As people get older, there are changes in their cells and tissues that may affect their ability to function. This can lead to increased death and age-associated disorders, like heart disease, cancer, and Alzheimer's disease. Studies in animal models have been able to identify drugs that slow the aging process, leading to a longer, healthier life. This study is focused on one such family of drugs, called mTOR inhibitors, and the investigators' goal is to test two of these drugs, Rapamycin (Sirolimus) and Everolimus (Afinitor), in healthy older adults to find a dose and dose timing that can be used to safely inhibit mTOR to the levels seen in young healthy persons. The investigators expect that the dose that works well in women may differ from the one that is best in men, so it is important to include both sexes in this research.

Start Date01 Jan 2025

Sponsor / Collaborator

University of Texas Health Science Center At San Antonio

[+1]

NCT04736953 / Not yet recruitingPhase 2IIT

Sirolimus Treatment of Patients With Systemic Lupus Erythematosus

Phase II Double-blind, placebo-controlled, randomized treatment trial with two arms: one SIROLIMUS arm with 92 patients and one placebo arm with 92 patients. The safety and therapeutic efficacy of SIROLIMUS will be determined within a dosage range of 1 mg/day to 4 mg/day, which will be titrated to tolerance during an initial 3-month open label period, relative to placebo in SLE patients over 12 months followed by a 1-month washout. The proposed study design, known as an enriched enrollment randomized withdrawal (EERW), has major advantages that (1) only people who tolerate SIROLIMUS are randomized, potentially reducing the percentage of dropouts in the randomized phase and (2) it allows participants to use an individualized dosage of study medication, which mimics clinical practice in terms of how SIROLIMUS would be administered. Healthy subjects receive no drugs and serve as controls for in vitro studies.

Start Date01 Jan 2025

Sponsor / Collaborator

SUNY Upstate Medical University

NCT06552169 / Not yet recruitingPhase 2

The RETIRE Trial: A Randomized Phase 2 Trial of Adoptive Therapy With Treg Adoptive Cell Transfer (TRACT) To Prevent Rejection in Living Donor Kidney Transplant Recipients

The goal of this multi-national, multi-center, open-label, randomized Phase 2 trial is to determine the safety and efficacy of administering expanded regulatory T cells (TRK-001) to prevent allograft rejection in living donor renal transplant recipients.
Enrolled subjects will be randomized to one of 2 study arms:
Arm 1 subjects will receive standard of care immunosuppression
Arm 2 subjects will receive initial standard of care (SOC) immunosuppression and a single infusion of TRK-001. Three months after the transplant, Arm 2 subjects may be able to begin reducing their immunosuppression medication to a 1-drug regimen.
The primary outcome measures of trial are to evaluate several components indicating immunologic problems with the transplanted organ at 1-year post-transplant and to evaluate the ability for the study subjects given TRK-001 to wean to a 1-drug immunosuppression regimen.
All enrolled subjects will be followed for 5 years post-transplant.

Start Date01 Dec 2024

Sponsor / Collaborator

TRACT Therapeutics, Inc.

[+1]

100 Clinical Results associated with Sirolimus

100 Translational Medicine associated with Sirolimus

100 Patents (Medical) associated with Sirolimus

25,105

Literatures (Medical) associated with Sirolimus

01 Dec 2025·Journal of Clinical Immunology

Novel Compound Heterozygous Variants in the FAS Gene Lead to Fetal Onset of Autoimmune Lymphoproliferative Syndrome (ALPS)

Article

Author: Yu, Meiping ; Wu, Bingbing ; Zhu, Li ; Chen, Yao ; Li, Qifan ; Wang, Chenghao ; Qian, Feng ; Zhou, Weitao ; Liu, Luyao ; Wu, Qi ; Hui, Xiaoying ; Hou, Jia ; Zhou, Qinhua ; Wang, Xiaochuan ; Sun, Bijun ; Wang, Ying ; Sun, Jinqiao ; Ying, Wenjing ; Wang, Wenjie

OBJECTIVEFAS gene defects lead to autoimmune lymphoproliferative syndrome (ALPS), which is often inherited in an autosomal dominant and rarely in an autosomal recessive manner. We report a case of a newborn girl with novel compound heterozygous variants in FAS and reveal the underlying mechanism.METHODSWhole-exome sequencing (WES) was used to identify pathogenic variants. Multiparametric flow cytometry analysis, phosflow analysis, and FAS-induced apoptosis assays were used to explore the effects of the variants on FAS expression, apoptosis, and immunophenotype. The HEK293T cells were used to assess the impact of the variants on protein expression and FAS-induced apoptosis.RESULTSThe patient was born with hepatosplenomegaly, anemia, and thrombocytopenia. She also experienced COVID-19, rotavirus infection, herpes simplex virus infection, and severe pneumonia. The proportion of double-negative T cells (DNTs) was significantly elevated. Novel FAS compound heterozygous variants c.310T > A (p.C104S) and c.702_704del (p.T235del) were identified. The apoptotic ability of T cells was defective, and FAS expression on the surface of T cells was deficient. The T235del variant decreased FAS expression, and the C104S protein remained in the endoplasmic reticulum (ER) and could not translocate to the cell surface. Both mutations resulted in loss-of-function in terms of FAS-induced apoptosis in HEK293T cells. The DNTs were mainly terminally differentiated T (TEMRA) and CD45RA⁺HLA-DR⁺, with high expression of CD85j, PD-1, and CD57. The percentage of Th1, Tfh, and autoreactive B cells were significantly increased in the patient. The abnormal immunophenotyping was partially attenuated by sirolimus treatment.CONCLUSIONSWe identified two variants that significantly affect FAS expression or localization, leading to early disease onset of in the fetus. Abnormalities in the mTOR pathway are associated with a favorable response to sirolimus.

01 Jun 2025·Neural Regeneration Research

Mechanism by which Rab5 promotes regeneration and functional recovery of zebrafish Mauthner axons

Article

Author: Cui, Jiantao ; Hu, Bing ; Shen, Yueru ; Fan, Dinggang ; Song, Zheng

JOURNAL/nrgr/04.03/01300535-202506000-00031/figure1/v/2024-08-08T040853Z/r/image-tiff Rab5 is a GTPase protein that is involved in intracellular membrane trafficking. It functions by binding to various effector proteins and regulating cellular responses, including the formation of transport vesicles and their fusion with the cellular membrane. Rab5 has been reported to play an important role in the development of the zebrafish embryo; however, its role in axonal regeneration in the central nervous system remains unclear. In this study, we established a zebrafish Mauthner cell model of axonal injury using single-cell electroporation and two-photon axotomy techniques. We found that overexpression of Rab5 in single Mauthner cells promoted marked axonal regeneration and increased the number of intra-axonal transport vesicles. In contrast, treatment of zebrafish larvae with the Rab kinase inhibitor CID-1067700 markedly inhibited axonal regeneration in Mauthner cells. We also found that Rab5 activated phosphatidylinositol 3-kinase (PI3K) during axonal repair of Mauthner cells and promoted the recovery of zebrafish locomotor function. Additionally, rapamycin, an inhibitor of the mechanistic target of rapamycin downstream of PI3K, markedly hindered axonal regeneration. These findings suggest that Rab5 promotes the axonal regeneration of injured zebrafish Mauthner cells by activating the PI3K signaling pathway.

01 Mar 2025·Current Radiopharmaceuticals

Response of Male Reproductive System Against Ionizing Radiation and Available Radio-protective Agents: Cellular and Molecular Insight

Article

Author: Jheeta, Kuldeep S ; Saini, Priya ; Parashar, Ronit ; Meena, Priyadarshi ; Patel, Dev Dutt ; Kumar, Raj ; Yadav, Sanwar Mal

Background:The reproductive organ, housing spermatogonial stem cells (SSCs), undergoes ongoing division impacted by the irradiation dosage and exposure duration. Within the male reproductive organ, germ stem cells (spermatogonia) and somatic cells (Sertoli and Leydig cells) are present. Lower doses of ionizing (>4-6 Gy) and non-ionizing radiation (radiofrequency and microwave range 900 MHz - 2.45 GHz) may cause sperm-related issues, while higher doses (15 Gy) may affect Leydig cells and testosterone production. Response to radiation varies with age and pubescence. Spermatogonial stem cells, crucial for regenerating the spermatogenic lineage, express molecular markers like Estrogen receptor, FSH (Follicular Stimulating Hormone) receptor, TLR-4 (Toll-like Receptor-4), TLR-5 (Toll-like Receptor-5), FGF2 (Fibroblast Growth Factor-2), KIT (Receptor Tyrosine Kinase), AT-1 (Angiotensin II Type-1 Receptor), LXRs-γ (Liver X Receptor-γ), TNF-β (Tumor Necrosis Factor-β), and PCNA (Proliferating Cell Nuclear Antigen), influencing stem cell activity in testes.Objective:This study aimed to review the various available radioprotective agents and their efficacy in targeting the male reproductive system from the available literature.Result:Various radioprotective herbal/synthetic/microbial/metallic extracts/formulations/ drugs [Septilin, Silymarin, Organic Turmeric, Oestrogen, Melatonin, Febuxostat, SQGD (Semiquinone glucoside derivative), Rapamycin, Entolimod, Zinc, Selenium, etc.] have been investigated up to exposure, but owing to effectiveness issues, they are unable to fulfil the aim to the fullest of restoring male fertility and normal testosterone levels during such eventuality.Conclusion:Further study is needed to optimize these tactics and fill knowledge gaps. Also, the effective components of herbal, synthetic drugs, etc., should be isolated and tested up to clinical levels, paving the way for successful radioprotection and radiomitigation strategies in the male reproductive system.

322

News (Medical) associated with Sirolimus

12 Nov 2024

·www.globenewswire.com

Orchestra BioMed Reports Third Quarter 2024 Financial Results and Provides a Business Update

NEW HOPE, Pa., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Orchestra BioMed Holdings, Inc. (Nasdaq: OBIO, “Orchestra BioMed” or the “Company”), a biomedical innovation company accelerating high-impact technologies to patients through risk-reward sharing partnerships, today reported its third quarter 2024 financial results and provided a business update. "The Orchestra BioMed team is focused on execution of the BACKBEAT global pivotal study alongside our strategic partner, Medtronic. We are excited about the enthusiasm we have seen from the clinical community for the potential of AVIM therapy to provide these typically older, higher risk hypertension patients with a potent, programmable, always on treatment option for managing their blood pressure,” commented David Hochman, Chairman, Chief Executive Officer, and Founder of Orchestra BioMed. “We received FDA approval for and recently started to implement an important amendment to our study protocol that improves patient engagement processes and significantly expands our window for screening and enrollment. We believe this amendment will help us execute the ongoing study and deliver regulatory-submission-ready clinical results to our partners at Medtronic.” Mr. Hochman continued, “In parallel with our work on AVIM therapy, we are continuing to advance our Virtue SAB program with active preparations to initiate coronary pivotal studies. The agenda at this year’s TCT conference highlighted the growing importance of drug-coated balloons in the future of coronary and peripheral interventions for artery disease. We view Virtue SAB as a revolutionary innovation that can elevate this rapidly accelerating therapeutic trend, offering superior drug delivery without the limitations of fragile balloon coatings. Overall, we expect 2025 to be a milestone year for Orchestra BioMed and remain steadfast in our commitment to employ our partnership-enabled business model to deliver innovative solutions that will make a meaningful impact on patient care." Recent Business Highlights: Continued site activation and patient enrollment of the BACKBEAT global pivotal study, in collaboration with Medtronic (NYSE: MDT), evaluating the efficacy and safety of atrioventricular interval modulation (“AVIM”) therapy in hypertensive pacemaker patients.Received FDA approval for and are now implementing an amendment to the BACKBEAT study protocol intended to improve patient engagement processes and expand the window for screening and enrolling patients.Preparing to initiate coronary pivotal studies for Virtue® Sirolimus AngioInfusionTM Balloon (“SAB”).Actively engaged with Terumo in Virtue SAB partnership restructuring discussions.Vivasure Medical Ltd. (“Vivasure”), a strategic holding of Orchestra BioMed, reported positive data from its U.S. IDE PATCH pivotal study at the Transcatheter Cardiovascular Therapeutics (“TCT”) 2024 annual conference demonstrating that its PerQseal® Closure Device achieved closure with very low rates of major vascular complications and rapid times to hemostasis. This data positions PerQseal for potential regulatory approval and commercial launch in 2025. Haemonetics Corporation (NYSE: HAE) has invested over $30 million in Vivasure and has an option to acquire the company. Financial Results for the Third Quarter Ended September 30, 2024 Cash and cash equivalents and Marketable securities totaled $66.9 million as of September 30, 2024. Following the close of Q3 2024, cash and cash equivalents increased in November 2024 by approximately $15 million from the initial draw on our credit facility with Hercules Capital, Inc. Operating cash runway is expected into second half of 2026 based on internal forecast reflecting operating priorities and certain potential future proceeds.Net cash used in operating activities and for the purchase of fixed assets was $13.8 million during the third quarter of 2024, compared with $10.3 million for the third quarter of 2023, with the primary driver of this increase being increased cash outflows for research and development during the third quarter of 2024.Revenue for the third quarter of 2024 was $1.0 million, compared with $0.4 million for the third quarter in 2023. The increase was primarily due to increased recognition of partnership revenues earned under the agreement with Terumo.Research and development expenses for the third quarter of 2024 were $11.6 million, compared with $8.6 million for the same period in 2023. The increase was primarily due to additional costs associated with the ongoing BACKBEAT global pivotal study.Selling, general and administrative expenses for the third quarter of 2024 were $5.7 million, compared with $6.3 million for the third quarter of 2023. The decrease was primarily due to reduced expenses related to stock-based compensation.Net loss for the third quarter of 2024 was $15.4 million, or $0.41 per share, compared with a net loss of $13.3 million, or $0.38 per share, for 2023. Net loss for the third quarter of 2024 included non-cash stock-based compensation expense of $2.4 million as compared with $3.5 million for the same period in 2023. About Orchestra BioMed Orchestra BioMed (Nasdaq: OBIO) is a biomedical innovation company accelerating high-impact technologies to patients through risk-reward sharing partnerships with leading medical device companies. Orchestra BioMed’s partnership-enabled business model focuses on forging strategic collaborations with leading medical device companies to drive successful global commercialization of products it develops. Orchestra BioMed’s lead product candidate is atrioventricular interval modulation (AVIM) therapy (also known as BackBeat Cardiac Neuromodulation Therapy (CNT™)) for the treatment of hypertension, a significant risk factor for death worldwide. Orchestra BioMed is also developing the Virtue® Sirolimus AngioInfusion™ Balloon (SAB) for the treatment of atherosclerotic artery disease, the leading cause of mortality worldwide. Orchestra BioMed has a strategic collaboration with Medtronic, one of the largest medical device companies in the world, for development and commercialization of AVIM therapy for the treatment of hypertension in pacemaker-indicated patients, and a strategic partnership with Terumo, a global leader in medical technology, for development and commercialization of Virtue SAB for the treatment of artery disease. For further information about Orchestra BioMed, please visit www.orchestrabiomed.com, and follow us on LinkedIn. References to Websites and Social Media Platforms References to information included on, or accessible through, websites and social media platforms do not constitute incorporation by reference of the information contained at or available through such websites or social media platforms, and you should not consider such information to be part of this press release. About AVIM Therapy AVIM therapy, also known as BackBeat CNT™, is an investigational therapy compatible with standard dual-chamber pacemakers designed to substantially and persistently lower blood pressure. It has been evaluated in pilot studies in patients with hypertension who are also indicated for a pacemaker. MODERATO II, a double-blind, randomized, pilot study, showed that patients treated with AVIM therapy experienced net reductions of 8.1 mmHg in 24-hour ambulatory systolic blood pressure (aSBP) and 12.3 mmHg in office systolic blood pressure (oSBP) at six months when compared to control patients. The BACKBEAT (BradycArdia paCemaKer with atrioventricular interval modulation for Blood prEssure treAtmenT) global pivotal study will further evaluate the safety and efficacy of AVIM therapy in lowering blood pressure in a similar target population of patients who have been indicated for, and recently implanted with, a dual-chamber cardiac pacemaker. About Virtue SAB Virtue SAB is a patented drug/device combination product candidate in development for the treatment of certain forms of artery disease that is designed to deliver a proprietary, investigational, extended-release formulation of sirolimus, SirolimusEFR™, to the vessel wall during balloon angioplasty without any coating on the balloon surface or the need to leave a stent or other permanent implant in the artery. Virtue SAB demonstrated positive three-year clinical data in coronary ISR in the SABRE study, a multi-center prospective, independent core lab-adjudicated clinical study conducted in Europe. Virtue SAB has been granted Breakthrough Device designation by the FDA for specific indications relating to coronary ISR, coronary small vessel disease and peripheral artery disease below-the-knee. Orchestra BioMed has a strategic partnership with Terumo (Terumo, TSE: 4543), a global leader in medical technology headquartered in Tokyo, Japan, as well as Terumo Medical Corporation, its U.S. subsidiary, to collaborate on the global development and commercialization of Virtue SAB in coronary and peripheral vascular indications. Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements generally are accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements relating to the initiation, enrollment, timing, implementation and design of the Company’s planned and ongoing pivotal trials and reporting of top-line results, realizing the clinical and commercial value of BackBeat CNT and Virtue SAB, the expected runway of the Company’s current cash, cash equivalents, marketable securities the Company’s ability to restructure its partnership agreement with Terumo, the potential safety and efficacy of the Company’s product candidates, regulatory approval Vivasure’s PerQseal® Closure Device and the timing of any acquisition of Vivasure, and the ability of the Company’s partnerships to accelerate clinical development. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of the Company’s management and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as and must not be relied on as a guarantee, an assurance, a prediction, or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and may differ from assumptions. Many actual events and circumstances are beyond the control of the Company. These forward-looking statements are subject to a number of risks and uncertainties, including changes in domestic and foreign business, market, financial, political, and legal conditions; risks related to regulatory approval of the Company’s commercial product candidates and ongoing regulation of the Company’s product candidates, if approved; the timing of, and the Company’s ability to achieve expected regulatory and business milestones; the impact of competitive products and product candidates; and the risk factors discussed under the heading “Item 1A. Risk Factors” in the Company’s annual report on Form 10-K for the year ended December 31, 2023, which was filed with the Securities and Exchange Commission (“SEC”) on March 27, 2024 , and under the heading “Item1A. Risk Factors” in Part II of the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on November 12, 2024. The Company operates in a very competitive and rapidly changing environment. New risks emerge from time to time. Given these risks and uncertainties, the Company cautions against placing undue reliance on these forward-looking statements, which only speak as of the date of this press release. The Company does not plan and undertakes no obligation to update any of the forward-looking statements made herein, except as required by law. ORCHESTRA BIOMED HOLDINGS, INC.Condensed Consolidated Balance Sheets(in thousands, except share and per share data)(Unaudited) September 30, December 31, 2024 2023ASSETS CURRENT ASSETS: Cash and cash equivalents $25,605 $30,559 Marketable securities 41,321 56,968 Strategic investments, current portion — 68 Accounts receivable, net 115 99 Inventory 234 146 Prepaid expenses and other current assets 1,278 1,274 Total current assets 68,553 89,114 Property and equipment, net 1,254 1,279 Right-of-use assets 1,714 1,555 Strategic investments, less current portion 2,495 2,495 Deposits and other assets 1,303 769 TOTAL ASSETS $75,319 $95,212 LIABILITIES AND STOCKHOLDERS’ EQUITY CURRENT LIABILITIES: Accounts payable $4,723 $2,900 Accrued expenses and other liabilities 7,034 5,149 Operating lease liability, current portion 395 649 Deferred revenue, current portion 4,066 2,510 Total current liabilities 16,218 11,208 Deferred revenue, less current portion 11,439 14,923 Operating lease liability, less current portion 1,443 1,038 TOTAL LIABILITIES 29,100 27,169 STOCKHOLDERS’ EQUITY Preferred stock, $0.0001 par value per share; 10,000,000 shares authorized; none issued or outstanding at September 30, 2024 and December 31, 2023. — — Common stock, $0.0001 par value per share; 340,000,000 shares authorized; 37,942,905 and 35,777,412 shares issued and outstanding as of September 30, 2024 and December 31, 2023, respectively. 4 4 Additional paid-in capital 339,840 316,903 Accumulated other comprehensive income (loss) 98 (10)Accumulated deficit (293,723) (248,854)TOTAL STOCKHOLDERS’ EQUITY 46,219 68,043 TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY $75,319 $95,212 ORCHESTRA BIOMED HOLDINGS, INC.Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(Unaudited) Three Months Ended September 30, 2024 2023 Revenue: Partnership revenue $803 $271 Product revenue 184 148 Total revenue 987 419 Expenses: Cost of product revenues 68 41 Research and development 11,595 8,558 Selling, general and administrative 5,666 6,344 Total expenses 17,329 14,943 Loss from operations (16,342) (14,524) Other income (expense): Interest income, net 916 915 Gain (loss) on fair value of strategic investments — 293 Total other income 916 1,208 Net loss $(15,426) $(13,316) Net loss per share Basic and diluted $(0.41) $(0.38) Weighted-average shares used in computing net loss per share, basic and diluted 37,621,495 35,243,598 Comprehensive loss Net loss $(15,426) $(13,316) Unrealized gain (loss) on marketable securities 121 19 Comprehensive loss $(15,305) $(13,297)

Financial StatementBreakthrough TherapyClinical Result

12 Nov 2024

·www.globenewswire.com

Biodexa Pharmaceuticals PLC (“Biodexa” or the “Company”) Regains Compliance For Continued Listing on Nasdaq

November 12, 2024 Biodexa Pharmaceuticals PLC (“Biodexa” or the “Company”) Regains Compliance For Continued Listing on Nasdaq Biodexa Pharmaceuticals PLC, (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs announces that the Company has been formally notified that the Nasdaq Hearings Panel (the “Panel”) of the Nasdaq Stock Market LLC (“Nasdaq”) determined that the Company has regained compliance with the $1.00 minimum bid price requirement for continued listing on Nasdaq as set forth in Nasdaq Listing Rule 5550(a)(2) (the “Minimum Bid Price Requirement”). As previously disclosed, the Company received notice from Nasdaq on August 27, 2024, informing the Company that it was not in compliance with the Minimum Bid Price Requirement. On October 14, 2024, the Panel granted the Company’s request for an exception to demonstrate compliance with the Minimum Bid Price Requirement for continued listing through October 31, 2024. The Company remains subject to a discretionary panel monitor through February 24, 2025, and is required to provide prompt notification during this exception period of any significant events that occur during this time that may affect the Company’s compliance with Nasdaq requirements. About Biodexa Pharmaceuticals PLC Biodexa Pharmaceuticals PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs. The Company’s lead development programs include eRapa, under development for Familial Adenomatous Polyposis and Non-Muscle Invasive Blader Cancer; tolimidone, under development for the treatment of type 1 diabetes; and MTX110, which is being studied in aggressive rare/orphan brain cancer indications. eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorigenesis. Tolimidone is an orally delivered, potent and selective inhibitor of Lyn kinase. Lyn is a member of the Src family of protein tyrosine kinases, which is mainly expressed in hematopoietic cells, in neural tissues, liver, and adipose tissue. Tolimidone demonstrates glycaemic control via insulin sensitization in animal models of diabetes and has the potential to become a first in class blood glucose modulating agent. MTX110 is a solubilized formulation of the histone deacetylase (HDAC) inhibitor, panobinostat. This proprietary formulation enables delivery of the product via convection-enhanced delivery (CED) at chemotherapeutic doses directly to the site of the tumor, by-passing the blood-brain barrier and potentially avoiding systemic toxicity. Biodexa is supported by three proprietary drug delivery technologies focused on improving the bio-delivery and bio-distribution of medicines. Biodexa’s headquarters and R&D facility is in Cardiff, UK. For more information visit www.biodexapharma.com. Forward-Looking StatementsCertain statements in this announcement may constitute “forward-looking statements” within the meaning of legislation in the United Kingdom and/or United States. Such statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and are based on management’s belief or interpretation. All statements contained in this announcement that do not relate to matters of historical fact should be considered forward-looking statements. In certain cases, forward-looking statements can be identified by the use of words such as “plans”, “expects” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might” or “will be taken”, “occur” or “be achieved.” Forward-looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability of the Company to control or predict, that may cause their actual results, performance or achievements to be materially different from those expressed or implied thereby, and are developed based on assumptions about such risks, uncertainties and other factors set out herein. Reference should be made to those documents that Biodexa shall file from time to time or announcements that may be made by Biodexa in accordance with the rules and regulations promulgated by the SEC, which contain and identify other important factors that could cause actual results to differ materially from those contained in any projections or forward-looking statements. These forward-looking statements speak only as of the date of this announcement. All subsequent written and oral forward-looking statements by or concerning Biodexa are expressly qualified in their entirety by the cautionary statements above. Except as may be required under relevant laws in the United States, Biodexa does not undertake any obligation to publicly update or revise any forward-looking statements because of new information, future events or events otherwise arising.

11 Nov 2024

·www.prnewswire.com

UPSHER-SMITH PRESENTS EXPANDED RARE DISEASE PORTFOLIO AT 2024 CHILD NEUROLOGY SOCIETY ANNUAL MEETING

Visit Our Booths to Learn More about the Company's Latest Product Offerings and Promise of Support™ Program MAPLE GROVE, Minn., Nov. 11, 2024 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced that it will attend the 2024 Child Neurology Society (CNS) Annual Meeting in San Diego, CA from November 11-14, 2024. Representatives from Upsher-Smith will be at our booths to speak with clinicians about its expanding portfolio of medications including newly acquired VIGAFYDE™ (vigabatrin) Oral Solution and recently launched TORPENZ™ (everolimus) Tablets, which became available in August 2024. VIGAFYDE™ is the first and only ready-to-use vigabatrin oral solution and represents a welcome expansion of options for the treatment of infantile spasms as the first new drug application approved for this condition in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ offers patients and caregivers a new option in liquid vigabatrin that does not require reconstitution by the caregiver. VIGAFYDE™, which became available to patients in September 2024, is indicated as monotherapy for the treatment of infantile spasms in pediatric patients 1 month to 2 years, where the potential benefits outweigh the potential risk of vision loss. TORPENZ™, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC), is the only everolimus backed by the Upsher-Smith Promise of Support™ Program to deliver reliable and consistent support to patients, caregivers, and healthcare providers in the TSC community. TORPENZ™ is indicated for the treatment of adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery and is also indicated in adult and pediatric patients aged 1 year and older with TSC for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected. "Since the successful launch of VIGADRONE® (vigabatrin) for Oral Solution to the specialty pharmacy channel in 2018, we have broadened our offerings to include VIGADRONE® (vigabatrin) Tablets, TORPENZ™ Tablets, and now VIGAFYDE™ ready-to-use solution," said Jim Maahs, Head of Commercial, Upsher-Smith. "As part of our commitment to supporting the rare disease community, Upsher-Smith is dedicated to not only maintaining, but enhancing the support and resources available to patients, caregivers, and healthcare practitioners. We encourage attendees to stop by our booths during this year's CNS meeting to learn more." About Upsher-Smith's Promise of Support™ Program Central to Upsher-Smith's commitment to rare disease communities is its Promise of Support™ Program, ensuring comprehensive assistance for patients, caregivers, and healthcare providers throughout the treatment journey. For nearly a decade, the program has offered resources for uninterrupted medication supply, prior authorization support, patient education materials, collaboration with a leading specialty pharmacy partner, and streamlined prescribing and enrollment procedures. It also includes copay assistance for eligible patients to help lessen the financial burden on patients and their families. To learn more about Upsher-Smith, its products, and the Promise of Support™ Program visit: . Vigabatrin REMS All vigabatrin products are governed by a Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA. The Vigabatrin REMS ensures that patients and healthcare providers make informed risk-benefit decisions about its use. Healthcare providers must be enrolled in the Vigabatrin REMS to prescribe these products, and patients must be enrolled to receive them. IMPORTANT SAFETY INFORMATION FOR VIGAFYDE™ (vigabatrin) What is the most important information I should know about VIGAFYDE? VIGAFYDE can cause serious side effects, including: Permanent vision loss: VIGAFYDE can damage the vision of anyone who takes it. Some babies can have severe loss, particularly to their ability to see to the side when they look straight ahead (peripheral vision). With severe vision loss, your baby may only be able to see things straight in front of them (sometimes called "tunnel vision"). Your baby also may have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if your baby might not be seeing as well as before starting VIGAFYDE or is acting differently than normal. Vision loss in babies: Because of the risk of vision loss, VIGAFYDE is used in babies 1 month to 2 years of age with infantile spasms only when you and your healthcare provider decide that the possible benefits of VIGAFYDE are more important than the risks. Parents or caregivers are not likely to recognize the symptoms of vision loss in babies until it is severe. Healthcare providers may not find vision loss in babies until it is severe. It is difficult to test vision in babies, but, to the extent possible, all babies should have their vision tested before starting VIGAFYDE or within 4 weeks after starting VIGAFYDE, and every 3 months after that until VIGAFYDE is stopped. Your baby also should have a vision test about 3 to 6 months after VIGAFYDE is stopped. Your healthcare provider will determine if your baby can be tested. If your baby cannot be tested, your healthcare provider may continue prescribing VIGAFYDE, but they will not be able to watch for any vision loss. Even if your baby's vision seems fine, it is important to get regular vision tests because damage can happen before your baby acts differently. Even these regular vision exams may not show the damage to your baby's vision before it is severe and permanent. All babies who take VIGAFYDE: Are at risk for permanent vision loss with any amount of VIGAFYDE. The risk of vision loss may increase with higher doses of VIGAFYDE, and the longer it is taken. It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting VIGAFYDE or any time during treatment. It may even happen after treatment has stopped. Because VIGAFYDE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called Vigabatrin Risk Evaluation and Mitigation Strategy (REMS). Your healthcare provider will explain the details of the Vigabatrin REMS to you. For more information, go to or call 1-866-244-8175. Magnetic resonance imaging (MRI) changes in babies with infantile spasms (IS): brain pictures taken by MRI show changes in some babies after they are given VIGAFYDE. It is not known if these changes are harmful. What are the possible side effects of VIGAFYDE? VIGAFYDE can cause serious side effects, including sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable) and weight gain without swelling. Serious side effects that happen in adults who take vigabatrin and are not known if they will happen in babies are low red blood cell counts, nerve problems, and swelling. Tell your baby's healthcare provider right away if your baby's seizures get worse. The most common side effects of VIGAFYDE in babies include sleepiness, ear infection, swelling of the bronchial tubes, and irritability. Tell your healthcare provider if your baby has any side effect that bothers them or that does not go away. These are not all the possible side effects of VIGAFYDE. Do not stop taking VIGAFYDE suddenly. This can cause serious problems. Talk to your healthcare provider for directions on how to stop treatment. What should I tell my healthcare provider before starting VIGAFYDE? Before giving VIGAFYDE to your baby for IS, inform your healthcare provider about all your baby's medical conditions. This includes whether your baby has ever had an allergic reaction to vigabatrin, such as hives, itching, or trouble breathing, as well as any history of vision problems, or kidney issues. Tell your healthcare provider about all the medicines your baby takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Interactions between VIGAFYDE and other medicines could lead to side effects. You may report side effects to the FDA at or call 1-800-FDA-1088 or Pyros at 1-855-406-1010. Please see full Prescribing Information, including BOXED WARNING, Medication Guide, and Instructions for Use for additional Important Safety Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT TORPENZ™ ( everolimus) TABLETS in Tuberous Sclerosis Complex? Do not take TORPENZ if you have had an allergic reaction to everolimus. Talk to your healthcare provider before taking TORPENZ if you are allergic to sirolimus or temsirolimus. Ask your provider if you do not know. TORPENZ can cause serious side effects, including: Lung or breathing problems. In some people, these may be severe and can be life threatening. Tell your healthcare provider right away if you have new or worsening cough, shortness of breath, chest pain, difficulty breathing or wheezing. Higher likelihood of infection such as pneumonia, or a bacterial, fungal, or viral infection. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some adults and children, these infections may be severe and can be life threatening. You may need to be treated as soon as possible. Tell your healthcare provider right away if you have a temperature of 100.5˚F or above, chills, or do not feel well. Symptoms of infection may include: Severe allergic reactions. Get medical help right away if you have signs of an allergic reaction, including rash, itching, hives, flushing, trouble breathing or swallowing, chest pain or dizziness. Possible increased risk for a type of allergic reaction called angioedema in people who take an Angiotensin-Converting Enzyme (ACE) inhibitor with TORPENZ. Talk with your healthcare provider before taking TORPENZ if you are not sure if you take an ACE inhibitor. Get medical help right away if you have trouble breathing or develop swelling of your tongue, mouth, or throat during treatment with TORPENZ. Mouth ulcers and sores. Mouth ulcers and sores are common during treatment with TORPENZ but can also be severe. When you start TORPENZ, your healthcare provider may tell you to also start a prescription mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your provider's instructions on how to use this mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your provider. Kidney failure. In some people, this may be severe and can be life threatening. Your healthcare provider should check your kidney function before and during treatment. Wound healing problems. Wounds may not heal properly during TORPENZ treatment. Tell your healthcare provider if you plan to have any surgery before starting or during treatment. You should stop TORPENZ at least 1 week before planned surgery. Your provider should tell you when to start taking TORPENZ again after surgery. Increased blood sugar and fat (cholesterol and triglyceride) levels in the blood. Your healthcare provider should check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment. Decreased blood cell counts. TORPENZ can cause you to have decreased red blood cells, white blood cells and platelets. Your healthcare provider should check your blood cell counts before you start and during treatment. Worsening side effects from radiation treatment that can sometimes be severe. Tell your healthcare provider if you have had or are planning to receive radiation therapy. Before taking TORPENZ, tell your healthcare provider about all of your medical conditions, including if you: Have or have had kidney or liver problems, have diabetes or high blood sugar, have high blood cholesterol, have any infections, or previously had hepatitis B. Are scheduled to receive any vaccinations. You should not receive a "live vaccine" or be around people who have recently received a "live vaccine" during your treatment with TORPENZ. If you are not sure about the type of immunization or vaccine, ask your provider. For children, work with your provider to complete the recommended childhood series of vaccines before your child starts TORPENZ. Are pregnant, can become pregnant, or have a partner who can become pregnant. TORPENZ can cause harm to your unborn baby. For females who are able to become pregnant, your provider will give you a pregnancy test before you start TORPENZ. Use effective birth control during treatment and for 8 weeks after your last dose of TORPENZ. Males with a female partner should use effective birth control during treatment and for 4 weeks after your last dose of TORPENZ. Are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of TORPENZ. Are planning to have or have had a recent surgery. You should stop taking TORPENZ at least 1 week before planned surgery. Have received or are planning to receive radiation therapy. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TORPENZ with some medicines can cause serious side effects. Keep a list of medications you take, and show it to your healthcare provider and pharmacist when you get a new medicine. Especially tell your provider if you take: St. John's Wort (Hypericum perforatum) Medicine for fungal or bacterial infections, tuberculosis, seizures, HIV-AIDS, heart conditions or high blood pressure, including ACE inhibitors Medicines that weaken your immune system (your body's ability to fight infections and other problems) Ask your provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your provider might need to prescribe a different medicine or your dose of TORPENZ may need to be changed. Tell your provider before you start any new medicine. You should not drink grapefruit juice or eat grapefruit during your treatment with TORPENZ. It may make the amount of TORPENZ in your blood increase to a harmful level. The most common side effect of TORPENZ in people who have SEGA or renal angiomyolipoma include respiratory tract infection. Other side effects that may occur with TORPENZ: Absence of menstrual periods (menstruation). Tell your healthcare provider if this happens. TORPENZ may affect fertility and may affect your ability to become pregnant if you are female or your ability to father a child if you are male. Talk to your provider if this is a concern. Tell your healthcare provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of TORPENZ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You can also visit TORPENZ.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. WHAT ARE TORPENZ (everolimus) TABLETS? TORPENZ is a prescription medicine used to treat the following types of benign (non-cancerous) tumors that are seen with a genetic condition called tuberous sclerosis complex (TSC): Brain tumors called subependymal giant cell astrocytoma (SEGA), when the tumor cannot be removed completely by surgery, in adults and children 1 year and older. Kidney tumors called angiomyolipoma, when the tumor does not require surgery right away, in adults. For additional information, including safety information, for non-TSC indications, see the full Prescribing Information, including Patient Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGADRONE® (vigabatrin)? Because VIGADRONE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your healthcare provider will explain the details of this Program to you. VIGADRONE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, you may only be able to see things straight in front of you (sometimes called "tunnel vision"). You may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if you (or your child): might not be seeing as well as before starting VIGADRONE; start to trip, bump into things, or are more clumsy than usual; are surprised by people or things coming in front of you that seem to come out of nowhere; or if your baby is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your (or your child's) vision before or within 4 weeks after starting VIGADRONE, and at least every 3 months during treatment until VIGADRONE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGADRONE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGADRONE, but they do allow VIGADRONE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in patients, until it is severe. If you do not have these vision tests regularly, your healthcare provider may stop prescribing VIGADRONE for you (or your child). Some people are not able to complete vision testing. If vision testing cannot be done, your healthcare provider may continue prescribing VIGADRONE, but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGADRONE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in autopsy examination of patients treated with vigabatrin. Risk of suicidal thoughts or actions. Like other antiepileptic drugs, VIGADRONE may cause suicidal thoughts and actions in some people (about 1 in 500 people). Call a healthcare provider right away if you (or your child) have any symptoms, especially sudden changes in mood, behaviors, thoughts or feelings, and especially if they are new, worse, or worry you. Do not stop VIGADRONE without first talking to a healthcare provider. Stopping VIGADRONE suddenly can cause seizures that will not stop. VIGADRONE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, nerve problems, weight gain, and swelling. Because VIGADRONE causes sleepiness and tiredness, do not drive, operate machinery, or perform any hazardous task, unless it is decided that these things can be done safely. VIGADRONE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGADRONE, tell your doctor about all of your (or your child's) medical conditions including depression, mood problems, suicidal thoughts or behavior, any allergic reaction to VIGADRONE, vision problems, kidney problems, low red blood cell counts (anemia), and any nervous or mental illnesses. Tell your doctor about all the medicines you (or your child) take. If you are breastfeeding or plan to breastfeed, VIGADRONE can pass into breast milk and may harm your baby. Breastfeeding is not recommended. If you are pregnant or plan to become pregnant, VIGADRONE can cause harm to your unborn baby. You and your healthcare provider will have to decide if you should take VIGADRONE while you are pregnant. The most common side effects of VIGADRONE in adults include: blurred vision, sleepiness, dizziness, problems walking or feeling uncoordinated, shaking (tremor) and tiredness. The most common side effect of VIGADRONE in children 3 to 16 years of age is weight gain. Also expect side effects like those seen in adults. The most common side effects of VIGADRONE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if you or your child have any side effect that bothers you or that does not go away. This is the most important information to know about VIGADRONE, but it is not all the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGADRONE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGADRONE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT IS VIGADRONE? VIGADRONE® (vigabatrin) is a prescription medicine used to treat: Infantile Spasms (IS) in patients 1 month to 2 years of age, if you and your healthcare provider decide the possible benefits of taking VIGADRONE are more important than the possible risk of vision loss. Refractory Complex Partial Seizures (CPS) in adults and children 2 years and older with refractory complex partial seizures (CPS) along with other treatments if: The CPS do not respond well enough to several other treatments, and You and your healthcare provider decide the possible benefit of taking VIGADRONE is more important than the risk of vision loss. VIGADRONE should not be the first medicine used to treat CPS. About Upsher-Smith Upsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit . About Bora Founded in 2007, Bora Pharmaceutical Co., Ltd. ("Bora" or "the Company", 6472.TW) now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit . TORPENZ, VIGADRONE, and Promise of Support are trademarks of Upsher-Smith Laboratories, LLC. VIGAFYDE is a trademark of Pyros Pharmaceuticals, Inc. SOURCE Upsher-Smith Laboratories, LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

100 Deals associated with Sirolimus

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KEGG	Wiki	ATC	Drug Bank
D00753	Sirolimus	L04AA10 S01XA23	DB00877

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Vascular Malformations	JP	Nobelpharma Co., Ltd.	18 Jan 2024
Vascular Neoplasms	JP	Nobelpharma Co., Ltd.	18 Jan 2024
Angiofibroma	EU	Pfizer Inc.	15 May 2023
Angiofibroma	IS	Pfizer Inc.	15 May 2023
Angiofibroma	LI	Pfizer Inc.	15 May 2023
Angiofibroma	NO	Pfizer Inc.	15 May 2023
Tuberous Sclerosis	CN	Nobelpharma Co., Ltd.	28 Mar 2023
Angiofibroma associated with tuberous sclerosis	US	Nobelpharma Co., Ltd.	22 Mar 2022
Lymphangiectasis	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioma	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Osteolysis, Essential	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioleiomyomatosis	IS	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	EU	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	NO	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	LI	Pfizer Europe MA EEIG	13 Mar 2001
Renal transplant rejection	US	PF Prism CV	15 Sep 1999

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Focal Cortical Dysplasia of Taylor	Phase 3	JP	Nobelpharma Co., Ltd.	-
Liver transplant rejection	Preclinical	CZ	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	PT	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	DE	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	CH	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	IT	Wyeth AB	01 Dec 2002
Allograft Rejection	Discovery	-	Wyeth AB	01 Aug 2002
Graft vs Host Disease	Discovery	US	Wyeth AB	20 May 2002
Graft vs Host Disease	Discovery	CA	Wyeth AB	20 May 2002
Kidney Diseases	Discovery	-	Wyeth AB	01 Jul 2000

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04339101 (1043, ASH2024)	Phase 2	Myelofibrosis \| acute leukemia \| Myelodysplastic Syndromes HGF \| IL17 \| TNFaR ... View more	59	Itacitinib + Tacrolimus/Sirolimus	ucdsalegrh(bezdscwizj) = hjwasogenv wqbbjvuubs (mkosaonyqg, 41 - 66) View more	Positive	09 Dec 2024
NCT04339101 (1043, ASH2024)	Phase 2		59	Tacrolimus/Sirolimus	ucdsalegrh(bezdscwizj) = xuwqypgrvm wqbbjvuubs (mkosaonyqg )	Positive	09 Dec 2024
ChiCTR1900020657 (1187Sirolimus, ASH2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic First line	-	HD-DXM plus sirolimus	tnmlakunsl(yebsbaliyu) = iheqnewvnl wdniejjzrs (zdmjmvuuav ) View more	Positive	07 Dec 2024
ChiCTR1900020657 (1187Sirolimus, ASH2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic First line	-	HD-DXM monotherapy	tnmlakunsl(yebsbaliyu) = lfcyazjplk wdniejjzrs (zdmjmvuuav ) View more	Positive	07 Dec 2024
NCT03095703 (CTgov)	Phase 2	Adenomatous Polyposis Coli	4	Sirolimus	jujwtdfxtq(hvhhkgiwon) = ofwnigktdd zrtzfwbtyi (yozqtnlyft, rcmsqvwtzq - sldjbhtist) View more	-	24 Oct 2024
NCT04348292 (CTgov)	Phase 1	Resectable Lung Non-Small Cell Carcinoma \| Non-small cell lung cancer stage IIIA	3	Sirolimus+Durvalumab	rxfumhoehf(zmbswicdzt) = ienjdtbtrz tevnzxkxpq (lfzzehprde, xgpdpfrhyz - yrgpkcusxy) View more	-	22 Aug 2024
NCT03077542 (CTgov)	Phase 1/2	Anemia, Sickle Cell	57	Filgrastim	cmcdbpashe(sxobhdxcux) = dnljvvhshd wxpocmreka (mcxsipkyzv, znfndihhxl - ajfnuolwrw) View more	-	07 Aug 2024
NCT04393454 (CTgov)	Phase 2	Metastatic Solid Tumor \| Mismatch repair-deficient Solid Tumors \| Microsatellite instability-high cancer	6	Sirolimus 2mg Tablet	hguwxpxgtl(mtauciqnfy) = esqryzekmw waphlvkpeq (vwdgiaknul, bdjirxrkyl - lofsqzoivl) View more	-	25 Jul 2024
NCT04230499 (Company_Website) Manual	Phase 2	Adenomatous Polyposis Coli	30	eRapa	(cqbzmwicut) = wriqywjchx upbdqjndip (wmxrnclotg ) View more	Positive	24 Jun 2024
NCT04230499 (Company_Website) Manual	Phase 2	Adenomatous Polyposis Coli	30	eRapa (0.5mg every other day)	(cqbzmwicut) = knqmgndyyn upbdqjndip (wmxrnclotg )	Positive	24 Jun 2024
NCT04339062 (CONTRAC, CTgov)	Phase 1/2	Advanced Skin Squamous Cell Carcinoma \| Advanced cancer \| Cutaneous Squamous Cell Carcinoma	12	Cemiplimab (Cohort 1 Cemiplimab)	pmaigdcaiv(bvufrwzygz) = rjneipcgsz cxyehddgox (lrmgumlvyg, ktuenyvsbw - lxkodisugl) View more	-	12 Jun 2024
NCT04339062 (CONTRAC, CTgov)	Phase 1/2		12	Prednisone+Cemiplimab+Sirolimus+everolimus (Cohort 2 Cemiplimab + Everolimus/Sirolimus + Prednisone)	vfdvxgufju(uezmbczlxz) = ucvguhhfrg bukflmgmzi (prbjzkxdgp, napooleydd - klcgsuchlu) View more	-	12 Jun 2024
NCT02584647 (phase II study, ASCO2024)	Phase 2	Neurofibrosarcoma	-	Pexidartinib 1000mg + Sirolimus 2mg	(dbltdfpbwz) = leukopenia and rash wwyowxguml (jvdbaobyoy )	Negative	24 May 2024
NCT04230499 (Corporate Publications) Manual	Phase 2	Adenomatous Polyposis Coli	30	eRapa	(euxmzbkfrv) = dzfguataoe eaaavvyhkq (hlvzihefnu ) View more	Positive	21 May 2024

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