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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that new results from its CD117-ADC patient preparation program were presented at the 61st Annual Meeting of the American Society of Hematology (ASH). These results, which were highlighted in an oral presentation at ASH by John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, showed the first-ever successful transplant of gene-modified cells in non-human primates using a targeted, single-agent antibody-drug conjugate (ADC), without the use of chemotherapy or radiation.

Todays conditioning regimens involve high doses of chemotherapy, often paired with radiation, to remove the disease-causing cells. As a result, patients undergoing gene therapy or stem cell transplant are all faced with a difficult choice: whether to endure severe toxicity and risk infertility and cancer for the chance for a cure. Magentas portfolio of targeted ADCs represents an extremely promising new option to prepare patients for gene therapy or transplant with no need for toxic chemotherapy or radiation, said Dr. Tisdale. The results presented today show that a single dose of single agent CD117-ADC achieves the same level of depletion as four doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the -globin gene, the gene that causes sickle cell disease and -thalassemia when mutated. Importantly, the animals undergoing preparation with CD117-ADC showed none of the damaging toxicities associated with busulfan conditioning.

Magenta is the only company with the people, platforms and a product engine committed to comprehensively transforming immune and blood system reset, which includes revolutionizing the toxic methods that are used to prepare patients for gene therapy and transplant today. said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. The gene therapy field has learned that higher levels of stem cell depletion, which meant higher doses of busulfan, were needed to ensure long-term engraftment of the gene-modified cells and persistence of gene therapy. Across all the modalities we have tested, we have seen that ADCs are most effective at achieving these high levels of stem cell depletion without chemotherapy to enable engraftment and long-term durability of the transplant. Todays impressive results provide important validation of the ADC approach as well as the CD117 target for patient preparation and underscore Magentas leadership in the field of conditioning.

Results from the CD117-ADC Patient Preparation Program

Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.

Magentas most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells in the bone marrow that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.

Results presented by Dr. Tisdale showed:

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug...

Glimpse of Health Care Player CSLs Gene Therapy

Wait, What? $2.1 million for just a drug? Yes, you heard it right!

Few months back in Belgium, a fundraising campaign was held to raise funds to pay for a one-time gene-therapy of a toddler suffering from an extremely rare spinal muscular atrophy (SMA)disorder. What has caught everyones attention, was the astonishing $2.1 million price of the drug. The question here is whether it is worth?

What is Gene Therapy?

First lets understand what gene therapy is. Gene therapy is one of the most cutting-edge medical technologies, possessing an untold potential on the brink of revolutionizing the treatment of most debilitating, rare and genetic ailments.

In gene therapy, the expression of a patients genes is modified either by deactivating/blocking or repairing the defective genes, and resuming their normal function, thereby, offering a novel and unique approach for treating life-long and devastating diseases.

One-off gene therapy is a one-time treatment, or a single dose/injection or infusion treatment given to patients.

Prices and One-off Gene Therapy

Although these novel gene therapies present significant advantages to patients with unmet medical needs and have been gaining a lot of praise for their innovative way of disease treatment, but they come at a skyrocket price. For instance,

Such high prices of drugs have ignited debate amid companies (those developed them), stakeholders, and patients.

Payers are still struggling to find out the long-term repercussions of these novel and expensive treatment options that may possibly succeed in addressing and curing disease in just a single dose.

But the companies who have developed these drugs are defending the record-breaking cost of the medicine, arguing that such treatments are still cheaper than the alternate treatment available in the market.

Life-time advantage squeezed down to one-time treatment -Zolgensma Vs Spinzara

There has been a recent fuss in the market when the leading biotech company Novartis joined the debate and defended Zolgensma high price that cures SMA which is a leading cause of mortality in a newborn, emphasizing on the fact that the one-time treatment is more valuable than the costly long-run treatments.

Novartis stated an example of its rival drug Spinraza (an alternative treatment to spinal muscular atrophy, developed by Biogen), which requires infusion in every 4 years at a price of $750,000 in the first year and a maintenance dose of $375,000 per year thereafter.

Me Lennon further emphasized that considering the alternate therapy costs $4 million over a span of ten years, while the one-time cost of Zolgensma is $2.1 million, designating Zolgensma the worlds most expensive drug is deceptive.

Connecting the dots-Reducing Financial Burden

Nevertheless, these novel and costly therapies have intensified the discussions on placing a value on gene therapy and how the government and the health care systems can aid in upfront payment for these one-off treatments.

Shedding light on this, Mr Lennon informed that the company is undergoing discussions with the government bodies and the health insurers for creating new payment models rendering the substantial cost reasonable for payers. With respect to this, a five-year payment plan has already been proposed by the company.

Stakeholders must come up with innovative approaches to price and reimburse the expensive treatment in order to enhance the patient access to these life-changing, novel gene therapies.

Moreover, to realizing the tremendous potential value gene therapies possesses, a reasonable access for all patients is important and so a flexible thinking about evaluating their value.

Key Players

A range of biotech companies have been leading the market leveraging gene therapy approaches while some drugs have entered the pharmaceutical market. Some are listed below-

Let us now have a glimpse of an Australian biotech player that had expanded its technology platform with gene therapy.

CSL limited (ASX: CSL)

Leading Australian Biotech Giant- CSL limited (ASX: CSL) is focused ondeveloping, manufacturing and commercialising novel protein-based pharmaceuticals, cell-culture media & human plasma fractions, with its two key businessesCSL Behring and Seqirus.

CSL has ventured into gene therapy in the fiscal year 2018 after acquiring Calimmune Inc, in 2017 that provide CSL a new technology platform and manufacturing process.

The Company acquired 100% of the Calimmune Incs equity, by making an upfront fee of $82 million and subsequent contingent payments subject to the achievement of development milestones.

Calimmune is a U.S. biotechnology company that has established a suite of gene therapy technologies with a potential to treat rare diseases.

CSLs in-vivo versus ex-vivo cell and gene therapy (Source: Company Presentation)

CSL gene therapy targets Sickle Cell Disease (CSL200), with high unment need and immune deficiencies such as Wiskott-Aldrich Syndrome (WAS).

Two year post the acquisition of Calimmune, CSL has completed the integration of this new technology into R&D with its first clinical program enrolling patients. CSL also has early stage gene therapy projects under pipeline.

On 13 December 2019, CSLs stock traded at $278.12, down 0.38%. The market cap of the company was noted at $126.72 billion with 453.87 million outstanding shares. The stock has a P/E ratio of 46.22x, with 0.95% of annual dividend yield.

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Would you pay $2.1 million for one-off gene therapy? - Kalkine Media