ProMetic's PBI-4050 Significantly Reduces Liver Fibrosis in Patients with Alström Syndrome

  • Significant reduction of established liver fibrosis demonstrated in all patients that completed 12 weeks of PBI-4050 treatment
  • Liver enzymes reduced to within normal ranges in all patients that completed 12 weeks of PBI-4050 treatment
  • Alström syndrome clinical program to be expanded to include further sites throughout Europe and North America

LAVAL, QC, Oct. 12, 2016 /CNW Telbec/ - ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) ("ProMetic" or the "Corporation") announced today that the Drug Safety Monitoring Board ("DSMB") recommended that patient enrolment should continue in the Corporation's ongoing Alström syndrome phase 2 clinical trial. This recommendation follows the DSMB's review of the safety data accumulated in the first eight (8) Alström syndrome patients that had received treatment with PBI-4050. The DSMB determined that no safety or tolerability issues had been observed in these patients.

The early efficacy results in this phase 2, open-label study demonstrate that the first five (5) patients (100%) who completed 12 weeks of treatment with PBI-4050 had a significant reduction of liver fibrosis, as measured by transient elastography (FibroScan®). This reduction was also sustained for the first patient, who continued to show reduced fibrosis after having completed 24 weeks of treatment. The initial efficacy results also demonstrate that those patients with the most elevated liver enzymes at baseline (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and alkaline phosphatase) [ALP], had a significant reduction in all of these enzymes, to within normal ranges, after completing 4, 8 and 12 weeks of treatment with PBI-4050.

Dr. Tarekegn Geberhiwot, principal investigator of the clinical trial from the Department of Endocrinology and Metabolism, Queen Elizabeth Hospital, Birmingham, UK commented: "PBI-4050 has shown encouraging findings in patients with Alström Syndrome, being well tolerated and safe for up to 24 weeks. There have been beneficial effects observed in glycaemic control and early potential anti-fibrotic effects on the liver. The rapidity of the effect is remarkable, with a reduction in the elevated levels of liver enzymes apparent within the first 4 weeks, and significant reductions in FibroScan scores within only 12 weeks in patients with established severe fibrosis"

"To me, patients with Alström syndrome display the most extreme features of Type 2 diabetes and liver fibrosis and a trial of PBI-4050 in this population is as challenging a test of the efficacy of PBI-4050 as I can imagine" stated Dr John Moran, ProMetic's Chief Medical Officer. "The progression of liver fibrosis is much more aggressive in patients with Alström syndrome than in "typical" metabolic syndrome patients with obesity, diabetes and fatty liver, and so the major improvement in FibroScan score in all 5 patients in only 12 weeks is way beyond our expectations. Non-alcoholic fatty liver disease ("NAFLD") is the manifestation of metabolic syndrome in the liver. Due to the worldwide obesity epidemic, NAFLD now affects 20–30% of the general population and thus has become by far the most common cause of chronic liver disease and we firmly believe that PBI-4050 will also be effective in that population", added Dr. Moran.

The ongoing Alström syndrome phase 2 clinical trial is an open label, single arm and single center study investigating the safety, tolerability and efficacy of ProMetic's small molecule lead compound PBI-4050 in a total of 20 patients. The trial is being performed at the specialty center for the care of UK patients with Alström syndrome at the Queen Elizabeth Hospital, Birmingham, UK. This center has recently published data showing that many of these patients show evidence of non-alcoholic fatty liver disease and advanced liver fibrosis at an early age, confirming previous publications showing a very high incidence of progression of NAFLD into liver cirrhosis with associated mortality in Alström patients.

 


Level of liver fibrosis
at baseline

Fibroscan value
at Baseline

Fibroscan value
at Week 12

Fibroscan
decrease from
baseline (%)

Patient 1

Severe (F3)

9.9

7.1

- 28%

Patient 2

Significant (F2)

7.4

6.1

- 18%

Patient 3

Mild (F1)

6.1

4.5

- 26%

Patient 4

Significant (F2)

8.1

5.3

- 35%

Patient 5

Mild (F1)

5.1

3.1

- 39%

 


Liver fibrosis
Score

Fibroscan
Pka range

Fatty liver

F0


Mild fibrosis

F1

2.5 - 7

Significant fibrosis

F2

7 – 9.5

Severe fibrosis

F3

9.5 – 12.5

cirrhosis

F4

>12.5

 

"We are absolutely thrilled to see the beneficial effects that PBI-4050 has in patients with Alstrӧm syndrome", stated Mr. Pierre Laurin, President and Chief Executive Officer of ProMetic. "These early results have motivated us to expand our program to Alström patients elsewhere in Europe and in North America. Furthermore, these results provide us with valuable clinical information as ProMetic pursues other unmet medical conditions where fibrosis plays a key role", added Mr. Laurin.

Conference Call and Webcast Information

ProMetic will host a conference call at 8:30am (ET) on Wednesday October 12, 2016 to discuss the Alström syndrome clinical results. The telephone numbers to access the conference call are (647) 427-7450 and 1-888-231-8191 (Toll-free). A replay of the call will be available from October 12, 2016 at 11:30 p.m. until October 19, 2016. The numbers to access the replay are 1-416-849-0833 (passcode: 96578110) and 1-855-859-2056 (passcode: 96578110). A live audio webcast of the conference call will be available through the following:
http://event.on24.com/r.htm?e=1285808&s=1&k=A64E1226736219C9785D21037FF18034

More about Alström syndrome:

Alström syndrome is a rare inherited autosomal recessive syndrome characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis, involving the liver, kidney and heart.

Alström syndrome is also characterized by a progressive loss of vision and hearing, a form of heart disease that enlarges and weakens the heart muscle (dilated cardiomyopathy), and short stature. This disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. Some individuals with Alström syndrome have a skin condition called acanthosis nigricans, which causes the skin in body folds and creases to become thick, dark, and velvety. The signs and symptoms of Alström syndrome vary in severity, and not all affected individuals have all of the characteristic features of the disorder.

About ProMetic      

ProMetic Life Sciences Inc. (www.prometic.com) is a long established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. ProMetic offers its state of the art technologies for large-scale purification of biologics, drug development, proteomics and the elimination of pathogens to a growing base of industry leaders and uses its own affinity technology that provides for highly efficient extraction and purification of therapeutic proteins from human plasma in order to develop best-in-class therapeutics. ProMetic is also active in developing its own novel small-molecule therapeutic products targeting unmet medical needs in the field of fibrosis, anemia, neutropenia, cancer and autoimmune diseases/inflammation as well as certain nephropathies. Headquartered in Laval (Canada), ProMetic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and commercial activities in the U.S., Europe, Russia, Asia and Australia.

Forward Looking Statements

This press release contains forward-looking statements about ProMetic's objectives, strategies and businesses that involve risks and uncertainties. These statements are "forward-looking" because they are based on our current expectations about the markets we operate in and on various estimates and assumptions. Actual events or results may differ materially from those anticipated in these forward-looking statements if known or unknown risks affect our business, or if our estimates or assumptions turn out to be inaccurate. Such risks and assumptions include, but are not limited to, ProMetic's ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the ability of ProMetic to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. You will find a more detailed assessment of the risks that could cause actual events or results to materially differ from our current expectations in ProMetic's Annual Information Form for the year ended December 31, 2015, under the heading "Risk and Uncertainties related to ProMetic's business". As a result, we cannot guarantee that any forward-looking statement will materialize. We assume no obligation to update any forward-looking statement even if new information becomes available, as a result of future events or for any other reason, unless required by applicable securities laws and regulations. All amounts are in Canadian dollars unless indicated otherwise.         

 

SOURCE ProMetic Life Sciences Inc.

For further information: Pierre Laurin, President and CEO, ProMetic Life Sciences Inc, p.laurin@prometic.com, 450.781.0115; Frederic Dumais, Senior Director, Communications & Investor Relations, ProMetic Life Sciences Inc., f.dumais@prometic.com, 450-781-0115

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