MedPath

Clinical Trial News

RAPT Therapeutics Halts Zelnecirnon Trials in Atopic Dermatitis and Asthma Following FDA Clinical Hold

  • RAPT Therapeutics has decided to close and unblind its Phase 2b trial of zelnecirnon in atopic dermatitis and its Phase 2a trial in asthma after an FDA clinical hold.
  • The clinical hold was initiated due to a serious adverse event of liver failure requiring a transplant in one patient during the atopic dermatitis trial.
  • RAPT Therapeutics plans to analyze the data collected from the trials, even if not statistically significant, to inform future steps and discussions with the FDA.
  • The company's first-quarter net loss of 2024 was $30.5 million, and as of March 31, 2024, it held $141.6 million in cash and cash equivalents.

RAPT Therapeutics Reports Q1 2024 Financial Results and Clinical Trial Update

  • RAPT Therapeutics reported a net loss of $30.5 million for Q1 2024, aligning with analyst estimates, while EPS was slightly better than expected at -$0.79.
  • Research and development expenses decreased to $24.8 million, while general and administrative expenses increased to $7.7 million due to higher personnel and consulting costs.
  • The company's Phase 2b and 2a clinical trials for zelnecirnon were halted due to a serious adverse event, with data analysis expected in Q3 2024.
  • RAPT maintains a strong cash position with $141.6 million in cash and marketable securities, supporting ongoing research and strategic initiatives despite clinical setbacks.

FibroGen Highlights Pamrevlumab's Potential in Pancreatic Cancer and Roxadustat's Growth in Q1 2024 Earnings Call

  • FibroGen anticipates top-line data from Phase 3 trials of pamrevlumab in metastatic and locally-advanced pancreatic cancer, addressing a significant unmet need.
  • Roxadustat sales in China increased by 24% year-over-year, driven by a 39% volume increase, with potential for further growth pending approval for chemotherapy-induced anemia.
  • FG-3246, a first-in-class ADC, shows promising Phase 1 results in metastatic castration-resistant prostate cancer, with plans for a Phase 2/3 study in late 2024.
  • FibroGen's strong cash position of $214.7 million is expected to fund operations into 2026, supporting the advancement of its clinical programs.

Vertex Pharmaceuticals Reports Strong Q1 2024 Performance, Advances Pipeline

  • Vertex Pharmaceuticals reported a 13% revenue increase in Q1 2024, reaching $2.7 billion, driven by cystic fibrosis (CF) drug sales and the launch of CASGEVY for sickle cell disease and beta-thalassemia.
  • The company completed regulatory submissions in the U.S. and EU for its vanzacaftor triple therapy for CF patients aged 6 years and older, potentially setting a new standard in CF treatment.
  • Vertex is advancing suzetrigine (VX-548) for acute and neuropathic pain, with a rolling NDA submission underway and Phase III trials planned for painful diabetic peripheral neuropathy (DPN).
  • The acquisition of Alpine Immune Sciences is expected to close this quarter, adding povetacicept (pove), a Phase III-ready molecule for IgA nephropathy (IgAN), to Vertex's portfolio.

Axsome Therapeutics Reports Strong Q1 2024 Financial Results and Pipeline Advancements

  • Axsome Therapeutics reported Q1 2024 total net product revenue of $75 million, marking a 160% year-over-year growth, driven by strong performance of its marketed products.
  • The company advanced its neuroscience pipeline, with AXS-12 achieving positive Phase 3 results in narcolepsy and progress in trials for migraine, fibromyalgia, and Alzheimer's disease agitation.
  • Axsome is on track for NDA resubmission for AXS-07 (migraine) and NDA submission for AXS-14 (fibromyalgia) this quarter, with multiple Phase 3 trial results expected in 2024 and 2025.
  • The company's cash balance of $331.4 million is expected to fund operations into cash flow positivity, supporting continued commercial expansion and pipeline development.

Opdivo Fails to Meet Primary Endpoint in CheckMate-73L NSCLC Study

  • Bristol Myers Squibb's CheckMate-73L study, evaluating Opdivo plus Yervoy following chemoradiotherapy, did not meet its primary endpoint of progression-free survival in unresectable stage III NSCLC.
  • The phase III trial compared Opdivo-based regimens to chemoradiotherapy followed by AstraZeneca’s Imfinzi in 925 patients with locally advanced non-small cell lung cancer.
  • No new safety signals were identified, with adverse events consistent with the known profiles of Opdivo and Yervoy, according to Bristol Myers Squibb.
  • Bristol Myers is conducting a thorough evaluation of the CheckMate-73L data, with plans to present the findings at an upcoming medical conference.

Efficacy and Safety of Piwei Peiyuan Prescription in Treating Chronic Atrophic Gastritis

A clinical trial demonstrates the effectiveness and safety of Piwei Peiyuan Prescription, a Traditional Chinese Medicine, in treating Chronic Atrophic Gastritis (CAG), a precancerous condition of the stomach. The study highlights the prescription's ability to improve symptoms, enhance gastrointestinal motility, and promote mucosal repair, with minimal adverse reactions.

Recursion Anticipates Phase 2 Data Readouts and Advances Platform Capabilities

  • Recursion expects to release Phase 2 data for REC-994 in Cerebral Cavernous Malformation in Q3 2024, with the SYCAMORE trial fully enrolled.
  • Preliminary Phase 2 data for REC-2282 in Neurofibromatosis Type 2 is anticipated in Q4 2024, with adult enrollment expected to complete in Q2 2024.
  • The company's BioHive-2 supercomputer nears completion, enhancing capabilities in constructing foundation models for biology and chemistry.
  • A multi-year agreement with Helix provides access to extensive genomic and health record data for AI model training and patient stratification.

DLL3-Guided Therapies Show Promise in Small-Cell Lung Cancer Treatment

Recent advancements in DLL3-targeted therapies, including antibody-drug conjugates, bispecific T-cell engagers, and chimeric antigen receptor T-cell therapies, are offering new hope for patients with small-cell lung cancer (SCLC). Despite challenges, these therapies have demonstrated potential in improving clinical outcomes, with ongoing clinical trials exploring their efficacy and safety.

Highlighted Clinical Trials:

NCT04471727RecruitingPhase 1
Harpoon Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Posted 12/14/2020
NCT05507593Unknown StatusPhase 1
Tianjin Medical University Cancer Institute and Hospital
Posted 9/1/2022

FDA Finalizes Guidance on Clinical Pharmacology Considerations for Antibody-Drug Conjugates

  • The FDA has finalized its guidance on clinical pharmacology considerations for antibody-drug conjugates (ADCs), providing a framework for drug developers focusing on the cytotoxic payload.
  • The guidance emphasizes bioanalytical methods, dosing strategies, exposure-response analysis, intrinsic patient factors, QTc assessments, immunogenicity, and drug-drug interactions (DDIs).
  • Key areas of interest include measuring ADC constituents and metabolites, conducting exposure-response analyses, and assessing intrinsic factors like renal or liver impairment.
  • The guidance also addresses QTc assessment for cardiac safety, drug-drug interaction risk, and dosing strategies to maximize the therapeutic index of ADCs.
© Copyright 2025. All Rights Reserved by MedPath