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.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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