Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — AVI Biopharma — Merger or Acquisition (March 2008)
9 slides from Sarepta Therapeutics's merger presentation, Mar 13, 2008
Slide 1: AVI plus Ercole Rationale and Strategy
1 AVI plus Ercole Rationale and Strategy March 13, 2008 Product Flow From Directed RNA Alternative Splicing
Slide 2: Safe Harbor Statement
2 Safe Harbor Statement Comments made by management during this presentation will include forward-looking statements within the meaning of Federal securities laws. These forward-looking statements involve material risks and uncertainties and include statements which may be preceded by the words “pot
Slide 3: AVI & Ercole – True Synergy
3 AVI & Ercole – True Synergy Complementary technologies and patents – Ercole • Fundamental expertise, patents and technology for directed alternative splicing • Patent applications on high-value targets – AVI • PMO chemistry well suited for directed alternative splicing (i.e., ESPRIT) • Dedicated i
Slide 4: Directing Outcome of Alternative Splicing
4 Directing Outcome of Alternative Splicing Mitigate impact of a mutation Direct RNA processing to a preferred splice variant Tip balance of RNA processing from promotion to inhibition; cell death, signal transduction Create novel protein in situ; for example endogenously solubilized transmembrane p
Slide 5: In Vivo Delivery and Localization
5 In Vivo Delivery and Localization Ercole EGFP mouse has proven to be key for delivery evaluation Has been used to match tissue localization and optimum chemistry AVI developed enhanced immune cell targeting – Penetration into T cells and dendritic cells in vivo Exploits advantages of the chemical
Slide 6: Range of New Discovery Targets Enabled by Combined IP Estate
6 Range of New Discovery Targets Enabled by Combined IP Estate RNA repair – Joint program in DMD already in clinic Soluble receptors – TNFR2 programs in rheumatoid arthritis already in progress in Ercole Ligand independent receptors – CTLA-4 program in type I diabetes in Discovery Research at AVI Tr
Slide 7: DMD Clinical Strategy
7 DMD Clinical Strategy Demonstrate expression, safety, duration and gain of function Systemic delivery, dose-response and safety; therapeutic window – Single exon intervention for a limited number of exons Spectrum of DMD treatment of the affected population will require a multi-exonic approach – D
Slide 8: TNF Receptor Superfamily
8 TNF Receptor Superfamily Approved TNFa Blockers Name Technology Enbrel® rDNA Humira®, Remicade® MAb Market size: $9.9B (>$4B for Enbrel alone) – Opportunity for a chemically synthesized TNFa blocker – Induction of soluble TNF receptor in situ – Well characterized therapeutic intervention points wi
Slide 9: AVI plus Ercole Product Flow From Directed RNA Alternative Splicing
9 AVI plus Ercole Product Flow From Directed RNA Alternative Splicing