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.

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

Endpoints Premium

Premium subscription required

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

Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

Endpoints Premium

Premium subscription required

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

Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

Bris­tol My­ers claims win with CAR-T ther­a­py Breyanzi in leukemia

Bristol Myers Squibb is looking to expand Breyanzi into more indications — and the pharma’s newest data readout makes progress on that front.

The Big Pharma put out word Thursday that the CAR-T cell therapy met the primary endpoint of complete response rate compared to historical control in a subset of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) that were refractory to a BTK inhibitor and pretreated with a BCL-2 inhibitor.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,400+ biopharma pros reading Endpoints daily — and it's free.

Vu Truong, Aridis Pharmaceuticals CEO (Aridis/Nasdaq)

Aridis' mon­o­clon­al an­ti­body fails PhI­II, but plans for sec­ond tri­al any­way

Aridis Pharmaceuticals’ monoclonal antibody missed the bar in a Phase III test in ventilator-associated pneumonia caused by the gram-positive bacteria S. aureus, the company announced Wednesday. 

But Aridis is planning for a second Phase III study anyway once it discusses the findings with the FDA and the European Medicines Agency. Execs blamed recruitment challenges stemming from Covid-19 and Russia’s invasion of Ukraine for the miss, cutting their enrollment target in half.

Al Gianchetti, XyloCor CEO

Xy­lo­Cor wraps up PhII for heart dis­ease gene ther­a­py, plans for piv­otal tri­al

XyloCor Therapeutics says patients with heart disease who got its gene therapy could exercise for longer and had fewer chest pain attacks. The biotech announced it completed a Phase I/II trial of the gene therapy Thursday morning, and plans to move forward with a pivotal trial.

In the Phase II portion of the trial, 28 patients with angina (or chest pain) caused by coronary artery disease and who had no other treatment options were enrolled and were given the highest tested dose from the first part of the trial. Patients were followed for six months.