XLRN Acceleron Pharma Inc.

100.68
-1.6  -2%
Previous Close 102.28
Open 102.83
52 Week Low 37.6
52 Week High 110.49
Market Cap $5,419,063,245
Shares 53,824,625
Float 38,856,277
Enterprise Value $5,115,660,645
Volume 355,863
Av. Daily Volume 704,334
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Upcoming Catalysts

Drug Stage Catalyst Date
Sotatercept - SPECTRA
Pulmonary arterial hypertension (PAH)
Phase 2
Phase 2
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Luspatercept - BEYOND
Non-transfusion-dependent beta-thalassemia
Phase 2
Phase 2
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Luspatercept - COMMANDS
First-line, lower-risk Myelodysplastic syndromes (MDS)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
Sotatercept - PULSAR
Pulmonary arterial hypertension (PAH)
Phase 2
Phase 2
Phase 2 trial met primary endpoint - January 27, 2020. Data presented at American Thoracic Society (ATS) June 24, 2020 noted PVR reductions of 21-34% compared with 2% for placebo.
Luspatercept - INDEPENDENCE
Myelofibrosis
Phase 3
Phase 3
Phase 3 trial to commence by end of 2020.
Luspatercept
Myelodysplastic syndromes (MDS) cancer
Approved
Approved
FDA Approval announced April 3, 2020.
ACE-083
Charcot-Marie-Tooth disease (CMT)
Phase 2
Phase 2
Phase 2 data did not meet primary endpoint - March 9, 2020.
Luspatercept
Beta-thalassemia
Approved
Approved
FDA Approval announced November 8, 2019.
ACE-083
Facioscapulohumeral muscular dystrophy
Phase 2
Phase 2
Phase 2 data did not achieve statistically significant improvements in functional endpoints relative to placebo - September 16, 2019.
ACE-2494
Healthy volunteers
Phase 1
Phase 1
Announced April 4, 2019 intention to discontinue development.
Dalantercept - DART Study
Renal Cell Carcinoma (RCC) - cancer
Phase 2
Phase 2
Phase 2 data released June 12, 2017 - primary endpoint not met.

Latest News

  1. Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it has issued an aggregate of 5,594,593 shares of common stock at a price of $92.50 per share at the closing of the Company's previously-announced public offering. The aggregate net proceeds to the Company of the public offering, including pursuant to the full exercise of the underwriters' option to purchase additional shares and after deducting underwriting discounts and commissions and estimated offering expenses, were approximately $492.5 million.

    J.P. Morgan, SVB Leerink and Cowen acted as joint book-runners for the offering…

    Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it has issued an aggregate of 5,594,593 shares of common stock at a price of $92.50 per share at the closing of the Company's previously-announced public offering. The aggregate net proceeds to the Company of the public offering, including pursuant to the full exercise of the underwriters' option to purchase additional shares and after deducting underwriting discounts and commissions and estimated offering expenses, were approximately $492.5 million.

    J.P. Morgan, SVB Leerink and Cowen acted as joint book-runners for the offering. Barclays, Credit Suisse, and Piper Sandler & Co. also acted as joint book-running managers. H.C. Wainwright & Co. acted as lead manager.

    The shares were offered by Acceleron pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on June 30, 2020. The final prospectus supplement relating to the offering was filed with the SEC on July 2, 2020. Copies of the final prospectus supplement and the accompanying prospectus can be obtained at the SEC's website http://www.sec.gov or from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204; or by email at ; SVB Leerink LLC, by mail at One Federal Street, 37th Floor, Boston, MA 02110, Attention: Syndicate Department, by telephone at (800) 808-7525, ext. 6218, or by email at ; and Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in the offering, nor shall there be any sale of these securities in any jurisdiction in which an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.

    Cautionary Note on Forward-Looking Statements

    Certain statements contained in this press release are forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those in the forward-looking statements, including statements about Acceleron's strategy, future plans and prospects. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, risks and uncertainties associated with the consummation of the proposed offering, changes in general economic or regulatory conditions, and the clinical development and commercialization of Acceleron's compounds. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

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  2. Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it has priced an underwritten public offering of 4,864,864 shares of common stock at a price to the public of $92.50 per share for gross proceeds of $450.0 million. In connection with this offering, Acceleron has granted the underwriters a 30-day option to purchase up to an additional 729,729 shares of common stock.

    J.P. Morgan, SVB Leerink and Cowen are acting as joint book-runners for the offering. Barclays, Credit Suisse, and Piper Sandler & Co. are also acting as joint book-running managers. H.C. Wainwright & Co. is acting…

    Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it has priced an underwritten public offering of 4,864,864 shares of common stock at a price to the public of $92.50 per share for gross proceeds of $450.0 million. In connection with this offering, Acceleron has granted the underwriters a 30-day option to purchase up to an additional 729,729 shares of common stock.

    J.P. Morgan, SVB Leerink and Cowen are acting as joint book-runners for the offering. Barclays, Credit Suisse, and Piper Sandler & Co. are also acting as joint book-running managers. H.C. Wainwright & Co. is acting as lead manager. The offering is expected to close on or about July 6, 2020, subject to the satisfaction of customary closing conditions.

    Acceleron expects to receive net proceeds from the offering after deducting underwriting discounts and commissions and estimated offering expenses payable by the company of approximately $428.2 million. Acceleron intends to use the net proceeds from the offering to conduct clinical trials and associated activities in connection with its therapeutic candidates in its pulmonary programs; to prepare for the potential launch and commercialization of sotatercept; and the remainder for general corporate purposes, including potential future development programs, capital expenditures and working capital, as well as potential acquisitions of rights to additional programs from third parties.

    The shares are being offered by Acceleron pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on June 30, 2020. The final prospectus supplement relating to the offering will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus can be obtained at the SEC's website http://www.sec.gov or from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204; or by email at ; SVB Leerink LLC, by mail at One Federal Street, 37th Floor, Boston, MA 02110, Attention: Syndicate Department, by telephone at (800) 808-7525, ext. 6218, or by email at ; and Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in the offering, nor shall there be any sale of these securities in any jurisdiction in which an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.

    Cautionary Note on Forward-Looking Statements

    This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the use of proceeds from the sale of common stock, the expected closing of the offering, the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, and the potential of REBLOZYL® (luspatercept-aamt) as a therapeutic drug. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

    Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, risks and uncertainties associated with the consummation of the proposed offering, changes in general economic or regulatory conditions, and the clinical development and commercialization of Acceleron's compounds. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

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  3. Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it intends to offer and sell, subject to market and other conditions, $400 million of its common stock in an underwritten public offering. As part of this offering, Acceleron intends to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares of common stock offered in the public offering. All of the shares in the proposed offering are to be sold by Acceleron.

    J.P. Morgan, SVB Leerink and Cowen are acting as joint book-running managers for the offering. Barclays, Credit Suisse…

    Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery, development and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that it intends to offer and sell, subject to market and other conditions, $400 million of its common stock in an underwritten public offering. As part of this offering, Acceleron intends to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares of common stock offered in the public offering. All of the shares in the proposed offering are to be sold by Acceleron.

    J.P. Morgan, SVB Leerink and Cowen are acting as joint book-running managers for the offering. Barclays, Credit Suisse, and Piper Sandler & Co. are also acting as joint book-running managers.

    Acceleron intends to use the net proceeds from the offering to conduct clinical trials and associated activities in connection with its therapeutic candidates in its pulmonary programs; to prepare for the potential launch and commercialization of sotatercept; and the remainder for general corporate purposes, including potential future development programs, capital expenditures and working capital, as well as potential acquisitions of rights to additional programs from third parties.

    The securities described above are being offered by Acceleron pursuant to its registration statement on Form S-3 filed with the Securities and Exchange Commission (the "SEC") on September 19, 2017, which became effective automatically when it was filed. The offering will be made only by means of a preliminary prospectus supplement and accompanying prospectus, copies of which may be obtained, when available, from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204; or by email at ; SVB Leerink LLC, by mail at One Federal Street, 37th Floor, Boston, MA 02110, Attention: Syndicate Department, by telephone at (800) 808-7525, ext. 6218, or by email at ; and Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926.

    The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in the offering, nor shall there be any sale of these securities in any jurisdiction in which an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension.

    Cautionary Note on Forward-Looking Statements

    This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the use of proceeds from the sale of common stock and the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, and the potential of Reblozyl® (luspatercept-aamt) as a therapeutic drug. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

    Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, risks and uncertainties associated with the consummation of the proposed offering, changes in general economic or regulatory conditions, and the clinical development and commercialization of Acceleron's compounds. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

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  4. Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions

    Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy

    Bristol Myers Squibb (NYSE:BMY) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of:

    • Adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy.
    • Adult patients with transfusion-dependent anemia associated…

    Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions

    Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy

    Bristol Myers Squibb (NYSE:BMY) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of:

    • Adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy.
    • Adult patients with transfusion-dependent anemia associated with beta thalassemia.

