On July 10, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the results of a sub-analysis from the first post-commercial utilization review of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, JUL 10, 2023, View Source [SID1234633144]). This study assessed the efficacy of JELMYTO in managing patients with larger volume disease where initial tumor burden may not be amenable to complete mechanical ablation or renal preservation. Findings from the study titled, The Ablative Effect of Mitomycin Reverse Thermal Gel: Expanding the Role for Nephron Preservation Therapy in Low Grade Upper Tract Urothelial Carcinoma, are published in Urologic Oncology: Seminars and Original Investigations online.

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The goal of this study was to explore alternatives to nephroureterectomy for patients with higher-volume low-grade disease to preserve kidney function and minimize complications. This study aimed to determine if higher volume, low-grade upper tract urothelial tumors (LG-UTUC) patients could achieve disease-free status by undergoing partial ablation or biopsy only before treatment with JELMYTO during the initial ureteroscopy (URS) procedure.

In this study of 116 patients from 15 centers no significant differences were found in the rates of rendered disease free (RDF) among those who underwent complete ablation (78.6%), partial ablation (57.6%), or biopsy only (66.7%) during the initial URS procedure (p=0.15). RDF was defined as patients with absence of any tumor or an almost complete response requiring only minimal mechanical ablation to render the upper tract clear of visible disease at first URS after JELMYTO.

The analysis also showed that tumor size prior to JELMYTO induction did not have a significant impact on RDF rates (p=0.09). Tumor size was <1 cm for completely ablated, 1-3 cm for partial ablation or >3 cm for biopsy only. Among the 112 renal units evaluated, 50 underwent complete ablation, 42 underwent partial ablation and 20 underwent biopsy only.

In the Olympus trial, patients were required to have a tumor size of 5 to 15 mm prior to enrollment, and lesions larger than 15 mm were eligible for endoscopic downsizing prior to primary treatment with JELMYTO. Thirty-seven percent (37%) of patients who underwent endoscopic downsizing before enrollment and 58% of Olympus patients achieved a complete response with JELMYTO treatment, defined as the complete absence of tumor three months after initiation of JELMYTO treatment.

"Implementing JELMYTO earlier in real-world scenarios for LG-UTUC may offer appropriate low-risk patients the choice of minimal ablation or biopsy alone for renal preservation," said Hristos Kaimakliotis, M.D., Associate Professor of Urology, Indiana University Medical Center, Indianapolis, IN. "This kidney-sparing approach aligns with the first-ever UTUC AUA/SUO Guideline, which includes JELMYTO and strongly recommends tumor ablation as the initial management approach for low-grade favorable UTUC."

Dr. Mark Schoenberg, M.D., Chief Medical Officer at UroGen, commented, "The findings are encouraging and provide evidence in support of a new treatment strategy utilizing JELMYTO for reducing the volume of larger low-grade tumors to help spare the kidney."

The primary outcome of this study was RDF rate at first post-JELMYTO URS, defined as complete response or partial response with minimal mechanical ablation to endoscopically clear the upper tract of visible disease. While acknowledging the limitations of the study, such as its retrospective design, variability in patient management and institution recordkeeping, lack of follow-up and small number of recurrences, the researchers also noted that additional research is required to better quantify the chemoablative impact and determine the relevant clinical factors for patient selection.

To address some of these areas, investigators are currently enrolling patients in the prospective and retrospective uTRACT Registry, which aims to capture data in a large-scale, standardized manner and provide more comprehensive insights into patient outcomes following JELMYTO treatment, including long-term follow-up.

About UTUC

Approximately 5-7% of urothelial cancer occurs in the upper lining of the kidney, called the calyx and renal pelvis. It could also occur in one or both of the ureter(s), the tubes that lead from the kidneys to the bladder. Cancer in the renal pelvis or ureter(s) is called upper tract. LG-UTUC is usually not very aggressive and is slow to spread but has a high recurrence rate. High-grade UTUC can be more aggressive. It may spread to other parts of the urinary tract, or to other parts of the body.

JELMYTO is approved for the treatment of adults with 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.

