Achillion announces positive POC data for ACH-3102...

Posted 9-27-12 on the Achillion website. Achillion announces positive proof-of-concept data with it's second generation pan-genotypic NS5A inhibitor, ACH-3102. A single-dose of ACH-3102 in GT1a resulted in a mean maximum 3.74 log10 reduction in HCV RNA (range 2.9 - 4.6 log10). In addition, ACH-3102 looks to have a unique resistance profile and generally is well-tolerated.  Two patients in the proof-of-concept trial were found to have baseline resistance mutations common to the first generation HCV NS5A inhibitor class - the L31M (patient had a maximum HCV RNA decline of 3.4 log10 with the 300mg dose) and Y93C mutation  (patient had a maximum HCV RNA decline of 4.6 log10 with the 300mg dose). Data in two patients with resistance mutations with a single-dose of ACH-3102 can't nearly be labeled as definitive, but it has positive implications in terms of sequential therapy.  That Gilead's co-formulated GS-7977/GS-5885 tablet will likely be on the market well before ACH-3102, 'first rescue' for GS-7977/GS-5885 failures would potentially be an attractive niche for ACH-3102. 

The company also expects results from it's Phase II trial looking at the interferon-free, all-oral regimen of ACH-3102 + RBV for 12  weeks in GT1b patients to be available in the 4th quarter of this year. 



September 27, 2012
Achillion Announces Positive Proof-of-Concept Data With ACH-3102

-Second-Generation Pan-Genotypic NS5A Inhibitor Achieves Potent Antiviral Activity
of Mean Maximum 3.74 Log10 Reduction Following a Single Dose -

- Initiated Enrollment in a Phase 2 Clinical Trial Evaluating ACH-3102 Plus Ribavirin for the Treatment of HCV Genotype 1b-

- Hosting Analyst Day Today With Live Webcast Beginning at 1:00 p.m. ET -

NEW HAVEN, Conn., Sept. 27, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced positive proof-of-concept results with ACH-3102, a second-generation pan-genotypic NS5A inhibitor being developed for the treatment of chronic hepatitis C viral infections (HCV). Administration of a single-dose of ACH-3102 to genotype (GT) 1a HCV-infected subjects resulted in a mean maximum 3.74 log10 reduction in HCV RNA (range 2.9 — 4.6 log10). Significant reductions in HCV RNA were achieved in subjects with resistant variants at baseline, including L31M and Y93C variants.

Based on these data, combined with safety and tolerability results from the Phase 1a trial in healthy subjects evaluating up to 14 days of ACH-3102, Achillion has initiated a pilot Phase 2 clinical trial evaluating ACH-3102 in combination with ribavirin for the treatment of patients with chronic GT 1b HCV.

"We believe these proof-of-concept results demonstrate the differentiation of ACH-3102 from first-generation NS5A inhibitors. The potency of ACH-3102 was successfully shown against genotype 1a, historically the hardest to treat HCV subtype," commented Michael Kishbauch, President and Chief Executive Officer of Achillion. "Furthermore, we believe the enhanced resistance profile of ACH-3102 observed in vitro has been validated in the clinic with robust antiviral activity against baseline mutations such as L31M. These results support our belief that this second-generation pan-genotypic NS5A inhibitor has the potential to become a cornerstone compound."

ACH-3102: Phase 1 Program

In May 2012, Achillion initiated a Phase 1a clinical trial evaluating the safety and tolerability of single and multiple ascending doses of ACH-3102 in healthy volunteers. To date, 42 healthy volunteers have received a single dose of ACH-3102, ranging from 25 mg to 1,000 mg. An additional 32 healthy volunteers have received 14 days of ACH-3102 once-daily evaluating various dosing regimens. Preliminary data from the single and multiple ascending dose groups demonstrated that ACH-3102 was well tolerated at all doses evaluated. There were no serious adverse events and no clinically significant changes in vital signs, electrocardiograms (ECGs), or laboratory evaluations. All reported adverse events were classified as mild or moderate and were transient in nature.

In August 2012, Achillion initiated a Phase 1b clinical trial enrolling a total of 14 patients infected with GT 1a chronic HCV, of which 2 received placebo and 12 received a single dose of 50 mg, 150 mg or 300 mg ACH-3102. No serious adverse events were reported and there were no patient discontinuations.

The mean maximum HCV RNA decline for each dose group is provided below:


      Mean maximal   Range decline
  Dose   decline HCV RVA HCV RNA
Genotype (mg) N (log10) (log10)
  50 4 3.78 3.35 — 4.16
1a 150 4 3.52 2.91 — 3.98
  300 4 3.93 3.40 — 4.60
  Placebo 2 0.72 --

An assessment of clinical virology was conducted on baseline samples from all 12 patients receiving a single-dose of ACH-3102. Sequencing revealed one patient had a baseline L31M mutation (300 mg dose group, maximum HCV RNA decline of 3.4 log10) and another patient had a baseline Y93C mutation (300 dose group, maximum HCV RNA decline of 4.6 log10). These mutations have been previously reported to convey a high level of resistance to first-generation NS5A inhibitors which was not observed following exposure to ACH-3102.

ACH-3102: All-oral, interferon-free pilot Phase 2 12-week trial of ACH-3102 and ribavirin for the treatment of HCV GT 1b

Achillion has initiated patient enrollment in an open-label Phase 2 pilot trial evaluating 12-weeks of once-daily ACH-3102 in combination with ribavirin for the treatment of HCV GT 1b. This study will initially enroll up to 16 treatment-naïve patients with GT 1b IL28B CC HCV. Patients will receive 225 mg of ACH-3102 on day 1 followed by 75 mg of ACH-3102 once daily on subsequent days in combination with twice daily ribavirin. The primary objective of the trial is to determine the safety and sustained virologic response 12 weeks after the completion of treatment (SVR12) with secondary endpoints assessing safety, pharmacokinetics, pharmacodynamics, and virologic endpoints including rapid virologic response (RVR) and extended RVR (eRVR). Achillion expects to report initial RVR results from this study during the fourth quarter of 2012.

Mr. Kishbauch further commented, "With the initiation of this all-oral 12-week study evaluating ACH-3102 and ribavirin for the treatment of HCV genotype 1b, we have rapidly advanced our portfolio and believe the attributes of ACH-3102, as well as sovaprevir, our Phase 2 protease inhibitor, have the potential to provide optimized compounds for the broad treatment of HCV."

Analyst Day Webcast

Achillion is hosting its inaugural Analyst Day and simultaneous webcast on Thursday, September 27, 2012 at 1:00 p.m. Eastern Time. To access a copy of the presentation and the live audio webcast of the event, please visit www.achillion.com. Please connect to Achillion's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. A replay of the webcast will be available on www.achillion.com beginning approximately 2 hours after the conclusion of the event.

About HCV

The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to the favorable activity and potential benefits of ACH-3102 and sovaprevir, and expectations about milestone achievement including the potential to report RVR results during the fourth quarter of 2012. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things, Achillion's ability to: replicate in later clinical trials the positive results found in nonclinical studies and earlier stage clinical studies of sovaprevir, ACH-2684, and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

CONTACT: Company Contact:

         Glenn Schulman

         Achillion Pharmaceuticals, Inc.

         Tel. (203) 624-7000

         gschulman@achillion.com



         Media:

         Christin Culotta Miller

         Ogilvy PR

         Tel. (646) 229-5178

         christin.miller@ogilvypr.com

       

         Investors:

         Mary Kay Fenton

         Achillion Pharmaceuticals, Inc.

         Tel. (203) 624-7000

         mfenton@achillion.com



         Investors:

         Seth Lewis

         The Trout Group, LLC

         Tel. (646) 378-2952

         slewis@troutgroup.com

Achillion Pharmaceuticals HCV pipeline suddenly looking attractive...

Posted 8/29/12 on Business Week.com. Achillion Pharmaceuticals, once regarded as an also ran by Big Pharma suddenly looks sexy again as clinical holds and pipeline failures stymie drug development with Idenix and BMS. Rumors of a takeover has made Achillion stock surge in the past couple of days. The HCV drug development space continues to be one of the most dynamic in pharma, there is definitely no shortage of spills and thrills. 

Achillion Deal Looming as Hepatitis Drugs Fail: Real M&A

By Ryan Flinn and Will Robinson on August 29, 2012

Achillion Pharmaceuticals Inc. (ACHN), the developer of hepatitis C treatments that was passed over by potential acquirers in the last year, is poised to draw renewed interest after setbacks by rival drugmakers.

Bristol-Myers Squibb Co. (BMY) last week said it was abandoning an experimental hepatitis C pill it obtained through its February purchase of Inhibitex Inc. after one patient died and others were hospitalized while taking the drug in a study. This week, Idenix Pharmaceuticals Inc. said U.S. regulators halted its study of a similar therapy, marking the second hold on clinical trials for the company this month.

With the market for new hepatitis C treatments projected to reach $20 billion by 2020 and Achillion facing no delays in two drugs under development, Piper Jaffray Cos. and William Blair & Co. say the $481 million company could gain fresh attention as a takeover candidate for Merck & Co. (MRK), Roche (ROG) Holding AG and Vertex Pharmaceuticals Inc. (VRTX) A suitor could pay a premium of as much as 79 percent to Achillion’s stock price and still acquire the New Haven, Connecticut-based company for less than its peak market value earlier this year, when takeovers and merger speculation spurred a surge in hepatitis C drugmakers’ shares.

“The frenzy has been taken out of the space, but I still think Achillion is very attractive” because its therapies have the potential to be the best of their type, Ted Tenthoff, a New York-based analyst for Piper Jaffray, said in a telephone interview. “We expect the wave of consolidation to continue. Achillion is clearly a target.”

Drug Development
Joe Truitt, Achillion’s chief commercial officer, said it wasn’t appropriate to comment on the company’s development plans, including the possibility of a takeover.

“We’ll make the best strategic options as they come to us, but for right now, we’re developing our drugs and getting them into combinations and making them available to patients,” Truitt said in a phone interview.

Today, shares of Achillion rose 3.5 percent to $6.86 at 9:45 a.m. in New York, the second-biggest gain among 116 stocks in the Nasdaq Biotechnology Index.

Hepatitis C is a viral infection that can cause liver damage and is estimated to affect 180 million people worldwide, according to the National Institutes of Health. Rising deaths among so-called baby boomers from the infection prompted U.S. health officials to declare in May that all of those born from 1946 to 1964 are at risk and should be tested.

Achillion is among several companies racing to develop hepatitis C cures that would replace the standard year-long injectable treatment that can cause flu-like symptoms.

Four Classes
There are four new classes of drugs under development to cure hepatitis C. Each work in different ways to stop the virus from replicating, and can be effective against one or several subtypes of the disease.

Drugmakers such as Abbott Laboratories (ABT), Achillion, Bristol- Myers, Gilead Sciences Inc. (GILD), Merck and Vertex have been testing these therapies, either alone or together, with varying degrees of success. The promise of a market that Achillion Chief Executive Officer Michael Kishbauch estimates will grow to $20 billion by 2020 spurred at least three acquisitions since October.

The biggest deal was Gilead’s $10.8 billion acquisition of Pharmasset Inc., announced in November, which came a month after Roche agreed to buy Anadys Pharmaceuticals Inc. for about $230 million. Bristol-Myers followed in January by announcing its $2.5 billion purchase of Inhibitex.

Fresh Look
Achillion’s Kishbauch said in November that the company was in “advanced discussions” with potential partners or acquirers. Its shares then reached a five-year high of $12.38 in February on takeover speculation before falling (ACHN) 46 percent since then as no deal materialized.

Now, with Bristol-Myers stopping development of the drug it bought from Inhibitex, and Idenix (IDIX) halting testing of a similar therapy, Achillion could attract a fresh look from companies seeking hepatitis treatments to use on their own or in combination with their existing therapies, said Liisa Bayko, a Chicago-based analyst with JMP Securities LLC.

Achillion is testing two types of drugs. By combining several classes of these new hepatitis C drugs, doctors may be able to limit the virus’ ability to infect, mimicking the strategy that a decade earlier helped turn HIV from a killer disease to a controlled one.

During the first quarter, Achillion will be reporting on how effective its two therapies work in combination. Good data could entice competitors to bid, Bayko said.

‘Well-Positioned’
“By the first quarter of next year, we could be a very different company,” Achillion’s Truitt said. “If that combination data comes through, then we really have a commercially viable, competitive combination that will put everybody on notice.”

“We’re pretty optimistic for Achillion,” Bayko said in a phone interview. “They’ll be well-positioned to be a candidate to be taken out, because right now, there are very few options if you want to get involved in hep C, in terms of combinations that are more advanced that are still in clinical development.”

Bayko said that while she expects a suitor to wait for the data on the drugs before making an offer, Achillion still could fetch as much as $10 a share if a company bid for it now, 51 percent more than its closing price yesterday.

Piper Jaffray’s Tenthoff said Achillion could lure suitors such as Merck, Roche and Vertex as they seek to compete against Gilead, which is seen by analysts as having the most promising hepatitis C drug. Gilead is poised to start testing two of its therapies together in a single pill this year, putting it on track to request U.S. regulatory approval for the drug in 2014.

Gilead Bid
Ronald Rogers, a spokesman for Merck, said the company doesn’t comment on speculation when asked whether the Whitehouse Station, New Jersey-based drugmaker was interested in Achillion, while an e-mail to Basel, Switzerland-based Roche’s media relations office wasn’t returned. Megan Pace, a spokeswoman for Cambridge, Massachusetts-based Vertex, declined to comment.

Even Gilead could seek to acquire Achillion as a way to remove a potential competitor and bolster its position, said Peter Kolchinsky, co-founder and general partner at RA Capital Management LLC, which oversees $300 million, including Achillion shares.

“Gilead could solidify its supremacy if it had Achillion’s drugs, each best in its respective class based on what we know so far,” Kolchinsky said in an interview. “Acquiring Achillion would also be a wise defensive move for Gilead, keeping it from falling into a competitor’s hands or from becoming an independent low-cost competitor.”

Safety Concerns
Cara Miller, a spokeswoman for Foster City, California- based Gilead, said the company (GILD) doesn’t comment on market speculation.

Brian Skorney, an analyst with Brean Murray Carret & Co. in New York, says Achillion won’t be a takeover target soon because it has “a lot more to prove” with clinical data next year. Other companies that developed hepatitis C treatments like Pharmasset and Inhibitex proved their drugs were effective before they were bought, and the only remaining question about their products was safety, he said.

The safety problems that challenged the drug Bristol-Myers bought from Inhibitex and the regulatory holds that Idenix faces show how much risk is still left in the market for hepatitis C treatments, said Les Funtleyder, a fund manager focused on the health-care industry at New York-based Poliwogg.

“What’s that phrase, ‘Once burned, twice shy?’” Funtleyder said in a phone call. “If someone was to repeat what happened to Bristol, shareholders would start to ask questions about management’s judgment.”

Cheaper Now
Still, after the drop in Achillion’s stock this year, a buyer would be taking on the risk of the therapies potentially failing at a lower price tag.

During the past 12 months, acquirers that announced deals for biomedical companies paid 65 percent more than the target’s average 20-day stock price in transactions greater than $500 million, according to data compiled by Bloomberg. A bidder for Achillion could offer a premium of as much 79 percent to yesterday’s stock price and still get the drugmaker for less than its record market value of $863 million in February.

The market for treating the viral infection is too big to be dominated by Gilead alone, so large drugmakers may have the appetite to acquire a company such as Idenix or Achillion once they produce sufficient data on the safety and effectiveness of their drugs, said Y. Katherine Xu, a New York-based analyst at William Blair. Kelly Barry, a spokeswoman for Cambridge, Massachusetts-based Idenix, didn’t return a voicemail message and e-mail sent after business hours about whether the company has been approached by suitors.

“Both Idenix and Achillion, their strategy is to sell themselves,” Xu said. “Timeline-wise, these two used to be similar, but now Achillion may be a little bit ahead.”

To contact the reporters on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net; Will Robinson in New York at wrobinson11@bloomberg.net.

To contact the editors responsible for this story: Sarah Rabil at srabil@bloomberg.net; Reg Gale at rgale5@bloomberg.net.

Achillion releases mid-stage data on sovaprevir; advance NS5A inhibtior...

Press release posted 8/7/2012 on Equities.com. I always root hard for Achillion Pharmaceuticals, the scrappy underdog from Connecticut, so it's good to hear that their once-a-day PI sovaprevir is advancing nicely through the clinic coupled with the start of enrollment for phase 1 trials with their pan-genotypic NS5A inhibitor ACH-3102. Actually, given the recent, untimely demise regarding BMS' nuc BMS-094, it's good to hear about *anything* advancing in HCV drug development.  Hopefully Achillion will end up wagging their fingers in the faces of naysayers and develop a fully proprietary interferon-free anti-HCV combination. 

