Achillion Reports Third Quarter and Nine Month 2014 Financial Results

Achillion Reports Third Quarter and Nine Month 2014 Financial Results

- Hepatitis C development program remains on track to initiate all-oral ribavirin-free regimens with ACH-3422, ACH-3102 and sovaprevir for HCV in 2015 -

- Advancement of novel platform for complement factor D inhibitors for the oral treatment of immune-related rare diseases -

NEW HAVEN, Conn., Nov. 4, 2014

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today reported financial results for the three and nine months ended September 30, 2014, outlined upcoming milestones in its development programs for chronic hepatitis C viral infection (HCV), and introduced a novel platform for advancing oral complement factor D inhibitors.

For the third quarter of 2014, Achillion reported a net loss of $15.7 million or $0.16 per share, compared with a net loss of $13.9 million or $0.14 per share for the third quarter of 2013. Cash, cash equivalents, marketable securities, and interest receivable as of September 30, 2014 were $127.1 million.

Hepatitis C Development Program
During the remainder of 2014, Achillion expects to achieve the following milestones in its HCV development program:

• Present Phase 2 SVR12 results following 8-weeks of treatment with the interferon-free, ribavirin-free regimen of ACH-3102, a second-generation NS5A inhibitor, and sofosbuvir in patients with treatment-naïve genotype 1 HCV. These results will be presented in a late breaker poster presentation and made available in a related press release along with development updates at The Liver Meeting 2014 (AASLD) which begins Saturday, November 8, 2014 in Boston, MA
   
• Present three posters on ACH-3422, a uridine-analog nucleotide NS5B polymerase inhibitor prodrug, at AASLD that will detail the preclinical profile of this Phase 1 direct-acting antiviral agent for HCV; and
   
• Report Phase 1 proof-of-concept results with ACH-3422 including safety following 14-day exposure in healthy volunteers and antiviral activity on treatment-naïve genotype 1 HCV patients.

"Over the course of 2014, we executed on our global HCV development plan and achieved several important milestones including the advancement of ACH-3422 into a Phase 1 trial, working with the FDA to remove the clinical hold on sovaprevir, our Phase 2 protease inhibitor, and continuing to demonstrate that, based upon results from three Phase 2 trials, ACH-3102 is a clearly differentiated NS5A inhibitor," commented David Apelian, M.D., Ph.D., Executive Vice President and Chief Medical Officer at Achillion. "As previously announced, we expect to report top-line results from our ongoing Phase 1 trial of ACH-3422 later this quarter and look forward to initiating a proprietary combination program evaluating ACH-3422, ACH-3102 and sovaprevir during 2015."

Complement Factor D Inhibitor Platform

Achillion also announced today that the Company has leveraged its internal discovery capabilities and a novel complement-related platform to develop oral inhibitors of complement factor D. Factor D is an essential serine protease involved in the complement pathway, a part of the innate immune system. The new complement platform is focused on advancing compounds that inhibit factor D, can be orally-administered, and potentially can be used in the treatment of immune-related diseases in which complement plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and age-related macular degeneration (AMD). Achillion anticipates that its platform could play a role to addressing the needs of all PNH patients, including patients who have suboptimal response to, or fail to respond to, the currently available treatment, as well as for patients suffering from other complement-mediated diseases.

Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion commented, "I am very proud of our accomplishments and we remain focused on transforming innovation into treatments that improve patients' lives. With our HCV compounds progressing through clinical development and on track to deliver additional results during the remainder of 2014, the Achillion discovery team has been focused on identifying and advancing exciting new candidates into our pipeline. We have now generated a portfolio of small molecule compounds that will be evaluated as orally administered inhibitors of complement factor D, potentially offering novel treatment options for patients with complement-related disease."