Acceleron Pharma, Inc. is an American clinical stage

biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, t ...

company based in

Cambridge Cambridge ( ) is a university city and the county town in Cambridgeshire, England. It is located on the River Cam approximately north of London. As of the 2021 United Kingdom census, the population of Cambridge was 145,700. Cambridge beca ...

, Massachusetts with a broad focus on developing medicines that regulate the

transforming growth factor beta Transforming growth factor beta (TGF-β) is a multifunctional cytokine belonging to the transforming growth factor superfamily that includes three different mammalian isoforms (TGF-β 1 to 3, HGNC symbols TGFB1, TGFB2, TGFB3) and many other ...

(TGF-β) superfamily of proteins, which play fundamental roles in the growth and repair of cells and tissues such as red blood cells, muscle, bone, and

blood vessel Blood vessels are the structures of the circulatory system that transport blood throughout the human body. These vessels transport blood cells, nutrients, and oxygen to the tissues of the body. They also take waste and carbon dioxide away from ...

Pipeline

Acceleron has four drugs in

clinical trial Clinical trials are prospective biomedical or behavioral research studies on human subject research, human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel v ...

s, and one in preclinical development. * Luspatercept (ACE-536) for anemia *

Sotatercept Sotatercept, sold under the brand name Winrevair is a medication used for the treatment of pulmonary arterial hypertension. It is an activin signaling inhibitor, based on the extracellular domain of the activin type 2 receptor expressed as a ...

(ACE-011) for kidney disease * Dalantercept (ACE-041) for kidney cancer * ACE-083 for muscular disorders

History

The company was formed in June 2003 in

Cambridge, Massachusetts Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. As part of the Greater Boston, Boston metropolitan area, the cities population of the 2020 United States Census, 2020 U.S. census was 118,403, making it the fourth most ...

as a Delaware corporation; the original name was Phoenix Pharma. The founders were scientists Jasbir Seehra,

Tom Maniatis Tom Maniatis (born May 8, 1943), is an American professor of molecular and cellular biology. He is a professor at Columbia University, and serves as the Scientific Director and CEO of the New York Genome Center. Education Maniatis received B. ...

Mark Ptashne Mark Ptashne (born June 5, 1940, in Chicago) is a molecular biologist. He is the Ludwig Chair of Molecular Biology at Memorial Sloan–Kettering Cancer Center in New York City. Ptashne grew up in Chicago. He earned his undergraduate degree at Re ...

, Wylie Vale, and scientific advisor

Joan Massague Joan may refer to: People and fictional characters *Joan (given name), including a list of women, men and fictional characters *:Joan of Arc, a French military heroine *Joan (surname) Weather events *Tropical Storm Joan (disambiguation), multiple ...

, and business people and investors John Knopf and

Christoph Westphal Christoph Westphal, M.D., Ph.D., is a biomedical entrepreneur. Background and training Westphal graduated from Columbia College of Columbia University, summa cum laude, in 1990 and finished the MD–PhD program at Harvard University in six y ...

of Polaris Venture Partners, who served as founding CEO. The company was founded to discover and develop drugs based on the scientific discoveries of the scientific founders in the field of

growth factors A growth factor is a naturally occurring substance capable of stimulating cell proliferation, wound healing, and occasionally cellular differentiation. Usually it is a secreted protein or a steroid hormone. Growth factors are important for re ...

and

transforming growth factor Transforming growth factor (, or TGF) is used to describe two classes of polypeptide growth factors, TGFα and TGFβ. The name "Transforming Growth Factor" is somewhat arbitrary, since the two classes of TGFs are not structurally or genetically r ...

s in the fields of

metabolic disorder A metabolic disorder is a disorder that negatively alters the body's processing and distribution of macronutrients, such as proteins, fats, and carbohydrates. Metabolic disorders can happen when abnormal chemical reactions in the body alter the ...

s like

obesity Obesity is a medical condition, sometimes considered a disease, in which excess body fat has accumulated to such an extent that it may negatively affect health. People are classified as obese when their body mass index (BMI)—a person's ...

diabetes Diabetes, also known as diabetes mellitus, is a group of metabolic disorders characterized by a high blood sugar level (hyperglycemia) over a prolonged period of time. Symptoms often include frequent urination, increased thirst and increased ...

