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Clinical Trial News

Brain-Inspired Supercomputer Deployed at Leipzig University to Accelerate AI-Driven Drug Discovery

AIDrug Discovery
  • SpiNNcloud has delivered the world's largest brain-inspired supercomputer system to Leipzig University in Germany, featuring 650,000 cores specifically designed for small-molecule drug discovery research.
  • The system can simulate up to 10.5 billion neurons and screen 20 billion molecules in less than an hour, representing a two-orders-of-magnitude speed improvement over traditional 1000 CPU core systems.
  • The supercomputer operates with 18 times greater energy efficiency than current GPU-based systems, utilizing a unique architecture with 10 million ARM-based processors and specialized neural network accelerators.
  • Leipzig University plans to use the system primarily for protein folding simulations to advance personalized medicine by identifying molecular interactions with patient profiles.

eXoZymes Achieves Breakthrough in NCT Production with AI-Driven Biomanufacturing Platform

  • eXoZymes and its spinout NCTx successfully produced 4 grams of N-trans-caffeoyltyramine (NCT) at 96% yield and over 99% purity using their AI-driven exozymes platform.
  • The company achieved gram-scale production of NCT in just 5 months at a fraction of normal synthetic biology R&D costs, overcoming traditional extraction limitations from hemp seeds.
  • NCT shows therapeutic potential for liver fat metabolism, gut barrier function, and mitochondrial activity, with preclinical studies demonstrating enhanced fat breakdown and no observed toxicity.
  • The company plans to scale production 100-fold over six months and prepare for GMP pilot scale production, targeting kilogram demand discussions for 2026.

ViroCell Biologics Partners with AvenCell Therapeutics to Advance Dual-Targeted Allogeneic CAR-T Therapy

CAR-TOncology
  • ViroCell Biologics has delivered a novel retroviral vector to AvenCell Therapeutics for their investigational CD19/CD20 dual-targeted CAR-T therapy AVC-203, designed for B cell malignancies and autoimmune diseases.
  • AVC-203 is an allogeneic "off-the-shelf" CAR-T cell therapy engineered to mitigate graft-versus-host disease and graft rejection, incorporating AvenCell's RevCAR receptor for additional antigen targeting with in vivo "off/on" capability.
  • The therapy is expected to enter a phase I clinical trial for relapsed/refractory B cell lymphoma in the second half of 2025, with potential advantages of improved efficacy, immediate treatment availability, and reduced cost compared to existing CAR-T therapies.

Bausch Health Acquires DURECT for $63M to Advance Breakthrough Alcoholic Hepatitis Treatment

  • Bausch Health announced the acquisition of DURECT Corporation for $63 million upfront, with potential milestone payments up to $350 million, to gain access to larsucosterol, an epigenetic modulator with FDA Breakthrough Therapy Designation for alcoholic hepatitis.
  • Larsucosterol represents a potential first-in-class treatment for alcoholic hepatitis, a life-threatening condition responsible for approximately 100 deaths per day in the US and 164,000 hospital admissions in 2021, with no currently approved therapies.
  • The acquisition strengthens Bausch Health's hepatology portfolio alongside their ongoing Phase 3 rifaximin SSD program, with a registrational Phase 3 trial for larsucosterol planned to evaluate 90-day survival in severe alcoholic hepatitis patients.

Centauri Therapeutics Secures Additional $5.1M from CARB-X to Advance Novel Antimicrobial ABX-01 to Clinical Trials

  • Centauri Therapeutics received an additional $5.1 million from CARB-X to advance their lead antimicrobial compound ABX-01 toward first-in-human clinical trials in early 2026.
  • The ABX-01 candidate employs a dual mechanism combining immunotherapeutic effects through complement fixation and phagocytosis with intrinsic antibacterial properties to target multidrug-resistant Gram-negative bacterial infections.
  • CARB-X's total funding support for Centauri's Alphamer platform and ABX-01 development has reached $12.3 million since 2019.
  • The broad-spectrum antimicrobial is specifically designed to combat clinically prevalent and multidrug-resistant bacterial strains affecting the most vulnerable patients with serious lung infections.

