On this Rare Dis­ease Day, let's all com­mit to help­ing rec­og­nize the life and work of biotech gi­ant Hen­ri A. Ter­meer

Last May the Cam­bridge com­mu­ni­ty and en­tire bio­phar­ma in­dus­try suf­fered a tremen­dous loss with the un­ex­pect­ed pass­ing of Hen­ri A. Ter­meer. Hen­ri was a for­mer chair­man, pres­i­dent and CEO of Gen­zyme Cor­po­ra­tion for near­ly three decades pri­or to its ac­qui­si­tion by the French drug mak­er Sanofi. Re­tir­ing from Gen­zyme in 2011, af­ter his 28-year tenure, Hen­ri led the com­pa­ny’s growth from a small start-up of 20 to 12,000 em­ploy­ees glob­al­ly serv­ing pa­tients in more than 90 coun­tries all while es­tab­lish­ing Mass­a­chu­setts as the mec­ca of biotech. He was known for his ser­vice to the rare dis­ease com­mu­ni­ty and his un­sur­passed en­tre­pre­neur­ial lead­er­ship that spurred the rise of an in­dus­try ded­i­cat­ed to in­no­v­a­tive treat­ments for or­phan dis­eases. Hen­ri set a stan­dard, al­ways putting pa­tients first, and he forged the path for build­ing a sus­tain­able rare dis­ease busi­ness, with many – in­clud­ing Al­ny­lam – fol­low­ing his foot­steps.  He was a men­tor, a col­league, and a friend.

John Maraganore

To hon­or Hen­ri’s lega­cy and cel­e­brate his many con­tri­bu­tions to the thriv­ing in­dus­try that he helped build, a num­ber of in­dus­try, aca­d­e­m­ic and com­mu­ni­ty lead­ers have come to­geth­er to form the Hen­ri A. Ter­meer Trib­ute Com­mit­tee, which we have the priv­i­lege of co-chair­ing, work­ing close­ly with Be­lin­da and Adri­ana Ter­meer.

The fo­cus of every­thing Hen­ri did was to al­ways do what’s right for pa­tients. Fit­ting­ly, to­day on Rare Dis­ease Day, a time ded­i­cat­ed to rais­ing aware­ness for rare dis­eases and im­prov­ing ac­cess to treat­ment and med­ical rep­re­sen­ta­tion for in­di­vid­u­als with rare dis­eases and their fam­i­lies, we will be re-nam­ing a square in his hon­or as the “Hen­ri A. Ter­meer Square,” lo­cat­ed just op­po­site from the Gen­zyme Cen­ter in Kendall Square, Cam­bridge. To­day would have al­so been Hen­ri’s 72nd birth­day.

Robert Cough­lin

We have al­so com­mis­sioned renowned sculp­tor Pablo Ed­uar­do to cre­ate a life-size sculp­ture of Hen­ri to serve as a last­ing re­mem­brance of all he did for pa­tients, next gen­er­a­tion lead­ers and the com­mu­ni­ty – the sculp­ture will be in­stalled on the square in 2019.

Our cur­rent ef­forts and fu­ture ini­tia­tives fo­cused on the de­vel­op­ment of our biotech com­mu­ni­ty and on the men­tor­ship of fu­ture lead­ers are in­tend­ed to con­tin­ue Hen­ri’s re­mark­able lega­cy.

To learn more about these ef­forts, please vis­it ter­meer­tribute.org.

— John Maraganore and Robert Cough­lin


John Maraganore is the CEO of Al­ny­lam Phar­ma­ceu­ti­cals. Robert Cough­lin is the pres­i­dent and CEO of Mass­Bio.


Im­age: Feb. 16, 2011: Hen­ri Ter­meer, chief ex­ec­u­tive of­fi­cer of Gen­zyme, lis­tens dur­ing a news con­fer­ence an­nounc­ing the deal where France’s Sanofi-Aven­tis agreed to buy Ter­meer’s com­pa­ny, end­ing a nine-month pur­suit of Gen­zyme. Bloomberg via Get­ty Im­ages

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.