Viridian Therapeutics, Inc.

Greetings and welcome to Viridian Therapeutics third quarter 2022 earnings call. At this time, all participants are in a listen only mode. A question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host. John Jordan, Vice President of Investor Relations. Please go ahead.

Thank you, Brock. Good morning, everyone, and welcome to the Viridian conference call to discuss the positive clinical data report earlier today for VRDN001 in patients with thyroid eye disease. Before we begin, I'd like to remind everyone that this conference call and webcast will contain forward-looking statements about the company. These statements are subject to risks and uncertainties that could cause actual results to differ. Please note that these forward-looking statements reflect our opinions only as of today. Except as required by law, we specifically disclaim any obligation to update or revise these forward-looking statements in light of new information or future events. Factors that could cause actual results or outcomes to differ materially from Those expressed in or implied by such forward-looking statements are discussed in greater detail in our most recent filings on Form 10-K and 10-Q and other reports on file with the SEC. I'm joined today by Dr. Jonathan Violin, President and CEO, Dr. Barry Katz, our Chief Medical Officer, and Christian Humer, our Chief Financial Officer and Chief Business Officer. I would now like to turn the call over to Jonathan.

Thanks, John, and good morning, everyone. Thanks for joining us. Over the last quarter, Viridian continued to execute on all pillars of our strategy, designed to deliver the most complete portfolio of products for the treatment of thyroid eye disease across the world. TED, as you know, is currently a $2 billion market in just the United States and is expected to grow to over $4 billion globally, with just one product currently approved. We're advancing a portfolio of intravenous and subcutaneous programs towards this opportunity. For our IV VRDN001 program, we're excited to share positive results from our second proof-of-concept cohort. Data we'll review today for 20 mg per kg confirm the profile we initially presented for 10 mg per kg a few months ago. The data compares favorably to TPEZA on each key endpoint, including proptosis response rate, overall response, change in clinical activity score, or CAS, proportion of patients achieving a CAS of 0 or 1, and resolution of diplopia. VRDN001's safety profile continues to look very promising, with all adverse events mild to moderate, no SAEs, no hearing impairment, no drug-related hyperglycemia, and no infusion reactions reported in the 20 mcg per kg cohort. I'm pleased to share that the three mcg per kg cohorts for VRDN001 is now fully enrolled, in fact, over-enrolled due to patient demand, and we plan to present top-line data in early January 2023. I'm also excited to share that this month we initiated our first phase three trial, the THRIVE trial, in active TED. Sites are now active and are screening patients. In the THRIVE program, we're evaluating two regimens of VRD on 001, the standard aid infusion course and a shorter patient-friendly five-dose regimen, which would be 43% shorter than the aid infusion to PESA regimen. Our confidence in the efficacy of the shorter five-dose regimen is enhanced by new 12-week data from our 10-week per kid cohort, showing that between week 6 and 12, with no further dosing, VRDN001 activity is maintained. This supports our assessment that five infusions of VRDN001 with a 12-week course will deliver the meaningful benefit patients need. In parallel to the VRDN001 program, we also continue to make progress with our subcutaneous programs, where we aim to deliver a convenient, low-volume auto-injector that patients can self-administer at home, and we aim to be the first to market with this offering globally. Today, we reported the final data for VRDM002 from our healthy volunteer study, confirming that VRDM002 PK exceeded our expectations with a half-life of up to 43 days, about four-fold better than Tepeza, VRDM001, and all other IGF-1R antibodies in development. This is actually longer than the initially estimated 30 to 40-day half-life we reported a few months ago and positions OO2 well for its upcoming subcutaneous proof-of-concept trial in TED patients. Both VRDN OO2 and our half-life extended version of VRDN OO1, called VRDN OO3, are on track and advancing quickly. We're poised to bring the first self-administered TED therapy to patients in need of better options. And we're very well funded to execute on these plans with cash to advance rapidly through multiple key milestones. We've ended the quarter with over $430 million in cash, providing a runway into the second half of 2025. Let's now discuss the data from the 20 mg per kg cohort in more detail. I'll hand the call over to Barrett Katz, Viridian's Chief Medical Officer.