Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week be­fore the FDA is set to de­cide on Mirum Phar­ma­ceu­ti­cals’ lead liv­er dis­ease drug — an old Shire cast-off called mar­al­ix­i­bat — Take­da is swoop­ing in to se­cure the rights in Japan.

Mar­al­ix­i­bat’s roots trace back to Lu­me­na, which was snapped up by Shire for $260 mil­lion-plus back in 2014. While the can­di­date had failed mid-stage stud­ies at Shire, Mirum be­lieves bet­ter tri­al de­sign and pa­tient se­lec­tion will de­liv­er the wins it needs. The drug is cur­rent­ly in de­vel­op­ment for Alag­ille syn­drome (a con­di­tion called AL­GS in which bile builds up in the liv­er), pro­gres­sive fa­mil­ial in­tra­hep­at­ic cholesta­sis (PF­IC, which caus­es pro­gres­sive liv­er dis­ease) and bil­iary atre­sia (a block­age in the ducts that car­ry bile from the liv­er to the gall­blad­der).

Mirum sub­mit­ted an NDA for the can­di­date to treat itch­i­ness in pa­tients with AL­GS in March, and reg­u­la­tors as­signed a PDU­FA date of Sept. 29.

“As we ap­proach po­ten­tial com­mer­cial­iza­tion in the Unit­ed States and com­plete the re­cent fil­ing for Alag­ille syn­drome in Eu­rope, our goal is to part­ner with top com­pa­nies out­side of North Amer­i­ca and Eu­rope to en­sure glob­al reach for pa­tients with these ter­ri­ble dis­eases,” CEO Chris Peetz said in a state­ment.

For an undis­closed amount, Take­da is get­ting de­vel­op­ment and com­mer­cial­iza­tion rights to mar­al­ix­i­bat in AL­GS, PF­IC and BA in Japan. The phar­ma will be re­spon­si­ble for reg­u­la­to­ry ap­proval and com­mer­cial­iza­tion in that coun­try, as well as de­vel­op­ment, in­clud­ing clin­i­cal stud­ies in cholesta­t­ic in­di­ca­tions. — Nicole De­Feud­is

Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

Ger­man bio­phar­ma Boehringer In­gel­heim an­nounced to­day that it bought pre­ci­sion on­col­o­gy firm Abexxa Bi­o­log­ics. The deal in­cludes an undis­closed up­front pay­ment, along with mile­stones and oth­er pay­ments.

Boehringer In­gel­heim has been busy, hav­ing just agreed less than two weeks ago to dole out more than $700 mil­lion in mile­stones to Twist Bio­science for ac­cess to its an­ti­body li­brary.

“The ac­qui­si­tion of Abexxa bol­sters our com­mit­ment to tu­mor-anti­gen dis­cov­ery and new ways of tar­get­ing in­tra­cel­lu­lar anti­gens,” said Boehringer In­gel­heim’s cor­po­rate SVP and head of dis­cov­ery re­search, Clive Wood, in a pre­pared state­ment.

In 2016, Boehringer In­gel­heim’s Ven­ture Fund award­ed Abexxa ini­tial in­vest­ment fund­ing. Lat­er that year, Abexxa won In­gel­heim’s In­no­va­tion Prize, which al­lowed Abexxa to ex­pand op­er­a­tions in­to a shared lab space in Cam­bridge, Mass­a­chu­setts.

Abexxa will con­tin­ue to op­er­ate in Ar­ling­ton, Texas, col­lab­o­rat­ing with their US-based col­leagues at Boehringer In­gel­heim’s fa­cil­i­ty in Ridge­field, Con­necti­cut. — Paul Schloess­er

Black Di­a­mond Ther­a­peu­tics strikes dis­cov­ery deal with soft­ware com­pa­ny Open­Eye Sci­en­tif­ic

Black Di­a­mond Ther­a­peu­tics an­nounced this morn­ing that it is en­ter­ing in­to a strate­gic part­ner­ship with sci­ence soft­ware firm Open­Eye, in­te­grat­ing the soft­ware com­pa­ny’s in-house mol­e­c­u­lar de­sign plat­form in­to Black Di­a­mond’s drug dis­cov­ery en­gine.

