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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 DRxTM (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 FalconTM platform, including ThecaFlex DRxTM ,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 FalconTM 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 . MEDIA & INVESTOR CONTACT: Alcyone Therapeutics PJ Anand + 1 978-709-1946 [email protected] SOURCE Alcyone Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Rugonersen, a novel RNA antisense oligonucleotide (ASO), has potential to be the first approved disease-modifying treatment for a disease affecting 500K individuals globally Encouraging clinical efficacy results on exploratory endpoints related to brain function and measures of cognition and communication support further investigation Pivotal Phase 3 study anticipated to be initiated in early 2026 CAMBRIDGE, MA, USA I April 15, 2025 I Oak Hill Bio, a biotechnology company focused on acquiring and developing life-changing rare disease therapeutics, has entered into an exclusive license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) to obtain global rights for rugonersen (RO7248824). Rugonersen is an ASO designed as a potential best-in-class treatment for individuals with Angelman syndrome, a rare, genetic neurodevelopmental condition for which there is no approved disease-modifying treatment. Oak Hill plans to initiate a Phase 3 study in early 2026. “We are excited to take on this program. We are incredibly impressed by the rigorous research Roche has conducted, and the promising clinical data generated to date,” said Josh Distler, Chief Executive Officer of Oak Hill. “We have also been moved by the dedication of the patients, families, and investigators in the Angelman community and look forward to working closely with them to fully evaluate rugonersen’s potential to improve the lives of individuals with Angelman syndrome and their families.” “I am very glad that the development of rugonersen will continue,” said Elizabeth Berry-Kravis, M.D., Ph.D., Pediatric Neurologist and Principal Investigator of the TANGELO trial at Rush University Medical Center. “Many of our trial participants have had meaningful improvements while in the rugonersen trial, and the TANGELO results suggest developmental and functional gains in multiple domains relative to natural history. We are very excited that the potential benefits of rugonersen will continue to be evaluated for these and other patients with Angelman syndrome.” Angelman syndrome affects an estimated 500,000 individuals worldwide. It typically presents during early childhood and is characterized by cognitive and developmental issues, including speech and communication difficulties, motor impairment, balance issues, and debilitating seizures. The condition is caused by a loss of function of the maternally inherited UBE3A gene. “We are incredibly excited and deeply grateful to see Oak Hill stepping in to continue the rugonersen program for Angelman syndrome. We, along with caregivers and providers, have seen the promise of rugonersen to help those with Angelman syndrome. This is a powerful signal to our community who have fought tirelessly for progress. Knowing that this work will continue means everything. We’re thankful for Oak Hill’s commitment to the community and for recognizing the urgent, unmet needs of individuals living with Angelman syndrome. The community stands ready to support this important effort in every way we can,” said Amanda Moore (CEO, Angelman Syndrome Foundation, ASF) and Ryan Fischer (COO, Foundation for Angelman Syndrome Therapeutics, FAST). About rugonersen Rugonersen is a novel RNA antisense oligonucleotide (ASO) developed as a potential treatment option for individuals with Angelman syndrome, a rare genetic neurodevelopmental condition that affects the central nervous system and causes severe physical and learning disabilities. Rugonersen has been designed to modify disease progression by boosting expression of the paternal version of the UBE3A gene. In the TANGELO study, an open-label, non-randomized, adaptive study led by Roche, rugonersen demonstrated encouraging exploratory effects compared to natural history on multiple clinical measures and on a pharmacodynamic biomarker of brain function, EEG delta power. These data were presented at FAST’s Annual Global Science Summit in November 2024 . Oak Hill believes these results support rugonersen’s potential to address multiple disease domains. Pending review of the final Phase 3 protocol by regulatory authorities, Oak Hill anticipates initiating a pivotal Phase 3 trial in early 2026 for rugonersen as a potential treatment for individuals with Angelman syndrome. About Oak Hill Bio Oak Hill Bio Ltd is a clinical-stage biotechnology company focused on acquiring and developing life-changing rare disease therapeutics. The company’s pipeline includes three programs in late-stage clinical development – potential treatments for Angelman syndrome and complications of extremely premature birth – as well as a preclinical program for diabetic macular edema. Oak Hill has operations in the United States and the United Kingdom. For more information, visit the company’s website at www.oakhillbio.com . SOURCE: Oak Hill Bio

