Full Press Release Details
HOOKIPA Interim Phase 1 Monotherapy
Data of HB-201 for the Treatment of Advanced HPV16+ Cancers Shows Promising Anti-Tumor Activity and Favorable Tolerability
York, US and Vienna, Austria, December 7, 2020 - HOOKIPA Pharma Inc. (NASDAQ: HOOK, HOOKIPA'), a company
developing a new class of immunotherapeutics based on its proprietary arenavirus platform, today announced positive interim Phase
1 data on HB-201, its replicating monotherapy for the treatment of HPV16+ cancers. The results are from the initial
dose escalation cohorts of an ongoing Phase 1/2 clinical trial (NCT04180215) evaluating HB- 201 as therapy for patients with
advanced HPV16+ metastatic cancers. HOOKIPA will host a conference call and live audio webcast today at 8:30am EST.
These interim data support proof of concept
for HB-201 monotherapy as a new immunotherapy for a difficult-to-treat patient population with multiple prior treatment failures.
As of December 4, 2020, 22 patients have been enrolled in the first two cohorts, of which 15 were eligible for evaluation. Among
the 15 evaluable patients, 11 patients had relapsed/refractory metastatic squamous cell head and neck cancer (HNSCC), all of whom
had progressed on prior therapy with a PD1 inhibitor. As per RECIST1.1, in patients with third-line or later HNSCC, HB-201 demonstrated
an unconfirmed response rate of 18% (one unconfirmed complete responder and one unconfirmed partial responder) and a 73% disease
control rate (six stable disease patients, in addition to the two unconfirmed responses referenced above).
Median progression-free survival (mPFS)
is currently measured at 72 days and is ongoing. Although not demonstrated in a head-to-head trial, these HB-201 results, in more
heavily treated patients who progressed on a PD1 inhibitor, compare favorably to the benchmark data of a 13% overall response rate
and a 60-day mPFS1 for nivolumab
in second-line PD1 inhibitor na ve HNSCC patients, based on data published from the third-party registrational study.
Encouraging efficacy signals were also
seen in the more heterogeneous group of all 15 evaluable patients with HPV16+ cancers treated in this trial, comprised
of the 11 HNSCC patients summarized above and four other patients with HPV16+ cervical, anal, or vaginal tumors. In
these 15 patients, HB-201 demonstrated an unconfirmed response rate of 13%, a disease control rate of 67%, and a median PFS
that is also ongoing and currently measured at 72 days.
Ferris R et al. Nivolumab for recurrent squamous-cell carcinoma of the head and neck. N Engl J Med. 2016; 375:1856-1867.
"We are thrilled by these preliminary
HB-201 data, as they show the potential of our arenavirus platform in oncology and represent future possible therapeutic options
for patients with HPV16+ cancers," said Joern Aldag, Chief Executive Officer of HOOKIPA. "The early response
with our single-vector HB-201 therapy highlights the potential of our replicating technology, especially as we explore alternating
two-vector therapy with HB-201/HB-202, and a future combination with a PD-1 inhibitor, both of which we hope will deliver even
Of the 22 patients enrolled on the trial
as of December 4th, preliminary safety data show that HB-201 has been well tolerated. Treatment-related adverse events
were reported by 41% of participants. Almost all reported events were Grade 1 and 2 and included fatigue, fever, decreased appetite,
constipation, nausea, and itching. Only one serious adverse event deemed related to HB-201, Grade 3 fatigue leading to hospitalization,
has been reported to- date. The rate of adverse events was consistent regardless of administration route.
"There remains a considerable unmet
need in the treatment of HPV16+ cancers, particularly those in head and neck, and these preliminary data on HB-201 as
a monotherapy are encouraging," said Alan L. Ho, MD, PhD, a medical oncologist at Memorial Sloan Kettering Cancer Center
and an investigator on the trial.
This Phase 1/2 clinical trial is an open-label
dose-escalation and dose-expansion trial in individuals with treatment-refractory HPV16+ cancers. The primary endpoint
of the Phase 1 trial is a recommended Phase 2 dose based on safety and tolerability. Secondary endpoints include anti-tumor activity
as defined by RECIST 1.1, immunogenicity, safety, and tolerability.
The trial is designed to evaluate different
dose levels of HB-201 as a single-vector therapy, as an alternating two-vector therapy together with HB-202, and in combination
with a PD-1 inhibitor. Dosing frequencies of every three weeks and every two weeks are being explored during dose escalation.
Since the trial opened in December 2019,
22 patients with metastatic HPV16+ tumors have been enrolled in the HB-201 monotherapy segment: 17 with squamous cell
head and neck tumors, two with cervical, one with nasopharyngeal, one with anal, and one with vaginal. Patients had received at
least two prior therapies, and most patients progressed on a PD-1 inhibitor, a platinum-containing regimen or both. Enrollment
is ongoing and HOOKIPA expects to share additional interim clinical data from the HB-201/HB-202 alternating two-vector therapy
segment in mid-2021.
HB-201 and HB-202 are engineered using
HOOKIPA's replicating arenaviral vector platform. They are designed to use different arenavirus backbones (LCMV
for HB- 201 and PICV for HB-202), while expressing the same antigen, an E7/E6 fusion protein derived from HPV16. In
pre-clinical studies, alternating administration of HB-202 and HB-201 resulted in a ten-fold increase in immune response and better
disease control than either compound alone.
HOOKIPA will host a conference call and
live audio webcast today at 8:30am EST to discuss the HB-201 monotherapy data from the interim analysis of the Phase 1 trial.
To access the conference call, please dial +1 877 870 9135 (from the US) or +44 2071 928338 (international) and refer to conference
ID 9747865. The webcast and the presentation will be available within the Investors & Media section of HOOKIPA's website
at https://ir.hookipapharma.com/events. An archived replay will be accessible for 30 days following the event.
Human Papillomavirus
Human Papillomavirus,
or HPV, is estimated to cause about 5 percent of the worldwide burden of cancers. This includes approximately 99 percent of cases
in cervical, up to 60 percent of head and neck, 70 percent of vaginal and 88 percent of anal cancers.
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
uptake can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn
predisposes these cells to become cancerous.
Pharma Inc. (NASDAQ: HOOK) is a clinical stage biopharmaceutical company developing a new class of immunotherapeutics based on
its proprietary arenavirus platform that reprograms the body's immune system. HOOKIPA's proprietary arenavirus-based
technologies, non-replicating (VaxWave ) and replicating (TheraT ), are designed induce
robust antigen-specific CD8+ T cells and pathogen-neutralizing antibodies. HOOKIPA's viral vectors target antigen presenting
cells in vivo to activate the immune system. Both technologies enable repeat administration to augment and refresh immune responses.
As a monotherapy, HOOKIPA's replicating arenavirus technology has the potential to induce CD8+ T cell response
levels previously not achieved by other immuno-therapy approaches.
non-replicating prophylactic cytomegalovirus (CMV) vaccine candidate is currently in a Phase 2 clinical trial for patients
awaiting kidney transplantation. To expand its infectious disease portfolio, HOOKIPA entered into a collaboration and licensing
agreement with Gilead Sciences, Inc. to research arenavirus-based functional cures for HIV and chronic Hepatitis B infections.
addition, HOOKIPA is building a proprietary immuno-oncology pipeline by targeting virally mediated cancer antigens, self-antigens,
and next-generation antigens. The lead replicating arenavirus oncology product candidates, HB-201 and HB-202, are in development
for the treatment of Human Papilloma Virus 16-positive cancers in a Phase 1/2 clinical trial.
out more about HOOKIPA online at www.hookipapharma.com.
HOOKIPA Forward Looking Statements
statements set forth in this press release constitute "forward-looking" statements within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended. Forward-looking statements can be identified by terms such as "believes,"
"expects," "plans," "potential," "would" or similar expressions and the negative
of those terms. Such forward-looking statements involve substantial risks and uncertainties that could cause HOOKIPA's research
and clinical development programs, future results, performance or achievements to differ significantly from those expressed or
implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the
drug development process, including HOOKIPA's programs' early stage of development, the process of designing and conducting
preclinical and clinical trials, the regulatory approval processes, the timing of regulatory filings, the challenges associated
with manufacturing drug products, HOOKIPA's ability to successfully establish, protect and defend its intellectual property,
risks relating to business interruptions resulting from the coronavirus (COVID-19) disease outbreak or similar public health crises,
the impact of COVID-19 on the enrollment of patients and timing of clinical results for HB-101 and other programs, and other matters
that could affect the sufficiency of existing cash to fund operations and HOOKIPA's ability to achieve the milestones under
the agreement with Gilead. HOOKIPA undertakes no obligation to update or revise any forward-looking statements. For a further
description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking