Developments – Page 2

Developments

Prevail Therapeutics doses first patient in Phase 1/2 frontotemporal dementia trial

Prevail Therapeutics (NASDAQ:PRVL) dosed the first patient in its Phase 1/2 PROCLAIM trial evaluating PR006 for the treatment of frontotemporal dementia patients with GRN mutations. Frontotemporal dementia is the second...

December 11, 2020

Developments

Geron begins enrollment in Phase 3 refractory myelofibrosis trial

Geron (NASDAQ:GERN) reported that its Phase 3 IMpactMF clinical trial, which is evaluating imetelstat for the treatment of refractory myelofibrosis, is open for patient screening and enrollment. The trial is designed to...

December 11, 2020

Developments

Lexicon gets FDA fast track designation for diabetic peripheral neuropathic pain

Lexicon Pharmaceuticals’ (NASDAQ:LXRX) LX9211 received FDA fast track designation for the treatment of diabetic peripheral neuropathic pain. The company is currently enrolling patients with diabetic peripheral...

December 11, 2020

Developments

Hepion completes 75 mg CRV431 dosing, initiates 225 mg dosing in AMBITION trial

Hepion Pharmaceuticals (NASDAQ:HEPA) completed patient dosing in the 75 mg cohort of its Phase 2a ‘AMBITION’ clinical trial, which is evaluating CRV431 for the treatment of non-alcoholic steatohepatitis (NASH). In...

December 10, 2020

Developments

Savara’s AeroVanc fails Phase 3 cystic fibrosis trial

Savara (NASDAQ:SVRA) reported that its Phase 3 AVAIL trial evaluating AeroVanc for the treatment of cystic fibrosis failed to meet its primary endpoint. The trial enrolled 55 cystic fibrosis patients with methicillin...

December 10, 2020

Developments

TG Therapeutics reports positive topline results from 2 Phase 3 MS trials

TG Therapeutics (NASDAQ:TGTX) reported positive topline data from its two Phase 3 trials evaluating ublituximab, an anti-CD20 monoclonal antibody, for the treatment of relapsing forms of multiple sclerosis (MS)...

December 10, 2020

Developments

Pluristem terminates Phase 3 critical limb ischemia trial

Pluristem Therapeutics (NASDAQ:PSTI) reported disappointing results from an interim analysis of its Phase 3 study evaluating PLX-PAD for the treatment of critical limb ischemia. Based on the interim analysis, the study...

December 9, 2020

Developments

Cytokinetics reports positive interim results from Phase 2 hypertrophic cardiomyopathy trial

Cytokinetics (NASDAQ:CYTK) reported positive interim results from the first cohort of its Phase 2 REDWOOD-HCM clinical trial evaluating CK-274 for the treatment of hypertrophic cardiomyopathy. In the first cohort, 21...

December 9, 2020

Developments

BELLUS Health doses first patient in Phase 2b chronic cough trial

BELLUS Health (NASDAQ:BLU; TSX:BLU) dosed the first patient in its Phase 2b study evaluating BLU-5937 for the treatment of refractory chronic cough. The study, called SOOTHE, will evaluate three doses of BLU-5937 in...

December 8, 2020

Developments

electroCore reports positive topline results from migraine prevention study

electroCore (NASDAQ:ECOR) reported positive topline results from its PREMIUM II study evaluating gammaCore, a non-invasive vagus nerve stimulation (nVNS) device, for the prevention of migraines. The study was designed...

December 8, 2020

Developments

Precision BioSciences reports positive interim data from Phase 1/2a lymphoma, leukemia study

Precision BioSciences (NASDAQ:DTIL) reported positive interim results from its Phase 1/2a study evaluating PBCAR0191, an allogeneic chimeric antigen receptor (CAR-T) cell therapy, for the treatment of...

December 4, 2020

Developments

Taysha gets FDA rare pediatric and orphan drug designations for epilepsy

Taysha Gene Therapies’ (NASDAQ:TSHA) TSHA-103 received FDA rare pediatric and orphan drug designations for the treatment of SLC6A1-related epilepsy. SLC6A1-related epilepsy is caused by the loss of function of one copy...

December 3, 2020

Developments

Protagonist Therapeutics gets FDA fast track designation for polycythemia vera

Protagonist Therapeutics’ (NASDAQ:PTGX) PTG-300 received FDA fast track designation for the treatment of polycythemia vera, a rare disease characterized by the excessive production of red blood cells. PTG-300 is an...

December 2, 2020

Developments

Cara Therapeutics completes enrollment in Phase 2 pruritis trial

Cara Therapeutics (NASDAQ:CARA) completed enrollment in its Phase 2 KARE trial evaluating KORSUVA for the treatment of moderate-to-severe pruritus, or itchy skin, in atopic dermatitis patients. The trial enrolled 400...

