AADI Aadi Bioscience Inc.

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Drug Stage Notes
FYARRO
PEComa​
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Approved November 23, 2021.
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Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.

Latest News

  1. LOS ANGELES, Jan. 04, 2022 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi"), a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that Founder, Chief Executive Officer and President, Neil Desai, Ph.D., will present at the H.C. Wainwright BioConnect Conference, to be held virtually January 10-13, 2022. The presentation will be available beginning on Monday, January 10, 2022 at 7:00 a.m. ET.

    The webcast can be accessed here. A replay will also be available on Aadi's website within the News/Events & Presentations section.

    About Aadi Bioscience

    Aadi is a commercial-stage biopharmaceutical company focused on precision therapies for genetically-defined…

    LOS ANGELES, Jan. 04, 2022 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi"), a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that Founder, Chief Executive Officer and President, Neil Desai, Ph.D., will present at the H.C. Wainwright BioConnect Conference, to be held virtually January 10-13, 2022. The presentation will be available beginning on Monday, January 10, 2022 at 7:00 a.m. ET.

    The webcast can be accessed here. A replay will also be available on Aadi's website within the News/Events & Presentations section.

    About Aadi Bioscience

    Aadi is a commercial-stage biopharmaceutical company focused on precision therapies for genetically-defined cancers. Aadi's primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations where other mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site. In November 2021, Aadi received FDA approval for FYARRO for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is an mTOR inhibitor bound to human albumin that has demonstrated significantly higher tumor accumulation, greater mTOR target suppression, and increased tumor growth inhibition over other mTOR inhibitors in preclinical models.

    Based on data from the AMPECT trial with FYARRO and following discussions with the FDA about other emerging data with FYARRO, Aadi plans to initiate a tumor-agnostic registrational trial in mTOR inhibitor-naïve solid tumors harboring TSC1 or TSC2 inactivating alterations by early 2022. Aadi also has ongoing studies to evaluate dosing of FYARRO in combination regimens. More information on Aadi's development pipeline is available on the Aadi website at www.aadibio.com.

    Contacts:

    Investors

    Irina Koffler



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    • FYARRO is the first and only approved therapy for adults for the treatment of malignant PEComa, an ultra-rare and aggressive form of sarcoma with a strong female predominance
    • FYARRO launch planned for Q1 2022; Investor call to be held today at 8:30 am ET    

    LOS ANGELES, Nov. 23, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that the U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic…

    • FYARRO is the first and only approved therapy for adults for the treatment of malignant PEComa, an ultra-rare and aggressive form of sarcoma with a strong female predominance
    • FYARRO launch planned for Q1 2022; Investor call to be held today at 8:30 am ET    

    LOS ANGELES, Nov. 23, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that the U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is the first and only FDA-approved treatment for advanced malignant PEComa in adults.

    Neil Desai, Ph.D., Founder, Chief Executive Officer and President of Aadi, stated, "We are thrilled to have received full FDA-approval of FYARRO. The approval of FYARRO is a momentous event not just for Aadi but, importantly for advanced malignant PEComa patients. We reiterate that all of us at Aadi are incredibly grateful to all of the people with advanced malignant PEComa, their families and caregivers, as well as the healthcare professionals who made the FYARRO clinical studies possible."

    "The approval of FYARRO, the first approved drug for advanced malignant PEComa, an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease," added Andrew Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute and the Principal Investigator in the pivotal AMPECT registrational trial. "In our AMPECT trial, FYARRO demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, with an acceptable and manageable safety profile. This is a drug that will be welcomed by the physician community as the only approved therapeutic option for patients with advanced malignant PEComa."

    In the Phase 2 registrational AMPECT trial the overall response rate as assessed by independent review was 39% (12/31) with 2 patients achieving a Complete Response after prolonged follow up. The median duration of response has not been reached with a median follow-up of 36 months, and a range of 5.6 to 55.5+ months and ongoing. Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to 2 years. As is the case with other therapeutics of the mTOR class, the FYARRO prescribing information includes warnings and precautions related to stomatitis, myelosuppression, infections, hypokalemia, hyperglycemia, interstitial lung disease, hemorrhage, and hypersensitivity reactions. Grade 3 non-hematologic events occurring in more than 10% of patients included stomatitis, rash, fatigue and infections. Grade 3 laboratory abnormalities occurring in more than 10% of patients that worsened from baseline included lymphocytopenia, increased glucose, and decreased potassium. For detailed important safety information, please see below.

    Brendan Delaney, Chief Operating Officer of Aadi, added, "We have built a strong commercial team and devised a thoughtful strategy in preparation for FYARRO's launch. With FYARRO's demonstrated clinical profile we believe it will become a standard of care for advanced malignant PEComa. We look forward to engaging with physicians to educate the market about this new treatment."

    Robert G. Maki, M.D., Ph.D., Clinical Director of the Sarcoma Program, and Professor of Medicine at the University of Pennsylvania, added, "Patients living with locally advanced or metastatic PEComa are in urgent need of new treatment options. The approval of FYARRO is a significant advancement for treating patients with this disease. Treating sarcoma patients in my practice, I have seen the need for a therapy that addresses the specific molecular alterations of advanced malignant PEComa. I am encouraged that FYARRO provided a clinically meaningful benefit in overall response rate, with some patients responding for up to several years. I am pleased to have FYARRO as a new therapeutic option to offer my advanced malignant PEComa patients."

    Aadi will hold a conference call and webcast to discuss today's announcement, Tuesday, November 23, 2021 at 8:30 a.m. ET.

    Investor conference call information:

    A listen-in only webcast of the conference call with corresponding slides can be accessed via Aadi's website, within the Investors & News/Events & Presentations section. A replay of the webcast can also be accessed at this link.

    Dial-in only information:

    Investors, U.S.: 877-407-9716 

    Investors, non-U.S.: 201-493-6779 

    Conference ID: 13725222 

    About Malignant PEComa

    Advanced malignant PEComa, defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,' are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. While there is no formal epidemiology for malignant PEComa, it is estimated that there are about 100-300 new patients per year in the United States. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract with a female predominance) and can have an aggressive clinical course including distant metastases and ultimately death. The estimated prognosis based on retrospective reports is 12-16 months. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.

    About FYARRO™

    FYARRO is an mTOR inhibitor indicated for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

    Important Safety Information

    Contraindication

    FYARRO is contraindicated in patients with a history of severe hypersensitivity to sirolimus, other rapamycin derivatives, or albumin.

    Warnings and Precautions

    Stomatitis

    Stomatitis, including mouth ulcers and oral mucositis, occurred in 79% of patients treated with FYARRO, including 18% Grade 3. Stomatitis was most often first reported within 8 weeks of treatment. Based on the severity of the adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Myelosuppression

    FYARRO can cause myelosuppression including anemia, thrombocytopenia and neutropenia. Anemia occurred in 68% of patients; 6% were Grade 3. Thrombocytopenia and neutropenia occurred in 35% of patients each. Obtain blood counts at baseline and every 2 months for the first year of treatment and every 3 months thereafter, or more frequently if clinically indicated. Based on the severity of the adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Infections

    FYARRO can cause infections. Infections such as urinary tract infections (UTI), upper respiratory tract infections and sinusitis occurred in 59% of patients. Grade 3 infections occurred in 12% of patients, including a single case each of a UTI, pneumonia, skin, and abdominal infections. Monitor patients for infections, including opportunistic infections. Based on the severity of the adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Hypokalemia

    FYARRO can cause hypokalemia. Hypokalemia occurred in 44% of patients including 12% Grade 3 events. Monitor potassium levels prior to starting FYARRO and implement potassium supplementation as medically indicated. Based on the severity of the adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Hyperglycemia

    FYARRO can cause hyperglycemia. Hyperglycemia occurred in 12% of patients treated with FYARRO, all of which were Grade 3 events. Monitor fasting serum glucose prior to starting FYARRO. During treatment, monitor serum glucose every 3 months in non-diabetic patients, or as clinically indicated. Monitor serum glucose more frequently in diabetic patients. Based on the severity of the adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Interstitial Lung Disease / Non-Infectious Pneumonitis

    FYARRO can cause interstitial lung disease (ILD) / non-infectious pneumonitis. ILD / non-infectious pneumonitis occurred in 18% of patients treated with FYARRO, of which all were Grades 1 and 2. Based on the severity of the adverse reaction, withhold, reduce the dose, or permanently discontinue FYARRO.

    Hemorrhage

    FYARRO can cause serious and sometimes fatal hemorrhage. Hemorrhage occurred in 24% of patients treated with FYARRO, including Grade 3 and Grade 5 events in 2.9% of patients each. Monitor patients for signs and symptoms of hemorrhage. Based on the severity of adverse reaction, withhold, resume at reduced dose, or permanently discontinue FYARRO.

    Hypersensitivity Reactions

    FYARRO can cause hypersensitivity reactions. Hypersensitivity reactions, including anaphylaxis, angioedema, exfoliative dermatitis, and hypersensitivity vasculitis have been observed with administration of the oral formulation of sirolimus. Hypersensitivity reactions including anaphylaxis have been observed with human albumin administration. Monitor patients closely for signs and symptoms of infusion reactions during and following each FYARRO infusion in a setting where cardiopulmonary resuscitation medication and equipment are available. Monitor patients for at least 2 hours after the first infusion and as clinically needed for each subsequent infusion. Reduce the rate, interrupt infusion, or permanently discontinue FYARRO based on severity and institute appropriate medical management as needed.

    Embryo-Fetal Toxicity

    Based on animal studies and the mechanism of action, FYARRO can cause fetal harm when administered to a pregnant woman. In animal studies, mTOR inhibitors caused embryo-fetal toxicity when administered during the period of organogenesis at maternal exposures that were equal to or less than human exposures at the recommended lowest starting dose. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to avoid becoming pregnant and to use effective contraception while using FYARRO and for 12 weeks after the last dose.

    Male Infertility

    Azoospermia or oligospermia may be observed in patients treated with FYARRO. FYARRO is an anti-proliferative drug and affects rapidly dividing cells such as germ cells.

    Immunizations and Risks Associated with Live Vaccines

    No studies in conjunction with immunization have been conducted with FYARRO. Immunization during FYARRO treatment may be ineffective. Update immunizations according to immunization guidelines prior to initiating FYARRO, if possible. Immunization with live vaccines is not recommended during treatment and avoid close contact with those who have received live vaccines while on FYARRO. The interval between live vaccinations and initiation of FYARRO should be in accordance with current vaccination guidelines for patients on immunosuppressive therapies.

    Risk of Transmission of Infectious Agents with Human Albumin

    FYARRO contains human albumin, a derivative of human blood. Human albumin carries only a remote risk of transmission of viral diseases because of effective donor screening and product manufacturing processes. A theoretical risk for transmission of Creutzfeldt-Jakob Disease (CJD) also is considered extremely remote. No cases of transmission of viral diseases or CJD have ever been associated with albumin.

    Adverse Reactions

    Adverse Reactions in PEComa

    The most common adverse reactions (≥30%) were stomatitis in 27 (79%) patients; fatigue and rash in 23 (68%) patients each; infection in 20 (59%) patients; nausea and edema in 17 (50%) patients each; diarrhea, musculoskeletal pain and decreased weight in 16 (47%) patients each; decreased appetite in 15 (44%) patients; cough in 12 (35%) patients; and vomiting and dysgeusia in 11 (32%) patients each.

    Laboratory Abnormalities in PEComa

    The most common Grade 3 to 4 laboratory abnormalities (≥6%) were decreased lymphocytes in 7 (21%) patients; increased glucose and decreased potassium in 4 (12%) patients each; decreased phosphate in 3 (9%) patients; and decreased hemoglobin and increased lipase in 2 (6%) patients each.

    Dosage interruptions

    Dose interruptions of FYARRO due to an adverse reaction occurred in 22 (65%) patients. Adverse reactions which required dosage interruption in >5% of patients included stomatitis in 6 (18%) patients, pneumonitis in 5 (15%) patients, anemia in 3 (9%) patients, and dehydration, dermatitis acneiform, and thrombocytopenia in 2 (6%) patients each.

