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): September 5, 2018
Bellerophon Therapeutics, Inc. |
(Exact Name of Registrant as Specified in Charter)
Delaware | 001-36845 | 47-3116175 |
(State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) |
184 Liberty Corner Road, Suite 302 Warren, New Jersey | 07059 |
(Address of Principal Executive Offices) | (Zip Code) |
Registrant’s telephone number, including area code: (908) 574-4770
(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 (see General Instruction A.2. below):
o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o 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).
x | Emerging growth company | |
x | If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. | |
Item 7.01. Regulation FD Disclosure.
Bellerophon Therapeutics, Inc. has prepared an investor presentation to be presented to members of the investment community, a copy which is attached to this Current Report on Form 8-K as Exhibit 99.1.
In accordance with General Instruction B.2 on Form 8-K, the information set forth in this Item 7.01 and the investor presentation attached to this report as Exhibit 99.1 is “furnished” and shall not be deemed to be “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 such information be deemed incorporated by reference in any filing under the Securities Exchange Act of 1934, as amended, or the Securities Act of 1933, as amended.
The investor presentation attached hereto as Exhibit 99.1 contains certain statements that may be deemed to be “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. In some cases, these forward-looking statements can be identified by the use of forward-looking terminology, including the terms “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or, in each case, their negative or other variations thereon or comparable terminology, although not all forward-looking statements contain these words.
By their nature, forward-looking statements involve risks and uncertainties because they relate to events, competitive dynamics, and healthcare, regulatory and scientific developments and depend on the economic circumstances that may or may not occur in the future or may occur on longer or shorter timelines than anticipated. Although we believe that we have a reasonable basis for each forward-looking statement contained in the presentation, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in the presentation as a result of, among other factors, the factors referenced in the “Risk Factors” section of our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 15, 2018, and as amended on May 17, 2018, and the “Risk Factors” sections of our Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission on May 10, 2018 and August 1, 2018, respectively. In addition, even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in the presentation, they may not be predictive of results or developments in future periods. Any forward-looking statements that we make in the presentation speak only as of the date of such statement, and we undertake no obligation to update such statements to reflect events or circumstances after the date of the presentation, except as required by law.
You should read carefully our “Cautionary Note Regarding Forward-Looking Statements” and the factors described in the “Risk Factors” sections of our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q to better understand the risks and uncertainties inherent in our business.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits:
Exhibit No. | Description | |
2
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
BELLEROPHON THERAPEUTICS, INC. | ||||||
Date: September 5, 2018 | By: | /s/ Fabian Tenenbaum | ||||
Name: Fabian Tenenbaum Title: Chief Executive Officer | ||||||
3
Company Presentation September 2018 Nasdaq: BLPH
