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

Idera Pharmaceuticals reports positive antiviral results from phase 1 study for Hep C drug

Idera Pharmaceuticals (Nasdaq: IDRA) announced Monday positive preliminary results from a phase 1 study for IMO-2125, the company's investigational drug treatment for chronic hepatitis C infection.

Hep C is an infectious disease affecting the liver. It is often asymptomatic, but once established, chronic infection can progress to scarring of the liver. In some cases, people will go on to develop liver failure or other complications, including liver cancer.

During the 4-week dose-ranging trial, the IMO-2125 drug was tested in combination with ribavirin, in 60 patients with the virus. The results indicated a "substantial decline" in virus levels at two days after the first dose of IMO-2125, and after four weeks of treatment, the company said.

In addition, liver enzymes decreased during the treatment period and were within the normal range by the end of the fourth week in the majority of IMO-2125-treated patients.

"In this study, IMO-2125 plus ribavirin was well tolerated with no treatment-related discontinuations, and demonstrated substantial antiviral activity," said vice president of clinical development, Robert Arbeit.

Idera develops drug candidates that act by modulating immune responses through specific Toll-like Receptors (TLRs).

TLRs, a family of immune system receptors and the immune system's first line of defense, recognize pathogens, or infectious agents, and initiate an immune response.

IMO-2125 is designed to stimulate the immune system, causing the body to generate natural interferons and other antiviral cytokines, or protein regulating molecules.

The company said it is now planning a 12-week phase 2 trial of the drug, which it expects will determine optimal dosing and provide longer term safety and antiviral activity data. Recruitment for the study is anticipated to begin in the first quarter of next year.

Idera was up more than 7% on Monday morning, trading at $2.53 as of 11:30am EST

Pharmasset Initiates Dosing in a Combination Study of PSI-7977 and PSI-938 for Chronic Hepatitis C...

First study to look at a combination of of a purine and a pyrimidine nucleotide analog (PSI-938 and PSI-7977 respectively). Pharmasset believes the combination of two 'nucs' has a potential higher barrier to resistance, making it attractive to use in combo with other STAT-C/DAA drugs. The company plans a Phase II trail looking at the combination without interferon mid-2011 - Chris

- Phase 1 combination study of a pyrimidine (PSI-7977) and purine (PSI-938) nucleotide analog in patients with chronic hepatitis C

- Interim data expected in first quarter of 2011

PRINCETON, N.J., Nov. 30, 2010 /PRNewswire-FirstCall/ -- Pharmasset, Inc. (Nasdaq: VRUS) announced today that dosing has begun in Part 2 of a Phase 1 study. This is the first clinical study combining a purine (PSI-938) and a pyrimidine (PSI-7977) nucleotide analog for HCV, and is designed to evaluate once daily doses of PSI-7977 and PSI-938 in patients with HCV who have not been treated previously.

"We are excited to be initiating this combination study with two proprietary nucleotide analogs for HCV," stated Michelle Berrey, MD, MPH, Pharmasset's Chief Medical Officer. "Based on our in vitro data, we believe the combination of two nucleotide analogs could provide potent antiviral activity across multiple HCV genotypes and could also have a higher barrier to resistance compared to other DAA combinations. We believe nucleotide analogs have a number of key attributes that may make them ideal partners for other DAA combinations, in addition to a 'nuc-nuc' combination."

About the Phase 1 Trial

In Part 1 of the Phase 1 multiple ascending dose study of PSI-938, suppression of HCV RNA below the limit of detection (LOD, <15 IU/mL) was observed in over half of the patients who received PSI-938 at daily doses of 200 mg or 300 mg for seven days. Part 2 of the study is designed to evaluate the combination of PSI-938 and PSI-7977. The primary objective is to assess the safety, tolerability and pharmacokinetics of PSI-938 administered alone for 14 days, and in combination with PSI-7977 for 7 to 14 days. The secondary objective is to evaluate viral kinetics of HCV RNA during monotherapy and combination nucleotide dosing. Approximately forty patients are expected to be enrolled into four cohorts as follows:

* PSI-938 QD administered as monotherapy for 14 days, followed by;
* PSI-938 QD for 7 days followed by PSI-938 plus PSI-7977 QD for 7 days and
* PSI-7977 QD for 7 days followed by PSI-938 plus PSI-7977 QD for 7 days, followed by;
* PSI-938 plus PSI-7977 QD for 14 days


We expect to report preliminary results from Part 2 of this Phase 1 study during the first quarter of calendar year 2011. We also expect to initiate a Phase 2 study of PSI-938 in combination with PSI-7977 during mid-2011. This Phase 2 study proposes to explore durations of PSI-938 and PSI-7977 in interferon-free combinations with an SVR endpoint.