Patient Inspired Science Establishing a leading, rare and orphan disease-focused biopharmaceutical company to deliver impactful new medicines to patients H.C. Wainwright & Company 22nd Annual Global Investment Conference September 14, 2020 September | 2020 Corporate Highlights | Focus on COVID-19 ARDS
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, Inc. (“Cerecor”) 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,” “might,” “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 2020 outlook; the development of product candidates or products; potential attributes and benefits of product candidates; strategic alternatives for neurological assets and Millipred; 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 integration of key personnel; drug development costs, timing and other risks, including reliance on investigators and enrollment of patients in clinical trials, which might be slowed by the COVID-19 pandemic; regulatory risks; Cerecor's cash position and the need for it to raise additional capital; risks related to potential strategic alternatives for our neurology assets and Millipred; general economic and market risks and uncertainties, including those caused by the COVID-19 pandemic 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|
Highlights • Recent Merger of Cerecor and Aevi has created a rich pipeline of novel, 1 st in class assets all with proven mechanistic rationale • Only known anti-LIGHT mAb in the clinic, offers potential to treat cytokine storm-induced COVID-19 ARDS in the near-term and broader ARDS indication beyond – CERC-002 entered the clinic in July for ARDS and is expected to deliver definitive topline POC data in Q4 2020 – CERC-002 resumed clinical trial for the treatment of severe pediatric onset Crohn’s disease in August • CERC-007 (anti-IL-18 mAb), unique molecular target, is expected to deliver initial data in multiple myeloma 1Q 2021 and top line data 2Q 2021; initial data in adult onset Still’s Disease by 2Q 2021. • CERC-006 (dual mTOR inhibitor), topline data expected 2Q 2021 and recently received ODD and RPDD from the FDA (August 2020) • CERC-800 series are monosaccharide replacement therapies for congenital disorders of glycosylation; all orphan disease designated; rare pediatric disease designated; with Priority Review Voucher(s) eligibility – CERC-801 pivotal trial expected start 4Q 2020, top line data expected 4Q 2021 – CERC-802 pivotal trial expected start 4Q 2020, top line data expected 3Q 2021 – CERC-803 pivotal trial expected start 1H 2021, top line data expected 2H 2021 3|
Clinical-Stage Pipeline Development Stage Core Research & Therapeutic Mechanism Upcoming Development Program Lead Indication Area of Action Pivotal Milestone Areas Preclin Phase 1 Phase 2 Trial Initial Data CERC-002 Anti-LIGHT mAb ARDS 4Q 2020 Severe Pediatric Initial Data CERC-002 Immunology Inflammation Anti-LIGHT mAb Onset Crohn’s 1Q 2021 Initial Data CERC-007 Anti-IL-18 mAb AOSD 2Q 2021 Multiple Oncology CERC-007 Anti-IL-18 mAb Blood Cancers Initial Data 1Q21 Myeloma Complex Complex Dual mTOR Initial Data CERC-006 Lymphatic Lymphatic inhibitor 2Q 2021* Malformations Malformations D-Galactose Pivotal Trial CERC-801 PGM1-CDG replacement Data 2021 Rare Genetic Disorders Congenital D-Mannose Pivotal Trial CERC-802 MPI-CDG Disorders of replacement Data 2021 Glycosylation L-Fucose CERC-803 LADII-CDG IND 2H20 replacement 4| *Milestone currently under evaluation due to COVID-19 related CMC availability
CERC-007 Phase 2-ready, anti-IL-18 monoclonal antibody for Multiple Myeloma & Adult Onset Still’s Disease
Phase II-Ready Asset for Multiple Myeloma (MM) and Adult Onset Still’s Disease (AOSD) First-in-class, only fully human anti-IL-18 mAb with potential to address multiple tumor types and inflammatory conditions Strong • Elevated IL-18 is correlated with poor survival in MM patients and disease Scientific severity in AOSD patients Rationale Need for • A need for improved durability of response and treatment relapse rate in Novel MOAs multiple myeloma • IL-18 allows tumor to evade immune destruction, and is a driver of Unique tumor growth Mechanism of Action • Demonstrated proof-of-concept with an IL-18 binding protein in AOSD • Unique MOA and safety profile makes it an ideal candidate for combination Clinical therapy in MM Differentiation • Completely new mechanism with strong correlation for disease severity in AOSD 6| Source: Nakamura Cancer Cell. 2018. 33(4):634-648.e5.; Kudela et al. (2019) BMC Rheumatol . 3:4
Strong Potential in Multiple Myeloma Elevated IL-18 levels correlate with poor survival in multiple myeloma patients IL-18 Levels Are Elevated in Many MM Patients and Correlate with Poor Survival (n=69) (n=76) • Patients with high IL-18 have significantly worse median survival (42 months vs. >84 months, p value= 0.0026, HR = 1.84) • Reducing IL-18 levels prolongs survival in rodent models of multiple myeloma 7| Source: Nakamura Cancer Cell. 2018. 33(4):634-648.e5
Additional Targets within IL-18-Mediated Autoimmune Disorders IL-18 levels correlate with AOSD severity Elevated Serum IL-18 Levels in AOSD Patients IL-18bp Response Rates 80 1000000 Serum IL-18 (pg/ml) 100000 Percentage of Responders 60 10000 * 1000 40 100 10 20 Active AOSD in AOSD in Comparison Comparison AOSD Partial Remission Group Group Remission CRP>5 CRP<=5 • IL-18 is a key driver of several orphan 0 auto-inflammatory diseases 80 mg 160 mg – Adult Onset Still’s Disease (AOSD) (Week 12) (Week 12) • Serum IL-18 correlates with disease severity Patients Received Subcutaneous – AB2 Bio clinical proof-of-concept in AOSD (n = 23) using Administration of 80 or 160 Mg IL-18bp (T1/2 = 40 h); 4/4 patients with undetectable Three Times per Week serum IL-18 had a clinical response 8| Kudela et al. BMC Rheumatol . 2019. 3:4. Gabay et al. Ann Rheum Dis. 2018. 77(6):840-847
Program Update as of June 2020 Anticipate proof-of-concept trial initiation Q4 2020 in both MM and AOSD • Pre-IND meeting with the FDA completed, concurrence on high-level design • CRO under contract (PRA); sites selected; study start anticipated early 4Q 2020 Multiple Myeloma • Classic 3+3 dose escalation design to determine recommended Phase 2 dose (anticipated 1Q 2021) followed by a treatment expansion portion to establish response rate (anticipated 2Q 2021) • Pre-IND meeting with the FDA completed, working through design details AOSD • CRO under contract (PRA); study start anticipated 4Q 2020 9|
CERC-006 Phase 2-ready, Dual mTORC 1/2 small molecule inhibitor for Complex Lymphatic Malformations
Phase II-Ready Asset for Complex Lymphatic Malformations Potential first-in-class potent inhibitor of mTORC1/mTORC2 High Unmet • Orphan disease(s) with combined US prevalence of 30 to 60k associated with Need high mortality rates of 20% to 50% over 3 to 7 years Demonstrated • Off label use of sirolimus (mTORC1 inhibitor) has demonstrated modest Proof of efficacy, hampered by significant safety issues Concept • Potent inhibitor of mTORC1/mTORC2 allowing for lower dosing to achieve Potent Dual efficacy and improve safety mTOR • Dual inhibition may prevent upregulation of AKT and PI3K, potentially leading Inhibition to less diabetes and mucositis Potential to Become • Potential to be the first pharmacologic therapy approved for complex Standard lymphatic malformations of Care 11| Source: ClearView market research
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