Cystic Fibrosis
Nuvara has Unlocked Alternative Chloride Channels Shown to be More Efficacious at Stimulating Chloride Secretion in the Absence of CFTR
Healthy FTR & ENaC Function in the Lungs
Ion channels CFTR and ENaC pass chloride (Cl-) and sodium (Na+) ions (respectively) through the cell membrane maintaining equilibrium in the cell and managing mucus appropriately.
Current CF Therapies:
CFTR Correctors and Potentiators
Correctors and Potentiators work only in the presence of an available CFTR Channel. They restore function to CFTR, promoting the passage of chloride ions from inside the cell membrane to outside, resulting in the breakdown of mucus. However, ENaC remains in a hyperactive state, pulling excess sodium ions and water into the cell.
Cystic Fibrosis – Class I Mutation
CFTR is non-existent. ENaC is hyperactive. Chloride ions are stuck inside the cell and excess sodium is being pulled in, causing hyperpolarization which increases cell negativity and thickens extracellular fluid, making mucous sticky and air exchange difficult
Current CF drugs target the CFTR ion channel. Due of the absence of CFTR in this case, there are currently no effective treatments for Cystic Fibrosis Class I Mutation.
Using RxAA as cell signalers, Nuvara is the first and only company to:
• Open both the SLC26A9 and TMEM16A chloride channels in addition to, or in the absence of, CFTR
• Reduce sodium absorption by restricting ENaC
Anti-Diarrheal: Worlds first anti-diarrheal/oral rehydration dual-action formula
• Ion channels and transporters such as CFTR (chloride) and NHE3 (sodium) are natural targets for antidiarrheal therapies.
• New compounds targeting intestinal transporters are in development for antidiarrheal therapies, including small molecules, natural compounds, and existing drugs.
• Antidiarrheal therapies might be useful as stand-alone therapy or together with oral rehydration solutions (dual-action)
Clinical Efficacy: Diarrhea
• Community-acquired diarrhea
• 300 children
• Q4 ’23
| Program Summary | Clinical trial to compare the efficacy and safety of multiple amino-acid based ORS “VS002A” with standard WHO-ORS in the management of non-cholera acute watery diarrhea in infants and young children |
| Primary Endpoint | Duration of diarrhea in hospital (hours) |
| Exploratory Secondary Endpoint | Total stool output, ORS intake, clinical success, tolerability, blood chemistry, gut biomarkers. |
| Protocol Summary | Randomized double-blinded two-cell clinical trial to evaluate the efficacy and safety of “VS002A” with the standard WHO-ORS in the management of non-cholera acute watery diarrhea. A total of 312 (156 in each arm) male children aged 6-36 months old with acute (onset <48 hours) non- bloody watery diarrhea will be included in this study. |
| First Patient In | June 16, 2021 |
| Interim Data Read Out | August 2023 |
| Final Data | August 2023 |
Chronic Constipation: IBS-C
• Ion channels and transporters such as CFTR (chloride) and NHE3 (sodium) are natural targets for constipation therapies.
• New compounds targeting intestinal transporters are in development for constipation therapies, including small molecules, natural compounds, and existing drugs.
• Novel therapeutics aimed at anti-absorptive and pro-secretory functions represent a first ever synergistic, dual-action treatment for constipation.
Preliminary Data Demonstrates Significant Improvement over Standard of Care (Linaclotide)
