UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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| Item 2.02 | Results of Operations and Financial Condition. |
On May 12, 2022, Viridian Therapeutics, Inc. (the “Company”), issued a press release reporting financial results for the three months ended March 31, 2022.
The press release is attached hereto as Exhibit 99.1, which is furnished under Item 2.02 of this Current Report on Form 8-K and shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Exchange Act or the Securities Act of 1933, as amended (the “Securities Act”), regardless of any general incorporation language in such filing.
| Item 7.01 | Regulation FD Disclosure. |
On May 12, 2022, the Company made available an updated investor presentation. A copy of the investor presentation is furnished herewith as Exhibit 99.2 and incorporated by reference herein.
The investor presentation is attached hereto as Exhibit 99.2, which is furnished under Item 7.01 of this Current Report on Form 8-K and shall not be deemed to be “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Exchange Act or the Securities Act, regardless of any general incorporation language in such filing.
| Item 9.01 | Financial Statements and Exhibits |
(d) Exhibits.
| Exhibit Number |
Exhibit Description | |
| 99.1 | Press release, dated May 12, 2022 | |
| 99.2 | Viridian Therapeutics, Inc. Investor Presentation, dated May 2022 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Viridian Therapeutics, Inc. | ||||||
| Date: May 12, 2022 | By: | /s/ Jonathan Violin | ||||
| Jonathan Violin | ||||||
| President, Chief Executive Officer, and Director | ||||||
Exhibit 99.1
VIRIDIAN THERAPEUTICS REPORTS FIRST QUARTER 2022
FINANCIAL RESULTS AND PROVIDES CORPORATE UPDATES
— Phase 1/2 clinical trial for VRDN-001 currently recruiting Thyroid Eye Disease (TED) patients at sites in the U.S. and Canada; top line proof of concept data expected in the third quarter of 2022 —
— Interim results for VRDN-001 in healthy volunteers show saturation of IGF-1 response at doses as low as 3 mg/kg, supporting potential efficacy of lower doses in TED —
— Adverse events reported to date for VRDN-001 in healthy volunteers are generally comparable to placebo, with no drug related events of hyperglycemia, hearing loss, or muscle spasms reported at any dose including top dose of 20 mg/kg —
— VRDN-002 Phase 1 clinical trial dose escalation complete: First-In-Human, healthy volunteer trial for VRDN-002 on track for top line data in the third quarter of 2022—
— Ended 1Q 2022 with cash, cash equivalents and short-term investments of $175 million, expected to fund operations into 2024 —
— Conference call today at 4:30 p.m. ET —
Waltham, Mass., May 12, 2022 — Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company advancing new treatments for patients suffering from serious diseases underserved by current therapies, today announced financial results for the first quarter ending March 31, 2022 and provided corporate updates, including interim results for the healthy volunteer portion of the Company’s ongoing proof of concept trial of VRDN-001 in Thyroid Eye Disease (TED).
“Our ongoing VRDN-001 and VRDN-002 clinical trials are poised to deliver data in the coming months that we believe will transform Viridian and significantly advance our goal of improving patient care in Thyroid Eye Disease,” said Jonathan Violin, Ph.D., President and CEO of Viridian Therapeutics. “Our first clinical results are highly encouraging. The VRDN-001 healthy volunteer interim data show saturation of IGF-1 response at all doses tested, confirming robust IGF-1R inhibition as seen in our preclinical data, with an excellent safety and tolerability profile. We look forward to seeing the first efficacy data from TED patients soon.”
First Quarter 2022 and Recent Highlights
VRDN-001: Viridian’s most advanced product candidate VRDN-001 is a differentiated humanized monoclonal antibody that binds and blocks the insulin-like growth factor-1 receptor (IGF-1R) with sub-nanomolar affinity. This mechanism of action is clinically and commercially validated for the treatment of TED. The Company’s ongoing first clinical trial for VRDN-001 is a Phase 1/2 proof of concept study that includes multiple randomized, placebo-controlled cohorts of TED patients. The trial is designed to assess the potential for VRDN-001 to provide rapid improvement of signs and symptoms of TED at six weeks, after two intravenous (IV) infusions of VRDN-001. The Company expects to announce top line proof of concept clinical data from two patient cohorts in the third quarter of 2022.
Dose escalation and healthy volunteer enrollment is complete, and the Company continues to enroll TED patients at sites in the U.S. and Canada. Each TED cohort includes eight patients randomized in a 3:1 ratio to receive VRDN-001 or placebo. The first cohort is evaluating two infusions of 10 mg/kg VRDN-001; the second cohort is evaluating two infusions of 20 mg/kg VRDN-001.
The healthy volunteer portion of the trial includes doses of 3 mg/kg, 10 mg/kg and 20 mg/kg in 13 subjects. No drug related adverse events associated with hyperglycemia, hearing loss or muscle spasms have been reported to date. Other adverse events have been generally comparable to placebo; to date, there have been no infusion reactions or serious adverse events. Interim data for plasma levels of IGF-1, a biomarker for target engagement, show a rapid increase that saturated after the first infusion at levels that were similar for all doses tested, including 3 mg/kg. Based on these results the Company now plans to enroll a cohort of TED patients at a dose of 3 mg/kg following the completion of the 10 mg/kg and 20 mg/kg cohorts in this trial. The Company expects to report top-line data from the 3 mg/kg cohort in the fourth quarter of 2022.
“The interim healthy volunteer data suggests robust activity of VRDN-001 at doses from 3 mg/kg to 20 mg/kg, with excellent safety and tolerability based on our observations to date. In TED patients we are assessing safety and tolerability as well as multiple efficacy endpoints and expect to report top line data on proptosis, clinical activity score and diplopia,” said Dr. Barrett Katz, M.D., M.B.A., Chief Medical Officer of Viridian Therapeutics. “These efficacy measurements will be assessed at six weeks, after two infusions of VRDN-001. We will report the same endpoints used to evaluate teprotumumab, focusing on mean change from baseline in proptosis reduction, but will also report proptosis responder rate, clinical activity score, and diplopia.”
VRDN-002: Viridian’s second product candidate, VRDN-002, is a distinct, next-generation IGF-1R antibody incorporating half-life extension technology, designed to support administration as a convenient, low-volume, subcutaneous injection for the treatment of TED. In March 2022, Viridian announced dosing of the first subject in a first-in-human, healthy volunteer Phase 1 clinical trial evaluating VRDN-002. This is a single ascending dose trial to explore safety, tolerability, pharmacokinetics and pharmacodynamics of intravenously administered VRDN-002 at doses of 3 mg/kg, 10 mg/kg, and 20 mg/kg in up to 16 healthy volunteers. The Company has completed dose escalation and expects to announce top line data from this Phase 1 trial in the third quarter of 2022. Results from this trial will confirm the feasibility of a low-volume subcutaneous dosing paradigm for TED patients; the company is planning a subcutaneous proof of concept trial in TED patients as the next step in VRDN-002 development. The Company believes a low-volume subcutaneous injection would improve convenience for patients and physicians, mitigate treatment burdens, and expand the settings of care for TED therapies.
Discovery Pipeline: Viridian’s pipeline expansion is focused on additional opportunities that leverage validated mechanisms and technologies in therapeutic areas underserved by today’s available medicines. The most advanced of these programs is VRDN-004, a therapeutic monoclonal antibody program currently in discovery stage for an undisclosed rare disease. VRDN-005 is a second discovery-stage program for another undisclosed indication in which the Company believes patient care can be advanced with a novel therapeutic monoclonal antibody.
First Quarter 2022 Financial Results
Cash Position: Cash, cash equivalents and short-term investments were $175 million as of March 31, 2022, compared with $197 million as of December 31, 2021.
After the first quarter ended, the Company entered into a debt financing agreement with Hercules Capital, Inc. for up to $75 million. Under the terms of the agreement, Viridian drew an initial $5 million at closing.
An additional $20 million is available at the Company’s request through June 15, 2023, with an additional $25 million available upon the Company’s achievement of certain milestones, and the remaining $25 million available subject to final lender approval. The Company is under no obligation to draw funds in the future.
