BridgeBio Pharma Reports Third Quarter 2023 Financial Results and Business Update

- Presented detailed positive results from the ATTRibute-CM Phase 3 study of acoramidis at the European Society of Cardiology (ESC) Congress, demonstrating that patients survived more and were hospitalized less than has been seen in other interventional studies of transthyretin amyloid cardiomyopathy (ATTR-CM) to the Company’s knowledge, including real-world evidence presented at recent cardiology medical meetings

- The ATTRibute-CM data presented at ESC also demonstrated proportions of improvement in the treated population on 6-minute walk distance and NT-proBNP greater than observed in prior interventional studies of ATTR-CM to the Company’s knowledge; these results support a differentiated profile for acoramidis in the contemporary care of ATTR-CM

- A New Drug Application (NDA) for acoramidis is expected to be filed with the U.S. Food and Drug Administration (FDA) by the end of 2023; if approved, this would make acoramidis the next treatment option available to patients with ATTR-CM

- Held positive regulatory meetings with the FDA and the European Medicines Agency (EMA) on the path for infigratinib in children with achondroplasia; reached alignment on a single pivotal one-year, randomized, placebo-controlled Phase 3 clinical trial, clearing the path for the expected initiation of the trial by the end of 2023

- Shared data from Phase 2 trial of BBP-418 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) at the Annual Congress of World Muscle Society, which showed that early changes in glycosylated alpha-dystroglycan (αDG) levels predicted subsequent ambulatory improvements, supporting the validity of glycosylated αDG as a potential surrogate endpoint for Accelerated Approval in FORTIFY, the ongoing Phase 3 study of BBP-418 in LGMD2I/R9

- Published Phase 2b trial data of encaleret in patients with autosomal dominant hypocalcemia type 1 (ADH1) in the New England Journal of Medicine; results demonstrated rapid and sustained restoration of physiologic mineral homeostasis in all 13 participants with ADH1; registrational Phase 3 trial CALIBRATE is ongoing with announcement of topline results expected in 2024

- Phase 1/2 trial of BBP-631 for treatment of congenital adrenal hyperplasia (CAH) is ongoing with the dose-escalation portion fully enrolled; Company expects to share data from the study in early 2024

- Three lead KRAS programs are advancing; first-in-class direct KRASG12C (ON) inhibitor BBO-8520, whose novel direct KRAS (ON) targeting mechanism was observed to be potentially superior to KRAS (OFF) as presented at the recent Triple Meeting, remains on track for an Investigational New Drug (IND) application in 2023; PI3Kα:RAS breaker candidate BBO-10203 and pan-KRAS program remain on track for IND and DC nomination, respectively, in 2024

- Signed a multi-year partnership with National Resilience, Inc. (Resilience) to advance BBP-631, BBP-812 and future gene therapy treatments; the deal is intended to reduce manufacturing costs, which have historically been approximately 50%-60% of the Gene Therapy budget, and is expected to help the Company expedite development of gene therapies going forward

- Raised gross proceeds of approximately $316 million via at-the-market (ATM) facility and a private placement equity financing (PIPE) from a syndicate of the largest investment management firms as detailed in the forthcoming S-3 filing

- Ended the third quarter with $522 million in cash, cash equivalents, and short-term restricted cash, and $38 million of investments in equity securities

PALO ALTO, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) --  BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today reported its financial results for the third quarter ended September 30, 2023, and provided an update on the Company’s operations.

“We continue to be extremely grateful to the patient and physician communities with whom we collaborate; their partnership has helped us to realize tremendous advancement recently across the programs that make up our portfolio,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. “We are excited about the progress from our late-stage pipeline, and have begun to highlight areas of differentiation for the upcoming, potentially blockbuster launch of our ATTR-CM asset. In particular, recent real-world evidence on ATTR-CM therapies presented at the HFSA scientific sessions suggests that the survival levels observed on acoramidis treatment in our Phase 3 study are indeed differentiated even in the context of the contemporary care setting as compared to other agents in the field. This continues to reinforce our hypothesis that better stabilization leads to better outcomes for patients with ATTR-CM.”

BridgeBio’s key programs:

• Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
  • In August 2023, the Company presented detailed positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM; a highly statistically significant result was observed on the primary endpoint with a Win Ratio of 1.8 (p