MedPath

Clinical Trial News

Blacksmith Medicines Advances Novel Antibiotic FG-2101 Targeting Drug-Resistant Gram-Negative Bacteria

  • Blacksmith Medicines' FG-2101 represents a first-in-class non-hydroxamate inhibitor targeting LpxC, a zinc-dependent metalloenzyme found exclusively in Gram-negative bacteria.
  • The antibiotic candidate has completed IND-enabling studies and is positioned to enter human trials later this year for treating drug-resistant Gram-negative infections.
  • FG-2101's novel mechanism spares beneficial Gram-positive bacteria in the gut microbiome, potentially reducing C. difficile infection risk compared to broad-spectrum antibiotics.
  • The compound addresses a critical unmet medical need, as no approved therapeutics currently target LpxC despite its attractiveness as an antibiotic target.

Novel Nanoparticle Formulations Enhance Paclitaxel Delivery to Cancer Tumors

Targeted Therapy
  • University of Arizona researchers developed Paclitaxome, a new nanovesicle formulation of paclitaxel that outperformed standard chemotherapy drugs Taxol and Abraxane in mouse models of triple-negative breast cancer and advanced pancreatic cancer.
  • The formulation chemically attaches paclitaxel to sphingomyelin to create nanovesicles that improve tumor delivery, extend circulation time, and reduce accumulation in healthy tissues like the liver and spleen.
  • Indian researchers created fluorescent nanoparticles coated with transferrin that selectively target cancer cells overexpressing transferrin receptors, releasing over 90% of paclitaxel over two days while sparing normal cells.
  • Both platforms demonstrate the potential for improved drug delivery systems that could reduce chemotherapy side effects while enhancing therapeutic efficacy across multiple cancer types.

Long-Term Syfovre Data Shows Early Treatment Benefits and Sustained Safety in Geographic Atrophy

  • New four-year data from OAKS, DERBY, and GALE trials demonstrates that patients starting Syfovre treatment earlier preserve more retinal tissue and visual function compared to those who delayed therapy by two years.
  • The GALE extension study confirms Syfovre's safety profile remains consistent over extended treatment periods, with low rates of intraocular inflammation and stable wet AMD conversion rates in single digits annually.
  • Every-other-month dosing appears to offer comparable efficacy to monthly injections while providing better tolerability and safety profile for long-term treatment.
  • Patients who develop wet AMD while on Syfovre can be successfully managed with concurrent anti-VEGF therapy without compromising vision outcomes.

Purgo Scientific Appoints James Bowman as CEO to Advance Novel Drug Delivery Platform

  • Purgo Scientific, a medical device company developing localized drug delivery technologies, has appointed James A. Bowman as its new Chief Executive Officer.
  • Bowman brings over 20 years of experience in leading early-stage life science companies and commercializing breakthrough medical technologies across multiple specialties.
  • The company's flagship innovation represents a new frontier in site-specific drug delivery with applications in both human and veterinary medicine.
  • Under Bowman's leadership, Purgo will accelerate product development and build strategic partnerships to advance its platform technology currently under regulatory development.

Hansa Biopharma's Imlifidase Enables Gene Therapy Access for Duchenne Patients with Pre-existing Antibodies

Rare Disease
  • Hansa Biopharma reported that imlifidase successfully reduced IgG antibodies by ≥95% and anti-AAV antibodies below 1:400 titre in three Duchenne muscular dystrophy patients, enabling treatment with Sarepta's ELEVIDYS gene therapy.
  • The SRP-9001-104 trial demonstrated that imlifidase pre-treatment allowed previously ineligible patients to receive gene therapy, though micro-dystrophin expression levels were lower than observed in other ELEVIDYS trials.
  • This represents the first clinical evidence that imlifidase can overcome antibody barriers that prevent approximately one in three patients from accessing AAV-based gene therapies.

