Tardy mar­ket­ing ap­pli­ca­tion fil­ing, tri­al fail­ure trou­ble Su­per­nus in­vestors

Two key up­dates sur­faced in CNS-fo­cused Su­per­nus Phar­ma­ceu­ti­cals third-quar­ter re­sults on Tues­day.

The drug­mak­er has not yet filed a mar­ket­ing ap­pli­ca­tion for its AD­HD drug SPN-812 — and a late-stage study test­ing an­oth­er ther­a­py, SPN-810, for use in im­pul­sive ag­gres­sion (IA) in pa­tients with AD­HD, has failed.

Shares of the Rockville, Mary­land-based com­pa­ny $SUPN — which al­so post­ed Q3 rev­enue and prof­it that un­der­whelmed Wall Street ex­pec­ta­tions — tum­bled more than 26% to $21.50 in Wednes­day pre­mar­ket trad­ing.

Based on pre­vi­ous man­age­ment com­men­tary,  Jef­feries an­a­lyst David Stein­berg said he had ex­pect­ed Su­per­nus to have filed the mar­ket­ing ap­pli­ca­tion for ‘812 by now. “Pre­vi­ous­ly, mgt had in­di­cat­ed a 2H19 fil­ing with good like­li­hood for launch dur­ing the crit­i­cal ‘back to school pe­ri­od’ next fall, when typ­i­cal­ly 40% of Rx switch­ing in AD­HD takes place.”

Su­per­nus said it would sub­mit the ap­pli­ca­tion this month.

The drug will now like­ly win ap­proval next Nov/Dec, with a launch in ear­ly 2021, Stein­berg pre­dict­ed. He cut his full-year 2023 sales es­ti­mate to $125 mil­lion from $173 mil­lion, not­ing that gener­ics for Su­per­nus’ an­ti-epilep­tic drug Tro­k­en­di are al­so ex­pect­ed to hit in 2023.

SPN-810, mean­while, was be­ing test­ed in a 325-pa­tient place­bo-con­trolled study in pa­tients aged 6 to 11. Two dos­es of the drug (18 mg and 36 mg) — which is a dif­fer­ent for­mu­la­tion of the ex­ist­ing an­tipsy­chot­ic molin­done hy­drochlo­ride — were eval­u­at­ed.

In the first tranche of the tri­al, the me­di­an per­cent re­duc­tion in the week­ly fre­quen­cy of im­pul­sive ag­gres­sion episodes from base­line for the 36 mg dose was 60% ver­sus the place­bo, which reg­is­tered at 48.6% — a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment (p= 0.029). How­ev­er, fol­low­ing the in­ter­im analy­sis, in the sec­ond stage of the study, the per­for­mance of the 36 mg arm dipped — ad­verse­ly im­pact­ing the com­bined re­sults and trig­ger­ing the study fail­ure.

“This was not a ma­jor sur­prise. We had pegged suc­cess at ~60%,” Stein­berg wrote on Wednes­day.

Over­all, 98% of the tri­al’s en­roll­ment was com­plete — but Su­per­nus is ceas­ing en­rolling more pa­tients un­til it an­a­lyzes the dataset. Mean­while, en­roll­ment in a sep­a­rate late-stage tri­al test­ing the drug in ado­les­cents has been paused un­til a fi­nal de­ci­sion is reached re­gard­ing the SPN-810 pro­gram in IA, the com­pa­ny said.

As of Sep­tem­ber 30, Su­per­nus had about $893 mil­lion in cash, cash equiv­a­lents, and se­cu­ri­ties.

“It’s pos­si­ble there could now be in­creased ur­gency for SUPN to ex­e­cute a trans­ac­tion tar­get­ing late-stage or on-mar­ket as­sets in neu­rol­o­gy/psy­chi­a­try. That said, mgt has been dis­cussing BD for sev­er­al years now and has been very dis­ci­plined in its ap­proach – re­fus­ing to over­pay for com­mer­cial as­sets and avoid­ing those that don’t meet its growth as­pi­ra­tions,” Stein­berg said.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edited cancer patients watch their treatments unfold, one of the first CRISPR companies is rejigging a major oncology deal.

Editas Medicine is amending its long-running collaboration with Celgene and their subsidiary Juno Therapeutics. The new deal will expand the focus of their work to cover a subset of immune cells that have become an increasingly hot target for immunotherapy: gamma-delta cells.

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%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Prakash Raman. Flagship

Flag­ship woos No­var­tis top deal­mak­er Prakash Ra­man in move to get the BD ball rolling ear­ly

Flagship Pioneering likes to be ahead of its times — so far ahead, perhaps, that it is often challenging to find partners for their startups while the scientific scaffolding is underway. But Prakash Raman is here to change that.

Raman, who most recently headed up business development at the Novartis Institutes for BioMedical Research, became Flagship’s first chief business development officer two weeks ago. By acting as a “central resource” for the 100 companies in the venture fund’s portfolio, he hopes to help entrepreneurs and management teams strategize about dealmaking to capture value beyond the near-term validation of their platform technologies, Raman told Endpoints News.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Bill Haney, Skyhawk

Cel­gene ex­ecs shell out $92M cash for a pair of R&D deals that will fit per­fect­ly in their new home at Bris­tol-My­ers

With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Reata's bar­dox­olone of­fers promise in pa­tients with rare kid­ney dis­or­der

After surprising Wall Street with positive data on its drug, omaveloxolone, in patients suffering from a notoriously hard-to-treat degenerative neuromuscular disorder last month, Reata Pharma on Monday unveiled pivotal results from a trial testing another drug, bardoxolone, in patients with a rare, genetic form of chronic kidney disease for which there exist no approved therapies.

Bardoxolone, like Reata’s other lead drug — omaveloxolone — is a small molecule engineered to bind to a gene called Keap1 to enhance the activity of the protein Nrf2 in order to defuse inflammation.

Am­gen ax­es 149 of its staff in Cam­bridge of­fice; Evotec, Mil­li­pore­Sig­ma en­ter re­search pact

→ Amgen has submitted a Worker Adjustment and Retraining Act (WARN) — a warning of impending mass layoffs 60 days in advance of the date — to the state of Massachusetts in the wake of the company’s exodus from the neurosciences R&D sector. David Reese, the company’s R&D chief, said at the time that the company is cutting ties in the field to focus on other undisclosed areas. In its WARN notice, the Cambridge-based company stated that 149 of its employees would be affected — among the total 180 being let go. The terminations will take effect on December 31, 2019.