On March 24, 2026 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its full-year financial results for the year ended December 31, 2025, and provides an update on its lead INTV asset TG4050 developed from its myvac platform together with upcoming plans for 2026.

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Alessandro Riva, MD, Chairman and CEO of Transgene, commented, "In 2025, we achieved important clinical progress with TG4050, the first product based on myvac, our individualized neoantigen therapeutic vaccine platform. The positive randomized Phase 1 results, which demonstrated durable disease-free survival and persisting neoantigen-specific immune responses in early-stage head and neck cancer, strengthen our confidence in its transformative potential for patients. All Phase 2 patients are close to being randomized , with the primary endpoint being two-year disease-free survival. In parallel, we are preparing a new Phase 1 trial in a second indication in an early treatment setting, reflecting our strategy to expand the clinical evaluation of myvac across operable solid tumors where significant unmet medical need remains.
"Together with the updated Phase 1 results for BT-001 presented at ESMO (Free ESMO Whitepaper) 2025, which support the program’s next development steps, and the successful fundraising completed at the end of 2025, we are well positioned to deliver key milestones while maintaining financial visibility. This strengthened position allows us to confidently advance our innovative pipeline in our mission to bring meaningful benefits to patients and reshape early‑stage cancer treatment with individualized neoantigen therapeutic vaccines."

TG4050: Data support TG4050’s potential role in preventing cancer relapse

Our individualized neoantigen therapeutic vaccine (INTV) TG4050 is currently under evaluation in a randomized multicenter Phase 1/2 clinical trial (NCT04183166), as a single agent in the adjuvant treatment of HPV-negative head and neck cancer (head and neck squamous cell carcinoma or HNSCC).

ASCO 2025: TG4050 meets all Phase 1 endpoints, with 100% disease-free survival (DFS) after more than 2 years of follow-up

Transgene presented positive data from the randomized Phase 1 part of the ongoing international Phase 1/2 trial in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO 2025) Annual Meeting (see press release). All patients who received TG4050 remained disease-free for at least 2-years (median follow-up: 30 months).

The results successfully met all trial endpoints (including safety, tolerability and feasibility).

Compelling translational findings from Phase 1 in TG4050-treated patients confirm durable, neoantigen-specific T-cell responses

Immunogenicity data presented at the 2025 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2025 (see press release), confirmed clinical proof of principle for TG4050. This includes the ability to induce neoantigen-specific cytotoxic CD8+ T cell responses capable of targeting and eliminating tumor cells, thereby contributing to the prevention of cancer relapse.
Translational data presented at SITC (Free SITC Whitepaper) 2025 showed that TG4050 induced neoantigen-specific T cell responses in the majority of treated patients (73% of 15 evaluable patients). These responses were durable (persisting 24 months after the start of treatment), with cytotoxic and effector phenotype markers expressed up to one year after the end of treatment.
A comprehensive analysis of the clinical and translational data from the Phase 1 part of the randomized Phase 1/2 trial of INTV-TG4050 was published on the preprint platform medRxiv2, in January 2026 (see press release). The article is under review by a peer-reviewed journal.

End of randomization in Phase 2 part close to being completed in the coming weeks – Next clinical readout expected 2 years following completion of randomization

The randomized Phase 2 part of the study for adjuvant HNSCC is nearly completed.
The primary endpoint of the trial is 2-year disease-free survival (DFS). This will be as soon as all patients achieve 2-year follow-up from randomization unless an event (relapse, death or lost to follow-up) occurs earlier.

3-year disease-free survival (DFS) follow-up for all patients is expected in Q2/Q3 2026.

myvac platform: Potential to reduce the risk of relapse across multiple operable solid tumor types

Transgene’s INTV platform, myvac, could improve treatment across a range of solid tumors where in many cases a significant unmet medical need remains. In parallel with the ongoing Phase 1/2 trial in HNSCC, Transgene has begun start-up activities for a new Phase 1 trial in a second indication in an early treatment setting, with the aim of initiating later in 2026.

Transgene is also optimizing its manufacturing processes and capabilities to prepare for a potential pivotal clinical trial.

VacDesignR: Transgene’s proprietary bioinformatics engine for cancer mutation selection and vector optimization

A poster on Transgene’s proprietary VacDesignR computational tool was presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) AI & Digital Oncology 2025 conference. Developed in-house, VacDesignR is a computational design engine that optimizes recombinant plasmid architecture for Modified Vaccinia Ankara (MVA) vectors, a core component of Transgene’s myvac platform. By minimizing unwanted homologous recombination and intelligently selecting peptide sequences for cassette assembly among targets that have previously been identified as potentially immunogenic, VacDesignR significantly improves production reliability and vector quality.

BT-001 (oncolytic virus for intratumoral administration): Updated Phase 1/2 data presented at ESMO (Free ESMO Whitepaper) 2025 demonstrated positive antitumoral activity

Transgene and partner BioInvent presented a poster on updated clinical results showing the positive antitumoral activity of BT-001 in patients with advanced refractory tumors at the ESMO (Free ESMO Whitepaper) Annual Meeting – see press release.

These updated data from the Phase 1 trial (NCT04725331) evaluating BT-001 in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy, KEYTRUDA (pembrolizumab)3 showed positive local, abscopal and sustained antitumoral activity in injected and non-injected lesions. Immune-mediated tumor shrinkage is consistent with the mechanistic hypothesis that BT-001, in combination with pembrolizumab, turns "cold" tumors into immunologically active or "hot" ones – see poster.

The data support further clinical development of BT-001.

Governance: recent additions to leadership team to further accelerate the development of the myvac platform

In April 2025, Simone Steiner joined Transgene as Chief Technical Officer (CTO) and member of the Executive committee.

In February 2026, Feriel Marin was appointed Chief Quality Officer ad interim. As of April 1, 2026, Sandrine Lemius will become Deputy CEO – Responsible Pharmacist ad interim.
They temporarily join the Executive committee following the retirement of Christophe Ancel, and will remain in these roles until the appointment of his successor.

In addition, in July 2025, the Board of Directors appointed a new independent director, Emmanuelle Quilès (see press release).

Key financial events

Business funded until early 2028, supporting key clinical milestones

In December 2025, Transgene completed a successful fundraising of circa €105 million and the conversion of its €39 million debt to TSGH into shares – see press release.

The net proceeds from the fundraising, combined with its existing cash, enables the acceleration of Transgene’s INTV myvac programs, and extends the Company’s financial visibility until early 2028.

Key financials for 2025

Operating revenue of €7.2 million in 2025 compared to €6.4 million in 2024.
Operating revenue was mostly comprised of the research tax credit (€6.7 million in 2025 compared to €6.0 million in 2024).

Net operating expenses of €42.3 million in 2025 compared to €42.0 million in 2024.
These reflect patient accrual in the ongoing Phase 2 part of the Phase 1/2 clinical trial of TG4050 in head and neck cancer. Research and development (R&D) expenses were at €33.9 million in 2025 versus €34.3 million in 2024. General and administrative expenses amounted to €7.3 million in 2025 versus €7.8 million in 2024.

Operating loss of €35.1 million in 2025, compared to an operating loss of €35.7 million in 2024.
Net loss of €37.5 million in 2025, compared to a net loss of €34.0 million in 2024.
Net cash burn of €38.2 million in 2025, compared to €27.7 million in 2024.
Cash, cash equivalents and other financial assets as of December 31, 2025: €111.9 million, compared to €16.7 million at the end of 2024, following the capital raise in December 2025.
Current and non-current liabilities of €17.1 million at the end 2025, compared to €27.0 million at the end of 2024, resulting from the conversion into shares of debt drawn down from the current account advance granted by the Company’s major shareholder TSGH for an amount of circa €39 million in December 2025. Following the completion of the Reserved Capital Increase, the current account advance agreement has been terminated.
Total shareholder’s equity of €121.7 million at the end of 2025, compared to €15.2 million at the end of 2024, resulting mainly from the completion of the capital increases in December 2025.
The financial statements for 2025 as well as management’s discussion and analysis are attached to this press release (appendices A and B).