Ac­cord­ing to a state­ment re­leased by the two com­pa­nies, Black Di­a­mond and Open­Eye will work to co-de­vel­op sam­pling ca­pa­bil­i­ties and de­liv­er in­for­ma­tion more quick­ly. Ul­ti­mate­ly, their goal is to ad­vance Black Di­a­mond’s ef­forts to de­vel­op can­cer ther­a­pies that can tar­get an en­tire fam­i­ly of mu­ta­tions — what they call a “Mas­terKey” in­hibitor, ac­cord­ing to a pre­pared state­ment.

The deal fol­lows a re­cent per­son­nel move at Black Di­a­mond, as then-CMO Rachel Humphrey was fired and CSO Christo­pher Roberts re­signed from the pre­ci­sion on­col­o­gy firm in mid-Ju­ly.

The arrange­ment be­tween Open­Eye and Black Di­a­mond in­volves an undis­closed up­front pay­ment, along with po­ten­tial down­stream eco­nom­ics for Open­Eye.

“We are be­gin­ning to see the mar­riage of com­pu­ta­tion and ge­nomics not just through se­quence analy­sis, but at the struc­tur­al lev­el,” said Open­Eye CEO and founder An­tho­ny Nicholls in a state­ment. “We’re very proud to be able to work with Black Di­a­mond to com­bine mol­e­c­u­lar sim­u­la­tion on our cloud plat­form, Ori­on, with their MAP drug dis­cov­ery en­gine to ac­cel­er­ate their search for nov­el can­cer ther­a­peu­tics.” — Paul Schloess­er

Pe­ter Thiel joins the board of a Medicare ad­vi­so­ry group

As pro­pos­als for drug pric­ing re­forms make their way through Con­gress, Sil­i­con Val­ley pow­er play­er Pe­ter Thiel is jump­ing on the board of Chap­ter, a Medicare ad­vis­ing group that helps Amer­i­cans find the best cov­er­age plans.

Chap­ter un­veiled a $17 mil­lion Se­ries A round Tues­day, led by Narya Cap­i­tal with a hand from Susa Ven­tures, Mav­er­ick Ven­tures, XYZ Ven­ture Cap­i­tal, Core In­no­va­tion Cap­i­tal and Health2047 Cap­i­tal Part­ners. Along with the round, the com­pa­ny added Thiel to its board of di­rec­tors.

“It is…com­mon­place that deal­ing with the gov­ern­ment is dif­fi­cult. It is un­com­mon that any­one does any­thing about it,” Thiel said in a state­ment. “Chap­ter’s abil­i­ty to help peo­ple nav­i­gate Medicare’s bu­reau­crat­ic maze makes it both ad­mirable and valu­able.”

While lega­cy Medicare bro­kers aren’t re­quired to search a min­i­mum num­ber of plans or dis­close the num­ber they do search, Chap­ter promis­es to search every plan — in­clud­ing Medicare Ad­van­tage, Medicare Sup­ple­ments and stand­alone Part D pre­scrip­tion plans. To do so, it’s part­nered with Thiel-found­ed Palan­tir Tech­nolo­gies to de­vel­op a com­pre­hen­sive Medicare da­ta plat­form.

The news comes a cou­ple of weeks af­ter Pres­i­dent Joe Biden’s ad­min­is­tra­tion called for new re­forms to in­clude Medicare drug price ne­go­ti­a­tions, as well as mea­sures to slow price in­creas­es over time on ex­ist­ing drugs.

“Most Amer­i­cans nav­i­gate Medicare alone or un­in­ten­tion­al­ly use re­sources that lim­it their op­tions,” Chap­ter CEO Co­bi Blu­men­feld-Gantz said in a state­ment. “Medicare cov­er­age is too im­por­tant to get wrong: sign­ing up too late can re­sult in life­time penal­ties. Fur­ther­more, plans have such sig­nif­i­cant dif­fer­ences in ben­e­fits and pre­mi­ums that it’s crit­i­cal to search every sin­gle op­tion.”  — Nicole De­Feud­is

Il­lu­mi­na ex­pects EU or­der to keep Grail a sep­a­rate com­pa­ny

Il­lu­mi­na said on Mon­day that it ex­pects the Eu­ro­pean Com­mis­sion to is­sue an or­der to keep re­cent­ly ac­quired Grail as a sep­a­rate com­pa­ny — a hand­ful of hours af­ter EU an­titrust reg­u­la­tors warned of in­ter­im mea­sures for clos­ing the deal be­fore their ap­proval.

