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Clinical Trial News

MSC-Derived Extracellular Vesicles Show Promise as Cell-Free Therapeutics Across Multiple Disease Areas

  • Mesenchymal stem cell-derived extracellular vesicles (MSC-EVs) demonstrate superior therapeutic advantages over traditional stem cell therapies, including enhanced safety profiles, easier storage, and versatile administration routes without risk of carcinogenesis.
  • Clinical trials show MSC-EVs effectively treat various conditions including COVID-19, ARDS, osteoarthritis, and stroke, with 64 registered trials currently investigating their therapeutic potential across multiple disease areas.
  • Menstrual blood-derived MSC-EVs emerge as a particularly promising source, offering unique advantages for female reproductive health applications while providing non-invasive collection methods and potential for personalized medicine approaches.

CMS Launches Multi-State Payment Model to Expand Access to Sickle Cell Gene Therapies

  • The Centers for Medicare & Medicaid Services unveiled a new outcomes-based payment model with 33 states to expand Medicaid access to gene therapies for sickle cell disease.
  • Participating states covering 84% of Medicaid sickle cell patients will receive guaranteed discounts and rebates from manufacturers if their drugs fail to deliver promised benefits.
  • The program addresses slow uptake of approved gene therapies Casgevy and Lyfgenia, which cost $2.2 million and $3.1 million respectively but have limited patient adoption since December 2023 approval.
  • CMS will negotiate outcomes-based agreements on behalf of state Medicaid agencies and provide up to $9.55 million in additional support per state for implementation.

SPRIM PRO and ObvioHealth Expand Partnership to Accelerate Digital Clinical Trial Innovation

  • SPRIM PRO and ObvioHealth, sister companies within the SPRIM Group, have expanded their collaboration to deliver a fully integrated clinical trial solution combining scientific expertise with advanced digital capabilities.
  • The partnership leverages SPRIM PRO's 24 years of experience supporting over 300 trials across 26 therapeutic areas with ObvioHealth's proprietary ObvioGo platform for decentralized and hybrid trials.
  • The collaboration enables next-generation digital endpoints through SPRIM PRO's development of over 300 custom instruments and ObvioGo's integration of wearables, biosensors, and telehealth visits.
  • The alliance aims to provide sponsors with streamlined, participant-centric research solutions that accelerate trial timelines while maintaining scientific rigor and regulatory compliance.

IBI343 Shows Promising Efficacy in CLDN18.2-Positive Gastric Cancer with Improved Safety Profile

  • IBI343, a next-generation CLDN18.2-targeting antibody-drug conjugate with Fc-silenced design, demonstrated manageable safety and promising efficacy in a phase 1 study of 127 patients with advanced gastric or gastroesophageal junction adenocarcinoma.
  • The study established 6 mg/kg every 3 weeks as the recommended phase 2 dose, showing confirmed objective response rates of 29.0% and 47.1% at 6 mg/kg and 8 mg/kg respectively in patients with high CLDN18.2 expression.
  • IBI343's unique Fc-silenced design resulted in notably lower gastrointestinal toxicities compared to other CLDN18.2-targeting therapies, with nausea occurring in 41.4% of patients versus higher rates seen with competing agents.
  • A phase 3 randomized trial (G-HOPE-001) comparing IBI343 monotherapy versus chemotherapy in previously treated patients with high CLDN18.2 expression is being initiated based on these encouraging results.

A Study of SHR-A1904 in Patients With Advanced Solid Cancer

NCT04877717Active, Not RecruitingPhase 1
Shanghai Hengrui Pharmaceutical Co., Ltd.
Posted 7/1/2021

A First-in-human Study of IBI343 in Subjects With Locally Advanced Unresectable or Metastatic Solid Tumors

NCT05458219RecruitingPhase 1
Innovent Biologics (Suzhou) Co. Ltd.
Posted 10/26/2022

A Multicenter, Phase 3 Study of IBI343 Monotherapy Versus Treatment of Investigator's Choice in Subjects With Previously Treated, Claudin (CLDN) 18.2-positive, HER2-negative, Gastric or Gastroesophageal Junction Adenocarcinoma

NCT06238843RecruitingPhase 3
Innovent Biologics (Suzhou) Co. Ltd.
Posted 6/30/2024

Sino Biopharm Acquires LaNova Medicines for $950M, Expanding Innovative Drug Pipeline

  • Sino Biopharm announced acquisition of remaining 95.09% stake in LaNova Medicines for up to $950 million, making it a wholly owned subsidiary.
  • LaNova possesses valuable innovative products including LM-299 (PD-1/VEGF) licensed to Merck and LM-305 (GPRC5D ADC) licensed to AstraZeneca.
  • The transaction represents the first 'China-to-China' merger and acquisition milestone for China's pharmaceutical industry.
  • Sino Biopharm's own pipeline includes TQC3721, a world's second-leading PDE3/4 inhibitor entering Phase III trials for COPD.

