ALEXION PHARMACEUTICALS, INC.

The 'Myositis - Pipeline Insight, 2024' report by ResearchAndMarkets.com offers insights into 18+ companies and 20+ pipeline drugs for Myositis, covering drug profiles, therapeutic assessments, and pipeline development activities.

Moderna, a biotech company specializing in mRNA technology, developed one of the first COVID-19 vaccines and researches treatments for infectious diseases, cancer, and rare genetic disorders. Founded in 2010, it became publicly traded in 2018 and has since expanded its mRNA-based vaccine research to include RSV, HIV, and Zika fever, among others.

Tenpoint Therapeutics and Visus Therapeutics merge to focus on ophthalmic therapeutics, including BRIMOCHOL™ PF for presbyopia, with a US launch planned for 1H 2026. The merger involves a syndicate of investors and appoints Henric Bjarke as CEO, aiming to advance innovative treatments for presbyopia, cataracts, and geographic atrophy.

Regeneron achieved significant trial results at the American Society for Hematology meeting, demonstrating better disease control in paroxysmal nocturnal hemoglobinuria with a combination therapy and potential best-in-class performance in follicular lymphoma with a bispecific antibody. Dr. Andres Sirulnik highlights Regeneron's science-driven approach and the company's focus on addressing difficult-to-treat problems in hematology.

Pharma leaders discussed clinical trial quality at SCOPE Europe, emphasizing the shift from reactive to proactive, risk-based approaches. Challenges include cultural resistance and high implementation costs, highlighting the need for senior leadership support and data-driven decision-making to ensure quality and efficiency in clinical trials.

IgA nephropathy affects 25-50 per million annually, with 20–40% progressing to end-stage kidney disease. Diagnosed cases in the US are expected to reach 135,000 by 2030. Treatments include ACE inhibitors, ARBs, corticosteroids, and immunosuppressants. FDA-approved treatments are TARPEYO, FILSPARI, and FABHALTA. The IgA nephropathy market is projected to reach ~USD 4.1 billion by 2034, driven by novel therapies like Novartis’ Atrasentan, Otsuka/Visterra’s Sibeprenlimab, Vertex/Alpine’s Povetacicept, and others.

Katherine Talcott, MD, discusses potential therapies for geographic atrophy (GA) secondary to age-related macular degeneration (AMD) at OSN New York 2024, highlighting ANX007, Danicopan ALXN 2040, AVD-104, JNJ-1887, ASP7317, RG6501, elamipretide, Glideuretinal, AREDS/AREDS2, and ONL1204. ANX007 showed significant protection from vision loss in the ARCHER Trial, while Danicopan ALXN 2040 is an oral agent inhibiting complement factor D. AVD-104 demonstrated visual acuity gains in the SIGLEC study. Gene therapies like JNJ-1887 and ASP7317 aim to protect retinal cells. Elamipretide showed positive effects on visual function in the ReCLAIM study. Glideuretinal and AREDS/AREDS2 are antioxidants, and ONL1204 is a Fas inhibitor reducing retinal cell apoptosis.

Iptacopan (Fabhalta) showed a clinically meaningful reduction in proteinuria and confirmed clinical benefit of alternative pathway inhibition in IgA nephropathy, with a 38.3% lower UPCR than placebo by month 9. The study also found significant decreases in complement pathway biomarkers, supporting selective alternative pathway inhibition.

PhotonPharma Inc. appoints Derek Brown as CEO, securing $2.5 million in seed funding for ovarian cancer therapy. Brown, with over 25 years of biopharmaceutical experience, replaces Alan Rudolph and will guide the company through its growth phase.

BIOTECanada welcomes Anne Woods, Tamer Mohamed, and Karen Heim to its Board of Directors, enhancing its commitment to advancing Canada's biotechnology sector.