Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the pivotal study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049).
On the strength of upside in best corrected visual acuity (BCVA) in patients with geographic atrophy (GA), Annexon Inc. officials will meet with the U.S. FDA to talk about what’s next for ANX-007, after the C1q inhibitor missed the phase II trial’s primary endpoint: mean rate of change, or slope, in the GA lesion area compared to sham.
“We clearly see an active drug here,” PTC Therapeutics Inc. CEO Matthew Klein said of the 15-lipoxygenase inhibitor vatiquinone for Friedreich’s ataxia (FA), tested in a phase III study called Move-FA that missed the primary endpoint of statistically significant change in modified FA Rating Scale score at 72 weeks. The company will “take one step at a time” decisions about the drug, analyzing the results and then consulting with the U.S. FDA regarding how to proceed, he said. Meanwhile, Wall Street wasn’t happy, and South Plainfield, N.J.-based PTC’s shares (NASDAQ:PTCT) closed May 24 at $46.95, down $11.46, or 19.6%.
Results from a new study suggest that treatment with Magnus Medical Inc.'s Saint neuromodulation system causes abnormal brain signals to become normal by reversing the direction brain signals flow in severely depressed individuals. The researchers also identified a new biomarker that could help doctors diagnose and treat major depressive disorder (MDD).
Jewelry used to adorn the human ear is not the stuff of legend where medical technology is concerned, but a clip-type device, similar to an earring, that can be worn on the tragus of the ear might soon become daily wear for those who suffer from postural orthostatic tachycardia syndrome (POTS). Researchers at the University of Oklahoma say their self-administered clip can relieve POTS with just one hour’s use per day over a period of two months, a finding that might prove an inexpensive and convenient treatment for as many as one million Americans who currently have few treatment options.
Bringing both ventricles back into synchrony has long been the subject of cardiological hopes in patients with heart failure, but current approaches to maximizing the function of the left ventricle leave many patients in a desperate state of cardiac dyssynchrony. However, Ebr Systems Inc., has reported the results of a study that demonstrates that its WiSE device can pace the left ventricle in patients who are unresponsive to conventional pacing in an effort to restore optimal left ventricular function, a development Sunnyvale, Calif.-based Ebr says will allow the company to conclude a premarket filing with the U.S. FDA.
While pricing a $67.8 million registered direct offering, Icosavax Inc. also released positive top-line interim data from a phase I study of IVX-A12 against respiratory syncytial virus (RSV) and human metapneumovirus in older adults.
Hanall Biopharma Co. Ltd.’s phase III trial VELOS-3 of tanfanercept did not show effects on the central cornea of the eye or in eye dryness in the treatment of moderate to severe dry eye disease, the primary endpoints. Data from the trial did show improvements in tear production, a secondary endpoint.
Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain.