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:
inhaled 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

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)

Organoid swelling is a surrogate marker for clinical results, predicting decreased sweat chloride level and increased FEV₁

Promising preclinical results as monotherapy and in combination with Kalydeco
Potential as an inhaled therapy with improved therapeutic index

LX-02 has >100 fold lung to plasma exposure when inhaled versus given subcutaneously
IND for nebulizer based (inhaled) delivery of ELX-02 initiated

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)

Organoid swelling is a surrogate marker for clinical results, predicting decreased sweat chloride level and increased FEV₁

Promising preclinical results as monotherapy and in combination with Kalydeco
Potential as an inhaled therapy with improved therapeutic index

LX-02 has >100 fold lung to plasma exposure when inhaled versus given subcutaneously
IND for nebulizer based (inhaled) delivery of ELX-02 initiated

RMAs have the potential to become
oral therapies to restore functional CFTR protein
in Class 1 CF patients

Over 40 Hits with greater than equal preclinical activity in Patient lung cells have been identified.
RMAs offer compelling benefit vs risk profile for chronic therapy.

Promising Preclinical RESULTS Paved the Way for our phase 2 trial

Organoid Swelling Charts

Promising preclinical results
paved the way for our Phase 2 Trial

Learn More About RDEB / JEB >

Learn More About FAP / CRC >

Explore Our Pipeline >

CLASS 1 CYSTIC FIBROSIS (CF) WITH NONSENSE MUTATIONS

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

RMAs have the potential to become oral therapies to restore functional CFTR protein in Class 1 CF patients

Promising Preclinical RESULTS Paved the Way for our phase 2 trial

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

RMAs have the potential to become
oral therapies to restore functional CFTR protein
in Class 1 CF patients