CHDI Foundation, Inc.

Longstanding alliance between Evotec and CHDI to enable the development of therapeutics for Huntington's disease One of Evotec's largest strategic drug discovery alliances HAMBURG, GERMANY / ACCESSWIRE / May 28, 2024 / Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809; NASDAQ: EVO) has announced the extension of its 20-year collaboration with CHDI Foundation, Inc. ("CHDI"). CHDI is a privately funded nonprofit biomedical research organisation exclusively dedicated to collaboratively developing therapeutics that will substantially improve the lives of those affected by Huntington's disease. The collaboration will further our fundamental scientific understanding of the disease and thereby aid the development of new effective treatments. The collaboration utilises Evotec's integrated neuroscience platform and broad expertise in CNS drug discovery and development. It covers a wide range of biology and chemistry services supported by compound and library management, target validation, stem-cell research, high-content screening, computational chemistry, pharmacology, protein production, and biomarker discovery and validation across multiple sites. Initiated in 2006, the long-standing collaboration is one of the largest strategic drug discovery alliances within Evotec. Dr Craig Johnstone, Chief Operating Officer of Evotec, commented: "We are delighted that CHDI recognises the value of our long and highly cooperative relationship. We are tremendously proud of our continued contribution to the fight against Huntington's disease with our colleagues at CHDI, which underscores our unwavering joint commitment to finding new therapies for people affected by this devastating condition." Dr Robert Pacifici, Chief Scientific Officer at CHDI, added: "Over the past 20 years, our colleagues at Evotec have been critical to CHDI's preclinical research programs and truly valued scientific allies in our mission to develop therapeutics for people with Huntington's disease. We look forward to continuing our longstanding and productive research collaboration into the foreseeable future." About Huntington's disease Huntington's disease is a familial disease caused by a mutation in the huntingtin gene. Each child of a parent carrying this mutation has a 50-50 chance of inheriting the condition. Due to the presence of the mutation, an individual´s brain cells undergo failure and subsequent death, resulting in cognitive and physical impairments. These challenges intensify over the course of the disease, significantly impairing the individual's quality of life and ultimately leading to death. Symptoms of Huntington's disease, which generally develop in midlife and become progressively more debilitating over time, can also emerge in infancy or old age. Once overt symptoms start, patients typically live for about 15 to 20 years. Currently, there is no way to delay the onset of symptoms or slow the progression of Huntington's disease. It is estimated that the disorder affects about 30,000 people in the United States, and at least 150,000 others have a 50% risk of developing Huntington's disease at some point. About CHDI Foundation, Inc. CHDI Foundation is a privately-funded, nonprofit biomedical research organization devoted to a single disease - Huntington's disease. Our mission is to collaboratively develop therapeutics that will substantially improve the lives of those affected by Huntington's disease. To achieve this CHDI manages a diverse portfolio of research projects through a novel virtual model that encourages scientific collaboration to more directly connect innovative research, drug discovery, and clinical development. This helps bridge the translational gap that often exists between academic and industrial research pursuits, and which adds costly delays to therapeutic development. Our activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials. To learn more, visit . About Evotec SE Evotec is a life science company with a unique business model that delivers on its mission to discover and develop highly effective therapeutics and make them available to the patients. The Company's multimodality platform comprises a unique combination of innovative technologies, data and science for the discovery, development, and production of first-in-class and best-in-class pharmaceutical products. Evotec provides high value pipeline co-creating partnerships and solutions to all Top 20 Pharma and over 800 biotechnology companies, academic institutions, as well as other healthcare stakeholders. Evotec has strategic activities in a broad range of currently underserved therapeutic areas, including e.g. neurology, oncology, as well as metabolic and infectious diseases. Within these areas of expertise, Evotec aims to create the world-leading co-owned pipeline for innovative therapeutics and has to-date established a portfolio of more than 200 proprietary and co-owned R&D projects from early discovery to clinical development. Evotec operates globally with more than 5,000 highly qualified people. The Company's 17 sites offer highly synergistic technologies and services and operate as complementary clusters of excellence. For additional information please go to and follow us on X/Twitter @Evotec and LinkedIn . Forward-looking-statements This announcement contains forward-looking statements concerning future events, including the proposed offering and listing of Evotec's securities. Words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "should," "target," "would" and variations of such words and similar expressions are intended to identify forward-looking statements. Such statements include comments regarding Evotec's expectations for revenues, Group EBITDA and unpartnered R&D expenses. These forward-looking statements are based on the information available to, and the expectations and assumptions deemed reasonable by Evotec at the time these statements were made. No assurance can be given that such expectations will prove to have been correct. These statements involve known and unknown risks and are based upon a number of assumptions and estimates, which are inherently subject to significant uncertainties and contingencies, many of which are beyond the control of Evotec. Evotec expressly disclaims any obligations or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Evotec's expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based. Media Gabriele Hansen SVP Head of Global Corporate Communications Gabriele.Hansen@evotec.com Hinnerk Rohwedder Director of Global Corporate Communications Hinnerk.Rohwedder@evotec.com Investor Relations Volker Braun EVP Head of Global Investor Relations & ESG Volker.Braun@evotec.com SOURCE: Evotec SE View the original press release on accesswire.com

