Heinrich, M.D., professor of medicine and cell/developmental biology at the Knight Cancer Institute, Oregon Health & Research University, Portland, Oregon. ‘Treatment with approved agents in the second or third-line settings is associated with a median time to progression of significantly less than six months for each collection of treatment. There is a great unmet need for additional treatments because of this population of patients. Stable disease at 16 weeks can be an essential milestone for refractory sufferers so I believe these early scientific responses to ponatinib are encouraging.’.. ARIAD reviews data from Iclusig Stage 2 trial in adult individuals with gastrointestinal stromal tumors ARIAD Pharmaceuticals, Inc.Dr. Harkness lately presented her results at the International Culture for Affective Disorders Conference in Toronto.. Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR Alnylam Pharmaceuticals, Inc. , a leading RNAi therapeutics company, announced today that it provides initiated dosing in its Phase I medical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin for the treatment of TTR-mediated amyloidosis . ATTR is due to mutations in the TTR gene which trigger abnormal amyloid proteins deposits to accumulate in a variety of tissues including peripheral nerves and heart, leading to neuropathy and/or cardiomyopathy.