Plasminogen &
Corporate Update
October 29, 2014
1
Safe Harbour
Forward Looking Statement
This presentation 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 forwardlooking 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 the Annual Information Form for the year ended
December 31, 2013, under the heading “Risk Factors”. 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.
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Disclaimer
ProMetic reserves the right to make improvements, corrections and/or changes to this presentation at
any time.
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AGENDA
• Plasminogen
•
•
Phase I and Phase II / III protocols
What is an Accelerated Approval Pathway
• PBI‐4050
•
•
Pages 14 ‐15
Status
• Summary •
•
Pages 9 ‐13
IPF – First Orphan indication targeted
Clinical program – next steps
• Key Development & Regulatory Milestones for 2014
•
Pages 4 ‐ 8
Pages 16 ‐ 17
Clinical programs & outlook for 2015
Guidance : what to expect before year end 2014
• Q & A
3
Plasminogen
4
Plasminogen Deficiency :
Multi‐system disease that affects the prevention and healing of tissue damage.
Eyes, ears,
Sinuses,
Gingiva
tracheobronchial tree
genitourinary tract
5
Plasminogen : Phase I
Open label, single ascending dose (intra‐venous administration)
6 patients suffering from hypoplasminogenemia
Objectives
Safety, tolerability and pharmacokinetics
Pharmacokinetics:
Determine the
½ life of Pg in plasma
and whether the ½ life is
dose dependant
Pg
Plasma
concentration
Time (days)
6
Plasminogen : Phase II / III
Open label, multiple doses (intra‐venous administration)
15 ‐ 18 patients suffering from hypoplasminogenemia
Objectives
Safety, tolerability
Dose regimen to achieve surrogate endpoint
Define the optimal dose
regimen to achieve the
surrogate end point =
defined Pg concentration
for each patient
Pg
Plasma
concentration
Surrogate
End point ‐
Concentration
threshold
Time (days)
7
Plasminogen Deficiency
FDA clearance of the IND
FDA acceptance of the Phase II / III surrogate end point for licensure* Plasminogen IV Clinical program for hypoplasminogenemia
Phase I
~ 6 patients
Safety & PK
Phase II/III
~ 15 ‐ 18 patients
Safety + surrogate end point
H1 2015
BLA
Market
accelerated approval Pathway* H2 2015
2016
*To secure an accelerated pathway approval, a drug must treat a serious condition, provide a meaningful advantage over available therapies and demonstrate an effect
on a surrogate endpoint that is reasonably likely to predict clinical benefit.
8
PBI-4050
Why targeting (IPF)?
9
Idiopathic Pulmonary Fibrosis (IPF)
Poor prognosis even when compared to many cancers
In the USA alone:
~150,000 IPF patients
> 50,000 deaths / year
10
PBI-4050: regulating multiple factors involved in pulmonary fibrosis
Adapted from Datta A., Scotton C.J. and Chambers R.C. Novel Therapeutic approaches for pulmonary fibrosis. Br. J. Pharmacol. 2011, 163:141-72.
