Dai­ichi says it’s ready to change the stan­dard of care for acute myeloid leukemia — but it’s a work in progress

Close to 4 years ago, Dai­ichi Sankyo swooped in to buy lit­tle San Diego-based Am­bit Bio­sciences for up to $410 mil­lion — $315 mil­lion of that in cash — to lay its hands on a leukemia drug called quizar­tinib.

Jorge Cortes

Over the week­end, in­ves­ti­ga­tors rolled out the late-stage da­ta that the Japan­ese com­pa­ny plans to take to reg­u­la­tors at the FDA. The re­sults are sta­tis­ti­cal­ly sig­nif­i­cant but un­spec­tac­u­lar, with 27% of the pa­tients suf­fer­ing from treat­ment-re­sis­tant acute myeloid leukemia with FLT3-ITD mu­ta­tions in the drug arm mak­ing it to 52 weeks com­pared to 20% of the chemo pa­tients in the con­trol arm.

The haz­ard ra­tio was 0.76, with a 24% drop in risk of death. The p-val­ue on that was pos­i­tive: 0.0177.

For Jorge Cortes at MD An­der­son, who led the study, the da­ta of­fer a com­pelling case for Dai­ichi’s drug as the on­ly sin­gle agent to prove ef­fi­ca­cy in this spe­cif­ic FLT3-ITD group. 

 “For 50 years, since the stan­dard of ther­a­py was in­tro­duced, noth­ing has changed since then,” Cortes tells me. “You could say a few weeks isn’t much,” but he sees this as a land­mark suc­cess that will change that long-run­ning stan­dard of care.

“I see this as very sig­nif­i­cant,” he adds, cit­ing the progress with an oral, sin­gle agent in a sal­vage set­ting with re­frac­to­ry pa­tients. “Any ben­e­fit for these pa­tients is very wel­come.”

That’s all true, but it’s worth not­ing that No­var­tis’ Ry­dapt (mi­dostau­rin) was ap­proved a year ago in com­bi­na­tion with chemo for front­line cas­es of AML with FLT3 mu­ta­tions (in­clud­ing ITD) while Nex­avar re­cent­ly demon­strat­ed ef­fi­ca­cy with chemo in the same spe­cif­ic pa­tient pop­u­la­tion. 

Dai­ichi is clear­ly in­ter­est­ed in see­ing how this drug can do with chemo as well, with at least two com­bo stud­ies on­go­ing, ac­cord­ing to clin­i­cal­tri­als.gov. One of the study de­scrip­tions notes the “tran­sient” na­ture of the sec­ond-gen drugs in the field. There’s al­so an ear­ly-stage study that was filed on the site a few days ago call­ing for a com­bi­na­tion of Dai­ichi’s MDM2 In­hibitor DS-3032 with quizar­tinib.

That chemo com­bo ap­proach may well prove ef­fec­tive in the front­line set­ting, says the in­ves­ti­ga­tor. And the MDM2 ap­proach could sig­nif­i­cant­ly ex­pand the pop­u­la­tion of AML pa­tients they can help.

The next stop for quizar­tinib: Reg­u­la­tors.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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