To date Clinuvel Pharmaceuticals' (CUV: $10.03) commercial business has been built on the success of its product Scenesse for the treatment of EPP, a disease characterized by severe sunlight intolerance.
Currently the company has the market to itself. In the first half of this financial year, the company generated revenue from this product totaling $36.9 million, up 4% from the PCP. Net profit for the half was down 26% to $10.4 million, due to the investment in its Phase III vitiligo program and commercialization of its ACTH generic. The company is also investing in its peptide drug delivery platform.
Clinuvel Maintains Monopoly as Disk Medicine Receives CRL
Last month one of its potential competitors in the EPP market, Disk Medicine, received a Complete Response Letter from the FDA for its accelerated approval application for the treatment of EPP with its drug candidate bitopertin.
Whilst the company's Phase II studies had showed a 40% reduction in the surrogate marker PPIX (protoporphyrin IX), the FDA's view was that a correlation between PPIX levels and increased sun exposure time had not been proved.
Disk Medicine is conducting a Phase III study in 150 subjects with EPP and XLP (X-linked protoporphyria) with results expected by the end of this year. However in the meantime the company plans to reduce is staff by 20% (from 150 staff) as a direct result of the FDA decision, mostly in its commercial and support functions. At the end of 2024, the company had just 84 employees.
Bitopertin had originally been developed as an anti-psychotic by Roche, at a 20mg dose. However Disk Medicine has to increase the dose to 60mg to achieve the drop in PPIX, which the FDA referred to as a 'relatively modest' decrease. Increasing the dose can have a negative impact on the liver, with EPP patients already having a higher risk of liver damage according to Clinuvel's head of North American operations, Linda Teng. Teng is also skeptical about the use of a compound that affects the CNS for lifetime use (as a treatment for EPP).
Clinuvel continues to maintain and grow its monopoly in the global EPP market.
Key Events for 2026
A key milestone for this year for Clinuvel will be results from the first Phase III vitiligo study, and filing of Neuracthel for approval in Europe.
Whilst the FDA has stated that it intends to move to the requirement of only one Phase III study for the approval of new pharmaceuticals, Clinuvel will still need to complete both of its trials in vitiligo before submitting Scenesse for approval for this new indication. The company will file in Europe first and then following with an FDA submission. Given the novelty in the company's approach, Clinuvel believes processing through the EMA first will aid in the FDA approval process.
With respect to competition in vitiligo, Clinuvel's Director of Global Clinical Affairs, Emilie Rodenburger, noted that one JAK inhibitor had been filed for approval for the treatment of vitiligo. However these drugs take a long time to take effect and work by suppressing the immune system.
By contrast, the safety profile of Clinuvel's Scenesse has been established from over a decade of use with over 21,500 doses delivered to patients. "Vitiligo is a natural home for afamelanotide, a pigment activating peptide, which is an analogue of a hormone that is naturally produced by your own body," said Rodenburger.
With the company's ACTH generic, called Neuracthel, the company will also file first in Europe for approval, in the second half of this year, with the product to be sold through specialty centres in Europe, which is where they are distributed (e.g. through endocrinologists).
Summary
Clinuvel finished last year with $233 million in cash. Its revenue from Scenesse in EPP is fairly evenly split between the US and Europe (53%/47%). The company is capitalised at $502 million.
CEO Philippe Wolgen said that the company had five core objectives. These are:
♦ To expand the EPP market
♦ Advance the Phase III vitiligo program (first Phase III read out later this year)
♦ Advance the ACTH program (European filing this year)
♦ Commercial launch of the photocosmetic products (in conjunction with vitiligo results), and
♦ Bring in-house manufacturing for new and next formulation products.
Bioshares recommendation: Buy
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