Achillion Reports 100% SVR12 From Second Cohort of Patients in the Previously-Completed Six Week Phase 2 Trial Evaluating Odalasvir (ACH-3102) and Sofosbuvir for Genotype 1 HCV ("Proxy Study")

Achillion Reports 100% SVR12 From Second Cohort of Patients in the Previously-Completed Six Week Phase 2 Trial Evaluating Odalasvir (ACH-3102) and Sofosbuvir for Genotype 1 HCV ("Proxy Study")

- 100% SVR12 reported for all patients treated for six- (n=18) or eight-weeks (n=12) —
- Odalasvir (ACH-3102) is the subject of an exclusive, worldwide development and commercialization license granted to Janssen -

NEW HAVEN, Conn., Sept. 17, 2015 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced additional interim results from a Phase 2 study evaluating odalasvir (also known as ACH-3102), a NS5A inhibitor, in combination with sofosbuvir, without ribavirin, for either six or eight weeks of treatment in patients with treatment-naïve genotype 1 chronic hepatitis C virus (HCV) infection. Of the patients treated for six weeks in this cross-over cohort, 100 percent (n=6/6) remained HCV RNA undetectable twelve weeks after completing therapy (SVR12). Previously, Achillion reported results from this study including 100 percent SVR24 for the initial cohorts including 12 patients treated for eight weeks and 100 percent SVR24 for 12 patients treated for six weeks.

In May 2015, Achillion announced it had granted Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, an exclusive, worldwide license to develop and, upon regulatory approval, commercialize HCV products and regimens containing one or more of Achillion's HCV assets which include odalasvir, ACH-3422, and sovaprevir.

ACH-3102 - 017: Phase 2 pilot study evaluating six- and eight-weeks of treatment in combination with sofosbuvir for genotype 1 treatment-naïve HCV

Achillion conducted a Phase 2, open-label, randomized, partial-crossover study to evaluate the efficacy, safety, and tolerability of eight weeks or six weeks of odalasvir and sofosbuvir, a marketed nucleotide polymerase inhibitor, without ribavirin, in treatment-naïve genotype 1 HCV-infected patients. The primary objective of the study was determination of sustained viral response 12 weeks (SVR12) after the completion of therapy. Eighteen patients were initially enrolled, including six observational patients (group 1). Twelve patients completed eight weeks of treatment consisting of 50 mg of odalasvir and 400 mg of sofosbuvir administered once daily while observational patients received no drug during this phase of the trial. Ten of the 12 patients receiving eight weeks of treatment had genotype 1a HCV. At baseline, the median HCV RNA was 7.15 log10 (range 5.5 — 7.8 log10). Of the 12 patients, 100 percent achieved SVR24. Odalasvir and sofosbuvir were well tolerated with no significant adverse events, ECG findings, or lab abnormalities observed during treatment.

Following achievement of the pre-specified response rate of 100 percent, the six observational patients plus six additional patients (group 2) were enrolled and received six weeks of treatment consisting of 50 mg of odalasvir and 400 mg of sofosbuvir administered once daily. Median HCV RNA at baseline was 6.95 log10 (range 6.2 — 8.0 log10) and six patients had GT 1a HCV. Of the 12 patients, 100 percent achieved SVR24.

Six additional rollover patients (group 3), enrolled into the final cohort, also received six weeks of treatment consisting of 50 mg of odalasvir and 400 mg of sofosbuvir administered once daily. Baseline characteristics included five of six patients with genotype 1a HCV, four of six patients with non-CC IL28B (two patients with IL28B TT), and a median baseline HCV RNA of 6.32 log10 IU/ml (range 6.0 — 7.3 log10 IU/ml). In all, a total of 18 patients (group 2 and 3) received six weeks of treatment and all subjects, 100 percent, achieved SVR12.

About the Achillion Worldwide HCV Collaboration with Janssen

On May 19, 2015, Achillion announced it had granted Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, an exclusive, worldwide license to develop and, upon regulatory approval, commercialize HCV products and regimens containing one or more of Achillion's HCV assets which include odalasvir (ACH-3102), ACH-3422, and sovaprevir. A key objective of the collaboration is to develop a short-duration, highly effective, pan-genotypic, oral regimen for the treatment of HCV. Achillion announced on August 3, 2015 that Alios Biopharma Inc., part of the Janssen Pharmaceutical Companies (Janssen) had initiated a Phase 1 clinical trial to evaluate the potential effect of simeprevir and odalasvir on the pharmacokinetics of AL-335 in healthy volunteers. Janssen previously stated its goal of initiating Phase 3 development with a triple regimen for HCV by early 2017.

About HCV

The hepatitis C virus (HCV) is one of the most common causes of viral hepatitis, which is an inflammation of the liver. It is currently estimated that more than 150 million people are infected with HCV worldwide including more than 5 million people in the United States. Three-quarters 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.