December 2, 2020

Developments

Axsome reports positive results from Phase 2 treatment-resistant depression trial

Axsome Therapeutics (NASDAQ:AXSM) reported positive results from its Phase 2 COMET-TRD trial evaluating AXS-05 for the treatment resistant depression (TRD). The trial evaluated 70 patients who had ongoing symptoms of...

December 2, 2020

Developments

Catalyst Biosciences gets FDA fast track designation for hemophilia

Catalyst Biosciences’ (NASDAQ:CBIO) activated marzeptacog alfa, or MarzAA, received FDA fast track designation for the treatment of episodic bleeding in subjects with hemophilia A or B who have developed coagulation...

December 2, 2020

Developments

Hepion reports CRV431 may reduce risk of NASH-associated ischemic stroke

Hepion Pharmaceuticals (NASDAQ:HEPA) reported results from a translational research study in which its lead drug candidate, CRV431, decreased formation of procoagulant platelets. The company noted that patients with...

December 2, 2020

Developments

Aeglea BioTherapeutics gets FDA rare pediatric disease designation for homocystinuria

Aeglea BioTherapeutics’ (NASDAQ:AGLE) ACN00177 received FDA rare pediatric disease designation for the treatment homocystinuria. Homocystinuria is a serious metabolic disorder characterized by elevated plasma...

December 1, 2020

Developments

Bellerophon enrolls first patient in Phase 3 INOpulse study

Bellerophon Therapeutics (NASDAQ:BLPH) enrolled the first patient in its Phase 3 REBUILD study evaluating INOpulse, a pulsed nitric oxide system, for the treatment of fibrotic interstitial lung disease (fILD). The study...

December 1, 2020

Developments

Athira initiates dosing in Phase 2 Alzheimer’s disease study

Athira Pharma (NASDAQ:ATHA) initiated patient dosing in its ACT-AD Phase 2 trial evaluating ATH-1017 in patients with mild-to-moderate Alzheimer’s disease (AD). ATH-1017 is a small molecule designed to regenerate...

December 1, 2020

Developments

Moleculin Biotech gets FDA rare pediatric disease designation for three indications

Moleculin Biotech’s (NASDAQ:MBRX) WP1066 received FDA rare pediatric disease indication for the treatment of diffuse intrinsic pontine glioma (DIPG), medulloblastoma and atypical teratoid rhabdoid tumor. WP1066 is an...

December 1, 2020

Developments

Ovid Therapeutics’ OV101 fails Phase 3 Angelman syndrome trial

Ovid Therapeutics (NASDAQ: OVID) reported that its Phase 3 NEPTUNE trial evaluating OV101 for the treatment of Angelman syndrome failed to meet its primary endpoint. Angelman syndrome is a rare genetic disorder that...

December 1, 2020

Prevail Therapeutics doses first patient in Phase 1/2 frontotemporal dementia trial

Geron begins enrollment in Phase 3 refractory myelofibrosis trial

Lexicon gets FDA fast track designation for diabetic peripheral neuropathic pain

Hepion completes 75 mg CRV431 dosing, initiates 225 mg dosing in AMBITION trial

Savara’s AeroVanc fails Phase 3 cystic fibrosis trial

TG Therapeutics reports positive topline results from 2 Phase 3 MS trials

Pluristem terminates Phase 3 critical limb ischemia trial

Cytokinetics reports positive interim results from Phase 2 hypertrophic cardiomyopathy trial

BELLUS Health doses first patient in Phase 2b chronic cough trial

electroCore reports positive topline results from migraine prevention study

Precision BioSciences reports positive interim data from Phase 1/2a lymphoma, leukemia study

Taysha gets FDA rare pediatric and orphan drug designations for epilepsy

Protagonist Therapeutics gets FDA fast track designation for polycythemia vera

Cara Therapeutics completes enrollment in Phase 2 pruritis trial

Axsome reports positive results from Phase 2 treatment-resistant depression trial

Catalyst Biosciences gets FDA fast track designation for hemophilia

Hepion reports CRV431 may reduce risk of NASH-associated ischemic stroke

Aeglea BioTherapeutics gets FDA rare pediatric disease designation for homocystinuria

Bellerophon enrolls first patient in Phase 3 INOpulse study

Athira initiates dosing in Phase 2 Alzheimer’s disease study

Moleculin Biotech gets FDA rare pediatric disease designation for three indications

Ovid Therapeutics’ OV101 fails Phase 3 Angelman syndrome trial

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