Viridian's drug cures eye disease in phase 3, challenging Amgen's Tepezza

Viridian Therapeutics has achieved primary and secondary endpoints in its phase 3 clinical trial for thyroid eye disease (TED) with its drug candidate veligrotug, also known as VRDN-001. The biotech company, based in Massachusetts, is aiming to differentiate its drug from Amgen's already marketed Tepezza. 

In earlier phase 2 trials, six-week data showed that viridian’s anti-IGF-1R antibody had comparable or superior results to Tepezza on key endpoints, prompting the company to progress to phase 3 trials. The phase 3 study compared VRDN-001 to a placebo, but with Tepezza as a competitor already on the market, Viridian knew they needed more than just positive control results to compete effectively.

The phase 3 results are promising. According to Viridian, 70% of veligrotug recipients experienced at least a 2 mm reduction in proptosis, the medical term for bulging eyes, after five infusions over 15 weeks. In comparison, Tepezza showed response rates of 71% and 83% at week 24 in its clinical trials. The placebo-adjusted response rate for veligrotug was 64%, which falls between the 51% and 73% seen in Tepezza studies.

On another measure, the second Tepezza study showed a 2.06 mm placebo-adjusted change in proptosis after 12 weeks, improving to 2.67 mm by week 18. Viridian observed a 2.4 mm placebo-adjusted change after 15 weeks in their trial.

A clearer distinction between the two drugs emerged on a secondary endpoint, though cross-trial comparisons can be unreliable. Viridian reported a complete resolution of diplopia, which is double vision, in 54% of patients treated with veligrotug, compared to 12% in the placebo group. The placebo-adjusted resolution rate was 43% for veligrotug, surpassing the 28% seen in the Tepezza studies.

Safety and tolerability present another potential differentiating factor for veligrotug. While all data have not yet been released, Viridian reported a 5.5% placebo-adjusted rate of hearing impairment events. This is lower than the 10% rate seen in the Tepezza studies, but this difference was driven by the rate in the placebo arm. The proportion of events in the veligrotug arm was 16%, higher than the 10% in the Tepezza studies.

Viridian anticipates having top-line data from a second study by the end of the year, aiming to file for approval in the second half of 2025. This news caused the company's share price to rise by 13%, reaching over $16 in premarket trading on Tuesday morning. 

As other companies also target Tepezza, questions about veligrotug's competitiveness could intensify. Argenx is conducting a phase 3 trial of FcRn inhibitor efgartigimod in TED, while Roche is evaluating its anti-1L-6R satralizumab in two phase 3 trials. Viridian itself is working on improving veligrotug, with a half-life-extended formulation currently in late-phase development.

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