~ 96 Percent of High-Risk Melanoma Patients Were Alive Three Years After Completing the Vaccine Series Compared to 77 Percent of those Treated with Placebo ~
~ 52 Percent of Patients Treated with the Vaccine Were Disease-Free at Three Years Compared to 27 Percent in the Placebo Arm ~
~ New Analysis Reveals Optimized Vaccine Formulation Results in Improved Clinical Outcomes ~
~ Treatment with Vaccine in Combination with Checkpoint Inhibitors Doubled Disease-Free Survival Rate Compared to Checkpoint Inhibitors Alone ~
AUSTIN, TX, August 5, 2020 – Elios Therapeutics, a biopharmaceutical company developing innovative personalized therapeutic cancer vaccines, today announced final data from a prospective, randomized, double-blind, placebo-controlled Phase IIb clinical trial evaluating adjuvant use of its personalized tumor lysate, particle-loaded, dendritic cell (TLPLDC) vaccine in patients with Stage III or Stage IV melanoma at high risk of recurrence following complete surgical resection.
“We now have long-term data demonstrating that use of the TLPLDC vaccine for the adjuvant treatment of high-risk melanoma correlates with a 93 percent increase in patients alive at three years without their disease returning. This trial also significantly improves our understanding of the optimal method of vaccine production,” said Buddy Long, chief executive officer of Elios Therapeutics. “These new data, combined with the doubled rate of disease-free survival among patients treated with the vaccine and standard of care checkpoint inhibitors, further strengthen our confidence that the personalized TLPLDC vaccine provides a clinically meaningful benefit for people with high-risk melanoma. We look forward to advancing this vaccine with a registrational Phase III trial that will move us one step closer to bringing this important treatment to patients as soon as possible.”
The TLPLDC vaccine is a personalized treatment that is created using a patient’s own blood and tumor cells. Samples are collected at resection, frozen, and sent to the lab where they are used to create autologous tumor lysate, which is loaded into yeast cell wall particles. This combination is then introduced to the patient’s dendritic cells, leading to the creation of the final TLPLDC vaccine. The time from resection to injection of the vaccine takes approximately three weeks.
The vaccine has been studied in a large randomized Phase IIb trial, and the newly reported data is from the pre-specified 36-month disease-free survival (DFS) and overall survival (OS) assessment by vaccine formulation, stage, and checkpoint inhibition. The analysis included all randomized patients in an intent-to-treat (ITT) analysis. Two versions of the vaccine, one produced by isolating dendritic cells (DCs) from 120 mL of blood (vaccine-A) and one with DCs isolated after a single injection of filgrastim followed by 50-70 mL of blood (vaccine-B), were tested in 144 participants who were randomized to receive either version of the vaccine or placebo to prevent recurrence.
A key finding showed that treatment with vaccine-B resulted in clinical outcomes similar to placebo. Producing the vaccine with filgrastim was intended to increase white blood cell and dendritic cell counts, requiring less blood to be drawn from patients to create the vaccine. While the use of filgrastim increased DC production, it takes only 72-hours to create the vaccine which was not enough time for the DCs to mature, rendering vaccine-B ineffective.
Importantly, vaccine-A, when compared to vaccine-B and placebo, resulted in a statistically significant improvement in 36-month DFS (51.8% vs. 23.4% vs 27.1%, respectively; p=0.027) and OS (92.9% vs. 62.8% vs 70.3%, respectively; p=0.022) in the ITT population.
Furthermore, the DFS improvement with vaccine-A was seen across both Stage III (49.7% vs. 29.4%; p=0.066) and IV (68.6% vs. 9.4%; p=0.0582) patients. Importantly, the addition of vaccine-A to current standard of care checkpoint inhibitors led to a statistically significant increase in 36-month DFS in the ITT population compared to treatment with checkpoint inhibitors alone (48.5% vs. 24.1%; p=0.039). As previously reported, treatment with the vaccine was well-tolerated with 34.7 percent of patients experiencing a treatment-related adverse event, and >90% being grades 1 or 2.
“To demonstrate a long-term survival benefit with low toxicity in a therapeutic is what we hope for in every clinical trial. Achieving this with an aggressive disease like melanoma offers great promise for patients,” said Mark B. Faries, M.D., co-director of the Melanoma Program and head of Surgical Oncology at Cedars-Sinai at The Angeles Clinic and Research Institute, and principal investigator of the study. “With data showing a two-fold increase in disease-free survival with the vaccine alone and in combination with checkpoint inhibitors, we hope to one day change the narrative for people with melanoma – turning this disease into a chronic condition that can be treated and managed over time.”
Melanoma is more likely to grow and spread than other types of skin cancer. When diagnosed and treated at an early stage, melanoma has a high cure rate, however patients with later stages of the disease carry a high risk for melanoma recurrence because some melanoma cells can remain in the body, even after surgery. In the U.S, the incidence of melanoma has increased over the past decades, with 91,270 estimated new cases and 9,320 related deaths in 2018^1.
About the Phase IIb TLPLDC Study
This Phase IIb study is a prospective, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of the TLPLDC (tumor lysate, particle-loaded, dendritic cell) vaccine in patients with resected Stage III and IV melanoma. The primary endpoint of the trial is two-year disease-free survival (DFS), and the secondary endpoint is three-year DFS and overall survival (OS).
In the study, 144 participants were randomized to receive either the vaccine or placebo to prevent recurrence. TLPLDC or placebo vaccines were initiated within three months of completion of standard of care (SoC) therapies and were given at 0, 1, 2, 6, 12, and 18 months. The protocol was amended to allow concurrent checkpoint inhibitor therapy once approved for the adjuvant setting. Study participants were followed for recurrence per SoC. The primary efficacy analysis was performed on the intent-to-treat (ITT) and the per treatment (PT) populations as co-primary analyses given the high early recurrence rate often seen in patients with advanced melanoma. Secondary endpoints include 36-month DFS and overall survival (OS) which will be compared between the vaccinated and control groups as well as by vaccine formulation.
The TLPLDC (tumor lysate, particle-loaded, dendritic cell) vaccine is a unique type of immunotherapy, both in how it is made and how it is delivered. The vaccine is personalized, meaning it is made from a patient’s tumor and blood. Every patient’s tumor has a unique antigenic profile unlike any other, and dendritic cells found in the blood are the most potent antigen-presenting cells in the body. Once TLPLDC is administered, it delivers the patient’s complete repertoire of tumor antigens to the immune system, creating a dual innate and adaptive immune response, activating fighter T cells, and triggering the immune system to recognize, and seek out and destroy any cells containing the antigens and specific mutations from their tumor.
Historically, autologous cancer vaccines have been rather onerous to develop, sometimes taking months between the tumor biopsy and administration. Elios has simplified the process so the time from resection to injection is approximately two weeks. This makes the vaccine highly feasible and will ultimately be easy for community and academic oncologists to adopt into their practices.
The TLPLDC vaccine is currently being studied as a monotherapy and in combination with standard-of-care checkpoint inhibitor therapies in a Phase IIb clinical trial for the treatment of late-stage melanoma at leading academic cancer centers in the United States.
About Elios Therapeutics, LLC
Elios Therapeutics, LLC, a wholly owned subsidiary of Perseus Holdings, LLC., is a biopharmaceutical company developing a portfolio of innovative personalized therapeutic cancer vaccines targeting unmet medical needs across a broad range of tumor types. Elios’ lead therapeutic candidate is the TLPLDC (tumor lysate, particle-loaded, dendritic cell) vaccine, a personalized therapeutic cancer vaccine that uses a proprietary particle delivery system to stimulate the body’s immune system to recognize cancer cells as foreign pathogens and fight a patient’s specific cancer. For more information, please visit www.eliostherapeutics.com.
This document contains forward‐looking statements relating to the Company’s strategy, objectives, business development plans and financial position. All statements other than statements of historical facts included in this document, including, without limitation, statements regarding the Company’s future financial position, strategy, anticipated investments, costs and results, status and results of clinical trials, size of patient population, plans, outcomes of product development efforts, and objectives of management for future operations, may be deemed to be forward‐looking statements. You can identify forward-looking statements by words such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would,” or the negative of those terms, and similar expressions that convey uncertainty or future events or outcomes. These forward‐looking statements involve known and unknown risks, uncertainties, and other factors that may cause the Company’s actual results, performance, or achievements or industry results to be materially different from those contemplated, projected, forecasted, estimated or budgeted, whether expressed or implied, by these forward‐looking statements. Given these risks and uncertainties, investors should not place undue reliance on forward‐looking statements as a prediction of actual results. None of these forward‐looking statements constitutes a guarantee of the future occurrence of such events or of actual results. These statements are based on data, assumptions, and estimates that the Company believes are reasonable. The forward‐looking statements contained in this document are made only as of the date hereof. Except as otherwise required by law, the Company expressly disclaims any obligation or undertaking to release publicly any updates of any forward-looking statements contained in this document to reflect any change in its actual results, assumptions, expectations or any change in events, factors, conditions, or circumstances on which any forward‐looking statement contained in this document is based.
Media Relations Vikki Christian
Elixir Health Public Relations
+1 (424) 610-1241