- Janssen HCV triple combination candidate JNJ-4178 reported 100% SVR12 after 6 weeks of therapy; global phase IIB study to begin 4Q 2016
- Achillion's phase I MAD study with oral factor D inhibitor ACH-4471 ongoing; strong complement inhibition observed in in vivo and ex vivo assays
- Company advancing small molecule candidates for the treatment of dry AMD
Achillion recognized in the third quarter of 2015 revenue of $33.8 million under the Janssen Agreement, representing a portion of the premium paid by JJDC. No revenue was recognized during the three months ended September 30, 2016.Research and development expenses were $16.7 million for the three months ended September 30, 2016, compared with $12.0 million for the same period of 2015. The increase for the three months ended September 30, 2016 was primarily due to increased manufacturing; clinical trial and consulting costs related to ACH-4471, partially offset by decreased manufacturing, clinical trial and consulting costs related to our HCV compounds which were licensed to Janssen in 2015. General and administrative expenses were $4.8 million for the three months ended September 30, 2016, compared with $4.9 million incurred during the same period in 2015. The decrease for the three months ended September 30, 2016 was primarily due to decreased business development consulting fees and corporate legal fees related to the Janssen Agreement, partially offset by increased personnel and non-cash stock costs largely related to the addition of personnel. Nine Month Results For the nine months ended September 30, 2016, Achillion reported a net loss of $57.3 million, compared to a net loss of $22.0 million in the same period in 2015. During the nine months ended September 30, 2015, Achillion recognized revenue of $34.5 million under the Janssen Agreement, representing a portion of the premium paid by JJDC. Research and development expenses were $44.1 million and $46.9 million for the nine months ended September 30, 2016 and 2015, respectively. The decrease for the nine months ended September 30, 2016 was primarily due to decreased manufacturing, clinical trial and consulting costs related to our HCV compounds, which were licensed to Janssen in 2015, partially offset by increased manufacturing, clinical trial and consulting costs related to ACH-4471. Personnel costs also increased due to the addition of personnel in our discovery and development groups.
General and administrative expenses in the period ending September 30, 2016 were $15.4 million, compared with $19.2 million incurred during the same period in 2015. The decrease for the nine months ended September 30, 2016 was primarily due to decreased consulting fees and corporate legal fees related to the Janssen Agreement, partially offset by increased personnel and non-cash stock compensation costs largely related to the addition of personnel.Complement Factor D Platform "ACH-4471 is the first factor D inhibitor to demonstrate complement alternative pathway inhibition in humans after oral dosing," said Dr. Deshpande. "Through pioneering research in complement biology, and data emerging from the ACH-4471 clinical program, we are gaining important insights into PK/PD relationships as well as in vivo biomarkers to guide development in patients. Upcoming presentations at ASH will highlight groundbreaking research with ACH-4471 regarding complement biology and factor D inhibition." Phase I Clinical Program In June 2016, Achillion initiated enrollment and dosing in an ongoing phase I multiple ascending dose (MAD) study of ACH-4471 in healthy volunteers. The goal of the MAD study is to evaluate safety and tolerability as well as to optimize the PK/PD profile and dosing regimens for further development in patients. In the three dose cohorts completed to date, strong complement inhibition using multiple in vivo as well as ex vivo assays was observed. The plasma trough concentrations of ACH-4471 exceeded the range the Company anticipates for potential treatment of patients. These current projections are based on emerging data including the benchmarking of ACH-4471 with eculizumab for inhibition of lysis of PNH red blood cells and the PK/PD profile of ACH-4471 from the phase I program. In the ongoing MAD study, Achillion is planning to assess additional dosing regimens to maintain the projected effective trough concentrations and safety of ACH-4471. To date in the MAD study, ACH-4471 has been generally well tolerated across three dose cohorts (200 mg, 500 mg or 800 mg given every 12 hours) with no treatment-related SAEs reported. Two cases of self-limited, ALT elevations (Grade 3 and 4) were observed post-treatment in the mid- and high dose groups, respectively, with neither subject exhibiting signs or symptoms of hepatic decompensation. Both subjects' ALT levels normalized without intervention during follow up. Further, no treatment-associated fever or infections were observed.Achillion anticipates announcing interim results from this phase I study in the first half of 2017.
Upcoming Presentations at the 2016 Meeting of the American Society of HematologyToday, the American Society of Hematology announced the titles of two ACH-4471 related posters to be presented at the 58th annual meeting in San Diego on December 3-6, 2016.
- 'Effect of complement inhibition by anti-C5 (eculizumab) or a small molecule inhibitor of Factor D (ACH-4471) on survival of meningococci in blood from vaccinated adults.' These data will be presented by Dr. Dan M. Granoff, UCSF Benioff Children's Hospital, a world expert on meningococcal infections.
- 'Evaluation of bacteria-mediated potential "Bystander" hemolysis of PNH red cells in vitro: No evidence of significant complement classical or lectin pathway-mediated hemolysis induced by microorganisms.' These data will be presented by Dr. Xuan Yuan from the laboratory of Dr. Robert Brodsky, Johns Hopkins University, a leading PNH expert.
About the Achillion Complement Factor D PlatformAchillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop small molecule drug candidates that are 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. Achillion's complement platform is focused on seeking to advance small molecule compounds that inhibit factor D and can potentially be used in the treatment of immune-related diseases in which complement alternative pathway plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and dry age-related macular degeneration (dry AMD). About HCV Globally, HCV infection is a leading cause of liver disease and liver related mortality. It is currently estimated that more than 150 million people are infected with HCV worldwide including approximately 3 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. Despite available treatments, there remains a significant unmet need for many patients infected with HCV. About Achillion Pharmaceuticals Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is a science-driven, patient-focused company seeking to leverage its strengths across the continuum from discovery to commercialization in its goal of providing better treatments for people with serious diseases. The company employs a highly-disciplined discovery and development approach that has allowed it to pursue best-in-class oral antiviral therapy for chronic hepatitis C (HCV) and build a platform of potent and specific complement inhibitors. Achillion is rapidly advancing its efforts to become a fully-integrated pharmaceutical company with a goal of bringing life-saving medicines to patients with rare diseases. More information is available at http://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," "target," "intend," "plan," "aim," "believe," "seek," " estimate," "can," "could" "focus," "will," "look forward," "goal," and "may" and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: Achillion's expected plans, timing, data readouts and results from ongoing and planned clinical trials of both ACH-4471 and HCV development candidates being advanced by Janssen under Achillion's collaboration with Janssen; the planned advancement of Achillion's other small molecule factor D inhibitors, including those for the treatment of dry AMD; and statements concerning Achillion's strategic goals, milestone plans, and prospects. 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: advance the preclinical and clinical development of its complement factor D inhibitors under the timelines it projects in current and future preclinical studies and clinical trials; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; obtain and maintain necessary regulatory approvals; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with third-parties, including the current collaboration with Janssen; compete successfully in the markets in which it seeks to develop and commercialize its product candidates and future products; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. Furthermore, because Janssen is solely responsible for the development and commercialization of Achillion's HCV assets under the exclusive worldwide license Achillion granted to it and has the deciding vote on all collaboration matters, Janssen generally has full discretion over all development plans and strategies and may not advance the HCV programs in the time frames Achillion or Janssen projects, or at all, including with regard to the planned phase IIb combination trial that include Achillion's licensed drug candidates. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the fiscal quarter ended June 30, 2016, and its subsequent SEC filings.
-- Financial Tables Attached --
|ACHILLION PHARMACEUTICALS INC. (ACHN)|
|Statements of Operations|
|(Unaudited, in thousands, except per share amounts)|
|Three Months Ended||Nine Months Ended|
|September 30,||September 30,|
|Research and development||16,701||11,983||44,133||46,912|
|General and administrative||4,848||4,856||15,443||19,226|
|Total operating expenses||21,549||16,839||59,576||66,138|
|Loss from operations||(21,549||)||16,981||(59,576||)||(31,607||)|
|Other income (expense):|
|Net income (loss) per share - basic||$||(0.15||)||$||0.19||$||(0.42||)||$||(0.18||)|
|Net income (loss) per share - diluted||$||(0.15||)||$||0.19||$||(0.42||)||$||(0.18||)|
|Weighted average shares outstanding - basic||136,681||136,439||136,647||121,896|
|Weighted average shares outstanding - diluted||136,681||140,024||136,647||121,896|
|(Unaudited, in thousands)|
|September 30,||December 31,|
|Cash, cash equivalents, marketable securities and interest and subscriptions receivable||$||409,550||$||460,540|
|Total stockholders' equity||401,211||449,636|
Investors:Glenn Schulman, PharmD, MPHExecutive Director, Investor RelationsAchillion Pharmaceuticals, Inc.Tel. (203) email@example.comMedia:Liz PowerSenior Director, Public RelationsAchillion Pharmaceuticals, Inc.Tel: (203) firstname.lastname@example.org