Cereno Scientific intends to initiate a phase II study in September
At the end of last week, Cereno Scientific announced that the timeline for the upcoming phase II trial with drug candidate CS1 has been set. The announcement comes after the signing of the final collaboration agreement with clinical research organisation (CRO) Worldwide Clinical Trials. According to the timeline, the first patient with the rare disease pulmonary arterial hypertension (PAH) will receive treatment in September 2021, and study results will be presented during the second half of 2022.
In mid-January, biotech Cereno Scientific signed a letter of intent (LOI) with the CRO Worldwide Clinical Trials for support and guidance in the final preparatory steps for as well as the entire execution of the phase II clinical study with drug candidate CS1 in PAH. In the meantime, an investigational new drug (IND) application to the FDA necessary for beginning the study in the US has been initiated, and, according to the established timeline, the study is expected to begin in September 2021.
CS1 in PAH
Cereno Scientific was granted orphan drug designation (ODD) by the FDA in March 2020. In September that same year, Cereno announced that the indication PAH would be prioritised for candidate CS1.
Based on an earlier pre-IND meeting with the FDA, which according to the company was successful, the chances Cereno being granted approval to initiate the clinical study look good. The purpose of this pre-IND-meeting was to confirm the study design and the clinical development plan for CS1.
The aim of the phase II study in PAH is to evaluate the safety and tolerability of CS1 in this severe disease and to evaluate the initial therapeutic effect of CS1 in this indication, as well as establishing the correct dose for further studies.
Looking onwards to phase III
In order to prepare for an upcoming phase II/III, the company states that, in addition to safety and tolerability, all relevant standard efficacy endpoints for this patient group will be evaluated and a risk score will be calculated and validated.
The dose to be used in later studies will be derived from cutting-edge technique with continuous monitoring of pulmonary pressure. The study will involve at least six different clinical centres in the United States and include 30 patients under the supervision of the study’s lead investigator, Dr Raymond Benza, a global thought leader in PAH and a member of Cereno’s Scientific Advisory Board (SAB). Read more about the key role that Dr Raymond Benza plays at Cereno Scientific here.
Strong need for new PAH treatments
PAH is a progressive and incurable disease leading to a gradually deteriorating lung function and oxygenation as well as increasing heart failure, which results in a significantly reduced quality of life and a high mortality rate. PAH is a rare disease that hits approximately 10/100 000 individuals in the USA, and more than half of the cases are attributed to idiopathic and hereditary causes. Other cases are associated with drugs, toxins and diseases such as HIV and congenital heart disease. Most patients are adults over 65 years of age, and more women are affected than men, but the disease can affect younger people as well.
The current standard treatment consists of different drugs that have limited efficacy and often need to be combined with each other to achieve acceptable treatment results. The medical need for new treatments is therefore significant, and the fact that Cereno Scientific has obtained ODD for CS1 in PAH strengthens the company ahead of its clinical development. Furthermore, previous pre-clinical studies have been able to demonstrate that the active substance in CS1 acts as an epigenetic modulator with anti-thrombotic, anti-inflammatory, anti-fibrotic and pressure-reducing properties, all of which are relevant in the disease profile for PAH.
