“We continue to achieve important progress in advancing the ongoing REBUILD Phase 3 trial of INOpulse® for the treatment of fibrotic Interstitial Lung Disease (fILD),” said
“With the recent
Clinical Program Highlights:
Fibrotic Interstitial Lung Disease (fILD)
- REBUILD Phase 3 Study: Phase 3 REBUILD registrational study enrollment of INOpulse for the treatment of fILD is approaching completion. The reduced study size is 140 fILD patients who will be treated with either INOpulse at a dose of iNO45 or a placebo. The new study size does not impact the trial’s principal objective or endpoints and maintains power of >90% (p-value < 0.01) for the primary endpoint of Moderate to Vigorous Physical Activity (MVPA) based on the effect size observed in Phase 2. 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. Following the evaluation of baseline MVPA characteristics, as measured by actigraphy, compliance to treatment, and review of safety data of randomized subjects in the ongoing Phase 3 REBUILD study, the trial’s independent Data Monitoring Committee (DMC) supported reducing the target study size from 300 to 140 subjects.
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 which showed benefits in multiple cardiopulmonary parameters, including pulmonary vascular resistance and improvement in MVPA.
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 iNO45 dosed chronically for six months in patients with PH-Sarc. Subsequently, the Company received FDA clearance to conduct the study and the Company is currently assessing the next steps for the study.
Third Quarter Ended
For the three months ended
Research and development expenses for the three months ended
General and administrative expenses for the three months ended
Nine Months Ended
For the nine months ended
Research and development expenses for the nine months ended
General and administrative expenses for the nine months ended
Balance Sheet
As of
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
Contacts
(212) 915-2578
britchie@lifesciadvisors.com
BELLEROPHON THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands except share and per share data)
| As of | As of | |||||||
| (Unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 11,317 | $ | 24,736 | ||||
| Restricted cash | 404 | 103 | ||||||
| Prepaid expenses and other current assets | 338 | 620 | ||||||
| Total current assets | 12,059 | 25,459 | ||||||
| Restricted cash, non-current | — | 300 | ||||||
| Right of use assets, net | 357 | 863 | ||||||
| Property and equipment, net | 6 | 67 | ||||||
| Other non-current assets | 186 | 186 | ||||||
| Total assets | $ | 12,608 | $ | 26,875 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,525 | $ | 1,192 | ||||
| Accrued research and development | 1,888 | 1,397 | ||||||
| Accrued expenses | 1,370 | 1,711 | ||||||
| Current portion of operating lease liabilities | 396 | 752 | ||||||
| Total current liabilities | 5,179 | 5,052 | ||||||
| Long term operating lease liabilities | — | 203 | ||||||
| Common stock warrant liability | 1 | 1 | ||||||
| Total liabilities | 5,180 | 5,256 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock, |
95 | 95 | ||||||
| Preferred stock, |
— | — | ||||||
| Additional paid-in capital | 254,395 | 253,771 | ||||||
| Accumulated deficit | (247,062 | ) | (232,247 | ) | ||||
| Total stockholders' equity | 7,428 | 21,619 | ||||||
| Total liabilities and stockholders' equity | $ | 12,608 | $ | 26,875 | ||||
BELLEROPHON THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (UNAUDITED)
(in thousands except share and per share data)
| Three Months Ended | Nine Months Ended | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 3,750 | $ | 3,030 | $ | 12,646 | $ | 9,853 | ||||||||
| General and administrative | 1,366 | 1,773 | 4,653 | 6,035 | ||||||||||||
| Total operating expenses | 5,116 | 4,803 | 17,299 | 15,888 | ||||||||||||
| Loss from operations | (5,116 | ) | (4,803 | ) | (17,299 | ) | (15,888 | ) | ||||||||
| Change in fair value of common stock warrant liability | — | 167 | — | 600 | ||||||||||||
| Interest and other income, net | 47 | 2 | 67 | 4 | ||||||||||||
| Pre-tax loss | (5,069 | ) | (4,634 | ) | (17,232 | ) | (15,284 | ) | ||||||||
| Income tax benefit | — | — | 2,417 | 1,800 | ||||||||||||
| Net loss and comprehensive loss | $ | (5,069 | ) | $ | (4,634 | ) | $ | (14,815 | ) | $ | (13,484 | ) | ||||
| Weighted average shares outstanding: | ||||||||||||||||
| Basic | 9,545,451 | 9,506,419 | 9,545,451 | 9,501,428 | ||||||||||||
| Diluted | 9,545,451 | 9,506,419 | 9,545,451 | 9,501,428 | ||||||||||||
| Net loss per share: | ||||||||||||||||
| Basic | $ | (0.53 | ) | $ | (0.49 | ) | $ | (1.55 | ) | $ | (1.42 | ) | ||||
| Diluted | $ | (0.53 | ) | $ | (0.49 | ) | $ | (1.55 | ) | $ | (1.42 | ) | ||||
Source: Bellerophon Therapeutics, Inc.