On November 30, 2020 Istari Oncology, Inc., a clinical-stage biotechnology company, reported the first patient was dosed in the LUMINOS-101 Phase 2 clinical trial, assessing the safety and efficacy of PVSRIPO in combination with the immune checkpoint inhibitor pembrolizumab (Keytruda) in patients with recurrent glioblastoma multiforme (rGBM) (Press release, Istari Oncology, NOV 30, 2020, View Source [SID1234571967]).

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PVSRIPO is a novel viral immunotherapy that activates a patient’s innate and adaptive immunity to facilitate a targeted anti-tumor immune response. The study seeks to determine whether PVSRIPO and pembrolizumab will be able to generate anti-tumor response in patients with rGBM, given their complimentary mechanisms of action.

"The initiation of this Phase 2 trial represents a significant milestone in the advancement of PVSRIPO and our quest to treat this formidable opponent," said Matt Stober, President and Chief Executive Officer at Istari Oncology. "Currently the treatment options for patients with rGBM are limited and outcomes are grim, so following the encouraging results of our Phase 1 trial, we are eager to see the effectiveness of PVSRIPO in combination with pembrolizumab."

"Combining PVSRIPO’s ability to generate antitumor immune response with a checkpoint inhibitor holds the promise of more effective therapy for this devastating disease," said W. Garrett Nichols, MD, MS, Chief Medical Officer at Istari Oncology. "Achieving rapid disease control is critical in patients with rGBM, one of the most aggressive and treatment-refractory tumors."

LUMINOS-101 is a Phase 2, single arm trial (clinicaltrials.gov NCT04479241) in patients with rGBM that aims to characterize the safety, tolerability and initial efficacy of PVSRIPO intratumoral infusion followed by intravenous pembrolizumab 14 to 28 days later, and every three weeks thereafter.

LUMINOS-101 follows a Phase 1 trial of PVSRIPO in rGBM, conducted at the Preston Robert Tisch Brain Tumor Center at Duke University Medical Center, that found survival rates were significantly higher in rGBM patients who received an intratumoral infusion of PVSRIPO compared to similar patients receiving standard treatment. Overall survival among patients who received PVSRIPO plateaued at 21 percent, 24 to 36 months after injection based on a publication of the interim trial results (Desjardins, et al., 2018 NEJM). The overall survival rate was sustained at 36 months in these patients. A multicenter Phase 2 study of PVSRIPO in patients with rGBM (n=120) completed enrollment in June 2020; follow-up of those patients is ongoing.

The Phase 2 LUMINOS-101 trial will be conducted across multiple research sites, including Ohio State University Comprehensive Cancer Center, University of California San Francisco, Baptist MD Anderson Cancer Center, Duke University Medical Center, Oregon Health and Science University and University Hospitals Seidman Cancer Center (UHSCC) in Cleveland, Ohio. The first patient has been dosed at UHSCC, which treated seven patients with rGBM as part of the previous Phase 2 trial.

"The Phase 1 trial of PVSRIPO yielded survival rates like we’ve never seen before," says Andrew E. Sloan, MD, FACS, Director of the Brain Tumor & Neuro-Oncology Center and the Center of Excellence in Translational Neuro-Oncology at UH Seidman Cancer Center and UH Neurological Institute, and Professor and Vice Chairman, Department of Neurosurgery at Case Western Reserve University School of Medicine. "Glioblastoma is one of the most aggressive tumors known to man. Following the encouraging results of PVSRIPO to date, we are very interested to see if those results improve further in combination with pembrolizumab."

For more information about Istari Oncology and their ongoing clinical trials, visit istarioncology.com.

About PVSRIPO
PVSRIPO is a virus based on the live attenuated Sabin type 1 polio vaccine that has been genetically modified for safety. Unlike other viral immunotherapies, PVSRIPO has a distinct target (the poliovirus receptor CD155), which is widely expressed in neoplastic cells of most solid tumors. Via CD155, PVSRIPO targets tumors with two primary mechanisms: 1) direct damage to and killing of cancerous cells; and 2) engaging innate and adaptive antitumor immune responses via sublethal infection of antigen presenting cells in the tumor, which unleashes an inflammatory cascade resulting in sustained systemic antitumor immunity. PVSRIPO has been granted Breakthrough Therapy Designation and Orphan Status by the FDA in recurrent glioblastoma.

About Glioblastoma
Glioblastoma is the most common and aggressive form of brain cancer, comprising 52% of patients with primary brain tumors. There are approximately 13,000 patients diagnosed with GBM in the United States annually and approximately 18,000 in the European Union. Despite aggressive treatment, survival for newly diagnosed glioblastoma patients is usually less than 20 months, and for patients with recurrence, which occurs in 98% of patients, survival is usually less than 12 months.

On November 30, 2020 FerGene Inc., reported that The Lancet Oncology published the Phase 3 data from the landmark U.S. clinical trial evaluating an investigational gene therapy, nadofaragene firadenovec (rAd-IFN/Syn3), for the treatment of patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) (Press release, FerGene, NOV 30, 2020, View Source [SID1234571990]). In the study, patients received nadofaragene firadenovec, an intravesical therapy given once every three months that is believed to target the patient’s own bladder wall cells to enhance the body’s natural defenses to fight cancer.1

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The Phase 3 study of 157 patients from 33 U.S. sites met its primary endpoint with more than half (53.4%) of CIS ± Ta/T1 patients (carcinoma in situ; with or without concomitant high-grade Ta or T1 disease) achieving a complete response (CR), all by three months. Of the patients who achieved a CR, 45.5% continued to remain free of high-grade recurrence at 12 months. In the study, nadofaragene firadenovec was administered directly into the patient’s bladder once every three months by a healthcare professional. The long-term follow-up phase of the four-year study is ongoing, and patients are continuing to be monitored.1

The most common adverse events (AEs) observed in the study that occurred in patients in order of decreasing frequency were: instillation site discharge, fatigue, bladder spasm, micturition urgency, and hematuria. The discontinuation rate due to AEs was 1.9%.1

"Once patients with high-grade, non-muscle invasive bladder cancer no longer benefit from their initial BCG treatments, patients often make an informed decision to decline cystectomy – a highly complex and life-altering bladder removal surgery – or are often medically ineligible for this complex operation, leaving them with limited options," said Colin P. N. Dinney, MD, Chairman, Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center and senior author of the publication. "These data published in The Lancet Oncology show that nadofaragene firadenovec, a first-of-its-kind therapy, may be an effective treatment option for BCG-unresponsive non-muscle invasive bladder cancer patients."

"As a practicing urologist, I’m encouraged by these efficacy and safety data which demonstrate the potential for a novel treatment option that fits within the urology practice and gives patients the choice of receiving treatment once every three months – which may be a particularly important consideration in this evolving healthcare environment," said Gennady Bratslavsky, MD, President of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). "Our organization is proud to have played a key role in the mid- and late-stage clinical studies for nadofaragene firadenovec."

Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.2,3 BCG remains the first-line standard of care for people living with high-grade NMIBC, however up to 50% of high-grade patients who receive initial treatment with BCG will experience recurrence and disease progression within one year – becoming BCG-unresponsive.4,5

"We believe the important clinical findings highlighted in The Lancet Oncology with this novel gene therapy may fulfill a significant unmet need for patients and have the potential to be practice-changing," said Vijay Kasturi, MD, Vice President of Medical Affairs at FerGene Inc. "We are extremely grateful to the investigators, the patients who participated in the study, FKD Therapies and the SUO-CTC for the important findings highlighted in this publication."

A Biologics License Application (BLA) for nadofaragene firadenovec is currently with the U.S. Food and Drug Administration (FDA).

About Nadofaragene Firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene to do its work. The internal gene/DNA machinery of the cells ‘picks up’ the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer. Nadofaragene firadenovec has been studied in a clinical trial program that includes 221 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.4 It is estimated that there will be approximately 81,000 new cases of bladder cancer in the U.S. in 20206; 75% of these cases present as NMIBC.3 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, up to 50% of high-grade patients will experience disease recurrence within one year.4,5 The outcome for BCG-unresponsive patients is poor, with chemotherapy and radiation or total cystectomy (complete removal of the bladder) often being the recommended next treatment options.7

About the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC)

Created, owned and operated by its members, the SUO-CTC is a clinical research investigator network of over 500 members from more than 200 clinical sites in the U.S. and Canada. This national alliance of leading academic and community based uro-oncologists is committed to furthering urology research. The SUO-CTC is a registered 501c3 not-for-profit corporation and has a cooperative relationship with the Society of Urologic Oncology (SUO). The SUO-CTC pursues clinical trials, in concert with sponsors, to investigate therapeutic interventions which address urological cancers including, but not restricted to: Bladder Cancer, Prostate Cancer and Renal Cancer. Together with industry, the SUO-CTC offers enhanced research options for ultimately delivering better quality of life to our patients.

On November 30, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported that an article highlighting pacritinib data was published in Blood Advances (Press release, CTI BioPharma, NOV 30, 2020, View Source [SID1234572006]). The article, titled "Determining the Recommended Dose of Pacritinib: Results from the PAC203 Phase 2 Dose-Finding Study in Patients with Advanced Myelofibrosis" is available online via this link.

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"Having recently announced the start of a rolling NDA submission for pacritinib as a treatment for myelofibrosis patients with severe thrombocytopenia, a patient population with reduced survival and limited therapeutic options, we continue to be committed to adding to the growing body of evidence underscoring the efficacy and safety profile of pacritinib," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "As our application will consist of a data package from the PERSIST-1, PERSIST-2 and PAC203 Phase 2 trials, with the ongoing Phase 3 PACIFICA trial expected to be completed as a post-marketing commitment, the data published today support our belief that pacritinib has the potential to become an important treatment in this disease setting, pending regulatory approval."

PAC203 Phase 2 was an open-label, randomized, dose-finding trial of pacritinib in patients with myelofibrosis who were previously treated with ruxolitinib. Patients were randomized 1:1:1 to pacritinib 100 mg daily (QD), 100 mg BID, or 200 mg BID. The trial demonstrated that pacritinib 200 mg BID had a favorable benefit risk profile. Spleen volume response (SVR) rates were highest among patients treated with pacritinib 200 mg BID who had a baseline platelet count of less than 50 x 109/L. Overall, the study data supported the selection of the pacritinib 200 mg BID for use in the ongoing Phase 3 PACIFICA study of pacritinib in patients with myelofibrosis with severe thrombocytopenia.

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

On November 30, 2020 Mersana Therapeutics, Inc. (Nasdaq: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported the appointment of Arvin Yang, M.D., Ph.D., as Senior Vice President and Chief Medical Officer effective immediately (Press release, Mersana Therapeutics, NOV 30, 2020, View Source [SID1234571968]). Dr. Yang succeeds Dirk Huebner, M.D., who has stepped down as Chief Medical Officer but will remain with the Company until January 15, 2021, to assist with a smooth transition.

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"We are excited to welcome Arvin to our leadership team as we enter this next crucial and exciting stage for Mersana," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "Arvin’s deep experience in leading late-stage global registration trials combined with his early stage and immuno-oncology experience will be instrumental in guiding the advancement of XMT-1536 into the planned fast-to-market registration-enabling study as well as driving the rest of Mersana’s first-in-class innovative pipeline."

Dr. Yang spent over a decade at Bristol Myers Squibb in various roles with increasing responsibility for the clinical development of oncology and immuno-oncology therapies. Most recently, he was Vice President and Head of Clinical Hematology, where he was responsible for the clinical development of the late stage hematology pipeline. Before that, he was Vice President and Development Lead for Melanoma and GU cancers, and played a critical role in the global approval of nivolumab and nivolumab plus ipilimumab combinations in a number of indications. Earlier, he was responsible for the nivolumab and ipilimumab life-cycle clinical development plans including those in gynecological cancers. Finally, he has held leadership roles overseeing a pipeline of early clinical programs as well as roles in medical affairs. Dr. Yang received his M.D. and Ph.D. from Rutgers Robert Wood Johnson Medical School and completed training in internal medicine at Beth Israel Deaconess, Harvard Medical School and in oncology at Memorial Sloan-Kettering Cancer Center.

