This trial studies the effect of pegloticase in reducing uric acid levels in patients with hyperuricemia (high blood levels of uric acid) caused by tumor lysis syndrome. Tumor lysis syndrome occurs when the breakdown products of cancer cells, such as uric acid, enter the blood stream. High levels of uric acid in blood may cause kidney damage and reduce kidney function. The goal of this trial is to learn if pegloticase may lower uric acid levels in blood when given to cancer patients with hyperuricemia caused by tumor lysis syndrome.

Start Date05 Apr 2022

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NL-OMON27662

/ SuspendedNot Applicable

Treatment of severe gout patients with rasburicase

Start Date03 May 2021

Sponsor / Collaborator-

100 Clinical Results associated with Rasburicase

100 Translational Medicine associated with Rasburicase

100 Patents (Medical) associated with Rasburicase

575

Literatures (Medical) associated with Rasburicase

03 Mar 2025·Journal of Applied Laboratory Medicine

Comparing Refrigeration to Immediate Room Temperature Testing for Uric Acid Monitoring in Rasburicase-Treated Patients

Article

Author: Wilson, Jeffrey ; Zuromski, Lauren M ; Lin, Leo ; Nguyen Sorenson, Anh ; Filtz, Michael ; Errigo, Roscoe ; Young, Brittany A

Abstract:

Background:

Rasburicase retains activity at room temperature (RT), so specimens collected for uric acid-level monitoring require cooling protocols. Our objective was to determine if we could ease these preanalytical requirements to improve compliance while maintaining accuracy.

Methods:

Fifty pairs of specimens were transported and stored either on ice or at RT. All were tested at 3 time points postcollection: immediately upon arrival to the laboratory (approximately 45 min), 90, and 135 min.

Results:

Uric acid concentrations are not clinically significantly different in RT or iced specimens, as long as specimens are tested within approximately 45 min postcollection. There was a negative bias in uric acid levels in a subset of specimens if they were held at RT and tested at 90 min (−9.1%) and 135 min (−17.5%). Specimens tested within 2 rasburicase half-lives postinfusion have an additional 24% decrease in uric acid levels if kept at RT for 90 min. Specimens from patients given a 6 mg dose had an 18% decrease in uric acid concentration compared to a 3 mg dose.

Conclusions:

Laboratories that can test uric acid levels rapidly after specimen collection may be able to validate alternative preanalytical methods to transporting and testing on ice.

01 Mar 2025·JOINT BONE SPINE

Optimizing gout treatment: A comprehensive review of current and emerging uricosurics

Review

Author: Chaiyakunapruk, Nathorn ; Kaufmann, Dan ; Schlesinger, Naomi

Gout is the most common inflammatory arthritis, affecting approximately 5.1% of adults in the United States (US) population. Gout is a metabolic and autoinflammatory disease. Elevated uric acid pools lead to the precipitation of monosodium urate (MSU) crystals in and around joints, as well as other tissues, and the subsequent autoinflammatory response. Since elevated serum urate (SU) levels (hyperuricemia) correspond with gout severity, urate-lowering therapies (ULTs) are the cornerstone of gout treatment. ULTs include xanthine oxidoreductase inhibitors, uricosurics, less commonly used in the US but widely used in Europe and Asia, including benzbromarone, dotinurad, and probenecid (the only US Food and Drug Administration (FDA) approved uricosuric in the US), and uricases, including rasburicase and pegloticase (available only in the US). Over 90% of the daily load of uric acid filtered by the kidneys is reabsorbed through renal transporters. These urate transporters include uric acid transporter 1 (URAT1), glucose transporter 9, and organic anion transporters 1, 3, and 4 (OAT1, OAT3, OAT4). They are the target of approved and in-the-pipeline uricosurics. Any drug that increases renal excretion of uric acid, independently of the mechanism through which it exerts its effect, may be considered a uricosuric drug. This review discusses drugs that increase renal excretion of uric acid, either approved or in development, as well as off-label drugs with uricosuric properties.

04 Feb 2025·ACS Nano

Lipid-Rapamycin Nanovaccines Overcome the Antidrug Antibody Barrier in Biologic Therapies

Article

Author: Liu, Hongzhuo ; Jin, Zheng ; An, Ni ; Zhong, Mingyuan ; Yasen, Guzailinuer ; Li, Ximu ; Wang, Yongjun ; Ma, Mengyao ; He, Zhonggui ; Yao, Xiaoxuan

Antidrug antibodies (ADAs) against biologics present a major challenge for sustained biotherapy, including enzyme replacement therapies and adeno-associated virus (AAV) gene therapies. These antibodies arise from undesirable immune responses, leading to altered pharmacokinetics, reduced efficacy, and adverse reactions. In this study, we introduced a rationally designed lipid-rapamycin (Rapa)-based nanovaccine to restore immune tolerance to biologics and overcome drug resistance. The nanovaccine significantly decreased ADA responses when used in a tolerogenic regimen with keyhole limpet hemocyanin (KLH), uricase, pegylated uricase, and AAV8 vector gene therapy. This approach facilitated three rechallenges with pegylated uricase after a 5 week rest from the nanovaccine, thereby enhancing its urate-lowering efficacy. Furthermore, the nanovaccine allowed for the successful intravenous readministration of AAV8 vector expressing secreted embryonic alkaline phosphatase (AAV8-SEAP), achieving sustained viral DNA and transcript levels in target tissues. The nanovaccine prompted antigen-presenting cells (APCs) in the liver to exhibit dynamic changes in CD80, CD86, MHCII, and PD-L1, which promoted the development of immunoregulatory T cells in response to biologic challenges. Notably, the nanovaccine exerted a minimal impact on CD8⁺ T cells, natural killer (NK) cells, and NK T cells, preserving the body's normal immune response to pathogens and tumors. Overall, the universal nanovaccine addressed biologic resistance by mitigating ADA-related issues, thereby enabling a prolonged therapeutic efficacy for antibodies, proteins, and gene therapies.

News (Medical) associated with Rasburicase

11 Dec 2024

·www.globenewswire.com

Prelude Therapeutics Presents Preliminary Results of Phase 1 Dose-escalation Study of PRT2527 as Monotherapy and in Combination with Zanubrutinib in Patients with Relapsed/Refractory Lymphoid Malignancies

