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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Tillman Gerngross (Adagio)

Till­man Gern­gross' Covid-19 an­ti­body moon­shot scores $336M with the help of new ace CFO. Is an IPO next?

Less than a year into its existence, serial biotech entrepreneur Tillman Gerngross’ antibody play Adagio has raced ahead into a pivotal trial for its lead drug for Covid-19 on the back of some very promising preclinical data. Now, crossover investors led by Peter Kolchinsky at RA are rolling up the Brinks truck — and that could spell an IPO in the offing for Adagio.

Adagio has bagged $336 million as part of a Series C round led by RA Capital to advance lead single-shot antibody ADG20 through a pivotal Phase I/II/III trial for the treatment of mild to moderate Covid-19 patients at high risk of infection, the biotech said Monday.

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UP­DAT­ED: New Kaiser analy­sis shows how lim­it­ing price ne­go­ti­a­tions to tar­get­ed drugs may bet­ter fo­cus up­com­ing leg­is­la­tion

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

Biotech's IPO raise ap­proach­es $5.5B as Nas­daq con­tin­ues to prove fruit­ful with 2 de­buts and three new fil­ings

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

It was another busy week in the biotech IPO market as the second quarter continues to churn out significant investment into the sector.

Recursion led the way with a $436 million raise on Friday, pricing its IPO at $18, the high end of its range. Our own John Carroll went in depth on that raise over the weekend. Also on Friday, preclinical cancer biotech Biomea Fusion debuted with a $153 million raise priced at its own high end of $17 per share. The two companies helped push the combined IPO raise for 2021 to nearly $5.5 billion.

When is a drug re­al­ly a de­vice? Court knocks down FDA ap­peal in try­ing to sort that grey area

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

Q1: A flood of in­vestor cash drove biotech's num­bers to new record highs, and the tor­rent of cash is mov­ing up­stream fast

If you thought biotech was booming last year, wait until you get a load of the numbers from Q1 2021.

On virtually every level, with one exception, the money engine was working around the clock in the first 3 months of this year. Venture capital has reached such a fever peak that the average B round now weighs in at an average mega-weight value of $100 million. The money flow is also finding its way to the mouth of the R&D river, where discovery work now merits the big bucks instead of cautionary seed funds.

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Jami Rubin (EQRx)

Ja­mi Ru­bin, once fa­bled for grilling bio­phar­ma ex­ecs, de­camps to head fi­nance at drug pric­ing dis­rupter

As Goldman Sachs’ top pharmaceutical analyst, Jami Rubin was known for asking the tough questions. Now, as she takes the lead on EQRx’s mission to rewrite the rules of drug pricing, we’ll see how good her answers are.

Rubin made the jump to biotech on April 5, becoming EQRx’s new CFO, the company said Monday. She’s coming from PJT Partners, where she’s been a partner providing strategic guidance for biotech and pharmaceutical companies for the last couple years. With EQRx’s recent $500 million Series B round in the books, it wouldn’t be a surprise if she was already lining up a public debut.

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Seagen gets Au­gust PDU­FA for Pad­cev ex­pan­sions; Adap­tate pulls in new cash for gam­ma delta T cell an­ti­bod­ies

Seagen is riding the wave of two new priority reviews straight to the FDA.

The Bothell, WA-based biotech and their partners at Astellas announced Monday that two supplemental BLAs for Padcev had been accepted by US regulators. FDA has set Aug. 17 as the PDUFA date for the reviews.

“With our recent regulatory submissions, we intend to provide the highest level of clinical evidence supporting Padcev use — overall survival data from a randomized Phase III trial — and expand availability in multiple countries where there is unmet medical need,” said Astellas oncology chief Andrew Krivoshik.