7:39 PM CDT, August 7, 2012


Achillion Announces Positive SVR4 Results From Phase 2 Study of Sovaprevir (Formerly ACH-1625) and Advancement of ACH-3102

Sovaprevir (formerly ACH-1625) achieves SVR4 of 85-100% of genotype 1 treatment naive patients treated with sovaprevir for 12 weeks followed by an additional 12 weeks of pegylated-interferon and ribavirin.

Enrollment of HCV-infected patients initiated in Phase 1 trial of ACH-3102, second generation pan-genotypic NS5A inhibitor

Conference call tomorrow August 8, 2012 at 10:00 a.m. EDT

NEW HAVEN, Conn., Aug. 7, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced sustained viral response (SVR4) results of 85 to 100 percent from an ongoing multi-dose Phase 2 trial evaluating 12 weeks of dosing with sovaprevir (formerly ACH-1625), a once-daily protease inhibitor, in combination with pegylated interferon plus ribavirin (P/R) followed by an additional 12 weeks of P/R. In addition, the Company announced today that ACH-3102, a second-generation pan-genotypic NS5A inhibitor, has been safe and well tolerated by healthy volunteers in both single and 14-day multiple ascending dose groups. Further, enrollment of patients in a Phase 1 proof-of-concept clinical trial to evaluate the safety and efficacy of ACH-3102 in patients with genotype 1 HCV has been initiated.

"We are very pleased to reach these important milestones in our HCV portfolio including positive SVR4 results with sovaprevir and the advancement of ACH-3102, our second-generation pan-genotypic NS5A inhibitor, through Phase 1 dose-escalation," commented Milind S. Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer. "The safety and efficacy seen with sovaprevir across dose groups provides us with confidence that this next-generation protease inhibitor will play an important role in an all-oral treatment for HCV. The safety and tolerability seen to date with ACH-3102, for which we expect to report proof-of-concept next month, combined with the compound's in vitro profile, lead us to we believe we have an in-house portfolio of optimized compounds that can successfully create an all-oral, interferon-free regimen for the treatment of genotype 1 HCV. We look forward to beginning combination studies with sovaprevir and ACH-3102 by the end of the year."

Sovaprevir: Updated Phase 2 results including SVR4
In June 2011, Achillion initiated a randomized Phase 2 trial evaluating three doses (200 mg, 400 mg, or 800 mg) of sovaprevir given once daily in combination with pegylated interferon plus ribavirin (P/R) for 12 weeks followed by an additional 12 or 36 weeks of P/R, for the treatment of genotype 1 HCV.

As previously reported in April 2012, of the 58 patients enrolled in this study, the majority had HCV genotype 1a (n=35 (60%)), with remaining patients having HCV genotype 1b (n=20) or genotype 1 (n=3). Approximately 71% of the patients were IL28B genotype CT/TT, the more difficult to treat mutation, 64% were male and 17% were African American. The complete early virologic responses (cEVR) across the 200 mg, 400 mg, and 800 mg sovaprevir dose groups were 100%, 94% and 100%, respectively.

Today, the Company reported SVR4 rates of 90%; 85%; and 100% in the 200 mg, 400 mg, and 800 mg dose groups, respectively, after 24 weeks of therapy consisting of 12 weeks of sovaprevir and P/R followed by additional 12 weeks of P/R. In all, 39 patients were assigned to receive an additional 12 weeks of P/R therapy with the remaining 14 patients assigned to receive an additional 36 weeks of P/R.

    Sovaprevir (ACH-1625) Virologic End Point 200 mg 400 mg 800 mg   n=19 n=20 n=19 RVR       (HCV RNA < 25IU/mL week 4) 79% (15/19) 89% (16/18)  90% (17/19) cEVR       (undetectable at week 12) 100% (18/18) + 94% (15/16) ++ 100% (19/19) Patients assigned to 12 weeks of sovaprevir and P/R followed by 12 weeks of P/R through week 12 12 13 14 Undetectable at end of treatment 100% (10/10) * 92% (12/13)  100% (14/14) SVR4       (undetectable at week 28) 90% (9/10) 85% (11/13) 100% (13/13)** + One patient withdrew for AE deemed unrelated to sovaprevir. ++ One patient withdrew consent, one patient moved, both undetectable at the time of withdrawal; two patients withdrew for AEs deemed unrelated to sovaprevir. * Two patients lost to follow-up, both undetectable at last assessment. ** One patient lost to follow up, undetectable at last assessment.

As previously reported, sovaprevir was generally well tolerated across all dose groups. Adverse events (AEs) in patients receiving sovaprevir were classified as mild to moderate and were transient. The most common AEs were consistent with P/R treatment.

Additional clinical trial results, including SVR4 and SVR12 for all patients treated with sovaprevir followed by an additional 12 weeks or 36 weeks of P/R, are expected be reported during the first quarter of 2013.

ACH-3102: Phase 1 trial in Healthy Volunteers and HCV-infected patients
In May 2012, Achillion initiated a Phase 1 clinical trial evaluating the safety and tolerability of single and multiple ascending doses of ACH-3102, a once daily, second-generation, pan-genotypic NS5A inhibitor, in healthy volunteers.

To date, 42 healthy volunteers have received a single dose of ACH-3102, ranging from 25 mg to 1,000 mg. An additional 24 healthy volunteers have received 14 days of once daily ACH-3102, with doses ranging from 25 mg to 75 mg. Preliminary data from both the single and multiple ascending dose groups demonstrated that ACH-3102 was well tolerated at all doses evaluated. There were no serious adverse events, no clinically significant changes in vital signs, electrocardiograms (ECGs), or laboratory evaluations. All reported adverse events were classified as mild or moderate, and were transient in nature.

Achillion announced today the initiation of enrollment of patients with genotype 1 HCV into a Phase 1 study to evaluate the safety, tolerability and antiviral activity of ACH-3102. The trial will initially evaluate the safety and antiviral activity of a single dose of ACH-3102. Initial results are expected to be reported during the third quarter of 2012.

Conference Call
Achillion will host a conference call and simultaneous webcast on Wednesday, August 8, 2012 at 10:00 a.m. EDT. To participate in the conference call, please dial (877) 266-0482 in the U.S. or (631) 291-4565 for international callers. The conference call ID is 13643523. A live audio webcast of the call will be accessible at www.achillion.com, under the News Center section of the website. Please connect to Achillion's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
A replay of the webcast will be available on www.achillion.com. Alternatively, a replay of the conference call will be available starting at 1:00 p.m. EDT on August 8, 2012, through 11:59 p.m. Eastern time on August 15, 2012 by dialing (800) 585-8367 or (404) 537-3406. The replay passcode is 13643523.
About HCV

The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.
About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to the expected safety, efficacy and potential benefits of sovaprevir, expectations about milestone achievement including the potential to achieve proof-of-concept for ACH-3102 and initiation of all-oral, interferon-free clinical trials evaluating regimens containing sovaprevir (ACH-1625) and ACH-3102 for the treatment of HCV. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage nonclinical studies and clinical trials of its drug candidates, including ACH-1625 and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

CONTACT: Company Contact: Glenn Schulman Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 gschulman@achillion.com Media: Christin Culotta Miller Ogilvy PR Tel. (646) 229-5178 christin.miller@ogilvypr.com Investors: Mary Kay Fenton Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 mfenton@achillion.com Investors: Seth Lewis The Trout Group, LLC Tel. (646) 378-2952 slewis@troutgroup.com Source: Achillion Pharmaceuticals, Inc.

Achillion Pharmaceuticals names ex-Vertex CCO to it's board of directors...

Posted on 6-11-12 on MarketWatch.com. True HCV Drug Development nerds will recognize Kurt Grave's name from his tenure as Executive Vice President, Head of Corporate and Strategic Development and Chief Commercial Officer at Vertex Pharmaceuticals from 2007 to 2009 - no easy task to take a small, primarily R&D company and ready it for commercialization. His tenure abruptly ended at Vertex as he and then-CEO Matthew Emmens butted heads. Some of you might argue with me, but it seems like Vertex could use Grave's leadership skills given the company's current level of dysfunction and dissatisfaction with it's current leadership.  Mr. Graves replacing Nicholas Simon on the Achillion board could also be interpreted in multiple ways, following the leadership shakeup when CMO Elizabeth Olek resigned last week.

PRESS RELEASE
June 11, 2012, 4:03 p.m. EDT
Achillion Appoints Kurt Graves to Its Board of Directors

NEW HAVEN, Conn., Jun 11, 2012 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. ACHN +0.65% today announced the appointment of Kurt Graves to its board of directors as an independent director. Mr. Graves brings more than two decades of experience in building, leading and advising top-performing pharmaceutical and biotechnology companies. Mr. Graves was appointed as a Class I director, with a term expiring in 2013, and will replace Nicholas Simon of Clarus Ventures LLC.

"We are very pleased to have Kurt join the Board of Directors, as his extensive strategic knowledge of hepatitis C and successful career in transforming pharmaceutical companies complements the expertise of the entire Achillion Board," commented David I. Scheer, Chairman of the Board of Achillion. "From his tenures at Merck, Novartis and Vertex Pharmaceuticals, Kurt brings a depth of industry expertise and a global business view that will serve us well as we move toward achieving a number of aggressive milestones including the initiation of all-oral, interferon-free clinical trials evaluating regimens containing our pan-genotypic protease inhibitor, ACH-1625, and NS5A inhibitor, ACH-3102, for the treatment of HCV."

Michael Kishbauch, President and Chief Executive Officer of Achillion further commented, "On behalf of the Board of Directors, I would also like to thank Nick Simon for his insight and guidance over the past three and a half years as Achillion matured into a clinical-stage pharmaceutical company, with a portfolio of proprietary hepatitis C compounds discovered by Achillion and advanced into clinical development."

Mr. Graves currently serves as Chairman, President and Chief Executive Officer of Intarcia Therapeutics. He is also Chairman of the Board of Radius, since April 2011, and a Director of Pulmatrix and Springleaf Therapeutics, since May 2010. In addition to his global experience in large pharmaceutical companies, Mr. Graves has played key leadership roles in building two highly successful early stage companies; at Astra Merck Pharmaceuticals and Vertex Pharmaceuticals. Mr. Graves was EVP, Head of Corporate and Strategic Development and Chief Commercial Officer at Vertex Pharmaceuticals from 2007 through 2009. Prior to his tenure at Vertex, he spent nearly ten years at Novartis Pharmaceuticals in senior leadership positions of increasing responsibility, and was most recently Global Head of the General Medicines Business Unit & Chief Marketing Officer for the Pharmaceuticals division. Prior to Novartis, Mr. Graves held commercial and general management positions of increasing responsibility at Merck and Astra Merck Pharmaceuticals where he led the GI Business Unit, with responsibility for Prilosec and Nexium.

Mr. Graves earned his B.S. in Biology from Hillsdale College and has attended executive leadership programs at Harvard, Wharton School of Management and University of Michigan.

About HCV

The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to the expected contributions of Mr. Graves to the Achillion Board of Directors and Achillion's expectations with respect to milestone achievement, and, in particular, its plans to initiate all-oral, interferon-free clinical trials evaluating regimens containing ACH-1625 and ACH-3102 for the treatment of HCV. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage nonclinical studies and clinical trials of ACH-2684, ACH-1625 and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Achillion Pharmaceuticals, Inc.



        CONTACT: Company Contact:
        Glenn Schulman
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        gschulman@achillion.com
        Investors:
        Mary Kay Fenton
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        mfenton@achillion.com
        Media:
        Christin Culotta Miller
        Ogilvy PR
        Tel. (212) 880-5264
        Christin.Miller@Ogilvy.com

Achillion Announces Resignation of Chief Medical Officer...

Press release posted 6/6/2012 on Market Watch.com:  

Achillion Announces Resignation of Chief Medical Officer

NEW HAVEN, Conn., Jun 6, 2012 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. ACHN +2.86% today announced the resignation of Elizabeth A. Olek, D.O., Senior Vice President of Clinical Development and Chief Medical Officer, effective June 18, 2012. Milind Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer, will assume responsibility for the oversight of ongoing and planned clinical trials evaluating Achillion's portfolio of compounds for the treatment of hepatitis C (HCV).

"Achillion's clinical programs have matured extensively during Liz's tenure, and during that time, our robust pipeline of HCV compounds, focused on pan-genotypic protease inhibitors and NS5A inhibitors, has advanced from discovery into early and mid-stage clinical trials. We thank Liz for her significant contributions to that development progress and wish her the best of luck in her future endeavors," said Michael D. Kishbauch, President and Chief Executive Officer of Achillion.

Mr. Kishbauch further commented, "As we move toward initiating all-oral clinical trials evaluating the combination of our protease inhibitor, ACH-1625, and our second-generation NS5A inhibitor, ACH-3102, later this year, we are confident that our development plans will remain on track by leveraging the talents of Dr. Deshpande, as well as physicians on both our staff and on our Board of Directors, and we plan to continue to achieve our aggressive milestones."

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to: Achillion's plans to initiate all-oral clinical trials evaluating the combination of ACH-1625 and ACH-3102, its beliefs that its development plans will remain on track by leveraging the talents of Dr. Deshpande and others, and its plan to continue to achieve its aggressive milestones. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage preclinical studies and clinical trials of ACH-1625 and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; retain key employees; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Achillion Pharmaceuticals, Inc.



        CONTACT: Company Contact:
        Glenn Schulman
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        gschulman@achillion.com
        Investors:
        Mary Kay Fenton
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        mfenton@achillion.com
     


(C) Copyright 2010 GlobeNewswire, Inc. All rights reserved.

Achillion discloses additional data on HCV protease inhibitor ACH-2684

Posted on 5/21/12 via MarketWatch.com. Look what happens when I go away for a week. The CDC changes it's guidelines to include one-time HCV testing for Baby Boomers (FYI, HCV drug developers had a hand in shaping this shift in public policy via the Viral Hepatitis Action Coalition for all you fans of government-corporate relationships) and Achillion continues to shore up their pipeline with decent drugs. OK, so a 3.73 log10 drop isn't exactly Telaprevir-like, but given the right backbone regimen - hopefully one also developed and manufactured by this currently unpartnered company - Achillion could hit it big. 

Achillion Announces Additional Proof-of-Concept Data With ACH-2684 for the Treatment of Hepatitis C

Achieves Up to 3.73 log10 Reduction in Genotype 1 HCV RNA After Three Days of Treatment With Once-Daily 400 mg ACH-2684 in Phase 1b Study

NEW HAVEN, Conn., May 21, 2012 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. ACHN +10.92% today reported proof-of-concept data from a Phase 1b clinical trial demonstrating that patients with chronic hepatitis C (HCV) genotype 1 (GT 1) treated with ACH-2684, a second-generation protease inhibitor, achieved a mean maximum 3.73 log10 reduction in HCV RNA after three-day 400 mg monotherapy with once-daily (QD) dosing. The compound also demonstrated good safety and tolerability both in healthy volunteers and in patients with HCV.

"We believe ACH-2684, with its potent antiviral activity achieved without boosting and once-daily dosing, is one of the most intriguing protease inhibitors in clinical development for the treatment of HCV," commented Michael D. Kishbauch, President and Chief Executive Officer of Achillion. "As we continue to focus our efforts on advancing our internally developed all-oral, interferon-free HCV regimen consisting of ACH-1625, our next generation protease inhibitor, in combination with ACH-3102, a second generation pan-genotypic NS5A inhibitor, we believe that the profile of ACH-2684 provides a significant strategic and therapeutic option for potential combinations with either ACH-3102 or other direct-acting antiviral agents."

ACH-2684 Phase 1 Clinical Trial

Achillion is evaluating ACH-2684 in a Phase 1 randomized, double-blind, placebo-controlled clinical trial to investigate the safety, tolerability, pharmacokinetic profile and antiviral activity in healthy subjects and HCV-infected patients. The trial consists of three segments: Phase 1a assessment of single ascending oral doses (SAD) in healthy volunteers, Phase 1a 14-day multiple ascending doses segment (MAD) in healthy volunteers, and Phase 1b evaluation of three days of oral repeat doses in subjects with GT 1 or 3 HCV. The MAD segment of this trial will be initiated during the second quarter and full trial results are expected to be presented at a medical meeting in the fourth quarter of 2012.

During the oral repeat doses segment in subjects infected with GT 1 HCV, a total of 30 patients were enrolled including 6 patients receiving placebo and 24 patients treated with three doses of 100 mg once daily (n=8), 400 mg once daily (n=8), or 400 mg twice daily (n=8) of ACH-2684. No serious adverse events (SAE) were reported and there were no patient discontinuations during treatment. The mean maximum GT 1 HCV RNA decline during therapy for the 100 mg QD, 400 mg QD, and 400 mg BID groups were 3.36 log10, 3.73 log10, and 4.16 log10, respectively, compared to a 0.68 log10 decline for patients receiving placebo. There were no viral breakthroughs observed during ACH-2684 monotherapy. Additional assessments of GT 3 activity are currently under development.

Safety data from the SAD and HCV-infected segments of the trial demonstrated ACH-2684 was well tolerated at all doses evaluated up to and including the maximum total daily dose of 1400 mg. There were no serious adverse events, no clinically significant changes in vital signs, electrocardiograms (ECGs), or laboratory evaluations. All reported adverse events were classified as mild or moderate, and were transient in nature. Dosing in the 14-day MAD segment is being initiated during the second quarter with longer term safety data expected to be available in the third quarter of 2012.

About ACH-2684

ACH-2684 is a next-generation HCV protease inhibitor designed and synthesized based on crystal structures of enzyme/inhibitor complex. ACH-2684 is a macro-cyclic, non-covalent, reversible inhibitor of NS3 protease. In preclinical studies, ACH-2684 demonstrated pico-molar potency, excellent pharmacokinetic properties and safety profile at high drug exposures. ACH-2684 also exhibits rapid and extensive partitioning to the liver, as well as high liver/plasma ratios in preclinical studies. ACH-2684 has shown pico-molar potency against NS3 protease that is specific to HCV. It has preclinical activity against the 6 known genotypes of HCV and exhibits equipotent activity against HCV genotypes 1a and 1b at an IC50 of approximately 100 pico-molar. The drug candidate was discovered internally and is being advanced by Achillion.

About HCV

The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to: the potency, safety, tolerability, effectiveness and other characteristics of Achillion's ACH-2684, ACH-1625 and ACH-3102; Achillion's expectations regarding timing for the commencement, completion and reporting of results of clinical trials of drug candidates including ACH-2684, ACH-1625 and ACH-3102; and Achillion's ability to advance a potentially best-in-class all-oral, interferon-free combination of ACH-1625 and ACH-3102 and ACH-2684 in combination with ACH-3102 or other direct acting antiviral agents. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage nonclinical studies and clinical trials of ACH-2684, ACH-1625 and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Achillion Pharmaceuticals, Inc.



        CONTACT: Company Contact:
        Glenn Schulman
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        gschulman@achillion.com
        Investors:
        Mary Kay Fenton
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        mfenton@achillion.com
        Media:
        Christin Culotta Miller
        Ogilvy PR
        Tel. (212) 880-5264
        Christin.Miller@Ogilvy.com

Achillion starts dosing of Phase 1 trial with 2nd generation NS5A inhibitor...

Posted on 5-9-2012 via MarketWatch.com. The folks at Achillion Pharmaceuticals are happy to announce that ACH-3102, their 2nd generation pan-genotypic NS5A inhibitor, began dosing in a 96 patient Phase 1 clinical trial. ACH-3102 boasts a unique resistance profile and QD dosing. Ultimately, the company would like to pair ACH-3102 with it's HCV protease inhibitor ACH-1625 currently in Phase II clinical trials. 

PRESS RELEASE
May 9, 2012, 7:00 a.m. EDT
Achillion Advances Second Generation Pan-Genotypic NS5A Inhibitor, ACH-3102, Into Clinical Development

Structurally Distinct NS5A Inhibitor Displays Potent Preclinical Activity Against Commonly Observed Resistant Variants

NEW HAVEN, Conn., May 9, 2012 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. ACHN +4.62% today announced that it has begun dosing ACH-3102 in a Phase 1 clinical trial. ACH-3102 is Achillion's second generation pan-genotypic NS5A inhibitor being investigated for the treatment of chronic hepatitis C virus (HCV) infection.

ACH-3102 is a structurally distinct small molecule compound that has demonstrated potent inhibition of the NS5A protein across all genotypes of HCV in preclinical studies. Furthermore, the unique chemical structure of ACH-3102 has resulted in enhanced potency in vitro against resistant mutants that have emerged during clinical studies with first generation NS5A inhibitors.

The randomized, double-blind, placebo-controlled Phase 1 trial will enroll approximately 96 healthy volunteers in the U.S. to investigate the safety, tolerability and pharmacokinetic profile of ACH-3102. The trial will assess dosing in single and multiple ascending oral doses for up to 28 days.

"We believe that NS5A inhibitors, in combination with protease inhibitors, will play an integral role in achieving the goal of an all-oral interferon-free treatment regimen for all segments of the HCV infected patient population," commented Michael D. Kishbauch, President and Chief Executive Officer of Achillion. "With our continued focus on compounds with potentially best-in-class characteristics, including safety and efficacy, broad genotypic effect with once-daily dosing and enhanced resistance profiles, we hope to move ACH-3102 through Phase 1 for HCV-infected subjects and toward combination studies with ACH-1625, our Phase 2 protease inhibitor, during the third quarter of 2012."

About NS5A Inhibitors and ACH-3102

The NS5A protein is a clinically validated target that serves multiple functions at various stages of the HCV life cycle including involvement in virion production, interaction with host proteins and association with interferon-resistance. ACH-3102, Achillion's second generation NS5A inhibitor, has demonstrated potent activity against all HCV genotypes in vitro and in preclinical studies achieved additive to synergistic activity when combined with NS3 protease inhibitors, NS5B polymerase inhibitors, interferon and ribavirin. In preclinical studies, ACH-3102 demonstrated excellent potency, in the pico-molar range, against wild type HCV RNA replication, as well as potency against resistant mutants that have been identified in clinical studies.

About HCV

The hepatitis C virus is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide including more than 5 million people in the United States, more than twice as widespread as HIV. Three-fourths of the global HCV patient population is undiagnosed; it is a silent epidemic and a major global health threat. Chronic hepatitis, if left untreated, can lead to permanent liver damage that can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to: the potency, safety, tolerability, effectiveness and other characteristics of Achillion's ACH-1625 and ACH-3102; Achillion's expectations regarding timing for the commencement, completion and reporting of results of clinical trials of ACH-1625 and ACH-3102; and Achillion's ability to advance a potentially best-in-class all-oral, interferon-free combination of ACH-1625 and ACH-3102. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion's ability to: replicate in later clinical trials positive results found in earlier stage preclinical studies and clinical trials of ACH-1625 and ACH-3102; advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; obtain necessary regulatory approvals; obtain patent protection for its drug candidates and the freedom to operate under third party intellectual property; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2011 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Achillion Pharmaceuticals, Inc.



        CONTACT:  Company Contact:
        Glenn Schulman
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        gschulman@achillion.com
        Investors:
        Mary Kay Fenton
        Achillion Pharmaceuticals, Inc.
        Tel. (203) 624-7000
        mfenton@achillion.com
        Media:
        Christin Culotta Miller
        Ogilvy PR
        Tel. (212) 880-5264
        Christin.Miller@Ogilvy.com

Seeking Alpha - Achillion Will Soar Higher On New Hepatitis C Drug In 2013

Posted on 4/16/2012 on Seeking Alpha.com. I'm trying to figure this guy out. He makes some valid points on Achillion's PI ACH-1625, but says nothing about their NS5A inhibitors. Then he mentions GSK's Promacta (Eltrombobag) as a 'competitor' and a 'Hepatitis C drug', which doesn't do much for this particular person's street cred. Anyway, I like to root for Achillion and I like it when people say nice things about the company, so I'm including it here for your reading pleasure. 

Achillion Will Soar Higher On New Hepatitis C Drug In 2013

By Vatalyst - Seeking Alpha.com

April 12, 2012

Achillion Pharmaceuticals (ACHN) has risen 51% since last year, which is a surprising number for a development stage pharmaceutical industry with no FDA approved drugs on the market. The news has been hyping up Achillion's hepatitis C drug ACH-1625, which is only just now ending Phase 2 clinical trials. Along with ACH-1625, are four other hepatitis C drugs and one bacterial infection antibiotic, ACH-702. The hepatitis C drug pipeline is composed of some very unique drugs and mechanisms for treating hepatitis C.

Should these drugs gain FDA approval, there would be a major upside for the company to sell ACH-1625 state side and worldwide to nearly corner the market. However, ACH-1625 is only just finishing Phase 2 and releasing results. I always highly advise against investing in any company with promising drugs that have not been FDA approved.

The first reason is that Achillion is getting great news hype about ACH-1625, which is causing the stock price to increase very well. But, Achillion is now starting Phase 3 clinical trials and will not have any more news reports that would cause a stock rise in the next year. The second reason is that more often than not, drugs fail to gain FDA approval for hundreds of different reasons, including toxicity, efficacy, and potency. It is never safe to bet on a drug before it is FDA approved so do not invest until there are conclusive and positive phase 3 trial results released.

