on November 5, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, reported its financial results for the third quarter ended September 30, 2020 and provided an update on the company’s operations (Press release, BridgeBio, NOV 5, 2020, View Source [SID1234570007]).
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BridgeBio announced a merger agreement last month with Eidos Therapeutics, Inc. (Nasdaq: EIDX), which is developing acoramidis (formerly AG10), a potential best-in-class transthyretin (TTR) stabilizer for patients with TTR amyloid (ATTR) cardiomyopathy and polyneuropathy. With this transaction, BridgeBio intends to fully and formally welcome Eidos back into its vibrant ecosystem of innovation and has agreed to acquire all of the outstanding common stock of Eidos it does not already own. The company expects to complete the proposed transaction in the first quarter of 2021, subject to certain conditions, including the receipt of stockholder approvals.
Since the company’s last quarterly update, BridgeBio had its first new drug application (NDA) accepted by the U.S. Food and Drug Administration (FDA) under Priority Review designation and initiated two new clinical trials, including a Phase 2 trial of encaleret (calcium sensing receptor antagonist) for autosomal dominant hypocalcemia type 1 (ADH1), one of BridgeBio’s four core value driver programs. It also entered into collaboration agreements with the Salk Institute and the University of Colorado Anschutz Medical Campus to advance the development of new therapies for genetically driven diseases.
BridgeBio held its first-ever R&D Day on September 29, 2020, which focused on the company’s drug engineering platform, its targeted oncology portfolio, and four highlighted programs where clinical data are anticipated in the next 12 to 18 months – acoramidis for ATTR, low-dose infigratinib (FGFR inhibitor) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia (CAH), and encaleret for ADH1.
"We are nearing a significant inflection point as a company as we approach the start of 2021. Our four key programs have critical data readouts within the next year and a half – in ATTR, achondroplasia, CAH and ADH1. We are progressing 17 ongoing clinical trials and we are preparing for commercialization, to bring our first investigational therapy to patients. There has never been a more exciting moment to be at the forefront of the revolution taking place in genetic medicine," said BridgeBio CEO and founder Neil Kumar, Ph.D.
Recent pipeline progress and corporate updates:
BridgeBio and Eidos Therapeutics enter into merger agreement: BridgeBio to acquire all outstanding shares of common stock of Eidos it does not already own; agreement unanimously approved by special committee of Eidos’ independent directors. Transaction removes the operational complexity of the current ownership structure and allows BridgeBio to fully invest in opportunities around the investigational drug, acoramidis, including subsequent studies to potentially broaden the evidence for its usage, and accelerate its commercial development using BridgeBio’s established infrastructure. Proposed transaction expected to be completed in the first quarter of 2021, subject to certain conditions, including approval by both BridgeBio and Eidos stockholders.
Fosdenopterin (formerly BBP-870/ORGN001) – Synthetic cPMP for molybdenum cofactor deficiency (MoCD) Type A: FDA acceptance of NDA under Priority Review designation with Breakthrough Therapy Designation and Rare Pediatric Disease Designation previously granted. There are currently no approved therapies for the treatment of MoCD Type A, which results in severe and irreversible neurological injury for infants and children. This is BridgeBio’s first NDA acceptance.
New academic partnerships: Established collaboration agreements with the Salk Institute and the University of Colorado Anschutz Medical Campus to advance the discovery of therapies for genetically driven diseases.
BridgeBio Pharma R&D Day: Held a virtual R&D Day on September 29, 2020. Presentation replay can be found on BridgeBio’s investor website here.
Major milestones anticipated over the next 12-18 months for BridgeBio’s four core value drivers:
Acoramidis (AG10) – TTR stabilizer for ATTR: Completed screening in September for pivotal Phase 3 ATTRibute-CM clinical trial of acoramidis in patients with ATTR cardiomyopathy. The study enrolled more than 600 subjects with either wild-type or variant TTR across more than 80 sites in 18 countries. Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 or early 2022 and from Part B in 2023. If Part A is successful, intend to file for regulatory approval of acoramidis in 2022.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Remain on track to report initial data from the ongoing Phase 2 dose ranging study by end of 2021. Achondroplasia is the most common form of genetic short stature and one of the most commonly known genetic diseases, with 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known therapy in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical stage development.
Encaleret – CaSR antagonist for ADH1: Initiated Phase 2 clinical study and dosed first patients, with topline proof-of-concept results anticipated in 2021. If the development program is successful, encaleret would 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 in the United States.
BBP-631 – AAV5 gene therapy candidate for CAH: Investigational New Drug (IND) application-enabling studies for AAV gene therapy proceeding. Remain on track to initiate a first in human Phase 1/2 study and report initial data in 2021. CAH is one of the most prevalent genetic diseases thought to be addressable with AAV gene therapy, with more than 75,000 cases in the United States and European Union.
Third quarter 2020 financial results:
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $710.7 million as of September 30, 2020, compared to $577.1 million at December 31, 2019. The net increase in cash balance of $133.6 million reflects $537.0 million in net proceeds received from the issuance of our 2.50% Convertible Senior Notes due 2027 (2027 Notes), $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares in capped call transactions in connection with the issuance of our 2027 Notes, $49.3 million payment related to capped call option, $13.3 million payments of interest on our debts, and $289.9 million primarily related to operating expenses.
Cash, cash equivalents and marketable securities, excluding restricted cash, decreased by $130.2 million compared to our balance as of June 30, 2020, which was $840.9 million. The decrease in cash reflects $9.2 million payments of interests on our debts and $121.0 million primarily related to operating expenses.
Operating Expenses
Operating expenses for the three and nine months ended September 30, 2020 were $128.1 million and $355.1 million, respectively, as compared to $81.3 million and $214.3 million, respectively, for the same periods in the prior year. The increases in operating expenses of $46.8 million and $140.8 million during the respective periods were attributable to the increase in external-related costs and increase in headcount to support the progression in our research and development programs, including our increasing research pipelines, and overall growth of our operations.
Operating expenses for the three months ended September 30, 2020 increased by $3.5 million when compared to the operating expenses for the three months ended June 30, 2020 of $124.6 million.
Our research and development expenses have not been significantly impacted by the global outbreak of COVID-19 for the periods presented. While we experienced some initial 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 of COVID-19 on our operating expenses is currently unknown.
The condensed consolidated balance sheet as of December 31, 2019 is derived from the audited consolidated financial statements as of that date. Certain reclassifications have been made to conform to the September 30, 2020 condensed consolidated balance sheet presentation.
December 31, 2019 amounts include long-term marketable securities of $31.1 million.