The U.S. Food and Drug Administration (FDA) has granted fast track designation to HDP-101 (pamlectabart tismanitin) for the treatment of multiple myeloma.
The development was announced in a news release issued by Heidelberg Pharma AG, the clinical-stage biotech company behind HDP-101 (pamlectabart tismanitin), which is a type of treatment known as an antibody-drug conjugate, or ADC.
The designation, according to the news release, was supported by both nonclinical and clinical data from an ongoing phase 1/2a study of HDP-101 that is evaluating the safety and antitumor activity of the treatment among patients with relapsed or refractory multiple myeloma.
“The FDA’s granting of Fast Track Designation is fantastic news for Heidelberg Pharma and underscores the potential of HDP-101 for the treatment of severely ill and heavily pretreated patients,” said professor Andreas Pahl, chief executive officer of Heidelberg Pharma, in a statement in the news release. “This designation will support our efforts to advance our lead ADC candidate efficiently toward patients with multiple myeloma who continue to face significant unmet medical needs.”
Glossary
Partial Response: when cancer treatment makes the tumors smaller or reduces signs of cancer in the body, but does not make them disappear completely. Sometimes also called a “partial remission.”
Complete Response: when all signs of cancer disappear after treatment. This means no cancer can be seen on scans or tests, though doctors will continue to monitor closely in case it returns.
Thrombocytopenia: a lower-than-normal number of platelets in the blood. Platelets help your blood to clot, so when they are low, you may bruise or bleed more easily.
Arthralgia: pain or aching in one or more joints (like knees, hips, or shoulders). It can sometimes be a side effect of cancer treatment.
Anemia: a condition in which you don’t have enough healthy red blood cells to carry oxygen through your body. It can make you feel tired, weak, or short of breath.
Neutropenia: a lower-than-normal number of neutrophils, a type of white blood cell that helps fight infection. When neutrophil counts are low, you may be more likely to get infections.
Fast track, as the FDA explains on its website, is a process designed to deliver important new therapies to patients earlier by facilitating the development and accelerating the review of drugs that treat serious conditions with unmet medical needs.
HDP-101 was shown to have produced significant preliminary efficacy with a manageable toxicity profile in data from the phase 1 HDP-101-01 trial shared at the 21st Annual International Myeloma Society Meeting and Exposition in 2024, according to a report from CURE’s sister publication, CancerNetwork.
In cohort 5 of the trial, in which patients received HDP-101 at 100 μg/kg, five of six patients saw a decrease in paraprotein percent changes from screening. Moreover, three had a partial response (PR) or better, and one experienced a complete response (CR), according to the report.
Common any-grade and grade 3 (severe) or 4 (life-threatening) treatment-emergent side effects, respectively, included thrombocytopenia (56% versus 39%), arthralgia (33% versus 0%), anemia (33% versus 22%), fatigue (28% versus 0%) and neutropenia (22% vs 17%).
The trial, according to its listing on clinicaltrials.gov, launched on Feb. 7, 2022 and has an estimated primary completion date of August 2025, followed by an approximate study completion date of May 2026.
According to the listing, the trial is still recruiting patients and will eventually enroll an estimated 78 patients with relapsed or refractory multiple myeloma. The trial is currently being conducted at 16 sites in the United States, Germany, Hungary and Poland, with U.S. locations including The University of Texas MD Anderson Cancer Center in Houston, Winship Cancer Institute of Emory University in Atlanta and The Tisch Cancer Institute at Mount Sinai in New York.
An antibody-drug conjugate such as HDP-101, as defined by the National Cancer Institute on its website, is a substance made up of a monoclonal antibody chemically linked to a drug. Upon administration, the monoclonal antibody binds to specific proteins or receptors found on certain types of cells, such as cancer cells, and the linked drug enters these cells and kills them without harming other cells.
HDP-101 is still an investigational product that has not received marketing approval from any regulatory authority, such as the FDA, the news release clarifies, noting that the safety and efficacy of the investigational compound are currently being evaluated and are not yet established.
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References:
- “PR: Heidelberg Pharma’s Lead ADC Candidate HDP-101 Granted Fast Track Designation by US FDA for the Treatment of Multiple Myeloma,” news release; https://heidelberg-pharma.com/en/press-investors/announcements/press-releases-and-ad-hoc-announcements/pr-heidelberg-pharmas-lead-adc-candidate-hdp-101-granted-fast-track-designation-by-us-fda-for-the-treatment-of-multiple-myeloma
- “Fast Track,” FDA; https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track
- “HDP-101 Demonstrates Preliminary Efficacy in R/R Multiple Myeloma,” CancerNetwork; https://www.cancernetwork.com/view/hdp-101-demonstrates-preliminary-efficacy-in-r-r-multiple-myeloma
- “Study to Assess Safety of HDP-101 in Patients With Relapsed Refractory Multiple Myeloma;” https://www.clinicaltrials.gov/study/NCT04879043?a=5#contacts-and-locations
- “Antibody-drug conjugate;” https://www.cancer.gov/publications/dictionaries/cancer-terms/def/antibody-drug-conjugate
