Rare Disease Report

FDA Orphan Drug Designation Granted to Potential Rare Cancer Treatment

APRIL 24, 2018
Mathew Shanley
This morning, Lin BioScience announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to LBS-007 for the treatment of acute lymphoblastic leukemia (ALL).

LBS-007, a targeted therapy for the treatment of ALL and other hematological cancers, has also been proven effective at combating solid tumors.

Per the National Institute of Health (NIH), approximately 876,000 people were affected by ALL globally in 2015, resulting in nearly 111,000 deaths. In the US, it is the most common cause of cancer and cancer-related deaths in children. The survival rates for leukemia vary depending on the type. Under the current standards of care, which typically include a combination of chemotherapy, radiation, and bone marrow transplantation, the overall 5-year survival rate for leukemias is approximately 60%.

"Our focus is to find treatments for untreatable diseases, and LBS-007 is the second candidate in our pipeline to receive Orphan Drug Designation in the last 6 months," stated Dr Tom Lin, CEO of Lin BioScience in a press release. "We are pleased to reach this regulatory milestone with LBS-007, and we look forward to advancing the anti-cancer program into Phase 1 clinical trials later this year."

LBS-007 is a natural, non-ATP cell cycle inhibitor with the potential to target a wide variety of cancers; it operates by inhibiting the S phase of the cell cycle. Its mechanism of action is to inhibit and block the kinase activity of a key regulator of the cancer cell cycle, known as CDC7. By inhibiting CDC7 and its role in DNA replication, the proliferation and division of tumor cells is prevented and cancer cells are killed.

Because CDC7’s activity is typically upregulated in cancer cells in comparison to healthy cells, it’s an idyllic candidate for a targeted anti-cancer therapy.

In preclinical studies, LBS-007 has exhibited very potent activity against leukemia and multiple solid tumors. Lin BioScience has stated its intentions to enter a first-in-human Phase 1 trial in drug-resistant and refractory acute leukemia patient population in Q4 of 2018.

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