University of Pennsylvania

STEVENAGE, United Kingdom, Sept. 05, 2024 (GLOBE NEWSWIRE) -- NRG Therapeutics, Ltd., an innovative neuroscience company targeting mitochondrial dysfunction, is pleased to announce that it has been awarded a non-dilutive grant from Target ALS Foundation. It is one of five recipients of the 2024 In Vivo Target Validation grants in collaboration with Biospective. The funding will be used to support its discovery programme for a treatment for Amyotrophic Lateral Sclerosis (ALS) (also known as Lou Gehrig’s disease or Motor Nuerone Disease). NRG Therapeutics is applying breakthrough science in the field of mitochondrial biology to develop first-in-class treatments for neurodegenerative diseases including ALS/MND. It has discovered a new class of small molecule mitochondrial permeability transition pore (mPTP) inhibitors that can block mitochondrial dysfunction. Its inhibitors are designed to penetrate the brain effectively when taken orally. Target ALS is committed to breaking down scientific research barriers to develop effective biomarkers and treatments for ALS. The In Vivo Target Validation Program aims to generate high-quality data on candidate therapeutics in animal models of ALS, provide access to in-kind funding for a resource-intensive step of drug discovery, and accelerate the progress of candidate therapeutics from preclinical testing towards clinical trials. Mitochondria, the batteries of cells, are crucial for energy production, especially in brain cells which have high energy demands. In diseases like ALS, mitochondria become defective, leading to brain cell death and disease progression. Recent findings have shown that dysfunctional TDP43 protein – found in nearly all ALS patients – disrupts mitochondrial function and undesirably activates the immune system. Announcing the awards, Target ALS said, “We are thrilled to announce the recipients of the Target ALS In Vivo Target Validation grants, focusing on proof-of-concept studies for promising therapeutics in a TDP-43 mouse model. This initiative, in collaboration with Biospective, is designed to accelerate the evaluation of potential therapeutics, bridging the gap from preclinical research to clinical trials.” Biospective has implemented a modified, slower progressing version of the TDP-43 NLS8 (or DELTA-NLS) mouse model that was originally developed at the University of Pennsylvania (Walker et al., 2015). This model mimics disease symptoms and pathology that people living with ALS experience, including cytoplasmic TDP-43 mislocalization, neurodegeneration, and motor deficits. Using this standardized model, the team at Biospective will conduct robust behavioral and molecular assessments of candidate therapeutics for these preclinical efficacy studies. Successful results in this mouse model will enable groups to move their therapeutic candidate toward the clinic. NRG Therapeutics’ co-founder and CEO, Dr Neil Miller, said, “Preliminary experiments using a mouse model of ALS which contains the human gene for TDP-43 showed that our mPTP inihibitor candidate therapeutic reduced brain cell death. However, due to the rapid disease progression in that mouse model, a full assessment of the therapeutic potential was not possible. This project aims to evaluate NRG’s small molecule inhibitor, in a modified ALS TDP-43 mouse model that progresses more slowly. This will allow for a comprehensive assessment of the molecule’s ability to protect mitochondria, reduce brain cell death, and decrease immune system activation. Additionally, the study will measure improvements in muscle function and strength. If successful, this project will provide crucial support for advancing our mPTP inhibitor into clinical trials for ALS.” Details of the Target ALS 2024 In Vivo Target Validation grant recepients can be found HERE. Media enquiries (for NRG Therapeutics)Sue Charles, Charles Consultants - +44 7968 726585 sue@charles-consultants.com About NRG Therapeutics – http://www.nrgtherapeutics.comNRG Therapeutics is a neuroscience drug discovery company building a pipeline of disease-modifying mitochondrial therapeutics to slow or halt the progression of neurodegenerative disorders such as Parkinson’s and Amyotrophic Lateral Sclerosis (ALS), also known as motor neurone disease (MND). The company’s pre-clinical pipeline of small molecule assets is based on inhibiting the mitochondrial permeability transition pore (mPTP) through a novel mechanism of action. Inhibition of the mPTP has been shown to protect neurones, reduce neuroinflammation and extend survival in pre-clinical disease models. Based at the Stevenage Bioscience Catalyst (SBC), UK, NRG Therapeutics is a private company with equity investment from Parkinson’s UK, Omega Funds and Brandon Capital. The company has also received grant funding from Innovate UK (Biomedical Catalyst Award), The Michael J. Fox Foundation and Target ALS, and is the industrial partner for a FightMND Drug Discovery grant awarded to WEHI (Walter and Eliza Hall Institute of Medical Research). Follow us on Twitter and LinkedIn.

