On July 27, 2023 Verrica Pharmaceuticals presented its corporate presentation (Presentation, Verrica Pharmaceuticals, JUL 27, 2023, View Source [SID1234633465]).

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On July 27, 2023 UroGen Pharma Ltd. (Nasdaq: URGN) (UroGen), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it has entered into a definitive securities purchase agreement in connection with a private placement (the Private Placement) to selected institutional and accredited investors (Press release, UroGen Pharma, JUL 27, 2023, View Source [SID1234633464]). RA Capital Management L.P. and Great Point Partners LLC led the Private Placement, which also included Acorn Bioventures, Monograph Capital and Horton Capital Partners Fund, LP. UroGen expects to receive gross proceeds of approximately $120 million, before deducting placement agent commissions and other offering expenses.

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UroGen intends to use the net proceeds of the Private Placement for non-clinical and clinical development activities for its product candidates, commercialization expense and general corporate purposes.

Pursuant to the terms of the securities purchase agreement, UroGen will issue 12,579,156 ordinary shares, or pre-funded warrants in lieu thereof, at a purchase price of $9.54 per share (less $0.001 for each pre-funded warrant), which reflects a premium to the closing price on July 26, 2023. Each pre-funded warrant has an exercise price of $0.001 per share and does not expire until exercised in full. The Private Placement is expected to close on or about July 28, 2023 subject to the satisfaction of customary closing conditions.

BofA Securities and H.C. Wainwright & Co. are acting as joint placement agents for the Private Placement. Ladenburg Thalmann & Co. Inc. acted as a financial advisor to UroGen for the Private Placement.

The securities described above are being offered and sold in a private placement and have not been registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. UroGen has agreed to file a resale registration statement with the United States Securities and Exchange Commission (SEC), for purposes of registering the resale of the ordinary shares issued or issuable in connection with the Private Placement.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor may there be any sale of any securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On July 27, 2023 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported positive topline data from its Phase 3 trials ATLAS and ENVISION studying UGN-102 (mitomycin) for intravesical solution in patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, JUL 27, 2023, View Source [SID1234633463]). Both ATLAS and ENVISION trials met their primary endpoints.

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In the ATLAS trial, UGN-102 met its primary endpoint of disease-free survival, reducing risk of recurrence, progression, or death by 55%. UGN-102 also showed a 64.8% complete response rate at three months for patients who only received UGN-102, compared to a 63.6% complete response rate at three months for patients who only received a TURBT.

The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.2% rate of complete response at 3-months following the initial treatment. 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.

"UGN-102 has demonstrated a robust and consistent therapeutic profile across multiple clinical trials, providing a compelling picture of its potential to be a transformational product and advance the standard of care away from repetitive surgery to a minimally invasive, non-surgical option for LG-IR-NMIBC," says Liz Barrett, President and Chief Executive Officer of UroGen. "If approved, we anticipate UGN-102 to be a significant growth driver for UroGen as the first-ever non-surgical treatment option for a disease afflicting approximately 82,000 new patients in the U.S. each year. We are on track to deliver on our previously shared guidance for JELMYTO and now find ourselves on the precipice of a new era in bladder cancer care, and that is a very exciting place to be."

In both trials, UGN-102, a sustained release, hydrogel-based formulation that is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means, was generally well tolerated, with a side effect profile similar to that of previous clinical trials.

"While TURBT is the standard treatment for bladder cancer, the recurrent nature of LG-IR-NMIBC means that patients will undergo multiple surgeries that come with risks for this older patient population," says Sandip Prasad, MD, M.Phil., Director of Genitourinary Surgical Oncology, Morristown Medical Center/Atlantic Health System, NJ. "Based on these compelling data, I am optimistic that UGN-102, if approved, may change the treatment paradigm for these patients who lack non-surgical options to manage the ongoing burden of this highly recurrent disease."

The Phase 3 ATLAS clinical trial, the predecessor to ENVISION, evaluated the efficacy, durability, and safety of UGN-102 with or without TURBT vs. TURBT alone in 282 patients with LG-IR-NMIBC. ENVISION evaluated the efficacy and safety of UGN-102 as a primary chemoablative therapy in 240 patients with LG-IR-NMIBC.

UGN-102 ATLAS & ENVISION Top-line Data Review

The company is hosting a data event featuring a panel discussion with leading bladder cancer experts on Thursday, July 27, 2023 at 10:00 a.m. Eastern Time to discuss these findings and will highlight topline results from the Phase 3 ATLAS and ENVISION clinical trials.

Please register for the webinar under the Events & Presentations section of the Company’s Investor Relations site (View Source).

Following the live webcast, a replay will be available on the Company’s website (View Source).

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. Assuming positive findings from the 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 ENVISION

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 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 ATLAS

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

On July 27, 2023 PharmaMar (MSE:PHM) reported total revenues of €80.2 million in 1H2023, compared with €101.4 million in 1H2022 (Press release, PharmaMar, JUL 27, 2023, View Source [SID1234633462]). Recurring revenues, the sum of net sales plus royalties from sales made by our partners, totaled €67.3 million, compared with €86.7 million reported to June 30th, 2022. This difference is mainly due to the entry into the European market of two generic trabectedin products (Yondelis), which have put significant pressure on prices. Yondelis reported net sales of €14.2 million in the first half of the year, compared with €35.9 million in the same period of the previous year. Yondelis, the standard treatment in Europe for second-line soft tissue sarcoma, received its first marketing authorization in 2007 and has therefore, been on the market for more than 15 years.

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Zepzelca (lurbinectedin) revenues in Europe from the early access program amounted to €21.0 million in the first half of the year in France, Austria and Spain, compared with €11.1 million in the same period of 2022. This increase is due to a positive adjustment made by the French authorities in relation to the previous year’s discounts. No further adjustments are expected. The number of units sold was similar to the same period of the previous year.

Also included within recurring revenues, sales of raw materials to our partners, both Yondelis and lurbinectedin, amounted to €8.2 million through June 30th, 2023, compared to €15.4 million reported in the first half of the previous year.

Royalty revenues totaled €22.8 million to June 30th, representing an increase of 6% compared with the first half of last year. These revenues include royalties received from our partner Jazz Pharmaceuticals for lurbinectedin sales in the US, which, through June 30th, amounted to €21.0 million (June 2022: €19.9 million). The royalties recorded for the first half of the year are an estimate, as information on sales by Jazz is not available at the date of publication of this report. Any discrepancies will be corrected in the following quarter.

In addition to the royalties received from Jazz Pharmaceuticals, royalties on Yondelis sales received from our partners in the United States and Japan totaled €1.8 million in the first half of this year (€1.6 million in the same period of 2022).

Non-recurring revenues, mainly from licensing agreements, totaled €12.7 million to June 30th, 2023, compared with €14.7 million through June 30th, 2022. In the first half of 2023 as well as 2022, the revenues recorded for this item came entirely from licensing agreements related to lurbinectedin.

In the first six months of this year, total R&D expenditure increased by 16% to €46.6 million. Of this figure, €39.0 million corresponds to the oncology area and is mainly related to the confirmatory Phase III trial of lurbinectedin in Small-Cell Lung Cancer, called "LAGOON", which is progressing in patient recruitment, as well as activities prior to the start of two other Phase III trials with lurbinectedin in the indications of mesothelioma and leiomyosarcoma, respectively. We continue to invest in the development of the Phase II clinical trial with ecubectedin in solid tumors, as well as in Phase I clinical trials with ecubectedin, PM534 and PM54 for the treatment of solid tumors.

PharmaMar realized net profit of €6.4 million to June 30th, 2023 (vs. €34.9 million in the same period of the previous year). This result is particularly noteworthy in view of the pressure on Yondelis sales prices and the Company’s growing R&D efforts to develop new treatments.

Finally, the PharmaMar Group ended the first half of 2023 with a cash and cash equivalents position of €202.3 million and total debt of €39.8 million, resulting in net cash of €162.5 million.

On July 27, 2023 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that the UPLIFT clinical trial of upifitamab rilsodotin (UpRi) did not meet its primary endpoint (Press release, Mersana Therapeutics, JUL 27, 2023, View Source [SID1234633461]). UpRi is an ADC targeting the sodium-dependent phosphate transport protein NaPi2b and was developed utilizing the company’s Dolaflexin platform.

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UPLIFT was a single-arm clinical trial that enrolled platinum-resistant ovarian cancer patients with one to four prior treatment regimens. The primary endpoint for UPLIFT was the investigator-assessed objective response rate (ORR) in the NaPi2b-positive population. NaPi2b-positive status was defined by a tumor proportion score (TPS) of ≥75%. Secondary endpoints for the trial included the investigator-assessed ORR among all patients in the trial, duration of response (DOR), and safety and tolerability. The trial also included an assessment of ORR and DOR by independent radiology review (IRR).

