On October 2, 2017 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported that during the company’s mid-cycle meeting with the U.S. Food and Drug Administration (FDA) the FDA indicated that, at this point, it is not planning to hold an Oncology Drugs Advisory Committee (ODAC) meeting to discuss the New Drug Application (NDA) for fostamatinib in patients with chronic or persistent immune thrombocytopenia (ITP) (Press release, Rigel, OCT 2, 2017, View Source [SID1234520747]). Additionally, the FDA indicated that it anticipates meeting the Prescription Drug User Fee Act (PDUFA) action date for the application review, which is April 17, 2018. In an earlier communication, the FDA had conditionally approved the proprietary name TavalisseTM.

“Since we submitted our NDA this spring, we have worked collaboratively with the FDA to answer routine questions as they arise,” said Anne-Marie Duliege, MD, executive vice president and chief medical officer of Rigel. “Our positive interactions with the FDA, including their customary biomedical monitoring (BIMO) inspections at our facilities and clinical sites, are in-line with our expectations and have progressed well. We will continue to work closely with the agency and remain committed to bringing fostamatinib to patients with ITP who are in need of new treatment options.”

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with increased risk of severe bleeding events that can result in serious medical complication, or even death. Currently approved therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients derive a benefit from existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

On October 2, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that the first patient has been dosed in the Company’s Phase I clinical trial of PRS-343, its lead proprietary immuno-oncology drug candidate. PRS-343 is a first-in-class bispecific antibody-Anticalin fusion protein and functions as a tumor-targeted immune costimulatory 4-1BB agonist (Press release, Pieris Pharmaceuticals, OCT 2, 2017, View Source [SID1234520746]). The trial, a multicenter, open-label, Phase I dose escalation study that will include expansion cohorts, is designed to determine the safety, tolerability and potential anti-cancer activity of PRS-343 in patients with advanced or metastatic HER2-positive solid tumors for which standard treatment options are not available, are no longer effective, are not tolerated, or the patient has refused standard therapy. Elevated HER2 expression is associated with multiple cancers, including gastroesophageal, bladder, breast and a range of other tumor types.

“We are very pleased to have commenced dosing PRS-343 in this Phase I trial,” said Louis Matis, M.D., Senior Vice President and Chief Development Officer of Pieris. “PRS-343 has been designed to selectively activate 4-1BB-expressing T cells within the tumor microenvironment, thus diminishing the likelihood of toxicity from systemic immune activation. PRS-343 has also exhibited HER2 inhibitory activity in preclinical studies, thereby demonstrating the potential to mediate dual anti-tumor effects.”

About PRS-343:
PRS-343 is a bispecific monoclonal antibody-Anticalin fusion protein comprised of a HER2 tumor-targeting antibody genetically linked to a potent Anticalin specific for the immune costimulatory TNF family receptor 4-1BB (CD137). PRS-343 is being developed as the first 4-1BB based bispecific therapeutic to mediate the activation of tumor-specific T lymphocytes selectively within the tumor microenvironment (TME). 4-1BB is a potent costimulatory immunoreceptor and an established marker for tumor-specific infiltrating T lymphocytes, and is, therefore, an attractive target for cancer immunotherapy. In in vivo preclinical tumor models, PRS-343 has demonstrated potent T lymphocyte activation localized to the TME of established HER2-positive tumors, indicating the potential for both enhanced safety and efficacy.

About HER2-Positive Malignancies:
HER2 is a tyrosine kinase receptor growth-promoting protein found on the surface of some cancer cells and is associated with aggressive disease progression. Multiple tumor types can express HER2 including breast, gastroesophageal, bladder, biliary (cholangiocarcinoma), colorectal, endometrial, ovarian, non-small cell lung, pancreatic, head and neck, and other cancers.

On October 2, 2017 NantKwest Inc. (Nasdaq:NK), a pioneering, next-generation, clinical-stage immunotherapy company focused on harnessing the unique power of the immune system using natural killer (NK) cells to treat cancer reported the clinical implementation of the company’s haNK cell therapy program to human clinical trials with the first participants treated in the first-in-human, Phase I haNK cell therapy clinical trial targeting a wide range of cancer types (Press release, NantKwest, OCT 2, 2017, http://ir.nantkwest.com/phoenix.zhtml?c=254059&p=RssLanding&cat=news&id=2303927 [SID1234520745]).

“Only about 10% of individuals are born with a high affinity Natural Killer cell type needed for the maximum killing effect when combined with monoclonal antibodies widely used in clinical practice today such as Trastuzamab, Rituxan and Avulamab. To maximize tumor cell death by this mechanism known as ADCC we have engineered our off the shelf natural killer cell line with a high affinity CD16 receptor. The potential for this haNK cell therapy is to improve patient outcomes for a significant percentage of the other 90% of individuals being treated with antibody therapy in a broad range of tumor types that can now be explored,” said Patrick Soon-Shiong, MD, Chairman and CEO of NantKwest.

Dr. Soon-Shiong added, “Our haNK cell therapy program was designed to optimize the unique properties of two immunotherapeutics (a cell based platform with a monoclonal antibody) used as a combination therapy. Multiple published preclinical studies have demonstrated the potential for haNK/antibody combinations to synergistically enhance antibody-dependent cell-mediated cytotoxicity (ADCC) activity, providing a sound scientific rationale for the transition of the haNK program into human clinical trials,” said Patrick Soon-Shiong, MD, Chairman and CEO of NantKwest.

haNK Cell Therapy Platform

NantKwest’s haNK cell therapy platform, an allogeneic, off-the-shelf therapy, was developed to optimize the key role of natural killer cells in mediating innate immunity, enhancing adaptive immune responses, and, specifically in the case of haNK, improve anti-tumor responses via antibody-dependent cell-mediated cytotoxicity (ADCC). ADCC is an important part of the human immune system associated with the synergistic interaction of natural killer cells with antibodies to directly kill a target cell that has been identified by an antigen-specific antibody. ADCC represents one of the key mechanisms that antibodies utilize to target and kill cancer cells.

Engineered to express the high-affinity variant of the CD16, high affinity Fc receptor (V158 FcγRIIIa), in multiple published preclinical studies, the combination of haNK cells with a variety of therapeutic antibodies has led to enhanced tumor cell killing when compared to use of the antibody as a single therapeutic agent, providing strong support for this novel combination immunotherapeutic approach.

haNK Phase I Study Background

The primary objective of the Phase I clinical study is to determine the safety of haNK cell therapy administered once per week in up to 16 patients with metastatic or locally advanced solid tumors. Secondary objectives include the determination of objective response rate, progression-free survival, overall survival, and any correlations between tumor molecular profiles (based on genomics, transcriptomics, and quantitative proteomics) and patient outcomes.

On October 02, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, continues to demonstrate an unmatched commitment to hereditary cancer risk assessment and genetic testing during Breast Cancer Awareness Month (Press release, Myriad Genetics, OCT 2, 2017, View Source [SID1234520744]). As the second leading form of cancer in the United States, breast cancer kills one woman every 13 minutes, with more than 250,000 new cases of breast cancer expected to be diagnosed this year alone.

“Myriad has been actively contributing to the fight against breast cancer since 1991. We have been at the forefront of a change in hereditary cancer risk assessment and witnessed remarkable progress in the last 25 years. More than ever before, our hereditary cancer risk assessment and genetic tests deliver critical answers, providing women with more power and control over their future,” said Johnathan Lancaster, M.D., Ph.D., chief medical officer, Myriad Genetics. “Conquering a devastating illness like breast cancer will require even more effort. Myriad will accomplish this through pioneering innovation, research collaborations and investment. We’re optimistic that we will make an even bigger difference for women in the years ahead.”

Our Portfolio of Leading Breast Cancer Tests
Myriad’s portfolio of personalized medicine tools to help doctors prevent and treat breast cancer include: myRisk Hereditary Cancer, riskScore, and EndoPredict. Additionally, the Company is advancing its companion diagnostics such as BRACAnalysis CDx and myChoice HRD for patients with breast cancer through ongoing research collaborations with multiple pharmaceutical companies.

Following is an overview of Myriad’s portfolio of personalized medicine tests for breast cancer:

myRisk Hereditary Cancer is a 28-gene panel and helps doctors understand an unaffected woman’s risk of developing hereditary breast cancer using genetic testing and family history. In contrast, for women already diagnosed with breast cancer, the myRisk test can help identify their risk of secondary cancers, help inform medical management, and may help prevent cancer in unaffected family members who inherited a cancer-causing mutation.

