PTC, ALS

PTC Scraps ALS Asset Utreloxastat After Mid-Stage Fail, Analysts Look to PKU Opportunity
Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.
PTC axes utreloxastat program after phase 2 ALS failure
But utreloxastat had little effect on ALS in the phase 2 trial. PTC powered the study to show whether twice-daily oral dosing of utreloxastat could achieve a two-point difference on ALSFRS, an instrument for evaluating the functional status of ALS patients. The drug candidate fell short of that bar.
PTC's utreloxastat fails Phase 2 clinical study for ALS
PTC Therapeutics (NASDAQ:PTCT) said a Phase 2 placebo-controlled study of its ALS drug candidate utreloxastat failed to meet its primary and secondary efficacy endpoints. The biotech company said the drug failed to demonstrate statistical significance in the slowing of disease progression,
PTC Therapeutics discontinues development of ALS drug after trial failure
PTC Therapeutics said on Tuesday it is discontinuing the development of its drug for a type of fatal neurodegenerative disease, as it failed to slow disease progression in a mid-stage trial. This marks the latest setback in a long list of roadblocks on the path to developing effective treatments against amyotrophic lateral sclerosis (ALS),
PTC Therapeutics: Phase 2 CardinALS study did not meet its primary endpoint
PTC Therapeutics (PTCT) announced that the global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing
PTC Therapeutics ALS drug fails to meet endpoints as trials shut down
The announcement means the company will be pulling the plug on the CardinALS trial after its amyotrophic lateral sclerosis drug failed to halt disease progression.
PTC Therapeutics' ALS drug fails mid-stage trial
PTC Therapeutics said on Tuesday its drug did not meet the main goal in a mid-stage trial evaluating it in patients with a type of fatal neurodegenerative disease called amyotrophic lateral sclerosis.
The Pharma Letter7h
PTC picks up $150 million via voucher sale
PTC Therapeutics has sold its rare pediatric disease priority review voucher (PRV) for $150 million, following FDA approval ...
PTC Therapeutics to sell Priority Review Voucher for $150M
PTC Therapeutics (PTCT) announced that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150M upon the closing of the transaction. PTC was granted the ...
FierceBiotech1d
PTC sells priority review voucher from Kebilidi approval for $150M
PTC Therapeutics may reeling from the failure of its amyotrophic lateral sclerosis (ALS) drug utreloxastat yesterday, but the ...
PTC Therapeutics: Sell Rating Due to Unmet Trial Goals and Overvaluation Concerns
Bank of America Securities analyst Tazeen Ahmad maintained a Sell rating on PTC Therapeutics (PTCT – Research Report) yesterday and set a price ...
PTC Therapeutics (NASDAQ:PTCT) Reaches New 12-Month High After Analyst Upgrade
PTC Therapeutics, Inc. (NASDAQ:PTCT – Get Free Report) reached a new 52-week high during mid-day trading on Wednesday after Robert W. Baird raised their price target on the stock from $48.00 to $52.00 ...
PTC Therapeutics Enters into Agreement to Sell Priority Review Voucher for $150 Million
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the ...
PTC To Sell Rare Pediatric Disease Priority Review Voucher For $150 Mln - Quick Facts
PTC Therapeutics (PTCT) has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million ...
BioPharma Dive1d
Kronos, Idorsia plan layoffs; PTC shelves ALS drug
Kronos is cutting 83% of its workforce, while Idorsia is considering cutting as many as 270 jobs. Elsewhere, PTC disclosed ...