He­mo­phil­ia groups warned of 5 deaths among pa­tients tak­ing Roche’s big new block­buster Hem­li­bra

The He­mo­phil­ia Fed­er­a­tion of Amer­i­ca has put out a state­ment not­ing that Roche alert­ed them of the deaths of 5 pa­tients who were tak­ing their new drug Hem­li­bra, cal­cu­lat­ed to be the biggest sin­gle drug launch of the year. And ac­cord­ing to Roche, the alert fol­lowed the death of two pa­tients ear­li­er this year, which they just learned about.

Ac­cord­ing to the state­ment, Roche’s Genen­tech in­formed them of the deaths Mon­day — adding 4 fa­tal­i­ties to the 1 death al­ready re­port­ed ear­ly last year.

The news trig­gered a 1.4% drop in Roche shares Wednes­day morn­ing.

Ac­cord­ing to the HFA:

Genen­tech has lit­tle in­for­ma­tion that they can present­ly share about the cir­cum­stances sur­round­ing the most re­cent pa­tient deaths; how­ev­er, Genen­tech could con­firm that the pa­tients had re­ceived Hem­li­bra as part of com­pas­sion­ate use and ex­pand­ed pa­tient ac­cess.

In a state­ment to End­points News late Tues­day, a spokesper­son for the com­pa­ny said that one of the pa­tients who died was in an ex­pand­ed ac­cess pro­to­col, three re­ceived it fol­low­ing com­pas­sion­ate use re­quests and one was in the HAVEN I tri­al. She added:

To date, five adults tak­ing Hem­li­bra have passed away. For all, the treat­ing physi­cian or in­ves­ti­ga­tor’s as­sess­ment was that the cause of death was un­re­lat­ed to Hem­li­bra (em­pha­sis pro­vid­ed by Roche). We pro­vid­ed the na­tion­al ad­vo­ca­cy groups with this up­date via phone yes­ter­day.

In a fol­lowup, Roche added a time­line:

  • In 2016, one adult tak­ing Hem­li­bra through a re­quest for com­pas­sion­ate use passed away.
  • In 2017, one adult par­tic­i­pat­ing in the Phase III HAVEN 1 clin­i­cal tri­al passed away and one adult tak­ing Hem­li­bra through a re­quest for com­pas­sion­ate use passed away.
  • In 2018, we just re­cent­ly learned that two adults tak­ing Hem­li­bra passed away. Again, the treat­ing physi­cian’s as­sess­ment for both pa­tients was that the cause of death was un­re­lat­ed to Hem­li­bra.

Roche $RHH­BY has had safe­ty is­sues to deal with be­fore on this drug.

The phar­ma gi­ant re­port­ed 2 cas­es of throm­boem­bol­ic events and 3 in­stances of throm­bot­ic mi­croan­giopa­thy from the Haven I Phase III tri­al. Roche rat­tled its most bull­ish fol­low­ers on this drug with the re­port that a pa­tient died on Feb­ru­ary 22, 2017 from a rec­tal he­m­or­rhage af­ter be­ing treat­ed with emi­cizum­ab and a by­pass­ing agent dur­ing a break­through bleed­ing episode. The in­ci­dents were at­trib­uted to the BPAs, which an­gered Shire and No­vo Nordisk, the two key ri­vals who are feel­ing the biggest im­pact from the ar­rival of Hem­li­bra.

Shire in par­tic­u­lar came out gun­ning against Hem­li­bra, which has a peak sales es­ti­mate of $4 bil­lion. Shire has claimed be­fore that Roche has is­sued “in­ac­cu­rate and mis­lead­ing” state­ments about the se­ri­ous ad­verse events tracked in its Phase III – events which con­spired to keep this would-be block­buster un­der a cloud.

Roche, though, nev­er blinked, bag­ging an ap­proval in the US No­vem­ber 17 for one of its biggest drugs in the pipeline, a key win that was bad­ly need­ed to ac­count for the loss of patent pro­tec­tion on 3 big fran­chise drugs. The EU fol­lowed with an OK Feb­ru­ary 18 and Japan fol­lowed suit March 18.

In the US, the FDA added a boxed warn­ing about safe­ty haz­ards. When the drug was ap­proved last fall, the agency not­ed:

Cas­es of throm­bot­ic mi­croan­giopa­thy and throm­bot­ic events were re­port­ed when on av­er­age a cu­mu­la­tive amount of >100 U/kg/24 hours of ac­ti­vat­ed pro­throm­bin com­plex con­cen­trate (aPCC) was ad­min­is­tered for 24 hours or more to pa­tients re­ceiv­ing emi­cizum­ab-kxwh pro­phy­lax­is. The pre­scrib­ing in­for­ma­tion con­tains a boxed warn­ing to mon­i­tor for throm­bot­ic mi­croan­giopa­thy and throm­bot­ic events when aPCC is ad­min­is­tered. If symp­toms oc­cur, aPCC should be dis­con­tin­ued and emi­cizum­ab-kxwh should be sus­pend­ed.

The HFA added:

Genen­tech has a Med­ical Com­mu­ni­ca­tions line at 1(800)-821-8590 for pa­tients, con­cerned com­mu­ni­ty mem­bers, and health­care providers who seek fur­ther in­for­ma­tion. HFA en­cour­ages you to reach out to Genen­tech and/or to your treat­ing physi­cian if you have ques­tions or con­cerns.

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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Un­lock the full End­points ex­pe­ri­ence for your com­pa­ny — and sup­port our mis­sion of in­de­pen­dent bio­phar­ma re­port­ing

I want to give readers a quick update on the most important part of our business model — premium subscriptions. We have some crucial financial goals we hope to achieve by the end of the year, and the team here in Lawrence is ready to ship some swag to kick off this limited December promotion.

We offer two premium plans — Enterprise for companies ($1,000/year, unlimited people), and Insider for individuals ($200/year). This month of December will be the last chance to enroll at the original rates — which have remained flat since we launched them in 2017.

One of Wall Street’s most high-pro­file hedge funds push­es Alex­ion's CEO to the auc­tion block — and he's not budg­ing

Fresh off buying Barnes & Noble and prodding AT&T with some heavy-handed criticism after picking up a $3.2 billion stake in the company, the activist — and supremely high profile — hedge fund Elliott Management has stepped up with some M&A advice for Alexion’s management team.
And the execs on the team $ALXN are giving them a polite — but very firm — stiff arm Friday morning.
In a release out early Friday, the big biotech said that the Elliott team had been in touch to encourage them to sell the company. But that’s not on the agenda.

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