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Clinical Trial News

Annexin Pharmaceuticals Receives UK Approval for Phase 2a Study of ANXV in Diabetic Retinopathy

  • Annexin Pharmaceuticals has received UK MHRA approval to initiate a Phase 2a proof-of-concept study evaluating ANXV in diabetic retinopathy, a serious eye disease affecting over 100 million people globally.
  • The adaptive study design will also include newly diagnosed retinal vein occlusion patients to evaluate shorter treatment cycles, addressing questions from potential licensing partners.
  • First patients are expected to be treated in the third quarter of 2025, with initial data anticipated approximately three months later and topline results expected before year-end.
  • The study represents a strategic expansion of ANXV's clinical portfolio beyond retinal vein occlusion, where the drug candidate previously demonstrated clinically relevant efficacy signals and favorable safety profile.

Llama-Derived Nanobody Shows Promise for Schizophrenia Cognitive Symptoms in Preclinical Study

  • Researchers at the Institute of Functional Genomics developed a llama-derived nanobody called DN13-DN1 that crosses the blood-brain barrier and improves cognitive deficits in schizophrenia mouse models after a single injection.
  • The nanobody targets mGlu2 receptors in the brain and demonstrated sustained therapeutic effects lasting up to seven days, significantly longer than existing treatments like LY379268 which wear off within 24 hours.
  • Unlike current antipsychotics that primarily address hallucinations and delusions, this nanobody specifically improved memory, attention, and sensorimotor gating deficits that are poorly treated by existing medications.
  • The therapy showed high selectivity with no effects in healthy mice and no toxicity signs during repeated dosing, offering a potential new approach for treating cognitive symptoms in schizophrenia.

Hillhurst Biopharmaceuticals Initiates Phase 2a Trial of Oral Carbon Monoxide Therapy for Sickle Cell Disease

Rare Disease
  • Hillhurst Biopharmaceuticals has dosed the first patient in a Phase 2a clinical trial evaluating HBI-002, an oral low-dose carbon monoxide therapy for sickle cell disease.
  • The open-label study will assess safety and tolerability while collecting biomarker and pharmacokinetic data to inform a larger Phase 2b trial planned for 2026.
  • HBI-002 represents a novel approach to treating sickle cell disease, targeting the prevention of painful vaso-occlusive crises in a condition with significant unmet medical need.
  • The therapy is designed for chronic home use and leverages Hillhurst's proprietary GLASS platform to deliver oral formulations of traditionally inhaled therapeutics.

Australia's National Reconstruction Fund Invests $150M in Brandon Capital to Retain Biotech Innovation Onshore

  • Australia's National Reconstruction Fund Corporation has committed $150 million to Brandon Capital's sixth biotech fund, bringing the total fund size to $439 million and making it the Melbourne-based firm's largest fund to date.
  • The investment aims to address a critical capital gap that has historically forced promising Australian biotech companies offshore just as they approach commercial viability, with two-thirds of NRF funding earmarked for late-stage companies.
  • The funding will support Australian-developed therapeutics, vaccines and medical devices, helping translate world-class research into commercial solutions while keeping highly skilled jobs and medical innovation within Australia.
  • The commitment pushes the NRF's total investments above its $550 million financial year target, representing a significant shift in how Australia funds its biotech sector.

Abivax Raises $747.5 Million in Public Offering to Advance Ulcerative Colitis Drug Development

  • Abivax completed a $747.5 million public offering of American Depositary Shares, with underwriters exercising their full option to purchase additional shares.
  • The biotechnology company will allocate approximately 23% of proceeds to fund development of obefazimod (ABX464) for ulcerative colitis treatment.
  • Obefazimod is currently in Phase 3 clinical trials for moderately to severely active ulcerative colitis, representing the company's lead therapeutic candidate.
  • The company had $71.4 million in cash as of June 30, 2025, which would fund operations only into the fourth quarter of 2025.

UK Becomes First Country to Enable Point-of-Care Manufacturing for Personalized Therapies

CAR-T
  • The UK has enacted world-first legislation allowing personalized medicines to be manufactured at hospitals, clinics, and near patients' homes, reducing treatment delivery times from months to days.
  • The Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations 2025 covers cell and gene therapies, tissue-engineered treatments, 3D-printed products, blood products, and medicinal gases.
  • The framework supports mobile manufacturing units and enables final manufacturing steps at the point of care, potentially preventing patients from becoming too unwell to receive CAR-T therapies due to extended wait times.
  • This legislation represents a significant advancement toward truly personalized medicine and is part of the NHS 10-year plan announced by the UK government.

Qu Biologics Completes Enrollment in Phase 2 Trial Testing Novel Immunomodulator for Age-Related Immune Decline

  • Qu Biologics has completed enrollment of 72 participants in the RESILIENCE Study, a randomized placebo-controlled Phase 2 trial evaluating QBKPN SSI in adults 65 years and older.
  • QBKPN SSI represents a first-in-class Site Specific Immunomodulator designed to restore innate immune function that naturally declines with aging.
  • The study addresses what the company calls "the largest unmet need in healthcare" by targeting age-related immune decline that increases risks of infections, inflammatory diseases, and cancer.
  • Previous proof-of-concept studies demonstrated QBKPN's ability to reverse immune dysregulation associated with aging, inflammatory diseases, and cancer.

Viatris Wins Patent Battle Against Novo Nordisk Over Generic Wegovy

FDA Approval
  • A federal district court in Delaware ruled that Viatris' generic version of Wegovy does not infringe on Novo Nordisk's patent for the GLP-1 receptor agonist semaglutide.
  • The court determined that Viatris' product label does not encourage use without other therapeutic agents, which was a key limitation in Novo Nordisk's patent claim.
  • This victory removes one patent obstacle for Viatris' generic Wegovy, which is currently awaiting FDA approval, while the global Wegovy market reached $8.4 billion in 2024.
  • The ruling comes amid ongoing patent disputes in the lucrative obesity drug market, with GlobalData forecasting the obesity market to exceed $173.5 billion by 2031.

Federal Court Dismisses Whistleblower Patent Fraud Lawsuit Against Valeant and Salix Over Ulcerative Colitis Drug Apriso

  • A federal judge dismissed a seven-year False Claims Act lawsuit against Valeant Pharmaceuticals and Salix Pharmaceuticals alleging fraudulent patent practices for ulcerative colitis drug Apriso.
  • Whistleblower Zachary Silbersher claimed the companies fraudulently obtained patents, excluded generic competitors, and overbilled Medicare for prescriptions.
  • The dismissal was without prejudice, allowing the plaintiff to amend and refile claims with more specific allegations against each defendant.

Autoantibodies Emerge as Key Predictors of Cancer Immunotherapy Response, Boosting Effectiveness Up to 10-Fold

  • A groundbreaking study published in Nature reveals that naturally occurring autoantibodies can dramatically influence cancer immunotherapy effectiveness, with some boosting patient response rates by five to ten-fold.
  • Researchers analyzed over 6,000 types of autoantibodies in 374 cancer patients receiving checkpoint inhibitors using the novel REAP technology platform.
  • Autoantibodies targeting interferons were linked to better anti-tumor responses, while others hindered treatment effectiveness, offering new targets for combination therapies.
  • The findings provide crucial insights into why checkpoint inhibitors work for some patients but not others, potentially extending benefits to more cancer patients.

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