Setrusumab

Ultragenyx is developing setrusumab for Osteogenesis Imperfecta, with Phase 3 study results expected soon. Praxis Precision Medicines is working on ulixacaltamide for essential tremor, also awaiting Phase 3 study results this quarter.

Mereo BioPharma, a biopharmaceutical company specializing in rare diseases, announced CEO Dr. Denise Scots-Knight's presentation at the 43rd Annual J.P. Morgan Healthcare Conference. The company focuses on innovative therapeutics, including setrusumab for osteogenesis imperfecta and alvelestat for AATD-LD, with partnerships and designations from regulatory bodies. Mereo also explores oncology treatments and has global licensing agreements for its product candidates.

H.C. Wainwright maintains a Buy rating for Ultragenyx Pharma with a $95 target, citing its Phase 3 Aspire study for GTX-102 targeting Angelman syndrome. Ultragenyx shows strong revenue growth and liquidity, with a 42% increase in Q3 2024 revenue but a $134M net loss. The company aims for GAAP profitability by 2026, with potential new therapies on the horizon.

Ultragenyx submitted a BLA to the FDA for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. The BLA is supported by data showing UX111's ability to reduce heparan sulfate levels in the CSF and improve cognitive development. UX111 is well-tolerated and has various designations in the U.S. and EU. Ultragenyx also dosed the first patient in the Aspire study for Angelman syndrome.

Mereo BioPharma Group plc, a $579M market cap biopharma, focuses on rare diseases like osteogenesis imperfecta (OI) and alpha-1 antitrypsin deficiency (AATD)-associated lung disease. Setrusumab, its lead asset for OI, shows promising Phase 2 results, with potential sales exceeding $750M. Alvelestat, targeting AATD, has encouraging Phase 2 data, paving the way for Phase 3 studies. With $81M in cash, Mereo's financial position supports operations into 2026/2027, and potential partnerships could enhance its financial flexibility. Analysts project significant market opportunities and upside potential for Mereo's stock.

Mereo BioPharma Group plc (NASDAQ:MREO) is a clinical-stage biopharmaceutical company focusing on rare diseases like osteogenesis imperfecta (OI) and alpha-1 antitrypsin deficiency (AATD)-associated lung disease. Its lead asset, setrusumab, a monoclonal antibody for OI, has shown promising Phase 2 results, with pivotal studies ongoing. Alvelestat, a small molecule for AATD-associated lung disease, has also shown encouraging Phase 2 data, with a Phase 3 trial expected to start in late 2024. Mereo's financial position is stable, with cash reserves estimated to fund operations into 2027. The company is actively seeking partnerships to advance its pipeline and commercialize its products, with significant revenue potential from successful drug approvals.

Mereo BioPharma's CEO, Dr. Denise Scots-Knight, to participate in a Fireside Chat at the Jefferies London Healthcare Conference on Nov 19, 2024, at 10:30am ET / 03:30pm GMT. Live audio webcast available on the Company’s website, with an archived replay for two weeks.

Mereo BioPharma's Q3 2024 report shows no revenue, $9.4M operating loss, and $15M net loss. Focus on rare disease therapeutics with setrusumab and alvelestat in development. Expects significant operating losses and needs additional funding for R&D and potential commercialization.

Mereo BioPharma reports $80.5 million cash as of Sept 30, 2024, expected to fund operations into 2027. Setrusumab receives Breakthrough Therapy designation from the FDA for osteogenesis imperfecta treatment. Alvelestat expected to be Phase 3-ready by end of 2024.