FDA re­jects Nabri­va a sec­ond time; Nabri­va pins it on trav­el re­stric­tions

A lit­tle over a year af­ter the FDA is­sued Nabri­va its first CRL for the an­tibi­ot­ic Con­tepo, the agency has is­sued an­oth­er.

By Nabri­va’s ac­count, the agency’s rea­son­ing hasn’t changed. They again cit­ed un­re­solved man­u­fac­tur­ing is­sues at the com­pa­ny’s CMO fa­cil­i­ties in Eu­rope and asked for no new clin­i­cal da­ta. The prob­lem, Nabri­va said, was that be­cause of trav­el re­stric­tions, the agency wasn’t able to do an on­site in­spec­tion of the fa­cil­i­ties to con­firm that the is­sues raised last year had been fixed.

Be­cause nei­ther com­pa­nies nor the FDA re­lease CRLs, it’s im­pos­si­ble to con­firm the claim, but it wouldn’t be the first time trav­el re­stric­tions have ap­peared to in­ter­fere with the re­view process. The agency warned in May that re­view times could be de­layed. And ear­li­er this month, No­var­tis an­nounced that the de­ci­sion date on its MS drug Arz­er­ra had been pushed from June to Sep­tem­ber. Though the com­pa­ny didn’t spec­i­fy rea­sons for the de­lay, one in­vestor at their Q1 call not­ed pos­si­ble out­stand­ing man­u­fac­tur­ing is­sues in Eu­rope that Covid-19 could have made dif­fi­cult to re­solve.

The re­jec­tion is a blow to a com­pa­ny that, like oth­er an­tibi­ot­ic mak­ers, has al­ready been ail­ing for some time. Al­though the Irish biotech reached $12 a share in 2017 off of pos­i­tive Phase III re­sults for the lead an­tibi­ot­ic and al­though that drug would soon be ap­proved, their shares tum­bled as oth­er suc­cess­ful an­tibi­ot­ic mak­ers filed for bank­rupt­cy and in­vestors be­gan to re­al­ize the grim fi­nan­cial log­ic of an­tibi­otics: To pre­vent re­sis­tance, you give new drugs on­ly as a last re­sort; a new drug, how­ev­er promis­ing, can be­come fa­tal­ly low-vol­ume.

The lat­est re­jec­tion cut shares by 15% pre-mar­ket, but it had al­ready en­tered pen­ny-stock ter­ri­to­ry.

The drug, known chem­i­cal­ly as fos­fomycin, has been avail­able in many coun­tries out­side the US since the ear­ly 1970s. Though of­ten giv­en oral­ly, Nabri­va had sub­mit­ted an in­tra­venous for­mu­la­tion for ap­proval in com­pli­cat­ed uri­nary tract in­fec­tions. The com­pa­ny ac­quired it from Za­vante in 2018 for 8.2 mil­lion shares — at the time worth around $25 mil­lion — and $100 mil­lion in mile­stones.

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

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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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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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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.

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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UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).

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.

UP­DAT­ED: In­ter­nal Trump-era emails re­veal a pletho­ra of celebs, com­pa­nies vy­ing for FDA’s at­ten­tion

The FDA on Thursday publicly released a trove of about 500 pages of internal and heavily redacted emails, showing how celebrities like Dr. Oz and Laura Ingraham vied for the FDA’s attention on Covid-related issues that ultimately proved to be a waste of time.

Many of the conversations, obtained via the Freedom of Information Act, involve major Trump-era health officials who, in retrospect, made crucial and often ill-advised decisions on increasing access to the over-hyped hydroxychloroquine.

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