On May 13, 2026 Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd., reported that the U.S. Food and Drug Administration (FDA) has approved INQOVI (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older or who are ineligible for intensive induction chemotherapy. INQOVI in combination with venetoclax is the first and only all-oral combination treatment regimen approved for this patient population, offering an alternative to parenteral hypomethylating agent–based regimens that require frequent clinic visits.

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The approval was supported by results from the Phase 2 ASCERTAIN-V study of INQOVI plus venetoclax in adult patients with newly diagnosed AML who were ineligible for intensive induction chemotherapy.1

Efficacy was established based on complete remission (CR) and the duration of CR (DoCR). Duration of remission was defined as the time from first CR until disease relapse or death from any cause, whichever occurred first. In combination with venetoclax, 42 patients achieved a CR (41.6%, 95% CI: 31.9, 51.8) with a median time to CR of two months (range: 0.4 to 15.3 months). The median duration of CR was not reached (range: 0.5 to 16.3 months).

The Prescribing Information contains Warnings and Precautions for myelosuppression and embryo-fetal toxicity. Please see the Important Safety Information and Prescribing Information for more details.

INQOVI is an orally administered hypomethylating regimen previously approved in the U.S. and Canada for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).2

"This FDA approval represents a significant milestone for patients with newly diagnosed acute myeloid leukemia who are not candidates for intensive induction chemotherapy," said Peter Melnyk, President and Chief Executive Officer of Taiho Oncology. "With the approval of an all-oral regimen, INQOVI in combination with venetoclax brings a new treatment option to this patient population and underscores our commitment to advancing innovative, patient-focused therapies in hematologic malignancies."

In 2026, an estimated 22,720 people in the U.S. will be diagnosed with AML, a cancer of the blood and bone marrow.3 More than half of those patients are likely to be ineligible for intensive induction chemotherapy due to advanced age or health concerns.4

"As a leader in the development of oral anti-cancer regimens, we are proud that INQOVI in combination with venetoclax will now be available for newly diagnosed patients with acute myeloid leukemia in the U.S.," said Harold Keer, MD, PhD, Chief Medical Officer of Taiho Oncology. "This approval marks an important step forward in expanding how treatment can be delivered for this patient population, offering an all‑oral option that can potentially reduce the overall treatment burden associated with receiving treatment in hospitals or infusion centers. We believe this approach has the potential to make a meaningful impact for patients and caregivers."

*ASCERTAIN-V Study: AStx727-07: decitabine + CEdazuRidine TreAtment IN AML, adding Venetoclax

About INQOVI

INQOVI is an orally administered, fixed-dose combination of the DNA hypomethylating agent decitabine together with cedazuridine,5 an inhibitor of cytidine deaminase.6 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for the oral delivery of decitabine to achieve comparable systemic exposure to that of IV decitabine.

Indications and Important Safety Information

INDICATIONS

INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

INQOVI is indicated in combination with venetoclax for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Myelosuppression

INQOVI as Monotherapy for MDS or CMML

In patients with MDS or CMML, INQOVI can cause severe myelosuppression, including fatal adverse reactions. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia are the most frequent cause of INQOVI dose reduction or interruption, occurring in 36% of patients. Permanent discontinuation due to myelosuppression (febrile neutropenia) occurred in 1% of patients. Myelosuppression and worsening neutropenia may occur more frequently in the first or second treatment cycles and may not necessarily indicate progression of underlying MDS.

Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.

Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti‑infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

INQOVI in Combination with Venetoclax for AML

In patients with AML, INQOVI can cause severe myelosuppression, including fatal adverse reactions, when given in combination with venetoclax. Based on laboratory values in Study ASTX727-07 Phase 2 new or worsening thrombocytopenia occurred in 70% of patients, with Grade 3 or 4 occurring in 69%. Neutropenia occurred in 48% of patients, with Grade 3 or 4 occurring in 48%. Anemia occurred in 54% of patients, with Grade 3 or 4 occurring in 50%. Febrile neutropenia occurred in 52% of patients, with Grade 3 or 4 occurring in 52%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia were a frequent cause of INQOVI and/or venetoclax dose reduction or interruption. Dose reductions of INQOVI due to neutropenia and thrombocytopenia occurred in 4% and 1% of patients, respectively. Dose interruptions of INQOVI due to neutropenia, febrile neutropenia, thrombocytopenia, and anemia occurred in 40%, 11%, 8%, and 2% of patients, respectively.

