On September 16, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for patent application no. 18/535,108 entitled "Material and Method for Treating Cancer (Press release, UroGen Pharma, SEP 16, 2024, View Source [SID1234646663])."

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The allowed claims relate to the combination of UroGen’s RTGel technology with medac’s licensed proprietary lyophilized mitomycin formulation and cover the use of UroGen’s UGN-103 and UGN-104 development programs in the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) and low-grade upper tract urothelial cancer (LG-UTUC), respectively. The U.S. patent, once issued, will have an expiration date in December 2041.

"Allowance of this patent application strengthens our intellectual property position for our next-generation investigational programs for patients with urothelial cancers," said Liz Barrett, President and CEO of UroGen. "We believe that the combination of UroGen’s RTGel technology with medac’s patent-protected lyophilized mitomycin has the potential to provide advantages related to production, cost, supply, and product convenience for patients. Our vision remains to develop innovative medicines that advance the care of patients, and this patent allowance is a key element of our strategy to build a long-term growth company."

In January 2024, UroGen entered into a licensing and supply agreement with medac to develop UGN-103 for LG-IR-NMIBC and UGN-104 for LG-UTUC. In April 2024, the FDA accepted UroGen’s IND application for UGN-103 and the company is currently onboarding clinical sites for the study. The company anticipates commencing a similar study for UGN-104, which is expected in early 2025.

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. UroGen anticipates potential FDA approval in early 2025 if the NDA is accepted for filing by the FDA and priority review is granted.

About UGN-103 and UGN-104

UGN-103 and UGN-104 are innovative mitomycin formulations in development by UroGen for the treatment of LG-IR-NMIBC and LG-UTUC, respectively. UGN-103 aims to streamline manufacturing and reconstitution processes while providing intellectual property coverage until December 2041; it utilizes UroGen’s RTGel technology for prolonged mitomycin exposure. UGN-104, also leveraging RTGel technology, is designed for treating low-grade upper tract urothelial cancer and is anticipated to enter Phase 3 trials in early 2025, offering a non-surgical treatment option with similar intellectual property protection.

On September 16, 2024 Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported positive, mature data from its ongoing phase 1b / 2a study of fostroxacitabine bralpamide (fostrox) + Lenvima in advanced liver cancer (hepatocellular carcinoma/HCC) at the ESMO (Free ESMO Whitepaper) (European Society of Medical Oncology) Congress in Barcelona, Spain (Press release, Medivir, SEP 16, 2024, View Source;lenvima-confirm-promise-of-improved-outcomes-in-advanced-liver-cancer-detailed-and-mature-data-presented-at-esmo-302248817.html [SID1234646679]).

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Today’s ESMO (Free ESMO Whitepaper) update, poster number 986P, presented by Dr Hong Jae Chon on Monday September 16, shows promising duration of benefit with 19% of patients continuing treatment for more than a year and the longest running patient remaining on treatment for over 2 years, with sustained partial response. The patients in the study had disease control on fostrox + Lenvima independent if they benefitted from previous line of therapy, showing potential for all second-line patients to benefit from the combination. The safety and tolerability profile continues to be encouraging with no unexpected adverse events. While hematological adverse events were common, they were temporary in nature. Decreases in neutrophil & platelet counts showed a cyclic pattern with recovery before next cycle of treatment, enabling patients to remain on treatment long-term. Importantly, no patient experienced febrile neutropenia or low platelet count with bleeding and there were no fostrox-related serious adverse events.

Results come despite very poor prognosis for most second-line HCC patients today, with just 5–10% responding to current standard of care treatment, and a typical TTP of only 3–4 months.

Dr. Pia Baumann, Chief Medical Officer at Medivir, said:
– "With three patients still remaining on study treatment, all of whom treated for more than a year, this data-set is now quite mature. At a median follow-up of 10.5 months, fostrox + Lenvima have clearly shown promise of improved outcomes beyond current alternatives for second-line liver cancer patients. Fostrox is designed to only target tumor cells locally in the liver, without harming healthy cells. It is therefore reassuring to see the tolerability profile of fostrox enabling the combination of two highly potent treatments, fostrox + Lenvima, without compromising patient safety. Patients were able to stay on treatment long-term, which evidently contributes to the extended duration of benefit and a median time to progression of 10.9 months, substantially longer than previously seen in second-line liver cancer. It is with reinforced confidence we continue our preparations for the initiation of the planned phase 2b study comparing fostrox + Lenvima with Lenvima alone in a randomized setting to confirm the benefit of the combination."

Dr Hong Jae Chon, Professor at CHA Bundang Hospital in Korea, and investigator in the fostrox + Lenvima study, commented:
"Treatment outcomes have improved in first-line with the use of immunotherapy combinations, resulting in more patients fit enough to receive second-line treatment. But with no treatments approved in second-line after immunotherapy, there is a significant unmet medical need for new treatments options for these patients. The phase 1b/2a data for fostrox + Lenvima show highly encouraging clinical benefits for patients, indicating that when adding fostrox to Lenvima, efficacy is better than expected from Lenvima alone. It is especially encouraging that in addition to patients experiencing benefit for an extended period of time, patients also responded to the treatment independent of outcome in previous line of therapy. I look forward to evaluating the efficacy of fostrox plus Lenvima in a randomized, controlled trial."

The data are from Medivir’s ongoing phase 1b/2a open-label, multi-center, dose-escalation and dose-expansion study, evaluating the safety and efficacy of fostrox in combination with Lenvima in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable.

HCC is the most common type of liver cancer, accounting for more than 80% of cases worldwide.2 There are approximately 660,000 patients diagnosed with HCC per year globally and current five-year survival is less than 20 percent3.

Medivir will host a webcast where Dr Chon and Dr Pia Baumann will present the data and answer questions. The webcast will take place Today, September 16, at 13.45 CET, and will be streamed via a link on the website: www.medivir.com/investors/presentations.

The poster and the presentation from the webcast will also be available on Medivir’s website after the presentation.

On September 16, 2024 Immutep Limited, a clinical-stage biotechnology company developing novel LAG-3 immunotherapiesfor cancer and autoimmune disease, reported positive efficacy and safety results from the TACTI-003 Phase IIb trial evaluating eftilagimod alpha (efti) in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) antiPD-1 therapy KEYTRUDA (pembrolizumab) as first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma patients (1L HNSCC) (Press release, Immutep, SEP 16, 2024, View Source [SID1234646626]).

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These results with a data cut-off of 11 March 2024 were selected as a Proffered Paper oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 and were presented by Claus Kristensen, M.D., Ph.D., Head of Section for Thoracic and Head and Neck Oncology, Rigshospitalet, Copenhagen, Denmark, on 15 September. The data adds to the previously reported overall response rates and safety data on 27 June and 12 July.

Dr. Kristensen stated, "The efficacy and safety data in TACTI-003 are very encouraging and show the significant potential of this novel immunotherapy combination to fight difficult-to-treat head and neck squamous cell carcinomas. The clinically meaningful improvement in responses for patients with high PD-L1 expression in the randomised portion of the trial, combined with the compelling response rates in patients with no PD-L1 expression, are a testament to the complementary nature of efti in combination with KEYTRUDA. I am particularly impressed that these higher response rates and clear increase in biological activity seen in the efti arm do not come at the expense of durability of response or lead to an increased toxicity profile, which is often the case when combining therapies in the search for more efficacious treatments for cancer patients."

Dr. Frédéric Triebel, CSO of Immutep, said "Through multiple clinical trials, we see the promise of efti to not only improve cancer patients’ clinical responses to immune checkpoint inhibitors, but also to expand patient populations who respond to them including patients with negative PD-L1 expression. Once again, the TACTI003 trial has reinforced efti’s positive impact on both these fronts. We are excited to see efti in combination with KEYTRUDA now driving a 1.9-fold increase in responses for head and neck cancer patients with high PDL1 expression as compared to KEYTRUDA alone, and a statistically significant increase in absolute lymphocyte count in the treatment arm showing efti’s biological activity in a randomised setting."

Marc Voigt, CEO of Immutep, added "As we move into the latter half of 2024, we will continue to follow the data in TACTI-003 and start to engage with regulatory authorities regarding potential paths forward. We are certainly pleased with durability we are seeing, which is consistent with other trials in which efti combined with KEYTRUDA achieves a high DOR, unlike many other therapeutic combinations. We are hopeful this positive duration of response continues and, as seen in first line non-small cell lung cancer in the TACTI-002 trial evaluating efti in combination with KEYTRUDA, eventually contributes to an overall survival benefit for patients with first line head and neck cancer."

