Scholar Rock announce SRK-015 orphan drug designation news and publish pre-clinical data

04 December 2018

Pharmaceutical company Scholar Rock has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion on their experimental drug for SMA, SRK-015. COMP is thus recommending that SRK-015 is designated as an Orphan Medicinal Product for SMA in the European Union (EU), which is similar to the Orphan Drug Status already approved for SRK-015 by the Food and Drug Administration (FDA) in the US (click here for more information).

Orphan Medicinal Product Designation provides incentives to companies developing drug treatments for diseases that affect fewer than five people in 10,0000 within the EU, which may help to expedite the development of SRK-015 as a potential therapeutic option for SMA.

SRK-015 is an SMN-independent, muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. SRK-015 works by specifically inhibiting an important protein called myostatin, which can restrict the growth and function of muscles. By targeting myostatin, SRK-015 can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit SMA patients.

In preparation for future trials with SMA patients, SRK-015 is currently being tested in a Phase I clinical trial in healthy volunteers (click here for more information).

Scholar Rock also recently published some pre-clinical data on the effects of an early-generation variant of SRK-015, called SRK-015P, in SMA mouse models. Although not exactly the same drug, the SRK-015P treatment has a very similar mode of action and is likely to have a comparable impact on muscles as SRK-015.

The work, which was published in the peer-reviewed scientific journal Human Molecular Genetics, showed that co-administration of SRK-015 with a drug that increases SMN levels called SMN-C1 (click here for more information) provided greater benefit than SMN-C1 treatment on its own.

The combination treatment resulted in increased size and strength of muscles, as well as improvements in bone deficiencies that have previously been reported in SMA mice.

These new data provide evidence that SRK-015 may prove beneficial to SMA patients, especially in combination with SMN-dependent therapies, such as Spinraza. Scholar Rock intend to initiate a Phase II proof-of-concept study with SMA patients in early 2019.


Further Information

Scholar Rock Press Release

Scholar Rock pre-clinical SRK-015 publication

Scholar Rock