Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne muscular dystrophy
- Neu-REFIX Beta glucan has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the US FDA for Duchenne muscular dystrophy (DMD).
- These designations will facilitate the transition of pre-clinical and clinical studies from Japan and India to clinical trials in the USA.
- Neu-REFIX Beta glucan has shown potential in reducing skeletal muscle inflammation, enhancing muscle regeneration, and improving plasma dystrophin levels in pre-clinical and clinical studies.
- The designations provide benefits such as market exclusivity, access to grants, and fast-track progress for regulatory approval.
Read more
FDA fast track designation for narmafotinib in advanced pancreatic cancer
- The US FDA has granted Fast Track Designation to Amplia's lead drug narmafotinib for advanced pancreatic cancer.
- Fast Track Designation facilitates the development and expedited review of investigational drugs.
- Narmafotinib is currently in an open-label Phase 2a trial in combination with gemcitabine and Abraxane.
- The ACCENT trial for narmafotinib is ongoing in Australia and South Korea, with plans for a US trial in advanced stages.
Read more
Edgewise Therapeutics announces positive top-line data from Phase 1 and Phase 2 CIRRUS-HCM trials
- Edgewise Therapeutics reported positive top-line data for EDG-7500 from Phase 1 and Phase 2 CIRRUS-HCM trials.
- In Phase 1, EDG-7500 was well tolerated with no significant changes in vital signs or LVEF across various doses.
- Phase 2 CIRRUS-HCM Part A showed significant reductions in LVOT pressure gradients and NT-proBNP levels in patients with obstructive HCM.
- The company has initiated the 28-day part of CIRRUS-HCM to further evaluate tolerability, pharmacokinetics, and effects on LVOT-G, LVEF, and biomarkers.
Read more
Tolebrutinib demonstrated a 31% delay in time to onset of confirmed disability progression in non-relapsing secondary progressive multiple sclerosis phase 3 study
- Tolebrutinib delayed the time to onset of 6-month confirmed disability progression (CDP) by 31% compared to placebo in the HERCULES phase 3 study.
- Secondary endpoints showed nearly a two-fold increase in confirmed disability improvement with tolebrutinib (10%) compared to placebo (5%).
- Adverse events included liver enzyme elevations (>3xULN) in 4.1% of tolebrutinib-treated patients versus 1.6% in the placebo group.
- Global regulatory submissions for tolebrutinib will begin in H2 2024.
Read more
Xspray Pharma announces positive FDA meeting and plans for Dasynoc NDA resubmission
- Xspray Pharma plans to resubmit the NDA for Dasynoc in Q4 2024, following a productive meeting with the FDA.
- FDA recommended adjustments to Dasynoc's tablet strengths to reduce medication errors, requiring new batches to be produced.
- The company has initiated production of the new batches and will provide further clarification on the manufacturing process.
- A final FDA decision on Dasynoc is expected within two to six months of the resubmission, potentially leading to a Q1 2025 launch.
Read more
CD (Suzhou) Biopharma announces FDA clearance for Phase I clinical trial of CD-001
- CD (Suzhou) Biopharma received FDA clearance for its IND application of CD-001.
- CD-001 is built on the company's proprietary Bispecific Fusion Protein (BsFP) platform.
- The therapy targets PD-1 positive CD8+ T cells using an anti-PD-1 antibody and engineered IL-21 mutant.
- Initial clinical data from the Phase I trial is expected in the upcoming months.
Read more
City of Hope reports positive results on phase 1 trial of personalized vaccine for lymphoplasmacytic lymphoma
- City of Hope and M.D. Anderson Cancer Center report safety and efficacy results from a Phase 1 trial of a personalized vaccine for lymphoplasmacytic lymphoma.
- The vaccine nearly doubled the disease-free progression time to an average of just under seven years.
- The trial enrolled nine patients, all of whom tolerated the therapy without negative side effects.
- Next steps include adapting the vaccine to an mRNA platform and combining it with other agents for enhanced efficacy.
Read more
Kiromic BioPharma advances Deltacel-01 into expansion phase following safety monitoring committee’s unanimous recommendation
- Kiromic BioPharma's Deltacel-01 trial moves to the expansion phase after unanimous SMC approval.
- The trial targets stage 4 metastatic non-small cell lung cancer (NSCLC) patients unresponsive to standard therapies.
- Safety data from the first two cohorts showed no dose-limiting toxicities, supporting the decision.
- Approximately nine new patients will be enrolled in the expansion phase, starting later this month.
Read more
InnoCare announces approval of clinical trial of BCL2 inhibitor ICP-248 for acute myeloid leukemia in China
- InnoCare Pharma received IND approval to conduct a clinical trial of BCL2 inhibitor ICP-248 in combination with azacitidine for AML in China.
- AML is a malignant hematological disease, accounting for about 80% of acute leukemia in adults and 15-20% in pediatric cases.
- ICP-248 is a novel, orally bioavailable BCL2-selective inhibitor that restores programmed cell death mechanisms.
- InnoCare aims to accelerate clinical development to benefit patients with hematological malignancies.
Read more
Apellis receives negative CHMP opinion for pegcetacoplan for geographic atrophy in the EU following re-examination
- The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) confirmed its negative opinion on Apellis' marketing authorization application for pegcetacoplan.
- Pegcetacoplan is an investigational therapy for geographic atrophy (GA) secondary to age-related macular degeneration.
- Despite broad support from the European retina community, the negative opinion leaves millions of Europeans without a treatment for this irreversible form of blindness.
- Apellis remains committed to expanding access to pegcetacoplan in the U.S. and other regions globally.
Read more
