CervoMed Announces Orphan Drug Designation Granted to Neflamapimod by U.S. Food and Drug Administration for the Treatment of Frontotemporal Dementia
27 11월 2024 - 9:00PM
CervoMed Inc. (NASDAQ: CRVO), a clinical-stage company focused on
developing treatments for age-related neurologic disorders, today
announced that its oral investigational drug neflamapimod has been
granted Orphan Drug Designation by the U.S. Food and Drug
Administration (FDA) for the treatment of frontotemporal dementia
(FTD).
“We are pleased to have received Orphan Drug Designation as it
implicitly recognizes the scientific rationale and potential for
neflamapimod to treat this debilitating condition. Patients
diagnosed with frontotemporal dementia have no available treatment
options, and this rare condition is extremely burdensome to
patients and caregivers alike,” said John Alam, MD, Chief Executive
Officer of CervoMed. “Within this year, there have been multiple
scientific presentations and publications that indicate
neflamapimod targets specific pathogenic mechanisms associated with
FTD. We are in active discussions with clinical thought leaders
regarding the design of a proof-of-principle study in FTD, while
continuing to prepare to advance neflamapimod into a Phase 3 trial
in DLB in mid-2025.”
FDA Orphan Drug Designation is granted to investigational
therapies addressing rare medical diseases or conditions that
affect fewer than 200,000 people in the United States.
Importantly, the FDA also requires sufficient preclinical and/or
clinical data to establish a medically plausible basis for
expecting the drug to be effective in the rare disease for which
orphan drug status is granted. Orphan Drug status provides benefits
to drug developers, including assistance in the drug development
process, tax credits for clinical costs, exemptions from certain
FDA fees and seven years of post-approval marketing
exclusivity.
About Frontotemporal DementiaFTD is a rare
neurodegenerative disease, but it is one of the most common causes
of early onset dementia. It affects an estimated 50,000 to 60,000
people in the United States and roughly 110,000 in
the European Union, with potentially higher prevalence
in Asia and Latin America. Pathologically FTD is
characterized by significant neuronal loss (i.e. atrophy by MRI or
at autopsy) in the frontal and temporal regions of the cortex. The
pathogenic drivers underlying the neuronal loss are in most cases,
in roughly equal proportions, intraneuronal inclusions in those two
regions of the brain containing either the protein tau or TDP-43
(transactive response DNA binding protein); intranuclear inclusions
of fused in sarcoma (FUS) are also seen, but in fewer than 10% of
patients. Patients with FTD frequently develop symptoms such
as behavioral changes, lapses in judgment, and diminished language
skills when they are in their 40s and 50s with the disease running
its course in 7-10 years. There are no FDA- or EMA-approved
treatment options available for any form of FTD.
About NeflamapimodNeflamapimod is an
investigational, orally administered small molecule brain penetrant
drug that inhibits alpha isoform of the p38MAP kinase. In
preclinical studies, neflamapimod reversed synaptic dysfunction,
including and particularly within the part of the brain most
impacted in DLB – the basal forebrain cholinergic system. In Phase
1 and Phase 2 clinical studies involving more than 300
participants, neflamapimod has been shown to be generally well
tolerated. Results from the AscenD-LB Phase 2a clinical study
demonstrated that neflamapimod significantly improved dementia
severity (assessed by Clinical Dementia Rating Sum-of-boxes, or
CDR-SB) compared to placebo and significantly improved functional
mobility (assessed by Timed Up and Go Test, or TUG test) compared
to placebo. At the highest dose evaluated, neflamapimod also
improved results on a cognitive test battery. The treatment
response in AscenD-LB in patients with early-stage DLB (i.e., those
without biomarker evidence of tau pathology in the brain) was
substantial (effect size > 0.7) and greater than the overall
patient population. Neflamapimod is currently being evaluated in
the ongoing RewinD-LB Phase 2b study, a randomized, 16-week,
double-blind, placebo-controlled clinical trial evaluating oral
neflamapimod (40mg TID) in 159 patients with early-stage DLB.
Topline results from the RewinD-LB study are expected in December
2024.
About CervoMed
CervoMed Inc. (the “Company”) is a clinical-stage company
focused on developing treatments for age-related neurologic
disorders. The Company is currently developing neflamapimod, an
investigational, orally administered small molecule brain penetrant
that inhibits p38 mitogen-activated protein kinase alpha.
Neflamapimod has the potential to treat synaptic dysfunction, the
reversible aspect of the underlying neurodegenerative processes
that causes disease in DLB and certain other major neurological
disorders. Neflamapimod is currently being evaluated in a Phase 2b
study in patients with early-stage DLB.
Forward-Looking Statements
This press release includes express and implied forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended, regarding the intentions, plans,
beliefs, expectations or forecasts for the future of the Company,
including, but not limited to, the therapeutic potential of
neflamapimod and the anticipated timing and achievement of clinical
and development milestones, including neflamapimod’s potential as a
treatment for FTD, the completion and achievement of primary
endpoints of the RewinD-LB Phase 2b clinical trial, the Company’s
announcement of topline or other data therefrom, and the Company’s
intent to initiate a Phase 3 trial in DLB in mid-2025 assuming
positive topline results in the RewinD-LB trial. Terms such as
“believes,” “estimates,” “anticipates,” “expects,” “plans,” “aims,”
“seeks,” “intends,” “may,” “might,” “could,” “might,” “will,”
“should,” “approximately,” “potential,” “target,” “project,”
“contemplate,” “predict,” “forecast,” “continue,” or other words
that convey uncertainty of future events or outcomes (including the
negative of these terms) may identify these forward-looking
statements. Although there is believed to be reasonable basis for
each forward-looking statement contained herein, forward-looking
statements by their nature involve risks and uncertainties, known
and unknown, many of which are beyond the Company’s control and, as
a result, actual results could differ materially from those
expressed or implied in any forward-looking statement. Particular
risks and uncertainties include, among other things, those related
to: the Company’s available cash resources and the availability of
additional funds on acceptable terms; the results of the Company’s
clinical trials, including RewinD-LB; the likelihood and timing of
any regulatory approval of neflamapimod or the nature of any
feedback the Company may receive from the U.S. Food and Drug
Administration; the ability to implement business plans, forecasts,
and other expectations in the future; general economic, political,
business, industry, and market conditions, inflationary pressures,
and geopolitical conflicts; and the other factors discussed under
the heading “Risk Factors” in the Company’s Annual Report on Form
10-K for the year ended December 31, 2023 filed with the U.S.
Securities and Exchange Commission (SEC) on March 29, 2024, and
other filings that the Company may file from time to time with the
SEC. Any forward-looking statements in this press release speak
only as of the date hereof (or such earlier date as may be
identified). The Company does not undertake any obligation to
update such forward-looking statements to reflect events or
circumstances after the date of this press release, except to the
extent required by law.
Investor Contact:PJ KelleherLifeSci
AdvisorsInvestors@cervomed.com617-430-7579
CervoMed (NASDAQ:CRVO)
과거 데이터 주식 차트
부터 10월(10) 2024 으로 11월(11) 2024
CervoMed (NASDAQ:CRVO)
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