Phase I

Abeona Therapeutics Inc., of New York


Gene therapy expressing COL7A1

Recessive dystrophic epidermolysis bullosa

Data published in JCI Insight showed 80% of 20 wounds had ≥50% healing and 70% had ≥75% healing after 3 years; 17% of 6 untreated wounds had ≥50% healing after 2 years; wounds with ≥50% healing had no pain and no itch after 3 years, compared to 53% with pain and 61% with itch at baseline

Immutep Ltd., of Sydney

Eftilagimod alpha

Soluble dimer of LAG-3

Metastatic melanoma

Eftilagimod alpha plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced an overall response rate of 50% in 6 patients in the dose-expansion phase at the recommended phase II dose

Oncosec Medical Inc., of San Diego


Plasmid expressing IL-12

Merkel cell carcinoma

Data published in Clinical Cancer Research showed an overall response rate of 25% in 12 patients with metastatic disease; 2 of 3 patients with locoregional disease were recurrence-free at 44+ and 75+ months

Principia Biopharma Inc., of South San Francisco


Covalent inhibitor of Bruton tyrosine kinase

Treatment-resistant and refractory immune thrombocytopenia

Interim results showed 39% of 26 patients had ≥ 2 consecutive platelet counts of ≥ 50,000/µL, separated by at least 5 days, and increased by ≥ 20,000/µL from baseline without requiring rescue medication; 46% of patients achieved any 2 platelet counts ≥ 50,000/µL; 50% of 5 patients who had completed 12 weeks met both thresholds

Phase II

Azurrx Biopharma Inc., of New York


Recombinant enzyme derived from yarrowia lipolytica lipase

Severe exocrine pancreatic insufficiency in cystic fibrosis patients

Treated first of about 24 patients testing escalating doses of the drug in combination with standard porcine enzyme replacement therapy; study expected to be complete in 2020

Can-Fite Biopharma Ltd., of Petach Tikva, Israel


Targets A3 adenosine receptor

Nonalcoholic fatty liver disease

Completed enrollment of 60 patients in study; top-line data expected in the first quarter of 2020

Collaborative Medicinal Development LLC, of Mill Valley, Calif.


Scavenger of peroxynitrite

Amyotrophic lateral sclerosis

Enrolled first of 80 patients in phase II/III study comparing drug to placebo for 6 months on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised and the Edinburgh Cognitive and Behavioral ALS Screen; results expected in about 18 months

Target Laboratories Inc., of West Lafayette, Ind.


Folate receptor-targeting imaging agent

Folate receptor-alpha positive ovarian cancer

Data published in Gynecologic Oncology showed the agent had a sensitivity of 97% when controlling for detection correlation of multiple lesions within a single patient; agent allowed surgeons to detect additional lesions in 48.3% of patients

Phasebio Pharmaceuticals Inc., of Malvern, Pa.


Monoclonal antibody antigen-binding fragment targeting ticagrelor

Antiplatelet reversal

Treated first of about 200 older and elderly patients, measuring the reversal of the antiplatelet effects of ticagrelor

Reata Pharmaceuticals Inc., of Irving, Texas


Activator of Nrf2

Friedreich's ataxia

In the Moxie study, omaveloxolone improved modified Friedreich's Ataxia Rating Scale (mFARS) by -1.55 points from baseline, compared to a worsening of +0.85 points for patients receiving placebo, resulting in a 2.4-point placebo-corrected improvement (p=0.014); when patients with pes cavus were included in the analysis, omaveloxolone produced a placebo-corrected 1.93-point improvement in mFARS (p=0.034)

Relmada Therapeutics Inc., of New York


NMDA receptor antagonist

Treatment-resistant major depressive disorder

At 2 different doses, the addition of REL-1017 to a stable background of antidepressant therapy produced effect size of 0.7 to 1 compared to placebo on the Montgomery-Asberg Depression Rating Scale from day 4 through day 14

Replimune Group Inc., of Woburn, Mass.


Oncolytic viral immunotherapy

Squamous cell carcinoma

First patient enrolled in trial of RP-1 in combination with Libtayo (cemiplimab, Regeneron Pharmaceuticals Inc. and Sanofi SA) vs. Libtayo alone

Phase III

Cel-Sci Corp., of Vienna, Va.


Leukocyte interleukin injection

Head and neck tumor

Independent data monitoring committee recommended continuation of trial until the appropriate number of events has occurred

Cumberland Pharmaceuticals Inc., Nashville, Tenn.

Vibativ (telavancin)

Semisynthetic lipoglycopeptide antibiotic

Bacterial pneumonia

A new publication in Infectious Diseases and Therapy showed numerically superior cure rates of Vibativ vs. vancomycin within a subset of patients who were enrolled in Attain trials and had hospital-acquired pneumonia caused by bacteria with low susceptibility to vancomycin

Novavax Inc., of Gaithersburg, Md.


Hemagglutinin nanoparticles with Matrix-M adjuvant

Influenza virus infection

First participants enrolled in pivotal trial; top-line data expected in the first quarter of 2020


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


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