“We are extremely pleased with the significant progress achieved with our clinical program for INOpulse® to address multiple unmet cardiopulmonary diseases. Most notably, we reported positive top-line results from Phase 2 of our ongoing Phase 2/3 clinical study (iNO-PF) of INOpulse for the treatment of PH-PF, completed our End-of-Phase 2 meetings with the
Clinical Program Highlights:
- Planned Pivotal Phase 3 Study:
Bellerophon Therapeuticssuccessfully completed its End-of-Phase 2 meetings with the FDA and finalized key elements of its planned pivotal Phase 3 study of INOpulse for the treatment of PH-PF. The Company intends to dose the first patient in the Phase 3 study shortly. If approved, INOpulse would become the first therapy to treat a broad PH-PF population that includes patients at low-, intermediate- and high-risk of pulmonary hypertension.
- iNO-PF Phase 2 Study:
Bellerophon Therapeuticsreported positive top-line results from Phase 2 of its ongoing Phase 2/3 randomized, double-blind, placebo-controlled clinical study (iNO-PF) of INOpulse for the treatment of PH-PF. Subjects treated with INOpulse (iNO30 or iNO45) maintained their activity levels, including moderate to vigorous physical activity (MVPA), while subjects on placebo deteriorated. Subjects treated with the higher dose of iNO45 chronically over four months demonstrated statistically significant improvement in MVPA of 14 minutes per day, representing a 20% improvement (p=0.02). Improvements in MVPA were supported by benefits in other activity parameters, as well as two patient reported questionnaires. The University of California, San Diego Shortness of Breath Questionnaire improved by 5 points and the St. George’s Respiratory Questionnaire total score improved by 3 points, with the Activity and Impacts domains improving by 5 and 6 points, respectively.
- PHPF-002 (ancillary study to iNO-PF):
Bellerophon Therapeuticsreported positive top-line data from PHPF-002, a recently completed ancillary dose escalation study assessing the acute hemodynamic effect of INOpulse in PH-PF via right heart catheterization. The study demonstrated that acute treatment with INOpulse provided statistically and clinically significant improvements in pre-specified hemodynamic parameters, including a 21% reduction in pulmonary vascular resistance, with increased benefit (p<0.01) on dose escalation from iNO30 to iNO45, and a 12% reduction in mean pulmonary arterial pressure. The acute hemodynamic benefits underpin the chronic benefit in exercise capacity demonstrated in the iNO-PF study and support utilizing the dose of iNO45 in the planned Phase 3 trial.
Pulmonary Hypertension-Sarcoidosis (PH-Sarc)
- Phase 2 Clinical Study:
Bellerophon Therapeuticsis conducting a Phase 2 dose escalation study in PH-Sarc. The safety and efficacy study is assessing the acute hemodynamic benefit of INOpulse via right heart catheterization. PH-Sarc is an unmet medical need with a median survival of approximately five years after diagnosis. Similar to PH-PF and PH-ILD, PH-Sarc cannot be treated with currently available systemic vasodilators.
- FDA Granted Emergency Expanded Access Program: Expanded access treatment with INOpulse was successfully used for the first time in a patient with a diagnosis of COVID-19 at the
University of Miami School of Medicineand the patient was subsequently discharged from the hospital. Published in vitro data have demonstrated that nitric oxide reduces viral load and prevents viral replication in SARS Coronavirus (SARS-CoV)¹, and a clinical study in patients infected with SARS-CoV revealed that treatment with inhaled nitric oxide can improve arterial oxygenation, reduce the need for ventilation support and reduce pneumonia infiltration². The genetic similarities between the two coronaviruses and the historical data support INOpulse’s potential to provide meaningful benefit for patients infected with COVID-19.
Corporate Finance Updates:
- Registered Direct Offering of Common Stock: In
April 2020, the Company closed a registered direct offering of 1,275,000 shares of its common stock at a purchase price of $12.00per share for total gross proceeds of $15.3 million, before deducting placement agent fees and offering expenses.
- Convertible Financing Facility: In
December 2019, the Company entered into a $10 millionconvertible financing facility led by existing institutional shareholders.
Fourth Quarter Financial Results:
For the fourth quarter ended
Net income for the three months ended
Research and development expenses for the fourth quarter ended
General and administrative expenses for the fourth quarter ended
2019 Year End Financial Results:
For the year ended
Net income for the year ended
Research and development expenses for the year ended of
General and administrative expenses for the year ended
Any statements in this press release about Bellerophon’s future expectations, plans and prospects, including statements about the clinical development of its product candidates, regulatory actions with respect to the Company’s clinical trials and expectations regarding the sufficiency of the Company’s cash balance to fund clinical trials, operating expenses and capital expenditures, and other statements containing the words “anticipate,” “believe,” “continue,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary or interim results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, the FDA’s substantial discretion in the approval process, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and in subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent Bellerophon’s views only as of the date of this release and should not be relied upon as representing the Company’s views as of any subsequent date. The Company specifically disclaims any obligation to update any forward-looking statements included in this press release.
At W2O Group:
- Akerstrom S et. Al. Nitric oxide inhibits the Replication Cycle of Severe Acute Respiratory Syndrome Coronavirus. J Virol 2005; 79(3):1966-9.
- Chen L. Inhalation of nitric oxide in the treatment of acute respiratory syndrome: a rescue trial in Beijing. Clinical Infectious Diseases 2004; 39(10):1531-5.
Consolidated Balance Sheets
(Amounts in thousands, except share and per share data)
|Cash and cash equivalents||$||9,874||$||16,645|
|Prepaid expenses and other current assets||405||650|
|Total current assets||10,382||17,396|
|Restricted cash, non-current||300||300|
|Right of use assets, net||2,110||—|
|Property and equipment, net||316||664|
|Liabilities and Stockholders' Equity|
|Accrued research and development||2,117||3,771|
|Current portion of operating lease liabilities||658||—|
|Total current liabilities||7,584||7,539|
|Long term operating lease liabilities||1,659||—|
|Common stock warrant liability||274||6,965|
|Commitments and contingencies (Note 11)|
|Additional paid-in capital||193,308||180,313|
|Total stockholders' equity||3,591||3,856|
|Total liabilities and stockholders' equity||$||13,108||$||18,360|
Consolidated Statements of Operations
(Amounts in thousands, except share and per share data)
|Research and development||$||11,032||$||20,259||$||17,854|
|General and administrative||6,441||7,621||6,745|
|Total operating expenses||17,473||27,880||24,599|
|Loss from operations||(17,473||)||(27,880||)||(24,599||)|
|Change in fair value of common stock warrant liability||2,682||24,877||(30,403||)|
|Warrant amendment charge||(674||)||—||—|
|Interest and other income, net||397||378||184|
|Income tax benefit||(1,801||)||(5,439||)||—|
|Net (loss) income||$||(13,267||)||$||2,814||$||(54,818||)|
|Weighted average shares outstanding:|
|Net (loss) income per share:|
Source: Bellerophon Therapeutics, Inc.