Last update 15 Nov 2024

Cyclophosphamide

Last update 15 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(+-)-Cyclophosphamide, (RS)-Cyclophosphamide, 2-[Bis(2-chloroethylamino)]-tetrahydro-2H-1,3,2-oxazaphosphorine-2-oxide

+ [24]

Target

DNA

Mechanism

DNA inhibitors(DNA inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesInfectious Diseases+ [13]

Active Indication

Graft vs Host DiseaseAmyloidosisLeukemia+ [140]

Inactive Indication

Acidemia, IsovalericAcute Erythroblastic LeukemiaAcute Graft Versus Host Disease+ [140]

Originator Organization

Baxter International, Inc.

Active Organization

Shionogi & Co., Ltd.Hikma Pharmaceuticals USA, Inc.

Pfizer Inc.

+ [31]

Inactive Organization

The Johns Hopkins University

Janssen Pharmaceutica NV

Xian Janssen Pharmaceutical Ltd.

+ [27]

Drug Highest PhaseApproved

First Approval Date

US (16 Nov 1959),

Acute Lymphoblastic LeukemiaAcute Monocytic LeukemiaAcute Myeloid Leukemia+ [12]

RegulationOrphan Drug (US), Orphan Drug (JP)

Structure

Molecular FormulaC7H17Cl2N2O3P

InChIKeyPWOQRKCAHTVFLB-UHFFFAOYSA-N

CAS Registry6055-19-2

3,816

Clinical Trials associated with Cyclophosphamide

NCT06660420 / Not yet recruitingPhase 1IIT

Phase 1 Dose Escalation and Expansion Study of PRAME T Cell Receptor (TCR) Engineered NK Cells in Participants With Recurrent and/or Refractory Melanoma (PRAMETIME-Mel)

To find the highest tolerable dose and recommended dose of PRAME-TCR-NK cells that can be given to participants with recurrent and/or refractory melanoma. The safety and tolerability of PRAME-TCR-NK cells will also be studied.

Start Date16 Apr 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NCT06094842 / Not yet recruitingPhase 1IIT

A Phase I Safety and Feasibility Study of Cellular Immunotherapy for Extensive Stage Small Cell Neuroendocrine Prostate Cancer Using Autologous T Cells Lentivirally Transduced to Express L1CAM-Specific Chimeric Antigen Receptor

This phase I trial studies the side effects and best dose of autologous CD8+ and CD4+ lentivirally transduced to express L1CAM-specific chimeric antigen receptor (CAR) and EGFRt mutation specific T cells and to see how well they work in treating patients with small cell neuroendocrine prostate cancer (SCNPC) that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be removed by surgery (unresectable) or has spread from where it first started (primary site) to other places in the body (metastatic). CAR T-cell therapy is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack tumor cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's tumor cells is added to the T cells in the laboratory. Some solid tumor cells have an L1CAM protein on their surface, and T cells can be modified with a receptor, called a chimeric antigen receptor (CAR), to help recognize this protein and kill these tumor cells. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. These L1CAM mutation specific T cells may help the body's immune system identify and kill L1CAM locally advanced and unresectable or metastatic small cell neuroendocrine prostate cancers' tumor cells.

Start Date15 Apr 2025

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+1]

NCT05582590 / Not yet recruitingPhase 1

A Phase 1 Open-Label Dose-Escalation Study of the Safety of Adoptively Transferred Autologous CD8+ T Lymphocytes Targeting HPV-16 E6/E7, HPV-18 E6/E7 and Survivin in Patients With Relapsed or Refractory HPV-related Oropharyngeal Cancers

This is a multicenter, open-label, Phase I, first-in-human trial to characterize the safety and clinical activity of an antigen-specific CD8+ T-cell product in patients with relapsed or refractory locally advanced or metastatic HPV-related oropharyngeal cancers. Patients must have received at least one prior standard treatment regimen consisting of systemic immunotherapy and/or chemotherapy. The investigative agent is an autologous adoptive T-cell product derived from the patient's endogenous cytolytic T cells that are directed toward HPV-16 E6/E7, HPV-18 E6/E7 antigens, and a tumor-associated antigen (Survivin) by ex vivo exposure to an artificial antigen presenting cell to which HLA-A2 antigen-peptides have been fit within the pocket of an MHC class 1 molecule. Patients must express HLA-A*0201.

Start Date31 Mar 2025

Sponsor / Collaborator

NexImmune, Inc.

100 Clinical Results associated with Cyclophosphamide

100 Translational Medicine associated with Cyclophosphamide

100 Patents (Medical) associated with Cyclophosphamide

52,654

Literatures (Medical) associated with Cyclophosphamide

01 Apr 2025·DEN open

Unique endoscopic features of primary biliary diffuse large B‐cell lymphoma: A case report with literature review (with video)

Article

Author: Ishigami, Keisuke ; Kameyama, Naohiro ; Nakase, Hiroshi ; Masaki, Yoshiharu ; Kawakami, Yujiro ; Sugawara, Taro ; Nakamura, Tomoya ; Satoh, Shuji ; Sugita, Shintaro ; Takada, Yumemi

Abstract:

A 67‐year‐old man visited our hospital complaining of dark‐colored urine and upper abdominal pain. Magnetic resonance cholangiopancreatography showed stricture of the distal bile duct, and contrast‐enhanced computed tomography showed irregular thickening of the distal bile duct wall. However, no enlarged lymph nodes, pancreatic tumors, or other neoplastic lesions were apparent around the bile duct. Endoscopic ultrasonography and intraductal ultrasonography showed irregular thickening of the inner hypoechoic layer without the disappearance of the innermost thin hyperechoic layer. On the basis of these findings, we considered that the bile duct lesion was of non‐epithelial origin. Thus, we repeatedly performed bile duct biopsies from the same site under fluoroscopy to obtain a sample of the submucosal tissue. The pathological diagnosis was diffuse large B‐cell lymphoma, and the patient received systemic chemotherapy (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone). After six courses of rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone, positron emission tomography‐computed tomography showed the disappearance of 18‐fluorodeoxyglucose uptake in the bile duct and endoscopic retrograde cholangiography showed improvement of the bile duct stricture. Endoscopic findings and repeated biopsies were useful in making the diagnosis of primary biliary diffuse large B‐cell lymphoma.

01 Jan 2025·CURRENT PROBLEMS IN CARDIOLOGY

Doxorubicin and cyclophosphamide mode of chemotherapy–related cardiomyopathy: Review of preclinical model

Review

Author: Ottaviani, Giulia ; Rizzo, Manfredi ; Sarrafzadegan, Nizal ; Kakturskiy, Lev ; Pogosova, Nana ; Avagimyan, Ashot

Over the past 70 years, there has been extensive research focused on preventing chemotherapy-related cardiovascular complications. However, the current state of cardio-oncology research has raised more questions than answers. Experimental studies often present data that are difficult to compare and, at times, contradictory. One notable limitation in translating experimental findings to clinical practice is the reliance on models that administer only one chemotherapeutic drug to experimental animals, despite the common use of multidrug cancer treatments in real clinical settings. This article aims to discuss our own experience in modeling an experimental rat model of cardiomyopathy induced by the administration of two chemotherapeutic drugs, doxorubicin (adriamycin) and cyclophosphamide (AC mode of chemotherapy) - Avagimyan A., et al model, along with a subsequent review of morphological changes based on our personal archive.

01 Jan 2025·JOINT BONE SPINE

Update on targeted treatments for ANCA-associated vasculitis

Article

Author: Puéchal, Xavier

Targeted therapy has revolutionized the management of ANCA-associated vasculitis (AAV) over the last fifteen years. Rituximab, an approved induction and maintenance agent for severe AAV, is no less effective than cyclophosphamide as induction therapy and particularly useful in relapsing or refractory disease, or in women. In patients with relapsing AAV, granulomatosis with polyangiitis or PR3-ANCA, it is more effective than cyclophosphamide. Rituximab maintenance is superior to the conventional immunosuppressive drugs that it replaces. Low-dose preemptive rituximab infusions are recommended every 6months for 18months, followed by re-evaluation to decide whether 4 additional biannual infusions should be administered, balancing the probability of relapse and the risk of serious infections on rituximab. A growing body of experimental and clinical data shows that C5a pathway inhibition is a promising therapeutic option for AAV, which could reduce glucocorticoids needs. Avacopan is a first approved oral C5A receptor antagonist, used when there is a high risk that glucocorticoids will cause serious adverse events. In eosinophilic granulomatosis with polyangiitis, the importance of IL-5 for eosinophil activation and survival led to evaluation and approval of mepolizumab, a humanized monoclonal antibody directed against IL-5. Mepolizumab showed a steroid-sparing effect. Its effectiveness in active vasculitis remains uncertain and is currently being evaluated. Benralizumab targeting the IL-5 receptor was recently shown to be noninferior to mepolizumab. Rituximab has had disappointing results in non-severe active vasculitis and is being evaluated as maintenance therapy. Plasma exchange is not indicated as first-line treatment but remains recommended when creatinine levels exceed 300μmol/L.

599

News (Medical) associated with Cyclophosphamide

12 Nov 2024

·www.globenewswire.com

Fate Therapeutics Reports Third Quarter 2024 Financial Results and Business Updates

Initial Clinical Data from Phase 1 Autoimmunity Study of FT819 1XX CAR T-cell Product Candidate to be Presented in 4Q24; Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning Chemotherapy Opened for Enrollment FT522 Multi-indication IND Application for Conditioning-free Treatment of B Cell-mediated Autoimmune Diseases Allowed by FDA; Initial Phase 1 Clinical Data in B-cell Lymphoma to be Presented at ACR Convergence Initial Low-Dose Cohort of FT825 / ONO-8250 Shows Favorable Safety Profile in Phase 1 Solid Tumor Study; Peripheral Blood from First Three Patients Demonstrates CAR T-cell Expansion with Maintenance of Activated State $331 Million in Cash, Cash Equivalents and Investments with Projected Operating Runway through YE26 SAN DIEGO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, today reported business highlights and financial results for the third quarter ended September 30, 2024. “We look forward to sharing initial Phase 1 clinical data of our off-the-shelf FT819 CAR T-cell product candidate in systemic lupus erythematosus at ACR Convergence and ASH. We continue to make great strides in our pursuit of therapeutic differentiation for patients with B cell-mediated autoimmune diseases, and patient enrollment is ongoing assessing FT819 with fludarabine-free conditioning as well as in a new treatment arm as add-on to maintenance therapy without conditioning chemotherapy,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “In addition, we are very pleased with the Phase 1 study design allowed by the FDA for our FT522 ADR-armed CAR NK cell product candidate in autoimmunity, which is designed to assess multiple doses of FT522 without conditioning chemotherapy across a basket of B cell-mediated autoimmune diseases, and we look forward to presenting an initial look at the clinical and translational data of FT522 in B-cell lymphoma at ACR.” FT819 iPSC-derived 1XX CAR T-cell Program Three Patients Treated in Phase 1 Autoimmunity Study using Fludarabine-free Conditioning. The ongoing multi-center, Phase 1 clinical trial for patients with moderate-to-severe systemic lupus erythematosus (SLE) is designed to evaluate the safety, pharmacokinetics, and anti-B cell activity of FT819, the Company’s off-the-shelf CD8αβ+ T-cell product candidate that incorporates a CD19-targeted chimeric antigen receptor (CAR) with a novel 1XX costimulatory domain into the T-cell receptor alpha constant (TRAC) locus (NCT06308978). The first three patients, all of whom presented with active lupus nephritis (LN) despite having been treated with multiple standard-of-care therapies, received fludarabine-free conditioning consisting of either bendamustine alone or cyclophosphamide alone, followed by a single dose of FT819 at 360 million cells (Regimen A). All three patients remain on-study, and there have been no dose-limiting toxicities (DLTs) and no events of any grade of cytokine release syndrome (CRS), immune effector-cell associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD). The Company plans to present clinical and translational data from the first three patients at the American Society of Hematology (ASH) Annual Meeting being held in San Diego, CA on December 7-10.First SLE Patient Case Study to be Presented at ACR Convergence. The first patient treated in the ongoing FT819 Phase 1 Autoimmunity study was a 27 year-old woman diagnosed with LN over ten years ago who received fludarabine-free conditioning followed by a single dose of FT819 at 360 million cells. The first patient has completed six-month follow-up and remains on-study. The Company plans to present clinical and translational data from the first patient at the American College of Rheumatology (ACR) Convergence being held in Washington, D.C. on November 16-19.Initiated Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning. The Company amended the clinical protocol of its FT819 Phase 1 Autoimmunity study to include a second treatment arm (Regimen B) to assess the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 as an add-on to maintenance therapy without conditioning chemotherapy in patients with SLE. The new arm is open for enrollment at a starting cell dose of 360 million cells, and is being conducted in parallel with Regimen A. FT825 / ONO-8250 iPSC-derived CAR T-cell Program Initial Phase 1 Clinical Data Presented at 2024 SITC. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). At the 2024 Society of Immunotherapy of Cancer (SITC) 39th Annual Meeting, the Company presented initial clinical data from three heavily pre-treated patients, all of whom were previously treated with at least five prior lines of therapy including HER2-targeted therapy. Each patient was administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 at the first dose level of 100 million cells. As of a data cutoff date of October 25, 2024, FT825 / ONO-8250 demonstrated a favorable safety profile with no DLTs and no events of any grade of CRS, ICANS, or GvHD. In addition, at Day 8 following treatment, peak CAR T-cell expansion was observed and phenotyping of FT825 / ONO-8250 sourced from the patients’ peripheral blood was indicative of an activated state (as evidenced by high levels of Granzyme B expression and maintenance of CAR expression) with no evidence of exhaustion (as evidenced by low levels of PD-1 and TIM3 expression). Enrollment is currently ongoing at the second dose level of 300 million cells as monotherapy and at the first dose level of 100 million cells in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy.New Preclinical Data Demonstrates Cancer-selectivity of Novel H2CasMab-2 CAR. FT825 / ONO-8250 incorporates seven synthetic controls of cell function including a novel cancer-selective H2CasMab-2 CAR, which has exhibited similar potency with greater specificity for cancer cells expressing HER2 compared to trastuzumab in preclinical studies. New preclinical data presented at SITC demonstrated potent HER2-specific, anti-tumor activity in both in vitro and in vivo settings with limited cytolytic targeting of HER2+ normal cells. The on-tumor selectivity of FT825 / ONO-8250 was attributed to its incorporation of a novel HER2-targeted antigen binding domain, which was derived from a cancer-specific monoclonal antibody H2CasMab-2 (Kaneko et al., 2024), that was shown to differentially and preferentially recognize both locally misfolded HER2 and p95 truncation variants of HER2 as compared to trastuzumab. FT522 iPSC-derived CAR NK Cell Program Initial Clinical Data from Phase 1 BCL Study to be Presented at ACR Convergence. FT522 is the Company’s off-the-shelf, CD19-targeted CAR NK cell product candidate and its first to incorporate Alloimmune Defense Receptor (ADR) technology, which is designed to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In its ongoing multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), the Company is currently enrolling patients in the second three-dose cohort at 900 million cells per dose with conditioning chemotherapy (Regimen A) and in the first three-dose cohort at 300 million cells per dose without conditioning chemotherapy (Regimen B). No DLTs, and no events of any grade of CRS, ICANS, or GvHD, have been reported in the Phase 1 study. The Company plans to present initial clinical and translational data from the Phase 1 BCL study at ACR Convergence.IND Application for Phase 1 Basket Study in Autoimmunity Allowed by FDA. The U.S. Food and Drug Administration (FDA) has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Phase 1 study is intended to treat patients with multiple doses of FT522 without conditioning chemotherapy as an add-on to rituximab induction therapy (Regimen A) and as an add-on to maintenance therapy in combination with rituximab (Regimen B). Dose escalation is expected to commence at 900 million cells per dose. The Company previously presented preclinical data from a novel re-challenge assay using peripheral blood mononuclear cells (PBMCs) from unmatched SLE donors, showing that FT522 uniquely drove rapid and deep CD19+ B cell depletion, maintained functional persistence, and eliminated alloreactive T cells, indicating that FT522 has the potential to function effectively in the presence of an unmatched host immune system. Third Quarter 2024 Financial Results Cash & Investment Position: Cash, cash equivalents, and investments as of September 30, 2024 were $330.5 million.Total Revenue: Revenue was $3.1 million for the third quarter of 2024, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under its collaboration with Ono.Total Operating Expenses: Total operating expenses were $55.5 million for the third quarter of 2024, including research and development expenses of $34.7 million and general and administrative expenses of $20.8 million. Such amounts included $11.8 million of non-cash stock-based compensation expense.Shares Outstanding: As of September 30, 2024, common shares outstanding were 113.9 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares. About Fate Therapeutics’ iPSC Product PlatformHuman induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be combined and administered with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the manufacture of cell therapies using patient- or donor-sourced cells. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications. About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived natural killer (NK) cell and T-cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com. Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Company’s results of operations, financial condition, anticipated operating expenses and cash runway, and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, including the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the availability of data from the Company’s clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, the Company’s clinical and product development strategy, and the Company’s expectations regarding progress and timelines, and the objectives, plans and goals of its collaboration with Ono. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, the risk that the Company may not comply with its obligations under and otherwise maintain its collaboration agreement with Ono, the risk that research funding and milestone payments received by the Company under its collaboration may be less than expected, and the risk that the Company may incur operating expenses in amounts greater than anticipated. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise. Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(unaudited) Three Months Ended Nine Months Ended September 30, September 30, 2024 2023 2024 2023 Collaboration revenue$3,074 $1,944 $11,771 $61,857 Operating expenses: Research and development 34,650 34,275 101,392 140,780 General and administrative 20,801 18,948 58,907 63,513 Total operating expenses 55,451 53,223 160,299 204,293 Loss from operations$(52,377) $(51,279) $(148,528) $(142,436)Other income (expense): Interest income 4,438 4,697 13,414 12,772 Change in fair value of stock price appreciation milestones (13) 1,049 149 3,160 Other Income 274 363 856 9,698 Total other income, net 4,699 6,109 14,419 25,630 Net loss$(47,678) $(45,170) $(134, 109) $(116,806)Other comprehensive income: Unrealized gain on available-for-sale securities, net 1,257 88 820 1,355 Comprehensive loss$(46,421) $(45,082) $(133,289) $(115,451)Net loss per common share, basic and diluted$(0.40) $(0.46) $(1.19) $(1.19)Weighted–average common shares used to compute basic and diluted net loss per share 117,768,161 98,568,012 112,305,430 98,342,898 Condensed Consolidated Balance Sheets(in thousands)(unaudited) September 30, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents$37,909 $41,870 Accounts receivable 4,127 1,826 Short-term investments 259,014 273,305 Prepaid expenses and other current assets 9,244 14,539 Total current assets$310,294 $331,540 Long-term investments 33,607 980 Operating lease right-of-use asset 58,441 61,675 Other long-term assets 92,628 112,022 Total assets$494,970 $506,217 Liabilities and stockholders’ equity Current liabilities: Accounts payable and accrued expenses$29,929 $32,233 Deferred revenue 600 685 Operating lease liability, current portion 6,893 6,176 Total current liabilities$37,422 $39,094 CIRM award liability 1,940 --- Operating lease liability, net of current portion 92,085 97,360 Stock price appreciation milestones 1,197 1,346 Stockholders’ equity 362,326 368,417 Total liabilities and stockholders’ equity$494,970 $506,217 Contact:Christina TartagliaPrecision AQ212.362.1200christina.tartaglia@precisionaq.com

Phase 1Cell TherapyImmunotherapyASHFinancial Statement

12 Nov 2024

·www.prnewswire.com

Heron Therapeutics Announces Third Quarter 2024 Financial Results and Narrows Financial Guidance

Reported positive YTD 2024 Adjusted EBITDA of $1.4 million Company expects Q4 2024 Net Revenue in the range of $37 million - $43 million ZYNRELEF® (bupivacaine and meloxicam) extended-release solution Vial Access Needle ("VAN") approved in September and on track for Q4 2024 launch CMS Final Rule Non-Opioid Policy for Pain Relief includes ZYNRELEF as a qualifying product for separate payment in both the hospital outpatient department and ambulatory surgical center settings of care SAN DIEGO, Nov. 12, 2024 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, today announced financial results for the three and nine months ended September 30, 2024, and highlighted recent corporate updates. "We are pleased to report that with our prudent financial management and continued revenue growth we were able to achieve positive Adjusted EBITDA for the quarter," said Craig Collard, Chief Executive Officer. "The future looks bright as we continue to grow all product revenue. The CrossLink partnership roll out, FDA approval of the VAN in September, and inclusion in the CMS Final Rule Non-Opioid Policy for Pain Relief, positions ZYNRELEF for significant growth within the surgical setting." "We continue to deliver on our commitment to financial efficiency while growing revenue. The team has made great strides in the transformation of Heron over the past year. We are looking forward to a strong fourth quarter which is off to a great start. As such, we are narrowing guidance for full-year 2024." Financial Guidance for 2024 The Company narrows its full-year 2024 guidance for Product Revenues, Net, Adjusted Operating Expenses and Adjusted EBITDA: Business Highlights The ZYNRELEF VAN will be available for initial use in the fourth quarter following approval by the U.S. Food and Drug Administration ("FDA") on September 24, 2024. The VAN will replace the current vented vial spike and has the potential to simplify aseptic preparation, while also significantly reducing ZYNRELEF's withdrawal time down to between twenty and forty-five seconds. ZYNRELEF will continue to receive separate payment from April 1, 2025, until at least the end of 2027 as the result of inclusion in the "CMS OPPS and ASC Final Rule CY 2025 Non-Opioid Policy for Pain Relief" by the Centers for Medicare & Medicaid Services ("CMS"). The payment limitation for ZYNRELEF is set at $2,267.26, in line with similar products, and it will be granted the status indicator of "K1" – for Non-Opioid Drugs and Biologicals For Post-Surgical Pain Relief. The goal of the policy is to ensure there are no financial incentives to use opioids instead of non-opioid alternatives like ZYNRELEF. To qualify under the new policy, ZYNRELEF needed to meet strict criteria establishing that it does not act upon the body's opioid receptors and that it successfully demonstrated the ability to replace, reduce, or avoid intraoperative or postoperative opioid requirements in clinical trials or peer reviewed literature. These criteria set a strong precedent from CMS that we expect other payors to follow. Cash and Cash Equivalents were $70.9 million as of September 30, 2024 , compared with $80.4 million on December 31, 2023. Acute Care Franchise Acute Care Franchise Net Product Sales: For the three and nine months ended September 30, 2024, acute care franchise Net Product Sales were $7.4 million and $19.7 million, respectively, compared to $4.7 million and $12.9 million, respectively, for the same period in 2023. ZYNRELEF Net Product Sales: Net Product Sales of ZYNRELEF for the three and nine months ended September 30, 2024 were $6.3 million and $17.1 million, respectively, compared to $4.4 million and $12.0 million, respectively, for the same period in 2023. APONVIE® Net Product Sales: Net Product Sales of APONVIE for the three and nine months ended September 30, 2024 were $1.1 million and $2.6 million, respectively, compared to $0.3 million and $0.9 million, respectively, for the same period in 2023. Oncology Care Franchise Oncology Care Franchise Net Product Sales: For the three and nine months ended September 30, 2024, oncology care franchise Net Product Sales were $25.4 million and $83.8 million, respectively, compared to $26.7 million and $79.9 million for the same period in 2023. CINVANTI® Net Product Sales: Net Product Sales of CINVANTI (aprepitant) injectable emulsion for the three and nine months ended September 30, 2024 were $22.6 million and $73.2 million, compared to $23.3 million and $70.6 million for the same period in 2023. SUSTOL® Net Product Sales: Net Product Sales of SUSTOL (granisetron) extended-release injection for the three and nine months ended September 30, 2024 were $2.8 million and $10.6 million, respectively, compared to $3.4 million and $9.3 million, respectively, for the same period in 2023. Conference Call and Webcast Heron will host a conference call and live webcast on Tuesday, November 12, 2024, at 8:00 a.m. ET. The conference call can be accessed by phone by utilizing the following registration link which will provide participants with dial-in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. The conference call will also be available via webcast under the Investor Relations section of Heron's website at . An archive of the teleconference and webcast will also be made available on Heron's website for sixty days following the call. About ZYNRELEF for Postoperative Pain ZYNRELEF is the first and only dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of nonsteroidal anti-inflammatory drug meloxicam. ZYNRELEF is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and significantly increased proportion of patients requiring no opioids through the first 72 hours following surgery compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. ZYNRELEF was initially approved by the FDA in May 2021 for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. In December 2021, the FDA approved an expansion of ZYNRELEF's indication to include foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures. On January 23, 2024, the FDA approved ZYNRELEF for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. Safety and efficacy have not been established in highly vascular surgeries, such as intrathoracic, large multilevel spinal, and head and neck procedures. Please see full prescribing information, including Boxed Warning, at . About APONVIE for Postoperative Nausea and Vomiting (PONV) APONVIE is a substance NK1 Receptor Antagonist (RA), indicated for the prevention of PONV in adults. Delivered via a 30-second IV push, APONVIE 32 mg was demonstrated to be bioequivalent to oral aprepitant 40 mg with rapid achievement of therapeutic drug levels. APONVIE is the same formulation as Heron's approved drug product CINVANTI. APONVIE is supplied in a single-dose vial that delivers the full 32 mg dose for PONV. APONVIE was approved by the FDA in September 2022 and became commercially available in the U.S. on March 6, 2023. Please see full prescribing information at . About CINVANTI for Chemotherapy Induced Nausea and Vomiting (CINV) Prevention CINVANTI, in combination with other antiemetic agents, is indicated in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC) including high-dose cisplatin as a single-dose regimen, delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC) as a single-dose regimen, and nausea and vomiting associated with initial and repeat courses of MEC as a 3-day regimen. CINVANTI is an IV formulation of aprepitant, an NK1 RA. CINVANTI is the first IV formulation to directly deliver aprepitant, the active ingredient in EMEND® capsules. Aprepitant (including its prodrug, fosaprepitant) is a single-agent NK1 RA to significantly reduce nausea and vomiting in both the acute phase (0–24 hours after chemotherapy) and the delayed phase (24–120 hours after chemotherapy). The FDA-approved dosing administration included in the U.S. prescribing information for CINVANTI include 100 mg or 130 mg administered as a 30-minute IV infusion or a 2-minute IV injection. Please see full prescribing information at . About SUSTOL for CINV Prevention SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-hydroxytryptamine type 3 RA that utilizes Heron's Biochronomer® drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical studies that evaluated SUSTOL's efficacy and safety in more than 2,000 patients with cancer. SUSTOL's efficacy in preventing nausea and vomiting was evaluated in both the acute phase (0–24 hours after chemotherapy) and delayed phase (24–120 hours after chemotherapy). Please see full prescribing information at . About Heron Therapeutics, Inc. Heron Therapeutics, Inc. is a commercial-stage biotechnology company focused on improving the lives of patients by developing and commercializing therapeutic innovations that improve medical care. Our advanced science, patented technologies, and innovative approach to drug discovery and development have allowed us to create and commercialize a portfolio of products that aim to advance the standard-of-care for acute care and oncology patients. For more information, visit . Non-GAAP Financial Measures To supplement our financial results presented on a GAAP basis, we have included information about certain non-GAAP financial measures. We believe the presentation of these non-GAAP financial measures, when viewed with our results under GAAP, provide analysts, investors, lenders, and other third parties with insights into how we evaluate normal operational activities, including our ability to generate cash from operations, on a comparable year-over-year basis and manage our budgeting and forecasting. In our quarterly and annual reports, earnings press releases and conference calls, we may discuss the following financial measures that are not calculated in accordance with GAAP, to supplement our consolidated financial statements presented on a GAAP basis. Adjusted EBITDA Adjusted EBITDA is a non-GAAP financial measure that represents GAAP net income or loss adjusted to exclude interest expense, interest income, the benefit from or provision for income taxes, depreciation, amortization, stock-based compensation, and other adjustments to reflect changes that occur in our business but do not represent ongoing operations. Adjusted EBITDA, as used by us, may be calculated differently from, and therefore may not be comparable to, similarly titled measures used by other companies. There are several limitations related to the use of adjusted EBITDA rather than net income or loss, which is the nearest GAAP equivalent, such as: adjusted EBITDA excludes depreciation and amortization and, although these are non-cash expenses, the assets being depreciated or amortized may have to be replaced in the future, the cash requirements for which are not reflected in adjusted EBITDA; we exclude stock-based compensation expense from adjusted EBITDA although: (i) it has been, and will continue to be for the foreseeable future, a significant recurring expense for our business and an important part of our compensation strategy; and (ii) if we did not pay out a portion of our compensation in the form of stock-based compensation, the cash salary expense included in operating expenses would be higher, which would affect our cash position; we exclude impairment of long-lived assets, the amount and/or frequency of which are not part of our underlying business. we exclude inventory write-downs (and write-ups should they occur), the amount and/or frequency of which are not part of our underlying business. adjusted EBITDA does not reflect changes in, or cash requirements for, working capital needs; adjusted EBITDA does not reflect the benefit from or provision for income taxes or the cash requirements to pay taxes; adjusted EBITDA does not reflect historical cash expenditures or future requirements for capital expenditures or contractual commitments; and we exclude restructuring expenses from adjusted EBITDA. Restructuring expenses primarily include employee severance and contract termination costs that are not related to acquisitions. The amount and/or frequency of these restructuring expenses are not part of our underlying business. Adjusted Operating Expenses Adjusted operating expenses is a non-GAAP financial measure that represents GAAP operating expenses adjusted to exclude stock-based compensation expense, depreciation and amortization, and other adjustments to reflect changes that occur in our business but do not represent ongoing operations. The Company has not provided a reconciliation of its full-year 2024 guidance for adjusted EBITDA or adjusted operating expenses to the most directly comparable forward-looking GAAP measures, in reliance on the unreasonable efforts exception provided under Item 10(e)(1)(i)(B) of Regulation S-K, because the Company is unable to predict, without unreasonable efforts, the timing and amount of items that would be included in such a reconciliation, including, but not limited to, stock-based compensation expense, acquisition related expense and litigation settlements. These items are uncertain and depend on various factors that are outside of the Company's control or cannot be reasonably predicted. While the Company is unable to address the probable significance of these items, they could have a material impact on GAAP net income and operating expenses for the guidance period. Forward-looking Statements This news release contains "forward-looking statements" as defined by the Private Securities Litigation Reform Act of 1995. Heron cautions readers that forward-looking statements are based on management's expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially. Therefore, you should not place undue reliance on forward-looking statements. Examples of forward-looking statements include, among others, statements we make regarding the potential market opportunities for ZYNRELEF, APONVIE, CINVANTI and SUSTOL; revenue, adjusted EBITDA and other financial guidance provided by the Company; the results of the commercial launch of APONVIE; the potential additional market opportunity for the expanded U.S. label for ZYNRELEF or inclusion of ZYNRELEF under the OPPS and the ASC payment system; the timing of the Company's development of the VAN program and receipt of required regulatory approvals; our ability to establish and maintain successful commercial arrangements like our co-promotion agreement with CrossLink Life Sciences, LLC ("CrossLink"); the outcome of the Company's pending ANDA litigation; whether the Company is required to write-off any additional inventory in the future; the expected future balances of Heron's cash, cash equivalents and short-term investments; the expected duration over which Heron's cash, cash equivalents and short-term investments balances will fund its operations and the risk that future equity financings may be needed; any inability or delay in achieving profitability. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q, and in our other reports filed with the Securities and Exchange Commission, including under the caption "Risk Factors." Forward-looking statements reflect our analysis only on their stated date, and Heron takes no obligation to update or revise these statements except as may be required by law. Investor Relations and Media Contact: Ira Duarte Executive Vice President, Chief Financial Officer Heron Therapeutics, Inc. [email protected] 858-251-4400 SOURCE Heron Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalFinancial StatementClinical Result

12 Nov 2024

·www.drugs.com

Actuate Announces FDA Rare Pediatric Disease Designation Granted to Elraglusib for Treatment of Ewing Sarcoma

CHICAGO and FORT WORTH, Texas, Nov. 12, 2024 (GLOBE NEWSWIRE) — Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company, focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to elraglusib, a novel GSK- 3β inhibitor for treatment of Ewing sarcoma (EWS). “Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with EWS and recognizes elraglusib’s transformative potential,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “Early clinical data from our ongoing Phase 1/2 trial show promising anti-tumor activity with objective tumor responses, including two ongoing durable Complete Responses (CRs) in the first six patients treated with relapsed/refractory EWS, reinforcing our confidence in elraglusib’s potential impact in this challenging disease setting. We are committed to advancing elraglusib’s clinical development with the ultimate goal of providing new therapeutic options where current approaches are unsatisfactory.” Ewing sarcoma (EWS) is a highly metastatic form of sarcoma, originating in bone with a peak incidence at the age of 15, that ranks as the second most prevalent primary malignant tumor of childhood and adolescence. Approximately 25% of new EWS patients have metastatic disease when first diagnosed, which is the most significant predictor of poor survival. The ongoing Phase 1/2 Trial (NCT 04239092), also referred to as Actuate-1902, is an open-label, multicenter study evaluating the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including EWS and EWS-related pediatric small round cell sarcomas. To date, the study has enrolled 8 patients with relapsed/refractory EWS (>1 remission) treated with the combination of elraglusib and topotecan/cyclophosphamide. Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for elraglusib for the treatment of Ewing sarcoma is approved by the FDA, Actuate will be eligible to receive a Priority Review Voucher (PRV) that could be utilized by the Company or potentially sold to another company for its use. About Actuate Therapeutics, Inc. Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug product, elraglusib (a novel GSK- 3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy including several DNA Damage Response (DDR) pathways. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com. Forward-Looking Statements This press release contains forward-looking statements about us, including our clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, and elraglusib may not achieve favorable results in clinical trials or preclinical studies or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities; and our ability to realize the benefits associated with Rare Pediatric Disease Designation, including receipt of a Priority Review Voucher or any value therefrom. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading “Item 1A. Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024 filed with the SEC on September 24, 2024 and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events. Source: Actuate Therapeutics, Inc. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Priority ReviewNDAFast TrackClinical ResultAccelerated Approval

100 Deals associated with Cyclophosphamide

External Link

KEGG	Wiki	ATC	Drug Bank
D07760	Cyclophosphamide	L01AA01	DB00531

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Graft vs Host Disease	JP	Shionogi & Co., Ltd.	09 Feb 2024
Amyloidosis	JP	Shionogi Pharma Co., Ltd.	25 Aug 2021
Leukemia	US	Eugia Pharma Specialities Ltd.	25 Aug 2021
Pheochromocytoma	JP	Shionogi Pharma Co., Ltd.	25 Mar 2013
Palindromic Rheumatism	JP	Shionogi Pharma Co., Ltd.	23 Feb 2011
Hematopoietic stem cell transplantation	JP	Shionogi Pharma Co., Ltd.	09 Oct 2003
Lymphoid Leukemia	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Nasopharyngeal Carcinoma	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Osteosarcoma	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Ovarian Cancer	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Rhabdomyosarcoma	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Testicular Neoplasms	CN	Jinghua Pharmaceutical Group Co., Ltd.	01 Jan 1982
Lung Cancer	JP	Shionogi Pharma Co., Ltd.	14 Mar 1962
Nephrotic Syndrome	JP	Shionogi Pharma Co., Ltd.	14 Mar 1962
Acute Lymphoblastic Leukemia	US	Baxter Healthcare Corp.	16 Nov 1959
Acute Monocytic Leukemia	US	Baxter Healthcare Corp.	16 Nov 1959
Acute Myeloid Leukemia	US	Baxter Healthcare Corp.	16 Nov 1959
Breast Cancer	US	Baxter Healthcare Corp.	16 Nov 1959
Burkitt Lymphoma	US	Baxter Healthcare Corp.	16 Nov 1959
Chronic Lymphocytic Leukemia	US	Baxter Healthcare Corp.	16 Nov 1959

