Vyluma, Inc. released positive top-line results from the second stage of the phase 3 CHAMP (Childhood Atropine for Myopia Progression) on its lead compound NVK002 during the American Academy of Optometry (AAOPT) annual meeting.
Refresh me on NVK002.
NVK002 (low-dose atropine 0.01%) is a proprietary, investigational, preservative-free eye drop intended to be administered on a nightly basis for patients ages 3 to 17.
The formulation features Vyluma’s proprietary technology to address stability, tolerability, and safety for the potential treatment of pediatric myopia.
Didn’t we hear news about this in the last few months?
We did! Vyluma announced in June 2023 that the FDA accepted its new drug application (NDA) for NVK002. See our coverage here.
Gotcha. Now, talk about this CHAMP study.
The 3-arm, randomized, multicenter, double-masked, placebo-controlled, phase 3 CHAMP study enrolled 576 pediatric patients (ages 3 to ≤ 17 years diagnosed with progressive myopia).
Two stages are included:
- Stage 1 (now completed): 3-year treatment period that evaluated the safety and efficacy of NVK002, after which enrolled patients were re-randomized for a masked study.
- Stage 2: a randomized crossover 1-year treatment period to characterize cessation of therapy.
To note, this recent data is based on stage 2 of the study.
Now, these patients and their respective dosages.
Study participants were required to be ages 3 to ≤ 17 years, with a spherical equivalent refraction (SER) of -0.50 D to -6.00 D and astigmatism no worse than -1.50 D.
Dosage-wise, patients were randomized 2:2:3 to receive either a once-daily placebo, NVK002 0.01%, or NVK002 0.02% eye drops for an initial 3-year period.
Before we get to this new data …was there any previous data reported?
Yup, there was! In May 2023, Vyluma reported:
NVK002 at a dose of 0.01% (compared to NVK002 0.02%) achieved statistically significant and clinically meaningful differences at Month 36 in its key outcome measures:
- Responder analysis
- Mean change from baseline in SER
- Mean change from baseline in axial length (AL)
See here for more details.
Now this 4-year data.
Per the company, both NVK002 doses “continued to exhibit a strong safety profile with a low level (8% incidence) of treatment-emergent adverse events (TEAEs).”
Further, no evidence of meaningful rebound was reported for patients who had stopped active treatment and were washed out over the course of a year.
Any new findings?
Not necessarily new, but certainly promising.
Data from NVK002 0.01% demonstrated a “continued widening” of the treatment’s previously-reported effect for the following (when compared to a matched historical placebo group):
- Mean change from baseline in SER
- Mean change from baseline in AL endpoints
And any signs of tachyphylaxis?
Tachyphylaxis—a progressive decrease in response to a dose following repeated administration of a drug—was not evident in NVK002’s 4-year study.
So what does this mean?
Well, according to Vyluma’s Founder and Chairman, Navneet Puri, PhD, the study’s two-stage phase completion indicates potential for NVK002 as “an important, first-in-class pharmaceutical treatment option” for pediatric myopic patients.
Lastly .. what’s next?
With an NDA already accepted by the FDA, a Prescription Drug User Fee Act (PDUFA) date has been set for January 31, 2024.
In other words, now we wait.
And the significance?
If approved, NVK002 has the potential to become the first pharmacological treatment option for myopic progression.
This data was released during the American Academy of Optometry (AAOPT) annual meeting in New Orleans, Louisiana, October 11-14, 2023.