Amicus Therapeutics Inc., of Cranbury, N.J., announced new positive data from both of its phase III studies of oral small-molecule pharmacological chaperone migalastat HCl for Fabry disease at WORLDSymposium in San Diego. The company is also presenting additional proof-of-concept data for its product candidate ATB200/AT2221 for Pompe disease. In an oral presentation and poster from study 011 (FACETS) in Fabry patients who were naïve to enzyme replacement therapy (ERT), migalastat demonstrated a consistent and statistically significant reduction in disease substrate (GL-3) in podocytes from baseline to month six (p=0.02). Podocytes play a key role in Fabry nephropathy, including proteinuria, and have shown more resistance than other kidney cell types to clear GL-3.The annualized change in glomerular filtration rate in the migalastat group at month 30 was comparable to the previously reported results for the migalastat and ERT groups through month 18. In addition, an oral presentation and poster describe updated preclinical results that informed the ongoing clinical study, ATB200-02, in Pompe patients to investigate a treatment paradigm (ATB200/AT2221) that consists of ATB200, an engineered recombinant human acid alpha-glucosidase enzyme with an optimized carbohydrate structure to enhance uptake, administered with a pharmacological chaperone (AT2221) to improve activity and stability. Previously presented preclinical data showed that ATB200 was associated with increased tissue enzyme levels and reduced substrate, which was further improved when co-administered with AT2221.

Cellceutix Corp., of Beverly, Mass., said dosing was completed in its phase II trial of Prurisol for the treatment of mild to moderate chronic plaque psoriasis. In the trial, physicians selected and evaluated a specific lesion while the patient was treated with a Prurisol or placebo tablet, as opposed to treating the lesion with a cream or gel, a common method used in clinical trials for the treatment of mild to moderate chronic plaque psoriasis. Subjects will now be followed for an additional 28 days without taking additional study medication. Cellceutix is developing Prurisol, a small molecule that acts through immune modulation and PRINS reduction, under the FDA's 505(b)(2) pathway.

Colucid Pharmaceuticals Inc., of Cambridge, Mass., said it received a special protocol agreement for its second pivotal phase III trial, called SPARTAN, from the FDA. The objective of the study is to evaluate the safety and efficacy of lasmiditan (50 mg, 100 mg and 200 mg) in comparison to placebo two hours after dosing on freedom from migraine headache pain, which is the primary endpoint, and on freedom from the most bothersome associated symptom of migraine (nausea, phonophobia or photophobia), which is the key secondary endpoint. The study is expected to treat a single migraine in up to 2,226 migraine patients with lasmiditan at approximately 130 sites in the U.S., U.K. and Germany.

Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel, said it randomized the first patient in the ARRIVE (ARamchol for the Reversal of HIV-AssociatEd Lipodystrophy and NAFLD) proof-of-concept study to evaluate the safety and efficacy of Aramchol, a synthetic fatty-acid/bile-acid conjugate, in up to 50 patients with HIV-associated lipodystrophy and nonalcoholic fatty liver disease. The phase IIa trial will compare pre- and post-treatment MRI-measured liver fat content and total body fat via dual energy x-ray absorptiometry. The primary endpoint will be an improvement in hepatic steatosis as measured by MRI. Secondary endpoints will be an improvement in total body fat, metabolic profile and liver biochemistry.

New Haven Pharmaceuticals Inc., of New Haven, Conn., reported clinical data showing that Durlaza is well tolerated, with a favorable safety profile comparable to immediate-release, low-dose aspirin, and delivers sustained antiplatelet control for a full 24-hour period in high-risk cardiovascular and diabetes patients. Data were presented at the American College of Preventive Medicine meeting in Arlington, Va. Durlaza received FDA approval in September.

Relmada Therapeutics Inc., of New York, said full results from a phase I trial of d-methadone (dextromethadone, REL-1017) in patients suffering from chronic pain were published in the Journal of Opioid Management and demonstrated that d-methadone appears to be safe and well tolerated. Findings will support a phase II trial of the NMDA antagonist in neuropathic pain.