Full Press Release Details
HOOKIPA Pharma Announces Positive Clinical Data
to be Presented at the American Society for Clinical Oncology 2024 Annual Meeting
YORK and VIENNA, May 23, 2024 - HOOKIPA Pharma Inc. (NASDAQ: HOOK, HOOKIPA'), a company developing a
new class of immunotherapeutics based on its proprietary arenavirus platform, today announced positive updated results from its Phase
1/2 clinical trial of HB-200 for the treatment of human papillomavirus 16 positive (HPV16+) head and neck cancers. The data were published
in the Company's abstract for the ASCO 2024 Annual Meeting and support the Company's pivotal Phase 2/3 trial design for HB-200
in combination with pembrolizumab in the first line setting.
The abstract reported
data as of January 12, 2024, and included 42 patients treated with HB-200 plus pembrolizumab. The treatment was generally well tolerated
with a low rate of treatment-related discontinuation and no treatment-related deaths.
Among a subpopulation of 17 evaluable patients
with CPS of 20 or higher, the updated data showed confirmed ORR of 53 percent, CR rate of 18 percent, and DCR of 82 percent. This subpopulation
is representative of patients eligible for the Company's pivotal Phase 2/3 trial, which will begin enrolling patients in the fourth
Additional data will be presented in the Head
and Neck Oral Abstract Session at the ASCO 2024 Annual Meeting on June 4, at 11:09 a.m. CDT. During the presentation, preliminary
progression-free survival and overall survival data will be shared for the first time.
are happy to provide an update on our clinical data and showcase the meaningful outcomes we are helping to drive for patients,"
said Joern Aldag, Chief Executive Officer of HOOKIPA. "The data exhibit strong evidence that has helped inform our pivotal Phase
2/3 trial design, which will begin enrolling patients later this year. This update gives us conviction that we are on the right path to
achieve our goals and help provide a new targeted therapeutic option for patients battling HPV16+ head and neck cancer."
in combination with pembrolizumab:
abstract presented data as of January 12, 2024, and included 42 first line patients with HPV16+, PD-L1 positive, recurrent or metastatic
head and neck squamous cell carcinoma.
The updated data continue to demonstrate
a favorable safety profile of HB-200 in combination with pembrolizumab and promising clinical activity
as a first line treatment. Median follow-up time was 5.6 months.
+ pembrolizumab were generally well tolerated. Grade 3 treatment-related adverse events (TRAEs) were reported in 6 (14%) patients,
serious TRAEs were reported in 3 (7%) patients, and TRAEs leading to treatment discontinuation were reported in 2 (5%) patients. No treatment-related
deaths were reported.
35 evaluable patients-those with 1 post-baseline tumor response assessment-3 confirmed complete responses, 9 confirmed
partial responses, and 3 unconfirmed partial responses were observed. Notably, among patients with PD-L1 CPS 20 (N=17), ORR, based
on confirmed responses, was 53%, CR rate was 18%, and DCR was 82%.
Abstracts at ASCO 2024 Annual Meeting:
HB-200 plus chemotherapy (neoadjuvant setting):
In addition, data from an Investigator Initiated
Trial (IIT), led by Dr. Ari Rosenberg of the University of Chicago Department of Medicine, will be presented on June 3, in the
Head and Neck Rapid Oral Abstract Session. The study concluded that neoadjuvant HB-200 plus chemotherapy for the treatment of patients
with non-metastatic HPV16+ oropharyngeal cancers (OPC) is safe and feasible, with early efficacy signal in this setting warranting further
Twenty-one patients with HPV16+ OPC were enrolled
and treated across multiple cohorts and dose levels. All patients completed neoadjuvant HB-200/chemotherapy and response-stratified locoregional
treatment. Deep responses following HB-200/chemotherapy were observed in 17/21 (81%) patients, and in 14/15 (93%) patients treated with
higher dose levels 1 or 2. All three patients who underwent transoral robotic surgery (TORS) had no viable tumor at time of surgery. Two
patients (9%) had persistent disease following chemoradiotherapy and underwent salvage surgery with no evidence of disease at last follow-up.
ctHPV-DNA and HPV16-specific T-cell response data will be presented at the meeting.
Enrollment to the subsequent randomized phase
II part is ongoing. Details of the rapid oral presentation are included below.
HB-700 preclinical data:
HB-700 is an investigational arenaviral immunotherapy
designed to treat KRAS-mutated lung, colorectal, pancreatic and other cancers. HB-700 is a replicating 2-vector therapy that targets the
most common KRAS mutations (G12D, G12V, G12R, G12C and G13D) and has the potential to benefit a broader patient population than single
mutation inhibitors.
A transgene cassette consisting of peptide stretches
including KRAS mutations G12D, G12V, G12C, G12R and G13D was generated by in silico aided antigen design. KRAS mutation specific CD8+
T cell expansion was evaluated in HLA transgenic mice treated with HB-700 and functionality of induced CD8+ T cells was evaluated by assessing
CD8+ T cell mediated killing of mutant KRAS peptide loaded target cells in vivo.
All treatment regimens
were well tolerated, and no mortalities or major adverse events were observed. The results indicate efficient induction of KRAS mutation
specific T cell responses in HLA transgenic mice. Expanded CD8+ T cells were capable of killing cells loaded with KRAS mutation specific
peptides in vivo indicating functionality of the induced T cell responses. No specific cytotoxicity towards target cells pulsed with KRAS
wild type peptides was observed in any of the groups. HOOKIPA's HB-700 investigational product candidate differs from KRAS
inhibitors and has a wide range of combinability options including with small-molecule inhibitors.
Based on these results, initiation of a Phase I study for the treatment of KRAS mutated cancers is planned.
Abstract details: ASCO 2024 Annual Meeting
arenavirus-based immunotherapy plus pembrolizumab as first-line treatment of patients with recurrent/metastatic HPV16-positive head and
neck cancer: Updated results
Presenter: Dr. Alan L. Ho, Head and
Neck Oncologist at Memorial Sloan Kettering Cancer Center and a trial investigator
Abstract Type: Oral abstract
Name: Head and Neck Cancer
Date and Time: June 4, 2024; 9:45 AM-12:45 PM CDT
Abstract Number: 6005
Investigator Initiated Trial:
HPV16-specific arenavirus-based immunotherapy HB-200 plus chemotherapy followed by response-stratified de-intensification in HPV16+ oropharyngeal
Presenter: Dr. Ari Rosenberg, Principal Investigator, TARGET-HPV Trial,
University of Chicago Medicine
Abstract Type: Rapid oral abstract
Name: Head and Neck Cancer
Date and Time: June 3, 2024; 8:00 AM-9:30 AM CDT
Abstract Number: 6017
of an arenavirus-based immunotherapy for treatment of KRAS mutant cancer
Abstract Type: Abstract only
Abstract Number: e14672
HB-200 is HOOKIPA's lead oncology candidate
engineered with the company's proprietary replicating arenaviral vector platform. It comprises two single-vector compounds with
arenaviral backbones based on lymphocytic choriomeningitis virus (LCMV) and pichinde virus (PICV). Both express the same transgene encoding
an E7E6 fusion protein derived from HPV16. HB-200 is an alternating 2-vector immunotherapy designed to further focus the immune response
against the encoded antigen.
HB-200 in combination with pembrolizumab received
Fast Track Designation from the U.S. Food and Drug Administration and PRIME designation from the European Medicines Agency for the treatment
of first-line HPV16+ recurrent/metastatic oropharyngeal squamous cell carcinoma. These designations are supported by preliminary clinical
evidence from the Phase 1/2, open-label, clinical trial (NCT04180215) evaluating safety, T cell response, and efficacy based on objective
response rate (ORR) and disease control rate (DCR) as defined by RECIST 1.1. and iRECIST.
HB-700 is an investigational arenaviral immunotherapy
designed to treat KRAS-mutated lung, colorectal, pancreatic and other cancers. HB-700 is a replicating 2-vector therapy that targets the
most common KRAS mutations (G12D, G12V, G12R, G12C and G13D) and has the potential to benefit a broader patient population than single
mutation inhibitors.
Pharma Inc. (NASDAQ: HOOK) is a clinical-stage biopharmaceutical company focused on developing novel immunotherapies, based on its proprietary
arenavirus platform, which are designed to mobilize and amplify targeted T cells and thereby fight or prevent serious disease. HOOKIPA's
replicating and non-replicating technologies are engineered to induce robust and durable antigen-specific CD8+ T cell responses and
pathogen-neutralizing antibodies. HOOKIPA's pipeline includes its wholly owned investigational arenaviral immunotherapies targeting