Neurofibromatosis 2

Hosted by the Children's Tumor Foundation, NF Summit and NF Conference are the largest gathering of the NF community in the world NEW YORK, June 22, 2023 /PRNewswire/ -- From June 21 through June 27, the largest gathering of NF researchers, clinicians, and patients in the world will take place at the Fairmont Scottsdale Princess in Scottsdale, Arizona. NF is a group of genetic disorders that causes tumors to grow on nerves throughout the body, and affects 1 in 2,000 births of all populations equally. As such, NF affects millions worldwide, but is underrecognized. While there is one approved treatment for a small subgroup of NF patients with plexiform neurofibromas, there is no cure yet, and the vast majority of NF patients face serious health issues because of the condition. This gathering in Arizona, comprising two meetings, offers novel insights into NF research, generates enthusiasm that effective therapeutics are on the horizon, and fosters a deeper sense of community. - NF Summit: patients, families, and caregivers will convene for three and a half days (June 21-24) to hear about promising research, meet with world-class NF experts, and participate in patient empowerment sessions. All forms of NF will be covered – neurofibromatosis type 1 (NF1) and all forms of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2), as well as quality of life issues for the myriad manifestations of NF, which can cause deafness, blindness, bone abnormalities, learning disabilities, disabling pain, and cancer. Notable sessions at the NF Summit include: Clinical trials in nutraceuticals that are leading to less toxic treatment and prevention strategies through a specific dietary component. Panel discussion on alternative & traditional approaches to chronic pain Seminar for parents and other caregivers about navigating chronic illness; this session will explore the emotional and psychological impact of NF on parents, siblings, and other family members, as well as the practical challenges that arise when a family member has a chronic illness. The NF Clinical Trials Consortium session will review the structure, discuss trials completed or ongoing, and plans for the future of the Department of Defense-funded program. - NF Conference: this four-day global meeting (June 24-27) attracts leading specialists from across a wide range of scientific disciplines and research and clinical backgrounds, all focused on improving outcomes for NF patients. This annual event is the most important date on the NF research calendar, and is critical to consensus building and advancing basic, translational, and clinical research in NF and related fields. Among the key advances to be presented at the NF Conference are: dedicated afternoon to gene therapy in NF with a targeted focus on developing curative therapies that address the underlying genetic abnormalities in NF patients focus on pain, from basic science to preclinical and translational testing & development to novel imaging biomarkers to clinical management a full day of programming dedicated to all types of schwannomatosis, which includes NF2-related schwannomatosis (NF2), covering alternative cell models, new therapeutic approaches, and microenvironment of nerve tumors and the role of neurons in tumor growth For more information about NF and the Children's Tumor Foundation, visit . For more information about the NF Summit, visit . For more information about the NF Conference, visit . ABOUT THE CHILDREN'S TUMOR FOUNDATION: The Children's Tumor Foundation is the world's leading nonprofit dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with NF, a group of genetic conditions that causes tumors to grow on nerves throughout the body. One in every 2,000 people is born with some type of neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. NF affects all populations equally, and while there is no cure yet, the Children's Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF. For more information, please visit: ctf.org. SOURCE Children's Tumor Foundation

Recursion Pharmaceuticals has agreed to acquire two biotechnology startups developing artificial intelligence tools for drug discovery for a combined $87.5 million, the company said Monday.The Salt Lake City company will pay $40 million for Cyclica and $47.5 million for Valence, and expects to add roughly 60 employees to its payroll.We are moving programs forward, but it's not at the pace that is possible with the right tools, said Chris Gibson, Recursions CEO and co-founder. We felt like if we built it ourselves, it was going to be two or three years slower than if we found the best teams in the world and brought them in-house.Recursion takes an approach to industrial drug discovery that Gibson describes as full stack, borrowing language more common to the technology sector than to biotech. In tech circles, full stack developers work on both the front-end the parts that users see and the back-end, or the systems that make a website or app functional.Cyclica and Valence are meant to help Recursion offer a more complete range of drug discovery services. According to Gibson, Recursion started with a list of more than 100 potential acquisition targets and, over the past 18 months, winnowed that down to Cyclica and Valence.Cyclica, based in Toronto, has two products that enable it to better predict and generate hits on drug targets in the development of small molecule medicines. Valence, based in Montreal, focuses on a machine learning technique known as few-shot learning classifying data with a limited number of samples.Valence is advised by Yoshua Bengio, a noted Canadian computer scientist who also serves as a scientific adviser for Recursion.In addition to partnering with other companies, Recursion has several drug candidates of its own in clinical testing for rare cancers and other conditions including neurofibromatosis type 2, an inherited disorder where benign tumors grow along nerves. It has more than a dozen programs in discovery stages for cancers, brain disorders, inflammation and other rare diseases.We wanted to de-bottleneck the downstream efforts and accelerate our ability to turn biology and early chemistry insights into real molecules that we will be able to, hopefully, test in humans, Gibson said.AI-enabled drug discovery is a hot area in biotech, despite some skepticism from industry veterans about how large its impact might really be. Companies like Exscientia, BenevolentAI and Insitro are working to build new models of drug discovery that draw on fields like computational biology and machine learning. Other biotechs, such as Relay Therapeutics, have put machine learning at the heart of their drug development work.Recursion is among the most well-funded companies in that group. It raised more than $400 million privately before adding another $436 million in an initial public offering two years ago, one of the largest new stock offerings for a biotech since 2018. The company had $473 million in cash and equivalents at the end of March.Shares of Recursion rose by as much as 10% through early Monday afternoon on news of its acquisitions and earnings. Still, at about $5.50 apiece, those shares have lost more than two thirds of their value since Recursion's Nasdaq debut in 2021. '

