Scholar Rock Secures U.S. Patent for Potential SMA Therapy Targeting Myostatin


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Scholar Rock SRK-015

The U.S. Patent and Trademark Office has issued a patent for antibodies that selectively bind and inhibit myostatin – a protein that naturally prevents muscle mass growth.

This new patent granted to Scholar Rock will protect its drug candidate, SRK-015, which the Cambridge, Massachusetts, company is developing to improve muscle strength and motor function in patients with spinal muscular atrophy (SMA) and other motor disorders.

“With this broad patent coverage and exclusivity through May 2034, Scholar Rock is strongly positioned to develop SRK-015 as the first and only clinical candidate that works through this unique mechanism of action,” Atsuko Polzin, head of intellectual property at Scholar Rock, said in a press release. that called the patent issuance an “important milestone.”

Previous studies have showed that people with mutated myostatin genes also have significantly larger muscle mass. This suggests that inhibiting myostatin’s activity could potentially treat several motor illnesses, including SMA.

Preclinical testing in non-human primates has shown SRK-015’s potential to improve muscle cell function and strength. Animals given SRK-015 improved lean muscle mass as well as the function of some specific muscle fibers that are often simpaired in many SMA patients.

Scholar Rock is working to take its lead candidate for treatment of SMA into clinical trials. The company plans to evaluate SRK-015’s safety and effectiveness in combination with other therapies designed to correct the genetic defects that lead to SMA. By mid-2018, Scholar Rock also intends to address SRK-015’s potential as a standalone therapy in patients with certain subtypes of the rare genetic disorder.

“Targeting the activation of protein growth factors [such as myostatin] offers a number of important potential benefits that we aim to realize in the clinic in our efforts to build a world-leading pipeline of medicines to treat patients suffering from a range of serious diseases, including SMA,” said Nagesh Mahanthappa, president and CEO of Scholar Rock.

The recently issued patent will provide broad protection for SRK-015 and other therapeutic antibodies that use the same mechanism of action. These antibodies can be explored as therapeutic strategies for several neuromuscular and other diseases.