Targeted Cancer Therapies

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Targeted Cancer Therapies
ASCO Presentation, June 6th, 2016
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Trademarks:
The trademarks included herein are the property of the owners thereof and are used for reference purposes only. Such use should
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2
ProNAi Therapeutics
A drug development
company advancing
targeted cancer therapies
• We are an ambitious oncology drug
development company oriented
towards registration and
commercialization.
NASDAQ: DNAI
• We have a world-class management
team with a proven track record in
oncology drug development.
Headquarters: Vancouver, BC
Development: San Francisco, CA
Research: Plymouth, MI
IPO: July 2015
Shares: 30.2M outstanding
34.3M fully diluted
Cash on hand (31/3/16): $140.9M
• We intend to build a broad and
diverse pipeline of promising
oncology assets against emerging
targets on the leading edge of cancer
biology.
• We have a healthy cash balance, that
we expect will allow us to achieve
meaningful development milestones.
3
Proven Leadership in Oncology Development
Nick Glover, PhD
President and CEO
Barbara Klencke, MD
Chief Development Officer
Angie You, PhD
Chief Business & Strategy Officer;
Head of Commercial
Sukhi Jagpal, CA, CBV, MBA
Chief Financial Officer
Keith Anderson, PhD
Senior Vice President, Technical Operations
Wendy Chapman
Senior Vice President of Clinical Operations
Diane Gardiner
Senior Vice President of Human Resources
Chris Hassig, PhD
Senior Vice President of Research
Chandra Lovejoy
Senior Vice President of Global Regulatory Affairs
and Head of Quality
Emma McCann
Senior Vice President of Program Management
Gregg Smith, PhD, MBA
Senior Vice President of Preclinical
4
PNT2258 Program Update
PNT2258: DNAi Drug Candidate
PNT2258
• Proprietary formulation of multiple
copies of PNT100 encapsulated within
a lipid nanoparticle, delivered
systemically via infusion.
PNT100 DNAi
• A single stranded 24-base DNAi
oligonucleotide rationally designed to
bind to a specific regulatory region
associated with the BCL2 oncogene.
6
PNT2258: Development Program - Status
PNT2258-01
Solid
Tumors
22 subjects
Initiated
Sep. 2010
PNT2258-02
r/r NHL
13 subjects
Continuation/Follow Up
Initiated
Jan. 2013
PNT2258-03 Wolverine
r/r DLBCL
45/61 subjects
Initiated
Jan. 2015
Interim
-02 Data
(ASH)
ProNAi Updated
Appoints -02 Data
Dr. Glover (ASH)
Crossover ProNAi
$60M
Appoints
Raised
Dr. You
2010 2012
2013
2014
PNT2258-04 Brighton
Richter’s 5/50 subjects
Initiated
ProNAi
Appoints Oct. 2015
Dr. Klencke
IPO
$158M
Raised
2015
Interim -03
Data
2016
7
Final Results of PNT2258-02:
A Pilot Phase 2 Study of PNT2258 for r/r NHL
PNT2258-02: Pilot Phase 2 Trial in r/r NHL
Primary Objective
• To characterize pilot antitumor activity and collect safety data on subjects with recurrent
or treatment refractory (r/r) NHL receiving single-agent PNT2258.
Population
• 13 subjects with metabolically active recurrent or treatment refractory B-cell NHL were
enrolled from three centers.
• All subjects were required to have received prior therapy with rituximab and cytotoxic
therapy.
Dosing Regimen
• Administered PNT2258 as a single agent IV, Days 1-5, every 21-days for 6 to 8 induction
cycles or until progression.
• Subjects with ongoing benefit were allowed to receive continuation therapy at a
modified dose and schedule until progression.
Efficacy Evaluation
• Antitumor activity according to the Cheson criteria (2007).
• FDG-PET and CT imaging evaluation were conducted at baseline, at the end of Cycle 2,
and after 6 to 8 cycles.
