tricaprilin (CER-0001) / Cerecin - LARVOL DELTA

Response to Aanwat et al. on chow et al. Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study JONS July 22, 2024. (PubMed, J Neurol Sci) - No abstract available

Journal • P2 data • CNS Disorders • Migraine • Pain

Letter to the Editor: Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study. (PubMed, J Neurol Sci) - No abstract available

Journal • P2 data • CNS Disorders • Migraine • Pain

Tricaprilin Phase 3 ALTER-AD (Alternative-Alzheimer Disease) Study (clinicaltrials.gov) - P3 | N=535 | Not yet recruiting | Sponsor: Cerecin | Trial completion date: Jan 2028 ➔ Aug 2028 | Trial primary completion date: Jan 2027 ➔ Aug 2028

Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • GFAP • NEFL • TARDBP

Tricaprilin Infantile Spasms Pilot Study (clinicaltrials.gov) - P1 | N=8 | Completed | Sponsor: Cerecin | Active, not recruiting ➔ Completed | Trial completion date: Jan 2025 ➔ Dec 2023 | Trial primary completion date: Jan 2025 ➔ Dec 2023

Trial completion • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy

Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study. (PubMed, J Neurol Sci) - P2 | "Results suggest positive directional promise over 2-3 months for CER-0001. A new formulation will be used for larger, fully powered phase 2/3 studies."

Journal • P2 data • CNS Disorders • Gastrointestinal Disorder • Migraine • Pain

Tricaprilin Phase 3 AD Study (clinicaltrials.gov) - P3 | N=535 | Not yet recruiting | Sponsor: Cerecin | Trial completion date: Jan 2027 ➔ Jan 2028 | Initiation date: Jan 2024 ➔ Jan 2025 | Trial primary completion date: Jan 2026 ➔ Jan 2027

Trial completion date • Trial initiation date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

Tricaprilin, an Investigational Ketogenic Drug in Refractory Infantile Spasms (AES 2023) - "Positive safety results and benefit observed in some subjects in this Phase 2a Tricaprilin study for the treatment of IS and warrant continued investigation."

Atrial Fibrillation • Cardiovascular • CNS Disorders • Epilepsy • Pediatrics • Pulmonary Disease • Respiratory Diseases

AC-OLE-01-VA Tolerability Study in Healthy Participants Under Different Dosing Conditions (clinicaltrials.gov) - P1 | N=18 | Completed | Sponsor: Cerecin | Recruiting ➔ Completed

Trial completion • Alzheimer's Disease • CNS Disorders

Tricaprilin Liquid Formulation PK Study (clinicaltrials.gov) - P1 | N=71 | Terminated | Sponsor: Cerecin | Recruiting ➔ Terminated; Part 1 was completed, Part 2 and Part 3 of the study were not conducted under this study protocol due to subject recruitment difficulty.

Trial termination • Alzheimer's Disease • CNS Disorders

A Phase 1, Two-part, Randomized Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a Liquid Formulation of Tricaprilin and Matching Placebo in Healthy Subjects (clinicaltrials.gov) - P1 | N=45 | Completed | Sponsor: Cerecin | Recruiting ➔ Completed | Trial completion date: Mar 2023 ➔ Oct 2022

Trial completion • Trial completion date • Alzheimer's Disease • CNS Disorders

Tricaprilin Infantile Spasms Pilot Study (clinicaltrials.gov) - P1 | N=8 | Active, not recruiting | Sponsor: Cerecin | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2023 ➔ Jan 2025 | Trial primary completion date: Mar 2023 ➔ Jan 2025

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy

Clinical Trial Data of Novel Metabolic Mechanism in Migraine: CER-0001, a Ketogenic Investigational Agent (PRNewswire) - P2 | N=83 | RELIEF (NCT04437199) | Sponsor: Cerecin | "Cerecin Inc...presented encouraging findings from a recent study on CER-0001 (tricaprilin)...indicating that certain individuals suffering from migraines may experience benefit. The data will be used to inform future studies. The study's findings were highlighted in a poster presentation at the 65th Annual Society Meeting of the American Headache Society, held in Austin, Texas from June 15-18, 2023....The primary endpoint was not met due to lack of statistical power, however, an efficacy signal was noted in sensitivity analyses. Some patients opted to enter the CER-0001 Compassionate Access Program which provides continued access to CER-0001 (tricaprilin) for up to one year after completion of the clinical study. Given that metabolic factors have been implicated in migraine etiology, the induction of ketosis via ketogenic drugs like tricaprilin could potentially offer an innovative approach to managing this condition."

P2 data • CNS Disorders • Migraine • Pain

Metabolic Mechanism in Migraine: Tricaprilin, a ketogenic agent (AHS 2023) - "Results of the pilot suggest directional promise over 2-3 months for tricaprilin, and a new formulation will be used for larger, fully-powered Phase 2/3 studies."

CNS Disorders • Migraine • Pain

Tricaprilin Phase 3 AD Study (clinicaltrials.gov) - P3 | N=535 | Not yet recruiting | Sponsor: Cerecin

New P3 trial • Alzheimer's Disease • CNS Disorders

A Phase III Multi Regional Clinical Trial (MRCT) of Tricaprilin in Mild to Moderately Severe Probable Alzheimer's Disease With Optional Open Label Extension (clinicaltrials.gov) - P3 | N=0 | Withdrawn | Sponsor: Cerecin | N=344 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Alzheimer's Disease • CNS Disorders • APOE

AC-OLE-01-VA Tolerability Study in Healthy Participants Under Different Dosing Conditions (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Cerecin | Not yet recruiting ➔ Recruiting

Enrollment open • Alzheimer's Disease • CNS Disorders

Cerecin received US FDA clearance to study CER-0001 under an Investigational New Drug (IND) application for Infantile Spasms (GlobeNewswire) - "Cerecin Inc...announced that they had received authorization from the US Food and Drug Administration for an Investigational New Drug (IND) application for CER-0001 in IS, also referred to as West Syndrome. CER-0001 is an investigational, oral drug designed to induce a state of ketosis and address the metabolic deficit in the brains of children with IS, a rare and serious form of childhood epilepsy....Cerecin’s pilot study of CER-0001 is ongoing and will have recruited the 10th and final patient by 1H 2023. Patients enrolled in the current study are children between the ages of 3 and 36 months. The Company will plan a Phase 2 study by 2H 2023."

Enrollment status • IND • New P2 trial • CNS Disorders • Epilepsy

Modeling digestion, absorption, and ketogenesis after administration of tricaprilin formulations to humans. (PubMed, Eur J Pharm Biopharm) - "A partial parameter sensitivity analysis elucidated various influences on the plasma ketone levels that are mainly responsible for the therapeutic effects of tricaprilin. Based on our findings, we concluded that dispersibility and lipolysis of tricaprilin together with the gastric emptying patterns are limiting ketogenesis, while other steps such as the conversion of octanoic acid to ketone bodies play a minor role only."

Journal • Alzheimer's Disease • CNS Disorders

AC-OLE-01-VA Tolerability Study in Healthy Participants Under Different Dosing Conditions (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Cerecin

New P1 trial • Alzheimer's Disease • CNS Disorders

Results of Pilot Study of CER-0001 in Migraine support advancement into a pivotal global Phase 2/3 study (GlobeNewswire) - P2 | N=83 | NCT04437199 | Sponsor: Cerecin | "Cerecin Inc...announced that data from RELIEF, a double-blind, randomized, placebo-controlled study investigating the safety and efficacy of twice daily administration of CER-0001 for the prevention of migraine, found an efficacy signal that warrants further development in a pivotal Phase 2/3 study....At month three, the study did not meet statistical significance due to power but efficacy was seen at earlier time points. The study was highly informative and provided information for the design of the next study....The Company will present the full study data at the CNS Summit, in Florida, United States, on November 19, 2022. Cerecin has received authorization from the U.S. Food and Drug Administration (FDA) to study CER-0001 in migraine under an Investigational New Drug (IND) application. This will allow the Company to conduct the Phase 2/3 study globally with trial sites in the United States..."

IND • New P2/3 trial • P2 data • CNS Disorders • Migraine

Tricaprilin Phase 2 Pilot Study in Migraine (clinicaltrials.gov) - P2 | N=83 | Completed | Sponsor: Cerecin | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Migraine

A Phase 1, Two-part, Randomized Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a Liquid Formulation of Tricaprilin and Matching Placebo in Healthy Subjects (clinicaltrials.gov) - P1 | N=45 | Recruiting | Sponsor: Cerecin | Not yet recruiting ➔ Recruiting

Enrollment open • Alzheimer's Disease • CNS Disorders

A Phase 1, Two-part, Randomized Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a Liquid Formulation of Tricaprilin and Matching Placebo in Healthy Subjects (clinicaltrials.gov) - P1 | N=45 | Not yet recruiting | Sponsor: Cerecin

New P1 trial • Alzheimer's Disease • CNS Disorders

Tricaprilin Infantile Spasms Pilot Study (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Cerecin | Trial completion date: Aug 2022 ➔ Mar 2023 | Trial primary completion date: Aug 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • CNS Disorders

Cerecin raises $40 Million Series II and IIA financing to support its programs in neurological disorders (Yahoo Finance) - "Cerecin Inc...today announced it has raised USD $40 million in an oversubscribed round of financing. A Series II investment was initially made by long-term investor, Nestle. In follow up to this round, Series IIA was led by SK Securities, Hana Financial Investment, Partners Investment, Arche Investment, and K&T Investment....The company initiated a pilot study to assess safety, tolerability and efficacy in infantile spasms, and has also commenced a phase 2 study in migraine prevention. The proceeds of this financing will fund the expansion of the company’s current studies and support the planning and initiation of a global phase 3 study in Alzheimer’s disease....Over the next 18 months, Cerecin anticipates achieving several inflection points and milestones, including the readout of current trials and initiation of later stage studies in epilepsy, migraine and Alzheimer’s disease."

Financing • New P3 trial • New trial • P2 data • Alzheimer's Disease • CNS Disorders • Epilepsy • Migraine • Pain