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Corcept Submits New Drug Application for Relacorilant as a Treatment for Patients with Platinum-Resistant Ovarian Cancer

On July 14, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported that it has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its proprietary, selective cortisol modulator, relacorilant, to treat patients with platinum-resistant ovarian cancer (Press release, Corcept Therapeutics, JUL 14, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-submits-new-drug-application-relacorilant-treatment-0 [SID1234654367]).

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Corcept’s filing is based on positive data from its pivotal Phase 3 ROSELLA and Phase 2 trials. In these trials, patients who received relacorilant plus nab-paclitaxel experienced improved progression-free and overall survival compared to patients who received nab-paclitaxel monotherapy, with no need for biomarker selection. Relacorilant was well-tolerated, consistent with its known safety profile. Importantly, the type, frequency and severity of adverse events in the combination arms were similar to those in the nab-paclitaxel monotherapy arms. Relacorilant did not increase the safety burden of the patients who received it.

"This submission is an important milestone for Corcept as we now have two New Drug Applications before the FDA: Relacorilant in combination with nab-paclitaxel as a treatment for people with platinum-resistant ovarian cancer and relacorilant as a treatment for patients with hypercortisolism," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. "Better treatment options are needed for the many patients living with these diseases. Our oncology and endocrinology business units are already working to make sure relacorilant is available immediately following regulatory approval."

About Relacorilant

Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body’s other hormone receptors. Corcept is developing relacorilant in ovarian cancer and a variety of other serious disorders, including endogenous hypercortisolism and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2025 for relacorilant as a treatment for patients with hypercortisolism.

About Cortisol’s Role in Oncology

Cortisol plays a role in tumor growth through several mechanisms. It helps solid tumors resist chemotherapy by inhibiting cellular apoptosis — the tumor-killing effect chemotherapy is meant to stimulate. In some cancers, cortisol promotes tumor growth by activating oncogenes in the cells to which it binds. Cortisol also suppresses the body’s immune response, which weakens its ability to fight all diseases, including cancer.

About Platinum-Resistant Ovarian Cancer

Ovarian cancer is the fifth most common cause of cancer death in women. Patients whose disease returns less than six months after receiving platinum-containing therapy have "platinum-resistant" disease. There are few treatment options for these women. Median overall survival following recurrence is approximately 12 months with single-agent chemotherapy. Approximately 20,000 women with platinum-resistant disease are candidates to start a new therapy each year in the United States, with at least an equal number in Europe.

Atara Biotherapeutics Provides Regulatory and Business Updates on Tabelecleucel (Tab-cel®)

On July 14, 2025 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported that it has resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for tabelecleucel (EBVALLO or tab-cel) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy (Press release, Atara Biotherapeutics, JUL 14, 2025, View Source [SID1234654366]). There are no FDA approved therapies in this treatment setting.

"The BLA resubmission for tab-cel represents the collaborative efforts with our partner, Pierre Fabre Laboratories, to address the third-party manufacturing facility observations outlined in the January 2025 Complete Response Letter," said Cokey Nguyen, President and Chief Executive Officer of Atara. "We look forward to continued engagement with the FDA throughout its review and with Pierre Fabre Laboratories as they actively prepare for the potential launch of this innovative therapy in the U.S."

Tab-cel is an allogeneic, EBV-specific T-cell immunotherapy designed to target and eliminate EBV-infected cells. The BLA is supported by pivotal and supportive data covering more than 430 patients treated with tab-cel across multiple life-threatening diseases, including the latest pivotal ALLELE study data that demonstrated a statistically significant 48.8% Objective Response Rate (ORR) (p<0.0001) and favorable safety profile consistent with previous analyses.

Tab-cel has been granted Breakthrough Therapy Designation for the treatment of rituximab-refractory EBV-associated lymphoproliferative disease by the U.S. FDA and has orphan drug designation for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disorders.

Corporate Updates

Tab-cel Transition Activities: The company is finalizing the transfer of the following clinical studies associated with tab-cel:

NCT03394365: Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Participants with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease (EBV+ PTLD) After Failure of Rituximab or Rituximab and Chemotherapy (ALLELE)
NCT04554914: A Study to Evaluate Tabelecleucel in Participants with Epstein-Barr Virus-associated Diseases. This study is a multicenter, multicohort, open-label, single-arm, Phase 2 study investigating the efficacy and safety of tabelecleucel for the treatment of EBV-associated diseases.
Upon completion substantially all operational activities and associated costs related to tab-cel will transfer to Pierre Fabre Laboratories.

The sponsorship of the BLA continues to be maintained by Atara.

Cash Runway and Future Tabelecleucel (Tab-cel) Milestone and Royalty Income: Atara projects that its cash, cash equivalents and short-term investments of approximately $22M as of June 30, 2025, combined with the cost reduction initiatives implemented in the first half of 2025, will enable funding of all currently planned operations, including one-time restructuring costs, into the first quarter of 2026, that Atara believes will be sufficient to fund the ongoing activities required to achieve potential BLA approval.