    Dose reduction

    Dose reductions of FYARRO due to an adverse reaction occurred in 12 (35%) patients. Adverse reactions which required dose reductions in >5% of patients included stomatitis and pneumonitis in 3 (9%) patients each.

    Drug Interactions

    Reduce the dosage of FYARRO to 56 mg/m2 when used concomitantly with a moderate or weak cytochrome P-450 3A4 (CYP3A4) inhibitor. Avoid concomitant use with drugs that are strong CYP3A4 and/or P-glycoprotein (P-gp) inhibitors and inducers and with grapefruit and grapefruit juice.

    Use in Specific Populations

    Pregnancy

    Based on the mechanism of action and findings in animals, FYARRO can cause fetal harm when administered to a pregnant woman. Advise females of the potential risk to a fetus and to avoid becoming pregnant while receiving FYARRO.

    Lactation

    Sirolimus is present in the milk of lactating rats. There is potential for serious adverse effects from sirolimus in breastfed infants based on mechanism of action. Because of the potential for serious adverse reactions in breastfed infants from FYARRO, advise women not to breastfeed during treatment with FYARRO and for 2 weeks after the last dose.

    Females and Males of Reproductive Potential

    FYARRO can cause fetal harm when administered to a pregnant woman. Verify the pregnancy status of females of reproductive potential prior to starting treatment with FYARRO. Advise females of reproductive potential to use effective contraception and avoid becoming pregnant during treatment with and for at least twelve weeks after the last dose of FYARRO. Advise males with female partners of reproductive potential to use effective contraception and avoid fathering a child during treatment with FYARRO and for at least twelve weeks after the last dose of FYARRO. Although there are no data on the impact of FYARRO on fertility, based on available clinical findings with oral formulation of sirolimus and findings in animals, male and female fertility may be compromised by the treatment with FYARRO.

    Pediatric

    The safety and effectiveness of FYARRO in pediatric patients have not been established.

    Geriatric Use

    Of the 34 patients treated with FYARRO, 44% were 65 years of age and older, and 6% were 75 years of age and older. Clinical studies of FYARRO did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients.

    Hepatic Impairment

    FYARRO is not recommended for use in patients with severe hepatic impairment. Reduce FYARRO dosage in patients with mild or moderate hepatic impairment.

    Full prescribing information can be found here.

    About Aadi Bioscience

    Aadi is a commercial-stage biopharmaceutical company focused on precision therapies for genetically-defined cancers. Aadi's primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations where other mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site. In November 2021, Aadi received FDA approval for FYARRO for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is an mTOR inhibitor bound to human albumin that has demonstrated significantly higher tumor accumulation, greater mTOR target suppression, and increased tumor growth inhibition over other mTOR inhibitors in preclinical models.

    Based on data from the AMPECT trial with FYARRO and following discussions with the FDA about other emerging data with FYARRO, Aadi plans to initiate a tumor-agnostic registrational trial in mTOR inhibitor-naïve solid tumors harboring TSC1 or TSC2 inactivating alterations by the end of 2021 or early 2022. Aadi also has ongoing studies to evaluate dosing of FYARRO in combination regimens. More information on Aadi's development pipeline is available on the Aadi website at www.aadibio.com.

    Forward-Looking Statements

    Aadi cautions you that certain statements included in this press release that 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: our plans and potential for success relating to commercializing FYARRO, the expectations regarding the beneficial characteristics, safety, efficacy and therapeutic effects of FYARRO, our plans related to further development and manufacturing of FYARRO, the timing of additional clinical trials, including the registrational trial in patients harboring TSC1 and TSC2 inactivating alterations whose initiation is expected by the end of 2021 or early 2022, the timing or likelihood of regulatory filings and approvals of FYARRO, including in potential additional indications for FYARRO and potential filings in additional jurisdictions, anticipated reception of FYARRO in the physician community, and the sufficiency of our existing capital resources to fund our future operating expenses and capital expenditure requirements. Actual results could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: risks related to Aadi's ability to successfully commercialize, including the timing of a commercial launch of FYARRO in advanced malignant PEComa; uncertainties associated with the clinical development and regulatory approval of FYARRO, including potential delays in the commencement, enrollment and completion of clinical trials for additional indications; the risk that interim results of clinical trials may not be reproduced and do not necessarily predict final results; the risk that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of commercializing, developing and testing FYARRO; risks associated with the failure to realize any value from FYARRO in light of inherent risks and difficulties involved in successfully bringing product candidates to market; risks related to Aadi's estimates regarding future expenses, capital requirements and need for additional financing; and risks related to the impact of the COVID-19 outbreak on Aadi's operations, the biotechnology industry and the economy generally.

    Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption "Risk Factors" and elsewhere in Aadi's reports and other documents that Aadi has filed, or will file, with the SEC from time to time and available at www.sec.gov.

    All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Aadi undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, 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 caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

    FYARRO™ is a trademark of Aadi Bioscience, Inc.

    Contacts

    Investors:

    Irina Koffler

    LifeSci Advisors LLC

    Media:

    Darren Opland, Ph.D.

    LifeSci Communications



    Primary Logo

    View Full Article Hide Full Article
    • In an Expanded Access Program, advanced malignant PEComa patients who previously progressed on other mTOR inhibitors showed a 25% partial response rate and 63% clinical benefit rate when treated with nab-sirolimus; the subset of patients with TSC1 or TSC2 alterations showed a 44% response rate
    • In a final analysis from the AMPECT registrational trial in mTOR naïve patients with advanced malignant PEComa, results of a 2.5 year-follow-up showed a median duration of response exceeding 36 months and conversion of 2 patients to complete responses
    • FYARRO™ has a November 26, 2021 PDUFA target date

    LOS ANGELES, Nov. 15, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision…

    • In an Expanded Access Program, advanced malignant PEComa patients who previously progressed on other mTOR inhibitors showed a 25% partial response rate and 63% clinical benefit rate when treated with nab-sirolimus; the subset of patients with TSC1 or TSC2 alterations showed a 44% response rate
    • In a final analysis from the AMPECT registrational trial in mTOR naïve patients with advanced malignant PEComa, results of a 2.5 year-follow-up showed a median duration of response exceeding 36 months and conversion of 2 patients to complete responses
    • FYARRO™ has a November 26, 2021 PDUFA target date

    LOS ANGELES, Nov. 15, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced two oral presentations that were made related to its lead candidate, FYARRO™ (ABI-009 or nab-sirolimus) at the Connective Tissue Oncology Society (CTOS) 2021 Annual Meeting, held virtually from November 10-13, 2021. CTOS is a multi-disciplinary group of specialized physicians, medical professionals and scientists from around the world who connect and share their knowledge, experiences and research for the advancement of treatment of sarcomas. Both studies, sponsored by Aadi, provided data in patients with advanced malignant PEComa, which is the clinical indication currently under review by the Food and Drug Administration (FDA) with a November 26, 2021 target Prescription Drug User Fee Act (PDUFA) date.

