Company

Product

Description

Indication

Status

Phase I

Beigene Ltd., of Beijing

Tislelizumab

Anti-PD-1 antibody

Solid tumor

Preliminary data from patients with microsatellite instability-high or mismatch repair-deficient solid tumors found objective response rate of 29% in 4 patients out of 25-patient cohort; median duration of response still maturing

Cardurion Pharmaceuticals Inc., of Cambridge, Mass.

CRD-733

PDE9 inhibitor

Heart failure

FDA accepted the IND, and a study in healthy volunteers was initiated

Cell Medica Ltd., of London

CMD-501

Autologous CAR-NKT therapy

Neuroblastoma

Dosed first patient in GINAKIT2, a first-in-human, dose-escalation evaluation in children with relapsed or refractory high-risk neuroblastoma

Exicure Inc., of Skokie, Ill.

AST-008

Spherical nucleic acid

Carcinoma

Doses up to 20 µg/kg found safe and well-tolerated; treatment-related AEs were short-lived, reversible and consistent with TLR9 activation; elicited high levels of certain cytokines and activation of important effector cells; preliminary data from phase Ib trial expected in late 2019

Galectin Therapeutics Inc., of Norcross, Ga.

GR-MD-02

Galectin-3-targeting polysaccharide

Melanoma

Investigator-initiated trial with Providence Cancer Institute found GR-MD-02 + Keytruda (pembrolizumab, Merck & Co. Inc.) yielded objective response rate and duration of response rate higher than expected with Keytruda alone

Nanobiotix SA, of Paris

NBTXR-3

Hafnium oxide crystal nanoparticles

Metastatic head and neck cancer

Strengthening of NBTXR-3 data showing the potential impact on survival in elderly and frail patients with locally advanced head and neck cancers; biomarker analysis and new preclinical data support NBTXR-3 mechanism of action in immune-oncology and use in combination with immune checkpoint inhibitors

Oxurion NV, of Leuven, Belgium

THR-687

Pan-RGD integrin antagonist

Diabetic macular edema

Enrolled the first patient; open-label, dose-escalation study will test single intravitreal injection; up to 18 patients to be enrolled

Phase II

Aivita Biomedical Inc., of Irvine, Calif.

Root of cancer

Patient-specific treatment consisting of autologous dendritic cells loaded with autologous antigens from patient's tumor-initiating cells

Advanced ovarian cancer

Dosed the first 10 patients in the 99-patient trial; treatment is administered via series of injections along with standard of care

Amygdala Neurosciences Inc., of San Francisco

ANS-6637

Selective ALDH2 inhibitor

Opioid use disorder

SEARCH studies, sponsored by the National Institute of Allergy and Infection Diseases with $12.4 million in funding, will test safety, efficacy and underlying mechanisms of craving reduction as a strategy to prevent opioid misuse, dependence and relapse, and improve the morbidity and reduce mortality

Cocrystal Pharma Inc., of Atlanta

CC-31244

Oral, non-nucleoside inhibitor

Hepatitis C virus

Completed patient enrollment in the phase IIa study for ultra-short treatment of HCV patients

ISR Immune System Regulation Holding AB, of Stockholm

ISR-048

GnRH agonist

HIV

Enrolled first patient in phase IIa study testing ISR-048 plus antiretroviral therapy (ART) vs. ART alone

Oxurion NV, of Leuven, Belgium

THR-317

Anti-PIGF antibody

Macular telangiectasia type 1

Enrolled the first patient in the study testing efficacy and safety of intravitreally administered drug, assessed via functional and anatomic endpoints

Phase III

Astellas Pharma Inc., of Tokyo

Roxadustat

Inhibits HIF prolyl hydroxylase activity

Chronic kidney disease with anemia

ALPS study met primary endpoints, showing superiority vs. placebo in both hemoglobin response rate in the first 24 weeks and Hb change from baseline at weeks 28 to 52

Erytech Pharma SA, of Paris

Eryaspase

L-asparaginase encapsulated inside donor-derived red blood cells

Second-line metastatic pancreatic cancer

First 3 patients enrolled in the pivotal TRYbeCA1 trial; 500-patient trial is testing eryaspase in combination with chemotherapy vs. chemotherapy alone; primary endpoint is overall survival

Innovent Biologics Inc., of Suzhou, China

Sintilimab

Fully human anti-PD-1 antibody

First-line nonsquamous non-small-cell lung cancer

Data from cohort D in combination with pemetrexed and cisplatin showed an objective response rate of 68.4% based on 19 patients with at least 1 radiological assessment among 21 patients total; as of data cut-off, median duration of response, progression-free survival and overall survival have not been reached

Mesoblast Ltd., of Melbourne, Australia

Remestemcel-L

Allogeneic mesenchymal stem cell candidate

Steroid-refractory acute graft-vs.-host disease

Pediatric study met the primary endpoint of day 28 overall survival, with 69% of patients achieving that endpoint vs. protocol-defined historical control rate of 45% (p=0.0003); in patients with positive overall response at day 28, survival was 87% at day 100; day 180 survival for those patients was 79%

Novo Nordisk A/S, of Bagsvaerd, Denmark

Oral semaglutide

GLP-1 analogue

Type 2 diabetes

Headline results from phase IIIa PIONEER 10 study in Japanese patients achieved primary objective, showing comparable number of adverse events with oral semaglutide vs. 0.75-mg dulaglutide


Notes

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