Company

Product

Description

Indication

Status

Phase I

Constellation Pharmaceuticals Inc., of Cambridge, Mass.

CPI-0209

Enhancer of zeste homolog 2 inhibitor

Solid tumors

Dosed first participant in phase I/II trial beginning with escalation phase to evaluate CPI-0209 as monotherapy in advanced, relapsed disease

Kadimastem Ltd., of Ness Ziona, Israel

Astrorx

Astrocytes derived from human embryonic stem cells

Amyotrophic lateral sclerosis

Interim results from cohort A (n = 5) of phase I/IIa trial showed no serious adverse events or dose-limiting toxicities 3 months post treatment with lowest dose (100x106 cells); muscle function, measured by ALS Functional Rating Scale-Revised, showed average change of +0.26 per month, or statistically significant difference between pre- and post-treatment slope (p=0.0023)

Oncolys Biopharma Inc., of Tokyo

Telomelysin (OBP-301)

Telomerase modulator

Esophageal cancer

6 participants received study drug in combination with radiation; data safety monitoring board determined that no adverse event required withdrawal of the drug

Replicel Life Sciences Inc., of Vancouver, British Columbia

RCS-01

Autologous cell therapy

Aging and sun-damaged skin

Findings from single-center safety study were published in Skin Pharmacology and Physiology; intradermal injections were well-tolerated for up to 1 year post-injection and study established biological link between the treatment and changes in expression of particular genes in the skin

Salarius Pharmaceuticals Inc., of Houston

Seclidemstat

Lysine specific histone demethylase 1 inhibitor

Ewing sarcoma, advanced solid tumors

Safety review committees overseeing open-label dose-finding phase I/II study in Ewing sarcoma and phase I study in AST approved their advancement to fourth level dosing cohort

Soricimed Biopharma Inc., of Toronto

SOR-C13

TRP cation channel V6 inhibitor

Solid tumors

Dosing initiated in first cohort of 3 participants with late-stage pancreatic cancer in investigator-initiated phase Ib trial

Phase II

Lidds AB, of Uppsala, Sweden

Liproca Depot

Androgen receptor antagonist

Hormone-dependent prostate cancer

Preliminary results from phase IIb dose-finding study showed strong maximum prostate-specific antigen (PSA) decrease and sustained PSA reduction, with maximum reduction during months 2-4

Neuralstem Inc., Germantown, Md.

NSI-566

Human neural stem cells

Chronic ischemic stroke

Enrollment completed with 22 participants randomized to treatment or sham surgery, who will be evaluated for efficacy measures for 12 months following surgery

Takeda Pharmaceutical Co. Ltd., of Osaka, Japan

TAK-620 (maribavir)

Serine threonine protein kinase UL97 inhibitor

Cytomegalovirus infection

The New England Journal of Medicine published results from 12-week study in individuals with CMV infection following hematopoietic cell transplant or solid organ transplant; 62% treated with any dose of maribavir showed effect within 3 weeks of treatment vs. 56% with valganciclovir; at 6 weeks, response rates were 79% and 67%, respectively

Urogen Pharma Ltd., of New York

UGN-102

Sustained-release mitomycin C gel

Bladder cancer

Trial completed enrollment; among interim cohort of 32 patients, 63% achieved a complete response

Phase III

Emmaus Life Sciences Inc., of Torrance, Calif.

Endari (levoglutamide)

Oral solution of L-glutamine

Sickle cell disease

Study showed 25% lower frequency of painful crises (p=0.005), 33% lower frequency of hospitalization (p=0.005) and > 60% decrease in frequency of acute chest syndrome among those treated with Endari (p=0.003)

Ocular Therapeutix Inc., of Bedford, Mass.

Dextenza (dexamethasone)

Glucocorticoid receptor agonist

Allergic conjunctivitis

First of about 80 participants dosed in trial evaluating safety and efficacy of study drug vs. placebo vehicle punctum plug; primary efficacy endpoint is ocular itching following insertion of Dextenza at multiple time points during 30-day period

Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy

PRX-102 (pegunigalsidase alfa)

Alpha-galactosidase stimulator

Fabry disease

Completed enrollment of 24-month Balance trial evaluating drug compared to agalsidase beta (Fabrazyme, Sanofi SA/Genzyme Corp.) on renal function in participants with Fabry and progressing kidney disease previously treated with agalsidase beta; outcome measures include rate of renal function deterioration as well as cardiac assessment, Lyso-Gb3, pain, quality of life, immunogenicity and clinical events

Urogen Pharma Ltd., of New York

UGN-101

Sustained-release mitomycin C gel

Ureter cancer

Pivotal Olympus trial showed UGN-101 demonstrated a 59% complete response rate in patients with low-grade upper tract urothelial cancer; estimated median time to recurrence was 13 months

Urovant Sciences Ltd., of Irvine, Calif.

Vibegron

Oral beta-3 adrenergic receptor agonist

Overactive bladder

Double-blind extension of Empowur study found vibegron further improved key OAB symptoms over 40-week extension period; 41% of patients on vibegron became "dry"; NDA filing planned by early 2020

Zealand Pharma A/S, of Glostrup, Denmark

Dasiglucagon

Glucagon receptor agonist; glucagon ligand

Hypoglycemia

Median time to blood glucose recovery was 10 minutes for dasiglucagon vs. 30 minutes for placebo (p<0.001); median time to recovery for Glucagen (Novo Nordisk A/S) was 10 minutes

Notes

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