Zokinvy™▼ increases the survival probability of patients with HGPS1

Zokinvy (lonafarnib) increases the survival probability of patients with HGPS compared with untreated patients from the natural history cohort.1

The efficacy of Zokinvy is based on the results of two phase 2 studies – ProLon1 and ProLon2. These studies were combined into a pooled analysis to evaluate the differences in survival between HGPS patients treated with Zokinvy and untreated patients matched from a historical cohort.2

The survival analysis showed that HGPS patients treated with Zokinvy have a higher probability of survival at 1, 3 and 5 years than untreated patients from the natural history cohort.1

Table 1: Survival analysis summary for patients with Hutchinson-Gilford progeria syndrome (lonafarnib treated versus external natural history cohort)

Difference in RMST* in years (95%-CI)
0.466 (0.204, 0.728) P1+P2
0.414 (0.042, 0.785) P1
0.172 (-0.101, 0.445) P2
Hazard ratio* (95%-CI)
0.28 (0.107, 0.756) P1+P2
0.15 (0.017, 1.263) P1
0.71 (0.199, 2.556) P2
Difference in RMST* in years (95%-CI)
4.338 (2.551, 6.126) P1+P2
Hazard ratio* (95%-CI)
0.28 (0.154, 0.521) P1+P2
Difference in RMST* in years (95%-CI)
0.237 (0.074, 0.401) P1+P2
Hazard ratio* (95%-CI)
0.29 (0.097, 0.838) P1+P2
Difference in RMST* in years (95%-CI)
0.094 (0.034, 0.154) P1+P2
Hazard ratio* (95%-CI)
0.20 (0.054, 0.732) P1+P2

CI = confidence interval; P1 = ProLon1; P2 = ProLon2; RMST = restricted mean survival time
There were 27 patients in ProLon1 and 35 patients in ProLon2.
* Estimates are based on matching as follows: for each lonafarnib patient a random match untreated patient was selected with the same sex and same continent.
Lonafarnib patients were matched sequentially from the lonafarnib patient with oldest age at start to the youngest. The age at start of treatment of the untreated patient within a matched pair was set to that of the lonafarnib patient. If an untreated patient had a longer follow-up than the lonafarnib treated patient in a matched pair, then this follow-up was censored at the length of the follow-up of the lonafarnib treated patient. Regression analysis for the RMST and Cox proportional hazard regression for the hazard ratio had sex and continent as stratification factors and age at start of treatment as covariate.

At the last follow up (August 2021) the mean lifespan for HGPS patients treated with Zokinvy increased by an average of 4.3 years (range: 2.551-6.126 ) vs. untreated patients.1

Did you know?

Treatment with Zokinvy resulted in increased restricted mean survival time up to 4.3 years vs. untreated patients.1

Study overview

Pro-Ion1:

  • A phase 2, open-label, single-arm trial that evaluated the efficacy and safety of Zokinvy (lonafarnib) in 28 patients (26 with classic HGPS, 1 with nonclassic HGPS and 1 with PL)
  • Patients received lonafarnib over 24 to 30 months
  • Patients initiated treatment with lonafarnib at 115 mg/m2 twice daily
  • After 4 months of treatment, patients who tolerated treatment had an increase in dosage to 150 mg/m2 twice daily
  • Among the 28 patients treated, 27 patients with progeria were included in the survival assessment (16 females, 11 males)
  • The median age at treatment initiation for the 27 patients was 7.5 years (range, 3 to 16 years)

Pro-lon2:

  • A phase 2, open-label, single-arm trial involving 35 patients (34 with classic HGPS, 1 with non-classic HGPS) to evaluate the efficacy and safety of Zokinvy (lonafarnib)
  • Patients received lonafarnib over 12 to 36 months
  • All 35 treated patients were included in the survival analysis
  • The median age at the start of treatment was 6.0 years (range, 2 to 17 years)

Get information on the safety profile of Zokinvy

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References

1. Data on file, Eiger Biopharmaceuticals.
2. Zokinvy Summary of Product Characteristics 2022