On November 4, 2025 AVEO Oncology, an LG Chem company (AVEO), and HiberCell, Inc. (HiberCell) reported that the companies have entered into an exclusive development and option agreement to develop HiberCell’s first-in-human human protein kinase RNA-like endoplasmic reticulum kinase (PERK) inhibitor, HC-5404, alone and in combination with AVEO’s potent and selective vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI), FOTIVDA (tivozanib). FOTIVDA is an anti-angiogenic agent approved by the U.S. Food and Drug Administration in March of 2021 for the treatment of adult patients with relapsed or refractory renal cell carcinoma (RCC) following two or more prior systemic therapies. Pre-clinical studies of HC-5404 in multiple solid tumor models, including gastric cancer and RCC, suggest the potential for enhanced anti-tumor efficacy when combined with an anti-angiogenic agent.

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Under the terms of the agreement, AVEO is responsible for conducting initial stage clinical development. During the Phase 2 clinical trial, AVEO will have the option to obtain an exclusive license for the development, manufacturing, and commercialization of HC-5404 in all therapeutic indications worldwide for an undisclosed option fee. In addition, if AVEO exercises its option, HiberCell will be eligible to receive milestone payments and royalties for global net sales of HC-5404, contingent upon successful development and commercialization.

"AVEO and LG Chem are excited to enter into this collaboration with HiberCell to advance the development of this first-in-human PERK inhibitor and to expand on our commitment to RCC patients," said Michael P. Bailey, President and CEO of AVEO. "HC-5404 has shown promising activity combined with VEGFR TKIs which makes pairing it with tivozanib in RCC a logical first phase of development. The combinability with our current portfolio in RCC, coupled with the opportunity to expand to other oncology indications, make this collaboration an important step in our vision to become a global leader in oncology."

"We are excited to be partnering with AVEO to help us explore the combination activity of HC-5404 and tivozanib in metastatic renal cell carcinoma," said Steven Gillis, Ph.D., Chairman and acting Chief Executive Officer at HiberCell. "AVEO brings considerable scientific and clinical development capabilities across oncology, including significant expertise in renal cell carcinoma. This collaboration is an important step in assessing the combination of HC-5404 and tivozanib in RCC and may inform broader development across multiple tumor types."

About FOTIVDA (tivozanib)
FOTIVDA (tivozanib) is an oral, next-generation vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI). It is a potent, selective inhibitor of VEGFRs 1, 2, and 3 with a long half-life designed to improve efficacy and tolerability. AVEO received U.S. Food and Drug Administration (FDA) approval for FOTIVDA on March 10, 2021, for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies, based on data from the TIVO-3 trial comparing FOTIVDA to sorafenib. FOTIVDA was approved in August 2017 in the European Union and other countries in the territory of its partner Recordati UK Ltd. for the treatment of adult patients with advanced RCC. FOTIVDA was discovered by Kyowa Kirin.

IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTION

Hypertension was reported in 45% of patients (22% ≥ Grade 3). Hypertensive crises were reported in 0.8% of patients. Do not initiate FOTIVDA in patients with uncontrolled hypertension. Monitor for hypertension and treat as needed. Reduce the FOTIVDA dose for persistent hypertension not controlled by anti-hypertensive medications. Discontinue FOTIVDA for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Cardiac failures were reported in 1.6% of patients (1% ≥ Grade 3); 0.6% of events were fatal. Monitor for signs or symptoms of cardiac failure during treatment with FOTIVDA. Manage with dose interruption, dose reduction, or discontinuation.

Cardiac ischemia were reported in 3.2% of patients; 0.4% of events were fatal. Arterial thromboembolic events were reported in 2.0% of patients, including death due to ischemic stroke (0.1%). Closely monitor patients at risk for, or who have a history of these events. Discontinue FOTIVDA in patients who develop severe arterial thromboembolic events, such as myocardial infarction and stroke.

Venous Thrombotic Events (VTE) were reported in 2.4% of patients, including 0.3% fatal events. Closely monitor patients who are at increased risk for these events. Discontinue in patients who develop serious VTEs.

Hemorrhagic Events were reported in 11% of patients; 0.2% of events were fatal. Use FOTIVDA with caution in patients who are at risk for or who have a history of bleeding.

Proteinuria was reported in 8% of patients (2% = Grade 3). Monitor during treatment with FOTIVDA. For moderate to severe proteinuria, reduce the dose or interrupt treatment. Discontinue in patients who develop nephrotic syndrome.

Gastrointestinal (GI) Perforation including fatal cases, has been reported in patients receiving FOTIVDA. Monitor for symptoms of GI perforation or fistula formation periodically throughout treatment with FOTIVDA. Permanently discontinue FOTIVDA in patients who develop severe or life-threatening GI perforation.

Thyroid Dysfunction events were reported in 11% of patients (0.3% ≥ Grade 3). Monitor thyroid function before and during treatment with FOTIVDA.

Wound Healing Complications: Withhold FOTIVDA for at least 24 days prior to elective surgery and do not administer for at least 2 weeks after major surgery and until adequate wound healing is observed.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS) can occur with FOTIVDA. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue if signs or symptoms of RPLS occur.

Embryo-fetal Toxicity: FOTIVDA can cause fetal harm. Advise patients of the potential risk to a fetus, to avoid becoming pregnant and to use contraception during treatment and for one month after the last dose of FOTIVDA. Advise males with female partners of reproductive potential to use effective contraception during treatment and for one month after the last dose of FOTIVDA.

Allergic Reaction to Tartrazine: FOTIVDA 0.89 mg capsule contains FD&C Yellow No. 5 (tartrazine) which may cause allergic-type reactions (including bronchial asthma) in certain susceptible patients.

ADVERSE REACTIONS

Common adverse reactions include fatigue/asthenia, hypertension, diarrhea, decreased appetite, nausea, dysphonia, hypothyroidism, cough, and stomatitis.

Serious adverse reactions include bleeding (3.5%), venous thromboembolism (3.5%), arterial thromboembolism (2.9%), acute kidney injury (2.3%), and hepatobiliary disorders (2.3%).

DRUG INTERACTIONS

Avoid coadministration with strong CYP3A4 inducers.

USE IN SPECIFIC POPULATIONS

Advise women not to breastfeed during treatment and for at least 1 month after the last dose.

The recommended dosage for patients with end-stage renal disease has not been established.

Reduce the FOTIVDA dose for patients with moderate hepatic impairment. The recommended dosage in patients with severe hepatic impairment has not been established.

To report SUSPECTED ADVERSE REACTIONS, contact AVEO Pharmaceuticals, Inc. at 1-833-FOTIVDA (1-833-368-4832) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for FOTIVDA (tivozanib).

(Press release, AVEO, NOV 4, 2025, View Source [SID1234659391])

On November 4, 2025 NEOK Bio, Inc., a biotechnology company focused on the development of novel antibody drug conjugates (ADCs) for improving outcomes for cancer patients, reported that it has emerged from stealth mode with $75 million in Series A financing. The company’s principal investor is ABL Bio, Inc., a leading Korean biotech company and a proven leader in antibody engineering. The funding will be used to advance two bispecific ADC programs into the clinic. The company aims to be a leading bispecific ADC company in the U.S.

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Leveraging ABL’s innovative ADC platform technology, NEOK is building a pipeline of bispecific ADCs by pursuing validated targets, while balancing high expression, selectivity, and target-related safety signals. NEOK utilizes a proprietary, linker-payload technology (SYNtecan E) that enables ADC generation with strong linker stability and superior biophysical properties. The company aims to overcome the efficacy and safety limitations of conventional ADCs through its bispecific approach which targets unique pairs of cancer targets.

"ADCs are a proven modality in treating select cancers, but historically have had limitations related to stability, selectivity, and therapeutic window. We believe our dual-targeting strategy has the potential to overcome drug resistance, target a wider range of tumors, increase internalization rates and cell killing, and improve the safety profile of ADCs by increasing selectivity and reducing off-tumor toxicity," said Mayank Gandhi, Chief Executive Officer of NEOK Bio. "The financing is a critical step in our journey to a clinical-stage company and enables the execution of a robust and efficient clinical development plan for our bispecific ADCs."

"Our investment in the formation of NEOK Bio underscores our commitment to deliver transformative therapeutic innovation to the dynamic and growing ADC landscape," said ABL Bio CEO Dr. Sang Hoon Lee. "We are excited to support an outstanding and experienced NEOK team as they aim to fulfill the significant untapped potential of bispecific ADCs to improve the lives of people with cancer."

The financing will support the initiation of clinical studies for NEOK’s two lead ADC candidates, which target proteins that are broadly expressed in multiple tumor types with significant unmet needs. They include NEOK001 (previously ABL206), a bispecific ADC targeting ROR1 and B7-H3, and NEOK002 (previously ABL209), a bispecific ADC targeting EGFR and MUC1 proteins. Both assets have the potential to demonstrate enhanced efficacy and safety over monovalent ADCs in large patient populations across thoracic, gastrointestinal, and gynecological cancers.

NEOK plans to file an Investigational New Drug (IND) application for both programs by early 2026 and initiate Phase 1 clinical trials in mid-2026 in the U.S. First data readouts from both programs are expected in 2027.

(Press release, Neok Bio, NOV 4, 2025, View Source [SID1234659407])

On November 4, 2025 Aligos Therapeutics, Inc. (Nasdaq: ALGS) a clinical stage biopharmaceutical company focused on improving patient outcomes through best-in-class therapies for liver and viral diseases, reported that members of management will present at two upcoming investor conferences.

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Jefferies London Healthcare Conference

Fireside Chat on Monday, November 17, 2025 at 3:00pm GMT
Piper Sandler 37th Annual Healthcare Conference

Fireside Chat on Wednesday, December 3, 2025 at 2:00pm ET
The presentations will premiere at the time listed above and can be accessible by visiting the Presentation & Events section on the "Investors" page of Aligos’ website at www.aligos.com. A replay of the webcast will be available following the presentation for at least 30 days.

(Press release, Aligos Therapeutics, NOV 4, 2025, View Source [SID1234659342])

On November 4, 2025 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a leading precision medicine oncology company, reported a business update and announced financial results for the third quarter ended September 30, 2025.

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"This quarter we continued to make significant progress across the pipeline and broader business, including the partnership with Servier that extends our runway into 2030 and enables potential commercialization of darovasertib outside of the United States. We have also provided multiple major medical conference clinical data updates at WCLC, ESMO (Free ESMO Whitepaper) and SMR, and completed our third IND filing in 2025 to further extend our industry leadership in precision medicine oncology," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

Selected Pipeline Developments and Upcoming Milestones

Darovasertib

•
Metastatic uveal melanoma (mUM)
o
Median progression-free survival (PFS) data from the Phase 2/3 trial (OptimUM-02) of darovasertib in combination with crizotinib in first line (1L) HLA*A2:01-negative mUM is on track to be reported by year-end 2025 to 1Q 2026; this data has the potential to enable an accelerated approval filing in the United States. The trial is nearing full enrollment, which remains on track to be completed by year-end.
o
In October 2025, data from the single-arm, Phase 2 trial (OptimUM-01) of darovasertib in combination with crizotinib were presented at the Society for Melanoma Research (SMR) Congress in Amsterdam, Netherlands. Data were from a total of 44 1L mUM patients, including both HLA*A2:01-negative and HLA*A2:01-positive patient subsets. Highlights include:
▪
21.1 month median overall survival (OS) and 7.0 month median progression free survival (PFS) across all patients
▪
In 41 efficacy-evaluable patients, confirmed overall response rate (ORR) of 34% (14/41) with a 9.0 month median duration of response (mDOR)
▪
Disease control rate (DCR) of 90% (37/41), with 85% (35/41) of patients achieving ‘any reduction’ in target lesions
▪
The combination continued to be well-tolerated with the most common treatment-related adverse events (TRAEs >30%) of diarrhea, nausea, edema, vomiting, dermatitis, hypoalbuminemia, and fatigue
•
Neoadjuvant therapy for primary uveal melanoma (UM)
o
In October 2025, the company presented positive data from the randomized Phase 2 trial (OptimUM-09) in a Proffered Paper Oral Presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in Berlin, Germany. Data were from a total of 95 patients, including 56 recommended for enucleation (EN) and 39 eligible for plaque brachytherapy (PB). Highlights include:
▪
Ocular tumor shrinkage in ~83% (78/94) of patients assessed, the majority of whom achieved ≥20% tumor shrinkage
▪
Among evaluable EN patients, the eye preservation rate was 57% (24/42), which increased to 95% (19/20) in patients achieving ≥20% tumor shrinkage
▪
In evaluable PB eligible patients, ~70% (26/37) achieved a reduction in predicted radiation dose to the eye from baseline, resulting in ~65% (24/37) having lower predicted risk of vision loss 3-years post-PB treatment
▪
During neoadjuvant treatment with darovasertib, ~55% (29/53) of EN and ~61% (23/38) of PB patients showed an improvement in baseline visual acuity scores (VAS), with a mean gain of 17 and 10 letters, respectively