• PRT2527 demonstrated activity across a range of relapsed/refractory lymphoid malignancies, including patients who received prior CAR-T therapy • Prelude plans to seek a partner for future development of PRT2527 in hematologic malignancies WILMINGTON, Del., Dec. 11, 2024 (GLOBE NEWSWIRE) -- Prelude Therapeutics Incorporated (Nasdaq: PRLD) (“Prelude” or the “Company”), a clinical-stage precision oncology company, today announced the presentation of the first interim clinical data from its ongoing open-label, dose-escalation trial of PRT2527, a potent and highly selective CDK9 inhibitor, as monotherapy and in combination with zanubrutinib in patients with relapsed/refractory lymphoid malignancies. The data were presented at a poster session of the 66th American Society of Hematology Annual Meeting in San Diego, California. The study investigators reported on the 46 patients that were enrolled, treated, and safety evaluable as of September 17, 2024. PRT2527 was generally well-tolerated through 4 dosing cohorts as monotherapy and 3 dosing cohorts in combination with zanubrutinib. PRT2527 monotherapy and in combination with zanubrutinib demonstrated an acceptable safety profile with evidence of preliminary activity in patients with relapsed/refractory lymphoid malignancies, including patients who received prior CAR-T therapy. “CDK9 has long been considered a potential therapeutic approach for treating hematologic malignancies and a highly selective CDK9 inhibitor was sought to minimize off target toxicity,” stated Jane Huang, M.D., President and Chief Medical Officer of Prelude. “We are encouraged by the results demonstrated to date by PRT2527 both as a monotherapy and particularly in combination with zanubrutinib resulting in an overall response rate of 38.5% including two patients with aggressive lymphomas who had received prior CAR-T therapy. These results represent a positive step for CDK9 inhibition as a possible future therapeutic approach for patients with aggressive hematologic cancers with limited treatment options.” PRT2527 Interim Phase 1 ResultsPRT2527 is an investigational, potent and highly selective CDK9 inhibitor being evaluated in select relapsed/refractory (R/R) hematologic malignancies as monotherapy and in combination with zanubrutinib. As of the cutoff date, 46 patients with relapsed/refractory lymphoid malignancies were treated with PRT2527. 29 patients were treated once weekly via intravenous infusion at four dose levels of PRT2527 monotherapy (9 mg/m2, 15 mg/m2, 18 mg/m2, 24 mg/m2) and 17 patients were treated once weekly at three dose levels of PRT2527 (9 mg/m2, 15 mg/m2, 18 mg/m2) in combination with zanubrutinib administered orally starting on C1D1 at 320 mg daily or 160 mg BID. Initial Safety DataThe most frequent treatment emergent adverse events (TEAEs) observed in ≥20% of patients were neutropenia (48%) and nausea (33%), and the most frequent grade ≥3 TEAEs (≥10% of patients) were neutropenia (46%) and anemia (11%). Five patients discontinued treatment due to TEAEs in the monotherapy cohort; 3 TEAEs in 1 patient were treatment related: grade 3 hypotension, grade 3 diarrhea, and grade 4 neutropenia (n=1 each). No TEAEs led to treatment discontinuation in the combination therapy cohort. PRT2527 dose interruptions due to TEAEs occurred in 17 patients (11 monotherapy; 6 combination therapy). Most dose interruptions were due to neutropenia and were managed with growth factor support. One DLT of grade 3 tumor lysis syndrome (TLS) occurred in a patient with primary cutaneous peripheral T cell lymphoma who had extensive disease at the 24 mg/m2 monotherapy dose level and did not receive ramp-up dosing. TLS was managed with rasburicase and IV fluids and resolved. The patient was able to resume study treatment as planned. No DLTs were observed in the combination therapy cohort. Dose level 3 (18 mg/m2) was selected for dose confirmation for monotherapy and in combination with zanubrutinib due to higher rates of grade 3/4 neutropenia and of dose interruptions and reductions in the 24 mg/m2 dose