Crouzon Syndrome With Acanthosis Nigricans

The figures for Rights of Use assets (6,620 KSEK) were missing in the previous report and have now been added. The total sums for Fixed Assets and the Balance Sheet remain the same. MÖLNDAL, Sweden, Feb. 29, 2024 /PRNewswire/ -- THIRD QUARTER 2023/24 (NOV - JAN) Net sales amounts to 30.1 (18.7) MSEK, which is an increase of 61% compared with the corresponding period last year. The growth in the US market is 43% compared with the corresponding period last year. Operating profit amounts to 0.3 (-13.8) MSEK (EBIT 1.1%). The operating profit amounted to 6.1 MSEK (EBIT 20.2%) excluding currency effects of -5.7 MSEK, most of which are unrealized internal accounts receivables. Profit/loss after tax amounts to 0.8 (-13.6) MSEK, which resulted in profit per share of SEK 0.05 (-0.74). Cash flow from operating activities amounts to 2.0 MSEK (-14.1), change in working capital was -2.9 MSEK (8.2) and investing activities -4.0 (-1.2). Total cash flow for the period was -5.0 (-7.1) MSEK. Liquid assets as of 2024-01-31 amount to 28.4 (46.3) MSEK, not including 29.2 MSEK in accounts receivables. Significant events in the third quarter The US Department of Defense decided to fully fund a clinical study of OPRA™ Implant System on up to 30 patients with transtibial (below-knee) amputations. The study will be performed by the Walter Reed National Military Medical Center. Integrum will supply OPRA™ Implant Systems on commercial terms. The company held a pre-submission meeting with the US Food and Drug Administration (FDA). Based on the conclusions from the meeting, Integrum plans to submit a Pre-Market Approval (PMA) application for Integrum's OPRA™ Implant System in transhumeral amputations based on existing clinical data. Integrum received its final MDR certification. The company's OPRATM Implant System product portfolio has thereby been granted MDR certification and may be freely distributed within the EU for treatment in individuals living with disabilities because of limb loss. The company established a Center of Excellence at the Center for Complex Endoprosthetics, Osseointegration and Bionics in Kyiv, with the intention to spearhead orthopedic treatment using OPRA™ Implant System in individuals who have suffered limb loss. 1 MAY - 31 JANUARY 2022/23 (9 MONTHS) Net sales amounts to 78.1 (56.1) MSEK, which is an increase of 39% compared with the corresponding period last year. The growth in the US market is 29% compared with the corresponding period last year Operating profit amounts to 3.9 (-12.5) MSEK (EBIT 5.0%). The operating profit amounted to 2.5 MSEK (EBIT 3.2%), excluding currency effects of 1.4 MSEK, most of which are unrealized internal accounts receivables. Profit/loss after tax amounts to 1.9 (-10.1) MSEK, which resulted in earnings per share of SEK 0.1 (-0.55). Cash flow from operating activities amounts to 2.6 (-17.1) MSEK. Total cash flow was -13.6 (-29.0) MSEK. During the year, The Centers for Medicare and Medicaid Services (CMS) granted the osseointegrated external prosthetic connection device Axor II™ a new reimbursement code. The new code is connected to a set reimbursement that will help to drive growth in the US. CEO's statement The third quarter has been characterized by record sales figures, mainly driven by our continued expansion in the US and a recent accelerating growth in emerging markets. Yet another quarter with record sales and profit Revenues amounted to 30.1 MSEK in the third quarter, corresponding to an increase of 61.2 percent compared to the same period last year. Excluding direct currency effects, operating profit amounted to 6.1 MSEK leading to an EBIT margin of 20.2%. Enhanced collaborations in the US In the US, which is our prioritized market, we have worked to deepen existing collaborations with hospitals offering treatment with our innovative products. The work is based on Integrum's network model to collect and connect healthcare professionals that surround the patient undergoing treatment with the OPRA™ Implant system. By building an ecosystem with all key players, we lessen hospitals' administrative burden and help them funnel patients through the treatment process. Ultimately, we believe this will increase patient turnover and, most importantly, improve the individual patient's healthcare experience. In November, we announced our plans to apply for pre-market approval (PMA) for the use of the OPRA™ Implant System in above-elbow treatment. Following a successful meeting with the FDA, we are working with high focus to compile the necessary regulatory documentation needed for a formal application. Expansion into new markets Integrum's innovative and bone-anchored OPRA™ Implant System restores mobility in a