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 List of cities in the United Kingdom, city and non-metropolitan district in the county of Cambridgeshire, England. It is the county town of Cambridgeshire and is located on the River Cam, north of London. As of the 2021 Unit ...

, 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 tubular structures of a circulatory system that transport blood throughout many Animal, animals’ bodies. Blood vessels transport blood cells, nutrients, and oxygen to most of the Tissue (biology), tissues of a Body (bi ...

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 (ACE-011) for pulmonary arterial hypertension * 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. It is a suburb in the Greater Boston metropolitan area, located directly across the Charles River from Boston. The city's population as of the 2020 United States census, ...

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.A ...

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 R ...

, Wylie Vale, and scientific advisor Joan Massague, and business people and investors John Knopf and Christoph Westphal 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 regu ...

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 ...

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, considered by multiple organizations to be a disease, in which excess Adipose tissue, body fat has accumulated to such an extent that it can potentially have negative effects on health. People are classifi ...

diabetes Diabetes mellitus, commonly known as diabetes, is a group of common endocrine diseases characterized by sustained high blood sugar levels. Diabetes is due to either the pancreas not producing enough of the hormone insulin, or the cells of th ...

osteoporosis Osteoporosis is a systemic skeletal disorder characterized by low bone mass, micro-architectural deterioration of bone tissue leading to more porous bone, and consequent increase in Bone fracture, fracture risk. It is the most common reason f ...

, 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 (VC) is a form of private equity financing provided by firms or funds to start-up company, startup, early-stage, and emerging companies, that have been deemed to have high growth potential or that have demonstrated high growth in ...

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 The activin type 2 receptors belong to a larger TGF-beta receptor family and modulate signals for transforming growth factor beta ligands. These receptors are involved in a host of physiology, physiological processes including, cell growth, growt ...

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 cow to potential inventors, as a way of illustrating the company's products' potential to develop muscle. In 2008 Acceleron and

Celgene Celgene Corporation, headquartered in Summit, New Jersey, was a pharmaceutical company that produced cancer and immunology drugs. Its primary products were Revlimid (lenalidomide), which is used in the treatment of multiple myeloma (63% of 2018 r ...

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 investm ...

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 ...

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 fac ...

. 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 Pulmonary hypertension (PH or PHTN) is a condition of increased blood pressure in the pulmonary artery, arteries of the lungs. Symptoms include dypsnea, shortness of breath, Syncope (medicine), fainting, tiredness, chest pain, pedal edema, swell ...

and luspatercept-aamt. 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 Pharmaceutical 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