On August 5, 2025 Intas Pharmaceuticals reported it has launched HETRONIFLY (Serplulimab), the first PD-1 inhibitor globally approved for the treatment of Extensive-Stage Small Cell Lung Cancer (ES-SCLC), in the Indian market (Press release, Intas Pharmaceuticals, AUG 5, 2025, View Source [SID1234654812]). This marks another major milestone, following the successful Launch in Europe.

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This launch follows a strategic licensing agreement between Intas Pharmaceuticals Limited and Shanghai Henlius Biotech, Inc., further strengthening Intas’ oncology portfolio and reinforcing its commitment to delivering cutting-edge therapies to patients in India.

Serplulimab is the first PD-1 inhibitor worldwide to receive approval for ES-SCLC, and is currently present in over 40 countries, including key European markets. Its efficacy is supported by the landmark ASTRUM-005 trial, which demonstrated a 40% reduction in the risk of death and remarkably higher overall survival rate versus the current standard-of-care Chemotherapy regimen. Notably, Serplulimab achieved an ESMO (Free ESMO Whitepaper)-MCBS score of 4/5—the highest among current immunotherapies, signifying highest clinical benefit in this indication.

The novel humanized mAb has a unique dual-blockade mechanism of PD-L1 and PD-L2, along with the highest PD-1 internalization, which sets a new benchmark in deep immune engagement for solid tumors. Globally, Serplulimab has been administered to over a lakh patients across a range of malignancies including SCLC, NSCLC, ESCC, and MSI-high cancers.

Despite the promise of immuno-oncology, cost remains a significant barrier in India. HETRONIFLY, introduced at approximately 75% lower cost than the currently available immunotherapies for this indication, underscores Intas’ commitment to providing high-quality innovative therapies at affordable prices to cancer patients in India.

Commenting on the launch, Binish Chudgar, Chairman & Managing Director of Intas Pharmaceuticals said, "The launch of HETRONIFLY reinforces our strategic position in oncology and reflects Intas’ commitment to accelerating access to globally validated therapies. It aligns with our operating model of delivering high-impact innovations to the Indian market with speed and cost-efficiency."

On August 5, 2025 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), an oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics, Triclonics and ADClonics), reported financial results for the second quarter and provided a business update (Press release, Merus, AUG 5, 2025, View Source [SID1234654811]).

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"We were excited to share the unprecedented efficacy of petosemtamab with pembrolizumab in first-line head and neck cancer at 2025 ASCO (Free ASCO Whitepaper), and were thrilled by the response from clinicians and KOLs, which we believe continues to drive strong phase 3 site activation and support our expectation that both phase 3 trials will be substantially enrolled by year end 2025," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "Additionally, I believe these data substantially enhance the likelihood of clinical success of petosemtamab. We are looking forward to providing initial clinical data on mCRC in the second half of 2025."

Petosemtamab (MCLA-158: EGFR x LGR5 Biclonics): Solid Tumors
LiGeR-HN1 phase 3 trial in 1L recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC) and LiGeR-HN2 phase 3 trial in 2/3L r/m HNSCC enrolling – with both trials expected to be substantially enrolled by YE25 and potential top line interim readout for one or both trials in 2026; phase 2 trial in 1L, 2L and 3L+ metastatic colorectal cancer (mCRC) enrolling; mCRC initial clinical data planned for 2H25

Merus provided updated interim clinical data from the phase 2 trial of petosemtamab with pembrolizumab as 1L treatment for PD-L1+ (CPS≥1) r/m HNSCC at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, demonstrating a 63% response rate among 43 evaluable patients and a 79% overall survival rate at 12 months. The presentation was detailed in our press release, Merus’ Petosemtamab with Pembrolizumab Interim Data Demonstrates Robust Efficacy and Durability in 1L PD-L1+ r/m HNSCC (May 22, 2025).

LiGeR-HN1, a phase 3 trial evaluating the efficacy and safety of petosemtamab in combination with pembrolizumab in 1L PD-L1+ r/m HNSCC compared to pembrolizumab, and LiGeR-HN2, a phase 3 trial evaluating the efficacy and safety of petosemtamab in 2/3L HNSCC compared to standard of care, are enrolling and we expect both trials to be substantially enrolled by YE25.

Merus believes a randomized registration trial in HNSCC with an overall response rate endpoint could potentially support accelerated approval and the overall survival results from the same study could potentially verify its clinical benefit to support regular approval for the Company’s phase 3 trial in 1L, and in phase 3 trial in 2/3L HNSCC. We expect to provide topline interim readout of one or both phase 3 registration trials in 2026.

In February 2025, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation (BTD) to petosemtamab in combination with pembrolizumab for the first-line treatment of adult patients with recurrent or metastatic programmed death-ligand 1 (PD-L1) positive HNSCC with combined positive score (CPS) ≥ 1. This designation was detailed in our press release, Petosemtamab Granted Breakthrough Therapy Designation by the U.S. FDA for 1L PD-L1 Positive Head and Neck Squamous Cell Carcinoma (February 18, 2025). BTD was also granted for petosemtamab monotherapy for the treatment of patients with recurrent or metastatic HNSCC whose disease has progressed following treatment with platinum based chemotherapy and an anti-programmed cell death receptor-1 (PD-1) or anti-programmed death ligand 1 (PD-L1) antibody, detailed in our press release, Petosemtamab granted Breakthrough Therapy Designation by the U.S. FDA (May 13, 2024).

Merus provided updated interim clinical data on petosemtamab monotherapy in 2L+ r/m HNSCC at the European Society for Medical Oncology Asia Congress, demonstrating a 36% response rate among 75 evaluable patients. The oral presentation was detailed in our press release, Merus’ Petosemtamab Monotherapy Interim Data Continues to Demonstrate Clinically Meaningful Activity in 2L+ r/m HNSCC (Dec. 7, 2024).

A phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 1L and 2L mCRC, and as monotherapy in heavily pretreated (3L+) mCRC, is enrolling. We expect to provide initial clinical data for petosemtamab in mCRC in 2H25.

BIZENGRI (zenocutuzumab-zbco: HER2 x HER3 Biclonics)
Approved by FDA for adults with pancreatic adenocarcinoma or non–small cell lung cancer (NSCLC) that are advanced unresectable or metastatic and harbor a neuregulin 1 (NRG1) gene fusion who have disease progression on or after prior systemic therapy

Merus has exclusively licensed to Partner Therapeutics, Inc. (PTx) the right to commercialize BIZENGRI for the treatment of NRG1+ cancer in the U.S. This was detailed in our press release, Merus and Partner Therapeutics Announce License Agreement for the U.S. Commercialization of Zenocutuzumab in NRG1 Fusion-Positive Cancer (December 2, 2024).

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Investigation of MCLA-129 is ongoing in METex14 NSCLC; phase 2 trial in combination with chemotherapy in 2L+ EGFR mutant (EGFRm) NSCLC enrolling

MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to develop MCLA-129, and potentially commercialize exclusively in China, while Merus retains global rights outside of China.

Collaborations

Incyte Corporation
Since 2017, Merus has been working with Incyte Corporation (Incyte) under a global collaboration and license agreement focused on the research, discovery and development of bispecific antibodies utilizing Merus’ proprietary Biclonics technology platform. For each program under the collaboration, Merus receives reimbursement for research activities and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved. During the second quarter of 2025, Merus received the milestone payment of $1 million for the candidate nomination of a discovery program under the collaboration.

Eli Lilly and Company
In January 2021, Merus and Eli Lilly and Company (Lilly) announced a research collaboration and exclusive license agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies utilizing Merus’ Biclonics platform and proprietary CD3 panel along with the scientific and rational drug design expertise of Lilly. The collaboration is progressing well with two programs advancing through preclinical development.

