Hear hear! Ear gene ther­a­py play­er Ak­ou­os draws $105M in fresh cap­i­tal

Come on, come on, lis­ten to the mon­ey talk.

Thriv­ing on the mo­men­tum of biotech­nol­o­gy com­pa­nies work­ing on ther­a­pies to ad­dress hear­ing loss, Boston-based Ak­ou­os has scored $105 mil­lion in an over­sub­scribed Se­ries B fi­nanc­ing, less than two years af­ter an NEA, 5AM Ven­tures-backed $50 mil­lion haul.

Man­ny Si­mons Ak­ou­os

Ak­ou­os — Greek for ‘to hear’ — is de­vel­op­ing gene ther­a­pies en­gi­neered to re­store, im­prove or pre­serve hear­ing. Along with its part­ners — the world’s largest hear­ing re­search cen­ter Mass­a­chu­setts Eye and Ear, as well as Swiss Alps-based vi­ral gene and cell ther­a­py mak­er Lon­za — Ak­ou­os is at the cusp of tak­ing its lead ther­a­py in­to the clin­ic.

The pro­gram, AK-OTOF, is de­signed to help in­di­vid­u­als with sen­sorineur­al hear­ing loss due to mu­ta­tions in the otofer­lin (OTOF) gene.

Led by Man­ny Si­mons, who con­sid­ered be­com­ing a mu­si­cian while at­tend­ing Har­vard, Ak­ou­os has an­oth­er Har­vard man on the ros­ter — co-founder and chief med­ical of­fi­cer Michael McKen­na. On Mon­day, the com­pa­ny added a third Har­vard voice to its board — Vic­ki Sato — who pre­vi­ous­ly served as pres­i­dent of Ver­tex Phar­ma­ceu­ti­cals. Heather Pre­ston of Piv­otal bioVen­ture Part­ners, which led the fresh cap­i­tal in­jec­tion, al­so earned a spot as di­rec­tor.

Oth­er in­vestors in the round in­clud­ed Cor­morant As­set Man­age­ment, Cowen Health­care In­vest­ments, EcoR1 Cap­i­tal, Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, Po­laris Founders Fund, Pags­group, Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), Wu Cap­i­tal, 5AM Ven­tures, New En­ter­prise As­so­ci­ates, No­var­tis Ven­ture Fund, Part­ners In­no­va­tion Fund, RA Cap­i­tal Man­age­ment and Sofinno­va In­vest­ments.

Michael McKen­na Ak­ou­os

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. Vy­ing to fill that gap, a pletho­ra of de­vel­op­ers are work­ing on ther­a­peu­tics in a bur­geon­ing field.

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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

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

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

David Coman, Science 37

Amid vir­tu­al tri­al craze, Sci­ence 37 earns uni­corn sta­tus and a trip to Nas­daq on the back of SPAC deal

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

As the Covid-19 pandemic made conventional trials impossible for some drugmakers, more and more companies moved to decentralize their clinical studies, accelerating business for tech developers like Science 37. Leveraging that boost, the company is on the verge of a SPAC merger, landing unicorn status and its very own stock ticker.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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