Excluding this $75 million credit facility, the Company believes that its current cash, cash equivalents and short-term investments will be sufficient to fund its operations into 2024.
R&D Expenses: Research and development expenses were $17.7 million during the first quarter of 2022, compared with $13.8 million for the same period last year. The increase in research and development expenses was primarily driven by personnel related costs, license fees and clinical trial costs for VRDN-001 and VRDN-002. These increases were offset by expenses related to manufacturing and IND-enabling studies for VRDN-001 and VRDN-002 that were incurred in the first quarter of 2021.
G&A Expenses: General and administrative expenses were $8.4 million during the first quarter of 2022, compared with $6.2 million for the same period last year. The increase in general and administrative expenses was driven by increases in personnel-related costs, including severance, share-based compensation charges, and consulting expenses.
Net Loss: The Company’s net loss was $25.7 million for the first quarter of 2022, compared with $18.5 million for the same period last year. The increase in net loss was driven by increased operating costs, as described above, as well as lower revenue from our collaboration with Zenas in the first quarter of 2022 compared to 2021.
Shares Outstanding: As of March 31, 2022, Viridian had approximately 42,883,007 shares of common stock outstanding on an as-converted basis, which included 27,169,422 shares of common stock outstanding and an aggregate of approximately 15,713,585 shares of common stock issuable upon the conversion of 212,566 and 23,126 shares of Series A and Series B preferred stock, respectively.
First Quarter 2022 Financial Results Conference Call
Viridian’s management will host a conference call today at 4:30 p.m. ET to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-270-2148 for domestic participants and 1-412-902-6510 for international participants. A live webcast of the conference call can be accessed through the “Events” page in the Investors section of the Viridian Therapeutics website. Following the live webcast, an archived version of the call will also be available on the website.
About Viridian Therapeutics, Inc.
Viridian Therapeutics is a biotechnology company advancing new treatments for patients suffering from serious diseases but underserved by today’s therapies. Viridian’s most advanced program, VRDN-001, is a differentiated humanized monoclonal antibody targeting insulin-like growth factor-1 receptor (IGF-1R), a clinically and commercially validated target for the treatment of thyroid eye disease (TED). Viridian’s second product candidate, VRDN-002, is a distinct anti-IGF-1R antibody that incorporates half-life extension technology and is designed to support administration as a convenient, low-volume, subcutaneous injection. TED is a debilitating autoimmune disease that causes inflammation and fibrosis within the orbit and behind the eye which can cause double vision, pain, and potential blindness. Patients with severe disease often require multiple remedial surgeries to the orbit, eye muscles and eyelids. Viridian is based in Waltham, Massachusetts.
Note Regarding 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 include, without limitation, statements regarding the Company’s expectations and guidance regarding its business plans and objectives for its product candidates and pipeline, including the therapeutic potential and clinical benefits thereof, the sufficiency of the Company’s financial position and its projected cash runway, the timing, progress and plans for the Company’s ongoing and future research and clinical development programs, trial design and protocols for ongoing clinical trials, and expectations regarding the timing of top line data for the VRDN-001 and VRDN-002 programs, the safety and efficacy of the Company’s product candidates, and the unpredictable relationship between preclinical study results and clinical study results. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. 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: uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; manufacturing risks; competition from other therapies or products; other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations; the Company’s future operating results and financial performance; the timing of pre-clinical and clinical trial activities and reporting results from same; the effects from the COVID-19 pandemic on the Company’s research, development and business activities and operating results, including those risks set forth under the caption “Risk Factors” in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 11, 2022 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 we, nor our 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 our views as of any date subsequent to the date hereof.
