An­oth­er Langer-backed biotech breaks out of stealth mode, aims for the clin­ic with hear­ing loss ther­a­py

David Lucchi­no

Over the last 18 months the team at Fre­quen­cy Ther­a­peu­tics’ Kendall Square of­fice has been mak­ing qui­et progress with its pre­clin­i­cal work on a new ap­proach to cur­ing hear­ing loss, ex­plor­ing the po­ten­tial of a ther­a­peu­tic tech­nique that emerged out of the lab of MIT’s se­r­i­al sci­ence en­tre­pre­neur Bob Langer and his col­league Jeff Karp. To­day, though, the stealthy ramp-up is over as the biotech de­buts its man­age­ment team and R&D strat­e­gy.

David Lucchi­no, the CEO, tells me that it all got start­ed years ago when Langer, who’s been work­ing in the re­gen­er­a­tive med­i­cine are­na, brought up the re­search he was do­ing on prog­en­i­tor cells.

“There are 15,000 hair cells in your ear” which are need­ed to hear prop­er­ly, says Chris Loose, co-founder and CSO. Noise and oth­er fac­tors de­stroy that in­tri­cate bi­o­log­ic ma­chin­ery need­ed for hear­ing. But the prog­en­i­tor stem cells that give life to those cells are ly­ing dor­mant. And Fre­quen­cy plans to de­vel­op a ther­a­py that can spur them back in­to busi­ness.

That ap­proach — like jump start­ing a dead bat­tery to get a car back on the road — gave birth to a plat­form coined prog­en­i­tor cell ac­ti­va­tion (PCA), and a strat­e­gy to de­vel­op small mol­e­cules to re­store hear­ing. Fre­quen­cy be­lieves the plat­form has the kind of ver­sa­til­i­ty that can take it in­to oth­er dis­ease fields, such as reti­nal dis­eases, skin ail­ments or di­a­betes.

Loose and Lucchi­no, who both worked on Sem­prus Bio­Sciences, one of more than 30 Langer-in­spired biotech star­tups which was sold in 2012, are part of a 12-mem­ber team that’s do­ing the pre­clin­i­cal work. They plan to jump in­to hu­man stud­ies in about 18 months. And they’re ea­ger to prove that re­viv­ing dor­mant stem cells could be a much more ef­fec­tive and ef­fi­cient ap­proach than the more com­plex sci­ence be­hind stem cell ther­a­peu­tics.

This isn’t your clas­sic biotech start­up mod­el, ex­plains Chair­man Marc Co­hen, an­oth­er well-con­nect­ed biotech play­er drawn by Fre­quen­cy’s po­ten­tial. Co­hen had been chair­man at Acety­lon be­fore Cel­gene ac­quired the com­pa­ny, and is chair­man at C4, which launched with a $73 mil­lion round last year. He and his broth­er run Co­bro Ven­tures. And in­stead of round­ing up ven­ture back­ers, they’ve been as­sem­bling the team and start­ing the work with some non­tra­di­tion­al back­ing.

“We have brought to­geth­er a group of an­gels, or su­per an­gels, I worked with in the past to pro­vide the ini­tial fund­ing of the com­pa­ny,” says Co­hen. “That’s a group of peo­ple with a sim­i­lar vi­sion, and we’re look­ing for­ward to ad­vance this, get it in­to the clin­ic.”

“What’s nice about this space is that the end­points are crys­tal clear,” says Loose. “You don’t need ex­treme­ly long-term out­comes stud­ies. That’s a ben­e­fit.”

Fre­quen­cy is ex­pand­ing, and it’s al­so mov­ing away from Kendall Square and in­to a new fa­cil­i­ty Alexan­dria built in Woburn. But while it may be a bit fur­ther from the cen­ter of the biotech hub, you can ex­pect the biotech to turn up the vol­ume on its next stage of de­vel­op­ment.

You’re like­ly to hear them loud and clear.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMedO

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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Covid-19 roundup: Af­ter promis­ing US its sum­mer sup­ply, Gilead in talks with EU over remde­sivir ac­cess; First in­haled remde­sivir study un­der­way

It wasn’t lost on European journalists or European doctors that the “amazing deal” the Trump Administration said it signed with Gilead to buy up remdesivir meant that they would have severely limited access to one of only two drugs proven to treat Covid-19. “This is the first major approved drug, and where is the mechanism for access?” Andrew Hill, a research fellow at Liverpool University, told The Guardian. “Once again we’re at the back of the queue.”

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Covid-19 roundup: Mod­er­na sticks to Ju­ly for its Phase III as ru­mors swirl; Fol­low­ing US lead, EU buys up Covid-19 treat­ments

The Phase III might be delayed from its original early July goal, but Moderna says it will still kick off the pivotal study for what could ultimately be the first Covid-19 vaccine before the end of the month.

A day after Reuters reported that squabbling between the Cambridge biotech and government regulators had held up the trial by about two weeks, Moderna released a statement saying that they had completed enrollment of their 650-person Phase II trial and were on track to begin Phase III by the end of the month. The protocol for that study, which is meant to prove whether or not the vaccine can prevent people from becoming sick, has been finalized, they said.