On February 20, 2025 Oxcia reported that the FDA (the United States Food and Drug Administration ) has granted ODD (orphan drug designation) to OXC-101 as a potential treatment option for patients with AML (acute myeloid leukemia) (Press release, Oxcia, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=oxc-101-has-been-granted-odd-status-by-the-fda-in-aml [SID1234650416]).

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OXC-101 is Oxcia’s lead clinical candidate, a first-in class mitotic MTH1 inhibitor with a unique dual mechanism of action. In short, OXC-101, fights cancer by taking advantage of one of the Achilles heels of cancer cells – the high endogenous oxidative stress and DNA damage. In preclinical studies, OXC-101 significantly prolongs survival and reduces tumor growth in AML disease models.

Clinical Phase 1 data in advanced hematological cancer patients is reassuring. Oxcia is now performing an expansion trial, a phase 1/2 study in a subpopulation (relapsed/recurrent AML), in combination with one of the standard treatments, idarubicin. The aim is to confirm the preliminary efficacy previously shown in AML to support further clinical development in a pivotal phase 2 trial that can be the base for regulatory fast-track approval.

Oxcia AB was in the beginning of 2023 awarded a grant of 3 million SEK by Swelife and Medtech4Health for this study. To ensure the recruitment of patients with this rare AML disease in a cost- and time-efficient manner, we are now adding additional clinical sites. Rigshospitalet in Denmark will be enrolled shortly. In addition, Bulgaria and Serbia will be added during 2025.

"We are delighted with the FDA’s decision to grant orphan drug designation to OXC-101 for AML. This is a significant milestone and underscores the significant unmet need for novel medicines and the unique approach by OXC-101. We believe OXC-101 holds potential to greatly improve treatment for patients who suffer from AML", says Ulrika Warpman Berglund, Oxcia’s CEO. We are presently preparing an application for ODD also with EMA (European Medicines Agency). Early engagement with the FDA and EMA is pivotal.

In the study we are also integrating precision medicine screening to help identify responders/non-responders. Precision medicine screening is a novel way of personalizing treatment for patients and thus identifying the best treatment strategy for individuals reducing unsuccessful treatments with unnecessary suffering and lack of effect.

Briefly about AML

Acute myeloid leukemia (AML) is an aggressive blood cancer associated with infection, anemia and bleeding. It is the most common acute leukemia in adults, accounting for more than 80% of the cases. In 2021, there were 162,200 incident cases of AML worldwide, and the number is forecasted to increase to 169,000 incident cases by 2027 (Datamonitor). Even though the overall survival rate among AML patients has been increasing in the past decades, it remains poor with an overall 5-year survival rate of 20% in selected subpopulations.

AML is a very heterogenous disease, which explains why classical intensive chemotherapy, constituting of a cytarabine /anthracycline backbone, remains the standard of care (SoC). The medical need is high. Although we have seen recent approvals for biomarker led AML subsets, the majority of patients are still in need of well-tolerated and effective novel therapies.

Briefly about Orphan Drug Designation

The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent a life-threatening rare disease or condition that affects fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

On February 20, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported financial results for the quarter and full year ended December 31, 2024 (Press release, Guardant Health, FEB 20, 2025, View Source [SID1234650415]).

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Fourth Quarter 2024 Financial Highlights

For the three-month period ended December 31, 2024, as compared to the same period of 2023:
•Total revenue of $201.8 million, an increase of 30%
•Reported approximately 57,300 oncology clinical tests (excluding Shield) and approximately 11,050 biopharma tests, an increase of 24% and 16%, respectively
•Reported approximately 6,400 Shield screening tests, with revenue of $4.1 million

Full Year 2024 Financial Highlights

For the twelve-month period ended December 31, 2024, as compared to the same period of 2023:
•Total revenue of $739.0 million, an increase of 31%
•Reported approximately 206,700 oncology clinical tests (excluding Shield) and approximately 40,500 biopharma tests, an increase of 20% and 35%, respectively
•Improved full year 2024 free cash flow burn to $275 million, compared to $345 million for the full year 2023

Recent Operating Highlights

•Received Medicare coverage for Guardant Reveal on Smart Liquid Biopsy platform for colorectal cancer surveillance
•Shield selected by NIH for inclusion in the Vanguard multi cancer detection study based on strong feasibility data in predicting presence of ten cancer types and cancer site of origin
•Shield selected for a government funded population scale screening program in Abu Dhabi and surrounding regions
•Entered into a collaboration with ConcertAI to create a differentiated data-as-a-service platform that integrates comprehensive EMR records with both genomic and epigenomic tumor profiling data to accelerate cancer therapy research and development
•Entered into a strategic collaboration with Boehringer Ingelheim to develop a companion diagnostic for zongertinib to detect specific mutations in non-small cell lung cancer
•Completed a convertible debt exchange to extend the maturity on $600 million of outstanding debt
"We fired on all cylinders throughout 2024, delivering remarkable revenue growth and upgrading our products to our smart liquid biopsy platform," said Helmy Eltoukhy, co-founder and co-CEO. "Last month, we were pleased to receive Medicare reimbursement for Guardant Reveal in the CRC surveillance setting, which was an important milestone for our MRD business. We are entering 2025 with a strong oncology product portfolio and anticipate volume acceleration across all our products this year."

"We made strong progress with Shield during Q4, our first full quarter since commercial launch," said AmirAli Talasaz, co-founder and co-CEO. "We screened thousands of patients with our simple blood test and are looking forward to scaling our impact quickly. Beyond CRC, the selection of Shield for the NIH Vanguard study underscores the strength of our platform and establishes our leadership in the field of multi cancer detection. With a robust pipeline of catalysts in front of us, we are excited to execute on the opportunities ahead in 2025."

Fourth Quarter 2024 Financial Results
Revenue was $201.8 million for the fourth quarter of 2024, a 30% increase from $155.1 million for the corresponding prior year period. Precision oncology revenue grew 30%, to $184.6 million for the fourth quarter of 2024, from $142.2 million for the corresponding prior year period, driven by an increase in the volume of clinical tests (i.e., Guardant360, TissueNext, Response, and Reveal tests) and biopharma tests, which grew 24% and 16%, respectively, over the prior year period. The increase in precision oncology revenue was also attributable to an increase in reimbursement for our tests, due to an increase in Medicare reimbursement for our Guardant360 LDT test to $5,000, effective January 1, 2024; and an increase in both Medicare Advantage and commercial payer reimbursement. Development services and other revenue was $17.2 million for the fourth quarter of 2024, compared to $12.9 million for the corresponding prior year period. Other revenue includes $4.1 million derived from approximately 6,400 Shield screening tests reported in the fourth quarter of 2024.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $124.2 million for the fourth quarter of 2024, an increase of $31.7 million from $92.5 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 62%, as compared to 60% for the corresponding prior year period. Precision oncology gross margin was 62% in the fourth quarter of 2024, as compared to 60% in the prior year period. Development services and other gross margin was 53% in the fourth quarter of 2024, as compared to 60% in the prior year period.

Non-GAAP gross profit was $126.4 million for the fourth quarter of 2024, an increase of $31.8 million, from $94.5 million for the corresponding prior year period. Non-GAAP gross margin was 63% for the fourth quarter of 2024, as compared to 61% for the corresponding prior year period.

Non-GAAP gross profit excluding screening was $126.3 million for the fourth quarter of 2024, an increase of $29.0 million, from $97.3 million for the corresponding prior year period. Non-GAAP gross margin excluding screening was 64% for the fourth quarter of 2024, as compared to 63% for the corresponding prior year period.
Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $250.2 million for the fourth quarter of 2024, as compared to $206.6 million for the corresponding prior year period. Other operating expense was $83.4 million for the fourth quarter of 2023, related to a non-recurring legal accrual. No such other operating expense was recorded for the same period of 2024. Non-GAAP operating expenses were $214.7 million for the fourth quarter of 2024, as compared to $183.1 million for the corresponding prior year period.
Net loss was $111.0 million for the fourth quarter of 2024, as compared to $187.0 million for the corresponding prior year period. Net loss per share was $0.90 for the fourth quarter of 2024, as compared to $1.58 for the corresponding prior year period. The year-over-year improvement in net loss was primarily due to the non-recurring $83.4 million legal accrual recorded in 2023.
Non-GAAP net loss was $77.3 million for the fourth quarter of 2024, as compared to $75.9 million for the corresponding prior year period. Non-GAAP net loss per share was $0.62 for the fourth quarter of 2024, as compared to $0.64 for the corresponding prior year period.
Adjusted EBITDA loss was $78.4 million for the fourth quarter of 2024, as compared to a $78.4 million loss for the corresponding prior year period.
Free cash flow for the fourth quarter of 2024 was $(83.4) million, as compared to $(82.8) million for the corresponding prior year period.

Full Year 2024 Financial Results

Revenue was $739.0 million for 2024, a 31% increase from $563.9 million for the corresponding prior year period. Precision oncology revenue grew 34%, to $687.9 million for 2024, from $514.2 million for the corresponding prior year period, driven by an increase in the volume of clinical tests (i.e., Guardant360, TissueNext, Response, and Reveal tests) and biopharma tests, which grew 20% and 35%, respectively, over the prior year period. The increase in precision oncology revenue was also attributable to an increase in reimbursement for our tests, due to an increase in Medicare reimbursement for our Guardant360 LDT test to $5,000, effective January 1, 2024; an increase in both Medicare Advantage and commercial payer reimbursement; and an approximately $22 million non-recurring, out of period revenue upside for 2024 resulting from better than expected cash collections from prior years. Development services and other revenue was $51.1 million for 2024, compared to $49.7 million for the corresponding prior year period. Other revenue includes $5.1 million derived from Shield screening tests reported in 2024 following the FDA approval.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $449.2 million for 2024, an increase of $112.3 million from $336.9 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 61%, as compared to 60% for the corresponding prior year period. Precision oncology gross margin was 62% in the year of 2024, as compared to 60% in the prior year period. Development services and other gross margin was 43% in the year of 2024, as compared to 57% in the prior year period.

