Acceleron Pharma Announces Discontinuation of Clinical Trials Program for CMT

On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo. As a result, Acceleron is discontinuing development of ACE-083 and its clinical trials program for CMT. Delivered by . . .

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NS Pharma Launches Expanded Access Program for Viltolarsen

On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for viltolarsen, its investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. The program is open to all DMD patients . . .

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FDA Accepts NS Pharma’s NDA for Viltolarsen for the Treatment of DMD Amenable to Exon 53 Skipping

On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .

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Antisense Therapeutics Announces Positive Results from Phase 2 Study of ATL1102 in DMD

Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its primary endpoint, which was to assess the safety and tolerability of ATL1102 in patients with DMD who are non-ambulatory (unable to walk independently). After six . . .

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MDA Awards More Than $1 Million in Venture Philanthropy Funding to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia

AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a gene-replacement therapy for the disease. MVP is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and . . .

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FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of amyotrophic lateral sclerosis (ALS). The expanded approval for use with PEG feeding tubes will enable patients who not only have difficulty swallowing but also require . . .

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Amylyx Pharmaceuticals Announces Positive Results from Phase 2 Study of AMX0035 for Treatment of ALS

On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment with the company’s investigational therapy, AMX0035, in patients with amyotrophic lateral sclerosis (ALS). The study met its primary endpoint, showing a statistically significant slowing of . . .

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