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): February 28, 2019
ALDEYRA THERAPEUTICS, INC.
(Exact name of Registrant as specified in its charter)
Delaware
(State or other jurisdiction of incorporation)
| 001-36332 | 20-1968197 | |
| (Commission File No.) | (IRS Employer Identification No.) |
131 Hartwell Avenue, Suite 320
Lexington, MA 02421
(Address of principal executive offices and zip code)
Registrant’s telephone number, including area code: (781) 761-4904
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. |
On February 28, 2019, Aldeyra Therapeutics, Inc. (“Aldeyra”) intends to make a slide presentation at its 2019 Research & Development Day in person in New York City and by webcast on Aldeyra’s website. A copy of Aldeyra’s slide presentation is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated by reference herein.
The furnishing of the attached slide presentation is not an admission as to the materiality of any information contained therein. The information contained in the slide presentation is summary information that is intended to be considered in the context of more complete information included in Aldeyra’s filings with the Securities and Exchange Commission (“SEC”) and other public announcements that Aldeyra has made and may make from time to time by press release or otherwise. Aldeyra undertakes no duty or obligation to update or revise the information contained in this report, although it may do so from time to time as its management believes is appropriate.
Various statements to be made during the conference call are “forward-looking statements” under the securities laws, including, but not limited to, statements regarding Aldeyra’s strategy, future operations, future, prospects, plans, and objectives and Aldeyra’s plans and expectations for its product candidates. In some cases, you can identify forward looking statements by terms such as, but not limited to, “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “aim,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties.
Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. All of Aldeyra’s development timelines may be subject to adjustment depending on recruitment rate, regulatory review, preclinical and clinical results, and other factors that could delay the initiation or completion of clinical trials. Important factors that could cause actual results to differ materially from those reflected in Aldeyra’s forward-looking statements include, among others, the timing of enrollment, commencement and completion of Aldeyra’s clinical trials, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; the ability to obtain and maintain regulatory approval of Aldeyra’s product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Aldeyra’s product candidates; the size and growth of the potential markets and pricing for Aldeyra’s product candidates and the ability to serve those markets; Aldeyra’s expectations regarding Aldeyra’s expenses and revenue, the sufficiency or use of Aldeyra’s cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra’s product candidates; Aldeyra’s expectations regarding competition; Aldeyra’s anticipated growth strategies; Aldeyra’s ability to attract or retain key personnel; Aldeyra’s ability to successfully integrate its new senior management team members; Aldeyra’s ability to establish and maintain development partnerships; Aldeyra’s expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries; Aldeyra’s ability to obtain and maintain intellectual property protection for Aldeyra’s product candidates; the anticipated trends and challenges in Aldeyra’s business and the market in which it operates; and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Aldeyra’s Annual Report on Form 10-K for the year ended December 31, 2017 and Aldeyra’s Quarterly Report on Form 10-Q for the
quarter ended September 30, 2018, both of which are on file with the SEC and available on the SEC’s website at www.sec.gov. Additional factors may be described in those sections of Aldeyra’s Annual Report on Form 10-K for the year ended December 31, 2018, expected to be filed with the SEC in the first quarter of 2019.
In addition to the risks described above and in Aldeyra’s other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra’s results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information conveyed on the conference call is provided only as of the date of the call, and Aldeyra undertakes no obligation to update any forward-looking statements presented on the call on account of new information, future events, or otherwise, except as required by law.
The information in Item 7.01 of this Form 8-K shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference under the Securities Act of 1933, as amended, except as expressly set forth by specific reference in such a filing, regardless of any general incorporation language in any such filing, unless Aldeyra expressly sets forth in such filing that such information is to be considered “filed” or incorporated by reference therein.
| Item 8.01 | Other Events. |
On February 28, 2019, Aldeyra issued a press release that provided an update on Aldeyra’s clinical development plans and pipeline. A copy of the press release is attached as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits
| Exhibit |
Description | |
| 99.1 | Slide presentation of Aldeyra Therapeutics, Inc. dated February 28, 2019. | |
| 99.2 | Press Release of Aldeyra Therapeutics, Inc. dated February 28, 2019. | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| ALDEYRA THERAPEUTICS, INC. | ||||
| By: | /s/ Joshua Reed | |||
| Name: | Joshua Reed | |||
| Title: | Chief Financial Officer | |||
Dated: February 28, 2019
R&D Day 2019 Update on Research Programs February 28, 2019 Exhibit 99.1
Disclaimers and Forward-Looking Statements This presentation and various remarks which may be made during this presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and Section 21E of the Securities Exchange Act of 1934, as amended, including statements regarding Aldeyra’s possible or assumed future results of operations, expenses and financing needs, business strategies and plans, research and development plans or expectations, trends, the structure, timing and success of Aldeyra’s planned or pending clinical trials, expected milestones, market sizing, pricing and reimbursement, competitive position, regulatory matters, industry environment and potential growth opportunities, among other things. Forward-looking statements include all statements that are not historical facts and, in some cases, can be identified by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “plan” or similar expressions and the negatives of those terms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Aldeyra’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect Aldeyra’s current views with respect to future events and are based on assumptions and subject to risks and uncertainties, including the development, clinical and regulatory plans or expectations for Aldeyra’s product candidates and Aldeyra’s continuing review and quality control analysis of clinical data. Important factors that could cause actual results to differ materially from those reflected in Aldeyra's forward-looking statements are described in Aldeyra’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, as well as Aldeyra’s subsequent filings with the Securities and Exchange Commission. All of Aldeyra's development timelines may be subject to adjustment depending on recruitment rate, regulatory review, preclinical and clinical results, and other factors that could delay the initiation, completion, or reporting of clinical trials. In addition to the risks described above and in Aldeyra's other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra's results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this presentation is provided only as of February 28, 2019, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this presentation on account of new information, future events, or otherwise, except as required by law.
