On May 12, 2025 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported financial results for the first quarter ended March 31, 2025 and highlighted progress on key clinical development programs (Press release, Karyopharm, MAY 12, 2025, View Source,-Symptom-Improvement,-Hemoglobin-Stabilization-and-Disease-Modificati [SID1234652885]).

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"We are pleased that our Phase 3 SENTRY trial in patients with JAKi-naïve myelofibrosis has passed its pre-specified futility analysis and continues as planned without modifications. Our conviction in this trial is further strengthened by our new data in hard-to-treat myelofibrosis patients where we observed spleen volume reduction, symptom improvement, hemoglobin stabilization and evidence of disease modification with selinexor monotherapy, addressing all four hallmarks of the disease," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "The outcomes that we have observed from our multiple pre-clinical and clinical trials continue to suggest that selinexor may be additive, if not synergistic, when combined with ruxolitinib in JAKi-naïve myelofibrosis patients. This combination holds the potential to be a much needed, new standard of care for patients with myelofibrosis."

First Quarter 2025 Highlights

XPOVIO Commercial Performance

Demand for XPOVIO increased 5% in the first quarter of 2025 compared to the first quarter of 2024, with demand growth in both the community and academic settings.
U.S. net product revenue was $21.1 million in the first quarter of 2025 compared to $26.0 million in the first quarter of 2024. Net product revenue in the first quarter of 2025 was impacted by a $5.0 million increase in the product return reserve due to atypical returns of expired 80 mg and 100 mg units from clinics and hospitals that had purchased these units following the 2020 approval by the U.S. Food and Drug Administration of XPOVIO 100 mg in combination with bortezomib and dexamethasone. The majority of XPOVIO that is prescribed today are 40 mg and 60 mg doses, and the Company expects product returns will revert to historical levels in future quarters.
Expanded global patient access for selinexor is translating into growth in royalty revenue from Menarini, Antengene and other international partners. Royalty revenue increased 57% to $1.7 million in the first quarter of 2025 compared to the first quarter of 2024.
Research and Development (R&D) Highlights

Myelofibrosis

The Phase 3 SENTRY trial (XPORT-MF-034; NCT04562389) continues as planned without any modifications following a pre-specified futility analysis conducted by the independent Data Safety and Monitoring Board. The trial is now approximately 80% enrolled. SENTRY is evaluating 60 mg once-weekly selinexor in combination with ruxolitinib compared to ruxolitinib plus placebo and is targeting 350 patients for enrollment.
Data from the XPORT-MF-035 (NCT04562870) Phase 2, randomized, open-label trial of selinexor versus physician’s-choice (PC) in hard-to-treat patients with heavily pretreated myelofibrosis indicated signs of single-agent clinical activity with selinexor, including spleen volume reduction, hemoglobin stabilization, symptom improvement and evidence of disease modification. Patients were randomized 1:1 to either selinexor monotherapy or PC. Selinexor was administered at 80 mg weekly for the first two cycles and then decreased to 60 mg weekly from cycle 3 onwards. An optional crossover was available for PC treated patients if they met predefined spleen progression criteria. In total, 12 patients were randomized to the selinexor arm and 12 patients to the PC arm; 6 patients crossed over from PC to selinexor. Inclusive of crossover, spleen volume reduction of 25% or more (SVR25) at anytime was achieved in 8/12 (67%) efficacy evaluable selinexor treated patients versus 3/8 (38%) efficacy evaluable patients treated with PC. Spleen volume reduction of 35% or more (SVR35) at anytime was observed in 4/12 (33%) efficacy evaluable patients treated with selinexor versus 1/8 (13%) efficacy evaluable patients treated with PC. Patients treated with selinexor had higher mean hemoglobin levels throughout the study duration and lower rates of red blood cell transfusions than those treated with PC. In addition, data on key cytokines that are relevant to myelofibrosis pathogenesis, symptom development, and anemia including IL-6, IL-8, TNFa, and hepcidin, demonstrated greater reductions at week 4 compared to baseline in patients treated with selinexor than patients treated with PC. Most common (≥25% overall) treatment emergent adverse events (TEAEs) in the randomized arms were decreased weight (selinexor: 50%; PC: 50%), anemia (25%; 58%), asthenia (42%; 25%), nausea (33%; 25%), thrombocytopenia (33%; 25%) and upper respiratory tract infection (25%; 33%). Most common (≥15% in any arm) Grade 3+ TEAEs were anemia (17%; 58%), cardiac failure (0%; 17%), decreased appetite (17%; 0%) and nausea (17%; 8%). No treatment discontinuations due to TEAEs were observed in the selinexor arm. In 2023, Karyopharm decided to stop enrollment at 24 patients in this trial to focus resources on the Phase 3 SENTRY trial.
The Company continues to enroll patients into the 60 mg cohort of the Phase 2 SENTRY-2 trial of selinexor monotherapy in JAKi-naïve patients with moderate thrombocytopenia (XPORT-MF-044; NCT05980806).
Endometrial Cancer

Enrollment continues in the Phase 3 XPORT-EC-042 (NCT05611931) trial evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. The Company expects to report top-line data from this event-driven trial in mid-2026.
Multiple Myeloma

Enrollment of approximately 120 patients in the Phase 3 XPORT-MM-031 trial (EMN29; NCT05028348) was completed in the fourth quarter of 2024. 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 in their immediate prior line of therapy. The Company expects to report top-line data from this event-driven trial in the first half of 2026.
Anticipated Catalysts and Operational Objectives in 2025

Myelofibrosis

Announce completion of target enrollment (N=350) of the Phase 3 SENTRY trial evaluating selinexor in combination with ruxolitinib in JAKi-naive myelofibrosis patients in June/July 2025.
Report preliminary data on a subset of participants in the 60 mg cohort from the Phase 2 SENTRY-2 trial evaluating selinexor as a monotherapy in patients with JAKi-naïve myelofibrosis with moderate thrombocytopenia in 1H 2025.
Report top-line results from the Phase 3 SENTRY trial in late 2025/early 2026.
Multiple Myeloma

Maintain the Company’s commercial foundation in the increasingly competitive multiple myeloma marketplace and drive increased XPOVIO revenues.
Continue global launches and regulatory and reimbursement approvals for selinexor by partners in ex-U.S. territories.
Continue to follow patients that are enrolled in the Phase 3 XPORT-MM-031 (EMN29) trial.
Endometrial Cancer

Continue to enroll patients into the Phase 3 XPORT-EC-042 trial of selinexor as a maintenance monotherapy for patients with TP53 wild-type advanced or recurrent endometrial cancer.
2025 Financial Outlook

Based on its current operating plans, Karyopharm expects the following for full year 2025:

Total revenue to be in the range of $140 million to $155 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 $240 million to $255 million, which includes approximately $20 million of estimated non-cash stock-based compensation expense.
The Company expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO net product sales, as well as revenue generated from its license agreements, will fund its planned operations into early first quarter of 2026.1
1Excluding re-payment of $24.5 million aggregate principal amount of the Company’s remaining senior convertible notes due October 2025 (the 2025 Notes) and $25.0 million minimum liquidity covenant under the Company’s senior secured term loan due 2028. Taking into account the repayment of the 2025 Notes and the minimum liquidity covenant, Karyopharm expects its cash, cash equivalents and investments will fund its operations into early fourth quarter of 2025. The Company is exploring various alternatives to extend its cash runway, which may not result in the consummation of any transaction.

First Quarter 2025 Financial Results

Please note: All share amounts and per share amounts in this press release have been adjusted to reflect a 1-for-15 reverse split of our common stock, which we effected on February 25, 2025.

Total revenue: Total revenue for the first quarter of 2025 was $30.0 million, compared to $33.1 million for the first quarter of 2024.

Net product revenue: Net product revenue for the first quarter of 2025 was $21.1 million, compared to $26.0 million for the first quarter of 2024. The decrease in net product revenue was due to an increase in the gross-to-net provision, primarily related to atypical product returns recorded in the first quarter of 2025, resulting in a $5.0 million increase in the product return reserve.

License and other revenue: License and other revenue for the first quarter of 2025 was $9.0 million, compared to $7.1 million for the first quarter of 2024. The increase was attributable to timing of revenue recognition for the reimbursement of development-related expenses from Menarini.

Cost of sales: Cost of sales for the first quarter of 2025 was $1.3 million, compared to $1.9 million for the first quarter of 2024. Cost of sales reflects the costs of XPOVIO units sold and the costs of products sold to our partners.

