Su­per­nus bags ear­ly-stage CNS drug for $15M up­front in re­newed epilep­sy R&D ef­forts

A few years in­to mar­ket­ing their two epilep­sy drugs, the neu­rol­o­gy and psy­chi­a­try ex­perts at Su­per­nus Phar­ma are go­ing back to the draw­ing board with a new as­set.

Su­per­nus $SUPN is pay­ing $15 mil­lion up­front to ac­quire Bis­cayne Neu­rother­a­peu­tics and their Phase I pro­gram, with de­vel­op­ment and sales mile­stones to­talling $73 mil­lion and $95 mil­lion re­spec­tive­ly. A low sin­gle dig­it roy­al­ty would al­so be due should the drug ever reach the mar­ket.

While the num­bers are small, it’s a con­sid­er­able win for Mi­a­mi-based Bis­cayne Neu­rother­a­peu­tics, which spun out of Bis­cayne Phar­ma less than two years ago with $3 mil­lion in fund­ing.

Steven Schachter

Their epilep­sy treat­ment — now called SPN-817 — is a syn­thet­ic form of hu­perzine A, a sup­ple­ment with roots in Chi­nese med­i­cine that peo­ple some­times take for mem­o­ry im­pair­ment. It in­hibits the acetyl­cholinesterase en­zyme, there­by reg­u­lat­ing a neu­ro­trans­mit­ter re­spon­si­ble for send­ing sig­nals to mus­cles called acetyl­choline.

“In ad­di­tion to its pow­er­ful an­ti­seizure ac­tiv­i­ty in pre­clin­i­cal mod­els of se­vere epilep­sy, BIS-001 has ex­hib­it­ed the cog­ni­tion-en­hanc­ing prop­er­ties seen with oth­er AChE drugs, but with much bet­ter cen­tral ner­vous sys­tem and sys­temic tol­er­a­bil­i­ty and safe­ty than cur­rent­ly avail­able agents,” Steven Schachter, a Har­vard pro­fes­sor who co-found­ed Bis­cayne, said at its launch. “At a min­i­mum, we are op­ti­mistic it will be de­void of the detri­men­tal ef­fects on cog­ni­tion seen with many ex­ist­ing antiepilep­tic drugs.”

Jack Khat­tar

Su­per­nus has a long way to go in prov­ing that op­ti­mism — and a num­ber of ri­vals to con­tend with. GW Phar­ma scored the very first cannabis-de­rived drug ap­proval weeks ago to treat Dravet syn­drome, the ex­act form of rare pe­di­atric epilep­sy that Su­per­nus plans to start with. Zo­genix is lin­ing up with their pitch for the same in­di­ca­tion with a ther­a­py backed by a slate of pos­i­tive Phase III da­ta.

But it’s al­so had some time to nav­i­gate the epilep­sy mar­ket with their ex­tend­ed re­lease pills Ox­tel­lar XR (ox­car­bazepine) and Tro­k­en­di (top­i­ra­mate), with which SPN-817 will be a “strong strate­gic fit” ac­cord­ing to CEO Jack Khat­tar.

The pri­or­i­ty now is to de­vel­op an ex­tend­ed re­lease oral dosage for the drug, while fin­ish­ing up a proof-of-con­cept in adult pa­tients with re­frac­to­ry com­plex par­tial seizures.

Oth­er pro­grams in Su­per­nus’ pipeline tar­get AD­HD, bipo­lar dis­or­der, and de­pres­sion.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.