Last update 21 Nov 2024

Ruxolitinib Phosphate

Last update 21 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Jakabi, RUX, Ruxolitinab

+ [13]

Target

JAK1 x JAK2

Mechanism

JAK1 inhibitors(Tyrosine-protein kinase JAK1 inhibitors), JAK2 inhibitors(Tyrosine-protein kinase JAK2 inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesCongenital Disorders+ [8]

Active Indication

Nonsegmental vitiligoDermatitis, AtopicThrombocythemia, Essential+ [54]

Inactive Indication

acute leukemiaAdenocarcinoma of LungAdvanced Malignant Solid Neoplasm+ [30]

Originator Organization

Eli Lilly & Co.

Active Organization

Constellation Pharmaceuticals, Inc.

Incyte Corp.

AbbVie, Inc.

+ [14]

Inactive Organization

Maruho Co., Ltd.

Novartis AG

Drug Highest PhaseApproved

First Approval Date

US (16 Nov 2011),

Myelofibrosis

RegulationAccelerated assessment (EU), Special Review Project (CN), Orphan Drug (JP), Priority Review (AU)

Structure

Molecular FormulaC17H18N6

InChIKeyHFNKQEVNSGCOJV-OAHLLOKOSA-N

CAS Registry941678-49-5

View All Structures (2)

428

Clinical Trials associated with Ruxolitinib Phosphate

NCT06619522 / Not yet recruitingPhase 2IIT

A Phase 2 Study of INCB57643 (BET Inhibitor) in Combination With Ruxolitinib in JAK Inhibitor-naïve Patients With Myelofibrosis

To assess INCB05643 + ruxolitinib JAKi-naive patients with myelofibrosis

Start Date01 Mar 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NCT06536166 / Not yet recruitingPhase 2IIT

Blocking Interferon-γ by Ruxolitinib for Treating Inclusion Body Myositis: a Phase IIb Trial

Refer to the "Detailed Description" section.

Start Date01 Jan 2025

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT06615050 / Not yet recruitingPhase 3

A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation

The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation

Start Date09 Dec 2024

Sponsor / Collaborator

Incyte Corp.

[+4]

100 Clinical Results associated with Ruxolitinib Phosphate

100 Translational Medicine associated with Ruxolitinib Phosphate

100 Patents (Medical) associated with Ruxolitinib Phosphate

2,865

Literatures (Medical) associated with Ruxolitinib Phosphate

01 Sep 2025·Neural Regeneration Research

High-dose dexamethasone regulates microglial polarization via the GR/JAK1/STAT3 signaling pathway after traumatic brain injury

Article

Author: Wu, Hongbin ; Yang, Mengshi ; Deng, Yu ; Liu, Baiyun ; Bai, Miao ; Niu, Fei ; Ge, Qianqian ; Zhuang, Yuan ; Dong, Xinlong ; Xu, Xiaojian ; Zhang, Bin ; Lu, Shenghua ; Li, Hao

JOURNAL/nrgr/04.03/01300535-202509000-00023/figure1/v/2024-11-05T132919Z/r/image-tiff Although microglial polarization and neuroinflammation are crucial cellular responses after traumatic brain injury, the fundamental regulatory and functional mechanisms remain insufficiently understood. As potent anti-inflammatory agents, the use of glucocorticoids in traumatic brain injury is still controversial, and their regulatory effects on microglial polarization are not yet known. In the present study, we sought to determine whether exacerbation of traumatic brain injury caused by high-dose dexamethasone is related to its regulatory effects on microglial polarization and its mechanisms of action. In vitro cultured BV2 cells and primary microglia and a controlled cortical impact mouse model were used to investigate the effects of dexamethasone on microglial polarization. Lipopolysaccharide, dexamethasone, RU486 (a glucocorticoid receptor antagonist), and ruxolitinib (a Janus kinase 1 antagonist) were administered. RNA-sequencing data obtained from a C57BL/6 mouse model of traumatic brain injury were used to identify potential targets of dexamethasone. The Morris water maze, quantitative reverse transcription-polymerase chain reaction, western blotting, immunofluorescence and confocal microscopy analysis, and TUNEL, Nissl, and Golgi staining were performed to investigate our hypothesis. High-throughput sequencing results showed that arginase 1, a marker of M2 microglia, was significantly downregulated in the dexamethasone group compared with the traumatic brain injury group at 3 days post–traumatic brain injury. Thus dexamethasone inhibited M1 and M2 microglia, with a more pronounced inhibitory effect on M2 microglia in vitro and in vivo. Glucocorticoid receptor plays an indispensable role in microglial polarization after dexamethasone treatment following traumatic brain injury. Additionally, glucocorticoid receptor activation increased the number of apoptotic cells and neuronal death, and also decreased the density of dendritic spines. A possible downstream receptor signaling mechanism is the GR/JAK1/STAT3 pathway. Overactivation of glucocorticoid receptor by high-dose dexamethasone reduced the expression of M2 microglia, which plays an anti-inflammatory role. In contrast, inhibiting the activation of glucocorticoid receptor reduced the number of apoptotic glia and neurons and decreased the loss of dendritic spines after traumatic brain injury. Dexamethasone may exert its neurotoxic effects by inhibiting M2 microglia through the GR/JAK1/STAT3 signaling pathway.

31 Dec 2024·Hematology

Presence of triple positive driver mutations in JAK2, CALR and MPL in primary myelofibrosis: a case report and literature review

Review

Author: Ma, Haizhen ; Zhao, Long ; Liu, Bei ; Chen, Juan ; Zhang, Hao

BACKGROUNDPrimary myelofibrosis (PMF) is the most advanced subtype among the classic Philadelphia chromosomenegative myeloproliferative neoplasms (MPNs). A majority of patients carry one of three mutually-exclusive somatic driver mutations: JAK2 (60-65%), CALR (20-25%), or MPL (5%). Co-occurrence of these mutations is rarely reported. Here we report a case with a triple positive combination of JAK2, CALR and MPL driver mutations.CASE PRESENTATIONA 69-year-old male was admitted to hospital for acute exacerbation of chronic obstructive pulmonary disease (COPD) and was found to have splenomegaly and leukocytosis. Nextgeneration revealed JAK2, CALR, MPL mutations, and additional variants in SF3B1, SRSF2, and STAG2. The patient was diagnosed with PMF and treated with ruxolitinib and COPD therapy. Due to nausea, the ruxolitinib dose was reduced. After therapy, spleen volume decreased and hematologic responses were poor. Another genetic mutation of ASXL1 was later found. After adjusting the medication and adding antiemetics, the patient's condition improved.CONCLUSIONSThe rare coexistence of JAK2, CALR, and MPL mutations challenges the assumption of their mutual exclusivity. Further study of these mutations is essential for developing better treatment strategies.

31 Dec 2024·Journal of Dermatological Treatment

Use of a topical Janus kinase inhibitor in immune checkpoint inhibitor-induced eczematous reaction: a case report

Article

Author: Powers, Camille M ; Verma, Hannah ; Gulati, Nicholas ; Orloff, Jeremy ; Lamb, Angela ; Tiersten, Amy ; Piontkowski, Austin J

In this report, we describe the case of a 28-year-old female with bilateral breast cancer in the setting of a BRCA1 mutation, who presented to dermatology with an eczematous reaction, ultimately diagnosed as a cutaneous immune-related adverse event (cirAE) secondary to an immune checkpoint inhibitor (ICI), pembrolizumab. Our case report highlights a novel therapeutic option for an eczematous cirAE: the topical JAK 1/2 inhibitor, ruxolitinib. CirAEs can occur in up to 55% of patients on ICIs, a class of medications seeing rapidly increasing use in cancer therapy, and prior research has demonstrated that ICI-induced dermatitis may involve different pathways than traditionally observed in their spontaneous counterparts. Specifically, marked Th1 skewing is noted in ICI-induced dermatitis, as opposed to a predominant Th2 response which typically characterizes spontaneous atopic dermatitis. To our knowledge, this is the first case report in the literature discussing use of a topical JAK inhibitor, ruxolitinib, in the treatment of topical steroid-refractory cirAEs. Furthermore, as topical JAK inhibitors are thought to not carry the risks of systemic JAK inhibitors, including malignancy, ruxolitinib cream is a promising therapeutic option for this challenging patient population.

380

News (Medical) associated with Ruxolitinib Phosphate

14 Nov 2024

·www.prnewswire.com

Mallinckrodt announces the 2024 Winner of the Extracorporeal Immunomodulation Award

This year's award marks 30 years since clinicians first used ECP to successfully treat chronic graft versus host disease (cGvHD).1 In recognition of this important milestone, this year's $75,000 grant was dedicated to advancing research in this important area. DUBLIN, Nov. 14, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced the winner of the Extracorporeal Immunomodulation Award (EIA). The award recognizes a project aimed at identifying the optimal timing for extracorporeal photopheresis (ECP) initiation and assessing its effectiveness when combined with other graft versus host disease (GvHD) therapies. To recognize the occasion, Mallinckrodt, which provides immunomodulatory therapy via ECP, dedicated the $75,000 grant to this research. This year's award holds special significance as it marks 30 years since clinicians first used ECP to successfully treat chronic graft versus host disease (cGvHD).1,2 The EIA-winning project was submitted by Dr Andrea Varkonyi, of the South-Pest Central Hospital, National Institute of Hematology and Infectiology's Department of Hematology and Stem Cell Transplantation, in Budapest, Hungary. Her team will carry out a retrospective and prospective study, comparing ruxolitinib alone to ruxolitinib combined with ECP therapy in the treatment of acute and chronic GvHD. They will also evaluate the impact of ECP on the development of transplant-associated thrombotic microangiopathy (TA-TMA). "Mallinckrodt is thrilled to recognize Dr. Andrea Varkonyi with this year's the Extracorporeal Immunomodulation Award and continuing the research in chronic GvHD. As the manufacturer of the THERAKOS™ CELLEX™ Photopheresis System we are committed to supporting research that seeks to improve patient outcomes and tackle unmet needs in this important area of medicine." said Peter Richardson, MRCP (UK), Executive Vice President & Chief Scientific Officer. Dr. Varkonyi said, "Acute and chronic GvHD, along with TA-TMA, remain unresolved issues following allogeneic bone marrow transplantation. Despite the intent of the procedure, these complications often prevent patients from achieving complete recovery. We are grateful to be receiving this award to advance research in this area. Thank you, Mallinckrodt, for the opportunity to further our research." The study's objective is to identify the optimal timing for introducing ECP and assess its effectiveness when combined with other therapies. It aims to address key areas of interest in allogeneic stem cell transplant-associated fundamental and clinical research. Entries to the EIA award were invited from clinicians and scientists working on translational or outcomes-based research, as well as collaborative projects, in the field of GvHD-focused ECP. Submissions were assessed on a range of criteria, including scientific merit, originality, and feasibility. The EIA award recognizes individuals and institutions whose research contributes to the continued advancement of knowledge in this area of medicine and is just one part of our ongoing commitment to the science of immunomodulation through ECP. In June, the THERAKOS™ CELLEX™ System technology secured CE certification under the European Union Medical Device Regulation (EU MDR). It involved a robust quality management system audit, technical and microbiological reviews, and a thorough clinical assessment, demonstrating the organization's ongoing commitment to ECP evidence generation, post approval. About THERAKOS™ CELLEX™ Photopheresis System The THERAKOS CELLEX Photopheresis System is the world's only fully integrated and validated ECP system.3 THERAKOS performs ECP using patented technology that collects, separates and treats a small amount of white blood cells (immune cells) while the patient is connected to the instrument. The treated cells are then returned to the patient where they help to modify the immune response in a process called immunomodulation. It is used to treat a range of immune-mediated diseases. THERAKOS Systems are used by over 300 treatment centres in over 30 countries worldwide.4 About Extracorporeal Photopheresis (ECP) Extracorporeal photopheresis (ECP) is an immunomodulatory therapy that has demonstrated efficacy in various T-cell and immune-mediated diseases.³ ECP is recommended by international and national guidelines for a spectrum of diseases, including cutaneous T-cell lymphoma (CTCL), acute and chronic graft-versus-host disease (aGvHD and cGvHD), chronic lung allograft dysfunction-bronchiolitis obliterans syndrome (CLAD-BOS) and after cardiac transplantation. 5,6,7,8,9,10,11,12,13,14,15, 16,17 EU INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR THE THERAKOS™ PHOTOPHERESIS PROCEDURE Indications The THERAKOS™ CELLEX™ Photopheresis System is indicated for patients older than 18 years of age for the administration of photopheresis in the following: Cutaneous T Cell Lymphoma (CTCL) Solid Organ Transplant Rejection (SOT) (heart, lung) The THERAKOS™ CELLEX™ Photopheresis System is indicated in patients older than 3 years of age for the management of: Acute and Chronic Graft versus Host Disease (aGvHD, cGvHD) Contraindications THERAKOS™ Photopheresis is contraindicated in: Patients possessing a specific history of a light sensitive disease Patients who cannot tolerate extracorporeal volume loss or who have white blood cell counts greater than 25,000 / mm3 Patients who have coagulation disorders or who have previously had a splenectomy Warnings and Precautions THERAKOS™ Photopheresis treatments should always be performed in locations where standard medical emergency equipment is available. Volume replacement fluids and/or volume expanders should be readily available throughout the procedure. Do not expose the device to a magnetic resonance (MR) environment. The device may present a risk of projectile injury, and thermal injury and burns may occur. The device may generate artifacts in the MR image, or may not function properly. Thromboembolic events, including pulmonary embolism and deep vein thrombosis, have been reported in the treatment of Graft versus Host Disease (GvHD). Special attention to adequate anticoagulation is advised when treating patients with GvHD. When prescribing and administering THERAKOS Photopheresis for patients receiving concomitant therapy, exercise caution when changing treatment schedules to avoid increased disease activity that may be caused by abrupt withdrawal of previous therapy. Adverse Events Hypotension may occur during any treatment involving extracorporeal circulation. Closely monitor the patient during the entire treatment for hypotension. Transient pyretic reactions, 37.7–38.9°C (100–102°F), have been observed in some patients within six to eight hours of reinfusion of the photoactivated leukocyte-enriched blood. A temporary increase in erythroderma may accompany the pyretic reaction. Treatment frequency exceeding labelling recommendations may result in anaemia. Venous access carries a small risk of infection and pain. Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator's Manual for a complete list of warnings and precautions. IMPORTANT SAFETY INFORMATION FOR METHOXSALEN USED IN CONJUNCTION WITH THERAKOS ™ PHOTOPHERESIS Consult the 8-methoxypsoralen (Methoxsalen (20 micrograms / mL)) professional leaflet or the oral 8-methoxypsoralen formulation package insert before prescribing or dispensing any medication. Warnings and Precautions Patients exhibiting multiple basal cell carcinomas or having a history of basal cell carcinoma should be diligently observed and treated. Methoxsalen may cause fetal harm when given to a pregnant woman. Women undergoing photopheresis should be advised to avoid becoming pregnant. Special care should be exercised in treating patients who are receiving concomitant therapy (either topically or systemically) with known photosensitizing agents. Oral administration of methoxsalen followed by cutaneous UVA exposure (PUVA therapy) is carcinogenic. Patients should be told emphatically to wear UVA absorbing, wrap-around sunglasses for twenty-four (24) hours after methoxsalen treatment. They should wear these glasses any time they are exposed to direct or indirect sunlight, whether they are outdoors or exposed through a window. Refer to the package insert for methoxsalen sterile solution (20 micrograms / mL) or the oral 8-methoxypsoralen dosage formulation for a list of all warnings and precautions. ABOUT MALLINCKRODT Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology, ophthalmology, and oncology; immunotherapy and neonatal respiratory critical care therapies; analgesics; and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit . CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS This release contains forward-looking statements, including with regard to Therakos, its potential to improve health and treatment outcomes, and its potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: the effects of Mallinckrodt's recent emergence from bankruptcy; satisfaction of, and compliance with, regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues or adverse side effects or adverse reactions associated with THERAKOS; and other risks identified and described in more detail in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Mallinckrodt's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law. CONTACT Media Inquiries Green Room Communications 908-577-4531 [email protected] Investor Relations Derek Belz Vice President, Investor Relations 314-654-3950 [email protected] Mallinckrodt, the "M" brand mark, and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. ©2024 Mallinckrodt. EU-2400258 10/24 References: Owsianowski M, et al. Bone Marrow Transplant. 1994;14(5), 845-848. Solh M, et al. (2023). Bone Marrow Transplant. 2023;58(2), 168-174. Knobler R, et al. J Eur Acad Dermatol Venereol. 2020;34(12):2693-2716. Data on File – Ref-07615. Mallinckrodt Pharmaceuticals. Hart JW, et al. Ther Adv Hematol. 2013;4:320-334. Knobler R, et al. J Eur Acad Dermatol Venereol. 2020;34(12):2693-2716. Trautinger F, et al. Eur J Cancer. 2017;77:57 74. Padmanabhan A, et al. J Clin Apher. 2019;34(3):171-354. Alfred A, et al. Br J Haematol. 2017;177:287-310. Cho A, et al. Front Med (Lausanne). 2018;5:236. Zeiser R. et al. Graft-versus-Host Erkrankung, akut. 2021. Available at: . Accessed August 2024. Bredeson C, et al. Curr Oncol. 2014;21(2):e310-325. Pierelli L, et al. Transfusion. 2013;53(10):2340-2352. Wolff D, et al. Biol Blood Marrow Transplant. 2011;17:1-17. Dignan FL, et al. Br J Haematol. 2012;158(1):30-45; 46-61. Knobler R, et al. J Eur Acad Dermatol Venereol. 2021;25(1):27-49. Costanzo MR, et al. J Heart Lung Transplant. 2010;29(8):914-956. Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Immunotherapy

12 Nov 2024

·www.globenewswire.com

Disc Medicine Reports Third Quarter 2024 Financial Results and Provides Business Update

Completed a successful end of Phase 2 meeting with the FDA for bitopertin in erythropoietic protoporphyria (EPP), reaching alignment on all proposed study parameters with the potential for accelerated approval based on existing dataPresented proof-of-mechanism data for Phase 1b trial of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia at the American Society of Nephrology (ASN) Kidney Week 2024Eight posters and an oral presentation across all three clinical-stage assets to be presented at the 66th American Society of Hematology (ASH) Annual Meeting and ExpositionStrong financial position ending Q3 with $487 million in cash, cash equivalents, and marketable securities, further strengthened by closing a $200 million non-dilutive debt financing in November 2024 increasing future financing optionality WATERTOWN, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today reported financial results for the third quarter ended September 30, 2024. “This is an exciting time for Disc as we have made significant progress in advancing our pipeline in recent months and remain keenly focused on execution as we prepare for multiple upcoming catalysts. We now have clarity on the path forward for bitopertin in EPP, with the potential for accelerated approval, and added to the data set supporting the potential of DISC-0974 in treating anemias of inflammation with positive SAD data in NDD-CKD,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “Additionally, with the recent completion of a debt financing with Hercules Capital, we have further solidified our strong financial position, enabling us to achieve our upcoming catalysts and to continue to work toward our goal of developing therapies with the potential to address a range of hematologic diseases.” Recent Highlights and Anticipated Milestones: Bitopertin: GlyTI Inhibitor (Heme Synthesis Modulator) Announced positive End of Phase 2 meeting with the FDA, reflecting alignment with the FDA on all proposed attributes of the APOLLO study, as well as the potential to pursue accelerated approval based on existing data using reduction of PPIX as a surrogate endpointThe Company will provide an update on discussions with the FDA around the design of a confirmatory trial in Q1 2025 and plans to initiate this trial by mid-2025.Announced four posters at ASH 2024: Additional clinical data from the AURORA and BEACON Phase 2 trialsPreclinical data on bitopertin’s effects on PPIX and phototoxicityReal-world evidence from the EPP LIGHT survey study on the burden of disease in EPP DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Presented proof of mechanism data from its ongoing Phase 1b single-ascending dose (SAD) study of DISC-0974 in non-dialysis-dependent chronic kidney disease (NDD-CKD) patients with anemia at ASN Kidney Week 2024, demonstrating that a single dose of DISC-0974 leads to consistent reductions in hepcidin and increases in transferrin saturation (TSAT), resulting in an increase in reticulocyte hemoglobin and hemoglobin Announced several data updates at ASH 2024: Oral presentation of final clinical data from the Phase 1b trial of DISC-0974 in anemia of myelofibrosis (MF)Pre-clinical data for DISC-0974 in anemia of inflammatory bowel disease (IBD)Pre-clinical data for DISC-0974 in combination with ruxolitinib The Company expects to initiate a Phase 2 study in anemia of myelofibrosis (MF) by the end of 2024 DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Announced two posters to be presented at ASH 2024: Data from the ongoing Phase 1 SAD/MAD study in healthy volunteers will be presented at ASH 2024Preclinical data for DISC-3405 in a mouse model of sickle cell disease The Company plans to initiate a Phase 2 study in polycythemia vera (PV) in 2025 Corporate: Successfully completed $200 million non-dilutive debt financing deal with Hercules Capital in November. An initial $30 million was funded at closing with an additional $80 million available to be drawn through the second half of 2026 at the Company's discretion. An additional $65 million is available subject to milestones and at the Company’s option. The financing provides minimum 48 months of interest-only.Expanded leadership team with the appointment of Steve Caffé, MD as Chief Regulatory Officer and Rahul Kaushik, Ph.D. as Chief Technical Officer Third Quarter 2024 Financial Results: Cash Position: Cash, cash equivalents and marketable securities were $487.4 million as of September 30, 2024. The Company is sufficiently financed to fund operational plans well into 2027.Research and Development Expenses: R&D expenses were $24.7 million for the quarter ended September 30, 2024, as compared to $14.4 million for the quarter ended September 30, 2023. The increase in R&D expenses were primarily driven by the progression of Disc’s portfolio, including bitopertin’s clinical studies and drug manufacturing, advancement of DISC-0974 and DISC-3405 programs deeper into development, and increased headcount.General and Administrative Expenses: G&A expenses were $8.2 million for the quarter ended September 30, 2024, as compared to $4.5 million for the quarter ended September 30, 2023. The increase in G&A expenses was primarily due to increased headcount.Net Loss: Net loss was $26.6 million for the quarter ended September 30, 2024, as compared to $14.1 million for the quarter ended September 30, 2023. About Disc Medicine Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com. Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at ir.discmedicine.com in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. Disc Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding: Disc’s anticipated use of net proceeds from the debt financing; the potential for accelerated approval and conducting a confirmatory trial for bitopertin; and future product development plans and projected timelines for the initiation and completion of preclinical and clinical trials and other activities. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the offering may not be completed on the timeline expected or at all; the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the SEC, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. DISC MEDICINE, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(In thousands, except share and per share amounts)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024202320242023Operating expenses: Research and development$24,685 $14,419 $71,874 $46,699 General and administrative 8,171 4,539 23,296 14,712 Total operating expenses 32,856 18,958 95,170 61,411 Loss from operations (32,856) (18,958) (95,170) (61,411)Other income (expense), net 6,371 4,856 15,449 8,628 Income tax expense (114) (20) (179) (67)Net loss$(26,599) $(14,122) $(79,900) $(52,850)Weighted-average common shares outstanding-basic and diluted 29,935,551 24,316,817 26,809,605 21,605,202 Net loss per share-basic and diluted$(0.89) $(0.58) $(2.98) $(2.45) DISC MEDICINE, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(In thousands)(Unaudited) September 30, December 31, 2024 2023Assets Cash, cash equivalents, and marketable securities$487,363 $360,382Other current assets 5,789 5,280Total current assets 493,152 365,662Non-current assets 1,993 2,334Total assets$495,145 $367,996Liabilities and Stockholders’ Equity Current liabilities$25,473 $21,439Non-current liabilities 1,712 1,436Total liabilities 27,185 22,875Total stockholders’ equity 467,960 345,121Total liabilities and stockholders’ equity$495,145 $367,996 Media Contact Peg RusconiDeerfield Grouppeg.rusconi@deerfieldgroup.com Investor Relations Contact Christina TartagliaPrecisionAQ Christina.tartaglia@precisionaq.com