Forward Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make due to a number of important factors, including risks and uncertainties relating to: the timing and outcomes of our ongoing and expected clinical trials for our product candidates; our ability to successfully develop, commercialize and market any of our product candidates; our ability to obtain, maintain and enforce intellectual property rights; competition; our reliance on third parties; our ability to obtain necessary financing; and those risk factors discussed in the “Risk Factors” section and elsewhere in our most recent Form 10-K and other periodic filings we make with the SEC. All forward-looking statements contained in this presentation reflect our current views with respect to future events. We assume no obligation, except as required by applicable law, to update any forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. 2
Bellerophon Therapeutics (BLPH) – Company Profile Clinical-Stage Company spun-off from Ikaria Biotherapeutics Focused on developing inhaled nitric oxide (iNO) based therapies for outpatient management of chronic pulmonary diseases Company Portable, lightweight delivery system allows for chronic home use PH-ILD Phase 2b study ongoing with readout around end of 2018 Novel Therapy PH-COPD Phase 2b study design finalized with FDA Addressing Unmet Medical Needs PH-Sarc Phase 2 study to be initiated in 4Q2018 Simplified regulatory approval pathway via existing nitric oxide NDA IPO on Nasdaq in February 2015 Financial Cash & Equivalents: $25.9M(1,2), No Debt(1) Summary Shares Outstanding = 57.8 million(1); Fully Diluted = 96.8 million(1) Notes: (1) Amounts as of June 30, 2018 per Quarterly Report on Form 10-Q filed August 1, 2018 (2) Includes cash, cash equivalents and marketable securities 3
Investment Highlights Therapeutic benefit of inhaled nitric oxide (iNO) has been demonstrated Approved for acute treatment of persistent pulmonary hypertension Proprietary INOpulse technology enables advanced clinical stage product candidate targeting several unmet indications, including multiple orphan opportunities, with simplified approval pathway due to existing iNO NDA Pulmonary hypertension associated with interstitial lung disease (PH-ILD) . Successful Phase 2 study in PH-IPF completed in May 2017 . Phase 2b study initiated with top line results expected around the end of 2018 Pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD) . Successful Phase 2 study completed in September 2017 . Phase 2b study design finalized in agreement with FDA Pulmonary hypertension associated with sarcoidosis (PH-Sarc) . Phase 2 study to be initiated in 4Q2018 Learnings from PAH program support INOpulse mechanism of action and benefit in PH associated with ILD, COPD and Sarcoidosis Each indication represents multi-billion dollar potential market opportunity Strong IP protection on core programs through 2033 4
Development Pipeline Development Stage Indication Market 2017 2018 2019 Key Milestones PH-IPF • 220,000 with ILD in US Ph 2a Phase 2 Trial completed • 35-40% with • Results presented in May 2017 PH-ILD associated PH at rest Phase 2b Trial: iNO-PF (WHO Group 3) • Unmet medical need iNO-PF • Trial underway • $2B+ potential market Ph 2b • Top line around end of 2018 PH-COPD • 1.2 million PH-COPD in Ph 2 Phase 2 Trial completed US • Trial completed in Sept 2017 PH-COPD • Unmet medical need PH-COPD Phase 2b Trial: iNO-COPD (WHO Group 3) • Multi billion dollar Ph 2b • Trial design finalized potential market • Timing TBD • 200,000 with sarcoidosis in US PH-Sarc • Up to 30% with PH-Sarc Phase 2 Trial (WHO Group 5) associated PH Ph 2 • To be initiated in 4Q2018 • Unmet medical need • $1B+ potential market 5
Highly Experienced Leadership Team 10 years experience as CFO at Jonathan Peacock Amgen and Novartis Pharma and Chairman 10+ years as partner in Strategy and Corporate Finance consulting Fabian Tenenbaum 15 years of executive-level Chief Executive Officer experience in finance, business development and operations 15 years experience in clinical Deborah Quinn, MD research specializing in PAH Chief Medical Officer and heart failure Peter Fernandes 25 years experience in global Chief Regulatory & Safety regulatory affairs specializing in Officer respiratory products Assaf Korner 15 years of financial experience Chief Financial Officer in medical device and consumer product companies Parag Shah, PhD 12 years experience in pharmaceutical product VP, Business Operations development Amy Edmonds 20 years experience global VP, Clinical Operations & clinical operations and Administration training Martin Dekker 17 years experience in new VP, Device Engineering & product development and 6 Manufacturing launch
