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Clinical Trial News

FDA Nears Decisions on Vanda, Zevra, Heron, Merck, BMS, Sanofi/Regeneron

  • Vanda Pharmaceuticals awaits FDA decision on tradipitant for gastroparesis, a condition affecting millions in the U.S., with a verdict expected by September 18.
  • Zevra Therapeutics anticipates potential approval of arimoclomol for Niemann-Pick disease type C (NPC) by September 21, following positive advisory committee support.
  • The FDA is set to decide on Heron Therapeutics' extended-release needle for Zynrelef by September 23, designed to simplify drug preparation and administration.
  • Merck seeks approval for Keytruda in pleural mesothelioma, with a decision due September 25, based on Phase II/III KEYNOTE-483 trial data showing improved survival.
  • Bristol Myers Squibb awaits FDA decision on KarXT for schizophrenia by September 26, potentially offering a novel mechanism of action targeting muscarinic receptors.
  • Sanofi and Regeneron anticipate a decision on Dupixent for COPD by September 27, supported by Phase III BOREAS and NOTUS trials demonstrating reduced exacerbations.

mRNA Vaccine Pipeline Heats Up: Late-Stage Candidates Target Flu, COVID, CMV, and Cancer

  • Pfizer and BioNTech are advancing an mRNA influenza vaccine, PF-07252220, demonstrating superiority over existing flu vaccines in Phase III trials.
  • Moderna's mRNA-1083, a combination COVID-19/influenza vaccine, showed strong immune responses in Phase III, potentially extending the sales of COVID-19 vaccines.
  • GSK acquired rights to CureVac's mRNA vaccines against COVID-19 (GSK4388067) and influenza (GSK4382276), both currently in Phase II trials.
  • Moderna's mRNA-4157, a therapeutic cancer vaccine, is being explored in Phase III in combination with Merck’s Keytruda, targeting head and neck cancer and melanoma.

FDA Nears Decisions on Key Therapies for Gastroparesis, Niemann-Pick Disease, Schizophrenia, COPD, Mesothelioma, and Post-Surgical Pain

  • The FDA is expected to decide on Vanda Pharmaceuticals' tradipitant for gastroparesis by September 18, potentially offering a novel NK-1R antagonist treatment option.
  • Zevra Therapeutics awaits a verdict by September 21 on arimoclomol for Niemann-Pick disease type C, an ultrarare neurodegenerative disorder with no approved therapies.
  • A decision is anticipated by September 26 on Bristol Myers Squibb's KarXT for schizophrenia, representing a new pharmacological approach targeting muscarinic receptors.

Dimerix Partners with UK National Registry to Accelerate ACTION3 Trial for FSGS

  • Dimerix collaborates with the UK's National Registry of Rare Kidney Diseases (RaDaR) to enhance patient recruitment for the ACTION3 Phase 3 trial.
  • RaDaR, the largest kidney registry globally, will identify and contact suitable FSGS patients in the UK for potential trial participation.
  • Dimerix has also received approval for its Investigational New Drug Application from the Thai Food and Drug Administration, paving the way for trial initiation in Thailand.

LuPSMA Therapy Shows Promise in Earlier-Stage Prostate Cancer

  • A Peter Mac-led study reveals that Lutetium-177 PSMA-617 (LuPSMA) therapy significantly improves outcomes for patients with newly diagnosed metastatic prostate cancer.
  • The UpFrontPSMA phase 2 trial demonstrated that adding LuPSMA to standard androgen deprivation therapy and chemotherapy led to higher rates of undetectable PSA levels.
  • 41% of patients receiving LuPSMA achieved undetectable PSA levels at 48 weeks, compared to only 16% with standard care, indicating a substantial improvement.
  • A larger Phase III clinical trial is underway to validate these findings and potentially change clinical practice for treating earlier-stage prostate cancer.

LuPSMA Therapy Shows Promise in Earlier Stages of Prostate Cancer

• A Peter Mac-led study reveals that Lutetium-177 PSMA-617 (LuPSMA) therapy significantly improves outcomes for patients with newly diagnosed prostate cancer when combined with standard care. • The UpFrontPSMA phase 2 trial demonstrated that adding LuPSMA to androgen deprivation therapy and chemotherapy led to undetectable PSA levels in 41% of patients, compared to 16% with standard care alone. • The trial's results, published in Lancet Oncology and presented at ESMO Congress 2024, suggest LuPSMA could be a game-changer in earlier-stage prostate cancer treatment, pending confirmation in larger phase 3 trials.

FDA Modernization Act 3.0 Introduced to Expedite Non-Animal Drug Testing

  • A bipartisan group of senators introduced the FDA Modernization Act 3.0 to accelerate the implementation of non-animal drug testing methods.
  • The bill mandates the FDA to update its regulations within six months to align with the FDA Modernization Act 2.0, passed in 2022.
  • FDA Modernization Act 2.0 allowed drug developers to use advanced methods like cell-based assays and computer modeling instead of animal testing.
  • The act aims to reduce unnecessary animal testing, improve the efficiency of drug development, and provide faster, more reliable treatments for patients.

Trastuzumab Deruxtecan Preserves Quality of Life and Delays Pain in HR+/HER2-Low/Ultralow Metastatic Breast Cancer

  • Trastuzumab deruxtecan (T-DXd) maintains global health and quality of life (QOL) in patients with hormone receptor–positive (HR+), HER2-low or -ultralow metastatic breast cancer (MBC).
  • T-DXd significantly reduces the risk of clinically meaningful deterioration in pain compared to treatment of physician’s choice (TPC) in MBC patients.
  • DESTINY-Breast06 trial data showed T-DXd delayed pain deterioration by 15.7 months compared to TPC, highlighting its potential as a new therapeutic option.
  • Gastrointestinal adverse events with T-DXd underscore the importance of antiemetic prophylaxis, though they do not negate overall QOL preservation.

Study of Trastuzumab Deruxtecan (T-DXd) vs Investigator's Choice Chemotherapy in HER2-low, Hormone Receptor Positive, Metastatic Breast Cancer

NCT04494425Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/24/2020

Trastuzumab Deruxtecan (DS-8201a) Versus Investigator's Choice for HER2-low Breast Cancer That Has Spread or Cannot be Surgically Removed [DESTINY-Breast04]

NCT03734029Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/27/2018

Digital Support App Launches in UK to Address Rising Youth Cancer Cases

  • Cancer rates among people under 24 have surged 22% since early 2000s, prompting the Ella Dawson Foundation and Careology to launch a specialized digital support app for young cancer patients.
  • The app offers comprehensive features including symptom monitoring, medication tracking, and wellbeing support, with content from leading organizations like Macmillan Cancer Support and Cancer Research UK.
  • Initially rolling out at London's Royal Marsden Hospital, the platform addresses the unique challenges faced by young cancer patients while enabling clinicians to monitor patient progress remotely.

Anlotinib and Penpulimab Combination Shows Promise in First-Line HCC Treatment

  • The Phase III APOLLO trial evaluated anlotinib plus penpulimab versus sorafenib in advanced hepatocellular carcinoma (aHCC).
  • The combination therapy significantly improved progression-free survival (PFS) to 6.9 months compared to 2.8 months with sorafenib.
  • Overall survival (OS) also saw improvement in the anlotinib plus penpulimab arm, with 16.5 months versus 13.2 months for sorafenib.
  • The safety profile of the combination was manageable, exhibiting fewer treatment-related adverse events than sorafenib.

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