Shares of biopharmaceutical company Scholar Rock Holdings (NASDAQ: SRRK) rose well over 300 per cent on Monday on the positive results form its Phase 3 clinical trial, SAPPHIRE.
The trial evaluated the effectiveness and safety of apitegromab, a novel treatment for spinal muscular atrophy (SMA), marking a potentially transformative moment for patients suffering from this debilitating condition.
SMA is a disease characterized by muscle weakness and atrophy due to lower levels of survival motor neuron (SMN) protein.
Conversely, Apitegromab is an inhibitor of myostatin activation. Scholar Rock is developing it as a targeted therapy to improve motor function in SMA patients.
The SAPPHIRE study met its primary endpoint, demonstrating both statistically significant and clinically meaningful improvements. This success has positioned apitegromab as a promising new option in the treatment landscape of SMA.
“We are thrilled that apitegromab met the primary endpoint in our Phase 3 SAPPHIRE clinical study. The results clearly demonstrate robust and clinically meaningful improvement in motor function in patients with SMA,” said Jay Backstrom, president and CEO of Scholar Rock.
The implications of these results extend beyond immediate medical benefits. For Scholar Rock, this breakthrough could mean a fast-tracked path to market. Further, the company has plans already in motion to submit a Biologics License Application (BLA) in the U.S. and equivalent documentation in the European Union.
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Drug given multiple designations by European Medicine Agency
The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations to apitegromab to treat with SMA.
The European Medicines Agency (EMA) has also awarded the drug Priority Medicines (PRIME) and Orphan Medicinal Product designations. The company also plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorization application (MAA) in Q1 2025.
The successful trial outcome validates Scholar Rock’s scientific approach and its business strategy in focusing on diseases where growth factor signalling plays a critical role. Consequently, investors and analysts alike have responded positively, with several investment firms upgrading their ratings on the stock.
“Declining motor function and hopes for reversing losses associated with muscle weakness are significant unmet needs, impacting activities of daily living, from breathing, eating, self-care, to working and social interactions,” said Kenneth Hobby, president of Cure SMA.
The broader impact on the SMA community could be profound. Current treatments for SMA have made significant strides, but there remains a need for therapies that can offer additive benefits or work in patients who might not respond well to existing options.
Apitegromab’s mechanism of action does not directly increase SMN protein but instead works to enhance muscle growth and function. Additionally, it could complement existing therapies or stand alone in treatment protocols.
Scholar Rock is continuing its analysis of the full Phase 3 SAPPHIRE data and plans to present detailed results at a medical conference in early 2025. The company will present preliminary baseline characteristics from the trial during a poster presentation at the 29th Annual Congress of the World Muscle Society. It’s scheduled for Friday, October 11, 2024, in Prague, Czech Republic.
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