Gemtuzumab Ozogamicin

Data showcased at SABCS and ASH reflect progress and growth of Jazz's research and development in oncology and demonstrate ongoing efforts to redefine what is possible for cancer treatment DUBLIN , Dec. 3, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company and its partners will present two abstracts at the 2024 San Antonio Breast Cancer Symposium (SABCS) from December 10-13 and 13 abstracts at the 66th Annual American Society of Hematology (ASH) Annual Meeting from December 7-10. A trial-in-progress poster presentation at SABCS outlines the trial design of the ongoing Phase 3 EmpowHER-303 trial (NCT06435429), which is evaluating the efficacy and safety of Ziihera® (zanidatamab-hrii) vs trastuzumab with chemotherapy in patients with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, trastuzumab deruxtecan. New data from a Phase 1b/2 study of zanidatamab plus evorpacept (NCT05027139) in patients with pre-treated HER2-positive and HER2-low metastatic breast cancer will be featured as a poster spotlight. "Following the recent FDA approval of Ziihera for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, we are pleased to present meaningful data at SABCS on the positive impact zanidatamab can have for patients with HER2-expressing cancers. We continue to advance our clinical program for zanidatamab with the goal to improve outcomes for patients with difficult-to-treat HER2-positive cancers. Our development program includes multiple ongoing Phase 3 trials evaluating 1L BTC, 1L GEA, with top-line PFS results expected in the second quarter of 2025, and metastatic breast cancer after T-DXd treatment, supporting the use of zanidatamab after other HER2-targeted therapies," said Rob Iannone , M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals . "Additionally, we look forward to highlighting 13 abstracts featuring oncology research at ASH 2024, which underscores our commitment to improving standards of care in blood cancer and other hematologic diseases." The full SABCS abstracts are available here. The Jazz and partner-supported presentations at SABCS 2024 are: Ziihera® (zanidatamab-hrii) Presentations Topic Author Presentation Details EmpowHER 303: A phase 3 study to evaluate the efficacy and safety of zanidatamab vs trastuzumab withchemotherapy in patients (pts) with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, trastuzumab deruxtecan Sara M Tolaney, et al. Presentation Type: Poster Session Abstract Number: SESS-1922 Date: Friday December 13, 2024 Time: 12:00-2:00 PM (PST) Zanidatamab in combination with evorpacept in HER2-positive and HER2-low metastatic breast cancer: Results from a phase 1b/2 study Alberto J Montero, et al. Presentation Type: Poster Spotlight Presentation Abstract Number: SESS-2007 Date: Thursday December 12, 2024 Time: 7:00-8:30 AM (PST) The full ASH abstracts are available here. The Jazz and partner-supported presentations at ASH 2024 are: Vyxeos® (daunorubicin and cytarabine) Presentations Topic Author Presentation Details A Randomized Comparison of CPX-351 and FLAG-Ida in Patients WithHigh-Risk Acute Myeloid Leukemia(AML)/Myelodysplastic Syndrome (MDS) And MDS-Related GeneMutations: A Subgroup Analysis of the UK NCRI AML19 Trial Priyanka Mehta , et al. Presentation Type: Oral Presentation Abstract Number: #55 Date: Saturday December 7, 2024 Time: 9:30 AM (PST) AML-MR Mutations Drive the Benefit of CPX-351 over 7+3 in the Pivotal Phase 3 AML Trial Shai O Shimony, et al. Presentation Type: Oral Presentation Abstract Number: #60 Date: Saturday December 7, 2024 Time: 10:45 AM (PST) Phase Ib/II Study of CPX-351 in Combination with Venetoclax in Patients with Newly Diagnosed, HighRisk Acute Myeloid Leukemia Emmanuel Almanza , et al. Presentation Type: Poster Presentation Abstract Number: #1511 Date: Saturday December 7, 2024 Time: 5:30-7:30 PM (PST) A Randomised Comparison of CPX-351 versus Standard Daunorubicin and Cytarabine plus FractionatedGemtuzumab Ozogamicin in Older AML Adults Without Known AdverseRisk Cytogenetics: Results of the NCRIAML18 Trial Steven Knapper , et al. Presentation Type: Oral Presentation Abstract Number: #59 Date: Saturday December 7, 2024 Time: 10:30 AM (PST) Phase 1/1b Dose Escalation and Expansion of CPX-351 in Combinationwith Gemtuzumab Ozogamicin in Newly Diagnosed Acute MyeloidLeukemia Onyee Chan , et al. Presentation Type: Poster Presentation Abstract Number: #4270 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) A Phase 3 Randomized Trial for Patients with de novo AML Comparing Standard Therapy IncludingGemtuzumab Ozogamicin (GO) to CPX-351 with GO – A report from the Children's Oncology Group Jessica A Pollard, et al. Presentation Type: Oral Presentation Abstract Number: #967 Date: Monday December 9, 2024 Time: 4:30 PM (PST) Combination of CPX-351 andGemtuzumab Ozogamicin (GO) inRelapsed Refractory (R/R) AcuteMyeloid Leukemia and PostHypomethylating Agent (HMA) FailureHigh-Risk Myelodysplastic Syndrome (HR-MDS) Jayastu Senapati , et al. Presentation Type: Poster Presentation Abstract Number: #2903 Date: Sunday December 8, 2024 Time: 6:00-8:00 PM (PST) Phase Ib/II Study of CPX-351 plusVenetoclax in Patients with Relapsed/Refractory Acute MyeloidLeukemia (AML) Vanthana Bharathi , et al. Presentation Type: Poster Presentation Abstract Number: #4272 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Rapid, Reliable, and ComprehensiveIdentification of MDS-DefiningCytogenetic Changes By a FISH-Panel Containing Six Probes in a Real-World Population of Patients with Suspected AML Katayoon Shirneshan, et al. Presentation Type: Poster Presentation Abstract Number: #4308 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Prospective Evaluation of the Impact ofMeasurable Residual Disease (MRD) by Error Corrected Next-GenerationSequencing (NGS) with CPX-351 inAcute Myeloid Leukemia (AML) David Sallman , et al. Presentation Type: Poster Presentation Abstract Number: #4332 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) A Phase 1 Study of CPX-351 Plus Gilteritinib in Relapsed or Refractory, FLT3-Mutated Acute Myeloid Leukemia Onyee Chan , et al. Presentation Type: Poster Presentation Abstract Number: #1520 Date: Saturday December 7, 2024 Time: 5:30-7:30 PM (PST) Defitelio® (defibrotide sodium) Presentations Topic Author Presentation Details Genetic Susceptibility in Sinusoidal Obstruction Syndrome/Veno-OcclusiveDisease Ioulia Mavrikou, et al. Presentation Type: Poster presentation Abstract Number: #4778 Date: Monday December 9, 2024 Time: 6:00-8:00 PM (PST) Defibrotide reduces hypercoagulablestate in patients with Sickle Cell Disease-Related Acute ChestSyndrome Edo Schaefer , et al. Presentation Type: Poster presentation Abstract Number: #2515 Date: Sunday December 8, 2024 Time: 6:00-8:00 PM (PST) About Ziihera® (zanidatamab-hrii) Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity ( CDC ), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.1 In the United States , Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.1 The U.S. Food and Drug Administration (FDA) granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).1 Zanidatamab is not approved anywhere else in the world. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule. The FDA granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Important Safety Information for ZIIHERA® WARNING: EMBRYO-FETAL TOXICITY Exposure to ZIIHERA during pregnancy can cause embryo-fetal harm. Advise patientsof the risk and need for effective contraception. WARNINGS AND PRECAUTIONS Embryo-Fetal ToxicityZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular DysfunctionZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to <50% in 4.3% of 233 patients. Left ventricular dysfunction (LVD) leading to permanent discontinuation of ZIIHERA was reported in 0.9% of patients. The median time to first occurrence of LVD was 5.6 months (range: 1.6 to 18.7). LVD resolved in 70% of patients. Assess LVEF prior to initiation of ZIIHERA and at regular intervals during treatment. Withhold dose or permanently discontinue ZIIHERA based on severity of adverse reactions. The safety of ZIIHERA has not been established in patients with a baseline ejection fraction that is below 50%. Infusion-Related ReactionsZIIHERA can cause infusion-related reactions (IRRs). An IRR was reported in 31% of 233 patients treated with ZIIHERA as a single agent in clinical studies, including Grade 3 (0.4%), and Grade 2 (25%). IRRs leading to permanent discontinuation of ZIIHERA were reported in 0.4% of patients. IRRs occurred on the first day of dosing in 28% of patients; 97% of IRRs resolved within one day. Prior to each dose of ZIIHERA, administer premedications to prevent potential IRRs. Monitor patients for signs and symptoms of IRR during ZIIHERA administration and as clinically indicated after completion of infusion. Have medications and emergency equipment to treat IRRs available for immediate use. If an IRR occurs, slow, or stop the infusion, and administer appropriate medical management. Monitor patients until complete resolution of signs and symptoms before resuming. Permanently discontinue ZIIHERA in patients with recurrent severe or life-threatening IRRs. DiarrheaZIIHERA can cause severe diarrhea. Diarrhea was reported in 48% of 233 patients treated in clinical studies, including Grade 3 (6%) and Grade 2 (17%). If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Withhold or permanently discontinue ZIIHERA based on severity. ADVERSE REACTIONS Serious adverse reactions occurred in 53% of 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA. Serious adverse reactions in >2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric UseSafety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric UseOf the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. About Vyxeos® (daunorubicin and cytarabine) liposome for injectionVyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor. In the U.S., Vyxeos (daunorubicin and cytarabine) liposome for injection is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older.3 More information about Vyxeos in the United States, including Full Prescribing Information and BOXED Warning, is available here. Important Safety Information for VYXEOS® WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products. VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients. VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding. VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as: shortness