Denali Therapeutics Presents Enzyme TransportVehicle™ Progress Across Three Clinical Programs for Treatment of Lysosomal Storage Disorders at 2026 WORLDSymposium™

Rhea-AI Summary

Denali Therapeutics (NASDAQ: DNLI) presented clinical and preclinical data across three lysosomal storage disorder programs highlighting Enzyme TransportVehicle (ETV) delivery to brain and body. Key points: tividenofusp alfa (DNL310) maintained CSF and urine heparan sulfate reductions through Week 201; DNL126 showed an 80% mean CSF HS reduction at Week 49; DNL952 Phase 1 design and preclinical glycogen reductions in muscle and brain were presented.

Positive

• CSF heparan sulfate reduced and normalized through Week 201
• DNL126 showed mean 80% CSF HS reduction at Week 49
• Urine HS reduced 83% with DNL126 at Week 49
• BLA for tividenofusp alfa is under Priority Review with PDUFA 4/5/2026

Negative

• Most common treatment-related adverse events were infusion-related reactions
• DNL126 safety is preliminary and based on short-duration cohorts (≤49 weeks)

Key Figures

CSF HS reduction: 80% mean reduction CSF GM3 reduction: 61% mean reduction Urine HS reduction: 83% mean reduction +5 more

8 metrics

CSF HS reduction 80% mean reduction DNL126 Phase 1/2, Sanfilippo syndrome type A, at Week 49

CSF GM3 reduction 61% mean reduction DNL126 Phase 1/2, biomarker of lysosomal function, at Week 49

Urine HS reduction 83% mean reduction DNL126 Phase 1/2, Sanfilippo syndrome type A, at Week 49

DNL126 participants 20 participants Ongoing Phase 1/2 Sanfilippo syndrome type A study

Study duration 25-week open-label Initial DNL126 Phase 1/2 period before 193-week extension

Extension period 193 weeks Open-label extension for DNL126 Phase 1/2 study

PDUFA date April 5, 2026 Tividenofusp alfa BLA Priority Review for Hunter syndrome

Week 201 follow-up Week 201 Tividenofusp Phase 1/2 follow-up maintaining biomarker improvements

Market Reality Check

Price: $20.02 Vol: Volume 1,830,915 vs 20-da...

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Technical Shares at $21.28 are trading above the $15.75 200-day moving average, reflecting a pre-existing uptrend.

Peers on Argus

DNLI is down 2.3% while key biotech peers like AGIO, BLTE, IDYA and TVTX also sh...

1 Up

DNLI is down 2.3% while key biotech peers like AGIO, BLTE, IDYA and TVTX also show single-day declines, but momentum scanner flags only 1 peer (APGE) moving up, supporting a stock-specific move rather than a broad sector rotation.

Historical Context

5 past events · Latest: Feb 02 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Feb 02	Webcast announcement	Positive	+4.6%	Announced webcast to review ETV presentations at 2026 WORLDSymposium.
Jan 29	Data presentations	Positive	+0.7%	Flagged upcoming tividenofusp, DNL126, and DNL952 data at WORLDSymposium.
Jan 06	2026 milestones	Positive	+7.4%	Outlined 2026 priorities including expected tividenofusp commercial launch.
Dec 30	NEJM publication	Positive	-1.0%	Published Phase 1/2 tividenofusp data in NEJM backing FDA Priority Review.
Dec 10	Equity offering	Negative	-10.6%	Priced public offering of common stock and prefunded warrants for funding.

Pattern Detected

DNLI has generally reacted positively to ETV platform and milestone updates, with occasional divergence such as a small drop on NEJM clinical data and a larger decline on an equity offering.

Recent Company History

Over the past few months, DNLI has repeatedly highlighted progress for tividenofusp alfa and its broader Enzyme TransportVehicle™ platform. A WORLDSymposium-focused webcast on Feb 2, 2026 and prior data-presentation announcements in late January saw modest to strong gains. A Dec 30, 2025 NEJM publication of Phase 1/2 tividenofusp data coincided with a slight decline, while a Dec 10, 2025 public offering linked to roughly $200 million in proceeds led to a sharper selloff. Today’s detailed clinical updates extend that same program narrative.

Market Pulse Summary

This announcement consolidates Denali’s Enzyme TransportVehicle™ story with extended Phase 1/2 data ...

Analysis

This announcement consolidates Denali’s Enzyme TransportVehicle™ story with extended Phase 1/2 data in Hunter and Sanfilippo syndromes and outlines the Phase 1 design in Pompe disease. It reinforces the April 5, 2026 PDUFA date for tividenofusp alfa and presents substantial biomarker reductions for DNL126, alongside an accelerated approval pathway. Investors may watch for upcoming regulatory decisions, Phase 3 planning, and any further financing or insider activity disclosed in SEC filings.

Key Terms

enzyme replacement therapies, cerebrospinal fluid, biologics license application, priority review, +4 more

8 terms

enzyme replacement therapies medical

"to enable the delivery of enzyme replacement therapies (ERT) to the whole body"

Enzyme replacement therapies are medical treatments that supply patients with missing or malfunctioning enzymes by administering lab-made versions, often through regular infusions. Like replacing a broken part in a machine to restore normal function, these therapies can transform chronic, debilitating conditions into manageable ones; for investors they matter because they often command high prices, face strict regulatory and manufacturing hurdles, and can drive predictable long-term revenue if widely adopted.

cerebrospinal fluid medical

"substantially reduced disease biomarkers in cerebrospinal fluid (CSF) and peripheral tissues"

A clear fluid that surrounds and cushions the brain and spinal cord, acting like a protective bath and cleanup system that removes waste and helps circulate nutrients. For investors, cerebrospinal fluid matters because it is a common source of diagnostic markers and a route for delivering or testing neurological drugs; changes in its composition can signal disease or affect a therapy’s development, approval prospects, and market value.

biologics license application regulatory

"support 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

"currently under Priority Review by the U.S. Food and Drug Administration (FDA)"

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.

prescription drug user fee act regulatory

"anticipation of April 5, 2026, Prescription Drug User Fee Act (PDUFA) date"

A federal program that lets drug makers pay fees to the U.S. regulator to fund and speed up the review of new medicines and label changes. Investors care because it affects how quickly a drug can move from testing to market and how predictable approval timelines and regulatory interactions are — like buying a faster lane at a busy checkpoint that can reduce uncertainty about a product’s commercial timing.

surrogate endpoint medical

"CSF HS may be considered a reasonably likely surrogate endpoint to predict clinical benefit"

A surrogate endpoint is a measurable substitute used in a clinical trial—like a lab test or imaging result—that stands in for a direct patient benefit, such as longer life or improved daily function. Investors care because regulators may accept these quicker, earlier signals to clear or fast-track a treatment, which can shorten development time, reduce costs and change a drug’s market prospects; think of it as using a thermometer to predict recovery instead of waiting for full healing.

accelerated approval regulatory

"may therefore be used to support an accelerated approval path for DNL126"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

pharmacokinetics medical

"intended to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DNL952"

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.

AI-generated analysis. Not financial advice.

• Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa (DNL310) to address full disease spectrum
• Launch readiness established in anticipation of April 5, 2026, Prescription Drug User Fee Act (PDUFA) date for tividenofusp alfa
• Preliminary Phase 1/2 study data show treatment with DNL126 (ETV:SGSH) substantially reduced disease biomarkers in cerebrospinal fluid (CSF) and peripheral tissues, including an 80% mean reduction in CSF heparan sulfate, in Sanfilippo syndrome type A (MPS IIIA); safety profile generally consistent with established enzyme replacement therapies
• Design of ongoing DNL952 (ETV:GAA) Phase 1 clinical study presented in addition to preclinical data that show therapeutic potential to treat both muscle and nervous system manifestations of Pompe disease
  
  

SOUTH SAN FRANCISCO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced data from programs in Hunter syndrome (mucopolysaccharidosis type II, MPS II), Sanfilippo syndrome type A (MPS IIIA) and Pompe disease that highlight the potential of its Enzyme TransportVehicle™ (ETV) to enable the delivery of enzyme replacement therapies (ERT) to the whole body, including the brain. Data were presented this week at the 22nd Annual WORLDSymposium™ in San Diego, California.

“The data presented at this year’s WORLDSymposium reflect the strong momentum of our Enzyme TransportVehicle franchise as we continue to prepare for the potential commercial launch of tividenofusp alfa for Hunter syndrome and make meaningful progress across lysosomal storage disorders,” said Peter Chin, M.D., Acting Chief Medical Officer and Head of Development of Denali Therapeutics. “From advancing our program in Sanfilippo to expanding into muscle disease with Pompe, we are continuing to broaden the reach of our ETV platform with the goal of delivering new medicines in areas of high unmet need. This progress is made possible through constructive collaboration with regulatory authorities and close partnership with the community, listening to and learning from them to better understand treatment journeys of individuals and their families living with these debilitating diseases.”

Key highlights from the presentations are summarized below.

Tividenofusp alfa (DNL310, ETV:IDS) for Hunter syndrome (MPS II)

An analysis from continued follow-up of the Phase 1/2 study of the investigational therapy tividenofusp alfa (DNL310) for MPS II showed that rapid, substantial reduction from baseline and normalization of cerebrospinal fluid heparan sulfate (CFS HS) and urine HS, both key biomarkers of disease, resulting from treatment was maintained through Week 201 (as of the clinical data cut-off of March 28, 2025). Stabilization or improvement in clinical endpoints including adaptive behavior, cognition and hearing, and normalization of liver volume was also observed through Week 201. Safety and tolerability data were consistent with previously reported results from the Phase 1/2 study. Data from this Phase 1/2 study support the Biologics License Application (BLA) for tividenofusp alfa that is currently under Priority Review by the U.S. Food and Drug Administration (FDA) with a target decision date of April 5, 2026.

Denali also highlighted data from a case study of two male siblings with non-neuronopathic MPS II enrolled in the Phase 1/2 trial, which further supports the potential of tividenofusp alfa to address the full disease spectrum.

DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA)

The ongoing Phase 1/2 study of the investigational therapy DNL126 (ETV:SGSH) is fully enrolled with a total of 20 participants and is an open-label, 25-week study followed by an open-label extension period through 193 weeks. Preliminary data as of the clinical data cut-off (June 4, 2025) were presented, including biomarker results from the dose-finding cohorts (n=8) and safety data from the dose-finding and efficacy cohorts (n=14). Preliminary biomarker results from dose-finding cohorts showed treatment with DNL126 resulted in a mean reduction in CSF HS of 80% (95% CI: 43% to 93%) and 61% in CSF GM3 (95% CI: 36% to 76%), a biomarker of lysosomal function, from baseline at Week 49. Normalization of levels of CSF HS and GM3 from baseline were observed in three of seven individuals and six of seven individuals, respectively, with CSF samples available at Week 49. A mean reduction in urine HS of 83% (95% CI: 77% to 87%) from baseline was observed at Week 49, and improvement in liver volume was observed as early as Week 25. Preliminary safety data from participants who had received at least 24 weeks of treatment in the dose-finding and efficacy cohorts demonstrates that the safety profile of DNL126 is generally consistent with established ERTs. The most common treatment-related adverse events in the study were infusion-related reactions.

“These promising data show for the first time that treatment with the brain-penetrant enzyme replacement therapy DNL126 substantially reduced biomarkers of substrate accumulation in cerebrospinal fluid and peripheral tissues,” said Elizabeth Jalazo, M.D., University of North Carolina at Chapel Hill and an investigator in the Phase 1/2 study. “Currently, there are no approved disease-modifying therapies for individuals living with Sanfilippo syndrome type A, which presents with developmental delay and behavior problems that progress to severe cognitive and motor decline. Many affected individuals do not live beyond adolescence.”

In August 2025, Denali announced that it reached alignment with the FDA that CSF HS may be considered a reasonably likely surrogate endpoint to predict clinical benefit and may therefore be used to support an accelerated approval path for DNL126 for MPS IIIA. Denali expects a BLA submission and potential approval for DNL126 for MPS IIIA in 2027. Planning for a global Phase 3 confirmatory study is ongoing.

DNL952 (ETV:GAA) for Pompe disease

Denali presented the Phase 1 clinical study design for the investigational therapy DNL952 (ETV:GAA) in participants with late-onset Pompe disease. The study includes planned cohorts evaluating different dose regimens of DNL952 in patients previously treated with second-generation ERTs, as well as optional additional cohorts including treatment-naïve patients. The study is intended to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DNL952. Preclinical data also presented at WORLDSymposium showed improved glycogen reduction compared to a second-generation ERT in both skeletal muscle and brain in a mouse model of Pompe disease.

About Hunter syndrome (MPS II)

Hunter syndrome, also known as MPS II, is a rare genetic lysosomal storage disease caused by mutations in the iduronate-2-sulfatase (IDS) gene. This results in a deficiency of the IDS enzyme, which is responsible for breaking down glycosaminoglycans (GAGs) such as heparan sulfate and dermatan sulfate. The accumulation of GAGs leads to progressive damage in multiple organs and tissues, including the brain. Symptoms of Hunter syndrome include developmental delays, cognitive decline, behavioral abnormalities and physical complications such as joint stiffness, hearing loss and organ dysfunction. Current standard-of-care enzyme replacement therapies do not cross the blood-brain barrier and therefore do not address the neurological symptoms of the disease. There is a significant unmet need for therapies that address both the central nervous system (CNS) and peripheral manifestations of Hunter syndrome.

About Tividenofusp Alfa

Tividenofusp alfa (DNL310) is composed of the iduronate 2-sulfatase (IDS) enzyme fused to Denali’s proprietary TransportVehicle™ (TV) platform, designed to deliver IDS into the brain and the body, with the goal of addressing behavioral, cognitive and physical symptoms of Hunter syndrome (MPS II). In addition to Rare Pediatric Disease Designation and Breakthrough Therapy Designation, the U.S. Food and Drug Administration has granted Fast Track and Orphan Drug designations to tividenofusp alfa for development in the treatment of MPS II. The European Medicines Agency has granted Priority Medicines designation to tividenofusp alfa.

Denali is conducting the Phase 2/3 COMPASS study in participants with MPS II in North America, South America and Europe to support global approval. Participants are randomized 2:1 to receive either tividenofusp alfa or idursulfase, respectively. More information about the COMPASS study can be found here.

Tividenofusp alfa is an investigational therapeutic and has not been approved for use by any Health Authority.

About Sanfilippo syndrome type A (MPS IIIA)

Sanfilippo syndrome type A, also known as MPS IIIA, is a rare, genetic lysosomal storage disorder characterized by severe neurocognitive deterioration during childhood, with many affected individuals not living past adolescence. MPS IIIA results from a deficiency of the sulfoglucosamine sulfohydrolase (SGSH) enzyme, which is responsible for degrading heparan sulfate in the lysosome. There are no approved disease-modifying therapies for MPS IIIA.

About DNL126 (ETV:SGSH)

DNL126 (ETV:SGSH) is an investigational, intravenously administered, Enzyme TransportVehicle™ (ETV)-enabled N-sulfoglucosamine sulfohydrolase (SGSH) replacement therapy designed to deliver SGSH into the brain and body, with the goal of addressing the behavioral, cognitive and physical manifestations of MPS IIIA. In 2024, the U.S. Food and Drug Administration (FDA) selected DNL126 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program, with the stated purpose to further accelerate the pace of development of novel drug and biological products that are intended to address an unmet medical need as a treatment for a rare disease.

