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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.

Bristol Myers Squibb (Alamy)

CVS re­sumes cov­er­age of block­buster blood thin­ner af­ter price drop fol­lows Jan­u­ary ex­clu­sion

Following some backlash from the American College of Cardiology and patients, Bristol Myers Squibb and Pfizer lowered the price of their blockbuster blood thinner Eliquis, thus ensuring that CVS Caremark would cover the drug after 6 months of it being off the major PBM’s formulary.

“Because we secured lower net costs for patients from negotiations with the drug manufacturer, Eliquis will be added back to our template formularies for the commercial segment effective July 1, 2022, and patient choices will be expanded,” CVS Health said in an emailed statement. “Anti-coagulant therapies are among the non-specialty products where we are seeing the fastest cost increases from drug manufacturers and we will continue to push back on unwarranted price increases.”