Effective & durable treatments for cancer and immunological diseases - Corporate Presentation April 2021
←
→
Page content transcription
If your browser does not render page correctly, please read the page content below
Effective & durable treatments for cancer and immunological diseases
Corporate Presentation April 2021
Disclaimer
This presentation is being provided for the sole purpose of providing the recipients with background information about Stamford Pharmaceuticals,
Inc.’s (“Stamford”) business. This presentation, including the information contained in this disclaimer, does not constitute an offer, invitation or
recommendation to subscribe for or purchase any security, and neither the presentation, disclaimer nor anything contained in them forms the basis
of any contract or commitment. This presentation does not purport to summarize all information that an investor should consider when making an
investment decision. Before making an investment decision you should consider whether it is suitable for you in light of your own investment profile
and objectives and financial circumstances and the merits and risk involved.
No representation, express or implied, is made as to the fairness, accuracy, completeness or correctness of information, opinions and conclusions
contained in this presentation, including the accuracy, likelihood of achievement or reasonableness of any forecasts, prospects, returns or statements
in relation to future matters contained in the presentation (“forward-looking statements”). Such forward-looking statements are by their nature
subject to significant uncertainties and contingencies and are based on a number of estimates and assumptions that are subject to change (and in
many cases are outside the control of Stamford and its Directors) which may cause the actual results or performance of Stamford to be materially
different from any future results or performance expressed or implied by such forward-looking statements. Forward-looking statements are provided
as a general guide only and should not be relied upon as an indication or guarantee of future performance.
To the maximum extent permitted by law, neither Stamford nor its related corporations, directors, employees or agents, nor any other person, accepts
any liability, including, without limitation, any liability arising from fault or negligence, for any loss arising from the use of this presentation or its
contents or otherwise arising in connection with it.
You represent and confirm by attending and/or retaining this presentation, that you accept the above conditions. This presentation does not
constitute an offer to sell or a solicitation of an offer to buy securities in the United States.
2 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
Executive Summary
Late-stage clinical development Lead product, SP-002, is a viral-vector SP-002 demonstrated histological
company founded in 2019, developing based immunotherapy encoding the clearance rates of 83% in Basal Cell
novel therapies for oncology and gene for human interferon gamma Carcinoma (BCC) lesions, as a
inflammatory diseases (IFNγ) and being developed for skin monotherapy, in a recent Phase 1/2a
cancers trial of 15 patients
IND opened and Orphan Fully funded pilot phase of Layered intellectual property strategy
Designation granted by the FDA Registrational combo trial for SP-002 covering compositions, formulations
+ Erivedge® in collaboration with and use patents plus biologic statutory
Roche/Genentech initiated in July – protection
Near-term readouts in 2021
3 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
Our Science & Technology 8/04/2021 4
SP-002 Mechanism of Action: Adenovirus Encoding Interferon-γ
Anti-Angiogenic Effect Immuno-oncology MOA
Decreased density of tumor blood vessels
Antigen Adaptive Innate
Presentation Immunity Immunity
Ad-null treated tumor Ad-IFN-γ treated tumor
shows increased number shows decreased number Induction of Induction of Induction of
of blood vessels of blood vessels multiple dendritic humoral and innate immune
cell populations cellular immunity effectors
Potent Cellular Response
Humoral Response Induced by SP-002:
Induction of antibody responses to tumor
antigens
Increased immune infiltrates in Increased immune infiltrates in
cutaneous T-cell lymphoma cutaneous B-cell lymphoma
complete responder at baseline complete responder at baseline
(L) and post-treatment (R) (L) and post-treatment (R)
5 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
Meeting an Important Need in Skin Cancers
6
8/04/2021
Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
6
Addressable BCC Patient Population
Patients with surgically treatable BCCs
Moderate disease burden: Patients presenting with multiple BCCs (3+ lesions/presentation)
~75k patients annually in the US
High disease burden: Patients with multiple BCCs/high number of lesions (6+ per presentation)
High BCC disease burden ~15,000 (each patient will have potentially 6+ lesions treated)
Patients with Basal Cell Nevus Syndrome
High BCC disease burden (median 30 lesions/patient/yr) ~11,000 patients in US alone
Patients with single BCC (incl. lesions in H-zone, high-risk subtypes and local. advance disease)
2-2.5M patients annually in US
Based on pre-filled syringe comprising a novel composition comprising SP-002 with a second small
molecule inhibitor
Incidence of BCC estimated to be increasing worldwide between 7-10% annually1,2
1. CSM Wong et al. BMJ. 2003; 327(7418): 794–798.
2 SC Flohill et al. JID 2013; 133(4), 913-918.
PAGE 7
Basal Cell Nevus Syndrome
“SP-002 has the potential to be a new treatment paradigm for nevus patients.”
Gregory Siller, MD – Basal Cell Carcinoma Key Opinion Leader
Unmet Need
A non-surgical modality that is curative for high-threat lesions and provides systemic control for lower risk and
new lesions
Basal Cell Nevus Syndrome
▪ BCNS is a rare orphan disease, ~30,000 patients worldwide and 10,300 patients in the US
▪ Patients have a genetic mutation in the Hedgehog signaling pathway that leads to growth of hundreds of primary BCCs
annually - median 29 lesions a year has been reported1
Current Management
▪ Surgical excision is the only definitive treatment to manage high risk tumors when they reach a certain size or threat level
o With current US reimbursement, it is common for three or fewer BCCs to be removed per visit2
o Patients may undergo multiple procedures on a weekly or monthly basis
o Surgery exerts significant morbidity, disfigurement and procedural fatigue on patients
▪ Hedgehog inhibitors (HHI) can manage BCCs systemically, but are poorly curative3 and have significant side-effects
o Discontinuation of HHI treatment also results in the recurrence of ‘’resolved’’ BCCs
1-Solis et al, JAMA Dermatol. 2017, 153:189; 2-Carriers reduce the reimbursement on additional surgeries when multiple procedures are performed during a visit; 3-Erivedge® has a cure rate of ~20% and has side-effects such as muscle cramps, hair
loss and taste loss
8 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
Nodular Basal Cell Carcinoma in Sporadic Patients
4 million cases 1 of BCC diagnosed in the US annually comprising ~75% nodular BCC and
~15% superficial BCC
• Majority of nodular BCC arises on the head & neck
• Main treatments are surgical options
Positioning of SP-002
• For patients concerned about scarring, morbidity & protracted recovery time
Most nBCC lesions occur If untreated, BCCs can
on the face, head and cause extensive
• A more effective, better tolerated and convenient alternative to imiquimod (and other topicals)
neck disfigurement
Target Physicians
• For the general dermatologist that would refer BCCs on the head/neck to Mohs surgeons (i.e., this
will be most general dermatologists in the US. Also ~80% of nodular BCCs are on head and neck)
Product profile and % of BCC population addressable, if:
Excision margins of 2-5 mm are recommended for
• An ~85% Histological Clearance rate could address ~10% of patients2 most BCC excisions
• Over 90% Histological Clearance rate can be a new standard of care for low risk superficial and
nodular BCCs
1JAMA Dermatol 2015, 151(10):1081; American Cancer Society. http://www.cancer.org/cancer/skincancer
2Physician survey that about a significant number of BCC patients are particularly concerned about disfigurement, morbidity and healing time and willing to accept a slightly lower cure rate
9 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com 9Development / Clinical Plan
10
8/04/2021
Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
10Development Strategy
Starting with low risk indications & progressing to more advance settings
BCCs Other Cancers Other Cancers
Label expansion Multi-lesional
BCC
Bowens Disease1 Ovarian Cancer
(BCNS/Sporadic
BCC)
(incl. lesions in
Single lesion H-zone, high-
Cutaneous SCC
Cutaneous T/B
Nodular & risk subtypes and (Locally
locally advance
Cell Lymphoma2
Superficial BCC advanced)
disease)
Cutaneous
Cutaneous Melanoma &
Breast Cancer Melanoma In
Situ2,3
1Including Keratoacanthomas (KA) Currently Funded
2Positive POC data for Cutaneous Lymphoma/Mycosis Fungoidis
3Positive POC data for Advance Melanoma plus TILs Additional financing(s)
11 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comClinical Development Plan
Treatment of Multiple BCCs in BCNS and Mult-lesional Sporadic BCC Patients
Completed Pilot Phase (NCT 04416516) Confirmatory Phase
Phase 1/2a Choose optimal BCNS (multiple lesions)
Study combination dose &
excision timepoint1 RCT
~10-14 months ~14-18 months
Pilot Phase Registration study Confirmatory Phase Registration
study: randomized controlled study
▪ Pilot study in collaboration with
comprising ~150 subjects2
Roche/Genentech being conducted in
Australia by our wholly owned
subsidiary, Ascend Biopharmaceuticals
Ltd.
▪ Adaptive design - up to 84 subjects
▪ Primary objective to evaluate the
tolerability & efficacy of SP-002 &
Hedgehog Pathway Inhibitors
▪ Initiated July 2020
RCT: Randomized controlled study design
1-Evaluation at week 24/32 and booster injection may improve histological clearance rate - FDA meeting planned with interim data time-point
2-Final design pending FDA EOP2/Type C meeting
12 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comPilot Phase Study Design: Safety and Efficacy of SP-002 Combined
with Vismodegib (Erivedge®), a Hedgehog Pathway Inhibitor
Arm 1 Single Target Tumor (single lesion)
Vismodegib: 150 mg daily for 4 weeks
1 3 Arm 3 Lower Dose SP-002: 0.5x1011 vp /target tumor1
Arm 5 Higher Dose SP-002: 1.5x1011 vp /target tumor1
SP-002 1.0x1011 vp/target tumor1
Adaptive Design2
Parallel
Week 25/33
Recruitment Excision & Assessments
Arm 2 Multiple Target Tumors (3 lesions)
Vismodegib: 150 mg daily for 4 weeks
1 3 Arm 4 Lower Dose SP-002: 0.5x1011 vp /target tumor1
Arm 6 Higher Dose SP-002: 1.5x1011 vp /target tumor1
SP-002 1.0x1011 vp/target tumor1
• Up to 84 subjects (arms n= 6 or 12) 3
• Primary endpoints - Clinical Response and
Histological Clearance, Safety and Tolerability
1 Dosing 1q/wk x 3 for SP-002 and concurrently administered with vismodegib: 150 mg daily for 4 wks
2 Based on Week 6 safety and tolerability data and week 17 clinical response
3 Arms 7 and 8 are SP-002 monotherapy and vismodegib monotherapy arms
13 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comCurrent Clinical Development Timeline – Pilot Study
Funded through Pilot Phase Study Readouts
2020 2021
Jun Jul Aug Sep Oct Nov Dec Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Dec
Recruitment
Pilot study (24 subjects currently approved to be recruited)
Pilot Phase of Registration Study
Interim data
FDA Type C Meeting *
▪ SP-002 pilot study initiated in July in collaboration with Roche/Genentech
o Fully funded through data readouts in 1Q and 4Q, 2021
▪ Initiation of SP-002 Registration trial phase planned for 2H, 2022 pending FDA Type C & EOP2 meetings
o Expect to initiate Registration trial phase with pharma partner
1
4
14 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comClinical Data: Phase 1/2a Trial
15
8/04/2021
Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.com
15Efficacy Results - BCNS and Sporadic BCC
Lesion at baseline Dose Lymphocytic
Subject # Cohort Histological Clearance
(mm) (VP) Infiltration
02-001 Low Dose 7 5x1010 Minimal No
02-003 Low Dose 9 5x10 10
Moderate Yes ▪ 83% histological
03-001 Low Dose 7 5x1010 Moderate No clearance rate1; the two
01-003 Intermediate Dose 8 1.5x1011 Moderate Yes patients not
03-004 Intermediate Dose 5.5 1.5x1011 Moderate No histologically cleared
02-006 Intermediate Dose 8 1.5x1011 Moderate Yes had Partial Responses
03-006 Intermediate Dose 6.5 1.5x10 11
Moderate Yes (16.7%)
Optimal Doses
11
02-008 Intermediate Dose 6 1.5x10 Moderate Yes
01-006 Intermediate Dose 8 1.5x1011 Moderate Yes ▪ Overall Response Rate
01-005 High Dose 16 3x1011 Dense Yes for the intermediate
03-005 High Dose 8.5 3x1011 Dense Yes and high dose cohort
02-007 High Dose 10 3x1011 Dense Yes was 100%2
11
03-008 High Dose 10 3x10 Dense Yes
11
03-009 High Dose 6 3x10 Moderate No
02-009 High Dose 6 3x1011 Moderate Yes
1-Based on subjects from intermediate and high dose cohorts
2-One excluded cohort IV subject was deemed to have micro-nodular BCC, an excluded subtype as per protocol
16 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comAbscopal Response - BCNS and Sporadic BCC
▪ SP-002 can cause clinical regression in non-injected BCCs
▪ Evidence of a systemic immune response directed at non-injected BCCs
1.00 ▪ 70% with clinically
100.00% Non-Target Lesion Response
n=20 assessible regressions
% CHANGE FROM BASE-LINE - CLINICAL
80.00% (14/20)
60.00%
▪ 45% with PR or CR
40.00% (9/20)
0.25
RESPONSE
0.20 0.17
20.00%
0.00 0.00
0.00%
1 2 3 4 5 6 7
-0.06 8 9 10 11 12 13 14 15 16 17 18 19 20
-20.00% -0.14 -0.17
-0.25 -0.29
-40.00% -0.30 -0.30
-0.38 -0.40 -0.40
-0.47 -0.50
-60.00%
-0.60
-80.00%
INDIVIDUAL LESIONS
-100.00%
-1.00
17 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comLocal Skin Responses - BCNS and Sporadic BCC
SP-002 is Well Tolerated
The 2 Subjects with the Highest Local Skin Reaction Scores
Subject 02-006
Week 1 Week 4 Week 16
Subject 02-009
18 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comSafety Results - BCNS and Sporadic BCC
SP-002 is Safe at All Doses Evaluated
▪ Toxicity profile in 15 completed eligible nodular BCC subjects treated with weekly injections x 3 and excision
at week 17
o Largely limited to:
- Grade 1-2 local reactions (erythema, induration, ulceration and pain) at the injection site
- Grade 1-2 flu-like symptoms (headache, malaise, fever, body aches and adenopathy) of 1-2 days
duration
o Only Grade 1 flu-like reactions were observed in the two lower dose cohorts
o Grade 2 flu-like reactions were observed only in the highest dose cohort in 2 of the 6 eligible subjects
o Similar safety profile to the 51 previously treated patients with cutaneous lymphoma
19 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comCompetitor Landscape
SP-002 has Best-in-Class Efficacy
HedgePath/
Sun/Novartis Roche Pelle Pharm Stamford
Mayne
Compound Sonidegib (Odomzo®) SUBA™-Itraconazole Vismodegib (Erivedge®) Patidegib SP-002
Locally advanced BCC Locally advanced BCC BCNS
Indication BCNS BCNS and sporadic BCC
(used off-label in BCNS) (used off-label in BCNS)
Route Oral Oral Oral Topical Intra-lesional
150 mg twice daily for 16
Schedule 200 mg once a day 150mg once a day Twice daily for 26 weeks Once a week for 3 weeks
weeks
20% CR 41% CR1 20.6% CR 27% CR 83% CR3
Efficacy Rate
35% PR 54% PR 22.2% PR PR unreported 17% PR
66 patients 38 patients 71 patients 17 patients 15 patients
Sample Size
Phase 3 Phase 2 Phase 3 Phase 2 Phase 1/2a
Mild to moderate flu like symptoms
Redness, itching and
AE profile Significant AEs Grade 2 or higher AEs Significant AEs2 only in the high dose cohort (in 2
swelling
subjects out of 6)
Novartis sold the rights to Leo Pharma provided Superior cure rates & tolerability
Odomzo® to Sun Pharma US$70m funding and has an compared to approved (Odomzo®
Notes for US$175m plus option to purchase and Erivedge®) and in development
milestones and royalty company for an additional products (Patidegib and SUBA-
payments US$690m itraconazole)
CR: Complete Response; PR: Partial Response
1-As of last interim report January 2018
2-Muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, pruritus, lymphopenia and anaemia
3-Study assessed responses by Histological Clearance which is an FDA approved endpoint
20 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comStamford Pharma Management Team & Board of Directors
Dr. Jacques Banchereau, PhD / Non-Executive Director
Clement Leong, PhD, MBA / Chief Executive Officer &
▪ Currently Director of Immunological Sciences and the Deputy
Executive Director
Director of JAX Genomic Medicine. Formerly Chief Scientific
▪ Pharma/biotech/venture capital background in Australia and US;
Officer of the Hoffmann-La Roche (Roche) & Director of the
led and completed multiple financings & successful clinical teams
Baylor Institute for Immunology Research in Dallas, Texas
▪ PhD from University of Western Australia; MBA from the
▪ PhD University of Paris, France
Australian Graduate School of Management
Dr. Edward McKenna, PhD / Non-Executive Director
Dr. Jacqui Waterkeyn / VP. Clinical Operations & Regulatory ▪ Previously, Principal Medical Science Director at Genentech with
Affairs responsibilities as Medical Lead for Dermatologic Oncology and
▪ Regulatory Affairs and Clinical Operations background (20+ the following Roche/Genentech products: Zelboraf, Cotellic.
years) in the Biotechnology and Pharmaceutical Industries, Tecentriq and Erivedge.
including Clinical Research Organizations ▪ PhD, University of Kentucky
▪ PhD from the University of Melbourne
Mr Andrew Strong, JD / Non-Executive Director
Dr. Geoffrey Pietersz, PhD / VP. Technology Development ▪ Andrew Strong a Pillsbury partner in the firm’s Houston and
▪ Formerly professor at the Burnet Institute, expert in bio- Austin offices, has extensive experience representing many of the
conjugation of therapeutics (e.g., vaccine; antibody drug firm's life sciences clients and routinely advises on corporate and
conjugates) securities, mergers & acquisitions, private and public financing.
▪ PhD from University of Melbourne Andrew was also the Founding President and CEO of Kalon
Biotherapeutics.
Dr. Satish Menon, PhD / Director CMC ▪ J.D., South Texas College of Law; B.S., Civil Engineering, Texas
▪ Consultant, Allergan; Senior Consultant, PVP Biologicals; Senior A&M University
Technology Consultant, Seq-Biomarque LLC
▪ PhD Biochemistry, Indian Institute of Science, Postdoctoral,
Harvard Medical School
21 Stamford Pharmaceuticals Inc. | stamfordpharmaceuticals.comEffective & durable treatments for cancer and immunological diseases
Stamford Pharmaceuticals Inc
Two Houston Center, 909 Fannin, Suite 2000
Houston, TX 77010-1028
Ph.: 203-516-7895
Email: info@stamfordpharmaceuticals.com
Web: stamfordpharmaceuticals.comYou can also read
