The clinical evidence of VIJOICE has been established across a median of 3.5 years and up to 6 years of treatment^1,2

Data from EPIK-P1 and EPIK-P3 were pooled (EPIK-P1 + EPIK-P3) and described for each patient from treatment start date in EPIK-P1 to the end of the EPIK-P3 retrospective period. Only EPIK-P1 data are included for patients who discontinued during EPIK-P1 or did not participate in EPIK-P3.²

Median duration of treatment with VIJOICE in the full population (N=57) was 42.0 months (range: 3.4-74.8 months)²

• 22.8% of patients were treated more than
60 months

• Despite the extended duration of treatment (median 43.3 months [range: 3.4-64.4]), pediatric patients continued to experience normal growth trajectory during treatment with VIJOICE

VIJOICE shrunk overgrowth (EPIK-P1)³

Durable response: The median duration of response was not reached as of the data cutoff. The median time to censoring for this analysis was 63 weeks (range: 1 day to 187 weeks).³

Response was defined as the proportion of patients achieving a ≥20% reduction from baseline in the sum of measurable target lesion volume (1 to 3 lesions) confirmed by at least 1 subsequent imaging assessment, provided that: none of the individual target lesions had a ≥20% increase from baseline, nontarget lesions had not progressed, and there were no new lesions, as determined by BICR.

In the pediatric and adult subgroups:

• 30.4% (7/23) of pediatric patients experienced a response,^*,† and 68.2% (15/22) experienced any reduction in the sum of target lesion volume

• 55.6% (5/9) of adult patients experienced a response,^*,† and 88.8% (8/9) experienced any reduction in the sum of target lesion volume

^*Confirmed response as determined by BICR.        
^†Patients without any response assessment at Week 24 were considered nonresponders.

Percentage of patients with improvements at the end of the pooling period in the most commonly reported signs and symptoms

Improvement was defined based on CTCAE grade reduction or resolution of the event. Percentages were calculated based on the number of patients who reported the event at baseline.

CTCAE, Common Terminology Criteria for Adverse Events.

EPIK-P1 + EPIK-P3

VIJOICE provided improvements in pediatric and adult PROS signs and symptoms that lasted through the longer-term analysis²

Pediatric patients (<18 years of age)²

In the longer-term pooled analysis of EPIK-P1 and EPIK-P3, pediatric patients experienced improvements in fatigue (100.0%, 28/28), vascular malformations (92.0%, 23/25), disseminated intravascular coagulation (89.5%, 17/19), lipomatosis (88.2%, 15/17), limb asymmetry (76.5%, 13/17), scoliosis (64.7%, 11/17), and other signs and symptoms.

Adult patients (≥18 years of age)²

In the longer-term pooled analysis of EPIK-P1 and EPIK-P3, adult patients experienced improvements in fatigue (100.0%, 14/14), limb asymmetry (100.0%, 12/12), vascular malformations (92.3%, 12/13), pain (90.0%, 9/10), disseminated intravascular coagulation (80.0%, 8/10), and other signs and symptoms.

EPIK-P1 + EPIK-P3

VIJOICE reduced the number of surgeries for most pediatric and adult patients²

Pediatric patients (<18 years of age)² 

Three pediatric patients required progression-related surgery during the longer-term evaluation² 

• Twelve pediatric patients (30.8%) underwent PROS-related surgeries after treatment initiation; 15 surgeries were performed

  • Since the start of the analysis, 3 patients underwent surgeries due to disease progression

  • Six surgeries were performed due to disease improvement

  • Seven surgeries were performed for other reasons 

Adult patients (≥18 years of age)² 

No adult patients required any PROS-related surgery during the longer-term evaluation²

• Two adult patients (11.1%) underwent PROS-related surgeries after treatment initiation, with 3 surgeries performed

  • One of the 3 surgeries was due to disease improvement; the other 2 were for other reasons

In EPIK-P1 + EPIK-P3

VIJOICE improved functionality that lasted through the longer-term analysis²

In EPIK-P1

At least* 50% (24/47) of patients experienced improved or stable function at Week 24³

*The performance status score was not reported for 23 of the 47 patients at Week 24.

In EPIK-P1 + EPIK-P3

Approximately 81% (38/47) of patients had an improved score by at least 20 points at the last available post-index assessment²

The median score at the index date was 70.0 (range: 20-100).

Performance status was recorded at baseline for 47 patients (33 pediatric patients; 14 adult patients) using ECOG, Karnofsky, and Lansky assessments.³ 

For the ECOG^† scale, improvement is defined as a decrease by at least 1 point and worsening is defined as an increase by at least 1 point. For the Karnofsky and Lansky^‡ scales, improvement is defined as an increase by at least 20 points, worsening is defined as a decrease by at least 20 points, and stable is defined as a change in score between -20 and 20 points.^2,3

No other scales were reported for change in score. Change in score is only applicable if both index and post-index date information are available.
Patients may have started with Lansky at index and switched to Karnofsky at a later time.  
^†ECOG scales assess level of functioning by measuring a patient’s daily activity, physical ability, and ability to care for oneself.^5  
‡Lansky and Karnofsky scales assess a patient’s ability to perform normal activities of daily living and functional status.^5,6  
ECOG, Eastern Cooperative Oncology Group.