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8-K - HUMANIGEN, INC | s2141708k.htm |
Exhibit 99.1
Advancing Medicines for Neglected and Rare Diseases Through Innovative Business Models OTC: KBIO www.kalobios.com | Corporate Overview 1
Forward-Looking Statements | Corporate Overview 2 This presentation contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct, and actual results could differ materially from the forward-looking statements.Words such as “will,” “expect,” “intend,” “plan,” “predict,” “potential,” “possible,” and similar expressions identify forward-looking statements, including, without limitation, statements related to the scope, progress, expansion, and costs of developing and commercializing the Company’s product candidates; opportunity to benefit from anticipated regulatory incentives for product candidates; and anticipated expenses related to development activities, clinical trials and the development and potential commercialization of product candidates.Forward-looking statements are subject to risks and uncertainties including, but not limited to, the Company’s lack of revenues, history of operating losses, limited cash reserves and ability to obtain additional capital to develop and commercialize its product candidates, including the additional capital which will be necessary to complete the clinical trials that the Company has initiated or plans to initiate, and continue as a going concern; the Company’s ability to execute its revised strategy and business plan; the ability of the Company to list its common stock on a national securities exchange, whether through a new listing or by completing a reverse merger or other strategic transaction; the availability of a 505(b)(2) development pathway for the potential approval by FDA of the Company’s benznidazole candidate as a treatment for Chagas disease remaining acceptable to FDA in the future; the fact that a 505(b)(2) pathway does not assure a product candidate will be deemed safe or effective, or that FDA approval will be obtained; the requirement that the Company be first to receive FDA approval for benznidazole as a treatment for Chagas disease as a prerequisite to the Company’s ability to apply for or receive a Priority Review Voucher in respect of that candidate; the potential timing and outcomes of clinical studies of benznidazole, lenzilumab, ifabotuzumab or any other product candidates and the uncertainties inherent in clinical testing; the commercial viability of the Company’s proposed drug pricing program; the ability of the Company to timely source adequate supply of its development products from third-party manufacturers on which the Company depends; the potential, if any, for future development of any of its present or future products; the Company's ability to successfully progress, partner or complete further development of its programs; the ability of the Company to identify and develop additional products; the Company's ability to attain market exclusivity or to protect its intellectual property; competition; changes in the regulatory landscape that may prevent the Company from pursuing or realizing any of the expected benefits from the various regulatory incentives at the center of its strategy, or the imposition of regulations that affect the Company's products; and the various risks described in the "Risk Factors" and elsewhere in the Company's periodic and other filings with the Securities and Exchange Commission.You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this presentation. The company has no obligation, and expressly disclaims any obligation to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.
Turnaround focused on high-value neglected and rare diseases, including pediatricPipeline with multiple potential near- and longer-term catalystsBenznidazole: expected 505(b)(2) development pathway for Chagas disease; possible PRVLenzilumab: recombinant mAb; possible PRVIfabotuzumab: recombinant mAb; pediatric and orphan blood and solid cancers; possible PRV; potential for partneringExperienced, focused new management with track record of efficient execution, commitment to leadership in responsible, transparent pricing Compelling Investment Opportunity | Corporate Overview 3
Leadership Cameron Durrant, MD, MBAChairman and CEOSenior pharmaceutical and biotech exec, turnaround specialistSenior exec roles at Pharmacia/Pfizer, J+J in US, Merck, GSK in Europe; experience as Exec Chairman, CEO and CFO; CEO roles at three specialty pharma groupsExpertise in anti-infectives, pediatrics, oncologyMorgan LamChief Scientific OfficerExtensive industry experience in clinical researchHead of Clinical Operations and Development KaloBiosExecutive Director, Medical Affairs, GeronDave Tousley, MBA, CPAInterim Chief Financial Officer More than 35 years experience in biotech, spec pharma, big pharmaSenior exec roles, President, COO, CFOPasteur, Merieux, Connaught, AVAX, airPharma, PediaMed, DARA Biosciences Tariq Arshad, MD, MBAConsultant focused on medical affairsExtensive industry experience in clinical developmentExperienced in orphan, pediatrics, oncology, pediatric oncology, immunologyPharmacia/Pfizer, Genentech, XomaNiv Caviar, MBAConsultant focused on corporate/business developmentSenior functional roles in marketing, business development, strategic planningSenior exec roles, CEO, EVP-CBO, CFO, VP Bus DevLa Jolla Pharma, Allergan, Suneva, SpineOvations, Affymetrix, AccentureChristopher BoweConsultant focused on corporate affairsDeep experience advising CEOs on articulating, executing strategy through corporate affairsFormer Strategic Affairs advisor at Schering-PloughIndustry thought leader, prior award-winning writer Financial Times Steve Pal, MBAConsultant focused on commercialGlobal pharma and consumer healthcare product commercialisationFormer Corporate VP Global Strategic Marketing, Health Outcomes, Strategy and Research, Global Medical Affairs, Allergan | Corporate Overview 4
Board of Directors Cameron Durrant, MD, MBAChairman | Corporate Overview 5 Ronald Barliant, JDOf Counsel at Goldberg Kohn, extensive experience representing debtors and creditors in complex bankruptcy casesUnited States bankruptcy judge for the Northern District of Illinois 1988 to 2002Dale Chappell, MD, MBAManaging member of Black Horse Capital Management, LLCPreviously associate at Chilton Investment Company specializing in healthcareTimothy Morris, CPACFO AcelRx Pharmaceuticals; also Head of Business DevelopmentCFO, SVP Finance and Global Corporate Development VIVUS, Inc.Ezra FriedbergFounder and general partner of Multiplier Capital, LP>20 years investing experience across public and private companies
Two-Pronged Value Model: a Unique Approach To Near-Term Return Propelling Long-Term Value Creation | Corporate Overview 6 Smart Investment in underappreciated assets Leverage FDA incentives for better efficiency Value Options From multiple FDA catalysts like PRV Value Creation Potential robust commercial return HuntLate-stage, neglected, rare/orphan assets NEAR-TERM LONG-TERM
Priority Review Vouchers (PRVs) Create Options For Significant Potential Return | Corporate Overview 7 Range of disclosed sale prices for PRVs is $67.5MM - $350MM Rare Pediatric Diseases (RPD) Neglected Tropical Diseases (NTD) Holder of PRV can receive priority review for any NDA/BLA PRV can be sold to company seeking a competitive jumpKaloBios open to novel potential PRV transaction structures
| Corporate Overview 8 Funding dead-end, winding down Oct 2015 Martin Shkreli investment late Nov 2015 Shkreli arrest, indictment Dec 2015 Chapter 11 filing Dec 2015 Delisted by Nasdaq Jan 2016 Two new board members Jan 2016 New CEO Mar 2016 Announced Responsible Pricing Model April 2016 Exit from Chapter 11 bankruptcy June 30, 2016 Lenzilumab Phase 1 start July 2016 Shkreli governance agreement July 2016 Shkreli stake extinguished Aug 2016 Current with SEC filings Sept 2016 San Francisco R&D company, mAb platform FDA meeting Dec 2016 and positive minutes Jan 2017 KaloBios Turnaround Momentum Builds: Rising from the Ashes
Current Pipeline | Corporate Overview 9 Pre-clin Phase I Phase II Phase III NDA/BLA Benznidazole (potential PRV)Chagas diseaseAnti-infective LenzilumabCMML Anti-GM-CSF mAb Lenzilumab (potential PRV)JMML (peds) – dependent on interim data in CMMLAnti-GM-CSF mAb Ifabotuzumab (potential PRV)Solid (peds) tumorsAnti-EphA3 mAb Partnering potential Intend to seek approval pursuant to 505(b)(2) pathway
Benznidazole a potential treatment for Chagas disease | Corporate Overview 10
Neglected tropical disease presenting in the US and other nations On FDA list of NTDs eligible for PRVCurrently no FDA-approved treatmentsImmediate need for reliable benznidazole supply in US and abroad CHAGAS disease | Corporate Overview 11 Insect carries the parasite called Trypanosoma cruzi Infected individuals in US* Can also affect swallowing, digestion and cause neurological complications 300,000 * Source: CDC A chronic, potentially life-threatening infectious disease spread by contact with feces of an infected triatomine insect, called “kissing bugs” KEY TAKEAWAYS Significant portion of chronic patients progress to serious heart illness
