NEW YORK - Tiziana Life Sciences Ltd (NASDAQ:TLSA), a biotech firm focused on immunomodulation therapies, has filed for Orphan Drug Designation with the FDA for its intranasal foralumab, intended for non-active secondary progressive Multiple Sclerosis (na-SPMS). The company claims this would be the first therapy for na-SPMS to achieve such designation.
The submission is supported by clinical and non-clinical evidence suggesting the effectiveness of foralumab in na-SPMS, with prevalence estimates partly based on data from the Brigham & Women’s Hospital, Boston. Foralumab is a fully human anti-CD3 monoclonal antibody, which has been observed to stimulate T regulatory cells when administered intranasally.
Currently, 10 na-SPMS patients have been treated under an Expanded Access Program, with 20 more recently granted FDA approval to join. These patients have reportedly either stabilized or improved after treatment, with 70% experiencing reduced fatigue, as measured by the Modified Fatigue Impact Scale.
Furthermore, intranasal foralumab is undergoing a Phase 2a trial, with results expected in 2025. The Orphan Drug Designation, if granted, would provide Tiziana with up to seven years of marketing exclusivity, along with other development incentives.
The FDA’s Orphan Drug Designation program, which reviews applications within 90 days, defines a rare disease or condition as one affecting fewer than 200,000 people in the U.S. The designation is a critical step for drugs targeting such diseases, providing significant value to the developers and potential treatments for patients with limited options.
Tiziana Life Sciences is developing therapies using drug delivery technologies to facilitate alternative routes of immunotherapy, with a focus on nasal delivery to potentially improve efficacy and safety. Intranasal foralumab, their lead candidate, has shown a favorable safety profile in studies so far. The company's approach to immunomodulation represents a novel treatment avenue for neuroinflammatory and neurodegenerative diseases.
This news is based on a press release statement from Tiziana Life Sciences Ltd.
InvestingPro Insights
Tiziana Life Sciences Ltd (NASDAQ:TLSA) has been making significant strides in the biotech industry, particularly with its recent application for Orphan Drug Designation for its promising intranasal foralumab therapy. Investors closely monitoring TLSA may find the following InvestingPro Data particularly intriguing:
- The company's Market Capitalization stands at $62.46 million USD, reflecting its current valuation in the market.
- Despite the potential in their pipeline, the company's Price to Earnings (P/E) Ratio is at -3.6, indicating that it is not currently generating profits relative to its share price.
- With a Price / Book Ratio of 11.29 as of the last twelve months ending Q4 2023, the stock is trading at a premium compared to its book value.
InvestingPro Tips suggest that while Tiziana Life Sciences holds more cash than debt on its balance sheet, which is a positive sign of financial stability, the company has not been profitable over the last twelve months. Management's aggressive share buybacks could be a signal of confidence in the company's future prospects. Additionally, the stock has experienced a strong return over the last month, with a 41.52% increase in price total return.
For readers interested in a deeper dive into Tiziana Life Sciences' financial health and stock performance, InvestingPro offers more insights, with a total of 11 additional InvestingPro Tips available at: https://www.investing.com/pro/TLSA. These tips could provide valuable context for investors considering the stock, especially in light of the company's recent developments.
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