Adrulipase Alfa Likely Global Name for CF Non-porcine PERT Candidate

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by Marisa Wexler, MS |

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The World Health Organization (WHO) proposed a compound name to better identify MS1819, an investigational therapy for exocrine pancreatic insufficiency in people with cystic fibrosis (CF), with scientists globally.

MS1819 may soon be called “adrulipase alfa.” The WHO is expected to make a final decision on the candidate’s International Nonproprietary Name (INN) by November.

“Assignment of an INN signifies that a pharmaceutical substance has reached a point in its development where it is now beneficial to specify to pharmacists and medical doctors, via nomenclature, the substances contained in a preparation,” James Sapirstein, the president and CEO of AzurRx BioPharma, the potential treatment’s developer, said in a press release.

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Exocrine pancreatic insufficiency, or EPI, occurs when the thick mucus that is characteristic of CF builds in the ducts of the pancreas, preventing the pancreas from releasing enzymes that are needed to properly digest foods, especially fats. The current standard treatment for EPI is porcine pancreatic enzyme replacement therapy (PERT), which involves administering pig-derived versions of the missing enzymes to aid in digestion.

MS1819 is a lipase — a fat-cleaving enzyme — that aims to improve fat digestion in CF patients with EPI. Notably, the enzyme is derived from yeast cells; MS1819 does not contain animal products.

Earlier this year, AzurRx reported mixed top-line results from a Phase 2 clinical trial, called OPTION 2 (NCT04375878), which compared the safety and efficacy of MS1819 against standard PERT. MS1819 alone had a good safety profile, but it was not as effective as traditional PERT at improving fat absorption in most participants.

Recently, the company shared top-line results of another Phase 2 trial (NCT04302662), which tested MS1819 in combination with porcine PERT in 20 CF patients with severe EPI. Findings showed that MS1819 plus PERT, given for 15 days, improved fat absorption — the trial’s main goal — increased mean body weight, and lowered stool weight. No serious adverse events were reported.

“The assignment of adrulipase alfa as the proposed International Nonproprietary Name for MS1819 is an important step in the ongoing development of this drug technology,” Sapirstein said.

“For MS1819, we view this as further validation of the technology. It is a clear point of differentiation as we continue the drug’s development s a potential treatment for EPI that could improve upon the current standard of care, porcine-derived pancreatic enzyme replacement therapy,” he added.

AzurRx also is working on an enteric-coated microbead formulation of MS1819, one designed to allow it active medication (the fat-digesting enzyme) to more effectively bypass the harsh, acidic environment of the stomach and get to the small intestine, where the bulk of digestion takes place.

The company expects to complete development of the new formulation by year’s end.