Denali Therapeutics Unveils Portfolio Goals for 2026, Stock Up

Denali Therapeutics Inc. DNLI recently outlined its roadmap for 2026 across its portfolio of investigational therapies for neurodegenerative diseases, lysosomal storage disorders and other serious diseases.Denali Therapeutics is preparing for the anticipated FDA approval and commercial launch of tividenofusp alfa, its investigational therapy for Hunter syndrome. The company expects 2026 to mark the first commercial validation of its platform.Shares of Denali Therapeutics gained 7.43% yesterday and were up 2.5% in after-market trading.More on DNLI’s Hunter Syndrome DrugDenali Therapeutics is developing DNL310 or tividenofuspalfa, an Enzyme Transport Vehicle (ETV)-enabled iduronate-2-sulfatase (IDS) replacement therapy, for the treatment of MPS II (Hunter syndrome). It is designed to cross the blood-brain barrier (BBB) and deliver the iduronate-2-sulfatase (IDS) enzyme throughout the body and brain.Denali Therapeutics submitted a biologics license application (BLA) for tividenofuspalfa under the FDA’s accelerated approval pathway based on data from the phase I/II study in participants with Hunter syndrome.However, in October 2025, the FDA extended its review timeline of the BLA seeking accelerated approval of tividenofusp alfa. The target date has been extended to April 5, 2026, from Jan. 5, 2026. The extension follows the company’s submission of updated clinical pharmacology information in response to an information request from the FDA as part of the standard review process and is not related to efficacy, safety or biomarkers.The regulatory body had previously granted Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations to this therapy.A potential approval will be a significant boost to the company’s growth prospects as there are very few effective therapeutic options available for patients with diseases such as Hunter syndrome.A phase II/III COMPASS study is ongoing. Data from this study is expected to generate confirmatory evidence and support global regulatory submissions.DNLI’s Other Key Milestones Targeted for 2026Denali Therapeutics is evaluating DNL126 for the treatment of Sanfilippo syndrome type A (MPS IIIA). In September, the company completed enrollment in the ongoing phase I/II study of DNL126. This study is designed to support an accelerated approval path in Sanfilippo syndrome type A.Initial data from DNL126 for Sanfilippo syndrome Type A are scheduled to be presented at the 2026 WORLDSymposium in February. A global phase III confirmatory study is being planned.Denali Therapeutics is also developing other candidates in partnership with Takeda TAK and Biogen BIIB.Denali Therapeutics and partner Takeda have collaborated to develop DNL593, an investigational therapeutic designed to deliver progranulin across the blood-brain barrier for the treatment of granulin (GRN) mutation-associated frontotemporal dementia (FTD-GRN).A phase I/II study on TAK-594/DNL593 is ongoing and initial FTD-GRN patient data are expected in 2026.DNLI is evaluating DNL628 (OTV:MAPT) for Alzheimer’s disease. Its clinical trial application for DNL628 for the phase Ib study of DNL628 has been approved and study start-up activities are underway. The approval marks a significant milestone in advancing the oligonucleotide Transport Vehicle (OTV) platform.Meanwhile, Denali Therapeutics and Biogen continue co-development of BIIB122. Biogen is leading the global phase IIb LUMA study, evaluating BIIB122's impact on disease progression in early-stage Parkinson’s disease (PD). The LUMA study is fully enrolled and a readout is expected in 2026.Denali Therapeutics is conducting the phase IIa BEACON study, specifically enrolling participants with LRRK2-associated PD to assess how LRRK2 inhibition may impact this disease.Denali Therapeutics recently announced that the FDA has lifted the clinical hold on the investigational new drug application for DNL952 for Pompe disease. The company plans to begin a phase I study. The therapy is engineered to enhance enzyme delivery into muscle tissues and the brain.Denali Therapeutics also stands to receive royalty payments for SAR443122/DNL758, which is licensed to Sanofi SNY and is in development for ulcerative colitis.Results from the Sanofi-partnered RIPK1 inhibitor eclitasertib (SAR443122/DNL758) in ulcerative colitis are expected in the first half of 2026.DNLI’s Strong Cash PositionAs of Sept. 30, 2025, the company held approximately $872.9 million in cash and marketable securities. In December, it raised an additional $200 million through equity financing and secured a royalty funding agreement with Royalty Pharma worth up to $275 million based on future potential sales of tividenofusp alfa.These moves provide the capital required to support regulatory activities, fund new clinical trials and prepare for commercialization activities for tividenofusp alfa.Road Ahead for DNLI2026 positions Denali Therapeutics at an inflection point. A potential FDA approval of tividenofusp alfa would transform the company into a commercial-stage biotech. Positive data readouts from ongoing studies will also provide impetus to the stock.Shares of DNLI have lost 19% in the past year against the industry’s 17.2% growth.Image Source: Zacks Investment ResearchHowever, any additional regulatory setback will be detrimental to DNLI’s growth prospects.DNLI’s Zacks RankDenali Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here. #1 Semiconductor Stock to Buy (Not NVDA)The incredible demand for data is fueling the market's next digital gold rush. As data centers continue to be built and constantly upgraded, the companies that provide the hardware for these behemoths will become the NVIDIAs of tomorrow.One under-the-radar chipmaker is uniquely positioned to take advantage of the next growth stage of this market. It specializes in semiconductor products that titans like NVIDIA don't build. It's just beginning to enter the spotlight, which is exactly where you want to be.See This Stock Now for Free >>Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Sanofi (SNY): Free Stock Analysis Report Biogen Inc. (BIIB): Free Stock Analysis Report Denali Therapeutics Inc. (DNLI): Free Stock Analysis Report Takeda Pharmaceutical Co. (TAK): Free Stock Analysis ReportThis article originally published on Zacks Investment Research (zacks.com).Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks