On November 14, 2022 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company reported financial results for the third quarter ended September 30, 2022 (Press release, Alaunos Therapeutics, NOV 14, 2022, View Source [SID1234624032]).

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"Our team has worked diligently over the past year to transform our promising technology and scientific foundation into meaningful clinical progress. We were excited to present early data from our TCR-T Library Phase 1/2 trial at CICON, where we showed for the very first time, an objective clinical response in a solid tumor using a non-viral TCR-T cell therapy. These initial safety, persistence and efficacy data reinforce the promise of our Sleeping Beauty TCR-T cell therapy to safely achieve measurable regression in solid tumors, even at the lowest dose," commented Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. "In tandem, we have successfully doubled our manufacturing capacity. We look forward to dosing the next patient in our TCR-T Library Phase 1/2 Trial as well as filing an IND amendment to expand our TCR Library and enhance the speed and flexibility of our manufacturing process using cryopreserved cell products in the fourth quarter."

Recent Developments and Upcoming Milestones

Presented encouraging clinical data from TCR-T Library Phase 1/2 Trial at CRI-ENCI-AACR Sixth International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) (CICON): In September 2022, the Company presented early data from its TCR-T Library Phase 1/2 trial targeting KRAS, TP53, and EGFR mutations across six solid tumor indications. The data represent the first report of a successful TCR-T cell therapy using the non-viral Sleeping Beauty system for solid tumors. The Company expects to dose the next patient in the study in 4Q22.

Key highlights include:

First patient dosed was diagnosed with NSCLC and was treated at dose level 1 with TCR-T cells targeting a KRAS G12D mutation. The patient achieved six-month progression-free survival, with a best overall response of objective, partial regression of greater than 50% of target lesions at 12 weeks post-cell therapy.

Second patient dosed was diagnosed with colorectal cancer and was treated at dose level 2 with TCR-T cells targeting a TP53 R175H mutation. This patient achieved a best overall response of stable disease at six weeks with 12-week progression-free survival.

Persistence of TCR-T cells was evident in both patients. Patient 1 had persistence at 24 weeks with approximately 30% of all T-cells being TCR-T cells in the blood. Patient 2 had persistence at 12 weeks with approximately 20% of all T-cells being TCR-T cells in the blood.

In both patients, the TCR-T cell therapy was well-tolerated and presented a manageable safety profile, with no dose limiting toxicities or immune effector cell-associated neurotoxicity syndrome (ICANS) observed.

Additional information about the trial is available at www.clinicaltrials.gov using the identifier: NCT05194735.

Expanded manufacturing capacity to produce two products simultaneously: The Company continues to execute on its multi-pronged strategy to expand manufacturing capacity. As a result of this initiative, the Company has doubled its existing manufacturing capacity to produce two products simultaneously.

Expect to file IND amendment in 4Q22 to expand its TCR Library and move from a fresh to cryopreserved manufacturing process: Alaunos expects to file an IND amendment in the fourth quarter, which will add two new TCRs to the Company’s TCR Library targeting frequent mutations and HLAs. This should allow the Company to increase the potential addressable market for its T-cell therapies. In addition, the Company has successfully completed process qualification runs using cryopreserved cell products to manufacture TCR-T cells, which reduces manufacturing process time from 30 days to 26 days, a 13% decrease. The IND amendment will enable the Company to move to a cryopreserved manufacturing process and add flexibility for patient scheduling and treatment.

Presented data highlighting potential of the Company’s hunTR platform to expand its TCR Library at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 37th Annual Meeting: In November 2022, the Company presented a poster at the SITC (Free SITC Whitepaper) annual meeting, highlighting its proprietary hunTR (human neoantigen T-cell Receptor) platform. hunTR is a high-throughput screening process that uses state-of-the-art bioinformatics and next generation sequencing to interrogate and deconvolute thousands of single T cells simultaneously. In the study, Alaunos evaluated hundreds of thousands of TCR+HLA+neoantigen permutations in nine patients across colorectal, endometrial and breast cancers. All patients screened had at least one detectable neoantigen-reactive TCR, including one shared KRAS mutation. Further screening of additional patients only for KRAS mutations resulted in discovery of KRAS-G12V reactive TCRs. The Company plans to expand the application of hunTR to screen for additional shared KRAS, TP53, and EGFR mutations in order to rapidly advance new TCR library candidates from the lab through to clinical translation.

Third Quarter Ended September 30, 2022 Financial Results

Collaboration Revenue: Collaboration revenue was $2.9 million for the third quarter of 2022, compared to $0.4 million for the third quarter of 2021, an increase of 631%. The increase was primarily due to the achievement of sales-based milestones of darinaparsin in Japan, which was largely offset by a one-time corresponding $2.5 million Research & Development expense.

Research and Development Expenses: Research and development expenses were $7.9 million for the third quarter of 2022, compared to $14.5 million for the third quarter of 2021, a decrease of approximately 46%. Research and Development expenses during the third quarter of 2022 included a one-time $2.5 million expense as a result of the achievement of sales-based milestones of darinaparsin in Japan.

General and Administrative Expenses: General and administrative expenses were $3.3 million for the third quarter of 2022, compared to $8.2 million for the third quarter of 2021, a decrease of approximately 60%.

Net Loss: Net loss was $8.9 million, or $(0.04) per share, for the third quarter of 2022, compared to a net loss of $22.7 million, or $(0.11) per share, for the same period in 2021.

Cash and Cash Equivalents: As of September 30, 2022, Alaunos had approximately $37.8 million in cash and cash equivalents and restricted cash of $13.9 million. Operating cash burn for the third quarter of 2022 was $6.1 million compared to $9.6 million in the third quarter of 2021, a decrease of $3.4 million or 36%.

Conference Call and Webcast

Alaunos will host a conference call and webcast today, November 14, 2022, at 8:30am ET. Participants may access the live webcast using the link here or by visiting the "Investors" section of the Alaunos website at www.alaunos.com. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. After the live webcast, the event will be archived on the Company’s website for approximately 30 days after the call.

On November 14, 2022 Yumanity Therapeutics, Inc. ("Yumanity" or the "Company") (Nasdaq: YMTX), a clinical-stage biopharmaceutical company, reported financial results for the third quarter ended September 30, 2022 and provided an overview of the Company’s recent corporate developments (Press release, Yumanity Therapeutics, NOV 14, 2022, View Source [SID1234624031]).

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Recent Corporate Developments

On November 10, 2022, Yumanity announced the effectiveness of a registration statement on Form S-4 (the "Registration Statement") with the U.S. Securities and Exchange Commission (the "SEC") in connection with Yumanity’s previously announced proposed sale of Yumanity’s lead clinical-stage product candidate, YTX-7739, as well as Yumanity’s unpartnered discovery-stage neuroscience product candidates and targets to Janssen Pharmaceutica NV ("Janssen") for $26 million in cash as well as Yumanity’s proposed merger with Kineta, Inc. ("Kineta") under which Kineta will become a wholly-owned subsidiary of Yumanity in an all-stock transaction, resulting in a combined publicly traded company re-named Kineta, Inc. Yumanity subsequently filed a definitive proxy statement and prospectus (the "Proxy Statement") which provides important information about Yumanity and the proposed transactions, both of which were announced on June 6, 2022. Yumanity is mailing the Proxy Statement to stockholders of record as of the close of business on November 4, 2022. The Proxy Statement will be accompanied by a voting instruction form or a proxy card relating to the special meeting of Yumanity’s stockholders to approve the asset sale and merger (the "Special Meeting") which will be held in a virtual-only format via live audio webcast at 10:00 a.m., Eastern Time, on December 13, 2022, at www.virtualshareholdermeeting.com/YMTX2022SM, unless postponed or adjourned to a later date.
Third Quarter 2022 Financial Highlights

Cash position: As of September 30, 2022, cash, cash equivalents and investments were $8.4 million, compared to $36.5 million as of December 31, 2021. The decrease was primarily due to a $12.8 million payment to extinguish the Company’s remaining debt during the first quarter of 2022, spending on the clinical development of YTX-7739 primarily in the first quarter of 2022, and costs related to being a public company. As of the issuance date of the condensed consolidated financial statements for the period ended September 30, 2022, the Company expects that, absent either strategic transaction, its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses early into the first quarter of 2023.

Research and development (R&D) expense: Research and development expense was $0.7 million compared to $6.6 million for the third quarter of 2021. The decrease in R&D expense was primarily due to the elimination of a significant portion of the Company’s R&D personnel in connection with the restructuring announced in February 2022 as well as pausing of clinical development efforts for YTX-7739 while the U.S. Food and Drug Administration partial clinical hold is in effect.

General and administrative expense: General and administrative expense was $4.2 million compared to $4.5 million for the third quarter of 2021. The decrease was primarily attributable to lower personnel costs resulting from the restructuring announced in February 2022, offset by higher legal fees and investment banking fees incurred in connection with the transactions contemplated by the merger agreement with Kineta and the asset purchase agreement with Janssen.

Net loss: The Company reported a net loss of $3.4 million, or $0.31 per basic and diluted share, compared to a net loss of $10.0 million, or $0.97 per basic and diluted share for the third quarter of 2021. The decrease in net loss was due primarily to the decrease in R&D expense as described above. The Company expects to continue to generate operating losses for the foreseeable future, although at reduced expected levels as a result of restructuring actions taken in the nine months ended September 30, 2022.

On November 14, 2022 VBL Therapeutics (Nasdaq: VBLT), a clinical stage biotechnology company developing targeted medicines for immune-inflammatory diseases, reported financial results for the third quarter ended September 30, 2022, and provided a corporate update (Press release, VBL Therapeutics, NOV 14, 2022, View Source [SID1234624030]).

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"VBL’s management team and board continue to make good progress on the evaluation of strategic options for the company’s assets, including the GMP manufacturing facility and VB-601 program, with the goal of maximizing shareholder value," said Dror Harats, M.D., Chief Executive Officer of VBL. "We recently submitted our regulatory filings for a first-in-human clinical trial for the VB-601 program, and expect to initiate this trial in the first quarter of 2023, subject to the outcome of our strategic process."

Third Quarter Highlights

●Filed a regulatory submission to the Israel Ministry of Health and institutional review board to conduct a Phase 1 first-in-human trial for its lead immunology product candidate,VB-601, a targeted antibody for immune-inflammatory applications.
●Retained Chardan Capital to act as financial advisor to explore and evaluate strategic options for maximizing shareholder value.
●Took steps to preserve capital, including the workforce reduction and ceasing internal development of ofra-vec.

Financial Results for the Third Quarter of 2022

●At September 30, 2022, VBL had cash, cash equivalents, short-term bank deposits and restricted bank deposits of $27.7 million. VBL expects that its cash, cash equivalents, short-term bank deposits, and restricted bank deposits will be sufficient to fund currently planned operating expenses and capital expenditures for at least 12 months. VBL’s ongoing review of its strategic options and any transactions resulting from such review may impact this projection.
●For the quarter ended September 30, 2022, VBL reported a net loss of $9.2 million, or ($0.12) per basic share, compared to a net loss of $6.5 million, or ($0.09) per basic share, in the comparable period in 2021.
●Revenues for the quarter ended September 30, 2022, were $0.5 million, as compared to $0.2 million in the comparable period in 2021.
●For the quarter ended September 30, 2022, total operating expenses were approximately $9.7 million, consisting of $6.0 million in research and development expenses, net, and $3.7 million in general and administrative expenses. This compares with total operating expenses of $6.6 million in the quarter ended September 30, 2021, which was comprised of $5.0 million in research and development expenses, net, and $1.6 million in general and administrative expenses.

On November 14, 2022 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported financial results for the third quarter ended September 30, 2022 and provided a corporate update on key programs (Press release, Vaccinex, NOV 14, 2022, View Source [SID1234624029]).

