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.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Brii Bio backs in­fec­tious dis­ease start­up while ink­ing deal for its lead TB drug, dou­bling down on an­tibi­otics

Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas.