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Efficacy and Safety

Omisirge was evaluated in a global, randomized phase 3 trial across a diverse population of patients

Phase 3 Trial Design
Phase 3 Trial Endpoints
Diagram depicting the different randomized treatment arms in the Omisirge phase 3 trial. Diagram depicting the different randomized treatment arms in the Omisirge phase 3 trial.
* 66% of the patients received a double UCB transplant and 34% received a single UCB transplant.
Inclusion Criteria
  • Age 12-65 years
  • Hematologic malignancies
  • Eligible for allo-HCT
  • No readily available matched related or matched unrelated adult donor

Patients who had haploidentical related donors or syngeneic donors were not excluded.

Patient Demographics
Over 40%
of patients enrolled were
from racially and ethnically
diverse backgrounds
Pie chart depicting the racial/ethnic backgrounds of participants in the Omisirge phase 3 trial. Pie chart depicting the racial/ethnic backgrounds of participants in the Omisirge phase 3 trial.
Diagnosis
  • AML (48%)
  • ALL (33%)
  • MDS (7%)
  • CML (5%)
  • Lymphoma (4%)
  • Rare leukemias (3%)
Risk Index
  • Moderate (42%)
  • High / Very high (34%)
HCT-CI*
  • ≥ 3 (50%)
  • 1-2 (30%)

* HCT-CI: Hemtopoietic cell transplantation-specific comorbidity index

Trial endpoints were selected for their significance to patient outcomes

Timeline of various endpoints studied in the Omisirge phase 3 trial.
Primary Endpoint
  • Time to neutrophil engraftment
Secondary Endpoints
  • Platelet engraftment by 42 days
  • First grade 2/3 bacterial or invasive fungal infections during the first 100 days
  • Days alive and out of hospital in the first 100 days
Additional Endpoints
  • Grade 3 viral infections by 180 days and 1 year following transplantation
  • Non-relapse mortality (NRM) by 210 days following randomization
  • Duration of primary hospitalization
  • Overall survival (OS) at 210 days and 15 months following randomization
  • Health-related quality of life (HRQL)

Omisirge demonstrated faster hematopoietic recovery with fewer infections

Patients transplanted with Omisirge experienced faster neutrophil recovery compared to those in the UCB control group (primary endpoint)

A bar chart comparing neutrophil recovery between Omisirge and standard umbilical cord blood (UCB) transplant. A bar chart comparing neutrophil recovery between Omisirge and standard umbilical cord blood (UCB) transplant.

Patients transplanted with Omisirge had fewer bacterial and fungal infections after transplantation (secondary endpoint)

A bar chart comparing incidence of bacterial and fungal infections after transplantation of Omisirge and standard umbilical cord (UCB) transplant. A bar chart comparing incidence of bacterial and fungal infections after transplantation of Omisirge and standard umbilical cord (UCB) transplant.

* Time to neutrophil recovery was defined as the time from transplantation to the earliest of 3 consecutive measurements on different days with absolute neutrophil counter greater than or equal to 0.5 GI/L assessed with 42 days of follow-up.

† Median time to neutrophil recovery was estimated by the Kaplan-Meier estimator.

Differences between the Omisirge and UCB arms for the Neutrophil Recovery and Bacterial and Fungal Infections endpoints were statistically significant.

Click to see additional endpoints evaluated in the phase 3 trial

Secondary Endpoints
Platelet
Engraftment
by Day 42

55% of patients achieved platelet engraftment by day 42 post-transplant (ITT)

35% of patients achieved platelet engraftment by day 42 post-transplant (ITT)

CI, Confidence interval;
ITT, Intent-to-treat population (n=125);
PP, Per protocol population (n=108)

Days Alive
and Out of
the Hospital
(ITT)

ITT, Intent-to-treat population (n=125)

Additional Endpoints
Non-Relapse
Mortality
Tertiary Endpoint
at 210 days (ITT)*

ITT, Intent-to-treat population (n=125)

* Not statistically significant. The study was
not powered to see a significant difference

Grade 3
Viral Infections
Exploratory Endpoint
by 1 year (ITT)

ITT, Intent-to-treat population (n=125)

