Cerebral Palsy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Parabilis Medicines, a clinical-stage biopharmaceutical company committed to creating extraordinary medicines for people living with cancer, today announced at the ESMO Congress 2025 the first-ever clinical data showing that its investigational FOG-001 therapy has successfully drugged β-catenin:TCF – a key cancer-driving node in the Wnt/β-catenin pathway, until now considered “undruggable.” In the ongoing Phase 1/2 trial of FOG-001 in patients with a range of Wnt/β-catenin-driven tumors, as of mid-August 2025, 12 patients with desmoid tumors had been dosed across three dose levels. Of the 10 patients who had at least one post-baseline scan, tumor reductions were observed at all dose levels with a 100% disease-control rate (DCR). Of the five patients with more than one post-baseline scan, an objective response rate (ORR) of 80% (4/5) was observed, per RECIST 1.1. Responses were seen in both gamma secretase-naive and –treated patients, and FOG-001 demonstrated clinically meaningful anti-tumor activity alongside acceptable safety and tolerability profiles. FOG-001 achieves its effect by blocking the interaction between β-catenin and the T-cell factor (TCF) family of transcription factors, the key driver of tumorigenesis in Wnt pathway-activated cancer cells. The data support further development of FOG-001 in patients with desmoid tumors, and suggest that FOG-001, unlike other available therapies for this disease, directly addresses the underlying mechanism of disease through inhibition of β-catenin. “For decades, scientists had said that the Wnt/β-catenin:TCF interaction couldn’t be drugged, but our data prove otherwise,” said Mathai Mammen, M.D., Ph.D., Chairman, CEO and President of Parabilis Medicines. “FOG-001 shows what bold science can achieve — taking on one of cancer’s most important drivers and opening the door to an entirely new class of therapies. This milestone validates the power of Parabilis’s distinctive Helicon peptides and marks an important step forward in our mission to create extraordinary medicines for patients.” The Wnt/β-catenin pathway was first identified over 30 years ago as a fundamental driver of cancer and is implicated in millions of cases each year, across both common cancers — including gastrointestinal cancers like colorectal, hepatocellular, and gastric cancers — as well as many rare cancers such as desmoid tumors and adamantinomatous craniopharyngioma (ACP). Attempts to target the β-catenin:TCF interaction, the key downstream node within the Wnt pathway, have repeatedly failed until now with FOG-001. Parabilis’s Helicon™ platform has overcome limitations of traditional small molecule therapeutics by designing stabilized α-helical peptides that gently penetrate cells and bind tightly to proteins with relatively flat binding surfaces. With FOG-001, Parabilis has achieved what decades of cancer research could not: directly drugging the “undruggable” β-catenin:TCF interaction. Beyond ESMO, additional clinical data on FOG-001 across a range of Wnt/β-catenin-driven tumors will be presented at several additional upcoming scientific meetings, including the AACR-NCI-EORTC International Conference on Molecular Targets (October 22-26, Boston, MA), the Connective Tissue Oncology Society (CTOS) 2025 Annual Meeting (November 12-15, Boca Raton, FL), and the Society for Neuro-Oncology (SNO) 2025 Annual Meeting (November 19-23, Honolulu, HI). The company will also share new preclinical data on its allosteric active androgen receptor (ARON) and ERG degrader discovery programs for the treatment of prostate cancer at AACR-NCI-EORTC and the Prostate Cancer Foundation (PCF) Scientific Retreat (October 23-25, Carlsbad, CA). Details of the presentations are as follows: ESMO mini oral information: Presentation Title: “A Phase 1/2 trial of FOG-001, a first-in-class direct β-catenin:TCF4 inhibitor: Safety and preliminary antitumor activity in patients with desmoid tumors” Date and Time: Friday, October 17, 2025, 4:00 – 5:30 p.m. CEST Session: Mini oral session: Sarcoma Location: Essen Auditorium, Hall 7.2A AACR-NCI-EORTC oral and poster information: Title: “A Phase 1/2 trial of FOG-001, a first-in-class direct β-catenin inhibitor, preliminary safety and efficacy in patients with solid tumors bearing Wnt pathway-activating mutations (WPAM+)” Oral Presentation Date and Time: Friday, October 24, 2025, 10:00 – 11:40 a.m. EDT Session: Plenary Session 4: Clinical Trials Plenary Session Location: Level 3, Ballroom AB Poster Presentation Date and Time: Friday, October 24, 2025, 12:30 – 4:00 p.m. EDT Session: Poster Session B Location: Level 2, Exhibit Hall