Bio-Path Holdings, Inc. · OTC:BPTH
Bio-Path — Investor Presentation (December 2016)
22 slides from Bio-Path's investor presentation, Dec 16, 2016
Slide 1: A new path in DNA-powered medicine
“ A new path in DNA - powered medicine ” Company Presentation December 2016 1
Slide 2: Forward Looking Statements
Forward Looking Statements This Presentation contains forward - looking statements with respect to business conducted by Bio - Path Holdings, Inc . By their nature, forward - looking statements and forecasts involve risks and uncertainties because they relate to events and depend on circumstances th
Slide 3: DNAbilize Technology
DNAbilize™ Technology x Enables the development and delivery of systemic antisense DNA treatments for a broad spectrum of cancers including hematological malignancies, solid tumors, as well as diseases outside of cancer » has the ability to address hard to treat diseases and unmet clinical needs in
Slide 4: Investment Highlights
Investment Highlights • Lead candidate, Prexigebersen, (BP1001), a Liposomal Grb2 Antisense in Phase II for acute myeloid leukemia and chronic myeloid leukemia • Second drug candidate being readied for IND to start Phase I • Demonstrated ability to deliver antisense DNA into target cells and down -
Slide 5: DNAbilize Antisense DNA: A Targeted Method for Treating Disease
» Antisense - molecules that interfere with the process of producing proteins inside cells (RNAi) » Does not use a toxic agent to kill cells, but blocks production of proteins » Advantage of specificity because it targets the disease - causing protein » No toxicity - In numerous animal studies and h
Slide 6: DNAbilize Technology proprietary antisense and delivery platform
DNAbilize ™ Technology is a proprietary antisense and delivery platform that solves the antisense industry dilemma P - ethoxy backbone Neutral liposomes DNAbilize™ SAFE : No toxicity from the DNA modification or the lipid delivery, no AEs observed to date SYSTEMIC : Allows for systemic distribution
Slide 7: Clinical Pipeline
*Prexigebersen (BP1001) has received orphan drug designation from the U.S. FDA for AML and CML and from the European Medicines Agency (EMA) for AML Clinical Pipeline Target Indications Preclinical IND Phase I Phase II Prexigebersen (BP1001) AML* Prexigebersen (BP1001) CML* Prexigebersen (BP1001) Sol
Slide 8: Continuing Improvement in Product Design and Manufacturing
Continuing Improvement in Product Design and Manufacturing 8 Manufacturing Development Product Design Advances CMC’s IND Lead Product Scale up from initial batch size 2 nd Generation Formulation Engineered Oligos Product Improvements Going Forward • Lyo process • Capacity Going Forward • Liposome pr
Slide 9: Manufacturing Improvements
Manufacturing Improvements 9 0% 20% 40% 60% 80% 100% 120% 140% 0 10 20 30 40 % Live Grb - 2, uM BV173 96 hr after dosage with Grb - 2 Lot 25, 29, 34, 35, 36 Series1 Series2 Series3 Series4 Series5 » Through our dose - escalating Phase 1 trial we upgraded our supplier base with the goal of increased
Slide 10: DNAbilize Lead Target and Indications: Grb2 and Myeloid Leukemia
DNAbilize ™ Lead Target and Indications: Grb2 and Myeloid Leukemia 10 Prexigebersen (BP1001) is an antisense DNA to the bridging protein Grb2 □ Ideal target for myeloid leukemia because it shuts down the Ras pathway in receptor activated myeloid cells without exerting adverse effects on Ras signalin
Slide 11: Summary of Phase I Monotherapy Clinical Trial Results for Prexigebersen
» AML, CML, & MDS Patients Refractory or Resistant to Current Therapies » Patients averaged 6 prior therapies » Dose escalating treatment cycle, 8 doses over 4 weeks, up to 90 mg/m 2 » Average reduction in circulating blasts was 67% in patient’s showing anti - leukemic response to drug » Drug was we
Slide 12: Prexigebersen Knocks Down Grb2 Protein in Target Cells
NS 1 = no sample collected 2 Fewer cells used in analysis because sample had less cells Grb2 levels decreased in 11 of 13 patient samples by end of treatment (EOT) pErk levels decreased in 7 of 13 patient samples by EOT Prexigebersen Knocks Down Grb2 Protein in Target Cells 12
Slide 13: Response to Treatment for Blast Crisis CML and Stable Disease in AML
• Patient 002 : 32 year - old, Hispanic male with myeloid blast crisis of CML • Prior therapies consisted of: ― Gleevec ― Dasatinib ― Nilotinib ― DCC - 2036 ― Cytarabine/Fludarabine/ ― Dasatinib/Gemtuzumab ― PHA - 739358 ― Clofarabine/Dasatinib • Patient taken off therapy, CNS disease was assumed (n
Slide 14: Phase II Prexigebersen + LDAC Combination Therapy Results
CR = Complete remission Safety Segment Phase II Prexigebersen + LDAC Combination Therapy Results Relapsed/refractory AML patients - 3 evaluable patients in each of 2 cohorts - Dose 60 mg/m 2 and 90 mg/m 2 respectively - Received drug 2x week for 4 weeks 5 of 6 patients responded: - 3 patients achiev
Slide 15: Ongoing Phase II Efficacy Trial Design for AML
interim analysis after 19 patients Ongoing Phase II Efficacy Trial Design for AML Prexigebersen Combination Therapy • Safety segment completed, demonstrated no negative synergies using prexigebersen together with frontline therapy (LDAC) – Efficacy trial will be conducted at 10 leading cancer center
Slide 16: Advances in the CML Program
Advances in the CML Program • Presentation at the 58th Annual American Society of Hematology Annual Meeting: – BP1001, a Novel Therapeutic for Chronic Myelogenous Leukemia • Prexigebersen decreased the proliferation of Gleevec ® ( imatinib ) - resistant CML cells in a dose - dependent manner • Prexi
Slide 17: Prexigebersen Treatment Paradigm
Prexigebersen Treatment Paradigm • Frontline therapy for de novo AML patients who are induction therapy ineligible and unfit for a stem cell transplant – Prexigebersen combination with LDAC – Current response rate frontline therapy alone less than 20% – Prexigebersen to improve response rates signif
Slide 18: Preclinical Pipeline
Preclinical Pipeline Validating DNAbilize™ with Key Opinion Leaders: • Establishing prexigebersen (BP1001) in triple negative and inflammatory breast cancer and advanced ovarian cancer with the MD Anderson Cancer Center • Developing clinical and preclinical targets for treatment of systemic lupus in
Slide 19: Achievements and Upcoming Milestones
Achievements and Upcoming Milestones Completed safety segment of the Phase II for AML Enrolled first patient in prexigebersen Phase II efficacy trial for AML Received orphan drug designation in the EU from the European Medicines Agency Expanded pre - clinical development with new target drug
Slide 20: Core Organization
Core Organization Peter Nielsen Co - Founder, President, Chief Executive Officer and Chief Financial Officer • Officer and Director since founding Company in 2007 • Manufacturing development and evolution of engineered product design Ulrich Mueller, PhD Chief Operating Officer • P reviously Vice Pre
Slide 21: Board of Directors
Jorge Cortes, M.D . Chairman • M.D. from la Facultad de Medicina , Universidad Nacional Autónoma de México • Jane and John Justin Distinguished Chair in Leukemia Research, Chief of the AML and CML sections, and Deputy Chair of the Department of Leukemia at The University of Texas MDACC • Has consult
Slide 22: IP and Financial Snapshot
Intellectual Property • Original patents licensed from MD Anderson • New composition and methods of use patents filed to cover DNAbilize technology, solely owned by Bio - Path Financial Snapshot • Ticker: NASDAQ: BPTH • Cash: $11.3 million as of September 30, 2016 • July 2016, $10 million registered
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