Nephrotic Syndrome

Two poster presentations demonstrate ANI’s commitment to research in membranous nephropathy carePRINCETON, N.J., Oct. 24, 2024 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that the Company will present new pre-clinical data highlighting its membranous nephropathy (MN) research during two poster presentations at the American Society of Nephrology (ASN) Kidney Week 2024 in San Diego, CA, October 23-27. "We are excited to share our data with the ASN 2024 community. These data shed light of the potential role of melanocortin receptors in the underlying disease pathologies within nephrotic syndrome,” said Mary Pao, MD, PhD, Chief Medical Officer of ANI “This research contributes to the development of valuable research tools and models for studying membranous nephropathy. The findings represent the first steps in ANI’s commitment to advancing the science underlying certain disease pathologies.” Poster Title: THSD7A-Associated Membranous Nephropathy Involves Both Complement-Mediated and Autonomous Podocyte InjuryPoster Session: Pathology and Lab Medicine - 1Abstract: FR-PO955Presentation Time: Friday, Oct. 25, 10:00 AM-12:00 PMLead Author: Jing Liu Poster Title: Activation of Podocyte-Specific MC5R Signaling by Melanocortin Therapy Protects Against THSD7A-Associated Membranous NephropathyPoster Session: Glomerular Diseases: Therapeutic StrategiesAbstract: SA-PO715Presentation Time: Saturday, Oct. 26, 10:00 AM-12:00 PMLead Author: Jing Liu The abstracts are available online at asn-online.org. For more information during the conference, visit the ANI Pharmaceuticals booth. About ANI Pharmaceuticals, Inc. ANI Pharmaceuticals, Inc. (Nasdaq: ANIP) is a diversified biopharmaceutical company serving patients in need by developing, manufacturing, and marketing high-quality branded and generic prescription pharmaceutical products, including for diseases with high unmet medical need. ANI is focused on delivering sustainable growth by scaling up its Rare Disease business, strengthening its Generics business with enhanced research and development capabilities, delivering innovation in Established Brands, and leveraging its U.S. based manufacturing footprint. For more information, visit www.anipharmaceuticals.com. Forward-Looking Statements This press release contains not only historical information, but also forward-looking statements made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements represent the Company’s expectations or beliefs concerning future events, including statements regarding the benefits of the acquisition of Alimera Sciences. These forward-looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “continue,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “shall,” “would” other words of similar meaning, derivations of such words and the use of future dates. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. The following factors, among others, could cause actual results to differ materially from those described in these forward-looking statements: (i) the ability to implement business plans, forecasts, and other expectations in connection with the acquisition and integration of Alimera Sciences, Inc. (“Alimera”) and identify and realize additional opportunities and, in particular, the possibility that the Company is unable to achieve anticipated synergies, (ii) costs and regulatory requirements relating to contract manufacturing arrangements, (iii) costs or delays associated with manufacturing (including the sources and any changes in sources thereof) of the Company’s products, (iv) delays or failures in retaining and obtaining continuing and future product approvals from the FDA, and other regulatory issues relating to the Company's business and products, (v) market trends for the Company’s products, and the ability to achieve anticipated sales for such products, (vi) risks that the acquisition of Alimera may disrupt current plans and operations of the Company and potential difficulties of the Company in retaining employees of Alimera and/or maintaining business relationships of Alimera, (vii) the impact of any litigation to which the Company is, or may become, a party, including in connection with the acquisition and integration thereof, (viii) volatility in the Company’s stock price, including as a result of the acquisition, (ix) changes in competitive and regulated industries in which the Company operates, variations in operating performance across competitors, changes in laws and regulations affecting the Company’s business, and changes in the Company’s capital structure as a result of the acquisition, (x) regulatory and other approvals relating to product development and manufacturing, (xi) the Company’s ability, and that of its suppliers, development partners, and manufacturing partners, to comply with laws, regulations and standards that govern or affect the pharmaceutical and biotechnology industries and/or the Company and its products, (xii) costs incurred in connection with the acquisition of Alimera and the possibility that the Company is unable to realize anticipated benefits of the acquisition or to realize estimated pro forma results and underlying assumptions, (xiii) delays in production, increased costs and potential loss of revenues if there is a change in manufacturers or manufacturing processes due to the limited number of suppliers for the Company’s raw materials, active pharmaceutical ingredients, excipients and other materials, (xiv) the Company’s reliance on single source third-party contract manufacturing supply for certain of its key products, (xv) changes in policy or actions that may be taken by the FDA, United States Drug Enforcement Administration, and other regulatory agencies, including among other things, drug recalls, regulatory approvals, facility inspections and potential enforcement actions, (xvi) the impact of legislative or regulatory reform on the pricing for the Company’s products, (xvii) the Company’ ability to maintain the services of its key executives and other personnel, and (xviii) general business and economic conditions, such as inflationary pressures, geopolitical conditions including, but not limited to, the conflict between Russia and the Ukraine, the conflict between Israel and Gaza, conflicts related to the attacks on cargo ships in the Red Sea, and the effects and duration of outbreaks of public health emergencies. More detailed information on these and additional factors that could affect the Company’s actual results are described in the Company’s filings with the Securities and Exchange Commission (“SEC”), including its most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other filings with the SEC. All forward-looking statements in this news release speak only as of the date of this news release and are based on the Company’s current beliefs, assumptions, and expectations. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Media Contact: Chris SaundersT: 484-794-6525E: chris.saunders@anipharmaceuticals.com Source: ANI Pharmaceuticals, Inc.

