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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Nkarta CEO Paul Hastings at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Nkar­ta un­der­scores safe­ty of CAR-NK, boasts ear­ly re­spons­es

The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses.

In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%.

Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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