EMA orphan drug designation is awarded to drugs intended to treat rare diseases that are life-threatening or chronically debilitating Preeclampsia, a serious obstetric complication, is a leading cause of near-term morbidity and mortality for mothers and their babies, and can lead to long-term consequences for both An investigational drug candidate, CBP-4888 leverages the precise targeting ability of siRNAs to downregulate sFlt1, a key pathogenic mediator of preeclampsia, in the placenta CBP-4888 represents the first potential molecularly targeted therapeutic for an obstetrical indication April 08, 2025 -- Comanche Biopharma Corp. today announced that the European Medicines Agency (EMA) has granted orphan drug designation for CBP-4888 for the treatment of sFlt1-mediated preterm preeclampsia. A serious obstetric complication, preeclampsia is estimated to impact approximately four in 10,000 pregnancies in the European Union each year. A novel siRNA drug candidate, CBP-4888 is designed to downregulate overexpression of the protein sFlt1, a known pathogenic mediator of preeclampsia, in the placenta. Currently an investigational drug candidate, CBP-4888 has the potential to be the first precision medicine for an obstetrical indication. “We are grateful to the EMA for granting orphan designation to CBP-4888 and recognizing preeclampsia as a life-threatening, significant unmet medical need,” said Allison August, M.D., Chief Medical Officer at Comanche Biopharma. “Beyond the acute morbidity and mortality risk that preeclampsia presents for mothers and their babies, it is also associated with serious, lifelong health complications. By targeting a root cause of preeclampsia with a precision medicine, our goal is to provide a therapeutic solution that can prevent the far-reaching, short- and long-term impact of this condition for mothers, babies, families and society at large.” The EMA grants orphan drug designation to drugs and biologics intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that affects fewer than five in 10,000 people in the European Union, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. Sponsors who obtain orphan drug designation benefit from protocol assistance, market exclusivity and regulatory fee reductions. Overexpression of the protein sFlt1 is believed to be a key pathogenic mediator of preeclampsia. When produced in excess by the placenta, sFlt1 is toxic, severely damaging the mother’s blood vessels and impairing the growth of new ones. This overproduction of sFlt1 and subsequent vascular damage result in the common maternal signs and symptoms of preeclampsia and in fetal growth restriction. CBP-4888 is designed to achieve sufficient uptake by the placenta to selectively downregulate the disease-causing overexpression of the sFlt-1 protein in patients with sFlt-1-mediated preterm preeclampsia. CBP-4888 is being investigated for the potential to ameliorate the acute, potentially life-threatening maternal symptoms of preterm preeclampsia. This, in turn, may enable the safe prolongation of pregnancy and continued fetal maturation, thereby minimizing the near-term morbidity and mortality for mothers and their babies, and the potential long-term consequences for both. In addition to receiving Orphan Drug Designation by the EMA, CBP-4888 has also received Fast Track Designation by the U.S. Food and Drug Administration, and Innovation Passport Designation by the U.K. Innovative Licensing and Access Pathway (ILAP) Steering Group. Preeclampsia is a serious pregnancy complication that affects up to 8% of pregnancies worldwide. It can lead to a range of acute, near-term complications for both mothers and their babies, including multi-organ damage, potential progression to eclampsia (onset of seizures) and premature births. Preeclampsia can also result in lifelong consequences, such as a higher risk of heart disease, chronic hypertension, cardiovascular disease and stroke for women, and learning disorders, cerebral palsy, epilepsy, blindness and deafness for infants born prematurely. Globally, there are an estimated 76,000 maternal deaths and 500,000 fetal and newborn deaths annually due to this complication of pregnancy. While preeclampsia can develop in any pregnancy, it disproportionately affects black individuals and those living in low resource settings. The signs and symptoms of preeclampsia can vary and include high blood pressure, acute kidney injury, swelling of the hands and face, severe headaches, vision changes and abdominal pain. Currently, premature delivery of the baby is the only available option for stopping the progression of preeclampsia. Comanche Biopharma is a clinical stage biopharmaceutical company working to make every pregnancy around the world safer by delivering innovative solutions and ensuring they are accessible to patients in need. We are currently developing CBP-4888, an innovative siRNA therapeutic candidate that, if approved, would be the world’s first treatment to target an underlying cause of preeclampsia, an obstetric complication globally responsible for a significant burden of maternal and infant morbidity and mortality, which can also lead to long-term consequences for both mothers and their babies. At Comanche, we are committed to the ethical representation of people of all colors and economic status globally in our clinical development programs. Visit us at comanchebiopharma.com. The content above comes from the network. if any infringement, please contact us to modify.