Clinical Trial News
FDA Accepts PTC Therapeutics' NDA for Sepiapterin to Treat PKU
PTC Therapeutics, Inc. has announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, supported by promising clinical trial data.
CARVYKTI® Approved as First BCMA-Targeted Therapy for Relapsed/Refractory Multiple Myeloma After One Prior Line
- CARVYKTI® (ciltacabtagene autoleucel) gains FDA approval for relapsed or refractory multiple myeloma after just one prior line of therapy.
- The approval is based on the Phase 3 CARTITUDE-4 study, which showed a 59% reduction in disease progression or death compared to standard therapies.
- This approval expands access to CARVYKTI®, offering a potential treatment-free period for more patients with multiple myeloma early in their treatment journey.
- CARVYKTI® is a personalized, one-time infusion that harnesses a patient's own T-cells to fight the disease, targeting B-cell maturation antigen (BCMA).
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 6/12/2020
AI-Driven Real-World Evidence Revolution: New Paradigm for Biotech Companies
- Biotech companies are adopting tech industry principles to generate real-world evidence (RWE) more efficiently, leveraging AI and cloud infrastructure instead of building expensive internal capabilities.
- Cross-functional teams led by specialists with dual expertise in computing and life sciences are essential for successful implementation of AI-powered RWE generation systems.
- Cloud-based infrastructure and machine learning technologies are enabling smaller biotechs to produce high-quality real-world data analysis without massive internal resource investment.
Harmony Valve Shows Excellent Real-World Outcomes in TPVR
- A real-world study demonstrates the self-expanding Harmony valve's effectiveness in transcatheter pulmonary valve replacement (TPVR), with high rates of technical success.
- After a median follow-up of 13 months, 98% of patients had mild or less pulmonary regurgitation, and 99% were free from hemodynamic dysfunction after one year.
- Thrombosis was observed in 3.4% of patients, and endocarditis was diagnosed or suspected in 2.6%, highlighting the need for vigilance regarding potential risks.
- The study confirms the short-term performance of self-expanding TPVR, showing successful elimination of pulmonary regurgitation and maintenance of hemodynamic stability.
Caribou Biosciences Expands Clinical Development of CB-010 with FDA Clearance of IND in Lupus
Caribou Biosciences has received FDA clearance for its IND application for CB-010, an allogeneic anti-CD19 CAR-T cell therapy, to treat lupus nephritis and extrarenal lupus. The GALLOP Phase 1 clinical trial is set to begin by the end of 2024, following encouraging results from the ANTLER trial for B cell non-Hodgkin lymphoma.
Highlighted Clinical Trials:
Caribou Biosciences, Inc.
Posted 5/26/2021
FDA Requires Fast Track Designation for Subcutaneous Formulation of Leqembi for Alzheimer's Disease
The FDA has informed Eisai Co Ltd and Biogen Inc that a Fast Track designation specific to the subcutaneous formulation of Leqembi is necessary for rolling review, altering Eisai's regulatory submission plans. Despite delays, the potential of Leqembi remains significant, with its non-subcutaneous formulation already receiving traditional FDA approval.
Court Rules in Favor of Arbutus in Patent Dispute Over Moderna's COVID-19 Vaccine Delivery Technology
- Delaware District Court Judge Mitchell Goldberg sided with Arbutus Biopharma and Genevant Sciences in their patent infringement case against Moderna regarding lipid nanoparticle technology used in COVID-19 vaccines.
- The ruling favored Arbutus and Genevant's interpretation of two disputed patent claims concerning lipid percentages and cationic lipid specifications, rejecting Moderna's more restrictive definitions.
- The lawsuit, initially filed in February 2022, seeks compensation for Moderna's alleged unauthorized use of patented lipid nanoparticle delivery technology that was crucial to its COVID-19 vaccine success.
IND Application Submitted to FDA for SynKIR-310 Trial in Relapsed/Refractory B-NHL
Verismo Therapeutics has submitted an Investigational New Drug (IND) application to the FDA for the phase 1 trial of SynKIR-310, targeting relapsed/refractory B-cell non-Hodgkin lymphomas (B-NHL). The trial aims to evaluate the efficacy of SynKIR-310 in treating various B-NHL subtypes, including DLBCL, FL, MCL, and MZL, with potential improvements in T-cell antitumor function and persistence.
Highlighted Clinical Trials:
Verismo Therapeutics
Posted 3/30/2023
Key Strategic Decisions Shaping the Future of Cell and Gene Therapy Development
- The global cell and gene therapy market, valued at nearly $23 billion in 2023, faces unique challenges in patient identification, requiring genetic screening and comprehensive pre-screening protocols for trial enrollment.
- Complex laboratory assessments throughout the CAGT product lifecycle demand early planning and validation, with sponsors increasingly relying on CROs and specialty lab partners for end-to-end management.
- Recent FDA initiatives, including the establishment of the Office of Therapeutic Products and updated regulatory guidance, are reshaping the development landscape for cell and gene therapies.
Behavioral Science Emerges as Key Strategy for Driving Adoption of Novel Therapies in Biopharma
- Pharmaceutical companies are increasingly turning to behavioral science principles to overcome barriers in prescribing patterns and drive adoption of new therapies, moving beyond traditional educational approaches.
- Research into Parkinson's disease treatment adoption reveals that physician capability is only a small factor in prescribing decisions, with motivation and perceived opportunity playing more significant roles.
- The COM-B model, featuring 93 different behavioral techniques, is being implemented to address complex barriers in healthcare decision-making, particularly for expensive novel therapies with limited patient populations.