A newly developed nanomaterial that mimics the behavior of proteins could be an effective tool for treating Alzheimer's and other neurodegenerative diseases. The nanomaterial alters the interaction between two key proteins in brain cells -- with a potentially powerful therapeutic effect. A newly developed nanomaterial that mimics the behavior of proteins could be an effective tool for treating Alzheimer's and other neurodegenerative diseases. The nanomaterial alters the interaction between two key proteins in brain cells -- with a potentially powerful therapeutic effect. The innovative findings, recently published in the journal Advanced Materials, were made possible thanks to a collaboration between University of Wisconsin-Madison scientists and nanomaterial engineers at Northwestern University. The work centers around altering the interaction between two proteins that are believed to be involved in setting the stage for diseases like Alzheimer's, Parkinson's and amyotrophic lateral sclerosis, or ALS. The first protein is called Nrf2, a specific type of protein called a transcription factor that turns genes on and off within cells. One of Nrf2's important functions is its antioxidant effect. While different neurodegenerative diseases result from separate disease processes, a commonality among them is the toxic effect of oxidative stress on neurons and other nerve cells. Nrf2 combats this toxic stress in brain cells, helping to stave off disease. Jeffrey Johnson, a professor in the UW-Madison School of Pharmacy, has been studying Nrf2 as a promising target for treating neurodegenerative diseases for decades alongside his wife Delinda Johnson, a senior scientist at the pharmacy school. In 2022, the Johnsons and another group of collaborators found that increasing Nrf2 activity in a specific cell type in the brain, the astrocyte, helped protect neurons in mouse models of Alzheimer's disease, leading to significantly less memory loss. While this previous research suggested that increasing Nrf2's activity could form the basis of an Alzheimer's treatment, scientists have found it challenging to effectively target the protein within the brain. "It's hard to get drugs into the brain, but it's also been very hard to find drugs that activate Nrf2 without a lot of off-target effects," says Jeffrey Johnson. Enter the new nanomaterial. Known as a protein-like polymer, or PLP, the synthetic material is designed to bind to proteins as if it were itself a protein. This nano-scale imitator is a product of a team led by Nathan Gianneschi, a professor of chemistry at Northwestern and faculty member at the university's International Institute for Nanotechnology. Gianneschi has designed multiple PLPs to target various proteins. This particular PLP is structured to alter the interaction between Nrf2 and another protein called Keap1. The proteins' interaction, or pathway, is a well-known target for treating many conditions because Keap1 essentially controls when Nrf2 responds to -- and combats -- oxidative stress. Bound together under unstressed conditions, Keap1 releases Nrf2 to do its antioxidant work when needed. Gianneschi and the Johnsons were connected via Robert Pacifici, chief science officer at the CHDI Foundation, which funds research aimed at treating Huntington's disease, another neurodegenerative condition. The foundation has funded both the Johnsons' and Gianneschi's work in the past. "Just in passing, Nathan and his colleagues at Grove Biopharma, a preclinical biotech startup focused on therapeutic targeting of protein-protein interactions, said to Robert that they were thinking about moving to target Nrf2," says Johnson. "And Robert said, 'If you're going to do that, you should call Jeff Johnson.'" Soon, the Johnsons and Gianneschi were discussing the possibility of the UW-Madison lab providing mouse model brain cells needed to test Gianneschi's protein-like nanomaterial. Jeffrey Johnson says he was initially somewhat skeptical about the PLP approach, given his unfamiliarity with it and the general difficulty of precisely targeting proteins in brain cells. "But then one of Nathan's students came up here with it and put it on our cells, and I'll be damned if it didn't work really well," he says. "We really dove into it then." The resulting research showed that Gianneschi's PLP was very effective at binding to Keap1, which freed up Nrf2 to accumulate in cells' nuclei, amping up its antioxidant function. Importantly, it did so without causing the unwanted off-target effects that have hampered other strategies aimed at better activating Nrf2. While that work was performed in cells in culture, the Johnsons and Gianneschi are now taking it a step further in mouse models of neurodegenerative diseases. It's a line of research that they hadn't expected to be involved in but are now excited to be pursuing. "We don't have the expertise in biomaterials," says Delinda Johnson. "So getting that from Northwestern and then moving forward on the biological side here at UW shows that these types of collaborations are really important."