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
acute leukemia	Phase 3	US	National Cancer Institute	25 Jun 2019
acute leukemia	Phase 3	US	National Heart, Lung & Blood Institute	25 Jun 2019
Lymphoma	Phase 3	US	National Heart, Lung & Blood Institute	25 Jun 2019
Relapse multiple myeloma	Phase 3	JP	Celgene Corp.	13 Sep 2017
Relapse multiple myeloma	Phase 3	HK	Celgene Corp.	13 Sep 2017
Relapse multiple myeloma	Phase 3	SG	Celgene Corp.	13 Sep 2017
Relapse multiple myeloma	Phase 3	KR	Celgene Corp.	13 Sep 2017
Relapse multiple myeloma	Phase 3	TW	Celgene Corp.	13 Sep 2017
Alveolar Rhabdomyosarcoma	Phase 3	US	National Cancer Institute	01 Jun 2016
Alveolar Rhabdomyosarcoma	Phase 3	AU	National Cancer Institute	01 Jun 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Hematopoietic stem cell transplantation	-	Post-transplant cyclophosphamide (PTCy)	pavnlxbkku(ytrojvzvdc) = nqourlcmfg fagchflmsh (fxhsnitwia )	-	09 Dec 2024
				Calcineurin inhibitor (CNI)	pavnlxbkku(ytrojvzvdc) = jsaaurlbpm fagchflmsh (fxhsnitwia )
ASH2024	Not Applicable	Hematopoietic stem cell transplantation	-	Cyclophosphamide (Monocyte Recovery >0.3 x 10^9/L at D+100)	elyzkxixcv(mgrvcbnrht) = ycfakgrdpb yfhlclyxev (eoyrxoqgaw, 50 - 65) View more	-	09 Dec 2024
				Cyclophosphamide (Monocyte Recovery <0.3 x 10^9/L at D+100)	elyzkxixcv(mgrvcbnrht) = dzsoglnlfd yfhlclyxev (eoyrxoqgaw, 15 - 45) View more
ASH2024	Not Applicable	-	-	Abatacept and Post Transplant Cyclophosphamide	sbluxpqicb(jueaucpdao) = psevweoilg subnolhfxg (wdoklbefpk ) View more	-	09 Dec 2024
ASH2024	Not Applicable	Peripheral Stem Cell Transplantation	-	PTCy 30 mg/kg on D3 and D4	odwrlpkfju(zdoocfoqpz) = mqlzzmrvjl trgfkmlcmj (vqcvwdbqsi, 4.3 - 9.6) View more	-	09 Dec 2024
ASH2024	Not Applicable	Hematopoietic stem cell transplantation	-	Posttransplant Cyclophosphamide (Peripheral Blood Stem Cell (PBSC) Graft)	efwfxpepkf(pkcurcheyz) = afoaezihpr npmjxahyej (wjrubvwzvg ) View more	-	09 Dec 2024
				Posttransplant Cyclophosphamide (Bone Marrow (BM) Graft)	efwfxpepkf(pkcurcheyz) = pkjpcwoffw npmjxahyej (wjrubvwzvg ) View more
ASH2024	Not Applicable	-	-	Post-transplant cyclophosphamide (PTCy) + calcineurin inhibitor (CnI) and mycophenolate mofetil (MMF)	wpcnigkoqw(dcsoqxddzf) = pqgkukcoup xhqdjucodp (vqycjufgce, 42 - 75) View more	-	09 Dec 2024
				Calcineurin inhibitor (CnI) and methotrexate (MTX)	wpcnigkoqw(dcsoqxddzf) = mkxpioztei xhqdjucodp (vqycjufgce, 10 - 27) View more
ASH2024	Not Applicable	Immune Reconstitution Inflammatory Syndrome \| Graft vs Host Disease	-	Calcineurin inhibitor plus methotrexate (CNI+MTX)	kiqtrqzwht(gqvzwrgyag) = buumpnezuv aezscgyvjx (gxtmvuydub )	-	09 Dec 2024
3543 (ASH2024)	Not Applicable	acute leukemia \| Myelodysplastic Syndromes	865	Post-Transplant Cyclophosphamide (Age >70 years)	agapyvsnzk(hvjpzhjhmn) = rtdfjwsufu cqdletevnj (rutsfwysiu ) View more	Negative	08 Dec 2024
				Post-Transplant Cyclophosphamide (Age 18-70 years)	agapyvsnzk(hvjpzhjhmn) = mvphcpjxpu cqdletevnj (rutsfwysiu ) View more
ASH2024	Not Applicable	Hodgkin's Lymphoma	-	Brentuximab Vedotin, Cyclophosphamide, Doxorubicin and Prednisone (B-CAP)	ntwoqrqtnb(hmroakcrpp) = Primary G-CSF support documented in 98% of patients spgnwbqydf (ztlwjfzryn ) View more	-	08 Dec 2024
3551Acute Lymphoblastic Leukemia (ALL) (ASH2024)	Not Applicable	Acute Lymphoblastic Leukemia Consolidation	-	Cyclophosphamide (ERA1)	jxmcdxvbnv(ogxmuywefv) = ibhagwdsds pxvnqrnuqu (fhqmpaxfkx ) View more	Positive	08 Dec 2024
				Cyclophosphamide (ERA2)	jxmcdxvbnv(ogxmuywefv) = osjjqwgpop pxvnqrnuqu (fhqmpaxfkx ) View more

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