Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biopharmaceutical
company focused on discovering and developing potential
best-in-class medicines for serious and rare diseases, today
announced positive topline clinical data from the third, low dose
cohort in its ongoing Phase 1/2 clinical trial of VRDN-001, an
anti-insulin-like growth factor 1 receptor (IGF-1R) antibody, in
patients with active thyroid eye disease (TED). The Company
believes this data further validate the differentiated and
potentially best-in-class clinical activity of VRDN-001. The data
also support the planned dosing interval for Viridian’s VRDN-002
and VRDN-003 subcutaneous programs of up to once monthly.
“The rapid and meaningful improvements in signs and symptoms of
TED observed with a low dose of VRDN-001 reinforce previously
reported findings in this trial, and suggest that VRDN-001 may
offer a differentiated efficacy profile,” said Roger Turbin, M.D.,
Professor of Ophthalmology and Visual Science within the Department
of Ophthalmology of Rutgers New Jersey Medical School, and an
investigator on the VRDN-001 trial. “The data also support
development of VRDN-001 as a patient-friendly low volume
subcutaneous injection, which could reduce the burden of care for
patients suffering from TED.”
VRDN-001 – Phase 1/2 proof-of-concept trialThe
proof-of-concept portion of this double-blind, placebo-controlled
Phase 1/2 trial evaluated two infusions of VRDN-001 administered
intravenously, three weeks apart, with efficacy measured six weeks
after the first dose. VRDN-001 was evaluated at doses of 3, 10, and
20 mg/kg, with each cohort designed to include six patients
randomized to drug, and two patients randomized to placebo. The
Company previously announced positive results from the first two
dose cohorts, which demonstrated a favorable safety profile. The
third cohort evaluated a VRDN-001 dose of 3 mg/kg with 6-week data
announced today. In the 3 mg/kg dose cohort, nine patients were
randomized to receive VRDN-001 to enable all consented patients who
were eligible following screening to participate in the trial, and
two patients were randomized to receive placebo. One patient
receiving placebo discontinued in the trial prior to the 6-week
evaluation.
VRDN-001 – Safety data VRDN-001 was generally
safe and well-tolerated by all patients treated in the three dose
cohorts. There were no reported serious adverse events (SAEs), no
discontinuations, and no infusion reactions in patients treated
with VRDN-001 as of December 19, 2022, the most recent cut-off date
for follow-up observation. The safety and tolerability profile at
the 3 mg/kg dose level was generally consistent with previously
reported results.
VRDN-001 – Clinical activity dataAll VRDN-001
treated patients (n=21) in the 3 mg/kg (n=9), 10 mg/kg (n=6) and 20
mg/kg (n=6) cohorts were treated for two full cycles and were
evaluated for changes in proptosis, clinical activity score (CAS)
and diplopia. Improvement in proptosis and CAS was generally
consistent across the three cohorts. A preliminary analysis of
systemic IGF-1 levels, a biomarker for target engagement, shows a
similar increase was also observed across the three cohorts. The
following activity was observed in the 3mg/kg cohort (n=9) and
across all three dose groups (n=21) at week 6:
Proptosis
- Proptosis responder rate, defined as a ≥2-millimeter (mm)
reduction in proptosis from baseline as measured by
exophthalmometry
- 67% in the 3mg/kg cohort
- 71% across all three dose groups
- Mean reduction in proptosis from baseline as measured by
exophthalmometry
- 2.7 mm in the 3mg/kg cohort
- 2.3 mm across all three dose groups
- Mean reduction in proptosis from baseline as measured by
blinded, centrally reviewed magnetic resonance imaging (MRI)
- 2.8 mm in the 3mg/kg cohort (MRI available for 7 patients)
- 2.76 mm across all three dose groups (MRI available for 16
patients)
Clinical Activity Score (CAS)
- Mean reduction in CAS from baseline on a 7-point measure of
signs and symptoms of TED
- 4.2-points in the 3mg/kg cohort
- 4.1-points across all three dose groups
- Maximal or near-maximal therapeutic effect on CAS, defined as
reaching a CAS of 0 or 1 on the 7-point composite measure of signs
and symptoms of TED
- 67% in the 3mg/kg cohort
- 62% across all three dose groups
Overall response
- Overall responder rate, defined as a ≥2 mm reduction in
proptosis and a ≥2 point reduction in CAS
- 56% in the 3mg/kg cohort
- 67% across all three dose groups
Diplopia
- Complete resolution of diplopia, defined as patients with
baseline diplopia who achieved a score of 0 on the Gorman
subjective diplopia scale
- 20% in the 3mg/kg cohort (5 patients with diplopia at
baseline)
- 54% across all three dose groups (13 patients with diplopia at
baseline)
“Data from this low dose cohort expand our overall data set to
21 drug-treated patients and build additional confidence in our
ongoing Phase 3 ‘THRIVE’ trial evaluating VRDN-001 in patients with
active TED.” said Barrett Katz, MD, MBA, Chief Medical Officer at
Viridian. “This low dose data also increases our confidence in our
planned subcutaneous program, which we are advancing as a
convenient, self-administered pen.”
