What are rare thrombotic disorders?
Rare thrombotic disorders are a group of uncommon but potentially life-threatening conditions characterized by abnormal blood clot formation in veins or arteries.
Unlike more common clotting events such as deep vein thrombosis (DVT) or pulmonary embolism (PE), these disorders often:
- Occur in unusual locations
- Are linked to genetic or autoimmune mechanisms
- Affect younger or otherwise healthy individuals
- Require specialized diagnostic testing
Examples of rare thrombotic disorders include:
- Antiphospholipid syndrome (APS)
- Heparin-induced thrombocytopenia (HIT)
- Thrombotic thrombocytopenic purpura (TTP)
Why these conditions are especially challenging
Rare thrombotic disorders frequently present with non-specific or atypical symptoms, which can delay diagnosis. Confirming the diagnosis often requires specialized tests, such as antibody testing or enzyme activity measurements. Despite being rare, these conditions can carry a high clinical burden, due to: Sudden or severe clotting events, Risk of recurrence, Potential for serious organ damage and Need for long-term treatment.
Current treatment approaches and limitations
For most rare thrombotic disorders, the standard of care relies on anticoagulant therapies to reduce clot formation. Commonly used treatments include aspirin,w arfarin and other vitamin K antagonists, and direct oral anticoagulants (DOACs) in selected patients. While these therapies can be effective in preventing large-vessel thrombosis, they are often associated with:
- Increased risk of bleeding
- Limited protection against small-vessel (microvascular) clotting
- Incomplete control of the underlying disease mechanisms
As a result, patients may continue to experience disease progression or complications despite ongoing treatment.
Antiphospholipid syndrome (APS) – a key example
Antiphospholipid syndrome (APS) is a systemic autoimmune thrombo-inflammatory disorder characterized by recurrent blood clots and, in some cases, pregnancy complications. APS is associated with the presence of antiphospholipid antibodies, which drive:
- Immune cell activation
- Platelet activation
- Formation of blood clots, particularly in small blood vessels
In rare cases, APS can progress to catastrophic antiphospholipid syndrome (CAPS), a severe and life-threatening condition involving widespread clotting across multiple organs. The estimated global prevalence of APS is around 50 per 100,000 people, with differences observed between males and females.
Unmet medical needs in rare thrombotic disorders
Across rare thrombotic disorders, there remains a significant need for new therapies that:
- Prevent thrombosis effectively and durably
- Reduce the risk of bleeding complications
- Address both large- and small-vessel clotting
- Are suitable for long-term use
A safer and more targeted treatment approach could meaningfully improve outcomes for patients living with these serious conditions.
A HDACi
Drug candidate CS1
An HDAC inhibitor, proprietary formulation of VPA being developed as a well-tolerated oral treatment with favorable safety profile and disease-modifying effects for the rare disease pulmonary arterial hypertension (PAH). A Phase IIa study has been successfully completed and preparations for Phase IIb are underway.
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Second HDACi
Drug candidate CS014
A HDACi, proprietary new chemical entity, employing a multimodal mechanism of action as an epigenetic modulator. A Phase I trial confirmed favorable safety and tolerability. A Phase II trial is planned to start in Q1 2027 evaluating CS014 in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD).
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Cereno Scientific's pipeline is focused on innovative epigenetic modulating HDAC inhibitors.