level. Analysis of Initial Clinical ActivityOf the 23 efficacy evaluable patients in the monotherapy cohort, complete responses (CRs) were observed in 1 patient (DLBCL) and 3 partial responses observed (TCL), with an overall response rate (ORR) of 17.4% (4 of 23). Of the 13 patients in the combination cohort who were evaluable for efficacy, complete responses (CRs) were observed in 3 patients (2 DLBCL, 1 MCL) and 2 partial responses (PRs) observed (DLBCL and CLL) with an overall response rate (ORR) of 38.5% (5 of 13). The above-noted presentation can be found at Publications - Prelude Therapeutics (preludetx.com). “We believe the clinical data presented today with PRT2527 confirm our hypothesis that a highly selective and potent inhibitor of CDK9 has the potential to offer meaningful clinical activity for patients with hematologic malignancies, while avoiding the off-target toxicities observed with less selective agents,” stated Kris Vaddi, Ph.D., Chief Executive Officer of Prelude. “However, given the significant progress and potential of our SMARCA degrader programs that are currently advancing in the clinic, along with our productive discovery organization, we plan to focus our resources towards the continued advancement of those programs. As a result, we will only advance the CDK9 program with a partner beyond completion of the current ongoing Phase 1 study.” About Prelude Therapeutics Prelude Therapeutics is a leading precision oncology company developing innovative medicines in areas of high unmet need for cancer patients. Our pipeline is comprised of several novel drug candidates including first-in-class, highly selective IV and oral SMARCA2 degraders, and a potentially best-in-class CDK9 inhibitor. We are also leveraging our expertise in targeted protein degradation to discover, develop and commercialize next generation degrader antibody conjugates (Precision ADCs) with partners. We are on a mission to extend the promise of precision medicine to every cancer patient in need. For more information, visit preludetx.com. Cautionary Note Regarding 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 discovery, preclinical and clinical development activities for Prelude’s product candidates, the potential safety, efficacy, benefits and addressable market for Prelude’s product candidates, and clinical trial results for Prelude’s product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The words “believes,” “anticipates,” “estimates,” “plans,” “expects,” “intends,” “may,” “could,” “should,” “potential,” “likely,” “projects,” “continue,” “will,” “schedule,” and “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are predictions based on the Company’s current expectations and projections about future events and various assumptions. Although Prelude believes that the expectations reflected in such forward-looking statements are reasonable, Prelude 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 Prelude's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to Prelude's ability to advance its product candidates, the receipt and timing of potential regulatory designations, approvals and commercialization of product candidates, clinical trial sites and our ability to enroll eligible patients, supply chain and manufacturing facilities, Prelude’s ability to maintain and recognize the benefits of certain designations received by product candidates, the timing and results of preclinical and clinical trials, Prelude's ability to fund development activities and achieve development goals, Prelude's ability to protect intellectual property, and other risks and uncertainties described under the heading "Risk Factors" in Prelude’s Annual Report on Form 10-K for the year ended December 31, 2023, its Quarterly Reports on Form 10-Q and other documents that Prelude 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 Prelude undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof, except as may be required by law.  Investor Contact: Robert A. Doody Jr.Senior Vice President, Investor Relations 484.639.7235rdoody@preludetx.com

