Shares of Forty Seven (NASDAQ:FTSV) rose as much as 95.6% today after the company provided an encouraging update for its lead drug candidate at the Annual Meeting of the American Society of Hematology. The oddly named company is developing the monoclonal antibody magrolimab as a treatment for two rare blood disorders: myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).
The development-stage company also provided an update for FSI-174, a preclinical asset on track to advance to a phase 1 clinical trial in the first quarter of 2020. It will be studied as a combination therapy with magrolimab as a conditioning regimen ahead of hematopoietic stem cell (HSC) transplantation. The all-antibody approach could be a significant advance, as the current standard of care relies on chemotherapy or radiation treatment to wipe out a patient's HSCs ahead of a transplant.
As of 12:45 p.m. EST, the pharma stock had settled to a 83.7% gain.
Forty Seven reported promising objective response rates (ORR) and complete response rates (CRR) for magrolimab. The ORR measures how many patients involved in the study showed any response to treatment, while the CRR measures how many had no evidence of disease (or who had significant recoveries in blood count) following treatment.
In untreated higher-risk MDS patients, magrolimab achieved an ORR of 92% and a CRR of 50%. In untreated AML patients who were ineligible for chemotherapy, the drug candidate achieved an ORR of 64% and a CRR of 55%.
The results are impressive considering only 25% of those affected with MDS and AML are eligible for existing treatment options. That could help the drug's path to market. Forty Seven told investors it remains on track to file a biologics license application with the U.S. Food and Drug Administration in late 2021.
Forty Seven ended September with $166.7 million in cash, but investors shouldn't be surprised if it wisely takes advantage of a soaring stock price to conduct a public stock offering. After all, the company has seven programs in clinical development and five more in preclinical stages of study. Raising funding now would pad the balance sheet and allow the business to focus on research and development efforts.