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AmideBio receives grant to advance work on hyperinsulinism

BOULDER — AmideBio LLC, a biopharmaceutical company in Boulder that makes peptides used in research to discover therapies for diabetes, has received a $338,387 grant that it will use to further develop a treatment for hyperinsulinism.

Hyperinsulinism is a disease in which infants and newborn babies suffer from persistent hypoglycemia, or low blood-sugar levels, that can lead to seizures and brain damage.

AmideBio received the two-year grant from the National Institute of Health through a Small Business Innovation Research grant.

“This grant will allow us to further develop and test AmideBio’s stable glucagons for treatment of patients with hyperinsulinism who fail to respond to current drug treatments,” said Misha Plam, CEO of AmideBio. “This therapeutic solution offers the potential for babies to avoid traumatic surgeries including a total pancreatectomy, providing the chance for them to live more normal lives.”

At a BizWest CEO Roundtable on life sciences in April, Plam said the company had begun working on a therapy to treat babies whose pancreases create too much insulin, driving down blood-sugar levels. The condition is considered an “orphan disease” because it affects fewer than 200,000 young people. Plam said developing a drug for an “orphan disease” is appealing because it requires less investment. But obtaining funding to advance the drug had been negatively affected by government budget cuts.

Now, with the grant in hand, AmideBio plans to develop and produce a series of glucagons and test them in hopes of identifying one suitable for clinical testing for long-term treatment. Glucagon is a peptide hormone produced by the pancreas that raises the concentration of glucose and fat in the bloodstream to offset too much insulin.

Glucagon has been shown to be an effective treatment for hyperinsulinism, but its inherent instability has limited its use due to complications associated with its administration through pumps, according to a statement by AmideBio. Using AmideBio’s BioPure process, the company has produced glucagon analogs that have in-use stability of at least six months and can be stored for extended periods of time.

AzurRx BioPharma and Mayoly-Spindler Announce Positive MS1819-SD Phase II Data in Exocrine …

BROOKLYN, NY, USA I September 27, 2017 I AzurRx BioPharma Inc.(NASDAQ:AZRX) (“AzurRx” or the “Company”), today provided an update on the first six treated patients in its ongoing Phase IIa trial of MS1819-SD, a recombinant lipase, for the treatment of exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis.

Importantly, the company observed both clinical activity and a clear dose response, where the highest MS1819-SD dose cohort showed greater than 21% improvement in CFA (coefficient of fat absorption) in evaluable patients.  Additionally, maximal absolute CFA response to treatment was up to 57%, with an inverse relationship to baseline CFA. Favorable trends were also observed on other evaluated endpoints, such as Bristol stoolhttp://boulderpeptide.org/2017/09/27/azurrx-biopharma…data-in-exocrine/ scale, number of daily evacuations and weight of stool, and these were consistent with the CFA results.

With regard to safety, no serious adverse events or notable mild to moderate events have been reported in the open-label, ascending dose Phase IIa trial.  Other markers relating to nutritional status including patients’ plasma albumen were unchanged with treatment.  Similarly, fecal nitrogen assessments showed favorable trends.

“We are encouraged by these Phase IIa data,” said Thijs Spoor, CEO of AzurRx BioPharma.  “They further confirm the activity of MS1819-SD and also demonstrate its dose response characteristics.  Additionally, secondary efficacy endpoints are consistently aligning with the CFA data and the safety profile of MS1819-SD remains favorable.”

Phase IIa Study
The open-label, dose escalation Phase IIa study is being conducted in Australia and New Zealand and has been designed to enroll 12-15 patients with EPI caused by chronic pancreatitis.

The primary objective of the Phase IIa study is to investigate the safety of escalating doses of MS1819-SD in patients with chronic pancreatitis. The secondary objective is to investigate the efficacy of MS1819-SD in these patients by analysis of the coefficient of fat absorption and its change from baseline. Safety is being assessed at the end of each treatment period with particular attention paid to immunoallergic effects, digestive symptoms and clinical laboratory tests.

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