ABIONYX Announces Positive Clinical Results From CER-001 in an Ultrarare Kidney Disease Published in the Annals of Internal M...
02 Mars 2021 - 7:30AM
Business Wire
- Demonstrated efficacy in renal and
ophthalmic indication
- Confirmation of the breakthrough therapy
status of CER-001 in kidney and ophthalmic diseases
- Systemic mechanism of action of
CER-001
Regulatory News:
ABIONYX Pharma (FR0012616852 - ABNX -
PEA PME eligible), a new generation biotech company dedicated
to the discovery and development of innovative therapies for
patients, today announces positive clinical results from CER-001 in
an ultrarare kidney disease published in the Annals of Internal
Medicine, the highest cited and ranked internal medicine
journal.
The patient, who was about to undergo dialysis due to the
rapid decline in renal function, was able to avoid the need for
dialysis during his treatment with CER-001. In addition, the
patient who was suffering from lipid deposits in the corneas saw
the visual blurring disappear. This clear improvement in visual
function is still observed after 1 year of follow-up.
As a reminder, the French Drug Safety Agency (Agence Nationale
de Sécurité du Médicament or ANSM), granted a named patient
Temporary Authorization for Use (“ATU nominative”) for CER-001 in
an untreated, ultra-rare renal disease early 2020.
The patient with inherited mutations in the lecithin-cholesterol
acyltransferase (LCAT) gene developed glomerulopathy and corneal
lipid deposits and displayed very low circulating levels of
high-density lipoprotein (HDL) and apoA-I. In this ATUn granted in
the familial LCAT deficiency disease the patient was treated with
CER-001, an apoA-I-containing HDL mimetic, to help in preventing
rapidly progressive kidney failure.
CER-001 was completely safe and very well tolerated at doses
of 10-30 mg/kg/week
CER-001 was given intravenously at a dose of 10 mg/kg thrice a
week for 3 weeks, then twice a week for 3 weeks and once a week for
3 weeks. Thereafter, the dose was increased to 20 mg/kg/week for 6
weeks to reach the weekly dose that stabilized eGFR.
Whereas eGFR rapidly decreased from 41 to 19 mL/min/1.73 m2
during the 9 months that preceded the start of CER-001, eGFR only
decreased from 19 to 17 mL/min/1.73 m2 during the 11 months
following treatment introduction.
Urinary protein-to-creatinine ratio (uPCr) decreased from 7 to
0.25 g/g at day 10, with undetectable albuminuria at that time, but
returned to initial values thereafter. However nephrotic syndrome
disappeared with serum albumin increased from 29 to 37 g/L during
the treatment period and then the follow-up.
The publication states that CER-001 prevented further kidney
function decline. The patient who was about to be dialysed due to
rapidly declining kidney function, was able to avoid the need for
dialysis during their treatment with CER-001.
No other treatment was introduced strongly suggesting that
stabilization of the renal function relied on the administration of
CER-001.
The publication confirms the breakthrough therapy status of
CER-001 in kidney diseases
The publication mentions that CER-001 effects on renal
progression should be assessed in more common proteinuric diseases,
including diabetic nephropathy or extra-membranous
glomerulonephritis (two diseases characterized by a reduction in
local LCAT activity, gloemrular lipid deposits, and inflammatory
processes), or other nephropathies related to lipid deposits.
The publication reveals the systemic mechanism of action of
CER-001 and broadens the field of breakthrough innovations to
ophthalmology
The publication mentions positive extra-renal clinical results
and the disappearance of visual blurring secondary to corneal
deposits. This clear improvement in visual function is still
observed after 1 year of follow-up.
The clear improvement of the blurred vision at the end of the
follow-up suggests that the anti-inflammatory properties and/or the
increase in the Reverse Cholesterol Transport (RCT) of CER-001 can
improve vision in FLD patients. This finding and previous data
showing the role of apoA-I in the development of corneal clouding
and blurring vision will pave the way to interventional studies
testing CER-001 in patients developing lipid corneal deposits from
other pathologies such as secondary lipid keratopathy or inherited
corneal dystrophy.
Prof. Stanislas Faguer, nephrologist at Department of Nephrology
and Organ Transplantation Reference Center for Rare Kidney
Diseases, Hospital Rangueil, CHU Toulouse, states : “These positive
clinical data demonstrate that CER-001 prevented significantly
kidney function decline. ApoA-I containing HDL mimetic CER-001 is
the first treatment that has proven its ability to slow the
progression of renal failure and reduce visual discomfort secondary
to corneal lipid deposits in a patient with FLD. We look forward to
conducting new clinical studies of CER-001 to assess its ability to
improve the prognosis of other orphan or common kidney diseases for
which no effective treatment is currently available.”
Cyrille Tupin, Managing Director of ABIONYX Pharma concludes :
“These positive clinical data published in one of the most cited
medical journals prove the efficacy of our bioproduct and reveal
the relevant repositioning of CER-001 in a severe renal indication
where there has been no breakthrough therapeutic innovation in the
last 10 years. This represents an important step forward in the
clinical development of CER-001. I would like to warmly thank
Professor Faguer for his major contribution and all the teams at
the Toulouse University Hospital for their continued collaboration.
We are very grateful to the French National Agency for Drug Safety
for accepting Prof. Faguer's request for ATUn, as well as to the
patient who fully supported the therapeutic project that led to
this discovery. The ABIONYX team continues to explore other
potential medical benefits associated with the CER-001, an HDL
mimetic. We are committed to advancing the clinical development of
the mimetic HDL as quickly and safely as possible while
investigating new indications in ophtalmology so that we can help
address new rare or orphan diseases with no existing
treatment.”
Data tables can be found in the article
https://www.acpjournals.org/doi/10.7326/L20-1300.
About Annals of Internal Medicine
Annals of Internal Medicine is an academic medical journal
published by the American College of Physicians (ACP). It is one of
the most widely cited and influential specialty medical journals in
the world. The most recent (2019) Impact Factor for Annals of
Internal Medicine is 21.317 (Clarivate Analytics). According
to the new 2019 Journal Citations Reports from Clarivate Analytics,
Annals is the highest cited and ranked internal medicine journal in
the category of Medicine, General and Internal.
About ABIONYX Pharma
ABIONYX Pharma is a new generation biotech company dedicated to
the discovery and development of innovative therapies for patients.
The biotech assets inherited from CERENIS Therapeutics constitute a
rich portfolio of valuable programs for the treatment of metabolic
diseases as well as with a HDL targeted drug delivery platform.
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NewCap Investor relations Louis-Victor Delouvrier
abionyx@newcap.eu +33 (0)1 44 71 98 53
NewCap Media relations Nicolas Merigeau abionyx@newcap.eu
+33 (0)1 44 71 94 98
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