On December 30, 2019 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported that the first patient has been dosed in a Phase 1/2 clinical trial (NCT04180215) of HB-201, an immunotherapy for the treatment of Human Papillomavirus 16-positive (HPV16+) cancers. This trial is HOOKIPA’s first clinical trial in immuno-oncology (Press release, Hookipa Pharma, DEC 30, 2019, View Source [SID1234553433]).
HB-201 is a TheraT platform-based vector (replication attenuated) from the arenavirus family expressing a non-oncogenic but highly antigenic E6/E7 fusion protein from HPV16. In preclinical studies, HB-201 was observed to be highly immunogenic, resulting in a robust CD8+ T cell response as compared to the levels induced by other approaches including adoptive cell therapies. In addition to strong immunogenicity, HOOKIPA observed robust anti-tumor activity in mouse models. HOOKIPA believes that HB-201 has the potential to provide therapeutic benefit to patients across the HPV16+ cancer setting.
Joern Aldag, Chief Executive Officer at HOOKIPA, commented: "We are excited to begin first-in-human testing with HB-201, our first clinical trial in immuno-oncology. Translating our promising preclinical data to cancer patients is an important milestone. We believe HOOKIPA’s approach can supercharge the natural defence mechanisms by inducing strong T cell responses to the benefit of patients affected by cancer and infectious diseases."
About the HB-201 Clinical Trial
The HB-201 Phase 1/2 trial is an open-label dose-escalation and dose-expansion trial in 100 patients with treatment-refractory HPV16+ cancers. It is designed to evaluate the safety and tolerability and preliminary efficacy of HB-201 as monotherapy and in combination with an immune checkpoint inhibitor.
For Phase 1 dose escalation, the patient population will be divided into two groups of 20 patients, each: the first group will include patients with progressing HPV16+ tumors who will receive monthly intravenous (IV) administration of HB-201; the second group will include patients with progressing HPV16+ tumors and an accessible tumor site who will receive one intratumoral (IT) administration of HB-201 followed by monthly IV administration of HB-201.
The primary endpoint of the Phase 1 portion of this trial will be to evaluate safety and tolerability to determine the recommended dose for the Phase 2 portion. Secondary endpoints will evaluate anti-tumor activity, as defined by RECIST (Response Evaluation Criteria In Solid Tumors) 1.1, and immunogenicity. The Phase 2 portion of the trial will also investigate the efficacy of HB-201 alone and in combination with a PD-1 inhibitor. HOOKIPA expects to provide interim safety, dose escalation, and efficacy data from HB-201 in late 2020 or early 2021. These data will be supplemented by a series of translational data sets designed to demonstrate the mechanism of action.
About Human Papillomavirus
Human Papillomavirus (HPV) is estimated to cause about 5% of the worldwide burden of cancer including approximately 99% of cervical cancers, 25% to 60% of head and neck cancers, 70% of vaginal cancers and 88% of anal cancers1. The majority of these cancers are caused by the HPV serotype 16. Most infections with HPV are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to becoming cancerous.