CBFA2T3

– Complete remission seen in 42% of patients with CBF/GLIS AML with ≥5% blasts and 75% of pediatric patients with CBF/GLIS AML with <5% blasts; treatment with luvelta was either alone or in combination – – Luvelta was generally well-tolerated – – Data supports potential further development in CBF/GLIS AML – SOUTH SAN FRANCISCO, Calif., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), today announced that its research collaborators presented data on anti-leukemic activity from the compassionate use of luveltamab tazevibulin (luvelta), a novel folate receptor-α (FR-α) targeting ADC, in pediatric patients with relapsed/refractory CBFA2T3-GLIS2 (CBF/GLIS) acute myeloid leukemia (AML), commonly known as RAM phenotype AML. Data demonstrated that treatment with luvelta produced meaningful clinical responses, including complete remission (CR); and prolongs overall survival (OS) enabling some patients to receive potentially curative therapies such as hematopoietic stem cell transplant. These patients were treated under the single patient IND mechanism. These data were featured in a poster presentation at the 65th American Society of Hematology Annual Meeting and Exposition (ASH 2023) in San Diego, CA. “Treatment with luvelta led to notable response in a significant subset of patients who had exhausted all therapeutic options,” said Soheil Meshinchi, M.D., Ph.D., presenter and primary author. “Response varied from deep remissions to disease stabilization with minimal toxicity – mostly in outpatient setting. Luvelta was well tolerated as long-term maintenance therapy with little to no hematopoietic toxicity.” CBF/GLIS subtype AML is a rare and highly lethal form of leukemia found exclusively in infants and young children, with the average age of onset at 18 months 1. There are no therapies specifically approved to target this form of leukemia and it is resistant to conventional chemotherapy, with an induction failure rate of over 80%2. Due to a lack of effective treatment, children diagnosed with the disease have a dismal two-year survival rate of 15%3. Recent studies have shown that FOLR1, which encodes for FolRα, is silent in normal hematopoiesis, but is uniquely induced by the CBF/GLIS fusion4. Under compassionate use, 25 pediatric patients with relapsed/refractory CBF/GLIS subtype AML were treated with luvelta at doses up to 4.3 or 5.2mg/kg every two to four weeks for a median duration of 15.9 weeks (3-73.1), with the majority of patients receiving at least five doses (68%). Of the 25 treated patients, 19 had ≥5% blasts (morphologic disease, or MD) and 8 had <5% blasts (sub-morphologic disease, or SMD)5. Collective results show that treatment with luvelta produced clinically meaningful and durable responses across a broad range of patients in various settings including in patients with or without prior stem cell transplant and in monotherapy or in combination with cytotoxic therapy. These data were generated by the treating physicians and collected and enabled for presentation by Sutro. “It is clear from these data that luvelta is providing an ongoing and promising impact on the lives of infants and young children with this rare leukemia,” said Bill Newell, Sutro’s Chief Executive Officer. “These results add to the growing body of research supporting the development of luvelta, which has now seen positive clinical results across three different tumor types, including those with potentially low or variable folate receptor-α expression.” ASH Presentation Highlights: Overall, anti-leukemic activity was seen with luvelta either as a single agent or in combination. 19 patients had ≥5% blasts and 8 patients had <5% blasts5. A CR/CRh was observed in 8 out of 19 (42%) patients with ≥5% blasts treated with luvelta, with 5 out of 8 CR/CRh patients reaching a minimal residual disease (MRD)-negative CR (63%).6 out of 8 patients with <5% blasts experienced an MRD-negative CR (75%). Patients whose leukemia experienced an MRD-negative CR had an improved outcome over those who did not experience an MRD-negative CR.Treatment with luvelta also enabled some children to bridge to stem cell transplant, which is potentially curative therapy.Luvelta was well-tolerated as a monotherapy agent and in combination with standard of care therapies with minimal hematopoietic toxicity and can be delivered as outpatient therapy.As of September 17, 2023, 8 patients remain on treatment, with 5 of the 8 (63%) in continued remission and on luvelta maintenance. The poster titled, “Anti-leukemic Activity of Luveltamab Tazevibulin (LT, STRO-002), a Novel Folate Receptor-α (FR-α)-targeting Antibody Drug Conjugate (ADC) in Relapsed/Refractory CBFA2T3::GLIS2 AML,” will be accessible through the News & Events page of the Investor Relations section of the company’s website at www.sutrobio.com. *1: National Institutes of Health [NIH], 2022; Quessada et al 2021; Masseti et al 2019*2: Smith JL, et al. Comprehensive Transcriptome Profiling of Cryptic CBFA2T3-GLIS2 Fusion-Positive AML Defines Novel Therapeutic Options: A COG and TARGET Pediatric AML Study. Clin Cancer Res. 2020 Feb 1;26(3):726-737. doi: 10.1158/1078-0432.CCR-19-1800. Epub 2019 Nov 12. PMID: 31719049; PMCID: PMC7002196.*3: Tang T, et al. Targeting FOLR1 in high-risk CBF2AT3-GLIS2 pediatric AML with STRO-002 FOLR1-antibody-drug conjugate, Blood Adv. 2022 Nov 22;6(22):5933-5937. doi: 10.1182/bloodadvances.2022008503. PMID: 36149945; PMCID: PMC9701621. *4: Le Q, et al. Targeting FOLR1 in High-Risk CBFA2T3-GLIS2 AML with Stro-002 FOLR1-Directed Antibody-Drug Conjugate, Blood, Volume 138, Supplement 1, 2021, Page 209, ISSN 0006-4971, https://doi.org/10.1182/blood-2021-153076.*5: Two patients initially presented with <5% blasts, received luvelta and proceeded to BMT. Both relapsed post-transplant with ≥5% blasts and re-started luvelta. About Luveltamab TazevibulinLuveltamab tazevibulin, abbreviated as “luvelta” and formerly known as STRO-002, is a FolRα-targeting antibody-drug conjugate (ADC) designed to treat a broad range of patients with ovarian cancer, including those with lower FolRα-expression who are not eligible for approved treatment options targeting FolRα. Developed and manufactured with Sutro’s cell-free XpressCF® platform, luvelta is a homogeneous ADC with four hemiasterlin cytotoxins per antibody, precisely positioned to efficiently deliver to the tumor while ensuring systemic stability after dosing. Sutro recently initiated REFRaME, a Phase 2/3 registration-directed study for patients with platinum-resistant ovarian cancer. The company has ongoing trials in patients with endometrial cancer and in combination with bevacizumab in patients with ovarian cancer. The company is also assessing the clinical path forward for CBF/GLIS2 acute myeloid leukemia, a rare subtype of pediatric cancer, as well as non-small cell lung cancer. The U.S. Food and Drug Administration (FDA) has granted luvelta a Fast Track designation for Ovarian Cancer, as well as Orphan and Rare Pediatric Disease designations for CBF/GLIS2 Pediatric AML. About Sutro Biopharma Sutro Biopharma, Inc., is a clinical-stage company relentlessly focused on the discovery and development of precisely designed cancer therapeutics, transforming what science can do for patients. Sutro’s fit-for-purpose technology, including cell-free XpressCF®, provides the opportunity for broader patient benefit and an improved patient experience. Sutro has multiple clinical stage candidates, including luveltamab tazevibulin, or luvelta, a registrational-stage folate receptor alpha (FolRα)-targeting ADC in clinical studies. A robust pipeline, coupled with high-value collaborations and industry partnerships, validates Sutro’s continuous product innovation. Sutro is headquartered in South San Francisco. For more information, follow Sutro on social media @Sutrobio, or visit www.sutrobio.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, anticipated preclinical and clinical development activities, timing of announcements of clinical results, trial initiation, and regulatory filings, potential benefits of luvelta and the Company’s other product candidates and platform, potential future milestone and royalty payments, the Company’s expectations about its cash runway, and potential market opportunities for luvelta and the Company’s other product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, the Company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the Company’s ability to advance its product candidates, the receipt and timing of potential regulatory designations, approvals and commercialization of product candidates and the Company’s ability to successfully leverage Fast Track designation, the market size for the Company’s product candidates to be smaller than anticipated, clinical trial sites, supply chain and manufacturing facilities, the Company’s ability to maintain and recognize the benefits of certain designations received by product candidates, the timing and results of preclinical and clinical trials, the Company’s ability to fund development activities and achieve development goals, the Company’s ability to protect intellectual property, the value of the Company’s holdings of Vaxcyte common stock, and the Company’s commercial collaborations with third parties and other risks and uncertainties described under the heading “Risk Factors” in documents the Company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. ContactEmily WhiteSutro Biopharma (650) 823-7681ewhite@sutrobio.com

- Updated luvelta data from Compassionate Use Access demonstrating anti-leukemic activity in pediatric patients with relapsed/refractory CBF2AT3-GLIS2 AML to be presented in a poster at ASH 2023 - - Luvelta demonstrated encouraging preliminary anti-tumor activity in initial data from the Phase 1 dose-expansion study for patients with endometrial cancer presented at ESMO 2023 - - Dr. Hans-Peter Gerber joined Sutro as Chief Scientific Officer in September 2023 - - As of September 30, 2023, Sutro had cash and investments of $321.1 million and shares of Vaxcyte common stock valued at $34.0 million, which together provide a projected cash runway into the first half of 2025 - SOUTH SAN FRANCISCO, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), today reported its financial results for the third quarter of 2023, its recent business highlights, and a preview of select anticipated milestones. “The last few months have been productive for Sutro, with the recent presentation of encouraging early data for luvelta in endometrial cancer, as well as the appointment of Dr. Hans-Peter Gerber as our CSO. Dr. Gerber’s extensive experience in discovering and developing novel ADCs further strengthens our position as a scientific leader in this important therapeutic modality,” said Bill Newell, Sutro’s Chief Executive Officer. “The recent encouraging update with luvelta in endometrial cancer gave us further confidence in the potential for luvelta to be a targeted treatment for a range of indications with significant unmet need. Additionally, we are pleased with the pace of enrollment of our Phase 2/3 trial for luvelta in patients with platinum resistant ovarian cancer, REFRaME-O1.” “Sutro’s remarkable platform has the potential to yield best in class product candidates from all of our ADC design concepts – including ADCs, iADCs, and ADC2. The diversity of product candidate attributes enabled by Sutro technology is what originally attracted me to the company,” said Hans-Peter Gerber, Sutro’s CSO. “After an in-depth review of the platform and programs developed at Sutro, I am excited to combine our industry leading technology with our established development capabilities to deliver fit-for-purpose molecules that could transform the lives of cancer patients with limited options.” Recent Business Highlights and Select Anticipated Milestones STRO-002, International Nonproprietary Name, “luveltamab tazevibulin,” abbreviated as “luvelta,” FolRα-Targeting ADC: Luvelta is being studied in the clinic globally for patients with ovarian and endometrial cancers. •Initial results from the Phase 1 dose-expansion