On October 22, 2025 Onco3R Therapeutics, a clinical-stage immunology and oncology biotech company dedicated to transforming patients’ lives with best-in-class medicines, reported that it will present preclinical data from its FGFR3, SMARCA2 and P53 Y220C small molecules programs in 3 posters at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, taking place October 22-26, 2025, in Boston.

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"First generation precision medicines are often suboptimal in the clinic due to low target coverage, off-target toxicity and emergence of resistance. At Onco3R, our vision is to design best-in-class medicines to address the unmet needs left by first generation drugs and unlock the full potential of therapeutic targets" said François Gonzalvez, PhD, CSO and co-Founder of Onco3R Therapeutics.

"We are thrilled to present the first preclinical data from our lead oncology programs FGFR3, SMARCA2 and P53 Y220C. Each program has identified best-in-class molecules which offer the potential to deliver transformational efficacy and improved tolerability for patients. Our FGFR3 and SMARCA2 candidates, G-012 and G-141 respectively, have reached the optimum potency and selectivity profile to mitigate dose-limiting toxicities while maintaining maximum target coverage. This has translated into robust anti-tumor activity in vivo. The poster presentations will highlight data supporting the advancement of these two candidates towards the clinic, as well as the discovery of unique small molecule P53 reactivators."

"These compelling preclinical results further validate our patient-centric drug discovery approach, which integrates deep translational science with rational, structure-based and AI-augmented drug design", Pierre Raboisson, PhD, CEO and co-Founder of Onco3R Therapeutics said. "We look forward to advancing these two candidates and remain on track to initiate IND-enabling studies in mid-2026. The identification of these candidates, alongside the continued clinical progress of our SIK3 inhibitor O3R-5671 in autoimmune indications, reinforces Onco3R’s strong strategic position. With a robust pipeline and clear execution momentum, we are confidently advancing toward our next value-driving milestones."

Presentation details

Title: Discovery of Best-in-Class FGFR3 small molecule inhibitors with high isoform selectivity and activity against gatekeeper mutations

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: Sandrine Vendeville, PhD

Key findings from preclinical studies include:

G-012 demonstrated best-in-class potency and selectivity with favorable drug-like properties.

Based on translational modelling, the compound reached the optimal selectivity against other FGFR isoforms to mitigate off-target toxicity and maintain maximal target coverage.

G-012 showed robust anti-proliferative activity in FGFR3-driven cancer cells and induced significant tumor regression in vivo.

G-012 is currently advancing in 14 days toxicology studies.

IND-enabling studies are anticipated in mid-2026.

Title: Discovery of novel SMARCA2 small molecule inhibitors with best-in-class potency and selectivity for the treatment of SMARCA4-mutant cancers

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: Lijs Beke, PhD

Key findings from preclinical studies include:

G-141 combined best-in-class cellular potency and selectivity to allow optimal target coverage and unlock the full therapeutic potential of SMARCA2 inhibition.

The compound showed synthetic lethality in SMARCA4-deficient cells and induced robust anti-tumor activity in vivo without signs of SMARCA4-related toxicity.

G-141 showed favorable drug-like properties and is currently advancing in 14 days toxicology studies.

IND-enabling studies are anticipated in mid-2026.

Title: Discovery of a Best-in-Class small molecule p53 Y220C reactivator: Breaking through the potency ceiling

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: François Gonzalvez, PhD

Key findings from preclinical studies include:

Onco3R patient-centric drug discovery approach identified unique small molecule P53 reactivators with best-in-class cellular potency.

Onco3R leads exhibit the optimal potency and residence time to induce deep and sustain target engagement and fully unlock the tumor suppressive function of P53 in cells.

This translated into robust anti-proliferative activity in P53 Y220C mutant cancer cell lines (single digit nanomolar IC50s) and tumor regression in a Y220C P53 mutant xenograft model.

Further characterization of the lead candidates is ongoing.