Fatal and serious infectious complications can occur during treatment with INQOVI and venetoclax. Pneumonia occurred in 25% of patients, with Grade 3 or 4 occurring in 20%. Sepsis occurred in 28% of patients, with Grade 3 or 4 occurring in 18%. Fatal pneumonia occurred in 2% of patients and fatal sepsis in 8%.

Obtain complete blood cell counts prior to initiation of INQOVI with venetoclax, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

Embryo-Fetal Toxicity

Advise patients of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with INQOVI and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with INQOVI and for 3 months after the last dose.

ADVERSE REACTIONS

INQOVI as Monotherapy for MDS or CMML

Serious adverse reactions occurred in 68% of patients who received INQOVI. Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions occurred in 6% of patients. These included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.

The most common adverse reactions (≥ 20%) were fatigue (55%), constipation (44%), hemorrhage (43%), myalgia (42%), mucositis (41%), arthralgia (40%), nausea (40%), dyspnea (38%), diarrhea (37%), rash (33%), dizziness (33%), febrile neutropenia (33%), edema (30%), headache (30%), cough (28%), decreased appetite (24%), upper respiratory tract infection (23%), pneumonia (21%), and transaminase increased (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased (81%), platelet count decreased (76%), neutrophil count decreased (71%), and hemoglobin decreased (55%).

INQOVI in Combination with Venetoclax for AML

Serious adverse reactions occurred in 82% of patients who received INQOVI+VEN. Serious adverse reactions in > 5% of patients included febrile neutropenia (31%), sepsis (22%), pneumonia (15%), infection (bacterial/viral) (10%), hemorrhage (9%), and dyspnea (6%). Fatal adverse reactions occurred in 8% of patients who received INQOVI+VEN. These included sepsis (5%), dyspnea (2%), myocardial infarction (1%), hemolytic anemia (1%), and tumor lysis syndrome (1%).

The most common adverse reactions (≥ 20%) were neutropenia (60%), febrile neutropenia (52%), thrombocytopenia (52%), hemorrhage (42%), anemia (41%), infection (bacterial/viral) (40%), diarrhea (38%), fatigue (36%), mucositis (36%), constipation (36%), arthralgia (35%), decreased appetite (31%), edema (31%), nausea (31%), dyspnea (30%), white blood cell count decreased (28%), sepsis (28%), pneumonia (25%), rash (25%), transaminitis (24%), myalgia (23%), arrhythmia (21%), and abdominal pain (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 20%) were decreased leukocytes (91%), decreased lymphocytes (81%), decreased platelets (69%), decreased hemoglobin (50%), and decreased neutrophils (48%).

USE IN SPECIFIC POPULATIONS

Lactation

Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for 2 weeks after the last dose.

Renal Impairment

No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).

(Press release, Taiho, MAY 13, 2026, View Source [SID1234665650])

On May 13, 2026 Takeda reported Financial Report For the Quarter Ended March 31, 2026.

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(Presentation, Takeda, MAY 13, 2026, View Source [SID1234666404])

On May 13, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported that it is approaching the first unblinding of data from its pivotal Phase 2B/3 "MIRACLE" trial evaluating Annamycin in combination with cytarabine for the treatment of subjects that have been relapsed or refractory to their primary line of treatment for acute myeloid leukemia (R/R AML). The trial incorporates two arms of Annamycin at different doses plus cytarabine compared to the control arm of cytarabine plus a placebo. The Company continues to expect this unblinding to occur prior to June 30, 2026, as previously communicated.

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The Company also reported that preliminary blinded efficacy data for the 45 subjects continue to approximate previously disclosed results, including a composite complete remission rate (CRc) exceeding 40% and a complete remission (CR) rate of approximately 30%. These results compare favorably to historical CR rates from two major trials of approximately 17-18% observed with cytarabine alone in similar patient populations. Cytarabine is a currently approved standard of care for second line treatment of AML. Based on preliminary data, the median age for subjects enrolled is in the mid-60’s, with over 30% entering the trial after becoming relapsed from or refractory (R/R) to a prior venetoclax regimen as first line therapy, which is considered a particularly challenging patient group. As of May 1st, a total of 56 subjects have been recruited in the MIRACLE trial to date keeping the Company on track to recruit the 90th subject in Part A in Q3 2026.

