5 Biotech Stocks With The Biggest Upside

In this article, we discuss the 5 biotech stocks with the biggest upside. If you want to read about some more biotech stocks with the biggest upside, go directly to 10 Biotech Stocks With The Biggest Upside.

5. Veru Inc. (NASDAQ:VERU)

Number of Hedge Fund Holders: 13      

Veru Inc. (NASDAQ:VERU) is a biopharmaceutical company that focuses on developing medicines for the management of COVID-19, and breast and prostate cancers. On November 9, Veru Inc declared the results of its meeting with the US Food and Drug Administration’s Pulmonary-Allergy Drugs Advisory Committee, which reviewed sabizabulin for Emergency Use Authorization in hospitalized moderate to severe COVID-19 patients who are at high risk for acute respiratory distress syndrome.

On November 10, H.C. Wainwright analyst Yi Chen maintained a Buy rating on Veru Inc. (NASDAQ:VERU) stock and lowered the price target on Veru to $21 from $24, noting that FDA might deny the company’s EUA application and requested additional clinical data.

Among the hedge funds being tracked by Insider Monkey, Chicago-based investment firm Perceptive Advisors is a leading shareholder in Veru Inc. (NASDAQ:VERU) with 3.9 million shares worth more than $44.8 million.  

4. Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX)

Number of Hedge Fund Holders: 18     

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) is a commercial-stage biopharmaceutical company that focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. On September 29, Catalyst Pharmaceuticals, Inc. revealed that the US Food and Drug Administration has approved the supplemental New Drug Application to expand the indicated age range for FIRDAPSE (amifampridine) Tablets 10 mg to include pediatric patients six years of age and older for the treatment of Lambert-Eaton myasthenic syndrome.

On November 10, Piper Sandler analyst Joseph Catanzaro maintained an Overweight rating on Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) stock and raised the price target to $18 from $16, noting that the company reported third-quarter Firdapse revenues of $57.2 million, well ahead of consensus, and increased Firdapse revenue guidance to $205 million-$210 million.

At the end of the third quarter of 2022, 18 hedge funds in the database of Insider Monkey held stakes worth $189 million in Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX), compared to 16 in the preceding quarter worth $138.3 million.

3. Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY)

Number of Hedge Fund Holders: 27    

Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) is a commercial-stage pharmaceutical company that develops and commercializes therapies for patients with rare neurological disorders in the United States. In early November, the company posted positive topline results from phase 2 data related to Wakix (pitolisant), a drug approved for daytime sleepiness in those with narcolepsy. The results found that the daytime sleepiness drug may be effective for patients with Prader-Willi syndrome.

On November 8, Mizuho analyst Graig Suvannavejh maintained a Buy rating on Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) stock and raised the price target to $70 from $60, highlighting that the company reported positive third-quarter results.

At the end of the third quarter of 2022, 27 hedge funds in the database of Insider Monkey held stakes worth $338.7 million in Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY), compared to 20 in the previous quarter worth $402.7 million.

2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

Number of Hedge Fund Holders: 27  

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that engages in developing and commercializing therapies for treating cystic fibrosis. In early December, the company announced a four-year global research collaboration with Entrada Therapeutics targeting muscular disorder myotonic dystrophy type 1 (DM1). Per the deal, the former will take over the global development, manufacturing, and commercialization of the DM1 program ENTR-701, which is currently in late-stage pre-clinical studies, of the latter. Entrada will receive $224 million in return as an upfront payment and $26 million as an equity investment.

On November 17, Argus analyst Jasper Hellweg maintained a Buy rating on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) stock and raised the price target to $340 from $320, highlighting that the company continues to benefit from high sales of Trikafta, its pipeline progress, and partnerships with other organizations.

Among the hedge funds being tracked by Insider Monkey, New York-based investment firm Renaissance Technologies is a leading shareholder in Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) with 2.1 million shares worth more than $619 million. 

In its Q3 2022 investor letter, Artisan Partners, an asset management firm, highlighted a few stocks and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) was one of them. Here is what the fund said:

“Biotechnology company Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) dominates the market for the treatment of cystic fibrosis (CF) with limited competition. In addition to solid growth in CF revenues that has driven better-than-expected results, positive progress in its development pipeline has lifted shares. At the time of our Q2 2021 purchase, the stock was under pressure due to regulatory hurdles and Vertex’s decision not to pursue late-stage development of VX-864 after an unexpectedly unfavorable outcome. VX-864 is designed to treat alpha-1 antitrypsin deficiency (AATD), which is an inherited disorder with a strong correlation to pediatric liver disease. Irrespective of Vertex’s AATD pipeline, the company has nearly two decades of patent protection remaining for its CF franchise. Management maintains a healthy reserve of cash and is focusing on research and development. We believe near-term growth is likely to be driven by Vertex’s expanding geographic presence and expansion of medicines to lower age groups, with long-term gains arising from the company’s diversifying pipeline.”

1. BioMarin Pharmaceutical Inc. (NYSE:BMRN)

Number of Hedge Fund Holders: 62    

BioMarin Pharmaceuticals Inc. (NYSE:BMRN) develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. On November 23, BioMarin Pharmaceutical Inc. announced advancements in the US Food and Drug Administration (FDA) review of the Biologics License Application of ROCTAVIAN (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A.

On November 29, Jefferies analyst Akash Tewari maintained a Buy rating on BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) stock and raised the price target to $120 from $100, noting that 80 patients in Germany may begin taking Roctavian once it is available, which would indicate sales well above predicted by the Street for fourth quarter 2022 to second quarter 2023.

At the end of the third quarter of 2022, 62 hedge funds in the database of Insider Monkey held stakes worth $3.1 billion in BioMarin Pharmaceuticals Inc. (NYSE:BMRN), compared to 59 in the previous quarter worth $2.9 billion.

In its Q2 2021 investor letter, Carillon Tower Advisers, an asset management firm, highlighted a few stocks and BioMarin Pharmaceuticals Inc. (NYSE:BMRN) was one of them. Here is what the fund said:

“BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) rose as the company announced a new approval in Japan for its drug to treat achondroplasia (dwarfism), and investors may have become more optimistic regarding its therapy for hemophilia following a positive opinion from European drug regulators. The drug is pending approval and has been delayed by the U.S. Food and Drug Administration to collect more data.”

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