Shares of Scholar Rock SRRK skyrocketed 362% on Monday after the company announced positive top-line results from the late-stage study, evaluating the efficacy and safety of its investigational muscle-targeted therapy, apitegromab, to treat spinal muscular atrophy (SMA).
Apitegromab is a fully human monoclonal antibody inhibiting myostatin activation. Myostatin is a protein that is primarily produced in skeletal muscle cells and acts as a negative regulator of muscle growth. SMA is a genetic disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. Despite progress in developing treatments targeting motor neuron loss, there remains a significant unmet need for therapies that directly address the progressive muscle weakness responsible for the decline in motor function in SMA.
SRRK’s Late-Stage SMA Study Meets Primary Goal
Per the data readout, the phase III SAPPHIRE study met its primary endpoint demonstrating a statistically significant and clinically meaningful improvement in motor function upon treatment of SMA patients with apitegromab compared with placebo at week 52. The metric used for measuring the same was the gold standard Hammersmith Functional Motor Scale Expanded (HFMSE).
Motor function outcomes showed meaningful and consistent improvements in both the main efficacy group (ages 2-12) and the exploratory group (ages 13-21), with apitegromab outperforming placebo. In the apitegromab group, 30% of SMA patients saw a 3 point or more improvement in HFMSE compared with 12.5% in the placebo group.
Patients on apitegromab also showed early motor function gains as soon as eight weeks, with further improvement at 52 weeks. Following the study, 98% of the SAPPHIRE study patient population (185 out of 188) joined the ongoing ONYX open-label extension study for the same indication.
Year to date, shares of Scholar Rock have surged 82.3% against the industry’s 2.8% decline.
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SRRK further reported that apitegromab was well-tolerated across all age groups, with no clinically significant differences in adverse events between the two evaluated dosage strengths (10 mg/kg and 20 mg/kg). The observed safety profile of the candidate was consistent with that observed in previous clinical studies. Serious adverse events were not attributed to treatment with apitegromab. Additionally, no patients discontinued the study drug due to adverse events.
Based on such encouraging results, Scholar Rock is currently gearing up to submit regulatory applications, seeking the approval of apitegromab for SMA in the United States and the EU in the first quarter of 2025.
Please note that the candidate already enjoys the Fast Track, Orphan Drug and Rare Pediatric Disease designations in the United States and the Priority Medicines and Orphan Medicinal Product designations in the EU for the same indication.
Scholar Rock anticipates presenting detailed data from the phase III SAPPHIRE study of apitegromab for SMA at an upcoming medical conference in early 2025.
Other Developmental Programs in SRRK’s Clinical Pipeline
Apart from the SMA indication,Scholar Rock is also currently evaluating apitegromab for cardiometabolic disorders. Last month, the company reported completing patient enrollment in its phase II EMBRAZE proof-of-concept study of the candidate to safely preserve lean muscle mass in individuals on GLP-1 receptor agonist therapy for obesity.
Per SRRK, the EMBRAZE study results, expected in the second quarter of 2025, will guide the development of SRK-439, an investigational selective myostatin inhibitor. The company intends to advance SRK-439 specifically for treating cardiometabolic disorders like obesity.
Scholar Rock has another candidate in its pipeline, SRK-181, an investigational selective inhibitor of latent TGFβ-1 activation, which is currently being evaluated in a phase I DRAGON proof-of-concept study to treat patients with advanced cancer across multiple tumor types. Enrollment in the DRAGON study was completed last December.
Scholar Rock Holding Corporation Price and Consensus
Scholar Rock Holding Corporation price-consensus-chart | Scholar Rock Holding Corporation Quote
Impact of SRRK News on Biohaven Stock
Interestingly, following the positive data readout from Scholar Rock’s late-stage SMA study, Biohaven BHVN also gained 10.8% on Monday. This is likely because the investors also anticipate a positive data readout from BHVN’s ongoing late-stage SMA study.
Biohaven is currently evaluating its investigational candidate, taldefgrobep alfa (BHV-2000), a novel anti-myostatin adnectin, in a phase III study for treating SMA. BHVN expects to report top-line data from the late-stage SMA study by the end of 2024.
SRRK’s Zacks Rank & Stocks to Consider
Scholar Rock currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals ANIP and Catalyst Pharmaceuticals CPRX, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share (EPS) have moved up from $4.69 to $4.81. EPS estimates for 2025 have improved from $5.37 to $5.86. Year to date, shares of ANIP have gained 1.3%.
ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Catalyst’s 2024 EPS have increased from $1.84 to $1.92. The consensus estimate for 2025 EPS has improved from $2.14 to $2.27. Year to date, shares of CPRX have soared 17.4%.
Catalyst’s earnings beat estimates in three of the trailing four quarters while matching on the remaining occasion, the average surprise being 13.88%.
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