On March 2, 2020 BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, reported its fourth quarter and full year 2019 financial results and recent progress across its portfolio, which now includes more than 20 drug development and discovery programs (Press release, BridgeBio, MAR 2, 2020, View Source [SID1234555041]).

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2019 was a year of significant growth and milestone achievements for BridgeBio. The company initiated its first New Drug Application (NDA) with the United States Food and Drug Administration (FDA) and added five new drug development and discovery programs to its pipeline, which target diseases in endocrinology, ophthalmology, otology, neurology and musculoskeletal therapeutic areas. Veteran biotech and pharmaceutical industry leaders joined BridgeBio’s board of directors and the company as senior leaders, including the recent board appointment of Ronald J. Daniels, president of Johns Hopkins University. The company went public on June 27, 2019 and raised approximately $401 million in gross proceeds in its initial public offering.

"BridgeBio was founded nearly five years ago on the back of two big ideas: that there had to be a better way to finance critical biomedical research and that there was a tremendous amount of medical innovation that was trapped, without a way to move into the clinic where it could help patients," said BridgeBio founder and CEO Neil Kumar, Ph.D. "Today BridgeBio has more than 20 drug development and discovery programs in our pipeline. We initiated our first new drug application with the FDA and we are on track to file our second later this year, along with the anticipated filing of multiple investigational new drug applications. We are demonstrating that our model is working and has the potential to produce an array of life-changing therapies for people in need."

Pipeline growth:

We recently disclosed five new genetic medicine product candidates in the pipeline:

Encaleret – Calcium sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1): IND application for the treatment of ADH1 became effective in late 2019; Phase 2 ready

Zuretinol – Synthetic retinoid for inherited retinal disease due to RPE65 or LRAT gene mutations: Phase 2/3 study initiating in 2020

BBP-418 – Substrate supplementation therapy for limb-girdle muscular dystrophy type 2i (LGMD2i)

BBP-472 – Brain-permeable inhibitor of PI3KB for children with autism-spectrum disorders (ASD) characterized by loss of the PTEN protein. This program is in preclinical development.

BBP-815 – AAV gene therapy for nonsyndromic hearing loss caused by recessive mutations in the TMC1 gene. This program is undergoing early proof of concept animal studies.

Fourth quarter 2019 and recent pipeline progress:

Mendelian

Low-dose infigratinib—FGFR1-3 inhibitor for achondroplasia: Initiated clinical program consisting of an observational study (PROPEL, NCT04265651) and Phase 2 dose-ranging study (PROPEL2, NCT04265651) in children with achondroplasia, the most common form of genetic short stature.

Fosdenopterin – cPMP replacement therapy for MoCD type A: Initiated a rolling submission of an NDA with the United States FDA for the treatment of patients with molybdenum cofactor deficiency (MoCD) type A.

Topical patidegib gel for Gorlin syndrome and high-frequency basal cell carcinoma1: Completed enrollment of pivotal Phase 3 clinical trial in patients with Gorlin Syndrome (NCT03703310) and initiated Phase 2 study in high-frequency basal cell carcinoma (NCT04155190).

BBP-265 (AG10) – TTR stabilizer for ATTR: Presented positive data from Phase 2 Open Label Extension (NCT03536767) in transthyretin amyloid cardiomyopathy at the American Heart Association’s 2019 Scientific Sessions.

Targeted Oncology

Infigratinib – FGFR1-3 inhibitor for FGFR+ cancer: Received FDA Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for the treatment of cholangiocarcinoma, and initiated Phase 3 study in advanced 1L cholangiocarcinoma as a first-line therapy (PROOF trial, NCT03773302).

BBP-398 – SHP2 inhibitor for treatment-resistant cancer: Presented data (link to poster) highlighting the discovery and preclinical activity of our potent and selective SHP2 inhibitor potentially both as monotherapy and in combination with approved agents.

Gene Therapy

BBP-812 – Gene therapy candidate for Canavan disease: Opened a natural history study in Canavan disease (treatcanavan.com) and presented preclinical data (link to poster) demonstrating intravenous (IV) dosing of BridgeBio’s experimental therapy for Canavan disease (BBP-812) achieved broad central nervous system delivery.

