Developing novel treatments in cardiovascular diseases
Cardiovascular disease is the number 1 cause of death globally, killing nearly twice as many people as cancer. It is a group of disorders of the heart and blood vessels. An estimated 17.8 million people died from cardiovascular diseases in 2017, a figure estimated to increase to 22.2 million people annually by 2022. Of these deaths, approximately 85% are due to heart attacks and the thrombotic stroke. There are both common and rare diseases among cardiovascular diseases.
Many cardiovascular diseases affect older patients and reduce their quality of life, then indirectly or directly lead to an early death. Unfortunately, existing treatment options are insufficient and the burden on society is high. In the EU, this disease group is estimate to cost €210 billion annually; in the US, $555 billion is estimated annually in 2017.
Cereno Scientific combines an extensive experience in cardiovascular diseases with epigenetic modulation in a novel approach to develop new treatments for patients in need. The aim is to use epigenetic modulating drugs to improve the health of cardiovascular patients, both in common and rare diseases.
Cereno’s focus is to develop novel treatments in cardiovascular diseases that offer better efficacy and fewer side effects compared with today’s available drugs.
Epigenetic modulation, the alteration of gene expression without altering genetic material
Epigenetic modulation is the alteration of gene expression without altering genetic material, in which histone deacetylase (HDAC) inhibition plays an important role. To carry out gene expression, cells must control the coiling and uncoiling of DNA around the terminal tails of core histones. HDAC inhibitors cause epigenetic modulation by blocking the actions that either leads to less compact and more transcriptionally active chromatin or form a condensed and transcriptionally silenced chromatin, resulting in either in the up-regulation or the repression of genes. Epigenetic modulation has in recent years played a critical role in new therapies within oncology, but the use of epigenetic modulation in cardiovascular diseases has just begun.
Around 95% of rare diseases are without approved treatment
There are around 6-8,000 rare diseases, affecting more than 300 million people around the world. Yet around 95% of rare diseases currently have no approved treatment. The definition of what constitutes a rare disease varies; in the US, a rare disease is defined as a condition that affects fewer than 200,000 people while in the EU it is when it affects fewer than 1 in 2,000 people.
Rare diseases became known as orphan diseases because drug companies were not interested in adopting them to develop treatments. In the US, the Orphan Drug Act was established to create financial incentives to encourage companies to develop new drugs for rare diseases.
PAH, pulmonary arterial hypertension
Pulmonary arterial hypertension (PAH) is a rare disease, that is one of five different types of pulmonary hypertension, affecting around 5-15 out of a 100,000 people globally. The disease presents patients with serious unmet needs, being progressive with unsatisfying treatment options. Patients with PAH have a poor prognosis with a 5-year mortality rate of around 30%, and the disease substantially decreases the quality of life of patients.
PAH is characterized by increase in pulmonary pressure is caused by thickening of the walls of the pulmonary arteries (blood vessels leading from the right side of the heart to the lungs) that become thick and stiff, narrowing the space for the blood to flow causing increased blood pressure in the lung, at later stages worsened by locally formed thrombosis. PAH is a serious, progressive disease with a variety of aetiologias that ultimately leads to right heart failure and poor lung function.
Most PAH cases have no specific known cause. The disease has major impact on the individuals functioning, as well as their physical, psychological and social well-being. There is currently no cure for PAH with the exception of lung transplantation, which at that point patients are often in a too precarious state. Today’s medication only improves function and a moderate delay of disease progression, causing the large unmet need for disease-modifying therapy.
Thrombosis, a blood clot forming in deep veins
The majority of cardiovascular disease complications are caused by thrombus formation, where blood clots obstruct the cardiovascular system. This is the reason why the global anti-thrombotic drug market is estimated to grow on average 7.5% annually, resulting in a $43.4 billion market by 2025.
Venous thromboembolism (VTE) is a condition in which a blood clot forms in deep veins before traveling and lodging itself in the lungs. Every year, VTE is diagnosed in 900,000 to 2,100,000 people globally, causing over 800,000 deaths only in the US and Europe. A stroke is caused by a blood clot traveling to the heart or brain. Stroke prevention in atrial fibrillation (SPAF) is a term used to describe stroke prevention in the management of atrial fibrillation (AF), which is defined as arrhythmia that can lead to stroke or other cardiovascular complications. AF is the most common arrhythmia, influencing the lives of approximately 38 million patients globally.
Anticoagulants are commonly used in the treatment of VTE and SPAF, however, major unmet needs remain. More specifically, current blood-thinning treatments involve serious bleeding risks, which calls for new preventive strategies.