FOXP3

LAPIX Therapeutics, Inc. (“LAPIX”), a biopharmaceutical company focused on developing novel, orally bioavailable immune system restoration therapies for autoimmune diseases, today announced that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial for its first-in-class, immune tolerance restoration small molecule, LPX-TI641, for the treatment of multiple sclerosis (MS). The clinical trial is expected to begin dosing in 4Q 2023. “We believe in empowering and restoring the immune system to fight disease and the FDA clearance of our IND application for LPX-TI641 represents momentum in our technology and scientific approach,” said Anas M. Fathallah, Ph.D., Chief Executive Officer and co-founder of LAPIX. “We are excited to begin our first-in-human clinical study evaluating LPX-TI641 and its potential to be a first of its kind non-immune suppressive therapeutic option for autoimmune diseases.” The Phase 1 trial is designed to evaluate the safety, tolerability, and pharmacokinetics of LPX-TI641 in healthy subjects after a single ascending dose. Data from the Phase 1 study along with LPX-TI641’s antigen-agnostic versatility could allow for the rapid pivot to other autoimmune indications such as rheumatoid arthritis. “We aspire to transform how the medical field treats patients diagnosed with serious and complex immune diseases and we are deeply committed to helping as many people as possible,” added Dr. Fathallah. “Our antigen-agnostic immune tolerance restoration approach centered on developing Tim (T-cell immunoglobulin and mucin domain) agonists, along with our in-depth knowledge of immunology and immune tolerance are the keys to unlocking transformative therapies for patients with additional consideration for their quality of life.” About LPX-TI641 LPX-TI641 is an investigational, novel, and proprietary orally bioavailable small molecule T cell immunoglobulin and mucin domain-containing protein (TIM) 3/4 receptor agonist. The TIM family of receptors play an important role in autoimmunity. LPX-TI641’s primary pharmacology is the upregulation of Foxp3+/DC4+ T-cells (T-regs) and of Tim1+/CD25+/CD19+ (B-regs) and inhibition/downregulation of Th17. The combined effects of LPX-TI641’s pharmacology is to allow the adaptive immune system to re-establish self-tolerance (T-reg/Th17 balance and B-regs) without affecting the innate immune system. The efficacy of LPX-TI641 versus standards of care has been evaluated in several animal models of MS and those models have shown favorable efficacy of LPX-TI641 in treatment escalation and treatment induction/maintenance paradigms mimicking current clinical management paradigms of MS without inducing neutropenia or lymphocytopenia. It is currently under development for neuro-autoimmune indications such as MS, with the intent to expand into rheumatoid arthritis amongst other autoimmune conditions. About LAPIX Therapeutics LAPIX Therapeutics Inc. is a Boston, MA-based company in the biopharma sector, focused on developing novel, orally bioavailable immune system restoration therapies, including immune tolerance restoration therapy for autoimmune diseases, immune tolerance induction therapies for gene therapy, and immune function restoration therapies for oncology. To learn more, visit www.lapix.com or follow us on LinkedIn and Twitter. The content above comes from the network. if any infringement, please contact us to modify.

STOCKHOLM, July 13, 2023 /PRNewswire/ -- Calliditas Therapeutics AB(NASDAQ: CALT) (NASDAQ Stockholm: CALTX) ("Calliditas") today announced interim data from the proof-of-concept Phase 2 trial in patients with squamous cell carcinoma of the head and neck (SCCHN) with its lead NOX 1 and 4 inhibitor product candidate, setanaxib. The analysis reflects encouraging early clinical progression-free survival (PFS) results and is supportive of the presumed anti fibrotic mode of action of setanaxib. The basis for the analysis consisted of a data set of 20 patients with recurrent or metastatic SCCHN, out of which 16 patients had evaluable tumor size and PFS related results. Twelve (12) patients had tumor biopsies before and after treatment that were evaluable for the biomarker analysis, which included transcriptomic analysis and also evaluated pathology markers such as SMA, Foxp3 regulatory T cells and PDL-1 CPS. Due to the small sample size and heterogeneity of the patient population, any inferences from the interim analysis should be treated with caution. The transcriptomic analysis showed that the two top pathways impacted by the treatment were fibrosis-related signaling pathways (the Idiopathic Pulmonary Fibrosis Signaling Pathway and Hepatic Fibrosis/Hepatic Stellate Cell Activation Pathway), providing support for the presumed mode of action relating to modulation of activated (myofibroblastic) fibroblasts, as well as the ongoing clinical programs. Pathology analysis showed preliminary evidence of an increase in immunological activity within tumors of patients treated with setanaxib, with favorable changes in Foxp3and PDL-1 CPS. As SMA levels at baseline were not balanced between the groups, and tumor biopsy samples were generally small, it was not possible to draw any conclusions regarding setanaxib's impact on SMA reduction. In terms of PFS, 7 out of the 16 evaluable patients were progression-free with either stable disease or partial response, out of which 6 were in the setanaxib arm and 1 was in the placebo arm. 6 of the 7 patients were still on the study drug at the time of the data read out with the longest period on drug being reported as 21 weeks, related to a patient in the setanaxib arm. "Based on the encouraging clinical and transcriptomic results, data clearly support the continuation of the trial, which will read out on tumor size and progression free survival in the full trial population next year. Also, it is interesting that the transcriptomic results clearly pointed to beneficial impact on 2 fibrosis-related signaling pathways, supporting the presumed mode of action as well as our pipeline programs. We are excited about the potential of setanaxib in disease areas where today treatment options are limited" said CEO Renée Aguiar-Lucander. "We are pleased with these encouraging interim data in a patient population where additional effective treatments are needed, and look forward to completing the study in collaboration with our excellent sites and investigators" said CMO Richard Philipson. The trial is a randomized, placebo-controlled, double-blind, proof-of-concept Phase 2 study investigating the effect of setanaxib 800 mg twice daily in conjunction with pembrolizumab 200mg IV, administered every 3 weeks (an accepted standard treatment regimen for SCCHN), in at least 50 patients with moderate or high CAF-density tumors. A tumor biopsy is taken prior to randomization and then again after at least 9 weeks of treatment. Treatment will continue until unacceptable toxicity or tumor progression, as is typical for oncology trials. The study is expected to read out final data in 2024. For further information, please contact: Åsa Hillsten, Head of IR, Calliditas Therapeutics Tel.: +46 764 03 35 43, Email: ir@calliditas.com About Calliditas Calliditas Therapeutics is a commercial stage biopharma company based in Stockholm, Sweden focused on identifying, developing and commercializing novel treatments in orphan indications, with an initial focus on renal and hepatic diseases with significant unmet medical needs. Calliditas' lead product, developed under the name Nefecon, has been granted accelerated approval by the FDA under the trade name TARPEYO® and conditional marketing authorization by the European Commission under the trade name Kinpeygo®. Kinpeygo is being commercialized in the European Union Member States by Calliditas' partner, STADA Arzneimittel AG. Additionally, Calliditas is conducting a Phase 2b/3 clinical trial in primary biliary cholangitis and a Phase 2 proof-of-concept trial in head and neck cancer with its NOX inhibitor product candidate, setanaxib. Calliditas' common shares are listed on Nasdaq Stockholm (ticker: CALTX) and its American Depositary Shares are listed on the Nasdaq Global Select Market (ticker: CALT). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Calliditas' strategy, commercialization efforts, business plans, regulatory submissions, clinical development plans, revenue and product sales projections or forecasts and focus, and the prospects for setanaxib as a treatment for SCCHN. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties, and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, any related to Calliditas' business, operations, continued and additional regulatory approvals for TARPEYO and Kinpeygo, market acceptance of TARPEYO and Kinpeygo, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, revenue and product sales projections or forecasts and other risks identified in the section entitled "Risk Factors" in Calliditas' reports filed with the Securities and Exchange Commission. Calliditas cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Calliditas disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent Calliditas' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The following files are available for download: View original content: SOURCE Calliditas Therapeutics Company Codes: Bloomberg:CALT@US, Bloomberg:CALTX@SS, ISIN:SE0010441584, NASDAQ-NMS:CALT, RICS:CALT.A, RICS:CALTX.ST, Stockholm:CALTX

BOSTON, June 14, 2023 (GLOBE NEWSWIRE) -- Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body, announced today that it will deliver an oral presentation on its Immuno-STAT™ (Selective Targeting and Alteration of T cells) platform and biologics as well as a poster on the company’s bispecific protein, CUE-401, for the treatment of autoimmune and inflammatory diseases at the Federation of Clinical Immunology Societies (FOCIS) 2023 Annual Meeting, being held June 20-23, 2023 in Boston, Massachusetts. Presentation Details Oral Presentation Session: Immuno-oncology and Cell TherapyTitle: Immuno-STAT Platform: TCR-selective Engagers for Selective Targeting of IL-2 to Tumor-Specific T CellsPresenter: Steven Quayle, Ph.D., vice president and head of Research Biology & Translational Medicine, Cue BiopharmaDate and Time: Wednesday, June 21, 2023, 4:45 p.m. – 5:00 p.m. EDT Dr. Quayle will describe the company’s CUE-100 series of Immuno-STAT biologics, designed to enable selective targeting of the immunostimulant cytokine interleukin-2 (IL-2) to tumor specific T cells for enhanced efficacy and safety profiles. Proof of concept for this platform has been achieved with clinical data from CUE-101, the company’s lead candidate. The data has demonstrated anti-cancer efficacy with a favorable tolerability profile and supports potential registrational paths for both CUE-101 as a monotherapy in third line (3L) recurrent/metastatic (R/M) human papillomavirus (HPV)16+ head and neck squamous cell carcinoma (HNSCC), and CUE-101 in combination with KEYTRUDA® (pembrolizumab) in first line (1L) R/M HPV16+ HNSCC. Additionally, Dr. Quayle will discuss the modularity of the Immuno-STAT platform, which has enabled rapid generation of additional Immuno-STAT candidates targeting other tumor antigens, such as mutated KRAS or Wilms’ Tumor 1 (WT1). This includes Cue Biopharma’s second CUE-100 series candidate, CUE-102, that is being evaluated in a Phase 1 trial for the treatment of WT1 positive malignancies. Taken together, the clinical de-risking achieved with CUE-101 supports broad applications of the Immuno-STAT platform to target diverse cancers. Poster Presentation Session: Exhibit & Poster Opening ReceptionTitle: CUE-401: A Novel IL-2/TGF-beta Fusion Protein for the Induction of CD4+ FOXP3+ Regulatory T CellsPresenter: Rich DiPaolo, Ph.D., Professor and Chair, Saint Louis University, High Ridge, Missouri, U.S.Date and Time: Tuesday, June 20, 2023, 6:00 p.m. – 7:45 p.m. EDT Cue Biopharma’s collaborator, Dr. DiPaolo, will discuss in vitro and in vivo data demonstrating the potential of CUE-401, the company’s novel bispecific protein designed to induce and expand regulatory T cells (Tregs) for the treatment of T-cell mediated autoimmune and inflammatory diseases. The ability of CUE-401 to effectively induce de novo FOXP3-expressing iTregs from both mouse and human CD4+ T cells – while also expanding existing Tregs – and suppress autoimmune inflammation represents a novel therapeutic approach. About the CUE-100 SeriesThe CUE-100 series consists of Fc-fusion biologics that incorporate peptide-MHC (pMHC) molecules along with rationally engineered IL-2 molecules. This singular biologic is anticipated to selectively target, activate and expand a robust repertoire of tumor-specific T cells directly in the patient’s body. The binding affinity of IL-2 for its receptor has been deliberately attenuated to achieve preferential selective activation of tumor-specific effector T cells while reducing the potential for effects on regulatory T cells (Tregs) or broad systemic activation, potentially mitigating the dose-limiting toxicities associated with current IL-2-based therapies. About CUE-401CUE-401 is a preclinical, bispecific fusion protein designed to induce and expand regulatory T cells (Tregs) through the delivery of transforming growth factor beta (TGF-β) and interleukin 2 (IL-2) with therapeutic potential across a range of T-cell mediated autoimmune and inflammatory diseases. Cue Biopharma entered into a strategic collaboration and option agreement with Ono Pharmaceutical Co., Ltd. (“Ono”) in February 2023 to support development of CUE-401. About Cue BiopharmaCue Biopharma, a clinical-stage biopharmaceutical company, is developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body. The company’s proprietary platform, Immuno-STAT™ (Selective Targeting and Alteration of T cells) and biologics are designed to harness the body’s intrinsic immune system as T cell engagers without the need for ex vivo manipulation or broad systemic modulation. Headquartered in Boston, Massachusetts, we are led by an experienced management team and independent Board of Directors with deep expertise in immunology and immuno-oncology as well as the design and clinical development of protein biologics. For more information please visit www.cuebiopharma.com and follow us on Twitter at https://twitter.com/CueBiopharma. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, that are intended to be covered by the safe harbor created by those sections. Such forward-looking statements include, but are not limited to, those regarding: the company’s beliefs about the potential benefits of CUE-101 and the CUE 100 series; the company’s belief that the Immuno-STAT platform stimulates targeted immune modulation through the selective engagement of disease-relevant T cells; and the company’s business strategies, plans and prospects. Forward-looking statements, which are based on certain assumptions and describe the company’s future plans, strategies and expectations, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “seek,” “intend,” “plan,” “goal,” “project,” “estimate,” “anticipate,” “strategy,” “future,” “likely” or other comparable terms, although not all forward-looking statements contain these identifying words. All statements other than statements of historical facts included in this press release regarding the company’s strategies, prospects, financial condition, operations, costs, plans and objectives are forward-looking statements. Important factors that could cause the company’s actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the company’s limited operating history, limited cash and a history of losses; the company’s ability to achieve profitability; potential setbacks in the company’s research and development efforts including negative or inconclusive results from its preclinical studies, its ability to secure required U.S. Food and Drug Administration (“FDA”) or other governmental approvals for its product candidates and the breadth of any approved indication; adverse effects caused by public health pandemics, including COVID-19, including possible effects on the company’s trials; negative or inconclusive results from the company’s clinical trials or preclinical studies or serious and unexpected drug-related side effects or other safety issues experienced by participants in clinical trials; the Company’s inability to replicate in later clinical trials any positive data demonstrated in earlier clinical trials; delays and changes in regulatory requirements, policy and guidelines including potential delays in submitting required regulatory applications to the FDA; the company’s reliance on licensors, collaborators, contract research organizations, suppliers and other business partners; the company’s ability to obtain adequate financing to fund its business operations in the future; operations and clinical the company’s ability to maintain and enforce necessary patent and other intellectual property protection; competitive factors; general economic and market conditions and the other risks and uncertainties described in the Risk Factors and in Management's Discussion and Analysis of Financial Condition and Results of Operations sections of the company’s most recently filed Annual Report on Form 10-K and any subsequently filed Quarterly Report(s) on Form 10-Q. Any forward-looking statement made by the company in this press release is based only on information currently available to the company and speaks only as of the date on which it is made. The company undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Investor ContactMarie CampinellSenior Director, Corporate CommunicationsCue Biopharma, Inc.mcampinell@cuebio.com Media ContactMaya RomanchukLifeSci Communicationsmromanchuk@lifescicomms.com