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Clinical Trial News

BRACELET-1 Trial Shows Promise for Oncolytic Virus Pelareorep in HR+ Breast Cancer

  • The BRACELET-1 trial demonstrated that pelareorep combined with paclitaxel achieved a 31% objective response rate and 12.1-month median progression-free survival in HR+ HER2- metastatic breast cancer patients.
  • Patients receiving the paclitaxel-pelareorep combination showed enhanced T-cell proliferation by cycle 4, suggesting improved immune activation compared to paclitaxel alone.
  • Adding avelumab to the combination paradoxically reduced efficacy and increased toxicity, with the triple regimen showing only 14% response rate and 5.8-month median PFS.
  • The findings support further investigation of pelareorep as an immunotherapy option for HR+ HER2- breast cancer, a subtype historically resistant to immune-based treatments.

A Study to Assess Overall Response Rate by Inducing an Inflammatory Phenotype in Metastatic BReast cAnCEr With the Oncolytic Reovirus PeLareorEp in CombinaTion With Anti-PD-L1 Avelumab and Paclitaxel - BRACELET-1 Study

NCT04215146Active, Not RecruitingPhase 2
Oncolytics Biotech
Posted 6/10/2020

Cue Biopharma Receives Positive FDA Feedback for CUE-401 Autoimmune Therapy, Advances Toward IND Filing

  • Cue Biopharma received positive Pre-IND feedback from the FDA for CUE-401, a first-in-class bispecific fusion protein designed to treat autoimmune diseases by inducing regulatory T cells.
  • The FDA reviewed the company's first-in-human trial design, dose escalation plan, and safety monitoring protocols, enabling Cue Biopharma to proceed with IND filing after completing final enabling studies.
  • CUE-401 combines TGF-β and IL-2 activities to convert autoreactive T cells into regulatory T cells, potentially establishing immune tolerance across multiple autoimmune conditions.
  • The novel therapy aims to provide durable immune rebalance by creating a "tolerance positive feedback loop" that specifically targets disease-causing autoantigens.

NHS Delivers First Brain-Infused Gene Therapy for Rare AADC Deficiency, Showing Promising Results in Three-Year-Old Patient

Rare Disease
  • Three-year-old Gunreet Kaur received Upstaza (eladocagene exuparvovec), the first NHS England commissioned gene therapy infused directly into the brain, at Great Ormond Street Hospital in February 2024.
  • The treatment targets aromatic l-amino acid decarboxylase (AADC) deficiency, a rare and often fatal genetic condition that prevents dopamine production and causes severe developmental delays.
  • Following gene therapy, Gunreet demonstrated significant improvements including better head control, coordination, reduced crying, and new motor skills like rolling over.
  • The therapy uses a virus to deliver a working AADC gene directly to brain nerve cells with millimetre precision using robotic surgery assistance.

Eli Lilly's Olomorasib Receives FDA Orphan Drug Designation for KRAS G12C-Mutated NSCLC Treatment

Targeted Therapy
  • The FDA has granted orphan drug designation to Eli Lilly's Olomorasib for treating non-small cell lung cancer with KRAS G12C mutations, providing market exclusivity and development incentives.
  • Olomorasib is currently in Phase 3 clinical trials as a first-line treatment combined with Keytruda (pembrolizumab), with or without chemotherapy, for advanced NSCLC patients.
  • Previous ASCO data demonstrated that Olomorasib (50 or 100mg BID) combined with Keytruda showed promising safety and anti-tumor activity in KRAS G12C-mutated NSCLC patients.
  • The designation addresses an unmet medical need for patients with KRAS G12C mutations, a common driver mutation in NSCLC with limited treatment options.

Guselkumab Demonstrates High Efficacy for Psoriasis Treatment Across All Skin Tones in Landmark VISIBLE Trial

Monoclonal Antibody
  • The VISIBLE trial represents the first prospective randomized clinical trial evaluating psoriasis treatment across the complete spectrum of objectively measured skin tones, addressing critical gaps in dermatology research for underrepresented populations.
  • Guselkumab achieved remarkable efficacy rates with approximately 70% of participants reaching clear or almost clear skin and over 50% achieving complete skin clearance by week 48, with mean improvements exceeding 94% in both body and scalp psoriasis measures.
  • The study successfully enrolled and retained over 90% of participants with skin of color through innovative trial design features, demonstrating that diverse clinical trial participation is achievable in dermatology research.
  • Safety profiles remained consistent with established guselkumab data, with infections being the most common adverse events and no serious safety signals identified across the diverse patient population.

Liquidia Secures $50 Million Funding Following YUTREPIA Commercial Launch and Legal Victory

Drug ApprovalRare Disease
  • Liquidia Corporation received $50 million from Healthcare Royalty following the first commercial sale of YUTREPIA and a favorable court ruling denying United Therapeutics' injunction request.
  • The company has now accessed $175 million of $200 million in total potential funding, with an additional $25 million available if YUTREPIA achieves net sales exceeding $100 million by June 2026.
  • YUTREPIA is an inhaled dry-powder formulation of treprostinil approved for treating pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.
  • The funding will accelerate launch execution, advance clinical pipeline development, and support manufacturing facility expansion as Liquidia targets profitability without additional capital needs.

Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil

NCT03399604CompletedPhase 3
Liquidia Technologies, Inc.
Posted 1/2/2018

An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in PH

NCT06129240Recruiting
Liquidia Technologies, Inc.
Posted 12/28/2023

Fortrea and Emery Pharma Partner to Enable FDA-Compliant Rifampin Use in Drug-Drug Interaction Studies

  • Fortrea and Emery Pharma announced a strategic collaboration to provide rapid testing of rifampin for 1-methyl-4-nitrosopiperazine (MNP) impurity levels to meet FDA guidelines for drug-drug interaction studies.
  • The partnership addresses regulatory challenges that emerged in 2021 when FDA found all tested rifampin batches exceeded previously accepted MNP limits, forcing researchers to explore alternative drugs with safety concerns.
  • The collaboration enables sponsors to use rifampin as the preferred CYP3A4 inducer in DDI studies, potentially reducing early-phase trial risks and accelerating drug development timelines.

PathPresenter Receives FDA 510(k) Clearance for Digital Pathology Clinical Viewer

Precision Medicine
  • PathPresenter has received FDA 510(k) clearance for its Clinical Viewer platform, enabling primary diagnosis using digital pathology workflows.
  • The clearance is specifically for use with Hamamatsu NanoZoomer S360MD slide scanners and Barco NV MDPC-8127 display devices.
  • The platform addresses a critical need in healthcare, as pathology guides nearly 70% of clinical decision-making processes.
  • This regulatory milestone advances the adoption of digital workflows in pathology and supports precision medicine initiatives.

Cigna Sues Bristol Myers Squibb Over Alleged Monopoly Practices for Cancer Drug Pomalyst

  • Cigna filed a federal lawsuit against Bristol Myers Squibb, alleging the company used anti-competitive strategies to maintain a monopoly on Pomalyst, a multiple myeloma treatment that generated $2.7 billion in U.S. sales last year.
  • The insurer claims Bristol Myers' subsidiary Celgene filed sham patent lawsuits and paid off generic manufacturers to prevent lower-cost alternatives from entering the market.
  • Cigna alleges Celgene defrauded the U.S. Patent and Trademark Office by withholding information about an existing patent and claiming unexpected positive results to strengthen patent protections.
  • The lawsuit seeks triple damages for alleged overcharges of "many hundreds of millions, if not billions, of dollars" caused by the monopolistic practices.

Trinity Biotech Receives WHO Approval for Offshore Manufacturing of HIV Rapid Test

  • Trinity Biotech received World Health Organization approval for offshored and outsourced upstream manufacturing of its TrinScreen HIV rapid test, marking a critical milestone in the company's transformation plan.
  • The regulatory approval enables transition from legacy in-house operations to a more cost-effective and scalable outsourced model while maintaining product integrity and regulatory compliance.
  • The strategic move is expected to deliver substantial improvements in gross margin and working capital while enhancing supply chain resilience and scalability.
  • Trinity Biotech plans to implement this new TrinScreen HIV manufacturing model in Q3 2025 as part of broader efforts to streamline operations and reduce fixed costs.

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