Category - Developments

Developments

AVROBIO doses first patient in Phase 1/2 gene therapy trial for cystinosis

AVROBIO (NASDAQ:AVRO) dosed the first patient in its Phase 1/2 clinical trial of AVR-RD-04 gene therapy for the treatment of cystinosis. Caused by a mutation in the cystinosin gene, cystinosis is a lysosomal storage...

October 8, 2019

Developments

FDA approves Pfenex osteoporosis drug

The FDA approved Pfenex’s (NASDAQ:PFNX) PF708 for the treatment of osteoporosis in patients at high risk of fracture. “The FDA’s approval of PF708 marks a major milestone in Pfenex’s history as our first approved...

October 8, 2019

Developments

Journals publish mechanism of action data for BeyondSpring’s Plinabulin

Two peer-reviewed scientific journals, Chem and Cell Reports, published new data that sheds light on the mechanism of action of BeyondSpring’s (NASDAQ:BYSI) lead Phase 3 asset, Plinabulin, which is being developed in...

October 8, 2019

Developments

Salarius adds two clinical sites to seclidemstat trial in Ewing Sarcoma

Salarius Pharmaceuticals (NASDAQ:SLRX) added Memorial Sloan Kettering Cancer Center in New York City and Nationwide Children’s Hospital in Columbus, OH as clinical trial sites in its ongoing Phase 1/2 clinical trial of...

October 8, 2019

Developments

Ritter Pharma engages AGP to explore strategic alternatives

Ritter Pharmaceuticals (NASDAQ:RTTR) has engaged Alliance Global Partners as a financial advisor to help the company evaluate strategic alternatives. The company is considering a variety of options, including an...

October 7, 2019

Developments

BriaCell doses first patient in advanced breast cancer combination study

BriaCell Therapeutics (TSXV:BCT; OTCQB:BCTXF)initiated dosing in a Phase 1/2a clinical study of its lead product candidate, Bria-IMT, in combination with Incyte’s INCMGA00012 and epacadostat in patients with advanced...

October 7, 2019

Developments

Ra Pharma doses first patient in pivotal Phase 3 myasthenia gravis study

Ra Pharmaceuticals (NASDAQ:RARX) dosed the first patient in its RAISE study, a pivotal Phase 3 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis (gMG). gMG is a chronic autoimmune...

October 2, 2019

Developments

ArQule doses first patient in registrational trial of miransertib

ArQule (NASDAQ:ARQL) dosed the first patient in its MOSAIC trial evaluating miransertib for the treatment of Proteus syndrome (PS) and phosphoinositide 3 kinase (PIK3CA)-related overgrowth spectrum disorders (PROS). PS...

October 2, 2019

Developments

FDA rejects Savara’s Molgradex for autoimmune pulmonary alveolar proteinosis

Following a Type C FDA meeting, Savara (NASDAQ:SVRA) reported that the agency did not recommend it submit a biologics license application (BLA) for Molgradex for the treatment of autoimmune pulmonary alveolar...

October 2, 2019

Developments

LumiThera enrolls first patient in U.S. multi-center trial in dry AMD

Closely-held LumiThera enrolled the first patient in its multi-center U.S. clinical trial in patients with dry age-related macular degeneration (AMD). The study, called LIGHTSITE III, will evaluate LumiThera’s Valeda...

October 1, 2019

Category - Developments

AVROBIO doses first patient in Phase 1/2 gene therapy trial for cystinosis

FDA approves Pfenex osteoporosis drug

Journals publish mechanism of action data for BeyondSpring’s Plinabulin

Salarius adds two clinical sites to seclidemstat trial in Ewing Sarcoma

Ritter Pharma engages AGP to explore strategic alternatives

BriaCell doses first patient in advanced breast cancer combination study

Ra Pharma doses first patient in pivotal Phase 3 myasthenia gravis study

ArQule doses first patient in registrational trial of miransertib

FDA rejects Savara’s Molgradex for autoimmune pulmonary alveolar proteinosis

LumiThera enrolls first patient in U.S. multi-center trial in dry AMD

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