ABIONYX Pharma Announces Its 2021 Half Year Financial Results and Provides an Update on Its Development Activities
09 Septembre 2021 - 6:55PM
Business Wire
- Orphan Drug Designation status granted for CER-001 for
Lecithin Cholesterol Acyltransferase (LCAT) Deficiency as both a
renal disease and an ophthalmic disease
- Progress in the fully-funded Phase 2a clinical trial,
RACERS, evaluating CER-001 in sepsis at high risk of developing
acute kidney injury
Regulatory News:
ABIONYX Pharma (Paris:ABNX) (FR0012616852 - ABNX - PEA
PME eligible), a next-generation biotech company dedicated to
the discovery and development of innovative therapies, announces
its 2021 half year financial results and provides an update on its
development activities.
Orphan Drug Designation status granted for CER-001 for LCAT
Deficiency as both a renal disease and an ophthalmic
disease
Following the receipt of a positive opinion from the Committee
for Orphan Medicinal Products (COMP) of the European Medicines
Agency (EMA) regarding the Orphan Drug Designation application for
its bio-HDL, the company has obtained the Orphan Drug Designation
(ODD) from the European Commission for its drug candidate CER-001,
as a potential treatment for LCAT deficiency. Two forms of LCAT
deficiency exist: Familial LCAT Deficiency (FLD) resulting from a
complete deficiency and clinically characterized by hemolytic
anemia, renal failure, most often leading to renal transplantation,
and by corneal opacities; and Fish Eye Disease (FED), resulting
from a partial deficiency and clinically characterized by corneal
opacities without renal involvement. The Orphan Drug Designation
obtained therefore covers both a renal and an ophthalmologic
indication.
Obtaining the ODD allows for assistance from the EMA with
clinical protocol design, access to a centralized marketing
authorization procedure valid in all EU member states, the
possibility for conditional marketing authorization, reduced
regulatory fees, and 10 years of market exclusivity from the time a
marketing authorization is granted. In addition to the development
advantages of obtaining the ODD, this designation marks a strong
and strategic regulatory recognition as the only bio-HDL to receive
this status in Europe.
The efficacy of CER-001 was announced last March when positive
clinical results were published in the scientific journal “Annals
of Internal Medicine” in this rare kidney disease. As a reminder,
the patient who was on the verge of dialysis due to the rapid
decline of his kidney function was able to avoid the need for
dialysis during treatment with CER-001. In addition, the patient
who suffered from lipid deposits in the corneas experienced the
disappearance of visual blur, a clear improvement in visual
function that continued to be observed after 1 year of
treatment-free follow-up.
Progress in the Phase 2a clinical trial, RACERS, evaluating
CER-001 in sepsis at high risk of developing acute kidney
injury
Following the announcement last June of the enrollment of the
first patient in the Phase 2a clinical trial evaluating CER-001,
the Bio-HDL, as a potential treatment for patients with sepsis at
high risk of developing acute kidney injury, RACERS, the study,
evaluating the safety and efficacy of CER-001 in a total of 20
patients, continues to advance. The primary endpoints of the study
will be the onset and severity of acute kidney injury according to
KDIGO criteria, as well as the safety and tolerability of dosing
regimens, in order to select the optimal dose of CER-001. The
clinical study is being conducted in partnership with the
University of Bari and the Consorzio per Valutazioni Biologiche e
Farmacologiche (CBVF) and is already fully funded.
Selected Financial Information (at June 30 / IFRS
Consolidated accounts)
Millions €
H1 2021
H1 2020
Sales
0.03
0
R&D expenses
(2.03)
(0.41)
Administrative and commercial
expenses
(0.58)
(0.63)
Operating income
(2.59)
(1.04)
Financial products
0.08
0.19
Financial expenses
(0.04)
(0.07)
Financial Result
0.04
0.12
Net income
(2.55)
(0.92)
Net income per share (€)
(0.10)
(0.04)
Net cash flow from operating
activities
(4.27)
0.57
Net cash flow from investing
activities
(0.01)
(0.02)
Net cash flow from financing
activities
(0.05)
(0.07)
(Decrease) / Increase in cash
position
(4.32)
0.48
Cash and cash equivalents at end of
period
4.83
8.81
Details of the main changes in the consolidated financial
statements
ABIONYX Pharma recorded its first sales of €27 K in Q1,
corresponding to the supply of CER-001 for the treatment of a
patient for one month in the context of a new ATUn, following
positive results obtained in March 2021 in the treatment of LCAT
deficiency in a patient in France.
The increase in research and development expenses, which
amounted to €2,029 K for the period, compared to €412 K in the
first half of 2020, corresponds mainly to the evolution of
subcontracting and consulting expenses which amounted to €1,584 K.
This increase is mainly related to the launch of a new production
campaign for the CER-001 bio product, initiated at the end of 2020,
and the restart of new R&D activities.
General and administrative expenses amounted to €584 K as of
June 30, 2021, compared to €629 K as of June 30, 2020. After taking
these expenses into account, the operating profit went from a loss
of €1,041 K as of June 30, 2020 to a loss of €2,586 K as of June
30, 2021. The financial income shows a surplus €41 K in the first
half of 2021, compared to the €121 K income recorded for the first
half of 2020. The net result shows a loss of €2,546 K as of June
30, 2021, compared to a loss of €920 K as of June 30, 2020. Cash
and cash equivalents amounted to €4.8 million on June 30, 2021.
About ABIONYX Pharma
ABIONYX Pharma is a new generation biotech company that aims to
contribute to health through innovative therapies in indications
where there is no effective or existing treatment, even the rarest
ones. Thanks to its partners in research, medicine,
biopharmaceuticals and shareholding, the company innovates on a
daily basis to propose drugs for the treatment of renal and
ophthalmological diseases, or new HDL vectors used for targeted
drug delivery.
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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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