Psy­che­del­ic re­search gains mo­men­tum, as ear­ly tri­al sug­gests mi­cro-dos­ing LSD is safe

Psy­che­delics have been long ne­glect­ed as the sub­ject of vig­or­ous sci­en­tif­ic re­search af­ter gov­ern­ments brand­ed them as il­le­gal he­do­nis­tic com­pounds with no ther­a­peu­tic po­ten­tial. But in re­cent years, de­spite tricky reg­u­la­tions, a resur­gence of in­ter­est from re­searchers has cul­mi­nat­ed in an FDA ap­proved ke­t­a­mine-de­rived de­pres­sion treat­ment, clin­i­cal tri­als test­ing the po­ten­tial of psilo­cy­bin in ‘mag­ic mush­rooms,’ and the set­ting up of a psy­che­del­ic re­search cen­ter at Johns Hop­kins.

The col­or­less, odor­less and taste­less drug, ly­ser­gic acid di­ethy­lamide (LSD) — or acid, as it is fond­ly known — is part of this re­search re­nais­sance. On Wednes­day, a small pri­vate­ly held com­pa­ny — Eleu­sis Ben­e­fit Cor­po­ra­tion — un­veiled da­ta from an ear­ly study in healthy old­er vol­un­teers that test­ed its mi­cro-dos­ing ap­proach with LSD. And if it all goes ac­cord­ing to plan — nev­er a sure thing in biotech — they’ve got plans to tar­get Alzheimer’s with the ap­proach.

Neilo­u­far Fam­i­ly

In the study, 48 vol­un­teers (mean age = 62.9 years) re­ceived ei­ther 5 μg, 10 μg, or 20 μg of LSD, or place­bo  — ad­min­is­tered in wa­ter — every four days in six ses­sions. Over­all, the LSD was well tol­er­at­ed, and the fre­quen­cy of ad­verse events was no high­er than the place­bo, the com­pa­ny said, while claim­ing this is the first ever pub­li­ca­tion of clin­i­cal study da­ta on mi­cro-dosed LSD.

PK da­ta showed that the half-life of the LSD dos­es was short. “So at 12 hours post-dose, there was no drug in the blood at any of the dos­es,” Neilo­u­far Fam­i­ly, the tri­al’s lead in­ves­ti­ga­tor, told End­points News. “And there al­so wasn’t any drug in the blood at base­line on the sixth dose.”

The da­ta sup­port fur­ther clin­i­cal de­vel­op­ment of LSD, whose psy­choac­tiv­i­ty is un­der­stood to be me­di­at­ed pri­mar­i­ly through the 5-HT2A re­cep­tor, Eleu­sis said. The com­pa­ny even plans to de­vel­op the drug to treat and pre­vent Alzheimer’s dis­ease, a field lit­tered with fail­ure and a pauci­ty of promis­ing ther­a­peu­tics in the late-stage pipeline.

But the brim­ming en­thu­si­asm comes with a healthy dose of skep­ti­cism. Crit­ics wor­ry that the bur­geon­ing psy­che­del­ic re­search could in­cen­tivize un­bri­dled use of non-phar­ma­ceu­ti­cal ver­sions of these drugs and that clin­i­cal tri­al da­ta could be cloud­ed by the fact that place­bo-con­trolled stud­ies are not nec­es­sar­i­ly dou­ble-blind­ed, be­cause it is far too easy to de­ter­mine which group of pa­tients have been giv­en a place­bo.

“The one thing that we did ex­pect — but is still re­mark­able — is the high place­bo re­sponse,” Fam­i­ly said. “Peo­ple were re­port­ing per­cep­tions of psy­choac­tive ef­fects, when lat­er on we found out they were on place­bo…but in any case, any per­cep­tions of psy­choac­tive ef­fects were very mild and they sub­sided by the end of the day, both in the ac­tive dose groups and the place­bo groups.”

Eleu­sis has a plan to hedge its Alzheimer’s bet, and to deal with the pesky prob­lem of di­ver­sion.

Be­fore div­ing in­to a Phase II ef­fi­ca­cy study in Alzheimer’s, the com­pa­ny is plan­ning an ear­ly-stage study with a com­pound — a “not-so-psy­che­del­ic” psy­che­del­ic sero­tonin 5-HT2A ag­o­nist — in oph­thal­mol­o­gy. At the mo­ment, the eye drug is at the pre­clin­i­cal stage of de­vel­op­ment.

Shlo­mi Raz

The Phase I tri­al, which is ex­pect­ed to kick off in ear­ly 2021, will pro­vide a key mech­a­nis­tic in­sight in­to how psy­che­delics could pre­vent neu­rode­gen­er­a­tion as­so­ci­at­ed with in­flam­ma­tion, Eleu­sis chief Shlo­mi Raz told End­points.

“The eye is a win­dow to the soul but al­so to the brain,” he said.”The reti­na, in par­tic­u­lar, gives us a very neat way of as­sess­ing how psy­che­delics could po­ten­tial­ly man­age neu­ro­pro­tec­tion, neu­roin­flam­ma­tion and pro­vides us a cost-ef­fec­tive proof-of-con­cept be­fore go­ing in­to — by all mea­sures —what seems to be the most ex­pen­sive type of clin­i­cal tri­al around, which is in Alzheimer’s dis­ease.”

The hope is to de­vel­op an LSD com­pound for ther­a­peu­tic use that can be used in the out­pa­tient set­ting, but psy­choac­tiv­i­ty is a risk that must be mon­i­tored, he said. The com­pa­ny says it is de­vel­op­ing a non­in­va­sive safe­ty mon­i­tor­ing tech­nol­o­gy that will be used in its clin­i­cal tri­als, and if the com­pound is ap­proved, for pa­tient use.

“In all cas­es, there’s a cal­cu­lus of safe­ty, ver­sus un­met need, and clin­i­cal util­i­ty,” he said.  “I think in the case of Alzheimer’s dis­ease, should we demon­strate that LSD in fact, is ef­fec­tive in slow­ing or halt­ing the pro­gres­sion of the dis­ease, then I think that there’s a clear jus­ti­fi­ca­tion for tak­ing that risk.”

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.