On April 23, 2025 Adcendo ApS ("Adcendo"), a biotech company focused on the development of first and best-in-class ADCs for the treatment of cancers with high unmet medical need, reported it will be presenting data on its two novel ADC programs ADCE-T02, targeting Tissue Factor, and ADCE-D01, targeting uPARAP, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held in Chicago, Illinois, from April 25th to April 30th, 2025 (Press release, ADCendo, APR 23, 2025, View Source [SID1234652044]).

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ADCE-T02 is a potential best-in-class Topo-1 inhibitor-based ADC targeting Tissue Factor (TF). Tissue Factor is clinically validated target overexpressed in a broad range of solid tumors, with limited expression in normal tissues. ADCE-T02 is composed of a next-generation TF targeting antibody conjugated to an exatecan-based T-1000 payload, a clinically validated linker-payload technology. ADCE-T02 shows strong anti-tumor activity in a wide range of solid tumor models and is well tolerated in non-human primate toxicology studies with no evidence of toxicity from earlier generation TF ADCs. A phase 1 clinical trial in advanced solid tumors is ongoing in both Australia and the United States. Australian recruitment is underway and enrollment in the United States will be initiated in the near future [NCT06597721].

ADCE-D01 is a first-in-class ADC targeting uPARAP conjugated to the Topo-1 inhibitor payload P-1021. uPARAP is a novel endocytic ADC target that is overexpressed in tumors of mesenchymal origin, such as sarcomas, while exhibiting restricted expression in normal tissues. ADCE-D01 is composed of a first-in-class uPARAP-targeting antibody conjugated to an optimized, clinically validated linker payload technology, selected for its superior efficacy and tolerability. Preclinically, ADCE-D01 shows strong anti-tumor activity in a range of mesenchymal tumor models including soft tissue sarcoma and is well tolerated in non-human primate toxicology studies with a favorable safety profile and no evidence of target-specific toxicity. A phase 1 clinical trial in advanced soft tissue sarcomas is enrolling in the United States and is under regulatory review in the European Union [NCT06797999].

Dominik Mumberg, PhD, Chief Scientific Officer at Adcendo, said: "We are excited to share the promising preclinical data for ADCE-T02 and ADCE-D01 demonstrating strong anti-tumor activity across a broad range of epithelial and mesenchymal tumor models with highly favourable tolerability profiles. We look forward to advancing both programs in our phase 1 Tiffany-01 and ADCElerate1 trials to help cancer patients in need of novel therapeutic options."

Details of the poster presentations are as follows:

Date & Time: April 29th, 9.00am – 12.00pm CT
Session Title: Antibodies and Antibody-Drug Conjugates
Poster Section: 36

Board Number: 14, Abstract number: 4778
Presentation title: ADCE-T02: A first-in-class topoisomerase-1 inhibitor-based antibody drug conjugate against tissue factor demonstrates excellent preclinical efficacy and tolerability
Authors: T. T. Poulsen1, Y. Zhang2, J. Zhang2, H. Shi2, S. Liu3, X. Meng2, M. Gillberg1, D. Mumberg1
1 Adcendo ApS, Copenhagen, Denmark. 2 Multitude Therapeutics, Shanghai, China. 3 Multitude Therapeutics, Redwood City, CA, USA

Board Number: 20, Abstract number: 4784
Presentation title: ADCE-D01: a first in class antibody-drug conjugate against urokinase plasminogen activator receptor-associated protein (uPARAP) demonstrates excellent preclinical efficacy and tolerability
Authors: J. Wardman1, A. Bie1, C. Côme1, P. Barkholt1, S. van Putten1, C. Løkke1, I. Gregersen1, J. Lange1, M. Gilberg1, K. Bannister1, M. Krogh-Madsen1, T. T. Poulsen1, C. Nielsen1, L. Engelholm2, N. Behrendt2, C. Lynch1, P. Hemmingsen1, D. Mumberg1
1 Adcendo ApS, Copenhagen, Denmark. 2 The Finsen Laboratory, Rigshospitalet/BRIC, Copenhagen University, Copenhagen, Denmark

Abstracts are available in an online itinerary planner (ADCE-T02 abstract; ADCE-D01 abstract), and will be available in an online only supplement to the AACR (Free AACR Whitepaper) journal Cancer Research one month after the conference. Electronic posters will be made available for registered conference participants from April 25th, 2025, through the conference web portal.

About antibody-drug conjugates (ADCs):

ADCs are a class of highly potent biopharmaceutical drug composed of a targeting antibody linked to a biologically active drug or cytotoxic compound. ADCs combine the unique and very sensitive targeting capabilities of antibodies, with the potent effects of the conjugated cytotoxic drugs, allowing sensitive discrimination between healthy and cancer tissues.

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.

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.

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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.

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]):

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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.

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.

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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.