Trieste, Italy – A single treatment with avigbagene parvec, an investigational gene therapy developed by Spur Therapeutics, may help improve bone health in adults living with Gaucher disease, according to new clinical trial data. The findings come from the completed Phase 1/2 GALILEO-1 trial and its ongoing long-term extension study,...
treatment News
Basel — New results from a one-year Phase III study have confirmed that the investigational biological therapy Ilaris® (canakinumab, formerly ACZ885)[*] produced rapid and sustained remission of symptoms in the majority of children and adults with a rare and potentially life-threatening auto-inflammatory disease called cryopyrin-associated periodic syndrome (CAPS)[1],[2],[3]. The study...
Primary central nervous system lymphoma (PCNSL) is a rare and aggressive lymphoma with no approved treatment in the U.S.1 In the Phase 2 PROSPECT Study, tirabrutinib demonstrated an overall response rate of 67%, complete response rate of 44%, and a manageable safety profile2 OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters:...
OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; “Ono”) today announced that Ono submitted an application for the manufacturing and marketing approval of ripretinib (DCC-2618), developed by Deciphera Pharmaceuticals, Inc. (“Deciphera”), for the indication of “gastrointestinal stromal tumor that has progressed following cancer chemotherapy.” This...
OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”) announced that efficacy signals for ONO-2808, a S1P5 receptor agonist, were observed in the interim analysis of the Phase 2 clinical trial (ONO-2808-03 study) which assessed the safety and efficacy of ONO-2808 compared to...
PRINCETON, N.J. & ALAMEDA, Calif. — Bristol Myers Squibb and Exelixis, Inc. today announced four-year follow-up results from the CheckMate -9ER trial evaluating Opdivo® (nivolumab) in combination with CABOMETYX® (cabozantinib) vs. sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). Results continued to show superior progression-free...
By Louise Rodino-Klapac – PhD President of Research & Development at Sarepta Therapeutics Cambridge, Mass. – Momentum in Duchenne muscular dystrophy was palpable at the Muscular Dystrophy Association Scientific Congress last month. With nearly 180 abstracts focused on Duchenne alone, spanning pre-clinical research to post-market experience, the field is at...
SOUTH SAN FRANCISCO, Calif. — Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, today announced that OPN-2853, a bromodomain and extra-terminal motif (BET) small molecule inhibitor, has been granted Orphan Drug Designation (ODD) for the treatment of myelofibrosis (MF) by the U.S....
SAN DIEGO, Calif. — Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer therapeutics, presented promising preclinical data in two programs, OPN-6602, a dual EP300/CBP inhibitor in multiple myeloma, and OPN-9840, an oral, non-covalent TEAD inhibitor in malignant mesothelioma and metastatic melanoma. Data were...
RESEARCH TRIANGLE PARK, N.C. — Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, today announced a strategic partnership with the Global RDH12 Alliance (the Alliance) to advance Opus’ gene therapy program...