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]).

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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.

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

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On January 25, 2023 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL RNAi platform technology is designed to make immune cells more effective in killing tumor cells, reported that the Company’s Board of Directors has approved a reverse stock split of its shares of common stock at a ratio of 1-for-12 (Press release, Phio Pharmaceuticals, JAN 25, 2023, View Source [SID1234626549]). The reverse stock split will become effective at 12:01 a.m. Eastern Time on January 26, 2023 and the Company’s common stock will open for trading on The Nasdaq Capital Market on a post-split basis on January 26, 2023 under the Company’s existing trading symbol, "PHIO." At such time, the Company’s common stock will also commence trading with a new CUSIP number, 71880W402.

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The reverse stock split is being implemented to increase the per share trading price of the Company’s common stock, for the purpose of ensuring a share price high enough to comply with the minimum $1.00 bid price requirement for continued listing on The Nasdaq Capital Market.

At the effective time of the reverse stock split, every twelve (12) shares of Phio common stock issued and outstanding will be combined into one (1) share of common stock issued and outstanding, with no change to the par value of $0.0001 per share. This will reduce the Company’s outstanding common stock from approximately 13.7 million shares to approximately 1.1 million shares. No fractional shares of common stock will be issued as a result of the reverse stock split and instead holders of Phio common stock will receive a cash payment in lieu of fractional shares to which they would otherwise be entitled. The shares underlying the Company’s outstanding equity awards and warrants will also be adjusted accordingly. The reverse stock split affects all stockholders uniformly and will not alter any stockholder’s percentage interest in the Company’s common stock, except for adjustments that may result from the treatment of fractional shares.

The Company has retained its transfer agent, Computershare Trust Company, N.A. ("Computershare"), to act as its exchange agent for the reverse split. Shareholders with shares held in certificate form will receive from Computershare instructions regarding the exchange of their certificates. Shareholders that hold shares in book-entry form or hold their shares in brokerage accounts are not required to take any action and will see the impact of the reverse stock split reflected in their accounts, subject to brokers’ particular processes. Beneficial holders of Phio common stock are encouraged to contact their bank, broker, custodian or other nominee with questions regarding procedures for processing the reverse stock split.

Additional information regarding the reverse stock split is available in the definitive proxy statement filed with the U.S. Securities and Exchange Commission on November 30, 2022 by the Company.

On January 25, 2023 Onxeo S.A. (Euronext Growth Paris: ALONX), hereafter "Onxeo" or the "Company", a clinical-stage biotechnology company specializing in the development of innovative tumor specific drugs targeting tumor DNA Damage Response (DDR) and driver oncogenes, reported an update on the clinical development program of its first-in-class drug candidate AsiDNATM (Press release, Onxeo, JAN 25, 2023, View Source [SID1234626548]).

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Onxeo activated its first US clinical study site, Next Oncology San Antonio. This phase 1b/2 multicenter, basket trial intends to assess the safety and preliminary activity of AsiDNATM in combination with olaparib in patients with recurrent ovarian, breast and metastatic castration-resistant prostate cancer (mCRPC) who have progressed on previous PARP inhibitor therapy. The primary endpoint of the study will assess the safety and tolerability of the combination as well as to determine the recommended Phase 2 dose. Key secondary endpoints will assess the preliminary activity and duration of response for the combination.

Shefali Agarwal, Chairwoman of the Board of Directors and CEO, said: "We are delighted with the initiation of this important clinical trial in the US to further explore the potential of our first-in-class drug candidate, AsiDNATM. This investigational product has been in clinical development in Europe for the last few years, in recurrent solid tumors. In clinical studies AsiDNATM appears to be well tolerated with encouraging clinical activity in the studied patients till date. The activation of this first study in the US is an important next step towards its global clinical development.

The recent encouraging activity observed from the preliminary data in the REVOCAN study indicates the potential of AsiDNATM to re sensitize patients to PARP therapy which potentially addresses an unmet need and could meaningfully impact patients living with recurrent ovarian cancer who have progressed on an initial treatment with a PARP inhibitor. Additionally, this lays a strong foundation for our next first in class drug candidate OX425 which is sourced from the same proprietary PlatON platform and is a PARP/DDR specific decoy agonist thereby possibly not inducing tumor resistance to treatment. This profile represents a potential differentiation in safety and activity from other targeted therapies such as PARP inhibitors and we are on track to file an IND in mid- 2023".

The REVOCAN study is an open label, multicenter, phase 1b/2 study evaluating the safety and efficacy of AsiDNATM, in combination with PARP inhibitors in patients with relapsed platinum sensitive ovarian cancer already under treatment with a PARP inhibitor. The study is sponsored by Gustave Roussy Cancer Campus, Grand Paris, led by Dr Patricia PAUTIER and supported by ONXEO. The study team recently completed its first interim analysis (IA) of 10 patients. The combination of AsiDNATM and PARPi was generally well tolerated with no new safety signals or dose limiting toxicities. The IA also demonstrated encouraging clinical activity with six patients achieving a stable disease (SD) and one patient demonstrating a complete response (CR) with disease control rate of around 70%. The study continues to enroll patients. The detailed results of the IA will be published by the investigator.

Additionally, AsiDNATM is being evaluated in Children and young adults with recurrent high-grade glioma (HGG). This phase 1b/2 trial, sponsored by Institut Curie, is being conducted within the framework of the European ITCC consortium. The trial is evaluating the safety and clinical activity of AsiDNATM in combination with radiotherapy in children or young adults with recurrent HGG. The trial has already been opened at 8 clinical trial sites in France and 5 patients have been enrolled. To date, the combination has been well tolerated. Further trial site activation is planned for 2023 in Italy, the Netherlands, and Germany.