Korro Announces Regulatory Filing for Initiation of KRRO-110 First-In-Human Study and Formation of Clinical Advisory Board
November 04 2024 - 6:25AM
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company
focused on developing a new class of genetic medicines based on
editing RNA for both rare and highly prevalent diseases, today
announced a submission to the Bellberry HREC for a Phase 1/2
clinical study of KRRO-110 for AATD. A Clinical Advisory Board
(CAB) comprised of distinguished lung and liver researchers and
experts in AATD has also been assembled to help guide the clinical
development strategy for KRRO-110.
“This regulatory filing reflects Korro’s
transition to a clinical-stage company, and another important
milestone in the development of KRRO-110,” said Kemi Olugemo, MD,
Chief Medical Officer at Korro. “There remains significant unmet
medical need for people living with AATD, and based on our
preclinical data, KRRO-110 has the potential to be a best-in-class
therapy given our differentiated approach for RNA editing. The
breadth of pulmonary and hepatic expertise in our CAB will be
invaluable to our clinical and regulatory strategy, ensuring we
consider the holistic needs of patients with AATD. We are honored
to work alongside the world's leading experts in AATD who share our
commitment to scientific excellence and improving patient
outcomes.”
Subject to approval by the HREC and acceptance
of the clinical trial notification (CTN) by Australia’s Therapeutic
Goods Administration (TGA), Korro anticipates dosing the first
participant in the Phase 1/2 study in the first quarter of 2025. An
interim data readout for the Phase 1/2 study of KRRO-110 is
anticipated in the second half of 2025, and completion of the study
is expected in 2026.
“This is a major achievement for a company
founded on RNA editing – to go from initial concepts and ideas to a
first-in-human clinical study. I am immensely proud of the progress
we have made since nomination of KRRO-110 for AATD in December 2023
and the contributions of the entire team at Korro,” added Ram
Aiyar, PhD, CEO and President of Korro. “Our robust pipeline of
fully owned and partnered programs showcases the transformative
potential of our OPERA™ platform. With a strong balance sheet to
support completion of the Phase 1/2 study and to advance the next
set of programs, I am excited to see the potential of our platform
realized.”
The newly appointed CAB members, listed
below, include:
- Daniel Chambers, MBBS (Hons
1, University Medal), MRCP, FRACP, MD, FQA, Professor of
Medicine, Thoracic & Transplant Physician, Interstitial Lung
Disease Expert and Translational Clinician Researcher at The
University of Queensland and The Prince Charles Hospital,
Australia. Prof. Chambers is an internationally recognized
authority in the fields of lung fibrosis, cell therapy for lung
disease and lung transplantation.
- Monica Goldklang,
MD, Assistant Professor of Medicine in Anesthesiology and
Director, Alpha-1 Foundation Clinical Resource Center, Columbia
University New York, USA. Dr. Goldklang’s main area of research
interest is in translational studies, investigating the
pathogenesis of smoking related lung disease and mechanisms of
protease upregulation in lung injury.
- Noel G. McElvaney,
MBBCh, FRCPI, DSc, Professor of Medicine
at the Royal College of Surgeons in Ireland, Head of the Irish
Centre for Genetic Lung Disease, Beaumont Hospital, Dublin,
Ireland, and Founder of the Alpha-1 Foundation of Ireland. Prof.
McElvaney’s main research areas are in AATD, chronic obstructive
lung disease and cystic fibrosis.
- Pavel Strnad, MD,
Professor of Translational Gastroenterology and Senior Physician at
the University Hospital Aachen, Department of Medicine III, Aachen,
Germany. Dr. Strnad currently leads a European initiative for the
study of AATD-associated liver disease.
- Jeffrey Teckman,
MD, Professor of Pediatrics, Biochemistry and Molecular
Biology at Saint Louis University, USA. Dr. Teckman’s research with
the Alpha-1 Foundation’s Liver Initiative focuses on adult Alphas,
and his work with the Childhood Liver Disease Research and
Education Network (ChiLDREN) focuses on rare pediatric liver
disease.
- Alice M. Turner,
MBChB (Hons), MRCP, PGCE (MedEd), PhD, Professor
of Respiratory Medicine and Department Director of Research
Knowledge and Transfer at the University of Birmingham Institute of
Applied Health Research, UK. Dr. Turner’s research interests are
predominantly in clinical aspects of AATD and chronic obstructive
pulmonary disease, fields in which she has published widely.
For full CAB member bios, visit
www.korrobio.com/our-team/.
