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Clinical Trial News

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.

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

Monoclonal AntibodyRare DiseaseDrug Approval
  • 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

FDA Approval
  • 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.

Combination Study With Eftilagimod Alpha (a Soluble LAG-3 Fusion Protein) and Pembrolizumab in Patients With Recurrent or Metastatic HNSCC

NCT04811027Active, Not RecruitingPhase 2
Immutep S.A.S.
Posted 8/27/2021

SOPHiA GENETICS and AstraZeneca Expand AI Partnership to Optimize Breast Cancer Treatment Outcomes

AIReal World Evidence
  • SOPHiA GENETICS and AstraZeneca announced a multi-year collaboration expansion to leverage multimodal AI Factories for generating evidence on breast cancer therapy efficacy and real-world impact.
  • The partnership will utilize AI to analyze genomics, imaging, and clinical data to develop predictive models aimed at optimizing treatment outcomes for breast cancer patients.
  • The collaboration will focus on real-world evidence generation across Europe and North America to identify key drivers of treatment efficacy and enhance clinical decision-making.
  • AstraZeneca will integrate SOPHiA GENETICS's AI platform to augment their frontier AI solutions across oncology clinical development and improve understanding of disease biology.

Yatiri Bio Acquires NGeneBioAI to Strengthen AI-Powered Proteomics Platform for Precision Medicine

AIPrecision Medicine
  • Yatiri Bio has successfully acquired NGeneBioAI, a San Diego-based company specializing in plasma proteomics and AI-powered diagnostics, to strengthen its position in biomarker discovery and translational drug development.
  • The acquisition expands Yatiri Bio's operational capabilities by integrating CLIA- and CAP-certified laboratory infrastructure and enhancing its AI-proteomics platform that uses deep learning to map disease biology.
  • The expanded platform will support biomarker-driven programs across oncology, immunology, and neurodegeneration while strengthening partnerships with biopharma companies seeking companion diagnostics.
  • Yatiri Bio's platform uses deep neural networks to integrate proprietary proteomics, clinical metadata, and functional testing to identify responsive patient populations for clinical candidates.

Israeli Researchers Achieve First Long-Term Growth of Human Kidney Organoids, Opening Path to Regenerative Therapies

  • Researchers at Sheba Medical Center and Tel Aviv University have successfully grown human fetal kidney components from tissue stem cells for up to 34 weeks, marking the first time such organoids have survived beyond four weeks in laboratory conditions.
  • The breakthrough enables scientists to study congenital kidney defects in real-time, test drug toxicity during pregnancy, and develop new treatments using human-like models instead of relying solely on mouse studies.
  • The team is now seeking funding, including from China, to advance the technology toward clinical trials, with hopes of developing regenerative therapies that could repair damaged kidneys and reduce transplant needs.

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