Achillion Reports Interim Results on ACH-3102

Mon Jan 7, 2013 6:55am EST

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- Enrollment completed in pilot Phase 2a study of ACH-3102 and ribavirin for genotype 1b CC
patients -

- Up to 12 weeks of once daily ACH-3102 appears safe and well tolerated with no on-treatment
virologic breakthrough observed to date -

- Profile of ACH-3102 supportive of continued development for genotype 1b and in combination with
other direct-acting antivirals for the broad treatment of HCV -

NEW HAVEN, Conn., Jan. 7, 2013 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced that it has completed enrollment in a pilot Phase 2a trial evaluating ACH-3102, a
second-generation pan-genotypic NS5A inhibitor, in combination with ribavirin for the treatment of
patients with chronic genotype 1b (GT 1b) hepatitis C virus (HCV) infection. The initial cohort
enrolled eight patients with GT 1b HCV, IL28b genotype CC, that are receiving twelve weeks of
ACH-3102 once daily in combination with ribavirin.

To date, five patients have completed 4 weeks of treatment, including three patients who have
completed 12 weeks of treatment. ACH-3102 has been well-tolerated by all patients with no serious
adverse events, subject withdrawals, or on-treatment viral breakthrough reported to date.
Treatment with ACH-3102 has resulted in rapid reduction in HCV RNA accompanied by normalization of
liver enzymes.

"We are very pleased to see that the profile of ACH-3102 continues to exceed our expectations for
providing a truly improved barrier to resistance. As the first-ever clinical trial to evaluate a
NS5A inhibitor as a single direct-acting antiviral in combination with ribavirin, we are extremely
encouraged by these initial results that demonstrate rapid suppression of the HCV GT1b virus and a
well-tolerated safety profile through 12 weeks of therapy," commented Milind Deshpande, Ph.D.,
President of Research and Development and Chief Scientific Officer of Achillion. "As this data set
continues to mature, we look forward to reporting initial SVR results at a medical meeting in the
second quarter, and are planning to expand enrollment in the study to include non-CC GT 1b
treatment-naïve patients later this quarter pending regulatory discussions."

Interim Results of Phase 2a Pilot Study of ACH-3102 and ribavirin

Achillion initiated in September 2012 patient enrollment into this open-label Phase 2a pilot trial
evaluating 12 weeks of once daily dosing of ACH-3102 in combination with ribavirin for the
treatment of chronic HCV GT 1b infection. The study has enrolled 8 treatment-naïve patients with
HCV GT 1b IL28B CC genotype. Patients received 225 mg of ACH-3102 on day 1 followed by 75 mg of
ACH-3102 once daily on subsequent days in combination with 1000-1200 mg dose of ribavirin. The
primary objective of the trial is to determine the safety of this dosing regimen, and the
sustained virologic response 12 weeks after the completion of treatment (SVR12) with secondary
endpoints assessing pharmacokinetics, pharmacodynamics, and other virologic endpoints including
rapid virologic response (RVR) and end of treatment response (ETR).

To date, following up to 12 weeks of treatment with ACH-3102 in combination with ribavirin, there
have been no reported serious adverse events, no treatment discontinuations and no clinically
significant changes in vital signs, electrocardiograms (ECGs), or laboratory evaluations with the
exception of one subject with an anemia requiring a ribavirin dose reduction.

                                              Interim Results:                                                                             
                                              Virologic End Point                                                                          
 Total 8 subjects enrolled                    RVR*                                         ETR**                                        
                                              n = 5                                        n = 3                                        
 # of subjects                                4/5                                          3/3                                          
 (%)                                          (80%)                                         (100%)                                      
 RVR = HCV RNA < LLOQ (< 25 IU/mL) at week 4                                                                                              
 ETR = HCV RNA < LLOD (< 10 IU/mL) at week 12                                                                                             
 *RVR: 3 patients treatment ongoing and have not yet reached week 4.                                                                      
 3 subjects had HCV RNA undetectable at week 4 and 1 subject had HCV RNA undetectable at week 3, HCV RNA < 25 IU/ml (detectable           
  at week 4, and undetectable at weeks 5-12.                                                                                              
 **ETR: 5 patients treatment ongoing and have not yet reached week 12.                                                                    


Clinical virology evaluations are ongoing including baseline and on-treatment virologic
sequencing. ACH-3102 in this study demonstrates sustained antiviral activity even in subjects with
multiple baseline mutations in the NS5A protein known to be highly resistant to first-generation
NS5A inhibitors.

Michael D. Kishbauch, President and Chief Executive Officer of Achillion, commented, "Given the
impressive safety profile and single agent activity seen to date, we feel ACH-3102 is a highly
competitive compound for treating genotype 1b in combination with ribavirin and has emerged as a
best-in-class agent that can be combined in multiple regimens, including sovaprevir or other DAAs,
for the broad treatment of HCV."

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. ACH-3102 was
deemed to be safe and well-tolerated in Phase 1 development and achieved mean maximal reductions
in HCV RNA of 3.78 log10 after a single dose. ACH-3102 has been granted fast track designation by
the FDA and is currently being evaluated in Phase 2 for the treatment of GT 1b HCV.

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 and/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
http://www.globenewswire.com/newsroom/ctr?d=10017239&l=17&a=www.achillion.com&u=http%3A%2F%2Fwww.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 ACH-3102; the potential of ACH-3102 to be
a highly competitive and best-in-class compound for treating genotype 1b in combination with
ribavirin and in combination with multiple regimens, including with sovaprevir and other DAAs, for
the broad treatment of HCV; Achillion's expectations regarding timing for the commencement,
completion and reporting of results of clinical trials of ACH-3102; and Achillion's ability to
advance trials of 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: complete its current trail of ACH-3102 and ribavirin, and to obtain
favorable results from such trial; replicate in later clinical trials positive results found in
earlier stage preclinical studies and clinical trials of 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) 752-5510
         gschulman@achillion.com
         
         Media:
         Christin Culotta Miller
         Ogilvy PR
         Tel. (212) 880-5264
         christin.miller@ogilvy.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
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