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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Anna Protopapas, Mersana CEO

In $1.36B biobuck deal with GSK, Mer­sana touts 'biggest pre­clin­i­cal ADC deal ever'

Days after Enhertu reeled in another FDA nod, with the first-ever green light for HER2-low breast cancer, another antibody drug conjugate biotech claims it has secured the largest preclinical ADC pact to date for a single asset.

AstraZeneca and Daiichi Sankyo made waves with their nearly $7 billion collaboration back in spring 2019, but at that point, Enhertu was already nearing the FDA’s doors with clinical data. The latest ADC tie-up to enter the biopharma fray centers around a preclinical asset, Mersana Therapeutics’ XMT-2056.

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

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