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EUROPEAN MEDICINES AGENCY (EMA) HAS GRANTED ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR AB8939 IN THE TREATMENT OF ACUTE MYELOID LEUKEMIA

On April 23, 2025 AB Science SA (Euronext – FR0010557264 – AB) reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia (AML) (Press release, AB Science, APR 23, 2025, View Source [SID1234652043]).

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AB8939 had already obtained orphan drug designation from the US Food and Drug Administration (FDA) in AML.

Granting of this orphan drug status in the EU is a significant milestone because it means that the COMP considered that AB8939 offers a significant benefit to people suffering from this condition in addition to existing treatments.

Indeed, the criteria to obtain orphan drug designation at EMA differ from those at FDA and are very stringent for the following reasons :

-There must be no satisfactory method of diagnosis, prevention or treatment of the condition or, if such a method exists, the medicinal product must offer a significant benefit to patients. -Because the application is based on an assumption of significant benefit, a comparison with authorized treatments is required.

-Significant benefit means that a medicine produces a clinically relevant advantage or makes a major contribution to patient care, as compared with existing methods to treat the condition. Thus, orphan designation is given to a product that will improve patients’ current treatment, having considered what else is available.

-To follow the spirit of the orphan legislation, which makes it clear that an orphan application may bemade at any stage of the development, significant benefit will be based on the available evidence at the stage of designation.

In support of the significant benefit of AB8939 in AML, AB Science has released preclinical data from mouse models demonstrating a significant benefit of AB8939 treatment over current therapies, such as cytarabine, azacitidine (Vidaza) and venetoclax (Venclexta). This included:

-Efficacy on resistant cells: AB8939 manages to have an effect on the cancer cells (blasts) of AML patients, even when these cells are resistant to other drugs such as vincristine or cytarabine. For example, 45% of vincristine-resistant cells and 66% of cytarabine-resistant cells still respond to AB8939, including in severe cases with complex genetic mutations (MECOM, TP53).

-Convincing results in xenograft models derived from refractory AML patients: In these mouse models which mimick human AML, AB8939 reduces tumors and prolongs survival, even when cells areresistant to cytarabine. -Additive effect with reference treatments: When used with other treatments (cytarabine, Vidaza or venetoclax), AB8939 further improves results. For example, with venetoclax, it eliminates cancer cells in the blood, spleen and bone marrow, without serious side effects

-Furthermore, unlike venetoclax, AB8939 does not cause blood toxicity (hematotoxicity) and appears to act synergistically with other treatments, reinforcing its efficacy.

AB Science also presented preliminary efficacy and safety data from phase 1 of AB8939 as a monotherapy, with a 3-day treatment cycle (stage 1 of phase 1) and a 14-day treatment cycle (stage 2 of phase 1).

Professor Olivier Hermine, President of AB Science’s Scientific Committee, member of the French Academy of Sciences and Head of the Hematology Department at Necker Hospital, commented: "This designation testifies to the potential of AB8939 for the treatment of AML. Indeed, AB8939 has shown activity as a monotherapy on Ara-C-resistant patient lines, including in unfavorable genetic situations (MECOM, TP53 mutations) that have resisted all treatments administered to date, as well as a synergistic effect with the reference treatments Vidaza and Venclexta. The ongoing Phase 1 trial will now evaluate the combination of AB8939 with these reference treatments in refractory patients".

About AB8939

AB8939 is a new synthetic molecule which jointly targets cancer cells, by destabilizing the microtubules essential for cell division, and cancer stem cells, by inhibiting enzymes (ALDH1A1 and ALDH2) essential for maintaining their physiological state and survival. The molecule ‘1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one’ is the chemical name of AB8939. The intellectual property of AB8939 is 100% owned by AB Science.

Protara Therapeutics to Host Conference Call and Webcast to Review Interim Data from Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC on Monday, April 28, 2025

On April 23, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported it will host a conference call and live webcast at 8:30 a.m. ET on Monday, April 28, 2025, to review updated safety and efficacy data from the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC), including data from patients who have reached the 12-month evaluation timepoint (Press release, Protara Therapeutics, APR 23, 2025, https://ir.protaratx.com/news-releases/news-release-details/protara-therapeutics-host-conference-call-and-webcast-review [SID1234652042]). The data will be featured during an interactive poster session at the American Urological Association 2025 Annual Meeting on Saturday, April 26, 2025, at 7:00 a.m. PT.

The live call can be accessed by registering as a participant here. Upon registration, participants will receive conference call dial-in information. A live webcast of the event can be accessed by visiting the Events and Presentations section of the Company’s website: View Source A replay of the webcast will be archived for a limited time following the event.

About ADVANCED-2

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-unresponsive (n≈100) and BCG-Naïve (n=31). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the U.S. Food and Drug Administration’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment, Draft Guidance for Industry.

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

Innate Pharma Regains Its Rights on CD123 Targeting ANKET® and Announces Sanofi’s Intention to Make a Strategic Investment in the Company

On April 23, 2025 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported review of their January 2016 Research Collaboration and License Agreement (the "2016 Agreement") with Sanofi (Press release, Innate Pharma, APR 23, 2025, View Source [SID1234652025]):

As previously disclosed and in alignment with its current strategic priorities, Sanofi will opt to pursue the development of SAR’514/IPH6401 (BCMA ANKET) in autoimmune indications under the terms of the 2016 License Agreement;
In alignment with both company’s current strategic priorities, Sanofi and Innate agreed to terminate the 2016 Agreement as it relates to SAR’579/IPH6101 (CD123 ANKET); Innate will regain its rights on SAR’579/IPH6101 (CD123 ANKET).
As part of these discussions, Sanofi and Innate have agreed to a potential investment by Sanofi of up to €15M in new shares of Innate. The size and price of this equity investment will be determined on the basis of the ongoing market conditions, if they are satisfactory.

The 2022 research collaboration and license agreement remain unchanged.

"We are very pleased that Sanofi has chosen to further strengthen our relationship through a potential strategic equity investment in the company. This would further validate the innovation and scientific progress at Innate Pharma delivered by our research and development. We acknowledge Sanofi’s portfolio prioritization, and we are encouraged to see our ANKET platform being pursued in autoimmune indications. We will continue to evaluate, plan and execute next steps for our proprietary ANKET programs in oncology and beyond," said Jonathan Dickinson, Chief Executive Officer of Innate Pharma.

SAR’579 (NCT05086315)/IPH6101

The originally Sanofi-led Phase 1/2 study with SAR’579 / IPH6101 (clinical study identifier: NCT05086315) is ongoing. Efficacy and safety results from the dose-escalation part, were shared in an oral presentation at the EHA (Free EHA Whitepaper) 2024 Congress. The data demonstrated that SAR’579 had clinical benefit and durable responses along with a favorable safety profile in patients with relapsed or refractory acute myeloid leukemia (AML), with 5 complete responses (4 CR / 1 CRi) achieved at 1 mg/kg, with durable CR (>10 months) observed in 3 patients.
In April 2024, Sanofi advanced SAR’579 / IPH6101 to the Phase 2 preliminary dose expansion of the trial.
The Parties will discuss a transition plan with regard to ongoing studies.
SAR’514/IPH6401:

The continued Sanofi-led Phase 1/2 study (clinical study identifier: NCT05839626) for the treatment of patients with relapsed or refractory multiple myeloma will be terminated early and SAR’514/IPH6401 will now be refocused to pursue development in autoimmune indications.
IPH62 and one additional target

IPH62 is a NK-cell engager program targeting B7-H3 under development from Innate’s ANKET platform. Following a research collaboration period and upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization.
Sanofi still retains the option of one additional ANKET target under the terms of the 2022 research collaboration and license agreement.

BriaCell Enrollment Pace Accelerating in Phase 3 Clinical Study in Advanced Metastatic Breast Cancer (Bria-ABC)

On April 22, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, reported that its ongoing pivotal Phase 3 clinical study (listed on ClinicalTrials.gov as NCT06072612 ) has consented over 100 and has enrolled over 75 patients (Press release, BriaCell Therapeutics, APR 22, 2025, View Source [SID1234652429]). BriaCell anticipates completing patient enrollment in late 2025 or early 2026, and may report top line data as early as H1-2026.

BriaCell’s pivotal Phase 3 clinical study is evaluating BriaCell’s lead clinical candidate, Bria-IMT, plus immune check point inhibitor versus physician’s choice in a dvanced metastatic b reast c ancer (Bria-ABC).

"We are pleased at the expanding patient enrollment in our Phase 3 study, and expect this to continue to grow," stated Dr. William V. Williams, BriaCell’s President & CEO. "We believe our novel therapeutic approach has the potential to transform cancer care for metastatic breast cancer patients, and are determined to bring our novel immunotherapy to market to help these patients."

