Glucose Intolerance

AstraZeneca’s rare disease portfolio generated $7.76bn in global sales in 2023, as per the company’s Q4 financials. Image Credit: EllaSt / Shutterstock. AstraZeneca has signed a definitive agreement to acquire Amolyt Pharma, which specialises in therapies for rare endocrine diseases, in a deal worth $1.05bn. As per the contract, AstraZeneca will pay $800m upfront along with potential regulatory milestone payments of $250m. The deal is expected to close in Q3 2024. Amolyt’s lead candidate, eneboparatide (AZP-3601), was a central part of the acquisition. Eneboparatide is a subcutaneous parathyroid hormone receptor 1 (PTHR1) agonist for the treatment of hypoparathyroidism. This condition is caused by a deficiency in the parathyroid hormone that results in decreased calcium and elevated phosphorus levels in the blood. Dysregulation of parathyroid hormone can lead to osteoporosis and chronic kidney disease. Amolyt launched a Phase III CALYPSO trial (NCT05778071) for eneboparatide in May 2023. Topline data from the trial is expected by the end of this year. Another candidate in Amolyt’s pipeline is AZP-3813. The therapy is a peptide growth hormone receptor antagonist, which is being developed as a potential add-on to somatostatin analogues for the treatment of acromegaly, a condition associated with abnormally high secretion of growth hormone. It can lead to a variety of complications including heart failure and impaired glucose tolerance. The company initiated a Phase I trial for AZP-3813 in June 2023. See Also: Almirall to license Eloxx’s ZKN-013 for rare skin conditions Risk adjusted net present value: What is the current valuation of AstraZeneca’s AZD-3427? AstraZeneca’s rare disease portfolio generated $7.76bn in global sales in 2023, as per the company’s Q4 financials. The UK pharma giant has been investing heavily in rare disease therapies starting with the $39bn acquisition of Alexion in 2021. Last year, the company paid $1bn to acquire an early-stage gene therapy portfolio for rare diseases from Pfizer. According to a GlobalData report, innovation in rare diseases is seen as a risky investment, with rare oncology indications dominating most of the rare disease pipeline. GlobalData is the parent company of Pharmaceutical Technology . Despite this, AstraZeneca has seen some success amongst rare diseases that are not cancer-related. In September 2023, the Phase III trial for a rare vasculitis therapy, Fasenra (benralizumab), met its primary endpoint. The study found that Fasenra was non-inferior to the current standard of care, Pfizer’s Nucala (mepolizumab). Earlier this year, AstraZeneca reported positive outcomes from its Japan Phase III clinical trial of acoramidis, an investigational treatment for a rare cardiomyopathy, transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

CHICAGO--(BUSINESS WIRE)-- Xeris Biopharma, Inc. (Nasdaq: XERS), a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products across a range of therapies, today announced that it has entered into an exclusive worldwide license agreement for Amgen to develop, manufacture, and commercialize a subcutaneous formulation of teprotumumab using Xeris’ XeriJect® technology in Thyroid Eye Disease (TED) - a serious, progressive and potentially vision-threatening rare autoimmune disease. Teprotumumab-trbw is known as TEPEZZA® in the United States. “We are very excited that our partner is moving forward with licensing the XeriJect technology to further the development of the XeriJect subcutaneous teprotumumab to potentially enhance the patient experience and delivery of the treatment for Thyroid Eye Disease. This agreement to license XeriJect further validates the potential value of our technology to enable large molecule subcutaneous injections that provide a more patient friendly regimen that is effective, safe, and more convenient, with potential for improved adherence,” said Paul R. Edick, Chairman and CEO of Xeris. “We will move quickly to support our partner in this important development program.” Under the terms of the License Agreement, Xeris has the potential to receive $75 million in development and regulatory milestones, plus sales-based milestones, as well as escalating single-digit royalties based on future sales of TEPEZZA using the XeriJect technology. About XeriJect® XeriJect formulations are innovative, ready-to-use, viscoelastic pharmaceutical suspensions that have the potential to improve drug delivery, lower treatment burden and improve patients' lives across a broad range of therapeutic categories. XeriJect suspensions maximize drug loadings at >400mg/mL, enable small volume subcutaneous injections and do not settle on storage. The suspensions use FDA-approved excipients and leverage known manufacturing processes. XeriJect formulation technology is well suited for drugs and biologics including large molecules such as proteins, monoclonal antibodies, and vaccines. The technology is protected by an extensive patent estate, trade secrets and know-how, and it is available for licensing. About Xeris Xeris (Nasdaq: XERS) is a growth-oriented biopharmaceutical company committed to improving patients’ lives by developing and commercializing innovative products across a range of therapies. Xeris has three commercially available products; Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia, Keveyis®, for a proven therapy for primary periodic paralysis, and Recorlev® for the treatment of endogenous Cushing’s syndrome. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect®, supporting long-term product development and commercial success. Xeris Biopharma Holdings is headquartered in Chicago, IL. For more information, visit , or follow us on X, LinkedIn, or Instagram. Forward-Looking Statements Any statements in this press release other than statements of historical fact are forward-looking statements. Forward-looking statements include, but are not limited to, statements about future expectations, plans and prospects for Xeris Biopharma Holdings, Inc., including the development and potential of XeriJect® subcutaneous TEPEZZA®, the expectations regarding future product development efforts between Xeris and Amgen, Xeris’ potential entitlements to milestone and royalty payments from Amgen, the potential utility of its formulation platforms such as XeriJect, the market and therapeutic potential of its products and product candidates, and other statements containing the words “will,” “would,” “continue,” “expect,” “should,” “anticipate” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on numerous assumptions and assessments made in light of Xeris’ experience and perception of historical trends, current conditions, business strategies, operating environment, future developments, geopolitical factors and other factors it believes appropriate. By their nature, forward-looking statements involve known and unknown risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. The various factors that could cause Xeris’ actual results, performance or achievements, industry results and developments to differ materially from those expressed in or implied by such forward-looking statements, include, but are not limited to, its financial position and need for financing, including to fund its product development programs or commercialization efforts, whether its products will achieve and maintain market acceptance in a competitive business environment, its reliance on third-party suppliers, including single-source suppliers, its reliance on third parties to conduct clinical trials, the ability of its product candidates to compete successfully with existing and new drugs, and its and collaborators’ ability to protect its intellectual property and proprietary technology. No assurance can be given that such expectations will be realized and persons reading this communication are, therefore, cautioned not to place undue reliance on these forward-looking statements. Additional risks and information about potential impacts of financial, operational, economic, competitive, regulatory, governmental, technological, and other factors that may affect Xeris can be found in Xeris’ filings, including its most recently filed Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, the contents of which are not incorporated by reference into, nor do they form part of, this communication. Forward-looking statements in this communication are based on information available to us, as of the date of this communication and, while we believe our assumptions are reasonable, actual results may differ materially. Subject to any obligations under applicable law, we do not undertake any obligation to update any forward-looking statement whether as a result of new information, future developments or otherwise, or to conform any forward-looking statement to actual results, future events, or to changes in expectations. About TEPEZZA INDICATION TEPEZZA is indicated for the treatment of Thyroid Eye Disease regardless of Thyroid Eye Disease activity or duration. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Infusion Reactions: TEPEZZA may cause infusion reactions. Infusion reactions have been reported in approximately 4% of patients treated with TEPEZZA. Reported infusion reactions have usually been mild or moderate in severity. Signs and symptoms may include transient increases in blood pressure, feeling hot, tachycardia, dyspnea, headache, and muscular pain. Infusion reactions may occur during an infusion or within 1.5 hours after an infusion. In patients who experience an infusion reaction, consideration should be given to premedicating with an antihistamine, antipyretic, or corticosteroid and/or administering all subsequent infusions at a slower infusion rate. Preexisting Inflammatory Bowel Disease: TEPEZZA may cause an exacerbation of preexisting inflammatory bowel disease (IBD). Monitor patients with IBD for flare of disease. If IBD exacerbation is suspected, consider discontinuation of TEPEZZA. Hyperglycemia: Increased blood glucose or hyperglycemia may occur in patients treated with TEPEZZA. In clinical trials, 10% of patients (two-thirds of whom had preexisting diabetes or impaired glucose tolerance) experienced hyperglycemia. Hyperglycemic events should be controlled with medications for glycemic control, if necessary. Assess patients for elevated blood glucose and symptoms of hyperglycemia prior to infusion and continue to monitor while on treatment with TEPEZZA. Ensure patients with hyperglycemia or preexisting diabetes are under appropriate glycemic control before and while receiving TEPEZZA. Hearing Impairment Including Hearing Loss: TEPEZZA may cause severe hearing impairment including hearing loss, which in some cases may be permanent. Assess patients’ hearing before, during, and after treatment with TEPEZZA and consider the benefit-risk of treatment with patients. ADVERSE REACTIONS The most common adverse reactions (incidence ≥5% and greater than placebo) are muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing impairment, dysgeusia, headache, dry skin, weight decreased, nail disorders, and menstrual disorders. Please see Full Prescribing Information or visit TEPEZZAhcp.com for more information.