Non-GAAP gross profit was $459.6 million for 2024, an increase of $114.7 million, from $344.9 million for the corresponding prior year period. Non-GAAP gross margin was 62% for 2024, as compared to 61% for the corresponding prior year period.
Non-GAAP gross profit excluding screening was $466.9 million for 2024, an increase of $111.2 million, from $355.7 million for the corresponding prior year period. Non-GAAP gross margin excluding screening was 64% for 2024, as compared to 63% for the corresponding prior year period.
Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $892.8 million for 2024, as compared to $818.2 million for the corresponding prior year period. Other operating expense was $83.4 million for 2023, related to the non-recurring legal accrual discussed above. No such other operating expense was recorded for 2024. Non-GAAP operating expenses were $757.3 million for 2024, as compared to $729.2 million for the corresponding prior year period.
Net loss was $436.4 million for 2024, as compared to $479.4 million for the corresponding prior year period. Net loss per share was $3.56 for 2024, as compared to $4.28 for the corresponding prior year period. The year-over-year improvement in net loss was primarily due to the non-recurring $83.4 million legal accrual recorded in 2023, $37.7 million year-over-year improvement in loss from operations, excluding the legal accrual impact, $29.1 million of impairment recorded for our non-marketable equity security investments and other related assets in 2023 and $18.3 million increase in interest income, partially offset by $124.1 million negative change in fair value adjustments recorded for our marketable equity security investment in Lunit, Inc.
Non-GAAP net loss was $247.2 million for 2024, as compared to $352.3 million for the corresponding prior year period. Non-GAAP net loss per share was $2.01 for 2024, as compared to $3.15 for the corresponding prior year period.
Adjusted EBITDA loss was $257.5 million for 2024, as compared to a $344.2 million loss for the corresponding prior year period.
Free cash flow for 2024 was $(274.9) million, as compared to $(345.5) million for the corresponding prior year period.
Cash, cash equivalents, restricted cash and marketable debt securities were $944.2 million as of December 31, 2024. In addition, the aggregate principal amount of the 2027 Convertible Senior Notes (the "2027 Notes") was $1.15 billion as of December 31, 2024.
In February 2025, Guardant Health entered into privately negotiated exchange agreements with certain holders of the outstanding 0% 2027 Notes, pursuant to which Guardant Health issued $600.0 million aggregate principal amount of 1.25% Convertible Senior Notes due 2031 (the "New Notes") in exchange for the retirement of $659.3 million aggregate principal amount of the 2027 Notes (the "Transaction"). Total transaction costs related to the issuance of the New Notes were approximately $12.4 million. In connection with the Transaction, Guardant Health repurchased $45.0 million of shares of its common stock from certain participants in the Transaction through a financial intermediary at a price of $46.09 per share, which was the last reported sale price of its common stock on February 6, 2025 (the "Stock Repurchase"). As of December 31, 2024, pro forma for the Transaction and the Stock Repurchase, cash, cash equivalents, restricted cash and marketable debt securities would have been $886.8 million, and the aggregate principal amount of the 2027 Notes would have been $490.7 million, and the aggregate principal amount of the New Notes would have been $600.0 million, respectively.
2025 Guidance
Guardant Health expects full year 2025 revenue to be in the range of $850 to $860 million, representing growth of 15% to 16% compared to full year 2024. Excluding non-recurring, out of period revenue recorded in 2024, this range implies total revenue growth of 19% to 20% in 2025.
Within this revenue range,
•Oncology revenue is expected to grow approximately 15% year over year in 2025. Excluding non-recurring, out of period revenue recorded in 2024, this range implies oncology revenue growth of approximately 20%. Oncology clinical volume is expected to accelerate to approximately 25% growth in 2025 compared to 20% growth in 2024.
•Screening revenue is expected to be in the range of $25 to $30 million, driven by 45,000 to 50,000 Shield test volume.
•Biopharma & data revenue growth is expected to be in the low double-digit range.

Guardant Health expects full year 2025 non-GAAP gross margin to be in the range of 62% to 63%, compared to 62% in 2024. Guardant Health expects total non-GAAP operating expenses to be in the range of $815 to $825 million, representing an 8 to 9% increase compared to 2024, due to the commercial ramp to support Shield while both R&D and G&A are expected to be approximately flat year over year. Guardant Health expects free cash flow burn to be in the range of $225 to $235 million in 2025, an improvement compared to $275 million for the full year 2024. This includes approximately $200 million of screening net cash burn. Guardant Health expects the remainder of the business excluding screening to reach free cash flow breakeven in the fourth quarter of 2025.

Webcast Information
Guardant Health will host a conference call to discuss the fourth quarter and full year 2024 financial results after market close on Thursday, February 20, 2025 at 1:30 pm Pacific Time / 4:30 pm Eastern Time. A webcast of the conference call can be accessed at View Source The webcast will be archived and available for replay for at least 90 days after the event.

On February 20, 2025 Genmab A/S (Nasdaq: GMAB) reported that the Japan Ministry of Health, Labour and Welfare has approved EPKINLY (epcoritamab) for the treatment of patients with relapsed or refractory (R/R) follicular lymphoma (FL; Grades 1 to 3A) who have received two or more prior lines of therapy (Press release, Genmab, FEB 20, 2025, View Source [SID1234650414]). With this additional indication, EPKINLY is now the first and only T-cell engaging bispecific antibody administered subcutaneously to be approved in Japan to treat both R/R FL and R/R large B-cell lymphomas, including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma and primary mediastinal large B-cell lymphoma, after two or more prior lines of therapy.