D Poster Title: “Distinct aspects of β-catenin biology drive multiple biologically rational FOG-001 combinations for MSS colorectal cancer” Date and Time: Saturday, October 25, 2025, 12:30 – 4:00 p.m. EDT Session: Poster Session C Location: Level 2, Exhibit Hall D Poster Title: “Degradation of the ETS transcription factor ERG by stabilized helical peptide (Helicon™) degraders enable pharmacological validation in ERG-fusion prostate cancer models” Date and Time: Friday, October 24, 2025, 12:30 – 4:00 p.m. EDT Session: Poster Session B Location: Level 2, Exhibit Hall D Poster Title: “Discovery of Helicon peptides for the selective degradation of the agonist-bound conformation of androgen receptor (ARON) in prostate cancer” Date and Time: Friday, October 24, 2025, 12:30 – 4:00 p.m. EDT Session: Poster Session B Location: Level 2, Exhibit Hall D PCF presentation and poster information: Poster Title: “Discovery of Helicon peptides for the selective degradation of the agonist-bound conformation of androgen receptor (ARON) in prostate cancer” Date and Time: Thursday, October 23, 2025, 7:30 – 10:30 PM PDT Location: Costa De La Luna Ballroom Presentation Title: “Discovery and Optimization of ERG Bifunctional, Stapled Peptide Degraders using Generative AI–Driven Multi-Property Modeling” Date and Time: Saturday, October 25, 2025, 12:45 – 1:00 PM PDT Location: Costa Del Sol Ballroom CTOS poster information: Poster Title: “A Phase 1/2 trial of FOG-001, a first-in-class direct β-catenin inhibitor: Safety and preliminary antitumor activity in patients with desmoid tumors (DT)” Date and Time: Thursday, November 13, 2025, 5:30 – 6:30 p.m. EST Session: Poster Reception: Soft Tissue Tumors SNO mini oral information: Presentation Title: “A Phase 1/2 trial of FOG-001, a first-in-class direct β-catenin:TCF4 inhibitor: Safety and preliminary antitumor activity in adamantinomatous craniopharyngioma (ACP) patients” Abstract Number: CTNI-10 Date and Time: Friday, November 21, 2025, 11:30 – 12:30 p.m. HST Session: Rapid Orals Location: Hawaii Convention Center, Kamehameha Exhibit Hall II & III About the Phase 1/2 trial of FOG-001 FOG-001 is being evaluated in a first-in-human Phase 1/2 multicenter, open-label study (NCT05919264) assessing its safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity. The trial includes dose-escalation and dose-expansion phases and is testing FOG-001 both as a monotherapy and in combination with other anticancer agents in patients with advanced or metastatic solid tumors likely or known to harbor a Wnt pathway–activating mutation (WPAM). About FOG-001 FOG-001 is an investigational first-in-class competitive inhibitor of β-catenin interactions with the T-cell factor (TCF) family of transcription factors and is currently in clinical development. By directly targeting the β-catenin:TCF protein-protein interaction, FOG-001 is intended to block the Wnt signaling pathway irrespective of the various APC and β-catenin mutations that typically drive disease. FOG-001 combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell where it binds directly to the key oncogenic driver β-catenin; and FOG-001 blocks the Wnt pathway at the key downstream node, disrupting the interaction between β-catenin and the TCF transcription factors, thereby abrogating the signal transmission by which Wnt pathway mutations are believed to drive oncogenesis. FOG-001 is currently being evaluated in a first-in-human Phase 1/2 clinical trial in patients with locally advanced or metastatic solid tumors. About Parabilis Medicines Parabilis Medicines is a clinical-stage biopharmaceutical company dedicated to creating extraordinary medicines that unlock high-impact protein targets long-considered undruggable. The company has developed a new class of stabilized, cell-penetrant alpha-helical peptides – Helicons™ – capable of modulating intracellular proteins that are inaccessible to traditional drug modalities. Headquartered in Cambridge, Mass., Parabilis is advancing a focused pipeline of multiple first-in-class therapies across both rare and common cancers. Its lead candidate, FOG-001, is the first direct inhibitor of the interaction between β-catenin and the T-cell factor (TCF) family of transcription factors, implicated in colorectal cancer, desmoid tumors, and a range of other Wnt/β-catenin-driven tumors. Parabilis is also advancing investigational degraders of ERG and ARON for the treatment of prostate cancer, as well as other preclinical programs. Learn more about how the company is advancing a new generation of precision cancer medicines with the potential to meaningfully alter the trajectory of disease for patients in need: www.parabilismed.com.