Stelara Biosimilars Capture Market Share as J&J's Blockbuster Drug Faces 33.7% Sales Decline

  • Johnson & Johnson's Stelara experienced a dramatic 33.7% year-over-year sales decline to $1.08 billion in Q1 2025, driven by aggressive biosimilar competition following patent expiration in September 2023.
  • Eight ustekinumab biosimilars have launched in the U.S. market with discounts of 46% to 90% versus Stelara's list price, with major PBMs excluding Stelara from formularies in favor of private-label biosimilars.
  • J&J faces additional pressure from the Inflation Reduction Act's Medicare Drug Price Negotiation Program, which mandates a 66% price cut for Stelara beginning January 1, 2026.
  • The company is countering with pipeline innovations including icotrokinra for psoriasis and Crohn's disease, plus promoting Tremfya as an alternative IL-23 targeting biologic in the same therapeutic areas.

£17.9 Million Wellcome Grant Funds Major Initiative to Combat Deadly Fungal Infections

Drug Discovery
  • A collaborative team from University of Dundee, GSK, and University of Exeter has received £17.9 million from Wellcome to develop new antifungal treatments over five years.
  • The project targets Cryptococcus neoformans, which causes meningitis and kills approximately 180,000 HIV patients annually, and Candida auris, a climate change-linked pathogen with over 40% fatality rates.
  • The initiative aims to generate two pre-clinical drug candidates for cryptococcal meningitis with broad-spectrum potential, addressing critical gaps in antifungal drug development.
  • Fungal infections cause around 2.5 million deaths worldwide annually, yet the drug development pipeline remains severely limited with increasing resistance and poor diagnostics.

Sanofi's SAR446597 Gene Therapy Receives FDA Fast Track Designation for Geographic Atrophy Treatment

  • The US FDA has granted fast track designation to Sanofi's SAR446597, a one-time intravitreal gene therapy for treating geographic atrophy due to age-related macular degeneration.
  • SAR446597 uses a dual-targeting approach to inhibit two critical complement pathway components (C1s and factor Bb) through a single injection, potentially eliminating the need for frequent treatments.
  • Geographic atrophy affects over 5 million people worldwide and causes permanent vision loss, with Sanofi planning to initiate phase 1/2 studies to evaluate the therapy's safety and efficacy.

A Safety and Efficacy Study of One-time SAR402663 in Adults With Neovascular Age-related Macular Degeneration

NCT06660667RecruitingPhase 1
Sanofi
Posted 11/21/2024

NCI Initiates $4.8 Million Clinical Trial of Senhwa's First-in-Class Cancer Drug CX-5461

Oncology
  • The U.S. National Cancer Institute has selected Senhwa Biosciences' Pidnarulex (CX-5461) for a five-year cancer research program, with the first patient enrolled in a monotherapy trial for advanced solid tumors.
  • The NCI will fully fund the clinical trial at approximately $4.8 million, investigating CX-5461's ability to induce DNA damage through G-quadruplex stabilization in patients with and without homologous recombination deficiency mutations.
  • Three additional NCI-sponsored studies are planned, including combination therapies with immunotherapy and antibody-drug conjugates, as well as a monotherapy study targeting MYC-driven lymphomas.
  • The global cancer immunotherapy market is projected to reach $224.3 billion by 2030, but current treatments benefit only 20-25% of patients, highlighting the need for novel combination approaches.

4Moving Biotech Initiates Phase 2a Trial of First-in-Class GLP-1 Therapy for Knee Osteoarthritis

  • 4Moving Biotech has enrolled the first patient in INFLAM MOTION, a Phase 2a trial evaluating 4P004, an intra-articular GLP-1 analog for knee osteoarthritis treatment.
  • The multicenter, randomized, placebo-controlled trial will enroll 129 patients across Europe, the United States, and Canada to assess both symptom relief and structural benefits.
  • The study represents the first human evaluation of a potential disease-modifying osteoarthritis drug targeting GLP-1 pathways, with topline results expected in the second half of 2026.
  • Primary endpoints focus on knee pain reduction at weeks 4 and 12, while secondary objectives include synovial health assessment via contrast-enhanced MRI.

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