GILEAD SCIENCES SL

Wesley Sundquist's research on HIV's molecular structure led to the development of lenacapavir, a long-lasting HIV prophylactic. Lenacapavir, effective for six months, has shown 100% efficacy in preventing HIV transmission in clinical trials.

The FDA identified additional safety concerns with Ocaliva, used to treat primary biliary cholangitis, including serious liver injury in non-cirrhotic patients. Since 2021, 20 more liver-related events were reported. The FDA advises frequent liver tests and discontinuation if disease progresses or if the drug is ineffective. Ocaliva, once a blockbuster prospect, faces setbacks with FDA rejections and competition from new drugs.

Lenacapavir, an injectable HIV drug targeting capsid protein, was named Breakthrough of the Year by 'Science'. It offers 6-month protection per shot and showed 100% efficacy in preventing HIV infections in African adolescent girls and young women. Regulatory approval is expected no earlier than mid-2025.

Science magazine names twice-yearly lenacapavir PrEP its 2024 Breakthrough of the Year, recognizing its potential to significantly reduce HIV acquisition risk. Lenacapavir, an HIV capsid inhibitor, is administered every six months and shows promise for broader viral disease applications. Gilead is developing longer-acting formulations, including a potential once-a-year injectable version. The breakthrough underscores ongoing efforts to combat HIV/AIDS, with lenacapavir offering a new, effective prevention option.

ASH 2024 highlights include GSK's Blenrep Phase 3 data supporting re-market, J&J's Darzalex Faspro delaying progression in smoldering multiple myeloma, Arcellx's anito-cel showing comparable safety to Carvykti, and J&J/Legend's Carvykti improving survival outcomes. Kura Oncology's ziftomenib showed high response rates in leukemia, Eli Lilly's Jaypirca reduced disease progression risk, and Merck's zilovertamab vedotin achieved high complete response rates in lymphoma. Beam Therapeutics' BEAM-101 showed durable effects in sickle cell disease, Novo Nordisk's etavopivat reduced crises, and Bristol Myers Squibb's arlo-cel demonstrated durable responses in multiple myeloma. Galapagos' GLPG5101 showed encouraging cell therapy results, Orca Bio's Orca-T improved survival in blood cancers, and Sanofi's rilzabrutinib improved platelet response in thrombocytopenia. Regeneron's drug combo showed better disease control than Ultomiris in PNH.

KEMRI researchers urge Kenya's Pharmacy and Poisons Board to approve Cabotegravir and Lenacapavir, injectable drugs with high efficacy in preventing HIV. Cabotegravir, requiring injections every eight weeks, and Lenacapavir, every six months, have shown significant prevention rates, approved by the FDA and EMA. Despite Cabotegravir's registration in Kenya, Lenacapavir's application is pending, and both drugs are costly, posing accessibility challenges. KEMRI appeals for faster approvals to reduce HIV infections in Kenya.

uniQure's QURE surged 109.7% after aligning with FDA on an accelerated approval pathway for AMT-130, a gene therapy for Huntington's disease. The FDA agreed that data from ongoing studies are sufficient for regulatory filing, avoiding additional pre-submission studies. The therapy's safety profile is manageable, and uniQure plans to submit a biologics license application in 2025.

ZUMA-5 analysis shows Yescarta's median progression-free survival of 62.2 months and median duration of response of 60.4 months after 5+ years follow-up, with 55% of patients alive without new therapy, suggesting curative potential for relapsed/refractory non-Hodgkin lymphomas.

Adenosine antagonists market growth driven by R&D in neurodegenerative diseases, oncology, and cardiovascular disorders. Innovations in formulation and delivery systems enhance efficacy and safety, increasing adoption. Key companies include Arcus Biosciences, Corvus Pharmaceuticals, Portage Biotech, Palobiofarm, iTeos Therapeutics. Promising drugs like Etrumadenant, Ciforadenant, PORT-6, and Inupadenant are in various clinical trial phases.