Sesen Bio tri­al marred by mis­con­duct — re­port; Bris­tol My­ers picks up de­vel­op­ment of AI out­fit's im­munol­o­gy drug

Sesen Bio said that it was not ex­pect­ing a CRL from the FDA for its blad­der can­cer can­di­date Vicineum. But the re­ject­ed fil­ing in­clud­ed thou­sands of vi­o­la­tions and in­ves­ti­ga­tor mis­con­duct, STAT News re­port­ed Wednes­day.

Thomas Can­nell

The 130-pa­tient study had more than 2,000 vi­o­la­tions, 215 of which were clas­si­fied as ma­jor, STAT said. In­de­pen­dent mon­i­tors al­so re­port­ed three in­ves­ti­ga­tors to the FDA for a “se­ri­ous non­com­pli­ance” that “placed sub­jects at risk of harm.” A pa­tient died in 2016 of liv­er fail­ure re­lat­ed to the drug, and two years lat­er, the com­pa­ny said that there were no drug-re­lat­ed deaths at a urol­o­gy con­fer­ence.

The com­pa­ny saw its stock crash, as it en­tered a halt at $6 per share last Fri­day and emerged bat­tered with a $0.86 price.

In June CEO Thomas Can­nell said that the drug was on its way to gen­er­at­ing be­tween $1 bil­lion and $3 bil­lion. Then Mon­day, he blamed “tox­ic” and crit­i­cal me­dia cov­er­age of the FDA’s ap­proval of Bio­gen’s Alzheimer’s treat­ment Aduhelm for the CRL. — Josh Sul­li­van

Bris­tol My­ers picks up de­vel­op­ment of AI out­fit’s im­munol­o­gy drug

Ru­pert Vessey

Bris­tol My­ers Squibb has opt­ed to pick up the de­vel­op­ment of an im­mune-mod­u­lat­ing drug as part of a re­search col­lab­o­ra­tion with UK-based AI dis­cov­ery out­fit Ex­sci­en­tia, the part­ners said Wednes­day.

Ex­sci­en­tia will re­ceive a $20 mil­lion mile­stone pay­ment as part of the pick up with ad­di­tion­al mile­stones and tiered roy­al­ties pos­si­ble. De­tails on the drug were scarce, but the part­ners said it tar­gets “a crit­i­cal im­muno­log­i­cal ki­nase.”

“We are pleased to in-li­cense our first drug can­di­date re­sult­ing from our strate­gic col­lab­o­ra­tion with Ex­sci­en­tia,” Ru­pert Vessey, Bris­tol My­ers’ pres­i­dent of ear­ly R&D said in a re­lease. “Ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing con­tin­ue to play im­por­tant roles in drug dis­cov­ery and Ex­sci­en­tia has de­liv­ered a promis­ing de­vel­op­ment can­di­date in the field of im­munol­o­gy. We look for­ward to our con­tin­ued col­lab­o­ra­tion and fur­ther ad­vanc­ing this can­di­date for the po­ten­tial ben­e­fit of pa­tients with un­met med­ical need.” — Kyle Blanken­ship

Were­wolf hops aboard the Keytru­da train with Mer­ck tri­al col­lab­o­ra­tion

Were­wolf Ther­a­peu­tics and Mer­ck are team­ing up for a new clin­i­cal tri­al.

Ran­di Isaacs

Look­ing at Were­wolf’s WTX-124 can­di­date tar­get­ing IL-2, the pair will col­lab­o­rate on a Phase I study eval­u­at­ing the pro­gram’s safe­ty and ef­fi­ca­cy in sol­id tu­mors, the com­pa­nies an­nounced Wednes­day. Re­searchers will ex­am­ine WTX-124 both as a monother­a­py and in com­bi­na­tion with Mer­ck’s Keytru­da.

“The clin­i­cal ben­e­fit of tar­get­ing IL-2 as a treat­ment for can­cer has long been es­tab­lished; how­ev­er, its util­i­ty has been lim­it­ed by chal­leng­ing tox­i­c­i­ties,” Were­wolf CMO Ran­di Isaacs said in a state­ment. “We be­lieve WTX-124 has the po­ten­tial to en­hance ther­a­peu­tic op­tions for can­cer pa­tients as a monother­a­py and when com­bined with check­point in­hibitors like Keytru­da.”

Were­wolf says it plans to sub­mit an IND for the WTX-124 pro­gram in the first half of 2022. Should the IND be cleared, the Phase I study will start “prompt­ly,” though no time­line was spec­i­fied. — Max Gel­man

Pen­ny stock sees shares jump at Phase II topline da­ta

Pen­ny stock play­er Azur­Rx got a sig­nif­i­cant bump Wednes­day af­ter it re­port­ed topline da­ta for a Phase II study.

James Sapirstein

Eval­u­at­ing the com­pa­ny’s MS1819 pro­gram for the treat­ment of se­vere ex­ocrine pan­cre­at­ic in­suf­fi­cien­cy in pa­tients with cys­tic fi­bro­sis, Azur­Rx said the can­di­date in com­bi­na­tion with stan­dard of care re­sult­ed in an av­er­age 6.57-per­cent­age point gain from base­line in the Co­ef­fi­cient of Fat Ab­sorp­tion, which the com­pa­ny says is a clin­i­cal­ly mean­ing­ful re­sult.

Pre­vi­ous lit­er­a­ture had said any­thing above a five-per­cent­age point im­prove­ment is clin­i­cal­ly mean­ing­ful. Da­ta from Wednes­day’s re­sults come from 20 pa­tients.

“Topline da­ta clear­ly show that com­bin­ing MS1819 to the dai­ly dose of PERT [stan­dard of care] had clin­i­cal ben­e­fits for all pa­tients and im­proved qual­i­ty of life,” CEO James Sapirstein said in a state­ment. “Our next step with the MS1819 pro­gram is to fi­nal­ize de­vel­op­ment of a new en­teric-coat­ed mi­crobead for­mu­la­tion, which we be­lieve will en­able more of the med­ica­tion to reach the small in­tes­tine there­by en­hanc­ing its ther­a­peu­tic po­ten­tial.”

Azur­Rx shares $AZRX sky­rock­et­ed 46% in pre-mar­ket trad­ing Wednes­day, set­tling in the 35% to 40% range af­ter the open­ing bell.

The for­mu­la­tion work for the new en­teric-coat­ed mi­crobead tech is ex­pect­ed to be com­plet­ed by the end of the year. Azur­Rx plans to launch a bridg­ing study in 2022 eval­u­at­ing the for­mu­la­tion as a monother­a­py. — Max Gel­man

So­ma­Log­ic does the Twist

Emi­ly Lep­roust

So­ma­Log­ic and Twist Bio­science have agreed to a new col­lab­o­ra­tion, an­nounced Wednes­day morn­ing. The deal will al­low Twist ac­cess to So­ma­Log­ic’s plat­form to dis­cov­er new an­ti­bod­ies and drug tar­gets.

No fi­nan­cial terms were dis­closed for the deal.

“We in­tend to use the an­ti­bod­ies dis­cov­ered for our in­ter­nal pipeline, mov­ing the most promis­ing can­di­dates through pre­clin­i­cal de­vel­op­ment and then po­ten­tial­ly out­li­cens­ing to a part­ner,” Twist CEO Emi­ly Lep­roust said in a state­ment. — Max Gel­man

Biotech de­vel­op­ing an­ti-in­flam­ma­to­ry COPD treat­ment lands €20M Se­ries A

An Ice­landic biotech that fo­cus­es on treat­ing chron­ic ob­struc­tive pul­monary dis­ease has se­cured a Eu­ro­pean Se­ries A fi­nanc­ing round, the com­pa­ny an­nounced Wednes­day.

Maria Bech

Epi­En­do raised has €20 mil­lion, or $23.4 mil­lion. The round was led by Flerie In­vest and Iðunn Ven­ture Fund. Ex­ist­ing in­vestor ABC Ven­tures par­tic­i­pat­ed, as well as the Eu­ro­pean In­no­va­tion Coun­cil Fund.

Funds will go to­ward mov­ing EP395 through clin­i­cal tri­als through Phase IIa. The drug en­tered Phase I tri­als in April.

“This fi­nanc­ing is a ma­jor step for Epi­En­do, not on­ly fund­ing our lead com­pound through clin­i­cal de­vel­op­ment in pa­tients with COPD, but al­so en­abling ex­plo­ration of oth­er im­por­tant po­ten­tial ther­a­peu­tic ap­pli­ca­tion ar­eas such as der­ma­tol­ogy and gas­troin­testi­nal dis­or­ders, where com­pro­mised ep­ithe­lial in­tegri­ty is known to con­tribute to dis­ease patho­phys­i­ol­o­gy,” CEO Maria Bech said in a press re­lease.

Sci­en­tists seek to make EP395 the first dis­ease mod­i­fy­ing non-an­tibi­ot­ic macrolide that can be used as a long-term treat­ment for COPD. It works by re­duc­ing in­flam­ma­tion in the ep­ithe­lial bar­ri­er, and in the fu­ture, the com­pa­ny will try the can­di­date on pa­tients with asth­ma, bronchiec­ta­sis and dif­fuse pan-bron­chi­oli­tis. — Josh Sul­li­van

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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