IntraBio Announces Submission of Supplemental New Drug Application for Levacetylleucine for Ataxia-Telangiectasia




IntraBio Announces Submission of Supplemental New Drug Application for Levacetylleucine for Ataxia-Telangiectasia

AUSTIN, Texas–(BUSINESS WIRE)–IntraBio Inc. today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), a rare, progressive, inherited neurodegenerative disorder.

This submission represents the first regulatory application submitted to the U.S. Food and Drug Administration seeking approval of a therapy for the treatment of Ataxia-Telangiectasia.

The sNDA seeks to expand the label of levacetylleucine, marketed as AQNEURSA®, to include A-T. AQNEURSA® is currently approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.

The submission is supported by data from a pivotal Phase III clinical trial evaluating levacetylleucine in adult and pediatric patients with A-T. In this randomized, double-blind, placebo-controlled, crossover study, levacetylleucine met its primary endpoint and key secondary endpoints with high statistical significance. Levacetylleucine was generally safe and well-tolerated, with no drug-related serious adverse events observed, consistent with its established safety profile.

About Ataxia-Telangiectasia

Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. It is characterized by degeneration of the cerebellum, resulting in worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, lung disease, and a markedly increased risk of cancer. There are currently no approved therapies for A-T.

About AQNEURSA®

U.S Indication

AQNEURSA® (levacetylleucine) is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.

U.S. IMPORTANT SAFETY INFORMATION

Embryo-Fetal Toxicity

• Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.

Pregnancy and Lactation

• For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.

• There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.

Adverse Reactions

• The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.

Drug Interactions

• Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.

• Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.

To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please click here for Full US Prescribing Information for AQNEURSA: https://www.aqneursahcp.com/wp-content/prescribing-information.pdf

EMA Indication

AQNEURSA® is authorized in the European Union for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat. EMA Indication and Important Safety Information

About IntraBio

IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading universities and institutions worldwide, including the University of Oxford and the University of Munich.

Contacts

For further information, please contact:
Cass Fields

Vice-President of External Affairs

ccfields@intrabio.com
www.intrabio.com

Qiagen Announces Form 20-F Annual Report Filing for 2025 Results




Qiagen Announces Form 20-F Annual Report Filing for 2025 Results

VENLO, the Netherlands–(BUSINESS WIRE)–$QGEN #QIAGEN–QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) announced today that it has filed its annual report, including its audited consolidated financial statements on Form 20-F, for the year ended December 31, 2025, with the U.S. Securities and Exchange Commission. The document can be accessed on QIAGEN’s website here.

QIAGEN will provide printed copies of the 2025 Annual Report to shareholders free of charge upon request. To obtain a printed copy of the 2025 Annual Report please use our contact form or send an email to ir@qiagen.com.

About QIAGEN

QIAGEN N.V., a Netherlands-based holding company, is a global leader in Sample to Insight solutions that enable customers to extract and analyze molecular information from biological samples containing the building blocks of life. Our Sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies prepare these biomolecules for analysis, while bioinformatics support the interpretation of complex data to deliver actionable insights. Automation solutions integrate these steps into streamlined, cost-effective workflows. QIAGEN serves more than 500,000 customers worldwide in the Life Sciences (academia, pharmaceutical R&D and industrial applications such as forensics) and molecular diagnostics (clinical healthcare). As of December 31, 2025, QIAGEN employed approximately 5,700 people across more than 35 locations. For more information, visit www.qiagen.com.

Source: QIAGEN N.V.

Category: Corporate

Contacts

Investor Relations
e-mail: ir@QIAGEN.com

Public Relations
e-mail: pr@QIAGEN.com

KalVista Pharmaceuticals to Present New EKTERLY® (sebetralstat) Data at the 2026 Global Angioedema Leadership Conference




KalVista Pharmaceuticals to Present New EKTERLY® (sebetralstat) Data at the 2026 Global Angioedema Leadership Conference

New interim data from the KONFIDENT-KID trial evaluating sebetralstat in children aged 2-11 accepted for late-breaking oral presentation

FRAMINGHAM, Mass. & SALISBURY, England–(BUSINESS WIRE)–KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that five abstracts, including one late-breaking submission, have been accepted for presentation at the 2026 Global Angioedema Leadership Conference taking place in Madrid, Spain from March 26–29, 2026.

The following late-breaking abstract will be presented during the poster session on Friday, March 27 and as an oral presentation on Saturday, March 28 at 11:50 am CET:

• On-demand Oral Sebetralstat for Hereditary Angioedema Attacks in Children Aged 2-11: Interim Analysis of KONFIDENT-KID: Emel Aygören-Pürsün, Adil Adatia, John Anderson, Shira Benor, Mélisande Bourgoin-Heck, Mauro Cancian, Bob Geng, Eisuke Inage, Aharon Kessel, Majed Koleilat, H. Henry Li, Michael E. Manning, Heloise Reumaux, Raffi Tachdjian, Paola Triggianese, Andrea Zanichelli, Erik Hansen, Ya-Hsiu Chuang, Matthew Iverson, Michael D. Smith, Paul K. Audhya, H. James Wedner.

The following poster presentations will take place during the poster session on Friday, March 27, beginning at 6:10 pm CET:

• Satisfaction with Sebetralstat for HAE Attacks in Patients Switching from Parenteral On-demand Treatments in KONFIDENT-S: Mauro Cancian, Laurence Bouillet, Teresa Caballero, Tariq El-Shanawany, Sorena Kiani-Alikhan, Markus Magerl, Michael E. Manning, Inmaculada Martinez-Saguer, Maeve E. O’Connor, Sinisa Savic, Daniel F. Soteres, Maria Staevska, James Hao, Paolo Bajcic, Paul Audhya, Raffi Tachdjian.
• On-demand Treatment Patterns of Hereditary Angioedema Attacks with Sebetralstat in the KONFIDENT-S Study: Mar Guilarte, Emel Aygören-Pürsün, Jonathan A. Bernstein, Danny M. Cohn, Vesna Grivcheva-Panovska, Tamar Kinaciyan, Damia Leguevaques, William R. Lumry, Michael E. Manning, Daniel F. Soteres, Petra Staubach, Raffi Tachdjian, Marcin Stobiecki, Marc A. Riedl, Anna Valerieva, Patrick F. K. Yong, Andrea Zanichelli, James Hao, Michael D. Smith, Paul K. Audhya, Henriette Farkas.
• Barriers to Treatment of Hereditary Angioedema Attacks Among Patients Using Subcutaneous On-Demand Therapies: Insights from an International Patient Survey: Thomas Buttgereit, Isabelle Boccon-Gibod, Alexis Bocquet, Laurence Bouillet, Paula Busse, Sandra Christiansen, Timothy Craig, Tariq El-Shanawany, Tomaz Garcez, Padmalal Gurugama, Rashmi Jain, Sorena Kiani-Alikhan, Markus Magerl, Inmaculada Martinez-Saguer, Maeve O’Connor, Cristine Radojicic, Sinisa Savic, Paola Triggianese, H. James Wedner, Patrick Yong, Andrea Zanichelli, Sherry Danese, Julie Ulloa, Paolo Bajcic, Paul K. Audhya, Mauro Cancian.
• Anxiety in Patients Using Injectable On-Demand Treatments for Hereditary Angioedema Attacks: Results from an International Patient Survey: Alexis Bocquet, Isabelle Boccon-Gibod, Laurence Bouillet, Paula Busse, Thomas Buttgereit, Sandra Christiansen, Timothy Craig, Tariq El-Shanawany, Tomaz Garcez, Padmalal Gurugama, Rashmi Jain, Sorena Kiani-Alikhan, Markus Magerl, Inmaculada Martinez-Saguer, Maeve O’Connor, Cristine Radojicic, Sinisa Savic, Paola Triggianese, H. James Wedner, Patrick Yong, Andrea Zanichelli, Sherry Danese, Julie Ulloa, Paolo Bajcic, Paul K. Audhya, Mauro Cancian.

Links to all presentations will be available on the KalVista website under Publications.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity.

About EKTERLY® (sebetralstat)

EKTERLY (sebetralstat) is a novel plasma kallikrein inhibitor approved in the United States, European Union, United Kingdom, Switzerland, Australia, Singapore and Japan for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older. EKTERLY is the first and only oral on-demand treatment for HAE, offering efficacious and safe treatment of attacks without the burden of injections. With a US regulatory filing planned for 2026 to expand use to children aged 2–11, and additional filings anticipated in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide. For more information, including the full US Prescribing Information, visit EKTERLY.com.

About KalVista Pharmaceuticals, Inc.

KalVista is a global pharmaceutical company dedicated to delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. The KalVista team discovered and developed EKTERLY®—the first and only oral on-demand treatment for hereditary angioedema (HAE)—and continues to work closely with the global HAE community to improve treatment and care for this disease around the world. For more information about KalVista, please visit www.kalvista.com and follow us on LinkedIn, X, Facebook and Instagram.

Forward-Looking Statements

This press release contains “forward-looking” statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “anticipate,” “intend,” “plan,” “goal,” “seek,” “believe,” “project,” “estimate,” “expect,” “position,” “strategy,” “future,” “likely,” “may,” “should,” “will” and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, our expectations about the safety and efficacy of sebetralstat, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2025, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

Contacts

Ryan Baker

Head, Investor Relations

(617) 771-5001

ryan.baker@kalvista.com

Molly Cameron

Senior Director, Corporate Affairs

(978) 339-3378

molly.cameron@kalvista.com

Fauna Bio Announces Target Designation Milestone in Obesity Discovery Collaboration




Fauna Bio Announces Target Designation Milestone in Obesity Discovery Collaboration

Achievement marks advancement of novel obesity target identified through Convergence™ AI platform

EMERYVILLE, Calif.–(BUSINESS WIRE)–Fauna Bio, a biotechnology company pioneering the use of extreme mammal biology and comparative genomics to discover novel therapeutic targets, today announced the designation of a target under its strategic obesity discovery collaboration with Eli Lilly and Company (Lilly), initiated in December, 2023.

The designated target, identified using Fauna Bio’s proprietary Convergence™ AI platform, represents a potential first-in-class approach to treating obesity by leveraging insights from hibernating mammals that naturally regulate body weight and metabolism. This milestone triggers a payment to Fauna Bio under the terms of the collaboration agreement,.

“This target designation validates the power of our Convergence™ platform to identify truly novel biology in metabolic disease,” said Ashley Zehnder, Ph.D., Chief Executive Officer and co-founder of Fauna Bio. “By studying how hibernating mammals achieve remarkable feats of metabolic regulation—including dramatic, healthy weight cycling—we are uncovering therapeutic targets that have eluded traditional discovery approaches.”

The Convergence™ AI platform analyzes genomic data from over 450 mammal species, including more than 60 hibernators, integrating this comparative biology with human patient data to identify disease-relevant targets. The platform’s proprietary Graph Neural Network enables rapid evaluation and prioritization of novel targets across multiple therapeutic modalities.

“We are pleased to advance this collaboration into its next phase,” continued Zehnder. “This achievement reflects the deep scientific expertise of both teams and reinforces the value of our unique approach to drug discovery.”

About Fauna Bio

Fauna Bio is a biotechnology company improving human health by leveraging animal genomics and extreme mammal biology. The company’s proprietary Convergence™ platform integrates comparative genomic analyses across hundreds of mammal species with human disease data to identify novel therapeutic targets and develop transformative medicines. By studying animals that naturally resist disease—including hibernators that exhibit remarkable metabolic resilience—Fauna Bio translates these discoveries into innovative therapies for patients in need. The company is based in Emeryville, California. For more information, visit www.faunabio.com.

Contacts

Direct media inquiries to:

(510) 545-3945

media@faunabio.com

Innate Pharma Announces Conference Call and Webcast for Full Year 2025 Financial Results




Innate Pharma Announces Conference Call and Webcast for Full Year 2025 Financial Results

MARSEILLE, France–(BUSINESS WIRE)–#Biotech–Regulatory News:

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”), today announces that the Company will hold a conference call on Thursday, March 26, 2026 at 2 p.m. CET / 9 a.m. EDT, following the release of its financial results for the full year ending December 31, 2025.

Participants during the call will be:

• Jonathan Dickinson, Chief Executive Officer
• Sonia Quaratino, Executive Vice President, Chief Medical Officer
• Yannis Morel, Executive Vice President, Chief Operating Officer
• Stéphanie Cornen, Vice President, Investor Relations, Communication and Commercial Strategy
• Frédéric Lombard, Senior Vice President, Chief Financial Officer

Details for the Virtual Event

The live webcast will be available at the following link:

https://events.q4inc.com/attendee/704730270

Participants may also join via telephone using the following registration link: https://events.q4inc.com/analyst/704730270?pwd=usHLLD39

This information can also be found on the Investors section of the Innate Pharma website, www.innate-pharma.com. A replay of the webcast will be available on the Company website for 90 days following the event.

About Innate Pharma

Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Leveraging its expertise on antibody-engineering and innovative target identification, Innate Pharma is developing innovative and differentiated next-generation antibody therapeutics.

Innate Pharma is advancing a portfolio of differentiated potential first and/or best-in-class assets, focused on areas of high unmet medical need, including IPH4502, a differentiated Nectin-4 ADC developed in solid tumors, lacutamab, an anti-KIR3DL2 antibody developed in cutaneous T cell lymphomas and peripheral T cell lymphomas, and monalizumab, an anti-NKG2A antibody developed in collaboration with AstraZeneca in non-small cell lung cancer.

Innate Pharma has established collaborations with leading biopharmaceutical companies, including Sanofi and AstraZeneca, as well as renowned academic and research institutions, to advance innovation in immuno-oncology.

Headquartered in Marseille, France with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq in the US.

Learn more about Innate Pharma at www.innate-pharma.com and follow us on LinkedIn and X.

Information about Innate Pharma shares

Disclaimer on forward-looking information and risk factors

For a discussion of risks and uncertainties, please refer to the Risk Factors (“Facteurs de Risque”) section of the Universal Registration Document filed with the French Financial Markets Authority (“AMF”), which is available on the AMF website http://www.amf-france.org or on Innate Pharma’s website, and public filings and reports filed with the U.S. Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 20-F for the year ended December 31, 2024, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public by the Company. References to the Company’s website and the AMF website are included for information only and the content contained therein, or that can be accessed through them, are not incorporated by reference into, and do not constitute a part of, this press release.

This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in Innate Pharma in any country.

Contacts

For additional information, please contact:
Investors & Media Relations
Innate Pharma
Stéphanie Cornen

stephanie.cornen@innate-pharma.fr

Investor Relations
investors@innate-pharma.fr

Media
communication@innate-pharma.fr

Additional Phase 2 Data of AL-S Pharma’s Lead Program AP-101 Further Demonstrate Clinically Meaningful Disease Modification and Prolonged Survival in ALS




Additional Phase 2 Data of AL-S Pharma’s Lead Program AP-101 Further Demonstrate Clinically Meaningful Disease Modification and Prolonged Survival in ALS

Results supported by key neuroaxonal injury biomarkers pNfH and NfL after six months of treatment

AP-101 is an investigational human-derived antibody therapeutic that selectively targets the misfolded, toxic form of SOD1 to disrupt progressive spread of ALS

Data featured in oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases

Preparations underway to advance AP-101 into confirmatory Phase 3 clinical trial with initiation aimed for late 2026

ZURICH–(BUSINESS WIRE)–AL-S Pharma AG, a clinical-stage biotechnology company discovering and developing human antibodies that target misfolded proteins implicated in amyotrophic lateral sclerosis (ALS), today announced the presentation of new clinical data from the global Phase 2 clinical trial (AP-101-02) evaluating the company’s lead program, AP-101, in patients with ALS. AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1). AP-101 is designed to inhibit the spread of SOD1 pathology in the central nervous system of people living with ALS, helping the body’s immune system clear these harmful proteins.¹

The global Phase 2 clinical trial met its primary endpoint related to safety and tolerability. New results provide additional evidence of clinically meaningful disease modification and prolonged survival supported by reductions in key neuroaxonal injury biomarkers, serum neurofilament light chain (NfL) and cerebrospinal fluid phosphorylated neurofilament heavy chain (pNfH) after six months of treatment. Adverse events were comparable to placebo, and no AP-101-induced antibody responses were reported.

The AP-101-02 clinical trial results will be featured in an oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders. The presentation will be delivered by Angela Genge, M.D., chief medical officer of AL-S Pharma, today at 8:40AM CET in Hall A2 (Session 5210).

“These data show something we rarely see in ALS – objective evidence of clinically meaningful disease modification that tracks directly with prolonged survival,” said Dr. Genge. “The Phase 2 study results with AP-101 are consistent with the hypothesis that targeting misfolded SOD1 is a disease-modifying approach in ALS.² At AL-S Pharma, we believe AP-101 could fundamentally transform the treatment of ALS. We look forward to continue advancing the AP-101 clinical program so that we can bring a much-needed new treatment option to people living with ALS rapidly.”

The prespecified analysis of an exploratory composite endpoint revealed that early treatment with AP-101 prolonged survival and delayed ventilatory support in comparison to study participants receiving placebo for six months followed by six months of treatment with AP-101. Positive treatment effects were observed in both the sporadic ALS cohort (p = 0.013) and the SOD1 mutation carrier cohort (p = 0.036). Effects on survival were accompanied by disease stabilization as measured by King’s staging. Functional decline measured by ALSFRS-R was reduced in study participants with elevated misfolded SOD1 at baseline and in SOD1 mutation carriers.

AL-S Pharma is currently preparing for a confirmatory Phase 3 clinical trial of AP-101 in ALS aimed to initiate end of 2026. AP-101 received Orphan Drug designations from the U.S. Food and Drug Administration, the European Medicines Agency, and Swissmedic.

About AP-101-02

The AP-101-02 clinical trial (NCT05039099) was a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the safety, tolerability, pharmacodynamic markers, and pharmacokinetics (PK) of AP-101 in 73 patients with sporadic ALS and in patients with mutations in the superoxide dismutase 1 (SOD1) gene. Study participants with sporadic (n=52) or mutant SOD1 ALS (n=21) were stratified and randomized 2:1 to intravenous AP-101 or placebo every three weeks. Key assessments included survival and ventilation endpoints, slow vital capacity, neurofilament levels, misfolded SOD1, PK, and anti-drug antibodies. After 24 weeks all participants entered a 24-week open-label extension with continued AP-101 treatment, followed by a 16-week safety follow-up period.

AP-101-02 was conducted in the U.S., Canada, the U.K., European Union, and South Korea. More information about the clinical trial can be accessed on www.clinicaltrials.gov.

About Amyotrophic Lateral Sclerosis and SOD1

Amyotrophic lateral sclerosis (ALS) is a relentless and progressive neurodegenerative disease that affects the motor neurons of the brain and spinal cord. Symptoms vary from person to person. Some forms of ALS begin with limb weakness, while others start with bulbar symptoms. All forms ultimately lead to loss of independence and a markedly shortened lifespan. Median survival remains three-to-five years, and diagnosis is often delayed.

Despite this diversity, many patients share common downstream pathologies involving axonal injury, inflammation, and protein misfolding. Superoxide dismutase 1 (SOD1) is a protective enzyme that helps cells manage oxidative stress. In ALS, structural changes can cause SOD1 to lose its proper function and misfold, taking on toxic conformations that disrupt cellular function. Such misfolded SOD1 can injure motor neurons, damage mitochondria, and impair axonal transport. Pathology can spread by the seeding of SOD1 misfolding.

Misfolded SOD1 represents a powerful therapeutic opportunity. Across different presentations of ALS, misfolded SOD1 can amplify axonal injury and accelerate disease progression, making these toxic conformations an important target for intervention.

About AL-S Pharma AG

AL-S Pharma is a clinical-stage biotech company developing AP-101 for the treatment of amyotrophic lateral sclerosis (ALS). Founded and co-owned by Neurimmune and TVM Capital Life Science, AL-S Pharma brings together a seasoned team of biotech and pharmaceutical leaders with expertise spanning drug discovery, translational research, and clinical development. AL-S Pharma is based in Zurich, Switzerland. For more information, visit www.al-spharma.com.

¹ Maier M et al., Sci Transl Med. 2018;10(470).

² Marlow TR et al., Neurobiol Dis. 2025;216:107124.

Contacts

Investor Contact

AL-S Pharma

Fabian Buller, Ph.D.

Chief Business Officer

fabian.buller@al-spharma.com

Media Contact(s)

AL-S Pharma

Kathy Vincent

Corporate Affairs

kathy.vincent@al-spharma.com

MC Services AG

Brittney Sojeva

al-spharma@mc-services.eu

FDA Approves BRAVECTO® QUANTUM (fluralaner for extended-release injectable suspension) from Merck Animal Health to Treat and Control Asian Longhorned Tick and Gulf Coast Tick for 12 Months in Dogs




FDA Approves BRAVECTO® QUANTUM (fluralaner for extended-release injectable suspension) from Merck Animal Health to Treat and Control Asian Longhorned Tick and Gulf Coast Tick for 12 Months in Dogs

Expanded label for once-yearly parasiticide treatment for dogs now includes Haemaphysalis longicornis (Asian longhorned tick) and Amblyomma maculatum (Gulf Coast tick) for 12 months

RAHWAY, N.J.–(BUSINESS WIRE)–Merck Animal Health, known as MSD Animal Health outside the United States and Canada, a division of Merck & Co., Inc., Rahway, N.J., USA (NYSE: MRK), today announced the U.S. Food and Drug Administration (FDA) approved an expanded label for BRAVECTO^® QUANTUM (fluralaner for extended-release injectable suspension) in dogs. The updated indication adds treatment and control of Asian longhorned tick (H. longicornis) and Gulf Coast tick (A. maculatum) for 12 months, when administered as a single, veterinarian‑delivered injection.

BRAVECTO QUANTUM was approved in the U.S. on July 10, 2025, and continues to be indicated to kill adult fleas, for the treatment and prevention of flea infestations, and for the treatment and control of Ixodes scapularis, Dermacentor variabilis, and Rhipicephalus sanguineus infestations for 12 months and A. americanum infestations for 8 months in dogs and puppies 6 months of age and older.

“For more than a decade, BRAVECTO has set the benchmark for extended‑duration flea and tick protection, and today’s label expansion reinforces our leadership in parasiticide innovation,” said Meg Conlon, DVM, executive director, U.S. Companion Animal Veterinary Services, Merck Animal Health. “With BRAVECTO QUANTUM, Merck Animal Health continues to deliver the longest‑lasting protection available in a single dose – now covering an even broader range of clinically important tick species. This advancement reflects our unwavering commitment to help veterinarians protect dogs with the most comprehensive, convenient, and science‑driven solutions on the market.”

Key Information:

• What’s new: BRAVECTO QUANTUM is now approved to provide 12 months of treatment and control for two additional tick species – Asian longhorned tick (H. longicornis) and Gulf Coast tick (A. maculatum) – following a single injection administered by a veterinarian. This approval expands the product’s already robust label, reinforcing BRAVECTO’s position as the longest‑lasting flea and tick protection available for dogs in the United States.
• Complete Parasite Protection Profile: BRAVECTO QUANTUM is indicated to kill adult fleas and for treatment and prevention of flea infestations. BRAVECTO QUANTUM also provides 12 months of protection against Asian longhorned ticks, Gulf Coast ticks, black‑legged ticks, American dog ticks, and brown dog ticks, along with 8 months of protection against lone star ticks following a single veterinarian administered‑injection.
• Why this matters: The extended‑release injectable formulation offers veterinarians and pet owners a simple, once‑yearly approach that supports improved compliance and consistent, uninterrupted protection. This label update helps veterinarians address the evolving parasite landscape, as both Asian longhorned and Gulf Coast ticks continue to expand geographically and present risks to canine health.
• Where it’s available: BRAVECTO QUANTUM remains available exclusively through licensed veterinarians, ensuring appropriate in‑clinic administration, guidance, and support.

The expanded indication underscores Merck Animal Health’s continued leadership in delivering innovative, extended‑duration parasite protection solutions. With BRAVECTO QUANTUM, veterinarians have an unmatched, once‑yearly option that now defends dogs against an even broader range of ticks – supporting year‑round protection and helping meet the evolving needs of pets and the people who care for them. For more information, visit us.bravecto.com or follow BRAVECTO on Instagram @bravecto.us.

About BRAVECTO^®

Since its introduction in 2014, BRAVECTO has provided longer-lasting flea and tick protection, with more than 450 million doses distributed in 100 countries over eight years. BRAVECTO is available in a variety of formulations, including products for both dogs and cats.

The flea lifecycle can last as long as 12 weeks, and monthly treatments may leave gaps in protection. Providing pets with continuous flea and tick protection is essential – whether the pet goes outside or not. Contrary to popular belief among pet owners, fleas and ticks are not only active in the spring and summer months and are a year-round risk. Fleas and ticks can easily latch onto dogs and cats and can spread serious diseases. Fleas are the most common external parasite found on pets. BRAVECTO products are available through licensed veterinarians.

About Merck Animal Health

Merck Animal Health, a division of Merck & Co., Inc., Rahway, N.J., USA, is a global animal health business committed to The Science of Healthier Animals™. For more than 130 years, we have pioneered groundbreaking science. Today, we are driven by continuous innovation to develop breakthrough medicines, vaccines and technology. Rooted in direct experience on the farm and in the clinic, we work hand in hand with our customers every step of the way. Our singular focus is to empower those who care for animals, helping them manage their vital responsibility with confidence. Because when it comes to animal health, no one sees it like we do. For more information, visit www.merck-animal-health.com and connect with us on LinkedIn, Facebook, X (formerly Twitter) and Instagram.

Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA

This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2025 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

FAQ:

• What changed with today’s approval?
  • The BRAVECTO QUANTUM label now includes treatment and control of H. longicornis (Asian longhorned tick) and A. maculatum (Gulf Coast tick) for 12 months following a single injection administered by a veterinarian, in addition to existing indications (fleas and other listed tick species) and the 8‑month indication for A. americanum (lone star tick).
• Why focus on Asian longhorned and Gulf Coast ticks?
  • These ticks are clinically relevant and expanding in geographic range. Adding them strengthens year‑round, single‑dose tick control options for more dogs across more U.S. regions.
• How is BRAVECTO QUANTUM administered?
  • BRAVECTO QUANTUM is administered via a single subcutaneous injection delivered by a licensed veterinarian.
• Is this the same BRAVECTO formulation that was introduced in 2025?
  • Yes. BRAVECTO QUANTUM remains the once‑yearly fluralaner extended‑release injectable. Today’s update expands the label’s tick species coverage.
• Is BRAVECTO QUANTUM safe?
  • BRAVECTO QUANTUM has undergone extensive safety testing. In fact, safety was demonstrated when dosed at 5 times the recommended dose every 4 months for a total of 6 doses. In a U.S. field study with client owned dogs, 0.9% (2 out of 225 dogs) experienced seizures. This rate is consistent with estimates reported in the general dog population. BRAVECTO QUANTUM (fluralaner) is a member of the isoxazoline class, which has been associated with neurologic adverse reactions, including tremors, ataxia, and seizures. Use with caution in dogs with a history of seizures or neurologic disorders.
• Where can veterinarians find full prescribing information?
  • Please see the Prescribing Information (PI) here and consult Merck Animal Health for training and ordering support.
• Can cats receive BRAVECTO QUANTUM?
  • No. BRAVECTO QUANTUM is approved only for dogs and puppies 6 months of age and older and must be administered by a licensed veterinarian. It is not approved for use in cats. For cats, Merck Animal Health offers BRAVECTO^® Plus, a topical formulation that provides broad‑spectrum, extended‑duration protection against fleas, ticks, and internal parasites.

IMPORTANT SAFETY INFORMATION:

BRAVECTO (fluralaner) Chews for Dogs: The most commonly reported adverse reactions include vomiting, lethargy, diarrhea, anorexia and pruritus. In some cases, adverse events have been reported following use in breeding females. BRAVECTO Chews has not been shown to be effective for 12-weeks’ duration in puppies less than 6 months of age. BRAVECTO Chews is not effective against lone star ticks beyond 8 weeks of dosing. Indicated for dogs 6 months of age and older. BRAVECTO 1-MONTH (fluralaner) Chews: The most commonly reported adverse reactions include itching, diarrhea, vomiting, decreased appetite, elevated ALT, lethargy, and weight loss. Not effective against lone star ticks in puppies less than 6 months of age. Indicated for dogs 8 weeks of age and older. BRAVECTO (fluralaner topical solution) for Dogs: The most commonly reported adverse reactions include vomiting, hair loss, diarrhea, lethargy, decreased appetite, and moist dermatitis/rash. BRAVECTO Topical Solution for Dogs has not been shown to be effective for 12-weeks’ duration in puppies less than 6 months of age. BRAVECTO Topical Solution for Dogs is not effective against lone star ticks beyond 8 weeks of dosing. For topical use only. Avoid oral ingestion. Indicated for dogs 6 months of age and older. BRAVECTO QUANTUM (fluralaner for extended-release injectable suspension) for Dogs: The most commonly reported adverse reactions in a US field study included lethargy, decreased appetite, vomiting, diarrhea, elevated liver enzymes and pruritus. BRAVECTO QUANTUM is not effective against lone star ticks beyond 8 months of dosing. Indicated for dogs 6 months of age and older.

BRAVECTO (fluralaner topical solution) for Cats: The most commonly reported adverse reactions include vomiting, itching, diarrhea, hair loss, decreased appetite, lethargy, and scabs/ulcerated lesions. BRAVECTO Topical Solution for Cats is not effective against American dog ticks beyond 8 weeks of dosing. BRAVECTO Topical Solution for Cats has not been shown to be effective for 12-weeks’ duration in kittens less than 6 months of age. The safety of BRAVECTO Topical Solution for Cats have not been established in breeding, pregnant and lactating cats. For topical use only. Avoid oral ingestion. Indicated for cats 6 months of age and older. BRAVECTO PLUS (fluralaner and moxidectin topical solution) for Cats: The most commonly reported adverse reactions include vomiting, hair loss, itching, diarrhea, lethargy, dry skin, elevated ALT, and hypersalivation. BRAVECTO PLUS has not been shown to be effective for 2 months in kittens less than 6 months of age. Use with caution in cats that are heartworm positive. The effectiveness of BRAVECTO PLUS to prevent heartworm disease after bathing or water immersion has not been evaluated. The safety of BRAVECTO PLUS have not been established in breeding, pregnant and lactating cats. Indicated for cats 6 months of age and older.

All BRAVECTO products contain fluralaner, which is a member of the isoxazoline class. This class has been associated with neurologic adverse reactions including tremors, ataxia, and seizures. Seizures have been reported in dogs receiving isoxazoline class drugs, even in dogs without a history of seizures. Use with caution in dogs with a history of seizures or neurologic disorders. Neurologic adverse reactions have been reported in cats receiving isoxazoline class drugs, even in cats without a history of neurologic disorders. Use with caution in cats with a history of neurologic disorders.

Contacts

Media Contact:

Laurel Sawicki

(908) 872-9783

laurel.mundth@merck.com

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

peter.dannenbaum@merck.com

Hayley Kasko

(732) 594-4237

hayley.kasko@merck.com

Guardian Pharmacy Services Announces Launch of Underwritten Public Offering of Class A Common Stock




Guardian Pharmacy Services Announces Launch of Underwritten Public Offering of Class A Common Stock

ATLANTA–(BUSINESS WIRE)–Guardian Pharmacy Services, Inc. (“Guardian”) (NYSE: GRDN) today announced the launch of a proposed underwritten public offering (the “Offering”) of 5,000,000 shares of its Class A common stock, consisting of 3,980,000 shares being offered by certain selling stockholders and 1,020,000 newly issued shares being offered by Guardian as part of a non-dilutive “synthetic secondary” transaction, as described below. In addition, the selling stockholders intend to grant the underwriters a 30-day option to purchase up to an additional 750,000 shares of Class A common stock at the public offering price, less the underwriting discount.

The proposed Offering is considered non-dilutive as Guardian intends to use all of the net proceeds it receives in the Offering to repurchase from certain stockholders a number of shares of Class A common stock equal to the number of shares being issued and sold by Guardian in the Offering, at a purchase price per share equal to the public offering price in the Offering, less the underwriting discount (the “Synthetic Secondary”). Accordingly, Guardian will not retain any proceeds from the Offering and, upon completion of the Offering and the Synthetic Secondary, the total number of outstanding shares of Class A common stock will remain the same. The shares to be repurchased by Guardian consist of shares of Class A common stock that were issued upon conversion of shares of Guardian’s Class B common stock that were originally issued in connection with its corporate reorganization in September 2024. Guardian will not receive any proceeds from the offering of shares by the selling stockholders in the Offering.

BofA Securities, Jefferies and Raymond James are acting as joint bookrunners for the proposed Offering. Stephens Inc. and Oppenheimer & Co. are acting as co-managers for the proposed Offering.

A shelf registration statement on Form S-3 relating to the shares being offered in the proposed Offering was filed with the U.S. Securities and Exchange Commission (the “SEC”) on October 14, 2025 and became effective on November 3, 2025. This press release does not constitute an offer to sell or the solicitation of an offer to buy any securities, and shall not constitute an offer, solicitation, or sale in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction. Any offers, solicitations or offers to buy, or any sales of securities will be made in accordance with the registration requirements of the Securities Act of 1933, as amended.

The proposed Offering will be made only by means of a preliminary prospectus supplement and accompanying prospectus. Copies of the preliminary prospectus supplement and the accompanying prospectus related to the proposed Offering can be obtained from: BofA Securities, Attention: Prospectus Department, NC1-022-02-25, 201 North Tryon Street, Charlotte, NC 28255-0001, by email at dg.prospectus_requests@bofa.com; or Jefferies LLC, Attn: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at 877-821-7388 or by email at prospectus_department@jefferies.com.

About Guardian Pharmacy Services

Guardian Pharmacy Services is a leading long-term care pharmacy services company that provides an extensive suite of technology-enabled services designed to help residents of long-term health care facilities (“LTCFs”) adhere to their appropriate drug regimen, which in turn helps reduce the cost of care and improve clinical outcomes. As of December 31, 2025, our 61 pharmacies, 54 of which are full-service, served approximately 205,000 residents in approximately 8,400 LTCFs across 38 states.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are all statements other than those of historical fact. Words such as “aims,” “anticipates,” “believes,” “contemplates,” “continues,” “estimates,” “expects,” “intends,” “may,” “plans,” “seeks,” “should,” “will,” “would” and similar expressions are often, but not always, used to identify forward-looking statements. These forward-looking statements include statements regarding the proposed Offering and the Synthetic Secondary, and Guardian’s use of the net proceeds to it from the proposed Offering. These forward-looking statements are based on management’s current expectations and beliefs and are inherently subject to risks and uncertainties, including, among others, uncertainties related to market conditions, and those other risks and uncertainties more fully described under “Risk Factors” in Guardian’s Annual Report on Form 10-K for the year ended December 31, 2025 and the preliminary prospectus relating to the proposed Offering. Except to the extent required by applicable law, Guardian undertakes no obligation to update or revise any information contained in this press release beyond the published date, whether as a result of new information, future events or otherwise.

Contacts

Ashley Stockton

Vice President, Investor Relations

IR@guardianpharmacy.net

GE HealthCare completes Intelerad acquisition – accelerating shift to cloud-first enterprise solutions to deliver precision care




GE HealthCare completes Intelerad acquisition – accelerating shift to cloud-first enterprise solutions to deliver precision care

• Expands GE HealthCare’s enterprise imaging footprint by delivering comprehensive, end‑to‑end solutions across ambulatory, teleradiology, and hospital care settings
• Advances GE HealthCare’s D3 strategy by accelerating the development of disease‑focused smart devices and solutions enabled by digital (cloud and software) and artificial intelligence (AI) capabilities
• Enhances a fully connected, cloud‑first imaging ecosystem with an expanded GE HealthCare portfolio of AI, digital tools, and SaaS offerings designed to improve clinical operations
• Tuck‑in acquisition expected to strengthen the Imaging portfolio, increase recurring revenue mix, and support sustainable top‑line growth and profitability over time

CHICAGO–(BUSINESS WIRE)–GE HealthCare today announced that it has completed the acquisition of Intelerad, a leading medical imaging software provider for the healthcare industry, for a base purchase price of $2.3 billion in cash (subject to customary adjustments). Intelerad’s technology and customer base will extend GE HealthCare’s reach into high-growth specialized clinics and ambulatory care environments, complementing the company’s strength in hospital-based imaging. These combined capabilities will create a more comprehensive, cloud-first and AI-enabled imaging offering, which is expected to help reduce imaging infrastructure costs and enable faster deployment times.

“We are excited to welcome Intelerad to the GE HealthCare team to create an end-to-end, cloud-first and AI-enabled enterprise imaging solution for customers,” said Roland Rott, President and CEO, Imaging, GE HealthCare. “Intelerad’s cloud-enabled software will support GE HealthCare’s imaging technologies and AI capabilities by simplifying complex workflows, and providing patients and customers with more precise, connected care across the continuum.”

“Intelerad enhances our ability to deliver a cloud-first enterprise imaging platform at scale. Together, we are connecting imaging across care settings with interoperable, AI-enabled solutions that simplify operations, improve clinical insight, and help our customers deliver more precise, personalized care,” said Scott Miller, CEO, Solutions for Enterprise Imaging, GE HealthCare.

With the closing of the acquisition, Intelerad will operate as part of GE HealthCare’s Imaging business and continue to serve its enterprise imaging solution customers in the US, Canada, the UK, and Oceania.

“Joining GE HealthCare accelerates our vision for a more intelligent, connected imaging ecosystem. Together, we can harness the cloud and AI to break down longstanding barriers in healthcare, empowering clinicians with faster insights and giving patients a more seamless, precise care experience,” said Jordan Bazinsky, CEO of Intelerad.

GE HealthCare estimates that Intelerad’s revenues in the first full year of ownership will be approximately $270 million, of which approximately 90% is recurring, and Adjusted Earnings before Interest, Taxes, Depreciation and Amortization (EBITDA) margin will be in excess of 30%. Intelerad revenue is growing in the low-double-digit range annually and is expected to accelerate under GE HealthCare ownership. GE HealthCare expects this transaction to be immediately accretive to top line growth and Adjusted Earnings before Interest and Taxes (EBIT) margin¹. Inclusive of the impact of financing costs, GE HealthCare expects the transaction to be slightly dilutive to Adjusted Earnings per Share (EPS)¹ in the short term, and the company plans to offset this with cost efficiencies. GE HealthCare expects a high-single-digit return on invested capital by year five.

GE HealthCare funded this transaction with cash on hand and proceeds from debt financing.

For more information on GE HealthCare’s Intelerad offerings and full portfolio of solutions, please visit https://www.intelerad.com/en/.

Forward-Looking Statements

This release contains forward-looking statements. These forward-looking statements might be identified by words, and variations of words, such as “will,” “expect,” “may,” “would,” “could,” “plan,” “believe,” “anticipate,” “intend,” “estimate,” “potential,” “position,” “forecast,” “target,” “guidance,” “outlook,” and similar expressions. These forward-looking statements may include, but are not limited to, statements about the transaction, the expected results of the transaction, and GE HealthCare Technologies Inc.’s (the “Company’s”) markets, business, products, financial performance, growth opportunities, and strategy. These forward-looking statements involve risks and uncertainties, many of which are beyond the control of the Company. Factors that could cause the Company’s actual results to differ materially from those described in its forward-looking statements include, but are not limited to, the Company may be unable to achieve the anticipated benefits of the transaction; operating costs and business disruptions (including, without limitation, difficulties in maintaining relationships with employees, customers, and suppliers) may be greater than expected; and the Company may assume unexpected risks and liabilities. Other factors that may cause such a difference also include those discussed in the “Risk Factors” section of the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission and any updates or amendments it makes in future filings. There may be other factors not presently known to the Company or which it currently considers to be immaterial that could cause the Company’s actual results to differ materially from those projected in any forward-looking statements the Company makes. The Company does not undertake any obligation to update or revise its forward-looking statements except as required by applicable law or regulation.

About GE HealthCare Technologies Inc.

GE HealthCare is a leading global healthcare solutions provider of advanced medical technology, pharmaceutical diagnostics, and AI, cloud and software solutions that help clinicians tackle the world’s most complex diseases. Serving patients and providers for 130 years, GE HealthCare is delivering bold innovations designed for the next era of medicine across its Imaging, Advanced Visualization Solutions, Patient Care Solutions, and Pharmaceutical Diagnostics segments to help clinicians deliver more personalized, precise patient care. We are a $20.6 billion business with approximately 54,000 colleagues working to create a world where healthcare has no limits.

GE HealthCare is proud to be among 2026 Fortune World’s Most Admired Companies™.

Follow us on LinkedIn, Facebook, Instagram, or visit our website for our latest news and perspectives.

¹ Non-GAAP financial measure; see our earnings release dated February 4, 2026, for definition.

Contacts

GE HealthCare Media Contact:
Linh Dinh

Linh.Dinh@gehealthcare.com
M +408 275 5682

GE HealthCare Investor Relations Contact:
Carolynne Borders

Carolynne.Borders@gehealthcare.com
M (631) 662-4317

Nia Therapeutics Receives FDA Breakthrough Device Designation for AI-Guided Brain Implant to Treat Memory Loss




Nia Therapeutics Receives FDA Breakthrough Device Designation for AI-Guided Brain Implant to Treat Memory Loss

The Smart Neurostimulation System is the first neurostimulation device to receive Breakthrough designation for TBI-related memory loss; the implant decodes memory states from neural activity on 60 channels spanning four brain regions and delivers AI-guided personalized stimulation therapy to lateral temporal cortex

BOSTON–(BUSINESS WIRE)–Nia Therapeutics announced that the U.S. Food and Drug Administration has granted Breakthrough Device Designation to its Smart Neurostimulation System (SNS) for the treatment of episodic memory loss in adult patients with prior moderate-to-severe traumatic brain injury (TBI) and persistent memory deficits. The SNS is the first device to receive Breakthrough designation for TBI-related memory loss. There is significant unmet need in this indication, with no FDA-cleared or approved therapies to treat memory loss and more than 4.3 million Americans living with TBI-related disability.¹

“The Breakthrough designation validates the approach we’ve spent a decade building—that memory can be improved by listening to the brain and stimulating at precisely the right moment,” said Michael Kahana, PhD, co-founder and CEO of Nia Therapeutics, and the Edmund and Louise Kahn Term Professor at the University of Pennsylvania. “This designation provides a framework to work closely with the FDA as we bring this technology from the laboratory into the clinic.”

A New Approach to Treating Memory Disorders

The SNS is a fully implantable, wireless neuromodulation platform that records neural activity from 60 channels across four brain regions. Using machine-learning classifiers trained on each patient’s own brain signals, the device detects moments of impaired memory encoding in real time and delivers targeted electrical stimulation to the lateral temporal cortex. This closed-loop approach improved recall by 19% in a randomized, sham-controlled study of neurosurgical patients with epilepsy and a history of moderate-to-severe TBI.² Randomly timed stimulation produced no benefit, underscoring the importance of delivering therapy at the right moment.³

“Memory depends on coordinated activity across widespread brain networks—so we built a device that can sense and respond across the entire network. With 60 channels across four brain regions, the SNS offers an order of magnitude improvement over currently approved DBS devices,” said Daniel Rizzuto, PhD, co-founder and CTO of Nia Therapeutics.

Path Forward

The designation provides Nia with prioritized review, increased FDA interaction, and senior management involvement in future submissions. Building on the first in vivo validation of the SNS platform in a large-animal model, published in Brain Stimulation in 2026 (link), this designation will support the company as it advances toward an Investigational Device Exemption (IDE) application this year to support a first-in-human early feasibility study.

“Patients with TBI-related memory loss represent a profoundly underserved population,” said Dr. Ramon Diaz-Arrastia, Presidential Professor of Neurology and Director of the TBI Clinical Research Center at the University of Pennsylvania, and advisor to Nia Therapeutics. “Their disability is invisible but devastating—it affects the ability to work, maintain relationships, and live independently. We are committed to bringing them the first treatment that directly restores the capacity to form new memories.”

About Nia Therapeutics

Nia Therapeutics develops implantable brain-computer interfaces for memory disorders. Founded in 2018, the company’s SNS platform enables closed-loop neuromodulation by detecting brain states linked to impaired memory encoding and delivering targeted stimulation. Visit www.niatx.com.

Sources

1. Center for Disease Control and Prevention. (2015). Report to Congress on traumatic brain injury in the United States: Epidemiology and rehabilitation (cdc:29215). https://stacks.cdc.gov/view/cdc/29215
2. Kahana, M. J., Ezzyat, Y ., Wanda, P. A., Solomon, E. A., Adamovich-Zeitlin, R., Lega, B. C., Jobst, B. C., Gross, R. E., Ding, K., & Diaz-Arrastia, R. R. (2023). Biomarker-guided neuromodulation aids memory in traumatic brain injury. Brain Stimulation, 16(4), 1086–1093. https://doi.org/10.1016/j.brs.2023.07.002
3. Ezzyat, Y ., Kragel, J. E., Solomon, E. A., Lega, B. C., Aronson, J. P., Jobst, B. C., Gross, R. E., Sperling, M. R., Worrell, G. A., Sheth, S. A., Wanda, P. A., Rizzuto, D. S., & Kahana, M. J. (2024). Functional and anatomical connectivity predict brain stimulation’s mnemonic effects. Cerebral Cortex, 34(1), bhad427. https://doi.org/10.1093/cercor/bhad427

Contacts

For media inquiries, contact media@niatx.com.