Denali Therapeutics

Denali reports early commercial traction for Avlayah after FDA approval, with roughly $3.6 million in revenue in Q2 2024 and a revenue forecast of $10–12 million for Q3 2024. The approval validates the transferrin receptor platform and strengthens prospects for enzyme replacement therapies, antibodies and oligonucleotides. The Sanfilippo A program DNL593/XAFI shows strong biomarker effects and is targeting a BLA filing in 2027, while Alzheimer’s and FTD programs remain important but carry higher uncertainties. U.S. manufacturing, partnership decisions and potential label expansions for adults remain key operational challenges. » More on de.investing.com

Denali reported AVLAYAH sales of $3.6m in the second quarter and forecasts $10–12m for the third quarter, indicating an accelerated commercial rollout. The company has about $1.1bn in cash, which management says should fund plans through 2029. The Phase III COMPASS study is fully recruited; study data are expected at the end of 2027 and are planned for publication by mid-2028. Several other clinical programs could deliver value‑enhancing data in 2027, while manufacturing and regulatory risks continue to determine execution. » More on de.investing.com

Denali shares fell after it was announced that the phase-2b LUMA study of the LRRK2 inhibitor BIIB122 (DNL151), developed with Biogen, failed to meet primary or secondary endpoints. The drug showed peripheral kinase inhibition >90% and reduced a biomarker in cerebrospinal fluid but produced no clinical benefit; BIIB122 will not be pursued for the treatment of idiopathic Parkinson’s. Denali will continue the independent phase-2a BEACON study in carriers of pathogenic LRRK2 variants; data are expected in H1 2027. In premarket trading, Denali stock dropped about 2.9% and Biogen about 1%. » More on de.investing.com

Takeda terminated the collaboration on DNL593 for strategic reasons unrelated to the drug’s efficacy or safety, returning full development and IP control to Denali. Denali says the TransportVehicle technology remains a promising approach for neurodegeneration and will present Phase I/II findings for DNL593 by the end of 2026. Management views sole ownership of DNL593 as positive for future development. » More on finance.yahoo.com

Denali Therapeutics received $200 million in gross proceeds from a synthetic royalty financing with Royalty Pharma; the payment was triggered by the accelerated FDA approval of Tividenofusp Alfa (Avlayah) on March 24 and the transaction closed on March 27. The company gave no further details on contract terms or planned use of the funds. Avlayah’s approval for treating neurological manifestations of Hunter syndrome was based on a median reduction of cerebrospinal fluid heparan sulfate concentration by 91% at week 24, with 93% of patients reaching normal levels. Following the milestone, several research houses raised their price targets for Denali but kept their ratings unchanged. » More on de.investing.com