On May 14, 2019 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the quarter ended March 31, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 14, 2019, View Source [SID1234536295]).
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"2019 is off to a great start with the initiation of patient dosing in our Phase 2 TOPAZ trial of SRK-015 in SMA and the presentation of additional preclinical data outlining the potential of SRK-181 to expand the number of cancer patients who can benefit from checkpoint blockade therapies," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "We remain focused on advancing these exciting programs while building an organization and team dedicated to the mission of developing novel therapies that can make a meaningful difference in the lives of patients."
Company Highlights and Upcoming Milestones
SRK-015 Program:
Commenced Patient Enrollment and Dosing in the TOPAZ Phase 2 Clinical Trial for SRK-015. In May, Scholar Rock announced the initiation of patient enrollment and dosing in the Phase 2 proof-of-concept trial to assess the safety and efficacy of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Approximately 55 patients are anticipated to be enrolled in the U.S., Canada, and Europe across three distinct and parallel cohorts and treated with SRK-015 either as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy. The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA, over a 12-month treatment period.
Preliminary pharmacokinetics (PK) and pharmacodynamics (PD) data from a subset of patients are anticipated by the end of 2019. Interim safety and efficacy results for a subset of patients with six months of treatment exposure are expected in the first half of 2020 with top-line data for the 12-month treatment period expected beginning in the fourth quarter of 2020.
Plan to Identify Second Indication for SRK-015 in 2020. Scholar Rock continues to see multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 Program:
Presented Additional Preclinical Data Further Supporting the Potential of SRK-181 to Overcome Primary Resistance to Checkpoint Blockade Therapy (CBT). At the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, Scholar Rock presented additional preclinical data showing that co-administration of SRK-181-mIgG1 (murine version of SRK-181) with an anti-PD1 antibody can drive tumor regression or control, resulting in a significant survival benefit compared to anti-PD1 monotherapy. This was demonstrated in the MBT-2 (bladder cancer) and Cloudman S91 (melanoma) mouse models that express TGFβ1, as well as in the EMT6 (breast cancer) mouse model, which expresses both TGFβ1 and TGFβ3 isoforms. Immune response data also revealed that combination treatment leads to infiltration and expansion of CD8+ T cells and a reduction of immunosuppressive myeloid cells, suggesting TGFβ1’s multiple contributions to primary resistance to CBT.
Plan to Initiate Phase 1 Clinical Trial of SRK-181 in Patients with Solid Tumors in Mid-2020. In March 2019, Scholar Rock selected SRK-181, a highly specific inhibitor of TGFβ1 activation, as the first product candidate in its TGFβ1 cancer immunotherapy program based on the strength of preclinical data and human translational insights. Scholar Rock plans to develop SRK-181 for the treatment of tumors resistant to CBTs, such as anti-PD(L)1 antibodies, and to initiate a Phase 1 trial in patients with solid tumors in mid-2020.
First Quarter 2019 Financial Results
For the quarter ended March 31, 2019, net loss was $10.8 million or $0.42 per share compared to a net loss of $8.9 million or $3.18 per share for the quarter ended March 31, 2018.
Research and development expense was $10.7 million for the quarter ended March 31, 2019 compared to $6.7 million for the quarter ended March 31, 2018. The increase year-over-year reflects preclinical and manufacturing costs for SRK-181, research costs associated with earlier pipeline programs, and increased personnel-related costs, slightly offset by a decrease year-over-year in manufacturing costs associated with SRK-015.
General and administrative expense was $4.1 million for the quarter ended March 31, 2019 compared to $2.3 million for the quarter ended March 31, 2018. The increase year-over-year was primarily attributable to increased headcount, stock compensation, and higher operational fees associated with being a public company.
As of March 31, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $159.7 million, compared to $175.6 million as of December 31, 2018.