The hypothesize of this research is that rapamycin is effective and well-tolerated in teenagers with familial adenomatous polyposis (FAP). Rapamycin could be effective in blocking the formation of adenomas and/or their evolution by decreasing their size and number. Researchers aim to assess the safety profile of rapamycin in FAP adolescents using a 2 low dose regimen.

Start Date01 Aug 2025

Sponsor / Collaborator

Toulouse University Hospital

NCT06084780

/ Not yet recruitingPhase 2IIT

Prospective Case Series of Intestinal and Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE)

The goal of this prospective phase 2 study is to assess the efficacy and safety of intestinal or multivisceral transplantation for participants with PMP not amenable to other curative-intent treatments. Participants will undergo intestinal/multivisceral transplantation. Participants will be followed for 12 months to assess efficacy and safety.

Start Date01 Jun 2025

Sponsor / Collaborator

The Case Comprehensive Cancer Center

NCT06799195

/ Not yet recruitingPhase 2IIT

A Phase II Randomized Trial to Optimize GVHD Prophylaxis After Allogeneic Hematopoietic Cell Transplantation in Older Adults With Hematological Malignancies: the PROMISE Trial

This study will compare post-transplant health-related quality of life following the use of standard versus attenuated dose of post-transplant cyclophosphamide in addition to two-drug graft-versus-host disease (GVHD) prophylaxis among recipients of allogeneic hematopoietic stem cell transplant.

Start Date01 May 2025

Sponsor / Collaborator

University of Nebraska

100 Clinical Results associated with Sirolimus

100 Translational Medicine associated with Sirolimus

100 Patents (Medical) associated with Sirolimus

32,945

Literatures (Medical) associated with Sirolimus

31 Dec 2025·Cancer Biology & Therapy

The RNA-binding protein ELAVL1 promotes Beclin1-mediated cellular autophagy and thus endometrial cancer development by affecting LncRNA-neat stability

Article

Author: Luo, Yanlu ; Fan, Jiangtao ; Zhong, Xueyan ; Sun, Xinzhao

Our study aims to investigate the roles of embryonic lethal abnormal vision-like 1 (ELAVL1) and long non-coding RNA (LncRNA) NEAT1 in endometrial cancer (EC), focusing on their underlying molecular mechanisms.We obtained EC cell lines (HEC-1A, Ishikawa, RL95-2, HEC-1B, and AN3CA) from ATCC. We used siRNAs (si-ELAVL1#1 and si-ELAVL1#2) and overexpression RNAs (OE ELAVL1 and OE-NEAT1) for knockdown or overexpression of ELAVL1 and LncRNA NEAT1. We also employed 3-MA (5mM) or rapamycin (100µM) to inhibit or promote autophagy. Moreover, we conducted RNA immunoprecipitation (RIP) assays to confirm the interaction between LncRNA NEAT1 and ELAVL1. Cell Counting Kit-8 (CCK-8) and transwell assays were utilized to assess cell proliferation and migration. Additionally, we measured the expression of ELAVL1 and Beclin1 through Western blotting and RT-qPCR.ELAVL1 was found to be highly expressed in EC. Furthermore, ELAVL1 promoted the proliferation, invasion, and migration of EC cells through the regulation of Beclin1-related pathways. RIP assays revealed a direct interaction between LncRNA NEAT1 and ELAVL1, with ELAVL1 stabilizing LncRNA NEAT1 mRNA in EC cells. Additionally, we observed that ELAVL1 influenced EC cell proliferation, invasion, and migration through the regulation of LncRNA NEAT1-mediated regulation of Beclin1 expression. Moreover, in an animal study, we determined that ELAVL1 influenced endometrial cancer tumor growth through its interaction with LncRNA NEAT1, which mediated Beclin1 expression in vivo.In summary, our study showed that ELAVL1 regulated the malignant behavior of endometrial cancer cells through the modulation of LncRNA NEAT1-mediated regulation of Beclin1 expression.

01 Dec 2025·Journal of Clinical Immunology

Report of the Italian Cohort with Activated Phosphoinositide 3-Kinase δ Syndrome in the Target Therapy Era

Article

Author: Montin, Davide ; De Rosa, Antonio ; Rivalta, Beatrice ; Ricci, Silvia ; Trizzino, Antonino ; Marinoni, Maddalena ; Moratti, Mattia ; Zecca, Marco ; Tommasini, Alberto ; Marzollo, Antonio ; Barzaghi, Federica ; Baselli, Lucia Augusta ; Badolato, Raffaele ; Cancrini, Caterina ; Conti, Francesca ; Lougaris, Vassilios ; Lodi, Lorenzo ; Giardino, Giuliana ; Pignata, Claudio ; Panza, Giuseppina ; Costagliola, Giorgio ; Martire, Baldassarre ; Milito, Cinzia ; Chinello, Matteo

BACKGROUNDActivated Phosphoinositide 3-Kinase (PI3K) δ Syndrome (APDS), an inborn error of immunity due to upregulation of the PI3K pathway, leads to recurrent infections and immune dysregulation (lymphoproliferation and autoimmunity).METHODSClinical and genetic data of 28 APDS patients from 25 unrelated families were collected from fifteen Italian centers.RESULTSPatients were genetically confirmed with APDS-1 (n = 20) or APDS-2 (n = 8), with pathogenic mutations in the PIK3CD or PIK3R1 genes. The median age at diagnosis was 15.5 years, with a median follow-up of 74 months (range 6-384). The main presenting symptoms were respiratory tract infections alone (57%) or associated with lymphoproliferation (17%). Later, non-clonal lymphoproliferation was the leading clinical sign (86%), followed by respiratory infections (79%) and gastrointestinal complications (43%). Malignant lymphoproliferative disorders, all EBV-encoding RNA (EBER)-positive at the histological analysis, occurred in 14% of patients aged 17-19 years, highlighting the role of EBV in lymphomagenesis in this disorder. Diffuse large B-cell lymphoma was the most frequent. Immunological work-up revealed combined T/B cell abnormalities in most patients. Treatment strategies included immunosuppression and PI3K/Akt/mTOR inhibitor therapy. Rapamycin, employed in 36% of patients, showed efficacy in controlling lymphoproliferation, while selective PI3Kδ inhibitor leniolisib, administered in 32% of patients, was beneficial on both infections and immune dysregulation. Additionally, three patients underwent successful HSCT due to recurrent infections despite ongoing prophylaxis or lymphoproliferation poorly responsive to Rapamycin.CONCLUSIONSThis study underscores the clinical heterogeneity and challenging diagnosis of APDS, highlighting the importance of multidisciplinary management tailored to individual needs and further supporting leniolisib efficacy.

01 Dec 2025·Molecular Biology Reports

Rapamycin and Niacin combination induces apoptosis and cell cycle arrest through autophagy activation on acute myeloid leukemia cells

Article

Author: Akçok, İsmail ; Subay, Lale Beril ; Gencer Akçok, Emel Başak

BACKGROUNDAcute myeloid leukemia (AML) is a heterogeneous hematological malignancy caused by disorders in stem cell differentiation and excessive proliferation resulting in clonal expansion of dysfunctional cells called myeloid blasts. The combination of chemotherapeutic agents with natural product-based molecules is promising in the treatment of AML. In this study, we aim to investigate the anti-cancer effect of Rapamycin and Niacin combination on THP-1 and NB4 AML cell lines.METHODS AND RESULTSThe anti-proliferative effects of Rapamycin and Niacin were determined by MTT cell viability assay in a dose- and time-dependent manner. The combination indexes were calculated by isobologram analysis. Furthermore, apoptosis was investigated by Annexin-V/Propidium Iodide(PI) double staining and cell cycle distribution was measured by PI staining. The expression levels of autophagy-related proteins were detected by western blotting. The combination of Rapamycin and Niacin synergistically decreased cell viability of AML cell lines. The combination treatment induced the apoptotic cell population of THP-1 and NB4 by 4.9-fold and 7.3-fold, respectively. In THP-1 cells, the cell cycle was arrested at the G2/M phase by 10% whereas the NB4 cells were accumulated at the G0/G1 phase. The combination treatment decreased Akt and p-Akt expression. Besides, the ATG7 expression was reduced by combination treatment on THP-1 cells. Similarly, the ATG5 level was downregulated in NB4 cells. The level of LC3B-II/LC3B-I, which is an indicator of autophagy flux, was upregulated in THP-1 and NB4 cells.CONCLUSIONAlthough further studies are required, the combination of Rapamycin and Niacin combats cell proliferation by inducing cellular apoptosis, cell cycle arrest and autophagy activation.

352

News (Medical) associated with Sirolimus

13 Mar 2025

·investors.quoinpharma.com

Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results

ASHBURN, Va., March 13, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced recent corporate accomplishments and provided an update on its fourth quarter and full-year 2024 progress. Quoin CEO Michael Myers said, "2024 was without a doubt the most significant year in Quoin’s history as our team delivered tremendous progress in clinical, regulatory and manufacturing settings. This progress was particularly exemplified in the clinical development of QRX003, our product candidate for Netherton Syndrome, and we are encouraged by the positive data our ongoing studies are generating. The significant improvements observed across multiple clinical endpoints, including a dramatic reduction in disease severity, tangible skin healing and barrier restoration as well as highly positive patient-reported outcomes, have provided further evidence of QRX003’s potential to address the underlying pathology of Netherton Syndrome. Our recently announced FDA-cleared ‘whole-body’ clinical study will represent the most extensive evaluation of QRX003 to date, and we believe will generate a very robust and comprehensive safety and efficacy data set that will become a central component of our regulatory approval submission. As a company, we remain steadfast in our mission to develop the first FDA-approved treatment for Netherton Syndrome, a disease with no approved therapies and a profound impact on patients and families. Additionally, our recent patent filings for novel topical rapamycin formulations mark an important step in expanding our pipeline to address additional rare dermatological diseases. With multiple clinical trials underway, and continued pipeline expansion, 2025 is already shaping up to become a highly pivotal year for Quoin.” Recent AccomplishmentsOn March 4, 2025, Quoin announced it had filed U.S. and international patent applications for novel topical rapamycin formulations targeting microcystic lymphatic malformations, venous malformations and angiofibromas. The products are being developed using Quoin’s in-licensed proprietary Invisicare® delivery technology, further expanding Quoin’s rare disease pipeline. A week earlier, Quoin announced highly positive whole body clinical data from its ongoing pediatric Netherton Syndrome study. The first patient to receive whole-body application of QRX003 showed transformational improvement after just two weeks, with clear visual evidence of skin healing. Crucially also, the patient was able to fully discontinue previously required medications, including all antihistamines, glucocorticoids, antivirals, and has not needed any antibiotic treatment. Furthermore, the patient is experiencing zero nightly sleep disturbances for the first time in her life. No adverse events were reported. Earlier in February, Quoin launched its ‘NETHERTON NOW’ awareness campaign, a multi-channel initiative designed to raise awareness of Netherton Syndrome and its impact on patients and families. The company introduced a dedicated website, providing educational resources and support for the Netherton community. In January, Quoin released further clinical evidence from its open label study supporting QRX003’s effectiveness. Data showed that within four weeks of discontinuation of treatment all positive clinical improvements observed after 12 weeks of treatment were completely reversed, reinforcing QRX003’s mechanism of action as a competitive broad spectrum serine protease inhibitor and the need for ongoing chronic treatment to maintain therapeutic benefits. Earlier in January, Quoin announced additional positive clinical data from its ongoing open-label Netherton Syndrome study. The first subject to complete 12 weeks of twice-daily dosing with QRX003 demonstrated sustained clinical improvement across all measured endpoints. No adverse events were reported throughout the study. Photographic evidence clearly showing direct evidence of skin healing may be accessed on Quoin’s website. Fourth Quarter 2024 HighlightsOn Dec. 20, 2024, Quoin announced the successful completion of a $6.8 million public offering, bolstering the company’s financial position to support ongoing clinical development and corporate initiatives. The offering included significant participation by Quoin’s entire executive team. A day prior, Quoin received FDA clearance to initiate a new whole-body clinical study for QRX003. This groundbreaking study, which will be led by Dr. Amy Paller at Northwestern University, will evaluate the safety and efficacy of twice-daily application of QRX003 to more than 80% of the body surface area in Netherton Syndrome patients, mimicking potential real-world use. This represents the most extensive clinical application of QRX003 to date, with data anticipated to become a cornerstone of the company’s regulatory submission package. On Dec. 18, 2024, Quoin announced positive interim data from two of its ongoing Netherton Syndrome clinical studies. The first subject dosed twice daily for six weeks in the company’s open-label study demonstrated a significant clinical response across multiple endpoints. In addition, data from Quoin’s ongoing pediatric study showed a rapid improvement in skin appearance within 12 days of treatment, with no adverse events reported. Financial Highlights Quoin had approximately $14.1 million in cash, cash equivalents and marketable securities as of December 31, 2024. This is expected to fund the Company’s operations into the second quarter of 2026. Net loss for the quarter ended December 31, 2024 was approximately $2.3 million compared to approximately $2.0 million for the quarter ended December 31, 2023. Net loss for the twelve months ended December 31, 2024 was $9.0 million compared to $8.7 million for the twelve months ended December 31, 2023. Investors are encouraged to read the Company’s Annual Report on Form 10-K when it is filed with the Securities and Exchange Commission (the “SEC”), which will contain additional details about Quoin’s financial results as of and for the period ended December 31, 2024. About Quoin Pharmaceuticals Ltd.Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, microcystic lymphatic malformations, venous malformations and others. For more information, go to: www.quoinpharma.com. Cautionary Note Regarding Forward-Looking StatementsThe Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “hope,” “plan,” “potential,” “anticipate,” “look forward,” “believe,” “may,” and “will,” among others. All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: QRX003’s potential to address the underlying pathology of Netherton Syndrome; whole-body’ clinical study generating a robust and comprehensive safety and efficacy data set that will become a central component of the Company’s regulatory approval submission; the Company developing the first FDA-approved treatment for Netherton Syndrome; the Company’s new U.S. and International patent applications for novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare diseases including microcystic lymphatic malformations, venous malformations and angiofibromas; the progress or success of Quoin’s ongoing clinical trials; and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, microcystic lymphatic malformations, venous malformations and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the clinical studies may not generate the results anticipated; the Company ability to recruit additional subjects; the clinical studies not generating data which is sufficiently robust and comprehensive to support an NDA filing; the Company’s ability to obtain regulatory approvals; the Company’s ability to protect its assets with the new patent applications; the Company’s ability to obtain regulatory approvals for the commercialization of its product candidates or to comply with ongoing regulatory requirements; the Company’s ability to deliver a safe and effective treatment for Netherton Syndrome; the Company may be unable to submit applications and initiate clinical development as and when planned; and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. For further information, contact:Quoin Pharmaceuticals Ltd.Michael Myers, Ph.D., CEOmmyers@quoinpharma.com Investor RelationsPCG AdvisoryJeff Ramson jramson@pcgadvisory.com(646) 863-6341

Clinical ResultFinancial Statement

04 Mar 2025

·investors.quoinpharma.com

Quoin Pharmaceuticals Files U.S. and International Patent Applications for Novel Topical Rapamycin Formulations to Treat Rare Disease Indications

Target Indications Include Microcystic Lymphatic Malformations, Venous Malformations and Angiofibromas Proprietary Invisicare Delivery Technology Designed to Optimize Local Skin Penetration Company Plans to Submit Investigational New Drug Applications and Initiate Clinical Testing This Year ASHBURN, Va., March 04, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, today announced it has filed U.S. and International patent applications for novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare diseases including microcystic lymphatic malformations, venous malformations and angiofibromas. The products are being developed using Quoin’s in-licensed proprietary Invisicare delivery technology. There are currently no FDA approved treatments for either microcystic lymphatic malformations or venous malformations. The proprietary Invisicare delivery technology, which Quoin has exclusive rights to for all orphan rare skin disease applications, is designed to optimize penetration of rapamycin deep into dermis where it can be most effective clinically. The Invisicare technology is also being utilized in Quoin’s QRX003 topical lotion, which is in late-stage clinical testing as a potential treatment for Netherton Syndrome, a rare genetic disease. Quoin believes QRX003 has the potential to become the first approved treatment for this disease. “As we continue to advance the clinical development of Quoin’s lead product, QRX003, for Netherton Syndrome, we are pleased to announce the filing of this patent and the initiation of the development of our novel formulations as potential treatments for these additional rare skin diseases. By combining the known clinical activity of rapamycin with this optimized delivery system, we believe our novel topical formulations may have the potential to effectively treat these diseases. We have seen other topical formulations of rapamycin underperform in clinical settings across a number of indications, which we believe may be as a result of suboptimal delivery of the drug at the target sites. The Invisicare technology is designed to overcome these limitations. We are now moving forward with our plans to submit IND applications for at least two of these target indications this year and to formally initiating clinical development as soon as possible ” said Dr. Michael Myers, Chief Executive Officer of Quoin. Quoin is currently enrolling patients in three clinical trials being conducted under its open Investigational New Drug (IND) application, evaluating its QRX003 topical lotion as a potential treatment of Netherton Syndrome. To date, Quoin remains the only company actively recruiting subjects into multiple NS clinical trials that are being conducted under an open IND. To find out more about Quoin’s clinical studies relating to Netherton Syndrome, please visit http://www.nethertonsyndromeclinicaltrials.com/. About Microcystic Lymphatic Malformations Microcystic lymphatic malformation is one subtype of lymphatic malformation (LM), a congenital malformation of the lymphatic vessels in soft tissues, including the skin. LM is classified into the macrocystic type, cysts larger than 2 cm with clear margins (previously known as cystic hygromas), and the microcystic type, consisting of cysts smaller than 2 cm, that appear diffuse, and grow without clear borders (previously known as lymphangioma circumscriptum). When the two types concur it is called the combined type. Microcystic lesions are commonly found inside the mouth, throat, and in the tongue, parotid gland and submandibular gland. Symptoms include deformity, and problems with breathing and feeding. The exact cause is unknown but is likely related to a malformation of the lymphatic system at six to ten weeks of gestation, when some lymphatic tissue fails to communicate with the lymphatic and venous system.1 Lymphatic malformations occurring in 1 in 6000 to 16,000 patients. 2 About Venous Malformations Venous malformation (VM) is the most common type of congenital vascular malformation (CVM) with an incidence of 1 to 2 in 10,000 and a prevalence of 1%. They can cause significant morbidity, pain and discomfort to patients as they can lead to serious local and systemic complications. Although present at birth, they are not always clinically evident until later in life and tend to grow in concert with the child and without spontaneous regression. VMs are composed of ectatic venous channels found usually in the head, neck, limbs, and trunk and are thought to be sporadic in most cases, though familial inheritance patterns exist. Accurate diagnosis has been a limiting factor in VM management. An increased emphasis has been placed on creating comprehensive classification systems for diagnostic and therapeutic purposes of this chronic condition. Doppler ultrasound (US) and magnetic resonance imaging (MRI) are key imaging methods used to characterize and diagnose VMs. Treatment options include surgery, sclerotherapy, and ablative therapies. 3 About Angiofibroma Cutaneous angiofibroma is a benign skin tumor characterized by fibrovascular tissue and presents as a group of lesions with varied clinical appearances but consistent histological features. These benign fibrous neoplasms exhibit a proliferation of stellate and spindled cells, thin-walled blood vessels with dilated lumina in the dermis, and concentric collagen bundles. These growths typically manifest as small, firm, reddish, or flesh-colored papules, most commonly on the face (often referred to as fibrous papules or adenoma sebaceum), particularly around the nose and cheeks.4 About Quoin Pharmaceuticals Ltd. Quoin Pharmaceuticals Ltd. is a clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa and others. For more information, go to: www.quoinpharma.com. Cautionary Note Regarding Forward-Looking Statements The Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “hope,” “plan,” “potential,” “anticipate,” “look forward,” “believe,” “may,” and “will,” among others. All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the Company’s new U.S. and International patent applications for novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare diseases including microcystic lymphatic malformations, venous malformations and angiofibromas; Invisicare delivery technology being designed to optimize penetration of rapamycin deep into dermis where it can be most effective clinically; the potential efficacy of QRX003 as a treatment for Netherton Syndrome; the progress or success of Quoin’s ongoing clinical trials; combining rapamycin with the Invisicare technology delivery system having the potential to effectively treat these diseases; plans to submit IND applications for at least two of these target indications this year and initiating clinical development as soon as possible; and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company’s ability to protect its assets with the new patent applications; the Company’s ability to obtain regulatory approvals for the commercialization of its product candidates or to comply with ongoing regulatory requirements; the Company’s ability to deliver a safe and effective treatment for Netherton Syndrome; the Company may be unable to submit applications and initiate clinical development as and when planned; and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. For further information, contact: Quoin Pharmaceuticals Ltd. Michael Myers, Ph.D., CEO mmyers@quoinpharma.com Investor Relations PCG Advisory Jeff Ramson jramson@pcgadvisory.com (646) 863-6341 1 Microcystic lymphatic malformation | About the Disease | GARD 2 Genetic and Molecular Determinants of Lymphatic Malformations: Potential Targets for Therapy - PMC 3 Venous malformations: clinical diagnosis and treatment - PMC 4 Cutaneous Angiofibroma - StatPearls - NCBI Bookshelf

Clinical Study

03 Mar 2025

·ir.aadibio.com

Aadi Bioscience Announces Approval of All Proposals at Special Meeting of Stockholders

MORRISTOWN, N.J., March 3, 2025 /PRNewswire/ -- Aadi Bioscience, Inc. (NASDAQ: AADI), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver advances in cancer treatment, today announced that the Company's stockholders have approved all proposals voted on at the Company's Special Meeting of Stockholders ("Special Meeting") held on February 28, 2025. Approved proposals include: Divestiture Proposal. The approval of the sale by Aadi to KAKEN INVESTMENTS INC. ("Kaken") of 100% of the outstanding shares of capital stock of Aadi Subsidiary, Inc. ("Aadi Sub") and thereby all or substantially all of Aadi's assets related to its FYARRO® (sirolimus protein-bound particles for injectable suspension) (albumin-bound) program (the "FYARRO Business"). The PIPE Financing Proposal. The approval of, for purposes of complying with the applicable provisions of Nasdaq Listing Rule 5635, the issuance of an aggregate of 21,592,000 shares of Aadi's common stock, par value $0.0001 per share, at a price of $2.40 per share and pre-funded warrants to purchase up to an aggregate of 20,076,500 shares of Aadi's common stock at a purchase price of $2.3999 per pre-funded warrant (the "PIPE Financing Proposal"). Equity Plan Increase Proposal. The approval of an amendment to the Aadi Bioscience, Inc. 2021 Equity Incentive Plan (the "2021 Plan") to (i) increase the shares available for issuance under the 2021 Plan by 6,300,000 shares from 2,000,284 shares to 8,300,284 shares and (ii) increase the 2021 Plan's default annual automatic share reserve increase occurring on January 1 of each year from 4% of outstanding shares on the last day of the immediately preceding fiscal year to 5% (the "Equity Plan Increase Proposal"). Non-Binding Advisory Vote on Golden Parachutes Proposal. To approve, on a non-binding, advisory basis, certain compensation that will or may become payable to our named executive officers in connection with the Divestiture (the "Non-Binding Advisory Vote on Golden Parachutes Proposal"). Final voting results from the Special Meeting will be included in a Form 8-K filed with the Securities and Exchange Commission. About Aadi Bioscience Aadi is a precision oncology company with a vision to make bold choices in applying technology to efficiently deliver improved precision oncology therapies for people living with difficult-to-treat cancers. More information on the Company is available on the Aadi website at www.aadibio.com and connect with us on LinkedIn. Contact: IR@aadibio.com View original content to download multimedia:https://www.prnewswire.com/news-releases/aadi-bioscience-announces-approval-of-all-proposals-at-special-meeting-of-stockholders-302389545.html SOURCE Aadi Bioscience

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	Japan	Nobelpharma Co., Ltd.	18 Jan 2024
Vascular Neoplasms	Japan	Nobelpharma Co., Ltd.	18 Jan 2024
Angiofibroma	Norway	Pfizer Inc.	15 May 2023
Angiofibroma	Liechtenstein	Pfizer Inc.	15 May 2023
Angiofibroma	Iceland	Pfizer Inc.	15 May 2023
Angiofibroma	European Union	Pfizer Inc.	15 May 2023
Tuberous Sclerosis	China	Nobelpharma Co., Ltd.	28 Mar 2023
Angiofibroma associated with tuberous sclerosis	United States	Nobelpharma Co., Ltd.	22 Mar 2022
Lymphangiectasis	Japan	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioma	Japan	Nobelpharma Co., Ltd.	27 Sep 2021
Osteolysis, Essential	Japan	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioleiomyomatosis	Iceland	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	European Union	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	Norway	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	Liechtenstein	Pfizer Europe MA EEIG	13 Mar 2001
Renal transplant rejection	United States	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	Japan	Nobelpharma Co., Ltd.	-
Liver transplant rejection	Preclinical	France	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	Portugal	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	United States	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	Switzerland	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	Italy	Wyeth AB	01 Dec 2002
Allograft Rejection	Discovery	-	Wyeth AB	01 Aug 2002
Graft vs Host Disease	Discovery	United States	Wyeth AB	20 May 2002
Graft vs Host Disease	Discovery	Canada	Wyeth AB	20 May 2002
Kidney Diseases	Discovery	-	Wyeth AB	01 Jul 2000

Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00866684 (PROSKIN, CTgov)	Phase 4	Skin Neoplasms	44	Sirolimus (1 Sirolimus)	uetavirmkn(iisustkjxk) = cjcjjawgua yxvcppdowu (soerfjhzme, ivrrfvekqn - rqvwqknbhr) View more	-	14 Mar 2025
				Ciclosporin+Tacrolimus+Azathioprine+Mycophenolate (2 Standard)	uetavirmkn(iisustkjxk) = egudyniovi yxvcppdowu (soerfjhzme, gdffvykosg - awabfschnv) View more
NCT05050149 (CTgov)	Phase 2	Lymphatic Abnormalities	12	PTX-022	tyzyhgzirp(ajihjmqymw) = mlinekhknr catxlmcrro (beicapfmlc, wrhjitlbck - vjfmkjqnqa) View more	-	17 Jan 2025
SIRONA (PRNewswire) Manual	Not Applicable	Peripheral Arterial Disease	482	MagicTouch PTA	fsufjunqqc(djxhrliocf) = lcyppaqkmz gzsuqpvztx (pzmhnrpepn ) Met View more	Similar	08 Jan 2025
				紫杉醇涂层球囊	fsufjunqqc(djxhrliocf) = swlkqfgjhf gzsuqpvztx (pzmhnrpepn ) Met View more
NCT04339101 (1043, ASH2024)	Phase 2	Myelofibrosis \| acute leukemia \| Myelodysplastic Syndromes HGF \| IL17 \| TNFaR ... View more	59	Itacitinib + Tacrolimus/Sirolimus	upzamrkqjm(hzxhvekbhn) = xnqqoazoec oeoqqmzrog (eboshaaglu, 41 - 66) View more	Positive	09 Dec 2024
				Tacrolimus/Sirolimus	upzamrkqjm(hzxhvekbhn) = tgghbnpepq oeoqqmzrog (eboshaaglu )
ChiCTR1900020657 (1187Sirolimus, ASH2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic First line	-	HD-DXM plus sirolimus	rzsxdnufre(rcrvfhmrld) = ebdwzhsduu vsocjjqaqm (pbqxteffry ) View more	Positive	07 Dec 2024
				HD-DXM monotherapy	rzsxdnufre(rcrvfhmrld) = yjykoavhzy vsocjjqaqm (pbqxteffry ) View more
NCT02105766 (CTgov)	Phase 2	Beta-Thalassemia \| Hematopoietic stem cell transplantation \| Anemia, Sickle Cell \| Graft vs Host Disease	56	Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor)	ghbcynnyph(knyjobmjzt) = byrxccbrwn essawoihlu (vbmuyztkax, molpdtaenm - qdnhhpucrr) View more	-	29 Nov 2024
				Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant)	okmkaebecj(fgbutsyrae) = ttgtfbzpfl uauqlgigcm (pdoijdgvcx, ejznzrwtja - smqtyoqyia) View more
NCT03095703 (CTgov)	Phase 2	Adenomatous Polyposis Coli	4	Sirolimus	gzdegkshpj(wnzszcoqxg) = ulmnsjezdk cpskqdnoxn (sgobhjsqgm, xsbirdyteb - pgbqweyfrf) 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	ikeqgwvgmg(gmvemadazr) = rwjxxswgbg nhqpmmkeva (dhhojfnryw, vzsqplxvqi - vkbgmpfasa) View more	-	22 Aug 2024
NCT03077542 (CTgov)	Phase 1/2	Anemia, Sickle Cell	57	Filgrastim	cuiyvvscfn(rqbytimngo) = tzcielxvgc bclhlsxfvf (ckeayzejej, fetlthrqtq - egnoshkrkz) 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	(npvowcxubl) = pgrtgctoch gwqiogeuby (crjhijkyrw, ljxgvgxois - yramuwtddq) View more	-	25 Jul 2024

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