(Press release, Onco3R Therapeutics, OCT 22, 2025, View Source [SID1234656951])

On October 22, 2025 Onco3R Therapeutics, a clinical-stage immunology and oncology biotech company dedicated to transforming patients’ lives with best-in-class medicines, reported that it will present preclinical data from its FGFR3, SMARCA2 and P53 Y220C small molecules programs in 3 posters at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, taking place October 22-26, 2025, in Boston (Press release, Onco3R Therapeutics, OCT 22, 2025, View Source [SID1234656928]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"First generation precision medicines are often suboptimal in the clinic due to low target coverage, off-target toxicity and emergence of resistance. At Onco3R, our vision is to design best-in-class medicines to address the unmet needs left by first generation drugs and unlock the full potential of therapeutic targets" said François Gonzalvez, PhD, CSO and co-Founder of Onco3R Therapeutics.

"We are thrilled to present the first preclinical data from our lead oncology programs FGFR3, SMARCA2 and P53 Y220C. Each program has identified best-in-class molecules which offer the potential to deliver transformational efficacy and improved tolerability for patients. Our FGFR3 and SMARCA2 candidates, G-012 and G-141 respectively, have reached the optimum potency and selectivity profile to mitigate dose-limiting toxicities while maintaining maximum target coverage. This has translated into robust anti-tumor activity in vivo. The poster presentations will highlight data supporting the advancement of these two candidates towards the clinic, as well as the discovery of unique small molecule P53 reactivators."

"These compelling preclinical results further validate our patient-centric drug discovery approach, which integrates deep translational science with rational, structure-based and AI-augmented drug design", Pierre Raboisson, PhD, CEO and co-Founder of Onco3R Therapeutics said. "We look forward to advancing these two candidates and remain on track to initiate IND-enabling studies in mid-2026. The identification of these candidates, alongside the continued clinical progress of our SIK3 inhibitor O3R-5671 in autoimmune indications, reinforces Onco3R’s strong strategic position. With a robust pipeline and clear execution momentum, we are confidently advancing toward our next value-driving milestones."

Presentation details
Title: Discovery of Best-in-Class FGFR3 small molecule inhibitors with high isoform selectivity and activity against gatekeeper mutations

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: Sandrine Vendeville, PhD

Key findings from preclinical studies include:

G-012 demonstrated best-in-class potency and selectivity with favorable drug-like properties.

Based on translational modelling, the compound reached the optimal selectivity against other FGFR isoforms to mitigate off-target toxicity and maintain maximal target coverage.

G-012 showed robust anti-proliferative activity in FGFR3-driven cancer cells and induced significant tumor regression in vivo.

G-012 is currently advancing in 14 days toxicology studies.

IND-enabling studies are anticipated in mid-2026.

Title: Discovery of novel SMARCA2 small molecule inhibitors with best-in-class potency and selectivity for the treatment of SMARCA4-mutant cancers

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: Lijs Beke, PhD

Key findings from preclinical studies include:

G-141 combined best-in-class cellular potency and selectivity to allow optimal target coverage and unlock the full therapeutic potential of SMARCA2 inhibition.

The compound showed synthetic lethality in SMARCA4-deficient cells and induced robust anti-tumor activity in vivo without signs of SMARCA4-related toxicity.

G-141 showed favorable drug-like properties and is currently advancing in 14 days toxicology studies.

IND-enabling studies are anticipated in mid-2026.

Title: Discovery of a Best-in-Class small molecule p53 Y220C reactivator: Breaking through the potency ceiling

Session: Poster Session C

Session Date and Time: Saturday, October 25, 12:30-4pm

Presenting author: François Gonzalvez, PhD

Key findings from preclinical studies include:

Onco3R patient-centric drug discovery approach identified unique small molecule P53 reactivators with best-in-class cellular potency.

Onco3R leads exhibit the optimal potency and residence time to induce deep and sustain target engagement and fully unlock the tumor suppressive function of P53 in cells.

This translated into robust anti-proliferative activity in P53 Y220C mutant cancer cell lines (single digit nanomolar IC50s) and tumor regression in a Y220C P53 mutant xenograft model.

Further characterization of the lead candidates is ongoing.

