NewAmsterdam Pharma Company N.V. (NewAmsterdam Pharma) operates as a late-stage biopharmaceutical company.
The company seeks to fill a significant unmet need for a safe, well-tolerated, and convenient low-density lipoprotein cholesterol (LDL-C) lowering therapy. In multiple Phase 3 trials, the company has investigated obicetrapib, an oral, low-dose, once-daily, highly selective cholesterol ester transfer protein (CETP) inhibitor, alone or as a fixed-dose combination with ezetimibe, as preferred...
NewAmsterdam Pharma Company N.V. (NewAmsterdam Pharma) operates as a late-stage biopharmaceutical company.
The company seeks to fill a significant unmet need for a safe, well-tolerated, and convenient low-density lipoprotein cholesterol (LDL-C) lowering therapy. In multiple Phase 3 trials, the company has investigated obicetrapib, an oral, low-dose, once-daily, highly selective cholesterol ester transfer protein (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 cardiovascular disease (CVD) with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated.
Obicetrapib is a next-generation, oral, low-dose, highly selective CETP inhibitor that the company is developing to potentially overcome the limitations of current LDL-C lowering treatments. In addition to LDL-C, obicetrapib has shown significant reductions in lipoprotein(a) (Lp(a)) and small LDL particles, all with safety comparable to placebo. In each of the company's Phase 3 clinical trials, BROADWAY and BROOKLYN, evaluating obicetrapib as an adjunct to high-intensity statin therapy, obicetrapib met its primary and secondary endpoints, with statistically significant reductions in LDL-C observed. In the company's Phase 3 TANDEM clinical trial, evaluating obicetrapib in combination with ezetimibe as an adjunct to high-intensity statin therapy, obicetrapib in combination with ezetimibe met its primary and secondary endpoints, with statistically significant reductions in LDL-C observed. In five of the company's Phase 2 clinical trials, TULIP, ROSE, OCEAN, ROSE2, and the Japan Phase 2b clinical trial, evaluating obicetrapib as a monotherapy or a combination therapy with ezetimibe 10 mg, the company observed statistically significant LDL-C lowering with side effects similar in frequency and severity to placebo, including muscle-related side effects and drug-related treatment-emergent serious adverse events (TESAEs).
The company's intention is to develop and commercialize an LDL-C lowering monotherapy and a fixed-dose combination therapy, which offers the advantage of a single, low dose, once-daily oral pill, and fulfills the significant unmet need for an effective and convenient LDL-C lowering therapy. If the company obtains marketing approval, it intends to commercialize obicetrapib for patients with ASCVD and/or HeFH and elevated levels of LDL-C despite being treated with available optimal lipid lowering therapy.
The company has partnered with Menarini, providing them with the exclusive rights to commercialize obicetrapib 10 mg, either as a sole active ingredient product or in a fixed-dose combination with ezetimibe, in the majority of European countries, if approved. Subject to receipt of marketing approval, the company's current plan is to pursue the development and commercialization of obicetrapib in the United States itself, and to consider additional partners for jurisdictions outside of the United States and the European Union (the EU), including in Japan and China. In addition to its partnership with Menarini, the company may in the future utilize a variety of types of collaboration, license, monetization, distribution, and other arrangements with other third parties relating to the development or commercialization, once approved, of obicetrapib or future product candidates or indications. The company is also continually evaluating the potential acquisition or license of new product candidates.
The company conducted two Phase 3 pivotal clinical trials, BROADWAY and BROOKLYN, to evaluate obicetrapib as a monotherapy used as an adjunct to maximally tolerated lipid-lowering therapies to potentially enhance LDL-C lowering in patients with ASCVD and/or HeFH. The company announced topline results for BROOKLYN in July 2024 and for BROADWAY in December 2024, both of which met the primary endpoint for the study with safety and tolerability comparable to placebo. The company currently expects to report additional data from each study over the course of 2025. In March 2022, the company commenced its Phase 3 PREVAIL CVOT, which is designed to assess the potential of obicetrapib to reduce occurrences of MACE, including cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and non-elective coronary revascularization in at least 9,000 patients. The company completed enrollment in PREVAIL in April 2024 and expects to complete the study by the end of 2026.
The company is also investigating obicetrapib as a fixed-dose combination with ezetimibe, an oral cholesterol absorption inhibitor and LDL-C lowering therapy, and plans to seek approval for this fixed-dose combination in parallel with obicetrapib monotherapy. In the Phase 3 TANDEM trial, the company evaluated the efficacy and safety of 10 mg obicetrapib and 10 mg ezetimibe as a fixed-dose combination used as an adjunct to diet and maximally tolerated lipid-lowering therapies to potentially enhance LDL-lowering in patients with HeFH, ASCVD, or ASCVD risk equivalents. In November 2024, the company reported data from the Phase 3 TANDEM trial, which met its primary and secondary endpoints, with safety and tolerability comparable to placebo.
The company plans to seek approval of obicetrapib in the United States, the EU, Japan, China, and the United Kingdom. The company conducted multiple Phase 3 trials simultaneously, with clinical plans that incorporate feedback from the FDA, the EMA, the Japan Pharmaceuticals and Medical Devices Agency (PMDA), and the China National Medical Products Administration (NMPA).
Clinically demonstrated anti-diabetic benefits have been observed with CETP inhibition in Phase 3 CVOTs that, if seen in obicetrapib, would differentiate it from current treatment alternatives, especially statin therapy. The company is planning preclinical studies to examine the potential of obicetrapib for patients suffering from diabetes and has included new onset of type 2 diabetes as an endpoint in its PREVAIL CVOT, as measured by AEs indicating Type 2 diabetes, initiation of anti-diabetes medication after confirmed diabetes diagnosis, or high levels of hemoglobin A1c and fasting plasma glucose. In the Phase 3 BROADWAY trial, the company observed a statistically significant improvement in these prespecified AEs of special interest after one year of treatment, which the company hopes to reconfirm in the PREVAIL CVOT trial.
Solution: Enhanced LDL-C Lowering Through CETP Inhibition with Obicetrapib
The company is developing obicetrapib as both a monotherapy and a fixed-dose combination therapy with ezetimibe and has structured its obicetrapib program to overcome the safety, potency, trial design, and commercial viability limitations of prior CETP inhibitors. Further, the company believes that obicetrapib’s oral delivery, demonstrated activity in low doses, chemical properties, and potential tolerability make it well-suited for combination approaches.
Obicetrapib has intrinsic properties, such as ionizable features and substantially reduced lipophilicity. The company has observed a favorable tolerability profile for obicetrapib in an aggregate of over 3,500 patients with dyslipidemia from Phase 1 through Phase 3 clinical trials. The company conducted two Phase 3 pivotal trials, BROADWAY and BROOKLYN, to evaluate obicetrapib as a monotherapy used as an adjunct to maximally tolerated lipid-lowering therapies to potentially enhance LDL-C lowering in patients with ASCVD and/or HeFH. The company completed enrollment for BROADWAY in July 2023 and for BROOKLYN in April 2023. Over 2,500 patients were randomized in the BROADWAY trial and over 350 patients were randomized in the BROOKLYN trial. The company reported top-line data from BROOKLYN in July 2024 and from BROADWAY in December 2024, and currently expect to report additional data from each study over the course of 2025. In March 2022, the company commenced its Phase 3 PREVAIL CVOT, which is designed to assess the potential of obicetrapib to reduce occurrences of MACE, including cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and non-elective coronary revascularization. The company completed enrollment in PREVAIL in April 2024 and expect to complete the trial by the end of 2026.
