Targeting rare diseases

At OCT, we are committed to harnessing the therapeutic power of cannabinoids to develop breakthrough therapies for a range of debilitating conditions.?We are excited that, soon, we will be starting our clinical programme with OCT130401, for Trigeminal Neuralgia (“TN”), as we recently announced completion of funding for our Phase 1 trial which will take place in Australia.

All of the conditions that we are targeting are in areas of key clinical need where people either do not respond to current treatments or there simply aren’t effective medicines licensed for their condition. Trigeminal Neuralgia is a good example.? While there are drugs approved for TN, their effectiveness is limited but because this painful condition affects a small number of people, there has been little interest in finding a new treatments for it.?

As a rare disease, it does not make the condition any less important and regulators, such as the FDA and EMEA are active in encouraging companies to commercialise medicines for these conditions offering a special ‘orphan designation status to a given medicine.?For us at OCT, finding treatments for these uncommon diseases is a key focus because of these advantages. First it gives us market exclusivity, which is vital to protect our research and intellectual property for the work we are doing on cannabinoids, a well-understood substance which without exclusivity would never be commercially developed. Whilst clinical studies are run for orphan designated medicines in the same way as for non-orphan designated medicines, the number of study patients required for approval may be lower, reflecting the relative rarity of the condition treated.?This can help in reducing development costs. The regulatory authorities also offer grants and tax credits to Companies like OCT to develop these medicines and actively engage with the Company at every stage. Pricing is likely to be favourable and offer a good return on investment for us and our shareholders.

We look forward to the start of our Phase 1 trial for OCT130401 and to bringing further compounds from Programmes 3 and 4 into development that will also aim to secure Orphan Designation.