A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis.

All Patients:
1. Patient is able to communicate well with the investigator, understands and
is willing to comply with all requirements of the study, and understands and
signs the written informed consent form (ICF).
2. For children to be eligible, one or both parents/legal guardians must sign a
parental permission form which provides information contained in the ICF.
Children capable of assent must express their willingness to participate by
signing an assent form.
3. If patient has received a COVID vaccination, the baseline visit must occur
at least one week or more after the second/booster vaccination.
4. Patients who have had active symptoms of COVID within 3 months prior to
screening and are now asymptomatic for the last 2 weeks but have tested COVID
PCR positive. If a patient is asymptomatic at screening but is COVID positive,
then rescreening can occur after a minimum of two weeks.
5. Both female patients, as well as female partners of male patients who are of
child-bearing potential must be willing to not become pregnant for the complete
duration of the study (>180 days) (90 days after the last dose of study
medication).
6. Males (including sterilized subjects) whose female partners have
child-bearing potential, must agree to use male contraception (condoms) during
the period from the time of signing the informed consent form (ICF) through 90
days after the last dose of study drug. They must agree to immediately inform
the investigator if their partner becomes pregnant during the study.

AS Inclusion Criteria (in addition):
7. Males and females with X-Linked AS and males and females with autosomal
inherited AS.
a. For countries that are enrolling pediatric patients: patients
from age 12 years and older.
b. For countries that are not enrolling pediatric patients: patients
from age 18 years and older.
8. Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For
patients enrolled in the US who meet all inclusion and exclusion criteria but
have not had their diagnosis confirmed by genetic testing or kidney biopsy, the
Sponsor will provide for patient*s genetic testing.
9. UPCR >=1.0 g/g.
10. eGFR >= 45 mL/min/1.73m2 (using CKD-EPI equation for adults and
Bedside Schwartz equation for children).
11. ACEi/ARB therapy at maximum tolerated dose stable for at least 4
weeks prior to screening. ACEi/ARB dose should remain stable over
the course of the study.

FSGS Inclusion Criteria (in addition):
12. Male or female patients,
a. For countries that are enrolling pediatric patients: 12 to 75 years old at
the time of signing the informed consent
b. For countries that are not enrolling pediatric patients: 18 to 75 years old
at the time of signing the informed consent
13. Primary FSGS (without any identifiable cause, and where the FSGS is
confirmed by renal biopsy) or FSGS where there is documentation of a genetic
mutation in a podocyte protein associated with FSGS.
14. Steroid-resistance defined as failure to achieve partial or complete
remission, or experienced adverse events without acceptable clinical benefit
after at least 8 weeks of adequate corticosteroid therapy for children and 12
weeks for adults.
15. UPCR between 3.5g/g and 12.0g/g.
16. eGFR > 45 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside
Schwartz equation for children).
17. If taking concomitant ACEi and/or ARB treatment, it should remain at a
stable dose for a minimum of 28 days prior to enrollment and during the course
of the study.

All Patients:
1. Uncontrolled diabetes mellitus as evidenced by an HbA1c >= 11%. For Germany:
HbA1c >= 8.5%.
2. Uncontrolled hypertension
a. Adults: (SBP >= 180mmHg and/or DBP >= 100mmHg). For Germany: (SBP >= 140mmHg
and/or DBP >= 100mmHg).
b. Children: >= 95th percentile or >= 130/80 mm Hg, whichever is lower, as
defined in Appendix 13.8.
3. Moderate or severe hepatic impairment as per Child Pugh score (See Section
9.5.4.7), except if (a) decreased serum albumin is directly related to the
renal disease (resulting in a Child Pugh score of 7), and (b) no other
Child-Pugh Score parameters are increased and (c) patient has no liver
pathology in medical history.
4. Presence of any active (i.e., with symptoms) and/or uncontrolled infection
(including COVID).
5. Presence of Human immunodeficiency virus (HIV).
6. BMI > 40. For Germany: BMI > 35 (Obesity Class II).
7. History of malignancy other than treated basal cell or squamous cell skin
cancer within the past 5 years.
8. 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.
9. Received an investigational agent within 30 days or 5 half-lives prior to
screening (whichever is longer).
10. History of non-compliance such that patient is unlikely to be compliant
with study visits, procedures or drug administration.
11. Patient has had an organ transplant, is currently on an organ transplant
waiting list or there is a reasonable possibility that the patient will have an
organ transplant in the 6 months after screening.
12. Participation in an interventional trial within the previous 3 months prior
to screening or concurrent participation in a research trial.
13. Patient is not suitable to participate in the study for any reason
(including, but not limited to co-morbidities, history of non-compliance with
study visits, procedures, or drug administration) in the opinion of the
investigator.
14. Females of childbearing potential (those who are not surgically sterilized
or post-menopausal for at least 1 year) are excluded from participation in the
study unless they agree to use highly effective contraception as described in
Section 13.3.
15. Females that are lactating.
16. History of hypersensitivity to study drug and/or any of its excipients.
17. Patients with hereditary galactose intolerance, total lactase deficiency or
glucose-galactose malabsorption.
18. Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide,
abatacept or sparsentan.
AS Exclusion Criteria (in addition):
19. Kidney disease apart from AS, e.g., diabetic nephropathy or lupus nephritis.
20. Use of Bardoxolone or sparsentan treatment in the 30 days prior to
screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for
at least 3 months prior to screening.
FSGS Exclusion Criteria (in addition):
21. Patient has collapsing variant of FSGS on renal biopsy.
22. Patient has FSGS secondary to another condition (e.g., obesity,
cardiovascular, infectious, or autoimmune disorder).
23. Use of Rituximab, cyclophosphamide or abatacept treatment in the 120 days
prior to screening. If taking other chronic immune-modulatory medications that
are small molecules, the dosage must be stable for 4 weeks prior to screening.
24. If previous Rituximab treatment is greater than 120 days from screening,
CD20 cell count should be within normal limits.
25. If previous other antibody treatment on a stable dose is greater than 120
days from screening, the investigator must deem administration of study drug to
be safe.
26. Use of sparsentan in the 30 days prior to screening. SGLT2 inhibitors are
allowed if the patient is on a stable dose for at least 3 months prior to
screening.