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Regeneron's Garetosmab Achieves 90% Reduction in Bone Formation in Rare Disease Trial

Monoclonal AntibodyRare DiseaseOrphan Drug
  • Regeneron's Phase 3 OPTIMA trial of garetosmab met its primary endpoint, demonstrating a 90% or greater reduction in new heterotopic ossification lesions in adults with fibrodysplasia ossificans progressiva at 56 weeks.
  • The monoclonal antibody targeting activin A achieved over 99% reduction in total volume of new bone lesions, with the Independent Data Monitoring Committee recommending placebo patients transition to active treatment.
  • The company plans to submit garetosmab for U.S. regulatory approval by the end of 2025, positioning it as a potential first-in-class therapy for this ultra-rare genetic disorder affecting fewer than 1 in 2 million individuals.

Cadrenal Therapeutics Acquires eXIthera's Factor XIa Inhibitor Portfolio in $15M Deal

Orphan Drug
  • Cadrenal Therapeutics acquired eXIthera Pharmaceuticals' portfolio of Factor XIa inhibitors, including Phase 2-ready frunexian and IND-ready EP-7327, targeting the $38 billion global anticoagulation market.
  • The acquisition positions Cadrenal as the only company developing both novel vitamin K antagonists and Factor XIa inhibitors, addressing critical gaps in anticoagulation therapy.
  • Factor XIa inhibitors offer mechanism-based anticoagulation with high potency and selectivity, potentially reducing bleeding risks compared to current broad-spectrum anticoagulants.
  • The deal includes up to $15 million in milestone payments and royalties, with Sichuan Haisco retaining Chinese rights to frunexian following successful Phase 1 trials.

Can-Fite Reports Complete Resolution of Esophageal Varices in Decompensated Cirrhosis Patient Treated with Namodenoson

Orphan Drug
  • Can-Fite BioPharma announced complete resolution of esophageal varices in a decompensated liver cirrhosis patient treated with Namodenoson under compassionate use, confirmed by endoscopic evaluation.
  • Esophageal varices are severe complications of advanced liver disease with high bleeding risk, and their resolution in decompensated cirrhosis is uncommon, suggesting potential disease-modifying effects.
  • Namodenoson is currently in Phase III trials for hepatocellular carcinoma and Phase IIb trials for MASH, targeting the A3 adenosine receptor with high selectivity.
  • The finding provides supplementary evidence for Namodenoson's broader therapeutic potential in a market projected to reach $15 billion by 2030.

Lion TCR Receives FDA IND Clearance for First-in-Class TCR-T Therapy Targeting Chronic Hepatitis B

Orphan Drug
  • Lion TCR has received FDA IND clearance to initiate phase 1b/2 clinical trials for LioCyx-M004, marking the first TCR-T therapy to enter clinical development for chronic hepatitis B.
  • The approval represents the third major FDA milestone for LioCyx-M004, which previously received Fast Track and Orphan Drug designations for hepatitis B virus-related hepatocellular carcinoma.
  • LioCyx-M004 is an innovative mRNA-encoded autologous cell therapy that targets hepatitis B virus antigens and has demonstrated promising antiviral activity in preclinical studies.
  • The therapy addresses a global population exceeding 290 million chronic hepatitis B patients and offers potential as a functional cure for this chronic infection.

Trump Administration Withdraws DEA Marijuana Rules, Clearing Path for FDA-Compliant Cannabis Trials

Orphan DrugFDA Approval
  • The Department of Justice withdrew 16 pending rulemakings and 38 projected rules under Trump's Executive Order 14192, including key DEA marijuana research initiatives that had stalled medical cannabis development for years.
  • MMJ BioPharma, which has been waiting since 2018 for DEA registration approval despite securing FDA Orphan Drug Designations for Huntington's disease and Multiple Sclerosis trials, now faces no regulatory excuses for continued delays.
  • The withdrawal eliminates DEA's procedural shield of "pending rulemakings" and forces decisions under existing Controlled Substances Act law, potentially accelerating FDA-compliant medical marijuana research.
  • With constitutional challenges to DEA's hearing process and the removal of reform proposals, Administrator Terry Cole must now decide whether to approve compliant applications or continue blocking legitimate pharmaceutical-grade cannabis research.

Arrowhead Pharmaceuticals Files Patent Lawsuit Against Ionis Over Plozasiran FCS Treatment

Orphan DrugRare Disease
  • Arrowhead Pharmaceuticals filed a declaratory judgment complaint against Ionis Pharmaceuticals in Delaware federal court, challenging the validity of Ionis's U.S. Patent No. 9,593,333 and asserting non-infringement by plozasiran.
  • The legal action stems from Ionis's threats of patent infringement litigation against Arrowhead's investigational RNAi therapeutic plozasiran, which is currently under FDA review for treating familial chylomicronemia syndrome.
  • Plozasiran has received multiple FDA designations including Breakthrough Therapy, Orphan Drug, and Fast Track status for FCS treatment, and has demonstrated triglyceride reductions in completed Phase 3 trials.
  • Arrowhead CEO Christopher Anzalone criticized Ionis for prioritizing corporate interests over patient needs, emphasizing that FCS is a severe rare disease that can lead to potentially fatal pancreatitis.

Bioxodes Reports Promising Phase 2a Results for BIOX-101 in Intracerebral Hemorrhage Treatment

Orphan Drug
  • Bioxodes' BIOX-101 demonstrated encouraging safety and efficacy signals in a Phase 2a trial of 23 intracerebral hemorrhage patients, with all treated patients showing hematoma volume reduction.
  • The first-in-class therapeutic candidate targets Factors XIa and XIIa while providing anti-inflammatory effects, potentially addressing a devastating condition with no approved therapies and 50% mortality at 30 days.
  • The Data Monitoring Committee unanimously endorsed proceeding to Phase 2b trials, with Bioxodes planning to launch Series B financing for a potentially registrational study that could bring the drug to market by 2030.

Diamyd Medical Secures Key Patents for Precision Medicine Approach to Type 1 Diabetes Treatment

Precision MedicineOrphan Drug
  • Diamyd Medical has received patent grants from the Eurasian Patent Office and Hong Kong for insulin-based antigen treatment targeting individuals with type 1 diabetes carrying the HLA DR4-DQ8 genetic marker.
  • The company now holds comprehensive patent protection for precision medicine treatments covering up to 90% of type 1 diabetes patients through two genetic markers: HLA DR3-DQ2 and DR4-DQ8.
  • Patent protection extends until 2038 across key global markets including Europe, Eurasia, Hong Kong, and South Korea, strengthening the company's intellectual property portfolio.
  • The HLA DR3-DQ2 targeted therapy Diamyd® is currently in Phase 3 trials with FDA Fast Track Designation for both symptomatic and pre-symptomatic type 1 diabetes treatment.

Analog Pharma Canada Launches Generic Pregnancy Nausea Treatment in Canadian Market

Orphan Drug
  • Analog Pharma Canada launched PrALOG-Doxylamine/Pyridoxine, its first generic product for managing nausea and vomiting in pregnant women.
  • The launch represents a generic version of Duchesnay Pharmaceutical Group's flagship product PrDiclectin, manufactured in Quebec facilities.
  • Founded in January 2024, Analog Pharma Canada aims to improve access to affordable orphan drugs and generic medications within the Canadian healthcare system.
  • The company is part of Duchesnay Pharmaceutical Group, winner of the 2024 Life Sciences Innovation Award by ADRIQ.

LISCure BioSciences Achieves World's First FDA NDI Notification for Hair Health Probiotic Mobiome®

FDA ApprovalOrphan Drug
  • LISCure BioSciences received the world's first FDA New Dietary Ingredient (NDI) notification for Mobiome®, a proprietary hair health probiotic targeting hair follicle health.
  • Clinical trials demonstrated statistically significant improvements in hair gloss and elasticity (p<0.05, 95% CI) through multiple mechanisms including enhanced antioxidant activity and hair follicle cell proliferation.
  • The FDA NDI notification represents a critical regulatory milestone that strengthens LISCure's competitive position for global expansion in North America, Europe, and Asian markets.
  • Beyond hair health applications, LISCure is advancing a pharmaceutical pipeline including LB-P8 for primary sclerosing cholangitis with FDA Orphan Drug and Fast Track designations.

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