On March 3, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s lead drug candidate, tipifarnib, for the treatment of adult patients with relapsed or refractory angioimmunoblastic T-cell lymphoma (AITL), follicular T-cell lymphoma (FTCL) and nodal peripheral T-cell lymphoma with T follicular helper (TFH) phenotype (Press release, Kura Oncology, MAR 3, 2020, View Source [SID1234555122]).

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!

"This important designation from the FDA comes just two months after tipifarnib was awarded Fast Track for the treatment of patients with HRAS mutant head and neck squamous cell carcinomas (HNSCC)," said Bridget Martell, M.A., M.D., Acting Chief Medical Officer of Kura Oncology. "We believe that this designation reflects tipifarnib’s significant potential in these devastating disease settings, and we are now actively preparing to initiate a second registration-directed trial of tipifarnib in advanced nodal lymphomas of TFH phenotype, including AITL."

Fast Track designation is granted by the FDA for products that are intended for the treatment of serious or life-threatening disease or conditions, which demonstrate the potential to address an unmet medical need. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application.

In December 2019, Kura reported updated clinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting showing robust and durable activity from tipifarnib as a monotherapy in relapsed or refractory AITL. The data demonstrated an objective response rate (ORR) of approximately 50% in a heavily pre-treated patient population, with a median of three prior regimens. Additionally, enhanced anti-tumor activity was observed in patients who carried mutations in the killer-cell immunoglobulin-like receptor, or KIR, a CXCL pathway-associated biomarker. These patients had an ORR of 70% and a complete response rate of 40%.

About T-Cell Lymphomas

Peripheral T-cell lymphomas compromise up to 20% of all aggressive non-Hodgkin lymphomas and consist of many different subtypes of fast-growing lymphomas, representing approximately 20,000 incident cases per year worldwide. Outcomes for these patients are poor, with 5-year survival of approximately 30%. Although several drugs have been approved in the relapsed and/or refractory setting, none has led to a survival benefit. In addition, the National Comprehensive Cancer Network guidelines currently recommend clinical trials for these patients. Significant advances in the genetic landscape of T-cell lymphomas have led to revisions to the World Health Organization classification and the introduction of new entities. As a result, cases of AITL, an aggressive form of T-cell lymphoma frequently characterized by high levels of CXCL12 expression, are now unified with FTCL under the classification of nodal lymphomas of TFH phenotype.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent, selective farnesyl transferase inhibitor in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients, showing compelling and durable anti-cancer activity in certain patient subsets. However, no molecular mechanism of action was determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next-generation sequencing and emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. The Company has received multiple issued patents for tipifarnib in the U.S. and foreign countries, including one issued by the U.S. Patent and Trademark Office in September 2019 that further extends Kura’s exclusivity to the use of any farnesyl transferase inhibitor for the treatment of CXCL12-expressing peripheral T-cell lymphoma and acute myeloid leukemia.

On March 3, 2020 PDC*line Pharma, a clinical stage biotech company developing a new class of potent and scalable active immunotherapies for cancers, reported that the first patient was dosed with its innovative medicinal product candidate PDC*lung01 in a phase I/II trial in non-small cell lung cancer (NSCLC) (Press release, PDC Line Pharma, MAR 3, 2020, View Source [SID1234555139]). This patient is under the supervision of Dr. Anne Sibille, principal investigator for the Liege University Hospital (Belgium).

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 objectives of the phase I/II trial (PDC-LUNG-101) are to assess the safety, tolerability, immunogenicity and preliminary clinical activity of the drug candidate, PDC*lung01, associated or not with anti-PD-1 treatment in NSCLC. A total of 62 evaluable HLA-A*02:01 positive NSCLC patients are expected in three clinical centers in Belgium and six in France.

PDC*lung01 is a cell suspension of a mix, in the same proportion, of seven active agents made of irradiated human plasmacytoïd dendritic cells (PDC*line) loaded separately with a distinct synthetic human leukocyte antigen serotype-restricted peptide (HLA-A*02:01) encoded by a tumour antigen. PDC*line is a potent professional antigen-presenting cell that is able to prime and boost the antitumor cytotoxic CD8+ T-cells in the patient’s immune system.

"We are delighted to have achieved this first important milestone in the clinical development of PDC*line Pharma with our lead cancer vaccine candidate," said Eric Halioua, president and CEO of PDC*line Pharma. "Based on preclinical studies there is a strong rationale for developing PDC*lung01 in this indication and we are currently exploring a number of additional indications, where our technology may provide potential benefit for patients."

"PDC*lung01 is an innovation in the field of cancer vaccines that appears particularly suitable for the treatment of NSCLC patients, an area where there is still a high unmet medical need," said Dr. Channa Debruyne, medical director of PDC*line Pharma.

About lung cancer
Worldwide, lung cancer is the most common malignancy for male patients and for both sexes combined (2.1 million new cases per year), and the third most common for female patients (after breast and colorectal cancers). Globally, deaths from lung cancer exceed those from any other cancer, with the number of lung-cancer-related deaths for 2018 estimated at 18.1 million; 18.4% of total cancer deaths. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, representing 80% of cases.

About PDC*line Pharma’s technology

PDC*line’s biological features provide unique advantages:

A professional antigen-presenting cell line, much more potent than conventional dendritic cells in priming and expanding antitumor-specific cytotoxic CD8+ T cells (conventional tumor antigens and neoantigens)
While allogeneic, PDC*line is not rejected by the host immune system and can be injected several times to boost the immune response
Easily produced on a large scale, with a fully mastered and simple manufacturing process (use of bioreactors with a synthetic medium without growth, differentiation or activation factors)
Easy to use: after thawing, the same off-the-shelf product is used to treat the whole target population with a cancer type expressing the target antigens
Very versatile: tumor antigens can be provided by peptide loading, mRNA transfection or retrovirus transduction of PDC*line and the target population can be extended beyond HLA-A2 (currently used as it is expressed by 50% of the Caucasian population) by using other HLAs, either already expressed by PDC*line or added by genetic modification. Moreover, new candidates can be validated for new cancer indications in a few weeks, with ex vivo testing using human peripheral blood mononuclear cells (PBMC)
Synergizes with anti-PD-1 to activate antitumor CD8 T cells

On March 3, 2020 Bracco Diagnostics Inc., the U.S. subsidiary of Bracco Imaging S.p.A., a leading global company in the diagnostic imaging business, reported its commitment to achieve a colorectal cancer screening rate for its employees of at least 80% (Press release, Bracco Diagnostics, MAR 3, 2020, View Source [SID1234555156]).

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!

Colorectal cancer is a major public health problem. Colorectal cancer is the second leading cause of cancer death, and a cause of considerable suffering among more than 145,000 adults diagnosed with colorectal cancer each year. Colorectal cancer screening can reduce colorectal cancer death rates by both preventing the disease through the detection and removal of precancerous polyps and through detection at early more treatable stages.1

The National Colorectal Cancer Roundtable (NCCRT), established by the American Cancer Society (ACS) and the Centers for Disease Control and Prevention (CDC), has evolved their awareness campaign to increase Colorectal Cancer screening rates implementing the 80% in every community initiative in 2019.2

"Bracco Diagnostics Inc. is committed to the health and wellbeing of our employees, not only by taking the 80% screening rate pledge but also providing market leading products that are used in CT Colonography procedures. CT Colonography, also known as CTC or virtual colonoscopy, is a visual, patient-friendly colorectal screening test that can detect both precancerous and cancerous colorectal abnormalities," said Vittorio Puppo, President and CEO of Bracco Diagnostics Inc. "We have proudly supported for many years the great work of the NCCRT, other patient advocacy organizations and the American College of Radiology Colorectal Cancer Committee. At Bracco Diagnostics we are committed to the effort of eradicating colorectal cancer. Consequently, it is a logical step for Bracco Diagnostics to join the growing number of organizations in New Jersey and across the U.S. that make the 80% In Every Community pledge. I encourage other business leaders to join us in making this pledge for their organizations."

To find a location that performs CT Colonography procedures visit the American College of Radiology CT Colonography center locator at View Source

On March 3, 2020 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of agents designed to activate immune response to cancers, reported it will release its fourth quarter and full year 2019 financial results before the market opens on Thursday, March 12, 2020 (Press release, Agenus, MAR 3, 2020, View Source [SID1234555107]). Agenus executives will host a conference call and webcast at 8:30 a.m. ET the same day.

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!

Conference Call and Webcast Information:
Date: Thursday, March 12, 2020
Time: 8:30 a.m. ET
Domestic Dial-in Number: 1-844-492-3727 (U.S.)
International Dial-in Number: 1-412-317-5118 (International)
Conference ID: Agenus

The call will also be webcast and will be accessible from the Company’s website at View Source or via the link: View Source

A replay will be available on the Company’s website approximately two hours after the call.

On March 3, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported financial results for the fourth quarter and full year ended December 31, 2019 and recent business highlights (Press release, Magenta Therapeutics, MAR 3, 2020, View Source [SID1234555123]).

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!

"2019 was a year marked by crucial progress towards our vision of immune reset, including the advancement of our two lead conditioning programs and our two clinical programs. We generated unprecedented data from our ADC-based targeted conditioning platform, and we are particularly pleased with our new MGTA-117 clinical candidate for targeted conditioning for stem cell transplant or gene therapy. Results presented last month at the TCT conference highlighted the potency, safety and broad therapeutic index of MGTA-117, well above that of currently approved ADCs at this stage of development. We look forward to moving this program into the clinic with initial clinical data expected in 2021," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta. "We also presented updated clinical data for our first-line stem cell mobilization program, MGTA-145. We have completed dosing in the Phase 1 trial and are moving forward with multiple Phase 2 studies this year. We are developing MGTA-145 as the new standard of care for first line stem cell mobilization and immune system rebuild with the potential to benefit all of the patients eligible for transplant each year."

Recent Business Highlights:

New MGTA-117 ADC clinical candidate for conditioning demonstrates broad therapeutic index; advancing MGTA-117 to generate patient clinical data in 2021: Magenta presented new data at the TCT conference in February 2020 demonstrating that MGTA-117’s chemically modified linker-toxin between antibody and payload resulted in potent depletion of stem and progenitor cells with an improved therapeutic index over prior molecules: potency ratio of 30 fold (therapeutic index; typical range for approved ADCs at this stage of development is two to six fold). MGTA-117 was developed under a partnership with Heidelberg Pharma that grants Magenta exclusive worldwide development and commercialization rights for ADCs using an amanitin payload and targeting CD117. The antibody and payload are advancing in GMP manufacture. Magenta is scaling up manufacturing of MGTA-117 and completing IND-enabling studies in 2020. The Company intends to move this new product candidate into the clinic with initial clinical data expected in 2021.

Reported first-ever successful gene therapy transplant of non-human primates with targeted single-agent CD117-ADC with no chemotherapy: Data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2019, showed the first-ever successful transplant of gene-modified cells in non-human primates using a tool molecule CD117-targeted, single-agent ADC, without the use of chemotherapy or radiation. These landmark results validate and advance Magenta’s conditioning platform.

Completed dosing in Phase 1 MGTA-145 trial, demonstrating rapid, single-day first line stem cell mobilization and collection; met all primary and secondary endpoints: At TCT, Magenta presented data from the Phase 1 trial of MGTA-145 in healthy volunteers. Data showed that MGTA-145 was safe and well tolerated as a single agent and in combination with plerixafor and demonstrated rapid, single-day mobilization and collection of sufficient numbers of stem cells. The Company has completed dosing in the Phase 1 trial and intends to move this program into multiple Phase 2 trials in patients in 2020. The Phase 2 trials will include both allogeneic and autologous transplant settings and will evaluate mobilization and collection of functional cells and engraftment of the cells after transplant to rebuild the immune system.

Presented first preclinical immune reset data with CD45-ADC at ACR: In November 2019, Magenta presented the first data on the use of targeted ADCs to reset the immune system and halt progression of autoimmune disease. Results showed that a single dose of CD45-ADC removed disease-causing cells, enabled successful reset and rebuild of the immune system and was well tolerated in models of multiple sclerosis, systemic sclerosis and inflammatory arthritis. Further, a single dose of CD45-ADC significantly delayed disease onset in a model of multiple sclerosis that has successfully provided preclinical proof of concept for clinically validated standard of care therapies. Magenta has identified a lead antibody and has progressed this program into IND-enabling studies, which the Company plans to further advance in 2020. On November 11, 2019, Magenta announced that it had exercised its option with Heidelberg Pharma for exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD45.

Presented additional data from Phase 2 study of MGTA-456 showing clinically meaningful durable benefits for patients with inherited metabolic disorders: In updated results presented at TCT, two patients with cerebral adrenoleukodystrophy treated with MGTA-456 in the Phase 2 study in inherited metabolic disorders showed early and durable resolution of disease at one year of follow-up, as measured by resolution of brain inflammation on MRI. The two patients also had stable Loes and neurological function scores, consistent with a halt in disease progression. Patients with Hurler syndrome showed normalized levels of blood a-L-iduronidase and had decreased levels of Hurler-specific urine glycosaminoglycans, the toxic metabolites implicated in disease. Magenta intends to complete enrollment in the Phase 2 trial in 2020 and continue dialogue with the FDA under the RMAT designation on design of a registration-enabling study, and to have discussions with the European Medicines Agency for development in Europe.

Appointed Chief People Officer and SVP of Manufacturing: In February, Magenta announced that it had expanded its senior leadership with two new strategic hires, Kristen Stants as Chief People Officer and Li Malmberg, Ph.D., as Senior Vice President, Head of Manufacturing. Ms. Stants is a seasoned human resources professional who joined Magenta from Alexion Pharmaceuticals, where she served as Head of Talent Strategy, responsible for organizational development and talent acquisition to expand the company’s therapeutic pipeline. Dr. Malmberg is an accomplished technical leader with more than 25 years of manufacturing experience, coming to Magenta from Celgene Corporation, where she served as Vice President, Head of Biologics Development and Manufacturing, responsible for the company’s manufacturing development and biologics manufacturing organization and advanced more than 20 biologics molecule and launched one commercial product.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019, were $145.7 million, compared to $142.6 million on December 31, 2018. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the fourth quarter of 2021.

Research and Development Expenses: Research and development expenses were $18.7 million in the fourth quarter of 2019, compared to $12.4 million in the fourth quarter of 2018. The increase was driven primarily by investments in manufacturing related to our conditioning programs and MGTA-456, increases in personnel to support a clinical-stage company, as well as clinical activities for MGTA-145.

General and Administrative Expenses: General and administrative expenses were $5.9 million for the fourth quarter of 2019, compared to $5.5 million for the fourth quarter of 2018. The increase was primarily due to an increase in personnel and facilities associated with the growth of the Company.

Net Loss: Net loss was $23.2 million for the fourth quarter of 2019, compared to net loss of $16.7 million for the fourth quarter of 2018.