9
PNT2258-02: Demographic and Baseline Characteristics
Disease Status
(Relapsed or
ECOG PS Refractory)*
No. Prior Lines
of Therapy
Best Response on
Last Prior Therapy
Time to Treatment
Failure with Last Prior
Therapy (months)
Refractory
2
SD
9.4
2
Relapsed
2
PR
21.6
65
1
Refractory
4
SD
5.5
MCL
65
0
Relapsed
4
PR
23.7
9
3
7
4
FL
FL
FL
FL
54
55
73
56
1
1
1
1
Refractory
Relapsed
Relapsed
Relapsed
5
1
2
2
SD
CR
PR
CR
5.1
21.2
35.2
48
1
FL
71
0
Relapsed
1
CR
57.9
13
11
12
10
DLBCL-B
DLBCL-RT
DLBCL
DLBCL
61
78
80
40
1
1
1
1
Relapsed
Relapsed
Relapsed
Refractory
1
2
1
1
CR
PR
CR
PD
19.9
6
87.6
2.3
Subject
No.
Diagnosis
Age
(years)
5
CLL
51
0
8
CLL/SLL
65
2
MCL
6
Legend
B = Burkitt’s-like
CLL = chronic lymphocytic leukemia
DLBCL = diffuse large B-cell lymphoma
FL = follicular lymphoma
MCL = mantle cell lymphoma
RT = Richter’s Transformation
Legend
ECOG PS = Eastern Cooperative
Oncology Group Performance
Status
Legend
CR = complete response
PR = partial response
SD = stable disease
PD = progressive disease
* Disease status classified per internal clinical review of baseline data.
10
PNT2258-02: Final Progression-Free Survival
8
2
PD
7
SD
FL
SD
4
CR
1
PR
13
PR
11
PR
12
10
On treatment
Subject off treatment but in follow-up without progression
1.3 Months
SD
3
3 Years
PFS duration
2.4 Months
PD
SD
2 Years
1.2 Months
SD
9
DLBCL
Subject Number
6
MCL
5
CLL/SLL
1 Year
14.2 Months
4.0 Months
Subject is still on treatment or active follow-up without progression
Disease progression
8.9 Months
32.8 Months*
27.4 Months
16.7 Months**
9.1 Months
9.5 Months
CR
26.0 Months
CR
30.3 Months
0
CLL = chronic lymphocytic leukemia
DLBCL = diffuse large B-cell lymphoma
FL = follicular lymphoma
12
Months
MCL = mantle cell lymphoma
SLL = small lymphocytic leukemia
24
36
Note: Response per investigator assessment (Cheson 2007).
* Subject remains on PNT2258 therapy.
** An additional 17.8 months of follow-up without progression
has been censored pending data entry
11
PNT2258-02: Outcome with Last Therapy vs. PNT2258
Best Response with Last Prior Therapy and Time Interval from Start of Last Prior Therapy
2
6
CLL/SLL
8
PD
SD
SD
PR
SD
PR
SD
SD
FL
CR
CR
PR *
CR
1
CR
13
12
SD
PR
4
11
SD
CR
3
PR
PR
DLBCL
Subject Number
9
7
PD
SD
MCL
5
Time to Progression and Best Response with PNT2258
PR
CR
CR
CR
PD
10
96
-96
84
-84
72
-72
60
-60
48
-48
36
-36
24
-24
12
-12
0
12
24
36
48
Months
* An additional 17.8 months of follow-up without progression has been censored pending data entry.
12
PNT2258-02: Final Efficacy Summary
PNT2258-02
(N = 13)
n (%)
Best response (Investigator assessment; Cheson 2007)
CR
3 (23.1)
PR
3 (23.1)
SD
5 (38.5)
PD
2 (15.4)
Overall response rate (best response ≥ PR)
(n [%], 95% CI)
6 (46.2%)
(19.2, 74.9)
Disease control rate (best response is ≥ SD)
(n [%], 95% CI)
11 (84.6%)
(54.6, 98.1)
Duration of response
Median months (min, max)
95% CI
23.4 m (3.0, 26.1*)
13, NE
* Indicates censored value.
13
PNT2258-02: Overview of Safety
• 12 of the 13 subjects have discontinued PNT2258 therapy.
• Median treatment duration on PNT2258 was 8.9 months (range: 1 to 33+ months).
• No subjects discontinued treatment due to AEs.
• One subject required a dose reduction.
• The majority of AEs were Grade 1 or 2 in severity.
• 85% of subjects experienced Grade 3 or 4 AEs.
• No Grade 5 AEs were observed.
• Most common AEs were nausea, chills, diarrhea, fatigue, headache, and vomiting.
• 31% of subjects experienced an SAE.
• 8% of subjects experienced a treatment-related SAE.
14
PNT2258-02: Conclusions
PNT2258 as a single-agent resulted in clinically significant, durable responses in recurrent
or treatment refractory NHL, including in four subjects with DLBCL.
• Overall response rate of 46.2% in the entire study population of 13 subjects.
• Noteworthy responses (CR, PR) and durable SD was also observed in follicular
lymphoma subjects.
Six subjects were progression free at 12 months; PFS extended to 2 years and beyond in
four subjects.
• Five of the six subjects who were progression free at 12 months with PNT2258 were
relapsed (not refractory) at study entry and had responded with their last prior therapy
with a median time to treatment failure (TTF) of 48 months (4 yrs), range 23.7-87.6
months.
• All four responding DLBCL subjects had received 1 or 2 prior therapies.
PNT2258 was safe and well tolerated in these NHL subjects and was administered for
extended periods without evidence of cumulative toxicity for 2 years and longer.
15
A Phase 2 Study of PNT2258 in Patients with r/r DLBCL:
An Initial Report from the Wolverine Study
PNT2258-03: Wolverine Phase 2 Trial in r/r DLBCL
Primary Objective
• Overall response rate (ORR).
Secondary Objectives
• DCR, DOR, TTR, OS, PFS.
• Safety.
• Identify predictors of outcome.
Population
• Previously treated subjects with metabolically active (r/r) DLBCL, defined as refractory to
prior therapy or relapsed after prior therapy.
• Prior exposure to CD20-targeted therapy, an alkylating agent, and a steroid.
Dosing Regimen
• Administered PNT2258 as a single agent IV, Days 1-5, every 21-days for up to 8 induction
cycles.
• Subjects with ongoing benefit were allowed to receive continuation therapy at a
modified dose and schedule until progression.
Efficacy Evaluation
• FDG-PET/CT imaging evaluations (Lugano) conducted at baseline and after Cycles 3, 5, 8.
17
PNT2258-03: Interim Study Populations
Safety Population*
N = 37
Subjects who received at
least 1 dose of PNT2258
Eligible Population*
n = 27
ECOG PS 0-1 and 1-3 lines
of prior therapy
Additional Subjects*
n = 10
ECOG PS 2 and/or
≥ 4 prior therapies
Response Evaluable
Population*
n = 19
At least 8 doses of
PNT2258 within 35 days
of starting therapy
Excluded from Response
Evaluable Population Due
to Early Discontinuation*
n=8
• Analysis performed on all subjects enrolled as of February 29th, and included all available
data as of April 25th
• This is an ongoing study with an open and active database, with outstanding, unresolved
queries.
* Interim.
18
PNT2258-03: Biomarker Assessments
Wolverine is designed to explore the
correlation between various patient
baseline characteristics and response rate.
Performance of the Lymph2Cx Assay as Previously Published
IHC:
• BCL2, c-MYC, Ki-67, BCL6.
FISH:
• BCL2, c-MYC, Ki-67, BCL6.
Cell of Origin (COO) in DLBCL
• Utilizes the Lymph2Cx assay (NanoString),
a digital 20 gene-expression based test for
COO assignment in formalin-fixed
paraffin-embedded tissue.
Blood. 2014;123(8):1214-17
19
PNT2258-03: Demographic and Baseline Characteristics
Clinical Characteristics
Age (median years [min, max])
ECOG PS
0
1
2
Patient Outcome with Prior Therapy
Number of prior lines (median [min, max])
PNT2258-03
(N = 37)
n (%)
64 (26, 84)
6 (16.2)
25 (67.6)
6 (16.2)
3 (2, 7)
Refractory to most recent prior therapy
19 (48.6)
Best response to last prior therapy
CR + PR
SD
PD
Other
15 (40.5)
2 (5.4)
17 (45.9)
3 (8.1)
Time to Treatment Failure on last prior therapy (median months [min, max])
Biomarker Results
FISH+ for MYC and BCL2 and/or BCL6 (Double hit)
3.9 (1.1, 71.5)
IHC+ for MYC and BCL2 (Double expressor)
16/29 (55.2%)
2/22 (9.1%)
20
PNT2258-03: Interim Efficacy
PNT2258-03
(N = 37)
n (%)
Subjects with
PS 0-1 and
≤ 3 Prior Therapies
(n = 27)
n (%)
Response
Evaluable Subjects
