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TARRYTOWN, N.Y., May 1, 2025 /PRNewswire/ -- Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, today announced that data from its Phase 2 clinical trial of lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, in patients with glioblastoma will be featured during an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 30-June 3, 2025, in Chicago and online. Presentation Details: Title: "Use of lucicebtide (ST101) in glioblastoma patients by antagonism of C/EBPβ-dependent mesenchymal cell transition and immunosuppressive M2 macrophage polarization" Abstract Number: 2016 Session Type and Title: Rapid Oral Abstract – Central Nervous System Tumors Session Date and Time: Saturday, May 31, 2025, 3:00 pm – 4:30 pm CDT Presenting Author: Fabio M. Iwamoto, MD, Division of Neuro-Oncology, New York-Presbyterian/Columbia University Irving Medical Center More information can be found on the 2025 ASCO Annual Meeting website. About Lucicebtide (formerly known as ST101) Lucicebtide, a first-in-class antagonist of C/EBPβ, has completed the main portion of a Phase 2 dose expansion study in recurrent glioblastoma (rGBM) (NCT04478279). An ongoing window-of-opportunity sub-study is evaluating lucicebtide in combination with radiation and temozolomide in patients with newly diagnosed GBM (ndGBM) and as a monotherapy in patients with rGBM, with patients receiving ST101 before and after surgical resection in both cohorts. ST101 has been granted Fast Track designation for rGBM from the U.S. Food and Drug Administration (U.S. FDA) and orphan designations for glioma from the U.S. FDA and the European Commission. About Sapience Therapeutics Sapience Therapeutics, Inc. is a privately held, clinical-stage biotechnology company focused on discovering and developing peptide therapeutics to address oncogenic and immune dysregulation that drive cancer. With in-house discovery capabilities, Sapience has built a pipeline of therapeutic candidates called SPEARs™ (Stabilized Peptides Engineered Against Regulation) that disrupt intracellular protein-protein interactions, enabling targeting of transcription factors which have traditionally been considered undruggable. Sapience can also direct cargo to cell surface targets with their new class of molecule called SPARCs™ (Stabilized Peptides Against Receptors on Cancer), enabling delivery of radioisotope payloads such as α-particles to cancer cells. Sapience is advancing its lead programs, lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, and ST316, a first-in-class antagonist of β-catenin, through Phase 2 clinical trials. For more information on Sapience Therapeutics, please visit and engage with us on LinkedIn. SOURCE Sapience Therapeutics, Inc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Patent Supporting Gout and Autosomal Dominant Polycystic Kidney Disease ProgramsCALGARY, Alberta, April 28, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat gout and progressive kidney disease , is pleased to announce receipt of notification that the patent “Xanthine Oxidase Inhibitor Formulations” will be granted by the European Patent Office. The patent covers compositions and methods of formulating using XORTX’s proprietary formulations of xanthine oxidase inhibitors (“XOI”) for the treatment of health consequences of chronically high uric acid, gout, renal, cardiovascular and other diseases where aberrant purine metabolism has been implicated in disease progression. Dr. Allen Davidoff, CEO of XORTX stated, “This patent covers compositions of formulations key to XORTX’s platform technology and this issuance strengthens our intellectual property portfolio in the European Union (“EU”). Importantly, this European patent broadens protection of our first-in-class program for gout as well as supporting our autosomal dominant polycystic kidney disease (“ADPKD”) program. This patent focuses on the health consequences that arise from uric acid crystal formation due to hyperuricemia in conditions including gout, kidney stone formation, cardiovascular disease and covers novel therapeutic solutions developed specifically to address these problems. The granting of this patent provides additional breadth of patent to XORTX for commercialization and partnering opportunities throughout Europe. Including this newly granted patent, XORTX now has five granted patents in the United States (“US”) and/or EU covering compositions and uses of uric acid lowering agents to treat gout, the health consequences of hyperuricemia, and progressive kidney disease.” About Hyperuricemia, Gout and Health Consequences It is estimated that 14% of the adult population has hyperuricemia – i.e. serum uric acid levels greater than the normal range. Approximately 3% of the US population suffer from gout as a result of their hyperuricemia, though not all individuals who have hyperuricemia develop gout. The factors that contribute to hyperuricemia include genetic factors(1), dietary factors and lifestyle choices. Nucleotides are normally broken down in the blood by the xanthine oxidase (“XO”) enzyme through the purine metabolic pathway. This results in the formation of uric acid. However, if an individual experiences chronically high blood uric acid concentrations (hyperuricemia), they often experience health consequences including gout, kidney stones, diabetes, cardiovascular disease, and renal failure. Evidence that unique health consequences may arise from hyperuricemia, independent of the health consequences of gout, suggest that aberrant purine metabolism, hyperuricemia, merits an increased therapeutic focus. Lowering blood levels of uric acid in gout patients is strongly correlated with improved health outcomes. Recent groundbreaking studies by Wang et al. suggests linkage of genetic factors to the overexpression of XO(1). Recently, emerging discoveries link genetic factors to specific populations and show that higher XO expression is associated with a variety of conditions including hyperuricemia(2), general sepsis, organ failure and sepsis associated with acute respiratory distress syndrome (ARDS)(3,4), kidney dysfunction(3,4), diabetes(5), polycystic kidney disease(1,5) and kidney failure(6,7). From a mechanistic standpoint, these studies advocate for a precision medicine approach in which genetic risk variants would guide treatment decisions(1). Addressable Gout Market Opportunity In North America, approximately 3.5 million people suffer from gout due to elevated uric acid levels in blood. The therapeutic options to lower uric acid levels include three major classes of drugs: 1) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; 2) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and 3) oral XOIs that are used to inhibit the production of uric acid by the purine metabolic pathway. XOIs are the preferred first-line treatment for gout. Allopurinol is the most commonly prescribed XOI, with approximately 3 million prescriptions written per year in North America, however 3 to 5% of patients cannot tolerate allopurinol. An alternative XOI, Febuxostat, was launched in the US in 2009 with the hope of treating allopurinol intolerant patients. While Febuxostat achieved peak sales of approximately US$450 million1, it now carries a Black Box warning due to its associated risk of sudden cardiovascular death and its use has declined significantly. This decline in Febuxostat use has created an opportunity for a novel XOI to address the underlying unmet medical need which the XRx-026 program aims to fill. References: Korsmo HW, Emerging roles of xanthine oxidoreductase in chronic kidney disease, Antioxidants, June 2024Major TJ, et all, Evaluation of the diet wide contribution to serum urate levels: Met-analysis of population based cohorts, BMJ, 363, k3952, 2018Gao, Li et al., Xanthine oxidoreductase gene polymorphism are associated with high risk of sepsis and organ failure, Respir. Res, 24, 177_2023Liu H, et al., Genetic variants in XDH are associated with prognosis off gastric cancer in a Chines population, 663, 196, 2013Wang et al., Genetic susceptibility to diabetic kidney disease is linked to promoter variants of XOR,” The authors identified an expression quantitative trait loci (QTL) in the cis-acting regulatory region of the xanthine dehydrogenase, or xanthine oxidoreductase (XO), a binding site for C/EBPβ, to be associated with diabetes-induced podocyte loss in diabetic kidney disease in male mice. They concluded that certain types of alleles of a gene that controls the expression of xanthine oxidase can be over expressed in CKD, diabetic kidney disease and polycystic kidney disease.Kudo M et al., Functional Characterization of Genetic Polymorphisms Identified in the Promotor Region of the Xanthine Oxidase Gene, Drug Metab. Pharmacokinet., 25, 599, 2010Boban M, et al., Circulating purine compound, uric acid, and xanthine oxidase/dehydrogenate relationship in essential hypertension and end stage renal disease., Ren. Fail., 36, 613, 2014 About XORTX Therapeutics Inc. XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our lead program XRx-026 program for the treatment of gout; 2) XRx-008 program for ADPKD; and 3) XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections. In addition, the Company is developing XRx-225, a pre-clinical stage program for Type 2 diabetic nephropathy. XORTX is working to advance products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, we are dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases. Additional information on XORTX is available at www.xortx.com. For more information, please contact: Allen Davidoff, CEONick Rigopulos, Director of Communicationsadavidoff@xortx.com or +1 403 455 7727nick@alpineequityadv.com or +1 617 901 0785 Neither the TSX Venture Exchange nor Nasdaq has approved or disapproved the contents of this news release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein. Forward Looking Statements This press release contains express or implied forward-looking statements pursuant to applicable securities laws. These forward-looking statements include, but are not limited to, the Company's beliefs, plans, goals, objectives, expectations, assumptions, estimates, intentions, future performance, other statements that are not historical facts and