Af­ter a $200M fi­nanc­ing, High­land Ther­a­peu­tics falls silent on AD­HD drug’s fate as PDU­FA sails by

David Lick­r­ish

Back at the be­gin­ning of this year, the folks at Toron­to-based High­land Ther­a­peu­tics cel­e­brat­ed a $200 mil­lion fi­nanc­ing com­plet­ed through Mor­gan Stan­ley. That cash, they said, would go to com­mer­cial­iz­ing their AD­HD drug HLD200 through their whol­ly owned sub­sidiary Iron­shore, tout­ing a drug they have fre­quent­ly her­ald­ed as a break­through in the field as it faced a Ju­ly 30 PDU­FA date. They even hired some Shire vets to help grab mar­ket share.

But that PDU­FA dead­line has come and gone and there’s no word on what the FDA has done with it. And the com­pa­ny CEO tells me there isn’t go­ing to be.

Con­tact­ed by phone, David Lick­r­ish told me ear­li­er to­day that “we’re still in dis­cus­sions with the agency.” Pressed that the FDA would ei­ther re­ject the drug, OK it or de­lay the de­ci­sion — pick one — Lick­r­ish told me I’d caught him at a bad time and he’d get back to me.

In an email, he stuck with no com­ment.

With re­spect to the fil­ing, as a pri­vate­ly held com­pa­ny, and for com­pet­i­tive rea­sons, we are not com­ment­ing on the sta­tus of our NDA.  Hav­ing said that our pack­age con­tained 10 tri­als in­clud­ing two piv­otal stud­ies which at­tempt to eval­u­ate AD­HD dur­ing both the ear­ly morn­ing rou­tine and evening rou­tine pe­ri­ods, us­ing rat­ing scales not cur­rent­ly in Clin­i­cal Out­comes and As­sess­ment Com­pendi­um.  Be­yond this we won’t be com­ment­ing fur­ther on the on­go­ing in­ter­ac­tions with the Agency.

I asked the FDA for com­ment, which has stuck with a strict no com­ment pol­i­cy of its own be­fore Scott Got­tlieb ar­rived and ad­vo­cat­ed pub­lish­ing the CRLs.

They haven’t changed — yet. Their re­sponse:

Please un­der­stand that in­for­ma­tion about an ap­pli­ca­tion is con­fi­den­tial un­less it has been pub­licly dis­closed by a spon­sor.

At the time of the fi­nanc­ing in Jan­u­ary, Craig Lewis, pres­i­dent of Iron­shore Phar­ma­ceu­ti­cals, cel­e­brat­ed the com­pa­ny’s loom­ing trans­for­ma­tion from an R&D com­pa­ny to a full-fledged op­er­a­tion with a com­mer­cial­iza­tion wing. HLD200, he said in a state­ment, was on its way to be­com­ing a stan­dard ther­a­py for AD­HD. He added: “Our com­mer­cial or­ga­ni­za­tion is be­ing pur­pose-built with this ob­jec­tive in mind.”

In the mean­time, the com­pa­ny al­so her­ald­ed the ar­rival of two Shire vet­er­ans — Bar­ry K. Her­man as SVP, head of med­ical af­fairs and Paul J. Casano­va as SVP of sales. Shire has a ma­jor AD­HD fran­chise.

I would sus­pect that af­ter all the buildup, an ap­proval would have been shout­ed from the roof tops. So there may well have been at least been a snag in their plans. Or worse. The way the rules work now, a pri­vate com­pa­ny is free to say what­ev­er it likes. Or noth­ing at all.

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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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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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.