Shares of Alnylam (ALNY 0.19%) recently dipped after Intellia (NTLA -1.37%) stole the spotlight with trial results for a revolutionary new gene therapy that could compete with Onpattro, Alnylam's top drug at the moment. It does look like Onpattro could be in for some serious competition, but that doesn't mean you should avoid shares of the biotech pioneer.
Alnylam's drug development engine is spewing out new drugs that interfere with the production of troublesome proteins so quickly that potential competition from Intellia probably isn't going to be an issue.
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Here are five of the most important reasons to buy Alnylam now.
1. Onpattro has a big lead
Intellia's lead candidate uses strands of messenger RNA (mRNA) that coax human liver cells into producing a CRISPR-Cas9 complex that permanently disables the transthyretin gene following a single administration. Intellia's potential new treatment for transthyretin amyloidosis with polyneuropathy (ATTR-PN) could have a big advantage over Onpattro, which is administered as an intravenous infusion once every three weeks at great expense.
Treatment with Onpattro costs around $450,000 per year -- and that's before you factor in expenses associated with frequent infusions. This is an expense that ATTR-PN patients will probably have to shoulder for at least a few more years. Intellia just reported data from the first six ATTR-PN patients in a phase 1 study designed to monitor patients for two years. That gives Onpattro lots of time to deliver rapidly rising revenue to the company's top line.
In the first quarter, Onpattro sales bounded 53% higher year over year to an annualized $408 million. Alnylam's drug employs small sequences of RNA that interfere with the production of a protein that carries around thyroid hormones and vitamin A called transthyretin. Interfering with transthyretin production is super useful for patients with severe cases of ATTR-PN, which is why the U.S. Food and Drug Administration (FDA) approved Onpattro in 2018 to treat this population.
2. Vutisiran's on the way
Early clinical trial data that drove Intellia's stock sky-high recently suggests the limited ATTR-PN population could have a new permanent treatment option in the foreseeable future. Luckily, Alnylam's working on a drug like Onpattro called vutisiran that requires no more than four simple subcutaneous injections per year.
The FDA began reviewing an application for vutisiran recently, and an approval decision is expected next April. Results of the 18-month Helios-A study showed that patients who received quarterly injections of vutisiran experienced outcomes in line with patients who received Onpattro infusions every three weeks.
3. Successful launches
Treating patients with ATTR-PN will be an important source of revenue for Alnylam for years to come, but it isn't the company's only revenue stream at the moment. In 2019, the company launched Givlaari, an RNA interference (RNAi) drug for patients with acute hepatic porphyria. Late last year, Alnylam launched its third RNAi drug, a treatment for primary hyperoxaluria called Oxlumo.
Sales of Alnylam's recent launches are rapidly reducing the company's reliance on the limited ATTR-PN population. Sales of Givlaari rocketed 368% higher year over year in the first quarter to reach an annualized $99 million. Oxlumo hit the ground running, generating over $9 million in sales during its first full quarter since earning FDA approval.
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4. High cholesterol
Alnylam expects to report between $610 million and $660 million in sales this year for Onpattro, Givlaari, and Oxlumo. The company's also about to receive a great deal of royalty revenue from a drug aimed at a much larger audience.
In Europe, Novartis (NVS 2.27%) earned approval for Leqvio, a drug that Alnylam discovered years ago for The Medicines Company. Treatment with just two doses of Leqvio per year cut low-density lipoprotein (LDL) cholesterol in half for patients who had stubbornly high LDL levels despite taking statins like Lipitor.
Unresolved facility inspection issues, not the drug's performance, prevented the FDA from granting Leqvio approval last December. In 2022, though, Novartis has a pretty good chance at launching Alnylam's fourth commercial-stage drug. With a potential user group of millions, instead of thousands, it could be the company's most important asset to date.
5. So much more to come
Four global new drug approvals since 2018 is an impressive achievement, but Alnylam isn't done giving investors reasons to cheer. In fact, vutisiran is one of eight potential new drugs in the company's clinical-stage pipeline.
Alnylam also boasts dozens of programs in pre-clinical stages with plans to move them into trials with real people at a rate of two to four per year. With plenty of new drugs barreling through the pipeline, this is a terrific biotech stock to buy now and hold for at least another decade.
