Mesoblast

FDA grants RMAT designation to Mesoblast’s rexlemestrocel-L for treating children with hypoplastic left heart syndrome (HLHS). The therapy, an allogeneic mesenchymal precursor cell therapy, previously showed significant increases in left ventricular volumes in a phase 1/2 trial and improved survival in patients with heart failure in the DREAM-HF trial.

FDA grants RMAT designation to Revascor® (rexlemestrocel-L) for treating children with HLHS, following RPDD and ODD designations. Clinical trial results show significant improvement in left ventricular volumes over 12 months, crucial for surgical correction and reducing risks of heart failure, liver failure, and death. RMAT designation includes benefits like Breakthrough and Fast Track designations, rolling review, and priority review eligibility for BLA.

The article lists six top ASX shares for December 2024, including Mesoblast Ltd, Lovisa Holdings Ltd, Brickworks Limited, TechnologyOne Ltd, NextDC Ltd, and BHP Group Ltd, with market capitalisations ranging from $2.02 billion to $205.85 billion. It provides brief insights into why each stock is considered a smart investment, focusing on their growth potential, market position, and financial performance.

DelveInsight's report on Crohn’s Disease Market Insights, Epidemiology, and Market Forecast-2034 details the market size, emerging therapies, and key companies like Takeda Pharmaceutical, Janssen Pharmaceuticals, UCB, Biogen, AbbVie, AstraZeneca, Tillotts Pharma, Gilead Sciences, Galapagos NV, Boehringer Ingelheim, Celgene (Bristol Myers Squibb), Eli Lilly and Company, RedHill Biopharma, Arena Pharmaceuticals, Mesoblast, and others. The market size in 7MM was ~USD 9,000 million in 2023, expected to increase by 2034, with key therapies like ENTYVIO, STELARA, VELSIPITY, Zeposia, LY-3074828, RHB-104, and others expected to launch. Positive results for mirikizumab and TREMFYA were reported in 2024, and Dong-A-ST's Imuldosa received FDA approval as a biosimilar to Stelara.

Longeveron Inc. appoints Devin Blass as CTO and SVP of CMC, effective December 2, 2024, to lead technological and manufacturing strategies. Blass brings over 15 years of experience in advanced therapies, most recently at New York Blood Center.

Longeveron Inc. appoints Devin Blass as CTO and SVP of CMC, effective December 2, 2024, to lead technological and manufacturing strategies. Blass, with over 15 years of experience in advanced therapies, previously served at NYBC and Talaris Therapeutics. He holds a BS in Biochemistry from Texas State University.

Mesoblast reported Q1 FY2025 milestones, including an expected FDA decision on Ryoncil for SR-aGvHD in children by 7 Jan 2025. The company secured manufacturing and distribution channels, completed a Pre-License Inspection, and arranged US$50m financing post-approval. Revascor achieved Rare Pediatric Disease and Orphan-Drug Designation, and Phase 3 trials for chronic low back pain are ongoing. Q1 net operating cash outflows were US$10.5m, a 26% reduction, with a cash balance of US$51.1m and US$60m in financing facilities pending approval.

AusBiotech 2024 will feature over 260 speakers and 60 sessions, addressing global biotech trends, research, and industry advancements. Key topics include artificial heart technology, quantum computing in biotech, and medical manufacturing. The event also highlights emerging biotechnologies in agriculture and cell and gene therapies, supported by government initiatives and strategic partnerships. Australia's biotech sector is rapidly advancing, driven by innovation, government support, and strategic geographical clustering near research institutions.

Mesoblast advances late-stage pipeline, prepares for Ryoncil® launch pending FDA approval, secures $50 million for commercialization, and progresses Phase 3 trial for rexlemestrocel-L.