* After five years of treatment, 91% of children were alive - without

    treatment, children with Type 1 SMA would not be expected to live past two
    years of age
  * 96% of Evrysdi-treated children could swallow, 80% could feed without a
    feeding tube and 59% could sit without support for at least 30 seconds

* Evrysdi is now approved in more than 100 countries with over 15,000 patients treated globally
Basel, 07 June 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new
five-year data confirming the sustained efficacy and safety profile of Evrysdi®
(risdiplam) in children with Type 1 spinal muscular atrophy (SMA) from the open-
label extension of the pivotal FIREFISH study. By the end of Year 5, 91% of
children treated with Evrysdi were alive, 81% were alive without permanent
ventilation and the majority were able to sit without support for at least 30
seconds (59%). At the end of year 5, seven children were able to stand, three
with support, four unaided and six could walk with support. Without disease
modifying treatment, natural history studies indicate that children with Type 1
SMA would not only never be able to reach such milestones, but also not
typically live past the age of two. The data were presented at the Cure SMA
Research & Clinical Care Meeting, June 5 - 7, 2024.
"These long-term findings confirm the ongoing benefit of Evrysdi for children
with Type 1 SMA," said Professor Giovanni Baranello, M.D., UCL Great Ormond
Street Institute of Child Health & Great Ormond Street Hospital, London, UK.
"Children treated with Evrysdi over five years have maintained or improved their
ability to sit, stand and walk - critical skills for development and daily
living. An overwhelming majority also maintained the ability to swallow and to
eat without a feeding tube."
Motor function abilities, as assessed by the Gross Motor Scale of the Bayley
Scales of Infant and Toddler Development Third Edition (BSID-III) and
Hammersmith Infant Neurological Examination 2 (HINE-2), were maintained or
continued to be achieved in those treated with Evrysdi. The FIREFISH results
showed most children treated with Evrysdi also maintained their feeding and
swallowing abilities; of those assessed at year 5, 96% were able to swallow and
80% were able to feed without a feeding tube.
"This is the final readout of the FIREFISH study, which has provided a wealth of
insights and data, helping to firmly establish Evrysdi as an important treatment
option, improving the lives of children across the globe living with SMA," said
Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product
Development. "This would not have been possible without the commitment and
dedication of the children and families who participated, as well as numerous
healthcare professionals and patient support organisations to whom we are
immensely thankful."
No treatment-related adverse events (AEs) led to treatment discontinuation or
withdrawal from the study. The overall rate of AEs decreased by 66% between Year
1 and the final year of study. The most common AEs were upper respiratory tract
infection (64%), pyrexia (64%) and pneumonia (50%). Hospitalisations declined
over the 5-year treatment period and 22% of children did not require
hospitalisation at all since beginning treatment with Evrysdi.
Evrysdi is the only oral, non-invasive small molecule SMA treatment designed to
be systemically delivered to both the central nervous system (CNS) & peripheral
tissues.
Roche leads the clinical development of Evrysdi as part of a collaboration with
the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat
SMA caused by mutations in chromosome 5q that lead to survival motor neuron
(SMN) protein deficiency. Evrysdi is administered daily at home in liquid form
either by feeding tube or by mouth.
Evrysdi is designed to treat SMA by increasing and sustaining the production of
SMN protein in the CNS and peripheral tissues. SMN protein is found throughout
the body and is critical for maintaining healthy motor neurons and core motor
functions such as swallowing, speaking, and breathing.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in
2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in
2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British
Pharmacological Society as well as the Society for Medicines Research Award for
Drug Discovery. Evrysdi is currently approved in more than 100 countries, and
the dossier is under review in a further 13 countries.
Evrysdi is currently being, or has been, evaluated in five global multicentre
trials in people with SMA:

  * FIREFISH (NCT02913482) - an open-label, two-part pivotal clinical trial in
    infants with Type 1 SMA. Infants were approximately 5.5 months of age
    (median) at the time of enrollment and of the 58 infants that completed the

first year of treatment, 52 entered the open-label extension study.The study met its primary endpoint.
* SUNFISH (NCT02908685) - a two-part, double-blind, placebo-controlled pivotal

    study in people aged 2-25 years with Types 2 or 3 SMA. The study met its
    primary endpoint.

* JEWELFISH (NCT03032172) - an open-label exploratory trial designed to assess

    the safety, tolerability, pharmacokinetics and pharmacodynamics in people
    with SMA aged 6 months to 60 years who received other investigational or
    approved SMA therapies for at least 90 days prior to receiving Evrysdi. The
    study has completed recruitment (n=174).
  * RAINBOWFISH (NCT03779334) - an open-label, single-arm, multicentre study,
    investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics
    of Evrysdi in babies (n=26), from birth to six weeks of age (at first dose)

with genetically diagnosed SMA who are not yet presenting with symptoms. The

    study met its primary endpoint.
  * MANATEE (NCT05115110) - a phase 2/3 clinical study to evaluate the safety

and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle

    growth, in combination with Evrysdi for the treatment of SMA in patients
    2-10 years of age. The FDA Office of Orphan Products Development granted
    GYM329 Orphan Drug Designation for the treatment of patients with SMA in
    December 2021. The study is currently recruiting.

About SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects
approximately one in 10,000 babies and is the leading genetic cause of infant
mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1)
gene, which leads to a deficiency of SMN protein. This protein is found
throughout the body and is essential to the function of nerves that control
muscles and movement. Without it, nerve cells cannot function correctly, leading
to muscle weakness over time. Depending on the type of SMA, an individual's
physical strength and their ability to walk, eat or breathe can be significantly
diminished or lost.
About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche. Our goal is
to pursue groundbreaking science to develop new treatments that help improve the
lives of people with chronic and potentially devastating diseases.
Roche is investigating more than a dozen medicines for neurological disorders,
including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica
spectrum disorder, Alzheimer's disease, Huntington's disease, Parkinson's
disease and Duchenne muscular dystrophy. Together with our partners, we are
committed to pushing the boundaries of scientific understanding to solve some of
the most difficult challenges in neuroscience today.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial
manufacturers of branded medicines, Roche has grown into the world's largest
biotechnology company and the global leader in in-vitro diagnostics. The company
pursues scientific excellence to discover and develop medicines and diagnostics
for improving and saving the lives of people around the world. We are a pioneer
in personalised healthcare and want to further transform how healthcare is
delivered to have an even greater impact. To provide the best care for each
person we partner with many stakeholders and combine our strengths in
Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavour to pursue a long-term perspective in all we do,
Roche has been named one of the most sustainable companies in the
pharmaceuticals industry by the Dow Jones Sustainability Indices for the
fifteenth consecutive year. This distinction also reflects our efforts to
improve access to healthcare together with local partners in every country we
work.
Genentech, in the United States, is a wholly owned member of the Roche Group.
Roche is the majority shareholder in Chugai Pharmaceutical, Japan.
For more information, please visit www.roche.com (http://www.roche.com).
All trademarks used or mentioned in this release are protected by law.
Roche Global Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
(mailto:media.relations@roche.com)

  Hans Trees, PhD           Sileia Urech
  Phone: +41 79 407 72 58   Phone: +41 79 935 81 48
  Nathalie Altermatt        Simon Goldsborough
  Phone: +41 79 771 05 25   Phone: +44 797 32 72 915
  Karsten Kleine            Nina Mählitz
  Phone: +41 79 461 86 83   Phone: +41 79 327 54 74
  Kirti Pandey              Yvette Petillon
  Phone: +49 172 6367262    Phone: +41 79 961 92 50
  Dr. Rebekka Schnell
  Phone: +41 79 205 27 03

Roche Investor Relations

 Dr. Bruno Eschli                        Dr. Sabine Borngräber
 Phone: +41 61 68-75284                  Phone: +41 61 68-88027
 e-mail: bruno.eschli@roche.com          e-mail: sabine.borngraeber@roche.com
 (mailto:bruno.eschli@roche.com)         (mailto:sabine.borngraeber@roche.com)

Dr. Birgit Masjost
Phone: +41 61 68-84814
e-mail: birgit.masjost@roche.com
(mailto:birgit.masjost@roche.com)
Investor Relations North America

  Loren Kalm
  Phone: +1 650 225 3217
  e-mail: kalm.loren@gene.com (mailto:kalm.loren@gene.com)

Â