An in­tro­duc­tion to the End­points 100

John Car­roll, Ed­i­tor

I’m well in­to a new project at End­points that I’d like to tell you about. It’s called the End­points 100. (Up­date: List of ex­ec­u­tives is at the bot­tom of this ar­ti­cle.)

For the 14 or so years that I’ve cov­ered biotech on a dai­ly ba­sis, I’ve seen some dra­mat­ic shifts in biotechs’ ac­cess to cap­i­tal, at­ti­tudes about reg­u­la­to­ry con­di­tions for new drug ap­provals (on both sides of the At­lantic), views on gen­der di­ver­si­ty, along with a whole new fo­cus on drug pric­ing and the fu­ture of the in­dus­try. Usu­al­ly, these trends and at­ti­tudes are all tracked in ret­ro­spec­tive analy­ses.

The End­points 100 is aim­ing at keep­ing a fin­ger on the pulse of the in­dus­try, to de­tect changes — pos­i­tive or neg­a­tive — as they oc­cur.

Over the last 10 days I’ve reached out to about 50 biotech CEOs who have agreed to par­tic­i­pate. By the end of the month, I’ll have the rest of the group in place; an in­vi­ta­tion-on­ly af­fair that will bring to­geth­er a broad cross sec­tion of top in­dus­try ex­ec­u­tives from small and large com­pa­nies. They’ll be ge­o­graph­i­cal­ly di­verse, though you can ex­pect a lot of East Coast/West Coast, UK and con­ti­nen­tal ex­ecs in the mix. The vast ma­jor­i­ty, though not all, will be CEOs.

From this group I plan to gath­er re­spons­es for a quar­ter­ly con­fi­dence sur­vey on biotech, of­fer­ing a col­lec­tive ex­pres­sion on the mood of the in­dus­try as we jour­ney through one of the most crit­i­cal pas­sages faced in drug de­vel­op­ment. It’s al­so a re­source for me to get some di­rect feed­back on End­points as my part­ner Ar­salan Arif and I craft this niche biotech news com­pa­ny over the com­ing year. And I’ll be ask­ing a month­ly poll ques­tion for the group.

There is one re­quire­ment: End­points 100 mem­bers need to be reg­u­lar End­points read­ers. That doesn’t mean read­ing every­thing from top to bot­tom (who does that with any pub­li­ca­tion?). But in or­der to par­tic­i­pate, you’ll need to be con­ver­sant with our cov­er­age. (Here’s our ded­i­cat­ed sub­scrip­tion page. There’s nev­er any cost to re­ceive End­points. We’re look­ing for in­volve­ment, not cash.)

If you’d like to nom­i­nate some­one, drop me a line. Just re­mem­ber, though, that many are called, few are cho­sen. It’s go­ing to take some bal­anc­ing to get the right group to­geth­er.

Look for the first sur­vey in Sep­tem­ber. And if you have any ques­tions, you can find me at: john@end­

– John Car­roll

Up­date: Here is the list of cur­rent End­points 100 ex­ec­u­tives.

