New Venture Launch: Funding for trials for important new discovery
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Paxerol is an FDA trial-bound solution for nocturia that affects 100 million people in the U.S., presenting a significant unmet medical need with no current effective treatments. The drug combines acetaminophen and ibuprofen in a unique extended-release formulation, aiming to increase nocturnal bladder capacity and significantly improve quality of life. With strong intellectual property protections, a world-class research team, and anticipated sales projections of $2-8 billion, the launch is strategically planned in 2015 through partnerships for distribution.
New Venture Launch: Funding for trials for important new discovery
- 1. 100M people in the US suffer from a problem for which we have a strong solution. The patents are issued, we are raising funds for FDA trial... PAXEROL™ for NOCTURIA 07.21.2014
- 2. Misery associated with nocturia Insomnia, fatigue, depression, lower quality of life US lost work time $60B/year Incontinence care currently 25% of the costs of nursing home care Heart disease, stroke, brain damage, hip fractures NOCTURIA is the #1 urology complaint of men and women 55-84 65%of people ages 55-84 100Msufferers in US (2.2B worldwide) THIS IS A SIGNIFICANT UNMET MEDICAL NEED AS NO CURRENT DRUG TREATS LOW NOCTURNAL BLADDER CAPACITY 2
- 3. Paxerol is a unique 8 hour extended release formulation that provides increased nocturnal bladder capacity (NBC) through the night. (+50-100%) HOW IT WORKS: For 76% of people suffering from nocturia, low NBC is a key issue. The cause is excess Prostaglandins (PGE2) that irritate the bladder. For sufferers of Nocturia and Over Active Bladder, a combination of APAP & ibuprofen will inhibit PGE2 production to increase comfortable bladder capacity. People are informally using the combination of already approved drugs. It is proving effective for 80%. Our next step is to prove efficacy in trials. Our use of these drugs for this purpose and the addition of the timed release aspect are protected by numerous broad patents. 3 INTRODUCING APAP ibuprofen + time release Paxerol has three synergistic mechanisms of action.
- 4. SCIENTIFIC ADVISORS Frank Rauscher, III, Ph.D., Chief Scientist: Dep. Director, Wistar Inst. Khurshid Iqbal, Ph.D., Pharma Chemist, J&J, Hoffmann-LaRoche LEADING CLINICAL EXPERTS Tony DelConte, MD, led Novartis Enablex FDA trials Thomas Garvey III, MD, NIH, FDA, drug safety expert LICENSING AND LEGAL Joy Barton, Ph.D., Novartis, Licensing Agent Ed Allera, Buchanan Ingersoll, FDA Focused Lawyer Ping Wang MD IP Medical Expert Andrews Kurth, (named top 1000 patent Lawyer) 4 World Class Team David Dill, CEO, IBM, CFO of 7 firms, 35 years as manager
- 5. 6 Patents issued in 2012-2014 (16+ year life) • Method of use claims; broad spectrum combinations. • Covers use with Over Active Bladder (OAB) • Extended release critical: 2-3 hour half life standard • Wide range dosing of APAP with antimuscarinics, diuretics, antidiuretics, spasmolytics, and/or NSAIDs. • PCT process for worldwide protection filed 12/2010 5 Intellectual Property 8,236,857 B2 – Extended-release formulation 8,236,856 B2 – Delayed release formulation 8,685,453 B2 – Low dose APAP and NSAIDs 8,445,015 B2 – Extended-release formulation 8,445,011 B2 – Delayed-release formulation 8,703,184 B2 – Delayed-release formulation US PATENTS ISSUED:
- 6. 6 Vesicare, Enablex, Myrbetriq • Effects often take months • Large % do not respond • Severe side effects • 43–83% stop in < 30 days • 10-15% fewer bathroom trips • Treats annoying problem • Significant competition OVERACTIVE BLADDER DRUGS 2M patients (0.1%) $2B SALES • Effects in as little as 30 mins. • 80% positive (anecdotal) • No side effects • 20% drop out (anecdotal) • 33-100% fewer nightly trips • Treats dangerous problem • No current competition $2-8B SALES ? Proving effective for OAB, but focused on treatment of bladder problems related to sleep
- 7. 7 Actionable Market $2-8B SALES 2018 Paxerol is a new product that is effective in ways that current OAB (over active bladder) products are not. *source 2013 Report by Debra E. Irwin et al / Company estimates NOT A SIGNIFICANT PROBLEM 1 x per night 1.2 Billion 423 Million Unaffordable 600 Million 130 Million No efficacy 50 Million Medical Issues $0.30 / dose/daily = $39B annual sales potential (retail) NOTE: Forecasting a new market is highly speculative. 2.4 Billion sufferers es'mated by 2018
- 8. Almost all regulatory bodies outside US permit use of unlicensed drugs (“Specials”). We have an agreement for such distribution of Paxerol. Contract signed April 2014 (Q1 2015 launch) with Ethicor Pharma, Ltd. (UK). Plan to launch Paxerol with support of key opinion leaders, education sessions, and published articles. Partnered with UDG, UK’s largest prescription drug distributor. Ethicor exits when big pharma enters markets. Top end price target – $5/dose controlled by Ethicor Gross profit estimate – 98% Only ages 60+, EU, MENA, and SA, with no advertising 8 Ethicor Short-term Revenues 2015 2016 2017 2018 2019 TOTAL $832 $6,611 $26,949 $50,538 $73,161 $151,091 $k Ethicor Projected Income
- 9. We have protocol feedback and indications of interest from key global pharmaceutical leaders. Licensee to complete trials, patents, and launch Paxerol. We are expecting an upfront payment after Phase 2 trial (mid-2015), milestone payments, 15% royalty. 9 Big Pharma License
- 10. Safe Drug, low risk Huge unmet need. Tight patent barrier through 2030 10 Strengths of This Investment This is not a new chemical entity. Approval can be attained with a simpler, relatively inexpensive trial: Avoids Phase 1 Trial safety tests, and years of risk from unexpected side effects. Strong Ethicor validation. International sales begin before trial completion, mitigates risk. Mid-2015 worldwide big pharma license. Strong ROI @ up to 94% net profit. $3M to positive Cash flow 12-15 months 1 2 3 4 5 $787K raised so far primarily through pooled due diligence of Keiretsu Forum.
- 11. 11 Use of Funds Primary use of funds will be in the management of the trial process and preparations for Ethicor launch. Ethicor GP Rev Share Expenses Mfg. Testing Clinical Trials Business Dev. Patents/ Legal WW G&A / other TOTAL Expenses Net Income 2014 Q1 Q2 Q3 $42 $125 $221 $500 – – – $612 $425 – – $108 $29 $33 $38 $396 $130 $130 $90 $475 $55 $50 $50 $2,095 $639 $213 $178 ($2,095) ($597) ($88) $43 Figures expressed as $k 2015 Profits in Q3 2015
- 12. 12 Projections Ethicor GP Rev Share Big Pharma Milestones Big Pharma Royalties TOTAL Revenue Expenses Net Income % 2014 2015 2016 2017 2018 2019 2020 2021 $1 $7 $27 $51 $73 – $13 $5 $5 $38 – – – – $15 $77 $307 $768 $0 $13 $12 $32 $103 $150 $307 $767 $2 $2 $1 $2 $6 $8 $16 $39 ($2) $11 $11 $30 $97 $142 $291 $729 – 84% 92% 94% 95% 95% 95% 95% Figures $Millions Key Assumptions 1. FDA approval 2018 (no accelerated approval designation) 2. $0.30 retail ($0.15 wholesale) in 2018 3. 15% License fee (15-18% tiered structure would add $120M in 2021) 4. Licensing Agent 5% fee 5. A $5B big pharma drug by 2021 (no OAB or bedwetting solution)
- 13. Patents filed (issued 2012, 2013, & 2014) FDA Confirms no Phase 1 safety tests required EU“Special”Ethicor contract (launch Q1/2015) Series A complete and trial contract signed 1st patient starts trial: 13 week crossover design; patients with 3+ voids/night 60 patient trial completed. Sign big pharma license (Series A cash back) FDA approval H1- Paxerol launch 6 weeks later 13 Milestones 7/2010 4/2012 4/2014 Q3/2014 Q2/2015 Q3/2015 2018 Q1/2015
- 14. • 22 patients informally tested Paxerol for a Johns Hopkins physician. 17 reported good results (fewer nightly bathroom trips). (77%) • 18 patients have since tested Paxerol, with 15 reporting good results (1-4 fewer nightly bathroom trips). • Placebo effects are always possible, but much less likely when sleeping. • 32 of 40 patients (80%) self-report success – without placebo controls. • 3 articles report OAB suffers’urine averages 700% higher PGE2 levels - Paxerol addresses PGE2 levels. 14 Anecdotal Evidence
- 15. Independent clinical studies consistently report that ~70% of nocturia patients experience improvement when taking appropriate doses of certain NSAIDs 80% of patients with neurogenic bladder and 74% of patients with BPH experience improvement in nocturia when taking appropriate doses of NSAIDs (consistent with animal studies) Johns Hopkins proves NSAIDs reduce bladder spasms Ohio State preclinical model of bladder function showed acetaminophen equal to NSAIDs in reducing inflammation, achieved through independent mechanisms of action Together acetaminophen and NSAIDs impact multiple independent molecular pathways to relieve nocturia symptoms 15 Supporting Research
- 16. No matter how nice your bathroom is, nobody wants to visit in the middle of the night
- 17. 17 OAB Market Segment $2.1B SALES 2011 *source 2013 Report by Debra E. Irwin et al / Company estimates Astellas VesiCare 3.8M 1.6 M Enablex In the 12 months ending January 2012, more than 17.8 million prescriptions were filled for drugs in the Over Active Bladder, accounting for almost $2.1 billion in sales. Oxybutynin 3.7M Oxybutynin ER 3.6M Pfizer Detrol LA 3.3 M 712 k Pfizer Toviaz
- 18. 18 A Serious Problem * Results from the Third National Health and Nutrition Examination Study 12 year survival odds for women ages 65-90 based on nightly voiding episodes: 0 voids 68% 1 void 60% 2 voids 50% 3+ voids 22% Association of Nocturia and Mortality: Results From the Third National Health and Nutrition Examination Study
- 19. 19 Clinical Trial Design Inclusion Criteria • Those who use bathroom 3+ times/night • Those with elevated PGE2 levels Exclusion Criteria • Congestive heart failure, diabetes, etc. • Those with major arthritis or muscle pains 13 week double blind crossover study • Each patient tests all alternatives (to normalize variables) • Paxerol, APAP, ibuprofen, placebo • Show Paxerol’s superiority in reducing nocturia • Delay time to first awakening • Improve quality of life
- 20. 20 Series A vs Series B Both share 50% of returns until made whole for initial investment Series A thereafter gets returns based on $1.10/share Series B gets 50% until 15X invested $ back (1-4 years) Inflated returns come from Founders, not Series A Series B thereafter get returns based on $2.20/share