RGT-61159

First-in-human Phase 1a/1b clinical trial will evaluate orally available, RGT-61159 designed to selectively target MYB RNA and inhibit oncogenic MYB protein production Preclinical data supporting the clinical trial were recently presented at the American Society of Clinical Oncology Annual Meeting (ASCO 2024) WOBURN, Mass., July 10, 2024 /PRNewswire/ -- Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML). "Clearance of our first IND application is a significant milestone in Rgenta's mission to develop oral, small molecule RNA-targeting medicines to treat previously incurable diseases," said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. "We look forward to initiating clinical studies of RGT-61159 with a first-in-human Phase1a/1b clinical study in adults with ACC and CRC to potentially provide a new therapeutic option for patients with these difficult-to-treat cancers." "Development of small molecule drugs targeting oncogenic drivers such as MYB, has proven challenging in the past," said Travis Wager, Ph.D., co-founder, president and chief scientific officer. "RGT-61159 demonstrates potent inhibition of oncogenic MYB protein production and significant inhibition of tumor growth at tolerated doses in preclinical models of ACC and other cancers, and we are excited to move this novel therapeutic into clinical evaluation." About RGT-61159 RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of oncogenic MYB protein production, which has the potential to inhibit proliferation or induce cell death of cancer cells that overexpress MYB protein. MYB acts as a master regulator of cell proliferation differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemias (AML), T-cell acute lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Additional information on the planned Phase 1a/1b clinical trial can be accessed at clinicaltrials.gov (NCT06462183). About Adenoid Cystic Carcinoma (ACC) It is estimated that approximately 200,000 people are living with ACC throughout the world including 11,000 in the US. While it is a rare cancer, ACC is the second most common cancer type arising in the salivary gland and is an aggressive malignancy with a tendency to infiltrate surrounding nerves and metastasize to distant sites. Overactivation of the MYB oncogene has been described as a hallmark of ACC and is noted in over 90% of ACC. Treatment for ACC is extremely challenging and may include surgery and/or radiation, which often fails to control local tumor recurrence and distant metastases. There are no effective targeted therapies available for patients with recurrent and/or metastatic disease. There is thus an unmet medical need for new therapeutic targets and treatment strategies for patients with this fatal cancer. About Colorectal Cancer (CRC) CRC is the third most prevalent cancer and the second leading cause of cancer-related mortality worldwide. According to the World Health Organization, in 2022, more than 1.9 million cases of CRC were diagnosed. Despite the improved early detection of CRC and the recent success of targeted therapeutics, approximately 15%-30% of patients present with metastases and 20%-50% of patients with initially localized disease will develop metastases. Patients with relapsed or refractory CRC who have exhausted all the available standard of care therapy options, have a very poor prognosis. MYB is significantly overexpressed in 80-85% of CRC and has been frequently found to be a predictive biomarker of tumor aggressiveness and poor prognosis. Developing novel therapies to treat patients with metastatic CRC remains a major unmet medical need. About Rgenta Therapeutics Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our lead programs and unique approach are unlocking the therapeutic potential of historically undruggable targets in human diseases. Learn more at . Contacts Investors: Sylvia Wheeler Wheelhouse Life Science Advisors [email protected] Elizabeth Wolffe, Ph.D. Wheelhouse Life Science Advisors [email protected] Media Aljanae Reynolds Wheelhouse Life Science Advisors [email protected] SOURCE Rgenta Therapeutics

The once-daily oral capsule has been primarily developed to prevent HAE attacks. Credit: Kmpzzz/Shutterstock. Pint Pharma has announced the approval in Peru of Orladeyo (berotralstat) for use as an oral therapy to prevent hereditary angioedema (HAE) attacks. Orladeyo is a product of BioCryst Pharmaceuticals . Pint Pharma owns its registration, marketing and distribution rights in Latin America (LATAM). Approved by Peru’s General Directorate of Medicines, Supplies and Drugs, it is intended for the prophylaxis of HAE attacks in adults and children aged 12 and above. Hereditary angioedema is a genetic disorder. It results in recurrent episodes of painful swelling of the mucous membranes, skin and submucosal tissues. Pint Pharma scientific director Dr Valnei Canutti stated: “The approval of Orladeyo by DIGEMID is excellent news for the community of patients with hereditary angioedema in Peru. See Also: Hong Kong approves Leqembi for Alzheimer’s treatment Rgenta Therapeutics’ RGT-61159 gains IND approval to treat cancers “This innovative oral therapy represents a breakthrough for those living with this rare condition, offering an effective and convenient long-term prophylaxis option, preventing hereditary angioedema attacks.” The once-daily oral capsule has been primarily developed to prevent HAE attacks by inhibiting plasma kallikrein activity. Pint Pharma CEO David Muñoz stated: “Besides providing direct benefits to patients, the availability of Orladeyo in Peru highlights our continuous commitment to the advancement of research and innovation in the field of medical care for rare diseases.” Pint Pharma LATAM North Cluster general manager Mauricio Botero stated: “The approval of Orladeyo is a great achievement for patients and proof of the successful collaboration among the medical community, regulators, and pharmaceutical companies in Peru to improve treatment options.” In 2023, BioCryst secured marketing authorisation from the Public Health Institute of Chile for ORLADEYO.