On August 13, 2024 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported its financial results for the second quarter of 2024, its recent business highlights, and a preview of select anticipated milestones (Press release, Sutro Biopharma, AUG 13, 2024, View Source [SID1234645827]).

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"We continue to make meaningful progress with the development of luvelta across multiple indications, including enrollment of a patient expansion cohort in combination with bevacizumab, nearing initiation of our second registration-enabling trial, REFRaME-P1, for pediatric patients with a rare form of acute myeloid leukemia (AML) and approaching site activation of a Phase 2 trial in non-small cell lung cancer (NSCLC)," said Bill Newell, Sutro’s Chief Executive Officer. "We plan to share supplemental data from our Phase 1b trial of luvelta in combination with bevacizumab at the ESMO (Free ESMO Whitepaper) meeting in September."

Mr. Newell added, "We are off to strong start in our new partnership with Ipsen for STRO-003 and continue to advance our preclinical pipeline of next-generation ADCs, including our tissue-factor targeting exatecan ADC, STRO-004. In parallel, we are exploring new partnership opportunities to maximize the potential of our platform and pipeline, led by our new Chief Business Development Officer Barbara Leyman. Additionally, we are delighted to welcome Sukhi Jagpal to our Board, as he brings a wealth of invaluable financial and strategic expertise."

Recent Business Highlights and Select Anticipated Milestones

Luveltamab Tazevibulin (luvelta), FRα-Targeting ADC Franchise:

Sutro will present updated data from the Phase 1b study of luvelta in combination with bevacizumab for patients with ovarian cancer in a poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 to be held September 13-17 in Barcelona, Spain.
Title: Luveltamab tazevibulin, an antifolate receptor alpha (FRα) antibody-drug conjugate (ADC), in combination with bevacizumab (bev) in patients with recurrent high-grade epithelial ovarian cancer (EOC): STRO-002-GM2 phase 1 study
Date: Saturday, September 14, 2024
Part 2 (randomized portion) of the Phase 3 trial, REFRαME-O1, for treatment of platinum-resistant ovarian cancer (PROC), is underway.
REFRαME-P1, a registration-enabling trial for pediatric patients with CBFA2T3::GLIS2 (CBF/GLIS; RAM phenotype) AML, is expected to be initiated in the second half of 2024.
A Phase 2 expansion study in combination with bevacizumab is ongoing, with data expected in the first half of 2025.
A Phase 2 trial for the treatment of NSCLC is expected to initiate in the second half of 2024, with initial data expected in the first half of 2025.
Additional Pipeline Development and Collaboration Updates:

In April 2024, Sutro announced a global licensing agreement for STRO-003, a ROR1-targeting ADC, with Ipsen.
Sutro plans to submit an IND for STRO-004 in 2025.
Sutro continues to seek to maximize the value of its proprietary cell-free platform by working with partners on programs in multiple disease spaces and geographies and has generated from collaborators an aggregate of approximately $970 million in payments through June 30, 2024, including equity investments.
Corporate Updates:

In August, Sutro strengthened its Board of Directors with the appointment of Sukhi Jagpal, MBA, CPA, CBV. Mr. Jagpal brings 20 years of experience in the life sciences industry, with expertise in financial management, communication, and organizational effectiveness, including financial analysis, mergers and acquisitions, and cost optimization.
In July, Sutro appointed Barbara Leyman, Ph.D., as Chief Business Development Officer, with a focus on building value and executing the Company’s business development strategy, in addition to serving on Sutro’s senior management team.
Upcoming Events: Sutro will participate in two upcoming investor conferences. Webcasts of the presentations will be accessible through the News & Events page of the Investor Relations section of the Company’s website at www.sutrobio.com. Archived replays will be available for at least 30 days after the events.

Wedbush PacGrow Healthcare Conference in New York, August 13-14, 2024
Wells Fargo Healthcare Conference in Boston, September 4-6, 2024
Second Quarter 2024 Financial Highlights

Cash, Cash Equivalents and Marketable Securities and Vaxcyte Common Stock
As of June 30, 2024, Sutro had $426.0 million, composed of cash, cash equivalents and marketable securities of $375.6 million and approximately 0.7 million shares of Vaxcyte common stock with a fair value of $50.4 million.