About Achillion Pharmaceuticals

Achillion is seeking to apply its expertise in biology and structure-guided design and a deep understanding of patient and clinician needs to develop innovative treatment solutions aimed at improving patients' lives. Achillion believes that its scientific excellence, integrated capabilities and experienced team position it to successfully achieve its goal of advancing new products along the entire continuum from the bench to the patient. Achillion's pipeline is currently focused on small molecule therapeutics for infectious disease and complement-related diseases. www.achillion.com

Cautionary Note Regarding 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. Achillion may use words such as "expect," "anticipate," "project," "intend," "plan," "aim," "believe," "seek," " estimate," "can," "focus," "will," and "may" and similar expressions to identify such forward-looking statements. Among the important 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: comply with its obligations under and otherwise maintain its collaboration agreement with Janssen on the agreed upon terms; demonstrate, either alone or through its collaborators, the requisite safety, efficacy and combinability of its drug candidates, and advance the preclinical and clinical development of its drug candidates under the timelines it projects in current and future clinical trials; obtain and maintain necessary regulatory approvals; obtain and maintain 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 effectively and successfully; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. 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 year ended December 31, 2014, its quarterly report on Form 10-Q for the quarter ended June 30, 2015, 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 duty to update any forward-looking statement, except as required by applicable law.

Hepatitis C - Janssen starts a study to evaluate simeprevir and odalasvir on AL-335 pharmacokinetics

Medivir announces that Janssen has started a phase I study to evaluate the effect of simeprevir and odalasvir on AL-335 pharmacokinetics

Stockholm, Sweden — Medivir AB (Nasdaq Stockholm: MVIR) today announces that Alios Biopharma Inc., part of the Janssen Pharmaceutical Companies (Janssen) has started a phase I clinical trial to evaluate the potential effect of simeprevir and odalasvir (also known as ACH-3102), on the pharmacokinetics of AL-335 in healthy volunteers.

This phase I study is an open-label, two-group study of simeprevir, odalasvir, a hepatitis C virus (HCV) NS5A inhibitor, and of AL-335, a nucleotide-based HCV polymerase inhibitor. The primary objective of the study is to investigate the potential effect of simeprevir and odalasvir on the pharmacokinetics of AL-335 when administered in combination to healthy volunteers.

Approximately 150 million people are chronically infected with HCV globally*. When left untreated, HCV causes progressive liver disease in many of those who are chronically infected, and this can lead ultimately to cirrhosis, hepatocellular carcinoma and a requirement for liver transplantation. However, combinations of antiviral agents, including e.g. a protease inhibitor such as simeprevir, have shown the potential to be curative and convenient regimens for patients infected with HCV.

Further information about the study can be found at www.clinicaltrials.gov

For further information, please contact:
Ola Burmark, CFO Medivir AB, mobile: +46 (0)725-480 580.

Medivir is required under the Securities Markets Act to make the information in this press release public.
The information was submitted for publication at 8.30 CET on 3 August 2015.

About Simeprevir (OLYSIO®)
Simeprevir is an NS3/4A protease inhibitor jointly developed by Janssen Sciences Ireland UC and Medivir AB and indicated for the treatment of chronic hepatitis C infection as a component of a combination antiviral treatment regimen. Simeprevir efficacy has been established in HCV genotype 1 and HCV genotype 4 infected patients with compensated liver disease, including cirrhosis. Janssen is responsible for the global clinical development of simeprevir and has exclusive, worldwide marketing rights, except in the Nordic countries. Medivir AB retains marketing rights for simeprevir in these countries under the marketing authorization held by Janssen-Cilag International NV. In November 2013, simeprevir was approved by the U.S. Food & Drug Administration and, in May 2014, it was granted marketing authorisation by the European Commission. Subsequent marketing authorisations have followed in several other countries around the world. Indications vary by market.

About Medivir
Medivir is a research based pharmaceutical company with a research focus on infectious diseases and oncology. We have a leading competence within protease inhibitor design and nucleotide/nucleoside science and we are dedicated to develop innovative pharmaceuticals that meet great unmet medical need. Our commercial organization provides a growing portfolio of specialty care pharmaceuticals on the Nordic market. Medivir is listed on the Nasdaq Stockholm Mid Cap List.

*http://www.who.int/mediacentre/factsheets/fs164/en/

AASLD - Achillion to Present HCV Data on ACH-3102/Sofosbuvir and Preclinical Profile of ACH-3422

Achillion to Present Updated Clinical HCV Data on ACH-3102 and Preclinical Profile of ACH-3422 at the American Association for the Study of Liver Diseases (AASLD) Annual Meeting

- Late breaker poster presentation will feature updated SVR results from the Phase 2 trial of ACH-3102, NS5A inhibitor, plus sofosbuvir for the eight-week treatment of genotype 1 HCV -

- Three preclinical posters on ACH-3422, uridine-analog nucleotide prodrug, to be presented –

NEW HAVEN, Conn., Oct. 8, 2014 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) today announced four abstracts have been accepted for presentation at The Liver Meeting® 2014, the 65th Annual Meeting of The American Association for the Study of Liver Diseases (AASLD), in Boston, November 7 – 11.

A late breaker poster presentation will be made providing updated results, including SVR12, from an ongoing Phase 2 proxy study evaluating ACH-3102, Achillion's second-generation NS5A inhibitor, in combination with sofosbuvir, without ribavirin, for eight weeks of treatment in patients with treatment-naïve genotype 1 chronic hepatitis C virus (HCV) infection. This trial is currently dosing patients in a follow-on six-week treatment cohort.

