Abivax SA, of Paris, said the first patient was enrolled in a trial testing the pharmacokinetics of ABX464 in HIV cellular reservoirs in 24 HIV patients and 12 healthy volunteers. During the ABX464-005 study (the phase was not disclosed), patients will receive ABX464 for 28 days in addition to their antiretroviral treatment. Rectal biopsies will be collected at certain intervals, allowing quantification of the viral load and the level of inflammation in that reservoir over time. The study may therefore provide a better understanding of the biological mechanism driving the long-term efficacy on the viral load rebound observed in preclinical models with ABX464, the company said. Initial results are expected in the third quarter of this year.
Asit Biotech SA, of Brussels, Belgium, reported results from its phase I/IIa double-blind, placebo-controlled trial in house dust mite rhinitis, which achieved the primary endpoint, with its hdm-ASIT+ candidate showing a good safety and tolerability profile. Secondary objectives, which assessed hdm-ASIT+'s impact on the immune system and on the reduction in reactivity to a conjunctival provocation test (CPT), showed an effect observed on the immune system in a limited number of patients. However, there was no difference overall between the treated group and the placebo group with regard to immunogenicity parameters. The trial showed a somewhat stronger reduction in CPT reactivity in the treated group compared to the placebo group. The study was not powered to show statistical significance, and the company said the absence of a larger reduction can be explained by a substantial response to placebo (55 percent), the limited number of patients, the short observation period in the perennial disease and/or the nature of the product.
Axon Neuroscience SE, of Vienna, said that during the second quarter it will start a pilot phase I study of its active tau vaccine, AADvac1, in patients suffering from the nonfluent variant of primary progressive aphasia (nfvPPA), a subgroup of frontotemporal dementia (FTLD) patients. The trial will be run in cooperation with the German FTLD Consortium. Data from Axon's phase I study in Alzheimer's disease (AD) demonstrated that the antibodies elicited by vaccination with AADvac1 could recognize pathological tau protein in AD brains. That data suggests that the antibody response in patients can stop or slow down the progression of the disease, the company said. The company is now working to confirm those results in an ongoing phase II study in AD.
CSL Behring LLC, of King of Prussia, Pa., announced the publication in the New England Journal of Medicine of results from the Compact study, a pivotal phase III trial evaluating the safety and efficacy of CSL830 (C1-esterase inhibitor [C1-INH] human replacement therapy) for the prevention of hereditary angioedema (HAE) attacks. The study met its primary efficacy endpoint, significantly reducing the time-normalized number of HAE attacks. It also met secondary endpoints, including the responder rate (patients who had at least a 50 percent reduction in their attack rate) and the number of rescue medication uses. If approved by the FDA, it would be the first and only subcutaneous preventive therapy for HAE, the company said. The FDA accepted the company's biologics license application for CSL830 on Aug. 30, 2016.
Erytech Pharma SA, of Lyon, France, said a phase II investigator-launched study will test eryaspase, marketed as Graspa, in patients with acute lymphoblastic leukemia (ALL). The single-arm study will take place in seven Nordic countries and be conducted in collaboration with the Nordic Society of Pediatric Hematology and Oncology. It will enroll about 30 patients. The main objectives are to evaluate the biological (pharmacokinetic and pharmacodynamic) activity, safety and immunogenicity profile of eryaspase in combination with the NOPHO ALL 2008 multi-agent chemotherapy protocol administered as second-intention treatment for children or adult ALL patients, ages 1 to 45, who experience hypersensitivity reactions to PEG-asparaginase or silent inactivation. The study is expected to start this month and continue for about two years.
Galapagos NV, of Mechelen, Belgium, announced three new phase II proof-of-concept studies investigating filgotinib in Sjögren's syndrome, ankylosing spondylitis (AS) and psoriatic arthritis (PA). All three studies will be multicenter, randomized, double-blind and placebo-controlled. The Sjögren's study is being led by filgotinib collaboration partner Gilead Sciences Inc., of Foster City, Calif. It will test the drug in adult patients with active syndrome, Galapagos said. About 140 patients will be randomized in 60 to 80 centers globally to receive either filgotinib, placebo or two other investigational regimens administered once daily for up to 48 weeks. The primary endpoint will be the percentage of patients on treatment fulfilling protocol-specified clinical response criteria at week 12. The phase II AS study, led by Galapagos, will study filgotinib in adult patients with moderate to severe active disease. About 100 patients are expected to be randomized in the study in a 1-to1 ratio to receive 200 mg of filgotinib once daily or a placebo once daily, administered for 12 weeks. The primary goal of the study, called Tortuga, will be to evaluate the effect of filgotinib vs. placebo on the signs and symptoms of AS, as assessed by the AS Disease Activity Score at week 12. The phase II PA study, also led by Galapagos, will be called Equator. It will asses filgotinib in adult patients with moderately to severely active PA. About 124 patients are planned to be randomized in the study in a 1-to-1 ratio to receive 200 mg or placebo once daily, administered for 16 weeks. The primary goal will be to evaluate the effect of filgotinib compared to placebo on the signs and symptoms of psoriatic arthritis, as assessed by the American College of Rheumatology 20 percent improvement score at week 16. The study will also explore the effects of filgotinib on psoriasis, dactylitis, tendinitis, spondylitis and patients' nails. Gilead stepped in to partner with Galapagos in December 2015 after North Chicago-based Abbvie Inc. turned its focus to an internal JAK inhibitor, ABT-494 (upadacitinib), in 2015. (See BioWorld Today, Dec. 18, 2015.)
