Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced new safety and efficacy data in 17
patients treated with EDIT-301, now known as renizgamglogene
autogedtemcel (reni-cel), in the RUBY trial for severe sickle cell
disease (SCD) (n=11) and in the EdiTHAL trial for
transfusion-dependent beta thalassemia (TDT) (n=6). The total
dataset of 17 treated patients includes 12 additional patients
since the data presentation at the European Hematology Association
(EHA) Annual Congress and in a Company-sponsored webinar this past
June. Reni-cel is being investigated in the RUBY and EdiTHAL
clinical trials as a potential one-time, durable gene editing
medicine for people living with severe SCD and TDT.
Editas Medicine will present the RUBY and EdiTHAL trial data
today at 1 p.m. ET in a Company-sponsored webinar.
The data will also be presented in a poster presentation at the
American Society of Hematology (ASH) Annual Meeting in San Diego,
CA, at 6:00 p.m. PT (9:00 p.m. ET).
In both the RUBY and EdiTHAL trials to date, reni-cel was
well-tolerated and continues to demonstrate a safety profile
consistent with myeloablative conditioning with busulfan and
autologous hematopoietic stem cell transplant by all patients in
the two trials (n=17). Since treatment with reni-cel, all RUBY
patients are free of vaso-occlusive events (VOEs) (n=11). All RUBY
patients with ≥5 months follow-up have maintained a normal
hemoglobin level and a fetal hemoglobin level of >40%. All
EdiTHAL patients had early and robust increase of total hemoglobin,
above the transfusion independence threshold of 9 g/dl (n=6).
“These new and promising data with a larger patient cohort
support our belief that reni-cel can be a clinically
differentiated, one-time, durable medicine that can provide
life-changing clinical benefits to patients with sickle cell
disease and beta thalassemia, specifically driving early and robust
correction of anemia and sustained increases in fetal hemoglobin,”
said Baisong Mei, MD, Ph.D., Senior Vice President and Chief
Medical Officer, Editas Medicine. “I would like to thank the
clinical trial participants, their families, clinicians, and
colleagues at collaborating institutions that contribute to the
RUBY and EdiTHAL trials. We look forward to dosing additional
patients and sharing further RUBY and EdiTHAL clinical updates in
mid-2024.”
“These preliminary results from the RUBY and EdiTHAL trials
are encouraging. This investigational gene editing therapy has
been well-tolerated and shows promising efficacy, and we look
forward to continuing to evaluate its effectiveness on this patient
population in need of new treatment options,” said Rabi Hanna,
M.D., Chairman of the Division of Pediatric Hematology
Oncology and Blood and Marrow Transplantation at Cleveland Clinic
Children’s.
SafetyReni-cel was well-tolerated and
demonstrated a safety profile consistent with myeloablative
conditioning with busulfan and autologous hematopoietic stem cell
transplant by all patients in the RUBY and EdiTHAL trials
(n=17).
After reni-cel infusion, all treated patients with >2 months
follow-up demonstrated successful neutrophil engraftment within one
month and platelet engraftment within 1.6 months. No serious
adverse events (SAEs) related to reni-cel treatment have been
reported.
EfficacyRUBY Trial in Severe Sickle
Cell DiseaseIn the RUBY trial, all treated patients are
free of VOEs since reni-cel infusion. Reni-cel treatment drives
early, robust increase of total hemoglobin and fetal hemoglobin.
The patients with ≥5 months follow-up have maintained a normal
hemoglobin level and a fetal hemoglobin level of >40% (n=6;
range 5-18 months follow-up). All treated RUBY patients with >1
month of follow-up followed a similar trajectory of total
hemoglobin and fetal hemoglobin increases (n=10).
EdiTHAL Trial in Transfusion-dependent Beta
ThalassemiaIn the EdiTHAL trial, patients with >1 month
follow-up (n=5) demonstrated early and robust total hemoglobin and
fetal hemoglobin increases, with total hemoglobin rising above the
transfusion independence threshold of 9 g/dL.
Webinar Presentation Details: The live and
archived webcast of the Company’s webinar presentation will be
accessible through this webcast link, or through the Events &
Presentations page of the “Investors” section of the Company’s
website.
A replay of the webinar will be available upon conclusion of the
webinar in the Investors section of the Editas Medicine website
at https://www.editasmedicine.com/.
ASH Presentation Details:
Title: AsCas12a Gene Editing of HBG1/2 Promoters
with EDIT-301 Results in Rapid and Sustained Normalization of
Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe
Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia
Presenting Author: Rabi Hanna, M.D., Department of
Pediatric Hematology Oncology and Blood and Marrow Transplantation,
Cleveland Clinic Children’s, Cleveland, OH, United
StatesDate/Time: Monday, December 11, 2023, 6:00 –
8:00 p.m. PT/9:00 – 11:00 p.m. ETLocation: San
Diego Convention Center, Halls G-HSession: 801.
Gene Therapies: Poster IIIPublication Number:
4996
The abstract can be accessed on the ASH website.
About renizgamglogene autogedtemcel
(reni-cel)Reni-cel, formerly known as EDIT-301, is an
experimental gene editing medicine under investigation for the
treatment of severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT). Reni-cel consists of
patient-derived CD34+ hematopoietic stem and progenitor cells
edited at the gamma globin gene (HBG1 and HBG2) promoters, where
naturally occurring fetal hemoglobin (HbF) inducing mutations
reside, by AsCas12a, a novel, proprietary, highly efficient, and
specific gene editing nuclease. Red blood cells derived from
reni-cel CD34+ cells demonstrate a sustained increase in fetal
hemoglobin production, which has the potential to provide a
one-time, durable treatment benefit for people living with severe
SCD and TDT.
About the RUBY TrialThe RUBY trial is a
single-arm, open-label, multi-center Phase 1/2 study designed to
assess the safety and efficacy of reni-cel in patients with severe
sickle cell disease. Enrolled patients will receive a single
administration of reni-cel. The RUBY trial marks the first time
AsCas12a was used to successfully edit human cells in a clinical
trial. Additional details are available
on www.clinicaltrials.gov (NCT# 04853576).
About the EdiTHAL TrialThe EdiTHAL trial is a
single-arm, open label, multi-center Phase 1/2 study designed to
assess the safety and efficacy of reni-cel in patients with
transfusion-dependent beta thalassemia. Patients will receive a
single administration of reni-cel. Additional details are available
on www.clinicaltrials.gov (NCT# 05444894).
About Editas MedicineAs a
clinical-stage genome editing company, Editas Medicine is focused
on translating the power and potential of the CRISPR/Cas12a and
CRISPR/Cas9 genome editing systems into a robust pipeline of
treatments for people living with serious diseases around the
world. Editas Medicine aims to discover, develop, manufacture, and
commercialize transformative, durable, precision genomic medicines
for a broad class of diseases. Editas Medicine is the exclusive
licensee of Broad Institute’s Cas12a patent estate and Broad
Institute and Harvard University’s Cas9 patent estates for human
medicines. For the latest information and scientific presentations,
please visit www.editasmedicine.com.
Forward-Looking Statements This press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995.
The words “anticipate,” “believe,” “continue,” “could,” “estimate,”
“expect,” “intend,” “may,” “plan,” “potential,” “predict,”
“project,” “target,” “should,” “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 the timing
for the Company’s receipt and presentation of data from its
clinical trials and preclinical studies, including further RUBY and
EdiTHAL clinical updates in mid-2024, and the potential of, and
expectations for, the Company’s product candidates. The Company 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 important
factors, including: uncertainties inherent in the initiation and
completion of preclinical studies and clinical trials, including
the RUBY and EdiTHAL trials, and clinical development of the
Company’s product candidates, including reni-cel (EDIT-301);
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; expectations for regulatory approvals to conduct
trials or to market products and availability of funding sufficient
for the Company’s foreseeable and unforeseeable operating expenses
and capital expenditure requirements. These and other risks are
described in greater detail under the caption “Risk Factors”
included in the Company’s most recent Annual Report on Form 10-K,
which is on file with the Securities and Exchange
Commission, as updated by the Company’s subsequent filings
with the Securities and Exchange Commission, and in other
filings that the Company may make with the Securities and Exchange
Commission in the future. Any forward-looking statements contained
in this press release speak only as of the date hereof, and the
Company expressly disclaims any obligation to update any
forward-looking statements, whether because of new information,
future events or otherwise.
Media and Investor Contact:
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
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