Med Check: FDA, Anti-Amyloid Beta Vaccine, Clinical Trials of Psychedelics, and Lumryz

The Food and Drug Administration (FDA) has granted fast track status to ACI-24.060, AC Immune’s investigational anti-amyloid beta active immunotherapy vaccine for the treatment of Alzheimer’s disease, the company announced in June. The FDA fast track is a process designed to facilitate the development of medications that treat serious conditions and fill an unmet medical need and to expedite their review. ACI-24.060 is thought to enhance the formation of polyclonal anti-amyloid beta antibodies, which may then inhibit plaque accumulation and increase plaque clearance and potentially reduce or prevent Alzheimer’s disease progression.

The fast track designation was supported by data from the phase 1b/2 ABATE trial, a double-blind, placebo-controlled study assessing ACI-24.060 in patients with prodromal Alzheimer’s disease. Patients were randomized to receive injections of either ACI-24.060 or placebo at predefined time points over 48 weeks. Enrollment in the trial is ongoing, but early results showed that ACI-24.060 elicited an anti-amyloid beta antibody response in the low-dose cohort as soon as week 6, two weeks after the second injection. The trial also found that treatment was safe and well-tolerated, with no serious safety concerns reported.

In June the FDA published draft guidance for researchers investigating the use of psychedelic drugs for the potential treatment of medical conditions, including psychiatric or substance use disorders. The draft guidance covers studies on “classic psychedelics” such as psilocybin and lysergic acid diethylamide (LSD), as well as “entactogens” or “empathogens” such as methylenedioxymethamphetamine (MDMA).

The draft guidance offers recommendations on providing chemistry, manufacturing, and controls information to ensure proper identification, quality, purity, and strength of the investigational substance.

The draft guidance also advises researchers to study the ways that psychedelic drugs in development might interact with other medications; for example, chronic use of selective serotonin reuptake inhibitors (SSRIs) or monoamine oxidase inhibitors (MAOIs) may reduce the effect of psychedelic drugs, and chronic use of tricyclic antidepressants or lithium and acute use of SSRIs or MAOIs may potentiate psychedelic effects. The draft guidance also provides recommendations for assessing the abuse potential of an investigational substance.

The public comment period for the draft guidance ends on August 25. Submit comments.

Lumryz (sodium oxybate) is now available in the United States for treating cataplexy or excessive daytime sleepiness in adults who have narcolepsy, Avadel Pharmaceuticals announced in June.

The FDA approved the drug on May 1 based on the results of a phase 3 trial that included 212 patients with narcolepsy. Patients were randomly assigned to receive 4.5 g, 6.0 g, 7.5 g, or 9.0 g of Lumryz or placebo once nightly for 13 weeks.

Compared with patients who took placebo, those who took Lumryz experienced greater improvements in their symptoms at week 13 as measured by the Maintenance of Wakefulness Test, Clinical Global Impression of Improvement in daytime sleepiness, and mean weekly cataplexy attacks recorded on the Sleep and Symptom Daily Diary.

Lumryz is supplied in packets with granules that are mixed with water and taken as a single dose orally at bedtime. The dosage strengths include 4.5 g, 6 g, 7.5 g, and 9 g per packet. ■