Boehringer In­gel­heim sharp­ens reti­nal strat­e­gy, scoop­ing up dry AMD drug from a Swiss team be­hind No­var­tis' Beovu

Boehringer In­gel­heim has inked a sec­ond deal for reti­nal dis­eases in a year — and this time it’s spe­cif­ic about which ail­ment to tar­get.

Chris­t­ian Leis­ner

In a deal worth up to $490 mil­lion (CHF 474.5 mil­lion), the Ger­man phar­ma has locked in an an­ti­body frag­ment-based drug for ge­o­graph­ic at­ro­phy from Zurich, Switzer­land-based CDR-Life. Boehringer al­so gets a li­cense to de­vel­op and com­mer­cial­ize oth­er com­pounds against the spe­cif­ic tar­get.

Per­haps bet­ter known as ad­vanced dry age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD), GA can re­sult in the ir­re­versible loss of reti­na and lead to blind­ness. Un­like wet AMD — of­ten a more se­ri­ous, though not mu­tu­al­ly ex­clu­sive, form of the dis­ease of­ten treat­ed by an­ti-VEGF drugs — there are no ap­proved treat­ments for the in­di­ca­tion.

Clive Wood

A num­ber of play­ers are look­ing to change that. Apel­lis Phar­ma has post­ed pos­i­tive Phase II da­ta for a C3 in­hibitor, and Bio­gen has bet on a sim­i­lar ap­proach at Cat­a­lyst Bio. Gy­ro­scope, J&J, and Bio­phytis have al­so en­tered the race with new ap­proach­es in­clud­ing cell and gene ther­a­pies.

CDR-Life has a track record go­ing for it, not­ed Clive Wood, Boehringer’s head of dis­cov­ery re­search. Chris­t­ian Leis­ner, the co-founder and CEO, man­aged tri­als for No­var­tis’ new wet AMD drug Beovu as a se­nior clin­i­cal leader and brought sev­er­al drug de­vel­op­ers with him to the new ven­ture. Both CSO Leonar­do Bor­ras and Rou­ven Bin­gel-Er­len­mey­er, the head of tech­ni­cal de­vel­op­ment, had stints at the phar­ma gi­ant fol­low­ing long ca­reers at ES­BAT­e­ch.

Zoom­ing out to the broad spec­trum of reti­nal dis­eases, Boehringer said it’s built a port­fo­lio of next-gen­er­a­tion ther­a­py ap­proach­es in var­i­ous stages of de­vel­op­ment up to Phase II, in­clud­ing di­a­bet­ic reti­nal dis­eases.

Last Sep­tem­ber it looped in In­flam­ma­some Ther­a­peu­tics to add a new drug de­liv­ery tech that turns drugs in­to a long-act­ing, biodegrad­able gel for­mu­la­tion.

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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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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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.

Prahlad Singh, PerkinElmer

PerkinElmer hits the check­book again, this time dol­ing out $260M for next-gen ther­a­py bioser­vices firm

When PerkinElmer iced a deal to pick up UK gene editing firm Horizon Discovery, it trumpeted its big move into next-gen therapeutics. Now, not content to sit on its laurels, PerkinElmer is dipping into the war chest again, this time for a firm specializing in cutting-edge bioservices.

Life sciences services giant PerkinElmer will shell out $260 million to acquire Lawrence, MA-based Nexcelom Bioscience, which offers clinical services for next-gen cell and gene therapies, immuno-oncology drugs and vaccines, the companies said Thursday.

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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).