Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering
company, presented clinical data from VBP101, its Phase 1/2a
multicenter, open-label, first-in-human study of trem-cel
(previously VOR33) in patients with acute myeloid leukemia (AML).
In the first patient, trem-cel maintained hematopoiesis
through three cycles of Mylotarg (gemtuzumab ozogamicin), which was
well-tolerated at the initial dose level of 0.5 mg/m2. A second
patient has successfully received a trem-cel transplant and
engrafted normally. These data were presented today by Miguel-Angel
Perales, MD, Chief, Adult Bone Marrow Transplant Service, Memorial
Sloan Kettering Cancer Center in a late-breaking poster at the 2023
Tandem Meetings (Transplantation & Cellular Therapy Meetings of
ASTCT™ and CIBMTR®) in Orlando, FL.
“These data continue to demonstrate the potential of trem-cel as
a next-generation hematopoietic stem cell transplant, which could
transform treatment for patients with blood cancers such as AML,”
said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “We are also
encouraged that a second patient successfully received a trem-cel
transplant and look forward to learning more as we treat additional
patients and dose escalate Mylotarg. In addition, we plan to file
an IND for VCAR33ALLO, a novel allogeneic CAR-T treatment which we
believe could be more efficacious than Mylotarg in combination with
a trem-cel transplant.”
Trem-cel Safety & DurabilityPatient 1
maintained neutrophil and platelet counts approximately five months
(147 days) after transplantation with trem-cel. Due to detectable
measurable residual disease (MRD), Patient 1 was moved to other
therapies following administration of the third dose of Mylotarg,
subsequently relapsed, and remains on study for long-term
follow-up.
Similar to Patient 1, Patient 2 successfully received a trem-cel
transplant and showed robust cell recovery with neutrophil
engraftment occurring at Day 11 and platelet recovery on Day 17.
Trem-cel was well tolerated in both patients, with no related and
no unexpected adverse events (AEs) reported.
No Hematological Toxicity Observed Through Repeated
Doses of MylotargIn Patient 1, neutrophil and platelet
cell counts were maintained following three sequential Mylotarg
doses at 0.5 mg/m2. This suggests potential protection from
Mylotarg-related hematotoxicity. The only AE observed possibly
related to Mylotarg through dose 3 was low grade nausea and
vomiting, a known side-effect of Mylotarg. Mylotarg first-dose
pharmacokinetics revealed 0.5 mg/m2 achieved Cmax and AUC
parameters equivalent to 1-2 and 4-5 mg/m2 accordingly, potentially
due to the decreased CD33 antigen sink.
Evidence of Mylotarg Causing CD33-negative Donor Cell
EnrichmentIn Patient 1, CD33-negative donor hematopoiesis
was enriched across hematopoietic cell types following Mylotarg
administration. In addition, the CD33 deletion was observed in
donor cells of myeloid and lymphoid origin which were both enriched
following Mylotarg, suggesting that CD33 is expressed in early
hematopoietic cells and that Mylotarg treatment enriches for edited
donor cells.
Interest in enrollment in VBP101 continues to be strong with a
high level of investigator enthusiasm at all nine study sites. The
company is moving forward with dose escalation of Mylotarg per the
3+3 dose escalation schema in the protocol. The Company is also
on-track to submit an IND in the first half of 2023 for VCAR33ALLO,
a CAR-T therapy using allogeneic healthy donor-derived cells, which
it intends use in combination with trem-cel as a Treatment
System.
About AMLAML is the most common type of acute
leukemia in adults and one of the deadliest and most aggressive
blood cancers, affecting 20,000 newly diagnosed patients each year
in the United States. Approximately half of patients with AML who
receive a hematopoietic cell transplant (HCT) suffer a relapse of
their leukemia, with two-year survival rates of less than 20%, and
relapse rates are higher for patients with certain adverse risk
features. The fragility of engrafted hematopoietic stem cells
prevents treatment following transplant, giving the cancer a chance
to return.
About the VBP101 Clinical TrialVBP101 is a
Phase 1/2a, multicenter, open-label, first-in-human study of
trem-cel in participants with AML who are undergoing human
leukocyte antigen (HLA)-matched allogeneic HCT. Trem-cel is an
allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and
progenitor cell (HSPC) therapy product, lacking the CD33 protein.
It is being investigated for participants with CD33+ AML at high
risk for relapse after HCT to allow post-HCT targeting of residual
CD33+ acute AML cells using Mylotarg without toxicity to engrafted
cells. Participants undergo a myeloablative HCT with matched
related or unrelated donor CD34-selected HSPCs engineered to remove
CD33 expression (trem-cel drug product). Mylotarg is given after
engraftment for up to four cycles. The primary endpoint is the
incidence of successful engraftment, defined as the first day of 3
consecutive days of absolute neutrophil count (ANC) 500 cells/mm2
by day 28. Part 1 of this study is evaluating the safety of
escalating Mylotarg dose levels to determine the maximum tolerated
dose (MTD) and recommended Phase 2 dose. Part 2 will expand the
number of participants to evaluate the Mylotarg recommended Phase 2
dose. For more information, visit:
https://clinicaltrials.gov/ct2/show/NCT04849910
About Trem-celTremtelectogene empogeditemcel
(trem-cel), formerly VOR33, is a genome-edited hematopoietic stem
and progenitor allogeneic donor product candidate where CD33 has
been deleted using genome engineering. Transplant with trem-cel is
designed to replace standard of care transplants for patients
suffering from AML and potentially other blood cancers. Trem-cel
has the potential to enable powerful targeted therapies in the
post-transplant setting including CD33-targeted CAR-T cells.
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,” “believe,” “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 feasibility of a trem-cel transplant to be
successfully manufactured, to engraft normally, to maintain blood
counts following treatment with Mylotarg following allogeneic
hematopoietic cell transplant and to be well tolerated, the
potential of Vor Bio’s platform, and timing expectations for
additional regulatory filings. 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; whether successful engraftment and platelet recovery
will ultimately lead to efficacy of the product candidate;
expectations for 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. The interim data presented in
this press release is based on two patients and future results for
these patients or additional patients may not produce the same or
consistent results. 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
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