Lit­tle Neu­rotrope makes some big claims on an­oth­er failed Alzheimer’s study, but shares col­lapse

Daniel Alkon, Neu­rotrope

A low pro­file, small biotech named Neu­rotrope $NTRP teased out a pock­et of da­ta from a small Phase II Alzheimer’s study which its in­ves­ti­ga­tors her­ald­ed as ‘pos­i­tive.’

So why did its stock just plunge 53%?

Be­cause the study failed, like vir­tu­al­ly every­thing else that’s hap­pened in Alzheimer’s R&D over the last 15 years.

To get to its “pos­i­tive” nugget of da­ta, in­ves­ti­ga­tors nar­rowed the field to a sub­group of mod­er­ate to se­vere pa­tients who com­plet­ed all six low dos­es of Bryo­statin-1, de­signed to boost synaps­es in the brain that de­te­ri­o­rate rapid­ly in the dis­ease, wreak­ing hav­oc in the brain.

It wasn’t sta­tis­ti­cal­ly sig­nif­i­cant, but the sub­group re­spond­ed bet­ter than the place­bo arm on the Se­vere Im­pair­ment Bat­tery test used to eval­u­ate cog­ni­tion. Af­ter 12 weeks their score went up 1.5 points while the place­bo arm ex­pe­ri­enced a 1.1 point de­crease, a 2.6 point gap fa­vor­ing the drug.

The num­ber of “com­pleters,” though, was so small, the im­prove­ment so low and the time the drug was test­ed was so short that the da­ta nev­er­the­less in­di­cat­ed that the gap — such as it was — could be the re­sult of chance.

That’s a failed study. But Neu­rotrope be­lieves that hint of ef­fi­ca­cy needs to be ex­plored in a larg­er tri­al — not the first time that’s hap­pened in Alzheimer’s.

In­vestors weren’t in a gen­er­ous mood, though. Big phar­ma has done a bat­tery of tests of var­i­ous new drugs. Roivant’s Ax­o­vant is one of the fur­thest along in the clin­ic, look­ing for da­ta be­fore the end of this year on an­oth­er drug aimed at pre­serv­ing cog­ni­tion which GSK had tried, and failed, with. And Bio­gen has one of the most close­ly watched drugs — ad­u­canum­ab — that looks to mod­i­fy the course of the dis­ease, which is now in late-stage tri­als.

Bil­lions have been spent on failed tri­als in Alzheimer’s. Neu­rotrope just added a small con­tri­bu­tion of their own, adding some big spin in the process.

“These re­sults, which show im­prove­ment in pa­tients with mod­er­ate to se­vere Alzheimer’s dis­ease, the pop­u­la­tion that is gen­er­al­ly rec­og­nized as the most dif­fi­cult to treat, pro­vide ex­cit­ing ev­i­dence of a new ther­a­peu­tic ap­proach po­ten­tial­ly could re­ju­ve­nate synap­tic net­works in the brain. Im­prove­ments across the range of im­por­tant man­i­fes­ta­tions of the un­der­ly­ing neu­rode­gen­er­a­tive dis­ease, as shown in this Phase II study, could po­ten­tial­ly rep­re­sent a shift in the par­a­digm to treat Alzheimer’s dis­ease,” said Daniel Alkon, Pres­i­dent and Chief Sci­en­tif­ic Of­fi­cer of Neu­rotrope.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

David Arthur, Salarius Pharmaceuticals CEO

Salarius Phar­ma­ceu­ti­cals sees with­drawals, 3 of 13 pa­tient re­spon­ders in sar­co­ma tri­al

The Houston-based biotech Salarius Pharmaceuticals is lifting the cover on data from a Phase I/II trial for a drug currently on voluntary hold after a patient death, and the results appear to have underwhelmed investors.

Salarius’ candidate, dubbed seclidemstat, is an oral LSD1 inhibitor that is meant to treat Ewing sarcoma and FET-rearranged sarcomas in patients under 12 years old. The biotech had presented data with 13 patients with “first- and second-relapse Ewing sarcoma” who were treated in combination with topotecan and cyclophosphamide.