healthcare sector secondary research … · 11 healthcare sector secondary research sample report...

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HEALTHCARE SECTORSECONDARY RESEARCHSAMPLE REPORT

Market Landscape Assessment:Epidemiology, Current Treatments and Unmet Needs, Clinical and Preclinical Pipeline, Review of Key Trials, Emerging Profile of New Therapies

Dan [email protected]

Will [email protected]

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Market Research Intelligence: Make More Informed Investment and Management DecisionsDectiva specializes in delivering quantitative and qualitative market research data and analysis to leading investment and business decision makers. By providing access to a a global panel of survey and interview respondents, Dectiva empowers its clients with proprietary knowledge and timely insights in the life sciences industry. Dectiva serves institutional investment, venture capital, private equity, and corporate entities.

Clients Use Dectiva's Primary Market Research Platform To:» Survey targeted physician and patient groups to forecast demand and rate of adoption for new products» Test new target product profiles to investigate strengths, weaknesses, opportunities, and positioning relative to the competitive set» Identify unmet therapeutic needs and determine potential barriers to new product adoption» Analyze existing target markets to understand usage rates of competing products where sales information is incomplete» Collect early feedback from physicians, payers, and patients to inform new product design and development of target product profiles» Evaluate key drivers of market share to guide product enhancements and post-marketing efforts

Clients Use Dectiva's Secondary Market Research Services To Better Understand:» Disease definition and epidemiology» Current treatments, market size by number of patients and sales volume, and unmet therapeutic needs» Clinical and preclinical pipelines» Status and results of key trials» Emerging profiles of competing new therapies» Pricing, coverage, and reimbursement issues

Clinical Trial Recruitment - Investigators and Patients:Leveraging our global network, Dectiva performs custom recruitment campaigns of clinical investigators and patients for enrollment in clinical trials.

Introduction of services

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Extensive literature review: » Scientific publications» Trade journal and databases» Securities analyst reports» Government sponsored databases» Syndicated databases

(based on client access)

Information distilled to deliver insights, as well as data

Completed within 5-7 days

Secondary ResearchIdeal for companies/investors exploring:

»New products in new areas»New applications for existing products

Collect and analyze technical data on:»Disease classification info »Diagnostic algorithm »Epidemiology »Market drivers »Competitive intelligence

(detailed pipeline and existing market overviews)

Structured and customized reports to support business planning and investment decisions

Completed within 3-5 days

Market Landscape Assessment

Secondary Research Detailed Overview

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Opportunity assessment» Market size, growth» Current treatments» Unmet needs

Competitive landscape analysis» Current treatments, advantages, limitations» Pipeline competitors, trial design and potential positioning

Client BackgroundPharmaceutical client evaluating multiple opportunities for in-licensingNeeded preliminary assessment of market attractiveness based on secondary data to

prioritize different opportunitiesNext steps would be to conduct primary research to validate findings and to quantify value

of specific asset opportunityDisease Area

ThrombocytopeniaDiverse patient populationMinimal syndicated market data available

Case Study - Overview

SECTOR OBJECTIVES

Life Sciences –Thrombocytopenia

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Disease Definition and EpidemiologyCurrent Treatments and Unmet NeedsClinical and Preclinical PipelineKey TrialsConclusionsEmerging Profile of New Therapies

Case Study - Table of Contents

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• Thrombocytopenia is a reduction in platelets (<100,000 platelets/µL), which can lead to excessive bleeding. The condition is caused by:

– Suppression of megakaryocytes (responsible for platelet production) in the bone marrow.– Immune disorders that produce antibodies that destroy platelets.– Medications that result in myelosuppression and/or immunological destruction of platelets.

THROMBOCYTOPENIA CAUSES

Platelet Production Platelet Destruction• Bone marrow diseases (leukemia, lymphoma,

MDS, multiple myeloma)• Myelosuppressive therapies (e.g., chemotherapy,

radiation)• Reduced thrombopoietin in the liver due to liver

disease (chronic hepatitis C, cirrhosis, liver failure)

• Systemic viral or bacterial infection – HIV, HCV, HCB– Dengue fever infects megakaryocytes

• Sepsis• Vitamin B12 or folic acid deficiency• Numerous hereditary syndromes

• Immune or idiopathic thrombocytopenic purpura(ITP)

• Autoimmune disorders (e.g., lupus, rheumatoid arthritis)

• Disseminated intravascular coagulopathy (DIC) associated with cancer, sepsis or infection

• Medications: drug-antibody complexes bind and activate platelets

– Heparin-induced thrombocytopenia is a rare, but serious example of this

Disease Definition: Diverse Causes

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• Medication induced thrombocytopenia has increased with introduction of new therapies that cause hematological complications (e.g., antiinflammatories, cardiac & ulcer drugs, antibiotics).

• Reduced thrombopoietin production in the liver results in thrombocytopenia in patients with chronic HCV infection and liver disease.

• Other forms of thrombocytopenia are more rare; congenital thrombocytopenia, though quite varied, accounts for <X% of all cases.

US INCIDENCE OF THROMBOCYTOPENIACause Comments Est. US 2008 Incidence Annual Growth

ITP1 • Scientific references cite incidence of X/100,000 in adults and X/1MM in children

• Non-scientific references cite X/1MM.

XXXXXBut may be as high as XXXXX

+X%

Chemotherapy induced thrombocytopenia2

• Based on Decision Resources estimates and projections XXXXX +X%

Heparin induced thrombocytopenia (HIT)3

• XX% of patients treated with unfractionated heparin• XX% develop HIT with thrombosis• Estimates based only on cardiovascular patients

XXXXX +X%

Myelodysplaticsyndromes (MDS)4

• MDS est 2008 incidence = XXXXX• Thrombocytopenia occurs in ~XX% of patients

XXXXX +X%

Chronic hepatitis C infection5

• XX M Americans chronically infected• XX% of chronic HCV patients affected

XXXXX +X%

HIV/AIDS6 • XXXXX diagnosed cases of AIDS in US in 200X• 1 yr incidence is XX% in patients with AIDS

XXXXX +X%

Epidemiology: 200,000+ US Cases Per Year

Source: 1. ITP: Frederiksen H, Schmidt K, 1999; 2. Decision Resources, 2007; 3. HIT: Ohman EM, et al, 2005; 4. MDS: Ma X, et al, 2007 (est. 2003 MDS incidence rate of 3.56/100,000 applied to 2008 US population); Hagop K, et al, 2007; 5. HCV: Armstrong GL, et al, 2006; Chong-Shan W, et al, 2004; 6. HIV: CDC, HIV/AIDS Surveillance Report, 2005; Moore R, www.hopkins-hivguide.org

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• ITP treatment guidelines established by American Society of Hematology (ASH) in 1996 are still in place today.• Following acute situations, many patients experience spontaneous remission.• Patients whose symptoms persist require further treatment; ~X% of patients become refractory.

DxDx

ObservationObservation

Drug TxDrug Tx

Platelets >30,000/µL, asymptomatic or minor purpura

Platelets 20,000-30,000/µL and/or significant mucous membrane bleeding

Platelets <20,000/µL and/or significant mucous membrane bleeding

Hospitalization & Drug Tx

Hospitalization & Drug Tx

Glucocorticoids

High dose IV glucocorticoids, IVIG, platelet transfusions

1st Line 2nd Line / 3rd Line

Platelets <30,000/µL after 4-6 weeks of treatment

Consider SplenectomyConsider Splenectomy

Rituximab Off-LabelRituximab Off-Label

Other Experimental TxOther Experimental Tx

ITP TREATMENT APPROACH

Cylcophosphamide / Chemo Comb.

Cylcophosphamide / Chemo Comb.

Current Treatment Approach: Disrupt antibody-mediated destruction

Source: George JN, et al, “ITP: A Practice Guideline Developed by Explicit Methods for the American Society of Hematology,” Blood, 1996

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• In other forms of thrombocytopenia, physicians must assess underlying disease or risk-benefit of treatments causing reduced platelet levels.

