British drugmaker AstraZeneca (AZN 0.01%) and U.S.-based Ionis Pharmaceuticals (IONS 0.38%) have been working jointly on a drug candidate for treating a rare disease known as transthyretin (ATTR) amyloidosis polyneuropathy. The potential medication, eplontersen, recently showed positive results in a phase 3 clinical trial.
What could this mean for AstraZeneca's sales if eplontersen is approved? Let's dig into the global ATTR amyloidosis market to find out.
Another treatment option for a challenging disease
Transthyretin (ATTR) amyloidosis is a rare condition that gets progressively worse. In amyloidosis, abnormal proteins known as amyloids accumulate in the peripheral nerves and organs within the body, which can lead to nerve damage.
ATTR amyloidosis is caused by a protein called transthyretin changing shape into fibrous clumps that are deposited into the organs and peripheral nerves. The condition can either be inherited from a parent, or develop due to aging.
This condition typically affects either the cardiovascular system or the peripheral nerves. When it mostly affects the cardiovascular system, it's referred to as ATTR amyloidosis cardiomyopathy. The symptoms can include fatigue, shortness of breath with exertion, and irregular heartbeat. And when the condition has more impact on the peripheral nerves, it's referred to as ATTR amyloidosis polyneuropathy; this is often characterized by muscle weakness, gastrointestinal issues, and carpal tunnel syndrome.
Early diagnosis and treatment is critical. Without treatment, ATTR amyloidosis polyneuropathy can result in motor disability within five years of diagnosis, and is usually deadly within a decade of diagnosis.
The good news is that pharmaceutical companies like AstraZeneca and Ionis have been hard at work in developing more potent treatments for these conditions. Eplontersen could be one of those drugs that receives approval from regulatory authorities in the not-so-distant future.
The two companies enrolled patients with hereditary ATTN amyloidosis polyneuropathy in a phase 3 clinical trial. Patients were randomized to receive either a monthly self-administered subcutaneous (under the skin) injection of eplontersen, or a placebo injection. At 66 weeks, patients in the treatment group met the primary endpoint of the clinical trial, measuring cranial and motor weakness, reflexes in the muscles, and loss of sensation in the fingers and toes, to gauge how much the disease has progressed.
The specific results of the trial will be presented at the American Academy of Neurology Annual Meeting later this month. But the fact that eplontersen met the primary endpoint of the clinical trial tells us that it helped more patients to stop their condition from worsening compared to placebo. That's a step in the right direction for a difficult-to-treat condition like ATTR amyloidosis polyneuropathy.
Image source: Getty Images.
Solid incremental sales potential
If eplontersen is approved, AstraZeneca and Ionis will split its sales in the U.S. And the former company will develop and commercialize the drug in the rest of the world (besides Latin America) on its own. It's estimated that throughout the world, there are 300,000 to 500,000 patients with ATTR amyloidosis cardiomyopathy and 40,000 patients with ATTR amyloidosis polyneuropathy.
This has attracted other companies into the space, such as Pfizer with Vyndamax and Alnylam Pharmaceuticals with Amvuttra. But since no one treatment can work for every patient, there's room for each drug to succeed. This is why Wall Street estimates that eplontersen could haul in $1.3 billion for AstraZeneca to share with Ionis.
That would boost AstraZeneca's revenue by $700 million. Against the $45.9 billion in revenue that analysts expect from the company in 2023, it would be a modest bump in the top line. And with over 170 other projects in its pipeline, AstraZeneca has plenty of other growth catalysts as well.
AstraZeneca is a growth-oriented stock on sale
Analysts believe that the British drugmaker's earnings will grow by 13% annually over the next five years. For context, that's almost twice the drug manufacturing industry's average earnings growth outlook of 6.8%.
This positions AstraZeneca as a legitimate growth stock -- yet shares of the stock look to be trading at a bargain. AstraZeneca's forward price-to-earnings (P/E) ratio of 17.4 isn't much higher than the drug manufacturing industry's average forward P/E of 14.1. This makes the stock an interesting buy for growth investors.
