Revance Therapeutics Inc (RVNC)

[mouton29]

Given the preoccupation of the neurologist on the slingshot call with the ASPEN-OLS (open label) trial, I tried to find some efficacy data from that trial. The primary outcome was safety and that seemed excellent and the secondary endpoints were merely efficacy measured at weeks 4 and 6 of each cycle. Results have been presented at a couple of conferences, including a 2022 American Academy of Neurology meeting (https://index.mirasmart.com/aan2022/PDFfiles/AAN2022-002424.html) but I didn't seem much on line data, the following in the journal Neurology is the most I found. It looks pretty good. https://n.neurology.org/content/100/17_Supplement_2/3566. I've highlighted some key points:

Abstract

Objective: To evaluate efficacy, safety, and immunogenicity over multiple treatments of DaxibotulinumtoxinA for Injection (DAXI; DAXXIFY™), a novel botulinum toxin type A, in subjects with cervical dystonia (CD).

Background: Pooled Phase 3 results are presented from placebo-controlled, single-dose (ASPEN-1) and open-label extension (ASPEN-OLS) studies.

Design/Methods: In ASPEN-1, 301 adults with moderate-to-severe CD were randomized 1:3:3 (placebo:DAXI 125U:DAXI 250U) and followed for =36 weeks. In ASPEN-OLS, 357 subjects (271 from ASPEN-1 and 86 newly enrolled subjects) received up to 4 treatments of DAXI 125U, 200U, 250U, or 300U based on investigator decision. Re-treatment in ASPEN-OLS was based on loss of 80% of peak treatment effect or subject request and investigator judgment.

Results: Across both studies, 382 subjects received 1240 DAXI treatments. Mean change from baseline (CFB) in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score, averaged over Weeks 4 and 6 post injection, increased over the 5 treatment cycles (-12.3 in ASPEN-1; -15.4, -17.7, -17.9, and -19.9 for Cycles 1–4 of ASPEN-OLS, respectively). Similar trends for increasing CFB over successive cycles were seen for TWSTRS subscales of pain (-3.0 to -5.8), disability (-3.5 to -6.0), and severity (-5.7 to -8.3). Clinical and Patient Global Impression of Change responder rates (=2-point improvement) at Weeks 4 or 6 also increased from ASPEN-1 (58.8% and 52.2%) to Cycle 4 of ASPEN-OLS (83.1% and 69.2%).

Treatment-related dysphagia was reported in 2.7% of DAXI-treated subjects in ASPEN-1 and in 3.9%, 4.3%, 4.7%, and 3.1% of subjects in ASPEN-OLS Cycles 1–4, respectively (4.2% of 985 treatments in ASPEN-OLS). The incidence of treatment-induced anti-drug antibodies was low, with no observed trend in incidence over successive doses.

Conclusions: Repeat treatments with DAXI were efficacious, safe and well tolerated, with a favorable immunogenicity profile.

Thus, results seem to improve over time, and there were 985 treatments over the 52 week of the ASPEN-OLS trial in 357 OLS subjects. I suppose it depends when in the course of the trial their first treatment was, but 985/357 = 2.76, so less than 3 treatments on average. If participants are injected at close to the beginning of the OLS trial, it's better than 20 weeks between treatments.