Please click here for JELMYTO Full Prescribing Information, including the Patient Information, for additional information and here for the Nephrostomy Administration Guide.

On July 10, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the results of a sub-analysis from the first post-commercial utilization review of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, JUL 10, 2023, View Source [SID1234633144]). This study assessed the efficacy of JELMYTO in managing patients with larger volume disease where initial tumor burden may not be amenable to complete mechanical ablation or renal preservation. Findings from the study titled, The Ablative Effect of Mitomycin Reverse Thermal Gel: Expanding the Role for Nephron Preservation Therapy in Low Grade Upper Tract Urothelial Carcinoma, are published in Urologic Oncology: Seminars and Original Investigations online.

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The goal of this study was to explore alternatives to nephroureterectomy for patients with higher-volume low-grade disease to preserve kidney function and minimize complications. This study aimed to determine if higher volume, low-grade upper tract urothelial tumors (LG-UTUC) patients could achieve disease-free status by undergoing partial ablation or biopsy only before treatment with JELMYTO during the initial ureteroscopy (URS) procedure.

In this study of 116 patients from 15 centers no significant differences were found in the rates of rendered disease free (RDF) among those who underwent complete ablation (78.6%), partial ablation (57.6%), or biopsy only (66.7%) during the initial URS procedure (p=0.15). RDF was defined as patients with absence of any tumor or an almost complete response requiring only minimal mechanical ablation to render the upper tract clear of visible disease at first URS after JELMYTO.

The analysis also showed that tumor size prior to JELMYTO induction did not have a significant impact on RDF rates (p=0.09). Tumor size was <1 cm for completely ablated, 1-3 cm for partial ablation or >3 cm for biopsy only. Among the 112 renal units evaluated, 50 underwent complete ablation, 42 underwent partial ablation and 20 underwent biopsy only.

In the Olympus trial, patients were required to have a tumor size of 5 to 15 mm prior to enrollment, and lesions larger than 15 mm were eligible for endoscopic downsizing prior to primary treatment with JELMYTO. Thirty-seven percent (37%) of patients who underwent endoscopic downsizing before enrollment and 58% of Olympus patients achieved a complete response with JELMYTO treatment, defined as the complete absence of tumor three months after initiation of JELMYTO treatment.

"Implementing JELMYTO earlier in real-world scenarios for LG-UTUC may offer appropriate low-risk patients the choice of minimal ablation or biopsy alone for renal preservation," said Hristos Kaimakliotis, M.D., Associate Professor of Urology, Indiana University Medical Center, Indianapolis, IN. "This kidney-sparing approach aligns with the first-ever UTUC AUA/SUO Guideline, which includes JELMYTO and strongly recommends tumor ablation as the initial management approach for low-grade favorable UTUC."

Dr. Mark Schoenberg, M.D., Chief Medical Officer at UroGen, commented, "The findings are encouraging and provide evidence in support of a new treatment strategy utilizing JELMYTO for reducing the volume of larger low-grade tumors to help spare the kidney."

The primary outcome of this study was RDF rate at first post-JELMYTO URS, defined as complete response or partial response with minimal mechanical ablation to endoscopically clear the upper tract of visible disease. While acknowledging the limitations of the study, such as its retrospective design, variability in patient management and institution recordkeeping, lack of follow-up and small number of recurrences, the researchers also noted that additional research is required to better quantify the chemoablative impact and determine the relevant clinical factors for patient selection.

To address some of these areas, investigators are currently enrolling patients in the prospective and retrospective uTRACT Registry, which aims to capture data in a large-scale, standardized manner and provide more comprehensive insights into patient outcomes following JELMYTO treatment, including long-term follow-up.

About UTUC

Approximately 5-7% of urothelial cancer occurs in the upper lining of the kidney, called the calyx and renal pelvis. It could also occur in one or both of the ureter(s), the tubes that lead from the kidneys to the bladder. Cancer in the renal pelvis or ureter(s) is called upper tract. LG-UTUC is usually not very aggressive and is slow to spread but has a high recurrence rate. High-grade UTUC can be more aggressive. It may spread to other parts of the urinary tract, or to other parts of the body.

