FDA Grants Orphan Drug Designation to JBI-802 for Small Cell Lung Cancer and Acute Myeloid Leukemia

The FDA gave the dual epigenetic modulator JBI-802 an orphan drug designation to treat small cell lung cancer and acute myeloid leukemia.

The FDA has granted JBI-802 orphan drug designation for patients diagnosed with small cell lung cancer (SCLC) and acute myeloid leukemia (AML), according to a press release from Jubilant Therapeutics.

The regulatory decision was supported by a handful of preclinical models. In particular, JBI-802 demonstrated unique clinical activity in patients with SCLC, not only in standard neuroendocrine models but in MYC-amplified variant models, as well.

The findings further support an ongoing phase 1/2 study (NCT05268666) in a population of patients with advanced solid tumors of neuroendocrine differentiation.

Unique activity was also observed in patients with AML in erythroleukemia models, a rare leukemia subset.

JBI-802 is a dual epigenetic modulator that was created in a single pharmacophore to yield ideal inhibition of the transcriptional regulator of CoREST—known to regulate cellular lineage development in neuroendocrine-based tumors and hematopoietic tumors such as SCLC and AML, respectively.

The phase 1/2 trial is set to determine the maximum tolerated dose and recommended phase 2 dose of JBI-802 in patients with solid malignancies. The study will also assess preliminary efficacy and exploratory pharmacodynamic biomarkers. The multicenter, first-in-human open-label trial has an estimated enrollment of 126 patients.

The expansion cohort will include patients with SCLC, neuroendocrine prostate cancer, and other neuroendocrine-derived cancer who will be treated at the RP2D. Treatment will start at 10 mg once daily using a 4 days on, 3 days off schedule.

The primary outcomes are maximum-tolerated dose and overall response rate, with key secondary end points including safety, duration of response, and overall survival.

To be included in the study, patients need to be 18 years or older at screening with an absolute neutrophil count of 1500 cells/mm3 or more, a platelet of 100,000 cells/mm3, and total bilirubin of 1.5xULN or less. Additional, inclusion criteria include having an alanine transaminase/aspartate aminotransferase level of 2.5xULN or less, creatinine clearance of 60 mL per minute or more.

Patients also needed to have at least 1 measurable lesion on CT or MRI and an ECOG performance status of 0 to 2.

Those with central nervous system disease that were not stable or treated or other severe or unstable medical conditions such as congestive heart failure, ischemic heart disease, uncontrolled hypertension, uncontrolled diabetes, or a psychiatric condition were not eligible for enrollment.

Other exclusion criteria included Ise of strong inhibitors or inducers of CYP3A within 14 days, inhibitors or inducers of CYP2D6, a history of or concurrent cancer, and surgery were also not eligible to enroll.


Jubilant Therapeutics Inc. receives orphan drug designation for JBI-802 for acute myeloid leukemia (AML) and small cell lung cancer (SCLC). News release. Jubilant Therapeutics. January 5, 2023. Accessed January 13, 2023. http://bit.ly/3H5xF7q