On May 5, 2026 Citius Oncology, Inc. ("Citius Oncology" or the "Company") (Nasdaq: CTOR), a specialty biopharmaceutical company focused on the development and commercialization of novel targeted oncology therapies, reported that it has entered into a senior secured term loan credit facility (the "Credit Facility") with Avenue Venture Opportunities Fund II, L.P., a fund of Avenue Capital Group ("Avenue"), providing for up to $25 million in capital to support the ongoing commercialization of LYMPHIR (denileukin diftitox-cxdl), approved by the Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory Stage I-III cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy, and has also entered into a definitive agreement for the immediate exercise of certain outstanding warrants, with expected gross proceeds to the Company of approximately $11.5 million. The combined financings are expected to provide enhanced financial flexibility to support commercial execution, working capital, and general corporate purposes.

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"This Credit Facility strengthens our ability to continue to execute on the LYMPHIR launch, aligning capital access with commercial performance, and underscoring the confidence that a global investment firm like Avenue Capital has in our commercial trajectory and the long-term potential of LYMPHIR. In parallel, the warrant exercise financing provides additional near-term capital to further support our commercial efforts Collectively, this provides the company with meaningful financial flexibility as we continue to scale our commercial infrastructure, drive adoption of LYMPHIR among treating physicians, and expand patient access to this important therapy for relapsed or refractory cutaneous T-cell lymphoma. We are pleased to have Avenue as a capital partner as we advance our mission to improve outcomes for patients with limited treatment options," said Leonard Mazur, Chairman and Chief Executive Officer of Citius Oncology and Citius Pharmaceuticals.

"Citius Oncology has a differentiated, FDA-approved therapy with a targeted commercial opportunity. We are excited to support the Citius team as they execute on their commercialization strategy and work to realize the full value of their first FDA-approved asset," said Chad Norman, Senior Portfolio Manager of Avenue Capital Group.

H.C. Wainwright & Co. is acting as the exclusive origination, structuring and placement agent to Citius Oncology for the financings.

Financings Summary

The Credit Facility has a term of 3.5 years and includes an initial tranche of $10 million, to be fully funded at closing, plus two additional tranches of up to an aggregate of $15 million, subject to achievement of predefined revenue milestones and liquidity conditions. The Company has agreed to issue to Avenue warrants to purchase up to 11,111,111 shares of the Company’s common stock, at an exercise price of $0.90 per share, exercisable for a period of five years following the effective date of stockholder approval of the issuance of the shares issuable upon exercise of the warrants. The Company will also issue to Avenue warrants to purchase shares of common stock equal to 10% of the amount funded in each future tranche, divided by the exercise price of $0.90 per share. Additionally, Avenue will have the right, at any time while any loan is outstanding, to convert up to $4.0 million of the outstanding principal under the credit facility into shares of the Company’s common stock at a price per share equal to 120% of the exercise price of the warrant, subject to certain terms and conditions, including beneficial ownership limitations.

Concurrently, the Company entered into definitive agreements with a single healthcare-focused institutional investor for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 12,777,778 shares of common stock, originally issued in July 2025, September 2025, and December 2025, at a reduced exercise price of $0.90 per share, with expected gross proceeds to the Company of approximately $11.5 million prior to deduction of placement agent fees and other offering expenses. In consideration for the immediate exercise of these warrants for cash, the Company will issue new unregistered warrants to purchase up to 25,555,556 shares of common stock. The new warrants will have an exercise price of $0.90 per share, will be exercisable beginning on the effective date of stockholder approval of the issuance of the shares issuable upon exercise of the new warrants and will expire five years after the later of (i) the date of stockholder approval and (ii) the effective date of the Resale Registration Statement (as defined below). The new warrant offering is expected to close on or about May 6, 2026, subject to satisfaction of customary closing conditions.

The Company intends to use the net proceeds from the financings primarily to fund ongoing LYMPHIR commercialization efforts such as sales force expansion, market access initiatives, medical affairs activities, and manufacturing supply chain support, with the remainder to be used for working capital and general corporate purposes.

Warrant Offering Disclosure

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act") and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants (the "Resale Registration Statement").

The Company also has agreed to amend certain existing warrants to purchase up to an aggregate of 15,697,024 shares of the Company’s common stock that were previously issued to the investor in December 2025, with an exercise price of $1.09 per share, effective upon the closing of the offering, such that the amended warrants will have a reduced exercise price of $0.90 per share, will be exercisable beginning on the effective date of stockholder approval of the issuance of the shares upon exercise of the warrants and will expire five years after the date of stockholder approval.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

About LYMPHIR (denileukin diftitox‑cxdl)

LYMPHIR is a targeted immune therapy for relapsed or refractory cutaneous T-cell lymphoma (CTCL) indicated for use in Stage I-III disease after at least one prior systemic therapy. It is a recombinant fusion protein that combines the IL-2 receptor binding domain with diphtheria toxin (DT) fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. After uptake into the cell, the DT fragment is cleaved and the free DT fragments inhibit protein synthesis, resulting in cell death. Denileukin diftitox-cxdl demonstrated the ability to deplete immunosuppressive regulatory T lymphocytes (Tregs) and antitumor activity through a direct cytocidal action on IL-2R-expressing tumors.

In 2021, denileukin diftitox received regulatory approval in Japan for the treatment of relapsed or refractory CTCL and peripheral T-cell lymphoma (PTCL). Subsequently, in 2021, Citius acquired an exclusive license with rights to develop and commercialize denileukin diftitox in all markets except for India, Japan and certain parts of Asia. LYMPHIR (denileukin diftitox-cxdl) was approved by the FDA and subsequently launched in the U.S. in December 2025.

(Press release, Citius Oncology, MAY 5, 2026, View Source [SID1234665145])

On May 5, 2026 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported the successful completion of the acquisition of Terns Pharmaceuticals, Inc. ("Terns") (Nasdaq: TERN).

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"The Terns acquisition reflects Merck’s continued focus on science‑driven, value‑enhancing business development aimed at bringing meaningful innovation to patients," said Robert M. Davis, chairman and chief executive officer, Merck. "We believe TERN‑701 has the potential to become a differentiated treatment option for certain patients with chronic myeloid leukemia, and we look forward to working with the Terns team to advance its clinical development."

TERN-701 was recently granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adults with Philadelphia chromosome-positive chronic myeloid leukemia (CML) in the chronic phase without the T315I mutation previously treated with two or more tyrosine kinase inhibitors (TKIs). The BTD designation for TERN-701 is based on data from the ongoing Phase 1/2 CARDINAL trial (NCT06163430).

Transaction details

Merck completed the cash tender offer, through a subsidiary, for all the outstanding shares of common stock of Terns at a purchase price of $53.00 per share, without interest and subject to any applicable tax withholding. As of the tender offer expiration at one minute after 11:59 p.m., Eastern Time, on May 4, 2026, 100,091,794 shares of Terns common stock were validly tendered and not validly withdrawn, representing approximately 86.36% of the total number of Terns’ issued and outstanding shares of common stock as of such date and time. All such shares have been accepted for payment in accordance with the terms of the tender offer, and Merck, on behalf of its subsidiary, will promptly pay for such shares.

Following the completion of the tender offer, Merck completed the acquisition of Terns through a merger of Merck’s wholly-owned subsidiary with and into Terns, with Terns being the surviving corporation, in which all shares of Terns common stock issued and outstanding at the effective time of the merger were converted into the right to receive cash equal to the $53.00 offer price per share, without interest and subject to any applicable tax withholding. At the completion of the merger, Terns became a wholly-owned subsidiary of Merck and Terns’ common stock will no longer be listed or traded on the Nasdaq Global Select Market.

The transaction is expected to be accounted for as an asset acquisition, resulting in a charge to research and development expense of approximately $5.8 billion, or approximately $2.35 per share, included in both second quarter and full year 2026 GAAP and non-GAAP results. Additionally, GAAP and non-GAAP EPS are expected to be negatively impacted by approximately $0.12 per share in 2026, representing costs associated with advancing TERN-701 and costs of financing.

About TERN-701

TERN-701 is a novel investigational oral allosteric BCR::ABL1 tyrosine kinase inhibitor (TKI) designed to bind to the ABL myristoyl pocket, with a potentially best-in-disease profile that could improve upon existing treatments for certain patients with chronic myeloid leukemia (CML).

About chronic myeloid leukemia

Chronic myeloid leukemia (CML) is a slow growing type of blood cancer that leads to an overproduction of white blood cells that accumulate in the blood and bone marrow, disrupting the production of healthy blood cells. CML is commonly associated with the Philadelphia chromosome, a translocation between chromosomes 9 and 22 that results in constitutive activation of the BCR::ABL1 fusion protein, which fuels cancer growth.

(Press release, Merck & Co, MAY 5, 2026, View Source [SID1234665144])

On May 5, 2026 Theriva Biologics, Inc. (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the first quarter ended March 31, 2026, and provided a corporate update.

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"The first quarter of 2026 was marked by encouraging regulatory progress," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We were pleased to receive the minutes from our end-of-Phase 2 meeting with the FDA and align on the major elements of our proposed pivotal Phase 3 trial to evaluate VCN-01 with gemcitabine/nab-paclitaxel standard-of-care (SoC) chemotherapy in patients with metastatic PDAC. The FDA feedback was consistent with the positive scientific advice previously received from the EMA, with both agencies agreeing to repeated dosing ‘macrocycles’ of VCN-01 and SoC chemotherapy. Additional data analyses presented in a poster at the AACR (Free AACR Whitepaper) Annual Meeting in April 2026 concluded that the additional data may reflect a potential immune-mediated mechanism of action for VCN-01 in metastatic PDAC. We believe that repeated macrocycle dosing of VCN-01 and chemotherapy may enhance this immune action, providing increased and more durable tumor responses and longer survival. As we finalize the pivotal Phase 3 study protocol, we plan to generate feasibility data for the intended Phase 3 macrocycle dosing regimen, by conducting a small study in metastatic PDAC patients administering more frequent VCN-01 doses for a longer period. This dosing feasibility study is expected to commence at a single site in Spain in the second half of this year. In parallel with our PDAC program, discussions are ongoing with clinicians and key opinion leaders to design a Phase 2/3 clinical trial protocol for the VCN-01 plus topotecan combination in retinoblastoma patients. We believe that intravitreal coadministration of VCN-01 with topotecan may provide a new treatment option for children with refractory retinoblastoma and vitreous seeds, which remains an unmet medical need in patients with this rare disease. In the first quarter of 2026, we made VCN-01 available to investigators for compassionate use in treating patients with retinoblastoma. We expect that outcomes from these compassionate use patients will provide valuable information on the feasibility and tolerability of this combination for use in a potential Phase 2/3 clinical trial. If a protocol is ultimately submitted to, and agreed by, the FDA, we expect the first patient to be enrolled in December 2026, with rolling Biologic Licensing Application (BLA) submissions expected to be made in 2029 (if successful), targeting potential approval of the BLA prior to September 30, 2029."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic PDAC:

As recently announced, Theriva received minutes from Type B End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding the design of a Phase 3 clinical study of lead clinical candidate VCN-01 in combination with standard-of-care chemotherapy for the treatment of metastatic PDAC. The FDA provided general agreement with Theriva’s proposed design for a Phase 3 clinical trial, which closely tracks the design of the successful VIRAGE Phase 2 trial. As announced in 2025, the VIRAGE trial met its primary endpoints, with metastatic PDAC patients receiving VCN-01 with SoC chemotherapy having improved overall survival (OS), progression-free survival (PFS) and duration of response (DoR) compared to SoC chemotherapy alone. Greater improvements in OS and PFS were observed in patients who received two doses of VCN-01, leading Theriva to plan the Phase 3 trial to include repeat dosing and an adaptive design aimed to optimize the trial’s timelines and outcomes.
Tumor response, biomarker, and subgroup analyses from the VIRAGE Phase 2b clinical trial were recently presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2026 annual meeting. The poster concluded that the additional data may reflect an immune-mediated mechanism of action for VCN-01, with later and more durable responses and improved overall survival and progression-free survival in patients treated with VCN-01 plus SoC chemotherapy, compared to SoC chemotherapy alone. Improved overall survival was observed in VCN-01-treated patients across multiple subgroups, including patients with liver metastases.
Retinoblastoma:

Made VCN-01 available to investigators for compassionate use in treating patients with retinoblastoma. Two patients have been treated with intravitreal VCN-01 in combination with intravitreal topotecan and are being followed by the treating physicians.
Continued discussions with clinicians and key opinion leaders to design a Phase 2/3 clinical trial protocol for the VCN-01 plus topotecan combination in retinoblastoma patients.
First Quarter Ended March 31, 2026 Financial Results

General and administrative expenses

General and administrative expenses increased to $2.1 million for the three months ended March 31, 2026, from $1.4 million for the three months ended March 31, 2025. This increase of 43% is primarily comprised of an increase in legal fees, investor relations costs, registration fees, and salary costs. The charge related to stock-based compensation expense was $111,000 for the three months ended March 31, 2026, compared to $54,000 for the three months ended March 31, 2025.

Research and Development Expenses

Research and development expenses decreased to $355,000 for the three months ended March 31, 2026, from approximately $3.0 million for the three months ended March 31, 2025. This decrease of 88% is primarily the result of lower clinical trial expenses related to the completion of our VIRAGE Phase 2b clinical trial of VCN-01 in PDAC, the recognition of the Spanish research and development rebate, lower indirect costs related to compensation and lower clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by higher patent expenses related to SYN-020. We anticipate research and development expense to decrease in the near future until we commence additional clinical trials as we focus on regulatory interactions regarding a proposed pivotal clinical trial of VCN-01 in retinoblastoma, continue with VCN-01 manufacturing scale-up activities, commence a proposed Phase 2a study in metastatic PDAC patients evaluating VCN-01 dosing frequency and continue limited preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program. The charge related to stock-based compensation expense was $24,000 for the three months ended March 31, 2026, compared to $46,000 for the three months ended March 31, 2025.

Other Income/Expense

Other income was $83,000 for the three months ended March 31, 2026 compared to other income of $93,000 for the three months ended March 31, 2025. Other income for the three months ended March 31, 2026 is comprised of interest income of $82,000 and an exchange gain of $1,000. Other income for the three months ended March 31, 2025 is comprised of interest income of $96,000 and an exchange loss of $3,000.

Cash and cash equivalents

Cash and cash equivalents totaled $14.4 million as of March 31, 2026, an increase of $1.4 million from December 31, 2025. During the year ended December 31, 2025 and the quarter ended March 31, 2026, the primary use of cash was for working capital requirements and operating activities, which resulted in a net loss of $23.7 million and $2.0 million for the year ended December 31, 2025 and the quarter ended March 31, 2026, respectively.

(Press release, Theriva Biologics, MAY 5, 2026, View Source [SID1234665143])

On May 5, 2026 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing stenoparib (2X-121)—a differentiated, dual PARP and WNT pathway inhibitor, reported that its active pharmaceutical ingredient (API) manufacturing campaign for stenoparib is progressing in line with the planned timeline for completion no later than the third quarter of 2026 at its world-class contract development and manufacturing organization (CDMO).

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This milestone represents a key operational advance as the Company works to secure robust drug supply while preparing for potential pivotal-stage clinical development. The decision to move forward with the campaign reflects the continuously growing confidence in stenoparib’s therapeutic potential, based on previously reported data showing extended overall survival in advanced, platinum-resistant ovarian cancer patients.

"With the Phase 3 manufacturing campaign of stenoparib approaching completion, we are taking an important step to ensuring timely advancement into potential pivotal-stage clinical trials," said Thomas Jensen, Chief Executive Officer of Allarity Therapeutics. "This campaign reflects our confidence in the long-term potential of the program, and is particularly important as we work to leverage the FDA Fast Track designation to accelerate the development and potential approval of stenoparib."

The CDMO site, located in Europe, operates in full compliance with GMP (Good Manufacturing Practice) standards set by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Drug manufacturing for late-stage clinical development requires heightened manufacturing standards that go above and beyond the standards necessary for phase 1 and phase 2 clinical development. Triggering this campaign now ensures that the higher standard API is ready when the company is ready to advance a pivotal trial for FDA approval.

From a financial standpoint, the company has completed all payments for the manufacturing and no additional cash outlays for manufacturing are anticipated.

The manufacturing campaign is expected to be completed well in advance of the anticipated generation of critical data from Allarity’s ongoing Phase 2 trial in advanced ovarian cancer. The ongoing phase 2 trial continues to enroll patients under the new protocol, generating enthusiastic investigator engagement.

About Stenoparib/2X-121
Stenoparib is an orally available, small-molecule dual-targeted inhibitor of PARP1/2 and tankyrase 1/2. At present, tankyrases are attracting significant attention as emerging therapeutic targets for cancer, principally due to their role in regulating the WNT signaling pathway. Aberrant WNT/β-catenin signaling has been implicated in the development and progression of numerous cancers. By inhibiting PARP and blocking WNT pathway activation, stenoparib’s unique therapeutic action shows potential as a promising therapeutic for many cancer types, including ovarian cancer, Small Cell Lung Cancer and colorectal cancer. Allarity has secured exclusive global rights for the development and commercialization of stenoparib, which was originally developed by Eisai Co. Ltd. and was formerly known under the names E7449 and 2X-121. Allarity has two ongoing Phase 2 trial protocols for stenoparib in Ovarian Cancer patients. In the first, patients who had had 2+ lines of therapy were enrolled on stenoparib and given drug twice daily. This protocol has been closed to further enrollment but continues for the enrolled patients who are still receiving benefit from stenoparib administration. The updated data from this study were presented at the AACR (Free AACR Whitepaper) special conference on advances in Ovarian Cancer in September 2025. Note that, as these data are from an ongoing trial, analyses may change as the study fully matures. An amended protocol designed expressly to capitalize on the emerging clinical experience with stenoparib in platinum resistant patients began enrolling patients in the summer of 2025. This amended protocol enrolls only platinum resistant or platinum-ineligible patients and is designed to accelerate the clinical development of stenoparib toward FDA approval. In parallel, a separate Phase 2 trial evaluating stenoparib in combination with temozolomide for relapsed small cell lung cancer (SCLC) began enrolling patients in early 2026 and is currently enrolling patients across multiple U.S. Veterans Administration (VA) sites.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, may have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be enhanced. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines, combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has shown an ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients across dozens of clinical studies (both retrospective and prospective). The DRP platform, which may be useful in all cancer types and is patented for dozens of anti-cancer drugs, has been extensively published in the peer-reviewed literature.

(Press release, Allarity Therapeutics, MAY 5, 2026, View Source [SID1234665142])

On May 5, 2026 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 March 31, 2026 and reaffirmed its financial guidance for 2026.

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"This is an important year for Ultragenyx with two BLAs under review and our Angelman Phase 3 readout coming soon. We have the opportunity to meaningfully accelerate our consistent commercial revenue growth over the last few years as we prepare for two potential gene therapy approvals and launches in two urgent diseases without any approved therapies," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "The latest long-term GTX-102 Phase 1/2 data further support the potential of the program, as it heads toward Phase 3 results later this year."

