BLUE BELL, Pa., May 09, 2019 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today reported its financial results for the three months ended March 31, 2019 and provided a portfolio update.
“We made significant progress advancing our portfolio of oral factor D inhibitors in the first quarter of 2019,” said Joe Truitt, President and Chief Executive Officer at Achillion. “Our team completed enrollment in our Phase 2 clinical trial of ACH-4471 for paroxysmal nocturnal hemoglobinuria (PNH) in combination with eculizumab. Interim data from this trial has been accepted for presentation at the New Era of Aplastic Anemia and PNH meeting in Napoli, Italy, on May 17, 2019. Our hypothesis for combination therapy is that if the alternative pathway is adequately inhibited then meaningful patient benefit can be achieved in fundamentally different ways than has been seen with C5 inhibitors alone. In addition, we expect to present the data from the recently completed Phase 2 PNH monotherapy trial at a medical meeting later this year.”
“In C3G, we have now enrolled 24 patients in our 6-month and 12-month proof of concept trials of ACH-4471,” continued Mr. Truitt. “Our goal is to assess interim efficacy and safety data from these two trials, along with a sizeable data set from real-world data derived from patients with C3G. If this data supports advancing into a Phase 3 program, we plan to meet with the U.S. Food and Drug Administration in an end-of-Phase 2 meeting during the fourth quarter of 2019. In addition, we expect to complete our ongoing ex-U.S. Phase 1 multiple ascending dose study for our more potent next-generation oral factor D inhibitor, ACH-5228, and anticipate submitting an Investigational New Drug (IND) Application in the U.S. in the fourth quarter of 2019. We believe successful development of our portfolio of factor D compounds has the potential to be transformative for patients and to deliver on the promise of alternative pathway inhibition.”
First Quarter 2019 Financial Results
For the three months ended March 31, 2019, the Company reported a net loss of $19.0 million, compared to a net loss of $20.6 million in the three months ended March 31, 2018.
Research and development expenses were $14.8 million for the three months ended March 31, 2019, compared to $14.0 million for the same period of 2018. The increase for the three months ended March 31, 2019 was primarily due to increased clinical trial costs related to ACH-4471 and ACH-5228. These amounts were partially offset by decreased non-cash stock-based compensation and personnel costs due to fewer employees as compared to the prior period.
For the three months ended March 31, 2019, general and administrative expenses totaled $5.2 million, compared to $6.0 million for the same period of 2018. The decrease for the three months ended March 31, 2019 was primarily due to decreased non-cash stock-based compensation combined with decreased intellectual property related legal fees.
Cash, cash equivalents, and marketable securities at March 31, 2019 were $254.1 million.
About ACH-4471, Complement Factor D Inhibitor for PNH and C3G
Achillion’s first-generation oral complement factor D inhibitor, ACH-4471, continues to be evaluated for safety and efficacy in multiple, global Phase 2 clinical programs in patients with PNH and C3G. Preliminary proof-of-concept has been demonstrated in both indications. The PNH program consists of a Phase 2 clinical trial evaluating ACH-4471 in combination with eculizumab in patients who are inadequately controlled or sub-optimally responding to eculizumab. Additionally, the Company continues to dose patients in its PNH monotherapy extension trial. The C3G program consists of two Phase 2 clinical trials which are a six-month blinded, placebo-controlled study and a 12-month open-label study. More information is available at http://www.achillion.com/patients-and-clinicians/.
About ACH-5228 and ACH-5548, Next Generation Complement Factor D Inhibitors
ACH-5228 and ACH-5548 are the Company’s next-generation oral factor D inhibitors currently in Phase 1 clinical trials. These compounds have demonstrated enhanced potency as well as improved pharmacokinetic properties that should allow for higher alternative pathway inhibition along with a reduced dosing frequency.
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company’s oral small molecule portfolio was discovered in its laboratories and is wholly owned. To advance its investigational product candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors. More information is available at http://www.achillion.com.
