Mursla Bio Introduces AI Precision Medicine Platform Built on Organ-Specific EV Isolation from Blood

Mursla Bio Introduces AI Precision Medicine Platform Built on Organ-Specific EV Isolation from Blood




Mursla Bio Introduces AI Precision Medicine Platform Built on Organ-Specific EV Isolation from Blood

  • Platform is built on biologically labelled, organ-specific multi-omics data carried by extracellular vesicles isolated from 2ml plasma samples
  • Enables differentiated AI-powered biomarker discovery and IVD translation for patient stratification, therapeutic monitoring, surrogate markers and other diagnostic development programs
  • Publication of pre-print data, with support from Evotec International GmbH and University College London demonstrates validation of Platform’s capability for liver diseases

CAMBRIDGE, England & BOSTON–(BUSINESS WIRE)–Mursla Bio, a leader in Extracellular Vesicle (EV) science on a mission to advance precision diagnostics and significantly improve cancer outcomes for at-risk patients, today announced the commercial launch of its AI Precision Medicine Platform, alongside a pre-print¹ reporting the first validated method for isolating hepatocyte EVs from plasma for organ-specific proteomic and miRNA profiling. Mursla Bio is engaging with potential partners to apply its Platform to additional disease areas, biomarker discovery and IVD translation programs.


The Platform addresses a major gap in liquid biopsy and precision medicine: enabling non-invasive access to organ-specific molecular information from blood with high spatial and biological resolution. Mursla Bio’s Platform is built on its ability to isolate and analyze EVs which are secreted by specific organs into the bloodstream. The Platform reduces non-target background by up to five orders of magnitude compared to standard bulk EV isolation and significantly improves data structure, enhancing prediction model generalization.

The pre-print presents the first multi-layered validation of hepatocyte EV isolation in humans, confirming liver origin through proteomic and nucleic acid markers. Key supporting proteomics data was generated in collaboration with Evotec International GmbH. Patient samples were provided by University College London, with Professor Brian Davidson, a global leader in liver disease surgery and clinical research, contributing as a co-author. The study shows that Mursla Bio’s method yields robust, organ-specific multi-omics data using minimal blood volume, offering a novel scalable approach for biomarker and AI development.

Mursla Bio’s Platform incorporates optimized multi-omics workflows, embedded AI pipelines, and IVD translation processes to convert complex biological signals into practical, regulatory-grade assays. These assays are deployable on standard commercial instruments using patented, PCR and ELISA-like steps designed for clinical adoption. The Platform also underpins the development of EvoLiver™, the Company’s lead clinical program for liver cancer surveillance in cirrhotic patients. Built on multi-omics data from over 300 patients, EvoLiver achieved 86% sensitivity and 88% specificity on a locked, IVD-compatible assay for early-stage hepatocellular carcinoma detection², and has received Breakthrough Device Designation from the FDA³.

Pierre Arsène, Founder and CEO of Mursla Bio, said: “EVs are nature’s native signal enrichment system for long-distance communication between cells and organs. Our platform harnesses this biology to non-invasively access organ-specific, multi-omics data from just a small blood sample. It produces structured, biologically labeled datasets that are optimized for AI and ready to support diagnostic translation. EvoLiver is our first proof point of real-world clinical impact, and we are now expanding access to the platform to advance our pipeline and enable partnerships across other disease areas.”

Mursla Bio is expanding its pipeline and collaborations, with additional organ programs underway in cardiometabolic, lung, and neurological indications. The Platform will support pharma development through dynamic patient stratification, therapeutic response monitoring, surrogate markers and molecular phenotyping across complex disease biology.

For further information on Mursla Bio’s AI Precision Medicine Platform, please visit https://mursla.com/ai-precision-platform/.

  1. Organ-specific isolation of hepatocyte extracellular vesicles from human plasma enables tissue-resolved proteomic and miRNA profiling”
  2. Mursla Bio’s EvoLiver surpasses current standards in liver cancer surveillance
  3. Mursla Bio receives FDA Breakthrough Device Designation for EvoLiver™ test

 

Contacts

Ben Rutter: ben.rutter@zymecommunications.com

Innate Pharma Announces Its Participation in H.C. Wainwright and Wolfe Research Healthcare Conferences

Innate Pharma Announces Its Participation in H.C. Wainwright and Wolfe Research Healthcare Conferences




Innate Pharma Announces Its Participation in H.C. Wainwright and Wolfe Research Healthcare Conferences

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


Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that members of its executive team will participate in the upcoming investor conferences, detailed below.

  • H.C. Wainwright 3rd Annual Immune Cell Engager Virtual Conference

    Dates: June 24, 2025 | Virtual
  • Wolfe Research Virtual Biotech Day

    Dates: June 26, 2025 | Virtual

About Innate Pharma

Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Its innovative approach aims to harness the innate immune system through three therapeutic approaches: multi-specific NK Cell Engagers via its ANKET® (Antibody-based NK cell Engager Therapeutics) proprietary platform and Antibody Drug Conjugates (ADC) and monoclonal antibodies (mAbs).

Innate’s portfolio includes several ANKET® drug candidates to address multiple tumor types as well as IPH4502, a differentiated ADC in development in solid tumors. In addition, anti-KIR3DL2 mAb lacutamab is developed in advanced form of cutaneous T cell lymphomas and peripheral T cell lymphomas, and anti-NKG2A mAb monalizumab is developed with AstraZeneca in non-small cell lung cancer.

Innate Pharma is a trusted partner to biopharmaceutical companies such as Sanofi and AstraZeneca, as well as leading research institutions, to accelerate innovation, research and development for the benefit of patients.

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

ISIN code
Ticker code
LEI

             

FR0010331421

Euronext: IPH Nasdaq: IPHA

9695002Y8420ZB8HJE29

Disclaimer on forward-looking information and risk factors

This press release contains certain forward-looking statements, including those within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. The use of certain words, including “anticipate,” “believe,” “can,” “could,” “estimate,” “expect,” “may,” “might,” “potential,” “intend,” “should,” “will,” or the negative of these and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. These risks and uncertainties include, among other things, the uncertainties inherent in research and development, including related to safety, progression of and results from its ongoing and planned clinical trials and preclinical studies, review and approvals by regulatory authorities of its product candidates, the Company’s reliance on third parties to manufacture its product candidates, the Company’s commercialization efforts and the Company’s continued ability to raise capital to fund its development. For an additional discussion of risks and uncertainties, which could cause the Company’s actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, 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.