VIRIDIAN THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(amounts in thousands, except share and per share data)
(unaudited)
| Three Months Ended March 31, |
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| 2022 | 2021 | |||||||
| Revenue: |
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| Collaboration Revenue - related party |
$ | 216 | $ | 1,451 | ||||
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| Total revenue |
216 | 1,451 | ||||||
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| Operating Expenses: |
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| Research and development |
17,746 | 13,806 | ||||||
| General and administrative |
8,359 | 6,160 | ||||||
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| Total operating expenses |
26,105 | 19,966 | ||||||
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| Loss from operations |
(25,889 | ) | (18,515 | ) | ||||
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| Other income |
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| Interest and other income |
196 | 55 | ||||||
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| Net loss |
(25,693 | ) | (18,460 | ) | ||||
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| Change in unrealized loss on investments |
(778 | ) | (13 | ) | ||||
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| Comprehensive loss |
$ | (26,471 | ) | $ | (18,473 | ) | ||
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| Net loss |
$ | (25,693 | ) | $ | (18,460 | ) | ||
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| Net loss per share, basic and diluted |
$ | (0.98 | ) | $ | (2.91 | ) | ||
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| Weighted-average shares used to compute basic and diluted loss per |
26,126,092 | 6,336,347 | ||||||
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VIRIDIAN THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(amounts in thousands)
(unaudited)
| March 31, | December 31, | |||||||
| 2022 | 2021 | |||||||
| Cash and cash equivalents |
$ | 30,858 | $ | 42,299 | ||||
| Short-term investments |
$ | 144,570 | $ | 154,666 | ||||
| Total assets |
$ | 183,165 | $ | 203,709 | ||||
| Total liabilities |
$ | 16,523 | $ | 15,993 | ||||
| Total stockholders’ equity |
$ | 166,642 | $ | 187,716 | ||||
| Total liabilities and stockholders’ equity |
$ | 183,165 | $ | 203,709 | ||||
Exhibit 99.2 Viridian Therapeutics May 2022
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements relating to Viridian Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates, the clinical utility of our therapeutic candidates and our intellectual property position. You can identify forward-looking statements by the use of forward-looking terminology including “believes,” “expects,” “may,” “will,” “should,” “seeks,” “intends,” “plans,” “pro forma,” “estimates,” or “anticipates” or the negative of these words and phrases or other variations of these words and phrases or comparable terminology. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. These forward-looking statements should not be relied upon as predictions of future events as we cannot assure you that the events or circumstances reflected in these statements will be achieved or will occur. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make due to a number of important factors, including uncertainty regarding our ability to raise additional capital and fund our development programs, risks inherent in conducting clinical trials and seeking to demonstrate safety and efficacy to the satisfaction of the applicable regulatory authorities, and those risks discussed in “Risk Factors” and elsewhere in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 11, 2022, subsequently filed periodic reports and in other filings we make with the SEC from time to time. The forward-looking statements in this presentation represent our views as of the date of this presentation. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. 2
