Startups Striving to Find a Cure for Chronic Kidney Disease: The Forefront of Treatment Stakeholders Should Know for Investment and Collaboration

(This article was co-written by Wataru Katagiri, Hiroki Takai, and Atsushi Ueda, who invest in the life sciences sector at Global Brain (GB), an independent venture capital firm.)

Did you know that the number of people suffering from chronic kidney disease (CKD) is increasing not only in Japan but around the world due to the growth of super-aged societies and the rise of lifestyle-related diseases?

Although there are treatments for CKD, they only slow the progression of the disease and there is no fundamental cure. This has led to increased medical costs and deteriorated quality of life (QOL) of the patients, posing a serious social issue. Given this background, there is a growing global need for drug development and treatment for CKD, and this area is also attracting attention in startup investment.

In this article, we will explain the latest trends in drug development and new treatments for CKD. By introducing a wide range of topics, from the pharmaceutical companies’ race in developing treatments, to the approaches of advanced treatment by Japanese startups, we hope to drive investment and corporate activities in this field.

Why CKD is attracting attention

CKD is a general term for conditions in which kidney function gradually declines, making it difficult to properly excrete waste products and excess water from the body. Typical diseases include diabetic nephropathy, chronic glomerulonephritis, nephrosclerosis, and polycystic kidney disease. As the disease progresses, dialysis or kidney transplantation may become necessary.

One in five people in Japan has kidney disease

In Japan, approximately one in five adults aged 20 or older is said to have CKD (document only in Japanese), which amounts to about 20 million people. Known as one of the world’s longest-living nations, Japan is facing an increasing number of patients as its population ages.

In addition, lifestyle-related diseases such as diabetes, hypertension, and dyslipidemia also place a significant burden on the kidneys and are risk factors of CKD.

A heavy burden on both the patients and the medical workers

As mentioned at the beginning, there is currently no cure for CKD, and treatment mainly focuses on slowing its progression. Thus, many patients suffer from a decline in their QOL, and healthcare costs across the country continue to rise.

In particular, dialysis treatment for patients whose kidney function has almost completely failed requires time, physical strength, and strict hygiene management, placing significant constraints on all aspects of daily life, including work and family life. Data show that Japan has the second highest number of dialysis patients per capita in the world after Taiwan, which highlights the magnitude of the situation.

There are also challenges in kidney transplantation, which is considered the final treatment option for CKD. The waiting period for receiving a kidney donation from a deceased donor (cadaveric kidney transplantation) is approximately 15 years on average in Japan. This is a significant burden for both patients and medical workers.

Against this backdrop, pharmaceutical companies around the world are focusing on developing new drugs and treatments for kidney disease, and competition is intensifying. GB also sees this sector as a growth market and is paying close attention to it.

Global trends in the treatment of kidney disease

New drug development is being carried out around the world using a variety of approaches. Here are some of the most notable advancements.

1. Repurposing drugs used to lower blood sugar levels

It has been discovered in recent years that a class of drugs originally used to treat diabetes, known as SGLT2 inhibitors, also have the effect of slowing the progression of CKD.

This drug lowers blood sugar levels by excreting excess sugar from the body through urine. Clinical trials (trials in which a considerable number of patients are given a new drug candidate to confirm its efficacy and safety) have also revealed that it has a protective effect on the kidneys.

For example, a drug developed by the British company AstraZeneca has proven to reduce the risk of death caused by the progression of CKD or vascular diseases [1]. Thanks to these outcomes, the drug has been approved by regulatory authorities in the US and Japan to treat CKD.

Likewise, a drug jointly developed by the German company Boehringer Ingelheim and the US company Eli Lilly has also been proven to slow the progression of CKD [2] and was also approved as a new treatment in Japan in February 2024.

2. Focusing on specific kidney diseases

There is a rare kidney disease called IgA nephropathy. This disease occurs when IgA, an immune substance that protects the body, accumulates in the kidneys, causing inflammation and impairing kidney function. Active research is also underway to develop treatments for this disease.

Budesonide, an oral medication developed by the Swedish company Calliditas Therapeutics and the US company Viatris, is a steroid drug designed to work in the intestines and is expected to slow the progression of IgA nephropathy. It was approved in the US in 2023 as a new drug for CKD.

In Japan, Viatris is currently conducting clinical trials (Japanese only), and the drug is eagerly awaited to be a new treatment option.

The competition to develop treatments for IgA nephropathy is particularly fierce, with companies actively seeking to obtain other companies’ technologies and drugs that are under development through acquisitions.

In Japan, Otsuka Pharmaceutical acquired the US company, Visterra, gaining a pipeline of drugs for the treatment of IgA nephropathy. Asahi Kasei Pharma also entered the IgA nephropathy market by making the aforementioned Calliditas Therapeutics a wholly owned subsidiary.

Overseas, Novartis and Vertex Pharmaceuticals are moving forward with corporate acquisitions.

3. Focusing on complement system, which plays a role in regulating the body’s defense system

Our blood contains a group of proteins known as the complement system, which plays an important role in regulating the body’s immune defences. New medication based on the idea of suppressing the progression of kidney diseases by regulating the function of complement are also appearing.

A drug called Iptacopan developed by Novartis of Switzerland, targets and inhibits the function of complement system, demonstrating efficacy against several rare kidney diseases.

In the US, it has already been approved as a treatment for multiple kidney diseases, including paroxysmal nocturnal hemoglobinuria, IgA nephropathy, and C3 nephropathy. In Japan, it is also starting to be used as a treatment for certain conditions, including paroxysmal nocturnal hemoglobinuria.

Although new drug development is being pursued through various approaches, the development of new drugs and large-scale M&A are not easy to achieve. There are cases where newly licensed drugs fail to obtain approval or suffer poor sales.

Furthermore, considering that lawsuits have been filed in connection with failed clinical trials, we must keep in mind that drug development involves significant risks.

Japanese startups taking on the challenge of developing groundbreaking treatments

Research and development in kidney disease treatment is also progressing in Japan. Here are two remarkable startups that GB is supporting.

Rege Nephro taking on kidney diseases with iPS cells

Rege Nephro is a startup that is developing two treatment methods for kidney disease based on the research results of Dr. Kenji Osafune, Deputy Director of the Center for iPS Cell Research and Application, Kyoto University. The company is now taking on the challenge of developing two new treatment options for kidney disease.

One is the development of a new drug for polycystic kidney disease, a hereditary kidney disease. Using iPS cells (pluripotent stem cells that can differentiate into various types of cells), researchers are working to reproduce the condition of the disease and identify safer and more effective drug candidates, a process that has already advanced a new drug into clinical trials.

The other is regenerative medicine, which involves using iPS cells to create kidney cells and transplant them into patients to treat CKD. If successful, this could be the world’s first breakthrough for treating CKD, a condition for which no fundamental cure has been available.

GB also views Rege Nephro as an innovative and challenging startup, and has decided to invest in it through its 321FORCE™, the CVC fund jointly established with Mitsui Chemicals.

PorMedTec finds a way for pig kidneys to save humans

PorMedTec is a startup spun out of Meiji University in Japan and is developing a treatment for kidney disease using medical-grade pigs raised under a highly controlled environment. This technique is called xenotransplantation, which is an attempt to transplant animal organs into humans.

In the US, genetically modified pig kidneys have already been successfully transplanted into humans. In March 2024, Dr. Tatsuo Kawai performed the world’s first pig kidney transplant into a living person at Massachusetts General Hospital.

Unfortunately, the patient died two months after the surgery, but according to the hospital, kidney failure was not the direct cause of the death and it is unlikely that the death was related to the transplant. And in January 2025, the second patient who underwent transplantation at Massachusetts General Hospital was discharged and is being monitored at home.

In collaboration with US-based eGenesis, PorMedTec successfully created genetically modified pigs for xenotransplantation in Japan for the first time. Further research and development is conducted for clinical application.

GB is supporting PorMedTec’s challenge that has the potential to save many patients that suffer from kidney disease through its JGC MIRAI Innovation Fund, the CVC fund jointly established with JGC.

How to accurately evaluate the technology for developing treatments

Finally, here are the points to focus on when evaluating startups that are developing treatments for CKD.

Even if new drugs or treatment options are discovered, they will not become immediately available to everyone. It requires time to carefully verify their effectiveness and, above all, their safety.

For this reason, in the case of CKD, the following items are the criteria for evaluation in clinical trials.

• Values that show the level of kidney function

  • Example: eGFR (estimated glomerular filtration rate), a measure of how well the kidneys are filtering blood

• Values that indicate the extent of damage to the kidneys

  • Example: UPCR (Urine Protein Creatinine Ratio) and UACR (Urine Albumin Creatinine Ratio), which measure the amount of protein and waste products in urine

• The percentage of patients who have progressed to a condition requiring dialysis or kidney transplantation (end-stage renal failure)

There are several points to note when verifying the figures. Careful judgement is required as eGFR may temporarily decrease after the initiation of certain medications, such as SGLT2 inhibitors, and may also decline with age even in healthy individuals. Additionally, different indicators are used for drugs that directly target conditions such as sudden nocturnal hemoglobinuria or polycystic kidney disease. It is advisable to evaluate the criteria while confirming how the startups are approaching kidney disease. There are cases where drugs are rapidly approved based on a reduction in protein levels in urine, and promising new treatments are highly anticipated to quickly reach patients.

Summary

As explained so far, there is still no cure for CKD and there are many challenges including the long waiting period for transplants.

To respond to these challenges, pharmaceutical companies around the world are actively pursuing new approaches to develop innovative drugs and treatments. Startups that are exploring regenerative medicine using iPS cells and kidney transplants using medical-grade pigs are emerging in Japan as well.

The field of kidney disease treatment will continue to grow as it is expected to deliver both substantial social impact by greatly improving people’s lives and strong financial returns.

At GB, we remain committed to supporting startups tackling the significant challenge of CKD and strive to bring a brighter future to as many patients as possible.

The information provided is current at the time of publication.
(Written by Wataru Katagiri, Hiroki Takai, and Atsushi Ueda, edited by the Universe Editorial Team)

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1. Heerspink HJL, Stefánsson BV, Correa-Rotter R, et al. Dapagliflozin in patients with chronic kidney disease. N Engl J Med 2020;383:1436-1446. ↩︎

2. The EMPA-KIDNEY Collaborative Group. Empagliflozin in patients with chronic kidney disease. N Engl J Med 2023;388:117-127. ↩︎