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Clinical Trial News

Rocket Pharmaceuticals Cuts 30% of Workforce, Refocuses on Cardiovascular Gene Therapies

  • Rocket Pharmaceuticals is laying off 80 employees, representing 30% of its workforce, to reduce cash burn by almost 25% and extend its runway into 2027.
  • The company is narrowing its pipeline focus to prioritize late-stage cardiovascular gene therapies for Danon disease and cardiomyopathy conditions following regulatory setbacks.
  • The strategic realignment comes after an FDA clinical hold on its pivotal Danon disease trial following a patient death and previous rejection of its Kresladi gene therapy.
  • Rocket's hematology programs, including RP-L102 for Fanconi anemia previously targeted for 2025 approval, face significant delays as resources shift to cardiovascular assets.

Leads Biolabs Raises $189 Million in Hong Kong IPO, Advances First-in-Class 4-1BB Immunotherapy

  • Leads Biolabs successfully listed on the Hong Kong Stock Exchange, raising $189 million to fund clinical trials and expand its immuno-oncology pipeline.
  • The company's lead candidate LBL-024 became the world's first 4-1BB-targeted molecule to reach registrational trials, showing 75% response rates in neuroendocrine carcinoma.
  • The biotech operates proprietary platforms including LeadsBody™ for T-cell engagers and X-body™ for 4-1BB agonists, with 14 drug candidates across oncology and autoimmune diseases.
  • LBL-024 received breakthrough therapy designation from China's NMPA and orphan drug status from the FDA for neuroendocrine neoplasms.

Absci Raises $50 Million to Advance AI-Driven Drug Discovery Platform and Internal Pipeline

AIDrug Discovery
  • Absci Corporation completed a $50 million public offering of 16.67 million shares at $3.00 per share to fund its AI-powered drug discovery programs and internal pipeline development.
  • The company's cash reserves increased to $134 million as of March 2025, extending its operational runway through 2027 while advancing preclinical candidates ABS-101 and ABS-201.
  • Absci's Integrated Drug Creation platform combines generative AI with synthetic biology and has secured partnerships with pharmaceutical giants including Merck and Moderna.
  • The AI-driven biologics market is projected to grow from $13.9 billion in 2024 to over $30 billion by 2030, positioning Absci in a rapidly expanding sector.

PROCEPT BioRobotics Expands Prostate Cancer Trial to Outpatient Setting with First ASC Procedures

  • East Valley Urology completed the first three prostate cancer procedures in PROCEPT BioRobotics' FDA-approved WATER IV trial using Aquablation therapy in an ambulatory surgery center setting.
  • The outpatient procedures were performed without complications and achieved same-day discharge, demonstrating potential for more scalable and cost-effective prostate cancer treatment.
  • The WATER IV trial compares Aquablation therapy to radical prostatectomy in men with localized prostate cancer (grade group 1 to 3).
  • Aquablation therapy uses robotically controlled waterjet technology to remove prostate tissue while potentially preserving erectile, ejaculatory, and continence functions.

Magnetically Steerable Drug Delivery Vesicles Show Promise for Precision Cancer Treatment

Precision MedicineTargeted Therapy
  • Researchers at the University of Illinois Urbana-Champaign have developed microscopic lipid vesicles containing magnetic particles that can be precisely steered to target locations using magnetic fields.
  • The system combines magnetic steering with light-triggered drug release, creating a comprehensive prototype for precision drug delivery that could significantly advance cancer treatment.
  • The lipid vesicles are designed to interact only with specific cell types, offering advantages for targeted cancer therapy while utilizing existing MRI technology for navigation.
  • The team is now preparing for in vitro studies using real drugs in microfluidic systems that simulate biological environments.

Janux Therapeutics Advances Multi-Platform Pipeline with Novel ARM Technology for Autoimmune Diseases

Oncology
  • Janux Therapeutics showcased its expanded immunotherapy pipeline at R&D Day, highlighting three proprietary platforms: TRACTr, TRACIr, and the novel ARM platform targeting autoimmune diseases.
  • The company's CD19-ARM demonstrated rapid, deep B-cell depletion with prolonged memory B cell reset in non-human primates while maintaining a large safety window, positioning it for first-in-human trials in H1 2026.
  • Clinical programs JANX007 and JANX008 continue enrollment with data updates expected in H2 2025, while new TROP2-TRACTr and PSMA-TRACIr programs advance toward clinical development.
  • The company reported $996.0 million in cash and investments as of Q2 2025, providing substantial runway for its expanding development programs.

Study of JANX007 in Subjects With Metastatic Castration-Resistant Prostate Cancer (ENGAGER-PSMA-01)

NCT05519449RecruitingPhase 1
Janux Therapeutics
Posted 9/15/2022

Study of JANX008 in Subjects With Advanced or Metastatic Solid Tumor Malignancies

NCT05783622RecruitingPhase 1
Janux Therapeutics
Posted 4/19/2023

NextCure's Anti-Siglec-15 Antibody NC605 Shows Promise for Osteogenesis Imperfecta Treatment in Preclinical Study

Monoclonal AntibodyRare Disease
  • NextCure's NC605, a novel anti-Siglec-15 antibody, demonstrated improved bone microarchitecture and reduced fracture incidence in preclinical studies of osteogenesis imperfecta mice.
  • The treatment showed superior bone quality outcomes compared to current anti-resorptive therapies by both inhibiting bone loss and producing new bone with increased quality and density.
  • NC605 represents a potential breakthrough for osteogenesis imperfecta patients, as there is currently no FDA-approved standard of care for this rare brittle bone disease.
  • NextCure is seeking financial support to advance NC605 toward an Investigational New Drug submission within 12 to 18 months.

Insmed's Brensocatib Wins FDA Approval as First Treatment for Non-Cystic Fibrosis Bronchiectasis

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved Insmed's brensocatib (Brinsupri) as the first treatment for non-cystic fibrosis bronchiectasis, a progressive lung condition that can cause permanent lung damage.
  • The approval represents a "best-case scenario" with both doses approved and no major warnings added, positioning the drug for a potentially rapid commercial launch.
  • Analysts project Brinsupri could generate $3.8 billion in U.S. sales and over $6 billion worldwide, with the drug priced at $88,000 annually.
  • Insmed is also developing brensocatib for additional inflammatory conditions including chronic rhinosinusitis and hidradenitis suppurativa, leveraging its DPP1 enzyme blocking mechanism.

Stevanato Group Secures €200 Million Financing to Expand Biologics Manufacturing Capacity

  • Stevanato Group secured €200 million in financing from three major European financial institutions to support capital expenditure projects in Italy and the United States.
  • The funding will enhance pre-filled syringe production capacity and establish ready-to-use cartridge manufacturing capabilities to meet growing customer demand for biologics programs.
  • CDP committed €50 million for Italian operations, while BNL BNP Paribas provided €100 million and Banco BPM contributed €50 million for scaling production across both regions.
  • The financing supports ongoing construction for device manufacturing in Fishers, Indiana, and capacity expansion at the Cisterna di Latina facility in Italy.

Bayer Initiates Phase Clinical Trial for BAY3018250 in Deep Vein Thrombosis Treatment

  • Bayer AG has launched a randomized, double-blind, placebo-controlled clinical study to evaluate BAY3018250, a novel drug designed to dissolve blood clots in patients with symptomatic proximal deep vein thrombosis.
  • The interventional study follows a quadruple-masked design comparing BAY3018250 against placebo to assess both efficacy in reducing clot burden and safety profile in DVT patients.
  • The trial began on January 15, 2024, and is currently recruiting participants, with potential positive results expected to enhance Bayer's competitive position in the cardiovascular treatment market.

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