Neuren Pharmaceuticals Annual Report 2022

Currently there are approximately 4,500 patients diagnosed with Rett syndrome in the US. Based on published prevalence studies, Neuren estimates that the total number of potential patients in the US may be up to 10,000. Acadia has projected that DAYBUE access will be covered by Medicaid for 60% of patients and by commercial health insurance for 30% of patients. It is anticipated that there will be nominal or zero out-of-pocket expense for families. The list price of DAYBUE will be $21.10 per mL and Acadia expects the average annual net realised cost per patient will be approximately US$375,000. This includes assumptions for the average weight of the expected patient population, compliance rates to therapy and mandatory government discounts. Development and commercialisation outside North America Acadia has exclusive rights to trofinetide in all indications for the United States, Canada and Mexico. Neuren retained all rights to trofinetide for countries outside North America and has a fully paid-up, irrevocable licence for use in those countries to all data generated by Acadia. There is urgent unmet need for a treatment for Rett syndrome around the world, evidenced by communications received from families, patient support groups and physicians. The estimated number of potential patients and currently identified patients are shown in the table below. Neuren expects rates of diagnosis to increase with greater awareness and accelerate with the availability of a treatment. Rett Syndrome Patients Europe Japan Israel China urban Other Asia Potential patients1 13,000 3,000 300 28,000 6,000 Patients currently identified 4,000 1,000 200 2,000 ‘00s 1 Potential patient estimates derived by applying the mid-point of the published prevalence estimate range to the populations under 60 years Neuren intends to pursue registration and commercialisation of trofinetide through partners and is currently advancing discussions with a number of third parties. About Rett syndrome Rett syndrome is a seriously debilitating and life-threatening neurological disorder. It is first recognized in infancy and seen predominantly in girls, but can occur very rarely in boys. At diagnosis, Rett syndrome has often been misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Most cases of Rett syndrome are caused by mutations on the X chromosome on a gene called MECP2. Rett syndrome strikes all racial and ethnic groups and has been estimated to occur worldwide in 1 of every 10,000 to 15,000 female births, causing problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. These problems can include learning, speech, sensory sensations, mood, movement, breathing, cardiac function, and even chewing, swallowing, and digestion. Rett syndrome symptoms appear after an early period of apparently normal or near normal development until six to eighteen months of life, when there is a slowing down or stagnation of skills. A period of regression then follows, with loss of communication skills and purposeful hand use, loss or impairment of walking, and the onset of stereotypic hand movements. Other problems frequently include seizures and erratic breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances. OPERAT I NG REV I EW CON T I NU E D i Neuren Pharmaceuticals Limited Annual Repor t 2022 7

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