People can find out if they’re candidates for myRisk Hereditary Cancer by going to HereditaryCancerQuiz.com. The quiz is a brief online questionnaire that helps people determine whether they should be further evaluated for hereditary breast cancer and other types of cancer. On average, the quiz takes less than 1 minute to complete.

riskScore is the newest addition to Myriad’s breast cancer portfolio and is available for certain patients who receive a negative myRisk Hereditary Cancer test result. riskScore combines genetic markers throughout the human genome with a woman’s family and clinical history to predict her 5-year and lifetime risk of developing breast cancer.

EndoPredict is a test that combines genetic and clinical data to identify women with low-risk breast cancer who can safely forego chemotherapy after surgery and help maintain their quality of life.
Myriad is known for pioneering scientific breakthroughs and exceptional science. We bring that same spirit to our partnerships with pharmaceutical companies to develop companion diagnostic tests. These personalized medicine tests will help identify patients who are likely to benefit from certain medicines. Our companion diagnostic tests include:

BRACAnalysis CDx is a companion diagnostic test that detects germline BRCA1 and BRCA2 mutations and helps indicate whether or not patients with cancer who may preferentially benefit from the PARP inhibitor class of drugs. Most recently, BRACAnalysis CDx was used to successfully identify patients with metastatic breast cancer that responded to PARP inhibitors and will be submitted to the FDA as a companion diagnostic for use in these patients.

MyChoice HRD is a test that assesses a cancer’s inability to repair DNA damage. The results will help doctors identify more patients who may preferentially benefit from DNA-damaging medicines and PARP inhibitors. myChoice HRD is being studied in ongoing clinical trials for breast cancer.
If you are interested in learning more about Myriad’s commitment to breast cancer, talk to your healthcare professional and visit www.myriad.com to learn more about myRisk Hereditary Cancer, riskScore, EndoPredict, BRACAnalysis CDx and myChoice HRD.

Our Commitment to Patient Advocacy and Access
Myriad is committed to advocating for the discovery and delivery of personalized medicine for patients with breast cancer. The company has a long track record of partnering with advocacy organizations that promote awareness and research on breast cancer and many other diseases. Additionally, we believe access to genetic testing is an essential component of personalized medicine and can help patients live longer, healthier lives, while saving the healthcare system money.

Patients can access our genetic tests a number of ways, including through health insurance coverage from an employer-sponsored health plan, a health insurance exchange plan or a program like Medicare or Medicaid. Myriad also offers financial assistance programs for those who may have trouble accessing tests or lack coverage. The Company has provided more than $76 million in financial assistance to patients over the past five fiscal years (Table 1).

Table 1: Myriad Financial Assistance Contributions

FY2017 FY2016 FY2015 FY2014 FY2013
$ 20,946,808 $ 17,800,000 $ 14,300,000 $ 13,300,000 $ 10,400,000

On October 02, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported new preclinical data for RXDX-106 – a novel immunomodulatory agent with potent anti-tumor activity, alone and in combination with checkpoint inhibitors, that appears to restore and enhance overall immune function by reversing immunosuppression of innate immune cells in the tumor microenvironment (TME) through TYRO3, AXL, and MER (TAM) receptor tyrosine kinase (RTK) inhibition (Press release, Ignyta, OCT 2, 2017, View Source [SID1234520743]). The data were presented in a poster session (abstract number A37) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in Boston, Massachusetts.

“We continue to be excited by the preclinical profile of RXDX-106 and its potential to elicit and potentiate an immune response to cancer, both as a single agent and combined with checkpoint inhibitors,” said Jonathan Lim, M.D., chairman and CEO of Ignyta. “TAM receptors are novel immuno-oncologic RTK targets for precision medicine therapies such as RXDX-106, which we are eager to bring into the clinic as soon as possible to potentially help patients in their fight against cancer.”

In the study, researchers demonstrated immune-mediated, single-agent anti-tumor activity of RXDX-106 in multiple tumor models. The anti-tumor effect was further enhanced by combination therapy with immune checkpoint inhibitors, potentially by reversing immunosuppression of innate immunity in the TME. The data also suggest that RXDX-106 has a novel mechanism of enhancing overall immune function by activating both innate and adaptive immunity, as observed by treatment-mediated changes in relevant cytokine levels and immune cell biomarkers, and regulating cross-talk between immune and cancer cells. These promising early findings support further development of RXDX-106 to potentially treat a wide variety of cancers.