Achillion Presents Results on ACH-3102 and ACH-3422 at EASL

Achillion Presents Detailed Clinical Results on ACH-3102 and ACH-3422 at the International Liver Congress

- Two late breaker presentations detail previously announced 100% SVR12 in Phase 2 trial evaluating 6- or 8-weeks of treatment with ACH-3102 and sofosbuvir in genotype 1 HCV patients and Phase 1 proof-of-concept results with ACH-3422 -

- Clinical virology presentation continues to support improved barrier to resistance with ACH-3102 -

VIENNA, Austria, April 25, 2015 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today presented two late breaker posters at the 50^th Annual Meeting of the European Association for the Study of the Liver (EASL) during The International Liver Congress 2015.

"We believe that the ability to achieve 100% SVR12 after six-weeks of treatment with ACH-3102 and sofosbuvir supports the potential for our proprietary doublet regimen to reduce treatment duration for HCV patients," commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. "We are also very pleased with the robust Phase 1 proof-of-concept data reported on ACH-3422, our proprietary NS5B nucleotide polymerase inhibitor, and the clinical virology presentation that continues to support the differentiated higher barrier to resistance for ACH-3102."

Data highlights from all of the presentations include:
LP06: Sustained virologic response after ACH-3102 and sofosbuvir treatment for 8 or 6 weeks: A phase 2 "proxy" study ACH102-017. ePoster Presentation, April 25, 3:30 pm CET, Hall B ePoster Area. Lead Author: Edward Gane.

• As previously reported, 100% of patients achieved SVR12 after six- (n=12) or eight-weeks (n=12) of treatment in this ongoing interferon-free, ribavirin-free study evaluating the efficacy, safety, and tolerability of 50 mg of ACH-3102 and 400 mg of sofosbuvir, a marketed nucleotide polymerase inhibitor, in treatment-naïve genotype 1 HCV-infected patients.
• This represents the first study to report 100% SVR12 in patients with chronic GT-1 HCV infection using a two-drug combination for 6 weeks.
• Complete virologic responses were seen in all patients, including those who were considered harder to treat than others (i.e. GT-1a, non-CC and VL > 6 million IU/mL).
• The combination of ACH-3102 with sofosbuvir was well tolerated, with no treatment discontinuations, a low incidence of AEs, and no reported significant AEs or SAEs during the treatment and follow-up periods.
• The present study provides support for future studies which will explore the use of ACH-3102 in sofosbuvir-sparing regimens with short-treatment durations.
• In parallel, further studies will explore the combination of ACH-3102 and ACH-3422 (with and without sovaprevir, an NS3 protease inhibitor) in interferon- and ribavirin-free regimens with short treatment durations across different patient populations.

LP27: Gane, E., et al. ACH-3422, a novel nucleotide prodrug inhibitor of HCV NS5B polymerase. ePoster Presentation, April 25, 3:30 pm CET, Hall B ePoster Area. Lead Author: Edward Gane.

• As previously announced, ACH-3422 achieved dose-related virologic responses in GT-1 HCV-infected patients. In the six patients who received 700 mg once daily for 14 days, mean maximal reduction from baseline was 4.6 log10, including three patients with target not detected.
• In all healthy volunteers and patients infected with HCV who received active treatment through 700 mg once daily, ACH-3422 was well-tolerated with no treatment-related serious adverse events, adverse event-related discontinuations, or clinically significant laboratory or ECG abnormalities.
• These results support further investigation of ACH-3422 with ACH-3102, a potent NS5A inhibitor, with or without the NS3/4A protease inhibitor sovaprevir, for the treatment of different patient populations with chronic HCV infection.

P0805: Achievement of SVR12 despite the presence of HCV variants resistant to first generation NS5A inhibitors in genotype-1 hepatitis C patients after 8-week therapy of ACH-3102 in combination with sofosbuvir. ePoster Presentation, April 24, 10:00 am CET, Hall B ePoster Area. Lead Author: Wengang Yang.

• Dominant NS5A variants found at baseline conferred significant resistance to ledipasvir and daclatasvir but not to ACH-3102, highlighting the mechanism underlying the improved clinical efficacy of ACH-3102.
• The clinical efficacy of ACH-3102 is also supported by its greater potency in vitro against a spectrum of GT-1a and -1b NS5A mutants as compared to the first generation of NS5A inhibitors.
• These data support further clinical exploration of ACH-3102.

