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Clinical Trial News

Acadia Pharmaceuticals Presents Real-World Data on DAYBUE™ (trofinetide) for Rett Syndrome

Acadia Pharmaceuticals Inc. announced interim data from the LOTUS™ study, evaluating the efficacy and tolerability of DAYBUE™ (trofinetide) in Rett syndrome patients, at the 2024 International Rett Syndrome Foundation Annual Scientific Meeting. The study highlights improvements in symptoms and caregiver strategies for managing gastrointestinal tolerability.

Cerevel Therapeutics Advances Emraclidine Development for Alzheimer's-Related Dementia and Psychosis

  • Cerevel Therapeutics is developing Emraclidine (CVL-231), a novel oral M4 muscarinic receptor positive allosteric modulator, for treating dementia and psychosis in Alzheimer's disease.
  • The drug candidate represents a potential breakthrough in addressing neuropsychiatric symptoms of Alzheimer's disease, targeting the muscarinic acetylcholine receptor M4 through a unique mechanism of action.
  • As a subsidiary of AbbVie Inc., Cerevel is expanding its neuroscience portfolio, with Emraclidine development programs spanning multiple neurological conditions including schizophrenia and Alzheimer's-related disorders.

Zanubrutinib Combination Therapies Show Promise in High-Risk CLL/SLL Treatment

  • Zanubrutinib combined with venetoclax achieved 100% overall response rate and 48% complete response rate in treatment-naïve high-risk CLL/SLL patients with del(17p) and/or TP53 mutations in the SEQUOIA phase 3 trial.
  • The combination of sonrotoclax and zanubrutinib demonstrated 97% overall response rate with 57% complete response rate across all doses in relapsed/refractory CLL/SLL patients, with 85% achieving undetectable minimal residual disease.
  • Both combination therapies showed manageable safety profiles with no new safety concerns identified, positioning zanubrutinib as a potential backbone therapy for high-risk CLL/SLL treatment.

A Study Comparing Zanubrutinib With Bendamustine Plus Rituximab in Participants With Previously Untreated CLL or SLL

NCT03336333Active, Not RecruitingPhase 3
BeiGene
Posted 10/31/2017

Study of Bcl-2 Inhibitor Sonrotoclax (BGB-11417) in Participants With Mature B-Cell Malignancies

NCT04277637Active, Not RecruitingPhase 1
BeiGene
Posted 3/24/2020

FDA Approves Merck's CAPVAXIVE: First 21-Serotype Pneumococcal Vaccine Designed Specifically for Adults

FDA Approval
  • The FDA has approved Merck's CAPVAXIVE, a novel 21-valent pneumococcal conjugate vaccine specifically designed to protect adults against serotypes causing the majority of invasive pneumococcal disease.
  • CAPVAXIVE includes eight unique serotypes not covered by other approved pneumococcal vaccines, targeting approximately 84% of invasive pneumococcal disease cases in adults over 50, compared to 52% covered by PCV20.
  • The single-dose vaccine demonstrated robust immunogenicity across various adult populations, with the CDC's Advisory Committee on Immunization Practices expected to make recommendations for its use later this month.

Safety and Immunogenicity of V116 in Pneumococcal Vaccine-naïve Adults (V116-003, STRIDE-3)

NCT05425732CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 7/13/2022

A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V116 When Administered Concomitantly With Influenza Vaccine in Adults 50 Years of Age or Older (V116-005, STRIDE-5)

NCT05526716CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 9/23/2022

A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V116 in Pneumococcal Vaccine-experienced Adults (V116-006, STRIDE-6)

NCT05420961CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 7/12/2022

Regeneron's Lynozyfic Receives FDA Approval for Multiple Myeloma with 70% Response Rate

Drug Approval
  • Regeneron's Lynozyfic (linvoseltamab-gcpt) received accelerated FDA approval for relapsed or refractory multiple myeloma patients with at least four prior treatments.
  • The first-in-class BCMAxCD3 bispecific antibody demonstrated a 70% overall response rate with 45% achieving complete response or better in the pivotal LINKER-MM1 trial.
  • The therapy features a flexible maintenance schedule that can extend to every four weeks based on patient response, potentially reducing treatment burden.
  • The approval includes a Boxed Warning for cytokine release syndrome and neurological toxicities, with availability through a Risk Evaluation and Mitigation Strategy program.

Phase 1/2 Study of REGN5458 in Adult Patients With Relapsed or Refractory Multiple Myeloma

NCT03761108RecruitingPhase 1
Regeneron Pharmaceuticals
Posted 1/23/2019

Merck's Keytruda Advances Development Program for Hepatocellular Carcinoma Treatment

  • Pembrolizumab (Keytruda) is being evaluated for both first-line and second-line treatment of hepatocellular carcinoma, expanding its potential role in liver cancer therapy.
  • The drug, already approved for multiple cancer indications, has demonstrated significant clinical potential in HCC patients previously treated with sorafenib.
  • Merck's development program for Keytruda in HCC represents a major advancement in immunotherapy options for liver cancer patients, addressing a significant unmet medical need.

PharmaMar's Trabectedin Expands Development Portfolio with Multiple Cancer Indications

  • Trabectedin (Yondelis), a marine-derived antitumor agent, is currently approved for advanced soft tissue sarcoma and relapsed platinum-sensitive ovarian cancer in combination therapy.
  • The drug is undergoing development for multiple new indications including mesenchymal chondrosarcoma, recurrent meningioma, and various gynecological cancers, expanding its therapeutic potential.
  • PharmaMar continues to investigate Trabectedin's applications, building on its success in treating liposarcoma and leiomyosarcoma patients who have received prior anthracycline-containing regimens.

Roche's Bevacizumab Under Evaluation for Endometrial Cancer Treatment

  • F. Hoffmann-La Roche is advancing bevacizumab as a potential therapeutic option for endometrial cancer, expanding the drug's application in gynecologic oncology.
  • The development program leverages Roche's extensive experience in biotechnology and oncology, with the company tracking phase transition and likelihood of approval metrics based on 18 years of historical data.
  • The assessment of bevacizumab for endometrial cancer considers multiple factors including drug attributes, company capabilities, and clinical trial design to determine potential approval success.

argenx Outlines 'Vision 2030' with Focus on Autoimmune Disease Therapies

  • argenx will present its 'Vision 2030' at its R&D Day, focusing on expanding its reach to 50,000 patients with severe autoimmune diseases.
  • The company plans to broaden its leadership in myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP) with VYVGART and empasiprubart.
  • Phase 2 data in Sjogren’s disease and multifocal motor neuropathy (MMN) support the advancement of efgartigimod and empasiprubart to Phase 3 development.
  • argenx has decided not to advance efgartigimod in PC-POTS after Phase 2 results showed no clinically meaningful improvement compared to placebo.

CAR-T Cell Therapy Shows Promising Results in Lupus and Autoimmune Diseases at EULAR Conference

  • Early clinical trial data presented at EULAR demonstrates CAR-T cell therapy's potential to induce remission in lupus and other autoimmune conditions, with some patients maintaining benefits for up to three years.
  • Multiple companies including Kyverna Therapeutics, Cabaletta Bio, and Novartis reported encouraging safety profiles and positive patient responses, though some participants still require maintenance medications.
  • While showing promise, experts emphasize the need for more extensive testing, particularly in diverse populations, and acknowledge challenges including manufacturing complexity and cost considerations.

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