ROCKVILLE, Md. & EDMONTON, Alberta--(BUSINESS WIRE)--Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (Aurinia or the Company), announced today the acceptance of six abstracts, including five poster presentations, at the American Society of Nephrology (ASN) Kidney Week 2024 taking place in San Diego, CA October 23-27. These data reinforce the clinical importance of LUPKYNIS ® (voclosporin), a second generation calcineurin inhibitor (CNI), for the treatment of adults with active lupus nephritis (LN). “The data featured at ASN elucidate the critical role LUPKYNIS plays in advancing the treatment of lupus nephritis, especially in populations disproportionately impacted by the disease. We’re particularly excited about moving our ENLIGHT-LN™ registry forward, as clinicians need insights into real-world treatment patterns. Additionally, we continue adding to our growing body of evidence that the unique mechanism of action of LUPKYNIS affects podocytes, resulting in significant impact on kidney health in adults with active LN,” said Dr. Greg Keenan, Chief Medical Officer of Aurinia. New real-world baseline data from ENLIGHT-LN, a U.S.-based prospective, observational registry of adult patients with LN treated with LUPKYNIS This initial analysis of baseline data from 123 patients with biopsy-confirmed LN who are initiating or have initiated treatment with LUPKYNIS within the last 12 months provides a critical foundation for understanding the real-world effectiveness and usage patterns of LUPKYNIS. The subset of patients includes high percentages of Black and Hispanic and/or Latino patients, reflecting the larger population of LN in the US and the communities disproportionately affected by lupus and LN. Two new studies uncover the potential effects of LUPKYNIS on podocytes in LN and idiopathic nephrotic syndrome compared to older treatments A new study explored the impact of cyclosporine A (CsA), tacrolimus (TAC), and LUPKYNIS on podocyte cell biology and biomechanics. Using an integrative approach that combined classical cell biological assay with transcriptomics, quantitative proteomics, and other analyses, the study found that all three therapies modulate podocyte cytoskeletal architecture, but LUPKYNIS enhanced podocyte cytoskeletal stability at lower concentrations than CsA and TAC. Another study investigated the effects of LUPKYNIS in a preclinical model of idiopathic nephrotic syndrome. Results showed that LUPKYNIS significantly improved proteinuria, reduced hypercoagulability, enhanced lipid pro and showed significantly better cell viability and cytoskeletal integrity, in comparison to CsA. Together, these findings highlight the important role that LUPKYNIS plays in preserving kidney function and provide further evidence differentiating LUPKYNIS from first-generation calcineurin inhibitors. Following is the complete guide to Aurinia’s accepted abstracts at ASN 2024: Title: Enlight-LN Registry: Baseline Demographics and Clinical Characteristics of an Initial Cohort of Patients Treated with Voclosporin for Lupus Nephritis in the United States Authors: Niloofar Nobakht, Laura B. Geraldino-Pardilla, Leanna M. Wise, Mohammad Kamgar, Lily Cipolla, Lucy S. Hodge, Keelin Dahl Date: Thursday, October 24, 2024 Time: 10:00 a.m. – 12:00 p.m. PT Poster Number: TH-PO653 Title: Disease Targeting Properties of Voclosporin in Renal Transplant and Lupus Nephritis Patients Authors: Simon Zhou, Linda M. Rehaume, Ernie Yap, Henry Leher, Lucy Hodge, Robert B. Huizinga Date: Thursday, October 24, 2024 Time: 10:00 a.m. – 12:00 p.m. PT Poster Number: TH-PO642 Title: Immunologic Changes Over Time in Repeat Kidney Biopsies from the AURORA Studies of Voclosporin in Lupus Nephritis Authors: Samir V. Parikh, Maddalena Bolognesi, Ivana Grbesa, Brad H. Rovin, Giorgio Cattoretti, Vincenzo L’Imperio, Arnon Arazi, Lucy S. Hodge, Ernie Yap Date: Friday, October 25, 2024 Time: 10:00 a.m. – 12:00 p.m. PT Poster Number: FR-PO839 Title: Integrative Systems Analyses Reveal Calcineurin Inhibitor-Mediated Control of Human Podocyte Biophysics and Physiology Authors: Jacob M. Wright, Anthony Mendoza, Jonathan C. Haydak, Jenny Wong, Linda M. Rehaume, Kirk N. Campbell, Evren U. Azeloglu, Maria Paola Santini Date: Saturday, October 26, 2024 Time: 10:00 a.m. – 12:00 p.m. PT Poster Number: SD-PO549 Title : Voclosporin Ameliorates Proteinuria and Directly Protects Podocytes in a Model of Non-Inflammatory Glomerular Disease Authors: Yu Kamigaki, Julie A. Dougherty, Amanda P. Waller, Katelyn J. Wolfgang, Linda M. Rehaume, Jennifer L. Cross, Laura E. Biederman, Zackary S. Stevenson, Eman Abdelghani, Bryce A. Kerlin, William E. Smoyer Date: Saturday, October 26, 2024 Time: 10:00 a.m. – 12:00 p.m. PT Poster Number: SA-PO709 Title : Characterization of Screening Patterns and Identification of Patients with Lupus Nephritis in a Community Rheumatology Setting Authors: Nehad Soloman, Jawad Bilal, Romy Cabacungan, Scott Milligan, Andrew Sharobeem, John Tesser, Henry Leher About Lupus Nephritis Lupus nephritis (LN) is a serious manifestation of systemic lupus erythematosus (SLE), a chronic and complex autoimmune disease. LN affects approximately 120,000 people in the U.S. and disproportionately affects women and people of color. People living with LN have high unmet needs and often face significant barriers to optimal care. If poorly controlled, LN can lead to permanent and irreversible tissue damage within the kidney. Medical guidelines recommend that all SLE patients receive routine LN screenings at every visit. Guidelines also note that delaying LN diagnosis has profound prognostic repercussions. Yet, research shows that approximately 50% of SLE patients are not screened for LN and 77% of people with LN go untreated. Aurinia is committed to improving health outcomes for people living with LN by educating patients and providers on the critical need for routine screening and transformative therapies that can help improve health outcomes. About LUPKYNIS LUPKYNIS is a second generation calcineurin inhibitor (CNI) with a dual mechanism of action, acting as an immunosuppressant through inhibition of T-cell activation and cytokine production and promoting podocyte stability in the kidney. The AURORA Clinical Program, comprised of the AURORA 1 pivotal trial and AURORA 2 extension trial, demonstrated the importance of LUPKYNIS plus standard of care to preserve kidney health in patients with active LN without reliance on chronic high-dose glucocorticoids. It is the only clinical program to include three years of LN treatment and follow-up with mycophenolate mofetil (MMF) and steroids. About Aurinia Aurinia Pharmaceuticals is a fully integrated biopharmaceutical company focused on delivering therapies to people living with autoimmune diseases with high unmet medical needs. In January 2021, the Company introduced LUPKYNIS ® (voclosporin), the first FDA-approved oral therapy dedicated to the treatment of adult patients with active lupus nephritis. Aurinia is also developing AUR200, a differentiated, potential best-in-class therapy for autoimmune diseases that targets both BAFF (B-cell Activating Factor) and APRIL (A Proliferation-Inducing Ligand). INDICATION AND IMPORTANT SAFETY INFORMATION INDICATION LUPKYNIS is indicated in combination with a background immunosuppressive therapy regimen for the treatment of adult patients with active lupus nephritis (LN). Limitations of Use: Safety and efficacy of LUPKYNIS have not been established in combination with cyclophosphamide. Use of LUPKYNIS is not recommended in this situation. IMPORTANT SAFETY INFORMATION BOXED WARNINGS: MALIGNANCIES AND SERIOUS INFECTIONS Increased risk for developing malignancies and serious infections with LUPKYNIS or other immunosuppressants that may lead to hospitalization or death. CONTRAINDICATIONS: LUPKYNIS is contraindicated in patients taking strong CYP3A4 inhibitors because of the increased risk of acute and/or chronic nephrotoxicity, and in patients who have had a serious/severe hypersensitivity reaction to LUPKYNIS or its excipients. WARNINGS AND PRECAUTIONS Lymphoma and Other Malignancies: Immunosuppressants, including LUPKYNIS, increase the risk of developing lymphomas and other malignancies, particularly of the skin. The risk appears to be related to increasing doses and duration of immunosuppression rather than to the use of any specific agent. Serious Infections: Immunosuppressants, including LUPKYNIS, increase the risk of developing bacterial, viral, fungal, and protozoal infections, including opportunistic infections which lead to serious, including fatal outcomes. Nephrotoxicity: LUPKYNIS, like other calcineurin inhibitors (CNIs), may cause acute and/or chronic nephrotoxicity. The risk is increased when CNIs are concomitantly administered with drugs associated with nephrotoxicity. Monitor eGFR regularly. Hypertension: Hypertension is a common adverse reaction of LUPKYNIS therapy and may require antihypertensive therapy. Monitor blood pressure regularly. Neurotoxicity: LUPKYNIS, like other CNIs, may cause a spectrum of neurotoxicities: severe include posterior reversible encephalopathy