Furthermore, three additional posters will be presented detailing the preclinical profile of ACH-3422, a uridine-analog nucleotide that continues to advance through its Phase 1 clinical development program.

David Apelian, M.D., Ph.D., Executive Vice Present and Chief Medical Officer, commented, "We are delighted that Achillion's ongoing research into HCV with ACH-3102 will be featured in a late breaker poster presentation during the 2014 Liver Meeting. Furthermore, as we work to complete the Phase 1 trial of ACH-3422, we are pleased to have the opportunity to present new preclinical research detailing the profile of ACH-3422."

Publication No. LB-23

Title: Interim sustained virologic response (SVR), safety and tolerability results of 8-week treatment with ACH-3102 and sofosbuvir in chronic hepatitis C (HCV), genotype-1 (GT-1), treatment-naïve patients: a Phase 2 "proxy" study

Authors: E. Gane, H. Kocinsky, C. Schwabe, et al.

Date/Time: Monday, November 10th, 8:00 a.m. - 5:30 p.m. ET.

Session: Late-Breaking Poster Session

Room: John B. Hynes Convention Center, Hall C

Publication No. 1978

Title: ACH-3422, a novel HCV NS5B RNA polymerase nucleotide inhibitor, demonstrates improved potency over sofosbuvir against HCV genotype-3 replicons in vitro

Authors: Y. Zhao, S. Podos, J. Fabrycki, et al.

Date/Time: Tuesday, November 11th, 8:00 a.m. – 12:00 p.m. ET.

Session: Hepatitis C: Preclinical Development Poster Session

Room: John B. Hynes Convention Center, Hall C

Publication No. 1982

Title: A novel approach to HCV resistance selection featuring short treatment duration and reduced interference from host cell adaptation

Authors: J. Fabrycki, Y. Zhao, D. Patel, et al.

Date/Time: Tuesday, November 11th, 8:00 a.m. – 12:00 p.m. ET.

Session: Hepatitis C: Preclinical Development Poster Session

Room: John B. Hynes Convention Center, Hall C

Publication No. 1985

Title: Antiviral activity and resistance emergence: combinations of the NS5B nucleotide inhibitor ACH-3422 with other antiviral agents in vitro

Authors: D. Patel, Y. Zhao, J. Fabrycki, et al.

Date/Time: Tuesday, November 11th, 8:00 a.m. – 12:00 p.m. ET.

Session: Hepatitis C: Preclinical Development Poster Session

Room: John B. Hynes Convention Center, Hall C

Additional information including the date and time of presentations are provided below. Reprints of the posters will be made available on the Company's website at www.achillion.com/resources and accessible after presentation at AASLD.

Hepatitis C-Achillion Begins Dosing In ACH-3422/ Initiated Phase 2 ACH-3102/Sofosbuvir trial

Achillion Advances ACH-3422, Uridine-Analog Nucleotide Inhibitor, Into Clinical Trial; Initiates Phase 2 Pilot Study With ACH-3102, NS5A Inhibitor, for HCV

April 30th, 2014

- Dosing Initiated in Phase 1 Study to Evaluate the Safety, Tolerability and Antiviral Activity of ACH-3422, NS5B Uridine-Analog Nucleotide Prodrug

- Initiated Phase 2 Study Evaluating ACH-3102, Second-Generation NS5A Inhibitor, With Sofosbuvir for 8 Weeks of Treatment or Less in Genotype 1 HCV Treatment-Naïve Patients

NEW HAVEN, Conn. (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced that is has begun dosing study participants with ACH-3422, Achillion's proprietary uridine-analog nucleotide inhibitor, in a Phase 1 clinical trial. ACH-3422 is being developed for use in combination regimens to treat chronic hepatitis C viral infection (HCV). Achillion also announced the initiation of dosing in a Phase 2 pilot study evaluating ACH-3102, Achillion's second-generation NS5A inhibitor, in combination with sofosbuvir for eight and potentially six weeks of treatment for patients with chronic genotype 1 treatment-naïve HCV.

"We believe that a nucleotide inhibitor and NS5A combination is the cornerstone for pan-genotypic commercially competitive regimens, having demonstrated high response rates and short duration of therapy. With the addition of a third direct-acting antiviral such as a protease inhibitor, we believe we can potentially shorten therapy to less than eight weeks," commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion.

ACH-3422: Phase 1 Study in Healthy Subjects and Proof-of-Concept in HCV-infected Patients

Achillion is conducting a Phase 1 randomized, double-blind, placebo-controlled trial to investigate the safety, tolerability, pharmacokinetics and antiviral activity of ACH-3422. Cohorts of healthy subjects will be enrolled at each dose level to receive a single-ascending dose followed by multiple-ascending doses for 14 days. At each dose level, patients with treatment-naïve genotype 1 HCV will receive 7 days of ACH-3422 to assess safety and antiviral activity. The starting dose in this trial will be 50 mg of ACH-3422 with the study expected to enroll a total of approximately 100 healthy volunteers and HCV-infected patients. Preliminary results, including safety and antiviral activity, are expected to be reported during the fall of 2014. This study is being conducted outside of the United States.