▪
Darovasertib continued to be well-tolerated, with a low rate of serious adverse events and TRAEs leading to discontinuation
o
IDEAYA has initiated a randomized Phase 3 registration-enabling trial of darovasertib as a single-agent in the neoadjuvant setting of primary UM. The trial, referred to as OptimUM-10, will enroll a total of approximately 450 patients in two cohorts of PB- and EN-recommended patients.
▪
Target enrollment was revised downward from our previous estimate of 520 patients due to a reduction of 70 patients in the PB cohort (prior guidance of 400, now 330 patients) based on additional FDA feedback on the statistical plan (alpha usage) across the two cohorts.
•
Adjuvant therapy for primary UM
o
In collaboration with Servier, IDEAYA plans to initiate a global Phase 3 combination trial of darovasertib and crizotinib as an adjuvant therapy for primary UM in the first half of 2026.
IDE397 (MAT2A)

•
Positive data were reported from the ongoing Phase 1/2 trial of IDE397 in combination with Gilead’s Trodelvy (sacituzumab govitecan-hziy), a Trop2-directed antibody-drug conjugate (ADC), in patients with MTAP-deleted urothelial cancer (UC).
o
57% ORR (4/7; 4cPR) at 30 mg IDE397 plus 7.5mg/kg Trodelvy (Dose level 2).
o
Median PFS and mDOR were not yet reached.
o
Manageable safety profile consistent with known adverse events of both drugs as single agents.
•
IDEAYA has selected Dose level 2 as the go-forward dose for the IDE397 and Trodelvy clinical combination in MTAP-deleted UC and has achieved first-patient-in (FPI) in NSCLC. The next clinical update from the combination trial is planned for a medical conference in the first half of 2026.
IDE849 (DLL3 TOP1i ADC)

•
IDEAYA’s partner, Hengrui Pharma, presented clinical safety and efficacy data from over 70 small-cell lung cancer (SCLC) patients from their Phase 1 clinical trial at the 2025 International Association for the Study of Lung Cancer ("IASLC") World Conference on Lung Cancer (WCLC) in Barcelona, Spain. Data included 87 patients with small-cell lung cancer (SCLC) and 13 patients with other neuroendocrine carcinomas (NEC) as of a cut-off date of June 20, 2025. A total of 71 refractory (2L+) SCLC patients were evaluated for efficacy at doses of IDE849 between 2.4mg/kg and 4.2 mg/kg. Highlights include:
o
At the 2.4 mg/kg expansion dose of IDE849, 2L patients demonstrated an 80.0% (8/10) ORR and 70.0% (7/10) confirmed ORR; across all lines of therapy (2L+) at this dose the ORR and confirmed ORR decreased modestly to 73.7% (14/19) and 57.9% (11/19) (1 pending confirmation), respectively.
o
Across all doses of IDE849 tested, 2L patients showed a 77.1% (27/35) ORR and 60.0% (21/35) confirmed ORR (4 pending confirmation) whereas a 73.2% (52/71) ORR and 47.9% (34/71) confirmed ORR (10 pending confirmation) was observed across all lines of therapy at all doses (≥2.4 mg/kg).
o
Patients with baseline brain metastases had an 83.3% (5/6) confirmed ORR at the 2.4 mg/kg dose and a 66.7% (12/18) confirmed ORR (1 pending confirmation) across all doses (≥2.4 mg/kg).
o
6.7 month median PFS achieved across all lines and all doses (≥2.4 mg/kg); the median PFS was not reached in 2L patients.
•
In May 2025, IDEAYA initiated a global Phase 1 trial of IDE849 and has achieved FPI in the United States. The company continues to enroll SCLC patients with plans to expand into patients with neuroendocrine tumors (NETs) and other DLL3-overexpressing tumors by the end of 2025.
Other programs

•
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.
•
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.
•
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 dose escalation in combination with niraparib, GSK’s small molecule inhibitor of PARP, is ongoing in patients with solid tumors.
o
Phase 2 dose expansion in BRCA-mutant solid tumors would trigger a $10 million milestone payment from GSK.
•
IDE892, a potential best-in-class MTA-cooperative PRMT5 inhibitor, is being developed for patients with MTAP-deleted lung cancer and other high priority MTAP-deleted solid tumor indications. IDEAYA received IND clearance from the FDA in the third quarter and expects to begin a Phase 1 dose escalation trial by the end of the year with the goal of advancing into combination trials with IDE397 in the first half of 2026.

In the fourth quarter IDEAYA submitted an IND for IDE034, a potential first-in-class B7H3/PTK7 bispecific TOP1i ADC, and is on track to file an IND for IDE574, a potential first-in-class KAT6/7 dual inhibitor, by the end of the year.
License agreement with Servier

•
IDEAYA entered into an exclusive license agreement with Servier for rights to darovasertib outside the United States. The company received an upfront payment of $210 million, and is eligible for up to $100 million in regulatory approval-based milestone payments and up to $220 million in commercial milestone payments, as well as double-digit royalties on net sales in all territories outside of the United States. IDEAYA and Servier will collaborate on the development of darovasertib and share the associated costs. IDEAYA retains all rights to darovasertib in the United States.
Financial Results for the Quarter Ended September 30, 2025

As of September 30, 2025, IDEAYA had cash, cash equivalents and marketable securities of approximately $1.14 billion, compared to $991.9 million as of June 30, 2025. The increase was primarily driven by the $210.0 million upfront payment received from Servier related to the exclusive license agreement for darovasertib, offset by the net cash used in operations.

Collaboration revenue for the three months ended September 30, 2025, totaled $207.8 million compared to zero for the three months ended June 30, 2025. Collaboration revenue was recognized for the performance obligations satisfied through September 30, 2025 related to the development and commercialization license recognized upon execution and the research and development services that will be recognized over time under the Servier exclusive license agreement for darovasertib. As of September 30, 2025, the remaining balance for the research and development services performance obligations is $143.1 million related to the clinical development cost reimbursements anticipated under the license agreement that will be recognized as IDEAYA collaboration revenue over time as the research and development services are completed.

Research and development (R&D) expenses for the three months ended September 30, 2025 totaled $83.0 million compared to $74.2 million for the three months ended June 30, 2025. The increase was primarily driven by higher clinical trial and CMC manufacturing expenses to support our programs.

General and administrative (G&A) expenses for the three months ended September 30, 2025 totaled $16.4 million compared to $14.6 million for the three months ended June 30, 2025. The increase was primarily due to higher legal expenses to support company growth and commercial expenses to support the darovasertib commercial preparation activities.

The net income for the three months ended September 30, 2025, was $119.2 million compared to the net loss of $77.5 million for the three months ended June 30, 2025. Total stock compensation expense for the three months ended September 30, 2025, was $12.2 million compared to $11.9 million for the three months ended June 30, 2025.

(Press release, Ideaya Biosciences, NOV 4, 2025, View Source [SID1234659358])

On November 4, 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 September 30, 2025.

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"We are a global commercial company with multiple products generating meaningful growth that is expected to accelerate from anticipated launches from our late-stage clinical pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We announced today that we bolstered our balance sheet with a royalty financing ahead of pivotal milestones expected over the next year to support multiple late-stage data readouts, multiple regulatory submissions, and multiple launches. This includes the highly anticipated phase 3 study readouts for UX143 in osteogenesis imperfecta around the end of the year."

Third Quarter 2025 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 47,003 $ 35,604 $ 136,810 $ 112,294
Royalty revenue – U.S. and Canada 57,186 55,985 177,122 163,432
Royalty revenue – Europe 7,754 6,258 21,282 18,376
Total Crysvita Revenue 111,943 97,847 335,214 294,102
Dojolvi 24,275 21,374 64,491 57,091
Evkeeza 16,717 10,657 42,321 21,788
Mepsevii 6,998 9,616 23,695 22,372
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353

Total Revenues
Ultragenyx reported $160 million in total revenue for the third quarter of 2025, which represents 15% growth compared to the same period in 2024. Crysvita revenue in the third quarter 2025 was $112 million, which includes product sales of $47 million from Latin America and Türkiye. Dojolvi revenue in the third quarter 2025 was $24 million. Evkeeza revenue in the third quarter 2025 was $17 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 September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353
Operating expenses:
Cost of sales 27,991 21,021 79,655 59,834
Research and development 216,212 170,109 546,720 510,099
Selling, general and administrative 86,620 80,351 261,063 239,115
Total operating expenses 330,823 271,481 887,438 809,048
Net loss $ (180,413 ) $ (133,516 ) $ (446,444 ) $ (435,798 )
Net loss per share, basic and diluted $ (1.81 ) $ (1.40 ) $ (4.55 ) $ (4.91 )

Operating Expenses

Total operating expenses for the third quarter of 2025 were $331 million, including non-cash stock-based compensation of $37 million.

Net Loss
For the third quarter of 2025, Ultragenyx reported net loss of $180 million, or $1.81 per share basic and diluted, compared with a net loss for the third quarter of 2024 of $134 million, or $1.40 per share basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $447 million as of September 30, 2025.

The company announced today that it received $400 million through the sale of an additional 25% of its royalty interest on the future sales of Crysvita in the United States and Canada, to OMERS. Payments to OMERS will begin in January of 2028. OMERS will also continue to receive 30% of Crysvita net sales in the U.S. and Canada following the achievement of the 2022 royalty purchase agreement transaction’s cap of 1.45 times the purchase price. Total payments to OMERS pursuant to the new agreement are capped at 1.55 times the 2025 purchase price.

For the three months ended September 30, 2025, net cash used in operations was $91 million and for the nine months ended September 30, 2025 was $366 million.

2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14-20% compared to 2024.

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
Ultragenyx also reaffirmed its net cash used in operations guidance for 2025, which is expected to modestly increase compared to 2024 and its path to full year GAAP profitability in 2027.

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 final analyses at which time patients will have been on therapy for at least 18 months. Data from these studies are expected around the end of 2025.

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

In July 2025, enrollment of the global Phase 3 Aspire study was completed 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. Data from this study are expected in the second half of 2026.

Enrollment has begun in the supportive Phase 2/3 Aurora study, which is evaluating GTX-102 in other Angelman syndrome genotypes and ages.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): expect to resubmit Biologics License Application (BLA) early in 2026

Following receipt of a Complete Response Letter (CRL), the company has had constructive formal and informal discussions with the FDA. The additional clinical data requested by the agency, and that will be included in the BLA, continues to show a durable treatment effect across multiple biomarkers and further clinical separation from natural history, while maintaining an acceptable safety profile.

The company plans to resubmit the BLA early in 2026 and will be followed by an up to 6-month review per FDA regulations.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA rolling submission underway, expect to complete in the fourth quarter of 2025

Rolling submission of a BLA for DTX401 for the treatment of GSDIa began in August 2025. The BLA will include data from the randomized, placebo-controlled Phase 3 study that demonstrated statistically significant and clinically meaningful reductions in daily cornstarch intake compared with placebo at Week 48. It will also include longer-term data that was announced in September 2025 that demonstrated patients showed an even greater reduction in mean daily cornstarch intake in the 48-week crossover period. Both the originally treated DTX401 group (n=20) and the crossover group (n=19) who received DTX401 at Week 48 had a mean reduction in daily cornstarch intake of 61% at Week 96. Quality of life improved following treatment with DTX401 for patients in both groups as measured by the Patient Global Impression of Change (PGIC). At Week 96, improvements in disease management were reported by 83% (10/12) of patients in the DTX401 group and 95% (18/19) of patients in the crossover group.

Rolling submission of the BLA is expected to complete in the fourth quarter of 2025.

UX701 AAV gene therapy for Wilson Disease: Cohort 4 enrollment complete, data expected in the first half of 2026

In September 2025, the company completed enrollment of 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. A total of five patients were enrolled in Cohort 4. These patients received 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. Data from this study are expected in the first half of 2026.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, November 4, 2025, at 2 p.m. PT/5 p.m. ET to discuss the third 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.

(Press release, Ultragenyx Pharmaceutical, NOV 4, 2025, View Source [SID1234659374])