Axsome Therapeutics wowed investors when it soared more than 80% this year through August. Investors cheered Axsome's first steps in the world of product commercialization. The biotech company has given up a lot of that gain, but it continues to defy the bear market.
If you missed out on that major movement, don't worry. Other stocks may get a lift from excitement about new products. And one of them in particular makes the perfect stock to buy and hold. I'm talking about Vertex Pharmaceuticals (VRTX 1.73%). Let's find out why.
Leadership in cystic fibrosis
Vertex is best known for its leadership in the world of cystic fibrosis (CF) treatment. The company sells four CF drugs, and the latest is driving blockbuster revenue. Trikafta generated more than $5.6 billion in product revenue last year. And that resulted in a profit of $2.3 billion.
Vertex has what it takes to stay in this leadership position until at least the late 2030s. That's thanks to Trikafta -- and thanks to a candidate in phase 3 development. This candidate may even beat Trikafta. For example, its dosage is once a day instead of twice.
But what may drive the next wave of share gains at Vertex could be another treatment area altogether. Vertex is in the process of submitting its candidate for blood disorders to regulators. It aims to complete submissions in the U.K. and Europe by the end of the year, and complete its U.S. submission during the first quarter of next year.
What makes this candidate so special? Exa-cel, developed with partner CRISPR Therapeutics, is designed as a one-time curative treatment for beta thalassemia and sickle cell disease. Today, treatment options for these disorders are limited. And these are illnesses that continue throughout a person's lifetime. So the idea of a cure is big news.
Exa-cel could eventually become a blockbuster for Vertex if approved and if doctors prescribe the treatment for their patients. Vertex has taken on 60% of the program's costs, but it also gets to keep 60% of the profits. If all goes smoothly, exa-cel could lift Vertex's billion-dollar revenue higher -- and that could result in share price gains.
What's the stock done so far?
Vertex went through a rough period from late 2020 all the way through last year. The company announced two clinical trial failures in its alpha-1 antitrypsin deficiency program. Investors also worried about Vertex's ability to expand beyond CF. But as the company spoke more and more about positive exa-cel data, the shares rebounded.
This year, Vertex stock has climbed more than 30%. This leaves it trading at 23 times trailing-12-month earnings. It traded for more than double that in early 2020, when candidates like exa-cel and others offered much less visibility to investors.
Speaking of other candidates, Vertex is working on five programs beyond CF and blood disorders. Two that are particularly interesting are pain management and type 1 diabetes. They are in phase 2 and phase 1/2, respectively. Both of these indications are common -- and there aren't many treatment options out there. So, the opportunity for Vertex is huge.
What does all of this mean for you as an investor?
There is still time to invest in Axsome, even if you missed out on the major movement earlier this year.
But you may also want to consider getting in on Vertex right now. The stock hasn't experienced one big surge on exa-cel news. Maybe it won't in the future either. But if all goes well, exa-cel could deliver significant revenue. As a potential cure for blood disorders, it could become very popular with doctors and patients.