- THRIVE VRDN-001 global phase 3 clinical trial
in active thyroid eye disease (TED) remains on track for topline
readout in September 2024 -
- THRIVE-2 VRDN-001 global phase 3 clinical
trial in chronic TED topline readout expected year-end 2024;
enrollment completed in July and exceeded its target -
- REVEAL-1 and REVEAL-2, global phase 3
clinical trials for subcutaneous VRDN-003 in patients with active
and chronic TED, on track to initiate in August 2024 -
- Investigational New Drug (IND) submission for
neonatal Fc receptor (FcRn) inhibitor VRDN-006 planned by year-end
2024; non-human primate (NHP) data anticipated for half-life
extended FcRn inhibitor VRDN-008 in the second half of 2024 -
- 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 -
Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology
company focused on discovering and developing potential
best-in-class medicines for serious and rare diseases, today
reported recent business highlights and financial results for the
second quarter ending June 30, 2024.
“Our team continues to execute across the board as shown by the
progress we have made this quarter, and we are proud to report
solid progress in our TED portfolio, with both VRDN-001 phase 3
trials, THRIVE and THRIVE-2, exceeding enrollment targets, and
VRDN-003 trials, REVEAL-1 and REVEAL-2, planned to initiate this
month,” said Steve Mahoney, Viridian President and Chief Executive
Officer. “All timelines within our FcRn portfolio remain on track,
and we anticipate submitting an IND for VRDN-006 by year-end, and
reporting NHP data for VRDN-008 in the second half of the year. We
look forward to delivering on these multiple key program milestones
this year, beginning with our THRIVE readout next month.”
RECENT PROGRESS
Thyroid Eye Disease Portfolio
VRDN-001, an intravenously
delivered anti-insulin-like growth factor-1 receptor (IGF-1R)
antibody
- THRIVE-2 Completed and Exceeded Enrollment in July 2024:
The THRIVE-2 global phase 3 clinical trial in patients with chronic
TED completed enrollment in July 2024 with a total of 188 patients,
exceeding its enrollment target of 159 patients due to patient
demand. Approximately 40% of the enrolled patients were from the
US.
- VRDN-001 Topline Data and BLA on Track: Both VRDN-001
global phase 3 clinical studies, THRIVE and THRIVE-2, are on track
for topline data readouts in September and by year-end 2024,
respectively. Viridian anticipates submitting a Biologics License
Application (BLA) in the second half of 2025, pending data, and
expects that its data package will support a marketing
authorization application in Europe.
VRDN-003, a potential
best-in-class, subcutaneous, half-life extended anti-IGF-1R
antibody designed as a low-volume, infrequent, and
self-administered subcutaneous injection
- REVEAL-1 and REVEAL-2 on Track to Initiate in August:
REVEAL-1 and REVEAL-2 are randomized, double-masked,
placebo-controlled, global phase 3 clinical trials assessing the
efficacy and safety of VRDN-003 in patients with active and chronic
TED, respectively. In each study, Viridian plans to administer
VRDN-003 subcutaneously every 4 weeks or every 8 weeks and assess
outcomes versus placebo. The trials are designed to have a primary
endpoint of proptosis responder rate, based on the achievement of
at least 2 mm improvement in proptosis from baseline at week 24,
and additional secondary outcome measures including changes from
baseline in proptosis, clinical activity score (CAS), and
diplopia.
- Topline Data and BLA in 2026: Viridian anticipates
topline data for both REVEAL-1 and REVEAL-2 to be available in the
first half of 2026 to enable a potential BLA submission by the end
of 2026. The company plans to launch VRDN-003 with a commercially
available auto-injector pen, if approved.
FcRn Inhibitor Portfolio
VRDN-006, a highly selective
anti-FcRn Fc fragment designed to be a convenient subcutaneous and
self-administered option for patients
- IND on Track for Year-End 2024: Viridian is on track to
submit an IND application for VRDN-006 by year-end 2024.
VRDN-008, a half-life
extended FcRn inhibitor designed to prolong IgG suppression and
provide a potentially best-in-class subcutaneous option for
patients
- NHP Data on Track for Second Half 2024: Viridian is on
track to provide VRDN-008 non-human primate data, including PK and
PD data, in the second half of 2024.
- Potential Best-in-Class Profile: As a half-life extended
FcRn inhibitor, VRDN-008 has the potential to enable deeper and
more durable suppression than existing therapies targeting
FcRn.