study for luvelta in patients with endometrial cancer were presented in a mini oral presentation at the 2023 European Society for Medical Oncology (ESMO) Congress held in Madrid, Spain in October 2023. Luvelta demonstrated encouraging preliminary anti-tumor activity in FolRα-selected patients, defined by a Tumor Proportion Score (TPS) of >25% FolRα expression, and the safety profile was consistent with prior data in patients with platinum-resistant ovarian cancer. •Previous data from the Phase 1 dose-expansion study for luvelta in ovarian cancer demonstrated meaningful clinical benefit in FolRα-selected patients, defined by a TPS of >25%, irrespective of staining intensity, in which the data collected has shown to represent approximately 80% of the advanced ovarian cancer patient population. •In June 2023, Sutro announced the initiation of Part I of REFRaME-O1, a Phase 2/3 registration-directed study for patients with platinum-resistant ovarian cancer. The trial is well underway, and sites have been activated globally. Sutro is also in discussions with both the FDA and EMA to refine the trial design for REFRaME-O1 to potentially support global registration of luvelta. •Patients with CBFA2T3::GLIS2 (CBF/GLIS; RAM phenotype) AML, a highly refractory and uniformly fatal subtype of acute myeloid leukemia found exclusively in infants and young children, were treated with luvelta under compassionate use. Preliminary results from compassionate use of luvelta in this ultra rare indication suggested that luvelta was well tolerated as a monotherapy agent and in combination with standard cancer therapies. Soheil Meshinchi, M.D., Ph.D., expects to present updated data from this program in a poster at the 65th American Society of Hematology Annual Meeting and Exposition (ASH 2023) to be held December 9-12, 2023 in San Diego, CA. Title: Anti-Leukemic Activity of Luveltamab Tazevibulin (LT, STRO-002), a Novel Folate Receptor-α (FR-α)-Targeting Antibody Drug Conjugate (ADC) in Relapsed/Refractory CBFA2T3::GLIS2 AML Session: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III Date & Time: Monday, December 11, 2023, 6:00 PM - 8:00 PM PT •Luvelta is also being studied in combination with bevacizumab for patients with advanced ovarian cancer. •Translational work is ongoing to support an Investigational New Drug (IND) application for the initiation of a study of luvelta for patients with non-small cell lung cancer (NSCLC), for which the protocol is under development. Additional Pipeline Development: STRO-003, a ROR1-targeting ADC, and STRO-004, a tissue factor-targeting ADC, have INDs planned for 2024 and 2025, respectively. •STRO-003, a novel, next-generation ADC that has been designed to target ROR1, features eight precisely placed β-Glucuronidase-cleavable linkers attached to next-generation exatecan warheads, which, when released, inhibit topoisomerase-1 (TOPO-1) and cause DNA disruption. •STRO-003 has demonstrated, in NSCLC and breast cancer patient-derived xenograft models, strong cell-killing activity in low and heterogeneous ROR1-expressing tumors. STRO-003 has also exhibited promising tolerability in preclinical studies involving rodents and non-human primates, with potentially reduced lung toxicity relative to other TOPO-1 inhibiting ADCs. Collaboration Updates: Sutro continues to seek to maximize the value of its proprietary cell-free platform by working with partners on programs in multiple disease spaces and geographies and has generated from collaborators an aggregate of approximately $785 million in payments through September 30, 2023, including equity investments. •In June 2023, Sutro announced a royalty monetization agreement with Blackstone Life Sciences, an affiliate of Blackstone, under which Sutro received $140 million upfront and is eligible to receive up to an additional $250 million in future milestone payments in exchange for the 4% royalty, or revenue interest, in potential future sales of Vaxcyte’s products. This transaction with Blackstone provides non-dilutive capital to Sutro for continued pipeline advancement. Sutro retains the right to discover and develop vaccines for the treatment or prophylaxis of any disease that is not caused by an infectious pathogen, including cancer. •In December 2022, Sutro and Vaxcyte expanded upon a nearly decade-long relationship through a new agreement, under which Vaxcyte acquired an option to access expanded rights to develop and manufacture cell-free extract, among other rights, and includes a $22.5 million upfront payment and, upon exercise of the option, up to an additional $135 million in option exercise and contingent payments. •Sutro’s collaboration with Astellas on the discovery of immunostimulatory antibody-drug conjugates (iADCs) for three targets is ongoing, for which Sutro receives financial support for its research efforts, potential milestone payments and royalties, and has an option to co-develop and co-commercialize product candidates in the U.S. •Sutro is manufacturing initial drug supply for its partners including for Merck’s MK-1484, currently in Phase 1 as monotherapy and in combination with pembrolizumab in advanced or metastatic solid tumors. Sutro is providing clinical drug supply to BioNova for clinical studies for BN301 (STRO-001) in Greater China. Sutro is currently supporting Tasly Biopharmaceuticals (Tasly), which recently received its first IND clearance by the National Medical Products Administration, or NMPA, for their initiation of clinical development activities in Greater China for STRO-002 and will provide initial drug supply for their Phase 1 study. Corporate Updates: Sutro strengthened and continues to build a world-class leadership team through the appointment of a new Chief Scientific Officer. •Hans-Peter Gerber, Ph.D., joined Sutro as Chief Scientific Officer in September 2023, overseeing the research and early discovery functions, with a focus on the design and discovery of new molecules to rapidly progress into the clinic, in addition to being a member of Sutro’s Senior Management