UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of The Securities Exchange Act of 1934
Date of Report (Date of Earliest Event Reported): January 8, 2018
Aerpio Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)
| DELAWARE | 000-53057 | 61-1547850 | ||
| (State or other jurisdiction of incorporation) |
(Commission File Number) |
(I.R.S. Employer Identification No.) | ||
| 9987 Carver Road Cincinnati, OH |
45242 | |||
| (Address of principal executive offices) | (Zip Code) | |||
Registrant’s telephone number, including area code: (513) 985-1920
Not Applicable
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☒
| Item 7.01. | Regulation FD Disclosure. |
Aerpio Pharmaceuticals, Inc. (the “Company”) is furnishing a corporate presentation, attached as Exhibit 99.1 to this Current Report on Form 8-K, which the Company intends to use from time to time in meetings with investors and others beginning on January 8, 2018. The corporate presentation will also be available in the investor relations section of the Company’s website at http://aerpio.com.
The information in this Item 7.01 and Exhibit 99.1 attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities and Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section, nor shall they be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, except as expressly set forth by specific reference in such filing.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits
| Exhibit No. |
Description | |
| 99.1 | Aerpio Pharmaceuticals, Inc., corporate presentation. | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Date: January 8, 2018 | AERPIO PHARMACEUTICALS, INC. | |||||
| By: | /s/ Stephen Hoffman | |||||
| Stephen Hoffman | ||||||
| Chief Executive Officer | ||||||
Exhibit 99.1
aerpio pharmaceuticals
Forward looking statement This presentation has been prepared by Aerpio Pharmaceuticals (“we”, “us” or, the “Company”), solely for information purposes and is for the recipient to familiarize itself with the Company, and the fact that this meeting has taken place and anything you hear or learn during this meeting are strictly confidential. By agreeing to attend this meeting, you agree to keep all such information confidential. Except for internal use, this information may not be excerpted from, summarized, distributed, reproduced or used without the prior written consent of the Company. This presentation does not constitute an offer or invitation for the sale or purchase of securities. The Company does not make any representation or warranty, express or implied, as to the accuracy or completeness of the information contained herein and shall not have any liability for such information. Interested parties should conduct their own investigation and analysis of the Company, its business, prospects, results of operations and financial condition. This presentation includes forward-looking statements. All statements contained in this presentation other than statements of historical facts, including statements regarding our product candidates, their therapeutic potential and development plans, our future results of operations and our financial position, our business strategy and plans and our objectives for future operations, are forward-looking statements. Forward-looking statements speak only as of the date hereof unless it is stated otherwise. Although we believe that the expectations reflected in these forward-looking statements are reasonable, these statements relate to our strategy, our intellectual property position, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements including those contained in our public filings with the Securities and Exchange Commission. This presentation also contains estimates and other statistical data made by independent parties and by us. Management bases all estimates and projections as to events that may occur in the future (including projections of revenue, development plans and timing of clinical trial results) upon their best judgment as of the date of this presentation. Whether or not such estimates or projections may be achieved will depend upon the Company achieving its overall business objectives and the availability of funds. The Company does not guarantee that any of these projections will be attained. Actual results will vary from the projections, and such variations may be material. New risks emerge from time to time, and except as required by law, neither we nor any other person makes any representation as to the accuracy or completeness of such data or undertakes any obligation to update such data after the date of this presentation. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. By attending or receiving this presentation you acknowledge that you will be solely responsible for your own assessment of the market and our market position and that you will conduct your own analysis and are solely responsible for forming your own view of the potential future performance of our business. 2
Corporate highlights Advancing first-in-class treatments for ophthalmic disease Non-proliferative diabetic retinopathy (NPDR) Primary open angle glaucoma (POAG) AKB-9778 is the most potent activator of the Tie2 pathway, proven essential for vascular stability Phase 2b DR data expected Q2 2019 Proof-of-concept in DR demonstrated in Phase 2a DME study Approximately 13 million DR patients in the US and 20 million in EU Multi-billion dollar market potential Strong rationale for efficacy in primary open angle glaucoma Phase 1b demonstration of intra-ocular pressure lowering Q3 2019 Potential for activity in systemic vascular complications of diabetes AKB-4924, a HIF-1a stabilizer in development for inflammatory bowel disease Proof-of-concept target engagement data expected Q4 2018 Aerpio holds global rights to all intellectual property 3
The Tie2 Pathway & Diabetic Eye Disease 4
The Tie2 pathway is validated as the major axis for vascular stability and quiescence Tie2 is a transmembrane receptor found on endothelial cells Tie2 activity Maintains integrity of endothelial cell junctions (blocks vessel leak) Enhances endothelial cell function and viability Inhibits vascular inflammation Active Tie2 = Vascular Stability 5
Activation of the Tie2 receptor is the goal of all the therapeutics in development Ang-2 Antibody Ang-1 (Tie2 activating ligand) Blood Vessel Lumen Ang-2/VEGF Antibody (extracellular) Intracellular Space Tie2 Receptor VE-PTP Small Molecule (Transmembrane receptor AKB-9778 Aerpio found on all vasculature) Ang-2: Angiopoietin-2 VEGF: Vascular endothelial growth factor VE-PTP: Vascular endothelial protein tyrosine phosphatase 6
In diabetic eye disease, VE-PTP and Ang-2 upregulation turns off Tie2 Blood Vessel Lumen Ang-2 (Competitive (extracellular) inhibitor of Ang-1) Intracellular Space VE-PTP (Deactivates Tie2 on intracellular side of receptor) Ang-1: Angiopoietin-1 Ang-2: Angiopoietin-2 VE-PTP: Vascular endothelial protein tyrosine phosphatase 7
VE-PTP inhibition activates Tie2 more robustly than Ang-2 inhibition -2 Mab Blood Vessel inds Ang-2) Lumen (extracellular) Intracellular Space AKB-9778 (Binds to and inhibits VE-PTP) Ang-2: Angiopoietin-2 Mab Monoclonal antibody VE-PTP Vascular endothelial protein tyrosine p 8
VE-PTP inhibition is the optimal approach to activating Tie2 AKB-9778 Nesvacumab (REGN910) robustly activates Tie2 minimally activates Tie2 in human endothelial cells in human endothelial cells Shen et al. JCI 124:4564-76, 2014 Daly et al. Cancer Research 73:108-18, 2012 9
Vascular changes seen in early diabetic eye disease are caused by loss of Tie2 function Diabetic eye disease is a progressive disease characterized by worsening vascular damage Vascular damage in the eye leads to leakage of fluid and proteins in the surrounding retinal tissue Eventually the damage is severe enough to cause significant vision loss and potentially blindness NPDR Non-proliferative diabetic retinopathy PDR Proliferative diabetic retinopathy DME Diabetic macular edema 10
AKB-9778 TIME-2 Clinical Data: Diabetic Macular Edema 11
TIME-2 tested AKB-9778 alone and with Lucentis® in a randomized, phase 2a study AKB-9778 Monotherapy N=144 AKB-9778 + Lucentis® Combo Therapy 1:1:1 Lucentis® Monotherapy D0 D28 D56 D84 15 mg AKB-9778 SC BID 0.3 mg Lucentis® Placebo SC BID Sham injection 12
TIME-2: Change in retinal thickness over time AKB-9778 (N=46) Lucentis® (N=47) AKB-9778 + Lucentis® (N=48) 0 -8 6 -10 (µm) -20 CST in -70 -91 -102 -110 -120 -106 Change p = 0.02 p = 0.008 -146 -164 -170 Month 1 Month2 Month 3 P1 data showed efficacy as monotherapy in DME prompting testing of monotherapy in P2 No efficacy seen as monotherapy in primarily VEGF-driven disease (DME) Vascular stabilizing effect of combination therapy significantly greater than either agent alone Campochiaro P, et al. Ophthalmology, Aug. 2016; Vol. 123. 13
AKB-9778 TIME-2 Clinical Data: Diabetic Retinopathy 14
Progression of diabetic eye disease is measured using a discreet 11-step scale (Diabetic Retinopathy Severity Score—DRSS) Non-proliferative diseaseProliferative disease ETDRS Severity Lev Steps Very MildMild Mod.Sev.VeryMild Mod.High Risk Sev. 2-step improvement in DRSS at one year is a FDA-accepted endpoint for approval Risk of vision-threatening events (diabetic macular edema, hemorrhage, retinal detachment, etc.) increases with worsening step progression Treatment of diabetic retinopathy is generally not initiated until later in disease due to the drawbacks of currently available interventions (e.g. laser, intraocular injection) Early intervention with AKB-9778 could slow or prevent disease progression and represents a potential paradigm change in the treatment of diabetic eye disease globally Klein R et al., Arch Ophthalmol., 2001, Vol. 119.15
Assessment of AKB-9778 on DRSS from the TIME-2 study Pre-specified, planned analysis comparing effect of AKB-9778 on DRSS in fellow eyes without DME SC AKB-9778 Treated N=144 1:1:1 SC Placebo Treated D0 D28 D56 D84 15 mg AKB-9778 SC BID Placebo SC BID 16
Assessment of AKB-9778 on diabetic retinopathy severity score from the TIME-2 study Percentage of Patients with a ³ 2-Step Improvement in DRSS @ 3 Months 15 2-step improvement expected to increase at one-year timepoint (on- 11.4going TIME-2b study) 10 Lucentis 2-step improvement from RISE/RIDE studies at M3 (16-18%), patientsM6 (27%), M12 (31-34%) 4.2 of5 Placebo rate of improvement consistent %with sham control in RISE/RIDE studies (3% at M3, M6 and 12) 0 Fellow Eye Consistent and bilateral 2-step improvement in DRSS from TIME-2 Placebo Arm (N=24)support biologic activity of AKB-9778 AKB-9778 Arms (N=70) DRSS Diabetic retinopathy severity score Campochiaro P, et al. Ophthalmology, Aug. 2016; Vol. 123.17
TIME-2 safety results AKB-9778 + AKB-9778Lucentis®Lucentis® (N=48)(N=47)(N=49) Number of Ocular AEs174548 Subjects w/ Ocular AEs, n (%)10 (20.8)19 (40.4)23 (46.9) Number of Non-Ocular AEs7689108 Subjects w/ Non-Ocular AEs, n (%)28 (58.3)30 (63.8)33 (67.3) Number of Serious AEs*202 Subjects w/ Serious AEs, n (%)2 (4.2)02 (4.1) * No serious AEs were considered drug-related by investigators Campochiaro P, et al. Ophthalmology, Aug. 2016; Vol. 123.18
AKB-9778 development: DR vs. DME Emergence of approval pathway for DR (2015 Lucentis & Eylea approvals) led us to pre-specify a DRSS analysis in the TIME-2 study AKB-9778 product attributes support a potential market-leading profile for patients with DR, good vision, and no center involved DME No need for intraocular injections No increase in frequency of visits to ophthalmologist Treats both eyes via systemic delivery (70% have bilateral disease) Potential to prevent vision threatening DME and PDR May improve diabetes-induced compromise of other vascular beds Commercial considerations DME Diabetic macular edema DR Diabetic retinopathy DRSS Diabetic retinopathy severity score 19 PDR Proliferative diabetic retinopathy
TIME-2b Study 20
TIME-2b: Clinical trial design Prospective, randomized, placebo-controlled study in pts with moderate to severe Non-Proliferative Diabetic Retinopathy (NPDR) without DME 150 patients: 15mg AKB-9778 once or twice/day vs. placebo 48 week treatment period 50 sites in United States 1o Endpoint: ³ 2-step improvement in DRSS at 48 weeks Key 2o Endpoints: development of DME/PDR, DR progression, renal function Enrollment commenced June 2017, data expected Q2 2019 DME Diabetic macular edema DR Diabetic retinopathy DRSS Diabetic retinopathy severity score PDR Proliferative diabetic retinopathy 21
Commercial Opportunity: AKB-9778 in Diabetic Retinopathy 22
DR represents a large unmet medical need with significant market potential Diabetes affects over 400M patients worldwide Over 40M patients in North America and 60M in Europe 143 1 in 3 diabetics have DRGloballyLucentis® approved Patients (millions)in July 2017 of 1 in 15 diabeticsNumber312015 Lucentis® have DMEand Eylea® global sales ï,» $1.5-2.0 B DRDME DR diabetic retinopathy DME diabetic macular edema Yau J. et al. Diabetes Care, March 2012, Vol 35. International Diabetes Federation. 2015 IDF Diabetes Atlas. Retrieved from http://www.diabetesatlas.org/ Cowen and Company. 2016 Therapeutic Categories Outlook. New York.23
Unmet need for the treatment of early diabetic eye disease is clear Lloyd Paul Aiello, MD, PhD Beetham Eye Institute, Joslin Diabetes Center 2015 FDA/NEI Workshop on Diabetic Retinopathy DME- Diabetic macular edema DR diabetic retinopathy FDA Food and Drug Administration NEI National Eye Institute VEGF Vascular endothelial growth factor Visiongain: Global Ophthalmic Drugs Market Forecast 2017-2027. 24
Opportunity to treat earlier stage disease Treating patients earlier in the disease process represents a large market opportunity with significant unmet need Vision-related concerns are the greatest health concern of patients with diabetes and a strong motivator 25
Patient preferred treatment modality Monthly intraocular injections of anti- VEGF for DR is not a sustainable treatment strategy In contrast, AKB-9778 is patient- administered via daily SC injectionPen Injector that you or someone Reduces treatment and visit burdenelse administers56% ONCE per day More acceptable treatment delivery, avoiding eye injections, for less symptomatic patientsAn injection in the eye, administered13% Exposure to systemic vasculature mayin the doctor’s office effectively treat both eyes and other vascular complications of diabetes SC SubcutaneousBase = DR Patients (n=101) VEGF Vascular endothelial growth factorAerpio data on file. Lucentis® package insert Kiss S, et al. Clin Ophthalmol. 2014;8:1611-1621.26
AKB-9778 market opportunity includes the largest segment of diabetic eye disease Sev PDR Moderate PDR 125 M diabetics progressionglobally have DRMild PDR diseaseVery Severe NPDR Severe NPDR WorseningModerate NPDR Mild NPDR DR Diabetic retinopathy NPDR Non-proliferative diabetic retinopathy PDR Proliferative diabetic retinopathy27
Systemic Tie2 stabilization may have implications for other diabetic complications Restoration of Tie2 activation in chronic diabetes Koh, G. Trends in Molec Med. January 2013, 19:1. 31-39. 28
AKB-9778: Primary Open-Angle Glaucoma 29
Loss of Tie2 function leads to increased IOP and glaucoma phenotype 30
Glaucoma is the leading cause of blindness in the world Expected to increase to between 53 and 65.5 million affected individuals by 2020 Weinreb, R. N. et al. (2016) Primary open-angle glaucoma. Nat. Rev. Dis. Primers doi:10.1038/nrdp.2016.67 Tham, Y.-C. et al. Global prevalence of glaucoma and projections of glaucoma burden through 2040: a systematic review and meta-analysis. Ophthalmology 121, 2081 2090 (2014). Kapetanakis, V. V. et al. Global variations and time trends in the prevalence of primary open angle glaucoma (POAG): a systematic review and meta-analysis. Br. J. Ophthalmol. 100, 86 93 (2016). 31
Inhibition of VE-PTP activates Tie2 & affects downstream Rho kinase and eNOS activity AKB-9778 could achieve effects of both of the recently approved glaucoma products 32
Stat sig reductions in IOP were observed in the TIME-2 study at every time point AKB-9778AKB-9778 +Lucentis® MonotherapyLucentis®monotherapy SEFESEFESEFE Mean Baseline IOP 15.815.415.916.115.215.8 (mmHG) Mean Ä from BL -1.4-1.4-1.0-1.50.1-0.1 (mmHG) t-test Ä BL-Mo 3 <0.01<0.01<0.05<0.010.880.84 (p-value) BL = baseline; SE = study eye; FE = Fellow eye; SD = standard deviation Reduction of IOP increased to 2-2.5 mm Hg in patients with baseline IOPs ³ 16 mm Hg, similar to prostaglandin analogs in normotensive glaucoma Systemically administered ï¢-blockers and nitrates, drugs used in glaucoma as topical drops, have shown a similar stat sig reductions in IOP when administered systemically, 1 mm Hg Dirks et al. Adv Ther. 23:3, 2006 Khawaja et al. Ophthalmology 121:1501, 2014 Stenkula and Wettrell, Graefe’s Arch Clin Exp Ophthalomol 218:96, 198233
Dose related IOP decrease observed after topical ocular administration of AKB-9778 Normotensive Rabbits 34
AKB-9778 in primary open angle glaucoma AKB-9778 Target Product Profile Primary IndicationReduction of elevated intraocular pressure (IOP) Target Pt. PopulationPrimary open angle glaucoma / ocular hypertension Route ofInitiateInitiate AdministrationTopical installationphase 1bphase 2a studystudy Dosing Schedule1 drop QD (blow fill seal, 0.3 ml, single-dose) In adjunctive setting: Additive effect of at least 1.5 mm Hg when used as adjunct to first-line treatment In monotherapy setting: IOP reduction of at least 5 mm Hg (or 20%) OR at least 3 mm Hg compared to Efficacyplacebo Lack of significant rate of systemic and ocular sideQ1 Q2Q3 Q4 Q1 Q2 Q3 Q4 Q1 effects seen with other classes of drugs (eg, Systemic:201820192020 cardiovascular, respiratory, headache, drowsiness, depression, dry mouth, taste disturbance; Ocular:Top-line File allergy, iris/skin discoloration, blurred vision, Safety/Tolerabilityhyperemia).INDphase 1b results First-line therapy of choice in adjunctive setting First-line for patients where an alternative to prostaglandin analog (PGA) desired: PGA-intolerant or PGA-nonresponsive patients; concerns re PGA- Positioningassociated cosmetic side effects IND investigational new drug35
AKB-4924: Inflammatory Bowel Disease 36
AKB-4924 in inflammatory bowel disease First-in-class, HIF-1a stabilizer for IBD Addresses major unmet needs in IBD Efficacy and safety in preclinical models and early human studies support a preferred profile for moderate/severe and potentially earlier stage disease vs. current standard of care Oral, once-daily route of administration Proof-of-concept data in Q3 2019 AE adverse event HIF-1a hypoxia inducible factor-1 alpha IBD inflammatory bowel disease 37
AKB-4924 in Inflammatory Bowel Disease: Novel Mechanism of Action AKB-4924 is a HIF-1α stabilizer that represents a new therapeutic class of potentially transformative therapeutics in IBD MOA enhances both mucosal wound healing and the resolution of inflammation HIF-1α regulates innate immune responses by supporting broad-spectrum bactericidal and phagocytic activities of epithelial cells, neutrophils and macrophages important for the resolution of inflammation1 HIF-1α elicits a protective barrier function in intestinal epithelia during tissue injury and inflammation supporting resolution of inflammation and restoration of normal gut homeostasis2-5 HIF-1α hypoxia inducible-1 alpha IBD inflammatory bowel disease MOA mechanism of action 1. Nizet and Johnson, Nature Reviews Immunol. 9:609,2009; 2. Furuta GT, Turner JR, Taylor CT, et al. J. Ex. Med. 2001;193:1027-1034; 3. Comerford KM, Wallace TJ, Karhausen J, et al. Cancer Res 2002;62:3387-94; 4. Synnestvedt K, Furuta GT, Comerford KM, et al. J. Clin. Invest. 2002;110:993-1002; 5. Eltzschig HK, Ibla JC, Furuta Gt, et al. J. Ex. Med. 2003;198:783-796; 6. Karhausen JO, Furuta GT, Tomaszewski JE, et al. J Clin Invest 2004;114:1098-1106; 7. Robinson A, Keely S, Karhausen J, et al. Gastroenterology 2008;134:145-55. 38