On October 22, 2025 GemPharmatech, a global leader in preclinical research solutions and genetically-engineered mouse models, reported a collaboration with Memorial Sloan Kettering Cancer Center (MSK) to accelerate the discovery of new therapeutic antibodies.

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Through the non-exclusive agreement, researchers at Memorial Sloan Kettering will have access to GemPharmatech’s extensive preclinical service capabilities and resources, utilizing the NeoMab platform — a next-generation transgenic mouse model designed for rapid, efficient discovery of fully human therapeutic antibodies. The collaboration will help scientists at Memorial Sloan Kettering who are already tapping a wide range of leading-edge preclinical resources to further accelerate the development of fully human therapeutic antibodies against high-value targets and address urgent, unmet needs in cancer treatment.

"Our mission has always been to enable transformative biomedical research through innovative mouse models and technologies," said Dr. Xiang Gao, founder of GemPharmatech. "Collaborating with MSK on antibody discovery represents a powerful opportunity to combine scientific excellence with cutting-edge mouse models such as NeoMab. Together, we can accelerate the development of novel therapeutics that address some of the most urgent challenges in cancer treatment."

Part of the world’s largest library of genetically engineered mouse models (GEMMs), the immunoglobulin gene-humanized mouse model- NeoMab, carries the full variable gene repertoire of human heavy and kappa light chains, and regulatory elements, in a BALB/c background. This innovative design enables the rapid generation of diverse, high-affinity fully human antibodies without the need for time-consuming sequence humanization steps. By skipping this process, the collaboration aims to shorten antibody development timelines and reduce immunogenicity risks.

"This collaboration underscores our mission to provide the global research community with advanced platforms to accelerate innovations in drug discovery," said Dr. Brandy Wilkinson, Chief Executive Officer of GemPharmatech. "We are excited to be supporting MSK’s pioneering work in oncology research with the NeoMab platform. It is an honor to contribute to their scientific mission and to help advance antibody programs with the potential to transform therapies and improve patient outcomes worldwide."

(Press release, Memorial Sloan-Kettering Cancer Center, OCT 22, 2025, View Source [SID1234656915])

On October 22, 2025 Kyowa Kirin International (KKI), a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin), a Japan-based global specialty pharmaceutical company, and the University Hospitals Birmingham NHS Foundation Trust, reported new overall survival (OS) insights from the PROCLIPI (PROgnostic Cutaneous Lymphoma International Prognostic Index) Study aligning with previous findings that suggest mogamulizumab may offer meaningful OS benefit for patients living with mycosis fungoides (MF) and Sézary syndrome (SS), two rare conditions and subtypes of cutaneous T-cell lymphoma (CTCL).

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Key findings2:

For patients with advanced-stage MF and SS (n=371), the median overall survival from the onset of treatment was higher for patients treated with mogamulizumab (n=72) at 64 months compared with those not receiving mogamulizumab (n=175) at 54 months (p<0.01).
Patients treated with mogamulizumab showed a trend toward improved OS compared to those not receiving mogamulizumab across risk-stratified groups according to the new Cutaneous Lymphoma International Prognostic Index (CLIPI).
For a subset of patients with SS (n=96), the median OS is around 6.5 years for patients treated with mogamulizumab (n=46) compared with around 3 years for systemic treatment but not mogamulizumab (n=50) (p<0.01).
This research was presented as part of the Scientific Proceedings at this year’s European Organisation for Research and Treatment of Cancer Cutaneous Lymphoma Tumour Group (EORTC-CLTG) Annual Meeting in Athens, Greece.2 The data contributes to a growing body of evidence suggesting potential OS benefits with mogamulizumab, 4,5 highlighting the importance of collaboration across the CTCL community in advancing understanding of survival outcomes and helping to shape the future of patient care.

"The PROCLIPI Study demonstrates the power of global collaboration in rare diseases. By bringing together data from across the world, we can generate insights that simply wouldn’t be possible in isolation," says Professor Julia Scarisbrick, Chief Investigator of the PROCLIPI Study and Honorary Professor of Dermatology, University Hospitals Birmingham NHS Foundation Trust. "We are proud to coordinate this initiative as we’re working to build rigorous scientific evidence while giving patients and their families a better understanding of what long-term survival looks like."

