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Corporate Presentation
                                                       ASH 2012

1   YM Corporate Presentation | ASH 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. 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 | ASH 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
    – Favourable preclinical profile




3    YM Corporate Presentation | ASH 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

4   YM Corporate Presentation | ASH 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 patients are transfusion dependent‡,
        majority of whom 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

5      YM Corporate Presentation | ASH 2012
CYT387: Target Product Profile


                    CYT387 is able to provide myelofibrosis
                   patients with clinically meaningful benefits:


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




6   YM Corporate Presentation | ASH 2012
Safety and Efficacy of CYT387
                                             Phase I/II Data – ASH 2012

7   YM Corporate Presentation | ASH 2012
Phase I/II Study of CYT387, a JAK1/JAK2
              Inhibitor for the Treatment of Myelofibrosis
                                                         1Mayo   Clinic, Rochester, MN
                                           2Stanford   University School of Medicine, Stanford, CA
                                                3Princess    Margaret Hospital, Toronto, ON
                                              4Royal   Melbourne Hospital, Parkville, Australia
                                                5Dana    Farber Cancer Institute, Boston, MA
                                                 6Jewish    General Hospital, Montreal, QC
                                                 7YM    BioSciences Inc., Mississauga, ON


                                              54th ASH Annual Meeting, December 9, 2012


    Animesh Pardanani1, MBBS, PhD; Jason Gotlib2, MD, M.S.; Vikas Gupta3, MBBS, MD, MRCP, FRCPath; Andrew W
    Roberts4, MBBS, PhD, FRACP, FRCPA; Martha Wadleigh5, MD; Shireen Sirhan6, MD, FRCP; Jun Kawashima7, MD;
    Linda M Bavisotto7, MD; Mark Kowalski7, MD, PhD; and Ayalew Tefferi1, MD.



8   YM Corporate Presentation | ASH 2012
A Phase I/II Open-Label, Non-Randomized,
    Dose-Escalation Study

                                       Core                                      Extension
                                       Study                                       Study
                                      (n=166)                                     (n=120)


            9 months of dosing in Core Study                            Continued long term dosing

            Dosing complete                                             Dosing ongoing

            100 mg QD           (n=3)                                   73 patients (61%) remain in the
            150 mg QD           (n=52)      *                       *   Extension Study
                                                150 mg BID (n=42)
            200 mg QD           (n=3)
            300 mg QD           (n=60)      *                           Data collection and analysis
            400 mg QD           (n=6)                                   ongoing

            DLT level: 400 mg QD
            MTD: 300 mg QD

    *Dose level targeted for dose expansion

9    YM Corporate Presentation | ASH 2012
Key Entry Criteria


 •     PMF or post-ET/PV MF as per revised WHO criteria

 •     High risk or Intermediate-2 risk MF (as defined by the International Prognostic
       Scoring System [IPSS]); or

 •     Intermediate-I risk MF (IPSS) associated with symptomatic
       splenomegaly/hepatomegaly and/or unresponsive to available therapy

 •     Evidence of acceptable organ function within seven days of initiating study drug,
       including:

         •    Absolute neutrophil count ≥ 500/μL
         •    Platelet count ≥ 50,000/μL




10   YM Corporate Presentation | ASH 2012
Key Clinical Endpoints

     Clinical Responses:
     •       Anemia response according to IWG-MRT criteria
         •     Minimum transfusion-free period of 12 weeks (transfusion dependency by IWG-MRT)
         •     Minimum 2 g/dL increase in hemoglobin (requires hemoglobin <10 g/dL at baseline)

     •       Spleen response according to IWG-MRT criteria
         •     Minimum 50% decrease from baseline in palpable spleen length lasting at least 8 weeks
               (for baseline spleens ≥10 cm)
         •     100% decrease from baseline in palpable spleen length lasting at least 8 weeks (for
               baseline spleens >5 cm and <10 cm)

     •       Improvement in constitutional symptoms

     Safety Assessments:
     •       MTD determination
     •       Adverse events
     •       Laboratory assessments

11   YM Corporate Presentation | ASH 2012
Subject Baseline Characteristics

Characteristic                              Value    Characteristic                                                      Value

                                                     Prior Therapy
Number of Subjects                           166       JAK2 inhibitor (ruxolitinib, TG101348, unspecified)                13%
                                                       IMiDs (pomalidomide, thalidomide, lenalidomide)                     9%

Age (years)
                                                     Palpable Splenomegaly > 10cm                                         79%
   Median                                    68
   Range                                    34-89
                                                     Spleen Size (cm) (Spleen Evaluable; n=145)
Sex                                                     Mean (STD)                                                     18.3 (6.4)
   Male                                     58%         Median                                                           18.0
   Female                                   42%
                                                     RBC Transfusion Dependent (Evaluable; n=68)                          44%
Myelofibrosis
  Primary                                   63%      Median Hemoglobin (g/dL)
  Post-polycythemia vera                    22%        All subjects (n=166)                                                9.4
  Post-essential thrombocythemia            15%        Transfusion independent subjects (n=93)                            10.3
                                                       Transfusion dependent subjects (n=73)                               8.7
JAK2V617F Positive                          77%
                                                     Platelet Count (109/L)
                                                        Median                                                             182
DIPSS-Plus Category*
                                            10%     * Based on available data; includes unfavorable karyotype, platelet count and
 Int-1
                                            62%     transfusion dependent status as additional prognostic factors.
 Int-2
                                            28%
 High


12   YM Corporate Presentation | ASH 2012
Subject Disposition


     •      Median follow-up (range) for Core and Extension: 16.9 months (0.8 – 34.2*)
     •      Study discontinuation during Core: n=42 (25%); 75% retention rate
     •      Reasons for discontinuation during Core (n=42)
            •      Unrelated comorbid conditions (n=14)
            •      Disease progression (n=8)
            •      Possibly or probably related adverse events (n=8)
                   • Neuropathy (n=2)           • Cough (n=1)
                   • Transaminitis (n=1)        • Thrombocytopenia (n=1)
                   • Hepatitis B (n=1)          • AST increased (n=1)
                   • Muscular weakness (n=1)

            •      Investigator’s decision (n=5)
            •      Subject withdrew consent (n=4)
            •      Stem cell transplant (n=2)
            •      Intercurrent illness (n=1)

     •      Deaths during Core: n=16 (10%)
     *Ongoing as of November 2012

13       YM Corporate Presentation | ASH 2012
Transfusion Independence Response

                                                                      150 mg QD   300 mg QD      150 mg BID       Total1
Response by Dose (Core Study)
                                                                        (n=52)      (n=60)         (n=42)        (n=166)

Transfusion dependent at baseline (evaluable)                            24          28             14             68

Transfusion independence rate (12 wks)                                  63%         75%            57%2           68%

Minimum 2 g/dL increase in hemoglobin level (8 wks)                     11%          8%             14%           13%

IWG-MRT anemia response rate                                            48%         55%             36%           48%

     •   Of the transfusion dependent patients who did not achieve a full transfusion independence response,
         23% achieved at least a 50% reduction in transfusion requirement in any 3-month period

Onset and Durability of Response (Core and Extension Study)                               Median          Min-Max
Time to confirmed response (12 weeks) (Core; days) 3                                        85                85-353

Duration of transfusion-free period (12 weeks) (Core and Extension; days) 3         Not yet reached           85-988*
     •   3 additional subjects achieved 12 week transfusion independence response during the Extension Study

1 Includes 100mg QD (n=3), 200mg QD (n=3), and 400mg QD (n=6) doses
2 Not statistically significant vs. 300mg QD
3 Data based on responders

* Ongoing as of November 2012

14   YM Corporate Presentation | ASH 2012
Maximum Duration of Transfusion-Free Period
  (responders with onset in Core Study; n=46)

                                                                                                                                                                    *
                                                                                                                                           *
                                                                                                                                          *
                                                                                                                                      *
                                                                                                                          *
                                                                                                                      *
                                                                                                                      *
                                                                                                                  *
                                                                                                              *
                                                                                                         *
                                                                                                    *
                                                                                                *                                                    Core
                                                                             *
                                                                         *
                                                                         *                                                                           Extension†
                                                                     *
                                                                     *
                                                                 *

                                                         *




                                                                                                                                                       30 months
                                             *




                                                                                                                                                ≥ 6 months: 74%
                                                   12 months




                                                                                    18 months




                                                                                                                          24 months
                            6 months




                                                                                                                                               ≥ 12 months: 50%
                                                                                                                                               ≥ 18 months: 28%
                                                                                                                                               ≥ 24 months: 9%

  0            100               200         300               400           500                        600           700                 800         900          1000
*ongoing                                                                     Days
†data collection ongoing



15    YM Corporate Presentation | ASH 2012
Percentage of Patients Receiving RBC Transfusions
in Prior 4 Weeks
                                50%

                                45%
 % Patients with Transfusions




                                40%                                                                      Week 0: 166 patients
                                                                                                        Week 40: 125 patients
                                35%

                                30%

                                25%

                                20%

                                15%

                                10%

                                  5%

                                  0%
                                         0            4                8   12   16    20     24    28     32       36       40
                                                                                Weeks Post Day 1

16                              YM Corporate Presentation | ASH 2012
Spleen Response

                                                                   150 mg QD     300 mg QD       150 mg BID       Total1
     Response by Dose (Core Study)
                                                                     (n=52)        (n=60)          (n=42)        (n=166)
     Spleen evaluable (n)                                                  47       51              37             145

     Spleen response (IWG-MRT)                                            32%      39%              38%           37%
     Median spleen decrease at six months2                                -36%     -38%            -46%           -38%

     •      3 additional subjects achieved spleen response during the Extension Study
     •      11 subjects were evaluable for MRI response at six months (≥ 35% decrease in spleen volume)
               •     45% response at six months
               •     41% median decrease from baseline at six months


     Onset and Durability of Response (Core and Extension Study)                         Median               Min-Max
     Time to IWG-MRT response (Core; days)                                                 22                  6-260
     Duration of response (Core and Extension; days)                                      744†                56-859*


 1 Includes 100 mg QD (n=3), 200 mg QD (n=3), and 400 mg QD (n=6) doses
 2 Based on patients with C6D29 spleen assessment reported
 * Ongoing as of November 2012
 † As of November 2012



17       YM Corporate Presentation | ASH 2012
Maximal Change in Palpable Spleen Size
(Core Study; n=145)

                           75%
                                         ≥25% decrease from baseline: 87%
                                         ≥50% decrease from baseline: 50%
                           50%           ≥75% decrease from baseline: 25%
                                         100% decrease from baseline: 17%
 % Change from Baseline




                           25%


                             0%


                          -25%


                          -50%


                          -75%


                    -100%

18                        YM Corporate Presentation | ASH 2012
Constitutional Symptoms Response

                100%
                                  Complete Resolution
                90%               Marked Improvement

                80%

                70%
% of Patients




                60%

                50%                 57%                 52%                            100%
                                                                    33%
                40%
                                                                              29%
                30%

                20%

                                                        24%         26%
                10%                 21%                                       18%

                  0%
                              Night Sweats             Pruritis   Bone Pain   Cough     Fever
                                 (n=62)                (n=46)      (n=43)     (n=28)   (n=10)

19              YM Corporate Presentation | ASH 2012
Related Hematologic Adverse Events



 Adverse Event *(n=166)                                Grade 1   Grade 2   Grade 3   Grade 4

 Anemia                                                  2%        2%        2%        0%

 Leukopenia                                              2%       <1%        0%       <1%

 Neutropenia                                            <1%        2%        1%        2%

 Thrombocytopenia                                       14%        8%       17%        7%
   Baseline platelets > 150 x 109/L (n=97)              18%        1%       9%         1%




*Events occurring in more than one subject
 At least possibly related to study drug
 Common Terminology Criteria for Adverse Events v3.0


20   YM Corporate Presentation | ASH 2012
Related Non-Hematologic Adverse Events


Adverse Event (n=166) Incidence ≥ 10%                 Grade 1   Grade 2   Grade 3   Grade 4

