A proposed model in the egyptian pharmacies presentation
Alzipill
1. Alzipill
Use as an investigational drug
outside of clinical trial
MEGAZYME
INTERNATIONAL
2. AGENDA
Introduction
Proposed indication: Alzheimer’s Disease
Major risks and challenges
About Megazyme new venture: Alzipill
Treatment use of Alzipill
2
Other options
Outlook of current statistical report analysis
3. 3
More than 5 million Americans are living
with Alzheimer’s.
It is the 6th leading cause of death in
United States.
1 in 3 seniors dies with Alzheimer’s or
another dementia.
It kills more than breast and prostrate
cancer combined.
Facts About Alzheimer’s Epidemic
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4
6
8
10
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14
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2010 2015 2020 2025 2030 2035 2040 2045 2050
Number of Americans with Alzheimer’s
InMillions
4. Overview of Alzheimer’s Disease
4
A number of theories have been
proposed to explain the cause of AD but
to date, no one theory can adequately
explain all aspects of the disease
Precise mechanisms for AD
progression are also unclear
There are 3 major theories:
(Cholinergic, Amyloid, Tau) that are currently
regarded as the most likely explanation for AD
They are being used as the basis
for therapeutic development
5. 5
1st Stage:
Normal adult.
No functional decline.
3rd Stage:
Early Alzheimer’s.
Functions at about 12 years of age.
2nd Stage:
Awareness of some
functional decline.
4th Stage:
Mild Alzheimer’s.
Requires some assistance.
6th Stage:
Needs 24/7 care.
Functions at 2-4 years of age.
5th Stage:
Needs help getting dressed.
Functions at 5-7 years of age.
7th Stage:
Severe.
Functions at level of a newborn.
The Stages of
Alzheimer’s
Disease
6. Alzipill
6
But we need more volunteers to
complete clinical trials and
increased federal funding of
research to ensure that fresh
ideas continue to fill the pipeline.
Our researchers are looking for
new ways to treat Alzheimer's.
The current drug help mask the
symptoms of Alzheimer's, but do
not treat the underlying disease
or delay its progression.
A breakthrough Alzheimer's drug would
treat the underlying disease and stop or
delay the cell damage that eventually
leads to the worsening of symptoms.
There are several promising drugs in
development and testing.
8. 8
› There are often no simple solutions to
complex problems and AD is a good case
in point
› Clinical trial results to date have been
disappointing for several reasons:
• Our understanding of the pathophysiology,
the range of causes and how AD
progresses from pre-clinical to advanced
disease is still limited.
• too great a focus on amyloid-directed
therapy – need to look at non-amyloid
targets such as tau,
antioxidants/neuroprotectants and others
• giving treatment too late in the disease
process
• Limitations will be adverse effects and
affordability
Outlook towards
the Disease
9. Current Statistical Report
9
The clinical trial is in its phase II trial and the results are yet to
be received.
Efficacy and additional safety reports have not been
established yet.
The future of this company is at stake.
10. 10
Question Being – Can the drug be made available for public use without FDA approval
Yes, through new expanded access IND submission for expanded access, which can be used if:
(1) There is no existing IND in effect for the drug or, more commonly
(2) There is an existing IND in effect for the drug, but the sponsor of the existing IND10
declines to be the sponsor of the expanded access use (e.g., for an individual patient use,
the sponsor of the existing IND may prefer that a patient’s physician take on the role of
sponsor-investigator and submit a separate individual patient IND).
11. Treatment Use of Alzipill
11
The company is considering treatment use of the investigation drug.
Prospective IRB review and informed consent
Consulting on protocol development
Regulatory document preparation
Site qualification
Rapid and targeted start-up activities
Initial and ongoing training
Customized patient & site/clinical
practice support tools
Access to and training on a dynamic
electronic data capture (EDC) system
Main Requirements
12. Considerations for Pre-Approval Access Programs for
Patients
12
Clinical
› Understand the global regulatory
environment governing EAP implementation
› EAP design and data collection: What are
the important data collection considerations
relative to efficacy and safety data?
› Enrollment considerations: Could enrollment
in the expanded access program impact the
clinical trial?
Commercial
› Key stakeholders: How can early engagement
among key stakeholders (clinical commercial &
supply chain) optimize patient
transition experience and prevent disruption of
therapy?
› Opportunity to gain understanding of the payer
landscape and anticipate barriers to access
› Consider site level requirements for IRB
approval of Patient Assistance Program
enrollment forms and optimize the sequence of
the review and approval timeframe
› Aggregation of fragmented data to
support timely and transparent reporting
13. Major Risks and Challenges
13
Any serious adverse reaction might put the ongoing
clinical trial at risk.1
A new protocol needs to be prepared which is a very
time consuming process.2
3
The clinical trial needs to go through another IRB review
and informed consent form.
The clinical staff might not be trained for expanded
access use of drug.4
14. Other Options
14
Enroll more patientsConsider due diligence to get
monetary support and buy
time for the company to
establish positive results.
Wait for Phase II clinical
reports to develop
positive results
In case of negative test reports
consider making changes to
the existing protocol