Financial StatementPhase 1ASHPhase 2

07 Nov 2024

·www.globenewswire.com

Secura Bio Announces Poster Presentation Highlighting New COPIKTRA® (duvelisib) Data for the Treatment of Patients With Peripheral T-Cell Lymphoma and Other Advanced Hematologic Malignancies at the 2024 American Society of Hematology Meeting

Nov. 06, 2024 -- Secura Bio, Inc. (www.securabio.com), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, today announced a poster presentation highlighting new data on the investigational use of COPIKTRA® (duvelisib) both as a single agent and in combination therapy for the treatment of Peripheral T-cell Lymphoma (PTCL) and advanced hematologic malignancies at the upcoming 2024 American Society of Hematology (ASH) Annual Meeting taking place on December 7-10, 2024 in San Diego, CA. The poster, entitled: Duvelisib in Patients with Relapsed/Refractory Peripheral T-cell Lymphoma: Final Results from the Phase 2 PRIMO Trial highlights the outcomes of 123 patients in the PRIMO dose expansion phase of the PRIMO clinical trial and will be presented by Dr. Neha Mehta-Shah, Washington University School of Medicine, on Sunday, December 8, 2024, from 6:00 PM to 8:00 PM (PST) in the San Diego Convention Center, Halls G-H. The abstract is now available here. The PRIMO clinical trial was a global, multicenter, open-label, parallel cohort, Phase 2 study sponsored by Secura Bio, Inc., that evaluated COPIKTRA for the treatment of adult patients with relapsed or refractory (R/R) Peripheral T-cell Lymphoma (PTCL). Based on the dose optimization phase results, an expansion group of 123 patients was added in which COPIKTRA was dosed at 75 mg twice daily for two cycles, followed by 25 mg twice daily. COPIKTRA will also be highlighted in a number of other poster presentations evaluating combination therapy, including: Dr Alison Moskowitz, Memorial Sloan Kettering Cancer Center, will be presenting an oral presentation entitled Dual-targeted therapy with ruxolitinib plus duvelisib for T-cell lymphoma. Dr Moskowitz will be presenting on Sunday, December 8, 2024, from 9:30 AM - 11:00 AM (PST) in the San Diego Convention Center, Ballroom 20CD Dr Jennifer Crombie, Dana-Farber Cancer Institute, will be presenting a poster entitled A Phase 2 Study of Duvelisib and Venetoclax in Patients with Relapsed or Refractory (R/R) Chronic Lymphocytic Leukemia (CLL) or Richter’s Syndrome (RS) on Monday, December 9, 2024, from 6:00 PM - 8:00 PM (PST) in the San Diego Convention Center, Halls G-H Dr Hayder Saeed, Moffitt Cancer Center, will be presenting a poster entitled A Phase I Study Of Duvelisib In Combination With Oral Azacitidine (BMS-986345) In Lymphoid Malignancy on Sunday, December 8, 2024, from 6:00 PM - 8:00 PM (PST) in the San Diego Convention Center, Halls G-H Dr Josie Ford, Massachusetts General Hospital, will be presenting a poster entitled Real-World Evidence for Duvelisib and Romidepsin Combination in Patients with Relapsed/Refractory Peripheral T-Cell Lymphomas on Monday, December 9, 2024, from 6:00 PM - 8:00 PM (PST) in the San Diego Convention Center, Halls G-H Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma that develops in mature white blood cells that circulate through the bloodstream and lymphatic system. PTCL accounts for between 10-15% of all non-Hodgkin lymphomas and generally affects people aged 60 years and older. Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, typically painless lymph nodes in the neck, armpit, and/or groin. There are currently no well-established standards of care for patients with relapsed or refractory disease. COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first United States FDA approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant cells. PI3K signaling may lead to the proliferation of malignant cells and is thought to play a role in the formation and maintenance of a supportive tumor microenvironment. COPIKTRA is a kinase inhibitor indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior lines of systemic therapy. Limitations of Use: COPIKTRA is not indicated or recommended for the treatment of any patients with CLL or SLL as initial or second line treatment due to an increased risk of treatment-related mortality. COPIKTRA is also being investigated for the treatment of lymphomas, including peripheral T-cell lymphomas (PTCL). COPIKTRA has been given Fast Track status in the United States for the treatment of adult patients with PTCL who have received at least one prior therapy. Additionally, COPIKTRA has received an Orphan Drug Designation for use in the treatment of T-cell lymphomas. Treatment of T-cell lymphomas is a disease category for which COPIKTRA is not currently indicated. COPIKTRA is being investigated in combination with other agents across several types of solid and hematologic malignancies, through investigator-sponsored studies. For more information on COPIKTRA, please visit https://copiktra.com/. Information about duvelisib clinical trials can be found on https://www.clinicaltrials.gov/. Secura Bio is an integrated, commercial-stage pharmaceutical company dedicated to the worldwide development and commercialization of significant oncology therapies for physicians and their patients. Its product, COPIKTRA, is currently approved for the treatment of multiple B-cell malignancies and is also being investigated for the treatment of PTCL, for which it has received Fast Track status in the United States and is being investigated in combination with other agents through investigator-sponsored studies. For more information on Secura Bio, please visit https://www.securabio.com/. The content above comes from the network. if any infringement, please contact us to modify.

Fast TrackOrphan DrugDrug ApprovalClinical Result

100 Deals associated with Ruxolitinib Phosphate

External Link

KEGG	Wiki	ATC	Drug Bank
D09960	Ruxolitinib Phosphate	L01XE18	DB08877

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Acute Graft Versus Host Disease	NO	Novartis Europharm Ltd.	23 Aug 2012
Acute Graft Versus Host Disease	LI	Novartis Europharm Ltd.	23 Aug 2012
Acute Graft Versus Host Disease	IS	Novartis Europharm Ltd.	23 Aug 2012
Acute Graft Versus Host Disease	EU	Novartis Europharm Ltd.	23 Aug 2012
Chronic graft-versus-host disease	LI	Novartis Europharm Ltd.	23 Aug 2012
Chronic graft-versus-host disease	IS	Novartis Europharm Ltd.	23 Aug 2012
Chronic graft-versus-host disease	EU	Novartis Europharm Ltd.	23 Aug 2012
Chronic graft-versus-host disease	NO	Novartis Europharm Ltd.	23 Aug 2012
Post-polycythemia vera myelofibrosis	LI	Novartis Europharm Ltd.	23 Aug 2012
Post-polycythemia vera myelofibrosis	NO	Novartis Europharm Ltd.	23 Aug 2012
Post-polycythemia vera myelofibrosis	EU	Novartis Europharm Ltd.	23 Aug 2012
Primary Myelofibrosis	IS	Novartis Europharm Ltd.	23 Aug 2012
Primary Myelofibrosis	LI	Novartis Europharm Ltd.	23 Aug 2012
Primary Myelofibrosis	EU	Novartis Europharm Ltd.	23 Aug 2012
Primary Myelofibrosis	NO	Novartis Europharm Ltd.	23 Aug 2012
Splenomegaly	IS	Novartis Europharm Ltd.	23 Aug 2012
Splenomegaly	LI	Novartis Europharm Ltd.	23 Aug 2012
Splenomegaly	EU	Novartis Europharm Ltd.	23 Aug 2012
Splenomegaly	NO	Novartis Europharm Ltd.	23 Aug 2012
Myelofibrosis	US	Incyte Corp.	16 Nov 2011

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Nonsegmental vitiligo	NDA/BLA	CN	China Medical System Holdings Ltd.	24 Sep 2024
Myelofibrosis	NDA/BLA	CN	Novartis Pharma Stein AG	27 May 2022
Myelofibrosis	NDA/BLA	CN	Beijing Novartis Pharma Co. Ltd.	27 May 2022
Myelofibrosis	NDA/BLA	CN	Novartis Pharma Schweiz AG	27 May 2022
Post-essential thrombocythemia myelofibrosis	Phase 1	CA	Incyte Corp.	01 Aug 2009
Post-essential thrombocythemia myelofibrosis	Phase 1	US	Incyte Corp.	01 Aug 2009
Post-essential thrombocythemia myelofibrosis	Phase 1	AU	Incyte Corp.	01 Aug 2009
Post-essential thrombocythemia myelofibrosis	Discovery	AU	Incyte Corp.	01 Aug 2009
Post-essential thrombocythemia myelofibrosis	Discovery	US	Incyte Corp.	01 Aug 2009
Post-essential thrombocythemia myelofibrosis	Discovery	CA	Incyte Corp.	01 Aug 2009

