#ASH17: Ve­rastem's Ab­b­Vie castoff is head­ed for the FDA — even af­ter miss­ing over­all sur­vival goal

When Ve­rastem $VSTM CEO Robert For­rester high­light­ed the pos­i­tive end­point from the piv­otal study of du­velis­ib a few months ago, he didn’t want to talk about the sec­ondary da­ta points. That, he said, was be­ing saved for a fu­ture sci­en­tif­ic con­fer­ence.

And to­day at ASH you can see the added da­ta isn’t some­thing you’d want to dis­cuss much.

Robert For­rester

The drug — which hit sta­tis­ti­cal sig­nif­i­cance on pro­gres­sion-free sur­vival — flunked the key over­all sur­vival sec­ondary, fail­ing to do any bet­ter than Arz­er­ra (ofa­tu­mum­ab) in ex­tend­ing the lives of pa­tients with re­lapsed or re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia/small lym­pho­cyt­ic lym­phoma.

The pos­i­tive da­ta that Ve­rastem is stick­ing with is for the me­di­an PFS: 13.3 months ver­sus 9.9 months for the con­trol. By the in­ves­ti­ga­tor as­sess­ment, there was an even bet­ter 17.6-month me­di­an PFS ver­sus 9.7 months for Arz­er­ra.

This is the da­ta that Ve­rastem is now hus­tling to the FDA, look­ing for an ap­proval to treat sec­ond-line cas­es. The p val­ue for the OS da­ta, p=0.4807, demon­strat­ed the drug group was no worse off, some­thing that was “like­ly due to oth­er avail­able ther­a­pies fol­low­ing pro­gres­sion,” ac­cord­ing to the com­pa­ny.

This was the drug that Ab­b­Vie $AB­BV paid $275 mil­lion up­front to part­ner on with In­fin­i­ty $IN­FI, but then walked away as the drug proved suc­cess­ful — but ex­treme­ly dis­ap­point­ing — in a Phase II study. For­rester picked up the rights to the drug for ex­act­ly noth­ing down as In­fin­i­ty re­struc­tured and re­fo­cused on some­thing else.

For­rester — and ex-In­fin­i­ty R&D chief Ju­lian Adams, for that mat­ter — be­lieve the drug can def­i­nite­ly win an ap­proval. And Ve­rastem be­lieves it can mar­ket the drug look­ing for peak sales of sev­er­al hun­dred mil­lion dol­lars a year.

Ve­rastem’s PR to­day out­lines the sales mod­el. Diep Le, Ve­rastem’s CMO, said:

CLL/SLL most­ly af­fects el­der­ly pa­tients and many are un­able or un­will­ing to be hos­pi­tal­ized or come in­to the clin­ic for fre­quent IV in­fu­sions. The CLL/SLL treat­ment land­scape there­fore is mov­ing away from chemother­a­pies and to­ward more tar­get­ed, prefer­ably oral reg­i­mens. While pa­tients are liv­ing longer many will be in­tol­er­ant to, or re­lapse fol­low­ing, their ini­tial ther­a­py em­pha­siz­ing the need for new op­tions. Oral du­velis­ib is the first PI3K in­hibitor to show ef­fi­ca­cy as an oral monother­a­py in a ran­dom­ized Phase 3 study in pa­tients with re­lapsed or re­frac­to­ry CLL/SLL and may of­fer an ap­peal­ing al­ter­na­tive for pa­tients who have pro­gressed or re­lapsed.

The NDA will be de­liv­ered in Q1.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.