Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced positive initial safety and
efficacy data from the first four patients with sickle cell disease
(SCD) treated with EDIT-301 in the RUBY trial and from the first
transfusion-dependent beta thalassemia patient treated in the
EdiTHAL trial.
The RUBY trial data will be presented in an oral presentation at
European Hematology Association (EHA) Hybrid Congress in Frankfurt,
Germany, and via live stream on Saturday, June 10, at 5:30 p.m.
CEST/11:30 a.m. EDT. Editas Medicine will present the RUBY trial
data and the EdiTHAL trial data on Monday, June 12, at 8 a.m. ET in
a Company-sponsored webinar.
In the RUBY trial, Patients 1 and 2 reached normal hemoglobin
levels five months post-treatment with EDIT-301, and both patients
have maintained a normal hemoglobin level at ten- and six-month
follow-up, respectively. Additionally, each of these patients has
seen fetal hemoglobin levels of greater than 40% persist during the
same time frame. Patients 3 and 4 in the RUBY trial saw increases
in total hemoglobin and fetal hemoglobin at three and two months of
follow up, respectively, that follow similar trajectories as those
seen in the first two patients. All four treated RUBY patients are
also free of vaso-occlusive events (VOEs) since infusion.
In the EdiTHAL trial, the first patient demonstrated successful
neutrophil and platelet engraftment, and, at one and a half months
post-infusion, the patient’s response resembles that of the first
four RUBY patients.
EDIT-301 was well-tolerated and demonstrated a safety profile
consistent with myeloablative conditioning with busulfan and
autologous hematopoietic stem cell transplant by the four patients
in the RUBY trial and the first patient in the EdiTHAL trial. After
EDIT-301 infusion, no serious adverse events occurred, and no
adverse events reported were related to treatment with
EDIT-301.
RUBY TrialSafetyAll treated
patients demonstrated successful neutrophil and platelet
engraftment. No serious adverse events (SAEs) were reported after
EDIT-301 infusion, and no adverse events (AEs) related to EDIT-301
treatment have been observed. All four treated patients in the RUBY
trial are free from vaso-occlusive events since infusion with
EDIT-301, with 2-10 months follow-up.
EfficacyPatient 1 (male) has had 10 months of
follow-up. Patient 1’s total hemoglobin returned to normal
physiological level of 16.4g/dL (male normal range: 13.6–18.0 g/dL)
at five months after infusion of EDIT-301 and has been maintained
at this level at the 10-month follow-up. In addition, Patient 1’s
fetal hemoglobin fraction increased from 5% at baseline to 45.4%
five months after treatment with EDIT-301 and 43.4% at the 10-month
follow-up.
Patient 2 (female) has had 6 months of follow-up. Patient 2’s
total hemoglobin reached normal physiological level of 12.7 g/dL
(female normal range: 12.0–16.0 g/dL) at five months after infusion
of EDIT-301 and fetal hemoglobin increased from 10.8% at baseline
to 51.3% at the 6-month follow-up.
Patient 3 (female) and Patient 4 (male) have had three and two
months of follow-up, respectively. Increases in total hemoglobin
and fetal hemoglobin fractions for Patient’s 3 and 4 are following
similar trajectories as seen in Patients 1 and 2 at the same
timepoints.
EdiTHAL TrialSafetyThe first
treated patient (male) in the EdiTHAL trial has had 1.5 months of
follow-up. Patient 1 had successful neutrophil and platelet
engraftment within 30 days of EDIT-301 infusion, and the safety
profile has been consistent with that of myeloablative busulfan
conditioning and autologous hematopoietic stem cell transplant. No
serious adverse events occurred after EDIT-301 infusion, and no
adverse events reported were related to treatment with
EDIT-301.
EfficacyThe first EdiTHAL patient’s experience
with EDIT-301 resembles that of the first four RUBY patients,
achieving a fetal hemoglobin fraction of 34.9% representing 4 g/dL
of total hemoglobin at 1.5 months post-treatment.
“These promising data support our belief that EDIT-301 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 long term, 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 remain on-track to
dose 20 RUBY patients by year-end, and we look forward to sharing
further RUBY and EdiTHAL clinical updates later this year.”
