On August 10, 2023 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the second quarter of 2023 (Press release, Y-mAbs Therapeutics, AUG 10, 2023, View Source [SID1234634226]).

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"The second quarter of 2023 marked another period of progress for DANYELZA with continued revenue growth and international expansion with regulatory approval in Brazil," said Thomas Gad, Founder, President, and Interim Chief Executive Officer. "As we continue patient enrollment in the Phase I GD2-SADA study, we are pleased to report that we have closed Cohort 1 and Cohort 2 and are now dosing Cohort 3, and we have administered a 200 mCi dose of 177Lu-DOTA and an interval between dosing of the protein and the payload of between two to five days. We look forward to presenting pk and imaging data from this study later this year at our R&D Day. Upon completion of our reorganization, we estimate our existing cash and cash equivalents to support our business operations as currently planned into 2026, positioning us well to deliver our therapies to more patients both in the U.S. and worldwide."

Second Quarter 2023 and Recent Corporate Developments

· On July 2, 2023, the Company’s partner in China, SciClone Pharmaceuticals, announced that they had officially launched DANYELZA.
· On May 26, 2023, Y-mAbs announced interim clinical data from study 201 with naxitamab in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF") in patients with relapsed or refractory high-risk neuroblastoma and presented such interim clinical data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting on June 2-6, 2023.
· Also at ASCO (Free ASCO Whitepaper), Y-mAbs presented the design of its Phase I clinical study evaluating the Company’s Self-Assembly DisAssembly Pre-targeted Radioimmunotherapy ("SADA Y-PRIT") Theranostic Platform for the treatment of certain GD2-positive solid tumors, including small cell lung cancer, sarcoma, and malignant melanoma.
· On May 23, 2023, Y-mAbs announced that the Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária ("ANVISA") granted marketing authorization for DANYELZA (naxitamab-gqgk).
· On April 18, 2023, Y-mAbs announced that positive preclinical data had been presented on naxitamab in triple-negative breast cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting.
· On April 5, 2023, Y-mAbs announced that the first patient had been dosed in the Phase I clinical study evaluating the Company’s SADA Y-PRIT Theranostic Platform for the treatment of certain GD2-positive solid tumors.

Financial Results

Revenues

Y-mAbs reported DANYELZA net product revenues of $20.8 million and $41.0 million for the quarter and six months ended June 30, 2023, which represented increases of 112% and 102%, respectively, over $9.8 million and $20.3 million in the comparable periods of 2022.

The DANYELZA net product revenues for the quarter ended June 30, 2023 represents an increase of 3% compared to the first quarter of 2023. The $0.5 million sequential increase was driven by international revenues and related royalties, which included $3.5 million of revenues and related royalties for the China commercial launch inventory stocking order from SciClone, which Y-mAbs does not anticipate recurring at this level each quarter. This increase was partly offset by a softening in new U.S. patients in the second quarter and the Company’s $2.5 million inventory stocking order from WEP in the first quarter.

As of June 30, 2023, Y-mAbs has delivered DANYELZA to 56 centers across the U.S., a sequential increase of 6% in the number of centers since the first quarter of 2023. During the second quarter of 2023, approximately 61% of the vials sold in the U.S. were sold outside of Memorial Sloan Kettering Cancer Center ("MSKCC"), which is in line with the first quarter of 2023.

Operating Expenses

Cost of Goods Sold

Cost of goods sold was $4.6 million and $1.1 million for the three months ended June 30, 2023 and 2022, respectively. The increased cost of goods sold was driven by increased product revenue in the three months ended June 30, 2023 and an inventory write-down of $0.5 million in the three months ended June 30, 2023. Cost of goods sold was $6.7 million and $3.0 million for the six months ended June 30, 2023 and 2022, respectively. The increase in cost of goods sold was primarily driven by increased product revenue.

The Company’s gross margin decreased in the three and six months ended June 30, 2023, compared to the same periods in 2022, as a result of increased revenues from geographic areas outside of the United States, which were at a lower gross margin. The Company’s cost of goods sold includes amounts related to materials, third-party contract manufacturing, third-party packaging services, freight, labor costs for personnel involved in the manufacturing process, third-party royalties for approved products, and indirect overhead costs.

Research and Development

Research and development expenses were $12.1 million for the three months ended June 30, 2023, a reduction of 54% compared to $26.4 million for the three months ended June 30, 2022. The $14.3 million decrease was primarily due to decreased spending on deprioritized programs in connection with the Company’s previously announced restructuring plan, resulting in a $6.6 million decrease related to outsourced manufacturing, a $3.2 million decrease in personnel-related costs, and a $2.5 million decrease in outsourced research and supplies.

For the six months ended June 30, 2023, research and development expenses were $25.5 million, a reduction of 48% compared to $49.3 million for the six months ended June 30, 2022. The $23.8 million decrease was primarily due to decreased spending on deprioritized programs as described above, resulting in a $12.3 million decrease related to outsourced manufacturing, a $4.8 million decrease in outsourced research and supplies, a $2.9 million decrease in clinical trials and a $2.1 million decrease in personnel-related costs.

Y-mAbs recorded a restructuring charge of $3.4 million in research and development expenses during the six months ended June 30, 2023, in connection with the restructuring plan.

Selling, General, and Administration

Selling, general, and administrative expenses were $11.3 million for the three months ended June 30, 2023, a reduction of 51.1% compared to $23.1 million for the three months ended June 30, 2022. The $11.8 million decrease in selling, general and administrative expenses was primarily attributable to a $10.9 million charge related to contractual severance related benefits for the Company’s former Chief Executive Officer in connection with his departure in the second quarter of 2022.

For the six months ended June 30, 2023, selling, general, and administrative expenses were $23.5 million, a reduction of 35.6% compared to $36.5 million for the six months ended June 30, 2022. The $13.0 million decrease in SG&A expenses was primarily attributable to the contractual severance-related benefits described above.

Y-mAbs recorded a restructuring charge of $1.1 million in selling, general, and administrative expenses during the six months ended June 30, 2023, in connection with the restructuring plan.

Net Loss

Y-mAbs reported a net loss for the second quarter ended June 30, 2023, of $6.3 million, or ($0.14) per basic and diluted share, compared to a net loss of $41.1 million, or ($0.94) per basic and diluted share, for the quarter ended June 30, 2022. For the six months ended June 30, 2023, the Company reported a net loss of $12.7 million, or $0.29 per basic and diluted share, compared to a net loss of $69.2 million, or $1.58 per basic and diluted share, for the six months ended June 30, 2022. The favorable decrease in net loss was primarily driven by an increase in DANYELZA U.S. product revenues in the second quarter and six months ended June 30, 2023, an incremental benefit from expanding into international markets, decreased research and development cost, and decreased selling, general and administration cost, partially offset by the unfavorable impact of restructuring charges, all as noted above.

Cash and Cash Equivalents

As of June 30, 2023, Y-mAbs had approximately $87.9 million in cash and cash equivalents which, together with anticipated DANYELZA product revenues, is expected to support operations as currently planned into 2026. This estimate reflects the Company’s current business plan that is supported by assumptions that may prove to be inaccurate, such that Y-mAbs could use its available capital resources sooner than it currently expects.

Financial Guidance

Management reiterates its full year 2023 financial guidance, as updated on May 8, 2023:

· Anticipated DANYELZA net product revenues expected to be between $80 million and $85 million;
· Anticipated operating expenses expected to be between $115 million and $120 million;
· Anticipated total annual cash burn expected to be between $40 million and $50 million; and
· Cash and cash equivalents anticipated to support operations as currently planned into 2026.

Webcast and Conference Call

Y-mAbs will host a conference call on Friday, August 11, 2023, at 9:00 a.m. EDT. To participate in the call, please use the following dial-in information.

On August 10, 2023 Veru Inc. (NASDAQ: VERU), a late clinical stage biopharmaceutical company focused on developing novel medicines for metastatic breast cancer and for viral induced acute respiratory distress syndrome (ARDS), reported financial results for its fiscal 2023 third quarter and provided a business update (Press release, Veru, AUG 10, 2023, View Source [SID1234634225]).

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"This quarter was marked by important Company efforts to seek additional regulatory clarity from FDA regarding our two lead development programs, enobosarm and sabizabulin and to secure funding sources," said Mitchell Steiner, M.D., Chairman, President, and Chief Executive Officer of Veru. "We recently received further FDA regulatory clarity on the first stage of the Phase 3 ENABLAR-2 clinical trial design in order to add enobosarm alone as well as enobosarm in combination with abemaciclib as a 2nd line treatment for metastatic breast cancer with objective tumor response rates (ORR) as the primary endpoint. If ORR improvement is significant, we plan to pursue the accelerated approval pathway. As for sabizabulin, although there is a new summer and expected fall/winter COVID-19 surge, we believe that sabizabulin, as a broad antiviral and anti-inflammatory agent, may address viral lung infections and ARDS caused by other common viruses as well. Accordingly, we plan to discuss with FDA expanding the sabizabulin Phase 3 confirmatory study to include all hospitalized adult patients who have any kind of viral induced lung infection (influenza and RSV in addition to COVID)

requiring supplemental oxygen and who are at risk for ARDS. FDA has granted this meeting for September 2023. Viral induced pneumonia and lung infection is a leading cause of hospitalization in the U.S. according to the American Thoracic Society. In the U.S. alone, it is common to see well over 2 million hospitalizations annually from viral induced lung infections."

