Phase I

Athenex Inc., of Buffalo, N.Y., and Guangzhou Xiangxue Pharmaceutical Co. Ltd., of Guangzhou, China

KX2-361 (formerly known as KX-02)

Src kinase inhibitor

Advanced malignant solid tumors

Started the dose-escalation study in China

Cook Regentec LLC, of Indianapolis

Hematrate blood filtration system

Produces autologous, peripheral blood-derived total nucleated cell concentrate for intramuscular injection

Critical limb ischemia

Enrolled first patient with CLI due to peripheral arterial disease; primary endpoint is clinical benefit through 12-month follow-up, defined as freedom from reintervention, major amputation or death

Phase II

Momotaro-Gene Inc., of Okayama, Japan


Gene therapy delivering the reduced expression in immortalized cells/dickkopf-3 gene

Relapsed malignant pleural mesothelioma

Treated first of up to 12 patients in the study testing MTG-201 with a PD-1 inhibitor; primary endpoint is objective response rate; disease stabilization, duration of response, progression-free survival and overall survival will be measured as secondary endpoints; safety and the exploratory biomarkers of activity and the immunogenicity of MTG-201 will also be measured

Phase III

Biogen Inc., of Cambridge, Mass.

Tecfidera (dimethyl fumarate)

Activates the nuclear erythroid 2-related factor 2 transcriptional pathway

Relapsing multiple sclerosis

In the Endorse extension study, 51% of patients remained relapse-free over 10 years; 64% had no confirmed disability progression; 79% maintained the ability to walk without significant disability; meta-analysis of 18 databases of large real-world studies found Tecfidera was significantly more effective than interferon-beta, glatiramer acetate and teriflunomide, had comparable effectiveness to fingolimod and was less effective than natalizumab and alemtuzumab in reducing annualized relapse rate and delaying time to first relapse

Biogen Inc., of Cambridge, Mass., and Alkermes plc, of Dublin

Diroximel fumarate

Activates the nuclear erythroid 2-related factor 2 transcriptional pathway

Relapsing multiple sclerosis

Interim results from 888 patients in the Evolve-MS-1 study showed the drug was generally well-tolerated; less than 1% of patients taking the drug discontinued treatment due to gastrointestinal side effects; drug reduced annualized relapse rate by 79.4% and the mean number of gadolinium-enhancing lesions by 64.3% from baseline to 24 months

Gedeon Richter plc., of Budapest, Hungary


Dopamine D3/D2 receptor partial agonist


CGI-I improvement was similar for cariprazine and risperidone, but more patients taking cariprazine had "much" and "very much" improvement; patients with predominant negative symptoms had significantly better clinical improvement with cariprazine compared to risperidone; improvement in PANSS Factor Score for Negative Symptoms correlated with improvement in Personal and Social Performance Scale

Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson


S1P1 receptor modulator

Relapsing multiple sclerosis

In the Optimum study, annualized relapse rate in patients taking ponesimod was reduced by 30.5% compared to Aubagio (teriflunomide, Sanofi SA) up to week 108 (p=0.0003); mean difference in Fatigue Symptoms and Impacts Questionnaire - Relapsing Multiple Sclerosis, favoring ponesimod over Aubagio, was -3.57 (p=0.0019); cumulative number of combined unique active lesions was reduced by 56% for ponesimod compared to Aubagio (p<0.0001)

Nevakar Inc., of Bridgewater, N.J.


Topical eye drop formulation of atropine


Completed enrollment in the 576-patient Champ study measuring myopia progression in children over 3 years

Roche Holding AG, of Basel Switzerland

Ocrevus (ocrelizumab)

Monoclonal antibody targeting CD20-positive B cells

Relapsing multiple sclerosis and primary progressive multiple sclerosis

In the Opera I study in RMS, Ocrevus reduced blood serum neurofilament light chain (NfL) by 43% from baseline to 96 weeks compared to a 31% reduction for interferon-beta-1a (p<0.001); in the Oratorio study in PPMS, Ocrevus reduced blood NfL levels by 16% from baseline to 96 weeks compared with a 0.2% reduction for placebo (p<0.001)


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


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