"Mersana’s XMT-1536 is poised to make a meaningful difference for people living with ovarian cancer. I am excited about the opportunity to work with the talented Mersana team to bring this important medicine to patients," said Dr. Yang. "I am equally excited about the potential of the NaPi2b target in non-small cell lung cancer adenocarcinoma, Mersana’s robust pipeline of early-stage candidates developed using innovative and differentiated ADC platforms, and the potential of these candidates to make a significant impact in the lives of people living with cancer."

"I would also like to take this opportunity to thank Dirk for his many contributions at Mersana. Over the past two years, Dirk has played a significant role in bringing XMT-1536 to proof of concept in ovarian cancer and in building a strong clinical development team. I wish him all the best in his future endeavors," added Anna Protopapas.

On November 30, 2020 PureTech Health plc (LSE: PRTC, Nasdaq: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, reported that it will host its first R&D Day on Friday, December 11, 2020, beginning at 9:00 a.m., Eastern Time (Press release, PureTech Health, NOV 30, 2020, View Source [SID1234571991]). The virtual program will showcase PureTech’s scientific leadership in lymphatics and related immune pathways and share insights across its Wholly Owned Pipeline, which includes LYT-100, a clinical-stage anti-fibrotic and anti-inflammatory product candidate being advanced for the potential treatment of interstitial lung diseases and lymphedema, and LYT-200, a product candidate targeting foundational immunomodulatory mechanisms for the potential treatment of solid tumors.

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In addition to presentations by PureTech’s senior team, the R&D Day will feature talks from renowned scientists and physicians, including:

Toby Maher, M.D., professor of clinical medicine and director of interstitial lung disease at Keck School of Medicine of the University of Southern California
Siddhartha Mukherjee, M.D., DPhil, clinician and researcher at Columbia University and Pulitzer Prize-winning author of The Emperor of all Maladies and The Gene
Stanley Rockson, M.D., Allan and Tina Neill professor of lymphatic research and medicine at Stanford University; chief of consultative cardiology and director of the Stanford Center for Lymphatic and Venous Disorders
Zev Wainberg, M.D., co-director of the UCLA GI Oncology Program and assistant professor of medicine at UCLA
"PureTech’s inaugural R&D Day is an opportunity to bring together field-leading experts within our expansive global network to discuss the unique therapeutic opportunities within lymphatics and related immunology," said Daphne Zohar, chief executive officer of PureTech Health. "We have made great progress across our Wholly Owned Pipeline, which now includes four product candidates and three discovery platforms, and we look forward to detailing our differentiated approach towards addressing major diseases."

The agenda for PureTech’s R&D Day is:

Opening Remarks

Welcome from Chris Viehbacher, chairman of PureTech Board of Directors, and Bob Horvitz, Ph.D., chairman of PureTech R&D Committee
Perspectives from Daphne Zohar
Lymphatic Science Overview

The science underlying our Wholly Owned Pipeline, including the biology of the BIG Axis, lymphatic system and related immunology: Joe Bolen, Ph.D., chief scientific officer at PureTech
A clinical perspective on lymphatics and the need for innovation in lymphatic disorders and beyond: Stanley Rockson, M.D. (includes Q&A)
Fibrotic and Inflammatory Conditions – Unmet Need and LYT-100

Deep dive into LYT-100 and its potential to treat a range of conditions involving fibrosis, inflammation and impaired lymphatic flow: Michael Chen, Ph.D., head of innovation at PureTech
A pulmonology perspective on interstitial lung diseases, including IPF and Long COVID, and the unmet need: Toby Maher, M.D., Ph.D. (includes Q&A)
Remarks from Eric Elenko, Ph.D., chief innovation officer at PureTech
Immuno-Oncology – Prioritizing Targets and Future Directions

Perspectives on the field of immuno-oncology, where we are today and the clinical landscape: Siddhartha Mukherjee, M.D., DPhil, and Zev Wainberg, M.D.
Deep dive into LYT-200 and targeting galectin-9 for a range of cancer indications: Aleksandra Filipovic, M.D., Ph.D., head of oncology at PureTech
Fireside chat: What’s next? The future of immuno-oncology with Drs. Zev Wainberg and Siddhartha Mukherjee (includes Q&A)
Closing Remarks

PureTech’s vision for future growth and Q&A session with the PureTech senior management team
A webcast of the event will be available on the Investors section of PureTech’s website under Events & Presentations.