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Corporate Presentation
                                                       Calendar Q2 2012

1   YM Corporate Presentation | Calendar Q2/2012
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 JAK1/JAK2 inhibitor CYT387, nimotuzumab and our
VDA small molecule CYT997 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.

 2   YM Corporate Presentation | Calendar Q2/2012
Corporate Information


     Net Cash (Mar 31, 2012)                            $137.2mm

     Cash used in last 12 months (Dec 31, 2011)         $26.7mm

     Market cap (May 8, 2012)                           $282mm

     Volume (3-month average)                           ~1.1mm shares/day


     Total shares outstanding (Mar 31, 2012)            157.4mm

     Warrants @ $1.60 (to Mar 10, 2015)                 7.4mm

     Options1 (Mar 31, 2012)                            8.2mm

     No debt or preferred shares
1   Weighted average exercise price $1.92

    3    YM Corporate Presentation | Calendar Q2/2012
YM BioSciences


        Product                                          Preclinical   Phase I   Phase II   Phase III
        CYT387
        JAK1/JAK2 inhibitor
        Positive interim Phase I/II myelofibrosis data
        presented at ASH 2011


        Nimotuzumab
        EGFR targeting antibody
        Multiple Phase II and Phase III trials



        CYT997
        Tubulin inhibitor
        IV + oral formulation development




        Kinase Inhibitor Library
        ~4000 molecules including JAK RA candidates
1   Weighted average exercise price $1.99

    4     YM Corporate Presentation | Calendar Q2/2012
Target Markets for JAK Inhibitors



    Autoimmune                                     Myeloproliferative          Cancer /
    Diseases                                       Neoplasms                   Hematology
    – Rheumatoid                                   – Myelofibrosis             – Leukemia and
      Arthritis                                                                  Lymphoa
                                                   – Polycythemia Vera
    – Psoriasis                                                                – Solid Tumors
                                                   – Essential
    – Graft vs. Host                                 Thrombocythemia           – Other Hematologic
      Disease                                                                    Disorders




    Chronic Disorders                              Clinical Proof of Concept     Acute Diseases


5   YM Corporate Presentation | Calendar Q2/2012
Our Significant Expertise in JAK Research

YM acquired original intellectual assets           Intellectual Property
in JAK field
                                                   CYT387 Composition of Matter
    – Lead product CYT387
                                                   US: Pending, 2028 expiry
    – Wholly-owned and un-partnered                EU: Pending, 2028 expiry

    – First group to publish crystal               JAK2 Crystal Structure
      structures of JAK2 and JAK1
                                                   US: Issued, 2025 expiry
    – Medicinal chemistry and molecular            EU: Pending, 2026 expiry
      modeling expertise
                                                   JAK2 Enzyme
    – Novartis collaboration for selective         US: Issued, 2015 expiry
      JAK3 inhibitors
                                                   EU: Issued, 2011 expiry


6   YM Corporate Presentation | Calendar Q2/2012
CYT387: A Potent, Selective JAK1/JAK2 Inhibitor


    – Highly specific, small molecule, ATP
      competitive, JAK1/JAK2 inhibitor

    – Rationally designed for optimal JAK binding

    – Inhibits cellular proliferation driven by JAK2
      or JAK1, but not JAK3 or other mechanisms

    – Inhibits STAT phosphorylation downstream
      of constitutively active JAK2 or cytokine
      stimulated JAK1 or JAK2

    – Orally active

    – Very favourable preclinical profile




7   YM Corporate Presentation | Calendar Q2/2012
Clinical Presentations of Myelofibrosis


    – Decreased erythropoiesis or thrombopoiesis
    – Proliferation of dysfunctional megakaryocytes
    – Hypercellular bone marrow leading to fibrosis
    – Extramedullary hematopoiesis
    – Elevated cytokine levels


    – Anemia – often requiring transfusions
    – Thrombocytopenia
    – Splenomegaly
    – Constitutional symptoms
      Fatigue, night sweats, pruritus, bone pain,
      weight loss, fever

8   YM Corporate Presentation | Calendar Q2/2012
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

9      YM Corporate Presentation | Calendar Q2/2012
CYT387: Target Product Profile


CYT387 is able to provide a clinically meaningful benefit to myelofibrosis
patients by:



 1. Converting                                      2. Reducing spleen      3. Improving
    transfusion dependent                              volume in patients      constitutional
    patients to transfusion                            with enlarged           symptoms and
    independent status                                 spleens                 quality of life



                           … while having a significantly lower risk of causing
                           or worsening hematological adverse events such as
                           thrombocytopenia, anemia and neutropenia.