ESMO Congress 2024 Proffered Paper Oral Presentation
Title: Primary Results from TACTI-003: A Randomized Phase IIb Trial Comparing Eftilagimod Alpha (soluble LAG-3) Plus Pembrolizumab Versus Pembrolizumab Alone in First-Line Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma with CPS ≥1

Clinical Highlights from Randomised Cohort A in 1L HNSCC Patients with Any PD-L1 Expression (CPS ≥1)
Efti leads to higher Objective Response Rates (ORR)
• Efti in combination with pembrolizumab (E+P) led to a 32.8% ORR (34.5% including one partial response reported after data cut-off) in evaluable patients with CPS ≥1 (N=58) compared to 26.7% for pembrolizumab in evaluable patients with CPS ≥1 (N=60), according to RECIST 1.1. Imbalances of prognostic markers towards the pembrolizumab alone arm included HPV status, smoking status, and primary tumour location.

• E+P outperformance in patients with any PD-L1 expression was strongest in high PD-L1 expressing patients (CPS ≥20) with a 31.0% ORR (N=29) versus an 18.5% ORR for pembrolizumab alone (N=27), along with a complete response rate of 6.9% in E+P arm versus 3.7% for pembrolizumab alone. An additional partial response was reported in CPS ≥20 after data cut-off leading to a 34.5% ORR, a 1.9- fold increase in responses over pembrolizumab alone in this patient group.

Efti maintains a high Duration of Response (DOR)
• Durability of response was achieved with the addition of efti with a median DOR of 17.5 months s in the E+P arm (N=58) as compared to 17.1 months in the pembrolizumab alone arm (N=60)
o Data compares favourably to other anti-PD-1 combinations with cytotoxic drugs like chemotherapy or EGFR inhibitors, including a historical DOR of ~6 to ~7 months from antiPD-1 combined with chemotherapy in 1L HNSCC1-4

Efti increases Biological Activity
• A statistically significant increase in absolute lymphocyte count (ALC), measured as an exploratory biomarker, was seen in the E+P arm as shown in the graphic below, indicating an effective eftiinduced immune response in this randomised setting
ALC increase is in line with data from other Phase II trials evaluating efti in combination with chemotherapy in metastatic breast cancer or pembrolizumab in non-small cell lung cancer5-6

Efti continues to have favourable safety profile
• Efti in combination with pembrolizumab continues to have a favourable safety profile with no new safety signals observed
o Discontinuation rate from treatment emergent adverse events was similar for both E+P (4.3%) and for pembrolizumab alone (4.4%)
o Unlike other combinations with anti-PD-1 therapy, E+P continues to have a comparable safety profile to pembrolizumab alone other than injection site reactions as expected with efti’s subcutaneous delivery.

Additionally, E+P drives a high ORR and Disease Control Rate (DCR) in 1L HNSCC patients regardless of PD-L1 expression. In Cohorts A and B together (N=89), E+P achieved a 33.7% ORR (34.8% including one partial response reported after data cut-off) including 31 patients in Cohort B with negative PD-L1 (CPS <1). E+P also achieved a higher DCR compared to pembrolizumab monotherapy across all PD-L1 expression levels, with a consistent increase from 58.1% DCR in CPS <1, to 69.0% DCR in CPS 1-19, to 75.9% DCR in CPS ≥20.

This new data adds to the body of evidence that efti’s activation of antigen-presenting cells provides a strong boost to the immune system, enhancing the potential of immune checkpoint inhibitors (ICI) such as KEYTRUDA. As the only MHC Class II agonist in clinical development today, efti generates a broad anti-cancer immune response in combination with ICIs regardless of PD-L1 expression, including for patients with negative PD-L1 expression, in a unique and safe manner across multiple different cancers.

On September 16, 2024 Cytovation ASA, a clinical stage oncology company focused on the development of its first-in-class bifunctional immunotherapy CY-101 (CyPep-1) reported that it will deliver a poster presentation at ESMO (Free ESMO Whitepaper) 2024 entitled "Safety and activity of CY-101 in patients with advanced solid tumors: the Phase I/IIa CICILIA trial (Press release, Cytovation, SEP 16, 2024, View Source;utm_medium=rss&utm_campaign=cytovation-to-present-full-safety-and-efficacy-data-from-the-cicilia-phase-i-iia-trial-evaluating-cy-101-in-solid-tumors-at-esmo-2024 [SID1234646647])." Data from this study, evaluating intratumoral (IT) administration of CY-101 monotherapy and in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with heavily pretreated advanced solid tumors, show that CY-101 is well tolerated at the recommended Phase 2 dose (RP2D) of 20 mg. Furthermore, these data demonstrate early signs of antitumor activity, especially in tumors with dysregulated Wnt/β-catenin signaling.

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CY-101 is a synthetic peptide with a dual mode of action that both inhibits the oncogenic Wnt/β-catenin pathway by activating Axin2 and has a pore-forming membranolytic effect on cancer cells that exposes antigens and triggers a systemic tumor-specific immune response. Interim results from CICILIA were reported in 2023 (link to press release here) confirming that all trial endpoints were met, with CY-101 demonstrating a consistent safety profile across tumor types and strong early signals of efficacy.

Data from the ESMO (Free ESMO Whitepaper) poster show that no dose-limiting toxicities were observed in the dose- escalation part of the study with the RP2D defined at 20 mg. Analyses of paired tumor biopsies demonstrated induction of cancer cell death in >70% across tumor types at the RP2D, translating to a clinical benefit in several different cancer types. Notably, among six patients with adrenocortical carcinoma (ACC) receiving CY-101 monotherapy, a disease control rate of 50% (n = 3/6) was observed, with durable responses (> 6 months) in two patients. Both patients expressed β-catenin and had somatic mutations in the Wnt/β-catenin pathway.

Separately, the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to CY-101 monotherapy for the treatment of ACC. Orphan Drug Designation is granted to investigational therapies that are intended for the treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the US. Orphan Drug Designation provides several benefits to drug developers, including certain development cost benefits in the US, increased FDA interaction and eligibility for seven-year market exclusivity following approval.

"We believe CY-101 is a potentially important new treatment option that offers a truly differentiated approach to targeting Wnt/β-catenin driven cancers, an oncogenic pathway dysregulated in more than 20% of cancers," said Lars Prestergarden, CEO of Cytovation. "Our early data clearly confirm that CY-101 warrants further investigation in larger studies, and we are very pleased to receive Orphan Drug Designation for CY-101 in ACC. We look forward to advancing towards our planned Phase 2 trial in ACC with registrational intent."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merk & Co., Inc., Rahway, NJ, USA.

On September 16, 2024 ScaleReady and Bio-Techne Corporation (NASDAQ: TECH) reported the launch of the G-Rex optimized ProPakTM GMP Cytokines, ideally tailored to high efficiency closed system cell and gene-modified cell therapy (CGT) manufacturing (Press release, Bio-Techne, SEP 16, 2024, View Source [SID1234646664]).

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The ProPakTM GMP Cytokine product consists of a weldable bag filled with liquid formulated GMP-grade cytokines, specifically interleukin-7 (IL-7) or interleukin-15 (IL-15), commonly used in the ex vivo manufacturing of CAR-T and TCR-T cells. The quantity of IL-7 and IL-15 contained in each ProPakTM is sufficient to dose a one (1) liter G-Rex bioreactor at the recommended 10ng/mL concentration. The use of ProPak GMP cytokines will enable manufacturers of CAR-T and TCR-T cell therapies to reduce operating costs and complexity by dramatically simplifying the process of acquiring, storing, preparing, and administering these critical reagents for use in cGMP manufacturing.

"Bio-Techne is deeply committed to highly efficient manufacture of lifesaving cell and gene-modified cell therapies," said Will Geist, President (Protein Sciences) at Bio-Techne Corporation. "ProPakTM GMP Cytokines were optimized for use with the leading G-Rex platform by providing the precise quantity of cytokines needed for highly simplified closed system expansion of CAR-T and TCR-T cell drug products with excellent cell characteristics."

"The G-Rex optimized ProPakTM GMP Cytokine is a differentiated product as it is uniquely designed to enable users of G-Rex to manufacture CAR-T and TCR-T drug products with far more simplicity than is possible with any other cytokine vendor," said Josh Ludwig, Global Commercial Director at ScaleReady. "It eliminates the need for cytokine reconstitution and all of the related headaches in favor of a hands-off ballroom style G-Rex manufacturing approach.

"With the incredible response to our G-Rex Grant Program moving G-Rex into the majority of CGT clinical trials, the G-Rex optimized ProPak is the perfect next step in G-Rex manufacturing simplicity. Grant applicant or not, we will help every G-Rex user cost effectively perform comparability studies to integrate the ProPak GMP Cytokines," concluded Josh Ludwig.

Parties interested in learning more and/or evaluating the ProPakTM GMP Cytokines can reach out to [email protected].