UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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| Item 7.01 | Regulation FD Disclosure. |
On July 29, 2024, NewAmsterdam Pharma Company N.V. (the “Company”) issued a press release announcing positive topline results from its Phase 3 BROOKLYN clinical trial evaluating obicetrapib in adult patients with heterozygous familial hypercholesterolemia (“HeFH”) and whose low-density lipoprotein cholesterol (“LDL-C”) is not adequately controlled, despite being on maximally tolerated lipid-lowering therapy. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The Company is hosting a live webcast and conference call to discuss the positive topline results of the BROOKLYN clinical trial on July 29, 2024 at 8:30 a.m., Eastern Time, and a live webcast of the call will be available through the Company’s website. A copy of the slide presentation to be used by the Company during the conference call is attached hereto as Exhibit 99.2 and incorporated herein by reference.
The information contained in this Item 7.01, including Exhibits 99.1 and 99.2, is being “furnished” and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that Section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”). The information contained in this Item 7.01, including Exhibits 99.1 and 99.2, shall not be incorporated by reference into any registration statement or other document pursuant to the Securities Act or into any filing or other document pursuant to the Exchange Act, except as otherwise expressly stated in any such filing.
| Item 8.01 | Other Events |
On July 29, 2024, the Company announced positive topline results from its BROOKLYN clinical trial, a 52-week, global, pivotal, Phase 3, randomized, double-blind, placebo-controlled multicenter trial to evaluate the efficacy and safety of 10 mg obicetrapib compared to placebo as an adjunct to maximally tolerated lipid-lowering therapies in patients with HeFH whose LDL-C is not adequately controlled. The trial met its primary endpoint, with the obicetrapib arm achieving a statistically significant reduction of LDL-C versus placebo at day 84. The observed reduction was sustained at day 365 versus the placebo. The trial also met several of its prespecified secondary endpoints with statistical significance. 51% of patients in the treatment arm achieved an LDL-C level below 70 mg/dl.
Topline results from the BROOKLYN trial are as follows:
LDL-C LS mean percentage change:
| % Change from Baseline | Obicetrapib % Change | |||||||
| Placebo (n=118) | Obicetrapib (n=236) | Compared to Placebo | p-value | |||||
Day 84 |
+0.3% | -36.1% | -36.3% | <0.0001 | ||||
Day 365 |
+10.3% | -31.1% | -41.5% | <0.0001 | ||||
The BROOKLYN trial was conducted at sites in North America, Europe and Africa. A total of 354 patients were randomized 2:1 to receive 10 mg obicetrapib or placebo dosed as a once-daily oral treatment, with or without food. The mean baseline LDL-C for enrolled patients in the obicetrapib arm was 123 mg/dL despite high intensity statin use reported by approximately 79% of patients during screening. Females comprised approximately 53% of the study population and the median age of participants at baseline was 57 years.
Obicetrapib was observed to be generally well-tolerated, with safety results comparable to placebo and no increase in blood pressure. The treatment discontinuation rate for the obicetrapib arm was 7.6% versus 14.4% for the placebo. The incidence of treatment emergent adverse events (“TEAEs”), study-drug related TEAEs and treatment-emergent serious adverse events (“TESAEs”) are summarized in the table below:
Placebo N=118 n (%) |
Obicetrapib 10 mg N=234 n (%) |
Total N=352 n (%) | ||||
Any TEAEs |
83 (70.3) | 149 (63.7) | 232 (65.9) | |||
Any study drug related TEAEs |
8 (6.8) | 10 (4.3) | 18 (5.1) | |||
Any TEAEs leading to discontinuation of study drug |
8 (6.8) | 10 (4.3) | 18 (5.1) | |||
Any TESAEs |
8 (6.8) | 13 (5.6) | 21 (6.0) |
The Company plans to present full results from BROOKLYN at an upcoming medical conference and to publish the data in a major medical journal.
