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Clinical Trial News

Bezuclastinib Achieves 87% Response Rate in Pivotal Trial for Non-Advanced Systemic Mastocytosis

Rare DiseasePrecision MedicineOncology
  • Cogent Biosciences' bezuclastinib demonstrated superior symptom improvement with a placebo-adjusted difference of 8.91 points in total symptom score at 24 weeks (p=0.0002) in the SUMMIT trial.
  • The drug showed remarkable efficacy on mast cell burden, with 87.4% of patients achieving at least 50% reduction in serum tryptase compared to 0% in the placebo group.
  • Bezuclastinib exhibited a favorable safety profile with most adverse events being low-grade, supporting its potential for chronic use in systemic mastocytosis patients.
  • The company plans to submit a New Drug Application to the FDA by the end of 2025 based on these positive results from the registration-directed trial.

(Summit) a Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients with Indolent or Smoldering Systemic Mastocytosis

NCT05186753Active, Not RecruitingPhase 2
Cogent Biosciences, Inc.
Posted 6/27/2022

Orchard Therapeutics Completes Enrollment in Pivotal Gene Therapy Trial for Hurler Syndrome

Rare Disease
  • Orchard Therapeutics has completed enrollment in the HURCULES registrational trial for OTL-203, an investigational hematopoietic stem cell gene therapy for MPS-IH (Hurler syndrome), nearly one year ahead of schedule.
  • The randomized controlled trial compares OTL-203 to standard allogeneic stem cell transplant across four clinical sites in the U.S. and Europe, with primary analysis anticipated two years post-treatment.
  • Previous proof-of-concept data showed robust metabolic correction and improvements in cognitive, motor, skeletal, ocular and auditory health, addressing clinical manifestations not fully covered by current treatments.
  • MPS-IH affects approximately 60% of children born with MPS-I, a rare disease occurring in 1 in 100,000 live births, with patients rarely surviving past age 10 when untreated.

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403Active, Not RecruitingPhase 3
Orchard Therapeutics
Posted 12/11/2023

BioSapien Secures $8 Million in Extended Pre-Series A Round for Revolutionary Cancer Treatment Platform

Precision Medicine
  • BioSapien has extended its pre-Series A funding round to over $8 million, adding Globivest as a new investor alongside existing backers Global Ventures, Golden Gate Ventures, and Dara Holdings.
  • The company's flagship product MediChip™ is a 3D-printed, slow-release drug delivery platform that can be attached to any tissue with minimal systemic side effects for localized cancer treatment.
  • The funding will support product development and global expansion, with 12 new executives hired across R&D, medical affairs, regulatory, and operations including key appointments of Joseph Borrello as CTO and Sonia Ramamoorthy as Head of Medical Affairs.
  • Founded in 2018 by Dr. Khatija Ali, BioSapien aims to make precision medicine both globally impactful and locally accessible through its innovative drug delivery technology.

Scottish Health Authority Approves Theramex's Abaloparatide for High-Risk Osteoporosis Treatment

Drug ApprovalDrug Approval
  • The Scottish Medicines Consortium has recommended Theramex's Eladynos (abaloparatide) for treating osteoporosis in postmenopausal women at very high risk of fracture through NHS Scotland.
  • Over 1,000 postmenopausal women in Scotland are expected to benefit from this anabolic therapy, which promotes new bone formation through daily injections for up to 18 months.
  • Hip fractures cost the NHS £2 billion annually, with total osteoporotic fracture costs exceeding £4.6 billion and projected to reach £6 billion by 2030.
  • The approval follows NICE's previous recommendation in August 2024 for England, Wales, and Northern Ireland, ensuring equitable access across the UK.

BioCryst Pharmaceuticals Appoints Babar Ghias as CFO and Head of Corporate Development

Rare Disease
  • BioCryst Pharmaceuticals has appointed Babar Ghias as chief financial officer and head of corporate development, bringing extensive deal-making experience from rare disease companies.
  • Ghias joins from AvenCell Therapeutics where he served as CFO since 2022, and previously raised over $1 billion in capital across multiple biotechnology companies.
  • The appointment comes as BioCryst seeks to deploy capital and accelerate growth driven by commercial momentum from ORLADEYO, their oral treatment for hereditary angioedema.
  • CEO Jon Stonehouse emphasized that Ghias's expertise in capital deployment and operational experience are essential for the company's sustainable growth strategy into the next decade.

Pakistan Approves Chinese Biosimilar Cancer Drug to Expand Access to Affordable Bevacizumab Treatment

Drug ApprovalOncology
  • Pakistan's drug regulator has approved a biosimilar version of bevacizumab, a costly cancer drug, manufactured by Chinese firm TOT Biopharm and marketed by Kexing Biopharm.
  • The approval addresses critical healthcare needs in Pakistan, where over 118,000 cancer deaths occur annually, with lung, colorectal, and ovarian cancers among major contributors.
  • The biosimilar offers a more affordable alternative to Roche's Avastin, which costs several hundred dollars per dose and remains unaffordable for most patients in the country of 240 million people.
  • This development strengthens Pakistan-China health cooperation and aligns with Pakistan's 2018 drug pricing policy promoting generic and biosimilar medicines to reduce healthcare costs.

Biosynth Opens Expanded GMP Bioconjugation Facility in Berlin to Support Next-Generation Therapeutics

  • Biosynth has opened an expanded GMP bioconjugation facility at its Berlin site, significantly enhancing manufacturing capabilities for conjugate vaccines, conjugate drugs, and polymer-based drug delivery systems.
  • The new commercial suite includes class D and C cleanrooms and extends production capacity from early clinical phases through late clinical phases and commercial supply.
  • The facility expansion enables comprehensive bioconjugate services from initial process development to commercial GMP supply, supporting the advancement of next-generation diagnostic and therapeutic solutions.

Chugai and Gero Form $250M Partnership to Develop AI-Driven Antibody Therapies for Age-Related Diseases

AIMonoclonal AntibodyDrug Discovery
  • Chugai Pharmaceutical and Singapore-based Gero have entered into a joint research and license agreement to develop novel antibody therapies targeting age-related diseases using AI-driven drug discovery.
  • The collaboration combines Gero's physics-based machine learning platform for target identification with Chugai's proprietary antibody engineering technologies to create first-in-class therapeutics.
  • Under the agreement, Chugai will pay up to approximately $250 million in milestone payments plus royalties, while gaining exclusive worldwide rights to develop and commercialize antibodies for Gero-identified targets.
  • The partnership represents a strategic move by Chugai to accelerate innovation through open collaboration as part of its TOP I 2030 growth strategy focused on global first-class drug discovery.

ALS Community Files Citizens' Petition Seeking FDA Approval for NurOwn Stem Cell Therapy

Rare Disease
  • A coalition of ALS patients and families has filed a 309-page Citizens' Petition with the FDA requesting approval of NurOwn, a neurotrophically-enhanced stem cell therapy developed by BrainStorm Cell Therapeutics.
  • The petition presents unprecedented survival data showing 100% five-year survival in NurOwn's Expanded Access Program participants compared to 20% in natural ALS history, with median tracheostomy-free survival of 7 years.
  • NurOwn demonstrated statistically significant changes in 23 cerebrospinal fluid biomarkers and up to 85% slowing of ALS progression, supported by real-world evidence from clinical trials and expanded access programs.
  • The petition argues NurOwn meets approval thresholds for traditional, accelerated, or conditional approval pathways based on survival data, respiratory function preservation, and biomarker evidence.

CorFlow Therapeutics Receives FDA Approval for MOCA-II Pivotal Trial Testing Novel Heart Attack Diagnostic Technology

FDA Approval
  • CorFlow Therapeutics received FDA investigational device exemption approval for its MOCA-II pivotal trial, enabling enrollment of over 200 STEMI patients at U.S. and European research institutions.
  • The trial will validate the diagnostic accuracy of CorFlow's CoFl system in detecting microvascular obstruction during primary PCI procedures, comparing results to cardiac MRI reference standards.
  • More than half of STEMI heart attack patients have microvascular obstruction, which drives adverse clinical outcomes, yet no approved diagnostic technologies currently exist for real-time detection during interventions.
  • The technology represents a potential breakthrough in heart attack care, as someone experiences a heart attack every 30 seconds in the U.S. with approximately 800,000 cases annually.

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