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Patients with the rare disease rhabdomyosarcoma (RMS) and investors interested in rare disease designations alike are showing excitement over a new designation granted.
Patients with the rare disease rhabdomyosarcoma (RMS) and investors interested in rare disease designations alike are showing excitement over a new designation granted.
On Wednesday (June 6), Cellectar Biosciences (NASDAQ:CLRB) received rare pediatric disease designation (RPDD) from the US Food and Drug Administration (FDA) for its drug candidate CLR 131 for the treatment of RMS, a rare pediatric cancer.
“There is a critical need for new therapies in the fight against deadly diseases such as RMS and we continue to increase our focus on delivering innovative solutions to patients suffering from such rare cancers,” John Friend, chief medical officer of Cellectar said in the release.
This is the second RPDD CLR 131 has received, with the first one issued at the beginning of May for the treatment of neuroblastoma. If the drug is approved by the FDA, the designation may allow Cellectar to receive priority review voucher. The voucher can be used for a future new drug application or biologics license application, reducing the FDA’s review time by six months.
If the company chooses not to use the voucher for its own products it can be sold to other entities; over the last 16 months vouchers were sold for as high as US$150 million.
The product candidate is a small-molecule which uses the company’s Phospholipid Drug Conjugate technology to deliver cytotoxic radiation directly and selectively to cancer cells. Aside from these indications, CLR 131 is being evaluated in Phase 1 and 2 trials for multiple myeloma, hematologic tumors and head and neck indications.
RPDD’s are granted to to disease indications for serious or life-threatening diseases for children to encourage development of new drugs a biologics for rare pediatric diseases.
RMS is a malignant tumor which is the most common soft tissue sarcoma in children. It accounts for about 40 percent of childhood soft tissue sarcomas in the US with about 4.5 cases per one million children aged younger than 15 years. There is a five-year survival rate of 64 percent and the median progression-free survival after the first recurrence or progression is about nine months.
Cellectar is focused on discovering, developing and commercializing drugs in oncology. Aside from CLR 131 the company has six other programs primarily in preclinical development. Other candidates have indications for hematologic tumors and solid tumors.
The company has a variety of collaborators and partnerships including Avicenna Oncology, Onconova Therapeutics (NASDAQ:ONTX) and the University of Wisconsin-Madison among others.
Investor Takeaway
After the announcement Wednesday Cellectar’s share price increased over 16 percent earlier in the day but evened out at 10.88 percent to $1.02 at market close. The company’s share price has seen a gradual downturn over the past year, but if the company continues to make announcements such as this the share price may make a gradual return back up.
With most of its drugs in preclinical development, investors interested in the company need to be patient with the company as it nears Phase 3 trials for CLR 131 in hematologic tumors and multiple myeloma.
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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.