Alexion Pharmaceuticals Inc., of New Haven, Conn., said researchers presented 24-week data from an ongoing, open-label, phase I/II trial testing intravenous SBC-103 (rhNAGLU enzyme), an enzyme replacement therapy, in children with mucopolysaccharidosis IIIB (also known as Sanfilippo B syndrome), a genetic and progressive rare lysosomal storage disease. Data showed a 26.2 percent mean reduction from baseline in total heparan sulfate (HS) levels in cerebrospinal fluid (CSF) at 24 weeks in the highest dose studied (3 mg/kg every other week). Those results were presented at the WORLDSymposium meeting in San Diego. The primary endpoint of the ongoing trial is safety and tolerability, and key secondary endpoints include changes from baseline in HS levels in CSF and serum as well as pharmacokinetics and pharmacodynamics. Additional secondary endpoints related to neurocognitive outcomes, brain structure and changes in HS levels in urine are also being evaluated.
Evoke Pharma Inc., of Solana Beach, Calif., said it has enrolled 93 percent – 186 of 200 patients – in its phase III trial testing EVK-001, a nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in women. The company also reported that it amended a covenant associated with the timing of phase III results under its $4.5 million loan and security agreement with Pacific Western Bank (as successor to Square 1 Bank), with the bank agreeing to extend the deadline under the credit facility to Sept. 30.
Glycomimetics Inc., of Rockville, Md., said investigators observed clinical responses in eight of the first 13 evaluable patients in its trial testing GMI-1271 in combination with chemotherapy in relapsed/refractory acute myeloid leukemia, for an overall response rate of 62 percent. Of the eight objective responses, seven achieved a complete response, with the eighth patient achieving a complete response with an incomplete blood count recovery. GMI-127, a small-molecule E-selectin antagonist, also was well tolerated.
Hutchison China Meditech Ltd., of London, said its R&D subsidiary started an open-label, phase II trial testing sulfatinib (HMPL-012), a VEGFR/FGFR1 inhibitor, in patients with locally advanced or metastatic radioactive iodine-refractory differentiated thyroid cancer or medullary thyroid cancer in China. The first patient was dosed on March 1. The study is expected to enroll a total of 50 patients, with about 25 in each tumor type, with a primary endpoint of objective response rate. Secondary and exploratory endpoints include safety and tolerability, other efficacy parameters, pharmacokinetics and tumor biomarkers.
Merck KGaA, of Darmstadt, Germany, said it started a phase III study of MSB11022, a proposed biosimilar of adalimumab (sold as Humira by Abbvie Inc.), in chronic plaque psoriasis. The randomized, double-blind, active-controlled AURIEL-Psoriasis study is evaluating the efficacy, safety and immunogenicity of MSB11022 compared with Humira. About 400 patients are expected to be recruited.
Miragen Therapeutics Inc., of Boulder, Colo., started a phase I study of MRG-106, a synthetic microRNA antagonist (LNA antimir) of microRNA-155, in patients with cutaneous T-cell lymphoma of the mycosis fungoides subtype. The study is designed to assess safety, tolerability and pharmacokinetics and will also explore the molecular signature of MRG-106 in the lesions of those patients.
Motif Bio plc, of London, said it dosed the first patient in two phase III trials of lead antibiotic candidate iclaprim for the treatment of acute bacterial skin and skin structure infections (ABSSSIs). The REVIVE (Randomized Evaluation Intra Venous Iclaprim Vancomycin TrEatment) trials will assess the efficacy of iclaprim compared to standard-of-care antibiotic vancomycin and will enroll a total of 1,200 adults hospitalized with ABSSSIs. The primary endpoint will be at least a 20 percent reduction in lesion size at 48 hours to 72 hours after treatment. The key secondary endpoint is clinical cure at one to two weeks after treatment.
Oncosec Medical Inc., of San Diego, said it completed patient enrollment in its phase II extension study of Immunopulse IL-12 for the treatment of advanced melanoma. The study’s objective is to evaluate an alternate dosing frequency and additional biomarkers to provide further understanding of Immunopulse IL-12 in an extension of the OMS-I100 phase II melanoma study. Consolidated data from both trials are expected in the third quarter. The Immunopulse technology is designed to enhance the local delivery and uptake of DNA-based immune-targeting agents such as IL-12.
Scpharmaceuticals Inc., of Lexington, Mass., said the pivotal trial for its ceftriaxone program met the targeted endpoints, defined as noninferior antimicrobial coverage when compared to the same dose given by intravenous infusion. Antimicrobial coverage refers to time over the minimum inhibitory concentration, a generally accepted measure of the adequacy of plasma levels of an antibiotic for treatment of infections due to susceptible bacteria. The study is part of the development program for a drug-device combination product comprising ceftriaxone and the Sc2wear Ceftriaxone Pump, for which the company anticipates submitting a 505(b)(2) new drug application in the second half of this year.
Viamet Pharmaceuticals Inc., of Research Triangle Park, N.C., reported results from the planned interim analysis of the phase IIb RENOVATE (REstoring Nail; an Oral VT-1161 Tablet Evaluation) study testing VT-1161, an orally available inhibitor of fungal CYP51, in onychomycosis of the large toenail. In the intent-to-treat analysis, up to 59 percent of patients treated with VT-1161 had a negative dermatophyte culture by week 24. There was a 48 percent average reduction in the percentage of nail involvement at week 24 across the VT-1161 arms, as compared to a 6 percent reduction in the placebo arm. The median reduction in percentage nail involvement in the VT-1161 arms was as high as 67 percent at week 24. Safety data from the 100-patient interim analysis population through the week 24 visit demonstrated that VT-1161 was well tolerated, with a favorable safety profile and no evidence of an adverse effect on liver function.
Wuxi Biologics Co. Ltd., of Shanghai, and Prima Biomed Ltd., of Sydney, said, through their supply partnership, Prima’s immuno-oncology candidate, IMP321, a LAG-3 Ig fusion protein manufactured at Wuxi’s cGMP facility, was dosed in a phase IIb trial. The AIPAC (Active Immunotherapy PAClitaxel) study is testing IMP321 in metastatic breast cancer.
Zealand Pharma A/S, of Copenhagen, reported top-line results from the phase II trial of danegaptide for the protection against cardiac reperfusion injuries in patients with an acute myocardial infarction, which showed the study did not meet the primary endpoint of reducing ischemic reperfusion injuries, as measured on the Myocardial Salvage Index. Danegaptide, a gap junction modifier, was shown to be safe and well tolerated.