Sept. 9 Clinical Quick Takes: Translate to discontinue enzyme deficiency candidate; plus Janssen, Neurotrope, AMT and Cassava

Translate dropping enzyme deficiency program
Translate Bio Inc. (NASDAQ:TBIO) fell $0.73 to $9.25 in after-hours trade Monday after the company said that it is discontinuing development of MRT5201 for ornithine transcarbamylase deficiency based on preclinical safety and PK profiles for the lipid nanoparticle-encapsulated mRNA. Translate will prioritize pulmonary programs including MRT5005, an mRNA therapy encoding functional CFTR in a Phase I/II cystic fibrosis trial for which data are expected next year.

Spravato reduces depression in suicidal patients
Janssen Pharmaceuticals Inc.'s Spravato esketamine met the primary endpoint in the Phase III ASPIRE I and II trials to treat moderate to severe major depressive disorder patients with suicidal intent. Spravato plus standard of care therapy reduced depression symptoms, measured on the Montgomery-Åsberg Depression Rating Scale (MADRS), vs. placebo plus SOC 24 hours after the first dose (p=0.006 in both trials). The Johnson & Johnson (NYSE:JNJ) unit presented data at the European College of Neuropsychopharmacology meeting in Denmark. Spravato is approved for treatment-resistant depression.

Neurotrope plummets on Phase II Alzheimer’s failure
Neurotrope Inc. (NASDAQ:NTRP) dropped $3.40 (77%) to $1, shedding about $40 million in market cap, after revealing bryostatin-1 missed the primary endpoint of reducing Severe Impairment Battery (SIB) total score from baseline to week 13 vs. placebo in a Phase II trial to treat moderate to severe Alzheimer’s disease. The PKCE activator also missed on secondary endpoints evaluating change in SIB score at weeks 5, 9 and 15.

AMT debuts with Phase I ulcerative colitis trial start
Applied Molecular Transport Inc. (South San Francisco, Calif.) announced its official launch and said it dosed the first ulcerative colitis patient in a Phase Ib trial of AMT-101. AMT also said the IL-10 fusion protein was well tolerated in the first 20 healthy volunteers in a Phase Ia trial. The company's platform shuttles proteins across the intestinal epithelium by co-opting a strategy and molecule used by bacteria (see "Hitchhiker's Guide to the Gut").

Cassava to start Alzheimer’s Phase IIb based on biomarker data
Cassava Sciences Inc. (NASDAQ:SAVA) reported that twice daily PTI-125 for 28 days led to lower CSF levels of neuroinflammation, pathology and neurodegeneration biomarkers vs. baseline (p<0.001 for all comparisons) in all 13 mild to moderate Alzheimer’s patients in its first-in-patient Phase IIa trial, which did not evaluate cognition. The company plans to start a Phase IIb trial measuring AD biomarkers this quarter and to present full data from the Phase IIa trial at Clinical Trials on Alzheimer’s Disease (CTAD) meeting in San Diego in December.

Targets: CFTR - Cystic fibrosis transmembrane conductance regulator; IL-10 - Interleukin10; PKCE - Protein kinase C ε

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