Collagen Diseases

CLEVELAND, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the Company has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or Agency) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). “We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance,” said Vish Seshadri, Chief Executive Officer of Abeona. “We have incorporated the Agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the Complete Response Letter, including observations from the completed Pre-License Inspection of our manufacturing facility. Upon acceptance of the BLA, we look forward to assisting the FDA in the completion of its review.” The BLA resubmission follows the Company’s Type A meeting in August 2024, where Abeona aligned with the FDA on the content of the resubmission, including additional information to satisfy all Chemistry Manufacturing and Controls (CMC) requirements outlined in the Complete Response Letter (CRL) received in April 2024. In the CRL, the FDA required that certain additional information needed to satisfy CMC requirements be provided before the application could be approved. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379). Upon acceptance of the BLA resubmission, Abeona expects the FDA to set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA is either two or six months from the resubmission date depending on the FDA's classification of a resubmission as Class 1 or Class 2, respectively. The FDA’s decision on acceptance of a BLA resubmission is typically made within 14 calendar days following the resubmission. About prademagene zamikeracel (pz-cel) Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene therapy, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. As a result of this defect, cells are unable to express functional collagen VII protein which is needed to form anchoring fibrils that bond the epidermis to the dermis. Lack of anchoring fibrils leads to fragile skin that blisters easily and patients suffer from years of painful wounds, itch and increased risk of infection and squamous cell carcinoma. Pz-cel is made from patients' own skin cells that are genetically corrected with a functional COL7A1 gene integrated into the skin cells’ genome to express collagen VII. These gene-corrected cells are expanded to form keratinocyte sheets to cover wound areas in a single surgical application. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features 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. 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, the timing and outcome of the FDA’s review of our BLA resubmission for pz-cel; the FDA’s grant of a Priority Review Voucher upon pz-cel approval; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with 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.

Plus, news about Astellas, Clover, ElevateBio, Monopar, Idorsia and Neurocrine Biosciences: Vera Therapeutics plans $300M offering: The California biotech is selling shares after presenting Phase 2b data for an experimental drug called atacicept over the weekend. The company shared 96-week data for patients with immunoglobulin A nephropathy. — Kyle LaHucik Abeona Therapeutics resubmits rare skin disease therapy: Back in April, the FDA rejected Abeona’s cell therapy, called pz-cel, requesting more information about manufacturing. Abeona is developing pz-cel for recessive dystrophic epidermolysis bullosa, a rare genetic skin disease. — Lei Lei Wu Astellas yanks Izervay’s submission in Europe: The company said it withdrew its marketing authorization application for Izervay, a C5 protein inhibitor for geographic atrophy secondary to age-related macular degeneration. Astellas did not detail feedback from the Committee for Medicinal Products for Human Use, but said it is still “confident” in Izervay’s risk-benefit profile and will continue to engage with regulators in Europe and elsewhere. There are currently no treatments approved for GA secondary to AMD outside of the US, where Izervay was greenlit in August of last year. — Ayisha Sharma Clover releases early RSV vaccine data: The Shanghai-based biotech said its bivalent vaccine candidate, SCB-1019, produced “comparable” levels of RSV neutralizing antibodies to GSK’s Arexvy at 28 days in a Phase 1 test. Based on these data, Clover plans to advance the asset into studies focused on re-vaccination next year. — Ayisha Sharma ElevateBio lays off 4%: The genetic medicines company, which employs about 500 people, is ending its preclinical cellular engineering work, a spokesperson told Endpoints News on Monday. The Waltham, MA-based biotech laid off about 13% of its staff in October 2023. It’s one of the most well-funded private biopharma startups after having raised about $1.25 billion, including its $401 million Series D last year. Partnerships with companies like Novo Nordisk, Moderna, Kyverna and Affini-T are unaffected, the spokesperson confirmed. — Kyle LaHucik Monopar Therapeutics prices $19.2M offering: The Chicago-area biotech sold shares to Janus Henderson, RA Capital and others. A few days ago, Monopar licensed a Wilson disease drug candidate from AstraZeneca’s Alexion. — Kyle LaHucik Idorsia ends work with Neurocrine Biosciences: The companies were developing Idorsia’s T-type calcium channel blocker for patients with a rare form of pediatric epilepsy. The treatment failed a Phase 2 study in 2022, and further analysis led to the decision to stop work. — Jaimy Lee

Vivoryon Therapeutics N.V. Presents Outstanding Phase 2b Results of Varoglutamstat on Kidney Function at ASN Kidney Week 2024 Selected for late-breaking oral presentation at ASN kidney week, the world’s premier nephrology meetingResults presented show a statistically significant and clinically meaningful improvement of the prospectively defined kidney function parameter eGFR1 by 3.4mL/min/year (p<0.0001) in the varoglutamstat arm compared to placebo Results in the subgroup of patients with diabetes2 showed an 8.2mL/min/year difference in favor of varoglutamstat (p=0.02)The results were consistent in several sensitivity analyses including using the CKD-EPI 2021 formula for both creatinine and cystatin-CVaroglutamstat demonstrated an excellent safety and tolerability profile and there were no signs of increased proteinuriaA new Phase 2 study is in planning to confirm the effect in patients with DKD3 stage 3b and 4 Halle (Saale) / Munich, Germany, October 26, 2024 – Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on the discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, today announced highlights from a late-breaking oral presentation held yesterday, October 25, 2024, at the American Society of Nephrology (ASN) Kidney Week 2024 in San Diego, California. The presentation by the Company’s CEO, Frank Weber, M.D. titled “Varoglutamstat Increases Glomerular Filtration in Elderly Patients without Signs of Proteinuria and Potentially Offers a New Approach to Treat Diabetic Kidney Disease (DKD)” featured Phase 2 clinical study data substantiating the opportunity to further develop varoglutamstat, Vivoryon’s Phase 2 investigational medicine with the potential to improve kidney function, in people with kidney disease. “We’re privileged to have been accepted to share the exciting results of varoglutamstat on kidney function with so many scientific and medical experts in the kidney field. Varoglutamstat showed statistically significant and clinically meaningful improvements of eGFR versus placebo and a sustained improvement of eGFR above baseline, potentially indicating partial recovery of the kidney. We are grateful for many fruitful discussions and extremely encouraged by the positive reactions we received from the community,” said Frank Weber, M.D., CEO of Vivoryon. “The efficacy and safety data presented at ASN represent a unique profile for an oral product for treating kidney disorders and guide the future development of varoglutamstat. Our primary focus is delivering a much-needed novel treatment option for patients suffering from DKD. Beyond this, we see potential for varoglutamstat across a broad range of kidney diseases including rare diseases affecting kidney function, such as Fabry disease and Alport syndrome.” Presentation Highlights Background: Varoglutamstat is a specific and selective inhibitor of glutaminyl cyclases and confers a novel mechanism of action that attenuates inflammation and fibrosis through reduction of pyroglutamized versions of chemokines and pro-fibrotic peptides, thereby positively impacting kidney function.Assessment of progression of kidney dysfunction as measured by the eGFR slope4 was included prospectively in VIVIAD (NCT04498650), Vivoryon’s Phase 2b multicenter, randomized, double-blind, placebo-controlled, parallel group dose-finding study in 259 patients in Alzheimer’s disease.The average age of participants in VIVIAD was >68 years, dosing was twice-daily with either 300mg or 600mg varoglutamstat, or placebo and treatment duration was 48-96 weeks; key kidney-related study features included a mean baseline eGFR of ~80mL/min/1.73m2, eGFR, urine dipstick and vital signs measured every 12 weeks; the diabetes subgroup comprised ~12% of VIVIAD patients. Compelling efficacy and safety data in elderly people at risk for kidney disease: eGFR improved significantly for varoglutamstat compared to placebo and above baseline in both total population and diabetes subgroup, with the latter revealing a substantially higher treatment effect5 of >8.2mL/min/1.73m2/year (p=0.02; varoglutamstat n=20 / placebo n=12) compared to the overall VIVIAD study population (3.4mL/min/1.73m2/year (p<0.001; varoglutamstat n=141 / placebo n=117)).Results and effect size were consistent using a set of diverse and validated methods for eGFR assessment (2021 CKD-EPI cystatin C, 2021 CKD-EPI creatinine-cystatin C 2021 CKD-EPI creatinine, MDRD6).Urine dipstick analysis showed no evidence of increased proteinuria in the treatment group compared to placebo, with the majority of study participants having no proteinuria through all time points measured in the study.Robust safety data confirmed varoglutamstat’s excellent safety profile consistent across two years study duration with no adverse kidney effects and no meaningful differences observed in renal and metabolic systems adverse events in both total population and diabetes subgroup. Next steps: Placebo-controlled Phase 2 study in stage 3b/4 DKD patients on top of SoC in planning to confirm the results to date and investigate additional endpoints including albuminuria/proteinuria, inflammation and fibrosis-related biomarkers.Pre-clinical investigation of additional rare/orphan kidney disorders (Alport syndrome, Fabry disease). 1 Estimated glomerular filtration rate (eGFR), a validated measure of kidney function, was calculated as a slope analysis across two years taking all available data into account. 2 Diabetes subgroup defined as patients having at baseline either medical history of diabetes (type 1 or 2) and/or comedication with drugs used in diabetes and/or untreated with an HbA1c > 6.5%. 3 The timing and execution of the planned Phase 2 study in diabetic kidney disease is subject to additional funding / partnership. 4 Measuring the eGFR slope via random coefficients (RC) analysis was the primary efficacy endpoint in recent FDA approvals in CKD, as well as in many ongoing Phase 3 studies. 5 Treatment effect – the between-group difference in eGFR slope between varoglutamstat and placebo. 6 Estimated glomerular filtration rate based on creatinine and calculated using modification of diet in renal disease (MDRD) method. ### Varoglutamstat in Kidney DiseaseVaroglutamstat (PQ912) is a proprietary, potent and selective inhibitor of human glutaminyl cyclases QPCT and QPCTL with therapeutic potential in indications including inflammatory and fibrotic diseases, neurodegenerative diseases, cancer and others. Initially advancing development aiming to treat Alzheimer’s disease (AD), varoglutamstat has been investigated in a number of different clinical studies, all of which have consistently demonstrated a favorable safety and tolerability profile both in healthy volunteers and patients with AD. Based on the known anti-inflammatory activity of varoglutamstat, the protocol for the Phase 2 VIVIAD study in AD, which was completed in the first half of 2024, included the investigation of kidney function and measurement of biomarkers of kidney inflammation and fibrosis to explore the role of QPCT/L inhibition on kidney function. Although patients in VIVIAD were selected for their AD status and not for their kidney function level, many of them had reduced kidney function due to age and/or comorbidities. Analysis showed a statistically significant benefit of varoglutamstat on a prospectively defined key kidney function endpoint (eGFR) and a significant reduction of the pro-inflammatory cytokine pE-CCL2. A substantially higher treatment benefit of varoglutamstat on eGFR was observed in a post-hoc diabetes subgroup, triggering plans to advance varoglutamstat into Phase 2 study in DKD, which is currently in planning. About Vivoryon Therapeutics N.V.Vivoryon is a clinical stage biotechnology company focused on developing innovative small molecule-based medicines. Driven by its passion for ground-breaking science and innovation, the Company strives to change the lives of patients in need suffering from severe diseases. The Company leverages its in-depth expertise in understanding post-translational modifications to develop medicines that modulate the activity and stability of proteins which are altered in disease settings. The Company has established a pipeline of orally available small molecule inhibitors for various indications including Alzheimer’s disease, inflammatory and fibrotic disorders, including of the kidney, and cancer. www.vivoryon.com. Vivoryon Forward Looking StatementsThis press release includes forward-looking statements, including, without limitation, those regarding the business strategy, management plans and objectives for future operations of Vivoryon Therapeutics N.V. (the “Company”), estimates and projections with respect to the market for the Company’s products and forecasts and statements as to when the Company’s products may be available. Words such as “anticipate,” “believe,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “project,” “predict,” “should” and “will” and similar expressions as they relate to the Company are intended to identify such forward-looking statements. These forward-looking statements are not guarantees of future performance; rather they are based on the Management’s current expectations and assumptions about future events and trends, the economy and other future conditions. The forward-looking statements involve a number of known and unknown risks and uncertainties. These risks and uncertainties and other factors could materially adversely affect the outcome and financial effects of the plans and events described herein. The Company’s results of operations, cash needs, financial condition, liquidity, prospects, future transactions, strategies or events may differ materially from those expressed or implied in such forward-looking statements and from expectations. As a result, no undue reliance should be placed on such forward-looking statements. This press release does not contain risk factors. Certain risk factors that may affect the Company’s future financial results are discussed in the published annual financial statements of the Company. This press release, including any forward-looking statements, speaks only as of the date of this press release. The Company does not assume any obligation to update any information or forward-looking statements contained herein, save for any information required to be disclosed by law. For more information, please contact: Investor ContactVivoryon Therapeutics N.V.Dr. Manuela Bader, Director IR & CommunicationEmail: IR@vivoryon.com Media ContactTrophic CommunicationsValeria Fisher or Verena SchossmannTel: +49 175 8041816 / +49 151 219 412 77Email: vivoryon@trophic.eu Attachment 20241026_Vivoryon_ASN_KidneyWeek_Data