XBI - EXCLUSIVE: Kidney Care Revolution? How Swedish Biopharma Calliditas Aims To Tackle A Rare Disease With 'Unique But Pretty Simple' Drug | Benzinga
Swedish biopharma Calliditas Therapeutics AB (NASDAQ:CALT) is making significant strides in addressing rare kidney diseases. In a recent interview with Benzinga, the company’s CEO, Renee Aguiar-Lucander, provided valuable insights on various aspects of its work.
Tarpeyo – A Unique Solution?
Aguiar-Lucander described Tarpeyo, its lead drug, as “beautiful, unique, but pretty simple,” adding that it has received accelerated FDA approval for treating IgA nephropathy, an autoimmune kidney disorder that results from the accumulation of antibodies in the organs. This in turn causes inflammation and kidney damage.
The Gut-Kidney Connection
Although IgA nephropathy is a kidney disease, its origin is in the gut, which houses much of the human immune system. The exact origin is in the portion called the ileum, which has a huge concentration of Peyer's patches, which are like lymphatic tissues. These have beta cells that secrete IgA1, which per se is not harmful.
A trigger, possibly bacterial, dietary, environmental, etc., can cause a sudden overproduction or over-proliferation or even leaking of these IgA antibodies, which then find their way into the blood in large numbers, triggering autoimmune reactions by the body and the release of other antibodies. These large autoimmune complexes won't be cleared by the liver and continue to circulate until they end up in the kidney, causing inflammation, fibrosis, necrosis, etc.
It takes a while for the progressive autoimmune disease to impact the kidney's performance. Ultimately, the affected party has a high risk of ending up with end-stage renal disease, and for some, it might take 10 years or even up to 20 years for a precipitation into end-stage disease. It might leave one who is only 40 years old and otherwise healthy at the mercy of dialysis for a lifetime.
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Tarpeyo's Mode Of Action
With Tarpeyo, Calliditas focuses on the origin of the disease, and the drug is delivered to the gut. The capsule formulation of the drug is coated in such a way that it remains intact until it reaches the ileum. It then releases the active ingredient called budesonide over a very short time of an hour or two.
Budesonide is a locally-acting corticosteroid that is similar to the one used for asthma, which when inhaled, treats the local mucosa in the lungs. Tarpeyo treats the local mucosa in the gut. Aguiar-Lucander stressed that this approach is far safer than administering high doses of systemic steroids.
Source: Calliditas
Flexible Treatment Duration
When discussing the treatment duration, Aguiar-Lucander mentioned that clinical trials usually administered Tarpeyo for nine months. However, in real-world scenarios, the duration varies based on individual patient profiles. She specified that ...