Uncovering New Hope: Advancing Treatment Options for Those Living with Rare Cardiovascular Disease.

The potential for the pulmonary arterial hypertension treatments market is huge, with estimates of nearly $11 billion by 2030 – significantly more than the projected $24 billion non-alcoholic steatohepatitis market by 2028 and the $30 billion obesity market could see by 2030. This sector of the health industry is gaining considerable attention, as the treatment of PAH is quickly becoming a major priority.

In 2021, Merck made a splash in the market, shelling out an impressive $11.5 billion to purchase Acceleron Pharma and their pulmonary arterial hypertension treatment, sotatercept. They aren’t alone in seeking a foothold in this arena though, as Bayer and Pfizer have both taken note of this valuable niche. United Therapeutics further exhibited their interest in 2018, when they paid Arena Pharmaceuticals a hefty sum of $1.2 billion to gain exclusive rights to ralinepag’s late stage PAH candidacy. Clearly, this sector has become increasingly attractive to biopharma—but just what is the big draw?

PAH is an incredibly rare disease, with an estimated 500-1,000 people annually diagnosed in the United States. This specific form of pulmonary hypertension arises when the lung’s small arteries thicken and constrict, forcing the heart to work harder in order to keep up with the increased demand. Unfortunately, this exertion can ultimately lead to heart failure, often within a few short years.

Healthcare analysts have noticed a huge potential opportunity in the cardiovascular and metabolic disorder market, and Akash Patel, an analyst in this field at GlobalData Healthcare, is ready to tap into it. His belief is that, despite its relative modesty compared to similar markets, the vast untapped potential is just waiting to be explored.

The current healthcare landscape provides a significant opportunity for the advancement of new therapies that directly address underlying causes of disease. Such therapies promise to move beyond the simple treatment of symptoms, offering substantial market potential for any pharma companies that can gain competitive advantage in this space – leading to profits of more than $2 billion.

Patients suffering from PAH experience a decreased production of prostacyclin synthase, resulting in lesser amounts of prostacyclin in the lungs’ blood vessels. This, in turn, causes a reduction of cAMP levels, narrowing the vessels and impeding blood flow.

Harnessing the power of the body’s own messenger molecules, PDE5 inhibitors like sildenafil are among the widely-used treatments for Pulmonary Arterial Hypertension (PAH). By raising cAMP levels, which triggers the relaxation of blood vessel muscles, they help to open up the constricted channels and ease the strain, offering much-needed relief.

The treatment options available for pulmonary arterial hypertension (PAH) offer some symptom relief, but many of them fail to address the underlying disease mechanism responsible for decreased prostacyclin synthase. This means that patients continue to suffer from the disease’s progression with no definitive treatment available.

To make matters worse, these treatments are often administered in combination with other medications such as prostacylin receptor agonists, which can lead to further vasoconstriction. Despite this, there remains a substantial market opportunity to create a therapy that actually modifies the course of the disease.

A New Wave of Therapies

Analysts say Sotatercept could be a game-changer in the fight against Pulmonary Arterial Hypertension (PAH) – a disease in which the arterial walls thicken due to cell proliferation. Instead of only promoting vasodilation, the drug hopes to tackle a root cause of the disease. It is predicted to reach sales of $1.4 billion by 2028.

Joerg Koglin, Merck Cardiovascular’s VP of Global Clinical Development, has hailed the supposed anti-proliferation approach of sotatercept – describing in awe how pre-clinical studies on mice showed vessels being practically its normal self for the first time. “I have never seen something like that,” he articulated, expressing his optimism for utilizing this method to reverse the course of the disease.

Excitement has reached fever pitch as of October 2022, with the unveiling of the top-line data from the Phase III trial of the drug.

Sotatercept trials not only proved to significantly improve how far patients on the treatment can walk in six minutes, but further demonstrated its capacity to slow the progression of clinical symptoms.

For the first time ever, a groundbreaking new clinical trial has revealed that a new drug for Pulmonary Arterial Hypertension (PAH) has the potential to not only slow down the progression of the disease, but critically help to actually improve the progression in a substantial number of patients! This is an incredible breakthrough in the treatment of PAH and the company is now investigating if the drug can also reduce deaths and hospitalization associated with the disease.

A new era of treatments for pulmonary arterial hypertension (PAH) is under way, as anti-proliferation therapies make inroads on the condition. Tim Noyes, CEO of Aerovate Therapeutics, which is pushing ahead with the development of an inhaled version of the cancer drug imatinib for PAH patients, has hailed a “new wave” of treatments.

“Interest has been growing in pulmonary arterial hypertension (PAH) since recent studies have uncovered novel anti-proliferative mechanisms, like the one we’ve developed. Many drugs on the market today are only capable of widening blood vessels so far, and the vessels are still more susceptible to getting clogged from the inside out. We believe that our research has the potential to provide an effective solution to this problem.”

Progress in medical research has been stifled by the more-severe side effects of anti-proliferation drugs in comparison to those of vasodilators. However, companies are now pushing forward to create drugs that target the effects more specifically. Moreover, combinations of these drugs could be used together to unlock their maximum potential and create powerful treatments.

Cancer and Alzheimer’s are two of the most devastating diseases of our lifetime, and as of yet there are no known cures. However, Dr. Paul Schulz, studies show there is hope for the future: if we can learn to effectively reverse and manage the symptoms of Alzheimer’s, it could become a chronic, manageable condition much like HIV. We have the power to potentially change the nature of this disease, and with further research, the future could offer renewed hope for those affected by it.

A Broader Opportunity

Koglin highlighted an additional ‘elephant in the room’ concerning PAH research, and this element may also account for the heightened commercial interest in this rare disorder.

Pulmonary hypertension is far from being an uncommon condition – with heart failure affecting over 64 million people worldwide, pulmonary hypertension can arise from this condition as well as pulmonary diseases such as Chronic Obstructive Pulmonary Disease which can affect 10% of people over 40. Although PAH is rare, other forms of pulmonary hypertension are not, highlighting the importance of being aware of the risks and symptoms associated with this condition.

The potential to control blood vessel vasoconstriction in Pulmonary Arterial Hypertension (PAH) by utilizing anti-proliferation drugs opens a remarkable door; one that offers the possibility of providing similar benefits to people suffering from even more commonly encountered related diseases.

Koglin is optimistic about the potential to open up a new space in medicine with the development of a drug that can improve pulmonary hypertension secondary to heart failure. Their preclinical data has strongly supported that the drug should be effective in this regard, making for a thrilling and promising breakthrough.

Leave a Comment