Phase 1 Trial

Calithera Biosciences Inc (NASDAQ: CALA) shared interim safety and efficacy results from a Phase 1b trial evaluating CB-280, the company's investigational arginase inhibitor, in adults with cystic fibrosis (CF).  The data were shared at the North American Cystic Fibrosis...

Mustang Bio Inc (NASDAQ: MBIO) has been awarded a grant of approximately $2 million from the National Cancer Institute of the National Institutes of Health (NIH).  The two-year award will partially fund the Phase 1 trial to assess the safety, tolerability, and efficacy of MB-...

Recently listed HCW Biologics Inc (NASDAQ: HCWB) shares almost doubled during premarket Thursday, after FDA signed off Phase 1b trial of its lead candidate in pancreatic cancer. The drug candidate, HCW9218, is an injectable, fusion protein complex designed to drive bifunctional...

TFF Pharmaceuticals Inc (NASDAQ: TFFP) has received approval from Health Canada to begin a Phase 1 trial of a dry powder formulation of niclosamide, an antiviral treatment with the potential to address COVID-19 and other infectious diseases. TFF Pharmaceuticals anticipates dosing to...

Edgewise Therapeutics Inc (NASDAQ: EWTX) has announced topline results from the MAD portion of Phase 1 trial of EDG-5506, designed to protect skeletal muscle fibers in Duchenne muscular dystrophy (DMD) and Becker Muscular Dystrophy (BMD). EDG-5506 was shown to be generally well-...

Nurix Therapeutics Inc (NASDAQ: NRIX) announced initial data from Phase 1a trial of NX-2127 in relapsed or refractory B cell malignancies. Initial data from the first two completed cohorts of 100 mg and 200 mg, which included six patients, showed BTK levels in peripheral blood...

Provention Bio Inc (NASDAQ: PRVB) has announced interim results from the PROVENT Phase 1 trial of PRV-101. PRV-101 is a polyvalent inactivated coxsackievirus B (CVB) vaccine candidate targeting all five key CVB strains associated with type 1 diabetes (T1D) autoimmunity. ...

4D Molecular Therapeutics Inc (NASDAQ: FDMT) has announced interim data from the Phase 1/2 trial of intravenous 4D-310 in Fabry disease patients. The Company said that 4D-310 demonstrated a manageable safety profile with the initial evidence of clinical activity. The Company said...

Sio Gene Therapies Inc (NASDAQ: SIOX) has announced interim data from its Phase 1/2 study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis. GM1 gangliosidosis is a genetic disorder that progressively destroys nerve cells in...

Zai Lab Limited (NASDAQ: ZLAB) announced topline results from Phase 1b study of a topical formulation of ZL-1102 in adults with mild-to-moderate chronic plaque psoriasis (CPP). ZL-1102 is an investigational, novel human VH antibody fragment targeting the IL-17A cytokine. In efficacy...

AstraZeneca Plc (NASDAQ: AZN) has paused an active Phase 1 trial of AZD5991, a direct inhibitor of MCL-1, citing a potential safety issue. Two years ago, the FDA hit Amgen Inc (NASDAQ: AMGN) with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity....

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced interim data updates from seven patients from the RP-L201 Phase 1/2 trial for Leukocyte Adhesion Deficiency-I (LAD-I). Data were shared at the Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). RP-L201...

Dermata Therapeutics Inc (NASDAQ: DRMA) announced topline results from its Phase 1b trial of DMT310 for mild-to-moderate psoriasis.  The percentage of treatment responders in Physician's Global Assessment (PGA) with a score of 0 or 1 was 29.6% at Week 8. PGA is a scoring system used...

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) has announced Day 90 data for the first patient from the Phase 1/2 trial of VX-880 for type 1 diabetes (T1D).  VX-880 is an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy. The...

Repare Therapeutics Inc (NASDAQ: RPTX) announced the presentation of preliminary Phase 1 monotherapy data from its Phase 1/2 TRESR trial of RP-3500 for solid tumors with specific synthetic-lethal genomic alterations. Data were presented at the AACR-NCI-EORTC Virtual International...