MedPath

Clinical Trial News

Akyso Therapeutics Advances Long-Acting Opioid Use Disorder Treatment with Successful Phase 1a Trial and $15M NIH Grant

  • Akyso Therapeutics successfully completed its first-in-human Phase 1a clinical trial of iSTEP-N®, a bioabsorbable 12-month naltrexone implant for opioid use disorder.
  • The Phase 1a trial demonstrated the implant was well-tolerated with no serious adverse events and sustained therapeutic plasma levels for three months.
  • The company received $15 million in federal funding from NIDA to develop iSTEP-B®, a parallel 6-12-month buprenorphine implant using the same platform technology.
  • Both implants target the $3 billion OUD treatment market in the US, where naltrexone and buprenorphine represent over 90% of treatment options.

Vitiligo Treatment Pipeline Shows Promise with JAK Inhibitors and Novel Immunotherapies in Phase 3 Trials

Monoclonal AntibodyTargeted Therapy
  • Over 18 companies are actively developing 20+ pipeline therapies for vitiligo treatment, with JAK inhibitors leading the charge in late-stage clinical trials.
  • AbbVie's Upadacitinib and Pfizer's Ritlecitinib have both advanced to Phase 3 trials, demonstrating promising repigmentation results in vitiligo patients.
  • Novel approaches include Clinuvel's Afamelanotide in Phase 3 trials and Amgen's AMG 714 anti-IL-15 monoclonal antibody in Phase 2 development.
  • The pipeline reflects a paradigm shift towards targeted therapies and personalized medicine approaches, offering new hope for patients with this challenging autoimmune condition.

Porosome Therapeutics Reports Breakthrough Alzheimer's Platform with FDA-Validated Organoid Studies and AI-Designed Therapeutics

AIDrug Discovery
  • Porosome Therapeutics announced key advancements in its first-in-class, disease-modifying neurological platform that targets the root causes of Alzheimer's disease rather than just symptom management.
  • The company demonstrated significant reduction of Tau protein levels, an FDA-approved biomarker, and rapid reversal of Alzheimer's pathology within two weeks using FDA-validated human brain organoid models.
  • Three distinct therapeutic classes have been identified including small molecules, biologics, and AI-designed decoy peptides that neutralize toxic beta amyloid peptides.
  • The company is actively exploring the FDA's accelerated approval pathway based on quantifiable, biomarker-driven improvements in their studies.

Imperial College's REACT Study Partners with Dementia Trials Accelerator to Transform Clinical Trial Recruitment

  • Imperial College London's REACT programme, which built a 2.5 million participant cohort during COVID-19, will partner with the Dementia Trials Accelerator to address chronic recruitment challenges in UK dementia clinical trials.
  • The collaboration with Health Data Research UK, UK Dementia Research Institute, and Inuvi will create an end-to-end recruitment process using blood-based biomarkers and cognitive assessments to pre-screen participants.
  • The partnership aims to establish a pilot group of 10,000 pre-screened participants by early 2027, potentially accelerating the development of new dementia treatments and therapeutics.

Merck's Ifinatamab Deruxtecan Receives FDA Breakthrough Designation for Small Cell Lung Cancer

AIDrug DiscoveryOncology
  • Merck and Daiichi Sankyo's ifinatamab deruxtecan received FDA Breakthrough Therapy Designation for extensive-stage small cell lung cancer patients who progressed after platinum-based chemotherapy.
  • The B7-H3 directed antibody-drug conjugate represents the first BTD for the collaboration and addresses significant unmet medical needs in SCLC, a disease with limited therapeutic options.
  • The designation follows positive Phase 2 IDeate-Lung01 trial data and accelerates regulatory pathways, potentially expediting approval timelines for this innovative cancer therapy.
  • Merck also announced a strategic AI partnership with Turbine to create virtual tumor models for hard-to-treat cancers, leveraging Simulated Cells™ technology to accelerate drug discovery.

Epicrispr Biotechnologies Doses First Patient with EPI-321, Novel Epigenetic Therapy for Facioscapulohumeral Muscular Dystrophy

Rare DiseaseOrphan Drug
  • Epicrispr Biotechnologies has dosed the first patient in a global first-in-human clinical trial of EPI-321, an investigational one-time epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD).
  • EPI-321 is the first investigational therapy designed to silence DUX4 expression via epigenetic modulation, targeting the root cause of FSHD which affects approximately 1 in 8,000 individuals worldwide.
  • The therapy has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, with initial data expected in early 2026.
  • FSHD is a progressive genetic disease leading to skeletal muscle degeneration and severe loss of function, with currently no approved disease-modifying therapies available.

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

NCT06907875RecruitingPhase 1
Epicrispr Biotechnologies, Inc.
Posted 5/8/2025

AC Immune Reports Strong Immunogenicity Data for Parkinson's Disease Vaccine in Phase 2 Trial

  • AC Immune's ACI-7104.056 anti-alpha-synuclein active immunotherapy demonstrated a 20-fold increase in anti-alpha-synuclein antibodies after four immunizations in the Phase 2 VacSYn trial for early Parkinson's disease.
  • The company maintains a robust cash position of CHF 127.1 million providing funding into Q1 2027, supporting advancement of three active immunotherapies in Phase 2 development.
  • Multiple clinical milestones are expected in H2 2025, including additional VacSYn trial data and the filing of an IND for the novel NLRP3 inhibitor ACI-19764.
  • The Alzheimer's disease program ACI-24.060 will reach 12 months of treatment in the AD3 cohort by December 2025, with interim results expected in early 2026.

SynOx Therapeutics Completes Enrollment in Phase 3 Trial of Emactuzumab for Rare Joint Tumor

Monoclonal AntibodyRare DiseaseOrphan Drug
  • SynOx Therapeutics has completed patient enrollment in its registrational Phase 3 TANGENT trial evaluating emactuzumab for tenosynovial giant cell tumors (TGCT), with top-line results expected in Q1 2026.
  • The study enrolled patients significantly ahead of projected timelines across multiple global sites, reflecting strong interest in this potentially best-in-class CSF-1 receptor inhibiting monoclonal antibody.
  • Emactuzumab has demonstrated substantial efficacy in earlier clinical studies with an objective response rate of approximately 71% and has received Fast Track Designation from the FDA.
  • TGCT is a rare, debilitating joint tumor that affects over 50% of patients with tumor recurrence within three years after surgery, representing a significant unmet medical need.

A Phase III, Multicentre, Randomised, Double-Blind Study to Assess the Safety and Efficacy of Emactuzumab vs. Placebo in Subjects with Tenosynovial Giant Cell Tumour

2023-510422-32-00Active, Not RecruitingPhase 3
Synox Therapeutics Limited
Posted 4/30/2024

A Study of RO5509554 as Monotherapy and in Combination With Paclitaxel in Participants With Advanced Solid Tumors

NCT01494688CompletedPhase 1
Hoffmann-La Roche
Posted 12/20/2011

Novel Bacterial Consortium AUN Achieves Tumor Eradication Without Immune System Dependence

  • Researchers at JAIST, Daiichi Sankyo, and University of Tsukuba developed AUN, a revolutionary bacterial cancer therapy that eliminates tumors without requiring immune cell function.
  • The therapy combines Proteus mirabilis and Rhodopseudomonas palustris bacteria that work synergistically to destroy tumor vasculature and cancer cells while minimizing side effects.
  • AUN represents a paradigm shift for immunocompromised cancer patients who cannot benefit from conventional immunotherapies, with clinical trials planned within six years.

Shattuck Labs Secures $103 Million to Advance First-in-Class DR3 Antibody SL-325 Through Phase 2 Trials

Monoclonal Antibody
  • Shattuck Labs raised up to $103 million in an oversubscribed private placement led by OrbiMed to fund development of SL-325, a potential first-in-class DR3 blocking antibody for autoimmune diseases.
  • The funding will support multiple Phase 2 clinical trials of SL-325 in inflammatory bowel disease and other autoimmune conditions, with Phase 1 enrollment expected to begin in Q3 2025.
  • SL-325 targets the Death Receptor 3 (DR3) pathway and preclinical studies demonstrate superior activity over TL1A antibodies, potentially offering improved efficacy and reduced immunogenicity.
  • Combined with existing cash, the financing is expected to fund operations through 2029 and advance the company's lead program targeting TNF superfamily receptors.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.