#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.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

For­bion spot­lights late-stage plays, carves out new €250M growth fund

Having staked its rep on picking out a mix of biotech investment opportunities across the “build,” “enable,” “growth” continuum, Forbion is launching its first fund dedicated to late-stage opportunities.

Forbion Growth Opportunities Fund’s first close brought in €185 million ($208 million). Existing investors Pantheon, KfW Capital and the European Investment Fund came on board, joined by new backers Eli Lilly, Horizon Therapeutics, Belgian Growth Fund and New Waves Investments.

Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.