Dr. Steiner added: "During the quarter, we continued to make great progress on reducing our cash burn as we focus on our prioritized Phase 3 clinical programs. The Company’s cash burn during the quarter was $7.3 million, a $16.1 million reduction compared to the prior quarter. We also increased available capital through the completed $20 million ENTADFI sale and the $100 million Lincoln Park Capital Fund common stock purchase agreement. I am also excited to report that our sexual health business is making great progress with the FC2 Female Condom telehealth platform prescription business in the U.S. which has turned around and is growing to provide additional cash that we can invest, as we have done historically, in our two Phase 3 clinical development programs for large market opportunities."

Oncology Program Update:

The Company’s oncology drug pipeline is focused on the clinical development of enobosarm, an oral selective androgen receptor agonist, for the 2nd line treatment of metastatic breast cancer.

Enobosarm is a new kind of endocrine therapy for advanced breast cancer. Enobosarm is an oral, new chemical entity, selective androgen receptor agonist that activates the androgen receptor (AR) in AR+ ER+ HER2- metastatic breast cancer, which suppresses tumor growth without the unwanted masculinizing side effects and increases in hematocrit seen with androgens.

Phase 3 clinical ENABLAR-2 study – Enobosarm +/- abemaciclib (CDK 4/6 inhibitor) combination versus estrogen blocking agent (active control) as a 2nd line treatment for AR+ ER+ HER2- metastatic breast cancer.

On March 30, 2023, the Company met with the FDA to gain further agreement on Phase 3 clinical trial design and program. The Phase 3 study has been amended to accommodate the FDA’s latest recommendations to support registration as a second line treatment for patients with AR+ ER+ HER2- metastatic breast cancer who have tumor progression while receiving a CDK 4/6 inhibitor plus an estrogen blocking agent (nonsteroidal aromatase inhibitor or selective estrogen receptor degrader). The Phase 3 ENABLAR-2 study has 2 distinct study stages:

In Stage 1 of the Phase 3 study which will enroll 160 patients, the objectives are to optimize the dose of enobosarm in the abemaciclib combination and to assess the efficacy of enobosarm as a monotherapy compared to an estrogen blocking agent active control. The primary endpoint for Stage 1 is ORR. The Stage 1 initial run-in enrolled 3 patients to assess the safety and pharmacokinetics of the abemaciclib + enobosarm 9mg combination. In this run-in portion, there were no drug-to-drug interactions between abemaciclib and enobosarm, and there were no new safety findings. Further, the early preliminary clinical results showed 2 partial responses and 1 stable disease in the first 3 patients based on local assessments, and all patients have been on study for over 9 months. Our current plan is to have Phase 3 Stage 1 clinical results by late 2024 or early 2025. If enobosarm monotherapy or abemaciclib + enobosarm combination therapy compared to estrogen blocking agent (active control) demonstrates significant improvement in ORR, which is considered a surrogate endpoint for clinical benefit, then the Company plans to meet with the FDA to consider an accelerated approval regulatory pathway based on the clinical data from the Stage 1 portion of the Phase 3 study.

In Stage 2 of the Phase 3 study, we plan to enroll approximately 200 subjects in a multicenter, open label, randomized (1:1), active control clinical study, to evaluate the efficacy and safety of enobosarm with or without abemaciclib therapy (depending on the outcome of Stage 1) versus an alternative estrogen blocking agent (selective estrogen receptor degrader or an aromatase inhibitor) in subjects with AR+ ER+ HER2- metastatic breast cancer who have failed a CDK 4/6 inhibitor plus an estrogen blocking agent (nonsteroidal aromatase inhibitor or selective estrogen receptor degrader). The primary endpoint for Stage 2 of the Phase 3 study is progression-free survival.

In January 2022, Veru entered into a clinical trial collaboration and supply agreement through which Eli Lilly supplies abemaciclib for the ENABLAR-2 trial.

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

We have agreement with FDA on the design of the Phase 3 confirmatory COVID-19 study, but given the current COVID-19 landscape and unmet need for other types of viral induced ARDS in general, the Company now plans to meet with the FDA to reach agreement on the design of a proposed expanded Phase 3 confirmatory study evaluating sabizabulin 9mg for the treatment of hospitalized adult patients who have any viral lung infection, including SARS-CoV-2, Influenza A and B, Respiratory Syncytial Virus (RSV) and other viruses, and who require oxygen support and are at high risk for ARDS. The FDA has granted this meeting with Veru for September 2023.

