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Clinical Trial News

32 Biosciences Secures $6 Million Funding to Advance Gut Microbiome Diagnostic and Therapeutic Platform

AI
  • 32 Biosciences raised $6 million in funding to advance its AI-powered gut microbiome diagnostic platform and first-of-its-kind non-antibiotic antimicrobial therapy.
  • The company's diagnostic tool uses mass spectrometry and machine learning to quantitatively assess gut microbiome functional health and is targeted for FDA clearance by late 2026.
  • Their therapeutic candidate represents a novel approach to preventing gut-derived bacterial infections without contributing to antibiotic resistance, having shown effectiveness in animal models.
  • The funding will support clinical validation, regulatory submissions, and commercial readiness as the company prepares for Series A fundraising and Phase I clinical trials.

Vanda Pharmaceuticals Initiates First-in-Human Trial of Personalized ASO Therapy VCA-894A for Ultra-Rare CMT2S

Rare Disease
  • Vanda Pharmaceuticals has dosed the first patient in a first-in-human clinical trial evaluating VCA-894A, an antisense oligonucleotide therapy designed for a specific patient with Charcot-Marie-Tooth disease Type 2S.
  • VCA-894A targets a patient-specific IGHMBP2 gene variant and has demonstrated ability to restore gene expression levels in neuromuscular junction systems derived from the patient's own cells.
  • CMT2S is an extremely rare inherited neuromuscular disorder with an estimated prevalence of less than 1 in 1,000,000 worldwide, causing progressive muscle weakness and motor function loss.
  • This personalized medicine approach represents a potential breakthrough for addressing rare genetic disorders using nucleic acid-based platforms to restore function where crucial gene expression has been disrupted.

Insmed's TPIP Achieves Breakthrough Results in Phase 2b PAH Trial with 35% Reduction in Pulmonary Vascular Resistance

Rare Disease
  • Insmed's treprostinil palmitil inhalation powder (TPIP) met its primary endpoint with a statistically significant 35% placebo-adjusted reduction in pulmonary vascular resistance in patients with pulmonary arterial hypertension.
  • The once-daily inhaled therapy demonstrated sustained 24-hour efficacy with a 35.5-meter improvement in six-minute walk distance and 60% reduction in cardiac stress biomarker NT-proBNP.
  • TPIP was well-tolerated with 75% of patients achieving the maximum dose, prompting Insmed to engage with FDA for Phase 3 trial design beginning in 2025-2026.

Caliway Unveils CBL-514 Preclinical Data for GLP-1 Weight Rebound Prevention at BIO 2025

  • Caliway Biopharmaceuticals will present first-time preclinical data showing CBL-514 combined with GLP-1 therapies significantly reduces post-discontinuation weight regain and fat replenishment rates.
  • The company addresses a critical unmet need as only 10% of patients maintain weight loss after stopping GLP-1 treatment, representing a significant clinical challenge.
  • CBL-514 works through selective adipocyte apoptosis to target subcutaneous fat directly, potentially serving as a complementary therapy to enhance long-term GLP-1 treatment outcomes.
  • Caliway plans to submit a Phase 2 IND application to the FDA for this weight rebound management indication in Q4 2025.

Monte Rosa Therapeutics Receives FDA Clearance for MRT-8102, First NEK7-Targeted Molecular Glue Degrader for Inflammatory Diseases

  • Monte Rosa Therapeutics received FDA clearance for its Investigational New Drug application for MRT-8102, a NEK7-directed molecular glue degrader targeting inflammatory diseases driven by NLRP3 inflammasome and IL-1β dysregulation.
  • MRT-8102 demonstrated nanomolar-level NEK7 degradation with no off-target activity and achieved near-complete inhibition of downstream inflammatory markers in non-human primate studies.
  • The company plans to initiate a Phase 1 healthy volunteer study in the coming weeks, with clinical results including safety, pharmacokinetics, and pharmacodynamic data expected in the first half of 2026.
  • Preclinical toxicology studies showed a greater than 200-fold exposure margin over the projected human efficacious dose, supporting MRT-8102's potential safety profile for clinical development.

Evogene Completes First-in-Class AI Foundation Model for Small Molecule Design with 90% Precision

AIDrug Discovery
  • Evogene Ltd. has completed its generative AI foundation model version 1.0 for small molecule design, developed in collaboration with Google Cloud, achieving approximately 90% precision compared to 29% in traditional GPT AI models.
  • The model addresses the core challenge of identifying novel small molecules that simultaneously meet multiple complex product criteria while being patentable, essential for both pharmaceutical and agriculture applications.
  • Built on a dataset of approximately 38 billion molecular structures and trained using Google Cloud's advanced AI infrastructure, the model expands ChemPass AI capabilities to generate truly novel molecular structures.
  • Development is already underway on version 2.0 with enhanced flexibility for multi-parameter optimization, incorporating customized parameters tailored to therapeutic contexts and specific agriculture requirements.

Silicogenix Partners with BioDuro to Accelerate AI-Driven Drug Discovery for Complex Diseases

AIDrug DiscoveryOncology
  • Silicogenix, a pioneer in AI-powered small molecule design technology, has formed a strategic partnership with global CRDMO BioDuro to streamline drug discovery for complex and rare diseases.
  • The collaboration combines SGX's proprietary polypharmacology frameworks with BioDuro's integrated drug discovery platform to reduce development costs and timelines from concept to preclinical stages.
  • SGX's technology enables access to the majority of the human proteome and supports novel therapeutic approaches including combination therapies with standard-of-care treatments.
  • The partnership aims to accelerate development of first-in-class oncology and immunology therapeutics that address significant unmet medical needs in complex disease areas.

ImmVira Initiates Phase II Trial for Novel Oncolytic Immunotherapy in BCG-Unresponsive Bladder Cancer

  • ImmVira has dosed the first patient in a multi-regional Phase II clinical trial evaluating MVR-T3011, a novel oncolytic immunotherapy for BCG-unresponsive non-muscle invasive bladder cancer.
  • Phase I data demonstrated an encouraging complete response rate of over 80% at the 2×10⁹ PFU dose cohort, with sustained 3-month recurrence-free survival exceeding 80% in 16 evaluable patients.
  • The FDA-approved Phase II study will enroll patients at 15-20 cancer centers across the United States and China to confirm the recommended dose and assess anti-tumor efficacy.
  • MVR-T3011 combines a replication-competent oncolytic virus with PD-1 antibody and IL-12 expression to enhance immune responses in the tumor microenvironment.

A Study of T3011 in Patients With BCG-Unresponsive NMIBC or BCG-Exposed, Chemotherapy-Unresponsive NMIBC

NCT06971614RecruitingPhase 2
ImmVira Pharma Co. Ltd
Posted 6/9/2025

FDA Approves XIFYRM Injectable Meloxicam for Moderate-to-Severe Pain Management

Drug Approval
  • The FDA has approved XIFYRM (meloxicam injection), a once-daily intravenous NSAID formulation for managing moderate-to-severe pain in adults.
  • The 30mg/mL injection is administered via IV bolus over 15 seconds and can be used alone or combined with non-NSAID analgesics.
  • XIFYRM addresses clinical needs for non-opioid alternatives in multimodal analgesia strategies, particularly amid ongoing efforts to reduce opioid dependence.
  • The drug is not recommended when rapid onset of analgesia is required due to its delayed onset profile.

FDA Places Clinical Hold on Gilead's Weekly HIV Combination Therapy Following Safety Signal

  • The FDA has placed a clinical hold on five Gilead HIV trials involving weekly combination therapy GS-1720 and GS-4182 after safety signals showed decreased CD4+ T-cell and lymphocyte counts in some patients.
  • The hold affects two Phase II/III WONDERS trials comparing the weekly combo to Biktarvy in both treatment-naive and virologically suppressed HIV patients, plus three Phase I studies.
  • Analysts expect no impact on the pending June 19 FDA decision for lenacapavir's HIV prevention indication, as GS-4182 is a different pro-drug formulation than the approved Sunleca version.
  • Gilead maintains confidence in resolving the hold and continues developing multiple other long-acting HIV treatment combinations with weekly, monthly, quarterly, and twice-yearly dosing schedules.

Study of Oral Weekly GS-1720 and GS-4182 Versus Biktarvy in People With HIV-1 Who Are Virologically Suppressed

NCT06544733Active, Not RecruitingPhase 2
Gilead Sciences
Posted 8/20/2024

Study of Oral Weekly GS-1720 and GS-4182 Compared With Biktarvy in People With HIV-1 Who Have Not Been Treated

NCT06613685Active, Not RecruitingPhase 2
Gilead Sciences
Posted 10/21/2024

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