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

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

The FDA has another adcomm coming down the pipeline — this time on Covid-19 oral antiviral molnupiravir.

The federal agency’s advisory committee will meet on November 30th to go over Merck and Ridgeback’s EUA request for their investigational antiviral drug, and discuss the available data supporting its use in Covid-19 patients.

This comes two weeks after Merck claimed that their antiviral pill reduced the chance that newly diagnosed Covid-19 patients would be hospitalized or die by 50%. The pharma made the announcement after interim data on 775 patients in their clinical trial showed the antiviral’s potential.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.