On April 10, 2026 Eisai Inc. reported the presentation of findings from a new survey, which evaluated the experiences and perspectives of 119 U.S.-based patients who have been diagnosed with endometrial cancer (EC) and who received at least one line of chemotherapy.† Conducted online in collaboration with the Endometrial Cancer Action Network for African-Americans (ECANA), Facing Our Risk of Cancer Empowered (FORCE), the Foundation for Women’s Cancer (FWC) and The Harris Poll, the survey aimed to quantify patients’ perspectives on topics including the impact of treatment on daily life, preferences regarding treatment options, the role of shared decision-making, and more.

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Endometrial cancer, the most common type of uterine cancer, is the sixth most common cancer in women across the globe, with more than 60,000 new diagnoses in the U.S. each year. In this survey, patients reported that EC and its treatment had negatively impacted not just their physical health, but also social, mental, sexual and financial wellbeing.

Chemotherapy – which is a standard component of the first systemic treatment patients receive ("first-line") – was reported as taking an emotional and physical toll on patients, manifesting in deeply personal ways. While it remains an important step in the clinical paradigm for EC treatment, chemotherapy is not the only option available to patients for whom another course of treatment ("second-line") is necessary. The survey uncovered a strong desire from patients to be more engaged in clinical decision-making, especially to be informed about all available treatment options and their respective side effects.

"These survey results quantify what many of us have been hearing from our patients for years: that they feel the burden of treatment and they want to be informed partners in decisions about their care," said lead author Ginger J. Gardner, MD, FACOG, Gynecologic Oncologist, Memorial Sloan Kettering Cancer Center and Chair, FWC. "These findings are a clear call to action for providers in our field to ensure patients understand their options and have a voice in treatment conversations at every stage of their journey."

Key Findings: Chemotherapy Experiences and Preferences

When looking at all respondents (N=119), the survey found:

70% of patients reported that the burden of chemotherapy treatment was almost as much as the burden of the disease itself, and of those patients who received multiple lines of chemotherapy (n=64)*, 41% reported that their second-line of chemotherapy was worse than the first.
75% of those patients who had received multiple lines of chemotherapy* reported that the treatments had taken a toll on their personal lives.
*Denotes results based on small sample size (n=50-99); should be interpreted as directional only
95% of patients experienced hair loss (alopecia) with their chemotherapy treatment and 82% said that they would prefer a treatment option that causes less hair loss than chemotherapy, if one were available and applicable to them.
Beyond the immediate impact of treatment, patients reported a range of long-term side effects — most commonly fatigue (51%), followed by neuropathy (42%), trouble concentrating or remembering things (35%) and alopecia (35%).
71% of patients reported that they would prefer not to receive chemotherapy again in the future.
Key Findings: Treatment Decision-Making and Patient Education

When looking at all respondents (N=119), the survey found:

Only around 2 in 5 patients (42%) reported that they shared decision-making equally with their doctor and 79% indicated they wish their provider spent more time discussing what matters to them when it comes to their treatment.
9 in 10 patients (91%) wanted to know more about EC treatment options beyond chemotherapy.
92% of patients said they would prefer a treatment plan personalized to them over a one-size-fits-all approach.
Other Findings of Note: Differences in Treatment Experiences and Perceptions*

When looking specifically at respondents who identified as a person of color (n=47), the survey found:

They were less likely to feel informed about their EC treatment options overall (62% vs. 79% of white respondents);
70% reported spending 5 or more hours for a typical chemotherapy visit including scheduling and confirming appointments, getting to and from the appointment, and receiving treatment, while 55% of white respondents reported the same; and
74% agreed that they would prefer not to receive chemotherapy again in the future vs. 69% of white respondents.
*Denotes results based on extremely small sample size (n<50); should be interpreted as directional only

"It’s important that clinicians understand and acknowledge that there is a ‘trust gap,’ where patients of color may understandably doubt that their voices are truly being heard," said study author Adrienne Moore, President, ECANA. "In terms of treatment options, when the harsh physical toll of chemotherapy is compounded by the pressures of family, finances, and outside stressors – which often fall more heavily on communities of color – the combined burden can lead many to seek alternatives over repeating a chemotherapy treatment experience."

"The results of this survey provide a powerful reminder to the medical community that behind every data point is a person navigating one of the most difficult experiences of their life," said study author Sue Friedman, MD, Executive Director, FORCE. "So many of the people I connect with describe feeling overwhelmed by their treatment experience and wishing they had more time, more information, and more of a voice in their care. I encourage all patients to speak up, ask questions, and work with their healthcare team to build a treatment plan that reflects what matters most to them."

Findings from the survey will be presented as a poster, Endometrial Cancer: The Patient Voice is Center Stage, at the SGO Annual Meeting on Women’s Cancer, taking place April 10–13, 2026, in San Juan, Puerto Rico. The poster presentation (Abstract #1356) will happen in Exhibit Hall B on Sunday, April 12, 2026 from 11:00 AM–12:00 PM AST and 4:00–4:45 PM AST.

About the Survey

The Endometrial Cancer Patient Experience Survey was conducted online within the United States between April 21 and July 7, 2025, by The Harris Poll on behalf of Eisai Inc. in collaboration with ECANA, FORCE, and FWC. A total of 571 individuals were recruited to participate through a Harris Poll affiliate and through ECANA’s patient list, of whom 484 underwent eligibility screening (participation rate = 85%). Eligible respondents were ≥18 years of age, assigned female at birth, residents of the United States, diagnosed by a healthcare provider with uterine/endometrial/womb cancer and had received at least one line of prior chemotherapy. Of the 484 who underwent eligibility screening, 119 met the criteria for survey completion and subsequent data analysis (ECANA patient list, n=21; Harris Poll affiliate, n=98).

Select demographic and clinical characteristics of survey respondents included:

Median age: 59 years
Race/ethnicity: 61% White, 22% Hispanic, 16% Black/African American, 1% Asian
Mean time from first experiencing symptoms to EC diagnosis: 3 years
Treatment history (ever received): chemotherapy (100%), surgery (93%), radiation therapy (85%), immunotherapy (50%), oral targeted therapy (42%), hormonal therapy (40%)
54% received multiple prior lines of chemotherapy; 46% received one prior line of chemotherapy
Raw data were not weighted and are therefore only representative of the individuals who completed the survey. For this study, the patient sample data are accurate to within ±8.9 percentage points using a 95% confidence level. This credible interval will be wider among subsets of the surveyed population of interest. All sample surveys and polls, whether or not they use probability sampling, are subject to other multiple sources of error which are most often not possible to quantify or estimate, including, but not limited to coverage error, error associated with nonresponse, error associated with question wording and response options, and post-survey weighting and adjustments.

About Endometrial Cancer

Endometrial cancer begins in the inner lining of the uterus, which is known as the endometrium, and is the most common type of cancer in the uterus. More than 90% of uterine body cancers occur in the endometrium. In the U.S., it is estimated there will be approximately 68,270 patients diagnosed with uterine body cancer and approximately 14,450 patient deaths from the disease in 2026. Globally, endometrial cancer is the sixth most common cancer in women and the 15th most common cancer overall. Despite these figures, endometrial cancer is severely underfunded relative to its rapidly increasing mortality rate. Following primary treatment, patients face a risk of their cancer returning, often as distant metastasis, which is associated with poorer outcomes.

(Press release, Eisai, APR 10, 2026, View Source [SID1234664303])

On April 10, 2026 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), a clinical-stage oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved antibody drug conjugate (ADC) cancer treatments, reported three posters from a real-world analysis supporting the therapeutic potential of targeting mucin 16 (MUC16) with a next-generation ADC for the treatment of ovarian and endometrial cancers at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancer, being held April 10-13, 2026, in San Juan, Puerto Rico.

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MUC16 is a cell-surface glycoprotein that promotes tumor cell proliferation, metastasis and immune evasion. MUC16 is overexpressed in multiple tumor types with limited expression in normal tissue. A large-scale RNA analysis demonstrated that MUC16 is highly and stably expressed across ovarian cancers and in the most aggressive and most common subtypes of endometrial cancer, including in later-stage disease, supporting its potential as a clinically meaningful ADC target and reinforcing the rationale for clinical development of HWK-016.

"Across both ovarian and endometrial cancers, MUC16 shows a high level of stable expression that is uncommon among many therapeutic targets," said Kathleen N. Moore, MD, Deputy Director and Director of Phase 1 Oncology Trials, The Fred & Pamela Buffett Cancer Center, University of Nebraska Medical Center. "The consistency of MUC16 expression across disease stage and platinum sensitivity supports the relevance of a MUC16-targeted ADC in earlier-line settings, as well as for heavily pretreated patients and as part of combination strategies."

