Trial Outcomes & Findings for BO-112 With Pembrolizumab in Unresectable Malignant Melanoma (NCT NCT04570332)

Last Updated: 2025-11-26

Results Overview

Percentage of patients achieving a complete response (CR) or partial response (PR) as best overall response, by independent radiological central review (IRCR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by CT or MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

42 participants

Primary outcome timeframe

From the first dose of study treatment to the date of CR or PR assessed up to 2 years

Results posted on

2025-11-26

Participant Flow

Participant milestones

Participant milestones
Measure	Single Arm Patients with advanced Stage III/IV melanoma, refractory or resistant to anti-PD-1 treatment, were treated with the combination of intra-tumoral (IT) BO-112 and Q3W IV pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W).
Overall Study STARTED	42
Overall Study COMPLETED	42
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=42 Participants Patients with advanced Stage III/IV melanoma resistant or refractory to anti-PD-1 treatment were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Age, Categorical <=18 years	0 Participants n=42 Participants
Age, Categorical Between 18 and 65 years	18 Participants n=42 Participants
Age, Categorical >=65 years	24 Participants n=42 Participants
Age, Continuous	64.67 years STANDARD_DEVIATION 15.12 • n=42 Participants
Sex: Female, Male Female	18 Participants n=42 Participants
Sex: Female, Male Male	24 Participants n=42 Participants
Region of Enrollment France	17 Participants n=42 Participants
Region of Enrollment Spain	25 Participants n=42 Participants

PRIMARY outcome

Timeframe: From the first dose of study treatment to the date of CR or PR assessed up to 2 years

Population: Modified intent-to-treat population - patients with baseline and at least one post-baseline assessment. 2 patients from the ITT did not have post-baseline assessment and were excluded from mITT.

Outcome measures

Outcome measures
Measure	Single Arm n=40 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Overall Response Rate	25.0 percentage of participants Interval 12.69 to 41.2

SECONDARY outcome

Timeframe: 2 years and 9 months

The National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), v5.0 was used for grading toxicities.

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Number of Participants With Adverse Events and Serious Adverse Events	42 Participants

SECONDARY outcome

Timeframe: From the first dose of study treatment to the date of best response (i.e. CR, PR, or SD) on study up to 2 years

Population: 2 participants did not have post-baseline imagining and were excluded from the mITT analysis.

Participants with complete response (CR), or partial response (PR), or stable disease (SD) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR) and modified RECIST 1.1 for immune-based therapeutics (iRECIST)

Outcome measures

Outcome measures
Measure	Single Arm n=40 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Disease Control Rate	67.5 percentage of participants Interval 50.87 to 81.43

SECONDARY outcome

Timeframe: From the date of CR or PR until the date of event or censoring up to 2 years

Population: ITT

Median DOR (CR or PR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR) by IRCR

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Duration of Response	NA months Interval 8.31 to Median DOR could not be determined by the end of the study due to insufficient number of participants with events

SECONDARY outcome

Timeframe: From the first dose of study treatment to the date of radiologic progression or death, whichever comes first, up to 2 years

Population: ITT

Progressive disease (PD) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR) by IRCR or death

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Progression Free Survival	3.71 months Interval 2.17 to 9.23

SECONDARY outcome

Timeframe: From the first dose of study treatment to death from any cause up to 2 years

Population: ITT population

Median overall survival for all patients treated with at least one dose of the study drug

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants Patients were treated with the combination of IT BO-112 and pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W); pembrolizumab Q3W was administered IV.
Overall Survival	NA months Interval 9.89 to Median could not be determined at the time of study end due to insufficient number of participants with events

Adverse Events

Single Arm

Serious events: 15 serious events

Other events: 42 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm n=42 participants at risk Patients with advanced Stage III/IV melanoma, refractory or resistant to anti-PD-1 treatment, were treated with the combination of intra-tumoral (IT) BO-112 and Q3W IV pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W).
Nervous system disorders Haemorrhage intracranial	2.4% 1/42 • Number of events 1 • 2 years and 9 months
General disorders Disease progression	2.4% 1/42 • Number of events 1 • 2 years and 9 months
General disorders General physical health deterioration	7.1% 3/42 • Number of events 3 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Pain in extremity	4.8% 2/42 • Number of events 2 • 2 years and 9 months
Infections and infestations Device related infection	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Infections and infestations Sepsis	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Injury, poisoning and procedural complications Infusion related reaction	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lymphangiosis carcinomatosa	2.4% 1/42 • Number of events 1 • 2 years and 9 months
General disorders Condition aggravated	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Nervous system disorders Epilepsy	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Nervous system disorders Cerebral hematoma	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Arthralgia	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Vascular disorders Hypertension	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Gastrointestinal disorders Vomiting	2.4% 1/42 • Number of events 1 • 2 years and 9 months
General disorders Asthenia	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Gastrointestinal disorders Abdominal pain	2.4% 1/42 • Number of events 1 • 2 years and 9 months
Respiratory, thoracic and mediastinal disorders Tracheal compression	2.4% 1/42 • Number of events 1 • 2 years and 9 months

