U.S. Food and Drug Administration Approves Perioperative Treatment of Neoadjuvant Opdivo® (nivolumab) and Chemotherapy Followed by Surgery and Adjuvant Single-Agent Opdivo for Resectable Non-Small Cell Lung Cancer (NSCLC)




U.S. Food and Drug Administration Approves Perioperative Treatment of Neoadjuvant Opdivo® (nivolumab) and Chemotherapy Followed by Surgery and Adjuvant Single-Agent Opdivo for Resectable Non-Small Cell Lung Cancer (NSCLC)

Approval is based on the CheckMate-77T trial, in which the Opdivo-based regimen demonstrated significantly longer event-free survival compared to the chemotherapy and placebo arm; a high pathologic complete response rate was also observed¹

Opdivo is the only approved PD-1 inhibitor for resectable NSCLC in both a neoadjuvant-only regimen and as part of a perioperative treatment regimen¹

This milestone adds to Bristol Myers Squibb’s thoracic portfolio and highlights the company’s commitment to advancing treatments for patients with early-stage disease

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #CheckMate—Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo^® (nivolumab) for the treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements, for neoadjuvant treatment, in combination with platinum-doublet chemotherapy, followed by single-agent Opdivo as adjuvant treatment after surgery – otherwise referred to as perioperative therapy, which is used before and after surgery.¹ The approval is based on results from the CheckMate-77T trial, the company’s second positive Phase 3 randomized trial with an immunotherapy-based combination for the treatment of resectable NSCLC.¹ Opdivo is now the only PD-1 inhibitor to demonstrate statistically significant and clinically meaningful benefits in this disease versus chemotherapy in both a neoadjuvant-only regimen and as part of a perioperative regimen.¹

“Given the rates of disease recurrence in patients with resectable NSCLC, there is a clear need for options that can be administered before and after surgery that may target micrometastasis, help reduce the risk of cancer returning and improve the chance of successful surgical treatment,” said Tina Cascone, MD, PhD, associate professor of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center.^2,3,4 “This approval is a step forward for patients with resectable disease, as the perioperative nivolumab plus neoadjuvant chemotherapy regimen can offer an improved event free survival (EFS) compared with neoadjuvant chemotherapy alone and has the potential for achieving a pathologic response (pCR) in one in four patients.”²

The CheckMate-77T trial evaluated the perioperative regimen of neoadjuvant Opdivo with platinum-doublet chemotherapy followed by surgery and adjuvant Opdivo monotherapy (n=229), compared to neoadjuvant platinum-doublet chemotherapy and placebo followed by surgery and adjuvant placebo (n=232) in adult patients with resectable NSCLC.² In the trial, the Opdivo arm improved EFS, a primary endpoint, compared to the chemotherapy and placebo treatment arm.² A high pCR rate was also observed as one of the pre-specified secondary endpoints.²

The risk of disease recurrence, progression or death was reduced by 42% (EFS Hazard Ratio [HR] 0.58; 95% Confidence Interval [CI]: 0.43 to 0.78; P=0.00025) in patients treated in the Opdivo arm, compared to the chemotherapy and placebo arm, with a median follow-up of 25.4 months.² In addition, 18-month EFS was demonstrated in 70% of patients in the Opdivo arm, compared to 50% of patients in the chemotherapy and placebo arm.² Furthermore, 25% of patients in the Opdivo arm achieved pCR, while 4.7% of patients in the comparator arm achieved pCR in the intent-to-treat population (estimated treatment difference of 20.5%; 95% CI,14.3 to 26.6).²

Opdivo is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, dermatologic adverse reactions, nephritis and renal dysfunction; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity.¹ Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue and dexamethasone is not recommended outside of controlled clinical trials.¹ Please see Important Safety Information below.

“This milestone expands the role of Opdivo-based treatments and builds upon the foundation set by the FDA approval of neoadjuvant-only Opdivo plus chemotherapy in resectable NSCLC based on the CheckMate-816 trial,” said Wendy Short Bartie, senior vice president of U.S. Oncology and Hematology at Bristol Myers Squibb.¹ “With this new Opdivo-based regimen, we are reinforcing our commitment to helping improve patient outcomes and expanding our thoracic portfolio in early-stage disease.”

The recommended dose for Opdivo in this indication is 360 mg with platinum-doublet chemotherapy on the same day every three weeks for up to four cycles or until disease progression or unacceptable toxicity, then continued as a single-agent Opdivo 480 mg every four weeks after surgery for up to 13 cycles (approximately one year) or until disease recurrence or unacceptable toxicity.¹ The FDA previously approved Opdivo for adult patients with resectable (tumors ≥4 cm or node positive) NSCLC in the neoadjuvant setting, in combination with platinum-doublet chemotherapy.¹ Opdivo and Opdivo-based combinations have been approved by the FDA in the neoadjuvant, adjuvant or perioperative settings across four cancers to date, including lung cancer, melanoma, bladder cancer and esophageal/gastroesophageal junction cancer.¹

About CheckMate-77T

CheckMate-77T is a Phase 3 randomized, double-blind, multi-center trial evaluating neoadjuvant Opdivo in combination with platinum-doublet chemotherapy followed by surgery and single-agent adjuvant Opdivo, compared to neoadjuvant platinum-doublet chemotherapy and placebo followed by surgery and adjuvant placebo in patients with resectable NSCLC.⁵

In the CheckMate-77T study, a total of 461 patients were randomized to receive either neoadjuvant Opdivo 360 mg with platinum-doublet chemotherapy every three weeks, or placebo and platinum-doublet chemotherapy every three weeks, until disease progression or unacceptable toxicity, for up to four cycles, followed by single-agent Opdivo 480 mg after surgery every four weeks or placebo every four weeks, until disease progression or unacceptable toxicity, for up to thirteen cycles (approximately one year).¹ The primary endpoint of the trial is event-free survival determined by Blinded Independent Central Review (BICR). Secondary endpoints of the trial include pathologic complete response and major pathologic response, both determined by Blinded Independent Pathological Review (BIPR), as well as overall survival and safety.²

Select Safety Profile from CheckMate-77T

The most common adverse reactions (reported in ≥20%) in patients receiving Opdivo in combination with chemotherapy (n= 228) were anemia (39.5%), constipation (32.0%), nausea (28.9%), fatigue (28.1%), alopecia (25.9%), and cough (21.9%).⁶

Serious adverse reactions occurred in 21% of patients who received Opdivo in combination with platinum-doublet chemotherapy as neoadjuvant treatment (n=228).¹ The most frequent (≥2%) serious adverse reaction was pneumonia.¹ Fatal adverse reactions occurred in 2.2% of patients, due to cerebrovascular accident, COVID-19 infection, hemoptysis, pneumonia, and pneumonitis (0.4% each).¹

In Checkmate 77T, 5.3% (n=12) of the OPDIVO-treated patients who received neoadjuvant treatment, did not receive surgery due to adverse reactions. The adverse reactions that led to cancellation of surgery in OPDIVO-treated patients were cerebrovascular accident, pneumonia, and colitis/diarrhea (2 patients each) and acute coronary syndrome, myocarditis, hemoptysis, pneumonitis, COVID-19, and myositis (1 patient each).

Serious adverse reactions occurred in 22% of the patients who received single-agent Opdivo as adjuvant treatment (n=142).¹ The most frequent serious adverse reaction was pneumonitis/ILD (2.8%).¹ One fatal adverse event due to COVID-19 occurred.¹ The perioperative regimen had a safety profile consistent with previously reported Opdivo studies in NSCLC and no new safety signals were identified.²

About Lung Cancer

Lung cancer is the leading cause of cancer deaths in the United States.⁷ The two main types of lung cancer are non-small cell and small cell.⁷ Non-small cell lung cancer (NSCLC) represents up to 85% of diagnoses.⁷ For some non-metastatic early-stage NSCLC patients, surgery may be able to be used as a singular option for treatment.⁸ However, 30% to 55% of patients can develop recurrence, contributing to a need for treatment options administered before surgery (neoadjuvant) and after surgery (adjuvant) to improve long-term outcomes.² Survival rates vary depending on the stage and type of the cancer when diagnosed.⁷

INDICATIONS

OPDIVO^® (nivolumab) is indicated for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stage IIB, Stage IIC, Stage III, or Stage IV melanoma.

OPDIVO^® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).

OPDIVO^® (nivolumab) in combination with platinum-doublet chemotherapy, is indicated for neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements, followed by single-agent OPDIVO^® as adjuvant treatment after surgery.

OPDIVO^® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.

OPDIVO^® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).

IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur after discontinuation of OPDIVO. Early identification and management are essential to ensure safe use of OPDIVO. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment with OPDIVO. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if OPDIVO interruption or discontinuation is required, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below.

Immune-Mediated Pneumonitis

OPDIVO can cause immune-mediated pneumonitis. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. In patients receiving OPDIVO monotherapy, immune-mediated pneumonitis occurred in 3.1% (61/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.9%), and Grade 2 (2.1%).

Immune-Mediated Colitis

OPDIVO can cause immune-mediated colitis. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid- refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients, including Grade 3 (1.7%) and Grade 2 (1%).

Immune-Mediated Hepatitis and Hepatotoxicity

OPDIVO can cause immune-mediated hepatitis. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients, including Grade 4 (0.2%), Grade 3 (1.3%), and Grade 2 (0.4%).

Immune-Mediated Endocrinopathies

OPDIVO can cause primary or secondary adrenal insufficiency, immune-mediated hypophysitis, immune-mediated thyroid disorders, and Type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Withhold OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism; initiate hormone replacement as clinically indicated. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism; initiate hormone replacement or medical management as clinically indicated. Monitor patients for hyperglycemia or other signs and symptoms of diabetes; initiate treatment with insulin as clinically indicated.

In patients receiving OPDIVO monotherapy, adrenal insufficiency occurred in 1% (20/1994), including Grade 3 (0.4%) and Grade 2 (0.6%).

In patients receiving OPDIVO monotherapy, hypophysitis occurred in 0.6% (12/1994) of patients, including Grade 3 (0.2%) and Grade 2 (0.3%).