Forward-Looking Statements
Certain statements included in this Current Report on Form 8-K 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,” “plan,” “predict,” “potential,” “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 Company’s commercial opportunity, 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 Current Report on Form 8-K, 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 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, including the Russia-Ukraine and Israel-Hamas conflict; the effects of competition on the Company’s future business; and those factors described in the Company’s public filings with the 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 Current Report on Form 8-K 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.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits.
EXHIBIT NUMBER |
EXHIBIT DESCRIPTION | |
| 99.1 | Press Release, dated July 29, 2024. | |
| 99.2 | NewAmsterdam Pharma Company N.V. July 29, 2024 BROOKLYN Presentation. | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| NewAmsterdam Pharma Company N.V. | ||
| By: | /s/ Michael Davidson | |
| Michael Davidson | ||
| Chief Executive Officer | ||
Dated: July 29, 2024
Exhibit 99.1
NewAmsterdam Pharma Announces Positive Topline Data from Pivotal Phase 3 BROOKLYN Clinical Trial
Evaluating Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia
— Achieved primary endpoint of LS mean reduction in LDL-C on top of maximally tolerated lipid modifying therapies
at day 84 with statistically significant reduction (p<0.0001 ), which was sustained at day 365 (p<0.0001) —
— Obicetrapib lowered LDL-C by 36.3% at day 84 and 41.5% at day 365, compared to placebo —
— Observed to be generally well-tolerated with safety results comparable to placebo —
— NewAmsterdam to host conference call at 8:30 a.m. ET, Today —
Naarden, the Netherlands and Miami, USA; July 29, 2024 – 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 positive topline data from the Company’s Phase 3 BROOKLYN clinical trial (NCT05425745). BROOKLYN, the first of four studies in NewAmsterdam’s pivotal clinical development program, was designed to evaluate obicetrapib in adult patients with heterozygous familial hypercholesterolemia (“HeFH”), whose LDL-C is not adequately controlled, despite being on maximally tolerated lipid-lowering therapy.
The BROOKLYN trial met its primary endpoint, achieving an LS mean reduction of 36.3% (p < 0.0001) compared to placebo at day 84, which was sustained at day 365 with an LS mean LDL-C reduction of 41.5% (p < 0.0001). The observed reductions in other biomarkers, including high-density lipoprotein cholesterol (“HDL-C”), non-HDL-C, lipoprotein(a) (“Lp(a)”), and apolipoprotein B (“ApoB”), met statistical significance and were consistent with data reported from the Company’s prior clinical trials.
LDL-C LS mean percentage change:
| % Change from Baseline | Obicetrapib % Change | |||||||||
| Placebo (n=118) | Obicetrapib (n=236) | Compared to Placebo | p-value | |||||||
| Day 84 |
+0.3% | -36.1% | -36.3% | <0.0001 | ||||||
| Day 365 |
+10.3% | -31.1% | -41.5% | <0.0001 | ||||||
“My career has been dedicated to the treatment of patients with familial hypercholesterolemia,” said John Kastelein, M.D., Ph.D., FESC, Chief Scientific Officer of NewAmsterdam. “Many of these patients have exhausted available treatment options and we are delighted by the topline data from BROOKLYN, with 51% of patients achieving an LDL-C level below 70 mg/dl. Our goal is to provide physicians and patients with a novel, once-daily, low dose, oral option that can potentially transform the treatment landscape.”
In the trial, obicetrapib was observed to be well-tolerated, with safety results comparable to placebo and no increase in blood pressure. The treatment discontinuation rate for the obicetrapib arm was 7.6% versus 14.4% for placebo. The incidence of treatment-emergent adverse events (“TEAEs”), study-drug related TEAEs, and treatment-emergent serious adverse events (“TESAEs”) are summarized in the table below.