of breath or trouble breathing swelling or fluid retention, especially in the feet, ankles, or legs unusual tiredness VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as: trouble breathing severe itching skin rash or hives swelling of the face, lips, mouth, or tongue VYXEOS contains copper and may cause copper overload in patients with Wilson's disease or other copper-processing disorders. VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site. VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS. The most common side effects are bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568. About Defitelio® (defibrotide sodium)In the U.S., Defitelio® (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication.6 Please see full Prescribing Information for Defitelio in the United States . In Europe, defibrotide is marketed under the name Defitelio® ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. ▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC (https://www.ema.europa.eu/en/documents/product-information/defitelio-epar-product-information_en.pdf) The full Summary of Product Characteristics of Defitelio in Europe is available here. Important Safety Information for Defitelio® Defitelio should not be given to patients who are: Currently taking anticoagulants or fibrinolytics Allergic to Defitelio or any of its ingredients Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision. Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately. The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds. Defitelio is not approved for the prevention of VOD. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy. About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases—often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information. Jazz Pharmaceuticals plc Caution Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to expectations of top-line PFS results in the second quarter of 2025 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with pharmaceutical product development, and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2023 , as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 , and future filings and reports by Jazz Pharmaceuticals . Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Jazz Media Contact: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc CorporateAffairsMediaInfo@jazzpharma.com Ireland +353 1 637 2141 U.S. +1 215 867 4948 Jazz Investor Contact: Jeff Macdonald Executive Director, Investor Relations Jazz Pharmaceuticals plc investorinfo@jazzpharma.com Ireland +353 1 634 3211 U.S. +1 650 496 2717 View original content to download multimedia:https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-to-present-advancements-in-solid-tumors-and-blood-cancer-research-at-san-antonio-breast-cancer-symposium-and-american-society-of-hematology-annual-meeting-302320929.html SOURCE Jazz Pharmaceuticals plc

Company to present trem-cel + Mylotarg clinical data update at American Society of Hematology (ASH) 2024 annual meetingHan Choi, M.D., LL.M, seasoned healthcare institutional investor, appointed as Chief Financial Officer CAMBRIDGE, Mass., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended September 30, 2024, and provided a corporate update. “We are pleased with our operational and clinical execution this quarter and look forward to sharing additional clinical data since our September data update at the ASH annual meeting in December,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. Corporate Updates Trem-cel + Mylotarg (VBP101) Clinical Trial 25 patients have been dosed with trem-cel and 6 patients have now received the third dose level of Mylotarg at 2 mg/m2. On September 5, 2024, the Company provided a clinical update on 18 patients treated with trem-cel in the VBP101 study. The data demonstrated: Reliable engraftment, robust platelet recovery, and high CD33 editing efficiency with full myeloid chimerism ​at Day 28.Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2.Broadened therapeutic index for Mylotarg with drug exposure represented by AUC which is related to efficacy, consistent with labeled Mylotarg doses, and with maximal concentrations, measured by Cmax and related to veno-occlusive disease, well below known toxic range.Early evidence suggesting patient benefit as measured by relapse-free survival when compared to published high-risk AML comparators1. The Company plans to share a further clinical data update in a poster presentation at the ASH Annual Meeting on Sunday, December 8, from 6-8pm PT. (Poster Session 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies. Publication number 2873). The poster will be made available on Vor Bio’s website when the ASH embargo lifts at 9am PT on the day of the presentation and the Company plans to host a webcast to review the data on Monday, December 9th at 5am PT/8am ET. Trem-cel is a shielded transplant in development for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to shield healthy cells and enable targeted therapies post-transplant such as Mylotarg and CAR-T therapy. VCAR33ALLO (VBP301) Clinical Trial Dosing is ongoing in the VBP301 study with continued strong enrollment.On September 5, 2024, the Company announced encouraging in vivo CAR-T