In patients receiving OPDIVO monotherapy, thyroiditis occurred in 0.6% (12/1994) of patients, including Grade 2 (0.2%).

In patients receiving OPDIVO monotherapy, hyperthyroidism occurred in 2.7% (54/1994) of patients, including Grade 3 (<0.1%) and Grade 2 (1.2%).

In patients receiving OPDIVO monotherapy, hypothyroidism occurred in 8% (163/1994) of patients, including Grade 3 (0.2%) and Grade 2 (4.8%).

In patients receiving OPDIVO monotherapy, diabetes occurred in 0.9% (17/1994) of patients, including Grade 3 (0.4%) and Grade 2 (0.3%), and 2 cases of diabetic ketoacidosis.

Immune-Mediated Nephritis with Renal Dysfunction

OPDIVO can cause immune-mediated nephritis. In patients receiving OPDIVO monotherapy, immune-mediated nephritis and renal dysfunction occurred in 1.2% (23/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.5%), and Grade 2 (0.6%).

Immune-Mediated Dermatologic Adverse Reactions

OPDIVO can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug rash with eosinophilia and systemic symptoms (DRESS) has occurred with PD-1/PD-L1 blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes.

Withhold or permanently discontinue OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

In patients receiving OPDIVO monotherapy, immune-mediated rash occurred in 9% (171/1994) of patients, including Grade 3 (1.1%) and Grade 2 (2.2%).

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received OPDIVO monotherapy or were reported with the use of other PD-1/PD-L1 blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions: cardiac/vascular: myocarditis, pericarditis, vasculitis; nervous system: meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barré syndrome, nerve paresis, autoimmune neuropathy; ocular: uveitis, iritis, and other ocular inflammatory toxicities can occur; gastrointestinal: pancreatitis to include increases in serum amylase and lipase levels, gastritis, duodenitis; musculoskeletal and connective tissue: myositis/polymyositis, rhabdomyolysis, and associated sequelae including renal failure, arthritis, polymyalgia rheumatica; endocrine: hypoparathyroidism; other (hematologic/immune): hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis (HLH), systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection, other transplant (including corneal graft) rejection.

Some ocular IMAR cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada–like syndrome, which has been observed in patients receiving OPDIVO, as this may require treatment with systemic corticosteroids to reduce the risk of permanent vision loss.

Infusion-Related Reactions

OPDIVO can cause severe infusion-related reactions. Discontinue OPDIVO in patients with severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Interrupt or slow the rate of infusion in patients with mild (Grade 1) or moderate (Grade 2) infusion-related reactions. In patients receiving OPDIVO monotherapy as a 60-minute infusion, infusion-related reactions occurred in 6.4% (127/1994) of patients. In a separate trial in which patients received OPDIVO monotherapy as a 60-minute infusion or a 30-minute infusion, infusion-related reactions occurred in 2.2% (8/368) and 2.7% (10/369) of patients, respectively. Additionally, 0.5% (2/368) and 1.4% (5/369) of patients, respectively, experienced adverse reactions within 48 hours of infusion that led to dose delay, permanent discontinuation or withholding of OPDIVO.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with OPDIVO. Transplant-related complications include hyperacute graft-versus-host-disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between OPDIVO and allogeneic HSCT.

Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with OPDIVO prior to or after an allogeneic HSCT.

Embryo-Fetal Toxicity

Based on its mechanism of action and findings from animal studies, OPDIVO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with OPDIVO and for at least 5 months after the last dose.

Increased Mortality in Patients with Multiple Myeloma when OPDIVO is Added to a Thalidomide Analogue and Dexamethasone

In randomized clinical trials in patients with multiple myeloma, the addition of OPDIVO to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Lactation

There are no data on the presence of OPDIVO in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment and for 5 months after the last dose.

Serious Adverse Reactions

In Checkmate 238, serious adverse reactions occurred in 18% of patients receiving OPDIVO (n=452). Grade 3 or 4 adverse reactions occurred in 25% of OPDIVO-treated patients (n=452). The most frequent Grade 3 and 4 adverse reactions reported in ≥2% of OPDIVO-treated patients were diarrhea and increased lipase and amylase. In Checkmate 816, serious adverse reactions occurred in 30% of patients (n=176) who were treated with OPDIVO in combination with platinum-doublet chemotherapy. Serious adverse reactions in >2% included pneumonia and vomiting. No fatal adverse reactions occurred in patients who received OPDIVO in combination with platinum-doublet chemotherapy. In Checkmate 77T, serious adverse reactions occurred in 21% of patients who received OPDIVO in combination with platinum-doublet chemotherapy as neoadjuvant treatment (n=228). The most frequent (≥2%) serious adverse reactions was pneumonia. Fatal adverse reactions occurred in 2.2% of patients, due to cerebrovascular accident, COVID-19 infection, hemoptysis, pneumonia, and pneumonitis (0.4% each). In the adjuvant phase of Checkmate 77T, 22% of patients experienced serious adverse reactions (n=142). The most frequent serious adverse reaction was pneumonitis/ILD (2.8%). One fatal adverse reaction due to COVID-19 occurred. In Checkmate 274, serious adverse reactions occurred in 30% of patients receiving OPDIVO (n=351). The most frequent serious adverse reaction reported in ≥2% of patients receiving OPDIVO was urinary tract infection. Fatal adverse reactions occurred in 1% of patients; these included events of pneumonitis (0.6%). In Checkmate 577, serious adverse reactions occurred in 33% of patients receiving OPDIVO (n=532). A serious adverse reaction reported in ≥2% of patients who received OPDIVO was pneumonitis. A fatal reaction of myocardial infarction occurred in one patient who received OPDIVO. In Checkmate 76K, serious adverse reactions occurred in 18% of patients receiving OPDIVO (n=524). Adverse reactions which resulted in permanent discontinuation of OPDIVO in >1% of patients included arthralgia (1.7%), rash (1.7%), and diarrhea (1.1%). A fatal adverse reaction occurred in 1 (0.2%) patient (heart failure and acute kidney injury). The most frequent Grade 3-4 lab abnormalities reported in ≥1% of OPDIVO-treated patients were increased lipase (2.9%), increased AST (2.2%), increased ALT (2.1%), lymphopenia (1.1%), and decreased potassium (1.0%).

Common Adverse Reactions

In Checkmate 238, the most common adverse reactions (≥20%) reported in OPDIVO-treated patients (n=452) vs ipilimumab-treated patients (n=453) were fatigue (57% vs 55%), diarrhea (37% vs 55%), rash (35% vs 47%), musculoskeletal pain (32% vs 27%), pruritus (28% vs 37%), headache (23% vs 31%), nausea (23% vs 28%), upper respiratory infection (22% vs 15%), and abdominal pain (21% vs 23%).

Contacts

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Media Inquiries:
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investor.relations@bms.com

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AngioDynamics Reports Fiscal Year 2025 First Quarter Financial Results




AngioDynamics Reports Fiscal Year 2025 First Quarter Financial Results

LATHAM, N.Y.–(BUSINESS WIRE)–AngioDynamics, Inc. (NASDAQ: ANGO), a leading and transformative medical technology company focused on restoring healthy blood flow in the body’s vascular system, expanding cancer treatment options, and improving quality of life for patients, today announced financial results for the first quarter of fiscal year 2025, which ended August 31, 2024.

Fiscal Year 2025 First Quarter Highlights

• GAAP gross margin of 54.4%
• GAAP loss per share of $0.31
• Adjusted loss per share of $0.11
• Submitted for FDA 510(k) clearance for Prostate Tissue indication for NanoKnife
• Received CE Mark Approval in Europe for the Auryon System
• Initiated RECOVER-AV Clinical Trial in Europe for AlphaVac

*Pro forma results exclude the Dialysis and BioSentry businesses divested in June 2023 and the PICC and Midline product portfolios divested in February 2024, as well as the discontinued Radiofrequency and Syntrax products in February 2024.

“We are pleased with our strong start to fiscal year 2025, particularly in our Med Tech segment, with Auryon and AlphaVac both delivering over 20% growth in the quarter,” commented Jim Clemmer, President and Chief Executive Officer of AngioDynamics, Inc. “We continue to view 2025 as an inflection point in the trajectory of our business. We expect to continue to deliver strong revenue growth within our Med Tech business as we execute on key commercial initiatives. We remain focused on executing our growth strategy and advancing our innovative product portfolio.”

Fiscal Year 2025 First Quarter Financial Results

Unless otherwise noted, all financial metrics and growth rates presented below are on a pro forma basis.

Net sales for the first quarter of fiscal year 2025 were $67.5 million, an increase of 1.1% compared to the prior-year quarter.

Med Tech net sales were $28.0 million, an 8.7% increase from $25.7 million in the prior-year period. Med Tech includes the Auryon peripheral atherectomy platform, the thrombus management platform, which includes the AlphaVac and AngioVac mechanical thrombectomy systems, and the NanoKnife irreversible electroporation platform.

Growth was driven by Auryon sales during the quarter of $13.7 million, which increased 24.9% and AlphaVac sales of $2.2 million, an increase of 21.1% over the prior year. NanoKnife sales were $5.1 million during the quarter, a decrease of 6.9% compared to the prior year period, primarily due to the timing of international orders during last year.

Med Device net sales were $39.5 million, a decrease of 3.6% compared to $41.0 million in the prior-year period. U.S. net sales of Med Device products grew 2.1% during the first quarter compared to last year.

U.S. net sales in the first quarter of fiscal 2025 were $59.5 million, an increase of 6.2% from $56.0 million a year ago. International net sales were $8.0 million, a decrease of 25.4%, compared to $10.7 million a year ago, primarily due to the timing of international orders during last year.

Gross margin for the first quarter of fiscal 2025 was 54.4%, which was 40 basis points down compared to the first quarter of fiscal 2024, and 10 basis points sequentially up from 54.3% in the fourth quarter of fiscal 2024.

Gross margin for the Med Tech business was 63.3%, a decrease of 160 basis points from the first quarter of fiscal 2024 due to increased capital placements and inflationary costs. Gross margin for the Med Device business was 48.2%, a decrease of 40 basis points compared to the first quarter of fiscal 2024 due to inflationary pressures and costs associated with the transition to outsourced manufacturing.

The Company recorded a GAAP net loss of $12.8 million, or a loss per share of $0.31, in the first quarter of fiscal 2025. Excluding the items shown in the non-GAAP reconciliation table below, adjusted net loss for the first quarter of fiscal 2025 was $4.4 million, or a loss per share of $0.11. This compares to an adjusted net loss during the fiscal first quarter of 2024 of $6.2 million, or a loss per share of $0.16.

Adjusted EBITDA in the first quarter of fiscal 2025, excluding the items shown in the non-GAAP reconciliation table below, was $(0.2) million, compared to $(1.1) million in the first quarter of fiscal 2024.

In the first quarter of fiscal 2025, the Company used $18.3 million in operating cash. The Company’s first fiscal quarter has historically exhibited the highest utilization of cash and the first quarter of fiscal 2025 was in line with the Company’s expectations.

At August 31, 2024, the Company had $55.0 million in cash and cash equivalents compared to $76.1 million in cash and cash equivalents at May 31, 2024.

NanoKnife System’s PRESERVE Study Results Submitted for FDA 510(k) Clearance

In September, the Company submitted results from its Pivotal Study of the NanoKnife System for Ablation of Prostate Tissue in an Intermediate-Risk Patient Population (PRESERVE) to the U.S. Food and Drug Administration (FDA) for 510(k) indication of its NanoKnife System in the ablation of prostate tissue in an intermediate-risk population. The comprehensive study enrolled and treated 121 patients across 17 facilities throughout the United States.

CE Mark Approval in Europe for the Auryon System

Prior to the end of the quarter AngioDynamics received European CE Mark approval for its Auryon Atherectomy System. This regulatory approval allows AngioDynamics to market the Auryon System in Europe for the treatment of Peripheral Artery Disease (PAD), including Critical Limb Ischemia (CLI) and In-Stent Restenosis (ISR). The Auryon System uses solid-state laser technology to treat PAD lesions and occlusions. It has been cleared by the FDA since 2020 and has treated over 50,000 patients in the United States. The system is designed to treat lesions of various types, lengths, and locations, both above and below the knee. This CE Mark approval expands AngioDynamics’ potential market reach, as the global PAD market is valued at $1.1 billion.

RECOVER-AV Clinical Trial

Subsequent to the end of the first fiscal quarter, the Company initiated its RECOVER-AV clinical trial, marking a significant step in evaluating the AlphaVac F18⁸⁵ System for treating acute, intermediate-risk pulmonary embolism (PE) in the European market. This multi-center, multi-national study will assess the efficacy, safety, and long-term functional outcomes of the system across up to 20 hospital sites in Europe. Following the successful APEX-AV study in the United States, RECOVER-AV aims to further demonstrate the system’s capabilities in a region where PE prevalence is notably higher. The trial will track patient outcomes over a 12-month period, focusing on key efficacy and safety endpoints.

Fiscal Year 2025 Financial Guidance

For fiscal year 2025, the Company continues to expect:

• Net sales to be in the range of $282 to $288 million, representing growth of between 4.2% – 6.4% over fiscal 2024 pro forma revenue of $270.7 million
• Med Tech net sales are expected to grow in the range of 10% to 12%
• Med Device net sales are expected to grow in the range of 1% to 3%
• Gross margin to be approximately 52% to 53%
• Adjusted EBITDA loss of $2.5 million to $0, compared to a pro forma adjusted EBITDA loss of $3.2 million in fiscal year 2024
• Adjusted loss per share in the range of $0.38 to $0.42, compared to pro forma adjusted loss per share of $0.45 in fiscal year 2024

Conference Call

The Company’s management will host a conference call at 8:00 a.m. ET the same day to discuss the results. To participate in the conference call, dial 1-877-407-0784 (domestic) or +1-201-689-8560 (international).

This conference call will also be webcast and can be accessed from the “Investors” section of the AngioDynamics website at www.angiodynamics.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A recording of the call will also be available, until Thursday, October 10, 2024 at 11:59 PM ET. To hear this recording, dial 1-844-512-2921 (domestic) or +1-412-317-6671 (international) and enter the passcode 13748896.

Use of Non-GAAP Measures

Management uses non-GAAP measures to establish operational goals and believes that non-GAAP measures may assist investors in analyzing the underlying trends in AngioDynamics’ business over time. Investors should consider these non-GAAP measures in addition to, not as a substitute for or as superior to, financial reporting measures prepared in accordance with GAAP. In this news release, AngioDynamics has reported pro forma results, adjusted EBITDA, adjusted net income and adjusted earnings per share. Management uses these measures in its internal analysis and review of operational performance. Management believes that these measures provide investors with useful information in comparing AngioDynamics’ performance over different periods. By using these non-GAAP measures, management believes that investors get a better picture of the performance of AngioDynamics’ underlying business. Management encourages investors to review AngioDynamics’ financial results prepared in accordance with GAAP to understand AngioDynamics’ performance taking into account all relevant factors, including those that may only occur from time to time but have a material impact on AngioDynamics’ financial results. Please see the tables that follow for a reconciliation of non-GAAP measures to measures prepared in accordance with GAAP.

About AngioDynamics, Inc.

AngioDynamics is a leading and transformative medical technology company focused on restoring healthy blood flow in the body’s vascular system, expanding cancer treatment options and improving quality of life for patients.

The Company’s innovative technologies and devices are chosen by talented physicians in fast-growing healthcare markets to treat unmet patient needs. For more information, visit www.angiodynamics.com.

Safe Harbor

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements regarding AngioDynamics’ expected future financial position, results of operations, cash flows, business strategy, budgets, projected costs, capital expenditures, products, competitive positions, growth opportunities, plans and objectives of management for future operations, as well as statements that include the words such as “expects,” “reaffirms,” “intends,” “anticipates,” “plans,” “believes,” “seeks,” “estimates,” “projects,” “optimistic,” or variations of such words and similar expressions, are forward-looking statements. These forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties. Investors are cautioned that actual events or results may differ materially from AngioDynamics’ expectations, expressed or implied. Factors that may affect the actual results achieved by AngioDynamics include, without limitation, the scale and scope of the COVID-19 global pandemic, the ability of AngioDynamics to develop its existing and new products, technological advances and patents attained by competitors, infringement of AngioDynamics’ technology or assertions that AngioDynamics’ technology infringes the technology of third parties, the ability of AngioDynamics to effectively compete against competitors that have substantially greater resources, future actions by the FDA or other regulatory agencies, domestic and foreign health care reforms and government regulations, results of pending or future clinical trials, overall economic conditions (including inflation, labor shortages and supply chain challenges including the cost and availability of raw materials), the results of on-going litigation, challenges with respect to third-party distributors or joint venture partners or collaborators, the results of sales efforts, the effects of product recalls and product liability claims, changes in key personnel, the ability of AngioDynamics to execute on strategic initiatives, the effects of economic, credit and capital market conditions, general market conditions, market acceptance, foreign currency exchange rate fluctuations, the effects on pricing from group purchasing organizations and competition, the ability of AngioDynamics to obtain regulatory clearances or approval of its products, or to integrate acquired businesses, as well as the risk factors listed from time to time in AngioDynamics’ SEC filings, including but not limited to its Annual Report on Form 10-K for the year ended May 31, 2024. AngioDynamics does not assume any obligation to publicly update or revise any forward-looking statements for any reason.

In the United States, the NanoKnife System has received a 510(k) clearance by the Food and Drug Administration for use in the surgical ablation of soft tissue and is similarly approved for commercialization in Canada, the European Union and Australia. The NanoKnife System has not been cleared for the treatment or therapy of a specific disease or condition.

Contacts

Investors:

AngioDynamics, Inc.

Stephen Trowbridge, Executive Vice President & CFO

(518) 795-1408

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Ferring Pharmaceuticals bolsters global gene therapy supply chain with European manufacturing facility




Ferring Pharmaceuticals bolsters global gene therapy supply chain with European manufacturing facility

• This milestone represents significant progress in Ferring’s manufacturing capabilities to meet anticipated growth in demand for Adstiladrin^® (nadofaragene firadenovec-vncg), a gene therapy for people with non-muscle invasive bladder cancer (NMIBC).

• With bladder cancer now the ninth most diagnosed cancer around the world ¹, securing the global supply of this innovative bladder-sparing treatment is vital.

SAINT-PREX, Switzerland–(BUSINESS WIRE)–Ferring Pharmaceuticals today announced the opening of a state-of-the-art global manufacturing hub in Finland for the drug substance of its intravesical non-replicating gene therapy Adstiladrin^® (nadofaragene firadenovec-vncg). This represents a significant milestone in Ferring’s capabilities and capacity to meet the current and expected growth in demand for this gene therapy for people with non-muscle invasive bladder cancer (NMIBC).

Adstiladrin^® (nadofaragene firadenovec-vncg) is approved in the U.S. for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours.

“In January this year, we announced full commercial availability of Adstiladrin^® in the U.S. We have now embarked on a broad programme of clinical research to enlarge the body of evidence for Adstiladrin^® and plan for regulatory submissions in other global markets. Today’s announcement marks an important milestone in ensuring stable and sustainable global supply of Adstiladrin^® to meet the anticipated growth in demand,” said Bipin Dalmia, Global Head, Uro-Oncology and Urology Franchise, Ferring Pharmaceuticals.

Armin Metzger, Executive Vice President and Chief Technical Operations Officer, Ferring Pharmaceuticals added, “The continued investment and expansion of our gene therapy manufacturing infrastructure reflects Ferring’s strong commitment to deliver end-to-end solutions for even the most highly complex manufacturing challenges as part of our mission to fulfil the unmet clinical needs for people with bladder cancer.”

According to the International Agency for Research on Cancer, bladder cancer is now the ninth most diagnosed cancer worldwide, previously ranked tenth.¹ This rising global incidence underscores the necessity to expand the supply of innovative treatments for this disease.

In addition to this European investment in drug substance, Ferring has further strengthened and diversified its nadofaragene firadenovec-vncg supply chain and is also nearing completion of a new manufacturing facility for drug product at Ferring’s U.S. campus in Parsippany, New Jersey. The bolstering of Ferring’s gene therapy manufacturing capabilities and footprint reinforces its commitment to ensuring the future supply of this therapy for patients.

Located in Kuopio, Finland, the new European manufacturing site features a cutting-edge manufacturing suite, fully integrated with modern technology to produce adenovirus vector-based gene therapy drug substance in large quantities. The 25,000 square metre facility features renewable energy solutions such as waste heat recovery with heat pumps and solar energy, complementing Ferring’s commitment to protect the environment by reducing its negative impact on the planet.

About Adstiladrin^® (nadofaragene firadenovec-vncg)

Adstiladrin^® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.² It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defences against the cancer.³

Adstiladrin^® (nadofaragene firadenovec-vncg) has been studied in a clinical trial programme that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).³

About non-muscle invasive bladder cancer (NMIBC)

NMIBC is a form of bladder cancer in which the tumour is not invading into or beyond the muscularis propia.⁴ Bladder cancer is the 9th most common cancer worldwide, with 614,298 new cases reported in 2022.⁵ 75% of bladder cancers present as NMIBC.⁶ In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care.⁷ Current treatment options for BCG-unresponsive patients are very limited outside of radical cystectomy (partial or complete removal of the bladder).⁷ In a patient choice experiment to determine preferred treatment options for NMIBC, 89% of patients never selected radical cystectomy as their preferred option; patients were willing to accept a 43.8% increased risk of progression and a 66.1% increase in the risk of serious side effects to increase the time to radical cystectomy from 1 year to 6 years.⁸

About gene therapy and the manufacturing process

Gene therapies aim to treat, prevent, or cure diseases by modifying or manipulating gene expression or altering the biological properties of living cells. ⁹ The concept involves techniques like adding new copies of a gene or replacing faulty genes with healthy ones.¹⁰ Over the decades, gene therapies have been applied to inherited genetic diseases, cancers, and other disorders. Viral vectors, bacterial vectors, plasmid DNA, human gene editing, and patient-derived cellular gene therapies are various forms of gene therapy.⁹ Viral vectors are particularly significant due to their high transduction efficiency and stable expression, offering potentially curative treatments with fewer applications.¹¹ Successful applications include treatments for bladder cancer, leukaemia, lymphoma, haemophilia, certain neurological disorders, retinal diseases, and metabolic disorders.^12-18

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a privately owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. We are leaders in reproductive medicine and maternal health, and in areas of gastroenterology and urology. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Ferring was founded in 1950 and employs more than 7,000 people worldwide. The company is headquartered in Saint-Prex, Switzerland, and has operating subsidiaries in more than 50 countries which markets its medicines in over 100 countries.

Learn more at www.ferring.com, or connect with us on LinkedIn, Instagram, YouTube, Facebook and X(Twitter).

© 2024 Ferring. Ferring and the Ferring Pharmaceuticals logo are trademarks of the Ferring group of companies.

References:

1. International Agency for Research on Cancer. GLOBOCAN 2022 factsheet. Available from: https://gco.iarc.who.int/media/globocan/factsheets/populations/900-world-fact-sheet.pdf [Accessed July 2024].
2. U.S. FDA. ADSTILADRIN. Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/adstiladrin [Accessed July 2024].
3. ClinicalTrials.gov. ADSTILADRIN in patients with high grade, Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). Gov Identifier: NCT02773849. Available from: https://clinicaltrials.gov/ct2/show/NCT02773849 [Accessed July 2024].
4. Matulewicz RS, Steinberg GD. Non-muscle-invasive bladder cancer: overview and contemporary treatment landscape of neoadjuvant chemoablative therapies. Rev Urol. 2020 Jul; 22(2):43-51.
5. International Agency for Research on Cancer. Cancer Today. Available from: https://gco.iarc.fr/today/en/dataviz/bars?mode=cancer&group_populations=1&key=total [Accessed July 2024].
6. Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013 May; 63(2):234-41.
7. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2021 Nov 27; 22:107-17.
8. Collacott H, Krucien N, Heidenreich S, Catto JWF, Ghatnekar O. Patient Preferences for Treatment of Bacillus Calmette-Guérin-unresponsive Non-muscle-invasive Bladder Cancer: A Cross-country Choice Experiment. Eur Urol Open Sci. 2023 Jan 31;49:92-99.
9. U.S. FDA. What is gene therapy? Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy [Accessed July 2024].
10. NIH. Gene Therapy. Available from: https://www.genome.gov/genetics-glossary/Gene-Therapy [Accessed July 2024].
11. Braendstrup P, Levine BL, Ruella M. The long road to the first FDA-approved gene therapy: chimeric antigen receptor T cells targeting CD19. Cytotherapy. 2020 Feb;22(2):57-69
12. Munis AM. Gene therapy applications of non-human lentiviral vectors. Viruses. 2020 Sep 29; 12:1106. doi: 10.3390/v12101106.
13. Miyake H, et al. Therapeutic efficacy of adenoviral-mediated p53 gene transfer is synergistically enhanced by combined use of antisense oligodeoxynucleotide targeting clusterin gene in a human bladder cancer model. Neoplasia. 2005 Feb; 7(2): 171-9. doi:10.1593/neo.04478.
14. Maude SL, Frey N, Shaw PA, et al. Chimeric antigen receptor t cells for sustained remissions in leukemia. New England Journal of Medicine. 2014 Oct 16; 371(16), 1507-1517.
15. Nathwani AC, Reiss UM, Tuddenham EGD, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. New England Journal of Medicine. 2014 Nov 20; 371(21), 1994-2004.
16. Bankiewicz, K. S., Eberling, J. L., Kohutnicka, M., et al. Convection-enhanced delivery of AAV vector in parkinsonian monkeys: In vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Experimental Neurology. 2000 Jul; 164(1), 2-14.
17. Russell, S., Bennett, J., Wellman, J. A., et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. The Lancet. 2017 Aug 26; 390(10097), 849-860.
18. Seker Yilmaz B, Gissen P. Genetic therapy approaches for ornithine transcarbamylase deficiency. Biomedicines. 2023 Aug 8; 11(8):2227. doi: 10.3390/biomedicines11082227.

Contacts

Matthew Worrall
Director of Corporate Communications and Public Affairs, Ferring Pharmaceuticals
Matthew.worrall@ferring.com

Matt Handcock
Senior Account Manager, Syneos Health Communications
Matt.handcock@syneoshealth.com

Henry Schein Opens Henry Schein Cares Foundation 2024 Relief Fund and Commits Up to $500,000 in Cash and Essential Health Care Supplies to Support the Southeast U.S.




Henry Schein Opens Henry Schein Cares Foundation 2024 Relief Fund and Commits Up to $500,000 in Cash and Essential Health Care Supplies to Support the Southeast U.S.

MELVILLE, N.Y.–(BUSINESS WIRE)–Henry Schein, Inc. (Nasdaq: HSIC) today announced the opening of the Henry Schein Cares Foundation 2024 Relief Fund to support the Southeast U.S. Contributions will help ensure critical supplies and resources can help people impacted by Hurricane Helene.

The Company and Henry Schein Cares Foundation will donate an initial $25,000 each to support relief efforts and the Company will match TSM contributions up to another $50,000, for a cash total of up to $150,000. The Company will also donate essential health care supplies up to an initial amount of $350,000 to our partner relief organizations operating on the ground, bringing Henry Schein’s total commitment to as much as $500,000.

“Team Schein is deeply saddened by the loss of life and devastation caused by Hurricane Helene throughout the Southeast U.S., and our thoughts are with all those who have been affected,” said Stanley M. Bergman, Chairman of the Board and Chief Executive Officer of Henry Schein. “In response to this emergency, we will again partner with trusted and long-standing relief and recovery charities to ensure the availability of the critical supplies and resources required to provide health care within affected communities. We offer our deepest sympathies to the people impacted by Hurricane Helene and remain committed to helping health happen in the wake of this tragedy.”

Anyone who wishes to contribute to the 2024 Relief Fund can donate by visiting www.henryschein.com/foundationdonate and selecting “2024 Relief Fund” for the gift designation.

For dental and medical professionals impacted by Hurricane Helene and in need of assistance, please call 800-999-9729, or click here to learn about the Company’s Customer Assistance Hotline.

About The Henry Schein Cares Foundation

Established in 2008, The Henry Schein Cares Foundation, Inc., fosters a rich culture of giving back to society and serving others. The Foundation advocates and supports efforts to advance health equity and empower health care professionals to promote a healthier tomorrow for all people around the world through advancing access to care, promoting a holistic model of health care, building capacity to empower health care professionals, and catalyzing innovative models for emergency preparedness & response.

The Foundation is a tax-exempt organization under Section 501(c)(3) of the Internal Revenue Code of 1986, as amended. To learn more about the work of the Henry Schein Cares Foundation, visit www.henryschein.com/hscaresfoundation.

About Henry Schein, Inc.

Henry Schein, Inc. (Nasdaq: HSIC) is a solutions company for health care professionals powered by a network of people and technology. With more than 25,000 Team Schein Members worldwide, the Company’s network of trusted advisors provides more than 1 million customers globally with more than 300 valued solutions that help improve operational success and clinical outcomes. Our Business, Clinical, Technology, and Supply Chain solutions help office-based dental and medical practitioners work more efficiently so they can provide quality care more effectively. These solutions also support dental laboratories, government and institutional health care clinics, as well as other alternate care sites.

Henry Schein operates through a centralized and automated distribution network, with a selection of more than 300,000 branded products and Henry Schein corporate brand products in our distribution centers.

A FORTUNE 500 Company and a member of the S&P 500® index, Henry Schein is headquartered in Melville, N.Y., and has operations or affiliates in 33 countries and territories. The Company’s sales reached $12.3 billion in 2023 and have grown at a compound annual rate of approximately 11.5 percent since Henry Schein became a public company in 1995.

For more information, visit Henry Schein at www.henryschein.com, Facebook.com/HenrySchein, Instagram.com/HenrySchein, LinkedIn.com/Company/HenrySchein, and @HenrySchein on X.

Contacts

Ann Marie Gothard

Vice President, Global Corporate Media Relations

annmarie.gothard@henryschein.com
(631) 390-8169

Ferring Demonstrates Effectiveness of a Treat-to-Target Approach in Mild-to-Moderate Ulcerative Colitis in First Major Study




Ferring Demonstrates Effectiveness of a Treat-to-Target Approach in Mild-to-Moderate Ulcerative Colitis in First Major Study

• OPTIMISE study found a treat-to-target (T2T) management strategy in people with mild-to-moderate ulcerative colitis (M2M UC) being treated with 5-aminosalicylic acid (5-ASA; mesalazine) utilising faecal calprotectin (FC) home monitoring over 12 months achieved a significantly higher rate of endoscopic and clinical remission compared to a symptom-based approach which did not utilise FC home monitoring.¹
• These data support clinical thinking that T2T is the preferred management strategy for inflammatory bowel disease (IBD)^2,3 and demonstrates that mesalazine therapy is an integral part of this approach in M2M UC.
• Further details of the study results to be shared by Principal Investigators of the OPTIMISE study, Prof Silvio Danese and Prof Laurent Peyrin-Biroulet, in an upcoming webinar on 8^th October 2024.

SAINT-PREX, Switzerland–(BUSINESS WIRE)–Ferring Pharmaceuticals today announces results from the OPTIMISE study showing the first real-world evidence of the effectiveness of a treat-to-target (T2T) approach based on faecal calprotectin (FC) in patients with mild-to-moderate ulcerative colitis (M2M UC). These results have been published in the Journal of Clinical Medicine.¹

UC is a chronic, inflammatory bowel condition which can cause recurring bloody diarrhoea, stomach pain and extreme tiredness.⁴ Of those living with UC, over 85% have mild-to-moderate disease.^5,6,7 Usually 5-ASA compounds are the first-line therapy for people with M2M UC, followed by a stepwise treatment approach in case of non-response or intolerance. There is currently limited guidance on timely escalation and de-escalation of therapies.

OPTIMISE was the first major study to investigate whether a T2T approach based on monitoring of non-invasive parameters, such as clinical symptoms and FC, can provide a significantly higher benefit for patients with M2M UC versus an entirely symptom-based approach. “The OPTIMISE study represents another milestone in the management of M2M UC and demonstrates Ferring’s continued commitment to science and improving the lives of people with inflammatory bowel disease (IBD),” said Pierre-Yves Berclaz, Chief Science & Medical Officer, Ferring Pharmaceuticals.

Results from OPTIMISE, a pragmatic, randomised controlled study, showed that people who had their 5-ASA/mesalazine treatment optimised (with or without steroids) by following the T2T approach had a 17–22% advantage at achieving combined endoscopic and clinical remission over a symptoms-only based approach. For the primary endpoint of Mayo Endoscopic Score (MES)=0 at 12 months, no significant difference was found, however it was noted by the investigators that the COVID-19 pandemic negatively impacted the amount of evaluable data.¹

“OPTIMISE has provided the first real-world evidence that a T2T approach can help people living with IBD to achieve long-lasting remission and have a greater quality of life,” said Kristine Paridaens, Senior Medical Director, Gastroenterology, Ferring Pharmaceuticals.

A T2T approach has been advocated by the International Organisation for the Study of IBD (IOIBD). In their Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) Consensus they recommended resolution of clinical signs of disease activity and endoscopic remission as the optimal targets.² This was further refined in STRIDE-II to include symptomatic relief and normalisation of serum and faecal markers as short-term targets for all therapies.³

The OPTIMISE results coincide with an international expert consensus focusing on the practical management of M2M UC, which was published in Expert Review of Gastroenterology and Hepatology.⁸ Professor Silvio Danese, a co-author of the Consensus and Principle Investigator of the OPTIMISE study, said, “The OPTIMISE study provides real-world evidence of the effectiveness of a T2T approach based on FC monitoring and how its implementation in clinical practice will allow clinicians to tightly monitor disease activity and promptly adapt treatment, helping to avoid complications and disease progression and enabling patients to achieve better disease control.”

To learn more, register here to attend the free ‘New avenues in mild-to-moderate UC treatment optimisation’ webinar on 8^th October, 2024. Principal Investigators of the OPTIMISE study, Prof Silvio Danese and Prof Laurent Peyrin-Biroulet, will provide an expert overview of the results and the implications for clinical practice. The webinar is intended for healthcare professionals only.

About the OPTIMISE study¹

OPTIMISE was a European-based, multi-centre, randomised (1:1) controlled study of 250 patients with M2M UC (global Mayo score 2–6) treated with ≤2.4g/day 5-aminosalicylic acid that compared the effectiveness of two management strategies with (T2T arm) and without (reference arm) FC home monitoring over 12 months follow-up. Treatment was optimised (escalated or de-escalated) in the T2T arm using FC values and clinical symptoms (PRO-2), whilst the reference arm used only PRO-2. In both arms, therapy was optimised in line with current ECCO guidelines for UC, including maximal doses of 5-ASA (oral and rectal).

A total of 193 patients completed the study despite the acknowledged impact of the COVID-19 pandemic on all clinical studies conducted during that time. Reduced patient contact during the COVID-19 period also affected the availability of data for analysis. For the primary endpoint of MES=0 at 12 months, there was no significant difference between arms, when patients with missing values were classified as non-responders. A subsequent analysis using Monte Carlo Markov Chain (MCMC) imputation found a numerical advantage for the T2T arm over the reference arm for the primary endpoint (37.0% vs 33.4%, respectively). The secondary endpoints, including clinical symptomatology and quality of life, were similarly impacted by missing data, but again displayed numerical superiority for MES≤1, RB=0 and SF≤1 at 12 months when using MCMC imputation.

A logistic regression analysis pooling results for MES, SF and RB at 12 months, using data derived from MCMC imputation, found a statistically significant advantage for the T2T arm over the reference arm (p<0.001). When these endpoints were combined in a fixed effects meta-analysis, the combined endpoint of MES=0, RB=0 and SF≤1 at 12 months was achieved at a significantly higher rate in the T2T than the reference arm (effect size [ES]: 0.17, 95% CI 0.02, 0.32; p<0.05). A similar result was obtained for MES≤1, RB=0 and SF≤1 at 12 months (ES: 0.22; 95% CI 0.07, 0.37; p<0.05).

References

¹ Danese S, Fiorino G, Vicaut E, et al. Pragmatic Randomised Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis – the OPTIMISE study. J Clin Med 2024;13:5147.

² Peyrin-Biroulet L, Sandborn W, Sands BE, et al. Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE): Determining Therapeutic Goals for Treat-to-Target. Am J Gastroenterol 2015;110:1324–38.

³ Turner D, Ricciuto A, Lewis A, et al. STRIDE-II: An Update on the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) Initiative of the International Organization for the Study of IBD (IOIBD): Determining Therapeutic Goals for Treat-to-Target strategies in IBD. Gastroenterology 2021;160:1570–83.

⁴ NHS. Ulcerative colitis. Available at: https://www.nhs.uk/conditions/ulcerative-colitis/. Last accessed: September 2024.

⁵ Raine T, Bonovas S, Kucharzik T, et al. ECCO Guidelines on Therapeutics in Ulcerative Colitis: Medical Treatment. J Crohns Colitis. 2022;16(1):2-17.

⁶ Fumery M, Singh S, Dulai PS, et al. Natural History of Adult Ulcerative Colitis in Population-based Cohorts: A Systematic Review. 2018;16(3):343-356.e3.

⁷ CCDS Pentasa All formulations. Version 18. 10 December 2022.

⁸ D’Amico F, Magro F, Dignass A, et al. Practical management of mild-to-moderate ulcerative colitis: an international expert consensus. Expert Rev Gastroenterol Hepatol 2024;18:421–30.

© 2024 Ferring. Ferring and the Ferring Pharmaceuticals logo are trademarks of the Ferring group of companies.

Contacts

Matthew Worrall
Director, Corporate Communications, Ferring
+44 7442 271 811

Matthew.Worrall@ferring.com

Kyowa Kirin Presents Real-World Evidence Demonstrating Clinically Meaningful Impact of Burosumab Treatment in Adults with X-linked Hypophosphatemia




Kyowa Kirin Presents Real-World Evidence Demonstrating Clinically Meaningful Impact of Burosumab Treatment in Adults with X-linked Hypophosphatemia

• Findings from UK burosumab early access program spotlighted in oral presentation at ASBMR annual meeting
• Statistically significant improvements seen in patient-reported measures of pain, stiffness, physical function, and health-related quality of life

GALASHIELS & MARLOW, United Kingdom–(BUSINESS WIRE)–Kyowa Kirin International (KKI), a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) and a Japan-based global specialty pharmaceutical company, presented new research showing statistically significant and clinically meaningful improvements in patient-reported outcomes (PROs) in adults with X-linked hypophosphatemia (XLH) following treatment with burosumab (CRYSVITA) in real-world clinical practice. These results were shared with attendees at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting in Toronto, Canada, 27-30 September 2024.

“The improvements we observed in patient-reported symptoms and health-related quality of life provide additional evidence of the real-world impact of treatment on outcomes relevant to patients and clinicians,” said Judith Bubbear, MD, at the Royal National Orthopaedic Hospital in London, UK, and chief study investigator and author. “These benefits were accompanied by sustained improvements in serum phosphate concentration, the hallmark deficiency in people with XLH.”

XLH is a rare genetic, progressive, metabolic bone disease that causes skeletal abnormalities, stiffness, pain, and impaired physical function.¹ In this retrospective, longitudinal, real-world study, researchers assessed the experience of 136 burosumab-naïve adults with XLH enrolled in the UK burosumab early access program.

Investigators observed statistically significant (p<0.05) and clinically relevant improvements in mean patient-reported outcomes after six and 12 months of burosumab treatment, including:

• Brief Pain Inventory-Short Form (BPI-SF) domains: Worst Pain, Pain Severity, and Pain Interference.
• Western Ontario and McMaster Universities Arthritis Index (WOMAC) for Stiffness, Physical Function, Pain, and Total Score.
• EuroQol 5-Dimension (EQ-5D) 5-Level utility and visual analogue scale scores, measures of health-related quality of life.

“XLH is a lifelong, progressive disease, and Kyowa Kirin is deeply committed to partnering with the clinical and XLH community to help advance our understanding of the disease and its impact on patients and families,” commented Ben Johnson, global HEOR lead for nephrology at Kyowa Kirin and study author. “These data enhance our understanding of the real-world effectiveness of burosumab treatment in adults with XLH, in terms of the impact on specific symptoms as well as general health-related quality of life.”

About X-linked hypophosphataemia (XLH)

XLH is caused by a genetic mutation which leads to overexpression of the protein FGF23, a protein involved in the regulation of phosphate concentration in the blood. In XLH, FGF23 is produced in excess leading to depletion of phosphate in the blood, known as hypophosphataemia.¹

Individuals living with the disease may display a multitude of symptoms including short stature, limb deformities, bone and joint pain, oral abscesses, and hearing loss.¹ To manage this wide variety of symptoms, the disease is managed through multi-disciplinary teams.²

About CRYSVITA (burosumab)

CRYSVITA (burosumab) is a recombinant human monoclonal antibody (mAb) that binds to the protein fibroblast growth factor 23 (FGF23). This has the impact of inhibiting the action of FGF23, allowing phosphate regulation in the body to be restored.³

About Kyowa Kirin

Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by severe and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across our four regions—Japan, Asia Pacific, North America, and EMEA/International.

You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com

References

¹Beck-Nielsen SS, et al. 2019. FGF23 and its role in X-linked hypophosphatemia-related morbidity. Orphanet Journal of Rare Diseases. 2022;14:1-25.

²Kubota T. X-linked hypophosphatemia transition and team management. Endocrines. 2022;3(3):411-418.

³European Medicines Agency. Summary of Product Characteristics (SmPC). CRYSVITA® (burosumab). 2023. Available here. [Last accessed September 2024].

Contacts

Stacey Minton

Stacey.Minton@kyowakirin.com

Arcturis and Guy’s and St Thomas’ NHS Foundation Trust Enter Strategic Agreement




Arcturis and Guy’s and St Thomas’ NHS Foundation Trust Enter Strategic Agreement

• Agreement to develop deep, enriched real-world datasets to support the development and approval of novel cancer therapies
• Initial focus on gynaecological, lung, prostate and bladder cancers

OXFORD, England–(BUSINESS WIRE)–Arcturis Data Limited (“Arcturis”), the UK leader in curating deep, multi-modal health data to support development of precision medicines, and Guy’s and St Thomas’ NHS Foundation Trust (“Guy’s and St Thomas’”), a global exemplar in clinical, research and digital health excellence, announce today that they have entered into a multi-year Strategic Research Agreement. This agreement will allow anonymised NHS data, from an established Research Ethics approved database, to be made available to Arcturis for the purposes of undertaking research that supports the development and approval of novel therapies for cancer patients.

This agreement allows investment from Arcturis to Guy’s and St Thomas’ to support the curation and enrichment of deep, longitudinal and multi-modal research datasets that enable a precision medicine approach. The initial focus of the agreement will be in oncology covering areas including gynaecological, lung, prostate and bladder cancers. Cancer is responsible for a significant burden of patient morbidity and mortality and consumes significant resources within healthcare systems annually. This agreement will facilitate valuable research and innovation, delivering benefits to the health service, patients and the regional economy, whilst safeguarding the confidentiality and privacy of patients.

Dr Anne Rigg, Co-Chair of the Guy’s Cancer Real World Evidence Committee, Consultant Medical Oncologist, Medical Director for Cancer and Surgery at Guy’s and St Thomas’ commented, “Arcturis has unique expertise in integrating molecular and clinical data and curating fit-for-purpose, regulatory-grade research datasets. This valuable work will help to improve the quality and completeness of the data available within Guy’s and St Thomas’ for world class research, driving real world impact. Our patient and public partners will oversee the work of this ambitious collaboration, ensuring a central focus on healthcare improvement and clinical impact.” Consultancy and analytics – Guy’s Cancer cohort | Guy’s and St Thomas’ NHS Foundation Trust (guysandstthomas.nhs.uk)

Christie Brooks, Director of Data Strategy and Partnerships at Arcturis commented, “Arcturis is delighted to enter this long-term strategic partnership with Guy’s and St Thomas’, one of the UK’s leading providers of hospital and community-based healthcare and medical research. The partnership will build on the expertise of both organisations through the convergence of innovations in technology, informatics and clinical excellence and will enhance our growing health data network in the UK. Arcturis is committed to using deep, regulatory grade patient data to generate unique data insights to enhance clinical trials, regulatory decision making and adoption of new therapies to improve patient outcomes, whilst allowing the NHS to benefit from the wealth of data that the health system generates.”

Patient, Monica Jefford added, “This agreement between Arcturis and Guy’s and St Thomas’ and the technological management of our data, can only positively impact and benefit patients. The ever-evolving technological landscape, together with the resources available, present opportunities to further develop and improve patient care and experience. Both of these organisations are well-respected with established practices in how data is appropriately collected, applied, utilised and stored. Most importantly, this agreement prioritises putting patient’s interests and wellbeing at the heart of everything they do.”

About Arcturis Data Limited

Arcturis is the UK leader in the curation and application of precision, multi-modal heath data to enhance decision making across drug discovery, clinical development and post approval studies. Our unique access to real-time, deeply enriched health data in the UK covers all therapeutic areas including oncology, respiratory, cardiovascular and metabolic diseases. The regulatory grade data insights we generate supports the development and adoption of new medicines to improve patient outcomes. For more information, visit www.arcturisdata.com.

Contact: Alistair Armstrong, Head of NHS Partnerships on info@arcturisdata.com.

About Guy’s and St Thomas’ NHS Foundation Trust

Guy’s and St Thomas’ provides 2.8 million patient contacts in acute and specialist hospital services and community services every year. The Trust includes Guy’s Hospital, St Thomas’ Hospital, Evelina London Children’s Hospital, Royal Brompton Hospital, Harefield Hospital, and adult and children’s community services in Lambeth and Southwark. Guy’s and St Thomas’ is part of King’s Health Partners Academic Health Sciences Centre (AHSC), a collaboration between King’s College London, and Guy’s and St Thomas’, King’s College Hospital and South London and Maudsley NHS Foundation Trusts. www.kingshealthpartners.org. Guy’s and St Thomas’ is one of the biggest NHS trusts in the UK, with an annual turnover of £2.9 billion, and employs around 23,600 staff. www.guysandstthomas.nhs.uk.

Contacts

Alistair Armstrong, Head of NHS Partnerships

e-mail: Alistair.armstrong@arcturisdata.com

Guy’s and St Thomas’ NHS Foundation Trust

tel: 020 7188 5577 or e-mail: press@gstt.nhs.uk
Out of hours, please call our pager bureau on 0844 822 2888, ask for pager number 847704 and give the pager operator your message.

ENHERTU® Granted Priority Review in the U.S. for Patients with HER2 Low or HER2 Ultralow Metastatic Breast Cancer Who Have Received at Least One Line of Endocrine Therapy




ENHERTU® Granted Priority Review in the U.S. for Patients with HER2 Low or HER2 Ultralow Metastatic Breast Cancer Who Have Received at Least One Line of Endocrine Therapy

• Based on DESTINY-Breast06 phase 3 trial which demonstrated a statistically significant and clinically meaningful progression-free survival benefit for ENHERTU
• If approved, Daiichi Sankyo and AstraZeneca’s ENHERTU will be the first HER2 directed therapy and antibody drug conjugate for patients prior to receiving chemotherapy for metastatic breast cancer

TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Daiichi Sankyo (TSE: 4568) and AstraZeneca’s (LSE/STO/Nasdaq: AZN) supplemental Biologics License Application (sBLA) for ENHERTU^® (fam-trastuzumab deruxtecan-nxki) has been accepted and granted Priority Review in the U.S. for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (IHC 0 with membrane staining) breast cancer who have received at least one endocrine therapy in the metastatic setting.

ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

The U.S. Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available treatment options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is February 1, 2025. The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA for ENHERTU based on data from the DESTINY-Breast06 phase 3 trial in August 2024.

Hormone receptor (HR) positive, HER2 negative is the most common breast cancer subtype, accounting for approximately 70% of all breast cancers.¹ Despite being classified as HER2 negative, many of these tumors still carry some level of HER2 expression.² It is estimated that up to 85% to 90% of tumors historically classified as HR positive, HER2 negative, may be HER2 low or HER2 ultralow.^3,4

The sBLA is based on data from DESTINY-Breast06 presented as a late-breaking oral session at the 2024 American Society of Clinical Oncology (#ASCO24) Annual Meeting and recently published in The New England Journal of Medicine. In the overall trial population, ENHERTU reduced the risk of disease progression or death by 37% by blinded independent central review (BICR) versus chemotherapy (hazard ratio [HR] 0.63; 95% confidence interval [CI]: 0.53-0.75; p<0.0001). Median progression-free survival (PFS) was 13.2 months with ENHERTU compared to 8.1 months with chemotherapy.

Results were consistent in patients with HER2 low expression and HER2 ultralow expression. In the primary endpoint analysis of patients with HR positive, HER2 low metastatic breast cancer, median PFS was 13.2 months in the ENHERTU arm compared to 8.1 months in the chemotherapy arm (HR 0.62; 95% CI: 0.51-0.74; p<0.0001), as assessed by BICR. In a prespecified exploratory analysis of patients with HER2 ultralow expression, ENHERTU showed a median PFS of 13.2 months versus 8.3 months, respectively (HR 0.78; 95% CI: 0.50-1.21).

The safety profile of ENHERTU in DESTINY-Breast06 was consistent with previous breast cancer clinical trials with no new safety concerns identified. The most common grade 3 or higher treatment related treatment emergent adverse events (TEAEs) occurring in 5% or more of patients treated with ENHERTU were neutropenia (20.7%), leukopenia (6.9%) and anemia (5.8%). Interstitial lung disease (ILD) or pneumonitis occurred in 11.3% of patients treated with ENHERTU. The majority of ILD or pneumonitis events were low grade (grade 1 [n=7; 1.6%] or grade 2 [n=36; 8.3%]). There were three grade 3 ILD events (0.7%), zero grade 4 events and three grade 5 events (0.7%) as determined by an independent adjudication committee.

“This Priority Review highlights the potential to expand the existing indication of ENHERTU in HER2 low metastatic breast cancer to include use in an earlier disease setting as well as in a broader patient population that includes HER2 ultralow,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “We look forward to working closely with the FDA with the goal of bringing ENHERTU to more patients as quickly as possible.”

“While endocrine therapies are widely used in the initial treatment of HR positive metastatic breast cancer, most patients see limited benefit with additional lines of treatment and subsequent chemotherapy is associated with poor response rates and outcomes,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology R&D, AstraZeneca. “The results from DESTINY-Breast06 show that ENHERTU has the potential to evolve the current HR positive treatment landscape and become the first targeted treatment for patients with HER2 low or HER2 ultralow expression following endocrine therapy.”

About DESTINY-Breast06

DESTINY-Breast06 is a global, randomized, open-label, phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus investigator’s choice of chemotherapy (capecitabine, paclitaxel or nab paclitaxel) in patients with HR positive, HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (defined as IHC 0 with membrane staining [IHC >0 <1+]) advanced or metastatic breast cancer. Patients in the trial had no prior chemotherapy for advanced or metastatic disease and received at least two lines of prior endocrine therapy in the metastatic setting. Patients also were eligible if they had received one prior line of endocrine therapy combined with a CDK4/6 inhibitor in the metastatic setting and experienced disease progression within six months of starting first-line treatment or received endocrine therapy as an adjuvant treatment and experienced disease recurrence within 24 months.

The primary endpoint of DESTINY-Breast06 is PFS in the HR positive, HER2 low patient population as measured by BICR. Key secondary endpoints include PFS by BICR in the overall trial population (HER2 low and HER2 ultralow), overall survival (OS) in patients in the HER2 low patient population and OS in the overall trial population. Other secondary endpoints include objective response rate, duration of response, time to first subsequent treatment or death, time to second subsequent treatment or death and safety. Analysis of the HER2 ultralow subgroup was not powered to demonstrate statistical significance.

DESTINY-Breast06 enrolled 866 patients (n=713 for HER2 low and n=153 for HER2 ultralow) at multiple sites in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.

About Breast Cancer and HER2 Expression

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.⁵ More than two million breast cancer cases were diagnosed in 2022 with more than 665,000 deaths globally.⁵ In the U.S., more than 300,000 cases of breast cancer are diagnosed annually.⁶ While survival rates are high for those diagnosed with early breast cancer, only about 30% of patients diagnosed with or who progress to metastatic disease are expected to live five years following diagnosis.¹

HR positive, HER2 negative is the most common breast cancer subtype, accounting for approximately 70% of all breast cancers.¹ HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors, including breast cancer.⁷ Patients with high levels of HER2 expression (IHC 3+ or IHC2+/ISH+) are classified as HER2 positive and treated with HER2 targeted therapies, representing approximately 15 to 20% percent of all breast cancers.⁸ Historically, tumors that were not classified as HER2 positive were classified as HER2 negative, despite the fact that many of these tumors still carry some level of HER2 expression.² It is estimated that approximately 60% to 65% of HR positive, HER2 negative breast cancers are HER2 low and potentially an additional 25% may be HER2 ultralow.^3,4

Endocrine therapies are widely given consecutively in the early lines of treatment for HR positive metastatic breast cancer. However, following two lines of endocrine therapy, further efficacy with additional endocrine treatment is often limited. ⁹ The current standard of care following endocrine therapy is chemotherapy, which is associated with poor response rates and outcomes.^9,10,11,12

Prior to the approval of ENHERTU following chemotherapy in HER2 low metastatic breast cancer based on the DESTINY-Breast04 trial, there were no targeted therapies approved specifically for patients with HER2 low expression.¹³ There are no targeted therapies specifically approved for patients with HER2 ultralow expression.¹⁴

About ENHERTU

ENHERTU (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

ENHERTU (5.4 mg/kg) is approved in more than 65 countries worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+ or in-situ hybridization (ISH)+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

ENHERTU (5.4 mg/kg) is approved in more than 65 countries worldwide for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

ENHERTU (5.4 mg/kg) is approved in more than 45 countries worldwide for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 trial. Continued approval in the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (6.4 mg/kg) is approved in more than 45 countries worldwide for the treatment of adult patients with locally advanced or metastatic HER2 positive (IHC 3+ or IHC 2+/ISH+) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric06 trials. Full approval in China for this indication will depend on whether a randomized controlled confirmatory clinical trial can demonstrate clinical benefit in this population.

ENHERTU (5.4 mg/kg) is approved in the U.S. for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02 trials. Continued approval for this indication in the U.S. may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About the ENHERTU Clinical Development Program

A comprehensive global clinical development program is underway evaluating the efficacy and safety of ENHERTU monotherapy across multiple HER2 targetable cancers. Trials in combination with other anticancer treatments, such as immunotherapy, also are underway.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and datopotamab deruxtecan in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and datopotamab deruxtecan.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and datopotamab deruxtecan (Dato-DXd), a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Datopotamab deruxtecan, ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

ENHERTU U.S. Important Safety Information

Indications

ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with:

• Unresectable or metastatic HER2-positive (IHC 3+ or ISH positive) breast cancer who have received a prior anti-HER2-based regimen either:
  
  – In the metastatic setting, or
  
  – In the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy

• Unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer, as determined by an FDA-approved test, who have received a prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy

• Unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

• Locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH positive) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen

• Unresectable or metastatic HER2-positive (IHC3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Contraindications

None.

Warnings and Precautions

Interstitial Lung Disease / Pneumonitis

Severe, life-threatening, or fatal interstitial lung disease (ILD), including pneumonitis, can occur in patients treated with ENHERTU. A higher incidence of Grade 1 and 2 ILD/pneumonitis has been observed in patients with moderate renal impairment. Advise patients to immediately report cough, dyspnea, fever, and/or any new or worsening respiratory symptoms. Monitor patients for signs and symptoms of ILD. Promptly investigate evidence of ILD. Evaluate patients with suspected ILD by radiographic imaging. Consider consultation with a pulmonologist. For asymptomatic ILD/pneumonitis (Grade 1), interrupt ENHERTU until resolved to Grade 0, then if resolved in ≤28 days from date of onset, maintain dose. If resolved in >28 days from date of onset, reduce dose one level. Consider corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥0.5 mg/kg/day prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), permanently discontinue ENHERTU. Promptly initiate systemic corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥1 mg/kg/day prednisolone or equivalent) and continue for at least 14 days followed by gradual taper for at least 4 weeks.

HER2-Positive or HER2-Low Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, ILD occurred in 12% of patients. Median time to first onset was 5.5 months (range: 0.9 to 31.5). Fatal outcomes due to ILD and/or pneumonitis occurred in 1.0% of patients treated with ENHERTU.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, ILD occurred in 10% of patients. Median time to first onset was 2.8 months (range: 1.2 to 21).

Neutropenia

Severe neutropenia, including febrile neutropenia, can occur in patients treated with ENHERTU. Monitor complete blood counts prior to initiation of ENHERTU and prior to each dose, and as clinically indicated. For Grade 3 neutropenia (Absolute Neutrophil Count [ANC] <1.0 to 0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then maintain dose. For Grade 4 neutropenia (ANC <0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then reduce dose by one level. For febrile neutropenia (ANC <1.0 x 10⁹/L and temperature >38.3º C or a sustained temperature of ≥38º C for more than 1 hour), interrupt ENHERTU until resolved, then reduce dose by one level.

HER2-Positive or HER2-Low Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, a decrease in neutrophil count was reported in 63% of patients. Seventeen percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 22 days (range: 2 to 939). Febrile neutropenia was reported in 1% of patients.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, a decrease in neutrophil count was reported in 72% of patients. Fifty-one percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 16 days (range: 4 to 187). Febrile neutropenia was reported in 4.8% of patients.

Left Ventricular Dysfunction

Patients treated with ENHERTU may be at increased risk of developing left ventricular dysfunction. Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including ENHERTU. Assess LVEF prior to initiation of ENHERTU and at regular intervals during treatment as clinically indicated. Manage LVEF decrease through treatment interruption. When LVEF is >45% and absolute decrease from baseline is 10-20%, continue treatment with ENHERTU. When LVEF is 40-45% and absolute decrease from baseline is <10%, continue treatment with ENHERTU and repeat LVEF assessment within 3 weeks. When LVEF is 40-45% and absolute decrease from baseline is 10-20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF has not recovered to within 10% from baseline, permanently discontinue ENHERTU. If LVEF recovers to within 10% from baseline, resume treatment with ENHERTU at the same dose. When LVEF is <40% or absolute decrease from baseline is >20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF of <40% or absolute decrease from baseline of >20% is confirmed, permanently discontinue ENHERTU. Permanently discontinue ENHERTU in patients with symptomatic congestive heart failure. Treatment with ENHERTU has not been studied in patients with a history of clinically significant cardiac disease or LVEF <50% prior to initiation of treatment.

HER2-Positive or HER2-Low Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, LVEF decrease was reported in 3.8% of patients, of which 0.6% were Grade 3.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, no clinical adverse events of heart failure were reported; however, on echocardiography, 8% were found to have asymptomatic Grade 2 decrease in LVEF.

Embryo-Fetal Toxicity

ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of ENHERTU. Advise females of reproductive potential to use effective contraception during treatment and for 7 months after the last dose of ENHERTU. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for 4 months after the last dose of ENHERTU.

Additional Dose Modifications

Thrombocytopenia

For Grade 3 thrombocytopenia (platelets <50 to 25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then maintain dose. For Grade 4 thrombocytopenia (platelets <25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then reduce dose by one level.

Adverse Reactions

HER2-Positive and HER2-Low Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

The pooled safety population reflects exposure to ENHERTU 5.4 mg/kg intravenously every 3 weeks in 1799 patients in Study DS8201-A-J101 (NCT02564900), DESTINY-Breast01, DESTINY-Breast02, DESTINY-Breast03, DESTINY-Breast04, DESTINY-Lung01, DESTINY-Lung02, DESTINY-CRC02, and DESTINY-PanTumor02. Among these patients, 65% were exposed for >6 months and 38% were exposed for >1 year.

Contacts

Media

Global/US:
Jennifer Brennan

Daiichi Sankyo, Inc.

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

Japan:
Daiichi Sankyo Co., Ltd.

DS-PR@daiichisankyo.co.jp

Investor Relations:
DaiichiSankyoIR@daiichisankyo.co.jp

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SunLink Health Systems, Inc. Announces Fiscal 2024 Fourth Quarter and Annual Results




SunLink Health Systems, Inc. Announces Fiscal 2024 Fourth Quarter and Annual Results

ATLANTA–(BUSINESS WIRE)–SunLink Health Systems, Inc. (NYSE American: SSY) today announced a loss from continuing operations of $652,000 (or a loss of $0.09 per fully diluted share) for its fourth fiscal quarter ended June 30, 2024 compared to a loss from continuing operations of $1,021,000 (or a loss of $0.15 per fully diluted share) for the fourth fiscal quarter ended June 30, 2023.

Earnings from discontinued operations were $4,940,000 (or earnings of $0.70 per fully diluted share) for the fourth fiscal quarter ended June 30, 2024 compared to a loss from discontinued operations of $405,000 (or a loss of $0.06 per fully diluted share) for the fourth fiscal quarter ended June 30, 2023. During the quarter ended June 30, 2024, the Company’s indirect subsidiary, Southern Health Corporation of Houston, Inc. (“Southern”) sold its Trace Extended Care & Rehab senior care facility and related real estate in Houston, Mississippi for net proceeds of $6,522,000 and recorded a gain on the sale of $5,584,000. Southern’s results are included in discontinued operations for the year ended June 30, 2024.

Primarily as a result of the sale of Trace Extended Care & Rehab, net income for the quarter ended June 30, 2024 was $4,288,000 (or $0.61 per fully diluted share) compared to a net loss of $1,426,000 (or a loss of $0.20 per fully diluted share) for the quarter ended June 30, 2023.

On January 22, 2024, Southern reached revised agreements for the sale of Trace Regional Hospital, a vacant medical office building and three (3) patient clinics in Chickasaw County Mississippi, (collectively “Trace Hospital Assets”) to Progressive Health of Houston, LLC (“Progressive”) pursuant to which (i) Southern sold certain personal and intangible property to Progressive for $500,000 under to an asset purchase agreement (“Trace Hospital Assets Sale”), (ii) entered into a six-month net lease of the real property of the hospital, medical office building and the clinics property (the “Trace Real Estate Lease”) for $20,000 per month, (iii) entered into a contract to sell the Trace Hospital real estate (the “Trace Hospital Real Estate”) to Progressive for $2,000,000 and (iv) engaged Progressive under a management agreement to manage the operations of Trace Hospital pending receipt of certain regulatory approvals, which was received February 29, 2024. The Company recorded a loss of $962,000 on the Trace Hospital Assets sale during the year ended June 30, 2024, which included sale expenses of $174,000. The completion of the Trace Hospital Real Estate sale has been extended by mutual agreement to October 4, 2024. As a result of the transactions (“Revised Agreement”), SunLink reported an impairment loss of $1,974,000 at December 31, 2023 to reduce the net value of the Trace Hospital Assets and Trace Hospital Real Estate to the estimated sale proceeds under the Revised Agreement. An impairment reserve of $1,695,000 remains at June 30, 2024 for the Trace Hospital Real Estate. There can be no assurance the completion of the sale of the Trace Hospital Assets Real Estate will be achieved.

Consolidated net revenues for each of the fiscal quarters ended June 30, 2024 and 2023 were $7,913,000 and $8,010,000, respectively, which consists primarily of pharmacy net revenues. Pharmacy net revenues for the quarter ended June 30, 2024 decreased $97,000, or 1%, from the same period last year as a result of lower pharmacy scripts dispensed. The quarter ended June 30, 2024 includes $34,000 of prior period sales tax credits relating to such sales tax refund claims.

SunLink reported an operating loss for the quarter ended June 30, 2024 of $675,000 compared to an operating loss for the quarter ended June 30, 2023 of $1,112,000. The decreased operating loss this year compared to last year resulted primarily from increased gross profit margin on pharmacy revenue.

SunLink reported a loss from continuing operations of $2,311,000 (or a loss of $0.33 per fully diluted share) for its twelve months ended June 30, 2024 compared to earnings from continuing operations of $198,000 (or $0.03 per fully diluted share) for the twelve months ended June 30, 2023.

Earnings from discontinued operations were $784,000 (or $0.11 per fully diluted share) for the twelve months ended June 30, 2024 compared to a loss of $1,993,000 (or a loss of $0.28 per fully diluted share) for the twelve months ended June 30, 2023, primarily due to the Trace Hospital Assets and Trace Extended Care & Rehab transactions and Trace Hospital Real Estate impairment charge described above.

SunLink reported a net loss of $1,527,000 (or a loss of $0.22 per fully diluted share) for its twelve months ended June 30, 2024 compared to a net loss of $1,795,000 (or $0.26 per fully diluted share) for the twelve months ended June 30, 2023.

Consolidated net revenues for each of the twelve months ended June 30, 2024 and 2023 were $32,440,000 and $34,280,000, respectively. Pharmacy net revenues for the twelve months ended June 30, 2023 included $2,615,000 from the reversal of reserves for certain sales taxes previously accrued. The Company determined during that quarter that, based on discussions and correspondence from taxing authorities and consultation with external legal counsel, it was more likely than not that such accrued sales taxes would not be payable. The twelve months ended June 30, 2024 includes $471,000 of prior period sales tax refunds. Excluding the effect of the sales tax refunds and reversal of sales tax accruals, net revenues decreased less than 1 % in the twelve months ended June 30, 2024 compared to the prior year.

SunLink reported an operating loss for the twelve months ended June 30, 2024 of $2,411,000 compared to operating profit for the twelve months ended June 20, 2023 of $74,000. The operating profit during the comparable twelve month period last year resulted primarily from the reversal of accrued sales tax reserves.

COVID-19 Pandemic

The Company continues to experience adverse after-effects of the COVID-19 pandemic in the quarter ended June 30, 2024 and believes such effects will likely continue to affect its assets and operations in the foreseeable future particularly from salaries and wages pressure, workforce shortages, supply chain disruption and broad inflationary pressures. Our ability to make estimates of any such continuing effects on future revenues, expenses or changes in accounting judgments that have had or are reasonably likely to have a material effect on our financial statements is very limited, depending as they do on the severity and length thereof; as well as any further government actions and/or regulatory changes intended to address such effects.

SunLink Health Systems, Inc. is the parent company of subsidiaries that own and operate a pharmacy business and an information technology business in the Southeast. For additional information on SunLink Health Systems, Inc., please visit the Company’s website.

This press release contains certain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 including, without limitation, statements regarding the company’s business strategy. These forward-looking statements are subject to certain risks, uncertainties, and other factors, which could cause actual results, performance, and achievements to differ materially from those anticipated. Certain of those risks, uncertainties and other factors are disclosed in more detail in the company’s Annual Report on Form 10-K for the year ended June 30, 2024 and other filings with the Securities and Exchange Commission which can be located at www.sec.gov.

Contacts

Robert M. Thornton, Jr.
Chief Executive Officer
(770) 933-7004

AHF: J&J Retreats From Its Scheme to Gut 340B Drug Pricing Program




AHF: J&J Retreats From Its Scheme to Gut 340B Drug Pricing Program

WASHINGTON–(BUSINESS WIRE)–The following is a statement from AIDS Healthcare Foundation (AHF):

Thanks to a clear warning (see U.S. Health Resources and Services Administration letter [HRSA] Response to J&J’s September 19, 2024 Letter) from the U.S government that J&J’s plan to destroy the 340B Drug Pricing Program is illegal, the drug industry giant sent a letter raising the white flag of retreat today (See J&J’s September 30, 2024 letter here).

J&J’s illegal scheme would have denied required 340B discounts to eligible healthcare safety net covered entities and instead provided rebates if and when it chose to do so. J&J’s scheme may or may not have reimbursed providers at a later date, cheating safety net providers, making every claim subject to dispute and protracted payment. J&J’s scheme violates the law and AHF is pleased that HRSA has swiftly responded.

J&J’s hypocritical blather about making 340B “sustainable” belies its actions. Multiple J&J annual price increases aren’t sustainable. J&J’s flurry of extra-legal restrictions on contract pharmacies isn’t sustainable.

J&J’s retreat shows its “340B is not sustainable” argument is nonsense and should be disregarded whenever it is raised. Drug companies will not quit the lucrative taxpayer-funded Medicaid and Medicare drug markets.

AIDS Healthcare Foundation (AHF), the world’s largest HIV/AIDS healthcare organization, provides cutting-edge medicine and advocacy to more than 2.1 million individuals across 47 countries, including the U.S. and in Africa, Latin America/Caribbean, the Asia/Pacific Region, and Eastern Europe. To learn more about AHF, visit us online at AIDShealth.org, find us on Facebook, and follow us on Instagram, Twitter, and TikTok.

Contacts

MEDIA CONTACTS:

Ged Kenslea, Sr. Director, Communications, AHF

1.323.791.5526 mobile

ged.kenslea@ahf.org