Subcutaneous programThe Company believes that
data from the 3 mg/kg dose cohort of VRDN-001 validate a low
volume, subcutaneous product profile for the Company’s
next-generation half-life extended anti-IGF-1R antibodies VRDN-002
and VRDN-003.
VRDN-002 is a novel anti-IGF-1R monoclonal antibody that
incorporates half-life extension technology. The Company previously
reported that VRDN-002 demonstrated a half-life up to 43 days in
healthy volunteers, supporting administration as a low-volume,
subcutaneous injection up to once-monthly.
VRDN-003 is an anti-IGF-1R monoclonal antibody with the same
amino acid sequence as VRDN-001, except for the addition of the
half-life extension technology that is incorporated in
VRDN-002.
The Company’s updated pharmacokinetic (PK) modeling support
feasibility of ongoing development of a self-administered pen for
subcutaneous administration, and a planned dosing interval of up to
once-monthly for VRDN-002 and VRDN-003.
A presentation of the VRDN-001 3 mg/kg data is available under
“Events and Presentations” on the Investors section of the Viridian
website at viridiantherapeutics.com.
Upcoming corporate priorities
- Initial VRDN-001 results from a proof-of-concept study in
patients with chronic TED are expected in the first half of
2023
- VRDN-003 IND filing with the US Food and Drug Administration is
planned for the second quarter of 2023, with Phase 1 results in
healthy volunteers expected in the fourth quarter of 2023.
- VRDN-002 results in patients with active TED are expected in
the second half of 2023
- The Company expects to select either the VRDN-002 or VRDN-003
subcutaneous program to advance to a pivotal Phase 3 trial in early
2024.
- Patient enrollment in the global THRIVE Phase 3 trial in
patients with active TED is ongoing and results are expected
mid-2024
About Viridian’s Thyroid Eye Disease Pipeline (VRDN-001,
-002, and -003)
Viridian’s lead product candidate, VRDN-001, is a differentiated
monoclonal antibody targeting insulin-like growth factor-1 receptor
(IGF-1R), a clinically and commercially validated target for the
treatment of thyroid eye disease (TED). In preclinical studies,
VRDN-001 was shown to be a full antagonist of IGF-1R, with more
complete receptor blockade than other anti-IGF-1R antibodies,
including the only currently approved TED therapy. Data from the
initial dose cohorts of the Phase 2 portion of the ongoing trial
established clinical proof-of-concept for VRDN-001 in patients with
active TED. Preliminary data from the ongoing trial showed
treatment with VRDN-001 led to clinically meaningful reductions in
proptosis, improvement in clinical activity score (CAS), and
diplopia resolution. VRDN-001 was generally safe and well tolerated
in the trial. The Company recently initiated its THRIVE Phase 3
trial in patients with active TED to support global marketing
registration.
VRDN-001 is also being evaluated in Phase 2 trial cohorts in
patients with chronic TED. Pending positive results, the Company
plans to start its THRIVE-2 Phase 3 trial in patients with chronic
TED.
The Company is advancing VRDN-002, a distinct anti-IGF-1R
antibody incorporating half-life extension technology, and
VRDN-003, a half-life extended version of VRDN-001. Both VRDN-002
and VRDN-003 are designed for administration as convenient,
low-volume, subcutaneous injections.
