Open label study demonstrated statistically
significant effects of early treatment with rivipansel on time to
discharge and time to discontinuation of intravenous opioids
Benefit of early intervention with E-selectin
antagonist in acute VOC now reproduced in independent,
contemporaneous dataset that includes both the all-ages and
pediatric populations
Company expects to initiate Investigational New
Drug-enabling work with GMI-1687 during 2021
GlycoMimetics (Nasdaq: GLYC) today in an oral presentation at
the 62nd American Society of Hematology (ASH) Annual Meeting and
Exposition disclosed new data reinforcing a now comprehensive set
of findings that underscore the improvement of rivipansel when
administered early during an acute vaso-occlusive crisis (VOC) in
individuals with sickle cell disease (SCD). The new data were part
of an analysis of an Open Label Extension (OLE) trial of
rivipansel, designed to evaluate real-world data on safety and
efficacy in patients treated first in the RESET Phase 3 trial
conducted by the company’s former collaborator Pfizer. The OLE
trial ran in parallel to the RESET trial and provides an
independent, contemporaneous dataset for comparison of clinically
meaningful efficacy outcomes with rivipansel. The data from the OLE
efficacy analysis, together with more extensive data from the RESET
trial post-hoc analysis previously reported, were presented at ASH
by University of California Davis’ Theodore Wun, M.D., Interim Vice
Dean for Research, Associate Dean for Research, School of Medicine;
Director, Clinical and Translational Science Center; and Chief,
Division of Hematology and Oncology.
In his oral presentation, Dr. Wun reviewed key findings from
several post hoc analyses of the Phase 3 RESET trial and a
subsequent pre-planned analysis of the OLE trial. Specifically,
these included the achievement of statistically significant
improvements in primary and key secondary endpoints, with early
treatment in the pediatric subgroup as well as the all-ages
treatment group and importantly, on several key secondary
endpoints, including time to discharge (TTD) and time to
discontinuation of IV opioids (TTDIVO).
Dr. Wun’s presentation highlighted data from the OLE trial
reproducing the RESET efficacy outcomes for patients treated early
with rivipansel. For the all-ages study population:
- On duration of hospital stay, the median TTD with early
rivipansel treatment in the OLE study was 80.89 hours compared to
103.97 hours with early placebo in the RESET study, a difference of
nearly one day (23.08 hours). This difference achieved significance
at the pre-specified 90% confidence level, with a P-value of
0.0617; and
- On duration of IV opioid use, the median TTDIVO for early
rivipansel treatment in the OLE study was 63.87 hours compared to
93.99 hours for the early placebo in the RESET study, a difference
of more than one day (30.12 hours). This difference achieved
significance at the pre-specified 90% confidence level, with a
P-value of 0.0867.
Dr. Wun also disclosed data for the pediatric population in the
RESET trial, representing 41% of the total data set, including
that:
- Children treated with rivipansel within 30 hours of onset of
VOC experienced a reduction in the primary endpoint median TTRFD by
29.3 hours (from 94.1 to 64.8 hours); and
- Early treatment with rivipansel led to more children ready for
discharge from the hospital by 24, 48, and 72 hours.
Rachel King, GlycoMimetics’ Chief Executive Officer, noted, “The
OLE analysis of efficacy outcomes for early treatment with
rivipansel confirms the observations made in post-hoc assessment of
the RESET trial, and does so for two important, clinically
meaningful endpoints. The consistency of these findings clearly
supports the validity of E-selectin as a target, our product
candidate’s ability to hit the target, and the use of an E-selectin
antagonist for treating patients early in VOC. While we fully
expect that additional clinical data will be required to achieve
registration for rivipansel, we plan to continue interactions with
the FDA to determine the clinical and regulatory path forward prior
to making any decision as to how or whether we will proceed in this
acute setting. As Dr. Wun presented at ASH, there remains a need
for therapy that will abrogate VOC and reduce the duration of
hospitalization and opioid use. We are unaware of any late-stage
clinical programs targeting the resolution of acute vaso-occlusive
episodes. In sum, our data point to the potential for early
administration of rivipansel to change the treatment paradigm from
delayed palliation to early intervention to reduce length of
hospitalization and IV opioid requirement.”
The OLE study results, presented for the first time at ASH,
began in December of 2015 and treated 81 patients ages six years or
older who had completed the double-blind Phase 3 RESET study. The
study was designed to evaluate the safety of rivipansel as a
treatment for one or more VOC events in hospitalized subjects with
SCD. In this context, the OLE protocol originally included
collection of data for TTD and TTDIVO analyses as part of the
safety assessment. Upon completion of the post hoc analysis of the
RESET trial dataset, GlycoMimetics recognized the opportunity to
leverage the OLE as a contemporaneous clinical trial to confirm the
RESET study early treatment efficacy findings for TTD and TTDIVO.
GlycoMimetics developed and submitted a Statistical Analysis
Addendum (SAA) to FDA to compare the single-arm OLE data to the
RESET trial data to assess if the RESET observations were
reproducible. The SAA was implemented prior to final reporting of
the OLE study datasets and outcomes by Pfizer, allowing the OLE
dataset to be evaluated for efficacy outcomes in a pre-planned
analysis. In effect, a prospectively planned and executed OLE
safety study was converted prior to final analysis by GlycoMimetics
into a study including efficacy outcomes. This allowed independent
confirmation of the improvements in TTD and TTDIVO observed with
early rivipansel treatment in acute VOC, using a contemporaneous
single-arm dataset.
