- Veligrotug (VRDN-001) achieved all primary
and secondary endpoints in THRIVE, the largest phase 3 trial
conducted to date of an anti-IGF-1R antibody in thyroid eye disease
(TED), with a week 15 proptosis responder rate (PRR) of 70% and a
placebo-adjusted PRR of 64% (p < 0.0001) -
- All secondary endpoints were highly
statistically significant (p < 0.0001), with clinically
meaningful patient outcomes, including complete resolution of
diplopia in 54% of patients (placebo-adjusted rate of 43%) and
reduction of Clinical Activity Score (CAS) to 0 or 1 in 64% of
patients (placebo-adjusted reduction of 46%) treated with
veligrotug -
- Veligrotug was generally well-tolerated with
no treatment-related serious adverse events (SAEs), and a 5.5%
placebo-adjusted rate of hearing impairment AEs -
- THRIVE-2 study of veligrotug in patients with
chronic TED fully enrolled with topline data readout on track for
year-end 2024; BLA submission anticipated in second half of 2025,
as planned -
- REVEAL-1 and REVEAL-2, global phase 3 trials
of VRDN-003 dosed every four (Q4W) or eight weeks (Q8W), initiated
in August; VRDN-003 is a subcutaneous, half-life-extended
anti-IGF-1R antibody with the same binding domain as veligrotug
-
- Cash, cash equivalents, and short-term
investments of $571.4 million as of June 30, 2024; provides cash
runway into the second half of 2026, beyond the planned veligrotug
BLA submission, expected REVEAL topline data, and multiple
anticipated readouts from Viridian’s FcRn inhibitor programs -
- Conference call and webcast to be held today,
September 10th at 8:00 a.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 topline data from the THRIVE phase 3 clinical
trial of VRDN-001, now known as veligrotug, an intravenously
delivered anti-insulin-like growth factor-1 receptor (IGF-1R)
antibody, in patients with active thyroid eye disease (TED). TED is
an autoimmune condition characterized by inflammation, growth, and
damage to tissues around and behind the eyes.
“We are thrilled to see these exciting THRIVE topline results
which met our high bars for both efficacy and safety. We were
particularly pleased to observe the rapid onset of clinically
meaningful responses across all endpoints, and veligrotug’s safety
profile exceeded our expectations. Veligrotug utilizes a
differentiated, abbreviated five infusion dosing regimen that has
the potential to bring a more convenient treatment option with
lower IV burden for patients, compared to the current standard of
care,” said Steve Mahoney, Viridian’s President and CEO. “Our
excitement with the THRIVE results extends well beyond veligrotug
to the other half of our clinical TED franchise, subcutaneously
administered VRDN-003, for which we initiated two phase 3 clinical
trials last month. We believe veligrotug and VRDN-003 have the
potential to become the preferred treatments for TED patients.
Further, the strong THRIVE results reinforce our confidence in
VRDN-003, which shares the same binding domain as veligrotug, as a
potential best-in-class infrequent subcutaneous injection that we
believe will be transformative for TED patients and expand the
market. Our team has executed extremely effectively with both
THRIVE and THRIVE-2, exceeding enrollment targets in both studies
and across geographies including in the United States, which gives
us confidence that we will swiftly enroll the VRDN-003 trials,
REVEAL-1 and REVEAL-2. In addition to our TED franchise, we are
excited about our emerging FcRn inhibitor portfolio that we look
forward to discussing more later this year.”
“These phase 3 clinical data demonstrate the robust clinical
activity of veligrotug. The data showed substantial improvements in
proptosis, clinical activity score, and diplopia in patients with
TED after just five infusions, and these strong results are
consistent with the clinical outcomes of IGF-1R antagonism in TED.
Veligrotug also showed a rapid onset of action which I believe is
meaningful for patients,” said Michael Yen, M.D., an investigator
in THRIVE and Professor of Oculoplastic Surgery and Ophthalmology,
at Baylor College of Medicine. “Veligrotug showed a favorable
safety profile in a large phase 3 clinical trial that closely
monitored safety, including the potential for hearing impairment.
As a THRIVE investigator, I am excited to see these results and
look forward to the REVEAL clinical trials for VRDN-003 to help
bring additional potential treatment options for patients living
with TED.”
VRDN-001 THRIVE Phase 3 Topline Results
THRIVE Clinical Activity Data
THRIVE met the primary and all secondary endpoints at 15 weeks
after five infusions of veligrotug, showing highly statistically
significant (p < 0.0001) improvements on all of the measured
signs and symptoms of TED. Veligrotug additionally showed a rapid
onset of action, with the majority (53%) of veligrotug-treated
patients achieving a proptosis response after just 1 infusion, or 3
weeks after start of therapy. THRIVE enrolled 113 patients,
randomized to veligrotug (n=75) and placebo (n=38).
Patients receiving veligrotug had statistically significant and
clinically meaningful improvements across the following key disease
endpoints at the primary efficacy analysis timepoint of 15
weeks:
Proptosis:
- Proptosis Responder Rate: 70%
proptosis responder rate (PRR) in patients receiving veligrotug,
compared with 5% of patients receiving placebo (64%
placebo-adjusted) (p < 0.0001). PRR is defined as at least a
2-millimeter (mm) reduction in proptosis from baseline in the study
eye without worsening in the fellow eye (≥2 mm increase), as
measured by exophthalmometry.
- Proptosis Mean Reduction: 2.9 mm
mean reduction in proptosis from baseline in patients receiving
veligrotug, compared with 0.5mm reduction in patients receiving
placebo (2.4mm placebo-adjusted) (p < 0.0001), as measured by
exophthalmometry.
Diplopia:
- Diplopia Complete Resolution: 54%
complete resolution of diplopia in patients receiving veligrotug,
compared with 12% of patients receiving placebo (43%
placebo-adjusted) (p < 0.0001). Diplopia resolution is defined
by patients achieving a score of 0 on the Gorman subjective
diplopia scale at week 15, among patients with diplopia at baseline
(n=76).
- Diplopia Response: 63% achieved a
diplopia response in patients receiving veligrotug, compared with
20% of patients receiving placebo (43% placebo-adjusted) (p <
0.0001). Diplopia response is defined by patients achieving a
reduction of at least 1 on the Gorman subjective diplopia scale at
week 15, among patients with diplopia at baseline.
Clinical Activity Score
(CAS):
- CAS Reduction to 0 or 1: 64% of
patients receiving veligrotug achieved maximal or near-maximal
therapeutic effect on CAS, compared with 18% of patients receiving
placebo (46% placebo-adjusted) (p < 0.0001), defined as reaching
a CAS of 0 or 1. CAS measures inflammatory signs and symptoms of
TED, providing a composite score of pain, as well as redness and
swelling of the eyelids and conjunctiva, on a scale from 0 to
7.
- CAS Mean Reduction: 3.4-point mean
reduction in CAS from baseline for patients receiving veligrotug,
compared with 1.7-point reduction in patients receiving placebo
(1.7-point placebo-adjusted) (p < 0.0001).
Overall Response:
- Overall Responder Rate: 67% of
patients receiving veligrotug achieved an overall response,
compared with 5% of patients receiving placebo (61%
placebo-adjusted) (p < 0.0001). Overall Responder Rate is
defined as achieving a proptosis response and a ≥2-point reduction
in CAS from baseline without worsening in the fellow eye in either
proptosis (2mm increase) or CAS (2-point increase).
THRIVE Safety Data
- Generally Well-Tolerated:
Veligrotug was generally well-tolerated with a safety profile
consistent with previous veligrotug studies. The majority of
adverse events (AEs) were mild, and there was a low rate (4%) of
discontinuations in the veligrotug arm. There were no
treatment-related serious AEs.
- Low Rate of Hearing Impairment:
There was a 5.5% placebo-adjusted rate of hearing impairment AEs in
THRIVE (16% incidence in patients receiving veligrotug, compared
with 10.5% incidence in patients receiving placebo).
Veligrotug: On-Track to Submit Biologics License Application
(BLA) in Second Half of 2025
The second phase 3 clinical trial of veligrotug, THRIVE-2, in
patients with chronic TED is ongoing. Viridian completed enrollment
of THRIVE-2 in July 2024, and topline data readout is on track for
year-end 2024. Viridian anticipates submitting a BLA for veligrotug
for the treatment of TED in 2H 2025, as planned.
VRDN-003 REVEAL Clinical Trials Initiated in August;
Potential Best-in-Class Program on Track for BLA Submission by
Year-end 2026, Approximately One Year After Veligrotug BLA
Submission
VRDN-003 is an IGF-1R antibody with the same binding domain as
veligrotug and is believed to be the only anti-IGF-1R in
development with an extended half-life.
Viridian believes these topline results from THRIVE provide
strong support for a potential best-in-class profile of VRDN-003,
with the potential to deliver clinical efficacy and safety
consistent with veligrotug in a low-volume, infrequent,
self-administered, subcutaneous injection that patients take at
home.
Viridian initiated two global phase 3 clinical trials for
VRDN-003 in August as planned: REVEAL-1 and REVEAL-2 in active and
chronic TED, respectively. Both trials will evaluate VRDN-003
subcutaneously administered every Q4W or Q8W and will assess
outcomes versus placebo. Viridian anticipates topline data from
both trials in the first half of 2026, with a BLA submission for
VRDN-003 for the treatment of TED by year-end 2026.
Conference call and webcast information
Viridian will host a conference call today at 8:00 a.m. ET to
discuss the THRIVE topline data. The dial-in number for the
conference call is (800) 715-9871 for domestic participants and
(646) 307-1963 for international participants. The conference ID is
8636908.
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 Therapeutics
Viridian 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 protein engineering enables the development of
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
is conducting a pivotal program for veligrotug (VRDN-001),
including two global phase 3 clinical trials (THRIVE and THRIVE-2),
to evaluate its efficacy and safety in patients with active and
chronic TED. Viridian is also advancing VRDN-003 as a potential
best-in-class subcutaneous therapy for the treatment of TED,
including two ongoing global phase 3 pivotal clinical trials,
REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of
VRDN-003 in patients with active and chronic TED.
In addition to its TED portfolio, Viridian is advancing a novel
portfolio of neonatal Fc receptor (FcRn) inhibitors, including
VRDN-006 and VRDN-008, which has the potential to be developed in
multiple autoimmune diseases.
Viridian is based in Waltham, Massachusetts. For more
information, please visit www.viridiantherapeutics.com. Follow
Viridian on LinkedIn and X.
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,” “on
track,” “plan,” “potential,” “predict,” “project,” “design,”
“should,” “target,” “will,” or “would” or other similar terms or
expressions that concern our expectations, plans and intentions.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on our
current beliefs, expectations, and assumptions. Forward-looking
statements include, without limitation, statements regarding:
preclinical and clinical development of Viridian’s product
candidates veligrotug (formerly VRDN-001), VRDN-003, VRDN-006 and
VRDN-008, including Viridian's view that the THRIVE data provide
strong support for VRDN-003's clinical profile; anticipated
enrollment timeline in VRDN-003 trials, REVEAL-1 and REVEAL-2;
milestones; timelines; anticipated data results and timing of their
disclosure, including topline results; regulatory interactions and
anticipated timing of regulatory submissions, including the
anticipated BLA submission for veligrotug; Viridian’s expectation
that its data package will support a BLA submission for veligrotug
in the second half of 2025, pending data; Viridian’s expectation
that its data package will support marketing authorization
applications in Europe for veligrotug; clinical trial designs,
including the REVEAL-1 and REVEAL-2, global phase 3 clinical trials
for VRDN-003; Viridian’s plans to launch VRDN-003, if approved; the
potential utility, efficacy, potency, safety, clinical benefits,
clinical response, convenience and number of indications of
veligrotug, VRDN-003, VRDN-006 and VRDN-008; Viridian’s product
candidates potentially being best-in-class and the preferred
treatment for TED patients; and that Viridian’s cash, cash
equivalents and short-term investments will be sufficient to fund
its operations into the second half of 2026.
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: potential
utility, efficacy, potency, safety, clinical benefits, clinical
response and convenience of Viridian’s product candidates; that
results or data from completed or ongoing clinical trials may not
be representative of the results of ongoing or future clinical
trials; that preliminary data may not be representative of final
data; the timing, progress and plans for our ongoing or future
research, preclinical and clinical development programs; changes to
trial protocols for ongoing or new clinical trials, including
adjustments that we may make to the VRDN-003 clinical trial designs
as a result of the veligrotug data; expectations and changes
regarding the timing for regulatory filings; regulatory
interactions expectations and changes regarding the timing for
enrollment and data; uncertainty and potential delays related to
clinical drug development; the duration and impact of regulatory
delays in our clinical programs; the timing of and our ability to
obtain and maintain regulatory approvals for our therapeutic
candidates; manufacturing risks; competition from other therapies
or products; estimates of market size; other matters that could
affect the sufficiency of existing cash, cash equivalents and
short-term investments to fund operations; our financial position
and projected cash runway; our future operating results and
financial performance; Viridian’s intellectual property position;
the timing of preclinical and clinical trial activities and
reporting results from same; and those risks set forth under the
caption “Risk Factors” in our most recent quarterly report on Form
10-Q for the quarter ended June 30, 2024, filed with the Securities
and Exchange Commission (SEC) on August 8, 2024 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240910052986/en/
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
Viridian Therapeutics (NASDAQ:VRDN)
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