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.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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