JELMYTO is approved for the treatment of adults with 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.

Please click here for JELMYTO Full Prescribing Information, including the Patient Information, for additional information and here for the Nephrostomy Administration Guide.

As previously disclosed, on June 24, 2021, Prothena Corporation plc ("Prothena") reported that Bristol Myers Squibb ("BMS") exercised its option under the terms of the ongoing global neuroscience research and development collaboration (the "Master Collaboration Agreement") to enter into an exclusive U.S. license for PRX005, and, on July 30, 2021, Prothena entered into a U.S. License Agreement (the "Tau U.S. License Agreement") granting BMS the exclusive license to develop, manufacture and commercialize antibody products in the United States targeting tau ("Tau Collaboration Products") for any and all uses or purposes with respect to any human or animal disease, disorder or condition (Filing, 8-K, Prothena, JUL 10, 2023, View Source [SID1234633143]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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On July 5, 2023 and pursuant to the option exercised by BMS under the Master Collaboration Agreement, Prothena and BMS entered into a Global License Agreement (the "Tau Global License Agreement") granting BMS the exclusive license to develop, manufacture and commercialize Tau Collaboration Products globally for any and all uses or purposes with respect to any human or animal disease, disorder or condition. The Tau Global License Agreement supersedes and replaces the Tau U.S. License Agreement in its entirety.

The Tau Global License Agreement includes an exercise fee payable to Prothena of $55 million. The Tau Global License Agreement replaces the regulatory and sales milestones under the Tau U.S. License Agreement, and increases the amounts payable under such milestones from up to $465 million to up to $562.5 million. The tiered royalties on net sales remain the same, and such exercise fees, milestones and royalty payments are subject to certain reductions as specified in the Tau Global License Agreement. In addition, Prothena will be eligible to receive reimbursement of certain chemistry, manufacturing and control (CMC) related expenses incurred by Prothena under the Master Collaboration Agreement, not to exceed $15 million.

Under the Tau Global License Agreement, BMS will continue to pay royalties on a product-by-product and country-by-country basis, until the latest of (i) expiration of certain patents covering the Tau Collaboration Products in such country of sale, (ii) expiration of any applicable regulatory exclusivity in such country of sale, and (iii) an agreed period of time after the first commercial sale of the Tau Collaboration Products in such country of sale (the "Royalty Term").

The term of the Tau Global License Agreement will continue on a product-by-product and country-by-country basis until the expiration of all Royalty Terms with respect to all Tau Collaboration Products. Either party is entitled to terminate the Tau Global License Agreement for material breach, bankruptcy or safety reasons. Prothena is entitled to terminate the Tau Global License Agreement for a failure by BMS to exercise due diligence with respect to its global rights for the Tau Collaboration Products under the Master Collaboration Agreement, and for certain patent challenges by BMS. The Tau Global License Agreement imposes certain post-termination rights and obligations on the parties, which vary based on the reasons giving rise to the termination.

The foregoing description of the Tau Global License Agreement is not a complete description thereof, and is qualified in its entirety by reference to the actual agreement that will be filed with the Securities and Exchange Commission (the "SEC") as an exhibit to Prothena’s Quarterly Report on Form 10-Q for the quarter ending September 30, 2023.

The foregoing descriptions of (i) the Master Collaboration Agreement and (ii) the Tau U.S. License Agreement are not complete descriptions thereof, and are qualified in their entirety by reference to the actual agreements that are filed with the SEC as Exhibits 10.8 and 10.10, respectively, to Prothena’s Annual Report on Form 10-K filed February 28, 2023.

On July 10, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the independent Data Safety Monitoring Board (DSMB) of the Phase III ASPIRE clinical trial for patients with untreated metastatic pancreatic ductal adenocarcinoma has completed its pre-specified review of safety data for treated patients in the trial (Press release, Panbela Therapeutics, JUL 10, 2023, View Source;utm_medium=rss&utm_campaign=panbela-announces-preliminary-safety-analysis-for-aspire-trial [SID1234633142]). The DSMB has recommended that the study continue without modification. The ASPIRE Trial is a global randomized, double-blind placebo-controlled clinical trial to evaluate ivospemin in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma. "We are pleased that no safety concerns were identified and the DSMB’s recommendation is to proceed without modification to the ASPIRE Trial. With a focus on enrollment and completing site initiations, we are looking forward to the interim analysis in early 2024." said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.

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About Panbela’s Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of anticipated catalysts with programs ranging from pre-clinical to registration studies.

Ivospemin (SBP-101)

Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial.

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increasing polyamine export and catabolism. In a Phase III clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase III trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose powder sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase I or Phase II investigatorinitiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

On July 10, 2023 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported preliminary financial and operational highlights for the second quarter ended June 30, 2023 (Press release, NanoString Technologies, JUL 10, 2023, View Source [SID1234633141]).

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"We are excited to report that our teams delivered strong preliminary revenue results for the second quarter, with preliminary results exceeding consensus estimates and the upper end of our guidance ranges," said Brad Gray, President and CEO of NanoString. "Customers are focused on the potential for spatial analysis to advance science, and value the RNA-plex and protein capabilities our spatial platforms provide. During Q2, a competitor launched a campaign using a ruling in the Munich regional court to try to frighten customers to cancel orders for our CosMx Spatial Molecular Imager, even though the preliminary ruling was limited to Germany. Despite this challenge, through the strength of our technology and the hard work of our team, we fulfilled or retained approximately 97% of our cumulative CosMx instrument orders and captured orders for new CosMx instruments at a faster pace than in Q1."

Preliminary Second Quarter Financial Results

•Revenue is expected to be over $44 million, representing year on year growth of approximately 37%, above the upper end of our guidance range of $40 to $42 million and the consensus revenue estimate of $41 million
•Spatial biology revenue is expected to be over $23 million, at approximately the midpoint of our guidance range of $23 to $24 million, and in line with the consensus revenue estimate of $23 million
•nCounter revenue, inclusive of all service and other revenue, is expected to be approximately $21 million, above the upper end of our guidance range of $17 to $18 million and the consensus revenue estimate of $18 million
Second Quarter Highlights
Spatial Biology
•Accelerated CosMx shipments during Q2, resulting in Q2 spatial biology instrument revenue growth of approximately 188% year-over-year
•Successfully defended substantial CosMx instrument order book, fulfilling or retaining approximately 97% of cumulative orders as of June 30, 2023
•Captured new orders for spatial biology instruments at a rate exceeding the Q1 pace, and retained more than $30 million in CosMx instrument order backlog as of June 30, 2023
•Recorded spatial biology consumables revenue growth of approximately 44% year-over-year, and approximately $85,000 of annualized pull-through, driven by a steady GeoMx consumable pull-through over a larger installed base supplemented by growing shipments of CosMx consumables

nCounter

•Recorded nCounter consumables revenue growth of approximately 8% year-over-year and approximately $46,000 of annualized pull-through
These preliminary results are based on management’s initial analysis of operations for the quarter ended June 30, 2023 and are subject to further internal review and review by the company’s external auditors.
Guidance
Management reiterated its full year revenue guidance of $175-185 million, representing annual revenue growth of 41% at the midpoint of the guidance range.

Second Quarter Conference Call

The Company plans to release full operating results for the second quarter of 2023 after the close of trading on Thursday, August 3, 2023. Company management will host a conference call beginning at 4:30pm ET to discuss those results and provide updated financial guidance.

Investors and other interested parties should register for the conference call in advance by visiting View Source Following registration, an email confirmation will be sent that includes dial-in details and unique conference call codes for entry. Registration is open throughout the call but to ensure connection for the full call, registration in advance is recommended.

The link to the webcast and audio replay will be made available at the Investor Relations website: nanostring.com.
A replay of the call will be available beginning August 3, 2023, at 7:30pm ET through midnight on August 17, 2023. To access the replay, dial (800) 770-2030 or (647) 362-9199 and reference Conference ID: 72369. The webcast will also be available on the Company’s website for one year following the completion of the call.