On October 28, 2024 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported the clearance of an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1 clinical trial to evaluate IDE275 (GSK959), a potential first-in-class and best-in-class Werner Helicase (WRN) inhibitor (Press release, Ideaya Biosciences, OCT 28, 2024, View Source [SID1234647459]). IDE275 (GSK959) has demonstrated robust and selective synthetic lethality preclinically in the high microsatellite instability (MSI-High) biomarker setting, and the Phase 1 clinical trial will enroll patients having tumors characterized by MSI-High.

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"IDE275 represents IDEAYA’s fifth potential first-in-class clinical program in our precision medicine oncology pipeline and has a potentially differentiated best-in-class profile that we are targeting to present at a future medical conference with GSK. The robust preclinical efficacy observed by IDE275 selectively in the MSH-High biomarker setting, including monotherapy regressions, provides a double-digit % prevalence target patient population across several major solid tumor types, including endometrial, colorectal and gastric cancer," added Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

IDE275 (GSK959) is a potential first-in-class small molecule inhibitor of Werner Helicase that was discovered by IDEAYA in collaboration with GSK. In preclinical studies, IDE275 has demonstrated robust and selective synthetic lethality in the MSI-High biomarker setting, including single-agent tumor regressions in-vivo in MSI-High CDX and PDX models derived from colorectal, endometrial and gastric cancers. Initiation of the Phase 1 trial for IDE275 is projected in the fourth quarter of 2024. GSK is the sponsor of the IND application and plans to develop IDE275 (GSK959) as both a monotherapy agent and in combination with a PD-1 inhibitor in a Phase 1 clinical trial for patients having MSI-High tumors. The percent prevalence of MSI-High in solid tumors, including endometrial, colorectal, and gastric cancers, has been reported at approximately 31%, 20%, and 19%, respectively (JCO Precision Oncology, September 2017).

GSK is responsible for 80% of global research and development costs for IDE275 (GSK959) and IDEAYA is responsible for 20% of such costs. IDEAYA is eligible to receive a $7 million milestone payment upon acceptance of the IND by the U.S. Food and Drug Administration (FDA), and a potential additional $10 million milestone payment upon initiation of Phase 1 clinical dose expansion. IDEAYA may potentially also receive up to $465 million in further later-stage development and regulatory milestones. Upon potential commercialization, IDEAYA will be eligible to receive up to $475 million of commercial milestones 50% of U.S. net profits and tiered royalties on global non-U.S. net sales of IDE275 (GSK959) – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.

On October 28, 2024 BioVaxys Technology Corp. (CSE: BIOV) (FRA: 5LB) (OTCQB: BVAXF) ("BioVaxys" or the "Company") a clinical-stage company developing a portfolio of immune-educating therapies based on its novel DPX platform to treat a range of cancers, infectious diseases, and immune modulated disorders reported that Chief Executive Officer James Passin has been invited to present live at the Emerging Growth Conference on October 30th, 2024 at 9:40 AM EST (Press release, BioVaxys Technology, OCT 28, 2024, View Source [SID1234647458]). This live interactive online event will give existing shareholders and the investment community the opportunity to interact with the Company’s CEO in real time.

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Please submit your questions in advance to [email protected] or ask your questions during the event.

If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available on EmergingGrowth.com and on the Emerging Growth YouTube Channel, View Source We will release a link to that after the event.

On October 28, 2024 Chime Biologics, a leading global CDMO that enables its partners’ success in biologics, reported a strategic collaboration with MedPacto, a South Korean clinically staged company listed on KOSDAQ (235980) (Press release, MedPacto, OCT 28, 2024, View Source [SID1234647457]).

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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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MedPacto, known for its focus on cancer therapeutics, will leverage Chime Biologics’ expertise in biologics development to streamline the process from tech transfer to cGMP clinical manufacturing for Investigational New Drug (IND) submissions. Under the agreement, Chime Biologics will advance the Chemistry, Manufacturing, and Controls (CMC) process development for the multiple preclinical drug candidates.

Dr. Jungwon Woo, President of MedPacto, stated," We are highly impressed by Chime Biologics’ development and manufacture expertise, which is crucial helping us expand into major global markets. Their ability to meet international standards will greatly support our efforts to advance our assets into a new stage. This collaboration is a key step forward, and we look forward to a successful and long-term partnership."

Dr. Jimmy Wei, President of Chime Biologics, added, "We are pleased to announce our strategic collaboration with MedPacto, a respected KOSDAQ-listed company with a strong portfolio of first-in-class assets. This partnership marks the beginning of a long-term relationship aimed at advancing pharmaceutical innovation. It aligns with our growth strategy in South Korea and aligns with our commitment to the region’s biopharmaceutical landscape. We look forward to working closely with MedPacto and contributing to the success of their innovative pipeline."

Chime Biologics is expanding its manufacturing capacity to 100,000 liters to solidify its position as a leading global CDMO. As part of this strategy, the company is actively entering the South Korean market and has signed agreements with several South Korean biotech companies to provide development and manufacturing services for antibody drugs and biosimilars.

On October 28, 2024 Oncotelic Therapeutics, Inc (OTCQB:OTLC) reported its fourth publication of its TGFB2 series (Press release, Oncotelic, OCT 28, 2024, View Source [SID1234647454]). Clinical data demonstrated that pancreatic cancer patients with high expression levels of either TGFB2, IRF9, or IFI27 showed improvement of median overall survival from 16-20 months to 72 months for patients with low levels of expression for both TGFB2 and either IRF9 or IFI27 validating that knockdown of IRF9 and IFI27 as therapeutic targets for TGFB2 therapeutics. View Source

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The publications of this series are available at Pubmed as follow:

1) TGFB2 mRNA Levels Prognostically Interact with Interferon-Alpha Receptor Activation of IRF9 and IFI27, and an Immune Checkpoint LGALS9 to Impact Overall Survival in Pancreatic Ductal Adenocarcinoma. Qazi S, Trieu V. Int J Mol Sci. 2024 Oct 18;25(20):11221. doi: 10.3390/ijms252011221. PMID: 39457004

2) Transforming Growth Factor Beta 2 (TGFB2) mRNA Levels, in Conjunction with Interferon-Gamma Receptor Activation of Interferon Regulatory Factor 5 (IRF5) and Expression of CD276/B7-H3, Are Therapeutically Targetable Negative Prognostic Markers in Low-Grade Gliomas. Trieu V, Maida AE, Qazi S. Cancers (Basel). 2024 Mar 19;16(6):1202. doi: 10.3390/cancers16061202. PMID: 38539537 Free PMC article.

3) Transforming Growth Factor Beta 2 (TGFB2) and Interferon Gamma Receptor 2 (IFNGR2) mRNA Levels in the Brainstem Tumor Microenvironment (TME) Significantly Impact Overall Survival in Pediatric DMG Patients. Qazi S, Talebi Z, Trieu V. Biomedicines. 2024 Jan 15;12(1):191. doi: 10.3390/biomedicines12010191. PMID: 38255296 Free PMC article.

4) High Intra-Tumor Transforming Growth Factor Beta 2 Level as a Predictor of Poor Treatment Outcomes in Pediatric Diffuse Intrinsic Pontine Glioma. Uckun FM, Qazi S, Trieu V. Cancers (Basel). 2023 Mar 9;15(6):1676. doi: 10.3390/cancers15061676. PMID: 36980562 Free PMC article.

Additional information on our current phase 3 can be found at: View Source

On October 28, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that new long-term durability of response results from the Phase 3 ENVISION study of investigational drug UGN-102 in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC) were published online in the Journal of Urology (Press release, UroGen Pharma, OCT 28, 2024, View Source [SID1234647453]).

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In ENVISION, UGN-102 treatment was associated with an unprecedented 82.3% (95% CI, 75.9%, 87.1%) 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved complete response (CR) at three months after the first instillation of investigational drug UGN-102 (mitomycin) for intravesical solution. The DOR Kaplan-Meier estimates at 15 months (n=43) and 18 months (n=9) after 3-month CR were both 80.9% (95% CI, 73.9%, 86.2%). The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102.

"These data demonstrate that treatment with UGN-102 results in a clinically meaningful CR rate and that the durability of the response in patients with LG-IR-NMIBC is robust," said Sandip Prasad, MD, M.Phil., Director of Genitourinary Surgical Oncology, Morristown Medical Center/Atlantic Health System, NJ and Principal Investigator of the ENVISION trial. "This study adds to the mounting evidence supporting UGN-102 as a potentially valuable treatment option for patients with recurrent LG-IR-NMIBC."

UroGen initiated the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 as a treatment for LG-IR-NMIBC in January 2024 and completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 with a PDUFA goal date of June 13, 2025.

Liz Barrett, President and CEO of UroGen, emphasized the significance of the DOR findings from the ENVISION study, stating, "LG-IR-NMIBC patients are typically elderly and currently treated with repeated surgeries under general anesthesia that are associated with physical and quality of life detriments. These new results continue to strengthen UGN-102’s potential as a treatment option for these patients that extends their recurrence-free interval. The highly recurrent nature of LG-IR-NMIBC underscores the urgent need for innovative options for patients facing this challenging disease."

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were typically mild-to-moderate in severity and resolved or resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S., bladder cancer is the second most common urologic cancer in men. LG-IR- NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).