Phase 2 Trial

Seagen Inc (NASDAQ: SGEN) announced full results from the phase 2 MOUNTAINEER trial, which showed Tukysa (tucatinib) combined with trastuzumab was well-tolerated with durable responses in patients with previously treated HER2-positive metastatic colorectal cancer (mCRC). ...

BioRestorative Therapies Inc (NASDAQ: BRTX) has selected the Center for Clinical Research in North Carolina as the company's second contract for its Phase 2 trial targeting chronic lumbar disc disease (cLDD).  BioRestorative develops therapeutic products using cell and tissue...

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced topline safety and efficacy data from its Phase 2 trial of RP-L201 gene therapy in severe Leukocyte Adhesion Deficiency-I (LAD-I). Data were shared at the American Society of Gene and Cell Therapy. LAD-I is a disorder that...

Applied Genetic Technologies Corp (NASDAQ: AGTC) reported three-month interim data from its ongoing Phase 2 Skyline trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP), an inherited condition causing progressive vision loss in boys and young men.  AGTC-501 is a...

Urovant Sciences' interim results from a Phase 2a trial of URO-902 gene therapy were presented at the annual meeting of the American Urological Association (AUA2022).  Urovant is a wholly-owned subsidiary of Sumitovant Biopharma Ltd. Sumitovant, the Sumitomo Pharma subsidiary, is...

Rezolute Inc (NASDAQ: RZLT) unveiled results from its Phase 2b RIZE study of RZ358 in patients with congenital hyperinsulinism (abnormally high insulin levels) at the Pediatric Endocrine Society 2022 Annual Meeting.  RZ358 showed a better than 50% reduction from baseline in...

Kazia Therapeutics Limited (NASDAQ: KZIA) has completed a Phase 2 study of paxalisib in glioblastoma. Kazia has received a final clinical study report, and an abstract summarizing the study results has been accepted for presentation at an upcoming international clinical oncology...

Windtree Therapeutics Inc (NASDAQ: WINT) has announced positive primary results with istaroxime in rapidly raising systolic blood pressure. Istaroxime is a first-in-class dual mechanism therapy designed to improve systolic and diastolic cardiac function.  Windtree is advancing multiple...

Acadia Pharmaceuticals Inc (NASDAQ: ACAD) announced topline results from a Phase 2 trial evaluating the efficacy and safety of ACP-044 for acute pain following bunionectomy surgery.  The primary endpoint, a comparison of cumulative pain intensity scores over 24 hours between the ACP-044...

Results from the Phase 1/2 dose-finding study evaluating Sanofi SA's (NASDAQ: SNY) rilzabrutinib in adults with heavily pre-treated immune thrombocytopenia (ITP) were published in the New England Journal of Medicine.  Results demonstrate that rilzabrutinib treatment led to...

Genmab A/S (NASDAQ: GMAB) and AbbVie Inc (NYSE: ABBV) announced topline results from the first cohort of the EPCORE NHL-1 phase 1/2 trial of epcoritamab (DuoBody-CD3xCD20).  Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab's DuoBody...

Longeveron Inc (NASDAQ: LGVN) has announced a Cooperative Research and Development Agreement (CRADA) with the United States Department of Veterans Affairs (VA) to add Miami VA clinical site to its ongoing Phase 2 Alzheimer’s Disease (AD) trial.  Why It Matters: This marks the...

AbbVie Inc (NYSE: ABBV) has announced new data from a Phase 2 trial of navitoclax combined with ruxolitinib in myelofibrosis patients at the American Association for Cancer Research annual meeting.  The results presented at AACR 2022 were from REFINE Phase 2 trial, which...

Repare Therapeutics Inc (NASDAQ: RPTX) presented updated data from its ongoing Phase 1/2 TRESR trial of RP-3500 for solid tumors with specific synthetic-lethal genomic alterations. The data were featured at the AACR Annual Meeting included monotherapy Phase 1 (Module 1) results from...

IO Biotech Inc (NASDAQ: IOBT) announced updated efficacy data and subgroup analyses from the MM1636 Phase 1/2 trial evaluating IO102-IO103 combined with Bristol Myers Squibb Co's (NYSE: BMY) Opdivo (nivolumab) in metastatic melanoma. The data showed that the three-year...