- Patient-friendly alternative to
continuous IV administration – proven safety and efficacy
- Targeting orphan, late-stage heart
failure indication with high unmet medical needs
Liège, Belgium – 8 October 2021 –
Hyloris Pharmaceuticals SA (Euronext Brussels:
HYL), a specialty biopharma company committed to
addressing unmet medical needs through reinventing existing
medications, today announces that it has acquired the global rights
from the Baker Heart and Diabetes Institute (“the Baker
Institute”), Melbourne, Australia, to CRD-102 (and related
intellectual property), a novel, clinical-stage, extended-release
Milrinone capsule in late-stage heart failure (HF) patients with an
implanted left ventricular assist device (LVAD1) who have developed
right HF. CRD-102 is the second innovative product candidate that
has been added to the portfolio this year, further delivering on
Hyloris’ promise to expand the R&D pipeline with 4 new product
candidates in 2021.
Milrinone is a positive inotrope, a group of
heart failure drugs that strengthen the heart’s contractions so it
can pump more blood with fewer heartbeats. Milrinone IV is
currently approved for use as an intermittent or continuous
infusion for treatment of up to 48 hours for acute decompensated
heart failure. Longer term off-label use exceeding 48 hours of
treatment duration has also been reported, requiring nursing
support and limiting the patient’s quality of life by IV
administration. Continuous IV use however exposes patients to an
increased risk of common IV equipment complications, such as
central line-associated bloodstream infections. Milrinone
IV was approved in 1987 and in 2020 more than 12 million vials and
infusion bags were sold, of which over 2 million in the U.S.
alone.
Prof. Dr.
David Kaye, Heart Failure Specialist, Head of the Heart
Failure Research Group at the Baker Institute and Director of the
Department of Cardiology at the Alfred Hospital, Melbourne,
Australia, commented: “Patients with end-stage HF, a
serious, debilitating disease, experience extreme difficulties
breathing, have a very poor quality of life and suffer from
multiple, severe co-morbidities. The current standard of care is
predominantly palliative and despite recent advantages with new
heart failure therapies, the effectiveness of these is most
pronounced in mild to moderate heart failure. CRD-102 has the
potential to address the current unmet needs of late-stage LVAD
patients by offering a patient-friendly and convenient oral
treatment option as compared to repeated, continuous IV infusions,
which is the current gold standard in many countries. Its efficacy
and safety have been established in earlier Phase 1 and Phase 2
studies2, and if these results can be repeated in pivotal studies,
CRD-102 would have important potential in this underserved patient
population.”
Stijn Van Rompay, Chief Executive
Officer of Hyloris, added: “We are very pleased to partner
with the Baker Institute and believe that CRD-102 has the potential
to dramatically change the lives of patients living with late-stage
HF, more notably, patients with an LVAD who have developed right
HF, a severe orphan HF indication. Earlier studies have
demonstrated that treatment with CRD-102 resulted in improved
quality of life and functional status of late-stage HF patients,
and CRD-102 was well-tolerated with no increased incidences of
arrhythmias being reported. CRD-102 perfectly fits within our
portfolio of value-added cardiovascular products and our strategy
for self-commercialisation in the U.S. We are now preparing the
next stages of development and anticipate the start of the pivotal
clinical study in LVAD patients with right HF towards end 2022 or
early 2023.”
Under the terms of the agreement, Hyloris will
be responsible for the further development, manufacturing,
regulatory affairs, and commercialisation of CRD-102. In return,
Hyloris will pay an upfront payment of $50,000 to the Baker
Institute plus sales-based milestone payments and tiered, single to
double digit net profit shares in markets where Hyloris intends to
self-commercialise CRD-102. In markets where Hyloris intends to
seek commercial partnerships, the Baker Institute is eligible to
receive tiered, single to double digit net profit shares on net
sub-license income.
About CRD-102 and earlier clinical
studies in Stage IV HF patients3CRD-102
is a novel, patented, extended-release Milrinone capsule that has
been developed for patient-friendly, twice a day convenient oral
dosing and provides a steady and predictable exposure of Milrinone.
Hyloris will initially pursue a new, longer term use indication in
patients with left ventricular assist devices (LVAD) who have
developed right heart failure. Orphan drug status4 has been granted
by the FDA in this latter indication and formulation patent claims
have been issued in the U.S., Japan and China, and are pending in
Europe. In a single dose PK study, patients treated with extended
release (ER) Milrinone at a dose of 14mg twice daily, exhibited
stable plasma levels within the therapeutic range. An open label
safety and tolerability study of ER Milrinone 14mg twice daily in
advanced (n=26) left heart failure patients with a history of
recurrent hospitalisation generated >5 years patient exposure
data and included a prolonged open-label compassionate use phase.
ER Milrinone was well tolerated, with no effect on heart rate or
blood pressure and was associated with improved functional activity
as defined by NYHA Classification.5 ER Milrinone treatment was also
associated with significant improvements in both quality of life
(Minnesota Living with Heart Failure Score) and functional capacity
(6-minute walk distance) with a trend towards improved renal
function.
About Heart Failure
(HF)6 and Standard of
Care Heart failure is a severe and chronic condition in
which the heart muscle is unable to pump enough blood to meet the
body’s need for blood and oxygen. HF usually develops because the
heart has been damaged by a heart attack, or because of other
conditions such as cardiomyopathy, a disease of the heart muscle.
It is the most rapidly growing cardiovascular disorder in the U.S.
with 870,000 new cases every year. HF is the most common cause of
hospitalisation in people aged over 65 years of age, with about 1
million hospitalisations in the U.S. per year, and 20% readmissions
following discharge. The average life expectancy is less than 5
years for 50% of all patients and 90% of patients with advanced HF
die within 1 year following diagnosis. Current standard of care
depends on disease severity and treatment of advanced HF is
predominantly palliative and includes the use of positive inotropes
(such as Milrinone IV), digoxin and opioids, as well as LVADs in
some cases, which are used either longer-term or as a bridge to
heart transplantation. In 2020, there were about 20,000 patients
with an LVAD implant in the U.S. and 30% of these patients
developed right HF. Over the next coming years, the LVAD patient
population is expected to grow at an average annual growth rate of
6% in the U.S.7
About Baker Heart and Diabetes
InstituteBaker Heart and Diabetes Institute is an
independent, internationally renowned medical research facility,
with a history spanning 95 years. The Institute’s work extends from
the laboratory to wide-scale community studies with a focus on the
diagnosis, prevention and treatment of cardiovascular disease,
diabetes, and their complications. www.baker.edu.au
About Hyloris Pharmaceuticals
Hyloris is a specialty biopharma company focused on innovating,
reinventing, and optimising existing medications to address
important healthcare needs and deliver relevant improvements for
patients, healthcare professionals and payors. Hyloris has built a
broad, patented portfolio of 13 reformulated and repurposed
value-added medicines that have the potential to offer significant
advantages over available alternatives. Outside of its core
strategic focus, the Company also has 3 high barrier generic
products in development and registration phase. Two products are
currently in initial phases of commercialisation with partners:
Sotalol IV for the treatment of atrial fibrillation, and Maxigesic®
IV, a non-opioid post-operative pain treatment. The Company’s
development strategy primarily focuses on the FDA’s 505(b)2
regulatory pathway, which is specifically designed for
pharmaceuticals for which safety and efficacy of the molecule have
already been established. This pathway can reduce the clinical
burden required to bring a product to market, and significantly
shorten the development timelines and reduce costs and risks.
Hyloris is based in Liège, Belgium. For more information, visit
www.hyloris.com and follow-us on LinkedIn.
For more information, please contact
Hyloris Pharmaceuticals: Marieke VermeerschVP Investor
Relations and Corporate Communicationsinvestorrelations@hyloris.com
Disclaimer and forward-looking statementsHyloris
means “high yield, lower risk”, which relates to the 505(b)(2)
regulatory pathway for product approval on which the Issuer
focuses, but in no way relates or applies to an investment in the
Shares.Certain statements in this press release are
“forward-looking statements.” These forward-looking statements can
be identified using forward-looking terminology, including the
words "believes", "estimates," "anticipates", "expects", "intends",
"may", "will", "plans", "continue", "ongoing", "potential",
"predict", "project", "target", "seek" or "should", and include
statements the Company makes concerning the intended results of its
strategy. These statements relate to future events or the Company’s
future financial performance and involve known and unknown risks,
uncertainties, and other factors, many of which are beyond the
Company’s control, that may cause the actual results, levels of
activity, performance or achievements of the Company or its
industry to be materially different from those expressed or implied
by any forward-looking statements. The Company undertakes no
obligation to publicly update or revise forward-looking statements,
except as may be required by law.
1 A left ventricular assist device (LVAD) is a
battery-operated, mechanical surgically implanted pump, which helps
the left ventricle (main pumping chamber of the heart) pump blood
to the rest of the body. It is used as a bridge to a heart
transplantation or as destination therapy2 Nanayakkara et al. Am J
Cardiol. 2018; Nanayakkara et al. J. Am. Heart Assoc. 2020; LVAD
study results: publication pending3 Nanayakkara et al, American
Journal of Cardiology, 2018
4 FDA’s Orphan Drug Designation Program provides
orphan status to drugs and biologics which are defined as those
intended for the safe and effective treatment, diagnosis or
prevention of rare diseases and disorders that affect < 200,000
people in the U.S. Orphan designation qualifies the sponsor of the
drug for various development incentives, including eligibility for
7 years of market exclusivity upon approval, exemption from FDA
application fees, tax credits for qualified clinical trials, and
other potential assistance in the development process.5 The New
York Heart Association (NYHA) Classification provides a way of
classifying the extent of heart failure. It classifies patients in
one of four categories based on their limitations during physical
activity; the limitations/symptoms in regard to normal breathing
and varying degrees in shortness of breath and or angina pain.6
Centers for Disease Control (CDC); Virani et al, Update report
American Heart Association (AHA), Circulation, 2020; Heart Disease
and Stroke Statistics, AHA; Argiriou et al, Journal of Thoratic
Disease, 20147 Grand View Research, Inc., 2021; Triangle Insights,
2016
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