Viridian Therapeutics: A Catalyst for Change in Rare Disease Therapeutics at Goldman Sachs Conference

On Monday, June 9, 2025, Viridian Therapeutics Inc. (NASDAQ: VRDN) will take the stage at the Goldman Sachs 46th Annual Global Healthcare Conference in Miami, FL. This presentation marks a pivotal moment for the biotech firm, as it aims to solidify its position as a leader in rare disease therapeutics. Investors should pay close attention to the company's pipeline updates, particularly its lead asset veligrotug and its FcRn inhibitor platform, which could redefine treatment paradigms for conditions like thyroid eye disease (TED) and autoimmune disorders.

Veligrotug: A Breakthrough for Thyroid Eye Disease

Veligrotug, Viridian's Phase 3 lead candidate, is front and center in this presentation. The drug targets active TED, a rare autoimmune disorder with limited treatment options. Recent data highlights its efficacy: 54% of patients achieved diplopia resolution compared to 27% in the placebo group, with 70% of responders maintaining proptosis improvements at 52 weeks. These results are compelling, as current therapies like monoclonal antibodies (e.g., teprotumumab) often require frequent injections and have shorter durability.

The Breakthrough Therapy Designation secured in May 2025 further underscores veligrotug's potential. This FDA designation could accelerate its timeline, with a Biologics License Application (BLA) expected in Q4 2025 and a potential U.S. launch by late 2026. Analysts estimate peak annual sales of $1.2 billion for veligrotug, given its superior efficacy and the ~250,000 TED patients in the U.S. alone.

Expanding the FcRn Inhibitor Platform: Beyond TED

Viridian's broader pipeline hinges on its FcRn inhibitor technology, which targets autoimmune diseases by selectively reducing pathogenic immunoglobulins. The company's second-generation candidates, VRDN-006 (myasthenia gravis) and VRDN-008 (chronic inflammatory demyelinating polyneuropathy, or CIDP), are poised to tap into a $10 billion addressable market.

• VRDN-006: Phase 1 data expected in Q3 2025 could validate the safety and pharmacokinetics of this subcutaneous formulation, positioning it as a more convenient alternative to IV infusions.
• VRDN-008: An IND filing is planned by year-end. Both programs leverage Viridian's proprietary platform, which avoids the off-target effects seen with broader immune suppressants.

Financial Fortitude and Undervalued Valuation

Viridian's financial position is robust, with a $753 million cash balance as of September 2024—enough for a two-year runway. This eliminates near-term dilution risks, a critical advantage in a capital-constrained biotech landscape.

At a $1.5 billion valuation, the stock appears undervalued relative to its pipeline's potential. Analysts project a 50–100% upside if the Goldman Sachs presentation resonates with investors, particularly on execution timelines and data milestones.

Investment Thesis: A Rare Opportunity in Rare Diseases

Viridian's upcoming presentation is a catalyst to revalue its stock. The combination of veligrotug's strong data, Breakthrough status, and FcRn pipeline depth positions it as a disruptor in rare disease therapeutics. Key risks include regulatory hurdles and competition, but the clinical and commercial advantages of its platform suggest a favorable risk-reward profile.

Investors seeking exposure to innovative rare disease therapies should consider VRDN as a speculative buy, with a focus on near-term catalysts like the Goldman Sachs presentation and Phase 1 data for VRDN-006. Historically, buying VRDN on the day of its Goldman Sachs presentation and holding for 30 days has delivered an average return of 114.92% from 2020 to 2024, though with notable volatility and a maximum drawdown of nearly 50%. This underscores the potential upside of the upcoming catalyst, though investors must be prepared for short-term swings. Analysts project a 50–100% upside if the presentation resonates, aligning with this historical performance. For the risk-tolerant, the reward profile remains compelling.

Stay tuned for the June 9 webcast—this could be the start of Viridian's ascent.