UPDATE: Cipher Pharmaceuticals - Incidental discovery involving piclidenoson in pre-clinical studies for the treatment of Lowe Syndrome
In an unexpected turn of events, it appears that piclidenoson could be applicable for the treatment of a rare genetic disease called Lowe syndrome which has no existing available drugs.
“Lowe syndrome (LS) or oculocerebrorenal syndrome of Lowe (OCRL) is a rare disorder characterized by multiple features occurring mainly in males. Its prevalence is approximately 1 in 500,000. Its systemic manifestations include mental retardation, hypotonia, and kidney dysfunction in the form of Fanconi syndrome. Some patients with LS also have bleeding disorders, with normal to low platelet count and impaired platelet function. Ocular manifestations can include congenital cataracts, corneal keloid, and infantile glaucoma.”
If you’re unaware of what piclidenoson is in the context of Cipher Pharmaceuticals, Cipher purchased canadian rights to the drug for considerations of $3.65mm USD in 2015 (upfront payment of US$1.65 million and is eligible for milestone payments of up to US$2.0 million + royalties from product sales).
Piclidenoson is an oral drug created for the purpose of treating moderate to severe plaque psoriasis (ongoing phase 3 trials) and rheumatoid arthritis (which has since been canceled after failure to meet its endpoints).
“Piclidenoson is the only compound we’ve found to date that has shown to be effective in pre-clinical studies”
“Having tested thousands of compounds in search of a treatment for Lowe Syndrome, Piclidenoson is the only compound we have found to date that has shown to be effective in pre-clinical studies. Importantly, we observed that Piclidenoson treatment in mouse models of Lowe syndrome leads to a significant decrease of the urinary loss of proteins in diseased animals,” De Matteis said. “We chose to investigate Piclidenoson based on the availability of extensive scientific data showing its excellent safety, coupled with efficacy in this disease which involves renal, cerebral, and ocular manifestations.”
Can-Fite Chairman Dr. Pnina Fishman commented, “We are hopeful that Piclidenoson can offer a much-needed treatment for infants, children, and young people living with Lowe Syndrome. Based on Piclidenoson’s proven safety profile in clinical trials to date, and because Lowe is a rare disease in dire need of treatment, we plan to move into an advanced stage clinical study which may open a path to approval. Dr. De Matteis and her team have made an impactful discovery with Piclidenoson and we look forward to working with her and Fondazione Telethon.”
I’m currently unaware of how reimbursement would work here should this turn out well in clinical trials - and I expect we will know fairly quickly due to no existing therapeutic options along with the already extensive safety data involving piclodenoson. Intuitively I would be shocked if Cipher would be able to reimburse the drug at conventional rates for rare disease, but you never know. I will provide an update when I find out.
Until next time,