Aadi Bioscience Announces the Publication of its Phase 2 Registration Trial (AMPECT) of nab-sirolimus in Patients with Perivascular Epithelioid Cell Malignancies in the Journal of Clinical Oncology


LOS ANGELESAadi Bioscience, Inc. (“Aadi”) (Nasdaq: AADI), a clinical-stage biopharmaceutical company focused on precision therapies for genetically defined cancers with alterations in mTOR pathway genes, announced the publication of “nab-Sirolimus for Patients with Malignant Perivascular Epithelioid Cell Tumors ”, detailing its AMPECT study of experimental ABI-009 in the American Society of Clinical Oncology Journal of Clinical Oncology. The authors concluded that investigational nab-sirolimus (ABI-009, formerly known as nab-rapamycin), if approved, could represent an important new treatment option in malignant PEComa, a rare cancer and a form aggressive sarcoma, with no currently approved treatment.

The content of the publication included the primary analysis, which occurred 6 months after the start of treatment with the last patient in the AMPECT study, as well as a one-and-a-half-year follow-up after the primary analysis with a data threshold of November 23, 2020. In the trial, 34 patients were treated with ABI-009. In the 31 evaluable patients, the independently assessed overall response rate was 39% (12 of 31; with 95% confidence interval, 22% to 58%) with 1 complete response and 11 partial responses. In addition, 52% (16 of 31) of patients had stable disease. Responses were durable and the median duration of response was not reached after a median follow-up of response of 2.5 years, and 7 of 12 responders with ongoing treatment (range 5.6 to 47.2 month). Responses were rapid onset (67% at week 6). The safety profile was found to be acceptable with most treatment-related adverse events characterized as low grade and manageable for long-term treatment.

André Wagner, MD, Ph.D., senior oncologist at Dana-Farber Cancer Institute and principal investigator of the study, said, “We are delighted to publish the data obtained from this trial. nab-Sirolimus provided very durable responses in patients with advanced PEComa. Most responder patients achieved a rapid response on their first assessment 6 weeks after initiation of treatment and these patients continued treatment for extended periods of time with an acceptable safety profile. We are encouraged by the results of this very first prospective clinical trial in patients with this extremely rare disease ”.

Neil desai, Ph.D., co-author of the article and founder, CEO and President of Aadi Biosciences, say it Aadi Biosciences The team is extremely grateful to the patients, families and clinical trial teams who help push the boundaries of available care through their participation in clinical trials. The publication of these results is an important step not only for patients with malignant PEComa, but also for the ongoing development of nab-sirolimus as part of the planned investigation in the TSC1 and TSC2 indications and other strategies of ‘association.’

A new drug application for investigational ABI-009 is under review by the United States Food and Drug Administration for the treatment of patients with PEComa and has a user of prescription drugs Action on costs target date of November 26, 2021. If approved, ABI-009 will be known as FYARRO.

On Aadi Biosciences and FYARRO

Aadi is a clinical-stage biopharmaceutical company developing precision therapies for genetically defined cancers. Aadi’s main goal is to provide transformational therapies to cancer patients with alterations in the motor of the mTOR pathway such as alterations in the TSC1 or TSC2 genes, where other mTOR inhibitors do not or cannot. not be operated effectively due to pharmacology, effective drug delivery, safety or efficacy issues targeting the disease site. Aadi’s lead product candidate is FYARROTM (sirolimus albumin-bound nanoparticles for suspension for injection; nab-sirolimus; ABI-009), a human albumin-bound mTOR inhibitor that has demonstrated significantly greater tumor accumulation increased suppression of the mTOR target and increased inhibition of tumor growth. compared to other mTOR inhibitors in preclinical models1.

Aadi’s FYARRO Registration Trial in Advanced Malignant PEComa (the “AMPECT Trial”) demonstrated clinically significant efficacy in malignant PEComa2, a type of cancer with the highest known rate of damage. TSC1 or TSC2 genes. FYARRO has received Breakthrough Therapy, Fast-Track and Orphan designations from the United States Food and Drug Administration (FDA). A continuous New Drug Application (NDR) was completed in May 2021 for this indication and the FDA accepted the NDA in July 2021 and granted Aadi Priority Review status with a Prescription Drug User Fee Act (‘PDUFA’) target action date of November 26, 2021.

Based on the AMPECT trial and emerging data for FYARRO in other solid tumors with alterations inactivating TSC1 or TSC23 and following discussions with the FDA, Aadi plans to initiate a solid tumor agnostic registration trial mTOR inhibitor naive harboring TSC1 or TSC2 inactivating modifications by the end of 2021. Aadi also has ongoing studies to evaluate the dosage of FYARRO in combination treatment regimens. FYARRO is an investigational drug that has not been approved by the FDA for commercial distribution in United States. Further information is available on Aadi’s website at www.aadibio.com.

Forward-looking statements

Aadi Bioscience, Inc. (“Aadi”, “The Company”) warns you that certain statements included in this press release which are not a description of historical facts are forward-looking statements. These statements are based on Aadi’s current beliefs and expectations. Forward-looking statements include statements regarding: FYARRO, including expectations regarding clinical responses and safety profile, regulatory approval and marketing, and the timing of the initiation of additional clinical trials. Actual results could differ materially from those anticipated in these forward-looking statements due to these risks and uncertainties, which include, but are not limited to: risks relating to Aadi’s ability to obtain, or the timing to obtain, the regulatory approval from the FDA and other regulatory authorities for FYARRO in advanced malignant PEComa; risks related to Aadi’s ability to market successfully, including the timing of the commercial launch of FYARRO in the advanced malignant PEComa; uncertainties associated with the clinical development and regulatory approval of FYARRO, including potential delays in the initiation, recruitment and completion of clinical trials; the risk that interim results of clinical trials may not be reproduced and may not necessarily predict final outcomes; the risk that one or more of the clinical outcomes may change significantly as patient recruitment continues, as a result of more comprehensive data reviews, and as more patient data becomes available; the risk that unwanted effects or unforeseen side effects may occur during the development and testing of FYARRO; risks associated with the inability to realize any value from FYARRO in light of the risks and difficulties inherent in successfully bringing product candidates to market; risks associated with Aadi’s estimates of future expenses, capital requirements and the need for additional financing; and the risks associated with the impact of the COVID-19 epidemic on Aadi’s operations, the biotech industry and the economy in general.

Additional risks and uncertainties that could cause actual results to differ materially from those contemplated by forward-looking statements are included under “Risk Factors” and elsewhere in Aadi’s reports and other documents that Aadi has. deposited or will deposit with SECOND from time to time and available at www.sec.gov.

All forward-looking statements contained in this press release are only current as of the date hereof and, except as required by applicable law, Aadi does not undertake to revise or update any forward-looking statement, or to make any other forward-looking statement, whether as a result of new information, future events or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement. This warning is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.



Irina Koffler

LifeSci Advisors LLC

E: [email protected]

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