PTC Therapeutics

PTC Therapeutics' Phase II trial of utreloxastat for ALS failed to meet primary endpoints, leading to trial termination. Utreloxastat was safe but lacked efficacy, prompting the company to halt further development. PTC sold its Rare Pediatric Disease Priority Review Voucher for $150 million to maintain operations.

PTC Therapeutics to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, granted by the FDA for KEBILIDI™ approval for AADC deficiency treatment.

FDA approves Kebilidi (eladocagene exuparvovec-tneq) for AADC deficiency, a rare genetic disorder affecting neurotransmitter production. Administered via four brain infusions, it's the first FDA-approved gene therapy for AADC deficiency, showing improvement in gross motor function in 8 of 12 pediatric patients at week 48.

FDA's increased flexibility in drug approvals, particularly for rare diseases, could boost biotech stocks. However, this trend raises concerns about taxpayer funding for potentially ineffective drugs and patient safety. Four biotech firms—Applied Therapeutics, Biohaven, PTC Therapeutics, and Soleno Therapeutics—could benefit from this trend, potentially seeing stock gains if their drug candidates are approved.

FDA accepts PTC Therapeutics' resubmitted NDA for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy (nmDMD), based on phase 3 trial data and STRIDE registry analyses. No review timeline provided. Therapy currently licensed in EU and Brazil. Significant improvements observed in key endpoints at 72 weeks.

Natco Pharma submitted an ANDA for Risdiplam oral solution, a generic of Genentech's Evrysdi, potentially eligible for 180 days of marketing exclusivity. Named in a lawsuit by Genentech, Roche, and PTC Therapeutics, Evrysdi had $571 million in U.S. sales for 2023.

Huntington's disease treatments face challenges due to rarity and trial failures, but recent milestones like Wave's WVE-003, Roche-Ionis' tominersen, uniQure's AMT-130, and PTC Therapeutics' PTC518 offer hope. Prilenia's pridopidine is under EMA review, while Annexon's ANX005 and Sage's dalzanemdor faced setbacks. Despite these, cautious optimism remains for effective treatments.

BioNJ launches 'Time Off for Clinical Trials' initiative, encouraging member companies to offer paid time off for employees to participate in clinical trials. Amicus Therapeutics, Bristol Myers Squibb, Genmab, PsychoGenics, PTC Therapeutics, and Sanofi are among the initial companies committed to implementing this policy.

FDA approves PTC Therapeutics' eladocagene exuparvovec for AADC deficiency, marking the first brain-administered gene therapy in the U.S. Abeona Therapeutics' BLA for EB-101 accepted by FDA. Neurogene's NGN-401 trial for Rett syndrome faces critical immune response issue. Adicet Bio initiates phase 1 trial for ADI-100 in lupus nephritis. CSL Behring to close Pasadena R&D facility, shifting focus from ex vivo gene therapy. Anixa Biosciences and Moffitt Cancer Center dose first patient in phase 1 trial for ovarian cancer therapy. Answer ALS and Cedars-Sinai release ALS data repository. MyoGene receives FDA ODD and RPD designations for MyoDys45-55 for Duchenne muscular dystrophy.

BioNJ launches 'Time Off for Clinical Trials' initiative, encouraging member companies to offer paid time off for employees to participate in clinical trials. Amicus Therapeutics, Bristol Myers Squibb, Genmab, PsychoGenics, PTC Therapeutics, and Sanofi are among the first to commit. The initiative aims to remove barriers to participation in clinical trials, particularly for hourly workers and underrepresented populations.