Initial data from the clinical trial is expected in 2024 PORTLAND, Ore.--(BUSINESS WIRE)-- Sparrow Pharmaceuticals, an emerging, clinical-stage biopharmaceutical company developing novel, targeted therapies to address unmet needs in both rheumatology and endocrinology, today announced that the first patient has been dosed in the Phase 2 ACSpire study (NCT number: NCT05436639) of SPI-62, a potent and selective HSD-1 inhibitor, for the treatment of autonomous cortisol secretion (ACS), a prevalent yet serious condition caused by the overproduction of cortisol from a benign tumor of the adrenal gland. This trial will complement the ongoing Phase 2 RESCUE study (NCT number: NCT05307328) for the treatment of ACTH-dependent Cushing’s syndrome. “ACS is a dangerous condition with high morbidity and risk of death, with patients often having unexplained metabolic disease, cardiovascular disease, or osteopenia, and experiencing a 35% higher rate of mortality over a five-year span,” said Frank Czerwiec, chief medical officer of Sparrow. “However, most patients don’t even know they have ACS and are only diagnosed after an appropriate endocrine workup following the discovery of an adrenal tumor on a CT or MRI scan for unrelated reasons. SPI-62 could represent the first treatment to be approved for this large yet underappreciated and underserved patient population.” The pilot, long-term, open-label study is evaluating the efficacy, safety, and pharmacological effect of SPI-62 in participants with hypercortisolism related to a benign adrenal tumor. The study will examine SPI-62's effect on morbidities of hypercortisolism including diabetes or impaired glucose tolerance, hyperlipidemia, hypertension, and osteopenia. Sparrow is actively enrolling up to 30 participants in ACSpire at sites in the United States, soon to be joined by sites in Romania, France, and the United Kingdom. Criteria for participation include adults with documented benign adrenal lesions with proven ACS, and documentation of treatment for, or evidence of, ongoing metabolic consequences for at least one of the following: hyperglycemia, hypertension, hyperlipidemia, or osteopenia, attributable to clinically significant hypercortisolism. Surgery as first-line therapy should be discussed with all eligible participants, who will be included only if they have failed or rejected available surgical therapy. To learn more about Sparrow Pharmaceuticals and its clinical trials, visit the website at . About Autonomous Cortisol Secretion Autonomous cortisol secretion (ACS) is caused by the overproduction of cortisol from a tumor of the adrenal gland. It is a somewhat milder form of hypercortisolism than Cushing’s syndrome, which is differentiated by a characteristic constellation of signs and symptoms. As patients with ACS lack these outward clinical features of Cushing’s but present with common, chronic conditions (diabetes, hypertension, high cholesterol, weakened bones), it is rarely diagnosed or addressed specifically. ACS is currently only discovered when the tumor shows up on a radiology scan for an unrelated health condition and the patient subsequently receives an adequate endocrine workup. As this infrequently occurs, most patients with ACS are dangerously exposed to excess cortisol for long periods, leading them to face major health complications, significant healthcare costs, and increased risk of early death. The signs and symptoms of ACS vary across a wide range of mental and physical effects – including diabetes, hypertension, bone fractures, and weight gain, as well as mood, cognition, and sleep disorders. About Sparrow Pharmaceuticals Sparrow Pharmaceuticals was founded to spare patients the ravages of steroids. Leveraging underappreciated scientific insights into glucocorticoid biology, the company is working to provide better treatment options for serious disorders of hypercortisolism, and to revolutionize the treatment of autoimmune and inflammatory conditions. Its lead product, SPI-62, is an oral, small molecule, novel therapeutic treatment designed to target the source of active intracellular glucocorticoids in key tissues.