We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Denali Plans to File for Accelerated Approval of Hunter Syndrome Drug
Read MoreHide Full Article
Denali Therapeutics Inc. (DNLI - Free Report) announced that its meeting with the Center for Drug Evaluation and Research (“CDER”) division of the FDA for the advancement of pipeline candidate tividenofusp alfa (DNL310) was successful.
Tividenofusp alfa (DNL310) is being evaluated for the treatment of MPS II (Hunter syndrome).
The meeting with CDER outlined a path for Denali to file a biologics license application (BLA) seeking accelerated approval of tividenofusp alfa (DNL310) for the treatment of MPS II and its subsequent conversion to full approval.
Shares of this biopharmaceutical company have gained 17.8% in the year to date against the industry’s decline of 1%.
Image Source: Zacks Investment Research
Denali’s Plans for Tividenofusp Alfa
Denali reached an agreement with the CDER that cerebrospinal fluid heparan sulfate (CSF HS) is reasonably likely to predict clinical benefit and can be used as a surrogate endpoint to support accelerated approval for tividenofusp alfa in MPS II.
As per discussions with CDER, Denali will include preclinical and clinical data on biomarkers (CSF HS and neurofilament light [NfL]) and safety in the BLA for tividenofusp alfa as a treatment of MPS II.
Denali plans to submit the BLA under the accelerated approval pathway in early 2025.
DNLI is currently enrolling in the phase II/III COMPASS study on tividenofusp alfa in North America, South America, Europe and Australia, based on supportive clinical and preclinical data to date. The study will likely enroll approximately 54 participants with MPS II (with and without neuronopathic disease). The participants are randomized 2:1 to receive either tividenofusp alfa or idursulfase.
Data from a phase I/II study showed treatment with tividenofusp alfa produced robust and durable effects, with normalization of key disease biomarkers and improvement or stabilization in associated CNS and somatic clinical endpoints.
Denali Presents New Data on Tividenofusp Alfa
Denali also announced that new interim data from the phase I/II study is currently being presented at the Symposium of the Society for the Study of Inborn Errors of Metabolism. The presentations include data from additional study participants (n=37) and longer duration of treatment with tividenofusp alfa (up to week 129) as well as new analyses on biomarkers and clinical outcomes.
The data showed a 90% mean reduction in CSF HS from baseline at week 24, with all participants having normal or near normal levels at week 24. CSF HS reduction was sustained through week 104.
The proportion of participants with normal total urine glycosaminoglycans (colorimetric method) increased to 77% at week 24 from 5% of participants at baseline. This effect was sustained through week 129.
Patients experienced a significant and sustained reduction of serum NfL from baseline. All participants who reached week 129 had normal or near-normal levels of serum NfL, suggesting a reduction of neuronal injury in participants with MPS II. Improvement or stabilization in adaptive behavior and cognitive scores, hearing, liver volume and growth outcomes was observed.
Our Take
The successful development of tividenofusp alfa will be a significant boost for the company, as Denali does not have any approved candidate in its portfolio.
The FDA had earlier granted Fast Track designation to DNL310 for the treatment of patients with MPS II. In May 2022, the European Medicines Agency granted Priority Medicines designation to the candidate.
Denali has collaborated with Biogen (BIIB - Free Report) for the development of BIIB122/DNL151, a LRRK2 inhibitor for the treatment of Parkinson’s disease (PD). Biogen is conducting the ongoing global phase IIb LUMA study of BIIB122 in participants with early-stage Parkinson’s disease.
However, last month, Denali announced that Biogen terminated its license to the Antibody Transport Vehicle Amyloid beta (ATV:Abeta) program enabled by Denali’s TfR-targeting technology against amyloid beta for the potential treatment of Alzheimer's disease.
Denali also has a collaboration agreement with Sanofi for some of its pipeline candidates.
Krystal Biotech’s earnings per share (EPS) estimate for 2024 has increased to $2.38 from $1.91 in the past 30 days. Shares of KRYS have surged 50.4% year to date.
In the past 30 days, EXEL’s EPS estimate for 2024 has moved north to $1.79 from $1.39. Year to date, shares of EXEL have risen 6.2%.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
Denali Plans to File for Accelerated Approval of Hunter Syndrome Drug
Denali Therapeutics Inc. (DNLI - Free Report) announced that its meeting with the Center for Drug Evaluation and Research (“CDER”) division of the FDA for the advancement of pipeline candidate tividenofusp alfa (DNL310) was successful.
Tividenofusp alfa (DNL310) is being evaluated for the treatment of MPS II (Hunter syndrome).
The meeting with CDER outlined a path for Denali to file a biologics license application (BLA) seeking accelerated approval of tividenofusp alfa (DNL310) for the treatment of MPS II and its subsequent conversion to full approval.
Shares of this biopharmaceutical company have gained 17.8% in the year to date against the industry’s decline of 1%.
Image Source: Zacks Investment Research
Denali’s Plans for Tividenofusp Alfa
Denali reached an agreement with the CDER that cerebrospinal fluid heparan sulfate (CSF HS) is reasonably likely to predict clinical benefit and can be used as a surrogate endpoint to support accelerated approval for tividenofusp alfa in MPS II.
As per discussions with CDER, Denali will include preclinical and clinical data on biomarkers (CSF HS and neurofilament light [NfL]) and safety in the BLA for tividenofusp alfa as a treatment of MPS II.
Denali plans to submit the BLA under the accelerated approval pathway in early 2025.
DNLI is currently enrolling in the phase II/III COMPASS study on tividenofusp alfa in North America, South America, Europe and Australia, based on supportive clinical and preclinical data to date. The study will likely enroll approximately 54 participants with MPS II (with and without neuronopathic disease). The participants are randomized 2:1 to receive either tividenofusp alfa or idursulfase.
Data from a phase I/II study showed treatment with tividenofusp alfa produced robust and durable effects, with normalization of key disease biomarkers and improvement or stabilization in associated CNS and somatic clinical endpoints.
Denali Presents New Data on Tividenofusp Alfa
Denali also announced that new interim data from the phase I/II study is currently being presented at the Symposium of the Society for the Study of Inborn Errors of Metabolism. The presentations include data from additional study participants (n=37) and longer duration of treatment with tividenofusp alfa (up to week 129) as well as new analyses on biomarkers and clinical outcomes.
The data showed a 90% mean reduction in CSF HS from baseline at week 24, with all participants having normal or near normal levels at week 24. CSF HS reduction was sustained through week 104.
The proportion of participants with normal total urine glycosaminoglycans (colorimetric method) increased to 77% at week 24 from 5% of participants at baseline. This effect was sustained through week 129.
Patients experienced a significant and sustained reduction of serum NfL from baseline. All participants who reached week 129 had normal or near-normal levels of serum NfL, suggesting a reduction of neuronal injury in participants with MPS II. Improvement or stabilization in adaptive behavior and cognitive scores, hearing, liver volume and growth outcomes was observed.
Our Take
The successful development of tividenofusp alfa will be a significant boost for the company, as Denali does not have any approved candidate in its portfolio.
The FDA had earlier granted Fast Track designation to DNL310 for the treatment of patients with MPS II. In May 2022, the European Medicines Agency granted Priority Medicines designation to the candidate.
Denali has collaborated with Biogen (BIIB - Free Report) for the development of BIIB122/DNL151, a LRRK2 inhibitor for the treatment of Parkinson’s disease (PD). Biogen is conducting the ongoing global phase IIb LUMA study of BIIB122 in participants with early-stage Parkinson’s disease.
However, last month, Denali announced that Biogen terminated its license to the Antibody Transport Vehicle Amyloid beta (ATV:Abeta) program enabled by Denali’s TfR-targeting technology against amyloid beta for the potential treatment of Alzheimer's disease.
Denali also has a collaboration agreement with Sanofi for some of its pipeline candidates.
DNLI’s Rank and Stocks to Consider
Denali currently carries a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the biotech sector are Krystal Biotech (KRYS - Free Report) and Exelixis (EXEL - Free Report) . While KRYS sports a Zacks Rank #1 (Strong Buy) at present, Exelixis carries a Zack Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Krystal Biotech’s earnings per share (EPS) estimate for 2024 has increased to $2.38 from $1.91 in the past 30 days. Shares of KRYS have surged 50.4% year to date.
In the past 30 days, EXEL’s EPS estimate for 2024 has moved north to $1.79 from $1.39. Year to date, shares of EXEL have risen 6.2%.