Ex-Bio­gen ex­ec Steve Holtz­man hears the call of Third Rock’s am­bi­tious Deci­bel

Steve Holtz­man is one of the best con­nect­ed biotech ex­ecs in the Boston/Cam­bridge hub. He and George Scan­gos got to­geth­er and wooed Doug Williams in­to Bio­gen as the turn­around team’s R&D chief, with Holtz­man head­ing up cor­po­rate de­vel­op­ment. And long be­fore then, back in the mid-90s, he be­came a close col­league with Bob Tep­per, Mark Levin, Kevin Starr – the founders of Third Rock — and John Maraganore, all ex­ecs at Mil­len­ni­um, prob­a­bly the biggest sin­gle biotech ex­ec­u­tive mill in the big and rapid­ly ex­pand­ing hub city.

Steven Holtz­man, CEO Deci­bel Ther­a­peu­tics

Now, af­ter “re­tir­ing” from Bio­gen last fall, Holtz­man — who was the found­ing CEO at In­fin­i­ty Phar­ma­ceu­ti­cals — is fol­low­ing a trail that Doug Williams took last fall, jump­ing back to the helm of a biotech start­up and be­gin­ning the process of turn­ing it in­to a promi­nent new R&D or­ga­ni­za­tion, this time fo­cused on new drugs for hear­ing loss.

In part, this was his 24-year-old son’s idea. His mo­ti­vat­ing com­ment:

“Dad, you’re ap­proach­ing the 5th an­niver­sary of your 3-year Bio­gen plan, what are you go­ing to do next?”

What’s next turned out to be Deci­bel Ther­a­peu­tics, a re­cent start­up large­ly fi­nanced by his old friends and col­leagues at Third Rock, manned by some of those Mil­len­ni­um ex­ecs he teamed with more than 20 years ago.

Deci­bel got start­ed in style with a whop­ping $52 mil­lion ven­ture round last Oc­to­ber. And Holtz­man is think­ing big about its fu­ture.

To­day, Deci­bel is a com­pa­ny with a fair­ly stan­dard, start­up staff of 20. By the end of next year, says Holtz­man, it should be near­ing 100. And among his first tasks is flesh­ing out Deci­bel’s ex­ec­u­tive team.

“Not all mon­ey is green,” says Holtz­man, you need tra­di­tion­al VCs, crossovers and oth­ers “who share your vi­sion, as op­posed to look­ing for a quick ex­it.” He’s sat­is­fied that Third Rock has his back on that score.

“The idea of build­ing the 1,051st dis­cov­ery com­pa­ny didn’t in­ter­est me at this point,” says Holtz­man.

Deci­bel, though, was a dif­fer­ent mat­ter. In­stead of a biotech fo­cused on a pure un­met med­ical need, he was drawn in by the chance of de­vel­op­ing new ther­a­pies that could ad­dress a range of trig­gers for hear­ing loss, from the nar­row­ly de­fined pa­tient groups whose hear­ing loss can be trig­gered by spe­cif­ic drugs, to hear­ing loss due to an ex­po­sure to loud nois­es, or the sim­ple wear and tear of the years. He counts him­self in that last group, and a sis­ter suf­fer­ing from a pro­found con­gen­i­tal hear­ing loss has helped sen­si­tive him to the rest of the field.

With Deci­bel, the peo­ple were right, the mon­ey was right and the sci­ence was right, says Holtz­man. In this case, the biotech is pur­su­ing the role that neu­rotrophins play in restor­ing synaps­es and re­pair­ing hear­ing, with three key sci­en­tif­ic founders: Charles Liber­man at Har­vard Med­ical School, Gabriel Cor­fas at the Uni­ver­si­ty of Michi­gan, Scripps’ Ul­rich Müller, and Al­bert Edge from Mass­a­chu­setts Eye and Ear.

The goal now: “Giv­en that we’re right at the be­gin­ning of the whole un­fold­ing sto­ry of hear­ing ther­a­peu­tics, I don’t see why we can’t be the world’s lead­ing hear­ing ther­a­peu­tics com­pa­ny.”

It’s all ahead.

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.