Su­per­nus touts AD­HD Phase III da­ta, but re­sults ap­pear to fall short of ex­pec­ta­tions

CNS-fo­cused Su­per­nus Phar­ma­ceu­ti­cals $SUPN re­port­ed pos­i­tive da­ta from keen­ly watched twin stud­ies test­ing its non-stim­u­lant AD­HD drug in chil­dren on Thurs­day, but the re­sults sug­gest­ed the treat­ment does not have a leg up over an ex­ist­ing gener­ic drug on the mar­ket.

Shares of the Rockville, Mary­land based-com­pa­ny were down about 13% in ear­ly trad­ing.

Two dos­es of the drug SPN-812 were test­ed against a place­bo in each study —  100mg/200mg in study P301, and 200mg/400 mg in study P303.

In the 6-week P301 study, SPN-812 100mg and 200mg had an ef­fect size of 0.54 and 0.57 re­spec­tive­ly, the com­pa­ny re­port­ed, meet­ing the main goal of re­duc­ing symp­toms on an AD­HD rat­ing scale. And in the 8-week P303 tri­al, pa­tients re­ceiv­ing 200mg and 400mg had an ef­fect size of 0.46 and 0.49.

In 2002, Lil­ly’s $LLY Strat­tera was the first non-stim­u­lant med­ica­tion ap­proved for AD­HD. It went gener­ic last year and will di­rect­ly com­pete against SPN-812 if Su­per­nus’ drug is ap­proved. Strat­tera achieved an ef­fect size of 0.4-0.6 in tri­als.

Su­per­nus Chief Jack Khat­tar re­it­er­at­ed his en­thu­si­asm for the drug on Thurs­day: “We be­lieve these da­ta from the two piv­otal Phase III stud­ies…demon­strate that SPN-812 is a well-dif­fer­en­ti­at­ed nov­el non-stim­u­lant treat­ment op­tion for many chil­dren with AD­HD.”

An­oth­er mea­sure that might help dif­fer­en­ti­ate SPN-812 is on­set of ac­tion. In study P301, SPN-812 showed a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment over the place­bo as ear­ly as the first week, a feat the drug was un­able to re­pro­duce in study P303.

De­spite in­vestor dis­con­tent, Cowen’s Ken Cac­cia­tore sug­gest­ed the da­ta was en­cour­ag­ing: “What the Street ap­pears to be miss­ing to­day is that par­ents/care­givers have very lim­it­ed op­tions in the treat­ment of AD­HD and re­lat­ed be­hav­ioral dis­or­ders, and there­fore there is a re­al and large need to add to the treat­ment par­a­digm,” he wrote.

“SPN-812 com­pares fa­vor­ably with mar­ket-lead­ing non-stim­u­lant Strat­tera, but un­like Strat­tera SPN-812 has a much more rapid on­set of ac­tion (1 week ver­sus 4-6 weeks), as well as what ap­pears to be a clean­er safe­ty pro­file. De­spite Strat­tera’s lim­i­ta­tions, it reached $535 mil­lion in the U.S. be­fore go­ing gener­ic. Ad­di­tion­al­ly, the re­sults com­pare fa­vor­ably to Shire’s non-stim­u­lant In­tu­niv, which with­in on­ly a few years on the mar­ket was on a run-rate of $340 mil­lion in sales be­fore it went gener­ic.”

SPN-812 is al­so be­ing test­ed in ado­les­cents. One phase III study in this pa­tient pop­u­la­tion will read­out by the end of the year, while an­oth­er is ex­pect­ed by the first quar­ter of 2019. The biotech ex­pects to sub­mit a mar­ket­ing ap­pli­ca­tion for the drug in the sec­ond half of next year.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

Michel Vounatsos, Getty Images

UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.