Via both acquisition and internal research, Celtaxsys is evolving a flexible and diversified pipeline focused around modulating innate immunity to provide first-in-class or best-in-class therapeutics for important diseases such as Cystic Fibrosis (CF) and Multiple Sclerosis (MS).

CTX-4430: Our flagship program, CTX-4430, is an inhibitor of the well-known pro-inflammatory enzyme Leukotriene A4 Hydrolyase (LTA4H). LTA4H simulates inflammation by producing a potent neutrophil chemoattractant and immune cell activator called Leukotriene B4 (LTB4). LTB4 is known to be elevated in the blood of patients with Cystic Fibrosis, certain types of Asthma, and Chronic Obstructive Pulmonary Disease (COPD). Based on this knowledge and emerging science around the status and role of neutrophils in promoting lung dysfunction, Celtaxsys is positioning CTX-4430 as first-in-class oral treatment for reducing or preventing pulmonary disease associated with Cystic Fibrosis.

CTX-4430 Follow-ons: Celtaxsys is also leveraging the diverse properties of its LTA4H inhibitor class to create specialized drug candidates that are optimized for other applications, such as central nervous system (CNS) penetration for treatment of active Multiple Sclerosis. Preclinical models suggest that brain penetration is not required for the therapeutic effect of CTX-4430 in MS, however more research is necessary to better equate the preclinical effects with likely outcomes in human disease by comparing localization of the drug to peripheral tissue versus brain. However, it is clear that the target of CTX-4430, LTA4H, is highly up-regulated in innate immune cells associated with actively degrading MS lesions, leading to the potential for a breakthrough treatment for Chronic Progressive MS (CPMS).

CTX-4398: Celtaxsys is developing advanced second-generation injected products that improve patient comfort and, thus, compliance and efficacy. This research is based on our internal research into the underlying cause of the inflammation that occurs after injection of biologic drugs. The first such product is CTX-4398, and improved biologic for treatment of Relapsing Remitting Multiple Sclerosis (RRMS).

Neutrophil Repellents: Emanating from Celtaxsys’s foundation in modulating innate immunity and its focus on neutrophils, two Neutrophil Repellent programs are being advanced in parallel. These programs differ in chemical nature, with one being a polypeptide (CTX-4273) and the other a small molecule (CTX-4342). CTX-4273 originated from a blood-borne protein isolated as the active neutrophil repellent in a human tumor sample and has since been converted to an active peptide mimic of the protein. CTX-4342 is less than 300 MW yet shows a level of neutrophil repelling activity that is comparable to the neutrophil attracting activity of interleukin-8 (IL-8).