10   YM Corporate Presentation | Calendar Q2/2012
Safety and Efficacy of CYT387
                                                    Updated Phase I/II Data – ASH 2011

11   YM Corporate Presentation | Calendar Q2/2012
Current Study Status
                                166 Patient Phase I/II Study
          Dose                                   Dose                 Dose
                                                                                                Dose
        Escalation                            Confirmation          Expansion
                                                                                             Extension
          Phase                                  Phase                Phase
                                                                                                Study
           (n = 21)                                 (n = 39)         (n = 106)                (n = 104)

Initiated Nov 2009                       Initiated Aug 2010     Initiated Dec 2010      Enrollment complete

100 mg QD (n=3)                                                                         Data collection and
150 mg QD (n=3)                         150 mg QD (n=18)       150 mg QD (n = 31)       analysis ongoing
200 mg QD (n=3)                         300 mg QD (n=21)       300 mg QD (n = 33)
300 mg QD (n=6)                                                150 mg BID (n = 42)      97% of patients
400 mg QD (n=6)                                                                         entered the Extension
                                                                                        phase as at ASH 2011

DLT level: 400 mg QD                                           Mayo Clinic (Rochester, Minnesota)
MTD: 300 mg QD                                                 Dana Farber Cancer Institute (Boston)
                                                               Stanford Cancer Center (Stanford)
Mayo Clinic                              Mayo Clinic           Royal Melbourne Hospital (Melbourne)
                                                               Princess Margaret Hospital (Toronto)
                                                               Jewish General Hospital (Montreal)

12   YM Corporate Presentation | Calendar Q2/2012
CYT387 Evaluated in an Advanced Population


                                                                  CYT387                      Ruxolitinib            Ruxolitinib          Ruxolitinib
Baseline Characteristics                                         Phase I/II                    Phase III              Phase III            Phase II
                                                                (ASH 2011)                    (Comfort-1)            (Comfort-2)           (NEJM)
Primary Myelofibrosis (%)                                            65%                           45%                    53%                  53%
Post-PV Myelofibrosis (%)                                            22%                           31%                    31%                  31%
Post-ET Myelofibrosis (%)                                            13%                           24%                    16%                  16%
Median Hemoglobin (g/dL)                                              9.5                          10.5                   10.4                 10.4
                               9
Median Platelets (10 /L)                                             187.5                         262               236 (all pts)              263
                       9
Median ANC (10 /L)                                                    8.3                    Not reported           Not reported               12.1
Spleen length (cm)                                                     17                           16                     15                    19
                           3
Spleen volume (cm )                                                  3667                         2595                   2381
                                                                                            < 21% (estd*)          < 21% (estd*)
                                                                                                                                          (unspecified
Transfusion dependent (IWG) (%)                                      44%                    (Unspecified           (Unspecified
                                                                                                                                            criteria)
                                                                                               criteria)              criteria)



* Product label cited that 21% of patients had RBC transfusion within 8 weeks of enrollment in the study. Maximum estimation of potential transfusion
  dependent patients

13   YM Corporate Presentation | Calendar Q2/2012
Subject Disposition
     –      Median follow-up (range) for Core and Extension: 10.4 months (0.8 – 25.6)
     –      Median follow-up (range) for Core: 9.0 months (0.8 – 9.0)
     –      Study discontinuation during Core: n=32 (19%); 81% retention rate
     –      Reasons for discontinuation during Core (n=32)
            >       Unrelated comorbid conditions (n=11)
            >       Subject withdrew consent (n=5)
            >       Possibly or probably related adverse events (n=5)
                    – Neuropathy (n=2)
                    – Cough (n=1)
                    – Transaminitis (n=1)
                    – Thrombocytopenia (n=1)
            >       Disease progression (n=4)
            >       Investigator’s decision (n=4)
            >       Stem cell transplant (n=2)
            >       Intercurrent illness (n=1)

     –      Deaths during Core n=12 (7%)
     –      Study discontinuation during Extension n= 16 (21%); 79% retention rate
14       YM Corporate Presentation | Calendar Q2/2012
Transfusion Independence Response


                                                                         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

15   YM Corporate Presentation | Calendar Q2/2012
Maximum Duration of Transfusion-Free Period*
     Responders




                  0                100              200             300   400   500
                                                          Time (days)

* To date

16   YM Corporate Presentation | Calendar Q2/2012
Spleen Response

                                                                         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

17   YM Corporate Presentation | Calendar Q2/2012
Maximal Change in Palpable Spleen Size*

                                                                  (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

18            YM Corporate Presentation | Calendar Q2/2012
Spleen Response: MRI vs. Palpation

                                                                                             MRI1 (n=11)      Palpation (n=11)

Response at three months                                                                        64%2               45%3

Median decrease from baseline at three months                                                   -41%               -45%

    –   Median spleen volume at baseline 3667 cm3

                            Spleen Response: Change from Baseline at Three Months
                                                       20%    Spleen Volume (n=11)   Spleen Length (n=11)

                                                        0%
                             % Change from Baseline




                                                       -20%

                                                       -40%                  -41%
                                                                                                       -45%

                                                       -60%

                                                       -80%

                                                      -100%
1 Includes 1 subject with CT assessment
2 Spleen response defined as a 35% reduction in spleen volume from baseline
3 Spleen response defined as a 50% reduction in spleen length from baseline


19      YM Corporate Presentation | Calendar Q2/2012
Constitutional Symptoms Response at Six Months


                                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)



20   YM Corporate Presentation | Calendar Q2/2012
Related Hematologic Adverse Events




 Adverse Event (n=166)                                All Grades   Grade 3   Grade 4

 Thrombocytopenia                                       33%         11%        6%
  Baseline platelets > 100 x 109/L                      26%          6%        2%
  Baseline platelets > 200 x 109/L                      12%          4%         0

 Neutropenia                                             6%          1%        2%

 Anemia                                                  4%          1%        0

 Leukopenia                                              2%         <1%       <1%
 Leukocytosis                                            1%         <1%        0




At least possibly related to study drug
Common Terminology Criteria for Adverse Events v3.0

21    YM Corporate Presentation | Calendar Q2/2012
Related Non-Hematologic Laboratory Adverse Events

Adverse Event (n=166) Incidence ≥ 10%                                      All Grades                  Grade 3   Grade 4

Dizziness1                                                                     20%                          0       0

Neuropathy peripheral                                                          20%                          0       0

Diarrhea                                                                       18%                          0       0

Nausea                                                                         17%                          0     <1%

Headache                                                                       12%                          1%      0


Adverse Event (n=166)                                                      All Grades                  Grade 3   Grade 4

First Dose Effect2                                                             20%                          0      0

    Dizziness                                                                  11%                          0      0

    Flushing                                                                    5%                          0      0

    Hypotension                                                                 6%                          0      0

1   Includes First Dose Effect                        At least possibly related to study drug
2   Only events occurring on first dose date          Common Terminology Criteria for Adverse Events v3.0

22     YM Corporate Presentation | Calendar Q2/2012
Related Non-Hematologic Laboratory Adverse Events




Adverse Event (n=166) Incidence ≥ 5%                  All Grades   Grade 3   Grade 4

ALT increased                                            8%          2%        0

Lipase increased                                         8%          4%        0

AST increased                                            7%          1%       <1%

Amylase increased                                        6%          0         0

Bilirubin increased                                      6%          0         0

Creatinine increased                                     6%          0         0




At least possibly related to study drug
Common Terminology Criteria for Adverse Events v3.0

23   YM Corporate Presentation | Calendar Q2/2012
CYT387 – Safe, Effective, Differentiated

CYT387 treatment results in significant, durable responses in anemia,
splenomegaly and constitutional symptoms at 150 mg QD, 300 mg QD and
150 mg BID dose levels

     – 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

24   YM Corporate Presentation | Calendar Q2/2012
CYT387 – Safe, Effective, Differentiated


CYT387 is well tolerated in myelofibrosis patients for dosing periods up to and
exceeding two years

     – Reported adverse effects include thrombocytopenia; transient, mild dizziness;
       mild peripheral neuropathy; and abnormalities in liver/pancreas-related laboratory
       tests


     – Treatment-emergent anemia and neutropenia are rare


Although additional assessments and analyses are ongoing, 300 mg QD
appears to be a safe and effective dosing regimen that warrants further clinical
development



25   YM Corporate Presentation | Calendar Q2/2012
CYT387 Has the Best Fit in Myelofibrosis


Variable                                            Diagnosis*    >1 year*     CYT387
     Anemia                                            38%          64%         Benefit

     Transfusion dependency                            25%          45%         Benefit

     Thrombocytopenia                                  18%          31%         Minimal

     Palpable spleen >10cm                             21%          46%         Benefit

     Constitutional symptoms                           29%          34%         Benefit


     – CYT387 has a superior profile to other JAK inhibitors in relation to clinical needs
       and overarching risk factors
        > Superior benefit on anemia and transfusion dependency
        > Comparable activity for spleen and symptoms
        > Less myelosuppression



* Mayo Clin Proc 2012;87(1): 25-33

26   YM Corporate Presentation | Calendar Q2/2012
CYT387
                                                    Next Steps

27   YM Corporate Presentation | Calendar Q2/2012
CYT387 BID Study Design

     Dose                         Starting at 200mg BID, increasing by 50mg BID
     escalation                   Recruiting approximately 60 patients
     study                        Initiated in calendar Q3 2011

     – Designed to explore BID schedule to complement QD MTD
     – Spleen response measured by palpation and MRI
     – Constitutional symptoms assessed using the Myelofibrosis Symptom Assessment
       Form (MFSAF)
     – To assess anemia response, a minimum three months of transfusion history will be
       collected
     – Comprehensive blood work and biomarkers being recorded for
       correlative/mechanistic studies

Mayo Clinic             MD Anderson                 Huntsman Cancer     Princess Margaret   Jewish General
 (Arizona)                (Texas)                    Institute (Utah)        (Ontario)        (Quebec)
* Mayo Clin Proc 2012;87(1): 25-33

28   YM Corporate Presentation | Calendar Q2/2012
CYT387 Myelofibrosis Development Pathway

                                                                                             Extension
                                                                                             study

166-patient
Phase I/II
                         Dec 2010                     Q2 2011       Q3 2011            Q4 2011           Q4 2012
                        Interim data                Interim data   Completed          Multicenter     Report final core
                           at ASH                     at ASCO      enrollment         data at ASH      study data &
                                                                                                      extension study
                                                                                                           data

                   ~60 patient Phase II BID
                                                                      Q3 2011                             Q4 2012
                                                               Initiated enrollment                      Report data


                                                                              Pivotal program
                                                                                                          H2 2012
                                                                                                    Initiate enrollment
29   YM Corporate Presentation | Calendar Q2/2012
CYT387 Development and Commercial Opportunities


     Myeloproliferative                             Hematological             Solid
     Neoplasms                                      Disorders                 Tumors
     – Myelofibrosis                                 – Myelodysplastic        – Prostate cancer
          First line                                   Syndromes (MDS)        – Hepatocellular
          Ruxolitinib failures                       – Multiple Myeloma       – NSCLC
          Combinations                               – AML, CML, ALL          – Gastric
                                                     – NHL, Lymphoma          – Colorectal
                                                     – PV/ET                  – Etc.
                                                     – Orphan MPNs


Clinical proof-of-concept                           Expand hematological    Market expansion
Speed to market strategy                            indications/franchise   Lifecycle management

30   YM Corporate Presentation | Calendar Q2/2012
Promising Pipeline – Exciting Future

              CYT387                                   Nimotuzumab                          CYT997
      JAK1/JAK2 inhibitor                                anti-EGFR mAb                   Tubulin inhibitor

 Oral, small molecule                               Prospect for enhanced safety   Small molecule vascular
                                                    in “billion dollar” class      disrupting agent (VDA)

 Wholly owned, unpartnered
 and differentiated                                 De-risked, over 10,000         Phase I clinical proof of
                                                    patients dosed                 concept demonstrated

 Potentially very broad utility
 (hematology, liquid tumors,                        Partners advancing Phase       Phase II (iv) in glioblastoma
 solid tumors)                                      II’s and III’s globally        multiforme


 Multicenter Phase I/II data                        YM retains North American      Reformulating as an oral
 in myelofibrosis reported                          rights                         drug
 at ASH 2011


                                   Kinase Inhibitor Preclinical Research Programs

31   YM Corporate Presentation | Calendar Q2/2012
32   YM Corporate Presentation | Calendar Q2/2012

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YM BioSciences Corp Pres May 9 2012

  • 1. Corporate Presentation Calendar Q2 2012 1 YM Corporate Presentation | Calendar Q2/2012
  • 2. 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 JAK1/JAK2 inhibitor CYT387, nimotuzumab and our VDA small molecule CYT997 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. 2 YM Corporate Presentation | Calendar Q2/2012
  • 3. Corporate Information Net Cash (Mar 31, 2012) $137.2mm Cash used in last 12 months (Dec 31, 2011) $26.7mm Market cap (May 8, 2012) $282mm Volume (3-month average) ~1.1mm shares/day Total shares outstanding (Mar 31, 2012) 157.4mm Warrants @ $1.60 (to Mar 10, 2015) 7.4mm Options1 (Mar 31, 2012) 8.2mm No debt or preferred shares 1 Weighted average exercise price $1.92 3 YM Corporate Presentation | Calendar Q2/2012
  • 4. YM BioSciences Product Preclinical Phase I Phase II Phase III CYT387 JAK1/JAK2 inhibitor Positive interim Phase I/II myelofibrosis data presented at ASH 2011 Nimotuzumab EGFR targeting antibody Multiple Phase II and Phase III trials CYT997 Tubulin inhibitor IV + oral formulation development Kinase Inhibitor Library ~4000 molecules including JAK RA candidates 1 Weighted average exercise price $1.99 4 YM Corporate Presentation | Calendar Q2/2012
  • 5. Target Markets for JAK Inhibitors Autoimmune Myeloproliferative Cancer / Diseases Neoplasms Hematology – Rheumatoid – Myelofibrosis – Leukemia and Arthritis Lymphoa – Polycythemia Vera – Psoriasis – Solid Tumors – Essential – Graft vs. Host Thrombocythemia – Other Hematologic Disease Disorders Chronic Disorders Clinical Proof of Concept Acute Diseases 5 YM Corporate Presentation | Calendar Q2/2012
  • 6. Our Significant Expertise in JAK Research YM acquired original intellectual assets Intellectual Property in JAK field CYT387 Composition of Matter – Lead product CYT387 US: Pending, 2028 expiry – Wholly-owned and un-partnered EU: Pending, 2028 expiry – First group to publish crystal JAK2 Crystal Structure structures of JAK2 and JAK1 US: Issued, 2025 expiry – Medicinal chemistry and molecular EU: Pending, 2026 expiry modeling expertise JAK2 Enzyme – Novartis collaboration for selective US: Issued, 2015 expiry JAK3 inhibitors EU: Issued, 2011 expiry 6 YM Corporate Presentation | Calendar Q2/2012
  • 7. CYT387: A Potent, Selective JAK1/JAK2 Inhibitor – Highly specific, small molecule, ATP competitive, JAK1/JAK2 inhibitor – Rationally designed for optimal JAK binding – Inhibits cellular proliferation driven by JAK2 or JAK1, but not JAK3 or other mechanisms – Inhibits STAT phosphorylation downstream of constitutively active JAK2 or cytokine stimulated JAK1 or JAK2 – Orally active – Very favourable preclinical profile 7 YM Corporate Presentation | Calendar Q2/2012
  • 8. Clinical Presentations of Myelofibrosis – Decreased erythropoiesis or thrombopoiesis – Proliferation of dysfunctional megakaryocytes – Hypercellular bone marrow leading to fibrosis – Extramedullary hematopoiesis – Elevated cytokine levels – Anemia – often requiring transfusions – Thrombocytopenia – Splenomegaly – Constitutional symptoms Fatigue, night sweats, pruritus, bone pain, weight loss, fever 8 YM Corporate Presentation | Calendar Q2/2012
  • 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 9 YM Corporate Presentation | Calendar Q2/2012
  • 10. CYT387: Target Product Profile CYT387 is able to provide a clinically meaningful benefit to myelofibrosis patients by: 1. Converting 2. Reducing spleen 3. Improving transfusion dependent volume in patients constitutional patients to transfusion with enlarged symptoms and independent status spleens quality of life … while having a significantly lower risk of causing or worsening hematological adverse events such as thrombocytopenia, anemia and neutropenia. 10 YM Corporate Presentation | Calendar Q2/2012
  • 11. Safety and Efficacy of CYT387 Updated Phase I/II Data – ASH 2011 11 YM Corporate Presentation | Calendar Q2/2012
  • 12. Current Study Status 166 Patient Phase I/II Study Dose Dose Dose Dose Escalation Confirmation Expansion Extension Phase Phase Phase Study (n = 21) (n = 39) (n = 106) (n = 104) Initiated Nov 2009 Initiated Aug 2010 Initiated Dec 2010 Enrollment complete 100 mg QD (n=3) Data collection and 150 mg QD (n=3) 150 mg QD (n=18) 150 mg QD (n = 31) analysis ongoing 200 mg QD (n=3) 300 mg QD (n=21) 300 mg QD (n = 33) 300 mg QD (n=6) 150 mg BID (n = 42) 97% of patients 400 mg QD (n=6) entered the Extension phase as at ASH 2011 DLT level: 400 mg QD Mayo Clinic (Rochester, Minnesota) MTD: 300 mg QD Dana Farber Cancer Institute (Boston) Stanford Cancer Center (Stanford) Mayo Clinic Mayo Clinic Royal Melbourne Hospital (Melbourne) Princess Margaret Hospital (Toronto) Jewish General Hospital (Montreal) 12 YM Corporate Presentation | Calendar Q2/2012
  • 13. CYT387 Evaluated in an Advanced Population CYT387 Ruxolitinib Ruxolitinib Ruxolitinib Baseline Characteristics Phase I/II Phase III Phase III Phase II (ASH 2011) (Comfort-1) (Comfort-2) (NEJM) Primary Myelofibrosis (%) 65% 45% 53% 53% Post-PV Myelofibrosis (%) 22% 31% 31% 31% Post-ET Myelofibrosis (%) 13% 24% 16% 16% Median Hemoglobin (g/dL) 9.5 10.5 10.4 10.4 9 Median Platelets (10 /L) 187.5 262 236 (all pts) 263 9 Median ANC (10 /L) 8.3 Not reported Not reported 12.1 Spleen length (cm) 17 16 15 19 3 Spleen volume (cm ) 3667 2595 2381 < 21% (estd*) < 21% (estd*) (unspecified Transfusion dependent (IWG) (%) 44% (Unspecified (Unspecified criteria) criteria) criteria) * Product label cited that 21% of patients had RBC transfusion within 8 weeks of enrollment in the study. Maximum estimation of potential transfusion dependent patients 13 YM Corporate Presentation | Calendar Q2/2012
