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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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