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PTC Therapeutics

PTC Therapeutics’ results in SMA has Biogen nervous

IMAGE: PTC Therapeutics

Biogen currently has the only product on the market approved to treat spinal muscular atrophy (SMA), in Spinraza, and that treatment is pretty central to the biotech’s portfolio, bringing in $364 million in revenue in the first quarter.

So, news that a competitor had posted strong results at Phase 1, with a more convenient method of delivery came as a blow to the company – enough to send shares down over 5%.

Duchenne drug poised to cost $35,000 but with major catch

For those that have not followed the story, Emflaza was successfully taken through the FDA regulatory process by Marathon as the first new treatment for Duchenne muscular dystrophy. It seemed great news until the price was revealed, at $89,000 per year when patients and carers had previously been paying around $1,000 a year by importing the drug.

NICE recommends PTC Therapeutics Duchenne muscular dystrophy treatment

PTC Therapeutics has announced that the National Institute for Health and Care Excellence has issued final guidance recommending Translarna (ataluren) for ambulatory patients aged five and over with nonsense mutation Duchenne muscular dystrophy in connection with a managed access agreement with NHS England.

Nice extends price-negotiation talks for PTC’ muscle wasting drug

UK regulators have extended the cost negotiation talks with PTC Therapeutics (Nasdaq: PTCT) by a week for the drug to treat a muscle wasting condition. 

The National Institute for Health and Care Excellence (Nice) on Wednesday agreed to extend price discussions for Translarna (ataluren) a therapy for Duchenne Muscular Dystrophy caused by nonsense mutation before publishing its recommendation.   

NICE recommends PTC Therapeutics' Translarna to treat Duchenne muscular dystrophy

Shares in drug maker PTC Therapeutics (Nasdaq: PTCT) jumped after the National Institute for Health and Care Excellence (NICE) backed its Translarna (ataluren) to treat muscle wasting disease in paediatric patients.

The UK regulator has called the drug a ‘step change’ in the management of Duchenne muscular dystrophy (DMD), which causes progressive muscle wasting and is usually fatal by age 30.

PTC Therapeutics to cut jobs after FDA rejects Duchenne drug

PTC Therapeutics has announced plans to reduce its operating expenses that will see it cut almost a fifth of its workforce.

The news comes just weeks after the FDA rejected its drug Translarna (ataluren) for the treatment of the rare muscle-wasting condition Duchenne Muscular Dystrophy (DMD). The US regulator said there was insufficient evidence that the drug was efficacious in treating the condition, after it failed to meet primary endpoints in two late-stage trials.

PTC Therapeutics to file new Phase III data for genetic muscle disorder drug in Canada

PTC Therapeutics (Nasdaq: PTCT) said it does not expect the Canadian regulators to review its trial drug to treat a genetic muscle-wasting condition in the first half of 2016 as it plans to submit results from a new trial.

The company said it plans a New Drug Submission (NDS) following the results of the late-stage trials for its Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).

PTC Therapeutics makes series of changes

Published on: 27/01/14

Biopharma firm PTC Therapeutics has expanded its senior management team with a raft of appointments.

Robert Spiegel (pictured) becomes its chief medical officer and Murad Husain vice president, Global Regulatory Affairs.

PTC has also announced the promotions of Joseph Colacino to senior VP, Drug Discovery, and Ernest Dietel to senior VP, Quality.

In addition, Jay Barth who is their senior VP of Clinical Development is leaving the firm to pursue other opportunities.

Roche inks $490 million deal with PTC Therapeutics

Roche picture

Roche has signed a $490 million deal with specialist biotech firm PTC Therapeutics.

Roche is paying PTC $30 million upfront for rights to its spinal muscular atrophy (SMA) programme, and could add $460 million in future milestone payments. 

Roche previously partnered with PTC in 2009 and has now extended this agreement to include exclusive global rights to three pre-clinical compounds in development against SMA. 

AstraZeneca signs cancer drug discovery collaboration

AstraZeneca has signed a deal with PTC Therapeutics to use the company’s proprietary drug discovery platform to discover new cancer treatments.

New Jersey-based PTC is focused on the discovery and development of oral, small-molecule drugs that target post-transcriptional control processes. These regulate the rate and timing of protein production and are of central importance to proper cellular function.

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