Viridian Therapeutics, Inc. has submitted a Biologics License Application (BLA) to the FDA for veligrotug (veli), its investigational candidate under development for the treatment of thyroid eye disease (TED).
The company reported that this submission was conducted in late October, following recent consultation with the federal agency—despitethe ongoing government shutdown.
How exciting! Let’s start with a rundown on veligrotug.
It’s definitely been a minute (just over 5 months, to be exact) since we last reported on this Breakthrough Therapy-designated (BTD) candidate.
What it is: An anti-insulin-like growth factor-1 receptor (IGF-1R) monoclonal antibody.
What it does: Blocks cell surface receptor activity and targets antibody and protein engineering for specific diseases such as TED.
- See here for why Viridian is focusing on IGF-1Rs (hint: it has to do with it being overexpressed in—and a clinically-validated target for—TED).
And how does it work?
Veli’s intended purpose (and potential) is to reduce the tissue swelling and inflammation associated with TED via an intravenous (IV) injection.
Its dosing: Five 30-minute infusions, administered 3 weeks apart.
Alrighty, now let’s talk BLA.
The submission was based on clinical data from two supporting pivotal phase 3 trials that evaluated veli’s safety and efficacy among patients with active and chronic TED.
- THRIVE (NCT05176639)
- See here for positive topline (15-week) data
- And click here for 52-week data
- THRIVE-2 (NCT06021054)
- Positive data can be found here
For both studies: Veli was found to be generally well tolerated, met all primary and secondary endpoints as well as demonstrated “a rapid onset of clinical benefit and statistically significant and meaningful effect on multiple diplopia endpoints.”
Hone in on those 52-week findings for a moment.
As we previously reported, the focus for the THRIVE study’s long-term durability data was on the proptosis responder rate.
In fact: Just shy of 75% of veli-treated patients who were proptosis responders at Week 15—and continued follow-up to the end of the study—maintained their proptosis response.
And how does veli compare to other TED therapeutics?
See here for clinical insights from Viridian’s President and CEO Steve Mahoney.
So what’s next for this BLA?
Thanks to veli receiving BTD, it could potentially receive an expedited clinical pathway toward potential regulatory approval and eventual commercial launch (with a 2026 target).
Specifically: The therapeutic is eligible for priority review, which would shorten the FDA’s review period from 10 months (as is standard) to 6 months.
- Take note: This only applies to drugs that could potentially offer significant improvements in safety or effectiveness over existing treatments.
And when will we know if Priority Review is granted?
Sometime soon. As Viridian noted its BLA submission was in “late October,” we should hopefully hear from the FDA anytime between now and next month.
To be more precise: The agency typically informs a BLA sponsor (Viridian, in this case) of its decision to either accept or reject the application for review—as well as whether Priority Review is granted—within 60 days of a submission.
So, as always… stay tuned!