- VRDN-001 data demonstrated clinically
meaningful and rapid improvement in signs and symptoms of chronic
TED at week 6 after receiving two infusions of VRDN-001 10 mg/kg or
3 mg/kg –
- Ongoing THRIVE Phase 3 trial in patients with
active TED amended to reflect Viridian’s confidence in 5-dose
regimen and key stakeholder feedback on evolving TED treatment
paradigm -
- THRIVE-2 Phase 3 trial in patients with
chronic TED expected to start in third quarter 2023 –
- 3 mg/kg data support low-volume subcutaneous
(SC) dosing profile for Company’s SC candidates in TED -
- Viridian plans to select lead SC program
candidate by year-end 2023; VRDN-001 SC IND amendment and VRDN-003
IND submitted to the FDA –
- Conference call and webcast to be held today,
Monday, July 10th at 5:00 p.m. ET -
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 preliminary data from its ongoing Phase 1/2
clinical trial of VRDN-001, an investigational full antagonist
antibody to the insulin-like growth factor 1 receptor (IGF-1R), in
patients with chronic thyroid eye disease (TED). The Company also
announced an amendment to its ongoing THRIVE Phase 3 trial design
and provided an update on recent progress of its SC program
candidates in TED.
“It is quite impressive that patients in the 10 mg/kg and 3
mg/kg dose cohorts experienced reductions in proptosis as well as
improvements in their clinical activity scores after receiving just
two infusions of VRDN-001,” said Kimberly Cockerham, M.D., an
Oculoplastic Surgeon specializing in neuro-ophthalmology, orbital
oncology and oculofacial restoration at the SENTA Clinic in San
Diego, California, and an investigator on the VRDN-001 clinical
trial. “Importantly, VRDN-001 was generally well tolerated among
all treated patients, who will continue to be evaluated for safety
and durability of response. Thus far, these data suggest that
VRDN-001 has the potential to become an important new treatment
option for managing the signs and symptoms of TED.”
VRDN-001 – Phase 1/2 proof-of-concept
trial in chronic TED The proof-of-concept portion of the
double-masked, placebo-controlled Phase 1/2 trial evaluated two
infusions of VRDN-001 administered intravenously (IV), three weeks
apart, with clinical activity endpoints measured six weeks after
the first infusion. VRDN-001 was evaluated at doses of 10 and 3
mg/kg, with each cohort designed to include six patients randomized
to drug, and two patients randomized to placebo.
Participant eligibility criteria included: chronic TED with
documented evidence of ocular symptoms or signs that began more
than one year prior to screening (mean duration of 7.8 years), and
proptosis of ≥3 mm above normal values for gender and race. Any
clinical activity score (CAS, 0 – 7) was allowed for randomization
(mean CAS was 3.3).
VRDN-001 – Safety data VRDN-001 was
generally well tolerated by all drug treated patients in both dose
cohorts. There were no reported serious adverse events (SAEs),
including no hearing impairment or hyperglycemia events, in
patients with chronic TED treated with VRDN-001 as of May 30, 2023,
the most recent cutoff date for follow-up observation. The safety
and tolerability profile was generally consistent with previously
reported results in patients with active TED treated with
VRDN-001.
VRDN-001 – Clinical activity data in
chronic TED Patients in the 10 mg/kg (n=6) and 3 mg/kg (n=6)
cohorts who were treated with two doses of VRDN-001 were evaluated
for changes in proptosis, CAS, and diplopia at week 6. Proptosis
changes from baseline were measured both by exophthalmometry and
magnetic resonance imaging (MRI, an exploratory measure). We
believe exophthalmometry and MRI together provide a robust
assessment of changes in proptosis. The following activity was
observed in the 10 mg/kg cohort (n=6), 3mg/kg cohort (n=6), and
across both dose groups (n=12):
Proptosis:
- Patients treated with VRDN-001 had lower mean proptosis at
baseline when compared with placebo (25.0 mm).
VRDN-001
10 mg/kg cohort
(n = 6)
VRDN-001
3 mg/kg cohort
(n = 6)
VRDN-001 combined
10 and 3 mg/kg
(n = 12)
Mean baseline proptosis
21.1 mm
23.4 mm
22.2 mm
Mean reduction in proptosis from
baseline (measured by exophthalmometry)
-1.8 mm
-1.5 mm
-1.6 mm
Mean reduction in proptosis from
baseline (measured by MRI*)
-1.5 mm
-2.6 mm
-2.0 mm
Proptosis responder rate
50%
33%
42%
*Masked, centrally reviewed MRI; MRI data is preliminary, MRI
data available for 4 of 6 VRDN-001 10 mg/kg treated patients, 4 of
6 VRDN-001 3 mg/kg treated patients, and 5 of 5 placebo-treated
patients (mean reduction in proptosis by MRI = -0.2 mm for
placebo).
CAS:
- Observed a 50% to 72% reduction in mean CAS at week 6 compared
with mean baseline levels in patients treated with VRDN-001.
VRDN-001
10 mg/kg cohort
(n = 6)
VRDN-001
3 mg/kg cohort
(n = 6)
VRDN-001 combined
10 and 3 mg/kg
(n = 12)
Mean baseline CAS
2.5
4.0
3.3
Mean reduction in CAS from
baseline, all patients (7-point measure)
-1.8
-2.0
-1.9
Mean reduction in CAS from
baseline, patients CAS>0 at baseline*
-2.8
-2.0
-2.3
*2 patients from the VRDN-001 10 mg/kg cohort with CAS of 0 at
baseline excluded from calculation.
Diplopia:
- Five out of the twelve VRDN-001 treated patients across both
dose cohorts had diplopia (double vision) at baseline. None of the
patients treated with VRDN-001 achieved complete resolution of
diplopia at week 6, defined as patients with baseline diplopia who
achieved a score of 0 on the Gorman subjective diplopia scale.
“We continue to be enthusiastic over the clinical trial data
we’re compiling on VRDN-001,” said Barrett Katz, M.D., M.B.A.,
Chief Medical Officer at Viridian. “As shown previously in our
proof-of-concept study in patients with active TED, and now in
those with chronic disease, VRDN-001 appears to be generally well
tolerated and shows clinically meaningful changes, making it a
promising lead candidate in our mission to develop therapies to
improve the lives of those with TED.”
THRIVE Phase 3 trials in TED
Following recent discussions with the US Food and Drug
Administration (FDA) regarding Viridian’s proposal to amend the
THRIVE Phase 3 trial design, THRIVE will now include the VRDN-001
5-dose treatment regimen and placebo arms only. The trial design
amendment reflects Viridian’s confidence in the 5-dose treatment
regimen of VRDN-001 and takes into consideration key stakeholder
feedback from the TED community expressing preference for a
shortened treatment regimen compared to an 8-dose treatment
regimen.
The primary efficacy endpoint for THRIVE, proptosis responder
rate, will be evaluated at week 15. The Company expects to announce
topline results from the THRIVE Phase 3 trial in the middle of
2024.
Viridian plans to initiate the THRIVE-2 Phase 3 trial to
evaluate the safety and efficacy of VRDN-001 in patients with
chronic TED in the third quarter of 2023. The Company expects to
announce topline results from the THRIVE-2 trial by year-end
2024.
Subcutaneous (SC) programs in
TED Viridian believes that data from the 3 mg/kg dose
cohorts of VRDN-001 in patients with active or chronic TED validate
a low-volume, SC product profile for the Company’s three SC
candidates. The Company’s recent progress and upcoming priorities
for its SC programs, include:
- Completed the SC formulation work for all three SC programs,
allowing for a concentration of 150 mg/mL for administration of a
300 mg/2 mL dose in its SC clinical trials.
- Filed an investigational new drug (IND) application for
VRDN-003 and an IND amendment for VRDN-001 SC with the FDA in June
2023. Following clearance of the submissions, Viridian plans to
initiate Phase 1 trials of VRDN-003 and VRDN-001 SC in healthy
volunteers in the third quarter of 2023, with initial data expected
in the fourth quarter of 2023.
- Completed enrollment of the Phase 1 healthy volunteer trial of
VRDN-002 single IV and single SC dose cohorts.
- Initiation of a pen device supply agreement with an experienced
drug delivery device manufacturer in the second half of 2023.
Viridian expects to select its lead subcutaneous program by
year-end 2023 and to advance the program into a pivotal Phase 2/3
trial in the middle of 2024.
VRDN-001, -002, and -003 are investigational therapies that are
not approved for any use in any country.
Conference call and webcast information
The Company will host a conference call today at 5:00 p.m. ET to
discuss the topline data and program updates in TED. The dial-in
number for the conference call is 1-888-330-3622 for domestic
participants and 1-646-960-0662 for international participants. The
conference ID is 3961606.
A live webcast of the conference call can be accessed through
the “Events” page in the Investors section of the Viridian
Therapeutics website. Following the live webcast, an archived
version of the call will also be available on the website.
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
Phase 2 portion of the ongoing trial established clinical
proof-of-concept for VRDN-001 in patients with active and chronic
TED. VRDN-001 was generally well tolerated in the trial.
The THRIVE Phase 3 trial in patients with active TED is ongoing.
The Company is currently planning to start its second Phase 3
trial, called THRIVE-2, in patients with chronic TED.
The Company is also advancing three candidates (VRDN-001 SC,
VRDN-002, and VRDN-003) designed for administration as convenient,
low-volume, SC injection for the treatment of TED.
Viridian’s goal is to bring a best-in-class IV therapy followed
by a first- and best-in-class SC therapy to the market for the
treatment of the TED.
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. In addition to its intravenously administered VRDN-001
program, the Company is advancing three 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 Statements
This 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 in patients with
chronic Thyroid Eye Disease 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; the Company’s ability to develop an SC
formulation; the Company’s plans regarding a lead SC program
candidate 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; and potential addressable market size,,
including those risks set forth under the caption “Risk Factors” in
the Company’s Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) on May 10, 2023 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20230710883161/en/
Investors: Louisa Stone, 508-808-2400 Manager, Investor
Relations IR@viridiantherapeutics.com
Todd James, 617-272-4691 Senior Vice President, Corporate
Affairs and Investor Relations IR@viridiantherapeutics.com
Media: Matt Fearer, 617-272-4605 Vice President, Corporate
Communications Media@viridiantherapeutics.com
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