SOUTH SAN FRANCISCO - Denali Therapeutics Inc. (NASDAQ:DNLI) has announced a pivotal step toward the expedited approval of its investigational treatment for MPS II, known as Hunter syndrome. Following a recent meeting with the U.S. Food and Drug Administration (FDA), the company plans to submit a biologics license application (BLA) for tividenofusp alfa in early 2025 under the accelerated approval pathway.
The FDA has agreed that cerebrospinal fluid heparan sulfate (CSF HS) can serve as a surrogate endpoint to support accelerated approval for the treatment. Denali's Chief Medical Officer, Carole Ho, MD, expressed gratitude for the FDA's guidance and highlighted the significance of this development for individuals and families affected by MPS II.
Denali's announcement coincides with the presentation of new Phase 1/2 study data at the SSIEM 2024 conference, which demonstrates the treatment's robust and durable biomarker responses and positive effects on clinical outcomes. The data includes a 90% mean reduction in CSF HS at Week 24, with sustained effects through Week 104, and improvements in urine glycosaminoglycans (GAGs), serum neurofilament light (NfL), and various clinical outcomes.
The safety profile of tividenofusp alfa has been described as generally well-tolerated, supporting its continued development. MPS II is a rare genetic disorder that leads to severe physical and cognitive impairments, with current treatments only partially addressing the symptoms.
Tividenofusp alfa is a fusion protein designed to deliver the enzyme iduronate-2-sulfatase across the blood-brain barrier to treat both the central nervous system and somatic symptoms of MPS II. The drug has received Fast Track designation from the FDA and Priority Medicines designation from the European Medicines Agency.
Denali is also enrolling participants for the Phase 2/3 COMPASS study across multiple continents, aiming to further evaluate the treatment's efficacy and safety.
This news is based on a press release statement from Denali Therapeutics and reflects the company's current expectations regarding regulatory pathways and clinical study outcomes for tividenofusp alfa. The treatment remains investigational and has yet to receive approval from health authorities.
In other recent news, Denali Therapeutics has made noteworthy strides in its drug development programs. The company's nonclinical data in Science Translational Medicine indicated the potential of its Oligonucleotide Transport Vehicle (OTV) platform to deliver antisense oligonucleotides to the central nervous system and muscle tissues, marking a significant milestone for the company.
Furthermore, Denali's investigational drug, DNL126, was selected for the FDA's Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program, which aims to fast-track the development of treatments for rare diseases. This inclusion is expected to expedite the development path for DNL126.
In terms of analyst notes, H.C. Wainwright revised its price target for Denali to $90 from $95, maintaining a Buy rating. This adjustment followed a review of Denali's development programs, particularly its work on therapies targeting amyloid beta plaques associated with Alzheimer's disease. Simultaneously, Stifel also revised its price target for Denali from $26.00 to $22.00, while maintaining a Hold rating, in light of discussions about an accelerated approval pathway for Denali's DNL310 treatment for Hunter syndrome.
These recent developments reflect Denali's ongoing commitment to addressing the needs of individuals with neurodegenerative diseases.
InvestingPro Insights
As Denali Therapeutics Inc. (NASDAQ:DNLI) makes strides towards the potential accelerated approval of tividenofusp alfa, investors and stakeholders are closely monitoring the company's financial health and market performance. Here are some insights based on real-time data and InvestingPro Tips that may be of interest:
InvestingPro Data shows that Denali has a market capitalization of approximately $3.5 billion, reflecting the market's valuation of the company. Despite an impressive return of 19.92% over the last three months, the company's revenue has experienced a significant decline of 99.63% in the last twelve months as of Q2 2024. This dramatic decrease is also reflected in the company's gross profit margin, which stands at -33,255.09% for the same period.
InvestingPro Tips highlight that Denali holds more cash than debt on its balance sheet, which is a positive sign of financial stability. This is reinforced by the fact that the company's liquid assets exceed its short-term obligations, suggesting a strong liquidity position. However, analysts have revised their earnings upwards for the upcoming period, indicating a potential shift in the company's financial trajectory.
Moreover, analysts do not anticipate Denali to be profitable this year, with net income expected to drop, and the company has not been profitable over the last twelve months. This aligns with the anticipated sales decline in the current year. Despite these challenges, the company is trading near its 52-week high, with the price at 94.36% of this peak.
For those interested in a deeper analysis, there are additional InvestingPro Tips available at https://www.investing.com/pro/DNLI, offering further insights into Denali's financials and market performance. These metrics and tips provide a valuable context for understanding the company's current position and future prospects as it navigates the regulatory pathway for its investigational treatment.
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