Stifel Nicolaus Healthcare Conference. September 2012

Transcription

1 Stifel Nicolaus Healthcare Conference September 212

2 Alnylam Forward Looking Statements This presentation contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of There are a number of important factors that could cause actual results to differ materially from the results anticipated by these forward-looking statements. These important factors include our ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of our drug candidates, obtain, maintain and protect intellectual property, enforce our patents and defend our patent portfolio, obtain regulatory approval for products, establish and maintain business alliances; our dependence on third parties for access to intellectual property; and the outcome of litigation, as well as those risks more fully discussed in our most recent quarterly report on Form 1-Q under the caption Risk Factors. If one or more of these factors materialize, or if any underlying assumptions prove incorrect, our actual results, performance or achievements may vary materially from any future results, performance or achievements expressed or implied by these forward-looking statements. All forwardlooking statements speak only as of the date of this presentation and, except as required by law, we undertake no obligation to update such statements. 2

3 RNAi Therapeutics Harness natural pathway» Catalytic mechanism» Mediated by small interfering RNAs or sirnas Treat disease with therapeutic gene silencing» Any gene in genome» Creates unique opportunities for innovative medicines RNA Interference (RNAi) A New Class of Innovative Medicines 3

4 % TTR mrna Silencing (Relative to Control) % TTR mrna Silencing (Relative to Control) Enables advancement of innovative medicines to patients RNAi Delivery to Liver Solved IV and SC Platforms Potent, rapid, and durable target gene silencing with proprietary lipid nanoparticle (LNP) technology and IV dosing Advancement of conjugate platform for clinical translation and SC dosing Control Control ALN-TTR2 (MC3-LNP) mg/kg ALN-TTRsc (GalNAc-Conjugate) mg/kg 4 Keystone: Adv in Biopharm., Jan 21; Oligo Ther Soc., Sep 211

5 % Serum TTR Knockdown Relative to Baseline at Nadir (Days 1-28) % Subjects Achieving LDL-c Target 1. Human Safety and PK >575 Subjects/patients enrolled overall Systemic delivery in human trials» ~1 Patients dosed» >325 Doses administered 2» >26 months of dosing RNAi therapeutics generally well tolerated Pharmacologically relevant human tissue levels achieved p<.1 RNAi Human Translation Achieved Multiple Alnylam Therapeutic Programs 2. Human Proof of Mechanism 3. Human Proof of Concept 4. Human Clinical Efficacy ALN-TTR Pre- Post- Positive, p<.1 5 location along VEGF transcript RNAi Cleavage ALN-PCS p=.3 ALN-VSP Control sirna *** *** *** Placebo ALN-TTR2 (mg/kg) 2 <.15 mg/kg.15 mg/kg (n=9) (n=15) ALN-PCS Dose Groups

6 RNAi Therapeutics Profile Single dose % Target Knockdown 2 4 Potential for Major Therapeutic Impact 6 8 Potent and rapid Durable and reversible (dosing q3-6 days) Day Post Dose 6 6

7 Alnylam 5x15 TM Focused Product Development Pipeline for Innovative Medicines RNAi for genetically defined disease as core strategy 5 Key products in clinical development through 215 Near term focus on TTR and AT3 programs Product characteristics Genetically defined target/disease Existing Alnylam delivery platform Early biomarkers in Phase I Clear and rapid development Significant commercial opportunity ALN-VSP ALN-RSV ALN-HTT Programs 1. ALN-TTR 2. ALN-AT3 3. ALN-PCS 4. ALN-HPN 5. ALN-TMP Partner Programs 7

8 Partner Programs Alnylam 5x15 Programs Pipeline Goals ALN-TTR2 IND/Phase I Clinical Data ALN-TTRsc H2 12 H1 13 ALN-AT3 213 Phase II Alnylam Goals Pivotal 213 ALN-PCS Partner ALN-HPN Partner ALN-TMP Partner ALN-RSV1 ALN-VSP /Partner Additional Corporate Goals 212 Additional partnerships Year-end Cash >$25M 8

9 Thank You 9