Gottron syndrome

RESCUE trial continues to enroll eligible patients; data 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 the first patient has elected to enroll in the open-label extension (OLE) phase of the RESCUE trial, a phase 2 clinical trial of SPI-62, a potent and selective HSD-1 inhibitor, for the treatment of ACTH-dependent Cushing’s syndrome. The long-term OLE will be open to patients who complete the initial RESCUE cross-over treatment protocol and then elect to continue to participate in the research. “Cushing’s syndrome can be life-threatening and the preferred first-line surgical treatment often fails. While medical therapies are increasingly used, there are many drawbacks and innovative medical approaches have lagged,” said Frank Czerwiec, MD, PhD, Chief Medical Officer of Sparrow. “We’re excited to graduate patients from the initial RESCUE study to a new phase of investigation of SPI-62, one which will allow us to deepen our understanding of the potential for long-term safety and efficacy of this completely novel mode of treatment for Cushing’s.” RESCUE is evaluating the pharmacologic effect, efficacy, and safety of SPI-62 in patients with adrenocorticotropic hormone (ACTH)-dependent Cushing’s syndrome. Evidence suggests that by inhibiting HSD-1, SPI-62 can lower intracellular levels of cortisol in key organs, thereby potentially mitigating the adverse effects of hypercortisolism. SPI-62 could represent the first new mechanism of action to treat Cushing’s syndrome in decades. The OLE will seek to gather new long-term safety and tolerability data, as well as allow continued monitoring of SPI-62’s effects on signs and symptoms of Cushing’s syndrome. Sparrow is actively enrolling the RESCUE trial at approximately 16 sites in the U.S., Bulgaria, and Romania. Criteria for participation include male and female subjects, ages 18 years or older, with active and consistent cortisol excess and documented diagnosis of ACTH-dependent Cushing’s syndrome. To learn more about the RESCUE trial (NCT05307328), please visit . About Cushing’s Syndrome and SPI-62 Patients develop Cushing’s syndrome due to an excess of glucocorticoids. In Cushing’s disease, this is due to a pituitary tumor that leads to high levels of adrenocorticotropic hormone, which stimulates excess cortisol secretion. ACTH, or its releasing hormone CRH, may also be produced by non-pituitary tumors. By targeting the intracellular activation of cortisol in key organs, SPI-62 can potentially mitigate multiple signs and symptoms of cortisol excess. HSD-1 inhibition would be the first new mechanism of action in decades to treat Cushing’s. In phase 1 studies, SPI-62 demonstrated the ability to reduce intracellular cortisol in the liver and to inhibit HSD-1 in both the brain and adipose tissue, which are three key tissues in which corticosteroid toxicity leads to morbidity. 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.

AstraZeneca has received the first warning letter of the year from the FDA’s promotion police over alleged efficacy claims for its COPD med Breztri Aerosphere. The warning is only the second letter of the year from FDA’s promo office, following an untitled letter in June to Xeris Pharmaceuticals for its Cushing’s syndrome drug Recorlev. The Office of Prescription Drug Promotion (OPDP) sent the AstraZeneca warning letter – the more serious of the two types of enforcement notices – regarding a Breztri “professional sales aid” during the first week of August. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

Achieved record revenue of $38.0M – a 14% increase from prior quarter, and a 50% increase from same period prior year Ended Q2 with over $80M in cash, cash equivalents and short-term investments Tightens full-year 2023 guidance: total net revenue of $145M-$165M; cash utilization of $57M-$67M; 2023 year-end cash balance of $55M-$65M Remains on track to achieve cash flow breakeven by year-end 2023 Hosting conference call and webcast today at 8:30 a.m. ET 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 financial results for the second quarter and six months ended June 30, 2023 and corporate highlights. “We posted another record quarter of revenue due to the continued strong demand of all three products, as well as contributions from our partnerships,” said Paul R. Edick, Chairman and CEO of Xeris Biopharma. “Our differentiated business model, exceptional year-to-date performance, and expectations for the remainder of the year gives us the confidence to tighten our 2023 guidance by raising the low end of the total revenue range to $145 to $165 million from $135 to $165 million; reducing