Cel­gene’s $710M deal on mon­gersen im­plodes as Phase III Crohn's study flops

Cel­gene’s $710 mil­lion cash roll of the dice on the in­flam­ma­to­ry bow­el dis­ease drug mon­gersen (GED-301) has come up snake eyes.

The big biotech $CELG an­nounced Thurs­day evening that it is ter­mi­nat­ing the Phase III on Crohn’s af­ter it failed to pass muster in an in­ter­im fu­til­i­ty analy­sis. An­oth­er planned Phase III study is be­ing scrapped, and the team there will make a fi­nal de­ter­mi­na­tion if there’s any fu­ture left for this drug af­ter they com­plete an analy­sis of Phase II da­ta on ul­cer­a­tive col­i­tis.

Cel­gene’s stock plunged 7% on the news, tear­ing out a $7 bil­lion-plus chunk of its mar­ket cap.

Scott Smith, Cel­gene

Cel­gene COO Scott Smith presided over the brief last rites, say­ing that “while we are dis­ap­point­ed with the re­sults of RE­VOLVE, we re­main com­mit­ted to ad­vanc­ing our port­fo­lio of nov­el med­i­cines for pa­tients suf­fer­ing from this dis­ease and oth­er in­flam­ma­to­ry bow­el dis­or­ders.”

Al­most ex­act­ly three years ago to the day, the biotech spelled out promis­ing mid-stage da­ta for Crohn’s, ex­plain­ing why Cel­gene’s then CEO (now ex­ec­u­tive chair­man) Bob Hug­in had com­mit­ted to a deal worth up to $2.6 bil­lion with the lit­tle known Irish com­pa­ny No­gra.

Leerink’s Ge­of­frey Porges wrote it off as an un­for­tu­nate out­come trig­gered by an in­ex­pe­ri­enced team of deal­mak­ers at Cel­gene who rushed in­to this three years ago with way too much mon­ey up front. Notes Porges:

This dis­ap­point­ment is like­ly to cost the com­pa­ny up to $2bn in long-term rev­enue, and up to 4-5% in val­ue; it is al­so a painful re­minder of the costs of a “shots on goal” ap­proach to busi­ness de­vel­op­ment, and the per­ils of heav­i­ly front-loaded in­vest­ments in­to cat­e­gories where the or­ga­ni­za­tion lacks tech­ni­cal and com­mer­cial ex­per­tise.

The com­pa­ny now will shift all of its fo­cus to ozan­i­mod af­ter of­fer­ing a peek at the Phase II re­sults for Crohn’s and ul­cer­a­tive col­i­tis post­ed at the World Con­gress of Gas­troen­terol­o­gy at ACG2017 in Or­lan­do.

For Cel­gene, the mon­gersen deal helped il­lus­trate its will­ing­ness to bet big on clin­i­cal-stage as­sets. Mon­gersen is the biotech’s most ad­vanced ex­per­i­men­tal drug in its in­flam­ma­tion and im­munol­o­gy pipeline, where the com­pa­ny had high hopes for some block­buster break outs.

This one proved an ex­pen­sive fail­ure.

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.

Roger Perlmutter, Merck

Mer­ck bags Ar­Qule's next-gen BTK can­cer drug with $2.7B buy­out deal — pay­ing more than dou­ble last close

Just 6 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 with Merck.

The pharma giant says it is scooping up the drug in an M&A deal for $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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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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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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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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J&J's Mathai Mammen at an Endpoints News event in Boston, June 2018 (Photo: Rob Tannenbaum for Endpoints News)

J&J fronts $750M cash to grab a failed can­cer drug that’s been re­pur­posed as a pow­er­ful an­ti-in­flam­ma­to­ry

J&J has stepped up with one of its blockbuster drug buys, agreeing to pay Austin-based XBiotech $XBIT $750 million in cash and up to $600 million more in milestones for their late stage-ready anti-inflammatory drug bermekimab — which some longtime biotech observers may recognize as a failed cancer therapy with a disaster-prone past.

The drug targets the IL-1a pathway. J&J $JNJ R&D chief Mathai Mammen is cutting a check for a drug that has produced positive mid-stage data in patients suffering from a skin condition called hidradenitis suppurativa with another mid-stage program underway for atopic dermatitis.

That puts J&J in charge of a drug on the threshold of pivotal — though pricey — R&D work for a broad patient group with other related fields to explore. And it’s a very busy development arena.

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Sangamo CEO Sandy Macrae

Pa­tient #9 has been a con­cern, but Sang­amo and Pfiz­er are bull­ish about win­ning the marathon he­mo­phil­ia A gene ther­a­py race

Patient number 9 has given Sangamo and its partners at Pfizer some heart palpitations in their high profile hemophilia A gene therapy program.

After watching his Factor VIII level rise following treatment like the rest, the crucial efficacy gauge they track saw a sudden and significant plunge. At week 13, the FVIII level had dropped below normal. Then it began to rise again.

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