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CTV News
5 hours ago
- CTV News
You can slow cognitive decline as you age, large study finds. Here's how
At 62, Phyllis Jones felt trapped in darkness. She was traumatized by her mother's recent death, ongoing pandemic stress and an increasingly toxic work environment. A sudden panic attack led to a medical leave. Her depression worsened until the day her 33-year-old son sadly told her, 'Mom, I didn't think I would have to be your caregiver at this stage in your life.' 'For me, that was the wake-up call,' Jones, now 66, told CNN. 'That's when I found the POINTER study and my life changed. What I accomplished during the study was phenomenal — I'm a new person.' The Protect Brain Health Through Lifestyle Intervention to Reduce Risk, or U.S. POINTER study, is the largest randomized clinical trial in the United States designed to examine whether lifestyle interventions can protect cognitive function in older adults. 'These are cognitively healthy people between the ages of 60 and 79 who, to be in the study, had to be completely sedentary and at risk for dementia due to health issues such as prediabetes and borderline high blood pressure,' said principal investigator Laura Baker, a professor of gerontology, geriatrics and internal medicine at Wake Forest University School of Medicine in Winston-Salem, North Carolina. Approximately one-half of the 2,111 study participants attended 38 structured team meetings over two years in local neighborhoods near Chicago, Houston, Winston-Salem, Sacramento, California, and Providence, Rhode Island. During each session, a trained facilitator provided guidance on how to exercise and eat for the brain, and explained the importance of socialization, the use of brain-training games, and the basics of brain health. The team leader also held the group accountable for logging blood pressure and other vitals. Physical and cognitive exams by a physician occurred every six months. At six team meetings, the other half of the study's participants learned about brain health and were encouraged to select lifestyle changes that best suited their schedules. This group was self-guided, with no goal-directed coaching. These participants also received physical and cognitive exams every six months. The two-year results of the US$50 million study, funded by the Alzheimer's Association, were simultaneously presented Monday at the 2025 Alzheimer's Association International Conference in Toronto and published in the journal JAMA. 'We found people in the structured program appeared to delay normal cognitive aging by one to nearly two years over and above the self-guided group — people who did not receive the same degree of support,' Baker said. 'However, the self-guided group improved their cognitive scores over time as well.' Exercise, diet and socializing are key Exercise was the first challenge. Like the other groups across the country, Jones and her Aurora, Illinois, team received YMCA memberships and lessons on how to use the gym equipment. Jones was told to use aerobic exercise to raise her heart rate for 30 minutes a day while adding strength training and stretching several times a week. At first, it wasn't easy. The study participants wore fitness trackers that monitored their activity, Jones said. 'After that first 10 minutes, I was sweating and exhausted,' she said. 'But we went slow, adding 10 minutes at a time, and we kept each other honest. Now I just love to work out.' Four weeks later, teams were given a new challenge — beginning the Mediterranean-DASH Intervention for Neurodegenerative Delay, or MIND diet. The diet combines the best of the Mediterranean diet with the salt restrictions of the DASH diet, which stands for Dietary Approaches to Stop Hypertension. 'They gave us a refrigerator chart with foods to limit and foods to enjoy,' Jones said. 'We had to eat berries and vegetables most days, including green leafy veggies, which was a separate item. We had to have 2 tablespoons of extra-virgin olive oil once every day.' Foods to limit included fried food, processed meat, dairy, cheese and butter. Restrictions were also in place for sugary sweets. 'But we could have dessert four times a week,' Jones added. 'That's awesome because you're not completely depriving yourself.' Another pillar of the program was requiring study participants to familiarize themselves with their vital signs, Wake Forest's Baker said. 'If at any point we asked them, 'What's your average blood pressure?' they should be able to tell us,' she said. 'We encouraged people to monitor their blood sugar as well.' Later came brain training, via memberships to a popular, Web-based cognitive training app. While some scientists say the benefits of such online brain programs have yet to be proven, Jones said she enjoyed the mental stimulation. Becoming better at socializing was another key part of the program. The researchers tasked teams with assignments, such as speaking to strangers or going out with friends. 'I found my best friend, Patty Kelly, on my team,' Jones said. 'At 81, she's older than me, but we do all sorts of things together — in fact, she's coming with me to Toronto when I speak at the Alzheimer's conference. 'Isolation is horrible for your brain,' she added. 'But once you get to a point where you are moving and eating healthy, your energy level changes, and I think you automatically become more social.' As the study progressed, the researchers reduced check-ins to twice a month, then once a month, Baker said. 'We were trying to get people to say, 'I am now a healthy person,' because if you believe that, you start making decisions which agree with the new perception of yourself,' she said. 'So in the beginning, we were holding their hands, but by the end, they were flying on their own,' Baker added. 'And that was the whole idea — get them to fly on their own.' 'Brain health is a long game' Because researchers tracked each team closely, the study has a wealth of data that has yet to be mined. 'On any given day, I could go into our web-based data system and see how much exercise someone's doing, whether they've logged into brain training that day, what's their latest MIND diet score, and whether they'd attended the last team meeting,' Baker said. 'We also have sleep data, blood biomarkers, brain scans and other variables, which will provide more clarity on which parts of the intervention were most successful.' Digging deeper into the data is important, Baker says, because the study has limitations, such as the potential for a well-known phenomenon called the practice effect. 'Even though we use different stimuli within tests, the act of taking a test over and over makes you more familiar with the situation — you know where the clinic is, where to park, you're more comfortable with your examiner,' she said. 'You're not really smarter, you're just more relaxed and comfortable, so therefore you do better on the test,' Baker said. 'So while we're thrilled both groups in US POINTER appear to have improved their global cognition (thinking, learning and problem-solving), we have to be cautious in our interpretations.' It's important to note the POINTER study was not designed to provide the more immersive lifestyle interventions needed for people with early stages of Alzheimer's, said Dr. Dean Ornish, a professor of medicine at the University of California, San Francisco. Ornish published a June 2024 clinical trial that found a strict vegan diet, daily exercise, structured stress reduction and frequent socialization could often stop the decline or even improve cognition in those already experiencing from early-stage Alzheimer's disease, not just for those at risk for it. 'The US POINTER randomized clinical trial is a landmark study showing that moderate lifestyle changes in diet, exercise, socialization and more can improve cognition in those at risk for dementia,' said Ornish, creator of the Ornish diet and lifestyle medicine program and coauthor of 'Undo It!: How Simple Lifestyle Changes Can Reverse Most Chronic Diseases.' 'It complements our randomized clinical trial findings which found that more intensive multiple lifestyle changes often improve cognition in those already diagnosed with early-stage Alzheimer's disease,' Ornish said. 'But the US POINTER study showed that more moderate lifestyle changes may be sufficient to help prevent it.' In reality, two years isn't sufficient to track brain changes over time, said study coauthor Maria Carillo, chief science officer of the Alzheimer's Association. 'We really want to make recommendations that are evidence based,' Carillo told CNN. 'That's why we have invested another $40 million in a four-year follow-up, and I believe over 80% of the original participants have joined. 'Brain health is a long game,' she added. 'It's hard to track, but over time, change can be meaningful.' By Sandee LaMotte, CNN
Globe and Mail
14 hours ago
- Globe and Mail
Cutaneous T-Cell Lymphoma Pipeline Outlook Report 2025: Key 25+ Companies and Breakthrough Therapies Shaping the Future Landscape
DelveInsight's ' Cutaneous T-cell lymphoma Pipeline Insight 2025 ' report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in the Cutaneous T-cell lymphoma pipeline landscape. It covers the Cutaneous T-Cell Lymphoma pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Cutaneous T-Cell Lymphoma pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Explore the comprehensive insights by DelveInsight and stay ahead in understanding the Cutaneous T-Cell Lymphoma Treatment Landscape. Click here to read more @ Cutaneous T-Cell Lymphoma Pipeline Outlook Key Takeaways from the Cutaneous T-Cell Lymphoma Pipeline Report In July 2025, Prescient Therapeutics Ltd. announced a phase 2 study to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmadynamics (PD), of PTX-100 monotherapy at 500 or 1000 mg/m2 in patients with relapsed/refractory Cutaneous T-Cell Lymphoma (CTCL). In July 2025, Dren Bio conducted a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and anti-tumor activity of DR-01 in adult patients with large granular lymphocytic leukemia or cytotoxic lymphomas. In July 2025, Soligenix announced a Phase 3 study is to evaluate the ability of an 18-week course of HyBryte and visible light to induce a Treatment Response in patients with patch/plaque phase CTCL compared to patients receiving placebo and visible study will evaluate the efficacy and safety of HyBryte (0.25% hypericin) gel or placebo gel applied twice weekly for 18 weeks. Treated lesions will be covered with opaque material (such as opaque clothing), followed 21 (±3) hours later by the administration of visible light. DelveInsight's Cutaneous T-Cell Lymphoma pipeline report depicts a robust space with 25+ active players working to develop 30+ pipeline therapies for