Company
Portfolio Data
Back to Company SearchSCHEDULE 1 THERAPEUTICS INC
UEI: TV68WM7PHYW3
Number of Employees: 1
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
SBIR/STTR Involvement
Year of first award: 2020
1
Phase I Awards
1
Phase II Awards
100%
Conversion Rate
$308,465
Phase I Dollars
$2,019,222
Phase II Dollars
$2,327,687
Total Awarded
Awards
Optimization of Cannabinoids for Development as FDA-Approved Migraine Therapeutics
Amount: $2,019,222 Topic: NIDA
Abstract Schedule 1 Therapeutics (S1Tx) is developing optimized cannabinoid therapeutics for FDA approval in migraine, the second most debilitating disease worldwide. The goal of this SBIR is to obtain IND clearance from FDA to begin a phase 1 clinical trial of S1Tx’s lead candidate S1-220 (100:1 CBD:THC fixed-dose combination) which demonstrated efficacy in preclinical migraine models in the company’s Phase 1 STTR, with no adverse effects. Migraine has a 15% global prevalence and is a leading cause of global disability. Current treatments are not sufficient with at most half of patients relieved from debilitating pain. Evidence supports potential efficacy of Δ9- tetrahydrocannabinol (THC) and cannabidiol (CBD) in migraine. However, these studies suffer from lack of proper controls or do not take a drug design approach to optimizing the combination as a migraine treatment. S1-220 will treat migraine via multiple novel mechanisms and meet the needs of millions for whom current therapies are ineffective or pose unacceptable risks. Rapid administration supports rescue of acute attacks, and S1-220 is an alternative to unmonitored self-medication of cannabis, which may pose health, safety, and legal harms to patients if misused. This SBIR will advance a novel therapeutic and will validate our model to develop optimized cannabinoid therapeutics for large pain and CNS disorders. S1-220 was identified in a Phase I STTR with partners at University of Iowa (Russo). This Phase II SBIR will select human doses for formulation into a sublingual spray that meets FDA requirements for a phase 1 clinical trial. Aim 1. Escalate S1-220 dosing (100:1 CBD:THC) in preclinical CGRP and SNP migraine models and assays (three migraine symptom and three adverse effect assays). Compare pharmacokinetic (PK) levels of THC, 11-OH-THC, THC-COOH, CBD, 7-OH- CBD, and 7-COOH-CBD with behavioral responses for use as a basis for allometric scaling to set targets for equivalent human PK levels and corresponding human doses of S1-220 for a phase 1 clinical trial. Aim 2. Design and manufacture a drug product suitable for use in a phase 1 clinical trial. Pre-formulation (solubility, stability) and formulation (drug-drug and drug-device compatibility) studies with S1-220 will be conducted to design and manufacture a sublingual spray clinical dosage form suitable for use a phase 1 trial. Aim 3: Synthesize findings from Aim 1 and Aim 2, and follow FDA pre-IND meeting guidance (2021) to prepare a phase 1 clinical protocol, establish a scientifically appropriate case for S1-220 safety, and submit an IND application for FDA clearance to begin a phase 1 clinical trial. Of ~40M migraine suffers in the U.S. (a conservative 12% prevalence), ~22M are diagnosed. Of these, ~ 7.3M are prescribed the standard of care triptans, of which > 80% have discontinued at 2 years (~6.2M). S1-220 is targeting 25% of these failures(~1.55M), at $1,460 annual spend per patient (~50% of gepant and ditan price), yielding $2.3B in peak sales. There are over 1 billion migraine sufferers, worldwide
Tagged as:
SBIR
Phase II
2024
HHS
NIH
Optimization of Cannabinoids for Development as FDA-Approved Migraine Therapeutics
Amount: $308,465 Topic: NIDA
Optimization of Cannabinoids for Development as FDA-Approved Migraine Therapeutics Project Summary: Migraine is a disorder that affects 15% of all people in the US, the majority of which are women. Migraine is one of the leading causes of missed days at work during which patients are unable to complete many daily activities. Currently approved treatments for migraine are not satisfying with at most half of the patients relieved from their pain. There is historical evidence for the efficacy of cannabis for the treatment of migraine. However, due partly to the regulatory status of cannabis in most countries worldwide, there are very few clinical or pre-clinical studies on its efficacy on migraine symptoms. The limited studies that do exist suffer from lack of controls or lack of knowledge of which of the many cannabinoids are present in the tested preparations and which of those cannabinoids are responsible for efficacy. Overall, there is a critical need for more rigorous preclinical and clinical studies of the effect of cannabis on migraine headache to better characterize the efficacy and adverse effects of its multiple components. The specific molecules extracted from cannabis that will be used in this study are Δ9-tetrahydrocannabinol (THC) and cannabidiol (CBD), the two primary plant- based cannabinoids. These molecules have different pharmacological and physiological effects and also have distinct adverse effect profiles. In an effort to provide proper preclinical scientific rationale for a formulation of cannabis-derived molecules that can be developed into an FDA-approved treatment of migraine, Schedule 1 Therapeutics is developing purified, orally administered CBD and THC molecules and proposes to test them as acute treatments for migraine. The objective of the proposed project is to determine the efficacy of purified CBD and THC in a preclinical model of migraine and identify a ratio of CBD:THC that maximizes efficacy and minimizes adverse effects. Our overall hypothesis is that while CBD and THC may show efficacy when used separately, a combination of the two will be more effective with fewer adverse effects than either alone. We propose two aims that use well established experimental behavioral approaches to validate CBD and THC as good candidate drugs to be further developed for the treatment of migraine symptoms. In Aim 1, we will determine the most efficient combination of CBD:THC to reduce CGRP-induced migraine-like phenotypes in mice. In Aim 2, we will determine the adverse effects of the different combinations of CBD:THC in mice. We propose to study THC and CBD separately as well as together in different combinations in order to find an optimal ratio that would have a high efficacy and low adverse effects. The outcome of these studies will add much-needed data from rigorous experiments to the scientific literature on the efficacy of cannabinoids for migraine and also constitute components of the development plan of cannabis-based drug by Schedule 1 Therapeutics. The optimal CBD:THC ratio determined by these studies will then serve as a lead product formulation for further development by Schedule 1 Therapeutics.PROJECT NARRATIVE - RELEVANCE TO PUBLIC HEALTH Migraine represents a significant burden to society. The proposed experiments will establish whether a cannabis extract of cannabidiol and Δ9-tetrahydrocannabinol, alone or in predefined combinations designed to optimize efficacy and minimize adverse effects, could be developed into valid FDA- approved therapeutics to treat migraine. FDA-approved cannabis-based drugs for migraine would provide an excellent alternative for the part of the patient population that doesn’t respond to existing treatments, as well as an alternative to the use of unregulated or illicit cannabis.
Tagged as:
STTR
Phase I
2020
HHS
NIH