This is my take ... Understand PKD and its Prevalence
Polycystic kidney disease (ADPKD) is an autosomal dominant disorder. It's a multisystem and progressive disease with cysts formation and kidney enlargement along with other organ involvement (e.g., liver, pancreas, spleen).
In the adult population, it is the most frequent genetic cause of renal failure. By the age of 60 years, 50% of them need renal replacement therapy. Cysts may be detected in childhood or in utero, but clinical manifestations appear in the third or fourth decade of life. [1]
PKD affects about 500,000 people in the U.S. [2]
Current PKD Treatment Options
Dialysis is a major treatment option for individuals diagnosed with end-stage renal disease (ESRD) such as PKD. It can be an incredibly costly procedure. In-centre Hemodialysis (HD) would be US$72,000 - $88,000 annually. Peritoneal Dialysis (PD) at home is less expansive at US$53,000 - $65,000. [3]
Dialysis is normally 3 times/week with each session lasting 3-4 hours. Some may be able to do dialysis at home daily using Peritoneal Dialysis (PD) but each session would be 10-12 hours. Dialysis does impact greatly on the quality of life of each PKD patient from around 30-40 years old which would be their golden age of their working life.
A better option is to have a Kidney Transplant if a compatible donor organ is available. One might have to go on dialysis for years until a match is found. In 2020, the average kidney transplant cost was US$442,500. [4]
Most people wait three to five years for a kidney from the national transplant waiting list in the United States. The typical waiting time in Australia.is 4 years. [5] [6]
PKD represents an addressable market of >US$10 billion p.a.? Let's check!
From PYC’s news release on 13.11.2023: “PKD affects 1 in every 1,000 people across the globe. There are currently no drugs available that address the underlying cause of the disease and approximately 50% of PKD patients will progress to end-stage renal failure by the age of 60.”
The US population in 2023 is 340,684,307. 1 in 1,000 prevalence means 340,684 PKD sufferers and 50% of them is 190,342. They would eventually have renal failure requiring dialysis or kidney transplant. PYC’s projected income of US$10b = US$52.5K per patient per year. The drug is 40% lower than the cost of dialysis at US$88,000 and 88% cheaper than a kidney transplant at US$442,500. Besides it is comforting to know that the drug could "arrest the course of the disease, enabling damaged kidneys to regenerate and restore function" according to the article.
With the other 50% of the PKD population, some would opt for the PYC drug well BEFORE their kidney fail. Therefore it is logical for PYC to estimate the income derived from the drug could be as high as US$15b. Obviously, PYC’s projected income figures are for global sales. The above calculations are only based on US data because of their abundance and accuracy to gauge if PYC’s projection is realistic or not.
The Global Market
When you add Northern America’s population of 378M, to Europe’s 742M, China’s 1.425 billion, and India’s 1.433M; [7] you are looking at 4 billion people. 2M would need PYC’s drug and divide into the projected income of US$10b p.a., that’s US$500 per patient. Even if only 10% of them received PYC treatment, it implies an ultra low cost of US$5,000 per head. I am sure their projected income is very conservative. If they price it on $10,000 per head or the uptake increases to 20%, annual income would grow to US$20-30b and so on.
For the developed countries, the question is: Would anyone still opt for dialysis instead of taking the PYC drug?
A Real Life Story of a PKD Patient
Today I interviewed a 45 y.o. PKD patient who received a kidney transplant 6 years ago. She said missing from work 3 half days a week to go to dialysis was destroying her career. Later she changed to Peritoneal Dialysis (PD) at home every night for 10-12 hours. With a day job for 8 hours and 1 hour transit to/from work, she only had 3 hour a day left to cook dinner and raise her kid. Sure she regained full employment but not much of quality of life to be had. After the transplant, she is now on a life-long cocktail of anti-rejection drugs and constant worries on her blood test results to monitor the organ’s rejection level. Her transplanted kidney is NOT a done and forget business. She is absolutely delighted to learn of PYC’s new drug for PKD. Because her teenage daughter is running a very high chance of getting PKD as well. It’s a psychological baggage on her shoulder and a ticking time bomb for her daughter. PYC-003 would change their lives! Bring it on !
(20min delay)
