LYFGENIA™ is a one-time transformational gene therapy with the potential to decrease or stop VOEs.1*

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

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

Most evaluable individuals (88%; 28/32 individuals) did not experience any VOEs between 6-18 months after treatment.1

*After the primary evaluation period to last follow-up, 4 of 28 patients who achieved complete resolution of VOEs experienced VOEs.1

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Primary Endpoint Post-infusion Neurologic Outcomes Globin Response

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Primary Endpoint
Post-infusion
Neurologic Outcomes
Globin Response
After LYFGENIA infusion almost all evaluable patients achieved complete resolution of VOEs (VOE-CR) and severe VOEs (sVOE-CR)1
Vaso-occlusive events (VOEs)1

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 hours1
  • acute chest syndrome (ACS)1
  • acute hepatic sequestration1
  • acute splenic sequestration1
Vaso-occlusive events (VOEs)1

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 hours1
  • acute chest syndrome (ACS)1
  • acute hepatic sequestration1
  • acute splenic sequestration1

Primary Endpoint1

Achieved VOE-CR

[n=28/32 (95% CI: 71,97]

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

Primary Endpoint1

Achieved VOE-CR

[n=28/32 (95% CI: 71,97]

88% of patients achieved complete resolution of vaso-occlusive events
Severe vaso-occlusive events (sVOEs)1

Severe VOEs (sVOEs) were defined as either of the following events:

  • VOE requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving intravenous medications at each visit1
  • priapism requiring any level of medical attention1
Severe vaso-occlusive events (sVOEs)1

Severe VOEs (sVOEs) were defined as either of the following events:

  • VOE requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving intravenous medications at each visit1
  • priapism requiring any level of medical attention1

Secondary Endpoint1

Achieved sVOE-CR

[n=30/32 (95% CI: 79, 99]

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

Secondary Endpoint1

Achieved sVOE-CR

[n=30/32 (95% CI: 79, 99]

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

AFTER INFUSION1

Duration of Follow-up1

[(min 12, max 61) n=36]

Median of

38

Months

AFTER INFUSION1

Duration of
Follow-up1

[(min 12, max 61) n=36]

Median of

38

Months

The efficacy outcomes were VOE-CR and sVOE-CR which were defined as the elimination of (s)VOEs between 6 and 18 months post-infusion with LYFGENIA.1

After the primary evaluation period to last follow-up, 4 of 28 patients who achieved VOE-CR experienced VOEs while maintaining globin response.1

Vaso-occlusive events before and after LYFGENIA infusion4

Graph showing presence of vaso-occlusive events before and 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
infusion4

Graph showing presence of vaso-occlusive events before and 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

STUDY 1-C

Neurologic outcomes1

Neurologic outcomes1

Five patients with a history of stroke or vasculopathy were treated in Study 1-C. All were at least 18 years old and on chronic transfusion therapy prior to LYFGENIA infusion. At 44-60 months’ follow-up, all five subjects remain transfusion independent without recurrent stroke.

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LYFGENIA maintained stable and durable HbAT87Q
levels from month 6 through month 481

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

Globin response was defined as meeting the following criteria for a continuous period of at least 6 months after drug product infusion:

  • weighted average hemoglobin A 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/dL.1

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 the 86% of patients that achieved globin response, 100% achieved globin response and maintained it

Patients Who Achieved Globin Response &
Maintained It1

(n=31/31)

Of the 86% of patients that achieved globin response, 100% achieved globin response and 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 infusion5

Median concentration of total Hb after LYFGENIA infusion5

Graph showing the mediation concentration of total hemoglobin after LYFGENIA infusion
Graph showing the mediation concentration of total hemoglobin after LYFGENIA infusion
Graph showing the mediation concentration of total hemoglobin after LYFGENIA infusion
Graph showing the mediation concentration of total hemoglobin after LYFGENIA infusion
Graph showing the mediation concentration of total hemoglobin after LYFGENIA infusion
Graph showing the mediation 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.

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