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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Greg Mayes, Antios Therapeutics CEO

An­tios' HBV col­lab axed af­ter clin­i­cal hold, but biotech be­lieves safe­ty in­ci­dent is not treat­ment-re­lat­ed

The FDA has placed a clinical hold on a Phase IIa study of Antios Therapeutics’ investigational hepatitis B med, CEO Greg Mayes confirmed to Endpoints News in an emailed statement.

A safety report was delivered to the biotech on May 17 after a patient dosed in a triple combination cohort of the study had experienced bradycardia and hypotension. The triple combo included Antios’ ATI-2173, Assembly Biosciences’ vebicorvir and Viread, an approved antiviral for HIV and hepatitis B.

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Tim Schoen, BioMed Realty CEO

Life sci­ences de­vel­op­er Bio­Med Re­al­ty buys San Fran­cis­co ho­tel for $75M — re­port

In a somewhat unconventional deal, life sciences real estate developer BioMed Realty has bought a 169-room Hilton Garden Inn in South San Francisco for $75 million, the San Francisco Business Times reported.

BioMed Realty, an affiliate of Blackstone, has multiple life sciences and technology office projects in the Bay Area, including three sites within a five-minute drive of the hotel.

While the sale of the hotel property was announced earlier this month, the sellers, Summit and GIC, did not identify the buyer at the time.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.