MedPath

Key Biotech Clinical Trials and Developments to Watch in the First Quarter

The first quarter sees significant biotech developments with Ultragenyx and Praxis Precision Medicines leading the charge. Ultragenyx is advancing its Phase 3 study on setrusumab for osteogenesis imperfecta, while Praxis Precision Medicines is set to report on ulixacaltamide for essential tremor.

The biotech sector is poised for significant developments in the first quarter, with several key clinical trials and treatments advancing. Ultragenyx is at the forefront with its Phase 3 study on setrusumab, an antibody treatment targeting osteogenesis imperfecta (OI), a genetic disorder that leads to decreased bone mass and increased fragility. Setrusumab works by inhibiting sclerostin, a protein that blocks bone formation. An interim analysis of this study, focusing on the drug's ability to reduce bone fractures in OI patients, is expected early this quarter.
On another front, Praxis Precision Medicines is making strides with ulixacaltamide, a drug aimed at treating essential tremor, a neurological condition characterized by involuntary shaking. The company is set to report a pre-planned interim analysis of its Phase 3 study this quarter, marking a critical step in the drug's development and potential approval process.
These developments underscore the dynamic nature of the biotech industry and its ongoing efforts to address complex medical conditions through innovative treatments.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Eli Lilly Seeks to Join Lawsuit on Compounded Drugs

Eli Lilly aims to join a lawsuit as a defendant against a compounding trade group's litigation with the FDA over the status of its drug tirzepatide, arguing it needs to protect its interests and ensure the swift resolution of the case.

Immutep's LAG-3 Agonist Shows Promise, Percheron and PYC Update on Neurological Disease Trials

Immutep's IMP-761, a LAG-3 agonist, demonstrates a favorable safety profile in Phase I trials, potentially addressing autoimmune diseases like rheumatoid arthritis.
Percheron Therapeutics anticipates Phase 2b trial results for ATL-1102 in Duchenne muscular dystrophy, a genetic condition affecting muscle protein production.

ASX Biotechs Gear Up for Critical Phase III Trial Results in 2025

Several ASX-listed biotech companies are anticipating crucial Phase III clinical trial results in 2025, which could significantly impact their market value and future prospects.
Dimerix expects results from its ACTION3 Phase III trial of DMX-200 for focal segmental glomerulosclerosis (FSGS) by mid-2025, a rare kidney condition with no approved treatments.

Solid Biosciences Poised for Next-Gen Duchenne Muscular Dystrophy Treatment with SGT-003

Solid Biosciences' SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD), utilizes a novel AAV capsid and nNOS binding domain to enhance dystrophin expression.
Phase 1/2 INSPIRE DUCHENNE trial results for SGT-003 are expected in Q1 2025, marking a significant milestone for Solid Biosciences.

uniQure Receives FDA Nod for Accelerated Approval Pathway for Huntington's Disease Gene Therapy

uniQure secured FDA agreement to pursue accelerated approval for its Huntington's disease gene therapy, potentially expediting treatment availability.
The FDA's decision reflects a more flexible approach to genetic medicines for severe rare diseases under Peter Marks.

Roche to Acquire Poseida Therapeutics; Merck, AstraZeneca, and Novartis Report Pipeline Success

Roche will acquire Poseida Therapeutics for $1.0 billion, expanding its CAR-T therapy portfolio, targeting hematological malignancies, solid tumors, and autoimmune diseases.
Merck's Winrevair phase III study met its primary endpoint, showing a statistically significant reduction in morbidity or mortality events in advanced pulmonary arterial hypertension.

Biotech Alert: Searches spiking for these stocks today | Markets Insider

Ultragenyx's Setrusumab Receives FDA Breakthrough Therapy Designation for Osteogenesis Imperfecta

Ultragenyx's setrusumab (UX143) has been granted Breakthrough Therapy Designation by the FDA to reduce fracture risk in osteogenesis imperfecta (OI) patients.
The FDA's decision was based on positive data from the Phase 2 Orbit study and Phase 2b ASTEROID study, demonstrating a significant reduction in fracture rates.

Ultragenyx to Pursue Accelerated Approval for UX111 Gene Therapy in MPSIIIA

Ultragenyx plans to seek accelerated approval for UX111 (ABO-102), an AAV-based gene therapy, for mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome.
The FDA has aligned with Ultragenyx on using cerebral spinal fluid (CSF) heparan sulfate (HS) as a surrogate endpoint for accelerated approval.

Sarepta's DMD Therapy Elevidys Receives Accelerated FDA Approval; Other Biotech Updates

Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec) gains accelerated FDA approval for treating ambulatory pediatric Duchenne muscular dystrophy (DMD) patients aged 4-5.
Bristol Myers Squibb's Camzyos (mavacamten) is approved in the EU for symptomatic obstructive hypertrophic cardiomyopathy (HCM) based on positive Phase III trial results.

Reference News

[1]
The biotech scorecard for the first quarter: 16 stock-moving events to watch
statnews.com · Jan 6, 2025

Ultragenyx is developing setrusumab for Osteogenesis Imperfecta, with Phase 3 study results expected soon. Praxis Precis...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy