Community Update from Genentech

We are pleased to share the following SMA community letter from our partners at Genentech on their clinical development programs and initiatives.

Dear SMA Community,

Summer is upon us and so are a number of developments we are pleased to share with the SMA community. We are enjoying interacting with the community at various walks and events this season and are looking forward to connecting later this month in Orlando at the Cure SMA Annual Conference. Not only will we be bringing the DoubleTake runway show to participants live, but are also sponsoring the “adult track” of the conference and providing important updates through both the Community and Research Conferences. We look forward to seeing you there! In the meantime, a few important updates are outlined below:

Clinical Trial & Research Developments

As we have previously shared, Genentech is currently studying an investigational medicine, GYM329, for use in combination with risdiplam. The combination treatment is being evaluated to target the underlying cause of SMA by increasing levels of functional SMN protein (risdiplam) while also evaluating muscle
growth (GYM329).The initial phase of the study- MANATEE Part 1- is currently underway which will evaluate the safety, muscle measurements and immune responses in order to select the dose for Part 2.

As a result of community and advocacy group feedback, we adjusted the eligibility criteria on March 18, 2023, to include not just ambulatory, but also non-ambulatory patients and have extended the age of eligible participants to 25 years old. Interested participants can find more information about the study here.

We are pleased to share that WeSMA (Phase IV Clinical Research Study) Long-term follow-up study of individuals with spinal muscular atrophy receiving risdiplam is currently ongoing and actively recruiting at over 30 participating sites in the US. WeSMA is designed to help doctors and researchers expand their
understanding of the safety and effectiveness of Evrysdi and add to the Evrysdi information already available. Additional information can be found here.

Updates in Understanding Fertility in the SMA Population:
We continue to be committed to further understanding how the fertility of males with SMA may be impacted by both the disease itself and available treatments and are in the process of working with physicians who are interested in conducting studies on the topic. This topic will be explored further at the upcoming Cure SMA Clinical & Research meeting in greater detail.

In addition, results from a recent study examining the fertility impact in male rats and monkeys exposed to Evrysdi further increased community understanding of the issue. The animals in the study were exposed to both Evrysdi and a similar molecule named RG7800. It concluded that:

● While Evrysdi initially impacted male reproductive tissues in male rats and monkeys, there was no evidence of damage to the spermatogonia (sperm stem cells).
● There was demonstrated reversibility in male rats and monkeys following the end of treatment.

While the results of this study are promising, they cannot be applied to humans. Genentech is supporting additional studies to better understand male fertility in those living with SMA.

Additional Study Results:

Real-world Data in Risdiplam-treated Individuals with SMA:
The results of a study evaluating adherence (whether individuals are treated on time) and persistence (whether individuals continue on treatment) over 12 months in people treated with Evrysdi in the US were released at this year’s MDA Clinical and Scientific Conference. Here are the primary results:

● At 12 months, 79-93% of individuals were adherent with Evrysdi
● The age group with the highest proportion of individuals that were adherent to Evrysdi were 3-5 years old, followed by 0-2 years old, 6-17 years old and ≥18 years old
● The majority of individuals (80%) remained on Evrysdi at 12 months

We are so appreciative of your ongoing collaboration, partnership and guidance. Your perspectives – whether as an individual living with SMA, family member or advocate – continue to help us understand and serve the needs of the SMA community. We thank you for your continued commitment.

Genentech SMA Team

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