Lisata Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to LSTA1 for the Treatment of Osteosarcoma
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The Orphan Drug Designation (ODD) by the FDA for Lisata's LSTA1 is a critical advancement in the field of oncology, particularly for rare cancers like osteosarcoma. From a medical perspective, the designation is a testament to the potential of LSTA1 in addressing the high morbidity and poor prognosis associated with osteosarcoma. The ODD not only highlights the drug's potential efficacy but also its significance in a therapeutic area with limited options. As a physician, the prospect of a new treatment option is promising, especially considering the aggressive nature of osteosarcoma and its tendency for early metastasis.
Furthermore, the ODD could enhance the drug's development process through closer FDA collaboration, which is often a bottleneck in bringing new therapies to market. The incentives provided, such as tax credits and exemption from certain fees, are likely to support the company's financial stability, allowing for more resources to be allocated to robust clinical trials. This could potentially translate to quicker access to LSTA1 for patients in need, pending successful trial outcomes.
From a research and development standpoint, the Orphan Drug Designation for LSTA1 is indicative of the drug's potential to fill an unmet medical need. The designation often precedes a more streamlined clinical trial process, which can lead to faster market entry if the drug proves to be safe and effective. This is of particular interest for investors as it may signal a reduced risk profile for the drug's development pathway and potentially a quicker return on investment.
It's important to note that the ODD also comes with a seven-year period of market exclusivity upon approval. This exclusivity is a significant competitive advantage, as it protects the drug from direct competition, potentially leading to a strong market position and higher profitability for Lisata. However, investors should be aware that the path from ODD to market approval is fraught with challenges and clinical success is not guaranteed.
Looking at the broader market implications, the ODD for LSTA1 could position Lisata Therapeutics as a notable player in the niche market of rare cancer treatments. The rarity of osteosarcoma, coupled with the lack of effective treatments, creates a high-value market opportunity for Lisata. The market exclusivity post-approval could allow the company to set a premium price for LSTA1, reflecting the drug's value in treating such a serious condition.
Investors should consider the potential impact on Lisata's stock, as the ODD might lead to increased investor confidence and a positive market perception of the company's pipeline. However, the long-term success will heavily depend on the outcomes of future clinical trials and the drug's efficacy and safety profile. As the company progresses with clinical development, it will be essential to monitor updates and results to gauge the likelihood of LSTA1 reaching the market and capturing the anticipated value.
BASKING RIDGE, N.J., April 09, 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 Orphan Drug Designation (“ODD”) 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 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.
“We are thrilled to have received another favorable regulatory designation from the FDA. This underscores the significant unmet medical need and demand for better treatments for patients diagnosed with osteosarcoma,” stated Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata. “Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis. Receiving an ODD from the FDA is an important milestone as we plan for future clinical expansion of LSTA1, and we believe it reflects the broad clinical utility of LSTA1 for the treatment of a wide array of solid tumors.”
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. The designation can provide for a seven-year window of exclusive marketing rights post-approval as well as exemption from user fees and eligibility for tax credits for qualified clinical trials. In addition to the financial benefits, it also may potentially shorten clinical development due to closer collaboration with the FDA.
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 resulting in tumors which are 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 ODD for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme and osteosarcoma in the U.S. It also received a Fast Track designation from the FDA for pancreatic cancer and, just recently, a Rare Pediatric Disease designation from the FDA for osteosarcoma.
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
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