  • 14. Subject Disposition – Median follow-up (range) for Core and Extension: 10.4 months (0.8 – 25.6) – Median follow-up (range) for Core: 9.0 months (0.8 – 9.0) – Study discontinuation during Core: n=32 (19%); 81% retention rate – Reasons for discontinuation during Core (n=32) > Unrelated comorbid conditions (n=11) > Subject withdrew consent (n=5) > Possibly or probably related adverse events (n=5) – Neuropathy (n=2) – Cough (n=1) – Transaminitis (n=1) – Thrombocytopenia (n=1) > Disease progression (n=4) > Investigator’s decision (n=4) > Stem cell transplant (n=2) > Intercurrent illness (n=1) – Deaths during Core n=12 (7%) – Study discontinuation during Extension n= 16 (21%); 79% retention rate 14 YM Corporate Presentation | Calendar Q2/2012
  • 15. Transfusion Independence Response 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 15 YM Corporate Presentation | Calendar Q2/2012
  • 16. Maximum Duration of Transfusion-Free Period* Responders 0 100 200 300 400 500 Time (days) * To date 16 YM Corporate Presentation | Calendar Q2/2012
  • 17. Spleen Response 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 17 YM Corporate Presentation | Calendar Q2/2012
  • 18. Maximal Change in Palpable Spleen Size* (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 18 YM Corporate Presentation | Calendar Q2/2012
  • 19. Spleen Response: MRI vs. Palpation MRI1 (n=11) Palpation (n=11) Response at three months 64%2 45%3 Median decrease from baseline at three months -41% -45% – Median spleen volume at baseline 3667 cm3 Spleen Response: Change from Baseline at Three Months 20% Spleen Volume (n=11) Spleen Length (n=11) 0% % Change from Baseline -20% -40% -41% -45% -60% -80% -100% 1 Includes 1 subject with CT assessment 2 Spleen response defined as a 35% reduction in spleen volume from baseline 3 Spleen response defined as a 50% reduction in spleen length from baseline 19 YM Corporate Presentation | Calendar Q2/2012
  • 20. Constitutional Symptoms Response at Six Months 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) 20 YM Corporate Presentation | Calendar Q2/2012
  • 21. Related Hematologic Adverse Events Adverse Event (n=166) All Grades Grade 3 Grade 4 Thrombocytopenia 33% 11% 6% Baseline platelets > 100 x 109/L 26% 6% 2% Baseline platelets > 200 x 109/L 12% 4% 0 Neutropenia 6% 1% 2% Anemia 4% 1% 0 Leukopenia 2% <1% <1% Leukocytosis 1% <1% 0 At least possibly related to study drug Common Terminology Criteria for Adverse Events v3.0 21 YM Corporate Presentation | Calendar Q2/2012
  • 22. Related Non-Hematologic Laboratory Adverse Events Adverse Event (n=166) Incidence ≥ 10% All Grades Grade 3 Grade 4 Dizziness1 20% 0 0 Neuropathy peripheral 20% 0 0 Diarrhea 18% 0 0 Nausea 17% 0 <1% Headache 12% 1% 0 Adverse Event (n=166) All Grades Grade 3 Grade 4 First Dose Effect2 20% 0 0 Dizziness 11% 0 0 Flushing 5% 0 0 Hypotension 6% 0 0 1 Includes First Dose Effect At least possibly related to study drug 2 Only events occurring on first dose date Common Terminology Criteria for Adverse Events v3.0 22 YM Corporate Presentation | Calendar Q2/2012
  • 23. Related Non-Hematologic Laboratory Adverse Events Adverse Event (n=166) Incidence ≥ 5% All Grades Grade 3 Grade 4 ALT increased 8% 2% 0 Lipase increased 8% 4% 0 AST increased 7% 1% <1% Amylase increased 6% 0 0 Bilirubin increased 6% 0 0 Creatinine increased 6% 0 0 At least possibly related to study drug Common Terminology Criteria for Adverse Events v3.0 23 YM Corporate Presentation | Calendar Q2/2012
  • 24. CYT387 – Safe, Effective, Differentiated CYT387 treatment results in significant, durable responses in anemia, splenomegaly and constitutional symptoms at 150 mg QD, 300 mg QD and 150 mg BID dose levels – 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 24 YM Corporate Presentation | Calendar Q2/2012
