Bay­er boost­ed as FDA OKs its PI3K drug Aliqopa for re­lapsed fol­lic­u­lar lym­phoma pa­tients

A lit­tle more than a year ago, Gilead ran in­to some se­ri­ous prob­lems when its PI3K can­cer pill Zy­delig was linked with a rash of deaths in front­line stud­ies, crip­pling what was once con­sid­ered a po­ten­tial block­buster sales ef­fort. Now Bay­er is com­ing along with an in­fused ri­val that will try and start to fol­low up on the suc­cess en­joyed by John­son & John­son $JNJ and Ab­b­Vie’s $AB­BV Im­bru­vi­ca, a po­ten­tial mega-block­buster play­er which has been scor­ing some big gains in leukemia and lym­phoma.

The FDA has ap­proved Bay­er’s co­pan­lis­ib, with will now be mar­ket­ed as Aliqopa for re­lapsed fol­lic­u­lar lym­phoma. Reg­u­la­tors were quick to fol­low up af­ter pro­vid­ing Bay­er’s can­cer prospect with a pri­or­i­ty re­view, lop­ping 4 months off the reg­u­la­to­ry process.

Bay­er has some lim­it­ed am­bi­tions here, say­ing ear­li­er that it ex­pects to rack up about $600 mil­lion a year, in line with an­a­lysts es­ti­mates for its peak sales po­ten­tial.

Richard Padzur

The pri­ma­ry end­point in Phase II, which is be­ing used for the ac­cel­er­at­ed ap­proval, was over­all re­sponse among treat­ment re­sis­tant pa­tients. And in all groups the ORR of 59.2%, with a 12% com­plete re­sponse rate and a me­di­an du­ra­tion of re­sponse of more than 98 weeks, or 687 days. But pulling out the 104 fol­lic­u­lar lym­phoma pa­tients in CHRONOS-1, co­pan­lis­ib scored an ORR of 58.7%, in­clud­ing a com­plete re­sponse of 14.4% and a me­di­an DOR of more than 52 weeks, or 370 days.

“For pa­tients with re­lapsed fol­lic­u­lar lym­phoma, the can­cer of­ten comes back even af­ter mul­ti­ple treat­ments,” said Richard Paz­dur, di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “Op­tions are lim­it­ed for these pa­tients and to­day’s ap­proval pro­vides an ad­di­tion­al choice for treat­ment, fill­ing an un­met need for them.”

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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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.
Provided there are no snafus or even modest stumbles now, they should get an OK within the 2020 timeline lined out in their $9 CVR for Celgene — the first in a trifecta of approvals required for a payout.
Whether they can go on to make it into a viable commercial therapy, though, is a whole other thing.
At first glance, there isn’t anything about the safety and efficacy data that would force a CRL or delay. In a large trial of patients with relapsed/refractory large B-cell lymphomas investigators tracked an outstanding 73% response rate and 53% complete response rate in heavily pretreated patients with few options.
That’s in line with Yescarta from Gilead’s Kite, which snagged an approval more than 2 years ago, just after Novartis’ Kymriah came through.
Then there’s safety. To be sure, the drug has safety issues. There were 4 patients in the study who died after treatment. Several others died for unrelated issues. But…with only a 2% rate of cytokine release syndrome, Bristol-Myers has a shot at a differentiated safety profile.
“There is a potential these patients can be treated on an outpatient basis,” says Samit Hirawat, the chief medical officer at Bristol-Myers. He noted that 23% of patients were never admitted, while 73% were admitted 4 days or later after therapy. The key is to treat them at a hospital with the infrastructure to provide care 24/7, so a patient can be admitted at any time later if needed.
How that will fly with payers after rivals have been on the market for about 3 years, with new evidence that earlier use of steroids can dramatically reduce CRS and considerable durability of response, will have to be seen.
But time is not on Bristol-Myers’ side. Liso-cel is the long delayed followup to the disastrous JCAR015, which killed a number of patients. Their setback threw them years off schedule. And rivals are advancing off-the-shelf alternatives or other new approaches that could knock the pioneers completely out of the game. In the meantime, Bristol-Myers is gathering its own durability data and will gradually see if their mix of CD4 and CD8 cells can do better.
The main interest now is in the timeline around the approval. Hirawat says they’ll ask for priority review, and there’s every reason to believe that regulators will move swiftly — barring a nasty surprise.

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.

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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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Samantha Budd Haeberlein. Biogen via YouTube

UP­DAT­ED: Skep­tics pounce as Bio­gen de­tails pos­i­tive sub­group analy­sis on ad­u­canum­ab — and both sides are dig­ging in

“Exhilarating.” “A major advance.” “A milestone achievement.” If one had just tuned into the panel comments on Biogen’s presentation at CTAD, it would seem that the biotech had an impressive, disease-modifying Alzheimer’s drug in aducanumab.

But off the stage, reactions to their admittedly complicated dataset and the biotech’s explanation for resurrecting a drug that failed its futility analysis were a lot more mixed, with analysts continuing to question whether the evidence is substantial enough to warrant an FDA approval and raising new doubts on the safety side.

In an investor call later in the day, execs noted that they are not planning another study and stood by their intention, publicized in October to much surprise, to submit regulatory filings based on what they have.

“We don’t file willy nilly,” said Al Sandrock, head of R&D. “We only go to filing when we believe that there is a benefit-risk argument based on science, based on data. And if you look at our history, we haven’t done filings right and left without good reason.”

Biogen had a theory going into the Clinical Trials on Alzheimer’s Disease meeting.

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Vas Narasimhan, Getty Images

No­var­tis CEO Vas Narasimhan's R&D up­date spot­lights next wave of drug stars as well as late-stage fa­vorites

As one of the biggest spenders in biopharma R&D, Novartis execs love to tout the scope of its late-stage pipeline, spotlighting the winners most likely to create blockbuster revenue streams in the near future.

Building on the 5 drug approvals the pharma giant expects to end the year with, Novartis CEO Vas Narasimhan — who’s done a slate of acquisitions topped by the recent $9.7 billion MedCo buyout — tapped the top emerging drugs as:

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