Dr. Deshpande further commented, "ACH-3422 has been rigorously evaluated in preclinical studies, which we believe support clinical advancement of ACH-3422. Preclinical data indicate that ACH-3422 has potency comparable to sofosbuvir against GT1 HCV, and has potency up to 7-fold higher against GT3 HCV. As we work to complete the healthy subject and HCV-infected patient cohorts in this ACH-3422 study, we are simultaneously exploring the characteristics of ACH-3102 in combination with sofosbuvir in a pilot trial that will evaluate an eight week or shorter treatment regimen and that we expect will be highly informative for initiation of combination studies of ACH-3422 and ACH-3102. We are eager to begin reporting preliminary results from these two programs starting late this summer and through the remainder of this year."

ACH-3102: Phase 2 Pilot Study Evaluating 8-week treatment in combination with sofosbuvir for genotype 1 treatment-naïve HCV

Achillion is conducting a Phase 2, open-label, randomized, partial-crossover study to evaluate the efficacy, safety, and tolerability of eight weeks or six weeks of ACH-3102 and sofosbuvir in treatment-naïve genotype 1 HCV-infected patients. The primary objective for the study is determination of sustained viral response 12 weeks (SVR12) after the completion of therapy. Twelve patients will be enrolled and receive eight weeks of treatment consisting of 50 mg of ACH-3102 and 400 mg of sofosbuvir administered once daily. The trial protocol also allows for the enrollment of additional HCV-infected patients who may be eligible to receive six weeks of treatment consisting of 50 mg of ACH-3102 and 400 mg of sofosbuvir administered once daily. Preliminary results from the eight-week treatment duration cohort are anticipated during the summer of 2014. This study is being conducted outside the United States.

David Apelian, M.D., Ph.D., Executive Vice President and Chief Medical Officer commented, "Our focus is to safely and expeditiously advance our all-oral regimens for the treatment of HCV. The initiation of our first clinical study with our nucleotide inhibitor ACH-3422 is an important milestone for the Achillion portfolio. We expect that evaluation of our NS5A inhibitor ACH-3102 in combination with sofosbuvir will provide significant insights for our ultimate use of ACH-3422 and ACH-3102 in combination. Furthermore, we believe the breadth of our portfolio, which includes our protease inhibitors, could enable us to potentially develop commercially-competitive regimens that can be safe, effective, ribavirin-free and that can be used for eight weeks or less to potentially cure HCV."

Read more here.....

EASL: Achillion Presents New Data on ACH-3102 to Treat Hepatitis C

Achillion Presents New Data on ACH-3102 to Treat Hepatitis C at the International Liver Congress

- Phase 1 Results Further Demonstrate Potency of ACH-3102 in Genotypes 1a and 1b and Against Resistant HCV-

- Study Shows ACH-3102 and Sovaprevir Can be Co-Administered Without Interaction -

- Total of Six Poster Presentations to be Made During EASL -

 

AMSTERDAM, The Netherlands, April 23, 2013 (GLOBE NEWSWIRE)

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)today announced the presentation of new data demonstrating the efficacy, safety, tolerability, and combinability of ACH-3102, a second-generation, pan-genotypic NS5A inhibitor, at the 48^th Annual Meeting of the European Association for the Study of the Liver (EASL) during the International Liver Congress 2013. ACH-3102 and sovaprevir are currently in Phase II combination development for the treatment of patients with chronic Hepatitis C infection (HCV). During the upcoming poster presentations, data will be reported that demonstrates ACH-3102 achieves a reduction in HCV RNA despite the presence of resistant variants. In addition, ACH-3102 demonstrates no drug-drug interaction with sovaprevir, Achillion's second-generation protease inhibitor.

Data highlights include:

Poster No. 876: A Second Generation NS5A Inhibitor, Demonstrates Potent Antiviral Activity in Patients with Genotype 1A HCV Infection Despite the Presence of Baseline NS5A-Resistant Variants. Friday, April 26, 2013: Poster Session — Category 08c: Viral Hepatitis C: Clinical (therapy).

• Treatment naïve patients with genotype 1 chronic HCV infection, patients received a single dose of ACH-3102 at various doses (Group 1: 50 mg, n=4; 150 mg, n=4; 300 mg, n=4 or placebo, n=2) or placebo and were evaluated for baseline NS5A-resistant viral variants, which was present in the majority of patients. Based on the robust anti-viral effect observed in Group 1, a lower dose (25 mg, n=6) of ACH-3102 or placebo (n=3) was studied in Group 2.
• All tested doses of ACH-3102 were safe and well-tolerated in subjects with chronic HCV Genotype 1 infection.
• Robust, rapid and sustained suppression of plasma HCV RNA levels was observed in all doses (mean reduction of 4.04 log₁₀, 3.78 log₁₀, 3.52 log₁₀, and 3.93 log₁₀ IU/mL for 25 mg, 50 mg, 150 mg and 300 mg doses, respectively, versus reduction of 0.68 log₁₀ IU/mL for placebo). Plasma HCV RNA levels sharply declined within the first two days post-treatment, and remained lower than baseline for at least 14 days.
• ACH-3102 retains potent anti-viral activity against both wild-type and NS5A-resistant viral variants, differentiating it from first-generation NS5A inhibitors.

