REMEDi4ALL highlights JNJ-37822681 – a promising therapeutic opportunity for rare epilepsies like KCNQ2-DEE

On 1 December 2025, REMEDi4ALL published a news article titled:

“REMEDi4ALL helping to refocus an existing experimental drug to treat rare childhood epilepsies”

In this article, REMEDi4ALL reports on the work of the international research consortium TREATKCNQ, which is investigating an existing experimental compound — JNJ-37822681 — as a potential therapy for KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE).

REMEDi4ALL itself does not conduct lab research; instead, it supports drug repurposing efforts to bring promising treatments for rare diseases forward more quickly and efficiently.

Who is involved?

The article emphasises collaboration between scientists, clinicians, families, and patient organisations, including:

• KCNQ2 Cure Alliance
• KCNQ2 e.V.

TREATKCNQ is a multidisciplinary consortium funded through the European Joint Programme on Rare Diseases (EJPRD), aiming to develop new therapeutic approaches directly targeting the mechanisms affected by KCNQ2 mutations.

What has been discovered?

REMEDi4ALL references a publication in the British Journal of Pharmacology, where the TREATKCNQ researchers demonstrate that:

• JNJ-37822681 can reverse a key functional deficit in neurons carrying KCNQ2 mutations.
• The compound reduces epileptic activity in patient-derived brain cells and in well-established epilepsy models.
• The drug has already undergone earlier clinical testing (for other purposes), meaning data on:
  – dosing,
  – safety,
  – pharmacology, and
  – manufacturing
  – already exist.

These legacy data could significantly accelerate the path toward clinical trials in children with KCNQ2-DEE, if access to the data is granted.

What is the main obstacle?

REMEDi4ALL clearly states the current challenge:

• The original developer company is no longer pursuing JNJ-37822681 commercially.
• The existing clinical trial data remain with the company.
• Without access to these data, many early safety and dosing studies would need to be repeated, causing major delays and costs.

The article calls for these data to be made accessible so that safe and efficient clinical trial planning for KCNQ2 children can begin.

The family perspective – featuring Nora

The REMEDi4ALL article shares the story of Nora, daughter of Aila and Oliver Coulmann, and describes her daily challenges living with KCNQ2-DEE:

• early-onset seizures
• severe hypotonia
• significant developmental delays

Nora’s parents are quoted as saying that this discovery brings them:

“a big step closer to a much-needed drug for children like ours.”

This highlights the urgent need for progress — and the human reality behind the science.

What exactly is REMEDi4ALL’s role?

REMEDi4ALL supports projects like TREATKCNQ by providing:

• its Drug Repurposing Concierge,
• regulatory and strategic guidance,
• expertise in data evaluation and trial design,
• a patient-centred approach to rare disease development.

It is a support and acceleration platform, not a laboratory — designed to help projects like TREATKCNQ move forward faster.

Links included in the REMEDi4ALL article

The article also links to:

• the Fraunhofer ITMP press release on JNJ-37822681
• the original British Journal of Pharmacology publication
• the websites of the KCNQ2 Cure Alliance and KCNQ2 e.V.

Our previous coverage as KCNQ2 e.V.

We have already informed our community about this important development:

• On 6 September 2025 and 8 September 2025, we reported on Facebook about the J&J compound and the Fraunhofer press release
  Example URL: facebook.com/share/p/1GMrHjB8bf/?mibextid=wwXIfr
• On our website, we published the article:
  “Neue Hoffnung durch Fraunhofer-Forschung – Ein möglicher Therapieansatz für KCNQ2”
  URL: https://kcnq2.de/neue-hoffnung-durch-fraunhofer-forschung-ein-moeglicher-therapieansatz-fuer-kcnq2/

The new REMEDi4ALL feature now amplifies this work at a European level, showing that the scientific and patient community is aligning behind this promising direction.

Conclusion

• REMEDi4ALL is not conducting the KCNQ2 experiments,
• but it supports and strengthens the progress made by TREATKCNQ and Fraunhofer.
• JNJ-37822681 is a concrete therapeutic candidate, showing positive effects in cells and models with KCNQ2 mutations.
• Access to existing trial data is now the decisive step needed to move toward clinical trials.
• Families, researchers and patient organisations — including KCNQ2 e.V. — play a vital role in ensuring that momentum is maintained.

💜 We will continue advocating and supporting this work — for Nora, and for all children living with KCNQ2-DEE.

Your KCNQ2 e.V. Team

Disclaimer

This article is for information only and does not replace medical advice. No liability is assumed for accuracy, completeness or timeliness.