A biopharmaceutical company headquartered in Australia, Neuren Pharmaceuticals Limited (ASX: NEU) is engaged in the development of new therapies for neurodevelopmental disorders with a high unmet need, characterised by impaired connections and signalling between brain cells.
Trofinetide (the leading drug candidate of Neuren, also known as NNZ-2566) is licensed to ACADIA Pharmaceuticals by Neuren for its development and commercialization in North America while Neuren retains all rights of the product outside North America. The company is also advancing the development of its second drug candidate NNZ-2591 which has shown positive results in models (prec-clinical) of several disorders, including Phelan-McDermid syndrome, traumatic brain injury, Fragile X syndrome, Parkinson’s disease and many more.
As indicated in the above product pipeline chart, Trofinetide is currently in Phase 2 development for Fragile X syndrome (completed the clinical trial) while it has completed Phase 2 development for Rett syndrome. The Phase 3 trial of Trofinetide for Rett syndrome is expected to commence in H2 2019 by ACADIA Pharmaceuticals.
Trofinetide has been granted orphan drug designation by the US Food and Drug Administration (US FDA) and European Medicines Agency in Fragile X syndrome and Rett syndrome. It has also received ‘Fast Track designation’ in Rett syndrome, Fragile X syndrome and moderate to severe traumatic brain injury (TBI) by the US FDA.
In a recent development, Neuren’s second drug candidate NNZ-2591 has demonstrated positive effects in pre-clinical models of two additional neurodevelopmental disorders including Angelman syndrome (AS) and Pitt Hopkins syndrome (PTHS), both currently with unavailability of specific treatment. In February 2019, NNZ-2591 also announced positive results in the Shank3 model of Phelan-McDermid syndrome (PMS).Neuren believes that Angelman syndrome (AS) as well as Pitt Hopkins syndrome (PTHS) meet the criteria for Orphan Drug designation.
The drug candidates of Neuren are protected by issued patents. For trofinetide, the company owns issued composition of matter patents in the US and Europe. These patents expiring in 2022 have the potential to be extended to 2027. Neuren also owns issued composition of matter patents for its other drug candidate, NNZ-2591 in the US, Europe and Japan. They will expire in 2024 and have the potential to be extended to 2029.
Neuren entered into a partnership with ACADIA Pharmaceuticals in August 2018 through which it secured significant funding and additional capabilities required to bring trofinetide to the United States market. The company received funds of $13.5 million as the first payment, which enabled it to advance NNZ-2591 as quickly as possible to clinical trials. The company has also received commitment from ACADIA of ~A$77 million investment in Rett Syndrome Phase 3.
For NNZ-2591, the company aims to file an Investigational New Drug application with the FDA and initiate the clinical trials in 2020.
According to the recently announced Annual Report 2018, Neuren ended 2018 in a much stronger financial position following the ACADIA partnership. For the year ended 31st December 2018, the company had cash reserves of $23.6 million in comparison to the $4.7 million cash reserves in the prior year. The cash inflow from operations and financing amounted to $6.4 million and $11.7 million, respectively during the period. The company sustained its annual profit (after tax) at ~$3 million.
The company’s stock has generated a return of 4.80 per cent in the last one month and 16.50 per cent in the last five days. It is currently trading at AUD 1.200 (As at 2:51 PM AEST, 23rd May 2019).
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