AlphaNavi Pharma Inc., Announces Initiation of a Phase 1/2 Clinical Trial of ANP-230 in Patients with Familial Infantile Episodic Limb Pain

AlphaNavi Pharma Inc. (Headquarter: Sakyo-ku, Kyoto, Japan; CEO: Yoshihiro Oyamada) today announced the initiation of a Phase 1/2 multicenter study of our lead drug candidate, ANP-230, in patients with familial infantile episodic limb pain. The study will enroll patients who have been diagnosed as familial infantile episodic limb pain, based on specific symptoms, family history, and SCN11A (voltage-gated sodium channel Nav1.9 gene) mutation. ANP-230 has demonstrated a tolerable safety profile and some clinical signals in several Phase 1 trials and this is the first trial to be initiated in patients with pain, to evaluate the Proof of Concept of it.
Preclinical evidences show that Nav1.9, one of the targets of ANP-230, involved to increase neuronal excitability and to increase the sensitivity to pain. Due to the inhibition of Nav 1.9, it is expected that ANP-230 might attenuate abnormal excitability and the intensity and the number of episodic pains.
“Evaluating ANP-230 in patients with familial infantile episodic limb pain is an important next step in our efforts to understand the potential of this investigational medicine to treat a treatment-resistant rare pain disease with SCN11A mutation,” said Hirotsugu Hayashi Ph.D., chief operating officer at AlphaNavi. “Based on the mechanism of action of ANP-230, we believe it has the greatest potential to benefit from treatment.”
For more information on this trial (jRCT2061200046), please visit:
This clinical development of ANP-230 was selected as one of the Cyclic Innovation for Clinical Empowerment (CiCLE) program under the application title: Study of a novel drug for familial episodic infantile limb pain syndrome, which is operated by the Japan Agency for Medical Research and Development (AMED).


About ANP-230
ANP-230 is a potent and selective voltage-gated sodium channel Nav1.7, Nav1.8 and Nav1.9 inhibitor, which has completed several Phase 1 studies in the UK, the US, and Japan. It is reported that Nav1.7, Nav1.8 and Nav1.9 are mainly expressed in peripheral nervous system and gain-of function mutations of those Nav induce treatment-resistant rare pain diseases, including erythromelalgia and familial episodic limb pain. ANP-230 might be only one published clinical-stage compound which inhibits Nav1.9 at this moment. In addition, due to high peripheral Nav selectivity and quite low penetration rate to blood-brain barrier, ANP-230 has less CV- and CNS-related side effects, while it still has potent analgesic efficacy.


About Familial Infantile Episodic Limb Pain
Familial infantile episodic limb pain is clinically characterized by attacks of pain in the limbs and joints, which starts during infancy, but no abnormal findings other than those of pain. This episodic pain is mainly induced by cold or bad weather, stress, fatigue, etc. It is reported that some patients suffering this episodic pain have SCN11A gain-of function mutations.


About AlphaNavi Pharma Inc.,
AlphaNavi Pharma has just established as a curve-out start-up company, which is focused on developing novel drugs to treat some kinds of pain. AlphaNavi’s goal is to deliver non-opioid pain therapies for people with treatment-resistant rare pain diseases. For more information, please visit AlphaNavi’ website at