Cereno Scientific submits request to the FDA for “compassionate use” of CS1 for treatment of patients with rare disease Pulmonary Arterial Hypertension (PAH)
Cereno Scientific (Nasdaq First North: CRNO B), a company developing innovative treatments for common and rare cardiovascular disease, today announced that the company has submitted a request to the FDA for Expanded Access, sometimes called “compassionate use”, to investigational drug CS1 for use in an extension of the ongoing Phase II trial evaluating CS1 in PAH. The initiative is prompted by a request from an investigator in the ongoing Phase II study. Cereno has submitted a request to the FDA under the ‘Expanded Access to Investigational Drugs for Treatment Use’ for expanded access to CS1, which initially will be limited to patients who have completed the Phase II study in PAH.
“I am very pleased to report that we now have submitted our request to the FDA for Extended Access to our innovative drug candidate CS1. By this action, we hope to be able to further extend Cereno’s support and contribution to patients with the rare disease PAH. The initiative to request “compassionate use” for CS1 was prompted by an investigator participating in the Phase II study and I am thankful to our team and collaborators who have worked hard the last couple of months to reach this point. In anticipation of an acceptance by the FDA, we have been making preparations to ensure that patients, who have been enrolled in the ongoing Phase II study, may experience no or as short as possible treatment pause with CS1. Such preparations include ensuring that CS1 is readily available immediately for eligible patients and that the drug will be available over a longer treatment period,” said Sten R. Sörensen, CEO, Cereno Scientific.
The Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. If Cereno’s request for expanded access to CS1 is authorized by the FDA and subsequently approved by the local hospital sites’ ethics committees, patients having completed the Phase II study will, in consultation with the investigator, have the option to continue with CS1 drug therapy. The expanded access will provide Cereno with the opportunity to, under a formal FDA-approved protocol, collect safety and efficacy data on extended long-term exposure to CS1 in patients. This allows gathering further information on CS1 use in this patient group, which can support later potential applications to the FDA such as fast-track designation/breakthrough therapy and the application to obtain the IND acceptance to start a Phase IIb/III study with CS1. According to regulatory guidelines, the FDA has 30 days to review the request, once received.
The Phase II study of CS1 in the rare disease PAH is actively running at 10 specialist clinics in the US with one new clinic currently in late-stage start-up process. The company has earlier this year reported positive findings from the ongoing study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH. First, a patient case study performed on the first patient having completed the study at a specific clinic showed remarkable efficacy data. In 12 weeks of treatment with CS1, the patient showed a 30% reduction in pulmonary pressure and a 20% increase in cardiac output. The patient’s overall functional status was changed from NYHA/WHO functional class II to I at the end of the treatment period, meaning that she had next to normal functional physical capacity with CS1. In addition, Cereno reported in October 2023 that a Data Quality Control Review (DQCR) was concluded with positive findings. The data quality of the CardioMEMS measurements was found satisfactory with adherence to study protocol and with timely data transfers from the patient's home to the clinic. Efficacy findings showed a clinically meaningful reduction of pulmonary pressure in several patients, included in the data quality control, of a similar or greater magnitude as in the Patient Case. The review included data obtained by the CardioMEMS HF System from the first 16 patients enrolled in the study and the reported findings can be read in full in a previous announcement.
For further information, please contact:
Tove Bergenholt, Director IR & Communications
Email: tove.bergenholt@cerenoscientific.com
Phone: +46 732-366 246
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
About Cereno Scientific AB
Cereno Scientific develops innovative treatments for common and rare cardiovascular disease. The lead drug candidate, CS1, is a HDAC (histone deacetylase) inhibitor that acts as an epigenetic modulator with pressure-reducing, reverse-remodeling, anti-inflammatory, anti-fibrotic and anti-thrombotic properties. A Phase II study is ongoing to evaluate CS1’s safety, tolerability, and efficacy in patients with the rare disease pulmonary arterial hypertension (PAH). A collaboration agreement with global healthcare company Abbott allows Cereno to use their cutting-edge technology CardioMEMS HF System in the study. Two initiatives performed during the ongoing Phase II study have shown positive findings suggesting the potential clinical benefit of CS1 in PAH patients. These initial findings are, however, not a guarantee of the final study results that are expected in Q2 2024. After requests by investigators in the Phase II study, a “compassionate use” application for CS1 is currently being pursued. Cereno also has two promising preclinical drug candidates in development through research collaborations with the University of Michigan. Investigational drug CS014 is a HDAC inhibitor in development as a treatment for arterial and venous thrombosis prevention. The innovative drug candidate represents a groundbreaking approach to antithrombotic treatment potentially without the associated increased risk of bleeding in humans. CS014 is a new chemical entity with a multi-fold mechanism of action as an epigenetic modulator – regulating platelet activity, fibrinolysis, and clot stability for the prevention of thrombosis without increased risk of bleeding as documented in preclinical studies. Drug candidate CS585 is a prostacyclin receptor agonist that has been documented in several preclinical studies to target the IP receptor for prevention of thrombosis without increased risk of bleeding, which also has been recognized in the medical community. CS585 was in-licensed from the University of Michigan in 2023. 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 Nasdaq First North (CRNO B). More information on www.cerenoscientific.com.