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Clinical Trial News

Cabometyx Demonstrates Significant Efficacy in Advanced Neuroendocrine Tumors

• Cabometyx (cabozantinib) significantly reduced the risk of disease progression or death in advanced pancreatic and extra-pancreatic neuroendocrine tumors (NETs). • The CABINET Phase III trial showed a 77% reduction in risk for pNETs and a 62% reduction for epNETs compared to placebo. • Median progression-free survival (PFS) was 13.8 months for pNETs and 8.4 months for epNETs treated with Cabometyx, versus 4.4 and 3.9 months with placebo, respectively. • Ipsen has submitted an extension of indication Marketing Authorization to the European Medicines Agency to expand treatment options for advanced NETs.

Enhanced TSOS Intervention Shows Promise for Trauma Survivors in Australia

  • Researchers in Australia assessed the feasibility of an enhanced TSOS intervention for trauma patients at risk of persistent PTSD and pain.
  • The study found the intervention was highly valued by participants and could be implemented cost-effectively within the Victorian trauma system.
  • Preliminary evidence suggests the intervention may reduce PTSD symptoms and pain-related disability, warranting further efficacy studies.
  • The study enrolled patients with complex clinical and social experiences, often excluded from traditional psychology-informed intervention trials.

Clene to Meet with FDA to Discuss CNM-Au8 for ALS

  • Clene Inc. will meet with the FDA before the end of November 2024 to discuss CNM-Au8's development for amyotrophic lateral sclerosis (ALS).
  • The FDA agreed to re-evaluate Clene's submission under the accelerated approval pathway after initial concerns regarding the briefing package.
  • Clene has accumulated over 700 patient-years of safety data for CNM-Au8, showing no significant safety concerns or trends.
  • No serious adverse events related to CNM-Au8 treatment have been identified by clinical trial investigators to date.

China Grants Breakthrough Therapy Designation to GSK's Blenrep for Multiple Myeloma

  • China's NMPA grants Breakthrough Therapy Designation (BTD) to GSK's Blenrep (belantamab mafodotin) combined with BorDex for relapsed or refractory multiple myeloma.
  • The BTD aims to expedite the development of treatments for severe diseases with limited options, showing potential for improved patient outcomes.
  • The designation is based on Phase III DREAMM-7 trial results, demonstrating statistically significant improvements in progression-free survival (PFS).
  • The trial compares belantamab mafodotin plus BorDex against daratumumab plus BorDex, showing clinically meaningful improvements across secondary efficacy endpoints.

FDA Approves Illumina's TruSight Oncology Comprehensive IVD with Velsera Technology

  • Illumina's TruSight Oncology Comprehensive IVD, supported by Velsera's tumor profiling technology, has received FDA approval, enhancing personalized cancer treatment.
  • Velsera's Knowledge Base is integral to the TSO Comprehensive test, classifying cancer mutations for more effective, targeted treatment plans.
  • The collaboration between Velsera and Illumina aims to advance cancer diagnostics and improve the accessibility of targeted therapeutics nationwide.
  • This approval marks a step forward in precision medicine, enabling faster and more precise biomarker identification for enhanced patient care.

Apellis Awaits EU Decision on Pegcetacoplan (Syfovre) Amidst Positive Phase 3 Kidney Data

• Apellis Pharmaceuticals awaits the European Medicines Agency's (EMA) decision on pegcetacoplan (Syfovre) approval, expected from the CHMP meeting on September 16-19. • Mizuho maintains a Neutral rating on Apellis, citing a cautious approach with only a 10% probability of success factored in for EU approval. • Apellis' Phase 3 VALIANT trial demonstrated a significant 68% reduction in proteinuria in patients with C3G and IC-MPGN kidney diseases. • Despite analysts' tempered expectations and a current lack of profitability, Apellis reported strong Q2 2024 growth with SYFOVRE and EMPAVELI sales.

HighField Biopharmaceuticals' HF1K16 Shows Promise in Phase 1 Trial for Refractory Metastatic Cancer

  • HighField Biopharmaceuticals presented positive Phase 1 data for HF1K16, an ATRA-encapsulated liposome, at the ESMO Congress, showing potential in refractory metastatic cancer patients.
  • The trial (NCT05388487) demonstrated encouraging immune modulation and efficacy signals, particularly in a R/R glioma cohort, with some patients surviving over two years.
  • HF1K16 targets myeloid-derived suppressor cells (MDSCs), converting them into active immune cells that stimulate T cells to attack cancer cells.
  • The company plans to advance HF1K16 into Phase 2 trials to determine the most beneficial indication, either as a monotherapy or in combination with standard treatments.

Tolerability and Safety of HF1K16 Injection in Patients With Refractory Solid Tumors

NCT05388487RecruitingPhase 1
HighField Biopharmaceuticals Corporation
Posted 2/16/2022

Passage Bio's PBFT02 Shows Sustained Progranulin Increase in FTD-GRN Patients

• Passage Bio's PBFT02 gene therapy demonstrated robust and sustained increases in CSF progranulin levels in FTD-GRN patients in the upliFT-D trial. • Dose 1 of PBFT02 was well-tolerated in patients receiving an enhanced immunosuppression regimen, with no serious adverse events reported. • CSF progranulin levels remained elevated for up to 12 months post-treatment, exceeding levels found in healthy adult controls. • These findings support PBFT02's potential as a progranulin-raising therapy for neurodegenerative diseases, warranting further investigation.

A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes

NCT04747431RecruitingPhase 1
Passage Bio, Inc.
Posted 9/14/2021

Bio-Path Holdings' BP1003 Demonstrates Therapeutic Potential in Diverse Cancers

  • Bio-Path Holdings announces a publication in Biomedicines highlighting BP1003's therapeutic potential across various cancer types.
  • BP1003, a STAT3 mRNA-targeting antisense drug, showed broad anti-tumor effects in preclinical models of breast, ovarian, and pancreatic cancer.
  • The study suggests BP1003 could enhance the efficacy of standard chemotherapies in treating difficult-to-treat cancers by targeting STAT3.
  • Bio-Path plans to file an IND application for BP1003, furthering its development as a specific STAT3 inhibitor.

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