An­oth­er old, cheap gener­ic is be­ing prepped for an FDA ap­pli­ca­tion — made over in­to a ‘new’ drug for AD­HD and nar­colep­sy

Alex Zwyer

Can an old, cheap gener­ic obe­si­ty drug pulled years ago from the US mar­ket be tweaked and made over in­to a brand new ther­a­peu­tic able to com­pete for a share of the multi­bil­lion-dol­lar AD­HD mar­ket?

NLS Phar­ma aims to find that out.

Mazin­dol was first de­vel­oped way back in the 1960s by San­doz as an im­me­di­ate-re­lease obe­si­ty drug de­signed to shed pounds fast. A lit­tle less than a decade ago, the FDA de­ter­mined that while the ther­a­py — sold as Sanorex —  had been pulled from the US mar­ket, it wasn’t due to safe­ty or ef­fi­ca­cy rea­sons, leav­ing it wide open to a gener­ic ap­pli­ca­tion from a dis­count sup­pli­er.

But now Swiss biotech NLS says it has tweaked the ag­ing gener­ic with a con­trolled re­lease for­mu­la­tion and has gath­ered promis­ing mid-stage da­ta to show how it can work as an al­ter­na­tive to the am­phet­a­mines used in cur­rent­ly mar­ket­ed AD­HD drugs.

Gre­go­ry Mat­ting­ly, an NLS study in­ves­ti­ga­tor, told Reuters that in a Phase II with 85 pa­tients the drug re­duced symp­toms of AD­HD by more than half, much bet­ter than the 15.8% rate of pa­tients in the place­bo arm.

NLS got its IND ap­proved for this drug — which acts like the brain chem­i­cal orex­in and is now dubbed NLS-1 — by the FDA last year, say­ing that it al­so had plans to de­vel­op the ther­a­py for rare cas­es of nar­colep­sy. And on Ju­ly 11, the biotech an­nounced that the FDA had fol­lowed up with an or­phan drug des­ig­na­tion for mazin­dol, which pro­vides a pack­age of in­cen­tives aimed at en­cour­ag­ing the de­vel­op­ment of new drugs.

NLS has tout­ed the drug as an al­ter­na­tive to the am­phet­a­mines used in drugs like Adder­all from Shire, able to es­cape the con­trolled sub­stance reg­u­la­tions that can make it hard­er to mar­ket these ther­a­pies. It is, in its orig­i­nal form, a stim­u­lant, in­creas­ing heart rate and blood pres­sure while tamp­ing down on ap­petite.

NLS CEO Alex Zwyer isn’t hid­ing just how old this drug is. In fact, he’s high­light­ed it as a def­i­nite plus in the com­pa­ny’s fa­vor. Zwyer not­ed in a re­lease last year:

Mazin­dol has been used off-la­bel in nar­colep­sy since the 1970’s, and it is our goal to make it avail­able to all nar­colep­tic pa­tients.

NLS still has reg­is­tra­tion stud­ies to com­plete be­fore it can an­gle for an ap­proval, but if it makes the last leg of the clin­i­cal jour­ney it has the po­ten­tial to re­mind peo­ple of Marathon, which took an over­seas gener­ic, hus­tled it through an ab­bre­vi­at­ed de­vel­op­ment plan for Duchenne mus­cu­lar dy­s­tro­phy and came up with an ap­proval to sell it for $89,000. The re­sult­ing scan­dal brought Marathon’s house down, and PTC Ther­a­peu­tics bought it for $140 mil­lion in cash. They’re pro­ceed­ing with the launch.

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.