Team. Upcoming Events: Sutro plans to participate in two upcoming investor conferences. Webcasts of the presentations will be accessible through the News & Events page of the Investor Relations section of the company’s website at www.sutrobio.com. Archived replays will be available for at least 30 days after the events. •Jefferies London Healthcare Conference in London, Wednesday, November 14-16, 2023 •Piper Sandler 35th Annual Healthcare Conference in New York, November 28-30, 2023 Third Quarter 2023 Financial Highlights Cash, Cash Equivalents and Marketable SecuritiesAs of September 30, 2023, Sutro had cash, cash equivalents and marketable securities of $321.1 million, as compared to $358.3 million as of June 30, 2023, and approximately 0.7 million shares of Vaxcyte common stock with a fair value of $34.0 million, which together provide a projected cash runway into the first half of 2025, based on current business plans and assumptions. Current market conditions provide a challenging financing environment. In this context, Sutro is evaluating its programs and spending as it fully develops its 2024 goals and financial plan. Unrealized Gain from Increase in Value of Vaxcyte Common Stock The non-operating, unrealized gain of $0.7 million in the quarter ended September 30, 2023 was due to the increase since June 30, 2023 in the estimated fair value of Sutro’s holdings of Vaxcyte common stock. Vaxcyte common stock held by Sutro will be remeasured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations. Revenue Revenue was $16.9 million for the quarter ended September 30, 2023, as compared to $25.1 million for the same period in 2022, with the 2023 amount related principally to the Astellas collaboration and the recognition of a contingent payment from Tasly. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments. Operating Expenses Total operating expenses for the quarter ended September 30, 2023 were $60.9 million, as compared to $46.4 million for the same period in 2022. The third quarter of 2023 includes non-cash expenses for stock-based compensation of $6.0 million and depreciation and amortization of $1.7 million, as compared to $6.8 million and $1.4 million, respectively, in the comparable 2022 period. Total operating expenses for the quarter ended September 30, 2023 were comprised of research and development expenses of $45.7 million and general and administrative expenses of $15.3 million. Royalty Monetization Agreement As related to the royalty monetization agreement between Sutro and an affiliate of Blackstone Life Sciences, Sutro received in June 2023 a $140.0 million upfront payment and is eligible to receive up to an additional $250.0 million in future milestone payments. Sutro recorded the $140.0 million upfront payment from Blackstone as a deferred royalty obligation related to the sale of future royalties on the Company’s condensed Balance Sheets as of June 30, 2023. Due to the Company’s ongoing manufacturing obligations, the Company accounted for the proceeds as imputed debt and will recognize future non-cash royalty revenues. Non-cash interest expense will be recognized over the estimated life of the royalty term arrangement using the effective interest method based on the imputed interest rate derived from estimated amounts and timing of future royalty payments to be received from Vaxcyte. As part of the sale, Sutro incurred approximately $3.8 million in transaction costs, which are being amortized over the estimated life of the royalty term arrangement using the effective interest method. As future royalties are earned from Vaxcyte by Blackstone, the balance of the deferred royalty obligation will be amortized over the estimated life of the royalty term arrangement. About Sutro Biopharma Sutro Biopharma, Inc., is a clinical-stage company relentlessly focused on the discovery and development of precisely designed cancer therapeutics, transforming what science can do for patients. Sutro’s fit-for-purpose technology, including cell-free XpressCF®, provides the opportunity for broader patient benefit and an improved patient experience. Sutro has multiple clinical stage candidates, including luveltamab tazevibulin, or luvelta, a registrational-stage folate receptor alpha (FolRα)-targeting ADC in clinical studies. A robust pipeline, coupled with high-value collaborations and industry partnerships, validates Sutro’s continuous product innovation. Sutro is headquartered in South San Francisco. For more information, follow Sutro on social media @Sutrobio, or visit www.sutrobio.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, anticipated preclinical and clinical development activities, timing of announcements of clinical results, trial initiation, and regulatory filings, potential benefits of luvelta and the Company’s other product candidates and platform, potential future milestone and royalty payments, the Company’s expectations about its cash runway, and potential market opportunities for luvelta and the Company’s other product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, the Company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the Company’s ability to advance its product candidates, the receipt and timing of potential regulatory designations, approvals and commercialization of product candidates and the Company’s ability to successfully leverage Fast Track designation, the market size for the Company’s product candidates to be smaller than anticipated, clinical trial sites, supply chain and manufacturing facilities, the Company’s ability to maintain and recognize the benefits of certain designations received by product candidates, the timing and results of preclinical and clinical trials, the Company’s ability to fund development activities and achieve development goals, the Company’s ability to protect intellectual property, the value of the Company’s holdings of Vaxcyte common stock, and the Company’s commercial collaborations with third parties and other risks and uncertainties described under the heading “Risk Factors” in documents the Company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. ContactEmily WhiteSutro Biopharma (650) 823-7681 ewhite@sutrobio.com Sutro Biopharma, Inc.Selected Statements of Operations Financial Data(Unaudited)(In thousands, except share and per share amounts) Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Revenues$16,924 $25,147 $40,010 $59,140 Operating expenses Research and development 45,669 31,714 126,660 94,036 General and administrative 15,269 14,643 45,780 44,825 Total operating expenses 60,938 46,357 172,440 138,861 Loss from operations (44,014) (21,210) (132,430) (79,721)Interest income 4,550 1,014 9,952 1,327 Unrealized gain on equity securities 694 3,496 2,023 323 Non-cash interest expense related to the sale of future royalties (5,936) - (6,378) - Interest and other income (expense), net (2,739) (2,788) (8,640) (4,039)Loss before provision for income taxes (47,445) (19,488) (135,473) (82,110)Provision for income taxes 1,839 - 2,385 2,500 Net loss$(49,284) $(19,488) $(137,858) $(84,610)Net loss per share, basic and diluted$(0.81) $(0.37) $(2.30) $(1.74)Weighted-average shares used in computing basic and diluted loss per share 60,599,025 52,345,732 59,894,181 48,622,258 Sutro Biopharma, Inc.Selected Balance Sheets Financial Data(Unaudited)(In thousands) September 30,2023(1) December 31,2022(2) Assets Cash, cash equivalents and marketable securities$321,108 $302,344 Investment in equity securities 34,043 32,020 Accounts receivable 16,627 7,122 Property and equipment, net 22,344 24,621 Operating lease right-of-use assets 23,996 26,443 Other assets 13,616 14,394 Total Assets$431,734 $406,944 Liabilities and Stockholders’ Equity Accounts payable, accrued expenses and other liabilities$36,312 $32,822 Deferred revenue 102,137 106,644 Operating lease liability 31,038 34,159 Debt 7,137 16,271 Deferred royalty obligation related to the sale of future royalties 142,763 - Total liabilities 319,387 189,896 Total stockholders’ equity 112,347 217,048 Total Liabilities and Stockholders’ Equity$431,734 $406,944 (1)The condensed balance sheet as of September 30, 2023 was derived from the unaudited financial statements included in the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, filed with the Securities and Exchange Commission on November 13, 2023.(2)The condensed balance sheet as of December 31, 2022 was derived from the audited financial statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 30, 2023.

SUZHOU, China, and ROCKVILLE, Md., Nov. 2, 2023 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, announced today that results from multiple clinical studies of the company's novel drug candidate, olverembatinib, have been selected for presentations, including two Oral Reports, at the 65th American Society of Hematology (ASH) Annual Meeting. Being selected for Oral Reports at the ASH Annual Meeting for the sixth consecutive year underscores the significant interest in the drug by the global hematology community. This year, results from multiple clinical studies on two of Ascentage Pharma's lead drug candidates (olverembatinib and lisaftoclax) have been selected for presentations at the ASH Annual Meeting. As a potential best-in-class drug, olverembatinib has broad therapeutic potential for the treatment of multiple solid tumors and hematologic malignancies. Currently, olverembatinib is being jointly commercialized by Ascentage Pharma and Innovent Biologics. Through an Oral Report at this year's ASH Annual Meeting, Ascentage Pharma will present the latest results from a randomized, controlled registrational Phase II study in patients with first- and second-generation tyrosine kinase inhibitor (TKI)-resistant chronic myeloid leukemia in the chronic-phase (CML-CP), led by Prof. Xiaojun Huang and Prof. Qian Jiang, from the Institute of Hematology of the Peking University People's Hospital. These data show that, in patients with CML-CP who were resistant/intolerant to TKIs, olverembatinib demonstrated statistically significant and clinically meaningful improvement in event-free survival (EFS), compared with the best available therapy (BAT), meeting the primary endpoint of the study. The other Oral Report featuring early results from a Phase II study of olverembatinib combined with venetoclax and reduced-intensity chemotherapy in treatment-naïve patients with Ph+ ALL, led by Prof. Xiaoyuan Gong of the Institute of Hematology and Blood Diseases Hospital, the Chinese Academy of Medical Sciences Also at this year's ASH Annual Meeting, Ascentage Pharma will release updated results from a US study of olverembatinib in a Poster Presentation. These data demonstrate the favorable potential clinical benefit of olverembatinib monotherapy and combination regimens in patients with heavily pretreated CML or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) and show the drug's therapeutic effect in patients who had failed prior treatment with ponatinib or asciminib, further demonstrating the drug's potential as a new treatment option to patients with CML or Ph+ ALL worldwide. In addition, multiple studies of olverembatinib were also selected for Poster Presentations, including one featuring a case series study of liposome mitoxantrone combined with venetoclax, homoharringtonine, and olverembatinib (the MVHO regimen) in pediatric patients with refractory or recurrent acute myeloid leukemia (AML), carried out by a team of investigators including Wenting Hu and Prof. Shuhong Shen of the Department of Hematology & Oncology, Shanghai Children's Medical Center of Shanghai Jiao Tong University School of Medicine (see the table below for details). The ASH Annual Meeting is one of the largest gatherings of the international hematology community, bringing together the latest and most cutting-edge scientific research in the pathogenesis and clinical treatment of hematologic diseases. The 65th ASH Annual Meeting will take place on December 9-12, 2023, local time, both online and in-person in San Diego, CA (United States). "This is the sixth consecutive year for the updated clinical