| Placebo N=118 n (%) |
Obicetrapib 10 mg n (%) |
Total N=352 n (%) | ||||
| Any TEAEs |
83 (70.3) | 149 (63.7) | 232 (65.9) | |||
| Any study drug related TEAEs |
8 (6.8) | 10 (4.3) | 18 (5.1) | |||
| Any TEAEs leading to discontinuation of study drug |
8 (6.8) | 10 (4.3) | 18 (5.1) | |||
| Any TESAEs |
8 (6.8) | 13 (5.6) | 21 (6.0) |
“Today’s announcement marks an important milestone for NewAmsterdam, the HeFH community, and CVD more broadly,” said Michael Davidson, M.D., Chief Executive Officer of NewAmsterdam. “Despite the widespread availability of lipid lowering therapies, CVD-related deaths have risen and patients remain above LDL-C targets. Patients and their doctors need additional options. We are very excited about the results from our BROOKLYN trial and believe they support obicetrapib’s potential to significantly reduce LDL-C in a challenging patient population, over a duration of one year. Adverse events and discontinuations due to side effects were similar to placebo, consistent with what was observed in Phase 2 studies. In the safety population, there was also no increase in blood pressure, nor any difference from placebo in liver enzymes, hs-CRP, or renal function. We look forward to building on these results with topline data from BROADWAY expected in the fourth quarter of 2024, and topline data from TANDEM expected in the first quarter of 2025.”
“The data announced today are another indication of obicetrapib’s potential to significantly reduce LDL-C in HeFH patients, a population already on multiple lipid-lowering therapies. I am incredibly encouraged by these results, which suggest that obicetrapib, if approved, has the potential to be a new oral option for a difficult-to-treat patient population and am excited to be a partner with the NewAmsterdam team on the remainder of the obicetrapib pivotal program,” said Stephen Nicholls, M.B.B.S., Ph.D., Director, Monash Victorian Heart Institute and Professor of Cardiology, Monash University.
“HeFH affects 1 in 250 people worldwide and leads to increased risk of major adverse cardiovascular events, including stroke, myocardial infarction, or death, in the prime of life because of life-long burden of high LDL-C. Many individuals living with HeFH are unable to attain guideline-recommended LDL-C levels, despite currently available treatment options,” said Katherine Wilemon, Founder and CEO of the Family Heart Foundation. “Familial hypercholesterolemia often requires multiple therapies to achieve safer levels of LDL cholesterol. We are highly encouraged with these results and the potential to have another efficacious oral option.”
NewAmsterdam plans to present full results from BROOKLYN at an upcoming medical conference and to publish the data in a major medical journal.
Design of the Pivotal Phase 3 BROOKLYN Clinical Trial
The 52-week, global, pivotal, Phase 3, randomized, double-blind, placebo-controlled multicenter study evaluated the efficacy and safety of 10 mg obicetrapib compared to placebo as an adjunct to maximally tolerated lipid-lowering therapies in patients with HeFH whose LDL-C is not adequately controlled. The study was conducted at sites in North America, Europe and Africa. A total of 354 patients were randomized 2:1 to receive 10 mg obicetrapib or placebo dosed as a once-daily oral treatment, with or without food. The mean baseline LDL-C for enrolled patients in the obicetrapib arm was 123 mg/dL despite high intensity statin use reported by approximately 79% of patients during screening. Females comprised approximately 53% of the study population and the median age of participants at baseline was 57 years.
The primary endpoint was percent change from baseline in LDL-C of obicetrapib 10 mg compared to placebo after 84 days. Secondary endpoints also included percent changes from baseline of obicetrapib 10 mg compared to placebo after 84 days in HDL-C, non- HDL-C, ApoB, and Lp(a). The trial also evaluated the safety and tolerability profile of obicetrapib.
Conference Call and Webcast Information
NewAmsterdam will host a live webcast and conference call to review the topline results from BROOKLYN at 8:30 a.m. ET today. To access the live webcast, participants may register here. The live webcast will be available under the “Events” section of the Investor Relations page of the NewAmsterdam website at ir.newamsterdampharma.com.
To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. While not required, it is recommended that participants join the call ten minutes prior to the scheduled start. An archived replay of the webcast will be available on NewAmsterdam’s website.