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FL is typically an indolent (or slow growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL, accounting for 20-30 percent of all cases.i There are approximately 19,000 patients currently living with FL in Japan.ii FL is considered incurable with current standard of care therapies.iii Patients often relapse and, with each relapse, the remission and time to next treatment is shorter.iv Over time, transformation to DLBCL, an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25 percent of FL patients.v

"In the treatment of follicular lymphoma, where options become limited with each relapse, there remains a high unmet need for third-line and subsequent therapies in the absence of a clear standard of care," said Dr. Koji Izutsu, Head of the Department of Hematology, National Cancer Center Hospital, who served as the principal investigator of the Japanese Phase 1/2 clinical trial (EPCORE NHL-3 trial). "The responses and tolerability demonstrated in this trial support the potential of epcoritamab to become an important option in future treatment strategies for relapsed/refractory follicular lymphoma."

The approval is based on results from the global Phase 1/2 EPCORE NHL-1 and the Japanese Phase 1/2 EPCORE NHL-3 clinical trials, which were open-label, multicenter studies to evaluate the safety and efficacy of EPKINLY as a monotherapy in patients with R/R mature B-cell non-Hodgkin’s lymphoma, including FL. In the Japanese trial, a 2-step step-up dosing (SUD) regimen was used. In the global trial, two different dose escalation methods were used – 2-step and 3-step SUD regimens – to mitigate a common adverse reaction from T-cell engaging cancer treatments known as cytokine release syndrome (CRS).

EPCORE NHL-1 Global Clinical Trial Results
Among the 128 evaluable patients with R/R FL in the EPCORE NHL-1 trial, the overall response rate (ORR) and the complete response (CR) rate were 82 percent (95 percent CI: 74.3-88.3) and 62.5 percent, respectively (data cut-off: April 21, 2023). Ninety-one patients were evaluable for a minimal residual disease (MRD) analysis, with 67 percent of patients achieving MRD negativity. Additionally, more than half of patients who responded to treatment in the study remained responsive to treatment at the time of data analysis (i.e., at a median follow-up of 14.8 months, median duration of response (DoR) was not reached).

Among the patients who received EPKINLY with the 2-step SUD regimen (n=128), adverse events were observed in 119 patients (93 percent). The most common treatment-emergent adverse events (TEAEs) (≥20 percent) included CRS (66.4 percent) and injection site reactions (36.7 percent).

As part of a separate dose-optimization cohort in the trial, a 3-step SUD regimen was evaluated in 86 patients with FL (Grades 1 to 3A). Adverse events were observed in 78 patients (90.7 percent). The most common TEAEs included CRS (48.8 percent) and injection site reactions (26.7 percent).

EPCORE NHL-3 Japanese Clinical Trial Results
Among the 21 evaluable patients with R/R FL in the Japanese trial, with a median follow up of 21.2 months, the ORR and the CR rate were 95.2 percent (95 percent CI: 76.2-99.9) and 76.2 percent, respectively. Additionally, 88.9 percent of patients achieved MRD negativity (n=18).

Among patients who received EPKINLY with the 2-step SUD regimen, the most common TEAEs included CRS (90.5 percent), injection site reactions (71.4 percent), rash (28.6 percent), neutropenia (28.6 percent), increased alanine aminotransferase (23.8 percent) and increased aspartate aminotransferase (23.8 percent).

