Chugai's Port Delivery System with Ranibizumab Shows Promise in Japanese Phase I/II Trial for Retinal Diseases
核心洞察
Chugai Pharmaceutical announced positive results from its Japanese phase I/II TEIEN study evaluating the Port Delivery System with Ranibizumab (搜索) (PDS) in patients with neovascular age-related macular degeneration (搜索) and diabetic macular edema (搜索).
The innovative intraocular implant demonstrated a mean change from baseline in best-corrected visual acuity of -1.4 letters at 24 weeks in nAMD patients, with a safety profile similar to previous overseas trials.
The PDS represents the first system of its kind in Japan, requiring drug refills only every 24 weeks compared to current treatments that need injections every 4 to 16 weeks.
Chugai Pharmaceutical Co., Ltd. announced positive results from its Japanese phase I/II TEIEN study evaluating the Port Delivery System with Ranibizumab (搜索) (PDS) in patients with neovascular age-related macular degeneration (搜索) (nAMD) and diabetic macular edema (搜索) (DME). The innovative intraocular implant demonstrated efficacy and safety profiles consistent with previous overseas clinical trials, marking a significant advancement in retinal disease treatment in Japan.
Clinical Trial Results
The TEIEN study enrolled 21 patients across two cohorts: 15 patients with nAMD and 6 patients with DME. In the nAMD patient population, the mean change from baseline in best-corrected visual acuity (BCVA) score at 24 weeks was -1.4 letters (95% CI: -5.7, 2.9). The safety profile was similar to previous overseas clinical trials in both the nAMD and DME patient populations.
The study's primary endpoints focused on safety and tolerability in both patient populations, along with the mean change from baseline in BCVA score at 24 weeks specifically in the nAMD cohort.
Revolutionary Treatment Approach
The Port Delivery System represents the first innovative intraocular implant of its kind in Japan for treating nAMD and DME. The system comprises an ocular implant surgically placed into the eye during a one-time procedure, along with four ancillary devices for initial filling, insertion, refilling, and removal if needed.
"The current standard of care for nAMD and DME requires intraocular injections at intervals of 4 to 16 weeks, and a less burdensome treatment for patients is desired," said Dr. Osamu Okuda, Chugai's President and CEO. "The PDS is expected to maintain long-term efficacy and reduce the burden of treatment by using an innovative intraocular implant that is the first of its kind in Japan and only requires refills with the drug once every 24 weeks."
Mechanism and Clinical Advantage
The PDS continually delivers a customized formulation of ranibizumab into the back of the eye. Ranibizumab is a VEGF (搜索) inhibitor designed to bind to and inhibit VEGF-A (搜索), a protein that plays a critical role in the formation of new blood vessels and vessel leakiness in patients with retinal vascular disorders, including nAMD and DME.
The system's key advantage lies in its extended dosing interval. While current intravitreal VEGF (搜索) therapies require injections as often as every 4 to 16 weeks, the PDS only requires drug refills every 24 weeks. This reduction in administration frequency is expected to lessen the treatment burden for patients, their families, and medical institutions.
Disease Burden and Market Impact
Neovascular AMD affects approximately 20 million people worldwide and represents the leading cause of vision loss in people over age 60. The condition develops when new and abnormal blood vessels grow uncontrolled under the macula, causing swelling, bleeding, and/or fibrosis. Without treatment, nAMD can cause rapid and severe vision loss.
DME affects around 29 million people globally and is a vision-threatening retinal condition associated with blindness and decreased quality of life when left untreated. The condition occurs when damaged blood vessels leak into and cause swelling in the macula. The number of people with DME is expected to grow as the prevalence of diabetes (搜索) increases.
Regulatory Path Forward
Chugai plans to file a new drug application in Japan based on the TEIEN study results combined with overseas phase III clinical trials conducted by Roche for patients with nAMD (Archway study) and DME (Pagoda study).
"This is a major advancement for patients with nAMD and DME, and represents a new treatment option that can also benefit physicians and patients' families," Dr. Okuda stated. "We are committed to working diligently toward filing for approval in Japan so that we can deliver this innovative value to patients as soon as possible."