Afferent Pharmaceuticals Advances Novel Molecule, AF-130, to Phase 1 Clinical Trial
Second Candidate from Company’s P2X3 Development Pipeline to Enter the Clinic
San Mateo, CA – December 8, 2015 – Afferent Pharmaceuticals, a leader in the development of small molecule compounds for the treatment of poorly managed and common neurogenic disorders, announced today initiation of the company’s Phase 1 trial of AF-130, a selective and differentiated P2X3 antagonist. Afferent’s Phase 1, double-blind, randomized, placebo-controlled trial is designed to assess the safety, tolerability and pharmacokinetics of single ascending doses and, in a second cohort, multiple ascending doses of AF-130 administered to healthy subjects. Further information regarding the study can be found at
Pending the progress of the Phase 1 trial, Afferent intends to evaluate AF-130 in multiple conditions, including treatment-resistant hypertension, and potentially in heart failure, migraine and visceral pain. The goal of treatment with a P2X3 inhibitor is to restore normal physiological function by blocking ATP activation of P2X3 channels that otherwise leads to sensitization of certain nerves and pathological signs and symptoms. Afferent’s lead product, AF-219, is currently being developed for the treatment of pathologic cough, including chronic cough, and cough in idiopathic pulmonary fibrosis (IPF) patients.
“Our broad platform and know-how in P2X3 receptor biology and chemistry has enabled us to generate well-differentiated candidates targeting this key mechanism and driver of pathologic signs and symptoms that affect millions of patients,” said Kathleen Sereda Glaub, chief executive officer of Afferent Pharmaceuticals. “AF-130 is the second lead compound from our development pipeline to advance to the clinic. This promising candidate has demonstrated distinctive pharmaceutical properties, and pending the results of the current Phase 1 study, we anticipate commencing a number of proof-of-concept trials, including in patients with treatment-resistant hypertension in 2016. Additional studies will evaluate other indications with substantial unmet needs, potentially including visceral pain, itch and migraine.”
About P2X3 Receptor-Mediated Sensitization
Afferent’s clinical candidates – AF-219 and AF-130 – are orally available, first-in-class compounds that selectively block P2X3 receptors.  P2X3 receptors play a key role in the sensitization of certain sensory nerves, notably C-fiber afferents. These nerves become activated and sensitized under pathological conditions mediated by a common cellular signal, ATP, when it is released in high concentrations due to cellular distress following injury or infection. Afferent’s compounds selectively block ATP activation of P2X3 channels, reducing a range of sensory signs and symptoms.
P2X3 receptor-mediated sensitization has been implicated in inflammatory, visceral and neuropathic pain states, as well as airways hyperreactivity, treatment-resistant hypertension, heart failure, migraine, itch and cancer pain.
About Afferent Pharmaceuticals
Afferent Pharmaceuticals is a clinical-stage biotechnology company and leader in the development of novel drugs for the treatment of a range of neurogenic disorders. These disorders affect millions of patients who suffer from chronic respiratory and urologic sensory pathologies, as well as chronic pain and cardiovascular disorders, and who have limited, if any, treatment options. These chronic pathologies arise when certain nerves become hyper-sensitized as a result of inflammation, distress, infection or tissue injury, which may remain chronically sensitized for months and even years.
Afferent is developing a portfolio of compounds that selectively block P2X3, which plays a key role in the sensitization of these nerves. Afferent’s lead molecule, AF-219, is in Phase 2 clinical development for the treatment of pathologic cough, including chronic cough and cough in idiopathic pulmonary fibrosis (IPF) patients. A second compound, AF-130, has entered Phase 1 clinical testing and will be evaluated in a number of non-respiratory conditions. For more information on the company, please visit Afferent’s website at
Afferent Pharmaceuticals
Amy Pfeiffer, 650-286-1276
Burns McClellan, Inc.
Justin Jackson, 212-213-0006, Ext. 327