The FDA is get­ting its hands on a ‘hu­man em­u­la­tion sys­tem,’ and Em­u­late hopes they nev­er let go

The fu­ture for the or­gans-on-chips in­dus­try will de­pend to a large ex­tent on the FDA’s abil­i­ty to un­der­stand how it works as well as its prac­ti­cal, near-term po­ten­tial for re­plac­ing an­i­mals in pre­clin­i­cal re­search as the sys­tem evolves. And now reg­u­la­tors are about to get some hands-on ex­pe­ri­ence with a col­lab­o­ra­tive ef­fort to build a hu­man em­u­la­tion sys­tem in­side the agency.

The FDA has agreed to work with Em­u­late, a spin­off of the Wyss In­sti­tute which has been de­vel­op­ing and im­prov­ing on the in­dus­try stan­dard in the field, snag­ging part­ner­ships with the likes of Mer­ck and J&J. The work will be done un­der a Co­op­er­a­tive Re­search and De­vel­op­ment Agree­ment (CRA­DA), cre­at­ing a plat­form for tox test­ing for prod­ucts like foods, di­etary test­ing and cos­met­ics.

“The fo­cus is to as­sess the sys­tem’s ca­pa­bil­i­ty,” says Geral­dine Hamil­ton, pres­i­dent and CSO, “putting the chips, soft­ware and in­stru­men­ta­tion in the hands of FDA re­searchers, so they can bet­ter un­der­stand its po­ten­tial in sur­pass­ing an­i­mals for this kind of work.”

The agree­ment calls for a full-time com­mit­ment of five staffers at the FDA for three years, with a sim­i­lar com­mit­ment com­ing from Em­u­late.

This part­ner­ship isn’t about re­plac­ing an­i­mals for tox­i­col­o­gy stud­ies in pre­clin­i­cal drug re­search, but when I sug­gest­ed that would be the ul­ti­mate goal for where the col­lab­o­ra­tion could be head­ed, giv­ing the FDA more con­fi­dence in its ap­pli­ca­tions, she quick­ly con­curred.

“That is ex­act­ly the pur­pose,” says Hamil­ton.

The tech al­so isn’t ex­act­ly brand new to the FDA.

“Suzanne Fitz­patrick, se­nior ad­vi­sor for tox­i­col­o­gy at the FDA,” adds the CSO, “has been in­volved in col­lab­o­ra­tive re­search ef­forts since the time the tech­nol­o­gy was un­der de­vel­op­ment at the Wyss In­sti­tute at Har­vard.” But it is a big step for­ward.

“This is the start­ing point,” says Hamil­ton. “We are al­ready do­ing sim­i­lar ef­forts with in­dus­try part­ners, in the food in­dus­try, on very sim­i­lar col­lab­o­ra­tions.”

Fitz­patrick had this to say in a blog post to­day:

In some ways, sci­ence is like a recipe in that both can go through a num­ber of in­car­na­tions be­fore they work. There’s a lot of ex­per­i­ment­ing and tweak­ing, col­lab­o­rat­ing and com­par­ing. And that’s what we’ll be do­ing at FDA with the or­gans-on-chips re­search. Sci­ence is the foun­da­tion of FDA’s de­ci­sions but many peo­ple don’t re­al­ize how much sci­en­tif­ic re­search is con­duct­ed by the agency. We’re ex­cit­ed to be at the fore­front of this ground-break­ing re­search, which may one day be rou­tine­ly used to safe­guard pub­lic health.

Birthing a new tech­nol­o­gy is no quick or easy prospect. But Em­u­late is mak­ing mea­sur­able progress and plans on more high-pro­file part­ner­ships lat­er in the year.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Mi­rati's KRAS drug looks like the fa­vorite in colon can­cer with new da­ta, putting the pres­sure square on Am­gen

With Amgen already providing proof-of-concept for KRAS inhibitors with its sotorasib, Mirati Therapeutics is piecing together a follow-up effort in lung cancer with data it thinks are superior. But in colon cancer, where solo sotorasib has turned in a dud, Mirati may now have a strong case for superiority.

Mirati’s adagrasib, dosed solo or in combination with chemotherapy cetuximab, showed response rates grater than sotorasib solo  and as part of combination study in a similar patient population also revealed this week at #ESMO21. Mirati’s data were presented as part of a cohort update from the Phase II KRYSTAL-1 study testing adagrasib in a range of solid tumors harboring the KRAS-G12C mutation.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; 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.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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The best of the rest: High­lights from the be­low-the-fold pre­sen­ta­tions at #ES­MO21

This year’s ESMO Congress has had a major focus on Big Pharma drugs — most notably candidates from Merck and AstraZeneca — but there have also been updates from smaller biotechs with data looking to challenge the big-name drugmakers.

Today, we’re highlighting some of the data releases that flew under the radar at #ESMO21 — whether from early-stage drugs looking to make a mark or older stalwarts with interesting follow-up data.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.