DalcA, Under-skin Injectable Factor IX Prophylactic, Showing Potential in Phase 2 Trial, Catalyst Says

A preventive treatment for hemophilia B called dalcinonacog alfa (DalcA) led to an increase of more than 12% in the levels of factor IX in two patients with severe disease treated in a Phase 2 trial, reaching the study’s main goal.

Researchers also found no immune reaction against the therapy.

Enrollment is ongoing in the single site trial in South Africa (NCT03995784), but all six participants have been identified, Nassim Usman, PhD, president and chief executive officer of Catalyst announced in an update (a webcast is available here through December) at the Cantor 2019 Global Healthcare Conference, held in New York on Oct. 2–4.

Final study results are expected in June 2020.

DalcA (also known as CB2679d) is a potent recombinant (artificial) factor IX prophylactic (preventive) therapy designed to prevent acute bleeding episodes in people with hemophilia B. Unlike other factor IX products, DalcA can be administered by an injection under the skin (subcutaneous injection), a less invasive method to conventional intravenous injections.

Patients in the Phase 2b trial will receive a single intravenous loading dose of DalcA, followed by daily doses given subcutaneously for 28 days. The study is open-label, meaning both the clinician and the patient will know what is being administered (that is, there will be no placebo).

Its main endpoint or goal is to assess the dose required to achieve steady-state FIX levels above 12%. Additional goals include evaluations of safety and tolerability, pharmacokinetics (what the body does to a drug) and pharmacodynamics (what a drug does to the body), and immunogenicity, or  a patient’s risk of producing antibodies against DalcA that can interfere with its efficacy.

Previous data from a small proof-of-concept Phase 1/2 study (NCT03186677) showed that DalcA had greater potency than BeneFIX, an approved prophylactic, with DalcA inducing a 22 times more potent response than that observed with BeneFIX. The immunogenicity risk and drug quality of DalcA was also found to be identical to other factor IX products on the market.

“We believe that DalcA may offer a conveniently-dosed subcutaneous prophylactic treatment option that could significantly improve the quality of life for those suffering from severe hemophilia B,” Usman said in a press release.

“We are very pleased with the FIX activity and lack of ADAs [anti-drug-antibodies] obtained and look forward to providing final data in the first half of 2020,” he added.

DalcA has been granted orphan medicinal product designation in Europe to treat individuals with hemophilia B, and orphan drug designation by the U.S. Food and Drug Administration (FDA) for the same indication.