Cutaneous T-Cell Lymphoma treatment. The leading Cutaneous T-Cell Lymphoma Companies such as HyBryte, Resminostat, Mundipharma International, Shanghai Pharmaceuticals Holding, Moleculin Biotech, Inc., BeiGene, Mundipharma Research Limited, Jiangsu Simcere Biologics Co., Ltd, Teva Pharmaceuticals USA, Pfizer, Galderma R&D, Merck Sharp & Dohme LLC, Bio-Path Holdings, Inc., Kymera Therapeutics, Inc., Karyopharm Therapeutics Inc and others. Promising Cutaneous T-Cell Lymphoma Pipeline Therapies such as E7777, CD11301 0.03%, Panobinostat, ONTAK (denileukin difitox, DAB389IL-2), Quisinostat, 12 mg, APO866, Enzastaurin, SGX301 (synthetic hypericin), Mogamulizumab, Romidepsin (depsipeptide, FK228), and others. Stay informed about the cutting-edge advancements in Cutaneous T-Cell Lymphoma Treatments. Download for updates and be a part of the revolution in oncology care @ Cutaneous T-Cell Lymphoma Clinical Trials Assessment Cutaneous T-cell lymphoma Emerging Drugs HyBryte™ (synthetic hypericin or SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. Synthetic hypericin sodium, the active ingredient in HyBryte™, has Orphan Drug designation in the United States for the treatment of T-cell lymphoma and CTCL and in Europe for CTCL. HyBryte™ has received Fast Track designation for the treatment of cutaneous t-cell lymphoma in the United States. AFM13: Affimed GmbH AFM-13 is under development for the treatment of refractory and relapsed Hodgkin lymphoma, CD30+ lymphoma such as transformed mycosis fungoides, peripheral and cutaneous T-cell lymphoma, large B-cell lymphoma, B-cell non-Hodgkin lymphoma, Hodgkin lymphoma combination with check point inhibitors and Hodgkin lymphoma combination with lenalidomide. The drug candidate is administered intravenously. AFM-13 is a bi-specific, tetravalent human antibody, it acts by targeting CD30/CD16A. The drug is currently in Phase II stage of its development for the treatment of Cutaneous T Cell Lymphoma. ASTX660: Otsuka Pharmaceutical Co., Ltd ASTX660 (Tolinapant) is a novel, orally administered, non-peptidomimetic antagonist of the cellular and X-linked inhibitors of apoptosis proteins (cIAP1/2 and XIAP). Inhibitors of apoptosis proteins (IAPs) are frequently overexpressed in tumor cells and contribute to tumor cell survival and chemo-resistance. By inhibiting IAPs, tolinapant promotes cell death. Tolinapant also acts via a newly described immunomodulatory mechanism, which works to enhance an anti-tumor immune response in T-cell lymphomas. The drug is in Phase I/II for the treatment of CTCL. WUCART007: Wugen WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. Currently, the drug is in Phase I stage of its clinical trial for the treatment of CTCL. The Cutaneous T-Cell Lymphoma pipeline report provides insights into The report provides detailed insights about companies that are developing therapies for the treatment of Cutaneous T-Cell Lymphoma with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cutaneous T-Cell Lymphoma Treatment. Cutaneous T-Cell Lymphoma Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Cutaneous T-Cell Lymphoma Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Cutaneous T-Cell Lymphoma market. Get a detailed analysis of the latest innovations in the Cutaneous T-Cell Lymphoma pipeline. Explore DelveInsight's expert-driven report today! @ Cutaneous T-Cell Lymphoma Unmet Needs Cutaneous T-Cell Lymphoma Companies HyBryte, Resminostat, Mundipharma International, Shanghai Pharmaceuticals Holding, Moleculin Biotech, Inc., BeiGene, Mundipharma Research Limited, Jiangsu Simcere Biologics Co., Ltd, Teva Pharmaceuticals USA, Pfizer, Galderma R&D, Merck Sharp & Dohme LLC, Bio-Path Holdings, Inc., Kymera Therapeutics, Inc., Karyopharm Therapeutics Inc and others. Cutaneous T-cell lymphoma pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Cutaneous T-Cell Lymphoma Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Discover the latest advancements in Cutaneous T-Cell Lymphoma Treatment by visiting our website. Stay informed about how we're transforming the future of oncology @ Cutaneous T-Cell Lymphoma Market Drivers and Barriers, and Future Perspectives Scope of the Cutaneous T-Cell Lymphoma Pipeline Report Coverage- Global Cutaneous T-Cell Lymphoma Companies- HyBryte, Resminostat, Mundipharma International, Shanghai Pharmaceuticals Holding, Moleculin Biotech, Inc., BeiGene, Mundipharma Research Limited, Jiangsu Simcere Biologics Co., Ltd, Teva Pharmaceuticals USA, Pfizer, Galderma R&D, Merck Sharp & Dohme LLC, Bio-Path Holdings, Inc., Kymera Therapeutics, Inc., Karyopharm Therapeutics Inc and others. Cutaneous T-Cell Lymphoma Pipeline Therapies- E7777, CD11301 0.03%, Panobinostat, ONTAK (denileukin difitox, DAB389IL-2), Quisinostat, 12 mg, APO866, Enzastaurin, SGX301 (synthetic hypericin), Mogamulizumab, Romidepsin (depsipeptide, FK228), and others. Cutaneous T-Cell Lymphoma Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Cutaneous T-Cell Lymphoma Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III For a detailed overview of our latest research findings and future plans, read the full details of Cutaneous T-Cell Lymphoma Pipeline on our website @ Cutaneous T-Cell Lymphoma Emerging Drugs and Companies Table of Content Introduction Executive Summary Cutaneous T-cell lymphoma: Overview Pipeline Therapeutics Therapeutic Assessment Cutaneous T-cell lymphoma– DelveInsight's Analytical Perspective Late Stage Products (Phase III) Drug Name: Company Name Mid Stage Products (Phase II) AFM13: Affimed GmbH Early Stage Products (Phase I) WUCART007: Wugen Preclinical Stage Products Drug Name: Company Name Inactive Products Cutaneous T-cell lymphoma Key Companies Cutaneous T-cell lymphoma Key Products Cutaneous T-cell lymphoma- Unmet Needs Cutaneous T-cell lymphoma- Market Drivers and Barriers Cutaneous T-cell lymphoma- Future Perspectives and Conclusion Cutaneous T-cell lymphoma Analyst Views Cutaneous T-cell lymphoma Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Globe and Mail
