Lisata Therapeutics Announces U.S. FDA Rare Pediatric Disease Designation Granted to LSTA1 for the Treatment of Osteosarcoma
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Insights
The FDA's Rare Pediatric Disease Designation (RPDD) for Lisata Therapeutics' LSTA1 is a significant regulatory milestone that could expedite the drug's development and review process. From a biotech industry perspective, the RPDD acts as an incentive for companies to invest in therapies for rare pediatric diseases, which often lack sufficient treatment options due to their lower prevalence and consequent lower commercial incentives. The potential to receive a priority review voucher upon successful approval of the New Drug Application (NDA) is a key aspect of this designation. These vouchers are highly coveted in the pharmaceutical industry as they can significantly reduce the FDA review time from the standard ten months to six months for a subsequent NDA, which can be a critical advantage in a competitive market.
For investors, the RPDD for LSTA1 increases the attractiveness of Lisata Therapeutics, as the designation can potentially lead to a faster time-to-market for its osteosarcoma treatment. However, it is important to note that while the RPDD is a positive development, it does not guarantee that the NDA will be approved. Investors should consider the clinical trial data and the drug's efficacy and safety profile in addition to the regulatory advantages conferred by the RPDD.
The announcement that Lisata Therapeutics' lead product candidate, LSTA1, has received RPDD for the treatment of osteosarcoma, should be carefully assessed by the market for its potential financial implications. The sale price of priority review vouchers has varied significantly over time, reaching as high as $350 million USD and more recently selling for $75 to $100 million USD. This variability in sale price reflects market demand and the strategic value companies place on expedited FDA review times. For Lisata, the prospect of obtaining such a voucher could represent a substantial financial asset.
Given that LSTA1 is still in the clinical development phase, the impact on the company's stock will likely be driven by investor speculation on the future success of the drug and the likelihood of obtaining and monetizing the voucher. The potential revenue from the sale of the voucher could provide Lisata with additional capital to fund ongoing research and development or to expedite the commercialization of LSTA1, should it be approved. Stakeholders should monitor the progress of LSTA1's clinical trials and the company's strategic use of the voucher if obtained, as these will be critical factors influencing the company's valuation and stock performance in the long term.
Osteosarcoma is a rare and aggressive cancer predominantly affecting children and young adults, creating a high unmet medical need due to limited treatment options. The RPDD granted to LSTA1 by the FDA underscores the potential of this therapy to address such a critical gap. In terms of medical research, the designation suggests that LSTA1 has shown promise in early-stage trials, though the detailed efficacy and safety data from these trials are essential for a thorough evaluation.
For the medical community and patients, the expedited development and review process could mean quicker access to a new treatment option, which is particularly important in the context of a life-threatening condition like osteosarcoma. It is important for ongoing and future clinical trials to robustly demonstrate the drug's benefits and manage any potential risks, as the success of LSTA1 will depend on its ability to improve patient outcomes in a safe and effective manner. The implications for stakeholders are significant, as positive trial results could lead to a new standard of care for osteosarcoma patients.
Achieves critical first step toward priority review voucher
BASKING RIDGE, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to LSTA1, the Company’s lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.
“LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumors, and we believe that potential extends to a beneficial impact in osteosarcoma, a rare cancer that usually develops in the osteoblast cells that form bone,” stated Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata. “Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1, and, more importantly, it underscores the high unmet medical need of this patient population.”
The FDA defines rare pediatric diseases as rare diseases (those with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals under 18 years of age. A substantial benefit of a RPDD is receipt of a priority review voucher, often referred to as a “golden ticket,” once the FDA approves the new drug application (“NDA”) for the product and indication having received the RPDD. Vouchers are especially valuable as they can be used to compel a priority review of an additional NDA or biologic license application for another product or indication, reducing the standard review time of approximately ten months to six months. The voucher may be used by the sponsor or sold to another sponsor for their use. Priority review vouchers have sold for as much as
About LSTA1
LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., covalently bound) anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. LSTA1 also has the potential to modify the tumor microenvironment, with the objective of making tumors more susceptible to immunotherapies. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability, and activity in clinical trials to enhance delivery of SoC chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively. LSTA1 has been granted orphan drug designation for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme (“GBM”) in the U.S. The product candidate has also received a Fast Track designation from the FDA for pancreatic cancer.
About Lisata Therapeutics
Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s lead product candidate, LSTA1, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Based on Lisata’s CendR Platform® Technology, Lisata has already established noteworthy commercial and R&D partnerships. The Company expects to announce numerous clinical study and business milestones over the next two years and has projected that its current business and development plan is funded with available capital through these milestones and into early 2026. For more information on the Company, please visit www.lisata.com.
Forward-Looking Statements
This communication contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, future operations, future financial position, future revenue, projected expenses and capital, prospects, plans and objectives of management are forward-looking statements. In addition, when or if used in this communication, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Lisata or its management, may identify forward-looking statements. Examples of forward-looking statements include, but are not limited to, the potential efficacy of LSTA-1 as a treatment for patients with osteosarcoma and other solid tumors, statements relating to Lisata’s continued listing on the Nasdaq Capital Market; expectations regarding the capitalization, resources and ownership structure of Lisata; the approach Lisata is taking to discover and develop novel therapeutics; the adequacy of Lisata’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; and the difficulty in predicting the time and cost of development of Lisata’s product candidates. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: results observed from a single patient case study are not necessarily indicative of final results and one or more of the clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available, including the risk that unconfirmed responses may not ultimately result in confirmed responses to treatment after follow-up evaluations; the risk that product candidates that appeared promising in early research and clinical trials do not demonstrate safety and/or efficacy in larger-scale or later clinical trials; the safety and efficacy of Lisata’s product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in Lisata’s clinical programs, Lisata’s ability to finance its operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of Lisata’s scientific studies, Lisata’s ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in Lisata’s markets, the ability of Lisata to protect its intellectual property rights; and legislative, regulatory, political and economic developments. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Lisata’s Annual Report on Form 10-K filed with the SEC on February 29, 2024, and in other documents filed by Lisata with the Securities and Exchange Commission. Except as required by applicable law, Lisata undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Contact:
Investors and Media:
Lisata Therapeutics, Inc.
John Menditto
Vice President, Investor Relations and Corporate Communications
Phone: 908-842-0084
Email: jmenditto@lisata.com
FAQ
What is the significance of Lisata Therapeutics, Inc. receiving Rare Pediatric Disease Designation (RPDD) for LSTA1?
What is the benefit of a priority review voucher received by Lisata Therapeutics, Inc.?
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