Vow­ing to help, Trump man­ages to spark an an­gry protest against poli­cies and a plan to re­vamp the FDA

End­points as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

 Pres­i­dent Don­ald J. Trump

Whether you love Trump or hate him, and you’ll find few peo­ple in be­tween those two sen­ti­ments, he’s made a huge im­pact on the bio­phar­ma world in the first few days as pres­i­dent. In many ways, it’s been a tox­ic af­fair right from the start.

First, Trump man­aged to raise quite a few bio­phar­ma hack­les with his ear­ly ex­ec­u­tive or­der ban­ning trav­el from 7 pre­dom­i­nant­ly Mus­lim coun­tries. True, none of these coun­tries are like­ly to reg­is­ter as big play­ers in sci­ence and drug de­vel­op­ment. But a poll of our read­ers, along with in­di­vid­ual com­ments from a large slate of ex­ec­u­tives, un­veiled a clear fear on the part of many green card hold­ers that their world could be turned up­side down in a heart­beat.

Fear and un­cer­tain­ty do not mix well with a fun­da­men­tal de­sire to re­cruit the best staff glob­al­ly.

The in­dus­try re­sponse to our sur­vey was ex­tra­or­di­nary, with 87% of more than 1,400 ex­ecs voic­ing their dis­ap­proval. That’s a big thumbs down from biotech.

  You say you want a rev­o­lu­tion?

Then came Trump’s sit-down with some very ap­pre­cia­tive phar­ma CEOs will­ing to nod their ap­proval as the pres­i­dent blast­ed the FDA and promised a whole­sale re­vi­sion of the reg­u­la­tions re­gard­ing drug de­vel­op­ment. No doubt, many crit­ics would have liked to have seen some de­fi­ance. The phar­ma ex­ecs, though, were on a lob­by­ing mis­sion. What they want, clear­ly, is to lim­it Trump’s ap­petite for slash­ing drug prices while gain­ing swift and im­me­di­ate tax re­lief, fat­ten­ing mar­gins and re­gain­ing bil­lions held over­seas for im­me­di­ate repa­tri­a­tion.

That’s all part of their job de­scrip­tion, so you shouldn’t act sur­prised when they go to DC to help make the sale.

As a group of biotech ex­ecs made crys­tal clear to me, though, FDA “re­form” isn’t need­ed or want­ed. Yes, there are con­tin­u­ing ef­forts to stream­line the reg­u­la­to­ry side of the busi­ness in PDU­FA VI, but leg­is­la­tion over the last few years has dra­mat­i­cal­ly al­tered many de­vel­op­ment time­lines. Trump’s loud protests to the con­trary, he’s ad­vo­cat­ing a rad­i­cal re­vi­sion at a time the in­dus­try is quite hap­py with where it is.

Re­form is a cov­er for sim­ply low­er­ing reg­u­la­to­ry stan­dards dra­mat­i­cal­ly. Once you do that, as just about any biotech CEO will tell you, you will cor­rupt a process that has be­come the gold stan­dard for the world. With­out an ob­jec­tive de­ci­sion that a drug’s ben­e­fits out­weigh ever present risks, you have chaos. And no one in this busi­ness with any in­tegri­ty wants to pit their good drugs against a tidal wave of fake cures that can be sold cheap.

 About those phar­ma jobs…

Trump likes to talk about bring­ing over­seas jobs back to the US. And the phar­ma rep­re­sen­ta­tives he met with were quick to oblige with some promis­es of their own. But Trump went look­ing for new jobs in the wrong place. Big Phar­ma has been zeal­ous in hold­ing the line on em­ploy­ment, which has fre­quent­ly called for sig­nif­i­cant cuts in the US.

As a quick re­view of the num­bers re­vealed, Eli Lil­ly, Mer­ck, Am­gen and oth­ers are bet­ter at cut­ting jobs than they are at adding them. And noth­ing Trump does or says is go­ing to get any­one re­al­ly en­thu­si­as­tic about putting the ax aside. Yes, we’ll get some lip ser­vice about man­u­fac­tur­ing and so on, but the new jobs in this in­dus­try are com­ing from biotech com­pa­nies, not Big Phar­ma.

Maybe that’s some­thing the pres­i­dent should think about be­fore he sets out to “help.”


Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; 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.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.