Acceleron and Fulcrum Therapeutics Announce Pulmonary Research and Discovery Collaboration Agreement - Associated Press
Acceleron and Fulcrum Therapeutics Announce Pulmonary Research and Discovery Collaboration Agreement - Associated Press |
- Acceleron and Fulcrum Therapeutics Announce Pulmonary Research and Discovery Collaboration Agreement - Associated Press
- Predicting Survival in Patients With Pulmonary Arterial Hypertension - Physician's Weekly
- Pulmonary Arterial Hypertension In Systemic Sclerosis: Challenges In D | OARRR - Dove Medical Press
| Posted: 30 Dec 2019 04:37 AM PST  CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec 30, 2019-- Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, and Fulcrum Therapeutics, Inc. (Nasdaq:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced they have entered into a collaboration and license agreement to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191230005047/en/ "This collaboration brings together Fulcrum's skill in identifying drug targets based on modulation of genetic pathways associated with disease and Acceleron's deep expertise in TGF-beta superfamily signaling in an effort to generate potentially disease-modifying therapeutics," said Habib Dable, Chief Executive Officer of Acceleron Pharma. "With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase 2 trials in pulmonary arterial hypertension—and ACE-1334, we underscore our growing commitment to the development of novel therapies for patients with pulmonary diseases of high unmet medical need." Under the agreement, Acceleron will have access to Fulcrum's unique, proprietary product engine and target identification platform with the potential to identify small molecules that control the expression of genes known to impact specific pathways associated with a pulmonary disease of interest. Acceleron and Fulcrum will collaborate on the identification of therapeutic targets and small molecule drug candidates for those targets. Acceleron will be responsible for all development and commercialization activities for any potential therapeutics identified via this platform. Fulcrum will receive a one-time, upfront payment of $10 million as well as reimbursement for relevant R&D costs. Fulcrum will also be eligible to receive research, development and commercial milestone payments of up to $295 million for a first product commercialized and up to a maximum of $143.5 million in additional milestone payments for all subsequent products commercialized. Fulcrum will additionally receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales. "We are very pleased to partner with Acceleron on this important research initiative," said Robert J. Gould, Ph.D., Chief Executive Officer of Fulcrum Therapeutics. "This collaboration builds on and extends the proven potential of our platform to identify therapies that can address the root cause of diseases, including our progress with losmapimod, currently in a Phase 2 clinical trial for FSHD and extensive pre-clinical and early stage research targeting other genetically defined diseases. This new opportunity to screen and identify pulmonary disease-specific therapies is another reflection of the broad potential applications of the Fulcrum platform in gene modulation." 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 research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol-Myers Squibb, are co-promoting newly approved REBLOZYL ® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension. For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn. About Fulcrum Therapeutics Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. Fulcrum has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia. Please visit www.fulcrumtx.com. Acceleron 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 success of a collaboration with Fulcrum Therapeutics. 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 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 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 will 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 will not receive regulatory approval or become commercially successful products, and that Acceleron's collaboration with Fulcrum Therapeutics will not be successful or result in any successful development candidates. 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, 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. Fulcrum Therapeutics Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding whether the collaboration will yield any targets, potential milestone payments or royalty payments in connection with the collaboration and the potential benefits of the collaboration. All statements, other than statements of historical facts, contained in this press release, including statements regarding Fulcrum's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "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. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with each party's ability to perform its obligations under the agreement, the sufficiency of Fulcrum's cash resources to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements on its expected timeline and other important factors discussed in the "Risk Factors" sections contained in Fulcrum's quarterly and annual reports on file with the Securities and Exchange Commission. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in Fulcrum's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum's views as of the date hereof and should not be relied upon as representing Fulcrum's views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum's views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so. View source version on businesswire.com:https://www.businesswire.com/news/home/20191230005047/en/ CONTACT: Acceleron Pharma Inc.Investors: Todd James, IRC, 617-649-9393 Vice President, Investor Relations and Corporate CommunicationsEd Joyce, 617-649-9242 Director, Investor RelationsMedia: Matt Fearer, 617-301-9557 Director, Corporate CommunicationsFulcrum TherapeuticsInvestor Contact: Stephanie Ascher Stern Investor Relations, Inc. stephanie.ascher@sternir.com 212-362-1200Media Contact: Adam Daley Berry & Company Public Relations adaley@berrypr.com 212-253-8881 KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Acceleron Pharma Inc. and Fulcrum Therapeutics Copyright Business Wire 2019. PUB: 12/30/2019 07:30 AM/DISC: 12/30/2019 07:30 AM http://www.businesswire.com/news/home/20191230005047/en  |
| Predicting Survival in Patients With Pulmonary Arterial Hypertension - Physician's Weekly Posted: 19 Dec 2019 12:00 AM PST A study has found that an updated risk calculator is a better discriminator of risk than other risk assessment strategies for patients with pulmonary arterial hypertension. When managing pulmonary arterial hypertension (PAH), regular risk assessments are recommended to guide treatment decisions and potentially improve morbidity and mortality. "Risk stratification is important in PAH because it can impact how patients are treated," explains Raymond L. Benza, MD. "Overestimating risks can result in overtreatment and substantially increase costs from therapy. Underestimating risk can result a greater risk of death. As such, ensuring that risk estimates are accurate is paramount in the management of PAH." Data from patient registries have been used to develop algorithms that estimate survival in PAH and to inform treatment guidelines. One such algorithm is the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) risk calculator, which uses up to 12 clinically relevant variables. Three additional risk assessment tools have also been developed using patient populations from the Swedish Pulmonary Arterial Hypertension Register (SPAHR), the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA), and the French Pulmonary Hypertension Registry (FPHR). These risk assessments utilize thresholds in 4 to 8 variables. A Comparative AnalysisTo enhance risk prediction in PAH, a new variable—all-cause hospitalization—and a revised variable—renal function measured by estimated glomerular filtration rate, or eGFR—were added to update the REVEAL risk calculator to REVEAL 2.0. Dr. Benza and colleagues had a study published in Chest that compared REVEAL 2.0 with the original REVEAL calculator and also compared risk discrimination with REVEAL 2.0 versus other contemporary risk assessment strategies. The authors analyzed a subpopulation from the REVEAL registry that survived 1 year or longer after being enrolled. Mortality estimates and discrimination were compared between REVEAL 2.0, COMPERA, and FPHR risk assessment strategies. The SPAHR strategy was not included because it is similar to the COMPERA strategy. A three-category REVEAL 2.0 score was computed in which patients were classified as low-, intermediate-, or high-risk.
