On November 10, 2022 Artiva Biotherapeutics, Inc., a clinical stage company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell-based therapies that are safe and accessible to cancer patients, reported the presentation of activity and efficacy preclinical data for AB-101 with several combinations of therapeutic monoclonal antibodies in multiple preclinical models of cancer (Press release, Artiva Biotherapeutics, NOV 10, 2022, View Source [SID1234623660]). The data were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37TH Annual Meeting in Boston.

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"These data provide additional support for the use of AB-101 as a highly scaled, off-the-shelf, ADCC-enhancing NK cell product candidate with optimized cellular profile but without the need for genetic modification," said Peter Flynn, Ph.D., cofounder and Chief Operating Officer of Artiva. "Specifically, these data support the use of AB-101 to enhance ADCC activity when combined with antibodies such as Rituxan, Gazyva, Erbitux and Herceptin, highlighting the broad therapeutic potential of AB-101."

The data presented highlight the use of Artiva’s AlloNKTM platform to generate NK cells with high and consistent expression of the high affinity variant of the receptor CD16 as well as other receptors involved in anti-tumor activity including NKG2D, DNAM-1, NKp30, and NKp46, all without the requirement to engineer the underlying NK cells. The resulting cryopreserved, off-the-shelf cell therapy candidate AB-101 combined with therapeutic monoclonal antibodies was seen to drive anti-tumor cell activity in a variety of solid tumor and hematopoietic model systems. Artiva is conducting a Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT04673617) to assess the safety and clinical activity of AB-101 alone and in combination with the anti-CD20 monoclonal antibody, rituximab. Artiva is also developing a combination therapy comprised of AB-101 and Affimed N.V.’s Innate Cell Engager AFM13 for treatment of patients with relapsed/refractory CD30-positive lymphomas.

The abstract is available on the Journal for ImmunoTherapy of Cancer website: View Source The poster is available for viewing by SITC (Free SITC Whitepaper) attendees on the SITC (Free SITC Whitepaper) virtual platform: View Source

The poster can also be viewed here.

About AB-101

AB-101 is a cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers. Artiva selects cord blood units with the high affinity variant of the receptor CD16 and a KIR-B haplotype for enhanced product activity. Artiva can generate thousands of doses of pure, cryopreserved, infusion-ready NK cells from a single umbilical cord blood unit while retaining the high and consistent expression of CD16 without the need for engineering. Artiva is conducting a Phase 1/2 multicenter clinical trial (ClinicalTrials.gov Identifier: NCT04673617) to assess the safety and clinical activity of AB-101 alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell-non-Hodgkin lymphoma (NHL) who have progressed beyond two or more prior lines of therapy. Artiva is also developing a combination therapy comprised of AB-101 and Affimed N.V.’s Innate Cell Engager AFM13 for treatment of patients with relapsed/refractory CD30-positive lymphomas.

On November 10, 2022 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported the presentation of positive interim results from the ongoing Phase 1 trial of the company’s MP0317 (FAP X CD40) program for the treatment of solid tumors at the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual meeting (Press release, Molecular Partners, NOV 10, 2022, View Source [SID1234623659]).

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While holding promise, the development of CD40 agonists for cancer therapy has faced challenges due to systemic CD40 activation leading to dose-limiting toxicity (DLT). MP0317 is designed to resolve these limitations by activating immune cells specifically within the tumor microenvironment through the simultaneous binding of the immune stimulator CD40 as well as a protein highly expressed within tumors named fibroblast activation protein, or FAP.

"This first clinical data supports the potential of MP0317 as a candidate able to achieve the goal of restricting CD40 activity to tumors. We are now progressing into dosages well above those that produced dose-limiting toxicities with non-DARPin CD40 agents, a significant achievement we hope to translate into observed clinical effect," said Nicolas Leupin, MD, Ph.D., CMO of Molecular Partners. "Our oncology programs continue displaying the potential of DARPin therapeutics to solve historical drug development challenges such as localizing potent immune activation to tumors in order to spare damage to healthy cells."

This Phase 1, first-in-human, multicenter, open label, dose escalation study enrolling patients with relapsed/refractory advanced solid tumors is intended to evaluate the safety of MP0317 and investigate a range of other biomarkers to better characterize the candidate’s mechanism and activity. At the point of data cutoff, 4 cohorts had received an intravenous dose of MP0317 every 3 weeks until disease progression, unacceptable toxicity, or other discontinuation criteria were met.

Key reported data:

MP0317 was seen to be safe and well tolerated with no dose limiting CD40-related systemic toxicities having been observed to date, and no signs of inflammatory cytokine release.
The most frequent adverse events were grade 2 infusion related reactions (e.g., rapidly resolved infusion site inflammation) in 3/13 dosed patients at the time of data cutoff, with no DLT observed. This spans cohorts 1-4, with dosages ranging from 0.03 mg/kg to 1 mg/kg.
Tumor biopsies from the earlier cohorts (1-3) already show evidence of MP0317 co-localization with both CD40 and FAP, in 3 of the 5 tumor biopsies available for analysis.
In addition, early PD data show signs of CD40-mediated immune activation.
These data support that MP0317’s mechanism is working as intended.
These results will be presented at SITC (Free SITC Whitepaper) 2022 in a poster, the details of which can be found below. The poster will be made available on Molecular Partners website, after the presentation on November 10.

Poster: "A phase 1 study to characterize the safety and tolerability of MP0317, a tumor targeting FAP dependent CD40 agonist DARPin, in patients with relapsed/refractory solid tumors"
Number: 1475
Timing: November 10, 2022
Presenter: Paul Baverel, PhD

The dose escalation of the Phase 1 remains ongoing and Molecular Partners expects the final data set to inform the therapeutic dose for evaluation in a potential Phase 2 trial. Subsequent to the data cutoff, current ongoing dosing levels include a 3 mg/kg cohort dosed every 3 weeks, as well as a 0.5 mg/kg weekly dosing cohort. A total of 19 patients have been enrolled in the Phase 1 study as of Nov. 1, 2022.

On November 10, 2022 Monopar Therapeutics Inc. (Monopar or the Company) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported third quarter 2022 financial results and summarized recent program developments (Press release, Monopar Therapeutics, NOV 10, 2022, View Source [SID1234623658]).

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Recent Program Developments

Validive – International Phase 2b/3 Trial, Interim Go/No-go Analysis on Track for Q1 2023

The VOICE trial continues to enroll patients and add additional clinical sites (now at 73 active sites and over 130 patients dosed across the U.S. and Europe).

Enrollment for the Phase 2b portion of the trial has been completed. The trial recently expanded to include sites in Germany and Poland, and enrollment for the Phase 3 portion of the trial has commenced.

The blinded interim analysis of clinical data from the Phase 2b patient cohort of the trial, to be performed by an independent data monitoring board, will be used to guide the Company as to whether or not to continue enrolling the Phase 3 portion of the trial. This analysis should be completed and reported out in Q1 2023.

Camsirubicin – Phase 1b Dose-Escalation Trial, Data to be Presented Next Week at CTOS 2022

Monopar is currently dosing the fourth dose-level cohort, which is approximately double the highest dose of camsirubicin ever tested in a prior trial.

Early results from the open-label camsirubicin Phase 1b clinical trial will be presented at the Connective Tissue Oncology Society (CTOS) Annual Meeting Conference being held on November 16-19, 2022 in Vancouver, BC.

MNPR-101 Radioimmunotherapeutic – Preclinical Studies Being Conducted to Support FIH Study

Monopar is actively conducting preclinical studies to support a submission for a first-in-human (FIH) study with an MNPR-101-based radioimmunotherapeutic/radiodiagnostic that the Company has generated with its partner NorthStar Medical Radioisotopes, LLC.

MNPR-202: Preclinical Data to be Presented at ASH (Free ASH Whitepaper) 2022 in December

Monopar’s collaborator, the Cancer Science Institute of Singapore at the National University of Singapore, has been testing MNPR-202 in preclinical cancer models with promising results.

Monopar and Cancer Science Institute of Singapore will present a poster of MNPR-202 preclinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting being held on December 10-13, 2022, in New Orleans, LA. The poster abstract can be found at the following link: View Source

Results for the Third Quarter Ended September 30, 2022, Compared to the Third Quarter Ended September 30, 2021

Cash and Net Loss

Cash and cash equivalents as of September 30, 2022, were $14.3 million. Monopar anticipates that its current cash and cash equivalents will fund: the completion of the Phase 2b portion of the VOICE clinical trial; the commencement of the Phase 3 portion of the VOICE clinical trial; and the Phase 1b camsirubicin clinical trial through at least November 2023. The Company plans to raise additional funds and/or engage a partner within the next 12 months to complete the VOICE clinical program and continue camsirubicin clinical development through and beyond the ongoing open-label, dose escalation Phase 1b clinical trial.

Net loss for the third quarter of 2022 was $2.4 million or $0.19 per share compared to net loss of $2.5 million or $0.20 per share for the third quarter of 2021.

Research and Development (R&D) Expenses

R&D expenses for the three months ended September 30, 2022, were $1,732,000, compared to $1,827,000 for the three months ended September 30, 2021. The decrease of $95,000 is attributed to (1) a decrease of $272,000 in R&D personnel expenses and (2) a $60,000 net decrease of other R&D expenses, partially offset by an increase of $237,000 in Validive clinical trial-related and clinical material manufacturing-related expenses.

General and Administrative (G&A) Expenses

G&A expenses for the three months ended September 30, 2022, were $675,000, compared to $632,000 for the three months ended September 30, 2021. The increase of $43,000 was primarily the result of an increase in G&A personnel expenses.

On November 10, 2022 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported financial results for the third quarter ended September 30, 2022, and provided a corporate update (Press release, VBI Vaccines, NOV 10, 2022, View Source [SID1234623657]).

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Jeff Baxter, VBI’s President and CEO commented:

"Our second quarter of the U.S. launch of PreHevbrio saw continued progress as our field teams worked to raise awareness of the new CDC adult hepatitis B (HBV) vaccination recommendations and the value proposition of our differentiated 3-antigen HBV vaccine. The launch is proceeding as planned, and we are pleased with the increased excitement, clinical demand, and overall reception our field teams have received. We have continued the implementation of critical market access and contracting infrastructure, all of which are key pieces of the initial groundwork needed to support mid-term commercial success.

"Outside of the U.S., we were very pleased to announce a marketing and distribution partnership with Valneva in certain European countries, and we look forward to working with their team to provide access to PreHevbri in these countries beginning in early 2023. For the rest of our pipeline, we continue to advance our lead candidates targeting chronic HBV, glioblastoma (GBM), and coronaviruses, and look forward to the anticipated meaningful clinical readouts and regulatory milestones in each program."

Recent Key Program Achievements and Projected Upcoming Milestones

Hepatitis B (HBV)

Prophylactic Hepatitis B Vaccine

●Notable commercial achievements for PreHevbrio in the U.S. over the past several months include:

●70% of Medicare-insured lives, 70% of commercially insured lives, and 60% of lives under state Medicaid plans are estimated to have coverage in place for the PreHevbrio specific Current Procedural Terminology (CPT) code
●Sales have been recorded in over 90% of our target territories

●September 2022: Following marketing authorization in the European Union/European Economic Area and in the United Kingdom, under the brand name PreHevbri [Hepatitis B vaccine (recombinant, adsorbed)], VBI and Valneva announced a marketing and authorization partnership in select European markets – these markets initially include the U.K., Sweden, Norway, Denmark, Finland, Belgium, and the Netherlands
●Beginning early 2023: VBI and Valneva expect to make PreHevbri available in certain European countries
●Regulatory filing under review by Health Canada

VBI-2601 (BRII-179): HBV Immunotherapeutic Candidate

●Year-end 2022: Interim topline data expected from Phase 2 human proof of concept combination study evaluating safety and efficacy of VBI-2601 (BRII-179) with BRII-835 (VIR-2218), an HBV-targeting siRNA
●Q3 2023: Interim topline results expected from part one of the two-part Phase 2a/2b combination study evaluating VBI-2601 (BRII-179) as an add-on to existing pegylated interferon (PEG-IFN-α) and nucleos(t)ide reverse transcriptase inhibitor (Nrtl) therapy in non-cirrhotic chronic HBV patients

Glioblastoma (GBM)

VBI-1901: Cancer Vaccine Immunotherapeutic Candidate

●Early 2023: Expected initiation of next phase of development in recurrent GBM setting, aiming to expand the number of patients in the ongoing Phase 1/2a study and adding a randomized control arm, with the potential for accelerated approval based on tumor response rates and improvement in overall survival
●Early 2023: Evaluation of VBI-1901 in combination with Agenus’ anti-PD-1, balstilimab, in the primary GBM setting expected to initiate as part of the Individualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT), a Phase 2 adaptive platform trial

COVID-19 & Coronaviruses

VBI-2901: Multivalent Coronavirus Vaccine Candidate

●Mid-year 2023: Interim data expected from Phase 1 study of VBI-2901, subject to speed of enrollment

Additional Corporate Updates

●Management Appointment: John Dillman appointed as VBI’s Chief Commercial Officer in July 2022
●Debt Financing: In September 2022, VBI closed a refinanced and upsized debt facility of up to $100 million with existing lender, K2 HealthVentures (K2HV), adding $20 million of non-dilutive funding to the balance sheet

Third Quarter 2022 Financial Results

●Cash Position: VBI ended the third quarter of 2022 with $83.6 million in cash compared with $121.7 million in cash as of December 31, 2021.
●Revenue: Revenue, net for the third quarter of 2022 was $0.3 million, compared to $0.1 million for the same period in 2021. The increase was due to an increase in U.S. product revenue related to the launch of PreHevbrio in the U.S.
●Cost of Revenue: Cost of revenues was $2.7 million in the third quarter of 2022 as compared to $2.5 million in the third quarter of 2021. The increase was due primarily to manufacturing costs for our 3-antigen HBV vaccine related to inventory build for the U.S. market and preparation for European launches.
●Research and Development (R&D): R&D expenses for the third quarter of 2022 were $5.0 million compared to $3.0 million for the same period in 2021. R&D expenses were offset by $2.4 million in the third quarter of 2022 and $5.1 million in the third quarter of 2021 due to government grants and funding arrangements. The increase in R&D expenses was mainly driven by the advancement of VBI-1901 as we prepare for the next clinical studies in recurrent and primary GBM patients.
●General and Administrative (G&A): G&A expenses for the third quarter of 2022 were $14.2 million compared to $9.7 million for the same period in 2021. The increase in G&A expenses, partially offset by government grants and funding arrangements, was a result of the increased commercial activities related to PreHevbrio, most notably the deployment of our promotional field team and development of our distribution infrastructure. Additional increased costs include increased insurance costs, increased professional costs, and increased labor costs.
●Net Cash Used in Operating Activities: Net cash used in operating activities for the nine months ended September 30, 2022 was $54.7 million, compared to $21.4 million for the same period in 2021. The increase was largely due to commercial expenses for the launch of PreHevbrio in the U.S., a decrease in net change in operating working capital related to purchase of inventory for commercial production of PreHevbrio, and $18.4 million of cash advanced from CEPI pursuant to the CEPI Funding Agreement during the nine months ended September 30, 2021, compared to $1.0 million cash advanced from the CEPI Funding Agreement for the same period in 2022.
●Net Loss and Net Loss Per Share: Net loss and net loss per share for the third quarter of 2022 were $25.2 million and $0.10, respectively, compared to a net loss of $15.9 million and a net loss per share of $0.06 for the third quarter of 2021.
●Net Loss and Net Loss Per Share, Excluding Foreign Exchange Loss: Net loss and net loss per share, excluding foreign exchange loss, for the third quarter of 2022 were $22.5 million and $0.09, respectively, compared to a net loss and a net loss per share, excluding foreign exchange loss, of $16.0 million and $0.06 for the third quarter of 2021. Foreign exchange loss for the third quarter of 2022 was $2.7 million as compared to a gain of $0.2 million for the third quarter of 2021. Certain intercompany loans between VBI Vaccines Inc. and our subsidiaries are denominated in a currency other than the functional currency of each entity. The primary driver of the increase in foreign exchange loss was the impact of the relative strengthening of the U.S. and Canadian Dollars against the New Israeli Shekel upon translation of these intercompany loans.

Use of Non-GAAP Financial Measures

Net Loss Excluding Foreign Exchange Loss and Net Loss per Share Excluding Foreign Exchange Loss are non-GAAP financial measures. VBI’s management believes that the presentation of Net Loss Excluding Foreign Exchange Loss and Net Loss per Share Excluding Foreign Exchange Loss is useful to investors because management does not consider foreign exchange loss, which is primarily driven by changes in exchange rates related to certain intercompany loans, when evaluating VBI’s operating performance. Non-GAAP financial measures are meant to supplement, and to be viewed in conjunction with, GAAP financial results. The presentation of these non-GAAP financial measures should not be considered in isolation or as a substitute for comparable GAAP financial measures and should be read only in conjunction with the Company’s financial statements prepared in accordance with GAAP. Reconciliations of the Company’s non-GAAP measures are included below.

About PreHevbrio

VBI’s hepatitis B vaccine is the only 3-antigen hepatitis B vaccine, comprised of the three hepatitis B surface antigens of the hepatitis B virus – S, pre-S1, and pre-S2. It is approved for use in the United States, European Union/European Economic Area, United Kingdom, and Israel. The brand names for this vaccine are: PreHevbrio (US), PreHevbri (EU/EEA, UK), and Sci-B-Vac (Israel).

U.S. Indication

PreHevbrio is indicated for prevention of infection caused by all known subtypes of hepatitis B virus. PreHevbrio is approved for use in adults 18 years of age and older.

U.S. Important Safety Information (ISI)

Do not administer PreHevbrio to individuals with a history of severe allergic reaction (e.g. anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of PreHevbrio.

Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of PreHevbrio.

Immunocompromised persons, including those on immunosuppressant therapy, may have a diminished immune response to PreHevbrio.

PreHevbrio may not prevent hepatitis B infection, which has a long incubation period, in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration.

The most common side effects (> 10%) in adults age 18-44, adults age 45-64, and adults age 65+ were pain and tenderness at the injection site, myalgia, fatigue, and headache.

There is a pregnancy exposure registry that monitors pregnancy outcomes in women who received PreHevbrio during pregnancy. Women who receive PreHevbrio during pregnancy are encouraged to contact 1-888-421-8808 (toll-free).

On November 10, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the third quarter ended September 30, 2022, and provided an overview of recent developments (Press release, UroGen Pharma, NOV 10, 2022, View Source [SID1234623656]).

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"From a consistent vision to pipeline advancement and product commercialization, we remain highly encouraged by the inroads we have made creating a new market in how urologic cancers are treated. Despite slightly lower revenue compared to the previous quarter, JELMYTO’s favorable growth trends, which include a substantial increase of year-over-year net product revenues, new site activations, and recent real-world outcomes data, continues to give us confidence in the LG-UTUC opportunity as we work to replicate the success we’ve had in certain key markets," said Liz Barrett, President and Chief Executive Officer of UroGen. "We anticipate that the FDA authorization this quarter to extend JELMYTO’s in-use period for the admixture from 8 hours to 96 hours following reconstitution will provide yet another significant opportunity towards driving market adoption and expansion."

"In parallel, we are rapidly advancing perhaps the most exciting investigational product in our pipeline, UGN-102, which has the potential to transform the treatment of bladder cancer and significantly broaden the utilization of our RTGel reverse-thermal hydrogel technology," continued Ms. Barrett. "ENVISION, our Phase 3 pivotal trial of UGN-102 in LG-IR-NMIBC, is on pace to complete enrollment as soon as the end of the month, bringing us one step closer to realizing UGN-102’s potential as the first non-surgical primary therapeutic to treat this important subset of bladder cancer patients. As we advance each of these programs, as well as our broader portfolio, we continue a disciplined and balanced approach to capital preservation in support of growing our business and maintaining a strong balance sheet."

Business Highlights:

Jelmyto (mitomycin) for pyelocalyceal solution in low-grade Upper Tract Urothelial Cancer (LG-UTUC):

Reported net product revenue of JELMYTO for the third quarter 2022 of $16.1 million, compared to $11.4 million in the third quarter of 2021, representing an increase of 41% from the same period last year.
Activated sites and repeat accounts as of November 1 were 930 and 177, respectively as compared to 893 and 144 on August 1, 2022.
FDA authorized an extension of the in-use period for JELMYTO admixture from 8 hours to 96 hours following reconstitution. This extension expands access to JELMYTO and gives urologists greater flexibility in choosing when to reconstitute and schedule instillations, including early morning instillation which we estimate is preferred by nearly all prescribing HCPs.
A multi-center retrospective analysis published in the British Journal of Urology International by Kyle Rose, M.D. Society of Urologic Oncology Fellow, Moffitt Cancer Center, and colleagues showed that 17 of 32 (59%) patients who received JELMYTO via antegrade administration had no evidence of disease at the primary disease evaluation and did not recur at a median follow up of 13 months. Importantly, ureteral stenosis occurred in just 3 (9%) of treated patients. This retrospective analysis concluded that antegrade administration of JELMYTO demonstrated a favorable safety profile, including a low rate of ureteral stenosis and can be administered without general anesthesia.
UGN-102 (mitomycin) for intravesical solution:

Full enrollment of approximately 220 patients across 90 sites in the single-arm Phase 3 ENVISION pivotal trial of UGN-102, for the treatment of low-grade, intermediate-risk NMIBC is expected to be completed as soon as the end of November 2022.
Hosted thought-leader webinar on UGN-102 and non-muscle invasive bladder cancers on October 18, 2022, which underscored the magnitude of the patient population and high unmet need in LG-IR-NMIBC.
UGN-301 (zalifrelimab) for intravesical solution:

UroGen continues to enroll subjects for its first-in-human, novel, multi-arm Phase 1 clinical trial of UGN-301, the Company’s anti-CTLA4- antibody, in high-grade NMIBC.
This Phase 1 clinical trial will utilize a Master Protocol to evaluate the safety and tolerability of UGN-301 as monotherapy and in combination with other immunomodulators, including UGN-201, the Company’s proprietary toll-like receptor 7 (TLR7) agonist, as well as other potential chemo and/or immune therapies in patients with NMIBC.
Board of Directors Appointments

Announced the appointment of two new independent members to the Board of Directors: Dr. Leana S. Wen, an emergency medicine physician and public health policy expert, and Dan Wildman, a seasoned executive with more than forty years of Medical Device and Pharmaceutical industry experience developing and commercializing meaningful innovations that changed the standard of care, including Auris Heath’s (a division of Ethicon Inc.) Monarch robotic surgery system
Third Quarter 2022 Financial Results:

Jelmyto Revenue: UroGen reported net product revenue of JELMYTO for the third quarter 2022 of $16.1 million, compared to $11.4 million in the third quarter of 2021, representing an increase of 41% from the same period last year.

R&D Expense: Research and development expenses for the third quarter 2022 were $13.1 million, including non-cash share-based compensation expense of $0.6 million as compared to $11.9 million, including non-cash share-based compensation expense of $1.0 million, for the same period in 2021.

SG&A Expense: Selling, general and administrative expenses for the third quarter 2022 were $19.1 million, including non-cash share-based compensation expense of $1.8 million. This compares to $21.6 million, including non-cash share-based compensation expense of $4.5 million, for the same period in 2021.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.8 million for the third quarter 2022.

Interest Expense on Long-Term Debt: Interest expense related to the up to $100 million term loan facility with funds managed by Pharmakon Advisors was $2.7 million for the third quarter of 2022. As the transaction closed in March 2022, there was no such expense in the third quarter of 2021.

Net Loss: UroGen reported a net loss of $25.8 million, or basic and diluted net loss per ordinary share of $1.13, for the third quarter 2022 as compared to $30.2 million, or basic and diluted net loss per ordinary share of $1.35, for the same period in 2021.

Cash & Cash Equivalents: As of September 30, 2022, cash, cash equivalents and marketable securities totaled $95.9 million. This includes the first $75 million tranche of the up to $100 million term loan facility with funds managed by Pharmakon Advisors, which closed in March 2022.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review the Company’s financial results and provide a general business update. The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source

About Jelmyto

Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters or nephrostomy tube. The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade intermediate risk NMIBC. Utilizing the RTGelTM Technology Platform, UroGen’s proprietary 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. The Company presented results from the Phase 2b OPTIMA II trial in September 2021.

About the Phase 3 ENVISION Trial

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) as primary chemoablative therapy in patients with low-grade, intermediate-risk NMIBC. The Phase 3 ENVISION trial is expected to enroll approximately 220 patients across 90 sites and study participants will receive six once-weekly intravesical instillations of UGN-102. The planned primary endpoint will evaluate the complete response rate at three months after the first installation, and the key secondary endpoint will evaluate durability over time in patients who achieve complete response at the three-month assessment. Based on discussions with the FDA, and enrollment expected by the end of 2022, assuming positive findings, UroGen anticipates submitting an NDA for UGN-102 in 2024.