Phase I

Avrobio Inc., of Cambridge, Mass.


CTNS gene stimulator


First of 6 expected adult participants dosed in single-arm phase I/II trial sponsored by academic collaborators at University of California San Diego; primary endpoints are safety and tolerability assessed up to 2 years following treatment and efficacy assessed by cystine levels in white blood cells; secondary endpoints include changes in cystine levels in blood, intestinal mucosa and skin, and cystine crystal counts in eye and skin

Bavarian Nordic A/S, of Copenhagen


Multivalent MVA-based vaccine

Equine encephalitis virus

First-in-human trial initiated to evaluate safety, tolerability and immunogenicity in 45 healthy adults in 3 treatment groups receiving different doses; top-line results expected in 2020

Briacell Therapeutics Corp., of Berkeley, Calif.


Breast cancer

GM-CSF receptor modulator

First of up to 24 participants dosed in phase I/II combination study with INCMGA-00012 (Incyte Corp.) and epacadostat in individuals with advanced disease; primary endpoint is safety and overall response rate; secondary endpoints include duration of response, progression-free survival and overall survival

Ideaya Biosciences Inc., of South San Francisco


Protein kinase C inhibitor

Melanoma; solid tumors

First participant with cutaneous melanoma harboring GNAQ or GNA11 mutation dosed to evaluate drug in solid tumors outside metastatic uveal melanoma as part of ongoing phase I/II basket trial

Onl Therapeutics Inc., of Ann Arbor, Mich.


CD95 antagonist

Macula-off rhegmatogenous retinal detachment

First-in-human trial initiated in Australia to assess safety and tolerability of 4 dose levels

Salarius Pharmaceuticals Inc., of Houston


Lysine specific histone demethylase 1 inhibitor

Ewing sarcoma

Memorial Sloan Kettering Cancer Center and Nationwide Children's Hospital added as sites in ongoing phase I/II trial; dose-escalation and expansion study currently enrolling fourth dose level (600 mg twice daily)

Phase II

Capricor Therapeutics Inc., of Los Angeles


Cardiosphere-derived allogenic stem cell therapy

Duchenne muscular dystrophy

Additional 6-month data from prespecified interim analysis of 20 teens and young men with advanced DMD (12 placebo, 8 treated) in ongoing HOPE-2 study showed improvements in Performance of the Upper Limb tool designed to assess high (shoulder), mid (elbow) and distal (wrist and hand) function in those who received multiple doses of drug

Minoryx Therapeutics SL, of Barcelona

Leriglitazone (MIN-102)

PPAR gamma agonist

Friedreich ataxia

Completed recruitment (n=39) in Frames trial

Novan Inc., of Morrisville, N.C.


Topical nitric oxide

Molluscum contagiosum infection

Data published in Journal of the American Academy of Dermatology showed drug demonstrated clinical efficacy with favorable safety profile; of those who completed 12 weeks of treatment (n=217), all MC lesions cleared in 20% of vehicle patients compared with 13.2%, 41% and 35.1% of those treated with twice-daily SB-206 4%, 8% or 12%, respectively, and 41.9% treated with once-daily SB-206 12%

Vistagen Therapeutics Inc., of South San Francisco


N-methyl-D-aspartate receptor glycine site antagonist

Major depressive disorder

Completed dosing of patients in the Elevate study; top-line results expected before the end of 2019

Phase III

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

CPI-613️ (devimistat)

Targets the mitochondrial tricarboxylic acid cycle

First-line metastatic adenocarcinoma of the pancreas

Enrolled 100 of an expected 500 patients in the Avenger 500 study testing CPI-613 with modified FOLFIRINOX


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


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