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])

On November 4, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported financial results for the third quarter ended September 30, 2025, including sales of TAVALISSE (fostamatinib disodium hexahydrate), GAVRETO (pralsetinib) and REZLIDHIA (olutasidenib), and recent business progress.

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"Our strong third-quarter performance demonstrates our strategic focus on commercial execution, pipeline development, and financial discipline. We are raising our full-year 2025 guidance due to our outstanding commercial performance year-to-date," said Raul Rodriguez, Rigel’s president and CEO. "Our development pipeline continues to advance, including our ongoing Phase 1b study evaluating R289 for the treatment of patients with lower-risk MDS. With enrollment complete in the dose escalation phase of the study, we look forward to presenting updated data in an oral presentation at ASH (Free ASH Whitepaper) in December. We’re also excited to have reached the next milestone of this program with the initiation of the dose expansion phase of the study."

Third Quarter 2025 Business Update

Commercial

Net product sales were $64.1 million, an increase of 65% from the same period of 2024.
TAVALISSE was commercially launched in South Korea in July by JW Pharmaceutical Corporation, the licensing partner of Kissei Pharmaceutical Co., Ltd. (Kissei), Rigel’s partner in certain Asian countries.
Clinical Development

Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a potent and selective dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor, in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). Enrollment in the dose escalation phase of the study was completed in July. In October, Rigel announced the first patient was enrolled in the dose expansion phase of the study, where up to 40 patients will be randomized to either 500 mg once daily or 500 mg twice daily to determine the recommended Phase 2 dose for future clinical trials.
Rigel announced one oral presentation and four poster presentations highlighting data from its commercial and clinical-stage hematology and oncology portfolio at the upcoming 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The abstract with updated data from the ongoing Phase 1b study of R289 showed R289 continues to be generally well tolerated in a heavily pretreated lower-risk MDS patient population, the majority of whom were high transfusion burden at study entry, and preliminary signs of efficacy in dose levels of at least 500 mg once daily and higher. Updated data as of an October 28, 2025, data cut off will be presented in the oral presentation. The company will also present additional data for olutasidenib in patients with R/R mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML).
The fifth study under the strategic alliance between Rigel and The University of Texas MD Anderson Cancer Center opened for enrollment in September. This Phase 2 multi-arm, multi-center, open-label, non-randomized clinical study will evaluate olutasidenib in combination with co-targeted therapies in patients with R/R IDH1-mutated myeloid malignancies harboring activated signaling pathway mutations (NCT07032727). The primary objectives of the study are to evaluate safety and the composite complete remission rate.
In October, the first patient was enrolled in the CONNECT Phase 2 TarGeT-D study evaluating olutasidenib in combination with temozolomide, followed by olutasidenib monotherapy as a maintenance regimen for newly-diagnosed adolescent and young adult patients with a high-grade glioma harboring an IDH1 mutation (NCT06161974).
Corporate

Eli Lilly and Company (Lilly) continues to advance ocadusertib (previously R552 or LY3871801), an investigational, potent and selective receptor-interacting protein kinase 1 (RIPK1) inhibitor. Enrollment in the Phase 2a clinical trial in adult patients with moderately to severely active rheumatoid arthritis is ongoing. Rigel will continue to be entitled to receive milestones and tiered royalty payments on future net sales of ocadusertib.
In early October, Rigel received notification from Lilly that it will terminate the central nervous system (CNS) disease program related to the collaboration between the two companies, which will become effective 60 days following notification.
Third Quarter 2025 and Year-to-Date Financial Update
For the third quarter ended September 30, 2025, total revenues were $69.5 million, consisting of $64.1 million in net product sales and $5.4 million in contract revenues from collaborations. Net product sales grew 65% compared to $38.9 million in the same period of 2024. TAVALISSE net product sales were $44.7 million, growth of 70% compared to $26.3 million in the same period of 2024. GAVRETO net product sales were $11.1 million, growth of 56% compared to $7.1 million in the same period of 2024. REZLIDHIA net product sales were $8.3 million, growth of 50% compared to $5.5 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $3.1 million of revenue from Grifols S.A. (Grifols) related to delivery of drug supplies and earned royalties, $1.8 million of revenue from Kissei related to the delivery of drug supplies, and $0.2 million of revenue from Medison Pharma (Medison) related to delivery of drug supplies and earned royalties.

Total costs and expenses were $41.0 million compared to $41.3 million for the same period of 2024. The decrease in costs and expenses was mainly due to lower cost of product sales, as the prior period included a sublicensing fee. This decrease was partially offset by increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289 and higher personnel-related costs.

Rigel reported net income of $27.9 million, or $1.55 basic and $1.46 diluted per share, compared to $12.4 million, or $0.71 basic and $0.70 diluted per share, for the same period of 2024.

For the nine months ended September 30, 2025, total revenues were $224.5 million, consisting of $166.6 million in net product sales and $57.9 million in contract revenues from collaborations. Net product sales grew 69% compared to $98.4 million in the same period of 2024. TAVALISSE net product sales were $113.3 million, growth of 54% compared to $73.8 million in the same period of 2024. GAVRETO net product sales were $31.9 million, growth of 252% compared to $9.0 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $21.4 million, growth of 38% compared to $15.6 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $9.9 million of revenue from Grifols related to delivery of drug supplies and earned royalties, $6.9 million of revenue from Kissei related to a milestone payment and delivery of drug supplies and $0.8 million of revenue from Medison related to delivery of drug supplies and earned royalties.

