In the world of rare diseases—which affect over 360 million people globally—Anavex Life Sciences Corp. is making significant strides in its approach to research and clinical trials, particularly for Rett syndrome. Anavex Life Sciences is a clinical-stage biopharmaceutical company that is developing therapeutics for the treatment of Alzheimer's disease and other central nervous system (CNS) diseases, including Rett syndrome.
While the COVID-19 pandemic disrupted many clinical trials in 2020 and 2021, Anavex forged ahead, conducting a Phase 2 clinical trial evaluating the safety and efficacy of its investigational therapy, ANAVEX®2-73 (blarcamesine), for Rett syndrome. The trial completed successfully, a notable achievement given the widespread impact of the pandemic on global clinical trials. This success generated significant excitement not only within the Rett syndrome community, but also within the larger rare disease advocacy sphere.
Timely Novel Trial Designs
One of the key figures driving this effort is Christopher U. Missling, PhD, President and CEO of Anavex. Under his leadership, Anavex embraced innovative approaches to trial design long before the pandemic made such adaptations necessary. For a community that has historically struggled to get the attention of biopharmaceutical companies, particularly for adults with rare neurodevelopmental disorders, Anavex’s proactive approach was encouraging.
In fact, rare disease advocates have long called for more patient-centered approaches to trial design. For years, many advocacy groups have pushed for decentralized trials that account for the unique challenges faced by patients with rare diseases, especially adults. The pandemic underscored the necessity of such approaches, but Anavex had already recognized the value in working closely with the patient community to develop more accessible and less burdensome trials.
Leveraging Partnerships
The Rare Advocacy Movement, a prominent organization advocating for rare disease patients, has pointed out that over 70% of people living with rare diseases are adults. Yet, many biopharmaceutical companies have historically overlooked these populations, assuming that the opportunity for treatment has passed. Anavex, however, chose to collaborate directly with the Rett syndrome community and international advocacy groups such as Rettsyndrome.org, Reverse Rett and Rett UK. Together, they designed a trial that was not only innovative but also tailored specifically to the needs of adult women with Rett syndrome.
Anavex’s collaboration with these advocacy groups was key to their success. By engaging with community-based advocates, Anavex Life Sciences was able to design a clinical trial that did not require patients to travel—an important consideration for individuals living with debilitating conditions. Even essential elements of the trial, such as blood work, were arranged to be conducted in the patients’ homes, minimizing the disruption to their lives and making participation more feasible.
Patient-Centricity Starting With Research
This patient-centric approach has set a new standard in the industry, particularly for drug developers working with neglected adult populations in rare diseases. Advocacy groups are now urging the broader biopharmaceutical sector to follow Anavex’s lead and to engage directly with rare disease communities when designing trials. By incorporating community insights and designing trials that address the real-world needs of patients, drug developers can significantly reduce risks and increase the chances of success in bringing new treatments to market.
Dr. Missling has been vocal about the responsibility of pharmaceutical companies to focus on "human-centered" protocols. His philosophy emphasizes that drug development must prioritize patient well-being, even in the early stages of research and clinical trials. This focus on compassionate, practical solutions aligns with the goals of many in the rare disease advocacy community, who have long argued for more consideration of the unique needs of adults living with rare diseases.
Listening to Patients
As a result, Anavex's work has earned the company widespread praise within the rare disease community. By moving forward during a time when many others were forced to pause, they not only gained a competitive edge, but deepened their connection with the patients and advocacy groups they serve. This approach—valuing patient input and prioritizing their needs—offers a blueprint for how biopharmaceutical companies can work more effectively with underserved populations.
Anavex’s results in the Rett syndrome trial illustrates a broader lesson: companies that listen to the communities they aim to serve and act proactively can make meaningful progress, even in the face of global challenges. Their work stands as a testament to the power of collaboration, innovation and patient-centered care in advancing treatments for rare diseases.