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 150 million people are infected with HCV worldwide including more than 5 million people in the United States. 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.

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. The Company 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, including without limitation statements with respect to: the potential to create shorter treatments for HCV patients and the potential therapeutic and other benefits of ACH-3422 and ACH-3102. 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: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; advance the preclinical and clinical development of its drug candidates, including ACH-3102, ACH-3422, and sovaprevir, 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 successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; 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 fiscal year ended December 31, 2014, 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.

Achillion, Gilead drug cocktail cures hepatitis C in six weeks

Achillion Achieves 100% SVR12 in Phase 2 Trial Evaluating 6-Week Combination Treatment With ACH-3102

- Achillion achieves 100% SVR12 in six-week regimen with combination of ACH-3102 and sofosbuvir for treatment-naïve genotype 1 HCV -

- Achillion to initiate 4-week treatment regimens based on the strength of ACH-3102 antiviral data -

NEW HAVEN, Conn., Feb. 9, 2015 (GLOBE NEWSWIRE) --Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced updated interim results from the ongoing interferon-free, ribavirin-free, Phase 2 study to evaluate the efficacy, safety, and tolerability of six weeks of 50 mg of ACH-3102 and 400 mg of sofosbuvir, a marketed nucleotide polymerase inhibitor, in treatment-naïve genotype 1 HCV-infected patients. The primary objective of the study is determination of sustained viral response 12 weeks (SVR12) after completion of therapy. One hundred percent of patients (12/12) in the six-week treatment duration arm achieved SVR12, which included patients with high baseline viral load.

"The ability to further shorten treatment duration to only six weeks and maintain excellent SVR12 rates remains the goal for clinicians and patients, and I am pleased that these Phase 2 results support that goal. The profile of ACH-3102, represents an important and exciting treatment option to shorten treatment duration for patients infected with HCV," commented Professor Edward Gane, M.D., Deputy Director and Hepatologist, New Zealand Liver Transplant Unit, Auckland City Hospital in New Zealand, and Lead Investigator in the Phase 2 study of ACH-3102 and sofosbuvir and the ACH-3422 nucleotide inhibitor program.

Dr. Milind Deshpande, President and Chief Executive Officer of Achillion, commented, "Our goal is to deliver short duration, widely accessible treatments to all HCV patients. We believe that these results with ACH-3102 represent the shortest duration and highest response achieved to date with any two-drug, direct-acting antiviral regimen for HCV. Given the exceptional profile of ACH-3102, we will now be evaluating four- and six-week treatment durations that leverage all of our HCV assets including ACH-3102, ACH-3422, and sovaprevir."

Overview of Phase 2 Proxy Study Design and Top-line Results
This ongoing study is a Phase 2 open-label, randomized, partial-crossover study to evaluate the efficacy, safety, and tolerability of eight- and six weeks of 50 mg of ACH-3102 and 400 mg of sofosbuvir, a marketed nucleotide polymerase inhibitor, once daily, in treatment-naïve genotype 1 HCV-infected patients. Initially, eighteen patients were enrolled, including six observational patients, into an eight-week treatment cohort.

Following the achievement of 100 percent SVR12 (12/12) in the eight-week cohort, the six-week treatment cohort was initiated. In all, eighteen patients were enrolled, including twelve active and six observational patients. Mean baseline HCV RNA viral load was 10 million (7 log₁₀) IU/ml, range 2 million (6.23 log₁₀) - 97 million (7.99 log₁₀) IU/ml, including seven patients with baseline HCV RNA viral load exceeding 6 million (6.78 log₁₀) IU/ml. Of the 12 active patients enrolled, seven patients were genotype 1a and five were genotype 1b.

Twelve weeks after the completion of therapy, 100 percent (12/12) achieved SVR12, independent of baseline viral load, gender, and IL28B status, in the six-week treatment arm. Additionally, one hundred percent of patients (12/12) in the eight-week treatment duration arm have achieved SVR24. The combination of ACH-3102 and sofosbuvir was well-tolerated with no serious adverse events, no discontinuations due to adverse events, and no clinically significant laboratory or ECG abnormalities.

"The achievement of 100% SVR12 after six weeks of treatment with a dual NS5A-nucleotide regimen, even in patients with high baseline viral load who would otherwise require extended duration treatments, supports our belief that ACH-3102 can unleash the potential of this combination to drive down treatment duration," commented Dr. David Apelian, Executive Vice President of Clinical Development and Chief Medical Officer at Achillion. "We are currently preparing to initiate our SPARTA Phase 2 program which evaluates short treatment durations with our proprietary once-daily regimens of ACH-3102 and ACH-3422, with or without sovaprevir, for treatment naïve genotype 1 HCV patients. In parallel, we plan on exploring sofosbuvir-sparing regimens that will leverage shorter durations of sofosbuvir in combination with ACH-3102 and sovaprevir as part of our global development program."

Achillion, Gilead drug cocktail cures hepatitis C in six weeks

(Reuters) - Achillion Pharmaceuticals Inc said its experimental hepatitis C drug, when used in combination with Gilead Sciences Inc's Sovaldi, eradicated signs of the virus after six weeks of therapy, sending its shares up before the opening bell.

If the combination of Sovaldi and Achillion's ACH-3102 continues to show this level of effectiveness, the treatment could eventually rival offerings from Gilead and AbbVie Inc to fight the liver-destroying virus.

Gilead's new-generation hepatitis C treatment, Harvoni, which combines Sovaldi with the company's own NS5A inhibitor, achieves this response after eight weeks of therapy. Sales of Harvoni totaled $2.11 billion in the quarter ended Dec. 31.

AbbVie's regimen, Viekira Pak, which won U.S. approval in December, takes 12 weeks to achieve a cure.

Shares of Achillion, one of the few companies developing hepatitis C therapies independently, rose about 10 percent in premarket trading on Monday.

The main goal of Achillion's ongoing mid-stage study is to achieve a sustained virological response 12 weeks after therapy, which would constitute a cure.

The study is testing a 50 milligram (mg) dose of Achillion's NS5A inhibitor in combination with 400 mg of Gilead's sofosbuvir in previously untreated genotype 1 hepatitis C patients.

The market for hepatitis C drugs has developed at a lightening pace in recent years, with several companies working on producing newer drugs to cure the disease, which affects about 150 million globally.

Achillion's data represents the shortest duration and highest response achieved to date with any two-drug, direct-acting antiviral regimen for hep C, Chief Executive Milind Deshpande said in a statement.

"We will now be evaluating four- and six-week treatment durations that leverage all of our (hepatitis C) assets including ACH-3102, ACH-3422 and sovaprevir," he said.

Achillion shares closed at $10.82 on the Nasdaq on Friday.

Gilead's stock was down about 0.5 percent at $96.98 premarket.

(Reporting by Natalie Grover in Bengaluru; Editing by Savio D'Souza and Ted Kerr)

AASLD- Achillion Reports 100% SVR12 With ACH-3102 and Sovaldi

Achillion Reports 100% SVR12 in a Phase 2 Combination Study With ACH-3102 at the Liver Meeting 2014 (AASLD)

Achillion Achieves 100% SVR12 in Eight-Week Phase 2 Trial Evaluating a Ribavirin-Free Regimen of ACH-3102 and Sofosbuvir for Genotype 1 HCV ("Proxy Study") Including Nine of 12 Patients With Viral Loads Higher Than 6 Million IU/ml at Baseline -

- Reports Additional Preclinical Results for ACH-3422, Uridine-Analog Nucleotide NS5B Polymerase Inhibitor -

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced the presentation of results from the ongoing Phase 2 study of ACH-3102 in a late breaker poster and data in three preclinical posters on ACH-3422. The poster presentations are being made at the 65th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), The Liver Meeting 2014, which takes place through November 11, 2014 in Boston, MA.

Late Breaker Poster Presentation: Phase 2 pilot study evaluating eight week treatment of ACH-3102 in combination with sofosbuvir for genotype 1 treatment-naïve HCV

In a late breaker poster presentation, Achillion reported updated interim results from an ongoing interferon-free, ribavirin-free, 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, a marketed nucleotide polymerase inhibitor, without ribavirin, in treatment-naïve genotype 1 HCV-infected patients. The primary objective of the study is determination of sustained viral response 12 weeks (SVR12) after the completion of therapy. Eighteen patients were enrolled, including six observational patients. Twelve patients completed eight weeks of treatment consisting of 50 mg of ACH-3102 and 400 mg of sofosbuvir administered once daily while observational patients received no drug during this phase of the trial.

Of the 12 patients treated, 100 percent (n=12/12) achieved SVR12. Of the 12 patients treated in this study, nine of 12 patients had a baseline viral load substantially greater than 6 million IU/ml at baseline. No on-treatment viral breakthrough or post-treatment viral relapse has been observed.

Preclinical poster presentations on ACH-3422

Achillion presented three posters at AASLD which reported updated preclinical results on ACH-3422. The in vitro results demonstrated that this nucleotide pro-drug has improved potency against genotype 3 HCV as compared to sofosbuvir. In addition, in a separate poster presentation, Achillion reported that ACH-3422 displays additive to synergistic activity when combined with ACH-3102 or sovaprevir, Achillion's Phase 2 NS3/4A protease inhibitor, in vitro. Furthermore, the high barrier to resistance for ACH-3422 was supported with the ability of the agent to block, in vitro, the appearance of resistant colonies in combination with other direct-acting antiviral agents.

"The antiviral activity and safety profile observed to date for ACH-3422 both in preclinical studies and in the ongoing 422-001 Phase 1 trial support further development with this nucleotide in combination with Achillion's other direct-acting antivirals, and represents an exciting treatment option for HCV," commented Professor Edward Gane, M.D., Deputy Director and Hepatologist, New Zealand Liver Transplant Unit, Auckland City Hospital in New Zealand, and Lead Investigator in the ACH-3422 Phase 1 proof-of-concept study and Phase 2 proxy study of ACH-3102 and sofosbuvir.

Reprints of the posters are available on the Company's website at www.achillion.com/resources.

AASLD-Achillion Reports 100% SVR12 in a Phase 2 Combination Study With ACH-3102 and Sofosbuvir

Achillion Reports 100% SVR12 in a Phase 2 Combination Study With ACH-3102 at the Liver Meeting 2014 (AASLD)

- Achillion Achieves 100% SVR12 in Eight-Week Phase 2 Trial Evaluating a Ribavirin-Free Regimen of ACH-3102 and Sofosbuvir for Genotype 1 HCV ("Proxy Study") Including Nine of 12 Patients With Viral Loads Higher Than 6 Milachlion IU/ml at Baseline –

- Reports Additional Preclinical Results for ACH-3422, Uridine-Analog Nucleotide NS5B Polymerase Inhibitor –

NEW HAVEN, Conn., Nov. 8, 2014 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced the presentation of results from the ongoing Phase 2 study of ACH-3102 in a late breaker poster and data in three preclinical posters on ACH-3422. The poster presentations are being made at the 65^th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), The Liver Meeting 2014, which takes place through November 11, 2014 in Boston, MA.

Late Breaker Poster Presentation: Phase 2 pilot study evaluating eight week treatment of ACH-3102 in combination with sofosbuvir for genotype 1 treatment-naïve HCV
In a late breaker poster presentation, Achillion reported updated interim results from an ongoing interferon-free, ribavirin-free, 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, a marketed nucleotide polymerase inhibitor, without ribavirin, in treatment-naïve genotype 1 HCV-infected patients. The primary objective of the study is determination of sustained viral response 12 weeks (SVR12) after the completion of therapy. Eighteen patients were enrolled, including six observational patients. Twelve patients completed eight weeks of treatment consisting of 50 mg of ACH-3102 and 400 mg of sofosbuvir administered once daily while observational patients received no drug during this phase of the trial.

Of the 12 patients treated, 100 percent (n=12/12) achieved SVR12. Of the 12 patients treated in this study, nine of 12 patients had a baseline viral load substantially greater than 6 million IU/ml at baseline. No on-treatment viral breakthrough or post-treatment viral relapse has been observed.

Preclinical poster presentations on ACH-3422
Achillion presented three posters at AASLD which reported updated preclinical results on ACH-3422. The in vitro results demonstrated that this nucleotide pro-drug has improved potency against genotype 3 HCV as compared to sofosbuvir. In addition, in a separate poster presentation, Achillion reported that ACH-3422 displays additive to synergistic activity when combined with ACH-3102 or sovaprevir, Achillion's Phase 2 NS3/4A protease inhibitor, in vitro. Furthermore, the high barrier to resistance for ACH-3422 was supported with the ability of the agent to block, in vitro, the appearance of resistant colonies in combination with other direct-acting antiviral agents.

"The antiviral activity and safety profile observed to date for ACH-3422 both in preclinical studies and in the ongoing 422-001 Phase 1 trial support further development with this nucleotide in combination with Achillion's other direct-acting antivirals, and represents an exciting treatment option for HCV," commented Professor Edward Gane, M.D., Deputy Director and Hepatologist, New Zealand Liver Transplant Unit, Auckland City Hospital in New Zealand, and Lead Investigator in the ACH-3422 Phase 1 proof-of-concept study and Phase 2 proxy study of ACH-3102 and sofosbuvir.
Reprints of the posters are available on the Company's website at www.achillion.com/resources.

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 150 million people are infected with HCV worldwide including more than 5 million people in the United States. 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.

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. The company's 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, including statements with respect to: the Company's reiteration that it remains on track to initiate all-oral ribavirin-free regimens with ACH-3422, ACH-3102 and sovaprevir for the treatment of HCV in 2015; the Company's expectations that the Phase 1 study of ACH-3422 could inform the potential initiation of combination studies of ACH-3422 and ACH-3102; and the Company's expectations that it may report preliminary results from its Phase 1 program during the fall of 2014. 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: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; advance the preclinical and clinical development of its drug candidates, including ACH-3422, ACH-3102 and sovaprevir, 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 successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; 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, 2013, 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.

Company Contact: Glenn Schulman Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 Media: Laurie Masonson Ogilvy PR Tel. (917)459-6164 Investors: Mary Kay Fenton Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 Investors: Tricia Truehart The Trout Group, LLC Tel. (646) 378-2953

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.

Achillion's hepatitis C drug shows promise in trial

Achillion's hepatitis C drug shows promise in trial

(Reuters) - Achillion Pharmaceuticals Inc said all patients treated with its experimental hepatitis C drug showed no detectable levels of the virus four weeks after the therapy, sending its shares up 18 percent.

The mid-stage trial tested Achillion's drug, ACH-3102, in 12 patients in combination with Gilead Sciences Inc's Sovaldi, also known as sofosbuvir.

Achievement of 100 percent cure rate confirms the competitive profile of NS5A, Wells Fargo Securities analyst Brian Abrahams wrote in a note.

ACH-3102 belongs to a promising new class of drugs that work by blocking the NS5A protein needed by the virus to replicate.

Achillion is also working on a different class of drugs known as nucleotide inhibitor, or "nuc", and which has been the focus of investors' attention.

"This class of drug (ACH-3102) is not as unique an asset as ACH-3422, that's the nuc. If they have positive data, then they are going to have a very high likelihood of takeout just like Idenix," FBR Capital Markets & Co analyst Andrew Berens told Reuters.

Merck & Co Inc agreed in June to buy Idenix Pharmaceuticals Inc for $3.85 billion to boost its hepatitis c drugs portfolio. Gilead paid $11 billion in cash to buy biotech company Pharmasset for Sovaldi.

Gilead said on Friday an arbitration panel ruled in its favor, rejecting patent infringement claims from Roche Holding AG related to Sovaldi.

Achillion is expected to report data from an early-stage trial of ACH-3422 at the end of this year.

Nucs, which comprise the backbone of current hepatitis C treatment, work by blocking a protein needed by the hepatitis C virus to replicate.

Hepatitis C drugs have a history of being expensive, largely because some 170 million people worldwide have the often-fatal liver disease and do not have good treatment options.

Sovaldi costs $84,000 for 12 weeks of treatment.

Achillion said it would begin treating 12 additional patients for six weeks with a once-daily dose of ACH-3102 and sofosbuvir. [ID:nGNXVBIEVa]

The trial excluded the older hepatitis C drug ribavirin, which can cause rashes, anemia and other side-effects.

