#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.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?