Walk­ing the walk, Al­ny­lam ap­points for­mer FDA head Mar­garet Ham­burg to the board

Right on the heels of an in­dus­try-wide no­tice from BIO to speed up gen­der di­ver­si­ty in the C-suite and board­room, Al­ny­lam is adding to its ros­ter with the ap­point­ment of for­mer FDA com­mis­sion­er Mar­garet Ham­burg to the board.

The let­ter, prompt­ed by a slate of con­tro­ver­sies over the lack of gen­der di­ver­si­ty and in­clu­sion dur­ing high-lev­el con­fer­ences such as JPM and BIO, was signed off by Al­ny­lam CEO John Maraganore, this year’s chair­man, along with Halozyme chief He­len Tor­ley and BIO chief Jim Green­wood.

Slat­ed to be­come a di­rec­tor on Jan­u­ary 10, 2019, Ham­burg takes the seat of John Clarke, who’s re­tir­ing from the board af­ter 16 years of ser­vice — in­clud­ing 13 years as the chair.

Ham­burg is the fourth woman on the cur­rent board of Al­ny­lam, join­ing Mar­sha Fanuc­cia, Amy Schul­man and Colleen Re­i­tan. As chair­man of in­dus­try group BIO, Al­ny­lam CEO John Maraganore has made di­ver­si­ty and in­clu­sion a pri­or­i­ty, with a goal of putting more women on cor­po­rate boards.

John Maraganore

“We are hon­ored to wel­come Peg­gy to our Board at a land­mark mo­ment for Al­ny­lam, hav­ing just re­cent­ly an­nounced the reg­u­la­to­ry ap­proval of the first RNAi ther­a­peu­tic,” said Maraganore.

Ham­burg’s most no­table stint is her time as FDA com­mis­sion­er dur­ing the Oba­ma years — 2009 to 2015 — where she served the sec­ond longest tenure. Pri­or to her lead role at the agency, she has spent time un­der the Clin­ton ad­min­is­tra­tion as as­sis­tant sec­re­tary for plan­ning and eval­u­a­tion at HHS as well as a gig as New York City’s health com­mis­sion­er. She is cur­rent­ly serv­ing as for­eign sec­re­tary of the Na­tion­al Acad­e­my of Med­i­cine, pres­i­dent-elect of AAAS and holds seats on a pletho­ra of oth­er boards, as well as a role on the Bill and Melin­da Gates Foun­da­tion Ad­vi­so­ry Com­mit­tee.

“I’m very pleased to be join­ing the Board — Al­ny­lam has kept a sus­tained fo­cus on cut­ting-edge bio­med­ical re­search, pi­o­neer­ing and lead­ing the field of RNAi ther­a­peu­tics to cre­ate a new class of med­i­cines,” said Ham­burg. “I look for­ward to work­ing with Al­ny­lam on in­no­v­a­tive ap­proach­es to help ex­pe­dite de­vel­op­ment of in­ves­ti­ga­tion­al ther­a­pies that ad­dress un­met med­ical needs.”


With con­tri­bu­tion from Am­ber Tong

Im­age: May­nard Clark; CC BY-SA 4.0

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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WIB22: Chas­ing af­ter ever-evolv­ing sci­ence takes a drug hunter across the pond

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Like many scientists, Fiona Marshall would tell you that she loved the natural world growing up — going to look at crabs running around the beach near her childhood home, pondering about the tides. But one thing about biology, in particular, stood out: It was constantly changing, and changing very quickly.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

WIB22: Suma Kr­ish­nan co-found­ed a com­pa­ny to de­vel­op a treat­ment for ‘the worst dis­ease you’ve nev­er heard of’

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

After spending time with patients who were diagnosed with what some call “the worst disease you’ve never heard of,” Suma Krishnan dedicated herself to finding a treatment.

Krishnan, who is the co-founder and chief operating officer of Krystal Biotech, spearheaded a gene therapy gel that showed promise in a late-stage clinical trial for dystrophic epidermolysis bullosa. The FDA is reviewing whether to approve the therapy and will decide by February.

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WIB22: She re­shaped mi­graine sci­ence and led key tri­als for new drugs, but Sheena Au­ro­ra isn’t done

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

For decades — even centuries — migraines were considered a disorder suffered by hysterical women.

Even when the father of modern-day migraine research pioneered a new understanding of migraine as a biological phenomenon having to do with blood vessels, he focused on explaining the condition for men but described women migraine sufferers, who make up the vast majority of the patient population, as inadequate wives and mothers.

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