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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Ken Greenberg, SonoThera CEO

Gene ther­a­py goes acoustic as ARCH-backed biotech launch­es with ul­tra­sound gene de­liv­ery plat­form

After co-founding two biotechs off virus-based therapies, one for pain and one for cancer, Ken Greenberg decided to go in a different direction for his newest biotech, SonoThera.

Based out of San Francisco, SonoThera announced Monday morning that it raised $60.75 million to develop new gene therapies — but delivered by ultrasound, which Greenberg says can address the major challenges facing more conventional viral gene therapies.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.