John Maraganore makes a bold promise ahead of #JPM21 — aiming to take Alnylam into the ranks of biotech’s best

Alnylam CEO John Maraganore is a big fan of five-year plans, and with JPM21 kicking off this week, he revealed his company’s newest ambitious goal — become a “top 5 biotech” by 2025.

The objective is one of many Maraganore outlined in his strategy for the next five years, which the Cambridge, MA-based company announced Sunday. Among the other goals, of which not-so-coincidentally there are five, he hopes to:

  • Have more than 500,000 patients taking RNAi therapeutics globally
  • Market six or more products in rare and prevalent diseases
  • Expand Alnylam’s pipeline to more than 20 clinical programs, with 10 or more in late stages and 4 or more INDs per year
  • Achieve a compound annual growth rate greater than 40%
  • Achieve “sustainable non-GAAP profitability” sometime within that time frame

Maraganore is measuring Alnylam by market cap and is shooting to become the size of companies like Regeneron and Vertex — somewhere between $50 billion and $100 billion — rather than the big pharma giants such as Pfizer and Merck, a spokesperson told Endpoints News. Currently, Alnylam’s market cap sits at $17 billion, ranking it 12th-largest among eligible “biotech” companies, according to Yahoo! Finance.

Novo Nordisk tops the list as of Monday morning with a whopping $162.3 billion market cap, nearly three times the size of Vertex, which comes in second at $61.6 billion. Regeneron, Moderna and Alexion round out the top five, with the latter clearing $34.4 billion.

Getting there would mean Alnylam will need to at least double its market cap in the next half-decade, assuming no growth for any of the 11 companies ahead. Such size could also put Alnylam on the level of a company like Gilead ($79.3 billion market cap), which was cited by the spokesperson but does not appear on Yahoo’s list. But Maraganore is confident in getting near that level given Alnylam says it “exceeded” all metrics of its last five-year plan that ran from 2016 to 2020.

With three drugs already on the market, Alnylam is aiming to double its portfolio size over the next five years, and is off to a fast start already. Just last week, its open-label Phase III results for vutrisiran came back positive, showing reduced symptoms of polyneuropathy tied to transthyretin-mediated amyloidosis.

Alnylam is prepping for an early 2021 NDA with a potential launch slated for early 2022 should the FDA give it the thumbs up. It’s also ready to follow a US filing with applications in other countries, including Brazil and Japan, and then in the EU after obtaining results of the Phase III study’s 18-month follow-up.

Things have been moving quickly for Alnylam in recent months. The vutrisiran data came less than two months after the company got its third approval for Oxlumo (lumasiran) in primary hyperoxaluria type 1, coming in both the US and EU. Maraganore pegged a peak sales estimate of $500 million and said he thinks Alnylam is 18 to 24 months ahead of its rivals at Dicerna.

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