Unrealized Gain from Increase in Value of Vaxcyte Common Stock
The non-operating, unrealized gain of $4.8 million for the quarter ended June 30, 2024 was due to the increase since March 31, 2024 in the estimated fair value of Sutro’s holdings of Vaxcyte common stock. Vaxcyte common stock held by Sutro will be remeasured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue
Revenue was $25.7 million for the quarter ended June 30, 2024, as compared to $10.4 million for the same period in 2023, with the 2024 amount related principally to the Astellas collaboration and the Vaxcyte agreement. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments.

Operating Expenses
Total operating expenses for the quarter ended June 30, 2024 were $74.4 million, as compared to $56.6 million for the same period in 2023. The 2024 quarter includes non-cash expenses for stock-based compensation of $6.2 million and depreciation and amortization of $1.8 million, as compared to $6.7 million and $1.7 million, respectively, in the comparable 2023 period. Total operating expenses for the quarter ended June 30, 2024 were comprised of research and development expenses of $62.0 million and general and administrative expenses of $12.4 million.

On August 13, 2024 AstraZeneca reported that Imfinzi (durvalumab) and Lynparza (olaparib) have been approved in the European Union (EU) as treatment for certain patients with primary advanced or recurrent endometrial cancer (Press release, AstraZeneca, AUG 13, 2024, View Source [SID1234645826]). Imfinzi plus chemotherapy as 1st-line treatment followed by Lynparza and Imfinzi has been approved for patients with mismatch repair proficient (pMMR) disease. Imfinzi plus chemotherapy followed by Imfinzi alone has been approved for patients with mismatch repair deficient (dMMR) disease.

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The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on a prespecified exploratory subgroup analysis by mismatch repair (MMR) status from the DUO-E Phase III trial, which was published in the Journal of Clinical Oncology.

In the trial, the Lynparza and Imfinzi regimen reduced the risk of disease progression or death for patients with pMMR disease by 43% (median 15.0 months versus 9.7 months, hazard ratio [HR] 0.57; 95% confidence interval [CI] 0.44-0.73) versus the control arm.1 The Imfinzi regimen reduced the risk of disease progression or death among patients with dMMR disease by 58% (median not reached versus 7.0 months, HR 0.42; 95% CI 0.22-0.80) versus the control arm.1

Els Van Nieuwenhuysen, Gynaecological Oncologist at the UZ Leuven, Belgium and DUO-E trial investigator, said: "This approval is welcome news for patients with advanced or recurrent endometrial cancer in Europe, especially those with mismatch repair proficient disease who have limited options. The olaparib and durvalumab as well as the durvalumab regimens now have the potential to improve outcomes for all patients in this setting in Europe, regardless of mismatch repair status."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: "This approval of Imfinzi and Lynparza regimens marks the first-ever approval for a combination of an immunotherapy and PARP inhibitor in endometrial cancer and a major step forward for patients. In Europe, endometrial cancer is the fourth most common cancer in women, and until now, the 70 to 80 per cent of patients who have mismatch repair proficient disease have had few available treatment options."

The safety profiles of both regimens were generally manageable, well-tolerated and broadly consistent with the known profiles of the individual agents.1,2,3

Regulatory submissions for Imfinzi and Lynparza are currently under review in Japan and several other countries based on the DUO-E trial. Imfinzi plus chemotherapy was recently approved for dMMR patients with primary advanced or recurrent endometrial cancer in the US.

Notes

Endometrial cancer
Endometrial cancer is a highly heterogeneous disease that originates in the tissue lining of the uterus and is most common in women who have already been through menopause, with the average age at diagnosis being over 60 years old.4-7

The majority of patients with endometrial cancer are diagnosed at an early stage of disease, where the cancer is confined to the uterus.8 They are typically treated with surgery and/or radiation, and the five-year survival rate is high (approximately 80-90%).9 Patients with advanced disease (Stage III-IV) usually have a much poorer prognosis, with the five-year survival rate falling to less than 20%.10 Immunotherapy combined with chemotherapy is emerging as a new standard of care for advanced endometrial cancer, particularly for patients with dMMR disease, who make up approximately 20-30% of all patients.11-14 There is a significant need for new treatment options, especially for the 70-80% of patients with pMMR disease.15,16

In Europe, nearly 125,000 women were diagnosed with endometrial cancer in 2022.17,18

DUO-E
The DUO-E trial (GOG 3041/ENGOT-EN10) is a three-arm, randomised, double-blind, placebo-controlled, multicentre Phase III trial of 1st-line Imfinzi (durvalumab) plus platinum-based chemotherapy (carboplatin and paclitaxel) followed by either Imfinzi monotherapy or Imfinzi plus Lynparza (olaparib) as maintenance therapy versus platinum-based chemotherapy alone as a treatment for patients with newly diagnosed advanced or recurrent endometrial cancer.

