Innovation & Business Idea
Pluvia is developing a first in class small-molecule therapy to treat PKU patients, based on a novel therapeutic concept and approach: a pharmacological chaperone (PC) treatment to prevent the phenylalanine hydroxylase (PAH) mutation-associated misfolding and thereby restoring the native enzymatic activity of the patient’s own dysfunctional PAH. PKU is the most common inborn error of metabolism. Individuals with Phenylketonuria (PKU) have reduced ability to convert the amino acid Phe to tyrosine, due to misfolding of phenylalanine hydroxylase (PAH) causing a toxic build-up of Phe. Left untreated, the PKU patients will develop chronic neurological disabilities through their childhood, severely affecting their ability to lead a normal life.
Customers / Target Market
The PKU population in the western world is well defined, as PKU is part of the newborn screening program: total population approximately 50,000, of which 28,000 are under active follow-up at centralised PKU clinics.
One drug treatment for PKU is Kuvan (Biomarin), approved in 2008. Kuvan is only effective in a sub-group of the patients (20-30%), and requires the patients to remain under strict dietary control. Another key competitor is Pegvaliase (Biomarin), a plant-based enzyme substitution therapy launched in 2018 and is expected to play a limited role given its side effect profile (immunogenicity), route of administration (daily injections), burden of titration (up to 32 weeks) and indications of loss of effect over time. Pegvaliase is only given to adults. Our molecules will be differentiated by targeting a larger sub-group of the patient population (efficacy >40%); No longer requiring medical diet for mild patients; target population >1 month of age.
Intellectual Property Status
Following promising proof of concept data, we have filed the patent application “HYPERPHENYLALANINEMIA AND TREATMENTS THEREOF“, international publication number WO 2017/029202 A1, international pub. date 23.02.2017 with several patent claims on the use of molecules.
Development Status & Future Steps
Pluvia is developing an integrated drug development program focusing on pharmacological chaperone based therapies. The company is currently in lead optimisation phase for the PKU program. The major objective for the company is to achieve the filing of a Clinical Trial Application (CTA) for Pluvia’s pharmacological chaperone for PKU treatment by end of 2021. Pluvia´s long-term strategic ambition is to leverage the company’s proprietary expertise and technology platform to address multiple medical indications that involve protein misfolding.
The company´s commercialisation plan is to position the PKU project for entering a collaboration agreement with a pharmaceutical partner as we approach the clinical stage. Pluvia has as its core strength the research capabilities on protein biochemistry and chaperones. We expect to be able to enter a collaboration agreement for the PKU project at attractive terms once we approach the clinical stage.
Pluvia’s management team is composed of experienced biotech executives with Torgeir Vaage as CEO of the company. Prof. Aurora Martinez is CTO with a longstanding experience in biochemistry. Dr Mikael Thomsen is director of drug development, having worked in the pharmaceutical area for close to 20 years. Dr Anne-Sophie Schillinger is responsible of the company program management and business development.
The future fundraising plans for the company total € 6-8 M, to cover the company´s costs associated our PKU drug development program and to accelerate the start-up of the clinical phase after the end of the preclinical development phase.
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