DirectHit Test Panel for Breast Cancer helps physicians predict which anticancer therapy treatments will be the most effective for individual breast cancer patients. Clinical trial results show that DirectHit predicts, with a high degree of accuracy, the tumor’s resistance and sensitivity to four commonly prescribed classes of breast cancer treatments.
OneMedForum New York 2010 Company Presentation: Transdel Pharmaceuticals, Inc., a specialty pharmaceutical company, develops non-invasive, topically-delivered medications in the United States. Learn More at: http://www.onemedplace.com/database/list/cid/13943
Experts from Informa’s Medtrack, Trialtrove and Strategic Transactions teams presented a panel discussion at the recent T3 Conference in Orlando, Florida.
DirectHit Test Panel for Breast Cancer helps physicians predict which anticancer therapy treatments will be the most effective for individual breast cancer patients. Clinical trial results show that DirectHit predicts, with a high degree of accuracy, the tumor’s resistance and sensitivity to four commonly prescribed classes of breast cancer treatments.
OneMedForum New York 2010 Company Presentation: Transdel Pharmaceuticals, Inc., a specialty pharmaceutical company, develops non-invasive, topically-delivered medications in the United States. Learn More at: http://www.onemedplace.com/database/list/cid/13943
Experts from Informa’s Medtrack, Trialtrove and Strategic Transactions teams presented a panel discussion at the recent T3 Conference in Orlando, Florida.
Targovax Next generation immune activators for solid tumorsRoarFredriksen1
Targovax (OSE:TRVX) is a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors. Targovax’s focus is to activate the patient’s immune system to fight cancer, and to bring benefit to cancer patients with few available treatment alternatives. Targovax is developing its product candidates in different cancer indications, including melanoma, mesothelioma, and multiple myeloma, and has demonstrated a favorable safety and tolerability profile.
Targovax’s lead clinical candidate, ONCOS-102, is a genetically modified oncolytic adenovirus, which has been engineered to selectively infect cancer cells and activate the immune system against the tumor. Following very encouraging clinical data in several indications, both as monotherapy and in combinations, ONCOS-102 is progressing into a randomized phase 2 trial in melanoma patients resistant to PD-1 checkpoint inhibitor treatment.
Building on successful clinical studies which have provided deep mechanistic insights into the tumor biology and the human immune systems, Targovax is researching circular RNA (circRNA) as novel cancer medicines. In addition, Targovax has a KRAS immunotherapy program, with lead cancer vaccine candidate, TG01, expected to enter the clinic in an enhanced format in the second half of 2022. Together this provides Targovax with a rich pipeline of innovative future immunotherapy product candidates to follow ONCOS-102.
User perspective for somatic variant analysis in VSClinical AMPGolden Helix
Somatic analysis is a complex and precise process that is constantly evolving. As the volume of available data and the accessibility of sequencing technology increase, so too does the value of a versatile, well-vetted, and efficient workflow solution. In this webcast, we will take a deep dive into the current state of our AMP interpretation software and explore various ways to optimize workflows. For anyone from grizzled VarSeq veterans to those seeing our software for the first time and labs of any size, we will provide a practical overview of our somatic analysis capabilities and how those capabilities scale with improving technology.
Throughout this webcast we will be discussing the following:
- Universal principles of somatic workflows, providing baseline recommendations
- Specific tumor-normal and somatic-only use cases
- VSClinical AMP interpretation hub and some variants of interest
- Opportunities for automation and how to decrease time to report for increased throughput
Join us as we show off the versatility and scalability of our AMP interpretation capabilities!
Targovax is developing two complementary and highly targeted approaches to cancer immunotherapy: a peptide-based targeted immunotherapy platform for patients with RAS-mutated cancers and a virus-based oncolytic immunotherapy platform based on engineered oncolytic viruses armed with potent immune-stimulating transgenes for patients with solid tumors.
Utilizing Doppler Flow to Investigate the Hemodynamic Effects of Anti-Cancer ...Scintica Instrumentation
Recent advances in targeted cancer therapies has led to the development of a number of novel approaches that have demonstrated some clinical advantages in terms of prolonging overall survival and progression free survival. One of these approaches involves inhibiting angiogenesis (the growth of blood vessels from a pre-existing vasculature), using therapies that inhibit the actions of vascular endothelial growth factor (VEGF). Although anti-VEGF therapies showed some promise in early trials and are now widely used in the treatment of a variety of diseases, including cancer, the cardiovascular side effects associated with these treatments are concerning.
The clinical community now faces the issue of using these long-term approaches to treat younger populations of patients, many of whom are now at risk of complications relating to the cardiovascular consequences of treatment, rather than the cancer itself. There is clearly a need to better understand the cardiovascular effects of anti-VEGF therapies in both acute and longer-term studies.
In this webinar, Prof Jeanette Woolard discussed the issues relating to anti-VEGF approaches, giving consideration to the signalling mechanisms by which this family of pro-angiogenic proteins evoke their effects. She contextualized this by focusing on the cardiovascular consequences of VEGF inhibition in both patient and preclinical conditions. Specifically, she reviewed the use of Doppler flowmetry models to explore the short-term cardiovascular effects of small molecule inhibitors of VEGF and expand this to longer-term consequences using implantable telemetry approaches.
Key topics covered during the webinar included:
What is the role of VEGF in normal and pathological angiogenesis?
What are the consequences of VEGF inhibition?
Why is Doppler flowmetry and unique and important model for exploring acute cardiovascular effects?
Why are chronic models important?
Where should this field of research be looking to in future?
https://www.scintica.com/webinar-utilizing-doppler-flow-to-investigate-the-hemodynamic-effects-of-anti-cancer-therapies/
This presentation contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements
include statements regarding future results of operations and financial position, business strategy, product candidates, planned
preclinical studies and clinical trials, results of clinical trials, research and development costs, regulatory approvals, timing and
likelihood of success, plans and objectives of management for future operations, as well as statements regarding industry
trends. Such forward-looking statements are based on ALX Oncology’s beliefs and assumptions and on information currently
available to it on the date of this presentation.
RETINA COMPANY SHOWCASE- Aerie PharmaceuticalsHealthegy
Panel Discussion by Aerie Pharmaceuticals at OIS@ASRS 2016.
Participant:
Casey Kopczynski, Chief Scientific Officer- Aerie Pharmaceuticals
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
Antibody drug conjugates (ADCs) are undoubtedly one of the hottest research areas in recent years. To date, there are 15 approved ADCs in the world, of which 13 have been approved by the FDA, one in China (Disitamab Vedotin) and one in Japan (Akalux).
2. FORWARD LOOKING STATEMENT
This presentation contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Such statements include, but are
not limited to, statements about future expectations, plans and prospects for the
development and commercialization of the Company's product candidates,
including patient enrollment in our clinical trials, present or future licensing,
collaborative or financing arrangements, expected outcomes with regulatory
agencies, and projected market opportunities for product candidates are subject
to a number of risks, uncertainties and assumptions, including those identified
under “Risk Factors” in the Company’s most recently filed Annual Report on Form
10-K and Quarterly Report on Form 10-Q and in other filings the
Company periodically makes with the SEC. Actual results may differ materially
from those contemplated by these forward-looking statements. The
Company does not undertake to update any of these forward-looking statements
to reflect a change in its views or events or circumstances that occur after the
date of this presentation.
