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Clinical Trial News

REGENXBIO Advances Gene Therapy to Pivotal Phase IIb/III Trial for Diabetic Retinopathy

  • REGENXBIO will initiate a pivotal Phase IIb/III clinical trial for surabgene lomparvovec in diabetic retinopathy using suprachoroidal delivery, following positive two-year safety and efficacy data from the Phase II ALTITUDE trial.
  • The gene therapy demonstrated durable safety with no drug-related serious adverse events and no intraocular inflammation through two years at the highest dose level with prophylactic steroids.
  • AbbVie will pay REGENXBIO $100 million upon first subject dosed in the Phase IIb/III trial and an additional $100 million upon first subject dosed in a second Phase III trial under an amended collaboration agreement.
  • Diabetic retinopathy affects nearly 10 million people in the United States and is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.

Valneva Overcomes FDA Safety Pause for IXCHIQ Chikungunya Vaccine, Strengthens Market Position

Drug Approval
  • The FDA temporarily paused IXCHIQ vaccine use in adults aged 60 and older in May 2025 after 17 serious adverse events globally, but lifted restrictions by August 2025 following safety review and updated labeling.
  • Valneva demonstrated regulatory agility by collaborating with authorities to refine safety data, securing approvals in Brazil and maintaining European market access through EMA approval.
  • The company achieved strong financial performance with Q1 2025 product sales surging 50.3% to €48.6 million while reducing operating cash burn by 67% in 2024.
  • Valneva's diversified pipeline includes VLA15 Lyme disease vaccine in Phase 3 trials with Pfizer partnership and S4V2 Shigella vaccine with FDA Fast Track designation.

Almirall and Absci Expand AI Drug Discovery Partnership with Second Dermatology Target

AIDrug DiscoveryMonoclonal Antibody
  • Almirall and Absci have expanded their AI drug discovery collaboration to include a second dermatology target following successful delivery of AI-designed antibody leads against a difficult-to-drug target.
  • The collaboration combines Absci's Integrated Drug Creation™ platform with Almirall's dermatology expertise to develop novel therapeutics for chronic and debilitating skin diseases.
  • Under the expanded partnership terms, Absci is eligible to receive up to approximately $650 million in milestone payments across both programs, plus royalties on potential product sales.
  • The partnership demonstrates the potential of AI de novo drug design to unlock novel biology where traditional drug discovery approaches have previously failed.

Expert Highlights Optimal Positioning of Bispecific T-Cell Engagers in Multiple Myeloma Treatment

CAR-TOncology
  • Bispecific T-cell engagers demonstrate variable effectiveness in multiple myeloma depending on treatment setting, with talquetamab showing promise as pre-CAR T therapy to reduce cytokine release syndrome and neurotoxicity.
  • These agents produce deeper and more durable responses compared to standard therapies but carry increased infection risk, particularly with BCMA-targeting bispecifics.
  • Approximately 30% of patients fail to respond to bispecific treatment, potentially due to host immune system factors, patient fitness, and cancer cell soluble BCMA levels.
  • Researchers are exploring fixed-duration treatment strategies followed by observation periods to mitigate prolonged adverse effects associated with infections.

A Study of Teclistamab in Combination With Daratumumab and Lenalidomide (Tec-DR) and Talquetamab in Combination With Daratumumab and Lenalidomide (Tal-DR) in Participants With Newly Diagnosed Multiple Myeloma

NCT05552222RecruitingPhase 3
Janssen Research & Development, LLC
Posted 10/25/2022

Phase 3 Study of Teclistamab in Combination With Lenalidomide and Teclistamab Alone Versus Lenalidomide Alone in Participants With Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation

NCT05243797RecruitingPhase 3
Stichting European Myeloma Network
Posted 9/8/2022

Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Participants With Previously Untreated Multiple Myeloma

NCT02252172CompletedPhase 3
Janssen Research & Development, LLC
Posted 2/16/2015

Cartherics Opens Advanced Cell Therapy Manufacturing Facility to Support CTH-401 Clinical Development

Oncology
  • Melbourne-based Cartherics has opened a state-of-the-art cleanroom facility in Victoria to support clinical-scale manufacturing of its cell therapy products, marking a critical milestone for the company's manufacturing capabilities.
  • The facility will enable production of clinical batches of CTH-401, the company's lead CAR-iNK cell therapy targeting TAG-72 for relapsed and refractory ovarian cancer treatment.
  • Cartherics plans to submit an investigational new drug (IND) application for CTH-401 to the US FDA by mid-2026, advancing its mission to develop off-the-shelf immunotherapies for women's health conditions.
  • The facility incorporates advanced manufacturing technologies including closed processing systems, bioreactors, and comprehensive environmental controls to meet strict cell therapy manufacturing standards.

Blood-Based Protein Analysis Achieves 85.8% Accuracy in Predicting Triple-Negative Breast Cancer Immunotherapy Response

Precision Medicine
  • Researchers at Fudan University developed the Plasma Immuno Prediction Score (PIPscore) using six immune-related plasma proteins to predict immunotherapy response in triple-negative breast cancer with 85.8% accuracy.
  • The study identified key biomarkers ARG1, NOS3, and CD28 from analysis of 92 proteins in 195 TNBC patients, offering a non-invasive alternative to tumor biopsies for treatment prediction.
  • The PIPscore demonstrated 96% precision in predicting 12-month progression-free survival, potentially enabling personalized treatment strategies and early identification of non-responders.
  • This plasma proteomics approach addresses limitations of current biomarkers like PD-L1 expression by providing dynamic assessment of systemic immunity rather than tumor-localized signatures alone.

Day One Biopharmaceuticals Reports 310% Revenue Surge for Pediatric Cancer Drug OJEMDA

Targeted Therapy
  • Day One Biopharmaceuticals reported OJEMDA net product revenue of $33.6 million in Q2 2025, representing a 310% year-over-year increase driven by expanding adoption among pediatric oncologists.
  • The company wrote more than 1,000 OJEMDA prescriptions during the quarter, marking a 15% sequential increase and 346% rise compared to the prior year period.
  • Management reaffirmed annual OJEMDA revenue guidance of $140-150 million for 2025 and confirmed sufficient cash reserves to fund operations without external financing.
  • Two pivotal trials for OJEMDA, FIREFLY-1 and FIREFLY-2, remain on track with key clinical data expected in late 2025 and early 2026.

Precision BioSciences Reports First Clinical Evidence of Hepatitis B Gene Editing Therapy PBGENE-HBV

Gene Editing
  • Precision BioSciences' PBGENE-HBV demonstrated substantial antiviral activity in all three patients in the lowest dose cohort, achieving 47-69% reductions in hepatitis B surface antigen levels.
  • The gene editing therapy showed a well-tolerated safety profile with no serious adverse events or dose-limiting toxicities observed across multiple dose administrations.
  • One patient maintained a durable 50% reduction in HBsAg levels seven months after initial treatment, providing first clinical evidence of sustained viral DNA editing.
  • PBGENE-HBV represents the first and only clinical modality designed to eliminate covalently closed circular DNA and inactivate integrated DNA for potential hepatitis B cure.

Lonza, Excellos, and Akadeum Form Strategic Alliance to Transform Cell Therapy Manufacturing Quality

  • Lonza, Excellos, and Akadeum Life Sciences have announced a collaborative project aimed at redefining cell therapy manufacturing standards by focusing on starting material quality and integrated workflows.
  • The partnership combines Lonza's automated Cocoon® Platform, Akadeum's flotation-based cell separation technology, and Excellos' donor-to-dose capabilities to create modular manufacturing solutions.
  • The collaboration emphasizes how upstream quality decisions in donor sourcing and cell processing directly impact downstream therapeutic performance and clinical consistency.
  • Industry experts will share findings through an August 12 symposium and August 21 webinar, exploring technical integration opportunities for scalable cell and gene therapy manufacturing.

Protagonist Therapeutics Files First NDA for Icotrokinra in Psoriasis, Advances Rusfertide for Polycythemia Vera

Drug ApprovalRare Disease
  • Protagonist Therapeutics submitted its first New Drug Application (NDA) for icotrokinra to treat moderate to severe plaque psoriasis in adults and adolescents 12 years and older in July 2025.
  • The company presented positive Phase 3 VERIFY trial data for rusfertide in polycythemia vera at ASCO's plenary session, with a U.S. NDA filing expected in Q4 2025.
  • Protagonist reported $673 million in cash and equivalents as of June 30, 2025, providing runway through at least the end of 2028, despite a Q2 net loss of $34.8 million.
  • The company is advancing early-stage assets including PN-881, a first-in-class oral IL-17 peptide antagonist, and PN-477, a GLP-1/GIP/GCG triple agonist for obesity treatment.

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