On August 10, 2021 Scholar Rock (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 second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 10, 2021, View Source [SID1234586232]).
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"Scholar Rock has had significant momentum in the first half of the year with further demonstration of apitegromab’s transformative potential to improve motor function for patients with SMA, as well as continued progression of dose escalation in the DRAGON trial evaluating SRK-181’s potential to overcome resistance to checkpoint inhibitors in solid tumors," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "I look forward to working closely with this exceptional team as we continue to advance our research and clinical programs to further elucidate the potential of our scientific platform and help make a difference in the lives of patients suffering from serious diseases."
Company Updates and Upcoming Milestones
Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).
Positive Top-line Results and Additional Supportive Exploratory Analyses from the TOPAZ Trial were Presented at the Cure SMA Annual Conference. In June 2021, positive top-line data from the TOPAZ Phase 2 trial (NCT03921528) were presented by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. Treatment with apitegromab in conjunction with nusinersen in patients with Type 2 and 3 SMA led to meaningful motor function improvements of up to 20 points as measured by Hammersmith Functional Motor Scale Expanded (HFMSE). The majority (74%, 23/31) of non-ambulatory patients showed a clinical improvement, as defined by at least a 1-point increase in HFMSE. Further evidence that improvements in motor function may be attributed to apitegromab was supported by a post-hoc analysis that showed no correlation between change in HFMSE and duration of prior maintenance nusinersen therapy. In addition, 7/35 non-ambulatory patients also showed major functional achievements as measured by World Health Organization (WHO) Motor Development Milestones, with one patient achieving two and one patient achieving three new WHO motor milestones.
Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a randomized, double-blind, placebo-controlled Phase 3 trial to evaluate apitegromab as an add-on therapy to nusinersen or risdiplam in patients with non-ambulatory Type 2 and Type 3 SMA. In the TOPAZ Phase 2 trial, non-ambulatory patients experienced the largest increases in motor function (HFMSE scores) following treatment with apitegromab as add-on to chronic maintenance nusinersen. Patients with non-ambulatory Type 2 and Type 3 SMA are estimated to represent approximately two-thirds of the overall prevalent SMA patient population.
Apitegromab Granted Fast Track Designation by the FDA for the Treatment of Patients with Spinal Muscular Atrophy. In May 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for apitegromab, offering Scholar Rock eligibility to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met. Fast Track designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and get new drugs to patients earlier. In addition to Fast Track designation, apitegromab had previously received Orphan Drug and Rare Pediatric Disease designations from the FDA as well as PRIME and Orphan Medicine Product designations from the European Medicines Agency for the treatment of SMA, all recognizing the unmet medical needs of patients with SMA.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which selectively inhibiting the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential indications outside of SMA.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.
Plan to Advance to Part B Dose Expansion Portion of the DRAGON Phase 1 Proof-of-Concept Trial Mid-Year. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Part A dose escalation, which is evaluating the safety and pharmacokinetics of SRK-181 as a single-agent and in combination with anti-PD-(L)1 therapy, continues to progress to identify the recommended dose for Part B of the trial. The Company is on-track to advance to Part B dose expansion, which will consist of multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients who have demonstrated primary resistance to anti-PD-(L)1 therapy and will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy. An update on dose escalation and initial clinical data from Part A of the DRAGON trial is anticipated by year-end.
Second Quarter 2021 Financial Results
For the quarter ended June 30, 2021, net loss was $30.7 million or $0.84 per share compared to a net loss of $19.3 million or $0.65 per share for the quarter ended June 30, 2020.
Revenue was $4.6 million for the quarter ended June 30, 2021 compared to $3.9 million for the quarter ended June 30, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $25.6 million for the quarter ended June 30, 2021 compared to $17.0 million for the quarter ended June 30, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab and higher personnel and facility-related costs, partially offset by lower manufacturing costs associated with SRK-181.
General and administrative expense was $9.3 million for the quarter ended June 30, 2021 compared to $6.4 million for the quarter ended June 30, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs and professional fees.
"We are executing towards key milestones and continue to work closely with regulatory authorities to finalize the design of the Phase 3 trial for apitegromab and to progress the DRAGON trial to Part B to evaluate SRK-181 across multiple tumor types," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We ended the second quarter with approximately $282 million in cash and cash equivalents, which will allow us to achieve meaningful milestones as we continue to execute against our plan."