ONGOING CLINICAL TRIALS

About ALK-001

Stargardt disease (STGD1) and dry age-related macular degeneration are currently untreatable disorders and leading causes of blindness affecting millions of people (dry AMD) and some 40,000 people (STGD) in the USA. In its early stage, symptoms include loss of central vision that is uncorrectable with glasses. The condition then progresses to legal blindness (20/200) or worse in the majority of the cases. In the case of STGD, most patients experience their first symptoms between the ages of 6 to 30 years of age.

ALK-001 is once-a-day oral drug candidate currently being tested for the treatment of geographic atrophy (advanced form of AMD) and Stargardt disease. Toxic vitamin A aggregates called "dimers" are known to rapidly form due to a genetic defect in Stargardt, or to accumulate with age in the case of dry AMD. These toxic dimers have been implicated in the degeneration of the retina seen in Stargardt and AMD. Dimers form when two molecules of vitamin A are chemically combined. ALK-001 is form of vitamin A that has been chemically-modified to prevent its aggregation into dimers.

In a completed Phase 1 clinical trial, we have assessed the safety and pharmacokinetics of ALK-001 in healthy volunteers. (Click here to read details about the Phase 1).

Stargardt Disease: ongoing Phase 2 Clinical Trial Enrolling Participants 8 years and older in the USA

We are currently enrolling patients who are 8 years of age and older and who have been diagnosed with Stargardt disease, to participate in a midstage (Phase 2) clinical trial taking place at multiple clinical sites in the United States. If you are interested or know someone who could be interested in participating, please email us at trials@alkeuspharma.com or register your name on our Stargardt registry mailing list.

For the most up to date information about this study, click on the following link:

http://www.clinicaltrials.gov/ct2/show/NCT02402660

Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD): ongoing Phase 3 Enrolling Participants in the USA

Dry-AMD is the leading cause of blindness for people over 50. There is no treatment or cure for dry-AMD. The late form of dry-AMD, also called Geographic Atrophy (GA) affects over 1 million people in the USA alone. Patients with GA have seriously impaired vision, with decreased visual acuity that is uncorrectable with glasses or lenses. GA progresses with time leading to legal blindness. Peripheral vision is usually preserved.

We are currently enrolling patients who have been diagnosed with Geographic Atrophy secondary to age-related macular degeneration, to participate in a Phase 3 clinical trial taking place at multiple clinical sites in the United States. If you are interested or know someone who could be interested in participating, please email us at trials@alkeuspharma.com

For the most up to date information about this study, click on the following link:

http://clinicaltrials.gov/ct2/show/NCT03845582

FUTURE CLINICAL TRIALS

If you are interested in learning more about future clinical trials, please send us an email at info@alkeuspharma.com. Alkeus Pharma intends to run clinical trials in serious diseases such as the following:

Intermediate dry-AMD

The intermediate form of dry-AMD affects approximately 8 million patients in the US alone. The severity of the disease is measured by a retinal specialist who attributes a disease grade (from 1 to 4).

If grade 1 and grade 2 have a relatively little chance of progression to late-AMD (dry or wet forms), grade 3 and grade 4 have respectivaly about 25% and 50% chance of progression to late-AMD after 5 years. These two groups of patients are at high risk of complications resulting of late-AMD.

Autosomal Recessive Retinitis Pigmentosa (RP)

Retinitis pigmentosa (RP) forms a group of rare diseases that lead to blindness preceded by night blindness and tunnel vision for several years or decades. The disease affects approximately 100,000 people in the US.
Although multiple genes when mutated can cause Retinitis Pigmentosa, about 5% of the patients with RP have mutated ABCA4 genes, which leads to improper processing of vitamin A by the eye. These patients could potentially benefit from Alkeus Pharma's technology.

Autosomal Recessive Cone-Rod Dystrophy (CORD3)

Cone-Rod dystrophy is a rare disease which affects the cones more seriously than the rods. The disease is usually diagnosed by evaluating the amplitudes of the retina's response to light signals and confirmed genetically. Multiple genes can cause Cone-Rod dystrophies. When the ABCA4 is defective, the disease is thought to be caused by the accelerated formation of toxic vitamin A aggregates in the eye. Because ALK-001 prevents the formation of toxic aggregates in the eye, it could possibly benefit patients with Cone-Rod dystrophy.