Verastem (VSTM)

[stockguard]

I now have to rethink my holding even though an FDA approval is likely.

https://endpts.com/ash17-verastems-abbvie-castoff-is-headed-for-the-fda-even-after-missing-overall-survival-goal/
#ASH17: Verastem’s AbbVie castoff is headed for the FDA — even after missing overall survival goal

by john carroll — on December 10, 2017 04:30 PM EST

PDF
PDF
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.

The best place to read end­points news? In your inbox.
Full-text daily re­ports for those who dis­cover, de­velop, and mar­ket drugs. Join 21,000+ bio­pharma pros who read End­points News by email every day.

free sub­scrip­tion
Bulletin Healthcare
RAPS Fundamentals
? Go back
In order to comment, you must be an Endpoints News subscriber. (It's free to subscribe.)

I'm already a subscriber I need to subscribe
Marcus Malkmus 4 hours ago
Just a short comment on the OS data: By the trial design it was virtually impossible to show am OS advantage, hence OS very unusually is only included as a secondary endpoint:
Taken together the DUO study (NCT02004522) and the follow-on crossover study (NCT02049515) make sure that almost every patient that progresses on either duvelisib or ofatumomab is then given the other treatment in the follow-on study.
In effect the OS data only say that there is no difference in OS whether a patient receives duvelisib followed by ofatumomab or the other way round.
This is both expected and actually a clinically useful result since it shows that doctors have more freedom in the sequencing of the two drugs without impacting OS.
Also CLL/SLL PFS is a very good predictor of OS, particularly in targeted therapy, hence approval on PFS data is a perfectly sensible regulatory response. This is much unlike the situation in some solid tumor data and certainly totally different from immunotherapies in solid tumors, where PFS becomes increasingly a suspect and for long tail rich treatments quite useless endpoint.



Reply
Dang T Huynh 17 hours ago
The data is quiet impressing since it was comparing with an active medication for CLL, instead vs. placebo. I believe if approved, it will be the only other oral drug for CLL besides Ibrutinib
Reply