Achillion: Positive Interim Data for ACH-4471 Phase 2 Trials; Clinical Update

ACH-4471, First Generation Oral factor D Inhibitor

– Proof of Concept Validated in both PNH as Monotherapy and in Combination w/C5 Inhibitor– Proof of Mechanism Validated in C3G, End of Phase 2 Meeting Targeted for 4Q 2019

ACH-5228, Next Generation Oral factor D Inhibitor
– Data Demonstrate 3x to 4x Greater Potency and Extended Half-life
– Phase 1Multiple Ascending Dose Trial targeted to begin January 2019
– USPTO has Issued Achillion a Patent Covering ACH-5528 Composition of Matter

Achillion to Host Conference Call and Webcast Today at 4:30 p.m. EST
Access to Webcast at www.achillion.com

Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement mediated disorders, today reported interim results for the Company’s Phase 2 trials of its first-generation oral factor D inhibitor, ACH-4471, as well as Phase 1 pharmacokinetics and potency data for its next-generation factor D inhibitors, ACH-5228 and ACH-5548.

“These compelling findings underscore the momentum for Achillion and our factor D portfolio as we advance three oral small molecule compounds through clinical development. Our oral factor D inhibitors have the potential to treat patients suffering from alternative pathway mediated diseases,” said Joe Truitt, President and Chief Executive Officer at Achillion. “In C3G, we have completed patient dosing in our 14-day biomarker/dose ranging study and we are currently enrolling patients in our 6 and 12-month PoC trials. We now have 18 clinical sites open globally and plan to open additional sites in 2019. We are also delighted that the FDA has recently agreed to allow adolescents in our trials as C3G is a disease with unmet medical needs that often begins in childhood. Our C3G plan is to enroll up to 20 patients in our 6 and 12-month trials and to present our data to the FDA at an End of Phase 2 meeting in Q4 2019.”

Truitt continued, “The PNH trial data show that factor D inhibition may play an important role in the future treatment paradigm for PNH patients. Our trials have demonstrated a positive impact on patient’s hemoglobin, reticulocyte counts, LDH, FACIT-fatigue scores and reduced blood transfusions both as a monotherapy and in combination with a C5 inhibitor. Our hypothesis has been reinforced that if the alternative pathway is adequately inhibited then patient benefit can be achieved in fundamentally different ways than has been seen with C5 inhibitors. We believe this is an unmet medical need and a market segment we will continue to evaluate. Additionally, based on the Phase 1 pharmacokinetic and potency data, our next generation compounds, ACH-5228 and ACH-5548, allow for higher alternative pathway inhibition along with a reduced dosing frequency. These compounds have the potential to be transformative both for patients and Achillion and to deliver on the promise of alternative pathway inhibition across a wide spectrum of diseases.”

The Company also confirmed today an expected YE 2018 cash and marketable securities balance of approximately $270 million and a projected 2019 cash burn of $80-85 million.

https://www.marketscreener.com/ACHILLION-PHARMACEUTICALS-35614/news/Achillion-Reports-Positive-Interim-Data-for-ACH-4471-Phase-2-Trials-and-Provides-Clinical-Developmen-27765648/