- VRDN-001 Phase 3 THRIVE and THRIVE-2 topline
clinical data readouts are expected for mid-year 2024 and year-end
2024, respectively -
- Subcutaneous VRDN-003 pivotal program in
thyroid eye disease expected to start mid-year 2024 pending
regulatory authority alignment, as previously shared -
- Fc receptor (FcRn) inhibitors are on track
with VRDN-006 Investigational New Drug Application (IND) submission
anticipated by year-end 2024 and VRDN-008 non-human primate data
expected in the second half of 2024 -
- Year-end 2023 cash balance of approximately
$477.4 million; in January 2024, raised approximately $150 million
in gross proceeds from a public offering which extended the
company’s operating 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
fourth quarter and full year ended December 31, 2023.
“Throughout 2023, we delivered important clinical results across
our TED portfolio, marking significant progress for the company,”
said Steve Mahoney, Viridian President and Chief Executive Officer.
“We also unveiled our FcRn inhibitor portfolio in October 2023. I
would like to thank the Viridian team for their commitment
throughout this past year as we work towards our mission of
delivering potential best-in-class medicines for patients with
serious rare and autoimmune diseases. The momentum we generated
throughout 2023 has placed us in a strong position to execute on
our upcoming 2024 milestones across our TED and FcRn inhibitor
portfolios.”
RECENT PROGRESS
Thyroid Eye Disease Portfolio: VRDN-001 and VRDN-003
- VRDN-001: Viridian’s lead product candidate is an
intravenously-delivered monoclonal antibody that acts as a full
antagonist of the insulin-like growth factor-1 receptor (IGF-1R).
Two ongoing registrational Phase 3 clinical trials, THRIVE and
THRIVE-2, are continuing to enroll patients with active and chronic
TED, respectively. Topline data readouts are expected mid-year for
THRIVE and year-end for THRIVE-2.
- VRDN-003: In December 2023, Viridian reported positive
data for VRDN-003 from a Phase 1 clinical study in healthy
volunteers and selected VRDN-003 to move forward as its
subcutaneous anti-IGF-1R product candidate with the potential to
become a best-in-class anti-IGF-1R. VRDN-003 has the same binding
domain as its parent VRDN-001 and was engineered to have a longer
half-life. The Phase 1 results showed a VRDN-003 half-life of 40-50
days which is 4-5x the half-life of VRDN-001. Further,
pharmacokinetic modeling predicted that convenient dosing regimens
of VRDN-003 (e.g., a subcutaneous injection once every two, four,
or eight weeks) could achieve exposure levels of VRDN-003 that are
equivalent to the exposure levels of VRDN-001 that produced
clinically meaningful results in TED. Viridian expects to initiate
a global pivotal program with VRDN-003 mid-year, with planned
trials in both active and chronic TED patients, pending regulatory
authority alignment.
FcRn Inhibitor Portfolio: VRDN-006 and VRDN-008
In October 2023, Viridian unveiled its development of a
portfolio of engineered FcRn inhibitors, including VRDN-006 and
VRDN-008. FcRn inhibitors have the potential to treat a broad array
of autoimmune diseases. 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.
- VRDN-006: VRDN-006 is a highly selective Fc fragment
designed to be a convenient subcutaneous and self-administered
option for patients. Viridian anticipates submitting an IND for
VRDN-006 by year-end 2024.
- VRDN-008: VRDN-008 is designed to be a half-life
extended FcRn inhibitor with the goal to prolong IgG suppression
and provide a potentially best-in-class subcutaneous option for
patients. The VRDN-008 program is on track to deliver
pharmacokinetic and pharmacodynamic non-human primate data in the
second half of the year.
Recent Financing Activity & Runway Extension
- On January 17, 2024, the company announced a public offering of
common stock with gross proceeds of approximately $150.0 million,
which consisted of the sale of 7,142,858 shares of common stock at
a public offering price of $21.00 per share.
- In December 2023 and January 2024, Viridian sold an aggregate
of 2,245,868 shares of common stock through its at-the-market (ATM)
facility resulting in net proceeds of approximately $14.8 million
in December, which is included in the company’s year-end reported
financials, and approximately $35.2 million in January 2024.
- The company believes that its current cash, cash equivalents,
and short-term investments, including proceeds from the ATM and
January financing, will be sufficient to fund its operations into
the second half of 2026.
UPCOMING PRESENTATIONS AT NANOS
Viridian plans to present encore VRDN-001 Phase 2 clinical data
and Phase 3 clinical trial designs via two abstracts at the 50th
Annual Meeting of the North American Neuro-Ophthalmology Society
(NANOS) to be held March 2-7, 2024 in Honolulu, Hawaii. Following
NANOS, the presentations will be available at
www.viridiantherapeutics.com/pipeline/scientific-presentations.
