Jefferies Virtual Global Healthcare Conference - November 2020
←
→
Page content transcription
If your browser does not render page correctly, please read the page content below
Jefferies Virtual
Global Healthcare Conference
November 2020
Safe Harbor Statement
In addition to historical information, this presentation contains forward-looking statements under the Private Securities Litigation Reform Act that involve
substantial risks and uncertainties. Such forward-looking statements within this presentation include, without limitation, statements regarding our drug
candidate SM-88 and its clinical potential and non-toxic safety profiles, our drug development plans and strategies, ongoing and planned clinical trials,
preliminary data results and the therapeutic design and mechanisms of our drug candidates; and readers can identify forward-looking statements by
sentences or passages involving the use of terms such “believes,” “expects,” “hopes,” “may,” “will,” “plan,” “intends,” “estimates,” “could,” “should,” “would,”
“continue,” “seeks,” or “anticipates,” and similar words (including their use in the negative) or by discussions of future matters such as the development and
potential commercialization of our lead drug candidate and of other new products, expected releases of interim or final data from our clinical trials, possible
collaborations, the timing, scope and objectives of our ongoing and planned clinical trials and other statements that are not historical. The forward-looking
statements contained in this presentation are based on management’s current expectations, which are subject to uncertainty, risks and changes in
circumstances that are difficult to predict and many of which are outside of TYME’s control. These statements involve known and unknown risks, uncertainties
and other factors which may cause the Company’s actual results, performance or achievements to be materially different from any historical results and future
results, performances or achievements expressed or implied by the forward-looking statements. These risks and uncertainties include, but are not limited to,
that the information is of a preliminary nature and may be subject to change; uncertainties inherent in research and development, including the ability to
achieve clinical study start and completion dates; the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of
existing data; risks associated with early, initial data, including the risk that the final Phase II data may differ from prior study data or preliminary Phase II data;
final results of additional clinical trials that may be different from the preliminary data analysis and may not support further clinical development; that past
reported data are not necessarily predictive of future patient or clinical data outcomes; whether and when any applications or other submissions for SM-88
may be filed with regulatory authorities; whether and when regulatory authorities may approve any applications or submissions; decisions by regulatory
authorities regarding labeling and other matters that could affect commercial availability of SM-88; the ability of TYME and its collaborators to develop and
realize collaborative synergies; competitive developments; and the factors described in the section captioned “Risk Factors” of TYME’s Annual Report on
Form 10-K filed with the U.S. Securities and Exchange Commission on May 22, 2020, as well as subsequent reports we file from time to time with the U.S.
Securities and Exchange Commission (available at www.sec.gov).
The information contained in this presentation is as of this date and TYME assumes no obligation to update forward-looking statements contained in this
presentation as a result of future events or developments.
2
TYME Technologies
TYME is an emerging biotechnology company
focused on exploring novel therapeutic
approaches designed to
