- Veligrotug, with recent positive phase 3
topline data in both active and chronic thyroid eye disease (TED),
has the potential to transform the standard of care in TED with a
differentiated clinical profile achieved with less drug and fewer
infusions; Biologics License Application (BLA) submission on track
for second half of 2025 -
- REVEAL-1 and REVEAL-2, phase 3 clinical
trials for subcutaneous (SC) VRDN-003 in active and chronic TED
respectively, are currently dosing patients; topline data for both
trials, which are evaluating VRDN-003 dosed Q4W or Q8W, is on track
for the first half of 2026 -
- Investigational New Drug (IND) submitted in
December 2024 for VRDN-006, an Fc fragment inhibitor of the
neonatal Fc receptor (FcRn); proof-of-concept IgG reduction
clinical data in healthy volunteers anticipated in the third
quarter of 2025 -
- VRDN-008, a bispecific FcRn inhibitor with an
extended half-life, progressing with preclinical studies as
planned; additional preclinical data expected in 2025 with an IND
submission planned for year-end 2025 -
- 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 VRDN-003, and multiple FcRn
inhibitor portfolio clinical catalysts -
Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology
company focused on discovering and developing potential
best-in-class medicines for serious and rare diseases, today
announces the company’s key priorities and catalysts for 2025.
“After a great year of execution across the portfolio, we enter
2025 poised to deliver on a number of important catalysts,” said
Steve Mahoney, Viridian’s President and CEO. “We believe that the
positive and better-than-expected topline data for veligrotug in
both THRIVE and THRIVE-2 confirms its potential to be the
treatment-of-choice for all forms of active and chronic TED at its
anticipated commercial launch. Our BLA and early commercial
preparations are well underway, and we are on track to submit the
BLA in the second half of this year. The 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 for VRDN-003 in
the second half of 2026.
“In addition to TED, we are equally excited by the potential of
our FcRn inhibitor portfolio. In Q3 of this year, we anticipate
showing proof-of-concept data, including IgG reduction, from our
planned VRDN-006 phase 1 clinical trial in healthy volunteers. We
also continue to advance our bispecific, half-life extended,
potential best-in-class VRDN-008 program. We previously showed
that, in non-human primates (NHPs), VRDN-008 had 3x the half-life
of efgartigimod and a more sustained IgG reduction. We anticipate
sharing additional data from ongoing VRDN-008 preclinical studies
later this year. We have another exciting year ahead and we look
forward to continued execution across our portfolio of derisked
programs.”
TED Portfolio: Well-Positioned with
Potential Best-in-Class Intravenous (IV) and Subcutaneous
Programs
Veligrotug, Potential IV Treatment-of-Choice for Active &
Chronic TED
Viridian’s lead product candidate, veligrotug, is a monoclonal
antibody that acts as a full antagonist of TED’s clinically and
commercially validated target of insulin-like growth factor-1
receptor (IGF-1R). In late 2024, Viridian reported positive topline
data for veligrotug in two of the largest global pivotal clinical
trials conducted to date in TED, THRIVE and THRIVE-2. Viridian
plans to submit a BLA for veligrotug for the treatment of patients
with active and chronic TED in the second half of this year.
- Phase 3 THRIVE Results in Active TED - Achieved All Primary
and Secondary Endpoints: Viridian announced positive topline
results for THRIVE in September 2024. Veligrotug met all the
primary and secondary endpoints at 15 weeks, including measures of
proptosis, clinical activity score (CAS), and diplopia, with a high
degree of statistical significance. Highlights of clinical efficacy
included 54% of treated patients achieving complete resolution of
their diplopia, or double vision, and a consistently rapid onset of
treatment effect across all disease endpoints, with a majority of
patients achieving a proptosis response after just one infusion, or
3 weeks after the start of therapy. Veligrotug was generally
well-tolerated with no treatment-related serious adverse events,
and importantly for a key area of interest in the class, had a low
5.5% placebo-adjusted rate of hearing impairment adverse events
(AEs).