"The data coming from MIRACLE is encouraging" said Walter Klemp, Chairman and CEO of Moleculin. "For decades, cytarabine monotherapy regimens, although one of the current standards of care, have set a relatively low bar in relapsed or refractory AML, with historical complete remission rates hovering around 17-18% in previously referenced clinical trials. With our trial comparing two arms of Annamycin in combination with cytarabine compared to cytarabine with a placebo, we are looking forward to the opportunity of showing Annamycin is additive to cytarabine alone in a head to head trial. Against this backdrop, the data emerging from MIRACLE highlight what could be a substantial advancement, suggesting that we may finally be moving beyond the limitations that have defined standard-of-care outcomes."

MIRACLE Trial Progress and Next Steps

The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. Part A of the MIRACLE trial is designed to evaluate the effectiveness of Annamycin in two dosing arms (190 mg/m² and 230 mg/m²) in combination with cytarabine (also referred to as Ara-C) as compared to a control arm of cytarabine plus placebo. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data of the three arms at 45 subjects in Part A, in addition to the conclusion of Part A (at 90 total subjects). The first early unblinding should yield approximately 30 subjects treated with Annamycin (190 mg/m2 and 230 mg/m2) in combination with cytarabine and 15 subjects treated with just cytarabine plus placebo. The MIRACLE trial is being offered only to AML patients who have had a single prior induction therapy (2nd line patients or 2L) including subjects that were treated with a Venetoclax regimen.

The unblinding of the first 45 subjects is on track to occur in late Q2 2026, as the data are subject to final data entry and audit. The second group of 45 subjects in Part A are expected to be fully recruited in the third quarter of 2026 with unblinding expected late in the second half of 2026. Recruitment of the second group of 45 subjects in Part A will continue uninterrupted while the first 45 subjects with efficacy are being unblinded. Unblinding for the full 90 subjects in Part A may require more time than for the first 45 as it involves more data to support the transition from Part A to Part B.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU can be found on euclinicaltrials.eu and the reference identifier is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

(Press release, Moleculin, MAY 13, 2026, View Source [SID1234665635])

On May 13, 2026 BeOne Medicines Ltd. ("BeOne") (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to BEQALZI (bee-KAHL-zee; sonrotoclax), a foundational, next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. BEQALZI was designed to enhance BCL2 inhibition—with greater potency, selectivity, and a pharmacologic profile with potential to improve efficacy, tolerability, and convenience over others in the class.

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Michael Wang, M.D., Global Principal Investigator, the Puddin Clarke Endowed Professor, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, said:

"The data supporting the approval of sonrotoclax in the U.S. confirm its role as a foundational therapy for mantle cell lymphoma in the post-BTK inhibitor setting, and demonstrate that it can deliver robust disease control when treatment choices are limited and outcomes are poor. From a clinical perspective, this provides physicians with an important new option grounded in both efficacy and tolerability, fundamentally changing how we think about sequencing therapy in this disease."

Data supporting approval

The accelerated approval of BEQALZI is supported by efficacy and safety data from the Phase 1/2 study, BGB-11417-201 (NCT05471843), which was presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition. The study included an independent review of efficacy data and demonstrated:

Overall response rate (ORR): 52% (95% CI, 42-62)
Complete response (CR) rate: 16% (95% CI, 9.1-24.0)
Median time to response (TTR): 1.9 months
Median duration of response (DOR): 15.8 months (95% CI, 7.4 months-NE) at a median response follow-up of 11.9 months (has yet to reach full maturity)
Safety: treatment with sonrotoclax monotherapy was generally well tolerated
Continued approval for this indication is contingent upon confirmation of clinical benefit in the confirmatory CELESTIAL-RRMCL trial (NCT06742996), which is underway. The U.S. FDA granted Breakthrough Therapy Designation (BTD) for sonrotoclax in this indication, as well as Fast Track Designation and Orphan Drug Designation.

Amit Agarwal, M.D., Ph.D., Chief Medical Officer, Hematology, BeOne Medicines, said:

"BeOne is leading the advancement and enhancement of BCL2 inhibition to revolutionize how we treat patients living with B-cell malignancies. Today’s approval of BEQALZI represents critical progress for patients with mantle cell lymphoma and reinforces our strategy of building foundational medicines designed to raise the standard of care in B-cell malignancies."

A new BCL2 option for a challenging R/R MCL post–BTK inhibitor setting

MCL is a rare and often aggressive subtype of non-Hodgkin lymphoma. In the United States, approximately 3,300 new cases of MCL are diagnosed each year.1 While many patients respond to initial therapy, relapse is common, and outcomes after progression can be poor, particularly after prior treatment with a BTK inhibitor. The accelerated approval of BEQALZI introduces a new targeted mechanism to the MCL treatment landscape and reinforces the importance of expanding therapeutic choices for patients as the disease evolves.

Meghan Gutierrez, Chief Executive Officer, Lymphoma Research Foundation, said:

"For people living with relapsed or refractory mantle cell lymphoma, each progression can bring uncertainty and questions regarding remaining treatment options. The FDA approval of sonrotoclax represents significant progress for the U.S. mantle cell lymphoma community, offering renewed hope for patients and families who have exhausted other available therapies. Advances like this underscore why continued research and innovation in this disease remain so critical."

Additional regulatory and development updates

BEQALZI is also approved in China for the treatment of R/R MCL, as well as adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) who have previously received at least one systemic therapy, including a BTK inhibitor. Data from the Phase 1/2 study of sonrotoclax in R/R MCL is also under review by the European Medicines Agency and other regulatory agencies.

The U.S. FDA also granted sonrotoclax Fast Track Designation for Waldenström macroglobulinemia (WM), as well as Orphan Drug Designation for the treatment of adult patients with WM, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.

Additionally, sonrotoclax is currently being studied in combination with other therapeutics, including zanubrutinib, as a potential treatment for CLL, with updated data expected to be presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

About BEQALZI (sonrotoclax)

BEQALZI (sonrotoclax) is a foundational, next-generation and potentially best-in-class B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Preclinical and clinical studies in early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, including chronic lymphocytic leukemia (CLL), and is in development as a monotherapy and in combination with other therapeutics, including zanubrutinib. To date, more than 2,200 patients have been enrolled across the broad sonrotoclax global development program.

INDICATION

BEQALZI (sonrotoclax) is a BCL-2 inhibitor indicated for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor.

This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

BEQALZI is contraindicated with strong CYP3A inhibitors at initiation and during the ramp-up phase due to the potential for an increased risk of tumor lysis syndrome (TLS).

WARNINGS & PRECAUTIONS

Tumor Lysis Syndrome (TLS): BEQALZI can cause rapid tumor reduction and changes in blood chemistries consistent with TLS, which may be serious or life-threatening and require prompt management. TLS may occur as early as 4 hours after the first dose, with dose increases, or upon reinitiation following treatment interruption. Laboratory or clinical TLS occurred in 7% of patients who followed the recommended dose ramp-up. Assess all patients for TLS risk and initiate prophylaxis, including adequate hydration and antihyperuricemics. For patients at high risk of TLS, consider hospitalization with intravenous hydration and employ frequent monitoring. Monitor blood chemistries closely and manage abnormalities promptly. Interrupt BEQALZI for TLS; upon reinitiation, follow dose modification guidance in the Prescribing Information.
Serious Infections: BEQALZI can cause fatal or serious infections. Serious infections occurred in 14% of patients; Grade 3-4 occurred in 17% (fatal: 2.6%). The most common Grade 3 or greater infection was pneumonia (10%). Monitor for signs and symptoms of infection and treat appropriately. Consider prophylactic antimicrobials and immunoglobulins. Interrupt, reduce dose, or permanently discontinue BEQALZI based on severity.
Neutropenia: BEQALZI can cause serious or severe cytopenias, including neutropenia. Grade 3 or 4 decreases in neutrophils occurred in 18% of patients (Grade 4: 6%); febrile neutropenia occurred in 1.7% of all patients. Monitor complete blood counts throughout treatment. Interrupt treatment, reduce the dose, or permanently discontinue BEQALZI based on severity.
Embryo-Fetal Toxicity: BEQALZI can cause fetal harm when administered to pregnant women. Advise patients of the potential risk to a fetus. Verify pregnancy status prior to initiation. Advise females to use effective contraception and males with female partners of reproductive potential to use effective contraception during treatment and for 1 week after the last dose.
ADVERSE REACTIONS

The most common adverse reactions (≥15%) are pneumonia (16%) and fatigue (16%). The most common Grade 3-4 laboratory abnormalities (≥15%) are decreases in lymphocytes (29%) and neutrophils (18%).

DRUG INTERACTIONS

Strong or Moderate CYP3A Inhibitors: Concomitant use increases BEQALZI exposure. Avoid use of strong CYP3A inhibitors during BEQALZI initiation and ramp-up. Avoid use of moderate CYP3A inhibitors at the 1 mg and 2 mg doses; for all other doses, reduce the BEQALZI dose with concomitant use. See approved labeling for dose modifications.
Strong or Moderate CYP3A Inducers: Concomitant use decreases BEQALZI exposure. Avoid use.
SPECIAL POPULATIONS

Lactation: Advise women not to breastfeed during treatment with BEQALZI and for 1 week after the last dose.

To report SUSPECTED ADVERSE REACTIONS, contact BeOne Medicines at 1-877-828-5596 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full U.S. Prescribing Information.

(Press release, BeOne Medicines, MAY 13, 2026, View Source [SID1234665651])

On May 13, 2026 Omeros Corporation (Nasdaq: OMER) reported recent highlights and developments as well as financial results for the first quarter ended March 31, 2026, which include:

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First Quarter and Recent Highlights

● In January 2026, we launched YARTEMLEA in the U.S. market. During the quarter, gross product sales were $11.1 million and associated net sales, after deduction of wholesaler distribution fees and chargebacks, were $9.9 million.

● Net income for the first quarter of 2026 was $56.1 million, or $0.78 per share, compared to a net loss of $33.5 million, or $0.58 per share, for the first quarter of 2025.

● First quarter results include a $73.1 million non-cash gain associated with the mark-to-market adjustment on the embedded derivatives related to our 2029 unsecured convertible notes (the "2029 Notes"). Excluding the non-cash change in our embedded derivatives, non-GAAP adjusted net loss for the three months ended March 31, 2026 was $17.1 million, or $0.24 per share.

● At March 31, 2026, we had $135.3 million of cash and short-term investments. This balance includes the February 2026 repayment at maturity of the remaining $17.1 million aggregate principal amount of our 2026 unsecured convertible notes (the "2026 Notes"). Following that repayment, our only remaining debt outstanding is $70.8 million aggregate principal amount of our 2029 Notes, which mature in June 2029.

● In April, the U.S. Centers for Medicare & Medicaid Services ("CMS") assigned a permanent Healthcare Common Procedure Coding System J-code specific for YARTEMLEA. This simplifies billing and reimbursement across payors. The J-code becomes effective on July 1, 2026. Also in April, CMS, in its Inpatient Prospective Payment System proposed rule, recommended approval of the New Technology Add-On Payment ("NTAP") for YARTEMLEA. NTAP provides additional payments to hospitals for certain high-cost, innovative technologies, helping bridge the gap until standard payment systems incorporate them. The final rule is expected in August, with NTAP expected to be effective October 1, 2026.

"The launch of YARTEMLEA has changed the trajectory of Omeros, both operationally and financially," said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief Executive Officer. "We are seeing strong early adoption across transplant centers, expanding formulary access, favorable reimbursement support, and growing physician experience with the first and only approved treatment for TA-TMA. At the same time, our Novo Nordisk transaction has strengthened our balance sheet and accelerated advancement of our pipeline, including next-generation MASP-2 programs, OncotoX-AML, OMS527 for cocaine use disorder under NIDA funding, and our T-CAT platform targeting multidrug-resistant pathogens. The progress achieved this quarter further demonstrates the strength of our science and the value we are creating across Omeros."

Recent Developments

● YARTEMLEA and our other MASP-2 inhibitor programs

o A marketing authorization application ("MAA") for YARTEMLEA for the treatment of TA-TMA is currently under review by the European Medicines Agency ("EMA") with a decision expected in mid-2026. If approved, the MAA authorizes the product to be marketed in all EU member states and European Economic Area countries.

o We are assessing opportunities for YARTEMLEA across indications involving lectin pathway activation, including acute respiratory distress syndrome (ARDS), sickle cell disease, acute kidney injury, solid organ transplant-related TMA, and delayed graft function.

o In parallel, we are finalizing selection of an indication for a Phase 2 clinical program for OMS1029, our long-acting antibody targeting MASP-2. In our MASP-2 small-molecule inhibitor program, we have selected a drug development candidate and are advancing to IND-enabling studies.

● OMS527 for the treatment of addiction — cocaine use disorder program funded by the National Institute on Drug Abuse ("NIDA")

o We are developing, at NIDA’s request, our lead orally administered phosphodiesterase 7 ("PDE7") inhibitor for the treatment of cocaine use disorder. Preclinical studies, designed with NIDA toxicologists, were completed and showed no drug-interaction or safety issues, supporting the scheduled in-patient human study of OMS527 in cocaine users.

o Following FDA’s request for additional nonclinical information and a subsequent meeting with FDA to discuss that request, we are working with FDA to streamline the path to initiate the in-patient clinical trial, targeted for initiation by year-end 2026.

