Corporate Overview
This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts and referenced statistics, contained in this presentation, including statements regarding future company strategy, operations, financial position, revenues, projected costs, plans and objectives of management, will be forward-looking statements.
• We envision a world Our Vision where all patients have a therapeutic option )
• We aspire to grow into an organization that fulfills the unmet needs of patients with rare diseases through the development of Our Mission pharmaceuticals and medical devices, either independently or through strategic partnerships )
• Commitment to Patients - We will constantly be seeking new therapeutic innovations for patients with unmet needs Our Values • Innovation - We will be dedicated to sourcing the next big breakthrough in rare disease treatments • Collaboration – We will seek alliances: • Patient advocacy groups • Charitable foundations • University think tanks • Other pharma organizations )
Why Orphan Drugs?
The Facts It’s estimated that 25-30 million Americans • The National Organization for Rare have a rare disease. That’s 1 in 10 people Diseases (NORD) helps quantify the need for orphan drugs • Many of us have friends and relatives affected by a rare disease. Some of More than 90% of rare diseases are without these include: an FDA approved treatment • Thyroid Cancer • Pediatric Crohn’s Disease • Multiple Sclerosis There are approximately • Cystic Fibrosis 7,000 • Muscular Dystrophy rare diseases • Hemophilia • ALS • All Pediatric Cancers National Organization for Rare Disorders, n.d., https://rarediseases.org/advocate/rareinsights/nord- rareinsights-rd-facts-2019-cover/ (Accessed 15 May 2020) ri 0).
More About Orphan Diseases • Approximately 80% of rare diseases are caused by underlying genetic defects, which can be hard to identify 1 • Others are caused by exposure to rare and unusual toxins. Some orphan diseases are so uncommon that afflicted individuals might not be correctly diagnosed for many years, and there are instances of afflicted individuals never receiving a correct diagnosis 2 1. Sharma, A. et al. (2010) Orphan drug: development trends and strategies. J. Pharm. Bioallied Sci. 2, 290 – 299 (Accessed 26 May 2020) 2. Schieppati, A. et al. (2008) Why rare diseases are an important medical and social issue. Lancet 371, 2039 – 2041 (Accessed 26 May 2020)
Why Does Big Pharma Usually Avoid Developing Orphan Drugs? • The life of a patent for a drug is 20 years, which begins when the patent is issued • Clinical trials can be lengthy and costly, which absorb much of the patent life: • This limits the length of time big pharma can commercialize a drug and therefore… • Reduces the potential profits for big pharma
• In the United States, the Orphan Drug Act of 1983 was a Orphan law passed to Drug Act of facilitate development of Orphan Drugs — 1983 drugs for rare diseases
FDA • The FDA determines if the Orphan Approval Drug is indeed “intended to treat a rare disease or condition.” Process for • The drug can either affect less than Orphan 200,000 people in the country, or… Drugs is • The drug affects more than 200,000 people in the country, but the FDA Fast- determines there is no reasonable expectation the company will exceed Tracked the costs of developing/producing the Compared drug through sales • The above are usually determined in to the early clinical testing stages Ordinary Approval
Advantages of Being an Orphan Drug Company ✓ The majority of Orphan Drugs qualify for fast-track approval with the FDA ✓ Orphan drugs approved by the FDA have a seven-year marketing exclusivity ✓ Less clinical trials mean a drug can be available to help patients sooner ✓ Manufacturers have pricing power during periods of exclusivity
• Orphan Drug Tax Credit – currently 25% credit on Financial clinical testing expense Incentives • User fee exemption for a new drug application for Orphan • Orphan drugs are also exempt from: ✓ Affordable Care Act Pharmaceutical Fee Drug ✓ Pediatric Research Equity Act Requirements (for Companies non-oncology drugs) ✓ 340 (b) pricing
Pipeline Sales Forecast Worldwide Pipeline sales forecast for orphan and non-orphan drugs worldwide from 2019 to 2024 (in billion U.S. dollars)* Mikulic, M. A. (n.d.). Orphan vs. non-orphan drugs pipeline sales forecast worldwide 2019-2024 . Statista. https://www.statista.com/statistics/866702/pipelines-sales-worldwide-orphan-vs-non-orphan-drugs-forecast/ (Accessed 29 May 2020)
Orphan Drug Designation Trends Note: Designations granted in a given year may include requests received from that year as well as previous years. Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
Orphan Drug Designations by Treatment Category in 2018 Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
Rare Pediatric Disease (RPD) Designation • Office of Orphan Product Development (part of FDA) roles/responsibilities: – Co-administered with Office of Pediatric Therapeutics – Grant special status (“RPD designation”) to products that meet eligibility criteria (prevalence less than 200,000; serious/life-threatening manifestations primarily affect those 18 years and under) • Designated products may qualify for RPD Priority Review Voucher Program • May be redeemed to receive a priority review of a subsequent marketing application for a different product • May be transferred (including by sale) Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
Rare Pediatric Disease (RPD) Designation Rubio, T. and Wolanski, N., n.d. Introduction To The Office Of Orphan Products Development (OOPD). [online] Fda.gov. Available at: <https://www.fda.gov/media/128018/download> [Accessed 27 May 2020].
Out-Licensing • Other countries also have orphan drug guidelines and therapeutic solutions can potentially be out-licensed. Some of these countries could include: - Australia - Brazil - Canada - European Union - Hong Kong - India - Japan - Singapore - South Korea - Taiwan
Potential Partnership Opportunities • We plan to build our portfolio of rare disease therapeutics principally based on collaborations and licenses with both academic and corporate partners • We will constantly seek new opportunities for partnerships at all phases of development with: ✓ Patient Advocacy Groups ✓ Universities ✓ Global Big Pharma ✓ NIH ✓ Charitable Foundations
How We Will Work • We will form strategic partnerships with various organizations • Utilize CROs (contract research organization) to reduce development costs • Take advantage of various grants • Capitalize on the fast track approval process under FDA guidelines for orphan drugs • Out-license other parts of the world to generate income (AKA non-diluted financing Asia/Europe)
Our Team
About Us ✓ Experienced management team with multiple transactions in the Orphan Drug Space ✓ Real-world medical expertise ✓ Developing a first-class scientific advisory board • In discussions with former industry leaders in the drug development field to establish robust board leadership ✓ Strong ties with multiple patient advocacy groups
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