Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.
“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.
Verge Genomics Inc. has entered a second big AI deal with a large drug company. Privately held Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties.
While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
Abcuro Inc. pulled down an oversubscribed $155 million series B financing co-led by Redmile Group and Bain Capital Life Sciences to advance cytotoxic T and natural killer cells therapies. Specifically, proceeds will back the phase II/III registrational trial of ABC-008, a first-in-class anti-killer cell lectin-like receptor G1 (KLRG1) antibody for inclusion body myositis (IBM) as well as fund continued development of other clinical programs.
There are plenty of companies chasing the first U.S. FDA-approved treatment for the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), but Ipsen SA stayed the course longer and won that approval Aug. 16 with Sohonos (palovarotene). Other companies have struggled to develop a treatment for FOP, which affects about 400 people in the U.S. and about 900 people worldwide. Some potential competitors are still in the clinic, trying to catch up.
The U.S. FDA has given its first approval for treating fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that creates bone formation outside the skeleton that can lead to immobility, life-threatening respiratory problems and a total locking of the jaw.
Variable muscle expression of frataxin (FXN) protein in muscle plus five adverse events (AEs) of injection-site thrombophlebitis in the multiple ascending-dose (MAD) phase I study hamstrung Design Therapeutics Inc. in its Friedreich’s ataxia (FA) bid with DT-216. Wall Street punished the Carlsbad, Calif.-based firm’s stock (NASDAQ:DSGN) with a knockdown of 70%, or $5.17, leaving shares at $2.17 when the market closed Aug. 15.
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