In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by the Company or any other person that the Company will achieve its objectives and plans in any specified time frame or at all. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

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
Innate Pharma
Henry Wheeler

Tel.: +33 (0)4 84 90 32 88

Henry.wheeler@innate-pharma.fr

Media Relations

NewCap
Arthur Rouillé

Tel.: +33 (0)1 44 71 00 15

innate@newcap.eu

D&D Pharmatech Announces Positive Phase 2 Results for DD01 in MASH with Robust Reductions in Liver Fat Accompanied by Improvements in Liver and Metabolic Health

D&D Pharmatech Announces Positive Phase 2 Results for DD01 in MASH with Robust Reductions in Liver Fat Accompanied by Improvements in Liver and Metabolic Health




D&D Pharmatech Announces Positive Phase 2 Results for DD01 in MASH with Robust Reductions in Liver Fat Accompanied by Improvements in Liver and Metabolic Health

  • After 12 weeks of treatment, the trial primary endpoint was met: 75.8% of DD01-treated subjects achieved at least a 30% reduction in liver fat, 72.7% achieved >50% reduction, and 48.5% achieved normalization of liver fat levels (<5%)
  • Treatment with DD01 was associated with a 62.3% relative reduction in liver fat compared to 8% with placebo
  • Statistically significant improvements in proC3 levels and liver stiffness (MRE), two non-invasive measures of MASH were observed at 12 weeks
  • DD01 was well tolerated; GI side effects were most common, generally mild to moderate, transient, and manageable with limited number of discontinuations
  • DD01 treatment resulted in significant reductions in HbA1c and body weight

GYEONGGI-DO, South Korea & GAITHERSBURG, Md.–(BUSINESS WIRE)–D&D Pharmatech, Inc. (D&D) (KOSDAQ: 347850), a clinical-stage biotechnology company developing breakthrough treatments for liver and metabolic diseases, today announced positive results from DD01-DN-02 study, an ongoing 48-week Phase 2 trial designed to evaluate the efficacy and safety of DD01 (a once-weekly dual GLP1/glucagon receptor agonist) in 67 overweight/obese subjects with MASH. DD01 treatment was initiated with two weeks of dosing at 20 mg, followed by the 40mg once-weekly maintenance dose.


Results of a planned 12-week assessment of safety and efficacy revealed DD01 was well tolerated, and the study’s primary endpoint was met. Following a 1:1 randomization of 40mg DD01 and placebo, 75.8% of subjects treated with DD01 achieved at least a 30% reduction in liver fat, 72.7% of subjects achieved greater than 50% reduction in liver fat, and 57.6% of subjects achieved greater than 70% liver fat reduction (in each case, with p < 0.0001). DD01 achieved a mean reduction of liver fat content of 62.3% vs 8.3% for placebo at 12 weeks of treatment, and 48.5% of DD01 subjects achieved normalization of liver fat fraction (defined as liver fat fraction of 5% or less by MRI-PDFF). No placebo subject achieved normalization.

12 Week Endpoints

DD01 40mg

n = 33

Placebo

n = 34

P-value

vs placebo

Primary Endpoint

 

 

 

Proportion of subjects achieving >30% Liver Fat Reduction

75.8%

11.8%

< 0.0001

Other Endpoints

 

 

 

Proportion of subjects achieving >50% Liver Fat Reduction

72.7%

5.9%

< 0.0001

Proportion of subjects achieving >70% Liver Fat Reduction

57.6%

0%

< 0.0001

Normalization: Proportion of subjects with Liver Fat Value <5%

48.5%

0%

< 0.0001

Treatment related reductions in Non-invasive markers of MASH progression were used to evaluate subjects at baseline and after 12 weeks of treatment. DD01 treatment was associated with statistically significant improvements in liver stiffness (MRE), pro-C3 levels, and ELF score.

Additional Endpoints

Twelve-week weight loss data were encouraging. While placebo-treated subjects had no significant weight loss, 42.4% of DD01-treated subjects achieving a greater than 5% reduction in weight. Also encouraging were the results of HbA1c testing. While the study population was not selected to be diabetic, following 12 weeks of DD01 treatment, HbA1c reduction was statistically significant compared to placebo.

Safety Results

DD01 was well tolerated. Gastrointestinal (GI) side effects were most common, generally mild to moderate, but transient and manageable. To date, only 3 subjects have discontinued treatment due to GI-related adverse events.

Implications

Seulki Lee, Ph.D., President and Chief Executive Officer of D&D Pharmatech, stated, “We have remarkably positive results for DD01 after only 12 weeks of treatment in MASH. The magnitude of improvements is equivalent to what has been achieved only after longer-term treatment with FGF and GLP-1 based drugs already validated with histology data in MASH.”

“Considering the combination of liver and metabolic benefits and the favorable tolerability profile, DD01 has the potential to provide patients and physicians with a MASH treatment that is easy to manage, encourages weight loss and is diabetes friendly.”

Mazen Noureddin, MD, MHSc, Professor of Medicine at Houston Methodist Hospital; Co-Chairman of the Board, Summit & Pinnacle Clinical Research; Director, Houston Research Institutes, commented, “The degree of liver fat reduction with DD01 is striking, with nearly three-quarters of patients achieving at least a 30% reduction and almost half reaching normalization within just 12 weeks. The observed improvement in liver stiffness by MRE, though early, adds further support to the biological activity of this dual GLP-1/glucagon approach, which is designed to act directly on the liver.”

“These consistent MRI-PDFF results provide strong confidence that DD01 has the potential to meet both key regulatory endpoints – MASH resolution and fibrosis improvement – as the program advances. Coupled with favorable metabolic effects and a very low treatment discontinuation rate, DD01 is emerging as a well-differentiated and promising therapy, both within its class and compared to other potent agents in development.”

Additional data will be presented at upcoming medical meetings.

About DD01

DD01 is a once-weekly dual GLP1/glucagon receptor agonist with a half-life of 7-8 days in obese/overweight patients with T2D and MASLD. Following a Phase 1 study that showed DD01 rapidly reduced liver steatosis, improved glucose tolerance, and encouraged weight loss in obese subjects with MASLD and type 2 diabetes, FDA granted DD01 Fast Track Designation for the treatment of MASH. Current Phase 2 results confirm and extend these findings revealing additional benefits in patients with MASH. The effect of DD01 is consistent with its dual-agonist pharmacology. Clinical results show the effects of DD01 are remarkably rapid. DD01 achieves robust results rapidly and with tolerability comparable to other validated MASH treatments.

The GLP1:glucagon potency ratio for DD01 is 11:1. GLP-1R potency is likely an important driver in glucose management for diabetic patients and in weight loss. Importantly, with this ratio, DD01 is clearly also a potent glucagon receptor agonist. Substantial liver fat reduction was observed after only 4 weeks in obese subjects with MASLD (Phase 1). Liver fat reduction by DD01 is clearly not secondary to weight loss at these early timepoints, differentiating it mechanistically from the pure incretin approach. In terms of the balance between efficacy and tolerability, the pharmacokinetics of DD01 are also unique. Good tolerability at clinically active doses is likely aided by very slow absorption (tmax = 6 days) and a long half-life (7-8 days), features which make the onset and rise in DD01 exposure gradual. Peaks associated with poor tolerability and troughs associated with diminished efficacy are avoided. As a result, the need for a lengthy titration period is eliminated and therapeutic levels are reached rapidly. A key differentiator for DD01 is the balanced constellation of clinical benefits afforded by a unique pharmacologic and pharmacodynamic signature. Current data suggest DD01 has the necessary attributes to offer patients and physicians a treatment that is easy to use, supports both liver and metabolic health.

About the DD01-DN-02 Trial

With the support of staff at Summit Clinical Research, the study investigators, and the study participants, the DD01-DN-02 study is fully enrolled and currently ongoing at 12 centers in the U.S. Baseline characteristics were well balanced between both treatment groups with nearly identical mean liver fat fractions (overall mean of 20.7%) and similar body weight (overall mean 99.4 kg) at baseline. Treatment is ongoing and the study includes non-invasive and histologic assessments of MASH resolution and change in fibrosis at 48 weeks. More information about the study is available at www.clinicaltrials.gov under the identifier NCT06410924.

About D&D Pharmatech

D&D Pharmatech is a clinical-stage biopharmaceutical company focused on developing revolutionary medicines for patients with a number of metabolic, fibrotic, and neurodegenerative diseases. DD01 and TLY012 are in development for fibrotic liver disease (MASH and cirrhosis). TLY012 has completed IND-enabling studies and has been shown to slow and even reverse established fibrosis in models of liver cirrhosis, chronic pancreatitis, and systemic scleroderma. Two products are in development for neurodegenerative diseases. NLY02 is a small molecule BBB penetrating kinase inhibitor targeting glial activation. It is a potent inhibitor of neurodegeneration. NLY01 is also a potent inhibitor of glial activation. It acts through the incretin pathway to reduce neuronal loss and slow functional decline in models of Parkinson’s, Alzheimer’s, and multiple sclerosis. In a recently completed Phase 2 study conducted in >250 Parkinson’s patients, 36-week of treatment with NLY01 resulted in a significant reduction in motor function decline (UPDRS III) in ~1/3 of trial subjects. NLY01 appeared to slow the progressive decline in motor function in newly diagnosed patients who were young (<60) and treatment naïve.

D&D Pharmatech’s ORALINK technology allows efficient oral uptake of therapeutic peptides. In partnership with Metsera, the company is developing orally active incretin-based peptide drugs for obesity.

Contacts

Neuraly Inc.

Ben Gibson

240-937-5876

bgibson@neuralymed.com

Genentech to Advance Prasinezumab Into Phase III Development for Early-Stage Parkinson’s Disease

Genentech to Advance Prasinezumab Into Phase III Development for Early-Stage Parkinson’s Disease




Genentech to Advance Prasinezumab Into Phase III Development for Early-Stage Parkinson’s Disease

– Results from Phase IIb PADOVA and longer term follow-up data suggest clinical benefit on top of symptomatic treatment in early-stage Parkinson’s disease –

– Prasinezumab is a potential first-in-class anti-alpha-synuclein antibody, targeting a known biological driver of Parkinson’s disease progression –

– Parkinson’s disease affects over 10 million people globally and significant unmet need remains –

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today its decision to proceed with Phase III development of prasinezumab, an investigational anti-alpha-synuclein antibody, in early-stage Parkinson’s disease. This decision is informed by data from the Phase IIb PADOVA study and ongoing open-label extensions (OLEs) of PADOVA and Phase II PASADENA studies.


“We are encouraged by the efficacy signals observed across the two Phase II trials and their open-label extensions, combined with the favorable safety and tolerability profile of prasinezumab,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We also recognize the substantial need for new treatment options, and the totality of data suggest that prasinezumab may have the potential to become the first disease-modifying treatment for people with Parkinson’s disease.”

Multiple endpoints from the PADOVA and OLE studies suggest a potential clinical benefit of prasinezumab when added to effective symptomatic treatment in early-stage Parkinson’s disease. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression, although missed statistical significance. Positive trends towards reduced motor progression at 104 weeks (two years) were observed; these effects appear to be sustained over longer treatment periods based on additional OLE data. The PADOVA study also provided the first biomarker evidence of prasinezumab impacting the underlying disease biology.

The PASADENA and PADOVA OLE studies, which are evaluating the long-term safety and efficacy of prasinezumab in over 750 people with early-stage Parkinson’s disease, are ongoing.

About prasinezumab

Prasinezumab is an investigational monoclonal antibody designed to bind aggregated alpha-synuclein and thereby reduce neuronal toxicity. By reducing the build-up of alpha-synuclein protein in the brain, prasinezumab can potentially prevent further accumulation and spreading between cells, which may slow progression of the disease.

Data from the Phase IIb PADOVA study suggest the possible clinical benefit of prasinezumab on top of effective symptomatic treatment in early-stage Parkinson’s disease. PADOVA investigated prasinezumab in 586 people with early-stage Parkinson’s disease, treated for a minimum of 18 months while on stable symptomatic treatment. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression with a HR=0.84 [0.69-1.01], although the study missed statistical significance (p=0.0657). In a pre-specified analysis, the effect of prasinezumab was more pronounced in the population treated with levodopa (75% of participants), HR=0.79 [0.63-0.99], p=0.0431 (nominal). Consistent positive trends across multiple secondary and exploratory endpoints were also observed. Trends towards reduced motor progression at 104 weeks (two years) were observed, showing 30-40% relative reduction versus placebo across the overall and levodopa-treated populations.

Prasinezumab continues to be well tolerated and no new safety signals were observed in the study. The safety database for prasinezumab consists of data from more than 900 Parkinson’s disease study participants that have been treated with the investigational medicine, of which more than 750 remain in open label treatment with over 500 treated for 1.5-5 years.

Roche/Genentech entered into a Licensing, Development, and Commercialization agreement with Prothena in December 2013 to develop and commercialize monoclonal antibodies targeting aggregated alpha-synuclein, such as prasinezumab, for the treatment of Parkinson’s disease.

About Parkinson’s disease

Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease characterized by the gradual loss of neurons that make dopamine and other nerve cells, and the development of motor and non-motor symptoms that may appear years before diagnosis. Today, Parkinson’s disease affects over 10 million people worldwide. The prevalence of Parkinson’s disease is increasing, and it has become one of the fastest-growing neurological disorders. Currently, symptomatic treatments that effectively alleviate motor symptoms are available today, having a significant impact on people’s quality of life; however, no available symptomatic therapies slow down or stop the clinical progression of Parkinson’s disease and the effects wear off over time as the disease progresses.

Genentech and Roche are evaluating multiple approaches to slow down disease progression and potentially prevent Parkinson’s disease that involve targeting underlying disease processes such as aggregated α-syn production, lysosomal dysfunction and neuroinflammation.

