Acceleron Pharma

[Michael Scally MD]

Acceleron Pharma
Acceleron Pharma

Since our initial financing in 2004, Acceleron’s goal has been to develop innovative therapies. Our company has built in-house drug development capabilities, allowing us to focus our creativity and innovation on the biology of the Growth and Differentiation Factor (GDF) protein family, while streamlining the development of novel therapies.

This strategy has already yielded a rich pipeline of compounds that have the potential to be therapeutic breakthroughs for a number of devastating diseases. Our approach has opened a completely new area of GDF-targeted therapies that can modulate the growth and repair of a variety of tissue systems, offering new hope and benefits to patients.

Focus on the GDF Family of Proteins

Acceleron is establishing itself as the premier company in GDF-targeted therapies. We have developed an unparalleled expertise in GDF biology, and our scientific advisory board consists of leaders and pioneers in the field. Our discoveries and technology have allowed us to unleash the therapeutic promise of this protein family, building a powerful and productive discovery platform.


ACE-031 (Soluble Activin Receptor Type IIB-IgG1) Increases Muscle Mass by Inhibiting Myostatin and Other Negative Regulators of Muscle: Non-Clinical and Clinical Studies. [See: OnLine First - Page 36 ]

[bigrobbie]

Impressive!

Thank you for posting...

[Michael Scally MD]

Koncarevic A, Kajimura S, Cornwall-Brady M, et al. A Novel Therapeutic Approach to Treating Obesity through Modulation of TGFβ Signaling. Endocrinology. A Novel Therapeutic Approach to Treating Obesity through Modulation of TGFβ Signaling

Obesity results from disproportionately high energy intake relative to energy expenditure. Many therapeutic strategies have focused on the intake side of the equation, including pharmaceutical targeting of appetite and digestion. An alternative approach is to increase energy expenditure through physical activity or adaptive thermogenesis. A pharmacological way to increase muscle mass and hence exercise capacity is through inhibition of the activin receptor type IIB (ActRIIB). Muscle mass and strength is regulated, at least in part, by growth factors that signal via ActRIIB. Administration of a soluble ActRIIB protein comprised of a form of the extracellular domain of ActRIIB fused to a human Fc (ActRIIB-Fc) results in a substantial muscle mass increase in normal mice. However, ActRIIB is also present on and mediates the action of growth factors in adipose tissue, although the function of this system is poorly understood. In the current study, we report the effect of ActRIIB-Fc to suppress diet-induced obesity and linked metabolic dysfunctions in mice fed a high-fat diet. ActRIIB-Fc induced a brown fat-like thermogenic gene program in epididymal white fat, as shown by robustly increased expression of the thermogenic genes uncoupling protein 1 and peroxisomal proliferator-activated receptor-γ coactivator 1α. Finally, we identified multiple ligands capable of reducing thermogenesis that represent likely target ligands for the ActRIIB-Fc effects on the white fat depots. These data demonstrate that novel therapeutic ActRIIB-Fc improves obesity and obesity-linked metabolic disease by both increasing skeletal muscle mass and by inducing a gene program of thermogenesis in the white adipose tissues.

[Michael Scally MD]

Attie KM, Borgstein NG, Yang Y, et al. A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers. Muscle Nerve. A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers - Attie - 2012 - Muscle & Nerve - Wiley Online Library

INTRODUCTION: ACE-031 is a soluble form of activin receptor type IIB (ActRIIB). ACE-031 promotes muscle growth by binding to myostatin and other negative regulators of muscle mass.

METHODS: This double-blind, placebo-controlled study evaluated the safety, pharmacokinetics, and pharmacodynamics of ACE-031 in 48 healthy, postmenopausal women randomized to receive 1 dose of ACE-031 (0.02-3 mg/kg SC) or placebo (3:1).

RESULTS: ACE-031 was generally well-tolerated. Adverse events included injection site erythema. Mean ACE-031 AUC(0-infinity) and C(max) increased linearly with dose; mean T((1/2)) was 10-15 days. Statistically significant increases in mean total body lean mass (3.3%; P = 0.03, by DXA) and thigh muscle volume (5.1%; P = 0.03, by MRI) were observed at day 29 in the 3 mg/kg group. Statistically significant changes in serum biomarkers suggest ACE-031 also improved bone and fat metabolism.

CONCLUSIONS: Single-dose ACE-031 treatment was generally well-tolerated and resulted in increases in muscle mass in healthy postmenopausal women.