Toad ven­om to treat de­pres­sion? RA Cap­i­tal wa­gers $125M on lat­est psy­che­del­ic biotech

Can an in­haled ver­sion of the psy­che­del­ic known as ‘toad ven­om’ be used to treat tough-to-crack de­pres­sion? For GH Re­search, that’s the $125 mil­lion ques­tion.

The Dublin-based biotech an­nounced Mon­day it closed a Se­ries B round to help ad­vance its lead pro­gram, an in­halant called GH001, for psy­chi­atric and neu­ro­log­i­cal dis­or­ders. GH is be­gin­ning with treat­ment-re­sis­tant de­pres­sion and has two as-yet-undis­closed in­di­ca­tions on tap.

Mon­day’s round was co-led RA Cap­i­tal and RTW In­vest­ments, along with ex­ist­ing in­vestor BVF Part­ners. GH de­clined to com­ment be­yond its press re­lease.

The drug in ques­tion is a sci­en­tif­ic mouth­ful — 5-Methoxy-N, N-di­methyl­trypt­a­mine is the of­fi­cial name — but is more com­mon­ly re­ferred to as 5-MeO-DMT or slangi­ly as toad ven­om, due to its pres­ence in a cer­tain toad species na­tive to the south­west­ern US and north­west­ern Mex­i­co. It can al­so be de­rived from plants or made syn­thet­i­cal­ly, with the plant ver­sion used as an en­theogen in some parts of Cen­tral and South Amer­i­ca.

Per a 2018 VICE News re­port, the psy­che­del­ic saw a rapid rise in pop­u­lar­i­ty in the mid-2010s among those try­ing to achieve the ‘ego death’ phe­nom­e­non, in which re­searchers be­lieve the part of the brain re­spon­si­ble for one’s sense of self shuts down tem­porar­i­ly. The drug saw heavy in­ter­est from users on Red­dit and YouTube, VICE re­port­ed, who en­gaged in an “arms race” to boast about their ex­pe­ri­ences get­ting high.

Psy­che­delics-fo­cused biotechs have seen a surge in in­vestor in­ter­est in re­cent years, most no­tably sur­round­ing the Pe­ter Thiel-backed ATAI Life Sci­ences. That com­pa­ny us­es a unique busi­ness mod­el for de­vel­op­ing ther­a­peu­tics cov­er­ing a range of men­tal health dis­or­ders by bring­ing port­fo­lio com­pa­nies un­der one um­brel­la.

Though ATAI has more than 13 pro­grams de­vel­op­ing drugs, GH is much more nar­row­ly fo­cused on its 5-MeO-DMT work. So far, GH001 has com­plet­ed a Phase I study in healthy vol­un­teers, the com­pa­ny said. Re­sults showed GH001 was well-tol­er­at­ed, and it’s now in a Phase I/II tri­al in pa­tients with treat­ment-re­sis­tant de­pres­sion.

The biotech is al­so work­ing on an in­jectable for­mu­la­tion of 5-MeO-DMT, which they are call­ing GH002 and for which they’re al­so keep­ing po­ten­tial in­di­ca­tions close to the vest.

Treat­ment-re­sis­tant de­pres­sion oc­curs in pa­tients who see lit­tle to no suc­cess with an­ti­de­pres­sants or psy­chother­a­py, ac­cord­ing to the Mayo Clin­ic. Symp­toms can range from mild to se­vere and there’s gen­er­al­ly not a sin­gle ap­proach that works for every pa­tient.

In ad­di­tion to RA Cap­i­tal and RTW In­vest­ments, oth­er new in­vestors in­clud­ed Acu­ta Cap­i­tal Part­ners, Box­er Cap­i­tal, Cor­morant As­set Man­age­ment, Deer­field Man­age­ment Com­pa­ny, Lo­gos Cap­i­tal, Sur­vey­or Cap­i­tal, Ven­rock Health­care Cap­i­tal Part­ners and Veri­tion Fund Man­age­ment.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

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By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

The IPO 4-1-1: Four fil­ings, a pric­ing and a with­draw­al head­line this week's Nas­daq ac­tion as raise ap­proach­es $7.5B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another week, another horde of biotechs is doing the Nasdaq dance.

This week saw four companies file their SEC paperwork ahead of expected debuts, another hit Nasdaq on Friday and a sixth formally withdrew its bid to go public. Aerovate Therapeutics, Ocean Biomedical and Acumen Pharmaceuticals all penciled in initial raises of $100 million, while Dermata Therapeutics is estimating a modest $18 million raise.

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Keiichi Fukuda, Heartseed CEO

Fresh off $598M deal with No­vo Nordisk, a Japan­ese stem cell com­pa­ny is on its way to the clin­ic with a dif­fer­ent ap­proach to treat­ing heart fail­ure

A common approach to treating heart failure with induced pluripotent stem cells involves grafting sheets of cells onto the surface of the heart to improve vascularization and blood flow. It’s the easiest method of transplantation — but you run the risk of not making an electrical connection with the heart and the cells not synchronizing with the patient’s heart muscle.

So what if you could inject spherical clusters of heart cells directly into the heart muscle wall? For Heartseed, that’s now the $37 million question.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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