rilpivirine oral suspension (SACT-1) / Aptorum Group - LARVOL DELTA

DIsease burden and treatment pattern of Recurrent or advanced Endometrial cancer in Korea: A naTIONwide database (DIRECTION) study (ESMO Asia 2024) - "EC-related cost, mostly inpatient cost, increased from 1,048 USD to 1,980 USD PPPM from SACT1 to 3 Table: 375O Top 3 regimens in the 1st- to 3rd-line of SACT SACT regimens (%) 1 st line (N=2,704) 2 nd line (N=837) 3 rd line (N=305) 1 T+P (42.0) D+P (25.7) Other P-based combinations a (31.8) 2 D+P (17.5) T+P (23.1) D+P (14.4) 3 P (16.0) Other P-based combinations a (21.2) T+P (14.4) 4 Others b (24.5) Others c (30.1) Others d (39.3) a P-based combinations other than T+P or D+P...1.0% T+P, taxanes+platinum; D+P, doxorubicin+platinum; P, platinum. . Conclusions Our study reveals an increase in HCRU and costs and a decrease in treatment duration and SACT-free interval with increased line of SACT, indicating a worsening prognosis as EC progresses. Thus, the application of appropriate treatments, including new therapeutic agents, in newly diagnosed a/r EC patients is important."

Metastases • Endometrial Cancer • Oncology • Solid Tumor

Aptorum Group Updates on a Phase 1b/2a Clinical Trial for Repurposed Small Molecule Drug SACT-1 for Neuroblastoma (Businesswire) - "Aptorum Group Limited...announced that the group has submitted the relevant Phase 1b/2a clinical trial protocol of SACT-1, an orally administered repurposed small molecule drug for the treatment of neuroblastoma to US FDA. The Phase 1b/2a study of SACT-1 submitted is for the combination with chemotherapy for first relapse or refractory high risk neuroblastoma....The targeted objectives of the Phase 1b part of the study based on neuroblastoma patients to be enrolled is to determine the recommended phase 2 dose (RP2D) based on safety, pharmacokinetics and efficacy and the Phase 2a part of the study based on neuroblastoma patients to be enrolled will be used to assess the preliminary efficacy of SACT-1. We will work closely with the regulatory agency in our journey towards the approval of our drug."

Clinical protocol • FDA event • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Announces Completion of End of Phase 1 (EOP1) Meeting with US FDA on its SACT-1, a Repurposed Small Molecule Drug Targeting Neuroblastoma (Businesswire) - "Aptorum Group Limited...announces the completion of an end of Phase 1 (EOP1) meeting with the US Food and Drug Administration ('US FDA'). SACT-1 is a repurposed small molecule drug formulated in oral suspension targeting neuroblastoma in pediatric patients and has also received orphan drug designation previously from the US FDA. The EOP1 meeting was focused on gaining alignment with the US FDA regarding the clinical and regulatory pathway for SACT-1 for the treatment of neuroblastoma in pediatric patients aged 2-18. The FDA generally agreed with the chemistry-manufacturing-control (CMC) strategy and our proposed clinical development plan for Phase 1/2 trials."

FDA event • Neuroblastoma • Oncology • Solid Tumor

A Study of Bioavailability and Food Effect of SACT-1 and Edurant® Tablets in Healthy Adult Volunteers (clinicaltrials.gov) - P1 | N=16 | Completed | Sponsor: Aptorum International Limited

New P1 trial

Aptorum Group Updates on Data from the Completed Phase 1 Clinical Trial of SACT-1, targeting neuroblastoma (Businesswire) - P1 | N=NA | "The study treatments were well tolerated and no subjects were discontinued from study participation because of adverse events. No serious adverse events were reported during the study. The phase 1 clinical data also suggested that any QT interval after oral administration of SACT-1 at 150mg was well within clinically acceptable limits....'We are planning to meet with the US FDA for an end of Phase 1 meeting as soon as possible and are targeting for submission for a Phase 1b/2a clinical trial in neuroblastoma patients'."

FDA event • New P1/2 trial • P1 data • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Receives FDA Orphan Drug Designation for its SACT-1 Repurposed Drug For The Treatment of Neuroblastoma (Businesswire) - "Aptorum Group Limited...is pleased to announce that the United States Food and Drug Administration (FDA) Office has granted Orphan Drug Designation to SACT-1, a repurposed small molecule compound for the treatment of patients with Neuroblastoma."

Orphan drug • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Granted The First Patent for its SACT-1 Repurposed Drug For Treatment of Various Cancer Including but Not Limited to Neuroblastoma (Businesswire) - "Aptorum Group Limited...is pleased to announce that the US Patent and Trademark Office has granted the first patent regarding Aptorum’s SACT-1 repurposed drug for the treatment of various cancers including but not limited to neuroblastoma (US Patent 11,166,962 B2)....The composition is administered alone or in combination with one or more chemotherapeutic agents, biological agents, and/or anticancer agents by various routes of administration and dosage forms."

Patent • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Announces Completion of Phase I Clinical Trials for ALS-4 and SACT-1, Small Molecule Drugs Targeted for Infections Caused by Staphylococcus Aureus and Neuroblastoma (Businesswire) - "Aptorum Group Limited...announces completion of the Phase I clinical trial for...assessing relative bioavailability and food effect of SACT-1 (a repurposed small molecule drug targeting Neuroblastoma and potentially other cancer types)....Aptorum is pleased to announce the successful completion of the trial, during which no SAE were observed. With the encouraging data in our trial so far, we are on track to submit an IND application to the US FDA this year seeking to initiate our planned Phase 1b/2a trial for SACT-1."

IND • Trial status • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Limited Has Received IND Clearance From the US FDA to Initiate Clinical Trials for Repurposed Small Molecule Drug SACT-1 for the Treatment of Neuroblastoma (Businesswire) - “Aptorum Group Limited…announced that the group has received clearance from the US FDA regarding the IND application to initiate clinical trials of SACT-1, an orally administered repurposed small molecule drug for the treatment of neuroblastoma....Based on the prior recommendations provided by the US FDA in our Pre-IND meeting, the IND-opening clinical trial, a bioavailability/food effect study which is believed to last for approximately four months, will be conducted in the United States, followed by a planned Phase 1b/2a trial in pediatric patients suffering from relapsed or refractory high-risk neuroblastoma, subject to further clearance by the US FDA. The objective of the bioavailability/food effect study is to compare the relative bioavailability of the newly developed SACT-1 pediatric formulation in healthy adult subjects.”

IND • Neuroblastoma • Oncology

[VIRTUAL] Aptorum Group Limited (BIO 2021) - "In particular, Aptorum Group is currently developing therapeutics in the areas of oncology, infectious diseases and women’s health. Targeted development milestones for some of the key programs include: ALS-4 (next-generation anti-infective for Staphylococcus aureus including MRSA via a non-bactericidal antivirulent approach): a new chemical entity (NCE) small molecule drug currently in a Phase 1 clinical trial, targeted for potential administration both on a standalone or a combined basis with other existing antibiotics such as vancomycin; SACT-1 (neuroblastoma & other solid tumours): a repurposed drug programme with prior human safety and toxicity data available, in preparation to open an IND with US FDA to commence clinical studies in 2021; (cont.)"

Infectious Disease • Neuroblastoma • Oncology • Solid Tumor • Women's Health

Aptorum Group Announces Positive Interim Phase I Clinical Trial Progress for ALS-4 Targeting Staphylococcus aureus and Positive Pre-IND Progress for SACT-1 Targeting Neuroblastoma (Businesswire) - “Aptorum Group…announces positive progress in respect of…its ongoing Pre-IND preparation for SACT-1, a repurposed small molecule targeting neuroblastoma, under which the Pre-IND meeting with the US FDA has been completed…SACT-1…has completed its Pre-IND meeting with the US FDA. With the guidance provided by the US FDA, SACT-1 is on track to open an IND to commence clinical studies in quarter 3 of 2021.”

IND • Trial status • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Limited Reports 2020 Fiscal Year End Financial Results and Provides Business Update (Businesswire) - “Aptorum Group completed a Pre-IND meeting with US FDA with encouraging outcomes regarding SACT-1, another lead program employing an orally administered small molecule repurposed drug for the treatment of neuroblastoma. The Company is now on track in its preparation to open an IND with US FDA for the commencement of clinical trials in 2021.”

IND • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group Announces Further Positive Data on SACT-1 Against Neuroblastoma and Other Potential Tumor Types (Businesswire) - "Aptorum...announced further positive data from its latest in vivo studies showing significant activity against neuroblastoma tumor reduction when treated with its lead compound SACT-1 in combination with standard of care (SOC) chemotherapy. Separately, SACT-1 was also screened for its in vitro activity against over 300 cancer cell lines and showed positive results in a number of cancer types including in particular colorectal cancer, leukemia and lymphoma, etc...SACT-1 is undergoing preparation for IND submission and is on track for regulatory application to target to commence phase 1b/2a clinical trials under the US FDA’s 505(b)(2) pathway."

IND • New P1/2 trial • Preclinical • Colorectal Cancer • Gastrointestinal Cancer • Hematological Malignancies • Leukemia • Lymphoma • Neuroblastoma • Oncology

Aptorum Group Appoints Dr. Herman Weiss as CEO of microbiome-based platform Claves Life Sciences and Dr. Robbie Majzner as scientific advisor to SACT-1 drug for Neuroblastoma and others (Businesswire) - "...Dr. Majzner will provide scientific advice and support for certain targeted clinical development aspects of Aptorum Group’s repurposed drug candidate SACT-1 for the treatment of neuroblastoma, a rare type of solid tumor cancer that develops predominantly in infants and young children, as well as other potential cancer indications."

Clinical • Neuroblastoma • Oncology • Solid Tumor

Aptorum Group announces significant progress of repurposed drug candidate, SACT-1 for neuroblastoma targeting IND submission in H2 2020 (Businesswire) - "Aptorum Group Limited...announces positive data and development in relation to its repurposed drug candidate, SACT-1, for the treatment of neuroblastoma...Subject to completion of current validation studies, Aptorum Group plans to leverage the 505(b)(2) pathway and submit an IND submission with the FDA for SACT-1 in H2 2020....SACT-1 has been shown to be effective against numerous neuroblastoma cell lines, of which 2 are MYCN-amplified cells, which represent the high-risk neuroblastoma patient group. In addition, by using a combination index as a quantitative measure of the extent of drug interaction"

IND • Preclinical