Actinium Pharmaceuticals (NYSE American:ATNM) is improving patient access to bone marrow transplants (BMT) and treating cancers with high unmet medical need by combining the precision of sophisticated proteins, with the cell killing ability of alpha-emitting isotopes, to ablate cancer cells and leave healthy tissue intact.
“We’re arming targeted agents, such as monoclonal antibodies, with isotope payloads and essentially creating heat seeking missiles against cancer,” Steve O’Loughlin, principal financial and accounting officer, says in an interview with BioTuesdays.
Actinium is focused on developing targeted therapies to treat diseases, such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and multiple myeloma (MM).
Mr. O’Loughlin explains that Actinium’s most advanced product candidate, Iomab-B, was licensed from the Fred Hutchinson Cancer Research Center, and is designed to be used, if approved, in preparing patients for a hematopoietic stem cell transplant, more commonly known as BMT, by conditioning the bone marrow without the need for intense chemotherapy.
Prior to the BMT procedure, physicians administer a conditioning regiment that ablates the bone marrow to destroy cancer cells, makes way for the new donor marrow, and decreases the body’s ability to reject the transplant.
“Often the only potential cure for patients with blood-borne cancers is a BMT,” Mr. O’Loughlin says.
Iomab-B has been granted orphan drug designation for relapsed or refractory AML in senior patients by the FDA and the European Medicines Agency (EMA).
“Currently, the only conditioning for BMT is salvage chemotherapy and/or total body irradiation, both of which really beat the patient up because they are so toxic, and kill healthy tissue,” he adds.
In an MD Anderson Cancer Center outcomes analysis of 100 elderly patients with relapsed or refractory AML, conditioned with salvage chemotherapy and followed by BMT, zero percent of the patients survived at the two-year mark.
On the other hand, Mr. O’Loughlin points out that data from a Phase 2 Fred Hutchinson trial where patients were conditioned with Iomab-B prior to BMT, demonstrated that nearly 20% lived to celebrate their second anniversary, and were told they would most likely die of causes unrelated to AML.
Actinium expects to complete enrollment in 2019 in a 150-patient, Phase 3 SIERRA study of Iomab-B in relapsed or refractory AML in the elderly.
“We are really looking forward to the SIERRA interim results that are expected as early as this fall,” Mr. O’Loughlin says. “Our plan is to file with the FDA in early 2020 and commercialize Iomab-B ourselves.”
In addition to Iomab-B, Mr. O’Loughlin contends that Actinium has created a potentially best-in-class CD33 program. “We’re the only company to have CD33 targeting agents going after multiple disease indications, including AML, MDS and MM.”
CD33 is an antigen found on hematopoietic cells in certain blood cancers. The Actinium CD33 program is currently being studied in five Phase 1 and Phase 2 clinical trials for elderly patients with AML, MDS, and MM.
Mr. O’Loughlin notes that underpinning Actinium’s CD33 program, is its Warhead Enabling (AWE) technology platform, where the company uses differentiated attributes of the potent cytotoxic radioisotope, actimium-225 (AC-225), in conjunction with select targeting agents.
Actinium’s lead AWE product candidate, Actimab-A, is a second-generation therapy from the HuM195-Alpha program developed at Memorial Sloan Kettering Cancer Center, Mr. O’Loughlin says.
“It’s being developed to induce remissions in elderly patients with AML, who lack effective treatment options, and often cannot tolerate the toxicities of standard frontline therapies,” he adds.
Actimab-A has been granted orphan drug designation from both the FDA and the EMA for newly diagnosed AML in elderly patients.
Another CD33 product candidate, Actimab-M, is being studied in a Phase 1 clinical trial for patients with relapsed or refractory MM. The trial will determine a maximum tolerated dose, assess adverse events, measure response rates, progression-free survival, and overall survival.
Mr. O’Loughlin says Actinium has also expanded its CD33 program beyond therapeutic use, and into targeted conditioning, with Actimab-MDS, “to provide treatment for the most difficult-to-treat MDS patients by rapidly and safely bridging them to transplant.”
In a Fred Hutchinson Phase 2 study, high-risk MDS patients were able to go to BMT after only one infusion of Actimab-MDS to condition bone marrow, he adds. “This drug is so targeted, we see very few side effects other than myeloablation, which is the burning out of the patient’s bone marrow, and which is necessary to the procedure.”
Actinium is finalizing discussions with the FDA for Actimab-MDS in order to set a clear path to a pivotal trial, Mr. O’Loughlin says.
Actimab-MDS is currently involved in a Phase 2 trial being conducted in collaboration with the MDS Clinical Research Consortium, which consists of the Cleveland Clinic, Johns Hopkins, Dana-Farber Cancer Institute, MD Anderson Cancer Center and Moffitt Cancer Center.
According to Mr. O’Loughlin, Actinium is the only company worldwide with a multi-product, multi-disease pipeline focused on improved transplant access and outcomes, via significantly enhanced conditioning.
“These are exciting times for Actinium as we’re working toward creating a world-class supply chain, and, by the 2020-21 timeframe, we expect to have two distinct drugs on the market, both of which will be in targeted conditioning but for different indications in 50 of the leading U.S. transplant centers,” he says.
via Features | BioTuesdays by Kilmer Lucas https://ift.tt/2Mla2Ia