MedPath

Clinical Trial News

PleoPharma's PP-01 Shows Significant Efficacy in Phase 2b Trial for Cannabis Withdrawal Syndrome

  • PleoPharma's PP-01, a dual-mechanism investigational agent containing nabilone and gabapentin, significantly reduced Cannabis Withdrawal Scores compared to placebo (P < 0.02) in a Phase 2b trial.
  • The treatment demonstrated 5-fold greater cannabis abstinence two weeks post-treatment compared to placebo in patients with Cannabis Use Disorder.
  • PP-01 has received FDA Fast Track designation and could become the first FDA-approved treatment for Cannabis Withdrawal Syndrome, addressing an unmet need for 19.2 million Americans with Cannabis Use Disorder.
  • The company is preparing to advance PP-01 into Phase 3 trials following these positive results from the CAN-002 study.

U.S. Pharmacopeia Recognizes Mass Photometry as Key Method for AAV Gene Therapy Quality Standards

  • The U.S. Pharmacopeia has officially recognized mass photometry as a key orthogonal method for characterizing adeno-associated virus (AAV) reference standards in gene therapy quality control.
  • Mass photometry is specifically referenced in the new USP general chapter <1067> as a recommended method capable of distinguishing between empty, full, and partially filled AAV capsids.
  • USP's new AAV8 reference standards were characterized using mass photometry alongside four other analytical methods in a comprehensive multi-laboratory study.
  • This recognition follows similar acknowledgment by the British Pharmacopeia in 2024, establishing mass photometry as an accepted analytical tool for AAV characterization.

KRAS G12C NSCLC Patients Face Limited CNS Treatment Options Despite 40% Brain Metastases Rate

Targeted Therapy
  • Central nervous system metastases affect approximately 40% of patients with KRAS G12C-positive non-small cell lung cancer, creating significant management challenges.
  • KRAS G12C inhibitors demonstrate 40% to 43% intracranial response rates in untreated brain metastases, but these rates remain below 50%.
  • Stereotactic radiosurgery often becomes the preferred approach for CNS lesions when systemic options are exhausted after platinum-based chemotherapy and immunotherapy.
  • KRAS G12C mutations represent 12% to 14% of all NSCLC diagnoses, with treatment decisions heavily dependent on PD-L1 expression levels.

Memo Therapeutics to Present Phase II Results for First-in-Class BK Polyomavirus Treatment at World Transplant Congress

Monoclonal Antibody
  • Memo Therapeutics will present interim Phase II results for potravitug, a monoclonal antibody targeting BK polyomavirus infection in kidney transplant recipients, at the World Transplant Congress in August 2025.
  • The SAFE KIDNEY II trial enrolled 95 patients across 22 U.S. sites to evaluate potravitug's efficacy against BKPyV infection, which affects up to 50% of kidney transplant recipients.
  • Potravitug represents a potential first-in-class therapy for BKV infection, addressing a significant unmet medical need with no currently approved treatment options available.
  • The drug received FDA fast-track designation in May 2023, recognizing the high medical need in this indication with market potential estimated at up to $2 billion annually.

Assess Safety, Tolerability and Pharmacokinetics of AntiBKV in Healthy Adult Volunteers.

NCT05358106CompletedPhase 1
Memo Therapeutics AG
Posted 5/9/2022

Safety, Tolerability and Efficacy of AntiBKV As Treatment of BKV Infection in Kidney Transplant Recipients

NCT05769582Active, Not RecruitingPhase 2
Memo Therapeutics AG
Posted 4/10/2023

DOE Supplies Accelerator-Produced Actinium-225 for First-Ever U.S. Cancer Therapy Clinical Trial

Targeted Therapy
  • The U.S. Department of Energy's Isotope Program will supply accelerator-produced actinium-225 to a U.S. company for the first clinical trial using this radioisotope for cancer therapy.
  • The clinical trial is scheduled to begin in summer 2025 and represents a significant milestone in radiopharmaceutical development by opening a new pipeline for this lifesaving isotope.
  • DOE has established a scalable production method using particle accelerators at Brookhaven National Laboratory and Los Alamos National Laboratory to address the current shortage of Ac-225.
  • This marks the transition from years of preclinical research and animal studies to the first human patient care application of accelerator-produced Ac-225.

Trial of 225Ac-DOTATATE (RYZ101) in Subjects With ER+, HER2-negative Unresectable or Metastatic Breast Cancer Expressing SSTRs.

NCT06590857RecruitingPhase 1
RayzeBio, Inc.
Posted 7/19/2024

Synfini and O2nix Bio Partner to Develop AI-Driven Cancer Metastasis Therapies Targeting FTSJ1

  • Synfini Inc. and O2nix Bio announced a strategic collaboration to develop novel drug candidates targeting FTSJ1, a tRNA-modifying enzyme implicated in metastatic cancer cell survival.
  • The partnership combines O2nix Bio's expertise in RNA epitranscriptomics and metastasis biology with Synfini's AI Cloud Foundry platform to accelerate drug discovery.
  • The collaboration aims to address cancer metastasis, which is responsible for over 90% of cancer-related deaths and remains largely untreatable by current therapies.
  • This marks O2nix Bio's first commercial drug discovery initiative as an independent entity, building on foundational research published in Nature Cancer.

FDA Cancels Advisory Committee Meeting for Capricor's Duchenne Therapy Amid Leadership Shake-up

  • The FDA's Center for Biologics Evaluation and Research director Vinay Prasad unilaterally canceled an advisory committee meeting for Capricor Therapeutics' Duchenne muscular dystrophy cell therapy due to skepticism about the treatment.
  • The cancellation occurred immediately before the ouster of Nicole Verdun, the FDA's chief regulator of cell and gene therapies, who had originally scheduled the meeting.
  • Capricor shares plummeted 40% to $7.22 following news of the canceled advisory committee meeting and regulatory uncertainty.
  • Long-standing tensions over Verdun's management style may have contributed to her placement on administrative leave, raising questions about the precise reasons for the leadership change.

MedHub-AI Receives PMDA Approval for AI-Powered Coronary Assessment Technology in Japan

AI
  • MedHub-AI's AutocathFFR® becomes the first fully automated AI-powered software to receive PMDA approval in Japan for calculating Fractional Flow Reserve values directly from X-ray angiograms without invasive procedures.
  • The approval is supported by a pivotal study of 504 vessels demonstrating 93.7% diagnostic accuracy, 90.2% sensitivity, and 94.9% specificity compared to traditional invasive FFR measurements.
  • The company has partnered with Terumo Corporation for Japanese market distribution and expects FDA approval in the near future to accelerate global expansion.
  • AutocathFFR® delivers FFR results in just 37 seconds, eliminating the need for guidewires and vasodilatory drugs while providing operator-independent decision support for cardiologists.

TGS and Viridien Launch Laconia Phase III Ocean Bottom Node Survey in Gulf of America

  • TGS and Viridien have commenced Laconia Phase III, a large-scale ocean bottom node survey spanning approximately 150 OCS blocks in the Gulf of America's central Keathley Canyon area.
  • The project utilizes advanced seismic technologies including TGS' ZXPLR™ nodes and Sercel's Tuned Pulse Source to deliver ultra-long offset data with enhanced low-frequency imaging capabilities.
  • The survey targets the increasingly attractive Paleogene play following recent industry success with 20k technologies, with initial products scheduled for delivery in Q1 2026.
  • Viridien's proprietary Elastic Full-Waveform Inversion and Reverse Time Migration imaging technologies will process the data to provide best-in-class seismic solutions for exploration and development activities.

HotSpot Therapeutics to Present IRF5 Small Molecule Inhibitor Data at FOCIS 2025

  • HotSpot Therapeutics will present preclinical data on selective small molecule IRF5 inhibitors at the 25th Annual FOCIS meeting in Boston on June 25, 2025.
  • The company's Smart Allostery platform uses computational approaches and AI-driven data mining to identify "natural hotspots" on proteins for drug targeting.
  • HotSpot is developing a pipeline of novel allosteric therapies for autoimmune diseases and cancer using their proprietary hotspot-targeting approach.
  • The IRF5 program represents part of the company's broader strategy to create oral, small molecule allosteric drugs with novel pharmacology.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.