On April 19, 2021 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company") (OTCQB:OTLC) reported a presentation at AACR (Free AACR Whitepaper)-2021 (American Association for Cancer Research/ Annual Meeting) entitled: Combination therapy of anti-sense oligonucleotide targeting TGF-β2 (TASO) and IL-2 (Proleukin) has anti-cancer effect in solid cancer (Press release, Oncotelic, APR 19, 2021, View Source [SID1234578197]).

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TGF-β (transforming growth factor-beta) is an essential cytokine for tumor proliferation and metastasis. The expression of TGF-Β correlates with malignancy of various cancers and involves immunosuppression and angiogenesis of a tumor. IL-2 is a major cytokine to proliferate T cells and NK cells which are major players of cancer immunity. However, the toxicity of high dose IL-2 limits its use in cancer therapy. Combination treatment of TGF-β inhibitor and IL-2 would have an anti-tumor effect by immune cells through diminishing immunosuppression by TGF-β and enforcement of immune cells by IL-2. Trabedersen is an anti-sense oligonucleotide targeting human TGF-β mRNA. It is shown that Trabedersen is well tolerable in cancer patients and effective reagent to treat pancreatic cancer, melanoma, and glioblastoma. Proleukin is the only approved IL-2 reagent to treat Renal cell carcinoma and Melanoma.

Trabedersen and Proleukin activated human PBMC (Peripheral Blood Mononuclear Cell) are treated to several solid cancer cell lines, such as Breast cancer, Pancreatic cancer, Melanoma, Lung cancer, and Colon cancer to see the cytotoxicity effect of combination therapy of Trabedersen and Proleukin. NSG mouse (NOD Scid Gamma mouse, NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) which are humanized by human PBMC engraftment and the tumor growth of Melanoma and TNBC (Triple Negative Breast Cancer cell) cell line are monitored.

The combination treatment of Trabedersen and low dose Proleukin decreased cancer cell viability in vitro experiment in solid cancer cell lines. Melanoma and TNBC tumor growth was delayed in humanized NSG mouse model by Trabedersen and low dose IL-2 combination therapy and tumor growth delay was statistically significant to Trabedersen alone or IL-2 alone group. Tumor infiltrating lymphocyte population was increased in Trabedersen treated group and FoxP3+ regulatory T cell population in blood and tumor microenvironment was decreased by treatment of Trabedersen.

TGF-β inhibitor (Trabedersen) and low dose IL-2 (Proleukin) combination treatment is expected to be an effective regimen in solid cancer treatment than individual treatment by alteration of tumor environment. Modulation of the dose of Proleukin expects to help reduce the toxicity of IL-2 and increase the anti-cancer effect by combination with Trabedersen.

Oncotelic has previously announced the regulatory approval from the Ministry of Food and Drug Safety of Korea for the phase 1b clinical trial of a patented OT-101/IL-2 combination. This phase 1b clinical trial will confirm the safety and effectiveness of OT-101/IL-2 in solid cancer patients in cooperation with the UK global pharmaceutical company Clinigen Group. The study will be conducted together with Autotelic BIO- a partner of Mateon on OT-101/IL-2 combination.

OT-101 has received orphan drug designation for glioblastoma, melanoma, and pancreatic cancer. Furthermore, FDA recently granted Rare Pediatric Designation for OT-101 against diffuse intrinsic pontine glioma (DIPG). OT-101 is also effective against coronavirus including COVID-19 and being deployed against the COVID-19 epidemic.

OT-101 has demonstrated robust efficacy against pancreatic cancer, glioblastoma, and melanoma during phase 2 clinical trials. The demonstration that OT-101 will synergize with IL-2 further demonstrate its utility as adjunct to other immunotherapies. Interleukin-2 (IL-2, Aldesleukin, PROLEUKIN) Immunotherapy is cancer treatment that stimulates the body’s immune system to fight cancer, such as melanoma.

On April 19, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that it will report its first-quarter 2021 financial results on Thursday, April 29, 2021 after the financial markets close (Press release, Vertex Pharmaceuticals, APR 19, 2021, View Source [SID1234578196]). The company will host a conference call and webcast at 5:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

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The conference call will be webcast live and a link to the webcast can be accessed through Vertex’s website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company’s website.

On April 19, 2021 Stand Up To Cancer (SU2C) and Mirati Therapeutics Inc. (NASDAQ: MRTX) – a late-stage targeted oncology company – reported that Mirati will contribute a $4 million grant to SU2C to develop new approaches to treat patients with KRAS mutant cancers, as a part of the SU2C Catalyst program (Press release, Stand Up To Cancer, APR 19, 2021, View Source;to-Support-Research-on-KRAS-Mutant-Cancers/default.aspx [SID1234578195]).

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The research will focus on cancer types with unmet medical needs, and the application of tumor-agnostic strategies and molecular testing to address those needs. Certain cancers can sometimes have mutations in the KRAS gene, which makes a protein involved in cellular growth and death. In KRAS mutant cancers, that protein can cause cancers to grow and spread. KRAS proteins were once considered undruggable, but recent research advances have resulted in the identification and development of investigational drugs against cancers that have specific KRAS mutations. Preliminary data from these investigational drugs have demonstrated early signs of clinical activity and studies are ongoing.

The grant from Mirati will leverage the SU2C Catalyst innovative research process, which uses funding and materials from the pharmaceutical, biotechnology, diagnostic and medical devices industries to accelerate research on cancer prevention, detection and treatment. The research will explore new treatment strategies with a KRASG12C inhibitor currently being developed by Mirati.

"The SU2C Catalyst program encourages a collaborative yet nimble and transparent process that accelerates the pace of research and identifies new or unexpected uses for cancer drugs and technologies," said Nobel laureate Phillip A. Sharp, PhD, chair of Stand Up To Cancer’s Scientific Advisory Committee and an institute professor at the David H. Koch Institute for Integrative Cancer Research at Massachusetts Institute of Technology. "Combined with our rigorous review and milestone-driven process, the program provides a unique framework with the objective to get treatments to patients as quickly and safely as possible."

SU2C’s Catalyst program began in 2016, with contributions from industry supporters Merck, Bristol Myers Squibb and Genentech, a member of the Roche Group. Through the program, companies donate funds to collaborative research studies, in which use of the companies’ products and materials is strongly encouraged. Those materials might include new pharmaceutical compounds that companies are developing or approved agents that can be investigated for other uses. A primary goal of SU2C Catalyst is to encourage collaborative research between academics and companies and shorten the time it takes to get new treatments to patients.

"We are excited to collaborate with Mirati Therapeutics and are thankful for their contribution to the SU2C Catalyst program," said Sung Poblete, PhD, RN, CEO of Stand Up To Cancer. "The opportunity to test the utility of KRAS inhibitors in several different cancer types is a great example of SU2C’s cancer-agnostic approach to research."

"We are proud to support the SU2C Catalyst program and the innovative research it makes possible," said Joseph Leveque, M.D., executive vice president and chief medical officer, Mirati Therapeutics, Inc. "So much progress has been made in understanding and treating cancers through collaborative research. Industry and academic scientists, working together, offer the best chance to move ideas forward that can make a real difference for patients."

As a part of the collaboration with Mirati, SU2C will convene a day-long innovation summit to bring together experts in KRAS signaling, clinical trial design, tumor agnostic approaches to cancer therapy development and other disciplines. The summit will be headed by members of SU2C’s scientific leadership team as well as Mirati representatives. These experts will consider the most pressing research needs in the field and define a grants process that provides a pathway for applicants, and the eventual grantees, to address those issues. Attention will be given to the current state of KRAS genetic testing in different cancer types and approaches that may be considered to appropriately expand testing both across tumor types and among traditionally underserved and minority populations.

On April 19, 2021 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of agents which includes checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) (Press release, Agenus, APR 19, 2021, View Source [SID1234578194]). The BLA has been submitted for the accelerated approval of balstilimab, Agenus’ anti-PD-1 antibody, for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy, and includes data from its pivotal Phase 2 single-arm clinical trial, presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020. These clinical data, along with preclinical data, suggest that balstilimab demonstrates differentiated features from other anti-PD-1 antibodies.

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"Women with recurrent or metastatic cervical cancer have a very poor prognosis and limited treatment options. Data suggest balstilimab may bring benefit to patients beyond what is available in this disease setting today," said Jennifer Buell, PhD, President and Chief Operating Officer at Agenus. "This submission also marks a significant step in our transition to a commercial company and the advancement of our oncology combination strategy."

The balstilimab BLA submission is based on an update to data presented at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020 and published in an Oncogene editorial, which demonstrate that balstilimab shows potential differentiation from other anti-PD-1 antibodies. This updated dataset includes maturation of late patient responses, with the overall data showing response rates of 20% in PD-L1 positive tumors, 15% in all tumors (PD-L1 positive and negative), and a median duration of response of 15.4 months.

"We expect that the potential approval of balstilimab will enable us to better pursue our oncology combination strategy for our own extensive pipeline of agents as well as for existing and future partner products," said Steven O’Day, MD, Chief Medical Officer at Agenus. "In particular, we hope to use this potential approval to allow us to rapidly proceed with our anti-CTLA-4 combination strategy, which we believe can add significantly to the benefit provided by our anti-PD-1 agent. There are currently limited treatment options available for recurrent or metastatic cervical cancer patients, and our vision is to bring effective treatments to these patients."

In April 2020, the FDA granted Fast Track designation for balstilimab in recurrent or metastatic cervical cancer based on its potential to provide benefit to patients with a serious condition and unmet medical need.

A global, randomized, Phase 3 confirmatory clinical trial designed to support global registration is planned.

About Cervical Cancer
Nearly 14,000 women are expected to be diagnosed with invasive cervical cancer in the United States this year and more than 4,000 are expected to die. Cervical cancer remains one of the leading causes of cancer death in women globally, annually killing more than 300,000 women worldwide.1 Despite advances in routine medical examinations and HPV vaccines, cervical cancer remains prevalent. When left undetected, recurrent or metastatic cervical cancer often develops, for which there are limited treatment options and a low chance of survival. Current therapies for recurrent or metastatic cervical cancer are limited to a small subset of patients with limited benefit.

About balstilimab
Balstilimab is a novel, fully human monoclonal immunoglobulin G4 (IgG4) designed to block PD-1 (programmed cell death protein 1) from interacting with its ligands PD-L1 and PD-L2. PD-1 is a negative regulator of immune activation that is considered a foundational target within the immuno-oncology market. Balstilimab is currently in clinical trials as monotherapy and in combination with Agenus’ anti-CTLA-4, zalifrelimab, in an ongoing Phase 2 study for recurrent/metastatic cervical cancer.

On April 19, 2021 Checkmate Pharmaceuticals Inc. (NASDAQ: CMPI) ("Checkmate"), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, reported the initiation of dosing in a Phase 2 proof-of-concept trial in patients with head and neck squamous cell carcinoma (HNSCC) (Press release, Checkmate Pharmaceuticals, APR 19, 2021, View Source [SID1234578193]). The trial will assess the efficacy and safety of vidutolimod (CMP-001) in combination with pembrolizumab for the treatment of patients with first-line relapsed or metastatic HNSCC. The results of this trial are expected in the 2nd half of 2022, with initial data in a subset of patients anticipated before the end of 2021.

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"The initiation of patient dosing in this head and neck cancer trial is an important milestone for our indication-expansion strategy for vidutolimod," said Barry Labinger, President and Chief Executive Officer of Checkmate. "Fewer than 20% of patients with relapsed or metastatic HNSCC respond to single-agent checkpoint blockade, which highlights the substantial need for a chemotherapy-free regimen with an improved rate and duration of response."

Checkmate is also conducting two clinical trials of vidutolimod in combination with nivolumab in the lead indication of melanoma. The first is a Phase 2 trial assessing the efficacy and safety of vidutolimod in combination with nivolumab for the treatment of patients with anti-PD-1 refractory melanoma. The second is a Phase 2/3 trial comparing the efficacy and safety of vidutolimod in combination with nivolumab to nivolumab monotherapy in patients with first-line metastatic or unresectable melanoma. Checkmate previously announced initiation of patient dosing in the first-line melanoma trial in March 2021. Trial sites have been activated and patient screening is underway in the refractory melanoma study.

Additional information about Checkmate’s HNSCC trial, including participating investigative sites, can be found here: View Source

Information about the Phase 2 anti-PD-1 refractory melanoma trial and the Phase 2/3 melanoma study can be found here: View Source