While Il­lu­mi­na fi­nal­ized the Grail takeover last month de­spite protest from the Com­mis­sion, the biotech said it would hold the com­pa­ny sep­a­rate while wait­ing for a de­ci­sion on the deal.

“We had an­tic­i­pat­ed that the Com­mis­sion would seek to im­pose a hold sep­a­rate or­der, and this is the rea­son why it has al­ready vol­un­tar­i­ly agreed to such an arrange­ment; their pro­pos­als are based on Il­lu­mi­na’s vol­un­tary un­der­tak­ings,” Il­lu­mi­na said in a state­ment. The com­pa­ny said it would dis­cuss cer­tain changes sug­gest­ed by the EU in the com­ing days.

Un­der EU merg­er rules, a com­pe­ti­tion watch­dog can or­der com­pa­nies to un­wind their deals or sell off shares or as­sets ac­quired for vi­o­la­tions.

Com­mis­sion VP Mar­grethe Vestager em­pha­sized Il­lu­mi­na’s ac­tions, say­ing it was the first time com­pa­nies had open­ly im­ple­ment­ed a deal while reg­u­la­tors were still in­ves­ti­gat­ing. She not­ed the com­pa­ny’s ac­tions were se­ri­ous.

“The stand­still oblig­a­tion is a cor­ner­stone of our ex-ante merg­er con­trol regime which aims at pre­vent­ing harm­ful ef­fects to com­pe­ti­tion while our re­view is on­go­ing,” she said in a state­ment.

The Com­mis­sion said the in­ter­im mea­sures aim to re­store and main­tain ef­fec­tive com­pe­ti­tion pend­ing a fi­nal de­ci­sion on the deal, which will be made af­ter re­ceiv­ing the com­pa­nies’ re­sponse to the state­ment of ob­jec­tions. — Paul Schloess­er

Stoke Ther­a­peu­tics sees ear­ly signs of ef­fi­ca­cy for Dravet syn­drome can­di­date

The num­bers are in on Stoke Ther­a­peu­tics’ lead an­ti­sense oligonu­cleotide for Dravet syn­drome.

Sin­gle dos­es of STK-001 up to 30 mg and mul­ti­ple dos­es at 20 mg were found to be well-tol­er­at­ed in a Phase I/IIa study, Stoke an­nounced on Tues­day. The in­ter­im analy­sis was based on 21 pa­tients treat­ed in sin­gle-dose co­horts who were fol­lowed for at least three months, and six pa­tients in the 20 mg mul­ti­ple-dose co­hort, most of whom had re­ceived three month­ly dos­es.

Dravet syn­drome is a se­vere and pro­gres­sive epilep­sy that be­gins with­in a new­born’s first year of life. Pa­tients in the Phase I/IIa study had a me­di­an of 17 con­vul­sive seizures in the four-week screen­ing pe­ri­od lead­ing up to treat­ment, ac­cord­ing to Stoke.

While there were no se­ri­ous side ef­fects re­lat­ed to STK-001, the most com­mon low­er-grade events were headache, ir­ri­tabil­i­ty, vom­it­ing, seizure and back pain.

In ad­di­tion, pri­ma­ry analy­ses sug­gest a trend to­ward a re­duc­tion in seizure fre­quen­cy in the sin­gle-dose group, Stoke an­nounced. The trend was more ev­i­dent in the 2- to 12-year-old age group.

“The ini­tial pos­i­tive safe­ty da­ta from MONARCH are high­ly en­cour­ag­ing and we now have greater clar­i­ty on the dose lev­els that are like­ly to be phar­ma­co­log­i­cal­ly ac­tive in pa­tients,” CMO Bar­ry Ti­cho said in a state­ment.

The com­pa­ny plans on giv­ing an­oth­er up­date at the Amer­i­can Epilep­sy So­ci­ety meet­ing in De­cem­ber and shar­ing da­ta from the pa­tients giv­en mul­ti­ple dos­es of 30 mg in the sec­ond half of next year.  — Nicole De­Feud­is

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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