Cereno Scientific reports progress of Expanded Access Program and announces timeline adjustment in the Phase II Study of CS1 in PAH
Cereno Scientific (Nasdaq First North: CRNO B), a company developing innovative treatments for common and rare cardiovascular disease, today announced an update on the progress of the Phase II study of CS1 in pulmonary arterial hypertension (PAH). Cereno has seen substantial interest in the FDA-approved Expanded Access Program (“compassionate use”) for patients who have completed the Phase II study, with investigators indicating that a majority of patients would be interested in continued access to CS1 following study completion. The company reports considerable progress in the study. Due to a longer start-up phase for the two new study sites than previously estimated, the study timeline has been negatively affected and top-line results are now expected in Q3 2024.
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 activated in late December and one new clinic currently in late-stage start-up process. To date, 25 patients have received the CardioMEMS HF System implantation, of which 23 patients have been randomized to CS1 drug therapy; and 19 patients have completed the study. This means that there are 7 patients remaining to be randomized to CS1 drug therapy, 2 of which are already on CardioMEMS.
Cereno Scientific has during 2023 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 the patient had next to normal functional physical capacity with CS1.
In addition, Cereno reported in October 2023 that a Data Quality Control Review (DQCR), of data obtained by the CardioMEMS HF System from the first 16 patients, 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.
Further, the Expanded Access Program (EAP) (“compassionate use”) approved by the FDA on 31st January 2024 is progressing according to plan, propelled by investigator and patient interest. Investigators indicate that close to two-thirds of the patients, having completed the study or are currently on therapy, have been judged to be interested in continued access to CS1 following study completion. Currently site-specific and IRB approvals are being progressed. The EAP will provide Cereno with the opportunity to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH. The EAP will thus allow Cereno to gather further documentation of CS1 use in patients suffering from PAH, which will help further inform the design of our Phase IIb/III pivotal study with CS1.
“We look very much forward to starting patients on CS1 treatment within the Expanded Access Program, after seeing the remarkable clinical results in the Patient Case and subsequently the positive findings seen in the DQCR. As investigators confirm an interest for a majority of the patients at their sites for continued access to CS1 following study completion, we move forward with manufacturing in larger quantities, so-called scale-up manufacturing, of CS1 with CDMO CordenPharma, to ensure timely supply, and safeguarding long-term availability of CS1 for the EAP and as preparation for the pivotal study,” said Sten R. Sörensen, CEO, Cereno Scientific.
While the company reports notable progress in the study, a slower recruitment pace than estimated during the last months and a longer start-up phase for the new sites have affected the study timeline. The updated study timeline now expects study completion and top-line results during Q3 2024.
“The reported interest from the investigators for the continuation of CS1 for the patients they have been treating with CS1 in the phase II study for 12 weeks is very encouraging and provides us with confidence that our drug has provided clinical utility in these patients as judged by the investigators so far. We are eager to finalize the randomization to CS1 drug therapy of the additional 7 patients and reach 30 patients to complete the study. We are actively engaging with the investigators and sites to ensure a timely progress for study completion during Q3 2024.”, said Sten R. Sörensen, CEO, Cereno Scientific.
For further information, please contact:
Henrik Westdahl, Director IR & Communications
Email: henrik.westdahl@cerenoscientific.com
Phone: +46 70-817 59 96
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
This information is information that Cereno Scientific AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 17:55 (CET) on February 21, 2024.
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 Q3 2024. Since January 2024, CS1 has been available under FDA’s Expanded Access Program (“compassionate use”) for continued CS1 treatment in patients who have completed the Phase II study. 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.