John Maraganore, Alnylam CEO (Ryan Emberley/Getty Images for Klick Inc.)

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

“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. 

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

Endpoints News

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An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

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.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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With patent con­cerns loom­ing, Roche gets a new pri­or­i­ty re­view on block­buster IPF drug

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.

Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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Covid-19 roundup: Italy won­ders aloud if it can sue Pfiz­er for vac­cine short­falls; Flood, dead­ly fire threat­en As­traZeneca vac­cine plants

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.

Mike Grey, Plexium chairman (Horizon Therapeutics)

Plex­i­um adds in­dus­try vet Mike Grey to the brain trust with new in­vestor cash fund­ing its pro­tein degra­da­tion play

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.