Clinical ResultPhase 1ASHCell TherapyImmunotherapy

24 Aug 2023

·www.globenewswire.com

Aprea Therapeutics Appoints Dr. Jean-Pierre Bizzari to its Board of Directors and Names Dr. Richard Peters as Chairman

DOYLESTOWN, Pa., Aug. 24, 2023 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE) (“Aprea”, or the “Company”), a clinical stage biopharmaceutical company focused on precision oncology through synthetic lethality, today announced it has appointed Dr. Jean-Pierre Bizzari to its Board of Directors and is naming Dr. Richard Peters as its new Chairman of the Board. Dr. Bizzari will replace Mr. Christian Schade, who is stepping down from the Board. “We warmly welcome Dr. Jean-Pierre Bizzari to our Board of Directors. Jean-Pierre is an esteemed industry thought leader and was the Group Head Clinical Development Oncology at Celgene where he was responsible for developing some of the most significant and successful oncology therapeutics,” said Dr. Oren Gilad, President and Chief Executive Officer of Aprea. “Jean-Pierre joins our Board as Chris Schade retires from his role as Chairman and member of the Board to focus on his other commitments. Dr. Richard Peters has served as independent Director of Aprea since June 2020 and Chair of the Compensation Committee since November 2020. Dr. Peters will now also assume the role of Chairman, and we welcome his leadership as he guides our diverse and experienced Board.” Dr. Jean-Pierre Bizzari joins the Aprea Board having led a remarkable and distinguished career in oncology. He has been responsible for numerous global approvals of several billion-dollar therapies, including: Fotomustine, Taxotere, Revlimid, Abraxane, Vidaza, PTCL, Eloxatin, CPT-11, Gliadel, Rasburicase, and Pomalidomide to name a few. He has been involved in acquisition and licensing agreements with several major pharmaceutical companies. Dr. Bizzari is a member and leader on many scientific committees and is currently a member of the Scientific Advisory Board for the National Cancer Institute in France; a Board member of the European Organization of Research and Treatment of Cancer (EORTC) and Chairman of the EORTC New Drug Advisory Committee. He currently is on the Boards of Halozyme, ADC Therapeutics, NETRIS Pharma, and Oxford BioTherapeutics. Most recently, Dr. Bizzari was the Group Head Clinical Development Oncology at Celgene. He led global clinical development conducting over 25 Phase 3 trials, was responsible for global operations of over 950 people and was chairman of the Hematology Oncology development committee. Prior to Celgene, Dr. Bizzari was the VP of clinical development oncology at Sanofi-Aventis where he was responsible for the worldwide clinical development and approvals. He also served as VP of clinical development oncology at Rhone-Poulenc Rorer where he shepherded a deep pipeline of oncology candidates. Dr. Bizzari holds a degree in mathematics, and completed his medical studies in Nice, France. He completed his residency in oncology at Pitie Salpetriere Hospital in Paris. Dr. Bizzari said, “I am excited to join this dedicated Board, and look forward to contributing to the success of their very promising therapeutics at the center of synthetic lethality and DNA Damage Repair drug development.” Mr. Christian Schade noted, “It has been an honor to serve on this dynamic Board. I leave knowing that the Board and Company are in good hands, and that everyone involved is committed to realizing the tremendous potential of this company.” Dr. Richard Peters added, “I thank Chris for his years of service to the Board and astute leadership as we successfully navigated the company through several challenges. I look forward to working closely with our Board and terrific management team as we move towards several meaningful Company milestones.” About ApreaAprea Therapeutics, Inc. is a clinical stage biopharmaceutical company headquartered in Doylestown, Pennsylvania, focused on precision oncology through synthetic lethality. The Company’s lead program is ATRN-119, a clinical-stage small molecule ATR inhibitor being developed for solid tumor indications. Our WEE1 inhibitor is being advanced to IND submission. For more information, please visit the company website at www.aprea.com. The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Forward-Looking Statement Certain information contained in this press release includes “forward-looking statements”, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended related to our study analyses, clinical trials, regulatory submissions, and projected cash position. We may, in some cases use terms such as “future,” “predicts,” “believes,” “potential,” “continue,” “anticipates,” “estimates,” “expects,” “plans,” “intends,” “targeting,” “confidence,” “may,” “could,” “might,” “likely,” “will,” “should” or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team and on information currently available to management that involve risks, potential changes in circumstances, assumptions, and uncertainties. All statements contained in this press release other than statements of historical fact are forward-looking statements, including statements regarding our ability to develop, commercialize and achieve market acceptance of our current and planned products and services, our research and development efforts, including timing considerations and other matters regarding our business strategies, use of capital, results of operations and financial position, and plans and objectives for future operations. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward-looking statements are submits to risks and uncertainties including, without limitation, risks related to the success, timing and cost of our ongoing clinical trials and anticipated clinical trials for our current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials, futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results )including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of our ongoing clinical trials, and the other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to update such forward-looking statement for any reason, except as required by law. Investor Contact: Mike MoyerLifeSci Advisorsmmoyer@lifesciadvisors.com

Executive ChangePhase 3

01 Dec 2022

·www.sanofi.com

Sanofi to showcase new data from oncology portfolio spanning marketed products and investigational compounds at ASH 2022

Sanofi to showcase new data from oncology portfolio spanning marketed products and investigational compounds at ASH 2022 Four Sarclisa ® (isatuximab) oral presentations and 11 posters featuring cutting-edge investigational and marketed therapies to be presented December 2 , 2022 . Data featured at the 64 th American Society of Hematology (ASH) Annual Meeting & Exposition from December 10-13, 2022, reinforce Sanofi’s commitment to transforming care for people living with multiple myeloma (MM) and other difficult-to-treat blood cancers. Peter C. Adamson , MD Global Head of Oncology Development “ Data to be presented at this year’s ASH Meeting demonstrate our commitment to improving the outcome for patients with cancer . This includes presentations for Sarclisa , an anti- CD38 antibody of choice for patients with multiple myeloma and a cornerstone of our strategy to broaden our portfolio in hematologic malignancies . W e are proud to be advancing knowledge and the treatment of patients with multiple myeloma , by sharing emerging data from our research efforts . ” The first Sarclisa oral presentation will detail results from a subgroup analysis of the Phase 3 IKEMA trial, which in May 2022 reported updated median progression-free survival results in combination with carfilzomib and dexamethasone. This new analysis compared patients with early versus late relapse. The second Sarclisa oral presentation highlights updated longer-term efficacy data following subsequent therapy in the pivotal Phase 3 ICARIA-MM trial. It is critical to advance scientific understanding of how individuals who relapse early will respond to subsequent lines of therapy because the earlier a person relapses, the more difficult they can be to treat. Sanofi is also presenting multiple abstracts from its investigational early pipeline of cutting-edge compounds, such as an open-label, first-in-human, dose-escalation study of Natural Killer Cell Engager (NKCE) SAR443579 as a monotherapy for the treatment of relapsed or refractory acute myeloid leukemia, B-cell acute lymphoblastic leukemia or high-risk myelodysplasia. Another abstract will highlight the potential of SAR’514, an anti-B cell Maturation Antigen (BCMA) NKCE, for controlling MM tumors in vivo. Since 2019, Sanofi’s oncology pipeline has doubled, with a dozen next-generation, potential first- or best-in-class compounds entering clinical trials. Much of this growth is a result of an acceleration of in-house research and development capabilities, as well as building external partnerships, particularly in early portfolio, including for MM and other hematologic malignancies. A bstracts accepted for presentation at ASH include: Isatuximab Isatuximab Plus Pomalidomide/Low-Dose Dexamethasone Versus Pomalidomide/Low-Dose Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma (ICARIA-MM): Characterization of Subsequent Antimyeloma Therapies Oral presentation Abstract #247 Dec. 10, 2-3:30 p.m. CST Isatuximab Plus Carfilzomib and Dexamethasone in Patients with Early Versus Late Relapsed Multiple Myeloma: IKEMA Subgroup Analysis Oral presentation Abstract #753 Dec. 12, 10:30 a.m.-12 p.m. CST Isatuximab, Carfilzomib, Lenalidomide, and Dexamethasone (Isa-KRd) in Patients with High-Risk Newly Diagnosed Multiple Myeloma: Planned Interim Analysis of the GMMG-Concept Trial Oral presentation by GMMG Abstract #759 Dec. 12, 10:30 a.m.-12 p.m. CST Bone Marrow Immune Signatures in Multiple Myeloma Are Linked to Tumor Heterogeneity and Treatment Outcome Oral presentation by GMMG Abstract #860 Dec. 12, 2:45-4:15 p.m. CST Subcutaneous Isatuximab Administration by an On-Body Delivery System (OBDS) in Combination with Pomalidomide and Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma: Phase 1b Expansion Study Results Poster Abstract #1923 Dec. 10, 5:30-7:30 p.m. CST Isatuximab Plus Carfilzomib and Dexamethasone in Relapsed Multiple Myeloma: IKEMA Subgroup Analysis By Number of Prior Lines of Treatment Poster Abstract #3176 Dec. 11, 6-8 p.m. CST Isatuximab in Combination with Lenalidomide and Dexamethasone in Patients with High-Risk Smoldering Multiple Myeloma: Updated Safety Run-in Results from the Randomized Phase 3 ITHACA study Poster Abstract #3253 Dec. 11, 6-8 p.m. CST Isatuximab Plus Pomalidomide and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma in Real-Life Context in France: IMAGE Subgroup Analysis Based on Prior Lines of Therapy and Refractory Status Poster Abstract #4928 Dec. 12, 6-8 p.m. CST Rasburicase Fatalities from Tumor Lysis Syndrome (TLS) After Anti-Hyperuricemic Monotherapy – Nationally Representative, Propensity Score Matched, Retrospective Study Comparison of Rasburicase and Allopurinol Poster Abstract #3632 Dec. 11, 6-8 p.m. CST Pipeline and other MAP4K2 Inhibition Reinforces the Iberdomide Sensitivity in MM Cells by Inducing IKZF1 Degradation Through a CRBN Independent Mechanism (Externally Sponsored Collaboration) Poster Abstract #1838 Dec. 10, 5:30-7:30 p.m. CST Real-World Multiple Myeloma Risk Factors and Outcomes by Race/Ethnicity in the United States Poster Abstract #2285 Dec. 10, 5:30-7:30 p.m. CST High Ex Vivo Response Rates to CD38/CD28xCD3 Trispecific T Cell Engager in Patients Relapsed After Anti-CD38 and Anti-BCMA Targeted Immunotherapies (Externally Sponsored Collaboration) Poster Abstract #3157 Dec. 11, 6-8 p.m. CST Real-World Multiple Myeloma Front-Line Treatment and Outcomes by Transplant in the United States Poster Abstract #3198 Dec. 11, 6-8 p.m. CST An Open-Label, First-in-Human, Dose-Escalation Study of SAR443579 Administered as Single Agent by Intravenous Infusion in Patients with Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-Cell Acute Lymphoblastic Leukemia (B-ALL) or High-Risk Myelodysplasia (HR-MDS) (Trial in Progress) Poster Abstract #3329 Dec. 11, 6-8 p.m. CST The Novel Trifunctional Anti-BCMA NK Cell Engager SAR’514 Has Potent in-Vitro and in-Vivo Anti-Myeloma Effect Through Dual NK Cell Engagement Poster Abstract #4486 Dec. 12, 6-8 p.m. CST Pegathor Lymphoma, a Phase 2 Study of SAR444245 as a Monotherapy or in Combination with Pembrolizumab for the Treatment of Adults and Adolescents with Relapsed or Refractory B Cell Lymphoma (Trial in Progress) Online abstract only About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media Relations Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com Sally Bain | + 1 617 834 6026 | sally.bain@sanofi.com Kate Conway | + 1 508 364 4931 | kate.conway@sanofi.com Investor Relations Eva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.com Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com Priya Nanduri | + 1 617 764 6418| priya.nanduri@sanofi.com Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com Sanofi Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that COVID-19 will have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2021. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Attachment PDF

Clinical ResultPhase 2Phase 3ImmunotherapyASH

100 Deals associated with Rasburicase

External Link

KEGG	Wiki	ATC	Drug Bank
D05704	Rasburicase	V03AF07	DB00049

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hyperuricemia	European Union	Sanofi Winthrop Industrie SA	23 Feb 2001
Hyperuricemia	Iceland	Sanofi Winthrop Industrie SA	23 Feb 2001
Hyperuricemia	Liechtenstein	Sanofi Winthrop Industrie SA	23 Feb 2001
Hyperuricemia	Norway	Sanofi Winthrop Industrie SA	23 Feb 2001

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hematologic Neoplasms	Phase 3	China	Sanofi	01 Oct 2007
Leukemia	Phase 3	United States	Sanofi	01 Apr 2004
Lymphoma	Phase 3	United States	Sanofi	01 Apr 2004
Solid tumor	Phase 3	United States	Sanofi	01 Apr 2004
Tumor Lysis Syndrome	Phase 3	United States	Sanofi	01 Apr 2004
Neoplasms	Phase 2	United States	Sanofi	01 Jan 2008
Acute Myeloid Leukemia	Phase 2	United States	Sanofi	01 Jul 2006
Blast Phase Chronic Granulocytic Leukemia	Phase 2	United States	Sanofi	01 Jul 2006
Aggressive Non-Hodgkin Lymphoma	Phase 2	Belgium	Sanofi	01 Jul 2002
Aggressive Non-Hodgkin Lymphoma	Phase 2	France	Sanofi	01 Jul 2002

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESMO2023	Not Applicable	Neoplasms	715	Rasburicase	emaecmzmmx(ikptjwszzp) = thzclkpbte lkwxuboyzj (txssmuekaa ) View more	-	23 Oct 2023
ESMO2023	Not Applicable	Neoplasms	715	Allopurinol	emaecmzmmx(ikptjwszzp) = qvqefqmjsa lkwxuboyzj (txssmuekaa ) View more	-	23 Oct 2023
ERA2023	Not Applicable	Cardio-Renal Syndrome hyperuricemia	23	Rasburicase	dsbqxdjbgr(crjlxrkovz) = qpcgqsxnyf rjttoowwja (ntiielnpte ) View more	-	15 Jun 2023
EULAR2022	Not Applicable	Arthritis, Gouty	17	Rasburicase 1.5mg/d	awzndunive(edygebrvij) = juzpoxefjv jzvzsfwuww (kzkdcesmfp ) View more	-	01 Jun 2022
EHA2020	Not Applicable	Tumor Lysis Syndrome \| Leukemia \| Lymphoma	55	Low dose Rasburicase	nhhtwipkoo(ujurebfajy) = dihczwsvlh xmvbewgloq (wdeoinnjoy )	Positive	14 May 2020
NCT01200485 (CTgov)	Phase 2	Tumor Lysis Syndrome \| Leukemia \| Lymphoma	55	Rasburicase (Arm A (Rasburicase))	wcwhmezudk = ojgvyytwhd bxeruvbwcp (exsemrmbos, mgtitsuuqg - zwiljqykfi) View more	-	31 Jan 2020
NCT01200485 (CTgov)	Phase 2	Tumor Lysis Syndrome \| Leukemia \| Lymphoma	55	Allopurinol (Arm B (Allopurinol))	wcwhmezudk = weznzfhdts bxeruvbwcp (exsemrmbos, ebbkylomxf - vlqowinojy) View more	-	31 Jan 2020
EHA2019	Not Applicable	Hematologic Neoplasms	-	Low dose rasburicase	vwecaaxbtk(iafbzkmchd) = sqiroaxjvz jrznbdsbbq (mtlevwzcii ) View more	-	16 May 2019
NCT00513474 (CTgov)	Phase 1	Myelodysplastic-Myeloproliferative Diseases \| Multiple Myeloma \| Myeloproliferative Disorders ... View more	46	allogeneic hematopoietic stem cell transplantation+cyclosporin-A+Etoposide+rasburicase+Fludarabine+Busulfan+cyclophosphamide+Tacrolimus+sirolimus+methotrexate (Rasburicase Group)	rfaiebzsiz = bembmkklnk pohqbbhgvy (nhbgtlzyyo, bzmhuavggx - dfqqfvmxyb) View more	-	11 Apr 2017
NCT00513474 (CTgov)	Phase 1		46	allogeneic hematopoietic stem cell transplantation+cyclosporin-A+Etoposide+Fludarabine+Busulfan+cyclophosphamide+Tacrolimus+sirolimus+allopurinol+methotrexate (Control Group)	rfaiebzsiz = mjzmeswbcb pohqbbhgvy (nhbgtlzyyo, cixqrohgvy - dqornfywsr) View more	-	11 Apr 2017
ASN2015	Not Applicable	Rhabdomyolysis CPK \| serum myoglobin	-	Rasburicase	kqdfdowlao(aotjaaovbw) = vmmzpqpmzh vdjdjabepz (trlmditanp )	Positive	03 Nov 2015
ASN2015	Not Applicable	Urate nephropathy	-	Rasburicase	bzcgdlmrrb(kmiqyhzxhj) = qtlbsrnozz lthavsymba (roooelxknm ) View more	Positive	03 Nov 2015
ASN2014	Not Applicable	Urate nephropathy Serum uric acid	-	Rasburicase	dngdqigyen(hyvwqwknho) = nsnbinwhyb befxrtdzhg (ebvngqkrbt )	Positive	11 Nov 2014

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