Argenx NV, of Breda, the Netherlands, started a phase II trial of ARGX-110, an antibody targeted CD70, as a monotherapy in patients with relapsed/refractory cutaneous T-cell lymphoma (CTCL). The company has previously observed evidence of biological activity and a promising safety profile in several CD70-positive CTCL patients treated in an ongoing phase I safety-expansion cohort, resulting in several patients with partial response or stable disease. Based on these preliminary results, Argenx opted to further evaluate ARGX-110 as a monotherapy in the exploratory phase II study to demonstrate the intrinsic activity of the drug in relapsed/refractory CTCL patients and to broaden the efficacy database. The trial will enroll up to 10 additional relapsed/refractory CTCL patients, and will be conducted at multiple centers in Europe. The primary endpoints of the trial are safety and efficacy and secondary endpoints include pharmacokinetics and immunogenicity. Interim data are expected by the end of 2017, and the company expects to report topline data in the second half of 2018.
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C., said it plans to explore a new oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks in patients with hereditary angioedema and has received initial regulatory approvals in Europe to start an exploratory trial with the new formulation. CEO Jon Stonehouse said he thinks the new formulation can fill an unmet need for patients with less frequent attacks who are looking for better ways to manage their illness. The upcoming trial, Zenith-1, is designed as a randomized, double-blind, placebo controlled, dose-ranging trial with BCX7353 self-administered at home to treat attacks.
Immunovaccine Inc., of Halifax, Nova Scotia, reported that all seven older adults provided with a 25 µg dose of the company's Depovax-based, small B-cell epitope peptide vaccine candidate for respiratory syncytial virus maintained the antigen-specific immune responses one year after receiving the booster dose. At one year, the antibody levels measured were still at peak with no sign of decrease. The data, from the high dose cohort of a phase I investigator-sponsored study, builds on top-line results from the low-dose cohort that the company released last year.
Ironshore Pharmaceuticals & Development Inc., of George Town, Grand Cayman, a subsidiary of Highland Therapeutics Inc., said that this year it intends to initiate two pivotal studies of HLD100, its experimental delayed- and extended release formulation of amphetamine for patients with attention-deficit/hyperactivity disorder after finding encouragement for the move in feedback it received from the FDA at an end-of-phase II meeting. The meeting covered the outcome of HLD100-103, a trial that found the drug may have a long duration of action without any discernible rebound effects that are sometimes reported with stimulant medications.
Kadmon Holdings Inc., of New York, reported that patients with moderate to severe psoriasis treated with its oral Rho-associated coiled-coil kinase 2 inhibitor, KD025, during an open-label phase II study saw improved clinical scores and skin pathology in psoriasis patients via concurrent modulation of the pro- and anti-inflammatory immune cell response. The 12-week study enrolled 38 patients with moderate to severe psoriasis. It demonstrated that KD025 affected the cellular mechanisms associated with psoriasis progression, as measured in both peripheral blood and the skin, leading to improvements in clinical scores of patients at 12 weeks, the company said. Specifically, KD025 significantly reduced peripheral blood levels of IL-17 and IL-23, two pro-inflammatory cytokines, and showed a correlation between changes in IL-17 levels and patients' clinical scores. The results were published in the April 7 edition of the Journal of Immunology.
Neuralstem Inc., of Germantown, Md., said an additional cohort of four patients will be added to its ongoing phase I trial evaluating the safety and feasibility of using lead cell therapy candidate, NSI-566 – spinal cord-derived neural stem cells – to repair chronic spinal cord injury. The company said the amended protocol was approved by the FDA and the Institutional Review Board at the study site. The four qualifying patients have AIS-A complete, quadriplegic, cervical injuries involving C5-C7 cord. The protocol calls for the injury to have occurred one to two years prior to treatment, which involves a single surgery involving six injections of NSI-566 into the affected area of the cord.