Agenda Opening WelcomeTodd Brady, CEO Corporate Strategy & Pipeline GrowthDavid McMullin, CCO Proliferative Vitreoretinopathy – A Rare Retinal DiseaseDean Eliott, M.D. Harvard Medical School Mass. Eye and Ear Infirmary Ocular Disease Area Program UpdatesDavid Clark, CMO Ocular Disease Area Market OpportunitiesChris Pearson, VP Commercial ConclusionTodd Brady, CEO Q&A
Our Mission Developing Next-Generation Medicines to Improve the Lives of Patients with Immune-Mediated Diseases ~7% Of Western Society Suffer from some form of immune-mediated disease, and incidence is increasing Disease control elusive despite existing therapies, and thus novel approaches are needed Unmet Needs Source: Lerner, Jeremias, and Matthias, International Journal of Celiac Disease, vol. 3, no. 4 (2015): 151-155; Shurin and Smolkin, Advances in Experimental Medicines and Biology 601:3-12, 2007; Kuek et al, Postgraduate Medical Journal 83(978): 251-260, 2007.
Immune System Imbalance Leads to Immune-Mediated Disease Stimulation Imbalance Inhibition Imbalance Autoimmune Cancer Immuno-proliferative Allergy Stimulation Inhibition Immune Balance Healthy Imbalance can result in… Immune-Mediated Diseases
Deliberate Focus on Ocular Diseases and Select Systemic Diseases 2004 May 2014 2015 2016 2017 2018 2019 … Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis PVR Retinal Disease Sjögren-Larsson Syndrome PTLD Mesothelioma and other rare cancers** Autoimmune Discovery & Preclinical Ocular Diseases Systemic Diseases RASP = Reactive Aldehyde Species Inhibitor DHFR = Dihydrofolate Reductase Inhibitor Hsp90 = Heat Shock Protein 90 Inhibitor PTLD = Post-Transplant Lymphoproliferative Disorder PVR = Proliferative Vitreoretinopathy RASP Hsp90 DHFR RASP RASP RASP RASP RASP Hsp90 IPO; First Phase 2 IND filing Founded First Phase 2 Initiations First Phase 2 Completions First Phase 3 Initiations Add’l Phase 2; Add’l Phase 3 Potential First Phase 3 Completions* *Contingent on funding, regulatory review, and other factors. **Initially supporting Investigator Sponsored Trials upon Hsp90 licensure.
Our Novel Approaches to Address Immune-Mediated Disease Cell Activation Cell Proliferation Immune Cell Stimulus Hsp90 Inhibitors RASP Inhibitors DHFR Inhibitors RASP = Reactive Aldehyde Species DHFR = Dihydrofolate Reductase Hsp90 = Heat Shock Protein 90 Reproxalap ADX-103 ADX-629 ADX-1612 ADX-1615 ADX-2191
Ocular Inflammation Deep and Innovative Pipeline Focused on Immune-Mediated Diseases Ocular Diseases Indication Compound Disease Area Preclinical Phase 1 Phase 2 Phase 3 Next Anticipated Milestone = Positive Phase 2 clinical trial data reported in 2016 – 2018 Trial initiations contingent on funding, regulatory review, and other factors ü RASP = Reactive Aldehyde Species Inhibitor DHFR = Dihydrofolate Reductase Inhibitor Hsp90 = Heat Shock Protein 90 Inhibitor PTLD = Post-Transplant Lymphoproliferative Disorder Proliferative Vitreoretinopathy ADX-2191 Reproxalap Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis ADX-103 Retinal Disease ADX-629 Autoimmune Disease ADX-1612 PTLD Mesothelioma Ovarian Cancer Reproxalap Sjögren-Larsson Syndrome Undisclosed Systemic Inflammatory Disease ADX-1615 Autoimmune Disease / Cancer Undisclosed Systemic Diseases ü ü ü ü ü ü ü Investigator-Sponsored Trial Research Collaboration Research Collaboration (undisclosed) Phase 3-Part 1 initiation H1 2019 Phase 3 results early 2019 Phase 3 results H2 2019 Phase 3-Part 1 results H2 2019 Phase 3-Part 1 initiation H2 2019 Phase 1 initiation H2 2019 Phase 2 initiation H1 2019 Phase 1/2 initiation 2020 Phase 2 initiation H2 2019 [Mechanism] [RASP] [RASP] [RASP] [RASP] [RASP] [Hsp90] [Hsp90] [DHFR]
Helio Vision Acquisition Expands Pipeline in Support of Our Strategic Growth Plans Retinal disease a strategic priority for pipeline growth Novel therapeutic approach leveraging an immunological mechanism that diminishes inflammation and cell proliferation Addition of Phase 3-ready clinical program Orphan drug designation for proliferative vitreoretinopathy, a potentially blinding disease with no approved treatment Potential applicability to a variety of other diseases
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
ADX-2191: Adaptive Phase 3 Proliferative Vitreoretinopathy Clinical Program Expected to Initiate H2 2019 Confirm endpoints, safety and tolerability Confirm sample size for subsequent trial Part 1 controlled, randomized trial design Adaptive Phase 3 Program Phase 3 Program Design Elements Adaptive Phase 3 (Part 1) PVR Trial Adaptive Phase 3 (Part 2) PVR Trial Confirmatory Phase 3 PVR Trial NDA Illustrative only Expected initiation H2 2019 PVR = Proliferative vitreoretinopathy
Primary objective: Evaluate efficacy of intravitreal ADX-2191 injections for prevention of recurrent retinal detachment due to proliferative vitreoretinopathy (PVR) Design: Multi-center, non-masked, randomized, controlled, two- part, adaptive Phase 3 clinical trial Inclusion highlights: Recurrent retinal detachment due to PVR, or Retinal detachment associated with open-globe trauma Dosing regimen: Weekly (x10) then every other week (x3) intravitreal ADX-2191 injections Endpoint: Retinal re-detachments due to PVR requiring re-operation within 6 months: OCT demonstrating fovea-off retinal detachment Photographic documentation retinal detachment ADX-2191: Adaptive Phase 3 (Part 1) Proliferative Vitreoretinopathy Clinical Trial Design Adaptive Phase 3 PVR Clinical Trial Design: Part 1 Operative Day 1 Post-Op Week 4 Month 3 ADX-2191 added to routine surgical care (N = 50) Control Arm: Routine surgical care (N = 50) ADX-2191 intravitreal injection treatment Post-Op Week 8 Post-Op Week 12 Post-Op Week 24 Post-Op Week 16 Month 4 Month 5 Month 6 Month 2 Month 1 OCT = Optical Coherence Tomography
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Reproxalap: Adaptive Phase 3 Dry Eye Disease Clinical Program Expected to Initiate H1 2019 Adaptive Phase 3 Program Phase 3 Program Design Elements Adaptive DED Phase 3 (Part 1) Adaptive DED Phase 3 (Part 2) Confirmatory DED Phase 3 NDA Illustrative only Expected initiation H1 2019 Phase 3 DED Safety Study Adaptive design, co-primary endpoints and innovative analysis strategy confirmed with FDA at EOP2 Meeting Confirm dosing regimen (QID vs. QID to BID taper) Confirm sample size for subsequent trial Confirm symptom and sign endpoints from Phase 2b trial DED = Dry eye disease BID = Two times daily QID = Four times daily EOP2 = End of Phase 2
Primary objective: Evaluate efficacy of reproxalap ophthalmic solution (0.25%) vs. vehicle to confirm dosing regimen and sample size for Part 2 Inclusion/exclusion criteria: Same as used for Phase 2b Moderate to severe dry eye disease Co-primary endpoints: Ocular dryness score (0-100mm VAS) and fluorescein nasal region staining Analysis strategy: Both co-primary endpoints will be assessed using Mixed Model Repeated Measures (MMRM) from week 2 to week 12 Both co-primary endpoints will be assessed based on separate pre-specified patient populations Ocular dryness score (OD4SS): baseline score of > 3 Fluorescein nasal staining: baseline score > 2 Reproxalap: Adaptive Phase 3 (Part 1) Dry Eye Disease Clinical Trial Design Vehicle -Wk 2 -Wk 1 Wk 1 Wk 2 Wk 3 Wk 4 Wk 5 Wk 6 Wk 7 Wk 8 Wk 9 Wk 10 Wk 11 Wk 12 Visit 1 Week -2 Visit 2 Day 1 Visit 4 Wk 2 Visit 5 Week 4 Visit 7 Week 8 Visit 9 Week 12 Screening Phase 3 Dry Eye Disease Clinical Trial: Part 1 Treatment Visit 3 Wk 1 Visit 6 Week 6 Visit 8 Week 10 Reproxalap 0.25% (N = 100) QID Vehicle (N = 100) QID Reproxalap 0.25% (N = 100) QID to BID Vehicle (N = 100) QID to BID VAS = Visual analog scale OD4SS = Ocular Discomfort 4-Symptom Score
Reproxalap: Adaptive Phase 3 (Part 2) Dry Eye Disease Clinical Trial Design Vehicle -Wk 2 -Wk 1 Wk 1 Wk 2 Wk 3 Wk 4 Wk 5 Wk 6 Wk 7 Wk 8 Wk 9 Wk 10 Wk 11 Wk 12 Visit 1 Week -2 Visit 2 Day 1 Visit 4 Wk 2 Visit 5 Week 4 Visit 7 Week 8 Visit 9 Week 12 Screening Treatment Regimen tbd (QID or QID to BID) Phase 3 Dry Eye Disease Clinical Trial: Part 2 Visit 3 Wk 1 Visit 6 Week 6 Visit 8 Week 10 Reproxalap 0.25% (N = 200-400) Vehicle (N = 200-400) Phase 3 Dry Eye Disease Clinical Trial: Part 1 Phase 3 Dry Eye Disease Clinical Trial: Part 2 Primary objective: Evaluate efficacy of reproxalap ophthalmic solution (0.25%) vs. vehicle on co-primary symptom and sign endpoints Population selection and design: Same as Part 1 Confirmed sample size Confirmed dosing regimen
Reproxalap: Dry Eye Disease Symptom and Sign Endpoints Achieved in Phase 2b Clinical Trial Mean Change from Baseline OD & 4-Symptom Questionnaire: Dryness (0-5) Baseline Score > 3 (N=69|69) Fluorescein Staining: Nasal (0-4) Baseline Score > 2 (N=62|56) Primary Symptom Endpoint for Phase 3 DED Primary Sign Endpoint for Phase 3 DED p values subject to change based on quality control analysis Source: Reproxalap DED Phase 2b clinical trial results * * ** * ** ** * *p<0.05 **p<0.01 OD = Ocular Discomfort MMRM = Mixed effect Model Repeated Measures MMRM p = 0.0048 MMRM p = 0.0007
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Reproxalap: ALLEVIATE Phase 3 Trial Design in Allergic Conjunctivitis Phase 3 Conjunctival Allergen Challenge Trial Primary objective: Evaluate efficacy of reproxalap ophthalmic solutions (0.25% & 0.5%) compared to vehicle for the treatment of ocular itching associated with acute allergic conjunctivitis Inclusion/exclusion highlights: Positive history of ocular allergies and positive skin test reaction to a seasonal allergen Positive bilateral conjunctival allergen challenge (CAC) reaction of ≥2.5 for itching and ≥2 for redness within 10 min of allergen instillation at first baseline visit Positive bilateral CAC reaction for at least two out of first three time points following challenge at second baseline visit Endpoints: Ocular itch score area under the curve (primary) Two-point responder comparison (key secondary) Results expected to be announced early 2019 Further information can be found on www.clinicaltrials.gov: Trial #NCT03494504. ALLEVIATE is the first of two required Phase 3 clinical trials, pending regulatory review. In preparation for a subsequent Phase 3 clinical trial, Aldeyra is conducting clinical method development studies to assess the feasibility of measuring ocular itching following environmental exposure to allergen. Reproxalap Administration Allergen Challenge Patient Reported Itch Assessment A-1 A0 A1 A2 A3 A4 A5 A6 A7 -10 10 20 30 40 50 60 Time (in minutes) Reproxalap 0.25% (N = 100) Vehicle (N = 100) Reproxalap 0.5% (N = 100)