Total costs and expenses were $122.2 million compared to $114.1 million for the same period of 2024. The increase in costs and expenses was mainly due to higher cost of product sales, increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289, and higher personnel-related costs.

Rigel reported net income of $99.0 million, or $5.52 basic and $5.38 diluted per share, compared to $3.1 million, or $0.18 basic and diluted per share, for the same period of 2024.

Cash, cash equivalents and short-term investments as of September 30, 2025 was $137.1 million, compared to $77.3 million as of December 31, 2024.

2025 Outlook
Rigel is updating its 2025 total revenue guidance to approximately $285 to $290 million, an increase from the previous range of approximately $270 to $280 million, which includes:

Net product sales of approximately $225 to $230 million, an increase from the previous range of approximately $210 to $220 million.
Contract revenues from collaborations of approximately $60 million.
The company anticipates it will report positive net income for the full year 2025, while funding existing and new clinical development programs.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

(Press release, Rigel, NOV 4, 2025, View Source [SID1234659373])

On November 4, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported that Mark A. Goldsmith, M.D., Ph.D., the company’s chief executive officer and chairman, will participate in two upcoming investor conferences.

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Details of the company’s participation are as follows:

Guggenheim 2nd Annual Healthcare Innovation Conference
Fireside Chat: Tuesday, November 11 at 9:00 a.m. ET
Jefferies Global Healthcare Conference
Fireside Chat: Tuesday, November 18 at 9:30 a.m. GMT
To listen to a live webcast of any of these events, or access archived webcasts, please visit: View Source Following the live webcasts, replays will be available on the company’s website for at least 14 days.

(Press release, Revolution Medicines, NOV 4, 2025, View Source [SID1234659372])

On November 4, 2025 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients living with inflammatory and immunological diseases, reported that members of the RAPT management team will participate in the following investor conferences in November:

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Guggenheim’s 2nd Annual Healthcare Innovation Conference – Fireside chat on Tuesday, November 11, 2025 at 3:30 p.m. ET

Stifel 2025 Healthcare Conference – Fireside chat on Wednesday, November 12, 2025 at 2:40 p.m. ET

TD Cowen Virtual Immunology & Inflammation Summit – Fireside chat on Thursday, November 13, 2025 at 2:30 p.m. ET
To access the live webcasts or subsequent archived recordings of the fireside chats, please visit the RAPT Therapeutics website at https://investors.rapt.com/events-and-presentations.

(Press release, RAPT Therapeutics, NOV 4, 2025, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-participate-multiple-upcoming-investor-0 [SID1234659371])

On November 4, 2025 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported results for the third quarter of 2025 and reaffirmed its outlook for solid profitable growth while raising its profitability targets.

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QIAGEN reaffirmed its FY 2025 outlook for net sales growth of about 4-5% CER (about 5-6% CER core sales excluding divestments) and raised its adjusted diluted EPS target to about $2.38 CER (previously about $2.35 CER). QIAGEN also expects an adjusted operating income margin of about 29.5% (about 30% CER) in 2025 while absorbing headwinds from currency movements and tariffs.

QIAGEN also announced two strategic initiatives to strengthen its Sample technologies portfolio and enhance shareholder value. The acquisition of Parse Biosciences provides entry into the rapidly growing single-cell market. QIAGEN also announced plans to complete a $500 million synthetic share repurchase in early January 2026 to further increase shareholder returns, bringing total returns to more than $1 billion since 2024 and well ahead of its 2028 goal.

"QIAGEN continues to deliver in a challenging environment, with another quarter of results above our outlook and among the fastest growth rates in the industry," said Thierry Bernard, CEO of QIAGEN. "We are pleased with the sustained growth from QIAstat-Dx and QuantiFERON, along with solid momentum in Sample technologies as we prepare to launch three new instruments. The acquisition of Parse Biosciences is an excellent strategic fit, strengthening our leadership in Sample technologies by providing access to the high-growth single-cell market and the ability to offer solutions to drive large-scale AI-driven biology. It reflects our commitment to invest in innovation offering accretive returns and expand our addressable markets. As we update our targets for 2025, we are moving ahead on our ambitions to deliver solid profitable growth," he said.

"Our strong profitability and cash generation are allowing QIAGEN to step up shareholder returns," said Roland Sackers, CFO of QIAGEN. "With the execution of the $500 million repurchase in January 2026 that shareholders approved at our last Annual General Meeting, we are returning more than $1 billion to shareholders well ahead of our 2028 goal. We remain focused on generating the highest returns and are reviewing how to increase this target while also strengthening our portfolio through organic investments and targeted acquisitions such as Parse. We anticipate that our ongoing capital allocation decisions will bring our leverage ratio toward the industry average of about 2x in 2026."

Please find the full press release incl. tables as a PDF for download at the top of this page.

Investor presentation and conference call

A conference call is scheduled for Wednesday, November 5, 2025, at 15:30 Frankfurt Time / 14:30 London Time / 9:30 New York Time. A live audio webcast will be available in the Investor Relations section of the QIAGEN website (www.qiagen.com), with a recording accessible after the event. The accompanying presentation will be published in advance under "Events and Presentations" in the same section.

(Press release, Qiagen, NOV 4, 2025, View Source;EPS-Target-Announces-Parse-Acquisition-and-500-Million-Share-Repurchase/default.aspx [SID1234659370])