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Clinical Trial News

Wingor Biotechnology Secures Tens of Millions in Funding to Advance Stem Cell and Gene Therapy Pipeline

  • Shenzhen Wingor Biotechnology raised tens of millions in CNY funding led by KC Eternium Management to advance its stem cell and gene therapy programs.
  • The company operates four therapeutic platforms including stem cell, exosome, immune cell, and gene engineering technologies with over ten R&D pipelines in development.
  • Wingor leads southern China in IND applications for stem cell drugs and has established partnerships with over 60 top-tier hospitals for clinical research projects.
  • Four clinical projects have received approval and begun patient recruitment, including participation in a national stem cell therapy project for liver failure.

OrganOx Acquired for $1.5 Billion in One of UK's Largest Medtech Exits

  • OrganOx, the Oxford-based medtech company behind pioneering liver perfusion technology, has been acquired by Terumo Corporation for approximately $1.5 billion.
  • The company's metra device enables donor livers to be maintained in a functioning state outside the human body for up to 24 hours, transforming transplant outcomes.
  • The technology has been used in more than 6,000 liver transplants to date, significantly increasing the number of viable organs available for transplant.
  • The acquisition represents one of the largest exits of a UK medtech company and delivered BGF's biggest return to date with a 10x money multiple.

Royalty Pharma Acquires $950 Million IMDELLTRA Royalty Rights from BeOne Medicines

Drug ApprovalOncology
  • Royalty Pharma acquired BeOne Medicines' royalty rights to IMDELLTRA (tarlatamab-dlle) for up to $950 million, with $885 million paid upfront and an option for additional $65 million within 12 months.
  • IMDELLTRA is a first-in-class DLL3-targeting bispecific T-cell engager approved for extensive-stage small cell lung cancer patients who progressed after platinum-based chemotherapy.
  • The drug generated $215 million in sales during the first half of 2025 and is projected to exceed $2.8 billion by 2035 according to analyst consensus.
  • BeOne retains China commercial rights and will share in royalties on annual sales above $1.5 billion, while maintaining rights to other collaboration assets including xaluritamig.

Leads Biolabs Advances First-in-China GPRC5D T-Cell Engager LBL-034 to Phase II Trial

Orphan Drug
  • Nanjing Leads Biolabs has successfully dosed the first patient in the Phase II trial of LBL-034, a GPRC5D/CD3 bispecific T-cell engager for relapsed/refractory multiple myeloma.
  • The therapy demonstrated CAR-T-like efficacy with robust objective response rates at higher doses and showed promising results in difficult-to-treat extramedullary plasmacytomas during Phase I trials.
  • LBL-034 is positioned to become the first domestic T-cell engager therapy targeting GPRC5D in China and is the second most clinically advanced globally.
  • The multi-center Phase II trial involves over 20 hospitals across China and aims to evaluate efficacy and safety in various relapsed/refractory plasma cell neoplasms.

Oculis Strengthens Leadership with Appointment of Two World-Renowned Ophthalmology Experts as Chief Medical Advisors

Precision Medicine
  • Oculis appointed Mark Kupersmith, M.D., as Chief Medical Advisor for Neuro-Ophthalmology following positive Phase 2 ACUITY trial results with Privosegtor (OCS-05) in acute optic neuritis.
  • Sebastian Wolf, M.D., Ph.D., was named Chief Medical Advisor for Ophthalmology as the company advances its Phase 3 DIAMOND trials of OCS-01 eye drops in diabetic macular edema.
  • The appointments strengthen Oculis' scientific leadership as the company progresses toward potential regulatory filings, with topline results from Phase 3 trials expected in Q2 2026.
  • Both advisors bring decades of clinical expertise, with Kupersmith having 40 years in neuro-ophthalmology and Wolf authoring over 400 peer-reviewed publications in retinal diseases.

