This document discusses a new treatment for hospital-acquired pneumonia (HAP) using a combination of SynAmp, a non-antibiotic compound that enhances antibiotic potency, and an antibiotic. HAP is a major problem with high mortality and increasing antibiotic resistance. The combination therapy is a first-line treatment for HAP that is dosed once daily and shows no resistance after 30 days. It has intellectual property protection and competitive advantages over current treatments. With expedited regulatory pathways, the company aims to launch the primary HAP indication with $200 million in annual sales and expand to other infections. Funding is sought to further develop the pre-clinical candidate.

1. Non-Resistant Antimicrobial
Therapy: Treating Superbugs
That Cause Nosocomial Pneumonia
August 28, 2020

2. What is the problem?
Hospital Acquired Pneumonia
“HAP/VAP is the biggest
problem among already sick
patients, it has the highest
medical complexity.”
-Daniel Hoft, MD, PhD
• 375,000 HAP cases/year in the U.S.
• High mortality rate (30% - 50%) due to
o co-morbidity
o Antibiotic treatment failures due to
antibiotic resistance
• MDR Gram-negative bacteria –
o increasing despite antibiotic
stewardship programs
o Few antibiotic treatment options
• Current treatments
• 14-25+ days long
• economic burden on patients,
hospitals, and insurers.

3. In vitro data:
• Half-life (t1/2)
SynAmp: 1.5 hrs
• CYP2B6 IC50: 7 µM
• Hepatocyte EC50: 2 µM
• ADMET & Safety data
How does product/service solve problem?
A New First-Line HAP Treatment
A non-resistant intravenous combination antibiotic prescribed
as first line treatment for HAP/VAP patients, dosed once daily.
Bacteria bacteria bacteria bacteria
+ SynAmp + antibiotic + SynAmp
+ antibiotic
64x potency No resistance Bioavailability
over 30 days
Enhancement

4. How does product/service solve problem?
Platform Technology
• Penetrates bacterial membrane, disrupts ion balance.
• Disrupts resistance mechanisms in bacteria and
allows more antibiotics to penetrate the bacteria.
• Works with various antibiotics and against various
microbes.
E. Coli SynAmp in membrane

5. What is the market use?
Current Product Price HAP/VAP Patients Addressable Market in US
$2500-$5500 375,000 $935 Million - $2.1 Billion
Initial Indication: HAP/VAP
• Interviewed payers showed willingness to cover
higher prices:
$5,000 - $12,000
• First Year Launch:
$5,000 x 37,000 patients = $185M
• SynAmp has a potential for other parallel indications,
including:
chronic Urinary Tract Infections ($2B market)

6. What competition exists?
Antibiotics for HAP Class
Non-
resistance
Nightmare
Pathogens
Cost
Cefepime, Meropenem
(Current treatments)
𝛃-lactams
Zerbaxa, Avycaz
(approved) 𝛃-lactam drugs +
𝛃-lactamase
InhibitorsMK-7655A
(Investigational)
SYNAMP
New Class of
adjuvants

7. What is the status of the intellectual property?
• US 10,463,044 (issued 2019) – A method of reversing
the resistance of a multi-drug resistant
bacterium….by three different classes of molecules
o One additional patent application allowed (US 2017-0347652); one
application pending (US 2019-0201379); and one currently being
drafted
• License of patent rights to be acquired by Upaya
Pharmaceuticals from UMSL.

8. Regulatory Strategies
• Single vial package: SynAmp + antibiotic.
• HAP is an unmet need
• Expedited pathways: Fast-track approval & priority
review.

9. Development Stage
• Recently funded by NIH via MU MBArC bootcamp
program with 100% match from University - $200,000 for
non-GLP pre-clinical studies (de-risk)
*Initial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT)

10. Upaya Pharmaceuticals Team Members
George W. Gokel, PhD
Principal Investigator
> 40 years of experience in
academia, industry and
pharmaceuticals expert witness
Mohit B. Patel, PhD
Biological Studies
Experienced in biological studies, life-
science entrepreneurship, product
development
Saeedeh Negin, PhD
Chemical Synthesis & Analysis
Experienced in academia and
industry in chemical synthesis,
manufacturing and analysis
Advisers
Bruce Hamper, PhD
Medicinal Chemist
Bill Simon, MBA
CEO, Traxxsson
Ron Dolle, PhD
Director, Center
for Drug Discovery
Team
Michael Kinch, PhD
WashU, Center for
Research Innovation

11. Summary
• Unmet need: Resistant HAP/VAP
• Product: A first of its kind non-resistant antibiotic
• Intellectual property: Platform technology: 1 issued +
additional pending & draft applications
• Competitive Advantages: New class and non-resistance
• Market: Primary indication launch $200 million, multiple
indications
• Regulatory: Expedited pathways (3-4 years, 300 patients)
• Team: Experienced and motivated pre-clinical team
• Seeking: Funding, development partner, experienced team
members (executive and drug developers)