Accure Therapeutics, a private translational R&D engine at clinical stage in the Central Nervous System (CNS) field, has announced the enrollment of the first patient in ACUITY phase II clinical trial on acute optic neuritis with its lead candidate ACT-01. The biotechnological company, based in the Barcelona Science Park and boosted by the Accelerator Programme of GAEM Foundation, expects results in the second half of 2022.

The ACUITY study is carried out within the neurology-opthalmology network of the Assistance Publique-Hôpitaux de Paris (AP-HP), with the leading site at Hôpital Pitie-Salpêtrière. The trial is under the supervision of lead investigator Dr. Céline Louapre, from the Institut du Cerveau et de la Moelle épinière (Institut du Cerveau – ICM), and scientific advisor Dr. Sophie Bonnin, from the Pitié-Salpétrière.

“This study is set to confirm the safety and tolerability of Accure’s candidate and to provide preliminary proof-of-efficacy – both as clinical and imaging evidenced by optical coherence tomography (OCT) – of ACT-01 in patients living with inflammatory demyelinating diseases such as acute optic neuritis,” said Dr. Celine Louapre. “These first efficacy results in patients will give us an estimate of the treatment effect and help us calculate the adequate sample size for the next clinical study.”

Preliminary efficacy of ACT-01 will be assessed over a six month period – post onset of AON symptoms. By this time, almost all subjects are expected to have come to the end of  their natural recovery; therefore this strategic timeline is used to compare the profiles of disease progression, and structural and functional outcomes between subjects in each treatment arm. For the majority of patients, thinning of the retinal layers in the affected eye occurs within the first six months of the onset of AON, as measured by the RNFL- retinal nerve fiber layer and the GCIPL – ganglion cell-inner plexiform layer, in an OCT scan.

“We are very excited to initiate this phase II study with our lead candidate ACT-01 in patients with acute inflammatory-demyelinating disorders,” said Dr. Rossella Medori, CMO at Accure Therapeutics. “The results from the phase II study have the potential to serve as a solid foundation for expanding future pivotal clinical development of our first-in-class disease modifying drug candidate, to address the challenges of residual and accrued disability in other chronic neurodegenerative CNS disorders.”

ACT-01 is a peptidomimetic first-in-class disease-modifying treatment for multiple sclerosis (MS) and acute optic neuritis, the latter with orphan drug designation by the US FDA and the EMA in the EU. It crosses the blood-brain barrier by active transport, to promote neuronal cell survival and myelination.

In vivo and phase I studies showed the safety, tolerability and therapeutic potential for ACT-01 in the treatment of neurodegenerative diseases, such as AON and MS.

Acute optic neuritis (AON) is an acute inflammatory demyelinating disorder of the optic nerve. The affected patients present with unilateral, subacute and painful visual loss. AON is considered a rare disease; with an incidence of one to five cases per 100,000 people per year. While in up to 20% of subjects, AON can be the initial presenting symptom of multiple sclerosis (MS), in half the cases, AON occurs as an MS relapse during the course of the disease.

Acute corticosteroids are the usual standard of care, but currently, there is no approved therapy that preserves vision or the integrity of ganglion/retinal nerve fibres after an episode of AON. A neuroprotective drug is therefore needed; one that could prevent long-term axonal loss and hopefully lead to a better visual outcome.

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