Confronting Human Protease-Driven Diseases
Verra transforms the treatment of major unsolved illnesses using an innovative small protein platform.
About Us
Verra Therapeutics is a privately held company near Ithaca NY, that was founded in 2017 with a mission of transforming the treatment of protease-driven diseases using an innovative small protein platform.
Verra is developing preclinical drug candidates for Chronic Bronchitis, Acute Lung Injury and Acute Kidney Injury by adapting nature’s highly evolved design for each target protease.
Dr. Christopher Prince brings decades of leadership experience to the team in managing technology companies through biotech and pharmaceutical development. Dr. Marcia Moss has a comprehensive background in drug discovery and development and is a thought leader on proteases as disease targets.
Verra’s goal is to create novel, selective and impactful drugs for patients with major unsolved medical illnesses.
WHY HUMAN PROTEASES?
- A largely untapped target class of about 600 members
- Dozens play key roles in disease and many lie at the heart of regulatory pathways
- They can be great targets: ACE ≈ $12 billion
- Proteases control the activity of most proteins in the body, including each other
- Dysregulation underlies major unsolved illnesses:
• cancer • lung, kidney and heart diseases
• brain disorders • autoimmune disorders
- Historical approaches usually fail. The latest trends come up short.
How to Intervene?
Small molecule inhibitors have largely failed for closely related proteases as they lack sufficient surface area for selectivity. Discriminating similar proteases with different activities is vital.
Antibodies can be selective, but are too large, exhibit limited tissue penetration and present substantial delivery challenges.
Both protein degraders and RNA-based silencing eliminate all domains of multi-functional and multi-domain proteases, not just the intended activity. Elimination has the real potential to create toxic effects and may limit therapeutic control.
...A Better Inhibitor Platform
- Based on nature's elegant design for each target. Native protein drugs promise better acceptance by the body.
- Meets and exceeds the thorny selectivity requirements of similar proteases. Small molecules don't.
- Better than antibodies:
- smaller by an order of magnitude
- bind only to activated targets
- promise vastly better tissue penetration...
poor penetration equals reduced efficacy
- few antibody drugs successful
for membrane-bound targets
- Simpler microbial production
- Subcutaneous or inhaled delivery
Pipeline
Chronic Bronchitis (in COPD)
COPD is a debilitating multi-phenotypic disease affecting about 400 million people worldwide, including as many as 24 million in the US.
Related health care expenses are estimated at $50 billion per year.
While phenotypes overlap, the most prevalent form is Chronic Bronchitis as it is diagnosed in up to 2/3 of COPD patients.
Sadly, current treatments address primarily symptoms; there are still no truly disease modifying drugs and Chronic Bronchitis-COPD remains an enormous unmet medical need.
A SCOURGE THAT IS MISUNDERSTOOD
- Second only to heart disease for cutting disability-adjusted life span
- Lifetime risk of succumbing is nearly 30% (US)
- Women and certain minorities are more vulnerable
- An underlying cause of many listed cardiac deaths
- The 3rd leading cause of death globally
- Up to 70% of COPD remains undiagnosed.
What’s new?
A specific protease culprit has been definitively linked with COPD’s three most important phenotypes: emphysema, small airway fibrosis and mucus hyper-secretion (chronic bronchitis).
It is up-regulated in the lungs of human patients in proportion to the severity of their disease.
Verra Therapeutics has developed a Game-changing inhibitor for this target.
Six month gold standard efficacy studies in mice demonstrate VTH245’s potential to greatly reduce mucus overproduction and damage in the lung. VTH245 exhibits favorable pharmacokinetics and is compatible with subcutaneous and inhaled routes of administration. Verra enjoys NIH support for this work. Among numerous disease-associated changes, abnormal mucus production and harmful elastin degradation are blocked with VTH245.
Improving Outcomes in ALI with VTH245
- Acute Lung Injury (ALI) is a serious life threatening illness that lacks adequate treatments
- ARDS (Acute Respiratory Distress Syndrome) is a more severe form of ALI and is the common cause of death in Covid and numerous other acute medical emergencies
- Causes range from lung infection to blood transfusions, surgeries, sepsis etc. (see below)
- The risk of death for patients with ALI is unacceptably high (as high as 40%)
- It causes over 2 million ICU days per year without a pandemic
- Survivors suffer a number of debilitating mental, physical and cognitive problems and a long period of dependency on others.
ALI is an unmet medical need as current therapeutics have not sufficiently impacted the risk of death or post-recovery quality of life.
- VTH245 and its target are new.
- The approach is supported by animal data.
- Development costs are partially born by overlap with the Chronic Bronchitis efforts.
- Verra intends to dramatically improve outcomes for ALI patients and their families.
ACUTE Kidney INJury
- Acute Kidney Injury (AKI) is another serious life threatening illness that lacks adequate treatments.
- We now know that specific proteases mediate the development of kidney damage.
- Verra has developed selective inhibitors for the most important of these.
- Development will closely track that for VTH245 - particularly CMC.
- VTH144 and its novel target are being validated collaboratively in vivo.