staff

Karyopharm Therapeutics Inc., a clinical pharmaceutical company, reported first-line positive results from the STORM phase 2b study that evaluated the effect of selinexor in heavily pre-treated patients with refractory multiple myeloma. Regarding the primary objective of the STORM study, oral selinexor achieved a global response rate (ORR) of 25.4%, which included two complete (CR) and 29 partial (PR) responses or very good partial responses (VGPR) in these patients with refractory myeloma. The median duration of the response (DOR), a key secondary objective, was 4.4 months. All responses were confirmed by an independent review committee (IRC). Selinexor recently obtained the Fast-Track designation by the US Food and Drug Administration (FDA) for this indication.

Aldeyra Therapeutics presented the results of a randomized, double-blind clinical trial of phase 2a on patients with ocular dryness treated with reproxalap at the ARVO annual meeting. The main objective of the experimentation, that is to select a formulation for the Phase 2b clinical trial, was reached with the progress of reproxalap at 0.1%. Compared to baseline, patients with dry eye disease treated with 0.1% reproxalap showed a statistically significant improvement in tear volume (Schirmer test), as well as a statistically and clinically significant improvement in the overall 4-symptom score and in the ocular disability score. Fifty-one subjects with active dry eye disease were randomized to receive ocular reproxalap for topical use at 0.1%, reproxalap at 0.5% or reproxalap formulated with 0.5% lipids four times a day. The results indicated statistically significant changes in the SANDE score (Assessment symptom in the case of dry eye), the ocular discomfort score, the overall score of 4 symptoms, the tear volume (Schirmer test), the osmolarity and the coloration of the cornea (Lissamine Green). A modest dose-dependent response was observed and improvement in symptoms was observed within one week of therapy. In January 2018 Aldeyra announced the start of a Phase 2b clinical trial that will recruit 300 patients with active disease, randomized to receive 0.1% or 0.25% reproxalap. The results of the study should be announced in the second half of 2018.

Alnylam Pharmaceuticals, an important company focused on RNAi therapies, announced that the Company has reached alignment with the US Food and Drug Administration (FDA) on a pivotal study project for lumasiran, an experimental RNAi for the treatment of primary hyperoxaluria type 1 (PH1). The Company and the FDA aligned on a primary endpoint for the registration study based on the reduction of six-month urinary oxalate, a biomarker directly related to the pathophysiology of PH1 and known to be well correlated with the progression of the disease. Furthermore, Alnylam and FDA have aligned themselves on a study size of about 25 patients with PH1. Based on discussions with the FDA, the Company is on track to begin the Phase 3 study in mid-2018, with plans to report topline results in 2019 and, if positive, to present an NDA at the beginning in 2020. Lumasiran was recently granted the designation of Breakthrough Therapy and Priority Medicines (PRIME) by the European Medicines Agency (EMA). Lumasiran offers hope to patients with PH1 who face health challenges. Alnylam’s alignment with the FDA key study project is an important step forward for patients suffering from this serious disease, as it recognizes the urgency of unmet need for effective treatment for PH1 and the potential for lumasiran to meet this necessity. The ongoing Phase I / IIb study is designed as a randomized, single-blind, placebo-controlled study. Patients with PH1, aged six or more, were enrolled.

miRagen Therapeutics has announced new provisional data from its phase 1 clinical trial of cobomarsen in patients with mycosis fungoides (MF). The data were presented in May at “2015 … 2018 T-cell lymphomas: we are close to finalization”, meeting doctor held in Bologna. Additional data obtained on cobomarsen provide further evidence that microRNA therapies have the potential to improve quality of life for patients living with mycosis fungoides. Cobomarsen continues to be safe and generally well tolerated at all doses tested in the Phase 1 study, with more patients receiving more than one year of therapy without serious adverse events attributed to the drug. Furthermore, the improvement in skin disease observed in the study appears to be lasting and is accompanied by improvements in metrics that measure patients’ quality of life. These data support the company’s plans to initiate a phase 2 clinical trial in patients with CTCL in the second half of 2018. Intermediate results presented at the conference include safety observations from long-term administration to 18 patients. The highlights presented are:

• cobomarsen treatment has produced a lasting improvement in quality of life, as measured by the total Skindex-29 score.
• 13 out of 18 subjects showed improvement in the first 100 days with cobomarsen.
• Improvement and stabilization remain durable in four subjects up to one year after treatment and one subject was stable after more than 400 days of cobomarsen.
• Cobomarsen continued to be generally well tolerated at all assessed dose levels.
• More patients received more than one year of therapy (up to 39 grams of cumulative dose) without serious adverse events attributed to cobomarsen.
• 300 and 600 mg intravenous infusions have similar efficacy and tolerability, offering the highest response rate based on mSWAT skin scores.