The PROCLIPI Study, now in its tenth year, aims to develop prognostic indices for MF and SS. The study collects comprehensive data, including clinical, haematological, pathological, imaging, treatment with responses, quality of life and survival data.1 Recently, a new prognostic index (CLIPI) for advanced CTCL was published to enable precise patient risk stratification.6

"For those of us in the CTCL community, survival isn’t just about numbers on a chart – it’s about being able to spend more time with our families, plan for the future, and live life with dignity", says Susan Thornton, CEO, Cutaneous Lymphoma Foundation. "That’s why initiatives like PROCLIPI are important, as the data collected reflects real lived experiences and can help inform future improvements in care for patients."

The independent international PROCLIPI Study aligns with Kyowa Kirin International’s commitment to generating real-world insights that advance the understanding of CTCL and turn these clinical insights into meaningful improvements in access, policy, and patient care.

Cutaneous T-cell lymphoma is a rare disease7, and innovation in this environment can be challenging due to the rarity of this disease.8 Yet despite these challenges, mounting evidence continues to emerge4,5 to help strengthen our collective understanding of potential treatment benefits.

"These insights into improved overall survival for patients living with CTCL mark an important step forward, providing a stronger clinical evidence base and reinforcing the value of international networks in rare disease research," says Dr Nick Kronfeld, Head of Medical Affairs, Kyowa Kirin International. "By working alongside scientific and patient communities in CTCL, we can gain a better understanding of real-world outcomes, enabling us to bring life-changing value to patients and families, not just today but over the long term."

Note to editors

The OS data from the PROCLIPI Study was presented as an oral presentation during the EORTC CTCL Annual Group Meeting 2025.2 KKI did not sponsor the PROCLIPI study or OS analysis.
The median OS for patients (n=72) receiving mogamulizumab compared to those not receiving mogamulizumab (n=175) was 64 months versus 54 months.2
Median OS data amongst risk stratifications according to CLIPI: 2,6
Low-risk: 67 months vs. ‘not reached’ for mogamulizumab versus those who had not received mogamulizumab
Medium-risk: 64 vs. 48 months for mogamulizumab versus those who had not received mogamulizumab
High-risk: 26 vs. 13 months for mogamulizumab and for those who had not received mogamulizumab

(Press release, Kyowa Hakko Kirin, OCT 22, 2025, View Source [SID1234656914])

On October 22, 2025 Charles River Laboratories International, Inc. (NYSE: CRL) and The Francis Crick Institute (Crick) reported a new collaboration around Antibody-Drug Conjugate (ADC) drug discovery and development, leveraging combined strengths to accelerate the development and delivery of next-generation targeted therapies.

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This agreement leverages an integrated approach to streamline discovery and characterization—bringing promising ADC candidates to the clinic faster and more efficiently. This end-to-end approach—spanning antibody discovery, conjugation, in vitro profiling, and preclinical studies—will be fully integrated and managed jointly by Charles River and the Crick to minimize timelines and maximize efficiency.

"This integrated ADC development agreement represents a powerful synergy between cutting-edge antibody discovery and rigorous safety profiling," said Justin Bryans, PhD, Chief Scientific Officer, Charles River. "Together, we’re enabling the development of smarter, safer therapeutics that are more likely to succeed in the clinic—ultimately supporting the mission of delivering life-changing therapies to patients."

This collaboration will feature antibody generation leveraging phage display libraries, ensuring high-affinity, target-specific antibodies as the foundation for ADC development. It will also utilize Charles River’s advanced Retrogenix platform, an in vitro profiling technology designed to evaluate off-target interactions and enhance safety and therapeutic index early in the development process.

David Allen, Director of Translation at the Crick, added, "This collaboration marks a powerful convergence of innovation and execution. Working together will allow us to translate our research discoveries into new treatments at a scale and speed that simply wouldn’t be possible alone."

(Press release, Charles River Laboratories, OCT 22, 2025, View Source [SID1234656913])