Cereno Scientific Receives FDA Clearance to Initiate Global Phase IIb Trial of CS1 in Pulmonary Arterial Hypertension (PAH)

Rhea-AI Summary

Cereno Scientific (B) announced that the U.S. FDA cleared initiation of its global Phase IIb trial of lead candidate CS1 for pulmonary arterial hypertension (PAH).

The randomized, double-blind, placebo-controlled, dose-finding study will enroll ~126 patients across ~65 sites in 10–12 countries, run 60 weeks total, and evaluate PVR by right-heart catheterization and 6-minute walk distance at Week 36. CS1 holds Orphan Drug and Fast Track designations. First patient in is planned for Q2 2026, with top-line data anticipated around Q4 2028 subject to enrollment timelines.

Positive

• FDA clearance granted to start Phase IIb
• Orphan Drug and Fast Track designations in U.S.
• Global enrollment planned: ~126 patients across 10–12 countries
• Primary endpoint: PVR measured by right-heart catheterization at Week 36

Negative

• Top-line data anticipated only around Q4 2028
• Readout timing subject to enrollment and possible delays
• Relatively small sample size: ~126 patients

News Market Reaction

+2.22%

1 alert

+2.22% News Effect

On the day this news was published, B gained 2.22%, reflecting a moderate positive market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Planned FPI: Q2 2026 Top-line data timing: Around Q4 2028 Phase IIb sample size: Approximately 126 patients +5 more

8 metrics

Planned FPI Q2 2026 First patient in for global Phase IIb CS1 PAH trial

Top-line data timing Around Q4 2028 Anticipated top-line CS1 Phase IIb readout, enrollment-dependent

Phase IIb sample size Approximately 126 patients Global multicenter CS1 PAH dose-finding trial

Initial treatment period 36 weeks Randomized CS1 vs placebo period before re-randomization

Total study duration 60 weeks Includes screening, treatment periods, and follow-up

Investigative sites Approximately 65 sites Planned global Phase IIb CS1 PAH trial footprint

Participating regions 10–12 countries U.S., Europe, and South America in CS1 trial

Trial phase Phase IIb Dose-finding study of CS1 in pulmonary arterial hypertension

Market Reality Check

Price: $48.73 Vol: Volume 7,468,472 is well ...

low vol

$48.73 Last Close

Volume Volume 7,468,472 is well below 20-day average of 19,342,149 (relative volume 0.39). low

Technical Price 40.02 is trading above 200-day MA at 32.57, indicating a pre-news uptrend.

Peers on Argus

While B is up 0.32%, key gold peers like WPM, FNV, AU, AEM, and KGC are down bet...

While B is up 0.32%, key gold peers like WPM, FNV, AU, AEM, and KGC are down between about 2–3%, pointing to stock-specific factors rather than a sector move.

Historical Context

5 past events · Latest: Dec 03 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 03	Product launch	Positive	-1.3%	Launch of ETQ Reliance Go cloud-native EQMS for smaller manufacturers.
Dec 02	Asset divestiture	Positive	-3.1%	Sale of Tongon mine and exploration assets for consideration up to $305M.
Dec 01	IPO evaluation	Positive	+2.4%	Board authorizes evaluation of IPO for North American gold assets subsidiary.
Nov 26	Asset divestiture	Positive	+4.8%	Completion of Hemlo mine sale with up to $1.09B in structured proceeds.
Nov 26	Board changes	Neutral	+4.8%	Lead Independent Director change and reaffirmation of strategic priorities.

Pattern Detected

Recent news has triggered mixed reactions: some strategic and portfolio updates saw gains, while other seemingly positive launches and divestitures coincided with declines.

Recent Company History

Over late November–December 2025, B’s news flow centered on portfolio reshaping and strategic initiatives. It completed divestitures of Tongon and Hemlo with structured consideration packages, explored an IPO of North American gold assets, and announced board leadership changes. A product launch via ETQ Reliance Go on December 3, 2025 drew a negative reaction. Against this backdrop of active portfolio management and governance updates, today’s FDA-related clinical news fits into a broader pattern of value-focused repositioning, though in a different industry context.

Market Pulse Summary

This announcement details FDA clearance for a global Phase IIb trial of CS1 in PAH, with around 126 ...

Analysis

This announcement details FDA clearance for a global Phase IIb trial of CS1 in PAH, with around 126 patients, a 36-week primary treatment period, and total duration of 60 weeks. It outlines key endpoints such as pulmonary vascular resistance and 6-minute walk distance, plus global execution across 10–12 countries and roughly 65 sites. Investors may watch enrollment progress, safety signals, and adherence to the Q2 2026 FPI and Q4 2028 top-line timelines.

Key Terms

pulmonary arterial hypertension, orphan drug designation, fast track designation, standard of care, +4 more

8 terms

pulmonary arterial hypertension medical

"lead drug candidate CS1 for the treatment of pulmonary arterial hypertension (PAH)."

Pulmonary arterial hypertension is a progressive medical condition in which the arteries that carry blood from the heart to the lungs become narrowed or stiff, causing high pressure in the lung circulation and extra strain on the heart — like a pump working against clogged pipes. For investors, it matters because the condition defines the need, market size, clinical trial design, regulatory hurdles and potential revenue or risk for companies developing drugs, devices or diagnostics to treat or manage it.

orphan drug designation regulatory

"CS1 has also been granted Orphan Drug Designation and Fast Track designation in the U.S."

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

fast track designation regulatory

"CS1 has also been granted Orphan Drug Designation and Fast Track designation in the U.S."

A "fast track designation" is a process that speeds up the review and approval of a product or project, allowing it to reach the market or be completed more quickly than usual. For investors, it can signal that a product may become available sooner, potentially leading to earlier revenue or benefits, and indicating a priority status that might influence company performance and market opportunities.

standard of care medical

"versus Placebo When Added to Standard of Care for the Treatment of Pulmonary"

Standard of care is the accepted medical treatment or clinical approach that most qualified doctors would use for a given condition today, based on available evidence, guidelines and common practice. For investors, it acts like the baseline product customers expect: a new therapy or device must match or improve on the standard of care to win market share, gain reimbursement and limit legal or regulatory risk.

right-heart catheterization medical

"effect of CS1 on pulmonary vascular resistance (PVR) at Week 36 via right-heart catheterization,"

A right-heart catheterization is a medical procedure in which a thin tube is threaded into the right side of the heart and nearby lung vessels to directly measure blood pressures and oxygen levels, like inserting a gauge into plumbing to check flow and pressure. For investors, these measurements are often key evidence in clinical trials, regulatory approvals, treatment decisions and reimbursement discussions, because they provide objective data on how a drug or device affects heart and lung function.

biomarker medical

"including measures of heart function, biomarker changes, clinical worsening,"

A biomarker is a measurable indicator found in the body, such as in blood or tissues, that provides information about health, disease, or how the body responds to treatment. For investors, biomarkers can signal the potential success or risk of medical products or therapies, influencing the value of related companies and industry trends. They act like signals or clues that help assess the progress of medical advancements and their market impact.

patient-reported outcomes medical

"clinical worsening, patient-reported outcomes, and pharmacokinetics."

Reports provided directly by patients about their symptoms, daily functioning, and quality of life—collected through surveys, apps, or interviews—reflecting how a treatment affects real people rather than lab measures. Investors care because these firsthand accounts help regulators, doctors and payers judge a product’s real-world value and can influence approval, pricing, adoption and long-term sales; think of them as customer reviews that show whether a medical product truly improves everyday life.

pharmacokinetics medical

"patient-reported outcomes, and pharmacokinetics. This dose-finding trial is expected"

Pharmacokinetics is the study of how a substance, such as a drug or chemical, moves through and is processed by the body over time. It tracks how it is absorbed, distributed, broken down, and eventually eliminated. For investors, understanding pharmacokinetics helps gauge the effectiveness, safety, and potential risks of new medications or treatments, which can influence a company’s success and valuation in the healthcare industry.

AI-generated analysis. Not financial advice.

GOTHENBURG, Sweden, Dec. 8, 2025 /PRNewswire/ -- 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 the U.S. Food and Drug Administration (FDA) has granted clearance to initiate the company's Phase IIb trial of its lead drug candidate CS1 for the treatment of pulmonary arterial hypertension (PAH). 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. The clearance follows constructive regulatory interactions and builds on the favorable safety, tolerability and encouraging disease-modifying signals observed in the Phase IIa study. CS1 has also been granted Orphan Drug Designation and Fast Track designation in the U.S.

"Our goal with CS1 is to address the underlying mechanisms that drive PAH, not simply manage symptoms," said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific. "The planned Phase IIb trial is designed to determine the optimal dose for a Phase III trial and to assess CS1's potential to meaningfully reduce pulmonary vascular resistance and improve functional capacity when added to today's standard therapies. Building on the encouraging signals observed in our Phase IIa study, we believe CS1 could offer a differentiated and disease modifying approach for patients living with this rare and fatal disease."

The Phase IIb trial is formally titled "A Phase 2b, Double-Blind, Randomized, Placebo-Controlled, Dose-Finding Study, to Compare the Efficacy and Safety/Tolerability of CS1 Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH)." It is a global, multicenter trial enrolling approximately 126 patients with PAH who are stable on background therapy. During the first 36 weeks of treatment, participants will be randomized to receive once-daily CS1 capsules at one of two dose levels or matching placebo. At Week 36, all participants will be re-randomized: those initially receiving CS1 will be assigned either to continue on their CS1 dose or switch to placebo, while those initially receiving placebo will be assigned to one of the two CS1 dose groups. This design ensures that all participants will receive active treatment at some point of the trial and allows CS1's previously observed disease-modifying signals to be further evaluated in a larger, controlled trial. All participants will then continue in a second treatment period and follow-up. The total study duration is 60 weeks, including screening and follow-up. The study will evaluate the effect of CS1 on pulmonary vascular resistance (PVR) at Week 36 via right-heart catheterization, changes in 6-minute walk distance at Week 36, and a range of additional evaluations including measures of heart function, biomarker changes, clinical worsening, patient-reported outcomes, and pharmacokinetics. This dose-finding trial is expected to be conducted across 10–12 countries in the U.S., Europe and South America at approximately 65 investigative sites.

"Receiving FDA clearance to proceed with our Phase IIb study is a significant catalyst for Cereno," said Sten R. Sörensen, CEO at Cereno Scientific. "This milestone strengthens the commercial potential of CS1, supports our positioning as a leader in epigenetic modulation for rare cardiovascular diseases, and creates additional momentum in our partnering discussions. With Fast Track designation and global site participation, we see clear opportunities to accelerate our path toward a potential disease-modifying therapy that can enhance and extend the lives of patients with PAH."

Cereno Scientific is now advancing global study-start activities, including site selection, regulatory submissions in participating countries, and operational readiness with its selected CRO partner. First patient in (FPI) is planned for Q2 2026, with top-line results anticipated around Q4 2028, subject to enrollment timelines. These activities mark the start of the largest and most comprehensive clinical program undertaken by the company to date.

For further information, please contact:

Tove Bergenholt, Head of IR & Communications
Email:  tove.bergenholt@cerenoscientific.com
Phone: +46 73- 236 62 46

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 22:25 (CET) on December 8, 2025.

About PAH

PAH is a rare, progressive and life-threatening disease characterized by high blood pressure in the pulmonary arteries that leads to right heart failure and premature death. Current standard treatments mainly focus on managing symptoms, leaving a significant unmet need for disease-modifying therapies that can change the course of disease and improve long-term outcomes.

About CS1

CS1 is an orally administered histone deacetylase inhibitor (HDACi) in development as a well-tolerated, disease-modifying therapy for pulmonary arterial hypertension (PAH) with favorable safety profile. Acting through epigenetic modulation, CS1 targets key disease-driving mechanisms such as vascular remodeling, fibrosis and inflammation. CS1 has shown disease-modifying potential in early clinical evaluation and is being evaluated as an add-on (on top of standard-of-care) therapy with the potential to improve outcomes for patients with high unmet medical needs. It has been granted Orphan Drug Designation (ODD) in both the U.S. and the EU and received Fast Track designation from the U.S. FDA in August 2025, underscoring its potential to address a serious condition with high unmet medical need.

CS1 has first-in-class potential and is currently in Phase II clinical development.

About Cereno Scientific AB

Cereno Scientific is pioneering treatments to enhance and extend life. The company's innovative pipeline offers disease-modifying drug candidates to empower people suffering from rare cardiovascular and pulmonary diseases to live life to the fullest.

Lead candidate CS1 is an HDAC inhibitor that works through epigenetic modulation and represents a novel therapeutic approach by targeting the root mechanisms of the pulmonary arterial hypertension (PAH). CS1 is a well-tolerated oral therapy with a favorable safety profile that has shown encouraging efficacy signals of reverse vascular remodeling, improvement of right heart function and enhanced patient quality of life in a Phase IIa trial in patients with PAH. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1. CS014, a new chemical entity with disease-modifying potential, showed favorable safety and tolerability profile in a Phase I trial. CS014 is a HDAC inhibitor with a multimodal mechanism of action as an epigenetic modulator having the potential to address the underlying pathophysiology of rare cardiovascular and pulmonary diseases with high unmet needs such as idiopathic pulmonary fibrosis (IPF). Cereno Scientific is also pursuing a preclinical program with CS585, an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular diseases. While CS585 has not yet been assigned a specific indication for clinical development, preclinical data indicates that it could potentially be used in indications like thrombosis prevention without increased risk of bleeding.

The Company is headquartered in GoCo Health Innovation City, in Gothenburg, Sweden, and has a US subsidiary; Cereno Scientific Inc. based in Kendall Square, Boston, Massachusetts, US. Cereno Scientific is listed on the Nasdaq First North (CRNO B). The Company's Certified Adviser is DNB Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.

This information was brought to you by Cision http://news.cision.com

https://news.cision.com/cereno-scientific/r/cereno-scientific-receives-fda-clearance-to-initiate-global-phase-iib-trial-of-cs1-in-pulmonary-arte,c4278561

The following files are available for download:

https://mb.cision.com/Main/14131/4278561/3831825.pdf

Press release

View original content:https://www.prnewswire.com/news-releases/cereno-scientific-receives-fda-clearance-to-initiate-global-phase-iib-trial-of-cs1-in-pulmonary-arterial-hypertension-pah-302635813.html

SOURCE Cereno Scientific

FAQ

What did Cereno Scientific (B) announce about CS1 on December 8, 2025?

The company said the FDA cleared initiation of a global Phase IIb trial of CS1 for PAH.

When is first patient in (FPI) planned for Cereno's CS1 Phase IIb trial (B)?

First patient in is planned for Q2 2026.

When does Cereno expect top-line results for the CS1 Phase IIb trial (B)?

Top-line data are anticipated around Q4 2028, subject to enrollment timelines.

How many patients and countries will Cereno's CS1 Phase IIb study (B) enroll and cover?

The trial will enroll approximately 126 patients across about 10–12 countries at ~65 sites.

What are the primary efficacy assessments in Cereno's CS1 Phase IIb trial (B)?

Primary assessments include PVR by right-heart catheterization and change in 6-minute walk distance at Week 36.

Does CS1 have any special regulatory designations in the U.S. for PAH (B)?

Yes. CS1 has received both Orphan Drug designation and Fast Track designation in the U.S.