A Phase I/II Study of Natalizumab as a Single Agent in Children, Adolescents and Young Adults With Recurrent, Refractory or Progressive Pulmonary Metastatic Osteosarcoma

The purpose of this study is to evaluate the safety and tolerability of Natalizumab in children, adolescent and young adult patients with pulmonary metastatic osteosarcoma (pOS) and to assess clinical response associated with this treatment as well as overall survival.

Start Date01 Dec 2024

Sponsor / Collaborator

The Case Comprehensive Cancer Center

NCT05658497

/ RecruitingNot Applicable

Vumerity (Diroximel Fumarate) Prospective MS Pregnancy Exposure Registry

The primary objectives of the study are to estimate the risk of major congenital malformations (MCMs) in infants born to women with multiple sclerosis (MS) who were exposed to diroximel fumarate (DRF) at any time from 2 weeks after the first day of their last menstrual period (LMP) up through the first trimester of pregnancy and to comparatively evaluate pregnancy outcomes with MCMs in women with MS who were exposed to DRF at any time from 2 weeks after the first day of their LMP through the first trimester of pregnancy with the following: i) women with MS who were unexposed to disease modifying therapies (DMTs) and, ii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries).
The secondary objective of the study is to evaluate pregnancy outcomes in women with DRF exposure at any time from 2 weeks after the first day of their LMP through the end of pregnancy compared with the following: i) women with MS who were unexposed to DMTs, ii) women with dimethyl fumarate (DMF) exposure, iii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries), and iv) women without MS (e.g., women from external, general population comparators).

Start Date27 Oct 2023

Sponsor / Collaborator

Biogen, Inc.

NCT05618301

/ CompletedPhase 1IIT

A Pilot Safety and Feasibility Study to Evaluate Motixafortide (CXCR4/SDF-1 Inhibition) and Natalizumab (VLA-4/VCAM-1 Inhibition) as a Novel Regimen to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)

Hematopoietic stem cell (HSC)-based gene therapies now offer curative potential for patients with sickle cell disease (SCD), with decreased toxicity compared to allogeneic hematopoietic cell transplantation. However, effective HSC-based gene therapy depends on collecting sufficient HSCs to generate the therapeutic product, and currently available mobilization regimens carry unacceptable risk for patients with SCD or do not reliably yield optimal numbers of HSCs for gene therapy.
The investigators hypothesize that HSC mobilization with motixafortide (CXCR4i) alone and the combination of motixafortide plus natalizumab (VLA-4i) will be safe and tolerable in SCD patients. In addition, the investigators hypothesize that combined CXCR4 and VLA-4 blockade with motixafortide plus natalizumab will result in a rapid, robust, and synergistic increase in HSC mobilization to peripheral blood (PB) in patients with SCD, when compared to motixafortide alone.

Start Date07 Jul 2023

Sponsor / Collaborator

Washington University School of Medicine

[+3]

100 Clinical Results associated with Natalizumab

100 Translational Medicine associated with Natalizumab

100 Patents (Medical) associated with Natalizumab

2,927

Literatures (Medical) associated with Natalizumab

01 Nov 2026·JOURNAL OF NEUROIMMUNOLOGY

Chitinase-1 in cerebrospinal fluid and white matter injury in patients with progressive multiple sclerosis

Article

Author: von Essen, M Rode ; Romme Christensen, J ; Buhelt, S ; Talbot, J ; Siebner, H R ; Lundell, H ; Ratzer, R ; Sellebjerg, F ; Mahler, M R ; Holm Hansen, R ; Kosa, P ; Højsgaard Chow, H ; Bielekova, B

OBJECTIVE:

In patients with progressive multiple sclerosis (PMS), previous studies reported increased cerebrospinal fluid (CSF) concentrations of inflammatory biomarkers, but these correlated only weakly with structural damage. We aimed to identify inflammatory CSF biomarkers that associate with tissue damage in PMS.

METHODS:

We performed four substudies: [1] A cross-sectional exploratory study of patients with primary (PPMS) or secondary progressive MS (SPMS) (n = 38) in whom we explored correlations between CSF concentrations of 1128 proteins and CSF neurofilament light chain (NFL) and myelin basic protein. [2] A cross-sectional confirmatory study where a protein of main interest, chitinase-1 (CHIT1), was analyzed in 104 patients with PPMS (n = 78) or SPMS (n = 26) and compared with 38 symptomatic controls. [3] Associations between CSF concentrations of CHIT1 and other disease biomarkers including CSF- and magnetic resonance imaging (MRI)-based measures of white matter injury in patients with PPMS (n = 59). [4] A longitudinal study of effects of treatment with methylprednisolone, natalizumab, dimethyl fumarate, or placebo on CHIT1 in CSF in patients with PMS.

RESULTS:

Substudy [1] identified three proteins that correlated with CSF NFL: soluble B-cell maturation antigen, CC chemokine ligand 22, and CHIT1. CHIT1 showed a strong correlation with CSF NFL (ρ = 0.61, q = 0.008) and was selected for further analyses. Substudy [2] showed that patients with progressive MS had higher CSF concentrations of CHIT1 than symptomatic controls (all p < 0.001). In substudy [3], CSF concentrations of CHIT1 correlated with increased lesion volume (p < 0.001) and decreased magnetization transfer ratio (p = 0.001) of lesions, decreased fractional anisotropy (p < 0.001) and increased mean diffusivity of normal-appearing white matter (p = 0.044) and lesions (p = 0.005) in patients with PPMS. Substudy [4] showed that natalizumab treatment reduced CSF CHIT1 concentrations (p = 0.005) in PMS.