Overall
Survival
Exploratory Endpoint
by 15 months (ITT)*

ITT, Intent-to-treat population (n=125)

* Not statistically significant. The study was
not powered to see a significant difference

Health-Related
Quality of Life (HRQL)

Exploratory Endpoint

Patients transplanted with
Omisirge reported clinically
meaningful differences in
health-related quality of life
(HRQL) parameters*

* FACT-G, FACT-BMT, EQ-5D-3L scores

Swipe to see additional endpoints evaluated in the phase 3 trial

Secondary Endpoint

Platelet Engraftment
by Day 42

55% of patients achieved platelet engraftment by day 42 post-transplant (ITT)

35% of patients achieved platelet engraftment by day 42 post-transplant (ITT)

CI, Confidence interval;
ITT, Intent-to-treat population (n=125);
PP, Per protocol population (n=108)

Secondary Endpoint

Days Alive and Out
of the Hospital
(ITT)

ITT, Intent-to-treat population (n=125)

Tertiary Endpoint

Non-Relapse
Mortality
at 210 days (ITT)*

ITT, Intent-to-treat population (n=125)

* Not statistically significant. The study was
not powered to see a significant difference

Exploratory Endpoint

Grade 3
Viral Infections
by 1 year (ITT)

ITT, Intent-to-treat population (n=125)

Exploratory Endpoint

Overall Survival
by 15 months (ITT)*

ITT, Intent-to-treat population (n=125)

* Not statistically significant. The study was
not powered to see a significant difference

Exploratory Endpoint

Health-Related Quality
of Life (HRQL)

Patients transplanted with
Omisirge reported clinically
meaningful differences in
health-related quality of life
(HRQL) parameters*

* FACT-G, FACT-BMT, EQ-5D-3L scores

Omisirge has an established safety profile consistent with the expected adverse events of allo-HCT following conditioning therapy

Adverse Events / Laboratory Abnormalities
Infections / GvHD
CTCAE ≥Grade 3 adverse reactions in ≥10% of patients following transplantation with Omisirge or UCB (PP)
Adverse Events Omisirge, n=52 (%) UCB, n=56 (%)
General disorders and administration site conditions
Pain* 33 18
Mucosal inflammation*† 31 34
Fatigue† 4 21
Fever 2 11
Vascular disorders
Hypertension*† 25 38
Hemorrhage 12 18
Gastrointestinal disorders
Gastrointestinal toxicity*† 19 34
Dysphagia 12 13
Renal and urinary disorders
Renal impairment 12 5
Respiratory, thoracic, and mediastinal disorders
Respiratory failure† 12 30
Dyspnea 8 16

* Most common Grade 3-5 AEs for Omisirge

† Most common Grade 3-5 AEs for UCB

Chemistry laboratory abnormalities in ≥10% of patients (PP)
Laboratory Abnormalities Omisirge, n=52 (%) UCB, n=56 (%)
Grade 1-4 Grade 3-4 Grade 1-4 Grade 3-4
Magnesium decreased 94 4 91 2
Aspartate aminotransferase increased 56 13 61 7
Alanine aminotransferase increased 56 13 57 9
Creatinine increased 50 4 57 2
Bilirubin increased 42 12 61 21
Alkaline phosphatase increased 42 0 54 2
Magnesium increased 15 2 29 9

* Most common Grade 3-5 AEs for Omisirge

† Most common Grade 3-5 AEs for UCB

Infections following transplantation with Omisirge or UCB (PP)
Infections Omisirge, n=52 (%) UCB,
n=56 (%)
Grade 1-3 Grade 2 Grade 3 Grade 1-3 Grade 2 Grade 3
Grade 1-3 2 3 1-3 2 3
Viral 75 48 8 80 32 27
Bacterial 65 27 8 80 46 23
Fungal 21 4 6 27 0 18
Acute and Chronic GvHD post treatment (PP)
GvHD Omisirge, n=52 (%) UCB, n=56 (%)
Grade II-IV acute GvHD 62 43
Grade III-IV acute GvHD 15 21
Chronic GvHD 35 25
Moderate to severe chronic GvHD 23 20

All patients received myeloablative preparative regimens and GvHD prophylaxis with tacrolimus or cyclosporin plus mycophenolate.

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