- VRDN-001 topline clinical results for THRIVE
and THRIVE-2 on track for mid-2024 and year-end 2024 in patients
with active and chronic thyroid eye disease (TED), respectively
-
- Subcutaneous VRDN-003 pivotal study in TED
anticipated to start in mid-2024, pending alignment with regulatory
authorities -
- Anti-neonatal Fc receptor (FcRn) portfolio
progressing as planned with VRDN-006 IND anticipated by year-end
2024 and VRDN-008 data in non-human primates in 2H 2024 -
- Tom Ciulla, M.D., M.B.A. promoted to Chief
Medical Officer to succeed Barrett Katz, M.D., M.B.A. -
Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology
company focused on discovering and developing potential
best-in-class medicines for serious and rare diseases, today
announced that President and Chief Executive Officer, Steve
Mahoney, will discuss the company’s progress at the 42nd Annual
J.P. Morgan Healthcare Conference in San Francisco, California.
As part of Viridian’s presentation at the J.P. Morgan Healthcare
Conference, Mr. Mahoney will provide key updates on the company’s
anti-insulin-like growth factor 1 receptor (IGF-1R) programs in
thyroid eye disease (TED) and its programs targeting FcRn. Viridian
aims to build a world-class portfolio in TED with a differentiated
and fast-to-market lead asset in VRDN-001, which is delivered
intravenously, and a potential best-in-class asset, VRDN-003, which
is designed to be self-administered subcutaneously at home as an
infrequent and low-volume injection. In addition to TED, the
company is also developing a novel portfolio of FcRn inhibitors
with VRDN-006, an Fc fragment, and VRDN-008, which is engineered to
have an extended half-life with the goal to prolong immunoglobulin
G (IgG) suppression.
“2024 is poised to be a year with major catalysts for Viridian
as we advance VRDN-001 towards topline data in two global Phase 3
studies in active and chronic TED, expected by the middle and end
of the year, respectively,” said Mr. Mahoney. “In addition, we were
thrilled by the positive data that exceeded our expectations from
our healthy volunteer studies in December 2023 that led to the
selection of VRDN-003 as our potential best-in-class subcutaneous
program. We plan to initiate the VRDN-003 pivotal program in
mid-2024, pending alignment with regulatory authorities, as we aim
to decrease the treatment burden on patients and their families
living with TED. We are equally excited by the progress in our FcRn
portfolio. We look forward to submitting an Investigational New
Drug Application (IND) for VRDN-006 by the end of the year and
providing additional details on VRDN-008 in the second half of the
year.”
Thyroid Eye Disease Portfolio: VRDN-001
and VRDN-003
Intravenous VRDN-001
Viridian’s lead product candidate, VRDN-001, is a monoclonal
antibody that acts as a full antagonist of IGF-1R, a clinically and
commercially validated target for TED that has current US annual
revenues close to $2 billion. Viridian is currently evaluating
VRDN-001 in two global Phase 3 clinical trials, THRIVE and
THRIVE-2, for the treatment of active and chronic TED,
respectively. THRIVE and THRIVE-2 are each designed to compare a
five-dose treatment arm of VRDN-001, dosed three weeks apart, to
placebo. This five-dose VRDN-001 regimen features fewer infusions
and a shorter time per infusion compared to the currently marketed
IGF-1R inhibitor. The company expects to report THRIVE and THRIVE-2
data in the middle of 2024 and by year end 2024, respectively.
THRIVE and THRIVE-2 data will look to confirm the Phase 2 data
Viridian reported throughout 2022 and 2023. In all dose cohorts of
a Phase 2 clinical trial, intravenous VRDN-001 was shown to improve
the signs and symptoms of TED at six weeks in patients with active
and chronic disease after two infusions. VRDN-001 was also
well-tolerated.
Subcutaneous VRDN-003
In December 2023, the company selected VRDN-003 as the potential
best-in-class subcutaneous anti-IGF-1R program for pivotal
development in TED following positive data in a Phase 1 clinical
trial in healthy volunteers. VRDN-003 has the same binding domain
as its parent molecule, VRDN-001, and was engineered to have a
longer half-life. VRDN-003 is designed to maintain the clinical
response of VRDN-001 IV while increasing patient convenience.
The Phase 1 clinical study showed VRDN-003 to have a prolonged
half-life of 40-50 days, which is four to five times that of its
parent molecule, VRDN-001. Because VRDN-001 and VRDN-003 are nearly
identical antibodies, the company expects VRDN-003 to have similar
clinical responses at the exposure levels of VRDN-001 that led to
robust clinical activity in its Phase 2 clinical study in TED.
Further, pharmacokinetic modeling of VRDN-003 predicted that dosing
regimens as long as every 8 weeks would achieve or exceed VRDN-001
exposure levels that were clinically meaningful. Based on these
results, the company expects to initiate a global pivotal program
with VRDN-003 in mid-2024, with planned trials in both active and
chronic TED patients, pending alignment with regulatory
authorities.
Anti-FcRn Portfolio: VRDN-006 and
VRDN-008
In October 2023, consistent with Viridian’s vision to develop
the next generation of best-in-class products for severe autoimmune
and rare diseases, the company unveiled a portfolio of engineered
FcRn inhibitors, VRDN-006 and VRDN-008. FcRn inhibitors have the
potential to treat a broad array of autoimmune diseases,
representing a significant commercial market opportunity.
