Allopurinol

XORTX to focus on late stage allopurinol intolerant gout program; discussion with FDA planned for first half 2025 regarding NDA filingCALGARY, Alberta, Jan. 06, 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 progressive kidney disease, announces the launch of a new late stage program to treat gout. The new drug development program - XRx-026 - will focus on the treatment of individuals who have gout and are intolerant to allopurinol. With the clinical development of XORLOTM 1 having advanced sufficiently, including completion of a pivotal program, XORTX is initiating discussions for the XRx-026 program with the US Food and Drug Administration (“FDA”) regarding preparation of a New Drug Application (“NDA”). Pending FDA feedback, the Company will also seek FDA orphan drug designation (“ODD”) and NDA marketing approval for the XRx-026 program. Previously, oxypurinol was granted ODD for allopurinol intolerant gout. Dr. Allen Davidoff commented, “Oxypurinol has been demonstrated to be safe and effective in clinical studies focused on the treatment of individuals with gout who are intolerant to allopurinol. Intolerance to allopurinol remains an important issue for many patients and physicians and the XRx-026 program has demonstrated the potential to address this unmet medical need. XORTX will consult with the FDA during the first half of 2025, regarding requirements to file a NDA.” About Hyperuricemia, Gout and Health Consequences The breakdown of nucleotides in the blood occurs through purine metabolism and results in the formation of uric acid by the xanthine oxidase enzyme. However, chronically high blood uric acid concentrations (hyperuricemia) have been associated with health consequences including gout, kidney stones, diabetes, cardiovascular disease, and renal failure. Worldwide, approximately 14% of individuals have hyperuricemia and an estimated 1 to 2% have gout. Lowering blood levels of uric acid in gout patients is strongly correlated with improved health outcomes. 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: (i) oral uricosurics that are used to decrease the reabsorption of uric acid by the kidney; (ii) intravenous uricase enzymes that are used to metabolize uric acid in the blood for excretion; and (iii) oral xanthine oxidase inhibitors (“XOIs”) that are used to inhibit the production of uric acid. 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, launched in the US in 2009 with the hope of treating allopurinol intolerant patients, however while Febuxostat achieved peak sales of approximately US$450 million2, 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. About XORTX Therapeutics Inc. XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our lead, XRx-008 program for ADPKD; 2) our XRx-026 program for the treatment of allopurinol intolerant gout; and 3) our secondary program in XRx-101 for acute kidney and other acute organ injury associated with Coronavirus / COVID-19 infection. In addition, XRx-225 is a pre-clinical stage program for Type 2 Diabetic Nephropathy. XORTX is working to advance its clinical development stage 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 to improve the quality of life and health of kidney disease patients and individuals with gout. 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 XORLOTM is XORTX’s proprietary formulation of oxypurinol, that has granted US and EU patents. 2 Source: Takeda Pharmaceutical Company 2018 Annual Report.