Merck Announces $3 Billion Cost-Cutting Initiative to Prepare for Keytruda Patent Expiration

  • Merck plans to slash $3 billion in costs by the end of 2027 to reinvest in new product launches and drug pipeline development ahead of Keytruda's patent expiration in 2028.
  • The company reported second-quarter revenue of $15.81 billion, missing Wall Street estimates for the first time since April 2021, driven by a 55% decline in Gardasil sales due to weak demand in China.
  • Keytruda sales grew 9% to $7.96 billion during the quarter, driven by higher uptake for earlier-stage cancers and strong demand for metastatic cancer treatment.
  • The restructuring program will eliminate certain administrative, sales, and R&D positions while reducing global real estate footprint and manufacturing network optimization.

Arrowhead Pharmaceuticals Receives $100 Million Milestone Payment for ARO-DM1 Myotonic Dystrophy Treatment

Rare Disease
  • Arrowhead Pharmaceuticals earned a $100 million milestone payment from Sarepta Therapeutics after reaching enrollment targets for ARO-DM1, an RNAi therapeutic for type 1 myotonic dystrophy.
  • The milestone was triggered by achieving the first of two prespecified enrollment targets and authorization for dose escalation in the Phase 1/2 clinical study.
  • A second enrollment target expected by end of 2025 would trigger an additional $200 million milestone payment under the global licensing agreement.
  • The collaboration covers multiple clinical and preclinical programs targeting rare genetic diseases of muscle, central nervous system, and lungs using Arrowhead's TRiM platform.

Celltrion Secures Preferred Bidder Status for US Manufacturing Facility to Navigate Tariff Challenges

  • Celltrion has been named the preferred bidder for a large-scale biologics manufacturing facility in the US, with a final agreement expected in early October.
  • The Korean pharmaceutical company plans to invest 700 billion won ($503 million) in the acquisition and operation, with potential additional investments of 300-700 billion won depending on US tariff policies.
  • The strategic move aims to eliminate tariff-related risks for Celltrion's 11 biosimilars currently sold in the US, with plans to expand the portfolio to 22 by 2030 and 41 by 2033.
  • The acquisition includes a contract manufacturing agreement covering 50% of the facility's capacity with exclusive rights to produce the seller's biologics for five years.

Syntara Initiates First-in-Human Trial of Topical Anti-Fibrotic Drug SNT-9465 for Hypertrophic Scars

  • Syntara Limited has dosed the first participant in a Phase 1a/b clinical trial of SNT-9465, a topical pan-lysyl oxidase inhibitor designed to treat hypertrophic scars.
  • The trial will initially assess safety and tolerability in healthy participants before moving to a Phase 1b extension evaluating improvements in scar appearance and composition after three months of daily treatment.
  • Current standard care for hypertrophic scars includes costly laser therapy or painful steroid injections requiring multiple treatments for only small incremental improvements.
  • Trial results expected in the first half of 2026 are anticipated to support an FDA IND application for what could become the first approved pharmacological treatment for skin scarring.

Alphamab Oncology's HER2-Targeting ADC JSKN003 Receives FDA Orphan Drug Designation for Gastric Cancer

FDA Approval
  • Alphamab Oncology's anti-HER2 biparatopic antibody-drug conjugate JSKN003 has been granted FDA Orphan Drug Designation for treating gastric cancer and gastroesophageal junction cancer.
  • The designation provides significant regulatory advantages including tax credits, fee waivers, accelerated review processes, and potential seven-year market exclusivity upon approval.
  • JSKN003 demonstrated favorable safety and promising efficacy in Phase I clinical studies, particularly in heavily pretreated patients with high HER2-expressing gastrointestinal tumors.
  • Gastric and gastroesophageal junction cancers represent a significant unmet medical need with poor survival outcomes, affecting approximately 960,000 new cases globally and 26,890 cases in the U.S. annually.

Safety and Tolerability of JSKN003 in Chinese Subjects With Advanced Solid Tumors

NCT05744427RecruitingPhase 1
Jiangsu Alphamab Biopharmaceuticals Co., Ltd
Posted 3/15/2023

First-In-Human Study in Subjects With Advanced or Metastatic Solid Malignant Tumors

NCT05494918CompletedPhase 1
Alphamab (Australia) Co Pty Ltd.
Posted 9/2/2022

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