BOSTON - In a significant development for patients with acute myeloid leukemia (AML), PureTech Health plc (NASDAQ:PRTC, LSE:PRTC) announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its therapeutic candidate LYT-200. This designation is a critical step in the development of new treatments for diseases affecting fewer than 200,000 people in the U.S., providing incentives such as tax credits and market exclusivity upon approval.
LYT-200, a fully human monoclonal antibody targeting galectin-9, is currently being evaluated in an ongoing Phase 1b clinical trial for patients with relapsed or refractory AML and high-risk myelodysplastic syndrome (MDS). The drug has shown promise in early clinical trials, demonstrating a favorable safety profile and potential clinical activity.
The orphan drug status underscores the FDA's recognition of the need for new AML treatments, as current long-term survival rates for relapsed or refractory AML are notably low. Dr. Amir Fathi, the lead investigator of the trial and Director of the Leukemia Program at Massachusetts General Hospital, highlighted the "tremendous unmet need for more effective therapies" in this patient population.
LYT-200 works by targeting galectin-9, a protein that plays a dual role as an oncogenic driver and an immunosuppressor in leukemia cells. Preclinical data have demonstrated the antibody's direct cytotoxic effects on leukemia cells and its potential for synergy with standard chemotherapy and venetoclax, a treatment already in use for certain types of leukemia.
The ongoing trial is also exploring LYT-200's use in combination with other standard-of-care treatments, such as hypomethylating agents (HMA), with additional data expected to be presented in 2024. Aleksandra Filipovic, MD, PhD, Head of Oncology at PureTech, expressed confidence in the novel approach of targeting galectin-9 and its potential to offer a better-tolerated and more effective treatment for patients.
PureTech's pipeline, which includes LYT-200, reflects the company's commitment to advancing new classes of medicine for devastating diseases through its research and development team and network of scientists and clinicians.
This article is based on a press release statement from PureTech Health plc.
InvestingPro Insights
As PureTech Health plc (NASDAQ:PRTC, LSE:PRTC) makes strides with its therapeutic candidate LYT-200 for AML, the financial landscape of the company presents a mix of challenges and strengths. With a market capitalization of $6.97 billion, PureTech's valuation reflects investor interest in its innovative approach to medical treatments. However, the company's P/E ratio stands at a negative 153.63, indicating that it is currently not generating profits relative to its share price.
InvestingPro Tips suggest that PureTech's management has been actively repurchasing shares, a sign of confidence in the company's future prospects. Additionally, PureTech holds more cash than debt on its balance sheet, providing a cushion for its operations and research endeavors. Yet, analysts caution that the company is quickly burning through its cash reserves and do not expect it to turn a profit this year. This is further underscored by the company's net income, which is projected to decline.
Despite these financial challenges, PureTech's strong performance over the last three months, with a 40.42% return, may signal investor optimism about the company's long-term potential, particularly as it advances its clinical trials and seeks to meet significant unmet medical needs.
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