NurOwn and the Therapy Development Process

24-01-2020

The ALS Liga Belgium and the wider ALS community are hopeful that several therapies currently in Phase 3 clinical trials will prove to be successful in slowing, halting, or reversing ALS. 

One of the therapies now in Phase 3 trials, NurOwn, has been the subject of considerable discussion on social media. Some of what is being stated on social media is unfortunately not accurate, and risks misleading people with ALS.

Here, we offer clarifications about NurOwn and the therapy development process. 

Phase 3 clinical trial with NurOwn ongoing in USA and Canada

NurOwn, a therapy developed by BrainStorm, showed promise in its Phase 2 trial and has now entered a Phase 3 clinical trial that is conducted in the USA and Canada. The study is fully enrolled, meaning that the maximum amount of 200 patients that meet the inclusion criteria are taken part. Finalizing of the phase 3 clinical trial is anticipated for the last quarter of 2020.

Access to a therapy is possible only after a successful Phase 3 Clinical trial and subsequent approval by the regulatory authority (FDA in the USA, EMA in the EU). In the USA, there are however two legal pathways for NurOwn to be made available to people with ALS while it is undergoing clinical trials — Expanded Access and Right to Try. In both cases, the company that makes the experimental drug has to grant permission and supply the treatment. BrainStorm has indicated on multiple occasions that it will not –with the exception of pALS Matt Bellina - provide NurOwn to individuals via Expanded Access or Right to Try. This is not uncommon, due to the costs and other factors that companies weigh when considering Expanded Access or Right to Try.

Therefore, until BrainStorm applies for a biologics license for NurOwn on the basis of positive phase 3 study results in the USA, there is nothing the FDA can do. BrainStorm has said it will not seek early approval of NurOwn and that it intends to complete its Phase 3 trial of NurOwn as scheduled.

Current situation of NurOwn in the EU, including Belgium

Currently, NurOwn is not approved for market authorization in the EU, including Belgium.

If BrainStorm intends to seek such market authorization for NurOwn in the EU, it has to address an application to the EU regulatory agency EMA. Upon this request, EMA will set the conditions for potential approval. This may require conducting a clinical trial in the EU.

Therefore, it is clear that the initiative to seek market authorization for NurOwn in the EU must be taken by BrainStorm, and not by EMA.

The ALS Liga Belgium cannot influence this process. Neither can we intervene to obtain the product.

However, Brainstorm launched an Hospital Exemption Program in Israel, where 13 pALS will be treated with the NurOwn therapy. According to this article in the Israeli newspaper Haaretz, 8 slots are for Israeli pALS at no cost, and 5 slots are reserved for foreign pALS that will each pay 300.000 USD.

While awaiting possible market authorization of NurOwn in the EU, we transparently offer pALS all information about e.g. NurOwn in the Research section of our website.

P.S. 1 The English press release of BrainStorm with their point of view regarding the Right To Try policy can be found here.

P.S. 2 Despite BrainStorm said it would no longer communicate as long as the phase 3 clinical trial in the USA and Canada was not finalized, their COO & CMO Ralph Kern gave this presentation at the 3rd Annual Neuroscience Innovation Forum at the JP Morgan Week.