Reproxalap: AC Ocular Itch Area Under The Curve and Responder Endpoints Achieved in Phase 2b Clinical Trial Source: Reproxalap AC Phase 2b clinical trial results (~30 patients per arm, seasonal allergy) Area Under the Curve: Ocular Itch Score (0-4) Probability of Response: Ocular Itch Score (0-4) Improvement in itch score over one hour after allergen exposure statistically greater for reproxalap vs. vehicle Clinically significant response rate of reproxalap statistically higher than that of vehicle AC = Allergic conjunctivitis
Reproxalap’s Novel Mechanism of Action has the Potential to Provide More Durable Activity Than Antihistamines Reproxalap has the potential to be uniquely effective in post-histaminic allergy, for which no drug is approved, and which affects all allergic conjunctivitis patients. Histaminic Phase Post-Histaminic Phase (RASP-Mediated) (Cells, cytokines, and other aldehyde-stimulated factors) Reproxalap Activity Antihistamine Activity Allergen 0 10 20 30 40 50 60 Minutes Following Single Exposure to Allergen RASP = Reactive Aldehyde Species
AC Phase 3 CAC Reproxalap: Parallel Dry Eye Disease and Allergic Conjunctivitis Phase 3 Clinical Programs Support Concurrent NDA Filings Adaptive DED Phase 3 (Part 1) Adaptive DED Phase 3 (Part 2) Confirmatory DED Phase 3 Expected initiation H1 2019 Phase 3 DED Safety Study Phase 3 AC Safety Study AC Phase 3 Design to be confirmed Results expected early 2019 NDA Dry Eye Disease Adaptive Phase 3 Clinical Program Allergic Conjunctivitis Phase 3 Clinical Program Illustrative only Type C FDA Meeting DED = Dry eye disease AC = Allergic conjunctivitis
Ocular Disease Area Program Updates Proliferative Vitreoretinopathy Dry Eye Disease Allergic Conjunctivitis Upcoming Milestones
Multiple Upcoming Ocular Disease Area Clinical Program Milestones 2019 2020 Early 2019: Reproxalap ALLEVIATE Phase 3 allergic conjunctivitis trial Results H1 2019: Reproxalap Phase 3 dry eye disease clinical trial program Initiate H2 2019: Reproxalap SOLACE Phase 3 noninfectious anterior uveitis trial Results H2 2019: ADX-2191 Phase 3 proliferative vitreoretinopathy clinical trial program Initiate 2020: ADX-103 Phase 1/2 retinal disease clinical trial Initiate *Contingent on funding, regulatory review, and other factors. Ocular Disease Area Anticipated Milestones*
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
Rare Common Low High Under-served Prevalence Unmet Medical Need DED AC We Intend to Target Unmet Medical Needs in Anterior and Posterior Ocular Diseases Anterior Opportunities Posterior Opportunities Rare Common Low High Under-served Prevalence Unmet Medical Need NAU PVR ADX-103 ADX-2191 Future Clear unmet and underserved medical need Novel treatment solutions Label expansion potential NAU = Noninfectious anterior uveitis DED = Dry eye disease AC = Allergic conjunctivitis PVR = Proliferative vitreoretinopathy
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
4,000 U.S. PVR is a rare disease, with ~4,000 patients per year in the U.S. and nearly twice as many in Europe and Japan PVR is the leading complication of retinal detachment surgery and prevents successful reattachment PVR is an serious condition leading to permanent vision loss in up to 75% of cases With no therapies available, PVR necessitates repeat surgery with a repeat surgery failure rate of ~50% PVR: A Rare Sight-Threatening Retinal Disease 50/50 Source: Aldeyra internal estimates based on primary and secondary market research; published literature PVR = Proliferative vitreoretinopathy
PVR: High Unmet Medical Need With No Approved Therapies ~50% failure rate 4,000 per year >28,000 per year Rhegmatogenous Retinal Detachment Non-traumatic Traumatic (open-globe injury) Proliferative Vitreoretinopathy U.S. Estimates Primary Surgery Etc. Repeat Repeat Repeat PVR Repeat Surgery Addt’l. PVR Repeat Surgeries ~50% failure rate PVR typically manifests 1-2 months after primary retinal detachment surgery Novel Approaches Needed PVR primary surgery failure rates vary depending on detachment etiology Today, PVR patients undergo 3-to-4 additional surgeries on average Vision and quality of life decreases with each procedure 10-50% failure rate PVR Source: Aldeyra internal estimates based on primary and secondary market research; published literature No FDA-approved therapy
ADX-2191: A Unique Approach and Novel Product Candidate for PVR A Unique Opportunity PVR: A Sight-Threatening Disease ADX-2191 A novel approach and potential therapeutic breakthrough in PVR treatment Granted U.S. orphan designation Tolerability and reattachment success during study period demonstrated in Phase 1b clinical trial Adaptive Phase 3 clinical trial expected to initiate H2 2019 Repeat surgery and subsequent vision loss currently the only possible course of action Left untreated, retinal detachment due to PVR can progress to permanent blindness No FDA- or EMA-approved therapy PVR = Proliferative vitreoretinopathy
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
DED and AC: Persistently Disturbing and Overlapping Disease Burdens Dry Eye Disease Allergic Conjunctivitis DED+AC Comorbidity Women are twice as likely to suffer from DED than men vs Significant negative quality of life impact AC can result in acute, intermittent, and chronic symptoms AC patients experience symptoms throughout all decades of adult life 20s AC 40s 60s 80s Significant negative quality of life impact x2 Significant negative quality of life impact DED Allergen exposure can contribute to DED seasonality Studies have shown that DED and AC can be interrelated and often overlap 50- 60% ~50-60% of DED and AC patients experience clinically significant itch and dryness 20 million or more adults in the U.S. suffer from DED 20 million Up to 30 million of AC sufferers in the U.S. do not respond adequately to or are dissatisfied with antihistamines 30 million DED increases with age, with those over age 50 three times more likely to suffer from DED Age 50+ >3x Source: Aldeyra internal estimates based on primary and secondary market research; published literature DED = Dry eye disease AC = Allergic conjunctivitis
DED and AC: Chronic Diseases With Inadequate Therapies Dry Eye Disease Allergic Conjunctivitis DED+AC Comorbidity Current Rx options may take up to six weeks or longer to achieve even modest efficacy Up to 75% of patients with DED are not satisfied with current prescription options Up to 75% Up to 50% of patients treated for DED with current therapies fail and discontinue Up to 50% ~2% of AC patients have severe symptoms and may be corticosteroid-dependent 2% Underserved Patient Population Underserved Patient Population Unmet Medical Need Antihistamines are not effective in an estimated 24% of treated AC patients 24% Many AC patients make significant sacrifices due to lack of drug activity Antihistamine use can cause and exacerbate eye dryness Differential diagnosis is difficult and treating both conditions together is complex DED, AC or both? Source: Aldeyra internal estimates based on primary and secondary market research; published literature DED = Dry eye disease AC = Allergic conjunctivitis Current Rx DED treatments are not effective against AC and vice versa
DED and AC: Large Market Opportunities With Unmet Medical Needs 10 million 20 million 30 million 100 million Up to 50% comorbidity Allergic Conjunctivitis Unsatisfied With SOC Dry Eye Disease U.S. Patient Estimates Significant negative quality of life Complex, overlapping, and difficult to treat chronic conditions Substantial unmet medical need with current treatments Novel Approaches Needed Source: Aldeyra internal estimates based on primary and secondary market research; published literature DED = Dry eye disease AC = Allergic conjunctivitis SOC = Standard of Care
Effective in post-histaminic allergy, for which no drug is approved Clinically significant and durable symptom response in Phase 2b clinical trial Reproxalap: A Unique and Novel Product Candidate for DED and AC Dry Eye Disease Allergic Conjunctivitis DED+AC Overlap Reproxalap in DED Reproxalap Reproxalap in AC Early and consistent symptom and sign improvements in Phase 2b clinical trial Broad symptom and sign improvements in Phase 2b clinical trial Observed improvements in both DED and AC Phase 2b clinical trials Both patients and physicians have a strong desire for better DED and AC treatments Novel mechanism of action and differentiated approach to treat DED and AC DED = Dry eye disease AC = Allergic conjunctivitis
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
NAU: A Severe Ocular Inflammatory Disease ~50% of NAU patients have recurrent or chronic conditions requiring multiple interventions per year 260K annually NAU is the most common form of uveitis with an estimated 260,000 U.S. patients per year Noninfectious anterior uveitis (NAU) is a severe ocular inflammation causing pain, photophobia, and vision loss Acute Recurrent 2-3/pt./yr. Chronic 4+/pt./yr. NAU dramatically impacts quality of life, leading to loss of work and significant economic burden Disease Burden Overview Source: Aldeyra internal estimates based on primary and secondary market research; published literature
