Armed with promis­ing alope­cia PhII, Con­cert edges for­ward with mod­i­fied JAK in­hibitor — but safe­ty will still be key

Con­cert Phar­ma has scored a fresh round of pos­i­tive Phase II da­ta for its JAK in­hibitor amid an on­go­ing ri­val­ry with Pfiz­er, paving the way for a late-stage show­down in alope­cia area­ta. It’s al­so cau­tious­ly stak­ing a claim about safe­ty at a time the whole class is un­der a cloud of un­cer­tain­ty.

The glow­ing re­sults — in which both the 8 mg and 12 mg twice-dai­ly dos­es of CTP-543 hit the pri­ma­ry end­point of re­liev­ing hair loss — re­couped some en­thu­si­asm for the drug lost in its in­ter­im read­out. In­ves­ti­ga­tors re­port­ed that 58% of pa­tients in the 12 mg co­hort achieved at least 50% rel­a­tive re­duc­tion in the over­all Sever­i­ty of Alope­cia Tool (SALT) while 47% in the 8 mg group saw that im­prove­ment, com­pared to 9% for place­bo (p <0.001 in both cas­es).

Shares $CNCE climbed 2.48% to $10.32.

Con­cert is drop­ping the 4 mg dose from the up­com­ing Phase III study af­ter con­clud­ing its dif­fer­ence with place­bo is not sta­tis­ti­cal­ly sig­nif­i­cant.

The biotech is go­ing up against two Pfiz­er drugs for alope­cia area­ta, an au­toim­mune dis­or­der that can cause hair loss not just on the scalp but the beard, eye­brows, and oth­er ar­eas of the body for both men and women. CTP-543 in­hibits JAK1 and JAK2, while PF-06651600 tar­gets JAK3 and PF-06700841 blocks TYK2 and JAK1.

While JAK, or Janus ki­nase, are be­lieved to play an im­por­tant role in in­flam­ma­to­ry process­es via sig­nal­ing for over 50 cy­tokines and growth fac­tors, drugs in the class have re­cent­ly been im­pli­cat­ed for se­ri­ous side ef­fects. These con­cerns were in part ex­em­pli­fied by Pfiz­er’s own block­buster Xel­janz af­ter FDA and EMA reg­u­la­tors re­strict­ed its use due to a high­er risk of blood clots and death. And Ab­b­Vie’s FDA ap­proval for upadac­i­tinib (Rin­voq) days ago came with a black box warn­ing about in­fec­tions and throm­bo­sis.

In its press re­lease Con­cert high­light­ed that “no throm­boem­bol­ic events were re­port­ed dur­ing the tri­al,” and the on­ly se­ri­ous ad­verse event pos­si­bly re­lat­ed to treat­ment was a bac­te­r­i­al in­fec­tion on the face (fa­cial cel­luli­tis).

Oth­er side ef­fects in­clude headache, na­sopharyn­gi­tis, up­per res­pi­ra­to­ry tract in­fec­tion and ac­ne. The break­down:

James Cas­sel­la Con­cert

Sim­i­lar­ly, Pfiz­er has ob­served in­fec­tions, gas­troin­testi­nal and skin/sub­cu­ta­neous tis­sue is­sues in their Phase II alope­cia area­ta study.

“We are high­ly fo­cused on the need for an ef­fec­tive and safe treat­ment for alope­cia area­ta, and we plan to ad­vance CTP-543 in­to Phase 3 test­ing next year,” Con­cert chief de­vel­op­ment of­fi­cer James Cas­sel­la said in a state­ment.

But Con­cert will have more than one bat­tle to fight. Since CTP-543 is a deuter­at­ed ver­sion of Jakafi, In­cyte has filed — and won — a patent chal­lenge that it is now ap­peal­ing.

So­cial im­age: Alope­cia, Shut­ter­stock


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.

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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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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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.

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.

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.

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.