CAMBRIDGE, Mass. - Scholar Rock (NASDAQ: SRRK), a biopharmaceutical company, today announced positive results from its Phase 3 SAPPHIRE clinical trial, evaluating apitegromab in patients with spinal muscular atrophy (SMA). The study met its primary endpoint, showing a statistically significant improvement in motor function in patients treated with apitegromab compared to those receiving a placebo.
The trial, which included patients aged 2-12, demonstrated a 30.4% improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) in those treated with apitegromab, versus 12.5% in the placebo group. This motor function benefit was observed as early as 8 weeks and sustained through 52 weeks. The trial also included an exploratory group of patients aged 13-21, where the results favored apitegromab over placebo.
Apitegromab was generally well-tolerated, with a safety profile consistent with previous studies. There were no significant differences in adverse events between the 10 mg/kg and 20 mg/kg doses, and no serious adverse events were attributed to the treatment. Following the trial, the majority of patients opted to continue in the ONYX open-label extension study.
Based on these findings, Scholar Rock plans to submit a Biologics License Application in the US and a marketing authorization application in the European Union in the first quarter of 2025. The drug has already received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Priority Medicines (PRIME) and Orphan Medicinal Product designations from the EMA.
The full data from the SAPPHIRE trial is set to be presented at an upcoming medical conference in early 2025, with preliminary baseline characteristics to be shared at the 29th Annual Congress of the World Muscle Society on October 11, 2024, in Prague.
Apitegromab is a monoclonal antibody designed to inhibit myostatin activation, targeting muscle growth to potentially improve motor function in patients with SMA. While not yet approved for use, these trial results suggest apitegromab could become a significant treatment option for SMA, a disease characterized by progressive muscle weakness and motor neuron loss.
This article is based on a press release statement from Scholar Rock.
In other recent news, Scholar Rock has made significant strides in its drug development programs. The biopharmaceutical company has announced the appointment of Beth Shafer, Ph.D., as Chief Business Officer, a role in which she will steer the company's long-term corporate and business development strategy. This strategic move comes as the company advances its pipeline of therapies in areas like cardiometabolic disorders.
Scholar Rock's Phase 2 TOPAZ extension study involving nonambulatory patients with Spinal Muscular Atrophy (SMA) reported encouraging 48-month data. Following these developments, BMO Capital Markets has expressed increased confidence in the forthcoming SAPPHIRE trial results. The company has also made progress with its apitegromab EMBRAZE study in obesity, with the initial data now expected in the second quarter of 2025, ahead of the previous mid-2025 guidance, due to the study enrolling participants ahead of schedule.
On their recent earnings call, Scholar Rock emphasized their on-track milestones, particularly with the development of apitegromab and SRK-439. These advancements, along with the sustained benefits reported from the TOPAZ trial and promising results from the SRK-439 program, have reinforced the company's confidence in their strategic pipeline. These are the recent developments in Scholar Rock's ongoing efforts to bring new treatments to market.
InvestingPro Insights
As Scholar Rock (NASDAQ: SRRK) announces positive results from its Phase 3 SAPPHIRE clinical trial for apitegromab, investors may be interested in additional financial insights. According to InvestingPro data, Scholar Rock has a market capitalization of $593.84 million, reflecting its position as a relatively small biopharmaceutical company with significant growth potential.
InvestingPro Tips reveal that Scholar Rock holds more cash than debt on its balance sheet, which could be crucial for funding further research and development of apitegromab and other pipeline candidates. This financial stability is particularly important as the company prepares for potential regulatory submissions in 2025.
However, it's worth noting that Scholar Rock is currently not profitable, with a negative gross profit of $150.76 million over the last twelve months as of Q2 2024. This is not uncommon for biotech companies in the development stage, but it underscores the importance of the positive SAPPHIRE trial results for the company's future prospects.
The stock has experienced a significant decline, with a -48.44% price return over the past six months. This volatility may reflect the high-risk, high-reward nature of biotech investments, especially as companies approach critical milestones like the recent trial results.
For investors seeking a more comprehensive analysis, InvestingPro offers 9 additional tips for Scholar Rock, providing a deeper understanding of the company's financial health and market position.
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