(n = 19)
N (%)
3 (8.1)
4 (10.8)
11 (29.7)
19 (51.4)
2 (5.4)
17 (45.9)
3 (8.1)
(1.7, 21.9)
7 (18.9)
(8.0, 35.2)
3 (11.1)
4 (14.8)
8 (29.6)
12 (44.4)
1 (3.7)
11 (40.7)
3 (11.1)
(2.4, 29.2)
7 (25.9)
(11.1, 46.3)
3 (15.8)
4 (21.1)
7 (36.8)
5 (26.3)
0
5 (26.3)
3 (15.8)
(3.4, 39.6)
7 (36.8)
(16.3, 61.6)
1.5 (0.07, 8.6)
0.03** - 5.3
1.9 (1.3, 2.4)
1.5 (0.07, 8.6)
0.03** - 5.3
2.0 (1.7, 3.6)
1.5 (0.85, 8.6)
0.03** - 5.3
2.0 (1.9, 3.6)
Best Response
≥ Partial Metabolic Response (PMR)
No Metabolic Response (NMR)
Progressive Metabolic Disease (PMD*)
N/E
Still on treatment
Discontinued treatment
Overall Response Rate
(n [%], 95% CI)
Disease Control Rate
(n [%], 95% CI)
Durability
Treatment Duration (median months [min, max])
DOR (range months)
PFS (median months, 95% CI)
*
**
One additional PMR incorrectly remained in the database at the time of the 25 April 2016 data cut-off date. This was subsequently corrected to PMD in the database
and thus, is displayed above as PMD as best response.
Indicates censored value.
21
PNT2258-03: Interim Data Summary for Responders
Characteristics of Responders:
• 3/3 male.
• 3/3 ECOG PS of 1.
• Median age 63 years (range: 63-68).
• 2/3 had received 2 prior lines of therapy.
• 1/3 had received 3 prior lines of therapy.
• 1/3 had a partial response to their last prior therapy.
• 2/3 progressed as their best response to prior therapy.
• One subject remains on therapy.
Biomarker Results:
• Cell of origin:
• 1 ABC, 1 GCB, 1 unknown.
• 1/3 MYC(+) by ICH, 0/3 MYC(+) by FISH.
22
PNT2258-03: Overview of Safety
• Median treatment duration on PNT2258 was 47 days (range: 2-263).
• Median treatment duration was 15 days in the 10 subjects with ECOG PS of 2 and/or
≥ 4 prior lines of therapy.
• 33 subjects (89%) have discontinued PNT2258 therapy.
• All 37 subjects experienced one or more AE.
• 65% of subjects experienced one or more Grade 3 or 4 AEs.
• Most common AEs were fatigue, nausea, pyrexia, anemia, diarrhea,
hypotension, and thrombocytopenia.
• 43% of subjects experienced an SAE.
• 35% of subjects experienced a treatment-related SAE.
• 22% of subjects died within 30 days of their last dose of PNT2258.
• 50% of subjects with ECOG PS of 2 and/or ≥ 4 prior lines of therapy died within 30
days of their last dose of PNT2258.
23
PNT2258-03: Conclusions - Interim Efficacy
PNT2258 showed modest single-agent activity in r/r DLBCL patients.
Response Evaluable
(n= 19)(%)
Safety Population
(N=37)(%)
Overall response rate
(n% [95% CI])
3 (15.8)
(3.4, 39.6)
3 (8.1)
(1.7, 21.9)
Disease control rate
(n% [95% CI])
7 (36.8)
(16.3, 61.6)
7 (18.9)
(8.0, 35.2)
• All three responses were observed in subjects with PS 0-1 and ≤ 3 prior lines
of therapy.
• Currently, 45 subjects have been enrolled.
• No additional responses seen to date in eight subjects enrolled after data cutoff.
• 39 subjects have discontinued treatment.
• Six subjects remain on PNT2258.
• one with an ongoing response after 10 cycles of therapy.
• five yet to be evaluated (on study for <9 weeks).
24
PNT2258-03: Conclusions - Interim Safety
Overall, the safety profile of PNT2258 appears acceptable in subjects with PS 0-1
and ≤ 3 prior lines of therapy.
Subjects with PS 0-1 and ≤ 3
prior lines of therapy (n = 27)
Subjects with PS 2 and/or ≥ 4
prior lines of therapy (n = 10)
47 days
15.5 days
SAE rate
37%
60%
Grade 5 AE rate
11%
50%
Median duration of
therapy on study
• We determined that the most advanced subjects, with PS 2 and/or having
received ≥ 4 lines of therapy, did not respond to therapy, and experienced
higher rates of serious adverse events and Grade 5 events.
25
The Brighton Phase 2 Trial:
PNT2258-04 Richter’s Transformation
PNT2258-04: Brighton Phase 2 Trial in Richter’s
The Brighton Phase 2 Trial
PNT2258-04 Richter’s Transformation [NCT02378038]
• A multicenter, single-arm, open-label study of PNT2258.
• PNT2258 administered as a 4-hour IV infusion on Days 1-5 of a 21-day cycle.
• Adults with Richter’s transformation.
Efficacy Evaluation
• FDG-PET/CT imaging evaluations conducted at baseline and after Cycles 3, 5, and 8.
• Antitumor activity interpreted according to the revised 2014 IWG criteria for lymphoma
(Lugano classification).
Primary Objective
• Overall response rate (ORR).
Secondary Objectives
• DCR, DOR, TTR, OS, PFS.
• Safety.
• Identify predictors of outcome.
27
PNT2258-04: Current Study Status
Five subjects enrolled to date.
• No responses have been observed to date.
Four subjects have discontinued study treatment.
• One subject received one dose and died on Study Day 2 (intracranial hemorrhage).
• One subject received one cycle and discontinued therapy on Day 24 for progressive
disease and died on Day 38.
• Two subjects discontinued for progressive disease after receiving three cycles of
PNT2258.
One subject continues on study treatment.
• Subject has completed two cycles of therapy.
28
PNT2258: Next Steps
PNT2258: Overarching Conclusions
• We have designed and run an appropriate, well-executed study, and have
sought to optimize the conduct of Wolverine in real-time in order to focus on
the most appropriate patient populations for treatment with PNT2258.
• Advanced DLBCL and Richter’s Transformation are challenging diseases to treat,
and PNT2258 did not markedly improve outcomes in these indications.
• Modest efficacy has been observed in Wolverine, with mostly limited duration
of response. No responses have been observed in Brighton.
• Clinical data in Wolverine are not trending to an outcome that supports the
likelihood of registration as monotherapy in late line DLBCL.
• In the Brighton study, although only a few patients have been enrolled to date in
this ultra-orphan indication clinical trial, we have also noted generally poor
outcomes in this advanced patient population, with limited signs of activity.
• In context of the interim Wolverine data, our emerging view is that Brighton is
unlikely to support registration of PNT2258.
30
Next Steps for PNT2258 and DNAi Programs
• On the basis of these interim assessments, we have decided to close the
Wolverine and Brighton studies to further enrollment of new subjects. On
behalf of ProNAi, we would like to thank the patients and their families,
investigators and staff involved in these studies for their participation and
support.
• Considering these data in context of the broader development of PNT2258, we
have decided to suspend all development of PNT2258 pending further review of
data in order to determine next steps for the asset.
• Similarly, we have halted all further investment in our DNAi research program at
this time given our emerging view that this research is unlikely to yield
additional ProNAi development candidates.
• We continue to maintain a strong balance sheet that we will manage prudently
as we focus our resources and activities on the advancement of PNT141 and
future assets. We plan to secure additional assets towards building a broad and
diverse pipeline of oncology assets under development.
31
PNT141: Targeting Cdc7
ProNAi Therapeutics: Focused on Building a Pipeline of
Promising Oncology Assets
• We are an ambitious oncology drug
development company oriented
towards registration and
commercialization.
• We have a world-class management
team with a proven track record in
oncology drug development.
• We intend to build a broad and
diverse pipeline of promising
oncology assets against emerging
targets on the leading edge of cancer
biology.
• We have a healthy cash balance, that
we expect will allow us to achieve
meaningful development milestones.
On May 26, 2016, we announced our
first transaction: AS141 (PNT141), a
Cdc7 inhibitor licensed from Carna
Biosciences.
• Initial upfront payment of
$0.9 million.
• Aggregate additional potential
payments upon achievement of
certain developmental, regulatory
and commercial milestones of up
to $270 million.
• ProNAi will also pay Carna
single-digit tiered royalties on the
net sales of any product
successfully developed.
33
PNT141: Selective Small Molecule Targeting Cdc7
PNT141 (formerly AS-141)
• A highly-selective and potent Cdc7 inhibitor, with favorable development
characteristics.
• Cdc7 is a key regulator of both DNA replication and DNA damage response, making it a
compelling emerging target for the potential treatment of a broad range of tumors.
