Inozyme Pharma | MedPath

BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder

6 days ago

• BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701. • INZ-701 is a Phase 3 enzyme replacement therapy being developed for ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones with no currently approved treatments. • The first pivotal data readout for INZ-701 in children is expected in early 2026, with potential regulatory approval and market launch in 2027.

Eli Lilly Partners with Rznomics in $1.3B Deal to Develop RNA Editing Therapy for Hereditary Hearing Loss

7 days ago

• Rznomics Inc. has secured a potential $1.35 billion global license option agreement with Eli Lilly to codevelop an RNA editing-based gene therapy targeting hereditary hearing loss. • Under the partnership structure, Rznomics will lead initial R&D efforts to discover and develop the therapy, while Lilly will handle subsequent development and commercialization phases. • The deal represents a significant expansion of Lilly's gene therapy pipeline for hearing disorders, though specific details regarding up-front payments and option terms remain undisclosed.

Inozyme's INZ-701 Shows Promise in ENPP1 Deficiency Trial with Sustained Phosphate Increases

8 days ago

• 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.

Takeda Spotlights Three Late-Stage Readouts as Potential "Inflection Point" Amid Vyvanse Generic Competition

13 days ago

• Takeda Pharmaceutical is positioning three upcoming late-stage clinical trial readouts as a strategic "inflection point" to offset revenue losses from Vyvanse's generic competition. • The Japanese pharmaceutical giant is focusing on new product launches and pipeline advancement to maintain growth momentum, with particular emphasis on rare disease and neuroscience therapeutic areas. • Industry analysts view Takeda's pipeline strategy as critical for the company's long-term financial stability, as it navigates the challenging transition period of losing exclusivity for one of its top-selling medications.

Inozyme's INZ-701 Shows Promise in ENPP1 and ABCC6 Deficiencies

4 months ago

• Inozyme Pharma's INZ-701 demonstrated improved survival rates in infants with ENPP1 Deficiency, with 80% surviving beyond one year compared to a historical 50%. • The ENERGY 3 pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency has completed enrollment, with topline data expected in early 2026. • Preliminary regulatory support has been received for the ASPIRE pivotal trial of INZ-701 in children with ABCC6 Deficiency, targeting major adverse clinical events. • INZ-701 was well-tolerated in trials, showing substantial reductions or stabilization of arterial calcifications and improvements in heart function in treated patients.

Tenpoint Therapeutics' BRIMOCHOL™ PF Shows Positive Phase 3 Results for Presbyopia

4 months ago

• Tenpoint Therapeutics' BRIMOCHOL™ PF met primary endpoints in the BRIO-II Phase 3 trial, demonstrating statistically significant near vision improvement over 8 hours. • The study showed BRIMOCHOL™ PF was well-tolerated with no serious adverse events during the 12-month at-home dosing period. • BRIMOCHOL™ PF also demonstrated statistically significant improvement in ocular hyperemia compared to carbachol alone and reduced pupil size. • Tenpoint plans to file a new drug application with the FDA in the first half of 2025, potentially launching in 2026.