“We are encouraged by the clinical and regulatory progress achieved with our INOpulse® program over the past several months, and look forward to reporting top-line results by the end of the year from Cohort 2 of our ongoing Phase 2/3 iNO-PF trial for the treatment of pulmonary hypertension associated with Interstitial Lung Disease (PH-ILD),” said
“In addition, we were pleased to receive Orphan Drug Designation in the third quarter from the
Key Recent Highlights
PH-ILD:
Cohort 1: Bellerophon presented results from Cohort 1 of iNO-PF, a randomized, double-blind, placebo-controlled clinical study of INOpulse for the treatment of PH-ILD, as a late-breaking abstract oral presentation at the
Cohort 1, the first of 3 cohorts, included 41 subjects randomized 1:1 to either iNO 30 (30 mcg/kg IBW/hr) or placebo, for a period of 8 weeks of blinded treatment. Top-line data from Cohort 1 demonstrated clinically and statistically significant improvement in MVPA, as well as other physical activity parameters measured by actigraphy, and was safe and well tolerated.
- MVPA (walking, stairs, yardwork, etc.) improved by 34% (8% increase on iNO vs. 26% decrease on placebo; p=0.04)
- 23% of subjects on INOpulse had a clinically significant improvement in MVPA, compared to 0% of subjects on placebo (placebo corrected difference of 23%)
- 39% of subjects on INOpulse had a clinically significant decline in MVPA, compared to 71% of subjects on placebo (placebo corrected difference of 32%)
- Proportion of awake time spent in MVPA improved by 38% (16% increase on INOpulse vs. 22% decrease on placebo; p=0.04)
- Overall activity improved by 12% (stable on INOpulse vs. 12% decrease on placebo; p=0.05)
- Responder analysis:
- 85% of subjects on placebo declined in MVPA, overall activity and non-sedentary activity
- 46% of subjects on INOpulse improved in MVPA, 62% in overall activity and 39% in non-sedentary activity (compared to only 15% of subjects on placebo in each category)
- OLE:
- Collectively, subjects (with an average of 27 weeks of OLE data) demonstrated maintenance of MVPA, overall activity and non-sedentary activity
- Subjects randomized to active treatment in the blinded portion of the trial continued to maintain their activity levels when transitioning to OLE over 27 weeks of open-label treatment
- Subjects randomized to placebo in the blinded portion of the trial transitioned from a decline during blinded treatment to stabilization of activity levels (MVPA, overall activity and non-sedentary activity) over 27 weeks of open-label treatment
Cohort 2: The Company completed recruitment in Cohort 2, which includes 44 subjects randomized 2:1 to either iNO45 (45 mcg/kg IBW/hr) or placebo. Subjects will complete 16 weeks of blinded treatment on iNO45 vs. placebo, and then continue onto open-label treatment. The Company expects to report top-line results from Cohort 2 by year-end 2019.
Cohort 3: The results from Cohorts 1 and 2 will be evaluated to determine the optimal dose between iNO30 and iNO45 to progress into pivotal Phase 3, with MVPA serving as the primary endpoint. The Company anticipates initiating the Phase 3 study in the first quarter of 2020, with approximately 300 subjects randomized 1:1 to active or placebo for a period of 16 weeks of blinded treatment.
Orphan Drug Designation: The
PH-Sarcoidosis: Bellerophon has initiated a Phase 2 dose escalation study in PH associated with sarcoidosis. The study is a dose escalation safety and efficacy study that will assess the acute hemodynamic benefit of INOpulse via right heart catheterization. PH-Sarcoidosis is an unmet medical need with a median survival of approximately five years after diagnosis. Similar to PH-ILD and PH-COPD, PH-Sarcoidosis cannot be treated with currently available systemic vasodilators.
PH-COPD: Following positive results from its Phase 2 study for INOpulse in PH-COPD, and agreement with the
Third Quarter Ended
For the three months ended
Net income for the three months ended
Research and development expenses for the three months ended
General and administrative expenses were
Nine Months Ended
For the nine months ended
Net income 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: 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 | |
| At Bellerophon: | At LifeSci Advisors: |
| Fabian Tenenbaum, Chief Executive Officer | Brian Ritchie |
| (908) 574-4767 | (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 | |||||||
| September 30, 2019 | December 31, 2018 | |||||||
| (Unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 12,838 | $ | 16,645 | ||||
| Restricted cash | 102 | 101 | ||||||
| Prepaid expenses and other current assets | 712 | 650 | ||||||
| Total current assets | 13,652 | 17,396 | ||||||
| Restricted cash, non-current | 300 | 300 | ||||||
| Right of use assets, net | 2,256 | — | ||||||
| Property and equipment, net | 401 | 664 | ||||||
| Total assets | $ | 16,609 | $ | 18,360 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,264 | $ | 2,755 | ||||
| Accrued research and development | 2,581 | 3,771 | ||||||
| Accrued expenses | 1,488 | 1,013 | ||||||
| Current portion of operating lease liabilities | 647 | — | ||||||
| Total current liabilities | 6,980 | 7,539 | ||||||
| Long term operating lease liabilities | $ | 1,828 | $ | — | ||||
| Common stock warrant liability | 452 | 6,965 | ||||||
| Total liabilities | 9,260 | 14,504 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Common stock, $0.01 par value per share; 200,000,000 shares authorized, and 68,906,765 and 58,679,492 shares issued and outstanding at September 30, 2019 and December 31, 2018, respectively | 689 | 587 | ||||||
| Preferred stock, $0.01 par value per share; 5,000,000 shares authorized, zero shares issued and outstanding at September 30, 2019 and December 31, 2018 | — | — | ||||||
| Additional paid-in capital | 192,343 | 179,765 | ||||||
| Accumulated deficit | (185,683 | ) | (176,496 | ) | ||||
| Total stockholders’ equity | 7,349 | 3,856 | ||||||
| Total liabilities and stockholders’ equity | $ | 16,609 | $ | 18,360 | ||||
BELLEROPHON THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)
(in thousands except share and per share data)
| Three Months Ended September 30, |
Nine Months Ended September 30, |
|||||||||||||||
| 2019 | 2018 | 2019 | 2018 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 3,259 | $ | 5,247 | $ | 8,193 | $ | 17,442 | ||||||||
| General and administrative | 1,332 | 1,584 | 4,965 | 5,754 | ||||||||||||
| Total operating expenses | 4,591 | 6,831 | 13,158 | 23,196 | ||||||||||||
| Loss from operations | (4,591 | ) | (6,831 | ) | (13,158 | ) | (23,196 | ) | ||||||||
| Change in fair value of common stock warrant liability | 215 | 17,840 | 2,504 | 21,201 | ||||||||||||
| Warrant amendment charge | — | — | (674 | ) | — | |||||||||||
| Interest and other income, net | 89 | 92 | 340 | 282 | ||||||||||||
| Pre-tax (loss) income | (4,287 | ) | 11,101 | (10,988 | ) | (1,713 | ) | |||||||||
| Income tax benefit | — | — | 1,801 | 5,439 | ||||||||||||
| Net (loss) income | $ | (4,287 | ) | $ | 11,101 | $ | (9,187 | ) | $ | 3,726 | ||||||
| Weighted average shares outstanding: | ||||||||||||||||
| Basic | 68,303,027 | 57,710,251 | 67,229,585 | 57,356,445 | ||||||||||||
| Diluted | 68,303,027 | 64,544,504 | 67,229,585 | 65,854,903 | ||||||||||||
| Net (loss) income per share: | ||||||||||||||||
| Basic | $ | (0.06 | ) | $ | 0.19 | $ | (0.14 | ) | $ | 0.06 | ||||||
| Diluted | $ | (0.06 | ) | $ | (0.10 | ) | $ | (0.14 | ) | $ | (0.27 | ) | ||||
Source: Bellerophon Therapeutics, Inc.