Latest news with #HoFH
Yahoo
14-07-2025
- Health
- Yahoo
Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)
First and only medicine approved by Health Canada for patients as young as 6-months of age with HoFH, an ultrarare, inherited form of high cholesterol TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor initially received Health Canada approval in September 2023 as an adjunct to diet and other LDL-C lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with HoFH, a disease associated with dangerously high levels of LDL-C. "Early detection and prompt initiation of effective treatment are essential to prevent the devastating consequences of very high cholesterol that begins at a very young age for patients with HoFH. However, conventional treatment options are challenging for infants and children,' stated Dr. Brian McCrindle, a pediatric lipid specialist at The Hospital for Sick Children, University of Toronto. 'This extended approval will enable the very early and effective lowering of lipid levels essential for these vulnerable patients." The pharmacokinetics and efficacy of the drug in pediatric patients aged 6 months to less than 5 years with HoFH have been predicted from a model-based extrapolation analysis. Results of these analyses show that pediatric patients aged 6 months to less than 5 years are predicted to experience a similar or higher magnitude of percent change in LDL-C at week 24 compared to older pediatric patients and adults, when receiving a 15 mg/kg dose every 4 weeks. In addition, supportive data for five patients who initiated treatment between 1 and 4 years of age via compassionate use showed a clinically meaningful reduction of LDL-C consistent with that observed in patients aged 5 years or older in clinical studies. Based on the currently available data, the safety profile in pediatric patients aged 6-months to 5 years old is expected to be similar to the safety profile in older pediatric patients. No new safety concerns have been identified in the compassionate use program. "HoFH is a severe, life-threatening condition that, if left untreated, can lead to heart disease and death as early as childhood. This makes diagnosing it and reducing the resulting high LDL-C levels an urgent need,' said Monty Keast, Vice President and General Manager at Ultragenyx Canada. 'We are proud to be able to provide children as young as 6-months old living with HoFH a medicine that could reduce LDL-C levels and will work collaboratively with healthcare providers and payers across the country to ensure it is accessible for families.' Evkeeza is reimbursed and commercially available to prescribe for appropriate patients with HoFH in Canada via private drug plans and through the public drug program in Quebec, the UK, U.S., Italy, Japan, the Netherlands, Spain and Luxembourg. It is also available via early access programs in 13 additional countries including Austria and France. About Homozygous Familial Hypercholesterolemia (HoFH)HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and 1 in 275,000 in the French Canadian population. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>10 mmol/L) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age. For more information about HoFH please go to About Evkeeza (evinacumab)Evinacumab, the active substance in the medicine, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. The medicine is delivered via an infusion every month (4 weeks). Regeneron Pharmaceuticals, Inc. discovered and developed Evkeeza, and commercializes the product in HoFH in the U.S. under the generic name evinacumab-dgnb, with dgnb as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Ultragenyx is responsible for development and commercialization efforts in countries outside of the U.S. What is Evkeeza used for? Evkeeza is used to treat adults and children aged 6 months and older with very high cholesterol caused by a condition called homozygous familial hypercholesterolemia (HoFH). Evkeeza is used with a low-fat diet and other medicines to bring down cholesterol levels. Do not use Evkeeza if: • Allergic to evinacumab or to any of the ingredients in this medicine. To help avoid side effects and ensure proper use, a healthcare professional should be consulted before taking Evkeeza. Talk about any health conditions or problems, including: • Allergic reactions (hypersensitivity), including a severe reaction known as anaphylaxis. Symptoms may include swelling of the lips, tongue, or throat that make it difficult to swallow or breathe, and may also include wheezing, feeling dizzy, or fainting. If any of these symptoms are noticed, stop taking Evkeeza and get immediate medical help. • If pregnant, might be pregnant, or plan to become pregnant, ask a healthcare professional for advice before taking Evkeeza. Evkeeza may harm an unborn baby. Tell a healthcare professional if become pregnant while using Evkeeza. • If breastfeeding or plan to breastfeed, ask a healthcare professional for advice before being given Evkeeza. It is not known if Evkeeza passes into breast milk. Tell a healthcare professional about all the medicines taken, including any drugs, vitamins, minerals, natural supplements, or alternative medicines. What are the possible side effects from using Evkeeza? These are not all the possible side effects when taking Evkeeza. If any side effects not listed here are experienced, tell a healthcare professional. • Abdominal pain • Back pain • Constipation • Decreased energy • Dizziness • Flu symptoms • Itchiness at the site of the injection • Nausea • Pain in legs or arms • Runny nose • Fatigue (for patients aged 5-11 years old) • Sore throat or sinus infection Reporting Side Effects: Any suspected side effects associated with the use of health products can be reported to Health Canada by: • Visiting the Web page on Adverse Reaction Reporting ( for information on how to report online, by mail or by fax; or • Calling toll-free at 1-866-234-2345. NOTE: Contact a health professional if information is needed about how to manage side effects. The Canada Vigilance Program does not provide medical advice. Please see PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION for more information. About Ultragenyx Pharmaceutical is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Ultragenyx Forward-Looking Statements and Use of Digital MediaExcept for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company's clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, risks related to reliance on third party partners to conduct certain activities on the Company's behalf; the potential for any license or collaboration agreement, including Ultragenyx's collaboration agreement with Regeneron to commercialize Evkeeza outside of the United States, to be terminated; uncertainty and potential delays related to clinical drug development; uncertainties and unpredictability of obtaining regulatory approval for the Company's product candidates and the scope of such potential regulatory approval; smaller than anticipated market opportunities for the Company's products and product candidates; fluctuations in buying or distribution patterns by distributors and specialty pharmacies; competition to the Company's products and product candidates; potential undesirable or serious side effects from the Company's products or product candidates; the Company's ability to effectively manage the expansion of its commercial organization; market acceptance of the Company's current or future products; uncertainties related to insurance coverage and reimbursement status of newly approved products; manufacturing risks and supply chain disruptions; competition from other therapies or products; and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the Company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website ( and LinkedIn website ( ContactsUltragenyx Pharmaceutical Higa+1-415-475-6370ir@ MediaJoey Fleury+1-925-784-5829media@
Business Wire
08-07-2025
- Business
- Business Wire
Arrowhead Pharmaceuticals Initiates Phase 3 YOSEMITE Study of Investigational Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease. Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies, after investigational drugs plozasiran, fazirsiran (licensed to Takeda) and olpasiran (licensed to Amgen). As an RNAi-based therapeutic targeting ANGPTL3, investigational zodasiran has the potential to treat HoFH in a fundamentally different manner from traditional LDL-C–lowering therapies. Share 'Patients living with HoFH are difficult to adequately treat and have a very high risk of developing atherosclerotic cardiovascular disease due to severely elevated LDL-C, often exceeding 500 mg/dL. As an RNAi-based therapeutic targeting ANGPTL3, investigational zodasiran has the potential to treat HoFH in a fundamentally different manner from traditional LDL-C–lowering therapies,' said James Hamilton, M.D., Chief Medical Officer and head of R&D at Arrowhead. 'In Phase 2 clinical studies, patients with HoFH receiving zodasiran achieved reductions from baseline in LDL-C, ApoB, non-HDL-C, and triglycerides, supporting its potential therapeutic role for the treatment of HoFH patients.' About Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia (HoFH) is an ultra-rare treatment‐resistant genetic condition characterized by elevated low density lipoprotein cholesterol (LDL-C) and early-onset cardiovascular disease. Most cases of HoFH are due to mutations in the low-density lipoprotein receptor gene (LDLR) coding for the LDL receptor (LDLR). Thus, HoFH represents a unique case where LDL-C lowering therapies not requiring functional LDL receptors may have benefit. If left untreated, individuals with HoFH can have median LDL-C levels above 500 mg/dL (13 mmol/L), leading to early clinical manifestations of coronary artery disease 1. Patients with HoFH may also have cholesterol deposits under the skin (xanthomas), around the eyes (xanthelasmas), or around the cornea (corneal arcus), but physical signs are not always present, particularly in children. HoFH remains challenging to treat and currently only patients with the more severe HoFH phenotypes get diagnosed and treated early. The estimated prevalence of HoFH globally is between 1:360,000 and 1:250,000 1. About YOSEMITE Phase 3 Study YOSEMITE (NCT07037771) is a Phase 3 multicenter, randomized, placebo-controlled study to evaluate the efficacy and safety of zodasiran in adolescent and adult patients with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) on maximally tolerated lipid lowering therapy. Approximately 60 subjects over the age of 12 will be randomized (2:1) to receive 4 doses (once every 3 months) of 200 mg zodasiran or placebo. The primary endpoint is the percent change from baseline to month 12 in fasting LDL-C. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension. About Zodasiran Zodasiran, previously called ARO-ANG3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of angiopoietin-like protein (ANGPTL3), which is a hepatocyte expressed regulator of lipid and lipoprotein metabolism with multiple potential modes of action, including inhibition of lipoprotein lipase (LPL) and endothelial lipase (EL) 5,6. ANGPTL3 is an emerging therapeutic target with relevance to hypercholesterolemia, hypertriglyceridemia, and mixed hyperlipidemia. Genetic studies suggest that individuals with ANGPTL3 loss-of-function variants have enhanced lipoprotein lipase and endothelial lipase activity, resulting in lower levels of atherogenic lipoproteins and a reduced risk of ASCVD 2-4. In prior clinical studies, investigational zodasiran was associated with dose-dependent reductions in triglycerides, triglyceride rich lipoprotein remnants, and total atherogenic lipoproteins, including LDL-C, in patients with homozygous (HoFH) and heterozygous (HeFH) familial hypercholesterolemia and mixed hyperlipidemia. Zodasiran also showed a favorable safety profile. In the Phase 2 GATEWAY study in patients with HoFH, there were no drug discontinuations, drug-related serious adverse events, or deaths. The most frequent adverse events were COVID-19, nasopharyngitis, upper respiratory tract infection, and dizziness. About Arrowhead Pharmaceuticals, Inc. Arrowhead Pharmaceuticals, Inc. develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company's email list and receive news directly, please visit Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as 'may,' 'might,' 'will,' 'expect,' 'believe,' 'anticipate,' 'goal,' 'endeavor,' 'strive,' 'hope,' 'intend,' 'plan,' 'project,' 'could,' 'estimate,' 'potential,' 'target,' 'forecast' or 'continue' or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding regulatory approval for and commercial launch of plozasiran; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc. ________________ 1. Cuchel, et al. Eur Heart J. 2023;44(25):2277-91 2. Dewey, et al. N Engl J Med. 2017;377 (3):211-21. 3. Minicocci, et al. J Lipid Res. 2013;54(12): 3481-90 4. Musunuru, et al. N Engl J Med. 2010; 363(23):2220-7 5. Adam, et al. J Lipid Res. 2020;61(9): 1271-86. 6. Rosenson. J Lipid Res. 2021:62:100060.
National Post
27-06-2025
- Health
- National Post
PRD Therapeutics Announces Initiation of First-in-Human Study for PRD001
Article content TOKYO — PRD Therapeutics, Inc., a clinical stage company focused on the development of novel lipid metabolism regulators targeting homozygous familial hypercholesterolemia (HoFH) and metabolic dysfunction associated fatty liver disease (MASH/MASLD), today announced that the company recently initiated dosing in a First-in-Human (FIH) clinical trial of PRD001, a first-in-class SOAT2 (formerly known as ACAT2) selective inhibitor. Article content 'We are excited to initiate dosing in this clinical trial of PRD001. Many clinical trials have been conducted on SOAT1/2 dual or SOAT1 selective inhibitors, but this is the first clinical trial of an SOAT2 selective inhibitor' said Kanji Hosoda, Ph.D., CEO and co-founder of PRD Therapeutics. 'Several results with SOAT1 or 2 knockout mice have been published, suggesting that knocking out or inhibiting only SOAT2 is crucial to demonstrate safety and efficacy. PRD001 is the world's first and only SOAT2-selective inhibitor and is expected to exhibit safety and efficacy in humans as well. Our preclinical animal models (LDL-R KO mice; HoFH model, and high-fat diets induced MASH model mice) have shown that PRD001 lowers blood and liver lipids and suppresses the progression of fatty liver and atherosclerosis with no adverse events. PRD001 has the potential to be the first-in-class effective and safe oral therapy for HoFH patients suffering from no or extremely low LDL receptor activity.' Article content This FIH Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and the signs of early efficacy (LDL-C lowing effect, and liver fat quantitative value using MRI-PDFF) of PRD001 in adult healthy volunteers. More information can be found at NCT07034183. Article content PRD001 is a first-in-class, orally available small molecule SOAT2-selective inhibitor. It uniquely controls three key pathways of lipid metabolism; cholesterol synthesis in the liver, cholesterol absorption in the small intestine, and uptake of blood LDL-C with a single drug, leading to a potent reduction in a blood LDL-C level independent of the LDL receptor. Article content This research and development is supported by Japan Agency for Medical Research and Development (AMED) under Strengthening Program for Pharmaceutical Startup Ecosystem (Project title: ' Development of First-in-class oral lipid metabolism regulator PRD001 and POC obtained for lipid metabolism disorder '). Article content Article content Article content Article content Article content
Business Wire
27-06-2025
- Health
- Business Wire
PRD Therapeutics Announces Initiation of First-in-Human Study for PRD001
TOKYO--(BUSINESS WIRE)--PRD Therapeutics, Inc., a clinical stage company focused on the development of novel lipid metabolism regulators targeting homozygous familial hypercholesterolemia (HoFH) and metabolic dysfunction associated fatty liver disease (MASH/MASLD), today announced that the company recently initiated dosing in a First-in-Human (FIH) clinical trial of PRD001, a first-in-class SOAT2 (formerly known as ACAT2) selective inhibitor. 'We are excited to initiate dosing in this clinical trial of PRD001. Many clinical trials have been conducted on SOAT1/2 dual or SOAT1 selective inhibitors, but this is the first clinical trial of an SOAT2 selective inhibitor' said Kanji Hosoda, Ph.D., CEO and co-founder of PRD Therapeutics. 'Several results with SOAT1 or 2 knockout mice have been published, suggesting that knocking out or inhibiting only SOAT2 is crucial to demonstrate safety and efficacy. PRD001 is the world's first and only SOAT2-selective inhibitor and is expected to exhibit safety and efficacy in humans as well. Our preclinical animal models (LDL-R KO mice; HoFH model, and high-fat diets induced MASH model mice) have shown that PRD001 lowers blood and liver lipids and suppresses the progression of fatty liver and atherosclerosis with no adverse events. PRD001 has the potential to be the first-in-class effective and safe oral therapy for HoFH patients suffering from no or extremely low LDL receptor activity.' This FIH Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and the signs of early efficacy (LDL-C lowing effect, and liver fat quantitative value using MRI-PDFF) of PRD001 in adult healthy volunteers. More information can be found at NCT07034183. About PRD001 PRD001 is a first-in-class, orally available small molecule SOAT2-selective inhibitor. It uniquely controls three key pathways of lipid metabolism; cholesterol synthesis in the liver, cholesterol absorption in the small intestine, and uptake of blood LDL-C with a single drug, leading to a potent reduction in a blood LDL-C level independent of the LDL receptor. This research and development is supported by Japan Agency for Medical Research and Development (AMED) under Strengthening Program for Pharmaceutical Startup Ecosystem (Project title: " Development of First-in-class oral lipid metabolism regulator PRD001 and POC obtained for lipid metabolism disorder ").
Yahoo
20-03-2025
- Business
- Yahoo
Esperion Aligns with U.S. Food and Drug Administration to Initiate Phase 3 Clinical Trials of Bempedoic Acid in Pediatric Heterozygous and Homozygous Familial Hypercholesterolemia
– Confirms Sufficient Data to Complete Phase 2 Clinical Study Enrollment and Advance to Phase 3 Studies in Both Heterozygous and Homozygous Familial Hypercholesterolemia – – Establishes Pediatric Path Forward to Start and Complete Phase 3 Trial and Secure Additional Six-Month Patent Extension Through June 2031 – ANN ARBOR, Mich., March 20, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that following meetings with the U.S. Food and Drug Administration (FDA), it has gained alignment on a regulatory path forward for initiating Phase 3 studies of bempedoic acid alone and in combination with ezetimibe in pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH, respectively). Based on these discussions with the FDA, the Company plans to initiate Phase 3 clinical studies this year. The FDA previously granted orphan drug designation for bempedoic acid in HoFH indication. 'We are delighted to further advance the development of bempedoic acid for children with familial hypercholesterolemia (FH) and are pleased the FDA has indicated that we have adequate data to proceed into Phase 3 clinical trials,' stated Sheldon Koenig, President and CEO of Esperion. 'In addition, this alignment supports our commitment to broaden the reach of our bempedoic acid products as part of our lifecycle management plan, providing the opportunity to extend our patent protection for an additional six months for these important therapies,' added Sheldon Koenig. About CLEAR Path 2 and CLEAR Path 3 StudiesCLEAR Path 2 (in children with HeFH) and CLEAR Path 3 (in children with HoFH) are Phase 3, randomized double-blind, placebo-controlled, multicenter studies to evaluate the efficacy and safety of bempedoic acid with and without concurrent ezetimibe in children with HeFH or HoFH and LDL-C ≥130 mg/dL (3.4 mmol/L) while on protocol defined optimum dose of a statin. Each study is a 52-week design where patients will be randomized 2:1 to treatment or control for 24 weeks, followed by 28 weeks of an open-label extension period. About Heterozygous Familial Hypercholesterolemia (HeFH) and Homozygous FH (HoFH)HeFH and HoFH are the two types of FH, a genetic condition that causes high cholesterol from birth and if untreated leads to early, aggressive atherosclerotic cardiovascular disease. HeFH is the more common form occurring in 1 in 250 births and results from inheriting a gene that causes FH from one parent. If left untreated, cardiovascular disease can develop during middle adulthood. HoFH is the rarer form of the disease, an orphan indication, occurring in 1 in 300,000 births and results from inheriting a FH gene from both parents. HoFH results in higher cholesterol levels, which are often more difficult to treat as cardiovascular disease can begin as early as in childhood if untreated. In both HeFH and HoFH, the processing of LDL-C is disrupted due to these gene mutations, which leads to dangerously high levels of LDL-C. Both HeFH and HoFH are underdiagnosed and undertreated but with early detection and continued development of LDL-C therapies for patients starting at an early age, there is hope that patients with FH can control their LDL cholesterol and reduce their risk of cardiovascular disease. About Esperion TherapeuticsEsperion Therapeutics, Inc. is a commercial stage biopharmaceutical company focused on bringing new medicines to market that address unmet needs of patients and healthcare professionals. The Company developed and is commercializing the only U.S. Food and Drug Administration (FDA) approved oral, once-daily, non-statin medicines for patients who are at risk for cardiovascular disease and are struggling with elevated low density lipoprotein cholesterol (LDL-C). These medications are supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial. Esperion continues to build on its success with its next generation program which is focused on developing ATP citrate lyase inhibitors (ACLYi). New insights into the structure and function of ACLYi fully enables rational drug design and the opportunity to develop highly potent and specific inhibitors with allosteric mechanisms. Esperion continues to evolve into a leading global biopharmaceutical company through commercial execution, international partnerships and collaborations and advancement of its pre-clinical pipeline. For more information, visit and follow Esperion on LinkedIn and X. Forward-Looking StatementsThis press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding marketing strategy and commercialization plans, current and planned operational expenses, future operations, commercial products, clinical development, including the timing, designs and plans for the CLEAR Outcomes study and its results, plans for potential future product candidates, financial condition and outlook, including expected cash runway, and other statements containing the words 'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'predict,' 'project,' 'suggest,' 'target,' 'potential,' 'will,' 'would,' 'could,' 'should,' 'continue,' and similar expressions. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements involve risks and uncertainties that could cause Esperion's actual results to differ significantly from those projected, including, without limitation, the net sales, profitability, and growth of Esperion's commercial products, clinical activities and results, supply chain, commercial development and launch plans, the outcomes and anticipated benefits of legal proceedings and settlements, and the risks detailed in Esperion's filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Esperion disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. Esperion Contact Information:Investors: Alina Veneziainvestorrelations@ (734) 887-3903 Media: Tiffany Aldrich corporateteam@ (616) 443-8438
