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Tyra Biosciences Initiates Phase 1/2 Trial of TYRA-300 in Metastatic Urothelial Carcinoma

  • Tyra Biosciences has commenced its SURF301 Phase 1/2 clinical trial, dosing the first patient with TYRA-300, an oral FGFR3-selective inhibitor.
  • The SURF301 study will evaluate TYRA-300's safety, tolerability, pharmacokinetics, and preliminary efficacy in patients with metastatic urothelial carcinoma.
  • TYRA-300 represents a novel, purpose-built therapy designed to overcome tumor resistance and improve outcomes in cancer patients with FGFR3 alterations.
  • The initiation of the SURF301 trial marks a significant milestone for Tyra Biosciences in advancing precision oncology treatments for urothelial carcinoma.

Vortioxetine Shows Promise in Glioblastoma Treatment by Targeting Tumor-Specific Vulnerabilities

  • A high-throughput drug screening identified vortioxetine, a neuroactive drug, as a potential treatment for glioblastoma, demonstrating anti-cancer activity in patient-derived cells.
  • Ex vivo drug testing revealed that vortioxetine selectively targets glioblastoma cells, reducing their viability while sparing other cell types, indicating a tumor-specific mechanism.
  • Mechanistic studies showed vortioxetine modulates calcium signaling and downregulates key genes involved in glioblastoma proliferation and survival, such as BTG1 and BTG2.
  • In vivo experiments in mice confirmed that vortioxetine can inhibit glioblastoma tumor growth, suggesting its potential as a novel therapeutic agent for this aggressive brain cancer.

Trabectedin Plus Ipilimumab and Nivolumab Shows Promise in Advanced Soft Tissue Sarcoma

  • A combination of trabectedin, ipilimumab, and nivolumab demonstrates potential as a safe and effective first-line treatment for advanced soft tissue sarcoma.
  • The SAINT study's phase 2 results showed an overall response rate of 24.7% and a disease control rate of 82.5% in previously untreated patients.
  • Median progression-free survival was 7.3 months, and median overall survival reached 32.0 months, indicating a clinically meaningful benefit.
  • Further randomized studies are recommended to confirm the superiority of this regimen compared to standard first-line therapies.

SAINT:Trabectedin, Ipilimumab and Nivolumab for Previously Treated Advanced Soft Tissue Sarcoma

NCT03138161RecruitingPhase 1
Sarcoma Oncology Research Center, LLC
Posted 4/13/2017

Third Harmonic Bio Appoints Geoff McDonough to Board of Directors, Advancing THB335 for Chronic Spontaneous Urticaria

  • Third Harmonic Bio appointed Geoff McDonough, M.D., to its Board of Directors, enhancing its leadership with extensive clinical and commercial experience.
  • The appointment supports the advancement of THB335, Third Harmonic Bio's lead product candidate, into clinical trials for chronic spontaneous urticaria.
  • Geoff McDonough's expertise and cultural alignment are expected to significantly benefit Third Harmonic Bio's strategic and operational initiatives.

FDA Approves Zevra Therapeutics' Miplyffa for Niemann-Pick Disease Type C, Stock Surges

  • The FDA has approved Zevra Therapeutics' Miplyffa for the treatment of Niemann-Pick disease type C, a rare neurodegenerative disorder.
  • This regulatory milestone marks a significant advancement in the treatment landscape for this progressive disease, offering new hope to patients.
  • Following the announcement, Zevra Therapeutics Inc. (ZVRA) experienced a surge in its stock price, reaching a new 52-week high.
  • The approval underscores the potential of Miplyffa to address the unmet medical needs of individuals affected by Niemann-Pick disease type C.

NCPA Intensifies Campaign for PBM Reform as Congressional Deadline Looms

  • The National Community Pharmacists Association (NCPA) is leading a critical push for PBM reform legislation, focusing on Medicaid Managed Care and Medicare Part D payment reforms before year-end Congressional deadline.
  • Independent pharmacies face significant challenges with approximately one pharmacy closing daily nationwide, potentially creating pharmacy deserts in underserved communities and impacting patient access to care.
  • NCPA's "Finish the Fight" campaign has mobilized nearly 40,000 patients to contact Congress, demonstrating strong bipartisan support for PBM reform to ensure fair compensation and transparent drug pricing practices.

Rare Shingles Reactivation Reported Post-Shingrix Vaccination

  • A recent study highlights a rare case of herpes zoster ophthalmicus (HZO) reactivation following recombinant zoster vaccine (RZV) administration in a 59-year-old male.
  • The patient developed a rash and paresthesia nine days post-vaccination, requiring hospitalization and treatment with valacyclovir and mupirocin.
  • While RZV (Shingrix) is generally safe and effective, this case underscores the need for ongoing monitoring for potential adverse events and rare reactivation.
  • Further research is necessary to understand how RZV's immunomodulatory effects may, in rare instances, provoke herpes zoster reactivation.

Capivasertib Shows Promise in ER+/HER2- Metastatic Breast Cancer with PIK3CA Mutations

  • Capivasertib combined with fulvestrant demonstrates significant progression-free survival benefit in patients with AKT pathway-altered metastatic breast cancer, showing a median PFS of 7.3 months versus 3.1 months with placebo.
  • The phase 3 CAPItello-291 trial revealed capivasertib offers better tolerability compared to alpelisib, particularly regarding blood sugar management and rash occurrence in PIK3CA-mutated breast cancer.
  • Expert analysis suggests capivasertib as a preferred treatment option for PIK3CA-mutated breast cancer, with data showing an overall survival benefit with HR of 0.74 across all patients regardless of mutation status.

Capivasertib+Fulvestrant vs Placebo+Fulvestrant as Treatment for Locally Advanced (Inoperable) or Metastatic HR+/HER2- Breast Cancer

NCT04305496Active, Not RecruitingPhase 3
AstraZeneca
Posted 4/16/2020

Global Metabolic Disruption Discovered in SMA Patients: N-acetylneuraminic Acid Shows Treatment Potential

  • A case-control study reveals widespread metabolic disruptions in SMA patients, affecting nucleotide, lipid, carbohydrate, amino acid, and vitamin metabolism pathways.
  • Researchers identified 118 significantly altered metabolites in SMA patients' cerebrospinal fluid, with N-acetylneuraminic acid showing the most significant differentiation (P = .0000549).
  • The findings suggest N-acetylneuraminic acid could serve as a potential therapeutic agent for improving functional outcomes in SMA patients.

Apellis Faces Setback as EMA Rejects Pegcetacoplan, Mizuho Lowers Price Target

  • Apellis Pharmaceuticals faces European market challenges as the EMA rejects pegcetacoplan (Syfovre) for regulatory approval, impacting potential revenue streams.
  • Mizuho Securities reduces Apellis' price target to $39, maintaining a Neutral rating due to the EMA's negative opinion and limited growth potential.
  • Despite the European setback, Apellis reports strong Q2 2024 growth, driven by robust sales of SYFOVRE and EMPAVELI in the United States.
  • Phase 3 VALIANT study shows Empaveli (pegcetacoplan) significantly reduces proteinuria in patients with rare kidney diseases, demonstrating potential beyond ophthalmology.

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