Safety, tolerability and pharmacodynamics of BoNT-E demonstrated in Phase I study1
PARIS–(BUSINESS WIRE)– Regulatory News:
Results of the first in-human study of a recombinant neurotoxin are being presented at the TOXINS International Conference in Copenhagen. Ipsen’s recombinant BoNT serotype E (rBoNT-E) was investigated in a phase I study that demonstrated its safety and tolerability profile in healthy volunteers1. The study also reported that it has a faster onset of action and a shorter duration of effect, as well as a quick time to peak activity, in comparison with established BoNT-A products. Further studies will be initiated to establish potential aesthetic and therapeutic uses of this investigational therapy.
“The application of recombinant techniques to create novel botulinum toxin-based medicines with different onsets and durations of action, will potentially offer clinicians the flexibility to choose the most appropriate neurotoxin for each patient, which is not an option today,” said Philippe Picaut, Pharm. D., PhD, Senior Vice President Research & Development for the Neuroscience Therapeutic Area, Ipsen.
Botulinum neurotoxins (BoNTs) are naturally occurring proteins (produced by Clostridium bacteria) that were first discovered in the 19th century. They are classified into seven serotypes (A-G) with the majority of commercialized BoNT products being serotype A. BoNTs are used in multiple different conditions after injection in skeletal muscles (eg cervical dystonia, hemifascial spasm, blepharospasm, spasticity in adult and children; aesthetic); smooth muscles (neurogenic detrusor overactivity, idiopathic bladder overactivity) or exocrine gland hyperfunction (eg sialorrhea, axillary hyperhidrosis).
Alexandre Lebeaut, M.D., Executive Vice President, Research & Development and Chief Scientific Officer, Ipsen stated: “Neurotoxin research is advancing at an unprecedented rate and we, at Ipsen, are at the forefront of this transformation, developing innovative therapeutic and aesthetic solutions that help patients take back control of their lives”.
Ipsen will have 50 posters at Toxins 2019 in Copenhagen, including:
- Outcomes of the first-in-human study with a recombinant botulinum toxin E (rBoNT-E): safety and pharmacodynamic profile of rBoNT-E compared with abobotulinumtoxinA (Dysport); Pons et al.
- New modified recombinant botulinum neurotoxin type F with enhanced potency; Burgina et al.
About the study1
This was a randomised, double-blind, placebo-controlled study, performed in male healthy volunteers at a single study centre in the UK (EudraCT: 2016-002609-20). Participants were healthy males (aged 18–49 years), who had not previously been treated with BoNT (any serotype) during the past 6 months. Overall, 65 subjects were screened and 28 were randomised to receive rBoNT-E or placebo (21 versus 7, respectively).
Subjects provided written informed consent prior to any study-related procedure and could withdraw at any time for any reason.
A baseline Extensor Digitorum Brevis CMAP total amplitude (peak-to-peak) ≥5 mV during electrophysiological examinations at screening and before study drug administration was required. Overall, rBoNT-E was well tolerated at the assessed doses. The majority of TEAEs were considered unrelated to treatment. There was no local diffusion to adjacent muscles for rBoNT-E at any dose level and no subject seroconverted following rBoNT-E injection.
1. Field, M. et al. AbobotulinumtoxinA (Dysport®), OnabotulinumtoxinA (Botox®), and IncobotulinumtoxinA (Xeomin®) Neurotoxin Content and Potential Implications for Duration of Response in Patients. Toxins (Basel). 10, 535 (2018).
Ipsen is a global biopharmaceutical group focused on innovation and specialty care. The group develops and commercializes innovative medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Diseases. Its commitment to Oncology is exemplified through its growing portfolio of key therapies for prostate cancer, neuroendocrine tumors, renal cell carcinoma and pancreatic cancer. Ipsen also has a well-established Consumer Healthcare business. With total sales over €1.9 billion in 2017, Ipsen sells more than 20 drugs in over 115 countries, with a direct commercial presence in more than 30 countries. Ipsen’s R&D is focused on its innovative and differentiated technological platforms located in the heart of the leading biotechnological and life sciences hubs (Paris-Saclay, France; Oxford, UK; Cambridge, US). The Group has about 5,400 employees worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information on Ipsen, visit www.ipsen.com.
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