Medical Needs redacted development procedures

Biomedical research

1. Development of Biotherapies to Address Unmet Medical Needs
Innocent Bekard PhD MBA
February, 2022

2. 2
Chatham House Rule
Whenameeting,orpartthereof,isheldundertheChathamHouseRule,
participantsarefreetousetheinformationreceived,butneithertheidentity
northeaffiliationofthespeaker(s),northatofanyotherparticipant,maybe
revealed.

3. The power of Science, Technology, Engineering, Mathematics (STEM)
3
Source: Google maps

4. Academiclife –towards a career in science
4
Institution: AdisadelCollege
Location : Cape Coast, Ghana
Course : Science (Physics, Chemistry, Biology)
Duration : 3 years
Institution: University of Ghana
Location : Accra, Ghana
Course : BSc (Hons) Biochemistry
Duration : 4 years
Institution: The University of Melbourne
Location : Melbourne, Australia
Course : PhD (Chem Eng), MBA
Duration : 3 years (PhD), 2 years (MBA)

5. Career path
5
Teaching Assistant
(Science)
National Service
(International
Student’s
Coordinator)
Business
Development
Officer
(Consulting Firm)
Research Assistant
(Chemical Eng.)
Scientist
(Process Design)
ADISADEL COLLEGE

6. Journey as a pharmaceutical research scientist
6
Scientist/Snr Scientist
Commercial plasma
fractionation process
support
Manager
Process design for
recombinant proteins
Technical Lead
Process support
for sa-mRNA and
flu vaccines
2011 -2017
2017 -date
2022 -date
Broadmeadows
Parkville

7. Why pharmaceutical companies exist –business view
7
Source:
https://companiesmarketcap.com/pharmaceuticals/large
st-pharmaceutical-companies-by-market-cap/
Carved list from top 454
pharmaceutical companies
To improve health
and quality of life
of patients

8. Why pharmaceutical companies exist –business view
8
Demand for plasma derived biotherapies

9. Biotherapy platforms
9
Plasma
Therapies
Recombinant
Therapies
Cell and Gene
Therapy
mRNA
Technology
Established
Vaccine
Technology

10. Examples of indications for plasma therapies
10
Therapy Condition treated
Coagulation factors e.g. Factor VIII, IX
•Bleeding from trauma
•Bleeding disorders e.g. haemophilia A and B
Immunoglobulins (IgG)
•Immunology: Immunodeficiencies (e.g. compromised immune system)
•Neurology: Immune mediated diseases (e.g. multi focal motor neuropathy)
•Haematology: e.g. Idiopathic thrombocytopenic purpura
•Dermatology: e.g. Dermatomyositis
Hyperimmune globulins
•Rabies, tetanus, hepatitis
•Rh negative pregnancy
•Transplant therapy
Alpha-1 proteinase inhibitor
•Alpha-1 antitrypsin deficiency: genetic deficiency resulting in lung or liver
disease
Albumin
•Cardiac surgery, liver disease, severe infections
•Emergency surgical medicine to treat shock, severe burns, and during
surgery
C1-esterase inhibitor (C1-INH) •Hereditary angioedema

11. Clinical presentation of C1 esterase inhibitor deficiency (example)
11
Hereditary angioedema (HAE), which presents as swelling beneath the surface of the skin
DidierG.EboandChrisH.Bridts.(2012)Disfiguring
Angioedema.ImagesinClinicalMedicine.New
EnglandJournalofMedicine367:1539
Cause
•deficient/dysfunctional C1 esterase inhibitor
•Swelling mediated by elevated levels of bradykinin
Treatment approaches
•C1 esterase inhibitor replacement therapy
•Administration of a kallikrein inhibitor

12. Phases of drug development and commercialisation
12
Drug
Discovery
Preliminary
Toxicology
Process
Design
Clinical Trials
Phase I, II, III
Registration
Commercial
Manufacture
Research (preclinical)
Process Development
Clinical Campaign
Market
Authorisation
Lifecycle
Management
Enabling functions
•Business development
•Project management
•Marketing
•Regulatory affairs
•Research scientist
•Chemistry, manufacturing, controls
•Engineering
•Manufacturing operations
Guidelines
ICH Q8 –Pharmaceutical Development
Good Laboratory Practice
Good Manufacturing Practice
•Quality Assurance
•Quality Control
•Legal –intellectual property
•Medical affairs
Guiding philosophy
Quality by Design (QbD)
Regulators

13. Phases of drug development and commercialisation
13
Drug
Discovery
Preliminary
Toxicology
Process
Design
Clinical Trials
Phase I, II, III
Registration
Commercial
Manufacture
Research
Considerations
•Moleculeofinterest:Outputfromresearchactivity
identifiesasset,whichisclinicallyfeasiblewith
commerciallyattractiveindication
•Targetproductprofile(TPP):Definesbasicproduct
requirementfromaclinicalstandpointe.g.indication,
routeofadministration
•Animalmodels:Acceptableproofofconceptinrelation
toefficacyandtoxicologicalprofilefrominvivoandin
vitrostudies

14. Phases of drug development and commercialisation
14
Drug
Discovery
Preliminary
Toxicology
Process
Design
Clinical Trials
Phase I, II, III
Registration
Commercial
Manufacture
Process Development
Clinical Campaign
Considerations
•Qualitytargetproductprofile(QTPP):Intendeduse,routeof
administration,dosage,containerclosuresystem,purity,sterility,
stability,bioavailabilityetc.
•Potentialcriticalqualityattributes(pCQAs):sterility,viralsafety,
concentration,stability
•Processperformanceandcharacterisation:Materialattributes
temperature,pHconductivity,flowrates
•Clinicaltrials–clinicaltrialapplication(CTA):
•PhaseI:2to80participants.Testforsafetyandsideeffects
•PhaseII:100to300participants.Testforefficacyandsafety
•PhaseIII:300to1000+participants.Monitoradverseeffects
•PhaseIV:Post-approvalstudiesandpharmacovigilance

15. Phases of manufacturing process design
15
Process
Design
Pilot Scale
Clinical
Manufacture
Process
Review
Process
Improvement
Clinical Trials
Phase I, Phase II, Phase III

16. Phases of drug development and commercialisation
16
Drug
Discovery
Preliminary
Toxicology
Process
Design
Clinical Trials
Phase I, II, III
Registration
Commercial
Manufacture
Market
Authorisation
Considerations
•Wellcharacterisedprocesswithsafeandefficacious
clinicalprofile
•Acceptablereturnoninvestment
•Biologicallicenceapplication(BLA)
•Processperformancequalification(PPQ)
•Nointellectualpropertycomplications

17. Phases of drug development and commercialisation
17
Drug
Discovery
Preliminary
Toxicology
Process
Design
Clinical Trials
Phase I, II, III
Registration
Commercial
Manufacture
Lifecycle
Management
Considerations
•Manufacturing facility
•Routine manufacture
•Continued process verification (CPV)
•Continued process improvement
•Pharmacovigilance

18. Key takeaways
•Biotherapies play a vital role in maintaining public health and
providing an improved quality of life for patients with rare and life-
threatening medical conditions
•The biotherapies segment of the pharmaceutical industry is vibrant
with demand expected to stay on an upward trajectory well into the
future
•There are still a number of unmet medical needs for which a
search for medical interventions are ongoing
•The process of drug development follows a well structured
methodology with strong collaboration between regulators and the
industry

19. BLANK