Mesoblast | MedPath

NextCell Pharma Strengthens Commercial Strategy with Appointment of MSC Therapy Expert Dr. Eric Strati

3 days ago

• NextCell Pharma has appointed Dr. Eric Strati, former executive at Takeda, Mesoblast, and Novartis, to its Board of Advisors to advance commercial strategy for ProTrans, its type 1 diabetes cell therapy. • Dr. Strati brings critical expertise from launching two approved MSC-based therapies (Alofisel and Ryoncil), strengthening NextCell's capabilities as it prepares for pivotal trials and partnership opportunities. • Recent analysis from the ProTrans-Young study shows promising trends in preserving insulin production in patients aged 12-21 years, with full trial results expected in the second half of 2026.

Myocardial Infarction Therapeutics Pipeline Shows Promise with Over 45 Companies Involved

4 months ago

• The myocardial infarction therapeutics pipeline is robust, featuring over 45 companies developing more than 50 potential drugs. • Key companies like Boehringer Ingelheim and Idorsia Pharmaceuticals are advancing novel therapies to improve outcomes post-myocardial infarction. • Several promising therapies, including Selatogrel and FDY-5301, are in Phase III trials, showing potential for significant advancements. • Recent clinical trial activities, such as the commencement of the PERFECT study and completion of enrollment in the Iocyte AMI-3 trial, highlight ongoing research efforts.

Wegovy and Zepbound Expected to Drive $20B Growth in Heart Failure Market

4 months ago

GlobalData predicts that the heart failure market will see a significant boost, adding $20 billion in value over the next decade, thanks to the introduction of GLP-1 drugs like Eli Lilly’s Zepbound and Novo Nordisk’s Wegovy. The market, which was valued at $13.5 billion in 2022, is expected to grow to $33.7 billion by 2032, driven by new treatments and mechanisms of action.

FDA Roundup: Approvals, Breakthrough Designations, and Clinical Holds in Neurology and Rare Diseases

5 months ago

• The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels. • Tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis based on phase 3 trial results showing delayed disability progression. • The FDA placed a clinical hold on PepGen’s PGN-EDO51 phase 2 study for Duchenne muscular dystrophy, pending further clarification from the agency.

FDA Approvals in 2024: Novel Therapies for MASH, WHIM Syndrome, Pediatric Glioma, MDS, and Bladder Cancer

5 months ago

• The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease. • X4 Pharmaceuticals' Xolremdi gained approval as the first targeted therapy for WHIM syndrome, a rare immunodeficiency, marking a milestone for the company. • Day One Biopharmaceuticals' Ojemda secured approval for BRAF-altered pediatric low-grade glioma, offering a new treatment option for this common childhood brain tumor. • Geron Corporation's Rytelo, a telomerase inhibitor, received FDA approval for lower- to intermediate-risk myelodysplastic syndromes (MDS), after 34 years in business. • ImmunityBio's Anktiva, a novel IL-15 superagonist, was approved for non-muscle invasive bladder cancer, providing a new therapeutic approach for BCG-unresponsive patients.

FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric Steroid-Refractory Acute Graft-versus-Host Disease

5 months ago

• The FDA has approved Ryoncil (remestemcel-L-rknd) as the first mesenchymal stromal cell (MSC) therapy for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients. • Ryoncil's approval was based on a Phase III trial demonstrating a 70% overall response rate in children with SR-aGVHD after 28 days of treatment. • This allogeneic, bone marrow-derived MSC therapy offers a new treatment option for children with SR-aGVHD who do not respond to steroid treatment. • Ryoncil is administered intravenously and should be monitored for infusion reactions; common side effects include infections, fever, hemorrhage, and abdominal pain.

FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric SR-aGVHD

6 months ago

• The FDA has approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States. • Ryoncil is indicated for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older. • Clinical trials showed a 70% overall response rate by Day 28 in children with SR-aGvHD treated with Ryoncil. • Ryoncil offers a new treatment option for a life-threatening condition with limited alternatives, improving outcomes for affected children.

Eli Lilly's Zepbound Demonstrates Superior Weight Loss Compared to Novo Nordisk's Wegovy in Head-to-Head Trial

6 months ago

• Eli Lilly's Zepbound (tirzepatide) led to a 20.2% average weight loss, significantly outperforming Wegovy (semaglutide) at 13.7% in a 72-week clinical trial. • The SURMOUNT-5 trial included overweight or obese adults without diabetes, showing Zepbound users experienced 47% more relative weight loss than Wegovy users. • Zepbound, a dual GIP and GLP-1 receptor agonist, helped 31.6% of participants achieve at least 25% body weight loss, compared to 16.1% with Wegovy. • The study's findings may influence treatment choices, with Zepbound potentially becoming a preferred option for greater weight loss outcomes, pending further data on tolerability.

Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025

7 months ago

• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD. • EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration. • Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option. • Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.