Investment highlights Viridian is engineering and developing best-in-class therapeutic antibodies for patients suffering from serious diseases that are underserved by current therapies Lead programs are novel and differentiated IGF-1R monoclonal antibodies being developed for Thyroid Eye Disease (“TED”), a >$3.5B estimated market opportunity • VRDN-001: Promising normal healthy volunteer (NHV) data, key proof of concept clinical data in TED in 3Q22, with potential to show meaningful improvements in proptosis, the key characteristic of TED • VRDN-002: Clinical data (IV, SAD, NHV) in 3Q22 that could de-risk clinical development of low volume subcutaneous injection Evaluating multiple opportunities to expand our pipeline by identifying and developing novel and differentiated monoclonal antibodies targeting robust and de-risked mechanisms of action Experienced management team and board backed by leading life science investors (1) Cash, cash equivalents and short-term investments $175M with cash runway into 2024, excluding $75M credit facility (2) 43M total common shares outstanding on an as converted basis – implied market (3) capitalization of $431M (1) As of March 31, 2022 (2) As of May 11, 2022, Viridian had approximately 42,908,763 shares of common stock outstanding on an as-converted basis, which included 27,927,423 shares of common stock and approximately 14,981,340 shares of common stock issuable upon the conversion of 201,583 and 23,126 3 shares of Series A and Series B preferred stock respectively (3) As of May 11, 2022 based on a stock price of $10.05 Investment Highlights
Viridian’s discovery engine leverages core expertise in antibody discovery and engineering 3 5 2 4 1 First entrant Significant market First entrant Viridian Purpose-built validates a new opportunity; leaves room for engineering clinical team to mechanism of serious disease improvement creates improved rapidly advance action with limited mAb targeted competition development plan 4 Strategy
Pipeline Phase 2 Preclinical Phase 1 Discovery Phase 3 Proptosis reduction VRDN-001 – Intravenous (potentially subcutaneous) PoC data 3Q 2022 Thyroid Eye FIH data (IV, SAD, NHV) Disease VRDN-002 – Subcutaneous 3Q 2022 (IGF-1R) VRDN-003 Rare Disease VRDN-004 (Undisclosed) Undisclosed VRDN-005 5 5 Pipeline
Substantial market opportunity in thyroid eye disease • Debilitating, progressive and sight-threatening orphan disease that significantly impacts quality of life 1 • 20-25,000 U.S. patients diagnosed with active disease each year 1 • Similar epidemiology ex-U.S. 2 • >$3.5B potential peak U.S. annual net sales (1) Viridian market research 6 (2) Horizon FY 2021 earnings release
Tepezza® launch reset TED landscape, creating opportunities to evolve treatment paradigms PEAK SALES ESTIMATES >$3.5B; POTENTIAL TO IMPROVE VALUE NO COMPETITION PROPOSITION OF LEAD MOLECULE ® TEPEZZA Revenues • More compelling product profile by targeting 5 3 lower dose and treatment burden – ~65% >$3.5B physicians prefer improved IV regimen over current options 4 ~$2.2B • Transform market by introducing low volume 5 SC injection - ~70% physicians prefer low 4 ~$1.7B volume SC injection offering over current options 3 $1.66B 2 • Expand market by targeting broader prescriber $820M 1 base and more accessible sites of care >$30-$40M 4 $0.5B Peak U.S. Annual • Opportunity to accelerate payer adoption and Initial 2020 2020A 2021A 2022E Net Sales guidance patient access Actual net sales Estimated (1) Horizon FY 2019 earnings release (5) Viridian U.S. market research (n=102 ophthalmologists (2) Horizon FY 2020 earnings release and endocrinologists who treat TED) 7 (3) Horizon FY 2021 earnings release (4) Horizon Q1 2022 earnings release