PARIS, Sept. 04, 2024 (GLOBE NEWSWIRE) -- PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology, is pleased to announce that it has established a scientific and clinical advisory board (SAB) of internationally recognized ophthalmology experts. The SAB members, from Europe, the USA and Australia, bring decades of clinical and scientific experience in both fundamental and clinical research in retinal and macular diseases. Prof Frank G. Holz, Professor and Chair of the Department of Ophthalmology, University of Bonn, Germany. Prof Holz will chair the SAB.Prof Francine Behar-Cohen, MD, PhD, Professor of Ophthalmology at Cochin Hospital, Paris, Director of research at INSERM, and Professor at the University of Paris Cité, France. Prof Behar-Cohen discovered and developed the technology; she is also a member of the Board, as an observer.Dr Joshua Dunaief MD PhD, Adele Niessen, Professor of Ophthalmology at The Perelman School of Medicine at the University of Pennsylvania, PA.Prof Robyn Guymer AM, Deputy Director, Head of Macular Research at CERA (Centre for Eye Research Australia), Melbourne, Australia, Professor of Ophthalmology at Melbourne University and senior retinal specialist at the Royal Victorian Eye and Ear Hospital.Prof Eleonora Lad, MD PhD Vice Chair, Associate Professor of Ophthalmology and Vice Chair, Clinical Research at the Duke Eye Center, NC, USA PulseSight’s lead program PST-611 in GA is ready to enter the clinic by the end of 2024, whilst its second program, PST-809 in wet AMD is at the very late stage of preclinical IND-enabling studies. Judith Greciet, CEO of PulseSight Therapeutics, said, “I am delighted to welcome these world-class seasoned experts as founding members of our SAB. Their depth and breadth of clinical and scientific expertise will bring invaluable contribution to our development plans, as we navigate early clinical development. Their enthusiastic agreement to join the SAB underlines the interest in our innovative programs and reinforces our confidence that PST-611, followed by PST-809, have the potential to significantly change the prognostic of these highly disabling, difficult to treat diseases.” Chairman of PulseSight Therapeutics, Dirk Sauer, added, “Having been in the ophthalmology field for more than two decades, I appreciate the challenges ahead of us as well as the potential of our novel non-viral approach. I would like to deeply thank the members of the SAB for their time and commitment to supporting us to unlock the full potential of our disruptive and innovative therapy platform.” Full details on the SAB members can be found on the PulseSight website – here https://pulsesight.com/#team About age-related macular degeneration (AMD)AMD is a disease with progressive loss of vision with a strong burden on patients’ everyday life, impacting their ability to read, recognize faces, and see objects and, ultimately, leading to irreversible vision loss in the elderly. After reaching an intermediate stage, AMD can progress to either ‘Wet AMD’ or ‘Dry AMD’, which can then evolve into GA (geographic atrophy), leading to irreversible blindness. In all its forms, AMD represents a compelling unmet need for more effective and durable treatment options, with a large and growing market, estimated to reach $27.5 Billion by 2031. About PulseSight Therapeutics PulseSight is clinical-stage biotech company committed to developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology to address severe eye diseases. Company’s current candidates focus on age-related macular degeneration (AMD) including wet AMD and geographic atrophy (GA) secondary to dry AMD. Already clinically validated for its safety and sustained activity, PulseSight’s technology platform delivers DNA plasmids encoding therapeutic proteins into the ciliary muscle using a user-friendly, injection like procedure based on electro-transfection. The ciliary muscle cells act as biofactories, expressing therapeutic proteins that reach the retina with high distribution, providing a safe and long-lasting treatment for major eye diseases. About PST-611 for GA PST-611 encodes the human transferrin protein, a crucial regulator of iron homeostasis and holds the potential to effectively address key pathological mechanisms in GA, whilst requiring re-treatment only every four to six months. This program is ready to enter the clinic by the end of 2024. About PST-809 for Wet AMD PST-809 is a dual-gene plasmid encoding for anti-VEGF aflibercept and decorin, an anti-angiogenic and anti-fibrotic native protein, showing superior efficacy against anti-VEGF gold standard while limiting the need for frequent reinjection. PST-809 is at the very late stage of preclinical IND-enabling studies. Based in Paris, France the company’s investors are Pureos Bioventures, ND Capital and Korea Investment Partners (KIP). For more information visit www.PulseSightTherapeutics.com Follow us on LinkedIn – https://www.linkedin.com/company/pulsesight-therapeutics/ Media contact Sue Charles, Charles Consultants T: +44 (0)7968 726585 E: sue@charles-consultants.com

