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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.