Other adverse events

Other adverse events
Measure	Single Arm n=42 participants at risk Patients with advanced Stage III/IV melanoma, refractory or resistant to anti-PD-1 treatment, were treated with the combination of intra-tumoral (IT) BO-112 and Q3W IV pembrolizumab. IT administration of BO-112 was performed once weekly (QW) for the first 7 weeks and then once every three weeks (Q3W).
General disorders Asthenia	69.0% 29/42 • Number of events 75 • 2 years and 9 months
General disorders Pyrexia	45.2% 19/42 • Number of events 90 • 2 years and 9 months
Gastrointestinal disorders Diarrhoea	42.9% 18/42 • Number of events 30 • 2 years and 9 months
Gastrointestinal disorders Nausea	31.0% 13/42 • Number of events 32 • 2 years and 9 months
Gastrointestinal disorders Vomiting	31.0% 13/42 • Number of events 27 • 2 years and 9 months
General disorders Chills	28.6% 12/42 • Number of events 25 • 2 years and 9 months
Nervous system disorders Headache	26.2% 11/42 • Number of events 17 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Pain in extremity	26.2% 11/42 • Number of events 14 • 2 years and 9 months
Gastrointestinal disorders Decreased apetite	23.8% 10/42 • Number of events 13 • 2 years and 9 months
General disorders Injection site pain	23.8% 10/42 • Number of events 12 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Arthralgia	21.4% 9/42 • Number of events 14 • 2 years and 9 months
Skin and subcutaneous tissue disorders Pruritus	21.4% 9/42 • Number of events 17 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Back pain	19.0% 8/42 • Number of events 13 • 2 years and 9 months
General disorders Oedema peripheral	16.7% 7/42 • Number of events 9 • 2 years and 9 months
Skin and subcutaneous tissue disorders Rash	16.7% 7/42 • Number of events 11 • 2 years and 9 months
Gastrointestinal disorders Constipation	14.3% 6/42 • Number of events 7 • 2 years and 9 months
Respiratory, thoracic and mediastinal disorders Cough	14.3% 6/42 • Number of events 6 • 2 years and 9 months
Vascular disorders Hypertension	14.3% 6/42 • Number of events 8 • 2 years and 9 months
Psychiatric disorders Insomnia	14.3% 6/42 • Number of events 6 • 2 years and 9 months
Musculoskeletal and connective tissue disorders Myalgia	14.3% 6/42 • Number of events 9 • 2 years and 9 months
Investigations Aspartate aminotransferase increased	11.9% 5/42 • Number of events 6 • 2 years and 9 months
Infections and infestations COVID-19	11.9% 5/42 • Number of events 7 • 2 years and 9 months
Infections and infestations Influenza like illness	11.9% 5/42 • Number of events 12 • 2 years and 9 months
Gastrointestinal disorders Dry mouth	9.5% 4/42 • Number of events 4 • 2 years and 9 months
General disorders General physical health deterioratio	9.5% 4/42 • Number of events 4 • 2 years and 9 months
Gastrointestinal disorders Abdominal pain	7.1% 3/42 • Number of events 3 • 2 years and 9 months
Endocrine disorders Adrenal insufficiency	7.1% 3/42 • Number of events 5 • 2 years and 9 months
Investigations Alanine aminotransferase increased	7.1% 3/42 • Number of events 3 • 2 years and 9 months
Blood and lymphatic system disorders Anemia	7.1% 3/42 • Number of events 4 • 2 years and 9 months
Investigations Blood alkaline phosphatase increased	7.1% 3/42 • Number of events 6 • 2 years and 9 months
Eye disorders Eye pruritus	7.1% 3/42 • Number of events 3 • 2 years and 9 months
Gastrointestinal disorders Fatigue	7.1% 3/42 • Number of events 5 • 2 years and 9 months
Investigations Gamma-glutamyltransferase increased	7.1% 3/42 • Number of events 4 • 2 years and 9 months
Gastrointestinal disorders Stomatitis	7.1% 3/42 • Number of events 4 • 2 years and 9 months
Nervous system disorders Tremor	7.1% 3/42 • Number of events 5 • 2 years and 9 months
Eye disorders Vision blurred	7.1% 3/42 • Number of events 3 • 2 years and 9 months

Additional Information

M. Quintero, CEO

Highlight Therapeutics Ltd.

Phone: +34686219355

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place