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H.C. Wainwright maintains a Buy rating for Ultragenyx Pharma with a $95 target, citing its Phase 3 Aspire study for GTX-102 targeting Angelman syndrome. Ultragenyx shows strong revenue growth and liquidity, with a 42% increase in Q3 2024 revenue but a $134M net loss. The company aims for GAAP profitability by 2026, with potential new therapies on the horizon.

Ultragenyx submitted a BLA to the FDA for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. The BLA is supported by data showing UX111's ability to reduce heparan sulfate levels in the CSF and improve cognitive development. UX111 is well-tolerated and has various designations in the U.S. and EU. Ultragenyx also dosed the first patient in the Aspire study for Angelman syndrome.

Ultragenyx submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo syndrome type A. The BLA is supported by data showing UX111 reduces heparan sulfate in cerebral spinal fluid and correlates with improved cognitive development. UX111 aims to address the underlying enzyme deficiency causing the disease.

Ultragenyx submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo Syndrome Type A (MPS IIIA). If approved, UX111 would be the first treatment in the U.S. for this condition. The submission follows FDA's acceptance of CSF heparan sulfate as a surrogate endpoint for accelerated approval. Clinical data showed UX111 led to rapid, sustained decreases in CSF heparan sulfate levels, correlating with improved cognitive development. Most treatment-related adverse events were mild to moderate liver enzyme elevations that resolved.

Ultragenyx reported Q3 2024 revenue of $139 million, up 42% YoY, with Crysvita® at $98 million and Dojolvi® at $21 million. The company reaffirmed 2024 revenue guidance of $530-$550 million and announced Breakthrough Designation for setrusumab (UX143) in osteogenesis imperfecta. DTX401 Phase 3 data showed a 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa).

Ultragenyx plans to submit a protocol amendment for the phase 1/2/3 Cyprus2+ clinical trial evaluating UX701, an AAV vector-based gene therapy for Wilson disease, to include a higher dose cohort and optimized immunomodulation regimen. The new cohort will be added to stage 1, which has already dosed 15 patients. Six patients have fully tapered off standard of care treatment, with stabilization of nonceruloplasmin bound copper levels and improved ATP7b gene function noted. The gene therapy has been well-tolerated with no significant safety events.

FDA grants Ultragenyx's setrusumab Breakthrough Therapy designation for osteogenesis imperfecta (OI) Type I, III, or IV, targeting fracture risk reduction in patients aged two and older. Setrusumab, a sclerostin inhibitor, is being evaluated in late-stage studies for OI, with preliminary data showing significant fracture rate reduction.