Key Findings Include:

MUC16 in Ovarian Cancer

MUC16 has high, tumor‑selective expression, with concordance between MUC16 mRNA expression and MUC16 protein abundance by IHC.
MUC16 is highly expressed across most histologic subtypes, including the most common subtype, high-grade serous ovarian carcinoma (HGSOC), which accounts for ~70% of cases.
In HGSOC, MUC16 expression is stable across disease stages, metastatic status and platinum sensitivity, underscoring relevance in early- and late-line treatment settings.
MUC16 has the highest median expression, ranging from 2- to >100-fold higher than other clinically validated and emerging ADC targets, including NaPi2b, HER2, FRα, TROP2, B7-H4, CDH6 and CLDN6.
MUC16 in Endometrial Cancer

MUC16 has high, tumor‑selective expression, with concordance with MUC16 protein abundance by IHC.
MUC16 is most highly expressed in serous adenocarcinoma (the most aggressive subtype accounting for ~40% of deaths), followed by endometrioid adenocarcinoma (the most common subtype, accounting for ~90% of cases).
In serous adenocarcinoma, MUC16 expression is stable across disease stages and metastatic status, and has the highest median expression, 1.4- to 3.7-fold higher than other clinically validated and emerging ADC targets, including NaPi2b, HER2, TROP2, FRα and B7H4.
Earlier MUC16‑directed ADCs targeted the cleaved extracellular portion of the protein (CA125), which is shed into circulation, limiting tumor delivery. HWK‑016, Whitehawk’s investigational, next‑generation MUC16‑targeted ADC, is designed to overcome this antigen sink effect by selectively targeting the membrane‑bound, non‑shed portion of MUC16. HWK‑016 leverages a stable, cleavable linker to deliver a novel topoisomerase I (TOP1) inhibitor payload. HWK-016 is currently being evaluated in a Phase 1 clinical trial in patients with advanced ovarian and endometrial cancers [NCT07470853]. Initial clinical data are expected in the first half of 2027.

"The real-world data reported at SGO reinforce MUC16 as a ‘super expressed’ and durable target for gynecological cancers, strengthening the biological rationale for HWK-016," said Margaret Dugan, MD, Chief Medical Officer of Whitehawk Therapeutics. "By combining selective targeting of the membrane‑bound, non‑shed portion of MUC16 with our differentiated carbon‑bridge cystine‑repairing linker‑payload technology, HWK‑016 exemplifies our broader strategy of applying advanced ADC engineering to well‑validated tumor biology to potentially generate meaningfully improved outcomes for patients."

Poster Presentation Details:

Title: MUC16 in ovarian cancer: high, stable expression across histologic subtypes, disease stages, and platinum sensitivity status supports antibody-drug conjugate development
Presenter: Ursula A. Matulonis, MD, Dana-Farber Cancer Institute
Date & Time: April 12, 2026, 11:00 AM – 12:00 PM and 4:00 – 4:45 PM

Title: MUC16 as a therapeutic target: advancing antibody-drug conjugates for ovarian cancer treatment
Presenter: David M. O’Malley, MD, The Ohio State University
Date & Time: April 12, 2026, 11:00 AM – 12:00 PM and 4:00 – 4:45 PM

Title: High and stable MUC16 expression in endometrial cancer highlights potential for targeted antibody-drug conjugate development
Presenter: Kathleen N. Moore, MD, University of Nebraska Medical Center
Poster Tour – Group 9: Targeted Tactics and Therapies
Date & Time: April 12, 2026, 11:30 AM – 12:00 PM

The analysis was conducted as part of a previously announced collaboration between Whitehawk and Tempus AI. The posters will be accessible on the Presentations page of the Investors & News section of the Company’s website at www.whitehawktx.com.

(Press release, Whitehawk Therapeutics, APR 10, 2026, View Source [SID1234664302])

On April 10, 2026 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company, reported plans to issue a joint IDEAYA and Servier pre-market press release and host a conference call and webcast on Monday, April 13, 2026 at 8:00 a.m. ET to disclose topline results from their ongoing Phase 2/3 registrational trial, OptimUM-02, evaluating darovasertib in combination with crizotinib in patients with first-line HLA*A2-negative metastatic uveal melanoma. The call will include members of IDEAYA’s management joined by a distinguished key opinion leader.

