Maze Therapeutics Drug shows a potential for a higher asset acquired by Otsuka for $ 800 million

An experimental therapeutic drug of the labyrinth has increased the urinary excretion of compounds which are biological indicators of metabolic disease, the results of early clinical trials which suggest that the molecule has the best class potential in a rare disorder with few treatment options as well as the possibility of introducing a new approach to chronic kidney disease.

Phase 1 test controlled by placebo has registered 112 healthy adults and evaluated a range of doses of the study medication, MZE782. The main objective was to measure the safety and tolerability and the labyrinth of the results reported on September 11 has not shown any serious adverse event or complications related to treatment leading to the stopping of the study under study. The secondary and exploratory evaluation criteria of the trial included urine measures as well as the measures of the renal function. These are the results that have excited investors.

The MZE782 of Maze is a small molecule designed to selectively inhibit SLC6A19, a gene that code for a transport protein which plays a key role in the absorption and reabsorption of intestinal and renal reabsorption of phenylalanine, an amino acid found in certain foods. The rare disease that the labyrinth aims to treat is phenylcetonuria, in which phenylalanin accumulates in the body and causes cognitive and behavioral problems. The accumulation stems from a hereditary deficiency of an enzyme necessary to break the amino acid. Phenylcetonuria is mainly managed with food changes to limit the contribution in phenylalanine. Biomarin Pharmaceutical Markets Two drugs approved by the FDA for rare disorder.

The results of phase 1 of Maze’s medication showed a dose-dependent excretion of phenylalanin and glutamine. Maze said that the higher excretion of these amino acids confirms that MZE782 has hired and inhibited SLC6A19. A single dose of 960 mg of MZE782 led to an increase of 39 times of the urinary excretion of phenylalanine over 24 hours. The company also said that an increase of 42 times in the urinary excretion of amino acid over 24 hours on day 7 was observed in the group which received the dose of 240 mg of the drug under study twice a day.

Multiple-ascending cohorts of the study also evaluated the estimated glomerular filtration rate (EGFR), a measure of the way the kidneys filter blood. The MZE782 led to an initial drop in the EGFR dependent on the dose over seven days which was similar to what was observed with the SGLT2 and Raas inhibitors, two classes of drugs currently used to treat chronic kidney disease. Maze said that with other kidney drugs, this initial decline is correlated with a slower rate of drop in EGFR and a better function of kidney function over longer periods in patients with chronic kidney disease.

With the positive results of phase 1, Maze said that he now planned to advance MZE782 to two phase 2 clinical trials of concept proof. The study of phenylcetonuria will measure the reductions of phenylalanine in the blood. The chronic study of kidney disease will measure the reduction of urinary proteins indicating disorder. The two studies should start in 2026.

In a note sent to investors, Leerink Partners’ analyst Joseph Schwartz said that the increase in phenylalanin excretion not only exceeds the previously established objective labyrinth, he also exceeds the measures obtained by JNT-57, the SLCA19 inhibitor which is the main asset of Jnana Therapeutics. Otsuka Pharmaceutical saw enough promises in this drug candidate to acquire JNANA last year for $ 800 million in advance with an additional $ 325 million linked to the realization of the milestones. The drug JNANA is currently in phase 3 tests in phenylcetonuria, but Schwartz considers the labyrinth medication as a strong competitor in this disease as well as on a chronic kidney disease.

“Overall, these results with the MZE782 of Maze suggest a profile the best in class, in our opinion, and we believe that this program will begin to obtain more credit from investors,” said Schwartz.

Maze followed data reading with a private investment that raised $ 150 million from new and previous investors. Biotechnology said it would use these products with its existing capital to finance the planned tests of phase 2 of MAZ782. The capital will also support phase 2 tests during the most advanced program of Maze, MZE829, which is in development to treat patients with renal disease mediated by APOL1.

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