completely different way than traditional socket prostheses. To this end, our ambition is to treat as many amputees as possible, as quickly as possible, with the OPRA™ Implant System to improve their quality of life. Hence, during the fall, alongside our US operations, we have focused on making the OPRA™ Implant System available in Ukraine and Turkey - two countries that, for various reasons, have seen rapidly growing groups of amputees. In December, we announced the establishment of a clinical center of excellence in Kyiv - aimed at training Ukrainian surgeons in using the OPRA™ Implant System. During the fall, I visited the center three times to perform surgeries, initially pro bono to swiftly get the center into operations. In January, the center independently performed seven surgeries, and is now conducting self-sufficient clinical activities offering the OPRA™ Implant System. The collaboration has generated orders already in the short term; at the end of January, Integrum sold and delivered OPRATM Implant Systems to a value of SEK 3.5 million. The need for Integrum's bone-anchored prosthetic treatments in Ukraine is partly driven by an increased incidence of the tourniquet syndrome, which primarily affects soldiers at the front. The syndrome occurs when treatment of extensive injuries to limbs is delayed. Due to the restricted blood flow during acute treatment with torniquets, the affected individual is usually left with a short stump. In these cases, socket prostheses use is almost impossible, unlike the OPRA™ Implant System which can restore full mobility. During my last visit in Kyiv, I met with Ukraine's Minister of Health, Viktor Liashko, who expressed the country's extensive need for high-standard prosthetic treatments and its willingness to invest in restoring the population's health. According to the Ukrainian Ministry of Health, nearly 100,000 people are expected to have an amputation at the end of 2024. In Turkey, since the end of October, we have been working with our partner Medikon to establish a business solution to offer treatments for people injured in the country's extensive earthquake in early 2023. A skilful organization to achieve high growth We have, time and again, shown the overturning potential in our innovative bone-anchored solutions - based both on a large body of scientific and clinical evidence, as well as important public recognition. As the organization has grown with skilled experts in key positions, we now see an important shift in our regulatory and commercial activities laying the foundation for meaningful growth. Integrum is the world's foremost company in in advanced amputation treatment, and we look forward to leading the way in restoring mobility globally. Mölndal, 29 February 2024 Rickard Brånemark Chief Executive Officer The interim report can be found here: This disclosure contains information that Integrum AB is obliged to make public pursuant to the EU Market Abuse Regulation (EU nr 596/2014). The information was submitted for publication, through the agency of the contact person, on 29-02-2024 09:39 CET. For more information please contact: Rickard Brånemark, CEO. Phone: +46 70 846 10 61, E-mail: [email protected] Jörgen Svanström, CFO. Phone: +46 70 734 96 60, E-mail: [email protected] Certified Adviser: The Company's Certified Adviser is Carnegie Investment Bank AB (publ). The following files are available for download: SOURCE Integrum AB

Expanded access program (EAP) data for omidubicel are consistent with Phase 3 trial results on rates of hematopoietic recovery and infections following stem cell transplant with Omisirge® (omidubicel-onlv) Preliminary data presented on GDA-201, Gamida Cell’s natural killer (NK) cell therapy candidate in ongoing Phase 1 study for non-Hodgkin lymphoma, show promising early evidence of anti-tumor activity BOSTON, Feb. 23, 2024 (GLOBE NEWSWIRE) -- Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, today presented data highlighting its expanded access program (EAP) for FDA-approved allogeneic stem cell therapy Omisirge® (omidubicel-onlv) and Phase 1 data for its allogeneic cryopreserved natural killer (NK) cell therapy candidate GDA-201 at the 2024 Tandem Meetings, Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR). The hybrid meetings take place February 21-24 virtually and in person in San Antonio, Texas. “The data presented at Tandem provide further evidence of the potential of Gamida Cell’s nicotinamide (NAM) technology to develop potentially curative therapies by expanding and enhancing cells,” said Ronit Simantov, MD, Chief Medical and Scientific Officer of Gamida Cell. “The EAP study provided the opportunity to treat patients after completion of enrollment of the Phase 3 trial and prior