GlobalData’s Investigative News team examines data generated by an internal model that combines machine learning and its proprietary algorithm. Likelihood of Approval (LoA) provides the likelihood of a drug gaining regulatory support; Phase Transition Success Rate (PTSR) indicates the likelihood of a drug advancing to the next stage of development. The model uses data points from individual drugs, clinical trials, regulatory milestones, company and financial databases.
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Albireo unveils data on liver disease
Albireo Pharma’s A3907 for liver disease saw its PTSR climb after the revelation of the first positive results from a phase I trial. The RPTP increased by 11 points in primary sclerosing cholangitis (PSC) and in primary biliary cholangitis (PBC), bringing their respective rates to 72% and 71%.
The Phase I study met its primary endpoints of no reported serious adverse events (AEs) or discontinuations due to treatment emergent AEs. The most common treatment-emergent AEs, all of which were mild, were abdominal symptoms such as loose stools. A3907 also had no clinically significant effects on hematology, clinical chemistry, physical examinations or electrocardiogram analyses.
Albireo announced its first results on December 16, and changes to the PTSR came into effect on December 20. The positive phase I did not affect the LoA of the drug, which was maintained at 0% and 3% for PSC and PBC.
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A3907 is an apical sodium-dependent bile acid transporter (ASBT) inhibitor that may inhibit ASBT expressed by cholangiocytes. Both CSP and PBC are chronic cholestatic liver diseases that can progress to end-stage liver disease over 10 to 20 years.
Roche makes progress in ulcerative colitis
Roche’s Efmarodocokin alfa for moderate to severe ulcerative colitis saw its PTSR jump nine points to 35% after completing its phase II trial. Efmarodocokin alfa targets the interleukin-22 receptor, generating potential anti-inflammatory effects.
ClinicalTrials.gov updated the Phase II trial (NCT03558152) from “active, not recruiting” to “completed” on December 20, and the PTSR change occurred on December 21. The 195-patient phase II was designed to compare efmarodocokine, also known as UTTR1147A, to Takeda Pharmaceutical’s Entyvio (vedolizumab) and a placebo.
The first part of the study focused on the induction of clinical remission of ulcerative colitis, while the second part tested the durability of remission. Phase II has as its primary endpoint the percentage of patients in clinical remission after eight weeks.
The completed phase II also boosted the drug’s LoA by three points to 11%. Efmarodocokin alfa is being investigated by Roche subsidiary Genentech.
Alzamend Neuro shares positive data on the first phase I line
Alzamend Neuro AL-001 for dementia associated with Alzheimer’s disease saw its PTSR jump 15 points to 73%. This follows the company’s announcement of positive results on December 17 from a phase I trial.
Phase I is designed to find an appropriate Phase II dose. The study data shows that AL-001, which has an oral lithium delivery system, is bioequivalent to lithium carbonate. Lithium carbonate is approved for bipolar disorder.
Phase I also shows that AL-001 and lithium carbonate had similar time curves on their respective plasma lithium concentration levels. AL-001 is an ionic co-crystal of lithium-salcylate-L-proline. The positive phase I also slightly increased the LoA of the drug by two points to 9%. The PTSR and LoA were updated on December 21.