Galena Biopharma, Inc., a biopharmaceutical company committed to the development and commercialization of hematology and oncology therapeutics that address unmet medical needs, announced the company has confirmed the regulatory pathway to advance GALE-401 (Anagrelide Controlled Release) into a pivotal, phase 3 trial. After a productive meeting with the US Food and Drug Administration (FDA), the company has confirmed the development programme and proposed clinical trial are appropriate for a New Drug Application (NDA) filing using the 505(b)(2) regulatory pathway.
The phase 3 clinical trial will enroll patients with essential thrombocythemia who have either failed or are intolerant to hydroxyurea, and will compare GALE-401 to the best available therapy (BAT) that will include a sizable subpopulation of patients treated with anagrelide immediate release. The company expects to finalize the phase 3 clinical trial protocol and initiate the trial in Q2, 2017.
“Confirmation of the 505(b)(2) regulatory pathway and preliminary agreement with the Agency on our phase 3 clinical trial design is a significant step forward for our late stage programme,” Mark W. Schwartz, Ph.D., president and chief executive officer. “Our controlled release version of anagrelide can offer a potential treatment option for patients with ET who have failed hydroxyurea. We remain on track to initiate the trial in the second quarter of 2017.”
GALE-401 is a controlled release formulation of anagrelide (Anagrelide CR) currently in clinical development for essential thrombocythemia. The currently available immediate release formulation (Agrylin or anagrelide IR) is approved by the FDA for the treatment of patients with thrombocythemia, secondary to myeloproliferative disorders, to reduce the elevated platelet count and the risk of thrombosis and to ameliorate associated symptoms including thrombo-hemorrhagic events. Adverse events associated with anagrelide IR, such as nausea, diarrhea, abdominal pain, palpitations, tachycardia, and headache, may be dose and plasma concentration dependent. GALE-401 is a reformulated, controlled release version of anagrelide that reduces the maximum plasma concentration (Cmax) of the drug and is expected to reduce side effects, but preserve efficacy. A phase 2 pilot study with GALE-401 has been completed.
Essential Thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by the overproduction of platelets by megakaryocytes in the bone marrow. The US prevalence of ET is between 135,000 and 175,000 with approximately seventy-five percent of patients receiving treatment. Common symptoms include headache, vision disturbances or migraines, dizziness or lightheadedness, coldness or blueness of fingers or toes, burning, redness, and pain in the hands and feet. Complications for patients with ET include blood clotting or bleeding or may be thrombotic in nature such as stroke, heart attack, or transient ischemic attack.
As with other MPNs, ET is a progressive blood cancer that can strike anyone at any age, and for which there is no known cure; and, there is no single treatment option that is appropriate or effective for all ET sufferers. While some ET patients may be asymptomatic and require no treatment, others may require various treatments and therapies based on the symptoms, their risk factors, and potential complications. The treatment options are limited and are generally hydroxyurea prescribed first line, followed by other treatments including anagrelide immediate release, interferon, aspirin or other agents depending on the patient’s condition. Of these, only anagrelide IR is approved for treatment of ET patients.