First patient with rare skin disorder receives Clinuvel drug
Health & Biotech
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Clinuvel Pharmaceuticals (ASX:CUV) says the first patient with a rare skin disorder that makes them extremely sensitive to sunlight has received its drug aimed at repairing their DNA and increasing melatonin levels.
Clinuvel said the patient with xeroderma pigmentosum (XP-C) had received its Scenesse (afamelanotide 16mg) implant under a special access program.
People with XP-C suffer extreme sunburns in just a few minutes in the sun, and are 10,000 times more likely to get skin cancer.
They have a very short life expectancy, with an average lifespan in the 30s. An estimated one in 450,000 individuals in Europe suffer from the genetic disease.
Clinuvel said the first patient would be observed by an expert clinician for safety, and then the study will progress to a phase 2a trial in six XP-C patients and 10 healthy volunteers.
“We seek to provide meaningful benefit to XP patients, and these results will serve a wider population of fair-skinned individuals at risk of developing skin cancers,” Clinuvel clinical operations manager Dr Pilar Bilbao said.
“The next 12 months will be exciting for many patients, their families, the clinical experts and our own teams.”
Scenesse, a dissolvable rod about the size of a grain of rice typically implanted under the skin around a patient’s hip, has been approved in Europe and the United States to treat a less severe form of light sensitivity called erythropoietic protoporphyria (EPP).
The drug is believed to work by regenerating the DNA of skin exposed to ultraviolet light, increasing melatonin levels.
The first results from the study are expected next year.
Elsewhere in the ASX biotech space, Bionomics (ASX:BNO) says a trial of its BNC210 drug candidate will begin in healthy volunteers in late December or early January.
If all goes well a second phase 2b trial will begin in mid-2021 to test how well the drug works to treat post-traumatic stress disorder (PTSD), after a previous drug trial showed mixed results.
Meanwhile, Cynata Therapeutics (ASX:CYP) says a paper describing a phase-one clinical trial of its possible stem cell treatment for graft-versus-host disease has been published in a prestigious medical journal.
The study, describing how 13 out of 15 patients treated with the stem cell infusion CYP-001 showed improvement, was published in Nature Medicine.
“We are delighted that this paper has been published in Nature Medicine, which is a major endorsement of our technology and the significance of this trial,” Dr Kilian Kelly, Cynata’s chief operating officer, said.
Dr Kelly said the company’s licensing partner Fujifilm is further developing CYP-001.