FREMONT, Calif. & BANGALORE, India--(BUSINESS WIRE)--VIVATRONIX Tech Pvt. Ltd. (Bangalore, India) and SpineX Inc. (Fremont, CA, USA) proudly announce that the Central Drugs Standard Control Organization (CDSCO), India’s national regulatory authority, has approved xStep (powered by SpineX) for clinical and home use in India. Empowering Independence. Restoring Confidence. Lets xStep Forward!! The xStep device delivers painless, noninvasive electrical stimulation to the spinal cord—without the need for surgery—to help restore motor function, mobility, and independence in individuals living with paralysis. Formerly known as SCiP™ or SCONE™, the xStep technology has been recognized by the U.S. Food and Drug Administration (FDA) as a Breakthrough Device for two distinct indications. It has demonstrated promising results in multiple global clinical trials, and SpineX continues to collaborate on investigator-initiated studies validating the safety, efficacy, and transformative potential of noninvasive spinal neuromodulation therapy. “xStep’s CDSCO approval represents a breakthrough for neurorehabilitation—bringing world-class, life-changing therapy within reach for patients in India and beyond,” said Dr. Sachin Kandhari, MD, a leading Key Opinion Leader in neuromodulation and neurorehabilitation. “This is a defining moment for us. With CDSCO approval, patients who have long been waiting for hope and recovery now have access to proven, noninvasive therapy. It’s no longer a question of if—it’s about making it happen now,” said Dr. Parag Gad, PhD, CEO of SpineX Inc. and VIVATRONIX Tech Pvt. Ltd. With CDSCO approval, SpineX Inc. and VIVATRONIX Tech Pvt. Ltd. are poised to expand access to advanced neurorehabilitation technology in India and worldwide—marking a major step toward transforming the standard of care for individuals living with paralysis. About SpineX Inc. SpineX Inc., headquartered in Fremont, California, is a clinical-stage neuromodulation company developing noninvasive spinal cord stimulation technologies aimed at restoring mobility and function for individuals living with neurological disorders such as paralysis and cerebral palsy. About VIVATRONIX Tech Pvt. Ltd. VIVATRONIX Tech Pvt. Ltd., based in Bangalore, India, is dedicated to bringing cutting-edge neurotechnology and rehabilitation solutions to patients across India and emerging markets through collaboration, innovation, and patient-centered care.

