Sarcoma vaccine(Memorial Sloan-Kettering Cancer Center)

WARREN, N.J., Aug. 15, 2022 (GLOBE NEWSWIRE) -- Bellerophon Therapeutics, Inc. (Nasdaq: BLPH) (“Bellerophon” or the “Company”), a clinical-stage biotherapeutics company focused on developing treatments for cardiopulmonary diseases, today provided a clinical program update and reported financial results for the second quarter ended June 30, 2022. “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 Naseem Amin, M.D., Chairman of Bellerophon’s Board of Directors. “Enrollment is steadily proceeding in our Phase 3 REBUILD study in fILD, which is evaluating the change in moderate to vigorous physical activity following treatment with INOpulse. In addition, we recently received clearance from the U.S. Food and Drug Administration (FDA) to conduct a follow-up exploratory Phase 2 chronic treatment clinical trial to evaluate the safety and efficacy of INOpulse in PH-Sarc patients.” 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, the University 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 the University 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. Second Quarter Ended June 30, 2022 Financial Results: For the three months ended June 30, 2022, the Company reported a net loss of $4.1 million, or $(0.43) per basic and diluted share, compared to a net loss of $3.4 million, or $(0.36) per basic and diluted share, for the three months ended June 30, 2021. Research and development expenses for the three months ended June 30, 2022 were $4.5 million, compared to $3.2 million in the prior year period. The increase was primarily due to the ongoing Phase 3 fILD trial. General and administrative expenses for the three months ended June 30, 2022 were $2.1 million, essentially flat as compared to $2.0 million in the prior year period. Six Months Ended June 30, 2022 Financial Results: For the six months ended June 30, 2022, the Company reported a net loss of $9.7 million, or $(1.02) per basic and diluted share, compared to a net loss of $8.9 million, or $(0.93) per basic and diluted share, in the six months ended June 30, 2021. Research and development expenses for the six months ended June 30, 2022 were $8.9 million, compared to $6.8 million in the prior year period. The increase was primarily due to the ongoing Phase 3 fILD trial. General and administrative expenses for the six months ended June 30, 2022 were $3.3 million, compared to $4.3 million in the prior year period. The decrease was primarily due to lower consulting, labor and stock-based compensation costs. Balance Sheet As of June 30, 2022, the Company had cash and cash equivalents of $16.3 million, compared to cash and cash equivalents of $24.7 million at December 31, 2021. About Bellerophon Bellerophon Therapeutics is a clinical-stage biotherapeutics company focused on developing innovative therapies that address significant unmet medical needs in the treatment of cardiopulmonary diseases. The Company is currently developing multiple product candidates under its INOpulse® program, a proprietary pulsatile nitric oxide delivery system. For more information, please visit . 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 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. Contacts LifeSci Advisors: Brian Ritchie (212) 915-2578 britchie@lifesciadvisors.com BELLEROPHON THERAPEUTICS, INC. Consolidated Balance Sheets (Amounts in thousands, except share and per share data) June 30, 2022 December 31, 2021 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, $0.01 par value per share; 200,000,000 shares authorized and 9,545,451 and 9,545,451 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively 95 95 Preferred stock, $0.01 par value per share; 5,000,000 shares authorized, zero shares issued and outstanding at June 30, 2022 and December 31, 2021 — — 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 BELLEROPHON THERAPEUTICS, INC. Consolidated Statement of Operations and Comprehensive Loss (Amounts in thousands, except share and per share data) Three Months Ended Six Months Ended June 30, June 30, 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 )

WARREN, N.J., Nov. 15, 2021 (GLOBE NEWSWIRE) -- Bellerophon Therapeutics Inc. (Nasdaq: BLPH) (“Bellerophon” or the “Company”), a clinical-stage biotherapeutics company focused on developing treatments for cardiopulmonary diseases, today provided a clinical program update and reported financial results for the third quarter ended September 30, 2021. “We remain focused on advancing the development of our INOpulse® inhaled nitric oxide therapy in fibrotic interstitial lung disease, or fILD, and sarcoidosis, or PH-Sarc, which are two areas of severe unmet medical need,” said Naseem Amin, M.D., Chairman of Bellerophon’s Board of Directors. “Our pivotal Phase 3 REBUILD study in fILD is continuing to enroll. Importantly, INOpulse is the first potential therapy to treat a broad fILD population that includes patients at low, intermediate, and high risk of associated pulmonary hypertension. In addition, we are pleased to have concluded enrollment in our PH-Sarc Phase 2 dose escalation study and expect the availability of top-line results before the end of this year. Finally, with $28.7 million in cash and cash equivalents, we believe that we are in a strong financial position to continue advancing our late-stage development programs.” Clinical Program Highlights: fILD REBUILD Phase 3 Study: In December 2020, Bellerophon announced that the first patient had been enrolled in its 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, the University of California, San Diego Shortness of Breath Questionnaire and the St. George’s Respiratory Questionnaire. Pulmonary Hypertension-Sarcoidosis (PH-Sarc) Phase 2 Clinical Study: Bellerophon has concluded enrollment in a Phase 2 dose escalation study in PH-Sarc and anticipates the availability of top-line data by the end of this year. 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 no approved therapies, and a median survival of approximately five years after diagnosis. Corporate Update: In September 2021, the Company presented at the H.C. Wainwright 23rd Annual Global Investment Conference. Third Quarter Ended September 30, 2021 Financial Results For the three months ended September 30, 2021, the Company reported an operating loss of $4.8 million, compared to $8.3 million in the three months ended September 30, 2020. For the three months ended September 30, 2021, the Company reported a net loss of $4.6 million, or $(0.49) per basic and diluted share, compared to a net loss of $7.9 million, or $(0.84) per basic and diluted share, for the three months ended September 30, 2020. Research and development expenses for the three months ended September 30, 2021 were $3.0 million, compared to $6.1 million for the three months ended September 30, 2020. The decrease was primarily due to the close-out of activities related to the development of INOpulse for the treatment of COVID-19. General and administrative expenses for the three months ended September 30, 2021 were $1.8 million, compared to $2.2 million for the three months ended September 30, 2020. The decrease was primarily due to a decrease in consulting and stock-based compensation costs. Nine Months Ended September 30, 2021 Financial Results For the nine months ended September 30, 2021, the Company reported an operating loss of $15.9 million, compared to $18.1 million in the nine months ended September 30, 2020. For the nine months ended September 30, 2021, the Company reported a net loss of $13.5 million, or $(1.42) per basic and diluted share, compared to a net loss of $16.7 million, or $(2.31) per basic and diluted share, for the nine months ended September 30, 2020. Net loss for the nine months ended September 30, 2021 included an income of $0.6 million due to a change in fair value of the Company’s common stock warrant liability, as compared to an expense of $0.8 million for the nine months ended September 30, 2020. Research and development expenses for the nine months ended September 30, 2021 were $9.9 million, compared to $11.8 million for the nine months ended September 30, 2020. The decrease was primarily due to the close-out of activities related to the development of INOpulse for the treatment of COVID-19. General and administrative expenses for the nine months ended September 30, 2021 were $6.0 million, compared to $6.4 million for the nine months ended September 30, 2020. The decrease was primarily due to a decrease in consulting and stock-based compensation costs. Balance Sheet As of September 30, 2021, the Company had cash and cash equivalents of $28.7 million, compared to $47.6 million at December 31, 2020. About Bellerophon Bellerophon Therapeutics is a clinical-stage biotherapeutics company focused on developing innovative therapies that address significant unmet medical needs in the treatment of cardiopulmonary diseases. The Company is currently developing multiple product candidates under its INOpulse® program, a proprietary pulsatile nitric oxide delivery system. For more information, please visit . 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 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. Contacts LifeSci Advisors: Brian Ritchie (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, 2021 December 31, 2020 (Unaudited) Assets Current assets: Cash and cash equivalents $ 28,714 $ 47,557 Restricted cash 103 103 Prepaid expenses and other current assets 898 420 Total current assets 29,715 48,080 Restricted cash, non-current 300 300 Right of use assets, net 1,026 1,504 Property and equipment, net 88 169 Other non-current assets 186 186 Total assets $ 31,315 $ 50,239 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 1,278 $ 3,725 Accrued research and development 1,314 3,699 Accrued expenses 1,795 2,305 Current portion of operating lease liabilities 740 704 Total current liabilities 5,127 10,433 Long term operating lease liabilities 396 956 Common stock warrant liability 1 601 Total liabilities 5,524 11,990 Commitments and contingencies Stockholders' equity: Common stock, $0.01 par value per share; 200,000,000 shares authorized and 9,506,419 and 9,491,111 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively 95 95 Preferred stock, $0.01 par value per share; 5,000,000 shares authorized, zero shares issued and outstanding at September 30, 2021 and December 31, 2020 — — Additional paid-in capital 253,671 252,645 Accumulated deficit (227,975 ) (214,491 ) Total stockholders' equity 25,791 38,249 Total liabilities and stockholders' equity $ 31,315 $ 50,239 BELLEROPHON THERAPEUTICS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) (in thousands except share and per share data) Three Months Ended Nine Months Ended September 30, September 30, 2021 2020 2021 2020 Operating expenses: Research and development $ 3,030 $ 6,065 $ 9,853 $ 11,754 General and administrative 1,773 2,196 6,035 6,376 Total operating expenses 4,803 8,261 15,888 18,130 Loss from operations (4,803 ) (8,261 ) (15,888 ) (18,130 ) Change in fair value of common stock warrant liability 167 319 600 (768 ) Interest and other income, net 2 9 4 50 Pre-tax loss (4,634 ) (7,933 ) (15,284 ) (18,848 ) Income tax benefit — — 1,800 2,125 Net loss $ (4,634 ) $ (7,933 ) $ (13,484 ) $ (16,723 ) Weighted average shares outstanding: Basic 9,506,419 9,491,111 9,501,428 7,228,349 Diluted 9,506,419 9,491,111 9,501,428 7,228,349 Net loss per share: Basic $ (0.49 ) $ (0.84 ) $ (1.42 ) $ (2.31 ) Diluted $ (0.49 ) $ (0.84 ) $ (1.42 ) $ (2.31 )