Polycystic Kidney, Autosomal Recessive

Robust and sustained efficacy in both adult PKD patients up to 30 months post-RP-L301; first pediatric patient results suggest efficacy similar to adult cohort with initial greater than five-point increase in hemoglobin Sustained genetic correction observed in eight of 12 evaluable Fanconi Anemia patients, and phenotypic correction and concomitant hematologic stabilization observed in seven of 12 patients 12-42 months after RP-L102 in Phase 2 pivotal trial 100% overall survival at 12 months post-RP-L201 and favorable safety pro all LAD-I patients with 12-24 months of follow-up Robust preclinical proof of concept studies showed RP-A601 for PKP2-ACM decreased arrhythmias and increased survival CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced positive data from the Pyruvate Kinase Deficiency (PKD), Fanconi Anemia, Severe Leukocyte Adhesion Deficiency-I (LAD-I) and Danon Disease clinical programs and PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) preclinical program presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California. “I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma. “The first pediatric PKD patient in our Phase 1 trial demonstrated initial hemoglobin increase of more than five points, and additional patients in our pivotal Fanconi Anemia trial have demonstrated robust engraftment and hematologic stabilization. All patients in the LAD-I pivotal trial continue to demonstrate improvements in clinical parameters. Regarding RP-A501, as of May 2023, all six patients with ongoing follow-up in the Danon Phase 1 trial continue to demonstrate improvements or stabilization at a time when these patients would most likely experience progressive disease or death.” Dr. Schwartz continued, “In addition, after recently receiving IND clearance from the FDA, we are excited to present robust preclinical proof of concept data from our PKP2-ACM program for the first time at a scientific meeting. PKP2-ACM is a devastating disease that is a frequent cause of sudden cardiac death and represents a significant unmet need given the limited and non-curative treatment options available to patients. Our data demonstrate decreased arrhythmias, improved cardiac structure and function, and increased survival; these results, and our rapid advancement to the first clinical gene therapy trial for PKP2-ACM, showcase our proven and consistent ability to elevate gene therapy research and our dedication to bringing these therapies to patients who need them most.” Global Phase 1 Study Results of Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency (PKD) The oral presentation includes positive updated data (cut-off May 3, 2023) from two adult patients followed up to 30 months and encouraging early data from the first pediatric patient treated with RP-L301, Rocket’s ex vivo lentiviral gene therapy candidate for Pyruvate Kinase Deficiency (PKD). Robust and sustained efficacy in both adult patients up to 30 months post-infusion demonstrated by normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, and red blood cell transfusion independence. Both adult patients reported improved quality of life with documented improvements via formal quality of life assessments. The safety pro highly favorable, with no RP-L301-related serious adverse events in either of the adult patients. Previously reported transient transaminase elevation seen in both adult subjects post conditioning and infusion with no clinical stigmata of liver injury have fully resolved. Insertion site analyses in peripheral blood and bone marrow in both adult patients through 24 months post-RP-L301 demonstrated highly polyclonal patterns and there has been no evidence of insertional mutagenesis. First pediatric results suggest similar efficacy as observed in long-term efficacy data in the adult cohort. The first pediatric patient infusion of RP-L301 was well tolerated, with engraftment achieved at day +15, hospital discharge less than one month following infusion, and no RP-L301-related serious adverse events. Hemoglobin normalized six weeks post-infusion and measured 13.4 g/dL at eight weeks (from median baseline of 7.9 g/dL). There were no red blood cell transfusion requirements following engraftment. Adult and pediatric enrollment is completed in the Phase 1 study. Phase 2 pivotal trial initiation is anticipated in the fourth quarter of 2023. Lentiviral-Mediated Gene Therapy for Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical Trials The oral presentation includes positive, updated data (cut-off April 17, 2023) from the ongoing Phase 2 pivotal trial of RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for Fanconi Anemia (FA). RP-L102 conferred sustained genetic correction in eight of 12 evaluable patients and comprehensive phenotypic correction in seven of 12 evaluable patients with ≥12 months of follow up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells and hematologic stabilization. The safety pro RP-L102 remains highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, continues to be well tolerated. No signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 have been observed. Polyclonal integration patterns have been observed in each of the seven patients with phenotypic, genetic, and hematologic evidence of engraftment. Pivotal trial enrollment and treatment have been completed, and the final two patients have been treated with commercial drug product in preparation for launch. Based on the positive efficacy and safety data from the Phase 2 pivotal FA trial, Rocket anticipates filing the Biologics License Application (BLA) with the FDA in the fourth quarter of 2023. Autologous Ex Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study The poster presentation includes positive, updated top-line data (cut-off November 2, 2022) at 12 to 24 months of follow-up after RP-L201 infusion for all nine patients. RP-L201 is Rocket’s ex vivo lentiviral gene therapy candidate for severe Leukocyte Adhesion Deficiency-I (LAD-I). Observed 100% overall survival at 12 months post-infusion via Kaplan Meier estimate for all nine LAD-I patients with 12 to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety pro RP-L201 was highly favorable in all patients with no RP-L201-related serious adverse events to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and are consistent with the safety profiles of those agents and procedures. Based on the positive efficacy and safety data from the Phase 2 pivotal LAD-I trial, Rocket anticipates filing the Biologics License Application (BLA) with the FDA during this second quarter of 2023. Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous (IV) Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy The spotlight oral presentation includes positive, previously disclosed data (cut-off July 11, 2022) in patients followed for up to 36 months in the ongoing Phase 1 clinical trial of RP-A501, Rocket’s investigational AAV gene therapy for Danon Disease. The presentation includes data from patients treated with the low dose (6.7 x 1013 GC/kg) in the pediatric (n=2) and adolescent/young adult (n=3) cohorts, and the high-dose (1.1 x 1014 GC/kg) adolescent/young adult cohort (n=2). As previously disclosed, RP-A501 was associated with favorable safety at the low dose with an appropriate immunomodulatory regimen. There have been no RP-A501-related or steroid-related serious adverse events reported to date in the pediatric cohort. Efficacy results continue to demonstrate sustained improvement or stabilization in all patients with preserved left ventricular systolic function at time of treatment (n=6, across all cohorts) across key clinical, biomarker, echocardiographic, and quality of life parameters. Improvement or stabilization of disease progression in these patients treated with RP-A501, as measured by natriuretic peptides, is in direct contrast to progressive worsening observed over three to 30 months in patients in the prospective natural history study. As of the presentation, all six patients that remain in follow-up continue to show signs of improvement or stabilization; additional follow-up data to be provided at a future date. The Phase 2 pivotal trial of RP-A501 for Danon Disease remains on track for initiation during this second quarter of 2023. Preclinical Efficacy of AAVrh.74-PKP2a (RP-A601): Gene Therapy for PKP2-associated Arrhythmogenic Cardiomyopathy The late-breaking abstract presentation includes robust preclinical proof of concept for RP-A601, Rocket’s investigational AAV gene therapy for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM), a devastating inherited heart disease that can lead to life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe. The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative. Even with treatment, life-threatening arrhythmias and progression of disease still occur. Preclinical proof of concept from a translationally relevant animal model has been demonstrated following Rocket-sponsored studies with academic partners. The preclinical studies with a cardiomyocyte-specific PKP2 knockout mouse model of ACM evaluated initial proof of concept and dose-related effects of AAV vectors (RP-A601), including survival, functional and anatomic benefits. Notably, the studies evaluated the delivery of RP-A601 at seven- and 14-days following induction of PKP2 knockout and subsequent disease onset. Results demonstrated increased survival and preserved cardiac function in the PKP2 knockout mouse model following administration of RP-A601. 100% of adult PKP2 knockout mice receiving RP-A601 seven days after knockout induction demonstrated survival to the five-month duration of evaluation, compared to 100% mortality by approximately day 50 in PKP2 knockout mice receiving formulation control. PKP2 knockout mice receiving RP-A601 displayed preserved ejection fraction and right ventricular area at 28 days, sustained to five months. Fourteen days following RP-A601 administration, PKP2 knockout mice demonstrated robust survival with a similar degree of cardiac benefit through five months. RP-A601 was also associated with mitigation of isoproterenol-induced premature ventricular contractions (PVCs) and arrhythmias, which are major morbidity components of ACM. Based on robust preclinical proof of concept that has demonstrated decreased arrhythmias and increased survival and the completion of extensive IND-enabling toxicology studies, Rocket has received IND clearance from the FDA for a Phase 1 study of RP-A601 that will assess the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death. Rocket is initiating Phase 1 study start-up activities and rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic. About Pyruvate Kinase Deficiency Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red blood cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the U.S. and Europe. Children are the most commonly and severely affected subgroup of patients. Patients with PKD have a high unmet medical need, as currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload. Recently, mitapivat, an oral enzyme activator, was approved for use in adult patients, however its efficacy is limited in more severely-afflicted patients, most notably in those who are splenectomized, transfusion-dependent, or whose disease results from deleterious mutations. RP-L301 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD). About Fanconi Anemia Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations, and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a ‘gold standard’ test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘natural gene therapy’ provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells. There is a high unmet medical need for patients with FA. About Leukocyte Adhesion Deficiency-I Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I. Rocket’s LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California. About Danon Disease Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease. There is a high unmet medical need for patients with Danon Disease. About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the U.S. and Europe. Patients living with PKP2-ACM have an urgent unmet medical need, as current medical, implantable cardioverter defibrillator (ICD), and ablation therapies do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by this devastating disease. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LV) based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit . Rocket Cautionary Statement Regarding Forward-Looking Statements Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as “aim,” “anticipate,” "believe," “can,” “continue,” “design,” “estimate,” "expect," "intend," “may,” "plan," “potential,” "will give," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2022, filed February 28, 2023 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. View source version on businesswire.com: Contacts Media Kevin Giordano kgiordano@rocketpharma.com Investors Brooks Rahmer investors@rocketpharma.com Source: Rocket Pharmaceuticals, Inc. View this news release online at:

University of Maryland School of Medicine Center is One of 28 Recognized Nationally BALTIMORE, Feb. 24, 2023 /PRNewswire/ -- The PKD Foundation recently recognized the University of Maryland School of Medicine's (UMSOM) clinical center that treats polycystic kidney disease (PKD) with a highly prestigious "Center of Excellence" designation. The designation recognizes the center as a leader in providing multidisciplinary, comprehensive clinical services for families affected by autosomal dominant polycystic kidney disease (ADPKD), a hereditary condition. It is one of just 28 clinical centers in the U.S. to receive this recognition. The Center of Excellence designation means UMSOM's PKD clinical center meets certain patient-centered, specialized criteria for the care of ADPKD; this is a common, life-threatening genetic disease that leads to cysts developing on a patient's kidneys, causing them to enlarge, malfunction and eventually fail. About 600,000 people in the U.S. have ADPKD, the fourth most common cause of kidney failure in the U.S., affecting all races and ethnicities. Many of these patients eventually require dialysis and kidney transplants. "This designation shows that we are providing gold-standard care to our patients, including access to an integrated healthcare team, to ongoing research and clinical studies, and to the latest therapeutic advances," said Terry Watnick, MD, the Joan B. and John H. Sadler Professor in Nephrology, Professor of Medicine at UMSOM, and Director of the newly renamed center - newly renamed the Maryland Polycystic Kidney Disease Clinical Center of Excellence. "We also are able to offer our patients - who live locally and those who travel from other states - a network of nephrologists, specialists, and other providers to meet their comprehensive care needs." In order to achieve this designation, the Maryland Polycystic Kidney Disease Clinical Center of Excellence had to demonstrate a wide-ranging expertise of its staff, including practitioners in the areas of nephrology, radiology, nutrition, hepatology, genetics, and pain management. A "Patient Advisory Panel" also was established to ensure the clinic receives feedback from patients and collaborates with them to provide education and advocacy in the surrounding community. These elements have enabled a center to provide coordination of care for people with ADPKD and to eliminate barriers to timely care. The PKD Foundation's selection of the UMSOM clinic demonstrates for the importance for giving patients access to high-quality care in a patient-centered environment. It also underscores the emphasis placed on medical research to address this condition, which could ultimately provide better treatments, and even a potential cure, for ADPKD. "We need to address a multitude of factors, including access to an experienced multi-disciplinary team of physicians and the opportunity to participate in clinical trials, to ensure that patients with this hereditary disease get access to state-of-the-art care," said UMSOM Dean Mark T. Gladwin, MD, Vice President for Medical Affairs, University of Maryland, Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor. "Currently there is only one FDA-approved treatment for ADPKD, so pre-clinical studies focused on dissecting the pathophysiology of cyst formation and testing of new compounds targeting metabolic pathways will hopefully accelerate the development of new treatments and better diagnostic tools." For Patients: To learn more about multidisciplinary care for patients with PKD at the University of Maryland Medical Center, visit umm.edu/pkd, or contact Karleen Schuhart at (410) 706-3455. About the University of Maryland School of Medicine Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest growing, top-tier biomedical research enterprises in the world -- with 46 academic departments, centers, institutes, and programs, and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research. With an operating budget of more than $1.3 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic, and clinically based care for nearly 2 million patients each year. The School of Medicine has nearly $600 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 students, trainees, residents, and fellows. The combined School of Medicine and Medical System ("University of Maryland Medicine") has an annual budget of over $6 billion and an economic impact of nearly $20 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity (according to the Association of American Medical Colleges profile) is an innovator in translational medicine, with 606 active patents and 52 start-up companies. In the latest U.S. News & World Report ranking of the Best Medical Schools, published in 2021, the UM School of Medicine is ranked #9 among the 92 public medical schools in the U.S., and in the top 15 percent (#27) of all 192 public and private U.S. medical schools. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visit medschool.umaryland.edu Issued on behalf of Newswise, online resource for knowledge-based news at SOURCE University of Maryland School of Medicine

NEW YORK, Oct. 19, 2022 /PRNewswire/ -- According to the research report, the Non-melanoma Skin Cancer Market will witness a YOY growth of 5.41 % in 2022 at a CAGR of 5.76% during the forecast period. The report is segmented by type (BCC and SCC) and geography (North America, Europe, Asia, and Rest of World (ROW)). For more insights on YOY and CAGR, Read FREE Sample Report. Continue Reading Technavio has announced its latest market research report titled Global Non-melanoma Skin Cancer Market 2022-2026 Non-melanoma Skin Cancer Market - Major Vendors Non-melanoma Skin Cancer Market is fragmented and the vendors are deploying various organic and inorganic growth strategies to compete in the market. Vendors are being driven by the intensifying