Massachusetts Startup Scholar Rock Scores $36 Million to Move Drug to First Clinical Trial

Massachusetts Startup Scholar Rock Scores $36 Million to Move Drug to First Clinical Trial
January 4, 2016
By Mark Terry, BioSpace.com Breaking News Staff

Cambridge, Mass.-based Scholar Rock, announced today that it has closed on a Series B financing round worth $36 million. The round was led by Fidelity Management and Research Company. It was joined by a new investor, Cormorant Asset Management. Existing investors also participated, including Polaris Partners, Timothy Springer, a co-founder, ARCH Venture Partners, EcoR1 Capital, and The Kraft Group.

Scholar Rock focuses on a protein called myostatin, which is involved in various wasting diseases that limit muscle growth. The company’s lead compound, SRK-015, is expected to start its first clinical trial early in 2017, and would be a safety trial in healthy volunteers.

“We are excited to have such strong support in this Series B financing, from an outstanding syndicate of life sciences investors with the expertise to help us build a leading innovative biotechnology company,” said Nagesh Mahanthappa, president and chief executive officer of Scholar Rock, in a statement. “This financing recognizes the power of our supracellular activation platform to create a compelling drug pipeline in a broad array of therapeutic areas, including fibrosis, muscle disease, immuno-oncology and autoimmune disease.”

Scholar Rock was founded in early 2013. In September 2014, it announced a $20 million investment, as well as an undisclosed seed round.

SRK-015 acts on myostatin in a way similar to Novartis ’s bimagrumab, that is currently in Phase III clinical trials for an inflammatory disease called inclusion-body myositis. Other similar drugs are being developed by GlaxoSmithKline , Sanofi , Eli Lilly , Pfizer , Regeneron Pharmaceuticals , and Bristol-Myers Squibb . Various diseases being investigated include Duchenne muscular dystrophy, cancer, and a type of age-related muscle loss, sarcopenia.

No drugs that block myostatin have been approved yet. Late in December 2015, Atara Biotherapeutics announced that its drug, PINTA 745, to treat protein energy wasting (PEW) in patients with end stage renal disease (ESRD), failed its Phase II trial, failing to meet its primary endpoint. As a result, it is suspending further development of the drug.

Mahanthappa notes that Scholar Rock’s approach is slightly different than most other companies’. They typically work on developing drugs that boost muscle growth by binding to myostatin directly or by targeting myostatin’s receptor. He points out that there are numerous growth factors that have similar structures to myostatin, and that the myostatin receptor has multiple functions for other growth factors. That means, basically, that developing a drug that only blocks myostatin without unintended side effects is difficult.

Scholar Rock’s approach is to maintain myostatin’s inactive form. Speaking of Atara’s experience, Mahanthappa told Xconomy, “That drug previously showed a pretty robust effect in cancer patients, so this really speaks to the fact that you have to think about clinical indication selection very carefully.”

In addition to SRK-015, the company is working with Johnson & Johnson ’s Janssen to develop drugs for autoimmune disease and cancer. It is also evaluating possible fibrosis treatments. “There’s a lot cooking within the company right now,” Mahanthappa told Xconomy.

Scholar Rock’s supracellular activation platform is a fundamentally new and improved approach to targeting growth factors that are biologically validated mediators of a broad spectrum of diseases with high unmet clinical need,” said Amir Nashat, managing partner of Polaris Partners and chairman of the board of Scholar Rock, in a statement. “Data generated in the company’s partnership with Janssen, as well as in its proprietary SRK-015 program for the treatment of muscle disease, demonstrate that Scholar Rock’s therapeutic candidates hold the potential for significantly improved outcomes for patients suffering from previously intractable diseases.”

Back to news