Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net

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Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Driven by Science
Inspired by Hope
Corporate Highlights

October | 2019
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Forward-Looking Statements

     This presentation may include forward-looking statements made pursuant to the Private Securities Litigation
     Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking
     statements are subject to significant risks and uncertainties that are subject to change based on various factors
     (many of which are beyond Cerecor’s control), which could cause actual results to differ from the forward-looking
     statements. Such statements may include, without limitation, statements with respect to Cerecor’s plans,
     objectives, projections, expectations and intentions and other statements identified by words such as “projects,”
     “may,” “will,” “could,” “would,” “should,” “continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,”
     “anticipates,” “estimates,” “intends,” “plans,” “potential,” or similar expressions (including their use in the
     negative), or by discussions of future matters such as: our 2019 outlook; the development of product candidates;
     timing and success of trial results and regulatory review (including as it may be impacted by government shut-
     downs); potential attributes and benefits of product candidates; the expansion of Cerecor’s drug portfolio; and
     other statements that are not historical.

     These statements are based upon the current beliefs and expectations of Cerecor’s management but are subject
     to significant risks and uncertainties, including: reliance on and the need to attract, integrate and retain key
     personnel; drug development costs, timing and other risks; Cerecor’s cash position and the potential need for it to
     raise additional capital; risks associated with acquisitions, including the need to quickly and successfully integrate
     acquired assets and personnel; and those other risks detailed in Cerecor’s filings with the Securities and Exchange
     Commission. Actual results may differ from those set forth in the forward-looking statements. Except as required
     by applicable law, Cerecor expressly disclaims any obligations or undertaking to release publicly any updates or
     revisions to any forward-looking statements contained herein to reflect any change in Cerecor’s expectations with
     respect thereto or any change in events, conditions or circumstances on which any statement is based.

2|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Overview

            1   Cerecor Transformation

            2   Cerecor Today

            3   Orphan & Neurological Pipeline

            4   Financial Highlights

            5   Strategic Growth Plans and Outlook

3|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
The New Cerecor
   Creating Near-Term Shareholder Value

  • 3 Completed
    Phase I Trials                                                                        CERC-800s 505(b)(2)
                                                                                3
                                                 Clinical-Stage                           programs for CDGS
  • Proof-of-Concept                                                     Fast-to-Market
                                                     Biotech               Programs       • NDA submissions in
    in patients
                                                                                            next 12 to 18 months
  • 2 Fast-Track
    Designations

                                                                                          CERC-800s granted
                                                     Novel                    3           RPDD from FDA
  • CERC-301 in OH                                                                          • Market value
                                                  Neurological            PRV-eligible
  • CERC-406 in PD                                  Pipeline               Programs           approximately
                                                                                                 $300M
                                                                                            ($80 to $130M)*

   RPDD = Rare Pediatric Disease Designation
4| Last 5 individual PRV’s sold between $80M and $130M; 2017 thru 2019
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Management Team
     Extensive Experience in Development and Commercialization

                         30+ years industry experience                                      20+ years industry experience
                         • President and CEO of Chelsea                                     • V.P. Global CMC & Development, Sucampo
                           Therapeutics                                                       Pharmaceuticals
      Dr. Simon          • Chief Business and Strategy Officer,        Dr. Pericles         • CSO, Pharming Group
       Pedder              Proprietary Products at Athenex                Calias            • Sr. Director Rare CNS Diseases and Device
                         • Vice President of Oncology Pharma                                  Lead, Shire plc
        Executive          Business at Hoffmann-LaRoche                     Chief
                                                                                            • Sr. Director Drug Delivery and Chemistry,
     Chairman of the                                                   Scientific Officer
                         • Franchise Head of Hepatitis at                                     Eyetech Pharmaceuticals
          Board                                                         & Head of R&D
                           Hoffmann-LaRoche                                                 • Ph.D., Tufts University, Bioorganic
                         • Faculty Department of Pharmacology                                 Chemistry
                           College of Medicine, University of
                           Saskatchewan

                         20+ years industry experience                                      25+ years industry experience
                         • Vice President of Finance, Sucampo                               • Sr. Vice President | Principal
                           Pharmaceuticals                                                    The NSCI Group
        Joseph           • Senior Director of Accounting, Qiagen         James A.           • General Manager Specialty
         Miller          • Chief Financial Officer, Eppendorf 5Prime      Harrell             Pharmaceuticals, Covidien
                         • Certified Public Accountant                      Chief           • Vice President Marketing Pediatric
          Chief                                                                               Infectious Disease, MedImmune
                                                                         Commercial
     Financial Officer
                                                                           Officer          • Sr. Director Marketing IMIDs, Centocor a
                                                                                              J&J Company
                                                                                            • Product Manager & Hospital Specialist,
                                                                                              ATOD Rhone Poulenc Rorer

5|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Transforming Cerecor to Deliver Shareholder Value
     Three Expected Orphan Disease Product Launches Within the Next Three Years
     a Neurological Pipeline in Markets of Substantial Size and Unmet Need

                     2017                      2018                           2019
 2016
 CERC-301
 misses
 primary           Acquisition               Acquisition               Pipeline Progression
 endpoint
 in Phase II       TRx Pharma                Avadel (AVDL)          CERC-301 IP extended to 2035,
 in Major            (Zylera)               Pediatric assets          positive Phase I PoC in nOH
 Depressive
 Disorder                                                               CERC-800s receive ODD
                                                                     CERC-801 & 802 receive FTD,
                                                                       completed Phase I in HVs

                   Out-license               Acquisition                  Asset Divestiture
               CERC- 501 licensed to           Ichorion                CERC-611 out-licensed to
                     Janssen             Rare Disease Pipeline             ES Therapeutics

                                                                         Pediatric Commercial
                                        Pipeline Progression          Portfolio sold for > $43M in
                                                                               total value
                                          CERC-301 re-purposed
                                       in Orthostatic Hypotension
                                        CERC-800s receive RPDD
6|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Recent Transaction Strengthens Balance Sheet
     Sale of Pediatric Portfolio Extends Financial Runway and Provides Non-Dilutive
     Financing for R&D

                                  Cerecor Transaction Highlights
     • Overall deal valued in excess of $43M
        – Composite of $17M in cash and preferred stock ($4.5M in cash & 12.5M of AYTU stock)
     • Assumption of Cerecor’s outstanding payment obligations payable to Deerfield CSF,
       LLC (“Deerfield Note”) and other liabilities in excess of $15 million
     • Elimination of existing royalty obligations & various commercial accruals of $11 million
     • The Pediatric Portfolio includes the following five product lines: Aciphex® Sprinkle™,
       Cefaclor for Oral Suspension, Karbinal® ER, Flexichamber™, Poly-Vi-Flor® and
       Tri-Vi-Flor™
     • Estimated annual expense reduction of $7 to $9 million associated with Commercial
       Sales organization transfer to AYTU
     • Company to fund its portfolio of development assets focusing on near-term value
       drivers; including NDA submissions for CERC-800s and the expansion of the CERC-301
       program

7|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Cerecor Today
     Focused on Advancing Emerging Clinical & Early-stage Pipeline to Critical Value
     Inflection Points

         Pediatric Orphan Disease Pipeline                    Neurological Pipeline
     • Substrate replacement therapies for Inborn   • Novel mechanisms-of-action with clinical
       Errors of Metabolism (IEMs)                    proof-of-concept and opportunity for strong
                                                      clinical differentiation
     • Three 505(b)(2) programs granted ODD and
       RPDD (PRV eligible)                          • Completed Phase I program demonstrating
                                                      robust and sustained BP effect in patients
     • Expedited path(s) to NDA with three            suffering from neurogenic Orthostatic
       anticipated NDA filings in 2021 & 2022         Hypotension (nOH)
     • Two completed Phase I programs with Fast     • Expanding clinical development in OH
       Track Designation (FTD)                        adjacencies of Diabetic OH (DOH) and
     • CDG FIRST retrospective study ongoing          Intradialytic Hypotension (IDH)
                                                    • Neurological markets with large commercial
                                                      opportunity & significant unmet needs

8|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Emerging Clinical & Early-Stage Pipeline
                                              Mechanism              Lead
                                Program                                                           Development Stage
                                               of Action          Indication
                                              D-Galactose
                               CERC-801                          PGM1-CDG         Phase 1 Complete                505(b)(2)
                                              replacement
       Metabolic Disorders

                                               D-Mannose
                               CERC-802                           MPI-CDG         Phase 1 Complete           505(b)(2)
                                              replacement

