On March 24, 2026 Ferring Pharmaceuticals reported that the U.S. Food and Drug Administration (FDA) has approved a label update for ADSTILADRIN (nadofaragene firadenovec-vncg), enabling an accelerated water-bath thawing method, completed in about 25 minutes, to enhance efficient clinical preparation for healthcare teams.

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ADSTILADRIN is the first and only FDA-approved non-replicating intravesical gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). In the United States, bladder cancer is the sixth most common cancer, with NMIBC comprising about 75% of all new cases.1,2

"The reduction in thawing time for ADSTILADRIN streamlines preparation and handling for healthcare providers treating patients with high-risk non-muscle invasive bladder cancer," said Vikram M. Narayan, MD, FACS, Associate Professor at Emory University Department of Urology, Chief of Urology at Grady Memorial Hospital. "This updated preparation process has the potential to save valuable time and enable healthcare providers and practices to deliver this treatment option to more patients efficiently."

ADSTILADRIN is shipped and stored as a sterile frozen suspension and must be brought to room temperature (20° C to 25° C [68° F to 77° F]) prior to use. The FDA’s approval is supported by a thawing and handling study demonstrating that frozen vials remained stable when thawed for about 25 minutes in a water bath maintained at 25°C (77°F). The storage conditions for ADSTILADRIN remain unchanged once thawing begins and permit storage for up to 24 hours at room temperature or up to 7 days refrigerated (2-8°C), including thaw time.

"At Ferring, we remain deeply committed to evolving our therapies in ways that address the real-world needs of patients and the healthcare providers who care for them," said Denise D’Andrea, MD, FACP, Senior Director, Medical Affairs Uro-Oncology, Ferring Pharmaceuticals. "ADSTILADRIN already offers convenient, once-every-three-months dosing that helps reduce visit frequency, travel burden and clinic workflow demands. The new 25 minutes at 25°C thaw option provides additional flexibility and represents another important step in our ongoing efforts to streamline operational workflow across care settings."

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall.3 In the United States, bladder cancer is the sixth most common cancer,1 fourth among men,4 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025.4 Historically, 75% of bladder cancer presents as NMIBC.2 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care; however, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease.5 Current treatment options for BCG-unresponsive patients are very limited and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).6

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC, who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849 and final five-year follow-up analysis published in The Journal of Urology).7-8

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions; including, laboratory abnormalities (>15%), were increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition (urination urgency), increased creatinine, hematuria (blood in urine), decreased phosphate, chills, pyrexia (fever) and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit View Source or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.

(Press release, Ferring Pharmaceuticals, MAR 24, 2026, View Source [SID1234663881])

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 Mosaic Therapeutics, Ltd, (‘Mosaic’, or ‘the Company’) a clinical-stage oncology therapeutics company building the category leader in Synergistic Precision Oncology, reported it will hold a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026, taking place April 17-22 in San Diego, CA.

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Poster Presentation Details:

Title: "Combination of the MDM2 antagonist ASTX295 and olaparib as a novel treatment option for BRCA2 mutant, TP53 wild-type solid tumors"
Abstract Number: 3053
Date and Time: Monday, April 20, 2026, 2:00PM – 5:00PM PST
Session Category: Novel Targets and Pathways
Session: PO.ET09.09
Location: Poster Section 15

For more information and to view the Company’s abstract, visit the AACR (Free AACR Whitepaper) Annual Meeting website.

About ASTX295

ASTX295 is a potent, highly selective, potentially best-in-class MDM2 antagonist that has completed a phase I study (NCT03975387) in over 100 patients with advanced solid tumors. Rationally designed to exhibit a short half-life, ASTX295 has demonstrated a compelling safety profile with minimal hematological toxicities, enabling use as a combination therapy.

MDM2 is a clinically validated target for cancer therapy, frequently overexpressed across multiple tumor types. As the primary negative regulator of the p53 tumor suppressor protein, MDM2 inhibition leads to p53 stabilization and re-activation, inducing cell cycle arrest and apoptosis.

Mosaic Therapeutics announced the in-licensing of ASTX295, alongside ASTX029 (an ERK1/2 inhibitor), from Astex Pharmaceuticals in April 2025, and holds full development and commercialization rights.

About MOS101

Plans for a Phase 1b/2a study evaluating the combination of ASTX295 with olaparib (an approved PARP inhibitor) for patients with biomarker-selected solid tumors are being finalized.

(Press release, Mosaic Therapeutics, MAR 24, 2026, View Source [SID1234663882])

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 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])