• Expanded collaboration with Nationwide Children's Hospital adds new funding and a third therapeutic candidate (CLN-301) to Alcyone's diversified portfolio of precision CNS therapeutics and delivery technologies
• CLN-301 is a clinical-stage transformative gene therapy in development for CLN3 Batten disease (Juvenile Neuronal Ceroid Lipofuscinosis), which has demonstrated potentially life-altering early efficacy and a robust safety profile in an ongoing Phase 1/2 trial

LOWELL, Mass., April 24, 2025 /PRNewswire/ -- Alcyone Therapeutics ("Alcyone"), a company committed to transforming pediatric care through innovation in precision CNS therapeutics and delivery, today announced closing strategic financing from Nationwide Children's Hospital. The new funding follows an expansion of the collaboration between Alcyone and Nationwide Children's, which added a clinical-stage CLN3 Batten disease gene therapy program (CLN-301) to Alcyone's therapeutic pipeline focused on pediatric neurological diseases. Existing investors also participated in the financing in continued support of Alcyone's development of the ThecaFlex DRx^TM (ThecaFlex) intrathecal drug delivery system. CLN-301 is a potential first-in-class AAV9 gene therapy in Phase 1/2 clinical development for CLN3 Batten disease (Juvenile Neuronal Ceroid Lipofuscinosis), a rare, fatal, inherited neurological disorder with no approved therapy. Treatment with CLN-301 is designed to deliver the coding sequence of the CLN3 gene to cells of the central nervous system to address an underlying enzyme deficiency that results in progressive cell damage and neurodevelopmental and physical decline.

Emily De Los Reyes, M.D., Director of the Batten Disease Center of Excellence at Nationwide Children's commented, "Families of children with Batten disease navigate challenges every day. Continued research and clinical investigation of this rare disease is needed in order to provide care and support to this patient population."

Initial results from a Phase 1/2 trial with CLN-301 demonstrated safety and therapeutic effects in a cohort of four patients compared to the natural progression of CLN3 Batten disease. On the Unified Batten Disease Rating Scale, patients in the CLN-301 study remained stable with a slope of -0.22 points per year over three years. By comparison, an increase of 2.86 points per year, leading to an overall 8.6-point increase in impairment over 36 months would be typical according to natural history data. Typical outcomes for patients with CLN3 Batten disease include rapid vision loss, cognitive and motor decline, and behavioral issues. The majority of patients treated with CLN-301 and followed for over five years to date have shown maintenance of skills in motor and cognitive function and overall health compared to natural history data.

"Based on observations from the patients dosed so far, addressing the underlying CLN3 deficiency with CLN-301 gene therapy has the potential to significantly alter the course of this devastating disease and to meaningfully impact life quality for CLN3 patients and their families," said Kathrin Meyer, Ph.D., Alcyone Therapeutics Chief Scientific Officer.

Amy Fenton Parker, President & CEO of the BDSRA Foundation (Batten Disease Support, Research, and Advocacy Foundation) said, "CLN3 is one of the most prevalent forms of Batten disease. The development of this important gene therapy program by Alcyone can potentially address a significant unmet need, and we are very excited to see this move forward."

"We are grateful for the opportunity to deepen our collaboration with Nationwide Children's Hospital," said PJ Anand, CEO of Alcyone Therapeutics. "With the addition of CLN-301, Alcyone has enriched its clinical-stage pipeline of precision CNS therapeutics with a program exhibiting promising safety and preliminary efficacy data and significant commercial potential as a first-in-class disease-modifying therapy."

CLN-301 is one component of Alcyone's diversified portfolio of CNS therapeutics, which also includes a next-generation genetic medicine for Rett Syndrome (XGR-101) in IND-enabling stage and a preclinical advanced precision-engineered small molecule targeting neurodegeneration (Efralex). The first-in-class and/or best-in-class potential of each of Alcyone's therapeutic assets is augmented by the Company's CNS precision delivery Falcon^TM platform, including ThecaFlex DRx^TM ,which is part of an ongoing co- development and co-promotion collaboration with Biogen. The Falcon intra-cerebrospinal fluid (CSF) drug transport modeling and delivery platform technology is central to Alcyone's therapeutic advancements. Falcon aims to enhance therapeutic efficacy and safety across therapeutic modalities by improving drug biodistribution as well as provide novel delivery options. The novel delivery solutions aim to provide a better quality of life to patients requiring CSF drug dosing, addressing a critical challenge in treating complex neurological conditions.

About CLN3 Batten Disease
Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system that usually begin during childhood. There are 13 known forms of Batten disease in which a defect in a specific gene triggers a cascade of problems that interfere with a cell's ability to recycle certain molecules. CLN3 Batten disease, also known as juvenile neuronal ceroid lipofuscinosis (JNCL), is caused by mutations in the CLN3 (ceroid lipofuscinosis, neuronal 3) gene. CLN3 encodes a transmembrane protein (embedded in the cell membrane) involved in the trafficking and recycling of proteins and other substances within cells.

The clinical course of Batten disease often involves progressive loss of vision and motor skills. Most affected children also experience, personality changes, behavioral problems, learning impairment and seizures. Children suffering from JNCL ultimately become bedridden and die prematurely.

About The Abigail Wexner Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report's 2024-25 list of "Best Children's Hospitals," Nationwide Children's Hospital is one of America's largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children's to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children's Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.

About Alcyone Therapeutics
Alcyone Therapeutics is a clinical-stage biotechnology company committed to transforming pediatric care through a diversified portfolio of precision CNS therapeutics and dosing platforms. The Company is advancing a therapeutic pipeline in collaboration with Nationwide Children's Hospital (AWRI) focused on severe pediatric neurological diseases. Alcyone's lead programs include a clinical-stage gene therapy for CLN3 Batten disease and a miRNA sponge for X chromosome reactivation to treat X-linked genetic disorders, including Rett syndrome. Alcyone's proprietary Falcon^TM precision intra-cerebrospinal fluid (CSF) drug transport modeling and delivery platform technology incorporates deep knowledge of CSF dynamics, computational modeling, and bioengineering. This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. For more information, visit www.alcyonetx.com.

MEDIA & INVESTOR CONTACT:

Alcyone Therapeutics
PJ Anand
+ 1 978-709-1946
ir@alcyonetx.com

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SOURCE Alcyone Therapeutics