Denali is conducting a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory clinical efficacy of DNL126 in participants with MPS IIIA. The core study period is approximately 6 months and is followed by an open-label extension for approximately 18 months. The study has five cohorts (two dose-finding cohorts and three efficacy cohorts). The primary endpoint of the Phase 1/2 study is percent change in baseline in cerebrospinal fluid heparan sulfate (CSF HS) at Week 49. More information about the study can be found here. Planning for a global Phase 3 confirmatory study is ongoing.

About Pompe disease

Pompe disease is a rare, progressively debilitating genetic disorder in which the body cannot break down glycogen, a complex sugar that is stored in cells for energy. This is caused by a deficiency of the lysosomal enzyme, acid alpha-glucosidase (GAA), which is responsible for the breakdown of glycogen. As a result, glycogen builds up in cells, especially in muscle tissue, including the heart, diaphragm and skeletal muscles, leading to muscle weakness, breathing difficulties and, in some cases, life-threatening complications.

About DNL952 (ETV:GAA)

DNL952 (ETV:GAA) is an investigational therapy being developed by Denali. This treatment is proposed to work by using the Enzyme TransportVehicle™ (ETV) to enhance delivery of the missing enzyme, GAA, into muscle tissues and across the blood-brain barrier into the brain. Denali is conducting a Phase 1 clinical study of DNL952 in participants with late-onset Pompe disease. More information about the study can be found here.

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 regarding Denali Therapeutics Inc.'s ("Denali" or the "Company") business strategy and business plans, including expected key milestones for Denali's therapeutic portfolio in 2026 and beyond and Denali’s ability to execute on its commercial strategies; plans, timelines and expectations related to Denali's ETV franchise and its therapeutic and commercial potential; plans, timelines and expectations relating to tividenofusp alfa (DNL310), including the timing, likelihood and scope of regulatory approvals and commercial launch, the therapeutic potential of tividenofusp alfa, and the likelihood of the Phase 2/3 COMPASS data to support confirmatory evidence for global regulatory submissions and approval; plans, timelines and expectations related to DNL126, including the timing and availability of data from the Phase 1/2 study, the therapeutic potential of DNL126, the likelihood of regulatory approval, and the plans to initiate a Phase 3 study; plans and expectations regarding DNL952, the design of the Phase 1 study and the program's therapeutic potential; expectations regarding Denali's leadership in developing transferrin receptor (TfR)-enabled and BBB-crossing therapeutics; and statements by Drs. Chin and Jalazo. 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 tividenofusp alfa; 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 significance of Denali's PDUFA date for tividenofusp alfa (DNLI)?

The PDUFA date is April 5, 2026, marking an FDA target decision for the BLA. According to the company, the BLA is under Priority Review and Week 201 biomarker and clinical stabilization data support the submission and potential regulatory decision.

How much did DNL126 (ETV:SGSH) reduce CSF heparan sulfate in the Phase 1/2 study?

DNL126 produced a mean 80% reduction in CSF heparan sulfate at Week 49. According to the company, this result came from dose-finding cohorts and included normalization in several participants, with safety consistent with established enzyme replacement therapies.

What clinical durability was reported for tividenofusp alfa (DNL310) in MPS II (DNLI)?

Tividenofusp alfa reductions in CSF and urine HS were maintained through Week 201. According to the company, this sustained biomarker normalization aligned with stabilization or improvement in adaptive behavior, cognition, hearing, and liver volume.

What does Denali report about the safety profile of DNL126 and DNL310?

Both DNL126 and DNL310 showed safety profiles generally consistent with established ERTs. According to the company, the most common treatment-related events were infusion-related reactions and no new safety signals were identified in presented data.

What evidence did Denali present for DNL952 (ETV:GAA) in Pompe disease (DNLI)?

Denali presented the Phase 1 study design and preclinical data showing improved glycogen reduction versus a second-generation ERT. According to the company, preclinical results showed glycogen reductions in both skeletal muscle and brain in a Pompe mouse model.