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PharmAust Gets a Nod from the FDA for MND Therapy Study

PharmAust Limited

The renowned clinical-stage biotech company PharmAust (ASX: PAA) is all set for a new program focusing on its trials for the primary drug candidate Monepantel (MPL). 

The company had conducted a Phase 1 study of monepantel to treat motor neuron disease (MND)/Amyotrophic Lateral Sclerosis (ALS). The Memory and Drug Enhancement (MEND) study aimed to get a green flag for the recommended Phase 2 dose based entirely on safety and preliminary efficacy data. PharmAust has already completed the four planned dose levels. 

The company has eagerly awaited the open-label extension (OLE) study, which is expected to begin early in 2024. This study will generate valuable safety and efficacy data for the coming months as the trials on the patients continue for better health initiatives. 

Now, the time has finally come with PharmAust getting positive feedback from the US Food and Drug Administration (FDA) for the next study phase. 

The company plans to use the data from the Phase 1 MEND study to commence the upcoming Phase 2 study. This has also supported its applications for an investigational new drug application and orphan drug designation (ODD) with the FDA.

PharmAust Latest News

The US FDA has proposed certain conditions for PharmAust (ASX: PAA) to grant the ODD to treat MND/ALS. This includes providing additional clinical data that proves that the particular drug that they are testing is effective in treating MND/ALS. 

PharmAust had sent the application for ODD to the FDA in November 2023. The company had already presented pre-clinical mechanistic data to prove that MPL can help treat the diseased cells in the human body. 

The FDA has also given PharmAust the liberty to use the old ODD request for the amendment instead of putting more effort into sending another fresh application. 

The company’s preclinical programs were a success. The results showed that MPL could activate molecules to help develop the MND therapeutic drug. If the study results come back positive, the MND/ALS patients can heave a sigh of relief because the drug would help prevent the loss of motor neurons in the human brain. 

Future Plans of PharmAust Limited ASX

PharmAust is already on the verge of releasing its Phase 1 MEND study results within the coming quarter. The company announced its collaboration with Berry Consultants, a leading clinical study design specialist, in January 2024. Their partnership aims to carry forward phases 2 and 3 of the MND therapy study with the help of Monepantel. 

However, Berry Consultants will be helping only with the statistical analysis of the MEND study data’s initial phase.

For the unversed, the Phase 1 MEND study by PharmAust involved 12 patients who were suffering from MND/ALS. This study came to an end in December 2023 with positive results. That is because the patients expressed their willingness to continue receiving MPL as a part of their therapy for the condition. These 12 individuals will also be included in the upcoming open-label extension study from February 2024. 

Future Plans of PharmAust (ASX: PAA)

The MPL therapy study is not the only program in PharmAust’s pipeline. The company’s clinical trials for other issues continue with collaborations and feedback from their worldwide partners. For instance, PharmAust has already announced its plan to open another investigational new animal drug (INAD) file to use the same MPL drug to treat canine B-cell lymphoma. 

The sponsors of this animal drug, including PharmAust, are on the verge of gathering pilot data to discuss the development with the FDA and get approval for the study. 

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