Santhera Announces First Half-Year 2021 Financial Results and
Updates on Corporate Progress
Ad hoc announcement pursuant to Art. 53 LR
- Cash and cash equivalents
of CHF 8.0 million (as of June 30, 2021)
- Net revenue CHF 4.5
million (H1-2020: CHF 7.8 million)
- Net result for period of
CHF -20.5 million (H1-2020:
CHF -31.8 million)
Pratteln, Switzerland, October 15,
2021 – Santhera Pharmaceuticals (SIX: SANN) announces the Company’s
financial results for the first half-year ended June 30, 2021,
and provides an update on corporate progress.
“Our achievements in the first half of 2021 have
moved us significantly closer to accomplishing key goals. The
highlights in the period under review were undoubtedly the positive
results of the Phase 2b VISION-DMD study at 24 weeks with
vamorolone compared to placebo in Duchenne muscular dystrophy,
paving the way for an NDA submission in the U.S. We are equally
excited about the favorable tolerability profile of vamorolone
versus prednisone, potentially addressing the high medical need for
steroid efficacy with a safety profile differentiated from steroids
on some key clinically meaningful parameters. On the financing
side, we successfully restructured the Company’s balance sheet and
secured adequate funding to take us to mid-2022 or beyond key
upcoming milestones,” said Dario Eklund, Chief Executive
Officer of Santhera. “These successes are crucial for both
patients who are in great need of a better tolerated steroid
therapy suitable for chronic treatment and for Santhera as they lay
the foundation for renewed future growth. We are grateful for the
continued support of patients and their caregivers, health care
staff and investors, which will allow us to move forward with our
strategy and execute our plans and ambitions.”
OPERATIONAL HIGHLIGHTS
Recent developments
- Statistically highly significant
24-week results across multiple endpoints with vamorolone in
pivotal Phase 2b VISION-DMD study
- Long-term treatment data with
vamorolone demonstrating maintenance of effect over 2.5 years
- Positive results with lonodelestat
in Phase 1b cystic fibrosis (CF) trial
- Primary endpoint met in Phase 4
LEROS trial with Raxone® in Leber’s hereditary optic neuropathy
(LHON)
- Restructuring activities (announced
in October 2020) completed
- Completion of 2017/22 convertible
bond exchange offer and issuance of a new 2021/24 convertible
bond
- Share capital increases implemented
as a basis to enable additional financing to secure operations,
fund prelaunch activities for vamorolone and support advancement of
pipeline
- Completion of financing to provide
up to CHF 42 million net of fees and expenses to fund
operations and current debt
obligations
Upcoming milestones
- Q4-2021: Completion of vamorolone
48-week VISION-DMD study providing additional safety data
- Q4-2021: Conclusion of Raxone PAMs
(post-authorization measures)
- Q1-2022: NDA (new drug application)
filing in the US for vamorolone in DMD
- Q2-2022: Regulatory submission in
Europe for vamorolone in DMD
Vamorolone—nearing regulatory submission
to the US FDA In June 2021, Santhera and ReveraGen
BioPharma announced positive 24-week results from the VISION-DMD
study, a pivotal Phase 2b study comparing vamorolone (2 or
6 mg/kg/day) to placebo and prednisone (0.75 mg/kg/day)
in the treatment of Duchenne muscular dystrophy (DMD). The results
demonstrated robust efficacy as the study met its primary endpoint
of superiority in change of time from supine positioning to
standing (TTSTAND) velocity with vamorolone 6 mg/kg/day versus
placebo (p=0.002) with a treatment difference of 0.06 rises/second
[95% CI: 0.02–0.10] from baseline. Likewise, the study also
demonstrated superiority of vamorolone versus placebo across
multiple secondary endpoints and established the efficacy of
vamorolone at 2 and 6 mg/kg/day. Vamorolone is the only steroid to
have shown efficacy for two doses across a three-fold dose range,
allowing physicians for the first time to tailor treatment to the
individual.
Already in April 2021, Santhera and ReveraGen
announced new clinical data of 2.5-year treatment outcome with
vamorolone in patients with Duchenne muscular dystrophy (DMD).
These Phase 2a long-term treatment data demonstrated a maintenance
of treatment effect, equivalent to a delay of about two years in
decline for time to stand (TTSTAND) velocity, and confirmed safety
and tolerability benefits of vamorolone over the 2.5-year follow up
period. In comparison to reports from clinical trials with other
corticosteroids, long-term treatment with vamorolone resulted in
fewer of the side effects that are typically observed with those
drugs.
Based on clinical trial results, including
long-term safety data up to 30 months, vamorolone at doses up to
6 mg/kg/day was generally well-tolerated. Vamorolone treatment
has been shown to preserve height trajectory and had a
significantly lower adverse impact on measures of bone health and
behavior changes compared to prednisone.
On the basis of the positive 24-week efficacy
results from the pivotal VISION-DMD study and the demonstration of
long-term benefits of vamorolone, Santhera is preparing for
submission of a New Drug Application (NDA) in the US in Q1-2022,
for which fast track designation was granted by the FDA.
In Q4-2021, the 48-week data readout delivering
supplementary safety and tolerability data for vamorolone is
expected upon completion of the VISION-DMD study. During the second
period of the study, where all participants receive vamorolone
treatment on either of the two dose levels, additional longer-term
tolerability data is captured. The 48-week data will support the
submission of a marketing authorization application in Europe in
Q2-2022.
In September 2021, the FDA awarded a
USD 1.2 million grant to ReveraGen under their “Clinical
Studies of Orphan Products Addressing Unmet Needs of Rare Diseases
(R01)” grants program to initiate a clinical trial of vamorolone in
adults and children with Becker muscular dystrophy (BMD), a
progressive muscle wasting disease similar to DMD, but usually
milder. The mechanisms of actions, which provide the basis of
vamorolone’s efficacy as demonstrated in the pivotal VISION-DMD
study in the more severe DMD, are hypothesized to be relevant to
BMD too.
Santhera intends to commercialize vamorolone for
the treatment of DMD through its own organization in the United
States and main markets in Europe, and is seeking collaborations
outside those regions for DMD and for additional indications
worldwide. Santhera estimates the peak product sales potential for
vamorolone in the indication DMD alone to be in excess of USD 500
million in the US and the largest five European countries
combined.
Lonodelestat—positive results in early
phase cystic fibrosis trial In March 2021, Santhera
announced positive results of a Phase 1b study with lonodelestat, a
potent and selective peptide inhibitor of human neutrophil elastase
(hNE) in development to treat cystic fibrosis (CF). Neutrophil
elastase is an enzyme associated with tissue inflammation, leading
to degradation of the lung tissue in cystic fibrosis and several
other acute and chronic inflammatory conditions of the lung where
neutrophils play a prominent role in the disease process. The
double-blind, placebo-controlled multiple ascending dose Phase 1b
study in patients with CF established a safe dose regimen and
provided promising data on the safety of lonodelestat. Furthermore,
the study demonstrated that lonodelestat is well tolerated at 40 mg
and 80 mg daily doses and achieves the desired effect of near
complete inhibition of elastase without any drug/metabolite
accumulation.
The results from the safety analyses and the
confirmed effect on the hNE biomarker by lonodelestat are very
encouraging for further development in CF and other inflammatory
lung diseases. On this basis, Santhera will now be refining the
further clinical development program to advance lonodelestat for
the treatment of CF and potentially for other inflammatory
pulmonary conditions, whether acute or chronic.
Post-authorization measures (PAMs) with
Raxone successful and nearing completion In June 2021,
Santhera announced positive topline results from its long-term
Phase 4 LEROS study with Raxone (idebenone) in the treatment of
LHON. The primary endpoint, the proportion of eyes with clinically
relevant benefit after 12 months treatment with Raxone versus
untreated patients from an external natural history control group,
was met with high statistical significance (p=0.002). The efficacy
data confirm and extend previous findings which demonstrated that
Raxone can prevent further vision loss and promote recovery of
vision in LHON patients.
The study, which was designed with guidance and
approval from the European Medicines Agency (EMA), was part of a
post-authorization commitment. The strong evidence of efficacy is
expected to support market access in countries where this is not
yet the case, allowing patients who have no therapeutic alternative
to benefit from treatment with Raxone.
Santhera holds the EU marketing authorization
for Raxone (idebenone) and out-licensed rights to the product
outside North America and France for the treatment of LHON to
Chiesi Group. Santhera is still commercializing Raxone for LHON in
France in a transitional phase and, as previously communicated, is
supplying the product free of charge since August 2021 following
its removal from the list of reimbursed products and while
reimbursement discussions are ongoing. The Company is entitled to
contingent variable near- to mid-term milestone payments from
Chiesi Group of up to EUR 49 million subject to the
achievement of certain commercial milestones for Raxone.
Corporate restructuring completed and
organization realigned to future priorities By the end of
March 2021, Santhera completed an organizational restructuring
which reduced costs and prioritized the Company’s resources for
vamorolone as a consequence of the termination of the Puldysa®
program in late 2020. The result is a lower cost base and
streamlined organization, focused on bringing vamorolone to
patients. In doing so, the core team will leverage its know-how in
the DMD space, regulatory experience with the EMA and FDA, strong
relationships with key clinical experts and the patient community
as well as its proven track record of successfully commercializing
a rare disease product.
On the back of the positive VISION-DMD study
results, paving the way for an NDA submission to the US FDA, the
Company has defined operational and organizational measures to
allow for a successful first launch of vamorolone in the US which
is expected earliest at the beginning of 2023. Attraction of key
talents with a focus on the US market for pre-commercialization
activities will begin as soon as additional funding has become
available.
Prioritization of the development
pipeline Given the resource limitations, and in parallel
to the alignment of its organizational structure, Santhera has
conducted a pipeline review and prioritization. Going forward, the
Company will focus on its lead clinical-stage projects vamorolone
in DMD and lonodelestat in CF and has decided to abandon the
further development of omigapil. In parallel, Santhera is
proactively pursuing collaborations with partners to assess and
exploit the potential of both clinical stage compounds in other
disease areas, beyond DMD and CF, as well as for its undertakings
in gene therapy for congenital muscular dystrophies (CMD).
Capital restructuring and share capital
increases implemented to enable adequate funding to execute
strategy In the first half-year 2021, the Company
implemented various measures to strengthen the capital structure of
Santhera and to secure sufficient flexibility to continue
operations and advance the pipeline as anticipated.
On May 4, Santhera announced completion of the
exchange offer in respect of its CHF 60 Million Convertible
Bonds due 2022 (SIX: SAN17) and the issuance of CHF 30,270,375
Senior Unsecured Convertible Bonds due 2024 (SIX: SAN21). The
restructuring of the 2017/22 Bonds enabled Santhera to proceed with
raising additional financing and was therefore crucial to preserve
the Company as a going concern until after such subsequent
financing.
Santhera’s shareholders gave consent to various
capital increases in the first half-year 2021. At the Extraordinary
General Meeting (EGM) held on March 18, the Board of Directors
(BoD) proposed to shareholders the authorization and issuance of
the shares for the upsized financing from a fund managed by
Highbridge Capital Management, LLC, and the restructuring of its
CHF 60 million Senior Unsecured Convertible Bonds. At the
Annual General Meeting (AGM) held on June 22, the BoD proposed
various capital increases to Santhera’s shareholders to give the
Company sufficient flexibility to raise additional capital to fund
ongoing development activities, increase pre-commercialization
activities and expand the organization in view of a US market
launch of vamorolone as early as the beginning of 2023, subject to
approval by the US FDA. Santhera’s shareholders approved all
motions by the BoD at both the EGM and the AGM, allowing the
Company to proceed with its strategy and plans as foreseen.
On September 20, 2021 the Company announced
a financing of up to CHF 45 million gross or CHF 42
million net of fees and expenses to provide funding through to
mid-2022 past the NDA filing planned for Q1-2022. The financing
comprised CHF 20 million in equity, CHF 15 million in a
new 2021/24 convertible bond to be used for settlement of the
CHF 15 million nominal value outstanding on the 2017/22
convertible bond maturing February 2022, together with a
CHF 10 million in new senior secured exchangeable notes. A
first tranche of CHF 2 million may be drawn after closing, on
October 14, 2021, subject to certain customary conditions. A
further tranche of CHF 5 million may be drawn if and when the
FDA supports an NDA for vamorolone in DMD in the United States upon
which a USD 5 million milestone payment to licensor ReveraGen
becomes due. The remaining tranche of CHF 3 million is
available subject to investor consent. The maturity of the term
loan will be May 2024.
However, considering the Company’s current
planned strategy for the next 12 months, additional funds will be
required to fund operations and material uncertainties remain as to
the Company’s ability to continue as a going concern until
June 30, 2022.
KEY FINANCIALS AS OF JUNE 30, 2021
- Cash and cash equivalents of
CHF 8.0 million
- Net revenue CHF 4.5 million
(H1-2020: CHF 7.8 million)
- Net result for period of
CHF -20.5 million (H1-2020: CHF -31.8 million)
Financial liquidity as of June 30,
2021As of June 30, 2021, the Company had cash and
cash equivalents of CHF 8.0 million compared to CHF 12.4
million as of December 31, 2020. The decrease was primarily
due to support of ongoing development and completion of
organizational restructuring activities.
Net cash used in operating activities was
CHF 18.6 million for the six months ended June 30, 2021,
compared to CHF 19.8 million for the six months ended
June 30, 2020.
Following financing activities, including the
convertible bond 2017/22 exchange for 2021/2024 convertible bond
overall net equity at June 30, 2021 increased to CHF 8.4 million
from a deficit of CHF 6.4 million as at December 31, 2020.
Financial results for the six months
ended June 30, 2021For the six months ended June 30,
2021, the Company recorded a net loss of CHF 20.5 million, or
CHF 0.92 per share, compared to a net loss of CHF 31.8
million or CHF 2.78 per share for the six months ended
June 30, 2020.
Total revenue was CHF 4.5 million and
CHF 7.8 million for the six months ended June 30, 2021,
and June 30, 2020, respectively. The majority of this revenue
reflects sales of Raxone for the treatment of LHON in France where
Santhera continues to supply the product following the outlicensing
and transfer to Chiesi Group in 2019. The decrease in revenues is
mainly attributable to a CHF 2.0 million adjustment to defer
revenues recorded in the first half-year 2021 due to uncertainties
around pricing and reimbursement in France, as well as an agreement
with the regulatory authorities in France to supply Raxone free of
charge from August 2021 while reimbursement discussions are
ongoing.
Development expenses were CHF 13.6 million
and CHF 17.7 million for the six months ended June 30,
2021, and June 30, 2020, respectively. The decrease in
expenses was primarily due to lower contract research organization
expenses and other third-party clinical trial expenses following
the termination of the Puldysa Phase 3 SIDEROS study, offset by
increased expenses to support the development of vamorolone to the
recently announced 24-week, in addition to a reduction in staff
costs following organizational restructuring.
Marketing and sales expenses were CHF 2.0
million and CHF 6.8 million for the six months ended
June 30, 2021 and June 30, 2020 respectively. The
decrease was primarily a result of the ceasing of Puldysa
activities following the termination of the program announced in
October 2020. Ongoing expenses relate to pre-commercialization
activities for vamorolone and meeting ongoing obligations in
relation to Raxone out-licensed to Chiesi Group.
General and administrative expenses were
CHF 6.3 million and CHF 7.2 million for the six months
ended June 30, 2021 and June 30, 2020 respectively, the
decrease was primarily related to the organization restructuring
announced in October 2020.
Half-year
ReportThe Santhera Half-year Report 2021 is
available for download on the Company’s website at
www.santhera.com/investors-and-media/investor-toolbox/financial-reports.
2021
Half-year Financial
Information
Santhera’s 2021 Half-year Report see
www.santhera.com/investors-and-media/investor-toolbox/financial-reports.
Condensed consolidated income statement(reviewed,
IFRS, for half-year ended June 30, in CHF thousands) |
1H-2021 |
1H-2020 |
Net sales |
2,853 |
6,133 |
Net sales to
licensing partner |
1,639 |
1,642 |
Revenue from contracts with customers |
4,492 |
7,775 |
Cost of goods sold (of which amortization intangible assets:
1H-2021 -1,519 / 1H-2020 -1,519) |
-2,031 |
-2,114 |
Development |
-13,592 |
-17,688 |
Marketing and
sales |
-2,008 |
-6,766 |
General and
administrative |
-6,307 |
-7,209 |
Operating expenses |
-21,938 |
-31,911 |
Operating result |
-19,477 |
-25,893 |
Financial result |
-389 |
-5,573 |
Income
taxes |
-653 |
-361 |
Net result |
-20,519 |
-31,827 |
Basic and diluted loss per share (in CHF) |
-0.92 |
-2.78 |
Condensed consolidated balance sheet (IFRS, in CHF
thousands) |
June 30,
2021(reviewed) |
Dec 31, 2020(audited) |
Cash and cash equivalents |
7,991 |
12,411 |
Other current
assets |
3,659 |
5,312 |
Noncurrent
assets |
68,567 |
70,964 |
Total assets |
80,217 |
88,687 |
Equity |
8,353 |
-6,354 |
Noncurrent
liabilities |
27,210 |
65,972 |
Current
liabilities |
44,654 |
29,069 |
Total equity and liabilities |
80,217 |
88,687 |
Condensed consolidated cash flow
statement(reviewed, IFRS, for half-year ended
June 30, in CHF thousands) |
1H-2021 |
1H-2020 |
Cash flow (used) in operating activities |
-18,607 |
-19,795 |
Cash flow from
investing activities |
-75 |
1,506 |
Cash flow from
financing activities |
14,276 |
6,405 |
Cash and cash
equivalents at January 1 |
12,411 |
31,358 |
Cash and cash
equivalents at June 30 |
7,991 |
19,353 |
Net change in cash and cash equivalents |
-4,420 |
-12,005 |
Share capital(number of shares with par value of
CHF 1) |
June 30,
2021(reviewed) |
Dec 31, 2020(audited) |
Shares issued |
31,303,512 |
19,429,696 |
Conditional
capital for equity rights |
5,537,052 |
687,052 |
Conditional
capital for convertible rights |
6,304,703 |
2,500,000 |
Authorized capital |
14,381,755 |
2,080,709 |
About SantheraSanthera
Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical
company focused on the development and commercialization of
innovative medicines for rare neuromuscular and pulmonary diseases
with high unmet medical need. Santhera has an exclusive license for
all indications worldwide to vamorolone, a first-in-class
dissociative steroid with novel mode of action, which was
investigated in a pivotal study in patients with DMD as an
alternative to standard corticosteroids. The clinical stage
pipeline also includes lonodelestat (POL6014) to treat cystic
fibrosis (CF) and other neutrophilic pulmonary diseases as well as
an exploratory gene therapy approach targeting congenital muscular
dystrophies. Santhera out-licensed rights to its first approved
product, Raxone® (idebenone), outside North America and France for
the treatment of Leber's hereditary optic neuropathy (LHON) to
Chiesi Group. For further information, please visit
www.santhera.com.
Raxone® is a trademark of Santhera
Pharmaceuticals.
For further information please
contact: public-relations@santhera.com orEva Kalias, Head
External CommunicationsPhone: +41 79 875 27
80eva.kalias@santhera.com
Disclaimer / Forward-looking
statements This communication does not constitute an offer
or invitation to subscribe for or purchase any securities of
Santhera Pharmaceuticals Holding AG. This publication may contain
certain forward-looking statements concerning the Company and its
business. Such statements involve certain risks, uncertainties and
other factors which could cause the actual results, financial
condition, performance or achievements of the Company to be
materially different from those expressed or implied by such
statements. Readers should therefore not place undue reliance on
these statements, particularly not in connection with any contract
or investment decision. The Company disclaims any obligation to
update these forward-looking statements.
# # #
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