Tuberous Sclerosis 2

Subgroup experienced efficacy and safety consistent with overall study population Advanced malignant PEComa tumors of gynecologic origin accounted for more than half of the evaluable patients enrolled in AMPECT Additional data presented highlight nab-sirolimus as potential approach for mTOR-driven gynecologic cancers LOS ANGELES, March 17, 2024 /PRNewswire/ -- Aadi Bioscience, Inc. (NASDAQ: AADI), a commercial-stage precision oncology company focused on developing and commercializing therapies for cancers with alterations in the mTOR pathway, today announced patients in the AMPECT trial whose malignant perivascular epithelioid cell tumor (PEComa) had gynecologic origins experienced efficacy and safety consistent with the overall study population. The AMPECT trial formed the basis for the FDA approval of the company's nab-sirolimus, FYARRO®, for advanced malignant PEComa regardless of mutational status. This new subgroup analysis will be presented during an oral plenary at the Society of Gynecologic Oncology (SGO) Annual Meeting in San Diego, CA on March 17, 2024. "In AMPECT, advanced malignant PEComa tumors originating from uterine, ovarian, pelvic or retroperitoneal sites had a response to nab-sirolimus consistent with that of the full study population, including overall response rate, onset and duration of tumor response," said Thomas J. Herzog, MD, Deputy Director, University of Cincinnati Cancer Center. "Malignant PEComa tumors of gynecologic or retroperitoneal origin accounted for more than half of the evaluable patients enrolled, offering important insights into this population and reinforcing the need for awareness and understanding of this rare but aggressive cancer among the gynecologic oncologist community." Oral plenary presentation details and study highlights include: Title: "Response to Treatment with nab-Sirolimus in Patients with Perivascular Epithelioid Cell Sarcoma (PEComa) of Gynecologic or Retroperitoneal Origin: Subgroup Analysis from AMPECT" Presenting Author: Thomas J. Herzog, MD Session Title: Focused Plenary V: Rare Care: Updates in Uncommon Cancers Location: Ballroom 20CD Date/Time: Sunday, March 17, 2024 – 1:45 PM to 2:45 PM Of the 31 patients enrolled in AMPECT, 16 had malignant PEComas originating from uterine, ovarian, pelvic or retroperitoneal sites Overall response rate to nab-sirolimus for the subgroup was 37.5% (6/16), consistent with the overall AMPECT population Subgroup responses were rapid and durable, with 1.4 months median time to response and 36.2 months median duration of response Safety profile of the subgroup was manageable and consistent with the overall AMPECT population Additional data presented at SGO further highlight nab-sirolimus as a potential approach for mTOR-driven gynecologic cancers. These presentations include a real-world study characterizing TSC1 and TSC2 inactivating alterations in patients with advanced gynecologic cancers; trial-in-progress updates for the ongoing, tumor agnostic, registration-intended PRECISION1 trial; and a Phase 2 study of nab-sirolimus in combination with letrozole for advanced or recurrent endometrioid-type endometrial cancer (EEC). "Overactivation of the mTOR pathway has been implicated in gynecological cancers," said Loretta Itri, MD, Chief Medical Officer at Aadi. "nab-Sirolimus is a nanoparticle albumin-bound (nab) mTOR inhibitor under investigation in TSC1- and TSC2-mutated tumors as well as other mTOR-driven tumors. We are diligently continuing to explore the potential of nab-sirolimus for this patient community in need of new therapies." Poster presentation details and highlights include: Title: "Analysis of inactivating TSC1 and TSC2 alterations in a real-world patient population with advanced gynecological cancers in the Foundation Medicine genomic database" Presenting Author: Lauren E. Dockery, MD, MS Session Title: Poster Session 1 Location: Exhibit Hall (Hall GH) Poster Number: 1176 Date/Time: Sunday, March 17, 2024 – 1:15 PM to 2:45 PM Registration-directed PRECISION1 study is enrolling patients with solid tumors harboring TSC1 and/or TSC2 inactivating alterations In a large real-world database of patients with advanced cancer, 1,342 (2.4%) of the 54,911 patients with gynecological cancers harbored at least one inactivating alteration in TSC1 or TSC2 TSC1 and/or TSC2 inactivating alterations were present in 3.6% of endometrial cancers, 2.0% of ovarian cancers and 1.5% of cervical cancers Title: "nab-Sirolimus Plus Letrozole in Advanced or Recurrent Endometrioid Endometrial Cancer: A Phase 2, Open-Label, Single-Arm, Prospective, Multi-Center Study" Presenting Author: Lauren E. Dockery, MD, MS Session Title: Poster Session 2 Location: Exhibit Hall (Hall GH) Poster Number: 2127 Date/Time: Monday, March 18, 2024 – 11:45 AM to 12:45 PM This ongoing phase 2, open-label, single-arm, multicenter study is evaluating nab-sirolimus in combination with letrozole for the treatment of patients with advanced or recurrent EEC Prior clinical studies with mTOR inhibitors and endocrine therapy have yielded promising results in EEC Title: "nab-Sirolimus for Malignant Solid Tumors Harboring Pathogenic Inactivating Alterations in TSC1 and TSC2 in a Phase 2, Multicenter, Open-Label Tumor-Agnostic Trial: PRECISION 1" Presenting Author: Debra L. Richardson, MD Session Title: Poster Session 2 Location: Exhibit Hall (Hall GH) Poster Number: 2126 Date/Time: Monday, March 18, 2024 – 11:45 AM to 12:45 PM TSC1 and/or TSC2 inactivating alterations have been observed in patients with gynecological cancers with a frequency of up to 5.0% in endometrial cancer, 2.2% in ovarian cancer and 1.5% in cervical cancer About Aadi Bioscience Aadi is a commercial-stage precision oncology company focused on the development and commercialization of therapies for cancers with alterations in the mTOR pathway, a key regulator of cell growth and cancer progression. To unlock the full potential of mTOR inhibition, Aadi uniquely combines nanoparticle albumin-bound (nab) technology with the potent mTOR inhibitor, sirolimus. Aadi received FDA approval and commercializes FYARRO® for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). Aadi is exploring nab-sirolimus in PRECISION1, a Phase 2 tumor-agnostic registration-intended trial in mTOR inhibitor-naïve malignant solid tumors harboring TSC1 or TSC2 inactivating alterations. Aadi is also exploring nab-sirolimus in two single-indication Phase 2 trials for difficult-to-treat mTOR-driven cancers: neuroendocrine tumors (NETs), and advanced or recurrent endometrioid-type endometrial cancer (EEC) in combination with letrozole. More information on the Company's development pipeline is available on the Aadi website at and connect with us on Twitter and LinkedIn. Forward-Looking Statements This press release contains certain forward-looking statements regarding the business of Aadi Bioscience that are not a description of historical facts within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based on the Company's current beliefs and expectations and may include, but are not limited to, statements relating to: expectations regarding the beneficial characteristics, safety, efficacy and therapeutic effects of FYARRO; expectations regarding the clinical responses and safety profile, regulatory approval and commercialization, and the sufficiency of the Company's existing capital resources and the expected timeframe to fund the Company's 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, those associated with the uncertainties associated with the clinical development and regulatory approval of FYARRO in additional indications; the risk that interim or subgroup analysis 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; and risks related to Aadi's estimates regarding future expenses, capital requirements and need for additional financing. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including under the caption "Item 1A. 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 . 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 cautionary statement is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contact: [email protected] SOURCE Aadi Bioscience

FDA assigns PDUFA target action date of March 8, 2024 New Drug Application supported by positive Phase III efficacy and safety clinical trial results Milestone builds upon Viatris' long-standing and continued commitment toward addressing the unmet needs of the MS community PITTSBURGH and NESS ZIONA, Israel, Aug. 7, 2023 /PRNewswire/ -- Viatris Inc. (NASDAQ: VTRS), a global healthcare company, and Mapi Pharma Ltd., a fully integrated, late-stage clinical development pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the companies' recently submitted New Drug Application (NDA) for GA Depot 40 mg. The product is a long-acting glatiramer acetate being investigated as a once-monthly injection for the treatment of relapsing forms of multiple sclerosis (RMS). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of March 8, 2024. Viatris President Rajiv Malik said, "The NDA filing acceptance for GA Depot is yet another example of our continuous commitment to look for opportunities to enhance existing therapies and innovation to support unmet medical needs. Our application is backed by Phase III efficacy and safety data, and we believe, when approved, GA Depot could improve patient experience through fewer injections, greater tolerability and increased compliance. This milestone gives us further confidence in the strength of our GA Depot clinical program, and we look forward to continuing to work closely with FDA to bring access to this important complex medicine to patients." According to a landmark study funded by the National Multiple Sclerosis Society, nearly one million people are living with MS in the U.S., which is more than twice as many people than was previously thought.1 Approximately 85 percent of people with MS are initially diagnosed with relapsing-remitting multiple sclerosis.2 The GA Depot NDA filing is supported by results from a multinational, double blind, placebo-controlled Phase III clinical trial evaluating the efficacy, safety and tolerability of GA Depot compared with placebo in patients with RMS. A total of 1,016 subjects were randomized into two groups, receiving either 40mg of GA Depot or placebo, via intramuscular injection (IM), once every 4 weeks for a total of 13 doses. The study concluded that the product offers a preferable schedule and with expected fewer Injection Site Reactions than other GA products. The study met its primary endpoint showing that GA Depot 40 mg statistically significantly reduced the annualized relapse rate by 30.1 percent compared to placebo (p=0.0066). "We are confident that GA Depot, when approved, will represent an important advancement in MS care by offering a convenient once-monthly option for patients which may potentially improve compliance and adherence, and the medicine is well positioned to deliver on this important unmet need," said Ehud Marom, CEO and Chairman, Mapi Pharma. "I commend the teams at Mapi and Viatris for the strong collaboration which has leveraged our collective expertise in complex products to deliver this novel medicine." About GA Depot GA Depot is a long-acting injection version of the approved Glatiramer Acetate (GA, commercially available as Copaxone®), designed to be administered as an intramuscular injection once every four weeks. GA Depot is intended to be used for treatment of Relapsing forms of Multiple Sclerosis (RMS). GA Depot is also currently being tested in Phase II for Primary Progressive Multiple Sclerosis (PPMS). References: About Viatris Viatris Inc. (NASDAQ: VTRS) is a global healthcare company empowering people worldwide to live healthier at every stage of life. We provide access to