Barcelona, Spain, March 1, 2021 – Accure Therapeutics, a private translational R&D engine at clinical stage in the Central Nervous System (CNS) field, today announces it has enrolled the first patient in its ACUITY phase II clinical trial – in patients with acute optic neuritis (AON). A total of 36 patients will be enroled in two randomized parallel groups. They will be followed for six months to measure and assess safety and preliminary signs of efficacy. Results are expected in the second half of 2022.
The ACUITY study is carried out within the neurology-opthalmology network of the Public University Hospital Group in Paris (APHP), with the leading site at La Pitié-Salpétrière hospital. The study is under the supervision of lead investigator Dr. Céline Louapre, from the Paris Brain Institute (Institut du Cerveau et de la Moelle épinière, ICM), and scientific advisor Dr. Sophie Bonnin, from the Department of Ophthalmology at La Pitié-Salpétrière. ICM, located at La Pitié-Salpetrière, brings patients, doctors and researchers together, with the aim of rapidly developing treatments for disorders of the nervous system and enabling patients to benefit from them as quickly as possible.
“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.”
A total of 36 eligible subjects will be randomized within ten days of the onset of AON (visual loss symptoms) to receive five intravenous infusions of ACT-01 (N=18) or placebo (N=18), over a five-day period. As is the standard of care for AON, all patients will receive intravenous corticosteroid as concomitant therapy – unless contra-indicated. Patients will be assessed by a neurologist and an ophthalmologist; clinically and with non-invasive routine imaging tests.
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.”
Join our webinar on Tuesday March 2, at 7:30pm CET/1:30pm ET/10:30am PST
and listen to expert speakers Prof. Shiv Saidha, Johns Hopkins University (Baltimore, US) and Prof. Fiona Costello, University of Calgary (Canada), as they discuss Acute Optic Neuritis. They will be covering the following:
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About acute optic neuritis (AON)
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.
There is wide variability in symptom severity and recovery, without predictors for poor recovery with residual dysfunction. It is reported that severity and recovery from the initial relapse are associated with severity and recovery for subsequent recurrences.
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.