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.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Sanofi, Cel­lec­tis team up to pair Lem­tra­da with al­lo­gene­ic CAR-Ts; Bris­tol My­ers joins Evotec's dri­ve to ad­vance UK sci­ence

Sanofi is partnering up with Cellectis on their allogeneic CAR-Ts.

Cellectis struck a deal to use the French pharma’s drug alemtuzumab (Lemtrada, an MS agent) as part of its lymphodepletion regimen — used to sideline host immune cells and improve CAR-T cell expansion and persistence.

The Sanofi drug is already used in some clinical trials, and now the pharma giant is on the hook for clinical studies as they engage in negotiations over a commercial pact. — John Carroll

Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.