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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Anthony Coyle (Pfizer via Youtube)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-Suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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