Pharma News: Pulmonary Arterial Hypertension (PAH) Treatments Driving Growth For Many Pharmaceutical Companies

Pharma News: According to data from the pharma industry, the global market for pulmonary arterial hypertension (PAH) treatments is on track to reach nearly US$11 billion by 2030, making PAH drugs highly sought-after assets in the biopharmaceutical industry. While the projected figures for other markets, such as non-alcoholic steatohepatitis and obesity, may be more substantial, the unique appeal of PAH lies in the significant unexplored potential waiting to be tapped. With promising developments on the horizon, biopharma companies are vying to establish their dominance in this rare disease sector.


 
Pulmonary Arterial Hypertension or PAH is classified as a rare disease, with only 500 to 1,000 new cases diagnosed each year in the United States. This specific type of pulmonary hypertension arises when the small arteries in the lungs thicken and narrow, putting additional strain on the heart to pump blood. Over time, this increased demand can lead to fatal heart failure within a few years.
 
In the early stages of PAH, one may not notice any symptoms at all. As the disease progresses, one will start to experience symptoms common to other lung diseases. The most common symptoms of PAH are:
-Increased shortness of breath
-Fatigue
-Edema, or swelling of the feet, legs and eventually the abdomen and neck
-Dizziness and fainting spells
-Chest pain
-Heart palpitations (racing or pounding)
-Lips and fingers turning blue
-Cough
-Hoarseness
 
An estimated prevalence of PH at the population level is approximately 1% to 3%. PAH is rare, with an estimated prevalence of 15 to 30 per million.20,33 The incidence and prevalence of both PH and PHA is increasing, likely for multifactorial reasons.
https://www.ajmc.com/view/pulmonary-arterial-hypertension-updates-in-epidemiology-and-evaluation-of-patients
 
https://www.jacc.org/doi/10.1016/j.jacasi.2022.04.008
 
According to Akash Patel, a healthcare analyst specializing in cardiovascular and metabolic disorders at GlobalData Healthcare, there remains a significant opportunity for pharmaceutical companies to develop therapies that address the underlying cause of PAH rather than merely alleviating its symptoms. He emphasizes the need for disease-modifying treatments that can potentially capture over US$2 billion in market share in the long run, offering substantial revenue for the companies that can establish a stronghold in this niche.
 
One of the key challenges in PAH treatment is the decreased expression of the enzyme prostacyclin synthase in patients, leading to reduced levels of prostacyclin in the blood vessels of the lungs. Consequently, the levels of cAMP, a molecule responsible for dilating blood vessels, also decrease. While existing PAH drugs, such as sildenafil, are PDE5 inhibitors that promote cAMP levels to induce vasodilation, they do not address the root cause of decreased prostacyclin synthase.
 
Patel highlights that these therapies are often administered in combination with prostacyclin receptor agonists, which prevent vasoconstriction and provide symptomatic relief from PAH, such as shortness of breath. However, they do not offer a disease-modifying effect, and the condition continues to progress.
 
A New Wave of Therapies
According to latest Pharma News reports, a potential game-changer in the PAH landscape is Sotatercept, a drug that tackles a root cause of the disease i.e. excessive cell proliferation leading to arterial thickening.
 
Pharmaceutical giant Merck acquired Acceleron Pharma, the developer of sotatercept, for U.S.$11.5 billion in 2021. The drug has shown significant promise in preclinical studies with mice, where blood vessels appeared almost normal after treatment, according to Joerg Koglin, vice president for global clinical development at Merck Cardiovascular.
 
Top-line data from the drug's Phase III trial, reported in October 2022, have generated excitement in the field. PAH trials typically measure improvements in patients' ability to walk six minutes while on treatment. However, sotatercept not only demonstrated significant benefits in this regard but also appeared to slow the progression of clinical symptoms.
 
“This was the first study where patients didn't only slow down their progression, but where actually a substantial proportion of patients improved their progression,” Koglin said. The company is currently conducting a trial to assess whether the drug can reduce deaths and hospitalizations, a feat never achieved with any other PAH drug.
 
The rise of anti-proliferation therapies is ushering in a "new wave" of PAH treatments, according to Tim Noyes, CEO of Aerovate Therapeutics. His company is developing an inhaled version of the cancer drug imatinib as an anti-proliferation therapy for PAH. While anti-proliferation drugs may have more severe side effects than vasodilators, researchers are finding ways to better target these effects. Moreover, combining different anti-proliferation drugs may maximize therapeutic benefits.
 
Noyes envisions a future where, similar to cancer treatment, PAH could be managed using combinations of therapies to reverse or arrest the disease, transforming it into a chronic condition that patients can effectively manage with medication.
 
A Broader Opportunity
Another factor driving interest in PAH research is the potential to address related diseases with similar mechanisms. While PAH itself is rare, other forms of pulmonary hypertension are more common. For instance, pulmonary hypertension can occur as a result of heart failure, which affects a staggering 64 million people worldwide. Additionally, pulmonary hypertension is associated with pulmonary diseases like chronic obstructive pulmonary disease (COPD), which affects over 10% of individuals above the age of 40.
 
Koglin points out that developing drugs that target blood vessel vasoconstriction in PAH may open up new avenues for treating these more common, and consequently more profitable, related diseases. The preclinical data suggest that such drugs could potentially benefit patients with pulmonary hypertension secondary to heart failure.
 
The future of PAH research appears promising, with innovative therapies like sotatercept leading the way in addressing the root causes of the disease. As biopharmaceutical companies strive to develop more effective disease-modifying treatments, the potential to expand into related disease areas further heightens interest in this rare but valuable cardiovascular segment. With a focus on anti-proliferation mechanisms and combination therapies, the PAH landscape is poised to witness transformative changes, offering hope for patients and attractive opportunities for drug developers alike.
 
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