Trial Outcomes & Findings for Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is Difficult to Apply (NCT NCT04182100)

Last Updated: 2025-09-15

Results Overview

The primary efficacy endpoint was the phlebotomy-free CHR rate at Months 9 and 12. The phlebotomy-free CHR rate was defined as the proportion of patients who achieved phlebotomy-free CHR at both Months 9 and 12 without phlebotomies during the previous 3 months. A responder in sense of the primary endpoint was a patient who met all of the following criteria at Months 9 and 12: Hematocrit \<45% phlebotomy-free (absence of phlebotomy during the previous 3 months) Platelet count ≤400 × 10\^9/L Leukocyte count ≤10 × 10\^9/L

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

29 participants

Primary outcome timeframe

12 months

Results posted on

2025-09-15

Participant Flow

Study was initiated at 8 sites in 1 country.

Eligible patients were to be treated with P1101, starting at 100 μg (or 50 μg in patients under another cytoreductive therapy). The dose was to be gradually increased by 50 μg every 2 weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelet count ≤400 × 10\^9/L, and leukocyte count ≤10 × 10\^9/L). The maximum recommended single dose is 500 μg injected every 2 weeks.

Participant milestones

Participant milestones
Measure	P1101 Eligible patients were to be treated with P1101, starting at 100 μg (or 50 μg in patients under another cytoreductive therapy). The dose was to be gradually increased by 50 μg every 2 weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelet count ≤400 × 10\^9/L, and leukocyte count ≤10 × 10\^9/L). The maximum recommended single dose is 500 μg injected every 2 weeks.At Week 36 (Month 9) and Week 52 (Month 12), the primary study endpoint, phlebotomy-free CHR, was to be analyzed. After completion of the 52-week study period, provision and administration of P1101, collection of the long-term follow up information (blood parameters, molecular and cytogenetic data, safety parameters, and as also the optional bone marrow data) would be continued.
Overall Study STARTED	29
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	P1101 Eligible patients were to be treated with P1101, starting at 100 μg (or 50 μg in patients under another cytoreductive therapy). The dose was to be gradually increased by 50 μg every 2 weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelet count ≤400 × 10\^9/L, and leukocyte count ≤10 × 10\^9/L). The maximum recommended single dose is 500 μg injected every 2 weeks.At Week 36 (Month 9) and Week 52 (Month 12), the primary study endpoint, phlebotomy-free CHR, was to be analyzed. After completion of the 52-week study period, provision and administration of P1101, collection of the long-term follow up information (blood parameters, molecular and cytogenetic data, safety parameters, and as also the optional bone marrow data) would be continued.
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is Difficult to Apply

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	P1101 n=29 Participants The dose was to be gradually increased by 50 μg every 2 weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelet count ≤400 × 10\^9/L, and leukocyte count ≤10 × 10\^9/L). The maximum recommended single dose is 500 μg injected every 2 weeks.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=99 Participants
Age, Categorical >=65 years	7 Participants n=99 Participants
Sex: Female, Male Female	16 Participants n=99 Participants
Sex: Female, Male Male	13 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	29 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	0 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment Japan	29 participants n=99 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Proportion of Subjects Who Achieved Durable Phlebotomy-free Complete Hematological Response (CHR) at Month 12	8 Participants

SECONDARY outcome

Timeframe: Baseline, 3 months, 6 months, 9 months and 12 months

Population: Intent-to-Treat (ITT) Population Number analyzed: the number of subjects with non-missing value at the visit

Hct will be recorded every 3 months.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Changes in Hct From Baseline Baseline	46.85 percent Standard Deviation 4.369
Changes in Hct From Baseline Change from Baseline at Week 12	-2.21 percent Standard Deviation 4.249
Changes in Hct From Baseline Change from Baseline at Week 24	-2.95 percent Standard Deviation 6.660
Changes in Hct From Baseline Change from Baseline at Week 36	-3.09 percent Standard Deviation 5.888
Changes in Hct From Baseline Change from Baseline at Week 52	-5.45 percent Standard Deviation 6.656

SECONDARY outcome

Timeframe: Baseline, 3 months, 6 months, 9 months and 12 months

Population: Intent-to-Treat (ITT) Population Number analyzed: the number of subjects with non-missing value at the visit

WBC count will be recorded every 3 months.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Changes in WBC Count From Baseline Baseline	17.07 10^9 cells/L Standard Deviation 8.824
Changes in WBC Count From Baseline Change from Baseline at Week 12	-8.50 10^9 cells/L Standard Deviation 7.607
Changes in WBC Count From Baseline Change from Baseline at Week 24	-10.57 10^9 cells/L Standard Deviation 7.842
Changes in WBC Count From Baseline Change from Baseline at Week 36	-11.29 10^9 cells/L Standard Deviation 8.214
Changes in WBC Count From Baseline Change from Baseline at Week 52	-11.71 10^9 cells/L Standard Deviation 8.379

