Scholar Rock Holding Corp

Scholar Rock Holding Corporation secured FDA approval on September 11 for ISEMBYLD, apitegromab-mstn, to treat spinal muscular atrophy in adults and children aged two and older currently receiving survival motor neuron 2-targeted therapy. In the Phase 3 SAPPHIRE trial, children aged 2 to 12 receiving the approved 10 mg/kg dose showed a 2.2-point advantage over placebo after one year on the Hammersmith Functional Motor Scale-Expanded, with a gain of at least three points occurring in 34.2% of treated patients versus 13.5% receiving placebo. Scholar Rock said its U.S. launch was underway, with shipments expected in the coming days, supported by a patient-support program for insurance coverage, financial assistance, and infusion logistics. Fractures occurred in 9% of patients receiving the approved dose versus 2% receiving placebo, and the safety information warns of fractures, including serious fractures, with clinicians possibly stopping treatment following a fracture. Insider Monkey's database showed 51 hedge funds holding Scholar Rock at the end of 2Q2026, up from 49 funds three months earlier.

Scholar Rock Holding's modelled fair value has edged higher to US$62.06 from US$59.31 following the FDA approval of ISEMBYLD and a wave of analyst revisions. Several firms, including BMO Capital, Citi, Truist, Raymond James, Evercore ISI, Piper Sandler, Wedbush and H.C. Wainwright, have lifted their price targets into the US$70s and even low US$80s, citing the breadth of the ISEMBYLD label and its use as a muscle-targeted treatment on top of SMN2 therapies. JPMorgan anchors its target at US$63, while Barclays downgraded the stock to Equal Weight even as it raised its target to US$58, arguing the approval outcome was largely priced in. The updated valuation assumes long-term revenue growth of 684.10%, up from 638.02%, a net profit margin of 17.73% versus 18.92%, a future P/E of 134.46x versus 140.37x, and a discount rate of 7.51% versus 7.22%.

U.S. stock index futures fell sharply on Monday, with technology shares under pressure after several leading artificial intelligence executives called for a slower pace of AI development. By 05:52 ET, Dow futures had fallen 150 points, or 0.3%, S&P 500 futures had dropped 63 points, or 0.8%, and Nasdaq 100 futures had slumped 531 points, or 1.8%. Rumble shares surged as much as 28% in premarket trading after reports that Anthropic had agreed to a $13.7 billion computing contract with the company, a deal Rumble had disclosed in an August securities filing without identifying the customer. Anthropic CEO Dario Amodei called on AI companies to slow the advancement of model capabilities in an essay posted on X on Saturday, warning that increasingly capable AI agents could create significant economic damage if misused, and OpenAI CEO Sam Altman and xAI chief Elon Musk said they agree with his concerns. Nvidia fell more than 2% in premarket trading, while Meta and Amazon each dropped more than 1%, and chipmakers Intel, AMD and Marvell Technology fell nearly 6%, 5% and 6%, respectively, even as software names ServiceNow, Adobe and Workday rose about 3%, 2.5% and 2.5%. Scholar Rock shares surged 9.1% to $60.45 after the FDA approved ISEMBYLD, also known as apitegromab-mstn, as the first and only muscle-targeted treatment for spinal muscular atrophy in patients aged two and older receiving an SMN2-targeted therapy, ahead of the September 30, 2026 PDUFA deadline. Elmet Group shares jumped 30.8% after announcing a $450 million committed investment from the U.S. Department of War to accelerate domestic tungsten manufacturing, while Olema Pharmaceuticals slid 15.1% to $8.68 after AstraZeneca said its Phase 3 SERENA-4 trial of camizestrant failed to show a statistically significant improvement in progression-free survival, and MARA Holdings fell 5.7% to $11.30 after JPMorgan double-downgraded the bitcoin miner to Underweight from Neutral and cut its price target to $11 from $13.

Scholar Rock has removed Catalent Indiana from all commercial fill-finish roles for apitegromab, with the FDA continuing to review its U.S. BLA using an alternate, previously inspected facility and the company preparing for launch upon a potential decision by the September 30, 2026 PDUFA date. The company also reset its global regulatory plan by withdrawing and planning to resubmit its European filing with the new facility, while securing PMDA agreement to file a Japanese NDA for apitegromab without local clinical trials, an unusual path that could simplify entry into one of the world's larger rare disease markets. Scholar Rock's narrative projects $402.0 million revenue and $76.0 million earnings by 2029, implying an earnings increase of about $484.7 million from -$408.7 million today. Three Simply Wall St community valuations span roughly US$59 to US$191 per share, showing how far apart individual expectations can sit.

Scholar Rock Holding Corporation is removing the Catalent Indiana fill-finish facility, now part of Novo Nordisk, from its apitegromab Biologics License Application after the FDA classified an April 2026 site inspection as Official Action Indicated. The company pivoted exclusively to its second fill-finish facility, submitted the data package ahead of schedule, and confirmed robust commercial supply, keeping the apitegromab BLA on track for its September 30, 2026 PDUFA date. Scholar Rock reported a narrower-than-expected Q2 2026 net loss of $0.84 per share, or $109.9 million, and exited the quarter with $492 million in cash. BMO Capital raised its price target to $76 from $70, citing confidence ahead of the apitegromab decision. Novo Nordisk, the parent of the Catalent site, reported an 11% increase in adjusted operating profit to DKK 33.38 billion, but its reported operating profit dropped 16% to DKK 27.06 billion due to non-cash impairment charges of DKK 6.3 billion and unfavorable comparisons to prior-year US 340B rebate provision reversals, leading Citi to lower its price target to DKK 310 from DKK 330 and call the quarter messy.

Scholar Rock announced that the FDA review of its apitegromab Biologics License Application for spinal muscular atrophy will progress solely with the company's second fill-finish facility, after Catalent Indiana received an Official Action Indicated classification from the FDA. The company received notification on August 7, 2026, that the FDA classified the April 2026 site inspection of Catalent Indiana as OAI, and under FDA guidance, Scholar Rock will remove that facility from the BLA. The data package for the second fill-finish facility, agreed upon at a March 2026 Type C meeting, has been submitted ahead of schedule, and robust commercial supply is ready for launch upon potential approval by the September 30, 2026 PDUFA action date. Scholar Rock is also engaging with the European Medicines Agency on including the second facility in its Marketing Authorisation Application.

Several biotech stocks reached 52-week highs on July 10, 2026, driven by regulatory updates and quarterly results. American Well Corporation hit $10.16, closing at $9.91, after a Nature report validated its SilverCloud platform and it facilitated a DarioHealth insurer agreement, though full-year 2026 revenue is expected at $195 million to $205 million, below 2025's $249.3 million. Cue Biopharma reached $45.50 and announced a roughly $50 million private placement, while advancing CUE-221 in a Phase 2 allergy study and planning an IND for CUE-401. ImmuCell climbed to $11.59 after reporting unaudited second-quarter sales of $7.2 million, an 11.5% year-over-year increase, with $4.9 million from Tri-Shield First Defense. Scholar Rock hit $58.49, up 3.33%, as its BLA for Apitegromab in SMA is under FDA review with a decision expected by September 30, 2026. Faeth Therapeutics touched $36.89, advancing the PIKTOR combination therapy in a Phase 1/2 breast cancer trial with interim data due in 2027 and a Phase 2 endometrial cancer trial with topline data expected in the second half of 2026.

Scholar Rock announced it will present new clinical data and research updates at two upcoming neuromuscular disease conferences. The presentations will feature analyses from the Phase 3 SAPPHIRE trial and the Phase 2 FORGE study of its investigational drug apitegromab. At the Cure SMA Annual Research and Clinical Care Meeting in Orlando, the company will share post hoc analyses of apitegromab in nonambulatory type 2 or 3 spinal muscular atrophy, an assessment of motor scale impacts on patient independence, and an evaluation of baseline characteristics on efficacy. At the FSHD Society's International Research Congress in Chicago, Scholar Rock will present the trial design for the Phase 2 FORGE study evaluating apitegromab in adults with facioscapulohumeral muscular dystrophy.