Karyopharm Reports First Quarter 2026 Financial Results and Completion of Phase 3 Endometrial Cancer Trial Enrollment

On May 14, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported financial results for the first quarter of 2026 and highlighted progress on key clinical development programs. These milestones reflect meaningful progress towards Karyopharm’s goal of advancing selinexor in late-stage indications and expanding the value of the franchise.

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"As we move through 2026, Karyopharm is in a pivotal period with meaningful clinical and regulatory milestones ahead," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Through the second and third quarters of 2026, we expect several important developments that have the potential to create significant value for the Company, while improving outcomes for patients. In endometrial cancer, we have completed enrollment in our Phase 3 XPORT-EC-042 trial and remain on track to report topline data in mid-2026. In myelofibrosis, the Phase 3 SENTRY results reinforced the potential of selinexor to meaningfully improve patient outcomes, including a statistically significant SVR35 benefit and a promising overall survival signal. With the endometrial cancer readout ahead and the myelofibrosis regulatory and guideline path coming into focus, we believe these programs could meaningfully expand the impact and long-term opportunity of the selinexor franchise."

First Quarter 2026 and Recent Company Highlights

XPOVIO Commercial Performance

U.S. net product revenue was $29.2 million for the quarter ended March 31, 2026 compared to $21.1 million for the quarter ended March 31, 2025.
Demand for XPOVIO was lower in the first quarter of 2026 compared to the first quarter of 2025, due to new competitive entrants. The community setting continued to represent approximately 60% of net product revenue.
Expanded global patient access for selinexor is translating into growth in royalty revenue from Menarini, Antengene and other international partners. Royalty revenue increased to $1.9 million in the first quarter of 2026 compared to $1.7 million in the first quarter of 2025, with selinexor now approved in more than 50 ex-U.S. countries and territories.
Research and Development (R&D) Highlights

Myelofibrosis

Results from the Phase 3 SENTRY trial in myelofibrosis were accepted for a late-breaking oral presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Reported topline results from the Phase 3 SENTRY trial, a randomized, double-blind trial evaluating 60 mg selinexor in combination with ruxolitinib in frontline myelofibrosis compared to ruxolitinib alone (n=353). The trial met the first co-primary endpoint of spleen volume reduction of 35% or more (SVR35) at week 24 and also demonstrated rapid, deep and sustained improvement in this endpoint over time. The trial did not meet its second co-primary endpoint of mean change in absolute total symptom score (Abs-TSS) at week 24 relative to baseline. Similar symptom improvement from baseline was observed in patients who received the combination of selinexor plus ruxolitinib compared to ruxolitinib alone. Importantly, a promising overall survival signal was also observed, which further reinforces the relevance of XPO1 inhibition in combination with ruxolitinib in frontline myelofibrosis. The combination of selinexor and ruxolitinib demonstrated a manageable safety and tolerability profile consistent with the known profile of each agent individually with no new safety signals observed.
Completed enrollment of the 60 mg cohort (n=29) of the Phase 2 SENTRY-2 trial (NCT05980806) and began enrolling patients into the 40 mg cohort.
Endometrial Cancer

Completed enrollment of the Phase 3 XPORT-EC-042 trial (NCT05611931), which is evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. Approximately 220 patients enrolled in the modified intent-to-treat (mITT) population and 257 enrolled in the intent-to-treat (ITT) population. Enrollment in the mITT population was focused on patients with either proficient mismatch repair status (pMMR) tumors or patients with deficient mismatch repair status (dMMR) tumors who are medically ineligible for checkpoint inhibitors.
Multiple Myeloma

Patients enrolled in the Phase 3 XPORT-MM-031 trial (EMN29; NCT05028348) continued to be followed for progression-free survival events contributing towards the primary endpoint. The trial is being conducted in collaboration with the European Myeloma Network and is evaluating the all-oral combination of selinexor 40 mg, pomalidomide and dexamethasone (SPd40) in patients with previously treated multiple myeloma who received an anti-CD38 as their immediate prior line of therapy.
Anticipated Catalysts and Operational Objectives

Myelofibrosis

Present results from the Phase 3 SENTRY trial in myelofibrosis at ASCO (Free ASCO Whitepaper) as a late-breaking oral presentation on June 2 at 9:45 a.m. Central Time during the "Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant" oral abstract session. The Company expects that its abstract titled "Selinexor plus ruxolitinib in JAK inhibitor–naïve myelofibrosis: Phase 3 SENTRY trial" (abstract number LBA6500) will be available on ASCO (Free ASCO Whitepaper)’s website on June 2 at approximately 8:00 a.m. Eastern Time / 7:00 a.m. Central Time. A copy of the SENTRY presentation will be available following its presentation at ASCO (Free ASCO Whitepaper) under "Publications and Presentations" in the Investors & Media section of the Company’s website.
Engage with the U.S. Food and Drug Administration (FDA) on the data from the SENTRY trial and the Company’s supplemental new drug application (sNDA) filing plan; engage with other global regulatory agencies in collaboration with the Company’s partners.
The Company believes the potential inclusion of the combination in relevant compendia could occur in the second half of 2026.
Announce topline data from all patients in the 60 mg cohort of the Phase 2 SENTRY-2 trial with at least 24 weeks of follow-up, expected in the second half of 2026.
Endometrial Cancer

Announce topline data from the event-driven, Phase 3 XPORT-EC-042 trial, expected in mid-2026.
Multiple Myeloma

Maintain the Company’s commercial foundation in the increasingly competitive multiple myeloma marketplace and drive increased XPOVIO revenues.

Support global launches by our partners following regulatory and reimbursement approvals for selinexor in ex-U.S. countries and territories.

Announce topline data from the event-driven, Phase 3 XPORT-MM-031 (EMN29) trial, expected in the second half of 2026.
2026 Financial Outlook

Based on its current operating plans, Karyopharm expects the following for full year 2026:

Total revenue to be in the range of $130 million to $150 million. Total revenue consists of U.S. XPOVIO net product revenue and license, royalty and milestone revenue earned from partners.
U.S. XPOVIO net product revenue to be in the range of $115 million to $130 million.
R&D and selling, general and administrative (SG&A) expenses to be in the range of $230 million to $245 million.
The Company expects its existing liquidity, including cash and cash equivalents, as well as cash flow from net product revenue and license and other revenue, will enable it to fund its current operating plans into late in the third quarter of 2026.
First Quarter 2026 Financial Results

Total revenue: Total revenue for the first quarter of 2026 was $35.1 million, compared to $30.0 million for the first quarter of 2025.

Net product revenue: Net product revenue was $29.2 million for the first quarter of 2026, compared to $21.1 million for the first quarter of 2025. The increase was primarily driven by lower gross-to-net adjustments, which were 21.8% in the first quarter of 2026 versus 45.0% in the prior-year period. The gross-to-net adjustments were impacted by an atypical product return adjustment in the first quarter of 2025 and lower realized discounts and returns in the first quarter of 2026.

License and other revenue: License and other revenue was $5.9 million for the first quarter of 2026, compared to $9.0 million for the first quarter of 2025. License and other revenue for the first quarter of 2026 primarily consisted of $3.5 million in milestone revenue and $1.9 million in royalties. For the first quarter of 2025, license and other revenue primarily consisted of $7.0 million related to the reimbursement of development-related expenses and $1.7 million in royalties.

Cost of sales: Cost of sales was $1.3 million for both the first quarter of 2026 and 2025.

R&D expenses: R&D expenses were $33.8 million for the first quarter of 2026, compared to $34.6 million for the first quarter of 2025.

SG&A expenses: SG&A expenses were $26.7 million for the first quarter of 2026, compared to $27.4 million for the first quarter of 2025.

Loss from operations: Loss from operations was $26.8 million for the first quarter of 2026, compared to $33.3 million for the first quarter of 2025, representing a 20% improvement. The decrease reflects improved operating efficiency and disciplined cost management.

Interest income: Interest income was $0.5 million for the first quarter of 2026, compared to $1.0 million for the first quarter of 2025. The decrease reflects lower investment balances during 2026 compared to 2025.

Interest expense: Interest expense was $12.6 million for the first quarter of 2026, compared to $11.0 million for the first quarter of 2025. The increase reflects higher outstanding debt and higher interest rates following the Company’s financing transactions executed in October 2025.

Other income, net: Other income, net was $16.4 million in the first quarter of 2026, compared to $19.8 million in the first quarter of 2025. The amounts primarily reflect non-cash fair value remeasurements of embedded derivatives and liability-classified common stock warrants related to the refinancing transactions completed in the second quarter of 2024 and the fourth quarter of 2025.

Net loss: Net loss was $22.4 million, or $1.02 per basic share and $1.24 per diluted share, for the first quarter of 2026, compared to $23.5 million, or $2.77 per basic and diluted share, for the first quarter of 2025. Net loss included non-cash stock-based compensation expense of $3.0 million in the first quarter of 2026 compared to $3.6 million in the first quarter of 2025.

Cash position: Cash, cash equivalents, and restricted cash as of March 31, 2026 totaled $91.2 million, including approximately $50 million of gross proceeds raised from a private placement of common stock and warrants and sales of common stock under the Company’s Open Market Sale Agreement during the first quarter of 2026, compared to $64.1 million as of December 31, 2025.

Conference Call Information

Karyopharm will host a conference call today, May 14, 2026, at 8:00 a.m. Eastern Time, to discuss the first quarter 2026 financial results, the financial outlook for 2026 and to provide other business updates. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website. An archived webcast will be available on the Company’s website approximately two hours after the event.

About the Phase 3 SENTRY Trial

SENTRY (XPORT-MF-034; NCT04562389) is a Phase 3 clinical trial evaluating a once-weekly dose of 60 mg of selinexor in combination with ruxolitinib compared to placebo plus ruxolitinib in JAKi-naïve myelofibrosis patients with platelet counts >100 x 109/L (N=353). Patients were randomized 2-to-1 to the selinexor arm. The co-primary endpoints for this trial are spleen volume reduction ≥ 35% (SVR35) at week 24 and the average change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline.

About Myelofibrosis

Myelofibrosis is a rare blood cancer that affects approximately 20,000 patients in the United States and 17,000 patients in the European Union1. The disease causes bone marrow fibrosis (scarring in the bone marrow), which makes it difficult for the bone marrow to make healthy blood cells, splenomegaly (enlarged spleen), progressive anemia which often leads to symptoms like fatigue and weakness, and other disease associated symptoms including abdominal discomfort, pain under the left ribs, early satiety, night sweats and bone pain. The only approved class of therapies to treat myelofibrosis are JAK inhibitors, including ruxolitinib.

About the Phase 3 XPORT-EC-042 Trial

EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind clinical trial evaluating selinexor as a maintenance-only therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer (N=257). Patients were randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The trial includes two patient populations, for which the primary endpoint of progression free survival will be tested sequentially: 1) a modified intent to treat population (mITT) that includes patients with either, a) TP53 wild-type tumors with proficient mismatch repair status (pMMR); or, b) TP53 wild-type tumors with deficient mismatch repair status (dMMR), who are medically ineligible to receive checkpoint inhibitors; and, 2) the trial’s original intent to treat (ITT) population, which includes all patients enrolled in the trial whose tumors are TP53 wild-type, regardless of MMR status. The key secondary endpoint of overall survival will be evaluated in the ITT population. The mITT population is expected to include approximately 220 patients. In connection with the EC-042 trial, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type.

About Endometrial Cancer

Endometrial cancer (EC) is the most common gynecologic malignancy in the U.S.1 In 2026, approximately 68,000 uterine cancers (predominantly endometrial) are expected to be diagnosed, with approximately 14,000 deaths.1 Worldwide there were about 420,368 cases with 97,723 deaths in 2022.2 Both incidence and mortality have continued to rise.3,4 Key risk factors include obesity, type 2 diabetes, high-fat diets, tamoxifen or oral estrogen use, and delayed menopause.5 TP53 is a well-recognized prognostic marker for EC; >50% of advanced or recurrent EC tumors are TP53wt (gene for tumor protein P53; wild-type), and ~40%-55% are both TP53wt and mismatch repair-proficient (pMMR).6-8 While immune checkpoint inhibitors have shown benefit in patients with mismatch repair–deficient (dMMR) and pMMR, the magnitude of benefit is greater for patients with dMMR tumors versus pMMR tumors.9-10 There remains an unmet need for targeted therapies for patients with pMMR EC.11

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor compound for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved and marketed by Karyopharm in the U.S. in multiple oncology indications, including: (i) in combination with VELCADE (bortezomib) and dexamethasone (XVd) in adult patients with multiple myeloma after at least one prior therapy; and (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO/NEXPOVIO is marketed in these respective ex-U.S. territories by Karyopharm’s partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in myelofibrosis and endometrial cancer.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
Use In Specific Populations

Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or www.fda.gov/medwatch.

(Press release, Karyopharm, MAY 14, 2026, View Source [SID1234665707])

CREATE Medicines Announces $122 Million Series B Financing to Advance In Vivo CAR Pipeline in Autoimmune Disease and Oncology

On May 14, 2026 CREATE Medicines, Inc. ("CREATE"), a clinical-stage biotechnology company pioneering in vivo immune programming, reported the closing of its $122 million Series B funding round, co-led by existing investors Newpath Partners, ARCH Venture Partners, and Hatteras Venture Partners, with participation from Alexandria Venture Investments and other current members of CREATE’s investor syndicate.

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The funding will support advancement of CREATE’s clinical pipeline across autoimmune disease and oncology. To date, CREATE has dosed more than 50 patients across its in vivo CAR clinical programs — the largest clinical dataset in the field — generating the translational foundation that informs development across both autoimmune disease and oncology. The company’s proprietary mRNA-LNP platform programs immune cells directly inside the body, enabling a rapid, iterative product engine designed to compress concept-to-clinic timelines.

In autoimmune, CREATE’s lead program CRT-402, a next generation CD19 targeted in vivo CAR-T therapy, has demonstrated deep and durable B cell depletion in non-human primates, with the potential to enable immune reset through the flexibility of repeat dosing. The company is also advancing a dual CAR CD19 x BCMA directed therapy designed to broaden therapeutic reach across refractory autoimmune indications.
In oncology, CREATE continues to advance therapies focused on areas of high unmet need. Early clinical data from the company’s MT-303 program in frontline hepatocellular carcinoma has demonstrated an extremely compelling response profile.
Collectively, these programs exemplify CREATE’s iterative platform that integrates clinically validated CAR architectures, optimized RNA design, and targeted delivery technologies with deep expertise across clinical development, translational medicine, manufacturing, and regulatory execution.
"CREATE was built as an iterative immune programming platform in which each clinical study informs and strengthens the next, and it is that work that has revealed the breadth of what in vivo immune programming can address," said Daniel Getts, PhD, Chief Executive Officer of CREATE Medicines. "Our autoimmune and oncology pipeline represents the convergence of years of platform development, clinical execution, and translational learning. We believe our ability to engineer multiple immune cell populations directly in vivo has the potential to fundamentally reshape treatment paradigms across autoimmune disease and oncology, and we look forward to this next chapter of innovation and growth."

In conjunction with the Series B Round, Ron Philip, a veteran biopharma leader, has joined CREATE as Executive Chairman. Brian Cuneo, Senior Partner at ARCH Venture Partners, and Tom Thomas, PhD of Newpath Partners, have joined the company’s Board of Directors.

"I’m excited to join CREATE at this pivotal moment in the company’s evolution," said Ron Philip. "CREATE has established meaningful in vivo clinical proof points and built a differentiated immune programming platform with the potential to redefine how engineered immune therapies are developed and delivered. The opportunity to translate these capabilities into transformative medicines across autoimmune disease and oncology is exceptionally compelling."

"I co-founded this company because the science was ahead of the field. More than fifty patients later, it still is. Most in vivo cell therapy companies will struggle to translate science into scalable manufacturing. CREATE owns its manufacturing infrastructure. I am backing CREATE because it can become the next great standalone pharmaceutical company," said Tom Cahill, MD, PhD, Founder and Managing Partner of Newpath Partners.

"CREATE is building one of the most differentiated in vivo immune engineering platforms in the field," said Brian Cuneo. "We believe the company’s clinical experience, repeat dose strategy, and broad immune programming capabilities position it to play an important role in the future of autoimmune disease and oncology."

(Press release, Create Medicines, MAY 14, 2026, View Source [SID1234665733])

MacroGenics to Participate in the Stifel 2026 Virtual Targeted Oncology Forum

On May 14, 2026 MacroGenics, Inc. (Nasdaq: MGNX), a biopharmaceutical company focused on developing innovative antibody-based therapeutics for the treatment of cancer, reported that Eric Risser, President and CEO of MacroGenics, will participate in a fireside chat at the Stifel 2026 Virtual Targeted Oncology Forum on Wednesday, May 20, 2026 at 10:30 a.m. ET.

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A webcast of the presentation may be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at View Source The Company will maintain an archived replay of the webcasts on its website for 30 days.

(Press release, MacroGenics, MAY 14, 2026, View Source [SID1234665708])

Ring Therapeutics® Presents its Vector Conjugate and VectorBricks™ Manufacturing Platforms at the 29th Annual American Society of Gene & Cell Therapy Conference

On May 14, 2026 Ring Therapeutics, a life sciences company pioneering a new class of targeted medicines, reported new data on its Vector Conjugate platform and VectorBricks in vitro manufacturing technology. The data, which are being presented as a poster and an oral presentation at the 29th Annual American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) conference, highlight significantly increased cargo capacity for the delivery of oligonucleotides using targetable vectors and VectorBricks as a scalable in vitro manufacturing platform for the modular production of novel vector conjugate medicines.

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Two distinct but related technical challenges have constrained the field of targeted therapeutics: payload ceiling and manufacturing complexity. Ring’s Vector Conjugate platform brings together distinct innovations in vector discovery, design, and engineering to expand payload capacity and improve cell and tissue targeting with the aim of unlocking new targets for drug delivery. Ring’s modular VectorBricks manufacturing technology spans multiple innovations to reduce the complexity of vector manufacturing to the simplicity of producing a single recombinant protein. With VectorBricks, Ring is able to make vectors and vector conjugates at greater cost efficiency and scale than existing technologies.

"Ring’s Vector Conjugate platform delivers orders of magnitude more payload than antibodies and can carry anything from small molecules to oligonucleotides," said Dmitriy Bobrovnikov, PhD, Chief Innovation Officer of Ring Therapeutics. "The data we are presenting at ASGCT (Free ASGCT Whitepaper) highlight how our vectors carry siRNA directly as Vector Oligo Conjugates (VOCs), as well as the underlying VectorBricks manufacturing technology that makes our platform uniquely possible."

Bobrovnikov continued, "Payload capacity is crucial to unlocking new tissues and targets for oligonucleotide therapy. Selective tissue targeting needs to move beyond the transferrin receptor to receptors that require more payload capacity to compensate for their lower expression levels. Our VOCs enable selective targeting and ultimately expand oligo-conjugate technology beyond rare genetic disorders."

Presentation details and key highlights below:

Poster Presentation:

Title: Vector Oligonucleotide Carriers (VOCs): A platform for targeted oligonucleotide delivery with redosable viral capsids
Abstract number: 3356

Presentation location, date, and time: Exhibit and Poster Hall (Halls B2-C), Thursday, May 14, 5:00 – 6:30 PM ET

This work introduces Vector Oligonucleotide Conjugates (VOCs), a novel modular platform for viral capsid–mediated delivery of oligonucleotides.
VOCs were successfully manufactured by direct conjugation of synthetic siRNA molecules onto recombinant capsid proteins followed by in vitro capsid assembly, with controllable surface decoration with targeting ligands at varying densities.
Conjugation of either a small targeting ligand (TL) or a targeting monoclonal antibody to the VOC surface drove robust internalization into target cells.
TL-conjugated VOCs delivered functional siRNA payloads that triggered specific target gene knockdown in vitro, with TL functionalization enhancing potency over untargeted VOC.
Oral Presentation:

Title: Pilot-scale production of in vitro assembled Anellovirus capsids to establish a novel therapeutic platform
Speaker: Sunaina Kiran Prabhu, PhD – Associate Director, Tech Ops at Ring Therapeutics

Session Title and Location: Vector manufacturing and analytics – Room 257AB (Level 2)

Presentation time: Thursday, May 14, 4:15 – 4:30 PM ET

The presentation outlines VectorBricksTM, a cost-effective, highly modular in vitro assembly platform that offers versatility in payload and targeting. It consists of two key building blocks: a recombinant capsid protein and a therapeutic payload.
Recombinant Anellovirus capsid protein was produced in high titers, achieving cell-specific productivity levels comparable to traditional monoclonal antibody processes.
The robustness and scalability of this process was demonstrated at a 50-liter scale.
Purified pentamer building blocks were assembled in vitro into monodisperse capsid particles with high yields.
This modular platform was shown to successfully package diverse payloads including small molecules, siRNA and DNA.

(Press release, Ring Therapeutics, MAY 14, 2026, View Source;cell-therapy-conference-302771719.html [SID1234665734])

Monopar Therapeutics Reports First Quarter 2026 Financial Results and Provides Business Updates

On May 14, 2026 Monopar Therapeutics Inc. ("Monopar" or the "Company") (Nasdaq: MNPR), a clinical‐stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, reported first quarter 2026 financial results and provided business updates.

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Recent Program Developments

ALXN1840 for Wilson Disease – NDA Submission on Track for Mid-2026

In April 2026, Monopar presented new analyses from the randomized controlled Phase 3 FoCus trial of ALXN1840 (tiomolibdate choline, TMC) at the American Academy of Neurology (AAN) Annual Meeting 2026. The late-breaker oral and poster presentation, titled "Greater clinical benefit with tiomolibdate choline versus standard-of-care in neurologic Wilson disease patients in the Phase 3 FoCus Trial," demonstrated greater neurologic benefit of ALXN1840 versus standard of care ("SoC") in Wilson disease patients with neurologic symptoms at baseline.

These results from the FoCus trial will also be presented in a poster at the 12th Congress of the European Academy of Neurology (EAN) in Geneva, Switzerland, June 27-30, 2026. Dr. Aurélia Poujois, MD, PhD, of the Adolphe de Rothschild Foundation Hospital, a leading authority in Wilson disease, will present on June 28 at 12:50 CEST.

Monopar will also present at the European Association for the Study of the Liver (EASL) Congress 2026, a leading global forum for liver disease research. The presentation, titled "Tiomolibdate choline stabilizes liver disease and improves neurological symptoms as well as quality-of-life in treatment-experienced Wilson disease patients," will be presented by UC Davis Professor Dr. Valentina Medici, MD, MAS, FAASLD. EASL Congress 2026 will take place in Barcelona, Spain, from May 27-30, 2026, with Dr. Medici presenting on May 29 at 08:45 CEST.

The Company remains on track with its plans to submit the New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") in mid-2026. Susan Rodriguez, the Company’s Chief Commercial and Strategy Officer who joined in March 2026, is leading commercial readiness activities in preparation for a potential launch.

Financial Results for the First Quarter Ended March 31, 2026, Compared to the First Quarter Ended March 31, 2025

Cash and Net Loss

Cash, cash equivalents and investments as of March 31, 2026, were $137.5 million. Monopar expects its current funds to support operations at least through December 31, 2027, including: (1) regulatory and potential commercial activities for ALXN1840; (2) continued development of MNPR-101 programs; and (3) internal research and development.

Net loss for the first quarter of 2026 was $3.9 million, or $0.46 per share, compared to net loss of $2.6 million, or $0.38 per share, for the first quarter of 2025.

Research and Development ("R&D") Expenses

R&D expenses for the first quarter of 2026 were $3,487,247 compared to $1,643,375 for the first quarter of 2025. This represents an increase of $1,843,872 primarily attributed to (1) an $825,972 increase in R&D contractor and consulting expenses, (2) a $799,593 increase in R&D personnel expenses including stock-based compensation and (3) a net increase of $218,307 in other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the first quarter of 2026 were $1,738,006 compared to $1,578,442 for the first quarter of 2025. This represents an increase of $159,564 primarily attributed to (1) a $134,599 increase in G&A personnel expenses including stock-based compensation and (2) a net increase of $24,965 in other G&A expenses.

Interest Income

Interest income for the first quarter of 2026 was $1,332,203 compared to $596,845 for the first quarter of 2025. The increase is attributed to interest earned on U.S. Treasury securities and commercial paper, and higher bank balances in 2026, due to the net proceeds of approximately $91.9 million from the September 2025 capital raise.

(Press release, Monopar Therapeutics, MAY 14, 2026, View Source [SID1234665709])