Psy­che­del­ic drug re­search breaks out of deep freeze as re­searchers head for the clin­ic with am­bi­tious tri­al plans

David Nichols en­tered grad­u­ate school in 1969 with dreams of crack­ing LSD. He was a child of the 60s, his friends drop­ping tabs and go­ing on about the vi­sions they saw – the big bang, god, in­fin­i­ty pro­ject­ed – and how it changed them in ways on­ly ma­jor life events could. Nichols was go­ing to fig­ure out how it all worked.

David Nichols Pur­due

And then in 1970, Con­gress passed the Con­trolled Sub­stance Act. LSD, which the gov­ern­ment had stud­ied ex­ten­sive­ly for two decades, be­came a Sched­ule I drug. The dol­lars washed up. For 30 years, psy­che­delics be­came all but un­touch­able for clin­i­cal tri­als.

“The Con­trolled Sub­stances Act was passed, and I thought, ‘Oh, well there goes that [idea],” Nichols told End­point News.

Now half a cen­tu­ry lat­er, Johns Hop­kins Med­i­cine is open­ing up the na­tion’s first ma­jor cen­ter for psy­che­del­ic re­search, a $17 mil­lion, pri­vate­ly-backed bet that re­flects a 20-year-long sea change in how psy­che­del­ic re­search is viewed by clin­i­cians and the promise the drugs have shown in treat­ing a host of psy­chi­atric dis­or­ders. The Cen­ter for Psy­che­del­ic and Con­scious­ness Re­search at Johns Hop­kins Med­i­cine will al­so ex­plore the drugs’ im­pact on cre­ativ­i­ty and well-be­ing.

“Oh, it’s huge,” says Nichols, who as a pro­fes­sor at Pur­due Uni­ver­si­ty be­came one of the on­ly psy­che­del­ic re­searchers (he fo­cused on an­i­mals) and one of the top ad­vo­cates for re­open­ing clin­i­cal tri­als dur­ing the three-plus decades the drugs were al­most en­tire­ly mar­gin­al­ized. “Where mon­ey has been the ob­sta­cle, now mon­ey is no longer the prob­lem.”

Af­ter years in the side­lines, psy­che­delics  be­gan­to creep back in­to the phar­ma­ceu­ti­cal imag­i­na­tion in 2000, when Johns Hop­kins ob­tained ap­proval to ad­min­is­ter psy­che­delics to healthy hu­man sub­jects.

Sci­en­tists at a hand­ful of promi­nent in­sti­tu­tions have since en­tered the field, con­duct­ing tri­als on how psy­che­delics can treat long-in­tractable dis­or­ders – some­times with dra­mat­ic suc­cess: psilo­cy­bin (the chem­i­cal found in mag­ic mush­rooms) for ad­dic­tion and a slew of ail­ments, LSD for de­pres­sion, MD­MA for PTSD.

Ear­ly re­sults have ap­peared as a panacea to psy­chi­atric pa­tients who have gone years on SS­RIs and oth­er treat­ments with­out sig­nif­i­cant im­prove­ment and opened the door to a string of star­tups seek­ing to ad­vance and cap­i­tal­ize on the emerg­ing mar­ket. Re­searchers tout­ed the re­sults of one pair of NYU and Hop­kins stud­ies on psilo­cy­bin to treat “ex­is­ten­tial dread” in can­cer pa­tients as “mind-blow­ing,”

“I think we’re look­ing at a par­a­digm shift,” says Nichols, who found­ed the Heffter Re­search In­sti­tute in 1993 to ad­vo­cate for and even­tu­al­ly fund clin­i­cal tri­als. “I think it’ll be a rev­o­lu­tion­ary change for peo­ple who suf­fer from these dis­or­ders. We didn’t have any­thing that worked un­til now.”

Re­searchers char­ac­ter­ized the cen­ter as a sign of how main­stream the field has be­come and an an­nounce­ment that will on­ly le­git­imize it fur­ther.

But im­me­di­ate­ly, the mon­ey will fu­el sev­er­al clin­i­cal tri­als at Hop­kins – in­clud­ing those for opi­oid ad­dic­tion, Alzheimer’s dis­ease, PTSD and co-oc­cur­ring mood and al­co­hol prob­lems – equip­ping sci­en­tists with funds that re­main scarce for psy­che­del­ic re­search.

Al­most all psy­che­delics are still Sched­ule I drugs, mean­ing of­fi­cial­ly they have “no cur­rent­ly ac­cept­ed med­ical use” and the NIH will not fund stud­ies on them. The prob­lem has plagued the field since its resur­gence.

Al­bert Gar­cia Johns Hop­kins

“Usu­al­ly sci­ence is a lot of beg­ging for mon­ey,” Al­bert Gar­cia, who will lead the Hop­kins Alzheimer’s study, told End­points News, “and this means we won’t have to do that for a cou­ple years at least.”

The fu­ture be­yond clin­i­cal tri­als re­mains murky, though, and the ex­u­ber­ance of some re­searchers and ad­vo­cates has led to push­back by crit­ics of the sci­ence (psy­che­del­ic stud­ies are nec­es­sar­i­ly not dou­ble-blind) and those, in­clud­ing health writer and pro­po­nent of psy­che­del­ic re­search Michael Pol­lan, who say ad­vo­cates for mag­ic mush­room de­crim­i­nal­iza­tion send a mes­sage that the drugs are not dan­ger­ous.

“This is not some sort of mag­ic bul­let that will be solv­ing all these men­tal health prob­lems,” Gar­cia said.

In March, the FDA ap­proved its first psy­che­del­ic drug: Es­ke­t­a­mine for treat­ing de­pres­sion. And in 2017 and 2018, the agency grant­ed break­through sta­tus to an MD­MA pro­to­col for treat­ing PTSD and to UK start­up Com­pass Path­ways’ psilo­cy­bin pro­to­col for treat­ing de­pres­sion. But there’s been lit­tle in­di­ca­tion of change to the un­der­ly­ing fed­er­al law.

Sim­i­lar ques­tions re­main about pre­cise­ly how these drugs would come to mar­ket, es­pe­cial­ly as the field has caught the eye of in­vestors in Sil­i­con Val­ley and Eu­rope. The list of Hop­kins donors in­cludes TOMS founder Blake My­coskie and pop­u­lar en­tre­pre­neur Tim Fer­riss but oth­er in­vestors have fun­neled mon­ey in­to pri­vate, for-prof­it ven­tures like Com­pass and ATAI.

In­g­mar Gor­man

“Scal­ing and ac­ces­si­bil­i­ty of treat­ments are ques­tions on every­one’s mind at the mo­ment,” In­g­mar Gor­man, di­rec­tor of the Psy­che­del­ic Ed­u­ca­tion and Con­tin­u­ing Care Pro­gram at the Cen­ter for Op­ti­mal Liv­ing and co-prin­ci­pal in­ves­ti­ga­tor on a stage III MD­MA study for treat­ing PTSD, told End­points News in an email.

Psy­che­delics such as psilo­cy­bin won’t op­er­ate on the pill-a-day for­mu­la that dom­i­nates most cur­rent psy­chi­atrics. Rather, a pa­tient would go in a few times per year to re­ceive dos­es in a con­trolled en­vi­ron­ment with med­ical pro­fes­sion­als. It’s un­clear yet what the costs would be, though Nichols and oth­ers are op­ti­mistic wide­spread knowl­edge of how to man­u­fac­ture psilo­cy­bin, for in­stance, will make it dif­fi­cult to patent and keep prices down.

Eka­te­ri­na Malievska­ia Com­pass

Last year, Com­pass Path­ways made head­lines af­ter in­sid­ers ac­cused them in Quartz of try­ing to form a mo­nop­oly on the glob­al sup­ply of psilo­cy­bin. They de­nied the charges but said se­cur­ing a large sup­ply was es­sen­tial to their busi­ness mod­el of in­te­grat­ing their ther­a­pies sys­tems.

They told End­points they weren’t a psy­che­del­ic com­pa­ny, but sim­ply look­ing for the best ways to tack­le men­tal health prob­lems.

“Right now, I don’t see any­thing more promis­ing than re­search in psy­che­delics,” says CIO and co-founder Eka­te­ri­na Malievska­ia. “But things can change.”

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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