On August 5, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended June 30, 2025 (Press release, Ultragenyx Pharmaceutical, AUG 5, 2025, View Source [SID1234654801]).

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"In the first half of the year, we delivered 20% revenue growth from our commercial therapies versus the prior year. We are continuing along our path to profitability in 2027, as we drive our top line growth and maintain our fiscal discipline," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are excited for the potential of UX143 in osteogenesis imperfecta to reduce fractures and meaningfully improve patients’ bone health and for GTX-102 in Angelman syndrome to transform the lives of patients and their families affected by this neurodevelopment disease."

Second Quarter 2025 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 34,727 $ 40,449 $ 89,807 $ 76,690
Royalty revenue – U.S. and Canada 79,083 67,045 119,936 107,447
Royalty revenue – Europe 6,596 6,176 13,528 12,118
Total Crysvita Revenue 120,406 113,670 223,271 196,255
Dojolvi 23,207 19,355 40,216 35,717
Evkeeza 14,573 7,856 25,604 11,131
Mepsevii 8,310 6,145 16,697 12,756
Total revenues $ 166,496 $ 147,026 $ 305,788 $ 255,859

Total Revenues
Ultragenyx reported $166 million in total revenue for the second quarter of 2025, which represents 13% growth compared to the same period in 2024. Second quarter 2025 Crysvita revenue was $120 million, which includes product sales of $35 million from Latin America and Türkiye. Dojolvi revenue in the second quarter 2025 was $23 million. Evkeeza revenue in the second quarter 2025 was $15 million as we continue to launch in the Ultragenyx territories outside of the United States.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Total revenues $ 166,496 $ 147,026 $ 305,788 $ 255,859
Operating expenses:
Cost of sales 23,002 21,280 51,664 38,813
Research and development 164,736 161,503 330,508 339,990
Selling, general and administrative 86,646 80,604 174,443 158,764
Total operating expenses 274,384 263,387 556,615 537,567
Net loss $ (114,951 ) $ (131,598 ) $ (266,031 ) $ (302,282 )
Net loss per share, basic and diluted $ (1.17 ) $ (1.52 ) $ (2.73 ) $ (3.54 )

Operating Expenses

Total operating expenses for the second quarter of 2025 were $274 million, including non-cash stock-based compensation of $39 million.

Net Loss
For the second quarter of 2025, Ultragenyx reported net loss of $115 million, or $1.17 per share basic and diluted, compared with a net loss for the second quarter of 2024 of $132 million, or $1.52 per share basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $539 million as of June 30, 2025, which includes $80 million of net proceeds raised through the At-The-Market (ATM) facility. For the three months ended June 30, 2025, net cash used in operations was $108 million and for the six months ended June 30, 2025 was $275 million.

2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14-20% compared to 2024. Net cash used in operations is now expected to modestly increase compared to 2024, related to timing delays and changes for UX111, DTX401, and UX143 impacting receipts and payments. The company reaffirms its path to GAAP profitability in 2027 and plans to continue to focus on growing revenues and prioritizing its spend, including stopping and delaying certain expenses prior to upcoming potential commercial launches.

Reaffirm for the full year 2025:

Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million

Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Final analysis for Phase 3 Orbit and Cosmic studies around the end of 2025

The Phase 3 Orbit and Cosmic studies, which evaluate setrusumab in pediatric and young adult patients with OI, are progressing towards their final analyses around the end of 2025. The randomized, placebo-controlled Phase 3 portion of the Orbit study was evaluated by the Data Monitoring Committee at an interim analysis in July 2025 and they informed the company that UX143 demonstrated an acceptable safety profile and that the study should continue to the final analysis. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2.

Data from the Cosmic study were not analyzed at the interim timepoint, consistent with the statistical analysis plan. Study conduct is going well and safety in this younger patient population is consistent with the safety profile in the other studies.

Patients will continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical studies with the final analyses to be conducted after patients have been on therapy for at least 18-months. The threshold for the Phase 3 Orbit final analysis is p<0.04 and for the Phase 3 Cosmic final analysis is p<0.05.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study fully enrolled; Phase 3 data expected in the second half of 2026

In June 2025, Breakthrough Therapy Designation (BTD) was granted by the FDA for GTX-102 as a treatment for Angelman syndrome. The FDA’s decision was based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients (4-17 years of age) with a full maternal UBE3A gene deletion, that showed participants have made consistent developmental gains with rapid, sustained and continuing improvements across multiple symptom domains when treated for up to 3 years. BTD aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.

In July 2025, enrollment of the global Phase 3 Aspire study was completed, ahead of plan due to patient and investigator interest, with 129 patients screened and randomized across 28 global sites. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a 10% allocation of alpha) is the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Data from this study are expected in the second half of 2026.

The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in the second half of 2025.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Working with FDA to resolve observations from Complete Response Letter (CRL)

In July 2025, the FDA issued a CRL for the Biologics License Application (BLA) for UX111 requesting additional information and improvements related to specific aspects of chemistry, manufacturing and controls (CMC) procedures and validation as well as observations from the recently completed manufacturing facility inspections. The company believes the observations are readily addressable and many have been addressed. The company will work with the FDA through a Type A meeting to agree on the planned resolution of the observations. Once agreement on the contents of a filing have been reached, the company expects to resubmit the BLA and anticipates up to a 6-month review period to follow the resubmission.

Clinical review had been ongoing and the FDA has acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence. The CRL did not note any review issues related to the clinical data package nor clinical inspections and specified that updated clinical data for particular endpoints from the current patients be included in the resubmission.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in the fourth quarter of 2025

A BLA for DTX401 for the treatment of GSDIa is planned to be submitted in the fourth quarter of 2025. The BLA will include data from the randomized, placebo controlled Phase 3 study and the previously disclosed 96-week data that demonstrated patients had even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-60%) and the Crossover Placebo to DTX401 group (-64%) when compared to the 48-week data. It will also include updates to proactively respond to related FDA observations identified in the UX111 CRL in the CMC section and at the company’s gene therapy manufacturing facilities.

UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; Cohort 4 enrollment ongoing, completion expected in second half of 2025

Enrollment is ongoing in the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. The company is on track to enroll five patients in Cohort 4 who will receive immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Enrollment in Cohort 4 is expected to complete in the second half of 2025.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, August 5, 2025, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.

On August 5, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the second quarter ended June 30, 2025, provided an update on its recent corporate activities and raised its 2025 financial guidance (Press release, Halozyme, AUG 5, 2025, View Source [SID1234654786]).

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"We are very excited to report another quarter of exceptional growth with a 65% increase in royalty revenue driven by our current three blockbuster therapies, DARZALEX SC, Phesgo, and VYVGART Hytrulo. In the second quarter, four notable approvals include RYBREVANT SC in Europe, VYVGART Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy in Europe and the VYVGART Hytrulo pre-filled syringe for gMG and CIDP in the U.S and Europe. Additional regulatory milestones were achieved with ENHANZE, including new indications and geographic expansion for DARZALEX SC, Opdivo SC, HYQVIA and Phesgo, which will further accelerate our future growth trajectory and enhance patient access. Based on the strong performance and growth trends, we are pleased to increase our full-year 2025 financial guidance ranges for the second time this year," said Dr. Helen Torley, President and CEO, Halozyme.

"The strong results highlight the momentum and durability across our business, powered by high-margin royalty streams and increasing global demand for our industry-leading ENHANZE drug delivery technology. In the quarter, we completed a total of $303 million in share repurchases, including the $250 million program that we announced in May. Our outperformance and strong cash generation supports a balanced capital allocation strategy, including investing in growth through M&A and returning capital to shareholders," concluded Dr. Torley.

Second Quarter and Recent Corporate Highlights:
•In June 2025, Halozyme initiated the third $250 million share repurchase tranche under the $750 million approved program from February 2024, of which $53.5 million was used to repurchase approximately 1.0 million shares at an average price of $52.40 per share in June 2025 for a total of $303.4 million of share repurchases in the second quarter.
•In May 2025, Halozyme announced a second $250 million share repurchase under the $750 million approved program from February 2024. The second $250 million share repurchase was completed in June 2025, resulting in a total purchase of 4.8 million shares at an average price of $52.09 per share.
•In April 2025, Halozyme filed a patent infringement lawsuit against Merck Sharp & Dohme Corp. ("Merck") in the U.S. District Court in New Jersey alleging that Merck is using Halozyme’s patented MDASE subcutaneous ("SC") drug delivery technology to develop SC Keytruda. Halozyme is seeking damages and injunctive relief to stop Merck’s infringement of Halozyme’s MDASE intellectual property.

Second Quarter and Recent Partner Highlights:
•In July 2025, Janssen announced the European Commission approved a new indication for DARZALEX SC as a monotherapy for the treatment of adult patients with smouldering multiple myeloma ("SMM") at high risk of developing multiple myeloma.
•In June 2025, Takeda announced the Ministry of Health, Labour and Welfare in Japan approved HYQVIA SC with ENHANZE for treatment of patients with chronic inflammatory demyelinating polyneuropathy ("CIDP") and multifocal motor neuropathy.
•In June 2025, argenx announced European Commission approval of VYVGART SC with ENHANZE for the treatment of adult patients with progressive or relapsing active CIDP after prior treatment with corticosteroids or immunoglobulins. VYVGART SC injection is available as a vial or prefilled syringe and can be administered by a patient, caregiver, or healthcare professional.
•In May 2025, Bristol Myers Squibb received European Commission approval of Opdivo SC, the SC formulation of Opdivo (nivolumab) developed with ENHANZE for use across multiple adult solid tumors, resulting in the recognition of $12.0 million in milestone revenue.
•In May 2025, argenx initiated a Phase 1 study to evaluate ARGX-213 with ENHANZE.
•In May 2025, Janssen announced the U.S. Food and Drug Administration ("FDA") Oncologic Drug Advisory Committee voted in favor of the benefit-risk profile of DARZALEX Faspro for the treatment of adult patients with high risk SMM.
•In April 2025, Roche received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use recommending an update to the European Union ("EU") label for Phesgo for human epidermal growth factor receptor 2-positive breast cancer. Administration of Phesgo outside of a clinical setting (such as in a person’s home) by a healthcare professional will be possible, once safely established in a clinical setting.
•In April 2025, argenx received FDA approval of VYVGART Hytrulo prefilled syringe for self-injection for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive and adult patients with CIDP.

•In April 2025, Janssen received European Commission marketing authorization of the SC formulation of RYBREVANT (amivantamab) with ENHANZE, in combination with LAZCLUZE (lazertinib), for the first-line treatment of adult patients with advanced non-small cell lung cancer ("NSCLC") with epidermal growth factor receptor ("EGFR") exon 19 deletions or exon 21 L858R substitution mutations. Additionally, RYBREVANT (amivantamab) is approved as a monotherapy for adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after the failure of platinum-based therapy. This represents the tenth partnered product with ENHANZE to be commercialized. In May 2025, amivantamab was made available to patients in the EU resulting in a $10.0 million milestone payment.
•In April 2025, Janssen received European Commission approval for an indication extension of DARZALEX SC in combination with bortezomib, lenalidomide, and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma regardless of transplant eligibility.

