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.”

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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WIB22: Chas­ing af­ter ever-evolv­ing sci­ence takes a drug hunter across the pond

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Like many scientists, Fiona Marshall would tell you that she loved the natural world growing up — going to look at crabs running around the beach near her childhood home, pondering about the tides. But one thing about biology, in particular, stood out: It was constantly changing, and changing very quickly.

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