- THRIVE VRDN-001 global phase 3 clinical trial
in active thyroid eye disease (TED) completed and exceeded its
target for enrollment in March 2024; topline readout expected in
September 2024 -
- THRIVE-2 VRDN-001 global phase 3 clinical
trial for patients with chronic TED remains on track for topline
readout by year-end 2024 -
- Positive VRDN-003 Type C meeting held with
the US Food and Drug Administration (FDA); pivotal program on track
to start mid-year 2024 -
- VRDN-006 FcRn inhibitor remains on track for
Investigational New Drug (IND) submission by year-end 2024;
non-human primate data expected for VRDN-008, a half-life extended
FcRn inhibitor, in the second half of 2024 -
- Conference call today at 8:00am ET -
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
first quarter ending March 31, 2024.
“It has been a remarkable first quarter of execution as we made
progress across our TED and neonatal Fc receptor (FcRn) inhibitor
programs,” said Steve Mahoney, Viridian President and Chief
Executive Officer. “Rapidly completing and exceeding enrollment in
THRIVE demonstrates the demand for new therapeutic options in TED
and reflects our team’s ability to execute as we look forward to
delivering on the exciting catalysts in the year ahead. We remain
on track for our key milestones across the portfolio as we aim to
deliver on our corporate priorities for the benefit of patients and
our other stakeholders.”
RECENT PROGRESS
Thyroid Eye Disease Portfolio
VRDN-001, an intravenously
delivered anti-insulin-like growth factor-1 receptor (IGF-1R)
antibody
- THRIVE Enrollment Completed in March 2024, Topline Data
Expected September 2024: The THRIVE phase 3 clinical trial in
patients with active TED completed enrollment in March 2024,
meeting and exceeding its enrollment target of 90 patients with
patient demand driving a total enrollment of 113 patients.
Approximately half of the enrolled patients were from the US and
half from Europe.
- THRIVE-2 Topline Data On Track for Year-End 2024: The
THRIVE-2 phase 3 clinical trial in patients with chronic TED
continues to enroll and remains on track for topline readout by
year-end 2024.
- VRDN-001 Safety Database and Preparation For Second Half
2025 Biologics License Application (BLA) Filing: To meet the
safety database requirement for BLA filing, Viridian is actively
enrolling patients into the recently initiated STRIVE clinical
trial. STRIVE is a global study of VRDN-001 in TED patients that
utilizes broad inclusion criteria (e.g., any severity or duration
of disease) and is randomized 3:1 (10 mg/kg IV with an active
control of 3 mg/kg IV). Viridian is also enrolling patients in an
open label extension study for non-responders in THRIVE and
THRIVE-2. Viridian anticipates filing a BLA in the second half of
2025, pending data, for marketing approval in TED 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 product
candidate designed to be a low-volume and infrequent subcutaneous
injection
- Pivotal Program On Track For Mid-Year 2024 Initiation
Following Positive FDA Meeting: Viridian completed a positive
Type C meeting with the FDA to discuss the VRDN-003 pivotal
development plan. The pivotal program remains on track to start
mid-year 2024, and Viridian plans to provide additional details,
including study designs, prior to the start of the program.
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 $613.2 million as of March 31, 2024, compared with
$477.4 million as of December 31, 2023. 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
$40.9 million during the quarter ended March 31, 2024, compared to
$50.7 million during the quarter ended March 31, 2023. The decrease
in research and development expenses was driven by a $15 million
license fee incurred during the quarter ending March 31, 2023. This
decrease was partially offset by increased clinical trials costs
associated with our ongoing THRIVE and THRIVE-2 clinical trials, as
well as increased personnel costs.
- G&A Expenses: General and administrative expenses
were $15.0 million during the quarter ended March 31, 2024,
compared to $21.8 million during the quarter ended March 31, 2023.
The decrease in general and administrative expenses was driven by a
reduction in severance and share-based compensation costs related
to separation agreements with former executive officers entered
into during the quarter ending March 31, 2023.
- Net Loss: The company’s net loss was $48.5 million for
the first quarter ended March 31, 2024, compared with $68.2 million
for the same period last year.
- Shares Outstanding: As of March 31, 2024, Viridian had
approximately 83,863,339 shares of common stock outstanding on an
as-converted basis, which included 63,798,536 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.
CONFERENCE CALL AND WEBCAST
Viridian will host a webcast and conference call to discuss its
first quarter 2024 financial results and provide a corporate update
today, May 8, at 8:00 a.m. ET.
The webcast can be accessed under “Events and Presentations” on
the Investors section of the Viridian website at
viridiantherapeutics.com. To participate in the conference call,
please dial 800-715-9871 (domestic) or 646-307-1963 (international)
and reference code 7373356. A replay of the webcast will be
available following the completion of the event.
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’s goal is to advance VRDN-001 as a potential best-in-class
intravenous therapy followed by VRDN-003 as a potential
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, 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,” “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,
including the initiation date of the VRDN-003 pivotal program;
alignment with regulatory authorities and anticipated regulatory
submissions, including the anticipated IND submission for VRDN-006
and the anticipated BLA submission for VRDN-001; Viridian’s
expectation that its data will support a BLA in the second half of
2025, pending data, for marketing approval in TED for VRDN-001;
Viridian’s expectation that its data package will support a
marketing authorization application in Europe for VRDN-001;
anticipated study designs and their disclosure; enrollment in
Viridian’s clinical studies; upcoming milestones and anticipated
data results, including topline results; 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; and
that the company’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;
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, preclinical 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
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 annual report on Form 10-K filed with
the Securities and Exchange Commission (SEC) on February 27, 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 March 31,
2024
2023
Revenue: Collaboration Revenue - related party
$
72
$
98
Total revenue
72
98
Operating Expenses: Research and development
40,944
50,740
General and administrative
15,025
21,831
Total operating expenses
55,969
72,571
Loss from operations
(55,897)
(72,473)
Other income Interest and other income
7,942
4,487
Interest and other expense
(587)
(165)
Net loss
(48,542)
(68,151)
Change in unrealized gain (loss) on investments
(705)
216
Comprehensive loss
$
(49,247)
$
(67,935)
Net loss
$
(48,542)
$
(68,151)
Net loss per share, basic and diluted
$
(0.79)
$
(1.61)
Weighted-average shares used to compute basic and diluted loss per
share
61,099,038
42,242,309
Viridian Therapeutics, Inc. Selected Financial
Information Condensed Condolidated Balance Sheets
(amounts in thousands) (unaudited) March
31, December 31,
2024
2023
Cash, cash equivalents and short-term investments
$
613,187
$
477,370
Other assets
17,194
13,054
Total assets
$
630,381
$
490,424
Total liabilities
47,851
48,402
Total stockholders’ equity
582,530
442,022
Total liabilities and stockholders’ equity
$
630,381
$
490,424
Source: Viridian Therapeutics, Inc.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240508566867/en/
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
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