Achillion Reports Third Quarter 2019 Financial Results and Provides Corporate Update
– Reached an agreement to be acquired by Alexion;
transaction expected to close in first half of 2020 –
– Danicopan (ACH-4471) received Breakthrough Therapy designation for treatment of PNH;
Phase 2 combination trial top-line results to be presented at 61st ASH Annual Meeting –
– ACH-5228 Phase 1 MAD study completed;
Phase 2 trial initiation planned for first half of 2020 –
– Cash and securities of
projected Year-End Cash and securities of
“In the third quarter of 2019 we reached a number of important milestones as part of our mission to bring oral complement factor D inhibitors to patients in need,” said
As previously announced on
Mr. Truitt continued, “We are making progress on the pending acquisition of Achillion by
Key Highlights and Development Updates:
The U.S. Food and Drug Administration( FDA) has granted Breakthrough Therapy designation for danicopan (ACH-4471) for treatment in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are sub-optimal responders to a C5 inhibitor alone. The FDA’s decision was based on interim data from the Company’s Phase 2 clinical trial assessing the safety and effectiveness of danicopan in combination with intravenous eculizumab. The top-line results from the Phase 2 clinical trial of danicopan in combination with eculizumab will be presented at the 61st ASH Annual Meeting. Global regulatory discussions are ongoing with initiation of a Phase 3 program for danicopan planned for early 2020.
- The Company completed enrollment in the danicopan Phase 2 clinical trials for C3 glomerulopathy (C3G) and C3G/immune complex-mediated membrane glomerulonephritis (IC-MPGN). In the third quarter of 2019, three adolescent patients were enrolled in the 12-month single-arm open-label trial. A total of 35 patients have been enrolled in the two trials. Pending an analysis of the C3G data, the Company plans to meet with the
FDAfor an End-of-Phase 2 meeting and the European Medicines Agency(EMA) for scientific advice in 2020.
- Further evaluation of pharmacokinetic and pharmacodynamic biomarker data from the Phase 2 PNH monotherapy study for untreated PNH patients was accepted for poster presentation at the 61st ASH Annual Meeting.
- 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 plans to initiate Phase 2 development of ACH-5228 in PNH in early 2020.
Third-generation Factor D Inhibitors
- The Company continues to evaluate 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 its third-generation factor D inhibitors for clinical development in 2020.
Pending Acquisition of Achillion by Alexion
Third Quarter 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
Nine Month Financial Results
For the nine 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 potential benefits of Achillion’s third generation factor D inhibitors and its plans to nominate a third generation inhibitor for clinical development; 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; the proposed acquisition of Achillion by Alexion, including the potential contractual contingent value right payment by Alexion in connection with the acquisition; 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. Risks and uncertainties related to the proposed acquisition of Achillion by Alexion include, among other things, risks related to the fact that the proposed acquisition may not be completed due to Achillion’s or Alexion’s failure to satisfy or waive the conditions to closing the proposed acquisition (including the failure to obtain necessary regulatory approvals) in the anticipated timeframe or at all, including uncertainties as to whether Achillion’s stockholders will approve the Merger and the possibility that the acquisition does not close; the possibility that competing offers may be made; risks related to obtaining the requisite consents to the acquisition, including, without limitation, the timing (including possible delays) and receipt of regulatory approvals from various governmental entities (including any conditions, limitations or restrictions placed on these approvals and the risk that one or more governmental entities may deny approval); the risk that the businesses will not be integrated successfully; disruption from the transaction making it more difficult to maintain business and operational relationships; negative effects of the announcement or the consummation of the proposed acquisition on the market price of Alexion’s common stock, Alexion’s credit ratings and/or Alexion’s operating results; significant transaction costs or unexpected expenses; unknown liabilities; the risk of litigation and/or regulatory actions related to the proposed acquisition.
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.
Note to Investors and Security Holders
This document does not constitute a solicitation of any vote or approval. In connection with the proposed acquisition of Achillion by Alexion, Achillion intends to file with the
Certain Information Regarding Participants
Alexion, Achillion and their respective directors and executive officers may be considered participants in the solicitation of proxies in connection with the proposed transaction. Information about the directors and executive officers of Alexion is set forth in its Annual Report on Form 10-K for the year ended
Senior VP, Corporate Communications
|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||14,993||12,842||45,744||37,915|
|General and administrative||6,061||4,447||16,326||17,924|
|Total operating expenses||21,054||17,364||62,725||57,739|
|Loss from operations||(21,054||)||(17,364||)||(62,725||)||(57,739||)|
|Other income (expense):|
|Net loss per share - basic and diluted||$||(0.14||)||$||(0.12||)||$||(0.42||)||$||(0.39||)|
|Weighted average shares outstanding - basic and diluted||139,589||138,586||139,025||138,344|
|(Unaudited, in thousands)|
|September 30,||December 31,|
|Cash, cash equivalents and marketable securities||$||228,962||$||270,977|
|Total stockholders' equity||217,216||266,012|
Source: Achillion Pharmaceuticals, Inc.