Celltrion receives U.S. FDA approval for STOBOCLO® (denosumab-bmwo) and OSENVELT® (denosumab-bmwo) biosimilars referencing PROLIA® and XGEVA®

On March 3, 2025 Celltrion reported that the U.S. Food and Drug Administration (FDA) has approved STOBOCLO (CT-P41, denosumab-bmwo) and OSENVELT (CT-P41, denosumab-bmwo), biosimilars referencing PROLIA (denosumab) and XGEVA (denosumab) respectively for all indications of reference products (Press release, Celltrion, MAR 3, 2025, View Source [SID1234650848]).

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"The approval of STOBOCLO and OSENVELT is another step forward in our efforts to deliver cost-effective and high-quality treatments that address critical unmet needs in osteoporosis-related fracture as well as cancer-related skeletal events," said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. "Patients deserve therapeutic options that have the potential to make real impacts on their care and their lives. We are committed to continuous innovation to meet these goals leveraging our experience and successful track record with biosimilar and novel biologics."

The FDA approval is based on robust clinical evidence, including results from Phase III clinical trials in postmenopausal women with osteoporosis designed to evaluate the efficacy, pharmacodynamics (PD), pharmacokinetics (PK), safety and immunogenicity of CT-P41 to reference denosumab. Study results demonstrated that CT-P41 had equivalent efficacy and PD to reference denosumab with similar PK and comparable safety and immunogenicity profiles.

"Denosumab is used to improve or protect bone health in patients with osteoporosis or those undergoing various cancer treatments and as a therapy for a lifetime for postmenopausal osteoporosis (PMO) patients," said Prof. Jean-Yves Reginster, Professor of Medicine, Protein Research Chair, Biochemistry Dept, College of Science, King Saud University, Riyadh, Kingdom of Saudi Arabia and Director WHO Collaborating Centre for Epidemiology of Musculoskeletal Health and Aging, Liège, Belgium. "Biosimilars have expanded into new therapeutic areas such as immunology, oncology and ophthalmology as they continue to offer significant cost-saving potential while expanding patient access. Having a denosumab product with a clinically proven track record in quality and safety is a valuable addition for my patients."

In accordance with a settlement agreement with Amgen Inc., STOBOCLO and OSENVELT are expected to be available in the U.S. in June 2025.

About STOBOCLO (denosumab-bmwo)

STOBOCLO (denosumab-bmwo) is a receptor activator of NF-κb ligand (RANKL) inhibitor referencing PROLIA (denosumab). STOBOCLO 60 mg/mL injection is approved by the FDA based on comprehensive data and clinical evidence confirming the therapeutic equivalence to PROLIA. In the U.S., STOBOCLO is approved to treat postmenopausal women with osteoporosis at high risk for fracture, to increase bone mass in men with osteoporosis at high risk for fracture, to treat glucocorticoid-induced osteoporosis in men and women at high risk for fracture, to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer, and to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer. STOBOCLO was also approved by the European Medicines Agency (EMA) in February 2025.

INDICATIONS

STOBOCLO (denosumab-bmwo) is a RANK ligand (RANKL) inhibitor indicated for treatment:

of postmenopausal women with osteoporosis at high risk for fracture
to increase bone mass in men with osteoporosis at high risk for fracture or in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer
of glucocorticoid-induced osteoporosis in men and women at high risk for fracture
to increase bone mass in women at high risk for fracture receiving a adjuvant aromatase inhibitor therapy for breast cancer
IMPORTANT SAFETY INFORMATION

WARNING: SEVERE HYPOCALCEMIA IN PATIENTS WITH ADVANCED KIDNEY DISEASE

Patients with advanced chronic kidney disease, including those on dialysis, face a higher risk of severe hypocalcemia after denosumab administration, with reported cases leading to hospitalization, life-threatening events, and fatalities.

The presence of chronic kidney disease-mineral bone disorder (CKD-MBD) markedly increases the risk of hypocalcemia in these patients

Before starting STOBOCLO (denosumab-bmwo) in advanced chronic kidney disease patients, assess for CKD-MBD. Treatment should be supervised by a healthcare provider experienced in diagnosing and managing CKD-MBD.

STOBOCLO is contraindicated in hypocalcemia, pregnant women, and in patients with known hypersensitivity to denosumab.

Severe Hypocalcemia: Ensure adequate calcium and vitamin D; monitor for severe hypocalcemia.

Drug Products with Same Active Ingredient: Do not use with other denosumab products.

Hypersensitivity: If an anaphylactic or other clinically significant allergic reaction occurs, initiate appropriate therapy and discontinue further use of STOBOCLO.

Osteonecrosis of the Jaw (ONJ): ONJ can occur in patients on STOBOCLO. Conduct oral exams before treatment; maintain oral hygiene; consider discontinuation of STOBOCLO if ONJ develops.

Atypical Subtrochanteric and Diaphyseal Femoral Fractures: Monitor for thigh, hip, or groin pain; evaluate for fractures. Interruption of STOBOCLO therapy should be considered, pending a benefit-risk assessment, on an individual basis.

Multiple Vertebral Fractures (MVF) Following Discontinuation of Treatment: Increased risk post-discontinuation of denosumab; transition to alternative therapy if discontinuing STOBOCLO.

Serious Infections: Higher risk in denosumab users; assess benefit-risk profile, especially in immunocompromised patients. Assess the benefit-risk profile before starting STOBOCLO and reconsider its use if serious infections develop.

Dermatologic Adverse Reactions: Consider discontinuing STOBOCLO if severe dermatitis, eczema, or rashes occur.

Musculoskeletal Pain: Consider discontinuation of STOBOCLO if severe pain develops.

Bone Turnover Suppression: In clinical trials in women with postmenopausal osteoporosis, denosumab significantly suppressed bone remodelling; patients should be monitored for these outcomes.

Hypercalcemia in Pediatrics Patients with Osteogenesis Imperfecta: Not for pediatric use; hypercalcemia reported in patients osteogenesis imperfecta treated with denosumab products.

Most common Adverse Reactions:

In (>5%) of patients with: Postmenopausal osteoporosis were back pain, pain in extremity, hypercholesterolemia, musculoskeletal pain, and cystitis. Pancreatitis has been reported in clinical trials. Male osteoporosis were back pain, arthralgia, and nasopharyngitis.
Glucocorticoid-induced osteoporosis (> 3%) were back pain, hypertension, bronchitis, and headache.
Bone loss due to hormone ablation for cancer (≥ 10%) were arthralgia and back pain. Pain in extremity and musculoskeletal pain have also been reported in clinical trials.
For more information, see Full Prescribing Information.

About OSENVELT (denosumab-bmwo)

OSENVELT (denosumab-bmwo) is a receptor activator of NF-κb ligand (RANKL) inhibitor referencing XGEVA (denosumab). OSENVELT 120 mg/1.7 mL (70 mg/mL) injection is approved by the FDA based on a robust clinical trial and comprehensive data confirming the therapeutic equivalence to XGEVA. In the U.S., OSENVELT is indicated to prevent skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors, to treat adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity, and to treat hypercalcemia of malignancy refractory to bisphosphonate therapy. OSENVELT was also approved by the European Medicines Agency (EMA) in February 2025.

INDICATION

OSENVELT (denosumab-bmwo) is indicated for:

Prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors.
Treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity.
Treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.
IMPORTANT SAFETY INFORMATION

Contraindications: Patients with hypocalcemia or with known clinically significant hypersensitivity to denosumab products.

Drug Products with Same Active Ingredient. Patients receiving OSENVELT should not receive other denosumab products concomitantly.

Hypersensitivity. If an anaphylactic or other clinically significant allergic reaction occurs, initiate appropriate therapy and discontinue further use of OSENVELT.

Hypocalcemia. Severe hypocalcemia can occur, and fatal cases have been reported. Monitor calcium levels and calcium and vitamin D intake.

Osteonecrosis of the Jaw (ONJ): ONJ can occur in patients on OSENVELT. Conduct oral exams and appropriate preventive dentistry before and during treatment; maintain oral hygiene and avoid invasive dental procedures; consider discontinuation of OSENVELT if ONJ develops.

Atypical Subtrochanteric and Diaphyseal Femoral Fractures: Monitor for thigh, hip, or groin pain; evaluate for fractures. Interruption of OSENVELT therapy should be considered, pending a benefit-risk assessment, on an individual basis.

Hypercalcemia Following Treatment Discontinuation in Patients with Giant Cell Tumor of Bone and in Patients with Growing Skeletons. Clinically significant hypercalcemia, potentially requiring hospitalization, can occur within a year after stopping denosumab in patients with giant cell tumor of bone or growing skeletons; monitor serum calcium and manage calcium and vitamin D needs post-discontinuation.

Multiple Vertebral Fractures (MVF) Following Treatment Discontinuation. Increased risk post-discontinuation of denosumab; evaluate for risk for vertebral fractures after discontinuing OSENVELT.

Embryo-Fetal Toxicity. Denosumab may cause fetal harm; verify pregnancy status before starting OSENVELT and advise effective contraception during treatment and for 5 months after the last dose.

Most common Adverse Reactions:

Bone Metastasis from Solid Tumors (≥ 25%) were fatigue/asthenia, hypophosphatemia, and nausea.
In patients (≥ 10%) with: Multiple Myeloma were diarrhea, nausea, anemia, back pain, thrombocytopenia, peripheral edema, hypocalcemia, upper respiratory tract infection, rash, and headache; Giant Cell Tumor of Bone were arthralgia, headache, nausea, back pain, fatigue, and pain in extremity.
Hypercalcemia of Malignancy (> 20%) were nausea, dyspnea, decreased appetite, headache, peripheral edema, vomiting, anemia, constipation, and diarrhea.