R&D expenses: R&D expenses for the first quarter of 2025 were $34.6 million, compared to $35.4 million for the first quarter of 2024. The decrease was due to a reduction in personnel costs, partially offset by increased clinical trial activity related to our myelofibrosis trial.

SG&A expenses: SG&A expenses for the first quarter of 2025 were $27.4 million, compared to $29.5 million for the first quarter of 2024. The decrease was primarily due to the realization of previously implemented cost reduction initiatives.

Interest income: Interest income for the first quarter of 2025 was $1.0 million, compared to $2.2 million for the first quarter of 2024. The decrease in interest income was due to a lower cash and investments balance quarter-over-quarter.

Interest expense: Interest expense for the first quarter of 2025 was $11.0 million, compared to $5.9 million for the first quarter of 2024. The increase was related to the term loan and convertible debt that were issued in the second quarter of 2024.

Other income: Other income for the first quarter of 2025 was $19.8 million due to recurring non-cash fair value remeasurements which related to the refinancing transactions that were completed in the second quarter of 2024. The Company had immaterial other income in the first quarter of 2024.

Net loss: Karyopharm reported a net loss of $23.5 million, or $2.77 per basic and diluted share, for the first quarter of 2025, compared to a net loss of $37.4 million, or $4.85 per basic and diluted share, for the first quarter of 2024. Net loss included non-cash stock-based compensation expense of $3.6 million and $5.0 million for the first quarters of 2025 and 2024, respectively.

Cash position: Cash, cash equivalents, restricted cash and investments as of March 31, 2025 totaled $70.3 million, compared to $109.1 million as of December 31, 2024.

Conference Call Information

Karyopharm will host a conference call today, May 12, 2025, at 4:30 p.m. Eastern Time, to discuss the first quarter 2025 financial results, the financial outlook for 2025 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 XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm 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; (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in adult patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. 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 endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: medicalinformation@karyopharm.com

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).
For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
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%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
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.

On May 12, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, reported new data on its proprietary γδ T manufacturing program (Press release, In8bio, MAY 12, 2025, View Source [SID1234652884]). The oral presentation shared exciting updates about its unique manufacturing processes, including deep analytics, genomics and repertoire analysis, presented at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting. The data, which earned IN8bio the prestigious Host Region (U.S. East) Abstract Award, as presented by Bruce Levine, PhD, ISCT Past President and a member of IN8bio’s Scientific Advisory Board. The award is a competitive recognition for scientific excellence and demonstrates how IN8bio’s technology and know-how creates consistent, powerful cellular therapies.

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The oral presentation highlights how IN8bio’s DeltEx Allo manufacturing process consistently induces donor derived T cells to express γδ T cell receptors (TCRs) and genes, associated with increased cancer cytotoxicity. Gene expression profiling confirmed a highly potent product across multiple manufacturing batches.

"Manufacturing abilities are paramount to the successful and safe development of cellular therapies. From process development through clinical manufacturing and product characterization, IN8bio uniquely maintains hands-on control of all steps in the process. These data and the Abstract Award further establish IN8bio as a leader in clinical grade γδ T cell manufacturing," said Kate Rochlin, Chief Operating Officer of IN8bio. "The power of our platform is our ability to consistently produce robust cell products with signatures associated with cytotoxicity, immune activation, and tissue trafficking. Our knowledge and know-how are key differentiators as we efficiently scale manufacturing to support our clinical trials and potential future commercialization."

Key Findings from the INB-100 Study Presented at ISCT:

Manufacturing-Driven TCR Reprogramming: All analyzed clinical batches showed a clear and consistent shift from αβ-TCR to γδ-TCR dominance, with enrichment of Vγ9 clones. This shift occurred independent of donor starting material, indicating the process—not the donor—drives final TCR composition.
Uniform Potency Markers Across Donors: All manufactured clinical products showed high expression levels of genes linked to increased cancer killing, immune activation, and tumor seeking migration.
Durable Remissions in AML Patients: All 10 patients in the initial cohort remained relapse-free for more than one year, with a median overall survival of 23.3 months as of January 2024.

These results highlight the strength and reproducibility of IN8bio’s cell therapy manufacturing processes. These products were administered clinically in the INB-100 clinical trial, which has demonstrated durable long-term remissions in adult AML patients with high-risk, complex disease characteristics. Further, long term expansion and persistence of the γδ T cells have been observed in these patients through 1 year, a first for an allogeneic cellular therapy product. IN8bio‘s manufacturing program has been automated, enabling rapid and reproducible production of cryopreserved cell therapy doses. The INB-100 trial continues to enroll an expansion cohort at the recommended Phase 2 dose (RP2D).

On May 12, 2025 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage company in Phase 3 development with its DNA-mediated immunotherapy, reported financial results for the three months ended March 31, 2025 and highlighted recent business updates, including progress in advancing Phase 3 clinical development of its lead candidate IMNN-001 in newly diagnosed advanced ovarian cancer (Press release, IMUNON, MAY 12, 2025, View Source [SID1234652883]).

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"IMUNON continues to make significant strides towards our goal of transforming the treatment landscape for women with advanced ovarian cancer. In 2024, our Phase 1/2 OVATION 2 Study delivered groundbreaking data, demonstrating that IMNN-001 is the first immunotherapy to extend both progression-free and overall survival in women newly diagnosed with ovarian cancer when combined with chemotherapy, and we continue to report promising results from the trial further supporting our therapeutic approach, including new translational and safety data this past quarter based on ongoing analyses," said Stacy Lindborg, Ph.D., president and chief executive officer of IMUNON.

"We look forward to building on this data and momentum, with a strong first quarter in 2025. The initiation of the first trial site for our Phase 3 OVATION 3 pivotal study marks a critical step toward our goal of delivering a new frontline treatment for women with limited options and urgent unmet medical needs. The acceptance of our OVATION 2 results for an oral presentation at ASCO (Free ASCO Whitepaper) also underscores the scientific community’s recognition of IMNN-001’s potential. These milestones reflect the strength of our clinical program, the dedication of trial investigators and patients, and our productive engagement with the FDA to finalize our Phase 3 study design. As we advance this historic opportunity, IMUNON remains committed to bringing safe, effective therapies to women battling ovarian cancer," Dr. Lindborg continued.

RECENT DEVELOPMENTS

IMNN-001 Immunotherapy

First Trial Site Initiated for Phase 3 OVATION 3 Study of IMNN-001 in Newly Diagnosed Advanced Ovarian Cancer – On May 8, 2025, the Company announced initiation of the first trial site for the OVATION 3 Study and is working with trial investigators to begin enrolling study participants.

The Phase 3 OVATION 3 trial will assess the safety and efficacy of IMNN-001 (100 mg/m2 administered intraperitoneally weekly) plus neoadjuvant and adjuvant chemotherapy (NACT) of paclitaxel and carboplatin compared to standard of care (SoC) NACT alone. Study participants will be randomized 1:1 and include women with newly diagnosed advanced ovarian cancer (stage 3C or 4) who are eligible for neoadjuvant therapy, the intent-to-treat (ITT) population, with a sub-group of women positive for homologous recombination deficiency (HRD), including BRCA1 or BRCA2 mutations. Participants who are HRD positive will receive poly ADP-ribose polymerase (PARP) inhibitors as part of standard maintenance therapy. The primary endpoint of the study is overall survival (OS), and secondary endpoints are surgical response score, chemotherapy response score, clinical response and time to second-line treatment. The study will also assess several exploratory endpoints.

IMNN-001 Data from Phase 1/2 OVATION 2 Study to be Published in Peer-Reviewed Journal Gynecologic Oncology – On May 6, 2025, the Company announced that data from the Phase 1/2 OVATION 2 clinical trial evaluating intraperitoneal IMNN-001 in combination with NACT in newly diagnosed patients with advanced epithelial ovarian cancer will be published in the peer-reviewed journal Gynecologic Oncology. The review of full data, entitled: OVATION-2: A Randomized Phase I/II study Evaluating the Safety and Efficacy of IMNN-001 (IL-12 gene therapy) with Neo/Adjuvant Chemotherapy in Patients Newly- Diagnosed with Advanced Epithelial Ovarian Cancer, is scheduled for publication on June 3, 2025.

IMNN-001 Abstract Accepted for Oral Presentation at 2025 ASCO (Free ASCO Whitepaper) Annual Meeting – On April 21, 2025, IMUNON announced that an abstract highlighting new data from the Phase 2 OVATION 2 Study of IMNN-001 to treat women with newly diagnosed advanced ovarian cancer was accepted for oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30 – June 3, 2025, in Chicago, Illinois. Details of the ASCO (Free ASCO Whitepaper) oral presentation are as follows:

Abstract Title: A phase I/II study of the safety and efficacy of intraperitoneal IMNN-001 in combination with neoadjuvant chemotherapy (NACT) of paclitaxel and carboplatin in patients newly diagnosed with advanced epithelial ovarian cancer (EOC): Updated survival analysis from OVATION-2 trial.

