NewAmsterdam Pharma Doses First Patient in Phase 3 TANDEM Clinical Trial Evaluating Fixed-Dose Combination of Obicetrapib and Ezetimibe in Patients with HeFH and/or ASCVD
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Insights
The initiation of the TANDEM Phase 3 clinical trial is a significant development in the field of cardiovascular medicine, specifically for patients with Heterozygous Familial Hypercholesterolemia and Atherosclerotic Cardiovascular Disease. The combination of obicetrapib, a Cholesteryl Ester Transfer Protein (CETP) inhibitor, with ezetimibe, which inhibits the absorption of cholesterol in the small intestine, could potentially offer a new therapeutic option for patients who are not achieving their LDL-C targets with current lipid-modifying therapies.
From a clinical standpoint, the primary endpoint of evaluating the effect of the fixed-dose combination on LDL-C levels is pertinent, as lowering LDL-C is strongly associated with reduced risk for cardiovascular events. The secondary endpoints, focusing on apolipoprotein B and non-HDL cholesterol, are equally important, as these are additional markers of cardiovascular risk. The safety and tolerability profile will be crucial for patient compliance and long-term adoption of the therapy, should it be approved.
Given the genetic component of HeFH and the chronic nature of ASCVD, the potential for a once-daily oral treatment that simplifies the management of dyslipidemia could have a substantial impact on patient quality of life and cardiovascular outcomes.
The advancement of NewAmsterdam Pharma's TANDEM trial into Phase 3 is a noteworthy event for investors, as it marks a critical stage in the drug's development pathway. The success of this trial could lead to a new product that addresses a significant unmet need in the market for lipid-lowering therapies.
Financially, the implications of a successful trial are considerable. The global market for lipid regulators is substantial and a new, effective treatment could capture a significant share, especially considering the high prevalence of dyslipidemia and cardiovascular diseases. Investors will be closely monitoring enrollment numbers and trial progress as indicators of potential future revenue streams.
However, it is essential to acknowledge the risks involved in drug development, including clinical, regulatory and commercialization challenges. Delays in trial progress, adverse safety findings, or failure to meet primary and secondary endpoints could negatively impact NewAmsterdam's stock performance and financial outlook.
The landscape of cardiovascular disease treatments is highly competitive, with many players seeking to introduce novel therapies. The TANDEM trial's focus on a fixed-dose combination of obicetrapib and ezetimibe positions NewAmsterdam Pharma to potentially enter the market with a differentiated product. The convenience of a once-daily pill could be a strong selling point in a market where patient adherence to treatment regimens is a known challenge.
Market adoption of the FDC, if approved, will depend on factors such as the clinical benefit over existing therapies, pricing, insurance coverage and physician prescribing habits. The anticipated enrollment of approximately 400 patients in the TANDEM trial will provide a significant dataset to support marketing efforts, but the company will need to carefully strategize its launch and positioning against established therapies and other emerging treatments.
Additionally, the parallel progression of NewAmsterdam's other Phase 3 trials, BROOKLYN, BROADWAY and PREVAIL, suggests a robust pipeline that could enhance the company's credibility and valuation in the eyes of stakeholders, assuming positive outcomes from these studies.
-- TANDEM is designed to evaluate obicetrapib and ezetimibe fixed-dose combination (“FDC”) as an adjunct to diet and maximally tolerated lipid lowering therapy in patients with HeFH, ASCVD or ASCVD risk equivalents requiring additional lowering of low-density lipoprotein cholesterol (“LDL-C”) --
-- Company expects to report topline data in the first quarter of 2025 --
-- TANDEM is being conducted in parallel with BROOKLYN, BROADWAY and PREVAIL pivotal Phase 3 studies; data from BROOKLYN and BROADWAY expected in 3Q and 4Q 2024, respectively --
NAARDEN, The Netherlands and MIAMI, March 12, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced the dosing of the first patient and initiation of TANDEM, a pivotal Phase 3 clinical trial to evaluate obicetrapib and ezetimibe FDC in adult patients with Heterozygous Familial Hypercholesterolemia (“HeFH”) and/or Atherosclerotic Cardiovascular Disease (“ASCVD”) or multiple risk factors for ASCVD, whose LDL-C is not adequately controlled despite being on maximally tolerated lipid-modifying therapies.
“Ezetimibe has become an established therapy for patients with elevated cholesterol at high risk of CVD; however, a significant proportion of these patients remain unable to achieve target LDL-C levels, highlighting a major unmet need. The ACC Expert Consensus set LDL-C goals of below the 70mg/dl for patients with ASCVD and below the 55mg/dl goal for ASCVD patients with very high risk. By combining obicetrapib, if approved, with ezetimibe in a once-daily pill, this FDC therapy could meaningfully improve the tools available to physicians for addressing elevated LDL-C, and lead to improved health outcomes for millions of people living with increased risk for CVD globally,” said Ashish Sarraju, M.D., Cardiovascular Medicine, Cleveland Clinic.
The primary objective of the placebo-controlled, double-blind, four-arm, randomized TANDEM trial is to evaluate the effect of 10mg obicetrapib and 10mg ezetimibe FDC on LDL-C levels, compared to both ezetimibe 10mg and obicetrapib 10mg monotherapy and to placebo. Secondary objectives include evaluating the effect of the FDC on apolipoprotein B (“ApoB”) and non-high-density lipoprotein cholesterol (“non-HDL-C”). The trial will also evaluate the safety and tolerability profile of the FDC.
