Company Product Description Indication Status
Phase I

Adaptimmune Therapeutics plc, of Oxfordshire, U.K.


Autologous T cells

Soft tissue sarcoma

Clinical responses in 7 of 14 patients with synovial sarcoma by RECIST 1.1; clinical benefit in 13 out of 14 patients

Anelixis Therapeutics Inc., of Cambridge, Mass.


Anti-CD40L antibody

Amyotrophic lateral sclerosis

Completed study in healthy volunteers and participants with ALS; drug was well-tolerated at all doses

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.


Double-stranded oligomer RNAi targeting APOC3


Dose-dependent reductions in serum APOC3 observed; no serious or severe adverse events

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.


Double-stranded oligomer RNAi targeting ARO-ANG3

Homozygous familial hypercholesterolemia

Dose-dependent reductions in serum ANGPTL3 observed; no drug-related severe or serious adverse events

Celldex Therapeutics Inc., of Hampton, N.J.


Kit tyrosine kinase inhibitor


Phase Ia study in health volunteers started

Enlivex Therapeutics Ltd., of Ness Ziona, Israel


Autologous mononuclear-enriched leukocytes plus the patients' own cells


Completed recruitment of 10 patients with severe sepsis for phase Ib trial

Kadimastem Ltd., of Ness Ziona, Israel


Astrocytes derived from human embryonic stem cells

Amyotrophic lateral sclerosis

Plans to submit amendment seeking permission to test repeated low dose instead of medium dose, as originally planned

Sensei Biotherapeutics Inc., of Gaithersburg, Md.


Nanoparticle-based immunotherapeutic cancer vaccine

Head and neck tumor

Dosed first patient in phase I/II trial in patients with ASPH-positive disease who previously received immune checkpoint inhibitors

Valneva SE, of Saint Herblain, France


Live-attenuated vaccine

Chikungunya virus infection

With sustained immunogenicity profile in all dose groups, plans to accelerate program to pivotal phase III trial in 2020 (subject to FDA agreement); 100% seroconversion achieved at day 14 after a single vaccination

Phase II

Amgen Inc., of Thousand Oaks, Calif.

Omecamtiv mecarbil

Cardiac myosin activator

Heart failure

Additional data from Cosmic-HF study showed measures of diastolic function were not different from placebo and, for some measures, trended toward improvement

Bridge Biopharma Inc., of San Francisco


Orally administered small molecule

Transthyretin amyloidosis cardiomyopathy

Adverse events reported in open-label combination study were generally consistent with the underlying disease state and no safety signals of potential clinical concern were associated with the administration of drug; 19 (40.4%) participants experienced a treatment-emergent serious adverse event, with congestive cardiac failure (10.6%) and acute kidney injury (8.5%) the most commonly reported 

Foamix Pharmaceuticals Ltd., of Rehovot, Israel


3% minocycline, 0.3% adapalene combination foam

Moderate to severe acne vulgaris

Enrollment complete

Karuna Therapeutics Inc., of Boston


Oral co-formulation of xanomeline (muscarinic receptor agonist) and trospium (muscarinic receptor antagonist)

Psychosis in schizophrenia

Turned up a statistically significant and clinically meaningful 11.6-point mean reduction in total Positive and Negative Syndrome Scale score compared to placebo (p<0.0001) and also showed good overall tolerability

Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda


Subcutaneously injected, recombinant fusion protein that blocks IL-1α and IL-1β signaling


Mean patient-reported pericardial pain on an 11-point numerical rating scale decreased from 4.5 at baseline to 0.5 at 24 weeks

Lidds AB, of Uppsala, Sweden


2-hydroxyflutamide, injected directly into the tumor region

Prostate cancer

Results confirm 90% of patients receiving 16 ml intraprostatic injection experienced a prostate-specific antigen reduction; showed 16 ml is the optimal dosage for future phase III studies

Sanifit Therapeutics SA, of Majorca, Spain


Binds to growth sites of hydroxy-apatite crystals


Compared to placebo, 52 weeks of treatment significantly attenuated progression of coronary artery calcification in patients with end-stage kidney disease receiving hemodialysis

Phase III

Aveo Oncology Inc., of Cambridge, Mass.


VEGF tyrosine kinase inhibitor

Refractory advanced renal cell carcinoma

Updated data from Tivo-3 study showed, as of Aug. 15, 2019, data cutoff date, median overall survival was 16.4 months for tivozanib and 19.7 months for sorafenib; as of second data cutoff date, 20 patients remained progression-free on tivozanib arm vs. 2 on sorafenib, with median duration on study of 32.5 months

Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Reduced ejection fraction, with or without type 2 diabetes

Analyses from Dapa-HF trial showed drug reduced risk of primary composite outcome of worsening heart failure, defined as hospitalization or an urgent visit, or death from cardiovascular causes vs. placebo, when added to standard of care

Caladrius Biosciences Inc., of Basking Ridge, N.J.


Autologous CD34+ cell therapy

Coronary microvascular dysfunction

Results from Escape-CMD trial showed single administration durably improves heart function and symptoms; patients experienced statistically significant (p=0.0087) increase in coronary flow reserve after a single intracoronary administration; treatment also resulted in statistically significant improvements in measures of patient symptoms and function using the Canadian Cardiovascular Society angina classification and Seattle Angina Questionnaire scores

Daiichi Sankyo Europe GmbH, of Munich, Germany

Bempedoic acid

Once-daily ATP citrate lyase inhibitor

Diabetes; hypercholesterolemia

2 pooled analyses from 4 studies showed bempedoic acid reduced HbA1c by 0.19% vs. placebo in in diabetes patients at 12 weeks; significantly lowered LDL-C in patients with hypercholesterolemia when added to maximally tolerated statin therapy, reducing by 18% vs. placebo, and by 25% vs. placebo in patients not on statin background therapy

Iterum Therapeutics plc, of Dublin



Complicated urinary tract infections

Completed patient enrollment in the Sure study; top-line results expected in early 2020

The Medicines Co., of Parsippany, N.J.


Cholesterol-lowering siRNA candidate

Heterozygous familial hypercholesterolemia

Results from Orion 9 met all primary and secondary efficacy endpoints; for primary endpoints, inclisiran delivered placebo-adjusted LDL-C reductions of 50% (71 mg/dL, p<0.0001) at day 510 and demonstrated time-averaged placebo-adjusted LDL-C reductions of 45% (63 mg/dL, p<0.0001) from days 90 through 540

Merck & Co. Inc., of Kenilworth, N.J., and Bayer AG, of Leverkusen, Germany


Soluble guanylate cyclase stimulator

Chronic heart failure

Victoria study to treat patients with worsening disease met the primary endpoint, reducing risk of the composite endpoint of heart failure hospitalization or cardiovascular death in patients with reduced ejection fraction vs. placebo when given in combination with available heart failure therapies

Novartis AG, of Basel, Switzerland

Entresto (sacubitril/valsartan)

Inhibits neprilysin and blocks angiotensin II type-I receptor

Heart failure with preserved ejection fraction

Subgroup analyses from Paragon-HF study show that, in specific subgroups, treatment may result in greater reductions in hospitalizations and cardiovascular death vs. valsartan; greater benefit seen in women with HFpEF and in HFpEF patients recently hospitalized for heart failure; in pooled analysis of Paragon-HF and Paradigm-HF trials, greater treatment benefit observed in patients with left ventricular ejection fraction below about 60%; data published in the Journal of the American College of Cardiology

Phase IV

Astrazeneca plc, of Cambridge, U.K.

Brilinta (ticagrelor)

Reversible, direct-acting P2Y12 receptor antagonist

Non-ST elevation acute coronary syndromes

Results from prespecified subgroup analyses of Twilight trial showed monotherapy reduced risk of clinically relevant bleeding vs. dual antiplatelet therapy over 12 months in high-risk patients; monotherapy associated with 53% relative reduction in risk of primary endpoint – Bleeding Academic Research Consortium (BARC) type 2, 3 or 5 bleeding – over 1 year, with an absolute risk reduction of 4%, compared to ticagrelor plus aspirin (3.6% vs. 7.6%); reduction in risk of BARC 3 or 5 bleeding in monotherapy group vs. ticagrelor/aspirin over 1 year (0.8% vs. 2.1%)

Correvio Pharma Corp., of Vancouver, British Columbia

Brinavess (vernakalant hydrochloride)

Antiarrhythmic drug

Recent onset atrial fibrillation

New post-hoc analysis from Spectrum study in emergency department setting, 1,289 Brinavess treatment episodes in 1,120 patients were assessed, with successful conversion in 70.2% of all treated episodes; treatment with Brinavess also showed median time to conversion of 12 minutes from start of first infusion among patients who converted

Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim GmbH, of Ingelheim, Germany


SGLT2 inhibitor

Type 2 diabetes, with and without cardiovascular disease

Interim analysis of 3-year data from Emprise real-world study showed treatment associated with decreased risk of hospitalization for heart failure and a similar risk of nonfatal atherosclerotic cardiovascular events vs. DPP-4 inhibitors and GLP-1 receptor agonists


For more information about individual companies and/or products, see Cortellis.

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