Ocular Therapeutix (NASDAQ:OCUL) shares tumbled 25% on Tuesday following publication of top-line results from the company’s Phase 3 SOL-1 superiority trial testing Axpaxli in patients with wet age-related macular degeneration. EyePoint (NASDAQ:EYPT) stock also moved lower, falling 15%, while Regeneron Pharmaceuticals (NASDAQ:REGN) shares remained essentially unchanged.
The SOL-1 study met its pre-specified primary endpoint. At 36 weeks, 74.1% of patients treated with Axpaxli maintained visual acuity compared with 55.8% of patients receiving Regeneron’s Eylea, an observed difference of 18.3% (p=0.0006). At the 52-week mark the gap widened, with 65.9% of Axpaxli patients maintaining vision versus 44.2% in the Eylea arm, an observed difference of 21.7% (p<0.0001).
Despite the statistical significance of those outcomes, investors reacted negatively. Market participants appeared to focus on the stronger-than-expected performance of Eylea in the control arm and on what they perceived as a smaller-than-anticipated efficacy differential between Axpaxli and the comparator.
Safety data in the top-line release indicated there were no treatment-related ocular or systemic serious adverse events reported. However, Axpaxli-treated patients experienced higher rates of certain non-serious ocular events compared with Eylea: vitreous floaters occurred in 12.4% of Axpaxli patients versus 1.2% with Eylea, and cataracts were reported in 7.1% of Axpaxli patients versus 2.9% in the control arm.
Analysts offered measured perspectives on the results. TD Cowen analyst Tara Bancroft said, "While the 18% benefit in the primary may have missed the investor bar, we believe the impressive BCVA data in rescue-free patients (-<3 letters at 6 & 9 mos.) and strong anatomical consistency more than offset."
Needham analyst Serge Belanger observed that, "There is the potential some retinal disease specialists may question the durability of Axpaxli vs. Eylea HD or Vabysmo, both of which are believed to offer slightly longer duration than Eylea."
RBC analyst Lisa Walter added, "We think the SOL-1 result should be positive for OCUL in the long term, but we understand investors might be initially disappointed by the high control arm response and minimal difference in BCVA among rescue-free patients."
The market reaction underscores investor sensitivity to both comparative efficacy margins and tolerability signals in late-stage ophthalmology trials. While the trial achieved its primary statistical goals, the perceived clinical differentiation versus established therapies and reported non-serious adverse event rates influenced near-term investor sentiment and stock performance.
Key takeaways
- Ocular Therapeutix announced positive top-line SOL-1 results for Axpaxli in wet AMD, meeting the trial's primary endpoint at 36 and 52 weeks.
- Despite statistical significance, OCUL stock fell sharply as investors reacted to a strong control-arm performance by Eylea and a narrower-than-expected efficacy gap.
- Safety signals showed no treatment-related serious adverse events, but higher rates of vitreous floaters and cataracts were reported with Axpaxli.
Sectors impacted: Biotechnology, Pharmaceuticals, Healthcare Equipment & Services.
Risks and uncertainties
- Investor sentiment may remain pressured by the unexpectedly robust response in the Eylea control arm, which affects perceived market differentiation - impacting biotech and pharmaceutical equities.
- Questions around the durability of Axpaxli versus other longer-duration retinal treatments could influence prescribing patterns and competitive positioning in ophthalmology markets.
- Reported higher incidences of vitreous floaters and cataracts with Axpaxli may weigh on regulatory review discussions and clinician adoption, affecting commercial prospects.