Scholar Rock Raises $47 Million to Advance SMA Therapy to Clinical Trials


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Scholar Rock financing

The biotech Scholar Rock has obtained $47 million in new financing to move its spinal muscular atrophy therapy SRK-015 into clinical trials during the first half of 2018.

Invus led the financing round, which included Redmile Group and a number of other investors.

“We are delighted by the strong support and recognition we received in this Series C financing and the expansion of our group of world-class life sciences investors, which now includes Invus and Redmile,” Dr. Nagesh Mahanthappa, president and chief executive officer of Scholar Rock, said in a press release.

Scholar Rock believes SRK-015 can improve SMA patients’ muscle strength and movement.

The trials will look at SRK-015 as a stand-alone therapy and in combinations with other treatments. The stand-alone tests will apply to certain groups of SMA patients. The combinations will involve SRK-015 and therapies that increase the production of SMN, the protein that is deficient in SMA.

Two of the hallmarks of SMA are muscle weakness and nerve cell deterioration, which affects patients’ ability to move.

SRK-015 targets faulty versions of the protein myostatin that inhibit muscle growth. Mutations of the myostatin gene are what produce the abnormal protein.

Studies have also suggested that inhibiting the production of faulty myostatin may be a way to treat neurological disorders such as SMA.

Scholar Rock hopes SRK-015 works on both the muscle and nerve-cell dimensions of SMA.

The company plans to test SRK-015 later as a potential treatment for cancer, iron deficiency anemias and fibrosis — or tissue-scarring — diseases.

“We expect 2018 to be a critical year of growth for Scholar Rock, as we advance the first clinical candidate from our groundbreaking platform into the clinic to address an important unmet need in SMA and continue to build out our [therapy] pipeline,” Mahanthappa said.

Scholar Rock has a patent on SRK-015 that runs through May 2034. The U.S. Patent and Trademark Office granted it in September 2017 after studies in primates showed that it had the ability to improve muscle cell function and strength.