Neurelis has the license to bring the ROCK inhibitor NRL-1049 to trials specifically for CCM. The medicine was developed by BioAxone as BA-1049 and licensed to Neurelis for trials. The medicine is in Phase 1 clinical trials in healthy volunteers.
Ovid therapeutics/graviton bioscience
Ovid and Graviton are working to develop a ROCK2-specific inhibitor known as OV-888 (formerly GV101) to target CCM. Graviton developed the medicine and has licensed it to Ovid for clinical trials. The medicine is in Phase 1 clinical trials in healthy volunteers.
Several medicines already on the market for other indications are seeking funding for Phase 2 or Phase 3 trials with our patients:
Propranolol has already completed a small multi-center safety trial in Italy. According to the research abstract:
Propranolol was safe and well tolerated in this population. Propranolol might be beneficial for reducing the incidence of clinical events in people with symptomatic familial cerebral cavernous malformations, although this trial was not designed to be adequately powered to investigate efficacy. A definitive phase 3 trial of propranolol in people with symptomatic familial cerebral cavernous malformations is justified.
Funding is being sought through multiple European agencies.
While it seems counter-intuitive, some researchers believe that antithrombotic medicines do not increase hemorrhage risk and that low-dose aspirin may offer protective effects against hemorrhage. Multiple retrospective case series studies have offered supporting evidence. Funding is being sought in the UK for clinical trial.
Rapamycin, also known as sirolimus, is an mTOR-inhibitor used in multiple disorders involving vascular malformations and has been tested in patients with other slow-flow vascular malformations. It has been tried in acute mouse models of CCM and appears to have an effect. Studies of chronic mouse models are pending. Funding for a clinical trial is being sought in the US.