Gilead Sciences
In March 2024, Merus and Gilead Sciences announced a collaboration to discover novel antibody based trispecific T-cell engagers using Merus’ patented Triclonics platform. Under the terms of the agreement, Merus will lead early-stage research activities for two programs, with an option to pursue a third. Gilead will have the right to exclusively license programs developed under the collaboration after the completion of select research activities. If Gilead exercises its option to license any such program from the collaboration, Gilead will be responsible for additional research, development and commercialization activities for such program. The collaboration is progressing well with two programs advancing through preclinical development.

Ono Pharmaceutical
In 2018, the Company granted Ono Pharmaceutical Co., Ltd. (Ono) an exclusive, worldwide, royalty-bearing license, with the right to sublicense, research, test, make, use and market a limited number of bispecific antibody candidates based on Merus’ Biclonics technology platform directed to an undisclosed target combination.

Biohaven
In January 2025, Merus and Biohaven announced a research collaboration and license agreement to co-develop three novel bispecific antibody drug conjugates (ADCs), leveraging Merus’ leading Biclonics technology platform, and Biohaven’s next-generation ADC conjugation and payload platform technologies. Under the terms of the agreement, Biohaven is responsible for the preclinical ADC generation of three Merus bispecific antibodies under mutually agreed research plans. The agreement includes two Merus bispecific programs generated using the Biclonics platform, and one program under preclinical research by Merus. Each program is subject to mutual agreement for advancement to further development, with the parties then sharing subsequent external development costs and commercialization, if advanced.

Corporate Activities
Completed public offering raising $345M gross proceeds
This equity raise is detailed in a press release: Merus N.V. Announces Pricing of Public Offering of Common Shares (June 4, 2025).

Cash Runway, existing cash, cash equivalents and marketable securities expected to fund Merus’ operations at least into 2028

As of June 30, 2025, Merus had $892 million cash, cash equivalents and marketable securities. Based on the Company’s current operating plan, the existing cash, cash equivalents and marketable securities are expected to fund Merus’ operations at least into 2028.

Second Quarter 2025 Financial Results

Total revenue for the three months ended June 30, 2025 increased by $1.5 million as compared to the three months ended June 30, 2024, primarily as a result of increases in Incyte revenue of $1.5 million, PTx revenue of $0.5 million, and Gilead revenue of $0.1 million, partially offset by a decrease in Lilly revenue of $0.6 million.

Research and development (R&D) expense for the three months ended June 30, 2025 increased by $44.8 million as compared to the three months ended June 30, 2024. The increase in R&D expense is primarily driven by an increase of $37.9 million in clinical trial support provided by contract manufacturing and development organizations and contract research organizations, most of which is related to the petosemtamab clinical trials. The increase is also due to increases in personnel related expenses including share-based compensation of $7.2 million.

General and administrative expense for the three months ended June 30, 2025 increased by $2.7 million as compared to the three months ended June 30, 2024, primarily as a result of increases in personnel related expenses including share-based compensation of $5.0 million, increases in facilities and depreciation expense of $0.7 million, increases in legal expenses of $0.6 million, and increases in travel expenses of $0.2 million, partially offset by decreases in consulting expenses of $3.6 million and decreases in intellectual property and license expenses of $0.3 million.

Total revenue for the six months ended June 30, 2025 increased by $20.1 million as compared to the six months ended June 30, 2024, primarily as a result of commercial material revenue sold to PTx of $13.3 million and increases in collaboration revenue of $6.8 million. The collaboration revenue increase is primarily due to increases in Biohaven upfront payment amortization of $5.1 million.

Research and development expense for the six months ended June 30, 2025 increased by $86.3 million as compared to the six months ended June 30, 2024. The increase in R&D expense is primarily driven by an increase of $73.7 million in clinical trial support provided by contract manufacturing and development organizations and contract research organizations, most of which is related to the petosemtamab clinical trials.