2
3. TARGETING AREAS OF UNMET MEDICAL NEED
Hematology
GALE-401
• Proprietary controlled release
version of anagrelide
• Targeting 3rd Line Essential
Thrombocythemia (ET)
• Phase 3 initiation expected in
Q2, 2017
Plan to develop under 505(b)2
pathway
Immunotherapy
NeuVax™ (nelipepimut-S)
• Development in key settings
Combination with trastuzumab
in HER2 1+/2+
Combination with trastuzumab
in HER2 3+
DCIS
Gastric
GALE-301/GALE-302
• Targeted development in
gynecological cancers
3
Diversified pipeline with multiple mid-to late stage clinical trials
focused in hematology-oncology
4. DEVELOPMENT PIPELINE
PRODUCT THERAPETIC AREA PHASE 1 PHASE 2 PHASE 3 BLA / NDA
Hematology
GALE-401 (Anagrelide CR) Essential Thrombocythemia
Immunotherapy: Breast Cancer
NeuVax™ + Herceptin® Node-positive or node negative/triple
negative, HER2 IHC 1+/2+
NeuVax™ + Herceptin® High risk, node-positive or negative,
HER2 IHC 3+
NeuVax™ Ductal Carcinoma in Situ (DCIS)
Immunotherapy: Gastric Cancer
NeuVax™ Gastric, HER2 IHC 1+/2+/3+
Immunotherapy: Gynecological Cancer
GALE-301 Ovarian & Endometrial
GALE-301 + GALE-302 Ovarian & Breast
*NeuVax is an investigational product. Efficacy has not been established. Herceptin is a registered trademark of Genentech.
Ongoing Planned
VADIS
4
2b
6. ANAGRELIDE
Anagrelide suppresses megakaryocytopoiesis by inhibiting PDE III-
dependent and PDE III-independent mechanisms
No DNA damaging or cytotoxic effect
Immediate release version indicated for the treatment of patients with
thrombocythemia, secondary to myeloproliferative disorders to reduce the
elevated platelet count and the risk of thrombosis and to ameliorate
associated symptoms including thrombo-hemorrhagic events
Approved to treat Myleoproliferative Neoplasms (MPNs) including Essential
Thrombocythemia (ET)
6
7. ANAGRELIDE IMMEDIATE RELEASE (IR) AEs
Related Adverse Events (AEs)
AGRYLINa
(n=942)
%
Cardiac 42
Generalc 83
Gastrointestinal 92
Respiratory, thoracic and mediastinal 18
Skin and subcutaneous tissue 14
Musculoskeletal and connective tissue 6
Nervous system 65
Vascular <5
Hepatobiliary <5
Blood and Lymphatic <5
Number of AEs/patient 3.3
a Anagrelide IR ADR data from the product label
c General AEs referred to fatigue, peripheral edema, and malaise
Most common
reasons for
discontinuation
7
Sources: Harrison et al N Engl J Med 2005;353:33-45; Mehta et al, (2014) Epidemiology of myeloproliferative neoplasms in the United
States, Leukemia & Lymphoma, 55:3, 595-600, DOI: 10.3109/10428194.2013.813500; Agrylin is a registered trademark of Shire.
Approximately 25% – 30%
of patients are intolerant
to Anagrelide IR
8. GALE-401 PHASE 1 TRIALS: CR FORMULATION
MAINTAINS PLATELET LOWERING & REDUCES Cmax
8Multiple Phase 1 studies in n=86 healthy volunteers; Agrylin is a registered trademark of Shire.
Anagrelide CR Plasma Levels
pg/mL
Anagrelide CR Platelet Lowering
GALE-401 is a proprietary, controlled release formulation of anagrelide
• Lower peak plasma concentration
• Maintain the Area Under the Curve (AUC)
9. GALE-401 DEMONSTRATES IMPROVED AE PROFILES
IN KEY CATEGORIES
Related Adverse Events (AEs)
GALE-401a
(N=18)
n (%)
AGRYLINb
(n=942)
%
Cardiac 6 (33) 42
Generalc 5 (27.8) 83
Gastrointestinal 9 (50) 92
Respiratory, thoracic and mediastinal 2 (11) 18
Skin and subcutaneous tissue 2 (11) 14
Musculoskeletal and connective tissue 1 (6) 6
Nervous system 9 (50) 65
Vascular 3 (16) <5
Hepatobiliary 2 (11) <5
Blood and Lymphatic 1 (6) <5
Number of AEs/patient 2.3 3.3
A Anagrelide CR Related AE data from the Phase 2 study; Source: Table 4, 2/12/2016
b Anagrelide IR ADR data from the product label
c General AEs referred to fatigue, peripheral edema, and malaise
9
10. GALE-401 PHASE 2 STUDY SUPPORTS
TARGETING IR INTOLERANT POPULATION
0
50
100
150
200
250
6001 2002 9001
On IR On GALE-401
Mean 7days 106 days
Median 7days 75 days
Max 7days 196 days
Min 7days 47 days
Three patients that discontinued IR were
on GALE-401 for average of 106 days
Two IR intolerant patients were on
GALE-401 for 660 days and 450 days
0
200
400
600
800
1000
1200
1400
1600
2001 6005
On GALE-401
On IR
10
11. GALE-401: TARGET PRODUCT PROFILE (TPP)
vs ANAGRELIDE IR
11
Anagrelide IR
GALE-401
TPP
GALE-401
Benefits
Therapeutic index* Narrow - dose escalation to
optimal effect is challenging
Wider
Possibility of achieving
desired effect with lower
dose
Pharmacokinetics (PK)
• Half life
• Cmax
• 2-3 hours
• 4x GALE-401
• 20 hours
• 25% of IR
Improved PK profile
Onset of Action As early as 4 weeks As early as 1 week Faster onset of action
Doses per day
2 to 4 times a day^
Label: starting at doses of 0.5-
2.0 mg every 6 hours
Target: 1 a day
2x/day based on P2
Targeting 1x/day in P3
Dosing regimen 2 to 10 mg per day^ Target: Mean 2 mg per
day
PK profile may improve
dosing regimen leading to
better patient compliance
Safety
• Related TEAEs
• # of AE/Patient
• 42.1%
• 3.3
• 30%
• 2.3
Better tolerated
*Therapeutic Index distance between therapeutic dose curve and toxic dose; ^Agrylin Package Insert
12. ESSENTIAL THROMBOCYTHEMIA (ET)
ET is a neoplastic stem cell
disorder causing dysregulated
production of large numbers
of abnormal megakaryocytes
resulting in elevated platelets
Chronic condition
• Median OS – 14.7 years
• Up to 50% of patients may be
asymptomatic at presentation
Associated with vascular
complications
• Increased risk of thrombosis and
cardiovascular events
• Patients older than 60 at greatest
risk of complications
12
Arrows:
Megakaryocytes
ET has Larger Number
of Megakayocytes
Source: Haematologica. 2009 June; 94(6): 865, Am J Hematology. 2008 May;83(5):359)
13. ESSENTIAL THROMBOCYTHEMIA (ET):
CURRENT U.S. STANDARD OF CARE
13
• Generally first line therapy for ET
• Cytotoxic Myelosuppressive drug (reduces
other blood cells as well)
• Increased risk of developing acute leukemia
after long term; avoided in younger patients
• About 25% of patients are
intolerant/refractory
• Off-label third line use
• Non cytotoxic drug
• Not used in most patients because requires
injection and is associated with flu like
symptoms
• Used mostly in pregnant women
• Generally second line therapy for ET
• Non cytotoxic drug
• Decreases platelets formation
• Not associated with increased risk of
leukemia
• Side effects: palpitations, headaches
• About one-third are intolerant to Anagrelide
IR
• Hydroxyurea and Anagrelide
Treatment Failure
Sources: Leukemia and Lymphoma Society: Essential Thrombocythemia Facts Cervantes, F. Hematology 2011; 215-221
1st Line: Hydroxyurea
2nd Line: Anagrelide IR
Interferon alpha
3rd Line: Unmet Need
14. GALE-401 ET DEVELOPMENT OPPORTUNITY
Clinical Need
Significant unmet medical need
• No approved therapies after
Anagrelide IR and Hydroxyurea
Diagnosed ET patient population
• US Prevalence: 135,000 - 175,000
• An estimated 9,000 3rd line patients in
the US
Chronic Disease
Development Path
Advantageous development and
regulatory path: 505(b) 2
Limited competition with very few
agents in development
Multiple life cycle management
opportunities
Next steps
• Finalize the Phase 3 clinical trial
design
• End of Phase 2 FDA meeting
• Initiate pivotal trial in Q2, 2017
14Sources: Harrison et al N Engl J Med 2005;353:33-45; Mehta et al, (2014) Epidemiology of myeloproliferative neoplasms in the
United States, Leukemia & Lymphoma, 55:3, 595-600, DOI: 10.3109/10428194.2013.813500
16. NEUVAX: HER2 IMMUNODOMINANT PEPTIDE
NeuVax contains the
immunodominant peptide derived
from the extracellular region of the
HER2 protein
Peptide (aa 369-377)
immunotherapy administered as
intradermal injection
MHC Class I: HLA A2/A3
16
K I F G S L A F L
17. ELICITS A STRONG CD8+ T-CELL RESPONSE
NeuVax binds to antigen
presenting cells (APCs)
NeuVax stimulates APCs to
activate CD8+ cytotoxic T
lymphocytes (CTLs)
CTLs rapidly replicate to seek out
and destroy HER2 expressing
tumor cells and micro-metastases
Booster series maintains long
term immunologic response
Demonstrated inter- and intra-
antigenic epitope spreading
17
Sources: Peoples GE, et al (2005) JCO, 23(300, 7536-7545; Mittendorf EA, et al (2006) Surgery, 139(3): 407-418. Peoples, et al, ASCO 2012 Poster Presentation
0.4
1.8
0.7
0.5
0.0
0.5
1.0
1.5
2.0
2.5
%NeuVaxspecificCD8+Tcells
NeuVax Specific CD-8 CTLs: Pre-, Post, Mean and
Long-Term (6 months)
Pre Max Mean Long-Term
19. IMMUNO-ONCOLOGY: COMBINATION STRATEGY
19
“There is growing evidence that a
multimodality approach targeting
different aspects of the immune
system may yield the greatest
clinical benefit.”
20. CORRELATION BETWEEN HER2 & MHC-1
There is an inverse
correlation between HER2
and MHC class I
HER2 overexpression is
associated with decreased
expression of components
of the antigen processing/
presentation pathway
Hypothesis:
Trastuzumab treatment will
enhance response to
vaccination by making tumor
cells more visible to T-
cells/immune system
20
21. COMBINATION IMMUNOTHERAPY
ENHANCES ANTIGEN PRESENTATION
Trastuzumab
HER2/neu
Breast
tumor cell
HER2/neu –derived peptide
presented on MHC-I
HER2/neu-
derived
peptide
Trastuzumab/HER2 complexes are internalized
and processed by proteasomes into short
peptides which are then presented on
MHC class I molecules.
Source: Mittendorf EA, et al (2006). Ann Surg Oncol;13:1085-98.
20.0
25.0
30.0
35.0
40.0
45.0
50.0
55.0
60.0
Average%Cytotoxicity51Cr
0 ug 10 ug 50 ug
* p=0.015
22
Trastuzumab
PBMC from HER2/neu peptide, E75,
vaccinated patients efficiently recognize
and lyse trastuzumab-treated HER2/neu-
expressing tumor cell lines
22. Interim
Analysis
at 1 Year
DFS
Standard of Care: Standard Herceptin
dosing every 3 weeks for 1 year
6 doses of NeuVax given every 3 weeks
starting with third dose of Herceptin
+ 1 booster
dose every
6 months
thereafter
+ Dosing to disease
progression;
36 mo follow up
Primary
Endpoint
DFS at
24 mos.
300 adjuvant breast cancer
patients, randomized 1:1
Single blind (subject)
Node positive or high risk
node negative
HLA A2/A3+
HLA A24/A26+
HER2 IHC 1+/2+
Stratified by nodal status
and HER2 status
Study Population
NEUVAX+TRASTUZUMAB:
HER2 1+/2+ PHASE 2 STUDY
GM-CSF
+ GM-CSF
22
23. NEUVAX: CURRENT CLINICAL TRIALS
Phase Treatment
HER2
Status
Indication Trial Status
Protocol
Defined
# of Patients
Collaborations
2b
Combination
with
trastuzumab
1+, 2+
BREAST
Node Positive or High
Risk Node Negative
HLA A2+, A3+,
A24+, A26+
Enrolling
U.S. only
33 centers
300
2
Combination
with
trastuzumab
3+ high
risk
BREAST
Node Positive
HLA A2+, A3+
Enrolling
U.S. only
28 centers
100
2
Single agent
VADIS Study
1+,
2+,3+
BREAST
Ductal Carcinoma in
Situ (DCIS)
HLA A2+
Suspended
U.S. only
4 centers
48
2 Single agent
1+,
2+,3+
GASTRIC
HLA A2+, A3+
Planned
India Only
50
23
25. GALE-301 & GALE-302
25Source: U.S. Ovarian Cancer http://seer.cancer.gov/statfacts/html/ovary.html
Targeted cancer
immunotherapy
Folate Binding Protein (FBP)
is over-expressed (20-80 fold)
in >90% of ovarian and
endometrial cancers
FBP has very limited tissue
distribution and expression in
non-malignant tissue making it
an ideal immunotherapy target
Current treatments are generic
• Carboplatin and paclitaxel
• High recurrence rate
Most patients relapse with
poor prognosis
26. GALE-301: OPTIMAL DOSE GROUP SHOWS
PRELIMINARY EFFICACY
Source: Peoples, et. al, Poster Presentation, American Society of Clinical Oncology 2016
26
Phase 1/2a trial ongoing
Phase 1: Determined optimal dose and
demonstrated safety and potent immune
response
Phase 2a Preliminary data:
• At 16 months median follow-up:
Overall recurrence rate was 44.8% in
the VG versus 54.5% in the CG
(p=0.58),
Recurrence rate of 23.5% in patients
who received booster inoculations.
• Two year DFS estimate in 1000 mcg dose
group is 73.5% vaccine vs. 38.1% control
(p=.03)
• GALE-301 plus GM-CSF is well tolerated
and elicits a strong in vivo immune
response with primarily Grade 1 and
Grade 2 toxicities
Estimated 24 months Disease Free Survival by
Dosing Cohort
28. LEADERSHIP TEAM
28
Mark W. Schwartz, Ph.D.
President & CEO
Apthera, Bayhill Therapeutics, Calyx
Therapeutics, Trega Biosciences, Incyte
Genomics, DuPont Diagnostics
Bijan Nejadnik, M.D.
Executive VP, Chief Medical Officer
Jazz Pharmaceuticals, Johnson & Johnson,
Stanford, Johns Hopkins, UC Davis
Remy Bernarda,
SVP, Investor Relations & Corporate
Communications
IR Sense, Hana Biosciences, Knight Equity
Markets, Bear Stearns, Goldman Sachs
John Burns, CPA
VP, Finance & Corporate Controller
Pixelworks, Moss Adams
Tom Knapp, Esq.
Interim General Counsel
Sucampo, Exemplar Law Partners,
NorthWestern Energy, Paul Hastings, The
Boeing Company
Joe Lasaga
VP, Business Development & Alliance
Management
Nektar Therapeutics, Rigel
Pharmaceuticals
29. FINANCIAL OVERVIEW
Cash Position (as of June 30, 2016) $19.6 million
Financing (July 13, 2016) + $11.7 million
Debt Financing (Amended Aug 22, 2016) + $24 million ($18.5M restricted cash)
Projected Quarterly Burn
Q3, 2016 $12 - $13 million
Q4, 2016 $8 - $10 million
Shares Outstanding (as of July 31, 2016) 214 million
Market Cap (as of September 20, 2016) ~$80 million
29
30. MILESTONES
30
PROGRAM MILESTONE
PROJECTED
DATE 2016
PROJECTED
DATE 2017
GALE-401
(anagrelide CR)
Present combined P1 & P2 safety data ✓
Confirmation of 505(b)2 pathway 2H
Phase 3 Initiation Q2
Phase 3 Dose Verification Q4
NeuVax™
(nelipepimut-S)
Fast Track Designation ✓
Initiate DCIS trial Q4
Combo H&N 1+/2+ Interim safety data Q4
Combo H&N Enrollment Complete Year-end
Combo H&N Interim Efficacy Q4
GALE-301
GALE-302
Present 301/302 booster data ✓
Present GALE-301 Phase 2a primary analysis ✓
Orphan Drug Designation ✓
Present GALE-301 Biomarker & Dosing Data Q4