The DUO-E trial randomised 699 patients with newly diagnosed advanced or recurrent epithelial endometrial carcinoma to receive either Imfinzi (1120mg) or placebo, given every three weeks in addition to standard-of-care platinum-based chemotherapy. After 4-6 cycles of chemotherapy, patients (whose disease had not progressed) then received either Imfinzi (1500mg) or placebo every four weeks as maintenance, plus 300mg Lynparza (300mg BID [2x150mg tablets, twice a day]) or placebo until disease progression.

The dual primary endpoint was progression-free survival (PFS) of each treatment arm versus standard-of-care chemotherapy alone, and both arms demonstrated a statistically significant and clinically meaningful improvement in PFS compared to standard of care in patients with newly diagnosed advanced or recurrent endometrial cancer.1 Key secondary endpoints included overall survival (OS), safety and tolerability. The trial continues to assess OS for both arms in the overall trial population. Mismatch repair (MMR) status, recurrence status and geographic location were stratification factors. The trial was sponsored independently by AstraZeneca and conducted in 253 study locations across 22 countries including the US, Europe, South America and Asia.

For more information about the trial, please visit ClinicalTrials.gov.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy and the global standard of care in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy. Imfinzi is also approved for the treatment of extensive-stage SCLC and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC.

Imfinzi also demonstrated statistically significant and clinically meaningful event-free survival results in patients with resectable early-stage NSCLC based on the AEGEAN Phase III trial. Imfinzi in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery is approved for patients in Switzerland and the UK based on this trial.

In limited-stage SCLC, Imfinzi demonstrated statistically significant and clinically meaningful improvements in the dual primary endpoints of OS and PFS compared to placebo in patients who had not progressed following standard-of-care concurrent chemoradiotherapy in the ADRIATIC Phase III trial.

In addition to its indications in lung cancers, Imfinzi is approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU and in combination with chemotherapy (carboplatin and paclitaxel) followed by Imfinzi monotherapy in primary advanced or recurrent endometrial cancer that is mismatch repair deficient in the US.

Since the first approval in May 2017, more than 220,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, breast cancer, bladder cancer, several gastrointestinal and gynaecologic cancers and other solid tumours.

Lynparza
Lynparza is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination-related (HRR) genes, such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as new hormonal agents [NHAs]).

Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza may also help enhance immunogenicity and increase the impact of anti-tumour immune responses.

Lynparza is currently approved in a number of countries across multiple tumour types, including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for germline BRCA mutation (gBRCAm), HER2-negative metastatic breast cancer (in the EU and Japan, this includes locally advanced breast cancer); for gBRCAm, HER2-negative high-risk early breast cancer (in Japan, this includes all BRCAm HER2-negative high-risk early breast cancer); for gBRCAm metastatic pancreatic cancer; in combination with abiraterone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) when chemotherapy is not clinically indicated (EU only) and for BRCAm mCRPC (US and Japan); and as monotherapy for HRR gene-mutated mCRPC in patients who have progressed on prior NHA treatment (BRCAm only in the EU and Japan). In China, Lynparza is approved for the treatment of BRCA-mutated mCRPC as well as 1st-line maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer.

Lynparza is being jointly developed and commercialised by AstraZeneca and MSD, both as a monotherapy and in combination with other potential medicines. Independently, the companies are developing and will commercialise Lynparza in combination with their respective PD-L1 and PD-1 medicines, Imfinzi (durvalumab) and Keytruda (pembrolizumab). Lynparza has been used to treat approximately 140,000 patients worldwide. Lynparza has a broad clinical trial development programme, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

On August 13, 2024 Medigene AG (Medigene or the "Company", FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, reported financial results for the six months ended June 30, 2024, and provided a corporate update (Press release, MediGene, AUG 13, 2024, View Source [SID1234645825]).

