Pfiz­er of­fers 3rd round of JAK da­ta, this time in kids. Can it com­pete?

Pfiz­er has re­leased the 3rd round of large and late-stage da­ta for their JAK in­hibitor and they say — well, they say pret­ty much what the first 2 stud­ies said, just in a new set of pa­tients.

The lat­est study, called JADE TEEN, was the first to study their JAK in­hibitor abroc­i­tinib ex­clu­sive­ly on chil­dren and ado­les­cents, test­ing it against place­bo in just un­der 300 atopic der­mati­tis pa­tients age 12 to 18. The youth pop­u­la­tion for AD is sig­nif­i­cant, with be­tween 15% and 20% of chil­dren af­fect­ed an­nu­al­ly by the in­flam­ma­to­ry con­di­tion world­wide.

As in the adult stud­ies — which al­lowed for pa­tients as young as 12 but had a mean age as high as 35 — the tri­al met its two pri­ma­ry end­points: a pair of met­rics that mea­sures how much the drug re­duces area of red­ness and itch­i­ness. But al­so like the adult stud­ies, the teens on the drug arm showed high­er rates of ad­verse ef­fects. The full de­tails weren’t dis­closed, but 62.8% of pa­tients on the high dose drug arm had side ef­fects, com­pared to 52.1% for place­bo.

In atopic der­mati­tis, Pfiz­er is go­ing up against an en­trenched com­peti­tor in Re­gen­eron and Sanofi’s Dupix­ent, an IL-4/IL-13 in­hibitor. In that strug­gle, the NJ phar­ma has a few things for them: It’s giv­en oral­ly as op­posed to sub­cu­ta­neous­ly, and over­all it has looked sim­i­lar in its abil­i­ty to treat the itch and pain that af­flict pa­tients.

“The ef­fect of abroc­i­tinib was fast on­set and could be ob­served as ear­ly as week 2, which con­curred our KOL’s im­pres­sion in pre­vi­ous in­ter­views,” Jef­feries’ Biren Amin wrote in a note to in­vestors af­ter the last round of abroc­i­tinib da­ta was pub­lished in JA­MA on June 3. “Our KOL con­sid­ers JA­Ki as an at­trac­tive op­tion for se­vere pts who are un­con­trolled by dupi.”

JAK in­hibitors, though, have broad­ly faced ques­tions about their safe­ty, ques­tions that have lim­it­ed the adop­tion of drugs that were long thought to have block­buster po­ten­tial. Ab­b­Vie’s JAK in­hibitor Rin­voq, for in­stance, got a black­box warn­ing af­ter stud­ies showed 1% of pa­tients got a se­ri­ous in­fec­tion, op­por­tunis­tic in­fec­tion, or her­pes zoster. In the JA­MA study, 1.9% of pa­tients on 100mg got a se­ri­ous in­fec­tion but none on 200mg. Two 200mg pa­tients, though, were in­fect­ed with her­pes.

There were al­so con­cerns about platelet re­duc­tion, with platelet counts drop­ping 26% for the 200mg dose and 19% for the 100mg group com­pared to es­sen­tial­ly no re­duc­tion on place­bo, though they even­tu­al­ly re­turned to nor­mal.

The lat­est top line read­out can’t an­swer those ques­tions. An­a­lysts will have to wait for the full da­ta. Still, with Sanofi and Re­gen­eron re­port­ing over $2 bil­lion in sales from Dupix­ent and atopic der­mati­tis af­fect­ing up to 20% of chil­dren and 3% adults, it’s a mar­ket that could have room for mul­ti­ple com­peti­tors.

Pfiz­er, which has gained a break­through ther­a­py des­ig­na­tion for the drug, is plan­ning to file for FDA ap­proval lat­er this year.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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