Viridian Therapeutics chooses VRDN-003 as lead subcutaneous therapy for Thyroid Eye Disease

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Viridian Therapeutics has announced the selection of VRDN-003 as its lead subcutaneous (SC) program for thyroid eye disease (TED).

In a press release from Viridian,¹ the company states the decision was made based on positive data from a Phase 1 clinical study in healthy volunteers. Furthermore, the data showed VRDN-003 has an extended half-life of 40-50 days, about 4-5 times longer than VRDN-001.

VRDN-001, another product from Viridian Therapeutics, is a differentiated monoclonal antibody targeting insulin-like growth factor-1 receptor (IGF-1R), for the treatment of thyroid eye disease (TED). According to the company, Viridian’s goal is to advance VRDN-001 as a best-in-class IV therapy followed by VRDN-003 as a first- and best-in-class SC therapy for the treatment of TED.¹

According to the company, “VRDN-003 utilizes the same binding domain as VRDN-001 and is engineered with 3 amino acid changes to the Fc region designed to extend the half-life of the parent antibody, and therefore potentially enabling less frequent and more convenient dosing. VRDN-003 is designed to maintain the clinical response of VRDN-001 IV while significantly increasing patient convenience.”¹

“The shared binding domain and pharmacology of VRDN-003 with VRDN-001 allow us to leverage the clinical activity of VRDN-001 IV at different exposure levels to confidently select doses for VRDN-003 that we expect to demonstrate an impactful clinical response,” said Tom Ciulla, Viridian’s Chief Development Officer. “We are enthusiastic about delivering a product with highly attractive, low-volume subcutaneous dosing schedules that we firmly believe will be preferred by patients.”

Steve Mahoney, President and CEO of Viridian Therapeutics, discussed the results in a press release.

“The VRDN-003 data exceeded our expectations as a potential best-in-class treatment option for patients affected by TED and support advancing dosing regimens as infrequently as once every eight weeks, which we believe could be transformative for TED patients who currently only have access to intravenous (IV) IGF-1R therapy,” said Mahoney. “The data reinforce our confidence in VRDN-003’s rapid development as a low-volume, self-administered, subcutaneous product. Given VRDN-003’s comparable pharmacology with VRDN-001, which has already generated compelling clinical data in TED patients, we are excited about the clinical potential of this program for patients and our ability to rapidly move toward pivotal development.”

VRDN-003 was dosed in 4, single-dose cohorts of healthy volunteers at a concentration of 150 mg/ml receiving 5 mg/kg IV (n=4), 300 mg SC (n=6), 15 mg/kg IV (n=4) and 600 mg SC (n=6). A fifth cohort of 2 doses of VRDN-003 (n=4) is ongoing.¹

Viridian stated that based on the results reported, the company expects to initiate global pivotal clinical trials of VRDN-003 in mid-2024 with planned trials in both active and chronic TED patients pending alignment with regulatory authorities. Both VRDN-001 SC and VRDN-002 SC development have been deprioritized.¹

References:

1. Viridian Therapeutics Announces Positive Clinical Data in Healthy Volunteer Study and Selects VRDN-003 as Potential Best-in-Class Subcutaneous anti-IGF-1R Program with Extended Half-Life for Pivotal Development in Thyroid Eye Disease. Press Release. Released December 18, 2023. Accessed December 18, 2023. https://www.businesswire.com/news/home/20231218302697/en/Viridian-Therapeutics-Announces-Positive-Clinical-Data-in-Healthy-Volunteer-Study-and-Selects-VRDN-003-as-Potential-Best-in-Class-Subcutaneous-anti-IGF-1R-Program-with-Extended-Half-Life-for-Pivotal-Development-in-Thyroid-Eye-Disease