Santhera Enters into License Agreement with Chiesi Group for Raxone in LHON Valued at up to CHF 105 Million
Santhera will hold a conference call today, May 23, 2019, at 13:00 CET, 12:00 GMT, 07:00 EST. Details at
the end of statement.
? Transaction allows Santhera to advance its long-term growth strategy by focusing on the
development of its clinical-stage neuromuscular and pulmonary programs
? Deal includes upfront cash payment of CHF 50 million (EUR 44 million) which is due after closing
of the transaction
Pratteln, Switzerland, May 23, 2019 ? Santhera Pharmaceuticals (SIX: SANN) announces that it has
entered into an exclusive license agreement with Chiesi Farmaceutici, an international research-focused healthcare group (Chiesi Group), under which Chiesi Group will in-license Raxone? for the
treatment of LHON for a total consideration of up to CHF 105 million (EUR 93 million), comprising an
upfront cash payment of CHF 50 million (EUR 44 million) and near- to mid-term sales milestone
payments of up to CHF 55 million (EUR 49 million).
Chiesi Group is in-licensing the rights to Raxone in LHON and all other ophthalmological indications
worldwide except the US and Canada, where Santhera retains rights. In a second step, following the
completion of certain reimbursement and post-regulatory commitments on the part of Santhera, Chiesi
Group has the option to fully acquire Santhera?s Raxone business. The transaction enables Santhera to
focus on the business areas core to its long-term growth strategy by advancing its clinical-stage
neuromuscular and pulmonary programs.
?Since the approval in 2015 we have successfully launched Raxone in Europe and reported strong yearon-year sales increases. In the past eighteen months we also strategically diversified our pipeline. This
license agreement for Raxone in LHON, our sole neuro-ophthalmology asset, will allow us to create
significant value for our shareholders as it provides financial resources enabling us to focus on delivering
innovation to patients with neuromuscular and pulmonary diseases of great unmet medical need,? said
Thomas Meier, PhD, Chief Executive Officer of Santhera.
?We have built a strong portfolio in neur
Santhera Enters into License Agreement with Chiesi Group for Raxone?
in LHON Valued at up to CHF 105 Million
May 23, 2019 / Page 2 of 4
?Chiesi Group has a strong commitment in the orphan diseases area, bringing innovative treatments
forward to patients affected by rare or ultra-rare disorders and improving the quality of life of people
with rare diseases is one of the most important goals. This strategic partnership reinforces this
commitment with a therapeutic offer for patients affected by LHON, a neurodegenerative disease
targeting the optic nerve and characterized by sudden vision loss?, commented Ugo Di Francesco, Chief
Executive Officer, Chiesi Group.
Santhera will license and transfer to Chiesi Group all of its rights for the development, commercialization
and distribution of Raxone for the treatment of LHON and any other potential ophthalmological
indications for all territories worldwide except the US and Canada.
The closing of the transaction is subject to customary approvals and is currently expected to occur in the
third quarter 2019. As of that date, the advance payment of CHF 50 million (EUR 44 million) will also
become due.
In an interim phase Santhera will continue to provide medical, technical, logistical and scientific support
with regard to ongoing market authorization activities and/or market access undertakings for several
months. In this context, the parties agreed that Santhera will continue to commercialize Raxone for LHON
in France until ongoing pricing and reimbursement negotiations have been finalized. Santhera will also
be responsible to complete the ongoing Post Authorization Measures, expected by 2021, in conjunction
with the centralized European Marketing Authorization granted in 2015.
Following the transaction, Santhera will focus its resources on advancing its late-stage clinical pipeline of
products for the treatment of neuromuscular and pulmonary diseases. In particular, Santhera intends to
fully exploit its strategic position in DMD therapy development, with ongoing programs to help all DMD
patients irrespective of causative mutations, disease stage or age. Addressing the needs of advanced
DMD patients experiencing respiratory dysfunction, Santhera plans to submit a conditional marketing
authorization application (MAA) for Puldysa? (idebenone) to the EMA. Pivotal study data for vamorolone
as treatment of younger, still ambulatory DMD patients is expected in 2020. In addition, Santhera?s
expertise in the respiratory area through its activities in the DMD space led to the acquisition of the
promising clinical-stage asset POL6014, which has the potential to treat cystic fibrosis (CF) and other
pulmonary diseases. Other pipeline elements include strategies to treat congenital muscular dystrophies.
Centerview Partners acted as exclusive financial advisor to Santhera for this license agreement.
Santhera Conference Call
Santhera will hold a conference call today, May 23, 2019 at 13:00 CET, 12:00 GMT, 07:00 EST to discuss
the license agreement with Chiesi Group for Raxone. Participants are invited to dial one of the following
numbers about 10 minutes before the call is due to start (no code required):
Europe: +41 58 310 50 00
UK: +44 207 107 0613
USA: +1 631 570 5613
Santhera Enters into License Agreement with Chiesi Group for Raxone?
in LHON Valued at up to CHF 105 Million
May 23, 2019 / Page 3 of 4
About Leber?s Hereditary Optic Neuropathy and the Therapeutic Use of Raxone
Leber?s hereditary optic neuropathy (LHON) is a heritable genetic disease causing profound vision loss
and blindness. The disease presents predominantly in young, otherwise healthy adult males as rapid,
painless loss of central vision, usually leading to permanent bilateral blindness within a few months of
the onset of symptoms. About 95% of patients harbor one of three pathogenic mutations of the
mitochondrial DNA, which cause a defect in the complex I subunit of the mitochondrial respiratory chain.
This defect leads to decreased cellular energy (ATP) production, increased reactive oxygen species (ROS)
production and retinal ganglion cell dysfunction, which cause progressive loss of visual acuity and
blindness.
Raxone (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme
NAD(P)H:quinone oxidoreductase (NQO1), circumvents the complex I defect, reduces and scavenges
ROS, restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity.
Current data demonstrate that up to 50% of patients benefit from treatment and are protected from
progression of visual acuity loss or experience a clinically relevant recovery of visual acuity.
Raxone for the treatment of LHON was granted orphan drug status in the EU, US, Switzerland and South
Korea.
About Chiesi Group
Based in Parma, Italy, Chiesi Farmaceutici is an international research-oriented group with over 80 years?
experience in the pharmaceutical sector, and is present in 27 countries. The group researches, develops
and commercialises innovative medicines in the respiratory disease, special care and rare disease
therapeutic areas. The Group?s Research & Development centre is based in Parma (Italy) and integrated
with 6 other important research and development groups in France, the USA, the UK and Sweden, to
promote its pre-clinical, clinical and registration programmes. The Group employs around 5,700 people.
www.chiesi.com.
About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the
development and commercialization of innovative medicines for rare and other diseases with high unmet
medical needs. The portfolio comprises clinical stage and marketed treatments for neuroophthalmologic, neuromuscular and pulmonary diseases. Santhera's Raxone? (idebenone) is authorized
in the European Union, Norway, Iceland, Liechtenstein, Israel and Serbia for the treatment of Leber's
hereditary optic neuropathy (LHON) and is currently commercialized in more than 20 countries. For
further information, please visit www.santhera.com.
Raxone? and Puldysa? are trademarks of Santhera Pharmaceuticals.
For further information please contact:
public-relations@santhera.com or
Eva Kalias, Head External Communications
Phone: +41 79 875 27 80
eva.kalias@santhera.com
Santhera Enters into License Agreement with Chiesi Group for Raxone?
in LHON Valued at up to CHF 105 Million
May 23, 2019 / Page 4 of 4
For Investors:
investor-relations@santhera.com or
Christoph Rentsch, Chief Financial Officer Hans Vitzthum, LifeSci Advisors
Europe: +41 61 906 89 65 US: +1 212 915 2568
christoph.rentsch@santhera.com hans@lifesciadvisors.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.