Clinical Results

Trailblazing trial design: LYFGENIA™ is the result of the longest-studied gene therapy program in SCD

LYFGENIA was evaluated in 2 clinical trials1

LYFGENIA was evaluated in 2 clinical trials1

The safety of LYFGENIA was based on patients with sickle cell disease in 1 open-label, single-arm clinical trial (Study 1: HGB-206) and 1 long-term follow-up study (LTF-307).1,3 Study 1 started in February 2015.

The efficacy of LYFGENIA was studied in a single-arm, 24-month, open-label, multicenter Phase 1/2 study (Study 1-C).1

In Study 1-C, 32 patients with a history of at least 4 VOEs in the 24 months prior to informed consent were evaluated for VOEs.1

LYFGENIA Safety and Efficacy

See experts lead an in-depth review of the clinical trial, efficacy data, and safety information for LYFGENIA

Transformational results. One-time treatment. Lasting impact.

LYFGENIA is a one-time transformational SCD gene therapy with the potential to decrease or stop VOEs.

[[Vaso-occlusive events (VOEs)1]]

[[Severe vaso-occlusive events (sVOEs)1]]

[[Vaso-occlusive events (VOEs)1]]

Primary Endpoint1

88% of patients achieved complete resolution of vaso-occlusive events

[[Severe vaso-occlusive events (sVOEs)1]]

Secondary Endpoint1

94% of patients achieved complete resolution of severe vaso-occlusive events

Follow-up Time1

Median duration of follow-up of 38 months

LYFGENIA is the only approved SCD gene therapy supported by a study design that used a fixed efficacy assessment window that was predefined, ensuring patients were evaluated for (s)VOEs between 6-18 months after infusion.1,4

• (s)VOE-CR was defined as the elimination of (s)VOEs between 6 and 18 months post infusion with LYFGENIA1
• In Study 1-C, 36 patients were infused with LYFGENIA (transplant population)1
• 32 of the 36 patients had a history of at least 4 VOEs in the 2 years prior to informed consent and were evaluated for VOEs1
• After the primary evaluation period to last follow-up, 4 of 28 patients who achieved VOE-CR experienced VOEs while maintaining globin response1
No clinically meaningful differences in efficacy or safety were observed between the adult and pediatric subgroups.1
 

LYFGENIA delivered powerful and lasting reduction in VOEs

Vaso-occlusive events before and after LYFGENIA infusion5

Graph showing presence of vaso-occlusive events 24 months before enrollment and 18 months after LYFGENIA infusion
Graph showing presence of vaso-occlusive events 24 months before enrollment and 18 months after LYFGENIA infusion

sVOEs were also counted as VOEs.
This figure represents patient-level data and is not included in the USPI.

Vaso-occlusive events before and after LYFGENIA infusion5

Graph showing presence of vaso-occlusive events 24 months before enrollment and 18 months after LYFGENIA infusion
Graph showing presence of vaso-occlusive events 24 months before enrollment and 18 months after LYFGENIA infusion

sVOEs were also counted as VOEs.
This figure represents patient-level data and is not included in the USPI.

 

Vaso-Occlusive Events (VOEs)1

In the LYFGENIA study, vaso-occlusive events (VOEs) were defined as any of the following events requiring evaluation at a medical facility:

  • an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours
  • acute chest syndrome
  • acute hepatic sequestration
  • acute splenic sequestration

Severe Vaso-Occlusive Events (sVOEs)1

In the LYFGENIA study, severe vaso-occlusive events (sVOEs) were defined as vaso-occlusive events (VOEs) requiring either of the following:

  • a hospitalization
  • multiple visits to an emergency department/urgent care over 72 hours and receiving IV medications at each visit
  • priapism requiring any level of medical attention
Neurologic

STUDY 1-C

The only approved SCD gene therapy with stroke outcome data included in the Prescribing Information

Demonstrated resilience in stroke patients — 5 patients remained stroke-free after LYFGENIA

Demonstrated resilience in stroke patients — 5 patients remained stroke-free after LYFGENIA

Five patients (≥18 years of age) in Study 1-C with a history of stroke or vasculopathy who were on chronic transfusion therapy prior to LYFGENIA infusion remained transfusion-independent and without recurrent stroke at 44–60 months follow-up.1

The only approved SCD gene therapy with stroke outcome data included in the Prescribing Information1,4

 

Stable. Durable. Proven.

LYFGENIA maintained stable and durable HbAT87Q levels from month 6 through month 48.1

All 36 patients infused in Study 1-C (transplant population) were evaluated for globin response. 31/36 (86%) achieved globin response. All patients who achieved globin response maintained it.1

[[Globin Response]]

Achieved Globin Response1

(n=31/36)

All members of the transplant population (n=36) were evaluated for globin response

86% of patients achieved globin response

Achieved Globin Response1

(n=31/36)

All members of the transplant population (n=36) were evaluated for globin response

86% of patients achieved globin response

Patients Who Achieved Globin Response
Maintained It1

(n=31/31)

Of patients that achieved globin response, 100% maintained it

Patients Who Achieved Globin Response
Maintained It1

(n=31/31)

Of patients that achieved globin response, 100% maintained it

Globin response, a secondary endpoint of the LYFGENIA clinical trials, was defined as meeting the following criteria for a continuous period of at least 6 months after LYFGENIA infusion:

  • Weighted average hemoglobin AT87Q percentage of non-transfused total Hb ≥30% AND1
  • Weighted average non-transfused total Hb (HbS + HbF + HbA2 + HbAT87Q) increase of ≥3 g/dL compared to baseline total Hb OR weighted average non-transfused total Hb ≥10 g/dL1

Median concentration of total Hb after LYFGENIA infusion6

Graph showing the median concentration of total hemoglobin after LYFGENIA infusion
Graph showing the median concentration of total hemoglobin after LYFGENIA infusion

Total Hb consists of HbS, HbAT87Q, fetal hemoglobin (HbF), and HbA2

Other Hb includes HbS, HbF, and HbA2

This data is from a secondary efficacy endpoint; however, there was no formal hypothesis testing.

Patients reported improvement in pain and fatigue7

At 36 months (n=20), more than half of patients reported clinically meaningful improvements in the following measures7:

57% of patients reported improvement in pain intensity
64% of patients reported clinically meaningful improvement in fatigue
64% of patients reported pain reduction

This exploratory analysis includes a subset of adult Study 1-C patients with available baseline and follow-up health-related quality of life (HRQOL) data (PROMIS-57).7

No efficacy conclusions should be drawn from these data.

Take a closer look at LYFGENIA

Back
to Top