On May 11, 2023 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported its financial results for the first quarter ended March 31, 2023 and provided a corporate update (Press release, Vincerx Pharma, MAY 11, 2023, View Source [SID1234631543]).

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"The ADC space is going through an exciting period of innovation," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "The therapeutic potential of new conjugates is being widely recognized, as reflected in the recent industry announcements of collaborations, acquisitions, and positive clinical results. So, for Vincerx, this is an exciting time to be a bioconjugation company with a next-generation platform and multiple programs in, or soon to be in, the clinic."

Dr. Hamdy continued, "2023 has already been a productive year for us, with the initiation of our Phase 1 dose escalation study for VIP236, our first-in-class αvβ3 SMDC. Most recently, preclinical data presented at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting highlighted compelling monotherapy efficacy data for VIP236 in patient-derived xenograft models (PDX) across a number of aggressive indications. The data also showed significant tumor growth inhibition for VIP236 when compared with ENHERTU, an approved ADC, independent of HER2 status. We are encouraged to see our SMDC show improved in vivo efficacy across a range of HER2 expression levels, including HER2 low and HER2 negative gastric models, suggesting a potential new treatment option for patients across various aggressive solid tumors.

"On the ADC front, we expect to file the first IND from our ADC program, VIP943, for CD123+ hematologic malignancies in mid-2023. In preclinical data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in 2022, our next-generation kinesin spindle protein inhibitor (KSPi) payload, unique linker, and CellTrapper technology showed significant improvement in safety over Mylotarg, an approved ADC for the treatment of acute myeloid leukemia (AML). The data also showed improved efficacy and survival for VIP943 in combination with venetoclax and azacitidine. This triple combination resulted in significant tumor regression, as demonstrated by an increased number of complete responses and overall survival, compared with venetoclax and azacitidine. These data support the opportunity to move to earlier lines of therapy once the dose and safety profile for VIP943 have been established.

"We believe our next-generation ADC technology represents a significant step forward in the treatment of cancer by solving problems associated with current ADCs. We look forward to sharing advances for our programs as we continue to position Vincerx as a leader in the ADC space," concluded Dr. Hamdy.

First Quarter 2023 Corporate Highlights

Bioconjugation Platform
VIP236, an αvβ3 integrin binder linked to an optCPT payload SMDC:

At AACR (Free AACR Whitepaper) 2023, Vincerx presented compelling preclinical data demonstrating that VIP236 had potent and durable antitumor activity in multiple mouse models implanted with tumor cells from cancer patients:
VIP236 treatment is efficacious in a PDX non-small cell lung cancer model, including durable complete responses (CRs).
Significant tumor growth inhibition in a PDX colorectal carcinoma (CRC) liver metastasis model and significantly reduced lung and brain metastasis in a PDX orthotopic triple-negative breast cancer (TNBC) model.
Significant tumor growth inhibition, independent of HER2 status, in gastric PDX and cell line-derived cancer models when compared with ENHERTU.
Dosed the first cohort of the ongoing Phase 1 first-in-human dose-escalation study with VIP236 monotherapy in the first quarter 2023 (ClinicalTrials.gov NCT05712889).
VIP943, CD123-KSPi ADC:

At ASH (Free ASH Whitepaper) 2022, Vincerx presented preclinical data on VIP943 in AML models demonstrating:
Superiority with significantly improved safety in monkeys when compared with Mylotarg (gemtuzumab ozogamicin).
AML ex vivo and in vivo monotherapy activity.
No signs of cytokine release in human blood cells.
Significant tumor regression in combination with venetoclax and azacitidine in a PDX AML model.
IND-enabling studies continue to advance with an IND filing on track for mid-2023.
VIP924, CXCR5-KSPi ADC:

IND-enabling studies continue to advance, with an IND filing planned for mid-2024.
Additional Platform Updates

At AACR (Free AACR Whitepaper) 2023, Vincerx presented preclinical data on the synthesis and characterization of novel SMDCs with different payloads. Data showed high elastase-dependent potency and cytotoxicity across several cancer cell lines:
The large scope of potential payloads and conjugation chemistries support the strategy for selective delivery of payloads to the tumor microenvironment, without requiring the tumor target to internalize.
Evaluation of linker variations with in vivo studies across different payload classes is ongoing.
Enitociclib, Positive Transcription Elongation Factor b (P-TEFb)/CDK9 inhibitor

First patient dosed in National Institutes of Health collaborative Phase 1 combination study with venetoclax and prednisone (VVIP) in diffuse large B-cell lymphoma (ClinicalTrials.gov NTC05371054).
Anticipate dosing first patient in Phase 1b study of enitociclib in combination with an approved BTK inhibitor in patients with a high-risk chronic lymphocytic leukemia (CLL) in second quarter 2023.
First Quarter 2023 Financial Results

Vincerx had approximately $39.8 million in cash, cash equivalents and marketable securities as of March 31, 2023, as compared to approximately $52.5 million as of December 31, 2022. Based on its current business plans and assumptions, Vincerx believes its available capital will be sufficient to meet its operating requirements into late 2024.

Research and development (R&D) expenses for the first quarter ended March 31, 2023 were approximately $10.6 million, as compared to approximately $16.0 million for the same period in 2022. This decrease is primarily the result of declines in stock-based compensation expense of approximately $3.1 million, payroll related costs of approximately $1.4 million as a result of our headcount reduction in June 2022, manufacturing services associated with our ADC program of approximately $1.2 million, and clinical services of approximately $0.6 million, partially offset by an increase in third party research and preclinical work of approximately $1.3 million.
General and administrative (G&A) expenses for the first quarter ended March 31, 2023 were approximately $4.5 million, as compared to approximately $5.7 million for the same period in 2022. This decrease is primarily driven by a decrease in stock-based compensation expense of approximately $1.1 million.

For the first quarter ended March 31, 2023, Vincerx reported a net loss of approximately $14.3 million, or $0.68 per share. For the first quarter ended March 31, 2022, Vincerx reported a net loss of approximately $16.4 million, or $0.79 per share.

On May 11, 2023 Veru Inc. (NASDAQ: VERU), a late clinical stage biopharmaceutical company focused on developing novel medicines for the treatment of breast cancer and for SARS-CoV-2 and other viral ARDS-related diseases, reported financial results for its fiscal 2023 second quarter and provided a business update (Press release, Veru, MAY 11, 2023, View Source [SID1234631542]).

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"This past quarter, we prioritized our clinical development program to focus on those indications with the largest market opportunities and with the potential for meaningful Phase 3 clinical data results in 2024 for both enobosarm for 2nd line AR+ ER+ HER2- metastatic breast cancer and sabizabulin for SARS-CoV-2 viral ARDS," said Mitchell Steiner, M.D., Chairman, President, and Chief Executive Officer of Veru. "As part of this prioritization, we are planning to also expand enobosarm into bone-only, metastatic breast cancer and sabizabulin into influenza-induced ARDS."

Dr. Steiner added, "On the regulatory front, we received FDA clarity to continue advancing our ENABLAR-2 Phase 2b/3 clinical trial for metastatic breast cancer and agreement to pursue our confirmatory Phase 3 clinical trial for hospitalized COVID-19 adult patients at high risk for ARDS. We also reduced our cash burn rate, and in parallel, we increased available capital through the completed ENTADFI sale and the Lincoln Park Capital transaction. We are also seeking partnerships for both our clinical drug candidates. In addition, we continue to invest in Veru’s FC2 Female Condom telemedicine portal and establish partnerships to grow our FC2 prescription business in the U.S."

Breast Cancer Program Updates

Enobosarm, a Novel Oral Selective Androgen Receptor Agonist, and Abemaciclib, a CDK 4/6 Inhibitor, Combination Therapy for the 2nd Line Treatment of AR+ ER+ HER2- Metastatic Breast Cancer

