REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline
• 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 Advances Antisense Therapy for Angelman Syndrome to Phase 3 Following Promising Clinical Results
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 Announces Positive 2024 Revenue and Advances Rare Disease Pipeline
• 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 Initiates Phase 3 Aspire Study of GTX-102 for Angelman Syndrome
• 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 Doses First Patient in Phase 3 Aspire Study of GTX-102 for Angelman Syndrome
Ultragenyx has initiated the Phase 3 Aspire study to evaluate GTX-102 for Angelman syndrome, a rare neurogenetic disorder.
Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A Receives FDA Priority Review
• 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). • The FDA's decision is expected by August 18, 2025, and the agency is not planning an advisory committee meeting for this application. • UX111 has demonstrated statistically significant improvements in cognitive and communication skills, correlated with reduced heparan sulfate levels in cerebrospinal fluid. • UX111, if approved, would be the first-ever treatment for Sanfilippo syndrome type A, addressing a critical unmet need for this rare, neurodegenerative disease.
Advancements in Gene and Cell Therapies Target Diverse Diseases
• 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's GTX-102 Shows Promise in Angelman Syndrome Phase 1/2 Data
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's GTX-102 Shows Positive Phase 1/2 Data for Angelman Syndrome, Phase 3 Enrollment to Begin by Year-End
Ultragenyx announced positive Phase 1/2 data for GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, at the FAST Global Science Summit.
Ultragenyx Reports Positive Q3 2024 Results and Clinical Updates
• Ultragenyx's Q3 2024 total revenue increased by 42% year-over-year to $139 million, driven by Crysvita and Dojolvi sales growth, reaffirming its 2024 revenue guidance of $530-$550 million. • The FDA granted Breakthrough Therapy Designation for setrusumab (UX143) in osteogenesis imperfecta, based on promising Phase 2 clinical trial results showing a significant reduction in fracture rate. • Phase 3 follow-up data for DTX401 in glycogen storage disease type Ia (GSDIa) demonstrated a substantial 62% mean reduction in cornstarch intake among crossover patients. • Ultragenyx anticipates filing a J-NDA for Dojolvi in Japan by mid-2025 and a BLA submission for DTX401 in mid-2025, marking significant progress in expanding treatment options for rare diseases.