sodium valproate delayed release (CS1) / Cereno Scientific - LARVOL DELTA

Cereno Scientific Receives FDA Clearance to Initiate Global Phase IIb Trial of CS1 in Pulmonary Arterial Hypertension (PAH) (PRNewswire) - "The FDA's decision enables Cereno to advance toward first patient in (FPI) in Q2 2026, with top-line data anticipated around Q4 2028, subject to enrollment timelines."

IND • New P2b trial • Pulmonary Arterial Hypertension

Safety Profile of the CardioMEMS HF System in Pulmonary Arterial Hypertension: A Pooled Adverse Event Analysis from Three Clinical Studies (AHA 2025) - P, P2, P4 | "However, its safety in patients with PAH remains undefined.Hypothesis:The use of the CardioMEMS is associated with an acceptable safety profile in patients with PAH.Adverse event data were combined from the completed CS1-003 (NCT05224531), on-going ARTISAN (NCT05203510), and on-going FIT_PH (NCT04078243) studies...The use of CardioMEMS in this combined PAH cohort has demonstrated a favorable safety profile, with few procedure-related adverse events and no enduring consequences. Future studies are warranted to investigate its long-term safety and efficacy in this population."

Adverse events • Clinical • Cardiovascular • Pulmonary Arterial Hypertension • Respiratory Diseases • Rheumatology

Cereno Scientific Granted FDA Fast Track Designation for CS1 in Rare Disease Pulmonary Arterial Hypertension (PAH) (TradingView)

Fast track • Pulmonary Arterial Hypertension

Cereno Scientific secures 100 MSEK through additional loan financing and conversion to advance HDAC inhibitors CS1 and CS014 in Phase II (Firstwordpharma Press Release) - "Cereno Scientific...announced entering loan financing agreements securing a total of 100 MSEK....The proceeds from the Addendum and the New Financing Agreement are primarily intended for: Advancing CS1 Phase IIb trial to initiation through preparation of regulatory documentation, obtaining approvals and conducting start-up activities; Initiating a Phase II program for CS014; Progressing the Expanded Access Program (EAP), including the Fluidda sub-study, to gain insights into the long-term use of CS1 in patients with pulmonary arterial hypertension (PAH); Continuing and expanding the company’s global partnering activities Continuing to strengthen footprint in the scientific community through publications and presentations."

Financing • Pulmonary Arterial Hypertension

Cereno Scientific receives endorsement from FDA on plans for Phase IIb trial of CS1 in rare disease pulmonary arterial hypertension (PAH) (Firstwordpharma Press Release) - "Cereno Scientific...announced that the US Food & Drug Administration (FDA) provided endorsement of the plans for the Phase IIb trial of CS1 as noted by the official meeting minutes from the Type C-meeting held on April 21. The next clinical development step of CS1 is a larger, placebo-controlled Phase IIb trial aiming to further evaluate the encouraging efficacy signs including reverse vascular remodeling and improvement of right heart function as observed in the Phase IIa trial....'We are now continuing steps for preparations ahead of the Phase IIb trial in PAH, with a target of initiating the trial in H1 2026.'"

FDA event • New P1 trial • Pulmonary Arterial Hypertension

The Effects of Drug Candidate CS1 on Lung Structure and Function Utilizing FRI (clinicaltrials.gov) - P=N/A | N=7 | Active, not recruiting | Sponsor: Prisma Health-Upstate | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Cereno Scientific completes successful FDA meeting discussing further clinical development of drug candidate CS1 in rare disease PAH (Cision) - "Cereno Scientific...today announced that the company has completed a Type C meeting with the U.S. Food and Drug Administration (FDA). The intention was to seek advice from the FDA to finalize the Phase IIb trial design and align on further clinical development steps for CS1 based on the encouraging signals suggesting reverse vascular remodeling effects observed in the Phase IIa trial. The discussions during the meeting indicate alignment between the FDA and Cereno Scientific on the plans. An update on the CS1 program will be shared following receipt of the official meeting minutes....The FDA Type C meeting that was held on April 21, 2025, focused on finalizing the design of the Phase IIb trial and aligning on further clinical development steps."

FDA event • Pulmonary Arterial Hypertension

The Effects of Drug Candidate CS1 on Lung Structure and Function Utilizing FRI (clinicaltrials.gov) - P=N/A | N=7 | Recruiting | Sponsor: Prisma Health-Upstate

New trial • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Cereno Scientific extends patent protection for drug candidate CS1 in the US and has filed to extend market exclusivity to 2045 (Cision) - "Cereno Scientific...announced that a new patent has been granted in the US for drug candidate CS1’s second patent family. Two patent applications have, additionally, been filed based on the encouraging efficacy signals observed in the recently completed Phase IIa trial of CS1 in the rare disease pulmonary arterial hypertension (PAH). These patent applications combined with the existing patent portfolio has the potential to extend the market exclusivity for CS1 in PAH to 2045."

Patent • Pulmonary Arterial Hypertension

Cereno Scientific extends patent protection for drug candidate CS1 in the US and has filed an application to extend market exclusivity until 2045 [Google translation] (Cision) - "Cereno Scientific...today announced that a new patent has been issued in the United States in the second patent family of its drug candidate CS1. Two additional patent applications have been filed based on the compelling efficacy signals observed in the recently completed Phase IIa study of CS1 in the rare disease pulmonary arterial hypertension (PAH). These patent applications, together with the existing patent portfolio, have the potential to extend market exclusivity for CS1 in PAH through 2045....The patent is entitled 'Valproic acid for the treatment or prevention of pathological conditions associated with excess fibrin deposition and/or thrombus formation' and has been assigned patent number 12,245,999 by the US Patent and Trademark Office."

Patent • Pulmonary Arterial Hypertension

Cereno Scientific secures FDA meeting to advance its development of CS1 for the treatment of rare disease PAH (Cision) - "Cereno Scientific (Nasdaq First North: CRNO B), an innovative biotech pioneering treatments to enhance and extend life for people with rare cardiovascular and pulmonary diseases, today announced that a Type C meeting has been scheduled on April 21, 2025, by the U.S. Food and Drug Administration (FDA)...An update on the results of the meeting with the FDA is planned to be shared upon receipt of the written meeting minutes. Drug candidate CS1 is an epigenetic modulating HDACi that aims to improve quality of life and expand life expectancy for patients with the rare disease pulmonary arterial hypertension (PAH)."

FDA event • Pulmonary Arterial Hypertension

Cereno Scientific received approval to initiate sub-study of the CS1 EAP using Fluidda’s innovative imaging technology to obtain insights on the long-term disease-modifying potential of CS1 in PAH (Cision) - "Cereno Scientific...today announced that a sub-study of the ongoing CS1 Expanded Access Program (EAP) will be initiated after obtaining all necessary regulatory approvals....The local Institutional Review Board (IRB) has approved the sub-study design, enabling Cereno Scientific to immediately initiate the study....The EAP, an FDA-approved protocol, enables Cereno to obtain valuable long-term safety and efficacy data of CS1 in PAH patients supporting required regulatory interactions and planning future Phase IIb or pivotal Phase III trials."

Trial status • Pulmonary Arterial Hypertension

Effect of CS1 in Subjects With Pulmonary Arterial Hypertension (clinicaltrials.gov) - P2 | N=25 | Completed | Sponsor: Cereno Scientific AB | Recruiting ➔ Completed

Trial completion • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Effect of CS1 in Subjects With Pulmonary Arterial Hypertension (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: Cereno Scientific AB | Trial completion date: Mar 2024 ➔ Sep 2024 | Trial primary completion date: Mar 2024 ➔ Sep 2024

Trial completion date • Trial primary completion date • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Safety and Efficacy of Continued CS1 Treatment of Patients With Pulmonary Arterial Hypertension (clinicaltrials.gov) - P=N/A | N=0 | Available | Sponsor: Cereno Scientific AB

New trial • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

CS1, a controlled-release formulation of valproic acid, for the treatment of patients with pulmonary arterial hypertension: Rationale and design of a Phase 2 clinical trial. (PubMed, Pulm Circ) - "The pharmacokinetic profile of CS1 will also be evaluated. Overall, the novel design and unique, extensive clinical phenotyping of participants in this trial will provide ample evidence to inform the design of any future Phase 3 studies with CS1."

Journal • P2 data • Cardiovascular • Fibrosis • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases