Though there are few treatments for pemphigus vulgaris, a rare disease, the Believe-PV trial of PRN1008 raises the possibility of a new, better option.
The Believe-PV trial in patients with the rare disease pemphigus vulgaris investigated PRN1008, a Bruton tyrosine kinase (BTK) inhibitor that successfully treated the condition with far fewer side effects.
Dedee Murrell, MD, professor and chair of dermatology at the University of New South Wales, Kensington, Australia, spoke with MD Magazine® about the data at the 2019 Annual Meeting of the American Academy of Dermatology (AAD) in Washington, DC.
“With this tablet, which is a BTK inhibitor, the patients were able to get better with extremely low doses of steroids and in some cases with no steroids,” Murrell told MD Mag.
The presentation, “Final Results of the Believe-PV Proof of Concept Study of PRN1008 in Pemphigus,” was given at a late-breaking session at the AAD Annual Meeting on Saturday, March 2, 2019.
MD Mag: What will your presentation of the Believe-PV trial cover?
Murrell: I'll be presenting results from the Believe Pemphigus trial. This is a phase 2 trial for the first time in humans. It's a medicine which is called a Bruton tyrosine kinase (BTK) inhibitor and it was given for 3 months with low dose or no steroids. And the patients had amazing responses in terms of control of disease activity and remission rates on extremely low doses of steroids—by 4 weeks with control of disease activity, by 12 weeks—remission—and the relapse in the following 3 months took several months to weeks to happen.
What is the significance of this trial for treating pemphigus vulgaris?
Patients with pemphigus have risk of dying from the current treatments that are available. Before those treatments were there—steroids essentially 50 years ago—they all died. Now, the patients, 5-10% of them are dying from the treatments we give to the patients, mostly [from] infections, diabetes and complications induced by the steroid treatments. With this tablet, which is a BTK inhibitor, the patients were able to get better with extremely low doses of steroids and in some cases with no steroids. And the treatment has much less side effects than steroids.