In addition to the rivipansel data, GlycoMimetics also presented
preclinical data in another oral presentation at the ASH meeting
showing that GMI-1687, the Company’s highly potent E-selectin
antagonist, was active in restoring blood flow in a mouse model of
VOC when administered subcutaneously. “With data from the
rivipansel program supporting targeting of E-selectin early in
acute vaso-occlusive crisis, a subcutaneously administered
E-selectin antagonist such as GMI-1687 could be ideally suited for
self-administration by patients,” Rachel King added.
GlycoMimetics expects to initiate Investigational New Drug
enabling work with GMI-1687 during 2021.
The presentation will be available on the Company’s website at
www.glycomimetics.com under Scientific Publications, and
Rivipansel. Details of the presentation are as follows:
Title: Early Initiation of Treatment with Rivipansel for
Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves
Earlier Discontinuation of IV Opioids and Shorter Hospital Stay:
Reset Clinical Trial Analysis (abstract #678) Presenter:
Theodore Wun, M.D., University of California Davis, Interim Vice
Dean for Research, Associate Dean for Research, School of Medicine;
Director, Clinical and Translational Science Center; and Chief,
Division of Hematology and Oncology. Session Name: 114
Hemoglobinopathies, Excluding Thalassemia—Clinical: Novel
Treatments for Sickle Cell Disease Date and Time: Monday,
December 7, 2020. 4:30 – 6:00 p.m. ET Presentation
Time: 4:45 p.m. ET
About Rivipansel
Rivipansel, the Company’s wholly-owned glycomimetic drug
candidate that binds to all three members of the selectin family
(E-, P- and L-selectin), was GlycoMimetics’ first drug candidate to
enter clinical development. After the Phase 3 RESET trial conducted
by Pfizer, GlycoMimetics’ former collaborator, did not meet its
primary or key secondary efficacy endpoints in 2019, new efficacy
data from a post hoc analysis of rivipansel were published in June
2020 and subsequently presented at the Foundation for Sickle Cell
Disease Research Meeting in September 2020. Additional new efficacy
data from an Open Label Extension trial of rivipansel were
presented at the 62nd American Society of Hematology Annual Meeting
and Exposition in December 2020. GlycoMimetics is engaging with the
FDA to identify what, if any, next steps to take in connection with
the development of rivipansel as a treatment for vaso-occlusive
crisis of sickle cell disease.
About GMI-1687
Discovered and developed by GlycoMimetics, GMI-1687 is a
highly-targeted, highly-potent E-selectin antagonist. It has been
shown in preclinical studies to be bioavailable via subcutaneous
administration. During 2020, data from oral presentations at major
scientific conferences pointed to the potential for a
self-administered drug to treat vaso-occlusive crisis of sickle
cell disease. The investigational drug represents a potential life
cycle extension opportunity for GlycoMimetics’ drug candidates
rivipansel and uproleselan.
About GlycoMimetics, Inc.
GlycoMimetics is a biotechnology company with two late-stage
clinical development programs and a pipeline of novel glycomimetic
drugs, all designed to address unmet medical needs resulting from
diseases in which carbohydrate biology plays a key role.
GlycoMimetics' drug candidate, uproleselan, an E-selectin
antagonist, was evaluated in a Phase 1/2 clinical trial as a
potential treatment for AML and is being evaluated across a range
of patient populations including a Company-sponsored Phase 3 trial
in relapsed/refractory AML under Breakthrough Therapy designation.
Rivipansel, a pan-selectin antagonist, is being explored for use in
treatment of acute VOC in sickle cell disease. GlycoMimetics has
also completed a Phase 1 clinical trial with another wholly-owned
drug candidate, GMI-1359, a combined CXCR4 and E-selectin
antagonist. GlycoMimetics is located in Rockville, MD in the
BioHealth Capital Region. Learn more at www.glycomimetics.com.
Forward-Looking Statements
This press release contains forward-looking statements. These
forward-looking statements include those relating to the planned or
potential clinical development of the Company’s product candidates,
including the Company’s engagement with regulatory authorities, as
well as the presentation of data from preclinical studies and
clinical trials and the potential benefits and impact of the
Company’s drug candidates. Actual results may differ materially
from those described in these forward-looking statements. For a
further description of the risks associated with these statements,
as well as other risks facing GlycoMimetics, please see the risk
factors described in the Company’s annual report on Form 10-K filed
with the U.S. Securities and Exchange Commission (SEC) on February
28, 2020, and other filings GlycoMimetics makes with the SEC from
time to time. Forward-looking statements speak only as of the date
of this release, and GlycoMimetics undertakes no obligation to
update or revise these statements, except as may be required by
law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201207005791/en/
Investor Contact: Shari Annes Phone: 650-888-0902 Email:
sannes@annesassociates.com
Media Contact: Jamie Lacey-Moreira Phone: 410-299-3310 Email:
jamielacey@presscommpr.com
GlycoMimetics (NASDAQ:GLYC)
Historical Stock Chart
From Mar 2024 to Apr 2024
GlycoMimetics (NASDAQ:GLYC)
Historical Stock Chart
From Apr 2023 to Apr 2024