Soligenix, Inc. (SNGX.OB)

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OneMedForum New York Company Presentation: Soligenix, Inc., a late-stage biopharmaceutical company, focuses on the development of products to treat life-threatening side effects of cancer treatments. …

OneMedForum New York Company Presentation: Soligenix, Inc., a late-stage biopharmaceutical company, focuses on the development of products to treat life-threatening side effects of cancer treatments. Learn More at: http://www.onemedplace.com/database/list/cid/14016

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  • 07/12/10 Contains forward looking statements that reflect Soligenix’s current expectations about its future results, performance, prospects and opportunities. I would encourage everyone to consult our most recent annual report to familiarize yourself with the risks associated with the type of work we do.
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  • 07/12/10 Thank you Dina. Again, that was Dina Lyaskowitz of Ibis Consulting, who does investor relations work for Soligenix, Inc. My name is Geoff Green, I am the President and Acting CEO of Soligenix. Before I get into the presentation I’d like to remind everyone (next slide) that this presentation contains
  • 07/12/10 Thank you Dina. Again, that was Dina Lyaskowitz of Ibis Consulting, who does investor relations work for Soligenix, Inc. My name is Geoff Green, I am the President and Acting CEO of Soligenix. Before I get into the presentation I’d like to remind everyone (next slide) that this presentation contains
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  • 07/12/10 Thank you Dina. Again, that was Dina Lyaskowitz of Ibis Consulting, who does investor relations work for Soligenix, Inc. My name is Geoff Green, I am the President and Acting CEO of Soligenix. Before I get into the presentation I’d like to remind everyone (next slide) that this presentation contains
  • 07/12/10
  • 07/12/10
  • 07/12/10 Thank you Dina. Again, that was Dina Lyaskowitz of Ibis Consulting, who does investor relations work for Soligenix, Inc. My name is Geoff Green, I am the President and Acting CEO of Soligenix. Before I get into the presentation I’d like to remind everyone (next slide) that this presentation contains
  • 07/12/10
  • 07/12/10 Thank you Dina. Again, that was Dina Lyaskowitz of Ibis Consulting, who does investor relations work for Soligenix, Inc. My name is Geoff Green, I am the President and Acting CEO of Soligenix. Before I get into the presentation I’d like to remind everyone (next slide) that this presentation contains
  • 07/12/10
  • 07/12/10
  • 07/12/10

Transcript

  • 1. OTC BB: SNGX
  • 2. Forward-Looking Statements
    • This presentation contains forward-looking statements that reflect Soligenix, Inc.'s current expectations about its future results, performance, prospects and opportunities. Statements that are not historical facts, such as "anticipates," "believes," "intends," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements. Soligenix cannot assure you that it will be able to successfully develop or commercialize products based on its technology, including orBec ® , SGX201, RiVax™, and LPM TM , particularly in light of the significant uncertainty inherent in developing vaccines against bioterror threats, manufacturing and conducting preclinical and clinical trials of vaccines, and obtaining regulatory approvals, that its cash expenditures will not exceed projected levels, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further grants and awards, maintain its existing grants which are subject to performance, enter into any biodefense procurement contracts with the US Government or other countries, that the US Congress may not pass any legislation that would provide additional funding for the Project BioShield program, that it will be able to patent, register or protect its technology from challenge and products from competition or maintain or expand its license agreements with its current licensors, or that its business strategy will be successful. Important factors which may affect the future use of orBec® for gastrointestinal GVHD include the risks that: the FDA's requirement that Soligenix conduct additional clinical trials to demonstrate the safety and efficacy of orBec ® will take a significant amount of time and money to complete and positive results leading to regulatory approval cannot be assumed; Soligenix is dependent on the expertise, effort, priorities and contractual obligations of third parties in the clinical trials, manufacturing, marketing, sales and distribution of its products; orBec ® may not gain market acceptance if it is eventually approved by the FDA; and others may develop technologies or products superior to orBec ® . Factors affecting the development and use of SGX201, RiVax™, and LPM TM are similar to those affecting orBec ® . These and other factors are described from time to time in filings with the Securities and Exchange Commission, including, but not limited to, Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.
  • 3. Soligenix, Inc. is a late-stage, biopharmaceutical company developing products to treat life-threatening side effects of cancer treatments and serious gastrointestinal diseases, and vaccines for certain bioterrorism agents Soligenix
  • 4. Value Proposition
      • Low current valuation of approximately $60 million
        • Potential for exponential growth with the achievement of a single milestone – A positive confirmatory Phase 3 clinical trial of orBec ®
        • Relatively low risk and late stage drug development proposition due to existing orBec ® Phase 3 data
      • Four active clinical trials
      • Clean capital structure with no debt or preferred stock outstanding
      • FDA SPA and EMEA agreement on Phase 3 protocol supports regulatory path to approval in US and EU
      • 35% royalty generating North American partnership with Sigma-Tau on orBec ®
      • orBec ® addresses a potential $400 million global GVHD market, where there is an unmet medical need with poor therapeutic options
      • Oral BDP application in other GI areas - Radiation Enteritis and Crohn’s Disease; markets in excess of $500 million worldwide 
      • LPM™ - novel oral delivery system for drugs such as leuprolide
      • Diversified portfolio: BioTherapeutics and BioDefense
        • BioDefense - Grant funded and revenue generating – New $9.4 million NIH grant
          • Soligenix is world leader in ricin toxin vaccine research
  • 5. Pipeline FAST TRACK and ORPHAN DESIGNATION ORPHAN DESIGNATION ORPHAN DESIGNATION FAST TRACK DESIGNATION ORPHAN DESIGNATION Programs highlighted in green are supported in whole or in part by NIH funding . Biotherapeutics   Preclinical Phase 1 Phase 2 Phase 3 orBec® Treatment of GVHD         orBec® Prevention of GVHD         orBec® Treatment of Chronic GVHD         SGX201 Radiation Enteritis         SGX203 Crohn’s Disease         LPM™ Leuprolide Prostate Cancer/Endometriosis         Biodefense   Proof of Concept Animal Phase 1 Phase 2/3 RiVax™ - Vaccine Ricin Toxin Exposure         SGX202 - Therapeutic Radiation Injury         FDA ANIMAL RULE FDA ANIMAL RULE
  • 6. Management Team Executive Yrs. Exp Industry Experience Christopher J. Schaber, PhD 21 Discovery Laboratories, Inc. (COO) President and CEO Acute Therapeutics, Inc. (Co-Founder) Ohmeda PPD, Inc. The Liposome Company, Inc. Wyeth Ayerst Evan Myrianthopoulos 15 CVL Advisors Group, Inc., - Life science Chief Financial Officer financial consulting firm Discovery Laboratories (CFO, VP Finance) Paramount Capital Investments, LLC Brian Hamilton, MD, PhD 30 Astra, USA Chief Medical Officer Wyeth Research Alkermes, Inc. University of Washington Robert Brey, PhD 27 Lederle-Praxis, division of American Chief Scientific Officer Cyanamid Vaxcel, Inc.
  • 7. orBec ® Gastrointestinal Graft-versus-Host Disease (GI GVHD) An Unmet Medical Need BioTherapeutics – Lead Program
  • 8. Gastrointestinal GVHD
    • Unmet medical need – orphan disease
    • Occurs in leukemia and other blood cancer patients who undergo bone marrow or stem cell transplantation
    • Patients’ tissue attacked by lymphocytes of donor marrow/stem cells
    • Most debilitating symptoms occur in stomach and small intestine
    • Symptoms include anorexia, nausea, vomiting, bloody stool, necrosis and exfoliation of endothelial cells
    • Treated with high dose systemic prednisone – for lack of better treatment
    Normal Upper GI Mucosa Acute GvHD
  • 9.
    • orBec ® (oral beclomethasone dipropionate or BDP)
      • Beclomethasone – Potent, topically active, anti-inflammatory steroid
        • Designed to treat inflammation within lining of GI tract
      • orBec ® strategy is to decrease need for prolonged use of high dose systemic prednisone
      • Treatment with prednisone: effective, but highly toxic
        • Increases susceptibility to opportunistic infection
        • Inhibits positive Graft-versus-Leukemia (GvL) effect
      • orBec ® clinical advantage gained by:
        • Fewer opportunistic infections
        • Enhanced GvL effect - fewer relapses, improved survival
        • Shortening of prednisone course
        • Sustained remission of GI GVHD
      • Fast Track and Orphan Drug Designations for treatment of acute GI GVHD
    orBec ®
  • 10. orBec ® - A Targeted Approach to GI GVHD
    • A Two-Pill System
      • Each tablet contains 1 mg BDP
      • 1 Immediate Release (IR) tablet designed to release in the upper GI tract and 1 Enteric Coated (EC) tablet designed to release in the lower GI tract
      • Total of only 8 mgs BDP per day
    Diagram showing dispersion of IR tablet in the stomach Diagram showing dispersion of IR and EC tablets in small intestine
  • 11. orBec ® - Risk/Benefit Analysis
    • Clinical Significance:
      • Statistically significant reductions in GVHD treatment failures
      • Statistically significant increase in daily caloric intake ability
      • Statistically significant reductions in mortality
    • Safety:
      • Well established – beclomethasone known to FDA
      • No major side effects seen in orBec ® trials
      • Superior safety profile compared to high dose prednisone
      • Beclomethasone use previously approved by FDA:
        • Becloforte ® – inhalant marketed by Glaxo and used to treat asthma
        • Beconase ® – nasal spray marketed by Glaxo for rhinitis
        • Propaderm ® – topical cream for psoriasis
    Positive Phase 2 and 3 clinical trial data provide strong support for Confirmatory, Pivotal Phase 3 Study
  • 12. McDonald et al . 1998. Gastroenterology
    • Primary endpoint achieved – Positive response to orBec ® therapy at Day 30
      • Positive response defined as the ability to consume > 70% of expected daily caloric requirements
      • Rapid control of GI GVHD not requiring additional prednisone
    • Secondary endpoint achieved
      • Positive treatment response through Study Day 40
    orBec ® - Phase 2 Study Results 60-patient randomized, double-blind, placebo-controlled, single center trial Endpoints orBec ® n=31 Placebo n=29 p-value Treatment Responders at Day 30 ( Primary endpoint ) 22 (71%) 12 (41%) 0.021 Treatment Responders at Day 40 16 (52%) 5 (17%) 0.005
  • 13. orBec ® - Previous Phase 3 Study Results Time to Treatment Failure through Day 80 p = 0.0226 129-patient randomized, double-blind, placebo-controlled, multicenter trial Hockenbery et al. 2007. Blood Endpoints orBec ® n=62 Placebo n=67 p-value Time to Treatment Failure through Day 50 ( primary endpoint ) 0.118 Treatment Failure Rate at Day 50 18 (31%) 30 (48%) 0.051 Time to Treatment Failure through Day 80 0.023 Treatment Failure Rate at Day 80 22 (39%) 39 (65%) 0.005 Mortality Rate at 200 Days Post-Transplant 5 (8%) 16 (24%) 0.014 Placebo BDP 0.00 0.25 0.50 0.75 1.00 Days since randomization 0 10 20 30 40 50 60 70 80 90
  • 14. orBec ® - Comprehensive Mortality Data Hockenbery et al. 2007. Blood Long-Term Survival Outcomes orBec ® Placebo p-value orBec ® vs. placebo Percentage reduction in mortality Mortality Rate at 200 days post transplant - Pivotal Phase 3 study 5 (8%) 16 (24%) .013 66% Mortality Rate at 200 days post transplant - Prior Phase 2 study 3 (10%) 6 (21%) 0.18 55% Mortality Rate among mismatched donors at 200 days post transplant Pivotal Phase 3 study 1 (4%) 10 (42%) 0.02 94% Mortality Rate at 1 year post randomization - Pivotal Phase 3 study 18 (29%) 28 (42%) 0.04 46% Mortality Rate at 1 year post randomization - Prior Phase 2 study 6 (19%) 9 (31%) 0.26 45% Mortality Rate at median time periods at 3.5 years – Both studies combined 37 (40%) 49 (51%) 0.03 37%
  • 15. orBec ® Clinical Development Path Forward
    • Special Protocol Assessment (SPA) for confirmatory, pivotal Phase 3 clinical trial cleared by FDA
      • Highly powered clinical trial of similar design to previous Phase 3 study is acceptable to support a regulatory submission seeking new drug approval
      • Enrollment sample size – approximately166 patients
      • New Primary Endpoint – Treatment failure rate at Study Day 80 – highly statistically significant in previous Phase 3 trial with p-value of 0.005
    • EMEA Agreement on Phase 3 protocol for potential EU approval
    • With 2 positive trials completed – strong basis for confidence in efficacy of confirmatory trial
    • Trial initiated; completion targeted for 1H 2011
  • 16. Confirmatory Phase 3 Replicates Prior Study
    • Improvements in design including change of primary endpoint increase likelihood of success
    • The key factors - drug, dose, patient population and expected control group outcomes - have remained constant since the prior Phase 3
    Prior Phase 3 Confirmatory Phase 3 Number of sites Multicenter Multicenter Number of patients 129 166 Patient population Allogeneic transplant patients with Grade 2 GI GVHD Same Powering 80% at a two-sided significance level of 0.05 90% at a two-sided significance level of 0.05 Primary endpoint Time to Treatment Failure Through Day 50 Treatment Failure Rate at Day 80 (p-value of 0.005 in prior Phase 3) Dosing regimen 8 mg BDP per day / 1 mg BDP per tablet / dosing duration 50 days Same Design 2 Randomized groups: High dose prednisone for 10 days followed by rapid taper with 50 days on placebo or drug Same
  • 17. Sigma-Tau Partnership and Commercialization
    • Marketing partnership with Sigma-Tau aimed at launching orBec ® in North America
      • 35% royalty on net sales back to Soligenix
      • $10 million in milestones with $1 million milestone received upon enrollment of first patient in confirmatory trial
      • Soligenix to lead all R&D and regulatory
    • Certain inherent market factors suggest quick launch and significant penetration
        • No competition – Nothing approved by FDA for GI GVHD
        • Rapid dissemination of information
        • Focused market – 15 transplant centers do 60% of all transplants
    • Soligenix seeking marketing partners in Europe and ROW
  • 18. Lifecycle Management – New BDP Trials Program Purpose # of Available Patients Status Prevention of GVHD Decrease incidence or severity of acute GVHD 10,000+ US Phase 2 – 140 patients enrollment completed 1H 2010 Radiation Enteritis Block inflammatory component of acute radiation enteritis in GI tract in rectal cancer patients receiving radiation therapy 50,000+ US Initiated. Supported by $500,000 NIH grant Fast Track granted Completion targeted 1H 2011 Chronic GI GVHD Decrease need for systemic immunosuppressive therapy in chronic GI GVHD 6,000+ US Phase 2 protocol Initiation targeted for 2H 2010 Crohn’s Disease Minimize inflammation in chronic GI conditions 500,000 US 100,000 Pediatric US TBD
  • 19. Worldwide Potential Oral BDP Market $300 $100 0 50 100 150 200 $ Millions 250 300 $200 orBec ® GVHD Treatment $120 400 450 350 orBec ® GVHD Prevention orBec ® GVHD Chronic Oral BDP Radiation Enteritis Oral BDP Crohn’s Disease >$500 500 Assumptions
    • ~ 20,000 allogeneic transplants annually
    • ~ 7% annual market growth
    • Acute GI GVHD Treatment
      • 5,000 Patients US
      • 5,000 Patients Europe
    • Acute GVHD Prevention
      • 10,000 Patients US
      • 10,000 Patients Europe
    • Chronic GI GVHD Treatment
      • 6,000 Patients US
      • 6,000 Patients Europe
    • Radiation Enteritis
      • 50,000 Patients US
      • 50,000 Patients Europe
    • Crohn’s Disease
      • 500,000 Patients US
      • 500,000 Patients Europe
  • 20. Lipid Polymer Micelle (LPM TM ) Delivery System for Improving Oral Drug/Peptide Absorption BioTherapeutics – Pipeline Expansion
  • 21. Lipid Polymer Micelles (LPM  )
    • Drug delivery system potentially applicable to a wide variety of poorly absorbable water-soluble drugs/peptides (500 - 5,000 daltons)
    • Stable reverse micelle composed of GRAS lipids and polymers
    • Transiently opens tight junctions in intestinal mucosa
    • High relative bioavailability
    • First candidate selected:
      • GnRH analog: Leuprolide (1200 daltons)
    • Pre-clinical proof-of-concept: Reproducible and consistent absorption in rats and dogs
      • LPM  achieved 20-40% bioavailability with leuprolide versus <5% with straight oral delivery
    • Next steps: Complete preclinical work and conduct Phase 1 human PK study to confirm preclinical findings
  • 22.
    • Paracellular Transport
    • Peptides and small proteins do not normally penetrate intestinal epithelial barriers
    • Tight junctions (TJ) are inter-cellular contact points that regulate intestinal absorption of solutes and nutrients
    • TJ consist of a belt of protein strands and lipids that surround the lateral membrane of epithelial cells and seals the outermost end of the intercellular space.
    • In the presence of LPM (polymer stabilized reverse micelles) tight junctions transiently open and allow the penetration of small proteins and peptides with a hydrodynamic radius in the range of small molecules, proteins and cells across these barriers in the range of 8–20 degrees.
    Reverse Micelles and Paracellular Transport Apical surface Intestinal epithelial cells Basal surface surface Paracellular transport of peptides Tight Junction
  • 23. Leuprolide Market
    • Annual US sales of Leuprolide approx. $1.7 billion
    • Other approved/marketed LHRH agonists :
      • Lupron (Lupron Depot, TAP)
      • Goserelin (Zoladex  , Astra/Zeneca), 1-3 month s.c. implant
      • Nafarelin (Synarel  , Pharmacia), intranasal solution
      • Histrelin (Supprelin  , Roberts), s.c. daily injection
    • Indications :
      • Prostate cancer
      • Endometriosis and puberty in children
    • Other depot formulations available (1,3,4 & 6 months)
    • No oral product on the market
  • 24. RiVax TM Ricin Toxin Vaccine (Phase 1) BioDefense – Lead Program Castor Bean
  • 25. Ricin - Evolution Of A Threat from “Ricin: Technical Background and potential Role in Terrorism,” Dana Shea and Frank Gottron, CRS Report for Congress; February 4, 2004 1940s – weaponized by UK military “ Compound W” 1978 – Umbrella Assassination in London of Bulgarian dissident Georgi Markov 1991 – Minnesota Patriots Council found with 0.7 g ricin; arrested and convicted 1995 – Thomas Lewis Lavy arrested for possession of 130 g ricin while crossing into Canada from Alaska 2002 – Kenneth Olson arrested and sentenced 13 years for producing ricin 2003 – Secret Service intercepted a letter contaminated with ricin addressed to the White House 2004 – US Senate closed - Ricin detected in mail sent to Senate Majority Leader Bill Frist 2004 - Reports of ricin in Afghanistan/Al Qaeda 2005 - Reports of ricin possession in Florida and Arizona 2007- Ricin highlighted in FBI terrorism report as top bioterror threat along with Anthrax 2008 - Roger Bergendorff put himself in coma after ricin exposure in Las Vegas Motel
  • 26. 2007 FBI Terrorism Report “ Ricin and the bacterial agent anthrax are emerging as the most prevalent agents involved in WMD investigations” FBI Terrorism Report , November 2007
  • 27. RiVax ™
    • Profile:
    • Soligenix is world leader in ricin toxin vaccine research
    • Vaccine is derived from a non toxic recombinant ricin A chain
    • Retains immunogenic properties: induces production of serum
    • antibodies capable of neutralizing native ricin toxin
    • Funding:
    • Recent $9.4 million NIH grant to develop heat stable vaccines
    • Received >$20 million NIAID grant funding to date
    • Partner UT Southwestern received $3.6 million grant funding
    • Status:
    • Phase 1 clinical trial complete: Demonstrated safety and
    • immunogenicity in humans
    • Non-human primate study initiated
    • Phase 1b clinical trial underway at UTSW: adjuvant formulation
    • Scale-up GMP process optimization and development completed
    • Next Milestones:
    • Complete Phase 1B clinical trial
    • Manufacture large scale commercial grade batches for future clinical trials and potential procurement contracts
    • Continue development under new $9.4 million heat stabilization grant
    Castor beans
  • 28. Milestone Events - Past & Future
  • 29. Investment Highlights
      • Low current valuation of approximately $60 million
        • Potential for exponential growth with the achievement of a single milestone – A positive confirmatory Phase 3 clinical trial of orBec ®
        • Relatively low risk and late stage drug development proposition due to existing orBec ® Phase 3 data
      • Four active clinical trials
      • Clean capital structure with no debt or preferred stock outstanding
      • FDA SPA and EMEA agreement on Phase 3 protocol supports regulatory path to approval in US and EU
      • 35% royalty generating North American partnership with Sigma-Tau on orBec ®
      • orBec ® addresses a potential $400 million global GVHD market, where there is an unmet medical need with poor therapeutic options
      • Oral BDP application in other GI areas - Radiation Enteritis and Crohn’s Disease; markets in excess of $500 million worldwide 
      • LPM™ - novel oral delivery system for drugs such as leuprolide
      • Diversified portfolio: BioTherapeutics and BioDefense
        • BioDefense - Grant funded and revenue generating – New $9.4 million NIH grant
          • Soligenix is world leader in ricin toxin vaccine research
  • 30. THANK YOU www.soligenix.com BioTherapeutics | BioDefense