On October 2, 2024 UroGen Pharma Ltd, a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the first patient was dosed in the Phase 3 clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in development for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, OCT 2, 2024, View Source [SID1234647005]). UGN-103 is a next-generation novel mitomycin-based formulation.

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"Reaching this Phase 3 trial milestone for UGN-103 highlights our drive to innovate and bring forward cutting-edge treatments for low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer, UroGen. "UGN-103 represents a significant step forward, offering potential improvements in manufacturing, convenience, and cost. We are excited about the potential of this next-generation formulation and look forward to furthering its development to advance the care of patients."

The UGN-103 formulation uses UroGen’s RTGel platform technology, a proprietary sustained-release, reverse-thermal hydrogel with the potential to improve the therapeutic profiles of existing drugs. The U.S. Food and Drug Administration (FDA) accepted the Investigational New Drug Application for UGN-103 in April 2024, permitting the drug’s investigational use in adults with LG-IR-NMIBC, a highly recurrent disease. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 (mitomycin) for intravesical solution for LG-IR-NMIBC. UroGen completed the New Drug Application (NDA) submission for UGN-102 ahead of schedule with potential FDA approval in early 2025, if the NDA is accepted for filing by the FDA and priority review is granted.

The UTOPIA study is a single-arm, multicenter study that will evaluate the efficacy and safety of UGN-103. UroGen is aiming to enroll 87 patients with LG-IR-NMIBC in the UTOPIA study. Patients will receive 75 mg of UGN-103 via intravesical instillation once a week for 6 weeks. Efficacy will be assessed by the complete response rate at the three-month visit. Patients who have a complete response at the three-month visit, defined as having no detectable disease in the bladder, will enter the follow-up period of the study. During the follow-up period, patients will return to the clinic every three months for evaluation of response. Patients will remain on study until disease recurrence, disease progression, death, or the last patient completes 12 months of follow-up (i.e., 15 months after the first instillation), whichever occurs first. To learn more about the UTOPIA trial, visit clinicaltrials.gov/NCT06331299.

About UGN-103

UGN-103 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin being developed for the treatment of LG-IR-NMIBC. Anticipated advantages of UGN-103 include a new formulation of mitomycin that may considerably shorten the manufacturing process and simplify the reconstitution procedure. UroGen announced on September 16, 2024, that it received from the United States Patent and Trademark Office (USPTO), a Notice of Allowance covering, among other things, the use of UGN-103 in the treatment of LG-IR-NMIBC. The U.S. patent, once issued, will have an expiration date in December 2041. UGN-103 will utilize UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.

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 for UGN-102 in August 2024, ahead of schedule, with a potential FDA decision as early as the first quarter of 2025, if the NDA is accepted for filing by the FDA and priority review is granted.

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 the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for the management of NMIBC include 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.

On October 2, 2024 Volastra Therapeutics, a clinical-stage biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to VLS-1488, the company’s internally discovered KIF18A inhibitor (Press release, Volastra Therapeutics, OCT 2, 2024, View Source [SID1234647004]). The designation has been granted for the treatment of patients with platinum-resistant high-grade serous ovarian cancer (HGSOC).

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"Platinum-resistant high-grade serous ovarian cancer is marked by poor prognosis, highlighting the urgent need for new therapies for this devastating disease," said Scott Drutman, M.D., Ph.D., Head of Research & Development and Chief Medical Officer. "Fast Track designation for VLS-1488 reaffirms the clear potential of KIF18A inhibition to address this unmet medical need and represents a critical step towards bringing these novel therapeutics to patients. We look forward to working closely with the FDA as we advance the development of inhibitors against this promising target."

Fast Track designation is a program intended to facilitate and expedite the development and review of new drugs for the treatment of a serious or life-threatening condition. To qualify for this designation, there must be clear data demonstrating the drug has potential to address unmet medical need in the designated condition.

In the U.S. alone, there are more than 20,000 new cases of ovarian cancer each year, over 75% of which are advanced. Most of these patients will see disease progression on platinum-based chemotherapy. Sovilnesib, the second of Volastra’s two KIF18A inhibitors, which was in-licensed from Amgen in 2023, has previously been granted Fast Track designation in this indication. Each of Volastra’s two KIF18A inhibitors, VLS-1488 and sovilnesib, has unique profiles and are both enrolling patients in early-phase studies.

The Phase I/II trial for VLS-1488 is evaluating safety, tolerability and preliminary efficacy in patients with advanced tumors, including HGSOC. Further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT05902988.

On October 2, 2024 Recursion, a leading clinical stage TechBio company decoding biology to industrialize drug discovery, reported that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for a Phase 1/2 clinical trial of REC-1245, a new chemical entity for the treatment of biomarker-enriched solid tumors and lymphoma (Press release, Recursion Pharmaceuticals, OCT 2, 2024, View Source [SID1234647003]).

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Chris Gibson, Ph.D., Co-founder and CEO of Recursion said, "REC-1245 is a prime example of using an expansive AI-enabled platform for drug discovery. After exploring many predicted biological and chemical relationships across our maps of biology, we identified RMB39 as a novel target that looks functionally similar to the well-known but hard to drug target CDK12. We also identified and optimized small molecules that target RBM39 without directly impacting CDK12 or CDK13 using these same AI-enabled maps. In under 18 months, leveraging some of our newer chemistry tools, Recursion rapidly progressed REC-1245 from novel target biology to preclinical drug candidate, more than twice the speed of industry average."

Recursion identified the novel regulatory role of RBM39 associated with CDK12 using its maps of biology and first reported this relationship in early 2023 at Download Day, Recursion’s R&D and investor event. Recursion believes the modulation of RBM39 may be associated with a therapeutic effect in certain biomarker-enriched solid tumors and lymphoma. Additionally, Recursion estimates that the initially addressable population for this potential therapeutic to be >100,000 patients in the US and EU5. REC-1245 is a potent and selective RBM39 degrader with a potential first-in-class profile. Preclinical data support that RBM39 degradation induces splicing defects which downregulate DNA Damage Response (DDR) networks and cell cycle checkpoints.

"RBM39 degraders may offer a promising therapeutic approach for patients with solid tumors, particularly those with limited treatment options," said Najat Khan, Ph.D., Chief R&D Officer and Chief Commercial Officer at Recursion. "Recursion’s platform was among the first to rapidly uncover the therapeutic potential of RBM39 degradation, a finding now validated by independent research. This mechanism provides new opportunities for targeting tumors, which are often resistant to conventional treatments. By advancing this research, we aim to deliver a critical option for patients facing significant unmet needs, ultimately improving their prognosis and quality of life."

The Phase 1/2 clinical trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential monotherapy efficacy of REC-1245, and is expected to initiate in Q4 2024.

On October 2, 2024 Processa Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs with improved efficacy and safety, reported that the first patient has been dosed in a Phase 2 clinical trial evaluating NGC-Cap for the treatment of advanced or metastatic breast cancer (Press release, Processa Pharmaceuticals, OCT 2, 2024, View Source [SID1234647002]).

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"Dosing the first patient in this Phase 2 trial is a significant step in the development of NGC-Cap as a more effective and better tolerated treatment than widely used capecitabine and 5-FU," stated David Young, PharmD, Ph.D., President of Research and Development at Processa. "We expect this Phase 2 trial to build upon NGC-Cap’s positive Phase 1b findings and we look forward to announcing the results from our interim analysis of this Phase 2 trial in mid-2025."

The Phase 2 trial (NCT06568692) is a global multicenter, open-label, adaptive designed safety-efficacy trial comparing two different doses of NGC-Cap to FDA-approved monotherapy capecitabine in approximately 60 to 90 patients with advanced or metastatic breast cancer. The trial is designed to evaluate the safety-efficacy profile of NGC-Cap versus monotherapy capecitabine, to determine the potential optimal dosage regimens of NGC-Cap as required by the FDA Project Optimus Initiative and to evaluate the possibility of personalizing NGC-Cap therapy.

To date, three clinical trial sites, including some with multiple clinical locations, have received institutional review board approval to participate in this study and are recruiting patients. Processa plans to activate approximately 30 sites worldwide.

Breast cancer is the second most common cancer and a leading cause of cancer-related death. More than 2 million cases of breast cancer were diagnosed in 2022 with more than 665,000 deaths globally. The five-year survival rate for those diagnosed with metastatic breast cancer is approximately 30%.

About Capecitabine Administered with PCS6422 (NGC-Cap)
NGC-Cap combines the administration of PCS6422, the Company’s irreversible dihydropyrimidine dehydrogenase (DPD) enzyme inhibitor, with low doses of capecitabine. Capecitabine is the oral prodrug of 5-fluorouracil (5-FU), and along with 5-FU is among the most widely used chemotherapy drugs, particularly for the treatment of solid tumors. When metabolized (after oral ingestion) it becomes 5-FU in the body, which, in turn, metabolizes to molecules called anabolites that actively kill duplicating cells, such as cancer cells, and to molecules called catabolites that only cause side effects. The presence of the DPD enzyme plays an integral role in the undesirable conversion of 5-FU to catabolites causing side effects while simultaneously decreasing tumor exposure to 5-FU and its cancer-killing anabolites.

On October 2, 2024 PDS Biotechnology, a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, reported that updated data from the IMMUNOCERV Phase 2 clinical trial evaluating Versamune HPV (formerly PDS0101) with chemoradiation to treat locally advanced cervical cancer were presented at the American Society for Radiation Oncology (ASTRO) Annual Meeting 2024 in an oral presentation by Adam Grippin, M.D., Ph.D., of The University of Texas MD Anderson Cancer Center (PubMed, PDS Biotechnology, OCT 2, 2024, View Sourcesec.gov/Archives/edgar/data/1472091/000114036124042537/ef20036539_ex99-1.htm" target="_blank" title="View Sourcesec.gov/Archives/edgar/data/1472091/000114036124042537/ef20036539_ex99-1.htm" rel="nofollow">View Source [SID1234647001]). The abstract was granted Basic/Translational Science Award from the ASTRO Annual Meeting Steering Committee.

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"HPV is responsible for virtually all cervical cancers and presents an opportunity for immunologic targeting1. However, there are currently no FDA-approved HPV-targeted immunotherapies to treat cervical cancer," said Ann Klopp, M.D., Ph.D., Professor of Radiation Oncology and Head of the Gynecologic Section at MD Anderson. "These data suggest that further investigation is warranted into the safety and efficacy of Versamune HPV in combination with standard of care in the treatment of locally advanced cervical cancer."

The IMMUNOCERV Phase 2 clinical trial (NCT04580771) evaluated the efficacy, safety and tolerability of Versamune HPV in combination with standard-of-care chemoradiotherapy for the treatment of locally advanced cervical cancer. The investigator-initiated study enrolled 17 newly diagnosed high-risk patients with large tumors of at least 5 cm in size. Highlights from the presentation include:

•All patients received at least 2 doses of Versamune HPV

•Median follow-up was 19 months

•36-month overall survival (OS) rate was 84.4%, and 100% for the eight patients who received all five doses of Versamune HPV. Historical published data show 36-month OS rate with chemoradiation in this population of approximately 64%.2

•36-month progression free survival (PFS) rate was 74.9%, among all patients and 100% for the eight patients who received all five doses of Versamune HPV. Historical published data show 36-month PFS rate with chemoradiation in this population of approximately 61%.2

•Complete metabolic response (CMR) was achieved in 15/17 (88%) patients

•Versamune HPV appeared to be safe and well-tolerated. The most common treatment-related toxicities were injection site reactions in twelve patients (71%).

"We are pleased that data from the Phase 2 IMMUNOCERV trial demonstrate compelling clinical activity and a promising safety profile," said Frank Bedu-Addo, Ph.D., President and Chief Executive Officer of PDS Biotech. "Based on our continued research in various HPV-positive cancers, Versamune HPV appears to work in combination with a variety of therapeutic agents to generate clinical responses and promote improved survival in patients with minimal toxicity. We look forward to the next steps in the development of Versamune HPV for locally advanced cervical cancer."

1. National Cancer Institute, Cervical Cancer Causes, Risk Factors and Prevention. View Source View Sourcecancer.gov/types/cervical/causes-risk-prevention

2. Rose PG, et al. Concurrent Cisplatin-Based Radiotherapy and Chemotherapy for Locally Advanced Cervical Cancer. N Engl J Med. 1999;340:1144-53.