Inozyme's INZ-701 Shows Promise in ENPP1 Deficiency Trial with Sustained Phosphate Increases
• Interim data from Inozyme Pharma's ENERGY 3 pivotal trial demonstrates INZ-701's potential to modify disease course in pediatric ENPP1 Deficiency patients, with sustained phosphate increases and favorable safety profile.
• The trial is progressing well with no patient dropouts, dose adjustments, or discontinuations, and remains on track for topline data in Q1 2026, positioning INZ-701 to potentially become the first approved therapy for this rare disease.
• Inozyme has appointed Petra Duda, M.D., Ph.D. as Chief Medical Officer, bringing over two decades of expertise in rare disease clinical development as the company advances toward late-stage development milestones.
Design Therapeutics Appoints Dr. Chris Storgard as Chief Medical Officer to Advance GeneTAC® Pipeline
• Design Therapeutics has appointed Chris Storgard, M.D., as Chief Medical Officer, bringing over two decades of leadership in drug development with experience advancing multiple assets through global regulatory approvals.
• Dr. Storgard previously served as CMO at ADARx Pharmaceuticals and Heron Therapeutics, where he secured U.S. and European approvals for several products in oncology and acute care.
• The appointment comes at a strategic time as Design Therapeutics advances its portfolio of GeneTAC® small molecules targeting serious degenerative genetic diseases, including its lead program for Friedreich ataxia.
Novartis's Oral MS Drug FTY720 Shows Promising Two-Year Efficacy Data, Potential 2010 Launch
• Novartis's experimental oral multiple sclerosis drug FTY720 (fingolimod) demonstrated significant reduction in disease relapses and progression over a two-year study period compared to placebo.
• The lower 0.5mg dose showed comparable efficacy to higher doses with improved safety profile, leading Novartis to pursue regulatory approval for this dosage.
• Novartis plans simultaneous FDA and EU submissions by end of 2009, with potential US market launch as early as 2010 pending expedited review.
Tolebrutinib Receives FDA Breakthrough Therapy Designation for Non-Relapsing Secondary Progressive Multiple Sclerosis
• The FDA has granted Breakthrough Therapy designation to tolebrutinib for adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS).
• The designation is based on positive results from the HERCULES phase 3 study, which demonstrated a 31% delay in disability progression compared to placebo.
• Tolebrutinib is the first brain-penetrant BTK inhibitor to receive this designation for MS, addressing a critical unmet need in delaying disability progression.
• Regulatory submissions for tolebrutinib are being finalized in the US and EU, with ongoing studies for primary progressive MS anticipated in H2 2025.
Patient-Centric Approaches and Technology Reshape Clinical Trial Landscape, Industry Experts Report
• Only 39% of clinical trial sites achieve enrollment targets according to Tufts Center, highlighting critical recruitment challenges in drug development.
• Industry leaders from INC Research, DrugDev, and EMD Serono emphasize the importance of non-traditional partnerships and patient-focused technology solutions to improve trial participation.
• Experts advocate for incorporating patient feedback through advisory boards and focus groups, while cautioning that technology solutions must be tailored to specific patient populations' needs.
© Copyright 2025. All Rights Reserved by MedPath