Temsirolimus Adventitial Delivery to Improve ANGioplasty and/or Atherectomy Revascularization Outcomes Below the Knee: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Effect of Temsirolimus Perivascular Injection 0.1 mg/mL on the Incidence of Ischemia-Driven Major Amputation, Clinically Driven Target Lesion Revascularization, and Clinically Relevant Target Lesion Occlusion After Revascularization of Lesions Below the Knee in Patients With Symptomatic Rutherford 3-5 Peripheral Artery Disease

A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the effect of Temsirolimus Perivascular Injection 0.1 mg/mL on the incidence of ischemia-driven major amputation, clinically driven target lesion revascularization, and clinically relevant target lesion occlusion after revascularization of lesions below the knee in patients with symptomatic Rutherford 3-5 peripheral artery disease. The primary safety endpoint will be gathered at 1-month post-index procedure. The primary efficacy endpoint will be gathered at 6 months post-index procedure. Participants will be followed for up to 5 years post-index procedure.

Start Date30 Jul 2025

Sponsor / Collaborator

Mercator MedSystems, Inc.

NCT04199026

/ RecruitingNot ApplicableIIT

Pilot Trial of an Implantable Microdevice for In Vivo Drug Sensitivity Testing in Patients With Sarcomas

This early phase I trial studies the side effects of implanting and removing a microdevice in patients with sarcomas that have spread to other places in the body (metastatic) or have come back (recurrent). Microdevices are rice-sized devices that are implanted into tumor tissue and are loaded with 10 different drugs that are delivered at very small doses, or "microdoses," which may only affect a very small, local area inside the tumor. The purpose of this study is to determine which drugs delivered in the microdevice affect tumor tissue in patients with sarcomas.

Start Date25 Feb 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

CTIS2024-520226-10-00

/ Not yet recruitingPhase 3IIT

Evaluation of the efficacy of treatment with mucosal bacterial vaccines(autovaccines) versus suppressive antibiotic treatment in subjects with hip or knee prosthesis infections.

Start Date16 Feb 2024

Sponsor / Collaborator-

100 Clinical Results associated with Temsirolimus

100 Translational Medicine associated with Temsirolimus

100 Patents (Medical) associated with Temsirolimus

1,271

Literatures (Medical) associated with Temsirolimus

01 Feb 2026·ANTI-CANCER DRUGS

Temsirolimus targets chemoresistant uveal melanoma via mammalian target of rapamycin inhibition and enhances chemotherapy

Article

Author: Huang, Qing ; Tao, Na ; Jiang, Xuan

Chemotherapy resistance remains a major challenge in the treatment of uveal melanoma, necessitating the identification of novel therapeutic strategies. In this study, we established chemoresistant uveal melanoma cell lines by exposing parental cells to dacarbazine, cisplatin, or gemcitabine and performed high-throughput drug screening incorporating normal human epidermal melanocytes (NHEMs) as a normal control to assess both efficacy and selectivity. Our screening identified temsirolimus and selumetinib as top candidates, with temsirolimus exhibiting strong tumor-selective cytotoxicity. Further in-vitro studies confirmed that temsirolimus induced apoptosis and suppressed clonogenic potential in chemoresistant uveal melanoma cells while having minimal effects on NHEM. Combination studies demonstrated synergy between temsirolimus and cisplatin or gemcitabine, reinforcing its role as an effective chemosensitizer. In a chemoresistant uveal melanoma xenograft model, temsirolimus significantly inhibited tumor growth without inducing systemic toxicity, as evidenced by stable biochemical markers of organ function. Mechanistically, temsirolimus downregulated mammalian target of rapamycin (mTOR) signaling, as indicated by reduced p-mTOR, p-S6, and p-4EBP1 expression in tumor tissues. These findings demonstrate that temsirolimus selectively targets chemoresistant uveal melanoma cells, enhances chemotherapy efficacy, and suppresses tumor growth via mTOR inhibition, supporting its potential clinical application as a novel therapeutic strategy for chemoresistant uveal melanoma.

01 Jan 2026·FERTILITY AND STERILITY

Temsirolimus and antimüllerian hormone protect fertility and the ovarian reserve against chemotherapy-induced damage in a murine model

Article

Author: Donnez, Jacques ; Zipponi, Margherita ; Goudmaeker, Apolline ; Dehoux, Jean-Paul ; Dolmans, Marie-Madeleine ; Houeis, Lara ; van der Plancke, Graziella ; Kluyskens, Candice ; Cacciottola, Luciana

OBJECTIVE:

To investigate the effects of temsirolimus (Tem) and recombinant antimüllerian hormone (AMH) on fertility and the ovarian reserve when associated with chemotherapy.

DESIGN:

Thirty-seven female mice were assigned to one of three treatment groups: control group; chemotherapy group; and gonadoprotection group. Five weeks after treatment, the mice underwent ovarian stimulation and were mated with males to evaluate residual fertility. Two days after mating, the animals were euthanized for embryo collection, and the ovaries were retrieved to assess the ovarian reserve and follicle damage.

SUBJECTS:

Thirty-seven Naval Medical Research Institute mice.

EXPOSURE:

The control group (n = 10) was given a 100-μL injection of phosphate-buffered saline. The chemotherapy group (n = 13) received a single 100-μL injection of chemotherapy (120-mg/kg cyclophosphamide and 12-mg/kg busulfan). The gonadoprotection group (n = 14) was administered five injections of 5-mg/kg Tem over 1 week prior to chemotherapy, followed by a chemotherapy injection and then five injections of 5-μg recombinant AMH over the next 24 hours.

MAIN OUTCOME MEASURES:

Embryo count, follicle count and morphology, and immunostaining for apoptosis (cleaved caspase-3), follicle activation (phospho-Akt), and proliferation (Ki67).

RESULTS:

The control group showed the highest mean number of retrieved embryos (41.40 ± 14.74), closely followed by the gonadoprotection group (36.27 ± 17.22), whereas the chemotherapy group exhibited a significant reduction (20.63 ± 12.12) compared to both groups. Regarding the ovarian reserve, the control group showed the highest total follicle count (897.4 ± 392.8), with no difference compared to the gonadoprotection group (714.4 ± 283.9); however, there was a significant decline in the chemotherapy group (320.7 ± 145.5) compared with those in both groups. In terms of follicle apoptosis and activation, no significant difference was observed between groups. Follicle proliferation rates were significantly higher in the chemotherapy group (59.38% ± 14.67%) than in the control group (20.72% ± 10.52%); but gonadoprotective treatment did curtail follicle proliferation (42.23% ± 18.37%) compared with the chemotherapy group.

CONCLUSION:

Our findings suggest that Tem and AMH show promise as gonadoprotective agents, potentially protecting the ovarian reserve against chemotherapy and thereby preserving fertility. This could offer patients who cannot benefit from currently available techniques brand new fertility preservation options.

31 Dec 2025·ANNALS OF MEDICINE

A novel glycogene-related signature for prognostic prediction and immune microenvironment assessment in kidney renal clear cell carcinoma

Article

Author: Zhao, Danni ; Cui, Hanxiao ; Ren, Xueting ; Zhao, Xuyan ; Kang, Huafeng ; Liu, Peinan ; Li, Zihao ; Lin, Shuai ; Zhou, Mingjing

BACKGROUND:

Kidney Renal Clear Cell Carcinoma (KIRC) is a prevalent urinary malignancies worldwide. Glycosylation is a key post-translational modification that is essential in cancer progression. However, its relationship with prognosis, tumour microenvironment (TME), and treatment response in KIRC remains unclear.

METHOD:

Expression profiles and clinical data were retrieved from The Cancer Genome Atlas and Gene Expression Omnibus databases. Consensus clustering, Cox regression, and LASSO regression analyses were conducted to develop an optimal glycogene-related signature. The prognostic relevance of this molecular signature was rigorously analyzed, along with its connections to tumour microenvironment (TME), tumour mutation burden, immune checkpoint activity, cancer-immunity cycle regulation, immunomodulatory gene expression patterns, and therapeutic response profiles. Validation was performed using real-world clinical specimens, quantitative PCR (qPCR), and immunohistochemistry (IHC), supported by cohort analyses from the Human Protein Atlas (HPA) database.

RESULTS:

A glycogene-associated prognostic scoring system was established to categorize patients into risk-stratified subgroups. Patients in the high-risk cohort exhibited significantly poorer survival outcomes (p < 0.001). By incorporating clinicopathological variables into this framework, we established a predictive nomogram demonstrating strong calibration and a concordance index (C-index) of 0.78. The high-risk subgroup displayed elevated immune infiltration scores (p < 0.001), upregulated expression of immune checkpoint-related genes (p < 0.05), and an increased frequency of somatic mutations (p = 0.043). The risk score positively correlated with cancer-immunity cycle activation and immunotherapy-related signals. The high-risk groups also showed associations with T cell exhaustion, immune-activating genes, chemokines, and receptors. Drug sensitivity analysis revealed that low-risk patients were more sensitive to sorafenib, pazopanib, and erlotinib, whereas high-risk individuals responded better to temsirolimus (p < 0.01). qPCR and IHC analyses consistently revealed distinct expression patterns of MX2 and other key genes across the risk groups, further corroborated by the HPA findings.

CONCLUSION:

This glycogene-based signature provides a robust tool for predicting prognosis, TME characteristics, and therapeutic responses in KIRC, offering potential clinical utility in patient management.

News (Medical) associated with Temsirolimus

02 Dec 2025

·www.globenewswire.com

Oncotelic and Sapu Nano Unveils Broad-Applicability Deciparticle™ Nanomedicine Platform Capable of Formulating Multiple Hydrophobic Peptide, Macrolide, and Polyketide Drugs

AGOURA HILLS, Calif., Dec. 02, 2025 (GLOBE NEWSWIRE) -- Oncotelic Therapeutics, Inc. (OTCQB: OTLC) 45% owned Joint Venture, Sapu Nano, a clinical-stage nanomedicine company, today announced new data demonstrating that its proprietary Deciparticle™ platform enables broad and consistent formulation of structurally diverse hydrophobic therapeutics—including macrolide mTOR inhibitors, cyclic peptides, linear peptides, ascomycin macrolactams, and polyketides—while maintaining a precise sub-20 nm nanoparticle profile suitable for intravenous (IV) delivery. These findings, presented at the 2025 San Antonio Breast Cancer Symposium (SABCS), confirm that the Deciparticle™ technology represents a chemically versatile, cGMP-ready delivery platform with the potential to support an expanding pipeline of oncology and immunology drug products. Presentation info: PS4-04-21. Deciparticle™ Everolimus (Sapu003): From Cytostasis to Cytotoxicity via a Single mPEG Polymer and Clinic-Ready Manufacturing. Sheng-Hao Min, Kevin Forero, Johnathan Anderson, William Putnam, Can Evizi, Cassidy McCallum, Robert Hoff, Mark Ostrander, and Kwun Ho. Deciparticle™ Platform Demonstrates Broad Formulation Capability Across Therapeutic Classes New data from a comprehensive screening effort show that Sapu Nano’s optimized polymer reproducibly forms monodisperse nanoparticles (~15 nm) capable of encapsulating multiple classes of hydrophobic drugs: Macrolide mTOR Inhibitors All five clinically relevant macrolide immunosuppressants formed stable Deciparticles™: SirolimusTemsirolimusRidaforolimusUmirolimusEverolimus Differences in substitution at the C-40/C-42 or C-33 positions did not impair nanoparticle formation, highlighting the platform’s tolerance for structural diversity within the macrolide family. Ascomycin Macrolactams Tacrolimus formed stable sub-20 nm Deciparticles™.Pimecrolimus, while encapsulated, produced particle sizes above the Deciparticle™ threshold—defining a steric upper bound for the platform and its suitability for optimized macrolactam design. Cyclic and Linear Peptides The Deciparticle™ platform successfully formulated two medically important peptide classes: Cyclosporine A (hydrophobic cyclic peptide)Exenatide (synthetic linear peptide) These findings demonstrate the platform’s ability to handle both compact hydrophobic macrocycles and amphipathic linear peptides—an important expansion for peptide-based therapeutic pipelines. Robust and Scalable cGMP Manufacturing The platform is supported by Sapu Nano’s ISO-5 cGMP facility capable of: One-pot bulk drug manufacturingSterile filtrationAutomated fill/finishLyophilization into a stable, clinic-ready drug productHigh batch reproducibilityDays to weeks post-reconstitution stabilityPrecision particle-size control with tight lot-to-lot consistency This cGMP infrastructure enables fast, reliable translation from formulation to Phase 1 supply, supporting multiple INDs annually. “With these new data, the Deciparticle™ platform has emerged as a broad, modular nanomedicine engine capable of formulating multiple classes of hydrophobic drugs that were previously constrained by solubility and delivery barriers,” said Dr. Vuong Trieu, Chief Executive Officer of Sapu Nano. “This platform advances beyond single-asset value to a multi-asset opportunity across oncology, immunology, and peptide therapeutics. Sapu003 is only the beginning.” About the Deciparticle™ Platform The Deciparticle™ platform is a proprietary nanotechnology engineered to encapsulate hydrophobic molecules as uniform, sub-20 nm nanoparticles for intravenous administration. The platform improves systemic exposure, reduces GI deposition, and supports precision delivery while maintaining manufacturability at clinical scale. About Sapu Nano Sapu Nano is a clinical-stage biotechnology company developing Deciparticle™ nanomedicine therapeutics designed to optimize the delivery of hydrophobic oncology agents and peptide-based therapeutics. The company operates an integrated ISO-5 cGMP manufacturing facility supporting rapid progression from formulation to clinical trial supply. For more information, visit www.sapunano.com. About Oncotelic Oncotelic (f/k/a Mateon Therapeutics, Inc.), was formed in the State of New York in 1988 as OXiGENE, Inc., was reincorporated in the State of Delaware in 1992, and changed its name to Mateon Therapeutics, Inc. in 2016, and Oncotelic Therapeutics, Inc. in November 2020. Oncotelic is seeking to leverage its deep expertise in oncology drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Oncotelic has rare pediatric designation for Diffuse Intrinsic Pontine Glioma “DIPG” (through OT-101) through its 45% joint venture, melanoma (through CA4P), and Acute Myeloid Leukemia “AML” (through OXi 4503). Oncotelic also acquired PointR Data Inc. in November 2019. Oncotelic acquired AL-101, during the 4th quarter of 2021, for the intranasal delivery of apomorphine. We intend to develop AL-101 for the treatment of Parkinson Disease ("PD"). Over 60,000 new patients are being diagnosed with PD in the United States and currently there are over 1 million patients in the US and expected to increase to over 1.2 million by 2030. In addition, approximately 10 million suffer from this disease globally. https://www.parkinson.org/Understanding-Parkinsons/Statistics. AL-101 is also being developed for Erectile Dysfunction ("ED"). ED is the most prevalent male sexual disorder globally. The percentages of men affected by ED are as follows: 14.3-70% of men aged 60 years, 6.7-48% of men aged 70 years, and 38% of men aged 80 years (Geerkens MJM et al. (2019). Eur Urol Focus. pii: S2405-4569(19)30079-3). However, with the increasing administration of PDE5 inhibitors in clinical practice, it was found that approximately 30-35% of ED patients are treatment failures (McMahon CN et al. (2006). BMJ, 332: 589-92). AL-101 is designed to target treatment failure ED patients who do not respond to PDE5 inhibitors. Through similar mechanism of action, AL-101 is being developed for Female Sexual Dysfunction ("FSD"). Female sexual dysfunction is a prevalent problem, afflicting approximately 40% of women and there are few treatment options. FSD is more typical as women age and is a progressive and widespread condition. (Allahdadi, KJ et al. (2009) Cardiovascular & hematological agents in medicinal chemistry, 7(4), 260-269). There is no available drug for the treatment of FSD. In June 2019, the U.S. Food and Drug Administration approved Vyleesi (bremelanotide) to treat acquired, generalized hypoactive sexual desire disorder ("HSDD") in premenopausal women. This is the only available drug treatment. Vyleesi has essentially replaced the only other drug for HSDD - however, it has a long list of drug-drug interactions, including commonly used antidepressants, such as fluoxetine and sertraline. In addition, it has a black box warning regarding its use with alcohol, a combination that has been associated with hypotension and syncopal episodes. Therefore, there is an urgent need for effective therapy against FSD and HSDD. Oncotelic's Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934 (the “Exchange Act”) that involve substantial risks and uncertainties. We generally identify forward-looking statements by terminology such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “would,” “intend,” “target,” “aim,” “project,” “believe,” “estimate,” “predict,” “potential,” “seek,” “indicate,” or “continue” or the negative of these terms or other similar words, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements regarding our or our management’s expectations, hopes, beliefs, intentions or strategies regarding the future, such as our estimates regarding anticipated operating income or losses, future performance, future revenues and projected expense, including that to fund our clinical and other development programs; our liquidity and our expectations regarding our needs for and ability to raise additional capital; our ability to continue as a going concern; our ability to select and capitalize on commercially desirable product opportunities as a result of limited financial resources; our ability to manage our expenses effectively and raise the funds needed to continue our business; our ability to retain the services of our current or future executive officers, directors and principal consultants; the competitive nature of our industry and the possibility that our products or product candidates may become obsolete or may not generate revenues as expected or at all; our ability to obtain and maintain regulatory approval of our existing products and any future products we may develop; the development of and the process of commercializing AI/Blockchain and other technologies for supporting the development of OT- 101 and Artemisinin for COVID-19, OT-101, including development of OT-101, Artemisinin, OXi4503, CA4P and our 2021 in-licensing of apomorphine; the initiation, timing, progress and results of our preclinical and clinical trials, research and development programs; regulatory and legislative developments in the United States and foreign countries; the timing, costs and other limitations involved in obtaining regulatory approval for any product; the further preclinical or clinical development and commercialization of our product candidates; the entering into any corporate transactions to develop our products through partnerships, joint ventures or other corporate transactions; our ability to make a proposed initial public offering between us and our joint-venture partners for the joint venture, statements about future plans related to the operations of the JV, taking the JV into an initial public offering or the success thereof: building and the success of our nanoparticle platform and the related success of launching the platform; the expected valuation of the JV, and therefore a corresponding increase in the valuation of the Company, by virtue of it’s ownership in the JV; the success of the launch of Pet2DAO, a corporation with a DAO infrastructure, the success of Pet2DAO and the plans surrounding the pet and animal health, the ability for the Company to register the tokens of Pet2DAO, the actual filing of a registration statement and approval of the tokens as registrable securities with the Securities and Exchange Commission (“SEC”) through a registration statement, the ability of the tokens to be tradable or any value such tokens may have if they become tradable; our ability to obtain and maintain orphan drug exclusivity for some of our product candidates; the potential benefits of our product candidates over other therapies; our ability to enter into and maintain any collaboration with respect to product candidates; our ability to continue to develop or commercialize our products or product candidates in the event any license agreements in place with third parties expire or are terminated; the performance and conduct of third parties, including our third-party manufacturers and third party service providers used in our clinical trials; our ability to obtain and maintain intellectual property protection for our products and operate our business without infringing upon the intellectual property rights of others; the potential liability exposure related to our products and our insurance coverage for such exposure; our ability to form alliances with other third parties to develop the products in our pipeline through partnerships, joint ventures, mergers or acquisitions; the successful development of our sales and marketing capabilities; the size and growth of the potential markets for our products and our ability to serve those markets; the rate and degree of market acceptance of any future products; the volatility of the price of our common stock; the ability to achieve secondary trading of our stock in certain states; the dilutive effects of potential future equity issuances; our expectation that no dividends will be declared on our common stock in the foreseeable future; our ability to maintain an effective system of internal controls; the payment and reimbursement methods used by private or governmental third-party payers; our ability to retain adequate staffing levels; unfavorable global economic conditions; unfavorable global epidemic and pandemic conditions; a failure of our internal computer systems or those of our contractors and consultants; potential misconduct or other improper activities by our employees, contractors or consultants; the ability of our business continuity and disaster recovery plans to protect us in the event of a natural disaster; and other factors discussed elsewhere in this document or any document incorporated by reference herein or therein. Contact Information:For Oncotelic Therapeutics, Inc.:Investor Relationsir@oncotelic.com

AcquisitionDrug Approval

18 Jun 2025

·www.einpresswire.com

Palvella Therapeutics Granted Sixth U.S. Patent Covering 0.1–20% Anhydrous Compositions of Rapamycin and Other mTOR Inhibitors

New intellectual property builds on Palvella’s multi-layered exclusivity strategy Patent term expected through at least 2038 WAYNE, Pa., June 18, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc . (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the United States Patent and Trademark Office (USPTO) issued patent No. 12,329,748 for claims related to the Company's lead product candidate QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin). The patent provides broad protection for the proprietary anhydrous topical composition and methods of use for QTORIN™ rapamycin. “Our sixth issued U.S. patent marks another meaningful milestone in solidifying the exclusivity position of QTORIN™ rapamycin,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We’re executing a multi-layered strategy to protect and maximize the long-term value of QTORIN™ rapamycin—anchored by strong intellectual property, proprietary formulation and manufacturing trade secrets, and regulatory exclusivities.” The newly granted U.S. patent encompasses a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors—including temsirolimus and everolimus—formulated in anhydrous compositions. It also covers treatment of a broad spectrum of dermatologic conditions, with emphasis on rare and difficult-to-treat diseases such as microcystic lymphatic malformations and venous malformations. QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track Designations by the FDA for the treatment of microcystic lymphatic malformations. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S. Palvella has also been named an awardee of an FDA Orphan Products Grant from the FDA’s Office of Orphan Products Development and is eligible to receive up to $2.6 million over the life of the grant to support the ongoing SELVA Phase 3 trial of QTORIN™ rapamycin. About Palvella Therapeutics Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter). QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication. Forward-Looking Statements This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Contact Information Investors Wesley H. Kaupinen Founder and CEO, Palvella Therapeutics wes.kaupinen@palvellatx.com Media Marcy Nanus Managing Partner, Trilon Advisors LLC mnanus@trilonadvisors.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Orphan DrugPhase 3Fast TrackBreakthrough Therapy

04 Apr 2025

·www.prnewswire.com

UPSHER-SMITH TO SHOWCASE RARE DISEASE PORTFOLIO AT 2025 AAN ANNUAL MEETING

Company to Feature First and Only Ready-to-Use Liquid Vigabatrin at Booth #846 MAPLE GROVE, Minn., April 4, 2025 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced its participation at the 2025 American Academy of Neurology (AAN) Annual Meeting, from April 5-9, 2025 in San Diego, California. Representatives from Upsher-Smith will be available at Booth #846 to share information with clinicians about its expanding portfolio of products for rare diseases, including the first and only ready-to-use liquid vigabatrin, VIGAFYDE™ (vigabatrin) Oral Solution. VIGAFYDE™ is indicated for infantile spasms (IS) as monotherapy in infants 1 month to 2 years of age for whom the potential benefits outweigh the risk of vision loss. "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™ (everolimus) Tablets, and 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 booth during this year's AAN meeting to learn more." About Upsher-Smith's Rare Disease Portfolio Upsher-Smith's rare disease portfolio continues to grow with the addition of high-quality medications that match or exceed the level of service provided by innovator brands. The Company offers three vigabatrin formulations including VIGAFYDE™ Oral Solution, VIGADRONE® Powder for Oral Solution and VIGADRONE® Tablets. VIGAFYDE™, the only ready-to-use vigabatrin oral solution, is the first new drug application (NDA) approved for infantile spasms in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ represents a welcome expansion of vigabatrin treatment options, offering patients and caregivers a simpler dose preparation process which may improve dosing accuracy. Upsher-Smith representatives will be available throughout the conference at exhibit booth #846 to engage with clinicians about their products including VIGAFYDE™, and VIGADRONE® and the Promise of Support™ Program, which has been providing comprehensive support to patients prescribed VIGADRONE® since 2018. Representatives will also be available to discuss Upsher-Smith's TORPENZ™ Tablets, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC). TORPENZ™, 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. 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. WHAT IS VIGAFYDE? VIGAFYDE™ (vigabatrin) Oral Solution is a prescription medicine used to treat: Infantile Spasms (IS) in babies 1 month to 2 years of age, if you and your baby's healthcare provider decide the possible benefits of taking VIGAFYDE are more important than the possible risk of vision loss. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGAFYDE™ (vigabatrin)? Because VIGAFYDE 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 baby's healthcare provider will explain the details of this Program to you. VIGAFYDE 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, your baby may only be able to see things straight in front of them (sometimes called "tunnel vision"). Your baby may also 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. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your baby's vision before or within 4 weeks after starting VIGAFYDE, and at least every 3 months during treatment until VIGAFYDE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGAFYDE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your baby's 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 VIGAFYDE, but they do allow VIGAFYDE 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 babies, until it is severe. If you do not have these vision tests regularly, your baby's healthcare provider may stop prescribing VIGAFYDE. Your baby may not be able to complete vision testing. If vision testing cannot be done, the healthcare provider may continue prescribing VIGAFYDE 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 VIGAFYDE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in postmortem examination of infants treated with vigabatrin. Risk of suicidal thoughts or actions. VIGAFYDE is approved for use in patients 1 month to 2 years of age [see WHAT IS VIGAFYDE?]. VIGAFYDE is not approved for use in adolescents or adults. Like other antiepileptic drugs, vigabatrin may cause suicidal thoughts and actions in some adolescents and adults (about 1 in 500 people). A healthcare provider should be called right away if a patient taking vigabatrin experiences any symptoms, especially sudden changes in mood, behaviors, thoughts, or feelings, and especially if they are new, worse, or worrying. Do not stop VIGAFYDE without first talking to a healthcare provider. Stopping VIGAFYDE suddenly can cause seizures that will not stop. VIGAFYDE is more concentrated than vigabatrin solutions prepared from powder. The volume of VIGAFYDE solution prescribed by your baby's healthcare provider may be less than the volume prescribed for other vigabatrin solutions prepared from powder. Always double check the volume (mL) that you are to give your baby when you get a new prescription filled. VIGAFYDE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, and weight gain. VIGAFYDE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGAFYDE, tell your baby's doctor about all of their medical conditions including any allergic reaction to vigabatrin, vision problems, kidney problems, or low red blood cell counts (anemia). Tell your doctor about all the medicines you baby is receiving. The most common side effects of VIGAFYDE 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 your baby has any side effect that bothers them or that does not go away. This is the most important information to know about VIGAFYDE, but not all of the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGAFYDE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGAFYDE.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. 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 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 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. 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 . VIGADRONE, TORPENZ 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 Temsirolimus

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KEGG	Wiki	ATC	Drug Bank
D06068	Temsirolimus	L01XE09	DB06287

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Advanced Renal Cell Carcinoma	South Korea	Pfizer Inc.	24 Oct 2008
Mantle-Cell Lymphoma	European Union	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Iceland	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Liechtenstein	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Norway	Pfizer Europe MA EEIG	19 Nov 2007
Renal Cell Carcinoma	United States	PF Prism CV	30 May 2007

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Locally advanced breast cancer	Phase 3	United States	Wyeth AB	01 May 2004
Locally advanced breast cancer	Phase 3	Canada	Wyeth AB	01 May 2004
Advanced Hepatocellular Carcinoma	Phase 2	United States	Pfizer Inc.	05 Oct 2012
Advanced cancer	Phase 2	Canada	Pfizer Inc.	09 Feb 2012
B-cell lymphoma refractory	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
B-cell lymphoma refractory	Phase 2	United States	Pfizer Inc.	01 Feb 2011
Multiple Myeloma	Phase 2	United States	Pfizer Inc.	01 Feb 2011
Multiple Myeloma	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
Non-Hodgkin's lymphoma refractory	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
Non-Hodgkin's lymphoma refractory	Phase 2	United States	Pfizer Inc.	01 Feb 2011

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01552434 (Pubmed \| Oncologist)	Phase 1	Ovarian Cancer \| Neoplasms	48	Bevacizumab + Temsirolimus	dxxlmxstgq(uqasexdzax) = qgewoncavs doldvquyae (ogevsmutqc ) View more	Positive	13 Dec 2025
				Bevacizumab + Temsirolimus (ovarian)	dxxlmxstgq(uqasexdzax) = bpwmxsuurg doldvquyae (ogevsmutqc ) View more
NCT01529593 (Phase I study of temsirolimus in combination with metformin, Pubmed \| Gynecol Oncol)	Phase 1	Recurrent Endometrial Cancer PI3K/AKT \| Ras/Raf/MEK/ERK pathways	-	Temsirolimus + Metformin	whabmyxinq(gpowmazffn) = reported in 32 patients qplnwzihff (vrnunjzfsi ) View more	Negative	01 Feb 2025
NCT Neuro Master Match (N2M2) (SNO2024)	Phase 1/2	CDK4 amplification \| CDKN2A/B codeletion \| mTOR activation	301	Temozolomide	gritxmmnpr(kexupknnvz) = vfeyjgwxmw hpbpbryrwx (eferenikjp ) View more	Positive	11 Nov 2024
				Asunercept	gritxmmnpr(kexupknnvz) = gjhunqxfoj hpbpbryrwx (eferenikjp ) View more
NCT03158389 (N2M2/NOA-20, ASCO2024)	Phase 1/2	Glioblastoma alectinib \| idasanutlin \| palbociclib ... View more	301	Palbociclib	sezyywgwbt(dqlmgcazue) = oxjpqxozbp knfuoaqquf (fjwhbaezwx ) View more	Positive	24 May 2024
				Temsirolimus	sezyywgwbt(dqlmgcazue) = nzxwiawnsn knfuoaqquf (fjwhbaezwx ) View more
NCT01010126 (phase II study of bevacizumab and temsirolimus in advanced extra-pancreatic neuroendocrine tumors, Pubmed \| Endocr Relat Cancer)	Phase 2	Extra-pancreatic neuroendocrine tumor	-	Temsirolimus 25 mg	peozezydxk(loaxusrdyw) = kxfybdpogn ourynmijcm (euzpnjprwy ) View more	Negative	01 Nov 2023
				Bevacizumab 10 mg/kg	oxmsyqbcpg(mtdzukpzdw) = wdqiuvxmtw lwchledsag (ytsrjworyo )
NCT01517243 (CTgov)	Phase 2	Metastatic Renal Cell Carcinoma	37	Sunitinib+Temsirolimus	autcuyoxey(vviwplemuo) = lbkjzjmvyf tziewrajxi (jyjuthducg, ngtjtiyhlp - cosgoddtjm) View more	-	21 Sep 2023
NCT01614197 (TACL 2014-001, CTgov)	Phase 1	Recurrent Adult Acute Lymphoblastic Leukemia \| Peripheral T-Cell Lymphoma \| Adult Lymphoblastic Lymphoma	16	Etoposide+Cyclophosphamide+Hydrocortisone+Methotrexate+Cytarabine+Temsirolimus (Dose Level 1)	oosvmvgdcx = jxppvbecyk okjctkqbty (gseyzhpjya, hnydlhaxvp - vsucjtujjm) View more	-	27 Jul 2023
				Etoposide+Cyclophosphamide+Hydrocortisone+Methotrexate+Cytarabine+Temsirolimus (Dose Level 2)	oosvmvgdcx = rhexhxylou okjctkqbty (gseyzhpjya, ogbjkfeifp - cdkgzxnzia) View more
NCT01552434 (Pubmed)	Phase 1	Advanced Malignant Solid Neoplasm	47	Bevacizumab	hpjjibhzxz(liivibacvk) = grade 3 infection, rash, mucositis, bowel perforation, elevated lipase, and grade 4 cerebrovascular ischemia ojishrmpnl (ebpxskhurf ) View more	-	13 Jun 2023
NCT02693535 (TAPUR, ASCO 12023 \| ASCO 22023) Manual	Phase 2	PIK3CA H1047R mutant Solid Tumors PIK3CA Mutation	41	Temsirolimus (BC: pts with breast cancer)	dzrardelio(iwgfwqlepq) = lwbzrcyqzi oqfxbyqnwb (tbgsoxgmab, 0.95 - 100) View more	Negative	26 May 2023
				Temsirolimus (HP: Low accruing histology-specific cohorts with PIK3CA and T tx were collapsed)	dzrardelio(iwgfwqlepq) = hijcrstjen oqfxbyqnwb (tbgsoxgmab, 17 - 100) View more
NCT00980460 (AHEP0731, Pubmed \| Pediatr Blood Cancer)	Not Applicable	Hepatoblastoma	36	Vincristine/irinotecan/temsirolimus (VIT)	jyawavoerc(uqcchtypdn) = cdiacpmcjj kpubytbqmp (ryzoldxyqy ) View more	Negative	19 Apr 2023

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