On January 13, 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 that recent progress across several programs and outlined goals for 2020 (Press release, Magenta Therapeutics, JAN 13, 2020, View Source [SID1234553070]). These updates will be discussed during a webcast presentation at the 38th annual J.P. Morgan Healthcare Conference on Wednesday, January 15th at 11:30 a.m. PT (2:30 p.m. ET).

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"In 2019 we generated landmark data from our ADC-based targeted patient preparation platform, which is delivering a new class of antibody-drug conjugates (ADCs) that have the power to bring one-time treatment to more patients with autoimmune diseases, blood cancers and genetic diseases. We also presented clinical data for our first-line stem cell mobilization program, MGTA-145, which we are developing as the new standard of care for stem cell mobilization with the potential to benefit all of the transplant-eligible patients each year," said Jason Gardner, D. Phil., President and Chief Executive Officer, Magenta. "As we begin 2020, we are particularly excited to unveil our MGTA-117 clinical candidate for targeted patient preparation for stem cell transplant or gene therapy. New results announced today highlight the potency, safety and broad therapeutic index of MGTA-117, well above that of currently approved ADCs. We believe that MGTA-117 is the optimal agent for depleting stem cells to enable safe immune reset. We look forward to moving this program into the clinic with initial clinical data expected in 2021."

Targeted Patient Preparation Programs

Current methods to condition patients before transplant and gene therapy are dependent on toxic, non-specific chemotherapy or radiation. These pre-transplant treatments are associated with significant side effects, including infertility, cancer, organ damage and death. Magenta is developing targeted, disease-modifying ADCs that are designed to precisely and rapidly remove the disease-causing cells in the body and enable immune system reset without the need for chemotherapy or radiation.

CD117-ADC Recent Progress

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 CD117-targeted, single-agent ADC from Magenta, without the use of chemotherapy or radiation. These unprecedented results validate and advance Magenta’s conditioning platform.

Building on this work, Magenta’s new clinical candidate, MGTA-117, is a CD117 antibody conjugated to amanitin. Results published today in an abstract for the Transplant and Cellular Therapy annual meeting show that MGTA-117 potently depleted stem and progenitor cells and demonstrated a wide tolerability: potency ratio of 30 fold (therapeutic index; typical range for approved ADCs at this stage is two to six fold). This program is advancing to the clinic and further validates Magenta’s antibody drug conjugate-based conditioning platform. MGTA-117 was developed under a partnership with Heidelberg Pharma that grants Magenta exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD117.

MGTA-117 in 2020

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.

CD45-ADC Recent Progress

Current standard treatment for patients with multiple sclerosis involves years of chronic dosing of medications that do not halt the progression of the disease. For patients with systemic sclerosis, a potentially fatal autoimmune disease, there are no approved therapies. Immune reset through stem cell transplant has demonstrated durable remissions in thousands of patients with autoimmune diseases such as multiple sclerosis and systemic sclerosis, and it is recommended by the European League Against Rheumatism (EULAR) in treatment guidelines for systemic sclerosis. The immune reset process involves two main steps: removing the disease-causing cells and replacing them with healthy cells to rebuild the immune system to a healthy state.

Magenta is developing targeted ADCs designed to precisely remove the disease-causing cells in the body without the need for chemotherapy or radiation. Magenta’s CD45-ADC program targets CD45, a protein expressed on immune cells and stem cells and is designed to remove the cells that cause autoimmune diseases in order to enable curative immune reset.

Data presented at the American College of Rheumatology (ACR) meeting in November 2019 showed that a single dose of CD45-ADC removed disease-causing reactive T cells, enabled successful immune reset and rebuild of the immune system and was well tolerated in three models of autoimmune disease, including the EAE model, the most reliable murine model of multiple sclerosis. Further, a single dose of CD45-ADC significantly reduced disease incidence and delayed disease onset in this model that has successfully provided preclinical proof of concept for many clinically validated standard-of-care therapies.

CD45-ADC in 2020

Magenta has identified a lead antibody and has progressed this program into IND-enabling studies, which the Company plans to further advance in 2020.

MGTA-145 First-Line Stem Cell Mobilization Therapy

MGTA-145 Recent Progress

Magenta is developing MGTA-145 as the new first-line standard of care for stem cell mobilization in a broad range of diseases, including autoimmune diseases, blood cancers and genetic diseases. MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, to harness the physiological mechanism of stem cell mobilization.

Magenta is currently studying MGTA-145 and plerixafor in a Phase 1 study in healthy volunteers. Data from the Phase 1 study presented at the ASH (Free ASH Whitepaper) annual meeting in December 2019 showed that MGTA-145 in combination with plerixafor successfully enables safe, same-day dosing, mobilization and collection of sufficient high-quality hematopoietic stem cells for transplant. Further, when cells collected from the first two apheresis subjects were transplanted into humanized mice, the cells engrafted more rapidly and at a five-fold higher level than cells from G-CSF-mobilized peripheral blood.

MGTA-145 in 2020

Magenta intends to complete the Phase 1 study and move this program into multiple Phase 2 studies in patients in 2020. The Phase 2 studies will include both allogeneic and autologous transplant settings and will evaluate mobilization and collection of high-quality cells and engraftment of the cells after transplant.

MGTA-456 Cell Therapy

MGTA-456 Recent Progress

MGTA-456 is a cell therapy designed to provide a high dose of stem cells that are well matched to the patient to enable safe immune and blood system rebuild and durable remissions in patients with blood cancers. In September, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for MGTA-456 for the treatment of multiple inherited metabolic disorders.

Magenta is currently studying MGTA-456 in a Phase 2 study in patients with inherited metabolic disorders, including cerebral adrenoleukodystrophy (cALD) and Hurler syndrome. These are rare, rapidly progressive neurologic disorders that are fatal when left untreated. Results in the first two evaluable patients with cALD updated in December 2019 showed early and durable resolution of the disease at 12 months’ follow-up. The Loes score and NFS score, which measure progress of the disease, remained stable, suggesting that progress of the disease has been halted in these patients. The early and durable resolution of disease with MGTA-456 is not consistently seen with other therapies, including standard stem cell transplant, gene therapy or enzyme replacement therapy.

MGTA-456 in 2020

Magenta intends to complete enrollment in the Phase 2 in 2020 and continue dialogue with the FDA under the RMAT designation, and to discuss with the European Medicines Agency (EMA) for development in Europe

On January 13, 2020 Guardant Health presented the corporate presentation (Press release, Guardant Health, JAN 13, 2020, View Source [SID1234553087]).

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On January 13, 2020 Phoenix Biotechnology’s (PBI) (www.phoenixbiotechnology.com) reported that New research findings presented in November at the Italian Cancer Society’s 61st annual meeting in Naples, Italy confirm the ability of drug PBI-05204 to act synergistically with coadministration of temozolamide and radiotherapy to kill Glioblastoma Multiforme (GBM) in mice with orthotopically implanted human GBM cells Italian Cancer Society’s 61st annual meeting in Naples, Italy. These data are currently being prepared for publication.

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The new research results are the latest in a series of evidence in support of PBI-05204, PBI’s lead anticancer drug derived from Nerium oleander, as a potential treatment for GBM, according to Robert A. Newman, Ph.D., Phoenix Biotechnology’s President and Chief Science Officer. The American Brain Tumor Association reports that GBM kills more than 16,000 people each year in this country. Both Senator John McCain and Senator Edward Kennedy died from complications of GBM.

The Italian research, conducted through the University of L’Aquila, shows an ability of PBI-05204 to synergistically interact with standard of care chemotherapy (temozolamide) and radiotherapy in extending the life of mice with human GBM, according to Dr. Claudio Festuccia, the lead investigator of the project.

GBM is the most common glial brain tumor and results in extremely low patient survival. A better understanding of GBM biology has recently shown some of the important reasons why current new drugs fail to produce major tumor responses, Newman said.

These include, for example, inabilities of a drug (1) to gain access to malignant tissue across the blood barrier, (2) to selectively target only malignant cells, (3) to eliminate GBM stem cells, (4) to induce the synthesis of brain derived neurotrophic factor (BDNF) recently shown to be important for a meaningful GBM response and (5) to interact in a synergistic manner with currently used standard of care therapeutic strategies, he said. In addition to these criteria, an ideal drug would also be orally bioavailable.

"Recent data from research with PBI-05204, a modified supercritical extract of Nerium oleander, has shown that it can meet each of these challenges," Newman said. "Data from Phoenix Biotechnology as well as Duke University have shown the facile ability of oleandrin, an important API within PBI-05204, to cross the blood brain barrier following oral administration to mice."1-3

"Once in the brain, PBI-05204 induces the synthesis of brain derived neurotrophic factor (BDNF), something which very few other drugs have been able to do," he said.4 "PBI’s lead drug also represents targeted delivery against malignant cells through their selective expression of a particular subunit of Na, K-ATPase."5-6

"Very recent and exciting research which is also being prepared for publication has shown that PBI-05204 is very potent in elimination of human GBM stem cells, possibly eliminating the recurrence of tumor after therapy," Newman continued, noting that other scientific investigators from Istanbul, Turkey and Pozzilli, Italy have suggested that oleandrin represents a new approach to brain cancer therapy.7-8

PBI-05204 is the only patented drug containing oleandrin that has been through Phase I and Phase II clinical cancer trials in the United States.

On January 13, 2020 Imaging Endpoints reported that the U.S. Patent and Trademark Office has issued Patent No. 10,332,634 – "Systems And Methods For Reliably Diagnosing Breast Cancer Using Quantitative Textural Analysis" to Imaging Endpoints and its inventor, Chief Medical Officer Ron Korn, M.D., PhD (Press release, Imaging Endpoints, JAN 13, 2020, View Source [SID1234553120]).

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This breakthrough radiomic evaluation tool allows for rapid diagnosis of the nature of a patient’s breast abnormalities from standard mammography images. The technology enables a real-time evaluation that is otherwise available only through an invasive biopsy and avoids the time involved in obtaining and processing the biopsied tissue.

Imaging Endpoints is honored and excited to share with the market this patented technology that could provide patients and physicians the advantage of faster, less invasive information that is critical to treatment decisions and patient outcomes.

Imaging Endpoints is a pioneer and global leader in the non-invasive interrogation of tumors and diseased tissues by analyzing diagnostic images to identify imaging patterns linked to tumor biology. In cancer patients, image analysis provides a non-invasive, low-risk approach to assessing tumor biology prior to therapy and an objective pathway for monitoring treatment responses. The method of using signals detected on image data to characterize tumor biology is based on underlying tumor biology, growth kinetics and other drivers of oncologic transformation which may have novel expression patterns on standard of care radiological images. When used alone or in combination with other factors, a unique imaging phenotype may be developed to characterize lesions using both qualitative and quantitative approaches. Thus insights into the biologic processes of tumors may be measured, derived, inferred or predicted with the discovery of imaging phenotypes. This coupled with imaging’s ability to provide a comprehensive and real-time assessment of the entire tumor and its micro-environment make quantitative imaging an attractive tool for rapid assessment and prognosis.

The Breast Imaging Reporting and Data System (BI-RADS) was established by the American College of Radiology to assign mammogram screenings into discrete categories of perceived severity. A follow-up biopsy is typically recommended for BI-RADS category 4 and higher. However, biopsies ultimately confirm that many BI-RADS 4 designations are benign. Consequently, many women undergo painful and expensive biopsies only to find that the tumor is benign due to the rate of false positives.

Ronald Korn, M.D., PhD commented: "A reliable imaging signature for differentiating between malignant and non-malignant BI-RADS 4 mammographic lesions has remained elusive until now. The Imaging Endpoints invention provides a biomarker signature for determining whether a lesion identified in a breast image is malignant. The signature is derived from processing mammography data using a Quantitative Textural Analysis platform (TexRAD); generating respective histograms and related quantitative metrics, and performing logistical regression to yield a model predictive signature. Imaging Endpoints believes that its technology offers a real-time advantage with rapid results, however additional studies are needed to further validate the correlation of the signature with pathologic variables."

Imaging Endpoints currently offers imaging CRO services for the use of its advanced imaging technologies in clinical trials, and is actively seeking partners to commercialize its technologies for routine patient care.

On January 13, 2020 Helix BioPharma Corp. (TSX: "HBP") ("Helix" or the "Company"), an immunooncology company developing innovative drug candidates for the prevention and treatment of cancer, reported it has closed a private placement financing of 2,940,000 units of the Company ("Units") and the disposition of shares of the Company’s Polish subsidiary, Helix Immuno-Oncology S.A. ("HIO"), representing an 8.5% stake in HIO (together with the Units, the "Purchased Securities") for aggregate gross proceeds of approximately CAD $3,000,000, or $1.02 per Purchased Security (Press release, Helix BioPharma, JAN 13, 2020, View Source [SID1234553139]).

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Each Unit is comprised of one common share and one common share purchase warrant. Each common share purchase warrant will entitle the holder to purchase one common share at an exercise price of $1.43 and have an expiry of five years from the date of issuance. The terms of the private placement also include the disposition by the Company of shares of HIO, representing 8.5% of the outstanding shares of HIO. Following the disposition, the Company owns 66.5% of the outstanding shares of HIO.

The Company intends to use the net proceeds of the private placement for working capital and research and development activities.

ACM Alpha Consulting Management AG provided financial advisory services to Helix in connection with the private placement