Madrigal Pharmaceuticals

In 2024, the FDA approved over 130 drugs, including 40 novel ones. Madrigal's Rezdiffra is the first treatment for MASH, X4's Xolremdi for WHIM syndrome, Day One's Ojemda for pediatric low-grade glioma, Geron's Rytelo for MDS, and ImmunityBio's Anktiva for NMIBC. These approvals mark significant milestones for these companies and address high unmet medical needs.

New data suggest AI, specifically metabolomics-based machine learning models, could revolutionize screening for metabolic dysfunction-associated steatohepatitis (MASH) candidates for resmetirom. Current noninvasive tests for noncirrhotic MASH with moderate-to-severe fibrosis have limitations, but machine learning models show potential to improve accuracy, sensitivity, and predictive ability. The study, led by Christos Mantzoros, assessed 28 biomarker-, imaging-, and algorithm-based noninvasive tests and developed machine learning models achieving higher AUC, sensitivity, and NPV. The addition of specific biomarkers to metabolomics-based models further enhanced performance.

FDA approved Madrigal's Rezdiffra for NASH, Italfarmaco's Duvyzat for DMD, and Cobenfy for schizophrenia. Genentech's Itovebi was approved for breast cancer. Lykos Therapeutics' MDMA for PTSD was rejected, leading to company reorganization.

Madrigal Pharmaceuticals' stock rose 82% in 12 months, driven by FDA approval of Rezdiffra for MASH, with over 6,800 patients and $62.2M in sales. The company aims to expand its market and is enrolling a trial for advanced liver scarring, potentially becoming the first to treat this condition. Madrigal's focus on MASH and network with specialist doctors could offer competitive advantages.

DelveInsight's report on Nonalcoholic Steatohepatitis (NASH) provides insights into historical and forecasted epidemiology, market trends, current treatment practices, emerging drugs, and market size from 2019 to 2032 in the United States, EU5, and Japan. Key companies and therapies in the NASH market are highlighted, including recent developments like Aligos Therapeutics' Phase 1 data presentation.

Madrigal Pharmaceuticals' Rezdiffra leads MASH treatment market, but opens opportunities for competitors like Inventiva Pharma's lanifibranor. GLP-1s, including Novo Nordisk's semaglutide and Eli Lilly's tirzepatide, show promise but face adherence issues. Inventiva sees potential in lanifibranor's anti-fibrotic and metabolic effects, aiming for market differentiation.

Life sciences and biopharma hedge funds saw gains in October, led by Casdin Capital's 16% surge. RTW Investments rose 13%, Madrigal Pharmaceuticals surged 22%, and Janus Henderson Biotech Innovation Fund climbed 2%. RA Capital Management gained 2%, while Avoro Capital Advisors and Soleus Capital added 1.6% and 2.1% respectively. Averill Partners lost 2.8%, and EcoR1 Capital dropped 3.4%, primarily due to Apellis Pharmaceuticals.

Trump's reelection may boost biotech innovation and M&A activity, potentially repealing the Inflation Reduction Act. Key roles in federal health agencies will influence drug pricing reforms. The Zacks Medical-Drugs industry, despite headwinds, shows promising trends with innovation in diabetes/obesity, inflammation, and neuroscience. Small drugmakers rely on collaboration partners and R&D funding, with notable stocks including Corcept Therapeutics, Madrigal Pharmaceuticals, Catalyst Pharmaceuticals, Theravance Biopharma, and Larimar Therapeutics.

Madrigal Pharmaceuticals (NASDAQ: MDGL) offers potential growth with its drug Rezdiffra, the first approved treatment for MASH, with a target market of 315,000 U.S. patients and a potential addressable market of 1.5 million. Rezdiffra sales reached $62.2 million in Q3 2024, and the company aims for EU approval in 2025. Despite competition from Novo Nordisk and Eli Lilly, Rezdiffra's tolerability gives it an edge, making Madrigal a promising long-term investment.