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

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; 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.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.