Strong commercial performance and significant pipeline milestones support sustained growth and innovation SAN FRANSISCO and SUZHOU, China, Aug. 28, 2024 /PRNewswire/ -- Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, announces its 2024 interim results and major business updates. Dr. Michael Yu, Founder, Chairman and CEO of Innovent, stated: "For the first half of 2024, our company's strategy of sustainable growth and global innovation continue executing very well: we achieved strong revenue growth, improved operational efficiency in all areas, and reached significant milestones in our late-stage pipeline to support our sustained growth. We also reported promising data readouts from early-stage assets, reinforcing our confidence in achieving global innovation and contributing to new long-term opportunities. The successful first half of 2024 has laid a solid foundation for achieving our full-year's growth. With strong commercial and financial execution, a high-value late-stage pipeline, and disciplined investments in next-generation innovation, we are well-positioned to deliver long-term value for patients, employees, shareholders and society." Enhanced operational efficiency and strong financial performance S trong revenue growth momentum: total revenue was RMB3,952.3 million in the first half of 2024, with a strong year-over-year growth of 46.3%, reflecting robust demand for our innovative portfolio and the advantage of our sustainable business model. Significantly improved financial performance: EBITDA Loss was significantly reduced, driven by strong revenue growth, enhanced operational efficiency and notable financial improvement. The gross profit margin of total revenue was 84.1%, a year-over-year increase of 1.8 percentage points The selling and marketing expenses of product revenue was 48.6%, a year-over-year decrease of 5.9 percentage points The administration and expenses of total revenue was 5.2%, a year-over-year decrease of 4.9 percentage points R&D expenses were RMB1,293.9 million; cash and short-term financial assets were RMB10,112.3 million, or approximately over USD1.4 billion, which enables us to focus on the long-term sustainable development EBITDA loss was RMB160.8 million, a notable year-over-year decrease of 39.9% Strong product revenue growth; preparing for CVM commercialization Product sales revenue reached RMB3 ,811.4 million in the first half of 2024, a strong year-over-year growth of 55.1%. Expansion of commercial portfolio into new approved products, new indications and broader NRDL coverage [1] , [2] and patient access: Eleven approved products: TYVYT®, BYVASDA®, SULINNO®, HALPRYZA®, PEMAZYRE®, Olverembatinib, CYRAMZA®, Retsevmo®, FUCASO® , SINTBILO® and DUPERT® (new product, KRAS G12C inhibitor) . TYVYT® and PEMAZYRE® were newly approved in the Macau market. TYVYT® and BYVASDA® were included in the NRDL for the treatment of EGFR-mutated NSCLC. Solidify oncology leadership; in active preparation for new commercial opportunities in general biomedicine. Oncology: we strengthened our leadership with a robust product portfolio, including TYVYT® (sintilimab injection), and expanded out oncology business with a 10th product, a ROS1 inhibitor, set for approval in the second half of 2024. General biomedicines: Following the approval of the first CVM product SINTBILO® (tafolecimab injection) in 2023, we have successfully submitted three new drug applications (NDA)—two for mazdutide, targeting obesity/overweight population and T2D, and one for IBI311 (IGF-1R) for thyroid eye disease (TED). As a key strategic priority, we are steadily building our commercialization capabilities in the CVM field with systematic approaches, aiming to unlock substantial commercial opportunities and drive sustainable growth. Material innovation delivery supports strategic goals 7 new assets are in NDA review or pivotal registrational clinical trials, and 18 assets are in early-phase clinical studies Substantial milestones delivered for key late-stage assets Encouraging progress in the next wave innovation of "IO+ADC" TYVYT® (sintilimab): submitted an NDA for its eighth indication, 2L endometrial cancer (EMC). New registrational clinical trials for neoadjuvant therapy in colon cancer and perioperative therapy in NSCLC have been initiated. Additionally, we are exploring the potential of combination therapies through multiple collaborations with novel modalities. IBI310 (CTLA-4 )：initiated a Phase 3 clinical trial for IBI310 in combination with sintilimab as neoadjuvant therapy in treating colon cancer. IBI343 (CLDN18.2 ADC)：Phase 1b positive data readout in GC and a Phase 3 trial is in preparation. Accelerating new launch momentum in general biomedicine to unlock significant opportunities Mazdutide (GLP-1R/GCGR) : first NDA for weight management in obese or overweight populations and second NDA for T2D treatment, both under NMPA review. We plan to develop new indications, including adolescent