In this article, we will take a look at the 5 Most Profitable Biotech Stocks to Buy Now. For a deeper discussion and an expanded list, please see 10 Most Profitable Biotech Stocks to Buy Now.
5. Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR)
Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) ranks among the most profitable biotech stocks to buy now. Following comments on Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR)’s obesity and metabolic illness initiatives, TD Cowen reiterated its Buy rating on March 26. The firm pointed out that Wave Life Sciences published more data from Arrowhead’s ARO-INHBE program, demonstrating that, despite the higher dosage, monotherapy WVE-007 produced weight and fat reduction measures that fell short of investor expectations.
Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) stated that research is being done to provide hypotheses regarding the optimal locations for INHBE and ALK7 inhibition. In order to further assess this prospect, the company expanded the INHBE studies in response to promising signals in patients with Type 2 diabetes and in combination with tirzepatide, following data disclosed in January.
According to TD Cowen, these methods are most likely to be used in maintenance and supplementary settings, or in other specific indications such as MASH and Type 2 diabetes. The firm stated that it anticipates more information in the latter part of the year, including closely watched sHTG data in the third quarter.
Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) develops medicines for the treatment of intractable diseases in the US. Its pipeline primarily includes Plozasiran, Zodasiran, ARO-DIMER-PA, and ARO-PNPLA3.
4. Corcept Therapeutics Incorporated (NASDAQ:CORT)
Corcept Therapeutics Incorporated (NASDAQ:CORT) ranks among the most profitable biotech stocks to buy now. At the Society of Gynecologic Oncology Annual Meeting on April 10, Corcept Therapeutics Incorporated (NASDAQ:CORT) reported the conclusive total survival results from its Phase 3 ROSELLA study of Lifyorli in combination with nab-paclitaxel for platinum-resistant ovarian cancer.
The FDA had approved Lifyorli for the treatment of individuals with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have had up to three prior systemic therapies, at least one of which included bevacizumab.
Based on the results of the study, patients who received Lifyorli in addition to nab-paclitaxel had a 35% lower risk of dying than those who only received nab-paclitaxel. Additionally, compared to nab-paclitaxel monotherapy, the combination showed a 30% lower risk of disease progression.
Following FDA approval, Wolfe Research boosted Corcept Therapeutics Incorporated (NASDAQ:CORT) to Peerperform from Underperform. The firm sees the approval as eliminating a key downside prospect for the stock. Nonetheless, Wolfe questions the long-term viability of Corcept’s main Cushing’s disease franchise.
Corcept Therapeutics Incorporated (NASDAQ:CORT), incorporated in 1998, is a California-based biopharmaceutical company that discovers and develops solutions for serious endocrinologic, oncologic, metabolic, and neurologic disorders.
3. PTC Therapeutics, Inc. (NASDAQ:PTCT)
PTC Therapeutics, Inc. (NASDAQ:PTCT) ranks among the most profitable biotech stocks to buy now. On April 28, PTC Therapeutics, Inc. (NASDAQ:PTCT) announced positive outcomes from a 24-month interim assessment of its PIVOT-HD extension trial, which assessed votoplam for the treatment of Huntington’s disease. The trial found dose-dependent improvements in disease progression in people with Stage 2 Huntington’s disease.
According to the Composite Unified Huntington’s Disease Rating Scale, patients on the 10 mg dose showed a 52% slowdown in disease progression compared to a natural history sample, whilst those on the 5 mg dose exhibited a 28% slowdown.
Meanwhile, on April 9, Raymond James began coverage of PTC Therapeutics, Inc. (NASDAQ:PTCT), with an Outperform rating and a $108 price target. The firm emphasized the introduction of Sephience, which is billed as the first and only Phenylketonuria medication that combines oral dose with phenylalanine management and diet adaptability.
Analyst Tiago Fauth noted that the therapy has merits over traditional standard-of-care therapies for the hereditary condition.
PTC Therapeutics, Inc. (NASDAQ:PTCT) is a global biopharmaceutical company that discovers, develops, and commercializes innovative medicines for rare genetic disorders.
2. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) ranks among the most profitable biotech stocks to buy now. On April 10, Piper Sandler reaffirmed an Overweight rating on Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) with a $875 price target. The assessment followed an Integrated Oncology Day event at the Sylvester Cancer Center in Miami. The event included discussions on oncology therapies and ophthalmology with key experts from the Bascom Palmer Eye Institute.
One of the event’s main themes revolved around optimism about BCMA T-cell engagers transitioning into second-line treatment with MAJESTEC-3 results. Leading experts claimed they were not worried about sequencing CAR-T therapy following BCMA T-cell engagers.
Meanwhile, Cantor Fitzgerald maintained its Overweight rating and $800 price target for Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) on April 8. The firm predicted that near-term fianlimab data will continue to dominate first-quarter 2026 findings.
Cantor Fitzgerald isn’t expecting data before the Q1 2026 earnings call. For the quarter, the firm expects a lower-impact print, led by a broadly in-line and well-anticipated Eylea HD result and a minor shortfall for Dupixent in what it calls the notoriously difficult first quarter.
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is a prominent biopharmaceutical company that discovers, develops, and commercializes medicines for a variety of diseases, including cancer, eye disorders, and allergies.
1. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) ranks among the most profitable biotech stocks to buy now. On April 13, Bank of America reiterated Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) as a top large-cap biotech pick for 2026, with a $598 price objective. The firm stated the company’s cystic fibrosis franchise has the ability to generate cash flow to support pipeline growth. BofA also cited the rare kidney disease franchise as a major long-term growth engine, citing encouraging results for povetacicept in IgA nephropathy.
In a similar vein, Argus boosted Vertex’s price target to $520 on March 19, highlighting the improved performance of its cystic fibrosis brand, especially triple-combination medicines like Trikafta/Kaftrio and the expanding Alyftrek regimen.
The ratings come around a timeline where the United States Food and Drug Administration approved Vertex Pharmaceuticals’ expanded use of ALYFTREK for treating patients with cystic fibrosis aged 6 and up. The label extension was backed up by clinical and/or in vitro results from 564 variants responding to ALYFTREK and 521 variants responding to TRIKAFTA.
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a global biotechnology company. It develops and commercializes therapies for serious diseases, with a primary focus on cystic fibrosis and genetic disorders.
While we acknowledge the potential of VRTX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than VRTX and that has 100x upside potential, check out our report about the cheapest AI stock.
READ NEXT: Starter Stock Portfolio: 14 Safe Stocks to Buy Now and 40 Most Popular Stocks Among Hedge Funds Heading Into 2026.
Disclosure: None. Insider Monkey focuses on uncovering the best investment ideas of hedge funds and insiders. Please subscribe to our free daily e-newsletter to get the latest investment ideas from hedge funds’ investor letters by entering your email address below.
