Ultragenyx México, S. de R.L. de C.V.

Up to 3,500 FDA staffers received termination notices following a Supreme Court ruling that found the government's HHS overhaul to be lawful.

• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.

Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.

• Ultragenyx reported preliminary 2024 revenue of $555 million to $560 million, exceeding previous guidance, driven by strong sales of Crysvita and Dojolvi. • The company anticipates total revenue between $640 million and $670 million in 2025, projecting continued growth and pipeline advancement. • Key clinical programs are progressing, including setrusumab for osteogenesis imperfecta (Phase 3) and GTX-102 for Angelman syndrome (Phase 3). • Ultragenyx expects a PDUFA decision for UX111 in Sanfilippo syndrome type A and plans to file a BLA for DTX401 in Glycogen Storage Disease Type Ia in 2025.

• Ultragenyx has commenced the Phase 3 Aspire study to evaluate GTX-102, an experimental treatment for Angelman syndrome, in children aged 4 to 17. • The Aspire study is a randomized, sham-controlled trial with a 48-week primary efficacy analysis period, measuring cognitive improvement via the Bayley-4 cognitive raw score. • Ultragenyx is also anticipating an interim analysis from the Phase 3 Orbit study for setrusumab, expected around the end of 2024 or early 2025. • Analysts maintain a strong buy consensus on Ultragenyx stock, with price targets ranging from $48 to $140 per share, reflecting optimism about the company's pipeline.

Ultragenyx has initiated the Phase 3 Aspire study to evaluate GTX-102 for Angelman syndrome, a rare neurogenetic disorder.

The FDA has granted priority review to Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA).

• Ultragenyx seeks accelerated FDA approval for UX111, a gene therapy for MPSIII, based on Phase 1/2/3 trial data. • Arbor Biotechnologies' CRISPR-based therapy ABO-101 receives clearance for US trial in Primary Hyperoxaluria Type 1. • Allogene Therapeutics' ALLO-329 cleared by FDA for Phase 1 trial in rheumatology indications including lupus. • uniQure progresses in trial for SOD1-ALS gene therapy AMT-162, advancing to the second cohort enrollment.

Ultragenyx presented Phase 1/2 data supporting the Phase 3 Aspire study of GTX-102 for Angelman syndrome at the FAST Global Science Summit.

Ultragenyx announced positive Phase 1/2 data for GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, at the FAST Global Science Summit.