"Patients living with relapsed or refractory follicular lymphoma in Japan deserve options, and we are proud that EPKINLY may help treat patients as their cancer returns or stops responding to other therapies," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "Over the last year, EPKINLY has been approved in the U.S., the European Union (as TEPKINLY) and Japan. With a dual indication in relapsed or refractory follicular lymphoma and diffuse large B-cell lymphoma after two or more prior therapies, we are committed to making epcoritamab available to patients in need and continuing its broad development as a potential core therapy across B-cell malignancies."

About the EPCORE NHL-1 Trial
EPCORE NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab that consists of three parts: a dose escalation part; an expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed or refractory B-cell non-Hodgkin’s lymphoma (B-NHL), including FL. In the expansion part, additional patients were enrolled to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed/refractory B-NHLs who have limited therapeutic options. The expansion part generated pivotal data from patients with FL and DLBCL. The optimization part evaluated additional CRS mitigation strategies during cycle 1. The primary endpoint of the expansion part was overall response rate (ORR) as assessed by an Independent Review Committee. Secondary efficacy endpoints included duration of response (DoR), complete response (CR) rate, duration of complete response (DoCR), progression-free survival (PFS), and time to response as determined by the Lugano criteria. Overall survival (OS), time to next therapy, and rate of minimal residual disease (MRD) negativity were also evaluated as secondary efficacy endpoints. The primary endpoint of the optimization part was the rate of ≥ Grade 2 CRS events and all grade CRS events from first dose of epcoritamab through 7 days following administration of the second full dose of epcoritamab.

About the EPCORE NHL-3 Trial
EPCORE NHL-3 is an open-label, multi-center safety and efficacy trial of epcoritamab that consists of a Phase 1 first-in-human dose escalation part and a Phase 2 expansion part. The Phase 2 expansion part evaluated subcutaneous administration of epcoritamab in Japanese patients with relapsed, progressive, or refractory mature B-cell NHL, including FL. The primary endpoint of the expansion part was ORR as assessed by IRC, and secondary efficacy endpoints included DOR, CR rate, DoCR, PFS, and time to response based on the Lugano criteria.

About EPKINLY (epcoritamab)
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.vi

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with R2 compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

On February 20, 2025 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported financial results for the fourth quarter and full year ended December 31, 2024 (Press release, Dynavax Technologies, FEB 20, 2025, View Source [SID1234650413]).

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"In 2024, we successfully executed on our strategic growth initiatives, achieving record HEPLISAV-B product revenue, advancing our pipeline programs, and maintaining a disciplined approach to capital allocation," said Ryan Spencer, Chief Executive Officer of Dynavax. "Looking ahead to 2025, we plan to drive significant top-line growth as we continue to establish HEPLISAV-B as the market share leader in the expanding hepatitis B vaccine market in the U.S. We also expect to advance our pipeline programs to key milestones this year, including providing top-line results from our shingles vaccine Phase 1/2 program in the third quarter, as well as completing our $200 million share capital return and pursuing external opportunities to generate sustainable long-term value for our shareholders and other stakeholders."

BUSINESS UPDATES
HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]
HEPLISAV-B vaccine is the first and only adult hepatitis B vaccine approved in the U.S., the European Union and the United Kingdom that enables series completion with only two doses in one month. Hepatitis B vaccination is universally recommended for adults aged 19-59 in the U.S.

HEPLISAV-B achieved record net product revenue of $268.4 million for the full year 2024, an increase of 26% compared to $213.3 million for the full year 2023.
HEPLISAV-B achieved quarterly net product revenue of $71.1 million for the fourth quarter of 2024, an increase of 39% compared to $51.1 million for the fourth quarter of 2023.
HEPLISAV-B total estimated market share in the U.S. increased to approximately 44% at the end of 2024, compared to approximately 42% at the end of 2023.
Dynavax continues to expect the hepatitis B adult vaccine market in the U.S. to expand to a peak of over $900 million in annual sales by 2030, with HEPLISAV-B expected to achieve at least 60% total market share. Additionally, Dynavax believes the HEPLISAV-B U.S. market opportunity will remain substantial beyond 2030 due to the ongoing penetration of the unvaccinated eligible adult population, observed revaccination practices by healthcare providers, and continued gains in market share.
Clinical Pipeline
Dynavax is advancing a pipeline of differentiated product candidates that leverage its CpG 1018 adjuvant, which has demonstrated its ability to enhance the immune response with a favorable tolerability profile in a wide range of clinical trials and real-world commercial use.

Shingles Vaccine Program:
Z-1018 is an investigational vaccine candidate being developed for the prevention of shingles in adults aged 50 years and older.

Dynavax is currently conducting a randomized, active-controlled, dose escalation, multicenter Phase 1/2 trial to evaluate the safety, tolerability, and immunogenicity of Z-1018 compared to Shingrix in 441 healthy adults aged 50 to 69.
In the fourth quarter of 2024, the Company completed enrollment in the trial, and Dynavax anticipates reporting top-line immunogenicity and safety data in the third quarter of 2025.
Plague Vaccine Program:
Dynavax is developing a plague (rF1V) vaccine candidate adjuvanted with CpG 1018 in collaboration with, and fully funded by, the U.S. Department of Defense (DoD).

Based on the results from a randomized, active-controlled Phase 2 clinical trial of the plague vaccine adjuvanted with CpG 1018, Dynavax and the DoD executed a new agreement for approximately $30 million through the first half of 2027 to support additional clinical and manufacturing activities, including a Phase 2 clinical trial expected to initiate in the third quarter of 2025.
HEPLISAV-B for Adults on Hemodialysis:
Dynavax is developing a four-dose HEPLISAV-B vaccine regimen for adults on hemodialysis.

In the fourth quarter of 2024, Dynavax received feedback from the FDA regarding the potential to conduct an observational retrospective cohort study to support its sBLA filing for adults on hemodialysis.
FOURTH QUARTER 2024 FINANCIAL HIGHLIGHTS

Total revenues were $72.0 million for the fourth quarter of 2024, a 30% increase compared to $55.6 million for the fourth quarter of 2023.
HEPLISAV-B net product revenue was $71.1 million for the fourth quarter of 2024, a 39% increase compared to $51.1 million for the fourth quarter of 2023.
Cost of sales – product for HEPLISAV-B were $13.4 million for the fourth quarter of 2024, compared to $8.7 million for the fourth quarter of 2023.
Research and development expenses (R&D) were $18.7 million for the fourth quarter of 2024, compared to $14.1 million for the fourth quarter of 2023.
Selling, general, and administrative expenses (SG&A) were $41.6 million for the fourth quarter of 2024, compared to $41.3 million for the fourth quarter of 2023.
GAAP net income was $7.1 million, or $0.06 per share (basic) and $0.05 per share (diluted) for the fourth quarter of 2024, compared to GAAP net income of $0.2 million, or $0.00 per share (basic and diluted) for the fourth quarter of 2023.
Adjusted EBITDA* (excluding stock-based compensation) was $13.4 million for the fourth quarter of 2024, compared to $4.1 million for the fourth quarter of 2023, representing a 225% increase year-over-year.
Cash, cash equivalents and marketable securities were $713.8 million as of December 31, 2024, compared to $742.3 million as of December 31, 2023.
Share Repurchase Program: In November 2024, Dynavax announced a $200 million share repurchase program authorized by its Board of Directors, including $100 million repurchased through an Accelerated Share Repurchase program, which was completed in the first quarter of 2025. The Company expects to complete the remainder of the $200 million share repurchase program by the end of 2025.
FULL YEAR 2024 FINANCIAL HIGHLIGHTS

Total revenues were $277.2 million for the full year 2024, a 19% increase compared to $232.3 million for the full year 2023.
HEPLISAV-B net product revenue was $268.4 million for the full year 2024, a 26% increase compared to $213.3 million for the full year 2023.
Cost of sales – product for HEPLISAV-B were $49.4 million for the full year 2024, compared to $50.2 million for the full year 2023.
Research and development expenses (R&D) were $61.6 million for the full year 2024, compared to $54.9 million for the for the full year 2023.
Selling, general, and administrative expenses (SG&A) were $170.4 million for the full year 2024, compared to $152.9 million for the for the full year 2023.
GAAP net income was $27.3 million, or $0.21 per share (basic) and $0.20 per share (diluted) for the full year 2024, compared to GAAP net loss of $6.4 million, or ($0.05) per share (basic and diluted) for the full year 2023.
Adjusted EBITDA* (excluding stock-based compensation) was $51.9 million for the full year 2024, compared to $12.1 million for the full year 2023, representing a 329% increase year-over-year.
FULL YEAR 2025 FINANCIAL GUIDANCE
Dynavax is providing its full year 2025 financial guidance, based on the Company’s current operating plan:

HEPLISAV-B net product revenue is expected in the range of $305 to $325 million.
Adjusted EBITDA* (excluding stock-based compensation) is expected to be at least $75 million.
* Non-GAAP financial measure. Please refer to the "Non-GAAP Financial Measures" section for details regarding this measure.

Conference Call and Webcast Information
Dynavax will host a conference call and live audio webcast on Thursday, February 20, 2025, at 4:30 p.m. ET/1:30 p.m. PT. The live audio webcast may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source A replay of the webcast will be available for 30 days following the live event.

To dial into the call, participants will need to register for the call using the participant call link. It is recommended that participants dial into the conference call or log into the webcast approximately 10 minutes prior to the call.

WHAT IS HEPLISAV-B?
HEPLISAV-B is a shot given to adults 18 years of age and older to help prevent infection caused by the hepatitis B virus. HEPLISAV-B is usually given in the arm muscle. HEPLISAV-B is given in 2 doses, 1 month apart, by a healthcare provider.

IMPORTANT SAFETY INFORMATION
Do not administer HEPLISAV-B to individuals with a history of severe allergic reaction (e.g., anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of HEPLISAV-B, including yeast.

Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of HEPLISAV-B.

Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to HEPLISAV-B.

Hepatitis B has a long incubation period. HEPLISAV-B may not prevent hepatitis B infection in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration.

The most common patient-reported adverse reactions reported within 7 days of vaccination were injection site pain (23%-39%), fatigue (11%-17%), and headache (8%-17%).

There are no adequate and well-controlled studies of HEPLISAV-B in pregnant individuals. Available data, primarily in individuals who received one dose of HEPLISAV-B in the 28 days prior to or during pregnancy, do not suggest an increased risk of major birth defects and miscarriage.

It is not known whether HEPLISAV-B is excreted in human milk.

Data are not available to assess the effects of HEPLISAV-B on the breastfed infant or on milk production/excretion.

Vaccination with HEPLISAV-B may not result in protection of all vaccine recipients.

Talk to your healthcare provider to determine if HEPLISAV-B is right for you.

On February 20, 2025 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), reported that company management will participate in two upcoming investment bank conferences (Press release, Crinetics Pharmaceuticals, FEB 20, 2025, View Source [SID1234650412]). The TD Cowen 45th Annual Healthcare Conference is being held in Boston, MA, and the Leerink Global Healthcare Conference is taking place in Miami, FL.

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                  Schedule Your 30 min Free Demo!

TD Cowen 45th Annual Healthcare Conference
Fireside chat on Monday, March 3, 2025 at 11:10 a.m. Eastern Time
Webcast link HERE

Leerink Global Healthcare Conference
Fireside chat on Monday, March 10, 2025 at 4:20 p.m. Eastern Time
Webcast link HERE

The live and archived webcasts will be accessible on the Events & Presentations page in the Investors section of the Crinetics’ website at www.crinetics.com/events.

If you are interested in arranging a 1×1 meeting with management, please contact your conference representative.