Vor Bio Reports Third Quarter 2024 Financial Results and Provides Company Update
Vor Bio reported Q3 2024 financial results and corporate updates. The company's net loss decreased to $27.6M from $33.2M year-over-year. Cash position stands at $62.8M, projected to fund operations into H2 2025. R&D expenses decreased to $21.8M from $27.6M, while G&A expenses reduced to $6.7M from $7.7M. Clinical progress includes 25 patients dosed with trem-cel and 6 patients receiving Mylotarg at 2 mg/m2. The company appointed Han Choi as CFO and plans to present updated clinical data at the upcoming ASH annual meeting.
Vor Bio ha riportato i risultati finanziari del terzo trimestre 2024 e aggiornamenti aziendali. La perdita netta dell'azienda è diminuita a $27.6 milioni rispetto a $33.2 milioni nell'anno precedente. La posizione di cassa è pari a $62.8 milioni, projected per finanziare le operazioni fino al secondo semestre del 2025. Le spese di R&D sono diminuite a $21.8 milioni rispetto a $27.6 milioni, mentre le spese generali e amministrative sono scese a $6.7 milioni rispetto a $7.7 milioni. I progressi clinici includono 25 pazienti trattati con trem-cel e 6 pazienti che ricevono Mylotarg a 2 mg/m2. L'azienda ha nominato Han Choi come CFO e prevede di presentare dati clinici aggiornati alla prossima riunione annuale dell'ASH.
Vor Bio reportó los resultados financieros del tercer trimestre de 2024 y actualizaciones corporativas. La pérdida neta de la empresa disminuyó a $27.6 millones desde $33.2 millones en el año anterior. La posición de efectivo es de $62.8 millones, proyectada para financiar operaciones hasta el segundo semestre de 2025. Los gastos en I+D disminuyeron a $21.8 millones desde $27.6 millones, mientras que los gastos administrativos y generales se redujeron a $6.7 millones desde $7.7 millones. El progreso clínico incluye 25 pacientes tratados con trem-cel y 6 pacientes que reciben Mylotarg a 2 mg/m2. La empresa nombró a Han Choi como CFO y planea presentar datos clínicos actualizados en la próxima reunión anual de la ASH.
Vor Bio는 2024년 3분기 재무 결과와 기업 업데이트를 보고했습니다. 회사의 순손실은 전년 대비 $33.2백만에서 $27.6백만으로 감소했습니다. 현금 보유고는 $62.8백만이며, 이는 2025년 하반기까지 운영 자금을 지원할 것으로 예상됩니다. R&D 지출은 $27.6백만에서 $21.8백만으로 감소했으며, G&A 지출은 $7.7백만에서 $6.7백만으로 줄었습니다. 임상 진행 상황에는 trem-cel 치료를 받은 25명의 환자와 2 mg/m2의 Mylotarg를 받은 6명의 환자가 포함됩니다. 회사는 Han Choi를 CFO로 임명했으며, 다가오는 ASH 연례 회의에서 업데이트된 임상 데이터를 발표할 계획입니다.
Vor Bio a rapporté ses résultats financiers du troisième trimestre 2024 ainsi que des mises à jour sur l'entreprise. La perte nette de l'entreprise a diminué, passant de 33,2 millions de dollars à 27,6 millions de dollars d'une année sur l'autre. La position de trésorerie est de 62,8 millions de dollars, prévue pour financer les opérations jusqu'au deuxième semestre 2025. Les dépenses de R&D ont diminué à 21,8 millions de dollars, contre 27,6 millions de dollars, tandis que les dépenses générales et administratives ont été réduites à 6,7 millions de dollars, contre 7,7 millions de dollars. Les progrès cliniques incluent 25 patients traités avec trem-cel et 6 patients recevant Mylotarg à 2 mg/m2. L'entreprise a nommé Han Choi au poste de CFO et prévoit de présenter des données cliniques mises à jour lors de la prochaine réunion annuelle de l'ASH.
Vor Bio hat die Finanzzahlen für das dritte Quartal 2024 und Unternehmensupdates veröffentlicht. Der Nettoverlust des Unternehmens sank von 33,2 Millionen USD auf 27,6 Millionen USD im Jahresvergleich. Die Liquiditätsposition beläuft sich auf 62,8 Millionen USD, die voraussichtlich die Betriebskosten bis zum zweiten Halbjahr 2025 decken wird. Die F&E-Ausgaben sanken von 27,6 Millionen USD auf 21,8 Millionen USD, während die allgemeinen und administrativen Ausgaben von 7,7 Millionen USD auf 6,7 Millionen USD reduziert wurden. Der klinische Fortschritt umfasst 25 Patienten, die mit trem-cel behandelt wurden, und 6 Patienten, die Mylotarg bei 2 mg/m2 erhalten. Das Unternehmen hat Han Choi zum CFO ernannt und plant, aktualisierte klinische Daten auf dem bevorstehenden ASH-Jahrestreffen zu präsentieren.
- Net loss improved by $5.6M year-over-year to $27.6M
- R&D expenses decreased by $5.8M compared to Q3 2023
- G&A expenses reduced by $1.0M year-over-year
- Clinical trial showing positive early evidence in patient relapse-free survival
- Strong enrollment continuing in VCAR33ALLO trial
- Cash position decreased from $137.2M to $62.8M since December 2023
- Operating loss of $28.5M in Q3 2024
- Interest income declined to $0.95M from $2.13M year-over-year
Insights
The Q3 2024 results show a concerning cash burn rate, with cash reserves declining from
The clinical progress of trem-cel shows promising signs with 25 patients dosed and 6 reaching the third dose level of Mylotarg at 2 mg/m2. Key positive indicators include reliable engraftment, maintained blood counts across multiple Mylotarg doses and encouraging relapse-free survival data compared to historical controls. The VCAR33ALLO trial demonstrates encouraging CAR-T expansion in initial patients. The new VADC45 program targeting CD45 expands the pipeline into broader therapeutic areas. However, while the data is encouraging, more mature clinical results are needed to fully validate the platform's potential.
- Company to present trem-cel + Mylotarg clinical data update at American Society of Hematology (ASH) 2024 annual meeting
- Han 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
VADC45
In 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 2024
In 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 2024
Additional 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 Immunotherapy
Format: Poster presentation
Session 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 Bio
Vor 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 Statements
This 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 & Media
Sarah Spencer
+1 857-242-6076
sspencer@vorbio.com
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