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Clinical Trial News

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.

GigaPath AI Model Shows Superior Accuracy in Predicting Cancer Mutations and Tumor Burden

  • GigaPath, a billion-parameter AI foundation model, demonstrates significant improvement in predicting cancer mutations, achieving an AUROC of 0.626 for lung adenocarcinoma, surpassing existing approaches.
  • The model, trained on over 1.3 billion image tiles from 171,189 H&E slides across 28 cancer centers, outperformed competing methods in pan-cancer gene prediction with 6.5% improvement in macro-AUROC.
  • Researchers have made GigaPath available as an open-source tool, enabling independent validation and potential integration with other imaging modalities and clinical data sources.

Nested Therapeutics' NST-628: A Novel Pan-RAF–MEK Molecular Glue for MAPK Pathway Inhibition

  • Nested Therapeutics is developing NST-628, an oral, brain-penetrant pan-RAF–MEK molecular glue designed to avoid paradoxical pathway activation.
  • NST-628 differentiates itself from other RAS/RAF/MAPK pathway inhibitors through its unique mechanism of action.
  • Preclinical data supports the clinical development of NST-628, suggesting its potential as an ideal combination therapy partner.
  • The novel mechanism and preclinical profile of NST-628 mark it as a significant advancement in targeting the MAPK pathway.

FDA Approves Roche's Tecentriq Hybreza, a Subcutaneous PD-L1 Inhibitor, for Multiple Cancers

  • The FDA has approved Roche's Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs), a subcutaneous formulation, for multiple cancer types previously approved for the IV form.
  • Tecentriq Hybreza, combining atezolizumab with Halozyme's Enhanze technology, offers a faster, approximately 7-minute injection compared to the 30-60 minute IV infusion.
  • Clinical trials demonstrated comparable drug levels, safety, and efficacy to IV Tecentriq, with 71% of patients preferring the subcutaneous formulation due to convenience.
  • This approval marks Tecentriq Hybreza as the first and only subcutaneous anti-PD-L1 cancer immunotherapy available in the US, enhancing treatment flexibility.

New Research Highlights Complexities of Central Centrifugal Cicatricial Alopecia in Diverse Populations

  • University of Pennsylvania study reveals that 72% of Central Centrifugal Cicatricial Alopecia (CCCA) cases show classic presentation, while 28% display atypical distributions, challenging current diagnostic approaches.
  • Research identifies significant comorbidities in CCCA patients, including high rates of hyperlipidemia (66%), hypertension (70%), and obesity (64%), suggesting potential links to cardiovascular disease and breast cancer.
  • Expert emphasizes the importance of culturally sensitive approaches to hair care modifications, particularly for African American patients, while calling for increased research in diverse populations.

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