Vicente Anido, Aerie CEO (University of West Virginia via YouTube)

Who needs pri­ma­ry end­points any­way? Aerie touts sil­ver lin­ings from failed PhI­Ib, plots PhI­II in dry eye dis­ease

Aerie’s dry eye dis­ease drug may not have met the pri­ma­ry end­points it’s cho­sen for a Phase IIb study, but that’s not stop­ping the biotech from pow­er­ing through Phase III — and steer­ing to­ward a field with plen­ty of ri­vals.

“While we did not achieve sta­tis­ti­cal sig­nif­i­cance at our pre-de­ter­mined pri­ma­ry end­points at Day 28, as a re­minder, this is a Phase 2b study where se­lect­ing pri­ma­ry end­points are not re­quired,” CEO Vi­cente Anido said in a state­ment.

In­stead, he high­light­ed how AR-15512, a TRPM8 ag­o­nist giv­en in the form of an eye drop, spurred sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments on “mul­ti­ple FDA-rec­og­nized symp­toms and signs” — things like oc­u­lar dis­com­fort, tear pro­duc­tion and eye dry­ness.

All of that, he added, points to a clear path to­ward ap­proval.

In­vestors aren’t so sure. Shares $AERI fell 17.83% af­ter the bells to $2.80.

Based on the re­sults, ex­ecs have de­cid­ed to bring the high­er con­cen­tra­tion of 0.003% BID in­to two Phase III tri­als that will stretch over three months.

With No­var­tis’ block­buster Xi­idra al­ready on the mar­ket, the FDA is in no rush to clear new treat­ments for dry eye dis­ease, re­ject­ing Kala’s ini­tial NDA and de­mand­ing a new Phase III be­fore fi­nal­ly ap­prov­ing its oc­u­lar cor­ti­cos­teroid, Ey­su­vis. But biotechs have ar­gued that the mar­ket re­mains un­tapped and there’s still need for more op­tions.

A to­tal of 369 pa­tients en­rolled in the COMET-1 study in­to one of three arms: AR-15512 (0.0014%), AR-15512 (0.003%) or AR-15512 ve­hi­cle. Dosed dai­ly over 84 days, they were eval­u­at­ed at days 1, 14, 28 and 84.

Un­usu­al for a da­ta read­out, Aerie didn’t pro­vide a p-val­ue on the pri­ma­ry end­points — or even men­tion them — for the topline. Ac­cord­ing to its post­ing on clin­i­cal­tri­, the pri­ma­ry out­come mea­sures of the COMET-1 study are im­prove­ment from base­line in ODS-VAS, or oc­u­lar dis­com­fort score on a vi­su­al ana­logue scale, and im­prove­ment on the anes­thetized Schirmer’s score. There was no word on how pa­tients fared on those two met­rics in a press re­lease.

In­stead, the com­pa­ny high­light­ed how the drug cleared the bar for oc­u­lar dis­com­fort at day 84 (p=0.028), SANDE (symp­tom as­sess­ment in dry eye) at days 14, 28 and 84 (p-val­ue be­tween 0.025 and 0.0005) and eye dry­ness at day 84 (p=0.03). It al­so re­port­ed pos­i­tive re­sults in signs like ef­fi­ca­cy af­ter the first dose, tear pro­duc­tion based on the unanes­thetized Schirmer’s score, con­junc­ti­val red­ness and oc­u­lar sur­face stain­ing.

Less than 3% of par­tic­i­pants dis­con­tin­ued the tri­al due to ad­verse events, Aerie added, and they con­clud­ed both for­mu­la­tions test­ed were safe and well-tol­er­at­ed.

Aerie will see what the FDA thinks of its plan in an end of Phase II meet­ing planned for ear­ly next year — al­though the rest of the world may not find out un­til much lat­er.

AR-15512 came to Aerie — which al­ready boasts of sev­er­al ap­proved drugs in glau­co­ma — via its 2019 buy­out of Spain’s Avi­zorex Phar­ma. The up­front pay­ment came in at $10 mil­lion, per­haps re­flect­ing the sales ex­pec­ta­tion.“We would look for AR-15512 to match the ef­fi­ca­cy of ap­proved dry eye dis­ease ther­a­peu­tics (in terms of both dry eye dis­ease signs and symp­toms) with peak sales of ~$230 mil (in­di­ca­tion would be worth $4). If ef­fi­ca­cy is sig­nif­i­cant­ly above on-mar­ket prod­ucts, our peak sales es­ti­mates could be ~$500 mil (in­di­ca­tion would be worth $9),” Mizuho an­a­lysts wrote in a re­cent note.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.