About NewAmsterdam’s Global Pivotal Phase 3 Program
NewAmsterdam’s global, pivotal Phase 3 clinical development program consists of four studies in over 12,250 patients, three for obicetrapib monotherapy and one for a fixed-dose combination (“FDC”) of obicetrapib and ezetimibe:
| • | BROOKLYN evaluated obicetrapib in patients with HeFH, whose LDL-C is not adequately controlled, despite being on maximally tolerated lipid-lowering therapy. NewAmsterdam reported topline data in the third quarter of 2024. |
| • | BROADWAY is evaluating obicetrapib in adult patients with established atherosclerotic cardiovascular disease (“ASCVD”) and/or HeFH, whose LDL-C is not adequately controlled, despite being on maximally tolerated lipid-lowering therapy. NewAmsterdam completed enrollment of over 2,500 patients in July 2023 and expects to report topline data in the fourth quarter of 2024. |
| • | TANDEM is evaluating obicetrapib as part of a FDC tablet with ezetimibe, a non-statin oral LDL-lowering therapy, in patients with established ASCVD or multiple risk factors for ASCVD and/or HeFH, whose LDL-C is not adequately controlled despite being on maximally tolerated lipid-lowering therapy. NewAmsterdam completed enrollment of over 400 patients in July 2024 and expects to report topline data in the first quarter of 2025. |
| • | PREVAIL is a cardiovascular outcomes trial evaluating obicetrapib in patients with a history of ASCVD, whose LDL-C is not adequately controlled, despite being on maximally tolerated lipid-lowering therapy. NewAmsterdam completed enrollment of over 9,500 patients in April 2024. |
About Obicetrapib
Obicetrapib is a novel, oral, low-dose CETP inhibitor that NewAmsterdam is developing to overcome the limitations of current LDL-lowering treatments. In each of the Company’s Phase 2 trials, ROSE2, TULIP, ROSE, and OCEAN, as well as the Company’s Phase 3 BROOKLYN trial, evaluating obicetrapib as monotherapy or combination therapy, the Company observed statistically significant LDL-lowering combined with a side effect profile similar to that of placebo. The Company is conducting an additional Phase 3 pivotal trial BROADWAY, to evaluate obicetrapib as a monotherapy used as an adjunct to maximally tolerated lipid-lowering therapies to provide additional LDL-lowering for CVD patients, and TANDEM, to evaluate obicetrapib and ezetimibe as a fixed-dose combination. The Company began enrolling patients in BROADWAY in January 2022 and in TANDEM in March 2024; completing enrollment of BROADWAY in July 2023, and TANDEM in July 2024. The Company also commenced the Phase 3 PREVAIL cardiovascular outcomes trial in March 2022, which is designed to assess the potential of obicetrapib to reduce occurrences of major adverse cardiovascular events, including cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and non-elective coronary revascularization. NewAmsterdam completed enrollment of PREVAIL in April 2024 and randomized over 9,500 patients. Commercialization rights of obicetrapib in Europe, either as a monotherapy or as part of a fixed dose combination with ezetimibe, for cardiovascular diseases have been exclusively granted to the Menarini Group, an Italy-based, leading international pharmaceutical and diagnostics company.
