Tagged News
FDA Questions Pfizer's Approach to Expand Talzenna's Indication in Prostate Cancer
• The FDA has raised concerns about Pfizer's strategy to expand Talzenna's indication to all castration-resistant prostate cancer patients, questioning the reliance on an "incompletely defined" subgroup without HRR gene mutations.
• Pfizer is seeking to broaden Talzenna's current first-line indication from only HRR-mutated castration-resistant prostate cancer to an all-comers population, representing a significant potential market expansion.
• The regulatory scrutiny highlights ongoing challenges in precision medicine approaches and biomarker-based treatment strategies in oncology drug development.
FDA Advisory Committee Rejects Pfizer's Talzenna Expansion for Broader Prostate Cancer Population
• Pfizer's application to expand Talzenna's indication to all castration-resistant prostate cancer (CRPC) patients was rejected by FDA's advisory committee due to insufficient data in biomarker-negative patients.
• The company sought to broaden Talzenna's current approval beyond patients with HRR gene mutations, but FDA questioned the reliance on an "incompletely defined" subgroup analysis.
• FDA oncology chief Richard Pazdur emphasized that chance effects could not be ruled out due to the lack of a formal efficacy analysis in patients without HRR mutations.
Breakthrough in Platelet Engineering Opens New Avenues for Targeted Drug Delivery
• University of Illinois researchers have successfully applied metabolic glycan labeling to platelets for the first time, overcoming the challenge of engineering these nucleus-free cell fragments.
• The novel chemical approach allows scientists to attach tags to platelet surfaces within hours, both in laboratory settings and living organisms, creating potential vehicles for targeted drug delivery.
• This breakthrough could revolutionize treatment for cancer, immune diseases, and blood clotting disorders, with the added benefit that platelets' short half-life ensures drugs clear from the body within days.
Taiwan to Expand National Health Insurance Coverage for Cancer Immunotherapies and Targeted Drugs
• Taiwan's National Health Insurance Administration will expand coverage to include three types of cancer immunotherapies for lung, colorectal, and breast cancers, benefiting up to 3,400 patients starting June 2025.
• The expansion will also cover two PARP inhibitors (olaparib and niraparib) for treating five cancer types, including ovarian, fallopian tube, peritoneal, breast, and prostate cancers, helping approximately 775 additional patients.
• Patients could save between NT$1.71 million to NT$2.47 million annually in medical expenses, with the government allocating NT$3.295 billion from a dedicated cancer fund for immunotherapies.
FDA's Approval of MRD as End Point Transforms Multiple Myeloma Research and Treatment
• The FDA's Oncology Drugs Advisory Committee unanimously approved minimal residual disease (MRD) as an end point for accelerated approval of multiple myeloma therapies in April 2024, potentially reducing trial timelines from 10-15 years to just 3 years.
• Pharmaceutical companies have rapidly adapted by implementing MRD as a coprimary end point in new trials and amending existing protocols, with the CEPHEUS trial being the first major study to read out with MRD as a coprimary endpoint.
• Researchers are now exploring MRD applications beyond drug approval, including using MRD status to guide treatment decisions, developing improved blood-based detection technologies, and expanding the approach to other hematologic malignancies.
Related Clinical Trials:
University of Miami
Posted 2/11/2020
Janssen Research & Development, LLC
Posted 11/6/2018
Stichting European Myeloma Network
Posted 12/14/2018
CellCentric Secures $120 Million Series C Funding to Advance Oral Multiple Myeloma Treatment
• CellCentric has raised $120 million in Series C funding to advance inobrodib, a first-in-class oral p300/CBP inhibitor for multiple myeloma treatment.
• The funding will support a Phase II/III study with potential for accelerated approval, as well as trials combining inobrodib with bi-specific antibodies and in maintenance settings.
• Inobrodib has received FDA Fast Track and orphan drug designation, with promising efficacy and safety data presented at the American Society of Hematology Annual Meeting.
GT Biopharma Advances GTB-3650 Phase 1 Trial to Second Cohort Following Promising Immune Activation Signals
• GT Biopharma has successfully completed dosing in the first cohort of its Phase 1 trial for GTB-3650, a second-generation TriKE immunotherapy, with no safety or tolerability issues observed.
• Both patients in Cohort 1 demonstrated early biological signs of immune activation, including increased natural killer cell activity and expansion, supporting the therapy's mechanism of action.
• The trial, which will evaluate approximately 14 patients across seven dose cohorts, aims to treat relapsed or refractory CD33-expressing hematologic malignancies through enhanced NK cell-mediated cancer destruction.
Related Clinical Trials:
Masonic Cancer Center, University of Minnesota
Posted 11/19/2024
Eascra Biotech Secures $100,000 MassVentures Grant for Novel Nanoparticle Drug Delivery Platform
• Eascra Biotech has received a $100,000 Stage I SBIR Targeted Technologies grant to advance their Janus Base Nanoparticles (JBNps) delivery platform for hard-to-reach tissues.
• The company has leveraged microgravity research, completing five missions on the International Space Station, becoming the first commercial entity to produce medical nanoparticles in space.
• Eascra's proprietary technology can deliver RNA therapeutics to challenging tissues like cartilage and solid tumors while maintaining mRNA stability at room temperature, eliminating cold chain requirements.
Fujifilm Validates Novel Peptide-Oligonucleotide Conjugates for Targeted Cancer Cell Delivery
• Fujifilm has successfully validated peptide-oligonucleotide conjugates that selectively target cancer cells, utilizing cyclic peptides from their proprietary library containing trillions of variants.
• The company developed a cyclic peptide that binds strongly to integrins overexpressed on cancer cell surfaces with a low dissociation constant of 1.6 nM, enabling higher cellular uptake compared to non-conjugated oligonucleotides.
• This breakthrough addresses delivery challenges in oligonucleotide-based therapies and will be presented at TIDES USA 2025 in San Diego, highlighting Fujifilm's expansion of global drug discovery services for peptide therapeutics.
Gustave Roussy Launches UMBRELLA Trial: Using Blood-Based Tumor DNA to Revolutionize Cancer Follow-Up Care
• Gustave Roussy has initiated UMBRELLA, a groundbreaking phase III trial that personalizes post-treatment cancer monitoring based on minimal residual disease (MRD) detected through blood-based circulating tumor DNA analysis.
• The trial will evaluate two strategies: pre-emptive immunotherapy (tislelizumab) for MRD-positive patients and reduced follow-up schedules for MRD-negative patients with non-metastatic lung, colorectal, pancreatic, and soft tissue sarcomas.
• UMBRELLA represents the first French multi-cancer trial using MRD status for therapeutic stratification, aiming to enroll over 700 patients across 10-11 centers in a collaborative effort involving Veracyte, IntegraGen, and BeiGene.
Related Clinical Trials:
Gustave Roussy, Cancer Campus, Grand Paris
Posted 4/16/2025