I was talking yesterday with some friends that have children with Dravet syndrome, and they asked me about what news we can expect in 2018 from those programs that are in clinical trials for Dravet syndrome.
For them, and for anyone interested, here is what we can expect in 2018 from the clinical programs from GW Pharmaceuticals, Zogenix, Ovid Therapeutics and Takeda, PTC Pharma and NYU, and OPKO Health, based on what these companies have communicated.
First quarter 2018
No big news expected this quarter around Dravet syndrome unless OPKO Health announces the initiation of the clinical trials with their antisense oligonucleotide OPK88001 [4]. This product was expected to start pilot trials in the second half of 2017 or first half of 2018. OPK88001 is the first disease-modifying treatment to be developed for Dravet syndrome and a potential game-changer.
Second quarter 2018
This is a very exciting quarter for Dravet syndrome. FDA has June 27 as the deadline to make a decision on the approval for GW Pharma Epidiolex to treat Dravet and Lennox-Gastaut syndromes [1]. Around the same time, Zogenix will announce the results of the second large Phase 3 clinical trial in Dravet syndrome with fenfluramine [2]. If the data are as strong as the first clinical trial the Dravet community will have fantastic news for their International Dravet Syndrome Awareness day (June 23rd).
Around the same time we expect also the results of the pilot study of ataluren in Dravet syndrome and CDKL5 deficiency disorder that is taking place at NYU [3]. This is an investigator-initiated trial, not a pivotal trial, but the placebo-controlled double-blind design makes it a very important trial to evaluate the potential efficacy of ataluren in children with nonsense mutations in SCN1A (Dravet syndrome) and CDKL5.
Third and fourth quarter 2018
The third quarter arrives with the required DEA rescheduling of cannabidiol (90 days after the FDA approval), followed by a potential launch in the last quarter. The epilepsy community meets every year during the first week for December for the American Epilepsy Society meeting and if there are no bad surprises Epidiolex should be the star of the 2018 meeting with an imminent launch.
Around the same time, Zogenix will file an NDA for the treatment of Dravet syndrome with ZX008, which a potential launch one year behind Epidiolex. Zogenix also has an advanced clinical trial program for Lennox-Gastaut Syndrome, with a Phase 3 trial ongoing based on a positive Phase 2 dose-finding study.
We also expect to hear before the end of 2018 about the results of the second large Phase 3 clinical trial of Epidiolex in Dravet syndrome and the Phase1b/2a clinical trial of OV935/TAK935 in a basket trial including patients with Dravet syndrome [4].
In summary: we expect in 2018 the first approval of a treatment for Dravet syndrome in the US, the results of clinical trials with three new therapeutics (ZX008, ataluren and OV935/TAK935), and the initiation of the first clinical trial ever done with a disease-modifying therapy designed to treat Dravet syndrome (OPK88001).
2018 is going to be a good year.
Ana Mingorance PhD
Sources:
[1] GW Investor Presentation, January 2018
[2] Zogenix Investor Presentation, January 2018
[3] Dr Devinsky at CDKL5 Forum, December 2017
[4] OPKO health press release August 8 2017
[5] Ovid Therapeutics Corporate Presentation, January 2018
Announcements within the same quarter ordered by drug name (alphabetic).