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 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,” “plan,” “predict,” “potential,” “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 Company’s commercial opportunity, 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 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, including the Russia-Ukraine and Israel-Hamas conflict; the effects of competition on the Company’s future business; and those factors described in the Company’s public filings with the Securities 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
Bryan Blatstein
P: 1-917-714-2609
bblatstein@spectrumscience.com
Investor Contact
Precision AQ on behalf of NewAmsterdam
Austin Murtagh
P: 1-212-698-8696
austin.murtagh@precisionaq.com
BROOKLYN Topline Results July 2024 Nasdaq: NAMS Exhibit 99.2
Disclaimer This presentation (together with oral statements made in connection herewith, this “Presentation”) is for informational purposes only. This Presentation shall not constitute an offer to sell, or the solicitation of an offer to buy, any securities, nor shall there be any sale of securities in any states or jurisdictions in which such offer, solicitation or sale would be unlawful. Forward Looking Statements Certain statements included in this Presentation 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,” “plan,” “predict,” “potential,” “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 by NewAmsterdam Pharma Company N.V. (“NewAmsterdam” or the “Company”) regarding estimates and forecasts and projections of market opportunity; the Company's business and strategic plans; expectations and timing related to the success, cost and timing of product development activities, including timing of initiation, completion and data readouts for clinical trials and the potential approval of the Company’s product candidate; the timing for enrolling patients; the timing and forums for announcing data; the size and growth potential of the markets for the Company’s product candidate; the therapeutic and curative potential of the Company’s product candidate; financing and other business milestones; the Company’s expected cash runway; and the Company’s plans for commercialization. These statements are based on various assumptions, whether or not identified in this Presentation, 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 related to the approval of NewAmsterdam’s product candidate and the timing of expected regulatory and business milestones; whether topline, initial or preliminary results from a particular clinical trial will be predictive of the final results of that trial and whether results of early clinical trials will be indicative of the results of later clinical trials; 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, including the Russia-Ukraine conflict, and the war in Israel; the effects of competition on NewAmsterdam’s future business; and those factors discussed in documents filed by the Company with the SEC. Additional risks related to NewAmsterdam’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; and the Company’s ability to continue to source the raw materials for its product candidate, together with the risks described in the Company’s filings made with the U.S. Securities and Exchange Commission from time to time. If any of these risks materialize or NewAmsterdam’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that are presently unknown by the Company or that NewAmsterdam 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 NewAmsterdam’s expectations, plans, or forecasts of future events and views as of the date of this Presentation and are qualified in their entirety by reference to the cautionary statements herein. NewAmsterdam anticipates that subsequent events and developments will cause the Company’s assessments to change. These forward-looking statements should not be relied upon as representing NewAmsterdam’s assessments as of any date subsequent to the date of this Presentation. Accordingly, undue reliance should not be placed upon the forward-looking statements. Neither NewAmsterdam nor any of its affiliates undertakes any obligation to update these forward-looking statements, except as required by law. Market Data Certain information contained in this Presentation relates to or is based on third-party studies, publications, surveys and NewAmsterdam’s own internal estimates and research. In addition, all of the market data included in this Presentation involves a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. Finally, while NewAmsterdam believes its internal research is reliable, such research has not been verified by any independent source and NewAmsterdam cannot guarantee and makes no representation or warranty, express or implied, as to its accuracy and completeness. 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HeFH Overview Heterozygous familial hypercholesterolemia (“HeFH”), a genetic disorder that affects 1 in 250 persons, or approximately 30 million people worldwide, is characterized by elevated levels of low-density lipoprotein cholesterol (“LDL-C”) from birth. Without treatment, the condition is associated with premature complications and death from accelerated development of atherosclerotic cardiovascular disease. Source: Eur Heart J, Volume 34, Issue 45, 1 December 2013, Pages 3478–3490
Study Design and Baseline Characteristics Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies: A Placebo-Controlled, Double-Blind, Randomized, Phase 3 Study to Evaluate the Effect of 10 mg Obicetrapib in Participants with a History of HeFH and LDL‑C ³70 mg/dL who are Not Adequately Controlled by Their Lipid‑Modifying Therapies Key Inclusion Criteria HeFH LDL-C ≥70 mg/dL Maximally tolerated lipid lowering therapy Key Exclusion Criteria HoFH Uncontrolled hypertension Endpoints Primary: LDL-C at day 84 Secondary: ApoB, Lp(a), non-HDL-C, HDL-C Safety: AE’s, vitals, laboratory Study Design Baseline Lipid Modifying Therapy Any statin 89% High intensity statin: 79% Ezetimibe: 54% PCSK9i 14% Other 8% Regions N. America S. Africa Europe Demographics 53% Female 57 years of age BMI: 29 kg/m2 Baseline Lipids (obicetrapib 10mg mean) Placebo Obicetrapib 10 mg (2:1 randomization) N = 354 13-months 1º endpoint Note: AEs: adverse events; ApoB: apolipoprotein B; HoFH: Homozygous FH
Disposition of All Randomized Participants Placebo Obicetrapib 10 mg Total Randomized 118 236 354 Completed treatment 101 (85.6) 218 (92.4) 319 (90.1) Discontinued treatment 17 (14.4) 18 (7.6) 35 (9.9) Discontinued due to AE’s 7 (5.9) 8 (3.4) 15 (4.2) Subject decision 4 (3.4) 4 (1.7) 8 (2.3) Withdraw of consent 3 (2.5) 3 (1.3) 6 (1.7) Death 2 (1.7) 2 (0.8) 4 (1.1) Lost to follow-up 1 (0.8) 1 (0.4) 2 (0.6) Completed the study 110 (93.2) 226 (95.8) 336 (94.9) Discontinued study early 8 ( 6.8) 10 ( 4.2) 18 ( 5.1) Adverse event 0 ( 0.0) 1 ( 0.4) 1 ( 0.3)
Additional Baseline Details of All Randomized Patients Placebo N=118 Obicetrapib 10 mg N=236 Total N=354 Mean Age (years) 56.6 57.0 56.9 Sex (F) n (%) 65 (55.1) 125 (53.0) 190 (53.7) Region n (%) EMEA 83 ( 70.3) 182 ( 77.1) 265 ( 74.9) North America 35 ( 29.7) 54 ( 22.9) 89 ( 25.1) Race n (%) White 110 ( 93.2) 219 ( 92.8) 329 ( 92.9) African American 3 ( 2.5) 3 ( 1.3) 6 ( 1.7) Height (mean) 169.4 169.9 169.8 Weight (mean) 85.2 85.2 85.2 BMI (mean) 29.6 29.3 29.4 HeFH Genotyping + or DLCN >8 points or SB definite 84 ( 71.2) 172 ( 72.9) 256 ( 72.3) SB possible FH diagnosis 33 ( 28.0) 61 ( 25.8) 94 ( 26.6)
Additional Baseline Details of All Randomized Patients Placebo N=118 Obicetrapib 10 mg N=236 Total N=354 Diabetes n (%) Yes 26 ( 22.0) 47 ( 19.9) 73 ( 20.6) No 92 ( 78.0) 189 ( 80.1) 281 ( 79.4) Statin treatment High Dose 80 ( 67.8) 186 ( 78.8) 266 ( 75.1) Low or Moderate Dose 19 ( 16.1) 23 ( 9.7) 42 ( 11.9) None 19 ( 16.1) 27 ( 11.4) 46 ( 13.0) Ezetimibe use Yes 59 ( 50.0) 127 ( 53.8) 186 ( 52.5) No 59 ( 50.0) 109 ( 46.2) 168 ( 47.5)
Statistically Significant LDL-C Reduction Observed at Primary Endpoint of Day 84 and Maintained Through Day 365 LS Mean % change vs. placebo LDL-C reduction over time (ITT population) 36.3% 41.5% 38.2% 38.3% Note: * = p<0.0001. Martin Hopkins mean changes at baseline, day 30, Day 180, Day 270 and LS mean by PUC at Day 84 and Day 365. * *
Greater Proportion of Patients in Obicetrapib Arm Achieved LDL-C Goal % of patients achieving LDL-C thresholds
LDL-C Responder Analysis in Obicetrapib 10mg arm 34% of patients in obicetrapib arm with >50% LDL-C reduction
Results Consistent with Data Reported from the Company's Prior Clinical Trials Note: LS mean change calculated by PUC. Obicetrapib 10mg for all studies
Overview of Treatment-Emergent Adverse Events – Safety Population Placebo N=118 n (%) Obicetrapib 10 mg N=234 n (%) Total N=352 n (%) Any treatment-emergent AEs (TEAEs) 83 ( 70.3) 149 ( 63.7) 232 ( 65.9) Any TEAEs by maximum severity Mild 47 ( 39.8) 84 ( 35.9) 131 ( 37.2) Moderate 28 ( 23.7) 57 ( 24.4) 85 ( 24.1) Severe 8 ( 6.8) 8 ( 3.4) 16 ( 4.5) Any study drug related TEAEs 8 ( 6.8) 10 ( 4.3) 18 ( 5.1) Any study drug-related TEAEs by maximum severity Mild 5 ( 4.2) 5 ( 2.1) 10 ( 2.8) Moderate 3( 2.5) 5 ( 2.1) 8 ( 2.3) Severe 0 ( 0.0) 0 ( 0.0) 0 ( 0.0) Any TEAEs leading to discontinuation of study drug 8 ( 6.8) 10 ( 4.3) 18 ( 5.1) Any treatment-emergent serious AEs (TESAEs) 8 ( 6.8) 13 ( 5.6) 21 ( 6.0) Any treatment-emergent non-serious AEs 82 ( 69.5) 145 ( 62.0) 227 ( 64.5) Any study drug-related TESAEs 0 ( 0.0) 0 ( 0.0) 0 ( 0.0) Any TEAEs leading to death 2 ( 1.7) 3 ( 1.3) 5 ( 1.4)
Overview of Treatment-Emergent Adverse Events >5% in Either Population Placebo N=118 n (%) Obicetrapib 10 mg N=234 n (%) Total N=352 n (%) Any non-serious treatment-emergent AEs 52 (44.1) 95 (40.6) 147 (41.8) Influenza 7 (5.9) 21 (9.0) 28 (8.0) Covid-19 8 (6.8) 15 (6.4) 23 (6.5) Hypertension 8 (6.8) 14 (6.0) 22 (6.3) Nasopharyngitis 5 (4.2) 15 (6.4) 20 (5.7) Diarrhea 8 (6.8) 9 (3.8) 17 (4.8) Upper respiratory tract infection 4 (3.4) 12 (5.1) 16 (4.5) Back pain 6 (5.1) 7 (3.0) 13 (3.7) Headache 6 (5.1) 7 (3.0) 13 (3.7) Fatigue 7 (5.9) 2 (0.9) 9 (2.6)
Overview of Adverse Events of Special Interest Placebo N=118 n (%) Obicetrapib N=234 n (%) AST or ALT > 3 x ULN 0 (0) 0 (0) Bilirubin > 2 x ULN 2 ( 1.7) 0 (0) CK > 5 x ULN 4 ( 3.4) 3 (1.3 ) NODM or worsening of glycemic control 26 (22.0) 48 (20.5) eGFR < 30 mL/min/1.73m2 or a 25% decrease in eGFR from baseline 10 (8.5) 10 (4.3) Increase of Serum Creatinine ≥ 0.3 mg/dL from baseline 9 (7.6) 5 (2.1) Macular degeneration 0 (0) 0 (0)