History of IGF-1R antibodies in thyroid eye disease Teprotumumab (R1507), River Vision Development FDA grants Breakthrough Tepezza® approved IGF-1R mAb, enters clinic founded to develop designation for January 2020 for oncology teprotumumab for TED teprotumumab for TED 2016 2020 2006 2011 2004 2009 2013 2017 2020 Smith et al identify Ph2 oncology trials fail to Teprotumumab enters Horizon Therapeutics Viridian licenses IGF1R blockade as a meet efficacy criteria. Roche Ph2 clinical trials for TED acquires River Vision Immunogen/Sanofi potential mechanism for discontinues teprotumumab and starts Ph3 trials IGF-1R mAb for TED treating TED* development. 8 8 *Smith TJ and Hoa N, J Clin Endocrinol Metab. 2004
An IGF-1R antibody elicits rapid, robust response with dramatic change in proptosis after two doses Opportunity For Early And Clear Proptosis PoC TEPROTUMUMAB PH3 PROPTOSIS RESPONSE CHANGE FROM BASELINE IN PROPTOSIS Randomized, double-masked, placebo controlled 83 active TED patients: 41 active treatment, 42 placebo IV infusion, first infusion 10 mg/kg, subsequent 7 infusions 20 mg/kg q3w Primary endpoint: proptosis responder rate 9 9 Data from Douglas et al, NEJM 2020
VRDN-001: previous clinical data and successful pre-IND meeting confirms rapid path to clinical proof of concept RAPID PROGRESS TO DATE NEAR TERM CLINICAL DATA • Identified AVE-1642 as a candidate for treating TED – Mechanism comparable to teprotumumab – Previously administered to >100 oncology patients with robust PK/PD, good tolerability and no antibody-elicited anti- drug antibodies; data accelerates clinical plans • Licensed exclusive worldwide rights to AVE-1642 from ImmunoGen Proof of concept data in TED patients (proptosis reduction at 6 weeks) • High solubility for potential low volume subcutaneous expected in 3Q 2022 injection achieved - 150 mg/ml • Tech transfer complete and manufacturing underway • Successful pre-IND meeting in July 2021 • FDA clearance of IND in November 2021 10 VRDN-001
AVE-1642 and teprotumumab showed similar profiles in oncology trials AVE-1642 TEPROTUMUMAB Half-life (in oncology (1) (4,5) 10 days 10 days patients) (1) (6) IGF-1 increase 6X 3X (2) (7) Affinity (K ) 0.21 nM 5 nM D 5-52% 11-50% Hyperglycemia (3) (4,6,8,9) Severe in 5.6% of patients (n=106, 2 trials) Severe in 3% of patients (n=335, 4 trials) (1) Soria et al, EJC, 2013; n = 27; solid tumor; 3-24 (6) Pappo et al, Cancer, 2014: n= 163; sarcoma; 9 mg/kg, q3w mg/kg, qw (2) AVE-1642 Investigator’s Brochure (7) Schanzer et al, JBC, 2014 (3) Sanofi-Aventis AVE-1642 FDA annual report 2010 (8) Rodon et al, JCO 2007: n=21, solid (4) Gore et al., MCT, 2009: n=36; solid tumor; 3-9 tumor/lymphoma; 1-16 mg/kg, q3w mg/kg qw, 16 mg/kg q3w (9) Pappo et al, JCO 2011: n=115, sarcoma; 9mg/kg (5) Kurzrock et al, CCR, 2010: n=37; solid qw or 27 mg/kg, q3w 11 tumor/lymphoma; 1-9 mg/kg, qw VRDN-001
IGF-1 levels generated in oncology patients suggest VRDN-001 target engagement at doses as low as 3 mg/kg 3 mg/kg 6 mg/kg 12 mg/kg 18 mg/kg 24 mg/kg IGF1 IGF2 IGFBP3 12 Soria et al, EJC, 2013; n = 27; solid tumor; 3-24 mg/kg, q3w VRDN-001
VRDN-001 Phase 1/2 trial design: PoC data from two cohorts of TED patients expected in 3Q 2022 POPULATION: • TED patients: active moderate to severe TED, same as teprotumumab Phase 3 • Normal Healthy Volunteers (NHVs): conducted in PROOF OF CONCEPT parallel to accelerate dose escalation decisions NHV run in (n=4) STUDY DESIGN: • Randomized placebo controlled NHV (n=4) TED cohort 1 • Multiple Ascending Dose in NHV and TED cohorts - 10mg/kg q3w q3w x 2 doses TED patients (n=8) nd q3w x 2 doses • Dose escalation after 2 NHV dose in each cohort Proptosis readout:6wks 12wks ENDPOINTS: NHV (n=4) • TED patients (efficacy at 6 and 12 weeks, follow up at TED cohort 2 24 and 52 weeks) - proptosis, clinical activity score, 20mg/kg q3w TED patients (n=8) diplopia, objective measures of orbital volume and q3w x 2 doses EOMs, ocular motility, QoL Proptosis readout:6wks 12wks • NHVs (6 weeks follow up) - safety, tolerability, PK, PD FLEXIBLE PROTOCOL: • Option for additional TED cohorts; dose / regimen to be selected by company based on PoC data 13 NHV run-in and TED Cohorts 1-2: 3:1 VRDN-001: PBO VRDN-001
