PureTech Receives Orphan Drug Designation for LYT-200 in Acute Myeloid Leukemia
PureTech Health plc receives orphan drug designation from the FDA for LYT-200 in the treatment of acute myeloid leukemia (AML), showcasing potential for a novel and effective therapy. The FDA recognition validates the innovative approach of targeting galectin-9 with LYT-200, offering hope for patients with relapsed or refractory AML.
The orphan drug designation for LYT-200 by the FDA is a significant milestone in the development of new therapies for AML. As an oncologist, it is encouraging to see advancements in treatments that target specific oncogenic drivers like galectin-9. Galectin-9's role in leukemia cells as a potent oncogenic driver and immunosuppressive protein makes it a compelling target for monoclonal antibody therapy. The direct cytotoxic effects of LYT-200, alongside its potential to modulate the immune system, could offer a dual mechanism of action that may enhance treatment efficacy for patients with limited options.
Moreover, the seven-year market exclusivity that comes with the orphan drug status, if approved, could incentivize PureTech to continue investing in the development of LYT-200. This exclusivity period could also potentially lead to higher pricing power for the company, reflecting the specialized nature of the treatment and the smaller patient population it serves. However, it is critical to balance the potential benefits with the need for affordable access to novel therapies for patients.
The ongoing Phase 1b trial of LYT-200 in relapsed/refractory AML and MDS is crucial for assessing the safety and efficacy of this novel monoclonal antibody. From a research perspective, the combination of LYT-200 with standard-of-care treatments like venetoclax and hypomethylating agents is of particular interest. The trial's design reflects a trend towards combination therapies in oncology, aiming to tackle the complexity of cancer through multiple pathways. Initial findings from the trial, expected to be presented in 2024, will provide valuable insights into the drug's clinical potential and could significantly impact PureTech's stock value if the results are positive.
It's important to note that the orphan drug designation, while promising, does not guarantee eventual FDA approval. The designation does, however, provide financial incentives that could reduce the cost of clinical development. For stakeholders, the progress of LYT-200 should be monitored closely as it may influence PureTech's future revenue streams and strategic direction in the oncology market.
The orphan drug designation for LYT-200 in the treatment of AML by the FDA can be seen as a strategic advantage for PureTech in the competitive landscape of oncology drugs. AML and high-risk MDS represent areas with high unmet medical needs and the introduction of a novel therapeutic agent like LYT-200 could disrupt the current market dynamics. From a market perspective, the potential approval of LYT-200 could lead to a significant expansion of PureTech's product portfolio and solidify its position in the oncology segment.
Furthermore, the exclusivity period could provide PureTech with a temporary monopoly in the AML treatment space, assuming LYT-200 demonstrates clinical efficacy and is approved. This could result in a favorable market position and potentially attract partnership opportunities or interest from larger pharmaceutical companies seeking to expand their oncology pipelines. However, the long-term impact will depend on the clinical trial outcomes and the ability of LYT-200 to demonstrate a clear benefit over existing therapies.
03/13/2024 - 07:00 AM
LYT-200 is being advanced in hematological malignancies such as acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), and locally advanced/metastatic solid tumors, including head and neck cancers
Phase 1b clinical trial evaluating LYT-200 in relapsed/refractory AML and MDS patients is ongoing
BOSTON --(BUSINESS WIRE)--
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LYT-200 for the treatment of AML.
“The current long-term survival rates of patients with relapsed or refractory AML are very poor, and there remains a tremendous unmet need for more effective therapies,” said Amir Fathi, MD, Director of the Leukemia Program at Massachusetts General Hospital and lead investigator of the trial.
LYT-200 is a fully human IgG4 monoclonal antibody (mAb) targeting galectin-9, a potent oncogenic driver in leukemia cells and an immunosuppressive protein. LYT-200 has demonstrated direct cytotoxic, anti-leukemic effects through multiple mechanisms and is being developed as a potential novel treatment for hematological malignancies, such as relapsed/refractory AML and high-risk MDS, as well as locally advanced/metastatic solid tumors that have poor survival rates, including head and neck cancers. LYT-200 is currently being evaluated in an ongoing Phase 1b clinical trial in relapsed/refractory AML and MDS, both as a single agent and in combination with standard-of-care venetoclax and hypomethylating agents (HMA). Initial findings from this trial were announced in 2023 , and additional data are expected to be presented in a scientific forum in 2024.
