The End­points 100 CEO Sur­vey: Ex­ecs of­fer a big thumbs up for Got­tlieb and some sug­ges­tions for mak­ing the FDA a bet­ter agency

Scott Got­tlieb, Com­mis­sion­er of Food and Drugs, ap­pears be­fore a US Sen­ate sub­com­mit­tee to re­view the FDA’s 2018 bud­get re­quest on Tues­day, June 20, 2017

AP Im­ages / End­points News

About the worst thing that any­body in our biotech ex­ec­u­tive sur­vey group has to say about FDA Com­mis­sion­er Scott Got­tlieb right now is that it’s still ear­ly in the game for him and they’re wait­ing to see how things de­vel­op be­fore they of­fer a firm opin­ion.

Af­ter that, it’s all good.

“Look­ing across the range of ad­min­is­tra­tion nom­i­nees, I’d say FDA did ex­cep­tion­al­ly well,” writes Jeff Jonker, pres­i­dent of NGM Bio­phar­ma­ceu­ti­cals, in a note that res­onat­ed through­out the re­spons­es we col­lect­ed. “To his cred­it, Dr. Got­tlieb seems ac­tive­ly com­mit­ted to the mis­sion of the agency.”

Al­to­geth­er 74 of 88 ex­ecs in our End­points sur­vey group — 84% — gave Got­tlieb their thumbs up. The rest are stick­ing to the side­lines with a neu­tral po­si­tion — for now.

There were 0 votes crit­i­ciz­ing the pick.

The re­spons­es for our most re­cent check­up on the biotech pulse range from an ap­pre­ci­a­tion of Got­tlieb’s pro­fes­sion­al ex­pe­ri­ence to his will­ing­ness to try new things to im­prove reg­u­la­to­ry over­sight of the drug de­vel­op­ment process. And for a group of ex­ecs who are, by and large, up­set and dis­ap­prov­ing of his boss — Pres­i­dent Don­ald Trump — Got­tlieb is quick to win ku­dos for his ap­pre­ci­a­tion of sci­ence and an in­sid­er’s un­der­stand­ing of how the agency works.

Says one: “Got­tlieb is one of the few mem­bers of this ad­min­is­tra­tion that ap­pears to un­der­stand and ap­pre­ci­ate sci­ence.”

There’s al­so a clear echo from an ear­li­er de­bate over who should run the FDA, as biotech ex­ecs gen­er­al­ly heaped abuse on Jim O’Neill and his no­tion that the ef­fi­ca­cy bar should lie on the ground. You could see that in one as­sess­ment — “ex­pe­ri­enced adult” — of Got­tlieb’s strengths.

The De­moc­rats’ ma­jor ob­jec­tion to Got­tlieb that his pre­vi­ous work as an in­vestor and con­sul­tant in the field left him hope­less­ly con­flict­ed is clear­ly not shared by biotech ex­ecs — of both po­lit­i­cal per­sua­sions.

Got­tlieb’s ear­ly pop­u­lar­i­ty is just one of sev­er­al is­sues we cov­ered in our lat­est in­dus­try sur­vey. By and large this group of ex­ecs is up­beat about the fu­ture of the busi­ness and their com­pa­nies, look­ing to make more hires in an era of fair­ly ready ac­cess to cap­i­tal. The lack of M&A so far this year, though, has some grow­ing con­cerns about val­u­a­tions, pric­ing re­mains a big is­sue with no easy so­lu­tions, and while Got­tlieb is draw­ing pos­i­tive re­views, there are al­so a num­ber of sug­ges­tions on how the FDA could do bet­ter.

Want a bet­ter FDA? Hire ex­cel­lent staffers. Lots of them

Got­tlieb clear­ly has a man­date from the pres­i­dent to im­prove the FDA and find a bet­ter way to hur­ry along the reg­u­la­to­ry process, in all ar­eas out­side of on­col­o­gy, which is wide­ly viewed by the in­dus­try — and Got­tlieb — as the mod­el for all oth­er di­vi­sions in the agency. So we asked the mem­bers of the E100 what they would do dif­fer­ent­ly at the FDA, and what they would be sure to leave un­mo­lest­ed.

Arie Bellde­grun

Staffing is clear­ly an is­sue for a num­ber of the ex­ecs who com­plet­ed the sur­vey. The FDA is un­der­staffed in key ar­eas, and most want to see gener­ics pushed through faster. Biotech ex­ecs want more reg­u­la­tors, and they want bet­ter staff with more ex­per­tise in their field.

That top­ic came up re­peat­ed­ly, in dif­fer­ent ways.

Arie Bellde­grun, the CEO of Kite, now in the last leg of its reg­u­la­to­ry re­view for a ground­break­ing CAR-T treat­ment, says it would be best to leave the re­view process alone. Pos­i­tive change, he adds, will come from ex­pe­ri­enced, high­ly mo­ti­vat­ed staffers at the FDA. And he sug­gests tak­ing a leaf from the new Chi­nese play­book.