About Genentech in Neuroscience

Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

Contacts

Media Contact:

Meghan Hindman (650) 467-6800

Advocacy Contact:

Jenee Williams (650) 303-2958

Investor Contacts:

Loren Kalm (650) 225-3217

Bruno Eschli +41 61 687 5284

Prothena’s Partner Roche to Advance Prasinezumab into Phase III Development for Early-Stage Parkinson’s Disease

Prothena’s Partner Roche to Advance Prasinezumab into Phase III Development for Early-Stage Parkinson’s Disease




Prothena’s Partner Roche to Advance Prasinezumab into Phase III Development for Early-Stage Parkinson’s Disease

  • Data from Phase IIb PADOVA study and longer term follow-up data suggest clinical benefit on top of symptomatic treatment in early-stage Parkinson’s disease
  • Prasinezumab is a potential first-in-class anti-alpha-synuclein antibody, targeting a known biological driver of Parkinson’s disease progression
  • Parkinson’s disease affects over 10 million people globally and significant unmet need remains

DUBLIN–(BUSINESS WIRE)–$PRTA #prothena–Prothena Corporation plc (NASDAQ:PRTA) today announced partner Roche will advance prasinezumab, an investigational anti-alpha-synuclein antibody, into Phase III development in early-stage Parkinson’s disease. This decision is informed by data from the Phase IIb PADOVA study and ongoing open-label extensions (OLE) from both PADOVA and the Phase II PASADENA study.


As pioneers in developing the first anti-alpha-synuclein targeting antibody, we are excited to see Roche advancing prasinezumab into Phase III development, with the potential to deliver the first disease-modifying treatment option to the millions of individuals living with Parkinson’s disease and their families,” stated Gene Kinney, Ph.D., President and Chief Executive Officer, Prothena.

Multiple endpoints from the PADOVA and OLE studies suggest a potential clinical benefit of prasinezumab when added to effective symptomatic treatment in early-stage Parkinson’s disease. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression, although missed statistical significance. Positive trends towards reduced motor progression at 104 weeks (two years) were observed; these effects appear to be sustained over longer treatment periods based on additional OLE data. The PADOVA study also provided the first biomarker evidence of prasinezumab impacting the underlying disease biology.

The PASADENA and PADOVA OLE studies, which are evaluating the long-term safety and efficacy of prasinezumab in over 750 people with early-stage Parkinson’s disease, are ongoing.

About Prasinezumab

Prasinezumab is an investigational monoclonal antibody designed to bind aggregated alpha-synuclein and thereby reduce neuronal toxicity. By reducing the build-up of alpha-synuclein protein in the brain, prasinezumab can potentially prevent further accumulation and spreading between cells, which may slow progression of the disease.

Data from the Phase IIb PADOVA study suggest the possible clinical benefit of prasinezumab on top of effective symptomatic treatment in early-stage Parkinson’s disease. PADOVA investigated prasinezumab in 586 people with early-stage Parkinson’s disease, treated for a minimum of 18 months while on stable symptomatic treatment. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression with a HR=0.84 [0.69-1.01], although the study missed statistical significance (p=0.0657). In a pre-specified analysis, the effect of prasinezumab was more pronounced in the population treated with levodopa (75% of participants), HR=0.79 [0.63-0.99], p=0.0431 (nominal). Consistent positive trends across multiple secondary and exploratory endpoints were also observed. Trends towards reduced motor progression at 104 weeks (two years) were observed, showing 30-40% relative reduction versus placebo across the overall and levodopa-treated populations.

Prasinezumab continues to be well tolerated and no new safety signals were observed in the study. The safety database for prasinezumab consists of data from more than 900 Parkinson’s disease study participants that have been treated with the investigational medicine, of which more than 750 remain in open label treatment with over 500 treated for 1.5-5 years.

In December 2013, Prothena and Roche entered into a worldwide collaboration to develop and commercialize antibodies that target alpha-synuclein, including prasinezumab. Roche has sole responsibility for developing and commercializing prasinezumab and has agreed to pay Prothena up to double-digit teen royalties on net sales. To date, Prothena has earned $135 million with up to $620 million in additional milestone payments that include regulatory and sales milestones. In addition, Prothena has an option to co-promote prasinezumab in the U.S.

About Parkinson’s disease

Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease characterized by the gradual loss of neurons that make dopamine and other nerve cells. Today, Parkinson’s disease affects over 10 million people worldwide. The prevalence of Parkinson’s disease is increasing, and it has become one of the fastest-growing neurological disorders. Currently, symptomatic treatments that effectively alleviate motor symptoms are available. However, no therapies slow down or stop the clinical progression of Parkinson’s disease.

About Prothena

Prothena Corporation plc is a clinical-stage biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases. Fueled by its deep scientific expertise built over decades of research, Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. Prothena’s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including ATTR amyloidosis with cardiomyopathy, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases. For more information, please visit the Company’s website at www.prothena.com and follow the Company on X (formerly Twitter) @ProthenaCorp.

Forward-Looking Statements

This press release contains forward-looking statements. These statements relate to, among other things, the treatment potential, design, and proposed mechanism of action prasinezumab; plans for ongoing and future clinical trials of prasinezumab; and amounts we might receive under our collaboration with Roche. These statements are based on estimates, projections and assumptions that may prove not to be accurate, and actual results could differ materially from those anticipated due to known and unknown risks, uncertainties and other factors, including but not limited to those described in the “Risk Factors” sections of our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 8, 2025, and discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC. We undertake no obligation to update publicly any forward-looking statements contained in this press release as a result of new information, future events, or changes in our expectations.

Contacts

Media

Michael Bachner, Senior Director, Corporate Communications

609-664-7308, michael.bachner@prothena.com

Investors

Mark Johnson, CFA, Vice President, Investor Relations

650-417-1974, mark.johnson@prothena.com

HekaBio, Japan-based Biopharma and MedTech Platform, Enters Strategic Partnership with Alfresa Holdings Corporation

HekaBio, Japan-based Biopharma and MedTech Platform, Enters Strategic Partnership with Alfresa Holdings Corporation




HekaBio, Japan-based Biopharma and MedTech Platform, Enters Strategic Partnership with Alfresa Holdings Corporation

~~Accelerating Access to Innovative Therapies in Japan through Alfresa Group’s ‘Total Supply Chain Service’~~


TOKYO–(BUSINESS WIRE)–HekaBio K.K. (Headquarters: Chuo-ku, Tokyo; President & CEO: Robert E. Claar; hereinafter “HekaBio”) today announced the signing of a strategic partnership agreement with Alfresa Holdings Corporation (Headquarters: Chiyoda-ku, Tokyo; Representative Director & President: Ryuji Arakawa; hereinafter “Alfresa Holdings”), the leading pharmaceutical wholesaler group in Japan. As part of this agreement, Alfresa Holdings has also made a capital investment in HekaBio. The partnership aims to accelerate the commercialization of innovative medical products in Japan.

Background and Purpose

In recent years, Japan has implemented policy reforms to address the growing access gap, where innovative therapies approved overseas are slow to come to the Japanese market.

In response to these developments, HekaBio has focused on introducing cutting-edge pharmaceuticals, medical devices, and regenerative medicine products from global markets into Japan and the Asia-Pacific markets. Leveraging a proprietary global network and partnership model, HekaBio evaluates over 200 assets annually across oncology, cardiology and CNS, and supports end-to-end commercialization from licensing to clinical and regulatory development, manufacturing and sales.

This partnership with Alfresa Group will enable HekaBio to advance and expand its portfolio and impact, particularly in CNS and regenerative medicine where Alfresa Group has strengths in manufacturing and in sales, including various other aspects of sales-related operations across the supply chain.

Outlook

Through this partnership, HekaBio and Alfresa Group aim to accelerate the introduction of high-impact, potential blockbuster therapies into the Japanese market, expanding treatment options for patients. The companies also plan to explore commercialization strategies across the Asia-Pacific region and beyond.

About HekaBio

HekaBio is a Japan-based, asset-light healthcare platform that accelerates patient access to global medicines and MedTech across Asia-Pacific. HekaBio reviews over 200 assets annually to in-license a select few, focusing on early-stage, de-risked opportunities in CNS, oncology, and healthy longevity. HekaBio synchronizes U.S. and Japan development timelines and leverages strategic partnerships to optimize commercial success. Japan, the world’s third-largest healthcare market, serves as its launchpad for regional expansion.

To learn more about HekaBio’s mission and portfolio, visit www.heka.bio

About Alfresa Holdings

Alfresa Holdings is the holding company for the Alfresa Group, which operates businesses ranging from the development, manufacture, and distribution of pharmaceuticals to the operation of dispensing pharmacies and is the top corporate group in Japan in terms of ethical pharmaceuticals wholesaling, with consolidated net sales exceeding ¥2.9 trillion in the fiscal year ended March 31, 2025. Guided by its corporate philosophy, “we create and deliver a fresh life for all,” the entire Group supports supply chains for pharmaceuticals and other products, a form of social infrastructure underpinning Japanese healthcare, contributing to a wide range of medical needs.

For more information: https://www.alfresa.com/eng/

Contacts

HekaBio K.K.

Corporate Communications

Mari Tanaka

Email: IR@heka.bio

Alfresa Holdings Corporation

Corporate Communication Department

Email: ir@alfresa.com

SINOVAC Board of Directors Alerts Shareholders to Advantech/Prime Success’ New Lawsuit, Which Threatens to Prevent Payment of US$55.00 Special Dividend to All Valid Shareholders

SINOVAC Board of Directors Alerts Shareholders to Advantech/Prime Success’ New Lawsuit, Which Threatens to Prevent Payment of US$55.00 Special Dividend to All Valid Shareholders




SINOVAC Board of Directors Alerts Shareholders to Advantech/Prime Success’ New Lawsuit, Which Threatens to Prevent Payment of US$55.00 Special Dividend to All Valid Shareholders

Vivo Capital Challenges SINOVAC’s Paying Agent on Distribution of the Dividend

Opposition Brief to be Filed Monday in Advance of Wednesday Hearing and Expected Friday Decision

BEIJING–(BUSINESS WIRE)–The Board of Directors of SINOVAC Biotech Ltd. (NASDAQ: SVA) (“SINOVAC” or the “Company”), a leading provider of biopharmaceutical products in China, today disclosed that on June 12, 2025, Advantech/Prime Success filed a Petition for Emergency Injunctive Relief against SINOVAC, et al., naming as relief parties Cede & Co., The Depository Trust Company, and Equiniti (the dividend paying agent retained by the SINOVAC Board), which may prevent the SINOVAC Board from paying the US$55.00 special dividend as scheduled on or about July 9, 2025.

We learned that Vivo Capital’s counsel informed the paying agent that Vivo would challenge the dividend and asked the paying agent to withdraw from facilitating the distribution of the US$55.00 dividend declared by the SINOVAC Board to all valid shareholders.

The SINOVAC Board has an obligation to answer this Petition on behalf of all SINOVAC common shareholders to stand up to the egregious actions of Prime Success. We expect to file our Opposition Brief today at 5:00 p.m. Atlantic Standard Time, followed by a hearing on Wednesday, June 18.

We continue to be shocked by the self-serving, unnecessary actions by Advantech and Vivo Capital (together known as the “Dissenting Investor Group”) against SINOVAC, threatening the rightful dividend payment to all valid SINOVAC common shareholders. These incomprehensible actions are unacceptable to the SINOVAC Board because:

  • The Dissenting Investor Group has already pocketed more than US$1.1 billion in dividends from SINOVAC’s operating subsidiary, while all SINOVAC’s valid shareholders received nothing.
  • An amount equal to the aggregate amount of cash that would be payable under the US$55.00 per common share special dividend in respect of the 2018 PIPE shares has been set aside by the SINOVAC Board in an escrow account managed by an independent third party pending final resolution of the legal proceedings, which the Dissenting Investor Group initiated. Thus, there is no risk whatsoever that the Dissenting Investor Group’s allocations of the dividend will not be paid if the courts ultimately decide in their favor regarding the PIPE shares.

These new actions by the Dissenting Investor Group go against their assertions of their support of the US$55.00 dividend payment in their letters to shareholders and raises the question – how can you trust anything they say in their letters or press releases?

The SINOVAC Board will continue to fight these frivolous lawsuits, all of which aim to accomplish one thing: to allow the Dissenting Investor group to double dip on dividends. We feel certain we will prevail quickly in this and other proceedings. The SINOVAC Board is on a mission to restore fairness and deliver value to ALL SINOVAC shareholders.

About SINOVAC

Sinovac Biotech Ltd. (SINOVAC) is a China-based biopharmaceutical company that focuses on the R&D, manufacturing, and commercialization of vaccines that protect against human infectious diseases.

SINOVAC’s product portfolio includes vaccines against COVID-19, enterovirus 71 (EV71) infected Hand-Foot-Mouth disease (HFMD), hepatitis A, varicella, influenza, poliomyelitis, pneumococcal disease, etc.

The COVID-19 vaccine, CoronaVac®, has been approved for use in more than 60 countries and regions worldwide. The hepatitis A vaccine, Healive®, passed WHO prequalification requirements in 2017. The EV71 vaccine, Inlive®, is an innovative vaccine under “Category 1 Preventative Biological Products” and commercialized in China in 2016. In 2022, SINOVAC’s Sabin-strain inactivated polio vaccine (sIPV) and varicella vaccine were prequalified by the WHO.

SINOVAC was the first company to be granted approval for its H1N1 influenza vaccine Panflu.1®, which has supplied the Chinese government’s vaccination campaign and stockpiling program. The Company is also the only supplier of the H5N1 pandemic influenza vaccine, Panflu®, to the Chinese government stockpiling program.

SINOVAC continually dedicates itself to new vaccine R&D, with more combination vaccine products in its pipeline, and constantly explores global market opportunities. SINOVAC plans to conduct more extensive and in-depth trade and cooperation with additional countries, and business and industry organizations.

Safe Harbor Statement

This announcement contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and as defined in the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by terminology such as “may,” “will,” “expect,” “anticipate,” “aim,” “estimate,” “intend,” “plan,” “believe,” “potential,” “continue,” “is/are likely to” or other similar expressions. Such statements are based upon current expectations and relate to events that involve known or unknown risks, uncertainties and other factors, including without limitation risks, uncertainties and factors related to the timing of filings, hearings and other litigation-related matters, all of which are difficult to predict and many of which are beyond the Company’s control, which may cause actual results, performance or achievements to differ materially from those in the forward-looking statements. Further information regarding these and other risks, uncertainties or factors is included in the Company’s filings with the U.S. Securities and Exchange Commission. The Company does not undertake any obligation to update any forward-looking statement as a result of new information, future events or otherwise, except as required under law.

Contacts

Investor and Media Contact
FGS Global

Sinovac@fgsglobal.com

QIAGEN and Incyte Announce Precision Medicine Collaboration to Develop Companion Diagnostics for Patients With Mutant CALR-expressing Myeloproliferative Neoplasms (MPNs)

QIAGEN and Incyte Announce Precision Medicine Collaboration to Develop Companion Diagnostics for Patients With Mutant CALR-expressing Myeloproliferative Neoplasms (MPNs)




QIAGEN and Incyte Announce Precision Medicine Collaboration to Develop Companion Diagnostics for Patients With Mutant CALR-expressing Myeloproliferative Neoplasms (MPNs)

  • QIAGEN to create a multimodal panel using next-generation sequencing (NGS) technology for detecting clinically relevant gene alterations in hematological malignancies
  • Companion diagnostic to identify key disease-causing mutations in patients with MPNs, with an initial focus on mutant CALR the second most common driver of MPNs
  • Panel to be validated on Illumina NextSeq 550Dx platform for use with whole blood samples
  • Partnership supports Incyte’s extensive portfolio in myeloproliferative neoplasms, including INCA033989, and enhances QIAGEN’s onco-hematology diagnostics pipeline

VENLO, Netherlands & WILMINGTON, Del.–(BUSINESS WIRE)–$QGEN #QIAGEN–QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) and Incyte (Nasdaq: INCY) today announced a new global collaboration to develop a novel diagnostic panel to support Incyte’s extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), a group of rare blood cancers, including Incyte’s monoclonal antibody INCA033989, targeting mutant calreticulin (mutCALR), which is being developed in myelofibrosis (MF) and essential thrombocythemia (ET).


Under the terms of the Master Collaboration Agreement with Incyte, QIAGEN will develop a multimodal panel using next-generation sequencing (NGS) technology for detecting clinically relevant gene alterations in hematological malignancies.

The panel will be validated using the next-generation sequencing (NGS) technology and the Illumina NextSeq 550Dx platform as part of QIAGEN’s partnership with Illumina (NASDAQ: ILMN) to leverage its NGS diagnostic platforms for patient testing by laboratories worldwide. QIAGEN will support regulatory submission processes and market access activities across the United States, European Union and Asia-Pacific regions.

Myeloproliferative neoplasms are a group of diseases representing about 40% of hematological malignancies, characterized by chronic accumulation of different mature blood cell types in blood.

Identifying genomic aberrations in clinically relevant biomarkers like CALR are shown to be key, especially in MPNs. Incyte is at the forefront of developing novel therapies, including INCA033989 for patients with mutCALR ET or MF, that target only malignant cells, sparing normal cells. The use of companion diagnostics helps guide clinicians in making treatment decisions that can lead to better patient outcomes.

“Following our presentation of positive, late-breaking data from our first-in-class mutCALR-targeted antibody at EHA, we are excited to announce this partnership with QIAGEN, which will facilitate CALR testing for patients with MPNs on a global basis. The development of companion diagnostics for mutCALR, coupled with the potential for new medicines to selectively target disease-initiating cells, is a critical step toward changing the course of disease in patients with ET and MF,” said Pablo J. Cagnoni, M.D., President and Head of Research and Development, Incyte. “As a partner, QIAGEN has the proven expertise in companion diagnostics development and approvals needed to support our ongoing work and commitment to transforming the treatment of patients with CALR-mutant MPNs.”

“Together with Incyte we are building a multimodal companion diagnostic using a powerful technology like next-generation sequencing to facilitate highly accurate testing for several blood cancer genes at once,” said Jonathan Arnold, Vice President and Head of Partnering for Precision Diagnostics at QIAGEN. “This new partnership strengthens our role in offering companion diagnostics for the growing number of biomarkers being discovered in onco-hematology and maximizing the clinical utility of the diagnostic for payor and patient benefit, thus supporting the work of innovative, science-driven companies like Incyte to improve patient outcomes.”

About Mutations in Calreticulin (mutCALR)

Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein production. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),i a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.ii,iii Among the two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases.i,ii

About QIAGEN

QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions, enabling customers to extract and gain valuable molecular insights from 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 software and knowledge bases can be used to interpret data to find actionable insights. Automation solutions bring these processes together into seamless and cost-effective workflows. QIAGEN serves over 500,000 customers globally in Life Sciences (academia, pharma R&D and industrial applications, primarily forensics) and Molecular Diagnostics for clinical healthcare. As of March 31, 2025, QIAGEN employed approximately 5,700 people in over 35 locations worldwide. For more information, visit https://www.qiagen.com.

QIAGEN is a pioneer in precision medicine and the leader in collaborating with pharmaceutical and biotechnology companies to develop companion diagnostics, having more than 30 master collaboration agreements with global pharmaceutical and biotechnology companies to develop and commercialize diagnostic tests. QIAGEN’s offering to these companies encompasses technologies ranging from polymerase chain reaction (PCR), near-patient testing and digital PCR (dPCR) to next-generation sequencing (NGS), and sample types from liquid biopsy to tissue. It also spans disease areas from cancer to non-oncology diseases such as neurodegenerative, inflammatory, and metabolic diseases – including 16 FDA-approved PCR-based companion diagnostics.

For more information about QIAGEN’s efforts in precision medicine please visit https://www.qiagen.com/us/clp/partnering-for-precision-diagnostics.

About Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia.

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

QIAGEN Forward-Looking Statement

Certain statements in this press release may constitute forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended. These statements, including those regarding QIAGEN’s products, development timelines, marketing and / or regulatory approvals, financial and operational outlook, growth strategies, collaborations and operating results – such as expected adjusted net sales and adjusted diluted earnings – are based on current expectations and assumptions. However, they involve uncertainties and risks. These risks include, but are not limited to, challenges in managing growth and international operations (including the effects of currency fluctuations, regulatory processes and logistical dependencies), variability in operating results, commercial development for our products to customers in the Life Sciences and clinical healthcare, changes in relationships with customers, suppliers or strategic partners; competition and rapid technological advancements; fluctuating demand for QIAGEN’s products due to factors such as economic conditions, customer budgets and funding cycles; obtaining and maintaining regulatory approvals for our products; difficulties in successfully adapting QIAGEN’s products into integrated solutions and producing these products; and protecting product differentiation from competitors. Additional uncertainties may arise from market acceptance of new products, integration of acquisitions, governmental actions, global or regional economic developments, natural disasters, political or public health crises, and other “force majeure” events. There is also no guarantee that anticipated benefits from restructuring programs and acquisitions will materialize as expected. For a comprehensive overview of risks, please refer to the “Risk Factors” contained in our most recent Annual Report on Form 20-F and other reports filed with or furnished to the U.S. Securities and Exchange Commission.

Incyte Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the potential for Incyte’s mut-CALR targeted antibody (INCA033989) to provide a potential treatment option for patients with ET or MF, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA, EMA, and other regulatory authorities; the efficacy or safety of Incyte and its partners’ products; the acceptance of Incyte and its partners’ products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in our reports filed with the U.S. Securities and Exchange Commission, including our annual report on Form 10-K and our quarterly report on Form 10-Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.

Source: QIAGEN N.V.

Category: Precision Medicine

i Raghavan, M., Wijeyesakere S.J., Peters L.R., Del Cid N. (2013) Calreticulin in the immune system: ins and outs. Trends in Immunology, 34(1):13-21. Link to source (https://www.cell.com/trends/immunology/abstract/S1471-4906(12)00131-7?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1471490612001317%3Fshowall%3Dtrue)

ii Nangalia J. Massie C.E., Baxter E.J., Nice F.L., et al. (2013) Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. New England Journal of Medicine, 369(25):2391-2405. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1312542?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

iii Klampfl T., Gisslinger, H., Harutyunyan A.S., et al. (2013) Somatic mutations of calreticulin in myeloproliferative neoplasms. New England Journal of Medicine, 369(25):2379-2390. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1311347?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

Contacts

Contacts QIAGEN:

Investor Relations

ir@QIAGEN.com

Public Relations

pr@QIAGEN.com

Contacts Incyte:

M
edia

media@incyte.com

Investors

ir@incyte.com

Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025

Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025




Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025

  • Data demonstrates the potential for INCA033989 to modify disease by directly inhibiting and eliminating oncogenic mutCALR cells, while sparing healthy cells and restoring normal blood cell production
  • In high-risk patients with essential thrombocythemia (ET) with a CALR mutation (mutCALR), 86% of INCA033989-treated patients at doses 400 mg and above achieved a complete or partial hematologic response with the majority (82%) realizing a complete response
  • A reduction in peripheral blood mutCALR variant allele frequency (VAF) was observed in 89% of evaluable patients correlating with hematologic response
  • Initial results demonstrate a favorable safety profile – no dose limiting toxicities were reported, a maximum tolerated dose was not reached and 98% of patients remained on treatment
  • Incyte will host an in-person analyst and investor event highlighting this data at EHA today, Sunday, June 15, 2025, from 6:00 – 7:30 a.m. EDT (12:00 -1:30 p.m. CEST)

WILMINGTON, Del.–(BUSINESS WIRE)–$INCY–Incyte (Nasdaq:INCY) today announced the first clinical data from two studies evaluating the safety, tolerability and efficacy of INCA033989, a novel, first in class, Incyte-discovered, targeted monoclonal antibody in patients with mutant calreticulin (mutCALR)-expressing myeloproliferative neoplasms (MPNs). These data – featured today in the Late-Breaking Oral Session (#LB4002) at the European Hematology Association 2025 (EHA2025) Congress in Milan, Italy – focus on the dose escalation portion of the studies in patients with high risk essential thrombocythemia (ET) who are resistant/intolerant to prior cytoreductive therapy.


The studies evaluated the safety and efficacy of INCA033989 in patients with ET as measured by hematologic response and reduction in mutCALR variant allele frequency (VAF).

Results as of April 4, 2025, showed rapid and durable normalization of platelet counts across all dose levels, with a trend toward improved responses in higher doses (>400 mg), in patients with ET treated with INCA033989. Notably, 86% of patients at doses 400 mg and above achieved a complete or partial hematologic response, with the majority (82%) of patients achieving complete response. Eighty-nine (89) percent of evaluable patients (34/38) showed a reduction in mutCALR VAF from baseline. A partial molecular response (>50% VAF reduction) was observed in 21% of evaluable patients (8/38) after only 3 cycles of treatment.

An exploratory study using single-cell DNA (scDNA) sequencing showed that INCA033989 directly targets and reduces cells carrying mutCALR. This reduction was seen in early blood-forming (CD34-positive) cells and cells in the myeloid-erythroid (ME) lineage. At the same time, there was a clear increase in healthy (wild-type CALR) cells, suggesting that the treatment supports the return of normal blood production. Bone marrow biopsies further confirmed these effects showing fewer megakaryocytes with mutCALR protein and a notable increase in megakaryocytes without mutCALR protein. Together, these findings demonstrate the selectivity of INCA033989, allowing for normalization of healthy hematopoiesis and disease modification.

“The late-breaking data presented today highlight the impact of INCA033989, a novel agent that selectively targets mutant CALR, to inhibit and eliminate cancer-causing cells in patients with essential thrombocythemia (ET), while sparing healthy cells and normalizing healthy blood production,” said Pablo J. Cagnoni, M.D., President, Head of Research and Development, Incyte. “These findings, and the further development of INCA033989, offer the potential to significantly transform the treatment of patients with CALR-mutant myeloproliferative neoplasms (MPNs).”

The results (N=49) showed that INCA033989 was well tolerated across all dose cohorts (24 to 2,500 mg), with no dose-limiting toxicities observed. Only one (1) patient discontinued treatment, and only one (1) dose reduction due to treatment-emergent adverse events (TEAEs) was observed. No infusion interruptions due to TEAEs were reported, and a maximum tolerated dose was not reached. Forty-two (42) patients across the dose cohorts reported a TEAE. The most common TEAEs were fatigue (26.5%) and upper respiratory tract infection (20.4%), all of which were Grade ≤2. Thirteen (13) patients had Grade >3 TEAEs, with transient asymptomatic lipase increase as the most common (6%).

“mutCALR is the second most common oncogenic driver of MPNs, yet the therapeutic landscape lacks a targeted agent for mutCALR expressing MPNs. Currently, ET treatments aim to prevent vascular complications and improve symptoms but are limited by toxicity and tolerability issues,” said John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mt. Sinai and Director, Center of Excellence for Blood Cancers and Myeloid Disorders, The Tisch Cancer Institute. “These data support the hypothesis that INCA033989 has the potential not only to normalize platelet counts and provide rapid and durable hematologic responses – but to induce molecular responses, which could potentially change the natural history of the disease.”

Additional data from the INCA033989 study in patients with myelofibrosis will be submitted for presentation at a future medical meeting. Discussions with regulatory authorities are planned with the goal to initiate a Phase 3 study by early 2026.

More information regarding the EHA2025 Congress and the data from Incyte’s hematology/oncology portfolio being featured at the meeting can be found on the EHA website: https://ehaweb.org/congress/eha2025-congress.

Incyte Conference Call and Webcast

Incyte will host an in-person analyst and investor event on Sunday, June 15, 2025, from 6:00 – 7:30 a.m. ET (12:00 – 1:30 p.m. CEST) to discuss key mutCALR data presented at EHA.

The event will be webcasted and can be accessed via the Events and Presentations tab of the Investor section of Incyte.com and it will be available for replay for 30 days.

About Myeloproliferative Neoplasms

Myeloproliferative neoplasms (MPNs) are a closely related group of blood cancers in which the bone marrow functions abnormally. The bone marrow is where the body’s blood cells are made. MPNs are progressive blood cancers that can strike anyone at any age, but they are more common in older adults. Estimates of the prevalence of MPNs vary, but analysis of claims data suggests there may be as many as 200,000 people in the U.S. living with the most prevalent MPNs: myelofibrosis, polycythemia vera or essential thrombocythemia (ET).1

About Mutations in Calreticulin (mutCALR)

Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),2 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.3,4 Among two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases.2,3 There are approximately 60,000 patients in the U.S. and Europe with mutCALR positive ET.5

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy.

About the INCA033989 Trial Program

The clinical trial program for INCA033989 includes two multicenter, open-label Phase 1 studies, INCA33989-101 (NCT05936359) and INCA33989-102 (NCT06034002), enrolling ~225 patients outside of the U.S. and ~140 patients in the U.S., respectively. The studies are evaluating the safety, tolerability, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD) and/or recommended dose(s) for expansion (RDE) of INCA033989 administered as a monotherapy or in combination with ruxolitinib in patients with myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET) and myelofibrosis (MF). The intent of Part 1A (dose escalation) is to identify the MTD and/or the RDE of INCA033989 among patients with MF and ET. In Part 1A INCA033989 is administered intravenously every two weeks at a protocol defined dose ranging from 24 mg. to 2,500 mg. In Part 1B (dose expansion), INCA033989 is administered at the RDE(s) identified during Part 1A.

The primary endpoint of the studies focuses on safety and tolerability as measured by: the number of participants with DLTs up to 28 days, the number of participants with treatment-emergent adverse events (TEAEs) up to 3 years and 60 days, and the number of participants with TEAEs leading to dose modification or discontinuation up to 3 years and 60 days. Secondary endpoints include response rates, mean change of ET total symptom score from baseline, percentage of MF patients achieving spleen volume reduction, MF patient anemia response, mean change in disease-related allele burden and various pharmacokinetics measures up to 3 years and 60 days.

For more information on the study, please visit: https://clinicaltrials.gov/study/NCT05936359 and https://clinicaltrials.gov/study/NCT06034002.

About Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia.

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data for Incyte’s anti-mutCALR monoclonal antibody (INCA033989), the potential this monoclonal antibody offers for patients, and expectations regarding ongoing and future clinical trials contain predictions, estimates, and other forward-looking statements.

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; determinations made by the FDA, EMA and other regulatory agencies; Incyte’s dependence on its relationships with and changes in the plans of its collaboration partners; the efficacy or safety of Incyte’s products and the products of Incyte’s collaboration partners; the acceptance of Incyte’s products and the products of Incyte’s collaboration partners in the marketplace; market competition; unexpected variations in the demand for Incyte’s products and the products of Incyte’s collaboration partners; the effects of announced or unexpected price regulation or limitations on reimbursement or coverage for Incyte’s products and the products of Incyte’s collaboration partners; sales, marketing, manufacturing and distribution requirements, including Incyte’s and its collaboration partners’ ability to successfully commercialize and build commercial infrastructure for newly approved products and any additional products that become approved; greater than expected expenses, including expenses relating to litigation or strategic activities; variations in foreign currency exchange rates; and other risks detailed in Incyte’s reports filed with the Securities and Exchange Commission, including its annual report on form 10-K and our quarterly report on Form 10-Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.

_____________________________
1 Data on file

2 Raghavan, M., Wijeyesakere S.J., Peters L.R., Del Cid N. (2013) Calreticulin in the immune system: ins and outs. Trends in Immunology, 34(1):13-21. Link to source (https://www.cell.com/trends/immunology/abstract/S1471-4906(12)00131-7?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1471490612001317%3Fshowall%3Dtrue)

3 Nangalia J. Massie C.E., Baxter E.J., Nice F.L., et al. (2013) Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. New England Journal of Medicine, 369(25):2391-2405. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1312542?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

4 Klampfl T., Gisslinger, H., Harutyunyan A.S., et al. (2013) Somatic mutations of calreticulin in myeloproliferative neoplasms. New England Journal of Medicine, 369(25):2379-2390. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1311347?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

5 Epidemiology Source: DRG, Prevalence 2026.

 

Contacts

Incyte Contacts:
Media
media@incyte.com

Investors
ir@incyte.com

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne




Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators


Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place

ENVISION study is paused while seeking a protocol amendment to incorporate additional immunosuppression

Sarepta to host investor call on June 16, 2025, at 8:00 am Eastern time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams.

Key Safety Initiatives

Evaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel’s recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance.

Suspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place.

For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after ELEVIDYS infusion, along with post-treatment monitoring, remains the same.

ENVISION Study Paused: Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303). FDA concurs with this action. The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume.

ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy. In the U.S., it serves as the confirmatory trial required under the FDA’s accelerated approval pathway for non-ambulatory patients.

“Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. “We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time. Duchenne muscular dystrophy is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients.”

Commitment to Long-Term Safety and Understanding

Sarepta remains committed to a thorough approach and the highest standards of patient safety and scientific rigor. The event has been reported to FDA and global health authorities and will inform ongoing discussions around a potential label update to reflect the risk of severe ALF and additional immune management strategies for non-ambulatory patients. While elevated liver enzymes are a known class effect of all AAV-based gene therapies, the exact mechanism behind AAV-related liver toxicity remains unclear. Current evidence suggests it is likely driven by an adaptive immune response. The Company will provide additional updates as appropriate.

Investor Conference Call Details

Sarepta will be hosting a conference call and webcast to discuss this update and provide an update on the Company’s business on Monday, June 16, 2025, at 8:00 am Eastern time. The event will be webcast live under the investor relations section of Sarepta’s website at: https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

  • For patients who are ambulatory and have a confirmed mutation in the DMD gene
  • For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

WARNINGS AND PRECAUTIONS:

Infusion-related Reactions:

  • Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
  • ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
  • Discontinue infusion for anaphylaxis.

Acute Serious Liver Injury:

  • Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
  • Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels).
  • Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated Myositis:

  • In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.
  • Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.
  • Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur.

Myocarditis:

  • Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.
  • If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.
  • Advise patients to contact a physician immediately if they experience cardiac symptoms.

Preexisting Immunity against AAVrh74:

  • In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.
  • Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.
  • ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

Adverse Reactions:

  • The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

For further information, please see the full Prescribing Information.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements

This statement contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients.

Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

Contacts

Investor Contact:
Ian Estepan

617-274-4052

iestepan@sarepta.com

Media Contacts:
Tracy Sorrentino

617-301-8566

tsorrentino@sarepta.com

Kara Hoeger

617-710-3898

KHoeger@sarepta.com