Shares of Catalyst Biosciences Inc. (CBIO) are up an impressive 100% year-to-date in sharp contrast to the NASDAQ Biotechnology Index, which is down nearly 2% during the same period.
Catalyst Biosciences is focused on developing therapies for hemophilia. Before we go into the details of the Company's pipeline, here's what hemophilia means in a nutshell.
Hemophilia is a rare but serious bleeding disorder. There are two main types - Hemophilia A, caused due to deficiency or dysfunction of clotting protein factor VIII, and Hemophilia B, caused due to deficiency or dysfunction of clotting protein factor IX.
The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor VIII or IX to control and prevent bleeding episodes.
But some people with hemophilia will develop inhibitors, making it difficult to treat. When a person develops an inhibitor, the body stops accepting the factor treatment product as a normal part of blood, thinking that the factor is a foreign substance and tries to destroy it with an inhibitor. (Source: CDC).
When we alerted our premium subscribers to CBIO on November 25, 2017 (Report titled "Will the Data Win over Investors?"), it was trading around $6.56. The stock touched an all-time high of $29.20 in intraday trading yesterday.
The Company's lead drug candidate is Marzeptacog alfa (activated) a potent, subcutaneously administered, next-generation Factor VIIa therapy being developed for prophylaxis in hemophilia A or B with inhibitors.
The phase II part of the phase II/III program of Marzeptacog alfa was initiated as recently as last month.
The trial is designed to enroll up to 12 individuals with hemophilia and an inhibitor across approximately ten clinical trial sites globally. Interim data is expected to be announced in the first half of 2018.
Next in the pipeline is Factor IX protease, CB 2679d, also known as ISU304, for the potential treatment of hemophilia B, under phase I/II testing.
This phase I/II clinical trial includes single intravenous and subcutaneous dosing cohorts, followed by daily subcutaneous injections of CB 2679d.
Data reported last September from the first Cohort of the study demonstrated a 22-fold higher potency and improved pharmacokinetics with intravenous dosing of CB 2679d compared with BeneFIX in individuals with hemophilia B. (*BeneFix is the most-prescribed recombinant factor IX treatment for hemophilia B).
The interim results from the phase I/II subcutaneous dosing Cohort reported last December demonstrated that subcutaneous delivery significantly increases half-life of CB 2679d to 98.7 hours, suggesting that CB 2679d may provide superior prophylaxis capabilities compared with intravenous extended half-life agents, with the potential to normalize FIX levels.
The Company will be presenting results from daily subcutaneous doses of CB 2679d on Factor IX blood levels today (Feb.9, 2018).
Catalyst Biosciences had cash of $27.5 million at September 30, 2017, and it raised gross proceeds of roughly $10.5 million in a public offering last December.
CBIO has traded in a range of $3.11 to $29.20 in the last 1 year. We are just into the second month of the New Year, and the stock has already had a pretty terrific performance. The stock closed Thursday's trading at $27.28, down 0.91%. Is there still more room to grow?
by RTT Staff Writer
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