Aprea Therapeutics, Inc. · NASDAQ:APRE
Aprea Therapeutics — J.P. Morgan 38th Annual Healthcare Conference 2020
20 slides from Aprea Therapeutics's conference presentation, Jan 13, 2020
Slide 1: J.P. Morgan 38th Annual Healthcare Conference
© 2020 Aprea Therapeutics, Inc. January 2020 J.P. Morgan 38 th Annual Healthcare Conference
Slide 2: Forward-Looking Statements
2 © 2020 Aprea Therapeutics, Inc. Forward - Looking Statements Certain information contained in this press release includes “forward - looking statements”, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended,
Slide 3: Aprea Therapeutics: The p53 Reactivator Company
Aprea Therapeutics (NASDAQ: APRE) Focused on p53-targeted anticancer therapy Headquarters in Boston, MA with research facilities in Stockholm, Sweden Gross proceeds of ~$98mm in October 2019 IPO Use of proceeds to expand and accelerate clinical development 5th best performing Biotech IPO in 20191 Si
Slide 4: p53: Guardian of the Genome
4 © 2020 Aprea Therapeutics, Inc. p53: Guardian of the Genome □ p53 is a critical tumor suppressor protein ▪ Normal function senses DNA damage and induces o Apoptosis, cell - cycle arrest, senescence □ The gene encoding p53 is the most commonly mutated gene in cancer ▪ Mutated in over half of all li
Slide 5: Our Approach to Reactivating p53
5 © 2020 Aprea Therapeutics, Inc. Our Approach to Reactivating p53 □ APR - 246 is converted to MQ, which covalently binds mutant p53 to restore wild - type conformation, resulting in cell cycle arrest and apoptosis Sources: Aprea unpublished data; Furukawa et al, Cancer Sci, 2018 p53 M237I tumors Gr
Slide 6: Clinical Development
6 © 2020 Aprea Therapeutics, Inc. Clinical Development
Slide 7: Current Pipeline
7 © 2020 Aprea Therapeutics, Inc. Current Pipeline 1 Investigator - initiated trial; 2 With or without azacitidine Molecule Indication Treatment Line Preclinical Phase 1 Phase 2 Phase 3 APR - 246 TP53 Mutant MDS Frontline TP53 Mutant MDS / AML Frontline (U.S. study) 1 Frontline (French study) 1 Post
Slide 8: TP53 Mutant MDS and AML are Diseases of Older Adults with Limited Treatment Options
8 © 2020 Aprea Therapeutics, Inc. TP53 Mutant MDS and AML are Diseases of Older Adults and Have Limited Treatment Options and Poor Outcomes Sources: 1 Decision Resources Group, 2019; 2 Lindsley et al, New England J Med, 2017, 536; 3 Dohner et al, Leukemia, 2018, 2546; Sallman et al, ASH poster #1817
Slide 9: Phase 1b/2 Trials in TP53 Mutant MDS/AML: Overview
9 © 2020 Aprea Therapeutics, Inc. Phase 1b/2 Trials in TP53 Mutant MDS/AML: Overview □ Two parallel investigator - initiated trials evaluating frontline APR - 246 + azacitidine (AZA) ▪ All patients confirmed as TP53 mutant by next - generation sequencing ▪ Patients receive 4 days APR - 246 followed
Slide 10: Data Summary of + AZA in TP53 Mutant MDS Patients
10 © 2020 Aprea Therapeutics, Inc. Data Summary of APR - 246 + AZA in TP53 Mutant MDS Patients □ Highly concordant responses observed across U.S. and French Trials □ 52% of evaluable MDS patients in U.S. trial discontinued study treatment for transplant □ APR - 246 + AZA combination regimen is well
Slide 11: Response and Duration in Evaluable Patients - U.S. Trial
11 © 2020 Aprea Therapeutics, Inc. Response and Duration in Evaluable Patients - U.S. Trial 1 (N=45) ASH 2019 Update 1 Sallman et al, ASH 2019 Median duration of follow - up = 10.8 months Overall MDS AML MDS - MPN + CMML Evaluable patients, n 45 33 8 4 Overall response rate, n (%) 39 (87) 29 (88) 7
Slide 12: Responses Observed Across p53 Mutation Types and Locations
12 © 2020 Aprea Therapeutics, Inc. Responses Observed Across p53 Mutation Types and Locations U.S. Trial 1 French Trial 2 ASH 2019 Update 1 Sallman et al, ASH 2019; 2 Cluzeau et al, ASH 2019
Slide 13: Response and Duration Compared to Published Data for AZA Alone
13 © 2020 Aprea Therapeutics, Inc. Response and Duration of APR - 246/AZA Compared to Published Data for AZA Alone TP53 Mutant MDS 1 Sallman et al, ASH poster #1817, 2018; Bally et al. Leuk Res. 2014; Takahashi et al. Oncotarget. 2016; Kulasekararaj et al, B r J Haematol, 2013; Bejar et al, Blood, 2
Slide 14: Randomized Phase 3 MDS Trial Topline Data Expected Q3 2020
14 © 2020 Aprea Therapeutics, Inc. Randomized Phase 3 MDS Trial 1 Topline Data Expected Q3 2020 1 ClinicalTrials.gov NCT03745716; 2 As of January 10, 2020 Randomized study of frontline AZA ± APR - 246 in TP53 mutant MDS □ Status ▪ 102/154 patients currently enrolled with 9 in screening 2 ▪ Full enro
Slide 15: Current Pipeline Update and Overview
15 © 2020 Aprea Therapeutics, Inc. Current Pipeline Update and Overview □ Phase 2 Post - Transplant Trial ▪ Only a minority of patients proceed to stem cell transplant (SCT) due to lack of sufficient response to initial therapies, advanced age, comorbidities and lack of a suitable donor ▪ APR - 246
Slide 16: Future Pipeline Plans
16 © 2020 Aprea Therapeutics, Inc. Future Pipeline Plans □ Hematological Malignancies ▪ Phase 1 in relapsed/refractory TP53 mutant non - Hodgkins lymphomas (CLL, MCL) o APR - 246 + venetoclax and APR - 246 + ibrutinib o Study evaluating safety and preliminary efficacy o Anticipate first patient enro
Slide 17: APR-548: Oral Next-Generation p53 Reactivator
17 © 2020 Aprea Therapeutics, Inc. □ Similar mechanism of action to APR - 246 (MQ) □ More potent than APR - 246 in vitro ▪ ≥ 40% reduction in IC 50 APR - 548: Oral Next - Generation p53 Reactivator □ IND enabling studies in progress ▪ No inhibition/induction of key metabolic enzymes, no hERG signal
Slide 18: Financial Overview
18 © 2020 Aprea Therapeutics, Inc. Financial Overview » ~$130.1 million of cash and cash equivalents (unaudited) at December 31, 2019 » No outstanding debt » Anticipated cash burn for 2020: $35.0 million – $40.0 million » Existing cash should fund operations into 2023 Balance Sheet Shares Outstandin
Slide 19: 2020 Anticipated Milestones
19 © 2020 Aprea Therapeutics, Inc. 2020 Anticipated Milestones Milestones Timeline Phase 3 Frontline MDS CR primary endpoint readout 3Q 2020 NDA Submission 4Q 2020 Phase 1/2 Frontline MDS/AML (French Trial) CR rate, duration, survival 1H 2020 Phase 2 MDS / AML post - transplant maintenance Full enro
Slide 20: Summary
20 © 2020 Aprea Therapeutics, Inc. Summary □ Continued strong execution of current APR - 246 clinical development plan ▪ Positive and concordant efficacy data in Phase 1b/2 trials for MDS/AML ▪ Randomized Phase 3 trial for MDS on track for full enrollment 1Q 2020 and primary CR endpoint readout 3Q 2
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