Will TOPAZ Add Sheen To Scholar Rock?

(RTTNews) - Shares of Scholar Rock Holding Corp. (SRRK) have gained nearly 39 percent in the last 1 month as the company gears up to report Spinal Muscular Atrophy trial data in the coming months.

The trial in focus is TOPAZ, a phase II study of SRK-015 in children and adults with spinal muscular atrophy.

Spinal muscular atrophy is an inherited neuromuscular disease caused by a mutation in the SMN1 gene, which is located on chromosome 5 and is characterized by muscle weakness. Novartis' gene therapy Zolgensma, Biogen's Spinraza, and Roche's Evrysdi are the FDA-approved treatments for SMA.

Scholar Rock's investigational SRK-015 is designed to improve muscle strength and motor function in patients with Spinal Muscular Atrophy (SMA).

The TOPAZ trial has enrolled 57 patients, with 54 of them having completed the 6-month visit for the interim efficacy and safety analysis. The company remains on track to report interim efficacy and safety data in the fourth quarter of 2020.

The top-line data from the TOPAZ trial are expected in the first half of 2021.

The second clinical program of Scholar Rock is SRK-181, which is under a phase I proof of concept trial in patients with locally advanced or metastatic solid tumors, dubbed DRAGON.

The clinical response and safety data from the DRAGON trial are anticipated in 2021.

Also in the pipeline are a couple of investigational drugs in the preclinical and research stage of development for a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis, and anemia.

Collaborations

The company has two research collaborations - one with Janssen Biotech, Inc., a subsidiary of Johnson & Johnson, executed in 2013, and the other is with Gilead, inked in December 2018.

Financials

Scholar Rock has incurred losses since inception and as of June 30, 2020, had an accumulated deficit of $194.2 million. The company has no marketed products and revenue is generated from its two collaborations.

Net loss widened to $19.28 million or $0.65 per share on revenue of $3.90 million in the second quarter of 2020. This compared with a net loss of $12.52 million or $0.48 per share and revenue of $5.04 million in the year-ago quarter.

The company ended June 30, 2020, with cash, cash equivalents, and marketable securities of $141.2 million.

SRRK has traded in a range of $6.95 to $21.92 in the last 1 year. The stock closed Wednesday's trading at $15.42, up 4.54%.