data of olverembatinib to be accepted for presentation at the ASH Annual Meeting, a new record for the drug," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "This year, results from multiple studies of olverembatinib have been selected for presentations, including two Oral Reports, at the meeting. This indicates the global hematology community's strong recognition of olverembatinib, a global best-in-class drug. The large body of clinical data we are bringing to this year's ASH Annual Meeting underscores our broad progress in new drug discovery and clinical development. Moving forward, we will continue to expeditiously advance our clinical development programs globally for the benefit of patients in China and around the world." Studies of Ascentage Pharma's Drug Candidates to be presented at ASH 2023 Abtracts on olverembatinib presented at the 2023 ASH Annual Meeting are as follows (for details on the abstracts featuring lisaftoclax, please refer to a separate press release published at the same time): Olverembatinib (HQP1351) Demonstrates Efficacy Vs. Best Available Therapy (BAT) in Patients (Pts) with Tyrosine Kinase Inhibitor (TKI)-Resistant Chronic Myeloid Leukemia Chronic-Phase (CML-CP) in a Registrational Randomized Phase 2 Study Format: Oral Report Abstract: #869 Session: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Novel Therapeutic Approaches Time: December 11, 2023, Monday, 3:45 PM (Pacific Time) / December 12, 2023, Tuesday, 7:45 AM (Beijing Time) Highlights: This is a multicenter, randomized, registrational Phase II study designed to assess the efficacy and safety of olverembatinib compared with the BAT in patients with CML-CP. As of April 30, 2023, a total of 144 patients were enrolled, of whom 69.4% were male and 30.6% were female. The median (range) age of these patients was 49.0 (18-77) years. A total of 96 patients were treated with olverembatinib, and 48 patients were treated with BAT. The median (range) duration of follow-up for the olverembatinib and BAT arms were 12.67 (0.0-40.9) months and 2.94 (0.0-40.4) months, respectively. 66 (45.8%) patients had ≥1 BCR::ABL1 mutations, and 39 (27.1%) had the "gatekeeper" BCR::ABL1T315I mutation, which confers resistance against all first- and second-generation tyrosine kinase inhibitors (TKIs). A total of 97 patients discontinued therapies (56 [58.3%] from the olverembatinib arm, 41 [85.4%] from the BAT arm) due to disease progression, treatment failure, adverse events (AEs), consent withdrawal, poor compliance, or death. Safety results: 82/96 (85.4%) patients receiving olverembatinib and 31/46 (67.4%) patients receiving BAT experienced grade≥3 AEs. AEs with an incidence >20% included thrombocytopenia; leukopenia; anemia; neutropenia; elevated creatine phosphokinase (CPK), alanine aminotransferase (ALT), and aspartate aminotransferase (AST); and hypertriglyceridemia. Serious AEs (SAEs) with an incidence >5% included thrombocytopenia. Common AEs (≥10%) that led to discontinuation, dose reduction, or treatment withdrawal were thrombocytopenia, neutropenia, and leukopenia. Efficacy results: Data showed that, in patients with CML-CP resistant/intolerant to prior treatment with TKIs, the olverembatinib arm, compared with the BAT arm, achieved statistically significant improvement in event-free survival (EFS), therefore meeting the primary endpoint of the study. The median (range) EFS was 21.22 (95% CI: 10.15 to not reached) months in the olverembatinib arm compared to 2.86 (95% CI 2.53-4.73) months in the BAT arm. Compared with the BAT control arm, olverembatinib reduced the risk of events by 65%. In the olverembatinib arm, the estimated EFS at 6, 12, and 24 months was 73% (95% CI, 62.5-81.0), 58.7% (95% CI, 47.5-68.2), and 46.9% (95% CI, 35.9-57.2), respectively. In the BAT group, it was 32.6% (95% CI,19.7-46.2), 26.1% (95% CI, 14.5-39.3), and 16.9% (95% CI, 7.7-29.2) at 6, 12 and 24 months, respectively. Neither the olverembatinib nor the BAT arm reached the median overall survival (OS). As of the data cutoff date, 34 (71%) patients in the BAT control arm were crossed-over to be treated with olverembatinib after reaching the EFS endpoint. The olverembatinib arm showed significantly better efficacy and higher response rates than the BAT control arm. Conclusions: Olverembatinib was observed to be better tolerated and more effective than BAT in patients with CML-CP resistant or intolerant to first- and second-generation TKIs. Update of Olverembatinib (HQP1351) Overcoming Ponatinib and/or Asciminib Resistance in Patients (Pts) with Heavily Pretreated/Refractory Chronic Myeloid Leukemia (CML) and Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL) Format: Poster Presentation Abstract: #1798 Session: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Poster I Time: December 9, 2023, Saturday, 5:30 PM - 7:30 PM (Pacific Time) / December 10, 2023, Sunday, 9:30 AM – 11:30 AM (Beijing Time) Highlights Olverembatinib, a novel, potent BCR::ABL1 tyrosine kinase inhibitor (TKI), has shown strong antitumor activity in patients with CML and Ph+ ALL. This abstract reports on the safety, efficacy, and pharmacokinetics (PK) of olverembatinib in patients with CML and Ph+ ALL outside China, particularly in patients previously treated with third-generation TKI ponatinib and/or allosteric STAMP inhibitor asciminib. Methods: Olverembatinib was administered orally once every other day (QOD) in 28-day cycles. In the monotherapy cohort, patients were enrolled after treatment failures on at least 2 prior TKIs and randomized to receive olverembatinib QOD at 30, 40, or 50 mg. In the combination cohort, patients with Ph+ B-cell precursor ALL (BCP ALL) or lymphoid CML-BP (CML-LBP) resistant to at least 1 TKI were enrolled and administered olverembatinib (30 or 40 mg) QOD in combination with blinatumomab. As of June 30, 2023, 76 patients were enrolled, including 57 with CML-CP and 19 with advanced Ph+ leukemia. The median (range) age was 54.5 (21-80) years and 56.6% of patients were male.11 (14.5%), 23 (30.3%) and 39 (51.3%) patients had received 2, 3, and ≥4 TKIs, respectively. A total of 52.6% of patients were previously treated with ponatinib, of whom 67.5% were resistant and 25.0% were intolerant to the drug, and 7.5% of patients failed for other reasons. A total of 27.6% of patients were previously treated with asciminib, of whom 71.4% were resistant and 19.1% were intolerant to the agent, and 9.5% failed for other reasons. At baseline, 32% of patients had T315I mutations, 38% had hypertension, and 17.1% had other cardiovascular comorbidities. The median (range) duration of treatment was 24.1 (0-134) weeks. PK analysis showed that western patients had a PK profile similar to historical data on Chinese patients. Safety results: 12 patients with CML-CP and 7 with advanced Ph+ leukemia discontinued treatment for reasons including AEs (n=4), disease progression (n=7), and other reasons (n=8). A total of 54 (83.1%) patients experienced treatment-related AEs (TRAEs) of any grade after receiving olverembatinib. Grade ≥3 AEs occurring in ≥3 patients (≥ 4.6% incidence) included thrombocytopenia (17%), neutropenia (13.8%), elevated blood creatine phosphokinase (13.8%), leukopenia (7.7%), anemia (4.6%), and elevated lipase (4.6%). 10 (15.4%) patients experienced olverembatinib treatment-related serious AEs (SAEs). 2 (3.1%) patients discontinued the study due to TRAEs, and no TRAE-related deaths were reported. Efficacy results: Among 50 efficacy-evaluable patients with CML-CP, 57% (25/44) achieved a complete cytogenetic response (CCyR), 43% (21/49) achieved a major molecular response (MMR), and efficacy continued to improve over time. The rate of MMR in patients with CML-CP treated for 6 months and 12 months was 66% and 88%, respectively. At 24 months, the rate of progression-free survival (PFS) was 75% (95% CI: 56.1-86.7) and the OS was 97.6% (95% CI: 90.8-99.4). Among patients whose disease failed ≥4 prior TKIs, CCyR and MMR rates were 57% (13/23) and 42% (11/26), respectively; In patients with CML-CP harboring the T315I mutation, CCyR and MMR rates were 60% (9/15) and 44% (7/16), respectively; In patients without the T315I mutation, CCyR and MMR rates were 55% (16/29) and 42% (14/33), respectively; In patients who had failed treatment with ponatinib, CCyR and MMR rates were 53% (8/15) and 38% (6/16), respectively; In patients who had failed treatment with asciminib, CCyR and MMR rates were 43% (3/7) and 38% (3/8), respectively. Among the 8 patients who were resistant/intolerant to both ponatinib and asciminib, 2 achieved MMR. Among the 13 efficacy-evaluable patients with advanced Ph+ leukemia, 3 (23%) achieved MMR, of whom 1 patient harbored the T315I mutation and 2 were T315I mutation negative and resistant to ponatinib. In the combination cohort, 2 patients with Ph+ BCP ALL received olverembatinib 30 mg QOD in combination with blinatumomab. Both patients achieved CCyR and 1 achieved negative minimal residual disease (MRD) status after 1 treatment cycle. Conclusions: Olverembatinib alone or combined with blinatumomab was efficacious and well tolerated in heavily pretreated patients with CML or Ph+ ALL. The efficacy of olverembatinib was not affected by prior exposure to ponatinib or asciminib, and the status of the T315I mutation. Olverembatinib may provide an effective treatment option for patients with CML or Ph+ ALL who have failed 2 or more TKIs. Olverembatinib Combined with Venetoclax and Reduced-Intensity Chemotherapy for Patients with Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia: Early results from a phase II study Format: Oral Report Abstract: #827 Session: 614. Acute Lymphoblastic Leukemia: Therapies, Excluding Transplantation and Cellular Immunotherapies: Optimal Frontline Treatment for ALL Time: December 11, 2023, Monday; 3:45 PM (Pacific Time) / December 12, 2023, Tuesday; 7:45 AM (Beijing Time) Highlights : Background: The combination of olverembatinib (HQP1351), a novel third-generation tyrosine kinase inhibitors (TKIs), with venetoclax generated high response rates in patients with relapsed/refractory Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph ALL). However, the efficacy and safety of these two agents-based regimens as frontline treatment remains unknown. Methods: This is a single-arm phase II study (NCT05594784) that enrolled patients (pts) ≥ 14 years (yrs) of age with newly diagnosed Ph+ ALL. Pts were treated with a combination of venetoclax (100 mg d1, 200 mg d2, 400 mg d3-28), olverembatinib 40 mg once every continuously other day, vincristine 1.4 mg/m2 (maximum dose 2 mg) on day 1, 8, 15, 22, and prednisone 60 mg/m2 on day 1-14; 40 mg/m2 on day 15-28 in cycle 1. In cycle 2-3, oral treatment with venetoclax 400 mg × 7 days, olverembatinib once every other day continuously and prednisone 60 mg/m × 7 days were administrated. Cycles were repeated every 28 days. During cycle 1, The dose of olverembatinib was reduced to 30 mg once every other day for pts achieving a complete molecular response (CMR). The primary endpoint of this study was the CMR rate at 3 months. CMR was defined as undetectable BCR:ABL1 transcripts by using the RT-PCR method with sensitivity of 0.001%. MMR was defined as more than 3-log reduction of BCR:ABL1 transcripts. From August 2022 to April 2023, a total of 31 pts were enrolled. All pts completed 3 cycles of treatment and could be assessed for the primary endpoint. The data cutoff date was July 25, 2023 with a median follow-up time of 5.8 months. The median age was 40 years (range, 20-66 years) and males accounted for 58.1%. Twenty-three pts (74.2%) expressed the p190 transcript and 8 pts (25.8%) expressed the p210 transcript. The median expression level of BCR:ABL1 was 96.33% (range, 70.79%-175.39%). Efficacy results: All patients achieved CR at the end of cycle 1 and no TLS or treatment-related deaths occurred. Molecular response at the end of cycle 1 was CMR in 17 patients (54.8%), and MMR in 8 (25.8%). Molecular response at 3 months was CMR in 19 patients (61.3%) and MMR in 10 (32.3%). No patients developed relapses or deaths at the last follow-up. Safety results: The regimen was well-tolerated and safe. Most side effects were grade 1-2. The demand for transfusion and the incidence of infections significantly decreased compared to our historic data. No patient discontinued olverembatinib or venetoclax due to toxicity. Conclusions: The combination of olverembatinib and venetoclax with reduced-intensity chemotherapy is a safe and effective regimen in patients with newly diagnosed Ph ALL. The regimen results in high rates of CMR in the absence of intensive chemotherapy or immunotherapy. Combination of Liposome Mitoxantrone, Venetoclax, Homoharringtonine, and Olverembatinib (HQP1351) (MVHO) in Pediatric Patients with Refractory or Recurrent Acute Myeloid Leukemia (AML): Case Series Format: Poster Presentation Abstract: #2840 Session: 613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster II Time: December 10, 2023, Sunday, 6:00 PM - 8:00 PM (Pacific Time) / December 11, 2023, Monday, 10:00 AM – 12:00 PM (Beijing Time) Highlights: Background: AML accounts for up to 25% of all pediatric acute leukemia cases. Although the OS in pediatric AML has increased to approximately 70% in recent years because of enhanced risk stratification and therapeutics, approximately 30% of patients experience relapse, and 5% to 10% die because of disease complications or untoward medication effects. Hence, pediatric AML continues to represent an unmet medical need. This study investigated the safety and efficacy of the MVHO therapy in pediatric patients with refractory or relapsed AML. Methods: The study enrolled patients with recurrent or newly diagnosed AML with poor prognosis secondary to related molecular abnormalities, including NUP98 rearrangements, FUS-ERG, CBFA2T3-GLIS2, and del(7/7q), who did not achieve complete remission (CR) after first-line induction therapy (i.e., DA, DAE, DAH, MAG, and CLAG). Patients were administered the MVHO regimen, which included 1 dose of liposome mitoxantrone at 8 mg/m2; venetoclax at 300 to 350 mg/m2 once daily on days (D) 1 through 7 (with dose escalation if high tumor burden); homoharringtonine at 2 mg/m2 once daily on D1 through 7; and olverembatinib at 20-30 mg/m2 every other day on D1, 3, 5, and 7. The remission rate after 1 cycle (28-35 days, depending on hematopoietic recovery), overall response rate, recurrence rate, EFS, hematologic toxicity, and infection rate of the MVHO protocol were evaluated. A total of 18 patients were enrolled (9 boys and 9 girls), with a median (range) age of 7.3 (4 months-13 years) years, and underwent a total of 27 cycles of the MVHO therapy. Half of the patients underwent 1 cycle and the other half underwent 2 cycles. Per French-American-British (FAB) classification criteria, patients had myelodysplastic syndrome (n = 2); mixed-phenotype acute leukemia (n = 2); and AML subtypes M7 (n = 4), M2 (n = 4), M5 (n = 4), M4 (n = 1), and M0 (n = 1). Efficacy results: The median (range) follow-up time was 131 (26-256) days. The objective response rate (CR + complete remission with incomplete hematological recovery [Cri] + partial response) was 83.3%, and the remission rate (CR + Cri) after 1 cycle was 72.2%. A total of 8 patients were minimum residual disease negative. For the 6 patients with relapsed AML, the remission rate after 1 cycle was 66.7%. A total of 3 patients discontinued because of no response or disease progression, and 12 patients underwent hematopoietic stem cell transplantation, after which 1 patient relapsed. The rate of recurrence was 6.7% (1/15). The 8-month mean (± SD) EFS was 60.1% (± 19%) and the OS was 100%. Safety results: Among the 26 cycles of treatment evaluated for toxicity, there were no associated fatal infections or bleeding events. Prophylactic levofloxacin and posaconazole were administered orally (or intravenously administered anti-infection treatment in the case of infection) during myelosuppression in each cycle. No breakthrough infection was observed in 5 of 26 cycles of MVHO. There was 1 case of septic shock, and the incidence of grade ≥ 3 infection was 80.7%, which included pneumonia and bloodstream infections. Common grade 4 TRAEs were neutropenia, which was experienced by 100% of patients, and grade 4 thrombocytopenia, which was experienced by 46.1% of patients. Platelets did not decrease to below 100,000/μL after 5 of 26 cycles of MVHO treatment. Conclusions: MVHO therapy was effective and reasonably well tolerated in pediatric patients with refractory or recurrent AML, suggesting that it may comprise a suitable first-line treatment option for pediatric patients with AML. * Olverembatinib is an investigational drug that has not been approved for any indication outside the Chinese mainland About Ascentage Pharma Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK. Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. The company has built a pipeline of 9 clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors (TKIs). Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 40 Phase I/II clinical trials in the US, Australia, Europe, and China. Ascentage Pharma has been designated for multiple Major National R&D Projects, including five Major New Drug Projects, one New Drug Incubator status, four Innovative Drug Programs, and one Major Project for the Prevention and Treatment of Infectious Diseases. Olverembatinib, the company's core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company's first approved product, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug had been included into the China 2022 National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU. To date, Ascentage Pharma has obtained a total of 16 ODDs, 2 FTDs, and 2 Rare Pediatric Disease (RPD) Designations from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, MSD, and AstraZeneca. The company has built a talented team with global experience in the discovery and development of innovative drugs and is setting up its world-class commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients. 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