Herantis Pharma has completed patient dosing in its Phase 2 trial evaluating the investigational gene therapy Lymfactin for secondary lymphedema — a common complication of breast cancer treatment caused by the extensive removal of lymph nodes in the armpit region.
The 39 patients included in the multicenter AdeLE trial (NCT03658967) — who were randomly assigned to receive Lymfactin or a placebo, given in combination with lymph node transplant surgery — will now be followed for one year to determine the treatment’s safety and efficacy. Top-line results are expected in the first quarter of 2021.
“The rapid enrolment completion demonstrates not only that there is a significant need for novel treatment options in lymphedema, but it is also an important milestone for our Lymfactin program as we are advancing into late-stage clinical development,” Pekka Simula, CEO of Herantis Pharma, said in a press release.
“We are truly grateful to the patients who participated in the AdeLE study, the supporting physicians and staff at the study sites, and to the international patient organization LE&RN, which fights to raise awareness for lymphedema as a significant unmet clinical need,” Simula said.
Approximately 20% of breast cancer patients who undergo lymph node removal as a part of cancer treatment develop secondary lymphedema, a condition characterized by the accumulation of fluid (lymph) that causes swelling, mostly in the arms or legs. Secondary lymphedema currently is treated with compression garments, special massages, and exercises — but these are only relief measures that fail to address the cause of lymphedema.
Lymfactin is an investigational gene therapy designed to repair the damage to the lymphatic system caused by cancer surgery and other treatments. The therapy delivers a natural human gene, called vascular endothelial growth factor C or VEGF-C, which is necessary for the growth of lymphatic vessels.
It is designed to be administered during a lymph node transplant surgery, in which lymph nodes are removed from the patient’s abdominal wall or groin region and implanted into the axilla of the affected arm. The axilla, or armpit region, is the area under the shoulder joint where the arm connects to the shoulder, and is home to lymph nodes, veins and arteries. Lymfactin is injected directly into the flap of tissue containing the lymph nodes, increasing lymphatic vessel recovery and the overall health of the lymphatic system.
In preclinical studies, Lymfactin was shown to promote the growth of new lymphatic vessels in damaged areas of the lymphatic system.
One-year data from an ongoing, Finland-based, open-label Phase 1 trial (NCT02994771), which is evaluating Lymfactin in 15 patients with breast cancer-associated lymphedema, showed that the treatment was safe and well-tolerated, with no severe adverse events, and improved quality of life.
Those positive results prompted Herantis to launch the AdeLE Phase 2 trial, designed to compare the effects of Lymfactin to those of a placebo in lymphedema patients undergoing lymph node transplant surgery.
AdeLE, ongoing in Finland and Sweden, is evaluating the treatment’s efficacy, as measured by three criteria. Those are the changes in the volume of the affected arm compared with the unaffected arm, the participants’ quality of life based on the Lymphedema Quality of Life Inventory, and the lymph flow of the affected arm.
Secondary measures include safety, onset of malignancies in the chest or abdomen, and whether the immune system reacts to Lymfactin. The anatomy and functionality of the lymphatic vessels also may be assessed with magnetic resonance imaging (MRI).
“The robust Phase 1 data, confirming safety and tolerability of Lymfactin, favorably positioned us to quickly move into Phase 2 clinical evaluation, which is an important achievement for this program and the company,” Simula said in a press release announcing the trial. “Secondary lymphedema remains a disease with a noteworthy and underserved patient population and as a locally administered single dose therapy, Lymfactin offers great potential to address this significant gap.”
“At the moment there is no curative treatment for lymphedema,” Anne Saarikko, MD, PhD, the Phase 1 study’s principal investigator, said at the start of the AdeLE trial. “Lymfactin stands out as a unique, easy-to-administer, and hopefully the first curative treatment option in a disease area where current therapies only relieve symptoms.”
The one-year follow-up period will be completed in December.
“Based on the cumulated safety data and assessments of the trial’s independent Data Safety Monitoring Board, we are encouraged by the established safety profile of Lymfactin,” Simula said in announcing the trial’s dosing completion. “We look forward to the efficacy read-outs that could bring new hope to lymphedema patients all around the world.”