    Abstract (ID: 1080984), lead-authored by Mark A. Dickson, M.D., medical oncologist at Memorial Sloan Kettering Cancer Center is entitled, "nab-Sirolimus in Patients with Malignant PEComa Previously Treated With mTOR Inhibitors: Emerging Experience from an Expanded Access Program". Sixteen patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa) were treated in an Expanded Access Program (NCT03817515) with nab-sirolimus, dosed intravenously at 100 mg/m2 given on day one and day eight of a 21-day cycle. The investigators concluded that nab-sirolimus showed encouraging clinical benefit including partial responses (PR) in 25% of patients previously progressing on other mTOR inhibitors and, in some cases, other targeted therapies. Disease control rate (DCR) as defined by complete or partial response + stable disease for ≥3 months, was 63%. In this study, the safety profile of nab-sirolimus was acceptable and allowed ongoing treatment for almost one year or more in several patients. In addition, consistent with results of the AMPECT trial, TSC1 or TSC2 alterations were associated with a higher response rate of 44% of patients, despite prior progression on other mTOR inhibitors and/or multiple lines of prior therapy. These results provide further rationale for investigation of nab-sirolimus in a tumor-agnostic study in patients with pathogenic inactivating TSC1 or TSC2 alterations.

    A second abstract (ID: 1080747), lead-authored by Andrew J. Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute, is entitled, "Final Analysis from AMPECT, an Open-Label Phase 2 Registration Trial of nab-Sirolimus for Patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)". This abstract provided a final analysis of the AMPECT study with a data cut of June 30, 2021, updating the recent publication in the Journal of Clinical Oncology which was based on a data cut of November 23, 2020. nab-Sirolimus demonstrated rapid and durable responses in mTOR-naïve patients with locally advanced unresectable or metastatic PEComa. Specifically, of 31 treated and evaluable patients, the independently assessed confirmed overall response rate (ORR) was 39% (12/31, 95% confidence interval: 22, 58); of which 7% (2/31) of patients had a complete response (CR) and 32% (10/31) had a PR. Disease control was achieved in 71% of patients. The patient responses demonstrated long-term durability with a duration of response (DOR) of 92% at 6 months and 66% at 36 months amongst the 12 patients with a response. At the final analysis, the median DOR has not been reached, 50% of patients had a DOR of over 36 months (range 5.6, 55.5+ months). By the final analysis, two patients converted from a PR to CR after 11 months and 34 months of treatment, respectively. Finally, nab-sirolimus demonstrated an acceptable safety profile with no grade 4 or 5 treatment-related adverse events (TRAE) and no unexpected adverse events or new safety signals. Some of the most common TRAEs were stomatitis, rash, fatigue, anemia, nausea, diarrhea and hyperglycemia.

    Dr. Dickson commented, "I am encouraged by the activity of nab-sirolimus not only in the AMPECT study, but in advanced PEComa patients previously progressing on other mTOR inhibitors, as well as in other solid tumor histologies with TSC1 or TSC2 inactivating alterations that has been previously presented. There is a strong rationale for conducting a broader investigation of nab-sirolimus in a tumor-agnostic setting."

    About Malignant PEComa

    Perivascular epithelioid-cell tumors (PEComa), defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,' are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. While there is no formal epidemiology for malignant PEComa, it is estimated that there are about 100-300 new patients per year in the United States. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract with a female predominance) and can have an aggressive clinical course including distant metastases and ultimate death. The estimated survival based on retrospective reports is 12-16 months. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.

    About Aadi Bioscience and FYARRO™

    Aadi is a clinical-stage biopharmaceutical company developing precision therapies for genetically-defined cancers. Aadi's primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations such as alterations in TSC1 or TSC2 genes, where other mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site. Aadi's lead product candidate is FYARRO™ (sirolimus albumin-bound nanoparticles for injectable suspension; nab-sirolimus; ABI-009), an mTOR inhibitor bound to human albumin that has demonstrated significantly higher tumor accumulation, greater mTOR target suppression, and increased tumor growth inhibition over other mTOR inhibitors in preclinical models.

    Aadi's registration trial of FYARRO in advanced malignant PEComa (the "AMPECT trial") demonstrated meaningful clinical efficacy in malignant PEComa, a type of cancer with the highest known alteration rate of TSC1 or TSC2 genes. FYARRO has received Breakthrough Therapy, Fast-Track and Orphan Designations from the U.S. Food and Drug Administration (FDA). A rolling New Drug Application (NDA) submission 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 TSC1 or TSC2 inactivating alterations, and following discussions with the FDA, Aadi plans to initiate a tumor-agnostic registrational trial in mTOR inhibitor-naïve solid tumors harboring TSC1 or TSC2 inactivating alterations by the end of 2021 or early 2022. Aadi also has ongoing studies to evaluate dosing of FYARRO in combination regimens. FYARRO is an investigational drug that has not been approved by the FDA for commercial distribution in the United States. More information is available on the Aadi website at  www.aadibio.com.

