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Clinical Trial News

FDA Approvals for Breast Cancer Drugs: Clinical Benefits, Development Time, and Pricing Analysis

• The FDA approved 26 unique cancer drugs for breast cancer across 42 indications between 2000 and 2023, with a median development time of 7.8 years. • A meta-analysis of RCTs showed new breast cancer indications significantly reduced the likelihood of death (HR 0.78) and improved median OS by 2.8 months. • There was a weak correlation between OS and PFS, suggesting that improvements in PFS may not always translate to better OS or QoL. • Drug prices averaged $16,013 per month in 2023, with no significant correlation between price and clinical benefits like OS or QoL improvements.

LUNAR Trial Update: Tisotumab Vedotin Shows Promise in Advanced NSCLC

  • Updated analysis of the Phase 3 LUNAR trial reinforces tisotumab vedotin's potential as a treatment for advanced non-small cell lung cancer (NSCLC).
  • The study highlights clinically meaningful improvements in overall survival and progression-free survival in previously treated NSCLC patients.
  • Researchers presented these findings at the IASLC 2024 World Conference on Lung Cancer, sparking discussions about integrating tisotumab vedotin into treatment paradigms.

India Introduces New Regulations for Clinical Research Organizations

  • The Indian government has established Standard Operating Procedures (SOPs) for Clinical Research Organizations (CROs) to ensure the safety and quality of clinical trials.
  • The new regulations, under the New Drugs and Clinical Trials (Amendment) Rules, 2024, define the roles, duties, and liabilities of CROs, effective April 1, 2025.
  • CROs must now apply to the Central Licensing Authority for registration to conduct clinical trials or bioavailability/bioequivalence studies, enhancing transparency and oversight.
  • The government aims to improve product quality, expedite clinical trials for new medications and vaccines, and create a repository of organizations involved in clinical research.

Donanemab Approved in US for Early Alzheimer's Treatment, UK Approval Uncertain

• The FDA approved donanemab in July 2024 for early symptomatic Alzheimer's, including mild cognitive impairment and mild dementia. • Clinical trials showed donanemab slowed cognitive decline by approximately 22% compared to placebo over 18 months. • UK approval is uncertain, with NICE potentially rejecting it due to high costs relative to modest benefits, following a similar decision on lecanemab. • Donanemab, developed by Eli Lilly, awaits UK approval, but sources suggest NICE may reject it due to cost concerns.

Glofitamab-gxbm (Columvi) Approved for Relapsed or Refractory Diffuse Large B-Cell Lymphoma

  • Glofitamab-gxbm, a CD20xCD3 T-cell engaging bispecific antibody, is approved for relapsed or refractory diffuse large B-cell lymphoma (LBCL) after two or more lines of systemic therapy.
  • The accelerated approval was based on a phase 1/2 study showing a 56% response rate and a median duration of response of 18.4 months in treated patients.
  • Glofitamab-gxbm is administered intravenously over a fixed duration, with premedication to reduce the risk of cytokine release syndrome (CRS) and infusion-related reactions.
  • Adverse events include CRS and Immune effector cell–associated neurotoxicity syndrome (ICANS), requiring careful monitoring and management in equipped facilities.

A Dose Escalation Study of Glofitamab (RO7082859) as a Single Agent and in Combination With Obinutuzumab, Administered After a Fixed, Single Pre-treatment Dose of Obinutuzumab in Participants With Relapsed/Refractory B-cell Non-hodgkin's Lymphoma

NCT03075696RecruitingPhase 1
Hoffmann-La Roche
Posted 2/21/2017

Wegovy Shows Promise in Treating Hidradenitis Suppurativa

  • A study at St Vincent’s University Hospital found that Wegovy (semaglutide) reduced flare-up frequency in hidradenitis suppurativa (HS) patients with obesity.
  • Patients receiving Wegovy experienced a reduction in flare-up frequency from once every 8.5 weeks to once every 12 weeks, alongside improved quality of life.
  • The study also noted weight loss among patients, with average BMI decreasing from 43.1 to 41.5 and a third of patients losing 10kg or more.
  • Lead researcher Dr. Daniel Lyons suggests semaglutide, even at modest doses, offers substantial benefits in managing HS, warranting larger randomized controlled trials.

Cinrebafusp Alfa Shows Promise in HER2-Expressing Solid Tumors: A Phase I Study

• Cinrebafusp alfa, a HER2/4-1BB bispecific antibody, demonstrates manageable safety and encouraging anti-tumor activity in patients with HER2-expressing solid tumors. • The Phase I trial established a recommended Phase II dose (RP2D) of 10 mg/kg every two weeks, showing dose-dependent increases in T-cell activation and proliferation. • Clinical responses, including confirmed partial responses and stable disease, were observed across various HER2-expressing cancers, suggesting potential therapeutic benefit. • This first-in-human study supports further investigation of cinrebafusp alfa in HER2-positive solid tumors, with ongoing trials exploring its efficacy in specific cancer subtypes.

Acasti Pharma's GTX-104 Trial for aSAH Completes Enrollment Early, Eyes 2026 Launch

  • Acasti Pharma's STRIVE-ON trial, evaluating GTX-104 for aneurysmal subarachnoid hemorrhage (aSAH), has completed enrollment ahead of schedule with approximately 100 participants.
  • Top-line data from the pivotal safety trial is expected by February, potentially leading to a New Drug Application (NDA) filing by June 2025.
  • H.C. Wainwright maintains a Buy rating for Acasti Pharma, projecting peak sales for GTX-104 to exceed $130 million, with potential FDA approval by April 2026.
  • GTX-104 is poised to become the new standard of care for aSAH in the ICU, given its product profile and previous data.

Longeveron to Explore Partnerships for Alzheimer's Cell Therapy at Cell & Gene Meeting

  • Longeveron will attend the Cell & Gene Meeting on the Mesa to seek partnerships for its Alzheimer's cell therapy program.
  • The company's Lomecel-B™ showed promising Phase 2a results, slowing disease worsening in mild Alzheimer's patients.
  • Lomecel-B™ has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA.
  • Longeveron also highlights its contract development and manufacturing capabilities at its cGMP facility.

Metabolic Reprogramming of T Cells Enhances Checkpoint Inhibitor Therapy

  • Researchers found that activating the pentose phosphate pathway in T cells enhances their anti-tumor activity when combined with checkpoint inhibitors.
  • Blocking the PKM2 enzyme boosts less mature T cells, providing a sustained supply of cytotoxic CD8+ T cells to attack tumors.
  • This metabolic reprogramming strategy significantly improved tumor control in animal models of lung cancer and melanoma.
  • The approach may enhance cell-transfer therapies like CAR-T by directly manipulating T cells in the lab to minimize off-target effects.

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