OR-449 / Orphagen - LARVOL DELTA

Tick diversity and molecular detection of Anaplasma, Babesia, and Theileria from Khao Kheow open zoo, Chonburi Province, Thailand. (PubMed, Front Vet Sci) - "A total of 10,436 ticks or 449 tick pools (1-50 ticks per pool) underwent screening for pathogen presence through conventional PCR and DNA sequencing...The findings could be valuable in developing programs for the treatment, prevention, and control of tick-borne illnesses in this area. However, additional research is required to determine the ticks' ability to transmit these pathogens and enhance the current understanding of the relationship among pathogens, ticks, and hosts."

Journal • Infectious Disease

Orphagen Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for OR-449 for the Treatment of Pediatric Adrenocortical Carcinoma (Businesswire) - "Orphagen Pharmaceuticals...announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC)....OR-449 is a selective, first-in-class, potent and orally bioavailable small molecule antagonist to steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland. Orphagen is developing OR-449 for both the adult and pediatric forms of ACC as well as other cancers known to express a high level of SF-1....'We currently plan to file an Investigational New Drug (IND) application with the FDA later this year to support initiation of a Phase 1 clinical trial'..."

FDA event • IND • Adrenal Cortex Carcinoma • Endocrine Cancer • Oncology • Solid Tumor

Downstream Targets of the Steroidogenic Factor-1 (SF-1) Antagonist OR-449 in PDX Models of ACC (ENDO 2022) - "Learning Objective 1: Identify and summarize the potential biomarkers that could be used to monitor the activity of SF-1 antagonists in an adrenocortical carcinoma clinical trial. Science Topic(s) for Tumor Biology: Cancer Targets and Therapies"

Adrenal Cortex Carcinoma • Endocrine Cancer • Genito-urinary Cancer • Oncology • Pediatrics • Solid Tumor

"#OrphagenPharmaceuticals Presents Data on Downstream Targets of #SF1 #Antagonist #OR449 at #ENDO22 https://t.co/ypuMDu7hFY" (@1stOncology)

Orphagen Pharmaceuticals Presents Data on Downstream Targets of SF-1 Antagonist OR-449 at ENDO 2022 (Businesswire) - "Orphagen Pharmaceuticals...announced that additional data from its preclinical program to develop OR-449 for the treatment of adrenocortical cancer (ACC) was presented at the 104th Annual Meeting of the Endocrine Society (ENDO 2022) being held in Atlanta from June 11 to 14. ACC is a rare endocrine malignancy frequently diagnosed at an advanced stage with very limited treatment options. OR-449 is in preclinical development for treatment of ACC with a first-in-human clinical trial targeted for early 2023....Not only does OR-449 inhibit the growth of this patient-derived xenograft, but it also regulates the levels of circulating adrenocortical tumor-derived steroids and steroid precursors."

New trial • Preclinical • Adrenal Cortex Carcinoma • Endocrine Cancer • Oncology • Solid Tumor

Antagonism of SF-1 as a potential targeted therapy for malignant Leydig cell tumors (AACR 2022) - "A novel steroidogenic factor-1 antagonist, OR-449, as a targeted therapy for adrenocortical cancer. ENDO 2021: J Endocr Soc, Vol5, Supplement_1, A1010"

Late-breaking abstract • Adrenal Cortex Carcinoma • Genito-urinary Cancer • Oncology • Solid Tumor • Testicular Cancer

Orphagen Pharmaceuticals Presents Preclinical Efficacy Data at AACR 2022 on OR-449, a First-in-Class Steroidogenic Factor 1 Inhibitor, for the Treatment of Leydig Cell Tumors (Businesswire) - "In cell lines lacking SF-1 expression, such as HEK293, OR-449 has no anti-proliferative activity up to 20 mM, indicating that the anti-proliferative effect on R2C is SF-1-mediated and is not due to cytotoxicity. OR-449 inhibits R2C xenograft tumor growth in immunocompromised mice at oral doses of 3, 10, and 30 mg/kg/day, with complete inhibition at a daily dose of 30 mg/kg....Orphagen has initiated IND-enabling studies of OR-449 with an anticipated IND-filing date at the end of 2022."

IND • Preclinical • Oncology • Solid Tumor

[VIRTUAL] A Novel Steroidogenic Factor-1 Antagonist, OR-449, as a Targeted Therapy for Adrenocortical Cancer (ENDO 2021) - "Furthermore, the non-specific adrenolytic and highly toxic FDA-approved chemotherapy drug, mitotane, is only marginally effective in adult and pediatric ACC. Importantly, in an exploratory 2-week mouse safety study, OR-449 showed no adverse effects when dosed up to 100 mg/kg. Taken together, these findings suggest that SF-1 antagonists could provide a safe and effective targeted therapy for ACC."

Adrenal Cortex Carcinoma • Genito-urinary Cancer • Oncology • Pediatrics • Solid Tumor