- Oral Presentation
- Title: Clinical Activity and Safety of VRDN-001, a Full
Antagonist Antibody to Insulin-like Growth Factor-1 Receptor, in
Active and Chronic Thyroid Eye Disease
- Session: Scientific platform Session III
- Date: Tuesday, March 5, 2024
- Time: 10:15 – 10:30 am HST
- Poster Presentation
- Title: THRIVE and THRIVE-2: Phase 3 Trials of VRDN-001 in
Thyroid Eye Disease: Next Generation Insulin-like Growth Factor-1
Receptor Blockade
- Session: Analytical Studies Poster Reception
- Date: Tuesday, March 5, 2024
- Time: 7:00 – 9:00 pm HST
CORPORATE HIGHLIGHTS
- In January 2024, the company promoted Dr. Thomas Ciulla to
Chief Medical Officer.
- In February 2024, the company appointed Jennifer Tousignant as
Chief Legal Officer.
FINANCIAL RESULTS
- Cash Position: Cash, cash equivalents, and short-term
investments were $477.4 million as of December 31, 2023, compared
with $424.6 million as of December 31, 2022. As of January 31,
2024, Viridian’s cash, cash equivalents, and short-term investments
were approximately $645.7 million, which includes January proceeds
from the public offering and its ATM. This is a preliminary number
that has not been audited and is subject to change pending
completion of the company’s financial statements for the quarter
ended March 31, 2024, which Viridian anticipates reporting on in
its first quarter earnings release.
- R&D Expenses: Research and development expenses were
$159.8 million during the year ended December 31, 2023, compared to
$100.9 million during the year ended December 31, 2022. The
increase in research and development expenses was driven by
increased costs associated with our ongoing THRIVE and THRIVE-2
clinical trials, increased costs associated with the selection of
our subcutaneous product candidate VRDN-003, as well as increased
personnel-related costs as a result of increased headcount.
- G&A Expenses: General and administrative expenses
were $95.0 million during the year ended December 31, 2023,
compared to $35.2 million during the year ended December 31, 2022.
The increase in general and administrative expenses was driven by
personnel-related costs, including share-based compensation and
$31.0 million of one-time severance costs, as well as professional,
accounting and licensing fees to support a growing organization and
preparation for commercial activities.
- Net Loss: The company’s net loss was $237.7 million for
the year ended December 31, 2023, compared with $129.9 million for
the same period last year.
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 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 potential best-in-class intravenous therapy followed
by VRDN-003 as a potential 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, 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 those related to the VRDN-003 pivotal program; 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; 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 pharmacokinetic modeling predicts that
convenient dosing regimens of VRDN-003 (e.g., a subcutaneous
injection once every two, four, or eight weeks) could achieve
exposure levels of VRDN-003 that are equivalent to the exposure
levels of VRDN-001 that produced clinically meaningful results in
TED; potential dosing schedules and trial designs; the Company’s
expected cash, cash equivalents and short-term investments of
$645.7 million as of January 31, 2024; 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, including 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 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.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS (amounts in thousands, except share and
per share data) (unaudited)
Three Months Ended December
31,
Twelve Months Ended December
31,
2023
2022
2023
2022
Revenue: Collaboration Revenue - related party
$
72
$
105
$
314
$
1,772
Total revenue
72
105
314
1,772
Operating Expenses: Research and development
38,558
39,317
159,765
100,894
General and administrative
32,993
9,854
94,999
35,182
Total operating expenses
71,551
49,171
254,764
136,076
Loss from operations
(71,479
)
(49,066
)
(254,450
)
(134,304
)
Other income Interest and other income
5,535
3,449
18,563
4,916
Interest and other expense
(916
)
(168
)
(1,847
)
(486
)
Net loss
(66,860
)
(45,785
)
(237,734
)
(129,874
)
Change in unrealized gain (loss) on investments
403
300
728
(233
)
Comprehensive loss
$
(66,457
)
$
(45,485
)
$
(237,006
)
$
(130,107
)
Net loss
$
(66,860
)
$
(45,785
)
$
(237,734
)
$
(129,874
)
Net loss per share, basic and diluted
$
(1.35
)
$
(1.13
)
$
(5.31
)
$
(4.05
)
Weighted-average shares used to compute basic and diluted loss per
share
49,681,803
40,541,507
44,755,475
32,087,293
Viridian Therapeutics, Inc. Selected Financial
Information Condensed Condolidated Balance Sheets
(amounts in thousands) (unaudited)
December 31,
2023
2022
Cash, cash equivalents and short-term investments
$
477,370
$
424,550
Other assets
13,054
10,541
Total assets
$
490,424
$
435,091
Total liabilities
48,402
40,027
Total stockholders’ equity
442,022
395,064
Total liabilities and stockholders’ equity
$
490,424
$
435,091
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240227846012/en/
Louisa Stone, 617-272-4604 Manager, Investor Relations
IR@viridiantherapeutics.com
Viridian Therapeutics (NASDAQ:VRDN)
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