Phase 2 Trial

Clene Inc (NASDAQ: CLNN) has announced multiple presentations of updated clinical trial results from the Phase 2 RESCUE-ALS and REPAIR trials at the 2022 MDA Clinical & Scientific Conference. RESCUE-ALS Phase 2 trial examined the efficacy, safety, pharmacokinetics, and...

BridgeBio Pharma Inc (NASDAQ: BBIO) has announced data from the Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i), a group of conditions that cause weakness and wasting of the muscles in the arms and legs. The Company plans to engage with...

Scholar Rock Holding Corp (NASDAQ: SRRK) will showcase data from an exploratory analysis of the ambulatory cohort in the Phase 2 TOPAZ trial of apitegromab in individuals with later-onset spinal muscular atrophy (SMA). The TOPAZ study assessed the safety, tolerability, PK/PD, and efficacy of...

Sanofi SA (NASDAQ: SNY) stock slid after its drug candidate, amcenestrant, to fight a common type of breast cancer, failed to slow the progression of the disease in the Phase 2 AMEERA-3 trial. Results from the Phase 2 trial showed that the drug amcenestrant, given as a pill, did not...

Moderna Inc (NASDAQ: MRNA) has dosed the first participant in the Phase 2 study of its omicron-specific bivalent booster candidate (mRNA-1273.214). The vaccine candidate combines Moderna's omicron-specific booster candidate (mRNA-1273.529) and the Moderna COVID-19 vaccine (mRNA-...

The FDA has completed a safety review of Inventiva SA's (NASDAQ: IVA) IND application and signed off Phase 2 combination trial with its lead drug candidate, lanifibranor. The planned Phase 2 trial, LEGEND3, has been designed as a proof-of-concept trial to assess the safety...

Alkermes plc (NASDAQ: ALKS) presented new data from the ongoing phase 1/2 ARTISTRY-1 trial for nemvaleukin alfa, engineered interleukin-2 (IL-2) variant immunotherapy for advanced renal cell carcinoma (RCC). The data were presented at the American Society of Clinical Oncology (ASCO...

Amgen Inc (NASDAQ: AMGN) announced the presentation of efficacy and safety data from the CodeBreaK 100 Phase 1/2 trial in KRAS G12C-mutated advanced pancreatic cancer patients who received Lumakras (sotorasib).  The data will be presented at the American Society of...

ImmunityBio Inc (NASDAQ: IBRX) announced data from its bladder cancer trial (QUILT-3.032) Phase 2/3 study of intravesical BCG plus Anktiva (N-803). The data showed sustained complete response rates in patients with BCG-unresponsive non-muscle-invasive carcinoma in situ (NMIBC CIS)...

BioXcel Therapeutics Inc (NASDAQ: BTAI) announced results from its ongoing Phase 2 trial of BXCL701 in metastatic castration-resistant prostate cancer (mCRPC) patients with either adenocarcinoma or small cell neuroendocrine carcinoma (SCNC) phenotype.  Results will be highlighted in two...

Opthea Limited's (NASDAQ: OPT) OPT-302 data were presented at the Bascom Palmer 19th annual Angiogenesis, Exudation, and Degeneration 2022 Conference.  The data presented was a prespecified subgroup analysis of a Phase 2b dose-ranging study of intravitreal OPT-302 in...

Regenxbio Inc (NASDAQ: RGNX) announced additional interim data from Cohorts 1-3 of Phase 1/2 trial of RGX-121 for patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II),  The data were shared at the 18th Annual WORLDSymposium.   RGX-121 is an...

4D Molecular Therapeutics Inc (NASDAQ: FDMT) has posted updated interim data from the Phase 1/2 trial of 4D-310 in Fabry disease at the 18th Annual WORLDSymposium. Following 4D-310 infusion, mean serum AGA enzyme activity was within, or significantly above, the normal range in all three...

Avrobio Inc's (NASDAQ: AVRO) gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion. The data comes from a Phase 1/2 trial testing the safety and efficacy of AVR-RD-04 for cystinosis who had...

NLS Pharmaceutics Ltd (NASDAQ: NLSP) shares are trading higher after the Company announced to present interim topline data from its Phase 2a trial evaluating its lead product candidate, Quilience (Mazindol ER), for narcolepsy at World Sleep Congress 2022. Narcolepsy is a chronic sleep...