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"Vaccinex continues to advance clinical development of our proprietary product candidate, pepinemab, a SEMA4D inhibitor, in oncology and neurodegenerative disease," said Maurice Zauderer, Ph.D., President and Chief Executive Officer of Vaccinex. "In the open label, Phase 1b/2 KEYNOTE B84 trial (NCT04815720) to evaluate immunotherapy with pepinemab and KEYTRUDA (pembrolizumab, a PD-1 inhibitor) in patients with recurrent or metastatic head and neck cancer (R/M HNSCC), we initially reported 2 confirmed complete responses (CRs) in patients whose tumors expressed low levels of PD-L1 biomarker (CPS<20), a subset of HNSCC patients who have had historically low response rates to anti-PD-1/L1 antibodies administered as single agents. We continue to observe a pattern of improved responses in this difficult to treat patient population and expect to report results of interim analysis from this study in 1Q 2023."

Dr. Zauderer continued, "Vaccinex is also very pleased to publish the full results of the SIGNAL phase 2 study of pepinemab treatment in Huntington’s disease (HD) in Nature Medicine (available here). While multiple prior clinical interventions in HD, including several recent trials of genetic interventions that aimed to inhibit expression of mutant huntingtin protein, were widely reported to have failed to provide benefit to patients, we believe pepinemab is the only clinical intervention to date that shows promise of preventing or reducing cognitive decline in HD patients. A major challenge in HD drug development has been that, in contrast to Alzheimer’s disease, there is currently no established measure of cognition in HD that is accepted as intrinsically meaningful by US and European regulators. We believe the SIGNAL study provided compelling evidence that the Huntington’s Disease-Cognitive Assessment Battery (HD-CAB) can be a useful measure of the ability to learn, which we believe is intrinsically meaningful to patients. We are currently preparing briefing materials and a request for a meeting with FDA in early 2023 to discuss (i) use of HD-CAB as a surrogate endpoint likely to predict clinical benefit, and that could potentially support accelerated approval, and (ii) incorporation of HD-CAB in the design of a larger confirmatory pivotal study to support regular approval. Separately, we expect to complete enrollment of patients in the randomized, double-blind, Phase 1/2a SIGNAL-AD clinical trial in Alzheimer’s Disease (NCT04381468) in 1H 2023 with topline results of 12-months treatment with pepinemab expected in 2024."

Recent Milestones:
Oncology:

Data from an investigator-sponsored study at The Winship Cancer Institute of Emory University evaluating neoadjuvant treatment with pepinemab in combination with nivolumab and/or ipilimumab in resectable Stage III melanoma followed by surgical resection and adjuvant treatment with nivolumab alone (NCT03769155) was presented at the 2022 European Society for Medical Oncology (September 2022). 100% of patients who received neoadjuvant treatment with the triple combination of pepinemab, nivolumab and ipilimumab were recurrence free at 24 months. This contrasted with recurrence free survival of less than 40% in patients who received neoadjuvant treatment with the dual combination of pepinemab and nivolumab or pepinemab and ipilimumab.
Neurodegenerative Disease:

Full results of the Phase 2 SIGNAL-HD study were published in Nature Medicine (Feigin et al., August 2022)
Results of detailed pepinemab mechanism of action studies in neurodegenerative disease were published in the Journal of Neuroinflammation (Evans et al., August 2022)
Posters related to the ongoing Phase 1/2a SIGNAL-AD study were presented at the Alzheimer’s Association International Conference (July 2022)
Poster related to the Phase 2 SIGNAL study of pepinemab as a treatment for early HD was presented at the European Huntington’s Disease Network 2022 (September 2022)
ActivMAb Platform Technology:

The company is engaged in multiple biopharmaceutical collaborations employing this enabling technology for drug discovery. An IND for the first such clinical product is expected to be filed in 2023.
Upcoming Milestones:
Head and Neck Cancer: Phase 1b/2 KEYNOTE B84 study and Oncology updates

Presentation of biomarker data from the investigator-sponsored study evaluating neoadjuvant pepinemab in combination with nivolumab and/or ipilimumab in resectable Stage III melanoma to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2022: November 11, 2022
Poster related to "Phase I Study of Adoptive T Cell Therapy Following HER2-Pulsed Dendritic Cell Vaccine and Pepinemab/Trastuzumab in Patients with Metastatic HER2-Positive Breast Cancer (MBC)" presented at SITC (Free SITC Whitepaper) 2022: November 10-11, 2022
Interim analysis of KEYNOTE B84: Expected Q1 2023
Alzheimer’s Disease: Phase 1/2a SIGNAL-AD study

Completion of enrollment: Expected in H1 2023 with topline results of 12-months treatment with pepinemab expected in 2024.
Financial Results for the Three Months Ended September 30, 2022:
Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on September 30, 2022 were $7.2 million, as compared to $8.6 million as of December 31, 2021.

Research and Development Expenses. Research and development expenses for the quarter ended September 30, 2022 were $3.4 million as compared to $3.6 million for the comparable period in 2021.

The essentially flat level of research and development expenses reflects consistent clinical trial costs to support the pepinemab Phase 1b/2 KEYNOTE B84 study in R/M HNSCC and Phase 1/2a study in Alzheimer’s Disease study along with continued careful cost control measures.

General and Administrative Expenses. General and administrative expenses for the quarter ended September 30, 2022 were $1.4 million as compared to $1.5 million for the comparable period in 2021.

Essentially flat level of general and administrative expenses reflects careful cost control measures.

Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the quarter ended September 30, 2022 was $4.8 million and $(0.11) compared to $5.2 million and $(0.17) for the comparable period in 2021.

Full financial tables are included below. For further details on Vaccinex’s financials, refer to its Form 10Q filed November 14, 2022 with the S.E.C.

About Pepinemab

Pepinemab is a humanized IgG4 monoclonal antibody that inhibits SEMA4D, which regulates the actin cytoskeleton of cells that plays an important role in tumor immunity and in inflammatory reactions in the brain. Preclinical and clinical data show that by preventing inflammatory reactivity of pepinemab during disease progression, pepinemab preserves normal function of astrocytes and microglia, two types of glial cells that play a crucial role in the development and maintenance of neurons in the brain. Additional data show that pepinemab promotes infiltration and activation of dendritic cells and CD8+ T-cells and reverses immunosuppression within the tumor microenvironment. Pepinemab is being evaluated in several studies in oncology and neurodegenerative disease.

About ActivMAb
Vaccinex has developed a proprietary mammalian cell-based antibody discovery platform with unique multi-pass membrane target capabilities. The ActivMAb technology now has four main applications: complex membrane antigen presentation, antibody or antigen discovery, and protein optimization. Vaccinex has entered into an antibody license with Surface Oncology (Cambridge, MA) and into Material Transfer Agreements for drug discovery or process development with two major pharmaceutical companies utilizing this technology.

On November 14, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that highlights results from a retrospective multi-center study of 32 patients evaluating the safety of antegrade administration of JELMYTO (mitomycin) for pyelocalyceal solution via percutaneous nephrostomy tube for the treatment of patients with upper tract urothelial carcinoma (UTUC) (Press release, UroGen Pharma, NOV 14, 2022, View Source [SID1234624028]). The study titled, "Antegrade Administration of Mitomycin Gel for UTUC via Percutaneous Nephrostomy Tube: A Multi-Institutional Retrospective Cohort Study," provides additional real-world evidence of a favorable safety and tolerability profile for JELMYTO when administered via percutaneous nephrostomy tube and is published in The British Journal of Urology International, November Issue.

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This retrospective study assessed the safety and feasibility of antegrade administration of JELMYTO via a percutaneous nephrostomy tube (PCNT) in 32 patients from four institutions. Each patient received at least one dose of JELMYTO via PCNT for UTUC, 29 of whom completed induction (at least 5 of 6 doses) and underwent primary disease evaluation (PDE) at a median 17 weeks after the last dose of therapy. At a median follow-up of 15 months following initiation of induction therapy, ureteral stenosis (defined as a discrete narrowing of the ureter on direct visual ureteroscopy, or a constriction identified on retrograde pyelogram at the time of ureteroscopy that required dilation or stenting to pass a ureteroscope for upstream visualization) occurred in three (9%) of patients. None of these patients had recurrent stenosis at a median 16 months follow- up. Other adverse events included fatigue (27%), flank pain (19%), urinary tract infection (12%), sepsis (8%), and hematuria (8%). No patient had impaired renal function during follow-up and there were no treatment-related deaths. Seventeen patients (59%) had no evidence of disease at PDE and had not recurred at a median follow-up of 13 months post induction.

"We found that antegrade administration of JELMYTO via a nephrostomy tube offered a low rate of ureteral stenosis and a favorable safety and tolerability profile overall," said Kyle Rose, M.D., Urologic Oncology Fellow at Moffitt Cancer Center in Tampa, Fla., and study investigator. "This study adds to the evidence that this form of administration is beneficial in many ways, including the ability to treat the elderly without the use of anesthesia, which can have negative cumulative effects when used repetitively."

In both retrograde and antegrade approaches, JELMYTO can be administered as an outpatient procedure in the clinic. Retrograde administration requires administration by a physician via a ureteral catheter which requires fluoroscopic guidance. Antegrade administration may be performed by trained nursing professionals under clean rather than sterile conditions and does not require fluoroscopy once tube placement is confirmed via nephrostogram at the first instillation.

"JELMYTO is approved for antegrade and retrograde administration in patients with LG-UTUC, which gives physicians the added convenience of choosing a method that works best for them and their patients," said Mark Schoenberg, Chief Medical Officer, UroGen. "Retrograde administration was the only method used in the pivotal OLYMPUS trial. Therefore, UroGen is pleased that this study adds to the growing body of real-world evidence demonstrating that antegrade administration of JELMYTO is a feasible alternative for patients with LG-UTUC."

About the Retrospective Study

In this multi-center study, patients undergoing antegrade administration of JELMYTO via PCNT were retrospectively included for analysis from four tertiary referral centers between 2020 and 2022. The primary outcome was safety profile graded by Common Terminology Criteria for Adverse Events (v5.0). Post-therapy disease burden was assessed by primary disease evaluation (PDE) via ureteroscopy. Thirty-two patients received at least one dose of JELMYTO via PCNT for UTUC, 29 of whom completed induction and underwent PDE. Thirteen (41%) patients had residual tumor present prior to induction therapy. At a median follow up of 15 months following first dose of induction therapy, ureteral stenosis occurred in 3 (9%) patients, all of which were treated without later recurrence or chronic stenosis. Other adverse events included fatigue (27%), flank pain (19%), UTI (12%), sepsis (8%), and hematuria (8%). No patients had impaired renal function during follow up and there were no treatment related deaths. Seventeen patients (59%) had no evidence of disease at PDE and had not recurred at a median follow up of 13 months post induction.

Limitations of this study include the retrospective nature and sample size. Some complications and side effects of the antegrade approach were contingent on patient reporting, and thus may be under-reported. Ureteral stenoses were noted incidentally at the time of PDE, and no patients presented with obstructive symptoms. There is a need for larger studies with longer follow-up to study more conclusively any potential advantages of antegrade JELMYTO administration when compared to retrograde instillation.

About the Pivotal OLYMPUS Study

OLYMPUS (Optimized DeLiverY of Mitomycin for Primary UTUC Study) was an open-label, single-arm Phase 3 clinical study of UGN-101 JELMYTO (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of JELMYTO in patients with low-grade Upper Tract Urothelial Cancer UTUC (LG UTUC). Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of JELMYTO administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were eligible for the maintenance phase of the trial, during which they could receive monthly maintenance instillations for up to 12 months and were assessed to determine the durability of response with JELMYTO.

In the OLYMPUS study, data was generated for the retrograde administration of JELMYTO. In that study, population ureteric obstruction was reported in 58% (n=41) of patients receiving JELMYTO, including 17% (n=12) of patients who experienced Grade 3 obstruction.

About LG UTUC

LG UTUC is a rare disease managed by endoscopic methods and radical nephroureterectomy. Endoscopic resection and laser ablation attempt to preserve the kidney, though there is a high risk of recurrence that may eventually necessitate removal of the kidney. Although kidney removal is the gold standard for treatment of high-grade UTUC, it may be over-treatment in LG UTUC, as kidney removal offers similar five-year survival as kidney-sparing procedures but is associated with significant morbidity. JELMYTO is efficacious as a primary chemoablative therapy in patients with LG UTUC.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for primary chemoablative treatment of LG UTUC in adults. It is recommended for primary treatment of biopsy-proven LG UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

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 U.S. Food and Drug Administration. 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.