Join us in our mission to improve the lives of those with autoimmune hepatitis (AIH).

The PORTOLA Phase 2a clinical trial is enrolling people living with autoimmune hepatitis.

Zetomipzomib (KZR-616) is an investigational product and has not been approved for commercial use in the United States or any other country for any indication.

Get Started

Join us in our mission to improve the lives of those with autoimmune hepatitis (AIH).

The PORTOLA Phase 2a clinical trial is enrolling people living with autoimmune hepatitis.

Learn More

Zetomipzomib (KZR-616) is an investigational product and has not been approved for commercial use in the United States or any other country for any indication.

THE PORTOLA CLINICAL TRIAL

PORTOLA is a randomized, double-blind, placebo-controlled phase 2a study with an open-label extension to evaluate the safety and efficacy of zetomipzomib (KZR-616) in people living with autoimmune hepatitis (AIH).

To learn more, scroll down or visit:

ClinicalTrials.gov

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Kezar Life Sciences is conducting the PORTOLA clinical trial to assess zetomipzomib for the treatment of autoimmune hepatitis, its side effects, and its potential effect on reducing liver inflammation or damage.

Autoimmune hepatitis is a rare, chronic disease in which the immune system mistakenly attacks the liver and causes inflammation and tissue damage, severely impacting patients’ physical health and quality of life.¹ This condition affects all ages, genders and ethnicities and requires lifelong maintenance therapy to avoid relapse, usually accompanied by burdensome side effects.¹ If left untreated, AIH can lead to cirrhosis, liver failure, and liver cancer.²
While several medications have been used for the treatment of autoimmune hepatitis, these may only benefit some people. They may also result in serious side effects, including an increased risk of infection.^3,4 Kezar Life Sciences is conducting the PORTOLA clinical trial to determine if zetomipzomib may be safe and effective for the treatment of autoimmune hepatitis.

What to Expect: Step-by-Step Process

After talking with your doctor and selecting a participating physician, you will then go through an informed consent process, in which the investigating physician explains the clinical trial to you in detail, ensures you understand the risks and requirements of participating, and gives you a chance to ask questions.

You will also be given written materials that provide additional clinical trial details to read and consider. If you want to participate and are deemed eligible, you must sign the consent form to give your permission to be included in the clinical trial.

The PALIZADE clinical trial is made up of 3 parts: screening, study treatment, and safety follow up.

A screening visit is conducted by a study coordinator or clinical research nurse to determine if you are eligible to participate in the clinical trial. This includes the informed consent process, medical history and physical examination, vital signs and measurements, laboratory tests, and any necessary additional assessments such as X-rays.
The study treatment will last for 52 weeks (1 year) with site visits occurring about every 4 weeks. At these visits, a study coordinator, clinical research nurse or physician will conduct assessments to determine your response to therapy.
Following the treatment period, there will be 1 follow-up visit to report any side effects or other safety information to the investigator.

There is a 2 in 3 chance of being placed in a patient group receiving zetomipzomib. All patients will be randomized into one of three groups to receive either a 30 mg dose of zetomipzomib, a 60 mg dose of zetomipzomib or placebo through a weekly injection under the skin. During the clinical trial, you will be able to continue taking any other medications that your physician has prescribed for treating your lupus nephritis.

The investigating physician, the hospital staff, and you will not know which treatment you receive. This “blinding” is meant to avoid bias in the clinical trial results.

SCREENING

UP TO 5 WEEKS

1 VISIT

STUDY TREATMENT

52 WEEKS

15-52 VISITS

FOLLOW-UP

4 WEEKS

1 VISIT

There is a 2 in 3 chance of being placed in a patient group receiving zetomipzomib. All patients will be randomized into one of three groups to receive either a 30 mg dose of zetomipzomib, a 60 mg dose of zetomipzomib or placebo through a weekly injection under the skin. During the clinical trial, you will be able to continue taking any other medications that your physician has prescribed for treating your lupus nephritis.

The investigating physician, the hospital staff, and you will not know which treatment you receive. This “blinding” is meant to avoid bias in the clinical trial results.

What to Expect: Step-by-Step Process

After talking with your doctor and selecting a participating clinical research physician, you will then go through an informed consent process, in which the investigating physician explains the clinical trial to you in detail, ensures you understand the potential benefits, risks and requirements of participating, and gives you a chance to ask questions.

You will also be given written materials that provide additional clinical trial details to read and consider. If you decide to participate and are deemed eligible, you will sign the consent form to give your permission to be included in the clinical trial.

The PORTOLA clinical trial is made up of 3 parts: screening, study treatment, and safety follow-up.

Screening

A screening visit is conducted by a study coordinator or clinical research nurse to determine if you are eligible to participate in the clinical trial. This includes the informed consent process, review of your medical history, physical examination, vital signs (blood pressure, temperature, pulse rate, and rate of breathing), laboratory tests, and any necessary additional assessments such as liver biopsy.

Study Treatment

Double-blind Treatment Period:

The double-blind treatment period lasts for 24 weeks.
All participants will be randomized into one of two groups to receive either zetomipzomib or placebo through a weekly injection under the skin.
There is a 2 in 3 chance that you will be placed in a group receiving zetomipzimib and a 1 in 3 chance that you will be assigned to receive placebo
While participating in this clinical study, you will, in most cases, be able to continue taking the same medications that your physician has been prescribing for treating your autoimmune hepatitis, together with your study medication.
During this period, the investigating physician, the hospital staff, and you will not know which treatment you are receiving. This is called “blinding” and is meant to avoid bias in the clinical trial results.

Open-label Extension Treatment Period:

Once the double-blind treatment period is completed, you will have the option to enter into the open-label extension and will be assessed for eligibility.
In this period, all eligible participants who have opted to participate in the open-label extension will receive zetomipzomib once weekly for up to 24 weeks.

Safety Follow-up

Following the treatment period, there will be 1 follow-up visit to report any side effects or other safety information to the investigator.

Who is Eligible?

To be eligible for the clinical trial:

Must be at least 18 years of age
Must have a clinical diagnosis of AIH and signs of active disease despite having received your current medication for ≥ 3 months, or have experienced a disease flare after experiencing a good prior treatment response
Must be willing to reduce the dose of your steroid therapy

Clinical Trial Locations

The PORTOLA clinical trial is being conducted across the United States.

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Participant Accommodations

We aim to make every participant’s PORTOLA clinical trial experience as easy as possible. Accommodations for trial participation vary by clinical trial location and may include the following:

Clinical trial assessments at no cost
Remote home trial services
Travel arrangements such as flights, hotels, taxi, or other travel-related needs for study visits

Man sitting in front of a laptop and smiling.

About Zetomipzomib

Zetomipzomib is a first-in-class, investigational product being studied for the treatment of autoimmune diseases, including autoimmune hepatitis, a rare and chronic autoimmune liver disease.

Zetomipzomib acts by inhibiting the immunoproteasome, a structure found in immune cells that helps to regulate the normal functioning of the immune system.⁵ In autoimmune diseases such as autoimmune hepatitis, the immune system mistakenly attacks and causes damage to the liver and other organs or tissues.

Research has shown that blocking (inhibiting) the immunoproteasome may rebalance the activity of the overactive immune system, and this may have a beneficial therapeutic effect on autoimmune hepatitis.

Zetomipzomib (KZR-616) is an investigational product and has not been approved for commercial use in the United States or any other country for any indication.

About Autoimmune Hepatitis

Autoimmune hepatitis (AIH) is a rare, chronic disease in which the immune system mistakenly attacks the liver and causes inflammation and tissue damage, severely impacting patients’ physical health and quality of life. This condition affects all ages, genders, and ethnicities and requires lifelong maintenance therapy to avoid relapse, usually accompanied by burdensome side effects.¹ If left untreated, AIH can lead to cirrhosis, liver failure, and liver cancer.²

The liver processes an individual’s blood, breaking down the nutrients and chemicals the blood carries. AIH occurs when an individual’s immune system attacks its liver, causing significant inflammation and tissue damage, severely impacting the liver’s function.²
Patients that have other autoimmune conditions have a higher chance of developing another autoimmune disease such as AIH.⁶
AIH affects all genders but affects females 9 times as often as males.⁶
Standard-of-care (SOC) treatment for AIH is long-term use of immunosuppressive medications such as steroids and other immunosuppressive agents like azathioprine.²

While steroids and other immunosuppressive drugs can help manage AIH, these treatments may have life-altering side effects—including osteoporosis, high blood pressure, mood changes, diabetes, and fatigue—and increase an individual’s risk of infection and developing cancers.⁷
Following treatment using current SOC, approximately 35% of patients do not go into remission, and approximately 50-87% of adults who go into remission relapse once treatment is discontinued.⁶
If left untreated, autoimmune hepatitis can result in cirrhosis, liver failure, and liver cancer.⁷ There is a significant unmet medical need for effective AIH treatments that reduce the need for chronic immunosuppression.

RESOURCES

Patient advocacy groups can offer additional educational resources and help you decide if a clinical trial is right for you. Check out these patient advocacy groups for more information and additional resources.

FAQs

About the PORTOLA clinical trial

Why is the PORTOLA clinical trial being conducted?

The PORTOLA clinical trial aims to learn more about zetomipzomib as a potential treatment for people living with autoimmune hepatitis.

Who is conducting the PORTOLA clinical trial?

Kezar Life Sciences is sponsoring the PORTOLA clinical trial. Kezar is a biotechnology company developing potential new treatments for autoimmune diseases and cancer. The company was founded in 2015 and is based in California. For more information, visit www.kezarlifesciences.com.

What ethical considerations are in place for the clinical trial?

Informed Consent: All participants will be provided an informed consent form before participation in the clinical trial. This form will provide information about the risks and benefits of the clinical trial, and a person must agree to participate voluntarily by signing the form if they choose to move forward.
Confidentiality: All information collected from participants will be kept confidential. This means it cannot be shared with anyone outside the clinical trial team without the participant’s permission.
Protection from harm: All participants will be protected from harm. This means the clinical trial team will take steps to minimize the risks associated with the clinical trial.

What safety measures are in place for the clinical trial?

Informed Consent: All participants will be provided an informed consent form before participation in the clinical trial. This form will provide information about the risks and benefits of the clinical trial, and a person must agree to participate voluntarily by signing the form if they choose to move forward.
Data safety monitoring: An independent data monitoring committee (IDMC) is a group of independent experts who review the data collected in a clinical trial on a regular basis. The IDMC is responsible for ensuring that the clinical trial is being conducted safely and that the participants are not subject to undue risk.
Adverse event reporting: All adverse events that occur in a clinical trial must be reported to the clinical trial team. An adverse event is any unwanted or unexpected medical occurrence that happens to a participant during a clinical trial.
Withdrawal from the clinical trial: Participants in a clinical trial have the right to withdraw at any time, for any reason. If a participant withdraws from the clinical trial, they must be followed up with to ensure that they are not at risk of harm.

These safety measures can help researchers ensure that clinical trials are conducted safely and that the data collected is accurate and reliable.

Is there placebo in this clinical trial?

Yes. Since this is a randomized placebo-controlled clinical trial, some participants may receive placebo in addition to the current standard-of-care treatment. A placebo looks identical to the study drug but contains no active ingredients. The purpose of placebo is to evaluate whether simply participating in a clinical trial affects clinical trial outcomes and to make sure the results of each group being studied are handled the same way.

There is a 1 in 3 chance that a participant will receive a placebo in the PORTOLA study during the double-blind treatment period. All participants who have completed the double-blind treatment period are eligible to roll over to the open-label extension period, where all patients will receive zetomipzomib for up to 24 weeks.

What is Kezar’s commitment to diversity in clinical trials?

Kezar-sponsored clinical trials aim to enroll representative numbers of participants from underrepresented racial and ethnic populations globally. Individuals from these populations are frequently underrepresented in biomedical research despite having a disproportionate disease burden for certain diseases relative to their proportional representation in the general population. Adequate representation of these populations in clinical trials helps ensure that the data generated in the development program reflects the racial and ethnic diversity of the population expected to use the medical product, if approved, and may potentially identify effects on safety and efficacy outcomes that may be associated with or occur more frequently within these populations. Kezar Life Sciences is dedicated to having a diverse and patient-centric approach.

Will my personal information be kept private?

The clinical trial team will take steps to protect your privacy, including:

Using pseudonyms or codes to identify you instead of your real name.
Storing your data in a secure location where unauthorized individuals cannot access it.
Requiring researchers to sign confidentiality agreements.

You will also have the right to withdraw from the clinical trial at any time, for any reason. However, data that was collected by your study doctor will be able to be used by the sponsor.

