Cara touts sub­group in failed atopic der­mati­tis tri­al, but stock craters

Cara Ther­a­peu­tics will join the grow­ing list of biotech to launch a Phase III de­spite fail­ing Phase II.

The itch-fo­cused com­pa­ny an­nounced Thurs­day that its cen­tral drug, a pill called Ko­r­su­va, failed to sig­nif­i­cant­ly al­le­vi­ate itch­ing in pa­tients with atopic der­mati­tis when com­pared with place­bo. The drug missed both the pri­ma­ry end­point, the change in av­er­age score on a stan­dard itch scale af­ter 12 weeks, and the key sec­ondary end­point: The pro­por­tion of pa­tients who im­proved by at least 4 points.

On the pri­ma­ry end­point, the study neared sta­tis­ti­cal sig­nif­i­cance, with a p-val­ue of 0.073 for the high dose and 0.111 for all dose. But it did not for the sec­ondary end­point, with p-val­ue of 0.55 and 0.59 for the mid and high dos­es.

The tri­al is one of sev­er­al Cara is run­ning for the drug across a va­ri­ety of dis­eases. The FDA is now eval­u­at­ing un­der pri­or­i­ty re­view an in­jectable form of Ko­r­su­va for dial­y­sis, a po­ten­tial­ly lu­cra­tive in­di­ca­tion that Vi­for spent $150 mil­lion up­front to com­mer­cial­ize in the US. Atopic der­mati­tis is one of two oth­er mid-stage ef­forts Cara is run­ning for Ko­r­su­va af­ter their pain pro­gram tanked, de­spite their best ef­forts to spin the da­ta.

Al­though they missed the two key end­points, Cara is ar­gu­ing that their re­sults in a sub-group of­fer a path to a piv­otal tri­al and even­tu­al ap­proval. In what the com­pa­ny refers to in their press re­lease as an­oth­er “Pri­ma­ry End­point,” the drug sig­nif­i­cant­ly im­proved symp­toms in a sub­group of pa­tients who had a more mild form of atopic der­mati­tis. This group, rep­re­sent­ing 257 pa­tients, or 64% of the 401-per­son study, saw sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in itch­ing symp­toms af­ter 12 weeks, with a p-val­ue of 0.036.

Joana Goncalves

Pa­tients in the milder group were al­so more like­ly to see an im­prove­ment by at least 4 points, with a p-val­ue of 0.03. In that group, 32% of pa­tients in the treat­ment group saw a 4-point im­prove­ment com­pared to 19% of place­bo pa­tients.

“We view this as a pos­i­tive out­come,” Cara CMO Joana Goncalves said on a con­fer­ence call “What we learned in the study is that [Ko­r­su­va] is most ef­fec­tive in the mild to mod­er­ate pa­tient pop­u­la­tion.”

Cara now plans to move for­ward with a Phase III for just the mild to mod­er­ate pa­tient pop­u­la­tion. In­vestors, how­ev­er, ap­peared un­con­vinced by the drug’s prospects, with the stock $CARA drop­ping 40%, from $25.67 t0 $15.67.

On Cara’s con­fer­ence call, an­a­lysts raised ques­tions about whether the ef­fect size they saw in the sub­group would be mean­ing­ful for pa­tients and whether they’d be able to charge as much as they would have for more se­vere pa­tients. They al­so point­ed out that, on one end­point, pa­tients who re­ceived a low dose per­formed bet­ter than pa­tients on the high dose — the op­po­site of what drug de­vel­op­ers gen­er­al­ly look for.

Derek Chalmers

“Does that make sense to you?” Stifel’s Annabel Samimy asked.

CEO Derek Chalmers said it was just an anom­aly. The in­di­vid­ual dose groups were sim­ply not large enough.

“We think that’s sim­ply a pow­er­ing is­sue,” said Chalmers.

The com­pa­ny al­so ar­gued that mild to mod­er­ate pa­tients still present a sig­nif­i­cant op­por­tu­ni­ty, as they can’t take in­jectable drugs like JAK in­hibitors and they aren’t suf­fi­cient­ly cov­ered by over-the-counter op­tions.

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.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? 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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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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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.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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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.