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.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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