    "Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients' quality of life," said Uwe Platzbecker, M.D., lead investigator of the MEDALIST study, Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital. "Today's approval of Reblozyl provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence."

    "While beta thalassemia remains an orphan disease, the lifelong blood transfusions often needed by patients can have a significant impact on the limited blood supply in their communities, and there are few treatment alternatives," said Maria Domenica Cappellini, M.D., lead investigator of the BELIEVE study, Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda. "The European Commission's approval of Reblozyl provides eligible adult patients with beta thalassemia a new, much needed treatment option for their anemia, and with it, the possibility of becoming less dependent on red blood cell transfusions."

    Reblozyl is the first and only erythroid maturation agent approved in the European Union, representing a new class of therapy for eligible patients. This approval is based on data from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

    "Across the EU, 25 million blood transfusions occur every year, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia," said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. "Reblozyl has the potential to address the ineffective erythropoiesis associated with MDS and beta thalassemia, decrease patients' dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients. Alongside our partners at Acceleron, we recognize the continuing need in disease-related anemias and are committed to working collaboratively with European health authorities to make Reblozyl available to these patients as quickly as possible."

    About MEDALIST

    MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of Reblozyl plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) MDS. All patients were RBC transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA naïve and unlikely to respond due to endogenous serum erythropoietin levels of ≥ 200 U/L, and had no prior treatment with disease modifying agents.

    The trial showed a statistically significant improvement in RBC transfusion burden with Reblozyl, the study's primary endpoint, with 37.9% of patients treated with Reblozyl achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared to 13.2% of patients on placebo. The trial also met the secondary endpoint of transfusion independence for at least 12 weeks within the first 24 and 48 weeks of the study, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo.

    The majority of treatment-emergent adverse events (TEAEs) were Grade 1-2. Grade 3 or 4 TEAEs were reported in 42.5% of patients who received Reblozyl and 44.7% of patients who received placebo. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 4.5% of patients who received Reblozyl. The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis and urinary tract infection.

    Results of the MEDALIST trial were first presented during the Plenary Session of the American Society of Hematology (ASH) Annual Meeting in December 2018 (ASH Abstract #001) and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.

    About MDS

    MDS are a group of hematologic malignancies characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections, and can progress to Acute Myeloid Leukemia (AML). People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of transfusion reactions, infections and iron overload. There are approximately 50,000 patients with MDS in the EU5 countries (France, Germany, Italy, Spain and the United Kingdom).

    About BELIEVE

    BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing Reblozyl plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia.

    The trial showed a statistically significant improvement in RBC transfusion burden during weeks 13 to 24 compared to the baseline 12-week interval prior to randomization (21.4% Reblozyl versus 4.5% placebo), meeting the study's primary endpoint. The trial also met the secondary endpoint of transfusion burden reduction of at least 33% (with a reduction of at least two units) during weeks 37 to 48, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo. The trial also met an exploratory endpoint, with 70.5% of patients treated with Reblozyl achieving at least a 33% reduction in RBC transfusion burden of at least two units for any 12 consecutive weeks compared to the 12-week interval prior to treatment, compared to 29.5% of patients on placebo.

    The majority of TEAEs were Grade 1-2. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 5.4% of patients who received Reblozyl. The most common adverse reactions (>10%) were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness.

    Results of the BELIEVE trial were first presented at the ASH Annual Meeting in December 2018 and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.

    About Beta Thalassemia

    Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemia—a condition that can be debilitating and can lead to other complications for patients—as well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

    About Reblozyl®

    Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

    • anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and
    • anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

    Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

    U.S. Important Safety Information

    WARNINGS AND PRECAUTIONS

    Thrombosis/Thromboembolism

    In adult patients with beta thalassemia, thromboembolic events (TEE) were reported in 8/223 (3.6%) REBLOZYL-treated patients. TEEs included deep vein thrombosis, pulmonary embolus, portal vein thrombosis, and ischemic stroke. Patients with known risk factors for thromboembolism (splenectomy or concomitant use of hormone replacement therapy) may be at further increased risk of thromboembolic conditions. Consider thromboprophylaxis in patients at increased risk of TEE. Monitor patients for signs and symptoms of thromboembolic events and institute treatment promptly.

    Hypertension

    Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated patients. Across clinical studies, the incidence of Grade 3 to 4 hypertension ranged from 1.8% to 8.6%. In patients with beta thalassemia with normal baseline blood pressure, 13 (6.2%) patients developed systolic blood pressure (SBP) ≥130 mm Hg and 33 (16.6%) patients developed diastolic blood pressure (DBP) ≥80 mm Hg. In adult patients with MDS with normal baseline blood pressure, 26 (29.9%) patients developed SBP ≥130 mm Hg and 23 (16.4%) patients developed DBP ≥80 mm Hg. Monitor blood pressure prior to each administration. Manage new or exacerbations of preexisting hypertension using anti-hypertensive agents.

    Embryo-Fetal Toxicity

    REBLOZYL may cause fetal harm when administered to a pregnant woman. REBLOZYL caused increased post-implantation loss, decreased litter size, and an increased incidence of skeletal variations in pregnant rat and rabbit studies. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 3 months after the final dose.

    ADVERSE REACTIONS

    Beta Thalassemia

    • Serious adverse reactions occurred in 3.6% of patients on REBLOZYL. Serious adverse reactions occurring in 1% of patients included cerebrovascular accident and deep vein thrombosis. A fatal adverse reaction occurred in 1 patient treated with REBLOZYL who died due to an unconfirmed case of acute myeloid leukemia (AML)
    • Most common adverse reactions (at least 10% for REBLOZYL and 1% more than placebo) were headache (26% vs 24%), bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and dizziness (11% vs 5%)

    Myelodysplastic Syndromes

    • Grade >3 (≥2%) adverse reactions included fatigue, hypertension, syncope and musculoskeletal pain. A fatal adverse reaction occurred in 5 (2.1%) patients
    • The most common (≥10%) adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis, and urinary tract infection

    LACTATION

    It is not known whether REBLOZYL is excreted into human milk or absorbed systemically after ingestion by a nursing infant. REBLOZYL was detected in milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because many drugs are excreted in human milk, and because of the unknown effects of REBLOZYL in infants, a decision should be made whether to discontinue nursing or to discontinue treatment. Because of the potential for serious adverse reactions in the breastfed child, breastfeeding is not recommended during treatment and for 3 months after the last dose.

    Please see full Prescribing Information and Summary of Product Characteristics for REBLOZYL

    Bristol Myers Squibb: Advancing Cancer Research

    At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients' quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

    Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

    About Bristol Myers Squibb

    Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

    Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of chronic anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.

    For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

    Bristol Myers Squibb Cautionary Statement Regarding Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that the outcome of pricing and reimbursement negotiations in individual countries in Europe may delay or limit the commercial potential of Reblozyl for the indications described in this release, and whether such product candidate for such additional indications described in this release will be commercially successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb's business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2019, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

    Acceleron Cautionary Statement Regarding Forward-Looking Statements

    This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, the expected timing for reporting of data from ongoing clinical trials, and the potential of Reblozyl® (luspatercept-aamt) as a therapeutic drug. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

    Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that the results of any clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds will take longer and/or cost more than planned, that Acceleron or its collaboration partner, Bristol-Myers Squibb Company will be unable to successfully complete the clinical development of Acceleron's compounds, that Acceleron or Bristol Myers Squibb may be delayed in initiating, enrolling or completing any clinical trials, and that Acceleron's compounds will not receive regulatory approval or become commercially successful products. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future.

    The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

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  5. - Data presented during ‘Breaking News' Session of the American Thoracic Society 2020 Virtual Conference (ATS 2020 Virtual) show the PULSAR trial achieved its primary endpoint: a statistically significant mean reduction in pulmonary vascular resistance (PVR) -

    - Patients on stable background therapy who were treated with 0.3 mg/kg or 0.7 mg/kg of sotatercept experienced mean PVR reductions of approximately 21% and 34%, respectively -

    - The trial also achieved a statistically significant all-dose mean improvement from baseline of 54 meters in the key secondary endpoint of six-minute walk distance (6MWD) and a placebo corrected improvement of 25 meters (all doses combined) -

    - Sotatercept was generally well tolerated; adverse events were

    - Data presented during ‘Breaking News' Session of the American Thoracic Society 2020 Virtual Conference (ATS 2020 Virtual) show the PULSAR trial achieved its primary endpoint: a statistically significant mean reduction in pulmonary vascular resistance (PVR) -

    - Patients on stable background therapy who were treated with 0.3 mg/kg or 0.7 mg/kg of sotatercept experienced mean PVR reductions of approximately 21% and 34%, respectively -

