On January 25, 2023 STORM Therapeutics Ltd. (STORM), the clinical stage biotechnology company discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases, reported new data on its lead candidate, the METTL3 inhibitor STC-15 (Press release, STORM Therapeutics, JAN 25, 2023, View Source [SID1234626558]). The new data in AML preclinical models was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference: Acute Myeloid Leukemia and Myelodysplastic Syndrome, hosted in Austin, Texas on the 23-25 January.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation entitled ‘STC-15, a novel METTL3 inhibitor, and its combination with venetoclax confer anti-tumour activity in AML models’ detailed the study of the pharmacological inhibition of METTL3 as monotherapy or in combination with venetoclax (FDA approved for AML treatment) in models of acute myeloid leukemia (AML) in vitro and in vivo.

Preclinical data demonstrated that:

STC-15 inhibited proliferation in some AML cell lines with sub- micromolar IC50 values
STC-15 inhibited the growth of 12 patient-derived AML samples in vitro with IC50 values reflecting a mean of approximately 1 micromolar
STC-15 reduced BCL2 protein levels in a dose-dependent manner in the majority of AML cell lines tested
STC-15 showed synergistic inhibition of tumor cell growth in vitro when combined with venetoclax
In an AML patient-derived in vivo model, STC-15 extended survival when compared to a vehicle-treated control group and a venatoclax-treated group of animals
In addition, STC-15 showed a decrease in circulating human CD45+ cells and decreased spleen weight when compared to vehicle treated animals
STORMS lead candidate STC-15 is advancing its ongoing Phase 1 study. The details of the study can be found on clinicaltrials.gov under the identifier NCT05584111.

Oliver Rausch, Chief Scientific Officer of STORM Therapeutics, said: "These studies provide evidence for the utility of METTL3 inhibitors as a new therapeutic approach to treat AML. We are delighted with the outcome of the data which further validates our previous publication that treatment with METTL3 inhibitors led to the downregulation of BCL2 protein levels in several AML cell lines and in vivo models and provides the rationale for conducting a clinical trial in AML with STC-15."

Jerry McMahon, Chief Executive Officer & President of STORM Therapeutics, said: "We are excited to present this new data with patient-derived tumor samples supporting the future clinical development of STC-15 in AML. Our ongoing Phase 1 multiple-ascending dose study in solid tumors is focused on establishing a potential dose and regimen of STC-15 to conduct future clinical studies in AML and solid tumors."

Details of the conference and presented poster are as follow:

Poster Presented Title: STC-15, a novel METTL3 inhibitor, and its combination with venetoclax confer anti-tumour activity in AML models

Presenting Authors: Lina Vasiliauskaitė1, Yaara Ofir-Rosenfeld1, Mark Albertella1, Coralie Hoareau-Aveilla2, Jerry McMahon1, Oliver Rausch1

Date and Time: Tuesday, 24 January, 07.15 – 21.30 ET

1Storm Therapeutics Ltd., Cambridge, UK

2Evotec SAS, Toulouse, FR

The Poster and Abstract are available on the STORM Therapeutics website: Publications.

STORM has developed potent and selective METTL3 inhibitors which include the first-in-class clinical candidate STC-15, an orally bioavailable, highly selective METTL3 inhibitor which commenced a clinical trial in cancer patients with solid tumors in November 2022.

On January 25, 2023 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it will host a webcast and conference call on February 6, 2023, at 4:30 p.m. ET to discuss its financial results for the fiscal 2023 first quarter ended December 31, 2022 (Press release, Arrowhead Pharmaceuticals, JAN 25, 2023, View Source [SID1234626557]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Webcast and Conference Call and Details

Investors may access a live audio webcast on the Company’s website at View Source A replay of the webcast will be available approximately two hours after the conclusion of the call.

For analysts that wish to participate in the conference call, please register at https://register.vevent.com/register/BI9b2661110f7a4b0ebc648bf42d2a403f. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.

On January 25, 2023 Oncotelic Therapeutics, Inc (OTCQB:OTLC) ("Oncotelic", the "Company" or "We"), a clinical stage biotechnology company, with a planned IPO of its JV this year, reported that it has submitted a clinical study protocol to the US Food and Drug Administration ("FDA") for the initiation of a Phase 2b/3 Trial (designated "P201") for OT-101, the Company’s transforming growth factor beta 2 ("TGF-β2") inhibitor, as a treatment for metastatic pancreatic cancer (Press release, Oncotelic, JAN 25, 2023, View Source [SID1234626556]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

P201: A Randomized Phase 2b/Phase 3 Study of the TGF-β2 Targeting Antisense Oligonucleotide OT-101 in Combination with FOLFOX Compared with FOLFOX Alone as Second-Line Therapy in Patients with Metastatic Pancreatic Cancer that has Progressed During or Following a First-Line Gemcitabine-Containing Regimen.

OT-101 is a first-in-class anti-TGF-β2 ribonucleic acid ("RNA") therapeutic that has exhibited single agent activity in relapsed/refractory cancer patients in multiple clinical trials. OT-101 has also demonstrated activity against the COVID-19 virus in our Phase 2 clinical trial- C001.

"Pancreatic cancer is the fourth leading cause of cancer-related mortality with a 5-year survival rate of approximately 10%. Incidence of pancreatic cancer has been steadily increasing despite advances in immunotherapy. Many well known names have succumbed to pancreatic cancer including Apple’s Steve Jobs and Justice Ruth Bader Ginsburg " said Dr. Vuong Trieu, CEO Oncotelic. "We are initiating late-stage clinical trials around pancreatic cancer and are excite to work with leading thought leaders to bring OT-101 to patients"

About OT-101 Pancreatic Cancer Program

Pancreatic cancer is associated with the poorest prognosis of gastrointestinal cancers and is expected to become the second leading cause of cancer-related mortality in the USA by 2030. Globally, over 400,000 people die of pancreatic cancer each year. Pancreatic cancer is traditionally considered to be an immune-resistant disease. There is a lack of effector T cells, an abundance of myeloid-derived suppressor T cells, and a dearth of key immune effector and regulatory cells. This may be part of the reason why single-agent checkpoint inhibitors are not as effective in comparison to other diseases. Here is where breaking immune tolerance by inhibiting TGF-β with OT-101 will have a significant impact.

The P001 trial was an open-label, multicenter dose-escalation study to evaluate the safety and tolerability of OT-101 (TGF-β2-specific Phosphorothioate Antisense Oligodeoxynucleotide) in adult patients with advanced tumors known to overproduce TGF- β2, which are not or no longer amenable to established therapies. Of the 61 patients treated, 37 had advanced treatment failure pancreas cancer, a very difficult-to-treat cancer with an overall survival rate that is measured in months even with the best available chemotherapy regimens. Disease control (complete response (CR)), partial response (PR) or stable disease (SD)) was achieved in 19 of 35 evaluable pancreas cancer patients (54%). Among liver mets only patients, there are exceptional single-agent activity and survival. Patient 1006 was pushed to complete response (CR) and survived as far out as 77 mos. This patient failed multiple lines of therapies: (1) surgery: Whipple’s procedure, (2) 1st line: 5-FU/LV, Dose 425 mg/m2, (3) 2nd line: 5-FU/LV, Dose 2600 mg/m2/24hr, (4) 3rd line: Gemcitabine, Dose 1000 mg/m2/week, and (5) went on to OT-101 with liver mets and complete response. Patient 1022 was pushed to stable disease ("SD") with overall survival of 40 months. This patient had also failed multiple lines of therapies: (1) surgery: Whipple’s procedure, (2) 1st line: radiation therapy (50 Gy), (3) 2nd line: 5FU, and (4) went on to OT-101 with liver mets and SD.

About OT-101

OT-101, is a first-in-class anti-TGF-β2 RNA therapeutic that exhibited single agent activity in some relapsed/refractory cancer patients in clinical trial settings. HGGs are characterized by a T-cell exhaustion signature and pronounced T-cell hypo responsiveness of their tumor microenvironment ("TME"). TGF-β2 has been implicated as a key contributor to the immunosuppressive landscape of the TME in HGG. OT-101 is designed to abrogate the immunosuppressive actions of TGF- β2. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult HGG patients, including young adults with Glioblastoma Multiforme or Amyloidosis.

OT-101 has been granted orphan designation by the FDA under the Orphan Drug Act ("ODA"). ODA provides for granting special status to a drug to treat a rare disease or condition upon request of a drug company. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. OT-101 also been granted Rare Pediatric Designation for DIPG. The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.

As previously reported, on March 31, 2022, we entered into a joint venture, or JV, with Dragon Overseas Capital Ltd. (Dragon Overseas) and GMP Biotechnology Ltd. (GMP Bio). The JV and Oncotelic will develop and ultimately market OT-101, individually and/or in combination with other products. Oncotelic would receive up to $50 million on sale of the RPD voucher, following marketing approval of OT-101 for diffuse intrinsic pontine glioma, or DIPG, by the US Food and Drug Administration.

On January 25, 2023 Magenta Therapeutics (Nasdaq: MGTA) reported that the latest participant dosed at the Cohort 3 level (0.08 mg/kg) in the ongoing MGTA-117 Phase 1/2 Dose-Escalation Clinical Trial in relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) experienced a Grade 5 Serious Adverse Event (SAE) (respiratory failure and cardiac arrest resulting in death) deemed to be possibly related to MGTA-117 (Press release, Magenta Therapeutics, JAN 25, 2023, View Source [SID1234626555]). The known information has been reported to the U.S. Food and Drug Administration (FDA) as a Suspected Unexpected Serious Adverse Reaction (SUSAR). After consultation with the trial’s safety Cohort Review Committee and with the highest regard for patient safety, Magenta has voluntarily paused dosing in the clinical trial and is working to evaluate the totality of available data and determine next steps for the development of MGTA-117.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On January 25, 2023 QSAM Biosciences presenting its Corporate Presentation (Filing, 8-K, QSAM Biosciences, JAN 25, 2023, View Source [SID1234626552]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!