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Orchestra BioMed Expands BACKBEAT Hypertension Trial Eligibility 24-Fold Following FDA Protocol Approval

FDA Approval
  • Orchestra BioMed received FDA approval for a protocol update that expands patient eligibility for the BACKBEAT pivotal study by more than 24-fold compared to the original protocol.
  • The expanded criteria now include any hypertensive patients with Medtronic Azure or Astra pacemakers, including device replacements and those with NYHA class I or II heart failure.
  • The company targets mid-2026 for completion of enrollment in the study evaluating AVIM therapy for uncontrolled hypertension in pacemaker patients.
  • AVIM therapy previously demonstrated net reductions of 8.1 mmHg in ambulatory systolic blood pressure and 12.3 mmHg in office systolic blood pressure in the MODERATO II pilot study.

Arcellx Reports Strong CAR-T Clinical Data Despite 72% Revenue Drop in Q2 2025

CAR-TFDA Approval
  • Arcellx's lead CAR-T therapy anito-cel achieved a 97% overall response rate and 93.3% minimal residual disease negativity in 117 multiple myeloma patients from the Phase 2 iMMagine-1 study.
  • The company's Q2 2025 collaboration revenue dropped 72% to $7.6 million, missing analyst expectations by 43.8% due to completion of clinical trial manufacturing activities.
  • Net loss more than doubled to $52.8 million as the company increased commercial readiness spending ahead of a planned 2026 product launch.
  • FDA authorized an investigational new drug application for ACLX-004, expanding Arcellx's pipeline into acute myeloid leukemia treatment.

FibroGen Receives FDA Approval for Phase 3 Trial of Roxadustat in Lower-Risk MDS Patients with High Transfusion Burden

FDA Approval
  • FibroGen has reached agreement with the FDA on key design elements for a pivotal Phase 3 trial of roxadustat in lower-risk myelodysplastic syndrome patients with high transfusion burden.
  • The oral HIF-PH inhibitor demonstrated promising efficacy in a post-hoc analysis, with 36% of high transfusion burden patients achieving transfusion independence compared to 7% on placebo.
  • The randomized, placebo-controlled trial will enroll approximately 200 patients and evaluate 8-week or 16-week transfusion independence as the primary endpoint.
  • FibroGen plans to submit the Phase 3 protocol to the FDA in the fourth quarter of 2025, addressing a significant unmet medical need in this patient population.

Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden

NCT03263091TerminatedPhase 3
FibroGen
Posted 1/29/2018

FDA Approves AJOVY as First CGRP Antagonist for Pediatric Migraine Prevention

FDA ApprovalDrug Approval
  • Teva Pharmaceutical Industries received FDA approval for AJOVY as the first CGRP antagonist indicated for preventive treatment of episodic migraine in children and adolescents aged 6-17 weighing at least 45 kilograms.
  • This approval expands Teva's neuroscience portfolio and provides a new treatment pathway for a pediatric population with previously limited preventive migraine options.
  • The approval supports Teva's Pivot to Growth strategy and contributed to the company raising its 2025 revenue guidance to $16.8-17.2 billion.
  • Analysts project Teva will reach $17.9 billion in revenue and $1.5 billion in earnings by 2028, representing a 2.6% annual revenue growth rate.

FDA Provides Positive Feedback on Immutep's Eftilagimod Alfa for Head and Neck Cancer Treatment

FDA Approval
  • Immutep received positive FDA feedback on eftilagimod alfa development for first-line treatment of head and neck squamous cell carcinoma patients with PD-L1 expression below 1.
  • The FDA agreed on the potential of efti combined with KEYTRUDA to address high unmet need in this patient segment, representing up to 20% of first-line HNSCC patients.
  • FDA outlined potential accelerated approval pathways including a randomized registrational trial or smaller single-arm study under Project FrontRunner initiative.
  • Current treatment options for PD-L1 CPS <1 patients are limited to chemotherapy, as anti-PD-1 therapy alone is only approved for patients with CPS >1.

Combination Study With Eftilagimod Alpha (a Soluble LAG-3 Fusion Protein) and Pembrolizumab in Patients With Recurrent or Metastatic HNSCC

NCT04811027Active, Not RecruitingPhase 2
Immutep S.A.S.
Posted 8/27/2021

First Commercial Brain Gene Therapy Treatments Delivered in US Using ClearPoint Neuro's SmartFlow Cannula

FDA ApprovalRare DiseaseNeurology
  • The first commercial gene therapy treatments ever delivered directly to the brain in the United States have been successfully performed using ClearPoint Neuro's SmartFlow Neuro Cannula to administer KEBILIDI for AADC deficiency.
  • KEBILIDI (eladocagene exuparvovec-tneq) is delivered directly to the putamen region of the brain through stereotactic neurosurgery, with the SmartFlow cannula being the only FDA-authorized device for this administration.
  • ClearPoint Neuro reported record clinical activity in July with 17 global patients treated across 11 different drug platforms, while nine of the company's partners have now been accepted into FDA expedited review programs.
  • New ICD-10 codes specific to neuro infusions will become effective October 1, 2025, to assist in tracking commercial use of brain-delivered therapies.

TransMedics Receives FDA Approval for Largest Heart Transplant Preservation Trial

FDA Approval
  • TransMedics received FDA conditional approval for its Investigational Device Exemption to initiate the Next-Generation OCS ENHANCE Heart trial, marking a significant regulatory milestone.
  • The two-part ENHANCE trial will exceed 650 patients and aims to demonstrate superiority of OCS Heart perfusion over static cold storage methods in donation after brain death cases.
  • The company plans to initiate both the ENHANCE Heart and DENOVO Lung trials in Q4 2025, positioning these studies as major catalysts for clinical adoption throughout 2026.
  • TransMedics believes this will constitute the largest heart preservation for transplant trial ever conducted worldwide, addressing critical unmet needs in cardiothoracic transplantation.

Anbogen Receives FDA Clearance for Phase 1/2 Trial of ABT-301 Triplet Therapy in Metastatic Colorectal Cancer

FDA Approval
  • Anbogen Therapeutics received FDA approval for its Investigational New Drug application to initiate a Phase 1/2 clinical trial of ABT-301 combined with tislelizumab and bevacizumab for metastatic colorectal cancer patients.
  • The open-label, multi-center international study will enroll 66 patients with proficient mismatch repair or non-microsatellite instability-high metastatic colorectal cancer in Taiwan and Australia.
  • ABT-301 is an oral HDAC1/2/3 inhibitor that converts "cold tumors" into "hot tumors" by promoting CD8+ T cell infiltration and enhancing immune checkpoint inhibitor efficacy.
  • The therapy targets approximately 95% of metastatic colorectal cancer patients who respond poorly to current immunotherapies, representing a potential $9 billion market opportunity.

Dose-Seeking Study of MPT0E028 in Subjects With Advanced Solid Malignancies Without Standard Treatment

NCT02350868CompletedPhase 1
Taipei Medical University
Posted 4/24/2015

MAGENTIQ EYE Secures Series A Funding to Advance AI-Powered Colonoscopy Technology

AIFDA Approval
  • MAGENTIQ EYE completed its Series A funding round led by aMoon to accelerate commercialization of its FDA- and CE-approved AI colonoscopy software MAGENTIQ-COLO in the USA and Europe.
  • The company's AI-powered software significantly improves adenoma detection rates during colonoscopies and offers real-time polyp analysis with size and type estimation capabilities.
  • Funding will support ongoing clinical trials for diagnostic tools targeting ulcerative colitis, Barrett's esophagus dysplasia, and gastric intestinal metaplasia while expanding global market access.
  • The investment reflects growing commercial traction with dozens of daily procedures and a robust pipeline of trial and purchase requests across international markets.

Alphamab Oncology's HER2-Targeting ADC JSKN003 Receives FDA Orphan Drug Designation for Gastric Cancer

FDA Approval
  • Alphamab Oncology's anti-HER2 biparatopic antibody-drug conjugate JSKN003 has been granted FDA Orphan Drug Designation for treating gastric cancer and gastroesophageal junction cancer.
  • The designation provides significant regulatory advantages including tax credits, fee waivers, accelerated review processes, and potential seven-year market exclusivity upon approval.
  • JSKN003 demonstrated favorable safety and promising efficacy in Phase I clinical studies, particularly in heavily pretreated patients with high HER2-expressing gastrointestinal tumors.
  • Gastric and gastroesophageal junction cancers represent a significant unmet medical need with poor survival outcomes, affecting approximately 960,000 new cases globally and 26,890 cases in the U.S. annually.

Safety and Tolerability of JSKN003 in Chinese Subjects With Advanced Solid Tumors

NCT05744427RecruitingPhase 1
Jiangsu Alphamab Biopharmaceuticals Co., Ltd
Posted 3/15/2023

First-In-Human Study in Subjects With Advanced or Metastatic Solid Malignant Tumors

NCT05494918CompletedPhase 1
Alphamab (Australia) Co Pty Ltd.
Posted 9/2/2022

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