                                                L-Fucose
                               CERC-803                         SLC35C1-CDG       IND-Enabling        505(b)(2)
                                              replacement
                                               Nucleoside          DGUOK
                               CERC-913                                           Pre-Clinical
                                              replacement         Deficiency
                                            NMDA receptor        Orthostatic
                               CERC-301
     Neurology

                                                                                  Phase 1 Complete in nOH
     Disorders

                                             antagonist          Hypotension
                                              CNS-targeted       Parkinson’s
                               CERC-406                                           Pre-Clinical
                                             COMT inhibitor       Disease

                             Denotes Pediatric Program and Expedited 505(b)(2) Approval Pathway
                             Denotes Pediatric Program
                             Denotes Neurology Program

9|
Driven by Science Inspired by Hope - Corporate Highlights October | 2019 - cloudfront.net
Pipeline Provides Strategic Optionality

                                                 World-Wide Estimated Patient Populations
                                                                                                                                         Clinical
                                                                                                                240M
                              Develop Internally &                                                                                  Proof-of-Concept
                                                                                                                185M
                           Commercialize Independently                                                          OH >65              Toward Strategic
                                                                                                                                      Partnership
                                    Orphan
                               Pediatric Diseases                                                                                      Larger CNS
                                                                                                                                       Indications
                                                                                                                 50M
                                                                                                               Diabetic
                                                                                                                  OH
                                                                                                                                      1.9M
                                                                                                                 2.9M
                                                                                                                SCI OH

                                                                                                                 1.7M
               500                                                                       500                     nOH
                                        250                       50                                             655k
                                                                                                                 IDH

          CERC-801                 CERC-802                CERC-803                 CERC-913                CERC-301               CERC-406
         PGM1-CDG                   MPI-CDG              SLC35C1-CDG                 DGUOK                 Orthostatic             Parkinson’s
                                                                                    Deficiency             Hypotension               Disease

10| IDH = Intra-Dialytic Hypotension; nOH = Neurogenic Orthostatic Hypotension; SCI = Spinal Cord Injury; DM = Diabetes Mellitus
R&D Milestones
    Multiple Potential Value-Generating Inflection Points Over Next 12 to 18 Months

                         Program      Target Indication         Upcoming Milestone

                                                               FDA Meeting Request YE19
                    CERC-801*            PGM1-CDG
                                                             Targeted NDA Submission 2021
    Metabolic
    Disorders

                                                               FDA Meeting Request 1H20
                    CERC-802*             MPI-CDG
                                                             Targeted NDA Submission 2021

                                                                    IND Filing 2020
                    CERC-803*           SLC35C1-CDG
                                                             Targeted NDA Submission 2022

                                                               Initiate Proof-of-Concept in
                     CERC-301      Orthostatic Hypotension    Additional Indication(s) YE19
    Neurology
    Disorders

                                                                   Initiate Phase II 2020

                     CERC-406        Parkinson’s Disease            IND Filing 2020

11| *505(b)(2) Pathway
Pediatric Orphan Disease Portfolio
CERC-800s
CERC-913
Congenital Disorders of Glycosylation (CDG)
      Genetic Diseases that Result in Impaired Glycoprotein Production and Function,
      but are Treatable with Substrate Replacement Therapy

                Glycosylation is essential for protein                                      Factor XI
                structure & function, particularly for                                     Thrombin
                circulating proteins and enzymes such                                    Liver enzymes
                as hormones and coagulation factors                                       Hemoglobin

                Some CDG patients lack the ability to
                effectively utilize monosaccharide            Normal Glycosylation
                                                          3
                                                              Pattern
                substrates for glycoprotein synthesis
                due to a single enzyme defect

                Substrate replacement with
                monosaccharides can compensate for                            Substrate replacement
                                                                          2
                                                                              w/ oral monosaccharides
                enzyme deficiencies

                Treatment with oral monosaccharides
                results in improvement in serum
                biomarkers of glycosylation, which can
                correlate with clinical benefit               Glycosylation Deficiency
                                                          1
                                                              due to CDG

13|
CERC-800s
      Substrate Replacement Therapies for CDGs
      Monogenic Disorders Resulting in Glycoprotein Defects with Broad Clinical Spectrum,
      Including Life-threatening Complications

                    Multi-system disease manifestation in PGM1-CDG
       CERC-801     D-Galactose leads to significant improvement in key clinical symptoms

