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Demonstrated favorable results in both pharmacodynamic and functional measures1

When it comes to efficacy, Lamzede has favorable results in several important disease categories.1

Trial 1 (rhLAMAN-05) results show the benefit of Lamzede for patients with alpha-mannosidosis, especially over time and in pediatric patients2

Studied in an even distribution of pediatric and adult patients1

Patients were 6-17 years old (pediatric) and 18-35 years old (adult), with confirmed diagnosis of alpha-mannosidosis.

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Lamzede achieved significant pharmacodynamic response1

Patients receiving Lamzede showed a favorable response in both pharmacodynamic and functional measures.

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Favorable results in prioritized secondary endpoints1

Patients receiving Lamzede showed favorable results in endurance walking and pulmonary function.

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secondary endpoints

Study design

Trial 1 (rhLAMAN-05) was a Phase III, international, multicenter, double-blind, randomized, placebo-controlled parallel-group trial which assessed Lamzede in patients with alpha-mannosidosis.2

Efficacy and safety assessed in 25 patients with this ultra-rare disease.2

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Eligibility criteria included confirmed α-mannosidase activity and no history of bone marrow transplantation2

Inclusion criteria1,2

  • Confirmed α-mannosidase activity (<10% of normal activity in blood leukocytes)
  • Ability to cooperate physically and mentally in trial assessments

Exclusion criteria2

  • Presence of known chromosomal abnormality and syndromes, other than alpha-mannosidosis
  • Inability to walk without support (the use of walking aids/wheelchair for partial support and longer distances was permitted)
  • History of bone marrow transplantation
  • Any psychotic disease, active or in remission
  • Total immunoglobulin E (lgE) >800 IU/mL

Eligibility was not limited by motor performance at baseline.

Demonstrated favorable response vs placebo in both co-primary endpoints.1

Co-primary endpoints2

  • Serum oligosaccharides change from baseline to week 52
  • 3-minute stair-climbing test (3MSCT)

Reduction in a key marker of impaired cellular function1

  • Improvements at 6 months that continued at 1 year

Serum oligosaccharides2,3
(95% CI: -4.4, -2.6)

Lamzede Placebo
Baseline (SD) 6.8 (1.2) 6.6 (1.9)
Mean relative
change % (SD)

Lamzede reduced oligosaccharides by 75.8%.1

Maintained endurance climbing stairs, as measured by the 3MSCT1

  • Patients who received placebo declined over time

(95% CI: -3.8, 9.1)

Lamzede Placebo
Baseline (SD) 52.9 (11.2) 55.5 (16.0)
Mean relative
change % (SD)

Lamzede patients showed a slight increase in steps per minute after 1 year.1,*

*Differences in 3MSCT were not statistically significant.

Trial 1 (rhLAMAN-05) prioritized secondary endpoints showed favorable results in measures of functional capacity vs placebo1

(95% CI: -30.7, 45.5)

Lamzede Placebo
Baseline (SD) 459.6 (72.3) 465.7 (140.5)
Mean relative
change % (SD)

Differences in 6MWT were not statistically significant.

(95% CI: -5.0, 16.1)

Lamzede Placebo
Baseline (SD) 81.7 (20.7) 90.4 (10.4)
Mean relative
change % (SD)

Differences in FVC test were not statistically significant.

Differences in 6MWT were not statistically significant.

Differences in FVC test were not statistically significant.