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 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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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.

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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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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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.

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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EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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