Ot­su­ka’s $886M-plus drug just flopped in its first cru­cial PhI­II tri­al for front­line AML

Five years ago Ot­su­ka swept in and scooped up Cal­i­for­nia-based As­tex for $886 mil­lion, de­ter­mined to get its hands on their promis­ing leukemia drug SGI-110 as it plugged the op­er­a­tions in­to its glob­al R&D ops. Now the drug is called guadecitabine, and it just flopped in the first piv­otal Phase III study, crash­ing co-pri­ma­ry end­points for com­plete re­spons­es and over­all sur­vival for front­line use among ad­vanced pa­tients with acute myeloid leukemia.

The drug is billed as a next-gen­er­a­tion DNA hy­pomethy­lat­ing agent, but it failed against a group of pa­tients who re­ceived one of sev­er­al al­ter­na­tive ther­a­pies: azac­i­ti­dine, decitabine, or low dose cy­tara­bine.

Mo­ham­mad Az­ab

Ot­su­ka was pulled in by promis­ing mid-stage da­ta on the drug, con­fi­dent that it had the kind of proof-of-con­cept da­ta in hu­mans that jus­ti­fied an am­bi­tious Phase III pro­gram. The Japan­ese com­pa­ny now has two oth­er Phase III stud­ies — AS­TRAL-2 and AS­TRAL-3 — in the clin­ic for ad­vanced AML and myelodys­plas­tic syn­dromes, MDS, or chron­ic myelomono­cyt­ic leukemia.

Ot­su­ka kept As­tex on as a sub­sidiary, with its head­quar­ters and de­vel­op­ment work in Pleasan­ton, CA, and its dis­cov­ery and pre­clin­i­cal ef­fort based in Cam­bridge, UK. The com­pa­ny has been vexed by fail­ures on Alzheimer’s, where it’s part­nered with Lund­beck. But Ot­su­ka al­so re­cent­ly nabbed Vis­ter­ra in a $430 mil­lion deal, with plans to add that Waltham, MA-based biotech to the glob­al net­work.

“We are dis­ap­point­ed in the out­come of the AS­TRAL-1 study,” said As­tex CMO Mo­ham­mad Az­ab. “The study used very strict cri­te­ria of in­el­i­gi­bil­i­ty to re­ceive in­ten­sive chemother­a­py based on age (over 75 years) or poor per­for­mance sta­tus (ECOG PS of 2 or 3) or co­mor­bidi­ties, which made it a dif­fi­cult pop­u­la­tion to show su­pe­ri­or ben­e­fit of guadecitabine. AS­TRAL-1 is the largest glob­al prospec­tive study ever con­duct­ed in this spe­cif­ic pa­tient pop­u­la­tion with low in­ten­si­ty ther­a­py, with 815 pa­tients ran­dom­ized, of whom about 90% were treat­ed with hy­pomethy­lat­ing agents or HMAs (guadecitabine, azac­i­ti­dine, or decitabine).

En­her­tu picks up an­oth­er win for As­traZeneca and Dai­ichi Sankyo, join­ing the pri­or­i­ty re­view lane for gas­tric can­cer

Five months after Enhertu received twin breakthrough therapy designations, AstraZeneca and Daiichi Sankyo are one step closer to nabbing another approval for their potential blockbuster drug.

The companies announced Wednesday morning that their billion-dollar antibody-drug conjugate has received priority review for HER2 positive metastatic gastric cancer. Already approved in the US for third-line metastatic breast cancer patients that are HER2 positive, Enhertu’s gastric cancer PDUFA date is scheduled for the first quarter of 2021.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Sci­en­tists warn Amer­i­cans are ex­pect­ing too much from a coro­n­avirus vac­cine

The White House and many Americans have pinned their hopes for defeating the Covid-19 pandemic on a vaccine being developed at “warp speed.” But some scientific experts warn they’re all expecting too much, too soon.

“Everyone thinks COVID-19 will go away with a vaccine,” said William Haseltine, chair and president of Access Health International, a foundation that advocates for affordable care.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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Three years in­to a PhI­II pro­gram for a failed Duchenne MD drug, Cataba­sis hauls down the flag and ad­mits de­feat

Three years ago, Catabasis CEO Jill Milne and the crew insisted they had found good reason for great cheer once they plumbed the data from their failed study for the Duchenne MD drug edasalonexent. Plunging into the extended open-label data, they said, you could find solid evidence of efficacy. And that justified a try in Phase III.

But they were wrong.

Monday, after the bell, the little biotech acknowledged that their pivotal attempt following the mid-stage flop was another failure. The primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance.