Neuren Pharmaceuticals (ASX: NEU) announced progress in its development programs for trofinetide in Rett syndrome and Fragile X syndrome.
The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has recommended Orphan designation be granted for trofinetide in both Rett syndrome and Fragile X syndrome.
Neuren said it anticipates the European Commission will provide formal confirmation of these designations in August.
Orphan designation in the European Union (EU) grants a number of benefits to sponsors, including ten years of market exclusivity once the medicine is on the market.
Neuren’s programs for Rett syndrome and Fragile X syndrome have already been granted Orphan Drug designation by the FDA, which provides seven years of market exclusivity in the US.
Neuren said it recently met with the FDA to discuss the remaining development requirements for trofinetide in Rett syndrome.
“The meeting provided meaningful guidance for Neuren in all areas of the development program. Importantly, this included guidance on efficacy endpoints for pivotal clinical trials, the requirements for Phase 3 trials and the testing of trofinetide in subjects younger than age 16,” said the company.
“Neuren and the FDA committed to reach agreement quickly on the primary efficacy endpoint to be used in a pivotal trial for Rett syndrome, with Neuren to propose a subset of items from the cliniciancompleted Motor Behavior Assessment (MBA).”
Neuren used the MBA in its Phase 2 clinical trial and has been used to assess over 1,100 children, adolescents and adults with Rett syndrome enrolled in the Rett Natural History Study, a study sponsored by the National Institutes of Health.
It said that, while two Phase 3 clinical trials are generally required to support the approval of a New Drug Application in the US, for rare diseases it may be acceptable to design a single Phase 3 trial to collect sufficient evidence of safety and efficacy.
“It was agreed that Neuren will submit a design proposal to the FDA for a single Phase 3 trial,” according to the company. “Neuren received encouragement to conduct a brief paediatric tolerability clinical trial in which higher doses of trofinetide will be tested in subjects below the age of 16. Neuren’s Phase 2 trial tested adults and adolescents aged 16 years and older.”
Neuren said it is currently well funded, with cash reserves of $17.7 million at the end of June, and the board will consider all strategic options for progressing the Phase 3 development as quickly and efficiently as possible.
“To date trofinetide has demonstrated an excellent safety and tolerability profile, with evidence of a pattern of clinical benefit across the broad phenotype of Rett syndrome, which is consistent with its normalising effects on brain biology”, said Neuren’s Chief Science Officer, Larry Glass.
Neuren is also presently completing a Phase 2 clinical trial in the US, collecting information on the safety, efficacy and pharmacokinetics of trofinetide in adults and adolescents with Fragile X syndrome. Enrolment of subjects has now been completed, with 72 subjects participating. The last subject is due to complete the trial at the end of September 2015. Neuren expects to release top-line results in December 2015.
“Following a productive and collaborative meeting with the FDA, we now have a pathway towards a New Drug Application for Rett syndrome”, said the company’s Executive Chairman, Richard Treagus. “We are also very pleased to have secured the important commercial incentive of Orphan designation in the European Union for both indications and we are on track to provide the top-line results from our Phase 2 trial in Fragile X before the end of the year”.