Chemotherapy Induced Thrombocytopenia

TREATMENT APPROACH FOR OTHER FORMS OF THROMBOCYTOPENIA

Heparin Induced Thrombocytopenia

Chronic Hepatitis C / Cirrhosis

Antiretroviral Therapy (AZT, ART)

Interferon TherapyLiver Transplant

Direct Thrombin Inhibitors (argatroban, lepirudin)

Platelet transfusionNeumega (oprelvekin, IL-11)

HIV

Current Treatment Approaches: Treat Underlying Cause

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Prednisone IV Gamma Globulin (IVIG)

Rho(D) Immune Glubulin (Anti-D)

Splenectomy Neumega(Oprelvekin)

Rituximab

MOA / Rationale

• Prevents bleeding• Raises platelet count

quickly

• Interrupts antibody mediated platelet destruction

• Approved for prevention of Rhimmunization but also shown to interrupt platelet destruction

• Spleen is site for antiplatelet antibody production and destruction of opsonized platelets

• IL-11 enhances growth and maturation of IL-3 dependent megakaryocytic progenitors

• Eliminate B-lymphocytes needed for autoantibody production

• May contribute to Fcreceptor blockade

Dosing / Admin

• Begin 1mg/kg IV for 3 days; taper to <10-15 mg/d

• 1g/kg/d IV for 2 d • 75µg/kg IM injection • Surgical procedure • 50µg/kg/d SC injection

• 375mg/m2 four times at weekly intervals

Duration of Therapy

• 6 months • Can be intermittent for 18 months

• Can be intermittent for 18 months

• N/A • Given in 10 and 21 day courses

• TBD

Efficacy • Platelet recovery within 4 days vs 16 days in untreated

• Platelet increase in 75% of pts within 3-4 weeks

• 50% achieved normal levels

• 68% response• Transiently increases

platelet counts for 2-3 weeks

• 75-85% of patients have initial response

• 25-40% relapse within 5-10 years

• 28-65% of patients did not require platelet transfusions, vs 7-41% in placebo arms

• 40-46% CRs• 63% overall response• 2-48 months duration

Safety • Adverse events associated with long-term steroid use (hyperglycemia, hypertension, weight gain, growth retardation)

• 15-75% of patients have mild headache, backache, nausea, fever

• Some risk of aseptic meningitis, alloimmunehemolysis

• Risk of alloimmunehemolysis

• Potential for renal failure

• Risks of infection (lifelong use of antibiotics recommended by UK guidelines but not US)

• Boxed warning for allergic reactions

• Constitutional toxicities, peri-pheraledema

• Low incidence of atrial arrhythmias and syncope

• 22% mild/mod adverse events

• 4% severe events

Source: George JN, et al, 1996; Cines DB, et al, “Congenital and Acquired Thrombocytopenia, Hematology, 2004; Beardsley DS, “ITP in the 21st Century,” Hematology, 2006; Panzer S, “New Therapeutic Options for Adult Chronic ITP, Vox Sanguinis, 2008

Current Treatments: Most Drugs are Immunomodulators

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• Current treatment options present risks of significant side effects and efficacy typically achieve 70% response rates or less.

• Even splenectomy, considered a “cure” for ITP, has efficacy of up to XX% but XX% may relapse within 5-10 years.

Source: Panzer S, 2008; Decision Resources, “Managing the Side Effects of Chemotherapy,” 2007

Safer therapies for patients at risk of bleeding or overt bleeding tendency, to postpone splenectomy

Drugs with more rapid onset of action

Effective therapies for refractory patients

Drugs for patients scheduled for minor or major surgery, who can not take steroids or IVIG Diagnostics to more quickly and effectively identify patients with ITP versus other conditions

UNMET NEEDS

Unmet Needs: Safer Treatments and Options for Refractory Cases

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• Even when the cause of thrombocytopenia is platelet destruction, patients may also be subject to decreased platelet production due to:

– Antiplatelet antibodies binding to megakaryocytes– Apoptosis of megakaryocytes– Low thrombopoietin (TPO) levels

• 1st generation TPO growth factors were recombinant megakaryocyte growth factors. These agents tended to be immunogenic and were discontinued.

• 2nd generation agents are TPO peptide and non-peptide mimetics and TPO agonist antibodies. All bind to TPO receptor but in different ways.

• Potential concerns with new TPO agents are thrombosis, development of cancer/leukemia, antibody production, marrow fibrosis and rebound effects when treatment is stopped.

• Of the 11 clinical and 8 preclinical compounds in development for thrombocytopenia, at least 6 target the TPO receptor.

• Two compounds are awaiting FDA approval:– Nplate (Romiplostim/AMG 531) and Promacta (Eltrombopag)– Both will receive fast track or priority review, with launch expected by 20XX

Clinical Pipeline: Focus is on TPO receptor

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Discontinued

Discontinued

TPO Receptor MOA2 MOA3 MOA4Mechanism

of Action

Clinical Pipeline: Two Near-Term Entrants; Very Crowded Post-20XX

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Source: Panzer S, 2008; ADIS R&D Insight; Amgen Press Release Jan 31, 2008

Clinical Pipeline

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Source: Panzer S, 2008; ADIS R&D Insight; GSK Press Release Dec 10, 2007

Clinical Pipeline

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• Three additional TPO agonists are in clinical development – the XXX compounds are follow-up candidates to Promacta.

• Immunomodulators have also generated interest. CD20 antibodies affect the immune system’s ability to make autoantibodies.

Drug Developer MOA Phase Indication ROA

XXXXX XXXXX Thrombopoietinreceptor agonist

X ITP Oral


X ITP Oral

XXXXX XXXXX Syk tyrosine kinaseinhibitor

X ITP Oral

XXXXX XXXXX CD20 antigen antagonist

X ITP IV


X ITP IV

XXXXX XXXXX Platelet activating factor agonist

X ITP IV


X ITP IV

XXXXX XXXXX Anti-RhDimmunoglobulin antagonist

X ITP IV


X Thrombocytopenia Oral

Source: ADIS R&D Insight; IDDB3

Clinical Pipeline

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• Several companies have active research programs targeting thrombocytopenia, but very little data are available to assess activity.

Drug Developer MOA Phase Indication ROA

XXXXX XXXXX IL-1 agonists X Thrombocytopenia Injectable

XXXXX XXXXX ADAM protein stimulants

X TTP Injectable


X Chemo-induced thrombocytopenia

Injectable

XXXXX XXXXX VPAC1 receptor antagonist

X Thrombocytopenia IV

XXXXX XXXXX LG543 growth factor agonist

X Thrombocytopenia Injectable

XXXXX XXXXX Anti-RhDimmunoglobulin antagonist

X ITP IV

XXXXX XXXXX Staphylococcal protein A down regulates human B-lymphocyte and macrophage activation

X ITP Injectable

XXXXX XXXXX Platelet activating factor agonist

X Thrombocytopenia IV

Source: ADIS R&D Insight; IDDB3

Preclinical Pipeline

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• Most pipeline competitors have focused on ITP as their lead indication for registration.– GSK fielded two phase III studies (up to 200 subjects) and Amgen’s phase III had 210 subjects.– Most trials involve patients who are relapsed after at least one standard therapy or are refractory.

• Thrombocytopenia related to hepatitis C or chronic liver disease is another area of investment, where trial sizes are much larger (up to 750 patients).

– HCV related studies aim to address thrombocytopenia such that patients can initiate and/or stay on antiviral therapy.

• Other thrombocytopenia studies in late phases address MDS and chemotherapy patients.

Key Trials Overview

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Compound Cancer Sponsor Phase Patients Design Primary Endpoints

Secondary Endpoints

Timing

Promacta ITP GSK III 200 EXTEND: Open-label, dose-adjustment, extension to evaluate safety and efficacy of eltrombopag for treatment of subjects with ITP previously enrolled in an eltrombopag trial. Study allows individualized dose and schedule for each subject. Ability to reduce the dose of concomitant ITP medications in the presence of eltrombopag, while maintaining platelet counts = 50,000/microL will be investigated.

Safety and tolerability, clinical lab tests, ocular exams and frequency of all AEs

Proportion of patients achieving target platelet counts and duration of count elevation; signs and symptoms of ITP; QOL

Start Jun 2006; end Jul 2010

Promacta ITP GSK III 189 RAISE: Randomized, double-blind, placebo-controlled study, to evaluate efficacy, safety and tolerability of eltrombopag, initially administered as 50 mg oral tablets once daily for six months in adult subjects with previously treated chronic ITP. Subjects randomized 2:1, eltrombopag to placebo, and will be stratified based upon splenectomy status, use of ITP medication at baseline and baseline platelet count less than or equal to 15,000/µL.

Pts w/ platelet counts between 50,000/uL and 400,000/uL during the 6 month treatment period

Duration of count elevation; frequency of AEs; need for rescue treatment or ITP med; ITP symptoms; safety & tolerability

Start Nov 2006; end Jul 2008

Promacta ITP GSK II 99 Randomized, double blind trial to assess efficacy, safety, tolerability in adults with refractory, chronic ITP. Entry platelet counts <30,000/microL for 6 mos who have failed at least one treatment; also included pts receiving chronic maintenance steroids.

Pts achieving platelet counts =50,000/microL after 42 days of treatment

Safety & tolerability; PK; PD; ITP symptoms, QOL

Start Apr 2005; complete

Source: ClinicalTrials.gov

Key Trials: Promacta (Eltrombopag) in ITP

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Secondary Endpoints

Timing

Promacta HCV GSK III 750 Randomized, placebo controlled study in pts w/ chronic HCV to assess ability of eltrombopag to maintain a platelet count sufficient to facilitate initiation of antiviral therapy (Peg IFN alfa-2a+ribavirin), to minimize antiviral therapy dose reductions and to avoid permanent discontinuation of antiviral therapy. The clinical benefit of eltrombopag will be measured by the proportion of subjects who are able to achieve a Sustained Virological Response (SVR). Entry platelet count of <75,000/microL.

SVR rate defined as percentage of subjects with non-detectable HCV-RNA at 24 weeks post-completion of the planned treatment period (i.e., Week 48 for genotype 2/3 or Week 72 for non-genotype 2/3)

Pts with a shift in platelet count from <75,000/µL to >/=90,000/µL; AEs, laboratory abnormalities, ocular examinations, 12-lead ECGs

Start Oct 2007; end Aug 2011

Promacta HCV GSK III 500 Randomized, double blind trial to assess platelet elevation to reduce need for platelet transfusions in chronic liver disease patients with thrombocytopenia undergoing elective invasive procedures; includes patients with HCV, HBV, HIV, non-alcoholic steatohepatitis, NASH. Patients with model of end stage liver disease score (MELD) of 24 or less; entry platelet count <50,000/microL

Need for platelet transfusion prior to, during and up to seven days following elective invasive procedures

Bleeding; AEs, lab abnormalities, ocular exams, ECGmb

Start May 2008


Key Trials: Promacta (Eltrombopag) in HCV-related Thrombocytopenia

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Secondary Endpoints

Timing

Promacta HCV GSK II 422 Randomized, double blind dose ranging study (30, 50, 75 QD for 12 weeks) in chronic HCV related thrombocytopenia who are potential candidates for antiviral therapy.

Pts shifting from baseline counts to 100,000/microL after 4 weeks

Mean increase in platelet counts; safety & tolerability; population PK; PD; effect of antiviral outcome measures

Start Feb 2005; complete

Promacta CIT GSK II 183 Randomized, double blind dose ranging study to assess efficacy, safety, PK in patients receiving multiple cycles of chemo. Included pts with advanced solid tumors scheduled to receive 1st line carboplatin/paclitaxel; subjects had no history of platelet or bleeding disorders.

Change in baseline platelet count from 1st day of 2nd cycle to lowest count observed in the cycle

Safety & tolerability; PD; platelet count change; dose intensity of carbo/paclitaxel

Start Apr 2005; end Feb 2007


Key Trials: Promacta (Eltrombopag) in HCV and Chemo-related Thrombocytopenia

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Secondary Endpoints

Timing

Nplate ITP Amgen II 100 Open label study in refractory ITP patients. Entry platelet count <=20,000/microL; failed at least 3 conventional treatments; on anti-coagulant medication if pt has AF history.

AEs; clinically significant changes in lab values and incidence of antibody formation

Platelet response; transfusions

Start Feb 2005; end Dec 2010

Nplate ITP Amgen III 210 3b, randomized, SOC-controlled, open-label, 52-week treatment study to compare AMG 531 to medical SOC for ITP, with a 6-month Safety Follow-up. Non-splenectomized subjects who are 18 years or older, are diagnosed with ITP according to the American Society of Hematology (ASH) guidelines, and who have received at least 1 prior therapy for ITP will be eligible to screen for this study. Eligible subjects will be randomized to AMG 531 or medical SOC for ITP if their platelet count is < 50,000 or their platelet count falls to < 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy. After the completion or discontinuation of the study treatment period, any subject who does not transfer in to another AMG 531 study will complete a 6-month Safety Follow-up period.

Number of pts undergoing splenectomy; number of treatment failures

Time to splenectomy; platelet response; change in ITP patient reported outcomes

Start Nov 2006; end Jan 2010


Key Trials: Nplate (Romiplostim) in ITP

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Secondary Endpoints

Timing

Nplate MDS Amgen II 240 Randomized, double blind, placebo controlled study evaluating efficacy and safety of treatment of thrombocytopenia in pts w/ low or intermediate-1 risk MDS. Starting dose of 750mcg up to 1000mcg or reduced to min of 250 mcg.

Efficacy Number of platelet transfusions, bleeding events; platelet hematological improvement; overall survival; disease progression to AML; neutralizing antibodies versus drug; all AEs

Start May 2008; end Jan 2011

Nplate MDS Amgen N/A 200 Open label extension study to evaluate safety of long term dosing in MDS. Patients with IPSS low or intermediate-1 risk MDS.

AEs Platelet response; transfusions; bleeding events

Start Jun 2007; end Sep 2010


Key Trials: Nplate (Romiplostim) in MDS

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Secondary Endpoints

Timing

XXXXX ITP XXXXX II 106 Open label, single arm study will evaluate the efficacy and safety of rituximab monotherapy in patients with refractory, relapsing or chronic idiopathic thrombocytopenic purpura (ITP). Patients will receive infusions of 1000mg i.v. on days 1 and 15. The anticipated time on study treatment is 3-12 months.

Overall response rate

Time to CR and PR; duration of response and time to new therapy; AEs; hematological toxicity; infections; infusion reactions

Start May 2007; end Nov 2009

XXXXX ITP XXXXX II 65 Double-blind, randomized, placebo-controlled, dose-ranging, parallel-group study will assess the efficacy, safety and tolerability. Once daily oral doses of 2.5, 5, 10, 20 mg or placebo for 28 days. Patients are refractory or relapsed after at least one prior therapy; entry platelet counts <50,000/mm3.

Platelet response PK; PK/PD relationship

XXXXX ITP XXXXX II 24 Randomized, double blind placebo controlled study in ITP with open label extension. Patients dosed 7.5 mg/day for 6 weeks. Includes patients with platelet counts <50,000/microL who have been treated with at least 1 ITP therapy.

Platelet counts Start Mar 2008; end May 2009

XXXXX ITP XXXXX II 66 Phase I/II, non-randomized, open label dose comparison study. Includes adults with chronic IPT that have failed at least one therapy. Entry platelet counts <30,000/microL and platelet levels <150,000/microL for >6 mos. Drug dosed twice, two weeks apart.

Start Nov 2007


Key Trials: Nplate (Romiplostim) in MDSKey Trials: Other Phase II Compounds

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• More than 200,000 patients in the US experience thrombocytopenia.• Drug-induced thrombocytopenia (heparin, chemo or other drugs) are most common.• Thrombocytopenia resulting from compromised liver function (chronic HCV infection and cirrhosis) is also a

significant contributor to incidence.• Estimates of ITP incidence span a large range (XXXX-XXXX/year), but condition is still considered rare.

– Small population allows for orphan designation and fast track or priority review– Unmet need qualifies for fast tract or priority review

Populations are small to moderate

Competition is poised to intensify• Treatment aims to address the underlying cause of thrombocytopenia (e.g., withdrawal of myelosuppressive therapy;

antiviral therapy, liver transplant etc.).

• In ITP, most treatments are immunomodulators targeting production or interaction of autoantibodies targeting platelets (e.g., IVIG, anti-D, rituximab).

• New TPO mimetics are poised to enter the market in 20XX. – Amgen and GSK will specifically address thrombopoiesis with new oral and injectable therapies which have reported high

(>75%) response rates in restoring platelets to safe levels.

– Both will significantly change competitive landscape with investments in advertising, promotion and sales to create share of voice.

Conclusions

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• Only 11 compounds have entered the clinic and 8 appear to be in preclinical development. • Several are focused on the TPO receptor, while others are immunomodulators or other MOAs.

– TPO agents hold the most promise at this time, with Amgen and GSK in the lead.

Clinical pipeline is limited

• Most competitors have focused on ITP as their lead indication for registration, due to potential for fast track or priority review.

– Phase II and III studies require 100-200 patients

• Companies have also fielded several studies in patients with chronic HCV infection and liver disease and Amgen has a large trial in MDS.

– Phase III studies with HCV patients are larger (~500-750 patients)

• Amgen, GSK and others are looking at chemotherapy induced thrombocytopenia, but investigation appears to be in phase II to date.

• Based on additional safety studies being fielded, long-term use and safety are significant concerns. Specific studies are evaluating ocular effects.

Clinical trials focus on ITP, but also HCV

Conclusions

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New agents must be effective, with relatively low toxicity

Emerging Profile based on Clinical Pipeline

MOA • Target TPO receptor or autoantibody activity

Indication(s) • ITP (lead)• Thrombocytopenia related to chronic HCV or liver disease• Chemotherapy-induced thrombocytopenia (CIT)

ROA and Dosing • Oral or subcutaneous dosing• QD

Efficacy • 75-90% response, measured by achievement of platelet counts ≥50,000/µL

• Duration of response ≥15 weeks• Reduced need for platelet infusions

Safety • Well tolerated; similar to placebo• Mild headaches and fatigue• Rare grade 3 or 4 toxicities (e.g., bleeding / thrombosis)• Ocular effects TBD

Emerging Profile

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• Armstrong GL, et al, “Prevalence of Hepatitis C Virus Infection in the United States, 1999-2002,” Ann Intern Med, 2006• Beardsley DS, “ITP in the 21st Century,” Hematology, 2006• CDC, HIV/AIDS Surveillance Report, 2005• Chong-Shan W, et al, “Strong Association of Hepatitis C Virus Infection and Thrombocytopenia,” Clinical Infectious

Diseases, 2004• Cines DB, et al, “Congenital and Acquired Thrombocytopenia, Hematology, 2004• Frederiksen H, Schmidt K, “Incidence of Idiopathic Thrombocytopenic Purpura in Adults Increases with Age,” Blood,

1999• Decision Resources, “Managing the Side Effects of Chemotherapy,” 2007• George JN, et al, “ITP: A Practice Guideline Developed by Explicit Methods for the American Society of Hematology,”

Blood, 1996 • Hagop K, et al, “Incidence and Impact of Thrombocytopenia in Myelodysplastic Syndromes,” Cancer, 2007• Ma X, et al, “Myelodysplastic Syndromes: Incidence and Survival in the US,” Cancer, 2007 • Moore R, www.hopkins-hivguide.org• Ohman EM, et al, “Identification, Diagnosis and Treatment of HIT and Thrombosis (CATCH Registry),” J Thromb

Thrombolysis, 2005• Panzer S, “New Therapeutic Options for Adult Chronic ITP, Vox Sanguinis, 2008

Key References

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Dan Meichenbaum415.828.5265

[email protected]

Will Anlyan347.334.2562

[email protected]

www.dectiva.com

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