Sarep­ta plays nice with the FDA, sets out plans for sec­ond ac­cel­er­at­ed Duchenne pitch by year's end

At­tempt­ing to mend a rather tense re­la­tion­ship with reg­u­la­tors, Sarep­ta has wrapped up talks with the FDA on how it should move for­ward with its new Duchenne mus­cu­lar dy­s­tro­phy drug: golodirsen. With notes from the FDA in hand, Sarep­ta $SRPT says it plans to seek rapid ap­proval, with an NDA filed by the end of this year.

El­lis Unger

The Cam­bridge, MA-based com­pa­ny stirred some con­tro­ver­sy at the agency back in 2016, when the FDA ap­proved Sarep­ta’s first DMD drug Ex­ondys 51. Some reg­u­la­tors were skep­ti­cal of the drug’s ben­e­fit to pa­tients, in­clud­ing El­lis Unger, di­rec­tor of the Of­fice of Drug Eval­u­a­tions. At the time, Unger warned that Ex­ondys 51 was “es­sen­tial­ly a sci­en­tif­i­cal­ly el­e­gant place­bo.” He was over­ruled, how­ev­er, and the drug came to mar­ket lat­er that year. It’s now a de­cent rev­enue dri­ver for the com­pa­ny, bring­ing in $57 mil­lion in Q4 sales last year.

Now, Sarep­ta wants a new ap­proval for a sub­set of DMD pa­tients. The ex­per­i­men­tal drug golodirsen is de­signed to treat pa­tients whose con­di­tion is caused by an er­ror in the DNA se­quence called ex­on 53. Da­ta on the drug shows it in­creas­es dy­s­trophin, the pro­tein miss­ing in Duchenne, to 1.2% of what nor­mal pa­tients would pro­duce. That’s a big im­prove­ment from the ob­served ben­e­fit for pa­tients on Ex­ondys 51, which makes 0.28% of the nor­mal amount of dy­s­trophin.

Doug In­gram

Sarep­ta wants ac­cel­er­at­ed ap­proval of the drug, and the FDA ap­pears to be open to it — as long as the drug­mak­er lat­er pro­duces da­ta that cor­re­lates the bio­mark­er with re­al clin­i­cal out­comes in pa­tients. The com­pa­ny be­lieves its ex­ist­ing Phase III tri­al, Essence, will do just fine to sat­is­fy the FDA’s re­quire­ment, as it’s de­signed to mea­sure dystophin pro­duc­tion and im­prove­ments in clin­i­cal out­comes by com­par­ing golodirsen to a place­bo.

“Ob­vi­ous­ly, whether golodirsen will ob­tain ac­cel­er­at­ed ap­proval is a re­view de­ci­sion that will come af­ter the sub­mis­sion and re­view of our NDA,” said Sarep­ta’s CEO Doug In­gram in a state­ment. “But we great­ly ap­pre­ci­ate the will­ing­ness of the Neu­rol­o­gy Di­vi­sion to en­gage and pro­vide clear di­rec­tion to us on the steps nec­es­sary to sup­port an NDA sub­mis­sion for ac­cel­er­at­ed ap­proval.”

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.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

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

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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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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented 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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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.

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.