Second Quarter 2025 Financial Highlights:
•Revenue was $325.7 million, compared to $231.4 million in the second quarter of 2024. The 41% year-over-year increase was primarily driven by royalty revenue growth and an increase in milestone revenues. Revenue included $205.6 million in royalties, an increase of 65% compared to $124.9 million in the second quarter of 2024, primarily attributable to increases in revenue of DARZALEX SC, VYVGART Hytrulo and Phesgo.
•Cost of sales was $46.4 million, compared to $39.6 million in the second quarter of 2024. The increase in cost of sales was primarily due to an increase in product sales and labor allocation initiatives.
•Amortization of intangibles expense remained flat at $17.8 million, compared to the second quarter of 2024.
•Research and development expense was $17.5 million, compared to $21.0 million in the second quarter of 2024. The decrease in research and development expense was primarily due to lower compensation expense driven by resource optimization and labor allocation initiatives, and timing of planned investments in ENHANZE related to the development of our new high-yield rHuPH20 manufacturing process.
•Selling, general and administrative expense was $41.6 million, compared to $35.7 million in the second quarter of 2024. The increase was primarily due to an increase in consulting and professional service fees, including $2.6 million of litigation costs incurred in connection with a patent infringement litigation case, and an increase in compensation expense.
•Operating income was $202.4 million, compared to $117.2 million in the second quarter of 2024.
•Net income was $165.2 million, compared to $93.2 million in the second quarter of 2024.
•EBITDA was $222.9 million, compared to $137.0 million in the second quarter of 2024. Adjusted EBITDA was $225.5 million, compared to $137.0 million in the second quarter of 2024.1
•GAAP diluted earnings per share was $1.33, compared to $0.72 in the second quarter of 2024. Non-GAAP diluted earnings per share was $1.54, compared to $0.91 in the second quarter of 2024.1
•Cash, cash equivalents and marketable securities were $548.2 million on June 30, 2025, compared to $596.1 million on December 31, 2024. The decrease was primarily a result of share repurchase activities during the year, partially offset by cash generated from operations.

On August 5, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the 24-month DOR of 72.2% (95% CI 64.1%, 78.8%) by Kaplan-Meier estimate in patients who achieved CR at three months from the Phase 3 ENVISION trial of ZUSDURI (mitomycin) for intravesical solution, a treatment for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, AUG 5, 2025, View Source [SID1234654802]). The median follow-up time after a three-month CR in this analysis was 23.7 months. The median DOR has not been reached.

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"This latest update from the pivotal ENVISION trial of ZUSDURI showed that, among patients who achieved a complete response at three months, the probability of remaining event-free 24 months after CR was 72.2%," said Sandip Prasad, M.D., M.Phil., Director of Genitourinary Surgical Oncology and Vice Chair of Urology at Morristown Medical Center/Atlantic Health System, NJ, and Principal Investigator of the ENVISION trial. "Among patients who achieved a complete response, the event rate over time has remained stable. This DOR result highlights the sustained efficacy of the therapy and reinforces its potential as a durable treatment option. For the first time, adult patients with recurrent LG-IR-NMIBC have an FDA-approved therapy. This marks a potentially transformative step forward in how we manage this chronic, highly recurrent, and disruptive cancer. ZUSDURI is a new, outpatient treatment option for patients that can help provide a clinically meaningful recurrence-free interval, which is compelling given the highly recurrent nature of LG-IR-NMIBC."

The existing standard of care for LG-IR-NMIBC is a surgical procedure typically conducted under general anesthesia called transurethral resection of bladder tumor (TURBT). Repeated TURBT procedures can impact patients’ physical health and quality of life and can even be associated with an increased risk in mortality. Due to high recurrence rates, LG-IR-NMIBC patients, who are typically elderly with comorbidities, will likely need multiple TURBTs under general anesthesia over the course of their lifetime. An estimated 59,000 patients with LG-IR-NMIBC recur annually.

According to Mark Schoenberg, M.D., Chief Medical Officer, UroGen, "The 24-month duration of response data from the ENVISION trial underscore the transformative potential of ZUSDURI (formerly known as UGN-102) for adult patients with recurrent LG-IR-NMIBC. This is a population that has long endured a cycle of recurrence and repeat surgeries, a burden that weighs heavily, particularly on older adults. With ZUSDURI now approved, we have a new treatment option that can help deliver durable complete responses and meaningfully extend event-free intervals. This represents a major advance in the way we care for these patients."

