Manny Simons, Akouos CEO (Harvard Business School via YouTube)

Add ear gene ther­a­py com­pa­ny Ak­ou­os to the ever-grow­ing list of IPOs amid Covid-19

For in­vestors look­ing to cash in on a bur­geon­ing ear ther­a­py space, the lat­est biotech an­gling for a pub­lic de­but could be mu­sic to their ears.

Fresh off a $105 mil­lion raise in March, ear gene ther­a­py com­pa­ny Ak­ou­os is look­ing for an­oth­er $100 mil­lion for a chance to dance on the Nas­daq well be­fore its lead prod­uct en­ters the clin­ic.

Hear­ing aids and cochlear im­plants do ad­dress ear dam­age caused by ge­net­ics, noise, ag­ing, or drugs, but noth­ing quite cures or in­deed tar­gets the bi­o­log­i­cal un­der­pin­nings of hear­ing loss — this is the gap Ak­ou­os and a hand­ful of oth­ers in the space want to bridge.

“I think some of the ear­ly ef­forts in the hear­ing space have been drawn to the largest af­fect­ed pop­u­la­tions where there hap­pens to be less clar­i­ty on the un­der­ly­ing bi­ol­o­gy mech­a­nism,” chief Man­ny Si­mons said in a pre­vi­ous in­ter­view with End­points News. “So we’re fo­cus­ing our at­ten­tion on forms of hear­ing loss that we feel are well-un­der­stood, well-char­ac­ter­ized, where we can po­ten­tial­ly ad­dress the un­der­ly­ing cause.”

The com­pa­ny’s lead ex­per­i­men­tal ther­a­py AK-OTOF is en­gi­neered to treat hear­ing loss due to mu­ta­tions in the gene that en­codes otofer­lin, a pro­tein that en­ables the sen­so­ry cells to ac­ti­vate au­di­to­ry neu­rons that car­ry elec­tron­i­cal­ly en­cod­ed acoustic in­for­ma­tion to the brain, which al­lows us to hear. Ak­ou­os plans to sub­mit an ap­pli­ca­tion to take the drug in­to hu­man stud­ies next year, and gen­er­ate ear­ly-stage da­ta in 2022.

Si­mons, who found­ed the com­pa­ny in 2016, ini­tial­ly flirt­ed with the idea of be­com­ing a mu­si­cian, grow­ing up play­ing the pi­ano and the trum­pet. He met his wife at a glee club at Har­vard. For his bach­e­lor’s de­gree, he had the op­por­tu­ni­ty to es­sen­tial­ly cre­ate his own course of study: to un­der­stand how the brain process­es mu­sic, on the ba­sis of imag­ing stud­ies. That path led to the ear — to de­ci­pher how sound is en­cod­ed in­to a neur­al im­pulse that can ex­tend deep in­to the brain.

Af­ter get­ting his first taste of en­tre­pre­neur­ship in the pro­lif­ic lab of drug de­liv­ery re­searcher Bob Langer, he got his bio­phar­ma train­ing wheels off with stints at Third Rock backed-Warp Dri­ve Bio and Voy­ager Ther­a­peu­tics (nei­ther of which were ear fo­cused). But when he learned that AAV vec­tors with po­ten­tial ap­pli­ca­tions for the ear were be­ing de­vel­oped in a lab­o­ra­to­ry at Mass­a­chu­setts Eye and Ear, Si­mons seized the op­por­tu­ni­ty to get a hear­ing-fo­cused gene ther­a­py com­pa­ny off the ground.

Af­ter se­cur­ing a sweet $7.5 mil­lion in seed fund­ing in 2018, Ak­ou­os scored $50 mil­lion in a Se­ries A round in 2018, led by 5AM and New En­ter­prise As­so­ci­ates.

Ak­ou­os, akin to some oth­ers in the gene and cell ther­a­py space, is in­vest­ing heav­i­ly in man­u­fac­tur­ing in­fra­struc­ture — hav­ing tak­en note that the com­plex man­u­fac­tur­ing process for these kinds of ther­a­pies has be­come some­thing of an Achilles heel in the field when it comes to adop­tion if the pro­duc­tion ap­pa­ra­tus is not up to scratch. For in­stance, the up­take of CAR-T ther­a­pies — No­var­tis’ Kym­ri­ah and Gilead’s Yescar­ta — un­der­whelmed ini­tial ex­pec­ta­tions, de­spite their abun­dant promise. The up­take of Kym­ri­ah was plagued by man­u­fac­tur­ing prob­lems, and de­spite No­var­tis’ at­tempt to ex­pand its ca­pac­i­ty, sales have dis­ap­point­ed com­mer­cial­ly, giv­ing Yescar­ta an edge in the mar­ket.

Ak­ou­os is build­ing its own in­fra­struc­ture to man­u­fac­ture vec­tors for its slate of ex­per­i­men­tal ther­a­pies, which al­so in­clude ge­net­ic med­i­cines for the most com­mon forms of hear­ing loss, such as age-re­lat­ed and noise-in­duced hear­ing loss. The com­pa­ny is al­so plan­ning on build­ing a plant to process gene ther­a­py batch­es to sup­port ac­tiv­i­ties through Phase I/II clin­i­cal tri­als for prod­uct can­di­dates be­yond AK-OTOF — part­ner Lon­za will help man­u­fac­ture AK-OTOF while it is shep­herd­ed through clin­i­cal de­vel­op­ment.

The com­pa­ny plans to list on the Nas­daq un­der the sym­bol ‘AKUS’ amid a broad­er rush of bio­phar­ma com­pa­nies that are mak­ing their way to the pub­lic mar­kets de­spite the dis­rup­tion of Covid-19. In­deed, in­vestor ap­petites have ap­peared seem­ing­ly in­sa­tiable giv­en the raft of splashy IPOs in re­cent weeks, in­clud­ing a $424 mil­lion de­but for a J&J-part­nered Chi­nese biotech Leg­end Biotech, mark­ing one of the largest pub­lic rais­es in biotech his­to­ry.

Mean­while, there are a host of ri­vals in the broad­er ear-fo­cused space. Al­so in Boston, Ak­ou­os’ home, is Deci­bel Ther­a­peu­tics, work­ing on re­gen­er­a­tion by tar­get­ing tiny hairs that grow in the in­ner ear to ad­dress con­gen­i­tal hear­ing loss or age-re­lat­ed bal­ance dis­or­ders. Fre­quen­cy Ther­a­peu­tics has a mid-stage hair cell re­gen­er­a­tion pro­gram us­ing prog­en­i­tor cells.

Across the At­lantic, UK-based Rin­ri Ther­a­peu­tics is work­ing on treat­ing hear­ing loss by trans­plant­i­ng ot­ic neur­al prog­en­i­tor cells in­to the in­ner ear. Am­s­ter­dam-based Au­dion Ther­a­peu­tics has a com­pound in-li­censed from Eli Lil­ly, which is de­signed to turn on a chem­i­cal switch to pro­duce new sen­so­ry hair cells from oth­er cells in the in­ner ear to im­prove hear­ing.

Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an­ti­body play­er

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer which has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.

Rajiv Shukla, Constellation Alpha Holdings

Can­del gets busy IPO week mov­ing with down­sized raise as Ra­jiv Shuk­la's third SPAC goes pub­lic

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

In a week that’s expected to see several biotechs price their IPOs, Candel Therapeutics got things kicked off Tuesday with a muted opener.

The company helmed by former GlaxoSmithKline vet Paul Peter Tak made its way to Nasdaq thanks to a $72 million raise, which was downsized by about 15% than originally anticipated, according to Renaissance Capital. Candel priced at $8 per share after initially seeking to launch in the $13 to $15 range.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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