Metal Metabolism, Inborn Errors

Potential vadadustat U.S. approval on PDUFA date of March 27, 2024 Strengthened balance sheet with $55.0 million term loan financing and $26.0 million in proceeds from ATM Reported 2023 Auryxia (ferric citrate) net product revenue of $170.3 million CAMBRIDGE, Mass., March 14, 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 fourth quarter and full year ended December 31, 2023 and recent business highlights. Akebia is preparing for a potential launch of vadadustat, which is currently under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) date of March 27, 2024. "We are eagerly awaiting the PDUFA date for vadadustat, now within weeks, and we believe the progress we have made over the past 12 months has positioned our team to successfully launch vadadustat in the U.S., if approved," said John P. Butler, Chief Executive Officer of Akebia. "A U.S. approval for vadadustat will be transformational for Akebia and a significant step toward our goal of bettering the lives of people impacted by kidney disease. Our team remains dedicated to delivering an innovative oral therapeutic treatment for anemia due to chronic kidney disease for patients on dialysis." Fourth Quarter 2023 and Recent Business Highlights: Appointed Nicholas Grund as Chief Commercial Officer, who brings years of expertise in customer-facing roles to Akebia. Mr. Grund's operational, commercial and strategic leadership experience across renal and specialty markets will be critical as Akebia prepares for the vadadustat launch in the U.S., if approved. Introduced new pipeline programs in acute care settings, potentially for acute kidney injury or acute respiratory distress syndrome (AKB-9090) and retinopathy of prematurity in neonates (AKB-10108). Closed a new debt facility with BlackRock that provides access to up to $55.0 million in borrowing capacity and used the proceeds from the first tranche of $37.0 million to pay down principal outstanding under the then loan agreement with Pharmakon Advisors, LP. The BlackRock debt facility, which closed on January 29, 2024, also extends the interest-only period in the event of vadadustat approval in the U.S. on or prior to June 30, 2024 without requiring any principal repayment until December 31, 2026. Concluded its offering of common stock under its "at-the-market" (ATM) sales agreement. Akebia raised approximately $26.0 million in gross proceeds. Akebia reported fourth quarter 2023 Auryxia® (ferric citrate) net product revenues of $53.2 million and full year 2023 revenues of $170.3 million, within Akebia's 2023 Auryxia net product revenue guidance of $170.0 - $175.0 million. "We are approaching a potential U.S. launch of vadadustat from an extremely strong financial position. We expect Auryxia net product revenue growth in 2024, with a quarterly revenue cadence that is similar to 2023, we executed a term loan with BlackRock and implemented other financial strategies that together we believe will support our business operations for at least two years if vadadustat is approved. As we move forward, we will continue to carefully manage expenses, while investing appropriately for a successful potential launch of vadadustat," Mr. Butler added. Financial Results Revenues: Total revenues were $56.2 million for the fourth quarter of 2023 compared to $55.8 million for the fourth quarter of 2022, and $194.6 million for the full-year 2023 compared to $292.5 million for the full-year 2022. Net product revenues were $53.2 million for the fourth quarter of 2023 compared to $50.3 million for the fourth quarter of 2022, and $170.3 million for the full-year 2023 compared to $176.9 million for the full-year 2022. License, collaboration and other revenues were $3.0 million for the fourth quarter of 2023 compared to $5.5 million for the fourth quarter of 2022, and $24.3 million for the full-year 2023 compared to $115.5 million for the full-year 2022. COGS: Cost of goods sold was $18.7 million for the fourth quarter of 2023 compared to a benefit of $3.4 million for the fourth quarter of 2022, and $74.1 million for the full-year 2023 compared to $85.6 million for the full-year 2022. Akebia continues to record 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 $9.9 million for the fourth quarter of 2023 compared to $32.1 million for the fourth quarter of 2022, and $63.1 million for the full-year 2023 compared to $130.0 million for the full-year 2022. SG&A Expenses: Selling, general and administrative expenses were $25.4 million for the fourth quarter of 2023 compared to $29.9 million for the fourth quarter of 2022, and $100.2 million for the full-year 2023 compared to $138.6 million for the full-year 2022. Net Income / Loss: Net income was $0.6 million for the fourth quarter of 2023 compared to a net loss of $6.1 million for the fourth quarter of 2022, and $51.9 million for the full-year 2023 compared to $94.2 million for the full-year 2022. Cash Position: Cash and cash equivalents as of December 31, 2023, were approximately $42.9 million. Akebia believes its existing cash resources and the cash it expects to generate from product, royalty, supply and license revenues as well as the borrowings and potential future borrowings that are available under the BlackRock debt facility and the working capital liability are sufficient to fund our current operating plan for at least twenty-four months if vadadustat is approved. 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 Vadadustat Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues. Vadadustat is not approved by the U.S. Food and Drug Administration. Vadadustat is approved in Europe and Australia for the treatment of symptomatic anemia due to CKD in adult patients on chronic maintenance dialysis and in Taiwan for the treatment of anemia due to CKD in adult patients on dialysis. In Japan, vadadustat is approved as a treatment for anemia due to CKD in both dialysis-dependent and non-dialysis dependent adult patients. IMPORTANT SAFETY INFORMATION FOR VAFSEO (vadadustat) For safety information, view the European Summary of Product Characteristics (SPC/SmPC) for Vafseo® (vadadustat) at , and and will be available via the Australian Therapeutic Goods Administration website. 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 see full Prescribing Information 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 financial results for the fourth quarter and full year ended December 31, 2023; the anticipated scheduled PDUFA date for vadadustat and the potential approval of vadadustat; Akebia's ability to enable a successful commercial launch of vadadustat, if approved; statements that a U.S. approval for vadadustat will be transformational for Akebia; Akebia's expectations and beliefs regarding the impact that the BlackRock debt facility will have on Akebia; Akebia's expectations for Auryxia revenue growth in 2024 and assumptions related thereto; Akebia's plans with respect to vadadustat as a treatment of anemia due to chronic kidney disease in patients on dialysis in the U.S.; Akebia's expectations with respect to Akebia's pipeline in acute care settings for acute kidney injury or acute respiratory distress syndrome (AKB-9090) and retinopathy of prematurity in neonates (AKB-10108) and market potential; 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, royalty, supply and license revenues as well as the borrowings and potential future borrowings that are available under the BlackRock debt facility and the working capital liability are sufficient to fund our current operating plan for at least twenty-four months if vadadustat is approved. 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: the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Auryxia, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia, 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, including the anticipated FDA decision on the NDA for vadadustat and the potential effects of a negative decision on Akebia's cash runway; the potential therapeutic benefits, safety profile, and effectiveness of vadadustat; the results of preclinical and clinical research; the direct or indirect impact of the COVID-19 pandemic on regulators and Akebia's business, operations, and 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 September 30, 2023, 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.

BALTIMORE, March 14, 2024 (GLOBE NEWSWIRE) -- Telomir Pharmaceuticals, Inc.(Nasdaq:TELO) (“Telomir” or the “Company”), a pre-clinical-stage pharmaceutical company focused on the development and commercialization of Telomir-1 as the first novel small molecule to lengthen the DNA’s protective telomere caps in order to potentially reverse age-related conditions, today highlighted its use of computer-aided testing in a pre-clinical setting to study and analyze the potential benefits of its lead product candidate, Telomir-1. Using in silico modeling, which deploys artificial intelligence-driven computational models to predict a compound’s therapeutic potential, biological activities and toxicity, Telomir continues to find evidence that the mechanism of action of Telomir-1 has the potential to reverse age-related conditions such as osteoarthritis by lengthening DNA's protective telomere caps. Telomir’s recent AI-powered research presented in Singapore last month not only supports but amplifies the therapeutic potential of Telomir-1. Additional AI-driven studies now being conducted to demonstrate Telomir-1's promising therapeutic potential, supporting Telomir’s hypothesis with solid computational evidence. Telomir’s advanced AI in silico model is seeking to enable a deeper understanding of Telomir-1's interactions at the molecular level and to affirm its ability to lengthen telomeres and potentially combat age-related diseases. As part of its work, Telomir works with third-party collaborators Frontage Laboratories and InSilicoTrials, each of whom utilize AI in-silico testing models. Following its previously announced presentation in Singapore, Telomir is excited to announce an upcoming presentation of its research in Las Vegas at the Global Longevity Federation on March 25-26, 2024. This conference is focused on continued discovery in the battle against aging. As additional research is conducted, Telomir is planning to present its findings at future scientific conferences. Chris Chapman, MD, Co-Founder, Chairman, Chief Executive Officer and President of Telomir, stated "Our continuing work on AI-driven research of Telomir-1 is not just an accomplishment for our company but for anti-aging science as a whole. Our innovative approach, combining cutting-edge AI technology with novel drug development, is paving the way for significant discovery in this important area of science. Our goal is to progress Telomir-1 through the pre-clinical process and into human trials to unlock Telomir-1's potential as a transformative treatment for aging and age-related diseases, instilling hope and improving the quality of life for individuals worldwide." To be added to the Telomir Pharmaceuticals email distribution list, please email telomir@kcsa.com with TELO in the subject line. About Telomir Pharmaceuticals, Inc. Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) is a pre-clinical-stage pharmaceutical company focused on the development and commercialization of Telomir-1 as the first novel small molecule to lengthen the DNA’s protective telomere caps in order to potentially reverse age-related conditions. Telomeres are the protective end caps of a chromosome made up of DNA sequences and proteins. As humans age, telomeres shorten, with metal reactivity accelerating the process, which presents us with an increased chance of contracting a number of degenerative and age-related diseases. Telomir’s goal is to develop and commercialize Telomir-1 (which is proposed to be dosed orally) for hemochromatosis (iron overload) and ultimately post-chemotherapy recovery and a broader range of other age-related inflammatory conditions such as osteoarthritis. Cautionary Note Regarding Forward-Looking Statements This press release and the statements of the Company’s management related thereto contains “forward-looking statements,” which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding Telomir-1’s potential in reversing age-related conditions. Any forward-looking statements in this press release are based on the Company's current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties (many of which are beyond the Company’s control) that could cause actual results (including the anticipated benefits of the Company’s pre-clinical testing and pre-clinical data discussed herein) to differ materially and adversely from those set forth in or implied by such forward-looking statements. These and other risks concerning the Company's programs and operations are described in additional detail in its registration statement on Form S-1 and other SEC filings, which are on the SEC at and the Company’s website at The Company explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. KCSA Strategic Communications Phil Carlson telomir@kcsa.com Telomir Pharmaceuticals info@Telomirpharma.com (813) 864-2558

A largescale new study has found that some people whose genetics are linked to the common iron overload condition haemochromatosis have substantially greater levels of liver, musculoskeletal and brain disease than previously reported, especially at older ages. A largescale new study has found that some people whose genetics are linked to the common iron overload condition haemochromatosis have substantially greater levels of liver, musculoskeletal and brain disease than previously reported, especially at older ages. Haemochromatosis causes a build-up of iron in the body which can cause harm to joints and organs -- although the extent of this harm is unclear, especially in older ages. The new research, led by a team at the University of Exeter and supported by the National Institute for Health and Care Research (NIHR) Exeter Biomedical Research Centre, found higher levels of disease plus greater mortality in males, especially after age 65. Published in BMJ Open, the research analysed data from more than 450,000 European Ancestry participants in UK Biobank, over a 13 year follow-up, making it the largest-scale study to look at disease outcomes linked to haemochromatosis. For the first time, the team provided data on clinical outcomes up to the age of 80 years on people who carry genes linked to haemochromatosis, providing insight into how the condition affects older age groups, including those who were undiagnosed at baseline. Dr Janice Atkins, Senior Research Fellow at the University of Exeter and study co-author, said: "Haemochromatosis is the most common genetic disorder in people of Northern European ancestry, yet we are still learning the extent to which it causes harm. Our study is the most comprehensive to date and reveals higher levels of death and organ and joint damage than previously known, including liver disease and joint replacements." In UK Biobank, 2,902 people (0.6 % of participants) had two copies of the genetic variant (C282Y) which mean they are at risk of the condition. Of these people, who had a mean age of 57, only a small proportion were diagnosed with haemochromatosis at the start of the study- 12% of males and 3.4% of females. By the times males with the two genetic variants had reached the age of 80, the study found that 33.1% had died, compared to 25.4% without thevariants. The study also found that 27.9% vs 17.1% had joint replacements, 20.3% vs 8.3% had liver disease, and there was higher delirium, non Alzheimer's dementia, and Parkinson's disease in those with the two gene variants. In females by age 80, those with both genetic variants had higher levels of liver disease (8.9% vs 6.8%), joint replacements and delirium. Haemochromatosis is more common in males, and particularly affects white people of Northern European ancestry. An estimated 175,000 men of European ancestry in the UK have the two gene variations that cause haemochromatosis. The disorder is known as the "Celtic curse" because it is particularly prevalent in Celtic bloodlines. Reliable tests are available to identify those at risk -- blood tests for measuring iron levels and genetic testing. Symptoms can include feeling tired all the time, muscle weakness and joint pains, meaning it is often misdiagnosed as the usual signs of ageing. Once diagnosed, the condition is easily treated by a process similar to donating blood several times a year, to lower iron levels. The blood can be donated to the NHS transfusion service once iron levels have reduced enough. Professor David Melzer, who leads the research team, said: "These findings highlight the need for earlier diagnosis of iron overload, as many people with the high risk genetic variants are currently diagnosed too late to prevent organ damage. The condition can be picked up with a simple blood iron test, and it is encouraging that more and more GPs are aware of the need to test for iron overload. The research results also contained very good news for people with the milder genetic variants (called C282Y/H63D and H63D homozygotes), with no excess of fatigue, liver fibrosis, diabetes or arthritis, adding to the evidence that these variants are benign. Of course, some people with these variants will unfortunately have fatigue, liver fibrosis, arthritis or diabetes, but this happens at the same rate as people without these gene variants and underlines the need to investigate the other causes of these conditions too." Study co-author Dr Jeremy Shearman, Consultant Hepatologist and Gastroenterologist at South Warwickshire University NHS Foundation Trust, said: "This important data supports the long-held belief that we should be using targeted genetic testing more consistently to make an earlier and more accurate diagnosis of haemochromatosis. It is a common condition here in the UK and can be easily treated to avoid serious harm."