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Phase 2 Study of Avacopan for Treatment of C3G, IND 132321, Orphan Designation 16-5590

Award Number: R01FD006342
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/10/2019
PERIOD OF PERFORMANCE END DATE: 01/31/2022

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Phase 2 Study of Avacopan for Treatment of C3G, IND 132321, Orphan Designation 16-5590 - Project Summary Complement 3 Glomerulopathy (C3G) is an orphan disease which includes C3 glomerulonephritis (C3GN) and the closely related dense deposit disease (DDD), forms of glomerulonephritis. This disease results from abnormal regulation of the alternative complement pathway resulting in unrestrained complement activation. The clinical presentation varies from mild proteinuria to rapid renal deterioration and about half of the individuals with C3G move the End Stage Renal Disease within in 10 years of diagnosis. There are no approved drugs for C3G currently The current treatment recommendations include control of hypertension with angiotensin-converting enzyme (ACE)/angiotensin receptor blocker (ARBs) to empirical use of plasma exchange or infusion. None of these treatments address the dysregulation of the complement system. Hence, there is a significant unmet medical need for novel drugs to treat C3G. Avacopan is an orally active drug that specific inhibitor of the C5a receptor (part of the complement system) and has been shown to be safe and efficacious in Phase 1 and Phase 2 human clinical trials in AAV, and is currently being tested in a Phase 3 AAV trial. More importantly, compelling clinical, laboratory, and kidney biopsy responses have been observed in C3G patients treated with avacopan under a compassionate use protocol. Taken together, these findings provide a strong justification for a human clinical trial to test the safety and efficacy of avacopan as a novel treatment of C3G. This study will enroll 44 patients with C3G at 40 clinical sites in 11 countries. The primary goal of the study is to evaluate the efficacy of avacopan compared to placebo based on histologic changes in kidney biopsies taken before and during treatment. Secondary goals of this study include the evaluation of: 1 The safety of avacopan compared to placebo based on the incidence of adverse events, changes in clinical laboratory measurements, and vital signs; 2 Changes in laboratory parameters of renal disease including estimated glomerular filtration rate (eGFR), proteinuria, and urinary excretion of monocyte chemoattractant protein-1 (MCP- 1) with avacopan compared to placebo; 3 Health-related quality-of-life changes based on Short Form-36 version 2 (SF-36 v2) and EuroQOL-5D-5L (EQ-5D-5L) with avacopan compared to placebo; 4 The pharmacokinetic profile of avacopan in patients with C3G. Additionally, exploratory evaluations studying changes from baseline in markers of alternative complement pathway involvement and other markers of inflammation may be assessed in plasma/serum or urine over the course of the treatment period.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2020	CHEMOCENTRYX, INC.	835 INDUSTRIAL RD STE 600	SAN CARLOS	CA	94070	SAN MATEO	USA	Food and Drug Administration Research	000	2	10/19/2021	NON-COMPETING CONTINUATION	$0
2022	2020	CHEMOCENTRYX, INC.	835 INDUSTRIAL RD	SAN CARLOS	CA	94070	SAN MATEO	USA	Food and Drug Administration Research	001	2	6/1/2022	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2020	CHEMOCENTRYX, INC.	835 INDUSTRIAL RD STE 600	SAN CARLOS	CA	94070	SAN MATEO	USA	Food and Drug Administration Research	000	2	8/30/2021	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $500,000 )
2020	2020	CHEMOCENTRYX, INC.	850 MAUDE AVE	MOUNTAIN VIEW	CA	94043	SANTA CLARA	USA	Food and Drug Administration Research	000	2	8/27/2020	NON-COMPETING CONTINUATION	$500,000
														Subtotal = $500,000

Issue Date FY: 2019 ( Subtotal = $500,000 )
2019	2019	CHEMOCENTRYX, INC.	850 MAUDE AVE	MOUNTAIN VIEW	CA	94043	SANTA CLARA	USA	Food and Drug Administration Research	000	1	9/10/2019	NEW	$500,000
														Subtotal = $500,000

Grand Total All Awards = $1,000,000

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Phase 2 Study of Avacopan for Treatment of C3G, IND 132321, Orphan Designation 16-5590

Award Number: R01FD006342

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/10/2019

PERIOD OF PERFORMANCE END DATE: 01/31/2022

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