Eli Lil­ly plots NDA as sec­ond JAK study backs baric­i­tinib for alope­cia area­ta; Al­ny­lam heads to FDA af­ter full vutrisir­an da­ta pan out

Eli Lil­ly has post­ed pos­i­tive da­ta from its sec­ond Phase III study test­ing its JAK in­hibitor baric­i­tinib in spurring hair re­growth among pa­tients suf­fer­ing from se­vere alope­cia area­ta.

In­ves­ti­ga­tors re­port­ed a dose-de­pen­dent re­sponse to the drug, with 35% of the sub­jects in the 4 mg arm achiev­ing 80% or more scalp hair cov­er­age against 22% in the 2 mg arm. That’s in line with the first Phase III read­out.

That leaves Lil­ly fol­low­ing up with a new drug ap­pli­ca­tion lat­er in the year, look­ing to cap­i­tal­ize on their break­through drug des­ig­na­tion.

Lo­tus Mall­bris

Yet to be seen, though, is what reg­u­la­tors will think re­gard­ing the safe­ty is­sues. The FDA barred the use of the 4 mg dose for rheuma­toid arthri­tis, wor­ried about the longterm safe­ty pro­file of the drug, which is shared by the en­tire class. Lil­ly had once in­sist­ed on us­ing the 4 mg dose for RA, to no avail.

Lo­tus Mall­bris, vice pres­i­dent of im­munol­o­gy de­vel­op­ment at Lil­ly, not­ed: “We look for­ward to dis­cussing with glob­al reg­u­la­tors da­ta from the BRAVE-AA clin­i­cal tri­al pro­gram for this im­por­tant po­ten­tial treat­ment, which could be the first ap­proved for peo­ple liv­ing with alope­cia area­ta.” — John Car­roll

Al­ny­lam heads to FDA af­ter full vutrisir­an da­ta pans out

Al­ny­lam is plow­ing ahead with its vutrisir­an pro­gram as it re­port­ed full da­ta from its Phase III study at AAN on Mon­day.

The da­ta con­firm the pre­vi­ous­ly re­port­ed topline re­sults from Jan­u­ary, show­ing vutrisir­an topped a his­tor­i­cal place­bo con­trol in re­duc­ing the symp­toms of polyneu­ropa­thy tied to a pro­tein fold­ing dis­or­der — transthyretin-me­di­at­ed amy­loi­do­sis — from base­line af­ter nine months. As such, the com­pa­ny has sub­mit­ted its NDA to treat the hered­i­tary ver­sion of the dis­ease, where the pri­ma­ry and sec­ondary end­points were al­so met.

Al­ny­lam had mea­sured the ex­per­i­men­tal drug against On­pat­tro, the com­pa­ny’s ap­proved RNAi ther­a­py for AT­TR. The treat­ments re­sult­ed in an av­er­age of a 17-point dif­fer­ence in a clin­i­cal test­ing scale, with the change fa­vor­ing vutrisir­an.

Should all go well with reg­u­la­tors, vutrisir­an stands ready to launch some­time in 2022. It would mark the fourth RNAi ther­a­py Al­ny­lam has pushed across the fin­ish line. — Max Gel­man

VC For­bion clos­es at hard cap as it rais­es $428 mil­lion for growth fund

In its fi­nal stage of fundrais­ing, For­bion has raised $428 mil­lion for its growth fund to fo­cus on in­vest­ing in late-stage Eu­ro­pean life sci­ence com­pa­nies, the com­pa­ny an­nounced Tues­day, and have an­nounced its first in­vest­ments.

For­bion has in­vest­ed in Ire­land-based Syn­Ox Ther­a­peu­tics, NewAms­ter­dam Phar­ma, of the Nether­lands and the UK’s Gy­ro­scope Ther­a­peu­tics, which filed for an IPO this week. Gy­ro­scope an­nounced in March that it was hus­tling through Phase II clin­i­cal tri­als for can­di­dates GT005, a gene ther­a­py for pa­tients with ge­o­graph­ic at­ro­phy.

These are the first three of be­tween 10-12 in­vest­ments in what the com­pa­ny calls “the most promis­ing Eu­ro­pean late-stage sci­ences com­pa­nies.” The ven­ture cap­i­tal firm is fo­cused on in­vest­ing in com­pa­nies that de­vel­op ther­a­pies for “ar­eas of high med­ical need.”

The fund — dubbed For­bion Growth Op­por­tu­ni­ties I — launched in Ju­ly 2020 and closed at the hard cap. In­vestors in­clude Pan­theon, Eli Lil­ly, Hori­zon Ther­a­peu­tics, the Bel­gian Growth Fund and New Waves In­vest­ments. — Josh Sul­li­van

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).