Trial Outcomes & Findings for A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT NCT04702568)

Last Updated: 2025-04-13

Results Overview

An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

From first dose of study drug up to 3 weeks after last dose (Week 147)

Results posted on

2025-04-13

Participant Flow

Participants who had participated in previous BCX9930 trials (BCX9930-101 \[NCT04330534\], BCX9930-202 \[NCT05116774\], or BCX9930-203 \[NCT05116787\]) for Paroxysmal Nocturnal Hemoglobinuria (PNH) and showed a benefit of treatment as determined by the investigator were enrolled in this long-term safety trial.

Participant milestones

Participant milestones
Measure	Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 milligrams (mg) orally twice daily (BID). Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Overall Study STARTED	12	7
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	12	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 milligrams (mg) orally twice daily (BID). Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Overall Study Transitioned to the BCX9930-205 roll-over study	10	4
Overall Study Pregnancy	1	0
Overall Study Bone marrow transplant	1	0
Overall Study Withdrawn due to Investigator decision	0	1
Overall Study Withdrawal by Subject	0	2

Baseline Characteristics

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	Total n=19 Participants Total of all reporting groups
Age, Continuous	29.3 years STANDARD_DEVIATION 7.82 • n=5 Participants	44.6 years STANDARD_DEVIATION 18.59 • n=7 Participants	34.9 years STANDARD_DEVIATION 14.50 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	2 Participants n=7 Participants	11 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	12 Participants n=5 Participants	7 Participants n=7 Participants	19 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	1 Participants n=5 Participants	1 Participants n=7 Participants	2 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	2 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	4 Participants n=7 Participants	7 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	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: The safety population included all participants who received at least 1 capsule or tablet of study drug.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	12 Participants	7 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: The mITT (modified intent-to-treat) population included all participants who received at least 1 capsule or tablet of study drug and had post baseline assessment of PNH symptoms and/or laboratory data. Participants with available data at each visit were included.

Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 96-none	6 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Baseline-moderate	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Baseline-severe	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 24-mild	2 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 24-moderate	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 48-none	6 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 72-none	7 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 96-mild	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 96-severe	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 120-none	8 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 120-moderate	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Baseline-none	8 Participants	4 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Baseline-mild	3 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 24-none	10 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 48-mild	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 48-moderate	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 72-mild	1 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue Week 72-moderate	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Baseline-none	11 Participants	6 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Baseline-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 24-none	11 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 24-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 48-none	6 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 48-mild	2 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 72-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 72-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 96-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 120-mild	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 96-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 96-moderate	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea Week 120-none	8 Participants	2 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Baseline-none	12 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Baseline-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 24-none	12 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 24-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 48-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 72-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 72-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 96-none	7 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 96-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 120-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 48-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 72-moderate	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort Week 96-severe	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Baseline-none	11 Participants	6 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Baseline-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 24-none	12 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 48-none	6 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 48-mild	2 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 96-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 120-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 24-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 72-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 72-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 96-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing Week 120-severe	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Baseline-none	10 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Baseline-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 24-mild	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 96-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 96-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Baseline-moderate	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 24-none	9 Participants	6 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 24-moderate	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 48-none	6 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 48-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 48-severe	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 72-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 72-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain Week 120-none	8 Participants	3 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 24-mild	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 48-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 96-none	7 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Baseline-none	10 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Baseline-mild	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 24-none	11 Participants	6 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 48-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 48-moderate	1 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 72-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 72-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 96-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 120-none	8 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 120-mild	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache Week 120-moderate	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 120-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 48-none	7 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 48-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 72-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 96-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 96-mild	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 120-none	7 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Baseline-none	9 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Baseline-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 24-none	9 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 24-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 72-none	7 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction Week 96-severe	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Baseline-none	10 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Baseline-missing severity	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 24-none	11 Participants	5 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 24-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 24-missing severity	1 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 48-none	6 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 48-moderate	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 48-missing severity	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 72-missing severity	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 96-none	6 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 96-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 120-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 120-missing severity	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Baseline-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 48-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 72-none	7 Participants	2 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 72-mild	0 Participants	1 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 96-missing severity	2 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria Week 120-none	7 Participants	3 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

