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Clinical Trial News

Engineered CD40 Antibody Achieves Complete Cancer Remission in Phase 1 Trial

Monoclonal Antibody
  • A modified CD40 agonist antibody called 2141-V11 demonstrated remarkable efficacy in a phase 1 clinical trial, with six of 12 patients experiencing significant tumor reduction and two achieving complete remission.
  • The drug showed systemic anti-tumor effects when injected directly into tumors, causing cancer disappearance at both injected and distant sites without the severe toxicities associated with previous CD40 therapies.
  • Tissue analysis revealed the formation of tertiary lymphoid structures within tumors, creating immune-rich microenvironments that replaced cancerous tissue with organized immune cell aggregates.
  • Nearly 200 patients are now enrolled in follow-up phase 1 and phase 2 trials investigating the drug's effectiveness against specific aggressive cancers including bladder cancer, prostate cancer, and glioblastoma.

A Study Investigating the Safety and Tolerability of an Immune Treatment in Cancer Patients With Lesions to the Skin

NCT04059588CompletedPhase 1
Rockefeller University
Posted 1/16/2020

Basilea Pharmaceutica Acquires Global Rights to Phase 3-Ready Oral Antibiotic for Drug-Resistant UTIs

  • Basilea Pharmaceutica has entered an exclusive licensing agreement with Venatorx Pharmaceuticals to acquire global rights to ceftibuten-ledaborbactam etzadroxil, a novel oral antibiotic combination targeting complicated urinary tract infections.
  • The drug demonstrates bactericidal activity against multidrug-resistant Enterobacterales pathogens and addresses a critical unmet need for oral treatment options in cUTI, which accounts for over 600,000 hospital admissions annually in the US.
  • Basilea plans to initiate a registrational Phase 3 clinical program within approximately 18 months, with the transaction potentially worth up to $325 million in milestone payments to Venatorx.
  • The antibiotic combination has received FDA Qualified Infectious Disease Product and Fast Track designations, positioning it as a promising solution for treating infections caused by extended-spectrum beta-lactamase producing bacteria.

VNRX-7145 SAD/MAD Safety and PK in Healthy Adult Volunteers

NCT04243863CompletedPhase 1
Venatorx Pharmaceuticals, Inc.
Posted 1/20/2020

Herantis Pharma Completes Phase 1b Trial of HER-096 for Parkinson's Disease, Topline Results Expected Within Nine Weeks

  • Herantis Pharma has completed the final patient visit in its Phase 1b clinical trial of HER-096, a first-in-class small peptide designed to mimic cerebral dopamine neurotrophic factor for Parkinson's disease treatment.
  • The trial evaluated safety, tolerability, and pharmacokinetics of repeated subcutaneous doses in Parkinson's patients, with topline results expected within six to nine weeks following data analysis.
  • HER-096 represents a potential breakthrough as the first disease-modifying therapy for Parkinson's disease, targeting over 10 million patients worldwide who currently lack treatments that can stop disease progression.

Clinical Study of HER-096 in Healthy Volunteer Subjects and Patients With Parkinson's Disease

NCT06659562Active, Not RecruitingPhase 1
Herantis Pharma Plc.
Posted 9/23/2024

Vedanta Biosciences Cuts 20% of Workforce After Microbiome Therapy VE202 Fails Phase 2 Ulcerative Colitis Trial

  • Vedanta Biosciences will lay off nearly 20% of its staff after experimental microbiome therapy VE202 failed to meet its primary endpoint in a Phase 2 ulcerative colitis study.
  • The VE202 treatment, licensed by Johnson & Johnson in 2015, was not significantly better than placebo at improving disease signs on endoscopic examination after eight weeks of treatment.
  • The company will now focus resources on VE303, a microbiome therapy in Phase 3 testing for recurrent C. diff infections, and a preclinical therapy for antibiotic-resistant bacterial infections.
  • The failure adds to mounting evidence of challenges in developing microbiome-based therapies for inflammatory bowel disease, following similar setbacks from competitors like Seres Therapeutics.

