Alzheimer's Disease Research and Development - Synaptogenix is focused on developing Bryostatin-1 for Alzheimer's disease, with ongoing clinical trials and pre-clinical evaluations for other neurodegenerative diseases[13] - A Phase 2 exploratory trial enrolled 147 patients, showing a net improvement of 2.6 points in the SIB score for the 20 µg dose compared to placebo in the Completer population[15][17] - The 20 µg dose demonstrated a sustained improvement in cognition, with a significant increase of 3.59 points in the mITT population at week 15 compared to controls[24] - The confirmatory Phase 2 trial did not achieve statistical significance on the primary endpoint, with an average increase of 1.3 points for the Bryostatin-1 group at week 13[32][33] - The Phase 2 trial results indicated that Bryostatin-1 could potentially treat Alzheimer's disease and help reduce its progression[22] - Neurotrope announced a statistically significant improvement of 4.8 points in the mean SIB at week 13 for the Bryostatin-1 treatment group, with a p-value of < 0.0076[35] - The extended confirmatory Phase 2 study of Bryostatin-1 did not achieve statistical significance on the primary endpoint, with an average increase in SIB total score of 1.4 points for the Bryostatin-1 group[38] - Statistical significance was achieved in nearly all pre-specified secondary endpoints for the most advanced and severe AD patient population (MMSE: 10-14) with a p-value of < 0.05[38] - The activation of the PKC ε – BDNF pathway in central nervous system neurons may improve neuronal vitality and function, potentially enhancing memory and cognition in AD patients[58] - Treatment with Bryostatin-1 for 12 weeks in genetically modified rodents demonstrated promotion of new synapse growth and preservation of existing synapses, reversing the decrease of PKC ε[67] Financial Overview and Funding - The total estimated budget for the confirmatory trial was approximately $7.8 million, with total expenses incurred of about $7.6 million[31] - A $2.7 million award from the National Institutes of Health was granted to support an additional Phase 2 clinical study focused on the moderate stratum showing improvement in the 203 study[36] - The total estimated budget for the current Phase 2 trial services is approximately $11.0 million, with an estimated net cost to Neurotrope of $9.3 million after NIH funding[37] - The company incurred approximately $11.2 million in cumulative expenses for the Phase 2 clinical trial of Bryostatin-1 as of December 31, 2023, with an estimated net budgeted cost of $8.6 million after a $2.7 million NIH grant[96] - The company has incurred approximately $1.6 million in cumulative expenses for the 2022 Study as of December 31, 2023[99] - The total estimated budget for the 2022 Study services was approximately $2.0 million, but the agreement was terminated in December 2022[98] Collaborations and Licensing - Synaptogenix has entered into licensing agreements with Stanford University for the use of synthetic bryostatin in neurodegenerative disease therapeutics[14] - The company has licensed technology from Stanford for the use of bryostatin derivatives in treating central nervous system disorders, with obligations including a $10,000 annual maintenance fee and royalties of 3% on net sales[78] - The company maintains an exclusive, non-transferable, royalty-bearing right to develop certain products for therapeutic applications for Alzheimer's disease and other cognitive dysfunctions under the CRE License[101] - The company has licensed an extensive intellectual property portfolio from CRE, including issued patents and pending applications for key pharmaceutical markets[108] Regulatory Environment - The FDA has placed the development of the IND for Fragile X on clinical hold pending further analytics[41] - The FDA's approval process for new drugs includes rigorous preclinical testing and clinical trials to demonstrate safety and efficacy[117] - The FDA charges an annual program fee exceeding $415,000 per product for approved NDAs[128] - The FDA's internal review goals are six months for priority NDAs and ten months for regular NDAs from the filing date[129] - The FDA may grant accelerated approval based on a surrogate endpoint that predicts clinical benefit for serious conditions[141] - The FDA may issue a complete response letter outlining deficiencies in an NDA submission, requiring additional testing or information[145] Market Potential and Competitive Landscape - Over 50 million people worldwide are estimated to have AD or other forms of dementia in 2023, indicating significant commercial potential for effective therapies[51] - The global sales of major prescription drug therapies for Alzheimer's Disease (AD) were approximately $2.8 billion in 2022, with a projected growth to approximately $6.8 billion by 2032, reflecting a compounded annual growth rate (CAGR) of 9.3%[55] - The markets for drugs to treat AD are primarily in developed regions, including North America, Europe, and Japan[54] - The company is pursuing a unique mechanism involving PKC ε activation to treat Alzheimer's disease, with no direct competitors currently in this field[206] Corporate Structure and Governance - The company has five full-time personnel, including three executive officers, and maintains good relations with employees and consultants[207] - The corporate headquarters is located in New York, with a lease expiring on June 30, 2024, at a cost of approximately $5,900 per month[209] - The company has no ongoing legal proceedings against it[210] - The company was incorporated in Delaware in 2012 and underwent a structural separation from Neurotrope in December 2020[211]
Synaptogenix(SNPX) - 2023 Q4 - Annual Report