                    Life-threatening gastrointestinal disorder in MPI-CDG
       CERC-802     D-Mannose rapidly resolves hematological & intestinal abnormalities

                    Immunodeficiency with CNS impairment in SLC35C1-CDG
       CERC-803     L-Fucose normalizes cell counts & reduces infection risk

               Ultra-orphan IEMs with Serious and Life-threatening Medical Needs

        • Symptoms evidenced by serum                 •
CERC-800s
      Substrate Replacement Therapies for CDGs
      Oral, Small Molecule, Naturally Occurring Monosaccharides Used as Standards-of-care
      for CDGs

      • Rapid onset of action
      • Safe and well-tolerated
      • Symptoms return upon
        treatment withdrawal
                                        D-Galactose        D-Mannose          L-Fucose

                Eligibility              CERC-801          CERC-802          CERC-803

      505(b)(2) NDA Pathway                  ✓                 ✓                 ✓
      NCE 5-yrs Exclusivity                  ✓                 ✓                 ✓
      FDA ODD 7-yrs Exclusivity              ✓                 ✓                 ✓
      EMA ODD 10-yrs Exclusivity             ✓                 ✓                 ✓
      Priority Review Voucher                ✓                 ✓                 ✓
15|
The Value of a Priority Review Voucher

                                 Recent PRV Sales with Purchase Amount (in millions)

                                                               The Mean purchase price over
                                                              the past 5 years has been $140M
            $200
                                                                                                The Median purchase price over
                                                                                                the past 5 years has been $125M

                              $125               $130              $125       $130
                                                                                        $110                           $105

                                                                                                $81         $80

            2016              2016               2017              2017       2017      2017    2018       2018        2019

16| Reference: https://priorityreviewvoucher.org/ downloaded April 24, 2019
CDG FIRST: Cerecor-Initiated Retrospective Study in CDG
      Retrospective Chart Reviews & Registry Data Have Been Successfully Used to Minimize
      or Obviate Prospective Clinical Studies

          Developer       Therapeutic (Indication)                        Pivotal Study Strategy
                                                            Retrospective case series summary (13/23 patients
                                  Carbaglu
                                                            with complete data) & 3 patients treated
                              (NAGS Deficiency)
                                                            prospectively (2010)
                                                            Case report form from retrospective chart review of
                                   Cholbam
                                                            patients in open-label, single-arm Expanded Access
                             (Bile Acid Disorders)          Protocol (2015)
                                                            Retrospective case reports from a multicenter chart
                                  ProVay Blue
                                                            review in addition to cases found in published
                        (Acquired Methemoglobinemia)
                                                            literature (2016)
                                                            Expanded label (from 10 mutations to 33) based on
                                  Kalydeco
                                                            registry data & mechanistic information from lab
                               (Cystic Fibrosis)            studies (2017)
                                                            Reference to survival data from an international
                                 Lumizyme
                                                            registry of infantile-onset disease demonstrating
                              (Pompe Disease)
                                                            mortality benefit (2010)

                                                   CDG Connect Patient Insights Network (PIN)
                                                      https://connect.invitae.com/org/cdg

17|
CDG FIRST Global Site Selection & Recruitment
      Multi-center, International, Non-interventional, Retrospective Study of CDG Patients to
      Collect Natural History and Treatment-related Data

                     North America                                         Europe
      Cerecor plans to leverage data from CDG FIRST Trial and other sponsor-initiated
       studies to accelerate development and approval under the 505(b)(2) pathway,
                     with our targeted NDA filing for CERC-801 in 2021

                                           CDG Connect Patient Insights Network (PIN)
                                              https://connect.invitae.com/org/cdg

18|
Key CERC-800s Milestones
    Multiple Potential Value-Generating Inflection Points Over Next 12 to 18 Months
    • Type B Meetings with FDA to clarify path to NDA
              – Briefing package expected to include data collected under CDG FIRST
              – Determine if pivotal study requirement and/or design

                             Program           Target Indication               Upcoming Milestone

                                                                         • Type B Meeting 2020
                           CERC-801
                                                   PGM1-CDG              • BTD Request 2020
                         (D-Galactose)
                                                                         • NDA Submission 2021
       505(b)(2) NCEs

                                                                         • Type B Meeting 2020
                            CERC-802                MPI-CDG              • BTD Request 2020
                          (D-Mannose)
                                                                         • NDA Submission 2021
                                                                         • IND Filing 2020
                            CERC-803
                                                  SLC35C1-CDG            • BTD Request 2020
                            (L-Fucose)
                                                                         • NDA Submission 2022

19| BTD = Break Through Therapy Designation
Deoxyguanosine Kinase (DGUOK) Deficiency
     Ultra-rare, Inborn Error of Metabolism Due to Loss-of-function Mutations in DGUOK
     • Inability to produce deoxyguanosine monophosphate (dGMP) results in depletion of
       mitochondrial DNA (mtDNA), leading to organ dysfunction

     • Most frequently mutated gene in patients with early hepatocerebral mtDNA depletion
       syndrome; estimated prevalence of 1 in 500,000

     • Severe depletion (>90%) of mtDNA relative to healthy individuals

     • Two forms: neonatal multisystem disorder that presents in early infancy and isolated
       hepatic disorder that presents later in infancy or childhood

     • Liver involvement is the most prominent feature, leading to liver cirrhosis and causing
       early-onset liver failure

     • Liver transplant can benefit patients with isolated hepatic disease, but less so for
       patients with moderate to severe neurological symptoms

     • 5-Year Overall Survival
CERC-913
      dGMP ProTide for DGUOK Deficiency
      Overcome Key Limitations of Direct Substrate Replacement
      • The ProTide nucleotide offers plasma stability, improved permeability and
        intracellular activation

               dGMP
          Unstable in Plasma
           Poor Permeability

                                                                                    Phenotypic
                                                                                      Rescue

             CERC-913
           Stable in Plasma
          Good Permeability

21|
Neurological Disorders
CERC-301
CERC-406
Orthostatic Hypotension (OH)
      Autonomic Nervous System Fails to Regulate Vasoconstriction Due to Underlying
      Neurological Disease
                 Rapid and Significant (20mm Hg) Drop in Systolic Blood Pressure (SBP)
                                      Induced by Postural Change
      • Increased risk of falls or injury, leading to decreased quality of life
      • Estimated approximately 300k patients in U.S. and 1.5mm WW

                   Two FDA-Approved Therapies, Northera® (droxidopa) & Midodrine
                          Northera®                                                Midodrine
      • Norepinephrine pro-drug acting upon                     • Vasoconstrictor / Hypertensive Agent
        adrenergic receptors                                    • α-adrenergic receptor agonist producing an increase
      • Effectiveness beyond 2 weeks of treatment                 in vascular tone and elevation of blood pressure
        has not been established                                • Pro-drug with half-life ~25 minutes
      • High rate (>1/3) of non-responders                      • Dosing Q6 to Q8 Daily
      • Estimated 40K TRx Annually in the US Jun’19-Jul’18      • Estimated 1.6M TRx Annually in the US Jun’19-Jul’18
      • Estimated Gross $349M Jun19-Jul’18                      • Estimated Gross $457M in Annual Sales Jun’19-Jul’18

                                    CERC-301 (Formerly Known as MK-0657)
      • Established safety profile in ~400 patients and healthy volunteers
      • Several studies demonstrated rapid and sustained increase in SBP without an increase
        in heart rate
23|
Phase I Study Design in PD Patients with nOH
      Opportunity to Efficiently Demonstrate Proof-of-concept in Patients

                 Phase I SAD in PD Patients with nOH                     Visit 1   Visit 2   Visit 3   Visit 4 Visit 5a

      Enrollment           • 12 active centers in US           Arm 1      pbo      8 mg      12 mg     16 mg    20 mg
                                                               (n = 5)
                           • N = 20 (8, 12, 16 & 20 mg)
                                                               Arm 2
                           • Double-blind, randomized,                    8 mg      pbo      12 mg     16 mg    20 mg
      Design                                                   (n = 5)
                             pbo-controlled
                           • Interim Analysis at 10 patients   Arm 3
                                                                         8 mg      12 mg      pbo      16 mg    20 mg
                                                               (n = 5)
                           • Safety, Tolerability & PK
      Endpoints            • BP measurement                    Arm 4
                                                                          8 mg     12 mg     16 mg      pbo     20 mg
                           • Symptomatic assessment            (n = 5)