statements identified by words such as "expects", "anticipates", "intends", "plans", "believes", "seeks", "estimates" or words of similar meaning. These forward-looking statements and their implications are based on the current expectations of the management of XORTX only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks, uncertainties, and other factors include, but are not limited to, our ability to obtain additional financing; the accuracy of our estimates regarding expenses, future revenues and capital requirements; the success and timing of our preclinical studies and clinical trials; the performance of third-party manufacturers and contract research organizations; our plans to develop and commercialize our product candidates; our plans to advance research in other kidney disease applications; and, our ability to obtain and maintain intellectual property protection for our product candidates. Except as otherwise required by applicable law and stock exchange rules, XORTX undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting XORTX is contained under the heading “Risk Factors” in XORTX’s Annual Report on Form 20-F filed with the SEC, which is available on the SEC's website, www.sec.gov (including any documents forming a part thereof or incorporated by reference therein), as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada, which are available on www.sedarplus.ca. 1 Source: Takeda Pharmaceutical Company 2018 Annual Report.

TARRYTOWN, N.Y., March 26, 2025 /PRNewswire/ -- Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, today announced four poster presentations at the American Association for Cancer Research (AACR) Annual Meeting 2025, taking place April 25-30, 2025, in Chicago, Illinois. Sapience will present non-clinical results from both of its clinical programs, lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, and ST316, a first-in-class antagonist of β-catenin and its co-activator, BCL9, as well as results from its preclinical Fra1 antagonist peptide (FraAP) program, a first-in-class antagonist of the activator protein 1 (AP-1) complex. "We are thrilled to be presenting a compendium of non-clinical data supportive of our clinical and preclinical SPEARs™ programs, including lucicebtide, ST316, and our FraAP program, at the upcoming AACR Annual Meeting," said Jim Rotolo, Ph.D., Sapience's Chief Scientific Officer. "Results from our lucicebtide and ST316 studies further elucidate their respective mechanisms of action and highlight their potential for combination strategies. Additionally, preclinical data demonstrate the potential of our FraAP program to induce anti-cancer activity by targeting the AP-1 complex, particularly in head and neck squamous cell carcinoma." Poster Presentation Details and Abstract Highlights: Title: "The clinical C/EBPβ antagonist peptide lucicebtide synergizes with molecularly targeted therapies in GBM" Session Title: Targeted Therapies and Combinations 1 Location: Poster Section 34 Abstract Number: 839 Date and Time: Sunday, April 27, 2025, 2:00 pm – 5:00 pm CDT Abstract Summary: In non-clinical studies, the mechanism of lucicebtide, which has previously demonstrated antitumor activity via direct cell death and immune activation within the tumor microenvironment, enabled a synthetic lethal effect with molecularly targeted therapeutics, such as EGFR inhibitors, that are typically not effective when used as monotherapies for the treatment of glioblastoma (GBM). Transcriptional analysis utilizing GBM genetics and signatures of C/EBPβ activity were performed to identify potential target populations likely to benefit from lucicebtide combinations. Title: "FraAP, a peptide antagonist against the activator protein 1 transcription factor complex, demonstrates cancer cell cytotoxicity and reduced invasion in vitro and tumor regression in vivo in HNSCC models" Session Title: Gene Regulation and Transcription Factors 2 Location: Poster Section 10 Abstract Number: 1415 Date and Time: Monday, April 28, 2025, 9:00 am – 12:00 pm CDT Abstract Summary: FraAP selectively binds to and inhibits key oncogenic drivers of the AP-1 transcription factor complex, resulting in the suppression of AP-1 driven pathways required for cancer cell cycle progression, proliferation, and invasion and promotes apoptosis in preclinical studies. Further, in a head and neck squamous cell carcinoma (HNSCC) xenograft model, administration of FraAP results in significant tumor growth inhibition, further demonstrating the anti-cancer potential of targeting the AP-1 complex. Title: "An ex vivo organoid study to identify biomarkers of sensitivity to ST316, a clinical-stage β-catenin antagonist peptide" Session Title: Biomarkers and Molecular Targets for Prevention Location: Poster Section 43 Abstract Number: 2361 Date and Time: Monday, April 28, 2025, 9:00 am – 12:00 pm CDT Abstract Summary: Analysis of 60 patient-derived tumor organoids revealed that tumors with TP53 WT and mutations in the MAPK pathway significantly correlate with sensitivity to ST316, a β-catenin antagonist peptide. Further, in non-clinical models, combining ST316 with encorafenib, a BRAF inhibitor, showed synergistic effects in BRAFV600E-mutant colorectal cancer (CRC) organoids, demonstrating a promising combination approach in this poor prognosis and underserved patient population. Across studies, findings support rational combinations of ST316 with clinically relevant MAPK inhibitors. Title: "Reprogramming of MDSCs by ST316, a clinical peptide antagonist of β-Catenin/BCL9, enhances anti-tumor immuno- and chemotherapy" Session Title: Modifiers of Tumor Microenvironment Location: Poster Section 30 Abstract Number: 3275 Date and Time: Monday, April 28, 2025, 2:00 pm – 5:00 pm CDT Abstract Summary: Results reveal a novel role for Wnt/β-catenin signaling in myeloid-derived suppressor cell (MDSC) biology, a key mediator of immune-suppression, whereby antagonism of β-catenin with ST316 leads to suppression of MDSC expansion. In combination studies, ST316 significantly enhances the anti-tumor efficacy of anti-PD-1 immunotherapy and standard-of-care chemotherapies for advanced CRC such as FOLFIRI. While anti-PD-1 and FOLFIRI alone each resulted in expansion of immunosuppressive MDSCs, combination with ST316 prevented this expansion. These data suggest that therapeutically targeting the β-catenin/BCL9 interaction may be an effective strategy to enhance both chemotherapy and immunotherapy in Wnt-driven tumors that respond poorly to monotherapy. More information can be found on the AACR Annual Meeting 2025 website. About Lucicebtide (formerly known as ST101) Lucicebtide, a first-in-class antagonist of C/EBPβ, has completed the main portion of a Phase 2 dose expansion study in recurrent GBM (rGBM) (NCT04478279). An ongoing window-of-opportunity sub-study is evaluating lucicebtide in combination with radiation and temozolomide in patients with newly diagnosed GBM (ndGBM) and as a monotherapy in patients with rGBM, with patients receiving ST101 before and after surgical resection in both cohorts. ST101 has been granted Fast Track designation for rGBM from the U.S. Food and Drug Administration (U.S. FDA) and orphan designations for glioma from the U.S. FDA and the European Commission. About ST316 ST316 is a first-in-class antagonist of the interaction between β-catenin and its co-activator, BCL9, a complex responsible for driving oncogene expression and immune exclusion in multiple cancers where aberrant Wnt/β-catenin pathway signaling is observed. ST316 exposure in cancer cells prevents BCL9-driven nuclear localization of β-catenin and formation of the enhanceosome protein complex. Disruption of this interaction selectively suppresses the transcription of oncogenic Wnt target genes that regulate proliferation, migration, invasion and the metastatic potential of tumor cells, as well as genes that regulate the immunosuppression of the tumor microenvironment. ST316 creates a pro-immune tumor microenvironment and in preclinical models has shown to be synergistic with checkpoint inhibition. Due to its selectivity and downstream modulation of the Wnt/β-catenin pathway, ST316 presents an opportunity to safely and effectively target Wnt/β-catenin driven pathologies without the toxicities previously seen with other Wnt pathway agents. ST316-101 (NCT05848739) is a first-in-human, open-label, Phase 1-2 Dose Escalation and Expansion study designed to determine the safety, tolerability, PK, PD and early efficacy of ST316. The Phase 1 Dose Escalation portion of the study tested various dose levels of ST316 in patients with select advanced solid tumors that are known to harbor abnormalities of the Wnt/β-catenin signaling pathway, including colorectal cancer (CRC). ST316 is currently being tested in the Phase 2 portion of the study in CRC patients in combination with relevant standards of care and in multiple lines of treatment. The U.S. Food and Drug Administration (U.S. FDA) has granted Orphan Drug Designation to ST316 for the treatment of familial adenomatous polyposis (FAP). About Sapience Therapeutics Sapience Therapeutics, Inc. is a privately held, clinical-stage biotechnology company focused on discovering and developing peptide therapeutics to address oncogenic and immune dysregulation that drive cancer. With in-house discovery capabilities, Sapience has built a pipeline of therapeutic candidates called SPEARs™ (Stabilized Peptides Engineered Against Regulation) that disrupt intracellular protein-protein interactions, enabling targeting of transcription factors which have traditionally been considered undruggable. Sapience can also direct cargo to cell surface targets with their new class of molecule called SPARCs™ (Stabilized Peptides Against Receptors on Cancer), enabling delivery of radioisotope payloads such as α-particles to cancer cells. Sapience is advancing its lead programs, lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, and ST316, a first-in-class antagonist of β-catenin, through Phase 2 clinical trials. For more information on Sapience Therapeutics, please visit and engage with us on LinkedIn. Media and Investor Contact: Melissa Forst/Brendan Burns Argot Partners 212-600-1902 [email protected] SOURCE Sapience Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED