Y-mAbs Therapeutics announced that the US Food and Drug Administration (FDA) has approved its Danyelza (naxitamab) antibody therapy for the treatment of cancer patients with neuroblastoma.
Specifically, Y-mAbs’ (YMAB) Danyelza in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) was granted FDA approval for the treatment of pediatric patients 1 year of age and older and adult patients, who are suffering from relapsed or refractory high-risk neuroblastoma in the bone or bone marrow and who have demonstrated a partial response, minor response, or stable disease to prior therapy. Following the US regulatory approval, Danyelza is expected to be available in the US in the coming weeks.
Danyelza is a humanized, monoclonal antibody that targets the ganglioside GD2, which is found in various neuroectoderm-derived tumors and sarcomas. The antibody is administered to patients three times in a week in an outpatient setting and the treatment s repeated every four weeks.
“We believe that Danyelza in combination with GM-CSF is a much-needed treatment for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow who have historically not had approved treatments available,” said Y-mAbs CEO Claus Moller. “This approval of Y-mAbs’ first BLA represents a key step in working towards our mission of becoming a world leader in developing better and safer antibody-based oncology products addressing unmet pediatric and adult medical needs.”
Y-mAbs said that the FDA approval is supported by clinical evidence from two pivotal studies in patients with high-risk neuroblastoma experiencing refractory or relapsed disease. Danyelza appears to be well tolerated with few discontinuations of treatment in the clinical trials and adverse events were clinically manageable, the biopharmaceutical company added.
The FDA granted approval under the accelerated approval regulation based on the overall response rate and duration of response. The continued approval for Danyelza may be contingent upon verification and description of clinical benefits in a confirmatory trial, the company said. The product has received Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations from the FDA.
In anticipation of the Danyelza approval, H.C. Wainwright analyst Robert Burns earlier this month raised the stock’s price target to a Street high of $65 (49% upside potential) from $52, while reiterating a Buy rating.
“Upcoming catalysts for the remainder of 2020 and 2021 include: (1) potential FDA approval of naxitamab by November 30, 2020; (2) sale of the Priority Review Voucher (PRV) that Y-mAbs shall receive upon naxitamab FDA approval by year-end 2020 or in 1Q21,” Burns wrote in a note to investors.
The rest of the Street is in line with Burns’ bullish outlook on the stock. The Strong Buy analyst consensus is based on 4 Buys and 1 Hold. With shares already up 39% so far this year, the average analyst price target stands of $51.40, implies upside potential of another 17% over the coming 12 months.