Eloxx Pharmaceuticals, Inc.

B usiness Company Overview We are a clinical-stage biopharmaceutical company developing novel, small molecule product candidates designed to modulate the ribosome and promote readthrough of premature stop codons induced by nonsense mutations ( NMs ) to enable the production of full-length proteins. Targeting ribosome subunits provides a therapeutic approach to addressing a number of genetic diseases. According to the Human Gene Mutation Database, NMs account for approximately 10% to 12% of patients with a given genetic disease. There are over 7,000 inherited genetic diseases that collectively affect 350 million people worldwide. Our immediate focus is to advance the clinical development of our lead product candidate, exaluren, for the treatment of rare kidney diseases and, through our collaboration with Almirall, S.A. ( Almirall ), ZKN-013 for the treatment of rare skin diseases. Exaluren is a novel eukaryotic ribosome-selective glycoside ( ERSG ) product candidate that is designed to induce premature termination codon ( PTC ) readthrough in the presence of NMs and promote the restoration of full-length proteins. We believe this mechanism has the potential to apply to all genetic diseases caused by NMs. We are initially developing exaluren to induce full-length Collagen IV protein for the treatment of Alport syndrome ( AS ) with NMs ( NMAS ).