AKB-4924 efficacy demonstrated in multiple models of IBD Pre-clinical proof-of-concept across multiple models of IBD in both the induction and maintenance setting TNBS-induced colitis Wild type mice (below) Chronic granulomatous disease mice DSS-induced colitis Genetic TNFα overexpression induced Crohn’s Disease Gut Graft Versus Host Disease DSS dextran sodium sulfate IBD inflammatory bowel disease TNBS trinitrobenzene sulphonic acid TNFa tumor necrosis factor alpha 39
AKB-4924 decreases colonic cytokine levels and enhances mucosal healing AKB-4924 significantly reduces AKB-4924 reduces levels of proinflammatory TNBS-induced leak of cytokines in target tissue (colon) luorescent dextran rom colon over 7 ays AKB-4924 reduces levels of circulating acterial endotoxin (LPS) from the gut secondary to storation of barrier unction Keely et al. Immunology 2014;7:114-123. 40
AKB-4924 profile in IBD supports a significant market opportunity Surgery +++ Biologics 2nd malignancy, opportunistic infection, SevereimmunogenicitySeverity AKB-4924Disease ³ efficacy profile, preferred safety and route of administration & ModerateCost Steroids (systemic/topical), azathioprine- - AEs: nausea, blood disorders, immune system compromise- Mild 5-ASA/mesalamine IBD Inflammatory bowel disease41
AKB-4924 in inflammatory bowel disease AKB-4924 Target Product Profile -inducing and maintaining clinical response & remission -improving endoscopic appearance of Primarythe mucosa Indication-achieving corticosteroid-free remission Target Pt. PopulationModerate/Severe Ulcerative Colitis Route of AdministrationPO (enteric-coated tablet) Dosing ScheduleOnce daily Efficacy³ to infliximab Safety/Lack of secondary malignancy, opportunistic Tolerabilityinfection, or immunogenicity reactions First-line therapy of choice in moderate to Positioningsevere ulcerative colitis Top-lineInitiate results fromphase 2 MAD studystudy Q1Q2Q3Q4Q1Q2Q3Q4Q1 201820192020 InitiateFile INDTop-line MAD studyresults phase 1b Initiatestudy phase 1b study IND investigational new drug MAD multiple ascending dose PO Oral 42
Pipeline 43
Aerpio pipeline based on novel mechanisms Stage of Development Product CandidateIndicationApproachPreclinicalPhase 1Phase 2aPhase 2bNear-Term Milestones Tie2 AKB-9778DRActivatorTop-line data available Q2 2019 (SC inj) Tie2 POAG/Phase Ib data available AKB-9778Activator OHTin Q3 2019 (eye drop) HIF-1Phase Ib data available AKB-4924IBD Stabilizerin Q3 2019 Tie2 Chronic ARP-1536ActivatorTBD Indications Antibody DR Diabetic retinopathy IBD Inflammatory bowel disease POAG/OHT Primary open angle glaucoma/ocular hypertension SC Subcutaneous44
Upcoming news events AKB-9778 in NPDRTop-line results from phase 1b study AKB-9778 in POAG (eye drops)InitiateInitiate 28-day AKB-4924 in UCphase 1bphase 2a study Complete enrollmentTop-line resultsInitiate in TIME-2b studyFile INDfrom TIME-2bphase 3 (phase 2b NPDR)(phase 2b NPDR)studies Q1Q2Q3Q4Q1Q2Q3Q4Q1 201820192020 InitiateTop-line MAD studyresults from MAD studyTop-lineInitiate resultsphase 2 File INDphase 1bstudy IND Investigational new drugInitiatestudy MAD Multiple ascending dosephase 1b NPDR Non-proliferative diabetic retinopathystudy POAG Primary open angle glaucoma UC Ulcerative colitis45
Corporate highlights Advancing first-in-class treatments for ophthalmic disease Non-proliferative diabetic retinopathy (NPDR) Primary open angle glaucoma (POAG) AKB-9778 is the most potent activator of the Tie2 pathway, proven essential for vascular stability Phase 2b DR data expected Q2 2019 Proof-of-concept in DR demonstrated in Phase 2a DME study Approximately 13 million DR patients in the US and 20 million in EU Multi-billion dollar market potential Strong rationale for efficacy in primary open angle glaucoma hase 1b demonstration of intra-ocular pressure lowering Q3 2019 Potential for activity in systemic vascular complications of diabetes AKB-4924, a HIF-1a stabilizer in development for inflammatory bowel disease Proof-of-concept target engagement data expected Q4 2018 Aerpio holds global rights to all intellectual property 46
www.aerpio.com 47