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

Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 48-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Baseline-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Baseline-missing severity	1 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 24-none	12 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 24-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 24-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 24-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 48-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 48-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 48-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 72-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 72-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 96-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 96-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 120-none	8 Participants	3 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Baseline-none	11 Participants	7 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Baseline-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Baseline-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 72-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 72-severe	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 96-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 96-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 120-mild	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 120-moderate	0 Participants	0 Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice Week 120-severe	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks, 24, 48, 72, 96, 120, and 144

Population: Participants in the mITT population with available data were analyzed.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=6 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Change From Baseline in Lactate Dehydrogenase (LDH) Baseline	475.6 Units per liter (U/L) Standard Deviation 210.98	248.3 Units per liter (U/L) Standard Deviation 125.71
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 24	-46.7 Units per liter (U/L) Standard Deviation 154.55	38.6 Units per liter (U/L) Standard Deviation 45.51
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 48	-62.5 Units per liter (U/L) Standard Deviation 80.97	38.5 Units per liter (U/L) Standard Deviation 65.76
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 72	-6.1 Units per liter (U/L) Standard Deviation 238.41	163.5 Units per liter (U/L) Standard Deviation 118.09
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 96	237.4 Units per liter (U/L) Standard Deviation 347.65	-4.0 Units per liter (U/L) Standard Deviation 134.35
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 120	195.4 Units per liter (U/L) Standard Deviation 355.03	31.0 Units per liter (U/L) Standard Deviation 125.87
Change From Baseline in Lactate Dehydrogenase (LDH) Change at Week 144	-59.0 Units per liter (U/L)	—

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, 120, and 144

Population: Participants in the mITT population with available data were analyzed.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Change From Baseline in Hemoglobin Change at Week 48	-0.61 grams per deciliter (g/dl) Standard Deviation 1.974	-0.95 grams per deciliter (g/dl) Standard Deviation 2.333
Change From Baseline in Hemoglobin Change at Week 72	-0.64 grams per deciliter (g/dl) Standard Deviation 2.327	-0.93 grams per deciliter (g/dl) Standard Deviation 1.484
Change From Baseline in Hemoglobin Change at Week 96	-0.33 grams per deciliter (g/dl) Standard Deviation 1.914	-0.70 grams per deciliter (g/dl) Standard Deviation 2.687
Change From Baseline in Hemoglobin Change at Week 120	0.38 grams per deciliter (g/dl) Standard Deviation 1.555	-0.47 grams per deciliter (g/dl) Standard Deviation 2.098
Change From Baseline in Hemoglobin Change at Week 144	-2.70 grams per deciliter (g/dl)	—
Change From Baseline in Hemoglobin Baseline	11.88 grams per deciliter (g/dl) Standard Deviation 1.688	11.53 grams per deciliter (g/dl) Standard Deviation 1.803
Change From Baseline in Hemoglobin Change at Week 24	0.75 grams per deciliter (g/dl) Standard Deviation 1.159	-1.32 grams per deciliter (g/dl) Standard Deviation 2.578

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=5 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Change From Baseline in Haptoglobin Baseline	0.325 grams per liter (g/L) Standard Deviation 0.2832	0.216 grams per liter (g/L) Standard Deviation 0.1350
Change From Baseline in Haptoglobin Change at Week 24	0.000 grams per liter (g/L) Standard Deviation 0.0447	-0.082 grams per liter (g/L) Standard Deviation 0.0844
Change From Baseline in Haptoglobin Change at Week 48	0.213 grams per liter (g/L) Standard Deviation 0.1126	—
Change From Baseline in Haptoglobin Change at Week 72	0.025 grams per liter (g/L) Standard Deviation 0.1165	-0.210 grams per liter (g/L) Standard Deviation 0.1414
Change From Baseline in Haptoglobin Change at Week 96	-0.013 grams per liter (g/L) Standard Deviation 0.0354	0.195 grams per liter (g/L) Standard Deviation 0.4313
Change From Baseline in Haptoglobin Change at Week 120	-0.013 grams per liter (g/L) Standard Deviation 0.0354	-0.100 grams per liter (g/L)