target cancer’s unique metabolism
TYME is advancing proprietary
Cancer Metabolism-Based Therapies (CMBTs™)
for difficult-to-treat cancers
3
TYME: Investment Rationale
A leader in Cancer Metabolism-Based Therapies (CMBTs™):
Over a decade of experience studying Cancer Metabolism-Based Therapies (CMBTs) with a strong patent portfolio
broadly covering compositions, methods, manufacturing and use extending beyond 2032
Differentiated MOA:
TYME is exploiting the extensively studied Warburg Effect by developing a first-in-class approach to kill cancer cells
through disrupting cancer metabolism through multiple mechanisms of action. TYME believes this unique
approach can provide broad therapeutic efficacy without unnecessary off-target toxicity
Lead Candidate, SM-88, in Pivotal Trials:
⬣ Enrolling patients in pivotal TYME-88-Panc Part 2 trial for third-line pancreatic cancer
⬣ Enrolling patients in Phase II/III Precision Promise pivotal trial in second-line pancreatic cancer
⬣ Enrolling patients in HopES Sarcoma Phase II trial for Ewing’s & high-risk sarcomas
⬣ Preparing SM-88 for clinical programs in cancers where it has previously shown responses in early clinical trials,
including breast, prostate and blood cancers
Large Growing Markets with Limited Therapeutic Options:
Targeting metastatic cancers for which there are limited options, including pancreatic, sarcoma, prostate, breast cancers and more
Fight Against COVID-19:
Initiating proof-of-concept RESPOnD trial to investigate TYME-19 for safety and efficacy in recently diagnosed,
symptomatic COVID-19 patients
4
Delivering on Key Milestones Positions
TYME for Long-Term Success Complete enrollment in
TYME-88-Panc pivotal study
Complete enrollment in HopES Sarcoma study
Initiate enrollment in PanCAN’s Precision
Advance SM-88 clinical programs into
PromiseSM adaptive randomized Phase II/III
other tumor types potentially including
trial in patients with pancreatic cancer using
metastatic breast, recurrent prostate
oral SM-88 in second-line monotherapy
and/or hematological cancers
Abstracts to be presented
on preclinical data for SM-88 Advance plans for
Advance & TYME-18 at AACR TYME-18 IND-
enrollment in enabling program
TYME-88-Panc
Pivotal Study Complete TYME-19
proof of concept trial
Publish SM-88 Phase II
prostate study
Present and/or publish
Orphan Drug final data from Part 1 of
Designation for SM-88 TYME-88-Panc study
Present Health Economic Outcomes study
Advance enrollment in the on total cost of care for pancreatic cancer
HopES Sarcoma Phase II Trial patients at ISPOR & ASCO
5
Advancing Innovative Pipeline of
Cancer Metabolism-Based Compounds (CMBTsTM)
PROGRAM FORMULATION INDICATION DEVELOPMENT STAGE PHASE I PHASE II PHASE II/III
Pancreatic: Third-Line TYME-88-Panc Pivotal Part 2: Enrolling
Pancreatic: Second-Line Monotherapy Precision Promise: Enrolling Shortly
Pancreatic: First-Line Combo w/GA Precision Promise: Initiate Following Second-Line
SM-88 Oral Prostate: Biomarker Recurrent Completed
Metastatic Sarcomas* HopES: Enrolling
Future Trials: Breast and Prostate
Future Trials: Hematology
SM-88i Injectable Digestively Compromised Patients
SM-88n Nasal Brain/Glioma
Intra-
TYME-18 tumoral
Solid Tumors
TYME-19 Oral Anti-viral
6
Expanding Breadth and Depth of Strong Patent Portfolio
Patents broadly cover compositions, methods, manufacturing
2032
and use of the Company’s pipeline to 2032, and beyond
EU
APAC
US
GLOBAL: 194 Patent Applications Granted and/or Pending
7
Expanding Opportunities for Cancer
Metabolism-Based Therapies to Transform
Treatment of Metastatic Cancers
PANCREAS PANCREAS SARCOMA
(Third-line) (First, Second and Third-line) (Ewing’s & High-Risk)
10K INCIDENCE 57K INCIDENCE 12K INCIDENCE
$0.9B MARKET
OPPORTUNITY $5.1B MARKET
OPPORTUNITY $1.1B MARKET
OPPORTUNITY
PROSTATE BREAST HEMATOLOGY
(Recurrent) (Metastatic) (DLBC, R/R AML)
450K PREVALENCE 150K INCIDENCE 48K INCIDENCE
$14.4B MARKET
OPPORTUNITY $19.4B MARKET
OPPORTUNITY $8.4B MARKET
OPPORTUNITY
source: IQVIA global oncology market trends 2019; American Cancer Society’s cancer facts & figures 2019; www.ncbi.nlm.nih.gov; 8
drugs.com; ajmc.com; boneandjointburden.org
UNIQUE
SCIENTIFIC APPROACH
9
Exploiting Warburg Effect Through Modified Dysfunctional
Amino Acids Targeting Cancer’s Unique Metabolism
1. Induce uptake of TYME’s
modified dysfunctional Tyrosine 2. Protein synthesis fails
SM-
88 3. Cell death from
88
SM - oxidative stress
SM
- 88
Free
4. Decreased
5. Reduce Immunosuppressive Radicals
cellular defenses
Cells By Blocking Upstream (ROS)
10CLINICAL TRIALS:
METASTATIC CANCER
11SM-88 Achieved Confirmed Clinical Responses
Across 15 Tumor Types
Success in pancreatic cancer may offer a path for
SM-88 development into many of the 15 advanced cancers
where imaging responses were demonstrated
Cancers with Demonstrated Responses to SM-88
Pancreatic Breast Ovarian
Prostate Colon Glioma/Glioblastoma
Ewing’s Sarcoma Renal Appendix
Soft-Tissue Sarcoma Thyroid Hodgkin’s Lymphoma
Lung Head & Neck Non-Hodgkin’s Lymphoma
*NCI.org statistics for 2018
12CLINICAL TRIALS:
PANCREATIC CANCER
13U.S. Pancreatic Treatment Paradigm
# of Patients
Diagnosed (U.S.) 55,400 Localized ~20%
Metastatic at Diagnosis ~80%
ASCO Guidelines (Metastatic) Historical Trial Medians
ORR Survival
®
 Gemzar / Abraxane® (“GA”); or
 FOLFIRINOX ~30% 8-11 months
Receive 1st Line > 41,000
 Single agent (ECOG 2)
 Onivyde® +5FU/LV (GA-failed)
 GA (5FU-failed) ~10% 4-6 months
Receive 2nd Line > 20,000
 Single agent (ECOG 2)
“No data are available to
recommend third-line (or greater) ~0% 2-3 months
Receive 3rd Line > 10,000
therapy with a cytotoxic agent”
Source: 2018 American Cancer Society patient statistics; Metastatic Pancreatic Cancer: ASCO Clinical Practice Guidelines; Abrams et al 2017; Bachet et al 2009
14TYME-88-Panc Overall Survival (OS)
and Clinical Benefit Rate (CBR)
RECIST Disease Control
HR: 0.08 (95% CI 0.01 – 0.63)
p = 0.02
Data cutoff as of 4/25/19 Data cutoff as of 4/25/19
 Third-line PDAC has no established therapy  44% (11/25) RECIST Clinical Benefit Rate (SD or PR)
 Previously reported survival for third line PDAC patients is approximately  Patients who achieved at least SD by first assessment demonstrated
2.0 – 2.5 months (Manax et al. ASCO GI Poster 2019) statistically significant greater survival than PD patients
 The preliminary median Kaplan-Meier (KM) derived overall survival of  Patients who achieved at least RECIST SD had a 92% reduction in risk of
death
the evaluable population is currently 6.4 months
ESMO GI Poster
15
SM-88 Therapy in High-Risk Poor Prognosis Pancreatic CancerTargeted Mechanism of Action Delivering
Favorable Safety Profile Compared With
Other Cancer Treatments
 SM-88 has demonstrated well tolerated safety profile with only 4% of patients
reporting serious adverse events (SAEs) across multiple clinical trials
 SM-88 has demonstrated a favorable safety profile in 15 different tumor types,
including solid tumors and hematologic malignancies across four separate studies
Data cutoff as of 4/25/19
16Enrolling Patients in TYME-88-Panc Pivotal Trial
SM-88 used with MPS in Patients with Metastatic Adenocarcinoma
PIVOTAL of the Pancreas Whose Disease Has Progressed or Reoccurred
Study Identifier: NCT03512756
KEY ELIGIBILITY CRITERIA DESIGN ENDPOINT(S)
⬣ Histologically confirmed Primary: OS
pancreatic adenocarcinoma SM-88 Secondary*: PFS, CBR,
Treatment until
(N=~125)
SCREENIN
unacceptable and QoL
⬣ Received second-lines of R1:1
toxicity, disease
prior systemic therapy Key Exploratory Endpoints*:
progression or
any treatment Biomarker analysis, including
CTCs
G
⬣ Adequate organ function Investigator-chosen Therapy discontinuation
criteria are met
(N=~125)
* Other secondary and
exploratory endpoints will
also be captured.
17CLINICAL TRIALS:
PRECISION PROMISESM
PANCREATIC CANCER
18PanCAN: World’s Largest Advocate Committed
to Curing Pancreatic Cancer
PanCAN Precision PromiseSM
Clinical Trial Consortium Sites
“ Precision Promise first
is the
response-adaptive randomized
clinical trial platform for pancreatic
cancer patients in the world and the Pancreatic
Cancer Action Network’s groundbreaking
initiative to dramatically improve outcomes for
pancreatic cancer patients and advance the
organization’s goal to double survival.
– Pancreatic Cancer Action Network
”
19CLINICAL TRIALS:
PROSTATE CANCER
20SM-88 Demonstrated Potential To Postpone
Hormone Therapy in Recurrent Prostate Cancer
⬣ At 6 months, 100% (23/23) of
patients were free of metastatic
progression, and 87% of patients
remained free of radiographic
progression
⬣ After 3 months, 78% (18/23) of
patients demonstrated a median
65% decrease in median CTCs from
baseline
⬣ 52% (12/23) of patients showed
improvement in median PSA
doubling time
⬣ No drug-related severe or life-
Data cutoff as of September 2019
threatening adverse events (grade 3
Benjamin Gartrell1 Mack Roach2 Giuseppe Del Priore3 Avi Retter4 Wen-Tien Chen5 Gerald H. Sokol6 Alexander Vandell3 Howard I. Scher7 or 4) were observed after cumulative
1)Albert Einstein College of Medicine/Montefiore Medical Center 2)University of California San Francisco 3)TYME Inc. 4)ECCC/NY Cancer and Blood
Specialists 5)LineaRx 6)Florida Cancer Specialist 7)Memorial Sloan-Kettering Cancer Center
dosing exposure of 149 months
ESMO Poster
21
Phase II Trial of SM-88 in Non-Metastatic Biochemical Recurrent Prostate CancerCLINICAL TRIALS:
SARCOMAS
22JAF HopES Trial: Enrolling Patients with
Ultra-Rare Metastatic Sarcoma
 TYME, Dr. Sant Chawla and The Joseph Ahmed Foundation (JAF) are addressing unmet
need in ultra-rare metastatic sarcoma
 JAF is funding the trial and is using its nationwide network to assist potential patients and their families
 Based on compassionate use results in two metastatic Ewing’s sarcoma patients who achieved CR or
PR, with no drug-related SAEs
 If proof-of-concept is demonstrated, a multi-site confirmatory study will be evaluated
Ewing’s and High-risk Sarcoma:
 Ewing’s accounts for 30% of bone cancers in
children
 Tumor of the bone or soft tissue, most
often in the pelvis, thigh, lower leg, upper
arm and chest wall
 30% 5-year survival rate
for metastatic disease
 All sarcomas represent 12,000 new cases
annually in U.S. alone
23SM-88 for Advanced Ewing's Sarcoma and
Salvage Therapy for Sarcoma Patients (HopES)
SM-88 Used With MPS in Patients With High-Risk
Phase 2 Ewing’s and Other Sarcoma Types
Study Identifier: NCT03778996
KEY ELIGIBILITY CRITERIA DESIGN ENDPOINT(S)
⬣ Bx proven previously Primary: Response Rate
treated Sarcoma SM-88 in Ewing’s Secondary*: PFS, CBR
SCREENIN (N=12) Treat until
⬣ High Risk for PD ie > 2 Progressive
disease or
prior lines of systemic
unacceptable
treatments SM-88 in Other Sarcomas toxicity
G
(N=12) * Other secondary and exploratory
endpoints will also be captured.
