• Alexion Pharmaceuticals Inc., of Cheshire, Conn., published data in the June 6, 2013, issue of the New England Journal of Medicine showing that chronic therapy with Soliris (eculizumab) was effective for hemolytic uremic syndrome. Results of two studies demonstrated that Soliris inhibited systemic complement-mediated TMA, decreased the need for TMA-related intervention, improved platelet count significantly, improved renal function and was associated with substantial kidney recovery. Chronic therapy with Soliris also led to reversal of vital organ damage and significant improvements in health-related quality of life. Earlier intervention improved clinical outcomes.

• ARCA Biopharma Inc., of Broomfield, Colo., published a paper in JACC: Heart Failure with results of a post-hoc analysis of a Phase III trial of Gencaro (bucindolol hydrochloride) in heart failure with reduced left ventricular ejection fraction, with regard to the impact of certain cardiac adrenergic receptor genotypes. The trial enrolled 2,708 patients and included a DNA substudy of 1,040 patients. The study found that patients who had received Gencaro had a 41 percent lower risk of new onset atrial fibrillation compared to placebo. The risk of new onset atrial fibrillation was reduced by 74 percent compared to placebo in patients with the beta-1 AR position 389 arginine homozygous genotype.

• BioLineRx Ltd., of Jerusalem, enrolled its first patient in a Phase II trial of BL-8040, for acute myeloid leukemia (AML). The company also received regulatory approval from the Israeli Ministry of Health to conduct the trial in Israel, and it is anticipating activating Israeli sites in the next few weeks. The open-label study will evaluate safety and efficacy of escalating doses of BL-8040 in adults with relapsed or refractory AML. Up to 50 patients will be enrolled.

• Inspiro Medical Ltd., of Tel Aviv, Israel, completed a trial of Inspiromatic, a dry powder inhaler for formoterol treatment of asthma in children. The trial enrolled 30 children, ages 8 to 18, for treatment with the Inspiromatic or a competing device. The study met its primary and secondary endpoints, showing superior results for delivery of the active drug. A lung function test showed a statistically significant improvement after formoterol delivery by the Inspiromatic, and it was highly rated by patients.

• Medivir AB, of Stockholm, Sweden, with its partner, Janssen Pharmaceutical R&D Ireland, a unit of New Brunswick, N.J.-based Johnson & Johnson, reported results from four Japanese Phase III studies of NS3/4A protease inhibitor simeprevir, showing sustained virologic response at 12 weeks after treatment in patients with genotype 1 hepatitis C. The study included treatment-naïve patients and nonresponders. Data were presented at the Japan Society of Hepatology's meeting in Tokyo.

• Proteostasis Therapeutics Inc., of Cambridge, Mass., said the Michael J. Fox Foundation awarded a grant for preclinical studies of a therapy for Parkinson's disease that is designed to clear alpha-synuclein. Proteostasis has a technology platform for identifying small molecules that modulate proteostasis network pathways. Studies by company co-founders showed significant changes in that pathway in brain samples from patients with Parkinson's disease.

Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Bayer HealthCare Pharmaceuticals Inc., of Wayne, N.J., disclosed positive top-line results for Eylea (aflibercept) Injection from the Phase III MYRROR study in myopic choroidal neovascularization. In that trial, patients receiving Eylea at an initial dose of 2 mg, followed by treatment on an as-needed basis, had a mean improvement in best-corrected visual acuity from baseline at week 24 of 12. 1 letters, compared to a loss of 2 letters in patients receiving sham injections (p < 0.0001). The most common adverse events observed in the MYRROR trial that occurred with a frequency of 2 percent or more were conjunctival hemorrhage, dry eye, eye pain, headache and nasopharyngitis. Eylea was approved in the U.S. for neovascular (wet) age-related macular degeneration in November 2011 and for macular edema following central retinal vein occlusion in September 2012. Shares of Regeneron (NASDAQ:REGN) gained $22.53, or 9.8 percent, to close Thursday at $252.17. (See BioWorld Today, Nov. 22, 2011.)

RXi Pharmaceuticals Corp., of Westborough, Mass., disclosed the unblinded results of the first of two placebo-controlled double-blind studies in volunteers with anti-scarring agent RXI-109, an sd-rxRNA compound that has been shown in vitro and in animals to reduce mRNA for connective tissue growth factor. Overexpression of that protein in wounds has been associated with abnormal scarring as seen in hypertrophic scars and keloids. The company was able to get a readout on a key biomarker for scarring in the study and said it will disclose more at an investors' meeting in July.

Vanda Pharmaceuticals Inc., of Washington, presented additional entrainment and patient-level clinical data at SLEEP 2013, the meeting of Associated Professional Sleep Societies in Baltimore, from its SET (Safety and Efficacy of Tasimelteon) and RESET (Randomized-withdrawal study of the Efficacy and Safety of Tasimelteon to treat non-24-hour disorder) Phase III studies of tasimelteon, a circadian regulator. In the SET study, tasimelteon achieved the primary endpoints of entrainment (synchronizing) of the melatonin (aMT6s) rhythm as compared to placebo and clinical response as measured by entrainment plus a score of greater than or equal to 3 on the Non-24 Clinical Response Scale. Tasimelteon also demonstrated significant improvement vs. placebo across a number of sleep and wake parameters, including measures of total sleep time, nap duration and timing of sleep, as well as in the Clinical Global Impression of Change, an overall global functioning scale.