NewAmsterdam anticipates enrolling approximately 400 patients on maximally tolerated lipid-modifying therapies with HeFH, ASCVD or ASCVD risk equivalents and who have a baseline LDL-C of at least 70 mg/Dl. Patients will be randomized to one of the following groups: placebo, 10 mg obicetrapib, 10mg ezetimibe, or obicetrapib 10mg and ezetimibe 10mg FDC tablet, all groups dosed as a once-daily oral treatment for an 84-day treatment period. HeFH is an inherited genetic disorder that causes dangerously high cholesterol levels. Patients with ASCVD have established cardiovascular diseases characterized by the buildup of plaque in the arteries, including coronary artery stenosis, myocardial infarction or stroke. Patients who have multiple risk factors for ASCVD are at high risk of experiencing a cardiovascular event in the near future.
“The initiation of the Phase 3 TANDEM trial represents a significant milestone for NewAmsterdam, as we are now progressing four pivotal Phase 3 trials – BROADWAY, BROOKLYN, PREVAIL, and TANDEM,” said Michael Davidson, M.D., Chief Executive Officer at NewAmsterdam Pharma. “Supported by compelling data from our Phase 2 ROSE2 trial, which demonstrated meaningful reductions in LDL-C, non-HDL, and ApoB in patients already using high-intensity statin therapy, we believe the combination of obicetrapib, if approved, and ezetimibe, in a simple and convenient once-daily tablet, is highly synergistic, with the potential to significantly improve cardiovascular disease treatment for millions of people living with dyslipidemia. TANDEM is a critical component of NewAmsterdam’s multi-pronged clinical strategy, and we are optimistic that favorable results from this trial, together with our other ongoing Phase 3 studies, can position us to advance our novel CETP inhibitor franchise as a much-needed therapeutic solution.”
About Obicetrapib
Obicetrapib is a novel, oral, low-dose CETP inhibitor that NewAmsterdam is developing to overcome the limitations of current LDL-lowering treatments. The Company believes that obicetrapib has the potential to be a once-daily oral CETP inhibitor for lowering LDL-C, if approved. In the Company’s Phase 2b ROSE trial, obicetrapib demonstrated a
About NewAmsterdam
NewAmsterdam Pharma (Nasdaq: NAMS) is a late-stage biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. We seek to fill a significant unmet need for a safe, well tolerated and convenient LDL-lowering therapy. In multiple phase 3 studies, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as preferred LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated.
Forward-Looking Statements
Certain statements included in this document that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements generally are accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “could,” “plan,” “predict,” “potential,” “position,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the Company’s business and strategic plans, the therapeutic and curative potential of the Company’s product candidate, the Company’s clinical trials and the timing for enrolling patients, the timing and forums for announcing data, the achievement and timing of regulatory approvals and plans for commercialization. These statements are based on various assumptions, whether or not identified in this document, and on the current expectations of the Company’s management and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as and must not be relied on as a guarantee, an assurance, a prediction, or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and may differ from assumptions. Many actual events and circumstances are beyond the control of the Company. These forward-looking statements are subject to a number of risks and uncertainties, including changes in domestic and foreign business, market, financial, political, and legal conditions; risks relating to the uncertainty of the projected financial information with respect to the Company; risks relating to the uncertainty of the projected financial information with respect to the Company; risks related to the approval of the Company’s product candidate and the timing of expected regulatory and business milestones, including potential commercialization; ability to negotiate definitive contractual arrangements with potential customers; the impact of competitive product candidates; ability to obtain sufficient supply of materials; global economic and political conditions; the effects of competition on the Company’s future business; and those factors described in the Company’s public filings with the U.S. Securities and Exchange Commission. Additional risks related to the Company’s business include, but are not limited to: uncertainty regarding outcomes of the Company’s ongoing clinical trials, particularly as they relate to regulatory review and potential approval for its product candidate; risks associated with the Company’s efforts to commercialize a product candidate; the Company’s ability to negotiate and enter into definitive agreements on favorable terms, if at all; the impact of competing product candidates on the Company’s business; intellectual property related claims; the Company’s ability to attract and retain qualified personnel; ability to continue to source the raw materials for its product candidate. If any of these risks materialize or the Company’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that the Company does not presently know or that the Company currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. In addition, forward-looking statements reflect the Company’s expectations, plans, or forecasts of future events and views as of the date of this document and are qualified in their entirety by reference to the cautionary statements herein. The Company anticipates that subsequent events and developments may cause the Company’s assessments to change. These forward-looking statements should not be relied upon as representing the Company’s assessment as of any date subsequent to the date of this communication. Accordingly, undue reliance should not be placed upon the forward-looking statements. Neither the Company nor any of its affiliates undertakes any obligation to update these forward-looking statements, except as may be required by law.
Company Contact
Matthew Philippe
P: 1-917-882-7512
matthew.philippe@newamsterdampharma.com
Media Contact
Spectrum Science on behalf of NewAmsterdam
Jen Gordon
P: 1-202-957-7795
jgordon@spectrumscience.com
Investor Contact
Stern Investor Relations on behalf of NewAmsterdam
Hannah Deresiewicz
P: 1-212-362-1200
hannah.deresiewicz@sternir.com
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