Roger Tung, Concert Pharmaceuticals CEO (Concert)

Con­cert gets BTD for alope­cia drug, set­ting up a late-stage show­down with gi­ant ri­val Pfiz­er

Con­cert Phar­ma­ceu­ti­cals’ path to de­vel­op­ing a drug that treats alope­cia area­ta has been bumpy, but the phar­ma com­pa­ny scored a win Wednes­day.

The FDA grant­ed Con­cert a Break­through Ther­a­py Des­ig­na­tion (BTD) for its oral Janus ki­nase in­hibitor, named CTP-543, paving the way for a Phase III study of the drug to be­gin in the fourth quar­ter of 2020. The news fol­lows pos­i­tive Phase II re­sults from last Sep­tem­ber, which saw the drug meet its pri­ma­ry end­point in both 8 mg and 12 mg twice-dai­ly dos­es.

The news spurred a 14% spike in the biotech’s share $CNCE price.

There are cur­rent­ly no drugs on the mar­ket to treat alope­cia area­ta and Con­cert, whose drug blocks JAK1 and JAK2, has been com­pet­ing with Pfiz­er to see who can reach shelves first. CTP-543 is go­ing up against PF-06651600, which in­hibits JAK3, and PF-06700841, which tar­gets TYK2 and JAK1.

Though the FDA has of­fi­cial­ly en­dorsed CTP-543’s po­ten­tial with the BTD, the drug’s suc­cess is far from guar­an­teed.

JAK in­hibitors, which can al­so be used to treat rheuma­toid arthri­tis and ul­cer­a­tive col­i­tis, have come un­der heavy scruti­ny in re­cent years. Both the FDA and EMA have slapped warn­ings on and re­strict­ed larg­er dos­es of such in­hibitors made by Pfiz­er (Xel­janz) and Ab­b­Vie (Rin­voq) af­ter ob­serv­ing high­er rates of blood clots and death. Pfiz­er end­ed up drop­ping its high­er-dose treat­ment of Xel­janz al­to­geth­er, and Eli Lil­ly avoid­ed sell­ing high dos­es of its Olu­mi­ant drug as it be­came avail­able.

Con­cert it­self ran in­to some trou­ble a few years ago when the FDA placed a clin­i­cal hold on the Phase IIa study for CTP-543, even though the agency did not men­tion safe­ty con­cerns in its no­tice. Af­ter quick­ly fix­ing the is­sue, the Phase IIa study was com­plet­ed in late 2018, but showed Con­cert’s in­hibitors, de­spite hit­ting their main goals, did not stim­u­late hair growth as quick­ly as Pfiz­er’s in da­ta re­leased in late 2018. The fi­nal da­ta, re­leased the next year, showed the drug to be more com­pet­i­tive and sug­gest­ed it might even out­per­form Pfiz­er.

Pfiz­er is al­so re­search­ing JAK in­hibitors in teens and young adults, test­ing their ef­fi­ca­cy in treat­ments for atopic der­mati­tis. Al­though these stud­ies hit their pri­ma­ry goals, they too re­port­ed high­er rates of side ef­fects than the place­bo.

Alope­cia area­ta is an au­toim­mune dis­ease that af­fects around 700,000 peo­ple in the Unit­ed States. The dis­ease tricks the im­mune sys­tem in­to at­tack­ing a body’s own hair fol­li­cles, re­sult­ing in par­tial or com­plete loss of hair. Though the scalp is the most com­mon­ly af­fect­ed area, hair loss can oc­cur on any part of the body.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

CMS spent more than $18B in four years on ac­cel­er­at­ed ap­provals with in­com­plete con­fir­ma­to­ry tri­als, in­spec­tor gen­er­al finds

The battle over whether and how to reform the FDA’s accelerated approval pathway is heating up again, just as the Senate punted any talks until the lame duck session just before the end of the year.

On Thursday, HHS’ inspector general released a new report reiterating concerns, also noted recently by the FDA’s Oncology Center of Excellence, about delayed or slowed confirmatory trials that are necessary to prove that the accelerated approvals were worth their salt in the first place.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA (Sipa via AP Images)

Sen­ate pass­es bill to re­work an­i­mal test­ing re­quire­ments for drug de­vel­op­ers

The US Senate passed via unanimous consent on Thursday afternoon a bipartisan bill that would alter a federal mandate for animal testing on new drugs, but stops short of removing animal testing entirely.

Touted as a much-needed modernization of FDA’s rules, co-sponsor Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) have said the bill will stop lots of needless suffering of animals.

Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.