Agios scores its sec­ond new drug ap­proval, ivosi­denib heads to the AML mar­ket

Agios has hit an­oth­er big goal in its decade-long track record. The FDA to­day sig­naled the ac­cel­er­at­ed ap­proval of ivosi­denib (AG-120) for ad­vanced acute myeloid leukemia.

David Schenkein

This marks the sec­ond drug ap­proval for Agios, a biotech com­pa­ny found­ed by David Schenkein, which he’s fond of say­ing got start­ed with a “blank sheet of pa­per.”

That sheet is get­ting quite crowd­ed now.

The new drug will be mar­ket­ed by Agios as Tib­so­vo, and the com­pa­ny is charg­ing $26,115 for a one-month sup­ply. Agios shares $AGIO were down just slight­ly — this was no great sur­prise — but the stock is up 56% 0f where it was a year ago.

Billed as “the first tar­get­ed treat­ment for R/R AML pa­tients with an IDH1 mu­ta­tion,” Agios sub­mit­ted it back in Feb­ru­ary with noth­ing but ear­ly-stage da­ta that had been on dis­play at ASH. 

The OK comes close to a year af­ter the biotech’s first ap­proval for Id­hi­fa, an IDH2 drug for an­oth­er slice of the AML mar­ket be­ing han­dled by its part­ners at Cel­gene. And just like Id­hi­fa, the FDA hur­ried this new ap­proval along with a pri­or­i­ty re­view.

To­geth­er, the two drugs cov­er 20% of the AML mar­ket.

This new drug was test­ed in an open la­bel study, with no con­trol arm. In­ves­ti­ga­tor found:

•CR+CRh rate of 32.8% (57 of 174 pa­tients) (95% CI: 25.8, 40.3).

•The CR rate was 24.7% (43 of 174 pa­tients) (95% CI 18.5, 31.8) and the CRh rate was 8% (14 of 174 pa­tients) (95% CI 4.5, 13.1).

•Me­di­an du­ra­tion of CR+CRh was 8.2 months (95% CI: range 5.6, 12 months).

•For pa­tients who achieved a CR or CRh, the me­di­an time to best re­sponse of CR or CRh was 2.0 months (range, 0.9 to 5.6 months).

•Among the 110 pa­tients who were de­pen­dent on red blood cell (RBC) and/or platelet trans­fu­sions at base­line, 41 (37.3%) be­came in­de­pen­dent of RBC and platelet trans­fu­sions dur­ing any 56-day post-base­line pe­ri­od.

“The FDA ap­proval of Tib­so­vo – our first whol­ly owned drug and the sec­ond ap­proved med­i­cine from our re­search plat­form in less than a year – is an in­cred­i­bly ex­cit­ing mile­stone for our com­pa­ny and, im­por­tant­ly, for the ap­prox­i­mate­ly 6-10% of AML pa­tients with an IDH1 mu­ta­tion who have been wait­ing for new treat­ment op­tions that work rad­i­cal­ly dif­fer­ent than con­ven­tion­al chemother­a­py,” said Schenkein.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.