Diarrhea                                               18%        4%        0%        0%

Dizziness1                                             22%        1%        0%        0%

Headache                                               14%        0%        1%        0%

Nausea                                                 20%       <1%        0%        0%

Neuropathy peripheral                                  25%        2%        0%        0%




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

1   Includes First Dose Effect

21     YM Corporate Presentation | ASH 2012
Related Non-Hematologic Laboratory Adverse Events



 Adverse Event (n=166) Incidence ≥ 5%                 Grade 1   Grade 2   Grade 3   Grade 4

 ALP increased                                          5%        1%        1%        0%

 ALT increased                                          9%        3%        2%        0%

 Amylase increased                                      7%        2%        0%        0%

 AST increased                                          9%        2%        1%        0%

 Bilirubin increased                                    7%        2%        0%        0%

 Creatinine increased                                   5%        3%        0%        0%

 Lipase increased                                       8%       <1%        4%        0%




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

22    YM Corporate Presentation | ASH 2012
CYT387 Conclusions

CYT387 treatment results in significant, durable responses in anemia, splenomegaly and
constitutional symptoms in MF patients

     – Substantial decrease in percentage of patients requiring transfusions during study (44% at
       baseline; <10% at week 40)

     – 68% of patients achieved a durable 12-week transfusion independence response rate with a
       maximal duration of response approaching 3 years; overall IWG anemia response rate was
       48%

     – 50% of transfusion independence responders achieved a transfusion-free period lasting ≥ 1
       year

     – 37% of patients exhibited a durable spleen response per IWG-MRT with a maximal duration
       of response of nearly 2.5 years

     – Complete resolution or marked improvement of common constitutional symptoms is achieved
       in the majority of subjects

23   YM Corporate Presentation | ASH 2012
CYT387 Conclusions



•    CYT387 is well tolerated in myelofibrosis patients for dosing periods currently up to
     three years and ongoing

     – Majority of adverse events are Grade 1

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

     – Treatment-emergent anemia and neutropenia are rare

•    300mg QD is a safe dosing regimen providing compelling efficacy and has been
     selected for use in the Phase III clinical development program




24   YM Corporate Presentation | ASH 2012
Acknowledgements

     • Animesh Pardanani, Mayo Clinic, Rochester, MN
     • Jason Gotlib, Stanford University School of Medicine, Stanford, CA
     • Vikas Gupta, Princess Margaret Hospital, Toronto, ON
     • Andrew Roberts, Royal Melbourne Hospital, Parkville, Australia
     • Martha Wadleigh, Dana Farber Cancer Institute, Boston, MA
     • Shireen Sirhan, Jewish General Hospital, Montreal, QC
     • Patients and their Families




25   YM Corporate Presentation | ASH 2012
CYT387 Next Steps


26   YM Corporate Presentation | ASH 2012
CYT387 Well Suited for 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 three years


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

27   YM Corporate Presentation | ASH 2012
CYT387 BID Dose Escalation Study

–     Designed to explore BID schedule to complement
      QD MTD (Starting at 200mg BID, increasing by
      50mg BID)
                                                                           – Enrolment of 61
–     Spleen response measured by palpation and MRI
                                                                             patients completed
–     Constitutional symptoms assessed using the                             Q3 2012
      Myelofibrosis Symptom Assessment Form
                                                                           – Median follow-up
      (MFSAF)
                                                                             (range): 5.3 months
–     To assess anemia response, a minimum three                             (0.5 – 13.9)
      months of transfusion history will be collected
–     Comprehensive blood work and biomarkers being
      recorded for correlative/mechanistic studies


                              MD              Huntsman         Princess       Jewish      Mayo
 Mayo Clinic
                           Anderson         Cancer Institute   Margaret       General     Clinic
  (Arizona)
                            (Texas)             (Utah)         (Ontario)     (Quebec)    (Florida)

28   YM Corporate Presentation | ASH 2012
Preliminary Efficacy


                                              MRI Spleen Response
        Response by Visit                                               C3D29

        Spleen evaluable (n)                                             53

        Spleen response* (≥35% decrease from baseline)                   57%
        Median spleen decrease                                          -47%


                                            Palpation Spleen Response
        Spleen Response                                                 C3D29

        Spleen evaluable (n)                                             47
        Insert Palpation Data??
        Spleen response*                                                60%




* Ongoing

29   YM Corporate Presentation | ASH 2012
CYT387: Next Steps

 ASH 2012
         • Phase I/II Core study results strongly reinforce differentiated profile

 Business Development
    • Explored multiple opportunities to develop CYT387 with other
       companies
         • Conducted a broad, robust business development process
         • Rationalized portfolio
         • Process on-track to conclude in the near-term
 Phase III
         • Preparations for Phase III advancing under assumption of
           collaboration/hand-off


30   YM Corporate Presentation | ASH 2012
31   YM Corporate Presentation | ASH 2012

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YM BioSciences CorpPres ASH2012 Dec 10 12

  • 1. Corporate Presentation ASH 2012 1 YM Corporate Presentation | ASH 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. 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 | ASH 2012
  • 3. 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 – Favourable preclinical profile 3 YM Corporate Presentation | ASH 2012
  • 4. 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 4 YM Corporate Presentation | ASH 2012
  • 5. 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 patients are transfusion dependent‡, majority of whom 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 5 YM Corporate Presentation | ASH 2012
  • 6. CYT387: Target Product Profile CYT387 is able to provide myelofibrosis patients with clinically meaningful benefits: 1. Converting 2. Reducing spleen 3. Improving transfusion volume in constitutional dependent patients with symptoms and patients to enlarged quality of life transfusion spleens independence 6 YM Corporate Presentation | ASH 2012
  • 7. Safety and Efficacy of CYT387 Phase I/II Data – ASH 2012 7 YM Corporate Presentation | ASH 2012
  • 8. Phase I/II Study of CYT387, a JAK1/JAK2 Inhibitor for the Treatment of Myelofibrosis 1Mayo Clinic, Rochester, MN 2Stanford University School of Medicine, Stanford, CA 3Princess Margaret Hospital, Toronto, ON 4Royal Melbourne Hospital, Parkville, Australia 5Dana Farber Cancer Institute, Boston, MA 6Jewish General Hospital, Montreal, QC 7YM BioSciences Inc., Mississauga, ON 54th ASH Annual Meeting, December 9, 2012 Animesh Pardanani1, MBBS, PhD; Jason Gotlib2, MD, M.S.; Vikas Gupta3, MBBS, MD, MRCP, FRCPath; Andrew W Roberts4, MBBS, PhD, FRACP, FRCPA; Martha Wadleigh5, MD; Shireen Sirhan6, MD, FRCP; Jun Kawashima7, MD; Linda M Bavisotto7, MD; Mark Kowalski7, MD, PhD; and Ayalew Tefferi1, MD. 8 YM Corporate Presentation | ASH 2012
  • 9. A Phase I/II Open-Label, Non-Randomized, Dose-Escalation Study Core Extension Study Study (n=166) (n=120) 9 months of dosing in Core Study Continued long term dosing Dosing complete Dosing ongoing 100 mg QD (n=3) 73 patients (61%) remain in the 150 mg QD (n=52) * * Extension Study 150 mg BID (n=42) 200 mg QD (n=3) 300 mg QD (n=60) * Data collection and analysis 400 mg QD (n=6) ongoing DLT level: 400 mg QD MTD: 300 mg QD *Dose level targeted for dose expansion 9 YM Corporate Presentation | ASH 2012
  • 10. Key Entry Criteria • PMF or post-ET/PV MF as per revised WHO criteria • High risk or Intermediate-2 risk MF (as defined by the International Prognostic Scoring System [IPSS]); or • Intermediate-I risk MF (IPSS) associated with symptomatic splenomegaly/hepatomegaly and/or unresponsive to available therapy • Evidence of acceptable organ function within seven days of initiating study drug, including: • Absolute neutrophil count ≥ 500/μL • Platelet count ≥ 50,000/μL 10 YM Corporate Presentation | ASH 2012
  • 11. Key Clinical Endpoints Clinical Responses: • Anemia response according to IWG-MRT criteria • Minimum transfusion-free period of 12 weeks (transfusion dependency by IWG-MRT) • Minimum 2 g/dL increase in hemoglobin (requires hemoglobin <10 g/dL at baseline) • Spleen response according to IWG-MRT criteria • Minimum 50% decrease from baseline in palpable spleen length lasting at least 8 weeks (for baseline spleens ≥10 cm) • 100% decrease from baseline in palpable spleen length lasting at least 8 weeks (for baseline spleens >5 cm and <10 cm) • Improvement in constitutional symptoms Safety Assessments: • MTD determination • Adverse events • Laboratory assessments 11 YM Corporate Presentation | ASH 2012
  • 12. Subject Baseline Characteristics Characteristic Value Characteristic Value Prior Therapy Number of Subjects 166 JAK2 inhibitor (ruxolitinib, TG101348, unspecified) 13% IMiDs (pomalidomide, thalidomide, lenalidomide) 9% Age (years) Palpable Splenomegaly > 10cm 79% Median 68 Range 34-89 Spleen Size (cm) (Spleen Evaluable; n=145) Sex Mean (STD) 18.3 (6.4) Male 58% Median 18.0 Female 42% RBC Transfusion Dependent (Evaluable; n=68) 44% Myelofibrosis Primary 63% Median Hemoglobin (g/dL) Post-polycythemia vera 22% All subjects (n=166) 9.4 Post-essential thrombocythemia 15% Transfusion independent subjects (n=93) 10.3 Transfusion dependent subjects (n=73) 8.7 JAK2V617F Positive 77% Platelet Count (109/L) Median 182 DIPSS-Plus Category* 10% * Based on available data; includes unfavorable karyotype, platelet count and Int-1 62% transfusion dependent status as additional prognostic factors. Int-2 28% High 12 YM Corporate Presentation | ASH 2012
  • 13. Subject Disposition • Median follow-up (range) for Core and Extension: 16.9 months (0.8 – 34.2*) • Study discontinuation during Core: n=42 (25%); 75% retention rate • Reasons for discontinuation during Core (n=42) • Unrelated comorbid conditions (n=14) • Disease progression (n=8) • Possibly or probably related adverse events (n=8) • Neuropathy (n=2) • Cough (n=1) • Transaminitis (n=1) • Thrombocytopenia (n=1) • Hepatitis B (n=1) • AST increased (n=1) • Muscular weakness (n=1) • Investigator’s decision (n=5) • Subject withdrew consent (n=4) • Stem cell transplant (n=2) • Intercurrent illness (n=1) • Deaths during Core: n=16 (10%) *Ongoing as of November 2012 13 YM Corporate Presentation | ASH 2012
  • 14. Transfusion Independence Response 150 mg QD 300 mg QD 150 mg BID Total1 Response by Dose (Core Study) (n=52) (n=60) (n=42) (n=166) Transfusion dependent at baseline (evaluable) 24 28 14 68 Transfusion independence rate (12 wks) 63% 75% 57%2 68% Minimum 2 g/dL increase in hemoglobin level (8 wks) 11% 8% 14% 13% IWG-MRT anemia response rate 48% 55% 36% 48% • Of the transfusion dependent patients who did not achieve a full transfusion independence response, 23% achieved at least a 50% reduction in transfusion requirement in any 3-month period Onset and Durability of Response (Core and Extension Study) Median Min-Max Time to confirmed response (12 weeks) (Core; days) 3 85 85-353 Duration of transfusion-free period (12 weeks) (Core and Extension; days) 3 Not yet reached 85-988* • 3 additional subjects achieved 12 week transfusion independence response during the Extension Study 1 Includes 100mg QD (n=3), 200mg QD (n=3), and 400mg QD (n=6) doses 2 Not statistically significant vs. 300mg QD 3 Data based on responders * Ongoing as of November 2012 14 YM Corporate Presentation | ASH 2012
  • 15. Maximum Duration of Transfusion-Free Period (responders with onset in Core Study; n=46) * * * * * * * * * * * * Core * * * Extension† * * * * 30 months * ≥ 6 months: 74% 12 months 18 months 24 months 6 months ≥ 12 months: 50% ≥ 18 months: 28% ≥ 24 months: 9% 0 100 200 300 400 500 600 700 800 900 1000 *ongoing Days †data collection ongoing 15 YM Corporate Presentation | ASH 2012
  • 16. Percentage of Patients Receiving RBC Transfusions in Prior 4 Weeks 50% 45% % Patients with Transfusions 40% Week 0: 166 patients Week 40: 125 patients 35% 30% 25% 20% 15% 10% 5% 0% 0 4 8 12 16 20 24 28 32 36 40 Weeks Post Day 1 16 YM Corporate Presentation | ASH 2012
  • 17. Spleen Response 150 mg QD 300 mg QD 150 mg BID Total1 Response by Dose (Core Study) (n=52) (n=60) (n=42) (n=166) Spleen evaluable (n) 47 51 37 145 Spleen response (IWG-MRT) 32% 39% 38% 37% Median spleen decrease at six months2 -36% -38% -46% -38% • 3 additional subjects achieved spleen response during the Extension Study • 11 subjects were evaluable for MRI response at six months (≥ 35% decrease in spleen volume) • 45% response at six months • 41% median decrease from baseline at six months Onset and Durability of Response (Core and Extension Study) Median Min-Max Time to IWG-MRT response (Core; days) 22 6-260 Duration of response (Core and Extension; days) 744† 56-859* 1 Includes 100 mg QD (n=3), 200 mg QD (n=3), and 400 mg QD (n=6) doses 2 Based on patients with C6D29 spleen assessment reported * Ongoing as of November 2012 † As of November 2012 17 YM Corporate Presentation | ASH 2012
  • 18. Maximal Change in Palpable Spleen Size (Core Study; n=145) 75% ≥25% decrease from baseline: 87% ≥50% decrease from baseline: 50% 50% ≥75% decrease from baseline: 25% 100% decrease from baseline: 17% % Change from Baseline 25% 0% -25% -50% -75% -100% 18 YM Corporate Presentation | ASH 2012
  • 19. Constitutional Symptoms Response 100% Complete Resolution 90% Marked Improvement 80% 70% % of Patients 60% 50% 57% 52% 100% 33% 40% 29% 30% 20% 24% 26% 10% 21% 18% 0% Night Sweats Pruritis Bone Pain Cough Fever (n=62) (n=46) (n=43) (n=28) (n=10) 19 YM Corporate Presentation | ASH 2012
  • 20. Related Hematologic Adverse Events Adverse Event *(n=166) Grade 1 Grade 2 Grade 3 Grade 4 Anemia 2% 2% 2% 0% Leukopenia 2% <1% 0% <1% Neutropenia <1% 2% 1% 2% Thrombocytopenia 14% 8% 17% 7% Baseline platelets > 150 x 109/L (n=97) 18% 1% 9% 1% *Events occurring in more than one subject At least possibly related to study drug Common Terminology Criteria for Adverse Events v3.0 20 YM Corporate Presentation | ASH 2012
  • 21. Related Non-Hematologic Adverse Events Adverse Event (n=166) Incidence ≥ 10% Grade 1 Grade 2 Grade 3 Grade 4 Diarrhea 18% 4% 0% 0% Dizziness1 22% 1% 0% 0% Headache 14% 0% 1% 0% Nausea 20% <1% 0% 0% Neuropathy peripheral 25% 2% 0% 0% At least possibly related to study drug Common Terminology Criteria for Adverse Events v3.0 1 Includes First Dose Effect 21 YM Corporate Presentation | ASH 2012
  • 22. Related Non-Hematologic Laboratory Adverse Events Adverse Event (n=166) Incidence ≥ 5% Grade 1 Grade 2 Grade 3 Grade 4 ALP increased 5% 1% 1% 0% ALT increased 9% 3% 2% 0% Amylase increased 7% 2% 0% 0% AST increased 9% 2% 1% 0% Bilirubin increased 7% 2% 0% 0% Creatinine increased 5% 3% 0% 0% Lipase increased 8% <1% 4% 0% At least possibly related to study drug Common Terminology Criteria for Adverse Events v3.0 22 YM Corporate Presentation | ASH 2012
