On August 8, 2023 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported financial results for the second quarter ending June 30, 2023 and highlighted recent progress (Press release, Verastem, AUG 8, 2023, View Source [SID1234634004]).

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"We made significant advancements in the second quarter, including presenting positive results from the RAMP 201 trial of avutometinib and defactinib in recurrent LGSOC and finalizing the design of the confirmatory Phase 3 trial. Both are important milestones in our plan to file for accelerated approval in LGSOC based on mature results from RAMP 201 and data from the investigator sponsored FRAME trial," said Dan Paterson, President and Chief Executive Officer, Verastem Oncology. "Our work to strengthen our balance sheet will enable our continued progress across our RAMP programs in LGSOC, non-small lung cancer and pancreatic cancer and support continued preparation for a potential commercial launch in LGSOC. We are encouraged by the progress we have made and believe we are well positioned to address significant unmet needs in RAS pathway-driven cancers."

Second Quarter 2023 and Recent Highlights

Low Grade Serous Ovarian Cancer (LGSOC)

The Company finalized the design of the Phase 3 confirmatory trial (RAMP 301) of avutometinib and defactinib in LGSOC versus standard of care (SOC) chemotherapy (pegylated liposomal doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole, anastrozole). RAMP 301 is an international collaboration between The GOG Foundation, Inc. (GOG) and the European Network of Gynaecological Oncological Trial groups (ENGOT) sponsored by Verastem Oncology. The trial will enroll approximately 270 patients who will be randomized to either the combination of avutometinib and defactinib or SOC. The primary endpoint is progression free survival (PFS) by blinded independent central review (BICR). Secondary endpoints include overall response rates, duration of response, disease control rate, safety and tolerability, patient reported outcomes and overall survival.
RAMP 301 is the follow-up confirmatory study being conducted for full regulatory approval in recurrent LGSOC. The Company intends to file for accelerated approval with the U.S. Food and Drug Administration (FDA) for the combination of avutometinib and defactinib based on mature data from the Company’s Phase 2 registration-directed trial, RAMP 201, together with the results of the investigator-initiated FRAME trial.
Data from Part A of the RAMP 201 trial were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting in June. Results included confirmed objective response rates (ORR) by BICR of 45% (13/29; 95% CI: 26%-64%).​ Overall, patients were heavily pretreated with a median of 4 prior systemic regimens (up to 11). Tumor shrinkage was observed in the majority of patients, 86% (25/29). The safety profile was tolerable and consistent with previously reported safety data. These results are consistent with the data that supported the Breakthrough Therapy Designation granted by the FDA for the combination in recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy.
Other Programs

In the Company’s RAMP 203 and RAMP 204 Phase 1/2 clinical trials, the combinations of avutometinib with Amgen’s LUMAKRAS (sotorasib) (RAMP 203) and with Mirati’s KRAZATI (adagrasib) (RAMP 204) are evaluated in patients with KRAS G12C mutant non-small cell lung cancer (NSCLC). RAMP 203 progressed to the recommendation of the Phase 2 dose (avutometinib 4 mg BIW PO and sotorasib 960 mg QD PO) and continues enrollment in Part B dose expansion in patients who are G12C inhibitor treatment naïve and in patients who experienced disease progression on prior G12C inhibitor monotherapy. Dose escalation is ongoing in RAMP 204.
Enrollment is ongoing in the Company’s RAMP 205 Phase 1b/2 clinical trial evaluating avutometinib and defactinib in combination with SOC chemotherapy (GEMZAR (gemcitabine) and ABRAXANE) in patients with metastatic adenocarcinoma of the pancreas. The trial is supported by the Company’s receipt of the first "Therapeutic Accelerator Award" from the Pancreatic Cancer Action Network (PanCAN).
Corporate Updates

Dan Paterson was promoted to President and Chief Executive Officer in July. During his tenure as President and Chief Operating Officer of Verastem Oncology, he spearheaded the acquisition of lead compound avutometinib and led strategic direction designed to accelerate the program’s advancement. In connection with his appointment, Dan was also appointed to the Board of Directors. Dan succeeds Brian Stuglik who has retired from his role as Chief Executive Officer but remains a member of the Company’s Board of Directors and leads the Board’s recently designated Commercialization Committee.
The Company strengthened the balance sheet in June 2023 by raising gross proceeds of approximately $97.8 million in a public offering of 8,489,409 shares of common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase an aggregate of 1,538,591 shares of common stock.
Karin Tollefson was elected to Verastem Oncology’s Board of Directors at the Company’s annual meeting, alongside returning Board members Robert Gagnon and Brian Stuglik. Dr. Tollefson is the Senior Vice President and Head of Global Medical Affairs at Seagen Inc. Karin has 30 years of experience in the pharmaceutical industry and is a proven leader in global oncology development and medical affairs.
Second Quarter 2023 Financial Results

