On August 14, 2023 Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, reported financial results for the three and six months ended June 30, 2023 and provides a business update (Press release, Biocept, AUG 14, 2023, View Source [SID1234634441]).

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"Biocept’s primary focus is establishing our proprietary cerebrospinal fluid assay CNSide as standard of care. We continue to diligently work towards submission to the National Comprehensive Cancer Network (NCCN) for consideration to include CNSide in their standard-of-care guidelines. We believe securing this status will broaden physician adoption and support reimbursement that reflects our test’s value in clinical decision-making," said Antonino Morales, Biocept President and CEO. "Our ongoing FORESEE clinical trial is powered to generate data in support of this goal by assessing CNSide’s impact on physicians’ treatment decisions. I’m exceptionally pleased that patient enrollment in FORESEE has passed the midpoint, with four clinical sites open around the country for patient recruitment and several additional medical centers expected to join in the coming weeks.

"We plan to provide further evidence of CNSide’s clinical utility through publication in peer-reviewed medical journals. We have submitted a manuscript with a description of our assay and its features, validation from pilot studies and compelling case studies showing actual use in patient management. Four additional manuscripts are being prepared in collaboration with leading neuro-oncologists for submission to scientific journals, including several documenting their clinical experiences with CNSide in their practices," Mr. Morales continued.

"We right sized our business to align with our primary focus, which is helping to extend our cash runway. Progressing towards standard of care, completing the FORESEE clinical trial, and reducing expenses are key to Biocept becoming a self-sustaining business," Mr. Morales added.

Earlier today Biocept announced that Priya U. Kumthekar, MD, a United Counsel for Neurologic Subspecialties (UCNS)-certified neuro-oncologist at Northwestern University, and David Piccioni, MD, PhD, Director of Neuro-Oncology at University of California, San Diego, discussed the use of CNSide in presentations at the 2023 SNO/ASCO CNS Cancer Conference, which was held last week.

Biocept intends to host a business update call later in August to present a progress report on the FORESEE trial and discuss other recent developments. Details of the call will be announced in a press release.

Second Quarter Financial Results

Net revenues for the second quarter of 2023 were $0.6 million, compared with $5.8 million for the second quarter of 2022, with the decline due to lower RT-PCR COVID-19 testing volume. As previously reported, the Company ceased providing COVID-19 testing services in February 2023. The number of commercial accessions delivered for the second quarters of 2023 and 2022 were 322 and 77,779, respectively.

Cost of revenues for the second quarter of 2023 was $2.6 million, compared with $8.0 million for the second quarter of 2022, with the decrease primarily due to the cessation of COVID-19 testing services and reduced headcount.

Research and development (R&D) expenses for the second quarter of 2023 were $0.4 million, compared with $1.7 million for the second quarter of 2022, with the decrease primarily due to a reduction in headcount and lower purchases of materials and supplies.

General and administrative (G&A) expenses for the second quarter of 2023 were $3.5 million, compared with $4.3 million for the second quarter of 2022. The decrease was primarily due to lower headcount and consulting fees.

Sales and marketing expenses for the second quarter of 2023 were $0.3 million, compared with $1.7 million for the second quarter of 2022, with the decrease primarily due to a reduction in headcount, and lower consulting, promotion and outside service-related expenses.

Non-cash change in the fair value of warrant liability for the second quarter of 2023 was $2.4 million. There was no comparable item for the second quarter of 2022.

Net loss attributable to common stockholders for the second quarter of 2023 was $3.6 million, or $3.50 per share, compared with a net loss attributable to common stockholders for the second quarter of 2022 of $10.0 million, or $17.82 per share.

Six Month Financial Results

Net revenues for the first half of 2023 were $1.3 million, compared with $25.8 million for the first half of 2022.

Operating expenses for the first half of 2023 were $14.5 million, and included cost of revenues of $5.6 million, R&D expenses of $1.4 million, G&A expenses of $6.5 million and sales and marketing expenses of $1.0 million.

Net loss attributable to common stockholders for the first half of 2023 was $10.8 million, or $13.25 per share, compared with net loss attributable to common stockholders for the first half of 2022 of $12.3 million, or $21.78 per share.

Biocept reported cash of $6.6 million as of June 30, 2023, compared with $12.9 million as of December 31, 2022. In May 2023, the Company received net cash proceeds of approximately $3.6 million from an underwritten public offering, after deducting underwriting discounts and other expenses payable by the Company.

About the FORESEE Clinical Trial

The multi-center, prospective FORESEE clinical trial is a longitudinal therapy response monitoring study in subjects with leptomeningeal metastases (LM) using CNSide (CSF Tumor Cells) compared to standard of care (CSF cytology, clinical evaluation, and imaging). The goal of the FORESEE trial is to evaluate the performance of CNSide in monitoring the response of LM to treatment and to assess the impact of CNSide on treatment decisions made by physicians. The trial is enrolling up to 40 patients with breast or non-small cell lung cancer (NSCLC) who have suspected or confirmed LM. Standard of care methods to diagnose or assess the treatment response of LM have limited sensitivity and specificity. This creates challenges for physicians to manage LM or determine the best course of treatment. CNSide is a Laboratory Developed Test (LDT) that is used commercially at the physician’s discretion in Biocept’s CLIA-certified, CAP-accredited laboratory.

On August 14, 2023 Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, reported a business update and provided financial results for the quarter ended June 30, 2023 (Press release, Gamida Cell, AUG 14, 2023, View Source [SID1234634410]).

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"Since the approval of Omisirge in April, Gamida Cell has made excellent progress on the launch," said Abbey Jenkins, President and Chief Executive Officer of Gamida Cell. "We have rapidly confirmed significant payer coverage and are seeing high interest from transplant centers, exceeding our plans. This is a testament to the unmet needs that Omisirge has the potential to address for patients, as well as the exceptional work of our experienced cell therapy team. We continue to progress our discussions in our efforts to secure a strategic deal to fully resource our Omisirge commercial efforts. We are optimistic that between our progress on the Omisirge launch and our discussions with potential partners, we can accelerate efforts to expand patient access to Omisirge."

The U.S. Food and Drug Administration (FDA) approved Omisirge in April 2023 for use in adult and pediatric patients 12 years and older with hematologic malignancies planned for umbilical cord blood transplantation. The Omisirge launch is ahead of expectations in terms of both transplant center onboarding and market access, with 12 transplant centers already onboarded, eight more in the onboarding process, and active engagement with virtually all of the top 70 transplant centers. The company has also confirmed coverage with payers that cover more than 85% of commercial lives, as well as confirmed coverage and reimbursement with Medicare from the Centers for Medicare and Medicaid Services (CMS). All documents are in place for coverage under the Department of Veterans Affairs, Department of Defense and Medicaid.

"We are very encouraged by the strong interest we have received from transplant centers to date, including centers that did not participate in our clinical studies," said Michele Korfin, Chief Operating and Chief Commercial Officer of Gamida Cell. "The enthusiasm for Omisirge is consistent with expectations from the market research we conducted both pre- and post-approval, supporting our assertion that Omisirge has the potential to capture approximately 20% peak market share. We anticipate this peak share will include patients who might have been otherwise transplanted with a different donor source, as well as patients who historically would not have been able to find an appropriate donor. We have onboarded more centers at this point in our launch than we initially anticipated and are ahead of our expectations in terms of payer coverage. We look forward to engaging with and onboarding additional transplant centers in the coming months to provide more access to Omisirge for patients."

Second Quarter Highlights and Recent Developments

Omisirge

Commercial progress:
The company is seeing strong demand from transplant centers and is ahead of its goal of onboarding 10-15 transplant centers in 2023. As of the morning of August 14, 2023, 12 transplant centers have been onboarded and eight additional transplant centers are in the onboarding process.
Patients are enrolled in the company’s Gamida Cell Assist program, which means that their transplanter has the intent to utilize Omisirge as the patient’s donor source.
The company has confirmed coverage with payers who cover more than 85% of commercial lives and is in ongoing discussions with additional commercial payers.
CMS coverage and reimbursement has been confirmed for patients covered by Medicare. All required documentation is in place for patients who are covered under Medicaid, Department of Defense and Veterans Affairs.
The company is ready to reliably deliver Omisirge within 30 days from the start of manufacturing.
Publication: The company announced the publication of a prospective sub-study of the Phase 3 clinical trial for Omisirge titled "Immune Reconstitution Profiling Suggests Antiviral Protection After Transplantation with Omidubicel: A Phase 3 Substudy." The publication is available online on the Transplantation and Cellular Therapy journal website.
Corporate Developments

Strategic review: The company continues to work with Moelis & Company LLC to engage and advance discussions with multiple parties as part of its efforts to explore alternatives to support a fully resourced launch.
Terry Coelho, MBA, appointed Chief Financial Officer: In May 2023, Gamida Cell announced the appointment of Terry Coelho as Chief Financial Officer. Ms. Coelho, a seasoned finance executive with more than 35 years of experience across all areas of finance and business development at emerging growth companies and established global companies, is positioned to support Gamida Cell’s transition into a commercial-phase company and its pursuit of a strategic partnership.
Jeremy Blank appointed to Board of Directors: In August 2023, the company’s Board of Directors appointed Jeremy Blank, Chief Investment Officer of Community Fund, to the Board, where he will serve on the Transactions Committee. "Having closely followed Gamida Cell for over a decade, it is a privilege to join the Board of Directors," said Mr. Blank. "I have high confidence in Gamida’s team and technology and our firm invested in both of Gamida’s most recent equity offerings. I look forward to supporting Gamida’s strategic process which is a great opportunity to create shareholder value." Mr. Blank will provide a valuable perspective to the Board as it aids the company in executing its corporate strategy. From 2005 to 2020, Mr. Blank also served as a partner at York Capital Management, a $15 billion global investment fund. Before that, he was a Vice President and credit analyst at Morgan Stanley. Mr. Blank currently serves as a Director on the boards of Insightec Ltd. and Advanced Emissions Solutions, Inc. (NASDAQ: ADES). Community Fund is a significant shareholder in Gamida Cell.
Strengthened balance sheet: The company ended the second quarter with $54.1 million in cash as a result of the net proceeds of $29.8 million from both the April equity offering and the at-the-market offering (ATM), thereby extending cash runway into the second quarter of 2024. Additionally, the company reduced its outstanding principal balance in the quarter by $9.0 million, from $94.0 million to $85.0 million.
Investor Day: Gamida Cell hosted an Investor Day on June 29, 2023. The event was attended by over 180 participants, both in-person and virtual, and included an update on the commercial launch of Omisirge as well as discussions with industry thought leaders. Steven Devine, M.D., Chief Medical Officer at the National Marrow Donor Program (NMDP)/Be The Match, discussed the unmet needs and barriers to care in stem cell transplant, and explained how Omisirge can help address some of these issues. Usama Gergis, M.D., MBA, Professor of Oncology & Director of Transplant and Cellular Therapy, Sidney Kimmel Cancer Center, Thomas Jefferson University, discussed the implications of a health outcomes economics research model developed in partnership with Gamida Cell that showed that a 20% peak market share of Omisirge may increase transplant access for eligible patients, particularly those from racially and ethnically diverse groups that are currently underserved.
GDA-201

Phase 1 study: Gamida Cell’s GDA-201, an intrinsic natural killer (NK) cell therapy candidate being investigated for the treatment of hematologic malignancies, is being evaluated in an ongoing Phase 1 study for treatment of non-Hodgkin lymphoma. The study is continuing to enroll patients at six sites in the U.S. Data are expected in the first quarter of 2024.
Data presented at International Society for Cell and Gene Therapy (ISCT) 2023 Annual Meeting: GDA-201 data presented at ISCT on June 2, 2023 demonstrated the robustness of cryopreserved GDA-201 and continued to support signs of strong lymphoid homing and decreased exhaustion of GDA-201 cells.
New data on nicotinamide (NAM) mechanism of action: Data published in Science Translational Medicine in the July 19 edition demonstrated that culturing NK cells with NAM, which the company uses to enhance and expand cells, and IL-15 increases metabolic fitness, energy charge and glucose flux within NK cells, providing a pathway for resistance to oxidative stress and increased potency compared to NK cells cultured with IL-15 but without NAM. Through preventing degradation of the protein FOXO1, the presence of NAM was found to increase levels of CD62L, an important lymph node homing marker. This expression cascade provides evidence for the strong lymph node homing, direct killing and antibody dependent cell-mediated cytotoxicity capabilities of NK cells cultured with NAM. The publication also includes outcome data from the Phase 1 dose escalation clinical trial for Gamida Cell’s NK cell therapy candidate GDA-201 in patients with non-Hodgkin lymphoma as well as translational data from biopsy specimens.
Second Quarter 2023 Financial Results

Research and development expenses were $8.7 million in the second quarter of 2023, compared to $10.6 million in the same quarter in 2022. The decrease of $1.9 million was primarily due to a $1.6 million reduction associated with the discontinuation of development of our engineered NK cell therapy pipeline, and $0.7 million in lower Omisirge Phase 3 spend, including a decrease in payments for manufacturing services, partially offset by a decrease of $0.4 million in IIA (Israeli Innovation Authority) income.

Commercial expenses were $3.9 million in the second quarter of 2023, compared to $3.2 million in the second quarter of 2022. The increase of $0.7 million was attributable to an increase in launch readiness activities.
General and administrative expenses were $6.3 million in the second quarter of 2023, compared to $4.3 million in the same period in 2022. The increase of $2.0 million was associated with higher professional services expenses, in part due to the April 2023 follow on offering and business development activities.

Financial expenses, net, were $12.9 million in the second quarter of 2023, compared to $0.5 million in the same period of 2022. The increase of $12.4 million was primarily due to non-cash expenses totaling approximately $10.4 million, including the fair value impact on our warrants liability of $4.9 million, the fair value impact on the 2022 convertible note of $4.3 million, and a decrease in capitalization of finance costs to fixed assets of $0.6 million. In addition, the increase was due to higher cash expenses of $2.0 million, including $1.7 million in issuance costs from our April 2023 underwritten public offering and an increase of $1.0 million in interest expenses associated with the 2022 convertible notes, partially offset by increased interest income of $0.5 million.
Net loss was $31.7 million in the second quarter of 2023, compared to a net loss of $18.6 million in the second quarter of 2022, driven primarily by the increase in financial expenses of $12.4 million.
Cash Position: As of June 30, 2023, Gamida Cell had total cash and cash equivalents of $54.1 million compared to $64.7 million as of December 31, 2022. The decrease of $10.4 million is due primarily to net cash used in operating activities of $44.3 million partially offset by $34.7 million in net cash proceeds from the issuance of shares and warrants from our underwritten public offering and the issuance of shares via the ATM facility. Subsequent to the quarter close and through August 9th, the company raised an additional $14.0 million in net proceeds via the ATM facility. The company expects its current cash and cash equivalents, including the funds raised subsequent to the close of the second quarter, to support its ongoing operating activities into the second quarter of 2024, based on Gamida Cell’s current operational plans and excluding commercialization activities beyond the initial launch of Omisirge, as well as any additional financing activities that may be undertaken.
Debt Position: As of June 30, 2023, the company had reduced its principal balance on the 2022 secured convertible note by $15.0 million, from $25.0 million as of December 31, 2022, to $10.0 million at the end of the second quarter. The company also holds a 2021 convertible senior note with an aggregate principal amount of $75.0 million.
Conference Call Information

Gamida Cell will host a conference call today, August 14, at 8:30 a.m. ET to discuss these financial results and company updates. To access the conference call by phone, please register here and be advised to do so at least 10 minutes prior to joining the call. A live conference call webcast can be accessed here and also in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A webcast replay will be available approximately two hours after the event for approximately 30 days.

On August 14, 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 Janssen Biotech, Inc. (Janssen’s) AKEEGA (niraparib and abiraterone acetate Dual Action Tablet), which was approved by the FDA for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC) (Press release, Foundation Medicine, AUG 14, 2023, View Source [SID1234634409]).

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Prostate cancer is one of the most common cancers in men.1 BRCA1- or BRCA2-mutated mCRPC is a particularly aggressive form of the disease,2 occurring in approximately 10-15% of diagnoses.3,4 Despite progress in developing new treatment options for this condition, BRCA1- or BRCA2-mutated mCRPC remains difficult to treat and patients often face a poor prognosis.

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

"With such a rapidly evolving therapeutic landscape in prostate cancer, high-quality companion diagnostics are important tools to support oncologists in the development of personalized treatment plans for each unique patient," said Mia Levy, MD, PhD, chief medical officer at Foundation Medicine. "This companion diagnostic specifically will help enable broader access to an important new therapy option in BRCA1/2+ mCRPC. We look forward to ongoing collaboration with Janssen to help bring more treatment options to patients facing a cancer diagnosis."

"BRCA1- or BRCA2-mutated metastatic castration-resistant prostate cancer has had a devastating impact on so many men and their families," said Shelby Moneer, VP Patient Programs and Education, ZERO Prostate Cancer. "We are so encouraged to see continued progress in advancing treatment options and diagnostics for this devastating condition."

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit www.F1CDxLabel.com.

On August 14, 2023 Biocept, Inc. (Nasdaq: BIOC) ("Biocept" or the "Company"), a leading provider of molecular diagnostic assays, products and services, reported that its CNSide cerebrospinal fluid assay was featured in two oral presentations at the 2023 SNO/ASCO CNS Cancer Conference on Saturday, August 12 (Press release, Biocept, AUG 14, 2023, View Source [SID1234634408]). The abstracts were presented by Priya U. Kumthekar, MD, a United Counsel for Neurologic Subspecialties (UCNS)-certified neuro-oncologist at Northwestern University, and David Piccioni, MD, PhD, Director of Neuro-Oncology at University of California, San Diego. The conference, sponsored by the Society for Neuro-Oncology (SNO) and the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), was held August 10-12 in San Francisco.

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"While patients with leptomeningeal metastasis (LM) have limited treatment options, select patients may benefit from targeted therapies," said Dr. Kumthekar. "Using CNSide in patients with solid tumors and LM to detect HER2 amplification could provide valuable information in treatment decision making." Dr. Kumthekar is the principal investigator at Northwestern University of Biocept’s ongoing FORESEE clinical trial, which is evaluating the performance of CNSide in monitoring the response of LM to treatment and assessing the impact of CNSide on treatment decisions made by physicians.

"Patients with leptomeningeal carcinomatosis (LMC) have limited survival and early detection by traditional CSF cytology is poor," said Dr. Piccioni. "From this study, we concluded that using CNSide was superior in detecting LMC, and its ability for molecular profiling on captured CSF tumor cells (CSF-TCs) could improve earlier detection and treatment outcomes."

A summary of the abstracts is as follows:

"The HER2 Flip: HER2 Amplification of Tumor Cells in the Cerebrospinal Fluid (CSF-TCs) of Patients with Leptomeningeal Metastasis having solid tumors; implications for treating the LM tumor with anti-HER2 therapy."

Results of this study indicated that HER2 amplification in the CSF can be detected in a substantial fraction of CSF-TCs from patients with LM having breast, upper GI and non-small cell lung cancer, and other solid tumors, and that an increased HER2 positivity in LM may imply HER2 as a driver for developing LM. The results suggest that prospective studies are needed to determine if evaluation of HER2 amplification in the CSF of patients with LM having solid tumors should be routinely considered, as it may offer viable treatment options otherwise not considered.
"Circulating tumor cell analysis from the cerebrospinal fluid informs early diagnosis, treatment and prognosis in leptomeningeal carcinomatosis (LMC)."

This study compared traditional CSF cytology and circulating tumor cell (CTC) analysis using CNSide to evaluate patients for presence of LMC. The results suggested that CTC provided superior sensitivity in detecting LMC compared with traditional cytology, and allowed for earlier detection and consequently earlier treatment. Molecular analysis of CTCs also allowed for identification of therapeutic targets specific to the CSF, which provides more information for treatment decision making and potentially improves survival versus historical controls.
The abstracts from both presentations are available on the Biocept website here.

On August 14, 2023 Pfizer Inc. (NYSE:PFE) reported the U.S. Food and Drug Administration (FDA) has granted accelerated approval to ELREXFIO (elranatamab-bcmm) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody (Press release, Pfizer, AUG 14, 2023, View Source [SID1234634407]). Approval was based on the results of the single-arm Phase 2 MagnetisMM-3 trial, and continued approval for this indication is contingent upon verification of clinical benefit in a confirmatory trial(s). ELREXFIO is a subcutaneously delivered B-cell maturation antigen (BCMA)-CD3-directed bispecific antibody (BsAb) immunotherapy that binds to BCMA on myeloma cells and CD3 on T-cells, bringing them together and activating the T-cells to kill myeloma cells.

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"ELREXFIO reflects our ongoing commitment to developing scientific breakthroughs that meaningfully improve outcomes for people with cancer. Discovered at Pfizer, we advanced this therapy from a first-in-patient trial to approval in less than five years, because we know that time is life for people living with multiple myeloma," said Angela Hwang, Chief Commercial Officer and President of Global Biopharmaceuticals Business, Pfizer. "With significant responses in a patient population with highly refractory disease, we believe ELREXFIO is poised to potentially become the new standard of care for multiple myeloma, as we plan to build upon this indication with continued development across the expansive MagnetisMM program."

The approval of ELREXFIO is based on data from response rates and duration of response. Data from cohort A (n=123) of the Phase 2 MagnetisMM-3 study (NCT04649359) showed meaningful responses among heavily pretreated RRMM patients who received ELREXFIO as their first BCMA-directed therapy. Among the patients in this study who received four or more lines of therapy prior to ELREXFIO (n=97), the overall response rate was 58%, with an estimated 82% maintaining the response for at least nine months. The median time to first response was 1.2 months. This study also established ELREXFIO as the first BCMA-directed therapy in the U.S. with once-every-other-week dosing for responding patients after 24 weeks of weekly therapy, which means less time at the clinic and potentially greater long-term treatment tolerability. The label also includes data from MagnetisMM-3 cohort B (n=64). Among the 63 patients in this cohort who received at least four prior lines of therapy, including a BCMA-directed therapy (CAR-T or antibody-drug conjugate), the overall response rate was 33% after a median follow-up of 10.2 months, with an estimated 84% maintaining the response for at least nine months.

In longer-term efficacy data for cohort A (n=123) presented at the 2023 European Hematology Association (EHA) (Free EHA Whitepaper) meeting, the objective response rate was 61%, and median duration of response, overall survival, and progression-free survival had not yet been reached at 14.7 months median follow-up. For the responding patients, the probability of maintaining a response at 15 months was 72%. Among responding patients who switched to every-other-week dosing at least six months prior to the data cut-off date (n=50), 80% maintained or improved their response after the switch, with 38% attaining a complete response or better after the switch.

"Most multiple myeloma patients will experience relapse or resistance of their disease to treatment, often facing increased symptom burden and lowering their chance of surviving longer with each attempted line of therapy," said MagnetisMM clinical trial investigator Ajay Nooka, MD, MPH, Director of the Multiple Myeloma Program at Winship Cancer Institute of Emory University. "By offering durable clinical response with an established safety profile and the convenience of subcutaneous administration, ELREXFIO provides a much-needed new option for heavily pre-treated multiple myeloma patients who are struggling with relapsed myeloma."

ELREXFIO’s label contains a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity (NT), including immune effector cell-associated neurotoxicity syndrome (ICANS), in addition to warnings and precautions for infections, neutropenia, hepatotoxicity and embryo-fetal toxicity. The most common adverse reactions to ELREXFIO (incidence ≥20%) are CRS, fatigue, injection site reaction, diarrhea, upper respiratory tract infection, musculoskeletal pain, pneumonia, decreased appetite, rash, cough, nausea, and fever (pyrexia). The most common Grade 3 to 4 laboratory abnormalities (≥20%) are decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased white blood cells, and decreased platelets. The step-up dose regimen (12/32/76 mg), combined with acetaminophen, dexamethasone, and diphenhydramine pre-treatment, is intended to reduce the incidence and severity of CRS. As a precaution, patients should be hospitalized for 48 hours following the first step-up dose and for 24 hours following the second step-up dose. No hospitalization is required for the third step-up dose. Given the risk of CRS and NT, including ICANS, ELREXFIO is available only through a restricted program called the ELREXFIO Risk Evaluation and Mitigation Strategy (REMS). A confirmatory trial (MagnetisMM-5) in the double-class exposed relapsed or refractory population involving 854 patients was initiated in 2022 to gather additional safety and efficacy data. Data will be shared as available.

"Accessibility is key to unlocking the potential impact of new treatment options. Unfortunately, novel therapies for triple-class-exposed multiple myeloma can be out of reach for medically underserved populations," said Jenny Ahlstrom, Founder and Chief Executive Officer of the HealthTree Foundation for Multiple Myeloma. "With the approval of ELREXFIO, patients have a new off-the-shelf treatment option that can be delivered on an ongoing basis in community clinics, where the majority of patients with multiple myeloma receive their care."

For patients who are prescribed ELREXFIO, Pfizer offers the support of Patient Access Navigators, through our Pfizer Oncology Together program, who provide personalized services for all aspects of treatment, including financial assistance resources, treatment support, and resources to navigate potential insurance and coverage issues.

ELREXFIO received Breakthrough Therapy Designation and Orphan Drug Designations and was approved under the FDA’s Accelerated Approval Program, which is designed to shorten the time of FDA review for drugs that treat serious conditions and fill an unmet medical need. The FDA review was also conducted under Project Orbis, a framework for the concurrent submission and review of oncology drugs among international partners to potentially expedite approvals. Currently, five countries (Switzerland, Brazil, Canada, Australia, and Singapore) are participating. A new drug application for ELREXFIO is being evaluated by the Japanese Ministry of Health, Labour and Welfare. Additionally, a marketing authorization application for ELREXFIO is currently being evaluated under the PRIME scheme by the European Medicines Agency (EMA).

The extensive MagnetisMM clinical development program is investigating ELREXFIO’s use across the entire spectrum of myeloma progression, from newly diagnosed multiple myeloma to RRMM. Ongoing registrational-intent trials are exploring ELREXFIO both as monotherapy and in combination with standard or novel therapies. These include the Phase 3 MagnetisMM-5 trial in the double-class exposed setting and MagnetisMM-7 with ELREXFIO as maintenance treatment in newly diagnosed patients after transplant.

View the full Prescribing Information, including the Boxed Warning and patient Medication Guide. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.

About Multiple Myeloma
Multiple myeloma (MM) is an aggressive and currently incurable blood cancer that affects plasma cells made in the bone marrow. Healthy plasma cells make antibodies that help the body fight infection.1 MM is the second most common type of blood cancer, with over 35,000 new cases of MM diagnosed annually in the U.S. and 176,000 globally.2,3 About half of those diagnosed with MM won’t survive beyond five years, and most will receive four or more lines of therapy due to relapse.4 While disease trajectory varies for each person, relapses are nearly inevitable.5 Real-world evidence shows that people with RRMM often become resistant to the three main classes of treatment – proteasome inhibitors, immunomodulatory agents and anti-CD38 monoclonal antibodies – after just a few rounds of therapy, and re-treating with these classes was common.6 The goal of therapy for people with RRMM is to achieve disease control with acceptable toxicity and improved quality of life.7

What is the most important information I should know about ELREXFIO?
ELREXFIO (elranatamab-bcmm) may cause side effects that are serious, life-threatening, or can lead to death, including cytokine release syndrome (CRS) and neurologic problems.

Tell your healthcare provider or get medical help right away if you develop any signs or symptoms of CRS or neurologic problems at any time during your treatment with ELREXFIO.

Cytokine release syndrome (CRS). Symptoms of CRS may include:

fever of 100.4°F (38ºC) or higher
trouble breathing
chills
dizziness or light-headedness
fast heartbeat
headache
increased liver enzymes in your blood
Due to the risk of CRS, you will receive ELREXFIO on a "step-up" dosing schedule and should be hospitalized for 48 hours after the first "step-up" dose and for 24 hours after the second "step-up" dose of ELREXFIO.

During the "step-up" dosing schedule:
For your first dose, you will receive a smaller "step-up" dose of ELREXFIO on day 1 of treatment
For your second dose, you will receive a larger "step-up" dose of ELREXFIO, which is usually given on day 4 of treatment
For your third dose, you will receive the first treatment dose of ELREXFIO, which is usually given on day 8 of treatment
If your dose of ELREXFIO is delayed for any reason, you may need to repeat the "step-up" dosing schedule
Before each dose of ELREXFIO you receive during the "step-up" dosing schedule, you will receive medications to help reduce your risk of CRS. Your healthcare provider will decide if you need to receive medications to help reduce your risk of CRS with future doses
Neurologic problems. Symptoms of neurologic problems may include:

headache
agitation, trouble staying awake, confusion or disorientation, or seeing or hearing things that are not real (hallucinations)
trouble speaking, thinking, remembering things, paying attention, or understanding things
problems walking, muscle weakness, shaking (tremors), loss of balance, or muscle spasms
numbness and tingling (feeling like "pins and needles")
burning, throbbing, or stabbing pain
changes in your handwriting
Your healthcare provider will monitor you for signs and symptoms of CRS and neurologic problems during treatment with ELREXFIO, as well as other side effects, and will treat you as needed. Your healthcare provider may temporarily stop or completely stop your treatment with ELREXFIO if you develop CRS, neurologic problems, or any other severe side effects.

If you have any questions about ELREXFIO, ask your healthcare provider.

See "What are the possible side effects of ELREXFIO?" section for more information about side effects.

ELREXFIO is available only through the ELREXFIO Risk Evaluation and Mitigation Strategy (REMS) Program due to the risk of CRS and neurologic problems. You will receive an ELREXFIO Patient Wallet Card from your healthcare provider. Carry the ELREXFIO Patient Wallet Card with you at all times and show it to all of your healthcare providers. The ELREXFIO Patient Wallet Card lists signs and symptoms of CRS and neurologic problems. Get medical help right away if you develop any of the signs and symptoms listed on the ELREXFIO Patient Wallet Card. You may need to be treated in a hospital.

Before taking ELREXFIO, tell your healthcare provider about all of your medical conditions, including if you:

have an infection
are pregnant or plan to become pregnant. ELREXFIO may harm your unborn baby. Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with ELREXFIO
You should use effective birth control (contraception) during treatment and for 4 months after your last dose of ELREXFIO
Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with ELREXFIO
are breastfeeding or plan to breastfeed. It is not known if ELREXFIO passes into your breast milk. Do not breastfeed during treatment and for 4 months after your last dose of ELREXFIO
Tell your healthcare provider about all of the medications you take, including prescription and over-the-counter medications, vitamins, and herbal supplements.

What should I avoid while receiving ELREXFIO?
Do not drive or operate heavy or potentially dangerous machinery for 48 hours after completing each of the 2 doses of ELREXFIO that are part of the "step-up" dosing schedule and the first treatment dose and at any time during treatment with ELREXFIO if you develop any new neurologic symptoms until the symptoms go away.

What are the possible side effects of ELREXFIO?
ELREXFIO may cause serious side effects, including:

Infections. Upper respiratory tract infection and pneumonia are common during treatment with ELREXFIO. ELREXFIO can cause bacterial and viral infections that are severe, life-threatening, or that may lead to death. Your healthcare provider will monitor you for symptoms of infection before and during treatment with ELREXFIO
Your healthcare provider may prescribe medications for you to help prevent infections and treat you as needed if you develop an infection during treatment with ELREXFIO
Tell your healthcare provider right away if you develop any signs or symptoms of an infection during treatment with ELREXFIO, including:
fever of 100.4°F (38ºC) or higher
chills
cough
shortness of breath
chest pain
sore throat
pain during urination
feeling weak or generally unwell
People with active infections should not start ELREXFIO
Decreased white blood cell counts. Decreased white blood cell counts are common during treatment with ELREXFIO and can also be severe. A fever can occur with low white blood cell counts and may be a sign that you have an infection. Your healthcare provider will treat you as needed
Liver problems. ELREXFIO can cause increased liver enzymes and bilirubin in your blood. These increases can happen with or without you also having CRS. Tell your healthcare provider if you develop any of the following signs or symptoms of a liver problem:
tiredness
loss of appetite
pain in your right upper stomach-area (abdomen)
dark urine
yellowing of your skin or the white part of your eyes
Your healthcare provider will check your blood and monitor you for signs and symptoms of these serious side effects before you start and during treatment with ELREXFIO and may temporarily or completely stop treatment with ELREXFIO if you develop certain side effects.

The most common side effects of ELREXFIO include:

tiredness
injection site reaction, such as redness, itching, pain, bruising, rash, swelling, and tenderness
diarrhea
muscle and bone pain decreased appetite rash
cough
nausea
fever
The most common severe abnormal lab test results with ELREXFIO include decreased white blood cells, red blood cells, and platelets.

These are not all of the possible side effects of ELREXFIO.

Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is ELREXFIO?
ELREXFIO is a prescription medication used to treat adults with multiple myeloma who have already received at least four treatment regimens (including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody) to treat their multiple myeloma, and their cancer has come back or did not respond to prior treatment.

ELREXFIO is approved based on patient response. Data are not yet available to show if ELREXFIO improves survival or symptoms.

It is not known if ELREXFIO is safe and effective in children.

Please read full Prescribing Information, including BOXED WARNING, for ELREXFIO.