Drugs

BrainStorm Gets FDA Buy-In on Special Protocol

BrainStorm Cell Therapeutics says it has received an FDA written agreement under a Special Protocol Assessment on the design of its planned Phase 3b trial of NurOwn in amyotrophic lateral sclerosis (ALS). The SPA agreement “validates the clinical trial protocol and statistical analysis” of the planned trial, “demonstrating their adequacy for addressing objectives that support a future BLA,” it says. “We believe that having this SPA in place will help de-risk certain regulatory aspects of the NurOwn clinical program.” The primary efficacy endpoint is the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating total score from baseline to Week 24, the company says.

BrainStorm submitted the SPA request following an in-person meeting that BrainStorm held with FDA in December after the company withdrew its BLA for NurOwn, a move the company says was coordinated with the agency. A BrainStorm statement at the time said the agency had invited it to request an expedited face-to-face meeting to discuss a path forward for NurOwn, which includes an additional pivotal trial.

BrainStorm expects to begin the Phase 3b study later this year after reviewing the protocol with investigators, securing study site IRB approvals, and engaging with appropriate members of the ALS community. The trial (Study BCT-006-US) will be a two-part, multicenter study designed to assess the efficacy and safety of NurOwn in patients who are earlier in the course of their disease and having the onset of ALS symptoms, the company says. The first part of the study is a double blind, placebo-controlled trial for 24 weeks. All eligible patients who complete the first part will have the option of entering the second part, an open-label extension where all participants will receive three repeated intrathecal injections of NurOwn once every eight weeks.

The company says the NurOwn technology platform (autologous MSC-NTF cells) “represents a promising investigational therapeutic approach to targeting disease pathways important in neurological disorders. MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process to secrete neurotrophic factors to target specific neurodegenerative diseases.”

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome