Freeline Therapeutics Reports FDA Clearance of Investigational New Drug Application

Jan 6, 2022 By MarketDepth

Business Headlines Healthcare Investing What's Hot

FDA Approval

Freeline Therapeutics Holdings plc (NASDAQ: FRLN) shares jumped about 10% after the clinical-stage biotechnology Company announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for FLT201 as an investigational gene therapy for the treatment of Gaucher disease Type 1.

“The FDA clearance of this IND is an important milestone for FLT201, which is the first AAV-mediated gene therapy for patients with Gaucher disease Type 1 in the clinic. Our FLT201 program harnesses our unique scientific platform capabilities – our highly potent, proprietary AAVS3 capsid, robust CMC and pre-clinical data across all our programs, and our advanced protein engineering capabilities – to develop a potentially transformative treatment for patients suffering from Gaucher disease. The entry of our third program into the clinic is important validation of the advantage of Freeline’s portfolio approach to development.”

Michael Parini, Chief Executive Officer of Freeline

The company’s advanced products candidate is FLT180a, a gene therapy product candidate that is in Phase 1/2 dose-finding clinical trials in adult males for the treatment of hemophilia B.

“This IND clearance is an important step toward bringing FLT201 to patients and is made possible by the extended safety database across our proprietary platform. Our extensive preclinical proof-of-concept studies suggest FLT201 has the potential to reach difficult to treat tissues, such as bone marrow and lung, which are not sufficiently addressed by standard-of-care enzyme replacement therapy. These data, along with the promise of sustained, endogenous production of GCase following a one-time intravenous infusion, suggest that treatment with FLT201 may be a transformative treatment that can significantly reduce or eliminate the need for enzyme or substrate replacement therapy.”

Dr. Pamela Foulds, Chief Medical Officer of Freeline

Freeline initiated the Phase 1/2 dose-finding trial of FLT201 at the end of 2021 in Europe and expects to dose two patients in the first dose cohort in the first half of 2022, with initial safety and biomarker data from the first cohort expected in Q3 2022. The Company expects to report data on all dosed patients, including those dosed in Q3, prior to year-end 2022.