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Key Developments in Biosimilars and Pharmaceuticals in 2024

2024 has seen significant advancements and approvals in the biosimilars and pharmaceutical sectors, including the launch of Wegovy® in Australia, new indications for Opdivo®, and approvals for biosimilars in Canada and Europe. Additionally, there have been notable developments in clinical trials and legal disputes over patents.

Brazil Emerges as Key Hub for Cancer Drug Clinical Trials

  • Brazil is increasingly becoming a prime location for oncology drug clinical trials due to its growing R&D capabilities.
  • Multinational pharmaceutical companies like Roche, Pfizer, and AstraZeneca are significantly contributing to Brazil's clinical trial landscape.
  • Phase III trials dominate the Brazilian clinical trial scene, indicating the country's importance in late-stage drug development.
  • Contract Research Organizations (CROs) play a crucial role in supporting and facilitating complex oncology trials in Brazil.

FDA Extends Review of Neurotech's NT-501 for Macular Telangiectasia Type 2

  • The FDA has extended the PDUFA goal date for Neurotech's NT-501 BLA by three months, now set for March 18, 2025, to allow for review of additional data.
  • NT-501 (revakinagene taroretcel) is under review for treating macular telangiectasia type 2 (MacTel), a rare neurodegenerative disease causing gradual vision loss.
  • Neurotech's NT-501 utilizes the ECT platform to deliver CNTF, aiming for long-term, sustained therapeutic protein delivery to treat chronic retinal diseases like MacTel.
  • The FDA had previously granted priority review for NT-501 in June, underscoring the urgency and unmet need in treating MacTel.

Sarclisa Demonstrates Improved Progression-Free Survival in Newly Diagnosed Multiple Myeloma

  • Sarclisa, combined with lenalidomide, bortezomib, and dexamethasone (RVd), significantly improved progression-free survival in transplant-eligible, newly diagnosed multiple myeloma patients.
  • The GMMG-HD7 phase 3 trial showed a statistically significant reduction in disease progression or death with the Sarclisa-RVd induction therapy.
  • These results support Sarclisa's potential as a backbone therapy in multiple myeloma, particularly in frontline settings, potentially becoming a new standard-of-care induction regimen.
  • Sanofi is pursuing further investigations of Sarclisa across the multiple myeloma treatment continuum to improve long-term outcomes for patients.

Trial on the Effect of Isatuximab to Lenalidomide/Bortezomib/Dexamethasone (RVd) Induction and Lenalidomide Maintenance in Patients With Newly Diagnosed Myeloma (GMMG HD7)

NCT03617731Active, Not RecruitingPhase 3
University of Heidelberg Medical Center
Posted 10/18/2018

A Study to Investigate the Clinical Benefit of Isatuximab in Combination With Bortezomib, Lenalidomide and Dexamethasone in Adults With Newly Diagnosed Multiple Myeloma Not Eligible for Transplant

NCT03319667Active, Not RecruitingPhase 3
Sanofi
Posted 12/7/2017

FDA Rejects Lykos Therapeutics' Midomafetamine for PTSD, Demands Additional Phase 3 Trial

  • The FDA issued a complete response letter for Lykos Therapeutics' NDA for midomafetamine capsules, intended to treat PTSD in adults, citing insufficient data.
  • Lykos plans to request a meeting with the FDA to discuss the agency's recommendations and seek reconsideration of the decision regarding midomafetamine.
  • The FDA's concerns echo those raised during an advisory committee meeting, including questions about data durability and expectancy bias from prior MDMA use.
  • Lykos maintains that existing data sufficiently demonstrate efficacy and durability, aligning with FDA guidance, and expresses disappointment for PTSD sufferers awaiting new treatments.

Invenio Imaging's AI-Based Image Analysis Module Enrolls First Patients in Pivotal Lung Cancer Study

  • Invenio Imaging initiated a pivotal US study (ON-SITE) to evaluate its AI-based image analysis module for rapid lung cancer detection during bronchoscopy.
  • The study combines Stimulated Raman Histology with AI, aiming to assist physicians in detecting cancer in lung biopsies where rapid-on-site tissue evaluation (ROSE) is unavailable.
  • The ON-SITE study is being conducted at multiple centers, including The University of Texas MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center.
  • The NIO Laser Imaging System allows rapid imaging of fresh tissue biopsies without staining, with the AI module intended to improve outcomes and personalize treatments.

ARS Pharma's Neffy Aims to Expand Pediatric Access for Type 1 Allergic Reactions

  • ARS Pharmaceuticals seeks FDA approval for Neffy 1 mg to treat type 1 allergic reactions, including anaphylaxis, in children weighing 33-66 lbs..
  • Neffy 1 mg offers a needle-free epinephrine delivery option, potentially reducing fear and accidental needle sticks in young patients.
  • Clinical trials showed adults could easily administer Neffy, and untrained children as young as 10 could self-administer, highlighting its ease of use.
  • Neffy's compact size and temperature stability offer convenience for families managing severe allergies, enhancing accessibility and adherence.

FDA Rejects MDMA for PTSD Treatment, Raising Questions for Psychedelic Therapies

  • The FDA has declined to approve MDMA, also known as ecstasy, as a treatment for post-traumatic stress disorder (PTSD), requesting an additional late-stage study.
  • Lykos Therapeutics, the company seeking approval, expressed disappointment and plans to request a reconsideration of the FDA's decision.
  • The FDA cited significant limitations in the application data, preventing the conclusion that MDMA is safe and effective for the proposed indication.
  • The decision casts uncertainty on the future of other psychedelic therapies in clinical trials, though some experts believe other substances may fare better.

FDA Approves Lymphir (denileukin diftitox-cxdl) for Relapsed/Refractory Cutaneous T-Cell Lymphoma

  • The FDA has approved Lymphir (denileukin diftitox-cxdl) for relapsed or refractory cutaneous T-cell lymphoma (CTCL) in adults after at least one prior systemic therapy.
  • Lymphir targets the interleukin-2 (IL-2) receptor on malignant T-cells and Tregs, offering a novel mechanism of action in CTCL treatment.
  • Clinical trial data showed an objective response rate of 36.2% in patients with Stage I-III CTCL, with a median time to response of 1.4 months.
  • Lymphir's approval provides a new treatment option for CTCL patients, aiming to reduce skin disease and control itching without cumulative toxicity.

A Trial of E7777 in Persistent and Recurrent Cutaneous T-Cell Lymphoma

NCT01871727CompletedPhase 3
Eisai Inc.
Posted 5/30/2013

WestGene's mRNA Cancer Vaccine WGc-043 Receives Dual IND Approvals for EBV-Related Cancers

  • WestGene's WGc-043, an mRNA therapeutic cancer vaccine, has received IND approvals from both China's NMPA and the US FDA, marking a global first for EBV-related cancers.
  • WGc-043 leverages AI-assisted antigen screening and a novel LNP delivery system to activate anti-tumor immunity, potentially offering efficacy comparable to CAR-T and monoclonal antibody therapies.
  • The vaccine has shown superior safety and efficacy in IITs for nasopharyngeal carcinoma and natural killer T-cell lymphoma compared to existing mRNA cancer vaccines.
  • Approved for EBV-positive solid tumors and hematologic malignancies, WGc-043 represents a significant advancement in mRNA immunotherapy, targeting a virus infecting over 90% of the global population.

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