An­drea Pfeifer AC Im­mune
Ron Co­hen Acor­da
Rick Orr Ad­ynxx
Mary Szela Aege­ri­on Phar­ma­ceu­ti­cals
David Schenkein Agios
Richard Pops Alk­er­mes
Alex­ey Mar­golin Al­lena Phar­ma­ceu­ti­cals
John Maraganore Al­ny­lam
John Crow­ley Am­i­cus
Mike Grey Am­plyx
Hamza Suria Anap­tys­Bio
Nor­bert Riedel Aptinyx
Joe Payne Arc­turus Ther­a­peu­tics
Tim Van Hauw­er­meiren Ar­genx
Bruce Booth At­las Ven­ture
Tito Ser­afi­ni Atre­ca
Na­tal­ie Holles Au­dentes Ther­a­peu­tics, Inc.
Jay Lichter Aval­on Ven­tures
Carmine N. Sten­gone Ave­las Bio­sciences, Inc.
Robert Con­nel­ly Ax­cel­la (Pronu­tria)
Howard Liang BeiGene
Jean-Jacques Bi­en­aimé Bio­Marin Phar­ma­ceu­ti­cal
Jef­frey Al­bers Blue­print Med­i­cines
An­drew J. Phillips C4 Ther­a­peu­tics
Gregg San­do Cell Med­ica
Prab­ha­vathi Fer­nan­des Cem­pra
Art Krieg Check­mate Phar­ma­ceu­ti­cals
Jeff Stein Cidara
Lau­ra Shawver Cleave Bio
Doug Williams Co­di­ak
Kei­th Dionne Con­stel­la­tion Phar­ma
Yu­val Co­hen Cor­bus Phar­ma
ROBERT I. BLUM Cy­to­ki­net­ics, Inc.
Sean Mc­Carthy Cy­tomX Ther­a­peu­tics
Steve Holtz­man Deci­bel Ther­a­peu­tics
Ryan Watts De­nali
Kevin Ju­dice DiCE Mol­e­cules
An­nal­isa Jenk­ins Di­men­sion Ther­a­peu­tics
Geral­dine Hamil­ton Em­u­late
Tim Mayleben Es­pe­ri­on
David Giljo­hann Ex­i­cure
Alexan­der Pas­teur F-Prime Cap­i­tal Part­ners
John Hau­rum F-star
Greg Ver­dine Fog Phar­ma
Sander Slootweg For­bion Cap­i­tal Part­ners
Steve Tre­gay For­ma Ther­a­peu­tics
Michael Pelli­ni Foun­da­tion Med­i­cine
Robert Gould Ful­crum Ther­a­peu­tics
Mark Vel­le­ca G1 Ther­a­peu­tics
Pier­lui­gi Parac­chi GENEN­TA Sci­ence
Chip Clark Geno­cea Bio­sciences
He­len Tor­ley Halozyme
Mal­colm Weir Hep­tares
Rich Hey­man Hey­man Biotech
Rus­sell Hern­don Hy­dra Bio­sciences
Eliot Forster Im­muno­core
Hervé Hop­penot In­cyte
Nes­san Berming­ham In­tel­lia Ther­a­puet­ics
Pe­ter Hecht Iron­wood
Kim Cobleigh Drap­kin Jounce Ther­a­peu­tics
Cameron Dur­rant Kalo­Bios
Arie Bellde­grun Kite Phar­ma
Troy Wil­son Ku­ra On­col­o­gy
Paul Sekhri Lyc­era Corp.
Kees Been Lyso­so­mal Ther­a­peu­tics
Denise Scots-Knight Mereo Bio­phar­ma
An­na Pro­topa­pas Mer­sana Ther­a­peu­tics
Ju­lia Owens Mil­len­do
Chuck Baum Mi­rati Ther­a­peu­tics
Stephane Ban­cel Mod­er­na
Praveen Tipir­neni Mor­phic Ther­a­peu­tic
Tas­sos Gi­anakakos MyoKar­dia
George P. Vla­suk Nav­i­tor Phar­ma­ceu­ti­cals, Inc.
David Mott NEA
Jeff Jonker NGM Bio
Don Nichol­son Nim­bus Ther­a­peu­tics
Jon Con­gle­ton Ni­valis Ther­a­peu­tics
Clif­ford J. Stocks On­coRe­sponse
Je­re­my Levin Ovid Ther­a­peu­tics
Bernard Coulie Pli­ant Ther­a­peu­tics
Kevin Bit­ter­man Po­laris Part­ners
Dale Schenk Prothena
John Bea­dle PsiOxus
Alan Auer­bach Puma
Dou­glas Tre­co Ra Phar­ma­ceu­ti­cals
Robert Ward Ra­dius
Olav Helle­bø ReNeu­ron Group plc
Robert Cof­fin Replimune
Clay Sie­gall Seat­tle Ge­net­ics
Glenn Ned­win Sec­ond Genome
Wern­er Cautreels Se­lec­ta
An­toine Pa­piernik Sofinno­va
Ar­mon Sharei SQZ Ther­a­peu­tics
Glyn Ed­wards Sum­mit Ther­a­peu­tics plc
Detlev Bin­iszkiewicz Sur­face On­col­o­gy
Kate Bing­ham SV Life Sci­ences
Nan­cy Si­mon­ian Sy­ros Phar­ma­ceu­ti­cals
Lon­nie Moul­der Tesaro
Mary Lynne Hed­ley Tesaro
Ed­uar­do Bra­vo Tigenix, SAU
Max­ine Gowen Treve­na
Niclas Stiern­holm Tril­li­um Ther­a­peu­tics
Bernat Olle Vedan­ta
Lau­rence Reid Warp Dri­ve Bio
Steve Yang WuXi AppTec
Rachel King Gly­coMimet­ics
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 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.