Unit­ed Ther­a­peu­tics pays $800M-plus to grab Are­na’s PhI­II PAH drug

Unit­ed Ther­a­peu­tics $UTHR is pay­ing an $800 mil­lion cash up­front in a big bet to stay among the lead­ers in pul­monary ar­te­r­i­al hy­per­ten­sion, grab­bing rights to Are­na Phar­ma­ceu­ti­cals’ ra­linepag in the deal. And the ex­ec­u­tive crew is will­ing to add hun­dreds of mil­lions more in mile­stones if they can stay on track through to an ap­proval and com­mer­cial­iza­tion.

Unit­ed CEO Mar­tine Roth­blatt says their 20 years of ex­pe­ri­ence in the field con­vinced her that they have a win­ner on their hands.

She not­ed:

We have con­duct­ed ex­ten­sive due dili­gence on ra­linepag, ap­ply­ing our two decades of knowl­edge about PAH. We are con­fi­dent that af­ter achiev­ing FDA ap­proval via at least one of its sev­er­al dif­fer­ent po­ten­tial reg­u­la­to­ry path­ways to suc­cess, this prod­uct will help greater than 10,000 pa­tients an­nu­al­ly from the 2020s and well in­to the 2030s, while com­ple­ment­ing our ex­ist­ing port­fo­lio of PAH ther­a­pies.

Are­na’s shares $AR­NA surged about 20% pre­mar­ket on the news.

The oral drug, which is cur­rent­ly in late-stage de­vel­op­ment, is ex­pect­ed to com­pete against John­son & John­son’s $JNJ se­lex­i­pag, which the bio­phar­ma gi­ant ac­quired as part of its $30 bil­lion takeover of Eu­rope’s biotech pow­er­house Acte­lion last year.

Amit Mun­shi

Are­na is el­i­gi­ble to re­ceive po­ten­tial mile­stone pay­ments of up to $400 mil­lion un­der the deal, in­clud­ing $150 mil­lion up­on first mar­ket­ing ap­proval of ra­linepag in a ma­jor non-U.S. mar­ket, and an­oth­er $250 mil­lion up­on U.S. mar­ket­ing ap­proval of an in­haled for­mu­la­tion of ra­linepag. The San Diego-based com­pa­ny is al­so el­i­gi­ble to re­ceive cer­tain roy­al­ties on po­ten­tial net sales.

The com­pa­ny has un­der­gone a mas­sive re­or­ga­ni­za­tion af­ter its gam­ble on the obe­si­ty pill Belviq turned awry as luke­warm sales and adop­tion of the drug led to a rene­go­ti­at­ed agree­ment with part­ner Ei­sai, which ac­quired the glob­al rights to the di­et drug in 2017. In a sep­a­rate 8-K fil­ing, Are­na said that it ex­pects “a sig­nif­i­cant por­tion of the tax­able gain that would oth­er­wise be trig­gered by the up­front pay­ment will be off­set by Are­na’s net op­er­at­ing loss­es”.

In pa­tients with PAH, the right side of the heart has to work hard­er to push blood through the lungs, be­cause the pul­monary ar­ter­ies in the lung are blocked, nar­rowed or de­stroyed. This cul­mi­nates in high­er pres­sure in the right ven­tri­cle that pumps blood around the lungs, which over time weak­ens the ven­tri­cle caus­ing symp­toms such as short­ness of breath, fa­tigue and chest pain. As the con­di­tion wors­ens, the symp­toms may lim­it all phys­i­cal ac­tiv­i­ty. Al­though treat­ments have been ap­proved to man­age the symp­toms of the life-threat­en­ing con­di­tion, PAH re­mains in­cur­able.

In a mid-stage study re­port­ed in Ju­ly last year, the long-act­ing ra­linepag con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in pul­monary vas­cu­lar re­sis­tance of near­ly 30 per­cent when com­pared to the place­bo, as well as gains in pa­tients’ abil­i­ty to ex­er­cise. Three Phase III tri­als are now set to test ra­linepag in a broad pa­tient pop­u­la­tion.

Joseph Schwartz

As part of an R&D day last month, Are­na laid out the mar­ket op­por­tu­ni­ty for ra­linepag with the help of key opin­ion lead­ers, as cit­ed by Leerink’s Joseph Schwartz. KOLs see ra­linepag as a mean­ing­ful play­er in the PAH field, and they ex­pect the drug’s bet­ter phar­ma­co­ki­net­ic pro­file could trans­late to a bet­ter ef­fi­ca­cy than se­lex­i­pag, which in it­self was an im­prove­ment over Unit­ed Ther­a­peu­tics’ Oren­itram, not­ed Schwartz.

Are­na will now fo­cus its re­sources on its ex­per­i­men­tal drugs etrasi­mod and olorinab that are be­ing test­ed for var­i­ous oth­er dis­or­ders.



Im­age: Mar­tine Roth­blatt. AP IM­AGES

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.

J&J team shows off 'break­through' BC­MA CAR-T da­ta, and that could cause a big headache at blue­bird and Bris­tol-My­ers

Just hours after J&J’s oncology team bragged about scoring a breakthrough therapy designation for their BCMA CAR-T drug, they pulled the wraps off of the multiple myeloma data for JNJ-4528 that impressed the FDA. And it’s easy to see why they may well be on a short path to a landmark approval — which may well be making the rival team at bluebird/Bristol-Myers more than a little nervous.

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Jake Van Naarden, Josh Bilenker, Nisha Nanda (Credit: Loxo, Aisling Capital)

Josh Bilenker and his Loxo crew are tak­ing the reins on on­col­o­gy R&D at Eli Lil­ly, culling the weak and map­ping a new path

Josh Bilenker, Jake Van Naarden and Nisha Nanda came out of Eli Lilly’s $8 billion Loxo Oncology buyout with a bundle of cash and plenty of choices on what they could do next. Start a new company, go public. Live on the beach in 5-star luxury. Contemplate the stars — in their own observatory.

So what are they doing?

They formed a new executive team that is taking over the management of Eli Lilly’s hundreds-strong oncology R&D group — essentially using Loxo as a base for a bold new experiment in Big Pharma R&D in an attempt to create a true biotech environment with the deep pockets of a top-15 industry player. They’ve recruited David Hyman from Memorial Sloan Kettering to join the team as chief medical officer. And the mandate includes culling out the oncology pipeline, highlighting their star prospects and going after new programs wherever they can find the best prospects.

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