In March 2023, the Company made the strategic decision to focus on the ENABLAR-2 Phase 2b/3 trial (combination of enobosarm + abemaciclib). The decision to focus on an earlier line of treatment is supported by the larger target patient population in a second line metastatic setting for AR+ ER+ HER2- breast cancer patients. On March 30, 2023, the Company met with the FDA to gain further regulatory clarity for the ongoing Phase 2b/3 clinical trial design and program. The Phase 2b/3 study has been amended to accommodate the FDA’s latest recommendations to support a potential registration. In the first stage of the trial, the dose of enobosarm in the abemaciclib combination is being optimized and the efficacy and safety of the combination therapy is being assessed compared to an estrogen blocking agent. The primary endpoint for stage 1 of the study is objective response rate (ORR), an endpoint that the FDA recognizes as an appropriate surrogate endpoint for clinical benefit for a possible accelerated approval. In Stage 2 of the Phase 2b/3 study, we plan to enroll approximately 210 subjects in a multicenter, open label, randomized (1:1), active control clinical study, to evaluate the efficacy and safety of enobosarm plus abemaciclib combination therapy versus an alternative estrogen blocking agent (SERD or an aromatase inhibitor) in subjects with AR+ ER+ HER2- metastatic breast cancer who have failed a CDK4/6 inhibitor plus an estrogen blocking agent (non-steroidal aromatase inhibitor or SERD). The primary endpoint is progression free survival. The regulatory strategy and clinical design for the Phase 2b/3 ENABLAR-2 clinical study could yield an accelerated approval from stage 1 and full approval from stage 2 for the 2nd line abemaciclib and enobosarm combination treatment of AR+ ER+ HER2- metastatic breast cancer. We anticipate having clinical data for the Phase 2b/3 ENABLAR-2 study in 2024. We have a collaboration and supply agreement with Eli Lilly and Company.

Enobosarm, a Novel Oral Selective Androgen Receptor Agonist, for the Treatment of Bone-only Nonmeasurable ER+ HER2- Metastatic Breast Cancer

The Company is planning a Phase 2b/3 study in bone-only nonmeasurable hormone receptor and HER2- metastatic breast cancer with enobosarm. Enobosarm has the ability to build both cortical and trabecular bone and muscle in clinical and/or nonclinical models, which may reduce the incidence of skeletal related events caused by bone metastases and positively impact quality of life for patients.

Infectious Disease: Viral Induced Acute Respiratory Distress Syndrome (ARDS) Program Updates

Sabizabulin, a Novel Oral Microtubule Disruptor, for the Treatment of Hospitalized Moderate to Severe COVID-19 Patients at High Risk for ARDS

In February 2023, the FDA declined to grant at this time an Emergency Use Authorization (EUA) for sabizabulin for hospitalized COVID-19 patients at high risk for ARDS because of the possibility of unknown influences or uncertainties in the study. Nonetheless, in communicating its decision, the FDA remains committed to working with the Company for the future development of sabizabulin.

In April 2023, the Company met with the FDA and reached an agreement on the trial design and path forward for a confirmatory Phase 3 study, which will include 408 subjects with a primary endpoint of all-cause mortality at Day 60. In addition, the study’s treatment population is expanded to include all hospitalized patients that require oxygen (WHO-4, WHO-5 and WHO-6) with no requirement to have a comorbidity. In order to get a potentially efficacious drug to patients in an efficient time frame, two planned interim efficacy analyses will be conducted: As requested by FDA, the first planned interim analysis will occur when 204 patients (50%) have completed the Day 60 primary efficacy endpoint, and the second planned interim analysis is expected to occur when 290 patients (71%) have completed the Day 60 primary efficacy endpoint. If either of the interim efficacy analyses meets the statistical significance criteria, the trial could be stopped for efficacy. Should the pre-specified primary efficacy endpoint analysis demonstrate a statistically significant effect on all-cause mortality favoring sabizabulin, the Company may consider a new request for an EUA and/or a submission of an NDA, "as the Company would potentially have two adequate and well controlled trials for review." The confirmatory Phase 3 clinical trial is expected to enroll in the second half of 2023, and the first planned interim analysis is expected to be conducted in 2024.

Sabizabulin, a Novel Oral Microtubule Disruptor, for the Treatment of Hospitalized Moderate to Severe Influenza Patients at High Risk for ARDS

In April 2023, the Company announced preclinical results of sabizabulin demonstrating robust anti-inflammatory activity with improved outcomes in an Influenza-Induced Pulmonary Inflammation Mouse ARDS model. As a result, Veru is planning a Phase 3 study of sabizabulin in hospitalized influenza patients at high risk for ARDS.

Sabizabulin, a Novel Oral Microtubule Disruptor, for the Treatment of Viruses that Pose Serious Worldwide Global Threats

In April 2023, the Company announced preclinical in vitro study results that demonstrate sabizabulin prevented both the release of Vaccinia poxvirus from infected cells and the spread of Vaccinia poxvirus to healthy cells. As a result, Veru is planning a pre-Investigational New Drug (IND) meeting with the FDA to discuss the development of sabizabulin for smallpox virus and Ebola virus under the Animal Rules FDA regulatory approval pathway.

Urev – Sexual Health Program Updates

FC2 Female Condom (internal condom)

In April 2023, the Company entered into a supply agreement with Afaxys Group Services, LLC (AGS), a healthcare company, to offer FC2 Female Condom through the AGS Group Purchasing Organization (GPO) for up to 31 million individuals that depend on public health centers for essential healthcare.

The Company continues to invest in and grow its direct to patient telemedicine portal and is focused on executing new contracts with additional telemedicine and internet fulfillment pharmacy partners to provide coverage in all 50 states in the U.S.

ENTADFI (finasteride and tadalafil) capsules for oral use, a New Treatment for Benign Prostatic Hyperplasia (BPH)

In April 2023, the Company sold ENTADFI, an FDA-approved oral, once daily product for BPH for men with an enlarged prostate experiencing the signs and symptoms of BPH for up to 26 weeks, to Blue Water Biotech for $20 million ($6 million upfront and the remaining $14 million in installments through Fiscal Year 2024), with the potential for up to an additional $80 million from sales milestones.

Corporate Updates

In May 2023, the Company entered into a common stock purchase agreement (Agreement) with Lincoln Park Capital Fund (LPC). Under the terms of the Agreement, LPC has committed to purchase up to $100 million of Veru’s common stock at Veru’s sole discretion from time to time over a 36-month period.

Second Quarter Financial Summary: Fiscal 2023 vs Fiscal 2022

•
Net revenues decreased to $6.6 million from $13.0 million

•
Gross profit decreased to $4.1 million from $11.2 million

•
Research and development expenses increased to $22.9 million from $15.5 million

•
Selling, general and administrative expenses increased to $12.8 million from $7.4 million

•
Operating loss, which included an impairment charge of $3.9 million and a provision for credit losses of $3.9 million, was $39.4 million versus $11.8 million

•
Net loss was $38.8 million, or $0.48 per share, compared to $14.2 million, or $0.18 per share

Year-to-Date Financial Summary: Fiscal 2023 vs Fiscal 2022

•
Net revenues decreased to $9.1 million from $27.2 million

•
Gross profit decreased to $4.8 million from $23.0 million

•
Research and development expenses increased to $41.6 million from $25.6 million

•
Selling, general and administrative expenses increased to $30.4 million from $14.1 million

•
Operating loss, which included an impairment charge of $3.9 million and a provision for credit losses of $3.9 million, was $75.0 million versus $16.7 million

•
Net loss was $75.6 million, or $0.94 per share, compared to $20.6 million, or $0.26 per share.

•
The net loss for the Company increased by $55 million for the current period. The main reason for the increase in the net loss relates to the Company preparing for the potential launch of sabizabulin for COVID-19 in the U.S. and outside the U.S. This required building a commercial team, engaging vendors to assist with the commercial launch, and manufacturing drug product for the launch upon EUA approval as required by the FDA. Since the declination, the majority of the employees hired for the commercial team have been terminated and the commercial launch sales and marketing related vendor contracts have been cancelled.

Balance Sheet Information

•
Cash and cash equivalents were $23.5 million as of March 31, 2023 versus $80.2 million as of September 30, 2022. Subsequent to the Company’s Fiscal Year 2023 second quarter, and as previously disclosed, Frost Gamma Investments Trust acquired $5 million of Company common stock in a private placement, and the Company sold its ENTADFI product to Blue Water Biotech for $6 million upfront, $14 million in notes receivable, and up to an additional $80 million if certain ENTADFI sales milestones are achieved.

•
Net accounts receivable were $4.2 million as of March 31, 2023 versus $3.6 million as of September 30, 2022

Event Details

The audio webcast will be accessible under "Investor Kit" in the Investors page of the Company’s website at www.verupharma.com. To join the conference call via telephone, please dial 1-800-341-1602 (domestic) or 1-412-902-6706 (international) and ask to join the Veru Inc. call. An archived version of the audio webcast will be available for replay on the Company’s website for approximately three months. A telephonic replay will be available on May 11, 2023 at approximately 12:00 p.m. ET by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) passcode 1592419 for one week.

On May 11, 2023 Werewolf Therapeutics, Inc. (the "Company" or "Werewolf") (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer, reported a business update and provided financial results for the first quarter ended March 31, 2023 (Press release, Werewolf Therapeutics, MAY 11, 2023, View Source [SID1234631541]).

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"In the first quarter, Werewolf has focused on execution by progressing our INDUKINE pipeline and enrolling ongoing first-in-human clinical trials for our lead programs, WTX-124 and WTX-330. In addition, preclinical data presented at AACR (Free AACR Whitepaper) and published in Cancer Immunology Research continues to demonstrate the robustness of our PREDATOR platform showing that Werewolf’s conditional activation technology results in potent anti-tumor activity alongside an improved therapeutic index," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "Looking ahead, in the fourth quarter we plan to share initial safety, tolerability, and preliminary efficacy data from our Phase 1/1b clinical trial of WTX-124 in solid tumor types."

"Finally, we’d like to express our deep appreciation to Reid Leonard, Ph.D., Werewolf’s Chief Operating Officer, who is retiring effective June 30, 2023, after a long and successful career in the biopharmaceutical industry. Reid is a founding member of the Werewolf Executive Team and has been instrumental in leading and advancing all aspects of organizational operations. We have benefited greatly from Reid’s significant expertise, and the strong team he has built and business process he has established will ensure continued operational excellence going forward. We wish Reid the very best in his retirement."

Recent Highlights and Upcoming Milestones

WTX-124: a systemically delivered, conditionally activated Interleukin-2 (IL-2) INDUKINE molecule being developed as monotherapy and in combination with checkpoint inhibitor therapy in multiple solid tumor types.

•Werewolf is progressing Study WTX-124×2101, its Phase 1/1b, multi-center, open-label clinical trial evaluating WTX-124 as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients with immunotherapy sensitive advanced or metastatic solid tumors who have failed standard of care, including checkpoint inhibitor therapy.
•Enrollment is ongoing in monotherapy dose-escalation cohorts, and the Company anticipates reporting interim safety, tolerability and preliminary efficacy data from these monotherapy cohorts in the fourth quarter of 2023.

•During the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023, the Company presented a poster entitled "Trial in progress: a multicenter phase 1/1b dose escalation study of WTX-124 as a monotherapy and in combination with pembrolizumab in patients with selected advanced or metastatic solid tumors."

WTX-330: a systemically delivered, conditionally activated Interleukin-12 (IL-12) INDUKINE molecule being developed in refractory and/or immunologically unresponsive tumors.

•Werewolf is progressing Study WTX-330×2101, its Phase 1, multi-center, open-label trial evaluating WTX-330 as a monotherapy in patients with immunotherapy insensitive or resistant advanced or metastatic solid tumors or non-Hodgkin lymphoma. Enrollment is ongoing in dose-escalation.
•During the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023, the Company presented a poster entitled "Trial in progress: a first-in-human, phase 1, multicenter dose escalation and dose expansion study of WTX-330 in adult patients with advanced or metastatic solid tumors or non-Hodgkin lymphoma."
•Also in April 2023, the Company announced the publication of preclinical data in Cancer Immunology Research Online First, demonstrating that mWTX-330 delivers IL-12 selectively to the tumor environment to inhibit tumor growth in mouse models. The preclinical data demonstrate that mWTX-330 stimulates a potent anti-tumor response in mice by activating tumor infiltrating lymphocytes and restoring the metabolic health of these lymphocytes and has the potential to minimize the toxicity previously associated with systemic IL-12 administration.

Early-Stage Pipeline:
•During the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023, The Company presented a poster entitled "Generation of IL-21 INDUKINE molecules for the treatment of cancer," which featured preclinical data supporting the recent nomination of Werewolf’s third wholly owned candidate, WTX-712, a conditionally activated IL-21 INDUKINE molecule in development for treatment of cancer. Highlights of the presentation include:
◦IL-21 INDUKINE polypeptides demonstrated improved tolerability and therapeutic window in mouse syngeneic tumor models.
◦Efficacy in these tumor models was linked to expansion and activation of tumor infiltrating T cells, increased polyfunctionality in CD8+ T cells, and signs of increased activation of B cells.

Financial Results for the First Quarter of 2023:

•Cash position: As of March 31, 2023, cash and cash equivalents were $147.9 million, compared to $129.3 million as of December 31, 2022. The Company also has restricted cash and cash equivalents of $21.2 million, compared to $1.2 million as of December 31, 2022. These increases are primarily due to proceeds from the Company’s at-the-market sales facility, the drawdown of remaining capital available under its term loan agreement with Pacific Western Bank, and funding received in connection with the Company’s licensing agreement with Jazz Pharmaceuticals ("Jazz"). The Company expects that its existing cash and cash equivalents, together with anticipated collaboration revenue, will be sufficient to fund its operational expenses and capital expenditure requirements through at least the fourth quarter of 2024.
•Collaboration revenue: Collaboration revenue was $4.5 million for the first quarter of 2023, compared to zero for the same period in 2022. Collaboration revenue is related to partial recognition of the $15.0 million upfront payment received in April 2022 upon the execution of Werewolf’s licensing agreement with Jazz and costs incurred for research services to be reimbursed by Jazz.
•Research and development expenses: Research and development expenses were $11.7 million for the first quarter of 2023, compared to $10.9 million for the same period in 2022. The increase in research and development expenses was primarily due to increased personnel costs.
•General and administrative expenses: General and administrative expenses were $5.0 million for the first quarter of 2023, compared to $4.4 million for the same period in 2022. The increase in general and administrative expenses was primarily due to increased costs to support the increasing size and complexity of Werewolf’s research, development and manufacturing activities.
•Net loss: Net loss was $12.0 million for the first quarter of 2023, compared to $15.3 million for the same period in 2022.

On May 11, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the first quarter ended March 31, 2023, and provided an overview of recent developments (Press release, UroGen Pharma, MAY 11, 2023, View Source [SID1234631540]).

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"2023 is off to a promising start as we reported double-digit year-over-year growth in worldwide JELMYTO net sales during the first quarter," said Liz Barrett, President, and Chief Executive Officer of UroGen. "JELMYTO adoption continues to grow, attributable to the growing body of evidence supporting its benefits in a real-world setting, combined with key initiatives that have expanded access and improved logistical efficiencies for physicians and patients. As we look ahead to summer 2023, we have several key events to prepare for, primarily topline results from both the ENVISION and ATLAS clinical trials investigating the potential of UGN-102 for the treatment of LG-IR-NMIBC. With prospective favorable results, we will look to submit a New Drug Application (NDA) for UGN-102 in 2024 to the U.S. Food & Drug Administration (FDA), for which we are already actively preparing."

Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

A topline data readout of the primary endpoint from the ENVISION Phase 3 pivotal study of UGN-102 evaluating the complete response (CR) rate of ~240 patients at 3-months after first instillation is expected this summer.
Topline data from the ATLAS clinical trial, the predecessor to ENVISION, is expected summer 2023 and will evaluate complete response, duration of response and safety from ~280 patients that completed the trial.
Announced preliminary results of a study to assess the feasibility of home instillation of UGN-102. In this study, UGN-102 was suitable to administer at home by a visiting nurse under the supervision of a treating physician and resulted in 75% (n=8) of patients achieving a complete response, defined as no detectable disease 3 months after starting treatment.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated net product revenue of $17.2 million for the first quarter of 2023, representing ~27% growth over the first quarter of 2022.
Activated sites on May 1, 2023 were 1,009, compared to 983 on March 1, 2023, while repeat accounts on May 1, 2023 were 235, compared to 214 on March 1, 2023.
A positive American Urologic Association (AUA) meeting was headlined by the first ever AUA and Society of Urologic Oncology (SUO) treatment guideline for LG-UTUC, recommending use of JELMYTO in certain clinical scenarios. The guideline states that tumor ablation should be the initial management option for patients with low-risk favorable UTUC, for which JELMYTO can be a treatment option as part of a kidney sparing approach intended to prevent radical nephroureterectomies (RNU) in low-risk UTUC patients.
New retrospective study presented at AUA 2023 reported on the use of JELMYTO in treating patients with UTUC after complete endoscopic ablation. Patients in this retrospective study who underwent complete endoscopic ablation followed by JELMYTO were more likely to be disease-free at first endoscopic evaluation than those who underwent chemoablation alone (69% vs. 40%). In the Phase 3 OLYMPUS study of JELMYTO, LG-UTUC patients in the primary chemoablation setting achieved a 58% complete response rate at first endoscopic evaluation.
New retrospective study presented at AUA 2023 reported similar outcomes when utilizing Jelmyto in treating LG-UTUC of the ureter compared to renal pelvic cancers. In this analysis, 47 patients had UTUC tumors involving the ureter, with 12 cases of ureteral tumor only (8.8%) and 35 cases of ureteral plus renal pelvic tumors (25.7%). The investigators reported no difference in outcomes at first endoscopic evaluation based on tumor location (p=0.644). JELMYTO is approved for the chemoablation of low-grade upper tract urothelial cancer (LG-UTUC) involving the renal pelvis and calyces.
First Quarter 2022 Financial Results:

Jelmyto Revenue: UroGen reported net product revenue of Jelmyto for the first quarter 2023 of $17.2 million, compared to $13.6 million in the first quarter of 2022.

R&D Expense: Research and development expenses for the first quarter 2023 were $12.5 million, including non-cash share-based compensation expense of $0.5 million as compared to $12.7 million, including non-cash share-based compensation expense of $0.7 million, for the same period in 2022.

SG&A Expense: Selling, general and administrative expenses for the first quarter 2023 were $24.5 million, including non-cash share-based compensation expense of $1.8 million. This compares to $21.3 million, including non-cash share-based compensation expense of $2.2 million, for the same period in 2022.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $5.2 million for the first quarter 2023. The rate applied to cash payments incurred in 2023 is 13% based on global net product sales of JELMTYO in 2022.

Interest Expense on Long-Term Debt: Interest expense related to the $100 million term loan facility with funds managed by Pharmakon Advisors was $3.6 million for the first quarter of 2023, compared to $0.3 million for the same period last year due to the transaction closing in March 2022 and the final $25 million draw down under the term loan facility in December 2022.

Net Loss: UroGen reported a net loss of $30.2 million, or basic and diluted net loss per ordinary share of $1.30, for the first quarter 2023 as compared to $28.4 million, or basic and diluted net loss per ordinary share of $1.25, for the same period in 2022.

Cash & Cash Equivalents: As of March 31, 2023, cash, cash equivalents and marketable securities totaled $75.2 million.

2023 Revenue, Operating Expense and RTW Expense Guidance: The Company reiterates anticipated full year 2023 net product revenues from JELMYTO to be in the range of $76 to $86 million. The Company reiterates anticipated full year 2023 operating expenses in the range of $135 to $145 million, including non-cash share-based compensation expense of $6.0 to $11.0 million, subject to market conditions. The Company reiterates anticipated full year 2023 non-cash financing expense related to the prepaid obligation to RTW Investments in the range of $21.0 to $26.0 million. Of this amount approximately $9.9 to $11.2 million is expected to be in cash.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On May 11, 2023 Theseus Pharmaceuticals, Inc. (NASDAQ: THRX) (Theseus or the Company), a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients through the discovery, development, and commercialization of transformative targeted therapies, reported business highlights and provided financial results for the first quarter ended March 31, 2023 (Press release, Theseus Pharmaceuticals, MAY 11, 2023, View Source [SID1234631539]).

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"We look forward to continued clinical execution and important pipeline advancements in the months ahead, most notably with two anticipated clinical readouts from our lead program, THE-630, in advanced GIST," said Tim Clackson, Ph.D., President and Chief Executive Officer of Theseus. "We maintain a strong financial position as we continue to progress our pipeline, with an anticipated IND submission for THE-349 in the fourth quarter of this year as well as advancement of our BCR-ABL and discovery programs."

Recent Pipeline Highlights and Upcoming Expected Milestones:

THE-630 is a pan-variant tyrosine kinase inhibitor (TKI) of the receptor tyrosine kinase KIT, designed for patients with gastrointestinal stromal tumors (GIST) that have developed resistance to earlier lines of therapy.
•As of March 31, 2023, Theseus is enrolling patients in cohort 7 of its ongoing phase 1/2 dose escalation and expansion clinical trial evaluating THE-630 in patients with advanced GIST.

•Theseus submitted its initial dose escalation results to the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place June 2-6, in Chicago, Illinois, and the abstract was selected for online publication. Theseus plans to present data with an updated cutoff date during a virtual investor event on May 25, 2023, at 5:30pm ET, after the public release of the abstract. The initial dose escalation data will include preliminary safety, pharmacokinetic (PK), and clinical activity data through cohort 6, as well as an analysis of circulating tumor DNA (ctDNA) through cohort 5.

•Theseus plans to present follow-up data at a scientific conference in the fourth quarter of 2023, which is expected to include data from additional dose escalation cohorts, including cohort 8, where Theseus expects to have reached the target systemic exposure for pan-variant KIT activity.

THE-349 is a fourth-generation (4G) epidermal growth factor receptor (EGFR) TKI development candidate, which has demonstrated activity against single-, double-, and triple-mutant EGFR variants, including T790M and C797X, found in EGFR-mutant non-small cell lung cancer (NSCLC) that has developed resistance to first- or later-line osimertinib.
•Preclinical data demonstrate THE-349 can potently inhibit all major classes of EGFR activating and resistance mutations observed in a post-first- or later-line osimertinib setting, possesses kinome and wild-type EGFR selectivity, and has central nervous system (CNS) activity.

•Theseus expects to submit an Investigational New Drug Application (IND) for THE-349 to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023 and to initiate the first-in-human trial as soon as possible thereafter, subject to clearance of the IND by the FDA.

BCR-ABL Program: Theseus aims to develop a potent and selective, next-generation, pan-variant BCR-ABL TKI candidate that optimizes the balance of safety and efficacy for patients with relapsed/refractory chronic

myelogenous leukemia (CML) and patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).

•Preclinically, Theseus lead molecules have been observed to show a high degree of potency against BCR-ABL and clinically relevant resistance mutations, such as the T315I gatekeeper mutation, and substantial kinome selectivity.
•Theseus expects to nominate a development candidate for this program by early 2024, with the goal of pursuing clinical development in patients with CML who have been previously treated with a second-generation TKI or have the T315I mutation, and in newly diagnosed patients with Ph+ ALL.

Business Highlights:

•In May 2023, Theseus announced the appointment of Franklin Vairinhos, Ph.D., as Vice President, Regulatory Affairs. Dr. Vairinhos has more than 30 years of experience in Regulatory Affairs, predominantly in developing regulatory strategies for oncology therapies.

First Quarter Financial Results:

Cash Position: As of March 31, 2023, Theseus had cash, cash equivalents, and marketable securities of $243.6 million, as compared to $211.8 million as of December 31, 2022. Theseus expects its current cash, cash equivalents, and marketable securities to fund operations and capital expenditures into the third quarter of 2025 based on its current operating plan.

R&D Expenses: Research and development expenses were $12.4 million for the first quarter of 2023, as compared to $6.5 million for the same period in 2022. This increase was primarily due to a $2.7 million increase in expense for preclinical studies and drug manufacturing to support THE-349 IND enabling studies, a $1.4 million increase in development expense related to discovery programs, as well as a $1.5 million increase in employee-related costs driven by an increase in headcount.

G&A Expenses: General and administrative expenses were $4.7 million for the first quarter of 2023, as compared to $4.0 million for the same period in 2022. This increase was primarily due to an increase in stock-based compensation expense and employee-related costs.

Net Loss: Net loss was $14.6 million for the first quarter of 2023, as compared to a net loss of $10.5 million for the same period in 2022.