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The half-year 2024 earnings report can be found on the website: View Source

Select Half-Year 2024 Financial Results:

Revenues in the reporting period amounted to EUR 4.5 million, an increase of 46% compared to EUR 3.1 million in the first half of 2023. This increase compared to the first half of 2023 is due to the reversal of the remaining contractual liability to Hongsheng Sciences HK Limited (EUR 1.5 million) following the termination of the partnership.

General & administrative expenses decreased by 5% to EUR 4.1 million in the first half of 2024 (6M 2023: EUR 4.3 million including selling expenses), in particular due to lower personnel expenses.

Research and development expenses increased by 21% to EUR 6.3 million in the first half of 2024 (6M 2023: EUR 5.2 million). The reason for the increase is the focus on the development of TCR-T therapies (MDG1015, MDG2011 and MDG2021) for the treatment of solid tumors and preparatory activities for clinical trials (MDG1015).

The net result of the first half of 2024 improved by EUR 0.3 million to EUR -6.7 million compared to the first half of 2023 (6M 2023: EUR -7.0 million).

As of June 30, 2024, cash and cash equivalents amounted to EUR 14.0 million (December 31, 2023: EUR 16.7 million including time deposits). There were no open credit lines.

"In the first half of 2024, we are delighted to have again delivered on all the elements of our corporate strategy. We significantly advanced our End-to-End Platform, adding new technologies such as our innovative Interferon-gamma (IFNγ) Biosensor and our novel TCR-specific antigen-antibody combination technology, UniTope & TraCR. We advanced our lead program MDG1015 towards a first-in-human clinical trial, which is expected to start by the end of 2024 subject to additional financing," said Selwyn Ho, Chief Executive Officer of Medigene." We also successfully closed an oversubscribed capital raise with subscription rights resulting in gross proceeds of approximately EUR 5.9 million, which allowed us to extend our cash runway into July 2025. In addition, we announced our new strategic partnership with WuXi Biologics last week, expanding the ability to apply Medigene’s TCRs into new modalities, such as TCR-guided T cell engagers, to create additional value beyond TCR-T therapies for both, patients and our shareholders."

Financial Guidance 2024:

Performance in the first half of 2024 was in line with the Executive Management Board’s expectations.

On May 8, 2024, the Company reported that it had successfully completed an oversubscribed capital raise with gross proceeds of approximately EUR 5.9 million. The Company therefore amended its guidance and extended its cash runway into July 2025 (initial 2024 guidance: cash runway into April 2025).

Research and development costs were also amended and are expected to increase from prior estimates of EUR 11.0 to 13.0 million to EUR 11.5 to 13.5 million in 2024 to support additional activities enabling first patient enrollment for MDG1015 Phase 1 clinical trial by the end of 2024, subject to further financing.

The Company maintained its guidance on expected revenues to be between EUR 9.0 and 11.0 million (unchanged) in 2024.

These projections include potential future milestone payments from existing partnerships that are highly likely to materialize(see Annual Report 2023). They do not include potential milestone payments from or future/new partnerships or transactions as the occurrence of such payments or their timing and size largely depend on third parties and cannot be controlled or influenced by Medigene.

Program development highlights:

MDG1015: MDG1015 is a first-in-class, third generation T cell receptor engineered T cell (TCR-T) therapy targeting NY-ESO-1/LAGE-1a (New York esophageal squamous cell carcinoma 1 / L Antigen Family Member-1a), armored and enhanced by the costimulatory switch protein PD1-41BB and targeting HLA-A*02 (HLA, human leukocyte antigen). Pre-clinical data presented in 2024 at the AACR (Free AACR Whitepaper) and CHI conferences demonstrated the clear potential of MDG1015 to improve clinical outcomes in solid tumors with the innovative approach of combining a 3S (specific, sensitive and safe) TCR with the Company’s PD1-41BB costimulatory switch protein technology as well as an optimized drug product composition.

Following positive EU and US preliminary regulatory interactions, the Company remains on track for IND-filing in Q3 and CTA-filing in Q4 2024. Subject to financing, the Company expects to initiate a first in-human trial for MDG1015 by the end of 2024.

