- Positive results from Phase 2 NASH Trial (DESTINY-1) for
PXL065 reported:
- Primary efficacy endpoint met: PXL065-treated patients
achieved statistically significant improvements in the relative
decrease in liver fat content at 36-weeks for all doses
- Paired liver biopsies confirmed strong improvement of
fibrosis and other parameters
- PXL065 was observed to be safe and well tolerated with no
dose-dependent increase in body weight and no increased lower
extremity edema vs. placebo
- Based on positive results from the DESTINY-1 trial, PXL065
will be prioritized for further development in NASH. PXL770
development will focus exclusively on rare diseases, driven by
promising data which showed strong potential in multiple rare
metabolic indications.
- Completion of preclinical studies support potential to
advance PXL770 into Phase 2 development for autosomal-dominant
polycystic kidney disease (ADPKD)
- Fast Track and Orphan Drug Designation for PXL065 and PXL770
in adrenoleukodystrophy (ALD) granted by the U.S. Food and
Drug Administration (FDA)
- Cash runway extended through at least February 2023 based
upon debt restructuring agreement with IPF Partners (IPF) and
equity-linked financing facility with Iris Capital Investment
(IRIS)
- As of June 30, 2022, cash and cash equivalents were EUR 16.1
million
The management team will host webcast conference calls on
Wednesday, September 21 at:
- 1:00 pm CEST, Paris time (7:00 am ET) in French
and
- 8:45 am ET, New York time (2:45 pm CEST) in
English.
A presentation will be available after the event on Poxel's
website in the Investor section.
To register for the webcast in French:
https://us02web.zoom.us/webinar/register/WN_z5Pgz18KRBqubDQ2QCjm7A
To register for the webcast in English:
https://us02web.zoom.us/webinar/register/WN_WC9_M_yvR_aKmJVcHJPXrg
POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage
biopharmaceutical company developing innovative treatments for
chronic serious diseases with metabolic pathophysiology, including
non-alcoholic steatohepatitis (NASH) and rare metabolic disorders,
today announced its financial results for the period ended June 30,
2022 and provided a corporate update.
Thomas Kuhn, Chief Executive Officer of Poxel, stated: “Thus
far, 2022 has been marked by important achievements for Poxel. On
the clinical front, our Phase 2 NASH DESTINY-1 trial for PXL065 met
its objectives demonstrating a statistically significant effect
with a favorable safety profile. Based on these positive results,
PXL065 will be prioritized for further development in NASH and we
will initiate discussions for a potential pivotal program in NASH.
In parallel, we will focus PXL770 development efforts exclusively
in rare diseases on the basis of our promising data which
demonstrated strong potential in multiple rare metabolic
indications. In addition, these recent PXL065 results have
validated our hypothesis that the deuterated-thiazolidinediones
(d-TZD) platform reduces PPARγ side-effects
while retaining the efficacy benefits of TZDs, and thus warrants
exploration in other diseases, such as ALD. Over the summer, we
also extended our cash runway through the restructuring of our debt
and an equity-linked financing facility. This accomplishment
provides us further flexibility to secure additional financing
solutions necessary to execute our rare disease strategy.”
H1 Key Events
Clinical Updates
- In ALD, PXL770 is prepared to advance into a Phase 2a biomarker
proof-of-concept (POC) clinical trial in male patients with
adrenomyeloneuropathy (AMN), the most common ALD subtype. The
12-week study will evaluate pharmacokinetics, safety and potential
for efficacy based on relevant disease biomarkers, such as the
effect on very long chain fatty acids (VLCFA), the characteristic
plasma marker of the disease. Considering the DESTINY-1 results for
PXL065 in NASH, which validated the deuterium-modified
thiazolidinedione (TZD) platform, a second identical study
continues to be planned in order to assess the potential of the
deuterium-modified TZD platform with PXL065 in ALD. ALD studies are
expected to initiate as soon as possible, subject to additional
financing.
- In February and April, the FDA awarded Fast Track Designation
(FTD) to PXL065 and PXL770 respectively, for ALD. The FDA grants
FTD to investigational drugs which treat a serious or
life-threatening condition, and which fill an unmet medical need.
Filling an unmet medical need is defined as providing a therapy
where none exists or providing a therapy which may be potentially
better than available therapy. The key benefits of FTD comprise
enhanced access to the FDA, with regular and more frequent
opportunities for consultation and discussion.
- In May, the FDA granted Orphan Drug Designation (ODD)1 to
PXL065 and PXL770 for ALD. ODD confers a company a potential
seven-year window of exclusive marketing rights following FDA
approval, along with a reduction in certain application fees, and
tax credits for expenses related to qualified clinical trials
conducted after orphan designation is received.
- Two preclinical articles on X-Linked Adrenoleukodystrophy (ALD)
for PXL065 and PXL770 were published:
- The article on PXL065 was published in The Journal of Inherited
Metabolic Disease (“JIMD”) and is entitled “Therapeutic potential
of deuterium-stabilized (R)-pioglitazone - PXL065 - for X-linked
adrenoleukodystrophy”. It is available here:
https://pubmed.ncbi.nlm.nih.gov/35510808/.
- The article on PXL770 was published in The Journal of
Pharmacology and Experimental Therapeutics (“JPET”), and is
entitled “Beneficial effects of the direct AMP-Kinase activator
PXL770 in in vitro and in vivo models of X-Linked
Adrenoleukodystrophy”. It is available here:
https://jpet.aspetjournals.org/content/early/2022/06/25/jpet.122.001208.
- In ADPKD, preclinical studies were completed and demonstrated
efficacy of PXL770 in in vitro cyst assays including ADPKD
patient-derived cells. In vivo efficacy in a classical animal model
of ADPKD was also observed including improvements in renal
function, kidney weight, cyst index, and other benefits in kidney
tissues. Initiation of development planning and regulatory
interactions is underway.
Corporate Update
- In June, the U.S. Patent and Trademark Office (PTO) issued a
new patent for PXL065 that describes a specific form of PXL065 with
unique properties. Importantly, this recently issued patent
provides additional protection through 2041 and could expand
protection for PXL065 worldwide, with the potential for an
additional 5 years through patent term extension.
- On June 21, Poxel held its annual general meeting. The
shareholders approved all the resolutions that were recommended by
the Board of Directors. For further information, please visit:
https://www.poxelpharma.com/en_us/investors/shareholder-information/annual-general-meeting-documents.
Significant Events after the Period
NASH
- In August, the topline results for the Phase 2 trial for the
treatment of NASH (DESTINY-1) for PXL065 were announced and
indicated that the primary efficacy endpoint was met.
PXL065-treated patients achieved statistically significant
improvements in the relative decrease in liver fat content measured
by magnetic resonance imaging estimated proton density fat fraction
(MRI-PDFF) at 36-weeks for all doses. PXL065 was observed to
be safe and well tolerated with no dose-dependent increase in body
weight and no increased lower extremity edema vs. placebo. The
safety profile is consistent with reduced
PPARγ-mediated side effects vs. published results of
pioglitazone.
- On September 21, the Company announced additional DESTINY-1
results including histology. Histology findings from paired liver
biopsies showed strong improvement in fibrosis without worsening of
NASH, consistent with dose-dependent reduction of all biomarkers
related to fibrinogenesis and fibrosis risk scores. Additional
favorable trends in other histology parameters were observed.
Additional dose-dependent benefits on glucose control and indices
of insulin sensitivity were also observed.
TWYMEEG® (Imeglimin)
- As of September 1st, initial launch year restrictions for
TWYMEEG which limited new products to two weeks prescriptions have
been lifted. Due to Sumitomo Pharma's promotional activities and
efforts since launch in September 2021, TWYMEEG is very well known
among prescribers. Sumitomo Pharma’s commercial efforts continue to
leverage TWYMEEG’s potential to be used both in combination with
other treatments, such as DPP4i’s, which are the most prescribed
treatments for Japanese Type-2-Diabetes patients, and as
monotherapy.
Financing
- In August, the Company announced that it restructured its debt
with IPF, resulting in the postponement of the Q3 2022 and Q4 2022
amortization payments under the existing debt facility, and
lowering certain financial covenants until the end of January 2023.
As part of the restructuring, the Company agreed to certain
additional commitments which include the increase of the amounts
due to IPF and potential partial early repayments of the debt.
- Concurrently, the Company entered into an equity-linked
financing arrangement with IRIS for an initial gross amount of EUR
4 million, with the option, at the latest on December 31, 2022 and,
at the Company’s sole discretion, to draw a second and third
tranche of up to EUR 1 million each.
- As a result of these two agreements, the Company’s expects that
its resources will be sufficient to fund its operations and capital
expenditure requirements through at least February 2023.
First Half 2022 Financial Results (IFRS standards)
Revenue
EUR (in thousands)
H1 2022
6 months
H1 2021
6 months
Sumitomo Pharma Agreement
83
13,274
Other
-
-
Total revenues
83
13,274
The review procedures by the auditors are
still ongoing.
Poxel reported revenues of EUR 83 thousand revenue for the six
months ended June 30, 2022, as compared to EUR 13.3 million revenue
during the corresponding period in 2021.
Revenue for the first half of 2022 reflects JPY 11 million (EUR
81 thousand) of royalty revenue from Sumitomo Pharma which
represents 8% of TWYMEEG net sales in Japan. Based on the current
forecast, Poxel expects to receive 8% royalties on TWYMEEG net
sales in Japan through the Sumitomo Pharma fiscal year 2022 (April
2022 to March 2023). As part of the Merck Serono licensing
agreement, Poxel will pay Merck Serono a fixed 8% royalty based on
the net sales of Imeglimin, independent of the level of sales.
Income Statement
EUR (in thousands)
2022
6 months
2021
6 months adjusted (*)
Revenue
83
13,274
Cost of sales
(83)
Gross margin
-
13,274
Research and development expenses**
(7,882)
(14,673)
General and administrative expenses
(4,295)
(5,434)
Operating gain (loss)
(12,178)
(6,833)
Financial income (loss)
(1,223)
(1,178)
Income tax
-
-
Net income (loss)
(13,401)
(8,011)
* Change in accounting policies related to
the application of IFRIC decision dated to April 20, 2021
**Net of R&D tax credit.
The review procedures by the auditors are
still ongoing.
R&D expenses totaled EUR 7.9 million for the first half of
2022, as compared to EUR 14.7 million for the corresponding period
in 2021. They primarily reflect the clinical study costs incurred
for the Phase 2 DESTINY study evaluating PXL065 in NASH.
R&D expenses are net of the R&D Tax Credit (CIR) that
resulted in an income of EUR 0.9 million for the first half of 2022
as compared to EUR 1.6 million for the corresponding period of
2021.
General and administrative expenses totaled EUR 4.3 million for
the first half of 2022, as compared to EUR 5.4 million for the
corresponding period in 2021.
The financial loss amounted to EUR 1.2 million for the first
half of 2022, unchanged from the first half of 2021. It primarily
reflected the interests attached to the Company indebtedness.
The net result for the financial period ending June 30, 2022,
was a net loss of EUR 13.4 million, as compared to a net loss of
EUR 8.0 million in the corresponding period in 2021.
First Half 2022 Cash and Cash equivalent
EUR (in thousands)
H1 2022
Q4 2021
Cash
16,143
28,753
Cash equivalents
-
3,534
Total cash and cash
equivalents*
16,143
32,287
* Net financial debt (excluding IFRS 16
impacts and derivative debts) was EUR 17.3 million at the end of Q2
2022 as compared to EUR 2.6 million at the end of Q4 2021.
The review procedures by the auditors are
still ongoing.
As of June 30, 2022, cash and cash equivalents were EUR 16.1
million, as compared to EUR 32.3 million as of December 31,
2021.
Net financial debt (excluding IFRS16 impacts and derivative
debts) was EUR 17.3 million as of June 30, 2022, as compared to EUR
2.6 million as of December 31, 2021.
Based on:
- its cash position at June 30, 2022,
- the current development plan of the Company including 1) the
completion of its Phase 2 NASH trial for PXL065 (DESTINY-1) but
excluding 2) the initiation Phase 2a clinical proof-of-concept
(POC) biomarker studies in adrenomyeloneuropathy (AMN),
- the cash forecast for the year 2022 approved by the Board of
Directors of the Company, that does not include, as a conservative
approach, any net royalties from Imeglimin in Japan,
- a strict control of its operating expenses, and
- the amendment to the IPF debt facility with the postponement of
the Q3 2022 and Q4 2022 amortization payments until end of February
2023, as well as a full drawdown of all tranches of the
equity-linked financing arrangement with IRIS for a total amount of
EUR 6 million, before December 31, 2022.
The Company expects that its resources will be sufficient to
fund its operations and capital expenditure requirements through at
least February 2023.
The Company is actively pursuing additional financing options,
including ongoing active partnership discussions related to its
programs.
Planned Presentations and Participation at the Following
Upcoming Events
- 5th European Workshop on AMPK and AMPK-related kinases,
Glasgow, UK, September 27-29
- H.C. Wainwright 6th Annual NASH Conference (virtual), October
17, 2022
- ALD Connect 2022 Annual Meeting & Patient Learning Academy,
November 11, 2022
- Jefferies Healthcare Conference, London, UK, November 15-17,
2022
Next Financial Press Release: Third Quarter 2022
financial results and Corporate Update on November 8, 2022
About Poxel SA
Poxel is a clinical stage biopharmaceutical company
developing innovative treatments for chronic serious diseases
with metabolic pathophysiology, including non-alcoholic
steatohepatitis (NASH) and rare disorders. For the treatment of
NASH, PXL065 (deuterium-stabilized R-pioglitazone) met its
primary endpoint in a streamlined Phase 2 trial (DESTINY-1). In
rare diseases, development of PXL770, a first-in-class direct
adenosine monophosphate-activated protein kinase (AMPK) activator,
is focused on the treatment of adrenoleukodystrophy (ALD) and
autosomal dominant polycystic kidney disease (ADPKD).
TWYMEEG® (Imeglimin), Poxel’s first-in-class product that
targets mitochondrial dysfunction, is now marketed for the
treatment of type 2 diabetes in Japan by Sumitomo Pharma and Poxel
expects to receive royalties and sales-based payments. Poxel has a
strategic partnership with Sumitomo Pharma for Imeglimin in Japan,
China, and eleven other Asian countries. Listed on Euronext Paris,
Poxel is headquartered in Lyon, France, and has subsidiaries in
Boston, MA, and Tokyo, Japan.
For more information, please visit: www.poxelpharma.com
All statements other than statements of historical fact included
in this press release about future events are subject to (i) change
without notice and (ii) factors beyond the Company’s control. These
statements may include, without limitation, any statements preceded
by, followed by or including words such as “target,” “believe,”
“expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,”
“project,” “will,” “can have,” “likely,” “should,” “would,” “could”
and other words and terms of similar meaning or the negative
thereof. Forward-looking statements are subject to inherent risks
and uncertainties beyond the Company’s control that could cause the
Company’s actual results or performance to be materially different
from the expected results or performance expressed or implied by
such forward-looking statements. The Company does not endorse or is
not otherwise responsible for the content of external hyperlinks
referred to in this press release.
1 For more information on Orphan Drug Designation, see:
https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220920006290/en/
Investor relations / Media
Aurélie Bozza Investor Relations & Communication Senior
Director aurelie.bozza@poxelpharma.com +33 6 99 81 08 36
Elizabeth Woo Senior Vice President, Investor Relations &
Communication elizabeth.woo@poxelpharma.com
NewCap Emmanuel Huynh or Arthur Rouillé poxel@newcap.eu +33 1 44
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