• Broad development scope in solid and liquid tumors; mono- and combo- therapy.
• PNT141’s selectivity profile offers possible differentiation and potential safety and
efficacy advantages.
• First-in-class and/or best-in-class opportunity.
Attractive Asset and Deal Terms
• Licensor: Carna Biosciences, Japan: expertise in kinase screening and chemistry drug
discovery (first validating deal with J&J in 2015).
• Attractive cost of entry, and ongoing deal and development costs typical for a smallmolecule oncology drug.
Intellectual Property
• PNT141 covered by composition of matter (2032 plus extensions) and combination
patents.
34
PNT141: Cdc7 Plays an Essential Role in Two Vitally
Important ‘Hallmarks of Cancer’
Cdc7 is a serine/threonine kinase with essential roles in both DNA replication
initiation [proliferation] and subsequent S-phase cell cycle checkpoint control
[genomic instability].
• Responsible for the “firing”
of replication origins leading
to initiation of DNA synthesis,
and is the final critical step
in growth regulatory control.
• Phosphorylates and activates
(i) the helicase complex
MCM2-7 (leading to the
unwinding of dsDNA during
the G1/S transition), (ii)
claspin, a Chk1 activator, and
(iii) RAD18 to mediate TLS.
Cellular
Energetics
Replicative
Immortality
Invasion /
Metastasis
Proliferation
Evading Growth
Inhibition
Tumor
Inflammation
Immune
Evasion
HALLMARKS
OF CANCER
Angiogenesis
Genome
Instability
Cell Death
Resistance
35
PNT141: Tumor Cells are Predisposed to Cell Death in
Response to Cdc7 Inhibition
Normal cells activate a p53-dependent cell cycle arrest in response to transient Cdc7
inhibition, whereas tumor cells undergo apoptosis via checkpoint failure – opportunity
for a favorable therapeutic window.
Cdc7 Inhibitor
Adapted from Carna Biosciences website.
36
PNT141: Clinical Development Opportunities and
Patient Selection Strategy
• Preclinical data and published literature suggest a variety of indications with
potential for response to Cdc7 inhibitors.
• Solid tumors – breast, ovarian, pancreatic, melanoma, colorectal, uterine, thyroid, etc.
• Hematological malignancies – AML, DLBCL, etc.
• A patient selection, biomarker-driven strategy focusing on drivers of replication
stress, genomic instability and proliferation (e.g. p53, BRCA, MYC, KRAS, H2AX
expression) will help facilitate clinical trial execution.
• Potential to exploit tumor-specific genetic defects and/or combination therapies
to effect cancer cell death via ‘synthetic lethality’.
• ProNAi plans to pursue a robust program of preclinical studies to further
evaluate tumor responses across a variety of indications and dosing regimens to
inform clinical development plans and patient selection strategies.
37
PNT141: Summary
• Cdc7 is a key regulator of both DNA replication and DNA damage response,
making it a compelling emerging target for the potential treatment of a broad
range of tumor types.
• PNT141’s promising pharmaceutical properties, combined with target utility, IP
runway (to 2032 plus extensions) and patient selection strategies, afford broad
development scope in solid and liquid tumors as both mono- and combination
therapy, with opportunity for first and/or best-in-class positioning.
• Deal metrics are highly competitive, with an attractive upfront, and ongoing
licensing and development costs typical for a small-molecule oncology drug.
• Importantly, there exists opportunity for ProNAi to add significant value to the
program, via its world-class preclinical and clinical development team.
• We anticipate filing an IND for PNT141 in H2 2017, with clinical studies
expected to begin by the end of 2017.
38
ProNAi Therapeutics: Focused on Building a Pipeline of
Promising Oncology Assets
• We intend to build a broad and diverse pipeline of promising oncology assets.
• PNT141 is our first transaction.
• We are actively engaged in ongoing business development activities, with two
additional promising assets under exclusive option to enable further validating
studies under MTA.
• These assets are consistent with our strategic in-licensing focus, namely:
• Orally bioavailable small molecules and next generation biologicals with exemplary
target selectivity.
• Assets with a foundation of strong, cancer-critical biology.
• Assets against emerging targets, where there is an opportunity to be first-in-class or
fast follower/best-in-class.
• Assets with potential as monotherapy and in combinations, across a variety of cancer
indications.
• Assets that could possibly form proprietary combinations with each other.
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Targeted Cancer Therapies
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