House lines up biotech lol­lipops as sup­port grows for an epic 21st Cen­tu­ry Cures Act

Now that Don­ald Trump is head­ed to the White House with plans to gut Oba­maCare, the House has field­ed a new ver­sion of the 21st Cen­tu­ry Cures Act in a bid to push through a bill now that in­cludes fresh promis­es of an even speed­i­er and more re­spon­sive FDA. And now that the De­moc­rats and Re­pub­li­cans have reached an agree­ment on how to fund the mea­sure, pluck­ing cash from the car­cass of the Af­ford­able Care Act, there are good odds that the House and Sen­ate could reach an agree­ment on a fi­nal bill in the not too dis­tant fu­ture.

Ac­cord­ing to Kaiser Health News, more than 1,400 lob­by­ists have tak­en a crack — for and against — var­i­ous sec­tions of this bill. And that helps ex­plain why this com­pro­mise bill weighs in at an epic 944 pages.

Rachel Sachs, a pro­fes­sor at Wash­ing­ton Uni­ver­si­ty who’s been fol­low­ing this bill close­ly, notes that some of the orig­i­nal good­ies for drug and de­vice mak­ers have been re­moved, in­clud­ing one sec­tion that would have al­lowed de­vel­op­ers to cir­cum­vent the FDA’s ap­proval process.

So what’s in? A spe­cial man­date for the NIH to sup­port young sci­en­tists look­ing for first-time grants; the pri­or­i­ty re­view vouch­er pro­gram that the FDA hates; an ex­tra $500 mil­lion in fund­ing for the FDA to move drugs and de­vices along faster over the next 10 years, and so on.

Here’s a 44-page sum­ma­ry to help di­gest what’s com­ing. You’ll find this in the sec­tion on speed­ed drug de­vel­op­ment, a promise that Trump made in his post-elec­tion game plan. The bill:

• Es­tab­lish­es a re­view path­way at FDA for bio­mark­ers and oth­er drug de­vel­op­ment tools that can be used to help short­en drug de­vel­op­ment time and re­duce the fail­ure rate in drug de­vel­op­ment.

• Al­lows spon­sors of ge­net­i­cal­ly tar­get­ed or vari­ant pro­tein tar­get­ed drugs to re­ly on da­ta for the same or sim­i­lar tech­nol­o­gy from pre­vi­ous­ly ap­proved ap­pli­ca­tions by the same spon­sor.

• Does not al­ter the ex­ist­ing ap­proval stan­dards for drugs.

There have been some high-pro­file scan­dals this year to sug­gest that the FDA may have act­ed hasti­ly in ap­prov­ing drugs or get­ting them back in­to the clin­ic af­ter a safe­ty mishap, most re­cent­ly with Juno’s new clin­i­cal hold in the wake of more pa­tient deaths. Far from slow­ing down the march at the FDA to low­er the bar for bio­phar­ma, Con­gress is in­tent on hit­ting the gas and forc­ing reg­u­la­tors to go even faster.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

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

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.