Ultragenyx Announces Initiation of a Phase 2 Study of KRN23 for Pediatric X-Linked Hypophosphatemia in the US and EU

Ultragenyx Announces Initiation of a Phase 2 Study of KRN23 for Pediatric X-Linked Hypophosphatemia in the US and EU

NOVATO, CA – July 1, 2014 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient screened and enrolled in the Phase 2 study of the human monoclonal anti-FGF23 antibody KRN23 (UX023) in pediatric patients with X-linked hypophosphatemia (XLH). XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness.

Ultragenyx Announces Results from Phase 1/2 Study of KRN23 in X-linked Hypophosphatemia in Adults

Treatment with KRN23 induces a sustained increase in serum phosphorus and increases in bone remodeling markers

Novato, CA— June 24, 2014 — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a multiple-dose study, conducted by Kyowa Hakko Kirin Pharma, Inc (KKP), of the investigational anti-FGF23 monoclonal antibody KRN23 (UX023) in adult patients with X-linked hypophosphatemia (XLH). XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness. The data was presented at the 2014 ICE/ENDO joint meeting of The Endocrine Society and The International Congress of Endocrinology in Chicago.

TESARO Announces Successful Achievement of Primary and All Secondary Endpoints in Third and Final Phase 3 Trial of Rolapitant

  • Achieved Primary Endpoint of Complete Response (CR) in the Delayed Period (24 to 120 Hours) Following Initiation of Chemotherapy
  • Achieved Key Secondary Endpoints of CR in the Acute and Overall Periods
  • Achieved All Secondary Endpoints, Including No Significant Nausea
  • Adverse Event Profile Consistent with Earlier Clinical Trials
  • New Drug Application (NDA) Submission to U.S. FDA On Track for Mid-2014

WALTHAM, Mass., May 12, 2014 (GLOBE NEWSWIRE) — TESARO, Inc. (Nasdaq:TSRO), an oncology-focused biopharmaceutical company, today announced positive top-line results from the third and final Phase 3 trial of rolapitant, an investigational neurokinin-1 (NK-1) receptor antagonist in development for the prevention of chemotherapy-induced nausea and vomiting (CINV). The rolapitant arm in this trial, which enrolled patients receiving cisplatin-based, highly emetogenic chemotherapy (HEC), successfully achieved statistical significance over the standard therapy arm for the primary and all secondary endpoints. The adverse event profile for rolapitant remains consistent with that seen in previous clinical studies.