Additionally, under its commercialization agreement with Pierre Fabre Laboratories, Atara is eligible to receive a $40 million milestone payment upon FDA approval of the tab-cel BLA, as well as significant double-digit tiered royalties as a percentage of net sales, and milestones related to commercial sales of EBVALLO.

The estimate of our cash, cash equivalents and short-term investments as of June 30, 2025, is preliminary, has not been audited and it is subject to change upon completion of our financial statement closure procedures. Our independent registered public accounting firm has not audited or completed any procedures with respect to this estimate. Such estimates are based on assumptions and plans that are subject to change and such changes could materially impact our expected cash runway. These assumptions include the completion of specific development and regulatory activities by us and actions taken by third parties, and are, therefore, uncertain at this time. Any delay in these activities will create additional expenses and cash needs for us.

We have not yet completed our quarter-end financial close process for the quarter ended June 30, 2025. This estimate of our cash, cash equivalents and short-term investments as of June 30, 2025, is preliminary and is subject to change upon completion of our financial statement closing procedures. Additional information and disclosure would be required for a more complete understanding of our financial position and results of operations as of and for the quarter ended June 30, 2025.

Alligator Bioscience comments on Henlius dosing first patient in the US in Phase 3 trial of HLX22 in gastric cancer

On July 14, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported on the announcement by Shanghai Henlius Biotech, Inc. that it has dosed the first patient in the United States in its global Phase 3 clinical trial (NCT06532006) of HLX22 in combination with trastuzumab and chemotherapy as a first-line treatment for HER2-positive metastatic gastric and gastroesophageal junction (GEJ) cancer (Press release, Alligator Bioscience, JUL 14, 2025, View Source [SID1234654365]).

HLX22 is an anti-HER2 monoclonal antibody being developed by Henlius under a license from AbClon, Inc., following a discovery collaboration which grants Alligator the right to participate in potential future revenues.

"We are encouraged to see Henlius dose the first patient in the United States in the Phase 3 trial of HLX22, reflecting continued momentum for this promising program in HER2-positive gastric cancer," commented Søren Bregenholt, CEO of Alligator Bioscience. "While Alligator is not directly involved in the development, we follow it closely as it represents an opportunity for meaningful revenue streams from milestones and royalties upon the future approval of HLX22."

Under the terms of the agreement, Alligator is entitled to 35% of AbClon’s revenue from its sublicense agreement with Henlius.

Two Additional Complete Responses and Three Partial Responses in azer-cel CAR T Phase 1b trial

On July 14, 2025 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported exciting new data from its Phase 1b clinical trial evaluating azer-cel (azercabtagene zapreleucel) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (Press release, Imugene, JUL 14, 2025, https://mcusercontent.com/e38c43331936a9627acb6427c/files/9dcc6e8a-a173-fb47-bc66-6b21c92ab09f/Two_Additional_Complete_Responses_in_azer_cel_Ph1b_trial.pdf [SID1234654361]).

In February 2025, Imugene announced that a total of four out of seven patients had achieved a Complete Response (CR), defined as the disappearance of all signs of cancer in response to treatment. Since then, two additional patients have also achieved a Complete Response, and three patients have achieved Partial Response (cancer reduction by at least 50%) bringing the best overall response rate to 75% and the CR rate to 55%. The duration of response continues to mature. These patients are being treated with azer-cel and interleukin 2 (IL -2).

Evaluable
patients

Treatment

Overall Response Rate (ORR)

Complete Response (CR)

At Day 60

Best Durability (Time of response)

DLBCL

Lymphodepletion (LD)1 +azer-cel

+Interleukin-2 (IL-2)

9/12 (75%)

6/11 (55%)

>450 days on going

For approved, autologous CD19 CART products, the average time to best response is 2-3 months with some patients taking up to 6 months to achieve their best response.

Azer-cel is being developed as a potential allogeneic, off-the-shelf, CAR T-cell therapy, addressing key limitations of approved autologous CAR T drugs, including geographical access to treatment centres, manufacturing complexity and time to receive treatment (on-demand).

Based on the updated response rate and maturing durability data, as well as having been awarded FDA Fast Track Designation for DLBCL in March 2025, Imugene will request a Type B (End of Phase 1) Meeting in Q4 2025, with the US FDA to present the data and to discuss designs for a pivotal / registrational trial for azer-cel.

Leslie Chong, Managing Director and CEO of Imugene, said:

"We are very pleased with the continued positive data coming from the azer-cel trial, which further reinforces its potential as a treatment for DLBCL patients who have failed several previous lines of therapy. The data also significantly improves our position from both a regulatory and commercial standpoint, and we look forward to expanding on these discussions with the FDA.

Additionally, given the positive results, we are opening the trial to other niche blood cancer indications, such as PCNSL and other subtypes of B Cell Lymphoma, for CAR T naïve patients. This is a high unmet need with potential to expedite and expand the scope of azer-cel."

Dr John Byon, Chief Medical Officer of Imugene, said:

"DLBCL remains one of the most aggressive forms of lymphoma, and despite the existing therapies, there are a large number of patients that still face relapse or resistance. We are seeing significant potential from azer-cel to date in its ability to provide a critical step forward for these patients who have relapsed on multiple therapies, offering deep and durable responses with a one-time treatment.

We remain deeply committed to transforming the standard of care in difficult-to-treat blood cancers, where significant unmet medical need still exists."

The FDA Fast Track Designation for DLBCL received for azer-cel is designed to facilitate the development and expedite the review of drugs that address serious or life-threatening conditions and meet an unmet medical need. Benefits of the designation include more frequent meetings with the FDA to discuss development plans, the option for rolling review of regulatory submissions, and potential eligibility for Accelerated Approval and Priority Review upon meeting relevant criteria.

Imugene continues to actively enrol patients into the Phase 1b azer-cel trial at ten US sites with up to six sites in Australia planned, after the first Australian patient was dosed in January 2025 at Royal Prince Alfred Hospital in Sydney, resulting in a Complete Response.

About the Phase 1b azer-cel trial

The azer-cel allogeneic CAR T trial is an ongoing, open-label, multi-centre Phase 1b clinical trial in the U.S. and Australia, for CAR T relapsed patients with DLBCL. The study has recently expanded to include and treat CAR T naïve patients diagnosed with a broad range of Non-Hodgkins lymphomas including primary central nervous system lymphoma (PCNSL), chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL), marginal zone lymphoma (MZL), Waldenstrom macroglobulinemia (WM) and follicular lymphoma (FL). Treatment with azer-cel, lymphodepletion (LD) and IL-2 is showing promising results with evidence of meaningful clinical activity, and durability of response. Additionally, the safety profile is manageable and generally well tolerated.

About diffuse large B cell lymphoma (DLBCL)

DLBCL is an aggressive and fast-growing type of non-Hodgkin’s lymphoma (NHL), a type of blood cancer. DLBCL is the most common type of NHL, with approximately 160,000[1] global cases per year and approximately 30,000 new cases per year in the U.S. Relapsed/refractory DLBCL has a high unmet medical need; ~60% of patients treated with approved autologous CD19 CAR T relapse.

About primary central nervous system lymphoma (PCNSL)

PCNSL is a rare and aggressive form of non-Hodgkin lymphoma (NHL), a type of blood cancer that originates in the brain, spinal cord, leptomeninges, or eyes, usually without evidence of systemic disease. In the U.S., there are approximately 1,500 to 1,800 new cases per year with limited approved treatment options and is a high unmet need. Currently, there are no CAR T-cell products approved for the treatment of PCNSL providing a unique opportunity for azer-cel to treat CART naïve patients.

About other types of B Cell Lymphoma

Other subtypes of non-Hodgkin lymphoma (NHL) include chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), the most common slow growing leukemia that can become resistant to therapy; marginal zone lymphoma (MZL), a slow-growing B-cell lymphoma that arises in lymphoid tissues associated with mucosal sites like the stomach and lung; Waldenström macroglobulinemia (WM), a rare slow-growing lymphoma characterized by excess IgM production, which can cause multiple complications ; and follicular lymphoma (FL), a common slow-growing NHL that can become more aggressive. While several targeted therapies and monoclonal antibodies are available for these types of B Cell Lymphoma, relapsed or refractory disease remains an ongoing challenge, highlighting the ongoing need for continued innovation and new and better treatments.

About Interleukin 2 (IL-2)

IL-2 is a cytokine (a protein that affects what happens between cells in the immune system) that helps T-cells (which are part of the immune system that help fight cancer) grow and survive. IL-2 has been shown to help T cells live longer and to enhance the cancer killing functions of CAR T cells, making them more effective at targeting and killing cancer cells.

CARsgen Successfully Defends Its GPC3 CAR-T Patent at the EPO

On July 13, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported a favorable outcome in opposition proceedings before the European Patent Office (EPO) concerning its European patent EP3445407, which covers its GPC3-targeted CAR-T cell therapy (Press release, Carsgen Therapeutics, JUL 13, 2025, View Source [SID1234654362]).

On July 3, 2025, a U.S.-based biotechnology — the sole appellant among the original two opponents — formally withdrew its appeal against the EPO Opposition Division’s earlier decision to maintain the patent. This withdrawal renders the EPO’s decision final and binding for the opponents, effectively concluding their part in the opposition process.

The patent was granted by the EPO in 2022 and opposed by two parties in 2023. Following oral proceedings, the EPO Opposition Division issued a decision to maintain the patent in amended form, upholding key claims related to the use of GPC3 CAR-T cell therapy following cyclophosphamide and fludarabine lymphodepletion pretreatment, in the treatment of liver cancer, lung cancer, ovarian cancer, breast cancer, gastric cancer, and thyroid cancer. Following the decision, only one opponent filed an appeal within the allowable period. That appeal has now been withdrawn.

Under EPO procedures, the opponents or any other third party can no longer challenge the patent at the EPO. This development further reinforces CARsgen’s intellectual property position in the field of GPC3-targeted CAR-T therapies, a promising and innovative approach for the treatment of solid tumors.