Neuren Pharmaceuticals (ASX: NEU) Extends Trofinetide US Patent Protection to 2041

Neuren Pharmaceuticals’ US patent protection for trofinetide is expected to run until March 28, 2041 after the US Patent and Trademark Office allowed an application covering the drug’s weight-banded dosing regimen.

The new patent would extend protection by almost five years compared with an existing method-of-use patent, which expires in July 2036. The 2041 date includes six months of paediatric exclusivity.

For Neuren, the longer protection period extends the expected US intellectual-property coverage supporting partner Acadia Pharmaceuticals’ Rett syndrome franchise. Neuren has granted Acadia an exclusive worldwide licence to develop and commercialise trofinetide.

The regulator’s Notice of Allowance means the application has met the requirements for patentability. The patent has not yet formally issued, with that step expected after standard administrative processes are completed.

Protection covers commercial dosing regimen

The application covers the weight-banded dosing of trofinetide for treating Rett syndrome. That regimen is used for the drug sold in the US as DAYBUE oral solution and DAYBUE STIX powder for adults and children aged two years and older.

The allowed application is titled “Methods and Compositions for Treatment of Rett Syndrome.” Its expected expiry in March 2041 would provide Neuren and Acadia with a longer period of US patent coverage for the dosing approach used in the marketed products.

DAYBUE oral solution is approved by the US Food and Drug Administration, Health Canada and Israel’s Ministry of Health. The US regulator has also approved DAYBUE STIX powder for Rett syndrome.

The announcement did not provide financial forecasts or changes to the commercial terms of Neuren’s licensing agreement with Acadia. Its immediate relevance is the addition of almost five years to the expected US patent protection period for trofinetide, subject to formal issuance of the patent.

Neuren’s clinical pipeline

Beyond trofinetide, Neuren is developing NNZ-2591, also known as ercanetide, as an oral treatment for several neurodevelopmental disorders. Its programs include Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome, as well as hypoxic ischemic encephalopathy, a serious condition caused by brain injury before or shortly after birth.

Neuren is conducting the Phase 3 Koala trial of NNZ-2591 in children aged three to 12 years with Phelan-McDermid syndrome. The randomised, double-blind and placebo-controlled study is assessing safety and efficacy and is accompanied by a 52-week open-label extension study.

The NNZ-2591 programs for Phelan-McDermid, Pitt Hopkins and Angelman syndromes have received orphan-drug designation in the US and European Union, along with Fast Track and Rare Pediatric Disease designations from the US regulator.

Neuren’s broader development strategy targets the role of insulin-like growth factor 1 in the brain, using orally administered analogues of naturally occurring peptides. The company is focused on serious neurological disorders caused by genetic abnormalities or brain injury where approved treatment options are limited or unavailable.