Dermatology company Botanix Pharmaceuticals (ASX: BOT) has received the green light from the US Food and Drug Administration (FDA) for its BTX 1503 phase 2 clinical acne trial.
Botanix is currently developing BTX 1503, as a new treatment for moderate to severe acne. The basis of its methodology is to use synthetic cannabidiol to target multiple pathologies involved in the development of acne utilising Botanix’s proprietary Permetrex drug delivery technology.
The trial is now expected to go ahead in the coming months with Botanix making immediate moves to begin the study by announcing plans to enrol the first acne patients this quarter. According to Botanix, the planned clinical acne trial will involve over 30 dermatology clinics in the US and Australia.
Botanix claims that the BTX 1503 phase 2 clinical trial is “fully funded” following its recent capital raising in February 2018 and it plans to enrol approximately 360 patients over the next few months with final completion date forecast for mid-2019.
The phase 2 trial is designed to generate sufficient scientific data to allow Botanix to explore licensing and other corporate opportunities upon its successful completion at the end of the second quarter of 2019.
Approving the battle against acne
To gain FDA approval for BTX 1503, Botanix submitted a comprehensive set of documentation regarding the drug candidate’s safety profile, and, importantly, its ability to treat acne.
In the first quarter of 2018, Botanix successfully completed a phase 1b study in patients with moderate to severe acne which demonstrated that, on average, BTX 1503 was able to reduce inflammatory acne lesions by 47%.
According to Botanix, this reduction is greater than any other FDA-approved topical acne product, for which data is available after 4 weeks of treatment — which makes it a significant achievement that warrants further investment.
The study achieved all the BTX 1503 program goals, indicating that BTX 1503 has an excellent safety profile, is very effective at reducing the number of inflammatory and non-inflammatory acne lesions, and possibly most crucial of all, provides “improvement in patient satisfaction.”
Acne is currently the most common skin disorder in the US affecting 40-50 million people every year and more than 250 million patients worldwide.
Acne has multiple pathogenic pathways including overproduction of oils, inflammation and bacterial infection, but the only approved products that can influence oil production and are currently available to patients (Accutane and Roaccutane) carry significant side effects, including birth defects, various lymphomas and a higher propensity for suicide amongst people taking Accutane or Roaccutane on a regular basis.
Botanix is confident its BTX 1503 treatment can avoid such severe side-effects reported by acne sufferers because it is not administered orally. The treatment is applied topically to the surface of the skin thereby offering localised delivery to only acne-affected areas, rather than the entire body via the bloodstream.
Botanix says that BTX 1503 has the potential to generate “similar or greater revenue” than the two leading topical acne products which are similarly applied to the surface of the skin only: Aczone which generated US$456 million in 2016 and Epiduo which generated US$494 million in the same year.
However, both products also had significant side-effects reported by patients.
Aczone can cause methemoglobinemia, a serious condition in which the amount of oxygen in the bloodstream becomes dangerously low, as well as allergic reactions such as hives and swelling. Meanwhile, Epiduo uses benzoyl peroxide which has been reported to cause serious allergic reactions and severe skin irritation.
Botanix says that its mission is to “improve the lives of patients battling acne”, atopic dermatitis and other skin diseases, by providing new treatment options for conditions that are currently inadequately addressed or are treated with counterproductive therapeutics that are burdened with side effects profiles.
“We are very pleased to clear this important hurdle with FDA and now have a clear and rapid path to completing the phase 2 program in the US following the recent success of our phase 1b study,” said Matt Callahan, executive director of Botanix.
Botanix’s share price was unchanged at A$0.14 in mid-morning trade.