Human Trials

Roche’s risdiplam meets primary endpoint in phase 3 SMA trial

Roche said that part 2 of the SUNFISH trial assessing risdiplam for the treatment of Type 2 or 3 spinal muscular atrophy (SMA) in people aged 2-25 years has met its primary endpoint.

Roxadustat significantly increased hemoglobin levels for chronic kidney disease patients with anemia in phase III OLYMPUS and ROCKIES trials

AstraZeneca today presented detailed results from the Phase III OLYMPUS and ROCKIES trials showing that roxadustat significantly increased hemoglobin (Hb) levels in both non-dialysis-dependent (NDD) and dialysis-dependent (DD) patients with anemia from chronic kidney disease (CKD), respectively.

Takeda’s dengue vaccine candidate demonstrates protection in children ages four to 16 years

Takeda Pharmaceutical announced that results from the primary endpoint analysis of the ongoing pivotal Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial of its dengue vaccine candidate (TAK-003) were published in the New England Journal of Medicine.

Axcella achieves initial milestones with its hematology product candidate AXA4010

Axcella Health, a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, announced key milestones in the development of AXA4010, the company’s first investigational hematology product candidate.

Halozyme’s PEGPH20 pancreas cancer drug candidate fails in HALO-301 trial

Halozyme Therapeutics has discontinued the development of PEGylated recombinant human hyaluronidase (PEGPH20) after the investigational drug failed to meet the primary endpoint in a phase 3 trial in metastatic pancreas cancer.

BioCryst begins phase 1 trial with BCX9250 for treatment of fibrodysplasia ossificans progressiva

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company has begun a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva (FOP).

Novartis’ Cosentyx delivers mixed results in EXCEED trial in active PsA

Novartis’ EXCEED phase 3b trial, which has been evaluating Cosentyx (secukinumab) in active psoriatic arthritis (PsA) in comparison to Humira (adalimumab), has yielded mixed results.

Daiichi Sankyo initiates clinical trial of DS-7300 in collaboration with Sarah Cannon Research Institute

Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and Sarah Cannon Research Institute (Sarah Cannon) announced today that the first patient has been dosed in a first-in-human phase 1/2 study evaluating DS-7300, an investigational B7-H3 targeting antibody drug conjugate (ADC), in patients with various advanced solid tumors that have progressed on standard treatments or for whom no standard treatment exists.

ACADIA Pharmaceuticals initiates phase 3 pivotal study of trofinetide in Rett Syndrome

ACADIA Pharmaceuticals, a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system (CNS) disorders, today announced that it has initiated the Phase 3 LAVENDER placebo-controlled study to evaluate the efficacy and safety of trofinetide for girls and young women with Rett syndrome.