    Forward-Looking Statements

    Aadi Bioscience, Inc. ("Aadi", "The Company") cautions you that certain statements included in this press release that 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 the clinical responses and safety profile, regulatory approval and commercialization, and the timing of additional clinical trials, including the registrational trial in patients harboring TSC1 and TSC2 inactivating alterations that is expected to begin by the end of 2021 or in early 2022; and the expectation that Aadi's cash and cash equivalents will fund operations into 2024. Actual results could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: risks related to Aadi's ability to obtain, or the timeline to obtain, regulatory approval from the FDA and other regulatory authorities for FYARRO in advanced malignant PEComa; risks related to Aadi's ability to successfully commercialize, including the timing of a commercial launch of FYARRO in advanced malignant PEComa; uncertainties associated with the clinical development and regulatory approval of FYARRO, including potential delays in the commencement, enrollment and completion of clinical trials; the risk that interim results of clinical trials may not be reproduced and do not necessarily predict final results; the risk that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of developing and testing FYARRO; risks associated with the failure to realize any value from FYARRO in light of inherent risks and difficulties involved in successfully bringing product candidates to market; risks related to Aadi's estimates regarding future expenses, capital requirements and need for additional financing; and risks related to the impact of the COVID-19 outbreak on Aadi's operations, the biotechnology industry and the economy generally.

    Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption "Risk Factors" and elsewhere in Aadi's reports and other documents that Aadi has filed, or will file, with the SEC from time to time and available at www.sec.gov.

    All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Aadi undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, 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 caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

    FYARRO™ is a trademark of Aadi Bioscience, Inc.

    Contacts

    Investors:

    Irina Koffler

    LifeSci Advisors LLC



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    • In an Expanded Access Program, advanced malignant PEComa patients who previously progressed on other mTOR inhibitors showed a 25% partial response rate and 63% clinical benefit rate when treated with nab-sirolimus
    • In a final analysis from the AMPECT registrational trial in mTOR naïve patients with advanced malignant PEComa, results of a 2.5 year-follow-up showed a median duration of response exceeding 36 months and conversion of 2 patients to complete responses
    • FYARRO™ has a November 26, 2021 PDUFA target date

    LOS ANGELES, Nov. 13, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway…

    • In an Expanded Access Program, advanced malignant PEComa patients who previously progressed on other mTOR inhibitors showed a 25% partial response rate and 63% clinical benefit rate when treated with nab-sirolimus
    • In a final analysis from the AMPECT registrational trial in mTOR naïve patients with advanced malignant PEComa, results of a 2.5 year-follow-up showed a median duration of response exceeding 36 months and conversion of 2 patients to complete responses
    • FYARRO™ has a November 26, 2021 PDUFA target date

    LOS ANGELES, Nov. 13, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced two oral presentations that were made related to its lead candidate, FYARRO™ (ABI-009 or nab-sirolimus) at the Connective Tissue Oncology Society (CTOS) 2021 Annual Meeting, held virtually from November 10-13, 2021. CTOS is a multi-disciplinary group of specialized physicians, medical professionals and scientists from around the world who connect and share their knowledge, experiences and research for the advancement of treatment of sarcomas. Both studies, sponsored by Aadi, provided data in patients with advanced malignant PEComa, which is the clinical indication currently under review by the Food and Drug Administration (FDA) with a November 26, 2021 target Prescription Drug User Fee Act (PDUFA) date.

    Abstract (ID: 1080984), lead-authored by Mark A. Dickson, M.D., medical oncologist at Memorial Sloan Kettering Cancer Center is entitled, "nab-Sirolimus in Patients with Malignant PEComa Previously Treated With mTOR Inhibitors: Emerging Experience from an Expanded Access Program". Sixteen patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa) were treated in an Expanded Access Program (NCT03817515) with nab-sirolimus, dosed intravenously at 100 mg/m2 given on day one and day eight of a 21-day cycle. The investigators concluded that nab-sirolimus showed encouraging clinical benefit including partial responses (PR) in 25% of patients previously progressing on other mTOR inhibitors and, in some cases, other targeted therapies. Disease control rate (DCR) as defined by complete or partial response + stable disease for ≥3 months, was 63%. In this study, the safety profile of nab-sirolimus was acceptable and allowed ongoing treatment for almost one year or more in several patients. In addition, consistent with results of the AMPECT trial, TSC1 or TSC2 alterations were associated with a higher response rate of 44% of patients, despite prior progression on other mTOR inhibitors and/or multiple lines of prior therapy. These results provide further rationale for investigation of nab-sirolimus in a tumor-agnostic study in patients with pathogenic inactivating TSC1 or TSC2 alterations.

    A second abstract (ID: 1080747), lead-authored by Andrew J. Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute, is entitled, "Final Analysis from AMPECT, an Open-Label Phase 2 Registration Trial of nab-Sirolimus for Patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)". This abstract provided a final analysis of the AMPECT study with a data cut of June 30, 2021, updating the recent publication in the Journal of Clinical Oncology which was based on a data cut of November 23, 2020. nab-Sirolimus demonstrated rapid and durable responses in mTOR-naïve patients with locally advanced unresectable or metastatic PEComa. Specifically, of 31 treated and evaluable patients, the independently assessed confirmed overall response rate (ORR) was 39% (12/31, 95% confidence interval: 22, 58); of which 7% (2/31) of patients had a complete response (CR) and 32% (10/31) had a PR. Disease control was achieved in 71% of patients. The patient responses demonstrated long-term durability with a duration of response (DOR) of 92% at 6 months and 66% at 36 months amongst the 12 patients with a response. At the final analysis, the median DOR has not been reached, 50% of patients had a DOR of over 36 months (range 5.6, 55.5+ months). By the final analysis, two patients converted from a PR to CR after 11 months and 34 months of treatment, respectively. Finally, nab-sirolimus demonstrated an acceptable safety profile with no grade 4 or 5 treatment-related adverse events (TRAE) and no unexpected adverse events or new safety signals. Some of the most common TRAEs were stomatitis, rash, fatigue, anemia, nausea, diarrhea and hyperglycemia.

    Dr. Dickson commented, "I am encouraged by the activity of nab-sirolimus not only in the AMPECT study, but in advanced PEComa patients previously progressing on other mTOR inhibitors, as well as in other solid tumor histologies with TSC1 or TSC2 inactivating alterations that has been previously presented. There is a strong rationale for conducting a broader investigation of nab-sirolimus in a tumor-agnostic setting."

    About Malignant PEComa

    Perivascular epithelioid-cell tumors (PEComa), defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,' are a rare subset of soft-tissue sarcomas, with an undefined cell of origin.  While there is no formal epidemiology for malignant PEComa, it is estimated that there are about 100-300 new patients per year in the United States. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract with a female predominance) and can have an aggressive clinical course including distant metastases and ultimate death.  The estimated survival based on retrospective reports is 12-16 months. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease.  Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.  

    About Aadi Bioscience and FYARRO™

    Aadi is a clinical-stage biopharmaceutical company developing precision therapies for genetically-defined cancers. Aadi's primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations such as alterations in TSC1 or TSC2 genes, where other mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site. Aadi's lead product candidate is FYARROTM (sirolimus albumin-bound nanoparticles for injectable suspension; nab-sirolimus; ABI-009), an mTOR inhibitor bound to human albumin that has demonstrated significantly higher tumor accumulation, greater mTOR target suppression, and increased tumor growth inhibition over other mTOR inhibitors in preclinical models.

    Aadi's registration trial of FYARRO in advanced malignant PEComa (the "AMPECT trial") demonstrated meaningful clinical efficacy in malignant PEComa, a type of cancer with the highest known alteration rate of TSC1 or TSC2 genes. FYARRO has received Breakthrough Therapy, Fast-Track and Orphan Designations from the U.S. Food and Drug Administration (FDA). A rolling New Drug Application (NDA) submission 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 TSC1 or TSC2 inactivating alterations, and following discussions with the FDA, Aadi plans to initiate a tumor-agnostic registrational trial in mTOR inhibitor-naïve solid tumors harboring TSC1 or TSC2 inactivating alterations by the end of 2021 or early 2022. Aadi also has ongoing studies to evaluate dosing of FYARRO in combination regimens. FYARRO is an investigational drug that has not been approved by the FDA for commercial distribution in the United States. More information is available on the Aadi website at www.aadibio.com.

    Forward-Looking Statements

    Aadi Bioscience, Inc. ("Aadi", "The Company") cautions you that certain statements included in this press release that 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 the clinical responses and safety profile, regulatory approval and commercialization, and the timing of additional clinical trials, including the registrational trial in patients harboring TSC1 and TSC2 inactivating alterations that is expected to begin by the end of 2021 or in early 2022; and the expectation that Aadi's cash and cash equivalents will fund operations into 2024. Actual results could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: risks related to Aadi's ability to obtain, or the timeline to obtain, regulatory approval from the FDA and other regulatory authorities for FYARRO in advanced malignant PEComa; risks related to Aadi's ability to successfully commercialize, including the timing of a commercial launch of FYARRO in advanced malignant PEComa; uncertainties associated with the clinical development and regulatory approval of FYARRO, including potential delays in the commencement, enrollment and completion of clinical trials; the risk that interim results of clinical trials may not be reproduced and do not necessarily predict final results; the risk that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of developing and testing FYARRO; risks associated with the failure to realize any value from FYARRO in light of inherent risks and difficulties involved in successfully bringing product candidates to market; risks related to Aadi's estimates regarding future expenses, capital requirements and need for additional financing; and risks related to the impact of the COVID-19 outbreak on Aadi's operations, the biotechnology industry and the economy generally.

    Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption "Risk Factors" and elsewhere in Aadi's reports and other documents that Aadi has filed, or will file, with the SEC from time to time and available at www.sec.gov.

    All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Aadi undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, 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 caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

    FYARRO™ is a trademark of Aadi Bioscience, Inc.

    Contacts

    Investors:

    Irina Koffler

    LifeSci Advisors LLC



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    • FYARRO™ under review with FDA with a November 26, 2021 PDUFA target date
    • Three key executive appointments made to the roles of Chief Operating Officer, Chief Medical Officer and Chief Financial Officer
    • Appointment of new board member
    • Registrational trial in patients harboring TSC1 and TSC2 inactivating alterations expected to be initiated by the end of 2021 or early 2022
    • Ended third quarter 2021 with $161.4 million in cash and cash equivalents with expected cash runway into 2024 following merger with Aerpio Pharmaceuticals, Inc. and concurrent PIPE financing

    LOS ANGELES, Nov. 10, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined…

    • FYARRO™ under review with FDA with a November 26, 2021 PDUFA target date
    • Three key executive appointments made to the roles of Chief Operating Officer, Chief Medical Officer and Chief Financial Officer
    • Appointment of new board member
    • Registrational trial in patients harboring TSC1 and TSC2 inactivating alterations expected to be initiated by the end of 2021 or early 2022
    • Ended third quarter 2021 with $161.4 million in cash and cash equivalents with expected cash runway into 2024 following merger with Aerpio Pharmaceuticals, Inc. and concurrent PIPE financing

    LOS ANGELES, Nov. 10, 2021 (GLOBE NEWSWIRE) -- Aadi Bioscience, Inc. ("Aadi") (NASDAQ:AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today reported financial results for the three and nine months ended September 30, 2021 and provided a general business update.

    Neil Desai, Ph.D., Founder, Chief Executive Officer and President of Aadi, commented, "Following the successful merger completed in the third quarter, we are now fortified on all fronts with a strengthened balance sheet and expanded management team ahead of our November 26 Prescription Drug User Fee Action ("PDUFA") target date for FYARRO. With a sizeable PIPE financing that closed in connection with our merger with Aerpio, we can effectively deploy capital toward the commercialization of FYARRO in advanced malignant PEComa and for our planned tumor-agnostic registrational trial in patients with solid tumors harboring inactivating alterations of TSC1 and TSC2 genes in the mTOR pathway. In addition, we are also planning for new studies to investigate ABI-009 in combination with other targeted agents in different cancers."

    Key Business Updates for the Third Quarter:

    Merger and PIPE Financing

    • On August 26, 2021, Aadi merged with Aerpio Pharmaceuticals, Inc., a publicly traded biotechnology company (previously traded on the Nasdaq Capital Market under "ARPO"), with Aadi being the surviving entity. As part of the merger, each share of Aadi common stock was converted into the right to receive 0.3172 shares of Aerpio common stock following a 15:1 reverse split of Aerpio's common stock. Aadi's common stock is traded on the Nasdaq Capital Market (Nasdaq) under the ticker symbol "AADI".
      • Concurrent to the closing of the merger, the combined company closed the previously announced $155 million private investment in public equity (PIPE) financing of its common stock.



