ACI SANT'ANTONIO, Italy, May 31, 2024 -- SIFI announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of AKANTIOR^® (polihexanide under the international non-proprietary name), for the treatment of acanthamoeba keratitis, a rare ophthalmic disease.

"We are very pleased to have received this positive opinion from the CHMP for AKANTIOR^®. If approved, this innovative therapeutic solution will help patients suffering from acanthamoeba keratitis, a condition that is very debilitating in daily life and could lead to corneal transplant or enucleation of the eye, if untreated. We have been innovating in ophthalmology for almost 90 years and AKANTIOR^® represents a significant milestone as well as a proof of our commitment to address unmet medical needs in our field. We are also very encouraged by the efficacy and safety of AKANTIOR^® being replicated in several early access programs, which enrolled over 180 patients during the last 17 months in 12 countries in Europe and beyond." said Fabrizio Chines, Chairman and CEO of SIFI.

The CHMP recommendation will now be reviewed by the European Commission (EC), which may decide on the marketing authorisation (MA) for AKANTIOR^® in August 2024. The decision will then be applicable to all member states of the European Union as well as to Iceland, Liechtenstein and Norway.

The CHMP positive opinion is based on positive data from the comprehensive development program including a Phase 1 and the largest Phase 3 trial in AK to date. The Phase 3 pivotal was a randomized, multicenter and double-blind study on 135 adults and adolescents with acanthamoeba keratitis (ClinicalTrials.gov identifier: NCT03274895). Study 043/SI assessed the efficacy, tolerability and safety at 12 months of polihexanide (at a concentration of 0.8 mg/ml (0.08%)) compared to an active control. Clinical resolution rate achieved was 86.7% with a median time to cure of 4 around months, with dramatic improvements in patients' vision and quality of life. The Phase I randomized, double-masked, controlled trial conducted on 90 healthy volunteers (ClinicalTrials.gov identifier: NCT02506257), was designed to study the effect of different concentrations of polihexanide or placebo. Results from this study confirm that polihexanide 0.08% had no statistically significant or clinically meaningful differences in adverse events between each dose tested and placebo.

"Following 15 years of research, culminating in a pivotal Phase 3 clinical trial, polihexanide 0.08% was developed as the first licensed treatment for AK. Our studies have shown that when used as monotherapy, together with the treatment delivery protocol established for use in the trial, medical cure rates of over 86% can be achieved. I expect this to become the standard of care for this severe disease." stated Professor John Dart (Moorfields Eye Hospital and the University College of London, Institute of Ophthalmology), principal investigator of the Phase 3 trial.

AKANTIOR^® is produced with a polihexanide polymer chain of an optimal length that provides a molecular weight shown to be highly efficacious as a monotherapy. SIFI has filed an Active Substance Master File for its proprietary Good Manufacturing Practices quality assured polihexanide, in connection with the marketing authorization application for AKANTIOR^®.

After the expected approval by the competent authorities, AKANTIOR® will be commercialized directly by SIFI in major European markets such as France, Germany, Italy, Romania, Spain, as well as United Kingdom and Turkey addressing a total population of 430 million, and by our commercial partner Avanzanite in other European Countries addressing an additional population of 180 million. Based on scientific publications, the incidence of acanthamoeba keratitis is estimated at 1 to 4 patients per million inhabitants.

ABOUT SIFI: SIFI is a leading international ophthalmic company, headquartered in Italy, featuring an integrated business model, from research & development to manufacturing and commercialization both in pharmaceutical and biomedical sectors. Since our foundation in 1935, our mission is to improve people's lives through meaningful innovation in eye care. SIFI exports to more than 40 countries worldwide with a direct presence in major European markets, Mexico and, through joint ventures, in China and the United Arab Emirates. More information available at sifigroup.com.

ABOUT AKANTIOR^®: AKANTIOR^® (polihexanide 0.08%) stands to become the first approved drug for the treatment of acanthamoeba keratitis in the world. It is an anti-amoebic polymer that acts on both the trophozoites and cysts of the protozoan acanthamoeba. It is formulated at a 0.8mg/ml (0.08%) concentration which makes it possible to administer as monotherapy eye drops in single-dose containers. In contrast, current treatment protocols include various non-standardized combination therapies involving unlicensed – compounded or imported – alternatives. The EMA and the US Food and Drug Administration granted SIFI Orphan Drug Designation in the treatment of Acanthamoeba keratitis.

ABOUT Acanthamoeba Keratitis (AK): AK is an ultra-rare, acute, severe parasitic corneal infection caused by acanthamoeba, a free-living amoeba, although incidence of AK has been rapidly growing in recent years. AK is an ocular emergency and requires urgent treatment to save the eye. It can lead to poor vision, potential blindness, or even eye loss and often requires single or multiple corneal transplants. It affects people of all ages, most of whom are young or middle-aged soft contact lens wearers. Patients report unbearable pain and extreme light sensitivity and can rarely work or lead normal lives until symptoms resolve.

Key Contact AKANTIOR^®:

Jelle Kleijn

Global Head of AKANTIOR^®

+31 615643708

jelle.kleijn@sifigroup.com