The most common (≥ 10%) adverse reactions (ARs), including laboratory abnormalities, that occurred in patients were dysuria, increased potassium, increased creatinine, decreased hemoglobin, increased eosinophils, increased aspartate aminotransferase, increased alanine aminotransferase, decreased lymphocytes, urinary tract infection, decreased neutrophils, and hematuria. ARs were mainly mild to moderate. Serious ARs occurred in 12% of patients, including urinary retention (0.8%) and urethral stenosis (0.4%).

About ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology (a sustained release, hydrogel-based formulation), ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of ZUSDURI (mitomycin) for intravesical solution as a chemoablative therapy in adult patients with recurrent LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of ZUSDURI. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluates durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

On August 5, 2025 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), an oncology company committed to advancing the discovery, development, and commercialization of transformative precision medicines to address unmet medical needs in cancer, reported a business update and announced financial results for the second quarter ended June 30, 2025 (Press release, Ideaya Biosciences, AUG 5, 2025, View Source [SID1234654787]).

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"We look forward to a catalyst rich period with six clinical data updates guided from now to year-end across three clinical stage programs, including two oral presentations that have been accepted at major medical conferences and our targeted top-line randomized median PFS results for the darovasertib and crizotinib combination in 1L HLA-A2 negative MUM to potentially enable our first accelerated approval filing in the U.S. We are also excited to host our 10-year Anniversary R&D Day in New York City on September 8th, where we will present multiple data updates across our potential first-in-class clinical pipeline and highlight our strategic vision and pioneering research in cancer biology and drug discovery," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

Selected Pipeline Developments and Upcoming Milestones

Darovasertib

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Metastatic uveal melanoma (MUM)
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Median progression-free survival (PFS) data from the Phase 2/3 trial of darovasertib in combination with crizotinib in first line (1L) HLA-A2-negative MUM is on track to be reported by year-end 2025; this data has the potential to enable an accelerated approval filing in the United States. Over 350 patients have been enrolled in the trial as of August 4, 2025, and the company expects to complete full enrollment of approximately 400 patients by year-end. Based on feedback from the U.S. Food and Drug Administration (FDA), IDEAYA plans to submit median overall survival (OS) data from this trial to support full U.S. approval in HLA-A2-negative MUM.
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Median OS data from a single-arm, Phase 2 trial of darovasertib in combination with crizotinib will be presented at a medical conference in the fourth quarter of 2025. The readout will include data in over 40 patients, including both HLA-A2-negative and HLA-A2-positive patients. IDEAYA continues to enroll HLA-A2-positive patients in this trial to assess the benefit of the darovasertib/crizotinib combination to support a potential real world evidence (RWE) regulatory submission and/or compendia listing. If granted, this has the potential to broaden the use of darovasertib in MUM patients, independent of HLA status.
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Neoadjuvant therapy for primary uveal melanoma (UM)
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IDEAYA is also evaluating darovasertib as a monotherapy in the neoadjuvant setting for primary UM, where the goal of treatment is to prevent enucleation (surgical eye removal), preserve vision prior to and post-plaque brachytherapy, and slow disease progression and metastasis. Initial safety and visual benefit data will be reported from the Phase 2 clinical trial from over 20 patients in the plaque brachytherapy-eligible cohort at IDEAYA’s R&D Day on September 8th, followed by additional data from over 90 patients in both the enucleation-eligible and plaque brachytherapy-eligible cohorts in a Proffered Paper Oral Presentation at ESMO (Free ESMO Whitepaper), taking place from October 17-21, 2025 in Berlin, Germany.
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Following a successful Type D meeting with the FDA in April 2025, the company initiated a randomized Phase 3 registration-enabling trial of darovasertib in the neoadjuvant setting for primary UM in the third quarter of 2025. The trial, referred to as OptimUM-10, will enroll a total of approximately 520 patients in two cohorts of plaque brachytherapy-eligible and enucleation-eligible patients.
IDE397 (MAT2A)

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IDEAYA is conducting a Phase 1/2 clinical trial pursuant to a clinical study collaboration and supply agreement with Gilead to evaluate IDE397 in combination with Trodelvy (sacituzumab govitecan-hziy), Gilead’s Trop-2 directed ADC, in patients with MTAP-deletion urothelial cancer, or UC, and non-small cell lung cancer, or NSCLC.
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In April 2025, the companies announced expansion of the IDE397 and Trodelvy (sacituzumab govitecan-hziy) combination trial in NSCLC. IDEAYA will provide initial Phase 1 safety and efficacy data from two expansion cohorts in the IDE397 and Trodelvy (sacituzumab govitecan-hziy) combination trial in MTAP-deletion UC patients at the company’s R&D Day September 8th, with additional data targeted for a medical conference in the first half of 2026.
IDE849 (DLL3 TOP1i ADC)

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IDEAYA’s partner, Hengrui Pharma, is conducting a multi-site, open label Phase 1 clinical trial for IDE849 in China for patients with small-cell lung cancer (SCLC). Hengrui will present clinical safety and efficacy data from over 70 patients in the trial at the International Association for the Study of Lung Cancer ("IASLC") 2025 World Conference on Lung Cancer (WCLC) taking place from September 6-9, 2025 in Barcelona, Spain. The presentation will include data from the dose escalation and multiple expansion doses.
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In May 2025, IDEAYA initiated a Phase 1 trial in the U.S. in SCLC. Patient dosing in NETs and other DLL3-expressing tumors is targeted by year-end 2025.
Other programs

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IDE161, a potential first-in-class small molecule poly-(ADP-ribose) glycohydrolase, or PARG, inhibitor is currently in Phase 1 dose optimization to inform future combination studies with IDE849 and other TOP1i-based ADCs where PARG inhibition may synergize with the payload to deepen responses. IDEAYA plans to initiate a Phase 1 combination trial of IDE849 and IDE161 by the end of 2025. The company will also share preclinical data in a poster presentation at WCLC providing combination mechanism and pre-clinical synergy data between TOP1-payload based ADCs and IDE161.
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IDE275 (GSK959), a potential first-in-class small molecule inhibitor of Werner Helicase, is being developed in collaboration with GlaxoSmithKline (GSK). A Phase 1 dose escalation in patients with MSI-High solid tumors is ongoing.
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IDE705 (GSK101), a potential first-in-class small molecule inhibitor of DNA Polymerase Theta Helicase, or Pol Theta, is being developed in collaboration with GSK. A Phase 1 clinical trial in combination with niraparib, GSK’s small molecule inhibitor of PARP, is ongoing in patients with BRCA-positive or other HRD-positive tumors.
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Phase 2 expansion in HRD-positive solid tumors would trigger a $10 million milestone payment from GSK.
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IDEAYA also plans to submit three investigational new drug, or IND, applications before the end of the year:
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IDE892, a potential best-in-class MTA-cooperative PRMT5 inhibitor, in mid-2025;
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IDE034, a potential first-in-class B7H3/PTK7 bispecific TOP1i ADC, in the fourth quarter of 2025; and
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IDE574, a potential first-in-class KAT6/7 dual inhibitor, in the fourth quarter of 2025.

R&D Day – September 8, 2025

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IDEAYA will host an in-person and virtual R&D Day on September 8th, 2025 from 8:00-10:00 AM ET in New York City. The company will present multiple clinical data updates across the pipeline and highlight future growth drivers and upcoming milestones. Speakers will include members of IDEAYA’s senior leadership team and key opinion leader(s). Additional agenda details will be provided by the end of August 2025.
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Registration for this event can be accessed here or at the investors section of the IDEAYA website at View Source
Other corporate updates

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IDEAYA continues efforts to scale the organization in preparation for the potential U.S. launch of darovasertib, including key hires within the commercial, medical affairs and market access functions.
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Gary Palmer, M.D., joined as Senior Vice President, Medical Affairs, where he will lead the company’s medical affairs activities. Gary joined IDEAYA with over 25 years of global leadership experience in medical affairs from biopharmaceutical companies of various sizes and stages, and across multiple therapeutic areas including oncology, pulmonary medicine, immunology and neurology. Most recently Gary was Senior Vice President of Medical Affairs at Pliant Therapeutics, and prior to that he was Senior Vice President of Global Medical Affairs Immunology & Neuroscience at Bristol-Myers Squibb Co (BMS) where he led the Worldwide Immunology, Fibrosis and Neuroscience Medical Affairs team covering a portfolio spanning four globally marketed medications and more than 15 development candidates across the areas of pulmonary fibrosis, dermatology, gastroenterology, rheumatology and neurology.

Financial Results for the Quarter Ended June 30, 2025

As of June 30, 2025, IDEAYA had cash, cash equivalents and marketable securities of approximately $991.9 million, compared to $1.05 billion as of March 31, 2025. The decrease was primarily driven by net cash used in operations.

Research and development (R&D) expenses for the three months ended June 30, 2025 totaled $74.2 million compared to $70.9 million for the three months ended March 31, 2025. The increase was primarily due to higher clinical trial expenses to support our clinical pipeline and personnel-related expenses.

General and administrative (G&A) expenses for the three months ended June 30, 2025 totaled $14.6 million compared to $13.5 million for the three months ended March 31, 2025. The increase was primarily due to higher personnel-related expenses to support our growth.

The net loss for the three months ended June 30, 2025, was $77.5 million compared to the net loss of $72.2 million for the three months ended March 31, 2025. Total stock compensation expense for the three months ended June 30, 2025, was $11.9 million compared to $10.2 million for the same period in 2024.

On August 5, 2025 SERB Pharmaceuticals ("SERB"), a global specialty pharmaceutical company focused on medicines for rare diseases and medical emergencies, and Y-mAbs Therapeutics, Inc. (Nasdaq: YMAB) ("Y-mAbs" or "the Company"), a commercial-stage biopharmaceutical company focused on the development and commercialization of antibody-based therapeutics for the treatment of cancer, reported that they have entered into a definitive merger agreement under which SERB will acquire Y-mAbs, including its lead commercial oncology asset, DANYELZA (naxitamab-gqgk), in an all-cash transaction, representing an equity value for Y-mAbs of approximately $412 million (Press release, Y-mAbs Therapeutics, AUG 5, 2025, View Source [SID1234654803]).

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Under the terms of the merger agreement, SERB will commence an all-cash tender offer to purchase all outstanding shares of Y-mAbs common stock. Holders of Y-mAbs common stock would receive $8.60 per share in cash, representing a premium of approximately 105% to Y-mAbs’ closing share price on August 4, 2025, the last full trading day prior to the transaction announcement.

The transaction was unanimously approved by the Y-mAbs Board of Directors following a review of strategic alternatives to maximize value for Y-mAbs stockholders, with the assistance of external advisors. The process included discussions with numerous potential buyers for Y-mAbs or for the DANYELZA or Radiopharmaceuticals businesses on a standalone basis. In addition, the Board reviewed potential sources of additional capital to support accelerating the further development of the Company’s pipeline.

With a focus on pediatric oncology, Y-mAbs successfully developed and commercialized the anti-GD2 therapy, DANYELZA (naxitamab-gqgk). DANYELZA is the first FDA-approved treatment for relapsed or refractory high-risk neuroblastoma – a rare and aggressive pediatric cancer – and was approved in the United States under accelerated approval based on overall response rate and duration of response. DANYELZA is indicated for the treatment of pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. In addition to inpatient use, doctors can choose to administer DANYELZA in an outpatient setting, which may reduce the logistical burden on patients and their families. Y-mAbs’ portfolio also includes an investigational therapy targeting GD2 in solid tumors and CD38 in circulating tumors in ongoing Phase 1 clinical trial from its Self-Assembly DisAssembly ("SADA") Pretargeted Radioimmunotherapy Platform ("PRIT").

Vanessa Wolfeler, Chief Executive Officer of SERB: "High-risk neuroblastoma is not only a rare and devastating pediatric cancer but also one of the most difficult to treat. DANYELZA is recognized as a critical treatment option for patients and expands the treatment pathways available to providers in an outpatient setting. Working together with the team from Y-mAbs, I believe we can continue generating data for this product, expand partnerships to additional oncology centers, and have a positive impact on the lives of more neuroblastoma patients and their families."

Jeremie Urbain, Chairman of SERB: "Following SERB’s expansion into the United States five years ago, this acquisition reflects another milestone in the execution of our growth strategy to build a leading global specialty pharma platform. DANYELZA is an excellent strategic fit for SERB as it strengthens our existing rare oncology portfolio and will allow us to leverage our existing global footprint and our medical, regulatory, and commercial expertise to expand the reach of DANYELZA to new markets."

SERB has a growing portfolio of medicines for rare emergency medicine, rare diseases, and CBRN preparedness, supplying healthcare providers around the world. The acquisition of DANYELZA broadens SERB’s existing Rare Oncology portfolio (Voraxaze, Vistogard, Xermelo) and furthers its mission of building a leading portfolio of medicines that improve the standard of care for patients globally.

Michael Rossi, President, Chief Executive Officer and a member of the Board of Directors, Y-mAbs: "Our Board regularly reviews our business, including our strategy, the current state of the biopharmaceutical sector and the time and resources required to execute on our strategic plans. Following the thorough process to explore all of the potential paths forward for the Company, we are now moving forward with this agreement with SERB that we believe reflects the most attractive option available to Y-mAbs, providing significant, immediate and certain value to our stockholders."

Mr. Rossi continued: "This transaction is a testament to our team’s hard work in building a strong foundation as a commercial organization with a differentiated, FDA-approved product in DANYELZA. We believe that Y-mAbs has made important progress advancing DANYELZA and our Radiopharmaceuticals platform. By combining our expertise and resources with SERB’s specialty commercial capabilities, we can extend our shared commitment of improving the lives of even more patients and families on a global scale."

Transaction Details

Under the terms of the merger agreement, SERB is obligated to commence a tender offer by August 19, 2025, to purchase all of Y-mAbs’ outstanding shares for $8.60 per share in cash. Assuming a majority of the outstanding Y-mAbs shares are tendered into, and not withdrawn from, the tender offer, and subject to the satisfaction of other customary conditions, including the receipt of a majority of Y-mAbs shares in the tender offer and expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, SERB is required to close the tender offer, following which, Y-mAbs will be merged with a subsidiary of SERB. After such merger, Y-mAbs shares that were not tendered in the tender offer will be converted into the right to receive the same $8.60 per share in cash paid for shares in the tender offer, and Y-mAbs’ stock will no longer be listed on the Nasdaq exchange.

Y-mAbs stockholders holding approximately 16% of Y-mAbs’ outstanding shares of common stock have entered into a tender and support agreement with SERB, pursuant to which such stockholders have agreed, among other things, to tender all of their shares of Y-mAbs common stock in the tender offer, subject to the terms and conditions of such agreement.

The transaction is expected to close by the fourth quarter of 2025.

Advisors

Rothschild & Co. is acting as exclusive financial advisor, Freshfields US LLP is acting as legal counsel and H/Advisors Abernathy is acting as strategic communications advisor to SERB.

Centerview Partners is acting as exclusive financial advisor, Cooley LLP is acting as legal counsel and Joele Frank, Wilkinson Brimmer Katcher is acting as strategic communications advisor to Y-mAbs.