On June 2, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported results from the ENVISION and ATLAS clinical studies exploring investigational therapy UGN-102 (mitomycin) for intravesical solution for the treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (Press release, UroGen Pharma, JUN 2, 2025, View Source [SID1234653626]). The data are featured at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL.

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UGN-102 data featured at ASCO (Free ASCO Whitepaper):

Duration of Response (DOR) Following Treatment with UGN-102 in Patients with Recurrent, Low-Grade, Intermediate-Risk, Non-Muscle Invasive, Bladder Cancer: 18-Month DOR Data from the Phase 3 ENVISION Trial: Abstract #4598, Poster #398
Impact of Tumor Burden or Focality in Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer on Response to Treatment with UGN-102: A Substudy of the Phase 3 ENVISION Trial: Abstract #4597, Poster #397
Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer with UGN-102: Results of the Phase 3 ATLAS and ENVISION Studies. Published Abstract 501822
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 recurrent 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 administered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the submission of the rolling new drug application (NDA) for UGN-102 in August 2024, ahead of schedule. The U.S. Food and Drug Administration (FDA) accepted the NDA for UGN-102 and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025. On May 21, 2025, an Oncologic Drugs Advisory Committee (ODAC) of the FDA voted 4 to 5 that the benefit/risk of UGN-102 (mitomycin) for intravesical solution was favorable for the treatment of recurrent LG-IR-NMIBC. See "Forward-Looking Statements" below.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. 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. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

About ENVISION

The Phase 3 ENVISION trial is an ongoing, 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 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluates 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).

On June 2, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA"), a Phase 3 immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported that Moffitt Cancer Center presented a Trial in Progress poster of the Company’s planned Phase 3 accelerated approval trial at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 1, 2025, in Chicago, Illinois (Press release, TuHURA Biosciences, JUN 2, 2025, View Source [SID1234653625]).

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The Company’s poster, titled "Multicenter, randomized, double-blinded, placebo-controlled trial of IFx-Hu2.0 (IFx) as adjunctive therapy with pembrolizumab (pembro) in checkpoint inhibitor (CPI)-naïve patients with advanced or metastatic Merkel cell carcinoma (MCC)" details the Company’s Phase 3 accelerated approval trial design of IFx-Hu2.0, its novel innate immune agonist. The Trial in Progress poster was presented by Andrew Brohl, M.D., Medical Oncologist at Moffitt Cancer Center, and highlighted the importance of innate immune system activation in Merkel cell carcinoma patients with primary resistance to checkpoint inhibitors (CPIs).

"Merkel cell carcinoma is a rare and aggressive tumor type. While checkpoint inhibitor therapy has markedly improved the outcome for patients with advanced or metastatic MCC, unfortunately for patients who don’t respond to first line checkpoint inhibitor therapy the survival is poor at less than 30 months.1 Based on the results from our Phase 1 clinical trials, IFx-Hu2.0 intralesional administration has demonstrated it can activate an innate immune response, resulting in the production and activation of tumor specific B cells and T cells, to overcome the primary CPI resistance in both advanced or metastatic MCC or melanoma," stated James Bianco, M.D., President and Chief Executive Officer of TuHURA Biosciences. "We believe we have provided data requested by the FDA that addresses the requirements listed in the partial clinical hold letter to allow us to initiate our Phase 3 accelerated approval trial this month. The FDA was constructive in the trial design, which can potentially satisfy both the requirements for accelerated and regular approval without the requirements for a post approval confirmatory trial. They continue to work with us under the SPA Agreement in preparing the trial’s initiation."

In the Phase 1b trial of IFx-Hu2.0, MCC among patients who progressed on pembrolizumab (anti-PD-1) or avelumab (anti-PDL-1) therapy, weekly administration of IFx-Hu2.0 for up to 3 doses followed by rechallenge with anti-PD(L)-1 therapy, demonstrated an overall response rate of 63% (2CR, 5 PR) with duration of responses ranging from 6 to 33 months with 5 ongoing responses as of last follow-up in June 2024.

The Company’s Phase 3 accelerated approval trial of adjunctive IFx-Hu2.0, to be conducted under a SPA agreement with the U.S. FDA, will evaluate IFx-Hu2.0 (0.1 mg) as an adjunctive therapy administered weekly for three weeks concurrent to pembrolizumab (200 mg) Q3W, compared to the same pembrolizumab regimen plus placebo. The pivotal trial is expected to enroll 118 CPI-naïve patients with advanced or metastatic MCC across approximately 22 to 25 U.S. sites. Trial participants will be randomized on a 1:1 basis and receive CPI therapy for up to two years, or until disease progression or CPI related toxicities. The primary endpoint for the trial is overall response rate (ORR) with a key secondary endpoint of progression free survival (PFS). Other secondary endpoints are safety, duration of response, and overall survival.

The Trial in Progress poster is available on the Scientific Publications page of TuHURA’s website.

On June 2, 2025 TG Therapeutics, Inc. (NASDAQ: TGTX) reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will participate in the Jefferies Global Healthcare Conference, being held at the Marriott Marquis, in New York City on June 3-5, 2025 (Press release, TG Therapeutics, JUN 2, 2025, View Source [SID1234653624]). The fireside chat is scheduled to take place on Wednesday, June 4, 2025, at 12:50 PM ET.

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A live webcast of the fireside chat will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source

On June 2, 2025 Taiho Pharmaceutical Co., Ltd. (hereinafter "Taiho Pharmaceutical") and Benz Sciences Inc. (hereinafter "Benz Sciences") reported that the two companies have entered into a license agreement for TAS1440, a LSD1 (lysine-specific demethylase 1) inhibitor discovered by Taiho Pharmaceutical (Press release, Taiho, JUN 2, 2025, View Source [SID1234653623]).

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Under the terms of agreement, Benz Sciences will obtain exclusive worldwide rights to develop, manufacture and commercialize TAS1440 (new development code SFG-03). Taiho Pharmaceutical will receive from Benz Sciences an upfront payment, milestone payments based on development progress and sales, and royalties.

TAS1440 is an orally administrable, small-molecule compound, reversible inhibitor of LSD1, an enzyme critical for hematopoiesis. It has been reported that the LSD1 gene is overexpressed in myeloproliferative neoplasms,1 diseases characterized by the excessive production of blood cells due to acquired genetic mutations in hematopoietic stem cells. Benz Sciences will undertake preparations in the United States for the Phase 1b/2 trial of TAS1440 for the treatment of myeloproliferative neoplasms.

About TAS1440
TAS1440 is a histone-competitive LSD1 inhibitor that binds to the histone H3 binding site of LSD1 to reversibly inhibit LSD1. LSD1, a critical enzyme that regulates the proliferation of hematopoietic stem cells and the maturation of progenitor cells,2 is expected to be a therapeutic target for diseases such as acute myeloid leukemia (AML) and myeloproliferative neoplasms. TAS1440 has completed a Phase 1 trial targeting AML in the United States, and the results of this trial are undergoing analysis at this time.

On June 2, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported preclinical efficacy of SLS009 (tambiciclib) in ASXL1 mutated colorectal cancer lines. The data are featured in a presentation, entitled "In vitro efficacy of CDK9 inhibitor tambiciclib (SLS009) in ASXL1 mutated colorectal cancer cell lines" at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30- June 3, 2025, in Chicago, Illinois (Press release, Sellas Life Sciences, JUN 2, 2025, View Source [SID1234653622]).

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In a panel of cell lines, SLS009 demonstrated potent anti-proliferative activity:

In 50% (4/8) of ASXL1 mutant cell lines showed an IC50<100 nM, compared to 0% (0/4) of ASXL1 wild-type lines
Among cell lines harboring ASXL1 frameshift mutations (FSMs), 75% (3/4) responded with IC50 <100 nM versus only 12.5% (1/8) in cell lines without FSMs
All cell lines (3/3) with ASXL1 FSMs in the 637-638 protein region responded to treatment with SLS009
In cell lines with IC50 <100 nM, 75% (3/4) also demonstrated IC99 values below 100 nM, indicating steep dose response curve
Importantly, effective concentrations were significantly lower than those achieved in patients treated at the recommended phase 2 dose determined to be safe, suggesting a broad therapeutic window.
"These results provide strong rationale for continued advancement of SLS009 as a potential treatment for ASXL1-mutated cancers," said Dr. Dragan Cicic, Senior Vice President, Chief Development Officer at SELLAS. "The ability to selectively target ASXL1-driven tumors at concentrations well below the known safety threshold opens the door for tolerable and effective therapy. Based on the findings, we believe that ASXL1 mutation status could serve as a potential biomarker for response to SLS009 inhibition, which may allow us to further refine patient selection and improve outcomes. We look forward to presenting these results at ASCO (Free ASCO Whitepaper)."

Poster presentation details:

Title: In vitro efficacy of CDK9 inhibitor tambiciclib (SLS009) in ASXL1 mutated colorectal cancer cell lines
Session Date and Time: Monday, June 2, 2025, 1:30 PM-4:30 PM CDT
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Location: Hall A – Posters and Exhibits
Abstract #: 3121
Poster Board #: 436

SLS009 is currently being investigated in a Phase 2 open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine including AML patients with ASXL1 mutations. Initial clinical safety and efficacy data are available. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit clinicaltrial.gov identifier NCT04588922.