According to a story from gurufocus.com, the biopharmaceutical company Scholar Rock has recently announced the release of preliminary results from a phase 2 clinical trial. This clinical trial is testing the company’s investigational product candidate SRK-015, which is being tested as a treatment for spinal muscular atrophy (SMA). Scholar Rock is focused on the development of therapies for diseases in which protein growth factors are a central component of the disease mechanism.
About Spinal Muscular Atrophy
Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. Without prompt treatment, the disease is lethal in many cases. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Zolgensma. To learn more about spinal muscular atrophy, click here.
About The Study
These preliminary results focused on pharmacodynamic and pharmacokinetic findings and includes data from 29 patients. In the trial, SRK-015 is administered every four weeks intravenously to spinal muscular atrophy patients. Enrollment for the study is still ongoing and it is expected to ultimately include around 55 participants. The trial will include three cohorts. Two will receive a dose level of 20 mg/kg and the third will be randomized with either the same level or 2 mg/kg.
Researchers noted increases by up to 100 times in serum myostatin levels. This suggests that the drug is engaging in target activity and that spinal muscular atrophy patients do have significant levels of latent myostatin. Findings so far have been in alignment with results from the earlier phase 1 trial, such as low dose variability and proportionality as well as a consistent safety profile. Further interim findings from this study are expected in the first half of next year.
About SRK-015
SRK-015 is being developed as an inhibitor of myostatin activation. Myostatin is mostly expressed by muscle cells and when its gene is absent and it is not expressed it has been linked to improvements in muscle strength in various animal models. The goal then with the treatment is to improve overall muscle strength.