NAU: Significant Repeat Episodes and Steroid Toxicity Creates the Need for Novel Approaches Acute Recurrent Chronic ~50% ~25% ~25% Single flare within a year Relapse 3-12 months after initial episode Relapse <3 months after initial episode Prevalence: Approximately 260,000 noninfectious anterior uveitis (NAU) patients in the U.S. NAU episode frequency: Corticosteroid treatment: 8-12 times/day tapered over 4-6 weeks Prolonged corticosteroid usage increases risks of serious side effects 1 regimen per year 4-6 weeks of treatment Steroid use: Safety risk: 8-12+ weeks of treatment 12-18+ wks. of treatment 2-3 regimens per year 4+ regimens per year Potential corticosteroid side-effects include glaucoma, cataracts, corneal ulceration, ptosis, delayed wound healing, and ocular infection U.S. Estimates Source: Aldeyra internal estimates based on primary and secondary market research; published literature NAU = Noninfectious anterior uveitis
Reproxalap: A Unique and Novel Product Candidate for NAU A Unique Opportunity NAU: A Serious Inflammatory Disease With Inadequate Current Therapy Reproxalap A novel and differentiated approach to treat NAU Reduced anterior chamber cell count observed in a Phase 2 clinical trial, statistically non-inferior to corticosteroid treatment Safety and tolerability without IOP increase in a Phase 2 clinical trial SOLACE Phase 3 clinical trial results expected H2 2019 Prolonged usage may lead to glaucoma, cataracts, corneal ulceration, and other serious side effects Acute Recurrent Chronic ~50% of noninfectious anterior uveitis (NAU) patients have recurrent or chronic conditions requiring multiple interventions per year Steroids Corticosteroids currently SOC and require monitoring due to serious toxicities Source: Aldeyra internal estimates based on primary and secondary market research; published literature NAU = Noninfectious anterior uveitis SOC = Standard of Care IOP = Intraocular pressure
Ocular Disease Area Market Opportunities Proliferative Vitreoretinopathy Dry Eye Disease and Allergic Conjunctivitis Noninfectious Anterior Uveitis Pathway to Commercialization
We Intend to Commercialize Directly and Through Partnerships Estimated U.S. Population* U.S. Healthcare Providers Competitive Value Proposition Infrastructure Requirement Dry Eye Disease Allergic Conjunctivitis Noninfectious Anterior Uveitis Sjögren-Larsson Syndrome Late Stage Programs Ocular Diseases Systemic Diseases Proliferative Vitreoretinopathy ~18,000 ophthalmologists and ~40,000 optometrists 260,000 20 million DED Up to 10 million with DED & AC 30 million AC Potential benefits over current therapies, which do not work well for many patients Medium sized sales force for national reach 4,000 1,000 ~200 U.S. uveitis sub-specialists Retina specialists at targeted centers Geneticists and ped. neurologists Effective non-steroid alternative Orphan: First and only Rx treatment Orphan: First and only Rx treatment Small targeted sales force Small specialized operation Small specialized operation Characterize the business model Prepare for commercialization Develop partnership options *Aldeyra estimates of the addressable market Source: Aldeyra internal estimates based on primary and secondary market research; published literature Commercial Planning Launch readiness Maximize value
Conclusion
Expected Development Milestones: Novel Approaches to Address Immune-Mediated Disease *Contingent on funding, regulatory review, and other factors. Reproxalap allergic conjunctivitis ALLEVIATE Phase 3 trial results early 2019 Ocular Diseases: Anticipated Milestones* Systemic Diseases: Anticipated Milestones* Reproxalap dry eye disease Phase 3 clinical trial program initiation H1 2019 Update Reproxalap noninfectious anterior uveitis SOLACE Phase 3 clinical trial results H2 2019 Update ADX-2191 Proliferative Vitreoretinopathy Phase 3 clinical program initiation H2 2019 Update ADX-103 retinal disease Phase 1/2 clinical trial initiation 2020 Update ADX-1612 mesothelioma Phase 2 clinical trial initiation 2019 ADX-1612 post-transplant lymphoproliferative disorder Phase 2 clinical trial initiation 2019 ADX-629 Phase 1 clinical trial initiation H2 2019 followed by NASH and/or IBD Phase 2a Update Reproxalap Sjögren-Larsson Syndrome RESET Phase 3 - Part 1 clinical trial results H2 2019 Update
Exhibit 99.2
Aldeyra Therapeutics Provides Update on Ophthalmic Programs at 2019 Research & Development Day
Phase 3 Results from ALLEVIATE Trial in Allergic Conjunctivitis Expected in Early 2019
Co-Primary Endpoints Confirmed for Phase 3 Clinical Trial in Dry Eye Disease, Expected to Begin in First Half of 2019
Phase 3 Clinical Trial of ADX-2191 in Proliferative Vitreoretinopathy Expected to Begin in Second Half of 2019
Phase 3 Results from SOLACE Clinical Trial in Noninfectious Anterior Uveitis Expected in Second Half of 2019
LEXINGTON, Mass., February 28, 2019 (PRNewswire) — Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to developing and commercializing next-generation medicines to improve the lives of patients with immune-mediated diseases, today updated progress on ophthalmic programs at a 2019 Research & Development Day in New York City. Presentations were given by members of the Aldeyra executive team along with Dean Eliott, M.D., the Stelios Evangelos Gragoudas Professor of Ophthalmology at Harvard Medical School, Director of the Retina Service at Massachusetts Eye and Ear Infirmary, and Director of the Retina Fellowship at Harvard and Massachusetts Eye and Ear Infirmary. Presentations covered the development and commercialization plans for novel product candidates in proliferative vitreoretinopathy, dry eye disease, allergic conjunctivitis, and noninfectious anterior uveitis.
A webcast of the presentation and slide deck will be available via Aldeyra’s investor relations website at http://ir.aldeyra.com until February 28, 2020.
“Over the past few years, we have deliberately expanded our pipeline in support of our corporate strategic initiatives. Today, we have six different compounds in development, representing three unique mechanisms of action, targeting ten potential clinical indications,” commented Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “With a deliberate focus on ocular disease and select systemic conditions, we now have five Phase 3 programs in progress or expected to be initiated this year. We look forward to updating investors on the first of the Phase 3 programs, the ALLEVIATE trial in allergic conjunctivitis, early this year.”
R&D Day Highlights
| • | ADX-2191 for Proliferative Vitreoretinopathy (PVR): The first Phase 3 clinical trial of ADX-2191, acquired this year for the prevention of PVR, is expected to begin in 2019. PVR is a rare inflammatory fibroproliferative disorder that leads to severe retinal scarring and blindness and is the leading cause of failure of retinal reattachment surgery. Over 50% of PVR cases result in severe uncorrectable vision loss, and 75% of PVR patients suffer from at least moderate uncorrectable vision loss. With no currently approved therapy available, PVR is a serious and sight-threatening disease that effects approximately 4,000 patients in the United States and nearly twice as many in Europe and Japan. Aldeyra plans to begin a two-part, multi-center, non-masked, randomized, controlled, adaptive Phase 3 clinical program in the second half of 2019, following discussions with regulatory authorities. ADX-2191 has received Orphan Drug Designation for the prevention of PVR. |
| • | Reproxalap for Dry Eye Disease (DED): In September 2018, Aldeyra reported Phase 2b results in DED that demonstrated statistical superiority of reproxalap versus vehicle across multiple DED symptoms and signs. Based on these results, Aldeyra plans to initiate Part 1 of a two-part adaptive Phase 3 clinical trial in the first half of 2019. Part 1 of the clinical trial will evaluate the efficacy of reproxalap ophthalmic solution (0.25%) vs. vehicle in 400 patients with moderate-to-severe DED. Results from Part 1 will confirm dosing and size for Part 2 of the Phase 3 clinical trial. The co-primary endpoints of this trial will be ocular dryness, and fluorescein nasal region staining in pre-specified moderate to severe patient subsets analyzed over twelve weeks of therapy using Mixed effects Model Repeated Measures (MMRM). In the Phase 2b clinical trial, the MMRM p values for the Phase 3 co-primary endpoints of dryness and staining were 0.0048 and 0.0007, respectively. DED impacts approximately 20 million adults in the United States and represents a highly underserved patient population with up to 50% of patients discontinuing treatment due to limited efficacy or slow onset with current treatment options. |
| • | Reproxalap for Allergic Conjunctivitis (AC): Aldeyra expects to report results of the Phase 3 ALLEVIATE trial in early 2019. ALLEVIATE is a multi-center, double-masked, parallel-group, vehicle-controlled Phase 3 clinical trial that will measure ocular itch score area under the curve and patient responder rate. In preparation for a subsequent Phase 3 clinical trial, Aldeyra is also conducting clinical method development studies to assess the feasibility of measuring ocular itch following environmental exposure to allergen. Allergic conjunctivitis represents a large and underserved market with an estimated 30 million patients in the United States who are inadequately treated with the current standard of care. In two Phase 2 clinical trials, reproxalap was observed to be well tolerated and demonstrated the potential to be effective in post-histaminic allergy, for which no drug is approved, and which affects all patients suffering from allergic conjunctivitis. |
