On December 3, 2024 Congruence Therapeutics reported that it has entered into a research collaboration agreement with Ono Pharmaceutical Co., Ltd. (Ono) to discover novel drug candidates leveraging Congruence’s proprietary drug discovery platform, Revenir (Press release, Ono, DEC 3, 2024, View Source [SID1234648771]). Congruence will apply its technology and expertise toward multiple cancer protein targets. Once the discovery effort advances to a prespecified stage, Ono has the option to acquire exclusive worldwide rights to further develop and commercialize small molecule correctors generated during the collaboration.

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"We believe that this collaboration with Congruence may help generating novel small molecule correctors for validated targets in the oncology area by leveraging their own technologies in protein dynamics and computational biology, leading to our development pipeline," said Seishi Katsumata, PhD, Corporate Officer / Executive Director, Discovery & Research of Ono. "We will be committed to delivering innovative new drugs to cancer patients as soon as possible."

The collaboration will leverage Congruence’s purpose-built computational drug discovery engine called Revenir, which captures the biophysical changes caused by mutations in proteins. By examining surface features and numerous biophysical descriptors of both the mutated and wild-type proteins, Congruence can derive novel insights regarding protein defects and how to correct them. Congruence’s current pipeline consists of both first-in-class and best-in-class potential therapeutic candidates that address significant unmet medical need in a number of high value indications.

"Congruence is thrilled to partner with Ono, which has established itself as a global leader in drug development, particularly in the oncology space. We believe that our Revenir platform and capabilities in protein dynamics will accelerate the discovery of novel therapies for compelling targets of interest to both companies," said Sharath Hegde PhD, Chief Scientific Officer of Congruence.

Under the terms of the collaboration, Congruence will receive an undisclosed upfront payment and is eligible to receive milestone payments upon the achievement of certain discovery, development, approval and sales events – as well as tiered royalties based on annual net sales of related products. Ono will additionally reimburse Congruence for the research costs it incurs in connection with the collaboration. Ono has the option to take an exclusive worldwide license to development candidates discovered under the collaboration.

About Revenir Drug Discovery Platform

Revenir, Congruence’s proprietary computational drug discovery platform, captures the dynamic biophysical changes caused by mutations in proteins, offering unique insights into protein defects and their correction. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir predicts small molecule induced correction of the underlying defect.

On December 3, 2024 SciTech Development, a clinical-stage pharmaceutical company focused on advancements in cancer treatment, reported promising updates from its clinical trial of ST-001 nanoFenretinide (ST-001) (Press release, SciTech Development, DEC 3, 2024, View Source [SID1234648770]).

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Preliminary Trial Findings
Preliminary data from the accelerated Phase 1a trial in patients with Cutaneous T-cell Lymphoma indicates favorable clinical improvements to ST-001 treatment, including earlier-than-anticipated stable disease and partial responses – one initial and one confirmed.

In addition, results from patients treated with ST-001 demonstrate that the advanced nanoparticle delivery system successfully achieves the desired pharmacokinetic profile. It enables rapid and targeted delivery of fenretinide directly to tissues while minimizing side effects and toxicity. These findings exceed expectations and mark a significant milestone in the continued development of ST-001.

ST-001 Clinical Trial Information
SciTech’s initial trial is targeting T-cell non-Hodgkin lymphoma (T-cell NHL) indications including all sub-types of Cutaneous T-cell lymphoma (CTCL), Mycosis Fungoides (MF), Sézary Syndrome (SS), Angioimmunoblastic T-cell lymphoma (AITL), and other types of systemic T-cell lymphomas. The trial is activated and enrolling at 8 prestigious cancer institutions across the U.S., supported by world-renowned scientific advisors and principal investigators specializing in T-cell NHL. View Source

"The early findings from the Phase 1a trial of ST-001 nanoFenretinide are incredibly promising. Achieving a partial response at this stage is a remarkable step forward and highlights the potential of this innovative treatment to address significant gaps in care for patients with Cutaneous T-cell Lymphoma (CTCL)," said Dr. Larisa Geskin, Director of the Comprehensive Cutaneous Oncology Center at Columbia University. "Fenretinide has shown an excellent safety profile so far, with minimal side effects, including no reported thyroid abnormalities or significant lipid issues. If responses continue to improve as doses are escalated, this trial could mark a turning point in CTCL therapy and bring renewed hope to patients."

"We are extremely encouraged by the preliminary data from the ST-001 trial," said Earle Holsapple, CEO of SciTech Development. "The findings to date strengthen our resolve to gather additional data and advance to the next phase of the trial, as we work toward bringing a new therapeutic option to oncology patients."

Trial Design and Advancements
The FDA approved the Phase 1a accelerated dose-escalation design based on extensive research, prior clinical testing, and the safety profile of fenretinide in more than 3,000 patients. This accelerated portion of the trial is designed to achieve early clinical development objectives including confirming safety, assessing pharmacokinetics, pharmacodynamics, and determine the optimal therapeutic dosing in patients with T-cell NHL.

Upcoming Trial Milestones
The accelerated Phase 1a portion is nearing completion. SciTech is preparing to progress to the next phase of the T-cell NHL study and plans to enroll approximately 45 additional patients. An upcoming clinical trial for ST-001 targeting small cell lung cancer (SCLC) is anticipated to commence in Q2 2025.

About ST-001
SciTech’s lead drug candidate, ST-001 nanoFenretinide, is a patented nanoparticle formulation of fenretinide and biocompatible phospholipids, allowing for the rapid infusion (IV) of high-dose fenretinide. ST-001 solves bioavailability challenges, avoids triglyceride toxicity of prior formulations, and optimizes therapeutic efficacy. ST-001 represents a novel therapeutic approach for targeting difficult-to-treat cancers, utilizing patented methods to improve drug safety and efficacy profiles while optimizing treatment outcomes.

On December 3, 2024 Duality Biologics ("DualityBio"), a clinical-stage biotech company focusing on the discovery and development of next-generation antibody-drug conjugate (ADC) therapeutics, reported that it has entered into an exclusive option agreement with GSK for a potentially best-in-class ADC candidate, DB-1324 (Press release, DualityBio, DEC 3, 2024, View Source [SID1234648769]). Under the agreement, DualityBio will grant GSK an exclusive option to obtain a license to develop and commercialize DB-1324 worldwide (excluding mainland China, Hong Kong, and Macau) (the "Option").

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DB-1324 is an ADC candidate built with DualityBio’s proprietary and clinically validated Duality Immune Toxin Antibody Conjugate (DITAC) platform. The molecule, currently in preclinical development, leverages DualityBio’s DITAC platform against a gastrointestinal (GI) cancer target. There is high unmet medical need for patients with GI cancer as it represents 35% of all cancer-related deaths and approximately 26% of the global cancer incidence.[1] This ADC molecule has the potential to unlock multiple combination therapy opportunities to strategically complement GSK’s oncology portfolio.

Under the terms of the agreement, GSK will pay $30 million upfront and additional pre-option milestone payments to obtain an exclusive option for exclusive worldwide rights for DualityBio’s ADC (excluding China’s mainland, Hong Kong, and Macau). Upon GSK exercising the Option, DualityBio will receive an option exercise fee as well as potential development, regulatory and commercial milestone payments totalling up to $975 million. Upon commercialisation, GSK will pay tiered royalties on DB-1324’s global net sales outside mainland China, Hong Kong, and Macau. GSK will receive royalties on net sales in mainland China, Hong Kong and Macau.

Hesham Abdullah, SVP, Global Head Oncology, R&D, GSK, said: "GSK has built a portfolio of novel antibody-drug conjugates underpinned by our deep expertise in tumour cell-targeting mechanisms. Given the unique ability of ADCs to address certain targets on tumour cells while sparing healthy ones, we are confident in our strategic focus on this modality as it could advance new therapeutic treatments for the most challenging tumour types."

