Bio­gen won’t ex­plain why FDA slapped down their bid for new Tysabri for­mu­la­tion

When it sub­mit­ted a sup­ple­men­tal Bi­o­log­ic Li­cense Ap­pli­ca­tion to the FDA, Bio­gen hoped they could of­fer pa­tients with mul­ti­ple scle­ro­sis an eas­i­er way of re­ceiv­ing and ad­min­is­ter­ing their ther­a­py Tysabri. But Thurs­day, it re­ceived a CRL deny­ing the ap­proval of its fil­ing, and it isn’t of­fer­ing any ex­pla­na­tion be­hind it.

Ma­ha Rad­hakr­ish­nan

The com­pa­ny turned down a re­quest for fur­ther ex­pla­na­tion from End­points News Fri­day.

“This re­sponse from the FDA does not af­fect the in­tra­venous ad­min­is­tra­tion of TYSABRI, a well-es­tab­lished high-ef­fi­ca­cy treat­ment with a well-char­ac­ter­ized safe­ty pro­file, which over the last 15 years has treat­ed more than 200,000 peo­ple world­wide with re­laps­ing MS,” CMO Ma­ha Rad­hakr­ish­nan said in a state­ment.

Bio­gen hopes to of­fer its drug through a sub­cu­ta­neous route of ad­min­is­tra­tion. In­jec­tions to treat MS were ap­proved in Eu­rope ear­li­er this month, though the drug was orig­i­nal­ly ap­proved in Eu­rope back in 2006. The in­jec­tions are dosed at 300 mg every four weeks by a health­care provider, with the goal of treat­ing pa­tients in a clin­i­cal set­ting be­yond in­fu­sion cen­ters.

Right now, it takes sev­er­al hours to have an in­fu­sion in a hos­pi­tal clin­ic. With sub­cu­ta­neous in­jec­tion, the time­frame will be short­er than an in­fu­sion, and can be ad­min­is­tered clos­er to a pa­tient’s home, like a hos­pi­tal or health cen­ter.

Bio­gen plans to pur­sue reg­u­la­to­ry fil­ings for Tysabri in ad­di­tion­al coun­tries, the com­pa­ny said in its re­lease, and it has been ap­proved in 80 dif­fer­ent coun­tries. The drug has proven to re­duce the for­ma­tion of new brain le­sions and cut re­laps­es.

Bio­gen’s hopes of ex­pand­ing the Tysabri fran­chise flopped in 2018, when plans to break it in­to is­chemic stroke failed in a Phase IIB tri­al. The elim­i­na­tion of the pro­gram height­ened pres­sure for Bio­gen to find suc­cess for the drug, which had been in tri­als to treat Alzheimer’s dis­ease.

The com­pa­ny agreed to a $22 mil­lion set­tle­ment with the De­part­ment of Jus­tice af­ter claims that it vi­o­lat­ed the False Claims Act by us­ing foun­da­tions to pay Medicare co­pays for pa­tients tak­ing 2 of its MS drugs: Tysabri and Avonex. The DOJ said that do­ing so vi­o­lat­ed the An­ti-Kick­back Statute, in­tend­ed to lim­it price hikes.

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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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.

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.”