"We measure success by the number of meaningful medicines we are able to develop and deliver to patients. By that metric, our most significant accomplishment of the quarter was the approval of TRUSELTIQ™ (infigratinib) for patients with cholangiocarcinoma - our second FDA approval as a company and our first for a cancer treatment. All drug approvals are a team effort that extends far beyond BridgeBio. We thank the patient community, physicians, scientists and advocates for their commitment and drive. They made this approval possible," said BridgeBio founder and CEO
Major milestones anticipated in the next 12 months for BridgeBio's four core value drivers:
- Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 and from Part B in 2023. The primary endpoint at Part A is the change from baseline in a 6-minute walk distance (6MWD) in trial participants receiving acoramidis or placebo after 12 months. In a previous Phase 3 study in ATTR-CM patients, participants receiving the current standard of care treatment demonstrated a decline in 6MWD of approximately 25 meters (m) from an average baseline of 351m at 12 months.1 From a comparable average 6MWD baseline in the ATTRibute-CM study, the Company is seeking to more potently halt the observed decline in acoramidis-treated participants. As a reminder, healthy elderly adults typically decline only 7m in 12 months.2 If the change from baseline in 6MWD in Part A is highly statistically significant (p < 0.01), BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA.
- Encaleret – Calcium-sensing receptor (CaSR) inhibitor for ADH1: Received Fast Track designation from the FDA. Early results from an ongoing Phase 2 proof-of-concept study shared at the
Endocrine Society's 2021 Annual Meeting (ENDO) inMarch 2021 showed normalization of blood calcium and urine calcium in 6 of 6 (100%) ADH1 participants. Additional data from the ongoing study are expected in the second half of 2021. If the development program is successful, encaleret could be the first approved therapy for ADH1, a condition caused by gain of function variants in the CaSR gene estimated to be carried by 12,000 individuals inthe United States alone.
- Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the first half of 2022. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with a prevalence of greater than 55,000 cases in the
United States andEuropean Union . Low-dose infigratinib is the only known product candidate in clinical development for achondroplasia that is designed to target the disease at its genetic source and the only orally administered product candidate in clinical-stage development.
- BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): Received Fast Track designation from the FDA in
May 2021 . Investigational New Drug (IND) application cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in mid-2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases estimated in theUnited States andEuropean Union . The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol production. BridgeBio's AAV5 gene therapy candidate is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.
Recent pipeline progress and corporate updates:
- FDA approval received for TRUSELTIQ™ (infigratinib) under the accelerated approval program for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as detected by an FDA-approved test. TRUSELTIQ is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. In the pivotal trial of patients with advanced, unresectable CCA, an aggressive malignancy with poor prognosis, TRUSELTIQ led to cases of tumor shrinkage. BridgeBio is eligible to receive upfront, regulatory and commercial milestone payments totaling up to approximately
$2.45 billion USD through its strategic collaboration withHelsinn Group to co-develop and commercialize infigratinib in certain oncology indications.
- Fast Track designation received from the FDA for BBP-812, BridgeBio's AAV9 gene therapy candidate for Canavan disease. IND cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing. Canavan disease is an extremely rare genetic condition starting in infancy with an incidence of approximately one in 100,000 births worldwide.
- Fast Track designation received from the FDA for infigratinib, an FGFR inhibitor, for the treatment of urothelial carcinoma (urinary tract and bladder cancer).
- New Drug Application (NDA) acceptance from the
Israeli Ministry of Health for NULIBRY™ (fosdenopterin) for injection to treat patients with molybdenum cofactor deficiency (MoCD) Type A. The FDA approved NULIBRY as the first therapy to reduce the risk of mortality in patients with MoCD Type A inFebruary 2021 . MoCD Type A is an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate.
- First-in-human Phase 1 trial of BBP-711, a glycolate oxidase (GO) inhibitor to treat patients with hyperoxaluria, initiated in
May 2021 .
- Research collaborations initiated with
MUSC Foundation for Research Development ,Stanford University and theUniversity of Pittsburgh to identify and advance therapies for genetic diseases and cancers for a total of 23 partnerships among BridgeBio and leading academic and research institutions to date.
- Non-exclusive, co-funded clinical collaboration initiated with Bristol Myers Squibb to study BBP-398, a potentially best-in-class
SHP2 inhibitor, in combination with OPDIVO® (nivolumab) in patients with advanced solid tumors with KRAS mutations.
- BridgeBio Pharma R&D Day: BridgeBio will hold a virtual R&D Day on
Tuesday, October 12, 2021 , from8:30 am ET –11:30 am ET . The event will be webcast and registration information can be found here.
Second Quarter 2021 Financial Results:
Cash,
Cash, cash equivalents and marketable securities, excluding restricted cash, totaled
Cash, cash equivalents and marketable securities, excluding restricted cash, decreased by
Revenues
Total revenues for the three and six months ended
Operating Costs and Expenses
Operating costs and expenses for the three and six months ended
Our research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While we experienced some delays in certain of our clinical enrollment and trial commencement activities, we continue to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with our contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on our operating costs and expenses is currently unknown.
|
||||||||
Condensed Consolidated Statements of Operations |
||||||||
(in thousands, except shares and per share amounts) |
||||||||
Three Months Ended |
Six Months Ended |
|||||||
2021 |
2020 |
2021 |
2020 |
|||||
(Unaudited) |
(Unaudited) |
|||||||
Revenue |
|
$ — |
|
$ — |
||||
Operating costs and expenses: |
||||||||
Research and development |
102,069 |
86,598 |
224,628 |
154,823 |
||||
Selling, general and |
45,970 |
37,969 |
91,377 |
72,231 |
||||
Total operating costs and |
148,039 |
124,567 |
316,005 |
227,054 |
||||
Loss from operations |
(94,015) |
(124,567) |
(261,519) |
(227,054) |
||||
Other income (expense), net: |
||||||||
Interest income |
323 |
934 |
717 |
2,875 |
||||
Interest expense |
(10,839) |
(10,754) |
(20,577) |
(14,764) |
||||
Other income |
2,457 |
(1,827) |
8,223 |
(1,353) |
||||
Total other income (expense), net |
(8,059) |
(11,647) |
(11,637) |
(13,242) |
||||
Net loss |
(102,074) |
(136,214) |
(273,156) |
(240,296) |
||||
Net loss attributable to |
5,726 |
15,180 |
13,729 |
27,412 |
||||
Net loss attributable to common stockholders of BridgeBio |
|
|
|
|
||||
Net loss per share, basic and diluted |
|
|
|
|
||||
Weighted-average shares used in computing net loss per share, basic and diluted |
146,754,299 |
117,012,062 |
142,713,463 |
117,407,750 |
|
||||
Condensed Consolidated Balance Sheets |
||||
(In thousands) |
||||
|
|
|||
2021 |
2020 |
|||
Assets |
(Unaudited) |
(1) |
||
Cash and cash equivalents and marketable securities (2) |
|
|
||
Receivable from licensing and collaboration agreements |
35,363 |
— |
||
Receivable from a related party |
8,962 |
— |
||
Prepaid expenses and other current assets |
27,883 |
35,731 |
||
Property and equipment, net |
26,272 |
20,325 |
||
Operating lease right-of-use assets |
15,964 |
16,508 |
||
Investment in equity securities |
18,894 |
— |
||
Other assets |
49,797 |
23,931 |
||
Total assets |
|
|
||
Liabilities, Redeemable Convertible |
||||
Accounts payable |
|
|
||
Accrued liabilities |
105,040 |
75,900 |
||
LEO call option liability |
— |
5,550 |
||
Operating lease liabilities |
22,562 |
18,472 |
||
Term loans, current portion |
— |
1,458 |
||
Term loans, net of current portion |
102,611 |
92,421 |
||
2029 Notes |
732,202 |
[ ] |
||
2027 Notes |
539,102 |
383,436 |
||
Other liabilities |
13,265 |
9,520 |
||
Redeemable convertible noncontrolling interests |
1,865 |
1,630 |
||
Total BridgeBio stockholders' equity (deficit) |
(464,294) |
57,906 |
||
Noncontrolling interests |
6,804 |
48,350 |
||
Total liabilities, redeemable convertible |
|
|
(1) |
The condensed consolidated financial statements as of and for the year ended |
|
(2) |
|
About
This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, the availability and success of topline results from Part A and Part B of our ATTRibute-CM trial of acoramidis, our plans to submit an application for regulatory approval of acoramidis, the availability of additional data from our ongoing study of encaleret for ADH1, the availability of initial data from our ongoing Phase 2 study of infigratinib for achondroplasia and our ongoing Phase 1/2 study of BBP-631 for CAH, our eligibility to receive future milestone payments under our strategic collaboration with the
Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended
1 Maurer, M., Schwartz, J., Gundapaneni, B., et al. "Tafamidis treatment for patients with transthyretin Amyloid cardiomyopathy".
2Enright, M., Duanel, S. "Reference equations for the six-minute walk in healthy adults".
Media Contact:
grace.rauh@bridgebio.com
917-232-5478
Investor Contact:
katherine.yau@bridgebio.com
516-554-5989
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SOURCE BridgeBio