BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a
commercial-stage biopharmaceutical company focused on genetic
diseases, today announced that the United States Food and Drug
Administration (FDA) has granted Regenerative Medicine Advanced
Therapy (RMAT) designation to BBP-812, an investigational
intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene
therapy for the treatment of Canavan disease. RMAT designation was
granted following the FDA’s review of clinical data from the
CANaspire Phase 1/2 clinical trial investigating BBP-812 as a
potential therapy to address the unmet medical needs of individuals
with Canavan disease.
RMAT is an expedited FDA program available to sponsors of
regenerative medicine therapies intended to treat, modify, reverse,
or cure serious conditions. Benefits of the RMAT designation
include all the advantages of the Fast Track and Breakthrough
Therapy Designation programs, including faster and more frequent
interactions with the FDA to achieve early alignment on critical
aspects of the program. FDA granted RMAT designation based on its
review of 12 months of safety and efficacy data from the first
eight patients with Canavan disease dosed with BBP-812 in the
CANaspire Phase 1/2 clinical trial.
“We are honored to be granted RMAT designation for BBP-812 and
are eager to work closely with the FDA and the Canavan community
with the goal of bringing our therapy to families living with
Canavan disease as fast as possible,” said Eric David, M.D., J.D.,
CEO at BridgeBio Gene Therapy. “We are beyond grateful to the
children and their families who are participating in CANaspire, as
well as to the study investigators. RMAT will allow us to work more
closely with FDA to ensure we are responding to the urgency that
families feel.”
To date, results from CANaspire show that all patients
dosed with at least one follow-up assessment
have demonstrated improvements in functional outcomes in key
areas important to caregivers such as head control, sitting
upright, reaching for and grasping objects, and visual tracking.
All patients dosed with BBP-812 with at least one follow-up
assessment have shown reductions in N-acetylaspartate (NAA),
both in urine and in the central nervous system, to levels
associated with mild disease. BBP-812 has been well-tolerated, with
a safety profile generally consistent with that of other AAV9 gene
therapy programs.
“Canavan disease is an extremely rare and rapidly progressive
neurodegenerative disease that prevents most children from meeting
basic developmental milestones, such as crawling, walking,
speaking, and even holding their heads up. It is a terminal
diagnosis with no approved treatment to date. The news of the RMAT
designation, coupled with the preliminary results seen in the
clinical trial, provides hope to children worldwide living with
Canavan disease and their families,” said Kathleen Flynn, CEO
of National Tay-Sachs & Allied Diseases Association, an
advocacy organization dedicated to driving research, forging
collaboration, and supporting families within the Tay-Sachs,
Canavan, GM1, and Sandhoff disease communities.
In addition to RMAT designation, BBP-812 has been granted Orphan
Drug, Rare Pediatric Disease (RPDD), and Fast Track Designations
from the FDA, as well as Orphan Drug Designation from the European
Medicines Agency. With RPDD, if approved, BridgeBio may qualify for
a Priority Review Voucher.
About CANaspireCANaspire is a Phase 1/2
open-label study designed to evaluate the safety, tolerability, and
pharmacodynamic activity of BridgeBio’s AAV9 gene therapy
candidate, BBP-812, in pediatric patients with Canavan disease.
Each eligible patient will receive a single IV infusion of BBP-812.
The primary outcomes of the study are safety, as well as change
from baseline of urine and central nervous system NAA levels. Motor
function and development will also be assessed.
For more information about the CANaspire trial, visit
TreatCanavan.com or ClinicalTrials.gov (NCT04998396).
About Canavan DiseaseAffecting approximately
1,000 children in the U.S. and European Union, Canavan disease is
an ultra-rare, disabling and fatal disease with no approved
therapy. Most children are not able to meet developmental
milestones, are unable to crawl, walk, sit or talk, and die at a
young age. The disease is caused by an inherited mutation of the
ASPA gene that codes for aspartoacylase, a protein that breaks down
a compound called NAA. Deficiency of aspartoacylase activity
results in accumulation of NAA, and ultimately results in toxicity
to myelin in ways that are not currently well understood. Myelin
insulates neuronal axons, and without it, neurons are unable to
send and receive messages as they should. The current standard of
care for Canavan disease is limited to supportive therapy.
About BridgeBio Pharma, Inc.BridgeBio Pharma,
Inc. (BridgeBio) is a commercial-stage biopharmaceutical company
founded to discover, create, test and deliver transformative
medicines to treat patients who suffer from genetic diseases.
BridgeBio’s pipeline of development programs ranges from early
science to advanced clinical trials. BridgeBio was founded in 2015
and its team of experienced drug discoverers, developers and
innovators are committed to applying advances in genetic medicine
to help patients as quickly as possible. For more information
visit bridgebio.com and follow us
on LinkedIn, Twitter
and Facebook.
BridgeBio Pharma, Inc. Forward-Looking
StatementsThis press release contains forward-looking
statements. Statements BridgeBio makes in this press release may
include statements that are not historical facts and are considered
forward-looking within the meaning of Section 27A of the Securities
Act of 1933, as amended (the “Securities Act”), and Section 21E of
the Securities Exchange Act of 1934, as amended (the “Exchange
Act”), which are usually identified by the use of words such as
“anticipates,” “believes,” “continues,” “estimates,” “expects,”
“hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,”
“should,” “will,” and variations of such words or similar
expressions. BridgeBio intends these forward-looking statements to
be covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Exchange Act. These forward-looking statements,
including statements relating to the timing and success of
BridgeBio’s Phase 1/2 clinical trial of BBP-812 for the treatment
of Canavan disease, expectations, plans and prospects regarding
BridgeBio’s regulatory approval process for BBP-812, the ability of
BBP-812 to be the first therapeutic treatment option for children
born with Canavan disease, reflect BridgeBio’s current views about
its plans, intentions, expectations, strategies and prospects,
which are based on the information currently available to BridgeBio
and on assumptions BridgeBio has made. Although BridgeBio believes
that its plans, intentions, expectations, strategies and prospects
as reflected in or suggested by those forward-looking statements
are reasonable, BridgeBio can give no assurance that the plans,
intentions, expectations or strategies will be attained or
achieved. Furthermore, actual results may differ materially from
those described in the forward-looking statements and will be
affected by a number of risks, uncertainties and assumptions,
including, but not limited to, BridgeBio’s ability to continue and
complete its Phase 1/2 clinical trial of BBP-812 for the treatment
of Canavan disease, BridgeBio’s ability to advance BBP-812 in
clinical development according to its plans, the ability of BBP-812
to treat Canavan disease, the ability of BBP-812 to retain Fast
Track Designation, Rare Pediatric Drug Designation, Regenerative
Medicine Advanced Therapy Designation and Orphan Drug Designation
from the U.S. Food and Drug Administration and Orphan Drug
Designation from the European Medicines Agency, and potential
adverse impacts due to global health emergencies, including delays
in regulatory review, manufacturing and supply chain interruptions,
adverse effects on healthcare systems and disruption of the global
economy, the impacts of current macroeconomic and geopolitical
events, including changing conditions from hostilities in Ukraine
and in Israel and the Gaza Strip, increasing rates of inflation and
rising interest rates, on our business operations and expectations
as well as those risks set forth in the Risk Factors section of
BridgeBio’s most recent Annual Report on Form 10-K, and BridgeBio’s
other filings with the U.S. Securities and Exchange Commission.
Moreover, BridgeBio operates in a very competitive and rapidly
changing environment in which new risks emerge from time to time.
These forward-looking statements are based upon the current
expectations and beliefs of BridgeBio’s management as of the date
of this press release and are subject to certain risks and
uncertainties that could cause actual results to differ materially
from those described in the forward-looking statements. Except as
required by applicable law, we assume no obligation to update
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.
BridgeBio Contact:Vikram
Balicontact@bridgebio.com(650)-789-8220
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