JPM: Alnylam CEO outlines aggressive plan to catapult into biotech's top 5

John Maraganore casual
Alnylam CEO John Maraganore, Ph.D., laid out his bold five-year vision for the biotech through 2025 at the annual J.P. Morgan healthcare conference. (Alnylam)

Alnylam CEO John Maraganore, Ph.D., officially has a new-and-lofty goal: Vault the RNAi-focused company into the top five biotechs by market cap—by 2025. 

What'll that take? Six or more products, more than 500,000 patients on its therapies, 20 clinical programs with 10 or more in late-stage development, and at least four INDs per year. Not to mention revenue growth of 40% during that same time, Maraganore said Monday at the virtual J.P. Morgan healthcare conference.

It's the next chapter for Alnylam, President and COO Yvonne Greenstreet, M.D., said Monday in an interview.The strategy is built on five P’s: patients,  products, pipeline, performance and profitability. Partners could be the sixth P; the company will partly depend on joint programs to hit that 500,000-patient goal, such as its Novartis-partnered cholesterol drug inclisiran.

RELATED: Alnylam's RNAi therapy Givlaari shows lasting effect one year into treatment, new data show

“We see a real opportunity to leverage our technology, not just for orphan diseases, but also for much larger diseases, and we’re already emboldened around the strategy by the success of the inclisiran program and now the [European] approval of Leqvio,” she said, referring to the EU brand name for the med, which hasn't yet won an FDA nod.

Along with the new programs, Alnylam is looking to extend existing products with new use trials for Onpattro, patisiran and lumasarin—and make profits doing so.

“Profitability allows us to own our own future, and there’s no need for us to tap the equity markets in order to access capital to fund opportunities in front of us,” she said.  

Alnylam's current portfolio of four therapies includes Onpattro and Gilvaari with $306 million and $55 million in 2020 revenue, respectively, as the company reported Monday. Onpatttro is approved to treat peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients, while Gilvaari’s nod is for the rare liver disease acute hepatic porphyria (AHP).

RELATED: Alnylam's Onpattro back in the game against Ionis, thanks to NICE U-turn

The company's most recent FDA approval came in November, for Oxlumo, the first medicine for primary hyperoxaluria type 1, or PH1, a severe rare disease that causes patients to suffer from recurrent kidney stones and often forces organ transplants. Oxlumo sales were $300,000 for 2020, Alnylam said Monday.

Meanwhile, the company is readying vutrisiran for an FDA filing after recent positive Phase 3 results. If approved, vutrisiran will become its fifth RNAi therapeutic to market.

The new plan, which Alnylam calls P5x25, continues the vision Alnylam began with its now wrapped Alnylam 2020 strategy.

CEO John Maraganore said in a statement that the company exceeded those 2020 goals and is “thrilled” with the debut of its new strategy, “aimed at Alnylam’s planned transition to a top 5 biotech in market capitalization.”

Editor's note: This story was updated with comments from Alnylam President and COO Yvonne Greenstreet.