Scholar Rock to start clinical trials in SMA

Scholar Rock to start clinical trials in SMAScholar Rock announced last week that SRK-015, the company’s lead clinical candidate, will be developed to improve muscle strength and function in patients with SMA.

What is Scholar Rock?

Scholar Rock is a biotechnology company which specialises in the discovery and development of innovative new medicines to treat a range of serious diseases with high unmet medical need, including neuromuscular diseases, cancer and fibrosis.

Scholar Rock plans to develop SRK-015 both in combination with therapies aimed at correcting the underlying genetic defect in SMA and as a single therapy in patients with certain subtypes of SMA.

How does SRK-015 work?

SRK-015 is an antibody which binds to a pre-cursor of a protein called myostatin, preventing the release of its mature form, myostatin. Myostatin prevents muscle growth.

What does SRK-015 do in SMA?

Previous work had shown that by blocking the activity of myostatin, SRK-015 increases muscle mass and force in healthy mice and prevented muscle loss in a model of atrophy.

Further pre-clinical data on the potential efficacy of SRK-015, this time in SMA, was presented in an oral presentation at the Cure SMA Annual Conference in Orlando, Florida last week:

  • SRK-015 substantially increases lean body mass in non-human primates, with a particularly notable effect on muscles with a high proportion of fast-twitch fibers, a muscle fiber type that is particularly affected in SMA.
  • In a mouse model of SMA, inhibition of myostatin activation, in combination with a SMN therapy which targets the underlying genetic defect, significantly improved the strength of the gastrocnemius relative to treatment with ‘corrector’ therapy alone.

For more information

Leave a Reply

Your email address will not be published. Required fields are marked *