Launch of a new Pharmaceutical Product
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Transcript of Launch of a new Pharmaceutical Product
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...
NOCTUROL™ for NOCTURIA
07.21.2014
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
100M sufferers in US
(2.2B worldwide)
THIS IS A SIGNIFICANT UNMET MEDICAL NEED AS NO CURRENT DRUG TREATS LOW NOCTURNAL BLADDER CAPACITY
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Nocturnal 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.
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INTRODUCING
APAP
ibuprofen
+ time release
Noctural has three synergistic mechanisms of action.
SCIENTIFIC ADVISORS Frank Rauscher, III, Ph.D., Chief Scientist: Dep. Director, Wistar Inst. Khurshid Iqbal, Ph.D., Pharma Chemist, J&J, Hoffmann-LaRoche
Tony DelConte, MD, led Novartis Enablex FDA trials Thomas Garvey III, MD, NIH, FDA, drug safety expert David Shantz, Marketing
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)
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World Class TeamDavid Dill, CEO, IBM, CFO of 7 firms, 35 years as manager
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
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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:
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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
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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
Almost all regulatory bodies outside US permit use of unlicensed drugs (“Specials”). We have an agreement for such distribution of Nocturol.
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
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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
We have protocol feedback and indications of interest from key global pharmaceutical leaders.
Licensee to complete trials, patents, and launch Nocturol. We are expecting an upfront payment after Phase 2 trial (mid-2015), milestone payments, 15% royalty.
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Big Pharma License
Safe Drug, low risk Huge unmet need. Tight patent barrier through 2030
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Strengths of This InvestmentThis 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
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5$787K raised so far primarily through pooled due diligence of Keiretsu Forum.
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Use of FundsPrimary use of funds will be in the management of the trial process and preparations for Ethicor launch.
2014 Q1 Q2 Q3
Ethicor GP Rev Share $42 $125 $221
Expenses
Mfg. Testing $500 – – –
Clinical Trials $612 $425 – –
Business Dev. $108 $29 $33 $38
Patents/ Legal WW $396 $130 $130 $90
G&A / other $475 $55 $50 $50
TOTAL Expenses $2,095 $639 $213 $178
Net Income ($2,095) ($597) ($88) $43
Figures expressed as $k
2015
Profits in Q3 2015
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Projections
2014 2015 2016 2017 2018 2019 2020 2021
Ethicor GP Rev Share $1 $7 $27 $51 $73
Big Pharma Milestones – $13 $5 $5 $38
Big Pharma Royalties – – – – $15 $77 $307 $768
TOTAL Revenue $0 $13 $12 $32 $103 $150 $307 $767
Expenses $2 $2 $1 $2 $6 $8 $16 $39
Net Income ($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)
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
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Milestones7/2010
4/2012
4/2014
Q3/2014
Q2/2015
Q3/2015
2018
Q1/2015
• 22 patients informally tested Nocturnal for a Johns Hopkins physician. 17 reported good results (fewer nightly bathroom trips). (77%)
• 18 patients have since tested Nocturol, 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 - Nocturnal addresses PGE2 levels.
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Anecdotal Evidence
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
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Supporting Research
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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
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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
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Clinical Trial DesignInclusion 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)
• Nocturol, APAP, ibuprofen, placebo
• Show Nocturol’s superiority in reducing nocturia
• Delay time to first awakening
• Improve quality of life
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Series A vs Series BBoth 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