expansion data from three patients treated to date, all at the lowest dose of 1 x 106 CAR+ cells/kg. VBP301, a Phase 1/2, multicenter, open-label, first-in-human study of VCAR33ALLO, is a transplant donor-derived anti-CD33 CAR-T cell therapy for patients with AML who have relapsed following a standard-of-care or trem-cel transplant. Preclinical Research Updates VADC45In September 2024, the Company announced a new preclinical asset, VADC45, which has several potential opportunities in oncology, gene therapy, and autoimmune disorders. VADC45 is an antibody-drug conjugate (ADC) designed to target the CD45 protein, a well-validated target for a wide variety of blood cancers with clinical poof of concept. The linker-payload used in VADC45 has also been clinically validated. Vor Bio is currently progressing IND-enabling studies to enable future Phase 1 studies. Preclinical data presentations further validate Vor Bio’s robust platform ESGCT 2024In October 2024, the Company presented preclinical data at the European Society of Gene and Cell Therapy Congress on the development and characterization of its novel CD33/CLL-1-directed CAR-T approach in AML. ASH 2024Additional analyses from the most comprehensive single cell AML atlas known to date (more than 400,000 cells from 26 AML patients and 10 healthy donors) were accepted for presentation at the ASH 2024 annual meeting in December. These data provide an unprecedented view of AML immunophenotypic heterogeneity, both across patients and within patients across clinical timepoints.Abstract Title: Multimodal Atlas of Paired Diagnosis and Relapse AML Reveals Surface Antigens for Multi-Specific ImmunotherapyFormat: Poster presentationSession Name: Acute Myeloid Leukemias: Biomarkers and Molecular Markers in Diagnosis and Prognosis Session date and time: Saturday, Dec 7, 5:30-7:30pm PT New Chief Financial Officer appointed Han Choi, M.D., LL.M., was appointed as Vor Bio’s new Chief Financial Officer. Dr. Choi brings more than 25 years of experience in investment management, business development, and corporate strategy within the pharmaceutical and biotechnology sectors to Vor Bio. His extensive expertise in forming corporate partnerships, managing complex transactions, and developing capital markets strategies will be pivotal as the Company advances its clinical pipeline and strengthens its leadership in cell and genome engineering. Upcoming Milestones Trem-cel + Mylotarg clinical data update planned at ASH 2024 annual meeting Third Quarter 2024 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $62.8 million as of September 30, 2024, which is projected to fund operations into the second half of 2025. Research & Development (R&D) Expenses: R&D expenses for the third quarter of 2024 were $21.8 million, compared to $27.6 million for the third quarter of 2023. The decrease of $5.8 million was due to a decrease in license payments, preclinical expenses, and manufacturing starting materials, offset in part by an increase in clinical trial costs to support our trem-cel and VCAR33ALLO programs.General & Administrative (G&A) Expenses: G&A expenses for the third quarter of 2024 were $6.7 million, compared to $7.7 million for the third quarter of 2023. The decrease of $1.0 million was primarily due to a decrease in professional fees and stock-based compensation expense. Net Loss: Net loss for the third quarter of 2024 was $27.6 million, compared to $33.2 million for the third quarter of 2023. Condensed Consolidated Balance Sheet Data (Unaudited) (in thousands) September 30, December 31, 2024 2023 Cash, cash equivalents and marketable securities$62,809 $137,175 Total assets 115,991 198,126 Total liabilities 43,036 47,402 Total stockholders' equity 72,955 150,724 Condensed Consolidated Statement of Operations (Unaudited) (in thousands, except share and per share data) Three Months Ended September 30, 2024 2023 Operating expenses: Research and development $21,817 $27,606 General and administrative 6,696 7,710 Total operating expenses $28,513 $35,316 Loss from operations $(28,513) $(35,316) Other income: Interest income 954 2,126 Total other income 954 2,126 Net loss $(27,559) $(33,190) Net loss per share attributable to common stockholders, basic and diluted $(0.40) $(0.49) Weighted-average common shares outstanding, basic and diluted 68,465,801 67,607,713 About Vor BioVor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, the timing and pace of patient enrollment and dosing in clinical trials and the availability of data therefrom, the potential of trem-cel to enable targeted therapies in the post-transplant setting including Mylotarg and CD33-targeted CAR-Ts, its potential upcoming milestones, its cash runway and expected capital requirements, and other statements that are not historical fact. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; uncertainties regarding regulatory approvals to conduct trials or to market products; the success of Vor Bio’s in-house manufacturing capabilities and efforts; and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements and Vor Bio’s ability to continue as a going concern. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law. Contact:Investors & MediaSarah Spencer +1 857-242-6076sspencer@vorbio.com