INOpulse Platform Overview Novel drug-device combination Potential to target therapy with dual mechanisms multiple unmet and NO of action underserved chronic • Targeted pulmonary pulmonary diseases Nitric Oxide is a well established vasodilation including areas where vasodilator approved for acute • Ventilation/Perfusion (V/Q) systemic vasodilators treatment of persistent pulmonary matching are ineffective or hypertension in hospitals poorly tolerated INOpulse® therapy allows for Current chronic use along with pulsatile Programs: targeted delivery to well ventilated PH-ILD sections of the lung PH-COPD PH-Sarc 7
INOpulse Provides a Unique and Differentiating Mechanism of Action Baseline Hypoxic pulmonary vasoconstriction prevents oxygen desaturation Systemic Vasodilators Systemic vasodilators can reverse hypoxic vasoconstriction leading to ventilation/perfusion (V/Q) mismatch and arterial O2 desaturation INOpulse Providing iNO early in the inspiratory phase allows for targeted vasodilation of only the well ventilated alveoliC thereby preventing V/Q mismatch and O2 desaturation NO 8
INOpulse: Portable Delivery System Allows Chronic iNO Therapy Commercial platform sold to Mallinckrodt for R&D Platform spun-out to form Bellerophon $2.3B Therapeutics • Continuous flow iNO delivery system • Pulsatile iNO delivery system • Approved for use in persistent pulmonary • Pulsed iNO can deliver equivalent dose as hypertension in neonates continuous delivery with 5% of the volume • Bulky device with large cylinders designed for • Dynamic pulse delivers the prescribed dose use in acute hospital settings accurately throughout the day • Small portable ~2.5 lbs. device allows ambulatory use in chronic in-home setting 9
INOpulse Delivery System: Lightweight, Portable and User Friendly Drug & Battery Cannula Power Indicators Alarm Key & Connector Swing Intuitive and engagement simple user with drug interface cartridge Oxygen Sources Lightweight Tri-lumen portable cannula design allows allows direct ease of connection transport with oxygen 10
Differences in PH Pathology, Standard of Care, and INOpulse Mechanism of Action PH Group 1 (PAH) PH Group 3 (PH-ILD & PH-COPD) PH Group 5 (PH-Sarc) Underlying • Vascular disease • Pulmonary disease (obstructive, • Inflammatory disease Disease fibrotic, etc.) (granulomas) PH • Normal lung function • Reduced lung function Pathology • PH driven by narrowing of • PH comorbidity is driven by lung structural and vascular changes pulmonary arteries Standard of • 13 approved drugs to treat PAH • No approved therapy to treat PH Care • Most patients treated with • PH Group 1 vasodilators ineffective multiple vasodilators INOpulse Single MOA Dual MOA Mechanism • Pulmonary vasodilation • Targeted pulmonary vasodilation of Action • Ventilation/Perfusion (V/Q) matching Clinical • Improvements in • Improvements in • Improvements in Findings hemodynamics and cardiac hemodynamics and 6MWD hemodynamics and 6MWD function verifies pulmonary verifies pulmonary vasodilation supports pulmonary vasodilation • Improvements in composite of vasodilation • Improvement in composite of 6MWD & SpO2 confirms V/Q 6MWD & SpO2 confirms V/Q matching matching • Results support dual • Improvement in 6MWD limited mechanism of action due to endpoint variability and multiple background vasodilators 11
Interstitial Lung Disease (PH-ILD) Represents a Significant Unmet Medical Need Interstitial Lung Disease (ILD) is a broad category of diffuse lung diseases characterized by variable amounts of inflammation and fibrosis Idiopathic Pulmonary Fibrosis (IPF) is the largest and most serious of the many fibrotic subsets of ILDs Patients with pulmonary fibrosis have thickening and scarring of the air sacs in the lungs, and often require supplemental oxygen to maintain adequate oxygen saturation 12
PH associated with Interstitial Lung Disease (PH-ILD) Significantly Reduces Survival Pulmonary hypertension as predictor of survival in IPF Approximately 40% of IPF patients exhibit symptoms of pulmonary hypertension at rest, including elevated pulmonary pressures Prognosis and survival are significantly worse for patients with pulmonary hypertension PH-IPF associated with a 3-fold increase in risk of death compared to IPF alone Rivera-Lebron, Advances in Pulmonary Hypertension, 2013 No approved therapy for treating PH in these patients INOpulse has the potential to provide targeted vasodilation to while avoiding concerns of V/Q mismatch which have prevented current PAH systemic vasodilators to be approved for this unmet medical need 13
PH-ILD Market Opportunity in the US PH-ILD • 220,000 ILD patients in the US 88% of PH- • IPF represents 44% of ILD patients IPF patients • Estimated prevalence of PH is 35-40% estimated • Prevalence of PH increases with severity to be on of underlying lung disease LTOT • Patients that are asymptomatic at rest may have PH during exercise Target US Population: • Approximately 60,000 PH-ILD on LTOT half of which have IPF • KOL feedback indicates high unmet medical need in PH-ILD • Addressable market estimated to be ~24,000 in the US • Pricing assessment supports a $2B+ market opportunity Data Monitor IPF Report 2015 LEK Market Assessment 14
Phase 2 Study Showed Immediate Benefit of iNO on Vasodilation and Hemodynamics Acute phase data showed: • Statistically significant (15.3%) increase blood vessel volume as compared to baseline • Significant correlation between ventilation and vasodilation, demonstrating selective vasodilation to better ventilated areas of the lung • Consistent and clinically meaningful reduction (14%) in systolic pulmonary arterial pressure (sPAP) Targeted Vasodilation Ventilation/Vasodilation Matching 15
Chronic iNO Treatment Provides Benefit on Oxygen Saturation and Exercise Capacity Chronic phase data showed: • Clinically meaningful improvement in exercise capacity and oxygen desaturation during 6MWT • Composite endpoint of oxygen saturation and exercise capacity shows consistent improvement in DSP and IDSP iNO Change in 6MWD 74.5 m Improvement in Nadir SpO2 5.5 % Improvement in Oxygen Desaturation 28.5 % Change in Distance Saturation Product (DSP) 78.1 m% Change in Integral Distance Saturation Product (IDSP) 85.9 m% Composite endpoints, combining oxygen saturation and walk distance during 6MWT, is potentially a better predictor of mortality in IPF (Lettieri et al, 2006) • DSP is the product of the distance walked and the lowest oxygen saturation (SpO2 nadir) during the 6MWT • IDSP is the product of the distance walked and the integral average (representative of AUC) of the oxygen saturation during the 6MWT 16
Next Steps in PH-ILD Phase 2b (iNO-PF) study initiated with Top Line Results around year end 40 subjects with pulmonary fibrosis: assessing subjects at low or intermediate/high risk of associated pulmonary hypertension at rest 8 weeks of blinded therapy (iNO 30 vs placebo) followed by open label treatment Multiple endpoints to be evaluated including: . 6MWD . Right ventricular function . Activity level (via activity monitors) . Oxygen saturation . Composite endpoints of oxygen saturation and exercise capacity (DSP, IDSP) Top line results will help finalize patient population, clinical endpoints and study size for Phase 3 Potential for a single Phase 3 trial to support NDA in underserved patient population 17
Chronic Obstructive Pulmonary Disease (PH-COPD) Represents a Large Unmet Medical Need Chronic obstructive pulmonary disease (COPD) which includes chronic bronchitis and emphysema is a group of lung diseases characterized by progressive airflow obstruction and chronic airway inflammation COPD is typically associated with smoking or exposure to other pollutants such as dust or chemicals Obstruction of the bronchioles and alveoli leads reduces the ability to get oxygen and ultimately leads to hypoxemia Hypoxemia and inflammation in COPD are thought to contribute to the development of associated pulmonary hypertension 18
Pulmonary Hypertension Independently Predicts Reduced Survival in Moderate-to-Severe COPD COPD patients with Pulmonary 5 Year Survival Hypertension have poor prognosis mPAP < 25 mmHg: 63% mPAP ≥ 25 mmHg: 37% and QoL Median life-expectancy is ~4 years with high hospitalization mPAP < 25 mmHg rates and impaired exercise capacity No approved therapy for treating PH in these patients mPAP ≥ 25 mmHg Existing PAH therapies lower pulmonary pressures but negatively influence oxygenation in PH-COPD Pulsed iNO can be targeted to the Survival years best ventilated alveoli only Dilation of best ventilated alveoli 2 Andersen KH et al. Prevalence, Predictors and Survival in Pulmonary Hypertension Related to End-stage Chronic Obstructive Pulmonary Disease. Journal of Heart and to reduce pulmonary pressure Lung Transplantation 2012; 31: 373-380. and prevent admixture of less oxygenated blood
PH-COPD Market Opportunity in the US COPD in US Prevalence: 27.8 million Diagnosed: 12.7 million (45.6%) Target US Population: PH-COPD on LTOT • Overall: 1,200,000 • Severe (Stage III/IV COPD): 900,00 Estimated prevalence of pulmonary hypertension (PH) in COPD is 27% • KOL feedback indicates high unmet medical need in PH-COPD • Addressable market estimated to be ~350,000 patients in the US • Multi-billion dollar market opportunity Data Monitor COPD Report 2010 LEK Opportunity Assessment 20
Demonstrated Benefit of Pulsed iNO on Vasodilation and Hemodynamics for PH-COPD Patients Baseline Corrected Change in Hemodynamics Vonbank et al, 2003 (Vonbank et al. 2003) • Sustained hemodynamic benefits, at three months, 2 50 PVRI (dyne PVRI(dyne s of pulsed iNO+O2 0 0 1 1 • Reduced mPAP and PVRI and increased -2 -50 2 cardiac output as compared to O2 alone -4 -100 - 1 without negative impact on hypoxemia cm -6 -150 - 5 • m Acute results replicated with INOpulse in a PH- Hg) mPAP(mm -8 -200 - 2 COPD Phase 2 study (COPD-201) ) -10 -250 O2 O2+iNO O2 O2+iNO 1. p-value <0.001; 2. p-value = 0.025 mPAP PVRI Change in Systolic Pulmonary Arterial Pressure (Mean 95% CI) COPD-201 Study 2 • Statistically significant change from baseline 0 for PASP at iNO 30 and iNO 75 dose -2 * * • Improvement in PASP is similar to results -4 * * * * * * from Vonbank study -6 ange in PASP (mm Hg) (mm PASP in ange • Verified iNO 30 as optimal dose with no h C -8 further improvement seen at iNO 75 Vonbank Study Study COPD-201 -10 10 15 20 25 30 3 10 15 30 75 iNO Dose (ppm) iNO Dose (mcg/Kg IBW/hr) Change in PASP significantly different from baseline (p < 0.05) * 21
High-Resolution Computed Tomography Imaging Study Demonstrated iNO effects on pulmonary vessels in PH-COPD patients Targeted Vasodilation • Acute Treatment with iNO 30 mcg/kg IBW/hr for at least 20 minutes (n=6) • No significant drop in blood oxygenation (SpO2) • All six patients showed increases in the blood volume in the vessel, Ventilation-Vasodilation Matching a surrogate for vasodilation • Patients reported significant improvement in symptoms for up to 24 hours Haijan et al., Int J of COPD, 2016 22
COPD-007 Phase 2 Study Showed Benefit of Chronic iNO Treatment on Exercise Capability and Hemodynamics • Subjects who completed 4 week chronic phase on iNO 30 showed: • Statistically significant increase in 6MWD at 2 weeks and 4 weeks (+50.7m) • Statistically and clinically significant decrease in sPAP at 4 weeks (-12.0 mmHg; 19.9% reduction) • sPAP increased to near baseline upon stopping treatment with iNO • Acute phase results showed a statistically significant increase of 4.2% in blood vessel volume compared to baseline and significant correlation between ventilation and vasodilation supporting targeted delivery to well ventilated alveoli Change in 6MWD sPAP p<0.05* p<0.05* p<0.05* *Statistically significant compared to baseline 23
Next Steps in PH-COPD Phase 2b study design reviewed and finalized with FDA U.S. based double-blind, placebo-controlled, study in approximately 90 PH-COPD patients Multiple endpoints to be evaluated including: . 6MWD . Right ventricular function . Time to clinical worsening . Time to clinical improvement . Oxygen saturation during 6MWT . Composite endpoints of oxygen saturation and 6MWT (distance saturation product) Target study start in 2019 Phase 2b study results will help finalize patient population, clinical endpoints and study size for Phase 3 24
Pulmonary Hypertension associated with Sarcoidosis (PH-Sarc) Represents an Orphan Unmet Medical Need Sarcoidosis is characterized by the growth of inflammatory cells (granulomas) most commonly in the lungs or lymphatic tissues Chest x-ray & CT of patient with PH-Sarc Prevalence of sarcoidosis is estimated at 200,000 in the US with up to 30% with associated pulmonary hypertension Patients with associated PH have significantly reduced survival 1 year survival 3 year survival 5 year survival PH-Sarc 84% 74% 59% Sarc 100% 96% 96% 25
INOpulse Mechanism of Action has the Potential to Provide Benefit to PH-Sarc Patients Systemic vasodilators exacerbate hypoxic vasoconstriction and cause hypoxemia No approved therapy for treating PH in these patients Inhaled nitric oxide has been shown to improve hemodynamics and exercise capacity in PH-Sarc Acute Hemodynamic Benefit on iNO Chronic Benefit on Exercise Capacity Parameter % Change mPAP -18 ± 4 PVR -31 ± 5 CO 12 ± 4 Preston et al., Chest, 2001 Phase 2 study designed to verify hemodynamic effect of INOpulse in PH-Sarc Acute dose escalation study with right heart catheterization Primary endpoint: change in mean PAP, PCWP, cardiac output and PVR Study to be initiated in 4Q2018 26
INOpulse – Intellectual Property Protection Patent Status Expiration Notes Method of NO US/EU: Issued Jan 2027 Covers consistent delivery of prescribed dose administration Other Territories: Issued/Pending independent of respiratory rate Breath Skipping & Pulse US: Issued Sept 2025 Covers skipping breaths or modifying pulse volume Volume Variation to ensure consistent dose independent of respiratory rate Method of Administering US/EU: Pending Mar 2033 Limits delivery rate of high concentration iNO to High Concentration NO Other Territories: Pending prevent safety concerns Optimized Pulse Shape US: Pending Oct 2035 Covers key parameters of pulse shape INOpulse Design US: Issued Apr 2028 Covers design of the INOpulse device Tip Purge US: Issued Apr 2033 Covers the use tip purge to ensure purity of iNO EU/Other Territories: Pending within the cannula Triple-Lumen Cannula US/ EU: Issued Dec 2033 Covers accurate dose delivery and reduced NO2 Other Territories: Pending formation Index valve US/EU: Issued May 2029 Ensures other cartridges cannot be used with Other Territories: Issued/Pending INOpulse • Orphan Drug designation for PH-IPF/PH-ILD would provide potential exclusivity for 7 years (US) and 10 years (EU) • Multiple provisional patent applications filed in 2017 and 2018 that can extend patent coverage into 2038 27
Financial Summary Amount (in millions) Cash and Cash Equivalents and Marketable Securities $25.9(1) Restricted Cash $0.6(1) Debt $0(1) Shares Outstanding 57.8(1) Fully Diluted 96.8(1) Current cash runway projected to cover cost of ongoing trials and key milestones into 2019, including top line results of Phase 2b Study in PH-PF 1) Amounts as of June 30, 2018 per Quarterly Report on Form 10-Q filed August 1, 2018 28
Investment Highlights Therapeutic benefit of inhaled nitric oxide (iNO) has been demonstrated Approved for acute treatment of persistent pulmonary hypertension Proprietary INOpulse technology enables advanced clinical stage product candidate targeting several unmet indications, including multiple orphan opportunities, with simplified approval pathway due to existing iNO NDA Pulmonary hypertension associated with interstitial lung disease (PH-ILD) . Successful Phase 2 study in PH-IPF completed in May 2017 . Phase 2b study initiated with top line results expected around the end of 2018 Pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD) . Successful Phase 2 study completed in September 2017 . Phase 2b study design finalized in agreement with FDA Sarcoidosis associated pulmonary hypertension (SAPH) . Phase 2 study to be initiated in 4Q2018 Learnings from PAH program support INOpulse mechanism of action and benefit in PH associated with ILD, COPD and Sarcoidosis Each indication represents multi-billion dollar potential market opportunity Strong IP protection on core programs through 2033 29
Thank you Investor Contacts: Fabian Tenenbaum Chief Executive Officer BTInvestorRelations@bellerophon.com Brian Ritchie LifeSci Advisors britchie@lifesciadvisors.com 212-915-2578