雅虎香港財經 Denali's (DNLI) Candidate Selected for FDA's START Pilot Program
Denali Therapeutics, Inc. DNLI announced that the FDA has selected experimental candidate DNL126 in support of clinical trials advancing the rare disease therapeutics (START) Pilot Program. The candidate is an investigational enzyme replacement therapy designed to cross the blood-brain barrier (BBB) for the potential treatment of MPS IIIA (Sanfilippo syndrome type A).
MPS III, also known as Sanfilippo syndrome, is a rare, genetic lysosomal storage disease that causes neurodegeneration.
The newly launched START Pilot program was initiated by the FDA on Sep 29, 2023, to further accelerate the pace of the development of novel drugs and biological products that are intended to address the significant need in the treatment of rare diseases.
The pilot program was launched jointly by the FDA Center for Biologics Evaluation and Research's Office of Therapeutic Products and Center for Drug Evaluation and Research's Office of New Drugs.
Some of the participants of the START Pilot Program are provided opportunities to obtain frequent advice and engage in more rapid ad hoc communication with the FDA review staff to address product-specific development issues.
Denali is conducting a multicenter, open-label, phase I/II 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 six months, followed by an open-label extension of approximately 18 months.
Since DNL126 has been selected as a START participant, it can interact with the FDA frequently. The company expects the increased level of engagement from the FDA, to facilitate alignment on the most efficient development path to ultimately support a marketing application for DNL126 in MPS IIIA.
Please note that Denali has another candidate in its pipeline, tividenofusp alfa (DNL310), which is engineered to cross the BBB via receptor-mediated transcytosis into the brain. The candidate is being developed as a potential treatment for people living with MPS II (Hunter syndrome). Enrollment in the phase II/III COMPASS study on the candidate is expected to be completed this year.
Shares of DNLI have lost 1.7% year to date compared with the industry’s 5.6% decline.
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The company is making an encouraging pipeline progress. It has collaborations with bigwigs like Biogen BIIB and Sanofi SNY for some of its candidates.
Denali and Biogen are jointly evaluating an LRRK2 inhibitor BIIB122/DNL151, which is in development for the treatment of Parkinson’s disease (PD).
In February, Denali executed a funding agreement with a third party related to a global phase IIa study of BIIB122/DNL151. Denali plans to operationalize the study to evaluate safety and biomarkers associated with BIIB122 in participants with PD and confirm pathogenic variants of LRRK2. The phase IIa study is expected to be initiated in 2024.
Meanwhile, Biogen is conducting the ongoing global phase IIb LUMA study of BIIB122 in participants with early-stage PD.
Denali and partner Sanofi are co-developing SAR443820/DNL788. While the ALS study on this candidate has been discontinued, Sanofi continues to evaluate SAR443820 in another phase II study for the treatment of multiple sclerosis.
Denali and Sanofi are also developing SAR443122/DNL758 (eclitasertib), a peripheralRIPK1 inhibitor, for the treatment of ulcerative colitis.
Zacks Rank and a Stock to Consider
Denali currently carries a Zacks Rank #3 (Hold).
A better-ranked stock in the healthcare sector is Krystal Biotech, Inc. KRYS which currently carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 30 days, the Zacks Consensus Estimate for KRYS’ earnings per share has increased 24 cents to $2.06. KRYS beat on earnings in two of the trailing four quarters and missed the mark in the other two, delivering an average negative surprise of 21.46%. Shares of Krystal Biotech have surged 31.3% year to date.
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