Fight for more ex­pe­ri­enced and high qual­i­ty man­pow­er to do the work and com­plete it in a time­ly man­ner. Keep your ex­am­in­ers sat­is­fied and feel­ing ac­com­plished. Learn from the Chi­nese FDA and CDE!! What a change there!! I have just re­turned from a very im­pres­sive vis­it and meet­ings with the leg­is­la­tors and ex­am­in­ers in Bei­jing. They are ex­cit­ed, en­gaged, full of en­er­gy, and have sig­nif­i­cant­ly ex­pand­ed their pro­fes­sion­al work­force. No com­plaints on be­ing short of staff or lack of great can­di­dates ( many of them trained in the US!). High­ly rec­om­mend a vis­it with them AS­AP.

There were sev­er­al bit­ing re­marks about the eteplirsen con­tro­ver­sy, with lin­ger­ing anger ev­i­dent that the agency chose to make an ex­cep­tion for Sarep­ta and its Duchenne drug. How­ev­er, it was al­so clear that there’s plen­ty of sup­port for re­spon­si­ble use of bio­mark­ers and sur­ro­gate end­points in com­ing up with faster ap­provals for many kinds of drugs, in­clud­ing those head­ed to the rare dis­ease field.

Nan­cy Si­mon­ian

Nan­cy Si­mon­ian, the CEO at Sy­ros Phar­ma­ceu­ti­cals, had this to say:

Keep the fo­cus on mov­ing in­no­v­a­tive prod­ucts swift­ly for­ward for dis­eases with high un­met need. Pro­vide more con­sis­tent guid­ance on bio­mark­er dri­ven strate­gies. Pro­mote ear­li­er ex­plo­ration of com­bi­na­tions and adap­tive tri­al de­signs to al­low more ef­fi­cient de­vel­op­ment. En­hance the per­spec­tive of the pa­tient in de­ci­sions on what con­sti­tutes clin­i­cal ben­e­fit and risk/re­ward.

From re­view­ing the sur­vey and mod­er­at­ing a re­cent con­ver­sa­tion on FDA re­form in Cam­bridge, MA, I’ll em­pha­size that there’s con­sid­er­able sup­port for mak­ing cer­tain that pa­tient per­spec­tives are re­spon­si­bly in­clud­ed in the re­view process, in­clud­ing a big­ger fo­cus on pa­tient re­port­ed out­comes in clin­i­cal tri­als.

Al­so, biotech R&D is in­creas­ing­ly less and less like­ly to be neat­ly di­vid­ed along the di­vi­sion­al bound­aries in­side the agency. That is caus­ing some added frus­tra­tion. Here’s one CEO, anony­mous­ly:

Change the di­vi­sion­al frame­work. It is an old fash­ioned way to di­vide a world that is in­creas­ing­ly mol­e­c­u­lar.

And don’t for­get about doc­tors and pa­tients, urges sci­en­tist and biotech in­vestor/en­tre­pre­neur Greg Ver­dine.

Em­pha­size the ed­u­ca­tion­al mis­sion of the FDA to help pa­tients and pre­scribers nav­i­gate the risk/ben­e­fit equa­tion for drugs.

We al­so asked CEOs about pub­lish­ing CRLs and found some sol­id back­ing for that.

Self re­straint on drug prices? It’s not enough

To be sure, there are some peo­ple in the in­dus­try who like the idea that com­pa­nies can rein in drug prices to help lance the boil on this is­sue. Up un­til a few days ago, the gen­er­al con­sen­sus was that the Trump ad­min­is­tra­tion would try some­thing de­fin­i­tive to slash drug prices. That is less and less like­ly, if the re­port­ing from Wash­ing­ton DC is ac­cu­rate. But self dis­ci­pline on pric­ing won’t be enough, ac­cord­ing to 63% of the sur­vey group.

“In­sin­cere,” re­marked one. You mean a cap of 10%? asks an­oth­er. That’s still way above in­fla­tion, say some. And the gen­er­al pub­lic won’t buy it now in any case, es­pe­cial­ly as long as a few price gougers ru­in pub­lic opin­ion about bio­phar­ma.

“The pub­lic’s per­cep­tion is now so neg­a­tive that more will be need­ed to turn it around. A few re­main­ing rot­ten ap­ples are enough to sour it for the rest.”

“I think it is a piece of the puz­zle, but on its own a com­mit­ment with­out demon­stra­ble change and ac­tion will not fun­da­men­tal­ly change the per­cep­tion. Need to keep the in­no­va­tion up and mak­ing drugs that have a big im­pact on pa­tients’ lives.”

Sev­er­al not­ed that the is­sue isn’t about drug pric­ing per se, but more about the stiff out-of-pock­et charges that are be­ing levied. As long as that re­mains in is­sue, man­u­fac­tur­ers can cut prices all they want with­out re­solv­ing the is­sue.

