Bruxism

Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 05, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and correcting mutations of interest. “The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome. This collaboration marks an important step in accelerating the development of Axiomer in CNS, one of our initial areas of strategic focus along with liver-originated diseases.” “ProQR’s leading RNA editing technology platform, coupled with their deep expertise in developing oligonucleotide therapies will offer an exciting pathway to develop new therapies for Rett Syndrome. RNA approaches can offer several advantages, including, for example, that because of their small size they do not require viral delivery,” said Bob Deans, RSRT’s Chief Technology Officer and Head of Research. “Building on work RSRT has been funding in the laboratory of Dr. Peter Beal at UC Davis, we are excited to now also work closely with ProQR to advance RNA editing as a potential therapeutic approach for Rett syndrome.” RSRT awarded ProQR approximately $1 million as a research grant for the initial phase of the project, which will encompass editing oligonucleotide design and optimization, including evaluation in in vivo models for editing efficacy and MECP2 protein recovery. It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work. The co-funding of the next phase of the collaboration would enable clinical development of an Axiomer-based therapeutic for Rett syndrome MECP2. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About Rett Syndrome Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements for ProQR This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our collaboration with RSRT, including the plan of the collaboration and the intended benefits thereof, our ability to complete the initial phase of the project and expand the collaboration, our business, and the further development and advancement of our Axiomer™ platform, including our anticipated strength and our continued investment in it, as well as the potential of our technologies and product candidates. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Eli Lilly and Company; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com

EMP-01 (R-MDMA) was generally well-tolerated and treatment-related adverse events (AEs) were as expectedThe PK profile of EMP-01 was dose-proportional, and a range of exploratory pharmacodynamic (PD) measures, including subjective reports and blood-based biomarkers, showed dose-dependent changes EMP-01 administration resulted in a differentiated subjective experience compared to racemic MDMA, a result that is likely to have therapeutic implications NEW YORK and BERLIN, Jan. 02, 2024 (GLOBE NEWSWIRE) -- atai Life Sciences (NASDAQ: ATAI) (“atai”), a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders, announced positive results from its Phase 1 study evaluating orally administered EMP-01, the R-enantiomer of MDMA (3,4-methylenedioxy-methamphetamine). The goals of this Phase 1 study were to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of EMP-01. The four-cohort, single-ascending dose, randomized, double-blind, placebo-controlled study enrolled 32 healthy participants who received 75mg, 125mg, 175mg or 225mg of EMP-01 or placebo in a 6+2 design. EMP-01 was well-tolerated, and treatment-related adverse events (AEs) were all expected and generally dose dependent. There were no study discontinuations, and no serious or severe AEs were observed in the study. Non-clinically significant increases in blood pressure and heart rate were observed, though such changes showed limited dose dependency. Further, the peak body temperatures observed fell within the normal range. Finally, bruxism was observed in only 1 of 24 subjects that received EMP-01. The PK profile of EMP-01 was dose-proportional. The PD measures included both subjective reports and blood-based biomarkers. Significant, consistent and dose-dependent changes were seen on several of these exploratory PD measures. EMP-01 administration resulted in a differentiated subjective experience compared to racemic MDMA on standard psychedelic experience questionnaires. Further, dose dependent changes on measures of emotional breakthrough, a phenomenon thought to be a key mediator of the long-term psychological changes associated with psychedelics, were noted in this healthy volunteer population. Detailed clinical data from the Phase 1 study of EMP-01 are expected to be presented at a future medical meeting. “I am grateful to the participants and investigators, as well as the members of the atai study team on the successful completion of this Phase 1 study,” said Florian Brand, CEO and Co-Founder of atai. “Building upon the decades of research into MDMA as a potential treatment for mental health disorders, including two positive Phase 3 studies in PTSD, we are encouraged by the unique characteristics of EMP-01 and exploring the implications for further clinical development.” “The two enantiomers of MDMA show markedly different and rich pharmacology. The present study is one of the first specifically focused on assessing the PK and PD of R-MDMA,” said Srinivas Rao, CSO of atai. “We found differences in the subjective experience R-MDMA in comparison to published reports involving racemic MDMA. If confirmed, these differences suggest that R-MDMA may have applicability in a broad array of mental conditions.” About atai Life Sciences atai 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. The words “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “could,” “would,” “project,” “plan,” “potentially,” “preliminary,” “likely,” and the negative of these terms and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements, including without limitation our expectations and projections regarding the success, potential uses and timing of development of EMP-01 and related trials and studies, and our business strategy and plans. Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: our limited operating history, historical losses, and anticipation that we will continue to incur significant losses for the foreseeable future; we will require substantial additional funding to achieve our business goals, including the development and any commercialization of our product candidates; we have never generated revenue and may never be profitable; our product candidates contain controlled substances, the use of which may generate public controversy; clinical and preclinical development is uncertain, and our programs may experience delays or may never advance to clinical trials; our reliance on third parties to assist in conducting our clinical trials and impact to such trials based on factors including failure by third parties to meet trial or testing deadlines; our reliance on qualified therapists working at third-party clinical trial sites to administer certain of our product candidates and failure to recruit and retain a sufficient number of therapists; the timing and outcome of regulatory review and/or approvals, which are necessary prior to commercialization; research and development of drugs targeting the central nervous system, or CNS, is particularly difficult, and it can be difficult to predict and understand why a drug has a positive effect on some patients but not others; significant competition; obtaining, maintaining and protecting our intellectual property; restricted operating activity as a result of covenants in any financing arrangements, including our loan agreement with Hercules Capital, Inc.; our aggregate tax burden based on our management and operational activity. These forward-looking statements are subject to a number of important factors that could cause actual results to differ materially from those in the forward-looking statements, including the risks, uncertainties, and assumptions described in our Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission (“SEC”) and our quarterly reports on Form 10-Q, as may be updated by other filings we file with or furnish to the SEC. Any forward-looking statements made herein speak only as of the date of this press release. Except as required by applicable law, we undertake no obligation to update any of these forward-looking statements for any reason after the date of this press release or to conform these statements to actual results or revised expectations. Contact InformationInvestor Contact:ir@atai.life Media Contact:PR@atai.life

LAUSANNE, Switzerland--(BUSINESS WIRE)-- Aesyra, a medical device company pioneering oral appliance technologies to improve sleep, today announced it has raised $3 million in seed financing. The round was led by Supermoon Capital (USA), the world’s first fund focused on sleeptech, and deep tech investor Creadd Ventures (Switzerland) with participation from existing investor Zürcher Kantonalbank. The new financing will support the development and commercialization of Aesyra’s AesyBite® line of products for accurate monitoring and treatment of sleep bruxism and sleep apnea. Sleep bruxism is a sleep disorder often linked to sleep apnea, which affects nearly a billion people worldwide. A spin-off of the Federal Institute of Technology of Lausanne (EPFL) in Switzerland, Aesyra has pioneered a powerful technology system — that combines a unique oral appliance device, proprietary sensors, and AI — that has demonstrated over 70% reduction of bruxism activity for users. Powering Aesyra’s AesyBite™ are technology developed by EPFL researchers and algorithms that leverage over 10 years of sleep research data. "This significant investment marks a pivotal moment for Aesyra,” said Marco Letizia, founder and CEO of Aesyra. “We are thrilled to partner with Supermoon Capital and Creadd Ventures, who share our vision of driving meaningful change within the sleep industry. With their support, we are well-positioned to accelerate the deployment of our groundbreaking solutions, ultimately improving patient outcomes and advancing healthcare practices." "Aesyra’s advanced technology is a tremendous breakthrough in the field of sleep science for the treatment of sleep bruxism and sleep apnea,” said Michael Masterson, Managing Partner at Supermoon Capital “We are impressed by the company’s unique approach, strong leadership team and unwavering commitment to revolutionize healthcare, and we are looking forward to working closely with Aesyra to support its mission.” "We selected Aesyra as the first investment of Creadd Ventures since it perfectly fits our philosophy to invest in companies that have the potential to make a profound impact on the quality of life of patients,” said Lorenzo Leoni, Founder and Managing Partner at Creadd Ventures. Masterson Leoni will join the Board of Directors of Aesyra, bringing their extensive industry expertise and strategic guidance to accelerate the company's growth and success. About Aesyra: Aesyra is a privately held, clinical-stage company dedicated to develop, manufacture and commercialize smart medical devices able to accurately monitor and relieve sleep bruxism and sleep apnea. The company has developed a line of devices (AesyBite®) using innovative and proprietary sensors and algorithms that allows intraoral measurements of several factors during sleep and is capable to actively reduce bruxism and improve patients’ quality of life. For more information, please visit . About Supermoon Capital: Supermoon Capital is the only venture capital firm in the world focused exclusively on the sleep technology market. The San Francisco-based firm partners with visionary innovators across the sleep market spectrum encompassing chronobiology, neurotech, biotech, pharmaceuticals, medtech, consumer interventions, and enterprise solutions. Supermoon and its global network of prominent academics, strategic partners, and renowned sleep scientists support companies across a range of functions, including product development, go-to-market strategy, and growth financing. For more information, please visit . About Creadd Ventures: Creadd Ventures is a new venture capital fund with a focus on investing Biomedical, DeepTech and SAAS companies based in Switzerland and Europe. The fund’s investment strategy involves targeting emerging early-stage companies with strong management teams, a solid and proprietary scientific foundation, and a clear go to market strategy. Creadd leverages the extensive experience and network of both founding partners as entrepreneurs and fund managers. For more information, please visit . About Zürcher Kantonalbank Zürcher Kantonalbank is an independent public-law institution of the Canton of Zurich, incorporated under the laws of the Canton of Zürich. Its registered corporate domicile and principal place of business is in Zurich, Switzerland. It has received top ratings from the rating agencies Standard & Poor’s, Moody’s and Fitch (AAA/Aaa). With more than 6,000 employees across the group, the bank’s core activities include financing businesses, asset and wealth management, trading, capital market transactions, deposits, payment transactions and card business. View source version on businesswire.com: Contacts Source: Aesyra View this news release online at: