Category - Developments

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

Category - Developments

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

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