Poster No. 1201: No Clinically Significant Pharmacokinetic Interaction between Sovaprevir and ACH-3102 in Healthy Volunteers. Saturday, April 27, 2013. Poster Session — Category 08d: Viral Hepatitis C: Clinical (new compounds, resistance).

• In a 24-patient healthy volunteer study, there was no clinically significant pharmacokinetic interaction between ACH-3102 and sovaprevir, Achillion's 2^nd generation NS3 Protease Inhibitor (PI), when co-administered with or without food.
• Co-administration of ACH-3102 and sovaprevir was deemed safe and well-tolerated and the results support a combination Phase 2 study which is currently underway

Poster No. 1199: Findings from Clinical Virology Studies on ACH-3102 are Consistent with Preclinical Observations on its Improved Potency Against Genotype-1A HCV and Resistant Variants. Saturday, April 27, 2013: Poster Session — Category 08d: Viral Hepatitis C: Clinical (new compounds, resistance).

• Single doses of ACH-3102 at 25 mg - 300 mg have previously shown robust antiviral activity in Genotype 1a patients, with reductions in HCV RNA observed in presence of baseline mutations associated with viral resistance to NS5A inhibitors.
• These results show that ACH-3102 administered in 50 mg, 150 mg, and 300 mg doses reduced viral load by 3.98-4.60 log₁₀ IU/mL in four patients with Y93C/D/H mutation, 3.35-4.02 log₁₀ IU/mL in four patients with M28T/V mutation, and 3.40 log₁₀ IU/mL in one patient with L31M mutation.
• Also in GT-1a patients, a comparison of 1-150 NS5A versus full-length NS5A showed less than five-fold differences in their susceptibility to ACH-3102, compared with up to a greater than 358-fold differences in susceptibility to daclatasvir.
• ACH-3102 retained potency (EC₅₀0.042 nM) in GT-1b patients with pooled NS5A mutations, all of whom carried the L31M and Y93H double mutation. In contrast, daclatasvir potency was reduced 2,200-fold (EC₅₀ 22 nM).

"The high barrier to resistance observed with ACH-3102 to date continues to confirm our view that ACH-3102 is unique from other NS5As and has the promise to be a cornerstone therapy in the treatment of Hepatitis C," said Milind Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer of Achillion. "The unique characteristics of ACH-3102, combined with the unique profile of our 2nd generation PI, sovaprevir, which also has a higher barrier to resistance than typical PIs, make for what we believe is a high potential combo, competitive against both current and emerging combinations."

Additional Poster Presentations at EASL:
Friday, April 26, 2013: Poster Session — Category 08c: Viral Hepatitis C: Clinical (therapy).

• Lawitz E., et al. ACH-2684 Demonstrates Potent Viral Suppression in Genotype 1 Hepatitis C Patients with and without Cirrhosis: Safety, Pharmacokinetic, and Viral Kinetic Analysis. Pres. #847.
• Agarwal A., et al. Viral Kinetics Modeling to Predict cEVR and Aid in Dose Selection Decisions for Phase 2/3 Clinical Trials. Pres. #784.

Saturday, April 27, 2013: Poster Session — Category 08d: Viral Hepatitis C: Clinical (new compounds, resistance).

• Fabrycki J., et al. Findings from Clinical Virology Studies on Sovaprevir, a Phase 2 HCV NS3A Protease Inhibitor, Indicate a High Pharmacological Barrier to Viral Resistance. Pres. #1200.

About 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 log ₁₀ 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 HCV.

About Sovaprevir
Sovaprevir, previously referred to as ACH-1625, is a pan-genotypic HCV protease inhibitor designed and synthesized based on crystal structures of enzyme/inhibitor complex. Sovaprevir is an open chain, non-covalent, reversible inhibitor of NS3 protease. In preclinical studies, sovaprevir demonstrated high potency, unique pharmacokinetic properties and an excellent safety profile at high drug exposures. Sovaprevir has rapid and extensive partitioning to the liver, as well as high liver/plasma ratios, and has shown low single-digit nanomolar potency that is specific to HCV. It is equipotent against HCV genotypes 1a and 1b at IC₅₀ of approximately 1nM. Sovaprevir is currently in Phase 2 clinical development and has shown clinical antiviral activity against genotypes 1 and 3. Fast Track status was granted to sovaprevir in 2012 for the treatment of chronic 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, making HCV 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 expected potency, safety, tolerability, effectiveness, combinability and other characteristics of sovaprevir and ACH-3102; and Achillion's expectations regarding timing for the commencement, completion and reporting of results of its clinical trials of both ACH-3102 in combination with ribavirin and sovaprevir in combination with ACH-3102. We may use words such as "expect," "anticipate," "project," "intend," "plan," "believe," "seek," " estimate," and "may" and similar expressions to identify such forward-looking statements. Among the important 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 clinical trials of sovaprevir, ACH-3102 and its other product candidates; 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, 2012 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 duty to update any forward-looking statement, except as required by applicable law.

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Hepatitis C - Achillion Reports Interim Results on ACH-3102

January 7, 2013

 

Achillion Reports Interim Results on ACH-3102

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* n = 5	ETR** n = 3
# of subjects (%)	4/5 (80%)	3/3 (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 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

» Return to Achillion Investor Relations

AASLD-Achillion Programs: Sovaprevir, ACH-3102 and ACH-2684

Achillion Provides Update on Clinical HCV Development Programs

Drug-Drug Interaction Study of Sovaprevir and ACH-3102 Completed -

- ACH-2684, Second-Generation Protease-Inhibitor, Achieves Comparable Activity in Cirrhotic Versus Non-Cirrhotic Patients -

- Posters Discussing Sovaprevir, ACH-3102 and ACH-2684 to be Presented at AASLD 2012 -

NEW HAVEN, Conn., Nov. 12, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today provided an update on the sovaprevir and ACH-3102 clinical development programs and announced new Phase 1b clinical trial results on ACH-2684.

Sovaprevir and ACH-3102 Development Update
Achillion announced today the completion of a drug-drug interaction (DDI) study evaluating the combination of sovaprevir and ACH-3102. The study, conducted in 24 healthy volunteers, concluded that there was no interaction between the two investigational compounds and that both sovaprevir and ACH-3102 were safe and well-tolerated by study participants with no significant adverse events reported.

"With the completion of this DDI study, we now plan to submit to the FDA a Phase 2 protocol that will evaluate an all-oral, interferon-free regimen containing sovaprevir and ACH-3102 for 12 weeks and, pending discussions with the regulatory agency, we intend to initiate this combination study with the goal of reporting rapid virologic response, or RVR, in the first half of 2013," stated Michael D. Kishbauch, President and Chief Executive Officer of Achillion.

Milind Deshpande, Ph.D., President of Research and Development and Chief Scientific Officer of Achillion commented, "We also continue to enroll and treat patients in our ongoing Phase 2 trial evaluating ACH-3102 and ribavirin for the treatment of genotype 1b CC HCV patients and, based upon emerging clinical trial results, we plan to initiate discussions with regulatory agencies to expand the enrollment to include additional genotype 1b patients and look forward to providing an update on this study later in the fourth quarter detailing RVR and safety results."

ACH-2684: Phase 1b study in Chronic HCV GT1 Cirrhotic Patients
Achillion also reported today additional proof-of-concept data from its Phase 1b clinical trial of ACH-2684, a second-generation protease inhibitor, which demonstrated that chronic genotype 1 HCV treatment-naive patients with cirrhosis treated with 400 mg

ACH-2684 once daily for three days achieved a mean maximum 3.67 log10 reduction in HCV RNA (range 3.10-4.40 log10) as compared to 0.22 log10 reduction for placebo. The antiviral activity achieved in this treatment-naive patient population was similar to the 3.68 log10 reduction in HCV RNA in non-cirrhotic genotype 1 HCV treatment-naive patients receiving the same dose of ACH-2684. The full dosing cohort (n=8; 6 active, 2 placebo) included one subject that received active drug who, based upon baseline viral population sequencing, was demonstrated to have nearly 100% substitution at position R155K. Presence of approximately 100% R155K mutation at baseline in subjects who have not been exposed to a protease inhibitor regimen is highly unusual and likely indicative of prior treatment that was not revealed at study enrollment. Further analysis is on-going and this patient's antiviral activity is excluded from the reductions above.

Safety data demonstrated that ACH-2684 was well tolerated in this patient population with 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.

"With the unique pharmacokinetic profile of ACH-2684, which we believe does not require active uptake of the compound into the liver, we are very pleased to have achieved the same robust antiviral activity in patients with compensated cirrhosis," commented Dr. Deshpande. "We believe these results support the continued development of this second-generation protease inhibitor and will look to fully leverage its activity in special populations of HCV patients."
Posters Presentations by Achillion at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD):

-- Poster 1171: Viral Kinetics Modeling to aid in Dose Selection Decisions
for Phase-2/3 Clinical Trials, A. Agarwal, et al. Session: Clinical HCV
2. Date: Tuesday, November 13, 2012.

-- Poster 1886: In vitro Studies Demonstrate a High Probability of Curing
Genotype-1 Hepatitis C with Combination of a Novel NS3 Protease
Inhibitor ACH-2684 and a Novel NS5A inhibitor ACH-3102, Y. Zhao, et al.
Session: HCV Therapy: Pre-clinical and Early Clinical Development. Date:
Tuesday, November 13, 2012.

About ACH-3102
ACH-3102, Achillion's second generation, pan-genotypic NS5A inhibitor, is a pico-molar potent compound that has been improved from first-generation NS5A inhibitors to maintain excellent potency against both wild type HCV as well as potency against resistant mutants that have been identified in clinical studies. In vitro, ACH-3102 has demonstrated potent activity against all HCV genotypes and shown additive to synergistic activity when combined with NS3 protease inhibitors, NS5B polymerase inhibitors, and ribavirin. ACH-3102 was shown to be safe and well tolerated in Phase 1 studies with potent antiviral activity achieved after a single dose. ACH-3102 is currently being evaluated in a Phase 2 trial in combination with ribavirin for 12 weeks as a treatment for chronic HCV genotype 1b.

