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Ocular Therapeutix announces positive topline Phase 1 data for Axpaxli in diabetic retinopathy
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According to the company, 46.2% of patients demonstrated a 1- or 2-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at 40 weeks in the Axpaxli arm, compared to 0% in the control arm.
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Ocular Therapeutix Inc announced positive topline results from the Phase 1 HELIOS study (NCT0569541) evaluating its investigational bioresorbable, hydrogel implant (Axpaxli) versus a sham control in patients with moderately severe to severe non-proliferative diabetic retinopathy without diabetic macular edema (DME).
The company said in a news release it plans to present the study results at an upcoming meeting.
The investigational bioresorbable, hydrogel implant (Axpaxli) incorporates axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet AMD, DR and other retinal diseases.1
According to the company, the Phase 1 HELIOS study is a multi-center, double-masked, randomized 2:1, parallel group clinical trial conducted at 10 centers in the U.S. The data set reported here is based on 21 evaluable subjects (one patient of the 22 enrolled subjects died from an unrelated event).
The study was designed to evaluate the safety, tolerability and efficacy of hydrogen implant compared to a sham control in subjects with moderately severe to severe NPDR without diabetic macular edema (DME).
The primary endpoint of the study is frequency of treatment emergent adverse events (TEAEs). Secondary study endpoints include changes in the DRSS, changes in best corrected visual acuity (BCVA), changes in central subfield thickness compared to baseline, and the portion of subjects receiving rescue therapy.1
The company noted the results showed the investigational bioresorbable, hydrogel implant was generally well tolerated with no inflammation observed including no incidence of iritis, vitritis or vasculitis.
The study’s efficacy results showed 6 of 13 (46.2%) patients in the hydrogel implant group experienced a 1 or 2-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at 40 weeks, with 2 of the 6 having a 2-step improvement.
Moreover, the company noted that no patients in the control group showed 1- or 2-step improvement at the same time point. No patients in the hydrogen stent group experienced any worsening in DRSS. 1 of 8 (12.5%) in the control group experienced worsening in the DRSS at 40 weeks.1
The study also found that a single injection of Axpaxli provided durable DRSS improvement up to 40 weeks.
The company noted that there were no patients in either arm who received rescue medication.
The next step, according to the company, is to move directly to a Phase 3 study with Axpaxli in patients with NPDR, pending discussions with the FDA.
Ocular Therapeutix said in the news release it plans to present topline results from the HELIOS study at a future meeting
Jeffrey S. Heier, chief scientific officer of Ocular Therapeutix, said the company was 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,’ he said in the news release. “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.”
Dilsher Dhoot, MD, of California Retina Consultants, pointed out it is promising that the efficacy signals seen to date in this Phase 1 study consistently favor the bioresorbable, hydrogel implant.
“These data demonstrated DRSS improvements and durability for up to 40 weeks,” Dhoot said in the news release. “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.”