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05371964 (IMproveMF, ASH2024) Manual	Phase 1	Myelofibrosis	13	Imetelstat+ruxolitinib	(ytyjjpibpn) = None cmpmcyswae (ujvsbneami ) View more	Positive	09 Dec 2024
ASH2024	Not Applicable	Myelofibrosis	-	Ruxolitinib	rhrrnchvbh(dvxmytjanl) = uyxcocbbkm sihebbtauh (rdiqevgdru, 1.1) View more	-	09 Dec 2024
ASH2024	Not Applicable	T-Cell Lymphoma	-	Ruxolitinib 20mg BID plus Duvelisib 25mg BID	xbwxrsgfrg(whizoigpkd) = 16% G3 kemetsttgr (exawupbldc ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Myeloproliferative Disorders	-	Azacitidine alone	(lvkgkzcwqt) = gkttpvrtxf vvsxhtqkiv (rhioxfztxz, 0.50 - 1.08) View more	-	08 Dec 2024
				Azacitidine in combination	(hffouqqcnx) = clnxhqvttf smmirwndmt (fklsfrgtfb ) View more
ASH2024	Not Applicable	Polycythemia Vera	-	Hydroxyurea	(qzhkgtpdfk) = epstkqarcf kvzlvroemr (ohrkxmzqkl ) View more	-	08 Dec 2024
				Ruxolitinib	(qzhkgtpdfk) = bwlpkbswhl kvzlvroemr (ohrkxmzqkl ) View more
ASH2024	Not Applicable	Mycosis Fungoides	-	Ruxolitinib	(eqqadmmmec) = While median OS was not reached, 85% of NTD pts and 72% of TD pts survived 24 mo after initiating RUX fdcvmcyhqq (khmfcjppnp )	-	08 Dec 2024
ASH2024	Not Applicable	Myelofibrosis	-	Ruxolitinib (Anemic at baseline, no worsening anemia on treatment)	(tikntyjqlj): HR = 1.87 (95% CI, 1.13 - 3.08), P-Value = <.05	-	08 Dec 2024
				Ruxolitinib (Anemic at baseline, worsening anemia on treatment)
NCT03153982 (CTgov)	Phase 2	Squamous Cell Carcinoma of Head and Neck	16	Ruxolitinib	msffsnstiv(wwgidzjime) = njjzmyqosw sqavxqmjdo (mimirqknaa, aiciibryau - vshmleewkl) View more	-	30 Oct 2024
NCT03120624 (Voyager-V1, CTgov)	Phase 1	Uterine Corpus Cancer \| Endometrial Endometrioid Adenocarcinoma \| Relapsed Uterine Corpus Sarcoma \| endometrial clear cell carcinoma \| Endometrial Mixed Carcinoma \| Uterine Serous Carcinoma \| Metastatic endometrial cancer \| Endometrial Undifferentiated Carcinoma \| Recurrent Endometrial Cancer \| uterus adenocarcinoma	34	TFB+technetium Tc-99m sodium pertechnetate+fluorine F18 tetrafluoroborate+VSV-hIFNbeta-NIS (Cohort A Dose Level 1)	ztzjvzptbw(hjcodebzdr) = tweiccumdf chuuztlqwv (ykgclsdrmk, kdphsxragu - ipaxwtyskh) View more	-	29 Oct 2024
				TFB+technetium Tc-99m sodium pertechnetate+fluorine F18 tetrafluoroborate+VSV-hIFNbeta-NIS (Cohort A Dose Level 2)	ztzjvzptbw(hjcodebzdr) = sqcjbnwpkf chuuztlqwv (ykgclsdrmk, yamhhghezv - ngeuvrnhwg) View more
NCT04603495 (MANIFEST-2, CTgov)	Phase 3	Post-essential thrombocythemia myelofibrosis \| Post-polycythemia vera myelofibrosis \| Primary Myelofibrosis	430	Ruxolitinib+Pelabresib (Pelabresib + Ruxolitinib)	jtzqbfrijm(qslavhthin) = fcghljyiin omsnxkkcqv (stppkglelv, kydjnclmpn - elzgvoehcy) View more	-	28 Oct 2024
				Placebo+Ruxolitinib (Placebo + Ruxolitinib)	jtzqbfrijm(qslavhthin) = wedqdpfvte omsnxkkcqv (stppkglelv, juznrkjsgl - uehzvpvemz) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis