5-MeO-DMT

Phase 2b clinical trial of GH001 in patients with treatment-resistant depression completed enrolment of the double-blind phase in Q3 2024Phase 1 clinical trial to evaluate proprietary aerosol delivery device in healthy volunteers is ongoing in the UKCash, cash equivalents, other financial assets and marketable securities of $193.8 million DUBLIN, Nov. 14, 2024 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today reported financial results for the third quarter ended September 30, 2024, and provided updates on its business. Business Updates GH001 in Patients with TRD GH001, our proprietary inhaled mebufotenin (5-MeO-DMT) product candidate, is currently being investigated in a multi-center, randomized, double-blind, placebo-controlled Phase 2b trial in approximately 80 patients with treatment-resistant depression (TRD) (GH001-TRD-201). GH001 is administered on a single dosing day, without mandated in-trial psychotherapeutic intervention, consistent with our previously completed trials. We completed enrolment of the double-blind phase in the third quarter of 2024, with top-line data expected to be available in the fourth quarter of 2024 or the first quarter of 2025. This trial includes a 6-month open-label extension which is on track for completion in the first quarter of 2025. GH001 Administered with Proprietary Aerosol Delivery Device Our Phase 1 clinical pharmacology trial to evaluate our proprietary aerosol delivery device for administration of GH001 in healthy volunteers (GH001-HV-106) is ongoing in the United Kingdom. This trial is designed to support our global program for GH001, by bridging to the clinical data generated with the commercially available device that we have used in our clinical trials to date. Update on IND for GH001 As previously announced, our investigational new drug application (IND) for GH001 administered using our proprietary aerosol delivery device was placed on clinical hold by the U.S. Food and Drug Administration (FDA). Based on interactions with the FDA, we believe we have a path to respond on the device element of the hold. The nonclinical studies to address the inhalation toxicology aspect are ongoing. Proof-of-Concept Trials with GH001 GH001 is being investigated in a proof-of-concept clinical trial in bipolar II disorder in patients with a current depressive episode (BDII) (GH001-BD-202). While increasing the number of sites has improved enrolment, recruitment has continued to be difficult and, for these reasons, the trial will end in the fourth quarter of 2024. GH001 is also being investigated in a proof-of-concept clinical trial in patients with postpartum depression (PPD) (GH001-PPD-203). We continue to expect GH001-PPD-203 completion in the fourth quarter of 2024. Third Quarter 2024 Financial Highlights Cash position Cash, cash equivalents, other financial assets and marketable securities were $193.8 million as of September 30, 2024, compared to cash, cash equivalents, other financial assets and marketable securities of $222.7 million as of December 31, 2023. Other financial assets are comprised of money market funds, and marketable securities are comprised of investment grade bonds. Research and development expenses R&D expenses were $8.4 million for the quarter ended September 30, 2024, compared to $7.1 million for same quarter in 2023. The increase is primarily due to increased expenses relating to our clinical development activities including clinical trials and nonclinical activities. General and administrative expenses G&A expenses were $4.2 million for the quarter ended September 30, 2024, compared to $2.6 million for the same quarter in 2023. The increase is primarily due to an increase in professional fees and employee expenses in our general and administrative functions to support our growth initiatives. Net loss Net loss was $12.1 million, or $0.23 loss per share, for the quarter ended September 30, 2024, compared to $5.6 million, or $0.11 loss per share, for the same quarter in 2023. About GH Research PLC GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. GH Research PLC's initial focus is on developing its novel and proprietary mebufotenin (5-MeO-DMT) therapies for the treatment of patients with treatment-resistant depression (TRD). GH Research PLC's annual report on Form 20-F filed with the U.S. Securities and Exchange Commission for the year ended December 31, 2023 is available at www.ghres.com and shareholders may receive a hard copy free of charge upon request. About GH001 Our lead product candidate, GH001, is formulated for mebufotenin (5-MeO-DMT) administration via a proprietary inhalation approach. With GH001, we have completed two Phase 1 healthy volunteer clinical trials and a Phase 1/2 clinical trial in patients with TRD. Based on the observed clinical activity, where 87.5% of patients with TRD achieved ultra-rapid remission with our GH001 individualized single-day dosing regimen in the Phase 2 part of the trial, we believe that GH001 has the potential to change the way TRD is treated today. About GH002 and GH003 GH002 is our mebufotenin (5-MeO-DMT) product candidate formulated for administration via a proprietary intravenous approach. We have completed a Phase 1 trial of GH002 in healthy volunteers. GH003 is our mebufotenin (5-MeO-DMT) product candidate formulated for administration via a proprietary intranasal administration approach. GH003 is currently in preclinical development. We anticipate developing GH002 and GH003 within our focus areas of psychiatric and neurological disorders. Forward-Looking Statements This press release contains statements that are, or may be deemed to be, forward-looking statements. All statements other than statements of historical fact included in this press release, including statements regarding our future results of operations and financial position, business strategy, product candidates, medical devices required to deliver these product candidates, research pipeline, ongoing and currently planned preclinical studies and clinical trials, regulatory submissions and approvals and their effects on our business strategy, including our plans and expectations related to addressing the clinical hold on the GH001 IND, research and development costs, cash runway, timing and likelihood of success, as well as plans and objectives of management for future operations, are forward-looking statements. Forward-looking statements appear in a number of places in this press release and include, but are not limited to, statements regarding our intent, belief or current expectations. Forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our management. Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including, but not limited to, those described in our filings with the U.S. Securities and Exchange Commission. No assurance can be given that such future results will be achieved. Such forward-looking statements contained in this press release speak only as of the date hereof. We expressly disclaim any obligation or undertaking to update these forward-looking statements contained in this press release to reflect any change in our expectations or any change in events, conditions, or circumstances on which such statements are based unless required to do so by applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Investor Relations: Julie RyanGH Research PLCinvestors@ghres.com GH RESEARCH PLCCondensed Consolidated Interim Statement of Comprehensive Income (Unaudited)(in thousands, except share and per share amounts) Three months endedSeptember 30, Nine months endedSeptember 30, 20242023 20242023 $’000$’000 $’000$’000 Operating expenses Research and development(8,397)(7,088) (26,810)(21,570)General and administration(4,224)(2,631) (10,558)(8,493)Loss from operations(12,621)(9,719) (37,368)(30,063) Finance income2,5352,438 7,7606,049Finance expense(181)(184) (538)(534)Movement of expected credit loss(2)(17) 451Foreign exchange (loss)/gain(1,845)1,833 (58)232Total other income5074,070 7,2095,748 Loss before tax(12,114)(5,649) (30,159)(24,315)Tax charge/(credit)-- --Loss for the period(12,114)(5,649) (30,159)(24,315) Other comprehensive income/(expense) Items that may be reclassified to profit or loss Fair value movement on marketable securities908(428) 258(1,216)Currency translation adjustment1,622(1,780) (113)(161)Total comprehensive loss for the period(9,584)(7,857) (30,014)(25,692) Attributable to owners: Loss for the period(12,114)(5,649) (30,159)(24,315)Total comprehensive loss for the period(9,584)(7,857) (30,014)(25,692) Loss per share Basic and diluted loss per share (in USD)(0.23)(0.11) (0.58)(0.47) GH RESEARCH PLCCondensed Consolidated Interim Balance Sheet (Unaudited)(in thousands) At September30,At December31, 20242023 $’000$’000ASSETS Current assets Cash and cash equivalents90,05978,420Other financial assets32,51755,615Marketable securities27,46127,525Other current assets4,9092,529Total current assets154,946164,089Non-current assets Marketable securities43,80661,142Property, plant and equipment8591,069Total non-current assets44,66562,211Total assets199,611226,300 LIABILITIES AND EQUITY Current liabilities Trade payables2,9463,490Lease liability275343Other current liabilities6,5662,868Total current liabilities9,7876,701Non-current liabilities Lease liability458631Total non-current liabilities458631Total liabilities10,2457,332 Equity attributable to owners Share capital1,3011,301Additional paid-in capital291,463291,463Other reserves4,8664,651Foreign currency translation reserve(10,620)(10,507)Accumulated deficit(97,644)(67,940)Total equity189,366218,968Total liabilities and equity199,611226,300

- The United States Food and Drug Administration cleared the investigational new drug application for VLS-01 (buccal film DMT); atai expects to initiate a Phase 2 study in treatment-resistant depression patients around YE’24 - Remain on track to initiate a Phase 2 study of EMP-01 (oral R-MDMA) in social anxiety disorder patients around YE’24 - Cash, marketable securities, and committed term loan funding expected to fund operations into 2026 NEW YORK and BERLIN, Nov. 13, 2024 (GLOBE NEWSWIRE) -- atai Life Sciences (NASDAQ: ATAI) (“atai” or “Company”), (NASDAQ: ATAI) (“atai” or “Company”), a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders, today announced third quarter 2024 financial results and provided corporate updates. “As we approach the end of 2024, we continue to see progress and momentum across our pipeline, both with our wholly owned programs and strategic investments,” stated Dr. Srinivas Rao, Co-Chief Executive Officer and Co-founder of atai. “We are on track to initiate Phase 2 trials for VLS-01 and EMP-01 around year-end and we look forward to topline Phase 2b data from Beckley Psytech’s BPL-003 in the second quarter of 2025. Our team is focused on executing these trials with the utmost scientific rigor and is driven by our goal of being the leader in developing new psychedelic treatment options to mental health patients in need of innovative, safe and effective solutions.” Recent Clinical HighlightsVLS-01: N,N-dimethyltryptamine (DMT) for Treatment-Resistant Depression (TRD) VLS-01 is a proprietary oral transmucosal film formulation of DMT applied to the buccal surface designed to fit within a two-hour in-clinic treatment paradigm.The United States Food and Drug Administration (FDA) cleared the investigational new drug (IND) application for VLS-01, allowing the Company to proceed with its plans to initiate a randomized, double-blind, placebo-controlled Phase 2 study to assess the safety, efficacy and durability of response of repeated doses of VLS-01 buccal film in patients with TRD.The Phase 2 study is expected to initiate the study in U.S. around year-end 2024. EMP-01: R-enantiomer of 3,4-methylenedioxy-methamphetamine (R-MDMA) for Social Anxiety Disorder (SAD) EMP-01 is an oral formulation of R-MDMA that demonstrated a unique, dose-dependent subjective effect profile in a Phase 1 trial that was generally found to be more similar to classical psychedelics than to racemic MDMA. atai expects to initiate an exploratory, randomized, double-blind, placebo-controlled Phase 2 study to assess the safety, tolerability and efficacy of EMP-01 in adults with SAD around year-end 2024.SAD is an area of high unmet medical need with approximately 18 million people in the U.S. diagnosed in the past year and no novel molecules approved in over two decades. IBX-210: Intravenous (IV)-Ibogaine for Opioid Use Disorder (OUD) IBX-210 is a novel IV formulation of ibogaine, which is an indole alkaloid with potential for clinical benefit for substance use disorderCompleted productive FDA pre-IND meeting to initiate discussions and alignment on a modern ibogaine IND.atai plans to run additional non-clinical studies prior to launching a Phase 1b study. Novel 5-HT2A Receptor Agonists Discovery program to identify novel, non-hallucinogenic 5-HT2AR agonists for TRD using artificial intelligence (AI)/machine learning (ML)-informed drug design and medicinal chemistry.Presented data at the Society for Neuroscience (SfN) annual meeting aimed to show that these compounds are promising chemical starting points for new analogs with further improved 5-HT2AR vs. 5-HT2BR agonist selectivity that maintain translational antidepressant-like activity with potential for non-hallucinogenic effects. RL-007: Pro-Cognitive Neuromodulator for Cognitive Impairment Associated with Schizophrenia (CIAS) RL-007 is an orally bioavailable compound that has demonstrated pro-cognitive effects in multiple pre-clinical and clinical studies, including two Phase 1 and two Phase 2 trials.The ongoing Phase 2b study is evaluating 20mg and 40mg of RL-007 vs. placebo in patients living with CIAS. Topline results are expected mid-2025. Recent Corporate UpdatesCompleted the acquisition of IntelGenx Corp. IntelGenx is a drug delivery company focused on the development and manufacturing of novel oral thin film products for the pharmaceutical market and manufactures VLS-01 (buccal film DMT).Neither equity nor cash from the Company was used to acquire IntelGenx. Anticipated Upcoming R&D Catalysts H2’24 VLS-01 TRD: Phase 2 initiation (around YE’24)EMP-01 SAD: Phase 2 initiation (around YE’24)BPL-003 alcohol use disorder (AUD): Phase 2a topline open-label dataELE-101 major depressive disorder (MDD): Phase 2a topline open-label data 2025 BPL-003 TRD: Phase 2b topline data (Q2’25)RL-007 cognitive impairment associated with schizophrenia (CIAS): Phase 2b topline data (mid’25)VLS-01 TRD: Phase 2 topline data (around YE’25)EMP-01 SAD: Phase 2 topline data (around YE’25) Consolidated Financial ResultsCash, cash equivalents, and short-term securities (primarily US treasuries and government agency securities): As of September 30, 2024, the Company had cash, cash equivalents, restricted cash and short-term securities of $101.0 million compared to $154.2 million as of December 31, 2023. The decrease of $53.2 million was primarily driven by $58.1 million net cash used in operating activities, $10.0 million for the Beckley Psytech investment, and $7.7 million investment to advance our programs; partially offset by $16.1 million in proceeds from the partial sale of our ADSs holdings in Compass Pathway, and $5.0 million in proceeds from our committed term loan with Hercules Capital, Inc. The Company expects its cash, short-term securities, public equity holdings, and committed term loan facility to be sufficient to fund operations into 2026. Research and development (R&D) expenses: R&D expenses were $12.4 million for the three months ended September 30, 2024, as compared to $13.3 million for the same prior year period. The year-over-year decrease of $0.9 million was primarily attributable to a decrease of $2.7 million in R&D personnel-related expenses, partially offset by an increase of $1.7 million in program-specific expenses. Within program-specific expenses, the increase was primarily driven by additional clinical trial expenses in the current year. The Company is anticipating R&D spend to increase as its R&D programs progress into later stage clinical trials. General and administrative (G&A) expenses: G&A expenses for the three months ended September 30, 2024, were $10.3 million as compared to $13.6 million in the same prior year period. The year-over-year decrease of $3.3 million was primarily attributable to a $3.5 million decrease in personnel-related expenses and administrative costs. The Company expects the reduction in G&A spend over prior years to continue. Net income (loss): Net loss attributable to stockholders for the three months ended September 30, 2024, was $26.3 million, which included $2.0 million of non-cash change in fair value of notes receivables and other investments and $5.0 million of non-cash share-based compensation. Net income attributable to stockholders for the three months ended September 30, 2023 was $44.2 million, which included a $69.0 million non-cash change in fair value of other investments related to an accounting change of our Compass Pathways plc investment and $8.3 million of non-cash share-based compensation. About atai Life Sciencesatai is a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders and was founded as a response to the significant unmet need and lack of innovation in the mental health treatment landscape. atai is dedicated to efficiently developing innovative therapeutics to treat depression, anxiety, addiction, and other mental health disorders. By pooling resources and best practices, atai aims to responsibly accelerate the development of new medicines to achieve clinically meaningful and sustained behavioral change in mental health patients. atai's vision is to heal mental health disorders so that everyone, everywhere can live a more fulfilled life. For more information, please visit www.atai.life. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). The words “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “anticipate,” “initiate,” “could,” “would,” “project,” “plan,” “potentially,” “preliminary,” “likely,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements include express or implied statements relating to, among other things: our business strategy and plans; the potential, success, cost and timing of development of our product candidates, including the progress of preclinical and clinical trials and related milestones; expectations regarding our strategic investment in Beckley Psytech and other investments; expectations regarding our cash runway; and the plans and objectives of management for future operations, research and development and capital expenditures. Forward-looking statements are neither promises nor guarantees, but involve known and unknown risks and uncertainties that could cause actual results to differ materially from those projected, including, without limitation, the important factors described in the section titled “Risk Factors” in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”), as such factors may be updated from time to time in atai's other filings with the SEC. atai disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by applicable law. Contact InformationInvestor Contact:IR@atai.life Media Contact:PR@atai.life -- Financial Statements Attached -- ATAI LIFE SCIENCES N.V.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(Amounts in thousands, except share and per share amounts) Three Months Ended Nine Months Ended September 30, September 30, 2024 2023 2024 2023 (unaudited) (unaudited)License revenue $40 $87 $313 $296 Operating expenses: Research and development 12,377 13,290 36,513 48,047 General and administrative 10,265 13,631 36,226 44,159 Total operating expenses 22,642 26,921 72,739 92,206 Loss from operations (22,602) (26,834) (72,426) (91,910)Other income (expense), net (3,861) 70,681 (36,795) 70,944 Net income (loss) before income taxes (26,463) 43,847 (109,221) (20,966)Benefit from (provision for) income taxes 178 (238) 163 (588)Losses from investments in equity method investees, net of tax (26) (238) (2,000) (3,199)Net income (loss) (26,311) 43,371 (111,058) (24,753)Net loss attributable to noncontrolling interests (25) (873) (747) (2,821)Net income (loss) attributable to ATAI Life Sciences N.V. stockholders $(26,286) $44,244 $(110,311) $(21,932)Net income (loss) per share attributable to ATAI Life Sciences N.V. stockholders — basic $(0.16) $0.28 $(0.69) $(0.14)Net income (loss) per share attributable to ATAI Life Sciences N.V. stockholders — diluted $(0.16) $0.25 $(0.69) $(0.14)Weighted average common shares outstanding attributable to ATAI Life Sciences N.V. stockholders — basic 160,621,817 155,792,490 159,973,201 155,793,601 Weighted average common shares outstanding attributable to ATAI Life Sciences N.V. stockholders — diluted 160,621,817 177,565,973 159,973,201 155,793,601 ATAI LIFE SCIENCES N.V.CONDENSED CONSOLIDATED BALANCE SHEET(Amounts in thousands) September 30, December 31, 2024 2023 (unaudited) (1)Assets Cash and cash equivalents $29,963 $45,034 Securities carried at fair value 55,957 109,223 Short-term restricted cash for other investments 15,000 - Committed investment funds - 25,000 Prepaid expenses and other current assets 7,454 5,830 Short-term notes receivable - related party, net 5,700 505 Property and equipment, net 865 981 Operating lease right-of-use asset, net 1,032 1,223 Other investments held at fair value 45,227 89,825 Other investments 33,893 1,838 Long-term notes receivable - related party, net - 97 Convertible notes receivable - related party - 11,202 Other assets 2,428 2,720 Total assets $197,519 $293,478 Liabilities and Stockholders' Equity Accounts payable 4,880 4,589 Accrued liabilities 11,953 15,256 Current portion of lease liability 257 275 Short-term convertible promissory notes and derivative liability - related party 925 — Short-term convertible promissory notes and derivative liability 1,481 — Other current liability 147 — Contingent consideration liability - related parties 650 620 Contingent consideration liability 1,388 1,637 Noncurrent portion of lease liability 808 990 Convertible promissory notes and derivative liability - related party — 164 Convertible promissory notes and derivative liability — 2,666 Long-term debt, net 20,336 15,047 Other liabilities 8,378 7,918 Total stockholders' equity attributable to ATAI Life Sciences N.V. stockholders 145,720 242,962 Noncontrolling interests 596 1,354 Total liabilities and stockholders' equity $197,519 $293,478 (1) The condensed consolidated financial statements as of and for the year ended December 31, 2023 are derived from the audited consolidated financial statements as of that date.