Eli Lilly has been on a roll. The big drugmaker won U.S. regulatory approval for weight loss drug Zepbound in November 2023. Over the last 12 months, Lilly's share price has skyrocketed more than 140%. Lilly has also claimed the mantle of the biggest healthcare company in the world based on market cap.
Don't worry if you feel as if you've missed out on Lilly's remarkable rise, though. Here are three healthcare stocks with big catalysts on the horizon.
1. Madrigal Pharmaceuticals
There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) for treating nonalcoholic steatohepatitis (NASH), a liver disease that affects an estimated 6% of Americans. That could soon change. The FDA set a PDUFA date of March 14, 2024, for deciding on accelerated approval for Madrigal Pharmaceuticals' (MDGL 0.90%) resmetirom in treating NASH patients with liver fibrosis.
NASDAQ: MDGL
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Although there's no guarantee that the FDA will approve resmetirom, Madrigal is optimistic. Chief Medical Officer Becky Taub said in the press release announcing the agency's acceptance of the approval application for the drug, "We believe that we have delivered a compelling data package to support the FDA's benefit-risk evaluation of resmetirom for the treatment of NASH with liver fibrosis." CEO Bill Sibold said in Madrigal's recent fourth-quarter update that the company is busy preparing for a potential U.S. launch.
Madrigal isn't limiting its hopes to the U.S. market. Just days ago, the company announced that its Marketing Authorization Application for resmetirom had been accepted by the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). This marks a key step toward potential approval for the NASH therapy in the European Union.
Estimates vary about how much money resmetirom will make if approved. However, SVB Securities thinks the drug could achieve peak annual sales of around $2.5 billion. Jefferies looks for resmetirom to eventually pull in $3 billion per year. With Madrigal's market cap currently hovering around $5 billion, this biotech stock could have a lot of room to run.
2. Regeneron Pharmaceuticals
Regeneron Pharmaceuticals (REGN +1.49%) might look like a big biopharmaceutical company that's past its prime. After delivering sizzling growth in the past, Regeneron's sales increased by a measly 1% year over year in the fourth quarter of 2023. But don't let this underwhelming performance fool you.
NASDAQ: REGN
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Sure, Regeneron's growth has been stymied by a sharp decline in demand for COVID-19 antibody therapy Ronapreve. However, excluding Ronapreve, the company's revenue jumped 14% year over year in Q4, with especially strong growth for cancer drug Libtayo, cardiovascular drug Praluent, and Zaire ebolavirus treatment Inmazeb.
Even better, Regeneron has multiple potential catalysts on the way. One of them is just around the corner. The FDA is expected to make an approval decision by March 31, 2024, on odronextamab in treating relapsed/refractory follicular lymphoma or diffuse large B-cell lymphoma. Another decision should be made only a few months later, with a PDUFA date of June 27, 2024, for Dupixent in treating COPD with type 2 inflammation. Hot on the heels of that news, the FDA should give its verdict on approval of linvoseltamab in treating relapsed/refractor multiple myeloma by Aug. 22, 2024.
More good news could be in store for Regeneron over the longer term. The company expects to advance Eylea HD into a pivotal study targeting retinal vein occlusion this summer. Around the same time, it plans to initiate a phase 2 study evaluating semaglutide (which is marketed by Novo Nordisk under the brand names Ozempic, Rybelsus, and Wegovy) in combination with trevogrumab in helping patients lose weight.
3. Vertex Pharmaceuticals
Vertex Pharmaceuticals (VRTX 0.73%) has already picked up key FDA and European approvals for Casgevy in treating sickle cell disease and transfusion-dependent beta-thalassemia. The big biotech appears to be in good shape to secure additional regulatory wins relatively soon.
NASDAQ: VRTX
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The EMA's CHMP recently gave a positive opinion for Vertex's cystic fibrosis (CF) therapy, Kalydeco, in treating infants ages one month and older. Kalydeco is already approved in Europe for treating CF patients ages four months and older. It seems likely that another approval expanding the drug's label could be on the way.
Vertex is also preparing to file for approvals in the U.S. and other countries of its newest CF therapy -- a triple combo featuring vanzacaftor, tezacaftor, and deutivacaftor -- in mid-2024. This combo could become the company's most profitable CF therapy yet if approved thanks to its impressive efficacy, convenience of administration, and low royalty burden.
That's not all. Vertex is planning to submit for FDA approval of VX-548 within the next few months as well. The non-opioid therapy should have tremendous commercial prospects in alleviating acute pain.
Vertex's pipeline features another promising program in pivotal development. Inaxaplin targets APOL1-mediated kidney disease (AMKD), which affects more patients worldwide than CF. There are currently no approved therapies for treating the underlying cause of AMKD.