Benznidazole: the Preferred Treatment Globally for Chagas | Corporate Overview 12 N N HN N
Benznidazole Strategy, Development Plan On Track | Corporate Overview 13 Positive FDA Guidance 505(b)(2) Pathway PRV Eligibility Confirmed Progress to Submission Received minutes from productive meeting Acceptable to FDAExpect no clinical efficacy or safety studies Currently expected to be eligible if approved for Chagas disease Expect to progress expeditiously toward a submission
2016 2017 H1 H2 H1 H2 Manufacturing (CMC) Analytical methods development Drug substance Drug product Stability studies Non-Clinical (Non-Human) Metabolism / PK Toxicity assessment T.cruzi biology Environmental assessment Clinical Summary of relevant clinical studies Right of Reference & data extraction for studies Human bioavailability (BA) Study Regulatory Pre-IND meeting & open IND Orphan drug status application Pediatric plan Pre-NDA meeting Assembly of NDA & NDA filing Benznidazole Target Approval Plan | Corporate Overview 14 Pre-IND meeting IND submission NDA filing 2018
Lenzilumab a potential pipeline in a product | Corporate Overview 15
High unmet needPatients typically unsuitable for stem cell transplant40-90% patients show hypersensitivity to GM-CSF CMML overview | Corporate Overview 16 Recently classified as separate disease with distinct natural history* Median overall survival rate in months Newly diagnosed US patients per year Age at diagnosis ~1,100 * Formerly classified as subtype of the myelodysplastic syndromes (MDS) 60+ a rare hematologic cancer 20 KEY TAKEAWAYS
No FDA-approved treatmentClear unmet needPotential for a rare pediatric disease PRVGM-CSF hypersensitivity is hallmarkPediatric oncology largely ignored in clinical development JMML overview | Corporate Overview 17 Event-free survival rate at 5 years (with bone marrow transplant) New US cases per year Age of majority of patients at diagnosis is 4 years or younger ~420 very rare, frequently lethal pediatric leukemia ~52% KEY TAKEAWAYS
Lenzilumab is a Potent Inhibitor of GM-CSF Function In Vitro | Corporate Overview 18 Inhibits GM-CSF-dependent Proliferation of Human TF-1 Leukemia Cells Blocks GM-CSF-induced STAT5 Phosphorylation in Human TF-1 Leukemia Cells Source: KaloBios laboratory notebook STAT5 phosphorylation detection in TF-1 Cells by Flow Cytometry Inhibition of STAT5 phosphorylation by lenzilumab in TF-1 Cells (+ 2 ng/mL GM-CSF) 0.001 0.01 0.1 1 10 Lenzilumab (µg/mL) 25 20 15 10 0 5 40 30 20 10 0 MFI 0.01 0.1 1 10 100 GM-CSF (ng/mL) Lenzilumab 0.1 1 10 100 1000 lgG (pM) 100 50 0 Lenzilumab Control antibody % Growth Source: KaloBios pre-clinical study MFI = mean fluorescence intensity
Lenzilumab: a Pipeline in a Drug Opportunity | Corporate Overview 19 JMML ALPS / RALD Cytokine Release Syndrome AMML RASopathies (e.g. CFC syndrome, Costello syndrome) Noonan Syndrome with PTPN11 CMML CML Plexiform Neurofibroma Pediatric Multiple Sclerosis US ADDRESSABLE POPULATION 325 350 500 600 700 800 1,900 5,800 24,000 40,000
Ifabotuzumab potential for partnering and development | Corporate Overview 20
1 2 3 4 Ifabotuzumab Has Four Postulated Mechanisms of Action | Corporate Overview 21 ADCC Mediated Killing of Tumor Cells Direct Apoptosis of Tumor Cells Disruption of Tumor Vasculature through Cell Rounding Anti-fibrotic Effect Ifabotuzumab Ifabotuzumab Ifabotuzumab Ifabotuzumab Humaneered® monoclonal antibody for Ephrin type-A receptor 3 (EphA3)
Capitalization Summary as of 2/14/17 | Corporate Overview 22 Debt $4.3 million Common Shares Outstanding 14.9 million Stock Options (WAEP $5.82) 2.1 million Warrants (WAEP $14.24) 0.3 million Fully Diluted Shares Outstanding 17.3 million Note: share and per share amounts as of 9/30/16 – last 10-Q filing Debt includes term loan financing closed on 12/21/16
Demonstrated History of Swift Execution in Challenging Circumstances | Corporate Overview 23 JUN JUL AUG SEP DEC Emerge in six months from complex Chapter 11 bankruptcy Lenzilumab Phase 1 trial started Extinguished Martin Shkreli’s stake in the company Current with Securities and Exchange Commission periodic reporting requirementsAssembled and filed three 10-Qs and a 10-K within three months Productive FDA meeting on benznidazole 30 24 29 23 6 2016
Summary Unique near-term/long-term potential value creation opportunity Thoughtful business strategy that leverages existing U.S. regulatory and development incentivesAttractive asset portfolio with multiple potential milestones and partnering prospectsDeeply focused new management team with an unwavering commitment to success | Corporate Overview 24