Interim results from healthy volunteer portion of Phase 1/2 TED proof of concept trial • Randomized double-blind placebo-controlled evaluation of safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers • Doses: two infusions of 3, 10, or 20 mg/kg q3w • Preliminary safety observations: no serious adverse events or infusion reactions, and no drug-related hyperglycemia, hearing loss, muscle spasms ‒ Adverse events were generally mild and similar in placebo and drug cohorts • Rapid increase in plasma IGF-1 levels indicate VRDN-001 maximally inhibits IGF1R at doses from 3-20 mg/kg • Proof of concept portion of study in TED patients is ongoing • Based on these results, we will evaluate a 3 mg/kg arm of VRDN-001 in TED patients in an additional cohort, which could enable subcutaneous dosing of VRDN-001 Safety/tolerability data are for 10 subjects who have completed the trial through Day 50, one subject with observations through Day 43, 1 subject who withdrew prior to second infusion for reasons unrelated to study drug and has observations through Day 35, and a replacement subject with observations through Day 8. 14 IGF-1 data are for 8 subjects who have completed the trial, and for 4 subjects (all in the 20mg/kg cohort) with samples through day 15 (n=2) or Day 29 (n=2) VRDN-001
Interim safety and tolerability observations of VRDN-001 in healthy volunteers (TEAE) ® TEPEZZA label Study VRDN-001-101, normal healthy volunteers VRDN-001 VRDN-001 Placebo Masked top dose cohort ® TEPEZZA Placebo Adverse Reactions 3 mg/kg 10 mg/kg (VRDN-001 20 mg/kg (n=84), n (%) (n=86), n (%) (n=3), n (n=3), n (n=2), n or PBO, n=5), n Muscle spasms 21 (25%) 6 (7%) 0 0 1 0 Nausea 14 (17%) 8 (9%) 0 0 0 0 Alopecia 11 (13%) 7 (8%) 0 0 0 0 Diarrhea 10 (12%) 7 (8%) 0 0 0 0 Fatigue 10 (12%) 6 (7%) 0 0 0 0 Hyperglycemia 8 (10%) 1 (1%) 0 1* 0 0 Hearing impairment 8 (10%) 0 (0%) 0 0 0 0 Dysgeusia 7 (8%) 0 (0%) 0 0 0 0 Headache 7 (8%) 6 (7%) 2* 0 0 0 Dry skin 7 (8%) 0 (0%) 0 0 0 0 ** Hypotension 2.5% N/A 0 1 0 0 ** Hypertension 1% N/A 1* 0 0 1* Infusion reactions 4% N/A 0 0 0 0 • No SAEs in any cohort The 20 mg/kg cohort is ongoing and remains masked; data shown includes both placebo and study drug *Deemed unrelated to study drug by the masked investigator **From FDA Clinical Review of BLA 761143; not listed on label as rates were below 5% Hyperglycemia event was mild and deemed unrelated to study drug by the masked investigator because hyperglycemia was also present at baseline Hypertension events were mild, and deemed unrelated to study drug by the masked investigator 15 Hypotension event was transient, and blood pressure changes were mild, resolving without treatment Muscle spasm event was transient, resolving without treatment, and deemed related to study drug by the masked investigator VRDN-001
Plasma IGF-1 levels in healthy volunteers indicate VRDN-001 inhibits IGF1R at doses from 3-20 mg/kg Plasma levels of IGF-1 • Rapid and sustained increases in in healthy volunteers administered VRDN-001 plasma IGF-1, a biomarker for IGF- 1R inhibition nd 2 dose 1000 • Similar rate and extent of IGF-1 3 mg/kg increase for all doses tested 10 mg/kg 20 mg/kg – 20 mg/kg data collection is PBO ongoing • Responses are ~ 7x baseline, 1,2 consistent with oncology trial data 100 1 43 22 Dosing Day (1) Soria et al, Eur J Cancer, 2013 16 (2) Moreau et al, Leukemia 2011 Note: Data are mean +/- SEM; data collection for 20m/kg cohort is ongoing VRDN-001 IGF-1 (ng/mL)