      •     5 visits (7 to 10 days apart), 4 single escalating doses of CERC-301 or placebo
      •     Assess safety, tolerability & pharmacokinetics
      •     Measure BP effects during orthostatic standing test (OST) pre- and post-dose
      •     Symptomatic assessment (OHSA Item #1) at each visit

24|   a   Randomized 4:1
CERC-301 Improves Blood Pressure Upon Standing
              20mg dose results in +29.8mm Hg increase in standing SBP over placebo at 6-hour post-
              dose during an Orthostatic Sanding Test

                                                  20mg vs Placebo
                                             pre-dose vs 6-hours post-dose
              155              Semi-Supine                          Standing

                                                                      +29.8
SBP (mm Hg)

              135

              115
                     CERC-301 reduces the
                     magnitude of SBP drop
                       following an OST
               95

                     SBP       SBP        SBP       SBP      SBP       SBP         SBP       SBP
                    Seated -10 mins     -5 mins Imed Prior +1 min     +3 min     +5 mins Seated
                       Placebo - PD        20 mg - PD       Placebo - 6hr         20 mg - 6 Hr

25|
CERC-301 PK Data
      Consistent and Predictable Pharmacokinetic Profile Across All Doses Studied

                                  CERC-301 Plasma Concentrations
              450

              400

              350

              300

              250
                                                                                    8 mg
      ng/Ml

              200                                                                   12 mg
                                                                                    16 mg
              150                                                                   20 mg

              100

               50

                0
                    0   0.5   1       2                           4             6
              -50
                                          Nominal Time in Hours

26|
Positive Phase I Data in nOH
      Final Phase l Results Depict a Rapid, Robust and Durable Increases in SBP
      in PD Patients with nOH

      • All doses demonstrated clinically meaningful increases in SBP over placebo within the
        6-hour post-dose timepoints
         – 20mg dose demonstrated a 15.2 mm Hg increase in SBP from baseline at 1-hour post-dose,
           which improved to 29.1 mm Hg from baseline at 4 hours
         – Early and sustained effect may differentiate CERC-301 from existing nOH / OH treatments

      • All doses tested were safe and well-tolerated with no serious adverse events

      • Opportunity to develop CERC-301 for additional indications characterized by
        hypotension (i.e. diabetics, hemodialysis, elderly populations)
         – Northera® limited to norepinephrine-deficient patient population(s)
         – CERC-301’s mechanism-of-action is potentially more broadly applicable
         – Initiate additional proof-of-concept study in OH population(s) by YE19

27|
CERC-301: A Pipeline Within a Product
     CERC-301’s Differentiated Profile Expands Addressable Patient Population to Additional
     Diseases Characterized by Low Blood Pressure
           Neurogenic Orthostatic                                Diabetic Orthostatic             Intradialytic Hypotension
               Hypotension                                          Hypotension                              (IDH)
         Estimated Patient Population                         Estimated Patient Population      Estimated Patient Population
                                                                                  46.2M
                                                                   3.0M

                                       1.5M

               220K                                                                                  250K               405K

             US               ROW                                    US               ROW               US               ROW
         Phase I Demonstrated Rapid,                           Initiating Phase I in Patients      Initiating Phase II in Patients
         Robust & Sustained BP Effect                                    by YE 2019                            in 2020
                  in Patients

     • Large target addressable markets with limited therapeutic options
           – Northera® and midodrine act as direct adrenergic agonists with narrow clinical utility, resulting
             in unmet medical needs
           – Clinical proof-of-concept throughout 2019 and 2020 will create an opportunity for strategic
             partnership(s)

28| Population estimates at mean values from the literature
Phase I Study Design in Diabetic OH
      Further Assess Durability of BP Effect to Facilitate Phase 2 Study Design

                                                            OST Performed @ 1, 2, 4 , 6, 12 and 24
      Phase I Single Dose Study in Diabetic OH                        Hours Post-dose

      Enrollment           • N = 10, single center                          Postural Change

                           • Single 20 mg dose or placebo          Supine                  Standing
      Design               • Double-blind, randomized,      -10      -5      0       +1       +3      +5
                             two-way crossover              min      min    min      min      min     min

                                                             X        X      X        X       X       X
                           • Safety (HbA1c monitoring )
                           • Tolerability & PK
      Endpoints                                                  X = measure systolic/diastolic blood
                           • BP measurement
                           • Symptomatic assessment                   pressure and heart rate

      • 2 visits (7 to 10 days apart), single 20mg dose of CERC-301 or placebo
      • Assess safety, tolerability & pharmacokinetics
      • Measure BP effects before and after an OST for up to 24 hours post-dose
      • Symptomatic assessment (OHSA Item #1) at each visit

29|   a Randomized   4:1
Phase 2 Study Design in Intradialytic Hypotension
      Well-defined Clinical Setting Facilitates Recruitment, Shared Opportunity for
      Hemodialysis (HD) Centers with Expected Study Start in 2020
      • Clinical Endpoint: change in the Number of Hypotension-induced Interventions During
        Hemodialysis (HD) Sessions
                                                         N = 75

                                                                  12mg /20mg 301 Group A
                                         Randomization

                                                                                                      End of Study
          2-week lead-in baseline                                 12mg/20mg 301 Group B
             evaluation period
          A = dose 1 hour prior to HD
          B = dose 2 hours prior to HD
                                                                      Placebo Group

                                                                   4-week treatment period
              Week 1         Week 2                      Week 3    Week 4      Week 5        Week 6
                     12 Week Study Duration with Hemodialysis 3x / week (18 sessions)

30|   a   Randomized 4:1
CERC-406
      COMT inhibitor for Parkinson’s Disease
      Highly-Selective with Greater CNS Permeability for Potentially Improved Efficacy
      • COMT regulates dopamine (levodopa) breakdown via the conversion of
        dihydroxyphenyl-acetic acid (DOPAC) to homovanillic acid (HVA)

       1st Gen COMT       Drawbacks /                                               CSF Biomarkers:
         Inhibitors      Marketing Status                               Single, 100 mg/kg CERC-406 Dose in Rats

                                                                              DOPAC                                           HVA
                        black box warning
        Tolcapone                                                     300                                         120
                             generic
                                                                      250                                         100

                                               DOPAC (% of Control)
                                                                                      **

                                                                                             HVA (% of Control)
                           peripherally-                              200                                          80
       Entacapone           restricted
                                                                      150                                          60
                             generic
                                                                      100                                          40
                                                                                                                                    **
                           peripherally-                               50                                          20
        Opicapone           restricted
                          NDA Filed 7/19                                0                                           0
                                                                            Veh   CERC-406                              Veh     CERC-406

31|
Key Financial Information
Financial and Investor Information
      Key Financial Indicators as of October 3, 2019 (Subject to Change)

                 Stock Information                           Investor Information
      • Founded January 31, 2011                   • 44.1M Shares Outstanding
      • NASDAQ listed CERC                         • Analyst Coverage (12 month target price)
      • IPO – October 16, 2016                        – Michael Higgins Ladenburg Thalmann ($9.00)
                                                      – Jason McCarthy Maxim ($10.00)
      • Initiated at $6.50
                                                      – Ram Selvaraju HC Wainwright ($11.00)
         – 4M Units @ $6.50 for $26M
                                                   • Added to Russell 3000 Index July 1, 2019
      • Stock Range (52 weeks 10/3/19)
         – 52 week high $7.66                               Financial Information
         – 52 week low $2.71
                                                   • Cash Position June 30, 2019 = ~$10M
      • Average Volume 80,390
                                                   • Ongoing Revenue from Millipred®
      • Market Cap in excess of $130M                 – Estimated 2019 Gross Margin of $5–$6M
                                                      – Provides Strategic Optionality

33|
2019 Growth Plans
      Recent Events Increase Shareholder Value by Strengthening Balance Sheet to Prioritize
      Pipeline Development

                   1                               2                            3

               Advance                                                 Accelerate Business
                                       Commercial Readiness
               Pipeline                                               Development Activity

         Advance CERC-800s to        Build commercial footprint for    Out-license or partner
         NDA filings and launch       pediatric orphan disease in       indications and/or
                                      preparation for 2021 launch           geographies
         Provide further clinical
           proof-of-concept in        Increase market knowledge          Acquire/in-license
      adjacencies for CERC-301 and      and insights for pricing /        complimentary
        initiate Phase 2 program          reimbursement and               pipeline assets
                                         distribution strategies
      Advance CERC-406 and CERC-
         913 into Phase I studies    Develop go-to-market strategy
                                            for CERC-800s

34|
Driven by Science
Inspired by Hope
NASDAQ:CERC
www.cerecor.com
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