The company also continues investigating obicetrapib as a fixed-dose combination with ezetimibe following the announcement of data from its Phase 3 TANDEM trial. In parallel with the ROSE2 trial, the company formulated two prototype fixed-dose combination tablets of obicetrapib and ezetimibe. These formulations were compared to the co-administration of obicetrapib and ezetimibe in a pilot bioequivalence trial, which was completed in the first half of 2023. Based on the results of this pilot bioequivalence trial and the data and learnings from the ROSE2 trial, the company selected a formulation for a fixed-dose combination tablet of obicetrapib and ezetimibe and initiated TANDEM, a Phase 3 pivotal trial, to evaluate 10 mg obicetrapib and 10 mg ezetimibe as a fixed-dose combination used as an adjunct to diet and maximally tolerated lipid-lowering therapies to potentially enhance LDL-C lowering in patients with HeFH, ASCVD, or ASCVD risk equivalents, in the first quarter of 2024 and announced topline data in November 2024.
Clinical Development Plan
The company conducted two Phase 3 pivotal trials – the BROADWAY and BROOKLYN trials – designed to measure obicetrapib’s ability to reduce LDL-C as a monotherapy administered as an adjunct to maximally tolerated lipid-modifying therapy. Following its end of Phase 2 meeting with the FDA in the fourth quarter of 2021, the company also commenced its Phase 3 PREVAIL CVOT for obicetrapib as a monotherapy administered as an adjunct to maximally tolerated lipid-modifying therapy in early 2022. In the Phase 2 ROSE2 trial, the company evaluated the effect of a fixed-dose combination of obicetrapib 10 mg with ezetimibe 10 mg on top of high-intensity statin therapy on reduction in LDL-C. In parallel with the ROSE2 trial, the company formulated two prototype fixed-dose combination tablets of obicetrapib and ezetimibe. These formulations were compared to the co-administration of obicetrapib and ezetimibe in a pilot bioequivalence trial, which was completed in the first half of 2023. Based on the results of this pilot bioequivalence trial and the data and learnings from the ROSE2 trial, the company selected a formulation for a fixed-dose combination tablet of obicetrapib and ezetimibe. The company initiated TANDEM, a Phase 3 pivotal trial, to evaluate 10 mg obicetrapib and 10 mg ezetimibe as a fixed-dose combination used as an adjunct to diet and maximally tolerated lipid-lowering therapies to potentially enhance LDL-lowering in patients with HeFH, ASCVD, or ASCVD risk equivalents, in the first quarter of 2024 and released topline data in November 2024.
Based on the lipid-modifying effects of CETP inhibition observed in the company's clinical trials for obicetrapib to date, the company has conducted preclinical assessments of obicetrapib to test its potential for the prevention and treatment of Alzheimer’s disease. Following a Type B meeting in June 2021, the FDA confirmed that the company's preclinical data are sufficient to support a proposed clinical trial of obicetrapib for this indication, and the company commenced a Phase 2a clinical trial in early 2022 in patients with early Alzheimer’s disease to evaluate the pharmacodynamic and pharmacokinetic effects, safety, and tolerability of obicetrapib. The company announced initial data from this trial in September 2023.
Obicetrapib for Cardiovascular Disease
The CTT collaboration conducted a meta-analysis of 26 statin clinical trials and showed that there is a consistent, linear decrease in MACE for every absolute unit of non-HDL (which is primarily composed of LDL-C) cholesterol reduction. The MACE reduction observed in REVEAL falls on the meta-regression line – specifically, the CTT metaregression line predicts that an absolute reduction of non-HDL of 17 mg/dl, as seen in REVEAL, would correspond to the 11% reduction in coronary death and myocardial infarction observed in REVEAL.
With these learnings in mind, the company is executing a Phase 3 clinical development plan for obicetrapib focused on patients with elevated baseline LDL-C and designed to support a broad CVD label, if successful. To date, the company has completed seven Phase 1 trials, five Phase 2 trials, and three Phase 3 trials of obicetrapib, and it is currently conducting a Phase 3 PREVAIL CVOT trial.
Planned and Ongoing Clinical Trials for Cardiovascular Disease
Phase 3 PREVAIL Cardiovascular Outcomes Trial
The company has initiated its PREVAIL trial (TA-8995-304), a Phase 3 CVOT, to evaluate the effects of 10 mg obicetrapib in participants with ASCVD on MACE, including cardiovascular death, myocardial infarction, stroke, and non-elective coronary revascularization. The company enrolled over 9,500 participants at sites in the United States, Canada, Europe, Asia, and Australia with established ASCVD and an LDL-C level of at least 55 mg/dL, and an additional risk enhancer in participants with an LDL-C level below 100 mg/dL, whose LDL-C levels are not adequately controlled despite maximally tolerated lipid-modifying therapies.
The company designed its PREVAIL trial based on insights gained from analyzing failures of prior CVOTs for other CETP inhibitors. The trial design targets patients above their LDL-C risk-based goal, despite treatment with maximally tolerated lipid-modifying therapies, which the company believes creates potential for greater observed absolute LDL-C reduction, particularly given the observed median LDL-lowering activity of 51% in its Phase 2b ROSE clinical trial. The company is focused on patients with elevated LDL-C levels who have at least one other risk enhancer (including recent myocardial infarction, Type 2 diabetes, high triglyceride levels, or low HDL-C), compared to prior CVOTs for CETP inhibitors that enrolled patients with low baseline LDL-C. The company is planning for a longer duration of follow-up to maximize opportunities to observe MACE reduction, with all patients to be followed for a minimum of 2.5 years.
Phase 3 REMBRANDT Imaging Trial
The company has initiated REMBRANDT, a Phase 3 cardiovascular computed tomography angiography imaging trial to evaluate the effect of obicetrapib and ezetimibe in fixed-dose combination (FDC) on coronary plaque. The placebo-controlled, double-blind, randomized, Phase 3 study is being conducted in adult participants with high-risk ASCVD who are not adequately controlled by their maximally tolerated lipid-modifying therapy, to assess the impact of the obicetrapib 10 mg and ezetimibe 10 mg FDC daily on coronary plaque and inflammation characteristics.
Phase 2 VINCENT Trial
VINCENT is a Phase 2 clinical study to evaluate the effects of obicetrapib alone and in combination with evolocumab on Lp(a) in patients with mild dyslipidemia. The single arm study will treat patients with obicetrapib 10 mg daily for 8 weeks followed by obicetrapib 10 mg daily plus evolocumab 140 mg/dL every other week for 8 weeks.
Completed Phase 3 Clinical Trials
The company has completed three Phase 3 clinical trials of obicetrapib for the treatment of cardiometabolic disease. TANDEM evaluated 10 mg obicetrapib and 10 mg ezetimibe as a fixed-dose combination, while BROADWAY and BROOKLYN were designed to measure the effect of obicetrapib 10 mg as monotherapy on top of maximally tolerated lipid-modifying therapy.
Phase 3 TANDEM Fixed-Dose Combination Trial
The company completed TANDEM, a Phase 3 pivotal trial, to evaluate 10 mg obicetrapib and 10 mg ezetimibe as a fixed-dose combination used as an adjunct to diet and maximally tolerated lipid-lowering therapies to potentially enhance LDL-lowering in patients with HeFH, ASCVD or ASCVD risk equivalents in November 2024. The company enrolled approximately 400 patients in the United States that had a baseline LDL-C of greater than or equal to 70 mg/dL. Following a 14-day screening period, patients were randomized 1:1:1:1 to obicetrapib 10 mg and ezetimibe 10 mg FDC, obicetrapib 10 mg monotherapy, ezetimibe 10 mg monotherapy or placebo for an 84-day treatment period.
TANDEM’s co-primary endpoints were percent change from baseline in LDL-C of the fixed-dose combination compared to each monotherapy arm after 84 days and obicetrapib 10 mg compared to placebo after day 84. Secondary endpoints incorporated percent changes from baseline in other biomarkers, including Lp(a), non-HDL-C and ApoB. The TANDEM trial met all co-primary endpoints. The safety and tolerability profile of the fixed-dose combination was observed to be comparable to placebo in the trial.
Phase 3 BROADWAY and BROOKLYN Lipid Trials
The company conducted two Phase 3 pivotal trials designed to measure obicetrapib’s LDL-C lowering capability and enrolled patients across both trials who require additional LDL-lowering on top of their maximum tolerated lipid-modifying therapies. BROADWAY, which completed enrollment in July 2023 and for which topline results were announced in December 2024, randomized approximately 2,500 patients in the United States, Europe, and Asia with HeFH (individuals genetically predisposed to very high cholesterol) or established ASCVD, and who have a baseline LDL-C of at least 55 mg/dL, and an additional risk enhancer in participants with an LDL-C level below 100 mg/dL (including other abnormal biometrics, a recent myocardial infarction, or Type 2 diabetes). BROOKLYN, which completed enrollment in April 2023 and for which topline results were announced in July 2024, enrolled HeFH patients in the United States, Canada, Europe, and Africa who had a baseline LDL-C of at least 70 mg/dL.
Completed Phase 2 Clinical Trials
The company has completed five Phase 2 trials of obicetrapib for the treatment of cardiometabolic disease. In these Phase 2 trials, obicetrapib was observed to robustly lower LDL-C and increase HDL-C from baseline across various treatment settings. Obicetrapib was also observed to be well-tolerated compared to placebo, in both the 5 mg and 10 mg doses and as a combination therapy with ezetimibe. The majority of treatment-emergent adverse events (TEAEs) were mild or moderate in severity, and there were no drug-related, treatment-emergent serious adverse events (AEs).
Phase 2b ROSE Trial
In the company's Phase 2b ROSE trial, it was observed that obicetrapib has robust LDL-C lowering capability as an adjunct to high-intensity statins at both 5 mg and 10 mg dosages. Based on the ROSE trial findings, the company is using a 10 mg dosage for its Phase 3 trials. In its Phase 2a TULIP trial, it was observed that a daily dose of up to 10 mg of obicetrapib alone significantly reduced LDL-C and increased HDL-C.
Phase 2a TULIP Trial
A Phase 2a TULIP trial of obicetrapib, which was completed in 2014, was a randomized, double-blind placebo-controlled trial among 364 patients with mild dyslipidemia and not on lipid-altering therapy at screening and involved once-daily oral dosing of obicetrapib up to 10 mg or a placebo alone and as a combination therapy with statins. The primary endpoints were the percent changes in LDL-C and HDL-C levels from baseline to week 12 of the trial, which were met for both doses. The 5 mg dose of obicetrapib resulted in a mean reduction of LDL-C by 45% and increased HDL-C by 161%, compared to placebo. In patients treated with 10 mg obicetrapib plus statin therapy (20 mg atorvastatin or 10 mg rosuvastatin), LDL-C levels were approximately 50% lower and HDL-C levels were approximately 140% higher, respectively, than those observed in patients receiving statin therapy alone.
Phase 2b ROSE Trial
The company’s Phase 2b ROSE trial, which was completed in August 2021, was a randomized, double-blind placebo-controlled trial among 120 patients with mild dyslipidemia who were already receiving high-intensity statin therapy. The trial involved a once-daily oral dose of obicetrapib at either 5 mg or 10 mg dose level for eight weeks. The primary endpoint of this clinical trial was LDL-C reduction from baseline and was met for both doses. Obicetrapib had a rapid effect, with LDL-C levels dropping dramatically in the first four weeks of the trial and remaining relatively steady for the remaining four weeks of the trial.
Based on the company's ROSE trial and the enhanced LDL-C reduction capability of a 10 mg dose compared with 5 mg, along with the safety profile observed, the company selected a 10 mg dose for its Phase 3 lipid trials and CVOT.
Phase 2b OCEAN Trial
The company's Phase 2b OCEAN trial, which was completed in June 2021, evaluated the effect of obicetrapib as a combination therapy with ezetimibe on LDL-C levels. This randomized, double-blind placebo-controlled trial involved 100 patients with mild dyslipidemia and assessed a once-daily oral 5 mg dose of obicetrapib both alone and in combination with 10 mg of ezetimibe, compared to both placebo and ezetimibe alone, for eight weeks.
ROSE2 Clinical Trial
On June 3, 2023, the company announced full results from its Phase 2 ROSE2 trial, which evaluated obicetrapib in combination with ezetimibe as an adjunct to high-intensity statin therapy. ROSE2 met its primary and secondary endpoints, with statistically significant reductions in LDL-C and ApoB observed. Statistically significant improvements in non-HDL-C and total and small LDL-P were also observed. The company also noted significant improvements in Lp(a). In addition, the combination of obicetrapib and ezetimibe was observed to be well-tolerated, with a safety profile comparable to placebo.
ROSE2 was designed as a placebo-controlled, double-blind, randomized Phase 2 clinical trial to evaluate the efficacy, safety and tolerability of obicetrapib 10 mg in combination with ezetimibe 10 mg as an adjunct to high-intensity statin therapy. Patients were randomized to receive combination therapy, obicetrapib 10 mg or placebo for a 12 week treatment period. A total of 119 patients enrolled in ROSE2, of whom 97 were included in the on-treatment analysis. Certain patients were excluded from the on treatment population as a result of suspected non-adherence to the trial protocol. Patients presented at baseline with a fasting LDL-C greater than 70 mg/dL and triglycerides less than 400 mg/dL and all were receiving a stable dose of high-intensity statin therapy.
The primary endpoint was the percent change from baseline to week 12 in Friedewald-calculated LDL-C for the obicetrapib plus ezetimibe combination treatment group compared with placebo. Secondary efficacy endpoints included the percent changes from baseline to week 12 in LDL-C for obicetrapib monotherapy compared with placebo and in ApoB for the obicetrapib plus ezetimibe combination compared with placebo and the obicetrapib monotherapy compared with placebo.
Japan Phase 2b Clinical Trial
On June 5, 2023, the company announced topline results from its Phase 2b Japan trial evaluating the effects of three doses of obicetrapib (2.5 mg, 5 mg, and 10 mg) on LDL-C levels. This was a randomized, double-blind, placebo controlled trial designed to evaluate the efficacy, safety and tolerability of obicetrapib as an adjunct to stable statin therapy in Japanese patients. The trial was conducted at hospitals and clinics across Japan. The primary endpoint was the percent change from baseline to end of treatment (day 56) in LDL-C for each obicetrapib group compared to placebo.
Obicetrapib for Other Therapeutic Areas
The company commenced a Phase 2a open-label and single-arm clinical trial in early 2022 in patients with early Alzheimer’s disease and the ApoE4 mutation to evaluate the pharmacodynamic and pharmacokinetic effects, safety, and tolerability of obicetrapib.
Marketing and Sales
The company entered into the Menarini License, pursuant to which Menarini has been granted the exclusive rights to commercialize obicetrapib 10 mg either as a sole active ingredient product or in a fixed-dose combination with ezetimibe in the majority of European countries, if approved. As any future product candidates near regulatory approval and potential commercial launch, the company plans to assess its options for commercializing each respective product candidate and may choose to commercialize them either independently or with a partner.
Menarini License
The company entered into the Menarini License, pursuant to which it granted Menarini an exclusive, royalty-bearing, sublicensable license under certain intellectual property and regulatory documentation to undertake post-approval development activities and commercialize multiple brands of obicetrapib in a single unit dose of 10 mg or less, either as a sole active ingredient product or in a fixed-dose combination with ezetimibe (the Licensed Products), for any use in the majority of European countries (the Menarini Territory). The company retained all rights to obicetrapib in all other territories and in other dosages.
Intellectual Property
All of the issued patents and pending patent applications in the company's patent portfolio are owned by its subsidiary, NewAmsterdam Pharma B.V., Dutch Chamber of Commerce registry number 55971946. As of December 31, 2024, the company owned 10 issued U.S. patents and 17 pending U.S. patent applications. The company also owned 132 granted European patents and five pending European patent applications, two granted Chinese patents, and 12 pending Chinese patent applications. In addition, the company owned 77 granted patents and 70 pending patent applications in other foreign jurisdictions, including international applications under the PCT.
Obicetrapib First Generation Patents
The company has two granted patents in the United States covering a genus of compounds that includes obicetrapib and claims that more narrowly cover the obicetrapib compound, pharmaceutical compositions, and methods of treatment. In Europe, the company has 17 granted patents. In Asia, the company has one granted patent in China, two granted patents in Japan, one granted patent in the Republic of Korea, one granted patent in Taiwan, and one granted patent in Singapore. The company also has one granted patent in India. In North America outside of the United States, the company has one granted patent in Canada and one granted patent in Mexico. Additionally, the company has 13 granted patents in other foreign jurisdictions. These patents are expected to expire between April 2025 and August 2027, without taking potential patent term extensions into account. The first-generation portfolio also includes a patent family covering a method of synthesizing obicetrapib. The company has one patent in the United States, five patents in Europe, including the United Kingdom, and one patent in Japan in this latter patent family. Patents in this family are expected to expire between March 29, 2027, and March 31, 2029, not including patent term extensions.
Obicetrapib Second Generation Patents
The company's second-generation obicetrapib patent portfolio includes a patent family directed to solid oral dosage forms containing 5 to 10 mg of obicetrapib, including tablet forms, and methods of treatment comprising administration of 1 to 25 mg of obicetrapib daily. The company has four granted patents in the United States and a pending application. The company has 39 granted patents in Europe. In Asia, the company has no granted patents in China, one granted patent in the Republic of Korea, one granted patent in Japan, one granted patent in Taiwan, one granted patent in Singapore, and one granted patent in Hong Kong. The company has one granted patent in India. In North America outside of the United States, the company has one granted patent in Mexico and one granted patent in Canada. Additionally, the company has 15 granted patents in other foreign jurisdictions. Patent applications are pending in Argentina, Brazil, China, Hong Kong, Colombia, Costa Rica, Egypt, Libya, Peru, Thailand, and Venezuela. Patents and patent applications, if granted, are expected to expire in February 2034, without taking potential patent term extensions or patent term adjustments into account.
The company also has a patent family directed to compositions that contain obicetrapib and a statin, methods of treating with compositions that contain obicetrapib and a statin, and in various foreign jurisdictions, methods of use in which obicetrapib and a statin are separately administered. The company has one granted patent in the United States, which is expected to expire in February 2034. The company has no granted patents in Europe. In Asia, the company has no granted patents in China, two granted patents in Japan, one granted patent in the Republic of Korea, and two granted patents in Taiwan. In North America outside of the United States, the company has one granted patent in Mexico and one granted patent in Canada. Additionally, the company has two granted patents in other foreign jurisdictions. Patent applications are pending in China, Hong Kong, Thailand, and Venezuela. Outside the United States, patents and patent applications, if granted, are expected to expire in August 2035, without taking potential patent term extensions or patent term adjustments into account.
The company has a patent family that claims a synthetic intermediate used in a synthetic process it intends to use commercially, as well as processes to make that intermediate. The company has one issued U.S. patent and 39 granted patents in Europe. In Asia, the company has one granted patent in China, one granted patent in Hong Kong, one granted patent in Japan, one granted patent in Singapore, one granted patent in the Republic of Korea, and one granted patent in Taiwan. The company also has one granted patent in India. In North America outside of the United States, the company has one granted patent in Mexico and one granted patent in Canada. Additionally, the company has 15 granted patents in other foreign jurisdictions. Patent applications are pending in Europe, Hong Kong, and Venezuela. Patents and patent applications, if granted, are expected to expire in July 2035, without taking potential patent term extensions or patent term adjustments into account.
Obicetrapib Third Generation Patents
The company has a third-generation patent family covering the solid salt form of obicetrapib that it intends to commercialize, a process for its commercial synthesis, and a novel intermediate used in that synthetic process. In the United States, the company has an issued patent with claims covering the solid salt form of obicetrapib, along with a pending application. Patent applications are pending worldwide in over 40 jurisdictions. The company's U.S. patent, and any patents granted on the pending applications, are expected to expire in July 2043, without taking potential patent term adjustments or extensions into account.
The company also has patent families directed to various compositions and methods of use of obicetrapib as a combination therapy. These families consist entirely of pending applications. Seven of these families are directed to combinations with ezetimibe, several of which cover the formulation of the company's intended commercial fixed-dose combination product. Two of these families further include the commercial formulation of the intended obicetrapib single active product. Patents, if granted, are expected to expire between February 2042 and November 2045, without taking potential patent term adjustments or extensions into account. Another family is directed to a combination with statins, for use in certain subpopulations, with patents, if granted, expected to expire in July 2042, without considering potential patent term adjustments or extensions. The company also has two families directed to combinations with SGLT2 inhibitors. If granted, these patents are expected to expire in December 2042 and April 2044, without taking potential patent term adjustments or extensions into account.
The company has an additional patent family directed to a new process for the synthesis of an intermediate in the manufacture of obicetrapib. This family currently consists of a U.S. provisional application. Any patents, if granted, are expected to expire around November 2045.
The company has two patent families covering methods of using obicetrapib to treat neurodegenerative diseases. The first of these families includes 31 granted patents in Europe, one granted patent in Hong Kong, and one granted patent in Israel. The company also has patent applications pending in this family in the United States, Europe, China, and other jurisdictions. The granted patents, as well as patent applications if granted, are expected to expire in March 2042, without taking potential patent term adjustments or extensions into account. The second family consists of a PCT application and a pending U.S. provisional application. Any patents that grant from this second family are expected to expire in September 2044, without considering potential patent term adjustments or patent term extensions.
Finally, the company has three patent families drawn to the treatment of other clinical indications. Patents, if granted, from these families are expected to expire in approximately October 2044, without taking potential patent term adjustments or patent term extensions into account.
Research and Development
The company's research and developments expenses were $151.4 million for the year ended December 31, 2024.
Government Regulation and Product Approval
Drug manufacturers and their subcontractors involved in the manufacture and distribution of approved drugs are required to register their establishments with the FDA and certain state agencies. They are also subject to periodic unannounced inspections by the FDA and certain state agencies for compliance with current Good Manufacturing Practices (cGMP), which impose certain procedural and documentation requirements upon the company and its third-party manufacturers.
Moreover, as a condition of participating in, and having products covered under, certain federal healthcare programs, such as Medicare and Medicaid, the company is subject to federal laws and regulations that require pharmaceutical manufacturers to calculate and report certain price reporting metrics to the government. These metrics include Medicaid Average Manufacturer Price (AMP), Best Price, Medicare Average Sales Price, the 340B Ceiling Price, and Non-Federal AMP reported to the Department of Veterans Affairs. With respect to Medicaid, the company is also required to pay statutory rebates on utilization of its products by Medicaid beneficiaries.
In the United States, numerous federal and state laws and regulations, including data breach notification laws, health information privacy and security laws such as The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), and federal and state consumer protection laws and regulations (e.g., Section 5 of the Federal Trade Commission Act), govern the collection, use, disclosure, and protection of health-related and other personal information. These laws could apply to the company's operations or the operations of its partners.
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