Cereno Scientific reports six activated clinical sites and updated timeline in the Phase II study in PAH with drug candidate CS1

Cereno Scientific (XSAT: CRNO B) today announced that six clinical sites across the US are activated and actively recruiting patients in the ongoing Phase II study in pulmonary arterial hypertension (PAH) with drug candidate CS1. To date, two patients have been randomized and entered the treatment period. An amendment to the study protocol is now in effect with the aim to primarily broaden the patient inclusion criteria and simplify the screening process while preserving high-quality data in the study. The study’s top-line results are expected in mid-2023 based on the adjusted timeline caused by an extended start-up phase.

“The Phase II study has been actively recruiting patients since July with two centers activated. We have previously reported that it has taken significantly longer to activate clinical sites for the study, which can be attributed to longer lead times for ethical approvals and contracting mainly due to the post-pandemic effects of personnel shortages or exchanged staff at academic centers. The Clinical Steering Committee has reviewed the study protocol and made amendments to the patient inclusion criteria as one way of increasing the recruitment pace as it allows for an expansion of the possible patient population in the study. We do not foresee any negative impact on the study based on this change and are now looking forward to seeing this amended study protocol coming into play,” said Björn Dahlöf, Chief Medical Officer (CMO) at Cereno.

The Phase II study in PAH with drug candidate CS1 has received accolades for its innovative design. The patient recruitment stage starts with the patient undergoing a screening process, implantation of the CardioMEMS HF System to monitor blood pressure in the pulmonary circulation and other cardio-pulmonary hemodynamics on a daily basis during the study, and a full baseline evaluation including a 6-minute walk test, echocardiography, biomarkers, patient-reported outcomes, and MRI to enable exploration of CS1’s efficacy. Once baseline evaluation is completed, the patient will receive their first dosing and undergo a 12-week drug treatment period and a two-week follow-up period. The study is designed to include 30 patients.

“The unmet medical need for patients with PAH is tremendous. We believe our drug candidate CS1 has the very highly sought-after potential to reverse the progression of PAH, and not only alleviate the symptoms as today’s available treatments. The impact the lingering covid-19 pandemic in the US has had on the study’s start-up timeline does not take away the importance or our drive to pursue a better treatment option for patients and families affected by PAH. The ongoing Phase II study with CS1 in PAH has the potential to make a major difference in the future treatment landscape,” said Sten R. Sörensen, CEO at Cereno.

In March 2020, Cereno received the US FDA’s orphan drug designation (ODD) for the clinical development program for CS1 in PAH. Through the granted ODD, the FDA has indicated that they believe CS1 has the potential to provide significant benefit to patients suffering from PAH. In September 2021, an investigational drug application (IND) was accepted by the FDA to start a Phase II multi-center PAH study in the US. Since then, many activities and processes have been executed culminating in the activation of clinical sites participating in the study and the subsequent patient enrollment. The first patient entered the study receiving the first dose in August 2022 and top-line results are expected in mid-2023.

For further information, please contact:

Hans Naess, Interim CFO
Phone: +46 793 40 79 00
Email: info@cerenoscientific.com
http://www.cerenoscientific.com/

About the Phase II study with CS1 in PAH

CS1 was granted an orphan drug designation (ODD) for the treatment of the rare disease PAH by the US FDA in March 2020 and in September 2021, Cereno obtained FDA acceptance of an investigational new drug (IND) application allowing initiation of the Phase II study. This study intends to evaluate drug candidate CS1’s safety, tolerability, dose, and exploratory efficacy in patients with PAH. A collaboration agreement with global healthcare company Abbott allows Cereno to use Abbott’s cutting-edge technology CardioMEMS HF System in the study. There will be a 12-week drug treatment period and a two-week follow-up period. The primary endpoint is safety and tolerability. All standard efficacy endpoints for this patient group will be explored as well as validated risk scores before and after treatment. The study includes at least ten different clinical sites in the US and aim to include 30 patients with PAH.

Clinical Steering Committee members: Dr Raymond Benza (Chair), Dr Phil Adamson, Dr Deepak Bhatt, Dr Björn Dahlöf, Dr Gunnar Olsson and Fredrik Frick (Secretary).

Further information is available at www.clinicaltrials.gov / (ClinicalTrials.gov: NCT05224531).

About Cereno Scientific AB

Cereno Scientific is a clinical stage biotech company within cardiovascular diseases. The lead drug candidate, CS1, is a Phase II candidate in development for the treatment of the rare disease pulmonary arterial hypertension (PAH). CS1 is an HDAC (histone deacetylase) inhibitor that acts as an epigenetic modulator with pressure-reducing, reverse-remodeling, anti-inflammatory, anti-fibrotic and anti-thrombotic properties, all relevant for PAH. A clinical Phase II study is ongoing to evaluate CS1’s safety, tolerability and efficacy in patients with PAH. A collaboration agreement with global healthcare company Abbott allows Cereno to use their cutting-edge technology CardioMEMS HF System in the study. Cereno also has two promising preclinical drug candidates in development for cardiovascular disease through research collaborations with the University of Michigan. Drug candidate CS585 is a stable, selective, and potent prostacyclin receptor agonist. It has been documented in preclinical studies to target the IP receptor for prevention of thrombosis without increased risk of bleeding. Drug candidate CS014 is a novel HDAC inhibitor with epigenetic effects. In preclinical studies it has been documented to regulate platelet activity, fibrinolysis and clot stability for prevention of thrombosis without increased risk of bleeding. The company is headquartered in Gothenburg, Sweden, and has a US subsidiary Cereno Scientific Inc. based in Kendall Square in Boston, Massachusetts, US. Cereno is listed on the Swedish Spotlight Stock Market (CRNO B). More information on www.cerenoscientific.com.