SECONDARY outcome

Timeframe: Baseline, 3 months, 6 months, 9 months and 12 months

Population: Intent-to-Treat (ITT) Population Number analyzed: the number of subjects with non-missing value at the visit

Plt count will be recorded every 3 months.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Changes in Plt Count From Baseline Baseline	747.3 10^9 platelets/L Standard Deviation 378.32
Changes in Plt Count From Baseline Change from Baseline at Week 12	-283.6 10^9 platelets/L Standard Deviation 325.58
Changes in Plt Count From Baseline Change from Baseline at Week 24	-423.0 10^9 platelets/L Standard Deviation 377.93
Changes in Plt Count From Baseline Change from Baseline at Week 36	-467.4 10^9 platelets/L Standard Deviation 396.44
Changes in Plt Count From Baseline Change from Baseline at Week 52	-493.6 10^9 platelets/L Standard Deviation 374.90

SECONDARY outcome

Timeframe: Baseline, 3 months, 6 months, 9 months and 12 months

Population: Intent-to-Treat (ITT) Population Number analyzed: the number of subjects with non-missing value at the visit

Spleen size will be recorded every 3 months.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Changes in Spleen Size From Baseline Change from Baseline at Week 12	2.587 square centimeter Standard Deviation 9.5810
Changes in Spleen Size From Baseline Change from Baseline at Week 24	1.847 square centimeter Standard Deviation 10.8476
Changes in Spleen Size From Baseline Change from Baseline at Week 36	2.604 square centimeter Standard Deviation 12.1256
Changes in Spleen Size From Baseline Change from Baseline at Week 52	5.043 square centimeter Standard Deviation 15.1477

SECONDARY outcome

Timeframe: Up to 12 months

Time to requiring no phlebotomy is recorded.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Time to Requiring no Phlebotomy Week 52	0.0 month Interval 0.0 to 1.0
Time to Requiring no Phlebotomy Baseline	0.3 month Interval 0.0 to 1.0
Time to Requiring no Phlebotomy Week 12	0.1 month Interval 0.0 to 1.0
Time to Requiring no Phlebotomy Week 24	0.0 month Interval 0.0 to 1.0
Time to Requiring no Phlebotomy Week 36	0.0 month Interval 0.0 to 1.0

SECONDARY outcome

Timeframe: Up to 12 months

Time required to first response is defined as time to achieve complete hematological response (CHR) without phlebotomy.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Time Required to First Response	11.90 month Interval 5.5 to 12.0

SECONDARY outcome

Timeframe: Up to 12 months

Duration of maintained complete hematologic response (CHR) since first achievement of CHR after administration of the study drug will be calculated.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Duration of Response Maintenance Initial response	3.38 month Standard Deviation 2.715
Duration of Response Maintenance Longest response	3.77 month Standard Deviation 2.881

SECONDARY outcome

Timeframe: Up to 12 months

Population: No thrombotic or hemorrhagic events related to PV were observed throughout the study period.

Thrombotic or hemorrhagic events will be recorded any time during the study. The proportion of subjects without thrombotic or hemorrhagic events is defined as the proportion of subjects experienced no thrombotic and hemorrhagic events during the study period (12 months).

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Proportion of Subjects Without Thrombotic or Hemorrhagic Events	0 Participants

SECONDARY outcome

Timeframe: Baseline, 3 months, 6 months, 9 months and 12 months

Population: Quantitative JAK2 measurements at screening, Months 3, 6, 9 and 12 (central laboratory) for subjects who signed consent form. Change of JAK2 allelic burden over time will be assessed.

Quantitative JAK2 measurements at screening, Months 3, 6, 9 and 12 (central laboratory) for subjects who signed consent form. Change of JAK2 allelic burden over time will be assessed.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Change of JAK2 Mutant Allelic Burden Over Time vs. Baseline Baseline	72.1881 percent Standard Deviation 23.30272
Change of JAK2 Mutant Allelic Burden Over Time vs. Baseline Change from baseline at Week 12	-4.9006 percent Standard Deviation 10.14497
Change of JAK2 Mutant Allelic Burden Over Time vs. Baseline Change from baseline at Week 24	-7.4963 percent Standard Deviation 15.72194
Change of JAK2 Mutant Allelic Burden Over Time vs. Baseline Change from baseline at Week 36	-12.4549 percent Standard Deviation 16.32228
Change of JAK2 Mutant Allelic Burden Over Time vs. Baseline Change from baseline at Week 52	-19.1748 percent Standard Deviation 22.64181

SECONDARY outcome

Timeframe: Up to 12 months

Trough concentration is the measured concentration of a drug at the end of a dosing interval at steady state every 2 weeks. Additionally, serum concentration is measured at hours 0, 24, 48, 96 and 168 after administration at Week 0 and Week 28.

