Laurence Reid (Third Rock)

Deci­bel Ther­a­peu­tics rais­es $82M as Lau­rence Reid looks to steer gene ther­a­py piv­ot

Lau­rence Reid could have picked a bet­ter time to take his lat­est CEO job.

It was Jan­u­ary 29 when Deci­bel Ther­a­peu­tics an­nounced they were bring­ing in the for­mer Al­ny­lam and Mil­len­ni­um ex­ec, and the coro­n­avirus was still a dis­tant threat, not even yet named. The job would have been tall enough with­out a pan­dem­ic; af­ter five years and $100 mil­lion, Deci­bel was prepar­ing to piv­ot its plat­form in a new di­rec­tion, work­ing to ex­pand on gene ther­a­py and re­gen­er­a­tion. Reid would need to raise the cash to do so.

Still, de­spite the ear­ly bumps of clear­ing out labs and fig­ur­ing out how to pitch an in­vestor via Zoom, Reid says they’ve weath­ered the storm. And to­day they an­nounced they’ve raised an $82 mil­lion Se­ries D led by Or­biMed, near­ly dou­bling their cap­i­tal to date and giv­ing Reid two years of run­way to get a plat­form they hope can trans­form hear­ing in­to the clin­ic.

“March, April, when we were still learn­ing so much, [there] was so much un­cer­tain­ty and a cer­tain de­gree of fear, so for any­one man­ag­ing peo­ple through that — for any com­pa­ny in any in­dus­try but cer­tain­ly for biotech — is a com­plete­ly unique chal­lenge,” Reid told End­points News.

“But I was re­al­ly im­pressed by my new col­leagues,” he said, not­ing they’ve got­ten labs and oth­er op­er­a­tions safe­ly back on track. “It’s been re­al­ly gal­va­niz­ing to see that ac­tu­al­ly.”

Deci­bel finds it­self among three ma­jor Boston area biotechs chas­ing cures for hear­ing dis­or­ders, next to the well-heeled gene ther­a­py up­start Ak­ou­os and the stem cell re­gen­er­a­tion de­vel­op­ers at Fre­quen­cy Ther­a­peu­tics. Ak­ou­os is ahead when it comes to gene ther­a­py, with a can­di­date near­ing the clin­ic to cor­rect hear­ing in pa­tients with mu­ta­tions in the OTOF gene.

Deci­bel spent years fo­cused on pre­vent­ing hear­ing loss, but they piv­ot­ed over the win­ter. Faced with what they char­ac­ter­ized as sur­pris­ing ad­vance­ments in ge­nom­ic and re­gen­er­a­tive tech­nol­o­gy and hav­ing failed to find a bio­mark­er that could let them run a pre­ven­ta­tive tri­al, they de­cid­ed to scrap key pro­grams and fo­cus on gene ther­a­pies that can re­store hear­ing loss.

De­vel­oped in part­ner­ship with Re­gen­eron, Deci­bel’s gene ther­a­py for the same pro­tein isn’t sched­uled to hit the clin­ic un­til 2022, but Reid tout­ed the ad­van­tage of the ba­sic sci­ence plat­form they built over the last five years.

“We’ve built this plat­form for in­te­grat­ing dif­fer­ent sin­gle cell ge­nom­ic tech­nol­o­gy, to look at DNA and RNA and splic­ing of RNA and we in­te­grate that to give us a com­plete mol­e­c­u­lar pic­ture of in­di­vid­ual cell types in the in­ner ear,” he said. They fo­cus that tech on the hair cells that trans­late sig­nals from the out­side world to the brain, and use gene ther­a­pies to re­store them. “It’s the com­bi­na­tion of those plat­form pieces that unique­ly de­fine Deci­bel,” he said.

Al­though they’re be­gin­ning with fix­ing a sin­gle gene in peo­ple with the OTOF mu­ta­tions, the longer term goal is to build cures for more gen­er­al hear­ing loss and bal­ance dis­or­ders. It’s an in­creas­ing­ly com­mon line among a sub­set of gene ther­a­py com­pa­nies, but first they’ll have to prove that they can just fix a sin­gle com­mon­ly dys­func­tion­al gene.

Reid said they now have enough mon­ey to get that pro­gram in­to the clin­ic. They’ll hope to fol­low with the broad­er ap­proach be­gin­ning at the end of 2022, grab­bing more cash as they do.

“We felt that giv­en where the com­pa­ny was, we want­ed to do a pri­vate fi­nanc­ing,” Reid said. “We’ll go to the pub­lic mar­ket when we have the need.”

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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