- Veligrotug met all primary and secondary
endpoints with high statistical significance in THRIVE-2, achieving
a week 15 proptosis responder rate (PRR) of 56% (placebo-adjusted
PRR of 48%, p < 0.0001) -
- THRIVE-2 is the first global phase 3 study in
patients with chronic TED to demonstrate a statistically
significant and clinically meaningful 56% diplopia responder rate
(placebo-adjusted rate of 31%, p = 0.0006) and 32% rate of diplopia
complete resolution (placebo-adjusted rate of 18%, p = 0.0152)
-
- Veligrotug was generally well-tolerated with
94% of patients completing their treatment course and a 9.6%
placebo-adjusted rate of hearing impairment -
- BLA submission for veligrotug on track for
second half of 2025; potential to transform the standard of care in
TED with differentiated clinical profile demonstrated in the
broadest population studied in a global phase 3 study across active
and chronic TED patients to date -
- REVEAL-1 and REVEAL-2, global phase 3
clinical trials of subcutaneous VRDN-003 evaluating every four-week
(Q4W) or every eight-week (Q8W) regimens, are both currently dosing
and on track for topline data in 1H 2026; VRDN-003 is a
half-life-extended anti-IGF-1R antibody with the same binding
domain as veligrotug -
- Strong cash position of $753 million as of
September 30, 2024; provides cash runway into the second half of
2027 through the anticipated commercial launch of veligrotug,
topline results and BLA submission for subcutaneous VRDN-003, and
multiple FcRn inhibitor clinical catalysts -
- Conference call and webcast to be held today,
December 16th 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-2 phase 3 clinical
trial of veligrotug (veli), an intravenously (IV) delivered
anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, in
patients with chronic thyroid eye disease (TED). TED is an
autoimmune condition characterized by inflammation, growth, and
damage to tissues around and behind the eyes.
“We are extremely pleased to announce better-than-expected
THRIVE-2 results generated in the broadest population of chronic
TED patients studied in a global phase 3 study to date. We believe
that these efficacy and safety results in only five infusions,
combined with our compelling data from THRIVE, confirm the
potential of veli to be the treatment-of-choice for all forms of
active and chronic TED,” said Steve Mahoney, Viridian’s President
and CEO. “The robustness and consistency of our data, similar to
THRIVE, showed strong and rapid responses in categories that we
believe matter most to patients including proptosis reduction,
diplopia resolution and improvements in Clinical Activity Scores.
This is the first product candidate to demonstrate a diplopia
response and resolution rate in a global chronic TED phase 3 study.
Our BLA preparation work is underway, and, if approved, we now
believe veli will offer a differentiated commercial product profile
to patients. The combined results of THRIVE and THRIVE-2, give us
even higher conviction that our subcutaneous VRDN-003 program will
deliver positive topline data in the first half of 2026, which
would enable a BLA submission in the second half of 2026.”
“These data represent an incredible step forward for TED
patients. I’ve been treating TED for over 30 years, and these
results in the broadest population of TED patients are highly
encouraging. Resolving double vision or even improving it in
chronic TED patients can really change their lives,” said Steven
Leibowitz, M.D., Associate Clinical Professor of Ophthalmology,
Stein Eye Institute, University of California Los Angeles and
THRIVE-2 investigator. “I see veligrotug’s potential product
profile as highly compelling with a rapid onset of treatment
effect, diplopia benefit across a broad TED population, shorter
dosing regimen, and favorable safety profile.”
THRIVE-2 Phase 3 Topline Results
THRIVE-2 Clinical Activity Data
THRIVE-2 met all primary and secondary endpoints at the 15-week
primary analysis timepoint after five infusions of veligrotug,
showing statistically significant responses on all of the measured
signs and symptoms of TED: proptosis, CAS, and diplopia. THRIVE-2
enrolled a total of 188 patients, randomized to veligrotug (n =
125) and placebo (n = 63). The mean time since onset of TED in this
patient population was 69.8 months.
The key data at the primary efficacy analysis timepoint of 15
weeks are as follows:
Proptosis:
- Proptosis Responder Rate (PRR):
56% in veligrotug patients, compared with 8% in placebo patients
(48% placebo-adjusted, p < 0.0001). PRR was statistically
significant at all time points, including as early as 3 weeks after
just one infusion, demonstrating a rapid onset of response. 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. PRR results as
measured by MRI/CT were consistent with those measured by
exophthalmometry at the primary efficacy analysis timepoint.
- Proptosis Mean Reduction: 2.34mm
mean reduction in proptosis from baseline in veligrotug patients,
compared with 0.46mm reduction in placebo patients (1.9mm
placebo-adjusted, p < 0.0001).
Diplopia:
- Diplopia Response: 56% of
veligrotug patients achieved a diplopia response, compared with 25%
of placebo patients (31% placebo-adjusted, p = 0.0006). Rapid onset
was observed as early as 6 weeks after just two infusions. Diplopia
response is defined as patients achieving a reduction of at least 1
on the Gorman subjective diplopia scale at week 15, for those
patients with diplopia at baseline (n = 102).
- Diplopia Complete Resolution: 32%
of veligrotug patients achieved complete resolution of diplopia,
compared with 14% of placebo patients (18% placebo-adjusted, p =
0.0152). Rapid onset was observed as early as 6 weeks after just
two infusions. Diplopia resolution is defined as patients achieving
a score of 0 on the Gorman subjective diplopia scale at week 15,
for those patients with diplopia at baseline.
Clinical Activity Score
(CAS):
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 Reduction to 0 or 1: 54% of
veligrotug patients achieved maximal or near-maximal therapeutic
effect on CAS, compared with 24% of placebo patients (29%
placebo-adjusted, p = 0.006), defined as reaching a CAS of 0 or 1,
among patients with a CAS of ≥ 3 at baseline (n = 104).
- CAS Mean Reduction: 2.9-point mean
reduction in CAS from baseline in veligrotug patients, compared
with 1.3-point reduction in placebo patients (1.6-point
placebo-adjusted, p < 0.0001), among patients with a CAS of ≥ 3
at baseline.
Overall Response:
- Overall Responder Rate: 56% of
veligrotug patients achieved an overall response, compared with 7%
of placebo patients (50% placebo-adjusted, p < 0.0001). Overall
Responder Rate is defined as achieving a proptosis response without
worsening of CAS from baseline (≥ 1 point increase) and without
worsening in the fellow eye in either proptosis (2 mm increase) or
CAS.
THRIVE-2 Safety Data
- Generally Well-Tolerated:
Veligrotug was generally well-tolerated with a safety profile
consistent with previous veligrotug studies including THRIVE. The
majority of adverse events (AEs) were mild, and 94% of
veligrotug-treated patients completed their treatment course.
- Low Rate of Hearing Impairment:
There was a 9.6% placebo-adjusted rate of hearing impairment AEs
(12.8% incidence in veligrotug patients, compared with 3.2%
incidence in placebo patients).
“Seeing the strong data presented today demonstrating that
diplopia can in fact be improved in patients with long standing
chronic TED is exciting. Together with the robust activity in both
THRIVE and THRIVE-2, favorable safety profile, and a shorter dosing
regimen, we believe veli is positioned to become a market leading
TED therapeutic,” said Tony Casciano, Chief Commercial Officer.
“Veli has the potential to be the only approved therapy with data
in chronic patients included in the labeling. We believe the
strength and completeness of veli’s pivotal program, the largest to
date in TED, could not only ensure advantageous market access, but
also expand utilization in patients unaddressed by current
therapies. Veli’s profile itself is inspiring, and if approved, our
commercial team is eager to get veli to patients.”
Subcutaneous VRDN-003, a Potential Best-In-Class Anti-IGF-1R,
On Track to Report Topline 1H 2026
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.
Based on this shared binding domain, Viridian believes the
topline results from THRIVE-2, in addition to the previously
reported THRIVE results, reinforces the potential best-in-class
profile of VRDN-003 to deliver clinical activity and safety
consistent with veligrotug in a low-volume, infrequent,
self-administered, subcutaneous injection that patients take at
home.
Viridian is currently dosing patients in two global phase 3
clinical trials for VRDN-003, REVEAL-1 and REVEAL-2, in active and
chronic TED, respectively. Viridian anticipates reporting topline
data from these studies in the first half of 2026 and submitting a
BLA 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-2 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
9934051.
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. Both THRIVE and THRIVE-2 reported positive topline
data, meeting all the primary and secondary endpoints of each
study. 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.
Cautionary 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,” “become,”
“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; the anticipated VRDN-003 topline data from THRIVE-2 in
the first half of 2026; anticipated BLA submissions for veligrotug
in the second half of 2025 and VRDN-003 in the second half of 2026,
pending data; Viridian’s expectation that its data package will
support a BLA for VRDN-003; the potential utility, efficacy,
potency, safety, clinical benefits, clinical response, convenience
and number of indications of veligrotug, VRDN-003, VRDN-006 and
VRDN-008; veligrotug’s potential to be a market-leading therapeutic
and the only approved therapy with data in chronic patients
included in the labeling; Viridian’s product candidates potentially
being best-in-class; whether veligrotug will serve an unmet need;
Viridian’s expectations regarding the potential commercialization
of veligrotug and VRDN-003, if approved; and that Viridian’s cash,
cash equivalents and short-term investments will be sufficient to
fund its operations into the second half of 2027.
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; 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; that our product candidates may not be
commercially successful, if approved and those risks set forth
under the caption “Risk Factors” in our most recent quarterly
report on Form 10-Q filed with the Securities and Exchange
Commission (SEC) on November 12, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20241215083644/en/
Anabel Chan, 617-458-8725 Vice President, Investor Relations
& Communications IR@viridiantherapeutics.com
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
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