Feb 27, 2015
  • 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.

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 reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that RTH258 will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that RTH258 will be commercially successful in the future. In particular, management's expectations regarding RTH258 could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and over-the-counter products. Novartis is the only global company with leading positions in these areas. In 2014, the Group achieved net sales of USD 58 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 130,000 full-time-equivalent associates. Novartis products are available in more than 180 countries around the world. For more information, please visit http://www.novartis.com.

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*Aflibercept is marketed under the brand name of Eylea® in the United States by Regeneron and outside the United States by Bayer, and is a prescription medicine for the treatment of patients with wet AMD, macular edema following retinal vein occlusion (RVO), and diabetic macular edema (DME).

[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/

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