Akcea Therapeutics Inc., of Cambridge, Mass., disclosed the publication in Diabetes Care of positive data from a phase II study of volanesorsen in patients with high plasma triglyceride (TG) levels and type 2 diabetes. Results from the study showed that, in addition to reductions in plasma TG and apolipoprotein C-III (ApoC-III), a key factor in TG regulation, volanesorsen improved insulin sensitivity and glucose control. Volanesorsen is currently being evaluated in two pivotal phase II studies: the APPROACH FCS study in patients with familial chylomicronemia syndrome and the BROADEN FPL study in patients with familial partial lipodystrophy. Also, the COMPASS study, a phase III trial designed to support global regulatory filings, is ongoing. An antisense drug, volanesorsen is designed to reduce the production of ApoC-III.
Allergy Therapeutics plc, of Worthing, U.K., disclosed findings from its exploratory phase II dose-ranging study (G204) for the U.S. Grassmatampl development program and said the results did not determine a recommended dose for the phase III trial. A further dose range-finding study will be implemented prior to proceeding into the planned pivotal phase III study, the company said. Grassmatampl is described as an ultra-short course subcutaneous allergen specific immunotherapy administered prior to the grass pollen season.
Boehringer Ingelheim GmbH, of Ingelheim, Germany, said a new analysis of the Inpulsis trials shows for the first time that disease progression is similar in idiopathic pulmonary fibrosis (IPF) patients identified using a broader diagnostic definition compared with the current diagnostic guidelines. The post-hoc analysis of these 1,061 patients has been published in the American Journal of Respiratory and Critical Care Medicine. Physicians typically use an imaging technique called high resolution computed tomography (HRCT) to help them identify the presence of scarring (fibrosis) and specifically the presence of usual interstitial pneumonia (UIP) pattern in the lungs to ensure an accurate diagnosis of IPF. Radiological changes called “honeycombing” are a key indicator for lung fibrosis and a feature of the UIP pattern visible on HRCT. However, it can be challenging to confirm that scarring in the absence of honeycombing on HRCT meets the strict guideline criteria for a definitive diagnosis of IPF. For a large group of patients who do not receive a confirmed diagnosis of IPF according to guidelines, including those not eligible for surgical lung biopsy, the clinical course of their condition and the effectiveness of IPF treatment remains unknown. The Inpulsis trials included patients with a classic diagnosis of IPF but also those patients with a clinical diagnosis of IPF who, in the absence of a surgical lung biopsy and honeycombing on HRCT, had a possible UIP pattern and the presence of traction bronchiectasis. Traction bronchiectasis is recognized as one of the most relevant signs of lung fibrosis. The analysis also shows that Boehringer’s oral kinase inhibitor, Ofev (nintedanib), is effective in slowing disease progression in both diagnostic subgroups which confirms the efficacy of Ofev across a broad range of IPF patient types studied in phase III and may be applicable to patients seen in clinical practice, the company said.
Santalis Pharmaceuticals Inc., of San Antonio, disclosed positive results from its single-center, open-label, efficacy and tolerability proof-of-concept study using a 10 percent East Indian sandalwood oil (EISO) serum formulation for the treatment of mild-to-moderate plaque psoriasis. The results of the study showed the EISO serum formulation to be safe, well tolerated, and efficacious for patients 18-65 years of age with a maximum of 10 percent treatable body surface area involvement of mild-to-moderate plaque psoriasis. Santalis said research has shown EISO is a broad-acting anti-inflammatory agent that strongly suppresses the activity of factors including PDE4 and IL-17 that are believed to contribute to psoriasis. EISO also has been shown to reduce the hyper-proliferation in psoriatic skin that leads to plaque formation, the company said.
Stealth Biotherapeutics Inc., of Boston, started two phase II studies evaluating elamipretide in heart failure: Progress-HF in patients with heart failure with reduced ejection fraction and Restore-HF in those with preserved ejection fraction. Top-line data from each are expected in the second half of 2017. The primary endpoint of Progress-HF is change from baseline in the volume of blood remaining in the left ventricle at end of contraction, as assessed by cardiac magnetic resonance imaging (MRI). Secondary endpoints will include additional measures of cardiac function, including ejection fraction, by echocardiography and MRI. The primary endpoints of Restore-HF are changes in diastolic heart function at rest and during submaximal stress, as assessed by echocardiography. Secondary endpoints include changes in exercise capacity, as measured by the six-minute walk test, and responses to the SF36 patient-reported health survey. Elamipretide is described as an investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress.
Twi Biotechnology Inc., of Taipei, Taiwan, completed patient enrollment in its phase II trial with AC-201CR for the treatment of gout. The company expects to report top-line results from this study in the fourth quarter of 2016. The positive interim results of the proof-of-concept study were made public late last year. AC-201 is described as a first-in-class, orally available small molecule which has shown the dual abilities to inhibit URAT1 as well as the production and activity of caspase-1 and the cytokine interleukin-1 beta through the inhibition of the assembly of the NLRP3 inflammasome.
Viromed Co. Ltd., of Seoul, South Korea, said its U.S. arm Vm Biopharma dosed the first patient in the recently initiated phase III study evaluating VM202, described as a DNA-based biopharmaceutical, in patients with painful diabetic peripheral neuropathy. This is the first pivotal gene therapy trial specifically targeting the most common cause of severe neuropathy, and follows the successful completion of a phase II trial conducted at Northwestern University and multinational sites, the firm said.