Rain Therapeutics Corporate Overview - September 2021 - Investor Relations | Rain Therapeutics Inc.
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September 2021
Rain Therapeutics
Corporate Over view
1 RAIN THERAPEUTICS INC.
This presentation contains “forward-looking statements” within the meaning of the federal securities laws, which statements are subject to substantial risks and uncertainties and are based on
estimates and assumptions. Other than statements of historical facts, all statements included in this presentation are forward-looking statements, including statements concerning the ability of our
clinical trials to demonstrate safety and efficacy of our product candidates and other positive results, the timing and focus of our ongoing and future preclinical studies and clinical trials and the
reporting of data from those studies and trials, our plans relating to commercializing our product candidates, if approved, including the geographic areas of focus and sales strategy, the size of the
market opportunity for our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting, our expectations regarding the approval and use of
our product candidates as first, second or subsequent lines of therapy or in combination with other drugs, the success of competing therapies that are or may become available, our estimates of the
number of patients that we will enroll in our clinical trials, the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates, the timing or likelihood of regulatory filings
and approvals, including our expectation to seek an accelerated approval pathway and special designations, such as orphan drug designation, for our product candidates for various diseases, our
ability to obtain and maintain regulatory approval of our product candidates, and our plans relating to the further development of our product candidates, including additional indications we may
pursue. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,”
“estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements involve known and unknown risks,
uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this presentation including our limited operating
history, the significant losses that we have incurred since inception, the substantial additional capital that we will require to finance our operations in the future, our ability to identify and acquire or in-
license products, our dependence on our lead candidates, our ability to enroll patients in our clinical trials and those risks described in “Risk Factors” and “Management's Discussion and Analysis of
Financial Condition and Results of Operations” in our Quarterly Report on Form 10-Q for the period ended June 30, 2021, filed with the Securities and Exchange Commission (SEC).
We cannot assure you that we will realize the results, benefits or developments that we expect or anticipate or, even if substantially realized, that they will result in the consequences or affect us or
our business in the way expected. Forward-looking statements are not historical facts, and reflect our current views with respect to future events. Given the significant uncertainties, you should
evaluate all forward-looking statements made in this presentation in the context of these risks and uncertainties and not place undue reliance on these forward-looking statements as predictions of
future events. All forward-looking statements in this presentation apply only as of the date made and are expressly qualified in their entirety by the cautionary statements included in this presentation.
We disclaim any intent to publicly update or revise any forward-looking statements to reflect subsequent events or circumstances, except as required by law.
We obtained the industry, market and competitive position data used throughout this presentation from our own internal estimates and research, as well as from industry and general publications,
and research, surveys and studies conducted by third parties. Internal estimates are derived from publicly available information released by industry analysts and third-party sources, our internal
research and our industry experience, and are based on assumptions made by us based on such data and our knowledge of the industry and market, which we believe to be reasonable. In addition,
while we believe the industry, market and competitive position data included in this presentation is reliable and based on reasonable assumptions, we have not independently verified any third-party
information, and all such data involve risks and uncertainties and are subject to change based on various factors. These and other factors could cause results to differ materially from those expressed
in the estimates made by the independent parties and by us.
This presentation concerns commercial products as well as discussion of investigational drugs that are under preclinical and/or clinical investigation and which have not yet been approved for
marketing by the U.S. Food and Drug Administration (FDA). They are currently limited by Federal law to investigational use, and no representations are made as to their safety or effectiveness for the
purposes for which they are being investigated.
2 RAIN THERAPEUTICS INC.
Rain Therapeutics: Overview
Rain's lead program, milademetan (RAIN-32), an oral MDM2 inhibitor, commenced a Phase 3 trial in 2H 2021
PR E C I S I O N O N C O LO GY M D M 2 I N H I B I TO R CASH
S T R AT E G Y (milademetan / R AIN-32) R U N W AY
• Phase 3 MANTRA trial commenced • Year-end 2021 cash guidance of
• Precision oncology, small molecule $137 – 147mm
strategy to evaluate milademetan in patients
with DD LPS
• Cash runway supports a multi-year
• Pipeline programs unified in targeting runway to support:
genetically-defined patient • “Pipeline in a Program”
▪ Phase 3 LPS registrational study
populations ▪ Phase 2 MDM2-amp basket trial
▪ Phase 2 Intimal Sarcoma trial
3 RAIN THERAPEUTICS INC.
Rain’s Corporate Leadership
Avanish Vellanki, Robert Doebele, Richard Bryce, Kolbot By, Vijaya Tirunagaru, Nelson Cabatuan,
MBS, MBA MD, PhD MBChB PhD PhD CPA
Cofounder, Cofounder, Executive VP & Senior VP, Senior VP, Senior VP,
Chairman and Chief President & Chief Chief Medical Officer Technical Operations Head of Research Finance &
Executive Officer Scientific Officer Administration
4 RAIN THERAPEUTICS INC.
Program Development Status
Rain commenced a Phase 3 study in WD/DD LPS and has more studies anticipated to start shortly, with clinical news flow
beginning in 2H 2022
COMMERCIAL PLANNED
PROGRAM INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 RIGHTS DATA
WD/DD Liposarcoma Enrolling 2023
Milademetan1 MDM2 Basket Study Planned: 2H 2021 2H 2022
MDM2 Inhibitor
Intimal Sarcoma Planned: By early 2022 Late-2022
IST: NCC (Tokyo, Japan)
RAD52
Research HRD+ Tumors Lead ID in
Program 2022
1Also known as RAIN-32 Rain anticipated clinical studies
See “Forward Looking Statements” in our SEC Filings for risks relating to timeline projections and other forward-looking statements.
5 RAIN THERAPEUTICS INC.
Milademetan
(Mila / RAIN-32)
RAIN THERAPEUTICS INC.
Milademetan (RAIN-32): Overview
Clinical stage, oral, small molecule MDM2 inhibitor
KEY RAIN PERSPECTIVES
Supportive Prior Clinical Data in Liposarcoma
Dosing Schedule Possibly Minimizing Toxicities
Potential to Translate to all MDM2 Dysregulated Cancers
7 RAIN THERAPEUTICS INC.
Milademetan: Clinical Trial Strategy
The clinical development plan is aimed at translating the unique potential therapeutic window of milademetan
into various MDM2-dependent indications in monotherapy settings
P L A N N E D C L I N I C A L I N D I C AT I O N S
Well-Diff / MDM2-Gene Intimal Merkel Cell GATA3- Mesothelioma
De-Diff Amplified Sarcoma Carcinoma Mutant ER+
Liposarcoma Pan-Tumor Breast
(LPS) Basket Cancer
Phase 3 Phase 2 Phase 2
2H 2021 Early 2022
Evaluating Evaluating Evaluating
8 RAIN THERAPEUTICS INC.
Milademetan Strategy: p53 WT Cancers
The milademetan strategy commences in MDM2-dependent tumors and may
progress to other p53 wildtype tumors in combination with other agents
p53 WT Cancer Opportunity MDM2-Dependent Cancers • Rain’s “1-2 Punch”
MDM2 Dependent, Cancer US Annual 1. MDM2-dependent
Incidence tumors
p53 WT2
2. Other wildtype p53
Possibly MDM2 MDM2 Dependent Cancers tumors
4%
8% Dependent, p53 WT3
WD / DD Liposarcoma 2,000 • At present, Rain
MDM2-amp (CN≥12)4 5,000
believes ~4% of p53
WT cancers exhibit
Other p53 WT Intimal Sarcoma 100 dependency on MDM2
p53 WT Annual 88%
Advanced Cancer - There may be more,
Merkel cell carcinoma 1,600 however, as Rain
Population (US):
continues to unravel
mGATA3 ER+ breast 11,000
~460,0001 add’l MDM2
Possibly MDM2 Dependent Cancers
dependencies
• In p53 WT tumors
CDKN2A-del cancers 37,000
without dependency on
Total 56,700 MDM2, combination
therapy likely the
1.
2.
Globocan 2020 Report, Rain estimates based on adv cancer annual treatable patients
Based on Rain estimates of WD/DD LPS, MDM2-amp solid tumors, intimal sarcoma,
preferred avenue
Merkel cell carcinoma and mGATA3 ER+ BC
3. Based on Rain estimates of CDKN2A-deleted solid tumors
9 4. Rain estimates a range of ~3-7.5k; using approximate mid-point
RAIN THERAPEUTICS INC.
Potential Mechanism: MDM2 – p53 Axis
Mechanisms of p53 loss RAIN-32 Mediated p53 Reactivation
Tumor Cell cycle arrest
Apoptosis
MDM2 binds
Milademetan
to p53
Degraded p53
MDM2 Milademetan
blocked
P53 MDM2 MIC1
transcription
targets
p53 Mutant p53
p53
p14 ARF
3 Mutated TP53 gene p53
2 CDKN2A p53 gene transcription
gene loss
MDM2 gene amp
1
MDM2 gene amp
Source: Rain Therapeutics
10 RAIN THERAPEUTICS INC.Evolutionary Convergence
Different alterations/pathways to generate the same final outcome
GENOTYPE PHENOTYPE
TP53 mutation
(loss of transcriptional activity)
MDM2 overexpression
- MDM2 amplification Loss of p53
-32
- ST antigen induced MDM2 expression function
- JAK2-mediated MDM2 expression
CDKN2A loss
(loss of p14ARF, negative
regulator of MDM2)
11 RAIN THERAPEUTICS INC.Liposarcoma Overview
TUMOR SCAN BIOLOGY & SUB-TYPES
• Liposarcomas are among the most common sarcomas in adults
(20% of all sarcomas)1
- Liposarcoma US annual incidence: ~3,0002
• WD/DD LPS is 65% of LPS
- p53 WT & MDM2 amplified in nearly 100% of WDLS/DDLS cases3-6
DDLS
APPROVED THERAPIES IN LPS
Efficacy in
WDLS Drug Company Approved Mechanism WD/DD LPS
(mPFS)
DNA binding /
2015 alkylator
2.2 mo7
Microtubule
2016 targeting
2.0 mo8
1 TM Mack. Cancer, 1995 Jan 1;75(1 Suppl):211-44. 7 Demetri et al., J Clin Oncol 34:786-793, 2015
2 Manji and Schwartz, J Oncol Pract, 2016 8 Demetri et al., J Clin Oncol 35:3433-3439. 2017
3 Pedeutour et al., Genes Chromosomes Cancer 24:30–41, 1999.
4 Italiano et al., Int. J. Cancer: 122, 2233–2241 (2008)
5 Kashima et al., Modern Pathology volume 25, pages 1384–1396(2012)
6 The Cancer Genome Atlas Research Network, Cell 171, 950–965, November 2, 2017
12 RAIN THERAPEUTICS INC.Phase 1 Trial Results: Safety
S E L E C T T R E AT M E N T E M E R G E N T A D V E R S E E V E N T S
Schedule A, B, and C
Schedule D Schedule D
Organ Class n (%) Cohorts
All doses (n=29)1 260mg (n=20)
(n=78) Dosing Schedule for 4
All Grades Grade ≥ 3 All Grades Grade ≥ 3 All Grades Grade ≥ 3 Cohorts
All drug-related TEAEs 74 (95) 43 (55) 25 (86) 5 (17) 18 (90) 4 (20) • Schd. A: QD 21/28
Blood and lymphatic • Schd. B: QD 28/28
disorders
Thrombocytopenia 52 (67) 27 (35) 13 (45) 4 (14) 9 (45) 3 (15) • Schd. C: QD 7/28
Anemia 33 (42) 14 (18) 5 (17) 0 4 (20) 0 • Schd. D: QD 3/14 x 2
Neutropenia 10 (13) 8 (10) 1 (3) 1 (3) 1 (5) 1 (5)
Gastrointestinal disorders
Nausea 57 (73) 2 (3) 20 (69) 0 16 (80) 0
Vomiting 22 (28) 2 (3) 13 (45) 1 (3) 10 (50) 1 (5)
Diarrhea 26 (33) 0 9 (31) 0 5 (25) 0
General disorders
Fatigue 36 (46) 3 (4) 12 (41) 0 8 (40) 0
Source: Gounder et al., 2020 EORTC-NCI-AACR (ENA) Conference. Interim data as of a July 2020 cutoff.
13 RAIN THERAPEUTICS INC.Phase 1 Trial Results: PFS
P RO G R E S S I O N - F R E E S U RV I VA L : S C H E D U L E D ( Q D 3 / 1 4 x 2 )
100
SCHEDULE D ≤ 260 mg 260 mg All Schd D
260 mg
90 (3/14 x 2) (n=17) (n=16) (n=18)
All QD 3/14 dosing
80 ≤260 mg
Median PFS, mo 8.0 7.4 7.4
(95% CI) (1.8, 28.9) (1.8, 14.6) (2.7, 28.9)
Progression-Free Survival (%)
70
60
50
40
30
20
Trabectedin Schd D (260mg)
10
Schd D (≤ 260mg)
0
0 3 6 9 12 15 18 21 24 27 30 33 36
Time (months)
Source: Gounder et al., 2020 EORTC-NCI-AACR (ENA) Conference. Interim data as of a July 2020 data cutoff.
The above table is for illustrative purposes only and is not a head-to-head comparison. Differences exist between trial designs and methodologies, and
caution should be exercised when comparing data across studies. The trabectedin data is from published data from third-party Phase 3 clinical trial.
14 RAIN THERAPEUTICS INC.Phase 1 Trial Results: Duration
L P S PAT I E N T T R E AT M E N T D U R AT I O N ( S c h e d u l e D O n l y )
• Vast majority of
Schedule D LPS
patients exhibited
treatment durations in
Schedule D (QD 3/14 x 2)
excess of trabectedin
• Three (3) patients
340 mg continued on therapy
on Schedule D as of
260 mg
July 2020
120 mg
Patient continuing therapy • Patient with longest
Milademetan U101 PFS duration on therapy
mPFS – 7.4 mo across all dose
Trabectedin Pivotal Trial schedules exceeded
mPFS – 2.2 mo 3 years as of July
2020
0 5 10 15 20 Months 25 30 35 40
Source: Gounder et al., 2020 EORTC-NCI-AACR (ENA) Conference. Interim data as of a cutoff of July 2020.
The above table is for illustrative purposes only and is not a head-to-head comparison. Differences exist between trial designs and methodologies, and
caution should be exercised when comparing data across studies. The trabectedin data is from published data from third-party Phase 3 clinical trial.
15 RAIN THERAPEUTICS INC.Comparative Hematologic Safety: Milademetan vs. Other MDM2 Programs
Treatment Related AEs, ≥ Gr 3
Thrombocytopenia Neutropenia Anemia
60% 60% 60%
50% 50%
50% 47% 50% 50%
42%
40% 40% 40% 37.5%
30% 30% 30%
24% 25%
23%
20% 20% 20%
15% 15%
12%
10% 10% 10% 8%
5%
NA 0%
0% 0% 0%
1 2 3 4 5
RAIN-32 HDM201 KRT-232 Idasanutlin APG-115 RAIN-32 HDM201 KRT-232 APG-115 Idasanutlin RAIN-32 KRT-232 HDM201 Idasanutlin APG-115
1. Gounder et al., ENA 2020; Dose/Schedule: 260mg QD 3/14 x 2; Tumor Type: Solid Tumors 4. Siu et al., ASCO 2014; Dose/Schedule: QD 100-800mg, BID 600mg 5/28; Tumor Type: Solid Tumors
2. Hyman et al., AACR 2017; Dose/Schedule: 15-200 mg, QD and intermittent; Tumor Type: Solid Tumors 5. Zhang et al., ASCO 2020; Dose/Schedule: QOD 150mg 21/28; Tumor Type: Solid Tumors
3. Wong et al., ASCO 2020; Dose/Schedule: 240 mg (QD 7/21, 5/28), 180 mg (5/28); Tumor Type: Merkel
Cell
The above tables are for illustrative purposes only and are not head-to-head comparison. Differences exist between trial designs and methodologies, and caution should be exercised when comparing data across studies.
16 RAIN THERAPEUTICS INC.Comparative Efficacy in WD/DD Liposarcoma
The Phase 1 study (n=107) derived an optimized dose schedule with improved safety
and efficacy in WD/DD LPS patients (n=53) vs. SOC and other MDM2 programs
mPFS of Approved Regimens and Developmental MDM2i’s in WD/DD LPS
• In n=53 patients, the
schedules with lowest
1
Eribulin N=31 2.0 observed mPFS was 6.3
SOC
mo (Schd A-B)
2
Trabectedin N=45 2.2 - ~3x that of the
standard of care,
KRT-232
3
N=17 2.0 3.9 Duration of Stable Disease (mo)
trabectedin
• Patients dosed under the
HDM201 4
MDM2 Inhibitors
HDM201
+ ribociclib
N=74 2.7 4.2 preferred dosing
schedule (260mg, 3/14 x
Mila (Schd A-B) 5 N=30 6.3 2) achieved an mPFS of
7.4 mo
Mila (Schd C-D) 5 N=23 7.4
Mila (Schd D, 260mg) 5 N=16 7.4 RP2D
- 1.0 2.0 3.0 4.0 5.0 6.0 7.0 8.0
1. Demetri et al., J Clin Oncol 35:3433-3439. 2017
2. Demetri et al., J Clin Oncol 34: 786- 793, 2016 mPFS (months)
3. Gluck et al., Invest New Drugs 2020, 240mg 7/21d: Tumor Type WD/DD LPS. Efficacy bars represent range of duration of SD for DD (2.0 months) or WD (3.9 months)
4. Razak et al.,AACR Annual Meeting 2018, multiple dose regimens (Poster) and combination with ribociclib
5. Gounder et al., 2020 EORTC-NCI-AACR (ENA) Conference
The above tables are for illustrative purposes only and are not head-to-head comparison. Differences exist between trial designs and methodologies, and caution should be exercised when
17 comparing data across studies. RAIN THERAPEUTICS INC.Beyond LPS: MDM2 Basket Trial Rationale
Case Study1,2 in Small Cell Lung Cancer from U101 Phase 1 Study
BASELINE PAT I E N T O U T C O M E S
• Patient with 3 prior
Patient ID 1005-1028
Tx’s (cis/VP-16,
Dose 340mg Ipi/Nivo, Temodar)
- Best response on
Schedule D (3/14 x 2) prior Tx was
progressive
Efficacy PR (-54%) disease (PD)
Duration 4 months • Patient did not
experience any Gr ≥3
Reason for Death (PE1) hematologic toxicities
C Y C L E 3 D AY 1
Discontinuation - Not progression
MDM2 Amp 36.6x
p53 Status WT
1 PE – Pulmonary Embolism
2 Scans: Gounder et al, 2018 CTOS conference
18 RAIN THERAPEUTICS INC.MDM2 Basket Trial: Mutual exclusivity of MDM2 CN and TP53 mutations
G E N I E / M S K I M PA C T F O R A L L C A N C E R S MDM2 AMP CONFERS POOR PROGNOSIS
Copy Number (CN) = 12
Total Number Median OS
cases deceased (Months)
100 MDM2 CNPatient Population: MDM2-amp
3,000
MDM2 CN ≥12 Incidence MDM2 CN≥12 & WT TP53
MDM2 CN ≥12 Adv incidence Adv Cancer • MDM2 CN of 12
Incidence
Incidence
2,500
Bladder Urothelial 2,578 309
based on mutual
Breast Inv 1,579 569 exclusivity with TP53
Lung Adeno 1,550 1,224 mutations in TCGA
2,000 Melanoma 464 60 and AACR-Genie
Esophageal 241 173
data
Sarcoma (No DD LPS) 236 80
Lung Sq 232 184 • Based on TCGA data
1,500
Testicular germ cell 202 61
Stomach Ad 190 118
analysis, patients with
Cholangio 147 147 MDM2 CN ≥ 12 range
1,000 Adrenocortical 4 4 between ~3,000 to
Total 7,423 2,929 7,500
500
0
Bladder Breast Inv Lung Adeno Melanoma Esophageal Sarcoma (No Lung Sq Testicular germ Stomach Ad Cholangio Adrenocortical
