Denali Therapeutics Announces Data Presentations on Enzyme TransportVehicle™ Programs for Hunter Syndrome, Sanfilippo Syndrome Type A and Pompe Disease at Upcoming 2026 WORLDSymposium™

Rhea-AI Summary

Denali Therapeutics (Nasdaq: DNLI) will present clinical and preclinical data for its Enzyme TransportVehicle (ETV) programs at the 22nd Annual WORLDSymposium, Feb 2-6, 2026. Highlights include Phase 1/2 follow-up for tividenofusp alfa (DNL310), preliminary Phase 1/2 DNL126 data, and a Phase 1 design for DNL952.

The U.S. FDA is conducting Priority Review of the tividenofusp alfa BLA with a decision expected by April 5, 2026. Multiple posters and two oral presentations will cover clinical outcomes, quality-of-life surveys, a sibling case study, and preclinical Pompe data.

Positive

• FDA Priority Review for tividenofusp alfa with decision due April 5, 2026
• Oral presentations of Phase 1/2 follow-up for DNL310 and preliminary DNL126 clinical data
• Phase 1 study design for DNL952 (ETV:GAA) presented with supporting preclinical data

Negative

• BLA decision pending creates near-term regulatory uncertainty
• Key program data remain early-stage (Phase 1/2 preliminary results)

Key Figures

WORLDSymposium dates: February 2–6, 2026 FDA decision date: April 5, 2026 Phase 1/2 tividenofusp: Phase 1/2 +5 more

8 metrics

WORLDSymposium dates February 2–6, 2026 22nd Annual WORLDSymposium™ meeting window

FDA decision date April 5, 2026 Expected FDA Priority Review BLA decision for tividenofusp alfa

Phase 1/2 tividenofusp Phase 1/2 Ongoing study follow-up for Hunter syndrome (MPS II)

Phase 1/2 DNL126 Phase 1/2 First-in-human open-label study in MPS IIIA

Phase 1 DNL952 Phase 1 Study design in adults with late-onset Pompe disease

Oral presentations 2 presentations Oral sessions for tividenofusp and DNL126 data

Poster presentations 5 posters Posters on MPS II, Pompe model, QoL and study design

Satellite symposium time 6:45–7:45 AM PST “Transforming Patient Care in MPS II” event on February 5, 2026

Market Reality Check

Price: $21.59 Vol: Volume 2,617,768 vs 20-da...

high vol

$21.59 Last Close

Volume Volume 2,617,768 vs 20-day average 1,653,526 (relative volume 1.58) ahead of the data presentations. high

Technical Price at $20.84, trading above 200-day MA at $15.53 and 14.4% below the 52-week high.

Peers on Argus

DNLI was at $20.84 (pre-news move -1.93%) while key biotech peers were mixed: AG...

1 Up

DNLI was at $20.84 (pre-news move -1.93%) while key biotech peers were mixed: AGIO -1.67%, BLTE -0.87%, IDYA -2.89%, FOLD 0%, TVTX +1.27%. Momentum scanner only flagged TVTX with a stronger move of +5.65%, suggesting DNLI’s setup was more stock-specific than sector-driven.

Historical Context

5 past events · Latest: Jan 06 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 06	2026 milestones outlook	Positive	+7.4%	Outlined 2026 priorities including planned commercial launch and multiple readouts.
Dec 30	NEJM clinical data	Positive	-1.0%	Published Phase 1/2 tividenofusp results supporting FDA Priority Review.
Dec 10	Equity offering priced	Negative	-10.6%	Priced common stock and pre-funded warrant offering targeting ~$200M proceeds.
Dec 09	Offering announced	Negative	-0.1%	Proposed $200M equity and warrant offering with additional $30M option.
Dec 04	Royalty funding deal	Positive	+6.8%	Announced $275M royalty funding agreement tied to tividenofusp sales.

Pattern Detected

DNLI has generally reacted positively to value-adding partnership and milestone news, while equity offerings have pressured the stock and one major clinical publication saw a mild negative reaction.

Recent Company History

Over the past months, DNLI has centered investor focus on tividenofusp alfa’s path toward potential approval, with a PDUFA date of April 5, 2026. A $275M royalty deal and a ~$200M financing strengthened its balance sheet, adding to cash of ~$872.9M as of Sept 30, 2025. Clinical data in NEJM for tividenofusp supported FDA Priority Review. Today’s WORLDSymposium™ data presentations on tividenofusp, DNL126, and DNL952 build directly on this theme of advancing TransportVehicle™ programs toward commercialization and broader clinical validation.

Market Pulse Summary

This announcement highlights expanding clinical and preclinical visibility for Denali’s Enzyme Trans...

Analysis

This announcement highlights expanding clinical and preclinical visibility for Denali’s Enzyme TransportVehicle™ programs, with multiple Phase 1/2 and Phase 1 updates and an upcoming FDA decision on tividenofusp alfa by April 5, 2026. Recent history includes substantial funding, a royalty agreement, and NEJM-published data, underscoring both scientific and financial momentum. Investors may watch WORLDSymposium™ data details, regulatory milestones, and any follow-up financing or partnership activity as key markers of execution risk and program durability.

Key Terms

phase 1/2, phase 1, biologics license application, priority review, +3 more

7 terms

phase 1/2 medical

"continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310)"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

phase 1 medical

"Phase 1 study design of DNL952 (ETV:GAA) for Pompe disease"

Phase 1 is the first stage of testing a new drug or medical treatment in people, focused primarily on safety, how the body handles the product, and finding a tolerated dose. Think of it as a short, tightly controlled experiment with a small group to check for dangerous side effects before wider testing; for investors it is an early milestone that reduces some uncertainty but still carries high risk and potential for both big value changes and setbacks.

biologics license application regulatory

"Priority Review of the Biologics License Application (BLA) for tividenofusp alfa"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

priority review regulatory

"tividenofusp alfa (DNL310), currently under FDA Priority Review"

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

open-label medical

"Phase I/II, First-in-Human, Open-Label Study of DNL126 in Children"

Open-label describes a situation where everyone involved in a study or process knows the full details, such as who is receiving a treatment or intervention. For investors, understanding whether a project or product is open-label helps gauge the level of transparency and potential biases, influencing trust and decision-making. It’s like knowing whether a test or experiment is conducted openly or behind closed doors.

pharmacokinetics medical

"Study Design to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics"

Pharmacokinetics is the study of how a substance, such as a drug or chemical, moves through and is processed by the body over time. It tracks how it is absorbed, distributed, broken down, and eventually eliminated. For investors, understanding pharmacokinetics helps gauge the effectiveness, safety, and potential risks of new medications or treatments, which can influence a company’s success and valuation in the healthcare industry.

pharmacodynamics medical

"Study Design to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics"

Pharmacodynamics is how a drug actually affects the body — the strength, type and duration of its effects and the relationship between dose and response. Think of it like how turning a thermostat changes room temperature: it shows what the drug does and how much is needed to get the desired effect. Investors care because these properties drive clinical success, dosing convenience, safety profile and competitive advantage, all of which influence commercial potential and regulatory approval.

AI-generated analysis. Not financial advice.

• Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently under FDA Priority Review
• Preliminary data from Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA) to be featured in oral presentation
• Phase 1 study design of DNL952 (ETV:GAA) for Pompe disease and supporting preclinical data will appear in poster session

SOUTH SAN FRANCISCO, Calif., Jan. 29, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced the presentation of clinical and preclinical data from its Enzyme TransportVehicle™ (ETV) programs at the upcoming 22nd Annual WORLDSymposium™ to be held February 2-6, 2026, in San Diego, California. These presentations will demonstrate the broad potential of the ETV to enable the delivery of enzyme replacement therapies to the whole body, including the brain.

Two oral presentations will discuss continued follow-up data from the Phase 1/2 clinical study of tividenofusp alfa (DNL310) for Hunter syndrome (mucopolysaccharidosis type II, or MPS II) and preliminary clinical data from the ongoing Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA). In addition, three posters will highlight a case study with a non-neuronopathic sibling pair from the Phase 1/2 study of tividenofusp alfa; a community survey conducted in partnership with patient advocacy organization Project Alive examining unmet needs among individuals living with MPS II and their caregivers; and a health outcomes analysis evaluating the clinical and economic burden among individuals treated for MPS II. The U.S. Food and Drug Administration (FDA) is conducting a Priority Review of the Biologics License Application (BLA) for tividenofusp alfa, with a decision expected by April 5, 2026. Denali will also detail the design of the Phase 1 study of DNL952 (ETV:GAA) for Pompe disease and supporting preclinical data in two posters.

Details on the WORLDSymposium presentations are below:

Platform Presentations

Title: Phase I/II Study of Intravenous Tividenofusp Alfa for Mucopolysaccharidosis Type II
Presentation #258
Date: Thursday, February 5, 2026
Session Time: 11:00 AM-noon PST

Title: Preliminary Results From Phase I/II, First-in-Human, Open-Label Study of DNL126 in Children With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Presentation #183
Date: Thursday, February 5, 2026
Session Time: 11:00 AM-noon PST

Poster Presentations

Title: Persistent Clinical Burden and Unmet Needs in Hunter Syndrome (MPS II) in the United States: A Retrospective Cohort Study
Poster #052
Date: Tuesday, February 3, 2026
Time: 3:30-5:30 PM PST

Title: Enhanced Correction of Skeletal Muscle and Brain Pathology in a Pompe Mouse Model Using Transferrin Receptor-Mediated Delivery of GAA
Poster #290
Date: Wednesday, February 4, 2026
Time: 3:30-5:30 PM PST

Title: Tividenofusp Alfa Treatment in a Male Sibling Pair with Non-neuronopathic Mucopolysaccharidosis Type II (MPS II)
Poster #065
Date: Thursday, February 5, 2026
Time: 3:30-5:30 PM PST

Title: Quality of Life (QoL), Unmet Needs, and Treatment Experience of People Living with Mucopolysaccharidosis Type II (MPS II) and Their Caregivers: A Community Survey
Poster #248
Date: Thursday, February 5, 2026
Session Time: 3:30-5:30 PM PST

Title: A Phase 1, Multi-center, Open-label Study Design to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease
Poster #034
Date: Thursday, February 5, 2026
Time: 3:30-5:30 PM PST

PDFs of the presentations will be available on the Events page in the Investor section of Denali’s corporate website once the WORLDSymposium embargo lifts.

Denali will sponsor a satellite symposium event titled “Transforming Patient Care in MPS II” on Thursday, February 5, 2026, from 6:45-7:45 AM PST. Featured speakers are Barbara Burton, M.D.; Gwen Gunn, Ph.D., M.S. and Paul Harmatz, M.D.

About the Denali TransportVehicle™ Platform

The blood-brain barrier (BBB) is essential in maintaining the brain’s microenvironment and protecting it from harmful substances and pathogens circulating in the bloodstream. Historically, the BBB has posed significant challenges to drug development for central nervous system diseases by preventing most drugs from reaching the brain in therapeutically relevant concentrations. Denali’s TransportVehicle™ (TV) platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes and oligonucleotides throughout the whole body, including the brain, by crossing the BBB after intravenous administration. The TV platform is based on engineered Fc domains that bind to specific natural transport receptors, such as transferrin receptor and CD98 heavy chain amino acid transporter, which are expressed at the BBB and deliver the TV and its therapeutic cargo to the brain through receptor-mediated transcytosis. In animal models, antibodies and enzymes engineered with the TV platform demonstrate more than 10- to 30-fold greater brain exposure than similar antibodies and enzymes without this technology. Oligonucleotides engineered with the TV platform demonstrate more than a 1,000-fold greater brain exposure in primates than systemically delivered oligonucleotides without this technology. Improved exposure and broad distribution in the brain may increase therapeutic efficacy by enabling widespread achievement of therapeutically relevant concentrations of product candidates. The TV platform has been clinically validated and five TV-enabled programs are currently in clinical development.

About Denali Therapeutics

Denali Therapeutics Inc. is a biotechnology company pioneering a new class of biotherapeutics designed to cross the blood-brain barrier using its proprietary TransportVehicle™ platform. With a clinically validated delivery platform and a growing portfolio of therapeutic candidates across all stages of development, Denali is advancing toward its goal of delivering effective medicines to transform the lives of people living with neurodegenerative, lysosomal storage and other serious diseases. For more information, please visit www.denalitherapeutics.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements by Denali Therapeutics Inc. (“Denali” or the “Company") regarding Denali’s planned presentations and events at the 2026 WORLDSymposium™; expectations related to Denali's TransportVehicle™ (TV) platform; the timing and availability of data from the ongoing Phase 1/2 study in DNL126 and data analysis from the Phase 1/2 study in DNL310; plans and expectations regarding the ongoing Phase 1 study in DNL952 and the availability of preclinical data for this study; and timelines and expectations related to the potential approval of DNL310. Actual results are subject to risks and uncertainties and may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: uncertainties related to the FDA’s policies and accelerated approval program, including risks that the PDUFA action date may be extended and the FDA may not approve DNL310; the possibility of events or changes that could lead to the termination of Denali’s collaboration agreements; Denali’s dependence on successful development and commercialization of its BBB platform technology and TV-enabled product candidates; Denali’s ability to initiate and enroll patients in its current and future clinical trials; Denali’s ability to conduct or complete clinical trials on expected timelines; Denali’s reliance on third parties for the manufacture and supply of its product candidates for clinical trials and commercial products; the potential for clinical trial results to differ from preclinical, early clinical, preliminary or expected results; the risk of significant adverse events, toxicities or other undesirable side effects; the risk that results from early clinical biomarker studies will not translate to clinical benefit in late clinical studies; the risk that product candidates may not receive regulatory approval necessary to be commercialized; developments relating to Denali’s competitors and its industry, including competing product candidates and therapies; Denali’s ability to obtain, maintain or protect intellectual property rights; and other risks and uncertainties. In light of these risks, uncertainties, and assumptions, the forward-looking statements in this press release are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely upon forward-looking statements as predictions of future events. Denali's product candidates are investigational, and their safety and efficacy profiles have not yet been established. No Denali product candidates have been approved by any health authority for any use. Information regarding additional risks and uncertainties may be found in Denali’s Annual and Quarterly Reports filed on Forms 10-K and 10-Q filed with the Securities and Exchange Commission (SEC) on February 27, 2025, and November 6, 2025, respectively, and Denali’s future reports to be filed with the SEC. Denali does not undertake any obligation to update or revise any forward-looking statements, to conform these statements to actual results or to make changes in Denali’s expectations, except as required by law.

Investor Contact:
Laura Hansen, Ph.D.
hansen@dnli.com

Media Contact:
Erin Patton
epatton@dnli.com

FAQ

What is the timeline for the FDA decision on Denali's tividenofusp alfa (DNLI)?

The FDA Priority Review decision for tividenofusp alfa is expected by April 5, 2026. According to Denali, the Biologics License Application is under Priority Review and a regulatory outcome is anticipated on that date, which could affect commercial timing and access.

What clinical data will Denali present for DNL126 (ETV:SGSH) at WORLDSymposium 2026?

Denali will present preliminary Phase 1/2 clinical results for DNL126 in children with MPS IIIA. According to Denali, an oral presentation on Feb 5, 2026 will report early safety and biomarker observations from the ongoing first-in-human study.

How will Denali report tividenofusp alfa (DNL310) results at WORLDSymposium 2026?

Denali will present continued follow-up Phase 1/2 data for tividenofusp alfa in oral sessions on Feb 5, 2026. According to Denali, presentations include longitudinal clinical outcomes and three related posters on burden, QoL, and a sibling case study.

What will Denali disclose about the DNL952 (ETV:GAA) Pompe program at the meeting?

Denali will detail the Phase 1 study design of DNL952 and show supporting preclinical Pompe data in posters. According to Denali, two posters will cover study design, safety assessments, pharmacokinetics, and transferrin receptor–mediated muscle and brain correction in mice.

Will Denali share patient-reported outcomes or quality-of-life findings at WORLDSymposium 2026?

Yes. Denali will present a community survey and a retrospective cohort study on MPS II burden and QoL. According to Denali, posters describe unmet needs, treatment experience, and health outcomes among people with MPS II and caregivers.