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Clinical Trial News

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

  • 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

  • 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.
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

  • 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.

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

  • 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.

SERB Pharmaceuticals to Acquire Y-mAbs Therapeutics for $412 Million, Expanding Rare Oncology Portfolio

  • SERB Pharmaceuticals announced an all-cash acquisition of Y-mAbs Therapeutics for $8.60 per share, representing a 105% premium and $412 million equity value.
  • The acquisition includes DANYELZA (naxitamab-gqgk), the first FDA-approved treatment for relapsed or refractory high-risk neuroblastoma in pediatric and adult patients.
  • DANYELZA expands SERB's rare oncology portfolio and provides outpatient treatment options for neuroblastoma patients who have shown partial response, minor response, or stable disease to prior therapy.
  • The transaction is expected to close by Q4 2025, with SERB leveraging its global footprint to expand DANYELZA's reach to new markets.

RIPK1 Inhibitor Pipeline Shows Promise Despite Sanofi Setback in Multiple Sclerosis

  • Over 10 companies are developing 12+ RIPK1 inhibitor therapies targeting inflammatory and neurodegenerative diseases, with key players including Sanofi, Rigel Pharmaceuticals, and GenFleet Therapeutics advancing promising candidates.
  • Sanofi discontinued its Phase 2 trial of oditrasertib in multiple sclerosis after failing to meet primary endpoints, highlighting the challenges in targeting neurodegeneration with RIPK1 inhibition.
  • Leading pipeline candidates include SAR443122 for cutaneous lupus and ulcerative colitis, GFH312 as China's first clinical-stage RIPK1 inhibitor, and R552 developed through Rigel's collaboration with Eli Lilly.
  • RIPK1 inhibitors represent a novel therapeutic approach by blocking inflammation and cell death pathways, offering potential treatments for autoimmune disorders, neurodegenerative conditions, and inflammatory diseases.

FDA Provides Positive Feedback on Immutep's Eftilagimod Alfa for Head and Neck Cancer Treatment

  • Immutep received positive FDA feedback on eftilagimod alfa development for first-line treatment of head and neck squamous cell carcinoma patients with PD-L1 expression below 1.
  • The FDA agreed on the potential of efti combined with KEYTRUDA to address high unmet need in this patient segment, representing up to 20% of first-line HNSCC patients.
  • FDA outlined potential accelerated approval pathways including a randomized registrational trial or smaller single-arm study under Project FrontRunner initiative.
  • Current treatment options for PD-L1 CPS <1 patients are limited to chemotherapy, as anti-PD-1 therapy alone is only approved for patients with CPS >1.

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