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Clinical Trial News

Harmony Biosciences' BP1.15205 Shows Breakthrough Potential as Best-in-Class Narcolepsy Treatment

  • Harmony Biosciences presented preclinical data for BP1.15205, a highly potent orexin 2 receptor agonist that demonstrated significant wake-promoting and cataplexy-suppressing effects in narcolepsy mouse models.
  • The investigational drug showed exceptional selectivity with >600-fold preference for OX2R over OX1R receptors and produced statistically significant effects at very low oral doses starting at 0.03 mg/kg.
  • A first-in-human study is planned for the second half of 2025 with topline data expected in 2026, following completion of regulatory applications to both European and U.S. authorities.
  • The compound demonstrated favorable safety profile with no adverse events observed in 3-month toxicology studies at doses up to 300 mg/kg/day across two species.

Walnut Hill Medical Selected as FDA TAP Program Advisor to Accelerate Medical Device Patient Access

  • Walnut Hill Medical has been selected as an advisor to the FDA's Total Product Life Cycle Advisory Program (TAP), launched in 2023 to transform how breakthrough medical devices reach patients.
  • The TAP program addresses delays in patient access to clinically promising medical technologies by connecting innovators with FDA experts and external advisors to streamline regulatory review and align evidence generation with payer expectations.
  • As a TAP advisor, Walnut Hill Medical will provide expertise in reimbursement strategy and market access to help companies design faster commercialization pathways for breakthrough-designated medical devices.
  • The program aims to solve persistent gaps in the innovation ecosystem where medical technologies face delays due to misaligned regulatory, coverage, and reimbursement pathways.

SeaStar Medical's SCD Therapy Receives $2 Million DoD Grant for Military Burn and Sepsis Research

  • The AREVA Institute secured a prestigious $2 million Department of Defense grant to evaluate SeaStar Medical's Selective Cytopheretic Device therapy for treating severe burns, inhalation injury, and sepsis in warfighters.
  • The three-year study, beginning July 2025, was selected as one of only four projects from 160 submissions to the Military Burn Research Program, highlighting the innovative potential of extracorporeal immunomodulation.
  • SeaStar Medical's SCD therapy is designed to neutralize destructive hyperinflammation and cytokine storms, potentially improving survival rates in both combat casualties and civilian patients with severe trauma and infection.
  • The device works alongside continuous renal replacement therapy to selectively target inflammatory immune cells and may reduce the need for dialysis while promoting long-term organ recovery.

Medable Launches AI-Powered Partner Program to Accelerate Clinical Trial Startup by 50%

AI
  • Medable has launched a Partner Program that provides contract research organizations with generative AI-driven electronic clinical outcome assessment build capabilities to reduce clinical trial timelines by at least 50%.
  • The program features Studio, an AI-powered tool for creating and reusing eCOA instruments, offering CROs flexible self-service, managed service, or hybrid support models.
  • The initiative aims to enable rapid trial-specific quote generation and pre-built digital studies for bid defense meetings, supporting Medable's vision of achieving one-day study startup.
  • Medable's digital clinical trials platform has been utilized in nearly 400 trials across 70 countries, demonstrating the company's established presence in clinical technology solutions.

Cellectar's Iopofosine I 131 Doubles Survival in Pediatric Brain Cancer Trial

Rare DiseaseOrphan Drug
  • Cellectar Biosciences reported that iopofosine I 131 achieved 5.4 months average progression-free survival in pediatric high-grade glioma patients, doubling the reported median of 2.25 months.
  • Patients receiving higher doses (minimum 55 mCi) showed 8.6 months overall survival, with three patients receiving additional cycles achieving 11.5 months average overall survival.
  • The radioligand therapy demonstrated a favorable safety profile with no cardiovascular, renal, or liver toxicities, and all hematologic adverse events were manageable and reversible.
  • The CLOVER-2 Phase 1 trial enrolled 14 pediatric patients with relapsed/refractory high-grade gliomas, including diffuse midline gliomas and diffuse intrinsic pontine gliomas.

Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma

NCT03478462Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 4/30/2019

Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG

NCT05610891Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 10/1/2023

Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma

NCT04987203Active, Not RecruitingPhase 3
AVEO Pharmaceuticals, Inc.
Posted 9/9/2021

Energenesis Biomedical Advances First-in-Class Parkinson's Disease Therapy ENERGI-F705PD Toward Phase I Data Readout

  • Energenesis Biomedical is developing ENERGI-F705PD, a first-in-class oral sustained-release therapy designed to address the underlying pathogenesis of Parkinson's disease with disease-modifying potential.
  • The company expects topline data from its Phase I trial in healthy subjects in September 2025, following successful completion of recruitment.
  • ENERGI-F705PD targets key pathological hallmarks of Parkinson's disease by reducing alpha-synuclein aggregation, enhancing antioxidant capacity, and boosting dopamine synthesis through cellular energy restoration.
  • The therapy utilizes a novel multi-pronged mechanism engaging purine salvage, glycolysis, and pentose phosphate pathways to enhance cellular ATP and antioxidant production.

Phase II Trial Combines Personalized Neoantigen Vaccines with Radiotherapy for Advanced Cancer Treatment

  • A novel phase II randomized trial is investigating the combination of individualized neoantigen peptide vaccines with precision radiotherapy in patients with advanced malignant tumors who have failed standard treatments.
  • The study employs a unique two-arm design with crossover capability, allowing control group patients to receive vaccine treatment upon disease progression, while comparing high-dose versus low-dose radiotherapy regimens.
  • The trial aims to enroll 154 patients across multiple cancer types, with progression-free survival as the primary endpoint, representing the first clinical investigation of radiotherapy combined with neoantigen vaccines for stage IV pan-cancer treatment.

Induction Chemoimmunotherapy Shows Non-Inferior Outcomes to Consolidation Therapy in Stage III NSCLC

  • A multicenter retrospective study of 262 patients with unresectable stage III NSCLC found that induction chemoimmunotherapy before chemoradiotherapy achieved comparable survival outcomes to consolidation immunotherapy with fewer treatment cycles.
  • The study demonstrated median progression-free survival of 25.5 months for induction therapy versus 25.9 months for consolidation therapy, with similar overall survival rates despite patients receiving only 4 cycles versus 10 cycles of immunotherapy respectively.
  • Induction chemoimmunotherapy showed a 66.1% objective response rate and 96.0% disease control rate, with only 4.0% of patients experiencing disease progression during the induction phase.
  • The findings suggest that upfront chemoimmunotherapy may provide a viable alternative treatment strategy for patients with high tumor burden or those ineligible for concurrent chemoradiotherapy.

Study of Pembrolizumab With Concurrent Chemoradiation Therapy Followed by Pembrolizumab With or Without Olaparib in Stage III Non-Small Cell Lung Cancer (NSCLC) (MK-7339-012/KEYLYNK-012)

NCT04380636Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 7/6/2020

A Global Study to Assess the Effects of Durvalumab With Oleclumab or Durvalumab With Monalizumab Following Concurrent Chemoradiation in Patients With Stage III Unresectable Non-Small Cell Lung Cancer

NCT05221840Active, Not RecruitingPhase 3
AstraZeneca
Posted 2/7/2022

A Study of Nivolumab and Ipilimumab in Untreated Participants With Stage 3 Non-small Cell Lung Cancer (NSCLC) That is Unable or Not Planned to be Removed by Surgery

NCT04026412CompletedPhase 3
Bristol-Myers Squibb
Posted 10/8/2019

Ascend Advanced Therapies Achieves GMP Certification for AAV Gene Therapy Quality Control in Munich

  • Ascend Advanced Therapies received GMP certification for Quality Control testing at its Munich facility following a joint inspection by the government of Upper Bavaria and the Paul-Ehrlich-Institute in September 2024.
  • The certification enables streamlined stability testing and release of higher quality AAV-based gene therapy products using advanced techniques including droplet digital PCR and automated ELISA.
  • The Munich facility can now perform commercial assays for DNA impurities, vector genome titer, and capsid titer, providing much-needed European capacity for AAV characterization and release.
  • Additional methods such as potency assays are planned to be added to the Munich GMP license through 2025, while GMP expansions continue in Alachua, Florida.

Cogent Biosciences Secures $400M Debt Financing to Advance Bezuclastinib Through Pivotal Trials

Precision MedicineRare Disease
  • Cogent Biosciences secured a $400 million non-dilutive debt financing facility with SLR Capital Partners, with an initial $50 million already drawn to support the company's growth strategy.
  • The company is on track to report results from three pivotal bezuclastinib trials in 2025, starting with SUMMIT results in July for NonAdvSM patients.
  • Additional funding tranches are tied to clinical milestones, with $100 million available upon successful SUMMIT and PEAK trial readouts and commercial success.
  • The financing enhances Cogent's financial flexibility as it prepares for the expected bezuclastinib launch in 2026 for genetically defined diseases.

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