# Ph 2 Multicenter study of seliciclib for Cushing Disease IND 120,848 (10/10/2017)

> **NIH FDA R01** · CEDARS-SINAI MEDICAL CENTER · 2023 · $467,775

## Abstract

PROJECT SUMMARY
The proposed phase 2 multicenter study will evaluate the small molecule selective CDK2/cyclin E inhibitor seliciclib
in patients with Cushing disease. This rare disease, with an estimated prevalence in the United States of
approximately 20,000 cases, results from an adrenocorticotrophic hormone (ACTH)-secreting pituitary corticotroph
adenoma and predisposes patients to serious morbidities and increased mortality, mostly due to cardiovascular
complications. The current multimodal standard of care of surgical, radiotherapeutic, and medical treatments for this
endogenous hypercortisolism is usually not sufficient to control disease, and can lead to serious sequelae. There is a
clear unmet need for a medical therapy that directly suppresses pituitary corticotroph ACTH secretion and
normalizes cortisol levels in patients with this rare disease.
 Preclinical discoveries led to identification of seliciclib (R-roscovitine), which directly targets processes
promoting ACTH overproduction and the consequent hypercortisolism that underlies the excess morbidity and
mortality associated with Cushing disease. Preliminary data from an NIH-funded pilot phase 2 study indicate efficacy
of oral seliciclib without conferring adrenal side effects seen with current medications used for this disease. The
proposed expanded phase 2 multicenter trial has an adaptive design and will evaluate the safety and efficacy of two
of three doses/schedules of seliciclib in patients with de novo or recurrent Cushing disease. Up to 30 subjects will
be recruited, consented, and enrolled in consecutive dose cohorts through a collaborative effort among all five major
specialty pituitary centers in greater Los Angeles. Results of this trial will lay the foundation for additional trials of
this novel, highly targeted agent for treatment of this rare disease.

## Key facts

- **NIH application ID:** 10003845
- **Project number:** 5R01FD006106-03
- **Recipient organization:** CEDARS-SINAI MEDICAL CENTER
- **Principal Investigator:** SHLOMO MELMED
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2023
- **Award amount:** $467,775
- **Award type:** 5
- **Project period:** 2018-09-01 → 2025-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10003845

## Citation

> US National Institutes of Health, RePORTER application 10003845, Ph 2 Multicenter study of seliciclib for Cushing Disease IND 120,848 (10/10/2017) (5R01FD006106-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10003845. Licensed CC0.

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