About Sovaprevir
Sovaprevir is a Phase 2 pan-genotypic HCV protease inhibitor designed and synthesized based on crystal structures of enzyme/inhibitor complex. Sovaprevir is an open chain, non-covalent, reversible inhibitor of NS3 protease. In clinical and preclinical studies, sovaprevir demonstrated high potency, unique pharmacokinetic properties and an excellent safety profile at high drug exposures. With low single-digit nanomolar potency specific to HCV, sovaprevir is equipotent against HCV genotypes 1a and 1b at IC50 of approximately 1nM. Sovaprevir achieved sustained viral response rates of approximately 80% in combination with pegylated-interferon and ribavirin for the treatment of HCV genotype 1, and will be evaluated in all-oral, interferon-free regimens, Fast Track status was granted to sovaprevir in 2012 for the treatment of chronic HCV.

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 nearly 4 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 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 that could cause actual results to differ materially from those indicated by such forward-looking statements, including statements with respect to:
the expected potency, safety, tolerability, effectiveness and other characteristics of sovaprevir, ACH-3102 and ACH-2684; and Achillion's plans and timing for advancing its clinical trials, including the combination trial of sovaprevir and ACH-3102, the ongoing trial of
ACH-3102 and ribavirin, and future studies of ACH-2684. 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 clinical trials 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; 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:
Lee Stern
The Trout Group, LLC
Tel. (646) 378-2922
lstern@troutgroup.com

Achillion Pharmaceuticals moves up as a contender in the HCV market

Achillion Pharmaceuticals moves up as a contender in the HCV market

Written by Dr. Ramya Kartikeyan, GlobalData's senior healthcare analyst covering infectious diseases.

GlobalData-  October 1 2012

On September 27, Achillion Pharmaceuticals released news of positive results surrounding Phase Ia trials testing ACH-3102, an NS5A inhibitor drug created to treat chronic Hepatitis C Virus (HCV) infections. In the past few months, developers in this space have faced serious concerns. Bristol-Myers Squibb’s HCV drug candidate BMS-986094, another NS5A inhibitor, recently caused the death of a patient during Phase IIb trials as a result of heart failure. The FDA, in response to this adverse event, moved to halt clinical trials on competitor Idenix Pharmaceuticals’ HCV NS5A inhibitor, IDX184.

 

Currently, the leading drugs on the market, Vertex’s Incivek (telaprevir) and Merck’s Victrelis (boceprevir) are only indicated with the use of Pegasys (pegylated interferon-alpha) and ribavirin, which fall short on multiple counts. Most important, however, are the toxicity issues associated with the use of ribavirin and Pegasys, and the fact these treatments do not intrinsically eliminate the virus, but instead stimulate the immune system to help deal with the viral infection. Both Incivek and Victrelis are NS5A inhibitors, which offer a glimpse into the market possibilities for a drug like Achillion’s ACH-3102. In contrast, Achillion has stated that further trials will test ACH-3102 in combination with ribavirin but not Pegasys. Nevertheless, this approach keeps in line with the trend of using NS5A inhibitors in combination with other Direct-Acting Antiviral (DAA) therapies.

 

In developing ACH-3102, Achillion focused on creating a drug that targets multiple genotypes of HCV. The different HCV genotypes pose a problem within the general population, where treatment towards a specific genotype of HCV is less likely to treat infections against other HCV genotypes.

 

Currently, there are 11 known genotypes of HCV, which makes the pan-genotypic treatment option of a drug like ACH-3102 very appealing to physicians and patients alike. Achillion’s research would appear to address the unmet need of drugs capable to treat multiple genotypes, in addition to creating drugs that deal with resistant strains of HCV. While seemingly a sound investment in research and development funds, it is interesting to note that many other players in this market still have active research programs focused upon treating HCV 1a.

 

The current HCV pipeline continues to be robust and growing. With most companies doubling down on their investments, research includes concurrent development in NS5A inhibitors, NS3 protease inhibitors, nucleotide inhibitors and polymerase inhibitors. With the pipeline leaning heavily in favor of polymerase and protease inhibitors, it would appear that the current marketed drugs have had a large role in determining the research focus of the newer products. More importantly, as products move to a fixed-dose combinatorial therapy, as seen in the HIV market, more companies are involved in expanding their pipeline to include different drug options and eliminate a split in potential drug revenue.

 

A growing population segment within the HCV therapeutics market is patients co-infected with HIV. This segment, largely seen as a growth market in countries with high prevalence of HIV, might allow companies to realize large profits within the seven major markets (US, UK, France, Italy, Germany, Spain and Japan), where prevalence of HIV is higher than its incidence. As companies such as Bristol-Myers Squibb, Gilead, Vertex and Abbott develop new HCV drugs, a likely secondary indication would be in patients infected with HIV.

 

With an early win, Achillion faces a long and turbulent path to the finish line. In the face of other bigger pharma players, Achillion might be seen as a potential acquisition or licensing opportunity, and given the debacle with Bristol-Myers Squibb’s HCV drug candidate, Achillion might not even think twice about offsetting some of the risk associated with the continued development of this drug. With a diverse HCV and antibacterial portfolio, Achillion is undoubtedly research-rich. Without assessing Achillion’s corporate strategy, the company will face important decisions with respect to marketing and commercialization approaches it chooses to adopt for their leading candidate drugs. However, as long as their drugs continue to trend positively, Achillion will enjoy the privilege of being courted by Big Pharma and seeking an alliance strongly in their favor.

 

http://www.globaldata.com/ExpertsInsightDetails.aspx?PRID=402&companyID=jpr/oAchillion

ACH-3102-Achillion Announces Positive Proof-of-Concept Data

Achillion Sees Positive Results in Hepatitis C Study
By Ben Fox Rubin
Achillion is among a handful of companies racing to bring an all-oral regimen for hepatitis C to the market. The new drugs being developed would eliminate an injectable drug used in the current standard treatment, interferon, which can be difficult for patients to tolerate. Major players working on new hepatitis C drugs include Gilead Sciences Inc. (GILD) and Bristol-Myers Squibb Co. (BMY).
Following positive results from a Phase 1a trial for its ACH-3102 drug, Achillion has initiated a pilot Phase 2 trial evaluating the drug. The company said the drug showed "robust antiviral activity" in its trial.
 *ACH-3102 received fast-track designation from the U.S. Food and Drug Administration in May.
Continue Reading....
 

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

http://ir.achillion.com/releasedetail.cfm?ReleaseID=709768

Achillion Pharma strikes deal to raise $41.8M as hep C results loom

Achillion Pharma strikes deal to raise $41.8M as hep C results loom

August 31, 2012 | By Ryan McBride
Fierce Biotech

Hepatitis C drug developer Achillion Pharmaceuticals ($ACHN) expects to haul in $41.8 million proceeds of a common stock sale to QVT Financial. And the funding comes ahead of some important clinical trial results from the New Haven, CT-based developer's pipeline of hep C treatments...

Achillion has several experimental oral meds in early- or mid-stage trials for hep C, a liver-damaging virus that afflicts an estimated 170 million people around the globe. The biopharma stampede is chasing all-oral regimens for the illness that can spare hep C patients from lengthy treatment on injections of interferon, which causes flu-like symptoms that make patients feel worse than the infectious disease does. And Achillion's contenders offer potential ingredients in cocktail therapies of oral meds to potentially wipe out the virus without those side effects from interferon....

Early next year Achillion is expected to report early data from an upcoming trial that tests a combo of the company's HCV NS3 protease inhibitor sovaprevir and a compound known as ACH-3102 as an interferon-free therapy for hep C, according a recent investor note from Cowen & Company...

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Hepatitis C-ACH-3102 Receives Fast Track Designation From the FDA

May 15, 2012, 7:00 a.m. EDT

ACH-3102 Receives Fast Track Designation From the FDA for the Treatment of Chronic Hepatitis C

NEW HAVEN, Conn., May 15, 2012 (GlobeNewswire via COMTEX) -- Achillion Pharmaceuticals, Inc. /quotes/zigman/100395/quotes/nls/achn ACHN -4.31% announced today the receipt of a Fast Track designation from the U.S. Food and Drug Administration (FDA) for ACH-3102 as part of an interferon-free regimen for the treatment of chronic hepatitis C (HCV). ACH-3102 is a pan-genotypic second-generation NS5A inhibitor against HCV that was discovered by Achillion and is currently being evaluated in a Phase 1 clinical trial.
Fast Track designation was requested for ACH-3102 for its potential to provide:

--  Improved safety as compared to the current standard of care;
          --  Potential for development in a once daily interferon-free fixed dose
              combination;
          --  Potent antiviral activity in vitro against HCV genotypes 1 through 6;
              and
          --  Low potential for drug-drug interactions and therefore greater potential
              to treat HCV patients with comorbidities, co-infected with HIV, or pre-
              or post-liver transplantation.   

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. "We are very pleased with the granting of a Fast Track designation for ACH-3102, which we believe highlights this second-generation NS5A inhibitor's attributes that include pan-genotypic coverage of HCV and potential for maintained activity against NS5A mutant strains of HCV," commented Michael Kishbauch, President and Chief Executive Officer of Achillion. "We are excited to leverage the superior profile of ACH-3102 in combination with our Phase 2 protease inhibitor, ACH-1625, as we seek to create an optimized, potentially best-in-class potent, well-tolerated, once-daily regimen to treat HCV, which will enter combination studies during the third quarter of this year."

Under the FDA Modernization Act of 1997, the Fast Track program facilitates interactions with the FDA before and during the submission of a New Drug Application (NDA) for therapeutics being investigated as a treatment of serious or life-threatening diseases which demonstrate the potential to address an unmet medical need for such a condition. The Fast Track program enables a company to file an NDA on a rolling basis as data becomes available. This permits the FDA to review the filing as it is received, rather than waiting for the entire document prior to commencing the review process. With a Fast Track designation, there is an opportunity for more frequent interactions with the FDA and the possibility of a priority review, which could decrease the typical development time and review period.
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. 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.

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-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-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 nonclinical 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.

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

EASL: PRECLINICAL CHARACTERISTICS OF ACH-3102: A NOVEL HCV NS5A INHIBITOR WITH IMPROVED POTENCY AGAINST GENOTYPE-1A VIRUS AND VARIANTS RESISTANT TO 1ST GENERATION NS5A INHIBITORS - (05/02/12)

NEW HAVEN, Conn., May 9, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) 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.

Read More At NATAP

Achillion Pan-Genotypic ACH-3102 begins dosing in a Phase 1 clinical trial

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. /quotes/zigman/100395/quotes/nls/achn ACHN +1.00% 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.