CONCLUSION:

CSF CHIT1 concentrations are associated with neuroaxonal and white matter injury in patients with PPMS and responsive to disease-modifying therapy in PMS.

01 Oct 2026·CLINICAL NEUROLOGY AND NEUROSURGERY

Longitudinal evolution of JC virus serological index in multiple sclerosis patients treated with natalizumab: Observational dynamics in a decade-spanning real-world cohort (2014–2024)

Article

Author: Sánchez-Rosales, Nayeli A ; Frías-Márquez, Francisco Javier ; Valdivia-Tangarife, Edgar R ; Rodríguez-Madera, Miriam Gisela ; Rodríguez-Ramírez, Larissa ; Mireles-Ramírez, Mario Alberto ; Verboonen-Salgado, Brenda Allison ; Contreras-Haro, Betsabe ; Marquez-Pedroza, Jazmin ; Torres-Mendoza, Blanca Miriam

BACKGROUND:

The anti-JC virus (JCV) antibody index is an established biomarker for estimating the risk of progressive multifocal leukoencephalopathy (PML) in patients with multiple sclerosis (MS) treated with natalizumab. However, long-term real-world data regarding the stability of JCV serostatus, particularly from Latin American populations, remain limited.

OBJECTIVE:

To evaluate longitudinal changes in the anti-JC virus (JCV) antibody index and their association with clinical and radiological outcomes in patients with relapsing-remitting multiple sclerosis (RRMS) treated with natalizumab during a 10-year follow-up in a Mexican public health tertiary center.

METHODS:

We conducted a retrospective observational cohort study including 104 RRMS patients treated with natalizumab between 2014 and 2024 at a single tertiary center in Guadalajara, Mexico. Anti-JCV antibody index was measured using the STRATIFY JCV™ DxSelect ELISA assay. Longitudinal serological patterns were categorized as persistent positive, persistent negative, seroconversion, indeterminate-to-positive transition, or seroreversion. Clinical and radiological outcomes were compared between treatment-naïve and previously treated patients.

RESULTS:

At baseline, 49 patients (47.1%) were treatment-naïve, and 55 (52.9%) had prior exposure to disease-modifying therapy. Overall, 42 patients (40.4%) were JCV seropositive, 49 (47.1%) seronegative, and 13 (12.5%) indeterminate. Median duration of natalizumab exposure was 5.2 years (IQR 3.1-7.4), and the median number of anti-JCV antibody determinations per patient was 8 (range 2-18). Persistent seropositivity (51.9%) and persistent seronegativity (37.5%) were the predominant longitudinal patterns. Seroconversion occurred in 6.7% of patients overall (4.1% naïve, 8.9% non-naïve), indeterminate-to-positive transition in 3.8%, and seroreversion in 1.0% of patients. No cases of progressive multifocal leukoencephalopathy were observed. Prior exposure to disease-modifying therapy, age at natalizumab initiation, and baseline disability were not associated with longitudinal serological evolution. Treatment-naïve patients exhibited fewer relapses (p = 0.0026), lower MRI activity (p = 0.03), and higher NEDA-3 rates (64.6% vs 32.7%, p = 0.002), whereas long-term disability outcomes did not differ significantly between groups.

CONCLUSIONS:

In this decade-spanning real-world cohort from a Latin American public health setting, anti-JCV antibody status remained predominantly stable during prolonged natalizumab therapy, confirming patterns previously reported in other populations. These findings add regional real-world evidence to the existing literature and support continued longitudinal serological monitoring. Early treatment initiation improved inflammatory disease control but did not significantly modify long-term disability trajectories.

01 Sep 2026·JOURNAL OF PHARMACOLOGICAL SCIENCES

Human blood–brain barrier spheroid models for the evaluation of drugs targeting brain vascular inflammation

Article

Author: Kunitomo, Furano ; Ebisawa, Ayuka ; Furihata, Tomomi ; Watanabe, Hiromu ; Morio, Hanae ; Rutkowska, Aleksandra ; Minasato, Asuka

The blood-brain barrier (BBB), primarily formed by brain microvascular endothelial cells (BMECs), restricts substance entry into the brain. BBB inflammation, involving barrier dysfunction and immune cell recruitment, contributes to neurological diseases and represents a therapeutic target. In this study, we characterized human immortalized cell-based multicellular spheroidal BBB (hiMCS-BBB) models as a tool for evaluating effects of BBB inflammation-targeted drugs. First, we examined the responses of the hiMCS-BBB models to the pro-inflammatory cytokine (TNF-α and IFN-γ) exposure. The cytokine treatment reduced claudin-5 protein expression levels at the BBB, accompanied by a 1.4 ± 0.1-fold increase in lucifer yellow permeability. Consistently, E-selectin protein expression was upregulated, accompanied by a 22.1-fold increase in THP-1 cell adhesion to the BBB. Then, we tested whether natalizumab (a monoclonal antibody used for multiple sclerosis treatment) and JPH203 (an inhibitor of L-type amino acid transporter 1) could modulate the immune cell adhesion. Both agents remarkably suppressed the cytokine-induced THP-1 cell adhesion to 0.2-fold relative to the respective controls. To summarize, our results demonstrate that the hiMCS-BBB models exhibit key features of BBB inflammation in response to inflammation stimuli and recapitulate clinically relevant responses to anti-inflammatory drugs, highlighting their potential to accelerate BBB-targeting drug development.

227

News (Medical) associated with Natalizumab

24 Aug 2026

·www.biospace.com

AEON Biopharma Designates Former FDA Neuroscience Leader Paul Lee, M.D., Ph.D. as Acting Chief Regulatory Officer

- Former Deputy Director of the FDA’s Office of Neuroscience - Dr. Lee has previously advised AEON through Clintrex Research LLC (Clintrex), an affiliate of BlueRidge Life Sciences, on the regulatory strategy for ABP-450 ALISO VIEJO, Calif., Aug. 24, 2026 (GLOBE NEWSWIRE) -- AEON Biopharma, Inc. (“AEON” or the “Company”) (NYSE American: AEON), a biopharmaceutical company advancing ABP-450 as a biosimilar to BOTOX ® (onabotulinumtoxinA) for therapeutic use to achieve full-label U.S. market entry, today announced the designation of Paul Lee, M.D., Ph.D. as the Company’s acting Chief Regulatory Officer, through Clintrex, strengthening the Company's regulatory leadership. Dr. Lee and Clintrex currently advise AEON on the regulatory and clinical development strategy for ABP-450, providing strategic guidance as the Company advances its biosimilar development program. In his acting Chief Regulatory Officer role, Dr. Lee will lead AEON’s regulatory strategy, help guide the Company’s interactions with the FDA and work closely with AEON’s analytical, CMC and clinical teams as it advances ABP-450 under the 351(k) biosimilar pathway. “Dr. Lee’s expanded role reflects our continued focus on strengthening the regulatory capabilities needed to successfully advance the ABP-450 biosimilar development program,” commented Rob Bancroft, President and Chief Executive Officer of AEON. “His senior FDA neuroscience leadership experience, direct experience with biosimilar science and detailed understanding of ABP-450 through his work with AEON and Clintrex, make him exceptionally well positioned to guide our regulatory strategy.” Dr. Lee spent more than seven years at the FDA’s Office of New Drugs, most recently serving as Deputy Director of the Office of Neuroscience from May 2024 to June 2025 and as Director of the Division of Neurology 2 from March 2023 to May 2025. In these roles, he oversaw the review and regulation of drug and biologic programs across a broad range of neurological and psychiatric conditions, including migraine, epilepsy, stroke, multiple sclerosis and neurodegenerative diseases. Dr. Lee also recently co-authored a publication describing the FDA review and approval of Tyruko ® (natalizumab-sztn), the first FDA-approved biosimilar to natalizumab. Before joining the FDA, Dr. Lee was a staff clinician at the National Human Genome Research Institute, where he served as an investigator in rare-disease clinical trials and as a reviewing physician for the Undiagnosed Diseases Network. Dr. Lee earned his M.D. and Ph.D. in Neuroscience from the University of Maryland and is board certified in neurology with special qualification in child neurology. Dr. Lee said, “I am pleased to take on this expanded role and work even more closely with the AEON team to advance a scientifically rigorous biosimilar development program. I look forward to bringing my regulatory, neuroscience and biosimilar experience to that work.” About the U.S. Biosimilar Pathway The 351(k) biosimilar pathway is grounded in a totality-of-the-evidence approach, with comparative analytical assessment providing the scientific foundation for establishing biosimilarity. The nature and extent of additional studies are informed by the totality of the evidence and any residual uncertainty remaining following comparative analytical assessment. Once biosimilarity is established, FDA may approve a biosimilar for additional indications of the reference product through scientifically justified extrapolation, without requiring separate clinical studies in each indication. About AEON Biopharma AEON Biopharma is a biopharmaceutical company pursuing full-label access to the U.S. therapeutic neurotoxin market via biosimilarity to BOTOX ® . The U.S. therapeutic neurotoxin market exceeds $3.0 billion annually, representing a major opportunity for biosimilar entry. ABP-450 is the same botulinum toxin complex currently approved and marketed for cosmetic indications by Evolus, Inc. under the name Jeuveau ® . ABP-450 is manufactured by Daewoong Pharmaceutical in a facility that has been authorized by the U.S. Food and Drug Administration, Health Canada, and European Medicines Agency for the manufacture of third-party botulinum toxin products. AEON has exclusive development and distribution rights for therapeutic indications of ABP-450 in the United States, Canada, the European Union, the United Kingdom, and certain other international territories. To learn more about AEON, visit . Forward-Looking Statements The foregoing material may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Forward-looking statements include all statements that do not relate solely to historical or current facts, including, without limitation, statements regarding the anticipated benefits of Dr. Lee’s appointment and his expected contributions to the Company; the timing, objectives and potential outcomes of the Company’s anticipated interactions with the U.S. Food and Drug Administration (“FDA”); the Company’s ability to obtain FDA feedback on, or alignment regarding, the remaining clinical and regulatory development program for ABP-450; the timing and advancement of the Company’s planned clinical development activities; the Company’s plans to advance ABP-450 as a proposed biosimilar to BOTOX ® (onabotulinumtoxinA) under the Section 351(k) biosimilar pathway; and the Company’s product development and business prospects. Forward-looking statements can be identified by the use of words such as “may,” “will,” “expect,” “project,” “estimate,” “anticipate,” “plan,” “believe,” “potential,” “should,” “continue,” “seek,” or the negative versions of those words or other comparable words. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements are based on information currently available to the Company and its current plans or expectations and are subject to a number of risks and uncertainties that could significantly affect current plans. Should one or more of these risks or uncertainties materialize, or the underlying assumptions prove incorrect, actual results may differ significantly from those anticipated, believed, estimated, expected, intended or planned. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, the Company cannot guarantee future results, performance or achievements. Except as required by applicable law, including the securities laws of the United States, the Company does not intend to update any of the forward-looking statements to conform these statements to actual results. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (i) whether the anticipated benefits of Dr. Lee’s designation are realized and whether he is able to contribute to the Company’s regulatory and development activities as anticipated; (ii) the Company’s ability to obtain additional and sufficient financing; (iii) the Company’s anticipated financial performance, including its cash and cash equivalents; (iv) the timing and outcome of the Company’s interactions with the FDA, including whether the FDA agrees with the Company’s proposed regulatory, analytical, clinical development and extrapolation strategies for ABP-450; (v) the possibility that the FDA may require additional analytical, nonclinical or clinical studies or otherwise modify the Company’s anticipated development plans; and (vi) other risks and uncertainties set forth in the sections entitled “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” in the Company’s filings with the Securities and Exchange Commission, which are available on the SEC’s website at Contacts Investor Contact: Hershel Berry Blueprint Life Science Group hberry@Bplifescience.com Source: AEON Biopharma

Executive ChangeDrug Approval

13 Aug 2026

·www.biospace.com

Zealand Pharma enters into a USD 100 million royalty purchase and sale agreement with Royalty Pharma for the economics related to rusfertide

Company announcement – No. 38 / 2026 Zealand Pharma enters into a USD 100 million royalty purchase and sale agreement with Royalty Pharma for the economics related to rusfertide Zealand Pharma will receive USD 100 million for the sale of its economic interests related to rusfertide, including royalty rights on the potential future global net sales of rusfertide. The agreement unlocks value from a non-core royalty entitlement for redeployment to Zealand Pharma’s key strategic priorities. Copenhagen, Denmark, August 12, 2026 – Zealand Pharma A/S (Nasdaq: ZEAL) (CVR-no. 20045078), a biotechnology company transforming the future of metabolic health, today announces that it has entered into a royalty purchase and sale agreement with Royalty Pharma plc (Nasdaq: RPRX) in exchange for Zealand Pharma’s economics related to rusfertide (PTG-300), a potential first-in-class therapy for polycythemia vera, a rare and chronic blood disorder. Under the terms of the royalty purchase and sale agreement, Zealand Pharma will receive USD 100 million for the sale of its economic interests related to rusfertide, including rights to one percent royalty on potential future global net sales of rusfertide and regulatory and commercial milestones. Of the USD 100 million, Zealand Pharma will receive an upfront payment of USD 50 million upon closing of the transaction. The remaining USD 50 million is due upon the first anniversary of the closing of the agreement. Zealand Pharma will retain the right to a 0.25% royalty on annual net global sales of rusfertide greater than USD 1.5 billion, while Royalty Pharma will retain a 0.75% royalty on sales above USD 1.5 billion. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); “ We are very pleased to work with Royalty Pharma, a leading funder of life science innovation, on this strategic financial transaction,” said Henriette Wennicke, Chief Financial Officer at Zealand Pharma. “ This agreement converts a future potential royalty stream into immediate capital that will be redeployed against future growth opportunities in line with our key strategic priorities under our Metabolic Frontier 2030 strategy. ” “ We are delighted to expand our relationship with Zealand Pharma through a second collaboration, supporting the company as it continues to advance its innovative pipeline of medicines for obesity and metabolic health ,” said Pablo Legorreta, Chief Executive Officer and Chairman of the Board of Royalty Pharma. “ This transaction underscores Royalty Pharma’s role as a long-term, trusted partner to biopharma companies as they advance important medicines. Furthermore, we believe rusfertide has the potential to become a transformative treatment option for patients with polycythemia vera, and we look forward to its continued progress through regulatory review .” The U.S. FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for the New Drug Application (NDA) for rusfertide for the treatment of adults with polycythemia vera in the third quarter of 2026 and Takeda will be responsible for global commercialization 1 . In June 2012, Zealand Pharma and Protagonist Therapeutics, Inc., entered into a research collaboration agreement to develop disulfide-rich peptides (DRPs). Under the agreement, Zealand Pharma was responsible for preclinical and clinical drug development of DRPs discovered under the collaboration. The research collaboration was terminated in 2014 and Protagonist’s payment obligations to Zealand Pharma were clarified in a settlement agreement reached in 2021. In January 2024, Protagonist and Takeda entered into a worldwide license and collaboration agreement for rusfertide. The purchase and sale agreement closed today, August 12, 2026. Goodwin acted as legal advisor to Zealand Pharma. Covington, Dechert, Jones Day and Kromann acted as legal advisors to Royalty Pharma. About rusfertide 1 Rusfertide is a first-in-class investigational subcutaneous treatment that mimics the action of hepcidin, a natural hormone that regulates iron homeostasis and red blood cell production. By targeting the underlying mechanism of iron dysregulation in polycythemia vera, rusfertide aims to reduce excess red blood cell production and help patients achieve sustained hematocrit control. Rusfertide is administered once weekly via subcutaneous self-injection and has been generally well-tolerated in clinical trials to date. About Zealand Pharma Zealand Pharma A/S (Nasdaq: ZEAL) is a biotechnology company focused on advancing medicines for obesity and metabolic health. Combining more than 25 years of peptide R&D expertise with a proprietary data platform that leverages advanced data driven and AI/ML approaches, Zealand Pharma aims to lead a new era in obesity and metabolic health. To date, more than ten Zealand Pharma invented drug candidates have entered clinical development, of which two products have reached the market and three candidates are in late-stage development. The Company has collaborations with global pharmaceutical and biotechnology partners for research, development, and commercialization. Founded in 1998, Zealand Pharma is headquartered in Copenhagen, Denmark, with a U.S. presence in Boston, Massachusetts. Learn more at . About Royalty Pharma plc Founded in 1996, Royalty Pharma is the largest buyer of biopharmaceutical royalties and a leading funder of innovation across the biopharmaceutical industry, collaborating with innovators from academic institutions, research hospitals and non-profits through small and mid-cap biotechnology companies to leading global pharmaceutical companies. Royalty Pharma has assembled a portfolio of royalties which entitles it to payments based directly on the top-line sales of many of the industry’s leading therapies. Royalty Pharma funds innovation in the biopharmaceutical industry both directly and indirectly – directly when it partners with companies to co fund late-stage clinical trials and new product launches in exchange for future royalties, and indirectly when it acquires existing royalties from the original innovators. Royalty Pharma’s current portfolio includes royalties on more than 35 commercial products, including Vertex’s Trikafta and Alyftrek, GSK’s Trelegy, Roche’s Evrysdi, Johnson & Johnson’s Tremfya, Biogen’s Tysabri and Spinraza, Servier’s Voranigo, AbbVie and Johnson & Johnson’s Imbruvica, Astellas and Pfizer’s Xtandi, Pfizer’s Nurtec ODT, and Gilead’s Trodelvy, and 20 development-stage product candidates. For more information, visit . Forward looking statements This company announcement contains “forward-looking statements”, as that term is defined in the Private Securities Litigation Reform Act of 1995 in the United States, as amended, even though no longer listed in the United States this is used as a definition to provide Zealand Pharma’s expectations or forecasts of future events regarding the research, development, and commercialization of pharmaceutical products, the timing of the company’s clinical trials and the reporting of data therefrom and the company’s significant events and potential catalysts in 2026 and financial guidance for 2026. These forward-looking statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would”, and other words and terms of similar meaning. You should not place undue reliance on these statements, or the scientific data presented. The reader is cautioned not to rely on these forward-looking statements. Such forward-looking statements are subject to risks, uncertainties and inaccurate assumptions, which may cause actual results to differ materially from expectations set forth herein and may cause any or all of such forward-looking statements to be incorrect, and which include, but are not limited to, unexpected costs or delays in clinical trials and other development activities due to adverse safety events or otherwise; unexpected concerns that may arise from additional data, analysis or results obtained during clinical trials; our ability to successfully market both new and existing products; changes in reimbursement rules and governmental laws and related interpretation thereof; government-mandated or market-driven price decreases for our products; introduction of competing products; production problems; unexpected growth in costs and expenses; our ability to effect the strategic reorganization of our businesses in the manner planned; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies, or may reject, fail to approve or may delay approval of our drug candidates or expansion of product labelling; failure to obtain regulatory approvals in other jurisdictions; exposure to product liability and other claims; interest rate and currency exchange rate fluctuations; unexpected contract breaches or terminations; inflationary pressures on the global economy; and political uncertainty. If any or all of such forward-looking statements prove to be incorrect, our actual results could differ materially and adversely from those anticipated or implied by such statements. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. All such forward-looking statements speak only as of the date of this press release/company announcement and are based on information available to Zealand Pharma as of the date of this release/announcement. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical advice. Zealand Pharma® is a registered trademark of Zealand Pharma A/S. Contacts Eric Rojas (Investors) Vice President, Head of Investor Relations Zealand Pharma Email: erojas@zealandpharma.com Neshat Anis Ahmadi (Investors) Investor Relations Manager Zealand Pharma Email: neahmadi@zealandpharma.com Rachel James-Owens (Media) Vice President, Corporate Communications & Media Relations Zealand Pharma Email: rjamesowens@zealandpharma.com Andreas Hylleberg Mølleskov (Media) Director, External Communications Zealand Pharma Email: ahylleberg@zealandpharma.com References 1. Takeda and Protagonist Announce U.S. Food and Drug Administration Accepts New Drug Application and Grants Priority Review for Rusfertide as a Potential First-in-Class Therapy for Polycythemia Vera. Takeda Pharmaceutical Company Limited. FDA Accepts NDA and Grants Priority Review for Rusfertide . Last accessed August 2026.

License out/inNDAPriority Review

11 Aug 2026

·www.prnewswire.com

Immunic, Inc. Reports Second Quarter 2026 Financial Results and Provides Corporate Update

– Leadership Team Expanded with Key Executive Appointments, Including Chief Executive Officer, Chief Medical Officer and Chair of the Board of Directors, to Support Clinical Development, as well as NDA and Commercial Readiness – – Pivotal Phase 3 ENSURE Trials of Vidofludimus Calcium in Relapsing Multiple Sclerosis on Track, with Top-Line Data Expected by End of 2026 – – Continued Preparations for Phase 3 Trial of Vidofludimus Calcium in Progressive Multiple Sclerosis, with Initiation Planned Later This Year – NEW YORK, Aug. 11, 2026 /PRNewswire/ -- Immunic , Inc. (Nasdaq: IMUX), a late-stage biotechnology company pioneering the development of novel oral therapies for neurologic diseases, today reported financial results for the second quarter ended June 30, 2026, and provided a corporate update. "During the second quarter, we continued to execute across our late-stage development priorities as we advance toward what we believe will be a transformational period for Immunic," said Erik Lundgren, Chief Executive Officer of Immunic. "As we prepare for the next stage of Immunic's growth, we have continued to strengthen our board and team with world-class multiple sclerosis (MS) experts whose extensive development, medical, regulatory and commercialization expertise will be instrumental as we execute on our clinical strategy and prepare for the potential regulatory filings and commercialization of vidofludimus calcium (IMU-838). Following the successful closing of our oversubscribed private placement financing earlier this year, we are well capitalized to execute on our key strategic priorities. We believe the milestones ahead position Immunic for a significant near-term value inflection for vidofludimus calcium in relapsing MS, while continuing to unlock the substantial long-term opportunity across the broader progressive MS patient population." Jason Tardio, President and Chief Operating Officer of Immunic, commented, "We remain highly encouraged by the consistent and differentiated profile of vidofludimus calcium, supported by additional data from our Phase 2 CALLIPER trial in progressive MS presented at this year's CMSC Annual Meeting, including favorable safety, tolerability, patient-reported, and exploratory efficacy data. Taken together, these findings reinforce our confidence in the potential of vidofludimus calcium to offer a differentiated benefit/risk profile through its novel dual mechanism of action, combining Nurr1 activation with selective DHODH inhibition. We believe this approach has the potential to deliver meaningful efficacy while maintaining the favorable safety and tolerability profile observed to date, positioning vidofludimus calcium as a potentially important new oral treatment option for people living with MS." "Our Phase 3 ENSURE trials of vidofludimus calcium in relapsing MS remain on track to report top-line data by the end of 2026," added Michael A. Panzara, M.D., M.P.H., Chief Medical Officer of Immunic. "With regulatory alignment and precedent surrounding the time to first relapse primary endpoint, the path to regulatory submission upon positive trial results is clear. In parallel, we are preparing to initiate our Phase 3 progressive MS trial later this year, reinforcing our commitment to evaluating the potential of vidofludimus calcium for anyone living with MS." Second Quarter 2026 and Subsequent Highlights Ongoing Phase 3 ENSURE Relapsing MS Program: Continued to execute the twin Phase 3 ENSURE-1 and ENSURE-2 trials of vidofludimus calcium in relapsing MS. Preparation of Phase 3 Progressive MS Program: Continued preparations for a confirmatory Phase 3 program in progressive MS, building on the Phase 2 CALLIPER trial data. Additional Phase 2 CALLIPER Data Presented at CMSC Annual Meeting: Presented one late-breaking and two additional posters of new data from the Phase 2 CALLIPER trial of vidofludimus calcium in progressive MS at the 2026 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting. The new analyses, which included a novel unified confirmed disability change (CDC) endpoint, up to 120 weeks of patient-reported outcomes, and safety and tolerability data, further reinforced the favorable profile of vidofludimus calcium and its potential to address underlying drivers of disability progression in progressive MS. Expansions in Management and Board of Directors: Appointment of Erik Lundgren as Chief Executive Officer, effective May 22, 2026, with employment having begun on June 1, 2026. Mr. Lundgren is a biopharmaceutical executive with nearly two decades of commercial leadership experience, including senior roles at Genentech and Roche supporting the launch of Ocrevus® for relapsing and primary progressive MS. On July 5, 2026, Mr. Lundgren was also appointed to serve as a Director of the Board. Appointment of Michael A. Panzara, M.D., M.P.H., as Chief Medical Officer, effective April 24, 2026. Dr. Panzara brings over 25 years of global neurology experience and proven leadership in advancing transformational therapies through development and regulatory approval processes. Dr. Panzara oversaw the global regulatory approvals of the MS drugs Lemtrada® and Aubagio® during his tenure at Sanofi Genzyme. During his time at Biogen, he served as global clinical lead for the development of Tysabri® and managed the late-stage MS portfolio. In addition, Michael W. Bonney has been appointed as Chair of the Board of Directors, effective May 16, 2026. Mr. Bonney is a seasoned biopharmaceutical executive with more than three decades of leadership experience, including senior commercial roles at Biogen tied to the launch and growth of Avonex® for relapsing MS and CEO experience at Cubist Pharmaceuticals. Simona Skerjanec, M.Pharm, M.B.A., transitioned from Interim Chairperson to continue serving as a Board member. Appointment of Elena Ridloff to the Board of Directors, effective August 6, 2026. Ms. Ridloff brings more than 20 years of leadership experience in finance, corporate development, investor relations, and capital markets across the biopharmaceutical industry. She currently serves as Chief Financial Officer at Sionna Therapeutics. Anticipated Clinical Milestones Vidofludimus Calcium in Relapsing MS: Top-line data from the twin Phase 3 ENSURE-1 and ENSURE-2 trials remains on track and is expected to be available by the end of 2026. If positive, Immunic plans to submit a New Drug Application (NDA) in the United States in mid-2027, with a targeted potential regulatory approval date in 2028. Vidofludimus Calcium in Progressive MS: The planned initiation of a Phase 3 program in progressive MS remains on track for later this year. IMU-381: The IMU-381 program, selected to leverage the Nurr1 platform for neurologic and autoimmune diseases, continues to be in preclinical testing with lead candidate identification ongoing. Financial and Operating Results Total Operating Expenses for the three and six months ended June 30, 2026 were $35.3 million and $68.6 million, respectively. Included in these results are $7.2 million and $11.7 million, respectively, of non-cash charges, primarily related to stock compensation expense. Research and Development (R&D) Expenses were $25.9 million for the three months ended June 30, 2026, as compared to $21.3 million for the three months ended June 30, 2025. The $4.6 million increase reflects (i) a $4.4 million increase in personnel expenses, $3.2 million of which was related to non-cash stock compensation, (ii) a $1.6 million increase due to drug-drug interaction studies in support of a potential NDA filing, (iii) a $1.1 million increase related to costs across numerous categories, partially offset by (iv) a $2.0 million decrease related to the CALLIPER clinical trial and (v) a $0.5 million decrease related to the ENSURE clinical trials. For the six months ended June 30, 2026, R&D expenses were $51.6 million, as compared to $42.9 million for the six months ended June 30, 2025. The $8.7 million increase reflects (i) a $5.4 million increase in personnel expenses, $3.8 million of which was related to non-cash stock compensation, (ii) a $3.9 million increase due to drug-drug interaction studies in support of a potential NDA filing, (iii) a $3.1 million increase for vidofludimus calcium drug supply and (iv) a $1.3 million increase related to costs across numerous categories, partially offset by (v) a $2.8 million decrease related to the CALLIPER clinical trial and (vi) a $2.2 million decrease related to the ENSURE clinical trials. General and Administrative (G&A) Expenses were $9.4 million for the three months ended June 30, 2026, as compared to $5.7 million for the same period ended June 30, 2025. The $3.7 million increase was due to (i) a $2.1 million increase related to personnel expenses, of which $1.8 million was related to non-cash stock compensation, (ii) a $0.7 million increase in legal and consultancy expenses, (iii) a $0.4 million increase in pre-commercial marketing expenses, and (iv) a $0.5 million increase in related costs across numerous categories. For the six months ended June 30, 2026, G&A expenses were $17.0 million, as compared to $11.0 million for the same period ended June 30, 2025. The $6.0 million increase was due to (i) a $4.2 million increase related to personnel expenses, of which $3.6 million was related to non-cash stock compensation, (ii) a $1.0 million increase in legal and consultancy expenses, (iii) a $0.6 million increase in pre-commercial marketing expenses, and (iv) a $0.2 million increase in related costs across numerous categories. Interest Income was $1.3 million for the three months ended June 30, 2026, as compared to $0.3 million for the three months ended June 30, 2025. The $1.0 million increase was due to a higher average cash balance as a result of the February 2026 Private Placement. For the six months ended June 30, 2026, interest income was $2.0 million, as compared to $0.4 million for the same period ended June 30, 2025. The $1.6 million increase was due to a higher average cash balance as a result of the February 2026 Private Placement. Other Income (Expense) was ($0.06) million for the three months ended June 30, 2026, as compared to $0.02 million for the same period ended June 30, 2025. For the six months ended June 30, 2026, Other Income (Expense) was ($0.2 million), as compared to $1.2 million for the same period ended June 30, 2025. The $1.4 million decrease was primarily attributable to (i) a $1.0 million grant income of the German Federal Ministry of Finance recognized in the first quarter 2025 and no grant income in 2026 and (ii) a $0.4 million decrease across various categories. Net Loss for the three months ended June 30, 2026, was approximately $34.1 million, or $0.80 per basic and diluted share, based on 42,513,839 weighted-average common shares outstanding, compared to a net loss of approximately $26.8 million, or $2.03 per basic and diluted share, based on 13,217,520 weighted-average common shares outstanding for the same period ended June 30, 2025. Net loss for the six months ended June 30, 2026, was approximately $66.7 million, or $1.83 per basic and diluted share, based on 36,359,274 weighted average common shares outstanding, compared to a net loss of approximately $52.3 million or $4.48 per basic and diluted share, based on 11,684,499 weighted average common shares outstanding for the same period ended June 30, 2025. Cash and Cash Equivalents as of June 30, 2026 were $155.1 million. With these funds, Immunic expects to be able to fund its operations into late 2027. About Immunic, Inc. Immunic, Inc. (Nasdaq: IMUX) is a late-stage biotechnology company pioneering the development of novel oral therapies for neurologic diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently being evaluated in Phase 3 clinical trials for the treatment of relapsing multiple sclerosis, with top-line data expected to be available by the end of 2026. Initiation of an additional Phase 3 clinical trial in progressive MS is expected later in 2026. Vidofludimus calcium has already shown therapeutic potential and a favorable safety and tolerability profile in Phase 2 clinical trials in relapsing-remitting multiple sclerosis, progressive multiple sclerosis and other diseases. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor-related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). The company's development pipeline also includes earlier-stage programs, including IMU-381 and IMU-856, aimed at building a broader therapeutics platform addressing neurodegenerative and autoimmune diseases. For further information, please visit: . Cautionary Statement Regarding Forward-Looking Statements This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, future financial position, future revenue, projected expenses, sufficiency of cash and cash runway, expected timing, development and results of clinical trials, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to Immunic's development programs and the targeted diseases; the potential for Immunic's development programs to safely and effectively target diseases; preclinical and clinical data for Immunic's development programs; the feasibility of advancing vidofludimus calcium to a confirmatory Phase 3 clinical trial in progressive multiple sclerosis; the timing of current and future clinical trials, anticipated clinical milestones and regulatory approvals; the nature, strategy and focus of the company and further updates with respect thereto; the development and commercial potential of any product candidates of the company; expectations regarding the capitalization, resources and ownership structure of the company; and the executive and board structure of the company. Immunic may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve substantial risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, increasing inflation, tariffs and macroeconomics trends, impacts of the Ukraine – Russia conflict and the conflict in the Middle East on planned and ongoing clinical trials, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient financial and other resources to meet business objectives and operational requirements, the fact that the results of earlier preclinical studies and clinical trials may not be predictive of future clinical trial results, any changes to the size of the target markets for the company's products or product candidates, the protection and market exclusivity provided by Immunic's intellectual property, risks related to the drug development and the regulatory approval process and the impact of competitive products and technological changes. A further list and descriptions of these risks, uncertainties and other factors can be found in the section captioned "Risk Factors," in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2025, filed with the SEC on February 26, 2026, and in the company's subsequent filings with the SEC. Copies of these filings are available online at or ir.imux.com/sec-filings. Any forward-looking statement made in this release speaks only as of the date of this release. Immunic disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. Immunic expressly disclaims all liability in respect to actions taken or not taken based on any or all of the contents of this press release. Contact Information Immunic, Inc. Jessica Breu Vice President Investor Relations and Communications +49 89 2080 477 09 [email protected] US IR Contact LifeSci Advisors Joyce Allaire [email protected] US Media Contact Real Chemistry [email protected] Financials SOURCE Immunic, Inc. 21% more press release views with Request a Demo

Phase 2Financial StatementExecutive ChangePhase 3

100 Deals associated with Natalizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D06886	Natalizumab	L04AA23	DB00108

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Crohn Disease	United States	Biogen, Inc.	14 Jan 2008
Multiple Sclerosis, Relapsing-Remitting	European Union	BIOGEN NETHERLANDS BV	27 Jun 2006
Multiple Sclerosis, Relapsing-Remitting	Iceland	BIOGEN NETHERLANDS BV	27 Jun 2006
Multiple Sclerosis, Relapsing-Remitting	Liechtenstein	BIOGEN NETHERLANDS BV	27 Jun 2006
Multiple Sclerosis, Relapsing-Remitting	Norway	BIOGEN NETHERLANDS BV	27 Jun 2006
Multiple Sclerosis	United States	Biogen, Inc.	23 Nov 2004

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Multiple Sclerosis, Secondary Progressive	Phase 3	United States	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Belgium	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Canada	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Czechia	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Denmark	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Finland	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	France	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Germany	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Ireland	Biogen, Inc.	13 Sep 2011
Multiple Sclerosis, Secondary Progressive	Phase 3	Israel	Biogen, Inc.	13 Sep 2011

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00493298 (TOP, Pubmed \| J Neurol Neurosurg Psychiatry)	Not Applicable	Multiple Sclerosis, Relapsing-Remitting	6,319	Natalizumab	hpdcjzmeua(tbsrwodeug) = ottkapqthq pptaubquth (hsllainuji ) View more	Positive	16 Apr 2026
NCT04048577 (Pubmed \| Ann Clin Transl Neurol)	Phase 2	Multiple Sclerosis	10	Natalizumab	icmlcnynpb(naqhlajxjx) = gikwzgqzqb oxeyescxnp (kqgqjxvnjj ) View more	Positive	01 Jan 2026
NCT05265728 (CTgov)	Phase 3	Multiple Sclerosis, Relapsing-Remitting	21	Natalizumab	wwuzfqjvxw(htxymgrehq) = dxuouadspm eytatgrlgq (ywnrwrdgmr, 0.60) View more	-	25 Nov 2025
EAN2025	Not Applicable	Multiple Sclerosis, Relapsing-Remitting	167	Natalizumab	jxqorstnsi(fgvyunjsbe) = kiwekvqocy rqqnkyxqvb (yjagopawhh ) View more	Positive	21 Jun 2025
EAN2025	Not Applicable	Multiple Sclerosis neurofilament light chain	125	low-efficacy DMTs (leDMTs)	iiwpzylchc(gwcrwonnre) = fkabqpvfbi uuqjnpifhd (xelafwpemh ) View more	Positive	21 Jun 2025
				natalizumab EID	lqhhfepvzf(btgqhvvhgf) = dfjgidwuku texaxnlima (csxzrwwqmk )
EAN2025	Not Applicable	Multiple Sclerosis	805	Platform therapies	auiowrpjaq(iqlqrcqtzo) = spgldolthi fsrkhjsyjy (vxzjjtbfqj ) View more	Positive	21 Jun 2025
				Intermediary active drugs	auiowrpjaq(iqlqrcqtzo) = ylryzckboc fsrkhjsyjy (vxzjjtbfqj ) View more
P8-1.016 (AAN2025)	Not Applicable	Multiple Sclerosis anti-CD20s \| natalizumab	188	Anti-CD20 therapy (Ocrelizumab)	mmtiwtfgox(cxcuoqjgej): rate ratio = 0.81 (95% CI, 0.48 - 1.36), P-Value = 0.42 View more	Positive	07 Apr 2025
				Natalizumab (TYSABRI)
NCT03689972 (NOVA, Pubmed \| Neurol Neuroimmunol Neuroinflamm)	Phase 3	Multiple Sclerosis, Relapsing-Remitting trough serum natalizumab concentration \| α4-integrin saturation	486	Natalizumab Q6W dosing	cxomyffmzw(yqgxnpyffw) = aqkjiwhhpw rbfgwewrev (vpaiiftlwg )	Positive	01 Nov 2024
				Natalizumab Q4W dosing	cxomyffmzw(yqgxnpyffw) = scacjcwcjo rbfgwewrev (vpaiiftlwg )
NCT03689972 (NOVA, CTgov)	Phase 3	Multiple Sclerosis, Relapsing-Remitting	585	Natalizumab (Part 1: IV Q4W)	vsksceidcf(ojqfjiammc) = qlbethgmvb danwfzpcuy (akncjgnhnm, oxgxdcfvzc - mjjixosxmr) View more	-	12 Jun 2024
				Natalizumab (Part 1: IV Q6W)	vsksceidcf(ojqfjiammc) = udbeugclpv danwfzpcuy (akncjgnhnm, sydjhqcusq - vtzqhxacfy) View more
S31.010 (AAN2024)	Not Applicable	Multiple Sclerosis	930	Fingolimod	gyearbrwcq(unzbtjwsoc) = ahwaetwilm rvpqvyypwe (iqtkrymehd )	Negative	09 Apr 2024
				Natalizumab	gyearbrwcq(unzbtjwsoc) = goonksljfg rvpqvyypwe (iqtkrymehd )

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