YM BioSciences 2012 Annual Meeting of Shareholders
1. YM BioSciences
AGM Presentation
November 2012
1 YM AGM Presentation | November 2012
2. YM BioSciences 2012 Annual General Meeting
A. Formal Business
1) Fix Number of Directors to be Elected
2) Election of Directors
David Allan, Thomas Allen, Kapil Dhingra, Mark Entwistle, Henry
Friesen, Nick Glover, Catherine Mackey, Nicole Onetto and Tryon
Williams
3) Appointment of Auditors
4) Further Business and Termination of the Meeting
B. Presentation from Management
2 YM AGM Presentation | November 2012
3. Safe Harbor
This presentation may contain forward-looking statements, which reflect the Company's current
expectation regarding future events. These forward-looking statements involve risks and uncertainties
that may cause actual results, events or developments to be materially different from any future results,
events or developments expressed or implied by such forward-looking statements. Such factors
include, but are not limited to, changing market conditions, the successful and timely completion of
clinical studies, the establishment of corporate alliances, the impact of competitive products and pricing,
new product development, uncertainties related to the regulatory approval process or the ability to
obtain drug product in sufficient quantity or at standards acceptable to health regulatory authorities to
complete clinical trials or to meet commercial demand, and other risks detailed from time to time in the
Company's ongoing quarterly and annual reporting. Certain of the assumptions made in preparing
forward-looking statements include but are not limited to the following: that our product candidates will
generate positive efficacy and safety data in future clinical trials, and that YM and its various partners
will complete their respective clinical trials within the timelines communicated. Except as required by
applicable securities laws, we undertake no obligation to publicly update or revise any forward-looking
statements, whether as a result of new information, future events or otherwise.
3 YM AGM Presentation | November 2012
4. Strategic Priorities for FY2012
1. Ensure optimization of the clinical and commercial potential of CYT387
• Myelofibrosis as proof-of-concept/path-to-market indication: PI/II – PIII transition
• Explore strategic partnering to potentially maximize value of the asset
• Initiate preclinical studies to explore anemia mechanism of action
2. Build a portfolio of products with clinical and commercial potential
• R&D collaborations
• YMBA Intellectual Property estate and small molecule libraries; JAK/kinase
screening project
• In-licensing and M&A opportunities
3. Minimize and rationalize resource expenditures
• Support ongoing development of nimotuzumab by sub-licencees
• Review clinical and commercial opportunity for CYT997
4 YM AGM Presentation | November 2012
5. CYT387: Our Promising Lead Asset
Significant Opportunity
• A potentially differentiated drug in the emerging JAK class
Strong Fundamentals
• Under-served initial disease with $B market potential
• Compelling clinical proof-of-concept data
Rapid Progress
• 2.5 years of expanding clinical development
• Transitioning to Phase III
• Oriented towards commercialization
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6. Our Significant Expertise in JAK Research
YM acquired original intellectual assets in Intellectual Property
JAK field
– Identified by Dr. Andrew Wilks, Ludwig CYT387 Composition of Matter
Cancer Institute, Melbourne, Founder of US: Pending, 2028 expiry
Cytopia (now YM Australia) EU: Pending, 2028 expiry
– First group to publish crystal structures
of JAK2 and JAK1 JAK2 Crystal Structure
– Medicinal chemistry and molecular US: Issued, 2025 expiry
modeling expertise EU: Pending, 2026 expiry
JAK2 Enzyme
US: Issued, 2015 expiry
6 YM AGM Presentation | November 2012
7. Target Markets for JAK Inhibitors
Autoimmune Myeloproliferative Cancer /
Diseases Neoplasms Hematology
– Rheumatoid – Myelofibrosis – Leukemia and
Arthritis Lymphoma
– Polycythemia Vera
– Psoriasis – Solid Tumors
– Essential
– Graft vs. Host Thrombocythemia – Other Hematologic
Disease Disorders
Chronic Disorders Clinical Proof of Concept Acute Diseases
7 YM AGM Presentation | November 2012
8. Clinical Presentations of Myelofibrosis
A chronic debilitating disease in which a
patient’s bone marrow is replaced by scar
tissue
– Anemia – often requiring transfusions
– Thrombocytopenia
– Splenomegaly
– Constitutional symptoms
Fatigue, night sweats, pruritus, bone pain,
weight loss, fever
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9. Anemia Impacts Survival in Myelofibrosis
Anemia at diagnosis Anemia at any time
– ~70% of myelofibrosis patients are Intermediate-II or High risk †
– Estimated that 30-50% of all myelofibrosis patient are transfusion dependent‡,
majority of which are Intermediate-II and High risk patients
† DIPSS-Plus; Gangat et al. JCO 2011; 29(4), 392
‡ Elena et al. Haematologica 2011 96(1) 167
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10. Transfusion Independence Response
(as at ASH 2011)
150 mg QD 300 mg QD 150 mg BID Total1
Response by Dose
(n=52) (n=60) (n=42) (n=166)
Transfusion dependent at baseline (evaluable; n) 25 26 14 68
Median time on study (days) 251 245 141 250
Transfusion independence rate (12 weeks)* 48% 65% 43%2 54%
Transfusion independence rate (12 weeks & Hgb≥8g/dL)* 40% 62% 29%2 46%
– >25% of subjects not receiving transfusions while on study experienced at least a 1 g/dL increase in Hgb
for ≥ 8 weeks
Time to Response Median Min-Max
Time to confirmed response (12 wks & Hgb≥8 g/dL) (days) 84 84-293
Duration of transfusion-free period (12 wks & Hgb≥8 g/dL) (days) not yet reached 82-506*
1 Includes 100 mg QD (n=3), 200 mg QD (n=3), and 400 mg QD (n=6) doses
2 Not statistically significant vs. 300 mg QD
* Ongoing
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11. Maximum Duration of Transfusion-Free Period
(as at ASH 2011, ongoing)
Responders
Clinically relevant maintenance of
transfusion independence period
0 100 200 300 400 500
Time (days)
* As at ASH 2011
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12. Spleen Response
(as at ASH 2011)
150 mg QD 300 mg QD 150 mg BID Total1
Response by Dose
(n=52) (n=60) (n=42) (n=166)
Spleen evaluable (n) 47 51 33 142
Median time on study* (days) 252 225 144 225
Spleen response* (IWG-MRT) 30% 33% 27% 31%
≥50% decrease in palpable spleen length at six months 28% 33% 39% 33%
Median spleen decrease at six months -35% -35% -39% -35%
Primary MF Post-PV MF Post-ET MF
Response by Diagnosis
(n=106) (n=36) (n=24)
Spleen evaluable (n) 88 34 20
Spleen response* (IWG-MRT) 28% 38% 30%
Time to Response Median Min-Max
Time to IWG-MRT response (days) 15 6-260
Duration of response (days) not yet reached 55-574*
1 Includes 100 mg QD (n=3), 200 mg QD (n=3), and 400 mg QD (n=6) doses
* Ongoing
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13. Maximal Change in Palpable Spleen Size
(as at ASH 2011, ongoing)
(Core Study; n=142)
80%
≥ 25% decrease from baseline: 87%
60% ≥ 50% decrease from baseline: 49%
≥ 75% decrease from baseline: 25%
100% decrease from baseline: 16%
40%
% Change From Baseline
20%
0%
-20%
-40%
-60%
-80%
-100%
* Ongoing
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14. Constitutional Symptoms Response at Six Months
(as at ASH 2011)
100%
Complete Resolution
Marked Improvement
90%
80%
Percentage of Patients
70%
60%
89%
50% 57%
52%
44%
40%
30%
30%
20%
10% 23% 22% 23%
19%
11%
0%
Night Sweats Pruritis Bone Pain Cough Fever
(n=62) (n=46) (n=43) (n=27) (n=9)
Complete resolution or marked improvement of common constitutional
symptoms is achieved in the majority of subjects
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15. CYT387 – Safe, Effective, Differentiated
CYT387 treatment results in significant, durable responses in anemia,
splenomegaly and constitutional symptoms at all doses evaluated.
– Therapeutic benefit and safety established in a population with multiple risk
factors, including anemia and thrombocytopenia
– CYT387 anemia benefit appears unique among the current class of JAK1 and
JAK2 inhibitors
– Clinically relevant maintenance of transfusion independence period
– MRI performed in a subset of subjects confirms the meaningful improvement in
splenomegaly measured by palpation
– Complete resolution or marked improvement of common constitutional
symptoms is achieved in the majority of subjects
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16. CYT387: Meets Clinical Needs in Myelofibrosis
Variable Diagnosis* >1 year* CYT387
Anemia 38% 64% Benefit
Transfusion dependency 25% 45% Benefit
Palpable spleen >10cm 21% 46% Benefit
Constitutional symptoms 29% 34% Benefit
CYT387 has a profile that addresses MF clinical needs and overarching risk factors
> Benefit on anemia and transfusion dependency
> Activity for spleen and symptoms
> Low myelosuppression
CYT387 is well tolerated for dosing periods up to and exceeding two years
* Mayo Clin Proc 2012;87(1): 25-33
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17. CYT387 Myelofibrosis Development Pathway
Aug 2010 Dec 2010 July 2011 Dec 2012
Dec 2011
CYT387 Reported Reported Report Final 9-
Reported Interim
Feb 2010 designated Interim 60 Interim 60 month 166
166 patient
Acquired Orphan patient data patient 12-week patient data at
multicenter data
Cytopia Drug at ASH data at ASCO ASH
at ASH
March 2010 Nov 2010 Sept 2011 June 2012 July 2012
Increased Increased trial Completed Completed Completed
CYT387 trial from 60 to 140 enrollment of dosing of 166 enrollment
from 21 to patients and 166 patient trial patient trial of 61
60 patients added BID & patient
cohort Initiated BID trial BID trial
Expand Clinical Development
Preclinical Preparation Activities
PIII & Commercial Readiness
Build Management FDA and EMA Discussions
Capsules to tablet transition
Partnering Campaign
Financing
17 YM AGM Presentation | November 2012
18. CYT387: Next Steps
ASH 2012
• Podium presentation at ASH 2012
• Phase I/II Core study results reinforce differentiated profile
Business Development
• Exploring opportunities to develop CYT387 with other companies
• Conducted a broad, robust business development process
• Actively exploring variety of options
Phase III
• Preparations for Phase III ongoing
• Flexibility to advance CYT387 with or without a partner
18 YM AGM Presentation | November 2012