News brief­ing: In­tel­lia hits clin­ic with CRISPR ther­a­py; Vi­for con­tin­ues to spend, pay­ing up to $340M for DGF pro­gram

An­oth­er CRISPR ther­a­py has en­tered the clin­ic. In­tel­lia Ther­a­peu­tics, the Cam­bridge gene edit­ing biotech, an­nounced to­day that they dosed their first pa­tient with a ther­a­py meant to treat transthyretin amy­loi­do­sis, a rare ge­net­ic dis­ease caused by a mu­ta­tion in a liv­er pro­tein.

Al­though a hand­ful of CRISPR ther­a­pies have al­ready en­tered the clin­ic at In­tel­lia and else­where, the new study is no­table for be­ing just the sec­ond study to in­ject CRISPR di­rect­ly in­to a pa­tient. Oth­er ap­proach­es, such as those meant to treat sick­le cell ane­mia, in­volve tak­ing cells out of a pa­tient, edit­ing them, and then in­fus­ing them back in.

Edit­ing cells in vi­vo, by con­trast, is more dif­fi­cult, as it’s de­pen­dent on the abil­i­ty to tar­get to CRISPR to the right part of the body. The liv­er, though, is one of the eas­i­est ar­eas to tar­get for any ge­net­ic ther­a­py. The eye is al­so a rel­a­tive­ly straight­for­ward tar­get, and Ed­i­tas test­ed an in vi­vo CRISPR treat­ment for blind­ness ear­li­er this year, mark­ing the first time the gene ther­a­py tool was used di­rect­ly in hu­mans.

In­tel­lia has plen­ty of com­pe­ti­tion for transthyretin amy­loi­do­sis. In the past few years, Pfiz­er, Io­n­is and Al­ny­lam have each de­vel­oped drugs proven to ame­lio­rate the con­di­tion. In­tel­lia, how­ev­er, is gun­ning for a cure. — Ja­son Mast

Vi­for con­tin­ues to spend, pay­ing up to $340M for DGF pro­gram

A few weeks af­ter shelling out $440 mil­lion to mar­ket a pru­ri­tus drug in the US, Vi­for is wheel­ing and deal­ing once again.

The Swiss com­pa­ny has agreed to li­cense and com­mer­cial­ize a pro­gram from An­gion Bio­med­ica for the treat­ment of de­layed graft func­tion (DGF) and car­diac surgery-as­so­ci­at­ed acute kid­ney in­jury. An­gion’s ex­per­i­men­tal drug, dubbed ANG-3777, will net the biotech up to $80 mil­lion in cash, eq­ui­ty in­vest­ment and clin­i­cal mile­stones.

An­gion can al­so re­ceive up to $260 mil­lion in reg­u­la­to­ry mile­stones, in­clud­ing if and when the drug scores ap­proval in the US and EU, as well as up to 40% in roy­al­ties on sales. Vi­for’s li­cense will ap­ply glob­al­ly ex­cept in Chi­na, Tai­wan, Hong Kong and Macau for all of ANG-3777’s nephrol­o­gy in­di­ca­tions.

While Vi­for will han­dle the mar­ket­ing for all ter­ri­to­ries where the drug could reach ap­proval, An­gion will con­tin­ue to run the clin­i­cal de­vel­op­ment pro­grams. The com­pa­nies will share re­spon­si­bil­i­ties for reg­u­la­to­ry fil­ings.

The com­pa­nies say that ANG-3777 was en­gi­neered to mim­ic the bi­o­log­i­cal ac­tiv­i­ty of the he­pa­to­cyte growth fac­tor, ac­ti­vat­ing crit­i­cal path­ways in the body’s or­gan re­pair process af­ter an acute in­jury. — Max Gel­man

In­ma­gene snags $21M Se­ries B, bring­ing to­tal raise to $40 mil­lion

Shang­hai-based In­ma­gene reeled in a $21 mil­lion Se­ries B to fu­el its mis­sion of in-li­cens­ing and de­vel­op­ing drugs in Chi­na.

The lat­est round, led by Ver­tex Ven­tures Chi­na, brings In­ma­gene’s to­tal raise up to $40 mil­lion. Ac­cord­ing to the com­pa­ny, two “en­gines” dri­ve its pipeline: in-li­cens­ing drugs from oth­er coun­tries that “fit Chi­na” to con­duct glob­al clin­i­cal tri­als, and con­duct­ing drug de­vel­op­ment. The com­pa­ny’s lead can­di­date, IMG-20, was cre­at­ed in part­ner­ship with Af­fi­body and Med­Im­mune, an As­traZeneca sub­sidiary. The can­di­date, which showed pos­i­tive safe­ty and ef­fi­ca­cy re­sults in tri­als with pso­ri­a­sis pa­tients, is about to en­ter glob­al reg­is­tra­tion tri­als, ac­cord­ing to In­ma­gene.

“We are grate­ful to Ver­tex, Panacea, Kun­lun, SCVC and oth­er in­vestors for their strong sup­port,” In­ma­gene chair­man and CEO  Jonathan Wang said in a state­ment. “This fi­nanc­ing should help strength­en In­ma­gene’s lead­ing po­si­tion in im­munol­o­gy drug de­vel­op­ment in Chi­na.” — Nicole De­Feud­is 

Day af­ter tak­ing poi­son pill, Apte­vo soars on ear­ly glance at bis­pe­cif­ic da­ta

Seat­tle-based Apte­vo Ther­a­peu­tics has adopt­ed a stock­hold­er rights plan — com­mon­ly called a poi­son pill on the Street — to fend off a po­ten­tial hos­tile takeover.

The move comes in the wake of Tang Cap­i­tal Part­ners’ dis­clo­sure that it now owns 54% of the biotech. Soon af­ter­wards, Apte­vo al­so tout­ed a sec­ond com­plete re­mis­sion re­port­ed from the Phase I tri­al of its CD123xCD3 bis­pe­cif­ic in acute myeloid leukemia, send­ing shares $AP­VO up 115.70% to $51.12.

Un­der the rights plan, share­hold­ers were giv­en one right for each share they own, which would be­come ex­er­cis­able on­ly if a per­son or a group ac­quires own­er­ship of 10% or more in a trans­ac­tion not ap­proved by Apte­vo’s board. The aim is to let stock­hold­ers pur­chase shares at dis­count, so as to ef­fec­tive­ly di­lute the own­er­ship of the hos­tile par­ty.

With­out nam­ing names, Apte­vo said it’s look­ing to pro­tect the com­pa­ny and its share­hold­ers from third par­ties that are not act­ing in their best in­ter­ests. — Am­ber Tong

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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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.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

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: 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).