Vertex to Announce First-Quarter 2021 Financial Results on April 29

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).

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 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.

Stand Up to Cancer Announces $4 Million Grant From Mirati Therapeutics to Support Research on KRAS Mutant Cancers

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]).

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 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.

Agenus Submits Balstilimab Biologics License Application to the U.S. FDA for Patients with Recurrent or Metastatic Cervical Cancer

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.

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!

"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.

Checkmate Pharmaceuticals Announces Initiation of Patient Dosing in Phase 2 Head and Neck Cancer Trial

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.

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 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

FDA Gives Go-Ahead for XBiotech’s Candidate Therapy for Phase I/II Double-blind Placebo Controlled Study in Pancreatic Cancer

On April 19, 2021 XBiotech (NASDAQ: XBIT) reported that the FDA has granted permission to commence clinical trials with its novel drug candidate for treating patients with pancreatic cancer (Press release, XBiotech, APR 19, 2021, View Source [SID1234578192]). From 1992 to 2018 the death rate from pancreatic cancer steadily increased in the USA. It is now predicted that pancreatic cancer will claim 48,220 lives and be the 3rd leading cause of cancer death in the USA in 2021 (National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program).

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!

Pancreatic cancer is typically identified at an advanced stage and treatment often includes surgery and aggressive chemotherapy. A current approved treatment involves combination chemotherapy including ONIVYDE and 5-fluorouracil, drugs that have significant toxicities and provide only modest response rates. XBiotech’s new drug candidate (XB2001) specifically targets a process potentially involved in the growth and spread of malignant tumors; and the drug also blocks inflammation associated with tissue injury, which may reduce toxicity associated with the chemotherapy and allow these drugs to be better tolerated and more effective.

The Phase I/II clinical study will evaluate XBiotech’s new drug candidate when added to the ONIVYDE/5-FU combination therapy. The clinical study is chaired by Dr. Shubham Pant, a leading researcher and oncologist at MD Anderson Cancer Center; and will involve at least 15 other top cancer centers around the United States. The Phase 1 portion of the study will examine increasing doses of XBiotech’s new drug and assess tolerability of the combination at escalating doses. Once a safe dose has been determined, the phase 2 portion will begin, enrolling 60 patients, which will be randomized to receive treatment with ONIVYDE/5-FU or ONIVYDE/5-FU combined with XB2001. Clinical endpoints in the study are safety, overall survival, objective response rate, progression free survival, time to treatment failure, clinical benefit response, number of severe adverse advents, as well as biological measures of experimental drug activity.

Dr. Razelle Kurzrock, M.D., Murray Professor of Medicine, Clinical Science Director, Center for Personalized Cancer Therapy, University of California, San Diego commented, "We need more effective treatments for pancreatic cancer and I believe IL-1a, the target of XB2001, represents an important novel target in oncology. This novel drug approach may both antagonize the biology of the tumor and mitigate chemotherapy-related toxicities—offering hope for improved outcomes in cancer, including tumors of the pancreas."

John Simard, Chairman and CEO of XBiotech, stated, "The launch of our new drug into this challenging area of oncology speaks to our strong conviction to the mechanism of this drug and the substantial unmet medical need for patients suffering from pancreatic cancer."

Several classes of therapeutics are undergoing development for pancreatic cancer, such as new cytotoxic chemotherapy, so called PKIs and immunotherapies. However, each of these treatments approaches are expected to provide, at best, modest improvements in survival and are not expected to replace existing current cytotoxic agents. Thus, significant opportunity exists for new drugs that could synergize with existing cytotoxic agents to reduce treatment and disease-related morbidity, and improve treatment outcomes. XBiotech believes its new drug is strongly positioned for this opportunity.

Cytotoxic chemotherapy agents result in systemic toxicity—which is considered a trade-off for potential anti-tumor activity. Toxicity is of acute importance clinically, but consequences of inflammatory responses induced by cytotoxic agents may also have a more profound impact, promoting tumor growth and compromising the efficacy and durability of the therapy itself. Cytotoxic agents upregulate inflammatory pathways, including activation of leukocytes, vascular endothelium and stromal cells of the tumor microenvironment. IL-1a may be a key player in the tumor and treatment related inflammatory pathway.

XBiotech’s new drug XB2001 is a naturally occurring antibody that potently neutralizes IL-1⍺ and is thus a safe and promising approach to block inflammation that occurs with advanced malignancies and chemotherapy. Unchecked, IL-1⍺ can stimulate angiogenesis, enhancing blood and nutrient supply to the tumor; IL-1⍺ may also act to recruit unwanted leukocytes (such as myeloid suppressor cells) into the tumor, that can suppress the ability of the body’s immune system to fight off the tumor; and systemically, IL-1⍺ can mediate metabolic dysfunction, and cause fatigue, anorexia, and anxiety. IL-1⍺ is thus a central player in paraneoplastic inflammation and XBiotech’s new drug therapy holds promise for treating a wide array of cancers.

About True Human Therapeutic Antibodies

XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.