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

 

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Covid-19 roundup: BAR­DA qui­et­ly pulls plug on IL-6 drugs; BioN­Tech and Fo­s­un be­gin mR­NA tri­al

IL-6 inhibitors showed some early promise in potentially treating Covid-19 patients, but recent trial flops have dashed hopes. Now it appears BARDA has officially pulled the plug.

The HHS office’s website has quietly updated to reflect that it is “no longer supporting product development” for Covid-19 in both Actemra (tocilizumab) and Kevzara (sarilumab), dealing a blow to the Roche and Regeneron/Sanofi drugs. This all but assures that IL-6 drugs repurposed to treat Covid-19 are essentially dead in the water.

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CF Foun­da­tion, Long­wood team on new in­cu­ba­tor for com­pa­nies with cut­ting-edge CF treat­ments

Nine months after launching a $500 million hunt for a cure for cystic fibrosis, the Cystic Fibrosis Foundation said it will use a portion of those funds to do something it has never done before: help launch new companies.

The CF Foundation, whose venture philanthropy efforts helped fund Vertex’s line of powerful CF drugs, is teaming with Longwood Fund to create a CF incubator. The incubator will identify new companies with platforms or technologies that can be applied in the rare genetic condition. The partners can then finance early development in exchange for a commitment from the companies to focus on applications in cystic fibrosis.

Kiersten Stead, John Hamer (DCVC Bio)

Deep tech, round 2: DCVC Bio bags $350M fund to chase the tip of the life sci­ence spear

It took one trip from San Francisco to Vancouver for Kiersten Stead and her DCVC Bio crew to feel confident about throwing their weight — and cash — behind AbCellera.

CEO Carl Hansen’s academic background and the potential of the platform, which combined machine vision and robotics with microfluidics, were promising. But the site visit sealed the Series A deal, where DCVC was the lead and only investor.

No­var­tis says Kym­ri­ah reach­es pri­ma­ry end­point in new PhII, al­though num­bers still to come

The race to develop CAR-T therapies has died down since Novartis’ Kymriah and Gilead’s Yescarta first crossed the finish line, though Tecartus also recently received approval. But the companies continue to expand their drugs’ applications, with Novartis preparing to conclude a new Phase II.

Interim data announced by the Swiss pharma show that Kymriah met its primary endpoint of complete response rate in treating patients with relapsed or refractory follicular lymphoma, the second-most common form of non-Hodgkin lymphoma. Based on preliminary trial findings, Kymriah had received RMAT designation from the FDA in April for r/r follicular lymphoma.

Ab­b­Vie shrugs off $134M cash deals, quit­ting a neu­ro R&D pact with Voy­ager Ther­a­peu­tics on vec­tor­ized an­ti­body treat­ments

It’s the end of the road for Voyager Therapeutics’ collaboration with AbbVie on tau and alpha-synuclein vectorized antibody development.

In two deals spanning the last two years, AbbVie dropped more than $134 million upfront for Voyager’s preclinical R&D of vectorized antibody treatments for diseases like Alzheimer’s and Parkinson’s. But Voyager says AbbVie is walking away now, without offering an explanation for why.

Mer­ck scoops up a PhII J&J dis­card in a bar­gain-base­ment deal. And this time they’re shoot­ing at NASH

When J&J turned to South Korea’s Hanmi for a GLP-1/glucagon dual receptor agonist obesity drug, the pharma giant paid $105 million in a cash upfront for the licensing rights and plotted a big clinical trial program to test it. A year ago, like a few of Hanmi’s big partners, J&J reviewed their trial data and walked away, handing it back.

Now Merck is stepping up to grab it for their NASH pipeline — and they got it a lot cheaper than J&J.