Finally, due to the fact that ACH-1625 is starting phase 3 trials in the beginning of 2012, we cannot expect to see the drug on the market until at least late 2013 or early 2014.

Phase 3 trials can take a year or longer, plus new drug application for the FDA takes a substantial amount of time. Therefore, I do not expect stock price to do much of anything until late 2013 at the earliest. If you are a long-term investor and already own stock with Achillion, sit tight until late 2013 when share price will see a major upside if the clinical results are positive. If you are short term, keep a close eye on the company until late 2013 and if the phase 3 results are positive then it is time to invest.

Achillion has a market cap of $773 million and a 52 week range of $4-$13. Achillion has a competitor in the hepatitis C drug Promacta by GlaxoSmithKline (GSK), which has a market capitalization of $223 billion, a 52-week range of $39-$47 and a price to earnings ratio of 27.45. Achillion's next competitor is a hepatitis C drug, TMC435, by Johnson & Johnson (JNJ) with a 52 week range of $59-$68, a price to earnings ratio of 18.74 and a market cap of $179 billion. The last competitor of Achillion are three hepatitis C drugs by Roche Holding AG (RHHBY.PK) with a price to earnings ratio of 14.3, a market cap of $145 billion and a 52-week range of $42-$43. For a company that has no FDA approved products on the market, Achillion seems to be in pretty good standing with its competitors.

Johnson & Johnson is set to be Achillion's biggest competitor. It currently has a hepatitis C drug, TMC435, entering Phase 3 trials. The results of the Phase 2 trials showed that the drug was effective, safe, and 83% of patients were able to discontinue treatment with TMC435 after 24 weeks. With the release of this positive clinical trial news, Johnson & Johnson is receiving equal amounts of positive hype over this hepatitis C drug as Achillion. It is possibly receiving even more hype when considering the other drugs currently in Phase 3 of the Johnson & Johnson pipeline. Share has stayed relatively the same over the past year with a nearly 10% increase. I feel that right now Achillion and Johnson & Johnson are going to mirror one another until the Phase 3 data is released. I recommend keeping an eye on both companies until then; once the data is released we will know which company has the better drug and which is worth investing in.

Roche Holding AG is currently developing three hepatitis C drugs in its pipeline. These drugs are all in Phase 2 testing and will not be ready for the market until late 2014 at the very earliest, most likely by 2016. This is assuming that the drugs even pass FDA regulation in the coming years. I see no real competition from Roche Holding AG in the next few years to hurt share price of Achillion, but Roche Holding AG would be a good company to keep an eye on and possibly invest in if ACH-1625 ends up failing FDA approval.

GlaxoSmithKline has developed an FDA approved drug, Promacta, used for the treatment of hepatitis C in compound with other treatments. I feel that this drug wont provide much competition for ACH-1625 should it become FDA approved. Promacta's main function is to increase blood platelet count in patients with hepatitis C while other drugs attack the hepatitis virus, whereas ACH-1625 directly treats hepatitis C with no need for compounding with other drugs. I feel that ACH-1625 would out-compete Promacta relatively easily and no reason to be wary of GlaxoSmithKline.

To sum up Achillion's stance on the market, it is too soon to be investing in ACH-1625. There is no current upside to raise share price substantially for at least the next year while the trials carry on. Considering Johnson & Johnson's position currently mirrors that of Achillion, it would be smart to let the research data of both company's respective drugs decide which is going to be the best bet in 2013. Lastly, I believe ACH-1625 will beat out GlaxoSmithKline's Promacta if it becomes FDA approved, so expect little competition on the market from GlaxoSmithKline.

Seeking Alpha - Achillion Pharmaceuticals: Hope, Hype, And Hep C

An article appearing 3/27/12 by Stephen Simpson on Seeking Alpha.com - one of the more level-headed articles appearing on that particular site in recent months, IMO (admittedly, coming from me, that might not mean much!). It's very hard to be prescient in regards to Achillion's future in HCV, but with a wholly-owned, advancing pipeline diversified among viral targets, it has to be considered at least somewhat of a target of companies like Merck, J&J and Vertex that need to fill holes in their pipeline or need compounds with synergistic activity. Mr. Simpson also gives a quick rundown of what's happening in the HCV drug development marketplace as well as some apocryphal statistics on HCV infection within South America that could feasibly boost the value of the HCV market considerably by 2016 over current thinking, in his estimation.   


Achillion Pharmaceuticals: Hope, Hype, And Hep C

By Stephen Simpson - Seeking Alpha

I've followed biotech for a long time now, and I have a hard time thinking of an example of another addressable market like hepatitis C (HCV) where investor interest has just exploded in the space of about a year. Like antisense, monoclonal antibodies, RNAi, genomics, stem cells and every other hot property in biotech, there has been no end of hope, hype, and hucksterism. Although HCV is likely to grow into a very financially significant drug target in the coming years, it's worth wondering just how much of the frenzy today can be justified in long-term valuations.

In particular, I'm curious about Achillion Pharmaceuticals (ACHN) these days. I've watched this stock for a while now and came close to buying on multiple times in the pre-2010 pre-$2 level. I underestimated just how fast this market would heat up, though, and had to re-learn a painful lesson - sometimes, you snooze and you lose.

Achillion jumps out as the only serious HCV play I'm aware of with a market cap below $1 billion (while Idenix (IDIX) is hardly a giant at $1.1 billion). Not only is it relatively small, but it also may be one of the relatively few players with its own home-grown effective combination therapy.

Of course everything Achillion is working on in HCV is still in the clinic and as Gilead (GILD) showed so clearly recently, "surprising" clinical results are not always positive surprises. The end result, then, is that this may be the next Pharmasset or just another biotech destined to flame out.

The Good - Interesting Compounds All Their Own

Achillion has at least two HCV antivirals well worth watching - ACH-1625 and ACH-3102.

ACH-1625 is a potentially pangenotypical NS3 protease inhibitor (PI) that has shown both solid efficacy and encouraging safety in early studies. Showing both strong efficacy and safety thus far, it could perhaps be the backbone for future combination therapies. That said, pangenotypical efficacy is not yet established and the compound still has not gone through a pivotal Phase 3 study.

The good news with antivirals is that, unlikely oncology and anti-inflammatory drugs, efficacy signals in early pilot studies often hold up through pivotal studies. What's more, these studies are relatively easy to enroll and can be completed expeditiously. What that all means for investors is that ACH-1625 could have a relatively quick path to market.

Achillion is also developing ACH-3102, a NS5a inhibitor, and expects to put it in human studies soon. This drug looks like it should be less subject to resistance and the company (as well as bulls) seem more excited about this drug than the more advanced ACH-2928 (also a NS5a inhibitor). Coupled with ACH-1625, this could be a very interesting combo therapy, but it's worth mentioning that almost every drug is interesting to bulls going into Phase 2 studies.

These aren't the only drugs in Achillion's HCV pipeline, and the company does have identified experimental compounds targeting bacterial infections and HIV. It should be noted too that Achillion's HCV pipeline is wholly-owned.

The Bad - Competition, And Expectations, Left Right And Center

Achillion is very definitely not going to be the first company to market with new HCV drugs, and there is apt to be extensive competition in the market. That is not only going to place a premium on the SVR efficacy data and safety profile, but also potentially on the marketing muscle of the company in question.

Since its acquisition of Pharmasset, Gilead was put in the pole position in the race to develop blockbuster HCV drugs. That is, until the company reported Phase 2 data on the superstar-to-be '7977 that showed it is not 100% perfect. Specifically, this Phase 2 study indicated that '7977 may not be effective in null responders (patients who have failed to respond to earlier treatments), and may give new hope to alternate approaches that include a NS5a inhibitor or NS3 protease inhibitor.

That said, a little perspective is in order. Maybe '7977 isn't flawless, but SVR-12 rates of 91% in genotype 1 and 100% in genotypes 2 and 3 in a thus far safe drug is still pretty impressive. Moreover, Gilead is looking at a variety of combination possibilities and while the company would certainly love to have "the one antiviral to rule them all", being a part of a blockbuster combination therapy is not a bad consolation prize.

Along Gilead and Achillion is a host of companies developing therapies and a sea of drugs known more by number than by name.

Bristol-Myers Squibb (BMY) has its NS5a inhibitor daclatasvir well along in studies, a PI ('032), and the NS5b inhibitor it acquired with the Inhibitex deal, as well as other earlier stage compounds. The NS5a inhibitor has shown solid efficacy, and its PI/NS5 combo showed 100% SVR without PEG-interferon or ribavarin.

Of course there are many more. Abbott (ABT) has its own all-oral combination (NS5a and PI), as does Roche (RHHBY.PK), although the efficacy in the Roche compounds has not been as encouraging. Johnson & Johnson (JNJ) has its TMC435 PI that it in-licensed from Medivir, and Boeringher Ingelheim has its PI as well. Indenix has a nucleotide inhibitor and NS5a inhibitor in trials and a non-nuc in preclinical development.

Last and not least are Vertex (VRTX) and Merck (MRK). These companies have brought the two newest HCV drugs to market (Incivek and Victrelis, respectively). Vertex has a PI in trials and two nucleotide inhibitors licensed in from Alios, while Merck's PI has seen a significant setback tied to safety issues.

A Quick Rundown On The Market, Prospects, And Deals

The oft-repeated statistic on HCV says that there are between 130 million and 170 million infected persons around the world. Approximately 4 million of those are in the U.S., with another 5 million the EU. By way of comparison, Brazil is thought to have at least 7 million HCV patients, India at least 10 million, and China 43 million.

Untreated HCV offer leads to severe chronic liver disease (including cirrhosis), but the long-used PEG-interferon and ribavirin therapy (dominated by Roche and Merck) has had success rates of below 50% in long-term usage.

That efficacy underlies a lot of the market expectation on HCV drugs. While the pre-Incivek/Victrelis HCV market was estimated to be about $3 billion (with $2.5 billion of that going to PEG-inteferon), analysts believe better efficacy could drive that figure close to $10 billion in 2016.

Admittedly, that's a large number. It may not be completely out of line, though, if these 90%+ SVR rates hold up in pivotal studies. Offer people with a serious lifelong illness a safer, more effective drug that is also easier to take and you can almost always charge a premium for it. Also, think about it this way - the U.S. and European insurance/payer systems are willing to pay several tens of thousands of dollars for cancer therapies that offer a few extra months of median survival benefit, so there's clearly a willingness to pay for better clinical outcomes.

What's this all mean for Achillion?

I could see ACH-1625 garnering perhaps up to a billion in sales if the early results hold all the way through studies. Moreover, there's the potential to be had from the combo therapy; whether that's in combination with another Achillion drug or a rival compound (like, say, Gilead's '7977). If Achillion could garner $1 billion in sales, that would translate into a fair value of $13 to $18 per share today based upon your forward multiple estimate (6x or 8x).

I'm sure $1 billion in sales will sound too small to Achillion bulls. Fair enough; if the early results hold up, maybe it's an even bigger blockbuster, but it looks like there are going to be a lot of competing therapies on the market and good marketing is going to deliver sales even in inferior compounds.

It may well be the case, though, that Achillion never makes it to the point where its sales matter. I would be quite surprised to see Achillion partner its drugs, but I think a buyout could well happen. Certainly there have been plenty of chatter on that subject, what with Gilead and Bristol-Myers paying up for HCV compounds (and Roche also making some deals of its own).

So how does the M&A scene break out? I don't think Bristol-Myers would need Achillion, and I don't think Gilead would go that route either unless a '7977/ACH-1625 really showed something special. Johnson & Johnson may think it needs non-PI partner compounds and may not want to pay for the overlap between their PI programs.

That said, I think there are a lot of names left that could be interested. Merck arguably needs a better protease inhibitor, and likewise Roche, Abbott, and Vertex may find that they need better compounds than they presently have. Companies like Sanofi (SNY) and GlaxoSmithKline (GSK) aren't doing much here today, but could find Achillion to be a ready-made HCV platform. Odds are, though, that Achillion holds out a bit for a desperate buyer - not because there's anything wrong with its pipeline, but because a desperate Merck or Roche may pony up a better deal if they feel pressure.

The Bottom Line

Taking a midpoint of that earlier $13-$18 range, I'd say Achillion may be worth about $15.50 a share today. Annoyingly, that's almost spot on with the current consensus number. I do believe there could be more upside as its pipeline matures and rivals run into problems, but I usually want more than 50% undervaluation before buying into a new biotech story.

Disclosure: I am long RHHBY.PK

Analysts weigh in on takeover odds of Achillion and Idenix...

Posted on Weds from Market Watch.com : It drives me nuts when 'nucs',  the popular shortened term for 'nucleoside analogs', is spelled 'n-u-k-e-s' instead. We're talking drug development, not nuclear arms proliferation in unstable Middle East nations. Probably a good indicator I need something else to take out my frustrations on. 

An article here from Market Watch talking to several analysts about possible mergers of Achillion and Idenix. No doubt that the slack of easing sales of Incivek and Victrelis will definitely be picked up by the 2nd and 3rd generation molecules, especially if developers can manage an interferon-free regimen. The HCV drug development space is incredibly hot right now, and with an estimated 170 million people infected, successful drugs are probable cash cows for many years to come.

Feb. 1, 2012, 12:01 a.m. EST

Achillion, Idenix could miss hep-C merger bonanza

By Val Brickates Kennedy, MarketWatch

BOSTON (MarketWatch) -- Investors tantalized by a recent string of lucrative takeover offers for hepatitis C drug-developers shouldn’t assume that an eye-popping bid for Idenix Pharmaceuticals and Achillion Pharmaceuticals is just around the corner, according to biotech analysts.

Both Idenix  were put in play earlier this month by news that Bristol-Myers Squibb intends to buy Inhibitex Inc for $2.5 billion in cash.The offer represents a dazzling 163% premium over Inhibitex’s pre-bid closing price.

Bristol’s bid also comes on the heels of two other lucrative takeovers in the hepatitis C virus, or HCV, arena. In October, Roche announced it was paying a stunning 256% premium, or $230 million, for tiny Anadys Pharmaceuticals. That was followed by Gilead Sciences’s whopping $11 bid for Pharmasset Inc., which carried an 89% premium.

Rumors have since swirled that other Big Pharma players are likewise eyeing the HCV space. And that speculation has helped push up Idenix shares by a hefty 80% and Achillion shares by 45% since the beginning of the year.

At stake is a market filled with a backlog of under-treated HCV patients that many analysts believe could reach $10 billion a year within the next five years.

But here’s the kicker -- because the new HCV drugs can actually cure the disease, their demand will likely drop over time after the backlog of patients is treated. Despite this, most analysts agree the market should be able to coast along at the $10 billion level for at least ten years. And because time is of the essence, companies with HCV drug candidates in mid-to-late stage development have been considered the hottest takeover targets.

Of the two companies, Idenix’s stock has seen the most action largely because its lead drug candidate hails from a highly-touted class of drugs called nucleotides, or “nukes.” Both Inhibitex’s and Pharmasset’s lead drug candidates are nukes, which is what made them particularly attractive acquisitions.

“I don’t think Idenix’s stock’s bid too high,” said Wedbush Securities analyst Duane Nash, who tracks Idenix. “But the caveat is that acquisitions generally take longer than most people anticipate.”
While Wedbush currently has Idenix’s fair market value listed at $15 a share, Nash believes that Idenix could fetch a takeout price of between $20 and $25 a share. The stock closed at $13.39 on Tuesday.

William Blair analyst Katherine Xu, meanwhile, said she believes Idenix’s current takeout range is probably between $15 and $20 a share. Xu currently has a price target of $10 on the stock.

Xu added that she could be raising her target into the mid-to-high teens if and when U.S. regulators give the green light to an Idenix’s clinical trial that has been placed on partial hold over safety concerns. The decision is expected within the next few weeks.

“I doubt people will take it out before the hold is removed,” she said.

But analysts also point out that Novartis AG’s roughly 30% equity stake in Idenix could hinder a takeover bid, especially as the Swiss pharmaceutical giant reportedly has options to some key drug candidates. Meanwhile, JMP Securities analyst Liisa Bayko thinks that investors have overvalued Idenix’s nuke drug candidate, which she says isn’t as potent as those being developed by Inhibitex and Pharmasset. Because of this, Bayko has a sell rating on the stock.

As for Achillion, the reason its shares haven’t been bid up as high as Idenix’s is largely because its lead drug candidate is a protease inhibitor, a class of drugs that includes Merck & Co.’s Victrelis and Vertex Pharmaceuticals’s Incivek, which were both launched last year. Several other drug developers already have protease inhibitors in their pipelines.

Xu said that while she currently has Achillion’s price target at $15 a share, its takeout range is probably between $15 and $20. The stock closed at $11.09 on Tuesday.

“I think it’s still undervalued at the moment,” she said.

Wells Fargo Securities analyst Brian Abrahams said that even though Achillion’s lead drug candidate isn’t a nucleotide, that doesn’t mean it isn’t an attractive acquisition target.

“Certainly nucleotides are an exciting class but they’re not the only class we believe will be used in HCV treatment,” said Abrahams, adding that doctors will be looking to use the drugs in combination to get the best results.

Achillion Pharmaceuticals updates investors on HCV protease inhibitor portfolio...

From RTT News: Short blurb on Achillion's HCV protease inhibitor portfolio - ACH-1625 and ACH-2684

Achillion Pharmaceuticals (ACHN) Rose Above Resistance At The Highs
1/10/2012 6:51 AM ET

(RTTNews) - Achillion Pharmaceuticals (ACHN: News ) reported new clinical trial results on its portfolio of protease inhibitors Monday morning. Based upon the results, the company is planning further exploration of ACH-1625 in combination with other oral antiviral agents for the treatment of all HCV genotypes and continues to evaluate ACH-2684 in a Phase 1 clinical trial.

Achillion Pharmaceuticals gapped open higher Monday and climbed further around the middle of the afternoon. The stock finished up by 1.80 at $9.72, with volume at a 6-month high. Achillion broke out of a 2 1/2 week range and set a new high for the year.

Achillion Pharmaceuticals announces preliminary proof-of-concept data with HCV NS5A inhibitor ACH-2928...

Looks like Achillion Pharmaceuticals' NS5A inhibitor is bearing fruit, with the company releasing three-day monotherapy proof-of-concept data for it's first generation NS5A inhibitor ACH-2928 as well as naming a second generation compound ACH-3102, which is slated for early trials in Q1/Q2 next year. CE0 Michael D. Kishbauch is aiming for "a proprietary interferon-free DAA combination regimen for the treatment of HCV within Achillion's pipeline." Looks like Achillion will have plenty to present at EASL 2012, as well as an intention to evaluate an all-oral comb including one of their protease inhibitors and an NS5A inhibitors.

Achillion Announces Preliminary Phase 1b Proof-of-Concept Data With ACH-2928 NS5A Inhibitor for the Treatment of Hepatitis C

Achieves 3.68 Log10 Reduction in HCV RNA After Three Days of Treatment

NEW HAVEN, Conn., Dec 5, 2011 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. ACHN +1.61% , a leader in the discovery and development of small molecule drugs to combat the most challenging infectious diseases, today reported proof-of-concept data from its Phase 1b clinical trial of ACH-2928, a first-generation NS5A inhibitor, demonstrating that patients treated with ACH-2928 achieved a mean maximum 3.68 log10 reduction in HCV RNA after three-day monotherapy of 60 mg once daily. The compound also demonstrated good safety and tolerability both in healthy volunteers and in patients with chronic hepatitis C (HCV).

ACH-2928, Achillion's first generation inhibitor of the NS5A protein, was discovered through the Company's NS5A inhibitor program. Achillion also recently nominated a second-generation NS5A inhibitor, ACH-3102, which is currently undergoing IND-enabling studies and is expected to be advanced into clinical trials during the first half of 2012.

"We believe NS5A inhibitors have emerged as an important component for an all-oral, direct acting antiviral (DAA) regimen," commented Michael D. Kishbauch, President and Chief Executive Officer of Achillion. "Furthermore, NS5A inhibitors, when combined with a protease inhibitor, have achieved sustained viral responses in clinical trials in tough to treat genotype 1 HCV populations. We believe this highlights the potential to form a proprietary interferon-free DAA combination regimen for the treatment of HCV within Achillion's pipeline."

ACH-2928 Phase 1 Program

In July 2011, Achillion initiated dosing in a randomized, double-blind, placebo-controlled phase 1a/1b clinical trial to investigate the safety, tolerability, pharmacokinetic profile and antiviral activity of ACH-2928. The trial consists of three segments: assessment of single ascending oral doses (SAD) in healthy volunteers, evaluation of 3 days of oral repeat doses in subjects with genotype 1a or 1b HCV, and a 5-day multiple ascending doses segment in healthy volunteers.

During the oral repeat doses segment in subjects infected with HCV, a total of 10 patients were enrolled with 2 patients (genotype 1a) receiving placebo and 8 patients (7 genotype 1a and 1 genotype 1b) treated with 3 doses of 60 mg ACH-2928 administered once daily. No serious adverse events (SAE) were reported and there were no patient discontinuations during treatment. The mean maximum HCV RNA decline during therapy was 3.68 log10 compared to a 0.54 log10 decline for patients receiving placebo. There were no viral breakthroughs observed during ACH-2928 monotherapy.

Preliminary data from the SAD trial segment demonstrated ACH-2928 was well tolerated at all doses evaluated up to and including the maximum dose of 500 mg. There were no serious adverse events, no clinically significant changes in vital signs, electrocardiograms (ECGs), or laboratory evaluations. All reported adverse events were classified as mild or moderate, and were transient in nature.

Based upon these preliminary results, the ongoing Phase 1 study will continue to evaluate the pharmacokinetic, pharmacodynamic, and antiviral profile of ACH-2928. These Phase 1 results have been submitted for presentation at a medical meeting being held during the second quarter of 2012. In parallel, Achillion is advancing its second generation NS5A inhibitor ACH-3102 through IND-enabling studies and the Company expects to initiate clinical development during the first half of 2012.

"As we continue to evaluate ACH-2928 in this Phase 1 study, we are also working rapidly to advance ACH-3102, which has shown in preclinical studies to possess the same potent activity against genotype 1a HCV as ACH-2928, as well as enhanced activity against resistant HCV mutants that have been observed in this patient population," stated Milind Deshpande, PhD, President of Research and Development and Chief Scientific Officer. "We believe these results validate our NS5A development program, and look forward to developing an all-oral combination for clinical evaluation that includes one of our protease inhibitors and an NS5A inhibitor next year."

About NS5A Inhibitors

The NS5A protein is a clinically validated target that serves multiple functions at various stages of the HCV life cycle including involvement in virion production, interaction with host proteins and association with interferon-resistance. Achillion's NS5A inhibitors, including ACH-2928 and ACH-3102, possess potent in vitro activity against all HCV genotypes and demonstrate, in preclinical studies, additive to synergistic activity when combined with NS3 protease inhibitors, NS5B polymerase inhibitors, and ribavirin. In preclinical studies, ACH-2928 and ACH-3102 have demonstrated excellent potency, in the pico-molar range, against HCV RNA replication, including potent activity against genotype 1a while ACH-3102 has been shown to possess enhanced activity against recognized genotype 1 resistant variants.

About HCV

The hepatitis C virus infects the liver and is the most common cause of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 170 million people are infected with HCV worldwide and The American Association of Liver Disease estimates that up to 80 percent of individuals become chronically infected following exposure to the virus. If left untreated, chronic hepatitis can lead to permanent liver damage, which can result in the development of liver cancer, liver failure or death. Few therapeutic options currently exist for the treatment of HCV infection. The current standard of care is limited by its specificity for certain types of HCV, significant side-effect profile, and injectable route of administration.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's proven discovery and development teams have advanced multiple product candidates with novel mechanisms of action. Achillion is focused on solutions for the most challenging problems in infectious disease including hepatitis C and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors, including statements with respect to the potency, safety and other characteristics of Achillion's NS5A inhibitors, which may not be duplicated in clinical studies, and Achillion's expectations regarding results, timing and duration of clinical trials and reporting of results from clinical trials of Achillion's NS5A inhibitors. Among the important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to: Achillion's ability to advance the development of its drug candidates under the timelines it anticipates in current and future clinical trials; to obtain patent protection for its drug candidates, and the freedom to operate under third party intellectual property; to establish commercial manufacturing arrangements and to identify, enter into and maintain collaboration agreements with appropriate third-parties; and to raise the substantial additional capital needed to achieve its business objectives. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any obligation to update any forward-looking statement, except as required by applicable law.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Achillion Pharmaceuticals, Inc.



CONTACT: Glenn Schulman
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
gschulman@achillion.com
Investors:
Mary Kay Fenton
Achillion Pharmaceuticals, Inc.
Tel. (203) 624-7000
mfenton@achillion.com
Media:
Christin Culotta Miller
Ogilvy PR
Tel. (212) 880-5264
christin.miller@ogilvy.com