Viridian Therapeutics Highlights Recent Progress and Reports Fourth Quarter and Full Year 2023 Financial Results
- Positive clinical results in 2023 across their TED portfolio.
- Upcoming Phase 3 data readouts for VRDN-001 and VRDN-003 in 2024.
- Successful financing activities with $150 million raised in January 2024.
- Cash balance of $477.4 million at year-end 2023.
- Plans to present clinical data at NANOS in March 2024.
- Significant increase in R&D expenses from 2022 to 2023.
- Substantial rise in net loss from 2022 to 2023.
- Increased general and administrative expenses driven by severance costs and personnel-related expenses.
Insights
The financial results reported by Viridian Therapeutics, Inc. indicate a substantial increase in their year-over-year R&D and G&A expenses, which is reflective of their aggressive development strategy. The jump from $100.9 million to $159.8 million in R&D expenses suggests a significant investment in advancing their clinical trials, particularly for their leading candidates VRDN-001 and VRDN-003. Additionally, the G&A expenses have more than doubled, largely due to the preparation for commercial activities and an expanding workforce.
Viridian's cash position, with a year-end balance of $477.4 million and the subsequent financing in January 2024 raising an additional $150 million, provides the company with a robust financial runway into the second half of 2026. This is critical for investors as it indicates that the company has secured sufficient funding to continue its operations and clinical programs without the immediate need for further financing, which could dilute existing shares.
However, the reported net loss of $237.7 million for the year ended December 31, 2023, compared to $129.9 million in the previous year, highlights the company's current stage of high cash burn due to its investment in R&D. This is a common characteristic of biotech firms in the clinical development phase and should be evaluated in the context of potential future revenues from successful product launches.
Viridian’s focus on Thyroid Eye Disease (TED) and its portfolio of FcRn inhibitors represents a strategic targeting of niche markets with high unmet medical needs. The progression of VRDN-001 and VRDN-003 into later stages of clinical development is significant, as these therapies could offer new treatment options for patients with active and chronic TED. The extended half-life of VRDN-003, as indicated by the Phase 1 study, could lead to less frequent dosing and improved patient compliance, potentially differentiating it from other treatments.
Furthermore, the anticipated Investigational New Drug (IND) submission for VRDN-006 by the end of 2024 marks a critical step towards clinical development for autoimmune diseases. The focus on subcutaneous and self-administered options reflects a trend in biologics aiming to improve patient quality of life. The non-human primate data for VRDN-008 expected in the second half of 2024 will be crucial in determining its pharmacokinetics and pharmacodynamics and thereby its potential efficacy and safety in humans.
From a research perspective, the upcoming presentations at NANOS will be an opportunity for the scientific community to scrutinize the data and trial designs, which could impact the perception of Viridian's candidates within the medical community.
The biotechnology sector is highly competitive and subject to rapid innovation. Viridian's emphasis on developing potential best-in-class medicines positions the company to potentially capture significant market share within the TED and autoimmune disease spaces, should their product candidates receive regulatory approval. The company's strategic alignment with regulatory authorities for the initiation of a pivotal program with VRDN-003 mid-year reflects a proactive approach to ensuring compliance and facilitating a smooth transition into later-stage trials.
Investors and stakeholders will be monitoring the outcomes of the THRIVE and THRIVE-2 clinical trials closely, as positive topline data readouts could significantly impact the company's valuation and market potential. The mid-year 2024 and year-end 2024 timelines for these readouts provide clear milestones for evaluating the company's progress and potential for future revenue generation.
It is also worth noting the importance of the company's recent financing activities in extending its operating runway. The ability to secure funding through a public offering and ATM facility demonstrates market confidence in Viridian's strategy and management. This extended runway allows for a focus on product development without the immediate pressure of raising additional capital, which can be reassuring for investors.
- 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
“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 $21.00
-
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 $35.2 million
- 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
-
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 $424.6 million $645.7 million
-
R&D Expenses: Research and development expenses were
$159.8 million $100.9 million
-
G&A Expenses: General and administrative expenses were
$95.0 million $35.2 million $31.0 million
-
Net Loss: The company’s net loss was
$237.7 million $129.9 million
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
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
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | |||||||||||||||
| (amounts in thousands, except share and per share data) | |||||||||||||||
| (unaudited) | |||||||||||||||
Three Months Ended
|
|
Twelve Months Ended
|
|||||||||||||
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
Source: Viridian Therapeutics, Inc.
FAQ
When are the topline clinical data readouts expected for VRDN-001 and VRDN-003?
What was the cash balance at year-end 2023?
How much did Viridian raise in gross proceeds from a public offering in January 2024?
What plans does Viridian have for presenting at NANOS in March 2024?
What was the net loss for Viridian for the year ended December 31, 2023?
What was the increase in research and development expenses from 2022 to 2023?
What was the increase in general and administrative expenses from 2022 to 2023?
