Immunicum AB (publ) Announces Completion of HCC Phase I/II Clinical Study

Press Release

28 September 2017

Immunicum AB (publ) Announces Completion of HCC Phase I/II Clinical Study

--Topline Safety and Immune Response Data Support Continued Development of Ilixadencel in Liver Cancer--

Immunicum AB (publ; First North Premier: IMMU.ST) a biopharmaceutical company advancing a novel immune-priming cancer treatment against a range of solid tumors, today announced positive topline results from the completed Phase I/II safety and tolerability clinical trial of lead drug candidate, ilixadencel, in 18 advanced liver cancer patients. Ilixadencel was shown to be safe and well tolerated in these patients when given both as a single treatment and in combination with the current first line standard of care treatment, sorafenib. In addition, the results provide evidence of tumor-specific immune activation in the majority of patients. Based on these positive data, Immunicum continues preparation for the next stage of clinical development, in which one focus will be on establishing ilixadencel's efficacy in treating liver cancer. Ilixadencel is a cell-based, off-the-shelf therapy designed to prime the patient's immune system to recognize and destroy cancer cells, overcoming the tumor cell's ability to avoid immune system detection and attack.

Advanced liver cancer, specifically metastatic hepatocellular carcinoma (HCC) and cholangiocarcinoma (CCA, also known as bile duct cancer), is an aggressive and difficult to treat form of cancer with high mortality rates and short time to death*. Currently available treatment options including chemotherapy, molecular targeted therapy, local interventional hepatic therapy or surgery, have limited impact in patients with advanced disease. These treatments are also constrained because liver cancer patients have low tolerance for treatments with high levels of toxicity. Priming the immune system to attack a tumor holds promise of offering increased therapeutic impact with reduced side effects.

Study Design
This was a single arm, open-label, Phase I/II trial including 18 patients with advanced liver cancer, 17 patients with mHCC and 1 patient with advanced CCA**. Patients were to be treated with three separate injections of ilixadencel directly into their primary tumor (at approximately Day 1, 14 and 42) and patients were followed for 6 months after last injection. The primary objective was to investigate safety and tolerability for ilixadencel in HCC as second line therapy for patients not responding to previous treatments, or first line therapy administered with or without sorafenib. The secondary objectives included several exploratory endpoints including immunological response as measured by systemic levels of tumor specific T cells as well as initial signs of clinical activity. The study was conducted in Sweden at the Sahlgrenska University Hospital, Gothenburg. (clinicaltrials.gov ID: NCT01974661)

The final patient disposition was as follows: 7 patients were treated with ilixadencel as second line treatment, 10 patients were treated as first line treatment of which 6 patients were treated in combination with sorafenib. 14 patients received all three injections.

Results - Safety     

There were no life-threatening or fatal treatment-related adverse events (AEs). Overall, with only one exception, all treatment-related AEs were mild-to-moderate in Common Terminology Criteria for Adverse Events grading (grade 1 - 2). The most common treatment-related AEs were described as fever and/or chills and could be easily managed. The exception was one patient receiving ilixadencel and sorafenib as combination therapy, who presented with a suspected sepsis event (grade 3) that subsequently recovered. As a reference, current standard of care, such as sorafenib or regorafenib, report in the literature and in prescribing information severe (grade 3) drug-related AEs in at least one in three HCC patients treated.

"The most important results of this phase of clinical development are the safety data and these provide us with a solid basis on which to continue testing ilixadencel as a potential treatment for advanced liver cancer, an indication with desperate need of additional therapies," said Peter Suenaert, MD, PhD, Chief Medical Officer at Immunicum. "The final topline results remain in line with our previously reported data, and we are very excited to conclude the study with this outcome. We look forward to the opportunity to publish the final data analysis from the study in a peer-reviewed scientific journal."

Results - Main Exploratory Endpoints
Evidence of systemic immunological response to the treatment, as measured by an increase in tumor-specific, interferon-gamma producing CD8+ T cells in the blood, was demonstrated in 9 out of 13 evaluable patients (69%). Overall survival ranged between 1.6 - 21.4 months in the total group of 17 HCC patients at closure of study with three patients still alive. More in-depth group and patient analyses are ongoing and will be included in a future scientific journal publication.

"The excellent safety observed in this trial, as in previous studies, support our overall strategy for ilixadencel as a possible backbone in solid tumor cancer treatment that can prime the immune system while being safe and well tolerated by patients. Beyond the safety profile, the immunological data confirm our mechanism of action and support our plans to move ilixadencel forward in HCC, an important part of our clinical development strategy," said Carlos de Sousa, MD, CEO of Immunicum. "We thank our clinical development team, the clinicians, the patients and their families involved in the study for their support."

* Bruix J et al. Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial; The Lancet Published online December 5, 2016
**Inclusion approved by the Swedish regulatory authorities (MPA)

About hepatocellular carcinoma
Hepatocellular carcinoma (HCC) is one of the most common malignant tumors worldwide and the most common cause of death in people with liver cirrhosis. Progression is very rapid and prognosis is poor due to the inability to completely remove the tumor through surgery in most cases. Malignant transformation of liver cells may occur as a consequence of various origins, such as chronic viral hepatitis, alcohol and metabolic disorders.

About ilixadencel
Ilixadencel cell therapy product (formerly known as INTUVAX®) is an off-the-shelf cancer immune primer, developed for the treatment of solid tumors. Its active ingredient is activated allogeneic dendritic cells, derived from healthy blood donors. Intratumoral injection of these cells is expected to lead to an inflammatory response which in turn leads to tumor-specific activation of the patient's cytotoxic T-lymphocytes.

About Phase I/II clinical trials
A Phase I clinical study is the first time a new treatment is administered to humans. The purpose of the first clinical trials is to determine whether the study subjects tolerate the drug and whether it behaves in the body in the way as indicated based on animal studies and other pre-clinical research. In cancer drug development, the study subjects are most often cancer patients whose disease is in an advanced stage and for whom not many other therapeutic options exist. The product is administered under strict medical conditions and the patients are followed up intensely.

The information is such information that Immunicum is obliged to make public pursuant to the EU Market Abuse Regulation. The information was released for public disclosure through the agency of the company's contact person on September 28, 2017 at 8:00 am CET.

For more information, please contact:

Carlos de Sousa, CEO, Immunicum
Telephone: +46 (0) 31 41 50 52
E-mail: info@immunicum.com

Investor relations, Sweden

Helena Stångberg
Hallvarsson & Halvarsson
Telephone: + 46 709 71 12 53
E-mail: helena.stangberg@halvarsson.se

Investor and Media Relations EU/US 

MacDougall Biomedical Communications
Gretchen Schweitzer or Stephanie May
Telephone: +49 89 2424 3494 or + 49 175 571 1562
E-mail: gschweitzer@macbiocom.com or smay@macbiocom.com

The Company's Certified Adviser is Redeye AB
Telephone: +46 (0) 8 545 013 31
www.redeye.se

About IMMUNICUM AB (PUBL)

Immunicum is establishing a unique immuno-oncology approach through the development of allogeneic, off-the-shelf cell-based therapies. Our goal is to improve survival outcomes and quality of life by priming the patient's own immune system to fight cancer. The company's lead product ilixadencel, consisting of pro-inflammatory allogeneic dendritic cells, has the potential to become a backbone component of modern cancer combination treatments in a variety of solid tumor indications. Founded and based in Sweden, Immunicum is publicly traded on the Nasdaq First North Premier. www.immunicum.com