As with chil­dren stud­ies, Su­per­nus AD­HD drug shows fast on­set of ac­tion in ado­les­cents

Pos­i­tive piv­otal da­ta on Su­per­nus’ $SUPN AD­HD drug in chil­dren gave in­vestors pause ear­li­er this month be­cause of the lack of dose re­sponse at the high­er 400 mg dose, which weighed on the shares. On Thurs­day, the drug de­vel­op­er fol­lowed up with large­ly sim­i­lar Phase III da­ta from an ado­les­cent study, but the drug’s swift on­set of ac­tion, as seen in pre­vi­ous tri­als with chil­dren, may be a key dif­fer­en­tia­tor from ex­ist­ing AD­HD non-stim­u­lant med­ica­tions.

Two dos­es (200 mg/400 mg) of the drug, SPN-812, were test­ed against a place­bo in the 310-pa­tient ado­les­cent study P302. At week 6, pa­tients re­ceiv­ing SPN-812 200 mg and 400 mg had a -16.0 point change (p=0.0232) and a -16.5 point change (p=0.0091) on an AD­HD rat­ing scale, ver­sus -11.4 for those on the place­bo — meet­ing the main goal of the study. Over­all, pa­tients re­ceiv­ing 200 mg and 400 mg had an ef­fect size of 0.47 and 0.50, re­spec­tive­ly. Sig­nif­i­cant­ly, the high­er dose start­ed to work as soon as the first week, the com­pa­ny said.

Jack Khat­tar

“These da­ta fur­ther re­in­force the ef­fec­tive­ness of SPN-812 in pa­tients with AD­HD…with a fa­vor­able safe­ty and tol­er­a­bil­i­ty pro­file,” said CEO Jack Khat­tar in a state­ment. “We now have pos­i­tive da­ta prov­ing the ef­fi­ca­cy and safe­ty of SPN-812 in all AD­HD pa­tient pop­u­la­tions; pos­i­tive Phase III da­ta in chil­dren 6-11 years old and ado­les­cents 12-17 years old, and pos­i­tive Phase IIa da­ta in adults.”

An­oth­er Phase III study (P304) in ado­les­cents is ex­pect­ed to read out in the first quar­ter of 2019. But buoyed by the da­ta so far, Su­per­nus ex­pects to sub­mit a mar­ket­ing ap­pli­ca­tion by the end of the first quar­ter of 2019 and to launch — pend­ing FDA ap­proval — in the sec­ond half of 2020, it said on Thurs­day. The com­pa­ny’s shares edged up about 2.5% be­fore the bell.

In the first week of De­cem­ber, twin stud­ies eval­u­at­ing the ex­per­i­men­tal non-stim­u­lant drug in chil­dren showed sim­i­lar ef­fect sizes. In the P301 study, the 100 mg and 200 mg demon­strat­ed an ef­fect size of 0.54 and 0.57, re­spec­tive­ly. In the P303 tri­al, pa­tients re­ceiv­ing 200 mg and 400 mg had an ef­fect size of 0.46 and 0.49. Three out of the four dos­es were ob­served to have start­ed work­ing in week 1. But the com­pa­ny’s stock fell be­cause of the lack of dose-re­sponse seen with the high­er 400 mg dose, and wor­ries that the drug may have not per­formed well enough to dif­fer­en­ti­ate it­self from ex­ist­ing AD­HD drugs such as Lil­ly’s Stat­tera and Shire’s In­tu­niv.

Lil­ly’s $LLY Strat­tera — which achieved an ef­fect size of 0.4 to 0.6 in tri­als — was the first non-stim­u­lant med­ica­tion ap­proved for AD­HD, but it can take more than 6 weeks to start work­ing. Last year it went gener­ic, and is used for both chil­dren and ado­les­cents.

In a note ear­li­er this month, Jef­feries’ David Stein­berg sug­gest­ed that the over­all pro­file of SPN-812 was favourable. If ap­proved, it is set to reap peak sales of $400 mil­lion, he said.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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