J&J racks up a fresh set of pos­i­tive piv­otal da­ta for de­pres­sion drug es­ke­t­a­mine — but ques­tions linger on safe­ty

J&J has tak­en an­oth­er big step for­ward in its quest to bring home a block­buster ap­proval for its nasal spray for­mu­la­tion of ke­t­a­mine as a new rem­e­dy for ma­jor de­pres­sion.

Just weeks af­ter the phar­ma gi­ant re­port­ed that es­ke­t­a­mine had es­sen­tial­ly aced short-term piv­otal stud­ies, re­searchers are back with ev­i­dence of its long-term ef­fi­ca­cy. But they will like­ly face care­ful scruti­ny over the sub­stan­tial side ef­fects spurred by the ther­a­py, in­clud­ing a set of se­ri­ous ad­verse events re­port­ed in the lengthy safe­ty study.

The ef­fi­ca­cy study fo­cused on 705 treat­ment-re­sis­tant pa­tients re­cruit­ed out of its Phase III pro­gram who were sta­ble af­ter 16 weeks of ther­a­py with es­ke­t­a­mine and an oral an­ti-de­pres­sant. Fol­low­ing an­oth­er stretch of treat­ment, the es­ke­t­a­mine com­bo proved sig­nif­i­cant­ly more ef­fec­tive in pre­vent­ing a re­lapse than a place­bo spray plus oral de­pres­sion drug.

About one in four (26.7%) of the sta­ble re­mit­ters in the es­ke­t­a­mine arm ex­pe­ri­enced a re­lapse com­pared to 45.3% of the place­bo group (p=0.003) — a strong­ly pos­i­tive out­come. It’s par­tic­u­lar­ly sig­nif­i­cant that the re­searchers were able to track longterm re­spons­es for a drug known for its ephemer­al im­pact.

The safe­ty da­ta are a lit­tle more com­plex. As seen ear­li­er, and com­plete­ly ex­pect­ed, there was a high rate of dis­so­ci­a­tion — 22.4%. That un­der­scores why J&J plans to have the drug in­fused in a clin­ic, with an ob­ser­va­tion pe­ri­od to make sure pa­tients were be­hav­ing nor­mal­ly be­fore they leave.

Es­ke­t­a­mine is a low dose of ke­t­a­mine, a par­ty drug and horse tran­quil­iz­er that’s of­ten abused. J&J here is try­ing to nav­i­gate its way through Phase III with an ac­cept­able for­mu­la­tion for a tough pa­tient pop­u­la­tion in a field known for wide­spread fail­ure.

The safe­ty study was open la­bel, with pa­tients aware they were get­ting the drug. Re­searchers re­port­ed:

Fifty-five (6.9%) pa­tients ex­pe­ri­enced 68 se­ri­ous treat­ment-emer­gent ad­verse events. Of these, five se­ri­ous treat­ment-emer­gent ad­verse events from four sub­jects were as­sessed by the in­ves­ti­ga­tor as es­ke­t­a­mine nasal spray-re­lat­ed. There were two deaths which the in­ves­ti­ga­tor de­ter­mined to be un­re­lat­ed to es­ke­t­a­mine nasal spray or oral an­ti­de­pres­sant use. Lab­o­ra­to­ry tests, phys­i­cal ex­am­i­na­tion, and nasal tol­er­a­bil­i­ty re­vealed no trends of clin­i­cal con­cern in pa­tients treat­ed with es­ke­t­a­mine nasal spray for up to 52 weeks. No clin­i­cal­ly mean­ing­ful changes in cog­ni­tion were found. No cas­es of in­ter­sti­tial or ul­cer­a­tive cys­ti­tis were re­port­ed.

I asked a com­pa­ny rep what the five se­ri­ous ad­verse events were, as it could be rel­e­vant to a reg­u­la­to­ry re­view. Her re­sponse: de­pres­sion, delir­i­um, anx­i­ety and delu­sion, sui­ci­dal ideation and sui­cide at­tempt.

J&J has list­ed es­ke­t­a­mine as one of its top late-stage de­vel­op­ment pro­grams, prep­ping ap­pli­ca­tions for both sides of the At­lantic. They’re not home free yet, but in de­pres­sion uni­form safe­ty and ef­fi­ca­cy is un­heard of, with reg­u­la­tors will­ing to bal­ance the risks with the ben­e­fits for a tough group to treat.

On that score, J&J is well ahead of the game.

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.

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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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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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.