Cerecor : CERC-002 COVID-19 ARDS STUDY RESULTS

CERC-002:Double-blind,Placebo-controlledProof-of-concept Trial in Patients with COVID-19 Mild to Moderate ARDS

January 2021

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: its 2021 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 its 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.

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Executive Summary

Successful Proof of Concept for CERC-002 in Patients Hospitalized with COVID-19 ARDS

• Proof of concept demonstrated in hospitalized patients with COVID-19 related ARDS
• • COVID-19patients treated with a single dose of CERC-002 demonstrated robust improvement in the primary endpoint (proportion of patients alive and free of respiratory failure over the 28-day study period) compared to placebo (n=62, odds ratio [OR] = 2.62, p=0.059)
  • A prespecified subgroup analysis of patients ≥ 60 years of age showed that CERC-002 treatment led to a greater than 3-fold likelihood of avoiding respiratory failure and death (n=33, OR = 3.38, p=0.054)
  • 28-daymortality was reduced by approximately 50% in patients treated with CERC-002 (3 patients) vs. placebo (6 patients). There was a total of 4 COVID-19 related deaths in patients on CERC-002 vs. 9 on placebo as of December 2020. These data will be updated and analyzed at the 60-day timepoint
  • Importantly, CERC-002 showed activity on top of corticosteroids in COVID-19 ARDS (>90% of patients in the trial received corticosteroids and >60% received remdesivir)
• CERC-002was well tolerated with no appreciable differences in immunosuppression or other SAE between CERC-002 and placebo
• CERC-002dramatically and rapidly reduced serum free-LIGHT levels
• • ~85% reduction in free LIGHT achieved in 1 day
• Plan to meet with the FDA regarding a registration trial and filing for Breakthrough Therapy Designation
• Additionally, the company is continuing its program in severe pediatric-onset Crohn's disease and is exploring the applicability of CERC-002 in non-COVID-19 ARDS

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LIGHT is a Central Driver of COVID-19 Related Cytokine Storm

Clinical Trial Initiated After Compelling Biomarker Study Completed June 2020

Association Between Elevated LIGHT and Mortality

Strongest in Patients Over 60

Key Implications

Free LIGHT Levels (pg/mL)

P = 0.021

10000

8000

1500

1000

500

0

Healthy Controls	Recovered	Deceased
Over 60	Over 60	Over 60
(n=14)	(n=5)	(n=23)

• In patients over 60, LIGHT levels were significantly higher in those that eventually died than in those patients that recovered (p=0.021)
• Observed mortality rate higher was higher for patients over 60 of age (82%) compared to patients <60 years (32%)

Elevated LIGHT levels in hospitalized COVID-19 patients were most strongly

associated with mortality in patients over 60

1. Perlin et al. (2020) mSphere. 5(4):e00699-20.

4| 2. Arunachalam et al. (2020) Science. 369(6508):1210-1220

CERC-002: A Novel First-in-ClassAnti-LIGHT (TNFSF14) mAb

The only known clinical stage anti-LIGHT antibody

• In-licensedfrom Kyowa Kirin Co.
• Positive toxicology profile
• • 8-weekmonkey toxicology study was well tolerated up to 100 mg/kg per week with NOAEL at 60 mg/kg
• Phase I trial successfully completed
• • Up to 1200 mg SQ in healthy volunteers (n=48) without significant toxicity
• Proprietary free LIGHT assay developed in collaboration with Myriad RBM enables a biomarker-based development approach

NH

2

23

VL

	VH			VH	22
	95			95
	149		CH	149
	136		205	136
88			88
134					134
		214	214
CL				CL
194		225	194
			231

263	263
323	323
369	369
427	427

Discovered at La Jolla Allergy Institute

and Licensed by Cerecor in 2016

NH

2

23

VL

5| SQ: Subcutaneous; NOAEL: No observed adverse effect level

CERC-002 Treatment of Cytokine Storm-InducedCOVID-19 ARDS

Primary Endpoint: Respiratory Failure and Mortality Over 28 Days

Proof-of-Concept Trial Design

Randomized, Double-blind,Placebo-controlled,Multi-Center,Proof-of-Concept Clinical Trial of CERC-002 in

Adults with COVID-19 ARDS

Inclusion Criteria			CERC-002 (16 mg/kg [maximum
			1200 mg]) on Day 1 by SQ injection +
Hospitalized Patients with Documented			Standard of Care at the site
COVID-19 Infection and	1:1
Clinical Evidence of Pneumonia with	Randomization
Mild to Moderate ARDS			Placebo-matched SQ injection +
			Standard of Care at the site
Enrollment (N=83)
Primary Endpoint			Key Secondary / Exploratory Endpoints

• The proportion of patients treated with CERC-002 compared with placebo in addition to standard of care at site, alive and free of respiratory failure over 28 days
• 80% power to show an absolute difference of 25% between cohorts

• 1-monthmortality
• Change in Pa02/Fi02 ratio
• Time to and duration of invasive ventilation
• LIGHT levels and other biomarkers of inflammation
• Viral load

6| PaO2 - Partial Pressure of Oxygen, FiO2 - Fraction of Inspired Oxygen

Patient Disposition Chart

Screening: Patients with COVID-19

associated Pneumonia and

Mild to Moderate ARDS

1:1 Randomization to

SoC + CERC-002 or Placebo

(n = 83)

Patients withdrawing

Informed Consent (n = 1)

Patients administered CERC-002 or Placebo (n = 82)

Patients free of High Flow O2		All Patients administered
or Positive Pressure O2		CERC-002 or Placebo
(n = 62)		(n = 82)
ITT Analysis for Primary		Full Analysis for Secondary
Endpoint		Endpoints and Safety

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Patient Demographics

Characteristic	CERC-002	Placebo
(n=41)	(n=42)
Age
Mean (SD)	59.2 (14.5)	58.1 (14.2)
Age Group
< 60 years (n, %)	20	(48.8%)	21 (50%)
≥ 60 years (n, %)	21	(51.2%)	21 (50%)
Gender
Male	25 (61%)	32	(76.2%)
Female	16 (39%)	10	(23.8%)
Free LIGHT Level at Baseline
Mean (range) pg/mL	348 (63 - 667)	273 (37 - 703)
Race
White	31	(75.1%)	37	(88.1%)
Black or African American	7 (17.1%)	3	(7.1%)
Asian	2	(4.9%)	0 (0%)
Other	1	(2.4%)	2	(4.8%)
Concomitant Medication
Systemic corticosteroids	38	(92.6%)	37	(88.1%)
Remdesivir	27	(65.9%)	29	(69.0%)

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A Single Dose of CERC-002 Reduced Free LIGHT Levels Dramatically and Rapidly

Free LIGHT Levels (pg/mL)

400

300

200

100

0

Free LIGHT Levels (pg/mL) Over Treatment Period

CERC-002 (n=36)

Placebo (n=34)

Baseline	Day 2	Day 5
(Day 0)

• Mean free LIGHT levels were comparable at baseline across cohorts
• Mean free LIGHT levels were about 100 pg/mL higher in the patients ≥ 60 years-old
• Free LIGHT levels reduced quickly in the active cohort and increased in the placebo cohort
• The pharmacodynamic effect was on top of standard of care where approximately 90% of patients received systemic corticosteroids

Free LIGHT is inhibited by Day 1 and remains low

9| Data on file

Robust Treatment Effect Demonstrated in Patients at Greatest Risk of Respiratory Failure and Death

Primary Endpoint: Percentage of patients alive and free of respiratory failure at Day 28

		100	p = 0.295
		p = 0.059
alivepatientsofPercentageand				p = 0.054	CERC-002
failurerespiratoryoffree	80
		Placebo
		60
		40
		20
		0
		Overall	Age < 60 years	Age ≥ 60 years
		(n = 62)	(n = 29)	(n = 33)

CERC-002 treatment led to a greater than 3-fold likelihood of avoiding respiratory failure and

death in patients ≥ 60 years (OR: 3.38, 90% CI: 0.98 - 11.68)

10| Data on file

Clear trend of CERC-002 reducing the need for invasive ventilation; this effect is driven by events in the ≥ 60-year-old subset of patients

Proportion/Percentage of Subjects Requiring Invasive Ventilation

		40		p = 0.17
RequiringSubjectsofProportion	VentilationInvasive
		CERC-002
		30		Placebo
		p = 0.13
		20
		10	p = 0.48
		0
		Overall	Age < 60 years	Age ≥ 60 years
		(n = 82)	(n = 41)	(n = 41)

Patients ≥ 60 years treated with CERC-002 were twice as likely to avoid invasive ventilation

(OR: 2.0, 90% CI: 0.61 - 6.6)

11| Data on file

28-day mortality was ~50% lower in patients treated with CERC-002 (3 patients) vs. placebo (6 patients)

COVID-19 related deaths: 4 on CERC-002 vs. 9 on placebo (December 2020)

			p = 0.26
Day 28	25
		CERC-002
by	20
	Placebo
Died
p = 0.19
who
15
Patientsof
10	p = 0.48
Percentage
0
	5
	Overall	Age < 60 years	Age ≥ 60 years
	(n = 80)	(n = 40)	(n = 40)

60-day follow up data in progress

12| Data on file

Safety and Tolerability

• CERC-002was well-tolerated at a single dose of 16 mg/kg
• No serious adverse events attributable to CERC-002
• Majority of AEs judged to be mild or moderate
• No evidence of increased infections or adverse events related to immunosuppression

	CERC-002	Placebo
	N = 40	N = 42
Subjects with ≥1 AE (%)	16 (40%)	21 (50%)
Subjects with ≥ Drug-related AE	8	(20%)	6	(14.3%)
AEs > 5%
Leukocytosis	6	(15%)	4 (9.5%)
Anemia	4	(10%)	3 (7.1%)
Hepatic enzyme increase	4	(10%)	2 (4.8%)
Acute kidney injury	3	(7.5%)	2 (4.8%)
Respiratory failure	3	(7.5%)	3 (7.1%)

13| Data on file

Next Steps

• 60-daysafety data expected 1Q 2021
• Plan to present full data at a future scientific meeting
• End of phase 2 meeting with FDA to discuss registration trial and filing for Breakthrough Therapy Designation
• Severe pediatric onset Crohn's disease - initial data 1Q 2021
• Currently exploring the applicability of CERC-002 in non-COVID-19 ARDS

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