Tillkännagivande • Jul 01
Abionyx Pharma Launches Largest Pivotal Clinical Trial for Cer-001 in Sepsis and Provides Updates on Cer-001 for Lcat Deficiency ABIONYX Pharma provided an update on the Combined General Meeting held at the company’s headquarters and the new phase of development beginning on June 30, 2026. Launch of the largest pivotal clinical trial for CER-001 in sepsis, the 3rd cause of death worldwide. Progress in regulatory and manufacturing phases toward approval of CER-001 for LCAT deficiency. The clinical development of CER-001 for sepsis; initiation of a pivotal Phase 2b clinical trial by the end of 2026, with the first results expected in the first half of 2028. Market access for CER-001 in LCAT deficiency; validation of the production of two GMP batches in 2027 for submission of a marketing authorization application to the EMA by early 2028. Adherence to clinical and GMP production timelines for sepsis and LCAT deficiency. Generating clinical data establishing CER-001 as the new standard of care for sepsis and LCAT deficiency. Leveraging and optimizing its recombinant apoA-I-based technology platform. Preparing for the next regulatory steps. For more than two decades, ABIONYX has been developing in-depth knowledge of apoA-I and its role in inflammatory and metabolic mechanisms. The data from its research and development programs now constitute a scientific asset designed to support the clinical development of CER-001, strengthen scientific collaborations, and open up new avenues for development. New Risk • Jun 18
New minor risk - Market cap size The company's market capitalization is less than US$100m. Market cap: €79.1m (US$90.7m) This is considered a minor risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 42% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (€40m net loss in 3 years). Share price has been volatile over the past 3 months (8.9% average weekly change). Revenue is less than US$5m (€4.1m revenue, or US$4.7m). Market cap is less than US$100m (€79.1m market cap, or US$90.7m). New Risk • May 27
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of German stocks, typically moving 8.7% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 42% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (€40m net loss in 3 years). Share price has been volatile over the past 3 months (8.7% average weekly change). Revenue is less than US$5m (€4.1m revenue, or US$4.8m). Tillkännagivande • May 27
ABIONYX Pharma SA has filed a Follow-on Equity Offering in the amount of €18.699502 million. ABIONYX Pharma SA has filed a Follow-on Equity Offering in the amount of €18.699502 million.
Security Name: Oridnary Shares
Security Type: Common Stock
Securities Offered: 7,056,416
Price\Range: €2.65
Transaction Features: Rights Offering Tillkännagivande • May 25
ABIONYX Pharma SA, Annual General Meeting, Jun 30, 2026 ABIONYX Pharma SA, Annual General Meeting, Jun 30, 2026. Location: 33 43 avenue georges pompidou bat d, balma France New Risk • Mar 28
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of German stocks, typically moving 8.7% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 48% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (€40m net loss in 3 years). Share price has been volatile over the past 3 months (8.7% average weekly change). Revenue is less than US$5m (€4.1m revenue, or US$4.7m). Tillkännagivande • Mar 27
ABIONYX Pharma Achieves Major Milestone in ApoA-I Biomanufacturing with Breakthrough in Synthetic Sphingomyelin Production ABIONYX Pharma announced a significant technological and industrial breakthrough combining high-performance apoA-I bioproduction with exclusive mastery of ultra-pure synthetic sphingomyelin, paving the way for a new class of sepsis therapeutics. As part of its strategy to enable large-scale industrial production of CER-001, ABIONYX Pharma has developed a new manufacturing process allowing: A threefold increase in sphingomyelin production yield, one of the two key components of CER-001; A synthetic component with exceptional purity and structural quality, meeting the most stringent pharmaceutical standards. All synthesis processes for this component are based on fully proprietary patents, providing ABIONYX Pharma with a major technological and competitive barrier. Sphingomyelin is a strategic lipid widely recognized for its structural and functional properties. Within the CER-001 biomedicine, it plays a central role: It stabilizes the ApoA-I protein in its optimal functional conformation; It gives CER-001 biological and therapeutic properties fully comparable to natural HDL found in the human body; It exhibits high intrinsic stability in pure form without added stabilizers—unlike conventional sphingomyelins—strengthening manufacturing robustness and pharmaceutical-grade quality. Sphingomyelin is also used as a key ingredient in other pharmaceutical applications, including advanced cosmetics, due to its ability to stabilize and protect biological structures. The use of 100% synthetic sphingomyelin, replacing traditional sources (egg or bovine), eliminates: Any risk of contamination; Variability in raw materials; Allergenic risks. With this achievement, the Company overcomes a major technological barrier in the control and production of CER-001. ABIONYX Pharma is advancing its platform in sepsis, a global medical emergency characterized by severe systemic dysregulation and a substantial unmet therapeutic need. The combination of highly purified apoA-I, proprietary synthetic sphingomyelin and optimized formulation technologies positions the company to deliver differentiated therapeutic approaches aimed at restoring essential biological functions in sepsis patients. This breakthrough in synthetic sphingomyelin production will also directly benefit the manufacturing of the two validation batches required ahead of Marketing Authorization (MAA) in LCAT deficiency. By ensuring high purity, reproducibility, and secured supply, this innovation significantly de-risks the production process and supports timely execution of these critical batches. These validation lots represent a key milestone for the Company, as they are essential to advancing CER-001 toward regulatory approval. This milestone reinforces ABIONYX Pharma’s positioning as a leader in advanced biomanufacturing within the biotech sector. The company demonstrates its ability to: Manufacture complex, high-value therapeutic proteins at scale; Control critical lipid components at an unprecedented level of purity; Integrate these elements into differentiated, functional drug formulations. This achievement validates the industrial scalability of the apoA-I platform, strengthens supply chain security, and supports upcoming clinical and regulatory milestones. It also creates new opportunities for strategic partnerships with global industry players. Reported Earnings • Mar 15
Full year 2025 earnings released Full year 2025 results: Revenue: €4.10m (down 9.9% from FY 2024). Net loss: €5.50m (loss widened 26% from FY 2024). Revenue is forecast to grow 68% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in Europe. New Risk • Mar 12
New minor risk - Revenue size The company makes less than US$5m in revenue. Total revenue: €4.3m (US$5.0m) This is considered a minor risk. Companies with a small amount of revenue are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 40% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (€26m net loss in 3 years). Revenue is less than US$5m (€4.3m revenue, or US$5.0m). Tillkännagivande • Dec 17
ABIONYX Pharma SA has filed a Follow-on Equity Offering in the amount of €1.799993 million. ABIONYX Pharma SA has filed a Follow-on Equity Offering in the amount of €1.799993 million.
Security Name: Oridnary Shares
Security Type: Common Stock
Securities Offered: 580,643
Price\Range: €3.1
Transaction Features: Subsequent Direct Listing Board Change • Nov 14
Less than half of directors are independent Following the recent departure of a director, there are only 3 independent directors on the board. The company's board is composed of: 3 independent directors. 5 non-independent directors. Independent Director Christmas Noël was the last independent director to join the board, commencing their role in 2017. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Tillkännagivande • Oct 22
ABIONYX Pharma Announces Study Published in Nature Confirms Genetic Causality of Apolipoprotein A-I (ApoA-I) in Sepsis ABIONYX Pharma announced the publication of a ground breaking study in Scientific Reports in Nature Portfolio titled "Plasma apolipoprotein A-I is a causal protective factor in sepsis. This landmark publication provides, for the first time, genetic proof of a causal linkage between higher plasma plasma apoA-I levels and a lower incidence of sepsis and lower mortality in patients who do develop sepsis. At the same time, the publication provides genetic validation of the well-studied mechanism of apoA-I's beneficial impact on sepsis - the sequestration of the bacterial lipid toxin, LPS, which is responsible for the manifestations of sepsis. The report provides evidence that apoA-I is also effective in reducing mortality in Gram positive sepsis driven by the bacterial toxin lipoteichoic acid. Importantly, the findings were replicated across three independent data sets, including both Caucasian and Asian sepsis sufferers. The structural protein which defines HDL is apoA-I. The study analyzed 442,601 participants from the UK Biobank, including 11,643 sepsis cases, and validated the findings across two large international cohorts. Results demonstrate that each standard deviation increase in plasma apoA- I levels reduces the incidence of sepsis by 13% and 28-day mortality by 27%. Using Mendelian randomization, the researchers confirmed that this protective effect is causal and independent of other lipid fractions (HDL-C, LDL-C, triglycerides). Mechanistically, higher apoA-I levels were linked to a reduction in circulating endotoxin (LPS) levels, reinforcing apoA-I's role as a central modulator of inflammatory response and innate immunity in sepsis. Board Change • Sep 02
Less than half of directors are independent Following the recent departure of a director, there are only 3 independent directors on the board. The company's board is composed of: 3 independent directors. 5 non-independent directors. Independent Director Christmas Noël was the last independent director to join the board, commencing their role in 2017. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Tillkännagivande • May 22
ABIONYX Pharma SA, Annual General Meeting, Jun 26, 2025 ABIONYX Pharma SA, Annual General Meeting, Jun 26, 2025. Location: 33 43 avenue georges pompidou bat d, balma France Board Change • Jan 03
Less than half of directors are independent Following the recent departure of a director, there are only 3 independent directors on the board. The company's board is composed of: 3 independent directors. 5 non-independent directors. Independent Director Christmas Noël was the last independent director to join the board, commencing their role in 2017. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Board Change • Dec 13
Less than half of directors are independent Following the recent departure of a director, there are only 3 independent directors on the board. The company's board is composed of: 3 independent directors. 5 non-independent directors. Independent Director Christmas Noël was the last independent director to join the board, commencing their role in 2017. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Tillkännagivande • Oct 25
ABIONYX Pharma RACERS Study Clinical Results in Brain-Fog Selected for Poster Presentation at the American Society of Nephrology (ASN) 2024 Annual Meeting Kidney Week ABIONYX Pharma announced that the Company's Phase 2b RACERS study in sepsis has been selected again as a poster presentation in the "AKI: Clinical, Outcome, and Trials- Epidemiology and Pathophysiology" poster session at the 2024 American Society of Nephrology Annual Meeting taking place October 23-27, 2024 in San Diego, California. The poster data will reveal exclusive new clinical results in brain-fog. CER-001 mitigates brain dysfunction by downregulating the Indolamine-2,3- dioxygenase 1 (IDO1) enzyme, a crucial mediator of the kynurenine pathway (KP) in sepsis. The IDO1 is significantly upregulated during inflammation and this has been linked to cognitive dysfunction. RACERS new clinical data suggest that IDO-1 is downregulated after treatment, thus reducing the production of potential neurotoxic metabolites. Clinical results showed significantly increased levels of tryptophan during treatment (p=0.0037), accompanied by observed increases in the neuroprotective Kynurenic Acid (KYNA), supporting the hypothesis of different regulation of tryptophan metabolism leading to neuroprotection. CER-001 is an engineered HDL particle which contains recombinant human apolipoprotein A-I (apoA-I), complexed with phospholipids. HDL particles have been shown to be highly effective scavengers of bacterial endotoxins, such as lipopolysaccharide (LPS), with the ability to inactivate LPS and target it for removal by the liver. In addition, its significant capacity to uptake cholesterol and lipids accumulated in tissues (particularly kidneys) makes CER-001 a important tool in the treatment of Norum disease. Board Change • Oct 01
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 4 experienced directors. 3 highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment. Board Change • Jun 24
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 4 experienced directors. 3 highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Tillkännagivande • Jun 15
ABIONYX Pharma Completes Pre-IND Meeting with the FDA for CER-001 Phase 2b/3 Clinical Trial for Patients with Sepsis ABIONYX Pharma announced that the company has completed a pre-IND (Investigational New Drug Application, IND) meeting with the US Food and Drug Administration and has received feedback to support an IND filing for its candidate drug. This is an important validation of the quality of the project and a significant step towards an application to include American study centers in future clinical trials. ABIONYX Pharma intends to file an IND application to the US authority in the coming months. latest phase 2a clinical study with CER-001, was published by BMC Medicine, in a translational research paper that included important in-vitro data, proof of concept in a pig model of sepsis and a four-arm controlled study in patients with sepsis. All sets of data showed statistically significant effects on parameters such as endotoxin levels, key inflammatory cytokines such as IL-6 and TNF-a and endothelial markers of inflammation such as sVCAM-1, sICAM-1 and MCP-1. Tillkännagivande • May 25
ABIONYX Pharma SA, Annual General Meeting, Jun 27, 2024 ABIONYX Pharma SA, Annual General Meeting, Jun 27, 2024. Location: 33 43 avenue georges pompidou bat d, balma France Tillkännagivande • May 18
ABIONYX Pharma SA to Report Q2, 2024 Results on Aug 22, 2024 ABIONYX Pharma SA announced that they will report Q2, 2024 results on Aug 22, 2024 Board Change • Apr 12
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 4 experienced directors. 3 highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. New Risk • Mar 18
New minor risk - Revenue size The company makes less than US$5m in revenue. Total revenue: €4.6m (US$5.0m) This is considered a minor risk. Companies with a small amount of revenue are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-€3.7m free cash flow). Shares are highly illiquid. Earnings are forecast to decline by an average of 43% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 2 years (€9.0m net loss in 2 years). Shareholders have been diluted in the past year (14% increase in shares outstanding). Revenue is less than US$5m (€4.6m revenue, or US$5.0m). Market cap is less than US$100m (€31.2m market cap, or US$34.0m). Reported Earnings • Mar 11
Full year 2023 earnings released Full year 2023 results: Revenue: €4.60m (down 12% from FY 2022). Net loss: €3.50m (loss narrowed 17% from FY 2022). Revenue is forecast to grow 35% p.a. on average during the next 3 years, compared to a 13% growth forecast for the Biotechs industry in Germany. New Risk • Mar 05
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 14% This is considered a minor risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-€2.5m free cash flow). Shares are highly illiquid. Earnings are forecast to decline by an average of 34% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 2 years (€7.7m net loss in 2 years). Shareholders have been diluted in the past year (14% increase in shares outstanding). Market cap is less than US$100m (€35.2m market cap, or US$38.2m). Board Change • Mar 05
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 4 experienced directors. 3 highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Tillkännagivande • Feb 16
ABIONYX Pharma SA Acknowledges the Clinical Results of the Phase 3 Aegis-Ii Study Evaluating the Efficacy and Safety of Csl Behring’S Human-Plasma-Derived Apoa-I, CSL112 ABIONYX Pharma acknowledges that the Phase 3 AEGIS-II study evaluating the efficacy and safety of CSL Behring’s human-plasma-derived apoA-I, CSL112, compared to placebo in reducing the risk of major adverse cardiovascular events (MACE) in patients following an acute myocardial infarction (AMI), did not meet its primary efficacy endpoint of MACE reduction at 90 days. In addition, CSL Behring announced that there are no plans for a near-term regulatory filing and added there were no major safety or tolerability concerns with CSL112. With over 18,000 patients treated, the AEGIS-II Trial results stand as a testament to the safety and tolerability of apoA-1-based treatments. The clinical results of the Phase 3 AEGIS-II Trial of human plasma-derived apolipoprotein A-I, CSL112 in acute myocardial infarction (AMI), strongly supports ABIONYX’ decision, made four years ago, to reposition the development of CER-001 out of the treatment of longstanding chronic diseases, such as coronary artery disease, and into acute conditions where the short-term dosing model followed by Abionyx has the potential to make a marked impact. ABIONYX Pharma has meticulously evaluated other diseases where apoA-I is known to have a beneficial or protective effect. Acute sepsis is an example where the beneficial effects of apoA-1 on mortality and other outcomes is supported by a wealth of epidemiological, genetic, animal and human data, including animal and human data with CER-001. Building on the safety demonstrated during CER-001 Phase 2 and 3 trials with 900 patients in cardiovascular diseases, Abionyx has strategically redirected its focus towards addressing high levels of unmet medical need in acute sepsis, acute renal, inflammatory and ophthalmic diseases. This pivotal decision demonstrates ABIONYX’ dedication to innovative treatments where therapy using recombinant apoA-1 can maximize impact on patient outcomes. About CER-001 CER-001 is a novel engineered recombinant human apoA-I that was designed to mimic the structural and functional biological properties of natural, nascent HDL, also known as pre-ß HDL, and has been shown to perform all steps of the Reverse Lipid Transport pathway (RLT), the only natural pathway responsible for lipid elimination. Administered CER-001 particles increase transient apoA-I and the number of HDL particles and promote the elimination of trapped cholesterol and lipids in tissues in the absence of LCAT enzyme for example, but also the elimination of bacterial lipid endotoxin (LPS) in the case of sepsis. HDL particles are then recognized by the liver, leading to the elimination of these transported lipids via a process called Reverse Lipid Transport. Tillkännagivande • Jan 09
ABIONYX Pharma Appoints Rob Scott as Chief Medical Officer and Head of R&D ABIONYX Pharma, announced the appointment of Dr. Rob Scott, former Chief Medical Officer and Head of Development at AbbVie, where he oversaw successful drug development for brands like Skyrizi, and Rinvoq, as Chief Medical Officer and Head of R&D with immediate effect. Dr. Rob Scott brings extensive clinical development and regulatory experience to ABIONYX Pharma. Tillkännagivande • Nov 25
ABIONYX Pharma Presents Preclinical Results for CER-001 in Brain Fog, At the 1st International Scientific Congress on Brain-Kidney Interaction in Naples on November 23-24, 2023 ABIONYX Pharma announced the presentation of new preclinical data for CER-001 in the treatment of brain dysfunction associated with acute kidney injury at the 1st International Congress on brain-kidney interaction: from physiology to clinics, to be held from November 23 to 24, 2023 in Naples, Italy. This lecture presentation follows the presentation of preclinical results on the effects of CER-001 in brain dysfunction induced by acute kidney injury, which were the subject of a dedicated poster at the 2023 Annual Meeting of the American Society of Nephrology (ASN) held from November 2 to 5, 2023, as part of Kidney Week. To date there are no appropriate preventive or therapeutic solutions to treat brain dysfunction related to kidney injury consecutive or concomitant with sepsis. A preclinical study has been sponsored by ABIONYX Pharma to assess the effects of recombinant apoA-I phospholipid complexes in reducing the inflammatory process and preventing sepsis-induced acute renal failure and brain dysfunction. In both CER-001 dose groups (20 mg/kg and 2x20 mg/kg), blood samples showed a significant reduction in Indolamine-2,3-dioxygenase enzyme activity, measured as the kynurenine/tryptophan ratio (p<0.05) and quinolinic acid levels (p<0.005) compared with the untreated control group. In addition, a significant decrease in systemic and brain interleukin-6 (IL-6) levels was observed after CER-001 treatment. Taken together, these data indicate that CER-001 treatment can reduce the inflammatory response, retention of residual substances and neuroactive compounds, thereby potentially improving renal and cognitive function in sepsis-induced acute renal failure. These results pave the way for future clinical trials in potential additional indications, such as neuropsychiatric lupus or, more importantly long Covid. Tillkännagivande • Nov 21
ABIONYX Pharma Announces New Positive Results in a Uveitis Model for the Strategic Development of the First Class of Biomedicines in Ophthalmology Based on its Recombinant apoA-I ABIONYX Pharma announced new positive results in ophthalmology for the first class of CER-001-based biomedicines for the treatment of ocular pathologies, and the appointment of Mr. Jérôme Martinez as ABIONYX Pharma's Senior Advisor in ophthalmology. New positive long-term preclinical results evaluating the efficacy of CER-001 after a single intraocular administration in a uveitis model with severe inflammation. Following the beneficial clinical findings related to the disappearance of visual blurring linked to corneal deposits in a patient suffering from LCAT deficiency treated under a Temporary Authorization for Use, and a marked improvement in the patient's visual function (results published exclusively in the scientific journal "Annals of Internal Medicine" in 2021), and still observed after more than a year of follow-up, ABIONYX Pharma conducted new preclinical studies in ophthalmology to qualify the efficacy spectrum of recombinant apoA-I alone and in combination with a corticosteroid, and to broaden its potential in new indications. After demonstrating the safety of CER-001, the recombinant apoA-I was tested again to assess its action in reducing inflammatory reactions and its tolerability after a single intraocular administration (IVT) in a model of LPS-induced uveitis. 74 animals participated in this study, divided into 8 groups. Six hours after LPS injection, statistically significant reductions in inflammation, measured by slit-lamp examination, were observed for the groups treated with CER-001 alone or in combination with a corticosteroid compared with the vehicle-treated group. CER-001 (cumulative score 3.1 ± 2.3, p= 0.0254) and best-in-class drug (cumulative score 3.1 ± 1.5, p= 0.0228) were superior to vehicle. No statistically significant differences were observed for the other groups treated with standard therapies. Twenty-four hours after induction, the significance observed at six hours for the CER-001 and best-in-class drug-treated groups versus the vehicle-treated group continued, demonstrating a reduction in inflammation (cumulative score 3.9 ± 1.7 and 4.9 ± 1.2, respectively and p < 0.0001 and = 0.0018, respectively). The downward trend observed at six hours compared with the vehicle-treated group was confirmed by statistical significance for CER-001 alone and CER-001 in combination (cumulative score 5.3 ± 1.3 and 4.6 ± 2.1, respectively and p = 0.0081 and = 0.0018, respectively). No statistically significant differences were observed for all other groups. Results for the CER-001 and CER-001 combination groups were comparable or better than those for the best-in-class alone group. Twenty-four hours after LPS injection, the high level of induced inflammation was reached in the vehicle-treated group, with median values of 5920 cells/µL. A statistically significant decrease in leukocyte infiltration was observed in the groups treated with CER-001 alone and CER-001 in combination with a corticosteroid, compared with the vehicle-treated group. For all other groups, no significance was observed. CER-001 alone or in combination with a corticosteroid, as tested in this preclinical study, proved safe and well tolerated on the ocular surface and inside the eye, when given by injection inside the eye. These new preclinical results reconfirm the major therapeutic potential of the only recombinant apoA-I in ophthalmology. The anti-inflammatory and/or reverse lipid transport-enhancing properties of CER-001, and these new preclinical results in uveitis, pave the way for clinical trials testing apoA-I in patients with other severe inflammatory ocular diseases. Tillkännagivande • Nov 03
Abionyx Pharma Racers Study Data in Sepsis Presented At the American Society of Nephrology (Asn) 2023 Annual Meeting "Kidney Week" ABIONYX Pharma SA announced the full results of the RACERS Phase 2 clinical trial of CER-001, an apoA-1-based therapy for the treatment of sepsis, in a late-breaking clinical trial poster presentation at the American Society of Nephrology (ASN) Kidney Week 2023. CER-001 rapidly and significantly eliminated endotoxins, and the result was maintained (p<0.05 on days 3, 6 and 9), whereas even by day 9, patients on standard care alone showed no decreases in endotoxin levels relative to baseline. Mortality for all patients at 30 days was 6.7% for the CER-001 group and 20.0% for patients treated with standard care alone. This represents a Relative Risk Reduction (RRR) of 65%. Among critical care patients, mortality rates were 14.7% compared to 50.0% for patients on standard care (RRR=71%). ICU patients treated with CER-001 were discharged earlier than patients receiving standard care, with an average ICU stay 5 days shorter than that of patients on standard care. Tillkännagivande • Oct 16
ABIONYX Pharma RACERS Study in Sepsis Selected for Late-Breaking Clinical Results Poster Presentation at the American Society of Nephrology 2023 Annual Meeting " Kidney Week" ABIONYX Pharma announced that the Company's Phase 2b RACERS study in sepsis has been selected as a late-breaking clinical trial poster presentation at the 2023 American Society of Nephrology Annual Meeting taking place November 2-5, 2023 in Philadelphia, Pennsylvania. Tillkännagivande • Jul 27
ABIONYX Pharma Successfully Manufactures a Second Batch of Recombinant Human ApoA-I CER-001 Using a New, Innovative and Robust Industrial Bioprocess ABIONYX Pharma announced the successful completion of a second batch for the GMP (Good Manufacturing Practice) industrial biomanufacturing of CER-001 using an innovative and robust method. ABIONYX has successfully manufactured a second batch of CER-001 under GMP conditions, using a novel and robust industrial process. This confirms that ABIONYX's new production line, based on an innovative and efficient approach, is ready to enter the Apotherapy market, based on the only natural recombinant ApoA-I protein. All stages of the biomanufacturing process are designed to increase production yields, enabling ABIONYX Pharma to serve its target markets in kidney disease, sepsis and ophthalmology. This second batch confirms the improved yield and simplification of the biomanufacturing process defined and followed for industrialization. This batch confirms that all industrial barriers have been crossed to ensure the generation of production volumes consistent with future needs. Before applying for marketing approval, based on the success of clinical trials conducted to assess the efficacy and safety of the biomedicine, at least 3 consecutive batches using the intended commercial process must be manufactured in accordance with Good Manufacturing Practices (GMP) in order to obtain regulatory approval. Biomedicines manufactured in compliance with GMP guarantee the quality, safety and reliability of the bioproduct in order to meet regulatory requirements. Finally, to obtain regulatory approval, the biotech must also provide data on the long-term stability of the biomedicine. This involves carrying out stability tests on 3 consecutive batches to determine the product's shelf life. In fact, due to the successful production of this second batch, the new manufacturing process has been reconfirmed, positioning on a biomanufacturing trajectory that will require the completion of 3 consecutive GMP pilot batches at the yield level of CER-001's future commercial operation. Tillkännagivande • Jul 12
ABIONYX Pharma Announces New Compassionate Access Authorization (CAA) for CER-001 in the Rare Disease LCAT Deficiency or Norum Disease in Europe ABIONYX Pharma announced that the company has been granted a new Compassionate Access Authorization (CAA) in Europe for CER-001 in the rare, untreated kidney disease LCAT Deficiency. This is the fourth patient suffering from LCAT deficiency to be granted Compassionate Access to CER-001, in development by ABIONYX Pharma, in Europe. The four patients suffering from this rare disease, which has a major impact on quality of life and on lifespan, represent very different stages in the evolution of the disease, for which CER-001 may prove effective, whether or not the patients are transplanted, and whatever their age. CER-001 would represent a unique therapeutic breakthrough for patients who need to be diagnosed as early as possible to avoid dialysis, followed by one or more transplants during their lifetime. The early administration of CER-001 in young patients with Norum disease could have a real beneficial effect on these patients, transforming their lives and, above all, delaying the end of their lives, while very significantly reducing the management of their disease, whether with dialysis or by one or more transplants. The safety and tolerability data for this treatment are as positive as ever, with no adverse events reported for any of the patients. Constructive discussions have been initiated with the European regulatory authorities to define a pathway for the rapid advancement of CER-001's clinical development in the patient population affected by this rare disease. The treatment of LCAT deficiency or Norum disease is a major medical challenge, given the constant progression of the disease, leading to rapid renal decline and often premature death, and the total absence of approved treatments to slow or modify the progression of the disease. ABIONYX Pharma continues to receive and fulfill Compassionate Access requests from hospitals around the world. The biomanufacturing of the latest and subsequent batches of CER-001 to the highest GMP quality standards will enable these requests to be met, and the clinical results of the first patients will be communicated exclusively through scientific publication. Tillkännagivande • May 25
ABIONYX Pharma SA, Annual General Meeting, Jun 28, 2023 ABIONYX Pharma SA, Annual General Meeting, Jun 28, 2023. Tillkännagivande • May 19
ABIONYX Pharma SA to Report Q2, 2023 Results on Aug 17, 2023 ABIONYX Pharma SA announced that they will report Q2, 2023 results on Aug 17, 2023 Tillkännagivande • May 11
ABIONYX Pharma Announces Successful Manufacturing of the First Batch of CER-001 with Recombinant Human ApoA-I Using aNew Innovative and Robust Industrial Bioprocess ABIONYX Pharma announced the successful GMP (Good Manufacturing Practices) industrial biomanufacturing of a batch of CER-001 using an innovative and robust method. This success confirms the relevance of the strategy to relocate biomanufacturing to France, which was implemented 4 years ago, not only to contribute to the country's industrial sovereignty in biomedicine, but also to ensure the independence of the Company's development from external constraints. This performance also testifies to the efforts and hard work of ABIONYX's teams who worked to optimize the industrial process, despite raw material inflation, a difficult context and limited financial resources. Finally, this major manufacturing improvement simplifies the process by making it more competitive, which increases the value of the bioproduct and of ABIONYX Pharma, which controls the manufacturing and intellectual property rights. First batch of recombinant human ApoA-I CER-001 produced by an innovative and robust industrial process ABIONYX successfully manufactured a GMP-compliant batch of CER-001 using a new innovative and robust industrial process. ABIONYX thus demonstrates that the new production line constitutes an innovative and efficient approach to access the apotherapy market, based on the only human recombinant ApoA-I protein. All the steps of the biomanufacturing process have been revisited, improved and requalified in order to increase the production yield and to serve the targeted markets such as renal diseases, sepsis or ophthalmology. As a reminder, biomedicines, which are often very complex to produce, play a crucial role in the development of new, highly innovative treatments, as demonstrated by CER-001. Examples of these biomedicines are interferons, coagulation factors, etc. These are large and highly complex molecules. In contrast to the production of classic drugs, a very specific know-how must be implemented during the manufacturing of therapeutic biomolecules due to their high potential sensitivity to changes in environmental conditions such as temperature, the composition of the culture medium, etc. In the case of CER-001, the natural protein ApoA-I is the most important part of the manufacturing process and requires this unique know-how that ABIONYX Pharma is the only company to master to date. The process was greatly simplified, making it more robust, with a concomitant positive effect on its cost, enabling the filing of a new patent in 2021 to continue to protect and extend its intellectual property. Performance improvement and simplification of the biomanufacturing process Since 2005, work on the biomanufacturing of CER-001 has crossed all industrial barriers. Thanks to the innovative process developed, ABIONYX ensures that the industrialization of CER-001 is now possible for production volumes consistent with future needs. ABIONYX’s ApoA-I is the only recombinant protein produced from cells and not an ApoA-I derived from purified plasma or blood. It displays the pleiotropic, anti-inflammatory, vascular integrity restoring and immune system modulating functional properties described for endogenous ApoA-I. This is a unique situation enabling ABIONYX to restore the normal level of ApoA-I to benefit patients with deficiency. ApoA-I deficiency can be due to rare genetic orphan diseases resulting in a severe and chronic deficiency, can be age-related as in ophthalmologic conditions, but can also can be due to acute severe inflammatory conditions such as bacterial or viral sepsis, including COVID-19. The known pleiotropic effects of natural ApoA-I enable CER-001 to address a wide range of diseases associated with reduced ApoA-I, and its mechanism of action is independent of endogenous levels of HDL to be effective. Board Change • Apr 24
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 5 experienced directors. 2 highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Tillkännagivande • Jan 17
ABIONYX Pharma Reports Positive Results from Phase 2A Pilot Clinical Trial ABIONYX Pharma reported that the pilot Phase 2a clinical trial evaluating CER-001, the only natural recombinant apoA-I, as a treatment for septic patients at high risk of developing Acute Kidney Injury (AKI) met its primary objective. There are no approved treatments for septic patients in the world. The RACERS study included 20 patients with gram-negative sepsis who were at high risk for acute kidney injury due to high levels of endotoxin activity and decline in function of one or more organ systems. Patients received either standard of care treatment alone, or in combination with one of three dosage regimens of CER-001 (five patients per group). The main objective of this pilot study was to investigate whether the use of CER-001 at different doses, in combination with standard of care (SOC) treatment, is safe and effective, providing a potential new strategy to treat septic patients, reducing the inflammatory response to endotoxin and preventing the progression to AKI according to KDIGO (Kidney Disease: Improving Global Outcomes) criteria, as well as safety and tolerability of the dosage regimens in order to select the optimal dose of CER-001. One of the metabolic characteristics of bacterial (like sepsis) or virus infections (like sars-Cov-2) is the strong decrease of circulating lipoprotein and particularly the High-Density Lipoprotein (HDL) with its main containing protein apolipoprotein A-I (apoA-I). As an example, apoA-I level was recently described as the biomarker predictive of long-term mortality after surgical sepsis1. The rational of Abionyx was to restore, using CER-001, the apoA-I levels to reestablish all the functionality of this individualized biomarker leading to potential benefit in sepsis pathology. RACERS pilot study has shown for the first time in a human pilot trial that the recovery of a normal apoA-I level in patient stop the cytokine storm and improve the clinical outcomes. CER-001 demonstrated rapid and sustained reduction in endotoxin levels and consequent reduction in the inflammatory cascade or “cytokine storm” relative to SOC alone. Endothelial biomarkers demonstrated a significant protective effect of CER-001. Trends towards fewer ICU days, lower requirement for organ support and improved 30-day survival. Evaluation of safety data, taken together with the pharmacokinetic and pharmacodynamic data, has identified the dose that will be used in subsequent studies. The observed safety and efficacy in RACERS were generally consistent with historical data including clinical results for CER-001 in COVID-19 that were published recently in the scientific journal "Frontiers in Medicine", a specialty medicine journal, in September 2022. The potential use of CER-001 in septic patients is currently under clinical development. These data will be discussed with regulatory authorities, starting with Europe but also the U.S. later this year in order to design an appropriate clinical and regulatory development strategy for this disease state that currently has no available treatment options. RACERS is a clinical trial named RACERS (a RAndomized study comparing short-term CER-001 infusions at different doses to prevent Sepsis-induced acute kidney