# Phase 2 study of ABI-009 Treatment of Advanced PEComa-IND 125669, 7/13/2015

> **NIH FDA R01** · AADI, LLC · 2020 · $499,631

## Abstract

PROJECT SUMMARY
Perivascular epithelioid cell tumors (PEComa) are a rare subset of soft tissue sarcomas. PEComas
arise most commonly at visceral (especially gastrointestinal and uterine), retroperitoneal, and
abdominopelvic sites. Most PEComa lesions are benign and slow-progressing, however, a small subset
of them are malignant PEComas, with an aggressive clinical course including distant metastases and
ultimate death. Malignant PEComa is extremely rare, with an estimated incidence of 0.12-
0.24/1,000,000 or approximately 42-84 new patients per year, and an estimated prevalence of 0.22-
0.48/1,000,000 or approximately 77-168 patients in the United States.
Currently, no effective medical treatment has been prospectively investigated or approved for advanced
malignant PEComa, including metastatic or locally advanced disease where surgery is not an option.
The prognosis for these patients is poor, with an estimated median survival of 12-17 months.
Chemotherapy and radiotherapy have not demonstrated significant clinical benefit. Therefore, a
significant unmet need exists for effective therapies to treat this aggressive and life-threatening disease.
The cytosolic kinase mTOR plays a major role in cell survival and proliferation. Limited case studies
suggest that the mTOR pathway is frequently deregulated in sporadic malignant PEComa (mostly with
mutation or loss of TSC2), making mTOR inhibition a promising therapeutic strategy. Rapamycin blocks
mTORC1 signaling, resulting in decreased protein translation and G1 cell cycle arrest. In some case
studies, patients with PEComa benefited from treatment with mTOR inhibitors, including oral rapamycin.
Oral mTOR inhibitors have low and highly variable oral bioavailability, poor solubility, and dose-limiting
GI toxicities and require therapeutic monitoring of blood levels to ensure adequate dosing. A novel
albumin-bound nanoparticle rapamycin (sirolimus) was developed (nab-rapamycin, ABI-009; originally
by Abraxis Bioscience/Celgene Corporation and licensed to AADi, LLC). In a phase 1 clinical study,
ABI-009 administered intravenously showed a promising safety despite high dose with evidence of
responses and stable disease in a variety of solid tumors. Albumin bound drugs have shown enhanced
tumor penetration, and ABI-009 shows significantly improved activity in animal models at equal dose
and superior pharmacological properties with significantly higher Cmax and AUC without compromising
safety as compared to the available mTOR inhibitors.
AADi, LLC, a small start-up company and applicant for this grant, has filed IND 125,669 (submitted Jul
13, 2015 and approved Aug 13, 2015) to conduct a single arm phase 2 clinical study (NCT02494570) to
assess the efficacy and safety of intravenous ABI-009 for advanced (locally advanced and metastatic)
malignant PEComa. There are no other ongoing trials for this patient population. Thirty-five patients will
be enrolled and the primary endpoint will be ORR; the secondary endpo...

## Key facts

- **NIH application ID:** 9999515
- **Project number:** 5R01FD005749-04
- **Recipient organization:** AADI, LLC
- **Principal Investigator:** Neil Desai
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $499,631
- **Award type:** 5
- **Project period:** 2017-07-15 → 2021-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9999515

## Citation

> US National Institutes of Health, RePORTER application 9999515, Phase 2 study of ABI-009 Treatment of Advanced PEComa-IND 125669, 7/13/2015 (5R01FD005749-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9999515. Licensed CC0.

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