Verastem Oncology ended the second quarter of 2023 with cash, cash equivalents and investments of $183.1 million. Total operating expenses for the three months ended June 30, 2023 (the "2023 Quarter") were $20.3 million, compared to $21.4 million for the three months ended June 30, 2022 (the "2022 Quarter"). Recent historical operating expenses have ranged between $16.0M and $20.0M per quarter, which the Company does not anticipate will change significantly in the near term as the RAMP 301 trial commences.

Research & development expenses for the 2023 Quarter were $12.9 million, compared to $14.9 million for the 2022 Quarter. The decrease of $2.0 million, or 13.4%, primarily resulted from a decrease in drug product and drug substance costs and contract research organization costs.

Selling, general & administrative expenses for the 2023 Quarter were $7.4 million, compared to $6.5 million for the 2022 Quarter. The increase of $0.9 million, or 13.8%, was primarily related to increased consulting and professional fees as well as additional costs in anticipation of a potential launch of avutometinib and defactinib in LGSOC.

Net loss for the 2023 Quarter was $24.3 million, or $1.37 per share (basic and diluted, each as adjusted for the Company’s reverse stock split), compared to net loss of $22.0 million, or $1.41 per share (basic and diluted, each as adjusted for the Company’s reverse stock split) for the 2022 Quarter.

For the 2023 Quarter, non-GAAP adjusted net loss was $18.8 million, or $1.06 per share (diluted, as adjusted for the Company’s reverse stock split), compared to non-GAAP adjusted net loss of $20.1 million, or $1.29 per share (diluted, as adjusted for the Company’s reverse stock split) for the 2022 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP adjusted net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three and six months ended June 30, 2023, and 2022 are included in the tables accompanying this press release, after the unaudited condensed consolidated financial statements.

About Avutometinib (VS-6766)

Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS pathway inhibition. Avutometinib is currently in late-stage development.

In contrast to other MEK inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK clamp avutometinib, with defactinib, its FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy.

Verastem Oncology is currently conducting clinical trials with its RAF/MEK clamp avutometinib in RAS- driven tumors as part of its (Raf And Mek Program). RAMP 201 is a registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC. Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) and KRAZATI (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively. As part of the "Therapeutic Accelerator Award" Verastem Oncology received from PanCAN, Verastem Oncology is conducting RAMP 205, a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer.

On August 8, 2023 XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its second quarter 2023 financial results and highlighted recent operational achievements as XOMA accelerates its differentiated biotech royalty and milestone acquisition strategy (Press release, Xoma, AUG 8, 2023, View Source [SID1234633997]).

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"XOMA is increasingly being recognized for enabling innovative biotech companies to unlock value in the face of a challenging financing environment. To date, the team has added three new assets to XOMA’s royalty and milestone portfolio to complement the growing cash flows generated by VABYSMO (faricimab)," stated Owen Hughes, Executive Chairman of XOMA. "And over the ensuing quarters, the potential approval of arimoclomol, a first-in-class molecule for Niemann-Pick Disease Type C (NPC) and tovorafenib for relapsed or progressive pediatric low-grade glioma, as well as the expansion of IXINITY’s label into the pediatric population with Hemophilia B, should address critical unmet needs for patients and drive increasing cash flows for XOMA shareholders."

"We see a great opportunity to continue expanding XOMA’s portfolio and building shareholder value, as the biotech industry’s desire for non-dilutive capital is greater than ever. Our team has been sourcing and reviewing more royalty and milestone acquisition opportunities than ever," said Brad Sitko, Chief Investment Officer of XOMA. "We are acting quickly on these opportunities to continue building XOMA’s portfolio with value-creating acquisitions."

Second Quarter 2023 Financial Results

XOMA recorded total revenues of $1.7 million for the second quarter of 2023 and $1.0 million for the second quarter of 2022. The increase for the three months ended June 30, 2023, as compared to the same period in 2022, was primarily due to $1.1 million of milestones earned during the quarter pursuant to the license agreement with Janssen.

Research and development ("R&D") expenses were $39,000 and $40,000, respectively, for the second quarters of 2023 and 2022.

General and administrative ("G&A") expenses were $5.8 million and $5.7 million, respectively, for the second quarters of 2023 and 2022. The second quarter of 2023 included an increase of $1.3 million in stock-based compensation expense and was offset by a decrease of $1.1 million in legal and consulting costs.

As a result of the announcement by Bioasis to suspend its operations and the termination of Bioasis’ research collaboration and license agreement with Chiesi, XOMA recorded a one-time, non-cash impairment charge of $1.6 million and a reduction of $1.6 million under long-term royalty receivables in the second quarter of 2023.

In the second quarter of 2023, G&A expenses included $2.2 million in non-cash stock-based compensation expense, as compared with $0.8 million in non-cash stock-based compensation expense in the second quarter of 2022. XOMA’s net cash used in operations in the second quarter of 2023 was $7.2 million, which included a one-time arbitration settlement cost of $4.1 million, as compared with $4.3 million during the second quarter of 2022.

Other income, net was $0.6 million for the second quarter of 2023, compared to other income, net of $0.1 million in the corresponding quarter of 2022. The $0.5 million difference primarily is due to an increase of $0.4 million in investment income combined with the change in fair value of $75,000 for the contingent consideration related to Bioasis.

Net loss for the second quarter of 2023 was $5.4 million, compared to net loss of $4.7 million for the second quarter of 2022.

On June 30, 2023, XOMA had cash and cash equivalents of $31.4 million. On July 17, 2023, the Company paid cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) equal to $0.53906 per share and cash dividends on the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO) equal to $0.52344 per depositary share. The Company ended December 31, 2022, with cash and cash equivalents of $57.8 million. Due to recent communications from several partners related to delays in achieving specific milestones that are associated with payments to XOMA, the Company currently anticipates lower cash receipts from milestone payments in 2023 than previously expected. Based upon the cash flows XOMA expects to receive from VABYSMO and IXINITY sales in addition to its current cash position, the Company continues to believe its current cash position will be sufficient to fund XOMA’s operations for multiple years.

On August 8, 2023 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported the publication of clinical data from an open-label, multicenter, Phase 1b/2 study of Nana-val in 55 patients with R/R EBV+ lymphoma in Blood Advances. Results showed complete responses (CRs) achieved and ongoing durable responses observed out to approximately 36 months across multiple EBV+ lymphoma subtypes, including some of the most aggressive cancers: peripheral T-cell lymphoma (PTCL), diffuse large B-cell lymphoma (DLBCL), and post-transplant lymphoproliferative disease (PTLD) (Press release, Viracta Therapeutics, AUG 8, 2023, View Source [SID1234633996]). This paper titled, "Targeted therapy with nanatinostat and valganciclovir in recurrent Epstein-Barr virus-positive lymphoid malignancies: a Phase 1b/2 study," can be found here.

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"Results from both retrospective and prospective clinical studies link EBV-positivity to significantly inferior survival in multiple lymphoma subtypes, highlighting the pressing need for novel therapies for this underserved patient population," said Pierluigi Porcu, M.D., Professor of Medical Oncology, Director of the Division of Hematologic Malignancies and Hematopoietic Stem Cell Transplantation, Department of Medical Oncology at Thomas Jefferson University, and corresponding author on the paper. "The newly published Phase 1b/2 study data showcase the potential of Viracta’s innovative ‘Kick and Kill’ approach to effectively address this need, with results demonstrating Nana-val’s favorable safety and tolerability profile and promising durable signal of efficacy in heavily pre-treated patients. These data served as a catalyst for the advancement of Nana-val into the confirmatory NAVAL-1 trial, which has an elegant multi-stage design to potentially support registration."

Darrel P. Cohen, M.D., Ph.D., Viracta’s newly appointed Chief Medical Officer added, "Having these Phase 1b/2 clinical trial data peer-reviewed and published in such a prestigious journal of the American Society of Hematology (ASH) (Free ASH Whitepaper) provides important external validation for Nana-val’s therapeutic potential. The substantial number, depth, and durability of Nana-val’s clinical responses with a favorable safety profile observed in this heavily pre-treated EBV-positive lymphoma patient population are impressive, several of which are still ongoing. This publication further supports the recently announced expansion of the NAVAL-1 trial’s EBV-positive peripheral T-cell lymphoma cohort, representing an exciting time for our clinical trial program, and we look forward to providing more updates on its progress in the future."