VRDN-001, -002, and -003 are investigational therapies that are
not approved for any use in any country.
About TED
TED is a serious and debilitating rare autoimmune disease that
causes inflammation within the orbit of the eye that can cause
double vision, pain, and potential blindness. TED is a progressive
disease consisting of an initial active phase, followed by a
transition to a secondary chronic phase. More than 50,000 and
200,000 people are estimated to suffer from active and chronic TED,
respectively, in the United States and Europe.
About Viridian Therapeutics
Viridian Therapeutics is a biopharmaceutical company focused on
engineering and developing potential best-in-class medicines for
patients with serious and rare diseases. Viridian’s expertise in
antibody discovery and engineering enables it to develop
differentiated therapeutic candidates for previously validated drug
targets in commercially established disease areas.
Viridian is advancing multiple candidates in the clinic for the
treatment of patients with thyroid eye disease (TED). The Company
recently initiated its first global Phase 3 trial called ‘THRIVE’
to evaluate the safety and efficacy of VRDN-001 in patients with
active TED. Viridian is also evaluating VRDN-001 in a Phase 2
proof-of-concept trial in patients with chronic TED. In addition to
its intravenously administered VRDN-001 program, the Company is
advancing two candidates for its subcutaneous strategy with the
goal of providing a more conveniently administered therapy to
patients with TED. Viridian is developing multiple preclinical
assets in autoimmune and rare diseases.
Viridian is based in Waltham, Massachusetts. For more
information, please visit https://www.viridiantherapeutics.com.
Follow Viridian on LinkedIn.
Note Regarding Forward-Looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
These statements may be identified by the use of words such as, but
not limited to, "anticipate," "believe," "continue," "could,"
"estimate," "expect," "intend," "may," "might," "plan,"
"potential," "predict," "project," "should," "target," "will," or
"would" or other similar terms or expressions that concern the
Company’s expectations, plans and intentions. Forward-looking
statements include, without limitation, statements regarding the
Company’s expectations, strategies, plans and intentions.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on the
Company’s current beliefs, expectations, and assumptions. New risks
and uncertainties may emerge from time to time, and it is not
possible to predict all risks and uncertainties. No representations
or warranties (expressed or implied) are made about the accuracy of
any such forward-looking statements. Such forward-looking
statements are subject to a number of material risks and
uncertainties including but not limited to: the potential efficacy
and safety of VRDN-001, VRDN-002 and VRDN-003 for the treatment of
TED; the relationship between the results from the positive data
from the Phase 1/2 clinical trial of VRDN-001 and the results of
ongoing or future clinical trials; the timing, progress and plans
for the Company’s ongoing and future research and clinical
development programs; trial protocols for ongoing or future
clinical trials, including the clinical trials for VRDN-001,
VRDN-002 and VRDN-003; expectations regarding the timing for data;
uncertainty and potential delays related to clinical drug
development; the duration and impact of regulatory delays in the
Company’s clinical programs; manufacturing risks; our ability to
develop a subcutaneous formulation; competition from other
therapies or products; other matters that could affect the
sufficiency of existing cash, cash equivalents and short-term
investments to fund operations; the Company’s financial position
and its projected cash runway; the Company’s future operating
results and financial performance; the timing of pre-clinical and
clinical trial activities and reporting results from same;
potential addressable market size; the effects from the COVID-19
pandemic on the Company’s research, development and business
activities and operating results, including those risks set forth
under the caption “Risk Factors” in the Company’s Annual Report on
Form 10-K filed with the Securities and Exchange
Commission (SEC) on March 11, 2022 and other
subsequent disclosure documents filed with the SEC. Any
forward-looking statement speaks only as of the date on which it
was made. Neither the Company, nor its affiliates, advisors, or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as a result of new
information, future events or otherwise, except as required by law.
These forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date hereof.
Investor and Media ContactTodd JamesViridian
Therapeutics, Inc.Senior Vice President, Corporate Affairs and
Investor Relations617-272-4691IR@viridiantherapeutics.com
Source: Viridian Therapeutics, Inc.
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