Sus­tain­able pric­ing? Hm­m­mm…

A sol­id ma­jor­i­ty of 60% be­lieve that the pric­ing on new drugs this year is sus­tain­able for all con­cerned. But a sig­nif­i­cant mi­nor­i­ty — 22% — said no.

Ac­cess to cap­i­tal

Close to 80% of our group in the E100 are bull­ish about ac­cess to cap­i­tal, with many rank­ing it good or ex­cel­lent.

“As an IO com­pa­ny with pos­i­tive hu­man da­ta, I feel I can get all the cap­i­tal I want.”

Oth­ers ex­pressed en­thu­si­asm about non-tra­di­tion­al cap­i­tal mak­ing it in­to the mar­ket, and sev­er­al re­marked that in this day and age in biotech, good com­pa­nies with good ideas have good ac­cess to cash.

But sev­er­al sound­ed a note of cau­tion. VCs ap­pear to be in­creas­ing­ly in­ter­est­ed on­ly in in­cu­bat­ing their own com­pa­nies, said a few, not look­ing out­side the ranks to in­vest in biotechs with lots of po­ten­tial.

And God help you if the da­ta sour — in­vestors ap­pear to be in­creas­ing­ly harsh in the face of a set­back, say some.

IPOs: Not bad, but not 2014

The biotech IPO mar­ket, which be­gan to show some fresh signs of live­li­ness af­ter the sur­vey went out, drew def­i­nite skep­ti­cism. 70% said the IPO mar­ket was av­er­age or on­ly fair. On­ly one in four thought it was good.

And there isn’t much be­lief that things will get much bet­ter in the short term, ac­cord­ing to 65%. The big boom of 2014 is def­i­nite­ly dead, said sev­er­al ex­ecs. And it’s not com­ing back soon.

Af­ter all, say some, gen­er­al­ist in­vestors are out of biotech and there are lots of fac­tors be­yond the con­trol of biotech that dic­tate in­vest­ment trends.

“The IPO mar­ket seems to be sup­port­ing high-qual­i­ty com­pa­nies, but I don’t see a re­turn to the 2014/2015 mar­ket.”

It will go up, Arc­turus CEO Joseph Payne writes: “The volatil­i­ty in­dex (VIX) has been con­sis­tent­ly sta­ble for the last 6 months (post elec­tion). This has been the my pre­ferred da­ta source as a pre­dic­tor of bub­bles pop­ping. In oth­er words, IPOs will be “the same” or “go up” as long as the VIX stays un­der 15.”

As I write this, the biotech sec­tor is ex­pe­ri­enc­ing a spike, and we’ll see how sus­tain­able it is by the time we ask this ques­tion again.

Hir­ing: Heck yes. But good tal­ent is hard to find

A big yes here. From the be­gin­ning of this sur­vey, which is now get­ting close to a year ago, these ex­ecs were in a hir­ing mode. In the lat­est round, 83% said they were adding staff in the sec­ond quar­ter; 86% will be adding staff lat­er in the year. No one is re­duc­ing staff. And that fits in with over­all in­dus­try pat­terns, where adding staff is now the name of the game.

“Both of the com­pa­nies I am build­ing are hir­ing ag­gres­sive­ly,” says one.

Brex­it, Schmex­it: “Good glob­al hir­ing in UK with no im­pact from Brex­it un­cer­tain­ties to date.”

“Mar­ket for tal­ent is get­ting very tight in San Fran­cis­co.”

“We’re hir­ing ag­gres­sive­ly but find­ing the right peo­ple is a chal­lenge with the sup­ply not keep­ing any­where near the de­mand,” notes Yu­val Co­hen.

Deal Watch: Show me the mon­ey

Talk to the av­er­age Big Phar­ma ex­ec, and you’ll get an ear­ful about how ex­pen­sive biotech val­u­a­tions are these days. Ask a biotech ex­ec, though, and things are just so-so.

More than half of the ex­ecs say that val­u­a­tions are on­ly av­er­age.

Why is that? De­spite big deals for Acte­lion and an ear­ly Ari­ad buy­out, M&A in biotech is in the slow lane. Tax re­form is still hangin­gin lim­bo and de­spite all the promis­es, we’re not see­ing any­thing like the ac­tiv­i­ty an­tic­i­pat­ed in 2017.

Right now it’s a case of “show me the mon­ey.” Once the deals and dol­lars (or eu­ros) flow, they’ll be ready to be­lieve again. In the mean­time, they’re on the side­lines with all the deal­mak­ers.

This is the third End­points 100 ex­ec­u­tive sur­vey. Pre­vi­ous sur­vey re­sults are avail­able here, and here. The End­points 100 is an in­vite-on­ly group of bio­phar­ma chief ex­ec­u­tives, polled once-a-quar­ter. 

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.