The Board of Directors of Transgene met on March 24, 2026, under the chairmanship of Dr. Alessandro Riva and closed the 2025 financial statements. Audit procedures have been performed by the statutory auditors and the auditor’s reports are in the process of being issued.

The Company’s universal registration document (URD), which includes the annual financial report, will be available early April 2026.

A conference call in English is scheduled today on March 24, 2026, at 6:00 p.m. CET (1:00 p.m. ET).

Webcast link to English language conference call:
View Source

Please log in to the following link to obtain your personal telephone IDs:
View Source

A replay of the call will be available on the Transgene website following the live event.

Next financial communications:

April 29, 2026: First Quarter 2026 Financial Results
May 22, 2026: Annual Shareholders’ Meeting

(Press release, Transgene, MAR 24, 2026, View Source [SID1234663866])

On March 24, 2026 Peptomyc reported the closing of a €5 million equity financing to advance the next stage of development of OMO-103, the first direct MYC inhibitor with clinical validation. The financing was led by Alta Life Sciences and Aurora Science, with participation from new investors, the EIC Fund, part of the European Innovation Council, and Laudecum, as well as follow-on participation from existing investor, CDTI Innovación, through its SICC Innvierte.

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The proceeds from the financing will be used to advance OMO-103 toward its next development milestones, including the completion of ongoing clinical studies, continued progress on manufacturing readiness for later-stage development, and ongoing strategic partnering activities. The financing reflects continued support for Peptomyc’s progress.

"We are grateful for the support of our investors and for the confidence they have placed in Peptomyc as we advance OMO-103 into later-stage clinical development," said Jesús Martin Garcia, CEO of Peptomyc. "This financing provides us with the resources to execute on our ongoing studies, strengthen our development capabilities, and support ongoing discussions with potential pharmaceutical partners."

Montserrat Vendrell, Partner at Asabys Partners and Board Member at Peptomyc, commented: "Peptomyc represents an important opportunity in oncology. OMO-103 is the first and only direct MYC inhibitor that has demonstrated a favorable safety profile in clinical trials, with promising signs of biological and clinical activity. MYC is a key oncogene in approximately 70% of cancers, and Peptomyc’s ability to target this previously perceived ‘undruggable’ target opens new therapeutic possibilities. We are excited to continue supporting the team at this important stage of development."

OMO-103 is the first direct MYC inhibitor with clinical validation, having demonstrated a favorable safety profile, target engagement, and early signs of clinical activity. Peptomyc is currently advancing OMO-103 across three ongoing clinical studies, including a Phase 1b trial in metastatic pancreatic cancer, a Phase 2 trial in osteosarcoma, and a Window-of-Opportunity study in pancreatic cancer.

In parallel, Peptomyc is continuing discussions with potential pharmaceutical partners to explore collaborations aimed at accelerating the development of OMO-103. Together with ongoing clinical execution and manufacturing readiness activities, these discussions are intended to support the company’s progress toward the next stage of development for OMO-103.

(Press release, Peptomyc, MAR 24, 2026, View Source [SID1234663865])

On March 24, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it has entered into a securities purchase agreement with RA Capital Management for a private placement that is expected to result in gross proceeds of approximately $30 million before deducting placement agent fees and offering expenses, and an additional approximately $44 million of gross proceeds if the accompanying warrants are exercised in full.

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In the private placement, the Company agreed to sell 1,030,354 shares of common stock at a price of $6.785 per share, 3,391,164 pre-funded warrants at a price of $6.7849 per pre-funded warrant, and accompanying warrants to purchase 4,421,518 shares of common stock with an exercise price of $10.00 per share. The pre-funded warrants will have an exercise price of $0.0001 per share of common stock, will be immediately exercisable and will not expire. The accompanying warrants will be immediately exercisable and will expire 30 days following the public announcement by the Company of topline results from the Phase 3 XPORT-EC-042 clinical trial of selinexor in patients with endometrial cancer.

