Vadadustat

Akebia to host conference call at 8:00 a.m. ET on May 9 Vafseo® (vadadustat) tablets FDA approved on March 27, 2024 Vafseo launch activities underway with availability expected in January 2025 Auryxia® (ferric citrate) net product revenues were $31.0 million for the first quarter 2024, Akebia expects 2024 Auryxia net product revenue growth versus 2023 CAMBRIDGE, Mass., May 9, 2024 /PRNewswire/ -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today reported financial results for the first quarter ended March 31, 2024. Akebia is launching Vafseo® (vadadustat) tablets, recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months, which will be available in January 2025. "The recent FDA approval of Vafseo represents a transformational milestone for Akebia as we initiate launch activities that we believe will enable widespread access and rapid adoption when Vafseo is available in January 2025," said John P. Butler, Chief Executive Officer of Akebia. "Upon FDA approval of Vafseo, we initiated work to drive demand from potential prescribers and to contract with dialysis providers. We are progressing plans for generating clinical data to identify additional areas of potential benefit to patients as we remain committed to the kidney community. We also expect to engage with the FDA on label expansion opportunities for Vafseo by the end of this year." Recent Business Highlights Akebia plans to submit its application for Transitional Drug Add-on Payment Adjustment (TDAPA) for Vafseo in June. Akebia expects the application to be accepted in July 2024 and expects TDAPA designation in January 2025. In March, Akebia presented posters at the 2024 Annual Dialysis Conference on the cardiovascular safety of vadadustat in patients new to dialysis with CKD-related anemia and the safety and efficacy of vadadustat in the treatment of anemia in U.S. patients with CKD. In April, Akebia's licensee Averoa, a biopharmaceutical company, submitted a marketing authorization application to the European Medicines Agency for Ferric Citrate Coordination Complex, and, if approved, Averoa will make the product available to patients in Europe. In April, Akebia drew down the second tranche of its $55.0 million BlackRock debt facility and received net proceeds of $7.5 million. Ten million dollars of borrowing capacity remains available under the debt facility until December 31, 2024, subject to the conditions in the loan agreement. In May, Akebia signed an amendment to its License Agreement with Vifor International Ltd. (Vifor) to modify the method of repayment of its working capital fund through tiered royalties based upon Akebia's sales of Vafseo, significantly simplifying the repayment terms. Akebia reported first quarter 2024 Auryxia® (ferric citrate) net product revenues of $31.0 million. Akebia reaffirms that it expects Auryxia net product revenue growth in 2024 versus 2023. The Centers for Medicare and Medicaid Services released guidance on incorporating phosphate binders, including Auryxia, into the dialysis bundle in January 2025. Akebia is accelerating contracting discussions with dialysis organizations with a goal to ensure broad access to Auryxia. "With continued revenue contributions from Auryxia, revenue from Vafseo beginning in 2025, and our current cash balance, we believe we have sufficient cash to support operations for at least the next two years while investing in the Vafseo launch," Mr. Butler added. Financial Results Revenues: Total revenues were $32.6 million for the first quarter of 2024 compared to $40.0 million for the first quarter of 2023. Net product revenues were $31.0 million for the first quarter of 2024 compared to $34.7 million for the first quarter of 2023. License, collaboration and other revenues were $1.6 million for the first quarter of 2024 compared to $5.3 million for the first quarter of 2023. COGS: Cost of goods sold was $11.6 million for the first quarter of 2024 compared to $20.2 million for the first quarter of 2023. Akebia continues to carry a non-cash intangible amortization charge of $9.0 million per quarter through the fourth quarter of 2024. R&D Expenses: Research and development expenses were $9.7 million for the first quarter of 2024 compared to $19.7 million for the first quarter of 2023. SG&A Expenses: Selling, general and administrative expenses were $25.4 million for the first quarter of 2024 compared to $25.1 million for the first quarter of 2023. Net Income / Loss: Net loss was $18.0 million for the first quarter of 2024 compared to a net loss of $26.9 million for the first quarter of 2023. Cash Position: Cash and cash equivalents as of March 31, 2024, were approximately $42.0 million. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including a U.S. Vafseo launch, for at least the next two years. Conference Call Akebia will host a conference call on Thursday, May 9 at 8:00 a.m. Eastern Time to discuss first quarter 2024 earnings. To access the call, please register by clicking on this Registration Link, and you will be provided with dial in details. To avoid delays and ensure timely connection, we encourage dialing into the conference call 15 minutes ahead of the scheduled start time. A live webcast of the conference call will be available via the "Investors" section of Akebia's website at: . An online archive of the webcast can be accessed via the Investors section of Akebia's website at approximately two hours after the event. About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at , which does not form a part of this release. About Vafseo® (vadadustat) tablets Vafseo® (vadadustat) tablets is a once-daily oral hypoxia-inducible factor prolyl hydroxylase inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin, increasing hemoglobin and red blood cell production to manage anemia. Vafseo is approved for use in 37 countries. INDICATION VAFSEO is indicated for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Limitations of Use VAFSEO has not been shown to improve quality of life, fatigue, or patient well-being. VAFSEO is not indicated for use: As a substitute for red blood cell transfusions in patients who require immediate correction of anemia. In patients with anemia due to CKD not on dialysis. IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat) tablets CONTRAINDICATIONS Known hypersensitivity to VAFSEO or any of its components Uncontrolled hypertension WARNINGS AND PRECAUTIONS Increased Risk of Death, Myocardial Infarction, Stroke, Venous Thromboembolism, and Thrombosis of Vascular Access A rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can increase these risks. Avoid use in patients with a history of myocardial infarction, cerebrovascular event, or acute coronary syndrome within the 3 months prior to starting VAFSEO. Targeting a Hb level of greater than 11g/dL is expected to further increase the risk of death and arterial and venous thrombotic events, as occurs with ESAs, which also increase erythropoietin levels. No specific Hb target level, dose of VAFSEO, or dosing strategy has been identified to avoid these risks. Use the lowest effective dose and adhere to dosing and Hb monitoring recommendations to avoid excessive erythropoiesis. Advise patients to seek immediate medical attention if they develop signs or symptoms of myocardial infarction, stroke, venous thromboembolism, or thrombosis of vascular access. Evaluate and manage promptly if these occur. Hepatotoxicity Hepatocellular injury attributed to VAFSEO was reported in less than 1% of patients, including one severe case with jaundice. All events were asymptomatic and resolved after discontinuation of VAFSEO. The time to onset was generally within the first 3 months of treatment. Elevated serum ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and 0.3% of CKD patients treated with VAFSEO, respectively. Measure ALT, AST, and bilirubin before treatment and monthly for the first 6 months, then as clinically indicated. Discontinue VAFSEO if ALT or AST is persistently elevated or accompanied by elevated bilirubin. Not recommended in patients with cirrhosis or active, acute liver disease. Hypertension Worsening of hypertension was reported in 14% (9.4 per 100 person-years [PY]) of patients receiving VAFSEO and 17% (11.8 per 100 PY) of patients receiving darbepoetin alfa. Serious worsening of hypertension was reported in 2.7% (1.7 per 100 PY) of patients receiving VAFSEO and 3% (1.8 per 100 PY) of patients receiving darbepoetin alfa. Cases of hypertensive crisis including hypertensive encephalopathy and seizures have also been reported in patients receiving VAFSEO. Monitor blood pressure. Adjust anti-hypertensive therapy as needed. Seizures Seizures occurred in 1.6% (1.0 per 100 PY) of patients who received VAFSEO and 1.6% (1.0 per 100 PY) of patients who received darbepoetin alfa. Following initiation of VAFSEO, monitor patients closely for premonitory neurologic symptoms. Monitor for new-onset seizures, premonitory symptoms, or change in seizure frequency. Gastrointestinal Erosion Gastric or esophageal erosions occurred in 6.4% (4.0 per 100 PY) of patients receiving VAFSEO and 5.3% (3.3 per 100 PY) of darbepoetin alfa-treated patients. Serious gastrointestinal (GI) erosions, including GI bleeding and the need for red blood cell transfusions were reported in 3.4% (2.1 per 100 PY) and 3.3% (2.0 per 100 PY) of those receiving VAFSEO and darbepoetin alfa, respectively. Consider the risk of GI erosion in high-risk patients, including those with a history of GI erosion, peptic ulcer disease, and tobacco or alcohol use. Advise patients of the signs and symptoms of erosions and GI bleeding and urge them to seek prompt medical care if present. Serious Adverse Reactions in Patients with Anemia Due to Chronic Kidney Disease and Not on Dialysis The safety of VAFSEO has not been established for the treatment of anemia due to CKD in adults not on dialysis and its use is not recommended in this setting. In large clinical trials in adults with anemia of CKD who were not on dialysis, an increased risk of mortality, stroke, myocardial infarction, serious acute kidney injury, serious hepatic injury, and serious GI erosions was observed in patients treated with VAFSEO compared to darbepoetin alfa. Malignancy VAFSEO has not been studied and is not recommended in patients with active malignancies. Malignancies were observed in 2.2% (1.3 per 100 PY) of patients treated with VAFSEO and 3.0% (1.8 per 100 PY) of patients treated with darbepoetin alfa. No evidence of increased carcinogenicity was observed in animal studies. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%) were hypertension and diarrhea. DRUG INTERACTIONS Iron supplements and iron-containing phosphate binders: Administer VAFSEO at least 1 hour before products containing iron. Non-iron-containing phosphate binders: Administer VAFSEO at least 1 hour before or 2 hours after non-iron-containing phosphate binders. BCRP substrates: Monitor for signs of substrate adverse reactions and consider dose reduction. Statins: Monitor for statin-related adverse reactions. Limit the daily dose of simvastatin (20 mg) and rosuvastatin (5 mg). USE IN SPECIFIC POPULATIONS Pregnancy: May cause fetal harm. Lactation: Breastfeeding not recommended until two days after the final dose. Hepatic Impairment: Not recommended for use in patients with cirrhosis or active, acute liver disease. Please note that this information is not comprehensive. Please click here for the Full Prescribing Information, including BOXED WARNING and Medication Guide. IMPORTANT U.S. SAFETY INFORMATION FOR AURYXIA (ferric citrate) CONTRAINDICATION AURYXIA (ferric citrate) is contraindicated in patients with iron overload syndromes, e.g., hemochromatosis. WARNINGS AND PRECAUTIONS Iron Overload: Increases in serum ferritin and transferrin saturation (TSAT) were observed in clinical trials with AURYXIA in patients with chronic kidney disease (CKD) on dialysis treated for hyperphosphatemia, which may lead to excessive elevations in iron stores. Assess iron parameters prior to initiating AURYXIA and monitor while on therapy. Patients receiving concomitant intravenous (IV) iron may require a reduction in dose or discontinuation of IV iron therapy. Risk of Overdosage in Children Due to Accidental Ingestion: Accidental ingestion and resulting overdose of iron-containing products is a leading cause of fatal poisoning in children under 6 years of age. Advise patients of the risks to children and to keep AURYXIA out of the reach of children. ADVERSE REACTIONS Most common adverse reactions with AURYXIA were: Hyperphosphatemia in CKD on Dialysis: Diarrhea (21%), discolored feces (19%), nausea (11%), constipation (8%), vomiting (7%) and cough (6%). Iron Deficiency Anemia in CKD Not on Dialysis: Discolored feces (22%), diarrhea (21%), constipation (18%), nausea (10%), abdominal pain (5%) and hyperkalemia (5%). SPECIFIC POPULATIONS Pregnancy and Lactation: There are no available data on AURYXIA use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage. However, an overdose of iron in pregnant women may carry a risk for spontaneous abortion, gestational diabetes and fetal malformation. Data from rat studies have shown the transfer of iron into milk, hence, there is a possibility of infant exposure when AURYXIA is administered to a nursing woman. To report suspected adverse reactions, contact Akebia Therapeutics at 1-844-445-3799. Please click to see the full Prescribing Information for AURYXIA. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia's plans with respect to the commercial launch of Vafseo; timing of the commercial availability of Vafseo, including the belief that launch activities will enable widespread access and rapid adoption when Vafseo is available in January 2025; expectations with respect to Akebia's application for Transitional Drug Add-on Payment Adjustment (TDAPA) for Vafseo and TDAPA designation, including the timing thereof; plans regarding potential label expansion; plans to generate clinical data to identify additional areas of potential benefits to patients; Akebia's expectations with respect to engagement with the FDA on label expansion and the timing thereof; Akebia's expectations for Auryxia net product revenue growth in 2024 and assumptions related thereto; Akebia's goal of ensuring broad access to Auryxia; and Akebia's goals, objectives and expectations with respect to its operating plan, expenses, cash resources and sources of funding for its cash runway, including its belief that its existing cash resources and the cash it expects to generate from product revenue are sufficient to fund its current operating plan, including a U.S. Vafseo launch, for at least the next two years. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: whether Vafseo will be commercially available when expected; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Auryxia® and Vafseo, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia and Vafseo, including potential generic entrants; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to implement cost avoidance measures and reduce operating expenses; decisions made by health authorities, such as the FDA, with respect to regulatory filings; the potential therapeutic benefits, safety profile, and effectiveness of Vafseo; the results of preclinical and clinical research; the direct or indirect impact of the COVID-19 pandemic on the markets and communities in which Akebia and its partners, collaborators, vendors and customers operate; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; and early termination of any of Akebia's collaborations. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics®, Auryxia® and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. and its affiliates. Akebia Therapeutics Contact Mercedes Carrasco [email protected] SOURCE Akebia Therapeutics, Inc.

关注并星标CPHI制药在线按照最近的标准，2024年第一季度在新药批准方面相对缓慢。今年前三个月只有10种新药获得授权（表1）。相比之下，2023年第一季度为13以上，这一数字与去年随后的每个季度持平或超过。              表1 | 2024年第一季度部分FDA新药批准（表格来源于nature）       尽管如此，第一季度的10项审批包括几项预期的重磅项目。Madrigal Pharmaceuticals的Rezdiffra（resmetirom）是甲状腺激素受体β激动剂，是第一个获准用于非酒精性脂肪性肝炎（NASH）患者的治疗方法，非酒精性脂肪性肝炎也称为代谢功能障碍相关脂肪性肝炎（MASH）。鉴于NASH的批准已被证明难以实现，而且该产品现已率先上市，人们对其期望很高，普遍预测2028年的销售额将超过20亿美元。美国食品和药物管理局（FDA）根据一项基于替代终点指标（衡量肝脏炎症和瘢痕形成程度）的加速批准路径，批准Rezdiffra用于患有中度至晚期肝瘢痕的非肝硬化NASH患者。Madrigal需要进行一项批准后研究以验证临床效益，该研究正在进行中。该产品还获得了适应症的突破性疗法、快速通道和优先审查称号。       根据Evaluate consensus的预测，默克公司的Winrevair（sota recept）进入市场后，预计2028年的销售额将接近30亿美元。该产品是一流的激活素信号抑制剂，在STELLAR试验中被批准用于治疗肺动脉高压（PAH），与单独使用背景疗法相比，运动能力显著提高。多环芳烃代表着尚未满足的临床需求，这反映在对该产品的商业期望以及该产品已从美国食品和药物管理局获得的突破性疗法称号中。Idorsia的内皮素A和B受体拮抗剂try VIO（aprocitentan）在本季度获得了进一步的高血压批准，这是第一个针对这一途径的产品被批准用于治疗顽固性高血压。       本季度的第三项批准预计将在2028年前实现重磅销售--Beigene的Tevimbra（tislelizumab），这是一种PD1靶向单克隆抗体，用于治疗既往全身化疗后不含PD-（L）1抑制剂的不可切除或转移性食管鳞状细胞癌（ESCC）成年患者。此前，受冠状病毒肺炎相关旅行限制的影响，Beigene推迟了批准时间。此前，Beigene与诺华的商业合作关系破裂，Beigene重新获得了该产品的全球使用权。该产品也正在接受ESCC食品和药物管理局的审查，将于今年晚些时候做出决定。tislelizumab（替雷丽珠丹康注射液）是一种抗人程序性死亡受体-1（PD-1）单克隆IgG4抗体，由BeiGene开发为免疫治疗抗肿瘤药物。Tislelizumab已在血液系统癌症和晚期实体瘤中进行了研究，导致其于2019年12月在中国获批用于至少二线化疗后复发或难治性经典霍奇金淋巴瘤患者。       在本季度获得批准的其他药物中，意大利制药公司的组蛋白去乙酰化酶抑制剂Duvyzat（givinostat）是第一个在美国获得批准的治疗杜氏肌营养不良症所有遗传变异的非甾体药物，而阿斯利康的voydeya（danicopan）是第一个用于治疗阵发性夜间血红蛋白尿症的D因子抑制剂。杜氏肌肉营养不良症( Duchenne Muscular Dystrophy，DMD)，乃遗传性肌肉萎缩病。它的基因( Dystrophin gene)存在于X性染色体中( Xp21 )，因此它是透过性连锁式隐性遗传型态传播的。       其余的批准包括用于传染性软疣的Ligand pharma ceuticals Zelsuvmi（berdazimer）、用于复杂尿路感染的Allecra Therapeutics复方抗生素Exblifep（头孢吡肟和恩美他唑巴坦）、用于慢性肾病所致贫血的Hugel美容药物Letybo（LetibotulinumtoxinA）和Akebia Therapeutics的vaf SEO（vadadustat）。       参考文献：       1.https://www-nature-com.libproxy1.nus.edu.sg/articles/d41573-024-00063-x?utm_source=xmol&utm_medium=affiliate&utm_content=meta&utm_campaign=DDCN_1_GL01_metadata.【智药研习社近期直播预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~