Amarani Therapeutics is developing a novel drug delivery system to treat atherosclerotic heart disease. They have conducted 12 interviews with physicians and pharmacists to understand the clinical need for alternative treatments to statins. Their drug candidate, D-PDMP, uses a biopolymer encapsulation to inhibit glycosphingolipid synthesis, an alternative mechanism to statins. Preclinical mouse studies show D-PDMP is 10 times more effective than the drug alone and has a different mechanism of action than competitors. Amarani plans to continue validating the clinical need and market opportunity through additional expert interviews to support further development and partnerships.

1. Amarani Therapeutics.Inc
• Grand Number of Interviews # 12
• Total Phone Interviews # 8 (Physicians)
• Total In person interviews # 4 (Pharma)
• Number of new interviews since Midpoint meeting # 2
• Atherosclerotic heart disease (AHD) & related diseases contribute to ~50% mortality in West
• One third of patients on Statins (Standard Treatment of Care for lipid reduction)are intolerant
• Novel approach to tackle AHD by inhibiting GSL synthesis, alternative to Statins
GSL(fatty compounds having sugars attached, enriched in atherosclerotic plaques)
• Biopolymer-encapsulated glycosphingolipid synthesis inhibitor drug in preclinical mouse model
Drug D-PDMP (glycosphingolipid synthesis inhibitor) + Drug delivery system SA-PEG (biodegradable encapsulated polymer)
• Drug delivery system increases efficacy of drug by ten- folds .
• Different Mechanism of Action than competitors (STATINS or PCSK9)
• Oral form of drug vs. an injectable monoclonal antibody
• Polymer components are FDA approved.
• Johns Hopkins University has filed a national phase PCT covering this technology in
April, 2015.

2. Team Members & Role
• Dr. Subroto Chatterjee
– Principal Investigator
PhD from the University of Toronto
Fellowship at Michigan State University
Professor of Medicine and Pediatrics at the Johns Hopkins University.
Has published ~150 peer reviewed publications. Has ~27 patents .
Has received national and international awards for his research and community service.
• Shubhda Roy
– Entrepreneurial Lead
Second Masters in Biotechnology from Johns Hopkins University (will complete in May 2016)
– Core Molecular Targets & Drug discovery & Entrepreneurship
First Masters in Information Science from Penn State
Currently working for Siemens Medical in Healthcare IT
• Nina Urban
– Mentor
Associate Director, Fast-forward. Baltimore, Maryland. Johns Hopkins.
Acting CEO – General Genomic, LLC

3. BUSINESS MODEL CANVAS – FIRST VERSION
• Decrease plaque
formation
• Lower Cholesterol
• Decrease High
Blood Pressure
• Lower chances of
cardiovascular
diseases
• Increase good
cholesterol(HDL)
• Decrease bad
cholesterol(LDL)
• AHD Patients
Key
Partners
Key
Activities
Value
Proposition
Customer
Relationships
Customer
Segments
Key
Resources
Channels
Cost Structure Revenue Streams

4. BUSINESS MODEL CANVAS – FINAL VERSION
• Lowers risk of
heart attack
for statin
intolerant
patients
• Unlike
statins D-
PDMP
maintains
normal blood
sugar levels
• Big Pharma
• Established
Biotech
• Angel Investors
End User
• AHD Patients
• Royalties from Intellectual Property
Key
Partners
Key
Activities
Value
Proposition
Customer
Relationships
Customer
Segments
Key
Resources
Channels
Cost Structure Revenue Streams

5. What we did….
– Shubhda
• Interviewed Physicians in different roles treating
patients with cardiovascular diseases
• To understand unmet clinical need for statin resistant
patient
• 5 cardiologists, 2 Hospitalist (Internal Medicine),
• 1 Cardiology fellow
– Dr. Chatterjee
• Met with Big Pharma scout

6. What we Learned….
– From interviewing Physicians
• Physician's quiet happy with statins. Treat most patients with one
class of statins or another.
• Very rare population allergic or intolerant to statin. It is about 5 %
of population.
• Physicians look at formulary, cost of the drug for prescribing drugs.
• For any new drugs, they look at clinical studies, specifically
outcome, study design, patient population used, adverse effects
• Most patients are over 65 and usually complain about muscle pain
from statins
• Prefer to use oral medication which is cheaper, as compared to
PCSK9 which is the new kid on the block. But it is monoclonal
antibody. No physician had prescribed it since they thought it was
too expensive. Not all insurances are covering it
– From interviewing Big Pharma Scout
• Interested to see data from big animal study

7. What we learned..
– Shubhda interviewed folks from Big Pharma
– Big Pharma looks for
• Head to head study with existing competing drugs in
the market – a TPP (Target Product Profile) and how
competitive is the TPP
• Mechanism of action, unmet clinical need, efficacy &
toxicity
• Clinical end point outcome.
– Will the drug cut hospitalization
– Will it bring down heart attack, lower lipids, Stroke
• Interested in Phase II Trials

8. Prepared a Head to Head Study for a
preclinical mouse model
Atorvastin
(Statin)
Alirocumab
(Antibody)
Atorva+Alirocumab D-PDMP
(AMARANI’s
Drug)
Cholesterol 15 46 58 80
Triglyceride Increased 35 No effect 60
Atherosclerosis 30 88 98 98
Atorvastatin(3.6mpk), alirocumab (10mpk) (PCSK-9 Antibody): ref(J Lipid Res 55; 2014 2103.
www.jlr.org/content/55/10/2103.full.pdf
D-PDMP(10mpk)
http://www.ncbi.nlm.nih.gov/pubmed/26111596

9. Next Steps ?
• Existing studies show 10 Million patients in US alone
are in need of alternative to statins.
• Continue to validate this by speaking to additional
cardiologists and lipid experts from around the country.
– Michael Miller, UMD
– Dan Rader, Upenn
– Joel S. Karliner, VA Med Ctr UC. Sanfransciso (spoken)
– Annabell Rodriguez-Oquendo, Un of Connecticut, Hartfort
– Samia Mora , Harvard
• Will continue to reach contacts in big pharma and
established biotech's.