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3 Niche Healthcare Stocks Tackling Rare Diseases

The healthcare industry is set for growth due to advancements in targeted treatments and personalized care and the rapid evolution of medical technologies. Hence, investors might consider investing in niche healthcare stocks, Immunocore Holdings (IMCR), Catalyst Pharmaceuticals (CPRX), and BioCryst Pharmaceuticals (BCRX). Read more…

The healthcare market is evolving due to technological advancements and global health challenges, with increasing demand for innovative solutions driven by aging populations, chronic conditions, and the shift toward value-based care.

Given this backdrop, it could be wise to consider investing in fundamentally strong healthcare stocks, Immunocore Holdings plc (IMCR), Catalyst Pharmaceuticals, Inc. (CPRX), and BioCryst Pharmaceuticals, Inc. (BCRX), that tackle rare diseases.

The increasing research and development (R&D) in personalized medicine, numerous advancements in genetic engineering, and escalating demand for sustainable agriculture are propelling the biotech market. The global biotechnology market is expected to grow at a CAGR of 6.3% by 2033.

Additionally, the digital health market’s growth is driven by ongoing technological advancements in the form of artificial intelligence, the emergence of the Internet of Things, robotics in the healthcare sector, and remote monitoring devices. The global digital health market is expected to grow at a CAGR of 11.7% by 2034.

Given these favorable industry trends, let’s look at the fundamentals of three Biotech stocks, starting with number 3.

Stock #3: Immunocore Holdings plc (IMCR)

IMCR is a commercial-stage biotechnology company that engages in the development of immunotherapies for the treatment of cancer, infectious, and autoimmune diseases. The company offers KIMMTRAK for the treatment of patients with unresectable or metastatic uveal melanoma.

On December 3, 2024, IMCR announced that KIMMTRAK (tebentafusp) had been recommended for funding through the National Health Service (NHS) in England by the National Institute for Health and Care Excellence (NICE).

During the third quarter that ended on September 30, 2024, IMCR’s total revenue increased 23.7% year-over-year to $80.25 million. The company’s net income before income taxes of $6.09 million indicates a significant growth from the prior year’s quarter.

In addition, the company’s net income per share increased significantly year-over-year to $0.17.

Analysts expect IMCR’s revenue for the fourth quarter (ended December 2024) to grow 20.7% year-over-year to $84.72 million.

IMCR’s shares have gained 11.8% over the past month to close the last trading session at $32.61.

IMCR’s prospects are reflected in its POWR Ratings. The stock has an overall rating of B, equating to a Buy in our proprietary rating system. The POWR Ratings are calculated by considering 118 different factors, each weighted to an optimal degree.

The stock has a B grade for Growth, Value, and Quality. Within the Biotech industry, IMCR is ranked #23 out of 330 stocks. Click here to access additional BDX ratings for Sentiment, Momentum, and Stability.

Stock #2: Catalyst Pharmaceuticals, Inc. (CPRX)

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 January 21, 2025, CPRX reported that its sub-licensee in Japan, DyDo Pharma, Inc., had launched FIRDAPSE (amifampridine) Tablets 10 mg in Japan for the indication of improving muscle weakness in patients living with Lambert-Eaton myasthenic syndrome (LEMS).

For the third quarter of 2024, which ended September 30, CPRX’s net product revenue increased 23.2% year-over-year to $126.42 million. The company’s non-GAAP net income amounted to $71.08 million compared to a net loss of $25.64 million in the previous year’s quarter.

In addition, the company’s non-GAAP income per share came in at $0.60 compared to the net loss per share of $0.24 in the previous year’s quarter.

Street expects CPRX’s revenue and EPS for the fourth quarter (ended December 2024) to increase 21.3% and 17.2% year-over-year to $134.08 million and $0.34, respectively. Moreover, the company topped the consensus revenue and EPS estimates in three of the four trailing quarters, which is impressive.

CPRX’s stock has gained 8% over the past month to close the last trading session at $23.19.

CPRX’s bright prospects are reflected in its POWR Ratings. The stock has an overall rating of B, equating to a Buy in our proprietary rating system.

CPRX has an A grade for Sentiment and a B for Value and Quality. It is ranked #16 in the same industry.

In addition to the POWR Ratings we’ve stated above, we also have CPRX ratings for Momentum, Stability, and Growth. Get all CPRX ratings here.

Stock #1: BioCryst Pharmaceuticals, Inc. (BCRX)

BCRX is a biotechnology company that develops oral small-molecule and protein therapeutics to treat rare diseases. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names, and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema.

On November 18, 2025, BCRX announced that the Health Services Executive (HSE) in Ireland had recommended ORLADEYO (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older. With this recommendation, HAE patients in Ireland would have access to the first oral, once-daily therapy for the reduction of recurrent HAE attacks.

BCRX’s total revenues increased 35% year-over-year to $117.09 million for the fiscal 2024 third quarter that ended September 30, 2024.  Its income from operations also increased to $7.69 million compared to a loss of $11.92 million in the previous year’s quarter. In addition, as of September 30, 2024, BCRX’s cash, cash equivalents, and investments amounted to $349.44 million, while total assets came in at $491.25 million.

The consensus revenue estimate of $131.10 million for the fiscal year quarter (ending December 2024) represents a 40.4% increase year-over-year. The company has an impressive earnings surprise history; it surpassed the consensus revenue estimates in each of the trailing four quarters.

Over the past nine months, the stock has surged 84.3%, closing the last trading session at $7.87.

BCRX’s POWR Ratings reflect its bright outlook. The stock has an overall rating of B, which translates to a Buy in our proprietary rating system. 

BCRX has a B grade for Value, Quality, and Growth. It is ranked #12 in the same industry. Click here to see the additional ratings for BCRX (Momentum, Sentiment, and Stability).

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CPRX shares closed at $22.56 on Friday, down $-0.63 (-2.72%). Year-to-date, CPRX has gained 8.10%, versus a 2.69% rise in the benchmark S&P 500 index during the same period.



About the Author: Nidhi Agarwal

Nidhi is passionate about the capital market and wealth management, which led her to pursue a career as an investment analyst. She holds a bachelor's degree in finance and marketing and is pursuing the CFA program. Her fundamental approach to analyzing stocks helps investors identify the best investment opportunities.

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