UPLIFT enrolled a total of 268 patients, 141 of whom were determined to be NaPi2b positive. The median prior lines of systemic therapy among all patients was three, with 31% of the population having received four prior lines of treatment. Additionally, 84% of patients received prior bevacizumab and 69% received prior PARP inhibitor treatment.

Top-line results from UPLIFT as of the May 31, 2023 data cutoff date are as follows:

NaPi2b-Positive Population (n=141)

Investigator Assessment IRR Assessment
ORR, n (%) [95% confidence interval] 22 (15.6%) [10.0%, 22.7%] 23 (16.3%) [10.6%, 23.5%]
Partial Response, n (%) 20 (14.2%) 16 (11.3%)
Complete Response, n (%) 2 (1.4%) 7 (5.0%)
Median DOR, months 7.4 Not Reached

Total Population (n=268)

Investigator Assessment IRR Assessment
ORR, n (%) 35 (13.1%) 35 (13.1%)
Partial Response, n (%) 32 (11.9%) 24 (9.0%)
Complete Response, n (%) 3 (1.1%) 11 (4.1%)
Median DOR, months 7.4 10.7

Safety and tolerability data in UPLIFT were generally consistent with prior disclosures. Following the completion of its analyses, the company plans to share detailed efficacy and safety data with the medical and scientific community in an appropriate forum.

"We are deeply disappointed that UPLIFT’s efficacy failed to replicate previous data from approximately 100 patients in the dose expansion portion of our Phase 1b clinical trial," said Dr. Arvin Yang, Senior Vice President and Chief Medical Officer of Mersana Therapeutics. "While the duration of response was longer than that from the dose expansion portion of UpRi’s Phase 1b clinical trial, the lower bound of the confidence interval for the primary endpoint did not meet our goal of excluding a 12% ORR seen with standard-of-care single-agent chemotherapy. We are in the process of conducting an in-depth analysis of various factors to better understand the results as well as the characteristics of patients who responded to UpRi therapy, particularly those whose responses were deep and durable. We extend our deepest gratitude to all of the patients, family members, caregivers and investigators who contributed to UPLIFT."

Strategic Reprioritization and Financial Update

"As an organization driven by a mission to discover and develop potentially life-changing ADCs for patients fighting cancer, UPLIFT’s outcome is clearly disappointing and requires us to reprioritize our areas of focus," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "In recent years, Mersana has advanced new product candidates and developed new partnerships utilizing its next-generation ADC platforms, Dolasynthen and Immunosynthen. These will be our primary focus moving forward. We plan to complete the dose escalation portion of our Phase 1 clinical trial of XMT-1660 in 2023 and initiate the dose expansion portion of the trial in 2024. Our team continues working diligently to address the clinical hold on the Phase 1 clinical trial of XMT-2056. Additionally, we intend to strongly support Mersana’s collaborators as they leverage our next-generation ADC platforms and advance their product candidates."

"We also are taking decisive action to extend our cash runway and ensure we have the resources to evaluate the clinical potential of our next-generation ADC product candidates," Ms. Protopapas continued. "Unfortunately, this necessitates reducing our workforce by approximately 50%. A very talented group of employees who helped to build Mersana will be departing as a result of this strategic reprioritization. We are grateful for their many contributions and intend to provide strong support through their transition."

Mersana’s restructuring plan includes a wind-down of UpRi-related development activities, including its UP-NEXT and UPGRADE-A clinical trials and the company’s regulatory and commercial readiness efforts. If analyses of data enable the identification of a path forward for UpRi, the company will consider strategic alternatives for the asset, including partnering.

Mersana estimates that its balance of cash, cash equivalents and marketable securities as of June 30, 2023 was $286.6 million. This figure is preliminary and is subject to completion of the company’s financial closing procedures. The company expects to report its cash, cash equivalents and marketable securities, as well as other information necessary for a complete understanding of its financial position, in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2023. The company expects that its available funds will be sufficient to support its current operating plan commitments into 2026.

Conference Call Notice
Mersana will host a conference call today at 8:00 a.m. ET. To access the call, please dial (877) 270-2148 (domestic) or (412) 902-6510 (international). A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com, and a replay of the webcast will be available in the same location following the conference call for approximately 90 days.