“Orphan drug designation from the FDA validates our belief that targeting galectin-9 with LYT-200 is a novel, promising approach that may offer patients a better tolerated, more effective treatment,” notes Aleksandra Filipovic, MD, PhD, Head of Oncology at PureTech.
The FDA grants orphan drug designation to novel drug and biologic products for the treatment, diagnosis, or prevention of conditions affecting fewer than 200,000 persons in the U.S. Orphan drug designation qualifies PureTech for incentives under the Orphan Drug Act, including tax credits for some clinical trials and eligibility for seven years of market exclusivity in the U.S. , if the drug is approved for AML.
About LYT-200
LYT-200 is a fully human IgG4 monoclonal antibody targeting galectin-9 for the potential treatment of hematological malignancies, such as acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), as well as locally advanced/metastatic solid tumors that have poor survival rates, including head and neck cancers. Galectin-9 is a potent oncogenic driver and immunosuppressor, and in AML it has been described to work via cytotoxic CD8 T cells and natural killer cells. A wide variety of preclinical data underscores the importance of galectin-9 as a target and suggests a potential opportunity for biomarker development. These data demonstrate high expression of galectin-9 across various blood cancers and solid tumor types and show that galectin-9 levels correlate with poor survival in several cancers.
LYT-200 has demonstrated direct cytotoxic, anti-leukemic effects through multiple mechanisms, as well as synergy with standard of care chemotherapy and venetoclax in preclinical models. In the ongoing, Phase 1b clinical trial evaluating LYT-200 as a single agent in AML and MDS, LYT-200 has demonstrated a favorable safety and tolerability profile as well as early signals of potential clinical activity. This trial is ongoing, in addition to a Phase 1/2 adaptive design trial for the potential treatment of advanced/metastatic solid tumors. Consistent with its hub-and-spoke model, PureTech intends to advance LYT-200 via its Founded Entity Gallop Oncology.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company dedicated to giving life to new classes of medicine to change the lives of patients with devastating diseases. The Company has created a broad and deep pipeline through its experienced research and development team and its extensive network of scientists, clinicians and industry leaders that is being advanced both internally and through its Founded Entities. PureTech's R&D engine has resulted in the development of 28 therapeutics and therapeutic candidates, including two (Plenity® and EndeavorRx® ) that have received both US FDA clearance and European marketing authorization and a third (KarXT) that has been filed for FDA approval. A number of these programs are being advanced by PureTech or its Founded Entities in various indications and stages of clinical development, including registration enabling studies. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points.
For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation those related to the LYT-200 development program and the timing for results from ongoing clinical trials of LYT-200, and our future prospects, developments and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption "Risk Factors" in our Annual Report on Form 20-F for the year ended December 31, 2022 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.
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What is the significance of the FDA granting orphan drug designation to LYT-200?
The FDA's orphan drug designation for LYT-200 in AML acknowledges its potential as a novel and effective treatment option for patients with relapsed or refractory AML.
Who is the lead investigator of the clinical trial evaluating LYT-200 in relapsed/refractory AML and MDS patients?
Dr. Amir Fathi, Director of the Leukemia Program at Massachusetts General Hospital, is the lead investigator of the trial.
What is the mechanism of action of LYT-200?
LYT-200 is a fully human IgG4 monoclonal antibody targeting galectin-9, an oncogenic driver in leukemia cells and an immunosuppressive protein, demonstrating direct cytotoxic and anti-leukemic effects.
What are the potential applications of LYT-200 beyond hematological malignancies?
LYT-200 is also being developed for locally advanced/metastatic solid tumors, including head and neck cancers, showing promise in improving survival rates for these challenging conditions.
What are the expected benefits of orphan drug designation for PureTech?
Orphan drug designation provides PureTech with incentives under the Orphan Drug Act, including tax credits for clinical trials and market exclusivity for seven years in the U.S. if LYT-200 is approved for AML.