First Quarter 2026 Revenue Highlights and 2026 Revenue Guidance

Total revenue in the first quarter of 2026 was $136 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches.
Crysvita revenue in the first quarter of 2026 was $93 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Brazil. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million.
Dojolvi revenue in the first quarter 2026 was $18 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.
Evkeeza revenue in the first quarter 2026 was $18 million, driven by increased demand from new country launches and early access.
Mepsevii revenue in the first quarter 2026 was $7 million.
Recent Clinical Milestones and 2026 Catalysts

GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): As of a March 2026 Phase 1/2 data cut-off date, a total of 74 patients had been treated with GTX-102, with 66 patients continuing in the long-term extension (LTE) study. Phase 1/2 patients have been on continuous treatment for an average of more than three years, with some patients now in their fifth year, generally receiving the 14 mg quarterly maintenance dose. Patients have continued to show positive improvements across multiple domains and continued to gain ground developmentally. GTX-102 has maintained a consistent safety profile, sustained over multiple years of chronic treatment, while demonstrating no new cases of transient lower extremity weakness nor any other recurring drug-related serious adverse events. These updated efficacy and safety data are planned to be presented at a future scientific meeting.

The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the second half of 2026.

Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began enrollment in October 2025 and is expected to complete enrollment in the second half of 2026.
DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In February 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa, granted the BLA Priority Review, and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. The FDA also recently informed the company that an Advisory Committee meeting is not anticipated at this time.

UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA. The resubmitted BLA included substantial longer-term data that were presented in February at the WORLDSymposium 2026 and included up to eight years of follow-up. These data demonstrated further clinical improvement relative to the decline observed in natural history studies, and showed a durable treatment effect across clinical evaluations and multiple biomarkers, while maintaining an acceptable safety profile. In February 2025, the FDA granted the BLA Priority Review and, in April 2026, assigned a PDUFA action date of September 19, 2026.

DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: As announced in March 2026, at Week 36 in the randomized, double-blind placebo-controlled period of the Phase 3 study, DTX301 patients (n=18) demonstrated a statistically significant and clinically meaningful 18% (p=0.018) reduction in 24-hour plasma ammonia (AUC0-24) into the normal range compared to placebo (n=19). Eight of nine patients with abnormal ammonia AUC0-24 at baseline also reached normal ammonia levels rapidly, which were generally maintained during this treatment period. At Week 24, patient global impression scale (PGIC) for overall OTC symptoms (n=15) showed 71% of DTX301 patients were much improved (equivalent to +3), compared to 0% of placebo patients. DTX301 was well tolerated with an acceptable safety profile.

Per the protocol, the study is continuing to its second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, across both the treatment and placebo-crossover groups following treatment with DTX301 through 64 weeks of follow-up. Data are expected in the first half of 2027.
UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in 2026.

UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The FDA cleared the Investigational New Drug (IND) application for UX016 and an externally funded Phase 1/2 study is expected to begin in the second half of 2026.
Summary of First Quarter 2026 Financial Results

Selected Financial Data (dollars in millions, except per share amounts), (unaudited)
Three Months Ended March 31,
2026 2025
Total revenues $ 136 $ 139
Operating expenses:
Cost of sales 30 29
Research and development 187 166
Selling, general and administrative 88 87
Total operating expenses 305 282
Net loss $ (185 ) $ (151 )
Net loss per share, basic and diluted $ (1.84 ) $ (1.57 )

Operating Expenses

Total operating expenses for the first quarter 2026 were $305 million, including $30 million of non-cash stock-based compensation and $30 million of expense related to the restructuring announced last quarter. The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses: compared to 2025, combined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits, and combined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%.

Net Loss

Net loss for the first quarter 2026 was $185 million, or $1.84 per share basic and diluted, compared with a net loss for the first quarter 2025 of $151 million, or $1.57 per share basic and diluted.

Cash Balance and Net Cash Used in Operations

Cash, cash equivalents, and marketable securities were $534 million as of March 31, 2026. For the three months ended March 31, 2026, net cash used in operations was $197 million and includes the payment of annual bonuses and $38 million of payments related to UX143 manufacturing activities.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, May 5, 2026, at 2 p.m. PT/5 p.m. ET to discuss the first quarter 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, MAY 5, 2026, View Source [SID1234665141])