First-in-Class TEAD Autopalmitoylation Inhibitor is Well Tolerated and Demonstrates Antitumor Activity in Advanced Mesothelioma and NF2-Mutant Cancers VT3989 is the First Therapeutic Targeting the Hippo Pathway to Demonstrate Clinical Activity in Patients with Cancer SAN MATEO, Calif., April 16, 2023 /PRNewswire/ -- Vivace Therapeutics, Inc., a small molecule discovery and development company developing first-in-class therapies targeting the Hippo pathway, today announced that the first clinical data for a cancer treatment targeting the Hippo pathway was presented at the American Association for Cancer Research (AACR) Annual Meeting 2023. Results from the company's Phase 1 clinical study of its first-in-class transcriptional enhanced associate domain (TEAD) autopalmitoylation inhibitor, VT3989, showed the compound to be well tolerated with durable antitumor responses in patients with advanced malignant mesothelioma and other tumors with neurofibromatosis 2 (NF2) mutations. These findings were presented by Timothy A. Yap, M.D., Ph.D., of The University of Texas MD Anderson Cancer Center, during an oral plenary session at the conference. The AACR conference is being held April 14-19, 2023, in Orlando, Florida. Seven (7) of 69 patients had reductions in tumor size meeting the rigorous criteria for being partial responses that persisted up to 21 months, and 34 had stable disease. Tumor response was observed in patients with mesothelioma with and without NF2 mutations and other solid tumors with NF2 mutations. Because mesothelioma patients often have NF2 mutations or loss of Merlin it was an a priori indication of interest. Six (6) of the seven (7) partial responses were in patients with mesothelioma. The other response was in a sarcoma patient with an NF2 mutation. Of the 69 patients presented, 43 had advanced malignant mesothelioma and 26 had other solid tumors. Patients were heavily pre-treated with a median of three prior lines of therapy. A total of 37 patients had NF2 mutations. Ongoing dose optimization cohorts of the study are investigating different doses and schedules in two-stage designs. "These are encouraging results that provide the first clinical proof-of-concept for drugging the Hippo-YAP-TEAD pathway," said Dr. Yap. "Preclinical data suggested that blocking the YAP-TEAD interaction could shrink tumors, and these clinical data validate that approach. These are early clinical trial data, but we look forward to future results from this trial and continuing to investigate the benefits to patients." VT3989 was well tolerated with no dose-limiting toxicities observed. The most common adverse events were albuminuria (a reversible increase in the protein albumin in the urine), swelling in the extremities, fatigue, and nausea. There were only seven grade 3 adverse events that were possibly treatment-related, which included albuminuria, swelling, fatigue, and increased liver enzymes alanine transaminase and aspartate aminotransferase, and one possibly related grade 4 cardiomyopathy. Based on robust efficacy seen in dose escalation and plasma levels of VT3989 at and exceeding the concentrations projected for activity, the study has now expanded in patients with mesothelioma to define the dose and schedule that will become the basis of registrational development in patient with mesothelioma. "It is gratifying to see the extensive preclinical data that we have generated for VT3989 translate so well into humans in both the anti-tumor activity and tolerability profile demonstrated in this clinical study. We view this as a significant milestone as these data position VT3989 as the very first cancer treatment targeting the Hippo pathway to show clinical relevance," said Sofie Qiao, Ph.D., chief executive officer of Vivace Therapeutics. "Based on this initial successful translation of preclinical data into humans, we now have high levels of confidence in a registrational path for VT3989 as a single agent in mesothelioma as well as in combination with targeted therapies to expand utility into major solid tumors. We look forward to continuing our development efforts in mesothelioma and proving the combination treatment hypothesis in a clinical setting." The AACR presentation will be available at after the conference. In additional news from the AACR conference, Vivace Therapeutics announced that Tracy Tang, Ph.D., the company's vice president of biology was named winner of the inaugural Molecular Cancer Therapeutics Award for Outstanding Journal Article by AACR. The award is given to the authors of a Molecular Cancer Therapeutics article published in 2021-2022 that has had significant impact on the fields represent by the journal. It was presented to Dr. Tang and her co-authors for the paper entitled, "Small Molecule Inhibitors of TEAD Auto-palmitoylation Selectively Inhibit Proliferation and Tumor Growth of NF2-deficient Mesothelioma," at the AACR Journals Reception during the conference. About Phase 1 study of VT3989 The Phase 1 study of VT3989 () is a multi-center, open label trial designed to evaluate the safety, tolerability, pharmacokinetics (PK) and biological activity of VT3989 in patients with refractory metastatic solid tumors, including refractory pleural malignant mesothelioma. The study includes both a dose escalation and a dose expansion phase, the latter of which is enrolling mesothelioma patients who have tumors with or without NF2 mutations. About Vivace Therapeutics, Inc. Vivace Therapeutics is a small molecule drug discovery and development company focused on targeting the Hippo pathway. The company is pursuing a first-in-class approach to treat human carcinomas of high unmet medical need. Based in San Francisco Bay Area, the company has raised $70 million to date, and is funded by leading biotechnology investors, including Canaan Partners, WuXi Healthcare Ventures, Cenova Capital, Sequoia Capital China, Boxer Capital and RA Capital Mangement. For more information, please visit . Contact information Sofie Qiao, Ph.D. President and CEO [email protected] 650.666.2753 Tim Brons Vida Strategic Partners (media) 646-319-8981 [email protected] SOURCE Vivace Therapeutics, Inc.