Company Overview
December 2022
Company Description
Urica Therapeutics Inc. is a clinical-stage biopharmaceutical company developing novel drugs to address
the significant unmet needs for the treatment of gout and other conditions associated with hyperuricemia
Urica’s lead asset, Dotinurad, is a differentiated oral URAT1 inhibitor approved in Japan with long patent
life, excellent long-term efficacy and a strong safety profile
In the U.S. market, Dotinurad may have $1B sales potential for the treatment of gout and potential for multi-
billion dollar expansion opportunities in other indications (Urica also owns exclusive rights for the EU, UK,
and Canada)
Dotinurad is being developed based on a proven regulatory pathway with Phase 1 trials currently underway
in the U.S., and a plan to launch a Phase 2 trial in 2023; successful Phase 2 trial may lead to significant
value creation
2
Dotinurad Products are investigational and not approved for use in the United States.
Lead Asset Dotinurad
Dotinurad is approved in Japan for hyperuricemia with or without gout
1,2
Japanese approval is based on 1000+ subjects treated in 17 clinical trials
500+ patients received Dotinurad in 3 Phase 3 trials up to 58 weeks in duration
4-6
4mg Dotinurad achieved 100% patient sUA <6mg/dL at 58 weeks
3
Well-tolerated with excellent adverse event profile
No significant cardiovascular, hepatic or renal adverse events
4-6
Label allows use in gout patients with mild-to-moderate CKD
Dotinurad has been widely licensed:
Eisai China & Southeast Asia
Standard Chem & Pharm Co. Ltd Taiwan
Urica U.S., EU, UK, and Canada
3
Dotinurad Products are investigational and not approved for use in the United States.
1. https://pubmed.ncbi.nlm.nih.gov/31754883/
2. https://www.fujiyakuhin.co.jp/medicine/m-fujiyakuhin/
3. 2mg Dotinurad resulted in 89.1% and 91.3% sUA under target Week 34 and 58
4. https://pubmed.ncbi.nlm.nih.gov/31980978/
5. https://pubmed.ncbi.nlm.nih.gov/31970593/
6. https://pubmed.ncbi.nlm.nih.gov/31875931/
Markets
Gout market is directly addressable by Dotinurad
More than 10 million U.S. adults are diagnosed with gout
Currently available gout drugs are limited by either moderate efficacy or considerable safety issues
Estimated 2.5 million U.S. gout patients could directly benefit from Dotinurad
Chronic kidney disease (CKD) market is potentially addressable by Dotinurad
Kidney disease is the leading cause of death in U.S. and ~37 million U.S. adults have CKD
There is a significant overlap between patients with gout and CKD
Japanese clinical data suggest that Dotinurad may confer a renal protective effect
Dotinurad has the potential to address the massive CKD market with a novel mechanism
4
Dotinurad Products are investigational and not approved for use in the United States.
Current and Future Treatment Algorithm for Gout
5
Severity of Disease
First-line therapy Second-line therapy Third-line therapy
Xanthine Oxidase Inhibitors (XOI) URAT-1 Inhibitors
(Currently not available in U.S.)
Uricases
Type of therapy
Allopurinol is the standard of care
Febuxostat is more potent than allopurinol
but used less because of black box warning
Lesinurad was approved in 2015 but
withdrawn due to dose-limiting side effects
and limited efficacy at tolerated doses
Ardea Bioscience, the developer of
Lesinurad, was acquired by AstraZeneca
for $1.3 billion in 2012 based on the
promise of the class after P2
Dotinurad is the successor with what
appears to be improved efficacy and safety
profile
Uricase, i.e. Krystexxa®,requires
supervised administration due to the
potential of life-threatening side effects
Sales of Krystexxa® were $566 million in
2021 with double digit year-on-year
growth
Treatment
overview
$100 - $1,000 ~$5,000 $300,000+Annual cost
Newly diagnosed patients XOI-refractory patients Chronic refractory patientsEligible patients
Beyond Gout, Hyperuricemia is associated with other Chronic Conditions
6
Diabetes CKD/Kidney Stone Hypertension/CVD Fatty Liver Disease
There is significant
overlap between HU
and type 2 diabetes
It has been
suggested that there
is a bidirectional
association between
gout and diabetes
HU can induce
nephropathy and uric
acid kidney stones
Reducing serum uric
acid can mitigate risk
of renal failure due to
nephropathy
Urate lowering
therapy has been
shown to reduce
albuminuria in CKD
1
HU is considered a
predictor for the
development of
hypertension and
CVD
Urate lowering
therapy has been
shown to reduce
blood pressure of
adolescents with HU
and essential
hypertension
2
HU increases the risk
of NAFLD
3
Urate lowering
therapy using
Dotinurad has been
shown to attenuate
diet-induced hepatic
steatosis and fat
metabolism in animal
models
4
1. Am J Kidney Dis . 2021 Apr;77(4):481-489; 2. JAMA . 2008 Aug 27;300(8):924-32; 3.
Eur J Gastroenterol Hepatol. 2016 Feb;28(2):132-8; 4. Mol Metab. 2022 Jan;55:101411
Development Plan
Phase 1 trials are currently underway to determine the pharmacodynamics,
pharmacokinetics and safety of Dotinurad in U.S. subjects
A Phase 2 proof of concept gout study is anticipated to launch in 2023
Successful completion of the funded trials is expected to lead to significant value
creation and the possibility of multiple potential exit options, such as an IPO or a
corporate partnership, by as early as 2024
7
Dotinurad Products are investigational and not approved for use in the United States.
The Company has Assembled an All-Star Team
8
Executive Management
Jay Kranzler, MD PhD
Chairman, Chief Executive Officer & Director
Founding Member, McKinsey Pharmaceutical Practice
24 years as CEO of 2 public companies (with 2 rheumatology product approvals), followed by senior executive position at Pfizer
Professor at NYU Medical & Business Schools
Founder, Board Member, and Advisor to an extensive number of companies and institutions
Raymond Zheng, PhD MBA
Founder and Chief Scientific Officer
Experienced Biotech Entrepreneur with combined 20+ years of experience in drug research, discovery and development
Previously held multiple business development roles at Fortress Biotech, Agenus, and Harvard Medical School
PhD from University of California, Riverside; Fellowship at Harvard Medical School; MBA from Rutgers University
Scott Baumgartner, MD
Interim Chief Medical Officer
Previously VP of Medical Affairs at AstraZeneca/Ardea Biosciences and Amgen
Experienced in the clinical development and post-launch activities of Lesinurad and Verinurad
MD from University of Washington with 20 years of clinical rheumatology experience
Board of Directors
Paul Brooke
Co - Chairman
Chairman Emeritus of the Board of Caelum Biosciences, acquired by AstraZeneca in Oct 2021
Founder and former Managing Partner of venBio, a pharmaceutical investment company
Former global head of healthcare research and strategy at Morgan Stanley
Lindsay Rosenwald, MD
Founder & Director
Chairman, President, and CEO at Fortress Biotech (NASDAQ: FBIO)
Previously, Founder of multiple biotech companies: Cougar Biotechnology (acquired by JNJ), Caelum Biosciences (acquired by Astra
Zeneca), Chelsea Therapeutics (acquired by Lundbeck), Indevus Pharma (acquired by Endo), Biocryst, Keryx, CorMedix, and Ziopharm
Vibeke Strand, MD
Director
Renowned rheumatologist and Consultant and Advisor for three decades to companies, FDA, and professional societies in the field
Participated in 30+ rheumatology drug approvals
Academic appointments at UCSF and Stanford, each for 10+ years with ~500 publications in the field
Scientific Advisory Board
Robert Terkeltaub, MD
Univ. of California, San Diego, Chief of Rheumatology
Professor of Medicine, UCSD, and Section Chief of Rheumatology at the VA Medical Center in San Diego
Associate Editor of Arthritis and Rheumatism, and serves on numerous NIH Study Sections in arthritis
Graduate of McGill University, where completed his medicine residency and rheumatology fellowship
Dr. Michael Pillinger, MD
New York Univ., Chief of Rheumatology
Chief of Rheumatology at VA NY Harbor Healthcare System, Professor of Medicine at NYC Grossman School of Medicine
Received medical degree from NYU School of Medicine; has practiced rheumatology for more than 20 years
Dr. Lee Simon, MD
Medical and Regulatory Consultant
FDA's Division Director of Analgesic, Anti-inflammatory and Ophthalmologic Drug Products (2001-2003)
Long-time NIH-funded investigator, two-term ACR Board of Directors, Chair of Education for the ACR and National Arthritis Foundation