We search for disease modifier medicines in CNS indications

Accure Therapeutics focuses on neurons that suffer a dysfunction, and on the glial environment that supports them, in an attempt to impede their degeneration and death. We work to find new medicines that correct the pathological changes that are responsible for the disease, and that produce beneficial outcomes (i.e. Disease Modifying Therapies). We specialize in developing drugs that promote neuroprotection and neurorepair by integrating the latest advances in neurobiology with state-of-the-art clinical research.

We support a targeted pipeline

We have currently built up a drug candidate portfolio that focuses on four areas: Optic Neuritis, Multiple Sclerosis, Parkinson’s Disease and Epilepsy.

Optic neuritis and glaucoma

Optic neuritis occurs when swelling (inflammation) damages the optic nerve that transmits visual information from your eye to your brain. Symptoms include pain on eye movement and vision loss in one eye. Optic neuritis can occur during the first relapse of multiple sclerosis (MS).

Glaucoma occurs when abnormally high fluid pressure in the eye, most commonly caused either by blockage of the channel through which aqueous humor drains or by pressure of the iris against the lens, traps the aqueous humor. That eye disease impairs the vision and may slowly cause eye damage and total loss of vision (blindness).

Parkinson’s disease

Parkinson’s disease occurs when death of cells in a region of the midbrain is associated with deficiency of the neurotransmitter dopamine. It mainly affects the motor system leading to the progressive emergence of tremor, muscular rigidity, and slow, imprecise movement, chiefly affecting middle-aged and elderly people. Upon progression of the disease can manifest cognitive impairments such as executive function deficits, attention difficulties, slowed thinking, difficulties in word-finding, and difficulties in learning and remembering information thus causing significant disability to patients and burdens for caregivers.

Multiple sclerosis

Multiple sclerosis (MS) occurs when an abnormal response of the body’s immune system causes inflammation that damages myelin, a sheath that surrounds and insulates the nerve fibers, as well as the nerve fibers themselves, thus altering or stopping messages within the CNS. Multiple sclerosis progression varies from person to person and can lead to severe disability, impairing the daily activities and requiring assistance with walking.


Epilepsy occurs when abnormal electrical activity in the brain lead to recurrent seizures which are sudden episodes of sensory disturbance, involuntary movements, loss of consciousness, or convulsions. These episodes can result in physical injuries and impact on the quality of life for people with the disease and their families.

Innovative Programmes

This portfolio can serve as the basis for further expansion towards future R&D programmes and targeted innovations


ACT-01, is a potentially disease-modifying first-in-class small molecule with neuroprotective activity that has shown positive results in animal models of neuroinflammation and neurodegeneration. Its mechanism of action is related to the activation of the trophic factor pathways such as IGF-1 and BDNF to promote cell survival, and endogenous and natural repair of damaged neurons. In ophthalmology, this action can protect the nerve axons in conditions where the optic nerve is affected, such as in acute optic neuritis and glaucoma, where ACT-01 could prevent chronic vision loss. Beyond optic neuropathies, ACT-01 has the potential to address a wider range of neurodegenerative diseases such a multiple sclerosis.

Based on positive preclinical data and results of a phase I safety and PK study in healthy volunteers, a phase 2a study (the ACUITY study) was initiated and is currently enrolling patients.

ACUITY is a two-arm, randomized, double-blind, placebo-controlled, monocentric study to evaluate the safety and tolerability of ACT-01 compared to placebo in patients with acute optic neuritis. In addition to safety, secondary outcome measures will include optic nerve anatomical measures, as well as visual function measures. The study is ongoing at the La Pitié-Salpêtrière hospital in Paris within the neurology-ophthalmology network of the Public University Hospital Group in Paris (APHP).

Accure has granted Oculis an exclusive global licensing agreement to develop and commercialize ACT-01.

“ACT-01 originated at IDIBAPS/CSIC.”


Is an orally active, brain penetrant and selective inhibitor of Prolyl oligopeptidase (also known as Prolyl Endopeptidase). These peptidases are involved in the catabolism of neurotransmitters and the modulation of certain protein-protein interactions that can affect cognitive functions and neurodegeneration. The drug candidate has demonstrated properties to improve cognition in preclinical models of brain disorders. ACT-02 is Accure’s lead program for Parkinson’s disease and further indications will also be explored.


Is an injectable, brain penetrant small molecule and selective inhibitor of matrix metalloproteinases that modulates neuroplasticity and neuroinflammation at the extracellular matrix level. The drug candidate demonstrated properties to reduce the number, the intensity and the duration of seizures in preclinical models of epilepsy. ACT-03 is Accure’s lead program for certain forms of epilepsy of inflammatory origin and further indications will also be explored.

R&D Portfolio and Pipeline

R&D Portfolio and Pipeline