VO659 is the only clinical-stage candidate targeting the CAG repeat expansion that causes these diseases VO659 was designed to preferentially reduce mutant HTT and spare wild-type HTT Phase 1/2a basket trial design and preclinical VO659 data to be presented at CHDI Foundation's 18th Annual Huntington's Disease Therapeutics Conference LEIDEN, Netherlands, April 3, 2023 /PRNewswire/ -- Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced that the first patient has been dosed in a Phase 1/2a clinical study evaluating VO659 for the treatment of Huntington's disease (HD), spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). VO659 is an antisense oligonucleotide (ASO) investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including HD, SCA1 and SCA3. "We are encouraged by the continued progress of our development program and very pleased to announce the first patient dosed in this Phase 1/2a study of VO659 in HD, SCA1 and SCA3," said Scott Schobel, MD, chief medical officer at Vico. "VO659 is the first allele-preferential ASO in clinical development with broad application to all CAG repeat expansion diseases. The robust preclinical data package for VO659 demonstrates favorable brain uptake, potency and durability of effect, and we look forward to assessing the translation of these characteristics in this clinical trial." VO659's unique target and dual mechanism of action enable potent target engagement across multiple disease models. In preclinical studies, significant and dose-dependent reductions of mutant huntingtin protein (mHTT) and improvement in motor function were observed in vivo in disease mouse models of HD. Allele-preferential reductions of mHTT were observed in vitro in HD patient cell models. Preclinical data also showed VO659 induced a significant reduction of mutant ATXN1 (mATXN1) and mutant ATXN3 (mATXN3) protein levels in vivo and in vitro in disease mouse models and patient cell models of SCA1 and SCA3, respectively. The Phase 1/2a clinical trial is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. Exploratory endpoints include the assessment of pharmacodynamic biomarkers (mHTT, total HTT, mATXN3, total ATXN3 and neurofilament light chain) in cerebrospinal fluid, plasma pharmacokinetics, and clinical outcome measures. The study is expected to enroll approximately 71 participants. "It is our mission to bring safe, effective therapies to patients and families affected by devastating neurological diseases, and today marks an important milestone in our journey to realize this mission. We are grateful to the trial investigators and patients participating in our Phase 1/2a study as we assess the potential of VO659 in HD, SCA1 and SCA3. We also look forward to sharing more details about the study at CHDI's Annual Huntington's Disease Therapeutics Conference later this month," said Micah Mackison, chief executive officer at Vico. The company also announced that researchers will be highlighting preclinical data of VO659 in an oral presentation along with poster presentations of the Phase 1/2a trial design and additional preclinical data of VO659 at the CHDI Foundation's 18th Annual Huntington's Disease Therapeutics Conference being held in Dubrovnik, Croatia from April 24-27, 2023. Details of Vico's presentations at CHDI's Annual Conference are as follows: Oral Presentation Title: VO659, an allele-preferential CAG repeat-targeting ASO with therapeutic potential for patients with Huntington's disease and spinocerebellar ataxia types 1 and 3 Presenter: Nicole Datson, PhD, Chief Scientific Officer, Vico Session: Session III - Novel mechanisms, modalities, and candidates for HD therapeutics Date and Time: Wednesday, April 26 at 9:00 am CET Poster Presentations Title: VO659, a CAG repeat-targeting ASO, blocks translation of polyQ proteins in a CAG repeat length dependent and allele preferential manner Presenter: Ruurd Verheul, Scientist, Vico Date and Time: Monday, April 24 – Thursday, April 27 Title: Design of a first-in-human, phase 1/2a trial of VO659, a CAG repeat-targeting ASO in patients with Huntington's disease and spinocerebellar ataxia types 1 and 3 Presenter: Katja Obieglo, PhD, Clinical Scientist, Vico Date and Time: Monday, April 24 – Thursday, April 27 About Vico Therapeutics B.V. Vico Therapeutics is a clinical-stage genetic medicines company developing antisense oligonucleotide (ASO) RNA modulating therapies for patients with severe neurological diseases. Vico's platform designs ASOs to modulate RNA through multiple approaches including inhibition of translation, modulation of splicing, editing, degradation, and activation. We apply precision chemistry to identify an RNA modulator that achieves the desired target engagement and safety profile for different diseases. Our lead product candidate, VO659, is currently in Phase 1/2a clinical development for the treatment of Huntington's disease and spinocerebellar ataxia types 1 and 3. For more information, visit . Media Contact Jenna Urban Berry & Company Public Relations [email protected] 212-253-8881 SOURCE Vico Therapeutics B.V.