The Company is developing sabizabulin 9mg, a novel oral microtubule disruptor, which has both host targeted antiviral and broad anti-inflammatory properties, as a two-pronged approach to the treatment of hospitalized patients with viral lung infection at high risk for ARDS and death. The Company has completed positive Phase 2 and positive Phase 3 COVID-19 clinical studies that have demonstrated that sabizabulin treatment resulted in a mortality benefit in hospitalized moderate to severe patients with COVID-19 viral lung infection at high risk for ARDS and death. On April 27, 2023, the Company met with the FDA and reached agreement on the design of the Phase 3 confirmatory COVID-19 clinical trial to evaluate sabizabulin treatment of hospitalized moderate to severe COVID-19 patients who are at high risk for ARDS and the path forward to submit a new EUA application and/or NDA.

On April 4, 2023, Veru announced positive results from a sabizabulin animal study conducted by a team of researchers at Labcorp Early Development Laboratories, Ltd, United Kingdom. The purpose of the study was to evaluate the efficacy of sabizabulin in the influenza H1N1 pulmonary inflammation mouse ARDS model. In the final study report, sabizabulin significantly reduced key cytokines involved in ARDS in H1N1 influenza pulmonary inflammation murine ARDS model. This positive preclinical influenza animal study further supports the potential use of sabizabulin as a broad antiviral and anti-inflammatory agent for viral induced ARDS.

As viruses that cause viral lung infection and ARDS do so in a similar way, the Company believes sabizabulin has the potential to be a treatment for all types of viral lung infections in hospitalized adult patients on oxygen who are at high risk for ARDS and death – not only SARS-CoV-2, but also influenza A or B, RSV, and other viruses. Although we have reached agreement with the FDA for the design of Phase 3 confirmatory COVID-19 clinical trial, the Company now plans to meet with the FDA again to reach agreement on the design of a proposed expanded Phase 3 confirmatory study evaluating sabizabulin 9mg for the treatment of hospitalized adult patients who have any kind of viral lung infection and on oxygen support who are at high risk for ARDS. The FDA has granted a meeting with Veru for September 2023. If we reach agreement with the FDA on the proposed ARDS sabizabulin study, we would not pursue the Phase 3 confirmatory COVID-19 only study or the influenza A or B only study.

Sabizabulin, as a host directed therapeutic, has been selected as a finalist drug candidate for consideration by The Influenza & Emerging Infectious Diseases Division of BARDA (The Biomedical Advanced Research and Development Authority of the U.S. Department of Health and Human Services) for a planned large multicenter, placebo-controlled clinical trial in hospitalized adult patients with ARDS. A decision by BARDA for final selection is expected during calendar Q4 2023. This clinical trial sponsored by BARDA plans to evaluate the safety and efficacy of up to 3 novel threat-agnostic and host-directed therapeutics representing different mechanisms of action that could address ARDS caused by known and unknown health security threats such as pandemic influenza, COVID-19, other emerging infectious diseases, and chemical, biological, radiological, and nuclear incidents.

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

On April 11, 2023, Veru announced positive results from a preclinical in vitro study conducted by a team of researchers led by Brian M. Ward, Ph.D., Associate Professor of Microbiology and Immunology, University of Rochester School of Medicine and Dentistry, Rochester, New York. The preclinical study evaluated the effects of sabizabulin against the prototypical poxvirus, vaccinia virus, which demonstrated that sabizabulin prevented both the release of poxvirus from infected cells and the spread of poxvirus to healthy cells. The Company plans to have preIND meetings with the FDA to discuss Animal Rule regulatory requirements for assessing the efficacy of sabizabulin for smallpox virus as well as Ebola virus. The smallpox virus pre-IND meeting has been granted and will take place in August 2023. Clinical human efficacy trials of drugs for preventing or treating viral infections, such as smallpox are not feasible and challenge studies in healthy subjects are unethical. Therefore, drugs for these indications are generally developed and approved under a regulatory pathway commonly referred to as the Animal Rule (21 CFR part 314, subpart I, for drugs and 21 CFR part 601, subpart H, for biologics). The FDA may grant marketing approval based on adequate and well-controlled animal efficacy studies when the results of those studies establish that the drug is reasonably likely to produce clinical benefit in humans.

Urev – Sexual Health Program Updates

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.

FC2 Female Condom (internal condom)

The Company sells FC2 in both the U.S. commercial sector and in the public health sector both in the U.S. and globally. FC2 is the only FDA approved female (internal) condom in the US. FC2 is a well-established business that has sold over 750 million female condoms worldwide. Since 2017, FC2 has generated over $213 million of net revenue. We have and plan to continue to invest the profits from the FC2 business to help fund the clinical development of our drug candidates, enobosarm and sabizabulin.

Telehealth is an important commercial strategy in the U.S. for access to birth control products especially FC2 as a nonhormonal and latex free option to prevent pregnancy and transmission of sexually transmitted infections. In order to maximize its reach and to have more direct control of the promotion, distribution, and sales of FC2, the Company made the decision last year to launch its own independent, FC2-dedicated telehealth digital portal. The Company continues to invest in and grow its direct to patient telehealth portal as well as adding new telehealth and internet fulfillment pharmacy partners to provide coverage in all 50 states in the U.S.