    - The trial also achieved a statistically significant all-dose mean improvement from baseline of 54 meters in the key secondary endpoint of six-minute walk distance (6MWD) and a placebo corrected improvement of 25 meters (all doses combined) -

    - Sotatercept was generally well tolerated; adverse events were consistent with previously published data on sotatercept in clinical trials in other patient populations -

    - Company-hosted investor and analyst conference call and webcast with guest PAH key opinion leaders to be held today, Wednesday, June 24th at 4:30 p.m. EDT -

    Acceleron Pharma Inc. (NASDAQ:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today presented topline results of the PULSAR Phase 2 trial of sotatercept in patients with pulmonary arterial hypertension (PAH).

    During the "Breaking News: Clinical Trials in Pulmonary Medicine" session of ATS 2020 Virtual, study investigators reported that patients on stable background PAH-specific therapies treated with sotatercept experienced a statistically significant reduction in pulmonary vascular resistance (PVR), the trial's primary endpoint, at week 24 versus placebo.

    "We're thrilled to report the impressive magnitude of the positive effects that sotatercept, in combination with current therapies, was able to achieve in patients with PAH," said Habib Dable, President and Chief Executive Officer of Acceleron. "Although the introduction over the past two decades of more than a dozen treatments for PAH has driven the development of today's combination-therapy strategies, the substantial morbidity still associated with PAH clearly signals the need for a new approach. As we work now with health authorities to move sotatercept into Phase 3 testing, we are increasingly encouraged that we will be able to deliver a truly innovative therapy to patients with this debilitating disease."

    In this Phase 2 double-blind, placebo-controlled study, 106 patients were randomized to receive placebo, 0.3 mg/kg of sotatercept, or 0.7 mg/kg of sotatercept subcutaneously every 21 days in combination with stable background PAH-specific therapies, including mono, double, and triple therapy over a 24-week treatment period. Of the 106 patients participating in the trial, 35% were receiving double background PAH-specific therapies and 56% were receiving triple background PAH-specific therapies. The trial achieved its primary endpoint, key secondary endpoint, and showed concordance of results across multiple additional endpoints and regardless of baseline characteristics.

    Primary Endpoint:

    Treatment*

    % Reduction in PVR

    P-Value

    Sotatercept 0.3 mg/kg (n=32)

    20.5%

    0.0027

    Sotatercept 0.7 mg/kg (n=42)

    33.9%

    <0.0001

    Placebo (n=32)

    2.1%

     

    *All cohorts include stable background PAH-specific therapies

    The trial also achieved the protocol-defined improvement in the key secondary endpoint of 6MWD at 24 weeks. Both sotatercept dose groups achieved at least a 50-meter (LS mean) increase from baseline, as demonstrated in the 0.3 mg/kg group (58 meters) and the 0.7 mg/kg group (50 meters), allowing for a pre-specified pooled analysis. Overall, treatment with sotatercept (pooled analysis) achieved a 54-meter (LS mean) change from baseline and a placebo-corrected (LS mean) difference of 25 meters (nominal p=0.03).

    "These results, seen on top of existing therapies in heavily pretreated patients, are consistent with sotatercept exerting its effects through a mechanism distinct from currently approved agents," said Dr. David Badesch, Professor of Medicine and Clinical Director of the Pulmonary Hypertension Center at the University of Colorado.

    Badesch, who presented the PULSAR trial data during today's ATS 2020 Virtual session, continued: "By selectively binding ligands of the TGF-beta superfamily, sotatercept is designed to rebalance key signaling pathways whose disruption has been shown to be important in PAH biology. If sotatercept's efficacy and safety are confirmed in the next phase of clinical development, I believe it has the potential to substantially alter the way we treat patients with PAH."

    Treatment with sotatercept also demonstrated improvement across multiple exploratory study endpoints at week 24, including a 51% reduction in amino-terminal brain natriuretic propeptide (NT-proBNP), and 20% reduction in mean pulmonary arterial pressure. In addition, 23% of subjects improved their World Health Organization (WHO) functional class.

    Sotatercept was generally well tolerated in the trial. Adverse events observed in the study were generally consistent with previously published data on sotatercept in clinical trials in other patient populations. Serious treatment-emergent adverse events (TEAEs) were reported in 6% (2/32) of patients receiving 0.3 mg/kg of sotatercept plus background therapy, 24% (10/42) of patients receiving 0.7 mg/kg of sotatercept plus background therapy, and in 9% (3/32) of patients receiving placebo plus background therapy.

