TOPAZ Trial (SRK-015) - Scholar Rock

UPDATE from February 15th, 2019

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The Scholar Rock team replied to us with the following:

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"Results from the TOPAZ trial will be used to inform and guide further development decisions for SRK-015, including future trials that may include a broader age group of adults and individuals with Type 1 SMA. We will continue our collaborative approach with updates surrounding progress and plans for the SRK-015 program, as results from the TOPAZ trial emerge and help guide the path forward"

 

UPDATE from January 29th, 2019

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SMA United for Treatment Options is engaging Scholar Rock and CureSMA regarding the Nusinersen / SRK-015 (TOPAZ) trial.

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We are thrilled about this development but concerned with the current age limit on type 2 and 3 and exclusion of the Type 1 Spinal Muscular Atrophy popoulation

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In order for the drug to be approved and paid for all, we need data. In order to have data, we need to have adults type 2 & 3 in the study NOW and we need a plan for including type 1.

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We are seeking answers and suggesting ways to address this issue.

The study is about to wrap up phase 2 and determine next steps - this is the time to intervene and inform further development!

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Preliminary information shows great results on efficacy and safety for type 2 and 3 children and teenagers currently in the study.

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OUR GOAL IS TO ENSURE ACCESS TO TREATMENTS FOR ALL!

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Advocating for the inclusion of adults over the age of 21 and the entire type 1 population.

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SRK-015 is a complementary treatment which, when used in conjunction with Nusinersen (Spinraza), improves muscle strength and function. Scholar Rock is the pharmaceutical company that is developing this medication.

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TOPAZ is the phase two clinical trial where SRK-105 is being tested on children with SMA type 2 and 3 up to age 21.

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Our current effort is aimed at connecting with Scholar Rock, CureSMA, and other entities to gain better understanding of the trial design, discuss next steps and ensure that adults and type 1 patients will have access to this drug.

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Coordination / Collaboration with CureSMA

 

As part of our general intent to establish SMA United’s presence as a valuable partner and stakeholder within the larger system of treatment options, we see the need to define our relationship with CureSMA as one of the starting points.

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CureSMA believes it might be difficult to include certain groups (such as adults and type 1 patients) in trials and obtain the results necessary for approval, and thus the way to go is to model the "Nusinersen / Spinraza" process. Our intent is to take what worked in that process and to avoid what didn't work; lack of data on certain sub-populations resulted in payors refusing to cover the treatment and, to this day, we have many who do not have access to Spinraza for this very reason. As an advocacy organization, we are reaching out to Scholar Rock and CureSMA to express our concerns and suggest solutions to ensure the drug wins the broadest approval and that it is paid for all ages and types once it hits the market.