Having taken the time to refine our marketing, drive operational improvements, and enhance the patient experience during the initial launch phase over the last nine months, there are increasing new prescriptions being written and filled through our FC2 telehealth portal. During the third quarter of fiscal 2023, we saw our acquisition costs remain stable with new prescriptions growing over 115%, providing prescriptions to approximately 4,400 patients in total. We believe these results support our strategy and demonstrate high demand for FC2. We plan to continue to grow and deepen our investment in a profitable way by further expanding our presence both in social media channels and online search.

In the U.S. public sector, the company has seen an 115% increase in volume for the third quarter fiscal 2023 versus the third quarter fiscal 2022. This growth is attributable to key US public sector partnerships including the Company’s recent announcement in April 2023 that it has entered into a Purchasing Agreement with Afaxys Group Services, LLC (AGS), the #1 provider of oral and emergency contraceptives in US clinics.

In the global public health sector outside the U.S., the Company markets FC2 to entities, including ministries of health, government health agencies, U.N. agencies, nonprofit organizations and commercial partners, that work to support and improve the lives, health and well-being of women around the world. We are currently supplying a large multi-year South African tender for female condoms, which is expected to continue until 2025 and have seen sales grow in the current year as the current tender launched. We also expect a formal Brazil tender process to commence later this year.

Corporate Updates

In June 2023, the Company announced that The University of Tennessee Health Science Center (UTHSC), oncology drug discovery research partner to Veru Inc., secured two additional grants to develop oncology therapeutics for indications of high unmet need. UTHSC was awarded $924,000 from the U.S. Department of Defense (DoD) and $3,074,470 from the National Cancer Institute’s Research Project Grant (NCI R01). These two new grants awarded to UTHSC bring the university’s aggregate oncology funding related to this project to over $10 million.

In May 2023, the Company entered into a common stock purchase agreement for the purchase of up to $100 million with Lincoln Park Capital Fund. In May 2023, the Company also entered into a common stock open market sale agreement (At-the-Market facility) with Jefferies LLC for the sale of up to $75 million.

Third Quarter Financial Summary: Fiscal 2023 vs Fiscal 2022

•
Net revenues decreased to $3.3 million from $9.6 million

•
Gross profit decreased to $1.2 million from $7.1 million

•
Research and development expenses decreased to $2.9 million from $18.1 million

•
Selling, general and administrative expenses increased to $10.9 million from $10.8 million

•
Operating income, which included a gain on the sale of the Company’s ENTADFI assets of $17.5 million, was $4.9 million versus operating loss of $21.8 million

•
Net income was $6.3 million, or $0.07 per share, compared to net loss of $22.2 million, or $0.28 per share

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

•
Net revenues decreased to $12.4 million from $36.8 million

•
Gross profit decreased to $6.0 million from $30.1 million

•
Research and development expenses increased to $44.5 million from $43.8 million

•
Selling, general and administrative expenses increased to $41.3 million from $24.9 million

•
Operating loss, which included an impairment charge of $3.9 million, a provision for credit losses of $3.9 million, and a gain on the sale of the Company’s ENTADFI assets of $17.5 million, was $70.1 million versus $38.6 million

•
Net loss was $69.3 million, or $0.83 per share, compared to $42.8 million, or $0.53 per share

Balance Sheet Information

•
Cash and cash equivalents were $16.2 million as of June 30, 2023 versus $80.2 million as of September 30, 2022

•
Net accounts receivable were $5.1 million as of June 30, 2023 versus $3.6 million as of September 30, 2022

•
Notes receivable, gross of imputed interest, from the sale of the Company’s ENTADFI assets are $14.0 million as of June 30, 2023

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 August 10, 2023 at approximately 12:00 p.m. ET by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) passcode 1699199 for one week.

On August 10, 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 second quarter ended June 30, 2023 (Press release, Werewolf Therapeutics, AUG 10, 2023, View Source [SID1234634224]).

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"Throughout the second quarter, we have continued advancing our INDUKINE pipeline with ongoing enrollment of our first-in-human clinical trials for WTX-124 and WTX-330, our two lead programs," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "We look towards the fourth quarter, when we anticipate sharing initial safety, tolerability, pharmacokinetics and biomarker data from our Phase 1/1b trial of WTX-124 in multiple solid tumors. Additionally, we are pleased that Jazz Pharmaceuticals has announced the Investigational New Drug (IND) application clearance of JZP898 (formerly WTX-613), an engineered IFNα INDUKINE molecule. Werewolf continues to operate from a position of financial strength with a cash runway projected to carry us through at least the fourth quarter of 2024."

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 KEYTRUDA (pembrolizumab) in multiple solid tumors.

•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 prior checkpoint inhibitor therapy.
•Enrollment is ongoing in monotherapy dose-escalation and combination therapy cohorts, and the Company anticipates reporting initial safety, tolerability, pharmacokinetics and biomarker data from monotherapy dose escalation in the fourth quarter of 2023.

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.

werewolflogohorizontalaa.jpg
JZP898 (Formerly WTX-613): an engineered IFNα INDUKINE pro-drug that is activated specifically within the tumor microenvironment where it can stimulate IFNα receptors on cancer-fighting immune effector cells.

•In April 2022, Werewolf entered into an exclusive global collaboration and license agreement with Jazz Pharmaceuticals under which Werewolf is responsible for certain pre-clinical development and other development activities with respect to JZP898.
•Jazz Pharmaceuticals recently disclosed that in July 2023, JZP898 received Investigational New Drug (IND) application clearance.

Financial Results for the Second Quarter of 2023:

•Cash position: As of June 30, 2023, cash and cash equivalents were $137.5 million, compared to $147.9 million as of March 31, 2023. The Company also had restricted cash and cash equivalents of $21.2 million as of June 30, 2023, and March 31, 2023, respectively. 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 $8.1 million for the second quarter of 2023, compared to $4.1 million 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 $9.6 million for the second quarter of 2023, compared to $13.9 million for the same period in 2022. The decrease in research and development expenses was primarily due to a decrease in the costs incurred with contract manufacturing associated with WTX-124 and WTX-330 and favorable adjustments recognized during the quarter upon the closeout of completed purchase orders.
•General and administrative expenses: General and administrative expenses were $4.6 million for the second quarter of 2023, compared to $5.2 million for the same period in 2022. The decrease in general and administrative expenses was primarily due to reduced insurance premiums and decreased utilization of outside advisors.
•Net loss: Net loss was $5.1 million for the second quarter of 2023, compared to $14.6 million for the same period in 2022.

On August 10, 2023 Vaccitech plc (NASDAQ: VACC), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel T cell immunotherapeutics designed to harness the power of the immune system to treat chronic infectious diseases, cancer and autoimmunity, reported its financial results for the second quarter of 2023 and provided an overview of the Company’s progress (Press release, Vaccitech, AUG 10, 2023, View Source [SID1234634223]).

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"In the second quarter of 2023, Vaccitech achieved important clinical milestones for two of our lead programs in HPV and HBV, which included the final analysis for the Phase 1b/2a trial of VTP-300 presented at EASL," said Bill Enright, Vaccitech’s Chief Executive Officer. "Looking ahead, we remain steadfast in our focus on HBV and are eagerly anticipating interim data from our ongoing Phase 2 trials which we expect to announce by the end of the year. We have been able to extend our cash runway into the second quarter of 2025, demonstrating Vaccitech’s ethos of agility and efficiency across our broad portfolio of programs."

Second Quarter 2023 and Recent Corporate Developments

Clinical developments

· In April 2023, at the 35th Annual International Papillomavirus Conference, the Company presented topline data from the HPV001 (NCT04607850) Phase 1b/2 clinical trial evaluating VTP-200 in women infected with human papillomavirus (HPV). Data included results for 42 women at Day 35, 7 days after the last dose of VTP-200. VTP-200 was generally well-tolerated and was administered with no product-related grade 3 unsolicited adverse events and no product-related severe adverse events. While the placebo group showed no antigen-specific T cell responses as measured by IFNg ELISpot, 26 of 29 women receiving varying doses of VTP-200 showed a response. The final dataset, including data on clearance of infection and cervical lesions at 12 months post-treatment, is expected in the second quarter of 2024.
· In June 2023, at the 2023 European Association for the Study of the Liver (EASL) International Liver Congress, the Company presented final Phase 2 HBV002 (NCT04778904) data showing that VTP-300, both as monotherapy and in combination with a single low dose of nivolumab (anti-PD-1 agent) at the time of the second dose, induced meaningful, durable reductions of hepatitis B surface antigen (HBsAg) and that reductions were most prominent in patients with lower baseline HBsAg. VTP-300 led to a decline in HBsAg in the majority of people infected with genotypes B and C viruses. In addition, VTP-300-induced T cells showed cross-reactivity to the core antigen from genotypes A to E in ELISpot assays using peripheral blood mononuclear cells from VTP-300-treated healthy subjects and genotype-specific peptides A-E.
· Also in June 2023, the Company announced dosing of the first patient in the PCA001 clinical trial evaluating its next generation product candidate, VTP-850, in prostate cancer (NCT05617040). PCA001 is a multi-center, Phase 1/2 trial designed to determine the recommended Phase 2 regimen and evaluate the safety, efficacy, as measured by prostate-specific antigen response (PSA), and T cell response of VTP-850 monotherapy in men with prostate cancer with rising PSA after definitive local therapy for their disease (i.e. biochemical recurrence).

Key operational updates

· In June 2023, the Company completed relocation of its U.S. team to a new, state-of-the-art laboratory and office facility in Germantown, Maryland.
· In July 2023, the Company announced the retirement of Dr. Margaret Marshall, Chief Medical Officer, effective immediately. In connection with her retirement, Dr. Marshall and the Company will enter into a consulting agreement.

Upcoming Milestones

· In the second half of 2023, the Company expects to:

o Announce interim efficacy data from HBV003 (NCT05343481), a Phase 2b clinical trial of VTP-300, that further evaluates its potential as a component of a functional cure for chronic Hepatitis B.
o Announce interim efficacy data from the Phase 2a clinical trial (ACTRN12622000317796) collaboration with Arbutus of VTP-300 in combination with Arbutus’ siRNA therapeutic candidate, AB-729 for chronic hepatitis B.
o Submit an Investigational New Drug (IND) application for VTP-1000, the Company’s lead SNAPvax technology candidate, for the treatment of celiac disease.

Q2 2023 Financial Highlights

· Cash position: As of June 30, 2023, the Company had cash and cash equivalents of $173.0 million, compared to $191.3 million as of March 31, 2023. The net cash used in operating activities was $17.0 million, primarily resulting from our net loss of $23.8 million adjusted by share based compensation of $2.0 million, depreciation and amortization of $1.3 million, and changes in our operating assets and liabilities. $3.0 million was used for investing activities, primarily from capital expenditures related to leasehold improvements on our new facility in Germantown, Maryland, consisting of laboratories and office space. $0.1 million was provided by financing activities being the proceeds received from the issuance of ordinary shares represented by American Depositary Shares through the Company’s "at-the-market" sales agreement, net of issuance costs. Based on current research and development plans, we expect our cash runway to fund our operating expenses and capital expenditure requirements into the second quarter of 2025.

· Revenue: Revenue consisted of $0.3 million in the second quarter of 2023 compared to $0.5 million in the first quarter of 2023. Revenue was comprised of the Company’s share of royalties received by Oxford University Innovation (OUI) as a result of commercial sales of Vaxzevria by AstraZeneca.
· Research and development expenses: Research and development expenses were $13.5 million in the second quarter of 2023 compared to $9.8 million in the first quarter of 2023, showing increased spend due to phasing of clinical and pre-clinical trials. VTP-300 HBV research and development expenses increased as a result of the Phase 2 HBV002 final data and the two ongoing phase 2b clinical trials. The research and development expenses related to SNAPvax candidates, VTP-1000/VTP-1100 Celiac/ HPV Cancer, increased due to IND-enabling studies. The quarter-on-quarter R&D expense per program is outlined in the following table.

Three months
ended
June 30, 2023 Three months
ended
March 30, 2023 Change
$000 $000 $000
Direct research and development expenses by program:
VTP-200 HPV 1,837 1,338 499
VTP-300 HBV 3,757 2,118 1,639
VTP-600 NSCLC1 79 275 (196 )
VTP-850 Prostate cancer 242 215 27
VTP-1000/VTP-1100 Celiac/HPV Cancer 3,018 1,572 1,446
Other and earlier stage programs 701 280 421
Total direct research and development expenses 9,634 5,798 3,836
Internal research and development expenses:
Personnel-related (including share-based compensation) 3,388 3,601 (213 )
Facility-related 202 371 (169 )
Other internal costs 319 44 275
Total internal research and development expenses 3,909 4,016 (107 )
Total research and development expense 13,543 9,814 3,729

1 The VTP-600 NSCLC Phase 1/2a trial is sponsored by Cancer Research UK.

· General and administrative expenses: General and administrative expenses were $13.1 million in the second quarter of 2023, compared to $12.1 million in the first quarter of 2023. The increase was mainly attributable to the unrealized foreign exchange loss of $4.2 million in the second quarter of 2023, compared to $3.5 million in the first quarter of 2023.
· Net loss: For the second quarter of 2023, the Company generated a net loss attributable to its shareholders of $23.8 million, or $0.62 per share on both basic and fully diluted bases, compared to a net loss attributable to shareholders of $18.2 million, or $0.48 per share on both basic and fully diluted bases in the first quarter of 2023.

On August 10, 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 second quarter ended June 30, 2023, and provided an overview of recent developments (Press release, UroGen Pharma, AUG 10, 2023, View Source [SID1234634222]).

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"We are in a transformative period for UroGen," said Liz Barrett, President and Chief Executive Officer of UroGen Pharma. "Recently, we announced positive topline results from the ATLAS and ENVISION Phase 3 clinical trials of UGN-102 in LG-IR-NMIBC. Both trials met their primary endpoints, demonstrating consistent, compelling results for UGN-102. If approved, we believe UGN-102’s promising efficacy and safety profile across multiple trials support a potential shift from frequent surgeries to a minimally invasive, non-surgical option for the more than 82,000 underserved LG-IR-NMIBC patients diagnosed annually in the U.S. alone, positioning it as a potential key growth driver for UroGen."

"Furthermore, increased adoption and expansion of JELMYTO use in a diverse LG-UTUC population, supported by a growing database of real-world outcomes data, translated to notable revenue growth during the second quarter of 2023," continued Ms. Barrett. "Building on these achievements, the recently announced $120 million private placement of ordinary shares and pre-funded warrants with a select group of biotech investors provides us with the resources to support our business, including our prospective pre-commercialization and launch strategy for UGN-102."

Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

The ENVISION Phase 3 clinical trial met its primary endpoint by demonstrating a 79.2% rate of complete response among ~240 LG-IR-NMIBC patients at 3-months after the first UGN-102 instillation.
Additional data evaluating the secondary endpoint of duration of response from ENVISION, and the submission of a New Drug Application (NDA) (assuming additional positive findings) to the U.S. Food and Drug Administration (FDA) are anticipated in 2024.
The Journal of Urology published a peer-reviewed article highlighting that the ATLAS Phase 3 clinical trial met its primary endpoint of disease-free survival, with topline results demonstrating a reduced risk of recurrence, progression, or death of 55% for UGN-102 + TURBT compared to TURBT monotherapy.

Patients who only received UGN-102 showed a 64.8% complete response rate at three months, compared to a 63.6% complete response rate at three months for patients who only received TURBT.

The estimated probability of remaining disease free 15-months after randomization was 72% for UGN-102 ± TURBT and 50% for TURBT monotherapy (hazard ratio 0.45).
UroGen hosted an event to discuss and highlight topline results from the Phase 3 ATLAS and ENVISION clinical trials on July 27th, 2023. A replay of the event is available on the UroGen website at View Source
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated record quarterly net product revenue of $21.1 million for the second quarter of 2023, representing ~27% growth over the second quarter of 2022.
Activated sites on August 1, 2023 were 1,058, compared to 1,009 on May 1, 2023, while repeat accounts on August 1, 2023 were 267, compared to 235 on May 1, 2023.
Results from a retrospective study investigating whether patients with higher-volume low-grade disease could achieve disease-free status using partial ablation or biopsy before JELMYTO treatment during initial ureteroscopy (URS) showed no significant difference in rendered disease-free rates between complete ablation (78.6%), partial ablation (57.6%), or biopsy-only (66.7%) groups during initial URS (p=0.15). The analysis also showed that tumor size prior to JELMYTO induction did not have a significant impact on rendered disease free (RDF) rates (p=0.09). The study aimed to find alternatives to nephroureterectomy for preserving kidney function and to assess JELMYTO’s efficacy in managing larger volume disease.
Private Placement of Ordinary Shares

Completed a private placement of ordinary shares and pre-funded warrants with gross proceeds of approximately $120 million before deducting placement agent commissions and other offering expenses to select institutional and accredited investors.
Second Quarter 2023 Financial Results:

JELMYTO Revenue: UroGen reported net product revenue of JELMYTO for the second quarter 2023 of $21.1 million, compared to $16.6 million in the second quarter of 2022.

R&D Expense: Research and development expenses for the second quarter 2023 were $11.6 million, including non-cash share-based compensation expense of $0.5 million as compared to $12.6 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 second quarter 2023 were $22.5 million, including non-cash share-based compensation expense of $1.7 million. This compares to $20.8 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 $5.3 million for the second quarter 2023, compared to $5.8 million for the same period in 2022. The rate applied to cash payments incurred in 2023 is 13% based on global net product sales of JELMYTO 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 $3.8 million for the second quarter of 2023, compared to $2.2 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 $24.1 million, or basic and diluted net loss per ordinary share of $1.03, for the second quarter 2023 as compared to $26.7 million, or basic and diluted net loss per ordinary share of $1.18, for the same period in 2022.

Cash & Cash Equivalents: As of June 30, 2023, cash, cash equivalents and marketable securities totaled $55.3 million. This figure does not include proceeds from the recent $120 million private placement of ordinary shares and pre-funded warrants.

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 also 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 also 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.

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 FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

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

About Upper Tract Urothelial Cancer (UTUC)

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

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of 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 secondary endpoint findings 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 the Phase 3 ENVISION Trial

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as primary chemoablative therapy in patients with low-grade, intermediate-risk NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the complete response rate at the 3-month assessment after the first instillation, and the key secondary endpoint will evaluate durability over time in patients who achieved a complete response at the three-month assessment. Based on discussions with the FDA, and assuming positive secondary endpoint findings, UroGen anticipates submitting an NDA for UGN-102 in 2024. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

About the Phase 3 ATLAS Trial

ATLAS was 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 + / – TURBT 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).