1. Scrip
  2. >>R&D

With Back-To-Back FDA Holds, Solid Hopes To Regain Footing For Duchenne Candidate

 
• By Joseph Haas

The US FDA wants more information on the first patient given SGT-001, who was hospitalized with a reduced platelet count after dosing. Solid says the patient is doing well and hopes to reply to the FDA's concerns within weeks.

Yellow road sign with a blue sky and white clouds: clinical trial

Solid Biosciences Inc.'s holding pattern, ongoing since its $125m initial public offering in January, continues – but the company hopes it can get its gene therapy candidate for Duchenne muscular dystrophy back on track now that it has received a letter from US FDA clarifying the reasons for a full clinical hold of its Phase I/II trial.

The Cambridge, Mass

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from R&D

Boehringer’s Next Generation IPF Drug Underwhelms

 
• By Andrew McConaghie

The company hoped its follow-up to blockbuster Ofev could clearly improve efficacy and safety benefits, but the full Phase III results from nerandomilast’s FIBRONEER-IPF fall short of a slam dunk.

Affimed’s ASCO ‘Hail Mary’ As Survival Chances Slim

 
• By Kevin Grogan

The German biotech has been forced into insolvency and NASDAQ suspension.

In Brief: Essence Raises Ionis Tryngolza Hypertriglyceridemia Filing Hopes

 
• By Sushmita Panda

Tryngolza has met endpoints in a moderate hypertriglyceridemia trial. Positive results in severe disease in Q3 could allow for a filing for a broader patient population than its currently approved indication of familial chylomicronemia syndrome.

Apnimed’s AD109 Could Be A Wake-Up Call For Sleep Apnea Market

 
• By Joseph Haas

The first of two Phase III trials in obstructive sleep apnea showed Apnimed’s combination pill can offer a therapeutic alternative to entrenched CPAP devices.

More from Scrip

Affimed’s ASCO ‘Hail Mary’ As Survival Chances Slim

 
• By Kevin Grogan

The German biotech has been forced into insolvency and NASDAQ suspension.

Stock Watch: Bayer, Bavarian Nordic Bring European Cheer To Pharma

 
• By Andy Smith

Investors welcomed Bavarian Nordic’s and Bayer’s earnings announcements. However, both stock prices declined over the day of their reports as the impact of recent pressures weighed.

In Brief: Essence Raises Ionis Tryngolza Hypertriglyceridemia Filing Hopes

 
• By Sushmita Panda

Tryngolza has met endpoints in a moderate hypertriglyceridemia trial. Positive results in severe disease in Q3 could allow for a filing for a broader patient population than its currently approved indication of familial chylomicronemia syndrome.