syndrome (PRES), delirium, seizure, and coma; others include tremor, paresthesia, headache, and changes in mental status and/or motor and sensory functions. Monitor for neurologic symptoms. Hyperkalemia: Hyperkalemia, which may be serious and require treatment, has been reported with CNIs, including LUPKYNIS. Concomitant use of agents associated with hyperkalemia may increase the risk for hyperkalemia. Monitor serum potassium levels periodically. QTc Prolongation: LUPKYNIS prolongs the QTc interval in a dose-dependent manner when dosed higher than the recommended lupus nephritis therapeutic dose. The use of LUPKYNIS in combination with other drugs that are known to prolong QTc may result in clinically significant QT prolongation. Immunizations: Avoid the use of live attenuated vaccines during treatment with LUPKYNIS. Inactivated vaccines noted to be safe for administration may not be sufficiently immunogenic during treatment with LUPKYNIS. Pure Red Cell Aplasia: Cases of pure red cell aplasia (PRCA) have been reported in patients treated with another CNI immunosuppressant. If PRCA is diagnosed, consider discontinuation of LUPKYNIS. Drug-Drug Interactions: Avoid co-administration of LUPKYNIS and strong CYP3A4 inhibitors or with strong or moderate CYP3A4 inducers. Co-administration of LUPKYNIS with strong CYP3A4 inhibitors is contraindicated. Reduce LUPKYNIS dosage when co-administered with moderate CYP3A4 inhibitors. Avoid use of LUPKYNIS with strong or moderate CYP3A4 inducers. ADVERSE REACTIONS The most common adverse reactions (≥3%) were glomerular filtration rate decreased, hypertension, diarrhea, headache, anemia, cough, urinary tract infection, abdominal pain upper, dyspepsia, alopecia, renal impairment, abdominal pain, mouth ulceration, fatigue, tremor, acute kidney injury, and decreased appetite. SPECIFIC POPULATIONS Pregnancy: Avoid use of LUPKYNIS. Lactation: Consider the mother’s clinical need for LUPKYNIS and any potential adverse effects to the breastfed infant when prescribing LUPKYNIS to a lactating woman. Renal Impairment: LUPKYNIS is not recommended in patients with baseline eGFR ≤45 mL/min/1.73 m 2 unless benefit exceeds risk. If used in this population, reduce LUPKYNIS dose. Hepatic Impairment: For mild or moderate hepatic impairment, reduce LUPKYNIS dose. Avoid use with severe hepatic impairment. Please see Prescribing Information , including Boxed Warning, and Medication Guide for LUPKYNIS. Contacts Media & Investor Inquiries: Andrea Christopher Corporate Communications & Investor Relations Aurinia Pharmaceuticals Inc. achristopher@auriniapharma.com General Investor Inquiries: ir@auriniapharma.com

Purespring Therapeutics raises £80/$105 million in a Series B financing to transform the treatment of kidney diseases Oversubscribed financing led by Sofinnova Partners in collaboration with Gilde Healthcare, Forbion, British Patient Capital and founding investor Syncona Funds raised will support Purespring in advancing its AAV pipeline of gene therapy programmes to treat kidney disease, including its lead programme targeting IgA Nephropathy (IgAN) London – 9 October 2024 - Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, today announces it has raised £80/$105 million in a Series B financing. The oversubscribed round was led by Sofinnova Partners, in collaboration with a prominent syndicate including Gilde Healthcare, Forbion, British Patient Capital and founding investor Syncona Limited. Purespring is the first company to successfully treat kidney disease models by directly targeting the podocyte, a specialised cell implicated in approximately 60% of renal diseases, through its proprietary adeno-associated viral (AAV) gene therapy platform. The Company currently has a pipeline of programmes in development. This includes its lead programme PS-002, in development for IgA Nephropathy (IgAN), and other complement mediated kidney diseases, a programme targeting nephrotic syndrome, and an undisclosed glomerular kidney disease programme. Purespring’s platform technology allows working copies of genes to be delivered with high efficiency and specificity to podocytes, opening up a new and highly differentiated modality for disease modifying treatments of a broad range of kidney diseases. Proceeds will be used to support Purespring’s pipeline including the initiation of a Phase I/II clinical trial for IgAN, a common, chronic kidney disease primarily affecting young adults. About one third of IgAN patients will go on to lose their kidney function within five years and require a kidney transplant or dialysis. Julian Hanak, Purespring’s Chief Executive Officer, commented: “Nearly one tenth of the world’s population, around 840 million people, suffer from chronic kidney disease. For many of them, there are few options beyond dialysis and transplantation. Our novel treatment platform and deep understanding of kidney disease puts us in a position to stop, reverse and even cure kidney disease. The funds raised will allow us to bring our novel treatments to patients in the clinic. “I’m really pleased to be able to welcome such a high-quality investor syndicate to support Purespring on this journey. Having sophisticated, experienced and committed investors on board, who understand drug development and the urgency of bringing these much-needed therapies to patients will maximise the opportunities for the company and the patients we treat.” Maina Bhaman, Partner at Sofinnova Partners, added: “Purespring Therapeutics is pioneering the treatment of kidney disease. The team’s ambition and calibre epitomise Sofinnova’s mission of scientific and entrepreneurial excellence in delivering transformative treatments to patients. We are excited to support the Purespring team as they bring their groundbreaking innovation to the global stage.” Michael Kyriakides, Investment Partner at Syncona Investment Management and Board Director of Purespring Therapeutics, said: “Syncona founded Purespring with the vision of creating the world’s leading kidney gene therapy company, and we have been impressed with its progress. Today’s financing and the strong syndicate of investors that the Company has attracted broadens Purespring’s financial scale and clearly validates its technology, strategy and execution to date. We look forward to working alongside Purespring and its new syndicate as it prepares for its Phase I/II clinical trial.” The financing follows critical scientific milestones for Purespring. Earlier this year, Purespring presented preclinical data at the 61st European Renal Association (ERA) Congress establishing the potential of AAV gene therapy to deliver transgenes to the podocyte to replace defective genes or to modulate protein production, underpinning the potential of AAV as an important novel modality to treat a broad range of kidney disease. In 2023, a paper published in Science Translational Medicine demonstrated that Purespring had successfully used AAV gene therapy to transduce the podocyte in vivo for the first time – a major milestone in overcoming the hurdles which have previously limited development in this area. Purespring will present its IgAN data publicly for the first time at the American Society of Nephrology (ASN) Kidney Week congress on October 26 in San Diego (abstract #SA-OR63). For further information, contact: Purespring: Julian Hanak, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn ICR Consilium Amber Fennell, Jessica Hodgsonpurespring@consilium-comms.com Notes to Editors About Purespring Purespring is developing gene therapies to halt or prevent kidney disease, one of humankind’s most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring’s platform approach enables streamlined gene therapy development for both monogenic and non-monogenic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for diseases caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and British Patient Capital and has raised £115M to date. For more information please visit: purespringtx.com and follow us on LinkedIn. About Syncona Syncona's purpose is to invest to extend and enhance human life. We do this by creating, building and scaling companies to deliver transformational treatments to patients in areas of high unmet need. We aim to build and maintain a diversified portfolio of 20-25 globally leading life science businesses, across development stage, modality and therapeutic area, for the benefit of all our stakeholders. We focus on developing treatments that deliver patient impact by working in close partnership with world-class academic founders and experienced management teams. Our balance sheet underpins our strategy, enabling us to take a long-term view as we look to improve the lives of patients with no or poor treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders. About Sofinnova Partners Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. Founded in 1972, Sofinnova Partners is a deeply-established venture capital firm in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners has over €2.8 billion under management. For more information, please visit: www.sofinnovapartners.com.