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

NCT06241950Enrolling by InvitationPhase 1
Sarepta Therapeutics, Inc.
Posted 1/29/2024

Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx

NCT04935177CompletedPhase 3
Hansa Biopharma AB
Posted 10/14/2021

Terbium-161 Radioimmunotherapy Shows Superior Efficacy Against Ovarian Cancer Stem Cells in Preclinical Study

Targeted Therapy
  • A novel radioimmunotherapy using Terbium-161 (¹⁶¹Tb) successfully eliminated ovarian cancer stem cells in preclinical models, outperforming the current gold standard Lutetium-177.
  • The ¹⁶¹Tb-DOTA-chCE7 conjugate completely eradicated all detected ovarian cancer stem cells and their differentiated progeny in live animal models, effectively preventing tumor growth.
  • The superior efficacy stems from ¹⁶¹Tb's emission of short-range conversion and Auger electrons alongside beta particles, enabling highly localized energy deposition and irreparable DNA damage within cancer stem cells.
  • This breakthrough offers renewed hope for targeting therapy-resistant cancer stem cells that drive tumor relapse, metastasis, and treatment resistance in ovarian cancer patients.

Allan Camaisa Joins Res Nova Biologics Advisory Board to Advance Breast Cancer Immunotherapy

  • Former Calidi Biotherapeutics Chairman and CEO Allan Camaisa has joined Res Nova Biologics' Advisory Board to accelerate development of breast cancer immunotherapies.
  • Res Nova is advancing FloraStilbene™, a proprietary combination of RU-486 and pterostilbene that showed tumor regression in early trials with no adverse events in four patients.
  • The company is also developing ValloVax Technology, a tumor-targeted delivery system using FDA-cleared blood vessel targeting to administer cancer-killing viruses intravenously.
  • Camaisa's expertise in digital health platforms and clinical trial optimization could streamline Res Nova's development processes and reduce costs for their 10-patient proof-of-concept trial.

Sucralose Artificial Sweetener Reduces Cancer Immunotherapy Effectiveness Through Gut Microbiome Disruption

  • University of Pittsburgh researchers found that sucralose consumption is associated with worse overall response and progression-free survival in cancer patients receiving anti-PD-1 immunotherapy.
  • The artificial sweetener disrupts gut microbiota, leading to depletion of arginine, an amino acid essential for T-cell function and anticancer activity.
  • Mouse studies demonstrated that arginine or citrulline supplementation can restore immunotherapy effectiveness even when sucralose consumption continues.
  • The findings, published in Cancer Discovery, suggest a potential therapeutic strategy to counteract dietary interference with cancer treatment without requiring patients to eliminate artificial sweeteners.

Spine BioPharma's SB-01 Fails to Meet Primary Endpoint in Phase 3 Trial for Chronic Low Back Pain

  • Spine BioPharma's Phase 3 MODEL trial evaluating SB-01, a TGF-β antagonist for chronic low back pain associated with degenerative disc disease, failed to meet its primary endpoint despite showing clinically meaningful improvements.
  • The 417-patient randomized controlled trial achieved a 67% success rate with SB-01 at Month 6, but this did not reach statistical significance compared to sham control due to unexpectedly high placebo response rates.
  • In sites with anticipated sham control responses, SB-01 nearly achieved statistical significance (p=0.051), suggesting the treatment may be effective when placebo effects are controlled.
  • The company plans to meet with the FDA to discuss potential approval pathways based on the Phase 3 results combined with earlier Phase 1 and 2 trial data.

Moderate - Severe Degenerative Disc Disease Evaluation of the Lumbar Spine

NCT05516992Active, Not RecruitingPhase 3
Spine BioPharma, Inc
Posted 8/19/2022

FDA Issues Second Rejection for Regeneron's Lymphoma Therapy Odronextamab Due to Manufacturing Issues

  • The FDA declined to approve Regeneron's lymphoma therapy odronextamab for the second time, citing manufacturing issues at a third-party Catalent facility rather than safety or efficacy concerns.
  • The rejection stems from problems discovered during an FDA inspection at Catalent's Indiana facility, which handles final drug preparation and packaging for odronextamab.
  • Despite FDA setbacks, odronextamab received European Union approval in August 2024 for treating relapsed/refractory follicular lymphoma and diffuse large B-cell lymphoma.
  • Regeneron must address the manufacturing concerns and resubmit its biologics license application while continuing its phase 3 OLYMPIA-1 trial.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.