Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter syndrome (MPS II).
Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).
Shares of DNLI have lost 1.9% in the past six months compared with the industry’s 8.3% decline.
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More on DNLI’s Tividenofusp Alfa
The FDA's above-mentioned designation is intended to expedite the development and review of therapeutic drugs being evaluated for the treatment of serious or life-threatening conditions.
Breakthrough Therapy Designation is generally granted to a drug when evidences suggest that the investigational drug may provide substantial improvement over available therapy on at least one clinically significant endpoint.
Data from the open-label phase I/II study have shown promising results, with positive effects on evidence-based surrogate endpoints and early signs of improved clinical outcomes in participants with Hunter syndrome.
This designation will provide Denali with more intensive FDA guidance, including involvement of senior reviewers, and eligibility for rolling and priority review of the marketing application.
In 2021, the FDA granted Fast Track designation to tividenofusp alfa for the treatment of patients with Hunter syndrome. In 2022, the European Medicines Agency granted Priority Medicines designation to tividenofusp alfa the.
In September 2024, Denali announced the outcome of a successful meeting with the FDA, providing a path to filing a biologics license application (BLA) for tividenofusp alfa for accelerated approval and subsequent conversion to full approval for the treatment of Hunter syndrome.
Denali expects to submit a BLA for tividenofusp alfa in early 2025 for regulatory review under the accelerated approval pathway.
DNLI’s Recent Pipeline Updates
Denali recently initiated dosing in a global mid-stage study, BEACON, on BIIB122 (DNL151).
BIIB122, an investigational drug leucine-rich repeat kinase 2 (LRRK2) inhibitor, is being evaluated in participants with LRRK2-associated Parkinson’s disease (LRRK2-PD).
The phase IIa study will evaluate safety and biomarkers associated with oral daily dosing of BIIB122 in approximately 50 participants with Parkinson’s disease and LRRK2 pathogenic mutations confirmed by genetic testing.
BIIB122 is also being evaluated in the ongoing global phase IIb LUMA study in participants with early-stage Parkinson’s disease with or without an LRRK2 mutation in collaboration with Biogen BIIB.
The LUMA study is being conducted by Biogen. This study is expected to enroll approximately 640 participants with early-stage Parkinson’s disease, including eligible ones with LRRK2 mutations.
However, Denali recently announced disappointing top-line results from an analysis of Regimen G of the phase II/III HEALEY ALS Platform Trial on pipeline candidate DNL343.
The HEALEY platform trial is evaluating eIF2B agonist DNL343 in the treatment of amyotrophic lateral sclerosis (ALS).
Results showed that the study did not meet the primary endpoint of efficacy in slowing disease progression as compared with placebo. The primary endpoint was evaluated as a change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival through week 24.
Key secondary endpoints, measuring muscle strength and respiratory function, were also not statistically different between the active and placebo groups at week 24.
Denali Therapeutics Inc. Price, Consensus and EPS Surprise
Denali Therapeutics Inc. price-consensus-eps-surprise-chart | Denali Therapeutics Inc. Quote
Earlier, partner Sanofi SNY discontinued the development of SAR443820/DNL788 for the treatment of ALS, based on the results of the phase II HIMALAYA study, which did not meet the primary endpoint.
Denali and partner SNY were co-developing SAR443820/DNL788 for other indications. However, Sanofi informed Denali that the phase II study evaluating the safety and efficacy of SAR443820/DNL788 on serum neurofilament light chain levels in participants with multiple sclerosis was discontinued. The decision was taken after the study did not meet the primary and key secondary endpoints.
DNLI's Zacks Rank and Stock to Consider
Denali currently carries a Zacks Rank #3 (Hold).
A better-ranked stock in the biotech sector is Halozyme Therapeutics HALO, which currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 90 days, estimates for Halozyme Therapeutics’ 2024 earnings per share have improved from $3.95 to $4.07. Estimates for 2025 earnings per share have increased from $4.73 to $4.81 during the same timeframe.
HALO’s earnings beat estimates in three of the trailing four quarters and met once, delivering an average surprise of 14.86%.
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