Urothelial DD LPS) cell
% CN ≥12 3.4 0.8 1.6 0.4 1.3 1.6 0.4 2.2 0.8 2.8 1.3
MDM2 CN frequency based on TCGA data from GDC data portal, downloaded on April 2020. https://portal.gdc.cancer.gov/
Incidence based on Globocan 2020 data; Advanced cancer based on NCI-SEER
20 RAIN THERAPEUTICS INC.Summary: Planned Trials for Mila in MDM2 Gene Amp Populations
TRIAL SCHEMAS E N D P O I N T S & P O P U L AT I O N
1
N ≈ 160
RAIN-32 • Primary / Secondary Endpoints: PFS, OS, ORR, DoR
Phase 3
N ≈ 80 260 mg, qd, 3/14 x 2
MANTRA
WD/DD • Patients with unresectable / metastatic WD/DD LPS
Randomize
Liposarcoma
Trabectedin • Disease progression within 6 months on systemic tx
N ≈ 80
1.5 mg/m2, IV over 24h, q3w
• Primary / Secondary Endpoints: ORR, PFS, OS,
Phase 2
MANTRA-2
BASKET: GMI, DoR
Pan Solid Tumors N ≈ 65 RAIN-32
MDM2 CN≥12 & 260 mg, qd, 3/14 x 2 • Population: Patients with relapsed/ refractory,
TP53 WT advanced or metastatic solid tumors
• Primary / Secondary Endpoints: ORR, PFS, OS,
Phase 2
GMI, DoR
Intimal N ≈ 30 RAIN-32
Sarcoma 260 mg, qd, 3/14 x 2 • Target Population: Patients with relapsed/ refractory,
advanced or metastatic intimal sarcoma
- No line of therapy restriction; No biomarker selection
1 Total patients randomized
21 RAIN THERAPEUTICS INC.MDM2 Competitive Landscape
SELECTED CLINICAL MDM2 INHIBITORS & ONGOING STUDIES
Indications
Company Drug Form.
LPS ST SCLC NSCLC SGC Merk AML MF PV Lymph Other
Milademetan Oral P3 P2 P2
(Basket) Intimal Sarcoma
P1b STS (IST)
KRT-232 / P1 MM (IST)
Oral P2 P2 P1b/2 P3 P2 P1b/2 P1 GB (IST)
AMG-232 P1 AML (x2; IST)
ALRN-6924 IV P1b P1b/2 P1 Ped (IST)
(IST)
Siremadin
Oral P2 P1b P1/2 P1 Uveal Melanoma
(HDM201) (IST) P1 CRC (IST)
APG-115 Oral P1b/2 P1b/2 P1b P2 T-PLL
(+Mel) P1 AML/MDS
BI 907828 Oral P1a/b
(x2)
ASTX295 Oral P1/2
Source: clinicaltrials.gov, as of Sept 2021
AML – Acute Myeloid Leukemia; CRC –Colorectal carcinoma; GB – Glioblastoma; LPS – Liposarcoma; Lymph – Lymphoma; MDS – Myelodysplastic Syndromes; Mel – Melanoma; Merk – Merkel Cell
Carcinoma; MF – Myelofibrosis; MM – Multiple Myeloma; MPNST – Malignant Peripheral Nerve Sheath Tumor; Ped – Pediatric Cancer; PLL – Prolymphocytic Leukemia; PV – Polycythemia Vera; SGC –
Salivary Gland Carcinoma; SCLC – Small Cell Lung Cancer; ST – Solid Tumors; and STS – Soft Tissue Sarcoma
Planned registrational trial
22 RAIN THERAPEUTICS INC.RAD52
Research Program
RAIN THERAPEUTICS INC.RAD52 Research Program: Overview
Synthetic Lethal Strategy Targeting RAD52 in DNA Damage Response (DDR) Pathway
KEY RAIN PERSPECTIVES
Few Strategies in DDR Space
Synthetically Lethal in HRD+ Tumors
No Clinical Competitors in RAD52
24 RAIN THERAPEUTICS INC.RAD52 Strategy
DNA Double Strand Break Repair
• RAD52 pathway likely to
be critical in BRCA
deficient tumors upon
relapse to PARP
RAD52 present in
therapy
pathways unique from
BRCA1/2 and PARP • No clinical competitors
in RAD52 space
Toma et al., Cancers (Basel). 2019 Oct 14;11(10).
25 RAIN THERAPEUTICS INC.RAD52 Synthetic Lethality and Potency Observed in Preclinical Models
Preliminary compounds, including RT-001, already exhibits selectivity in BRCA-deficient tumors vs. BRCA WT
RAD52i SELECTIVITY FOR MDA-MB-436, BRCA1(-/-) MDA-MB-436, BRCA1(+/+)
BRCA1-DEFICIENCY
140 BRCA1+ 35 Control
Talazoparib, 0.33 mg/ kg 35 Control
Tumor volume (fold increase)
BRCA1-
Tumor volume (fold increase)
RT-001,5050mg/kg
D-I03, mg/kg Talazoparib ++D-I03
Talazoparib RT-001
120 30
Talazoparib
Talazoparib++D-I03
RT-001 30
Clonal survival, %
100 25 25
80 20 20
60 15 15
40 10 10
20 5 5
0
0 0
0.0 2.5 5.0 7.5 10.0 0 1 2 3 4 5
0 1 2 3 4 5
D-I03,
RT-001,uM
mM Weeks
Weeks
Huang et al., Nucleic Acids Res. 2016 May 19;44(9):4189-99
26 RAIN THERAPEUTICS INC.Summary: Rain’s Event Calendar
Rain anticipates a steady stream of clinical data beginning in 2H 2022
O P E R AT I O N A L E X E C U T I O N
Event Program Timeline
Complete FDA / EMA Meetings Milademetan 1Q 2021
Complete Rain IPO Corporate 2Q 2021
Enroll First Patient: Phase 3 LPS Milademetan 2H 2021
Enroll First Patient: Phase 2 MDM2-amp Basket Milademetan 2H 2021 On track
Enroll First Patient: Phase 2 Intimal Sarcoma Milademetan By early-2022 On track
Select Development Candidate RAD52 2022 On track
C L I N I C A L D ATA T I M E L I N E S
Event Program Timeline
Interim Data: Phase 2 MDM2-amp Basket Milademetan 2H 2022 No Change
Interim Data: Phase 2 Intimal Sarcoma Milademetan Late-2022 No Change
Complete Data: Phase 3 LPS Milademetan 2023 No Change
27 RAIN THERAPEUTICS INC.Appendix
RAIN THERAPEUTICS INC.Rain’s Corporate Governance
Board of Directors
Avanish Vellanki, MBA Franklin Berger, CFA Peter Radovich, MBA
Chairman & CEO, Rain FMB Research COO, Mirum
Therapeutics
Aaron Davis, MBA Tran Nguyen, MBA
CEO, Boxer Capital CFO & COO, Prothena
Gorjan Hrustanovic, PhD Stefani Wolff
Managing Director, BVF LP COO & EVP of
Product Development, Nurix
Scientific Advisory Board
Robert Doebele, MD, PhD Lori Kunkel, MD Simon Powell, MD, PhD
President & CSO, Rain Fmr CMO, Loxo & Memorial Sloan Kettering
Therapeutics Pharmacyclics
Trever Bivona, MD, PhD Chris Kirk, PhD
Professor in Residence, UCSF President & CSO, Kezar
29 RAIN THERAPEUTICS INC.You can also read
