Company

Product

Description

Indication

Status

Phase I

Noxopharm Ltd., of Sydney

Veyonda (idronoxil)

Suppository dosage formulation of synthetic flavonoid derivative

End-stage, metastatic, castration-resistant prostate cancer

Interim results from phase Ib Darrt-1 study showed Veyonda combined with externally delivered radiotherapy had durable anticancer response rate lasting at least 6 months in a high proportion of men

Otonomy Inc., of San Diego

OTO-413

Sustained-exposure formulation of brain-derived neurotrophic factor

Hearing loss

Started a single ascending-dose phase I/II trial to test safety and exploratory efficacy in patients with speech-in-noise hearing difficulty

Poxel SA, of Lyon, France

PXL-065

Deuterium-stabilized R-stereoisomer of pioglitazone, targets mitochondrial pyruvate carrier inhibition

Metabolic disorders, including type 2 diabetes and nonalcoholic steatohepatitis

Started phase Ib study in healthy subjects to test safety, tolerability and pharmacokinetics as well as support dose selection for pivotal study; trial also aims to confirm predicted relative exposure and dose proportionality; top-line data expected in fourth quarter of 2019

Revolution Medicines Inc., of Redwood City, Calif.

RMC-4630

Inhibits activity of SHP2

Relapsed/refractory solid tumors harboring specific genomic mutations

Dosed first patient in open-label phase Ib/II dose-escalation and dose-expansion study in combination with Cotellic (cobimetinib, Roche Holding AG)

Sensorion SA, of Montpellier, France

SENS-111

Histamine type 4 receptor antagonist

Inner ear diseases

Study in healthy volunteers showed no sedative effects and consequences on vigilance, cognition and working memory vs. placebo, confirmed by Vigtrack and Pepsy tests; data confirmed by patients' self-assessed measures on Stanford Sleepiness Scale

Phase II

Acceleron Pharma Inc., of Cambridge, Mass.

ACE-083

Targets TGF-beta protein superfamily

Facioscapulohumeral muscular dystrophy

Treatment did not achieve functional secondary endpoints; though drug demonstrated statistically significant increase in mean total muscle volume, the primary endpoint, the increase failed to translate to statistically significant improvements in functional tests; Acceleron will not conduct further clinical trials of ACE-083 in FSHD

Concert Pharmaceuticals Inc., of Lexington, Mass.

CTP-543

Oral JAK1/2 inhibitor

Alopecia areata

Completed patient enrollment of open-label trial testing once-daily 24-mg dosing vs. twice-daily 12-mg dosing; top-line data expected in first half of 2020

Durect Corp., of Cupertino, Calif.

DUR-928

Epigenetic regulator

Alcoholic hepatitis

Completed phase IIa trial, showing that 19 patients treated with DUR-928 had statistically significantly greater reductions from baseline in bilirubin (day 7 and 28) and Model of End-Stage Liver Disease score (day 28), as well as statistically significantly lower Lille scores vs. historical control group

Generex Biotechnology Corp., of Miramar, Fla.

AE-37

Peptide vaccine

Metastatic triple-negative breast cancer

Enrolled first patient in trial to test safety and tolerability of AE-37 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.), as well as objective response rate

Leap Therapeutics Inc., of Cambridge, Mass.

DKN-01

Anti-Dickkopf-1 antibody

Advanced gynecological malignancies

Findings from P204 study showed, as of data cutoff, 1 patient with Wnt signaling alterations had her monotherapy partial response deepen into complete response; across study, patients with Wnt activating mutations demonstrated longer progression-free survival (n=21, 175 days) vs. those without Wnt activating mutations (n=67, 63 days); patients with DKK1-high tumors had longer PFS

Oramed Pharmaceuticals Inc., of New York

ORMD-0801

Oral insulin capsule

Type 2 diabetes

Treated last patient in primary cohort of phase IIb HbA1c trial; top-line data expected in fourth quarter of 2019

Solasia Pharma KK, of Tokyo

Darinaparsin

Mitochondrial targeted agent

Relapsed or refractory peripheral T-cell lymphoma

Patient registration reached target number of cases in Japan, South Korea, Taiwan and Hong Kong; pivotal study is testing monotherapy, with tumor response as primary outcome measure; results expected in 2020

Phase III

Aclaris Therapeutics Inc., of Wayne, Pa.

A-101 45% topical solution

High concentration of topical hydrogen peroxide solution

Common warts (verruca vulgaris)

Thwart-2 study met primary endpoint (p<0.0001), with higher proportion of subjects having all identified common warts reported as clear at day 60 vs. placebo; all secondary endpoints achieved statistical significance, including complete clearance at day 137 (p=0.0001), mean per subject percent of treated warts cleared at day 137 (p<0.0001) and time to complete clearance of all warts (p<0.0001); data from second trial, Thwart-1, expected in fourth quarter of 2019

Centrexion Therapeutics Corp., of Boston

CNTX-4975

Synthetic, ultra-pure intra-articular injection of trans-capsaicin

Chronic moderate to severe knee osteoarthritis pain

Completed patient enrollment in third trial, Victory-3, involving 857 patients with pain in 1 or both knees; top-line results expected in first quarter of 2020

Novartis AG, of Basel, Switzerland

Cosentyx (secukinumab)

Inhibits interleukin-17A

Non-radiographic axial spondyloarthritis

Data showed ongoing Prevent trial met primary endpoint of ASAS40 at week 16, showing significant and clinically meaningful reduction in disease activity vs. placebo

Novo Nordisk A/S, of Bagsvaerd, Denmark

Fiasp

Fast-acting insulin aspart

Advanced type 2 diabetes

Data showed that, for people not optimally controlled on basal-bolus regimen, Fiasp reduced overall blood sugar levels vs. conventional insulin aspart; patients on Fiasp also reported superior reduction in 1-hour post-mean blood sugar increment vs. those on conventional insulin aspart; Fiasp showed statistically significantly lower rate of severe or blood sugar-confirmed hypoglycemia vs. conventional insulin aspart

Novo Nordisk A/S, of Bagsvaerd, Denmark

Ozempic (once-weekly semaglutide)

GLP-1 analogue

Type 2 diabetes

Results from 2 head-to-head trials showed Ozempic was superior to SGLT2 inhibitor canagliflozin in reducing HbA1c and body weight in people uncontrolled on metformin in Sustain 8 trial and superior to Victoza (liraglutide) in reducing HbA1C and body weight in patients in Sustain 10 study; data published in The Lancet Diabetes & Endocrinology

Novo Nordisk A/S, of Bagsvaerd, Denmark

Oral semaglutide

GLP-1 analogue

Type 2 diabetes

Exploratory analyses from phase IIIa Pioneer program showed 3-mg, 7-mg and 14-mg doses improved glycemic control across baseline HbA1c levels; greater reductions in HbA1c were shown with 7-mg and 14-mg doses vs. all comparators, including placebo, Jardiance (empagliflozin 25 mg), Januvia (sitagliptin 100 mg) or Victoza (liraglutide 1.8 mg)

Rafael Pharmaceuticals Inc., of Cranbury, N.J.

CPI-613 (devimistat)

Targets mitochondrial tricarboxylic acid cycle

Metastatic adenocarcinoma of the pancreas

Expanded Avenger 500 trial into France; pivotal study is evaluating CPI-613 in combination with modified FOLFIRINOX as first-line therapy

Notes

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