      • Immediately following the merger, PIPE financing, and reverse split, Aadi had approximately 20.8 million shares of common stock outstanding, with prior Aadi stockholders collectively owning approximately 29.2% of the combined company, prior Aerpio stockholders owning approximately 15.2% of the combined company, and the PIPE investors collectively owning approximately 55.6% of the combined company, each on a fully-diluted basis.

    NDA Acceptance

    • In July, the U.S. Food and Drug Administration (FDA) accepted Aadi's New Drug Application (NDA) for its nanoparticle albumin-bound mTOR inhibitor, FYARRO™ (sirolimus albumin-bound nanoparticles for injectable suspension, nab-sirolimus ABI-009) for the treatment of advanced PEComa, and has granted the company Priority Review status with a PDUFA target action date of November 26, 2021.

    Key Leadership Appointments

    • In September, Aadi announced key appointments of an additional independent board member as well as a Chief Operating Officer:
      • Emma Reeve was appointed to Aadi's Board of Directors and as chair of the Audit Committee. Ms. Reeve brings over 25 years of value creation in pharmaceutical, medical device and bio-pharma service companies and a successful track record of transitioning companies from private to public.



      • Brendan Delaney was appointed to the role of Chief Operating Officer. Brendan has had an established career in oncology-focused commercial leadership roles, launching multiple groundbreaking new products and building effective and cohesive commercial teams.

    Recent Updates Following the Close of the Quarter:

    • In the fourth quarter, the Company made two more key executive appointments:



      • Loretta M. Itri, M.D., FACP® was appointed to the role of Chief Medical Officer. Dr. Itri's extensive career spans clinical and regulatory global-leadership roles at both major pharmaceutical and biopharmaceutical companies. Most recently, Dr. Itri was Chief Medical Officer at Immunomedics, Inc., where she oversaw the development program and approval of TRODELVY®, the first TROP-2 directed antibody-drug conjugate for the treatment of unresectable locally advanced or metastatic triple-negative breast and urothelial cancers. Immunomedics was subsequently acquired by Gilead Sciences, Inc.

         
      • Scott M. Giacobello, CPA, was appointed to the role of Chief Financial Officer and Treasurer, effective November 28, 2021. Most recently, Mr. Giacobello was the Chief Financial Officer of GW Pharmaceuticals plc until its $7.2 billion acquisition by Jazz Pharmaceuticals. Mr. Giacobello joined GW Pharmaceuticals in 2017 and played a key role in the buildout of the U.S. operations and commercial readiness for the company. While Chief Financial Officer, Mr. Giacobello was instrumental in devising the financing strategy to support the development, approval and highly successful launch of GW's lead product, EPIDIOLEX®, which had sales of over $296 million in its first full year of commercialization. Mr. Giacobello also raised over $620 million via follow-on offerings prior to the company's acquisition by Jazz Pharmaceuticals.
    • Also in October, the Company presented at a medical conference and announced the publication of its registrational study of FYARRO (ABI-009) in a major medical journal:



      • Lead researchers at Aadi presented a poster at the Virtual International Conference on Molecular Targets and Cancer Therapeutics held from October 7th -10th. The poster consisted of preclinical data evaluating nab-sirolimus (ABI-009) in PTEN-deleted and TSC2-deleted cancer models and demonstrated that ABI-009 showed significantly higher tumor accumulation, increased inhibition of downstream mTOR targets S6 and 4EBP1 and greater tumor growth suppression in comparison with other mTOR inhibitors sirolimus and everolimus at equal dose.



      • The Company also announced the publication of "nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors" in the American Society of Clinical Oncology's Journal of Clinical Oncology based on the results of the AMPECT registrational trial. The authors concluded that investigational nab-sirolimus (ABI-009), if approved, may represent an important new treatment option in malignant PEComa, a rare cancer and aggressive form of sarcoma, with no currently approved treatment.

    Third Quarter 2021 Financial Highlights

    As of September 30, 2021, cash and cash equivalents totaled $161.4 million, an increase from $4.5 million as of December 31, 2020, resulting primarily from the consolidated capital and proceeds received from the PIPE financing. Based on our current plans, we expect cash and cash equivalents to fund operations into 2024.

    For the three months ended September 30, 2021, operating expenses totaled $87.3 million, an increase of $84.4 million compared to $2.9 million for the same period in 2020. For the nine months ended September 30, 2021, operating expenses totaled $95.4 million, an increase of $84.0 million compared to $11.4 million for the same period in 2020. The increase in operating expenses for the three- and nine-month periods ended September 30, 2021, is due primarily to a non-cash impairment charge related to an acquired contract intangible asset of $74.2 million incurred in conjunction with the merger compared to the same period in 2020.

    Research and development expenses for the three months ended September 30, 2021, increased approximately $3.4 million, to $5.8 million compared to $2.4 million for the same period in 2020. Research and development expenses for the nine months ended September 30, 2021, increased approximately $2.7 million, to $12.4 million compared to $9.7 million for the same period in 2020. This increase was primarily the result of increased expenses associated with our clinical drug manufacturing process in the three and nine-month periods ended September 30, 2021, compared to the same periods in 2020.  

    General and administrative expenses for the three months ended September 30, 2021, increased approximately $6.9 million to $7.4 million compared to $0.5 million for the same period in 2020. General and administrative expenses for the nine months ended September 30, 2021, increased approximately $7.1 million, to $8.8 million from $1.7 million, in the nine months ended September 30, 2020. This increase was primarily the result of increased personnel expenses, including approximately $2.0 million of compensation expense related to former Aerpio executives as a result of the merger.

    Net loss attributable to common stockholders for the three and nine months ended September 30, 2021, was $87.2 million and $94.7 million, respectively, primarily driven by the non-cash impairment charge of $74.2 million discussed above.

    About Aadi Bioscience and FYARRO™

    Aadi is a clinical-stage biopharmaceutical company developing precision therapies for genetically-defined cancers. Aadi's primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations such as alterations in TSC1 or TSC2 genes, where other mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site. Aadi's lead product candidate is FYARROTM (sirolimus albumin-bound nanoparticles for injectable suspension; nab-sirolimus; ABI-009), an mTOR inhibitor bound to human albumin that has demonstrated significantly higher tumor accumulation, greater mTOR target suppression, and increased tumor growth inhibition over other mTOR inhibitors in preclinical models.

    Aadi's registration trial of FYARRO in advanced malignant PEComa (the "AMPECT trial") demonstrated meaningful clinical efficacy in malignant PEComa, a type of cancer with the highest known alteration rate of TSC1 or TSC2 genes. FYARRO has received Breakthrough Therapy, Fast-Track and Orphan Designations from the U.S. Food and Drug Administration (FDA). A rolling New Drug Application (NDA) submission 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 TSC1 or TSC2 inactivating alterations, and following discussions with the FDA, Aadi plans to initiate a tumor-agnostic registrational trial in mTOR inhibitor-naïve solid tumors harboring TSC1 or TSC2 inactivating alterations by the end of 2021 or early 2022. Aadi also has ongoing studies to evaluate dosing of FYARRO in combination regimens. FYARRO is an investigational drug that has not been approved by the FDA for commercial distribution in the United States. More information is available on the Aadi website at www.aadibio.com.

    Forward-Looking Statements

    Aadi Bioscience, Inc. ("Aadi", "The Company") cautions you that certain statements included in this press release that 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 the clinical responses and safety profile, regulatory approval and commercialization, and the timing of additional clinical trials, including the registrational trial in patients harboring TSC1 and TSC2 inactivating alterations whose initiation is expected by the end of 2021 or early 2022; and the expectation that Aadi's cash and cash equivalents will last into 2024. Actual results could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: risks related to Aadi's ability to obtain, or the timeline to obtain, regulatory approval from the FDA and other regulatory authorities for FYARRO in advanced malignant PEComa; risks related to Aadi's ability to successfully commercialize, including the timing of a commercial launch of FYARRO in advanced malignant PEComa; uncertainties associated with the clinical development and regulatory approval of FYARRO, including potential delays in the commencement, enrollment and completion of clinical trials; the risk that interim results of clinical trials may not be reproduced and do not necessarily predict final results; the risk that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of developing and testing FYARRO; risks associated with the failure to realize any value from FYARRO in light of inherent risks and difficulties involved in successfully bringing product candidates to market; risks related to Aadi's estimates regarding future expenses, capital requirements and need for additional financing; and risks related to the impact of the COVID-19 outbreak on Aadi's operations, the biotechnology industry and the economy generally.

    Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption "Risk Factors" and elsewhere in Aadi's reports and other documents that Aadi has filed, or will file, with the SEC from time to time and available at www.sec.gov.

    All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Aadi undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, 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 caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

    FYARRO™ is a trademark of Aadi Bioscience, Inc.

    Contacts

    Investors:

    Irina Koffler

    LifeSci Advisors LLC





        
    AADI BIOSCIENCE, INC.
    CONDENSED CONSOLIDATED BALANCE SHEETS
    (In thousands)
        
     September 30, December 31,
      2021   2020 
    Assets   
    Current assets:   
    Cash and cash equivalents$161,375  $4,455 
    Accounts receivable -   14,149 
    Prepaid expenses and other current assets 643   81 
    Total current assets 162,018   18,685 
    Property and equipment, net 14   21 
    Operating lease right-of-use assets 597   119 
    Intangible asset, net 3,880   - 
    Other assets 2,263   - 
    Total assets$168,772  $18,825 
        
    Liabilities and shareholders' equity (deficit)   
    Current liabilities:   
    Accounts payable$5,205  $2,392 
    Accrued liabilities 6,250   4,099 
    Payable to related party 22   14,314 
    Convertible related party promissory notes payable at fair value -   9,029 
    Operating lease liabilities, current portion 90   125 
    Other current liabilities -   99 
    Total current liabilities 11,567   30,058 
    Convertible promissory notes payable at fair value -   1,102 
    Payable to related party 5,757   - 
    Operating lease liabilities, net of current portion 523   - 
    Other liabilities -   97 
    Total liabilities 17,847   31,257 
        
    Stockholders' equity (deficit):   
    Series A preferred stock -   1 
    Common stock 2   1 
    Additional paid-in capital 277,618   20,161 
    Accumulated deficit (126,695)  (32,595)
    Total stockholders' equity (deficit) 150,925   (12,432)
    Total liabilities and stockholders' equity (deficit)$168,772  $18,825 
        





    AADI BIOSCIENCE, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
    (In thousands, except shares and earnings per share amounts)
            
     Three months ended Nine Months Ended
     September 30, September 30,
      2021   2020   2021   2020 
    Grant revenue$-  $231  $120  $431 
            
    Operating expenses:       
    Research and development 5,754   2,395   12,443   9,684 
    General and administrative 7,401   499   8,793   1,700 
    Impairment of intangible asset 74,156   -   74,156   - 
    Total operating expenses 87,311   2,894   95,392   11,384 
    Loss from operations (87,311)  (2,663)  (95,272)  (10,953)
    Other income (expense)       
    Change in fair value of convertible promissory note 380   -   1,585   - 
    Gain upon extinguishment of debt -   -   196   - 
    Interest income -   1   1   41 
    Interest expense (157)  (229)  (608)  (585)
    Total other income (expense), net 223   (228)  1,174   (544)
    Loss before income taxes (87,088)  (2,891)  (94,098)  (11,497)
    Income tax expense -   (1)  (2)  (1)
    Net and comprehensive loss (87,088)  (2,892)  (94,100)  (11,498)
    Cumulative dividends on convertible preferred stock (154)  (247)  (647)  (740)
    Net and comprehensive loss attributable to common stockholders$(87,242) $(3,139) $(94,747) $(12,238)
    Net and comprehensive loss per share attributable to common stockholders, basic and diluted

    $(9.17) $(1.23) $(19.37) $(4.81)
            
    Weighted average number of common shares outstanding used in computing net and comprehensive loss per share attributable to common stockholders, basic and diluted 9,510,379   2,542,358   4,890,556   2,542,358 
            



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