Oncolytics Biotech Inc., of Calgary, Alberta, disclosed an initial registration pathway and development plan for Reolysin, its immuno-oncology viral agent. At an end-of-phase II meeting with regulators, the company plans to present data from its randomized, open-label phase II study assessing Reolysin, dosed intravenously in combination with paclitaxel vs. paclitaxel alone, in patients with advanced or metastatic breast cancer. The 74-patient study showed a statistically significant improvement in median overall survival (OS) in the intention-to-treat patient population, from 10.4 months on the control arm to 17.4 months on the Reolysin arm (hazard ratio 0.65, 80 percent CI 0.46-0.91, p=0.1). Oncolytics Biotech will request scientific advice to support a registration pathway, with features of future studies expected to include OS as a primary endpoint, exploratory endpoints to identify potential markers of response and a trial design ensuring sufficient enrollment to reach a statistically significant outcome. In parallel, the company expects to advance Reolysin in collaboration with biopharma partners in immunotherapy and immunomodulatory indications, initially with cancer charity Myeloma U.K. and Celgene Corp., of Summit, N.J., through an agreement reached last month for a phase Ib dose escalation trial that will study Reolysin in combination with Celgene's Imnovid (pomalidomide) or Revlimid (lenalidomide) as a rescue treatment in relapsing myeloma patients. The study will evaluate safety and tolerability of the combinations and investigate whether the addition of Reolysin extends disease control in patients. Oncolytics Biotech also is running its first study of Reolysin in combination with checkpoint inhibitors.
Protalix Biotherapeutics Inc., of Carmiel, Israel, reported phase II data showing that treatment with alidornase alfa, a plant cell-expressed, chemically modified recombinant DNase enzyme resistant to inhibition by actin, resulted in clinically meaningful lung function improvement in cystic fibrosis patients (CF), as demonstrated by a mean absolute increase in the percent predicted forced expiratory volume in one second (ppFEV1) of 3.4 points from baseline. The 28-day switchover study enrolled 16 CF patients previously treated with Pulmozyme (dornase alfa), with a two-week washout period before receiving alidornase alfa via inhalation. Data also showed a mean absolute increase in ppFEV1 of 2.8 points observed in patients participating in the trial when compared to measurements taken from patients at initiation before the switch from Pulmozyme to alidornase alfa. Correlation between improvement in sputa parameters and pulmonary function was observed.
Saniona AB, of Ballerup, Denmark, said Mexican regulators approved the phase III study by partner Productos Medix, S.A de S.V, of Mexico City, to test tesofensine, a triple monoamine reuptake inhibitor, in obese Mexican patients. The study will include 372 patients and is expected to start following importation and subsequent release of the drug product. It will be designed as a randomized, double-blind, placebo-controlled, parallel-arm trial, with a primary endpoint of absolute and percent change in body weight over the treatment period. The study is expected to be completed within two years from initiation.
Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan, reported top-line results from a phase III study testing dasotraline (SEP-225289), a dopamine norepinephrine reuptake inhibitor, in children, ages 6 to 12, with attention deficit hyperactivity disorder (ADHD). The trial met the primary endpoint, defined as change from baseline at day 15 in ADHD symptoms as measured by mean SKAMP-Combined Score obtained from an average of seven assessments collected across the 12-hour laboratory classroom day (12-24 hours post-dose). Dasotraline 4 mg/day demonstrated sustained efficacy over the 12- to 24-hour time period post-dose and was associated with an acceptable safety profile over the two-week study duration. Based on the results, Sunovion Pharmaceuticals Inc., a U.S. subsidiary of Sumitomo Dainippon, plans to submit an NDA to the FDA this year for adult and pediatric populations.
Therapeutics Solutions International Inc., of Oceanside, Calif., said its subsidiary, Emvolio Inc., filed an IND for use of its Stemvacs cancer immunotherapeutic in patients with solid tumors. Stemvacs, which is generated using dendritic cell progenitors isolated from patients, is then activated by a proprietary process to endow dendritic cells with the ability to produce natural cytokines designed to kill cancer cells. The trial seeks to establish safety and immune response of the cancer, targeting a new personalized dendritic cell vaccine.
Transgene SA, of Strasbourg, France, said the first patient with soft tissue sarcoma (STS) was treated in the phase II part of the METROmaJX phase I/II trial testing the tolerability and efficacy of the co-administration of Pexa-Vec with metronomic cyclophosphamide (low doses given with high frequency) in patients with advanced solid tumors such as breast cancer and STS. In part one, the combination demonstrated a satisfactory tolerability profile. The second part of the open-label trial will enroll patients with STS and HER2-negative-breast cancer and will primarily assess antitumor efficacy. Pexa-Vec is a GM-CSF-expressing vaccinia-derived oncolytic virus co-developed by Transgene and Sillajen Co. Ltd., of South Korea.