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@end­pointsnews.com.

– 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

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.