Hepatera LLC, of Moscow, a portfolio company of Maxwell Biotech Venture Fund, and development partner Myr GmbH, of Burgwedel, Germany, said they completed recruitment for the phase IIb MYR 202 and 203 trials investigating Myrcludex B in chronic hepatitis delta infection. The program enrolled 180 subjects in 20 centers in Russia and Germany. Interim results from the MYR 202 study are expected by year-end. Myrcludex B is an entry inhibitor designed to treat chronic hepatitis B and D infections.
Mesoblast Ltd., of Melbourne, Australia, and Boston Children's Hospital, the pediatric teaching hospital of Harvard University, said they've gained FDA approval for starting a 24-patient trial combining Mesoblast's allogeneic mesenchymal precursor cells (MPCs) with corrective heart surgery in children under the age of 5 with hypoplastic left heart syndrome (HLHS). The trial is sponsored and funded by the hospital with support from the Bulens and Capozzi Foundation and the Ethan Lindberg Foundation. In the randomized, controlled trial, Mesoblast's candidate MPC-150-IM will be injected into the left ventricle of children with HLHS during surgical recruitment procedures of the small ventricle with the intent of improving ventricular mass and function leading to higher likelihood of biventricular conversion. MPC-150-IM is also under evaluation in a phase III trial in up to 600 patients with New York Heart Association (NYHA) class II/III chronic heart failure (CHF) and in a phase IIb trial of 159 patients with NYHA class IV CHF in conjunction with implantation of a left ventricular assist device.
Replicel Life Sciences Inc., of Vancouver, British Columbia, reported interim data from its phase I study evaluating RCS-01 to treat aging and sun-damaged skin. The primary objective was to establish a safety profile for intradermal injections of RCS-01, the company's type 1 collagen-expressing, hair follicle-derived fibroblasts, six months post-injection. Participants in the Germany-based study did not report any serious adverse events at the interim evaluation. Although the study was not powered for or expected to show statistically significant efficacy results, interim findings showed a nearly twofold increase in gene expression of collagen-related biomarkers in the skin following a single injection of RCS-01, deemed by the company as statistically significant. Gene expression markers, such as tissue inhibitor of metalloproteinases, also showed changes expected to correlate with increased collagen fibers.
Therapeuticsmd Inc., of Boca Raton, Fla., said the pivotal phase III Replenish trial of TX-001HR (estradiol 1 mg/progesterone 100 mg and estradiol 0.5 mg/progesterone 100 mg) met its co-primary efficacy endpoints. The bio-identical hormone therapy combination of 17-beta-estradiol and progesterone in a single oral softgel is designed to treat moderate to severe vasomotor symptoms due to menopause. Replenish showed that two doses of TX-001HR achieved statistically significant and clinically meaningful reduction in both the frequency and severity of hot flashes compared to placebo at weeks four and 12. Secondary endpoint data showed statistically significant improvement in total and vasomotor menopause-specific quality of life questionnaire, or MENQOL, scores at week 12 that were maintained through six and 12 months for the two study doses. A poster analyzing endometrial safety in the trial showed that TX-001HR resulted in no cases of endometrial hyperplasia or malignancy across treatment groups, meeting the recommendations established by the FDA in its draft guidance. The most common adverse events (>5 percent) across the active treatment groups in the trial included headache, nasopharyngitis, breast tenderness and upper respiratory infection. No unexpected safety signals occurred. The findings were presented at ENDO 2017 in Orlando, Fla.
Verona Pharma plc, of London, said the first patient was enrolled and dosed in a phase IIa study evaluating RPL554 to treat cystic fibrosis (CF). Conducted at a single site in the U.K., the double-blind, placebo-controlled study is evaluating the pharmacokinetic and pharmacodynamics profile and tolerability of RPL554 in up to 10 CF patients and examining the effect on lung function. RPL554 is an inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4, or PDE3/PDE4. (See BioWorld Today, June 21, 2016.)