Lundbeck's Bexicaserin Shows Sustained 59% Seizure Reduction in Rare Epilepsy Extension Study

Rare Disease
  • Lundbeck will present complete results from the PACIFIC trial extension study showing bexicaserin achieved a median 59.3% reduction in countable motor seizure frequency over 52 weeks in patients with developmental and epileptic encephalopathies.
  • The FDA Breakthrough Therapy-designated drug demonstrated sustained efficacy with 55% of participants experiencing ≥50% seizure reductions compared to baseline, with over 90% completion rate indicating strong tolerability.
  • New preclinical data reveals bexicaserin significantly reduced seizures and respiratory arrest in SUDEP models, addressing the leading cause of premature death in epilepsy patients.
  • The company has advanced bexicaserin into global Phase 3 trials (DEEp program) with DEEpSEA recruiting Dravet syndrome patients and DEEpOCEAN enrolling patients with various developmental and epileptic encephalopathies.

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)

NCT06719141RecruitingPhase 3
Longboard Pharmaceuticals
Posted 11/11/2024

A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

NCT06660394RecruitingPhase 3
Longboard Pharmaceuticals
Posted 10/2/2024

Neuren Pharmaceuticals Launches First-Ever Phase 3 Trial for Phelan-McDermid Syndrome

Orphan DrugRare Disease
  • Neuren Pharmaceuticals has initiated the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, marking the first-ever Phase 3 trial for this severe neurodevelopmental disorder.
  • The randomized, double-blind, placebo-controlled trial will assess 13 weeks of treatment in approximately 160 children aged 3 to 12 with PMS, with all participants eligible for a 12-month open-label extension.
  • Phelan-McDermid syndrome affects an estimated 1 in 8,000 to 1 in 15,000 people and currently has no approved treatments available.
  • The trial program is fully funded by Neuren's existing cash reserves, following FDA alignment on the Phase 3 trial design including primary efficacy endpoints.

GSK Launches First PD-1 Immunotherapy and PARP Inhibitor for Gynecological Cancers in India

OncologyDrug Approval
  • GlaxoSmithKline has introduced Jemperli and Zejula therapies in India, marking the company's entry into the oncology segment with treatments for gynecological cancers.
  • Jemperli becomes the first and only approved PD-1 immunotherapy for second-line treatment of mismatch repair-deficient/microsatellite instability-high advanced or recurrent endometrial cancer in India.
  • Zejula is positioned as the only PARP inhibitor approved as first-line monotherapy maintenance for all biomarker types in advanced ovarian cancer in India.
  • Both therapies are already trusted and approved in over 40 countries globally, addressing critical unmet medical needs in women's cancer care.

VYVGART Achieves Primary Endpoint in Phase 3 Trial for AChR-Ab Seronegative Myasthenia Gravis

Monoclonal Antibody
  • argenx's VYVGART met its primary endpoint in the ADAPT SERON study with a statistically significant improvement in MG-ADL scores (p-value=0.0068) for AChR-Ab seronegative generalized myasthenia gravis patients.
  • The study represents the first global Phase 3 trial to demonstrate clinically meaningful improvements across all three seronegative subtypes: MuSK+, LRP4+, and triple seronegative patients.
  • argenx plans to submit a supplemental Biologics License Application to the FDA by end of 2025 to expand VYVGART's label to include all AChR-Ab seronegative gMG patients.
  • The treatment was well tolerated with no new safety concerns identified, consistent with VYVGART's established safety profile in other indications.

Expecto Health Science Partners with VertisPro to Deploy AI-Driven Patient Recruitment in Asia-Pacific Clinical Trials

AI
  • Expecto Health Science, an Asia-Pacific Contract Research Organisation, has announced a strategic collaboration with VertisPro to integrate AI-driven patient acquisition solutions into clinical trials.
  • The partnership aims to address patient recruitment challenges in rare disease and oncology trials by improving patient identification accuracy and shortening recruitment timelines.
  • VertisPro's artificial intelligence platform will be integrated into Expecto's service offerings to help sponsors achieve better trial enrollment success rates across the region.
  • The collaboration combines Expecto's clinical operations expertise with VertisPro's proven recruitment technology to accelerate patient access to innovative therapies.

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