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Change From Baseline in Reticulocytes Baseline	0.1066 10^6 cells per microliter (μL) Standard Deviation 0.02363	0.0889 10^6 cells per microliter (μL) Standard Deviation 0.03939
Change From Baseline in Reticulocytes Change at Week 24	0.0164 10^6 cells per microliter (μL) Standard Deviation 0.02339	0.0913 10^6 cells per microliter (μL) Standard Deviation 0.09144
Change From Baseline in Reticulocytes Change at Week 48	0.0048 10^6 cells per microliter (μL) Standard Deviation 0.03518	0.0507 10^6 cells per microliter (μL) Standard Deviation 0.03161
Change From Baseline in Reticulocytes Change at Week 72	0.0044 10^6 cells per microliter (μL) Standard Deviation 0.03643	0.0463 10^6 cells per microliter (μL) Standard Deviation 0.03008
Change From Baseline in Reticulocytes Change at Week 96	0.0409 10^6 cells per microliter (μL) Standard Deviation 0.03805	0.0607 10^6 cells per microliter (μL) Standard Deviation 0.03585
Change From Baseline in Reticulocytes Change at Week 120	0.0173 10^6 cells per microliter (μL) Standard Deviation 0.03303	0.0653 10^6 cells per microliter (μL) Standard Deviation 0.02380

SECONDARY outcome

Timeframe: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: Participants in the mITT population with available data were analyzed.

Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=12 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Participants With Blood Transfusions or Thromboses Blood transfusions	3 Participants	4 Participants
Number of Participants With Blood Transfusions or Thromboses Thromboses	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: Participants from mITT population who required blood transfusions.

Number of blood transfusions were reported.

Outcome measures

Outcome measures
Measure	C5-INH Naïve Group n=3 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=4 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Number of Blood Transfusions	13 Number of blood transfusions	25 Number of blood transfusions

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 48, 72, and 96

Population: Participants in the mITT population 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	C5-INH Naïve Group n=11 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 Participants This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score Baseline	43.82 Scores on a scale Standard Deviation 9.400	40.14 Scores on a scale Standard Deviation 13.533
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score Change at Week 48	-0.63 Scores on a scale Standard Deviation 2.387	2.33 Scores on a scale Standard Deviation 3.215
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score Change at Week 72	-0.38 Scores on a scale Standard Deviation 2.875	-4.64 Scores on a scale Standard Deviation 12.016
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score Change at Week 24	0.09 Scores on a scale Standard Deviation 4.110	-3.36 Scores on a scale Standard Deviation 8.066
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score Change at Week 96	-1.13 Scores on a scale Standard Deviation 4.357	-18.00 Scores on a scale Standard Deviation 27.185

Adverse Events

Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group

Serious events: 6 serious events

Other events: 12 other events

Deaths: 0 deaths

C5-INH Inadequate Response Group

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group n=12 participants at risk This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	C5-INH Inadequate Response Group n=7 participants at risk This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
Blood and lymphatic system disorders Breakthrough haemolysis	25.0% 3/12 • Number of events 5 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Blood and lymphatic system disorders Haemolysis	8.3% 1/12 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Blood and lymphatic system disorders Febrile neutropenia	8.3% 1/12 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Infections and infestations Boutonneuse fever	8.3% 1/12 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Infections and infestations Pyelonephritis	0.00% 0/12 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Infections and infestations Sepsis	0.00% 0/12 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Infections and infestations Soft tissue infection	8.3% 1/12 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Gastrointestinal disorders Crohn's disease	0.00% 0/12 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Gastrointestinal disorders Gastritis	8.3% 1/12 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
General disorders Pyrexia	0.00% 0/12 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Epstein-Barr virus associated lymphoma	0.00% 0/12 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	14.3% 1/7 • Number of events 1 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
Renal and urinary disorders Acute kidney injury	8.3% 1/12 • Number of events 2 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.	0.00% 0/7 • From first dose of study drug up to 3 weeks after last dose (Week 147) The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.

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