Let me start out by acknowledging that predicting the stocks in any industry that will deliver the best performance over the next decade is a crapshoot. You can take whatever odds there are against successfully doing so and probably double or triple them when it comes to picking the biotech stocks that will deliver the highest returns.
There are simply too many variables at play in the biotech world. Clinical trials can flop. The prospects for regulatory approvals can evaporate into thin air. Rivals can launch better drugs.
But there are factors we can look at that help improve the chances of choosing biotech stocks that are likely to succeed over the next 10 years. We can look at the company's lead pipeline candidates as well as earlier-stage long-shot candidates. We can look at the market potential for the indications those pipeline drugs target. And we can evaluate the risks the companies face.
So what are the best biotech stocks to buy for the next decade? Here are my top three picks.
1. Vertex Pharmaceuticals
If there's any biotech that has a clear path to deliver strong revenue and earnings growth, it's Vertex Pharmaceuticals (NASDAQ:VRTX). The company dominates the cystic fibrosis (CF) market with its four FDA-approved drugs. Sales for a couple of these drugs — Orkambi and Symdeko — are sure to increase significantly over the next few years thanks to reimbursement deals recently signed in important European markets.
Vertex's newest CF drug, Trikafta, hasn't won European regulatory approval yet, but I think it's a really safe bet that it will. Assuming I'm right, the drug will expand the biotech's addressable CF patient population by more than 50%. No other potential rivals are even close to being as far along as Vertex is. As I said, Vertex has a clear path to strong revenue and earnings growth — and this based on its CF franchise alone.
I fully expect Vertex will also have drugs on the market within the next decade that treat diseases other than CF. The company's pipeline includes experimental drugs targeting pain and several rare genetic diseases such as alpha-1 antitrypsin deficiency (AATD) and APOL1 mediated kidney diseases. Vertex is working with CRISPR Therapeutics to develop gene-editing therapies targeting rare blood diseases beta-thalassemia and sickle cell disease.
Then there's Vertex's swing for the fences. The company acquired Semma Therapeutics last year to scoop up Semma's program focused on curing type 1 diabetes (T1D). If Vertex develops a safe and effective cure for T1D, its tremendous success in CF could pale in comparison to what lies ahead.
2. bluebird bio
Until recently, bluebird bio (NASDAQ:BLUE) didn't have any approved products on the market. That changed with the biotech's January launch in Germany of Zynteglo (also known as LentiGlobin), a gene therapy that targets the treatment of transfusion-dependent beta-thalassemia (TDT), following securing European approval in June 2019. Zynteglo isn't approved in the U.S. yet, but Bluebird expects to file for FDA approval in the first half of this year.
The company also plans to evaluate Zynteglo in a phase 2/3 clinical study targeting sickle cell disease. And it has another gene therapy in late-stage testing, Lenti-D, for treating rare genetic disease cerebral adrenoleukodystrophy (CALD).
I especially like the prospects for Bluebird's experimental multiple myeloma drugs ide-cel and bb21217. Both drugs are licensed to Celgene, which was acquired by Bristol-Myers Squibb in November 2019, and should have blockbuster sales potential.
Bluebird's market cap currently stands around $5 billion, a level that gives it plenty of room to run, in my view. If Zynteglo, Lenti-D, ide-cel, and bb21217 achieve the success that I think they will, the biotech should be a huge winner over the next 10 years.
3. Editas Medicine
I admit that I'm stepping out on a limb with this pick. Editas Medicine‘s (NASDAQ:EDIT) lead pipeline candidate, EDIT-101, is only in early-stage clinical testing as a treatment for Leber congenital amaurosis type 10 (LCA10), the leading genetic cause of blindness. And because Editas has licensed rights to EDIT-101 to Allergan, the small biotech won't make all of the revenue the gene-editing therapy might generate if it's eventually approved.
But all of Editas' chips aren't riding on just EDIT-101. Editas and Allergan are taking a similar approach with EDIT-102 in treating another genetic eye disease, Usher syndrome type 2A, as they're taking with EDIT-101.
Like Vertex and CRISPR Therapeutics, Editas is also developing a CRISPR gene-editing therapy targeting beta-thalassemia and sickle cell disease. Editas hopes to file for FDA approval later this year to begin clinical testing of the therapy. The company's approach of editing the HBG1 and HBG2 genes could be more effective than CRISPR Therapeutics' approach of editing the BCL11Ae gene.
My rationale in choosing Editas, though, is that its CRISPR gene-editing platform holds promise for treating a wide range of diseases. The biotech is working with Bristol-Myers Squibb to develop off-the-shelf T cell therapies targeting cancer as well as developing its fully owned cancer cell therapies. And if its gene-editing approach is successful in treating eye diseases, the company thinks that it could potentially target heart, liver, neuromuscular, and nervous system diseases, too.