AzurRx BioPharma Dips on Phase 2 Trial Results for MS1819

Shares of AzurRx BioPharma (NASDAQ:AZRX) took a substantial hit on Wednesday (September 25) following the release of data from a Phase 2 clinical trial of MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency.

The Phase 2 OPTION trial of MS1819 demonstrated that the primary endpoint of coefficient of fat absorption (CFA) was similar to a previous Phase 2 study of the same dose of MS1819 in patients with chronic pancreatitis.

As part of an agreement with the US Food and Drug Administration (FDA), both studies dosed 2 grams per day of the treatment as a bridging dose.

While no statistical significance was demonstrated in the Phase 2 study, the company said these results show that MS1819 was still effective in that 50 percent of the patients demonstrated enough CFA to achieve non-inferiority with porcine enzyme replacement therapies (PERT). PERTs are digestive enzymes taken as tablets that help with digesting fats and proteins.

Despite the company’s positive outlook on the results, shares of AzurRx BioPharma dipped 21 percent on Wednesday from the previous close on Tuesday (September 24) to close the session at US$0.70.

“We are thrilled to have seen such favorable safety and meaningful efficacy data in this Phase (2) study.  Importantly, the data were consistent and confirm results seen in prior clinical studies,” Jim Pennington, chief medical officer of AzurRx, said in a press release. “We are eager to move forward with what we consider a logical and promising next trial to increase the dose for CF patients.”

The open label Phase 2 Option trial took place at 14 sites across the US and Europe with 32 patients aged 18 and older participating. They were randomly assigned to either MS1819 for three weeks or the pre-study dose of PERT for three weeks.

Once the samples were collected, patients then moved over to the alternative method of treatment. AzurRx BioPharma noted that CFA in the MS1819 cohort was 56 percent compared to 86 percent of the PERT treatment cohort.

“With these data showing MS1819 to be safe and to have the potential to support fat absorption, we have considerable reason to be optimistic for the next steps in non-porcine enzyme development,” said Michael Konstan, principal investigator for the trial and a professor of pediatrics at the Case Western Reserve University School of Medicine.

From here, the company plans to meet with the FDA before the end of the year to plan for a potential Phase 2b/3 trial.

Cystic fibrosis is a genetic disorder that results in a defective protein, leading to negative effects on sweat and mucus production and the body’s cells and tissues.The most serious problems associated with cystic fibrosis are found in the lungs and lead to respiratory problems and potentially to lung infections.

As noted by Healthline, roughly 90 percent of people who are diagnosed with cystic fibrosis eventually develop exocrine pancreatic insufficiency, which is when pancreatic enzymes are blocked from entering the small intestine by thick mucus.

By 2025, the overarching cystic fibrosis treatment market is projected to reach US$7.6 billion, up from US$2.1 billion in 2015, according to a report from GlobalData.

Fueling that growth will be treatments such as Vertex Pharmaceuticals’ (NASDAQ:VRTX) Orkambi, which first received FDA approval in 2015 for patients aged 12 and older.

Since then, it has been approved to treat children aged between two and five with cystic fibrosis. As a leader in the cystic fibrosis space, the company also submitted a new drug application to the FDA in July for a triple combination regimen of its treatment VX-445.

That being said, GlobalData’s report indicates that additional cystic fibrosis treatments will enter the market during the forecast period — potentially including AzurRx BioPharma’s treatment options.

Despite the share price slump on Wednesday, AzurRx BioPharma is currently ranked a “moderate buy” on TipRanks based off of two analyst ratings. The company has an average price target of US$5.50, which is 682.47 percent higher than its current price.

Year-to-date, shares of AzurRx BioPharma are down 31.54 percent.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.