BBP-631 – Gene therapy candidate for CAH: Presented preclinical update (link to poster) for gene therapy candidate BBP-631 in congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency wherein IV dosing of non-human primates with BBP-631 resulted in durable delivery and expression of the gene product to the adrenal tissue.

PellePharm, which is focused on developing patidegib topical gel, 2% entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

Upcoming milestones:

Anticipate disclosing additional new product candidates and filing multiple new INDs in 2020

BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa: Plan to share topline data from the ongoing Phase 1/2 study (NCT03752905) in 2020.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Anticipate dosing of first child in Phase 2 dose-ranging study (PROPEL2) in the coming months.

BBP-265 (AG10) – TTR stabilizer for ATTR: On track to complete enrollment in the Phase 3 study of AG10 in ATTR-CM in 2H20 (ATTRibute-CM). A Phase 3 study of AG10 in ATTR-PN (ATTRibute-PN) is on track to begin in 1H20.

Encaleret – Calcium sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1): Phase 2b ready.

Infigratinib – FGFR1-3 inhibitor for FGFR+ cancer: Plan to present updated results at a major oncology meeting in 2020. Remain on track to submit new drug application for FDA approval in cholangiocarcinoma in 2020. Plan to dose first patients in a Phase 3 study in FGFR3+ adjuvant urothelial carcinoma (NCT04197986) and a Phase 2 tumor-agnostic study in patients with fusions or activating mutations in the FGFR1, 2 or 3 genes in 2020.

Organizational Growth:

Ronald J. Daniels, board member: President of Johns Hopkins University joined board of directors.

Jennifer Cook, board member and senior advisor: Genentech veteran who led Roche Pharma’s European commercial business joined board of directors and serves as a special advisor to BridgeBio.

Eli Wallace, chief scientific officer in residence for oncology: Medicinal chemist and strategic executive leader who oversaw entire research organizations at companies such as Peloton Therapeutics and Array BioPharma joined BridgeBio to lead oncology research.

Fourth quarter and full-year 2019 financial results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $577.1 million as of December 31, 2019 compared with $436.1 million at December 31, 2018.

License revenue

License revenue for the fourth quarter of 2019 was $13.8 million and for the full year 2019 was $40.6 million. The full year 2019 revenue was comprised mainly of the upfront payment recognized by our subsidiary company, Eidos Therapeutics, Inc. upon execution of its License Agreement with Alexion Pharmaceuticals, Inc.

Operating Expenses

Operating expenses for the fourth quarter of 2019 were $92.5 million, as compared to $65.6 million for the same period in the prior year. The increase in operating expenses of $26.9 million was primarily attributable to increase in headcount and external-related costs to support the growth of our operations.

Full year 2019 operating expenses were $306.8 million, as compared to $183.7 million for the full year in 2018. The increase of $123.1 million was mainly due to the increase in external-related costs to support the progression in our research and development programs and growth of our operations as well as increase in headcount.

Other Income (Expense), Net

Other income (expense), net for the fourth quarter of 2019 was ($5.5) million, as compared to $16.3 million for the same period in 2018. The change of $21.8 million was primarily attributable to a gain of $19.3 million that we recognized on deconsolidation of our controlled variable interest entity, PellePharm, Inc. in 2018.

Other income (expense), net for the full year 2019 was ($22.3) million, as compared to $14.2 million for the full year in 2018. The change of $36.5 million was primarily attributable to an increase in our net loss of equity method investments by $20.9 million in 2019 and the gain of $19.3 million on deconsolidation of our controlled variable interest entities, PellePharm, Inc.

Upon the closing of our IPO on July 1, 2019, we completed a reorganization (the "Reorganization"), whereby all unitholders of BridgeBio Pharma LLC, our predecessor entity ("BBP LLC") exchanged their units for shares of common stock of BridgeBio Pharma, Inc. (the "Corporation"), and BBP LLC became a wholly-owned subsidiary of the Corporation. Subsequent to the Reorganization, as the sole managing member, BridgeBio operates and controls all of BBP LLC’s businesses and affairs. The condensed consolidated financial statements as of and for the year ended December 31, 2018 are derived from the audited consolidated financial statements as of that date and were retroactively adjusted, including shares and per share amounts, as a result of the Reorganization. See the BridgeBio Pharma, Inc. Registration Statement on Form S-1 (File No. 333-231759) for additional details.

December 31, 2019 amount includes long-term marketable securities of $31.1 million.