Presenting Author: Premal H. Thaker, M.D., Interim Chief of Gynecologic Oncology, David & Lynn Mutch Distinguished Professor of Obstetrics & Gynecology, Director of Gynecologic Oncology Clinical Research, Washington University School of Medicine, OVATION 2 Study Chair

Date: Tuesday, June 3, 2025

Session Time: 8:00-9:30 a.m. CT

Session Title: Gynecologic Cancer

Abstract Number: 5516

Translational Data from OVATION 2 Study of IMNN-001 in Newly Diagnosed Advanced Ovarian Cancer – reinforce dose-dependent mechanism with IMNN-001 100 mg/m2 dose and continue to validate TheraPlas technology, demonstrating DNA-mediated production of key anti-cancer immune cytokines following treatment – On February 19, 2025, IMUNON announced new translational data from ongoing analyses of results from the Phase 2 OVATION 2 Study of IMNN-001 for the treatment of newly diagnosed advanced ovarian cancer. Results reinforced the dose-dependent mechanism with the IMNN-001 100 mg/m2 dose (administered intraperitoneally weekly) and continue to validate the Company’s TheraPlas technology, demonstrating DNA-mediated production of key anti-cancer immune cytokines following treatment. The results also demonstrated a 20% increase in IL-12 levels in women treated with IMNN-001 plus SoC NACT compared to IL-12 levels in women treated with IMNN-001 (79 mg/m2). In this analysis, increases in IL-12 levels were sampled in the peritoneal fluid cavity, which is the primary tumor microenvironment. Little to no changes were observed in the systemic blood stream of treated patients. In addition, the rise in IL-12 levels was accompanied by local increases in interferon-gamma (IFN-γ) and tumor necrosis factor-alpha (TNF-α), key downstream anti-cancer immune cytokines. Results showed no reports of serious immune-related adverse events including cytokine release syndrome.

PlaCCine DNA Vaccine Technology

New Immunogenicity Data from Phase 1 Proof-of-Concept Clinical Trial of PlaCCine DNA Vaccine in COVID-19 – On February 26, 2025, the Company announced new safety and immunogenicity data from the first Phase 1 proof-of-concept clinical trial of IMNN-101, an investigational DNA plasmid vaccine based on the Company’s proprietary PlaCCine technology platform. The Phase 1 study was conducted in 24 healthy volunteers as a seasonal COVID-19 vaccine, targeting the SARS-CoV-2 Omicron XBB1.5 spike antigen. IMNN-101 was administered as a single dose vaccine without a booster dose in study participants who were previously vaccinated against the Omicron XBB1.5 variant. Results demonstrated that IMNN-101 is safe and well-tolerated with no serious adverse effects. IMNN-101 induced a persistent 2- to 4-fold increase in serum neutralizing antibody (NAb) titers from baseline through Week 4, further increasing NAb titers between Week 2 and Week 4. The immune response was observed against the XBB1.5 variant and many newer variants following treatment, demonstrating the IMNN-101 vaccine’s cross-reactivity. Study participants had high baseline immune characteristics presumably from prior infection and multiple previous vaccinations against COVID-19 and ongoing infection as evidenced by the rise in viral nucleocapsid antigen during the study period. Modest increases in T cell responses were observed in this setting of trial participants having received multiple immunizations prior to the study.

The Phase 1 clinical data of IMNN-101 is consistent with strong evidence of immunogenicity and protection for the PlaCCine platform in rodents and non-human primates, with prior preclinical results showing that protection exceeded 95% in non-human primates, which is comparable to mRNA vaccines. The robust immunogenicity profile, expected durability of protection, comparative ease of manufacturing, and stability at workable temperatures (up to one year at 4°C and one month at 37°C) suggest that a vaccine based on the PlaCCine technology platform may be a potential viable alternative to available messenger RNA (mRNA) vaccines. The Company plans to seek potential partners for further development of IMNN-101.

Corporate Highlights

Addition to Leadership Team to Support Future Clinical Programs – On February 10, 2025, the Company announced that Douglas V. Faller, M.D., Ph.D., was appointed Chief Medical Officer of IMUNON, effective February 18, 2025. Dr. Faller has more than 30 years of industry, academic and laboratory experience, with specialized expertise in oncology and immunology. Dr. Faller is responsible for leading the Company’s clinical strategy including advancing the IMNN-001 program for the treatment of newly diagnosed advanced ovarian cancer.

FIRST QUARTER 2025 FINANCIAL RESULTS

Net loss for the first quarter of 2025 was $4.1 million, or $0.28 per share, compared with a net loss of $4.9 million, or $0.52 per share, for the first quarter of 2024. Operating expenses were $4.1 million for the first quarter of 2025, a decrease of $0.9 million or 18% from $5.0 million for the first quarter of 2024.

Research and development (R&D) expenses were $2.2 million for the first quarter of 2025, a decrease of $1.1 million from $3.3 million for the first quarter of 2024. The decrease was due primarily to lower costs associated with the OVATION 2 Study and the Phase 1 proof-of-concept PlaCCine DNA vaccine trial, a decrease in costs associated with the development of IMNN-001 to support the OVATION 2 Study, and lower costs associated with development of the PlaCCine DNA vaccine technology platform.

General and administrative expenses were $2.0 million for the first quarter of 2025, compared with $1.7 million for the first quarter of 2024. The increase was primarily attributable to higher employee-related expenses, partially offset by lower legal expenses.

Net cash used for operating activities was $2.8 million for the first quarter of 2025, compared with $5.9 million for the same period last year. This decrease was primarily due to lower R&D expenses and higher accounts payable and accrued liability balances.

As of March 31, 2025, cash and cash equivalents were $2.9 million. The Company believes it has sufficient capital resources to fund its operations into late second quarter of 2025.

Conference Call and Webcast

The Company is hosting a conference call to review first quarter 2025 financial results and provide a business update today, May 12, 2025, at 11:00 a.m. ET. To participate in the call, please dial 833-816-1132 (Toll-Free/North America) or 412-317-0711 (International/Toll) and ask for the IMUNON First Quarter 2025 Financial Results Call. A live webcast of the call will also be available here.

The call will be archived for replay until May 26, 2025. The replay can be accessed at 877-344-7529 (U.S. Toll-Free), 855-669-9658 (Canada Toll-Free) or 412-317-0088 (International Toll), using the replay access code 2322959. An audio replay of the call will also be available here for 90 days.

On May 12, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, reported financial results for the quarter ended March 31, 2025 and provided a business update (Press release, Immunome, MAY 12, 2025, View Source [SID1234652882]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Immunome continued to build momentum in the first quarter of 2025," said Clay B. Siegall, Ph.D., President and Chief Executive Officer. "We remain on track to share topline data for the RINGSIDE trial of varegacestat in the second half of 2025, and we are optimistic that varegacestat will demonstrate differentiated efficacy across multiple endpoints."

"Varegacestat is complemented by a robust pipeline of differentiated programs with first-in-class or best-in-class potential. The dose escalation portion of the Phase 1 clinical trial of IM-1021, our ROR1 ADC, is ongoing in solid tumors and lymphoma. Following the recent clearance of the IND for IM-3050, our FAP radiotherapy, we expect to start a clinical trial for that program in the second half of this year. We are making progress on IND-enabling manufacturing for IM-1617, IM-1335 and IM-1340, each of which incorporate our differentiated TOP1 inhibitor, HC74."

Pipeline Highlights

Varegacestat: Full enrollment for the Phase 3 RINGSIDE Part B study of varegacestat for the treatment of desmoid tumors was completed in February 2024, and Immunome expects to report topline data for RINGSIDE Part B in the second half of 2025. In parallel, Immunome is performing the manufacturing and pharmacology work required to support a new drug application filing for varegacestat.

IM-1021: The Phase 1 clinical trial of IM-1021 is ongoing, with the first patient dosed in February 2025. The trial is an open-label, multicenter dose-escalation and expansion study that is expected to include participants with advanced B-cell lymphomas and advanced solid tumors.

IM-3050: Immunome received IND clearance for IM-3050 in April 2025 and expects to initiate a Phase 1 clinical trial in the second half of 2025.

Preclinical Pipeline: Immunome’s three preclinical ADCs against solid tumor targets, IM-1617, IM-1340 and IM-1335, each of which incorporates HC74, are currently undergoing IND-enabling work. Additional undisclosed ADCs are in discovery and lead optimization.

First Quarter 2025 Financial Results

· As of March 31, 2025, cash, cash equivalents and marketable securities totaled $317.3 million. This total includes net proceeds of $161.7 million from the January 2025 financing and reflects approximately $11 million in payments during the quarter related to non-recurring IM-1021 milestones and 2024 annual performance bonuses. Immunome expects its current cash position to fund operations into 2027.
· Research and development expenses for the quarter ended March 31, 2025 were $36.9 million, including stock-based compensation costs of $2.4 million.
· General and administrative expenses for the quarter ended March 31, 2025 were $10.7 million, including stock-based compensation expense of $3.3 million.
· Immunome reported a net loss of $41.6 million for the quarter ended March 31, 2025.

On May 12, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported certain operational results following approval of the permanent J-code (J9028) in January 2025, as well as its financial results for the first-quarter ended March 31, 2025 (Press release, ImmunityBio, MAY 12, 2025, View Source [SID1234652881]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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With the issuance of the permanent J-code (J9028) in January 2025, ImmunityBio has seen increased sales momentum supporting a trend of increases month-over-month as well as quarter-over-quarter, with March unit sales volume increasing 69% over February, and Q1 2025 unit sales volume exceeding unit sales volume achieved for all of fiscal year 2024.
ImmunityBio earned net product revenue of approximately $16.5 million during the three-month period ended March 31, 2025, which represented an increase of 129% over the $7.2 million of net revenue earned during the fourth quarter of 2024.
"We are seeing a steady growth in revenue as urologists increase their use of ANKTIVA to treat NMIBC carcinoma in situ (CIS) patients, particularly since we addressed the BCG shortage with the launch of our rBCG EAP in February," said Richard Adcock, President and CEO of ImmunityBio. "Nearly 200 urological practices are in early stages of implementation or have already begun administering rBCG to patients, many of them in rural areas where patients otherwise would not have access to this treatment. This not only lets us help more patients, it opens a new marketplace for ImmunityBio’s therapies."
"ANKTIVA’s increasing use by urologists shows the real-world benefits of our unique approach to immunotherapy," said Dr. Patrick Soon-Shiong, Founder, Executive Chairman, Global Chief Scientific & Medical Officer of ImmunityBio. "We’re making excellent progress in developing our pipeline, and now have multiple sites open for our second-line lung cancer study as well as having submitted an EAP protocol for ANKTIVA for the treatment of lymphopenia. We are confident that we will continue to deliver more advanced treatment candidates based on our solid science."

First-Quarter Ended March 31, 2025 Financial Summary and Comparison to Prior Year Quarter
Product Revenue, Net

Product revenue, net increased $16.5 million during the three months ended March 31, 2025, as compared to the three months ended March 31, 2024, due to sales of ANKTIVA, which was approved in April 2024.

Research and Development Expense

Research and development (R&D) expense decreased $5.1 million to $48.2 million during the three months ended March 31, 2025, as compared to $53.3 million during the three months ended March 31, 2024. The decrease was primarily driven by lower external manufacturing costs, research agreement expenses, stock-based compensation expenses, equipment expenses, and consulting costs.

Selling, General and Administrative Expenses

Selling, general and administrative expense (SG&A) decreased $9.2 million to $32.7 million during the three months ended March 31, 2025, as compared to $41.9 million during the three months ended March 31, 2024. The decrease was due to lower costs related to litigation settlements and commercial consulting activities.

Net Loss Attributable to ImmunityBio Common Stockholders

Net loss attributable to ImmunityBio common stockholders was $129.6 million during the three months ended March 31, 2025, compared to $134.1 million during the three months ended March 31, 2024. The reduction of loss was primarily driven by product revenue, lower R&D and SG&A expense described above, lower related-party interest expense, and changes in the fair value of derivative liabilities, partially offset by changes in the fair value of our related-party convertible note and an increase in interest expense related to the revenue interest liability.

Cash and Marketable Securities Position

As of March 31, 2025, on a pro forma basis, the Company had consolidated cash and cash equivalents, and marketable securities of $136.4 million after giving effect to net proceeds of $74.8 million from an equity financing in April.