Solaxa Announces Phase 3 Clinical Trial for SCA27B
Bethesda, MD — March 28, 2025 – Solaxa Inc., a biopharmaceutical public benefit corporation focused on rare neurological conditions, has announced plans to conduct a registrational clinical trial evaluating its investigational therapy, SLX-100, for spinocerebellar ataxia type 27B (SCA27B).
This pivotal-powered study will be funded in part by a $7.3 million dollar grant awarded to Dr. Susan Perlman, MD, at the University of California, Los Angeles by the Congressionally Directed Medical Research Program. Solaxa will serve as the regulatory sponsor of the trial and partner with leading ataxia centers of excellence. Additional clinical trial sites will be announced later this year.
There are currently no FDA approved therapies for SCA27B, a rare hereditary ataxia. “We are thrilled to move one step closer to impacting the lives of people living with SCA27B,” said Christian Walker, CEO & Founder of Solaxa.
For more information about Solaxa and the upcoming clinical trial, please contact:
CONTACT:
Solaxa Inc.
Bethesda, MD 20814
7272 Wisconsin Avenue Floor 9
info@solaxa.com
To the SCA27B community,
My name is Christian Walker, and I am the CEO & Founder of Solaxa. I am writing to address your questions about Solaxa and whether our plans to seek FDA approval for 4-Aminopyridine (4-AP) for people with ataxia will impact access or cost of the drug. First, please know that I share your concerns that price and access to medicines you depend on are critical issues.
Individuals buying their 4-AP from Mark Cuban’s Cost-Plus Pharmacy might not know that the company operates as a public benefit corporation (PBC). Similarly, Solaxa is a public benefit corporation, and we firmly believe that doing good can coexist with being profitable. Our charter and bylaws stipulate that the medicines we promote must be both impactful and accessible. What does this mean for you?
Solaxa’s goal is to lower the price each of you pay for 4-AP. We will work to achieve this in various ways, including a robust patient assistance program ensuring that whether you are insured, underinsured, or uninsured, you have the most affordable option available which may be as low as a $0 copay or free.
What about access? There has only ever been one dosage of 4-AP approved, and it can only be used in adults. Solaxa doesn’t believe that 4-AP is a one-size-fits-all drug. We plan on bringing to market multiple dosages and formulations of 4-AP to serve adults and children with the many forms of ataxia where 4-AP has been shown to work.
I have not yet earned your trust that our commitment to lowering the costs borne by patients and increasing access is central to everything we do at Solaxa. What I can promise is that we will be transparent about how we intend to lower prices and work with the SCA27B community to be a good partner to earn your trust.
I look forward to continuing to answer your questions and trying to address your concerns, as well as share additional details about our upcoming 4-AP trial in the months ahead.
Respectfully,
Christian Walker
Sites are now enrolling SCA27B patients in a Natural History Study. Watch the recorded webinar and follow the links to see if you are eligible and how to enroll.
Natural history studies collect health information over time to track the progression of symptoms and to better understand a given medical condition. For this study, patients would go to one of the host ataxia clinics (once per year) for a physical exam and would also fill out several ataxia-related surveys. Blood samples will be drawn and some sites will include an MRI. This study will help to better define SCA27B and will be used to teach the medical community more about this rare disease and direct research for the treatment of SCA27B. A natural history study is not a clinical trial, so it does not involve giving patients treatments/drugs.
CRC-SCA Natural History Study
The natural history of the SCAs and factors that may modify them are not completely understood. There is a need to define natural history in geographically distinct areas and develop additional methods to document progression that may be more sensitive and reliable. There is also limited knowledge of factors that may modify symptoms of SCA.
The CRC-SCA natural history study and biomarker development is growing and adapting during this exciting new era in ataxia research as pharmaceuticals are advancing to therapy development for ataxias. CRC-SCA plans to add specific clinical end-point assessments and biospecimen collection. This will increase the strength of this study allowing the consortium to partner with stakeholders in a meaningful way that will include patients as research collaborators.
The Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) continues to recruit research participants who have a confirmed diagnosis of SCA 1, 2, 3, 6, 7, 8, 10, 27B, or RFC1 Ataxia/CANVAS. This is an opportunity for anyone in the United States (and now Canada) with these forms of SCA at any stage of the disease to participate.
You can use NAF’s new CRC-SCA Eligibility Screening Tool to confirm your eligibility to participate in this natural history study. Click this link for the eligibility tool and to learn more.
Contact the research coordinator at a site near you to register to participate in the CRC-SCA.
All About SCA27B
Nov 8, 2023 - 12:30 PM in Central Time (US & Canada)
Do you or a loved one have SCA27B? Join our webinar to learn about the mechanisms of the disease, the diagnostic journey, and what to expect for clinical care with this diagnosis.
Research and Treatment Development for SCA27B
Dec 12, 2023 - 12:30 PM in Central Time (US & Canada)
Do you or a loved one have a SCA27B? Our expert will teach us how SCA27B is studied and give an overview of the current state of research and drug development for the disease.
NAF Member’s Story - Mary Hogan
One family’s story of genealogy, research, and the discovery of SCA27B
Newly discovered ataxia SCA27B shows promising response to a treatment
Posted on UK Ataxia (ataxia.org.uk) on May 31, 2023
Late-onset spinocerebellar ataxia 27B (SCA27B) is newly discovered ataxia which is caused by a mutation in a gene called fibroblast growth factor 14 (FGF14). This discovery may lead to more diagnoses for people with ataxia of unknown cause. It is a late onset ataxia and, in some people, the condition starts with episodes of ataxia. To find out more about SCA27B click here.
A paper was recently published which showed that treatment with the drug 4-aminopyridine (4-AP) led to reduced daily symptoms and symptom severity in people with SCA27B. The mechanism of how this might work in SCA27B is not fully understood, but the researchers propose that 4-AP, which blocks potassium channels, could compensate for a problem with brain cells in the cerebellum transmitting signals.
This finding was part of a larger study, which looked at the symptoms and progression of people with SCA27B in Germany. The researchers found that six out of seven people in the study who had received 4-AP treatment (86%) reported a response to the treatment that was relevant to their everyday living. In 3 participants, the researchers looked at the differences in symptoms when participants were ‘on’ the treatment versus when they were ‘off’ the treatment. During periods ‘on’ the treatment, reductions in the time experiencing symptoms per day and in the number of days affected by severe symptoms were reported. When participants went ‘off’ the treatment, the beneficial effect of 4-AP went away. The results suggest that 4-AP is more likely a drug that alleviates symptoms, rather than one that affects disease progression.
In this study only a very small number of people were treated, and the effectiveness of the drug was not compared with taking a placebo. Therefore, a larger trial with comparison against a placebo would be the next step needed to test the drug’s effectiveness.
The abstract of the scientific paper can be found here.
National Ataxia Foundation 2023 Annual Ataxia Conference
Remote and in person, Las Vegas, NV
March 30 - April 1, 2023