The private placement is expected to close on or about March 26, 2026, subject to the satisfaction of customary closing conditions. The private placement was priced at-the-market under Nasdaq rules. The Company expects that the net proceeds of the private placement, together with its existing liquidity, including cash, cash equivalents and investments, 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 Q3 2026.

The Company intends to use the proceeds from the private placement for general corporate purposes, including to support the Company’s ongoing and planned clinical trial activities.

Jefferies and Piper Sandler acted as placement agents for the private placement.

The offer and sale of the shares of common stock, pre-funded warrants, warrants, or any other securities (including the shares of common stock issuable upon exercise of the pre-funded warrants and warrants) are not being registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state securities laws. The shares of common stock, pre-funded warrants, warrants, or any other securities (including the shares of common stock issuable upon exercise of the pre-funded warrants and warrants) may not be offered or sold in the United States except pursuant to an exemption from the registration requirements of the Securities Act and any applicable state securities laws.

This press release does not constitute an offer to sell or the solicitation of an offer to buy shares of common stock, pre-funded warrants, warrants, or any other securities, nor shall there be any offer, solicitation or sale of shares of common stock, pre-funded warrants, warrants, or any other securities (including the shares of common stock issuable upon exercise of the pre-funded warrants and warrants) in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful.

(Press release, Karyopharm, MAR 24, 2026, View Source [SID1234663864])

On March 24, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported topline results from its Phase 3 SENTRY trial, a randomized, double-blind, placebo-controlled trial of 60 mg selinexor in combination with ruxolitinib in frontline myelofibrosis (n=353). The trial met the first co-primary endpoint, demonstrating statistically significant improvement in spleen volume reduction of 35% or more (SVR35) for patients treated with the combination of selinexor plus ruxolitinib, with rapid, deep and sustained spleen volume reduction rates seen in the combination arm. The mean change in absolute total symptom score (Abs-TSS) at week 24 relative to baseline was comparable across the two arms with similar symptom improvement relative to baseline; the difference across the two arms was not statistically significant. Importantly, the topline results suggest a promising signal in overall survival (OS) for the combination arm.

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Spleen Volume: 50% of patients who received the combination of selinexor plus ruxolitinib achieved a statistically significant improvement in SVR35 at week 24 compared to 28% of patients who received ruxolitinib alone (one-sided p<0.0001). Patients on the combination achieved rapid spleen reduction with 49% already achieving SVR35 at week 12 compared to 20% who received ruxolitinib alone. Spleen volume reduction was sustained, with 47% of patients on the combination achieving SVR35 at week 36 compared to 23% who received ruxolitinib alone.

Symptoms: Similar symptom improvement from baseline was observed in patients who received the combination of selinexor plus ruxolitinib compared to ruxolitinib alone as measured by Abs-TSS at week 24. Patients who received the combination reported a 9.89 point improvement in Abs-TSS compared to a 10.86 point improvement in patients who received ruxolitinib alone.

Overall Survival: Promising OS signal was observed in patients who received the combination of selinexor plus ruxolitinib compared to ruxolitinib alone with a hazard ratio of 0.43 (95% CI [0.19, 1.00] nominal one-sided p=0.0222). The Company intends to continue to follow OS to maturity to further evaluate this signal.

Overall Survival Associated with SVR35: Post-hoc landmark analyses at weeks 12 and 24 suggest SVR35 may predict overall survival.

Variant Allele Frequency (VAF) Reduction: Evidence of potential disease modification from a pre-specified exploratory endpoint was observed at week 24 from baseline in the combination arm as 32% of patients who received the combination achieved a ≥20% reduction in VAF for JAK2, MPL, and CALR compared to 24% of patients who received ruxolitinib alone (n=261).

Other Secondary and Exploratory Endpoints: Across other secondary and exploratory endpoints of progression-free survival, hemoglobin stabilization, and bone marrow fibrosis improvement, no meaningful difference was observed between the trial arms as of the data cut-off of February 20, 2026. The Company intends to further evaluate these endpoints as they mature.
Patients were randomized 2:1 to 60 mg of selinexor once weekly plus ruxolitinib or placebo plus ruxolitinib. The ruxolitinib dose was determined based on the patients’ baseline platelet count per the drug’s prescribing information. All data presented are as of the data cut-off of February 20, 2026.

"The results from SENTRY are an important development for patients as the combination of selinexor plus ruxolitinib meaningfully improved spleen response and we observed a promising signal in overall survival. Reducing spleen volume remains one of the most important treatment goals in myelofibrosis since achieving SVR35 is associated with improvement in overall survival," said Dr. John Mascarenhas, Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders. "While the symptom endpoint did not reach statistical significance, patients treated in both arms achieved similar symptom improvement relative to baseline. Importantly, while JAK inhibitors have been the backbone of therapy, continued progress requires new therapies that target additional biological pathways. Inhibition of XPO1 represents a differentiated mechanism that has the potential to address these pathways and evolve treatment beyond JAK inhibition alone."

"For patients with myelofibrosis, improvements in spleen and symptoms are expected outcomes from JAK inhibitors such as ruxolitinib. The SENTRY topline results suggest that the combination of selinexor and ruxolitinib delivers superior spleen reduction, which may predict overall survival, while offering similar symptom improvement, and may offer an important advance for our patients," said Dr. Claire Harrison, Professor of Myeloproliferative Neoplasms and Deputy Chief Medical Officer of Research, Data, and Analytics at Guy’s and St. Thomas’ NHS Foundation Trust in the United Kingdom.

"Selinexor’s differentiated mechanism provides a complementary approach to JAK inhibition and highlights the importance of targeting additional biological pathways beyond JAK signaling to further advance outcomes for patients with myelofibrosis. I am encouraged by the speed and magnitude of spleen response, and the promising overall survival signal and evidence of potential disease modification. In totality, these data underscore selinexor’s potential to meaningfully improve clinical outcomes for patients with myelofibrosis," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research of Karyopharm. "On behalf of Karyopharm, I would like to thank the patients, families, caregivers, investigators, and clinical trial team who participated in this trial. We are excited to share our results with regulatory authorities, key opinion leaders and patient advocacy organizations."

"The myelofibrosis community is waiting for new treatment options that can build upon the benefit of JAK inhibitors. Improving overall survival is the ultimate goal for people living with myelofibrosis and I am incredibly encouraged by these results," said Kapila Viges, Chief Executive Officer of the MPN Research Foundation. "These results are an exciting development for the myelofibrosis community."

Safety and Tolerability

The combination demonstrated a manageable safety and tolerability profile consistent with the known profile of selinexor and ruxolitinib individually. No new safety signals were observed.

The five most common all-grade treatment emergent adverse events (TEAEs) in the selinexor plus ruxolitinib arm were thrombocytopenia (selinexor plus ruxolitinib arm: 59%; placebo plus ruxolitinib arm: 43%), anemia (57%; 58%), nausea (57%; 17%), constipation (32%; 36%) and neutropenia (27%; 9%) (n=234; n=116). The rate of grade 3+ TEAEs was 70% in the selinexor plus ruxolitinib arm compared to 50% in the placebo plus ruxolitinib arm. The rate of TEAEs leading to treatment discontinuation was 15% in the selinexor plus ruxolitinib arm and 9% in the placebo plus ruxolitinib arm. The rate of confirmed leukemic transformations was the same across both arms of the trial at 1.7%.

Next Steps

The Company will be meeting with the U.S. Food and Drug Administration (FDA) to discuss the totality of the data from the SENTRY trial and its supplemental new drug application (sNDA) filing plan.

The Company plans to share additional data from the Phase 3 SENTRY trial at an upcoming medical meeting and expects to submit a manuscript to a peer-reviewed medical journal. The Company believes that potential inclusion in relevant compendia could occur in the second half of 2026.

Conference Call Information

Karyopharm will host a conference call today, March 24, 2026, at 8:00 a.m. Eastern Time, to discuss the results of its Phase 3 SENTRY trial in myelofibrosis. 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. 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 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 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 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).

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.

