CD23

6 November 2024 Roquefort Therapeutics plc ("Roquefort Therapeutics" or the "Company") STAT-6 siRNA Presentation at Advanced Therapy Development Congress Roquefort Therapeutics (LSE:ROQ), the Main Market listed biotech company focused on developing first in class medicines in the high value and high growth immunology and oncology markets, is pleased to announce that the latest experimental results for its STAT-6 siRNA program will be presented at the Advanced Therapy Development Congress in London today. STAT-6 siRNA Immunology / Oncology Medicine siRNA therapeutics are an innovative new class of medicines based on RNA interference (RNAi), whereby a gene is downregulated by disrupting its messaging machinery, which was recognised with the 2006 Nobel Prize in Physiology or Medicine. Roquefort Therapeutics' siRNA programs target STAT-6. Signal Transducer and Activator of Transcription (STAT) proteins are both signalling proteins and transcription factors that play a role in cell growth, differentiation and function. STAT-6 is a key nodal transcription factor that selectively mediates downstream signalling of IL-4 and IL-13, dominant and central cytokines in the pathophysiology of Type 2 inflammatory diseases[1]. As previously announced, Roquefort Therapeutic's siRNA programs have demonstrated in vivo proof of concept and efficacy in oncology. This new set of experiments investigated the use of the siRNA in the inflammation and immunology ("I&I") field. STAT-6 is a novel I&I target validated by Sanofi and Recludix Pharma through a significant ($100M) licensing transaction in 20231. Our preliminary experiments demonstrated a significant reduction in the levels of total STAT-6. Presentation of STAT6 siRNA results at the Advanced Therapy Development Congress At the Advanced Therapy Development Congress today, the Company will present the new results of the STAT-6 siRNA experiments in the I&I indication in a scientific presentation titled Development of First-In-Class Advanced Therapies For Immunology and Oncology. The results presented today show that in a validated THP-1 model of I&I disease, the STAT-6 siRNA dampened the immune response. Following IL-4 stimulation, the THP-1 cells were treated with siRNA eliciting an ~10 fold reduction in total STAT-6 with a corresponding reduction in phospho-STAT-6 and modification of the key biomarkers CCL-17 and CD-23. Roquefort Therapeutics CEO Ajan Reginald commented: "The results presented today highlight the Company's internal research and development expertise in the Immunology and Inflammation field and the potency of our siRNA program, in the highly attractive STAT-6 niche. A number of leading Biotech companies have focused their research in the STAT-6 field and so, we sought advice and designed our experiments based on feedback from our potential Pharma partners. The results demonstrate our siRNA is highly competitive in a head-to-head comparison with the leading STAT-6 companies, with potentially better absolute efficacy. These are the types of results we were seeking to validate our business model of discovering early breakthrough innovation from which we develop medicines that are highly attractive to Big Pharma. We are progressing with the Company's near-term imperative, which remains, to complete meaningful partnerships with one or more Big Pharma companies." ENDS Roquefort Therapeutics plc +44 (0)20 3918 8633 Stephen West (Chairman) / Ajan Reginald (CEO) SP Angel Corporate Finance LLP (Broker) David Hignell / Vadim Alexandre / Devik Mehta +44 (0) 20 3470 0470 Buchanan (Public Relations) Ben Romney / Jamie Hooper / George Beale +44 (0)20 7466 5000 Peak IR (Investor Relations) Seb Wykeham +33 (0)7 44 44 15 42 LEI: ‎254900P4SISIWOR9RH34 About Roquefort Therapeutics Roquefort Therapeutics (LSE:ROQ) is a Main Market listed biotech company developing first in class drugs in the high value and high growth immunology and oncology markets prior to partnering with big pharma. Roquefort Therapeutics' portfolio consists of five novel patent-protected pre-clinical anti-cancer medicines. The highly complementary profile of five best-in-class medicines consists of: · Midkine antibodies with significant in vivo efficacy and toxicology studies; · Midkine RNA therapeutics with novel anti-cancer gene editing action; · Midkine mRNA therapeutics with novel anti-cancer approach; · STAT-6 siRNA therapeutics targeting solid tumours with significant in vivo efficacy; and · MK cell therapy with direct and NK cell-mediated anti-cancer action For further information on Roquefort Therapeutics, please visit www.roquefortplc.com and @RoquefortTherap on X (formerly Twitter). [1] https://recludixpharma.com/recludix-pharma-enters-into-a-strategic-collaboration-with-sanofi-to-advance-novel-oral-stat6-inhibitor-in-multiple-immunological-and-inflammatory-indications/

SAN DIEGO, March 12, 2024 /PRNewswire/ -- Longbio Pharma (Suzhou) Co., Ltd. (referred to as "Longbio Pharma"), a leading biotech company dedicated to developing innovative protein treatments for allergy, respiratory, dermatology, hematology, ophthalmology, and other autoimmune and rare diseases, proudly announced the Phase I data of LP-003 (new generation of anti-IgE antibody) at the 2024 American Academy of Dermatology Association annual meeting (2024AAD). The presentation of the poster titled "A Phase 1 study in healthy subjects of LP-003, a novel long-acting, high affinity anti-IgE antibody" by Longbio Pharma marked a significant moment during the conference. Title: A Phase 1 study in healthy subjects of LP-003, a novel long-acting, high affinity anti-IgE antibody Abstract Submission ID: 50484 Presentation Time: Mar 8-12, 2024 Location: Poster Exhibits Center Results: In a randomized, double-blind, single-ascending-dose Phase I clinical study (CTR20221413), 32 healthy subjects were randomly divided into 5 groups to receive a single intravenous dose of 0.3 mg/kg, 1 mg/kg, 3 mg/kg, 6 mg/kg and 10 mg/kg respectively. Safety, pharmacokinetics, and pharmacodynamics (free IgE levels) profile was evaluated. LP-003 exhibited a non-linear PK characteristic with T1/2 ranging from 44.6 days to 76.5 days, which is approximately 2～3 fold of Omalizumab. Moreover, the free-IgE was suppressed to below detection range for more than 168 days at 1 mg/kg group (except one with high baseline IgE level) to 10 mg/kg group. LP-003 appears to show good safety profile. No Grade 3 and above TEAE was reported, establishing a solid foundation for further investigation and advancement. Chronic spontaneous urticaria (CSU) stands as a prevalent skin disorder affecting approximately 1–2% of the global population. Despite SoC treatment, the efficacy and duration of relief remain significant challenges for patients and healthcare providers alike. Anti-IgE therapy was approved as a second-line treatment, however, only approximately 40% of patients achieving complete response following 300 mg Omalizumab dosing. Thus, a novel anti-IgE treatments offering higher efficacy and prolonged relief is needed. The successful Phase II study of Ligelizumab points a way that anti-IgE antibody with higher IgE affinity and more potent efficacy (FcεRI inhibition) retains the potential to be superior against Omalizumab. In this study, Longbio Pharma developed a new generation of anti-IgE antibody, LP-003, of higher IgE binding affinity, stronger FcεRI inhibition bioactivity, compared to Omalizumab and even Ligelizumab. Meanwhile, LP-003 has a much longer half-life (approximately 45∽76 days) compared to Omalizumab (approximately 17∽20 days). Moreover, a Phase II clinical trial (NCT06228560) of total 200 adult refractory CSU patients, despite up-dosing anti-histamine (H1) treatment, are being enrolled into 5 distinct groups (n=40/group), including three LP-003 treatment groups, one omalizumab treatment group, to provide a head-to-head comparison, and one placebo group. The topline results are expected to be released in early 2025. About LP-003 LP-003, a novel monoclonal anti-IgE antibody was generated, humanized, and engineered by Dr. Sun, Nai-chau, who is the inventor of Omalizumab, and co-founder of Tanox. Based on the 30+ years R&D experience on IgE target, Dr Sun developed a new generation of anti-IgE antibody (LP-003). Its extensive characterization including: affinity, bioactivity (FcεRI and FcεRII/CD23 inhibition), off-target binding and animal study (Tox and efficacy) was conducted. LP-003 just accomplished its Phase II study of allergic rhinitis. The current data shows LP-003 has the potential to become a best-in-class treatment. About Longbio Pharma LongBio Pharma is a biotech company located in Shanghai/Changshu, China. The company, which was founded in 2018, focuses on autoimmune and complement diseases, serving patients and society. For more information, please visit: or please contact: [email protected]. SOURCE Longbio Pharma

STOCKHOLM, Nov. 6, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) ("Calliditas"), today announced data presentations highlighting additional analyses from the Phase 3 NefIgArd study with Nefecon in adults with primary IgA nephropathy (IgAN), as well as pre-clinical data on the treatment of Alport syndrome with setanaxib, a novel NOX inhibitor, presented at the American Society of Nephrology (ASN) Kidney Week 2023 in Philadelphia, PA on November 1-5, 2023. "The additional analyses of data from the NeflgArd Phase 3 trial that we and our scientific collaborators presented at ASN Kidney Week showcased our commitment to advancing the science in understanding IgA nephropathy and to shaping a better future for patients with rare diseases," said Richard Philipson, Chief Medical Officer at Calliditas. Oral Presentation Analyses NefIgArd Phase 3 Trial full analysis The Phase 3 double-blind, randomized NefIgArd study evaluated the impact of Nefecon, a novel targeted-release formulation of budesonide, vs placebo on eGFR in adults with IgAN. The 2-year study period consisted of nine months of treatment with Nefecon (16 mg/day) or placebo, followed by a 15-month follow-up period off the study drug. The full analysis of the 364 patients randomized 1:1 to Nefecon or placebo showed: A statistically significantly smaller proportion of Nefecon-treated patients saw a 30% eGFR reduction compared to placebo-treated patients A delayed progression to a confirmed 30% eGFR reduction was observed in the Nefecon arm compared to the placebo arm Use of rescue medication did not alter the response to Nefecon vs placebo according to a pre-defined analysis Late-Breaking Poster Presentation Analyses NefIgArd Phase 3 trial population sub-analysis The 2-year NefIgArd trial included 62 patients with IgAN from mainland China (n=32 in Nefecon arm, n=30 in the placebo arm), with similar baseline characteristics as the global study population. In these patients, Nefecon treatment for 9 months resulted in: Lower time-weighted average change in eGFR from baseline in the Nefecon arm 66% less deterioration in renal function over the 2-year study period in the Nefecon arm, as well as reduced 2-year eGFR total slope 31% greater mean reduction in urine: protein creatinine ratio at 9 months in patients treated with Nefecon vs placebo, which was sustained at 2 years A higher proportion of Nefecon-treated patients did not display microhematuria during the observational follow-up period compared to patients who received placebo Poster Presentations Analyses Additional analyses were conducted from Part A of the Phase 3 NefIgArd trial(n=160) Serum samples collected from patients enrolled in Part A of the NefIgArd study were analyzed for functional protein interaction and showed that Nefecon modulates serum biomarkers associated with proteins known to play a role in the intestinal immune network for IgA production and control of B cell activation. These data further support a disease-modifying effect of Nefecon at the site of IgA synthesis and reinforce the link between the ileal gut-associated lymphoid tissue (GALT) and the kidneys. Levels of circulating anti-gliadin IgA and anti-casein IgA were reduced in IgAN patients treated with Nefecon vs placebo in Part A of the NefIgArd study at the 3-, 6- and 9-month mark after randomization. Levels of secretory IgA and fatty acid-binding protein, a gut permeability marker, were unchanged at these same time points. Reduction in IgA antibodies directed at dietary antigens supports a local disease-modifying effect for Nefecon via targeted action in the ileal GALT rather than modulation of gut permeability and antigen exclusion. Levels of three soluble biomarkers known for modulating B cell maturation, CD23, CD27, and CD30, were reduced in response to Nefecon 16 mg/day at the 3-, 6-, and 9-month post-randomization mark when compared to placebo responses. The degree of reduction in soluble CD23, CD27, and CD30 correlated with the magnitude of B-cell activating factor (BAFF) reduction at the same three study timepoints. Reduction in CD30 levels also correlated with the magnitude of IgA/IgG immune complex reduction at the 6- and 9-month timepoints. Together, these biomarker data add to the body of evidence supporting a disease-modification effect for Nefecon, including modulation of immune complex formation. Additional analysis from the full Phase 3 NefIgArd trial Modeling analyses leveraging the two-year eGFR total slope from 352 patients enrolled in the Phase 3 trial were applied to the records of 192 real-world IgAN patients to evaluate the long-term clinical potential of Nefecon. Using published linear regression analysis, Nefecon was predicted to substantially delay progression to renal failure, with a modeled 62 % risk reduction vs placebo and a median delay to progression of 12.8 years. Setanaxib The potential of setanaxib, a novel dual NOX inhibitor to modulate renal function and fibrosis was evaluated in a mouse model of Alport syndrome, a rare genetic disease characterized by fibrosis and progressive kidney damage. Setanaxib was evaluated alone or in combination with standard of care ramipril, an ACE inhibitor. Combined daily oral administration of setanaxib 60mg/kg and ramipril 10mg/kg resulted in a statistically significant reduction of urine albumin and albumin/creatinine ratio compared to vehicle alone after a 2-week and 4-week treatment duration. Histological analysis showed that this combination treatment decreased fibrosis and glomerular sclerosis. In silico and proteomics analyses demonstrated a reduction in both glomerular basement membrane proteins and collagen proteins in mice treated with both setanaxib and ramipril All presentations are available on the Presentations and Publications page on the Calliditas' corporate website. CONTACT: For further information, please contact: Åsa Hillsten, Head of Investor Relations & Sustanaibility, Calliditas Tel.: +46 76 403 35 43, email: [email protected] The information was sent for publication, through the agency of the contact persons set out above, on November 6, 2023 at 20:30 p.m. CET. The following files are available for download: SOURCE Calliditas Therapeutics