    Hemoglobin increase was reported in one patient (3%) in the 0.3 mg/kg sotatercept dose group and in 6 patients (14%) in the 0.7 mg/kg sotatercept dose group. No patient in the placebo group experienced an increase in hemoglobin. Two patients (6%) in the 0.3 mg/kg sotatercept dose group and 5 patients (12%) in the 0.7 mg/kg sotatercept dose group experienced thrombocytopenia. TEAEs occurring in 10% or more of all patients in any arm were headache, diarrhea, peripheral edema, dizziness, fatigue, hypokalemia, and nausea.

    As of June 22, 2020, 94 of 97 patients who opted to participate in the 18-month extension period of the trial were still enrolled; 64 patients have now been treated with sotatercept for at least 12 months.

    Dr. Badesch's presentation from ATS 2020 Virtual is available in the Publications section under "Science & Pipeline" on the Company's website at acceleronpharma.com.

    Sotatercept is an investigational therapy that is not approved for any use in any country.

    Dr. Badesch is the principal investigator of the PULSAR trial and a paid consultant to Acceleron.

    About the PULSAR Trial

    The PULSAR Phase 2 trial is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of sotatercept in PAH patients. The primary endpoint of the trial is the change from baseline in pulmonary vascular resistance (PVR) over a 24-week treatment period. PVR, as measured by right heart catheterization, is the resistance that the heart must overcome to pump blood through the pulmonary circulatory system. The key secondary endpoint was six-minute walk distance (6MWD); a measure of functional capacity/endurance. Other exploratory analyses included change in amino-terminal brain natriuretic propeptide (NT-proBNP), a hormone secreted by cardiac muscle cells in response to stretching caused by increased blood volume in the heart; mean pulmonary arterial pressure, a hemodynamic measure of average pressure in the main pulmonary arteries, which is elevated in PAH patients; and WHO functional class. A total of 106 patients were randomized in a 3:3:4 ratio to receive placebo, sotatercept 0.3 mg/kg, or sotatercept 0.7 mg/kg subcutaneously every 21 days with standard-of-care therapies in combination. The trial was powered to detect an 18% reduction in the primary endpoint of PVR and a 24-meter improvement in the secondary endpoint of 6MWD.

    Following the 6-month double-blind treatment period, participants in the trial were eligible to continue in the 18-month extension period.

    Conference Call and Webcast

    The Company will host a webcast and conference call to discuss the topline results from the PULSAR Phase 2 trial on June 24, 2020, at 4:30 p.m. EDT.

    The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company's website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron ATS 2020 Conference Call."

    The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

    About Sotatercept

    Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, which is a key molecular driver of PAH. In preclinical research recently published in Science Translational Medicine, sotatercept exhibited consistent effects across multiple components of disease, including suppressed proliferation of pulmonary arterial smooth muscle and microvascular endothelial cells, reduced pulmonary pressures, lessened right ventricular hypertrophy, improved right ventricular function, and attenuated vascular remodeling. Sotatercept, which is part of a licensing agreement with Bristol Myers Squibb, is also being evaluated in the exploratory, open-label SPECTRA Phase 2 trial in patients with PAH. For more information, please visit www.clinicaltrials.gov.

    Sotatercept, which has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration and Priority Medicine (PRIME) designation from the European Medicines Agency in PAH, is an investigational therapy that is not approved for any use in any country.

    About PAH

    PAH is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The 5-year survival rate for patients with PAH is approximately 57%. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-beta signaling as a primary driver of PAH in familial, idiopathic, and acquired forms of the disease.

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension.

    For more information, please visit www.acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.

    Forward-Looking Statements

    This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development of sotatercept in PAH, the timeline for clinical development and regulatory approval of sotatercept in PAH, the expected timing for reporting of data from ongoing clinical trials, and the potential of Acceleron's compounds as therapeutic drugs. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "possible," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

    Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that preclinical testing of Acceleron's compounds and data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds may take longer and/or cost more than planned, that Acceleron may be unable to successfully complete the clinical development of Acceleron's compounds, that Acceleron may be delayed in initiating, enrolling or completing any clinical trials, that Acceleron's compounds may not receive regulatory approval or become commercially successful products, and that Breakthrough Therapy or PRIME designation may not expedite the development or review of sotatercept. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings that Acceleron has made and may make with the SEC in the future.

    The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

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