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

“We’ve had this decade-long her­itage of mak­ing these five-year goals, and this is the third in­stall­ment,” Maraganore told End­points News. “Each one has al­ways seemed like a tall moun­tain to climb every time we put them for­ward, but we end up climb­ing the moun­tain and then some, as we have in the past two, and we’re cer­tain­ly aim­ing to do that here.”

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 in the US and a fourth in Eu­rope, Al­ny­lam is aim­ing to add at least two drugs to its port­fo­lio 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.

If Al­ny­lam does man­age to ac­com­plish this lofty goal, Maraganore says it like­ly won’t be through in-li­cens­ing pro­grams. The com­pa­ny’s “or­gan­ic” pipeline af­fords them the lux­u­ry of get­ting to that point all on its own, he says.

“There’s no deal we need to do, ei­ther to grow mar­ket cap or from the stand­point of build­ing our busi­ness,” Maraganore said. “We’re one of these rare beasts out there in the in­dus­try I would say. Not a lot of us out there, maybe Re­gen­eron as an­oth­er ex­am­ple, that have an abil­i­ty of gen­er­at­ing sus­tain­able in­no­va­tion, and as a re­sult, we feel we can grow in­to that type of pro­file.”

This ar­ti­cle was up­dat­ed to in­clude com­ments from John Maraganore. 

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Seven years after the FDA first approved Esbriet, the blockbuster Roche IPF drug is getting an expedited review for a second indication.

On Thursday, the agency gave Esbriet priority review for unclassified interstitial lung diseases, or forms of pulmonary inflammation and scarring that don’t fit easily into the over 200 known types of ILD. The move comes 10 months after Esbriet received breakthrough designation and sets Roche up for a decision by May.

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.