amyotrophic lateral sclerosis
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After ALS Drug Fails in Phase 3, Amylyx Weighs Pulling the Product From Market
Amylyx Pharmaceuticals co-CEO Justin Klee previously said the company would withdraw ALS drug Relyvrio from the market if it failed its Phase 3 clinical trial. The biotech has set an eight-week timeline to evaluate its next steps.
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Sanofi, Denali Neuro Drug Fails Mid-Stage Trial in ALS; MS Study Is Continuing
Sanofi, which is leading development of the Denali Therapeutics-partnered molecule, disclosed little about the clinical trial results in ALS. But this Phase 2 failure follows the 2020 clinical trial pause of a different partnered molecule that addresses the same target.
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Merck Neuroscience Acquisition Brings a Startup With Parkinson’s, ALS Programs
Merck’s acquisition of Caraway Therapeutics adds programs for genetically defined neurodegenerative diseases. One of those programs addresses a target that was the focus of a different startup purchased by Merck in 2019.
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BrainStorm Cuts 30% of Staff to Refocus on ALS Cell Therapy’s New Phase 3 Trial
A month after an FDA advisory committee voted against recommending approval of BrainStorm Cell Therapeutics’ stem cell therapy for ALS, the company is preparing to try again with a new Phase 3 study. To help pay for it, the biotech is restructuring and laying off 30% of its staff.
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BrainStorm’s ALS Data Fail to Persuade FDA Advisors, Who Vote Down the Stem Cell Therapy
NurOwn, BrainStorm Cell Therapeutics’ experimental stem cell therapy for ALS, did not win the backing of an independent panel of FDA advisors. Many advisory committee members said they want to see more data from another clinical trial, the same guidance the FDA has given the biotech for nearly three years.
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Biogen Drug Wins Accelerated FDA Nod for Treating Rare Genetic Form of ALS
The Biogen amyotrophic lateral sclerosis drug, Qalsody, treats patients whose disease is driven by mutations to a gene called SOD1. It’s the first drug approved to target a genetic cause of ALS.
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Cytokinetics Stops ALS Program After Analysis Finds No Evidence of Effect
Cytokinetics drug reldesemtiv has failed a pivotal clinical trial in amyotrophic lateral sclerosis. The small molecule had previously failed in Phase 2, but the biotech believed adjustments could lead to better results in Phase 3.
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Drug Approvals in 2022: A Recap of Notable FDA Regulatory Decisions
Fewer new drugs won FDA approvals in 2022 compared to previous years, but some of the products that passed regulatory muster are indicative of trends expected to continue into 2023. For example, more gene and cell therapies are on track to face regulatory decisions in the coming year.
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FDA Again Spurns BrainStorm’s ALS Data, This Time With Official Refusal
BrainStorm Cell Therapeutics’ stem cell therapy for amyotrophic lateral sclerosis received an FDA refuse-to-file letter, correspondence that informs a company its application seeking approval is incomplete. The notice means BrainStorm might need to run another clinical trial.
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Trayt Health Seeks to Increase Access to Diagnoses and Treatments
CEO Malekeh Amini explains how Trayt Health can bridge the gap for patients seeking neurological care.
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Cost of Amylyx ALS drug revives pricing debate for new meds in rare diseases
In pricing its new amyotrophic lateral sclerosis drug Relyvrio, Amylyx Pharmaceuticals chose a sum that’s just below the current cost of the last ALS drug to win FDA approval. But a drug price watchdog group argues that prices should be set based on therapeutic value, not according to the price of existing medications.
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Amylyx ALS drug’s winding regulatory path leads to long-awaited FDA approval
An Amylyx Pharmaceuticals drug developed to slow the progression of amyotrophic lateral sclerosis is now approved, making it just the third FDA-approved treatment for the disease. Amylyx will market its new product under the name “Relyvrio.”
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Biogen bolsters case for ALS drug under FDA review with new published data
A Biogen drug designed for a genetically defined form of amyotrophic lateral sclerosis led to patient improvement at a 12-month analysis, according to data now published in the New England Journal of Medicine. Biogen had already filed an application seeking FDA approval of the ALS drug, tofersen; a regulatory decision is expected by January.
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FDA sets decision date for Amylyx Pharma ALS drug that improves neuron survival
The FDA has accepted Amylyx Pharmaceuticals’ submission for amyotrophic lateral sclerosis drug AMX0035 and it expects to issue a regulatory decision by late June. A lot will happen before then. Amylyx recently filed for an IPO and the company’s drug is slated for an advisory committee meeting ahead of an FDA decision.
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Artificial Intelligence, BioPharma
Eli Lilly joins Verge Genomics’ $98M round as AI-discovered ALS drug nears clinic
Three life science companies unveiled Series B rounds of funding Thursday, early Christmas gifts that top $219 million combined. Along with Verge Genomics, the other companies that raised new capital are Tasso and Brainomix.
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ALS drugmaker steps up with FDA filing, but yet another biotech stumbles in clinic
Amylyx Pharmaceuticals has submitted its amyotrophic lateral sclerosis (ALS) drug for FDA review based on positive Phase 2 data after discussions with the agency over the summer. Meanwhile, a Clene Nanomedicine ALS drug failed its Phase 2 study, but the biotech points to encouraging results in a subgroup of patients.