Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

Inclusion Criteria:

All Patients:

1. Patient is able to communicate well with the investigator, understands and iswilling to comply with all requirements of the study, and understands and signs thewritten informed consent form (ICF).

2. For children to be eligible, one or both parents/legal guardians must sign aparental permission form which provides information contained in the ICF. Childrencapable of assent must express their willingness to participate by signing an assentform.

3. If patient has received a COVID vaccination, the baseline visit must occur at leastone week or more after the second/booster vaccination.

4. Patients who have had active symptoms of COVID within 3 months prior to screeningand are now asymptomatic for the last 2 weeks but have tested COVID PCR positive. Ifa patient is asymptomatic at screening but is COVID positive, then rescreening canoccur after a minimum of two weeks.

5. Both female patients, as well as, female partners of male patients who are ofchild-bearing potential must be willing to not become pregnant for the completeduration of the study (>180 days) (90 days after the last dose of study medication).

6. Males (including sterilized subjects) whose female partners have child-bearingpotential, must agree to use male contraception (condoms) during the period from thetime of signing the informed consent form (ICF) through 90 days after the last doseof study drug. They must agree to immediately inform the investigator if theirpartner becomes pregnant during the study. Alport Syndrome Patients Inclusion Criteria (in addition):

7. Males and females with X-Linked AS and males and females with autosomal inheritedAS.

8. For countries that are enrolling pediatric patients: patients from age 12 yearsand older.

9. For countries that are not enrolling pediatric patients: patients from age 18years and older.

10. Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For patientsenrolled in the US who meet all inclusion and exclusion criteria but have not hadtheir diagnosis confirmed by genetic testing or kidney biopsy, the Sponsor willprovide for patient's genetic testing.

11. UPCR ≥1.0 g/g.

12. eGFR ≥ 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartzequation for children).

13. ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior toenrollment and during the study. Focal Segmental Glomerulosclerosis Patients Inclusion Criteria (in addition):

14. Male or female patients,

15. For countries that are enrolling pediatric patients: 12 to 75 years old at thetime of signing the informed consent

16. For countries that are not enrolling pediatric patients: 18 to 75 years old atthe time of signing the informed consent

17. Primary FSGS, (without any identifiable cause, and where the FSGS is confirmed byrenal biopsy) or FSGS where there is documentation of a genetic mutation in apodocyte protein associated with FSGS.

18. If on steroids, the dose should remain stable for at least 4 weeks prior toenrollment and during the study. Subjects who are steroid-resistant, defined asfailure to achieve partial or complete remission, or subjects who experiencedadverse events without acceptable clinical benefit after at least 8 weeks ofadequate corticosteroid therapy for children and 12 weeks for adults are eligible.

19. UPCR between 1.5g/g and 12.0g/g.

20. eGFR > 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartzequation for children).

21. If taking concomitant ACEi and/or ARB treatment, it should remain at a stable dosefor at least 4 weeks prior to enrollment and during the study.

Exclusion Criteria:

All Patients:

1. Uncontrolled diabetes mellitus as evidenced by an HbA1c ≥ 11%. For Germany: HbA1c ≥ 8.5%.

2. Uncontrolled hypertension

3. Adults: (SBP ≥ 180mmHg and/or DBP ≥ 100mmHg). For Germany: (SBP ≥ 140mmHgand/or DBP ≥ 100mmHg).

4. Children: ≥ 95th percentile or ≥ 130/80 mm Hg, whichever is lower

5. Moderate or severe hepatic impairment (Child-Pugh B or C), except if (a) decreasedserum albumin is directly related to the renal disease (resulting in a Child Pughscore of 7), and (b) no other Child-Pugh Score parameters are increased and (c)patient has no liver pathology in medical history.

6. Presence of any active (i.e., with symptoms) and/or uncontrolled infection (including COVID).

7. Presence of Human immunodeficiency virus (HIV).

8. BMI > 40. Note - For Germany: BMI > 35 (Obesity Class II).

9. History of malignancy other than treated basal cell or squamous cell skin cancerwithin the past 5 years.

10. History of alcohol abuse in the last 5 years or currently drinks in excess of 21 and 14 units per week for males and females, respectively.

11. Received an investigational agent within 30 days or 5 half-lives prior to screening (whichever is longer).

12. History of non-compliance such that patient is unlikely to be compliant with studyvisits, procedures or drug administration.

13. Patient has had an organ transplant, is currently on an organ transplant waitinglist or there is a reasonable possibility that the patient will have an organtransplant in the 6 months after screening.

14. Participation in an interventional trial within the previous 3 months prior toscreening or concurrent participation in a research trial.

15. Patient is not suitable to participate in the study for any reason (including, butnot limited to co-morbidities, history of non-compliance with study visits,procedures, or drug administration) in the opinion of the investigator.

16. Females of childbearing potential (those who are not surgically sterilized orpost-menopausal for at least 1 year) are excluded from participation in the studyunless they agree to use highly effective contraception.

17. Females that are lactating.

18. History of hypersensitivity to study drug and/or any of its excipients.

19. Patients with hereditary galactose intolerance, total lactase deficiency orglucose-galactose malabsorption.

20. Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide, abatacept, orsparsentan Alport Syndrome Patients Exclusion Criteria (in addition):

21. Kidney disease apart from AS, e.g. diabetic nephropathy or lupus nephritis.

22. Use of Bardoxolone or sparsentan treatment in the 30 days prior to screening. SGLT2inhibitors are allowed if the patient is on a stable dose for at least 3 monthsprior to enrollment and during the study. Focal Segmental Glomerulosclerosis Patients Exclusion Criteria (in addition):

23. Patient has collapsing variant of FSGS on renal biopsy.

24. Patient has FSGS secondary to another condition (e.g. obesity, cardiovascular,infectious, or autoimmune disorder).

25. Use of Rituximab, cyclophosphamide or abatacept treatment in the 120 days prior toscreening. If taking other chronic immune-modulatory medications that are smallmolecules, the dosage must be stable for 4 weeks prior to screening and during thestudy.

26. If previous Rituximab treatment is greater than 120 days from screening, CD20 cellcount should be within normal limits.

27. If previous other antibody treatment on a stable dose is greater than 120 days fromscreening, the investigator must deem administration of study drug to be safe.

28. Use of sparsentan in the 30 days prior to screening. SGLT2 inhibitors are allowed ifthe patient is on a stable dose for at least 3 months prior to enrollment and duringthe study.