On March 17, 2025 Genmab A/S (Nasdaq: GMAB) reported updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5) in heavily pre-treated ovarian cancer (OC) patients regardless of FRα expression levels (Press release, Genmab, MAR 17, 2025, View Source [SID1234651181]). With a median on-study follow-up of 48 weeks, 1 out of 10 patients experienced disease progression and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). The data are from the dose expansion cohort of the multi-part study evaluating the safety and efficacy of Rina-S as a single agent in solid tumors that are known to express FRα and were presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women’s Cancer (SGO) in Seattle, Washington.

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"The antitumor activity observed in the dose expansion cohort continues to demonstrate the potential for a much-needed treatment option for patients with PROC, who have historically had poor prognosis. I am hopeful that further exploration of Rina-S will lead to advancements in the treatment landscape." said Elizabeth Lee, M.D., a medical oncologist in the gynecologic oncology program at Dana-Farber.

The B1 cohort is a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer or fallopian tube cancer). Rina-S 120 mg/m2 Q3W at a median on-study follow-up of 48 weeks showed encouraging antitumor activity; the confirmed ORR was 55.6% (95% CI: 30.8-78.5), the disease control rate (DCR) was 88.9% (95% CI: 65.3-98.6), and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). In the 18 patients evaluable for response treated with 120 mg/m2 Q3W, complete responses were observed in 4 patients (2 confirmed; 2 unconfirmed) and 8 patients experienced confirmed partial responses (44.4%). Most responses with Rina-S 120 mg/m2 were observed early (at week 6). Only one patient in the 120 mg/m2 treatment arm was not evaluable. In the Rina-S 100 mg/m2 Q3W treatment arm (N=22), at a median on study follow-up of 46 weeks, the confirmed ORR was 22.7% (95% CI: 7.8-45.4), the DCR was 86.4% (95% CI: 65.1-97.1), and the mDOR was not reached (95% CI, 16.3-NR). Partial responses were observed in 4 patients (18.2%) and 1 patient (4.5%) experienced a complete response. Rina-S 120 mg/m2 has been selected for further evaluation in the RAINFOL-01 and Phase 3 RAINFOL-02 trials for patients with platinum resistant ovarian cancer (PROC).

In this Phase 1/2 study, common treatment-emergent adverse events (TEAEs) included anemia, nausea, neutropenia, leukopenia, fatigue, thrombocytopenia, vomiting, diarrhea, alopecia, and hypokalemia. Dose reductions and treatment discontinuations were infrequent and no new safety signals were observed.

"The updated results reinforce the potential of Rina-S and further validate our development approach in advanced ovarian cancer," said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. "We are excited to keep moving forward with the ongoing Phase 3 trial, to evaluate the potential of Rina-S as a treatment option for patients facing this challenging disease."

The safety and efficacy of rinatabart sesutecan has not been established for these investigational uses.

About the RAINFOLTM -01 Trial
RAINFOL-01 (NCT05579366) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) as a single agent Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose-escalation cohorts; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; and Part D combination therapy cohorts.

Part B of the trial includes the B1 cohort, a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer). Patients were randomized 1:1 to 100 mg/m2 and 120 mg/m2 dose cohorts. Median age was 62.5 and 64.5 years in the 100 mg/m2 and 120 mg/m2 cohorts, respectively. Study participants were previously treated with a median of 3 prior lines of therapy (range 1-4). Patients received prior treatment with bevacizumab (90.9% in the 100 mg/m2 group and 90.0% in the 120 mg/m2 group respectively), PARP inhibitors (68.2%; 65%), and mirvetuximab soravtansin (18.2%; 19%). Initial results from Part B of this trial were presented during a mini-oral session at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 (ESMO) (Free ESMO Whitepaper).

About Ovarian Cancer
Ovarian cancer is a major global health issue, with over 320,000 new cases diagnosed annually worldwide.i It ranks as the eighth most common cancer and the eighth leading cause of cancer-related deaths among women globally.ii The disease is often diagnosed at an advanced stage due to its subtle and non-specific symptoms, such as abdominal bloating, pelvic pain and difficulty eating.iii Standard of care for platinum resistant ovarian cancer typically involves single agent chemotherapy (pegylated liposomal doxorubicin (PLD), topotecan, gemcitabine or paclitaxel).iv Approximately 70-90% of women with advanced-stage ovarian cancer worldwide experience a recurrence after initial treatment.v Ovarian cancer has a low five-year survival rate, which varies significantly by region, but generally hovers around 30-50%.vi,vii

About Rinatabart Sesutecan (Rina-S; GEN1184)
Rinatabart sesutecan (Rina-S; GEN1184) is a FRα-targeted, TOPO1 ADC, currently being evaluated for the potential treatment of ovarian cancer and other FRα-expressing cancers. A Phase 3 trial (RAINFOL-02, NCT06619236) evaluating Rina-S in patients with platinum resistant ovarian cancer compared to treatment of investigator’s choice is ongoing. In January 2024, the U.S. Food and Drug Administration granted Fast Track designation to Rina-S for the treatment of patients with FRα-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer.

Please visit www.clinicaltrials.gov for more information.

On March 17, 2025 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the fourth quarter and full year 2024 and provided an update on the company’s recent developments (Press release, FibroGen, MAR 17, 2025, View Source [SID1234651180]).

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"We entered 2025 optimistic about our future, highlighted by the planned initiation of the Phase 2 monotherapy trial of FG-3246, our first-in-class ADC targeting CD46 for the treatment of mCRPC, by mid-2025," said Thane Wettig, Chief Executive Officer. "Through the successful implementation of our cost reduction plan, and upon the closing of our recently announced sale of FibroGen China, we will be a leaner and more focused organization, with a stronger financial position and a cash runway that takes us into 2027, with multiple potential value-creating milestones in sight."

Recent Developments and Key Highlights of 2024:

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Announced the sale of FibroGen China to AstraZeneca for a total consideration of approximately $160 million, representing an enterprise value of $85 million plus estimated net cash held in China at closing of approximately $75 million. The transaction is expected to close by mid-2025.
o
Upon closing, FibroGen will repay its term loan to Morgan Stanley Tactical Value, further simplifying the Company’s capital structure.
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FibroGen maintains its rights to roxadustat in the U.S. and in all markets outside of China, South Korea, and those licensed to Astellas.
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Appointed David DeLucia, CFA, as Senior Vice President and Chief Financial Officer.
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Completed previously announced cost reduction program.

Upcoming Milestones:

FG-3246 (CD46 Targeting ADC) and FG-3180 (CD46 Targeting PET Imaging Agent)

•
Anticipate initiation of Phase 2 monotherapy dose optimization study of FG-3246 in mCRPC by mid-2025.
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Phase 2 trial will include a sub-study of FG-3180 to enable assessment of its diagnostic performance and the potential correlation between CD46 expression and response to FG-3246.
•
Topline results from the Phase 2 portion of the investigator-sponsored Phase 1b/2 study conducted by UCSF of FG-3246 in combination with enzalutamide in patients with mCRPC expected in 2H 2025.
o
Phase 2 portion of the study will include data on FG-3180.
Roxadustat

•
Plan to meet with FDA in 2Q 2025 to determine the potential next steps for the development of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS), an indication with significant unmet medical need, in the U.S.Financial:

•
Total revenue from continuing operations for the fourth quarter of 2024 was $3.1 million, as compared to $3.6 million for the fourth quarter of 2023.
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Total revenue from continuing operations for the full year 2024 was $29.6 million, as compared to $46.8 million for the full year 2023.
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Net loss from continuing operations for the fourth quarter of 2024 was $8.7 million, or $0.08 net loss per basic and diluted share, compared to a net loss of $62.5 million, or $0.63 net loss per basic and diluted share, one year ago.
•
Net loss from continuing operations for the full year 2024 was $153.1 million, or $1.53 net loss per basic and diluted share, compared to a net loss of $323.0 million, or $3.32 net loss per basic and diluted share, for the full year 2023.
•
At December 31, 2024, FibroGen reported $51.0 million in cash, cash equivalents and accounts receivable in the U.S. and $121.1 million in total consolidated cash, cash equivalents and accounts receivable.
•
Upon closing of the announced sale of FibroGen China, the Company expects its cash, cash equivalents and accounts receivable to be sufficient to fund operations into 2027.

Conference Call and Webcast Presentation

The FibroGen management team will host a conference call and webcast presentation to discuss the financial results and provide a business update. A live Q&A session will follow the brief presentation. Interested parties may access a live audio webcast of the conference call here. To access the call by phone, please register here, and you will be provided with dial in details. A replay of the webcast will also be available for a limited time on the Events & Presentations page on FibroGen’s website.

About FG-3246

FG-3246 (FOR46) is a potential first-in-class fully human antibody-drug conjugate (ADC), exclusively in-licensed from Fortis Therapeutics, and is being developed by FibroGen for metastatic castration-resistant prostate cancer and potentially other tumor types. FG-3246 binds to an epitope of CD46, a cell receptor target, that induces internalization upon antibody binding, is present at high levels in prostate cancer and other tumor types and demonstrates very limited expression in most normal tissues. FG-3246 is comprised of an anti-CD46 antibody, YS5, linked to the anti-mitotic agent, MMAE, which is a clinically and commercially validated ADC payload. FG-3246 has demonstrated anti-tumor activity in both preclinical and clinical studies.

FG-3246 is currently in an ongoing Phase 1b/2 study being conducted at UCSF as an investigator-sponsored trial to evaluate FG-3246 in combination with enzalutamide. An additional investigator-sponsored radiopharmaceutical marker trial using a zirconium-89 positron emission tomography (PET) tracer for CD46 that utilizes the YS5 antibody is also underway at UCSF. The initiation of the Phase 2 monotherapy dose optimization trial for FG-3246 in metastatic castration-resistant prostate cancer is anticipated by mid-2025. FG-3246 is an investigational drug and not approved for marketing by any regulatory authority.

About Roxadustat

Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority.

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa.

On March 17, 2025 The European Organisation for Research and Treatment of Cancer (EORTC) reported the launch of the EORTC-2334-BTG LUMEN-1 clinical trial, a groundbreaking study aimed at finding potential new treatment options for patients with recurrent Meningioma (Press release, EORTC, MAR 17, 2025, View Source [SID1234651179]). Meningioma is a type of brain tumour that can return after surgery and radiotherapy, leaving patients with limited treatment options.

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The LUMEN-1 trial will investigate the effectiveness of a new treatment called [177Lu]Lu-DOTATATE, which targets a specific receptor found in most meningiomas. This treatment has shown promising results in smaller studies and is now being tested in a larger, randomised trial involving 136 patients across 35 sites in 10 European countries. This is a phase II study, meaning that it is a study aiming to show the activity of this therapy in treating recurrent Meningioma.

Patients participating in the trial will be randomly assigned to receive either [177Lu]Lu-DOTATATE or the current standard of care. The primary goal of the study is to determine whether [177Lu]Lu-DOTATATE can improve progression-free survival, allowing patients to live longer without their disease worsening. Secondary goals include assessing overall survival, safety, quality of life, and neurological function.

The trial also includes a comprehensive research program to explore various aspects of the treatment, such as dosimetry, imaging, and tissue analysis. The study is expected to be completed by 2029, with the hope of guiding the way forward to potential new, effective treatment options for patients with recurrent meningioma.

On March 17, 2025 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported its financial results for the fourth quarter and full year ending December 31, 2024 (Press release, Akoya Biosciences, MAR 17, 2025, View Source [SID1234651177]).

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"Akoya navigated a challenging 2024 in the life science tools market, which was constrained by subdued capital equipment purchases, by successfully strengthening gross margins, reducing operating expenses and advancing our companion diagnostics programs throughout the year. We remain optimistic about the long-term growth outlook of Akoya’s leading spatial biology solutions," said Brian McKelligon, CEO of Akoya. "In 2024, Akoya achieved multiple milestones, including expanding our market-leading installed base to 1,330 instruments, launching our Manufacturing Center of Excellence to drive improvements in gross margins and the expansion of our content menu into new markets like neurobiology, and continued advancement of our clinical partnerships led by Acrivon and NeraCare."

Fourth Quarter 2024 Financial Highlights

Revenue was $21.3 million in the fourth quarter of 2024, compared to $26.5 million in the prior year period; a decrease of 19.4%. This topline revenue decrease was primarily due to a decline in instrument revenue.
Gross margin was 67.4% in the fourth quarter of 2024, compared to 62.7% in the prior year period. The increase in gross margin was primarily driven by operational efficiency from in-house reagent manufacturing and product mix.
Operating expenses were $20.1 million for the fourth quarter of 2024, compared to $26.1 million in the prior year period; an improvement of 22.9%. The improvement was primarily driven by further realized operating leverage and efficiencies.
Operating loss was $5.7 million for the fourth quarter of 2024, compared to an operating loss of $9.4 million in the prior year period; an improvement of 39.5%.
$35.0 million of cash, cash equivalents, and marketable securities as of December 31, 2024.
Business Highlights

Announced the pending acquisition of Akoya Biosciences by Quanterix Corporation (Nasdaq: QTRX), which, if consummated, would create the first integrated solution for ultra-sensitive detection of blood and tissue-based protein biomarkers.
Announced a strategic product roadmap, powered by the success of IO60 and upcoming launch of neurobiology panels to solidify leadership in spatial proteomics across new research verticals.
Announced an exclusive global license agreement with NeraCare for the development and commercialization of the Immunoprint test for early-stage melanoma patient treatment decisions.
Nature Methods named spatial proteomics "Method of the Year 2024" — a key milestone which reaffirms Akoya’s leadership position in the spatial proteomics field with the largest installed base and publications volume in the industry.
Financial Highlights

Full year 2024 revenue was $81.7 million, compared to $96.6 million in the prior year; a decrease of 15.5%.
Full year 2024 reported gross margin was 58.6% while non-GAAP adjusted gross margin(1) was 61.1% when excluding the write-off from discontinued legacy products in the first quarter of 2024. Both GAAP and non-GAAP gross margin were 58.3% in the prior year.
Full year 2024 operating expenses were $94.6 million while non-GAAP operating expenses(1) were $88.6 million when excluding the impairment charge for facility consolidation and restructuring associated with reductions in force completed in the first quarter of 2024 and third quarter of 2024, respectively. Both GAAP and non-GAAP operating expenses were $114.0 million in the prior year.
Full year 2024 loss from operations was $46.7 million while non-GAAP loss from operations(1) was $38.6 million excluding the items noted above. Both GAAP and non-GAAP loss from operations were $57.7 million in the prior year.
Instrument installed base of 1,330 as of December 31, 2024 (400 PhenoCyclers, 930 PhenoImagers), compared to an installed base of 1,183 in the prior year (342 PhenoCyclers, 841 PhenoImagers); an increase of 12.4%.
1,733 total publications citing Akoya’s technology as of December 31, 2024, compared to 1,160 total publications in the prior year; an increase of 49.4%.

(1) See discussion of "Non-GAAP Financial Measures" below.
In light of the pending acquisition by Quanterix Corporation, Akoya will not be hosting an earnings conference call or providing forward guidance at this time.

On March 17, 2025 EsoBiotec SA, a biotechnology company pioneering in vivo cell therapies that has demonstrated promising early clinical activity, reported it has entered into a definitive agreement to be acquired by AstraZeneca (Press release, EsoBiotec, MAR 17, 2025, View Source [SID1234651164]). The EsoBiotec Engineered NanoBody Lentiviral (ENaBL) platform empowers the immune system to attack cancers and could offer many more patients access to transformative cell therapy treatments delivered in just minutes rather than the current process which takes weeks.

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ENaBL uses highly targeted lentiviruses to deliver genetic instructions to specific immune cells, such as T cells, which programme them to recognise and destroy tumour cells for cancer treatment or autoreactive cells for potential use in immune-mediated diseases. This approach enables cell therapies to be administered through a simple IV injection and without the need for immune cell depletion.

Traditional cell therapies require cells to be removed from a patient, genetically modified outside the body, and then readministered to the patient as a medicine after immune cell depletion, typically taking weeks. By engineering immune cells directly within the patient’s body, the EsoBiotec in vivo approach has the potential to address many of the barriers associated with traditional cell therapies, reducing complexities and manufacturing timelines, thereby increasing access for patients.

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "We are excited about the acquisition of EsoBiotec and the opportunity to rapidly advance their promising in vivo platform. We believe it has the potential to transform cell therapy and will enable us to scale these innovative treatments so that many more patients around the world can access them. EsoBiotec will accelerate and expand the impact of our recent investments and marks a major step forward in realising our ambition to harness the full potential of cell therapy."

Jean-Pierre Latere, PhD, CEO, EsoBiotec, said: "We look forward to working with AstraZeneca, a global leader in drug development, to advance our shared goal of bringing transformative cost-effective cell therapies to more patients globally. By combining our expertise and resources, we can accelerate the development of our in vivo platform which has a novel delivery technology we believe will have broad therapeutic applicability."

EsoBiotec will become a wholly owned subsidiary of AstraZeneca, with operations in Belgium.

Financial considerations
AstraZeneca will acquire all outstanding equity of EsoBiotec for a total consideration of up to $1,000m, on a cash and debt free basis. This will include an initial payment of $425m on deal closing, and up to $575m in contingent consideration based on development and regulatory milestones.

The transaction is expected to close in the second quarter of 2025, subject to customary closing conditions and regulatory clearances.

Centerview Partners UK LLP is acting as exclusive financial advisor to EsoBiotec.

Cooley LLP is acting as legal advisor to EsoBiotec. Covington & Burling LLP is acting as legal advisor to AstraZeneca.