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Clinical Trial News

Exonate Advances EXN407 Eye Drop to Phase IIb Trial for Diabetic Retinopathy Treatment

  • Exonate plans to initiate the CLEAR-DE Phase IIb clinical trial for EXN407, a first-in-class topical SRPK1 inhibitor eye drop for non-proliferative diabetic retinopathy.
  • The Phase IIb trial will enroll 140 patients across Australia, the Middle East and China, beginning in early 2026 following encouraging Phase Ib/IIa results.
  • EXN407 demonstrated safety and tolerability with no drug-related serious adverse events and showed reduction in vascular leakage in the earlier study.
  • The twice-daily eye drop represents a potential breakthrough as the first non-invasive therapy for early-stage diabetic eye disease, avoiding monthly intraocular injections.

Artbio Raises $132 Million Series B to Advance Lead-212 Radiopharmaceutical for Prostate Cancer

Targeted TherapyOncology
  • Artbio secured $132 million in Series B funding to advance AB001, a lead-212 based radiopharmaceutical targeting metastatic castration-resistant prostate cancer.
  • The company's AB001 drug uses Pb-212 isotope technology designed to deliver radiation more quickly to tumors compared to existing treatments like Novartis' Pluvicto.
  • Funding will support mid-stage clinical trials and manufacturing network expansion, with a Phase 1 trial beginning imminently this year.
  • The Cambridge and Oslo-based startup, led by former Novartis executive Emanuele Ostuni, aims to compete in the rapidly growing radiopharmaceutical oncology market.

Relief Therapeutics to Merge with NeuroX in CHF 1 Billion Deal Creating AI-Driven Neurotherapeutics Platform

AIPrecision Medicine
  • Relief Therapeutics announced a binding agreement to merge with NeuroX Group, which recently acquired MindMaze's digital neurotherapeutics assets, in a transaction valued at CHF 1 billion for NeuroX and CHF 100 million for Relief.
  • The combined entity will create an AI-powered therapeutics platform integrating software-based neurorehabilitation with pharmacological treatments for neurological conditions including stroke, Parkinson's disease, and dementia.
  • Relief shareholders will own approximately 9% of the merged company, which will continue trading on the SIX Swiss Exchange with closing expected in Q4 2025.
  • NeuroX brings a clinically validated portfolio of digital neurotherapeutics backed by over USD 350 million in investment and partnerships with leading pharmaceutical companies across multiple clinical trials.

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.

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