cash utilization range to $57 to $67 million from $57 to $77 million; improving year-end cash range to $55 to $65 million. And, we expect to be at the cash flow breakeven point in the fourth quarter and continue being a self-sustaining enterprise.” Second Quarter 2023 Highlights Three Months Ended June 30, Change 2023 2022 $ % Product revenue (in thousands): Gvoke $ 15,638 $ 11,479 $ 4,159 36.2 Keveyis 14,088 12,812 1,276 10.0 Recorlev 7,167 969 6,198 nm Product revenue, net 36,893 25,260 11,633 46.1 Royalty, contract and other revenue 1,115 46 1,069 nm Total revenue $ 38,008 $ 25,306 $ 12,702 50.2 nm - not meaningful Commercial Products Gvoke®: Second quarter 2023 net revenue was $15.6 million as compared to $11.5 million in the second quarter of 2022 – an increase of approximately 36%. Gvoke prescriptions topped 51,000 for the first time, growing 50% compared to the same period in 2022. Gvoke’s market share of the retail TRx glucagon market grew to approximately 29% at the end of July. Keveyis®: Second quarter net revenue was $14.1 million – an increase of 10% compared to the same period in 2022. The average number of patients on Keveyis increased approximately 11% from the same period in 2022. Recorlev®: Second quarter net revenue was $7.2 million – an increase of $6.2 million compared to the second quarter of 2022, the first full quarter since its launch. The average number of patients on Recorlev increased over 400% from the same period in 2022. Pipeline Program XeriSol® levothyroxine (XP-8121): Xeris began dosing patients in its Phase 2 clinical study in the second quarter. The Company anticipates completing the study in the first half of 2024. Year-to-Date 2023 Financial Results Six Months Ended June 30, Change 2023 2022 $ % Product revenue (in thousands): Gvoke $ 30,671 $ 23,932 $ 6,739 28.2 Keveyis 26,843 22,136 4,707 21.3 Recorlev 11,644 1,102 10,542 nm Product revenue, net 69,158 47,170 21,988 46.6 Royalty, contract and other revenue 2,046 209 1,837 nm Total revenue $ 71,204 $ 47,379 $ 23,825 50.3 nm - not meaningful Gvoke®: Net revenue was $30.7 million for the six months ended June 30, 2023, a 28% increase compared to prior year. Gvoke prescriptions for the first half of 2023 were over 97,000 TRx’s, growing 50% compared to the same period in 2022. The growth in product demand was partially offset by a decrease in net pricing. Keveyis®: Net revenue was $26.8 million for the six months ended June 30, 2023, a 21% increase from last year. This increase was driven by a 9% increase in sales volume and an increase in net pricing. Recorlev®: Net revenue was $11.6 million for the six months ended June 30,2023, a $10.5 million increase from last year, driven primarily by increases in the number of patients on therapy. Cost of goods sold increased by $2.7 million or 57.1% for the three months ended June 30, 2023 compared to the same period ended June 30, 2022. The increase was attributable to higher product sales and product mix. Cost of goods sold increased by $1.8 million or 16.2% for the six months ended June 30, 2023 compared to the same period ended June 30, 2022. The increase was attributable to higher product sales and product mix, partially offset by a one-time contract credit in the first quarter of 2023. Research and development expenses increased by $2.4 million or 63.7% and $1.0 million or 9.6% for the three and six months ended June 30, 2023, respectively, compared to the same periods ended June 30, 2022. The increases in both periods were due to higher spending on the Open-label Treatment in Cushing’s Syndrome (OPTICS) study for Recorlev and higher product development costs. Selling, general and administrative expenses increased by $4.7 million or 14.1% and $2.3 million or 3.4% for the three and six months ended 2023, respectively, compared to the same periods ended June 30, 2022, due to higher personnel costs, marketing expenses and rent expenses related to the new lease commenced in April 2023. Net Loss for the three months ended June 30, 2023, was $19.8 million, or $0.14 per share, and a net loss of $36.7 million, or $0.27 per share, for the six months ended June 30, 2023. Cash, cash equivalents, and short-term investments at June 30, 2023 was $80.7 million compared to $122.0 million at December 31, 2022. Total shares outstanding at July 31, 2023 was 138,065,993. Conference Call and Webcast Details Xeris will host a conference call and webcast today, Tuesday, August 8, 2023, at 8:30 a.m. Eastern Time. To pre-register for the conference call please use this link: After registering, a confirmation email will be sent, including dial-in details and a unique