(Press release, Karyopharm, MAR 24, 2026, View Source,-Demonstrating-Statistically-Significant-Improvement-in-Spleen-Volume-Reduction [SID1234663863])

On March 24, 2026 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, reported its financial results for the fiscal year ended December 31, 2025 and provided a business update.

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"Our 2025 results validate that our strategy is clear and the foundation we built over the past year is now propelling Fennec into its next chapter of growth. We delivered record net product sales, achieved significant growth within our Fennec HEARS program, and advanced independent clinical evidence generation for PEDMARK – all while driving quarter-over-quarter growth in every quarter in 2025. These results demonstrate increasing PEDMARK adoption across key accounts and patient segments, effective field execution, and sustained progress across the organization," said Jeff Hackman, chief executive officer of Fennec Pharmaceuticals. "Concurrently, we strengthened our financial position through prudent operating decisions and strategic financial initiatives, including the closing of public and private offerings and the completion of full debt redemption."

Business Highlights:

● Continued Growth Within Key PEDMARK Accounts: Adoption continues to accelerate across new and existing accounts, including multiple Adolescent and Young Adult (AYA) patients across several tumor types receiving PEDMARK. Strong adoption trends reflect growing confidence in PEDMARK’s clinical value and reinforce its potential to help reshape the standard of care for patients receiving cisplatin-based treatment, demonstrating that the Company’s growth strategies are well aligned with market opportunities.

● Expanded Field Team to Accelerate Growth: In the fourth quarter, given the positive momentum Fennec observed over 2025, the Company made the strategic decision to further enhance its execution by increasing its customer facing team to achieve greater reach and frequency with its customers so the organization can ultimately help more cancer patients protect their hearing.

● New Real-World Data in Adults with Head and Neck Cancer (HNC): In February 2026, Fennec announced it presented the first new data since the pivotal clinical program at the 2026 Multidisciplinary Head and Neck Cancers Symposium (MHNCS). Findings supporting the potential use of PEDMARK in adults with head and neck cancers (HNC) were observed in a multi-institutional retrospective review of 15 adults with HNC. The data showed that PEDMARK could be safely given ≥ six hours after cisplatin dosing and was easy to incorporate into the real-world care plan for adults with HNC. This strict post-cisplatin timing is a validated approach intended to preserve cisplatin antitumor activity and no disruption to curative-intent cisplatin-based treatment delivery was observed as part of the study review.

● Initiation of Two Institution-Led Clinical Studies: In December 2025, Fennec announced that City of Hope, a U.S. cancer research and treatment organization, is evaluating PEDMARK for the prevention of cisplatin-induced ototoxicity (CIO) in adult men with stage II-III metastatic testicular germ cell tumors. In March 2026, Fennec announced that Tampa General Hospital (TGH) Cancer Institute is initiating a study evaluating the real-world clinical utility of PEDMARK in reducing the risk of ototoxicity in AYA and adult cancer patients receiving cisplatin-based treatment. Additional investigator-initiated studies supporting the use of PEDMARK in additional tumor types and patient populations, including AYA cancer, have been submitted to Fennec and are currently under review.

● STS-J01 in Japan: In December, Fennec announced positive topline results from the investigator-initiated Phase 2/3 STS-J01 clinical trial evaluating PEDMARK for the reduction of cisplatin-induced ototoxicity in pediatric and adolescent and young adult (AYA) patients with non-metastatic solid tumors in Japan. The results were from the first large-scale pediatric and adolescent and young adults (AYA) trial in Japan and demonstrated that PEDMARK can protect hearing without compromising cisplatin’s efficacy or introducing any concerning side effects. The Company is pursuing registration in Japan and is also exploring partnering or licensing opportunities for PEDMARK.

Upcoming Events:

● Piper Sandler Spring Biopharma Symposium: The management team will host one-on-one investor meetings at the annual Piper Sandler Spring Biopharma Symposium being held April 15–April 16, 2026 at the Convene One Boston Place.