Conclusions Primary endpoint met with LS mean reduction in LDL-C at day 84 versus placebo of 36.3% (p<0.0001). LDL-C reduction sustained at day 365 of 41.5% (p<0.0001) 77% of patients receiving obicetrapib achieved LDL-C below 100 mg/dl, with 51% below 70 mg/dl, and 24% below 50 mg/dl 34% of patients in the treatment arm achieved a greater than 50% reduction from baseline in LDL-C at day 84 Reduction in LDL-C seen regardless of background therapy or number of therapies Observed to be generally well-tolerated with safety results comparable to placebo, with no increase in blood pressure or any difference from placebo in liver enzymes, hs-CRP, or renal function Consistent results for non-HDL-C, ApoB, and Lp(a) compared to data observed in Phase 2 studies that will be presented at an upcoming scientific conference
Study Design and Baseline Characteristics of Ongoing Phase 3 Trials Key Inclusion Criteria ASCVD or HeFH LDL-C ≥55 mg/dL w/risk factors, or LDL-C≥ 100 mg/dL Maximally tolerated lipid lowering therapy 13-months Placebo Obicetrapib 10 mg (2:1 randomization) N = 2532 13-months 1º endpoint – day 84 Baseline Lipids (mean) Baseline Lipid Modifying Therapy Any statin 91% High intensity statin: 65% Ezetimibe: 26% PCSK9i 4% Other 11% Key Inclusion Criteria ASCVD LDL-C ≥55 mg/dL w/risk factors, or LDL-C≥ 100 mg/dL Maximally tolerated lipid lowering therapy Placebo Obicetrapib 10 mg (1:1 randomization) N = 9541 54-months LDL-C endpoint Baseline Lipids (mean) Baseline Lipid Modifying Therapy Any statin >90% High intensity statin: 70% Ezetimibe: 23%
Note: Other than as noted, the pipeline represents trials that are currently ongoing. Projections are subject to inherent limitations. Actual results may differ from expectations. The timing of regulatory submissions is subject to additional discussions with regulators. Phase 3 BROADWAY Trial Lipid Mono Study (HeFH or ASCVD; LDL-C ≥ 55 mg/dL; n=2,532) Phase 3 BROOKLYN Trial Lipid Mono Study (HeFH; LDL-C ≥ 70 mg/dL; n=354) Phase 3 CVOT PREVAIL Trial (ASCVD or HeFH; LDL-C ≥ 55 mg/dL; n=9,541) Phase 2b Japan Trial (LDL-C ≥ 70 mg/dL; n=100) Phase 2b ROSE2 Trial (LDL-C ≥ 70 mg/dL; n=114) Phase 3 FDC Trial (LDL-C ≥ 70 mg/dL; n=407) Phase 2a Alzheimer’s Disease Trial (ApoE4 carrier; n=10–15) Ezetimibe FDC Product (obicetrapib 10mg + ezetimibe 10mg) Alzheimer’s Product (proprietary dose/formulation incorporating obicetrapib) Cardiovascular Neuro- metabolic 2022 2023 2024 2025 2026 1H 2H 1H 2H 1H 2H 1H 2H 1H 2H Numerous catalysts expected throughout 2024-2026 Enrollment complete Ph3 readout CVOT readout Initiation Ph2 readout LEGEND Alzheimer’s Ph2a TANDEM FDC Ph3 ROSE2 Ph2b Obicetrapib Monotherapy Product (obicetrapib 10mg) BROADWAY Ph3 BROOKLYN Ph3 PREVAIL CVOT LDL regulatory filing MACE regulatory filing Japan Ph2b LDL regulatory filing Multiple Potential Pivotal Data Readouts in Next 12 Months
LDL-C at day 84 BROOKLYN LDL-C Reduction Applied to PREVAIL Baseline Data Continues to Suggest Hypothetical 20%+ MACE Benefit ~37 mg/dL drop in absolute LDL-C anticipated ~6% drop for a 10mg/dL move Expected to result in more absolute LDL-C reduction LDL-C PREVAIL baseline LDL-C lowering in BROOKLYN 37 mg/dL 103 mg/dL(1) 36% 22% drop Estimated MACE** benefit Note: Actual results may differ from hypothetical calculation. Source: Cholesterol Treatment Trialists Collaboration. Lancet. 2010 376:1670-81 Circulation. 2021;144:e564–e593 17065: Obicetrapib Lowers LDL-C in Patients Taking High Intensity Statins. (1) Represents estimated average baseline LDL to be enrolled, not entry criteria. ** MACE includes cardiovascular death, myocardial infarction, stroke and non-elective coronary revascularization in adults. LDL-C at day 365 Expected to result in more absolute LDL-C reduction LDL-C PREVAIL baseline LDL-C lowering in BROOKLYN 43 mg/dL 103 mg/dL(1) 41% 25% drop Estimated MACE** benefit