Scholar Rock Does Its Homework For Successful SMA Launch

On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.

Spinal muscular atrophy
• Source: Shutterstock

Scholar Rock has laid out regulatory and commercialization plans for its closely-watched spinal muscular atrophy (SMA) candidate, apitegromab, at the J.P. Morgan Healthcare conference in San Francisco and is eyeing a US launch by the end of the year.

More from Start-Ups & SMEs

Major Cash Injection Advances Augustine’s HDAC6 Hopes

 
• By 

The Belgian firm banks nearly €77.7m to push its Charcot-Marie-Tooth to proof-of-concept.

Roche Adds To Its Antibody Armoury With OBT Alliance

 
• By 

Deal Snapshot: The antibody-drug conjugate field continues one of the hottest dealmaking spaces and Roche continues to invest heavily. Its latest pact could be transformative for the UK firm which already has a decent number of big pharma partners.

New Duchenne Results Put Avidity On Course For Filing

 

The potential first-in-class antibody oligonucleotide conjugate could treat a form of the muscle wasting disease, and could be the first of Avidity’s trio of rare disease drugs to gain approval.

Big Pharma Backing For Sofinnova Biotech Acceleration Plans In Europe

 
• By 

The Biovelocita II fund is supported by the likes of Amgen, Bristol Myers Squibb and Pfizer

More from Therapy Areas

Japan T-Reg Venture RegCell Raises New Funds, Relocates To US

 
• By 

Kyoto-based venture moves HQ to California to expand R&D and business outreach for its regulatory T-cell technology, as it raises around $46m in public and private funding.

Axsome Scores In ADHD, But Differentiation In Generic-Heavy Market Unclear

 

The FOCUS study met its primary and key secondary endpoints, but lack of statistical significance on the higher dose and a crowded market raised questions.

GSK’s Blujepa Brings Innovation To The Antibiotic Space

 

The US FDA approved Blujepa for the treatment of uncomplicated urinary tract infections, marking the first new mechanism of action for the infection in more than 30 years.