Trial Outcomes & Findings for BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy (NCT NCT05116774)

NCT ID: NCT05116774

Last Updated: 2025-03-07

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Baseline, Week 24

Results posted on

2025-03-07

Participant Flow

A total 12 participants were randomized and treated.

The study was conducted in France, Hungary, Italy, Spain and United Kingdom.

Participant milestones

Participant milestones
Measure	BCX9930/BCX9930 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	C5-Inhibitor (C5-INH)/BCX9930 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
Part 1 (up to 24 Weeks) STARTED	8	4
Part 1 (up to 24 Weeks) COMPLETED	7	3
Part 1 (up to 24 Weeks) NOT COMPLETED	1	1
Part 2 (up to 52 Weeks) STARTED	7	3
Part 2 (up to 52 Weeks) COMPLETED	3	2
Part 2 (up to 52 Weeks) NOT COMPLETED	4	1

Reasons for withdrawal

Reasons for withdrawal
Measure	BCX9930/BCX9930 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	C5-Inhibitor (C5-INH)/BCX9930 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
Part 1 (up to 24 Weeks) Withdrawal by Subject	0	1
Part 1 (up to 24 Weeks) Adverse Event	1	0
Part 2 (up to 52 Weeks) Adverse Event	1	0
Part 2 (up to 52 Weeks) Miscellaneous	3	1

Baseline Characteristics

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BCX9930/BCX9930 n=8 Participants Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	C5-INH/BCX9930 n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.	Total n=12 Participants Total of all reporting groups
Age, Continuous	58.8 years STANDARD_DEVIATION 17.66 • n=99 Participants	48.5 years STANDARD_DEVIATION 14.25 • n=107 Participants	55.3 years STANDARD_DEVIATION 16.71 • n=206 Participants
Sex: Female, Male Female	4 Participants n=99 Participants	3 Participants n=107 Participants	7 Participants n=206 Participants
Sex: Female, Male Male	4 Participants n=99 Participants	1 Participants n=107 Participants	5 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=99 Participants	2 Participants n=107 Participants	7 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=99 Participants	2 Participants n=107 Participants	5 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	2 Participants n=99 Participants	0 Participants n=107 Participants	2 Participants n=206 Participants
Race (NIH/OMB) White	5 Participants n=99 Participants	2 Participants n=107 Participants	7 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants

PRIMARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the all subjects as treated (ASaT) population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=8 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Hemoglobin at Week 24 Change at Week 24	3.48 grams per deciliter (g/dL) Standard Deviation 0.674	0.72 grams per deciliter (g/dL) Standard Deviation 0.884
Part 1: Change From Baseline in Hemoglobin at Week 24 Baseline	9.12 grams per deciliter (g/dL) Standard Deviation 1.096	9.13 grams per deciliter (g/dL) Standard Deviation 0.847

SECONDARY outcome

Timeframe: From Week 4 to Week 24

Population: Participants in the ASaT population in Part 1 were analyzed.

The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.

Outcome measures

Outcome measures
Measure	BCX9930 n=8 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Participants Who Were Transfusion-free	7 Participants	4 Participants

SECONDARY outcome

Timeframe: From Week 4 to Week 24

Population: Participants in the ASaT population in Part 1 were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=8 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Units of pRBCs Transfused	0 units of pRBCs	0 units of pRBCs

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

Outcome measures

Outcome measures
Measure	BCX9930 n=6 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=2 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score Change at Week 24	-0.3 Scores on a scale Standard Deviation 12.86	1.0 Scores on a scale
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score Baseline	36.3 Scores on a scale Standard Deviation 9.69	40.0 Scores on a scale Standard Deviation 8.49

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=7 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=2 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Percent Change From Baseline in Lactate Dehydrogenase	24.3 percent change Standard Deviation 53.07	-21.0 percent change Standard Deviation 4.03

SECONDARY outcome

Timeframe: Prestudy (12 months prior to screening) and from Week 4 to Week 24

Population: Participants in the ASaT population in Part 1 were analyzed.

The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate \* 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.

Outcome measures

Outcome measures
Measure	BCX9930 n=8 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused	100 percent reduction	NA percent reduction No participants received pRBC transfusion at pre-study in the "C5-INH" arm, and therefore the percentage reduction, based on the formula provided, was not calculable.

SECONDARY outcome

Timeframe: Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=6 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)	6 Participants	0 Participants

SECONDARY outcome

Timeframe: From Week 4 to Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.

Outcome measures

Outcome measures
Measure	BCX9930 n=5 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Participants Who Achieved Hemoglobin Stabilization	5 Participants	4 Participants

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: No data was collected for this endpoint.

Red blood cells opsonized by C3 were to be assessed by flow cytometry .

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.

Outcome measures

Outcome measures
Measure	BCX9930 n=6 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=3 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size Baseline	80.51 % of PNH-RBC within total RBC population Standard Deviation 16.94	83.40 % of PNH-RBC within total RBC population Standard Deviation 14.34
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size Change at Week 24	23.18 % of PNH-RBC within total RBC population Standard Deviation 11.81	-6.34 % of PNH-RBC within total RBC population Standard Deviation 8.986

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.

Outcome measures

Outcome measures
Measure	BCX9930 n=4 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=2 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size Baseline	0.8128 ratio Standard Deviation 0.16528	0.9059 ratio Standard Deviation 0.12113
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size Change at Week 24	0.2336 ratio Standard Deviation 0.11595	-0.0668 ratio Standard Deviation 0.09462

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=7 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) Baseline	0.2933 10^6 cells/microliter (μL) Standard Deviation 0.10453	0.3129 10^6 cells/microliter (μL) Standard Deviation 0.16201
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) Change at Week 24	-0.2142 10^6 cells/microliter (μL) Standard Deviation 0.05384	-0.0462 10^6 cells/microliter (μL) Standard Deviation 0.01823

SECONDARY outcome

Timeframe: Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported.

Outcome measures

Outcome measures
Measure	BCX9930 n=6 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Participants With ARC in the Normal Range	4 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=7 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Haptoglobin Baseline	0.126 grams per liter (g/L) Standard Deviation 0.0732	0.105 grams per liter (g/L) Standard Deviation 0.0268
Part 1: Change From Baseline in Haptoglobin Change at Week 24	-0.024 grams per liter (g/L) Standard Deviation 0.0409	-0.015 grams per liter (g/L) Standard Deviation 0.0268

SECONDARY outcome

Timeframe: Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.

Outcome measures

Outcome measures
Measure	BCX9930 n=7 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Participants in the ASaT population in Part 1 with available data were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=7 Participants Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.	C5-INH n=4 Participants Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.
Part 1: Change From Baseline in Total Bilirubin Baseline	3.44 milligram per deciliter (mg/dL) Standard Deviation 3.679	1.10 milligram per deciliter (mg/dL) Standard Deviation 0.424
Part 1: Change From Baseline in Total Bilirubin Change at Week 24	-2.66 milligram per deciliter (mg/dL) Standard Deviation 3.672	-0.02 milligram per deciliter (mg/dL) Standard Deviation 0.250

Adverse Events

BCX9930/BCX9930

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

C5-INH

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

BCX9930 After C5-INH

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BCX9930/BCX9930 n=8 participants at risk Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	C5-INH n=4 participants at risk Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH was 24 weeks.	BCX9930 After C5-INH n=3 participants at risk Participants who were initially randomized to continue C5-INH therapy received BCX9930 monotherapy after they had completed 24 weeks on C5-INH, or earlier after the date when sponsor decided to halt enrolment in the study permanently and terminate the study. Initially participants were to receive BCX9930 500 mg BID orally. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 was 197 days.
Investigations Blood creatinine increased	12.5% 1/8 • Number of events 1 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	0.00% 0/4 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	0.00% 0/3 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.
Renal and urinary disorders Acute kidney injury	12.5% 1/8 • Number of events 1 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	0.00% 0/4 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	0.00% 0/3 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.
Skin and subcutaneous tissue disorders Rash erythematous	0.00% 0/8 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	0.00% 0/4 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.	33.3% 1/3 • Number of events 1 • Baseline up to Day 407 The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.

Other adverse events

Additional Information

Study Director

BioCryst Pharmaceuticals Inc

Phone: +1 919-859-1302

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place