Psilocybin mushrooms (via The Denver Post)

In a key step for psy­che­del­ic re­search, mag­ic mush­room com­pound clears first clin­i­cal safe­ty hur­dle

Ex­as­per­at­ed with the of­ten-in­ef­fec­tive ex­ist­ing slate of an­ti­de­pres­sants, COM­PASS Path­ways set up shop in Lon­don 2016 — and made a bee­line for psilo­cy­bin, the psy­choac­tive in­gre­di­ent in mag­ic mush­rooms.

On Wednes­day, the start­up said its man-made ver­sion of the chem­i­cal — which is il­le­gal across ge­o­gra­phies in its nat­ur­al fun­gi form — had been well-tol­er­at­ed in an ear­ly-stage, place­bo-con­trolled tri­al in 89 healthy vol­un­teers.

Al­though pre­vi­ous re­search sup­ports the use of psilo­cy­bin in re­liev­ing symp­toms of de­pres­sion, small­er stud­ies are not al­ways place­bo-con­trolled. The tri­al test­ing the COM­PASS com­pound is the largest con­trolled study of psilo­cy­bin to date, said the study’s lead in­ves­ti­ga­tor, James Ruck­er of King’s Col­lege Lon­don’s In­sti­tute of Psy­chi­a­try, Psy­chol­o­gy & Neu­ro­science, in a state­ment.

Ex­ist­ing an­ti­de­pres­sants typ­i­cal­ly come in a pill form, and take weeks to kick in. J&J’s phar­ma­ceu­ti­cal con­coc­tion of ke­t­a­mine — the no­to­ri­ous par­ty drug that is al­so a horse and cat tran­quil­iz­er — was ap­proved ear­li­er this year, comes in the form of a nasal spray.

Tra­cy Che­ung

In the COM­PASS tri­al, vol­un­teers were ran­dom­ized to re­ceive a 10 mg or 25 mg dose of the syn­the­sized chem­i­cal (en­cap­su­lat­ed in a pill) or giv­en place­bo. Once dosed, vol­un­teers were giv­en in­di­vid­ual sup­port from ther­a­pists in groups of six in ses­sions that last­ed up to six hours.

The pa­tients who got the drug will most like­ly have ex­pe­ri­enced some psy­che­del­ic ef­fect, which is why the ther­a­pists were avail­able on hand, COM­PASS’ chief com­mu­ni­ca­tions of­fi­cer Tra­cy Che­ung not­ed in an in­ter­view with End­points News.

“Some­times that can be a lit­tle bit fright­en­ing or a lit­tle bit in­tense and the ther­a­pist is just there to hold your hand if that’s what’s re­quired and just pro­vide some sup­port or just to kind of say it’s all right, I’m here.”

No se­ri­ous ad­verse events emerged, and the most com­mon side ef­fects — as ex­pect­ed — were in the psy­che­del­ic realm, in­clud­ing changes in sen­so­ry per­cep­tion. The com­pound, dubbed COMP360, al­so had no im­pact on cog­ni­tive and emo­tion­al func­tion­ing, the com­pa­ny said.

COM­PASS is al­so con­duct­ing a Phase II tri­al test­ing its psilo­cy­bin com­pound in 216 pa­tients with treat­ment-re­sis­tant de­pres­sion across sites in North Amer­i­ca and Eu­rope. The com­pa­ny ex­pects to re­port da­ta from this study, which does not in­clude a place­bo arm, by ear­ly 2021.

Psilo­cy­bin is a sub­stance that in most re­gions is clas­si­fied as hav­ing no med­i­c­i­nal val­ue, falling in the same cat­e­go­ry as chem­i­cals such as LSD.

“At the mo­ment…we can use it but there’s an aw­ful lot of pa­per­work and we have to get li­cens­es for each of the coun­tries that we’re work­ing on do­ing the clin­i­cal tri­al,” Che­ung said, not­ing that the com­pa­ny has raised £28 mil­lion so far to in­ves­ti­gate the drug.

Psy­choac­tive in­gre­di­ents, whether de­rived from cannabis, LSD or mag­ic mush­rooms, have long cap­ti­vat­ed men­tal health re­searchers. Nav­i­gat­ing the com­plex le­gal hur­dles to ac­cess these com­pounds has thawed the pace of re­search but with mo­ti­vat­ed sci­en­tists and a grow­ing bur­den of poor­ly treat­ed men­tal health con­di­tions, the ecosys­tem of psy­che­del­ic re­search has ex­plod­ed. In Sep­tem­ber, John Hop­kin’s un­veiled it had scored $17 mil­lion to open its very own cen­ter of psy­che­del­ic re­search to ex­plore the im­pact of psy­che­del­ic com­pounds on cre­ativ­i­ty and well-be­ing.

But the brim­ming en­thu­si­asm comes with a healthy dose of skep­ti­cism. Crit­ics wor­ry that the bur­geon­ing 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.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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