Follow the Money: Acceleron Pharma, Inc.

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Follow the Money highlights a newly funded bio/pharmaceutical company. This includes important insights into the featured company’s product pipeline, manufacturing and business relationships, and likely sourcing opportunities.

Acceleron Pharma is a U.S. public biopharmaceutical company that discovers and develops protein therapies for the treatment of musculoskeletal, metabolic and cancer-related diseases. As reported in the January 7, 2016 issue of the PharmSource Lead Sheet, they recently planned to raise $150 million in the completion of a secondary public offering.

Corporate Highlights

  • Acceleron was incorporated in Delaware in June 2003 as Phoenix Pharma, Inc., and later became Acceleron Pharma, Inc. in February 2004.
  • Corporate Headquarters: Cambridge, MA – approximately 94,500 square feet of office and laboratory space.
  • As of March 2, 2015, they had 83 employees, of whom 65 were engaged in R&D or manufacturing.

Sourcing Opportunities

  • Acceleron Pharma plans to use the proceeds for general expenses and to support R&D efforts and clinical trials of its pipeline, including:
    • Dalantercept (ACE-041) – complete a Phase I/II study:
      • For the treatment of hepatocellular carcinoma
      • Dosage Form: Parenteral
      • Nature of API: Biologic – Protein – Recombinant
      • Therapeutic Area: Oncology – Other
    • ACE-083 – complete a Phase II study:
      • For the treatment of facioscapulohumeral muscular dystrophy
      • Dosage Form: Parenteral
      • Nature of API: Biologic – Protein – Recombinant
      • Therapeutic Area: Muscular/Musculoskeletal
  • The company contracts with contract research organizations (CROs) to conduct clinical trials.
  • Acceleron Pharma owns and operates a manufacturing facility that develops all required materials for preclinical, Phase I, and Phase II studies, however they rely on third party contract manufacturers for all materials for Phase III studies.
  • Acceleron Pharma has no sales, marketing or distribution experience.

Business Relationships

  • On February 20, 2008, Acceleron and Celgene entered into a collaboration agreement for the development and commercialization of Sotatercept (ACE-011). Under the terms of the agreement, Acceleron received an upfront payment of $45 million and $5.0 million of equity with the eligibility of receiving up to $360 million based on future development, regulatory and sales milestones.
  • On September 8, 2009, Acceleron and Shire plc entered into a development and commercialization agreement for ACE-031. Under the terms of the agreement, Acceleron received an upfront payment of $45 million and an additional $9.0 million in R&D payments. In April 2013, Acceleron and Shire determined not to further the advancement of ACE-031, ending the collaboration agreement on June 30, 2013 with all rights retained by Acceleron.
  • On December 3, 2009, Acceleron and Alkermes entered into a collaboration and license agreement for the Medifusion technology, designed to extend the circulating half-life of proteins and peptides. Under the terms of the agreement, Alkermes gained the worldwide rights to the Medifusion technology in return for an upfront payment and equity investment in Acceleron, as well as future development, regulatory and sales milestones and royalties on product sales. Acceleron retained all rights to the technology for products derived from the TGF-beta superfamily. Financial terms were undisclosed.
  • On August 2, 2011, Acceleron and Celgene entered into a second collaboration agreement for the development and commercialization of Luspatercept (ACE-001). Acceleron received an upfront payment of $25 million and as of December 31, 2014, a total of $44.4 million has been received in R&D funding and milestone payments.1

Pipeline

Product Candidate Indication Dosage Form Status Next Anticipated Step
ACE-083 Facioscapulohumeral
Muscular Dystrophy
Parenteral Phase I Initiate Phase II trial
In mid-2016
Dalantercept
(ACE-041)
Hepatocellular
Carcinoma
Parenteral Phase Ib TBA
Sotatercept/Luspatercept
(ACE-011)
Beta-Thalassemia Parenteral Phase II TBA
Sotatercept/Luspatercept
(ACE-011)
Myelodysplastic
Syndromes
Parenteral Phase II TBA
Sotatercept/Luspatercept
(ACE-011)
Chronic Kidney Disease Parenteral Phase II TBA
Dalantercept
(ACE-041)
Renal Cell Carcinoma Parenteral Phase II TBA

Finances

(In $ thousands) 2014 2013
Revenues
14,632
57,230
R&D Expenditures
50,897
36,051
General & Administrative Expenditures
14,199
14,227
Total Operating Expenses
70,096
50,278

1Acceleron Pharma, Inc., 3/2/15 10-K, SEC.gov

Contact Information

Acceleron Pharma, Inc. Key Officers
128 Sidney Street John Knopf, PhD, CEO
Cambridge, MA 02139 USA Matthew L. Sherman, MD, CMO
Phone: +1-617-649-9200 Ravi Kumar, PhD, CSO
Fax: +1-617-649-9988 James Desiderio, PhD, VP, Regulatory Affairs
Web: www.acceleronpharma.com Christopher Rovaldi, SVP, Program Management & Operations

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Scotty is a Research Analyst at PharmSource. He holds a BS degree in Biology from George Mason University and an MPH degree from Eastern Virginia Medical School. He’s experienced in data collection, analysis and management, and has done laboratory analysis and safety program development.

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