Epizyme dis­cards a failed pro­gram for tazeme­to­stat as ex­ecs work to get an FDA hold lift­ed -- shares crater

Epizyme’s woes with its lead pro­gram for tazeme­to­stat just got much worse.

Robert Baze­more

The FDA stepped in and slapped a par­tial hold on their $EPZM lead drug — halt­ing en­roll­ment in clin­i­cal tri­als — close to three months ago af­ter a pe­di­atric pa­tient de­vel­oped a sec­ondary T-cell lym­phoblas­tic lym­phoma. To­day in its Q2 re­port Epizyme added that it is shut­ting down a top-ranked de­vel­op­ment pro­gram for dif­fuse large B-cell lym­phoma, DL­B­CL, af­ter con­clud­ing that it wouldn’t suc­ceed.

Their com­ment:

Epizyme has con­duct­ed an in­ter­im as­sess­ment of da­ta from this tri­al and con­clud­ed that the clin­i­cal ac­tiv­i­ty seen in these co­horts is not suf­fi­cient to war­rant fur­ther de­vel­op­ment of tazeme­to­stat in DL­B­CL as a monother­a­py or in com­bi­na­tion with pred­nisolone.

Two oth­er com­bi­na­tion stud­ies for DL­B­CL are con­tin­u­ing.

The bad news was held back in their re­lease un­til Epizyme CEO Robert Baze­more was able to tal­ly the up­beat things hap­pen­ing at the biotech. Re­searchers say they’ve been work­ing with the FDA on the hold, and ex­pect they are with­in close range of get­ting it lift­ed.

It didn’t help. Epizyme shares end­ed the day down 24%.

Epizyme is al­so lin­ing up an ap­pli­ca­tion for tazeme­to­stat for ep­ithe­lioid sar­co­ma, but has had to de­lay it un­til H1 2019 be­cause of the hold. And that won’t please their in­vestors, ei­ther.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Lund­beck sounds taps on an­oth­er CNS drug, re­treat­ing from a mine field still oc­cu­pied by a Mer­ck team

Lundbeck has snipped another clinical-stage branch of its CNS research, dumping a schizophrenia program after determining that their therapy would have no positive influence on the disease.

Designed originally as a 240-patient study, researchers set out in early 2019 to see if a homegrown drug dubbed Lu AF11167 could make it through a proof-of-concept study. The drug is a PDE10Ai inhibitor, targeting an enzyme which it said at the time offered a new pathway to retuning the body’s neurotransmitter dopamine. The big idea was that by hitting their target, the drug would modulate “dopamine D1 and D2 receptor-mediated intraneuronal signaling without binding to these receptors,” influencing negative symptoms of schizophrenia.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie aban­dons a pi­o­neer­ing CRISPR R&D al­liance with Ed­i­tas as Brent Saun­der­s' deal is cast out

A little more than 3 years ago Allergan paid $90 million in a cash upfront to partner with gene editing player Editas on a CRISPR alliance focused on the eye. The lead program centered on LCA10, a rare, inherited retinal degenerative disease that appears in childhood and leads to blindness.

Allergan then went to AbbVie $ABBV in a buyout, and the pharma giant has no interest in moving forward on the gene editing front. The company punted it all back to Editas Thursday, with the biotech $EDIT noting in a statement after the market closed Thursday that it is regaining all rights for its ocular medicines, including EDIT-101.

President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Le­vo Ther­a­peu­tics miss­es pri­ma­ry end­point in PhI­II tri­al of Prad­er-Willi drug — the lat­est set­back in a dis­as­ter-prone field

Marking yet another setback in the Prader-Willi Syndrome field, Levo Therapeutics failed to hit its primary endpoint in a Phase III study of intranasal carbetocin. But the biotech is now shifting its focus to the secondary endpoints in an effort to pluck victory out of the jaws of defeat.

The disorder, characterized by a false sense of starvation, is caused by the absence or deletion of a father’s chromosome 15. Illinois-based Levo’s potential therapy involves a selective oxytocin-receptor agonist.

Leonard Schleifer (AP Images)

Re­gen­eron adds more pos­i­tive PhI­II da­ta for its NGF pro­gram — but safe­ty is still a big con­cern

Two years after fasinumab hit its first late-stage bar, Regeneron is standing by its “high-risk, high reward” bet on the NGF antibody. But while new Phase III data solidified the potential reward, the risk is still threatening to blow it all up.

Regeneron execs and analysts alike now have their eyes set on a rival drug from Pfizer and Eli Lilly, whose fate at the FDA will likely set the scene for the class.