Benzinga | Oct 28, 2020 07:12AM EDT

ALX Oncology Announces First Patient Dosed in ASPEN-02, a Phase 1/2 Study of ALX148 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome

ALX Oncology Holdings Inc. ("ALX Oncology") (NASDAQ:ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, today announced the first patient has been dosed in the Phase 1/2 ASPEN-02 study evaluating the combination of ALX148, a next-generation CD47 blocker, with azacitidine for the treatment of patients with higher-risk myelodysplastic syndrome ("MDS").

The Phase 1 part of the study is expected to characterize the safety of ALX148 in combination with azacitidine in patients with relapsed/refractory or previously untreated higher-risk MDS. Upon completion of the Phase 1, the Phase 2 component of the study will be initiated to evaluate the efficacy of the combination in patients with previously untreated higher-risk MDS.

This study has been initiated based on promising preclinical data of ALX148 in combination with azacitidine in myeloid leukemia models, and clinical data generated by ALX Oncology in an ongoing Phase 1 trial (NCT03013218) evaluating ALX148 in combination with other anti-cancer agents in over 150 patients with various malignancies, including non-Hodgkins lymphoma, gastric/gastroesophageal junction cancer ("GC"), and head and neck squamous cell carcinoma ("HNSCC"). Data from the Phase 1 study provides the basis for ALX148's Fast Track Approvals in GC and HNSCC granted by the U.S. Food and Drug Administration.

"We are looking forward to evaluating the addition of ALX148 to azacitidine in patients with advanced MDS who are in need of effective new therapies," said Guillermo Garcia-Manero, M.D., Professor and Chief of Section of Myelodysplastic Syndromes, Department of Leukemia at MD Anderson Cancer Center. "ALX148 was designed for use in combination with a range of agents to maximize anti-cancer activity while minimizing associated toxicity."

"Through blockade of the CD47 myeloid checkpoint pathway, ALX148 bridges the innate and adaptive immune responses to cancer. This study builds upon the compelling combination activity observed in patients with ALX148 and multiple other anti-cancer agents," said Sophia Randolph, M.D., Ph.D., Chief Medical Officer of ALX Oncology. "Our goal is to transform treatment options for patients with cancer by developing ALX148 as a foundational checkpoint immunotherapy."

The ASPEN-02 trial is registered under NCT04417517. ALX Oncology owns worldwide commercial rights to ALX148.

About Myelodysplastic Syndrome ("MDS")

MDS represents a group of blood cancers characterized by ineffective production of healthy red blood cells, white blood cells, and platelets. This can lead to anemia with a potential need for frequent transfusions, infections, and a risk for transformation into leukemia. Over 10,000 people are estimated to be diagnosed with MDS in the U.S. each year. The average survival rate for those with higher-risk MDS is approximately 18 months.