Long on $DAWN!
$DAWN received a priority review with a PDUFA on April, 30, 2024 based on FIREFLY-1 Phase II readout. In the meantime, $DAWN was awarded Priority Review Voucher(PRV) upon approval. It is reported to have a sell-price ranging from 67M to 350M, a huge value addition upon approval.
Tovorafenib is the candidate under evaluation. It is a type II RAF inhibitor, targeting both mutation and fusion BRAF alterations. This pan-RAF inhibitor is superior to the marketed first gen RAF inhibitors, including dabrafenib, encorafenib, and vemurafenib, which have already showed clinical benefit in BRAF V600 mutation-driven melanoma patients.
The indication it is going after is pLGGs(pediatric Low grade gliomas), the most common brain tumor in children (representing ⅓ of pediatric CNS tumors), and BRAF mutation is the main driver behind the disease. The total addressable patient population is estimated ~2000-3000 per year.
The Phase II result looks pretty good, and got published in nature medicine. A few takeaways are in the following:
Clinical study subjects involved 77 of >=1 prior line treated patients aged 6 months to 25 years.
Efficacy: primary endpoint ORR hit 67% based on RANO-HGG assessment. The newly published RANO assessment also gave a 51% ORR. IMO, pretty impressive considering the patients with radiographic progression after >=1 prior line treatment.
Very manageable safety profile: Hair color changes were the main side effect, along with anemia and fatigue.
Once weekly dosing (oral tablet or liquid formulation) children-friendly
Cash runway into 2026.
Disclaimer: Community is offered by Moomoo Technologies Inc. and is for educational purposes only.
Read more
Comment
Sign in to post a comment
