sialic acid extended release (SA-ER) / Ultragenyx, Nobelpharma - LARVOL DELTA

Machine learning approaches for assessing medication transfer to human breast milk. (PubMed, J Pharmacokinet Pharmacodyn) - "Further validation and refinement by incorporating larger datasets can enhance their reliability and applicability. Advancing these techniques can support safer medication use and informed clinical decision-making for lactating individuals."

Journal

Efficacy Confirmation Study of NPC-09 (clinicaltrials.gov) - P3 | N=14 | Completed | Sponsor: Nobelpharma | Active, not recruiting ➔ Completed | N=10 ➔ 14

Enrollment change • Trial completion • Myositis

Efficacy Confirmation Study of NPC-09 (clinicaltrials.gov) - P3; N=10; Active, not recruiting; Sponsor: Nobelpharma; Recruiting ➔ Active, not recruiting

Enrollment closed • Myositis

Efficacy Confirmation Study of NPC-09 (clinicaltrials.gov) - P3; N=10; Recruiting; Sponsor: Nobelpharma; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Myositis

Efficacy Confirmation Study of NPC-09 (clinicaltrials.gov) - P3; N=10; Not yet recruiting; Sponsor: Nobelpharma

Clinical • New P3 trial • Myositis

Ultragenyx reports third quarter 2014 financial results and corporate update (TWST) - "The FDA agreed with the proposed Phase 3 study design, including the primary endpoint of a composite of upper extremity muscle strength, with supportive secondary endpoint data from a patient-reported outcome such as the GNEM-FAS (GNE myopathy-functional activity scale), both of which were studied in the Phase 2 trial. The company plans to initiate the Phase 3 trial by mid-2015."

Anticipated new P3 trial • Inflammation • Myositis

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (clinicaltrials.gov) - P3; N=89; Completed; Sponsor: Ultragenyx Pharmaceutical Inc; Active, not recruiting ➔ Completed

Trial completion • Biosimilar • Immunology • Inflammation • Myositis

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (clinicaltrials.gov) - P3; N=80; Active, not recruiting; Sponsor: Ultragenyx Pharmaceutical Inc; Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Biosimilar • Myositis

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid (ER Tablets + IR Capsules) in Patients With GNE Myopathy (clinicaltrials.gov) - P2; N=59; Completed; Sponsor: Ultragenyx Pharmaceutical Inc; Active, not recruiting ➔ Completed

Trial completion • Biosimilar • Immunology • Inflammation • Myositis

"#Ultragenyx Announces Top-Line Results from Ph3 Study of Ace-ER in #GNE_Myopathy https://t.co/9ynfkngBaO @eurordis @RareDiseases" (@CheckOrphan)

Biosimilar