Ar­cutis show­cas­es pos­i­tive PhI­II da­ta show­ing its top­i­cal cream scratched the sweet spot Ote­zla or steroids could­n't

The goal at Ar­cutis Bio­ther­a­peu­tics $AR­QT had been clear and straight­for­ward from day 1: to prove that it can take ex­ist­ing com­pounds against well es­tab­lished der­ma­tol­ogy tar­gets and make re­for­mu­la­tions that hit a sweet spot not yet scratched by any of its myr­i­ad block­buster, gener­ic or ex­per­i­men­tal ri­vals.

The first proof came Mon­day in the form of pos­i­tive topline re­sults from a pair of piv­otal Phase III tri­als. In­vestors re­act­ed cheer­ful­ly, send­ing the stock up 28.14% to $34.97.

ARQ-151, a top­i­cal cream of PDE4 in­hibitor rof­lu­mi­last (you may re­call it as the ac­tive in­gre­di­ent in As­traZeneca’s COPD drug Dalire­sp), beat place­bo in clear­ing plaque pso­ri­a­sis, Ar­cutis re­port­ed.

Mark Leb­wohl, the dean for clin­i­cal ther­a­peu­tics at Ic­ahn School of Med­i­cine at Mount Sinai, was a par­tic­i­pant in the tri­al. He high­light­ed a “pro­found im­pact on itch” and quick ef­fect as a key part of the da­ta.

“The im­pact of plaque pso­ri­a­sis ex­tends be­yond the se­ri­ous phys­i­cal bur­dens of the dis­ease, with many pa­tients ex­pe­ri­enc­ing ad­verse psy­cho­log­i­cal and so­cial ef­fects as well,” he not­ed in a state­ment. “The ex­ist­ing top­i­cal treat­ments pre­scribed to pso­ri­a­sis pa­tients have sig­nif­i­cant short­com­ings, which lead to dif­fi­cult trade-offs be­tween ef­fi­ca­cy, safe­ty, and tol­er­a­bil­i­ty.”

Patrick Bur­nett

Look­ing at the pri­ma­ry end­point, as as­sessed by the in­ves­ti­ga­tor, rof­lu­mi­last cream 0.3% had an IGA suc­cess rate of 42.4% com­pared to 6.1% on ve­hi­cle in DER­MIS-1; in DER­MIS-2, the dif­fer­ence was 37.5% ver­sus 6.9% (p<0.0001 for both tri­als). These pa­tients had their plaque pso­ri­a­sis cleared or al­most cleared and saw at least a 2-grade im­prove­ment from base­line.

The cream al­so hit all the sec­ondary end­points, in­clud­ing In­ter­t­rig­i­nous IGA (I-IGA) Suc­cess, Pso­ri­a­sis Area Sever­i­ty In­dex-75 (PASI-75), re­duc­tions in itch as mea­sured by the Worst Itch-Nu­mer­i­cal Rat­ing Scale and pa­tient per­cep­tions of symp­toms as mea­sured by the Pso­ri­a­sis Symp­toms Di­ary (PSD).

Among those who were giv­en rof­lu­mi­last cream, 90% com­plet­ed the full 8-week treat­ment — with on­ly 5 dis­con­tin­u­a­tions in DER­MIS-1 and 1 dis­con­tin­u­a­tion in DER­MIS-2 due to side ef­fects. None were se­vere.

But the re­al test ARQ-151 is fac­ing won’t be lim­it­ed to place­bo, and Ar­cutis knows it. In an in­vestor pre­sen­ta­tion, the Fra­zier-backed com­pa­ny con­tex­tu­al­ized its da­ta by com­par­ing them di­rect­ly against com­mon­ly pre­scribed drugs.

“These da­ta re­in­force our con­vic­tion that top­i­cal rof­lu­mi­last is ex­cep­tion­al­ly well suit­ed to ad­dress the un­met needs in the top­i­cal treat­ment of pso­ri­a­sis, of­fer­ing an ide­al com­bi­na­tion of ef­fi­ca­cy com­pa­ra­ble to the re­sults of pub­lished clin­i­cal stud­ies of high-po­ten­cy steroid/cal­cipotriene or high-po­ten­cy steroid/tazarotene com­bi­na­tion prod­ucts, the abil­i­ty to use the drug chron­i­cal­ly in any anatom­i­cal area, and a very fa­vor­able safe­ty and tol­er­a­bil­i­ty pro­file,” CMO Patrick Bur­nett said.

He ex­pects fil­ing an NDA in the sec­ond half of 2021, set­ting up the first in an an­tic­i­pat­ed se­ries of wide-rang­ing ap­provals in big-mar­ket in­di­ca­tions CEO Frank Watan­abe has mapped out.

So­cial: Frank Watan­abe, Ar­cutis CEO

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, 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.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

Look­ing to ce­ment its lead in packed MS mar­ket, Roche's Ocre­vus un­corks new da­ta in ear­ly-stage pa­tients

Among a positively jam-packed multiple sclerosis market, Roche’s Ocrevus has managed to stand out for what the Swiss drugmaker is calling the most successful launch in its long history. But in order to press its advantage, Ocrevus is looking to earlier-stage patients, and new interim data should help build its case there.

After 48 weeks on Roche’s Ocrevus, 85% of newly diagnosed primary progressing or relapsing MS patients without a history of disease modifying therapy posted no disease activity, including disease progression or relapse, according to interim data set to be presented this weekend at the virtual American Academy of Neurology meeting.

J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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