● Oncology platform — OncotoX-AML

o We continue to progress preclinical studies within our novel oncology program. The lead indication for development is acute myeloid leukemia ("AML"), an aggressive and highly fatal bone marrow and blood cancer. We have completed selection of a drug development candidate in the OncotoX-AML program, and IND-enabling studies are underway.

o OncotoX-AML shows broad application across AML regardless of genetic mutation, including TP53, NPM1, KMT2A, and FLT3, collectively found in approximately 90% of AML patients. In human tumor-bearing animal and in vitro human AML cell-line studies, our AML therapeutic candidate has demonstrated superior efficacy to current AML standard of care treatments.

o In February 2026, we announced the successful completion of our initial study in nonhuman primates evaluating the efficacy and safety of OncotoX-AML. Administration of only one course of OncotoX-AML treatment to immunocompetent primates demonstrated the desired pharmacologic response, selectively reducing myeloid progenitor cells, which can mutate and lead to AML, by up to 99%. OncotoX-AML was well tolerated. There were no observed safety signals or meaningful changes in blood chemistry values.

● Targeted Complement Activating Therapy ("T-CAT") platform

o Our T-CAT platform is a new class of recombinant antibodies designed to target and directly kill pathogens, including bacteria, fungi, viruses, and parasites. Our initial focus is on multidrug-resistant organisms ("MDROs"), one of the most critical unmet needs in medicine.

o Data from our T-CAT platform were recently featured in a podium presentation at the annual congress of the European Society of Clinical Microbiology and Infectious Diseases.

o The seminal manuscript describing our T-CAT technology was accepted for publication in Science Translational Medicine.

Financial Results

Commercial distribution and sales of YARTEMLEA commenced in January 2026. Gross product sales for the three months ended March 31, 2026 were $11.1 million, with net sales of $9.9 million. Revenue for the period reflects sales of YARTEMLEA to U.S. wholesalers.

Net income for the first quarter of 2026 was $56.1 million, or $0.78 per share, compared to a net loss of $33.5 million, or $0.58 per share for the first quarter of 2025.

The change in fair value of financial instruments as shown in our statement of operations and comprehensive income (loss) reflects marking to market the embedded derivative on our 2029 Notes under GAAP. Excluding the net gain on the change in the fair value of our financial instruments, which is non-cash, our non-GAAP adjusted net loss for the three months ended March 31, 2026 was $17.1 million, or $0.24 per share.

At March 31, 2026, we had $135.3 million of cash and short-term investments. Upon their maturity in February 2026, we repaid the remaining $17.1 million outstanding principal balance of our 2026 Notes and currently have only $70.8 million aggregate principal amount outstanding of our 2029 Notes, which mature in June 2029.

Total operating expenses for the three months ended March 31, 2026 were $27.3 million compared to $35.0 million for the three months ended March 31, 2025. The $7.7 million decrease was primarily due to reduced OMS906-related research and development work as a result of the zaltenibart asset sale and licensing agreement with Novo Nordisk in November 2025.

Interest expense increased $2.2 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. The increase primarily relates to interest incurred on the 2029 Notes and, to a lesser extent, a non-cash remeasurement charge taken on our OMIDRIA royalty obligation in the prior year, offset by decreased interest incurred on our 2026 Notes, which were repaid in February 2026.

Interest and other income was $1.5 million for the three months ended March 31, 2026 compared to $1.1 million for the three months ended March 31, 2025 due to holding higher cash and investment balances in the current period.

Net income from discontinued operations, net of tax, was $4.8 million, or $0.07 per share, for the three months ended March 31, 2026 compared to $4.1 million, or $0.07 per share, in the prior year period.

During the three months ended March 31, 2026, we repurchased and retired approximately 0.4 million shares of common stock pursuant to our share repurchase program, at an average cost of $11.70 per share, for an aggregate purchase price of $4.2 million.

Conference Call Details

Omeros’ management will host a conference call and webcast to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time.

For online access to the live webcast of the conference call, please register at the following URL View Source or go to Omeros’ website at View Source

A replay of the call will be made accessible online for 90 days at View Source

(Press release, Omeros, MAY 13, 2026, View Source [SID1234665636])