“I am encouraged by the results from the RUBY trial, which
indicate this investigational gene editing treatment has been
well-tolerated and efficacious in treated trial participants thus
far. While we need to let the trial finish and enroll many
other patients to understand the overall benefits and risks of this
treatment, I am pleased to see transformative results at this stage
in the study,” said Rabi Hanna, M.D., Department of Pediatric
Hematology Oncology and Blood and Marrow Transplantation, Cleveland
Clinic Children’s.
EDIT-301 uses AsCas12a, a novel, proprietary, highly efficient,
and specific gene editing nuclease, to edit the promoter regions of
gamma globin gene 1 and 2 to increase the expression of HbF to
mimic the naturally occurring mechanism of hereditary persistence
of fetal hemoglobin to treat SCD. The RUBY trial marks the first
time AsCas12a was used to successfully edit human cells in a
clinical trial.
EHA Oral Presentation Details:
Title: EDIT-301 Shows Promising Preliminary Safety
and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of
Patients with Severe Sickle Cell Disease Using Highly Specific and
Efficient AsCas12a Enzyme 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: Saturday, June 10,
2023, 4:30 – 5:45 p.m. CEST/ 10:30 – 11:45 a.m.
EDTLocation: Harmonie 1, Messe
FrankfurtSession: s437 Gene therapy and cellular
immunotherapy – Clinical
Virtual Event InformationEditas Medicine will
host a virtual event on Monday, June 12, at 8:00 a.m. ET to present
the data for the RUBY and EdiTHAL trials. The live and archived
webcast of the presentation will be accessible through this webcast
link, or through the Events & Presentations page of the
“Investors” section of the Company’s website.
EDIT-301 is currently being investigated in clinical studies in
patients with severe sickle cell disease (RUBY trial, NCT04853576)
and transfusion-dependent beta thalassemia (EDITHAL trial,
NCT05444894). In addition to the clinical data update from the RUBY
trial at EHA and in a Company-sponsored webinar, the Company
expects to present a further clinical update from the RUBY and
EdiTHAL trials by year-end.
About Sickle Cell DiseaseSickle cell disease is
an inherited blood disorder caused by a mutation in the beta-globin
gene that leads to polymerization of the sickle hemoglobin protein
(HbS). In sickle cell disease, the red blood cells are misshapen in
a sickle shape instead of a typical disc shape. The abnormal shape
causes the red blood cells (RBCs) to have shortened lifespan and to
block blood flow causing anemia, pain crises, organ failure, and
early death. There are an estimated 100,000 people in the United
States currently living with sickle cell disease. Higher levels of
fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing
the clinical manifestation of RBCs sickling.
About Beta Thalassemia Beta thalassemia is an
inherited blood disorder caused by mutations that reduce or
abrogate beta globin gene expression. Insufficient beta globin
production leads to ineffective red blood cell production, chronic
hemolytic anemia, compensatory extramedullary hematopoiesis
(creation of blood cells outside of the bone marrow), and
requirement for regular blood transfusion support in patients with
transfusion-dependent beta thalassemia (TDT). TDT is the most
severe form of beta thalassemia, and chronic red blood cell
transfusions are complicated by iron overload leading to organ
dysfunction and failure. It is estimated that there are
approximately 1,000 people in the United States currently living
with transfusion-dependent beta thalassemia. Higher levels of fetal
hemoglobin (HbF) ameliorate anemia thereby reducing the need for
regular red blood cell transfusions.
About EDIT-301EDIT-301 is an experimental cell
therapy medicine under investigation for the treatment of severe
sickle cell disease (SCD) and transfusion-dependent beta
thalassemia (TDT). EDIT-301 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 a highly specific
and efficient proprietary engineered AsCas12a nuclease. Red blood
cells derived from EDIT-301 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 RUBYThe RUBY trial is a single-arm,
open-label, multi-center Phase 1/2 study designed to assess the
safety and efficacy of a single administration of EDIT-301 in
patients with severe sickle cell disease. Additional details are
available on www.clinicaltrials.gov (NCT# 04853576).
About EDITHALThe EDITHAL trial is a single-arm,
open label, multi-center Phase 1/2 study designed to assess the
safety and efficacy of a single administration of EDIT-301 in
patients with transfusion-dependent beta thalassemia. 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
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 StatementsThis press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995,
including statements regarding the Company’s plans to continue
development of EDIT-301 and share further clinical updates in 2023,
and its belief about EDIT-301’s potential for clinical
differentiation. 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. 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 trial, and clinical development of the
Company’s product candidates, including 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
ir@editasmed.com
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