MDG2011: MDG2011 is a potential best-in-class third generation TCR-T therapy targeting KRAS G12V (HLA-A*11), further armored and enhanced by the PD1-41BB costimulatory switch protein. Pre-clinical data on MDG2011 was presented at the International Neoantigen Summit and the CIMT (Free CIMT Whitepaper) Annual Meeting in 2024.

MDG2021 MDG2021is the second candidate within the KRAS library targeting KRAS G12D (HLA-A*11) for which the Company announced the lead selection in June 2024.

MDG2012: The lead selection for the Company’s third announced KRAS-targeted program MDG2012,KRAS G12V (HLA-A*03), is expected in 2025.

Corporate development highlights:

In May 2024, Medigene successfully closed an oversubscribed capital raise with subscription rights resulting in gross proceeds of approximately EUR 5.9 million.

Subsequent to the quarter on August 8, 2024, the Company announced it has entered into a three-year, multi-target strategic partnership to design and co-research T cell receptor (TCR)-guided T Cell Engagers (TCR-TCEs) for the treatment of difficult-to-treat tumors with WuXi Biologics. The collaboration combines the respective expertise of each company with Medigene’s 3S TCR generation and characterization capabilities and WuXi Biologics’ unique anti-CD3 mAb, its industry-leading TCE platform and proprietary bispecific antibody platform WuXiBody. The companies aim to co-research TCR-TCE constructs, which will be owned by both cooperation partners with options to Medigene to further advance their development.

Expansion of patent portfolio:

The advancement of its End-to-End Platform allowed the Company to extend and strengthen its patent portfolio with new technologies as well as to expand existing patents into additional jurisdictions, such as the submission of three patents to the European Patent Office protecting the Company’s innovative Interferon-gamma (IFNγ) Biosensor and its novel T cell receptor (TCR)-specific antigen-antibody combination technology, UniTope & TraCR, the patent grant by the European Patent Office and Japan Patent Office protecting the inducible Medigene T cell receptor (iM-TCR) technology as well as the patent grant by the European Patent Office protecting the Company’s TCR targeting NY-ESO-1 and LAGE 1a. As of June 30, 2024, the patent portfolio consisted of 97 issued patents and 124 pending patent applications (December 31, 2023: 28 patent families, 112 issued patents and 131 pending patent applications).

Conference call and webcast:

The Company will host a conference call today, August 14, at 3:30 pm CEST / 9:30 am EDT. In addition to the financial results for the first six months of 2024, Medigene will provide a corporate update including its new partnership with WuXi Biologics (see press release on Aug 8, 2024).

Full details for the conference call and webcast are as follows:

Date Wednesday, August 14, 2024
Time 3:30 pm CEST (9:30 am EDT)
Conference call Registration conference call here
Webcast Join the live webcast here
Participants may pre-register and will receive dedicated dial-in details to easily and quickly access the call with the above registration link for the conference call.
Please dial in 10 minutes ahead of time to ensure a timely start of the conference call.
Following the call, an archived webcast will be accessible on the Investors & Media section on Medigene’s website: View Source

On August 13, 2024 XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its second quarter 2024 financial results and highlighted recent activities (Press release, Xoma, AUG 13, 2024, View Source [SID1234645824]).

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"The second quarter was marked by pipeline progress, the realization of several cash milestones, the addition of three commercial or late-stage programs, and the acquisition of Kinnate Pharmaceuticals," stated Owen Hughes, Chief Executive Officer of XOMA Royalty. "Most important, children suffering from relapsed or refractory low-grade glioma (pLGG) have a new option with the approval of Day One’s OJEMDA, which is now our fifth commercial royalty. And finally, with a robust cash position in hand, we look to further solidify our foundation for future growth via a disciplined approach to capital deployment."

Key Second Quarter Events

Partner

Event

Day One Biopharmaceuticals

The U.S. Food and Drug Administration (FDA) approved Day One’s OJEMDA (tovorafenib) for use in patients with pediatric low-grade glioma (pLGG). XOMA Royalty earned a $9.0 million milestone upon the approval and recorded $0.4 million in income resulting from OJEMDA sales in the second quarter of 2024. In addition, XOMA Royalty received an $8.1 million payment related to Day One’s sale of its priority review voucher.
Daré Bioscience

XOMA Royalty added economic interests to three best- or first-in-category assets to its portfolio. XACIATO vaginal gel 2% is commercially available and marketed by Organon. Bayer holds the U.S. rights to commercialize Ovaprene, a hormone-free monthly intravaginal contraceptive, currently in Phase 3 clinical trials. XOMA Royalty also acquired a synthetic royalty in Sildenafil Cream, 3.6%, a Phase 3-ready asset for female sexual arousal disorder. Daré recently published the efficacy results from its Phase 2b study of Sildenafil Cream, 3.6% in the publication Obstetrics & Gynecology.
Rezolute

RZ358—Dosed first patient in its Phase 3 trial of RZ358; XOMA Royalty earned a $5.0 million milestone associated with the event.

Presented Phase 2 RIZE study sub-analysis at the 2024 Pediatric Endocrine Society Annual Meeting.

RZ402—Presented positive topline results from its Phase 2 proof-of-concept study of RZ402 in patients with diabetic macular edema (DME). The data indicate RZ402 could be an effective oral therapy for patients with DME prior to anti-VEGF injections. Rezolute announced its intention to seek a partner for the next stage of development and future commercialization activities.

Takeda

Announced late-breaking data from Takeda’s Phase 2b study of mezagitamab demonstrating its potential to transform the treatment of primary immune thrombocytopenia1. In the study, patients receiving mezagitamab showed rapid and sustained increases in platelet counts that persisted 8 weeks after the last dose through to week 162.
Kinnate Pharmaceuticals

XOMA Royalty added several potential royalty streams, as well as more than $9.5 million to its cash balance as it completed the acquisition of Kinnate Pharmaceuticals.
LadRx

Regained development and commercialization rights to aldoxorubicin from ImmunityBio. XOMA Royalty is eligible to receive a low single-digit percent royalty on future sales of aldoxorubicin and a portion of any future milestone payments LadRx receives.

1
View Source

2
Kuter D, Pulanic D, et al. Safety, tolerability, and efficacy of mezagitamab (TAK-079) in chronic or persistent primary immune thrombocytopenia: Interim results from a phase 2, randomized, double-blind, placebo-controlled study. In: International Society on Thrombosis and Haemostasis (ISTH) Congress; June 22-26, 2024; Bangkok, Thailand. Abstract LB 01.1.

Subsequent Events
Partner

Event

Zevra Therapeutics

FDA convened a meeting of its Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to discuss the New Drug Application (NDA) for arimoclomol as a treatment in adults and pediatric patients 2 years and older with Niemann-Pick Disease Type C (NPC). The GeMDAC Advisory Committee voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with NPC. The Committee’s recommendation will be considered by FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the Agency.
Anticipated 2024 Events of Note
Partner

Event

Zevra Therapeutics

September 21, 2024 – FDA PDUFA action date for arimoclomol NDA
Takeda

In its press release dated June 22, 2024, Takeda announced plans to initiate a global Phase 3 trial of mezagitamab in ITP in the second half of fiscal year 2024.1
Second Quarter 2024 Financial Results

XOMA Royalty recorded total income and revenues of $11.1 million for the second quarter of 2024, which included $4.9 million in estimated income associated with two commercial products in our portfolio, $0.5 million in income from the $9.0 million milestone payment received from the FDA approval of OJEMDA, and $5.0 million in revenue from contracts with customers related to a milestone payment from Rezolute. In the second quarter of 2023, XOMA Royalty reported total income and revenue of $1.7 million, which included $1.1 million of revenue from contracts with customers related to a milestone earned from Janssen.

Research and development (R&D) expenses were $1.2 million in the second quarter of 2024, reflecting the ongoing clinical activities related to Kinnate’s Phase 1 clinical trial of KIN-3248, which XOMA Royalty assumed upon completing the Kinnate merger. The Company expects to incur additional R&D costs as this trial winds down in the second half of 2024. R&D expenses in the second quarter of 2023 were $39,000.

General and administrative ("G&A") expenses were $11.0 million for the second quarter of 2024 compared with $5.8 million in the second quarter of 2023. The increase of $5.2 million was driven primarily by expenses associated with our acquisition of Kinnate, which included $3.6 million in severance costs paid to Kinnate senior leadership, $1.0 million in consulting fees, and $0.8 million in other administrative costs.