General and administrative expense for the six months ended June 30, 2025 increased by $8.7 million as compared to the six months ended June 30, 2024, primarily as a result as a result of increases in personnel related expenses including share-based compensation of $10.3 million, increases in facilities and depreciation expense of $1.0 million, increases in legal expenses of $0.8 million, increases in finance and human resources expenses of $0.1 million and increases in travel expenses of $0.1 million, partially offset by decreases in consulting expenses of $3.4 million.

Other income (loss), net consists of interest earned and fees paid on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange (losses) gains on our foreign denominated cash, cash equivalents and marketable securities. Other gains or losses relate to the issuance and settlement of financial instruments.

MERUS N.V.
CONDENSED CONSOLIDATED BALANCE SHEETS
(UNAUDITED)
(Amounts in thousands, except share and per share data)

June 30,
2025 December 31,
2024
ASSETS
Current assets:
Cash and cash equivalents $ 442,791 $ 293,294
Marketable securities 267,433 243,733
Accounts receivable 16,889 1,261
Prepaid expenses and other current assets 41,169 30,784
Total current assets 768,282 569,072
Marketable securities 181,729 187,008
Property and equipment, net 11,346 10,770
Operating lease right-of-use assets 10,320 9,254
Intangible assets, net 1,797 1,679
Equity Investment 2,430 —
Deferred tax assets 758 1,520
Other assets 3,514 3,390
Total assets $ 980,176 $ 782,693
LIABILITIES AND SHAREHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 21,075 $ 4,164
Accrued expenses and other liabilities 41,387 43,957
Income taxes payable 478 7,317
Current portion of lease obligation 2,245 1,704
Current portion of deferred revenue 26,394 29,934
Total current liabilities 91,579 87,076
Lease obligation 8,754 8,208
Deferred revenue, net of current portion 38,107 39,482
Total liabilities 138,440 134,766
Commitments and contingencies – Note 6
Shareholders’ equity:
Common shares, €0.09 par value; 105,000,000 shares authorized at June 30, 2025 and December 31, 2024; 75,565,138 and 68,828,749 shares issued and outstanding as at June 30, 2025 and December 31, 2024, respectively 7,647 6,957
Additional paid-in capital 2,038,795 1,664,822
Accumulated other comprehensive income 18,373 (55,465 )
Accumulated deficit (1,223,079 ) (968,387 )
Total shareholders’ equity 841,736 647,927
Total liabilities and shareholders’ equity $ 980,176 $ 782,693

MERUS N.V.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(UNAUDITED)
(Amounts in thousands, except share and per share data)

Three Months Ended
June 30, Six Months Ended
June 30,
2025 2024 2025 2024
Commercial material revenue $ — $ — $ 13,331 $ —
Collaboration revenue 8,828 7,332 21,976 15,221
Royalty revenue — — 9 —
Total revenue 8,828 7,332 35,316 15,221
Operating expenses:
Research and development 93,926 49,119 174,042 87,703
General and administrative 25,252 22,587 47,364 38,701
Total operating expenses 119,178 71,706 221,406 126,404
Operating loss (110,350 ) (64,374 ) (186,090 ) (111,183 )
Other income, net:
Interest income, net 7,122 7,130 14,325 12,047
Foreign exchange gains (loss) (51,854 ) 9,519 (76,170 ) 18,053
Other expense (1,265 ) — (3,031 ) —
Total other income (loss), net (45,997 ) 16,649 (64,876 ) 30,100

Net loss before income taxes (156,347 ) (47,725 ) (250,966 ) (81,083 )
Income tax expense 1,871 2,317 3,726 3,415
Net loss $ (158,218 ) $ (50,042 ) $ (254,692 ) $ (84,498 )
Other comprehensive loss:
Currency translation adjustment 50,733 (8,978 ) 73,838 (16,366 )
Comprehensive loss $ (107,485 ) $ (59,020 ) $ (180,854 ) $ (100,864 )
Net loss per share attributable to common shareholders:
Basic and diluted $ (2.23 ) $ (0.81 ) $ (3.64 ) $ (1.41 )
Weighted-average common shares outstanding:
Basic and diluted 71,096,937 61,851,260 69,996,121 59,968,338

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.

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 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.