How common is autoimmune hepatitis?

Autoimmune hepatitis affects approximately 140,000 individuals in the United States, with rates of the disease increasing.⁸ Patients that have other autoimmune conditions have a higher chance of developing another autoimmune disease such as AIH. AIH affects all genders but affects females 9 times as often as males.⁶

What causes autoimmune hepatitis?

Immune cells that normally respond to foreign pathogens are mistakenly attacking healthy liver tissue and causing inflammation that damages the liver.

Has zetomipzomib been studied before?

Yes, zetomipzomib was observed to be generally safe and tolerable in preclinical, phase 1, and phase 2 studies. Based on these clinical trials, Kezar is studying zetomipzomib in multiple placebo-controlled clinical trials.

What is an immunoproteasome?

Immunoproteasomes are structures found in immune cells that are important in regulating the immune system. Research has shown that blocking or inhibiting the immunoproteasome may rebalance the activity of the overactive immune system.

About Clinical Trials

What is a clinical trial?

A clinical trial is conducted to evaluate the safety, effectiveness, and side effects of potential new treatments or interventions in humans. Clinical trials are typically conducted in phases, starting with small studies including a limited number of people and then progressively moving to larger controlled studies to determine if an investigational product is safe and effective in treating a medical condition. Clinical trials are essential for advancing medical knowledge and improving patient care.

What are the benefits of participating in a clinical trial?

Access to potential new treatments: Clinical trials offer the opportunity to receive access to new medical treatments or interventions that are not yet available to the general public.
Contributing to medical knowledge: Clinical trial participants can make a significant contribution to medical research by helping to advance our understanding of diseases and how to treat them.
Personal satisfaction: For some people, participating in a clinical trial can provide a sense of empowerment and a feeling of actively contributing to their own health and well-being, as well as to the health of others.

How do clinical trials work?

Video: See How Clinical Trials Work

What is an investigational product?

An investigational product refers to a preventative (vaccine), a therapeutic treatment (drug or biologic), device, or diagnostic tool that is being tested in clinical trials to determine whether it is safe and effective. Investigational products are not yet approved for commercial use outside of clinical trials, and their safety and effectiveness have not yet been fully established.

Who oversees clinical trials?

Several entities oversee clinical trials to ensure that they are conducted ethically, safely, and with the highest possible scientific standards. Clinical trials are conducted with oversight from the United States Food and Drug Administration (FDA) and other regulatory agencies in other countries. Each hospital also maintains an ethics board to oversee the conduct of the clinical trial and the investigating physician, nurses, and healthcare professionals. Finally, an independent data monitoring committee (IDMC) is a group of independent experts who review the data collected in a clinical trial on a regular basis to ensure that the clinical trial is being conducted safely and that the participants are not subject to undue risk.

CONTACT US

If you have any questions or for further information, please complete the form below or contact us at 650-741-7200.

¹https://liverfoundation.org/for-patients/about-the-liver/diseases-of-the-liver/autoimmune-hepatitis/ ²https://www.aihep.org/what-is-autoimmune-hepatitis ³Tesar V, et al. Nephrol Clin Pract. 2014;128:205-21 ⁴Hoover PJ, et al. Kidney Int. 2016;90(3):487-492 ⁵Basler M, et al. Clin Exp Rheumatol. 2015;33(Suppl. 92):S74-S79 | ⁶Mack et al. Hepatology. 2020;72(2):671-722. ⁷https://www.niddk.nih.gov/health-information/liver-disease/autoimmune-hepatitis ⁸Terziroli Beretta-Piccoli et al. Cell Mol Immunol. 2022;19(2):158-176|

Zetomipzomib (KZR-616) is an investigational product and has not been approved for commercial use in the United States or any other country for any indication.

Get Started

Join us in our mission to improve the lives of those with autoimmune hepatitis (AIH).

Join us in our mission to improve the lives of those with autoimmune hepatitis (AIH).

THE PORTOLA CLINICAL TRIAL

What to Expect: Step-by-Step Process

What to Expect: Step-by-Step Process

Who is Eligible?

Clinical Trial Locations

Participant Accommodations

About Zetomipzomib

About Autoimmune Hepatitis

RESOURCES

FAQs

About the PORTOLA clinical trial

About Clinical Trials

CONTACT US

Contact Us