Kim Stratton, Orphazyme

Near­ly two years af­ter their nar­row PhI­II fail­ure, Or­p­hazyme gets speedy look at the FDA

Or­p­hazyme’s bet that, de­spite miss­ing both pri­ma­ry end­points on its piv­otal study, they would find wel­com­ing ears at the FDA has paid off, for now.

The Dan­ish biotech an­nounced to­day that the FDA has ac­cept­ed the ap­pli­ca­tion for their ex­per­i­men­tal drug ari­mo­clo­mol in the rare neu­rode­gen­er­a­tive con­di­tion Nie­mann-Pick dis­ease Type C and giv­en it pri­or­i­ty re­view. Al­though drugs that fail piv­otal stud­ies rarely re­ceive a pri­or­i­ty look at the agency, the dis­tinc­tion should come with lit­tle sur­prise. Or­p­hazyme has sig­naled re­peat­ed­ly since the 2019 re­sults came out that the FDA viewed an ap­pli­ca­tion fa­vor­ably and, the FDA gave it break­through des­ig­na­tion that No­vem­ber.

The agency set a PDU­FA date for March 17, 2021. Or­p­hazyme’s stock jumped 8.38% on the news, from 83.5 kro­nes to 90.5 kro­nes ($13.3 to $14.3).

“We look for­ward to col­lab­o­rat­ing with the FDA as they com­plete their re­view of this NDA to ad­dress the un­met med­ical need in NPC, and mean­while are work­ing to ex­pand our U.S. ac­tiv­i­ties in prepa­ra­tion for po­ten­tial com­mer­cial avail­abil­i­ty next year,” CEO Kim Strat­ton said in a state­ment.

Or­p­hazyme, a one-drug com­pa­ny tar­get­ing a string of neu­rode­gen­er­a­tive dis­or­ders, came out it with re­sults from a 50-per­son Phase II/III study on Nie­mann-Pick type C pa­tients, their lead in­di­ca­tion, in Jan­u­ary 2019. Ari­mo­clo­mol failed to hit both pri­ma­ry end­points, both a stan­dard scale for as­sess­ing symp­toms in pa­tients, called the NPC Clin­i­cal Sever­i­ty Scale, and the Clin­i­cal Glob­al Im­pres­sion of Im­prove­ment, a self-rat­ing tool that the FDA re­quest­ed. It al­so failed sev­er­al sec­ondary end­points.

Still, the drug on­ly nar­row­ly missed sig­nif­i­cance on the first pri­ma­ry end­point, where they saw both a 74% re­duc­tion in dis­ease sever­i­ty and a p-val­ue of 0.0506. And the com­pa­ny not­ed that two pre-spec­i­fied sub­group analy­ses reached sig­nif­i­cance, with dis­ease pro­gres­sion slow­ing in pa­tients over the age of 4 and in pa­tients who had been tak­ing an Acte­lion drug some­times pre­scribed for the dis­ease. All end­points were against place­bo.

In a de­gen­er­a­tive con­di­tion with no sig­nif­i­cant treat­ments, Or­p­hazyme was will­ing to bet that was enough to take to reg­u­la­tors. So far, they’ve been proven right.

Or­p­hazyme, eye­ing funds for com­mer­cial­iza­tion, filed for its first US of­fer­ing ear­li­er this month, pen­cil­ing in an ini­tial fig­ure of $115 mil­lion for the raise. In ad­di­tion to their lead in­di­ca­tion, they al­so have piv­otal stud­ies on­go­ing for ALS and spo­radic in­clu­sion body myosi­tis, a pro­gres­sive mus­cle dis­or­der.

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.