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 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 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 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'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,
An artist with ALS creates beautiful portraits of people with the fatal disease
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He runs Upstate NY’s first Chick-fil-A without moving or speaking a word: ‘I’m still climbing’
Since Jimmer Szatkowski opened Upstate New York’s first Chick-fil-A in 2018, he’s been at the restaurant most every day. He’s ...
Inspiring Ogden High football coach Erik Thompson calling it a career — and what a career it was
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'Exercise juice' released by muscles helps nerves grow, study finds
In a new study of mouse cells, scientists found that both the biochemical and mechanical effects of exercise may help injured ...
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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 ...
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Macquarie Uni biotech spin out raises $16.8m Series A
Celosia Therapeutics is hoping to use gene therapy to treat neurodegenerative diseases such as Motor Neurone Disease (MND).
MaaT Pharma Announces Positive Phase 1b Results, Meeting Primary Endpoint in the Evaluation of MaaT033 in Amyotrophic Lateral Sclerosis (ALS)
MaaT Pharma Announces Positive Phase 1b Results, Meeting Primary Endpoint in the Evaluation of MaaT033 in Amyotrophic Lateral ...
IIT-BHU developing med device with potential to cure sclerosis
A team from IIT-BHU is developing a revolutionary cell-based device using 3D bioprinting technology to potentially cure ...
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Macquarie Spinout Lands $16.75M for ALS Gene Therapy
Sydney, Australia, 28 November 2024 – Celosia Therapeutics, an Australian biotech company developing advanced gene therapies for neurodegenerative diseases, has completed one of the largest Series A ...