Second Quarter 2021 Conference Call - August 16, 2021
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Second Quarter 2021 Conference Call
August 16, 2021
1
On Today’s Call
Agenda Topic Speakers
Welcome Olivia Bloom, EVP and Chief Financial Officer
Introductory Remarks John A. Scarlett, M.D., Chairman and Chief Executive Officer
Second Quarter 2021 Financials Olivia Bloom, EVP and Chief Financial Officer
Phase 3 Clinical Trials Updates Aleksandra Rizo, M.D., Ph.D., EVP and Chief Medical Officer
Imetelstat Market Opportunity in Anil Kapur, EVP Corporate Strategy and Chief Commercial Officer
Lower Risk MDS
Closing Remarks John A. Scarlett, M.D., Chairman and Chief Executive Officer
Q&A All
2
Forward Looking Statements
Except for the historical information contained herein, this presentation contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities
Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) that Geron expects IMerge Phase 3 to be fully enrolled in the
fourth quarter of 2021; (ii) that Geron expects top-line results for IMerge Phase 3 to be available in the first quarter of 2023; (iii) that Geron expects to conduct an interim analysis for
IMpactMF in 2024 and a final analysis in 2025; (iv) that Geron expects its financial resources, with the expected non-dilutive funding under the current debt facility, to fund operations
through the end of the first quarter of 2023; (v) that Geron expects to grow to 80-85 employees in 2021; (vi) that Geron plans to engage over 180 sites for IMpactMF; (vii) that IMerge
Phase 3 and IMpactMF have registrational intent; (viii) that imetelstat has the potential to demonstrate disease-modifying activity in patients and to target the malignant stem and
progenitor cells of the underlying disease; (ix) that the Company expects imetelstat to be a highly differentiated product in the lower risk MDS commercial marketplace; (x) that the
Company expects that the shortened 12-month follow-up period will still provide a sufficiently mature data set to demonstrate safety and efficacy, including transfusion independence,
of imetelstat to support any application for regulatory approval; (xi) that Geron expects the peak revenue in the United States and the five largest European markets for imetelstat in
lower risk MDS to be $1.2 billion; and (xii) other statements that are not historical facts, constitute forward-looking statements. These forward-looking statements involve risks and
uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and
uncertainties related to: (a) whether the current or evolving effects of the COVID-19 pandemic and resulting global economic and financial disruptions will materially and adversely
impact Geron’s business and business prospects, its financial condition and the future of imetelstat; (b) whether Geron overcomes all of the potential delays and other adverse impacts
caused by the current or evolving effects of the COVID-19 pandemic, and overcomes all the enrollment, clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing
and regulatory challenges in order to have the financial resources for, and to meet the expected timelines and planned milestones in (i) through (vi) above; (c) whether regulatory
authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (d) whether imetelstat is demonstrated to be safe and efficacious in
IMerge Phase 3 and IMpactMF to enable regulatory approval; (e) whether any future efficacy or safety results may cause the benefit-risk profile of imetelstat to become unacceptable;
(f) whether imetelstat actually demonstrates disease-modifying activity in patients and the ability to target the malignant stem and progenitor cells of the underlying disease; (g) that
Geron may seek to raise substantial capital in order to complete the development and commercialization of imetelstat, including to meet all of the expected timelines and planned
milestones in (i) through (vi) above; (h) whether regulatory authorities require an additional clinical trial for approval even if IMerge Phase 3 or IMpactMF meet their respective primary
endpoints; (i) whether there are failures or delays in manufacturing or supplying sufficient quantities of imetelstat or other clinical trial materials in a timely manner; (j) whether
imetelstat is able to maintain patent protection and have freedom to operate; (k) whether the shortened follow-up period of 12 months for the IMerge Phase 3 primary analysis results
in not obtaining adequate data to demonstrate safety and efficacy, including transfusion independence, in the primary analysis; (l) whether Geron can accurately project the timing of,
or attain complete enrollment in IMerge Phase 3 or IMpactMF, whether due to the current or evolving effects of the COVID-19 pandemic or otherwise; (m) whether Geron is able to
enroll IMerge Phase 3 and IMpactMF at a pace that would enable the financial resources for, and to meet the expected timelines and planned milestones in (i) through (vi) above; and
(n) that Geron may be unable to successfully commercialize imetelstat to achieve the peak revenue in (xi) above due to competitive products, or otherwise. Additional information on
the above risks and uncertainties and additional risks, uncertainties and factors that could cause actual results to differ materially from those in the forward-looking statements are
contained in Geron’s filings and periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors” and elsewhere in such filings and reports, including
Geron’s quarterly report on Form 10-Q for the quarter ended June 30, 2021 and future filings and reports by Geron. Undue reliance should not be placed on forward-looking statements,
which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any
obligation to update these forward-looking statements to reflect future information, events or circumstances.
3Introductory Remarks
John A. Scarlett, M.D., Chairman and Chief Executive Officer
4Key Takeaways
• Achieved 91% enrollment in IMerge Phase 3
• Expect to complete enrollment in 4th Quarter this year
Significant
Progress • Accelerated timing of Top-Line Results by 3 months
Since Last • Now expect Top-Line Results in 1st Quarter 2023
Quarter
• Current and projected financial resources expected to
be sufficient to Top-Line Results
5Q2 2021 Financials
Olivia Bloom, EVP and Chief Financial Officer
6Phase 3 Clinical Trials Updates
Aleksandra Rizo, M.D., Ph.D., EVP and Chief Medical Officer
7Imetelstat Market Opportunity in Lower Risk MDS
Anil Kapur, EVP Corporate Strategy and Chief Commercial Officer
8Lower Risk Myelodysplastic Syndromes (LR MDS)
LR MDS OPPORTUNITY TYPICAL LR MDS PATIENT
>100,000 patients living with LR MDS (US/EU5) NEEDING TREATMENT
~25,000 patients diagnosed annually (US/EU5)
Median age @ diagnosis ~70 years
~70% of MDS patients are LR MDS
Hemoglobin ~9 g/dL
Healthy: Men (13-18), Women (12-16)
KEY DISEASE CHARACTERISTICS Transfusion burden 2-5 RBC units/8 weeks
• Ineffective hematopoiesis in the bone marrow Typically transfused 1-3 units every 4 weeks
• Chronic anemia leading to transfusion dependency
• Presence or absence of deletion 5q Baseline EPO level Less than 500 mU/mL
Normal adult population: 3-19 mU/mL
• Patient sub-types: Ring Sideroblasts (RS+/RS-),
EPO levels, transfusion burden 5-year survival ~32%
Lower Risk MDS: IPSS-R: Very low, low, intermediate; WPSS: Very low, low, intermediate; EPO: erythropoietin; AML: acute myeloid leukemia; References on slide 18
Patients experience frequent transfusions, infections, higher risk of
progression to AML, poor quality of life and shortened survival 9Expected Broad Imetelstat Opportunity in LR MDS
Lower Risk MDS (~70%) ~30% High Risk MDS
Symptomatic Anemia
del(5q) Non-del(5q)
(~10% ) (~90% )
ESA Eligible ESA Ineligible
Serum EPO ≤500 mU/mL Serum EPO >500 mU/mL
(~90% ) (~10% )
Lenalidomide ESAs
ESA Failed and ESA Ineligible
Ring Sideroblast Positive Ring Sideroblast Negative (RS-)
(RS+) (~25%) (~75%)
Luspatercept NO APPROVED THERAPY
ESA: erythropoiesis stimulating agent;
REBLOZYL – tradename for
BMS/Acceleron’s drug luspatercept;
Expected Broad Imetelstat Opportunity
REVLIMID – tradename for BMS’s
drug lenalidomide
References on slide 18 10Excitement about Imetelstat
IMerge Phase 2 data and expected profile at launch received favorably by
practicing hematologists in both US and key European markets
Broad Efficacy Across Patient Subtypes
(RS+ and RS-)
Key aspects of
imetelstat that Durability of Transfusion Independence
resonated most
strongly with
hematologists in both
the community and Novel MOA, Non-Overlapping with Current Treatments
academic settings
Potential for Disease Modification
Geron Physician Market Research (US/EU5); stimuli included IMerge Phase 2 LR MDS data and expected target
product profile at launch; MOA: mechanism of action
11Market Opportunity for Imetelstat in LR MDS
USD $1.2 billion in potential peak revenue across US & key European markets
Key Addressable Patient Segments in LR MDS for Imetelstat
22,000 ESA Relapsed/Refractory, RS- Patients
7,000 ESA Relapsed/Refractory, RS+ Patients
3,700 ESA Ineligible Patients
TBD Luspatercept Experienced Patients Imetelstat expected
to become part of the
standard of care
in LR MDS
Company projections: based on treated prevalence estimates for imetelstat eligible patient populations in LR MDS; DRG syndicated data and Geron
analysis using assumptions for a) expected target product profile at launch, b) obtaining regulatory approvals and favorable reimbursement in US
and key European markets, c) duration of treatment and d) potential market penetration
12Stage-Gated Commercial Enterprise Build
Integrated U.S.
Commercial Organization
Established
U.S. Commercial
Team Expansion
EU Execute US
Commercialization “go-to-market”
Strategy finalized strategy
Establish Commercial Leadership
(Access, Medical Affairs, Marketing and Sales)
Build HR & IT, CMC and Supply & Distribution infrastructure to support launch
2020 2021 2022 2023-2024
TLR LR MDS Imetelstat LR MDS
Investment in Launch Preparations LR MDS NDA and MAA Launch
(Q1 2023*) Filings US (1H 2024*)
(2023*) EU (2H 2024*)
* Expected timing based on current planning assumptions, subject to ongoing COVID pandemic
and other risks; assumes priority review in the US and EU TLR: top-line results; NDA: new drug application; MAA: marketing authorization application
13Closing Remarks
John A. Scarlett, M.D., Chairman and Chief Executive Officer
14Virtual Investor Day – November 2021
Stay tuned for further details in October
Imetelstat’s potential for disease modification in
lower risk MDS and refractory MF
Join us and key Expected path to commercializing imetelstat
opinion leaders
for these
Expansion of imetelstat development plans, including
discussions new studies in additional indications
Geron’s early discovery program in second generation
telomerase inhibitors
15Q&A Session
16Thank you for joining us
References
Slide 9
• Incidence and Outcomes for Low Risk Myelodysplastic Syndrome; ASH 2012; Abstract 4944
• Revised International Prognostic Scoring System for Myelodysplastic Syndromes; Greenberg, et al, Blood 2012 Sep 20
• MDS Landscape and Forecast; DRG Clarivate; Nov 2020
• NCCN Guidelines v3, 2021
• Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents; Fenaux, et al; JCO
2017 May 10
• Long-term outcome of anemic lower-risk myelodysplastic syndromes without 5q deletion refractory to or relapsing after erythropoiesis-stimulating agents;
Kalaidi, et al; Leukemia 2013, 27
• Treatment of MDS; Platzbecker; Blood 2019 Mar 7
• UCSF Health Center and Mayo Clinic
Slide 10
• Revised International Prognostic Scoring System for Myelodysplastic Syndromes; Greenberg, et al, Blood 2012 Sep 20
• Clinical and biological implications of driver mutations in myelodysplastic syndromes; Malcovati et al; Blood 2013 Nov 21
• NCCN Guidelines v3, 2021
• Treatment of MDS; Platzbecker; Blood 2019 Mar 7
• MDS Landscape and Forecast; DRG Clarivate; Nov 2020
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