CK2

TAIPEI and SAN DIEGO, Sept. 13, 2024 /PRNewswire/ -- Senhwa Biosciences, Inc. (TPEx: 6492), a new drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and infectious diseases, today announced that its new drug Silmitasertib (CX-4945) was granted a rare pediatric disease designation (RPDD) by US FDA for the treatment of neuroblastoma. This is the recognition obtained again after Silmitasertib (CX-4945) received RPDD in medulloblastoma from the FDA in July 2020, demonstrating the great potential of Silmitasertib (CX-4945) in the development of treatments for rare pediatric cancers. Silmitasertib is a first-in-class small molecule drug that targets the CK2 protein and acts as a CK2 inhibitor that exhibited antitumor activity in pre-clinical study of neuroblastoma. The RPD designation will qualify the drug for the priority review voucher (PRV) program meant to encourage development of novel therapies for rare pediatric diseases. Silmitasetib has previously granted 1 RPD and 3 orphan drug designations (ODD) by the FDA. "The RPD designation offers sponsors the additional incentive by requesting priority review vouchers (PRVs) for their future marketing applications, and this approach encourages the drug development for the treatment of rare pediatric diseases," said Jin-Ding Huang, PhD, CEO of Senhwa Biosciences, Inc. Neuroblastoma, an embryonic tumor of the peripheral sympathetic nervous system, is the third most common pediatric cancer and the most common cancer in infants. It is mostly diagnosed in infancy at an average of 1 to 2 years old. There are an estimated 700 to 800 new cases of neuroblastoma each year in the United States. While low and intermediate-risk neuroblastoma have a very high rate of survival, high-risk neuroblastoma has a 5-year survival rate of about 50%, and it has a high likelihood of recurrence even with intense multimodal treatments. There is currently no standard treatment for patients with relapsed/refractory neuroblastoma. SOURCE Senhwa Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

New study will use Unravel's AI platform to stratify patients into subgroups based on their likelihood to respond to prioritized drugs identified in earlier phase of research. BOSTON, Aug. 28, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, and the CSNK2A1 Foundation ("Foundation") today announced that they are expanding their drug discovery collaboration to now stratify Okur-Chung Neurodevelopmental Syndrome (OCNDS) patients into subgroups based on their predicted response to selected drugs. Through a previously announced collaboration, Unravel's proprietary BioNAV™ drug discovery platform used RNA data from patient-derived cells and mouse models to identify in silico drug hits. The Unravel study revealed that patient response to predicted drugs may vary significantly based on the location of their specific CSNK2A1 gene mutation causing OCNDS. The Foundation independently confirmed several in silico hits in a Drosophila fruit fly animal model of OCNDS that have the potential for clinical benefit through a screen with Dr. Clement Chow at the University of Utah. As part of its rareSHIFT™ discovery services, Unravel will utilize its proprietary nasal swab collection methodology in the new project to collect RNA transcriptomes from OCNDS patients and matched healthy family members. The data will be processed using BioNAV™ to identify drug candidates most likely to work for patients with specific genetic mutations. This project focuses on the most prevalent OCNDS-causing mutation, K198R, located in the activation segment of CSNK2A1. Currently the Foundation has over 70 individuals registered with this specific variant. "Given that even monogenetic disorders have substantial variability in how they clinically present and respond to drugs, having a data-driven preview of drug response in real patients is essential to clinically derisk preclinical programs like this," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "By matching each patient to a drug candidate, we will increase the probability of success in clinical trials and reduce the time and cost of developing meaningful therapies for OCNDS patients." "This innovative approach allows us to move beyond a one-size-fits-all model, offering the possibility of more personalized and effective therapies for our community," explained Gabrielle Rushing, Ph.D., CSNK2A1 Foundation Science Program Director. "The insights gained from stratifying patients by their specific genetic mutations will be invaluable in our ongoing mission to accelerate the development of meaningful treatments for OCNDS." First described in 2016, OCNDS is an ultra-rare genetic syndrome caused by a mutation on chromosome 20 in the CSNK2A1 gene. OCNDS affects every aspect of life, with symptoms ranging from mild to severe and can include the inability to speak or delayed speech, global developmental delay, epilepsy, autism spectrum disorder traits, behavioral challenges, hypotonia, feeding difficulty, and severe sleep disruption. To date, there is no treatment for OCNDS. While the overall prevalence of OCNDS is not fully known, over 250 individuals worldwide are currently registered with the Foundation. About Unravel Biosciences Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. and About CSNK2A1 Foundation CSNK2A1 Foundation is focused on finding a cure for Okur-Chung Neurodevelopmental Syndrome and ensuring affected individuals have the opportunities and supports necessary for happy and full lives. CSNK2A1 Foundation is a 501(c)(3) non-profit organization that is operated and funded by a committed team of volunteers, advocates, and researchers. To date, the foundation has registered over 250 individuals with OCNDS in its patient registry worldwide. SOURCE Unravel Biosciences, Inc.

TAIPEI, July 11, 2024 /PRNewswire/ -- Senhwa Biosciences, Inc. (TPEx: 6492), announced IND Submission to US FDA for the Phase I/II study of Silmitasertib (CX-4945) in combination with chemotherapy in children and young adults with relapsed refractory solid tumors. This investigator-initiated trial (IIT) will be conducted by the Penn State Health Children's Hospital and the prestigious Beat Childhood Cancer Research Consortium, a group of over 50 universities and children's hospitals, based at Penn State College of Medicine in Hershey, Pa., that offers a worldwide network of pediatric cancer clinical trials. The funding is sponsored by the Four Diamonds Foundation, with Senhwa Biosciences providing the investigational drug, Silmitasertib (CX-4945). The clinical trial is conducted in two phases: the first phase focuses on establishing the safety and dosage of Silmitasertib (CX-4945) in pediatric patients with relapsed or refractory solid tumors, while the second phase evaluates its efficacy and potential as a novel treatment option. As high CK2 activity is noted across several pediatric cancers, including neuroblastoma, Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, medulloblastoma, and liposarcoma. Recent study has shown that CK2 is one of the key kinases that is essential for maintaining the stabilization of MYCN protein, the oncogenic driver in neuroblastoma. In view of the anti-tumor activity of CK2 inhibitor, the Beat Childhood Cancer Research Consortium at The Pennsylvania State University regards Silmitasertib (CX-4945) high therapeutic potential of treating pediatric cancers. Neuroblastoma is the most common type of solid malignant tumor in children, aside from brain tumors and lymphomas. Over 90% of cases are diagnosed before the age of 5. 70% of patients already have metastatic disease by the time symptoms appear, and the 20-year survival rate is only around 30%. In the US, there are 700-800 new cases each year, accounting for about 6% of childhood cancers, meeting the definition of a rare disease. Senhwa Biosciences is planning to apply for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD) for Silmitasertib (CX-4945) for the treatment of neuroblastoma. If these designations are granted and the drug is successfully commercialized, the company would obtain a Priority Review Voucher (PRV). The holder of a PRV can designate any future human drug application to receive priority review, potentially shortening the review time to 6 months, which could accelerate the timeline for the company (or its partners) to bring other products to market. The clinical trial design also includes Ewing's sarcoma and osteosarcoma, which are common pediatric bone cancers with poor prognoses, representing unmet medical needs. SOURCE Senhwa Biosciences, Inc.