Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — AVI BioPharma — Corporate Update (March 2008)
11 slides from Sarepta Therapeutics's corporate update, Mar 12, 2008
Slide 1: AVI BioPharma Business Strategy and Corporate Priorities
1 AVI BioPharma Business Strategy and Corporate Priorities March 12, 2008
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: The Opportunity
3 The Opportunity Compelling parallels between emerging therapeutic utility of monoclonal antibodies and potential for antisense oligomers Insights from beyond the genome and RNAi highlight the central importance of alternative splicing; 26,000 genes vs. 150,000 proteins Indications of pending techn
Slide 4: Company Goals
4 Company Goals Advance AVI’s clinical development programs – Evidenced by successful program partnering and commercialization Adapt infrastructure to support flow of product candidates – Augment Biological Research capability – Strengthen Exploratory Development in Product Development flow Review r
Slide 5: Alternative Splicing
5 pre-mRNA mRNA protein Alternative Splicing Translation 70% genes alternatively spliced 26,000 genes; 150,000 proteins Several proteins from a single gene Related, frequently opposing, functions – Promote . inhibit cell death – Promote . inhibit signal transduction Key for Control of Gene Expressio
Slide 6: Cook Partnership
6 Cook Partnership Cook continues to pursue applications with AVI-5126 (anti-c-myc PPMO product) for restenosis, including catheter delivery kit and coated stent AVI is supporting Cook with product and analytics Cook is performing required studies to support its regulatory applications Cook may purs
Slide 7: Business Development
7 Business Development Advance AVI’s programs to Phase II for partnering and commercialization Increase access to external funding for DMD program R&D partnership with pharma or large biotech Consider additional government funding opportunities
Slide 8: AVI-5126 CABG Program Status
8 AVI-5126 CABG Program Status Efficacy measure is to demonstrate that AVI-5126 exposure of saphenous vein donor graft prior to CABG will reduce subsequent graft failure by 50% compared to placebo in a double-blinded, randomized, 600-subject clinical study Per protocol, review of clinical data requi
Slide 9: AVI-4658 DMD Program Status
9 AVI-4658 DMD Program Status 2008 2009 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q AVI AVI-4658 IM 4658 IM AVI AVI-4658 IV 4658 IV UK – 4Q07 MHRA indicated that existing data package should support proposed IV study – 1Q08 CTA submitted for a systemic (IV) dose – ranging safety and de novo dystrophin production assess
Slide 10: Government Programs
10 Government Programs AVI programs are funded by the DoD, as well as the Defense Threat Reduction Agency’s Transformational Medical Technologies Initiative Initial efforts are targeting Category A and B Bioterror Threats, with work in hemorrhagic fever viruses, pathogens and alternative approaches
Slide 11: 2008 Clinical/Regulatory Milestones
11 2008 Clinical/Regulatory Milestones 1Q AVI-6003 (Marburg Musoke) – pre-IND filed – AVI-4658 IV(DMD) – CTA to be filed for a systemic study – AVI-6002 (Ebola Zaire) – pre-IND to be filed 2Q – PMO-based exon 50 product (DMD) – pre-IND to be filed 3Q – AVI-4658 IV(DMD) – anticipate dosing of first p