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

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Danish biotech Gubra announced a research collaboration and license agreement with Bayer to develop peptide therapeutics to treat cardiorenal diseases. The collaboration will utilize Gubra’s peptide drug discovery platform to identify potential candidates.

This is not the first time Gubra has partnered with a company on peptide therapeutics — they partnered with Boehringer Ingelheim back in 2017 to create peptide therapeutics to treat obesity.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.