On December 15, 2025 FibroGen, Inc. (NASDAQ: FGEN) reported that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS).

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"The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response," said Thane Wettig, Chief Executive Officer of FibroGen. "Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments. Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025."

There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life. Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden.

The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. following drug approval.

About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS.

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 approved in 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.

(Press release, FibroGen, DEC 15, 2025, View Source [SID1234661421])

On December 15, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA" or the "Company"), a Phase 3 immuno-oncology company developing novel therapeutics to overcome resistance to cancer immunotherapy, reported that Kintara’s REM-001 clinical trial of ten metastatic cutaneous breast cancer patients met its primary endpoint demonstrating safety with signs of clinical efficacy following eight weeks of follow-up for such patients.

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Pursuant to the terms of the Contingent Value Rights Agreement dated as of October 18, 2024, by and between TuHURA Biosciences, Inc. and the rights agent, Equiniti Trust Company, LLC (the "CVR Agreement"), as a result of the trial and the completion of the follow-up, the milestone required for the release of an aggregate of 1,539,958 shares of TuHURA common stock to legacy Kintara Therapeutics stockholders has been achieved. The holders of CVRs are entitled to receive shares of TuHURA common stock, subject to the terms of the CVR Agreement and any applicable withholding. Pursuant to the CVR Agreement, the shares of TuHURA common stock are expected to be distributed to the CVR holders within the next ten business days.

(Press release, TuHURA Biosciences, DEC 15, 2025, View Source [SID1234661441])

On December 15, 2025 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported an approximately 80% recurrence rate reduction in the open label non-HLA-A*02 arm of FLAMINGO-01.

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A preliminary analysis of recurrence rates by two methods to estimate the reduction in recurrence rate shows an approximately 80% reduction in recurrence rate in the fully enrolled, 250 patient non-HLA-A*02 arm of FLAMINGO-01. This arm does not have a direct placebo comparator arm, thus these two methods were used.
This observation is trending similarly to the Phase IIb trial results and hazard ratio where HLA-A*02 patients were treated and where breast cancer recurrences were reduced up to 80% compared to a 20-50% reduction in recurrence rate by other approved products. See the summary of the Phase IIb results below.
The first non-HLA-A*02 patient has completed the 11 primary and booster vaccinations over the first 3 years.

In addition to announcing this first analysis of the recurrence rate data in the open label non-HLA-A*02 arm of FLAMINGO-01, the Company previously reported promising observations showing that the immune response at baseline prior to any GLSI-100 treatment, the increasing immune response during the primary immunization series, and the safety profile of non-HLA-A*02 patients is trending similarly to the HLA-A*02 arms of FLAMINGO-01 and to the Phase IIb study.

The Primary Immunization Series (PIS), which includes the first 6 GLSI-100 injections over the first 6 months and is required to reach peak protection, is followed by 5 booster injections given every 6 months to prolong the immune response, thereby providing longer-term protection.

In the 250 patient non-HLA-A*02 data set, all patients received GLSI-100, which is 5 times more treated patients and recurrence rate data than the approximately 50 patients treated in the Phase IIb trial. Since the 250 non-HLA-A*02 patients do not have a placebo arm for a direct comparison, the following two methods were used, yielding the 80% reduction in recurrence rate observation:

Method 1: The recurrence rate of these 250 treated patients after completing the PIS was compared to the expected historical recurrence rate per year reported for this population in the Katherine study who received TDM1 (Kadcyla), which is about 3.5-4% recurrences per year or higher in the initial years of the Katherine study. The majority of the treated patients in FLAMINGO-01 also received TDM1 followed by GLSI-100. The data suggests an approximately 80% reduction in recurrence rate after the GLSI-100 PIS is completed as compared to the TDM1 arm of the Katherine study at a similar timepoint.
Method 2: The recurrence rate during the first 6 months of vaccination or PIS period when the patient may not be fully protected or protected at all, which covered an exposure period of 100 patient years, was compared to the recurrence rate after the PIS is completed and after peak immunity is believed to be achieved, which covered an exposure period of 132 patient years. The exposure period (patient years) is defined as the cumulative number of years all patients are followed in a period. The data suggests that the peak immunity is lowering the recurrence rate by approximately 80% after the PIS is completed as compared to the recurrence rate during the PIS period.
For the first 6 months of vaccination or PIS, the theoretical maximum patient years would be 250 patients at 0.5 years each or 125 patient years. Thus 100 completed patient years represents 80% of the maximum potential patient years and within 3 to 6 months all vaccinations during this period may be completed.
For the period after the PIS is completed, the theoretical maximum patient years would be 250 patients at 3.5 years of follow-up each or 875 patient years. Thus 132 completed patient years represents 15% of the maximum potential patient years and represents an average of 9 months of follow-up time after the PIS for patients who have completed the PIS.
Analysis of the open label data from FLAMINGO-01 has been conducted in a manner that maintains the study blind. The open label recurrence rate, immune response, and safety data is based on the patients enrolled to date in FLAMINGO-01 and the data provided by the clinical sites so far, which is not completed or fully reviewed, and is thus preliminary. While comparing any preliminary FLAMINGO-01 data to the Phase IIb clinical trial data may be possible, these preliminary results are not a prediction of future results, and the results at the end of the study may differ.

CEO Snehal Patel commented, "We are very excited to announce in our 100th press release since going public that the first patient has completed the full primary and booster vaccination series in FLAMINGO-01. We are equally excited to see a positive trend in the recurrence rate in the non-HLA-A*02 arm of FLAMINGO-01, as assessed multiple ways, that is trending similarly to the Phase IIb trial results where HLA-A*02 patients were treated. Despite these being preliminary results that will mature and can change with time, seeing a reduction in expected recurrence rates that is trending towards a low HR of 0.2, which represents an 80% reduction in recurrence rate, is very encouraging and which is why along with the similarly promising safety and immune response data to date, we will seek to continue to treat non-HLA-A*02 patients in a placebo controlled manner in the study. We will continue to analyze the data and may provide updates at any time."

Previously Published Phase IIb Data

In the prospective, randomized, single-blinded, placebo-controlled, multi-center (16 sites led by MD Anderson Cancer Center) Phase IIb clinical trial of HLA-A*02 breast cancer patients, 46 HER2/neu 3+ over-expressor patients were treated with GLSI-100, and 50 placebo patients were treated with GM-CSF alone. After 5 years of follow-up, there was an 80% or greater reduction in cancer recurrences in the HER2/neu 3+ patients who were treated with GLSI-100, followed, and remained disease free over the first 6 months, which we believe is the time required to reach peak immunity and thus maximum efficacy and protection. The Phase IIb results can be summarized as follows:

80% or greater reduction in metastatic breast cancer recurrence rate over 5 years of follow-up with a peak immune response at 6 months and well-tolerated safety profile.
The PIS elicited a potent immune response as measured by local skin tests and immunological assays.

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US and European clinical sites from university-based hospitals and academic and cooperative networks with plans to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients are planned to be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types are planned to be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

(Press release, Greenwich LifeSciences, DEC 15, 2025, View Source [SID1234661422])

On December 15, 2025 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the U.S. Food and Drug Administration (FDA) has approved additional indications for its PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody and VENTANA HER2 Dual ISH DNA Probe Cocktail tests. These tests are now approved to aid in identifying HER2-positive metastatic breast cancer (mBC) patients who may be eligible for treatment with ENHERTU (trastuzumab deruxtecan), a specifically engineered HER2-directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialised by Daiichi Sankyo and AstraZeneca.

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Until now, Roche’s PATHWAY HER2 (4B5) test had been approved for identifying mBC patients with HER2-low and HER2-ultralow expression. With this expanded approval, Roche’s PATHWAY HER2 (4B5) test in combination with the VENTANA HER2 Dual ISH DNA Probe Cocktail can now be used to identify patients across the full spectrum of HER2 expression for potential eligibility for ENHERTU. This approval reflects the vital role of advanced diagnostics in guiding precise treatment decisions to address the diverse needs of mBC patients.

"Metastatic breast cancer remains a significant challenge," said Laura Apitz, Head of Pathology Lab at Roche Diagnostics. "Diagnostic advances like these bring much-needed hope for patients. With this approval, our breast diagnostic portfolio can further guide therapy decisions for clinicians, enabling a more personalised approach."

Advancing Science in HER2 Breast Cancer
Breast cancer now represents one in four of all cancers diagnosed in women worldwide.1 Despite advances in care, metastatic breast cancer poses a growing challenge, especially in younger populations,2,3 where cases among women aged 25 to 39 increased by 32% between 2009 and 2015.2,3

However, treatment for metastatic breast cancer is evolving alongside a deeper understanding of HER2 biology, which now shows that HER2 expression exists on a continuous spectrum rather than as distinct "positive" or "negative" categories.4

These diagnostics strengthen Roche’s broader breast cancer portfolio, which offers solutions that cover the full spectrum of breast cancer management, including important predictive assays. Roche remains committed to supporting patients with advanced diagnostic tools that help clinicians improve outcomes and deliver more personalized care at every stage of the disease.

About PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody
The PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody delivers timely, clear and reliable results, driving diagnostic certainty and enabling therapeutic decisions that can lead to better outcomes for patients. Previously indicated as an aid to identify certain breast cancer patients eligible for HER2-targeted treatment with Herceptin, KADCYLA, or ENHERTU,5 the test is used in combination with the fully automated BenchMark ULTRA and BenchMark ULTRA PLUS staining platforms.

The assay standardizes all IHC processes from baking through staining, and reduces the possibility of human error.5 It also minimizes inherent variability resulting from individual reagent dilution and other processes found in manual and semi-automated IHC methods. The HER2 (4B5) clone achieves consistently high proficiency assessment scores compared to other clones6 and demonstrates high concordance with HER2 FISH,7.8 empowering laboratories to employ the most widely adopted and reliable HER2 IHC primary antibody.

About the VENTANA HER2 Dual ISH DNA Probe Cocktail assay
The VENTANA HER2 Dual ISH DNA Probe Cocktail assay is a fully automated, ready-to-use brightfield solution for determining HER2 gene status. VENTANA HER2 Dual ISH helps identify breast cancer patients eligible for personalized treatment with HER2-targeted therapies. The VENTANA HER2 Dual ISH DNA Probe Cocktail assay is optimised for use with the VENTANA Silver ISH DNP Detection Kit and the VENTANA Red ISH DIG Detection Kit on the fully-automated BenchMark ULTRA and BenchMark ULTRA PLUS staining platforms.9

The VENTANA HER2 Dual ISH DNA Probe Cocktail assay is an enhanced version of the previous-generation test. New oligonucleotide probes and highly sensitive detection kits provide clear results to pathology labs more quickly, allowing clinicians to make treatment decisions earlier.

(Press release, Hoffmann-La Roche, DEC 15, 2025, View Source [SID1234661442])

On December 15, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, reported positive topline results from the global pivotal Phase 3 RINGSIDE trial of varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), in patients with progressing desmoid tumors.

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The trial met its primary endpoint of improving progression-free survival, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death (hazard ratio (HR) = 0.16, 95% CI: 0.071, 0.375; p<0.0001). The confirmed objective response rate (ORR) based on RECIST v1.1 was 56% with varegacestat vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review. In an exploratory analysis, varegacestat demonstrated a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review. In addition, the trial met all key secondary endpoints, with varegacestat achieving statistically significant improvements vs. placebo in landmark tumor volume reduction and worst pain intensity.

Varegacestat was generally well tolerated, with a manageable safety profile consistent with the GSI class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most events were grade 1 or 2.

Based on these data, Immunome plans to submit a New Drug Application to the U.S. Food and Drug Administration in Q2 2026.

"RINGSIDE is the largest and most comprehensive clinical trial conducted to date in patients with desmoid tumors, and the topline results represent the highest objective response rate observed in a randomized clinical trial in this patient population," said Immunome’s CEO Clay Siegall, Ph.D. "These findings demonstrate the potential of varegacestat to offer best-in-class results in a convenient, once-daily, oral medicine that may help patients reclaim their lives."

"Desmoid tumors can have a devastating physical and emotional impact on patients given their unpredictable nature and the limitations of current treatment options," said Mrinal M. Gounder, M.D., sarcoma medical oncologist and drug development specialist at Memorial Sloan Kettering Cancer Center, and RINGSIDE primary investigator. "The progression-free survival benefit, high response rate and reduction in tumor volume with varegacestat in the RINGSIDE trial are striking. These findings elevate the role of GSIs and confirm varegacestat could become standard of care in the treatment of desmoid tumors."

Dr. Siegall added, "The RINGSIDE results represent a major milestone for Immunome as we advance our emerging pipeline of targeted oncology therapies that have exceptional potential to meaningfully improve the lives of patients."

Immunome plans to share additional data from the RINGSIDE trial at an upcoming major medical conference.

Webcast, Presentation Slides and Conference Call Information

Immunome will host a webcast and conference call on Monday, December 15, 2025, at 8:30 a.m. ET / 5:30 a.m. PT to discuss the Phase 3 RINGSIDE trial topline results. A live webcast, which will include presentation slides, can be accessed using this link or by visiting the Events and Presentations section of the Immunome website at View Source The conference call can be accessed by clicking on the call link and completing the online registration form, which will enable the selection of a dial-in number or callback from the system. A live question-and-answer session will follow the prepared remarks. Participants wishing to ask a question must do so via the conference call; the webcast will be listen-only. After the live webcast, the event will remain archived on the Immunome website for 90 days.

About the RINGSIDE Trial

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled secondary endpoints were ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity as determined using a patient reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors (also known as aggressive fibromatosis or desmoid-type fibromatosis) are aggressive non-metastatic soft tissue tumors that are prone to recurrence. Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients. Those affected face debilitating pain, deformity and, in some cases, life-threatening organ damage. The chronic pain and physical limitations associated with desmoid tumors lead to a high clinical burden and impaired quality of life. Although desmoid tumors are not considered cancerous, they often require systemic treatment to prevent permanent disability and alleviate disease burden.

About Varegacestat

Varegacestat (formerly AL102) is an investigational, oral, once-daily gamma secretase inhibitor. In December 2025, Immunome reported positive topline results for the Phase 3 RINGSIDE trial of varegacestat in adults with progressing desmoid tumors. Immunome plans to submit a New Drug Application for varegacestat to the U.S. Food and Drug Administration in Q2 2026.

(Press release, Immunome, DEC 15, 2025, View Source [SID1234661423])