  • 23. CYT387 Conclusions CYT387 treatment results in significant, durable responses in anemia, splenomegaly and constitutional symptoms in MF patients – Substantial decrease in percentage of patients requiring transfusions during study (44% at baseline; <10% at week 40) – 68% of patients achieved a durable 12-week transfusion independence response rate with a maximal duration of response approaching 3 years; overall IWG anemia response rate was 48% – 50% of transfusion independence responders achieved a transfusion-free period lasting ≥ 1 year – 37% of patients exhibited a durable spleen response per IWG-MRT with a maximal duration of response of nearly 2.5 years – Complete resolution or marked improvement of common constitutional symptoms is achieved in the majority of subjects 23 YM Corporate Presentation | ASH 2012
  • 24. CYT387 Conclusions • CYT387 is well tolerated in myelofibrosis patients for dosing periods currently up to three years and ongoing – Majority of adverse events are Grade 1 – Common reported adverse events include thrombocytopenia; transient, mild dizziness; mild peripheral neuropathy; and abnormalities in liver/pancreas-related laboratory tests – Treatment-emergent anemia and neutropenia are rare • 300mg QD is a safe dosing regimen providing compelling efficacy and has been selected for use in the Phase III clinical development program 24 YM Corporate Presentation | ASH 2012
  • 25. Acknowledgements • Animesh Pardanani, Mayo Clinic, Rochester, MN • Jason Gotlib, Stanford University School of Medicine, Stanford, CA • Vikas Gupta, Princess Margaret Hospital, Toronto, ON • Andrew Roberts, Royal Melbourne Hospital, Parkville, Australia • Martha Wadleigh, Dana Farber Cancer Institute, Boston, MA • Shireen Sirhan, Jewish General Hospital, Montreal, QC • Patients and their Families 25 YM Corporate Presentation | ASH 2012
  • 26. CYT387 Next Steps 26 YM Corporate Presentation | ASH 2012
  • 27. CYT387 Well Suited for 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 three years * Mayo Clin Proc 2012;87(1): 25-33 27 YM Corporate Presentation | ASH 2012
  • 28. CYT387 BID Dose Escalation Study – Designed to explore BID schedule to complement QD MTD (Starting at 200mg BID, increasing by 50mg BID) – Enrolment of 61 – Spleen response measured by palpation and MRI patients completed – Constitutional symptoms assessed using the Q3 2012 Myelofibrosis Symptom Assessment Form – Median follow-up (MFSAF) (range): 5.3 months – To assess anemia response, a minimum three (0.5 – 13.9) months of transfusion history will be collected – Comprehensive blood work and biomarkers being recorded for correlative/mechanistic studies MD Huntsman Princess Jewish Mayo Mayo Clinic Anderson Cancer Institute Margaret General Clinic (Arizona) (Texas) (Utah) (Ontario) (Quebec) (Florida) 28 YM Corporate Presentation | ASH 2012
  • 29. Preliminary Efficacy MRI Spleen Response Response by Visit C3D29 Spleen evaluable (n) 53 Spleen response* (≥35% decrease from baseline) 57% Median spleen decrease -47% Palpation Spleen Response Spleen Response C3D29 Spleen evaluable (n) 47 Insert Palpation Data?? Spleen response* 60% * Ongoing 29 YM Corporate Presentation | ASH 2012
  • 30. CYT387: Next Steps ASH 2012 • Phase I/II Core study results strongly reinforce differentiated profile Business Development • Explored multiple opportunities to develop CYT387 with other companies • Conducted a broad, robust business development process • Rationalized portfolio • Process on-track to conclude in the near-term Phase III • Preparations for Phase III advancing under assumption of collaboration/hand-off 30 YM Corporate Presentation | ASH 2012
  • 31. 31 YM Corporate Presentation | ASH 2012