New treatment for Netherton syndrome
|Coordinator||SIXERA PHARMA AB|
|Funding from Vinnova||SEK 500 000|
|Project duration||May 2016 - July 2017|
Purpose and goal
The funding enabled Sixera to advance the development program on novel treatment of Netherton syndrome in relation to chemistry, manufacturing and control (CMC) and nonclinical development. The project resulted in intense collaborations between Sixera and industrial partners (RISE and Syntagon for product manufacturing; CiTox Laboratories for GLP-regulated nonclinical safety studies). Close interactions with a network of key consultants and clinical KOLs was established. New investments by original owners were made and dialogues with new potential investors are ongoing.
Expected results and effects
The main project achievements: - Scientific advise with Swedish Medicinal Product Agency to ensure the regulatory compliance of the planned nonclinical toxicology studies and clinical trials with EMA requirements for dermal products (21st Sept. 2016; MPA Ref 4.2.3-2016-049443) -Manufacturing of the Active Pharmaceutical Ingredient (API; SXR1096) for GLP-toxicology studies and finalization of the plan for GMP production of clinical trial material -The GLP-regulated systemic toxicity study in rats to characterize the safety of SXR1096 and to provide a basis for pivotal studies
Planned approach and implementation
Overall, the project activities, time plan, and budget have followed the original project plan. However, based on dialogue with Swedish MPA, the scope of the pivotal toxicology studies was expanded. In addition, the cost for API manufacturing was significantly higher than anticipated. Therefore, only the first GLP-regulated systemic toxicology studies could be performed in the frames of this project while additional studies need to be conducted to provide necessary nonclinical safety documentation prior clinical trials in humans can commence.