On August 11, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, reported its financial results for the second quarter ending June 30, 2020 and provided an update on the company’s operations (Press release, BridgeBio, AUG 11, 2020, View Source [SID1234563437]).
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Since the beginning of 2020, BridgeBio has initiated four company-sponsored clinical trials, progressed its additional 11 ongoing clinical trials, submitted three Investigational New Drug (IND) applications to the U.S. Food and Drug Administration (FDA), and completed the rolling submission of its first New Drug Application (NDA) with the FDA. During the quarter, BridgeBio strengthened its corporate governance by adding three world-class independent directors to its board. In addition, it recently entered into a partnership with Perceptive Advisors-founded company LianBio, expanding BridgeBio’s global reach into China.
BridgeBio remains on track with each of its four core value drivers – acoramidis (formerly AG10, TTR stabilizer) for ATTR cardiomyopathy, low-dose infigratinib (FGFRi) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia (CAH), and encaleret (CaSRi) for autosomal dominant hypocalcemia type 1 (ADH1) – and the company believes it is adequately financed through key readouts for each of these programs. Notably, BridgeBio dosed the first child in its Phase 2 clinical trial of infigratinib in achondroplasia in July.
The strategic collaboration with LianBio will initially focus on targeted oncology. BridgeBio’s near-term economics includes a total of $26.5 million in upfront and milestone payments. BridgeBio will additionally receive up to $505 million in future milestone payments, tiered royalty payments ranging from single- to double-digits and increase its equity interest via investment in LianBio. BridgeBio CEO Neil Kumar has also been appointed to the LianBio board of directors.
Across the company, BridgeBio’s drug engineering platform continues to deliver. Strengthening its ability to discover new targets, it established collaboration agreements with Johns Hopkins University and University of Florida and continues to assess a wide variety of new programs in the genetic disease space. BridgeBio’s pre-clinical platform has expanded to include additional modalities such as antisense oligonucleotides and deepened expertise in critical areas such as molecular modeling and novel statistical approaches to genetics. Its clinical platform has grown and now encompasses more than 350 trial sites in over 25 countries.
"On a risk-adjusted basis we are in a great position to produce meaningful medicines for patients and meaningful returns to investors over the next 18 to 24 months. This is exemplified by our disease-modifying, first or best-in-class therapeutic candidates for ATTR, achondroplasia, ADH1, and CAH. We intend to deliver on this goal by expanding our industry-leading target identification and research engine and global clinical development infrastructure, and look forward to delivering our medicines, once approved, to patients through our growing commercial organization," said BridgeBio CEO and founder Neil Kumar, Ph.D.
Recent pipeline progress and corporate updates:
Low-dose infigratinib – Selective FGFR inhibitor for achondroplasia: Dosed first child in the Phase 2 clinical program (PROPEL 2) (NCT04265651).
BBP-418 – Glycosylation substrate pro-drug for LGMD2i: Dosed first subject in Phase 1 clinical trial in healthy volunteers.
Expansion into China through partnership with LianBio: BridgeBio entered into a strategic collaboration with Perceptive Advisors-founded LianBio, expanding its reach into China and other major Asian markets. The initial focus of the collaboration will be targeted oncology. Under the terms of the agreements, LianBio receives commercial rights in China and selected Asian markets and will participate in clinical development activities for BridgeBio’s Phase 3 FGFR inhibitor infigratinib and Phase 1-ready SHP2 inhibitor BBP-398. BridgeBio’s near-term economics includes a total of $26.5 million in upfront and milestone payments. BridgeBio will additionally receive up to $505 million in future milestone payments, tiered royalty payments ranging from single- to double-digits and increase its equity interest via investment in LianBio. BridgeBio CEO Neil Kumar has also been appointed to the LianBio board of directors.
Three new independent directors added to BridgeBio’s board:
○ Brent Saunders, former Allergan CEO and biopharma deal-maker
○ Randy Scott, Ph.D., genomics pioneer and entrepreneur
○ Andrew Lo, Ph.D., renowned economist and BridgeBio co-founder
New academic partnerships: Established collaboration agreements with Johns Hopkins University and University of Florida to accelerate the development of new medicines in genetically driven diseases.
BridgeBio Pharma R&D Day: BridgeBio will hold a virtual R&D Day on Tuesday, Sept. 29, 2020 from 8:30 am ET – noon. The event will be webcast, with a link available on the event calendar at View Source
Major milestones anticipated over the next 18-24 months for BridgeBio’s four core value drivers:
Acoramidis (formerly AG10) – TTR stabilizer for ATTR: Remain on track to complete enrollment in the Phase 3 ATTRibute-CM study in ATTR cardiomyopathy (ATTR-CM) in the first half of 2021, with topline data expected in the first half of 2022. Acoramidis is a potentially best in class TTR stabilizer for ATTR-CM, a large and growing disease affecting >400K patients globally, and one of the first drug candidates to arise from BridgeBio’s drug engineering platform.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Remain on track to report initial data from the ongoing Phase 2 dose ranging study by YE2021. Achondroplasia is the most common form of genetic short stature and one of the most commonly-known genetic diseases, with >55K cases in the US and EU. 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 Autosomal Dominant Hypocalcemia Type 1 (ADH1): Remain on track to initiate the planned Phase 2 study in 2020, with potential proof-of-concept data available 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 calcium-sensing receptor gene estimated to be carried by 12k individuals in the US.
BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): IND-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 >75K cases in the US and EU.
Second quarter 2020 financial results:
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $840.9 million as of June 30, 2020 compared to $577.1 million at December 31, 2019. The net change in cash balance of $263.8 million reflects $537.0 million in net proceeds received from the issuance of our 2.50% Convertible Senior Notes due 2027, $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, $49.3 million payment related to capped call option and the remaining payment of $173.0 million primarily related to operating expenses.
Operating Expenses
Operating expenses for the second quarter and first half of 2020 were $124.6 million and $227.1 million, as compared to $69.3 million and $133.1 million, respectively, for the same periods in the prior year. The increases in operating expenses of $55.2 million and $94.0 million during the 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.
Our research and development expenses have not been significantly impacted by the global outbreak of COVID-19 for the periods presented. While we have 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.