medicines, advance sustainable operations, develop innovative solutions and leverage our collective expertise to connect more people to more products and services through our one-of-a-kind Global Healthcare Gateway®. Formed in November 2020, Viatris brings together scientific, manufacturing and distribution expertise with proven regulatory, medical, and commercial capabilities to deliver high-quality medicines to patients in more than 165 countries and territories. Viatris' portfolio comprises more than 1,400 approved molecules across a wide range of therapeutic areas, spanning both non-communicable and infectious diseases, including globally recognized brands, complex generic and branded medicines, and a variety of over-the-counter consumer products. With more than 38,000 colleagues globally, Viatris is headquartered in the U.S., with global centers in Pittsburgh, Shanghai and Hyderabad, India. Learn more at viatris.com and investor.viatris.com, and connect with us on Twitter, LinkedIn, Instagram and YouTube. About Mapi Pharma Mapi Pharma is a clinical stage pharmaceutical company, engaged in development of high barrier-to-entry and high added-value life cycle management ("LCM") products and AB Rated Depot injectable products that target large markets that include complex active pharmaceutical ingredients ("APIs") and formulations. Mapi Pharma partnered with Viatris for GA Depot in an agreement under which Viatris was granted an exclusive license to commercialize the GA Depot injection product for relapsing forms of multiple sclerosis. The Company is also marketing its own generic versions of Fingolimod (Gilenya®) and Apremilast (Otezla®) in specific geographic markets. Mapi's portfolio also includes a leading development of Depot drugs for Schizophrenia, GLP-1 for diabetes, weight control, Parkinson's disease and potentially Alzheimer's with innovative intellectual property. Mapi is built on strong chemical and pharmaceutical R&D capabilities, a deep understanding of the global market and of regulatory needs. Mapi is headquartered in Israel, with R&D facilities in Israel and China, and an API production facility, and an aseptic manufacturing and a Fill & Finish facility for injectable Finished Dosage Forms, all in Israel. Mapi has a strong IP position, filing numerous patent applications for APIs and formulations. For more information, please visit . Forward-Looking Statements This press release includes statements that constitute "forward-looking statements." These statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward looking statements may include statements regarding FDA acceptance of NDA filing for GA Depot for the treatment of relapsing forms of multiple sclerosis; FDA assigns PDUFA target action date of March 8, 2024; the NDA filing acceptance for GA Depot is yet another example of our continuous commitment to look for opportunities to enhance existing therapies and innovation to support unmet medical needs; our application is backed by Phase III efficacy and safety data, and we believe, when approved, GA Depot could improve patient experience through fewer injections, greater tolerability and increased compliance; this milestone gives us further confidence in the strength of our GA Depot clinical program, and we look forward to continuing to work closely with FDA to bring access to this important complex medicine to patients. Factors that could cause or contribute to such differences include, but are not limited to: the possibility that the Company may be unable to realize the intended benefits of, or achieve the intended goals or outlooks with respect to, its strategic initiatives; the possibility that the Company may be unable to achieve expected benefits, synergies and operating efficiencies in connection with acquisitions, divestitures, or its global restructuring program, within the expected timeframe or at all; impairment charges or other losses related to the divestiture or sale of businesses or assets; the Company's failure to achieve expected or targeted future financial and operating performance and results; the potential impact of public health outbreaks, epidemics and pandemics, including the ongoing challenges and uncertainties posed by COVID-19; actions and decisions of healthcare and pharmaceutical regulators; changes in healthcare and pharmaceutical laws and regulations in the U.S. and abroad; any regulatory, legal or other impediments to Viatris' ability to bring new products to market, including but not limited to "at-risk" launches; Viatris' or its partners' ability to develop, manufacture, and commercialize products; the scope, timing and outcome of any ongoing legal proceedings, and the impact of any such proceedings; any significant breach of data security or data privacy or disruptions to our information technology systems; risks associated with international operations; the ability to protect intellectual property and preserve intellectual property rights; changes in third-party relationships; the effect of any changes in Viatris' or its partners' customer and supplier relationships and customer purchasing patterns; the impacts of competition; changes in the economic and financial conditions of Viatris or its partners; uncertainties and matters beyond the control of management, including general economic conditions, inflation and exchange rates; failure to execute stock repurchases consistent with current expectations; stock price volatility; and the other risks described in Viatris' filings with the Securities and Exchange Commission (SEC). Viatris routinely uses its website as a means of disclosing material information to the public in a broad, non-exclusionary manner for purposes of the SEC's Regulation Fair Disclosure (Reg FD). Viatris undertakes no obligation to update these statements for revisions or changes after the date of this release other than as required by law. SOURCE Viatris Inc.