injury) with CER-001 in septic patients at high risk of developing acute kidney injury. Following the positive signals observed in the Temporary Authorization for Named Use (ATUn) in an ultra-rare kidney disease, the study assessed the role of CER-001, a novel, in preventing Acute Kidney Injury (AKI) in septic patients. The core component of the program is the launch of a 30-day Phase 2a clinical dose-finding trial with the Company's lead product candidate, CER-001, in the prevention of AKI in septic patients. Researchers have demonstrated that in humans, reconstituted HDLs have a scavenger role in reducing circulating endotoxin, as well as major anti-inflammatory and endothelial activity. These important effects were also demonstrated with CER-001 in a rigorous preclinical model of sepsis-induced AKI developed in collaboration with an Italian Veterinarian Hospital (Surgical Section, Chief: Prof. Antonio Crovace). Several other AKI/sepsis models showed that HDL is a critical factor in modifying the disease. This clinical study, designed in concert with expert Italian nephrologists (Nephrology, Dialysis and Transplantation Unit, Chief: Prof. Loreto Gesualdo) and intensivists (Anesthesiology and Resuscitation Unit, Chief: Prof. Salvatore Grasso), was a randomized, open labelled, placebo-controlled, parallel-group study evaluating the safety and efficacy of intravenously administered CER-001 in patients with sepsis at high risk for AKI based on their endotoxin levels and Sequential Organ Failure Assessment (SOFA score). A total of 20 patients were randomized to receive 8 doses of CER-001 over 6 days on top of standard of care, or standard of care alone. The primary endpoint of the study was the onset and severity of AKI according to KDIGO criteria as well as safety and tolerability of the dosage regimens in order to select the optimal dose of CER-001. The clinical study was partnered with the University of Bari. Board Change • Jan 03
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 7 experienced directors. No highly experienced directors. CEO, GM & Director Cyrille Tupin was the last director to join the board, commencing their role in 2019. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Tillkännagivande • Oct 05
ABIONYX Pharma SA Announces Final Patient Enrollment in Phase 2a Clinical Trial with CER-001, Bio-HDL for the Treatment of Patients with Sepsis at High Risk of Developing Acute Kidney Injury ABIONYX Pharma SA announced that the last patient has been enrolled in the Phase 2a clinical trial evaluating CER-001, Bio-HDL, as a potential treatment for patients with sepsis at high risk of developing acute kidney injury. RACERS is a randomized, open-label, placebo-controlled, parallel-group, Phase 2a study evaluating the safety and efficacy of CER-001 administered intravenously to critically ill patients with sepsis who are at high risk of acute kidney injury (AKI) based on their Sequential Organ Failure Assessment (SOFA) score. A total of 20 patients were randomized to receive CER-001 or placebo over 6 days. The primary endpoint of the study was the onset and severity of acute renal failure according to KDIGO criteria, as well as the safety and tolerability of the dosing regimens, to select the optimal dose of CER-001. Additional efficacy parameters included changes from baseline levels of endotoxin and key inflammatory biomarkers. The clinical study is being conducted in partnership with the University of Bari. The company reconfirms that the final topline results will be reported by the end of fall 2022. Tillkännagivande • Apr 07
ABIONYX Pharma Reports Positive Interim Results from Phase 2A Clinical Trial Evaluating CER-001 in the Treatment of Septic Patients At High Risk of Developing Acute Kidney Injury ABIONYX Pharma reported positive interim results of an open-label Phase 2a clinical trial evaluating CER-001 as a treatment for septic patients at high risk of developing Acute Kidney Injury (AKI). The RACERS study aims to include 20 patients with gram-negative sepsis who are at high risk for acute kidney injury due to high levels of endotoxin activity and decline in function of one or more organ systems. Completion of the study has been delayed due to the COVID pandemic, which has limited availability of critical care beds and medical staff for non-COVID patients, since the first study patient was enrolled in June 2021. To date, thirteen patients have been enrolled and seven remain to be recruited. An interim review for the first ten patients to complete the study has been performed. Compared to standard of care therapy, CER-001 rapidly improved biomarkers of inflammation including leukocytosis, and endothelial dysfunction, preventing septic patients’ decline into acute kidney injury. CER-001 treatment was well tolerated at all dose levels (5, 10 and 20 mg/kg, twice a day). No treatment-related serious side effects were seen in this critically ill patient population. Tillkännagivande • Mar 30
ABIONYX Pharma Receives FDA Orphan Drug Designation (ODD) for CER-001 for the Treatment of LCAT Deficiency Presenting as Kidney Dysfunction and/or Ophthalmologic Disease ABIONYX Pharma SA announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to the Bio-HDL CER-001 for the treatment of lecithin-cholesterol acyltransferase (LCAT) deficiency. The designation covers both partial LCAT deficiency, presenting as Fisheye Disease, and complete LCAT deficiency presenting with renal symptoms and corneal opacities. Progression of LCAT deficiency, for which there is no approved treatment, can ultimately lead to renal failure requiring dialysis or kidney transplant, and/or to complete corneal opacification requiring transplant. The European Medicines Administration (EMA) granted ODD status to CER-001 for the treatment of LCATdeficiency in July 2021. Positive clinical results from CER-001 in LCAT disease have previously been published. In the Annals of Internal Medicine in March 2021, a case study of a patient who was about to undergo dialysis due to the rapid decline in renal function was described. The patient was able to avoid the need for dialysis during her treatment with CER-001 and in addition, lipid deposits in her corneas which had caused significant visual blurring, improved with treatment. The improvement in visual function was still observed after 1 year of follow-up. A second case was described in the Journal of Internal Medicine in November 2021 and showed that CER-001 reduced glomerular lipid deposits and slowed the patient’s decline in renal function. Furthermore, CER-001 remodeled his plasma lipoproteins by reducing the level of LpX, large abnormal lipid complexes known to be renally toxic. Tillkännagivande • Jan 05
ABIONYX Pharma Announces That the French Drug Safety Agency Has Granted a Compassionate Access Authorization for the bio-HDL (CER-001) in COVID-19 Disease ABIONYX Pharma announced that the French Drug Safety Agency (Agence Nationale de Sécurité du Médicament or ANSM), granted a Compassionate Access Authorization for the bio-HDL (CER-001) in COVID-19 disease. COVID-19 is associated with respiratory symptoms characterized by acute lung injury, rapidly progressing to acute respiratory distress syndrome. The pulmonary dysfunction is rapidly accompanied by a major "cytokine storm" during which inflammatory cytokines are released abundantly into the bloodstream leading to host tissue damage. Decreased levels of total cholesterol, LDL and HDL have been observed in patients with COVID-19 infections. Patients with low HDL at hospital admission had an increased risk of developing severe disease compared with patients with high HDL. With recovery from COVID-19 infections, serum lipid levels return to pre-infection levels. These lipid abnormalities could be modified by pharmacological agents that increase plasma ApoA-I and HDL levels, but more importantly increase the number of functional HDL particles. Thus, CER-001, a recombinant bio-HDL, may have the potential to improve the clinical outcome of patients with COVID-19. The current data do not allow to presume a favorable benefit-risk ratio for the use of CER-001 in the context of this authorization. Tillkännagivande • Jun 08
ABIONYX Announces First Patient Enrolled in Phase 2a Clinical Study With CER-001, the Bio-HDL for the Treatment of Septic Patients at High Risk of Developing Acute Kidney Injury ABIONYX Pharma announced that the first patient has been enrolled in a Phase 2a clinical study evaluating CER-001, the Bio-HDL, as a potential treatment for septic patients at high risk of developing acute kidney injury. RACERS is a randomized Phase 2a, open labelled, placebo-controlled, parallel-group study evaluating the safety and efficacy of intravenously administered CER-001 in ICU patients with sepsis at high risk for AKI based on their Sequential Organ Failure Assessment (SOFA score). A total of 20 patients will be randomized to receive 8 doses of CER-001 or placebo over 6 days. The primary endpoint of the study will be the onset and severity of AKI according to KDIGO criteria as well as safety and tolerability of the dosage regimens in order to select the optimal dose of CER-001. The clinical study is partnered with the University of Bari and the Consorzio per Valutazioni Biologiche e Farmacologiche (CBVF) and is already fully funded. Valuation Update With 7 Day Price Move • Jun 01
Investor sentiment improved over the past week After last week's 26% share price gain to €1.44, the stock trades at a trailing P/E ratio of 16.3x. Average forward P/E is 45x in the Biotechs industry in Europe. Total loss to shareholders of 25% over the past three years. Valuation Update With 7 Day Price Move • Apr 29
Investor sentiment improved over the past week After last week's 21% share price gain to €1.18, the stock trades at a trailing P/E ratio of 11.3x. Average forward P/E is 35x in the Biotechs industry in Europe. Total loss to shareholders of 39% over the past three years. Valuation Update With 7 Day Price Move • Mar 30
Investor sentiment improved over the past week After last week's 15% share price gain to €1.06, the stock trades at a trailing P/E ratio of 12x. Average forward P/E is 34x in the Biotechs industry in Europe. Total loss to shareholders of 40% over the past three years. Tillkännagivande • Mar 03
ABIONYX Announces Positive Clinical Results From CER-001 in an Ultrarare Kidney Disease Published in the Annals of Internal Medicine ABIONYX Pharma SA announced positive clinical results from CER-001 in an ultrarare kidney disease published in the Annals of Internal Medicine, the high cited and ranked internal medicine journal. The patient, who was about to undergo dialysis due to the rapid decline in renal function, was able to avoid the need for dialysis during his treatment with CER-001. In addition, the patient who was suffering from lipid deposits in the corneas saw the visual blurring disappear. This clear improvement in visual function is still observed after 1 year of follow-up. As a reminder, the French Drug Safety Agency (Agence Nationale de Sécurité du Médicament or ANSM), granted a named patient Temporary Authorization for Use (“ATU nominative”) for CER-001 in an untreated, ultra-rare renal disease early 2020. The patient with inherited mutations in the lecithin-cholesterol acyltransferase (LCAT) gene developed glomerulopathy and corneal lipid deposits and displayed very low circulating levels of high-density lipoprotein (HDL) and apoA-I. In this ATUn granted in the familial LCAT deficiency disease the patient was treated with CER-001, an apoA-I-containing HDL mimetic, to help in preventing rapidly progressive kidney failure. CER-001 was completely safe and very well tolerated at doses of 10-30 mg/kg/week. CER-001 was given intravenously at a dose of 10 mg/kg thrice a week for 3 weeks, then twice a week for 3 weeks and once a week for 3 weeks. Thereafter, the dose was increased to 20 mg/kg/week for 6 weeks to reach the weekly dose that stabilized eGFR. Whereas eGFR rapidly decreased from 41 to 19 mL/min/1.73 m2 during the 9 months that preceded the start of CER-001, eGFR only decreased from 19 to 17 mL/min/1.73 m2 during the 11 months following treatment introduction. Urinary protein-to-creatinine ratio (uPCr) decreased from 7 to 0.25 g/g at day 10, with undetectable albuminuria at that time, but returned to initial values thereafter. However nephrotic syndrome disappeared with serum albumin increased from 29 to 37 g/L during the treatment period and then the follow-up. The publication states that CER-001 prevented further kidney function decline. The patient who was about to be dialysed due to rapidly declining kidney function, was able to avoid the need for dialysis during their treatment with CER-001. No other treatment was introduced strongly suggesting that stabilization of the renal function relied on the administration of CER-001. The publication mentions that CER-001 effects on renal progression should be assessed in more common proteinuric diseases, including diabetic nephropathy or extra-membranous glomerulonephritis (two diseases characterized by a reduction in local LCAT activity, gloemrular lipid deposits, and inflammatory processes), or other nephropathies related to lipid deposits. The publication reveals the systemic mechanism of action of CER-001 and broadens the field of breakthrough innovations to ophthalmology. The publication mentions positive extra-renal clinical results and the disappearance of visual blurring secondary to corneal deposits. This clear improvement in visual function is still observed after 1 year of follow-up. The clear improvement of the blurred vision at the end of the follow-up suggests that the anti-inflammatory properties and/or the increase in the Reverse Cholesterol Transport (RCT) of CER-001 can improve vision in FLD patients. This finding and previous data showing the role of apoA-I in the development of corneal clouding and blurring vision will pave the way to interventional studies testing CER-001 in patients developing lipid corneal deposits from other pathologies such as secondary lipid keratopathy or inherited corneal dystrophy. Is New 90 Day High Low • Mar 02
New 90-day high: €1.24 The company is up 33% from its price of €0.93 on 02 December 2020. The German market is up 8.0% over the last 90 days, indicating the company outperformed over that time. It also outperformed the Biotechs industry, which is up 6.0% over the same period. Valuation Update With 7 Day Price Move • Mar 02
Investor sentiment improved over the past week After last week's 42% share price gain to €1.24, the stock is trading at a trailing P/E ratio of 12.9x, up from the previous P/E ratio of 9.1x. This compares to an average P/E of 33x in the Biotechs industry in Europe. Total return to shareholders over the past three years is a loss of 38%. Tillkännagivande • Dec 24
ABIONYX Pharma SA Initiates a Phase 2a Clinical Trial With CER-001 in Septic Patients at High Risk of Developing Acute Kidney Injury ABIONYX Pharma announced that it has received authorization from the Italian authorities to launch a clinical trial named RACERS (a RAndomized study comparing short-term CER-001 infusions at different doses to prevent Sepsis-induced acute kidney injury) with CER-001 in septic patients at high risk of developing acute kidney injury. Following the positive signals observed in the Temporary Authorization for Named Use (ATUn) in an ultra-rare kidney disease, the study will assess the role of CER-001, a HDL-mimetic, in preventing Acute Kidney Injury (AKI) in septic patients. The core component of the program will be the launch of a 30-day Phase 2a clinical dose-finding trial with the Company's lead product candidate, CER-001, in the prevention of AKI in septic patients. Researchers have demonstrated that in humans, reconstituted HDLs have a scavenger role in reducing circulating endotoxin, as well as major anti-inflammatory and endothelial activity. These important effects were also demonstrated with CER-001 in a rigorous animal model of sepsis-induced AKI. Several other AKI/sepsis models showed that HDL is a critical factor in modifying the disease. This clinical study, designed in concert with expert Italian nephrologists (nephrology Dialysis and Transplantation Unit, Chief: Prof. Loreto Gesualdo) and intensivists (Anesthesiology and Resuscitation Unit, Chief: Prof. Salvatore Grasso), will be a randomized, open labelled, placebo-controlled, parallel-group study evaluating the safety and efficacy of intravenously administered CER-001 in ICU patients with sepsis at high risk for AKI based on their Sequential Organ Failure Assessment (SOFA score). A total of 20 patients will be randomized to receive 8 doses of CER-001 or placebo over 6 days. The primary endpoint of the study will be the onset and severity of AKI according to KDIGO criteria as well as safety and tolerability of the dosage regimens in order to select the optimal dose of CER-001. The secondary endpoints will include changes in endotoxin and IL-6 levels from baseline to Day 3, Day 6 and Day 9, changes in the SOFA score from baseline to Day 3, Day 6 and Day 9, changes in other key inflammatory markers (e.g., CRP, IL-8, MCP 1 and TNF-a), and changes in AKI biomarkers (TIMP-2 and IGFBP7). Enrollment of patients in the study is expected to begin in the first half of 2021. The clinical study is partnered with the University of Bari and the Consorzio per Valutazioni Biologiche e Farmacologiche foundation (CBVF) and is already fully funded. Tillkännagivande • Dec 17
ABIONYX Pharma Announces Publication of Pre-Clinical Data in the Journal Metabolism Demonstrating CER-001 Improves Lipid Profile and Kidney Function for an Ultra-Rare Kidney Disease ABIONYX Pharma announced the publication of a set of pre-clinical data in the journal Metabolism demonstrating that CER-001 improves lipid profile and kidney function in an emphasis kidney disease model representative of LCAT (lecithin-cholesterol acyltransferase) deficiency. CER-001 is an HDL mimetic previously tested in different pathological conditions, but never in a kidney disease prior to ABIONYX Pharma's ATUn. In a genetically modified mouse model of LCAT deficiency, CER-001 had beneficial effects not only on lipid profile but also on renal function, reducing urinary albumin to creatinine ratio and restoring nestin and nephrin content in the glomerulus. These new data give a new opportunity for the use of CER-001 for the treatment of renal diseases. The company will continue to work on strengthening the understanding the mechanism of action of CER-001 in kidney disease. This study was designed to investigate the catabolic fate of CER-001, and to evaluate the effects of CER-001 on kidney disease. Wild-type mice received CER-001 for 2 weeks. Plasma lipid/lipoprotein profile and HDL subclasses were analyzed. In a second set of experiments, mice were induced to develop the ultra-rare renal LCAT disease and treated with CER-001. Lipid profile, renal function, and kidney histology were evaluated. As a conclusion, three major outcomes have emerged from this study: Treatment with CER-001 ameliorates the dyslipidemia, reducing plasma triglycerides and increasing HDL levels. More importantly, treatment with CER-001 ameliorates renal function in a mouse model with ultra-rare renal disease, reducing urinary albuminuria and restoring podocyte functionality. The data set the basis for the potential use of CER-001 in other renal diseases. ABIONYX is awaiting data from other ongoing preclinical studies. Is New 90 Day High Low • Nov 26
New 90-day high: €1.05 The company is up 20% from its price of €0.87 on 28 August 2020. The German market is up 2.0% over the last 90 days, indicating the company outperformed over that time. It also outperformed the Biotechs industry, which is down 9.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. Tillkännagivande • Nov 26
ABIONYX Pharma Announces the Observation of Positive Therapeutic Signals in Temporary Authorizations for Use (ATUn) in France and Italy for an Ultra-Rare Kidney Disease ABIONYX Pharma announced the observation of positive therapeutic signals in named patient Temporary Authorizations for Use (ATUn), using CER-001 in patients with an ultra-rare kidney disease. Based on the positive therapeutic signals detected in in-depth post-hoc analyses, ABIONYX confirms that the data set of the two ATUn granted in France and Italy supports a promising therapeutic activity of CER-001 in the progression of an ultra-rare renal disease. Due to the severity of their renal disease, patients who were about to be dialysed due to rapidly declining kidney function, were able to avoid the need for dialysis during their treatment with CER-001. These therapeutic signals on CER-001 confirm the potential for CER-001 to be used in severe indications, mainly renal at the moment. In studies currently being considered for publication by peer-reviewed journals, researchers have shown that HDLs have a major anti-inflammatory role and impact on kidney function. These important effects were demonstrated in a genetically modified mouse model of kidney failure. Several studies of other models of renal pathology have previously shown that HDLs stimulate renal remodeling, a critical factor contributing to disease progression. As a reminder, last January and February, ABIONYX received requests to provide CER-001 in France and Italy under named patient Temporary Authorizations for Use (ATUn) in an ultra-rare renal disease without existing treatment. These ATUn were granted at the request and under the sole responsibility of prescribing physicians, and are usually requested when the drug is likely to be of benefit to the patient. ABIONYX has conducted an in-depth analysis of all data that will be included in the publications and will share these results with regulatory authorities in order to redefine the development plan for CER-001 in the treatment of renal diseases without existing treatment. ABIONYX will work closely with patients, investigators, ethics committees and regulatory authorities to identify the next steps to be taken, in the best interest of patients, while ensuring the consent of all stakeholders. These ATUn data do not allow a definitive conclusion on the efficacy endpoint at this time, but are likely to be of benefit to patients with severe kidney disease without existing treatment. Is New 90 Day High Low • Oct 29
New 90-day low: €0.68 The company is down 8.0% from its price of €0.74 on 30 July 2020. The German market is down 5.0% over the last 90 days, indicating the company underperformed over that time. However, it outperformed the Biotechs industry, which is down 15% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. Tillkännagivande • Oct 03
ABIONYX Pharma SA announced that it expects to receive €1.859997 million in funding from Domundi SC and other investors ABIONYX Pharma SA (ENXTPA:ABNX) announced private placement of up to 2,695,648 shares at a price of €0.69 per share for gross proceeds of €1,859,997.12 on October 2, 2020. The shares will issued at premium of €1,725,215.12 on nominal value of €134,782. The investors will acquire 10.94% stake in the transaction. The transaction will include participation from existing investors Domundi SC for 768,115 shares to hold 12.12% stake, Cyrille Tupin, chief executive officer for 376,811 shares to hold 3.4% stake, Luc Demarre for 427,536 shares to hold 3.64% stake, new investor Christian Chavy, board member for 173,913 shares to acquire 0.71% stake, and 949,273 shares for other investors. All shares to be issued are subject to hold period of 6 months. The subscription period will begin on October 5, 2020 and will end on October 16, 2020. The transaction is approved by the board of directors of the company. Is New 90 Day High Low • Sep 26
New 90-day low: €0.71 The company is down 17% from its price of €0.85 on 26 June 2020. The German market is up 3.0% over the last 90 days, indicating the company underperformed over that time. It also underperformed the Biotechs industry, which is down 1.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. 