to the approval of Omisirge. It allowed for institutional variability in conditioning regimens and supportive care, more closely reflecting the real-world environment. Data were consistent with previous studies, showing that patients transplanted with omidubicel had rapid hematopoietic recovery and a low rate of serious infections post-transplant.” “The Phase 1 data on our natural killer (NK) cell candidate GDA-201 further support the anti-tumor activity of our NAM-enhanced cellular therapy, and today marked the first presentation of study results using our cryopreserved, readily available formulation,” Simantov added. Reporting on omidubicel’s EAP data, principal investigator Mitchell E. Horwitz, MD, Stem Cell Transplant Specialist and Professor of Medicine at Duke Cancer Institute, said, “I am highly encouraged by the results in the omidubicel expanded access program. The potential for rapid hematopoietic recovery post-transplant observed with omidubicel could make a meaningful impact on patient health. I am pleased to see that the EAP results are consistent with data from the Phase 3 study.” Additional details on the presentations are as follows: Title: Omidubicel-onlv for Allogeneic Transplantation (allo-HCT) in Patients with Hematologic Malignancies: Results of a Multicenter Open-Label Expanded Access Program (EAP)Abstract Number: 313Lead Author: Mitchell E. Horwitz, MD, Stem Cell Transplant Specialist and Professor of Medicine at Duke Cancer InstituteTime: February 22, 6:45-7:45 p.m. CT Presentation highlights: In this expanded access program (EAP) evaluating outcomes in 29 patients with hematologic malignancies following allogeneic hematopoietic stem cell transplant with omidubicel, outcomes were overall consistent with those from omidubicel’s Phase 3 study. Eligible patients ≥12 years of age received myeloablative conditioning, prophylactic medications and supportive care per individual institutional standards. Median time to neutrophil and platelet engraftment were 12 and 34 days, respectively. Results were also similar to the Phase 3 study for infection (first grade 2-3 bacterial / invasive fungal infections at 100 days posttransplant: 18%), graft-versus-host-disease (grades 3-4: 19%), disease-free survival (79%) and overall survival (87%). Demographics of transplanted patients were 55% White, 21% Asian, 17% Black, and 7% other, consistent with the Phase 3 trial, in which >40% of the study participants were racially or ethnically diverse. Omidubicel was approved under the brand name Omisirge® (omidubicel-onlv) by the U.S. FDA in April 2023 for allogeneic stem cell transplant. Title: A Phase I/II Study of GDA-201, Cryopreserved Nicotinamide-Enhanced Allogeneic Natural Killer Cells, in Patients with Relapsed/Refractory B-cell LymphomaAbstract Number: 255Lead Author: Brian C. Shaffer, Associate Professor of Medicine and Head of the Adult Mismatched Donor Sub-Program at Memorial Sloan Kettering Cancer Center in New YorkDate and Time: February 22, 6:45-7:45 p.m. CT Presentation highlights: In this ongoing multicenter Phase 1 study of allogeneic cryopreserved NK cell therapy candidate GDA-201 in patients with relapsed/refractory B-cell CD20 positive non-Hodgkin lymphoma, preliminary data for the first 12 patients were presented. Patients were heavily pretreated with a median of six prior lines of therapy including CAR-T cell therapy and hematopoietic stem cell transplant. Patients were treated with doses up to 2x108 cells/kg GDA-201 in combination with rituximab. There were no infusion reactions, dose-limiting toxicities or related serious adverse events. Seven patients exhibited a decrease in tumor burden. Efficacy evaluation using Lugano criteria showed three patients with complete response, two with partial response and two with stable disease. Cytokine release syndrome was reported in two patients (grade 1 and grade 2, respectively). There were no cases of immune effector cell associated neurotoxicity syndrome or graft versus host disease reported. Treatment and evaluation of patients in the fourth cohort of the study at the highest dose level of 2x108 cells/kg is ongoing. Full results are expected in Q1 2024. Posters are available at: https://www.gamida-cell.com/our-rd/ About Gamida CellGamida Cell is a cell therapy pioneer working to turn cells into powerful therapeutics. The company’s proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. These include Omisirge® (omidubicel-onlv), an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy, and GDA-201, an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram. About GDA-201GDA-201 is an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. NAM-modified NK cells have previously been shown to have enhanced metabolic fitness, resistance to oxidative stress and potent cytotoxicity. A multicenter Phase 1 study of GDA-201 for the treatment of non-Hodgkin lymphoma is ongoing (NCT05296525). Results are expected in Q1 2024. GDA-201 is an investigational cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. Omisirge® (omidubicel-onlv) IndicationOmisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection. Important Safety Information for Omisirge BOXED WARNING: INFUSION REACTIONS, GRAFT VERSUS HOST DISEASE, ENGRAFTMENT SYNDROME, AND GRAFT FAILURE Infusion reactions may be fatal. Monitor patients during infusion and discontinue for severe reactions. Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin or bovine material.Graft-versus-Host Disease may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD.Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids.Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery. ContraindicationsOMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine products. Warnings and PrecautionsHypersensitivity ReactionsAllergic reactions may occur with the infusion of OMISIRGE. Reactions include bronchospasm, wheezing, angioedema, pruritis and hives. Serious hypersensitivity reactions, including anaphylaxis, may be due to DMSO, residual gentamicin, Dextran 40, human serum albumin (HSA) and bovine material in OMISIRGE. OMISIRGE may contain residual antibiotics if the cord blood donor was exposed to antibiotics in utero. Patients with a history of allergic reactions to antibiotics should be monitored for allergic reactions following OMISIRGE administration. Infusion ReactionsInfusion reactions occurred following OMISIRGE infusion, including hypertension, mucosal inflammation, dysphagia, dyspnea, vomiting, and gastrointestinal toxicity. Premedication with antipyretics, histamine antagonists, and corticosteroids may reduce the incidence and intensity of infusion reactions. In patients transplanted with OMISIRGE in clinical trials, 47% (55/117) patients had an infusion reaction of any severity. Grade 3-4 infusion reactions were reported in 15% (18/117) patients. Infusion reactions may begin within minutes of the start of infusion of OMISIRGE, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of infusion reactions during and after OMISIRGE administration. When a reaction occurs, pause the infusion and institute supportive care as needed. Graft-versus-Host DiseaseAcute and chronic GvHD, including life-threatening and fatal cases, occurred following treatment with OMISIRGE. In patients transplanted with OMISIRGE Grade II-IV acute GvHD was reported in 58% (68/117). Grade III- IV acute GvHD was reported in 17% (20/117). Chronic GvHD occurred in 35% (41/117) of patients. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, be monitored for signs and symptoms of GvHD, and treated if GvHD develops. Engraftment SyndromeEngraftment syndrome may occur because OMISIRGE is derived from umbilical cord blood. Monitor patients for unexplained fever, rash, hypoxemia, weight gain, and pulmonary infiltrates in the peri-engraftment period. Treat with corticosteroids as soon as engraftment syndrome is recognized to ameliorate symptoms. If untreated, engraftment syndrome may progress to multiorgan failure and death. Graft FailurePrimary graft failure occurred in 3% (4/117) of patients in OMISIRGE clinical trials. Primary graft failure, which may be fatal, is defined as failure to achieve an absolute neutrophil count greater than 500 per microliter blood by Day 42 after transplantation. Immunologic rejection is the primary cause of graft failure. Monitor patients for laboratory evidence of hematopoietic recovery. Malignancies of Donor OriginTwo patients treated with OMISIRGE developed post-transplant lymphoproliferative disorder (PTLD) in the second-year post-transplant. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. The etiology is thought to be donor lymphoid cells transformed by Epstein-Barr virus (EBV). Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. One patient treated with OMISIRGE developed a donor-cell derived myelodysplastic syndrome (MDS) during the fourth-year post-transplant. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. If a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478. Transmission of Serious InfectionsTransmission of infectious disease may occur because OMISIRGE is derived from umbilical cord blood. Disease may be caused by known or unknown infectious agents. Donors are screened for increased risk of infection, clinical evidence of sepsis, and communicable disease risks associated with xenotransplantation. Maternal and infant donor blood is tested for evidence of donor infection. See full Prescribing Information, Warnings and Precautions, Transmission of Serious Infections for list of testing performed. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. Product manufacturing includes bovine-derived reagents. All animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use. These measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Test results may be found on the container label and/or in accompanying records. If final sterility results are not available at the time of use, Quality Assurance will communicate any positive results from sterility testing to the physician. Report the occurrence of transmitted infection to Gamida Cell at (844) 477-7478. Transmission of Rare Genetic DiseasesOMISIRGE may transmit rare genetic diseases involving the hematopoietic system because it is derived from umbilical cord blood. Cord blood donors have been screened to exclude donors with sickle cell anemia, and anemias due to abnormalities in hemoglobins C, D, and E. Because of the age of the donor at the time cord blood collection takes place, the ability to exclude rare genetic diseases is severely limited. ADVERSE REACTIONSThe most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion reaction. Please see full Prescribing Information, including Boxed Warning. About Gamida CellGamida Cell is a cell therapy pioneer working to turn cells into powerful therapeutics. The company’s proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. These include Omisirge® (omidubicel-onlv), an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy, and GDA-201, an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Facebook, X and Instagram. Cautionary Note Regarding Forward-Looking StatementsThis press release may contain forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the potentially life-saving or curative therapeutic and commercial potential of Omisirge® (omidubicel-onlv), and the Company’s cell therapy candidate, GDA-201. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions including those related to clinical, scientific, regulatory and technical developments and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 14, 2023, and other filings that Gamida Cell makes with the SEC from time to time (which are available at www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements. Omisirge® is a registered mark of Gamida Cell Inc. © 2024 Gamida Cell Inc. All Rights Reserved. Media Contact:  Dan Boyle  Orangefiery  media@orangefiery.com1-818-209-1692  Investor Contact:  Chuck Padala  LifeSci Advisors  Chuck@lifesciadvisors.com1-646-627-8390

Phase 2 data published in NEJM show potential of frexalimab as high-efficacy therapy in relapsing MS High-dose frexalimab significantly reduced disease activity, demonstrating 89% reduction in new brain lesions Phase 3 trials in relapsing MS and non-relapsing secondary progressive MS currently underway Paris, February 15, 2024.The New England Journal of Medicine published results from a positive Phase 2 clinical trial demonstrating frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis (MS), corresponding to 89% and 79% reduction in new gadolinium-enhancing (GdE) T1 brain lesions at Week 12 in the high- and low-dose treatment arms compared to placebo, meeting the study’s primary endpoint. Findings also showed both doses of frexalimab provided significant reduction in new or enlarging T2 lesions, a secondary endpoint of the study. Frexalimab is Sanofi’s novel second-generation investigational anti-CD40L antibody that has a unique method of action with the potential to address both acute and chronic neuroinflammation in MS without causing lymphocyte depletion. These data were previously presented at Consortium of Multiple Sclerosis Centers annual meeting 2023. Patrick Vermersch, MD, PhD University of Lille, CHU Lille, France “These published Phase 2 results for frexalimab represent important data in not only the potential treatment of MS but to the broader MS community. Of note, at Week 12, both doses of frexalimab provided reduction of new lesions – a standard measure of active inflammation in MS – and was well-sustained over time and well tolerated, especially at the high dose of frexalimab where 96% of patients were free of new active lesions after 24 weeks of treatment.” Results published in NEJM stem from the Phase 2 clinical trial that randomized 129 adults with relapsing MS to receive one of two doses of the anti-CD40L antibody frexalimab (n=52 and n=51, in the high- and low-dose treatment arms, respectively) or matching placebo (n=12 and n=14, respectively; pooled for efficacy analyses). In the high-dose treatment arm, participants received 1200 mg of frexalimab intravenously every 4 weeks with an 1800 mg loading dose. In the low-dose treatment arm, participants received 300 