| • | Reproxalap for Overlapping Treatment of Dry Eye Disease and Allergic Conjunctivitis: Studies have shown that DED and AC are interrelated, with up to 50% of the patient population suffering from DED/AC comorbidity. Reproxalap has demonstrated efficacy against both DED and AC in separate Phase 2b clinical trials. Aldeyra plans to advance parallel Phase 3 programs in DED and AC that could support concurrent New Drug Application filings with the U.S. Food and Drug Administration for both conditions. |
| • | Reproxalap – Noninfectious Anterior Uveitis (NAU): NAU is a rare ocular disease caused by an inflammatory response that leads to surface irritation, pain, photophobia, and in some cases vision loss, and affects approximately 260,000 patients in the United States per year. An estimated 50% of patients suffer from repeat or chronic NAU episodes, increasing the risk of serious ocular toxicity as a result of prolonged exposure to corticosteroids, the current standard of care. The anti-inflammatory product profile of reproxalap has the potential to treat patients without the toxicities commonly associated with corticosteroids. The Phase 3 SOLACE clinical trial of reproxalap in NAU is currently ongoing, and results are expected to be announced in the second half of 2019. |
About Aldeyra Therapeutics
Aldeyra Therapeutics is a biotechnology company devoted to developing and commercializing next-generation medicines to improve the lives of patients with immune-mediated diseases. Aldeyra’s lead product candidate, reproxalap, is a first-in-class treatment in late-stage development for dry eye disease, allergic conjunctivitis, noninfectious anterior uveitis, and Sjögren-Larsson Syndrome. The company is also developing other product candidates for proliferative vitreoretinopathy and other retinal diseases, post-transplant lymphoproliferative disease, autoimmune disease, metabolic disease, and cancer. None of Aldeyra’s product candidates have been approved for sale in the U.S. or elsewhere.
Safe Harbor Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding Aldeyra’s strategy, future operations, future, prospects, plans, and objectives and Aldeyra’s plans and expectations for its product candidates, including plans to initiate further clinical testing, the timing of results from clinical programs, and its rgulatory plans. Aldeyra intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “target,” “design,” “estimate,” “predict,” “potential,” “aim,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. All of Aldeyra’s development timelines may be subject to adjustment depending on recruitment rate, regulatory review, preclinical and clinical results, and other factors that could delay the initiation or completion of clinical trials. Important factors that could cause actual results to differ materially from those reflected in Aldeyra’s forward-looking statements include, among others, the timing of
enrollment, commencement and completion of Aldeyra’s clinical trials, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; updated or refined data based on Aldeyra’s continuing review and quality control analysis of clinical data, Aldeyra’s ability to design clinical trials with protocols and endpoints acceptable to applicable regulatory authorities, the ability to obtain and maintain regulatory approval of Aldeyra’s product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Aldeyra’s product candidates; the size and growth of the potential markets and pricing for Aldeyra’s product candidates and the ability to serve those markets; Aldeyra’s expectations regarding Aldeyra’s expenses and revenue, the sufficiency or use of Aldeyra’s cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra’s product candidates; Aldeyra’s expectations regarding competition; Aldeyra’s anticipated growth strategies; Aldeyra’s ability to attract or retain key personnel; Aldeyra’s ability to establish and maintain development partnerships; Aldeyra’s expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries; Aldeyra’s ability to obtain and maintain intellectual property protection for its product candidates; the anticipated trends and challenges in Aldeyra’s business and the market in which it operates; and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Aldeyra’s Annual Report on Form 10-K for the year ended December 31, 2017 and Aldeyra’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, both of which are on file with the Securities and Exchange Commission (SEC) and available on the SEC’s website at www.sec.gov. Additional factors may be set forth in those sections of Aldeyra’s Annual Report on Form 10-K for the year ended December 31, 2018, to be filed with the SEC in the first quarter of 2019.
In addition to the risks described above and in Aldeyra’s other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra’s results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
Corporate Contact:
David McMullin
Aldeyra Therapeutics, Inc.
Tel: 781-761-4904 ext. 218
dmcmullin@aldeyra.com
Investor Contact:
Chris Brinzey
Westwicke Partners
Tel: 339-970-2843
Chris.brinzey@westwicke.com