code for entry. The Company recommends registering a minimum of ten minutes prior to the start of the call. Following the conference call, a replay will be available until Tuesday, August 22, 2023 at US:1 929 458 6194, US Toll Free: 1 866 813 9403, UK: 0204 525 0658, Canada: 1 226 828 7578, or all other locations: +44 204 525 0658 Access Code: 729515. In addition, a live audio of the conference call will be available as a webcast. To join the webcast, please visit “Events” on investor relations page of the Company’s website at or use this link: About Xeris Xeris (Nasdaq: XERS) is a growth-oriented biopharmaceutical company committed to improving patient 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®, the first and only FDA-approved 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 Twitter, LinkedIn, or Instagram. Forward-Looking Statements Any statements in this press release about future expectations, plans and prospects for Xeris Biopharma Holdings, Inc. including statements regarding the financial outlook for 2023, including projections regarding year-end 2023 cash estimates and total revenue, the Company’s expectations regarding its cash flow breakeven timeline, the market and therapeutic potential of its products and product candidates, continued growth of Gvoke, Keveyis and Recorlev, continued use of Xeris’ formulation sciences in development and partnered programs, potentially new partnerships and collaborations, expectations regarding clinical data or results from planned clinical trials, the timing of clinical trials, including advancement of its levothyroxine clinical program and a related Phase 2 study results in the first half of 2024, the potential utility of its formulation platforms, cash management, becoming a self-sustaining enterprise, and other statements containing the words “will,” “would,” “continue,” “expect,” “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 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 information about potential impacts of COVID-19, 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 Annual Report on Form 10-K 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 believed to be 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. XERIS BIOPHARMA HOLDINGS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share data) (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Product revenue, net $ 36,893 $ 25,260 $ 69,158 $ 47,170 Royalty, contract and other revenue 1,115 46 2,046 209 Total revenue 38,008 25,306 71,204 47,379 Costs and expenses: Cost of goods sold 7,555 4,810 12,874 11,083 Research and development 6,087 3,718 10,925 9,968 Selling, general and administrative 37,635 32,984 71,240 68,897 Amortization of intangible assets 2,710 2,710 5,421 5,421 Total costs and expenses 53,987 44,222 100,460 95,369 Loss from operations (15,979 ) (18,916 ) (29,256 ) (47,990 ) Other expense (4,538 ) (7,608 ) (8,095 ) (12,656 ) Net loss before benefit from income taxes (20,517 ) (26,524 ) (37,351 ) (60,646 ) Benefit from income taxes 675 339 675 747 Net loss $ (19,842 ) $ (26,185 ) $ (36,676 ) $ (59,899 ) Net loss per common share - basic and diluted $ (0.14 ) $ (0.19 ) $ (0.27 ) $ (0.44 ) Weighted average common shares outstanding - basic and diluted 137,338,071 135,529,968 137,250,465 135,282,749 XERIS BIOPHARMA HOLDINGS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands) June 30, 2023 December 31, 2022 (unaudited) Assets Current assets: Cash and cash equivalents $ 46,170 $ 121,966 Short-term investments 34,498 — Trade accounts receivable, net 30,225 30,830 Inventory 36,538 24,735 Prepaid expenses and other current assets 8,310 9,287 Total current assets 155,741 186,818 Property and equipment, net 6,552 5,516 Intangible assets, net 115,186 120,607 Goodwill 22,859 22,859 Operating lease right-of-use assets 23,632 3,992 Other assets 4,808 4,730 Total assets $ 328,778 $ 344,522 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 11,621 $ 4,606 Current operating lease liabilities 1,935 1,580 Other accrued liabilities 19,413 36,786 Accrued trade discounts and rebates 17,034 16,818 Accrued returns reserve 11,320 11,173 Current portion of contingent value rights 16,637 — Other current liabilities 1,718 2,658 Total current liabilities 79,678 73,621 Long-term debt, net of unamortized debt issuance costs 188,182 187,075 Non-current contingent value rights 6,911 25,688 Non-current operating lease liabilities 34,871 9,402 Deferred tax liabilities 2,843 3,518 Other liabilities 2,652 31 Total liabilities 315,137 299,335 Total stockholders’ equity 13,641 45,187 Total liabilities and stockholders’ equity $ 328,778 $ 344,522