John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

'I want to be a grand­dad': Why John Maraganore left and where he (and Al­ny­lam) are go­ing

John Maraganore knew that peo­ple would be sur­prised when Al­ny­lam an­nounced his de­par­ture this morn­ing af­ter 19 years as CEO of the com­pa­ny. But he said it shouldn’t come as too much of a shock.

“Nine­teen years as a CEO lead­ing Al­ny­lam has been the great­est joy of my life, but 19 years has been a long time,” he said in an in­ter­view Thurs­day morn­ing. That’s “be­ing in the 24/7 op­er­a­tional mode of run­ning a com­pa­ny for 19 years.”

Maraganore, 59, start­ed as CEO when Phillip Sharp and a hand­ful of sci­en­tists and in­vestors de­cid­ed to launch a new com­pa­ny around RNAi, a new tech­nol­o­gy for si­lenc­ing genes that would go on to win the No­bel Prize. He re­mained CEO as the com­pa­ny faced tech­no­log­i­cal hur­dles, laid off em­ploy­ees, and lost the in­ter­est of in­vestors and large phar­mas.

But be­gin­ning about a decade ago, Maraganore out­lined a se­ries of five-year plans that, by 2018, re­sult­ed in the first ap­proved RNAi drug. An­oth­er ap­proval fol­lowed each suc­ces­sive year, and a fourth is like­ly to come in 2022.

In the process, he won a rare de­gree of trust from the once skep­ti­cal in­vestors, which ex­plains why the stock $AL­NY dropped 12.5% on news of Maraganore’s de­pa­ture, a $3 bil­lion swing for the big biotech.

So Maraganore will like­ly have his share of of­fers, though he’ll prob­a­bly turn many down. His CEO days are done, he said: Too much time, too much en­er­gy, too much com­mit­ment.

“My wife would punch me in the gut,” he said.

In­stead, he’ll drift for the ven­ture world, he said, even if he nev­er ful­ly joins any one firm. He be­lieves the hey­day of bio­med­ical in­no­va­tion is just be­gin­ning and he wants to ad­vise new com­pa­nies at the fore­front.

“It’s like a grand­fa­ther, right?  You get the ben­e­fit of lov­ing your grand­chil­dren, but not hav­ing to take care of them all the time,” Maraganore said. “I want to be a grand­dad.”

Yvonne Green­street

He leaves the keys to Al­ny­lam with Yvonne Green­street, who joined the biotech in 2016 af­ter stints at Pfiz­er and GSK and who was pro­mot­ed last year to pres­i­dent and COO.

Green­street, al­so 59, will be re­spon­si­ble for ex­e­cut­ing Al­ny­lam’s lat­est five-year plan. Un­veiled be­fore last year’s JPM, it promised to leave as a top five biotech by mar­ket cap by 2025, with six drugs on the mar­ket, 20 mol­e­cules in the clin­ic, and 500,000 pa­tients tak­ing RNAi ther­a­peu­tics glob­al­ly.

The new move won’t change that plan, Green­street said. Al­ny­lam had been ear­mark­ing her as a suc­ces­sor for “a bit of time,” Maraganore said, and she helped craft the lat­est plan.

Ex­e­cut­ing will re­quire com­pet­ing with oth­er new modal­i­ties. RNAi was one of the on­ly ge­net­ic tech­nolo­gies avail­able when Al­ny­lam was found­ed, along­side an­ti­sense. Now, thanks in part to some of the in­no­va­tions the com­pa­ny helped pi­o­neer, there are more than a half dozen, in­clud­ing mR­NA, gene ther­a­py, gene edit­ing.

This year, In­tel­lia, one of the first CRISPR com­pa­nies, showed proof-of-con­cept for a one-time treat­ment for amy­loi­do­sis, threat­en­ing Al­ny­lam’s biggest rev­enue gen­er­a­tor: On­pat­tro, an amy­loi­do­sis treat­ment that has to be giv­en every three weeks. The com­pa­ny’s next big drug is a new amy­loi­do­sis treat­ment that has to be giv­en quar­ter­ly.

Green­street and Maraganore are con­fi­dent, though, that Al­ny­lam will re­main com­pet­i­tive in any dis­ease that in­volves knock­ing down a gene. They’re work­ing on med­i­cines with even longer du­ra­tions, re­search­ing ways to de­liv­er in­to the ner­vous sys­tem with Re­gen­eron, and be­gin­ning to push their own drugs in more com­mon dis­or­ders.

“I’m go­ing to con­tin­ue ex­e­cut­ing with­out los­ing any mo­men­tum,” Green­street said in an in­ter­view. “We’ve on­ly just re­al­ly start­ed see­ing the pos­si­ble is well be able to de­liv­er to pa­tients with RNAi.”

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with Rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.