BMS : An Anti-Myostatin Adnectin Targeting Duchenne Muscular Dystrophy
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1 BMS : An Anti-Myostatin Adnectin Targeting Duchenne Muscular Dystrophy Leslie Jacobsen, MD Medical Lead GS Tirucherai, M Ahlijanian, F Luo, C Bechtold and the BMS Anti-Myostatin Team PPMD Connect Conference June 26-29, 2016
2 Disclosure and Disclaimer q q q q Dr. Jacobsen is a full time employee of, manufacturer of BMS BMS is currently in development as a treatment for DMD;; it is not approved for sale in any country This presentation is intended for dissemination and discussion of scientific information only and is not intended as a recruitment tool Disclosure information for Session Chairs and Planning Committee members is included in the Disclosure Insert of the program guide. All conflicts of interest have been resolved in accordance with ACCME Standards for Commercial Support 2
3 BMS : an Anti-Myostatin Adnectin q BMS is a fully human anti-myostatin adnectin q Sub nm affinity for Myostatin, supporting subcutaneous dosing q Equipotent in rodent, non-human primate and human q Being developed as a treatment for Duchenne Muscular Dystrophy q Clinical trial in Duchenne is ongoing 3
4 Myostatin: A Negative Regulator of Muscle Growth q Myostatin (GDF-8) discovered as novel member of TGF-b superfamily q Removal of the myostatin gene in mice (knock-out): q 2-3x increase in body weight, wide spread increase in skeletal muscle q Decreased fat mass, increased bone density q No increase in cardiac muscle q Myostatin gene sequence and function is conserved across species;; knockout produces a similar phenotype in: q Mice, Cattle, Sheep, Zebrafish, Dog, & Human 4
5 Anti-myostatin antibody improves phenotype of mdx mouse Muscle Weight Muscle Strength Rotorod Performance Serum Creatine Kinase treated control Weekly subcutaneous treatment for 3 months with an anti-myostatin antibody increased muscle weight and strength, improved locomotor performance and decreased serum creatine kinase in mdx mice Bogdanovich, Nature,
6 Multiple doses of BMS increase muscle volume in Cynomolgus monkeys 6
7 BMS : Phase 1 Safety, PK, & Pharmacodynamic Efficacy in Healthy Adults
8 BMS : Phase 1 Safety q Single and multiple ascending dose panels in healthy adults have completed q Preliminary findings: q Single and multiple weekly subcutaneous doses of up to 180 mg BMS appear to be generally safe and well tolerated q Most common adverse effects were mild injection site erythema, rash and injection site reaction q Increases in thigh muscle volume (measured using MRI) were observed following multiple dosing
9 PK & Target Engagement of BMS (SAD) BMS concentration (PK) Free myostatin - Percent of baseline BMS has a Tmax of 3-5 days post SC administration. Exposure increases were generally dose proportional up to 90 mg and greater than dose proportional at 180 mg BMS binds to and reduces free myostatin in serum. Extent and duration of free myostatin suppression increases with dose 9
10 BMS increases thigh muscle volume in healthy volunteers Increases in muscle volume appear to correlate with extent of free myostatin suppression. > 95% > 95% > 95% 67% > 95% >95% 81% +19% 13% 5 weekly doses;; last dose day 28 Time and dose-dependent effects 2.7% Embedded values represent % reduction in free serum myostatin 10
11 Summary of Preliminary Phase I Findings q Phase I data suggest that BMS is generally safe and well tolerated in single and weekly multiple subcutaneous doses of up to 180 mg q Exposure increases of BMS were dose proportional up to 90 mg and greater than dose proportional above 90 mg q Extent and duration of free myostatin suppression increased with dose q Treatment with 5 weekly doses of 45 mg or more of BMS was associated with increases in thigh muscle volume in healthy adults 11
12 Study of BMS in Duchenne 12
13 Ongoing Study of BMS in Boys with Duchenne q Multi-site, randomized, placebo-controlled, double-blind, multiple ascending weekly subcutaneous dose study to assess safety and tolerability of BMS in ambulatory boys with Duchenne q Outpatient 24 week double blind phase (randomized to BMS or PBO), followed by 48 week open label phase q All subjects will receive active drug during the open label phase q Sites in US & Canada 13
14 Study Objectives u Primary: q Assess safety and tolerability of multiple SC doses of BMS in boys with Duchenne u Secondary: q Evaluate PK, immunogenicity, free myostatin and myostatin drug complex q MRI/MRS measure of thigh muscle fat/lipid fraction 14
15 Key Criteria Inclusion q Diagnosis of DMD, confirmed by genotyping q Ambulatory boys ages > 5 to 11 years q Corticosteroid use for the past 6 months q 4 stair climb < 8.0 seconds Exclusion q EF < 55% on echo q Cognitive impairment or behavioral issues that interfere with subject ability to complete study procedures q Treatment with Ataluren or any investigational drug within 3 months or 5 half lives prior to the start of study drug q Major surgery within 6 weeks of starting study drug 15
16 Dosing and Procedures q Weekly subcutaneous dosing q Procedures include: q Vital signs, physical exam, ECG, echocardiograms, blood draws q Imaging studies q Measures of function, including TFTs, 6MWT, NSAA 16
17 Further Information Visit Study Connect Patient and Care Giver Portal to learn more about the DMD clinical study DMDtrial.com or Visit 17
18 Thank You LESLIE JACOBSEN, MD & THE BMS ANTI-MYOSTATIN TEAM
Roche/Genentech Anti-Myostatin Adnectin RG6206 Development Program in Duchenne
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