VRDN-002: a novel, next-generation IGF-1R mAb for TED • Treatment paradigm inconvenient: 8 IV infusions in 24 weeks (q3w) Current dosing • Dose is not conducive to low volume SC injection: 20mg/kg q3w paradigm suboptimal maintenance dose translates to ~500 mg/week • High affinity for IGF-1R offers potential to reduce dose VRDN-002 designed • Half-life extension provides potential for lower, fewer and/or less frequent doses to optimize dosing • 150mg/mL solubility achieved, enables potential low volume SC injection and delivery • First in human data (IV, SAD, NHV) expected 3Q22; data showing successful extension of mAb half-life would support development of a low-volume SC injection Near term • SAD trial will explore safety, tolerability, PK and PD of IV administered placebo or clinical data VRDN-002 at doses of 3, 10, and 20 mg/kg in up to 16 healthy volunteers • SC drug product ready in early 4Q22, and SC PoC trial initiation in patients shortly thereafter 17
VRDN-002 preclinical data supports potential best-in-class subcutaneous product profile NHP PHARMACOKINETICS NHP PK • VRDN-002 binds IGF-1R with high affinity • IV and SC PK in non-human primates show 100 extended half-life and sustained exposure vs. teprotumumbab and VRDN-001 – VRDN-002 plasma concentrations 3x 3-fold higher 28 days after dosing 10 VRDN-002, 10 mg/kg IV VRDN-002, 10 mg/kg SC – VRDN-002 cyno half life at 10 mg/kg Teprotumumab, 10 mg/kg IV ~2.2x teprotumumab AVE-1642, 10 mg/kg IV* 1 0 10 20 30 Time (days) 18 *historical data from AVE-1642 IND plasma [mAb] ug/mL
Leadership Management team Jonathan Violin, Ph.D. President & CEO Vahe Bedian, Ph.D. Chief Scientist Kristian Humer Chief Financial & Business Officer Barrett Katz, M.D. Chief Medical Officer Deepa Rajagopalan, M.D. SVP, New Product and Portfolio Development Janielle Newland SVP, Human Resources Lara Meisner, J.D. SVP, General Counsel Angela She, Ph.D. VP, R&D Operations and Chief of Staff 19 Viridian Leadership
Leadership Board of Directors Tomas Kiselak Chairman of the Board Managing Member, Fairmount Funds Management, LLC Peter Harwin Director Managing Member, Fairmount Funds Management, LLC Arlene Morris Director Chief Executive Officer, Willow Advisors, LLC Jennifer Moses, CPA Director, Audit Committee Chair Chief Financial Officer, G1 Therapeutics Jonathan Violin, Ph.D. Director Chief Executive Officer, Viridian Therapeutics, Inc. 20 Viridian Leadership
2022 milestones IV VRDN-001 IV VRDN-001 IV VRDN-001 NHV data Proptosis PoC data 3mg/kg data VRDN-002 IV VRDN-002 IND clearance FIH data Q1 Q2 Q3 Q4 21 Milestones
Investment highlights Viridian is engineering and developing best-in-class therapeutic antibodies for patients suffering from serious diseases that are underserved by current therapies Lead programs are novel and differentiated IGF-1R monoclonal antibodies being developed for Thyroid Eye Disease (“TED”), a >$3.5B estimated market opportunity • VRDN-001: Promising normal healthy volunteer (NHV) data, key proof of concept clinical data in TED in 3Q22, with potential to show meaningful improvements in proptosis, the key characteristic of TED • VRDN-002: Clinical data (IV, SAD, NHV) in 3Q22 that could de-risk clinical development of low volume subcutaneous injection Evaluating multiple opportunities to expand our pipeline by identifying and developing novel and differentiated monoclonal antibodies targeting robust and de-risked mechanisms of action Experienced management team and board backed by leading life science investors (1) Cash, cash equivalents and short-term investments $175M with cash runway into 2024, excluding $75M credit facility (2) 43M total common shares outstanding on an as converted basis – implied market (3) capitalization of $431M (1) As of March 31, 2022 (2) As of May 11, 2022, Viridian had approximately 42,908,763 shares of common stock outstanding on an as-converted basis, which included 27,927,423 shares of common stock and approximately 14,981,340 shares of common stock issuable upon the conversion of 201,583 and 23,126 22 shares of Series A and Series B preferred stock respectively (3) As of May 11, 2022 based on a stock price of $10.05 Investment Highlights
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