PTC Therapeutics

Transthyretin amyloidosis cardiomyopathy drug development, including Pfizer's Vyndamax and BridgeBio's acoramidis, is a major focus. FDA decisions on acoramidis, AstraZeneca's dato-dxd for lung cancer, Vertex's vanza triple for cystic fibrosis, PTC's Upstaza for AADC deficiency, and Bristol Myers' subcutaneous Opdivo for solid tumors are imminent, with significant market implications.

PTC Therapeutics announces FDA acceptance of NDA for sepiapterin, a potential treatment for pediatric and adult phenylketonuria (PKU), based on phase 3 APHENITY trial results showing significant Phe level reductions and diet liberalization potential.

PTC Therapeutics maintained a positive outlook from Baird with a reiterated Outperform rating and a $44.00 price target after the FDA granted Fast Track Designation to PTC518 for Huntington's disease. The designation expedites the review process, acknowledging the urgent need for new treatments. PTC518 showed promising results in the PIVOT-HD study, with a 43% reduction in mutant Huntingtin protein levels. Baird's maintained price target reflects confidence in the drug's potential and the company's prospects.

FDA grants Fast Track designation to PTC518 for Huntington's disease treatment. PTC518, discovered via PTC's splicing platform, demonstrated durable dose-dependent lowering of mutant Huntingtin protein in blood cells and CSF, with favorable clinical effects and safety in PIVOT-HD study. Fast Track designation aims to expedite development of therapies addressing unmet medical needs.

PTC Therapeutics announced FDA Fast Track designation for PTC518, a potential treatment for Huntington's disease, following positive 12-month interim data from the PIVOT-HD study.

PTC Therapeutics' PTC518 for Huntington's disease granted Fast Track designation by FDA; interim data from PIVOT-HD study shows 43% reduction in mutant Huntingtin protein levels at 10-mg dose, with favorable clinical effects and good tolerability. PTC Therapeutics reports $187 million in Q2 2024 revenue, updates full-year guidance to $700-$750 million, and makes progress in clinical trials and regulatory submissions. Stock performance shows 62.12% return over the last year, but caution advised due to overbought RSI.

Savara Inc. appoints Braden Parker as Chief Commercial Officer to oversee global strategy for molgramostim in aPAP, with a potential BLA submission to the FDA in H1 2025.