Ultragenyx Announces Positive Interim Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7

Rapid and Sustained Reduction in Urinary Substrate Excretion Observed Over 12 Weeks

NOVATO, Calif., Sept. 3, 2014  -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of positive interim data from the Phase 1/2 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome). The data are being presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Innsbruck, Austria.

Infinity And AbbVie Announce Global Strategic Collaboration To Develop And Commercialize Duvelisib (IPI-145) In Oncology

- Infinity to Receive $275 Million Upfront Payment and $530 Million in Potential Milestones -
- Companies to Jointly Develop and Commercialize Duvelisib in U.S., with Equal Profit Share; Infinity to Receive Royalties on Sales Outside the U.S.

CAMBRIDGE, Mass. and NORTH CHICAGO, Ill., Sept. 3, 2014  -- Infinity Pharmaceuticals, Inc. (INFI) and AbbVie Inc. (ABBV) today announced that they have entered into a global collaboration to develop and commercialize duvelisib (IPI-145), Infinity's oral inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, for the treatment of patients with cancer. Duvelisib has shown clinical activity across a broad range of blood cancers, including indolent non-Hodgkin lymphoma (iNHL) and chronic lymphocytic leukemia (CLL). Infinity is conducting registration-focused trials evaluating the safety and efficacy of duvelisib, including DYNAMO^TM, a Phase 2 study in patients with iNHL, and DUO^TM, a Phase 3 study in patients with CLL.

Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease

NEW YORK & CARMIEL, Israel-- Pfizer Inc. (PFE) and Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX, TASE:PLX) announced today that the U.S. Food and Drug Administration (FDA) approved ELELYSO™ (taliglucerase alfa) for injection for pediatric patients. ELELYSO is therefore now indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.

Androxal(R) Achieves Superiority in Top Line Analysis for Both Co-Primary Endpoints and Various Secondary Endpoints Versus Marketed Topical Gel in First of Two Identical Studies in the Treatment of Secondary Hypogonadism

• 23.8% of Topical T subjects below 10 million sperm/mL at end of study versus 2.3% and 2.4% respectively for Androxal^(R) and placebo ITT (Androxal^(R) vs. Topical T, p = 0.0031)
• 33.3% reduction from baseline sperm concentration for Topical T subjects versus 5.9% increase for Androxal^(R) subjects ITT (p=0.0004)