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Conference Call and Webcast Information

The webcast registration information can be accessed using this link or by visiting the Events section of the IDEAYA website. A replay of the webcast will be available on IDEAYA’s website for 30 days following the live event.

(Press release, Ideaya Biosciences, APR 10, 2026, View Source [SID1234664301])

On April 10, 2026 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported two-year median follow-up data from the Phase 2 RAMP 201 clinical trial that evaluated AVMAPKI FAKZYNJA combination therapy (avutometinib capsules; defactinib tablets) in patients with recurrent low-grade serous ovarian cancer (LGSOC) will be presented today during an oral plenary session at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancers taking place in San Juan, Puerto Rico, April 10-13, 2026.

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"Patients who remained on treatment with avutometinib plus defactinib for two years were able to maintain the same level of response and duration of therapy as seen in the primary analysis, suggesting that patients can stay on the combination for a long period of time, derive benefit and have manageable toxicity during long-term administration of these medications," said Rachel Grisham, M.D., RAMP 201 Presenting Investigator, Section Head, Ovarian Cancer at Memorial Sloan Kettering Cancer Center (MSK) in New York, NY, and Global Lead Principal Investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301. "As we approach the one-year FDA approval anniversary of avutometinib in combination with defactinib, this analysis reinforces progress in bringing a durable and clinically meaningful option to patients."

In the updated analysis, with ongoing patients presenting a median follow-up of 24.9 months, efficacy measures, including median duration of response (mDOR) and median progression free survival (mPFS), and safety, are consistent with the primary analysis, which was conducted more than 13 months prior:

Long-Term Median Follow-Up
(data cutoff of August 12, 2025)

(N=17)

Primary Analysis*
(data cutoff of June 30, 2024)

(N=109)

Total
(N=17)

KRAS mt
(N=12)

KRAS wt
(N=5)

Total
(N=109)

KRAS mt
(N=57)

KRAS wt
(N=52)

mDoR

31.1 months

31.1 months

12 months

31.1 months

31.1 months

9.2 months

mPFS

12.9 months

19.6 months

12.7 months

12.9 months

22 months

12.8 months

*The primary analysis was published in the Journal of Clinical Oncology (Banerjee et al, July 11, 2025).

Fifty percent of patients with KRAS-mutated and 30 percent with KRAS wild-type LGSOC remained on therapy for more than one year. Adverse events (AEs) were consistent with the primary analysis with no new safety signals observed, and a 12 percent discontinuation rate due to adverse events.

"With two years of follow-up demonstrating durable benefit, these analyses reinforce confidence in avutometinib plus defactinib in the real-world setting for patients living with recurrent LGSOC," said Bradley Monk, M.D., FACOG, FACS, RAMP 201 Investigator and Gynecologic Oncologist with Florida Cancer Specialists & Research Institute and Director GOG-Partners. "Notably, seeing one-third of patients without KRAS mutations remain on therapy for over a year is clinically meaningful, especially in a patient population with limited treatment options. The data also underscore the combination’s manageable safety profile and how dose holds can be used effectively to maintain dose intensity, helping distinguish this treatment strategy from prior MEK-based approaches."

Exposure-Response Analysis for Avutometinib in Combination with Defactinib in LGSOC

Efficacy analyses included patients with LGSOC (N=158) from the FRAME and RAMP 201 studies, while safety analyses included a broader population (N=303) from the FRAME, RAMP 201, and RAMP 202 studies.

All efficacy endpoints, including overall response rates, duration of response, and best target lesion response, suggest the best therapeutic effect is achieved with the FDA approved dose of avutometinib 3.2 mg twice weekly plus defactinib 200 mg twice daily. While a lower avutometinib dose may mitigate treatment emergent adverse events (TEAEs) (the most frequent being grade ≥2 skin disorders (39.9%; n=121), followed by grade ≥2 gastrointestinal toxicity (34.0%; n=103), grade ≥2 liver function tests (25.1%; n=76), and grade ≥3 creatine phosphokinase elevation (14.5%; n=44)), it may also compromise the efficacy. Importantly, these analyses demonstrated that TEAEs can be monitored and managed with dose interruptions, and subsequently resume treatment at the approved dose level, to allow patients to stay on treatment.

Verastem has an exhibition booth (#608) at the meeting to provide an overview of its approved therapy and ongoing cancer research. The full schedule and poster titles are available online at the SGO 2026 Annual Meeting on Women’s Cancer website.

About RAMP 201

RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction.

About Low-Grade Serous Ovarian Cancer (LGSOC)

LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication

AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions

The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.

(Press release, Verastem, APR 10, 2026, View Source [SID1234664300])

On April 10, 2026 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported that the company received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) for the Company’s Biologics License Application (BLA) for RP1 in combination with nivolumab for the treatment of advanced melanoma.

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Replimune disagrees with the FDA about whether the data set, upon which breakthrough therapy designation was awarded, is sufficient to allow this promising medicine to be made available to advanced cancer patients. In the IGNYTE trial, patients with confirmed progression on an anti-PD-1 based regimen who received RP1 plus nivolumab had a 34% response rate with a median duration of 24.8 months with a favorable safety profile.

"It is deeply disappointing that the FDA has not exercised regulatory flexibility to meet patients’ needs given the data supporting strong efficacy and the favorable safety profile. Approximately 8,500 Americans with advanced melanoma die every year. The country’s foremost melanoma specialists stood behind the RP1 data. Patients and caregivers pleaded for urgency. All of it was met with inconsistent communication and a fragmented and slow-moving regulatory process which clearly puts U.S. innovation at risk," said Sushil Patel, Ph.D., CEO of Replimune. "As we previously communicated, without timely accelerated approval, the development of RP1 will not be viable. We are devastated for our committed employees who have worked tirelessly for patients but at this point we have no choice but to eliminate jobs, including substantially scaling back our U.S. based manufacturing operations. A treatment desperately needed by patients will not be available. Not because the medicine failed. Because the system did."

Inconsistent agency process and communication thwarts innovation
With the CRL, the company learned that a different review team was appointed for the resubmission and replaced the prior team who had interacted with the company. A senior member of the prior review team stated publicly that the "BLA clinical team thought the applicant had provided adequate evidence to support contribution of effect of RP1 plus nivolumab but leadership did not agree." The new team did not meet with the company during the review process despite the company offering.

In the CRL, the agency appears to have contradicted their positions expressed at the September 2025 Type A meeting, including on the following points:

After testimony from melanoma experts, the agency did not raise further concerns about the heterogeneity of the patient population in IGNYTE and acknowledged that randomizing patients to an anti-PD1 only arm in the confirmatory study was not feasible.
Following an agency suggestion, the company submitted a proposal for a descriptive analysis from IGNYTE-3 supporting contribution of components. The company also included data from IGNYTE showing median progression free survival on RP1 plus nivolumab was 30.6 months compared to 4.4 months on their prior PD-1 based regimen. The company requested feedback, however, the FDA did not respond and subsequently accepted the resubmission as a complete response to the July 2025 CRL.
The FDA raised several points related to tumor assessment methodology. As requested by the FDA, responses in IGNYTE were assessed using RECIST 1.1 without modifications. In addition, the company provided detailed analyses showing no material difference in response rates between injected and non-injected lesions. The company also provided a comprehensive analysis which showed that biopsies and surgical interventions did not impact tumor response.
Prior to the original BLA submission, standard regulatory meetings were conducted to discuss trial design, patient population, and the BLA package requirements. While a randomized controlled trial was preferred, the FDA suggested in the March 2021 Type B minutes that if the data was sufficiently compelling, a single arm trial could be acceptable for consideration under accelerated approval. At the subsequent pre-BLA meeting, the FDA stated "we do not object to your proposal to submit a BLA based primarily on data from the cohort of patients (n=140) in the Phase 2 IGNYTE trial who had advanced melanoma and progressed while being treated with prior anti-PD-1 based therapy." The company subsequently submitted a BLA which was accepted with breakthrough therapy designation and granted priority review. Based on feedback from the FDA, the company initiated a resource-intensive global Phase 3 trial, IGNYTE-3, to satisfy the regulatory requirement that a confirmatory study be underway for an accelerated approval.

About Melanoma
Melanoma is the fifth most common cancer, with approximately 112,000 new cases estimated in the U.S. in 2026, and the most lethal form of skin cancer, accounting for nearly 8,500 deaths annually. Standard of care therapy includes treatment with immune checkpoint blockade, to which approximately half of patients will not respond or will progress after treatment. Melanoma is considered advanced when the cancer spreads beyond the primary tumor to other parts of the body.

About RP1
RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

(Press release, Replimune, APR 10, 2026, View Source [SID1234664299])