Data published from the study showed that CRs were achieved across multiple EBV+ lymphoma subtypes, with a reported overall response rate (ORR)/CR rate of 40%/19% in 43 evaluable patients. In patients with EBV+ PTCL, which was recently established as the leading indication in Viracta’s pivotal NAVAL-1 trial, ORR/CR rates of 67%/50% were reported (n=6 including both EBV+ PTCL-not otherwise specified [PTCL-NOS] and angioimmunoblastic T-cell lymphoma [AITL] patients). Of note, one of the CRs was achieved in a patient whose disease never responded to second-line histone deacetylase inhibitor (HDACi) treatment. In 6 patients with EBV+ DLBCL, a rare aggressive and distinct B-cell lymphoma subtype characterized by an adverse clinical outcome, ORR/CR rates of 67%/33% were reported. Of note, one of the CRs was achieved in a patient whose disease never responded to first-line R-CHOP chemotherapy.

The published paper includes an additional 10-month follow-up period, which demonstrated durable response durations across multiple EBV+ lymphoma subtypes. As of the expanded data cutoff date of September 1, 2022, multiple patients remained in an ongoing durable response in excess of 30 months, with two patients in an ongoing response of approximately 36 months. The median time to response was 1.8 months, and the median duration of response was approximately 10 months in a heavily pre-treated patient population. Overall, trial participants received a median of two prior therapies before entering the trial, with 75% (41/55) being refractory to their last therapy.

Data also showed that all-oral Nana-val was well tolerated with reversible low-grade AEs. The most commonly observed treatment-emergent AEs were reversible cytopenias, low-grade creatinine elevations, and gastrointestinal symptoms. Initial data from the Nana-val Phase 1b/2 clinical trial were previously presented at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

About Nana-val (Nanatinostat and Valganciclovir)
Nanatinostat is an orally available HDACi being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which are key to inducing viral genes that are epigenetically silenced in Epstein-Barr virus (EBV)-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing clinical trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed or refractory EBV+ lymphoma (NAVAL-1) as well as in combination with pembrolizumab in a multinational Phase 1b/2 trial in patients with recurrent or metastatic EBV+ nasopharyngeal carcinoma and other EBV+ solid tumors.

About EBV-Associated Cancers
Approximately 90% of the world’s adult population is infected with EBV. Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of cells for the duration of the patient’s life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV+ lymphomas. EBV is estimated to be associated with approximately 2% of the global cancer burden, including lymphoma, nasopharyngeal carcinoma, and gastric cancer.

On August 8, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the second quarter ended June 30, 2023 (Press release, Veracyte, AUG 8, 2023, View Source [SID1234633995]).

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"We delivered outstanding second quarter results, with revenue and test volume that exceeded our expectations. We also generated a record $17 million in cash from operations this quarter, driven by our exceptional top-line performance and strong cash collections," said Marc Stapley, Veracyte’s chief executive officer. "The compelling clinical data we shared this quarter demonstrates our focus on producing further evidence that our tests are helping to better guide patient care at pivotal moments in the race to diagnose and treat cancer."

Key Business Highlights

Increased second quarter total revenue by 24% to $90.3 million, compared to the second quarter of 2022.
Grew total test volume to 31,809, an increase of 28% compared to the second quarter of 2022.
Presented 12 abstracts for our diagnostic tests and biopharmaceutical offerings at major medical conferences. Study findings demonstrate our tests’ positive real-world impact on patient care, and also advance the scientific understanding of a number of diseases that we and our biopharmaceutical customers address.
Published real-world Decipher Prostate Genomic Classifier findings in JNCI Cancer Spectrum from a population-based study of the National Cancer Institute’s SEER program database. The findings reinforce the ability of our Decipher Prostate Genomic Classifier to help guide personalized treatment approaches for men with prostate cancer.
Generated approximately $17 million of cash from operating activities, ending the second quarter with cash, cash equivalents and short-term investments of $191 million, compared to $178 million at the end of the first quarter.
Second Quarter 2023 Financial Results

Total revenue for the second quarter of 2023 was $90.3 million, an increase of 24% compared to $72.9 million reported in the second quarter of 2022. Testing revenue was $81.7 million, an increase of 37% compared to $59.7 million in the second quarter of 2022 driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $4.0 million, an increase of 29% compared to $3.1 million in the second quarter of 2022. Biopharmaceutical and other revenue was $4.6 million, a decrease of 55% compared to $10.0 million in the second quarter of 2022.

Total gross margin for the second quarter of 2023, including the amortization of acquired intangible assets, was 62%, compared to 59% in the second quarter of 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 67%, compared to 66% in the second quarter of 2022.

Operating expenses, excluding cost of revenue, were $63.9 million, an increase of 19% compared to the second quarter of 2022. Non-GAAP operating expenses, excluding cost of revenue, amortization of acquired intangible assets, other acquisition related expenses and other restructuring costs, were $59.3 million compared to $49.0 million in the second quarter of 2022.

Net loss for the second quarter of 2023 was $8.4 million, an improvement of 12% compared to the second quarter of 2022. Basic and diluted net loss per common share was $0.12, an improvement of 8% compared to the second quarter of 2022. Net cash provided by operating activities in the first six months of 2023 was $14.5 million, an improvement of $23.7 million compared to the same period in 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2023 Financial Outlook

The company is raising full-year 2023 total revenue guidance to $342 million to $350 million, representing year-over-year growth of 15% to 18%, and an improvement compared to prior guidance of $330 million to $340 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BI62f03c15042b4d5dadeed4e37ed31fd8

On August 8, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported that The Journal of Urology published data from the Phase 3 ATLAS trial for investigational agent UGN-102 (mitomycin) for intravesical solution in patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC), that demonstrated superiority to transurethral resection of bladder tumor surgery (TURBT) with a 55% reduction of risk for recurrence, progression, or death in patients who received UGN-102 (Press release, UroGen Pharma, AUG 8, 2023, View Source [SID1234633994]). Tumor-free complete response rate at three months was 65% for patients who only received UGN-102, compared to a 64% complete response rate at three months for patients who underwent TURBT. TURBT is the current standard of care for patients with this type of cancer.

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"The publication of our ATLAS data in The Journal of Urology adds another chapter to our understanding of the potential of UGN-102 as a treatment for patients with LG-IR-NMIBC, who often face multiple surgeries due to the recurring nature of this cancer," said Mark Schoenberg, M.D., Chief Medical Officer at UroGen. "Our hope is that one day urologists may have an effective, non-surgical therapy option beyond TURBT that can be used to address this large patient population."

The Phase 3 ATLAS clinical trial, which met its primary endpoint, evaluated the efficacy, durability, and safety of UGN-102 with or without TURBT vs. TURBT alone in 282 patients with LG-IR-NMIBC. UroGen recently announced positive topline data from ATLAS and the Phase 3 ENVISION trial. The ENVISION trial evaluated the efficacy and safety of UGN-102 as a primary chemoablative therapy in 240 patients with LG-IR-NMIBC and met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.2% rate of complete response at 3-months following the initial treatment.

The Journal of Urology publication includes the following findings from ATLAS:

Tumor-free complete response 3 months after initial treatment was achieved by 92 patients (65%) who received UGN-102 and 89 patients (64%) treated by TURBT.
UGN-102 was generally well tolerated, with a side effect profile similar to that of previous clinical trials.
The estimated probability of remaining event free 15 months after randomization was 72% for UGN-102 ± TURBT and 50% for TURBT monotherapy [hazard ratio 0.45].

"While TURBT is the standard treatment for bladder cancer, the recurrent nature of LG-IR-NMIBC means that patients will undergo multiple surgeries that come with risks for this older patient population," says Sandip Prasad, M.D., M.Phil., Director of Genitourinary Surgical Oncology, Morristown Medical Center/Atlantic Health System, NJ, and Chief Investigator in the ATLAS trial. "It is exciting to consider what a potential non-surgical therapeutic alternative could mean for both patients and doctors who are eager for additional options."

Additional data evaluating the secondary endpoint of the ENVISION trial, Duration of Response, is expected in 2024. Assuming positive findings, a New Drug Application (NDA) is anticipated to be submitted to the U.S. Food and Drug Administration (FDA) in the same year.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. Assuming positive findings from the durability of response endpoint from the ENVISION Phase 3 study, UroGen anticipates submitting a New Drug Application (NDA) for UGN-102 in 2024. If approved, UGN-102 would be the first non-surgical primary therapeutic to treat a subset of bladder cancer characterized by high recurrence rates and multiple surgeries.

About ATLAS

ATLAS is a global, open-label, randomized controlled Phase 3 trial designed to assess the efficacy and safety of UGN-102, with or without TURBT, vs. TURBT alone in patients diagnosed with LG-IR-NMIBC. The trial enrolled 282 patients in clinical sites in the U.S., Europe and Israel. Patients were randomized 1:1 to either UGN-102 or TURBT. Patients in the UGN-102 arm were treated with six weekly intravesical instillations of UGN-102. At the 3-month time point, patients were assessed for response. Patients who demonstrated a complete response to either UGN-102 or TURBT, were assessed for long-term follow-up for evidence of recurrence. Patients who demonstrated presence of persistent disease at 3-months, in either arm, underwent a TURBT and continued for long-term follow-up for evidence of recurrence. The primary endpoint of the study is disease-free survival. Learn more about the ATLAS trial at www.clinicaltrials.gov (NCT04688931).