A Phase I Study of the Mesothelin-Targeted Immunotoxin LMB-100 in Combination With SEL-110 in Subjects With Malignant Pleural or Peritoneal Mesothelioma

Background:
Mesothelioma is cancer of the tissue that lines some organs. A new drug, LMB-100, may bind to a protein on mesothelioma tumors and kill cancer cells. But sometimes the body makes antibodies that reduce how well LMB-100 works. Researchers want to see if adding the drug SEL-110 to LMB-100 will prevent these antibodies from forming.
Objective:
To learn how safe and tolerable LMB-100 plus SEL-110 is in people with advanced mesothelioma.
Eligibility:
Adults ages 18 and older who have pleural or peritoneal mesothelioma that has not responded to prior platinum-based therapy
Design:
Participants will be screened with
Medical history
Physical exam
Blood and urine tests
Sample of tumor tissue. This can be from a previous procedure.
Scan of the chest, abdomen, and pelvis. Participants will lie on a table in a scanner that takes pictures. A special dye may be injected in a vein.
Positron emission tomography (fludeoxyglucose positron emission tomography (FDG-PET)) scan. A sugar attached to a chemical that gives off a signal will be injected before the scan.
Heart function tests
The study will be done in 21-day cycles. Participants will get the study drugs for up to 4 cycles. They will get them through an intravenous (IV) catheter (a tube inserted in a vein, usually in the arm):
LMB-100 for about 30 minutes on day 1, day 3, and day 5 of each cycle
SEL-110 for about 1 hour on day 1 of each cycle
Participants will get standard medicines to help prevent side effects.
Participants will repeat some screening tests during each cycle and about 5 weeks after the last dose of study drug.

Start Date28 Feb 2018

Sponsor / Collaborator

National Cancer Institute

NCT02959918

/ CompletedPhase 2

An Open Label Phase II Multiple Dose Safety, Pharmacokinetic and Pharmacodynamics Study of SEL-212 Followed by Open Label Administration of SEL-037 in Subjects With Symptomatic Gout and Elevated Blood Uric Acid

This is an open label multicenter study to evaluate the safety and tolerability of multiple doses (3 monthly IV infusions) of SEL-212 ( a combination of pegsiticase (SEL-037) and SVP-rapamycin (SEL-110)) followed by multiple doses (2 monthly IV infusions) of pegsiticase (SEL-037) alone for a total of 5 treatment cycles in subjects with symptomatic gout and hyperuricemia. Additional participants will be treated with multiple doses ( 5 monthly IV infusions) of pegsiticase (SEL-037) alone.
Participants will be monitored for safety endpoints through the 5th treatment cycle plus 30 days .Pharmacokinetic samples will be drawn at pre-determined time points in addition to weekly serum uric acid levels.

Start Date01 Oct 2016

Sponsor / Collaborator

Selecta Biosciences, Inc.

NCT02648269

/ CompletedPhase 1

A Phase I Single Ascending Dose Safety, Pharmacokinetic and Pharmacodynamics Study of SEL-212 in Subjects With Elevated Blood Uric Acid

This Study will first evaluate the safety and pharmacokinetics of a single intravenous dose of SEL-110, a nanoparticle containing rapamycin, in subjects with elevated blood uric acid levels. This will be followed, in separate subjects, by evaluation of the safety, pharmacokinetics, pharmacodynamics and immunogenicity of a single intravenous dose of SEL-212, SEL-037 (pegsiticase) plus SEL-110, in subjects with elevated blood uric acid levels. Uricase is an enzyme that converts uric acid to the readily soluble allantoin that is then excreted and SEL-110 is designed to prevent unwanted anti-drug-antibodies (ADAs) from forming.

Start Date01 Dec 2015

Sponsor / Collaborator

Selecta Biosciences, Inc.

100 Clinical Results associated with SEL-110

100 Translational Medicine associated with SEL-110

100 Patents (Medical) associated with SEL-110

Literatures (Medical) associated with SEL-110

01 Dec 2024·Molecular Therapy - Methods & Clinical Development

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I

Article

Author: Rudi de Waart, Dirk ; Ronzitti, Giuseppe ; Bosma, Piter ; Guerchet, Nicolas ; Lebrun, Pierre-Romain ; Mingozzi, Federico ; Shi, Xiaoxia ; Kishimoto, Takashi Kei ; Veron, Philippe ; Bloemendaal, Lysbeth Ten ; Duijst, Suzanne ; Collaud, Fanny ; Muro, Andrés F ; Bortolussi, Giulia ; Sellier, Pauline

Crigler-Najjar syndrome is an ultra-rare monogenic recessive liver disease caused by UGT1A1 gene mutations. Complete UGT1A1 deficiency results in severe unconjugated hyperbilirubinemia in newborns that, if not treated, may lead to brain damage and death. Treatment is based on intensive phototherapy, but its efficacy decreases with age, rendering liver transplantation the only curative option. Adeno-associated virus (AAV)-mediated gene therapy has shown long-term correction in adult patients, but loss of viral DNA and therapeutic efficacy are expected in younger patients associated with liver growth. Effective vector re-administration is hindered by anti-AAV neutralizing antibodies generated during the first administration. Here, we investigated AAV vector re-administration by modulating the immune response with rapamycin-loaded nanoparticles (ImmTOR) in Gunn rats (Ugt1a ^-/- ) and Ugt1a ^-/- mice. We administered a liver-specific AAV8 vector expressing a codon-optimized hUGT1A1 cDNA (1.0E11 vg/kg) in P25-P28 mutant animals and, upon loss of efficacy after 3 to 5 weeks, a higher second dose (1.0E12 or 5.0E12 vg/kg) was given. ImmTOR co-administration reduced anti-AAV neutralizing antibodies and immunoglobulin Gs generation in male animals of both models allowing effective re-dosing, underscored by a significant and long-term decrease in plasma bilirubin, although efficacy was affected by low-titer residual anti-AAV antibodies suggesting that re-administration in patients may require combination with other methods.

01 Nov 2024·Natural Product Research

Protective effect of scorpion venom oligopeptides in human umbilical vein endothelial cells under benzo(α)pyrene exposure

Article

Author: Li, Daixu ; Chen, Ruimin ; Lu, Lin ; Zheng, Man ; Chen, Wei

Inflammation and oxidative stress play pivotal role in the process of atherosclerosis. Scorpion venom is widely used as anti-cancer agent, however, the anti-inflammatory and antioxidant activities of scorpion venom peptides (SVPs) are rarely explored. In the current study, seven novel SVPs were isolated in a protective activity tracking isolation method in a cell model of benzo(α)pyrene (BaP)-induced human umbilical vein endothelial cells (HUVECs). The current study showed that SVP-1 [Tyr-Thr-Trp-Glu-Ala] significantly attenuated BaP-induced reactive oxygen species (ROS) over-production and inflammatory cytokines (IL-6, IL-1β, TNF-α, NO and PGE2) over-expression. Furthermore, SVP-1 attenuated BaP-induced adhesion molecules over-expression and inhibited the NF-κB and AhR signalling pathways activation. Collectively, the present study, for the first time, shows that SVPs inhibit the NF-κB and AhR signalling pathways in HUVECs under BaP-exposure, which strongly suggests the therapeutic potential of SVPs against atherosclerosis.

01 Aug 2024·Archives of Biochemistry and Biophysics

Modulation of keap-1/Nrf2/HO-1 and NF-ĸb/caspase-3 signaling pathways by dihydromyricetin ameliorates sodium valproate-induced liver injury

Article

Author: Gebril, Sahar M ; Emad, Doaa ; Ali, Doaa Mohamed Elroby ; Bayoumi, Asmaa M A ; Waz, Shaimaa

Nuclear factor erythroid factor 2 (Nrf2) is the key regulatory of the antioxidant response elements. Also, Nrf2 interacts with nuclear factor kappa B (NF-ĸB) to inhibit subsequent inflammatory cascade. Activation of Nrf2 signaling ameliorates drug-induced liver injury. Sodium valproate (SVP) is an anti-epilepsy drug with a hepatotoxic adverse effect that restricts its clinical use. In this study, coadministration of Dihydromyricetin (DHM), a natural flavonoid, with SVP to rats upregulated gene expression of Nrf2 and its downstream gene, heme oxygenase 1 (HO-1), while suppressed the Nrf2 repressor, Keap-1. Additionally, DHM led to downregulation of proinflammatory factors in liver tissues, including NF-ĸB, interleukin 1 beta (IL-1β), and tumor necrosis factor alpha (TNF-α). This was accompanied by a decrease in the proapoptotic protein (cleaved caspase-3) expression level. Furthermore, biochemical and histopathological studies showed that DHM treatment improved liver function and lipid profile while decreased inflammatory cell infiltration, congestion, and hepatocellular damage. According to our knowledge, prior research has not examined the protective effect of DHM on the liver injury induced by SVP. Consequently, this study provides DHM as a promising herbal medication that, when used with SVP, can prevent its induced hepatotoxicity owing to its potential anti-oxidative, anti-inflammatory, and anti-apoptotic properties.

News (Medical) associated with SEL-110

08 Jan 2025

·www.prnewswire.com

Clarivate Identifies Eleven Potential Blockbuster and Transformative Drugs in Annual Drugs to Watch Report

Anticipated advancements in obesity, oncology, gene therapy and other areas poised to revolutionize patient care LONDON, Jan. 8, 2025 /PRNewswire/ -- Clarivate Plc (NYSE:CLVT) a leading global provider of transformative intelligence, today announced the release of the twelfth annual Drugs to Watch™ report, a trusted guide to the therapies poised to redefine the future of healthcare. This year, the highly anticipated resource highlights 11 drugs projected to achieve blockbuster status or revolutionize treatment paradigms within five years. Since its inception, Drugs to Watch has identified over 98 transformative therapies, cementing its role as an essential resource for navigating the ever-evolving pharmaceutical landscape. This year's report, powered by insights from the Clarivate Cortellis intelligence suite of products, highlights 11 therapies that have recently launched or are set to debut in 2025. These innovations, addressing critical challenges in areas such as obesity, oncology, and gene therapy, are forecast to achieve blockbuster sales by 2030 or dramatically improve patient outcomes on a global scale. The report also explores pivotal trends shaping the industry, including the surging demand for obesity treatments, the transformative potential of gene editing, and the growing impact of regulatory innovation. The report offers an in-depth analysis of the chronic disease market in Mainland China, spotlighting five therapies expected to exceed $1 billion in annual sales over the next five years or deliver transformative outcomes for patients. It also explores critical topics shaping global healthcare, including regulatory advancements, the role of radiopharmaceutical theranostics in oncology, and the growing use of real-world data (RWD) and patient-reported outcomes (PROs) to drive health equity and enhance regulatory submissions. Henry Levy, President, Life Sciences & Healthcare, Clarivate, remarked: "Innovation in the life sciences is reaching unprecedented heights, and this year's Drugs to Watch™ report once again demonstrates the industry's ability to deliver therapies that address unmet medical needs and challenge existing paradigms of care. At Clarivate, we take pride in the precision and reliability of our predictions—last year, we identified 13 molecules as Drugs to Watch, with 12 already approved and launched and one poised for launch. This track record reflects the strength of our comprehensive data and deep expertise. By continuing to provide actionable insights, we empower the sector to navigate opportunities, overcome challenges and drive progress in advancing global health." Mike Ward, Global Head of Thought Leadership, Life Sciences & Healthcare, Clarivate, stated: "2025 represents a turning point for the life sciences sector as it embraces cutting-edge technologies such as AI and machine learning to enhance drug discovery and development. This year's report captures the dynamic forces at play, including groundbreaking progress in precision oncology, the rise of radiopharmaceuticals, and the growing focus on addressing global health disparities." The 2025 Drugs to Watch™ report highlights key trends reshaping the life sciences landscape, emphasizing the transformative impact of emerging technologies and therapeutic breakthroughs. Advances in AI and machine learning are streamlining drug discovery, clinical trials, and real-world data integration, enabling precision medicine approaches. The obesity market is undergoing a revolution driven by next-generation GLP-1 therapies, while radiopharmaceutical theranostics are redefining cancer treatment with a "see it and treat it" paradigm. Gene editing technologies are unlocking new opportunities in personalized medicine, and evolving regulatory frameworks are fostering greater emphasis on patient-reported outcomes and health equity. Together, these trends showcase the sector's resilience and its ability to navigate challenges while driving innovation to improve patient care worldwide. This year's drugs to watch exemplify the fusion of innovation and dedication to advancing patient care in an increasingly complex healthcare ecosystem. The Drugs to Watch™ 2025 list include: AWIQLI® (LAI 287; insulin icodec) developed by Novo Nordisk | Type 1 and type 2 diabetes mellitus AWIQLI®, the first once-weekly, subcutaneous insulin, has launched in Australia, Canada, the EU, Mainland China, and Japan. Its weekly dosing offers a significant advantage over daily basal insulin, potentially reducing the treatment burden for patients with type 1 or type 2 diabetes (T1DM and T2DM). CagriSema (cagrilintide + semaglutide) developed by Novo Nordisk | Obesity and type 2 diabetes mellitus CagriSema, combining cagrilintide, a long-acting amylin analog, with semaglutide, promises superior efficacy over semaglutide (OZEMPIC/WEGOVY®) and tirzepatide (MOUNJARO/ZEPBOUND®) in treating obesity and type 2 diabetes. This next-generation GLP-1 therapy leverages the benefits of GLP-1s, such as enhanced insulin secretion and appetite reduction, while incorporating amylin's effects, including slowed glucose absorption and release. If approved, CagriSema will be the first fixed-dose combination of amylin and GLP-1 receptor agonists in the obesity and T2DM markets. COBENFY™ (KarXT; xanomeline-trospium) developed by Bristol Myers Squibb | Schizophrenia and psychosis related to Alzheimer's disease Amid setbacks for emerging schizophrenia treatments, the approval of COBENFY marks a transformative milestone as the first drug in over 30 years with a novel mechanism of action for treating schizophrenia. Combining xanomeline and trospium, COBENFY selectively targets M1 and M4 receptors, rather than traditional dopamine pathways, while minimizing cholinergic side effects. While further data is needed to assess its effectiveness in Alzheimer's disease-related psychosis, COBENFY shows strong commercial potential if proven effective in treating AD-related hallucinations and delusions. EBGLYSS™ (lebrikizumab) developed by Eli Lilly and Co and Almirall | Atopic dermatitis EBGLYSS™, the third biologic targeting IL-13 for atopic dermatitis, follows DUPIXENT® (dupilumab) and ADBRY®/ADTRALZA® (tralokinumab) to market. Its less frequent dosing, more selective IL-13 inhibition, and strong efficacy and safety data position it as a likely first-line treatment for moderate-to-severe atopic dermatitis when topical corticosteroids are inadequate. Fitusiran developed by Alnylam® Pharmaceuticals Inc and Sanofi | Hemophilia A and B Fitusiran, shown to be effective in phase 3 trials for both hemophilia A and B, regardless of inhibitor status, has the potential to offer a new approach to hemophilia treatment. This small interfering RNA (siRNA) therapy works by inhibiting SerpinPC1 mRNA, reducing antithrombin levels, promoting thrombin generation, and helping to rebalance hemostasis to prevent bleeds. Leveraging Alnylam® Pharmaceuticals' ESC-GalNAc conjugate technology, fitusiran could become the first antithrombin-lowering therapy based on a double-stranded RNA molecule, pending approval. GSK-3536819 (MenABCWY) developed by GSK plc | Meningococcus GSK plc's GSK-3536819 vaccine candidate, a 5-in-1, first-generation formulation, targets the five groups of Neisseria meningitidis (A, B, C, W, and Y) responsible for most invasive meningococcal disease (IMD) cases worldwide. It combines the antigenic components of GSK's licensed meningococcal vaccines, BEXSERO (MenB) and MENVEO (MenACWY), both of which have established efficacy and safety profiles. IMDELLTRA™ (tarlatamab-dlle) developed by Amgen | Small-cell lung cancer (SCLC) IMDELLTRA™ is a first-in-class immunotherapy for extensive-stage small cell lung cancer (ES-SCLC). Using Amgen's bispecific T cell engager (BiTE®) molecules, it targets CD3 on T cells and DLL3 on tumor cells, enabling T cells to attack and lyse the tumor. DLL3 is expressed on the surface of SCLC cells in more than 85% of patients but is minimally expressed on healthy cells making it an attractive target. This mechanism positions IMDELLTRA as a potential standard of care for previously treated ES-SCLC. mRESVIA (mRNA-1345) developed by Moderna Inc | RSV With its U.S. FDA approval in May 2024, mRESVIA® joined AREXVY and ABRYSVO, both featured in Drugs to Watch 2024, as respiratory syncytial virus (RSV) vaccines currently available for adults ages 60 years and older, helping further support the public health initiative to reduce the RSV-related disease burden. Even with available vaccines, RSV infections continue to be a public health concern, particularly for infants and older adults (65 years and older). SEL-212 developed by Sobi® and Cartesian Therapeutics Inc/Selecta Biosciences Inc | Gout SEL-212 is a novel, once-monthly treatment combining pegylated uricase (pegadricase; SEL-037) with ImmTOR™, an immune tolerance technology designed to inhibit the formation of anti-drug antibodies (ADAs). For this application, ImmTOR consists of SEL-110.36, an inhibitor of uricase-specific ADA. This approach may help overcome the limitations of reduced efficacy and tolerability seen with other biologic treatments, such as KRYSTEXXA® (pegloticase), in patients with chronic gout. Vepdegestrant (ARV-471) developed by Arvinas Inc and Pfizer Inc | Breast cancer A global collaboration between Arvinas Inc and Pfizer Inc, vepdegestrant may become the first PROteolysis Targeting Chimera (PROTAC®) protein degrader on the market. Designed to target and degrade the estrogen receptor (ER) protein, early studies suggest PROTAC-induced degradation is more complete than with oral selective estrogen receptor degraders (SERDs). This offers potential for overcoming endocrine resistance in breast cancer. Label expansions, including combination with IBRANCE® (palbociclib), are being explored. Zanzalintinib (XL092) developed by Exelixis Inc | Colorectal cancer, renal cell carcinoma and squamous cell carcinoma of head and neck Zanzalintinib is a third-generation oral tyrosine kinase inhibitor targeting VEGF receptors, MET, and TAM kinases involved in tumor growth and immunosuppression. Currently in phase 3 trials for non-clear-cell renal cell carcinoma (nccRCC), colorectal cancer (CRC) and squamous cell carcinoma of the head and neck (SCCHN), the company anticipates one potential zanzalintinib launch per year starting as early as 2026. Compared to CABOMETYX® (cabozantinib), zanzalintinib may offer benefits, including approval for nccRCC histology and a broader patient population. Access the Drugs to Watch 2025 report from Clarivate, here. For more Drugs to Watch updates and analyses throughout the year, visit the Drugs to Watch web page and follow Clarivate for Life Sciences & Healthcare on LinkedIn and X. Join the conversation, using #DrugstoWatch. To learn more about how Clarivate can help healthcare companies inform and shape the drug discovery, development and delivery process, visit here. Methodology for the Clarivate Drugs to Watch 2025 Report To identify this year's Drugs to Watch, Clarivate drew from the expertise of over 160 analysts covering hundreds of diseases, drugs and markets, along with 11 integrated data sets that span the R&D and commercialization lifecycle, including: Cortellis Competitive Intelligence™, Disease Landscape & Forecast, Epidemiology Intelligence, BioWorld™, Cortellis Regulatory Intelligence™, Drug Timeline & Success Rates, Cortellis Clinical Trials Intelligence™, Cortellis Deals Intelligence™, Access & Reimbursement payer studies, Clarivate Real-World Data and Analytics, Web of Science™, Derwent Innovation™, and other industry sources including biopharma company press releases, filings and peer-reviewed publications. Candidate drugs in phase 2 or phase 3 trials, at pre-registration or registration stage, or already launched in 2024 were selected for analysis, including both novel treatments and already-marketed drugs pursuing new indications that could be particularly impactful. Drugs launched prior to 2024 were excluded. The dataset was filtered for drugs that had total forecast sales of $1 billion or more by 2030. Clarivate experts and analysts evaluated each drug in its individual context, based on factors such as expected approval or launch dates, competitive landscape, regulatory status, trial results, market dynamics and other key factors, and added novel drugs that, while likely to fall short of blockbuster status, are poised to be therapeutic game-changers. Please note that Clarivate analysts generated the data shown in this report prior to December 31, 2024. The Drugs to Watch 2025 Report and the treatments referenced in this release are based on Clarivate's current expectations per existing data, but actual results derived from the drugs named in the report and here may differ significantly. Clarivate is committed to comprehensively supporting customers across the entire drug, device and medical technology lifecycles to advance human health. By combining patient journey data, therapeutic area expertise, artificial intelligence and analytics in ways that unlock hidden insights, data-driven decisions and accelerating innovation, Clarivate's end-to-end research intelligence is designed to enable customers to make informed evidence-based decisions. About Clarivate Clarivate™ is a leading global provider of transformative intelligence. We offer enriched data, insights & analytics, workflow solutions and expert services in the areas of Academia & Government, Intellectual Property and Life Sciences & Healthcare. For more information, please visit . Media Contact: Catherine Daniel Director, External Communications, Life Sciences & Healthcare [email protected] SOURCE Clarivate Plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

VaccinePhase 3Immunotherapy

02 Jul 2024

·www.prnewswire.com

Sobi initiates rolling biologics license application to FDA for SEL-212 for the potential treatment of chronic refractory gout

STOCKHOLM, July 2, 2024 /PRNewswire/ -- Sobi ® today announced the initiation of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for SEL-212. The submission is based on the results of the DISSOLVE I and II pivotal studies. SEL-212 is an innovative biologic therapy in development for the treatment of chronic refractory gout, a debilitating condition characterised by the persistent and painful buildup of urate crystals in the joints. This significant milestone follows the FDA's Fast Track designation of SEL-212 in March 2024, underscoring the urgent need for new treatment options for patients with chronic refractory gout. The FDA's Fast Track program is intended to facilitate the development and expedite the review of medicines that treat serious conditions and fill an unmet medical need. "We are very pleased to initiate the rolling submission of the BLA for SEL-212, bringing Sobi one step closer to providing a potential new, effective treatment option for patients with chronic refractory gout," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi. "The Fast Track designation confirms the previously released SEL-212 phase 3 clinical data, highlighting the significant need for innovative therapies in this space and reinforcing our commitment to transforming the lives of patients with rare diseases." SEL-212 is a novel investigational combination medicine designed to reduce serum urate (SU) levels in people with chronic refractory gout, potentially reducing harmful tissue urate deposits which can lead to gout flares and joint deformity when left untreated. Sobi licensed SEL-212 from Selecta Biosciences (now Cartesian Therapeutics) in June 2020 and is responsible for development, regulatory and commercial activities in all markets outside of China. About SEL-212 SEL-212 is a novel investigational combination medicine designed to reduce serum urate (SU) levels in people with chronic refractory gout, potentially reducing harmful tissue urate deposits which when left untreated can lead to debilitating gout flares and joint deformity. SEL-212 consists of pegadricase, Selecta's proprietary pegylated uricase, co-administered with ImmTOR™, designed to mitigate the formation of anti-drug antibodies (ADAs). ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including chronic refractory gout. About Chronic refractory gout Gout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed with gout, which is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from chronic gout refractory to conventional medicines, a painful and debilitating condition in people with SU levels above 6 mg/dL and therefore have several flares per year and can develop nodular masses of uric acid crystals known as tophi. Elevated SU levels have been associated with diseases of the heart, vascular system, metabolism, kidney and joints. About Cartesian Therapeutics Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases. The Company's lead asset, Descartes-08, is a potential first-in-class mRNA CAR-T in phase 2b clinical development for patients with generalised myasthenia gravis. Additional phase 2 studies are planned in systemic lupus erythematosus under an allowed IND, as well as basket trials in additional autoimmune indications. The Company's clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care. Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download:

Phase 2Phase 3Fast Track

21 Mar 2023

·www.fiercebiotech.com

Selecta, Sobi rout gout in pair of phase 3 trials that could challenge Horizon

A filing for approval of Selecta Biosciences and Sobi's gout drug in the U.S. is planned for the first half of next year. Selecta Biosciences and Sobi have laid down a marker in their attempt to challenge Horizon for the gout market, delivering phase 3 data that suggest their SEL-212 candidate can match the efficacy of Krystexxa despite being administered less frequently. The Selecta drug candidate, which Sobi licensed for $100 million upfront in 2020, is a formulation of the immunogenic enzyme pegadricase and ImmTOR. Administering ImmTOR, biodegradable nanoparticles encapsulating the immunomodulator rapamycin, is designed to cut the immune response to pegadricase and prevent the development of antidrug antibodies that would render the enzyme ineffective. Krystexxa comes at the problem from a different angle, coupling the uricase enzyme to polyethylene glycol to create a pegylated molecule with reduced immunogenicity. Sales of Krystexxa grew 27% last year to hit a new high of $716.2 million. Selecta and Sobi want a piece of the action. To muscle in on the market, Selecta ran a pair of phase 3 trials that compared two doses of ImmTOR, combined with a fixed dose of pegadricase, to placebo in patients with gout that was refractory to conventional therapy. None of the participants had previously received Krystexxa or other experimental or approved therapies based on uricase. In the first trial, the response rates in the low- and high-dose cohorts were 48% and 56%, respectively, compared to 4% in the placebo cohort. The difference in the second trial was smaller, with 41% and 47% response rates in the low- and high-dose cohorts playing off against 12% in the placebo group, but was still statistically significant. Both doses in both clinical trials met the primary endpoint. The trials defined response as maintaining a reduction in serum uric acid less than 6 mg/dL for at least 80% of the time during month six. The phase 3 trials of Krystexxa looked at the proportion of people who had plasma uric acid of less than 6 mg/dL for at least 80% of the time during month three and Month 6. The response rates at the commercial Krystexxa regimen in the trials were (PDF) 47% and 38%. Krystexxa is given every two weeks—a four-week regimen failed in one of the phase 3 trials—and that gives Selecta and Sobi an advantage. SEL-212 was given every four weeks in Selecta’s phase 3 program. With patients staying on Krystexxa for up to one year, halving the administration frequency could spare people from receiving six to 12 doses. One question going into the SEL-212 trial was whether the candidate could reduce gout flares, which can occur as enzyme therapies break down urate deposits. In the first three months of the Krystexxa trials, 74% of patients on the two-week regimen experienced gout flares, compared to 51% of people on placebo. The situation reversed over the next three months, with the Krystexxa rate falling to 41% as the placebo rate rose to 67%, but the data still offered a potential point of differentiation for Selecta and Sobi. The SEL-212 release states there was “one gout flare in both the high and low dose treatment group” and that “there was no difference in gout flares when both treatment groups were compared to placebo.” Selecta and Sobi are holding off on sharing a closer look at the data until an upcoming medical meeting. A filing for approval in the U.S. is planned for the first half of next year. Shares in Selecta rose 22% to $1.57 in premarket trading, while Sobi opened down slightly in trading in Sweden.

Phase 3Clinical ResultImmunotherapy

100 Deals associated with SEL-110

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Methylmalonic Acidemia	Preclinical	US	Selecta Biosciences, Inc.	02 May 2022
Gout	Discovery	US	Selecta Biosciences, Inc.	01 Dec 2015

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ACR2017	Phase 2	Gout	60	SEL-212 (0.4mg/kg pegsiticase + 0.08mg/kg SVP-R)	hipnxgeelm(houiruwqtk) = Of 7 observed serious infusion reactions, 4 were associated with patients receiving pegsiticase alone or the lowest dose of SEL-212, as expected, and two were related to dosing errors bwyraqywzi (msrjbptmlu )	Positive	07 Nov 2017
ACR2017	Phase 2	Gout	60	Pegsiticase alone (0.4mg/kg)		Positive	07 Nov 2017
FOCIS2017	Phase 1	Hyperuricemia	5	SVP-R alone	ioocfkcutr(lwqordzhhi) = pvottmsvyb eyjyckpboi (ghgpnvjmzt )	Positive	14 Jun 2017

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