"Enrollment pace and clinical investigator interest in our Bria-ABC study is above any I have seen," noted Giuseppe Del Priore, MD, MPH, BriaCell’s Chief Medical Officer. "We would like to thank our dedicated clinical investigators and patients for participating in this important study. Through their efforts, we will advance our novel cancer immunotherapy to other MBC patients whose medical needs remain unmet."

About BriaCell’s Pivotal Phase 3 Clinical Study of Bria-IMT Combination Regimen in MBC patients

Fifty-four clinical sites in the US are actively enrolling patients in BriaCell’s pivotal Phase 3 study in metastatic breast cancer. Additional sites are in various stages of start-up.

Interim data will be analyzed once 144 patient events (deaths) occur, comparing the overall survival (OS) in patients treated with the Bria-IMT combination regimen versus those treated with physician’s choice as the primary endpoint. Positive results of the pivotal Phase 3 study could result in full approval and marketing authorization for Bria-IMT in MBC patients. BriaCell recently announced positive Phase 2 survival data in a similar MBC patient population treated with the same Bria-IMT combination regimen . The Bria-IMT combination regimen has received FDA Fast Track designation.

For additional information on BriaCell’s pivotal Phase 3 study of Bria-IMT and an immune check point inhibitor in metastatic breast cancer, please visit ClinicalTrials.gov NCT06072612.

Egle Therapeutics to Highlight Novel Preclinical Findings for Regulatory T Cells Targeting Programs EGL-001 and EGL-002 With Poster Presentations at the 2025 AACR Annual Meeting

On April 22, 2025 Egle Therapeutics, a clinical-stage biotechnology company developing therapies targeting regulatory T cells (Tregs) for immuno-oncology and autoimmune diseases, reported that it will present two posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR) (Free AACR Whitepaper) 2025 being held in Chicago April 25-30, 2025 (Press release, Egle Therapeutics, APR 22, 2025, View Source [SID1234652036]).

In the poster, entitled "Preferential tumor uptake and retention of EGL-001, an anti-CTLA4-IL2 mutein fusion antibody achieving selective tumor Treg depletion".

Egle Therapeutics unveils preclinical biodistribution data on EGL-001, a novel anti-CTLA4-IL2 mutein fusion antibody designed to selectively deplete tumor-infiltrating regulatory T cells (Tregs). EGL-001 mode of action combines competitive inhibition of IL-2 signaling and potent downregulation of surface CD25, leading to induction of Treg apoptosis.

In vivo EGL-001 preferentially bound to the surface of Tregs due to their high CTLA-4 and CD25 expression and depleted them from the tumor microenvironment without affecting other immune cells. In a biodistribution study, EGL-001 accumulated and persisted in tumors while rapidly clearing from healthy tissues. This targeted approach resulted in deep tumor Treg depletion and was associated with compelling anti-tumor efficacy in multiple mouse tumor models.

EGL-001 is currently under evaluation in a First-In-Human clinical trial (NCT06622486), offering a new therapeutic strategy for alleviating immune suppression mediated by Treg to overcome resistance to immune checkpoint inhibitors.

Session Title: Antibodies 3: Multi-Target Checkpoint Inhibitors and Immune Activators; Session Date and Time: Tuesday, Apr. 29, 2025 02:00 PM – 05:00 PM; Location: Poster Section 37; Poster Board Number: 21 Presentation Number: 6080

In the poster, entitled "Enhanced anti-tumor efficacy through prolonged plasma membrane retention of a novel anti-CCR8/IL2 mutein fusion antibody".

Egle scientists present evidence on EGL-002, a novel anti-CCR8/IL2 mutein fusion antibody engineered to enhance Treg depletion in solid tumors. Via the dual binding of CCR8 and CD25 EGL-002 showed prolonged retention on the surface of tumor-infiltrating Tregs, avoiding the rapid internalization that limited the efficacy of conventional CCR8 antibodies. This led to superior ADCC and ADCP potency, resulting in near complete tumor Treg depletion and potent anti-tumor activity in mouse models and ex vivo human tumors. These findings position EGL-002 as a best-in-class anti-CCR8 and a promising monotherapy or combination partner for immune checkpoint blockade.

Session Title: Enhanced Antibodies, TCR Constructs, Cytokines and Chimeric Proteins; Session Date and Time: Monday, Apr. 28, 2025 02:00 PM – 05:00 PM; Location: Poster Section 35; Poster Board Number: 26 Presentation Number: 3767