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  • Letter to the Shareholders

    The effort and investment that have gone into our proprietary research and development activities over the past several years paid off handsomely in 2013 and led to licensing agreements with Celgene and GlaxoSmithKline.

  • Product Pipeline

    The advances in our proprietary pipeline and the partnered pipeline were the main drivers of the Company’s value in 2013. With gantenerumab, bimagrumab, and guselkumab, there are a number of candidates in our pipeline that address pressing medical problems.

  • Financial Results

    The progress of our product pipeline has also shaped our financial performance in 2013. The alliances with Celgene and GlaxoSmithKline, the sale of AbD Serotec, and the capital increase have all contributed to the rise in our liquidity to approximately €400 million.

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Engineering the medicines
of tomorrow

MorphoSys has built a reputation in the pharmaceutical industry as the
partner of choice in the search for new antibody drugs. A number of very
successful research alliances and proprietary development activities have
led to more than 20 therapeutic antibodies in clinical trials. We will
build on this success and continue to develop and expand our
proprietary portfolio.

We believe that successful drug development is not a matter of chance,
but based on a profound understanding of disease biology, extensive
experience in the selection, characterization, and development of
compound candidates, and also on excellent technologies.

We have applied our skills and knowledge to build a proprietary portfolio
of drug candidates and will concentrate even more on proprietary product
development in the future. In 2013, we gained two prestigious partners,
Celgene and GlaxoSmithKline, for the further development of two
candidates from our portfolio.

Engineering

The foundation for the success or failure of a therapeutic
project is, in many cases, set at the very start. Correctly
+ interpreting the biology of the disease and selecting the
best approach for treatment, form the scientific
prerequisite. But even with the right treatment approach,
it’s the quality of the compound that is often decisive
for the success or failure of the project.

MorphoSys’s scientists are antibody experts. Our core
capabilities, however, can be applied to a much broader
range of molecules and drug classes. With more than
20 years’ + experience in antibody drug discovery and
development, MorphoSys can give therapeutic
projects a head start.

+ Technology is an essential part of this process. Smarter
tools provide faster, more flexible, and, ultimately,
better-quality access to drug candidates. With its platform
technologies Ylanthia and Slonomics, MorphoSys is pushing
the boundaries of antibody generation in its pursuit to
generate antibody drugs with superior development
potential and product characteristics.

100.000.000.000.
Antibodies

Both the HuCAL library and the Ylanthia library include the
blueprints for several billion different antibodies and form an
immense range of therapeutically relevant antibodies thanks to
their additional optimization opportunities. The key challenge
is to filter out the best antibody from this extensive variety of
possibilities and then develop this antibody thoroughly,
proactively, and rapidly.

  • MorphoSys’s Ylanthia technology is setting new standards in the
    generation of therapeutic antibodies. Its structural diversity
    allows for the selection of antibodies to target molecules that
    were previously not addressable and to achieve unique coverage
    of all possible therapeutic approaches.

  • By means of the HuCAL technology, MorphoSys has been able
    to establish what is currently the most successful antibody
    library in the biopharmaceutical industry.

  • Slonomics facilitates pinpoint modification of the
    gene sequence and the production of precisely defined
    gene libraries.

  • MorphoSys has gained far-reaching experience in the selection and
    use of significant in vivo models in the field of inflammatory diseases
    and oncology through its proprietary programs, and possesses
    expertise in an even broader spectrum of disease through
    its partnerships.

  • To identify the most suitable antibody, it is important to determine
    its biophysical properties and, in particular, its functional
    characterization. Can the antibody kill cancer cells in laboratory
    tests? Is the antibody able to penetrate tumor tissue?

Numerous criteria for an antibody molecule may
be optimized to improve the development potential
in each area of disease and raise the entire
project’s probability of success.

Aggregation behavior

How stable does the antibody remain in the solution? Could it be stored in this manner for a longer period of time?

Solubility

How much of the antibody dissolves in predetermined quantities of liquid? Is subcutaneous admini­stration possible?

Cross-reactivity

Does the antibody also recognize the target molecule in other species?

Binding strength

How strongly does the antibody bind its target molecule?

Specificity

To what extent does the antibody specifically recognize its target molecule? Does it bind to other structures in the body as well?

Characteristics of production

How high are the yields when the antibody is produced on a larger scale?

MorphoSys has an 80 % success rate in
selecting target molecules that result
in lead candidates.

MEDICINES

Due to their unique properties, therapeutic antibodies are
still one of the fastest growing drug classes in human
medicine. Until a compound is approved as a medication on
the market, it must withstand a highly critical examination
in clinical trials and fulfill numerous requirements and
governmental requests. MorphoSys has acquired extensive
knowledge in order to plan and perform the clinical trials
of its proprietary programs internationally.

All disciplines and teams within the MorphoSys Group
work closely together to facilitate the rapid advancement of
programs from the point at which the compound was
discovered through to clinical development. Additionally,
the Company has set the course for its evolution into a
commercial organization in the medium term.

  • Antibody technologies: the industry’s leading antibody libraries and supplementary systems
  • Antibody screening and selection: different strategies for identifying the most suitable antibodies
  • Characterization of the antibody, assay development, and biological expertise: every compound is put to the test
  • Antibody production and CMC management: development of the best production method and route of administration
  • Preclinical development: evaluation of efficacy and safety in animal models

Clinical development: testing the
compound on humans

Clinical Phase 1

Trials with a small number of healthy probands or patients. Initial findings with regard to dosage, safety, and tolerability

16 ongoing clinical trials in phase 1 with MorphoSys antibodies

Clinical Phase 2

Trials with a limited number of patients. Evaluation of the efficacy of the product for specific indications. Determination of the optimal dosage, and the identification of risks and side effects

24 ongoing clinical trials in phase 2 with MorphoSys antibodies

Clinical Phase 3

Administration of the product to a larger number of patients over a longer period of time in order to assess the underlying risk-benefit ratio. Usually, two or more phase 3 studies are necessary to achieve regulatory approval.

4 ongoing clinical trials in phase 3 with MorphoSys antibodies

  • To date, MorphoSys has initiated and successfully led
    clinical trials at more than 40 clinical centers in 12 countries.

  • MorphoSys possesses profound experience in
    the area of manufacturing processes and in the
    execution of formulation and stability studies.

  • MorphoSys has developed intravenous and subcutaneous
    routes of administration in the context of the MOR103 project.

  • As part of its MOR103 and MOR202 programs,
    MorphoSys has successfully carried out various
    toxicology and preclinical efficacy studies.

Tomorrow

In many ways, future drugs must be better than the
preparations that are currently available. Clearly differentiated
compounds that stand out from the mass of approaches
and break new ground will form the key value drivers in the
pharmaceutical industry. MorphoSys’s technologies and the
expertise of its people support the search for drug
candidates with unique properties.

Cell membrane
Target moleculeCause of disease

The interaction of receptors on the cell surface and in the
cell membrane is a natural process that can, however, cause
several diseases, including cancer, when brought out of
balance. A therapeutic antibody that binds to the best-possible
region, usually a unique epitope on the receptor protein, is
able to alter the course of the disease significantly.

Unique starting points: The MorphoSys Ylanthia antibody library has
all the necessary prerequisites for selecting antibodies against all
possible starting points of disease-relevant target
molecules due to its structural range and diversity. If one can
succeed in exhausting all of the possibilities for binding promising
target molecules, this can result in unique product opportunities
and compound candidates that can intervene at the crucial
points in a disease process.

Cell membrane
Target moleculeGPCR molecule

So-called GPCR molecules represent the largest class of
disease-relevant target molecules for the pharmaceutical
compounds currently on the market. GPCRs are found in
every cell of the body and transmit signals from the cellular
environment through the membrane into the cell interior to
downstream signaling cascades and allow communication
between cells and organs.

Previously unattainable target molecules: As a result
of the powerful technologies of MorphoSys and its partners,
therapeutic approaches are now becoming possible with
antibodies that previously could not be pursued or only pursued
to a very limited extent. MorphoSys believes this could lead
to a new wave of future antibody drugs.

The future
of healthcare will be
built on truly differentiated drugs.

Simon Moroney, CEO of MorphoSys AG
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