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Tenosynovial Giant Cell Tumors Treatment Market to See Significant Growth by 2034

The Tenosynovial Giant Cell Tumors (TSGCTs) treatment market, valued at approximately USD 300 million in 2023, is expected to grow significantly by 2034. This growth is driven by the increasing prevalence of TSGCTs, advancements in treatment options, and the introduction of novel therapies. Key developments include SynOx Therapeutics securing USD 75 million in Series B funding for the development of emactuzumab, a promising treatment for TSGCTs.

Global Clinical Trials Landscape Reveals Promising Advances in Non-Small Cell Lung Cancer Treatment

  • Non-small cell lung cancer affects approximately 2.1 million people globally, with the Asia-Pacific region showing the highest incidence and leading clinical trial activities.
  • Current treatment landscape includes targeted therapies like Lumakras and Tagrisso, with promising Phase III pipeline drugs such as KN046 and ivonescimab advancing personalized medicine approaches.
  • Clinical outcomes vary significantly by stage, with early-stage diagnosis showing 45-76% five-year recurrence rates, while advanced stages face more challenging survival rates of 35% for locally advanced and 7% for metastatic cases.

More Men with Advanced Prostate Cancer Could Benefit from Precision Drug Olaparib

Scientists have discovered that a genetic aberration in some prostate cancers could allow more men to be successfully treated with the targeted drug olaparib. This finding could expand the use of PARP inhibitors, like olaparib, to a broader group of men with advanced prostate cancer, potentially improving survival and quality of life.

Annamycin Plus Cytarabine Shows Promise in AML Treatment: FDA Review Complete

  • Moleculin Biotech completed an end-of-phase 2 meeting with the FDA for annamycin combined with cytarabine in acute myeloid leukemia (AML).
  • A phase 1b/2 trial showed a 45% composite complete remission rate in efficacy-evaluable AML patients treated with the combination.
  • In second-line AML patients, the combination therapy achieved a 60% composite complete remission rate, indicating enhanced efficacy.
  • Annamycin plus cytarabine was well-tolerated, with myelosuppression and infections being the most common adverse effects observed.

Study of Liposomal Annamycin in Combination with Cytarabine for the Treatment of Subjects with Acute Myeloid Leukemia (AML)

NCT05319587Active, Not RecruitingPhase 1
Moleculin Biotech, Inc.
Posted 9/29/2022

Eton Pharmaceuticals' ET-400 (Hydrocortisone Oral Solution) Receives FDA Acceptance

  • Eton Pharmaceuticals' New Drug Application (NDA) for ET-400, an oral hydrocortisone solution, has been accepted by the FDA.
  • The FDA has assigned a PDUFA target action date of February 28, 2025, for ET-400, a treatment for adrenocortical insufficiency.
  • ET-400 is a room-temperature stable, patented formulation, potentially capturing a significant portion of the oral hydrocortisone market.
  • Eton anticipates combined peak sales of over $50 million annually for ET-400 and ALKINDI SPRINKLE® upon approval.

KYV-101 Receives FDA Fast Track Designation for Stiff Person Syndrome Treatment

  • Kyverna Therapeutics' KYV-101, a CAR-T cell therapy, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for stiff person syndrome.
  • The RMAT designation will facilitate closer collaboration with the FDA to accelerate the development, review, and potential approval of KYV-101.
  • The FDA's decision was based on promising early clinical trial results, indicating KYV-101's potential to address the unmet needs in treating stiff person syndrome.
  • KYV-101 aims to "reset" the immune system in autoimmune diseases and is currently in Phase 2 trials for stiff person syndrome, multiple sclerosis, and myasthenia gravis.

Maralixibat Demonstrates Efficacy in Treating Pruritus and Reducing Bile Acids in PFIC Patients

  • A Phase 3 trial (MARCH-PFIC) reveals that maralixibat significantly improves pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
  • The study showed a significant reduction in serum bile acid concentrations in PFIC patients treated with maralixibat compared to placebo.
  • Maralixibat represents a potential non-surgical treatment option for managing PFIC by interrupting enterohepatic circulation.
  • The most common adverse event associated with maralixibat was mild to moderate, transient diarrhea.

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

NCT03905330CompletedPhase 3
Mirum Pharmaceuticals, Inc.
Posted 7/9/2019

Eli Lilly's Kisunla Receives FDA Approval for Alzheimer's Treatment

  • Eli Lilly's donanemab, branded as Kisunla, has been granted FDA approval for treating Alzheimer's disease, offering a new therapeutic option.
  • The market had largely anticipated the approval, which explains the muted stock response following the FDA's decision.
  • Eli Lilly's stock performance is currently driven more by its GLP-1 treatments for weight loss and diabetes, like Zepbound and Mounjaro, than by Alzheimer's drug approvals.
  • Kisunla is projected to generate substantial revenue, potentially reaching $5 billion annually, contributing to Eli Lilly's revenue diversification.

Novel Approaches Reshape Rare Disease Drug Development: Experts Outline Path to Success

  • Experts highlight the growing acceptance of non-interventional and observational studies in rare disease research, with regulators showing increased flexibility for orphan drug development pathways.
  • Quality of life endpoints and innovative trial designs are gaining traction, with FDA's Complex Innovative Trial Design program and EMA's reflection paper supporting novel methodological approaches.
  • Patient-centric strategies, including telemedicine integration and early consideration of access pathways, are becoming crucial for successful rare disease therapy development.
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