Krystal Biotech (KRYS 1.54%) and ARS Pharmaceuticals (SPRY 1.49%) have been gaining a lot of attention over the past year, thanks to the potential in their groundbreaking -- and for now, only -- commercial therapies.
In 2023, Krystal pioneered Vyjuvek, the first topical genetic therapy and first treatment approved by the Food and Drug Administration (FDA) for dystrophic epidermolysis bullosa, a rare skin disorder. In 2024, ARS Pharma got FDA approval for Neffy, a nasal spray that's the first epinephrine product for the treatment of anaphylaxis that is not administered by injection.
Both biotechs are working to expand the labels for their lead therapies while using their growing revenues to fund their pipelines. Like any biotech with only one commercialized treatment, each stock presents significant risks, but has considerable upside potential as well.
Krystal has a clear path to growth from its pipeline
Krystal Biotech turns viruses on their head, using their ability to spread through the human body to its advantage. The biotech develops gene therapies that use viral vectors -- basically, modified viruses -- to deliver genetic material to treat diseases, including cystic fibrosis, other lung diseases, and certain cancers.
NASDAQ: KRYS
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Krystal Biotech's KB707 candidate is intended to be a treatment for advanced or metastatic non-small cell lung cancer. The company recently got good news when the FDA gave KB707 a Regenerative Medicine Advanced Therapy designation, which could speed up its development and approval.
The company won't announce its fourth-quarter earnings until Feb. 17. However, it recently released preliminary numbers pointing to dramatically increased sales for Vyjuvek, a gel that treats dystrophic epidermolysis bullosa, a genetic disorder that causes blistering and scarring of the hands. The company said it expects to report annual sales of $388 million to $389 million, which would be a 34% increase at the midpoint.
While it didn't offer guidance for annual earnings per share (EPS), analysts predict it will report annual EPS of between $6.46 and $8.92, a jump of 156% over the prior year at the midpoint. That extra money will help it develop the other gene therapies in its pipeline.
The stock is up more than 10% year to date and up nearly 80% over the past year. It's trading at around $270 per share, so $300 would buy you just one share.
ARS Pharma has a nose for growth
ARS Pharma focuses on protecting patients from allergic reactions that could lead to anaphylaxis, a severe reaction in which the immune system releases a flood of chemicals that can cause the body to go into shock.
NASDAQ: SPRY
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The company recently got in trouble with the FDA over its advertising for Neffy. The agency said ARS Pharma's ads inaccurately made it seem that the drug completely negated the need for injections in emergency treatment of allergic reactions.
ARS Pharma's stock is down more than 24% both year to date and over the past 12 months. It's a riskier investment than Krystal Biotech because it isn't close to being profitable. However, the quick uptake in orders for its lead product shows it also has a lot of room for growth.
Through the first nine months of 2025, ARS Pharma reported $51.8 million in revenue, compared to only $568,000 over the same time frame in 2024. While it reported a nine-month loss of $1.32 per share, it has $288.2 million in cash on its books -- enough, the company said, to sustain it until it becomes profitable. Its potential is huge, as the global market size for epinephrine in 2025 was $2.48 billion, according to Fortune Business Insights.
ARS Pharma is looking to add indications for Neffy. In the meantime, its pipeline also contains ARS-2, an intranasal form of ephinephrine intended to be used to treat chronic urticaria, a skin disorder that can flare up from allergies.
Trading at $8.81 per share, you could buy 34 shares of ARS Pharma with $300.
