Trial Outcomes & Findings for BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy (NCT NCT05116787)

NCT ID: NCT05116787

Last Updated: 2025-01-03

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Baseline, Week 12

Results posted on

2025-01-03

Participant Flow

The study was conducted in Malaysia, South Africa, and Korea.

A total 12 participants were randomized and treated.

Participant milestones

Participant milestones
Measure	BCX9930/BCX9930 Participants received BCX9930 monotherapy in double blind (DB) manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days.	Placebo/BCX9930 Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.
Part 1:DB Phase (up to 12 Weeks) STARTED	10	2
Part 1:DB Phase (up to 12 Weeks) COMPLETED	8	1
Part 1:DB Phase (up to 12 Weeks) NOT COMPLETED	2	1
Part 2:Open-label Phase (up to 52 Weeks) STARTED	8	1
Part 2:Open-label Phase (up to 52 Weeks) COMPLETED	4	1
Part 2:Open-label Phase (up to 52 Weeks) NOT COMPLETED	4	0

Reasons for withdrawal

Reasons for withdrawal
Measure	BCX9930/BCX9930 Participants received BCX9930 monotherapy in double blind (DB) manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days.	Placebo/BCX9930 Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.
Part 1:DB Phase (up to 12 Weeks) Withdrawal by Subject	0	1
Part 1:DB Phase (up to 12 Weeks) Pregnancy	1	0
Part 1:DB Phase (up to 12 Weeks) Adverse Event	1	0
Part 2:Open-label Phase (up to 52 Weeks) Miscellaneous	4	0

Baseline Characteristics

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BCX9930/BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. 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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days.	Placebo/BCX9930 n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum treatment duration in Part 1 was 12 weeks. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.	Total n=12 Participants Total of all reporting groups
Age, Continuous	37.8 Years STANDARD_DEVIATION 12.70 • n=5 Participants	37.0 Years STANDARD_DEVIATION 9.90 • n=7 Participants	37.7 Years STANDARD_DEVIATION 11.87 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	2 Participants n=7 Participants	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	7 Participants n=5 Participants	0 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Participants in the All Subjects as Treated (ASaT) population (all participants who received at least 1 dose of study drug and had a post baseline laboratory assessment) in Part 1 were analyzed.

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Hemoglobin at Week 12 Baseline	8.49 grams per deciliter (g/dL) Standard Deviation 1.556	9.48 grams per deciliter (g/dL) Standard Deviation 1.721
Part 1: Change From Baseline in Hemoglobin at Week 12 Change at Week 12	2.23 grams per deciliter (g/dL) Standard Deviation 2.146	-1.23 grams per deciliter (g/dL) Standard Deviation 0.330

SECONDARY outcome

Timeframe: From Week 4 to Week 12

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 12, 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=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Participants Who Were Transfusion-free	8 Participants	1 Participants

SECONDARY outcome

Timeframe: From Week 4 to Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Units of pRBCs Transfused	1 units of pRBCs	0 units of pRBCs

SECONDARY outcome

Timeframe: Baseline, Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Percent Change From Baseline in Lactate Dehydrogenase	-69.8 percent change Standard Deviation 26.00	9.1 percent change Standard Deviation 45.47

SECONDARY outcome

Timeframe: Baseline, Week 12

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=9 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score Change at Week 12	-2.3 Scores on a scale Standard Deviation 13.48	-2.0 Scores on a scale Standard Deviation 0.00
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score Baseline	34.3 Scores on a scale Standard Deviation 13.47	13.0 Scores on a scale Standard Deviation 8.49

SECONDARY outcome

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

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

The rate of pRBC units transfused from Week 4 to Week 12 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=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused	89 percent reduction	100 percent reduction

SECONDARY outcome

Timeframe: At Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL	2 Participants	0 Participants

SECONDARY outcome

Timeframe: From Week 4 to Week 12

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 12.

Outcome measures

Outcome measures
Measure	BCX9930 n=8 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Participants Achieving Hemoglobin Stabilization	8 Participants	2 Participants

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Participants in the ASaT population Part 1 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=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size Change at Week 12	30.76 % of PNH-RBC within total RBC population Standard Deviation 25.925	-2.15 % of PNH-RBC within total RBC population Standard Deviation 0.032
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size Baseline	50.16 % of PNH-RBC within total RBC population Standard Deviation 30.386	33.72 % of PNH-RBC within total RBC population Standard Deviation 3.765

SECONDARY outcome

Timeframe: Baseline, Week 12

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 (i.e, 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=7 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=1 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size Baseline	0.6425 ratio Standard Deviation 0.28016	0.6860 ratio
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size Change at Week 12	0.2905 ratio Standard Deviation 0.26628	-0.3052 ratio

SECONDARY outcome

Timeframe: Baseline, Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) Baseline	200.01 10^9 cells/microliter (μL) Standard Deviation 81.094	261.47 10^9 cells/microliter (μL) Standard Deviation 13.529
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) Change at Week 12	-111.01 10^9 cells/microliter (μL) Standard Deviation 80.799	-21.43 10^9 cells/microliter (μL)

SECONDARY outcome

Timeframe: Week 12

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

Number of participants with ARC in the normal range (50 - 100 x 10\^9 cells/L) were reported.

Outcome measures

Outcome measures
Measure	BCX9930 n=9 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=1 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Participants With ARC in the Normal Range	5 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Haptoglobin Baseline	0.182 grams per liter (g/L) Standard Deviation 0.0711	0.195 grams per liter (g/L) Standard Deviation 0.1485
Part 1: Change From Baseline in Haptoglobin Change at Week 12	0.493 grams per liter (g/L) Standard Deviation 0.6948	0.000 grams per liter (g/L) Standard Deviation 0.0000

SECONDARY outcome

Timeframe: Week 12

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

Number of participants with haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range	5 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline, Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Total Bilirubin Change at Week 12	-0.97 milligram per deciliter (mg/dL) Standard Deviation 1.706	0.80 milligram per deciliter (mg/dL) Standard Deviation 0.283
Part 1: Change From Baseline in Total Bilirubin Baseline	1.66 milligram per deciliter (mg/dL) Standard Deviation 1.944	3.45 milligram per deciliter (mg/dL) Standard Deviation 2.475

SECONDARY outcome

Timeframe: Baseline, Week 12

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

Outcome measures

Outcome measures
Measure	BCX9930 n=10 Participants Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks. 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.	Placebo n=2 Participants Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).
Part 1: Change From Baseline in Aspartate Aminotransferase (AST) Change at Week 12	-68.83 Units per liter (U/L) Standard Deviation 58.333	13.00 Units per liter (U/L) Standard Deviation 36.487
Part 1: Change From Baseline in Aspartate Aminotransferase (AST) Baseline	107.43 Units per liter (U/L) Standard Deviation 71.295	83.60 Units per liter (U/L) Standard Deviation 6.505

Adverse Events

BCX9930/BCX9930

Serious events: 5 serious events

Other events: 10 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

BCX9930 After Placebo

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BCX9930/BCX9930 n=10 participants at risk Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. 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 was 378 days.	Placebo n=2 participants at risk Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1).	BCX9930 After Placebo n=1 participants at risk Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the 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 was 281 days.
Infections and infestations Enterovirus infection	0.00% 0/10 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	100.0% 1/1 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Infections and infestations Influenza	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Infections and infestations Pneumonia fungal	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Infections and infestations Tuberculosis	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Infections and infestations Urinary tract infection	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Blood and lymphatic system disorders Breakthrough haemolysis	10.0% 1/10 • Number of events 2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Hepatobiliary disorders Budd-Chiari syndrome	0.00% 0/10 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	50.0% 1/2 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Renal and urinary disorders Renal failure	10.0% 1/10 • Number of events 1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/2 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.	0.00% 0/1 • From Day 1 up to 408 days The safety population included all participants who received at least 1 dose of study drug, whether placebo 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