Achillion Reports Second Quarter 2019 Financial Results and Provides Corporate Update
– Danicopan (ACH-4471) Phase 2 combination therapy interim data showed clinically meaningful improvements in laboratory parameters of PNH; End-of-Phase 2 meeting activities initiated with
– ACH-5228’s near complete and sustained inhibition of the alternative pathway and its tolerability support US IND submission Q4 2019 –
– Cash and securities of
“We continued to advance our complement factor D inhibitors in the second quarter of 2019 by efficiently executing on our global development and regulatory strategies, while continuing to strengthen our intellectual property position. As we maintain discipline on our spending, we continue to increase our chemistry, manufacturing, and controls (CMC) capabilities in preparation for our expanding portfolio,” said
Key Highlights and Development Updates:
- The ongoing Phase 2 PNH clinical trial assessing the safety and effectiveness of danicopan (ACH-4471) in combination with intravenous eculizumab is expected to be completed in the third quarter of 2019. Interim data, presented at The
New Eraof Aplastic Anemia and PNH Meeting in May 2019, demonstrated clinically meaningful improvements in laboratory parameters of PNH including hemoglobin, reticulocyte count, and bilirubin, as well as meaningful improvements in health-related quality of life FACIT-Fatigue scores relative to baseline scores on eculizumab. The addition of danicopan nearly eliminated the patients’ needs for blood transfusions in this study. The full data set is expected in the fourth quarter of 2019. Dosing with ULTOMIRIS™ (ravulizumab) has been initiated in the extension study in combination with danicopan.
- The Company completed enrollment in the danicopan Phase 2 clinical trials for C3 glomerulopathy (C3G) and C3G/immune complex-mediated membrane glomerulonephritis (IC-MPGN). A total of 32 patients have been enrolled in the two trials; 13 C3G patients in the 6-month double-blind, placebo-controlled trial and 19 C3G/IC-MPGN patients in the 12-month single-arm open-label trial. Pending an analysis of the C3G data, the Company plans to meet with the
U.S. Food and Drug Administration( FDA) for an End-of-Phase 2 meeting and the European Medicines Agency(EMA) for scientific advice, in 2020.
Next-generation Factor D Inhibitors
- Results from the completed ACH-5228 Phase 1 multiple ascending dose study demonstrated that ACH-5228, when dosed 120 mg twice a day (BID) or higher, achieved near complete and sustained alternative pathway (AP) inhibition with a mean value of >95% at steady state concentrations as measured by AP Hemolysis and AP Wieslab assays. In the study, ACH-5228 was generally well-tolerated over the dose ranges tested, which included the doses expected to be evaluated in Phase 2 clinical trials. The Company expects to submit an investigational new drug (IND) application to the
FDAfor ACH-5228 in the fourth quarter of 2019.
- The Company has identified a series of third-generation factor D inhibitors, with unique pharmacokinetic attributes, which are believed to provide additional optionality and durability for our factor D development program. The Company plans to nominate one of the third-generation factor D inhibitors for clinical development in 2020.
Second Quarter 2019 Financial Results
For the three months ended
For the three months ended
Non-cash stock compensation expense totaled
Cash, cash equivalents and marketable securities as of
Six Month 2019 Financial Results
For the six months ended
General and administrative expenses were
Non-cash stock compensation expense totaled
About the Achillion Complement Factor D Portfolio
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop oral small molecule drug candidates that are inhibitors of complement factor D. Factor D is an essential serine protease involved in the alternative pathway (AP) of the complement system, a part of the innate immune system. Achillion's complement platform is focused on seeking to advance oral small molecules that inhibit the AP and can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role. Potential indications currently being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
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,” “continue,” “goal,” “strategy,” “objective,” “may,” “potential,” and similar expressions to identify such forward-looking statements. These forward-looking statements include statements about: the potential benefits of factor D inhibition as a treatment for complement-mediated diseases, including danicopan (ACH-4471) for PNH; the potential benefits of, and indications for, Achillion’s compounds that inhibit factor D, including danicopan and ACH-5228; Achillion’s belief that its portfolio of compounds could expand factor D portfolio opportunities, provide strategic optionality or create significant value; the status of enrollment in Achillion’s ongoing clinical trials; Achillion’s expectations regarding the advancement of, and timeline for reporting results from, clinical trials of its product candidates (including danicopan and ACH-5228) as well as its ability to advance additional compounds; Achillion’s expectations regarding the timing of regulatory interactions and filings; Achillion’s anticipated cash expenditures for 2019 and the sufficiency of its existing cash resources; and other statements concerning Achillion’s strategic goals, efforts, 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: demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its product candidates, including danicopan and ACH-5228; 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; whether interim results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials; enroll patients in its clinical trials on its projected timelines; obtain and maintain patent protection for its product candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals, and the granting of orphan designation does not alter the standard regulatory requirements and process for obtaining such approval; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration and other commercial agreements with third-parties; 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. These and other risks are described in the reports filed by Achillion with the
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.
Senior VP, Corporate Communications
|ACHILLION PHARMACEUTICALS INC. (ACHN)|
|Statements of Operations|
|(Unaudited, in thousands, except per share amounts)|
|Three Months Ended||Six Months Ended|
|June 30,||June 30,|
|Research and development||15,932||11,023||30,750||25,072|
|General and administrative||5,109||7,463||10,266||13,478|
|Total operating expenses||21,041||18,561||41,671||40,375|
|Loss from operations||(21,041||)||(18,561||)||(41,671||)||(40,375||)|
|Other income (expense):|
|Net loss per share - basic and diluted||$||(0.14||)||$||(0.12||)||$||(0.28||)||$||(0.27||)|
|Weighted average shares outstanding - basic and diluted||138,761||138,426||138,739||138,221|
|(Unaudited, in thousands)|
|June 30,||December 31,|
|Cash, cash equivalents and marketable securities||$||241,305||$||270,977|
|Total stockholders' equity||231,643||266,012|
Source: Achillion Pharmaceuticals, Inc.