mg of frexalimab subcutaneously every 2 weeks with a 600 mg loading dose. After 12 weeks of treatment, both doses of frexalimab led to significant reductions in: The number of new GdE T1-lesions at Week 12, providing rate ratios of 0.11 (95% CI, 0.03 to 0.38) and 0.21 (95% CI, 0.08 to 0.56), corresponding to 89% and 79% reduction in the high- and low-dose treatment arms versus placebo, the primary endpoint. The number of new/enlarging T2-lesions at Week 12, providing rate ratios of 0.08 (95% CI, 0.03 to 0.26) and 0.14 (95% CI, 0.05 to 0.41), corresponding to 92% and 86% reduction in the high- and low-dose treatment arms versus placebo, respectively, a secondary endpoint. The total number of GdE T1-lesions at Week 12 providing rate ratios of 0.12 (95% CI, 0.04 to 0.36) and 0.20 (95% CI, 0.07 to 0.53) corresponding to 88% and 80% reduction, respectively, another secondary endpoint. The effects on the primary endpoint were sustained over time across both treatment arms, with even greater reduction seen in the high-dose frexalimab treatment arm, as 96% of these study participants were free of new GdE T1-lesions at Week 24. Exploratory endpoints looked at changes in the Multiple Sclerosis Impact Scale 29 (MSIS-29), a patient-reported outcome, plasma neurofilament light chain (NfL), which has been identified as a biomarker of neuroaxonal damage and MS disease activity, as well as plasma levels of CXCL13, a biomarker of inflammatory activity. Over 12 weeks of treatment, patient-reported outcome MSIS-29 physical impact scores improved significantly in participants receiving the higher dose of frexalimab. The least-square [LS] mean difference (95% confidence interval) was -7.9 (-14.7,-1.2), compared to pooled placebo. Both doses of frexalimab achieved a reduction in NfL levels relative to baseline (24% and 18% in the high- and low-dose treatment arms, respectively) and in CXCL13 levels relative to baseline (21% and 30% in the high- and low-dose treatment arms, respectively) compared to pooled placebo at Week 12. Frexalimab was well-tolerated, and 125 (97%) participants completed Part A and continued to the open-label Part B. The most common adverse events (≥5%) in any frexalimab-treated group were COVID-19 (n=5 [9.8%] in the lower-dose group; all uncomplicated cases of mild or moderate intensity) and headache (n=1 [2.0%] and n=3 [5.8%] in the low- and high-dose group, respectively). Sanofi has initiated Phase 3 clinical trials of frexalimab in relapsing MS and non-relapsing secondary progressive MS. About the Phase 2 trial The Phase 2 trial was a randomized, double-blind, placebo-controlled trial evaluating frexalimab in participants with relapsing MS. Participants were randomized (4:4:1:1) to receive either high or low doses of frexalimab or matching placebo for 12 weeks (Part A). The primary endpoint was the reduction in the number of new GdE T1 MRI brain lesions at Week 12. Secondary endpoints included additional MRI-based efficacy measures as well as the safety, tolerability and pharmacokinetics of frexalimab. After Week 12, participants receiving placebo switched to respective frexalimab arms and entered the open-label Part B, which is currently ongoing. About frexalimab Frexalimab (SAR441344) is a potentially best-in-disease second generation investigational anti-CD40L antibody that blocks the costimulatory CD40/CD40L pathway which is important for activation and function of adaptive (T and B cells) and innate (macrophages/microglia and dendritic cells) immunity. Through this unique upstream mechanism of action, frexalimab has the potential to address both acute and chronic neuroinflammation in MS, without causing lymphocyte depletion. Sanofi is developing frexalimab under an exclusive license from ImmuNext Inc. Frexalimab is being evaluated in Phase 3 clinical trials for Multiple Sclerosis and Phase 2 clinical trials for Sjogren’s Syndrome, Systemic Lupus Erythematosus, and Type 1 Diabetes, and its safety and efficacy have not been reviewed by any regulatory authority. For more information on frexalimab clinical trials, please visit About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media Relations Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com victor.rouault@sanofi.com Sally Bain | + 1 617 834 6026 | sally.bain@sanofi.com Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com Investor Relations Eva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.com Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com Tarik Elgoutni| + 1 617 710 3587 | tarik.elgoutni@sanofi.com Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com Sanofi Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2022. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Attachment Press_Release