UCP’s Research Grant Program fuels innovation by funding projects that improve early diagnosis, treatment, and outcomes for children with cerebral palsy. This year’s record-breaking response underscores the urgency and creativity driving the research community. These projects stand out for their potential to transform how CP is identified and treated.” — Valerie Pieraccini, co-chair of UCP’s Research Council VIENNA, VA, UNITED STATES, September 30, 2025 / EINPresswire.com / -- United Cerebral Palsy (UCP) , a leader in providing resources and assistance to individuals with disabilities, is proud to announce the winners of the 2025 “Change is Possible for CP” Research Grant Program . Two recipients have been selected after a record number of applications were received for its annual program. The“Change is Possible for CP” grant cycle is designed to advance clinical and translational research that bridges the gap between science and care. Starting in January, researchers and clinicians across the U.S. and Canada were invited to submit letters of intent for funding opportunities aimed at transforming the lives of children with cerebral palsy and related neurodevelopmental disabilities. UCP’s Research Council — made up of medical professionals, parents, individuals with disabilities, clinician-researchers, affiliate leaders, and community advocates — guides the Research Grant Program. The program emphasizes “knowledge translation,” applying research insights to real-world care, and funds projects that drive advances in early diagnosis, treatment, and rehabilitation. 2025 Winning Projects UCP’s Research Grant Program fuels groundbreaking studies, enabling clinicians and scientists to explore innovative approaches that can redefine care for individuals with cerebral palsy. The 2025 winning projects exemplify the transformative potential of these grants. California Primary Care Provider Education of Early Identification and Intervention for Infants Primary Investigator: Christiana Butera, University of Southern California This project will expand training for primary care providers across California to improve early detection of cerebral palsy and other developmental disabilities. Building on the Early Identification and Intervention for Infants Network (EI3) initiative, the project will adapt a pilot program from Los Angeles County into a statewide virtual training program, ensuring accessible, evidence-based tools are available to providers and families. Translating Early Diagnosis into Practice: Clinical Validation of BabyOSCAR for Selective Motor Control Assessment in Infants with Probable Cerebral Palsy Primary Investigators: Theresa Sukal Moulton and Colleen Peyton, Northwestern University This study will validate BabyOSCAR, a novel assessment tool that evaluates joint-specific movement using observational scoring of video-recorded spontaneous infant movement. By analyzing 250 retrospective infant videos and testing reliability across clinicians, the project aims to establish BabyOSCAR as a scalable, clinically feasible tool for earlier, more accurate CP diagnosis and individualized intervention. UCP: Driving Innovation in Early Detection and Treatment Each winning project will receive $20,000 in funding to advance its work. Both projects strongly reflect UCP’s priority areas of early diagnosis and early treatment for cerebral palsy, advancing research that translates directly into improved care for children and families. “This year’s record-breaking response underscores the urgency and creativity driving the CP research community,” said Valerie Pieraccini, co-chair of UCP’s Research Council. “The two selected projects stand out for their potential to transform how cerebral palsy is identified and treated — accelerating the path from scientific discovery to clinical impact.” “This year’s awardees represent what our research program strives to achieve,” said Armando Contreras, president and CEO of United Cerebral Palsy. “By supporting projects that expand early detection training for clinicians and validate new diagnostic tools, we are investing in research that will have a direct and lasting impact on children and families. These innovative studies reflect UCP’s commitment to advancing science that doesn’t just remain in the lab but is translated into practice — helping providers deliver earlier, more effective interventions and giving children with cerebral palsy the best chance to thrive.” This year, the UCP Research Council saw a 270% surge in submissions compared to last year — a clear sign of the accelerating momentum, creativity, and commitment driving the future of cerebral palsy research. Learn more about UCP’s Research Grant Program and past awardees at www.ucp.org/research/research-grant-program/ . About United Cerebral Palsy United Cerebral Palsy (UCP) was founded in 1949, and today, its network of affiliate organizations in the United States and Canada provides a wide range of services annually to approximately 200,000 children and adults with cerebral palsy and other disabilities. These services include information, resource referral, advocacy, research, educational instruction, early intervention, therapies, job coaching, integrated employment, home and community-based services, recreational opportunities, and housing assistance. UCP is committed to the mission that all people with disabilities should be treated as equal members of an inclusive society. Learn more at UCP.org. Sarah Peiper United Cerebral Palsy Association +1 800-872-5827 email us here Visit us on social media: LinkedIn Instagram Facebook YouTube Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.