No big biotech stock performed better than Vertex Pharmaceuticals (NASDAQ:VRTX) did in 2017, with a market cap that more than doubled. CEO Jeff Leiden was certainly justified in highlighting Vertex's achievements when he spoke at the J.P. Morgan Healthcare Conference in San Francisco on Monday.
But Leiden didn't just focus on the past. He also laid out exactly what Vertex intends to accomplish in 2018, with 10 well-defined goals falling into three broad categories: Achieve the company's vision in cystic fibrosis (CF), expand the pipeline beyond CF, and build Vertex's financial strength. Let's take a closer look at those categories and points.
Achieve vision in cystic fibrosis (CF)
1. Report phase 2 data for triple combinations in CF patients. Vertex announced positive data from phase 1 and phase 2 studies of three different triple-drug combination treatments for CF in July 2017. The biotech advanced a couple of these triple combos into phase 2 -- VX-445 and VX-659, both in combination with tezacaftor and ivacaftor. Data from both phase 2 studies are expected in early 2018.
2. Initiate pivotal development of up to two triple-drug combos. Out of the four potential triple-drug combos being tested, Vertex plans to move forward with late-stage development of up to two of them. These triple combos are expected to be a huge catalyst for the biotech's future growth, potentially enabling Vertex to treat roughly 90% of CF patients.
3. Win approval for tezacaftor/ivacaftor combo. While the company moves ahead with its triple combos, Vertex has a final two-drug combo waiting on regulatory approval. The FDA should announce its decision on tezacaftor/ivacaftor by Feb. 28. Vertex has also submitted the combo for approval in Europe.
4. Advance additional next-generation correctors into development. In addition to its triple combos, Vertex has several next-generation correctors in pre-clinical testing. Leiden said the company intends to move some of these experimental drugs in to phase 1 clinical studies this year.
Expand the pipeline beyond CF
5. Advance at least one candidate into clinical development. Vertex dominates the CF market right now, but the biotech doesn't want to limit itself to just one indication. Leiden noted that what Vertex has learned in its efforts to treat CF help it in exploring therapies for other diseases. He highlighted several diseases the company is currently targeting, including adrenoleukodystrophy, alpha-1 antitrypsin deficiency, and polycystic kidney disease. Like CF, all are genetic diseases.
6. Begin clinical development of CRISPR-Cas9 treatment for two indications. Two indications that Leiden said Vertex would definitely advance to clinical stage are beta-thalassemia and sickle-cell disease. Vertex and partner CRISPR Therapeutics (NASDAQ:CRSP) are using CRISPR-Cas9, a powerful gene-editing approach, to treat both diseases. The two companies plan to begin a phase 1/2 trial evaluating CRISPR Therapeutics' CTX001 in treating beta-thalassemia in Europe and a phase 1/2 study of the therapy for treating sickle cell disease in the U.S.
Build financial strength
7. Significantly increase 2018 CF revenue. Leiden said Vertex should report 2017 revenue of $2.1 billion or so. Although he didn't provide guidance for this year, he indicated that the revenue trend of past years should continue into 2018 and beyond.
8. Secure reimbursement for Orkambi in more countries. Although Orkambi has already won European approval, reimbursement requires country-by-country negotiations. Vertex has secured reimbursement in several European nations, but the company is still in discussions with a key one -- France. Leiden didn't specifically say that the company would complete a deal with the French government this year, but it's without question a top priority for Vertex.
9. Manage expenses. While revenue has soared, Vertex has done a pretty good job of keeping research and development as well as sales, general, and administrative expenses under control. Expect continued growth in expenses, but Leiden said will focus on effectively managing the growth trend.
10. Increase operating margin and cash flow. Assuming Vertex does indeed keep expenses under control while revenue continues to grow, the company's operating margin will improve. So will its cash flow. In 2017, Vertex had an operating margin of around 24%. That should increase this year.
When Leiden presented at last year's J.P. Morgan conference, he outlined eight goals for 2017. Vertex achieved all of them. If the company can deliver on the 10 goals he detailed on Monday, Vertex stock should again generate impressive gains in 2018. I think it will -- and I consider Vertex one of the best big biotech stocks to buy right now.