Ocular Therapeutix Releases Topline Results for Phase 1 Trial of Axpaxli for Diabetic Retinopathy
Ocular Therapeutix announced positive topline results from the phase 1 HELIOS study, which is evaluating the safety, tolerability, and efficacy of drug candidate Axpaxli versus a sham control in patients with moderately severe to severe nonproliferative diabetic retinopathy (NPDR) without diabetic macular edema (DME). The phase 1 HELIOS study is a multicenter, double-masked, randomized 2:1, parallel group, clinical trial conducted at 10 centers in the United States. The currently reported data set is based on 21 evaluable patients (one patient of the 22 enrolled died from an unrelated event).
The primary endpoint of the study is frequency of treatment emergent adverse events. Secondary study endpoints include changes in the Diabetic Retinopathy Severity Scale (DRSS), changes in best corrected visual acuity, changes in central subfield thickness compared to baseline, and the portion of patients receiving rescue therapy.
According to Ocular Therapeutix, Axpaxli (axitinib intravitreal implant, also known as OTX-TKI) is an investigational bioresorbable, hydrogel implant incorporating axitinib. Axitinib is a small molecule multitarget, tyrosine kinase inhibitor with anti-angiogenic properties. It is being evaluated for the treatment of wet age-related macular degeneration (AMD), diabetic retinopathy (DR), and other retinal diseases. Axpaxli is based on the company’s Elutyx bioresorbable hydrogel-based formulation technology.
The company advised it plans to present the study results at an upcoming meeting.
As summarized in the company’s press release, the topline phase 1 HELIOS results include the following:
- Safety. Axpaxli was generally well tolerated with no inflammation observed including no incidence of iritis, vitritis, or vasculitis.
- Efficacy. Six of 13 (46.2%) patients in the Axpaxli group experienced a one- or two-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at 40 weeks, with two of the six having a two-step improvement. Additionally:
- No patients in the control group showed one- or two-step improvement at the same time point.
- No patients in the Axpaxli group experienced any worsening in DRSS.
- One of eight patients (12.5%) in the control group experienced worsening in the DRSS at 40 weeks.
- Durability of Effect. A single injection of Axpaxli provided durable DRSS improvement up to 40 weeks.
- Rescue Medication. No patients in either arm received rescue medication.
- Next Steps. The positive results from HELIOS support decision to move directly to a phase 3 study with Axpaxli in patients with NPDR, pending discussions with the FDA.
- Data Release. Ocular plans to present topline results from the HELIOS study at a future meeting.
Ocular Therapeutix noted that Axpaxli is currently in the first of two planned pivotal phase 3 trials for wet AMD, the SOL-1 trial, and a phase 1 clinical trial for the treatment of NPDR.
“It is promising that the efficacy signals seen to date in this phase 1 study consistently favor Axpaxli,” commented Dilsher Dhoot, MD, in the Ocular Therapeutix press release. “These data demonstrated DRSS improvements and durability for up to 40 weeks. In addition, these results, combined with the results in the AMD studies of Axpaxli, make me even more enthusiastic to be a principal investigator in the SOL-1 study.” Dr. Dhoot is from California Retina Consultants in Santa Barbara, California.
Jeffrey S. Heier, MD, Chief Scientific Officer of Ocular Therapeutix, added, “We are pleased to announce positive topline data for Axpaxli in patients with diabetic retinopathy. In an effort to get Axpaxli to patients as quickly as possible, we unmasked this study, and based on the observed safety, efficacy results and durable treatment effect, we have decided to accelerate the DR program to phase 3. We look forward to providing additional information on our timeline for next steps, including our plans to present these results at an upcoming meeting in the coming months.”