Three Significant Trofinetide Announcements from Neuren Pharmaceuticals: An overview

November 24, 2015

Neuren Pharmaceuticals has made three significant announcements in the last 2 months regarding trofinetide, the drug candidate they are developing as a treatment for Rett syndrome.  We at would like to take an opportunity to summarize these recent activities that give us great hope for a rising treatment.  In the spirit of Thanksgiving, we give thanks to those who made these advancements possible:  Neuren Pharmaceuticals and their shareholders, scientists, clinicians, physicians, nurses, study coordinators, government regulatory officials, our donors and the trial participants, girls and women along with their families affected by Rett syndrome. 

October 12, 2015

“New US patent covering use of trofinetide for Rett syndrome”

The US Patent and Trademark Office issued a Notice of Allowance for a new patent concerning the use of trofinetide to treat Rett syndrome.  The patent is expected to expire in January 2032, which offers Neuren Pharmaceutical additional protection to their current patent.

Trofinetide has orphan drug designation in the US and in Europe, which gives Neuren market exclusivity following market authorization for 7 and 10 years, respectively.

Read full press release.

October 13, 2015

“IRSF confirms financial support for Neuren's clinical trials”

Neuren Pharmaceuticals announced the financial support of for continued development of trofinetide as a treatment for Rett syndrome.  The Phase 2 trial in adults with Rett syndrome showed success in clinical benefit, as observed in core symptoms of Rett syndrome as well as caregivers and caregiver assessments.

With the funding from, Neuren Pharmaceuticals will conduct a pediatric trial in children below the age of 16.  The study will determine the tolerability of higher doses and length of treatment in this age group, which will enable Neuren to determine the optimal dose to be used in the subsequent Phase 3 trial.

Neuren is working with the Food and Drug Administration (FDA) on the Phase 3 trial design that would support the potential approval of a New Drug Application.

Read full press release.

November 9, 2015

“Shareholders provide further support for Rett syndrome”

With the financial commitment of, the shareholders of Neuren agreed to provide an additional level of funding that will accelerate the development of trofinetide for Rett syndrome.

Neuren Pharmaceutical will issue ~70M new shares to sophisticated investors, and these shares will represent 4% of Neuren’s existing issued shared capital.  Proceeds will fund development of trofinetide for Rett syndrome through into 2017.

In 2016, key activities will include: the pediatric trial, pre-clinical safety studies to support a New Drug Application, and completion of the optimization and scale-up of the commercial manufacturing process.

Read full press release.