John Zhu, Chief Executive Officer of Duality Biologics, said: "DualityBio is dedicated to becoming a leading global next-generation ADC company. We are very glad to have entered into this agreement with GSK. Through this collaboration, we will work together to advance our innovative ADC program in gastrointestinal cancer to address unmet medical needs. DualityBio’s unique and validated ADC technology platform can continue to empower more partners around the world and provide innovative treatment options for global patients."

On December 3, 2024 Medigene AG (Medigene or the "Company", FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, reported a USD $1 million milestonepayment from Regeneron Pharmaceuticals, Inc. was triggered (Press release, MediGene, DEC 3, 2024, View Source [SID1234648766]). Regeneron purchased the MAGE-A4-TCR program as part of its acquisition of2seventy bio Inc.’s pre-clinical and clinical oncology and autoimmune cell therapy pipeline, which closed in April 2024. The payment was triggered by a development milestone for a trial in China led by JW Therapeutics (also a prior collaborator of 2seventy bio, Inc.) of Regeneron’s MAGE-A4 cell therapy, which contains a Medigene generated T cell receptor (TCR) targeting MAGE-A4.

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"We are excited that this program is moving forward, incorporating our TCR directed at MAGE-A4 into the clinic and potentially providing the first clinical validation of our unique TCR discovery and generation capabilities and our End-to-End Platform technologies," said Dr. Selwyn Ho, Chief Executive Officer of Medigene. "We look forward to the results of this trial and remain committed to delivering best-in-class, differentiated T cell receptors for use in multiple TCR-guided therapies for patients with solid tumors."

This payment has been included in the current financial guidance provided by the Company, and therefore confirmation of this milestone does not change 2024 guidance.

On December 3, 2024 Gilead Sciences, Inc. (Nasdaq: GILD) and Tubulis reported that they have entered into an exclusive option and license agreement to discover and develop an antibody-drug conjugate (ADC) against a solid tumor target (Press release, Gilead Sciences, DEC 3, 2024, View Source [SID1234648765]).

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Through this agreement, Gilead will gain access to Tubulis’ proprietary Tubutecan and Alco5 platforms. The companies will collaborate to select the best technology to utilize, with Tubulis leading discovery and development efforts to design a topoisomerase I inhibitor-based ADC candidate with superior biophysical properties and stability to address current treatment challenges such as durability and off-target toxicity.

"As we expand our oncology portfolio to address the greatest gaps in care, accessing novel technologies is critical to advancing our pipeline," said Flavius Martin, MD, Executive Vice President, Research, Gilead Sciences. "With Gilead’s ongoing focus on innovating with next-generation therapies and combinations, we are excited to partner with Tubulis to explore a range of solutions that may help increase the therapeutic value of the ADC modality."

"Gilead has established a long track record of developing drugs that provide a significant step-up in therapeutic value, making them a great collaborator for leveraging our technology platforms, in line with our vision of fundamentally changing the ADC landscape," said Dominik Schumacher, PhD, CEO and co-founder of Tubulis. "Tubulis remains primarily focused on driving value through our own clinical development programs while selectively building partnerships with leaders in the industry."

Terms of the Agreement

Under the terms of the agreement, Tubulis will receive an upfront payment of $20 million and, if Gilead exercises its option, a separate option exercise fee of $30 million. In addition, Tubulis will be eligible for development and commercialization milestone payments totaling up to $415 million, plus mid-single to low double-digit tiered royalties on sales of marketed products resulting from the collaboration. Tubulis will lead early-stage research and development activities for the ADC program. If Gilead exercises its option to exclusively license the program, Gilead will be responsible for further development and commercialization activities for all products resulting from the collaboration.

Gilead does not exclude acquired IPR&D expenses from its non-GAAP financial measures. This transaction with Tubulis is expected to reduce Gilead’s GAAP and non-GAAP 2024 EPS by approximately $0.01.