On November 20, 2023 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, reported its unaudited financial results for the three and nine months ended September 30, 2023 and key corporate highlights (Press release, Legend Biotech, NOV 20, 2023, View Source [SID1234637885]).

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Legend Biotech shared the latest updates from its portfolio and pipeline, alongside its financial performance, including detailing Legend Biotech’s license agreement with Novartis. The license agreement grants Novartis the exclusive, worldwide rights to certain potential CAR-T therapies selectively targeting DLL3.

"We continuously explore the full potential of our products and technologies. The out-license agreement with Novartis affirms that our next-generation therapy, LB2102, has the potential to be a differentiated treatment for eligible patients with small cell lung cancer," said Ying Huang, Chief Executive Officer of Legend Biotech. "We also remain committed to meeting the demand for CARVYKTI, in collaboration with Janssen, and have progressively increased manufacturing capacity, which has led to an incremental increase in sales."

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1 ClinicalTrials.gov. DLL3-Directed Chimeric Antigen Receptor T-cells in Subjects With Extensive Stage Small Cell Lung Cancer. Available at: View Source Last accessed Aug 2023.
2 ClinicalTrials.gov. A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma (CARTITUDE-6). Available at: View Source

Financial Results for Quarter Ended September 30, 2023

Cash and Cash Equivalents, Time Deposits, and Short-Term Investments

As of September 30, 2023, Legend Biotech had approximately $1.4 billion of cash and cash equivalents, time deposits, and short-term investments.

Revenue

License Revenue

License revenue for the three months ended September 30, 2023 was $20.1 million compared to no license revenue for the three months ended September 30, 2022. The increase was due to the achievement of a milestone under our collaboration and license agreement (Janssen Agreement) with Janssen Biotech, Inc. (Janssen) during the three months ended September 30, 2023. License revenue for the nine months ended September 30, 2023 was $35.2 million, compared to $50.0 million for the nine months ended September 30, 2022. This decrease of $14.8 million was primarily driven by the nature and timing of milestones achieved as outlined in the Global Development Plan under the Janssen Agreement for cilta-cel.

Collaboration Revenue

Collaboration revenue for the three and nine months ended September 30, 2023 was $75.9 million and $170.4 million, respectively, compared to $27.3 million and $39.2 million for the three and nine months ended September 30, 2022. The increases of $48.6 million and $131.2 million for the three and nine month periods, respectively, were due to an increase in revenue generated from sales of CARVYKTI in connection with the Janssen Agreement.

Operating Expenses

Collaboration Cost of Revenue

Collaboration cost of revenue for the three and nine months ended September 30, 2023 was $43.5 million and $111.8 million, respectively, compared to $25.5 million and $42.4 million for the three and nine months ended September 30, 2022. The increases of $18.0 million and $69.4 million for the three and nine months periods, respectively, were a combination of Legend Biotech’s share of the cost of sales in connection with CARVYKTI sales under the Janssen Agreement and expenditures to support expansion in manufacturing capacity that could not be capitalized.

Research and Development Expenses

Research and development expenses for the three and nine months ended September 30, 2023 were $95.9 million and $276.5 million, respectively, compared to $104.5 million and $254.9 million for the three and nine months ended September 30, 2022, respectively. The decrease of $8.6 million for the three months ended September 30, 2023 compared to three months ended September 30, 2022 was due to timing of expenses incurred in connection with the Global Development Plan under the Janssen Agreement. The increase of $21.6 million for the nine months ended September 30, 2023 compared to the nine months ended September 30, 2022 was primarily due to continuous research and development activities in cilta-cel, including higher patient enrollment for Phase 3 clinical trials for cilta-cel, and an increase in research and development activities for other pipeline items. The other pipeline expenses include continued investment in Legend Biotech’s solid tumor programs, which include two Investigational New Drug approvals that advanced into Phase 1 development.

Administrative Expenses

Administrative expenses for the three and nine months ended September 30, 2023 were $28.1 million and $78.1 million, respectively, compared to $23.2 million and $54.0 million for the three and nine months ended September 30, 2022, respectively. The increases of $4.9 million and $24.1 million for the three and nine month periods, respectively, were primarily due to the expansion of administrative functions to facilitate continuous business growth and continued investment in building Legend Biotech’s global information technology infrastructure.

Selling and Distribution Expenses

Selling and distribution expenses for the three and nine months ended September 30, 2023 were $21.1 million and $60.5 million, respectively, compared to $18.9 million and $67.6 million for the three and nine months ended September 30, 2022. The increase of $2.2 million for the three months ended September 30, 2023 compared to the three months ended September 30, 2022 was due to costs associated with the commercialization of CARVYKTI. The decrease of $7.1 million for the nine months ended September 30, 2023 compared to the nine months ended September 30, 2022 was primarily due to non-recurring launch expenses incurred during the nine months ended September 30, 2022 to support the commercial launch of CARVYKTI in the U.S market.

Other Income and Gains

Other income and gains for the three and nine months ended September 30, 2023 were $35.8 million and $49.8 million, respectively, compared to $3.9 million and $4.7 million for the three and nine months ended September 30, 2022, respectively. The increases of $31.9 million and $45.1 million for the three and nine month periods, respectively, were primarily attributable to an increase in interest income, fair value gain on financial assets and foreign currency exchange gain.

Other Expenses

Other expenses for the three and nine months ended September 30, 2023 were $0.1 million and $0.2 million, respectively, compared to $2.0 million and $9.5 million for the three and nine months ended September 30, 2022. The decrease in both comparative periods was primarily due to an unrealized foreign currency exchange gain in 2023 and an unrealized foreign currency exchange loss in 2022.

Finance Costs

Finance costs for the three and nine months ended September 30, 2023 were $5.7 million and $16.0 million, respectively, compared to $3.2 million and $5.9 million for the three and nine months ended September 30, 2022. The increase in both comparative periods was primarily due to interest on advance funding, which is interest-bearing borrowings funded by Janssen under the Janssen Agreement and constituted of principal and applicable interests upon such principal.

Fair Value (Loss)/Gain of Warrant Liability

There was no fair value (loss)/gain of warrant liability for the three months ended September 30, 2023 compared to a gain of $61.2 million for the three months ended September 30, 2022, because the warrant was exercised on May 11, 2023. Fair value loss of warrant liability for the nine months ended September 30, 2023 was $85.8 million, compared to a fair value gain of $30.2 million for the nine months ended September 30, 2022. The increase was due to the fair value loss recorded on the full exercise of the warrant, which took place on May 11, 2023.

Loss for the Period

For the three months ended September 30, 2023, net loss was $62.2 million, or $0.17 per share, compared to net loss of $85.0 million, or $0.26 per share, for the three months ended September 30, 2022. For the nine months ended September 30, 2023, net loss was $373.4 million, or $1.07 per share, compared to a net loss of $310.5 million, or $0.99 per share, for the nine months ended September 30, 2022.

Webcast/Conference Call Details:

Legend Biotech will host its quarterly earnings call and webcast today at 8:00am ET. To access the webcast, please visit this weblink.

A replay of the webcast will be available on Legend Biotech’s website at View Source

On November 20, 2023 Calidi Biotherapeutics, Inc. (NYSE American: CLDI or "Calidi"), a clinical-stage biotechnology company developing a new generation of targeted immunotherapies, reported the issuance of a new patent for the company’s SuperNova technology, strengthening its intellectual property portfolio and positioning Calidi to advance its CLD-201 program into the clinic (Press release, Calidi Biotherapeutics, NOV 20, 2023, View Source [SID1234637884]). In addition, the company also provided an update on the timing of its anticipated clinical milestones.

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The United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,655,455 titled, "Enhanced systems for cell-mediated oncolytic viral therapy," directed to Calidi’s SuperNova platform composed of adipose-derived mesenchymal stem cells loaded with oncolytic vaccinia virus.

"This latest patent granted to Calidi covering meaningful features of our SuperNova platform represents an important milestone as we prepare to advance CLD-201 into the clinic," said Allan Camaisa, CEO and Chairman of the Board of Calidi Biotherapeutics. "We are pleased to continue strengthening an already robust intellectual property portfolio, and feel we are well-positioned to capture the potential of our efforts to develop a universal and off-the-shelf SuperNova platform to radically transform the landscape for patients with advanced solid tumors."

The patent covers a composition where cells are incubated with an oncolytic virus for a defined period allowing the expression of at least one immunomodulatory protein or recombinant therapeutic protein encoded by the virus, by virtue of association of the virus, with the carrier cell. Early Calidi research has shown the potential ability of the SuperNova platform to shield the viral payload from the immune system, supporting efficient delivery to tumor sites and effectively potentiating oncolytic viruses’ therapeutic efficacy.

"We see great potential in our SuperNova technology to address the vast unmet need for effective treatments of solid tumor cancers," said Dr. Antonio F. Santidrian, Chief Scientific Officer at Calidi Biotherapeutics. "We look forward to initiating our first-in-human clinical trial of our off-the-shelf and allogeneic therapy in 2024."

In addition, Calidi also provided updates on the timing for certain anticipated clinical milestones for its NeuroNova and SuperNova platforms.

Anticipated Clinical Milestones

1H 2024: First patient dosed in CLD-101 (NeuroNova) Phase 1b/2 trial in collaboration with Northwestern University for newly diagnosed high-grade glioma patients
2H 2024: First patient dosed in CLD-201 (SuperNova) Phase 1 trial

On November 20, 2023 Therapeutic Solutions International, Inc. (TSOI), reported the publication of a peer-reviewed paper entitled "Advancing personalized medicine in brain cancer: exploring the role of mRNA vaccines" which appeared in the November 2023 issue of the Journal of Translational Medicine (Press release, Therapeutics Solutions International, NOV 20, 2023, View Source [SID1234637883]). The article was co-authored by Therapeutic Solutions International Board Member Dr. Thomas Ichim, Chief Scientific Officer Dr. Feng Lin, and Scientific Advisory Board Members Drs Santosh Kesari and Francesco Marincola.

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In the publication, advances in stimulation of cancer-specific and patient-specific immunity to brain cancer are discussed, as well as methods of augmenting existing immunity. The paper provides several explanations regarding the advantages of mRNA immunization, as well as discusses the means of integrating immunotherapy with other currently used approaches to brain cancer.

"Therapeutic Solutions International has a long history of success in cancer immunotherapy going back to 2016 when the Company patented the use of pterostilbene combinations for enhancing the ability of the immune system to kill cancer2," said Dr. Thomas Ichim. "We are honored to continue our work in this field with Key Opinion Leaders such as our esteemed coauthors."

Currently, the Company is developing its clinical-stage immune-oncology assets through the spin-off Res Nova Biologics, which has treated cancer patients under the Right to Try law with both its ValloVax3 and FloraStilbene4 products.

"Our Company is guided by science. In order to remain at the cutting edge, it is fundamental to collaborate with other companies and institutions in understanding the state of the art and knowing which areas need advancing," said Timothy Dixon, President, and CEO. "I am grateful for the team that put together this excellent publication and look forward to numerous publications as our products continue advancing towards commercialization."

On November 20, 2023 Foundation Medicine Inc. reported that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic for AstraZeneca’s Truqap (capivasertib) in combination with Faslodex (fulvestrant) which has been contemporaneously approved for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations following progression on at least one endocrine-based regimen in the metastatic setting or recurrence on or within 12 months of completing adjuvant therapy (Press release, Foundation Medicine, NOV 20, 2023, View Source [SID1234637882]).

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Approximately 70% of advanced breast cancers are HR-positive,1 and PI3K-AKT pathway alterations occur in the tumors of up to 50% of these HR-positive patients.2 Endocrine therapy, the mainstay first line treatment in combination with CDK4/6 inhibition, does not stop disease progression for most of these patients and treatment thereafter remains a clinical challenge. CAPItello-291, a double-blind, phase 3, randomized trial showed that the addition of capivasertib to fulvestrant therapy resulted in a significant improvement in progression-free survival among patients with HR-positive, HER2-negative PIK3CA/AKT1/PTEN-altered advanced breast cancer whose disease had progressed during or after previous aromatase inhibitor therapy with or without a CDK4/6 inhibitor.3 FoundationOne CDx will be the first FDA-approved test to identify this new, prevalent subset of breast cancer patients for treatment with capivasertib.4

"We are thrilled to see an additional therapeutic option approved to help treat the high number of HR-positive breast cancer patients," said Troy Schurr, Chief Biopharma Business Officer at Foundation Medicine. "This companion diagnostic approval adds to the growing utility of Foundation Medicine’s high-quality diagnostic test portfolio in treating advanced breast cancer."

Foundation Medicine is the only company with an FDA-approved portfolio of tissue and blood-based comprehensive genomic profiling tests. Using a tissue sample, the FDA-approved FoundationOne CDx test analyzes more than 300 cancer-related genes for genomic alterations in a patient’s tumor. The test currently has over 35 companion diagnostic indications, five of which are in breast cancer.

"The prevalence of HR-positive breast cancer means the introduction of new targeted treatment options for this patient population will have an exponential impact," said Shehzin Tietjen, Associate Director, Corporate Relations at Living Beyond Breast Cancer. "Biomarker testing is such an important component of getting patients on the right therapy for their specific cancer, and we’re encouraged to see additional companion diagnostic indications being approved to help increase patient access to precision medicine."

Foundation Medicine is the global leader in companion diagnostic approvals. The company has 60% of all U.S. companion diagnostic approvals for next generation sequencing (NGS) testing.

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

On November 20, 2023 The Menarini Group ("Menarini"), a leading Italian pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group focused on bringing transformational oncology treatments to cancer patients, reported that they will present new data related to ORSERDU (elacestrant) and ELZONRIS (tagraxofusp-erzs) in two upcoming congresses (Press release, Menarini, NOV 20, 2023, View Source [SID1234637881]).

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ORSERDU, a once-daily oral endocrine monotherapy, for the treatment of postmenopausal women or adult men, with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, was approved by the U.S. Food and Drug Administration in January 2023 under its priority review and fast track designation. In September 2023, ORSERDU was approved by the European Commission.

Updated data, including a spotlight discussion on additional biomarker and clinical subgroup analyses from the Phase 3 EMERALD trial, along with new safety updates evaluating elacestrant in multiple combination settings, will be presented at the San Antonio Breast Cancer Symposium (SABCS), December 5-9, 2023.

ELZONRIS was approved by the FDA in December 2018 for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adult and pediatric patients two years and older, in both treatment-naïve and previously-treated populations. In January 2021, ELZONRIS was approved by the European Commission. Updated data, including an oral presentation of updated real-world results in treatment-naïve patients with BPDCN demonstrating durable outcomes and a manageable safety profile leading to prolonged survival, will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, December 9-12, 2023.

"The breadth of data on our novel oncology therapies, spanning solid tumors and hematologic malignancies, underscore our commitment to addressing significant unmet medical needs in difficult-to-treat cancers," said Elcin Barker Ergun, CEO of the Menarini Group. "At Menarini Stemline, we are dedicated to driving innovation in oncology to deliver targeted therapies that bring value to people living with cancer and to the healthcare providers who care for them."

See below for full details of the Menarini Group/Stemline Therapeutics’ upcoming presentations:

San Antonio Breast Cancer Symposium 2023

Lead Author​

Abstract Title and ID​

Presentation Details​

Bardia, A​

Elacestrant vs standard-of-care in ER+/HER2- advanced or metastatic breast cancer (mBC) with ESR1 mutation: key biomarkers and clinical subgroup analyses from the phase 3 EMERALD trial​

1576519/PS17-02

December 8, 2023​

7-8am CT

Poster Spotlight Discussion

Hemisfair Ballroom 1-2

Rugo, H​

ELEVATE: A phase 1b/2, open-label, umbrella study evaluating elacestrant in various combinations in patients (pts) with estrogen receptor-positive (ER+), HER2-negative (HER2-) locally advanced or metastatic breast cancer (mBC)​

1576517/PO2-05-04

December 6, 2023

5-7pm CT​

Poster

Halls 2-3

Ibrahim, N​

ELECTRA: An open-label, multicenter, phase 1b/2 study of elacestrant in combination with abemaciclib in patients with brain metastasis (mets) from estrogen receptor-positive (ER+), HER2-negative (HER2-) breast cancer (BC)

1576518​/PO2-05-05

December 6, 2023​

5-7pm CT​

Poster

Halls 2-3

Patnaik, A​

SUMIT-ELA: Phase 1b/2 combination of cyclin-dependent kinase 7 inhibitor (CDK7i) samuraciclib and selective estrogen receptor degrader (SERD) elacestrant in advanced hormone receptor positive (HR+) breast cancer after CDK4/6i*

NA/PO3-04-13

December 7, 2023

12-2pm CT​

Poster

Halls 2-3

Bellet, M​

A phase 2 randomized pre-operative, window of opportunity trial investigating the effect of elacestrant with/without triptorelin in premenopausal patients with HR+/HER2- breast cancer – SOLTI-2104-PremiÈRe trial*​

1580190/PO3-19-08

December 7, 2023​

12-2pm CT​

Poster

Halls 2-3

*Denotes investigator sponsored research or collaborative research

65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2023

Lead Author

Title and Publication Number

Session Name

Presentation Details

Angelucci et al.

Durable Outcomes with Manageable Safety Leading to Prolonged Survival with Tagraxofusp for Treatment-Naive Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm: Updated Results from a European Named Patient Program

Publication Number: 547

906. Outcomes Research – Myeloid Malignancies: Real-World Treatment Patterns and Outcomes

Oral Presentation

December 10, 2023

12:00 PM

Marriott Marquis San Diego Marina, Marriott Grand Ballroom 11-13

Minetto et al.

Single Agent Tagraxofusp in Relapsed/Refractory CD123-Positive Acute Myeloid Leukemia: An Interim Analysis of Italian GIMEMA AML2020 Trial*

Publication Number: 2918

616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II

Poster Presentation

December 10, 2023

6:00 PM – 8:00 PM

San Diego Convention Center, Halls G-H

Lane et al.

Tagraxofusp in Combination with Azacitidine and Venetoclax in Newly Diagnosed CD123+ Acute Myeloid Leukemia, Expansion Cohort of a Phase 1b Multicenter Trial*

Publication Number: 4277

616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III

Poster Presentation

December 11, 2023

6:00 PM – 8:00 PM

San Diego Convention Center, Halls G-H

Boichut et al.

Analysis of tagraxofusp activity in AML-PDC as a single agent and in combination with BCL2 inhibitors*

Publication Number: 2783

604. Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms: Poster II

Poster Presentation

December 10, 2023

6:00 PM – 8:00 PM

San Diego Convention Center, Halls G-H

Navarro Vicente et al.

Clinical Features and Treatment in Patients Diagnosed with Blastic Plasmacytoid Dendritic Cell Neoplasm: Interim Analysis from the Pethema Epidemiologic Registry (EPI-BLAS study)*

Publication Number: 4234

613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster III

Poster Presentation

December 11, 2023

6:00 PM – 8:00 PM

San Diego Convention Center, Halls G-H

*Denotes investigator sponsored research or collaborative research

About the EMERALD Phase 3 Study (NCT03778931)
The EMERALD Phase 3 trial is a randomized, open label, active-controlled study evaluating elacestrant as second- or third-line monotherapy in ER+, HER2- advanced/mBC patients. The study enrolled 478 patients who had received prior treatment with one or two lines of endocrine therapy, including a CDK4/6 inhibitor. Patients in the study were randomized to receive either elacestrant or the investigator’s choice of an approved hormonal agent. The primary endpoints of the study were progression-free survival (PFS) in the overall patient population and in patients with estrogen receptor 1 gene (ESR1) mutations. In the group of patients whose tumors had ESR1 mutations, elacestrant achieved a median PFS of 3.8 months vs 1.9 months on the SOC, and reduced the risk of progression or death by 45% (PFS HR=0.55, 95% CI: 0.39, 0.77) vs SOC.

About ORSERDU (elacestrant)
U.S. Indication: ORSERDU (elacestrant), 345 mg tablets, is approved by the U.S. Food & Drug Administration (FDA) for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

Full prescribing information for the U.S. can be found at www.orserdu.com

Important Safety Information, ORSERDU
Warning and Precautions

Dyslipidemia: Hypercholesterolemia and hypertriglyceridemia occurred in patients taking ORSERDU at an incidence of 30% and 27%, respectively. The incidence of Grade 3 and 4 hypercholesterolemia and hypertriglyceridemia were 0.9% and 2.2%, respectively. Monitor lipid profile prior to starting and periodically while taking ORSERDU.

Embryo-Fetal Toxicity: Based on findings in animals and its mechanism of action, ORSERDU can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the final dose.

Adverse Reactions

Serious adverse reactions occurred in 12% of patients who received ORSERDU. Serious adverse reactions in >1% of patients who received ORSERDU were musculoskeletal pain (1.7%) and nausea (1.3%). Fatal adverse reactions occurred in 1.7% of patients who received ORSERDU, including cardiac arrest, septic shock, diverticulitis, and unknown cause (one patient each).

The most common adverse reactions (>10%), including laboratory abnormalities, of ORSERDU were musculoskeletal pain (41%), nausea (35%), increased cholesterol (30%), increased AST (29%), increased triglycerides (27%), fatigue (26%), decreased hemoglobin (26%), vomiting (19%), increased ALT (17%), decreased sodium (16%), increased creatinine (16%), decreased appetite (15%), diarrhea (13%), headache (12%), constipation (12%), abdominal pain (11%), hot flush (11%), and dyspepsia (10%).

Drug interactions

Concomitant use with CYP3A4 Inducers and/or inhibitors: Avoid concomitant use of strong or moderate CYP3A4 inhibitors with ORSERDU. Avoid concomitant use of strong or moderate CYP3A4 inducers with ORSERDU.

Use in specific populations

Lactation: Advise lactating women to not breastfeed during treatment with ORSERDU and for 1 week after the last dose.

Hepatic Impairment: Avoid use of ORSERDU in patients with severe hepatic impairment (Child-Pugh C). Reduce the dose of ORSERDU in patients with moderate hepatic impairment (Child-Pugh B).

The safety and effectiveness of ORSERDU in pediatric patients have not been established.

To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Elacestrant is also being investigated in several clinical trials in metastatic breast cancer disease, alone or in combination with other therapies: ELEVATE (NCT05563220); ELECTRA (NCT05386108); and ELCIN (NCT05596409). Elacestrant is also being evaluated in early breast cancer disease.

About ELZONRIS (tagraxofusp-erzs)
U.S. Indication: ELZONRIS (tagraxofusp-erzs) is a prescription medicine used to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients 2 years and older.

Full prescribing information for the U.S. can be found at www.elzonris.com

IMPORTANT SAFETY INFORMATION, ELZONRIS
Boxed WARNING: CAPILLARY LEAK SYNDROME

Capillary Leak Syndrome (CLS) which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended.
Warnings and Precautions
Capillary Leak Syndrome

Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range – 1 to 46 days), and all but 5 patients experienced an event in Cycle 1.

Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability.

Hypersensitivity Reactions

ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur.

Hepatotoxicity

Treatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption.

Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved.

Adverse Reactions

Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST.

Please see full Prescribing Information, including Boxed WARNING.
To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.