Scott Got­tlieb to bio­phar­ma: You want­ed us to help mod­ern­ize R&D, now go out and do it

FDA com­mis­sion­er Scott Got­tlieb is us­ing the fi­nal days of his brief term to scold the drug R&D world for fail­ing to keep up. Wide­ly praised for his work aimed at mod­ern­iz­ing the re­search tech and strate­gies used to ad­vance new drugs, Got­tlieb says that drug de­vel­op­ers aren’t tak­ing ad­van­tage of the new, more ef­fi­cient ap­proach­es to clin­i­cal work — and that needs to change.

It’s not just the drug mak­ers, says Got­tlieb. CROs, who do much of the heavy lift­ing in the clin­ic, share a con­sid­er­able por­tion of the blame.

“Un­for­tu­nate­ly, we’ve seen a con­tin­ued re­luc­tance to adopt in­no­v­a­tive ap­proach­es among spon­sors and clin­i­cal re­search or­ga­ni­za­tions,” not­ed Got­tlieb in a state­ment. “In some cas­es, the busi­ness mod­el adopt­ed by the clin­i­cal tri­al es­tab­lish­ment just isn’t com­pat­i­ble with the kind of pos­i­tive but dis­rup­tive changes that cer­tain in­no­va­tions can en­able. We ap­pre­ci­ate that sci­en­tif­ic and tech­ni­cal com­plex­i­ty is a re­al and on­go­ing chal­lenge, but in­dus­try and acad­e­mia al­so need to in­vest in and lever­age these ap­proach­es and de­vel­op new in­cen­tives that re­ward col­lab­o­ra­tion and da­ta shar­ing across the clin­i­cal re­search en­ter­prise.”

He goes on in a jar­gon-laden style:

New re­search par­a­digms are need­ed to break down bar­ri­ers be­tween re­al world da­ta and clin­i­cal re­search, so that ev­i­dence can be shared rapid­ly to im­prove both do­mains across a learn­ing health care sys­tem. For in­stance, more tri­als can in­cor­po­rate da­ta from elec­tron­ic health records, and adopt elec­tron­ic in­formed con­sent, to en­roll more pa­tients in clin­i­cal tri­als clos­er to where they live and work. This can re­duce bar­ri­ers to clin­i­cal tri­al par­tic­i­pa­tion and ac­cel­er­ate re­searchers’ abil­i­ty to ask and an­swer im­por­tant ques­tions.

Got­tlieb goes on with the lat­est in a se­ries of guid­ances aimed at stream­lin­ing R&D. The list in­cludes the use of com­put­er­ized sys­tems for over­sight, ad­vice on adding pa­tients to tri­als with more “chal­leng­ing con­di­tions,” to make tri­als bet­ter re­flect re­al world con­di­tions and more about avoid­ing an­i­mals in drug de­vel­op­ment work.

The in­dus­try has been cheer­ing Got­tlieb and the FDA on for this kind of work. Now Got­tlieb, who winds up his tenure at the agency at the end of this month, wants the bio­phar­ma world to put it to prac­tice.

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.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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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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.

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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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

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.

In­cyte’s PD-(L)1 in­hibitor head­ed for an ODAC show­down next month

The FDA’s Oncologic Drugs Advisory Committee will spend a half day on June 24 reviewing Incyte’s PD-(L)1 inhibitor retifanlimab as a treatment for locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) for those who have progressed on or who are intolerant of platinum-based chemotherapy.

The eighth PD-(L)1 entrant in January nabbed a priority review and an orphan designation from the FDA, which sets the agency’s final decision date as July 25.

Third time’s the charm as Heron wins FDA nod for non-opi­oid anes­thet­ic Zyn­relef

With an aim to reduce the use of opioids in the post-op setting, Heron Therapeutics on Thursday announced that it finally won FDA approval — after two prior CRLs — for its long-lasting local anesthetic, which is a combination of bupivacaine and the NSAID meloxicam.

Heron will price the drug, known commercially as Zynrelef, somewhere between 22% and 28% less than its direct competitor, Pacira Biosciences’ Exparel (bupivacaine liposome injectable suspension), Heron CEO Barry Quart told Endpoints News in an interview.