obesity, metabolic dysfunction-associated steatohepatitis (MASH), obstructive sleep apnea (OSA), and heart failure with preserved ejection fraction (HFpEF). Teprotumumab (IGF-1R) : the NDA for TED is under NMPA review. With a longstanding lack of innovative TED treatments in China, Teprotumumab is set to be a transformative therapy for this significant unmet need once approved. Picankibart (IL-23p19) : the only IL-23p19 that reported over 80% subjects achieving ≥90% improvement in Psoriasis and Severity Index (PASI90) in 16 weeks of treatment, along with strong long-term skin clearance maintenance and quarterly dosing interval advantage. We plan to submit an NDA in the second half of 2024. IBI128 (XOI) : potential best-in-class XOI for the treatment of hyperuricemia in gout patients. It is currently undergoing overseas Phase 3 clinical trials overseas with our partner LG Chem, and we have completed patient enrollment for a Phase 2 clinical trial in China. IBI302 (VEGF/C) : Phase 3 study initiated for the treatment of nAMD, following stable and robust visual benefit observed with an extended dosing interval and potential macular atrophy inhibition in two Phase 2 studies. Abundant early-stage pipeline to support long-term growth and global ambition Encouraging data readouts from multiple oncology assets in Phase 1 studies, eyeing most difficult-to-treat cancers IBI363 (PD-1/IL-2 α -bias ) : preliminary positive signals in multiple IO-failed/cold tumor types; further investigations across different rumor types are ongoing; a Phase 2 clinical trial in the U.S. has been initiated. IBI343 (CLDN18.2 ADC) : encouraging positive signal in pancreatic cancer, with FDA fast track designation granted. Plans are underway for a clinical trial in the U.S. IBI389 (CLDN18.2/CD3) : encouraging and differentiated signals in GC and PDAC in Phase 1 studies; Phase 1b study is continuing. Multiple programs ongoing including IBI3003 (GPRC5D/BCMA/CD3), IBI115 (DLL3/CD3); IBI3004 (DR5/CEA); IBI3001 (EGFR/B7H3 ADC), IBI130 (TROP2 ADC), IBI133 (HER3 ADC) Develop next-generation general biomedicine programs to improve chronic disease treatment IBI3016 （ AGT siRNA ） : a new-generation siRNA drug candidate, entered into a Phase 1 clinical trial for hypertension. IBI355 (CD40L), IBI356 (OX40L) and IBI3002 (IL-4R α / TSLP) : innovative autoimmune molecules entered into first-in-human studies to explore other unmet medical needs in various types of autoimmune diseases. IBI324 (VEGF-A/ANG-2) and IBI333 (VEGF-C/VEGF-A) : both are in the Phase 1 stage to explore the potential differentiation clinical values versus existing therapy. Research innovation published in high-impact scientific journals and medical conferences, such as: AACR, ASCO, ESMO GI and ESMO plenary for oncology pipeline innovation, including 10+ oral presentations ADA, APAO, ICE, CSE for general biomedicine pipeline material progress, such as mazdutide and teprotumumab Facilitates and Manufacturing capacity adhering to high-standard quality: Shanghai R&D center (medical) is newly operational in August 2024 First manufacturing site: 60,000L antibody production capacity and ADC production lines in operation Second manufacturing site: first phase of 80,000L completed construction to secure CDMO business Devoted to responsible business practices and enhancing ESG management practices We remain committed to sustainable development, corporate responsibility and ethical business practices. We newly launched our ESG website to enhance our efforts in sustainability, corporate responsibility and ethical business conduct. The new platform highlights our initiatives, policies and performance in key ESG areas, including "Excellent Governance", "Enjoying Good Health", "Ensuring High-Quality Products", "Empowering Employees", and "Embracing Ecology". Innovent is graded 'A' level in MSCI ESG rating, ranking at the forefront of the biotechnology industry. About Innovent Innovent is a leading biopharmaceutical company founded in 2011 with the mission to empower patients worldwide with affordable, high-quality biopharmaceuticals. The company discovers, develops, manufactures and commercializes innovative medicines that treat some of the most intractable diseases. Its pioneering therapies treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has 11 products in the market, 3 new drug applications under the NMPA review, 4 assets in Phase III or pivotal clinical trials and 18 more molecules in early clinical stage. Innovent partners with over 30 global healthcare leaders, including Eli Lilly, Roche, Sanofi, Adimab, Incyte and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit , or follow Innovent on Facebook and LinkedIn. Statement: Innovent does not recommend the use of any unapproved drug (s)/indication (s). Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent Biologics ("Innovent"), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or is otherwise inaccurate. SOURCE Innovent Biologics