Rare Daily Staff

Scholar Rock, a biotechnology company focused on discovering and developing drugs that selectively modulate growth factor activation it completed a $47 million Series C financing.

New investor Invus led the round with Redmile Group also participating as a new investor. All of the existing investors in Scholar Rock also participated in this financing, including Polaris Partners, Timothy Springer, ARCH Venture Partners, EcoR1 Capital, The Kraft Group, Fidelity Management and Research Company, and Cormorant Asset Management.

Proceeds from the financing will be used to advance SRK-015, the company’s lead clinical candidate for spinal muscular atrophy and other neuromuscular disorders, into clinical development in the first half of 2018.

SMA is a rare, and often fatal, genetic disorder that affects approximately 1 in every 10,000 births. The condition is caused by defects in the SMN1 gene that produces SMN, a protein important for the survival and function of lower motor neurons. Deterioration and loss of lower motor neurons that innervate skeletal muscle lead to significant muscle atrophy, particularly in fast-twitch fibers. Muscle weakness is the most common and prominent feature of SMA, leaving many patients suffering from difficulty in performing many basic motor functions.

While there has been meaningful progress in the development of therapeutics that address the underlying SMA genetic defect, there continues to be a high unmet need for therapeutics that directly address muscle atrophy. The company said directly targeting the weakening of skeletal muscle may lead to improvements in muscle strength and motor function that could positively impact patients with SMA.

SRK-015 is a selective and local inhibitor of the supracellular activation of latent myostatin. Myostatin is a growth factor that is expressed primarily in skeletal muscle cells, is a genetically validated target that regulates muscle mass. Scholar Rock is actively working to advance SRK-015 into clinical trials that will evaluate the potential to improve muscle strength and motor function in patients with SMA.

Scholar Rock plans to develop SRK-015 both in SMA patients who are on therapies aimed at upregulating the expression of SMN and as monotherapy in certain subpopulations of SMA patients.

The company also expects to use the funding to progress additional development candidates in disease areas such as fibrosis, immuno-oncology, and anemias associated with iron restriction.

“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 pipeline,” said Nagesh Mahanthappa, president and CEO of Scholar Rock.

January 3, 2018

Photo: Nagesh Mahanthappa, president and CEO of Scholar Rock

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