OUR SOLUTION
Since 2017, Patient One has been working on the development of a novel sustained-release cysteamine bitartrate formulation, that is accessibly priced and has a good treatment experience.
We now have a novel formulation that shows great potential in the pharmacokinetic-pharmacodynamic model for Cystinosis.
OUR JOURNEY
We would like to take you along on our journey towards bringing to market this new cystinosis treatment.
1970’s
CYSTEAMINE
Since the late 70s, we know that cysteamine can be effective in slowing down the development and progression of kidney damage and can enhance growth in children.
The first immediate-release cysteamine formulation was registered in 1997. The immediate-release formulation needs to be administered every six hours to prevent irreversible damage.
2017
FOUNDATION OF PATIENT ONE
In 2017, Patient One is founded to develop and market a novel sustained-release cysteamine bitartrate treatment, making the administering less invasive, e.g. not breaking up nights.
2018
FORMULATION PO-001
In 2018 a new formulation of cysteamine is developed in the lab. PO-001 is designed to provide prolonged and consistent levels of the drug in the body, while minimizing adverse effects.
2019
DEVELOPMENT
The new formulation is produced in the pharmaceutical plant, preparing the novel formulation for the first clinical trial.
2019
PROOF OF CONCEPT
In 2019 a first clinical trial in healthy volunteers is conducted to examine the pharmacokinetics and the safety of the novel formulation.
In the double-blinded trial, 9 healthy volunteers are given PO-001 and the two commercially available cysteamine bitartrate formulations, in a random order with a week wash-out between every dose.
The trial concludes that the novel formulation showed pharmacokinetics of a sustained-release formulation, which may be beneficial for the treatment of cystinosis patients, the product can however be further optimized.
2020 – 2022
RE-FORMULATION and Development
A new, improved formulation of the novel sustained-release cysteamine bitartrate formulation, PO-003, is finalized.
Currently the team is working on developing and validating the analytical method. When the product is proven to be stable, a clinical batch will be produced. It is expected that a first clinical trial will be conducted in 2023 with PO-003.
2023
PK TRIAL
To examine the properties of PO-003, a clinical trial will be conducted in healthy volunteers, in which the novel sustained release cysteamine formulation will be compared with the existing imideate release cysteamine bitartrate formulation.
2024
CLINICAL TRIAL
In case the novel formulation shows the required pharmacokinetic properties in healthy volunteers, an additional trial will be conducted with cystinosis patients to examine the safety and efficacy of the new product.
2025 – 2026
REGISTRATION & COMMERCIAL MANUFACTURING
After the clinical trial, production of the final product at a commercial scale shall be initiated, including gathering the necessary information for the product dossier.
Simultaneously, all data on pre-clinical and clinical will be added to the product dossier, resulting in a marketing authorisation application on the basis of a hybrid application of an existing imideate release formualtion, which will be filed at the European Medicines Agency (EMA).
ACCESSIBLE PRICING
We would like to take you along on our journey towards bringing to market this new cystinosis treatment.
GOOD PATIENT EXPERIENCE
Working closely with patients and patient representatives, we strive to make our treatment – next to accessibly priced – as friendly to take as possible.
WILL YOU
JOIN US?
To make our next steps in the development process possible, we need additional investments.
Will you join our cause?