  • 25. CYT387 – Safe, Effective, Differentiated CYT387 is well tolerated in myelofibrosis patients for dosing periods up to and exceeding two years – Reported adverse effects include thrombocytopenia; transient, mild dizziness; mild peripheral neuropathy; and abnormalities in liver/pancreas-related laboratory tests – Treatment-emergent anemia and neutropenia are rare Although additional assessments and analyses are ongoing, 300 mg QD appears to be a safe and effective dosing regimen that warrants further clinical development 25 YM Corporate Presentation | Calendar Q2/2012
  • 26. CYT387 Has the Best Fit in Myelofibrosis Variable Diagnosis* >1 year* CYT387 Anemia 38% 64% Benefit Transfusion dependency 25% 45% Benefit Thrombocytopenia 18% 31% Minimal Palpable spleen >10cm 21% 46% Benefit Constitutional symptoms 29% 34% Benefit – CYT387 has a superior profile to other JAK inhibitors in relation to clinical needs and overarching risk factors > Superior benefit on anemia and transfusion dependency > Comparable activity for spleen and symptoms > Less myelosuppression * Mayo Clin Proc 2012;87(1): 25-33 26 YM Corporate Presentation | Calendar Q2/2012
  • 27. CYT387 Next Steps 27 YM Corporate Presentation | Calendar Q2/2012
  • 28. CYT387 BID Study Design Dose Starting at 200mg BID, increasing by 50mg BID escalation Recruiting approximately 60 patients study Initiated in calendar Q3 2011 – Designed to explore BID schedule to complement QD MTD – Spleen response measured by palpation and MRI – Constitutional symptoms assessed using the Myelofibrosis Symptom Assessment Form (MFSAF) – To assess anemia response, a minimum three months of transfusion history will be collected – Comprehensive blood work and biomarkers being recorded for correlative/mechanistic studies Mayo Clinic MD Anderson Huntsman Cancer Princess Margaret Jewish General (Arizona) (Texas) Institute (Utah) (Ontario) (Quebec) * Mayo Clin Proc 2012;87(1): 25-33 28 YM Corporate Presentation | Calendar Q2/2012
  • 29. CYT387 Myelofibrosis Development Pathway Extension study 166-patient Phase I/II Dec 2010 Q2 2011 Q3 2011 Q4 2011 Q4 2012 Interim data Interim data Completed Multicenter Report final core at ASH at ASCO enrollment data at ASH study data & extension study data ~60 patient Phase II BID Q3 2011 Q4 2012 Initiated enrollment Report data Pivotal program H2 2012 Initiate enrollment 29 YM Corporate Presentation | Calendar Q2/2012
  • 30. CYT387 Development and Commercial Opportunities Myeloproliferative Hematological Solid Neoplasms Disorders Tumors – Myelofibrosis – Myelodysplastic – Prostate cancer First line Syndromes (MDS) – Hepatocellular Ruxolitinib failures – Multiple Myeloma – NSCLC Combinations – AML, CML, ALL – Gastric – NHL, Lymphoma – Colorectal – PV/ET – Etc. – Orphan MPNs Clinical proof-of-concept Expand hematological Market expansion Speed to market strategy indications/franchise Lifecycle management 30 YM Corporate Presentation | Calendar Q2/2012
  • 31. Promising Pipeline – Exciting Future CYT387 Nimotuzumab CYT997 JAK1/JAK2 inhibitor anti-EGFR mAb Tubulin inhibitor Oral, small molecule Prospect for enhanced safety Small molecule vascular in “billion dollar” class disrupting agent (VDA) Wholly owned, unpartnered and differentiated De-risked, over 10,000 Phase I clinical proof of patients dosed concept demonstrated Potentially very broad utility (hematology, liquid tumors, Partners advancing Phase Phase II (iv) in glioblastoma solid tumors) II’s and III’s globally multiforme Multicenter Phase I/II data YM retains North American Reformulating as an oral in myelofibrosis reported rights drug at ASH 2011 Kinase Inhibitor Preclinical Research Programs 31 YM Corporate Presentation | Calendar Q2/2012
  • 32. 32 YM Corporate Presentation | Calendar Q2/2012