In­cyte nabs 2nd ever ap­proval ahead of sched­ule as FDA in­sists it can still re­view on time

Al­most a full year af­ter In­cyte broke off a trou­bled part­ner­ship with Eli Lil­ly to fo­cus on its own in­ter­nal en­gine, one of the drugs from that en­gine will en­ter the mar­ket.

Pemi­ga­tinib, now brand­ed as Pe­mazyre, ob­tained FDA ap­proval as a sec­ond-line treat­ment for a rare form of can­cer known as cholan­gio­car­ci­no­ma, or bile duct can­cer. For In­cyte, a biotech that rose to promi­nence on the strength of a sin­gle drug — Jakafi — and a slew of Big Phar­ma part­ner­ships, the ap­proval marks the first of what they hope will be a se­ries of in­ter­nal suc­cess­es and the first sign of a new phase for the com­pa­ny, al­beit one that is un­like­ly to make a huge com­mer­cial splash.

The ap­proval came a month be­fore the May 30 PDU­FA date and on the same day that a Seat­tle Ge­net­ics drug was cleared four months ahead of its date. FDA of­fi­cials, hav­ing been hound­ed for over a month with ques­tions about how the Covid-19 cri­sis would af­fect the re­view process for non-Covid drugs, were clear about what the reg­u­la­to­ry an­nounce­ments should sig­nal.

“This ap­proval demon­strates that while we con­tin­ue to fo­cus our ef­forts on ad­dress­ing the COVID-19 pan­dem­ic, the FDA re­mains com­mit­ted to the im­por­tant work of re­view­ing treat­ments for pa­tients with can­cer and oth­er se­ri­ous con­di­tions,” Richard Paz­dur, CDER’s top on­col­o­gy of­fi­cial, said in a state­ment.

For all its con­fi­dent state­ments and now mul­ti­ple ear­ly ap­provals, though, the agency ac­knowl­edged last week that with staff in­creas­ing­ly fo­cused on re­view­ing Covid-19 work, “it is pos­si­ble that we will not be able to sus­tain our cur­rent lev­el of per­for­mance in­def­i­nite­ly.”

The In­cyte drug had re­ceived both break­through ther­a­py sta­tus and pri­or­i­ty re­view. Af­ter ES­MO last year, Eval­u­ate pegged peak sales for the drug at $127 mil­lion across in­di­ca­tions.

Pem­ga­tinib is an in­hibitor of FGFR, or fi­brob­last growth fac­tor, the same ki­nase tar­get­ed by J&J’s blad­der can­cer drug erdafi­tinib. In Phase II tri­al da­ta re­leased last year, In­cyte showed the drug led to a pro­gres­sion-free sur­vival of 6.9 months and over­all sur­vival of 21.1 months in pa­tients with an FGFR2 mu­ta­tion. Two oth­er small­er co­horts with dif­fer­ent forms of FGFR lived for 6.7 months and 4.0 months, in line with the dis­ease’s stan­dard prog­no­sis. The fi­nal du­ra­tion of re­sponse re­sult was 9.1 months.

In­cyte hopes to soon show ef­fec­tive­ness in blad­der can­cer, test­ing the drug in sev­er­al Phase II tri­als for that in­di­ca­tion, along with tri­als for pa­tients with any tu­mors that have FGFR mu­ta­tions, ir­re­spec­tive of type. Op­er­at­ing in re­verse, J&J is now test­ing their drug against bile duct can­cer, while In­cyte push­es to show ef­fi­ca­cy as a first-line treat­ment. Agios al­so has an ex­per­i­men­tal bile duct treat­ment that tar­gets the en­zyme IDH1.

If the FDA keeps up its rapid pace, In­cyte could soon see an­oth­er big can­cer ap­proval. No­var­tis’ cap­ma­tinib, which the Swiss gi­ant li­censed from In­cyte back in 2009, has al­so been grant­ed break­through sta­tus and pri­or­i­ty re­view for MET-14 breast can­cer, with a PDU­FA date in Au­gust. That deal pro­vid­ed for over $500 mil­lion in mile­stones if No­var­tis suc­cess­ful­ly de­vel­oped the drug, plus 12-14% of sales.

The ap­proval could help shore up in­vestors’ testy faith in In­cyte’s abil­i­ty to ex­pand be­yond Jakafi. The com­pa­ny’s stock has swung wild­ly over the past year, par­tic­u­lar­ly af­ter the with­draw­al from the strug­gling Lil­ly drug baric­i­tinib and, in Jan­u­ary, a large Phase III fail­ure for a graft-ver­sus-host-dis­ease drug. It was up 4.54% from Thurs­day’s mar­ket close, to a flat $100.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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Doug Throckmorton speaks via video conference to the Senate Finance Committee, June 2, 2020 (Andrew Caballero-Reynolds, AP Images)

FDA de­fends its over­sight of for­eign drugs amid Sen­ate, GAO crit­i­cism

During a Senate Committee on Finance hearing Tuesday, officials from the FDA responded to criticism from senators and a new report from the Government Accountability Office (GAO) on its oversight of foreign drug manufacturers.

The hearing follows FDA’s move to halt most foreign inspections in March as a result of the coronavirus disease (COVID-19) pandemic.

Much of the criticism centered on the agency’s practice of giving foreign facilities advanced notice of inspections while most domestic surveillance inspections are unannounced, as well as US reliance on foreign drug manufacturing.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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