CLASS 1 CYSTIC FIBROSIS (CF) WITH NONSENSE MUTATIONS

CF is caused by a mutation in the cystic fibrosis transmembrane conductance (CFTR) gene, which codes for the protein that regulates salt and water balance. It is characterized by the buildup of mucus in the respiratory and digestive track leading to progressive damage to these organs. There are five classes of CF representing more than 1,700 different mutations in the CFTR gene.

Pipeline

Class 1 nonsense mutations result in no production of CFTR protein
• 12% of mutations are class 1, affecting between 7,000 and 9,000 people globally¹

Pipeline

There are no approved treatments for Class 1 CF; management is palliative

We have 2 programs to
address Class 1 CF: ELX-02 and oral TURBO-ZM™ based RMAs. Pipeline

Cystic Fibrosis Foundation

ELX-02: Potential for Transformative
Efficacy in Patients with Class 1 CF

Granted orphan drug and Fast Track designation by the FDA

ELX-02 Phase 2 Trial

(learn more at clinicaltrials.gov)

ELX-02 Phase 2 Trial

• Potential as an inhaled therapy with improved therapeutic index

—ELX-02 has >100 fold lung to plasma exposure when inhaled versus given subcutaneously

—IND for nebulizer based (inhaled) delivery of ELX-02 initiated

• Potential as an inhaled therapy with improved therapeutic index

—ELX-02 has >100 fold lung to plasma exposure when inhaled versus given subcutaneously

—IND for nebulizer based (inhaled) delivery of ELX-02 initiated

Existing Data Paved the Way for Our
Clinical Development of ELX-02

Patients with Class 1 CF treated with the aminoglycoside, gentamicin, had a 22% decrease in sweat chloride level vs patients treated with placebo
ELX-02 Class 1 organoid swelling response similar to Class 2 response for Symdeko (tezacaftor/ivacaftor and ivacaftor)