Phase II study met primary endpoint, demonstrating promising visual acuity gains in patients with neovascular (wet) age-related macular degeneration



Data shows the potential for less frequent dosing and reduced treatment burden with RTH258 compared to aflibercept



Phase III clinical trial program initiated in December 2014, with 1700 patients expected to be enrolled in more than 50 countries



Basel, February 27, 2015 - Alcon, the global leader in eye care, presented positive results from its second Phase II clinical study of RTH258 during the 38th Annual Macula Society Meeting in Scottsdale, Arizona. This study evaluated the efficacy and safety of the compound versus aflibercept in patients with neovascular (wet) age-related macular degeneration (AMD). RTH258 (formerly known as ESBA1008) is a novel, single-chain antibody fragment developed to treat wet AMD.

The Phase II study met its primary endpoint, demonstrating promising visual acuity gains that were non-inferior to aflibercept, with numerically greater reduction and rapid improvement in abnormal retinal fluid observed in RTH258-treated patients. Patients treated every three months with RTH258 also experienced a prolonged duration-of-action, potentially leading to a reduced treatment burden. A total of 90 patients diagnosed with wet AMD participated in the prospective, randomized, double-masked multicenter, two-arm study. The primary objective was to compare the efficacy of RTH258 6mg versus aflibercept 2mg with the primary endpoint being the mean change in best corrected visual acuity (BCVA), from Baseline to Week 12. Secondary endpoints included the change assessment in BCVA and central subfield foveal thickness (CSFT) as measured by spectral domain optical coherence tomography (SD-OCT). Both RTH258 and aflibercept were well tolerated and no new safety signal was reported during the study.

"Alcon is deeply committed to addressing unmet patient needs in retina, and RTH258 demonstrates the potential to improve visual outcomes and reduce the treatment burden for patients with wet AMD," said Sabri Markabi, Senior Vice President, Research & Development for Alcon. "We have initiated our Phase III study program based on the encouraging results we received from the Phase II studies with RTH258."

RTH258 Phase III Study Program Initiated
With these positive Phase II results, Alcon has initiated its Phase III clinical study program to evaluate the efficacy and safety of RTH258 versus aflibercept in patients with wet AMD. As part this innovative study program, Alcon expects to enroll approximately 1,700 patients in more than 50 countries worldwide. The primary objective of the first Phase III study is to compare the efficacy of RTH258 3mg and 6mg versus aflibercept 2mg, with the mean change in BCVA, from Baseline to Week 48 as the primary endpoint. The second study within the Phase III trial program will also compare the efficacy of RTH258 versus aflibercept, and is expected to commence in 2015. Patients participating in the Phase III studies will be dosed every three months with RTH258, while a bi-monthly-dosing regimen will be followed for those patients considered unsuitable for a quarterly dosing schedule due to disease activity.
Aflibercept will be dosed according to its approved label. Please visit www.clinicaltrials.gov for further details about the RTH258 Phase III clinical trial program.

AMD is a leading cause of severe vision loss in people over age 50,[1],[2] and the United Nations estimates that it impacts 25 to 30 million people worldwide.[3] AMD occurs when the center of the retina, known as the macula, deteriorates leading to gradual loss in central vision.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by words such as "potential," "expected," "promising," "potentially," "committed," "encouraging," "to evaluate," "expects," "will," or similar terms, or by express or implied discussions regarding potential marketing approvals for RTH258, or regarding potential future revenues from RTH258. You should not place undue
...
References
[1] National Eye Institute. Facts About Age-Related Macular Degeneration. http://www.nei.nih.gov/health/maculardegen/armd_facts.asp.
[2] Klein R, et al "Incidence of visual impairment over a 20-year period" Ophthalmology 2013; DOI: 10.1016/j.ophtha.2013.11.041. http://bmctoday.net/practicaldermatology/dermwire/view.asp?20130405-amd_remains_leading_cause_of_vision_loss.
[3] BMJ, 2003 March 1; 326(7387): 485-488. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1125371/