4SC Announces EMA’s Feedback relating to Resminostat’s (Kinselby) Marketing Authorisation Application

On March 3, 2025 4SC AG ("4SC") (Frankfurt Stock Exchange, Prime Standard: VSC; ISIN: DE000A3E5C40) reported that it was informed by the European Medicines Agency (EMA) based on its review of the final data and the Company’s responses to questions, the application for resminostat (Kinselby) an orphan medicinal product for the treatment of patients with CTCL is currently not approvable since "major objections" preclude a recommendation for marketing authorisation at this stage (Day-180 Assessment Report) (Press release, 4SC, MAR 3, 2025, View Source [SID1234650847]).

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4SC will now evaluate the feedback and decide how best to respond to EMA’s objections and list of outstanding issues in line with the given response deadline of one month.

Imfinzi-based perioperative regimen recommended for approval in the EU by CHMP for resectable non-small cell lung cancer

On March 3, 2025 AstraZeneca reported that Imfinzi (durvalumab) in combination with chemotherapy has been recommended for approval in the European Union (EU) for the treatment of adults with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence and no epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements (Press release, AstraZeneca, MAR 3, 2025, View Source [SID1234650846]). In this regimen, patients are treated with Imfinzi in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery.

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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from the pivotal AEGEAN trial, which were published in The New England Journal of Medicine.

Results from a planned interim analysis of event-free survival (EFS) showed a statistically significant and clinically meaningful 32% reduction in the risk of recurrence, progression events or death versus neoadjuvant chemotherapy alone in patients treated with the Imfinzi-based perioperative regimen (32% data maturity; EFS hazard ratio [HR] 0.68; 95% confidence interval [CI] 0.53-0.88; p=0.003902). In a final analysis of pathologic complete response (pCR), treatment with Imfinzi plus neoadjuvant chemotherapy before surgery resulted in a pCR rate of 17.2% versus 4.3% for patients treated with neoadjuvant chemotherapy alone (difference in pCR 13.0%; 95% CI 8.7-17.6).

Additionally, interim overall survival (OS) results presented at the 2024 World Conference on Lung Cancer showed a favourable trend with the Imfinzi-based perioperative regimen (35% data maturity; median OS: not reached [NR] versus 53.2 months; HR=0.89; 95% CI 0.70-1.14). The OS data were not tested for statistical significance at this interim analysis and will continue to be assessed as a key secondary endpoint at final analysis.

Professor Martin Reck, Head of the Department of Thoracic Oncology at the Lung Clinic Grosshansdorf, Germany, member of the AEGEAN Steering Committee and investigator in the trial, said: "Adding perioperative durvalumab to neoadjuvant chemotherapy significantly improved outcomes for patients with resectable non-small cell lung cancer, who experience high rates of recurrence and poor outcomes. Today’s recommendation marks an important step towards patients and their clinicians in Europe gaining access to an innovative treatment that should become a backbone combination approach in this curative-intent setting."

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "This recommendation highlights the potential of Imfinzi to address an unmet need for patients with resectable lung cancer who need new treatment options that increase the time they live without recurrence or progression. AEGEAN underscores our commitment to transforming care in the early stages of lung cancer where there is the greatest potential for cure."

Each year in Europe, there are more than 450,000 people diagnosed with lung cancer.1 Around 25-30% of all patients with NSCLC, the most common form of lung cancer, are diagnosed early enough to have surgery with curative intent.2-3 However, the majority of patients with resectable disease will develop recurrence and only 36-46% of patients with Stage II disease will survive for five years.4-5 This decreases to 24% for patients with Stage IIIA disease and 9% for patients with Stage IIIB disease, reflecting a high unmet medical need.4

Imfinzi was generally well tolerated, and no new safety signals were observed in the neoadjuvant and adjuvant settings. Further, adding Imfinzi to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not compromise patients’ ability to complete surgery versus chemotherapy alone.

Imfinzi is approved in the US and several other countries in this setting based on the AEGEAN results. Regulatory applications are also currently under review in China, Japan and additional countries.

Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy (CRT) based on the PACIFIC Phase III trial.

Notes

Lung cancer
Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths.6-7 Lung cancer is broadly split into NSCLC and small cell lung cancer (SCLC), with 80-85% of patients diagnosed with NSCLC.8-9 An estimated 28,000 people are treated for resectable NSCLC across the five major European countries each year.10

Early-stage lung cancer diagnoses are often only made when the cancer is found on imaging for an unrelated condition.11-12 The majority of patients with resectable disease eventually develop recurrence despite complete tumour resection and adjuvant chemotherapy.5

AEGEAN
AEGEAN is a randomised, double-blind, multi-centre, placebo-controlled global Phase III trial evaluating Imfinzi as perioperative treatment for patients with resectable Stage IIA-IIIB (Eighth Edition AJCC Cancer Staging Manual) NSCLC, irrespective of PD-L1 expression. Perioperative therapy includes treatment before and after surgery, also known as neoadjuvant/adjuvant therapy. In the trial, 802 patients were randomised to receive a 1500mg fixed dose of Imfinzi plus chemotherapy or placebo plus chemotherapy every three weeks for four cycles prior to surgery, followed by Imfinzi or placebo every four weeks (for up to 12 cycles) after surgery. Patients with known EGFR or ALK genomic tumour aberrations were excluded from the primary efficacy analyses.

In the AEGEAN trial, the primary endpoints were pCR, defined as no viable tumour in the resection specimen (including lymph nodes) following neoadjuvant therapy, and EFS, defined as the time from randomisation to an event like tumour recurrence, progression precluding definitive surgery, or death. Key secondary endpoints were major pathologic response, defined as residual viable tumour of less than or equal to 10% in the resected primary tumour following neoadjuvant therapy, disease-free survival, OS, safety and quality of life. The final pathologic response analyses were performed after all patients had the opportunity for surgery and pathology assessment per the trial protocol. The trial enrolled participants from 264 centres in more than 25 countries including in the US, Canada, Europe, South America and Asia.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its indications in resectable, early-stage (IIa-IIIB) NSCLC and unresectable, Stage III NSCLC, Imfinzi is also approved for use in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy (etoposide and either carboplatin or cisplatin) for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU.

Imfinzi is also approved in combination with chemotherapy (carboplatin and paclitaxel) followed by Imfinzi monotherapy in primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR) in the US. In the EU, Imfinzi plus chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient (pMMR) advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi alone is approved for patients with dMMR disease. In Japan, Imfinzi plus chemotherapy followed by Imfinzi monotherapy has also been approved as 1st-line treatment in primary advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi and Lynparza has been approved for patients with pMMR disease.

Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, breast cancer, several gastrointestinal and gynaecologic cancers, and other solid tumours.

Moleculin Abstract Accepted for Poster Presentation at the Acute Leukemias XIX (ISALXIX) International Symposium

On March 3, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported its abstract has been accepted for poster presentation at the Acute Leukemias XIX (ISALXIX) International Symposium being held March 16-19, 2025 in Munich, Germany (Press release, Moleculin, MAR 3, 2025, View Source [SID1234650844]).

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Details for the poster presentation are as follows:

Abstract: P002
Session: Clinical Studies in AML and ALL
Title: Liposomal Annamycin in Combination with Cytarabine for Treatment of Acute Myeloid Leukemia (AML)
Presenting Author: Cristina Papayannidis, MD, PhD, Adjunct professor, Department of Medical and Surgical Sciences, University of Bologna, Bologna Italy

For more information about the ISALXIX International Symposium, visit the event website.

Valneva to Present at Upcoming TD Cowen and Van Lanschot Kempen Healthcare Investor Conferences

On March 3, 2025 Valneva SE (Nasdaq: VALN; Euronext Paris: VLA) a specialty vaccine company, reported that its senior management will participate at upcoming investor conferences in the United States and Europe (Press release, Valneva, MAR 3, 2025, View Source;25.htm [SID1234650842]). CEO Thomas Lingelbach and CFO Peter Bühler will present at the TD Cowen 45th Annual Health Care Conference, taking place March 3 – 5, 2025, in Boston, MA. Mr. Lingelbach and Mr. Peter Bühler will also meet with institutional investors during the Van Lanschot Kempen Life Sciences Conference, held April 2 – 3, 2025, in Amsterdam, The Netherlands.

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Management will discuss Valneva’s pipeline of potential first-in-class vaccine candidates, including its lead Lyme disease vaccine, VLA15, and its Shigella and Zika programs alongside the Company’s portfolio of marketed vaccines, which are expected to generate €170-180 million in revenue in 2025.

The TD Cowen presentation will be webcast live and available as a replay in the "investor" section of the Valneva website at www.valneva.com.

TD Cowen 45th Annual Health Care Conference
Date/Time: March 4, 1:10pm ET
Format: Presentation and investor meetings
Location: Boston, Massachusetts
Webcast Link: https://wsw.com/webcast/cowen177/vla/1855281

Van Lanschot Kempen Life Sciences Conference
Date/Time: April 3
Format: Investor meetings
Location: Amsterdam, The Netherlands

Attending institutional investors who would like to meet with Valneva management are asked to submit a request to their representative at the respective bank.