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First self-administered antibody therapy for HIV in late-stage clinical trials
Investor Presentation
March 2017
(OTCQB: CYDY) 1 www.cytodyn.com
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(OTCQB:CYDY)
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Forward-Looking Statements
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This presentation includes forward-looking statements and forward-looking information within the meaning of United States securities laws. These statements and information represent CytoDyn’s intentions, plans, expectations and beliefs, and are subject to numerous risks, uncertainties and other factors, of which many are beyond CytoDyn’s control. These factors could cause actual results to differ materially from such forward-looking statements or information. The words “believe,” “estimate,” “expect,” “intend,” “attempt,” “anticipate,” “foresee,” “plan,” and similar expressions and variations thereof, identify certain of such forward-looking statements or forward-looking information, which speak only as of the date on which they are made.
CytoDyn disclaims any intention or obligation to publicly update or revise any forward-looking statements or forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable law. Readers are cautioned not to place undue reliance on these forward-looking statements or forward-looking information. While it is impossible to identify or predict all such matters, these differences may result from, among other things, the inherent uncertainty of the timing and success of and expense associated with research, development, regulatory approval, and commercialization of CytoDyn’s products and product candidates, including the risks that clinical trials will not commence or proceed as planned; products appearing promising in early trials will not demonstrate efficacy or safety in larger-scale trials; future clinical trial data on CytoDyn’s products and product candidates will be unfavorable; funding for additional clinical trials may not be available; CytoDyn’s products may not receive marketing approval from regulators or, if approved, may fail to gain sufficient market acceptance to justify development and commercialization costs; competing products currently on the market or in development may reduce the commercial potential of CytoDyn’s products; CytoDyn, its collaborators or others may identify side effects after the product is on the market; or efficacy or safety concerns regarding marketed products, whether or not scientifically justified, may lead to product recalls, withdrawals of marketing approval, reformulation of the product, additional pre-clinical testing or clinical trials, changes in labeling of the product, the need for additional marketing applications, or other adverse events.
CytoDyn is also subject to additional risks and uncertainties, including risks associated with the actions of its corporate, academic, and other collaborators and government regulatory agencies; risks from market forces and trends; potential product liability; intellectual property litigation; environmental and other risks; and risks that current and pending patent protection for its products may be invalid, unenforceable, or challenged or fail to provide adequate market exclusivity. There are also substantial risks arising out of CytoDyn’s need to raise additional capital to develop its products and satisfy its financial obligations; the highly regulated nature of its business, including government cost- containment initiatives and restrictions on third-party payments for its products; the highly competitive nature of its industry; and other factors set forth in CytoDyn’s Annual Report on Form 10-K and other reports filed with the U.S. Securities and Exchange Commission.
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CytoDyn and the HIV Landscape
Late-stage development of antibody for HIV therapies (two Phase 3 trials underway)
Large market of approximately $20 billion(1)
Big players with significant market share (2015)(2)
Gilead 56%
GSK 16%
BMS 12%
JNJ 9%
Merck 8%
CytoDyn’s PRO 140 offers strong and compelling differentiators for the market and its
1.3 million (3) patients (US only)
Safe
Efficacious
Minimal toxicity
Minimal side effects
Ease of compliance
Source: (1) http://www.transparencymarketresearch.com/hiv-market.html
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Compilation from MedAdNews, Aug. 2016 from 2015 annual reports |
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GlobalDataEpiCastSeptember 2015 |
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HIV is a virus called Human Immunodeficiency Virus
Once HIV is transmitted, it can only survive by entering T-cells
HIV half life = 24 hours
Viral Load (VL) is number of virus (HIV) particles per mL of blood
IfVL<40 copies/mL then the Transmission Rate ~ Zero (1)
High viral load is a major risk factor for transmission of HIV
Effective antiretroviral therapy can reduce transmission of HIV by more than 96% (1)
Treatment options (HAART –Current standard of care)
HIV replication process is called HIV life cycle and has 7 stages
Different classes of antiviral drugs are designed to stop HIV at different stages of HIV life cycle
HAART is comprised of 3 drugs from 2 classes –Today’s HIV drugs are oral pills
Problems with HAART
Compliance
Side Effects
Toxicity
Source: (1) https://www.hivlawandpolicy.org/sites/www.hivlawandpolicy.org/files/Undetectable%20Blood%20Viral%20Load%20and%20HIV%20Transmission%20Risk%20-%20Results%20of%20a%20Systematic%20Review%20(CATIE).pdf
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PRO 140 Advantages vs. Currently Approved Therapies
* The prevalence of antiretroviral drug resistance in the United States. http://www.ncbi.nlm.nih.gov/pubmed/15199315
PRO 140
HAART No drug resistancein patients on monotherapy for ~24 months 76% of patients have resistanceto 1 or more drugs*
Once-a-week, simple, painless, sub-cutaneous injections Lifelong adherence with only 30% of patients achieving suppressed viral load
No negative impact on immune function
No serious adverseevents (SAEs) Incomplete recoveryof immune function
No serious side effectsseen in 200 patients in more than 7 clinical trials Toxicityranges from mild to severe. Many problems with long-term toxicity.
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PRO 140 –A Monoclonal Antibody that Binds to theHIV Entry Receptor -CCR5
Humanized monoclonal antibody
Binds to CCR5 co-receptor on white blood cells
Blocks HIV entry into white blood cells
PRO 140
CCR5
CD4
Cell
HIV6
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PRO 140 Viral Load Reduction in >200 HIV Patients (7 clinical trials)
Subcutaneous Administration
First proof of concept for a long-acting,
self-administrable weekly HIV drug
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PRO 140 Overview
FDA Fast Track designation
Currently under development in combinationwith HAART
and for an alternative to HAART
Development of PRO 140
PRO 140 evaluated in 7 clinical trials with >200 patients
Development of PRO 140 for treatment of HIV-R5 strain
Viral load drop as much as 2.5 log (>300 fold) with a single dose
Very low toxicities or side effects
No resistance observed in clinical trials
HIV R5 strain represents 90% of newly diagnosed patients and ~70% overall
Ongoing Phase 2b monotherapy extension study has exceeded 2 years of viral suppression
Phase 2b/3 Pivotal Trial –30-patient study for combination therapy w/HAART
Phase 2b/3 Investigative Trial –300-patient study for long-term monotherapy
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PRO 140 for HIV: Clinical Trial OverviewTrial Stage
Study # patients Design / Findings Status P-Cl. Ph1 Ph2 Ph3
2 Phase 1 studies 54 Healthy patients, no safety concerns Complete
1302 IV Phase 1 study 39 Intravenous, single-dose VL reduction for
3 weeks Complete
2301 IV Phase 2 study 31 Intravenous, single-dose VL reduction for
3 weeks Complete
2101 SC Phase 2 study 44 Subcutaneous,long-acting, self-administered, proof-of-concept shown Complete
CD01 Phase2b 40 12-week drug-substitution monotherapy
Long-termmonotherapyextension: 14 patients with VL suppression at 12 weeks Complete Jan. 2015 Ongoing
CD02 Phase 2b/3Pivotal-Fastest path to approval 30 Combination therapy in HAART failures,
1 week efficacy + 24 weeks durability Injectionof 1stpatientOct.2015
CD03 Phase 2b/3 InvestigativeTrial –
Largest market size 300 Long-term monotherapy Injection of 1st patients Dec. 2016
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Phase 2b Monotherapy Trial –PRO 140 Substitution for HAART
40 patients previously stable on daily oral combination antiretroviral therapy
Trial completed in January 2015
No drug-related serious adverse events
Evaluate the efficacy, safety and tolerability of PRO 140 monotherapy for the maintenance of viral suppression
Shifted to PRO 140 monotherapy; weekly subcutaneous injections for up to 12 weeks Trial Results: Suppressed VL
Time of
Evaluation
PRO 140 Monotherapy
TreatmentInterruption Study Data(1)
4 weeks
98%
50%
11 weeks
75%
0%
After11 weeks, 21 patients were qualified for extension
Patient failures due to infections unrelated to PRO 140 were excluded
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Viral load measured with enhanced, single RNA copy assay
7 patientsViral load < 1 cp/mL
1 patient Viral load ~ 2 cp/mL
1 patient Viral load ~ 3 cp/mL
1 patient Viral load ~ 40 cp/mL
10 patients analyzed with assay capable of detecting a single virus
Successful Monotherapy for over 2 years
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Phase 2b/3 Investigative Monotherapy Trial
Protocol cleared by FDA; first of several patients treated in Dec. 2016
300 patients well-controlled by HAART at up to 30 clinical sites
PRO140toreplaceHAART
48-weekInvestigativetrial
Failure criteria: viral suppression of >200 cp/mL blood two consecutive weeks
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Phase 2b/3PivotalCombination Therapy Trial
First path for approval for PRO 140
To enroll 30 treatment-experienced HIV patients poorly controlled by HAART therapy. About 40 clinical sites have been cleared for enrollment Injected the 1st patient in Oct. 2015 Primary endpoint expected in 1H17: reduction in HIV viral load from baseline (0.5 log in 1 week) Secondary endpoints: Tolerability and Safety (24 weeks) Safety data to be generated from concurrent Phase 2b/3 Investigative monotherapy trial
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PRO 140 Clinical and Regulatory Strategy (HIV) Phase 2b/3 Pivotal
Combination Therapy Phase 2b/3 Investigative
Monotherapy
1st patient injected in October 2015 1stpatients injected in December 2016
Several patients concludedand are now in a rollover study due to request to receive continued access to PRO 140 48-week investigative trial
Clinical trial for first approval
underway Supported by long-term viral load reductions in ongoing Phase 2b extension study
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PRO 140 for Immunologic Indications (Non-HIV)
The target of PRO 140 is CCR5, the receptor for the chemokine responsible for immune cell trafficking, T-cell migration to sites of inflammation
PRO 140 binds to CCR5 without triggering migration signals and exhibits no agonist activity when it binds (no direct stimulation) PRO 140 is a competitive inhibitor of molecules that trigger CCR5 immunologic activity
CCR5 triggering plays a crucial role in inflammatory responses: Regulation of cancer cell killing Transplantation rejection reactions
Autoimmunity
Chronic inflammation
Animal models (mice) for these diseases can be used since PRO 140 recognizes human and mouse CCR5 equivalently
The transplantation reaction, GvHD, is our first non-HIV indication for clinical investigation
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Transplantation Indication – GvHD—Phase 2b
GvHD: Graft versus Host Disease, a rejection reaction by the transplanted bone marrow (BM) stem cells producing a life-threatening reaction against the patient
Bone marrow transplant patients (AML & MDS patients) have poor survival rate due to GvHD
CCR5 has been implicated as a central feature in GvHD and positive data in an animal model is being published PRO 140 has potential to reduce the severity of GvHD and expand the patient pool for BM transplantation
FDA approved protocol: randomized, double-blind, placebo-controlled, multicenter 100-day study Enrollment of 60 BM transplant patients with AML or MDS undergoing BM stem cell transplantation Evaluate safety and efficacy of PRO 140 for prophylaxis of acute GvHD
1st patients expected to enroll in 2H17
(OTCQB: CYDY) 16 www.cytodyn.com
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Experienced Management Team
Anthony Caracciolo, Executive Chairman: Former Gilead Senior Vice President of Manufacturing and Operations, member of Gilead executive committee, over 30 years of executive and senior leadership in the pharmaceutical sciences industry.
Nader Pourhassan, Ph.D., CEO: Led the development pathway for PRO 140 and was instrumental in leading the Company through several rounds of financing.
Michael Mulholland, CFO: Financial executive with 30 years of senior financial leadership with public companies in several industries. Experienced in strategic planning, corporate finance and M&A.
Denis Burger, Ph.D., Vice Chairman, CSO: Former academic Immunologist, successful biotech CEO, experienced with public company financing
Tom Boyd, Ph.D., BLA Team Leader: Formerly SVP Product Development, Progenics Pharmaceuticals Inc.; ex-Boehringer Ingelheim, Wyeth and Alteon. Experienced in project leadership and nonclinical drug development of small molecules and biologics from discovery through registration.
Nitya Ray, Ph.D., SVP-Manufacturing & CMC Team Leader: Formerly SVP Manufacturing, Progenics Pharmaceuticals Inc.; ex-Hoffmann-La Roche and Ortec International. Experienced in process development, manufacturing and quality control of biopharmaceutical, small molecule and radiopharmaceutical drugs.
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World-Class HIV Expert Advisors
Robert Schooley, MD
Paul Maddon, MD, PhD
Daniel Kuritzkes, MD
Professor of Medicine, Chief of Division of Infectious Diseases, Academic Vice Chair Department of Medicine, University of California, San Diego
An inventor of PRO 140; discovered CD4 and CCR5 interaction with HIV, Trustee of Columbia University
Professor of Medicine at Brigham & Women’s Hospital, Infectious Disease/Partners AIDS Research Center, Infectious Diseases Professor at Harvard Medical School, Principal Investigator and Chair of AIDS Clinical Trials Group (ACTG), Associate Editor for Journal of Infectious Diseases
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Potential Addressable U.S. HIV Markets for PRO 140
~$1b
~$11b
~$0,5b Projections for 2018
Combination Therapy population size Monotherapy population size
2ndline therapy* 350,000 Patients with VL<40 cp/mL 411,380
3 classes of resistance 20,000 to 25,000
* 2ndline therapy is for patients who are onsecond HIV drug regime and primarily have one drug resistance.
Sources:(GlobalData.com –CDC.gov –AIDS.gov)
Assumes that the total HIV population in US (2018) ~ 1.365 million patients
Assumes 67% of patients with R5 strain
CombinationTherapy:
Assumes that 20,000 –25,000 patients have at least 3 drug classes of resistance
Total number of patients on HAART = 1.365 million x 0.57 ~ 778,100 patients
Assumes 45% as 2nd-line orhigher which yields: ~ 778,100 patients x 0.45 ~ 350,000patients
Monotherapy:
Assumes that patients with VL<40 cp/mL = 40% x 1.365 million = 614,000 patients
Patientsfor monotherapy population = 67% x 614,000 = 411,380 patients
Source: https://www.cdc.gov/hiv/pdf/library/reports/surveillance/cdc-hiv-surveillance-report-2000-vol-12-1.pdf
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Upcoming Milestones
ODD: Orphan Drug Designation
BTD: Breakthrough Therapy DesignationEvents 1H 2017 2H 2017 2018
Phase 2b/3 Combination Therapy PIVOTAL Trial Primary Endpoint BTD ODD Submit BLA
Phase 2b/3 Monotherapy Investigative Trial 100 patients enrolled for safety arm of Combination Trial for BLA 300 patient enrollment completed Pre-BLA meeting with FDA for label expansion
Phase 2b Monotherapy extension arm 2 publications Presentation at ASM
Phase 2 GvHD 1st patient treated ODD
Non-HIV indications 3 publications
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Investment Highlights
U.S. market size for HIV therapies is about $20 billion (1)
Only 56% on HAART (US only)
PRO 140 addresses HAART shortcomings: No serious side effects or toxicities No drug resistance,
Once-weekly, self-administered subcutaneous injection addresses compliance issues
Phase 2b/3 Pivotal combination therapy trial underway –Primary Endpoint results expected in 1H17 – several patients successfully completed and now in rollover study due to their request to receive continued access to PRO 140
Phase 2b/3 Investigative monotherapy trial – first several patients treated in Dec. 2016
Pipeline opportunities for Non-HIV indications: transplantation, autoimmunity, cancer and chronic inflammation Phase 2 clinical trial underway for GvHD
Source: (1) http://www.transparencymarketresearch.com/hiv-market.html
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