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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.

Paul Grayson, Tentarix CEO (Versant)

Phar­ma vet­er­ans re­group with $50M and a plan to dis­cov­er new mul­ti-specifics

While a horde of drugmakers develops bispecific antibodies to more directly target tumor cells — there were about 100 programs in or nearing clinical trials back in May — a new company is emerging to go one step further.

On Thursday, Tentarix Biotherapeutics unveiled a $50 million Series A round to support its next-gen multi-specifics platform. While the field has largely focused on bispecifics, which engage two targets, Tentarix believes its multifunctional programs have the potential to be even more specific, since more conditions must be met for potent activity to occur.

Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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