⬣ Ewing’s patients may
be treated in SD
immediately following
1st or 2nd line therapy
24CLINICAL TRIALS:
BREAST CANCER
25Compelling SM-88 Data in Patients
With Metastatic Breast Cancer
 SM-88 demonstrated clinical benefit in metastatic breast cancer (mBC) with a favorable safety and
quality of life profile. There was no indication of cross-resistance based on hormone receptor profile,
prior treatments, or metastatic site
 A total of 25 mBC patients were treated with SM-88 between 2012–2017
 All subjects had previously treated progressing mBC
 Subjects had a median of 3 prior therapies (range of 1 – 8)
 Overall response rate was 44% (11/25)
 16% (4/25) Experienced a Complete Response
 79% Improved ECOG Performance Status
 57% Pain Reduction (NRS-11)
 There were no unanticipated or drug-related adverse events
26TYME-19
DEVELOPMENT PROGRAM
27Clinical Development Plan
 Initiating a proof-of-concept study to evaluate efficacy
DEVELOPMENT PARTNERS
of oral TYME-19 versus placebo (“RESPOnD”)
 Targeting newly diagnosed, symptomatic patients who
are high-risk, but do not require hospitalization
 Evaluating safety as well as change in patient
symptoms, viral levels, hospitalization and other
efficacy endpoints
 Protocol developed in partnership with researchers
from Mass General and Cornell
 Will be run as an investigator-initiated trial
 TYME has sourced cGMP drug substance to allow
rapid clinical development
 The Company is in discussions with the FDA to
determine the appropriate regulatory strategy
28Why is This Important and Different?
 Doctors need more tools between hospital care and vaccines
 A simple, well tolerated anti-viral therapeutic could be used early after diagnosis to prevent
the need for hospitalization
 Vaccines are a key part of the solution for a pandemic, but there needs to be better
therapies available while the vaccines are being developed
 By using an endogenous cellular regulator that targets basic functions of viruses, there is
potential for broad spectrum anti-viral effect
 It will be critical to have readily-available, effective drugs when the next viral outbreak or
pandemic occurs
 Potential use of TYME-19 or related drugs in other viruses
 TYME-19 is expected to be a shelf stable compound, relatively inexpensive to manufacture
and suitable for global distribution
 TYME-19’s mechanism may support prophylactic use to protect our first responders and most
at-risk populations
29TYME-19’s Antiviral Mechanisms
1 2
Inhibits Inhibits
Production of Production
Viral Proteins of Viral Lipids
3 4
Upregulates Physically
Innate Degrades
Immunity Viruses
30Next Steps
 Initiating Proof-of-Concept RESPOnD Trial
 Ongoing discussions with FDA
 Pursuing government funding opportunities to advance TYME-19
development
 Building portfolio of antiviral metabolic agents
31KEY MILESTONES FOR
FISCAL YEAR 2021
322020 Creates Pivotal Inflection Point
with Multiple Value Drivers
Milestone Timing
Advance enrollment in TYME-88-Panc pivotal study,
2021
and the HopES Phase II Trial
Advance enrollment in PanCAN’s Precision PromiseSM adaptive randomized Phase II/III
2021
Trial in patients with pancreatic cancer using oral SM-88 in second-line monotherapy
Clinical Trial Advance SM-88 clinical program as a potential oral treatment for patients with metastatic
Milestones 2021
breast, recurrent prostate and/or hematological cancers
Explore Opportunity with PanCAN’s Precision PromiseSM adaptive Phase II/III trial
evaluating SM-88 in patients with first-line pancreatic cancer in combination with 2021
gemcitabine and Abraxane
Complete enrollment in TYME-88-Panc pivotal study Not before 2022
Present preclinical data for SM-88 1H’2020
Present preclinical data for TYME-18 1H’2020
Preclinical &
Clinical Data Publish SM-88 Phase II prostate study 2H’2020
Milestones
Present and/or publish final data from Part 1 of TYME-88-Panc study 1H’2021
Complete TYME-19 proof of concept trial 1H’2021
Present preliminary Health Economic Outcomes study on total cost of care
1H’2020
for pancreatic cancer patients
Other
Orphan Drug Designation for SM-88 2H’2020
Advance plans for TYME-18 IND-enabling program 1H’2021
331 Pluckemin Way, Suite 103 Bedminster, NJ 07921 NASDAQ: TYME media@tymeinc.com investorrelations@tymeinc.com TYMEinc.com
You can also read
