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Clinical Trial News

Smartphone App Delivers Breakthrough in Huntington's Disease Clinical Trial Efficiency

  • A new smartphone-based digital motor score (HDDMS) developed by UCL and Roche researchers demonstrates twice the sensitivity of current clinical measures in detecting Huntington's disease progression.
  • The five-minute home-based assessment could reduce clinical trial participant requirements and study durations, potentially accelerating drug development for this devastating neurodegenerative condition.
  • Research involving 1,048 participants across four studies shows the digital biomarker approach offers convenient, reliable monitoring of motor symptoms outside clinical settings.

RenaissThera Advances AI-Designed Oral Obesity Drug Targeting GIPR to Lead Optimization

AIDrug Discovery
  • RenaissThera achieved a major discovery milestone in developing oral small molecule drugs targeting the glucose-dependent insulinotropic polypeptide receptor (GIPR) for obesity treatment.
  • The company's AI and machine learning platforms successfully designed and screened novel GIPR modulators that demonstrated in-vitro and in-vivo activity in mice.
  • These oral small molecules offer a more affordable and convenient alternative to existing peptide-based obesity therapies in a market projected to reach $38 billion by 2032.
  • RenaissThera is now advancing these "hits" to lead optimization and plans to push candidates toward IND-readiness next year.

Alexion Partners with Pangaea Data to Develop AI System for Hypophosphatasia Detection

AIRare Disease
  • Alexion, AstraZeneca's rare disease division, has entered a collaboration with AI platform provider Pangaea Data to develop an artificial intelligence clinical decision support system for detecting hypophosphatasia in adults.
  • The AI system will analyze electronic health records to improve detection of hypophosphatasia, a rare inherited disorder affecting bone mineralization that often remains undiagnosed due to diverse and non-specific symptoms.
  • Hypophosphatasia affects approximately one in 100,000 people in the general population, with patients typically waiting years for accurate diagnosis due to the condition's complex nature and variable clinical presentation.
  • Pangaea Data's AI platform has previously been configured to detect 42 other hard-to-diagnose conditions and will be reconfigured specifically for hypophosphatasia detection through this Alexion-funded partnership.

Taconic Biosciences Secures Exclusive CRISPR License from Helmholtz Munich to Enhance Animal Model Generation

Gene Editing
  • Taconic Biosciences has entered into an exclusive license agreement with Helmholtz Munich to acquire patented CRISPR-based gene editing technology for improved genetically modified animal model generation.
  • The partnership enhances Taconic's ExpressMODEL® platform to deliver high-quality, precise CRISPR/Cas9-generated custom animal models on an accelerated timeline to preclinical researchers.
  • The collaboration aims to accelerate the creation of precise models that uncover the genetic basis of diseases, supporting scientific discovery and paving the way for future medical breakthroughs.
  • Taconic has been a pioneer in CRISPR/Cas9 gene editing services for over a decade and strengthens its position as a leader in commercializing cutting-edge CRISPR gene editing services.

FDA Grants Fast Track Designation to Syntara's SNT-5505 for Myelofibrosis Treatment

Rare Disease
  • The US FDA has granted Fast Track designation to Syntara's SNT-5505 for treating myelofibrosis patients with inadequate response to JAK inhibitor therapy.
  • SNT-5505 represents a novel therapeutic approach by inhibiting lysyl oxidases, addressing significant unmet medical needs in this rare bone marrow cancer.
  • The designation enables more frequent FDA interactions, eligibility for Priority Review and Accelerated Approval, potentially expediting patient access to treatment.
  • Clinical studies have demonstrated that SNT-5505 improves patient quality of life with an excellent safety and tolerability profile.

MacroGenics Secures $70 Million in ZYNYZ Royalty Deal, Extends Cash Runway Through 2027

Oncology
  • MacroGenics received a $70 million upfront payment from Sagard Healthcare Partners through a royalty purchase agreement for ZYNYZ, a PD-1 inhibitor approved for anal canal carcinoma and Merkel cell carcinoma.
  • The deal extends MacroGenics' cash runway through the first half of 2027, providing financial stability as the company advances its pipeline of antibody-drug conjugates and bispecific therapeutics.
  • Under new CEO Eric Risser's leadership, MacroGenics is focusing on advancing key programs including lorigerlimab for prostate cancer, MGC026 and MGC028 ADCs for solid tumors, and preparing MGC030 for clinical trials.
  • The company reported $176.5 million in cash reserves as of June 30, 2025, with total revenue of $22.2 million in Q2 2025, primarily driven by increased contract manufacturing services.

Phase II Trial of Niraparib-Dostarlimab Combination Fails to Meet Primary Endpoint in Recurrent Head and Neck Cancer

  • A phase II trial evaluating niraparib and dostarlimab combination therapy in recurrent/metastatic head and neck squamous cell carcinoma was terminated early for futility after achieving only 20% clinical benefit versus the target of 50%.
  • The study enrolled 10 patients with a median age of 62.5, achieving a 10% overall response rate with median progression-free survival of 3.8 months and overall survival of 10.1 months.
  • Nine of the ten patients had previously failed anti-PD-1/PD-L1 therapy, highlighting the challenge of treating this patient population with poor prognosis.
  • Researchers suggest that biomarker-driven treatment selection may improve outcomes for this PARP inhibitor and immune checkpoint inhibitor combination approach.

National Resilience Winds Down Six Manufacturing Facilities Despite $2.25 Billion in Total Funding

  • National Resilience announced plans to wind down six manufacturing facilities across California, Massachusetts, and Florida, citing that capacity expansion has outpaced industry demand.
  • The biotech manufacturing startup secured an additional $250 million in bridge financing from existing investors to support its streamlined operations focused on Cincinnati and Toronto facilities.
  • The company will concentrate resources on high-growth segments including cell-based medicines, biologics, and aseptic drug products while pursuing additional debt funding for long-term growth.
  • An affiliate holding leases for the shuttered facilities is commencing legal proceedings for bankruptcy, though National Resilience itself is not filing for bankruptcy.

AriBio and Arcera Forge $600 Million Partnership for Novel Alzheimer's Drug AR1001

  • AriBio and Arcera have signed exclusive licensing and supply agreements for AR1001, an investigational oral therapy for Alzheimer's disease, with a total deal value estimated at $600 million.
  • AR1001 is a novel, once-daily oral phosphodiesterase-5 (PDE5) inhibitor currently in a global Phase 3 clinical trial (POLARIS-AD) for early-stage Alzheimer's disease treatment.
  • Arcera will have exclusive commercialization rights in Latin America, the Middle East, Southern Africa, Ukraine and select Eurasian countries, while AriBio retains manufacturing responsibilities.
  • The Phase 3 trial has reached its enrollment target with topline results expected in the first half of 2026, representing a significant milestone for disease-modifying Alzheimer's therapies.

Sentio BioSciences Announces $10.6 Million Expansion to Boost Domestic Generic Drug Manufacturing

  • Sentio BioSciences will invest more than $10.6 million to expand its Maryland Heights facility, creating 58 new jobs by 2026.
  • The expansion will increase capacity for domestic manufacturing of generic medicines, including key starting materials, APIs, and finished drug products.
  • The company currently manufactures three products and is submitting a fourth to the FDA, with expanded capabilities to include contract development services.
  • Sentio recently achieved OSHA SHARP certification, recognizing its commitment to workplace safety and operational excellence in pharmaceutical manufacturing.

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