Assessing Key FindingsThe study showed that REVEAL 2.0 demonstrated similar discrimination as the original calculator, provided excellent separation of risk among the risk categories, and predicted clinical worsening and mortality. "Importantly, the REVEAL 2.0 three-category score had greater discrimination of risk than COMPERA or FPHR," says Dr. Benza. When compared with REVEAL 2.0, COMPERA and FPHR both underestimated and overestimated risk (Table). In the REVEAL 2.0 low-risk group, COMPERA and FPHR overestimated risk in 51% and 60% of patients, respectively. For the REVEAL 2.0 intermediate-risk group, COMPERA and FPHR underestimated risk in 22% and 15% of patients, and overestimated risk in 4% and 19% of patients, respectively. In the highest risk group, COMPERA and FPHR underestimated risk in 80% and 58% of patients, respectively. Importantly, the REVEAL 2.0 three-category score discriminated risk better than the COMPERA and FPHR risk assessment strategies in a mixed group of patients that included those with newly diagnosed PAH and those with a previous diagnosis. "REVEAL 2.0 is a simple, more pragmatic platform for risk assessment in PAH because it is more discriminatory than other tools," Dr. Benza says. "This information is critical because it can help guide treatment decisions." Looking AheadThe REVEAL 2.0 risk calculator can be a valuable asset to standard clinical assessments because they provide a quantitative measure that can be easily tracked over time. REVEAL 2.0 more accurately stratifies individual patient risk than other assessments because it includes multiple, weighted modifiable and nonmodifiable variables. It also offers the advantage of predicting clinical worsening, which may enhance the ability to identify high-risk patients earlier in the disease course and support more informed treatment decisions. "For clinicians who already routinely use risk assessment algorithms, the REVEAL 2.0 calculator can be a useful addition to patient care," says Dr. Benza. "Other available risk assessment strategies can be used as screening tools in PAH. In the future, efforts are needed to incorporate REVEAL 2.0 into clinical guidelines so that this tool will be used more routinely in clinical practice." ReferencesBenza RL, Gomberg-Maitland M, Elliott CG, et al. Predicting survival in patients with pulmonary arterial hypertension: The REVEAL Risk Score Calculator 2.0 and comparison with ESC/ERS-based risk assessment strategies. Chest. 2019 Feb 14 [Epub ahead of print]. Available at: https://journal.chestnet.org/article/S0012-3692(19)30152-7/fulltext. Benza RL, Lohmueller LC, Kraisangka J, Kanwar M. Risk assessment in pulmonary arterial hypertension patients: the long and short of it. Adv Pulm Hypertens. 2018;16:125-135. Benza RL, Miller DP, Foreman AJ, et al. Prognostic implications of serial risk score assessments in patients with pulmonary arterial hypertension: a Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management (REVEAL) analysis. J Heart Lung Transplant. 2015;34:356-361. Benza RL, Miller DP, Barst RJ, Badesch DB, Frost AE, McGoon MD. An evaluation of long-term survival from time of diagnosis in pulmonary arterial hypertension from the REVEAL Registry. Chest. 2012;142:448-456. Farber HW, Benza RL. Risk assessment tools in pulmonary arterial hypertension. Prognosis for prospective trials? Am J Respir Crit Care Med. 2018;197:843-845. Benza RL, Miller DP, Gomberg-Maitland M, et al. Predicting survival in pulmonary arterial hypertension: insights from the Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management (REVEAL). Circulation. 2010;122:164-172.  |
| Pulmonary Arterial Hypertension In Systemic Sclerosis: Challenges In D | OARRR - Dove Medical Press Posted: 26 Dec 2019 04:52 PM PST Didem Saygin,1 Robyn T Domsic2 1Department of Medicine, University of Pittsburgh Medical Center, Pittsburgh, PA, USA; 2Division of Rheumatology and Clinical Immunology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA Correspondence: Robyn T Domsic Abstract: Systemic sclerosis (SSc) is a chronic, multisystem autoimmune disease characterized by vasculopathy, fibrosis and immune system activation. Pulmonary hypertension and interstitial lung disease account for majority of SSc-related deaths. Diagnosis of SSc-PAH can be challenging due to nonspecific clinical presentation which can lead to delayed diagnosis. Many screening algorithms have been developed to detect SSc-associated pulmonary arterial hypertension (SSc-PAH) in early stages. Currently used PAH-specific medications are largely extrapolated from IPAH studies due to smaller number of patients with SSc-PAH. In this review, we discuss the current state of knowledge in epidemiology and risk factors for development of SSc-PAH, and challenges and potential solutions in the diagnosis, screening and management of SSc-PAH. Keywords: scleroderma, pulmonary hypertension, screening
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