Achillion shares gave up most of their early gains and were up 8 percent at $9.13 on Friday morning on the Nasdaq. The stock touched an 18-month high of $9.94.

About 12 million shares changed hands by 11:00 a.m. ET, more than four times their 10-day average volume.

(Reporting by Anand Basu in Bangalore; Editing by Simon Jennings)
Reuters

Press Release

Achillion Achieves 100 Percent Sustained Virologic Response Rate (SVR4) From an Eight Week Phase 2 Trial Evaluating a Ribavirin-Free Regimen of ACH-3102 and Sofosbuvir for Genotype 1 HCV ("Proxy Study")

Achillion to Begin a Six Week Treatment Regimen With Its Second-Generation NS5A Inhibitor ACH-3102 and Sofosbuvir

NEW HAVEN, Conn., Aug. 15, 2014 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced interim results 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. Of the 12 patients treated, 100 percent (n=12/12) remained HCV RNA undetectable four weeks after completing therapy (SVR4). Based upon these results, 12 additional patients will begin treatment with six weeks of once daily ACH-3102 and sofosbuvir.

"ACH-3102 continues to demonstrate good safety and tolerability through three Phase 2 studies. We believe these studies also confirm a differentiated efficacy profile for an NS5A inhibitor. Achieving 100% SVR4 with eight weeks of treatment with sofosbuvir serving as a nucleotide proxy indicate that dosing 50 mg once daily of ACH-3102 plus a nucleotide inhibitor has the potential to achieve commercially competitive results for curing HCV in a short duration, ribavirin-free doublet," commented David Apelian, M.D., Ph.D., Executive Vice President and Chief Medical Officer at Achillion. "In addition, understanding how ACH-3102 performs with sofosbuvir provides valuable insight for the design of our proprietary combination trial with ACH-3102 and ACH-3422, a uridine-analog nucleotide that continues to advance through its Phase 1 clinical development program."

Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion commented, "As we continue to achieve clinical milestones, we remain focused on execution of the broader clinical development strategy for our HCV portfolio. We expect that Phase 1 proof-of-concept results with ACH-3422 will be reported during the fall of this year, which we anticipate will lead to the start of a Phase 2 combination program to evaluate our proprietary doublet regimen for an eight week, or potentially shorter, treatment regimen for HCV that will begin before the end of 2014."

ACH-3102 - 017: Phase 2 pilot study evaluating eight 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, a marketed nucleotide polymerase inhibitor, without ribavirin, in treatment-naïve genotype 1 HCV-infected patients. The primary objective of the study is determination of sustained viral response 12 weeks (SVR12) after the completion of therapy. Eighteen patients were enrolled, including six observational patients. Twelve patients completed eight weeks of treatment consisting of 50 mg of ACH-3102 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 with median HCV RNA at baseline of 7.22 log10 (range 5.5 - 7.8 log10). No on-treatment viral breakthrough or post-treatment viral relapse has been observed to date. ACH-3102 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 will be enrolled and receive six weeks of treatment consisting of 50 mg of ACH-3102 and 400 mg of sofosbuvir administered once daily. Achillion anticipates that SVR4 results from the crossover cohort will be reported by the end of 2014.

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 150 million people are infected with HCV worldwide including more than 5 million people in the United States. 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.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion's discovery, clinical development, and commercial teams have advanced multiple novel product candidates with proven mechanisms of action into studies and toward the market. 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.

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, including statements with respect to: the Company's expectations that the Phase 1 study of ACH-3422 could inform the potential initiation of combination studies of ACH-3422 and ACH-3102; the Company's expectations that it may report preliminary results from its Phase 1 program during the fall of 2014; the Company's plan to initiate a Phase 2 combination study of ACH-3422 with ACH-3102 by year-end 2014; the Company's goal to safely and expeditiously advance its all-oral regimens for the treatment of HCV and its expectation that the breadth of its portfolio could enable it 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. 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: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; advance the preclinical and clinical development of its drug candidates, including ACH-3422, ACH-3102 and sovaprevir, 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 successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; 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, 2013, 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.

http://ir.achillion.com/releaseDetail.cfm?ReleaseID=866418 

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.....