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
PK of P1101	50.3 ng/mL Standard Deviation 17.8

OTHER_PRE_SPECIFIED outcome

Timeframe: 0 month, 12 months

Population: In myelographic findings, Patient 001-006 showed improvement with the disappearance of findings considered to be MPN; Patients 001-009 and 006-002 showed no change in pathological findings; and Patient 001-005 showed evidence of fibrosis.

Status of bone marrow histological remission defined as the disappearance of hypercellularity (age-adjusted), trilineage growth (panmyelosis) and absence of \>grade 1 reticulin fibrosis

Outcome measures

Outcome measures
Measure	P1101 n=29 Participants Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Status of Bone Marrow Histological Remission (Optional) Showed no change in pathological findings	2 Participants
Status of Bone Marrow Histological Remission (Optional) Showed improvement with the disappearance of findings considered to be MPN	1 Participants
Status of Bone Marrow Histological Remission (Optional) Showed evidence of fibrosis	1 Participants

Adverse Events

P1101

Serious events: 0 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	P1101 n=29 participants at risk Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.
Skin and subcutaneous tissue disorders Alopecia	55.2% 16/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Eczema	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Pruritus	10.3% 3/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Rash	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Hand dermatitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Hyperkeratosis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Onychoclasis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Skin and subcutaneous tissue disorders Urticaria	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Fatigue	27.6% 8/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Influenza like illness	27.6% 8/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Injection site reaction	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Pyrexia	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Malaise	10.3% 3/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
General disorders Oedema peripheral	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Diarrhoea	31.0% 9/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Dental caries	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Abdominal pain	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Stomatitis	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Abdominal discomfort	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Chronic gastritis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Colitis ischaemic	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Constipation	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Enterocolitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Food poisoning	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Gastritis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Hyperaesthesia teeth	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Gastrointestinal disorders Nausea	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Nasopharyngitis	17.2% 5/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Gastroenteritis	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Hordeolum	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Appendicitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Cystitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Dermatophytosis of nail	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Fungal infection	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Herpes simplex	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Herpes zoster	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Paronychia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Pharyngitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Infections and infestations Pneumonia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Myalgia	17.2% 5/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Arthralgia	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Back pain	13.8% 4/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Bone pain	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Musculoskeletal and connective tissue disorders Musculoskeletal discomfort	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Blood and lymphatic system disorders Anaemia	10.3% 3/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Blood and lymphatic system disorders Haemorrhagic diathesis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Blood and lymphatic system disorders Lymph node pain	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Blood and lymphatic system disorders Neutropenia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Blood and lymphatic system disorders Thrombocytopenia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Eye disorders Dry eye	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Eye disorders Photophobia	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Eye disorders Retinal haemorrhage	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Eye disorders Swelling of eyelid	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Eye disorders Vitreous floaters	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Cardiac disorders Palpitations	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Cardiac disorders Mitral valve incompetence	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Cardiac disorders Supraventricular extrasystoles	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Cardiac disorders Ventricular hypokinesia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Nervous system disorders Dizziness	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Nervous system disorders Headache	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Nervous system disorders Taste disorder	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Psychiatric disorders Insomnia	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Psychiatric disorders Anxiety	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Psychiatric disorders Irritability	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Psychiatric disorders Suicidal ideation	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Endocrine disorders Hypothyroidism	6.9% 2/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Endocrine disorders Silent thyroiditis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Respiratory, thoracic and mediastinal disorders Laryngeal pain	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Respiratory, thoracic and mediastinal disorders Vasomotor rhinitis	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Injury, poisoning and procedural complications Arthropod bite	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Injury, poisoning and procedural complications Chillblains	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Injury, poisoning and procedural complications Heat illness	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Metabolism and nutrition disorders Dyslipidaemia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Metabolism and nutrition disorders Hyperuricaemia	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Hepatobiliary disorders Hepatic function abnormal	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Renal and urinary disorders Hypertonic bladder	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.
Vascular disorders Subgaleal haematoma	3.4% 1/29 • Up to 12 months All AEs reported on or after the date of first dose until 30 days (inclusive) after the last dose of the study drug were to be considered treatment-emergent adverse events (TEAEs) and were to be summarized.

Additional Information

Hiroaki Kawase

PharmaEssentia Japan KK

Phone: +81-3-6910-5103

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place