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

Investing Daily.com: Hepatitis C Drug Companies and All-Oral Treatment - A $20 Billion Market

Posted 8/28/12 on Investing Daily.com. Author and investor Jim Finks takes a look at the current HCV Drug Development space. Of note is the projected $20 billion worldwide HCV drug market which he feels will greatly be increased from the current $5 billion if drugs with better tolerability and efficacy make it to market, coupled with the benefit of interferon-free regimens for some patients.    

Hepatitis C Drug Companies and All-Oral Treatment: A $20 Billion Market

by JIM FINK on AUGUST 28, 2012
in STOCKS TO WATCH

Last week I wrote about 3 Stock Plungers and only liked the future prospects of one of the stocks: Idenix Pharmaceuticals (NasdaqGM: IDIX). My main reason for thinking a sustainable stock rebound is possible was because it’s a top-ten holding of value-investing legend Seth Klarman, who owns a 9.2% stake in the company (10 million shares) at an average purchase price of $8 per share (30% higher than the stock’s current price of $6.15). Klarman’s Baupost Group has bought shares in Idenix for five consecutive quarters.

This begged the question why Klarman is so enamored with Idenix. I couldn’t get an interview with Klarman to ask him and Baupost Group’s 2011 shareholder letter (dated Jan. 31, 2012) doesn’t mention Idenix — even though Klarman started buying the stock in Q2 2011. Consequently, I needed to research Idenix myself.

Hepatitis C Virus is a Global Killer
I discovered that the Idenix investment story is all about the market opportunity for treating Hepatitis C, a virus that infects the liver and is life-threatening – slowly destroying the liver over 20-30 years (resulting in cirrhosis or cancer). For those that are unsure, the liver is a vital organ that we cannot live without.

There is no vaccine for Hepatitis C, so the number of people who will get infected (e.g., sexual intercourse, tattoos, blood transfusions/dialysis, sharing dirty needles, mother/child) is predictable and not likely to go down. Furthermore, the body does not develop immunity from experiencing the disease, so people can get infected multiple times. In other words, the market demand for Hepatitis C drug cures is high and long-lasting. Below are some facts about Hepatitis C:

Worldwide, 180 million people are infected and 350,000 die each year.
In the U.S., 3.2 million people are infected, between 32,000 and 110,000 are newly infected each year, and 15,000 to 20,000 die each year.
75% of infections are curable, but up to 75% of all infected people don’t realize that they have the disease because they don’t look or feel sick – until it is too late.

Annual global sales of Hepatitis C drugs is currently $5 billion, but analysts project that new drug treatments with fewer side effects and oral application could generate a worldwide market opportunity of $20 billion.

The U.S. Centers for Disease Control and Prevention (CDC) recently recommended that all baby boomers born between 1946 and 1964 (76 million people aged 47 to 67) be tested for Hepatitis C infection.

Treating Hepatitis C with Interferon Injections is Unpleasant
Prior to 2011, the standard treatment for Hepatitis C was a combination of immune-boosting peginterferon injections – offered by Merck (NYSE: MRK) and Roche Holding AG (OTC Markets: RHHBY) — and ribavirin anti-viral pills (ribavirin patent has expired, so generic versions are available from several companies). Course of treatment was long at 48 weeks and costs $15,000 to $30,000, the cure rate was only 46% and there were severe side effects (e.g., flu-like symptoms, anemia, depression).

Treating Hepatitis C with Protease Inhibitors Is Better But Still Requires Interferon
Then, in May 2011, the Food & Drug Administration (FDA) approved two new Hepatitis C drugs called “protease inhibitors” that promise much higher cure rates (75%) and shorter treatment protocols (half as long at 24 weeks). Both of these drugs are taken orally in pill form but only work in conjunction with the standard peginterferon injections and ribavirin treatment. The two new drugs are:

Victrelis by Merck, which has a 66% cure rate, costs between $26,400 and $48,400 per course of treatment, and requires 12 pills per day.

Incivek by Vertex Pharmaceuticals (NasdaqGS: VRTX), which has a 79% cure rate, costs $49,200 per course of treatment, and requires 6 pills per day.

Based on the higher cure rate and simpler dosing from Incivek, it should come as no surprise that Incivek is outselling Victrelis 3-to-1.

Treating Hepatitis C with All-Oral Nucleotide Drugs is the Holy Grail

But Vertex’s dominance with Incivek could be short-lived because other drug companies are working on new Hepatitis C drugs called “nucleotides” that offer the holy grail of therapy: pill-only treatment that does not require the peginterferon/ribavirin injection albatross.

Perhaps in the forefront of all-oral Hepatitis C treatment is Gilead Sciences (NasdaqGS: GILD), which in November 2011 acquired biotech company Pharmhasset in a monstrous $10.8 billion all-cash deal. Pharmhasset’s Hepatitis C drug in Phase III trials is called PSI-7977 and could be on the market by 2014. According to Gilead, PSI-7977 is “way ahead of everybody else” and promises a cure in only 8-to-12 weeks of treatment. However, news in February that patients experience a relapse of Hepatitis C symptoms after stopping treatment with PSI-7977 caused Gilead’s stock price to suffer its largest one-day drop in 11 years. Since February, Gilead has recovered to all-time highs, which suggests investors have concluded that PSI-7977 will remain the core treatment for Hepatitis C – perhaps in combination with other drugs — despite the relapse issue.

Gilead’s drug may be best, but it won’t be the first all-oral treatment on the market because Roche Holdings is expected to offer an all-oral drug cocktail possibly consisting of setrobuvir (obtained in its acquisition of Anadys Pharmaceuticals in October 2011), danoprevir (purchased from InterMune (NasdaqGS: ITMN) in October 2010), and Merck’s Victrelis.

With Bristol-Myers Squibb’s (NYSE: BMY) abandonment of the Hepatitis C drug (BMS-986094) acquired from Inhibitex for $2.5 billion and the FDA clinical hold on Idenix’s Hepatitis C drug (IDX-19368) pending additional safety data, the path to riches in Hepatitis C treatment is proving to be a tortuous one. Other drug companies in the race for Hepatitis C drug treatments or vaccines include:

Achillion Pharmaceuticals (NasdaqGS: ACHN)
Abbott Laboratories (NYSE: ABT)
Dynavax Technologies (NasdaqCM: DVAX)
Peregrine Pharmaceuticals (NasdaqCM: PPHM)
Inovio Pharmaceuticals (NYSE: INO)
Medivir AB (OTC Markets: MVRBF) –  in partnership with Johnson & Johnson (NYSE: JNJ)

Achillion is a Prime Takeover Candidate
Of all of these companies, the best speculative buy right now may be Achillion. It is a pure-play on Hepatitis C with two promising drugs under development (one protease inhibitor and one NS5). There are no FDA clinical holds on Achillion’s drug pipeline like there are on Idenix and Achillion’s small market cap of $481 million makes the company an easily digestible acquisition for big boys like Gilead, Roche, Merck, or Abbott.

Furthermore, I have a soft spot for Achillion because it is located in New Haven, Connecticut (where I lived for four years) and is in partnership with my alma mater, Yale University. Good things happen to people and companies associated with a top-notch educational institution like Yale.

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 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.

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

Small Cap Network.com on Hepatitis C drug development....

The Hepatitis C Race Is On: MRK, VRTX, VRUS, ACHN, PFE, IDIX

By James E. Brumley

Published: September 21, 2011 10:24:49 AM PDT

The race for an effective hepatitis C treatment may not be as high-profile as, say efforts to find a cure for cancer or HIV. It's a bigger market than most may imagine though, judging from the number of companies doing R&D in the arena. Here's a quick look at the key one.

Merck & Co., Inc. (NYSE:MRK) has one of only two FDA-approved hepatitis C drugs. Its version is called Victrelis - a protease inhibitor. It was only approved in May, so there's no sales momentum yet. If the efficacy seen in Phase II testing is any clue though, it should be a revenue driver. Merck said two-thirds of patients receiving Victrelis in combination with other treatments had a strong virologic response in that the virus was no longer detected in the blood six months weeks after the treatment was stopped.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has the other FDA-approved hepatitis C treatment. Theirs is called Incivek - another protease inhibitor. Vertex's entry in the race seems even better than Merck's; nearly 80% of patients saw no sign of the irus in the blood 24 weeks after treatment.

Pharmasset, Inc. (NASDAQ:VRUS) is anything but a household name, and its drug is only in Phase II testing. But, PSI-7977 (a nucleoside polymerase inhibitor) is very promising. When used in combination with interferon and ribavirin, Pharmasset produces a 12-week sustained virological response rate of 91% for previously untreated hepatitis C patients.

Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) actually has several versions of its treatment currently anywhere from pre-clinical studies to Phase II trials. They're all built on the same basic platform though; all act as protease inhibitors. The underlying mechanism for these compounds is novel, targeting the NS4A protein of HCV. By inhibiting this target, Achillion Pharmaceuticals says the formation of a functional replicase complex (a key step in viral replication of the viral RNA genome) is blocked. ACH-1625 is the one to watch closely now.

Update/Correction: Achillion is exploring two distinct programs - protease inhibitors and NS5A inhibitors. The protease inhibitor program includes its lead Phase 2 compound ACH-1625, while the Phase 1 program is evaluating a novel pan-genotypic compound designated ACH-2684. The second class of compounds the company is developing are NS5A inhibitors, and is the backbone ACH-2928 (also in Phase 1 development).

Pfizer Inc. (NYSE:PFE) is in the hepatitis C race, but for a company of it size and financial backing, it's surprisingly lagging. Its polymerase inhibitor (PF-868554) is only in Phase II trials right now. Given the lateness of the effort and the fact that Pfizer has multiple partners on this front, it's hard to say HCV is a priority its going to push forward in a meaningful way.

Finally, Idenix Pharmaceuticals, Inc. (NASDAQ:IDIX) has a small army of hepatitis C treatment in the hopper (four in all, but they're all significantly different than one another). The one that's furthest along is IDX184, currently in Phase II, while work on IDX320 has been halted for the time being. Idenix Pharmaceuticals began 12-week trials of IDX184 in July of this year, and is expected to share interim results sometime in the fourth quarter. IDX184 has also been halted - albeit temporary - in the past by the FDA over safety concerns.

The hepatitis C market could be worth as much as $10 billion within five years, according to industry analysts. It affects tens of million of people worldwide every year.