NEA, 5AM back a $50M play to steer gene ther­a­pies to the clin­ic at Ak­ou­os — the lat­est start­up in hear­ing

Hear­ing is get­ting to be a trendy R&D top­ic in biotech.

Eight months af­ter putting to­geth­er a $7.5 mil­lion seed round and lin­ing up AAV gene ther­a­py tech­nol­o­gy out of Mass­a­chu­setts Eye and Ear and Lon­za, the start­up Ak­ou­os has come back with a fast $50 mil­lion A round de­signed to get them through their pre­clin­i­cal phase and up to the thresh­old of their first hu­man study.

Michael McKen­na

Ini­tial­ly fo­cused on a rare ge­net­ic mu­ta­tion that leads to deaf­ness in new­borns, Ak­ou­os CEO Man­ny Si­mons tells me that the biotech has its eyes on a line­up of mono­genet­ic ail­ments re­lat­ed to hear­ing. And they’re look­ing to re­store hear­ing with a pipeline of ther­a­pies, even­tu­al­ly ex­pand­ing in­to broad­er caus­es of hear­ing loss for an ag­ing so­ci­ety.

“Where we’ve been fo­cused is mak­ing sure we are able to de­liv­er vec­tor to sen­so­ry cells through­out the cochlea in the hu­man ear,” the shell-like sphere where vi­bra­tions be­come sound, says Si­mons, a biotech vet with stints at Voy­ager — an­oth­er gene ther­a­py spe­cial­ist — and Warp Dive. This is his first turn run­ning a biotech, and his back­ground helped him line up a ma­jor league crew of back­ers.

Arthur Tzian­a­bos

5AM and New En­ter­prise As­so­ci­ates led the round, with Part­ners In­no­va­tion Fund step­ping in with new in­vestors:Sofinno­va Ven­tures, RA Cap­i­tal Man­age­ment and No­var­tis Ven­ture Fund.

Like most star­tups, the CEO at Ak­ou­os is stay­ing qui­et about their first tar­get for now. But af­ter hav­ing time to ex­plore their tech in an­i­mal mod­els, in­clud­ing non-hu­man pri­mates, he’s al­so ea­ger to note that their first pro­gram puts them in the open­ing stages of ex­plor­ing a field with 150 mono­genet­ic trig­gers for hear­ing loss.

Christo­pher Smith

Ak­ou­os is join­ing a rel­a­tive­ly small but grow­ing group of drug de­vel­op­ers fo­cused on hear­ing in a world where deaf­ness is be­com­ing in­creas­ing­ly com­mon.

Not far from where Ak­ou­os makes its home, Deci­bel has been pur­su­ing its own ap­proach to build­ing a tech plat­form for new drugs to re­store lost hear­ing. Just weeks ago Steve Holtz­man and his crew pieced to­geth­er a $55 mil­lion C round — though they’re de­vel­op­ing a pipeline of com­pounds. Just this morn­ing Fre­quen­cy Ther­a­peu­tics spot­light­ed a move in­to a Phase I/II study for a hear­ing restora­tion drug. A host of aca­d­e­m­ic groups, mean­while, have been look­ing to de­liv­er a gene ther­a­py for hear­ing via a vec­tor — as that field steadi­ly deep­ens its roots fol­low­ing a land­mark ap­proval for Spark.

Si­mons is ex­pand­ing his team with the new mon­ey. One of his sci­en­tif­ic founders at Har­vard, Michael McKen­na, is com­ing on as chief med­ical of­fi­cer. And he has some fa­mil­iar biotech/ven­ture ex­ecs join­ing the board: Arthur Tzian­a­bos, the CEO of Ho­mol­o­gy Med­i­cines, and Christo­pher Smith, for­mer CEO of Cochlear.


In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

CEO Harith Rajagopalan (Fractyl)

Af­ter a decade in the type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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An­oth­er busy week for IPOs be­gins with an off-the shelf cell ther­a­py play­er sniff­ing around uni­corn sta­tus

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

A flurry of biotechs are expected to hit Nasdaq this week, with two companies, Ambrx Biopharma and Century Therapeutics, setting the terms for their public debuts, with expected raises at $126 million and $200 million, respectively. Alzamend Neuro is also joining in with a $12.5 million raise and two preclinical Alzheimer’s treatments in tow.

Alexander Vos, VectorY CEO

Start­ing fresh in man­u­fac­tur­ing, For­bion start­up re­fu­els to steer next-gen gene ther­a­py ap­proach for ALS, Alzheimer's in­to clin­ic

Forbion laid out its case for a next-generation gene therapy approach when it took the wraps off VectorY Therapeutics and its vectorized antibody tech in February. Now, the Dutch VC has tapped an experienced hand at cell and gene therapy manufacturing to steer the ship — and pulled a marquee syndicate for a €31 million ($37.6 million) seed round.

Alexander Vos, the new CEO, is a venture partner at BioGeneration Ventures and jumps immediately from VarmX, a BGV portfolio company developing an anticoagulant. But before that, he had led Dutch CDMO PharmaCell for eight years until it was bought out by Lonza.