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Magenta Championing Innovative Approaches to Stem Cell Transplant

09 Dec Magenta Championing Innovative Approaches to Stem Cell Transplant

Magenta Therapeutics Inc (NASDAQ: MGTA), a Company pioneering research in stem cell transplant to reset the immune system in order to combat autoimmune, blood cancers and genetic diseases, provided a development update for MGTA-145, a CXCR2 agonist that is intended as a first-line therapy for mobilization and collection of hemopoietic stem cells (HSCs) on the same day.

MGTA-145, works in conjunction with plerixafor, a CXCR4 antagonist and has the potential to be a first-in-class therapy approved for use of stem cells for treatment of genetic and autoimmune diseases, replacing granulocyte-colony stimulating factor (G-CSF). The Company announced the final results from the phase 1 clinical trial which was completed in the first half of 2020.

Magenta has entered into a clinical collaboration with bluebird bio (NASDAQ: BLUE) for evaluating the efficacy of MGTA-145, in combination with plerixafor, for the treatment of Sickle Cell Disease in adults and kids through use of Stem Cells. The Data from the clinical trial will also serve as proof-of-concept. The company’s intention to use it as a combination therapy aligns well with bluebird bio’s experience in the use of plerixafor.

John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics, commented

“Achieving reliable and rapid stem cell mobilization and a simplified collection process can ensure the entire patient experience is optimal with respect to therapeutic outcome. The incorporation of bluebird bio’s experience in this area of treatment will be immensely valuable in further developing MGTA-145 plus plerixafor to address the remaining unmet needs in gene therapy approaches for diseases like sickle cell disease. We look forward to collaborating with bluebird bio to evaluate MGTA-145 as the preferred mobilization option for people with sickle cell disease.”

Furthermore Magenta has also initiated the enrollment in an ongoing phase 2 trial for autologous stem cell transplantation of multiple myeloma patients. In addition, the company is likely to initiate a phase 2 clinical trial in early 2021 to test the efficacy of MGTA-145 for the treatment of Myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and ALL and also explore the potential for lowering the risk of Graft-versus-Host Disease (GvHD) in the allogeneic transplant setting.

The Company presented final clinical data from the MGTA-145 stem cell mobilization Phase 1 clinical trial at the annual ASH meeting. The results showed that a single dose of MGTA-145, in combination with plerixafor rapidly and reliably mobilized high numbers of stem cells in a single day without the need for G-CSF for potential use in diseases that can benefit from autologous and/or allogeneic stem cell transplantation.

The candidate has been granted an orphan drug designation by the FDA in May 2020, for the mobilization of hemopoietic stem cells (HSCs) to the peripheral blood for collection and transplant.

Magenta Therapeutics Inc (NASDAQ: MGTA)

Market Cap: $369.05M; Current Share Price: 7.64 USDChart
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An inherited autosomal recessive disorder, Sickle Cell Disease (SCD) is characterized by the lack of sufficient red blood cells, to carry oxygen throughout the body. The disease causes red blood cells to lose their flexibility and acquire the shape of a sickle or crescent moon, thereby affecting their ability to navigate through blood vessels and consequently block blood flow and oxygen. Patients suffering from this disorder have chronic pain, severe anemia, painful swelling in the hands and feet, compromised immunity, retarded growth and vision. Though there is no cure for this condition, if left untreated, it can lead to stroke, pulmonary hypertension, organ damage, ulcers and blindness.

According to an estimate by the Centers for Disease Control and Prevention (CDC), SCD affects over 100,000 Americans and is a major public health concern as it resulted in 75,000 hospitalizations between 1989 through 1993, and cost over $475 million. The Medical expenditure of children with SCD averaged $11,702 for those with medical coverage, and $14,772 for children with employer-sponsored insurance, with at least one hospital stay in 2005.

There is no cure for this disorder and the treatment is aimed at alleviating the symptoms and delaying the progression of the disease. New Born Screening and blood tests are used for initial diagnosis and are followed by medication and monitoring to prevent complications. Steam Cell transplant, bone marrow transplantation and blood transfusions are the more advanced treatment options but are limited by the availability of donors, efficacy of treatment in adults and costs.

The field of Sickle Cell Treatment is witnessing the emergence of groundbreaking research in the form of gene therapy, nitric oxide therapy and inhibition of the formation of sickle cells by boosting hemoglobin production. We take a look at few companies that are at the forefront of innovation in this area.

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Magenta is advancing stem cell research to revolutionize the practice of transplant medicine, through its portfolio of highly differentiated potential first-in-class therapeutic candidates in autoimmune, genetic diseases and blood cancers. The Company aims to build a comprehensive portfolio of innovative therapies that can completely transform the treatment landscape in both autologous and allogeneic transplant. The scope of its medicine covers multiple facets of a transplant journey such as patient preparation, stem cell collection, cell dose and safe immune regeneration.

CD45-ADC, CD117-ADC, and C300, the three profiles being developed by the Company can remove disease-causing cells with precision, eliminating the need for toxic treatments such as chemotherapy and radiation. This not only helps prevent the side-effects such as infertility, organ damage and cancer but also facilitates the immune system to rebuild itself.  The CD45-ADC program is exploring the possibility of targeted antibody-drug conjugates (ADCs) being able to reset the immune system, by targeting CD45, a protein expressed on immune cells and blood stem cells. On the other hand, its CD117-ADC program targets CD117, a protein expressed on blood stem cells, to promote blood and immune system rebuild in patients with genetic diseases undergoing gene therapy or stem cell transplant.

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Furthermore, Magenta is also pursuing the development of MGTA-456, a cell therapy aimed at rebuilding immune systems offering a better cell dose and higher probability of well matched cells. G100, its program targeting Graft-versus-host disease (GvHD), is seeking to eliminate cells that cause acute GvHD so that patients can be dosed precisely at the time of transplant and are able to rebuild their immunity.

The Company has a strategic collaboration with AVROBIO (NASDAQ: AVRO) for evaluating MGTA-117, in combination with AVROBIO’s investigational lentiviral gene therapies for the treatment of lysosomal storage disorders. This would allow them to combine Magenta’s expertise in ADC-based conditioning with AVROBIO’s lentiviral gene therapies, while allowing the companies to hold the rights to their respective candidates and pursue additional partnerships.

Furthermore it has also entered into a clinical collaboration with Beam Therapeutics (NASDAQ: BEAM) for developing MGTA-117 as a target conditioning agent for the treatment of Sickle Cell Disease and Beta-Thallasemia.

In September 2020, the Company received a grant from the National Institutes of Health (NIH) as part of a multi-project, interdisciplinary U19 grant along with the University of Southern California, University of Washington and Fred Hutchinson Cancer Research Center, Harvard University and Massachusetts General Hospital and the Ragon Institute, for research in conditioning agents with gene editing approaches for the treatment of HIV.

As per its Q3, 2020 financial results, the Company had Cash, cash equivalents and marketable securities worth $161.7 million as of September 30, 2020.

Key Takeaways

  • There is a large unmet need for less toxic approach to preparing patients for stem-cell or gene based therapies. The current methods such as granulocyte-colony stimulating factor (G-CSF) not only take 5 to 7 days for administration but also have severe side-effects on the patient. This severely limits the options of elderly patients or those who suffer from Sickle Cell Disease. There is a pressing need for an alternative that can eliminate the limitations of existing conditioning of transplant patients. Magenta estimates that the total addressable market of transplants is nearly 2 billion and it has a great shot at transforming the market with its novel approach.
  • Strategic collaborations and partnerships are key to the success of a clinical-stage company as they bring in much needed product development, regulatory and marketing expertise to the table. Magenta has ongoing clinical collaborations with bluebird bio, AVROBIO and Beam Therapeutics and The National Marrow Donor Program. The Company still retains the rights to its programs with opportunities for further explorations and collaborations.
  • The Company has a diverse pipeline of candidates which not only minimizes its risk but also helps make optimal use of its resources. The Comprehensive approach to the developments of its pipeline that encompasses candidates that address Mobilization, Conditioning, Cell Therapy and Post-Transplant complications have the potential to be a game changer in the transplant industry.
  • Magenta has faced setbacks in terms in enrollments for its clinical trials, owing to the impact of COVID-19 on its clinical sites. This has forced them to shift its Phase 2 trial in inherited metabolic disorders (IMDs) to 2021, while its multiple Phase 2 trials of MGTA-145 may now be staggered through the year on account of the pandemic.
  • Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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