Scholar Rock, a biotechnology company, has recently showcased its experimental drug, apitegromab, which targets spinal muscular atrophy (SMA), a rare genetic disease characterized by progressively worsening muscle weakness due to mutations in the SMN gene important for motor neuron health. Unlike existing therapies, apitegromab focuses directly on combating muscle atrophy by inhibiting myostatin, a protein that restricts muscle growth.
The promising results from the Phase 3 study of apitegromab, showcased significant improvements in muscle function among SMA patients, which sent the company’s shares soaring by over 300%. In response to these results, Scholar Rock has successfully raised $300 million through a stock offering to fund regulatory submissions and prepare for the drug’s potential market launch as their first product.
Existing treatments for SMA include Biogen’s Spinraza, the first approved drug that boosts SMN protein production; Novartis’s Zolgensma, a one-time gene therapy; and Roche’s Evrysdi, developed by PTC Therapeutics, an oral treatment aimed at increasing SMN protein production. However, these therapies primarily aim to preserve motor neuron function and do not directly address muscle atrophy, a gap that apitegromab aims to fill.
The pivotal trial for apitegromab involved 188 patients aged 2 to 21 years, who were already on standard treatments like Spinraza or Evrysdi. The drug, administered intravenously every four weeks, showed a clinically meaningful improvement in muscle function at 12 months as measured by a recognized scale. Notably, 30% of the patients treated with apitegromab exhibited a significant improvement in their scores, compared to only 12.5% in the placebo group. The therapy was also well-tolerated with no serious adverse events reported.
Scholar Rock’s CEO Jay Backstrom emphasized the urgency and potential transformative impact of apitegromab for SMA patients globally. The company plans to present detailed results at the World Muscle Society meeting and anticipates regulatory submissions in the U.S. and Europe by early next year. If approved, Scholar Rock aims for a commercial launch of apitegromab by the fourth quarter of 2025.
Furthermore, Scholar Rock is exploring additional applications for apitegromab, including preserving muscle mass in patients undergoing treatment with GLP-1 drugs for weight loss, another area where muscle loss is a noted side effect. The company’s ongoing research in this area aligns with its broader strategy to address muscle-related conditions and complications.
The funds raised from the recent stock offering, along with existing capital, are expected to support the commercialization of apitegromab, advance other programs, and sustain operations into the fourth quarter of 2026. This capital boost follows the company’s engagement in the stock market, where it priced over 10.2 million shares, alongside offering pre-funded warrants and an additional option for underwriters to purchase more shares, potentially raising even more funds.
As Scholar Rock progresses towards the potential launch of their myostatin-inhibiting therapy, they are not only contributing a novel approach to SMA treatment but are also setting a precedent with the first Phase 3 success of an anti-myostatin therapy, marking a significant milestone in muscular disease management. This positions Scholar Rock at a crucial juncture, potentially reshaping SMA therapy by offering a treatment that directly targets muscle health in addition to existing neuron-focused therapies.
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