FINANCIAL RESULTS
- Cash Position: Cash, cash equivalents, and short-term
investments were $571.4 million as of June 30, 2024, compared with
$613.2 million as of March 31, 2024. The company believes that its
current cash, cash equivalents, and short-term investments will be
sufficient to fund its operations into the second half of
2026.
- R&D Expenses: Research and development expenses were
$56.2 million during the quarter ended June 30, 2024, compared to
$40.1 million during the quarter ended June 30, 2023. The increase
in research and development expenses was driven by increased
clinical trials costs associated with our ongoing THRIVE and
THRIVE-2 clinical trials, as well as increased personnel costs to
support our pipeline development.
- G&A Expenses: General and administrative expenses
were $16.1 million during the quarter ended June 30, 2024, compared
to $19.3 million during the quarter ended June 30, 2023. The
decrease in general and administrative expenses was driven by a
decrease in personnel-related costs, primarily due to decreased
share-based compensation expense in the current year.
- Net Loss: The company’s net loss was $65.0 million for
the second quarter ended June 30, 2024, compared with $55.1 million
for the same period last year.
- Shares Outstanding: As of June 30, 2024, Viridian had
approximately 83,944,478 shares of common stock outstanding on an
as-converted basis, which included 63,879,675 shares of common
stock and an aggregate of approximately 20,064,803 shares of common
stock issuable upon the conversion of 157,435 and 143,522 shares of
Series A and Series B preferred stock, respectively.
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 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
planned global phase 3 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 VRDN-001, VRDN-003, VRDN-006 and VRDN-008; anticipated
start dates of studies, including the initiation date of the
REVEAL-1 and REVEAL-2, global phase 3 clinical trials for VRDN-003;
milestones; timelines; anticipated data results and timing of their
disclosure, including topline results; regulatory interactions and
anticipated timing of regulatory submissions, including the
anticipated IND submission for VRDN-006 and the anticipated BLA
submission for VRDN-001; Viridian’s expectation that its data
package will support a BLA submission for VRDN-001 in the second
half of 2025, pending data; Viridian’s expectation that its data
package will support a marketing authorization application in
Europe for VRDN-001; 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 with a commercially available
auto-injector pen, if approved; the potential utility, efficacy,
potency, safety, clinical benefits, clinical response, convenience
and number of indications of VRDN-001, VRDN-003, VRDN-006 and
VRDN-008; Viridian’s product candidates potentially being
best-in-class; 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 VRDN-001 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 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.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS (amounts in thousands, except share and
per share data) (unaudited) Three
Months Ended June 30, Six Months Ended June 30,
2024
2023
2024
2023
Revenue: Collaboration Revenue - related party
$
72
$
72
$
144
$
170
Total revenue
72
72
144
170
Operating Expenses: Research and development
56,193
40,083
97,136
90,823
General and administrative
16,066
19,264
31,091
41,095
Total operating expenses
72,259
59,347
128,227
131,918
Loss from operations
(72,187
)
(59,275
)
(128,083
)
(131,748
)
Other income Interest and other income
7,791
4,378
15,732
8,865
Interest and other expense
(597
)
(166
)
(1,184
)
(331
)
Net loss
(64,993
)
(55,063
)
(113,535
)
(123,214
)
Change in unrealized gain (loss) on investments
(176
)
1
(881
)
217
Comprehensive loss
$
(65,169
)
$
(55,062
)
$
(114,416
)
$
(122,997
)
Net loss
$
(64,993
)
$
(55,063
)
$
(113,535
)
$
(123,214
)
Net loss per share, basic and diluted
$
(1.02
)
$
(1.27
)
$
(1.82
)
$
(2.88
)
Weighted-average shares used to compute basic and diluted loss per
share
63,854,514
43,253,457
62,476,777
42,753,476
Viridian Therapeutics, Inc. Selected Financial
Information Condensed Consolidated Balance Sheets
(amounts in thousands) (unaudited) June
30, December 31,
2024
2023
Cash, cash equivalents and short-term investments
$
571,366
$
477,370
Other assets
14,297
13,054
Total assets
$
585,663
$
490,424
Total liabilities
56,325
48,402
Total stockholders’ equity
529,338
442,022
Total liabilities and stockholders’ equity
$
585,663
$
490,424
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240805076289/en/
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
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