STOCKHOLM, March 15, 2021 /PRNewswire/ -- Calliditas
Therapeutics AB (NASDAQ: CALT, Nasdaq Stockholm: CALTX)
("Calliditas") today announced the submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for Nefecon, a novel oral formulation targeting down regulation of
IgA1 for the treatment of primary IgA Nephropathy (IgAN).
Calliditas is seeking accelerated approval under Subpart H for the
505(b)(2) application.
"This is a key milestone in the company's development and we are
looking forward to engaging with the agency. This is the first time
a drug specifically designed for IgAN is being submitted for
approval to the FDA and I believe that we are delivering a very
robust data package based on the successful outcome of our pivotal
Phase 3 trial and our large Phase 2b
trial, which also met both the primary and key secondary endpoints.
Calliditas has long been pioneering a treatment for IgAN based on
precision and disease modification that focuses on the origin of
the disease, with the hope of bringing help to thousands of
patients, so today is truly a special day," said CEO Renée
Aguiar-Lucander.
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. As previously reported, the study
achieved its primary endpoint of proteinuria reduction compared to
placebo, as well as showing stabilisation of eGFR at 9 months. The
NefIgArd trial also showed that Nefecon was generally
well-tolerated with a safety profile in keeping with the Phase
2b results. 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.
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.
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, which is supported by the statistical
framework based on the meta-analysis of clinical studies where an
intervention was carried out in patients with IgAN, as updated by
Thompson A et al, published in 20191. A
confirmatory study designed to provide data on long-term renal
benefit is fully recruited and is expected to read out in early
2023.
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 98 12 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 March 15, 2021 at 07:30
a.m. CET.
1. Clin J Am Soc Nephrol. 2019;14:469-81.
About Calliditas
Calliditas Therapeutics is a specialty pharmaceutical 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 adults with the autoimmune
renal disease primary IgA nephropathy (IgAN), for which there is a
high unmet medical need and there are no approved treatments.
Calliditas has recently read out topline data from Part A of
its global Phase 3 study in IgAN and, if approved, aims to
commercialize Nefecon in the United
States. Calliditas is also planning to start clinical trials
with NOX inhibitors in primary biliary cholangitis and head and
neck cancer in 2H 2021. 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
stabilisation 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 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 filing and the success 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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