Most smaller biotech companies would love to follow in the footsteps of Vertex Pharmaceuticals (VRTX 0.68%). The company is the global leader in cystic fibrosis (CF) treatments. And these products help Vertex generate billions of dollars in annual revenue and profit. The stock also has rewarded investors with a gain of more than 120% over the past five years.
Let's turn to a smaller biotech that Vertex knows very well: CRISPR Therapeutics (CRSP 4.83%). The two companies are partners in a blood disorders program and recently submitted their treatment candidate for regulatory approval. If they win a nod, CRISPR may enter a big phase of growth.
But could CRISPR become the next Vertex? Let's find out.
Vertex's market-cap growth
First, to become the next Vertex, a company would have to grow to a similar market capitalization. Today, Vertex is worth $85 billion.
Vertex, of course, didn't start out at that size. About 10 years ago, its market value totaled less than $25 billion. And in the years preceding the approval of its first CF drug -- so around 2010 -- Vertex's market cap was about $7 billion.
Second, a company aiming to follow Vertex would have to sell at least one blockbuster product and maybe even become a leader in its industry. Vertex's CF blockbuster Trikafta generated more than $7 billion in sales last year. It also sells other CF treatments and has a full pipeline of candidates in other treatment areas. So a company that wants to fill Vertex's shoes should have a solid pipeline, too.
Now let's see if CRISPR might make it in terms of market value, products, and pipeline. I'm going to start with products and pipeline.
Today, CRISPR doesn't have any marketed products. But this may be about to change.
As mentioned above, CRISPR and Vertex have filed for the regulatory approval of exa-cel. They aim to win approval in adults with blood disorders beta thalassemia and sickle cell disease. They're also conducting phase 3 trials in children, so this could broaden the potential patient population.
Exa-cel is a one-time curative treatment using CRISPR's gene editing technology. This could be an important product for more than one reason.
Today there are limited treatment options for these particular blood disorders. So exa-cel could become a sought-after therapy and eventually a blockbuster. Exa-cel also demonstrates the performance of CRISPR's gene editing technique.
NASDAQ: CRSP
Key Data Points
A gene editing specialist
Speaking of gene editing, CRISPR uses this technology in its other pipeline programs. While Vertex is a specialist in CF, CRISPR could become known as a specialist in gene editing.
CRISPR also may generate revenue by licensing its technology to other companies. It's already done this with Vertex. Earlier this spring, CRISPR licensed its gene editing technology to Vertex for use in Vertex's type 1 diabetes program.
As for pipeline, CRISPR has several candidates in development. The biotech is studying gene editing candidates in immuno-oncology and regenerative medicine. One immuno-oncology candidate -- CTX-110 -- is even approaching the finish line. It's involved in a phase 2 trial that may support a regulatory submission.
If all goes well, CRISPR could have two products on the market -- exa-cel and CTX-110 -- within the next few years.
We've shown that CRISPR may bring blockbuster products to market, could become a gene-editing leader, and has a pipeline that might continue to generate new treatments. This sets it on the Vertex Pharmaceuticals path.
What about market value, though? Today, CRISPR is worth $5 billion. That's close to Vertex's value before it launched its CF drugs.
Over time, if successful drugs bring CRISPR revenue growth, it's easy to imagine the company's market value rising significantly. It's important to note that CRISPR's market cap reached $15 billion a few years ago -- and at that time, the company was further away from product commercialization.
All of this means CRISPR could become the next Vertex Pharmaceuticals -- a big biotech company with billion-dollar revenue that leads in its field. Of course, this won't happen overnight, and nothing is guaranteed.
Failure of a potential product always represents risk. But overall, the pieces to the puzzle are all there. They just have to fall into place.
