Amid ap­pli­ca­tion tsuna­mi, FDA's top cell and gene ther­a­py leader to re­tire in March

The FDA is in a bind and what it needs most right now is the ex­per­tise nec­es­sary to vet a mas­sive pipeline of cell and gene ther­a­pies com­ing down the pike.

Less than 30 of these ther­a­pies have so far been ap­proved by the FDA, and Pe­ter Marks, head of the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, told End­points News in an emailed state­ment that there are now 2,500 ap­pli­ca­tions pend­ing, and many of the meet­ings with spon­sors are falling be­hind.

“Cur­rent­ly, there are more than 1,300 ac­tive in­ves­ti­ga­tion­al new drug (IND) ap­pli­ca­tions for gene ther­a­pies, and over 1,200 ac­tive IND ap­pli­ca­tions for cell ther­a­pies,” Marks said.

Wil­son Bryan

Adding salt to the wounds of their em­ploy­ment needs (of at least 100 new po­si­tions), FDA’s di­rec­tor of the Of­fice of Tis­sues and Ad­vanced Ther­a­pies, now known as the Of­fice of Ther­a­peu­tic Prod­ucts, Wil­son Bryan, will re­tire in March, CBER’s chief of con­sumer af­fairs Paul Richards con­firmed yes­ter­day. The news was first re­port­ed yes­ter­day by the Pink Sheet’s Der­rick Gin­gery.

Bryan will de­part with more than 20 years of FDA ex­pe­ri­ence, af­ter com­ing to the FDA from the neu­rol­o­gy fac­ul­ty of the Uni­ver­si­ty of Texas South­west­ern Med­ical School for more than a decade. More re­cent­ly, he’s been up close and per­son­al with this grow­ing cell/gene ther­a­py pipeline, hav­ing served since 2016 as the of­fice di­rec­tor.

“The re­view­ers and project man­agers in cell and gene ther­a­pies are re­al­ly stretched,” he said at the Amer­i­can So­ci­ety of Gene and Cell Ther­a­py’s pol­i­cy sum­mit last Sep­tem­ber, not­ing that the meet­ing work­load, in par­tic­u­lar, has grown at a pace that far ex­ceeds per­son­nel in­creas­es, “and that cre­ates a stress­ful sit­u­a­tion.”

He al­so not­ed some key per­son­nel de­par­tures from OTAT, and the fact that the in­dus­try can quick­ly poach peo­ple from his of­fice for much high­er salaries.

“As OTAT has grown, the or­ga­ni­za­tion­al struc­ture has been a bit of a prob­lem,” Bryan said. “We have some branch­es that grew to where a branch chief was su­per­vis­ing over 20 peo­ple…I’m very wor­ried about turnover due to work­load and re­im­burse­ment due to salaries not be­ing com­pet­i­tive. We lost some re­al­ly good peo­ple be­cause they can go some­where else and make a lot of mon­ey.”

And this crunch on staff has been go­ing on for a while.

“We are meet­ing most PDU­FA UF [user fee] dead­lines, but at the ex­pense of staff work­ing con­sid­er­able over­time and fac­ing sig­nif­i­cant burnout,” not­ed 30-year FDA vet Christo­pher Jo­neck­is, CBER’s as­so­ciate di­rec­tor for re­view man­age­ment, in April 2021.

Even still, the lat­est user fee deal in­fus­es sig­nif­i­cant new sums of mon­ey (i.e. mil­lions of dol­lars) to try to re­cruit top new tal­ent in­to the cell/gene ther­a­py of­fice.

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.

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.

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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.

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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.

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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.

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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.

Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Mark Womack, BioCina CEO

Q&A: BioCi­na’s new CEO Mark Wom­ack on the CD­MO he says is 'worth trav­el­ing over'

A handful of CDMOs have made changes at the top over the past few weeks, including Genezen and Curia.

That also includes Australian CDMO BioCina, which announced last week that Mark Womack would be taking the helm. Womack previously served as chief business officer at AGC Biologics, CEO of Indian manufacturer Stelis Biopharma and most recently, CEO at CDMO KBI Biopharma and Selexis SA.

BioCina completed the takeover of a Pfizer manufacturing facility in Adelaide in 2021 and is now prepping for wider growth. Endpoints News sat down with Womack to discuss his new role, plans for the future, and how to compete in the wider CDMO market. This interview has been edited for brevity and clarity.

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.

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