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.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Severin Schwan (AP Images)

Roche CEO Schwan puts 3-time Alzheimer's los­er gan­tenerum­ab back in the spot­light as FDA's con­tro­ver­sial Aduhelm OK in­spires block­buster fore­casts

Four years after Roche opted to resurrect its late-stage effort on their Alzheimer’s program for gantenerumab following a clear failure, Roche CEO Severin Schwan is signaling some fresh enthusiasm for its blockbuster prospects in the wake of the controversial Aduhelm OK.

Schwan told reporters that company execs are engaged in ongoing talks with the FDA as speculation continues to percolate around its chances based on biomarker data alone, now that regulators have established a precedent using an accelerated approval pathway process with no convincing evidence of efficacy in helping patients retain their cognitive skills. In Aduhelm’s case, the drug reduced the amount of amyloid in the brain and Biogen was able to win the accelerated approval on what the FDA called a reasonable expectation of success.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.