, osteoporosis, and muscle-wasting conditions. The company began with a seed round from Polaris of $250,000 and then had a Series A

venture capital Venture capital (often abbreviated as VC) is a form of private equity financing that is provided by venture capital firms or funds to start-up company, startups, early-stage, and emerging companies that have been deemed to have high growth poten ...

investment of $25 million that it used to open its first laboratory in December 2003. Glenn Batchelder was appointed president and CEO in June 2004. It started its first clinical trial in June 2006; the product was ACE-011 (which eventually was named "sotatercept"), a protein therapeutic that was an activin type 2 receptor antagonist intended to treat bone loss. ACE-011 was a chimeric protein, created by fusing the binding portion of the activin type 2 receptors to part of an antibody; the resulting protein binds to

activin Activin and inhibin are two closely related protein complexes that have almost directly opposite biological effects. Identified in 1986, activin enhances FSH biosynthesis and secretion, and participates in the regulation of the menstrual c ...

and prevents it from acting. Belgische blauwe

Knopf took over as CEO in 2007. He became known for showing pictures of a

Belgian Blue The Belgian Blue (french: 'Blanc-Bleu Belge', nl, 'Belgisch Witblauw', both literally meaning "Belgian White-Blue") is a breed of beef cattle from Belgium. It may also be known as the , or nl, dikbil, label=none (literally "fat buttocks" in ...

cow to potential inventors, as a way of illustrating the company's products' potential to develop muscle. In 2008 Acceleron and Celgene started a partnership to jointly develop and market ACE-011 in which Celgene paid Acceleron $50 million up front and bought $5 million of Acceleron stock, and agreed to buy $7 million more in stock if Acceleron went public, and agreed to pay up to $510 million in milestones. In a separate deal done at the same time, Celgene acquired an option to license three products in Acceleron's pipeline directed to cancer and cancer-related bone loss. In 2011 Acceleron extended their partnership with Celgene to include ACE-536, a development candidate for anemia; Celgene paid $25 million upfront, with potential downstream payments of $217 million in milestones and royalties over 10%. The company held its

initial public offering An initial public offering (IPO) or stock launch is a public offering in which shares of a company are sold to institutional investors and usually also to retail (individual) investors. An IPO is typically underwritten by one or more investme ...

in September 2013. At that time, the company had three protein therapeutic candidates being studied in 12 Phase 2 clinical trials, including sotatercept and luspatercept (ACE-536) which promoted red blood cell production and were being tested as potential treatments for anemia in people with thalassemia and myelodysplastic syndromes (MDS); its other candidate was dalantercept (ACE-041), an

angiogenesis inhibitor An angiogenesis inhibitor is a substance that inhibits the growth of new blood vessels (angiogenesis). Some angiogenesis inhibitors are endogenous and a normal part of the body's control and others are obtained exogenously through pharmaceutical dru ...

as a potential cancer drug. In September 2016, Knopf retired, and the company hired Habib Dable as CEO; at that time, the company's lead product luspatercept was in Phase III testing for MDS and beta-thalassemia. Acceleron experienced a drop in its share prices in 2019, after announcing that it will discontinue the development of an experimental drug meant to treat the rare genetic disease called

facioscapulohumeral muscular dystrophy Facioscapulohumeral muscular dystrophy (FSHD) is a type of muscular dystrophy, a group of heritable diseases that cause degeneration of muscle and progressive weakness. Per the name, FSHD tends to sequentially weaken the muscles of the face, ...

. The termination of the drug's development has been partly due to shareholders' concerns regarding the costs associated with it. In September 2021, Merck & Co. announced it would acquire Acceleron for $11.5 billion, gaining control over Sotatercept, used in the treatment of pulmonary hypertension and

luspatercept-aamt Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Me ...

. The acquisition closed on November 19, 2021, making Acceleron a subsidiary of Merck. In October 2022, Merck & Co. announced the results from a Phase III trial evaluating sotatercept.

References

{{Merck&Co American companies established in 2003 Biotechnology companies established in 2003 Pharmaceutical companies established in 2003 Biopharmaceutical companies Biotechnology companies of the United States Companies based in Cambridge, Massachusetts Pharmaceutical companies of the United States Companies formerly listed on the Nasdaq 2003 establishments in Massachusetts Health care companies based in Massachusetts 2013 initial public offerings 2021 mergers and acquisitions Merck & Co. American corporate subsidiaries