REC-994 also demonstrates encouraging trends in objective MRI-based exploratory efficacy measures at the highest dose and the company plans to advance development of REC-994 for the potential treatment of symptomatic CCM in subsequent studies.SALT LAKE CITY, Sept. 03, 2024 (GLOBE NEWSWIRE) -- Recursion (NASDAQ: RXRX), a leading clinical stage TechBio company decoding biology to radically improve lives, today announced top-line results of the SYCAMORE trial, a 12-month Phase 2 randomized double-blind, placebo-controlled, safety, tolerability and exploratory efficacy study for REC-994 in symptomatic CCM patients. REC-994 met its primary endpoint of safety and tolerability, demonstrating a similar profile across placebo and both 200mg and 400mg dosage-arms with regard to the frequency and severity of adverse events after 12 months of treatment. Magnetic resonance imaging-based secondary efficacy endpoints showed a trend towards reduced lesion volume and hemosiderin ring size in patients at the highest dose (400mg) as compared to placebo. Time-dependent improvement in these trends at the 400mg dose was also observed in this signal-finding study. Improvements in either patient or physician-reported outcomes were not yet seen at the 12 month time point. A meeting with the FDA is anticipated as soon as practical to discuss plans for an additional clinical study. Recursion plans to present data from this trial at a forthcoming medical conference and intends to submit these data for publication in a peer reviewed scientific journal. "These studies are making significant strides in the development of therapeutics for CCM. The data from this readout is an impressive start and will provide a valuable contribution to the existing CCM literature and strongly supports the need for a future study, with a longer duration and a larger patient cohort,” said Dr. Jan-Karl Burkhardt, MD, Division Head, Cerebrovascular Surgery, University of Pennsylvania and Principal Investigator of the study. Connie Lee, Psy.D., founder and CEO of the Alliance to Cure Cavernous Malformation added: “I speak for the patients who have participated in the trial and those who have been cheering from the sidelines while waiting for news. This promising start is a critical step forward and will bring hope to thousands of families who currently have no options but brain or spinal cord surgery. The Alliance to Cure Cavernous Malformation looks forward to partnering with Recursion as they move to the next stage of the REC-994 program.” “We are encouraged by the recent data from our signal-finding Phase 2 study in CCM, where the trial successfully met its primary safety endpoint and became the first investigational therapy to demonstrate safety alongside some promising trends in exploratory efficacy endpoints. These results provide critical insights that will inform our next study design, including exploring study duration, higher doses, and a larger cohort of patients," said Najat Khan, Ph.D., Chief R&D Officer and Chief Commercial Officer of Recursion. "This is the first of several key clinical readouts for the company and represents an early proof-of-platform milestone for our constantly evolving Recursion OS, as we build upon our success in drug discovery with expertise and execution in mid-phase development. We are deeply grateful to the patients and investigators, and we are committed to advancing potential transformational therapies for CCM and beyond.” Background on Cerebral Cavernous Malformation (CCM) CCM is a neurovascular condition that impacts approximately 360,000 symptomatic individuals in the US and EU5. The disease is often underdiagnosed and potentially affects over 1 million patients worldwide. CCM manifests as vascular malformations of the spinal cord and brain characterized by abnormally enlarged capillary cavities without intervening brain parenchyma. Patients with CCM lesions are at substantial risk for seizures, headaches, progressive neurological deficits, and potentially fatal hemorrhagic stroke. Currently, only non-pharmacologic treatments including microsurgical resection and stereotactic radiosurgery are available options for this high unmet need patient population. However, surgical resection or stereotactic radiosurgery is not always feasible because of location and may not be curative. About REC-994 REC-994 is an orally bioavailable, superoxide scavenger small molecule under development for the treatment of symptomatic CCM. The potential of REC-994 in CCM was demonstrated using the earliest version of what would become the foundational technology underlying the Recursion OS. Subsequently, REC-994 demonstrated preclinical activity in models for CCM and tolerability and suitability for chronic dosing in Phase 1 single ascending dose escalation (SAD) and multiple ascending dose escalation (MAD) trials in healthy volunteers directed and executed by Recursion. Recursion has sought and received Orphan Drug Designation for REC-994 in symptomatic CCM in the US and Europe. About the Trial Our Phase 2 SYCAMORE clinical trial is a randomized, double-blind, placebo-controlled study of two doses of REC-994 in participants with CCM. The primary endpoint of the study is safety and tolerability. Secondary efficacy endpoints include MRI-based endpoints, clinician and patient reported outcomes, as well as selected biomarkers. This trial was fully enrolled in June 2023 with 62 participants, and 80% of participants who completed 12 months of treatment have entered the long-term extension study. This signal-finding study was not powered to demonstrate statistical significance. About Recursion Recursion (NASDAQ: RXRX) is a clinical stage TechBio company leading the space by decoding biology to radically improve lives. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously generate one of the world’s largest proprietary biological and chemical datasets. Recursion leverages sophisticated machine-learning algorithms to distill from its dataset a collection of trillions of searchable relationships across biology and chemistry unconstrained by human bias. By commanding massive experimental scale — up to millions of wet lab experiments weekly — and massive computational scale — owning and operating one of the most powerful supercomputers in the world, Recursion is uniting technology, biology and chemistry to advance the future of medicine. Recursion is headquartered in Salt Lake City, where it is a founding member of BioHive, the Utah life sciences industry collective. Recursion also has offices in Toronto, Montréal, London, and the San Francisco Bay Area. Learn more at www.Recursion.com, or connect on X (formerly Twitter) and LinkedIn. Media ContactMedia@Recursion.com Investor ContactInvestor@Recursion.com Forward-Looking Statements This document contains information that includes or is based upon "forward-looking statements" within the meaning of the Securities Litigation Reform Act of 1995, including, without limitation, those regarding Recursion’s anticipated meeting with the FDA; Recursion’s plans to present SYCAMORE trial data at a medical conference and submit the data for publication; the clinical relevance of the SYCAMORE trial data and obtaining additional confirmatory data; promising trends in REC-994 efficacy endpoints; advancing potential transformational therapies for CCM and beyond; subsequent REC-994 studies and their results and advancing Recursion’s REC-994 program further; the size of the potential CCM patient population; Recursion OS and other technologies potential and advancement of the future of medicine; business and financial plans and performance; and all other statements that are not historical facts. Forward-looking statements may or may not include identifying words such as “plan,” “will,” “expect,” “anticipate,” “intend,” “believe,” “potential,” “continue,” and similar terms. These statements are subject to known or unknown risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements, including but not limited to: challenges inherent in pharmaceutical research and development, including the timing and results of preclinical and clinical programs, where the risk of failure is high and failure can occur at any stage prior to or after regulatory approval due to lack of sufficient efficacy, safety considerations, or other factors; our ability to leverage and enhance our drug discovery platform; our ability to obtain financing for development activities and other corporate purposes; the success of our collaboration activities; our ability to obtain regulatory approval of, and ultimately commercialize, drug candidates; our ability to obtain, maintain, and enforce intellectual property protections; cyberattacks or other disruptions to our technology systems; our ability to attract, motivate, and retain key employees and manage our growth; inflation and other macroeconomic issues; and other risks and uncertainties such as those described under the heading “Risk Factors” in our filings with the U.S. Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, our subsequent Quarterly Reports on Form 10-Q, and our Current Reports on Form 8-K. All forward-looking statements are based on management’s current estimates, projections, and assumptions, and Recursion undertakes no obligation to correct or update any such statements, whether as a result of new information, future developments, or otherwise, except to the extent required by applicable law.