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Denali (DNLI) Begins Frontotemporal Dementia-Granulin Study

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Denali Therapeutics Inc. (DNLI - Free Report) announced that it has initiated a phase I/II study of its pipeline candidate DNL593 for the potential treatment of frontotemporal dementia (FTD) caused by mutations in the granulin gene (GRN).

FTD is the most common form of dementia in people under 60 years of age and FTD-GRN is characterized by progranulin (PGRN) deficiency in the brain. DNL593 is an investigational brain-penetrant, recombinant PGRN replacement therapy aiming to slow or prevent progression of FTD-GRN by increasing intracellular and extracellular levels of functional PGRN.

DNLI expects to begin dosing individuals with FTD-GRN in the second half of the year, pending initial clinical data from the healthy volunteer portion of the phase I study.

The phase I/II study is a three-part (Parts A-C) multicenter, randomized, double-blind, placebo-controlled clinical trial exploring safety, pharmacokinetics and pharmacodynamics of DNL593 in healthy volunteers and patients with FTD-GRN. Part A is a single ascending dose study in healthy volunteers and dosing has begun in this study.  Part B is a multiple dose, 25-week study in patients with FTD-GRN while part C is an 18-month open label extension study of patients who participated in Part B.

We note that Denali has a strategic collaboration with Takeda (TAK - Free Report) to co-develop and co-commercialize DNL593. DNLI may receive future milestone payments from Takeda upon achieving certain clinical and regulatory milestone events as well as certain sales-based milestones. Both companies will share the development and commercialization costs equally, subject to the terms of the collaboration agreement.

The Medicines & Healthcare products Regulatory Agency (MHRA) approved the Clinical Trial Application (CTA) in the United Kingdom in January 2022, which triggered a $12-million milestone from Takeda (received in February 2022).

Shares of Denali have declined 30.6% in the year so far compared with the industry’s 17% decrease.

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Denali is developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases. It forged alliances with bigwigs like Takeda and Biogen among others to advance its pipeline.

DNLI also has a collaboration agreement with Biogen (BIIB - Free Report) for its Parkinson’s disease (PD) candidate. In August 2020, DNLI and BIIB announced the selection of BIIB122/DNL151, a small molecule inhibitor of LRRK2, to advance into two late-stage studies.

While the phase IIb LUMA study will evaluate the inhibitor in PD patients without LRRK2 mutations, the phase III LIGHTHOUSE study will evaluate DNL151 in PD patients with LRRK2 mutations. Biogen will lead both studies.

Denali currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Vertex Pharmaceuticals (VRTX - Free Report) , which currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

The consensus estimate for Vertex’s 2022 earnings has increased $1.20 over the past 60 days to $14.52. Shares of VRTX have gained  9.4% in the past year.

 


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