“The Bellerophon team continues to advance our late-stage INOpulse® inhaled nitric oxide therapy platform for the treatment of fibrotic interstitial lung disease, or fILD, and pulmonary hypertension associated with sarcoidosis, or PH-Sarc,” said
Clinical Program Highlights:
Fibrotic Interstitial Lung Disease (fILD)
- REBUILD Phase 3 Study: Enrollment is continuing in Bellerophon’s Phase 3 REBUILD registrational study of INOpulse for the treatment of fILD. The REBUILD study plans to enroll 300 fILD patients who will be treated with either INOpulse at a dose of iNO45 or placebo. The primary endpoint is change in moderate to vigorous physical activity (MVPA). If approved, INOpulse would become the first therapy to treat a broad fILD population that includes patients at low-, intermediate- and high-risk of pulmonary hypertension.
The Phase 3 program builds on positive top-line results from the Company’s previously reported Phase 2 studies for INOpulse for the treatment of fILD. Acute treatment with INOpulse showed benefit in multiple cardiopulmonary parameters, including pulmonary vascular resistance, which improved by 21%, and mean pulmonary arterial pressure, which improved by 12%. Chronic treatment with INOpulse at a dose of iNO45 assessed over four months showed an average improvement in MVPA of 20% as compared to placebo. The improvements in MVPA were supported by benefits in overall activity, as well as two patient reported questionnaires, theUniversity of California , San Diego Shortness of Breath Questionnaire and the St. George’s Respiratory Questionnaire.
Pulmonary Hypertension-Sarcoidosis (PH-Sarc)
- Phase 2 Clinical Study: In
December 2021 , Bellerophon reported positive top-line data from the completed Phase 2 dose escalation study of INOpulse evaluating the acute hemodynamic benefit of INOpulse via right heart catheterization for the treatment of pulmonary hypertension associated with sarcoidosis (PH-Sarc). PH-Sarc is an unmet medical need with no approved therapies, and a median survival of approximately five years after diagnosis. The Phase 2 trial was designed as a proof-of-concept study to determine if iNO could demonstrate hemodynamic benefit in PH-Sarc.
All eight subjects demonstrated decreases in mean pulmonary arterial pressure (mPAP) and pulmonary vascular resistance (PVR) across the doses of INOpulse utilized in the study. The dose of iNO45 (45 mcg/kg IBW/hr) resulted in a median drop of 20% (-54% to +22%) in PVR, compared to a median baseline PVR of 329 dyne/cm.sec-5; a reduction of 20% or more in PVR is generally considered to be clinically meaningful. Along with the improvements in PVR, mPAP decreased by a median of 6-10% across the doses of iNO30 to iNO125, compared to a median baseline mPAP of 37.2 mmHg. No treatment-emergent adverse events (TEAEs) or serious adverse events (TESAEs) occurred during the acute hemodynamic dose escalation phase of the study.
Based on the results of the acute dose escalation study, Bellerophon designed and submitted to the FDA a proposed exploratory Phase 2 double-blinded placebo-controlled study to investigate the safety and efficacy of inhaled nitric oxide/INOpulse dosed chronically for six months in patients with PH-Sarc. Subsequently, the Company received FDA clearance to conduct the study.
Corporate Update:
- Dr.
Robert Baughman M.D ., Professor of Medicine at theUniversity of Cincinnati , was the lead author for a poster presented at the American Thoracic Society 2022 International Conference summarizing the positive top-line data from the completed Phase 2 dose escalation study of INOpulse evaluating the acute hemodynamic benefit of INOpulse via right heart catheterization for the treatment of PH-Sarc.
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Balance Sheet
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About Bellerophon
Forward-looking Statements
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: risks and uncertainties relating to INOpulse®, 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
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Consolidated Balance Sheets
(Amounts in thousands, except share and per share data)
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 16,328 | $ | 24,736 | ||||
| Restricted cash | 403 | 103 | ||||||
| Prepaid expenses and other current assets | 257 | 620 | ||||||
| Total current assets | 16,988 | 25,459 | ||||||
| Restricted cash, non-current | — | 300 | ||||||
| Right of use assets, net | 529 | 863 | ||||||
| Property and equipment, net | 27 | 67 | ||||||
| Other non-current assets | 186 | 186 | ||||||
| Total assets | $ | 17,730 | $ | 26,875 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,007 | $ | 1,192 | ||||
| Accrued research and development | 1,512 | 1,397 | ||||||
| Accrued expenses | 1,344 | 1,711 | ||||||
| Current portion of operating lease liabilities | 586 | 752 | ||||||
| Total current liabilities | 5,449 | 5,052 | ||||||
| Long term operating lease liabilities | — | 203 | ||||||
| Common stock warrant liability | 1 | 1 | ||||||
| Total liabilities | 5,450 | 5,256 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock, |
95 | 95 | ||||||
| Preferred stock, |
— | — | ||||||
| Additional paid-in capital | 254,178 | 253,771 | ||||||
| Accumulated deficit | (241,993 | ) | (232,247 | ) | ||||
| Total stockholders' equity | 12,280 | 21,619 | ||||||
| Total liabilities and stockholders' equity | $ | 17,730 | $ | 26,875 | ||||
Consolidated Statement of Operations and Comprehensive Loss
(Amounts in thousands, except share and per share data)
| Three Months Ended | Six Months Ended | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 4,488 | $ | 3,239 | $ | 8,897 | $ | 6,823 | ||||||||
| General and administrative | 2,053 | 1,987 | 3,286 | 4,262 | ||||||||||||
| Total operating expenses | 6,541 | 5,226 | 12,183 | 11,085 | ||||||||||||
| Loss from operations | (6,541 | ) | (5,226 | ) | (12,183 | ) | (11,085 | ) | ||||||||
| Change in fair value of common stock warrant liability | — | 36 | — | 433 | ||||||||||||
| Interest and other income, net | 19 | 1 | 20 | 2 | ||||||||||||
| Pre-tax loss | (6,522 | ) | (5,189 | ) | (12,163 | ) | (10,650 | ) | ||||||||
| Income tax benefit | 2,417 | 1,800 | 2,417 | 1,800 | ||||||||||||
| Net loss and comprehensive loss | $ | (4,105 | ) | $ | (3,389 | ) | $ | (9,746 | ) | $ | (8,850 | ) | ||||
| Weighted average shares outstanding: | ||||||||||||||||
| Basic | 9,545,451 | 9,506,419 | 9,545,451 | 9,498,892 | ||||||||||||
| Diluted | 9,545,451 | 9,506,419 | 9,545,451 | 9,498,892 | ||||||||||||
| Net loss per share: | ||||||||||||||||
| Basic | $ | (0.43 | ) | $ | (0.36 | ) | $ | (1.02 | ) | $ | (0.93 | ) | ||||
| Diluted | $ | (0.43 | ) | $ | (0.36 | ) | $ | (1.02 | ) | $ | (0.93 | ) | ||||
Source: Bellerophon Therapeutics, Inc.