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Clinical investigation of topical delivery of a muscarinic receptor antagonist for the prevention of chemotherapy-induced peripheral neuropathy

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R44CA265565-01
Agency Tracking Number: R44CA265565
Amount: $2,000,000.00
Phase: Phase II
Program: SBIR
Solicitation Topic Code: 102
Solicitation Number: PA20-262
Solicitation Year: 2020
Award Year: 2021
Award Start Date (Proposal Award Date): 2021-09-01
Award End Date (Contract End Date): 2023-08-31
Small Business Information
4685 CONVOY ST #210
San Diego, CA 92111-2339
United States
DUNS: 078868444
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (858) 546-9044
Business Contact
Phone: (858) 336-8094
Research Institution

Chemotherapy-induced peripheral neuropathy (CIPN) is a debilitating condition that afflicts 70% of cancer
patients undergoing chemotherapy and limits the dose and duration of treatment. Symptoms range from sensory
loss to painful neuropathy and are accompanied by electrophysiological and structural indices of nerve damage.
The American Society of Clinical Oncology currently makes no recommendations for the prevention or reversal
of CIPN and provides only a moderate recommendation for symptomatic pain relief. CIPN is therefore a major
unmet clinical concern. WinSanTor (WST)’s founders have published preclinical studies revealing that peripheral
nerve metabolism and growth is retrained under both in vitro and in vivo conditions by cholinergic suppression
of mitochondrial activity acting via neuronal M1 receptors. Removal of this cholinergic “brake” by muscarinic
antagonists promotes nerve growth and protects against neuropathy in multiple animal models of diabetes,
chemotherapy and HIV-induced neuropathy. Proof of concept clinical data obtained via NIH R21 funding
demonstrates that topical treatment with a muscarinic receptor antagonist can significantly reverse loss of intra-
epidermal nerve fibers (IENF) in the skin of patients with diabetic neuropathy and improve multiple indices of
neurological function and quality of life, as well as relieving diabetic neuropathic pain. Supported by our funded
NCI Phase I and II STTR grants and WST's internal funding, WST has achieved four major milestones
in developing a novel topical formulation of Pirenzepine (PZ), a selective M1R antagonist: 1) Phase 1 clinical
trial was completed in Australia in November 2018, 2) A Phase 2 clinical trial in diabetic patients has been
initiated (NCT04005287) in Canada in July 2019, 3) Pre-IND meeting with FDA was completed in September
2019, and FDA clearly informed us that WST will be able to conduct exploratory studies in CIPN under the
requested IND, and 4) IND filing completed on July 29, 2020 for a double-blind, randomized Phase 2 clinical
trial in diabetic patients with painful neuropathy, and IND was approved on August 28, 2020 (IND
144090). Our preclinical and exploratory clinical data encourages the present SBIR Direct-to-Phase II
application to evaluate the safety and efficacy of topical PZ in oncology patients administered cisplatin and
paclitaxel for the prevention of dose-limiting CIPN. The results of this project will result in clinical proof-of-
concept data to support additional investments and partnerships for an FDA registration trial of the first
disease-modifying treatment for painful CIPN.

* Information listed above is at the time of submission. *

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