market rivalry to implement a variety of growth methods, such as promotional efforts and spending on advertising, to increase the exposure of their services. In order to remain competitive in the market, several suppliers are also implementing inorganic expansion tactics like M&As. The report analyzes the market's competitive landscape and offers information on several market vendors, including: 3M Corp. Almirall SA Amgen Inc. Bausch Health Companies Inc. Bristol Myers Squibb Co. Elekta AB Eli Lilly and Co. F. Hoffmann La Roche Ltd. GlaxoSmithKline Plc iCAD Inc. Merck and Co. Inc. Find additional highlights on the growth strategies adopted by vendors and their product offerings, Buy Sample Report . Non-melanoma Skin Cancer Market - Geographical Analysis One of the most prevalent malignancies in the US is skin cancer. It is responsible for between 35% and 45% of all malignancies in Caucasians. The most prevalent form of cancer in the US and Canada is NMSC. During the projected period, the strong prevalence of BCC is anticipated to play a significant role in the market's expansion in the area. Numerous organizations in North America, including the Skin Cancer Foundation and the American Academy of Dermatology (AAD), launch projects and campaigns to raise public awareness of the prevention and early detection of skin cancer. During the anticipated period, these variables are anticipated to fuel market expansion. Furthermore, countries such as the US, Canada, UK, Germany, Japan, and China are expected to emerge as prominent markets for Non-melanoma Skin Cancer Market during the forecast period. Download Free Sample Report. Non-melanoma Skin Cancer Market - Key Segment Analysis The BCC segment's market share rise in non-melanoma skin cancer will be high. About 80% of NMSC is made up of BCC. According to estimates, the incidence of BCC has been increasing by 5% annually in Europe and 2% annually in the US during the previous few years. Australia has the greatest proportion of BCC patients who have received a diagnosis by the age of 70 in APAC. Surgical procedures have become more common in recent years, and both the general public and doctors are becoming more aware of BCC. Despite having a low death rate, BCCs can cause significant physical damage. Non-melanoma Skin Cancer Market - Key Drivers & Challenges The NMSC coverage and support is one of the major drivers fueling growth in the non-melanoma skin cancer market. Direct medical expenses for treating NMSC include pathology fees, facility fees, diagnosis fees, treatment fees, repair fees, and other incidentals. Medicare and the majority of payers reimburse the costs of Mohs surgery, excision, and destruction as part of treatment. Compared to surgery, the majority of pharmacological techniques demand significant effort and several doctor visits. Most of the medications for NMSC are paid for by the manufacturing business in the form of patient support and discount vouchers for low-income populations. However, the market for non-melanoma skin cancer will face significant challenges due to the small number of NMSC-specific medicines in development. Get a free sample for highlights on market Drivers & Challenges affecting the Non-melanoma Skin Cancer Market. Related Reports: Polycystic Kidney Disease Drugs Market by Type and Geography - Forecast and Analysis 2022-2026: The high prevalence of chronic kidney diseases is notably driving the polycystic kidney disease drugs market growth. The report extensively covers polycystic kidney disease drugs market segmentation by type (ADPKD and ARPKD) and geography (North America, Europe, Asia, and Rest of World (ROW)). Breast Cancer Therapeutics Market by Product and Geography - 2022-2026: The rising prevalence of breast cancer is notably driving the breast cancer therapeutics market growth. The report extensively covers the breast cancer therapeutics market segmentation by product (targeted therapy, hormonal therapy, and chemotherapy) and geography (North America, Europe, Asia, and the Rest of the World (ROW)). Table of Contents 1 Executive Summary 1.1 Market Overview Exhibit 01: Executive Summary – Chart on Market Overview Exhibit 02: Executive Summary – Data Table on Market Overview Exhibit 03: Executive Summary – Chart on Global Market Characteristics Exhibit 04: Executive Summary – Chart on Market by Geography Exhibit 05: Executive Summary – Chart on Market Segmentation by Type Exhibit 06: Executive Summary – Chart on Incremental Growth Exhibit 07: Executive Summary – Data Table on Incremental Growth Exhibit 08: Executive Summary – Chart on Vendor Market Positioning 2 Market Landscape 2.1 Market ecosystem Exhibit 09: Parent market Exhibit 10: Market Characteristics 3 Market Sizing 3.1 Market definition Exhibit 11: Offerings of vendors included in the market definition 3.2 Market segment analysis Exhibit 12: Market segments 3.3 Market size 2021 3.4 Market outlook: Forecast for 2021-2026 Exhibit 13: Chart on Global - Market size and forecast 2021-2026 ($ million) Exhibit 14: Data Table on Global - Market size and forecast 2021-2026 ($ million) Exhibit 15: Chart on Global Market: Year-over-year growth 2021-2026 (%) Exhibit 16: Data Table on Global Market: Year-over-year growth 2021-2026 (%) 4 Five Forces Analysis 4.1 Five forces summary Exhibit 17: Five forces analysis - Comparison between2021 and 2026 4.2 Bargaining power of buyers Exhibit 18: Chart on Bargaining power of buyers – Impact of key factors 2021 and 2026 4.3 Bargaining power of suppliers Exhibit 19: Bargaining power of suppliers – Impact of key factors in 2021 and 2026 4.4 Threat of new entrants Exhibit 20: Threat of new entrants – Impact of key factors in 2021 and 2026 4.5 Threat of substitutes Exhibit 21: Threat of substitutes – Impact of key factors in 2021 and 2026 4.6 Threat of rivalry Exhibit 22: Threat of rivalry – Impact of key factors in 2021 and 2026 4.7 Market condition Exhibit 23: Chart on Market condition - Five forces 2021 and 2026 5 Market Segmentation by Type 5.1 Market segments Exhibit 24: Chart on Type - Market share 2021-2026 (%) Exhibit 25: Data Table on Type - Market share 2021-2026 (%) 5.2 Comparison by Type Exhibit 26: Chart on Comparison by Type Exhibit 27: Data Table on Comparison by Type 5.3 BCC - Market size and forecast 2021-2026 Exhibit 28: Chart on BCC - Market size and forecast 2021-2026 ($ million) Exhibit 29: Data Table on BCC - Market size and forecast 2021-2026 ($ million) Exhibit 30: Chart on BCC - Year-over-year growth 2021-2026 (%) Exhibit 31: Data Table on BCC - Year-over-year growth 2021-2026 (%) 5.4 SCC - Market size and forecast 2021-2026 Exhibit 32: Chart on SCC - Market size and forecast 2021-2026 ($ million) Exhibit 33: Data Table on SCC - Market size and forecast 2021-2026 ($ million) Exhibit 34: Chart on SCC - Year-over-year growth 2021-2026 (%) Exhibit 35: Data Table on SCC - Year-over-year growth 2021-2026 (%) 5.5 Market opportunity by Type Exhibit 36: Market opportunity by Type ($ million) 6 Customer Landscape 6.1 Customer landscape overview Exhibit 37: Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria 7 Geographic Landscape 7.1 Geographic segmentation Exhibit 38: Chart on Market share by geography 2021-2026 (%) Exhibit 39: Data Table on Market share by geography 2021-2026 (%) 7.2 Geographic comparison Exhibit 40: Chart on Geographic comparison Exhibit 41: Data Table on Geographic comparison 7.3 North America - Market size and forecast 2021-2026 Exhibit 42: Chart on North America - Market size and forecast 2021-2026 ($ million) Exhibit 43: Data Table on North America - Market size and forecast 2021-2026 ($ million) Exhibit 44: Chart on North America - Year-over-year growth 2021-2026 (%) Exhibit 45: Data Table on North America - Year-over-year growth 2021-2026 (%) 7.4 Europe - Market size and forecast 2021-2026 Exhibit 46: Chart on Europe - Market size and forecast 2021-2026 ($ million) Exhibit 47: Data Table on Europe - Market size and forecast 2021-2026 ($ million) Exhibit 48: Chart on Europe - Year-over-year growth 2021-2026 (%) Exhibit 49: Data Table on Europe - Year-over-year growth 2021-2026 (%) 7.5 Asia - Market size and forecast 2021-2026 Exhibit 50: Chart on Asia - Market size and forecast 2021-2026 ($ million) Exhibit 51: Data Table on Asia - Market size and forecast 2021-2026 ($ million) Exhibit 52: Chart on Asia - Year-over-year growth 2021-2026 (%) Exhibit 53: Data Table on Asia - Year-over-year growth 2021-2026 (%) 7.6 Rest of World (ROW) - Market size and forecast 2021-2026 Exhibit 54: Chart on Rest of World (ROW) - Market size and forecast 2021-2026 ($ million) Exhibit 55: Data Table on Rest of World (ROW) - Market size and forecast 2021-2026 ($ million) Exhibit 56: Chart on Rest of World (ROW) - Year-over-year growth 2021-2026 (%) Exhibit 57: Data Table on Rest of World (ROW) - Year-over-year growth 2021-2026 (%) 7.7 US - Market size and forecast 2021-2026 Exhibit 58: Chart on US - Market size and forecast 2021-2026 ($ million) Exhibit 59: Data Table on US - Market size and forecast 2021-2026 ($ million) Exhibit 60: Chart on US - Year-over-year growth 2021-2026 (%) Exhibit 61: Data Table on US - Year-over-year growth 2021-2026 (%) 7.8 Canada - Market size and forecast 2021-2026 Exhibit 62: Chart on Canada - Market size and forecast 2021-2026 ($ million) Exhibit 63: Data Table on Canada - Market size and forecast 2021-2026 ($ million) Exhibit 64: Chart on Canada - Year-over-year growth 2021-2026 (%) Exhibit 65: Data Table on Canada - Year-over-year growth 2021-2026 (%) 7.9 UK - Market size and forecast 2021-2026 Exhibit 66: Chart on UK - Market size and forecast 2021-2026 ($ million) Exhibit 67: Data Table on UK - Market size and forecast 2021-2026 ($ million) Exhibit 68: Chart on UK - Year-over-year growth 2021-2026 (%) Exhibit 69: Data Table on UK - Year-over-year growth 2021-2026 (%) 7.10 Germany - Market size and forecast 2021-2026 Exhibit 70: Chart on Germany - Market size and forecast 2021-2026 ($ million) Exhibit 71: Data Table on Germany - Market size and forecast 2021-2026 ($ million) Exhibit 72: Chart on Germany - Year-over-year growth 2021-2026 (%) Exhibit 73: Data Table on Germany - Year-over-year growth 2021-2026 (%) 7.11 Japan - Market size and forecast 2021-2026 Exhibit 74: Chart on Japan - Market size and forecast 2021-2026 ($ million) Exhibit 75: Data Table on Japan - Market size and forecast 2021-2026 ($ million) Exhibit 76: Chart on Japan - Year-over-year growth 2021-2026 (%) Exhibit 77: Data Table on Japan - Year-over-year growth 2021-2026 (%) 7.12 China - Market size and forecast 2021-2026 Exhibit 78: Chart on China - Market size and forecast 2021-2026 ($ million) Exhibit 79: Data Table on China - Market size and forecast 2021-2026 ($ million) Exhibit 80: Chart on China - Year-over-year growth 2021-2026 (%) Exhibit 81: Data Table on China - Year-over-year growth 2021-2026 (%) 7.13 Market opportunity by geography Exhibit 82: Market opportunity by geography ($ million) 8 Drivers, Challenges, and Trends 8.1 Market drivers 8.2 Market challenges 8.3 Impact of drivers and challenges Exhibit 83: Impact of drivers and challenges in 2021 and 2026 8.4 Market trends 9 Vendor Landscape 9.1 Overview 9.2 Vendor landscape Exhibit 84: Overview on Criticality of inputs and Factors of differentiation 9.3 Landscape disruption Exhibit 85: Overview on factors of disruption 9.4 Industry risks Exhibit 86: Impact of key risks on business 10 Vendor Analysis 10.1 Vendors covered Exhibit 87: Vendors covered 10.2 Market positioning of vendors Exhibit 88: Matrix on vendor position and classification 10.3 3M Corp. Exhibit 89: 3M Corp. - Overview Exhibit 90: 3M Corp. - Business segments Exhibit 91: 3M Corp. - Key news Exhibit 92: 3M Corp. - Key offerings Exhibit 93: 3M Corp. - Segment focus 10.4 Almirall SA Exhibit 94: Almirall SA - Overview Exhibit 95: Almirall SA - Product / Service Exhibit 96: Almirall SA - Key news Exhibit 97: Almirall SA - Key offerings 10.5 Bausch Health Companies Inc. Exhibit 98: Bausch Health Companies Inc. - Overview Exhibit 99: Bausch Health Companies Inc. - Business segments Exhibit 100: Bausch Health Companies Inc. - Key news Exhibit 101: Bausch Health Companies Inc. - Key offerings Exhibit 102: Bausch Health Companies Inc. - Segment focus 10.6 Bristol Myers Squibb Co. Exhibit 103: Bristol Myers Squibb Co. - Overview Exhibit 104: Bristol Myers Squibb Co. - Product / Service Exhibit 105: Bristol Myers Squibb Co. - Key offerings 10.7 Elekta AB Exhibit 106: Elekta AB - Overview Exhibit 107: Elekta AB - Business segments Exhibit 108: Elekta AB - Key news Exhibit 109: Elekta AB - Key offerings Exhibit 110: Elekta AB - Segment focus 10.8 F. Hoffmann La Roche Ltd. Exhibit 111: F. Hoffmann La Roche Ltd. - Overview Exhibit 112: F. Hoffmann La Roche Ltd. - Business segments Exhibit 113: F. Hoffmann La Roche Ltd. - Key news Exhibit 114: F. Hoffmann La Roche Ltd. - Key offerings Exhibit 115: F. Hoffmann La Roche Ltd. - Segment focus 10.9 Mylan N.V Exhibit 116: Mylan N.V - Overview Exhibit 117: Mylan N.V - Product / Service Exhibit 118: Mylan N.V - Key offerings 10.10 Sun Pharmaceutical Industries Ltd. Exhibit 119: Sun Pharmaceutical Industries Ltd. - Overview Exhibit 120: Sun Pharmaceutical Industries Ltd. - Product / Service Exhibit 121: Sun Pharmaceutical Industries Ltd. - Key offerings 10.11 Varian Medical Systems Inc. Exhibit 122: Varian Medical Systems Inc. - Overview Exhibit 123: Varian Medical Systems Inc. - Business segments Exhibit 124: Varian Medical Systems Inc. - Key offerings Exhibit 125: Varian Medical Systems Inc. - Segment focus 10.12 Vidac Pharma Exhibit 126: Vidac Pharma - Overview Exhibit 127: Vidac Pharma - Product / Service Exhibit 128: Vidac Pharma - Key offerings 11 Appendix 11.1 Scope of the report 11.2 Inclusions and exclusions checklist Exhibit 129: Inclusions checklist Exhibit 130: Exclusions checklist 11.3 Currency conversion rates for US$ Exhibit 131: Currency conversion rates for US$ 11.4 Research methodology Exhibit 132: Research methodology Exhibit 133: Validation techniques employed for market sizing Exhibit 134: Information sources 11.5 List of abbreviations Exhibit 135: List of abbreviations About Us: Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website:  SOURCE Technavio