Praxis Precision Medicines, Inc updated our SCN2A community on the status of their three SCN2A programs in development and their CIITIZEN partnership.

by Shawn Egan, Ph.D. (parent to a child with SCN2A-DEE and extensive experience as a Biotech Equity Research Analyst)

Posted on March 25,2021


PRAX-562

PRAX-562 is currently in phase 1 clinical trials (meaning it is being tested in humans). This agent has completed its single-ascending dose (SAD) portion of the phase 1 study and is now being evaluated in a multiple-ascending dose (MAD) portion of the trial. The SAD study was completed up to the maximum planned dose with no dose limiting toxicities (meaning the safety profile was good) and Praxis is currently at the highest preplanned dose in the MAD study with plans to dose escalate further if it continues to be well tolerated. Praxis plans to present initial PRAX-562 proof-of-concept data in mid-2021.

PRAX-562 is a small molecule inhibitor medicine that acts as a persistent sodium current blocker. Praxis is developing this agent in SCN2A and SCN8A and it has the potential to be therapeutic for individuals that have gain-of-function mutations. PRAX-562 has been granted the Rare Pediatric Disease Designation by the FDA.

PRAX-222

PRAX-222 is currently in IND-enabling, GLP (good laboratory practices) toxicology studies (these are pre-clinical/being tested in animals and not current being tested in humans). If successful, Praxis plans to file an IND (Investigational New Drug) application with the FDA in 2022 and move the drug into human clinical testing.

PRAX-222 is an antisense oligonucleotide medicine that acts to block the production of SCN2A protein. Praxis is developing this agent in SCN2A and it has the potential to be therapeutic for individuals that have gain-of function mutations. PRAX-222 has been granted both the Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA

Loss-of-Function Program

This program is in collaboration with the Florey Institute, and is currently in discovery studies (meaning the drug is in early design and preclinical studies, and not currently being tested in humans. The Praxis team indicated that this program is still a few years away from clinical studies (being tested in patients).
This program is an antisense oligonucleotide medicine that is in development for loss-of-function SCN2a patients.

CIITIZEN

US SCN2A families have access to the CIITIZEN platform (see links below for easy access, either link will take you to the correct location). CIITIZEN is a healthcare technology company that gathers the complete medical records (labs, doctor notes, EEGs, imagining) of enrolled participants into a single portal. Information can be accessed by patients/caregivers/and providers that have been granted access by the caregiver.

Praxis has partnered with CIITIZEN to guide the development of biomarkers and to advance the understanding of SCN2A

To Register :

https://www.ciitizen.com/scn2a/
https://scn2a.com/research/scn2a-observational-study/

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Frequently Asked Questions

Drugs typically have to clear multiple clinical trial hurdles in order to support approval. The typical trial phases are 1, 2, and 3 with lower number being earlier trials and larger numbers being more advanced trials.

Phase 1 trials are typically in healthy volunteers and are evaluating initial safety, and dosing in humans. These trials help ensure that the drug is safe enough to be evaluated in patients and also are used to identify an appropriate dosing range for subsequent trials.

Phase 2 trials are typically used to provide proof of concept (that the drug has an efficacy signal, is providing a benefit to patients) and also tests safety in the patient population. These trials often are used to help design and power registrational phase 3 trials.

Phase 3 trials are often the trials used to support the approval of the agent and may be the final clinical hurdle a drug needs to pass in order to be approved by the FDA.

This is a designation granted by the FDA when a drug is being developed to treat a pediatric condition that is serious and life-threatening and occurs in under 200k people in the US.

This is a program used by the US government to incentivize drug development for pediatric rare diseases. If a drug is approved that has been granted the rare pediatric disease designation, then the developing company receives a voucher that grants priority review of a future drug (which can accelerate the developmental time of a drug by ~4 months; for example: if drug#1 is approved and had been granted the rare pediatric disease designation then said company would receive a priority review voucher that they could use on drug#2 ).

This is a designation granted by the FDA when a drug is being developed to treat a rare disease (occurs in under 200k people in the US).

This is a program used by the US government to incentivize drug development for rare diseases. Drugs awarded this designation received a number of incentives by the FDA including: 1. Market exclusivity for 7 years post approval, 2. A waiver of application of user-fees, and 3. A 50% tax credit for clinical testing expenses

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