Financial Results for the Fourth Quarter and Full Fiscal Year Ended December 31, 2025

● Net Product Sales – For the fourth quarter of 2025, the Company recorded net product sales of $13.8 million compared to $7.9 million in the fourth quarter of 2024, representing an increase of approximately 75%. For the full fiscal year (FY) 2025, the Company recorded net product sales of approximately $44.6 million compared to $29.6 million in 2024, representing an increase of approximately 50%. The increase in net product sales is attributable to growth across both new and existing accounts with notable success in conversion and adherence of PEDMARK patients.
● Selling and Marketing Expenses – The Company recorded $6.1 million in selling and marketing expenses in the fourth quarter of 2025 compared to $3.9 million in the fourth quarter of 2024. The increase in selling and marketing expenses is largely related to increased payroll and additional marketing expenses as we focused on expanding our commercial team and preparing for additional outreach to community oncology centers and the adolescent and young adult (AYA) population. For the FY 2025, the Company recorded $18.6 million in selling and marketing compared to $18.4 million in fiscal year 2024. The year- over-year slight increase is largely related to increased payroll and marketing expenses in the comparable period offset by the elimination of European expenses after the announcement of the Norgine transaction in March 2024.

● General and Administrative (G&A) Expenses – The Company recorded $8.9 million in G&A expenses fourth quarter of 2025 compared to $4.2 million in the fourth quarter of 2024. For the FY 2025, the Company recorded $28.8 million in G&A expenses compared to $23.1 million in fiscal year 2024. G&A expenses increased in both the comparable quarterly and fiscal years due to increased intellectual property-related legal expenses, increased payroll expenses as headcount increased and increased non-cash expenses associated with equity-based remuneration.

● Cash Position – Cash and cash equivalents were $36.7 million as of December 31, 2025. For the FY 2025, there was a $10.2 million increase in cash and cash equivalents between December 31, 2024 and December 31, 2025. The net increase in cash was primarily due to the approximately $42.0 million in net proceeds from equity offerings and net cash collected from net product sales offset by operating expenses and the $21.5 million debt paydown in November of 2025. As of December 31, 2025 the company had $0 in debt outstanding.

Fourth Quarter and Full-Year 2025 Conference Call Information

Date:Tuesday, March 24, 2026

Time:8:30 a.m. Eastern Time

Webcast Link: View Source

Participant Link: View Source

1Financial Update
The selected financial data presented below is derived from our unaudited condensed consolidated financial statements, which were prepared in accordance with U.S. generally accepted accounting principles. The complete unaudited condensed consolidated financial statements for the period ended December 31, 2025, and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.

About Cisplatin-Induced Ototoxicity

Cisplatin and other platinum-based chemotherapies are widely used to treat solid tumors and have been vital in improving survival rates. Unfortunately, these life-saving treatments often result in permanent, irreversible hearing loss, also known as ototoxicity.i

Hearing loss from cisplatin treatment is not rare. Studies show that between 60-90% of patients treated with cisplatin may develop hearing loss, depending upon the dose and duration of chemotherapy.ii Many of those treated with cisplatin will require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.iii Treatment-induced hearing loss can reduce quality of survivorship as it impacts many aspects of life, such as speech and language skills, academic performance, social-emotional development, career potential and the ability to live independently.iv,v While audiologic monitoring is recommended to help manage ototoxicity, it is currently underutilized in certain cancer patient populations.

PEDMARK (sodium thiosulfate injection)

PEDMARK is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients 1 month of age and older with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children’s Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

Additionally, PEDMARK is recommended for the adolescent and young adult (AYA) population by the National Comprehensive Cancer Network, or NCCN, with a 2A endorsement.

Approximately 500,000 patients in the U.S. are diagnosed annually with cancers that could be treated with a platinum-based chemotherapy.vi,vii The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of those treated will require lifelong hearing aids. Until the FDA approval of PEDMARK, there were no preventative agents for this hearing loss. Patients with hearing loss resulting from cancer treatment have a statistically significant worse quality of life compared with peers who have no hearing loss.viii,ix

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage
PEDMARK (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use
The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information
PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor

serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

Please see full Prescribing Information for PEDMARK at: www.PEDMARK.com.

(Press release, Fennec Pharmaceuticals, MAR 24, 2026, View Source [SID1234663862])