STOCKHOLM, April 28, 2021 /PRNewswire/ -- Calliditas
Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX)
("Calliditas") today announced that the U.S. Food and
Drug Administration (FDA) has accepted the submission and granted
Priority Review for the New Drug Application (NDA) for Nefecon, a
down regulator of IgA1 for the treatment of IgA nephropathy (IgAN.)
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date
of September 15, 2021.
"We are very excited about being granted priority review, which
reflects the unmet medical need of IgAN. We look forward to
engaging with the agency and work towards an accelerated approval
later this year so that we will be in a position to provide the
first approved medication for IgAN patients," said CEO Renée
Aguiar-Lucander.
As previously reported, Calliditas filed an NDA with the FDA on
March 15, 2021. The NDA submission is
based on positive data from Part A of the NefIgArd pivotal Phase 3
study, a randomized, double-blind, placebo-controlled,
international multicenter study designed to evaluate the efficacy
and safety of Nefecon compared to placebo in 200 adult patients
with IgAN. The NefIgArd study read out topline data in November 2020 and achieved its primary endpoint
of proteinuria reduction compared to placebo as well as showing
stabilization of eGFR at 9 months. The submission also includes
clinical data from the Phase 2 NEFIGAN trial, which also met the
same primary and secondary endpoints as the NefIgArd study. Both
studies showed that Nefecon was generally well-tolerated, with a
similar safety profile across both sets of results.
"Having a target action date provides us with a clear timeline
as we continue to expand our US organization and prepare for
commercialization in the fourth quarter of this year, subject to
approval," said Head of North America Commercial Andrew Udell.
Calliditas has applied for accelerated approval, which allows
drugs targeting serious conditions that fill an unmet medical need
to be approved based on a surrogate endpoint. The surrogate
endpoint in the pivotal Phase 3 trial NefIgArd was reduction of
proteinuria versus placebo. The confirmatory Part B of the NefIgArd
study, designed to provide data on long-term renal benefit, is
fully recruited and is expected to read out in early 2023.
Calliditas is the only company which has achieved positive data
in randomized, double-blind, placebo-controlled Phase 2b and Phase 3 clinical trials in IgAN. If
approved, Nefecon would become the first therapy specifically
designed and approved for the treatment of IgAN, with the potential
to be disease modifying. Subject to approval by the FDA, Calliditas
intends to commercialize Nefecon for IgAN on its own in
the United States.
For further information, please contact:
Marie Galay, IR Manager,
Calliditas
Tel.: +44 79 55 12 98 45, email: marie.galay@calliditas.com
The information in the press release is information that
Calliditas is obliged to make public pursuant to the EU Market
Abuse Regulation. The information was sent for publication, through
the agency of the contact persons set out above, on April 28, 2021 at 09:50
a.m. CET.
About Calliditas
Calliditas Therapeutics is a biopharma company based in
Stockholm, Sweden focused on
identifying, developing and commercializing novel treatments in
orphan indications, with an initial focus on renal and hepatic
diseases with significant unmet medical needs. Calliditas' lead
product candidate, Nefecon, is a proprietary, novel oral
formulation of budesonide, an established, highly potent local
immunosuppressant, for the treatment of the autoimmune renal
disease IgA nephropathy, or IgAN, for which there is a high unmet
medical need and there are no approved treatments. Calliditas is
running a global Phase 3 study within IgAN and, if approved, aims
to commercialize Nefecon in the United
States. Calliditas is listed on Nasdaq Stockholm (ticker:
CALTX) and the Nasdaq Global Select Market (ticker: CALT). Visit
www.calliditas.com for further information.
About the NefIgArd Study
The global Phase 3 clinical trial NefIgArd, which investigated
the effect of Nefecon versus placebo in patients with primary IgA
nephropathy (IgAN), consists of two parts.
Part A, which was designed to provide the basis for regulatory
submissions and approvals, evaluates data on the efficacy and
safety of Nefecon. The first patient in the NefIgArd trial was
randomized by Calliditas in November
2018, and in December 2019,
Calliditas announced the full recruitment of Part A, across
approximately 146 sites in 19 countries. Calliditas read out
topline data for Part A in November
2020.
The trial met its primary objective of demonstrating a
statistically significant reduction in urine protein creatinine
ratio, UPCR or proteinuria, after 9 months of treatment with 16 mg
of Nefecon compared to placebo, with significant continued
improvement at 12 months. The primary endpoint analysis showed a
31% mean reduction in the 16 mg arm versus baseline, with placebo
showing a 5% mean reduction versus baseline, resulting in a 27%
mean reduction at 9 months (p=0.0005) of the 16 mg arm versus
placebo. The trial also met the key secondary endpoint, showing a
statistically significant difference in estimated glomerular
filtration rate or eGFR after 9 months of treatment with Nefecon
compared to placebo. The key secondary endpoint, eGFR, showed a
treatment benefit of 7% versus placebo at 9 months, reflecting
stabilization in the treatment arm and a 7% decline of eGFR in the
placebo arm (p=0.0029). This reflected an absolute decline of 4.04
ml/min/1.73m2 in the placebo group over 9 months compared to a 0.17
ml/min/1.73m2 decline in the treatment group. Nefecon was also
generally well-tolerated, and the safety profile was in keeping
with the Phase 2b results and
consistent with the known safety profile of budesonide.
Part B of the NefIgArd study is designed to be a confirmatory
post-market observational trial to confirm long-term renal
protection and assess the difference in kidney function between
treated and placebo patients as measured by eGFR over a two-year
period from the start of dosing of each patient. The 360-patient
population of the complete Phase 3 trial includes another 160
patients enrolled in addition to the 200 patients from Part A. The
trial was fully recruited in January
2021, and aims to read out data in early 2023, after all
patients have completed 2 years in the trial.
About Nefecon
Nefecon is a patented oral formulation of a potent and
well-known active substance – budesonide – for targeted release.
The formulation is designed to deliver the drug to the Peyer's
patch region of the lower small intestine, where the disease
originates, as per the predominant pathogenesis models. Nefecon is
derived from the TARGIT technology, which allows for the substance
to pass through the stomach and intestine without being absorbed,
and to be released in a pulse like fashion only when it reaches the
lower small intestine.
The combination of dose and optimized release profile is
required to be effective in patients with IgAN, as shown in a large
Phase 2b trial, completed by
Calliditas. In addition to its potent local effect, another
advantage of using this active substance is that it has very low
bioavailability, i.e. around 90% of it is inactivated in the liver
before it reaches the systemic circulation. This means that a high
concentration can be applied locally where needed but with only
very limited systemic exposure and side effects.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, statements
regarding Calliditas' strategy, business plans, regulatory
submissions and focus. The words "may," "will," "could," "would,"
"should," "expect," "plan," "anticipate," "intend," "believe,"
"estimate," "predict," "project," "potential," "continue," "target"
and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Any forward-looking statements in this
press release are based on management's current expectations and
beliefs and are subject to a number of risks, uncertainties and
important factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, any related to Calliditas' business, operations, the
potential for FDA acceptance for and the success and timeline of
its regulatory marketing application for Nefecon, clinical trials,
supply chain, strategy, goals and anticipated timelines,
competition from other biopharmaceutical companies, and other risks
identified in the section entitled "Risk Factors" in Calliditas'
reports filed with the Securities and Exchange Commission.
Calliditas cautions you not to place undue reliance on any
forward-looking statements, which speak only as of the date they
are made. Calliditas disclaims any obligation to publicly update or
revise any such statements to reflect any change in expectations or
in events, conditions or circumstances on which any such statements
may be based, or that may affect the likelihood that actual results
will differ from those set forth in the forward-looking statements.
Any forward-looking statements contained in this press release
represent Calliditas' views only as of the date hereof and should
not be relied upon as representing its views as of any subsequent
date.
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SOURCE Calliditas Therapeutics