15 hours ago
- Globe and Mail
FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older
Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits Broad label includes adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrence Well-established safety profile, consistent across >2,200 patient years in approved indications WALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI ® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States. 1 'I'm excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,' said Carla Nester, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. 'With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.' In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. 'EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,' said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. 'As Apellis' third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients' lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.' 'The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,' said Josh Tarnoff, chief executive officer, NephCure. 'We recognize Apellis' commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.' The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease: Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo. Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function compared to placebo (nominal p=0.03) as measured by estimated glomerular filtration rate (eGFR). Reduction of C3 staining: A majority of EMPAVELI-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) compared to placebo. 71% of EMPAVELI-treated patients achieved zero C3 staining intensity, demonstrating complete clearance of C3 deposits. The safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist ® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information. Conference Call and Webcast Apellis will host a conference call and webcast to discuss the FDA's approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the 'Events and Presentations' page of the 'Investors and Media' section of the company's website. A replay of the webcast will be available for 30 days following the event. About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis therapy. 2 -4 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence. 5 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe. 1 About the VALIANT Study The VALIANT Phase 3 study (NCT05067127) was a randomized, placebo-controlled, double-blinded, multi-center study that evaluated EMPAVELI® (pegcetacoplan) efficacy and safety in 124 patients who were 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include pediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received EMPAVELI. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline. About EMPAVELI ® /Aspaveli ® (pegcetacoplan) EMPAVELI ® /Aspaveli ® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases. EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipients For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients' vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at or 1-888-343-7073. Infusion-Related Reactions Systemic hypersensitivity reactions (eg, facial swelling, rash, urticaria, pyrexia) have occurred in patients treated with EMPAVELI, which may resolve after treatment with antihistamines. Cases of anaphylaxis leading to treatment discontinuation have been reported. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved. Interference with Laboratory Tests There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels. ADVERSE REACTIONS Most common adverse reactions in adult and pediatric patients 12 years of age and older with C3G or primary IC-MPGN (incidence ≥10%) were infusion-site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. USE IN SPECIFIC POPULATIONS Females of Reproductive Potential EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose. Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company leading the way in complement science to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit or follow us on LinkedIn and X. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the potential market opportunity of EMPAVELI for C3G and IC-MPGN. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the clinical trial results of EMPAVELI for C3G and IC-MPGN indicate an effect that is greater than the actual positive effect; and any other factors discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Media: Tracy Vineis media@ 617.420.4839 Investors: Neil Carnahan, CFA 617.977.5703 References 1. Data on file using literature consensus. 2. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143. 3. Servais A, et al. Kidney Int. 2012;82(4):454-464. 4. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117. 5. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474. A photo accompanying this announcement is available at
