SP1707 is the development name/number of the Company's first drug candidate undergoing clinical development that contains the active ingredient used by Dr. Alan Shackelford in our initial targeted epilepsy population. SP1707 is being developed, ultimately, as a proprietary oral cannabinoid formulation that is amenable to being dosed once daily in patients.
Pre-Clinical Development and Real-World Patient Experience
SP1707 underwent a series of pre-clinical trials to test its effectiveness in epilepsy animal models and was successful at achieving results that warranted this drug candidate being moved forward in the development process.
The Company also evaluated patient data from Dr. Shackelford's real-world experience and discovered that similar human dose equivalents effective in the Company's animal trials were also effective at significantly impacting patients suffering from epilepsy in many patients. The Company is initially focusing on a specific seizure type for its first indication for SP1707. This indication is backed by ~356 patient treatment years of data.
Dr. Shackelford's real-world patient experience shows that in adult and pediatric patients with a specific seizure type, patients taking his cannabinoid-based treatment for at least one year resulted in clinically significant positive outcomes. The analysis of results (as of January 12, 2023) is shown in the table below. Published data from Jazz Pharmaceuticals plc.’s cannabidiol (“CBD”) based product, EPIDIOLEX®, is shown as a comparator.
Dr. Shackelford’s Real-World Experience Compared With Published Data from Jazz Pharmaceuticals plc.
Key messages from this analysis
1) Over 350 patient years of real-world data from Dr. Shackelford’s database in epilepsy. Adult and pediatric patients had been suffering from epilepsy for up to 46 years and 16 years, respectively, prior to being put on SP1707, suggesting overall, this has been a treatment for a refractory population.
2) Responder rates (meaning at least a 50% reduction in seizure frequency over a 1-year treatment period) were 71% in adults and 60% in pediatrics --- which is aligned with the response rates initially seen in FINTEPLA® (fenfluramine) open label studies and better than those observed with EPIDIOLEX®.
3) Importantly, 1 year seizure remission rates, which were similar between adults and children were 40-44% (greater than those observed with EPIDIOLEX®). Compared to baseline or expected 1 year seizure remission rates for this type of seizure, SP1707 elicited ~9-fold improvement.
4) The safety and tolerability data for SP1707 has been positive, with only 2% of adult patients and no children reporting any adverse events. This compares favorably to EPIDIOLEX® and other antiepileptic medicines.
While this real-world experience is on a relatively small data set, this data gives Shackelford a strong reason to believe that the Company is on the right path to develop a medicine that can potentially transform the way patients suffering from this devastating seizure type, across adult and pediatric seizure disorders, are treated. Our next step, once additional capital is raised, is to move into a clinical study.
Shackelford intends to follow-up its September 2022 FDA Pre-Investigational New Drug meeting with an Investigational New Drug submission to the FDA in the second half of 2023. This will allow the company to proceed to its planned Phase 2 trial pending FDA response and funding. The initial Phase 2 study of SP1707 in epilepsy patients will use an immediate-release oral formulation of SP1707 that will mimic the release characteristics of the cannabinoid that Dr. Shackelford used in his real-world experience treating patients.
In parallel to this safety and efficacy study, the Company plans to complete a clinical formulation screening / pharmacokinetic ("PK") bridging study, in order to facilitate novel formulation selection for the pivotal Phase 3 program. Upon successful conclusion of the Phase 2 and formulation screening / PK bridging studies, the Company will progress additional preparatory enabling activities required for its pivotal Phase 3 trial(s).
The Company has hired a reputable intellectual property firm as their IP counsel, who worked with the Company's management team to conduct an IP landscape review and a subsequent Freedom to Operate Opinion ("FTO"). The FTO opinion was received in Q2 2022. Shackelford submitted its full US nonprovisional patent and PCT application related to the use of SP1707 for the treatment of seizure disorders, either alone or as adjunctive therapy. This application included supportive preclinical data and real-world experience.
Undue reliance on forward-looking statements, and, in evaluating these forward-looking statements, readers should specifically consider various factors, including the risks outlined under the heading “Risk Factors” in documents we file with the SEC.
Invest in Shackelford
Shackelford Pharma’s Regulation A Financing is now open. Investors are required to fill out the form below to begin the investment process.
Please check your JUNK/SPAM email folder if your invitation email from Dealmaker/Shackelford does not arrive in your inbox.