Aquestive Therapeutics Secures $75M Strategic Funding from RTW for Anaphylm Commercial Launch

  • Aquestive Therapeutics announced a $75 million strategic funding agreement with RTW Investments to support the commercial launch of Anaphylm, contingent on FDA approval.
  • Anaphylm is positioned as potentially the first and only oral rescue medication for severe allergic reactions including anaphylaxis, offering a novel alternative to injectable treatments.
  • The funding will provide critical capital through 2027 and includes a tiered single-digit percentage of annual net sales in the U.S. for type I allergic reactions treatment.
  • Anaphylm features a postage stamp-sized sublingual film format that dissolves on contact without requiring water or swallowing, packaged in a credit card-thin carrier.

EvolveImmune Receives FDA Clearance for Novel Trispecific T Cell Engager EVOLVE104 Targeting Solid Tumors

  • EvolveImmune Therapeutics announced FDA clearance of its Investigational New Drug application for EVOLVE104, a novel trispecific T cell engager with integrated CD2 costimulation targeting ULBP2/5/6 antigens.
  • The company plans to initiate a Phase 1 clinical trial of EVOLVE104 in 2025, marking the first program from its EVOLVE platform to advance into clinical development.
  • Preclinical studies demonstrated compelling single-agent and combination activity with anti-PD-1 therapy, along with the ability to reinvigorate dysfunctional T cells for improved tumor growth inhibition.
  • EVOLVE104 represents a first-in-category approach designed to overcome cancer cell resistance to current immunotherapies in solid tumors and hematologic malignancies.

Goa Becomes India's First State to Implement Pricing Policy for High-Cost Cancer and Rare Disease Therapies

Rare Disease
  • Goa government launches India's first state-level pricing policy to negotiate lower prices for expensive cancer and rare disease treatments with pharmaceutical companies.
  • The initiative targets therapies costing over Rs 50 lakh annually, starting with lung cancer treatments, and aims to extend access to more patients through confidential pricing agreements.
  • A specialized committee will oversee price negotiations and approvals, addressing Goa's growing healthcare burden of 1,500 new cancer cases annually with 5% yearly increase.
  • The policy framework focuses on proprietary drugs and equipment already procured by state medical institutions, setting a national precedent for equitable healthcare access.

Taiwan Accepts Everest Medicines' VELSIPITY Application for Ulcerative Colitis Treatment

Drug Approval
  • Taiwan's Food and Drug Administration has officially accepted Everest Medicines' New Drug Application for VELSIPITY (etrasimod) to treat moderately to severely active ulcerative colitis.
  • The acceptance builds on prior approvals in Singapore, Hong Kong SAR, and Macao SAR, marking significant progress in VELSIPITY's commercialization across Asia.
  • Clinical data supporting the application comes from Phase 3 studies including the ENLIGHT trial, which enrolled 340 Asian patients and achieved mucosal healing in 51.9% of treated patients.
  • With approximately 800,000 UC patients in China in 2024 projected to reach 1 million by 2030, the drug addresses a significant unmet medical need in the region.

Reprieve Cardiovascular Secures $61 Million Series B Funding, Initiates Pivotal Heart Failure Trial

  • Reprieve Cardiovascular closed an oversubscribed $61 million Series B financing led by Deerfield Management to advance its intelligent decongestion management therapy for acute decompensated heart failure.
  • The company enrolled its first patient in the FASTR II pivotal clinical trial, which will evaluate the Reprieve system's efficacy compared to optimal diuretic therapy in up to 400 patients across the United States and Europe.
  • The Reprieve system combines real-time physiological monitoring with automated recommendations to personalize fluid removal therapy, addressing significant challenges in current heart failure management where nearly 1 in 4 patients are readmitted within 30 days.

Liquidia's YUTREPIA Achieves Rapid Market Uptake with 900+ Prescriptions in 11 Weeks Post-Approval

Drug ApprovalRare Disease
  • Liquidia Corporation's YUTREPIA (treprostinil) inhalation powder achieved over 900 unique patient prescriptions and 550 patient starts within 11 weeks of FDA approval for treating PAH and PH-ILD.
  • The ASCENT trial interim data demonstrated sustained efficacy with median improvements in six-minute walk distance of 31.5 meters at Week 16, with no discontinuations due to drug-related adverse events.
  • The company reported $6.5 million in product revenue for Q2 2025 and maintains a strong financial position with $173.4 million in cash and cash equivalents.

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