- Phase 3 THRIVE-2 Results in Chronic TED - Achieved All
Primary and Secondary Endpoints: Viridian announced positive
topline results for THRIVE-2 in December 2024. THRIVE-2 studied the
broadest population of chronic TED patients in a global phase 3
clinical trial to date and, consistent with THRIVE, veligrotug met
all the primary and secondary endpoints at 15 weeks with a high
degree of statistical significance. Additionally, veligrotug is the
first product candidate to demonstrate a statistically significant
and clinically meaningful reduction and resolution of diplopia in
patients with chronic TED. Also consistent with THRIVE, veligrotug
demonstrated a rapid onset of treatment effect, showing a
statistically significant proptosis response as early as 3 weeks
and diplopia reduction and resolution as early as 6 weeks. Similar
to THRIVE, veligrotug was generally well-tolerated in THRIVE-2 and
showed a low 9.6% placebo-adjusted rate of hearing impairment
AEs.
Together, THRIVE and THRIVE-2 evaluated veligrotug in the
largest and broadest population of active and chronic TED patients
studied to date in global phase 3 clinical trials. Viridian
believes that these robust and consistent clinical efficacy and
safety results, after only five infusions of veligrotug, support a
differentiated product profile and the potential for veligrotug to
be the IV treatment-of-choice for all forms of active and chronic
TED.
Subcutaneous VRDN-003, a Potential Best-In-Class Anti-IGF-1R
for Active and Chronic TED
VRDN-003 is a monoclonal antibody targeting IGF-1R and shares
the same binding domain as veligrotug. VRDN-003 is engineered to
have an extended half-life, shown to be 40-50 days, or 4-5x that of
veligrotug, enabling evaluation of convenient dosing, as
infrequently as once every 8 weeks.
With the positive results from THRIVE and THRIVE-2, Viridian
views subcutaneous VRDN-003 as further derisked with the potential
to be a best-in-class treatment for TED based on:
- Same Binding Domain as Veligrotug IV: VRDN-003 (SC) has
the same binding domain as veligrotug (IV) and is therefore
expected to inhibit IGF-1R as a full antagonist in the same
manner;
- Half-life Extension: VRDN-003's confirmed extended
half-life is expected to support infrequent Q4W or Q8W dosing;
- Sustained Pharmacodynamic (PD) Effects: VRDN-003 showed
sustained serum increases in IGF-1 levels, a PD biomarker for
inhibiting IGF-1R;
- Derisked Dosing Regimens: each REVEAL study is
evaluating Q4W and Q8W dosing regimens of VRDN-003, which are each
predicted to achieve drug exposure levels that have shown robust
clinical activity with veligrotug in TED; and
- Autoinjector Delivery for Patient Convenience and
Access: Viridian expects to launch VRDN-003 with a commercially
validated autoinjector pen to enable at-home patient
self-administration.
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. Topline data for both REVEAL-1 and
REVEAL-2 remains on track for the first half of 2026, which would
enable a BLA submission by year-end 2026.
FcRn Inhibitor
Portfolio:
In October 2023, Viridian first unveiled VRDN-006 and VRDN-008
in its portfolio of engineered FcRn inhibitors. FcRn inhibitors
have the potential to treat a broad array of autoimmune diseases,
representing significant commercial market opportunities.
VRDN-006, a Highly-Selective Fc Fragment that Inhibits
FcRn
VRDN-006 is an Fc fragment that inhibits FcRn and is designed to
be a convenient subcutaneous and self-administered option for
patients. In NHPs, VRDN-006 showed specificity for blocking
FcRn-IgG interactions while sparing albumin and low-density
lipoprotein (LDL).
- IND Submitted in December 2024: As planned, Viridian
submitted an IND for VRDN-006 in December 2024 and, upon clearance,
anticipates initiating a phase 1 clinical trial in healthy
volunteers in early 2025.
- Proof-of-Concept Phase 1 Clinical Data On-Track for Q3
2025: Viridian expects data from the phase 1 clinical trial in
Q3 2025, including proof-of-concept IgG reductions in healthy
volunteers. These data have historically been translatable to
patient populations.
VRDN-008, a Half-life Extended Bispecific FcRn
Inhibitor
VRDN-008 comprises an Fc fragment and an albumin-binding domain
designed to prolong IgG suppression as a subcutaneous,
self-administered, and potential best-in-class option for
patients.
- Confirmed Half-Life Extension and Sustained PD Effects:
Viridian announced in November 2024 that VRDN-008 demonstrated 3x
the half-life of efgartigimod in a single, high-dose, head-to-head
study in NHPs. Additionally, VRDN-008 showed a deeper and more
sustained IgG reduction in the same study with peak IgG reductions
that were 20% deeper than efgartigimod. VRDN-008 spared albumin and
LDL, consistent with efgartigimod.
- Additional Preclinical Data Expected in 2025: Additional
NHP studies are ongoing to generate data with lower doses and
multiple doses of VRDN-008. Once completed, Viridian plans to use
the totality of VRDN-008’s NHP data to build a robust
pharmacokinetic and pharmacodynamic model to enable the prediction
of potential human dosing regimens for VRDN-008.
Corporate Updates
Viridian announces the appointment and promotion of Seth Harmon
to the role of Chief Financial Officer. Mr. Harmon joined Viridian
in 2023 as the Senior Vice President of Finance and Accounting and
has over 20 years of strategic finance, accounting, and operations
experience. Prior to joining Viridian, Mr. Harmon served as the CFO
of BioNTech US, where he oversaw the general and administrative
functions for BioNTech SE’s US subsidiary. Prior to that, Mr.
Harmon held multiple positions of increasing seniority in finance
at Neon Therapeutics and Merrimack Pharmaceuticals, after starting
his career at Ernst and Young, where he spent 10 years in the audit
and assurance practices. Mr. Harmon holds an M.S. in Accounting and
M.B.A. from Northeastern University and obtained his certified
public accountant license while working at Ernst and Young, LLP. He
received his B.A. in Economics and Mathematics from Bowdoin
College.
Notice of Inducement
Grants
Today Viridian announces that a majority of the independent
directors serving on the Compensation Committee of the company’s
Board of Directors approved the grant of non-qualified stock
options to purchase an aggregate of 98,500 shares of the company’s
common stock to four new employees (the “Inducement Grants”) on
January 2, 2025 (the “Grant Date”). The Inducement Grants have been
granted outside of the company’s Amended and Restated 2016 Equity
Incentive Plan (the “Plan”) but remain subject to the terms and
conditions of such Plan. The Inducement Grants were granted as an
inducement material to these individuals entering into employment
with Viridian in accordance with Nasdaq Listing Rule
5635(c)(4).
The Inducement Grants have an exercise price per share that is
equal to the closing price of Viridian’s common stock on the Grant
Date. The Inducement Grants will vest over a four-year period, with
25% of the shares vesting on the one-year anniversary of the
employee’s start date, and thereafter the remainder of the shares
vest in 36 equal monthly installments, subject to each employee’s
continued employment with Viridian through the applicable vesting
dates.
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.
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 development, clinical development, and anticipated
commercialization of Viridian’s product candidates veligrotug
(formerly VRDN-001), VRDN-003, VRDN-006 and VRDN-008, including
Viridian's view that the THRIVE and THRIVE-2 data provides strong
support for VRDN-003's clinical profile; anticipated start dates of
studies, including the initiation of the phase 1 clinical trial for
VRDN-006; anticipated data results and timing of their disclosure,
including VRDN-003 topline data from the REVEAL-1 and REVEAL-2
trials in the first half of 2026, anticipated VRDN-006
proof-of-concept clinical data in the third quarter of 2025, and
anticipated VRDN-008 preclinical data in 2025; regulatory
interactions and anticipated timing of regulatory submissions,
including the anticipated BLA submissions for veligrotug in the
second half of 2025 and VRDN-003 in the second half of 2026, and
the anticipated IND submission for VRDN-008 by year-end 2025,
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 transform the standard of care
and the potential for veligrotug to be the IV treatment-of-choice
for all forms of active and chronic TED; Viridian’s product
candidates potentially being best-in-class; Viridian’s view that
its portfolio programs are derisked, including VRDN-003; 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250108385710/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
Viridian Therapeutics (NASDAQ:VRDN)
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