Merck Grants Exclusive License to Vertex for Two DNA Damage Response Inhibitors

Darmstadt, Germany (ots/PRNewswire) -

Not intended for UK- or US-based media

- Exclusive license grants Vertex one clinical and one pre-clinical
DNA-PK inhibitor for use in gene-editing applications in six
specific genetic disease areas
- Merck retains the rights to both compounds in all other disease
areas, including oncology
- Merck retains the rights to utilize and license this technology in
gene editing outside of the six specified disease areas

Merck, a leading science and technology company, today announced
that it has entered into an exclusive licensing agreement with Vertex
Pharmaceuticals Incorporated, Boston, USA (NASDAQ: VRTX), for two
DNA-dependent protein kinase (DNA-PK) inhibitors - M9831 (formerly
known as VX-984) and an additional pre-clinical compound - in the
field of gene editing for six specific genetic disease indications.
Merck will receive an upfront payment in addition to milestones and
royalties on future net sales and retains the rights to both assets
in all other disease areas, including oncology, with the ability to
develop both these compounds in-house, or to license them to future
partners in the gene editing field. Vertex has the option to add
indications to the license grant. Both molecules were acquired in a
licensing agreement from Vertex in 2017, and are part of Merck's
broad portfolio of DNA Damage Response (DDR) inhibitors.

"This transaction illustrates our determination to maximize value
creation from our pipeline," said Belén Garijo, Member of the
Executive Board and CEO Healthcare, Merck. "We are rapidly advancing
our leading-edge DDR portfolio in oncology and are delighted to see
the potential benefit of DNA-PK in genetic diseases through the
enhancement of CRISPR/Cas9-mediated gene editing."

Merck is investing significant resources into the promising area
of DDR, and has considerable expertise and experience in developing
DDR molecules, with the objective of becoming one of the leading
players in this therapeutic area. The company is currently
investigating four DDR molecules, including two ATR inhibitors, an
ATM inhibitor and an investigational small-molecule of DNA-PK. DNA-PK
is a key enzyme that could potentially enhance the efficacy of many
commonly used DNA-damaging agents such as radiotherapy and
chemotherapy.

Pre-clinical studies have shown that DNA-PK inhibitors can enhance
CRISPR/Cas9-mediated gene editing. CRISPR/Cas9 is a technology used
to modify genetic sequences and is being investigated for the
treatment of various genetic disorders. This collaboration licenses
two compounds to study the potential DNA-PK-inhibitor-mediated
enhancement of gene editing for the treatment of six genetic diseases
included in the license grant to Vertex.

About Vertex

Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now
located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to work,
including being named to Science magazine's Top Employers in the life
sciences ranking for nine years in a row.

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About Merck

Merck, a vibrant science and technology company, operates across
healthcare, life science and performance materials. Around 51,000
employees work to make a positive difference to millions of people's
lives every day by creating more joyful and sustainable ways to live.
From advancing gene editing technologies and discovering unique ways
to treat the most challenging diseases to enabling the intelligence
of devices - Merck is everywhere. In 2017, Merck generated sales of
EUR 15.3 billion in 66 countries.

Scientific exploration and responsible entrepreneurship have been
key to Merck's technological and scientific advances. This is how
Merck has thrived since its founding in 1668. The founding family
remains the majority owner of the publicly listed company. Merck
holds the global rights to the Merck name and brand. The only
exceptions are the United States and Canada, where the business
sectors operate as EMD Serono in healthcare, MilliporeSigma in life
science, and EMD Performance Materials. For more information about
Merck visit http://www.merckgroup.com.

Contact:

gangolf.schrimpf@merckgroup.com

Phone: +49-6151-72-9591

Investor Relations

investor.relations@merckgroup.com

Phone: +49-6151-72-3321

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