In the second quarter of 2024, as a result of communications with Aronora, XOMA Royalty evaluated the status of the partnered programs for potential impairment and recorded a one-time, non-cash impairment charge of $9.0 million and a reduction of royalty receivables of $9.0 million associated with Aronora. In 2023, as a result of the announcement by Bioasis to suspend its operations and the termination of its research collaboration and license agreement with Chiesi, XOMA Royalty recorded a one-time, non-cash impairment charge of $1.6 million and a reduction of $1.6 million under long-term royalty receivables in the second quarter of 2023.

In the second quarters of 2024 and 2023, G&A expenses included $2.7 million and $2.2 million, respectively, in non-cash stock-based compensation expenses.

XOMA Royalty recorded a $19.3 million gain on the acquisition of Kinnate in the second quarter of 2024 due to the fair value of net assets that exceeded total purchase consideration.

During the second quarter of 2024, XOMA Royalty recognized an $8.1 million change in the fair value of an embedded derivative related to the payment of $8.1 million for the sale of a priority review voucher by Day One that was earned pursuant to XOMA Royalty’s RPA with Viracta.

Interest expense in the second quarter of 2024 was $3.4 million, representing interest related to the Blue Owl Loan established in December 2023.

The Company reported total other income, net, of $2.1 million in the second quarter of 2024, as compared to total other income, net, of $0.6 million in the corresponding period of 2023. The $1.5 million increase reflects a $1.2 million increase in investment income due to higher cash balances and higher market interest rates on our investments, as well as the change in the market price of Rezolute’s common stock.

Net income for the second quarter of 2024 was $16.0 million, compared to a net loss of $5.4 million for the second quarter of 2023.

On June 30, 2024, XOMA Royalty had cash and cash equivalents of $149.9 million (including $6.0 million in restricted cash). On December 31, 2023, XOMA Royalty had cash and cash equivalents of $159.6 million (including $6.3 million in restricted cash). During the second quarter of 2024, XOMA Royalty received $22.6 million in cash from royalty and milestone payments and deployed $22.0 million to acquire new royalty and milestone economic interests. Net cash used in operating activities during the quarter was $2.2 million. On July 15, 2024, the Company paid a total of $1.4 million in cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) and the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO).

On August 13, 2024 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, 2024, and provided an overview of recent developments (Press release, UroGen Pharma, AUG 13, 2024, View Source [SID1234645823]).

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"Our immediate priority is completing the submission of a New Drug Application in the very near term for UGN-102, which we believe has the potential to be a practice-changing therapy for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "The compelling body of clinical data, including the ENVISION trial, which demonstrated an unprecedented 82.3% 12-month duration of response by Kaplan-Meier analysis in patients who had previously achieved a complete response at three months, reinforces the opportunity for UGN-102 to be the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer."

Ms. Barrett continued, "We estimate that approximately 82,000 patients suffering from this highly recurrent disease each year may benefit from an innovative treatment, creating an estimated five-billion-dollar market opportunity. Our immediate commercial focus is preparing for UGN-102’s potential approval and launch with the goal to establish our leadership in urothelial cancers."

Q2 2024 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

In June 2024, UroGen reported positive 12-month duration of response (DOR) data from the Phase 3 ENVISION pivotal trial evaluating UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The 12-month DOR was 82.3% (95% CI, 75.9%, 87.1%) by Kaplan-Meier estimate in patients who had achieved complete response (CR) at three months from the first instillation of investigational drug UGN-102. The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102. UGN-102 was well tolerated, with a safety profile that was consistent with previous clinical trials.
The ENVISION 12-month DOR data were presented in a virtual event "New Horizons in Bladder Cancer" hosted by UroGen on June 13. This event included presentations by company management and several key opinion leaders with expertise in urology. There was also a panel discussion on the treatment of LG-IR-NMIBC and insights from a patient from the ENVISION trial. A replay of the event can be accessed here.
The latest DOR data is expected to support a New Drug Application (NDA) for UGN-102 as a treatment for LG-IR-NMIBC, which the Company plans to complete in the very near term. There is potential for an FDA decision as early as the first quarter of 2025, assuming the FDA grants priority review. UroGen initiated submission of the rolling NDA for UGN-102 in January 2024.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated net product revenue of $21.8 million in the second quarter of 2024, compared to $21.1 million in the second quarter of 2023.
JELMYTO was featured in three presentations at the AUA 2024 Annual Meeting. Independent long-term, real-world analyses explored use of the product in broad patient types, and with different methods of administration. The results showed that JELMYTO treatment appears to demonstrate favorable recurrence-free survival rates for patients with LG-UTUC who respond to initial induction. There does not appear to be a recurrence difference according to the intent of JELMYTO induction, original tumor size, multifocality or tumor location.
Next-generation novel mitomycin-based formulation for urothelial cancers

UroGen is developing UGN-103 and UGN-104, next-generation novel mitomycin-based formulations for UGN-102 and JELMYTO, respectively. These candidates combine UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH in an agreement signed in January 2024. The development programs potentially offer both manufacturing efficiencies and additional intellectual property protection for the Company’s low-grade urothelial cancer franchise.
In April 2024, the U.S. FDA accepted the Company’s Investigational New Drug (IND) application for UGN-103. If approved, UGN-103 is expected to provide several advantages related to production, cost, supply, and product convenience.
UroGen has initiated the Phase 3 study and has onboarded three clinical sites to explore the safety and efficacy of UGN-103 in LG-IR-NMIBC. UroGen plans to initiate a Phase 3 study of UGN-104 in LG-UTUC early next year.
Corporate

In June 2024, UroGen appointed David Lin as Chief Commercial Officer and member of the Executive Leadership Team. Mr. Lin is spearheading UroGen’s commercial strategy and will be leveraging his extensive experience to prepare for the potential launch of UGN-102, if approved.
Public offering of ordinary shares and pre-funded warrants

In June 2024, the Company completed an underwritten public offering of 5,000,000 ordinary shares at a price to the public of $17.50 per ordinary share, and, to certain investors in lieu of issuing ordinary shares, pre-funded warrants to purchase 1,142,857 ordinary shares at a purchase price of $17.499 per pre-funded warrant, which equals the public offering price per ordinary share less the $0.001 per share exercise price for each pre-funded warrant. Gross proceeds to UroGen from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $107.5 million.
In July 2024 the underwriters exercised their option to purchase the full 921,428 additional shares. This yielded further gross proceeds to the Company of $16.1 million, before deducting underwriting discounts and commissions and estimated offering expenses.
Second quarter 2024 financial results

JELMYTO Revenue: JELMYTO net product revenues were $21.8 million and $21.1 million for the three months ended June 30, 2024, and 2023, respectively.

R&D Expense: Research and development expenses for the second quarter of 2024 were $15.4 million, including non-cash share-based compensation expense of $0.6 million as compared to $11.6 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2023.

SG&A Expense: Selling, general and administrative expenses for the second quarter of 2024 were $30.1 million, including non-cash share-based compensation expense of $3.0 million. This compares to $22.5 million, including non-cash share-based compensation expense of $1.7 million, for the same period in 2023.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $5.8 million in the second quarter of 2024, compared to $5.3 million in the same period in 2023.

Interest Expense on Long-Term Debt: Interest expense related to the up to $200 million term loan facility with funds managed by Pharmakon Advisors was $3.5 million in the second quarter of 2024, compared to $3.8 million in the same period in 2023.

Net Loss: UroGen reported a net loss of $33.4 million or ($0.91) per basic and diluted share in the second quarter of 2024 compared with a net loss of $24.1 million or ($1.03) per basic and diluted share in the same period in 2023.

Cash & Cash Equivalents: As of June 30, 2024, cash, cash equivalents and marketable securities totaled $241.3 million.

2024 Revenue, Operating Expense, and RTW Expense Guidance: With respect to the Company’s previously provided full-year 2024 JELMYTO revenue guidance, the Company sees a path toward the lower end of the guidance range. With respect to the Company’s previously provided full-year 2024 operating expense guidance, the Company expects to be toward the higher end of the guidance range, with a revised non-cash share-based compensation expense of $9 to $13 million, subject to market conditions. The anticipated full year 2024 non-cash financing expense related to the prepaid obligation to RTW Investments is unchanged and expected to be in the range of $21 to $26 million. The rate for the cash component of the RTW obligation will be 13% of global net product sales of JELMYTO in 2024.

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 the treatment of adult patients with LG-UTUC. 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 innovative drug formulation of mitomycin, currently 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 by a trained healthcare professional. UroGen anticipates completing its NDA submission for UGN-102 in the very near term with a potential FDA decision as early as the first quarter of 2025, assuming priority review.