About alpha-1 antitrypsin deficiency
(AATD) and KRRO-110AATD is a genetic disorder caused by a
single missense mutation (G-to-A) in the SERPINA1 gene. Affected
adult individuals experience pulmonary emphysema and/or hepatic
cirrhosis, as well as end organ manifestations. KRRO-110 is the
first RNA editing oligonucleotide product candidate from Korro’s
proprietary RNA editing platform, Oligonucleotide Promoted Editing
of RNA (OPERA™). KRRO-110 is designed to co-opt an endogenous
enzyme, Adenosine Deaminase Acting on RNA’s (ADAR), to edit the “A”
variant on SERPINA1 RNA, repair an amino acid codon, and restore
secretion of normal AAT protein. This repair of the endogenous
protein has the potential to clear protein aggregates from within
liver cells to create a potentially clinically differentiated
benefit for liver function and to preserve lung function by
providing an adequate amount of normal AAT protein.
About KorroKorro is a
biopharmaceutical company focused on developing a new class of
genetic medicines for both rare and highly prevalent diseases using
its proprietary RNA editing platform. Korro is generating a
portfolio of differentiated programs that are designed to harness
the body’s natural RNA editing process to affect a precise yet
transient single base edit. By editing RNA instead of DNA, Korro is
expanding the reach of genetic medicines by delivering additional
precision and tunability, which has the potential for increased
specificity and improved long-term tolerability. Using an
oligonucleotide-based approach, Korro expects to bring its
medicines to patients by leveraging its proprietary platform with
precedented delivery modalities, manufacturing know-how, and
established regulatory pathways of approved oligonucleotide drugs.
Korro is based in Cambridge, Massachusetts. For more information,
visit korrobio.com.
Forward-Looking
StatementsCertain statements in this press release may
constitute “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements include, but are not limited to, express
or implied statements regarding expectations, hopes, beliefs,
intentions or strategies of Korro regarding the future including,
without limitation, express or implied statements regarding:
approval by the Australian Bellberry Human Research Ethics
Committee of Korro’s regulatory filing to commence a Phase 1/2
clinical trial of KRRO-110 for AATD; acceptance by Australia’s
Therapeutic Goods Administration of Korro’s clinical trial
notification for the Phase 1/2 clinical trial of KRRO-110 for AATD;
the timing of Korro dosing the first participant, interim data
readout and completion of the Phase 1/2 clinical trial, including
its ability to complete such trial; KRRO-110’s potential to be a
best-in-class therapy; the transformative potential of Korro’s
OPERA™ platform; Korro’s ability to advance its next set of
pipeline programs and the strength of its balance sheet to support
completion; among others. In addition, any statements that refer to
projections, forecasts, or other characterizations of future events
or circumstances, including any underlying assumptions, are
forward-looking statements. The words “anticipate,” “believe,”
“continue,” “could,” “estimate,” “expect,” “intend,” “may,”
“might,” “plan,” “possible,” “potential,” “predict,” “project,”
“should,” “strive,” “would,” “aim,” “target,” “commit,” and similar
expressions may identify forward-looking statements, but the
absence of these words does not mean that statement is not forward
looking. Forward-looking statements are based on current
expectations and assumptions that, while considered reasonable are
inherently uncertain. New risks and uncertainties may emerge from
time to time, and it is not possible to predict all risks and
uncertainties. Factors that may cause actual results to differ
materially from current expectations include, but are not limited
to, various factors beyond management’s control including risks of
conducting a first-in-human clinical trial; challenges with
addressing any regulatory concerns necessary to proceed with
enrollment and dosing; risks associated with enrolling sufficient
participants and other risks inherent in biopharmaceutical
development; risks associated with conducting pre-clinical studies
and clinical trials and risks of replicating results from
pre-clinical studies in clinical trials; and other risks associated
with obtaining regulatory approvals and protecting intellectual
property; as well as risks associated with general economic
conditions; and other risks and uncertainties indicated from time
to time in Korro’s filings with the SEC, including Part II Item 1A.
“Risk Factors” in Korro’s Quarterly Report on Form 10-Q filed with
the SEC on August 13, 2024, as such may be amended or supplemented
by its other filings with the SEC. Nothing in this press release
should be regarded as a representation by any person that the
forward-looking statements set forth herein will be achieved or
that any of the contemplated results of such forward-looking
statements will be achieved. You should not place undue reliance on
forward-looking statements in this press release, which speak only
as of the date they are made and are qualified in their entirety by
reference to the cautionary statements herein. Except as required
by law, Korro does not undertake or accept any duty to release
publicly any updates or revisions to any forward-looking statements
to reflect any change in its expectations or in the events,
conditions or circumstances on which any such statement is based.
This press release does not purport to summarize all of the
conditions, risks and other attributes of an investment in
Korro.
Korro Bio Contact
Information
Investor & Media ContactTim
PalmerIR@korrobio.com
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