1111
Preclinical data in pulmonary fibrosis induced by bleomycin in mice
Significant reduction of the % of leukocyte infiltration in lung
determined in Masson’s trichrome staining (morphometric evaluation)
C O N F I D E N T I A L P R E S E N T A T I O N
12
PBI-4050: Clinical Program
H2 2014
Phase Ib
Multiple doses DKD patients
Safety & PK
(Canada) 2015
Phase Ib / II DKD patients
Multi Center Study
Placebo controlled – double blind
(Canada & USA)
Phase Ib / II Idiopathic Pulmonary Fibrosis (IPF) patients
Multi Center Study
Open Label
(Canada & UK)
Phase Ib / II Orphan condition (TBA)
Multi Center Study
Open label
(Canada & UK & USA)
Objectives of the program:
Primary end points:
• Safety & tolerability in patients
• Effects on biomarkers for fibrosis in blood & urine
• Effects on diabetes (when applicable)
• Tissue analysis / biopsies (when applicable)
Information on dosing
Set the stage for phase II/III
13
Key Development & Regulatory Milestones in 2014
Successfully Completed
PBI‐4050
Plasminogen
IGIV
In 2014
• Pre‐CTA meeting Health Canada for the proposed clinical program and manufacturing process
• Manufacturing of GMP product
• CTA granted for Phase I in Canada
• Completed Phase I – no serious adverse Events
• Pre‐IND meeting with the FDA for the proposed Canada‐USA multicentre trials
• Enrollment of clinical sites
• Pre‐IND meeting with the FDA to agree on clinical‐regulatory pathway & manufacturing process
• Manufacturing of the GMP product
• Preparation of the IND package
• Enrollment of clinical sites – Principal Investigators
• IND filing for clinical trials Phase I‐II‐III
• IND for phase I cleared
• Protocol for Phase II / III to follow an accelerated Approval Pathway
• Patients enrollment already initiated
• Pre‐IND meeting with the FDA to agree on clinical‐regulatory pathway & manufacturing process
• Successful scale up of the PPPS™ process
• Manufacturing of the GMP product
• Preparation of the IND package
• Enrollment of clinical sites – Principal Investigators
• The FDA confirmed that ProMetic IGIV successfully met QC Thrombogenic testing, a necessary step before filing the IND
• CTAs for the Phase II studies
• Patients enrollment
• Orphan Drug Designation
• IND filing for bioequivalence trial Phase I‐III
(on‐target in Q4)
14
Key Development & Regulatory Milestones in 2014
Successfully Completed
Fibrinogen
• Successful scale up of the PPPS™ manufacturing process
• Manufacturing of the GMP products
• Preparation of the Technical Specifications In 2014
• Commercial launch of GMP Fibrinogen for use in various medical applications
e.g. harvesting and culturing stem cells, wound
healing products, hemostatic bandages and drug
delivery systems)
On‐going & on target AAT
• Successful optimization of the PPPS™ process
to enable the manufacturing of AAT and other orphan Rx candidates
• Preparation of the Technical Dossier for the
Pre‐IND meeting with the FDA
•
•
•
•
•
Pre‐IND meeting with the FDA
Manufacturing of the GMP product
Preparation of the IND package
Enrollment of clinical sites – Principal Investigators Patients enrollment in H1 2015
On‐going & on target Orphan Rx
• Successful optimization of the PPPS™ process
to enable the manufacturing of orphan Rx candidates
• Preparation of the Technical Dossier for the
Pre‐IND meeting with the FDA
• Disclosure of 2 New Orphan Drug Candidates On‐going & on target 15
Therapeutics - Pipeline Development Status
R&D pre‐clinical scale up Clinical Programs Phase I
Phases II & III*
PBI‐4050 – Diabetic Kidney Disease (DKD)
Phase Ib/II 2015
PBI‐4050 – Idiopathic Pulmonary Fibrosis (IPF)
Phase Ib/II 2015
PBI‐4050 – Other Orphan Indication (TBA)
Phase Ib/II 2015
Plasminogen – Hypoplasminogenemia
Phase II / III 2015
IGIV – Primary Immune Deficiency (PID)
Phase III 2015
AAT
Phase III 2015
Orphan Rx TBA in H2 2014
Orphan Rx TBA in H2 2014
*Phases II & III clinical trials scheduled for 2015
16
Guidance - Before Year End 2014 the Company expects:
•
PBI‐4050 to progress in patients – phase Ib
•
To disclose New PBI‐4050 data at ASN (American Society of Nephrology Annual Meeting Nov 12‐15)
•
To disclose an additional Orphan indication for PBI‐4050
•
To file the IGIV IND with the FDA
•
To disclose 2 additional PPPS™ derived Orphan Rx
•
To close partnering deals providing :
•
•
•
•
Financial contribution
Access to market Additional manufacturing capacity Revenue range for H2 2014 ($15 M ‐ $20 M)
•
•
Subject to revenue recognition on licensing fees
Revenue significantly higher in Q4
17
Q&A
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