Viridian’s multi-pronged engineering approach has resulted in a
portfolio of FcRn-targeting molecules that leverage the clinically
and commercially validated mechanism of FcRn inhibition while
potentially addressing the limitations of current agents such as
incomplete IgG suppression and needed improvements in safety.
VRDN-006 is a highly-selective Fc fragment. In non-human
primates, VRDN-006 showed specificity for blocking FcRn-IgG
interactions while showing no decreases in albumin or increases in
low-density lipoprotein (LDL) levels. VRDN-008 is a novel,
first-in-class FcRn inhibitor that aims to pair IgG suppression
with extended half-life technology. VRDN-008, with its extended
half-life, has the potential to more deeply and durably suppress
IgG as compared to existing anti-FcRn therapies and pipeline
candidates. VRDN-006 and VRDN-008 are both designed to be
convenient, self-administered, subcutaneous products.
Summary of Anticipated 2024 Key Program Milestones
Thyroid eye disease (TED) Portfolio
- Mid-2024
- THRIVE Phase 3 trial topline results in active TED for VRDN-001
IV
- Initiate subcutaneous pivotal program for VRDN-003, pending
regulatory alignment
- Year End 2024
- THRIVE-2 Phase 3 trial topline results in chronic TED for
VRDN-001 IV
Anti-FcRn Portfolio
- 2H 2024
- IND submission for VRDN-006 by year end
- VRDN-008 NHP data
Corporate Updates
Today, the company announced the promotion of Dr. Thomas Ciulla
to Chief Medical Officer to succeed Dr. Barrett Katz. Dr. Katz will
continue to work with Viridian as a consultant to the company. Dr.
Ciulla joined Viridian in early 2023 as Chief Development Officer,
where he has been leading the clinical development efforts for all
programs. Dr. Ciulla has held numerous leadership roles in medical
and clinical development and research, including serving most
recently as Chief Medical Officer and Chief Development Officer of
Clearside Bio prior to joining Viridian.
“Dr. Ciulla has been an integral part of the Viridian team
throughout his tenure and will continue to lead our clinical
development efforts as Chief Medical Officer,” said Mr. Mahoney.
“We are grateful to Dr. Katz for his years of dedicated service to
Viridian and look forward to working with him as an advisor to the
company.”
Notice of Inducement Grant
Today Viridian announced 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 an employee to purchase up to 62,000 shares of the
company’s common stock (the “Inducement Grant”) on January 2, 2024
(the “Grant Date”). The Inducement Grant has been granted outside
of the company’s Amended and Restated 2016 Equity Incentive Plan
(the “Plan”) but remains subject to the terms and conditions of
such Plan. The Inducement Grant was granted as an inducement
material to this individual entering into employment with Viridian
in accordance with Nasdaq Listing Rule 5635(c)(4).
The Inducement Grant has an exercise price per share that is
equal to the closing price of Viridian’s common stock on the Grant
Date. The Inducement Grant 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 the 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 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
is conducting two global Phase 3 clinical trials (THRIVE and
THRIVE-2) to evaluate the safety and efficacy of VRDN-001 in
patients with active and chronic TED. Viridian’s goal is to advance
VRDN-001 as a best-in-class IV therapy followed by VRDN-003 as a
first- and best-in-class subcutaneous therapy for the treatment of
TED. In addition to its TED portfolio, Viridian is advancing a
novel portfolio of neonatal Fc receptor (FcRn) inhibitors, 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,” “plan,”
“potential,” “predict,” “project,” “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 VRDN-001, VRDN-003,
VRDN-006 and VRDN-008; anticipated start dates of studies;
alignment with regulatory authorities and anticipated regulatory
submissions; enrollment in Viridian’s clinical studies, including
the THRIVE and THRIVE-2 Phase 3 clinical studies; upcoming
milestones and anticipated data results, including topline results;
that THRIVE and THRIVE-2 data will look to confirm the Phase 2 data
Viridian reported throughout 2022 and 2023; the potential utility,
efficacy, potency, safety, clinical benefits, clinical response and
convenience of VRDN-001, VRDN-003, VRDN-006 and VRDN-008;
Viridian’s product candidates potentially being best-in-class; that
because VRDN-001 and VRDN-003 are nearly identical antibodies, the
company expects VRDN-003 to have similar clinical responses at the
exposure levels of VRDN-001 that led to robust clinical activity in
a Phase 2 clinical study in TED; that pharmacokinetic modeling of
VRDN-003 predicts that dosing regimens as long as every 8 weeks
would achieve or exceed the same VRDN-001 exposure levels that were
clinically meaningful; the time to market and commercial viability
of Viridian’s product candidates; and potential dosing schedules
trial designs. 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;
the relationship between the results from the positive data from
completed or ongoing clinical trials and 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, pre-clinical and clinical
development programs; trial protocols for ongoing clinical trials;
expectations regarding the timing for regulatory filings;
expectations 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 its
projected cash runway; our future operating results and financial
performance; Viridian’s intellectual property position; the timing
of pre-clinical and clinical trial activities and reporting results
from same, including those risks set forth under the caption “Risk
Factors” in our Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) on November 13, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240108920225/en/
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
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