Market Newsdesk

– ZEVASKYN, the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), now commercially available in the U.S. –

– Ann & Robert H. Lurie Children’s Hospital of Chicago, a top-ranked hospital, is ready to evaluate patients for ZEVASKYN treatment –

– Abeona Assist™ comprehensive patient services program in place to offer personalized support for eligible patients and their families throughout ZEVASKYN treatment journey –

CLEVELAND and CHICAGO, May 14, 2025 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO) and Ann & Robert H. Lurie Children’s Hospital of Chicago today announced that Lurie Children’s is now activated as the first Qualified Treatment Center (QTC) for ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets. This groundbreaking therapy will be used to treat wounds associated with recessive dystrophic epidermolysis bullosa (RDEB) – a rare skin disorder characterized by severe, painful wounds that can lead to systemic complications impacting the length and quality of life. Lurie Children’s has completed QTC start-up activities enabling it to begin patient identification for scheduling of ZEVASKYN treatment. Treatments are expected to begin in the third quarter of 2025.

On April 29, 2025, Abeona announced approval from the U.S. Food and Drug Administration (FDA) for ZEVASKYN as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB. There is no cure for RDEB and ZEVASKYN is the only FDA-approved product to treat RDEB wounds with a single application.

“Lurie Children’s is a top-ranked hospital, known for its expertise in treating patients with epidermolysis bullosa, and we are pleased to announce that ZEVASKYN is now commercially available with the activation of Lurie Children’s as our first QTC,” said Madhav Vasanthavada, Ph.D., M.B.A., Chief Commercial Officer of Abeona. “This site activation comes just a few weeks after the FDA approval of ZEVASKYN, underscoring the collaborative relationship between Lurie Children’s and Abeona, and our shared conviction that ZEVASKYN is an important treatment option for people living with RDEB.”

Amy Paller, MD, head of the epidermolysis bullosa research and care program at Lurie Children’s, and Chair of the Department of Dermatology at Northwestern University Feinberg School of Medicine, said, “Lurie Children’s is proud to be the first qualified treatment site in the U.S. to offer this groundbreaking treatment for RDEB patients. Grafting gene-corrected cellular sheets onto chronically open wounds of patients with RDEB promises the potential to provide long-term healing of wounds, reduction in pain and reduced risk of infection.”

Lurie Children’s has been a center for excellence for genetic skin diseases for more than 30 years. As one of the largest North American centers for epidermolysis bullosa (EB), caring for more than 150 affected children and adults, Lurie Children’s has been a member of the EB Clinical Research Consortium since its inception and continues to conduct cutting-edge bench and clinical research to better understand the disease and find new treatment options. For more information about receiving ZEVASKYN treatment at Lurie Children’s, email the Gene and Cellular Medicine Program at [email protected].

Lurie Children’s has been providing FDA-approved gene therapies since 2019, and the program is actively growing. Currently, gene therapies are available for neuromuscular disorders, eye disorders, and cancer and blood disorders. ZEVASKYN will be the tenth gene therapy offered at Lurie Children’s, with the first patient expected to be biopsied in July 2025 and to receive this treatment in August 2025.

Abeona is committed to enabling access to ZEVASKYN for eligible patients in the U.S. and has deployed services to provide information and resources to make informed decisions about treatment with ZEVASKYN for RDEB wounds. Abeona’s comprehensive patient support program, Abeona Assist™, offers personalized support, including helping patients understand their insurance benefits and financial assistance options, and providing travel and logistical assistance. For more information on how to access ZEVASKYN and learn about patient support services offered through Abeona Assist, visit www.abeonaassist.com, call 1-855-ABEONA-1 (1-855-223-6621) or email [email protected].

About Recessive Dystrophic Epidermolysis Bullosa

Recessive dystrophic epidermolysis bullosa (RDEB), a rare blistering disorder without a cure, is characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in both copies of the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin.

About ZEVASKYN™ (prademagene zamikeracel) gene-modified cellular sheets or pz-cel

ZEVASKYN is the first and only autologous cell sheet-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin disease caused by a defect in both copies of the COL7A1 gene resulting in the inability to produce functional type VII collagen. Without functional type VII collagen and anchoring fibrils, the skin is fragile and blisters easily, leading to wounds that continually open and close, or fail to heal altogether. Patients often have large open wounds that can lead to serious life-threatening complications. ZEVASKYN incorporates the functional type VII collagen-producing COL7A1 gene into a patient’s own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds. ZEVASKYN has demonstrated clinically meaningful wound healing and pain reduction with a single surgical application. For more information, visit www.ZEVASKYN.com and www.AbeonaAssist.com.

Indication

ZEVASKYN^TM (prademagene zamikeracel) is an autologous cell sheet-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

Important Safety Information

• Serious allergic reactions to ZEVASKYN can occur. Patients should get medical help right away if they experience symptoms like itching, swelling, hives, difficulty breathing, runny nose, watery eyes, or nausea. In rare cases, a severe reaction called anaphylaxis may happen.
• There is a potential risk that treatment with ZEVASKYN may contribute to the development of cancer because of how the therapy works. Patients should be monitored for the rest of their lives to check for any signs of cancer.
• ZEVASKYN is made using human and animal materials. Although these materials are tested before use, the risk of passing on infections cannot be eliminated.
• The most common side effects are pain from the procedure and itching.

This is not a complete list of side effects. Patients should call their care team for medical advice about side effects. Side effects may be reported to Abeona at 1-844-888-2236 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

See full Prescribing Information.

About Lurie Children’s Gene and Cellular Medicine Program

Lurie Children’s Gene and Cellular Medicine Program is committed to advancing treatment of conditions that are amenable to gene and cell-based therapy. Our program looks to bring hope to patients who live with various blood disorders, neurogenerative conditions, and other genetic disorders. Our integrated team of clinical researchers, infusion center experts, and other healthcare professionals collaborates to provide a seamless continuum of care for our pediatric patients and their families.

About Lurie Children’s

Ann & Robert H. Lurie Children’s Hospital of Chicago is a nonprofit organization committed to providing access to exceptional care for every child. It is the only independent, research-driven children’s hospital in Illinois and one of less than 35 nationally. This is where the top doctors go to train, practice pediatric medicine, teach, advocate, research and stay up to date on the latest treatments. Exclusively focused on children, all Lurie Children’s resources are devoted to serving their needs. Research at Lurie Children’s is conducted through Stanley Manne Children’s Research Institute, which is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s is the pediatric training ground for Northwestern University Feinberg School of Medicine. It is ranked as one of the nation’s top children’s hospitals by U.S. News & World Report.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company’s fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company’s development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit www.abeonatherapeutics.com.

ZEVASKYN^TM, Abeona Assist^TM, Abeona Therapeutics^®, and their related logos are trademarks of Abeona Therapeutics Inc.

Forward-Looking Statements

This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, our ability to commercialize ZEVASKYN, the therapeutic potential of ZEVASKYN, whether the unmet need and market opportunity for ZEVASKYN are consistent with the Company’s expectations, continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor and Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
[email protected]

BOSTON, May 14, 2025 (GLOBE NEWSWIRE) — Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company developing next-generation ADC therapeutics for difficult-to-treat cancers, today announced that Lara S. Sullivan, M.D., President, Chief Executive Officer and Chief Medical Officer, will participate in fireside chats and be available for one-on-one meetings with investors at the following two upcoming investor conferences.

• 2025 RBC Capital Markets Global Healthcare Conference in New York, NY on Wednesday, May 21 at 2:05 p.m. ET
• Jefferies Global Healthcare Conference in New York, NY on Thursday, June 5 at 4:55 p.m. ET

A live webcast and replay of the fireside chats will be available on the Events & Presentations page in the Investor Relations section of Pyxis Oncology’s website, ir.pyxisoncology.com.

About Pyxis Oncology, Inc.

Pyxis Oncology, Inc. is a clinical stage company focused on defeating difficult-to-treat cancers. The Company is efficiently building next generation therapeutics that hold the potential for monotherapy and combination indications. Its lead candidate, micvotabart pelidotin (MICVO, formerly PYX-201), has been evaluated in ongoing Phase 1 clinical studies in multiple types of solid tumors with a go-forward development focus on treating patients with recurrent and metastatic head and neck squamous cell carcinoma (R/M HNSCC) based on the strength of the HNSCC signal that emerged. Additionally, the Company initiated a Phase 1/2 combination study of MICVO and Merck’s anti-PD-1 therapy, KEYTRUDA^® (pembrolizumab), in patients with R/M HNSCC and other advanced solid tumors.

To learn more, visit www.pyxisoncology.com or follow us on Twitter and LinkedIn.

Pyxis Oncology Contact

Pamela Connealy
CFO and COO
[email protected]

GAITHERSBURG, Md., May 14, 2025 (GLOBE NEWSWIRE) — Altimmune, Inc. (Nasdaq: ALT), a late clinical-stage biopharmaceutical company developing novel peptide-based therapeutics for liver and cardiometabolic diseases, today announced that members of the Company’s management team will participate and be available for 1×1 meetings at the following investor conferences:

• H.C. Wainwright 3
  
  ^rd
  
  Annual BioConnect Investor Conference (New York, NY)
  Tuesday, May 20, 2025
  Fireside Chat at 10:00 a.m. Eastern Time

• Jefferies Global Healthcare Conference (New York, NY)
  Wednesday, June 4, 2025
  Fireside Chat at 9:55 a.m. Eastern Time

The sessions will be webcast and can be accessed by visiting the Events section of the Altimmune website.

About Altimmune

Altimmune is a late clinical-stage biopharmaceutical company focused on developing novel peptide-based therapeutics for liver and cardiometabolic diseases. The Company’s lead program is pemvidutide, a GLP-1/glucagon dual receptor agonist for the treatment of MASH, obesity, Alcohol Use Disorder (AUD) and Alcohol Liver Disease (ALD). For more information, please visit www.altimmune.com.

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Company Contact:

Greg Weaver
Chief Financial Officer
Phone: 240-654-1450
[email protected]

Investor Contact:

Lee Roth
Burns McClellan
Phone: 646-382-3403
[email protected]

Media Contact:

Jake Robison
Inizio Evoke Comms
Phone: 619-849-5383
[email protected]

This press release was published by a CLEAR® Verified individual.

Multiple long-term LM survivors in those patients receiving multiple doses of REYOBIQ

RNA sequencing data show early tumor apoptosis and activation of innate immune responses

Updated ReSPECT data presented at the 2025 Nuclear Medicine and Neurooncology Conference

HOUSTON, May 14, 2025 (GLOBE NEWSWIRE) — Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, announces the presentation of new data on its lead drug REYOBIQ™ (rhenium Re¹⁸⁶ obisbemeda) during both an oral presentation and a poster presented at the Nuclear Medicine and Neurooncology Conference. The meeting was held May 9-10, 2025 in Vienna, Austria.

“This newly presented data shows safety, clinical benefit and data supporting the underlying mechanism of action for REYOBIQ in patients with Leptomeningeal Metastases,” said Marc H. Hedrick, M.D., Plus Therapeutics President and Chief Executive Officer. “Furthermore, multiple doses of REYOBIQ were administered under compassionate use, mirroring how REYOBIQ may one day be used post approval and can contribute to long term survival in LM.”

The study, titled, “Rhenium Obisbemeda (REYOBIQ) in Leptomeningeal Metastases,” highlights additional data from the Company’s now complete Phase 1 ReSPECT-LM dose escalation trial. The presented data added further detail to the previously reported data from the submitted abstract, which demonstrated:

• Dose dependent increase in the average absorbed dose to the cranial and spinal subarachnoid space reaching 253Gy in Cohort 5
• Neuroimaging response data was available for 17 patients as of the data cutoff with five of those (29%) showing a partial response
• An additional eight patients showed stable disease by neuroimaging through day 112 for a Clinical Benefit Rate (complete response + partial response + stable disease) of 76% (13/17 – five partial responses and eight stable disease). Additionally, 87% of subjects demonstrated clinical response based on the physician evaluation (13/15 – 2 response and 11 stable disease)
• No dose limiting toxicity (DLT) observed in the first four cohorts, with a grade 4 DLT (thrombocytopenia), one in each of Cohorts 5 and 6
• RNA sequencing of LM cells showed early induction of apoptosis, with an innate immune response followed by an increase in T cells and an adaptive immune response by Day 28.
• In addition, of the seven patients who received a response of better than 80% in reduction of LM tumor cells in the cerebrospinal fluid, five survived at least one year following initial treatment.
• The study reports that three of those five were retreated via compassionate use.

Further details from the poster can be found here.

About Leptomeningeal Metastases (LM)
LM is a rare complication of cancer in which the primary cancer spreads to the cerebrospinal fluid (CSF) and leptomeninges surrounding the brain and spinal cord. All malignancies originating from solid tumors, primary brain tumors, or hematological malignancies have this LM complication potential with breast cancer as the most common cancer linked to LM, with 3-5% of breast cancer patients developing LM. Additionally, lung cancer, GI cancers and melanoma can also spread to the CSF and result in LM. LM occurs in approximately 5% of people with cancer and is usually terminal with 1-year and 2-year survival of just 7% and 3%, respectively. The incidence of LM is on the rise, partly because cancer patients are living longer and partly because many standard chemotherapies cannot reach sufficient concentrations in the spinal fluid to kill the tumor cells, yet there are no FDA-approved therapies specifically for LM patients, who often succumb to this complication within weeks to several months, if untreated.

About REYOBIQ™ (rhenium Re¹⁸⁶ obisbemeda)
REYOBIQ™ (rhenium Re¹⁸⁶ obisbemeda) is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. REYOBIQ™ has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. REYOBIQ™ is being evaluated for the treatment of recurrent glioblastoma and leptomeningeal metastases in the ReSPECT-GBM and ReSPECT-LM clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT).

About Plus Therapeutics

Headquartered in Houston, Texas, Plus Therapeutics, Inc. is a clinical-stage pharmaceutical company developing targeted radiotherapeutics for difficult-to-treat cancers of the central nervous system with the potential to enhance clinical outcomes. Combining image-guided local beta radiation and targeted drug delivery approaches, the Company is advancing a pipeline of product candidates with lead programs in leptomeningeal metastases (LM) and recurrent glioblastoma (GBM). The Company has built a supply chain through strategic partnerships that enable the development, manufacturing, and future potential commercialization of its products. For more information, visit https://plustherapeutics.com/.

Investor Contact

CORE IR
[email protected]

CARSON CITY, Nev., May 14, 2025 (GLOBE NEWSWIRE) — BioVie Inc. (NASDAQ: BIVI), (“BioVie” or the “Company”), a clinical-stage company developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders and advanced liver disease, today announced that it will host a virtual key opinion leader (KOL) event featuring Suzanne de la Monte, MD, MPH (Brown University Warren Alpert Medical School, Providence VA Medical Center) and Mark Stacy, MD (Medical University of South Carolina College of Medicine), who will join company management to discuss the unmet need and current treatment landscape for Parkinson’s disease on Wednesday, May 28, 2025 at 12:00 PM ET. To register, click here.

The event will provide an update on BioVie’s Phase 2 trial (SUNRISE-PD; sunrisepd.com) evaluating bezisterim (NE3107) for the treatment of Parkinson’s disease. Bezisterim is a novel, orally-administered small molecule that has been shown to reverse insulin resistance and selectively inhibit inflammation-driven ERK- and NF-κB-stimulated inflammatory mediators without inhibiting their homeostatic functions. Inflammation and the associated insulin resistance are important to the presentation of Parkinson’s symptoms. BioVie is currently enrolling patients recently diagnosed with Parkinson’s disease who have not yet begun treatment in order to measure bezisterim’s potential impact on clinical signs and effects on biomarkers that may correlate with disease-modification for the SUNRISE-PD trial.

A live question and answer session will follow the formal presentations.

About Suzanne de la Monte, MD, MPH

Suzanne de la Monte, MD, MPH is Professor in Pathology and Laboratory Medicine, Neurology, & Neurosurgery at the Warren Alpert Medical School of Brown University and Chief of Pathology & Laboratory Medicine at the Providence VA Medical Center. Dr. de la Monte is also a medical staff member at the Rhode Island Hospital and Women and Infants Hospital of Rhode Island and formerly a research fellow at the National Institutes of Health. She received residency training in Anatomic and Pediatric Pathology at Johns Hopkins and fellowship training in Neuropathology at the Massachusetts General Hospital (MGH). Dr. de la Monte leads programs in basic, translational, and clinical research on mechanisms and the neurological consequences of brain insulin resistance and metabolic dysfunction, and coined the term ‘Type 3 Diabetes’ which refers to the hypothesis that insulin resistance in the brain causes neurodegenerative disorders. She has over 300 peer-reviewed articles published.

About
Mark Stacy, MD

Mark Stacy, MD is a Professor in the College of Medicine at Medical University of South Carolina. He received his MD from the University of Missouri, Columbia, Missouri, and completed his residency training at Hahnemann (Drexel) University. Dr. Stacy also has a fellowship in Movement Disorders at Baylor College of Medicine. His prior positions include assistant professor at University of Missouri, Director of the Muhammad Ali Parkinson Research Center at the Barrow Neurological Institute in Phoenix, Arizona, and Director and subsequently Vice Dean of Parkinson Disease and Movement Disorders at Duke University. Dr. Stacy also completed a sabbatical in the Human Motor Control Section at the National Institutes of Health and served as Dean of Brody School of Medicine and Vice Chancellor of the Division of Health Sciences at East Carolina University from 2017 – 2021. He has published more than 300 journal articles and book chapters on the topics of clinical and neurobiological aspects, complications, and treatment options in Parkinson Disease; dystonia; Tourette’s syndrome; and movement disorders.

About Parkinson’s Disease

Parkinson’s disease (PD) is a progressive neurodegenerative disorder that primarily affects movement. It is characterized by the loss of dopamine-producing neurons in the substantia nigra, a region of the brain critical for motor control. Core symptoms include tremors, muscle rigidity, bradykinesia (slowness of movement), postural instability, and difficulties with speech.^1-2 Non-motor symptoms, such as cognitive impairment, mood disorders, and autonomic dysfunction, also significantly impact patients’ quality of life.³ In the early stages, symptoms are often mild and may include subtle tremors, slight movement difficulties, and changes in handwriting or facial expressions. As the disease progresses, motor symptoms become more pronounced, leading to difficulties with balance, speech, and daily activities, often requiring full-time care in later stages.⁴

Since the 1960s, when dopamine’s critical role in Parkinson’s disease was first identified, levodopa has remained the cornerstone of treatment, providing significant symptom relief by replenishing dopamine levels in the brain.^5-6 However, long-term use is associated with complications such as motor fluctuations—where symptom control becomes less stable—and dyskinesia, involuntary movements that can become debilitating.^7-9

Emerging research highlights the role of chronic inflammation and insulin resistance in the onset and progression of Parkinson’s disease. Neuroinflammation, driven by activated microglia and elevated levels of pro-inflammatory cytokines, contributes to oxidative stress and accelerates neuronal degeneration.^10-12 Additionally, insulin resistance, which impairs the brain’s ability to regulate glucose metabolism, has been linked to increased neurodegeneration and worsening motor symptoms.^13-14 These metabolic dysfunctions create a harmful cycle that exacerbates disease progression, underscoring the potential of anti-inflammatory and insulin-sensitizing therapies as new avenues for treatment.

About Bezisterim

Bezisterim (NE3107) is an orally bioavailable, blood-brain barrier (BBB)-permeable modulator of inflammation and insulin-sensitizer. In addition, it is not immunosuppressive and has a low risk of drug-drug interaction. By binding to ERK and selectively modulating NFκB activation and TNF-α production, BioVie believes that bezisterim may offer clinical improvements in several disease indications, including Alzheimer’s disease, Parkinson’s disease and long COVID.

In Parkinson’s disease, BioVie is currently enrolling patients in the Phase 2 SUNRISE-PD clinical trial evaluating the safety and efficacy of bezisterim on motor and non-motor symptoms in patients who have not been treated with carbidopa/levodopa, with topline data expected in late 2025 or early 2026. A previous Phase 2 study of bezisterim in Parkinson’s disease (NCT05083260) completed in 2022, and data presented at the AD/PD™ 2023 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders in Gothenburg, Sweden in March 2023 showed significant improvements in “morning on” symptoms and clinically meaningful improvement in motor control in patients treated with a combination of bezisterim and levodopa versus patients treated with levodopa alone, and no drug-related adverse events.

In long COVID, bezisterim has the potential to reduce neurological symptoms including fatigue and cognitive dysfunction. Persistently circulating viral spike proteins are believed to trigger TLR-4 driven activation of NFκB and the subsequent expression of inflammatory cytokines (IL-6, TNF, IFNg). BioVIe’s Phase 2 ADDRESS-LC study, is a randomized (1:1), placebo-controlled, multicenter trial in approximately 200 patients to evaluate the safety, tolerability and potential efficacy of 3 months of treatment with bezisterim to reduce the neurocognitive symptoms associated with long COVID, including difficulty concentrating or remembering things (“brain fog”) and fatigue.

In Alzheimer’s disease, BioVie conducted and reported efficacy data on its Phase 3 randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate bezisterim in patients who have mild-to-moderate Alzheimer’s disease (NCT04669028) in 2023. Results of a Phase 2 investigator-initiated trial (NCT05227820) showing bezisterim-treated patients experienced improved cognition and biomarker levels were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) annual conference in December 2022. An estimated six million Americans suffer from Alzheimer’s disease.

About BioVie Inc.

BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders (Long COVID, Alzheimer’s disease and Parkinson’s disease) and advanced liver disease. In neurodegenerative disease, the Company’s drug candidate bezisterim inhibits inflammatory activation of extracellular signal-regulated kinase and the transcription factor nuclear factor-kB, and the associated neuroinflammation and insulin resistance but not ERK and NFkB homeostatic functions (e.g., insulin signaling and neuron growth and survival). Both neuroinflammation and insulin resistance are drivers of AD and PD. Persistent systematic inflammation and neuroinflammation are key features in patients with neurological symptoms of Long COVID. In liver disease, the Company’s Orphan drug candidate BIV201 (continuous infusion terlipressin), with FDA Fast Track status, is being evaluated and discussed with guidance received from the FDA regarding the design of Phase 3 clinical testing of BIV201 for the reduction of further decompensation in participants with liver cirrhosis and ascites. The active agent is approved in the U.S. and in about 40 countries for related complications of advanced liver cirrhosis. For more information, visit www.bioviepharma.com .

References

1 Cleveland Clinic. Parkinson’s Disease. Last reviewed: 2022 Apr 15.
2 Jankovic J. J Neurol Neurosurg Psychiatry. 2008;79(4):368-376.
3 Postuma RB, Berg D, Stern M, et al. Mov Disord. 2015;30(12):1591-1601.
4 Kalia LV and Lang AE. Lancet. 2015;386(9996):896-912.
5 Hornykiewicz O. Angew Chem Int Ed. 2002;41(17):2934-2941.
6 Olanow CW, Obeso JA and Stocchi F. Lancet Neurol. 2006;5(8):677-687.
7 Ahlskog JE and Muenter MD. Mov Disord. 2001;16(3):448-458.
8 Espay AJ, Morgante F, Merola A, et al. Ann Neurol. 2018;84(6):797-811.
9 Cilia R and Akpalu A. J Neural Transm. 2020;127(5):889-916.
10 Jurcau A, Andronie-Cioara FL, Nistor-Cseppento DC, et al. Int J Mol Sci. 2023;24:14582.
11 Pajares M, Rojo AI, Manda G, et al. Cells. 2020;9:1687.
12 Isik S, Kiyak BY, Akbayir R, et al. Cells. 2023;12:1012.
13 Zagare A, Hemedan A, Almeida C, et al. Mov Disord. 2025;40(1):67-76.
14 Ruiz-Pozo VA, Tamayo-Trujillo R, Cadena-Ullauri S, et al. Nutrients. 2023;15(16):3585.

Forward-Looking Statements

This press release contains forward-looking statements, which may be identified by words such as “expect,” “look forward to,” “anticipate” “intend,” “plan,” “believe,” “seek,” “estimate,” “will,” “project” or words of similar meaning. Although BioVie Inc. believes such forward-looking statements are based on reasonable assumptions, it can give no assurance that its expectations will be attained. Actual results may vary materially from those expressed or implied by the statements herein due to the Company’s ability to successfully raise sufficient capital on reasonable terms or at all, available cash on hand and contractual and statutory limitations that could impair our ability to pay future dividends, our ability to complete our pre-clinical or clinical studies and to obtain approval for our product candidates, our ability to successfully defend potential future litigation, changes in local or national economic conditions as well as various additional risks, many of which are now unknown and generally out of the Company’s control, and which are detailed from time to time in reports filed by the Company with the SEC, including quarterly reports on Form 10-Q, reports on Form 8-K and annual reports on Form 10-K. BioVie Inc. does not undertake any duty to update any statements contained herein (including any forward-looking statements), except as required by law.

For Investor Relations Inquiries:

Bruce Mackle
Managing Director, LifeSci Advisors, LLC
[email protected]

For Media Relations Inquiries:

Melyssa Weible
Managing Partner, Elixir Health Public Relations
[email protected]

• Clear Improvement in Patient’s Skin Appearance Observed in Study after 12 weeks Compared to Baseline
• Key endpoints including Investigator’s Global Assessment (IGA), Modified Ichthyosis Area Severity (M-IASI) and Children’s Dermatology Life Quality Index (CDLQI) all demonstrated improvement from baseline
• QRX003 is being well tolerated and no adverse events have been reported
• Patient is expected to continue to be treated with QRX003 to assess progress
• Company continues to advance QRX003 in its late-stage Netherton Syndrome clinical studies
  

ASHBURN, Va., May 14, 2025 (GLOBE NEWSWIRE) — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, today announces positive initial clinical data from its ongoing Investigator Pediatric Peeling Skin Syndrome clinical study.

As the table below illustrates, after 12 weeks of treatment, clear evidence of skin healing in the area treated with QRX003 compared to baseline was observed.

End Point	Baseline	12 weeks (End of treatment period)
M-IASI*	36	12
IGA**	4 (Severe)	2 (Mild)
CDQLI***	19	11

*

M-IASI

: Modified Ichthyosis Area of Severity Index, a score used to assess the severity and extent of skin symptoms associated with ichthyosis. Lower scores indicate improvement.

**

IGA

: Investigator’s Global Assessment, which uses descriptive categories (e.g., clear, mild, moderate, severe) to evaluate the overall severity of disease symptoms.

***

CDQLI

: The Children’s Dermatology Life Quality Index is a validated clinical tool designed for children aged 4-15 that is used to measure the impact of their skin disease on a child’s quality of life in terms of symptoms, leisure activities, sleep, school, personal relationship and treatment. The scale for the CDQLI is 0-30.

QRX003 is being well tolerated by the patient and no adverse events have been reported. As a result of these positive initial results, the patient is expected to continue to be treated with QRX003 with a further clinical assessment by the investigator scheduled after 24 weeks of treatment.

Quoin CEO, Dr. Michael Myers, said, “We are very pleased to announce such positive initial data across a number of clinical endpoints for the pediatric subject in this study, which we believe may be the first formal clinical study for this disease. The two grade improvement, which is accepted as being clinically meaningful in both the Investigator’s Global Assessment (IGA) (severe to mild) and the M-IASI (moderate to clear) after 12 weeks of QRX003 application is very encouraging. We fully support the investigator’s decision to continue treatment. In addition, the validated CDQLI questionnaire results to date indicate that the pediatric subject in the study is experiencing a distinct positive improvement in their quality of life as a result of ongoing treatment with QRX003. Consistent with what we are observing in our Netherton Syndrome studies, QRX003 is well tolerated with no adverse events reported. We look forward to expanding this study to include additional pediatric subjects in other countries and to advancing the clinical development of the product for this disease. There is currently no approved treatment or cure for peeling skin syndrome and there are no clinical studies listed on clinicaltrials.gov as actively recruiting and dosing subjects, thus presenting a further opportunity for Quoin to potentially achieve the first regulatory approval for another rare genetic disease. We are also continuing to advance QRX003 in our late-stage Netherton Syndrome clinical studies.”

About Peeling Skin Syndrome (PSS)
Generalized inflammatory peeling skin syndrome (PSS) is a rare autosomal recessive genodermatosis caused by loss-of-function disease-causing variants of the corneodesmosin gene (CDSN), resulting in excessive shedding of the superficial layers of the epidermis. Patients generally suffer from a variety of conditions including severe pain and chronic pruritus (itch). There is currently no approved treatment for PSS, and patients try to manage symptoms using over-the-counter emollients.

About Quoin Pharmaceuticals Ltd.

Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. For more information, visit: www.quoinpharma.com or LinkedIn for updates.

Cautionary Note Regarding Forward Looking Statements

The Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances, such as “expect,” “intend,” “hope,” “plan,” “potential,” “anticipate,” “look forward,” “believe,” “may,” and “will,” among others. All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the patient continuing to be treated with QRX003 to assess progress; Quoin’s Pediatric Peeling Skin Syndrome clinical study being the first formal clinical study for this disease, expanding the study to include additional pediatric subjects in other countries, advancing the clinical development of a product for Peeling Skin Syndrome, achieving the first regulatory approval for another rare genetic disease, advancing QRX003 in Quoin’s late-stage Netherton Syndrome clinical studies and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company’s ability to pursue its regulatory strategy; the Company’s ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company’s ability to complete clinical trials on time and achieve desired results and benefits as expected; and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law.

For further information, contact:

Investor Relations

PCG Advisory
Jeff Ramson
[email protected]
(646) 863-6341

NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) — Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today announced that its President & Chief Executive Officer, Christopher U. Missling, PhD, will participate at both the H.C. Wainwright 3rd Annual BioConnect Investor Conference at NASDAQ and H.C. Wainwright 6^th Annual Neuro Perspectives Conference. Both conferences will take place in New York City (NY).

• H.C. Wainwright 3rd Annual BioConnect Investor Conference at NASDAQ
  
  When: Tuesday, May 20^th, 2025 at 2:30 pm ET.
  A webcast is available to clients of the firm at https://hcwevents.com/.

• H.C. Wainwright 6
  
  ^th
  
   Annual Neuro Perspectives Conference
  
  When: Tuesday, June 17^th, 2025 at 7:00 pm ET.
  A webcast is available to clients of the firm at https://hcwevents.com/.

About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer’s disease, Parkinson’s disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX^®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX^®2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX^®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX^®2-73 for the treatment of Parkinson’s disease. We believe that ANAVEX^®3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX^®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter,Facebook, Instagram, and LinkedIn.

Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

For Further Information:

Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: [email protected]

Investors:

Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: [email protected]

WILMINGTON, N.C., May 14, 2025 (GLOBE NEWSWIRE) — nCino, Inc. (NASDAQ: NCNO), the leading provider of intelligent, best-in-class banking solutions, will report financial results for its first quarter ended April 30, 2025, after the market close on Wednesday, May 28, 2025. nCino will host a conference call and webcast that day at 4:30 p.m. ET to discuss its financial results.

Event: nCino’s First Quarter Fiscal Year 2026 Financial Results Conference Call
Date and Time: Wednesday, May 28, 2025 at 4:30 p.m. ET
Webcast Link: https://investor.ncino.com/
Replay: A webcast replay will be available on the Investor Relations section of nCino’s website following the call.

About nCino

nCino (NASDAQ: NCNO) is powering a new era in financial services. The Company was founded to help financial institutions digitize and reengineer business processes to boost efficiencies and create better banking experiences. With over 2,700 customers worldwide – including community banks, credit unions, independent mortgage banks, and the largest financial entities globally – nCino offers a trusted platform of best-in-class, intelligent solutions. By integrating artificial intelligence and actionable insights into its platform, nCino is helping financial institutions consolidate legacy systems to enhance strategic decision-making, improve risk management, and elevate customer satisfaction by cohesively bringing together people, AI and data. For more information, visit www.ncino.com.    

INVESTOR CONTACT

Harrison Masters
[email protected]

Media Contacts  
Natalia Moose 
[email protected]  

Forward-Looking Statements:

This press release contains forward-looking statements about nCino’s financial and operating results, which include statements regarding nCino’s future performance, outlook, guidance, the benefits from the use of nCino’s solutions, our strategies, and general business conditions. Forward-looking statements generally include actions, events, results, strategies and expectations and are often identifiable by use of the words “believes,” “expects,” “intends,” “anticipates,” “plans,” “seeks,” “estimates,” “projects,” “may,” “will,” “could,” “might,” or “continues” or similar expressions and the negatives thereof. Any forward-looking statements contained in this press release are based upon nCino’s historical performance and its current plans, estimates, and expectations and are not a representation that such plans, estimates, or expectations will be achieved. These forward-looking statements represent nCino’s expectations as of the date of this press release. Subsequent events may cause these expectations to change and, except as may be required by law, nCino does not undertake any obligation to update or revise these forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially including, but not limited to risks associated with (i) adverse changes in the financial services industry, including as a result of customer consolidation or bank failures; (ii) adverse changes in economic, regulatory, or market conditions, including as a direct or indirect consequence of higher interest rates; (iii) risks associated with acquisitions we undertake, (iv) breaches in our security measures or unauthorized access to our customers’ or their clients’ data; (v) the accuracy of management’s assumptions and estimates; (vi) our ability to attract new customers and succeed in having current customers expand their use of our solution, including in connection with our migration to an asset-based pricing model; (vii) competitive factors, including pricing pressures and migration to asset-based pricing, consolidation among competitors, entry of new competitors, the launch of new products and marketing initiatives by our competitors, and difficulty securing rights to access or integrate with third party products or data used by our customers; (viii) the rate of adoption of our newer solutions and the results of our efforts to sustain or expand the use and adoption of our more established solutions; (ix) fluctuation of our results of operations, which may make period-to-period comparisons less meaningful; (x) our ability to manage our growth effectively including expanding outside of the United States; (xi) adverse changes in our relationship with Salesforce; (xii) our ability to successfully acquire new companies and/or integrate acquisitions into our existing organization; (xiii) the loss of one or more customers, particularly any of our larger customers, or a reduction in the number of users our customers purchase access and use rights for; (xiv) system unavailability, system performance problems, or loss of data due to disruptions or other problems with our computing infrastructure or the infrastructure we rely on that is operated by third parties; (xv) our ability to maintain our corporate culture and attract and retain highly skilled employees; and (xvi) the outcome and impact of legal proceedings and related fees and expenses.