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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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US sup­ports ex­ten­sion for Covid-19 IP waiv­er de­ci­sion

After much debate, the US government is now calling for a deadline extension to discuss a controversial potential IP waiver for Covid-19 diagnostics and therapeutics.

Over the last five months, the Office of the United States Trade Representative said it has consulted with members of Congress, public health advocates, organized labor groups, academics, think tanks, companies and trade associations on the WTO’s recent TRIPS agreement, which established a 5-year waiver of certain patent requirements on Covid-19 vaccines.

Sum­i­to­vant sub­sidiaries En­zy­vant and Al­ta­vant merge in­to com­bined com­pa­ny

Two Sumitovant Biopharma entities are merging under one name, effective immediately.

Enzyvant Therapeutics and Altavant Sciences announced they have merged to form a singular entity focused on developing therapies for patients with rare diseases. The combined company will keep the name Enzyvant and along with clinical development will eventually include in-house manufacturing.

Bill Symonds, the current CEO of both Altavant and Enzyvant, is now CEO of the merged company.

Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Mar­ket­ingRx roundup: Phar­mas lay off Twit­ter ads for an­oth­er week; WPP un­cov­ers LGBTQ+ mar­ket­ing find­ings

When Twitter’s new owner Elon Musk tweeted this weekend, “Just a note to thank advertisers for returning to Twitter,” he likely wasn’t talking about big pharma companies. The vast majority of the top spending pharma advertisers had not returned last week, according to updated tracking data Pathmatic for Endpoints News.

Only three pharma advertisers spent any money at all, which is about the same as the past several weeks. AstraZeneca rejoined the active advertiser list, although at $700 spent hardly worth a personal Musk expression of gratitude. GSK remained active with $3,500 spent ad much lower than its previous spending, according to the Pathmatics data. Only Bayer spent any significant amount in advertising, with $244,000 spent last week, but that’s a considerable drop from almost $500,000 spent on OTC, prescription and corporate Twitter ads in each of the previous two weeks.

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Bob Duggan, Summit Therapeutics co-CEO

Bounc­ing from ma­jor set­back, Sum­mit hands out $500M cash for can­cer drug — thanks to a loan from bil­lion­aire CEO

After hitting a dead end with Summit Therapeutics’ lead program, Bob Duggan has found the drug that he believes will usher into a compelling second act. So compelling, in fact, that it involves $500 million cash — and he’s taking money out of his own pocket to fund the deal.

Striking a partnership with Akeso Therapeutics out of China, Summit is bringing in a bispecific antibody that blocks both PD-1 and VEGF called ivonescimab. Akeso, which has a PD-1/CTLA-4 bispecific approved in China, has already taken ivonescimab into multiple clinical trials, including a Phase III in lung cancer.

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