Vancouver BC, Canada (February 6, 2023) — Shackelford Pharma Inc. (“Shackelford” or the “Company”), a pharmaceutical company developing medicines for neurological diseases with significant unmet patient needs, is pleased to share the compilation and retrospective analysis of additional real-world data backing its development of SP1707, a small molecule oral formulation targeting an undisclosed seizure type. SP1707 is the development name/number of the Company’s first drug candidate that contains the active ingredient used by Dr. Alan Shackelford. Ultimately, SP1707 is being developed as a proprietary oral cannabinoid formulation that is amenable to being dosed once daily in patients.
“This analysis of Dr. Shackelford’s real-world experience strongly supports our reason to believe we have a path to Phase 2 clinical trials,” stated Dr. Susan Learned, Head of R&D at Shackelford. “This exciting human efficacy and safety data will be used to support our Investigational New Drug (“IND”) submission to the FDA. The observed 9-fold improvement in 1 year seizure remission rates over what is typically expected in this seizure type gives us confidence that we may see clinically significant effectiveness when we progress into Phase 2. Importantly, this retrospective data seems to be tracking closely with what was observed with FINTEPLA® (fenfluramine) in open label studies and better than those observed with EPIDIOLEX®”.
Compiled from Dr. Shackelford’s real-world experience, the collected data shows that in adult and pediatric patients with a specific seizure type, patients taking his cannabinoid-based treatment for at least one year experienced clinically significant positive outcomes. The analysis of results (as of January 12, 2023) are shown in the table below. Published data from Jazz Pharmaceutical plc.’s cannabidiol (“CBD”) based product, EPIDIOLEX®, are shown as a comparator.
Key insights from this analysis:
1) Over 350 patient years of real-world data from Dr. Shackelford’s database in treating patients with 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 an approximate 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.
Dr. Shackelford’s Real-World Experience Compared with Published Data from Jazz Pharmaceuticals plc.
SP1707 | SP1707 | EPIDIOLEX® | EPIDIOLEX® | |
| Adult | Pediatric | Pediatric RCTs | EAP |
Demographics |
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Number of Patients | 82 | 13 | 61-76 per indication | 892 |
Male/Female | 47/35 | 8/5 | N/A | 464/428 |
SZ/Epilepsy Duration Prior to SP1707 Treatment | Mean= 15.55; Up to 46 yrs | Mean=6.6; Up to 16 yrs | <18 yrs | N/A (age range 0-75, median 12 yrs) |
Duration of Follow-Up (years) | 307 | 49 | ~14-20 | 1755 |
Disease Outcomes: Benefit Comparison |
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Indication | Undisclosed | Undisclosed | DS, LGS, TSC | TRE |
Responder Rate (>/=50% reduction in seizure frequency over 1 year period) | 71% (24/34) | 60% (6/10)) | ~45% @ 14 wks | 53% @48 wks |
1 YR Seizure Remission Rate (in patients with 1+ seizures in prior year with at least 1 year follow up post SP1707 treatment initiation) | 44% (15/34) | 40% (4/10) | 6-7% @14 wks | 11% @48 wks |
Expected 1 YR Seizure Remission Rate | 4-5% | 4-5% | 4-5% | 4-5% |
Increase in 1 YR Seizure Remission Rate over expected | ~9X | ~9X | ~1.5X | ~2X |
Disease Outcomes: Safety |
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Adverse Events | 2% (2/82) | 0% (0/13) | Up to 12% | 7% |
Key:
RCT: randomized controlled trial
EAP: expanded access program
N/A: not available
SZ: Seizures
DS: Dravet Syndrome
LGS: Lennox-Gastaut Syndrome
TSC: Tuberous Sclerosis Complex
TRE: Treatment Resistant Epilepsy
A Pre-Investigational New Drug Meeting with the FDA occurred in September 2022 and the Company intends to submit an Investigational New Drug submission 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.
Questions related to this announcement, or general inquires to the Company can be made by calling us directly at 1-888-377-4225 Ext.1., or by emailing us at info@shackelfordpharma.com. Additional company information is available on our website www.shackelfordpharma.com, where you are also be able to sign up to receive updates from Shackelford Pharma Inc. as we advance our clinical program.
On behalf of the Board of Directors
Mark Godsy, CEO
This news release does not constitute an offer to sell or a solicitation of an offer to buy any securities in the United States. In the United States under the Securities Act of 1933, any offer to sell securities must either be registered with the United States Securities and Exchange Commission (SEC) or meet certain qualifications to exempt it from such registration. An offering circular on Form 1-A was filed with the Securities and Exchange Commission (“SEC”) and was qualified by the SEC as of December 12, 2022, as amended. The offering of these securities is made only by means of an offering circular on Form 1-A.
About Shackelford Pharma Inc.
Based upon Dr. Alan Shackelford’s real-world experience, Shackelford Pharma, Inc., was formed to develop medicines for neurological diseases with significant unmet patient needs. Members of its world class R&D team collectively have over 320 years of drug development experience. This includes having developed 43 FDA approved drugs, 7 with peak annual revenue over $1B USD, and 6 anti-epileptic drugs. Shackelford’s first drug candidate, SP1707, is a small molecule, oral formulation targeting a specific seizure type.
Please visit us at www.shackelfordpharma.com
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