- Announced consistently positive results from the Phase 3 ATTRibute-CM study of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), including a highly statistically significant result observed on the primary endpoint with a Win Ratio of 1.8 (p<0.0001) and clinically meaningful and consistent separation observed on measures of mortality, morbidity, function, and quality of life; the Company intends to file a New Drug Application (NDA) for acoramidis with the U.S. Food and Drug Administration (FDA) by the end of 2023
- Presented updated six-month results from Cohort 5 of PROPEL2, a Phase 2 trial of infigratinib in children with achondroplasia, at the Endocrine Society 2023 Annual Conference (ENDO 2023), demonstrating a continued potentially best-in-class efficacy and well-tolerated safety profile, and a mean increase in annualized height velocity (AHV) of 3.38cm/year with no treatment-related adverse events
- Dosed the first participant in FORTIFY, a global Phase 3 study of BBP-418 in patients with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) and met with the FDA to discuss the use of glycosylated alpha-dystroglycan (αDG) levels as a surrogate endpoint; based on this meeting, the Company believes there is potential to pursue Accelerated Approval in the U.S. for BBP-418
- Shared 18-month data from the long-term extension of the Phase 2 study of encaleret in patients with autosomal dominant hypocalcemia type 1 (ADH1) at ENDO 2023, including observation of a rapid and sustained treatment effect; Phase 3 CALIBRATE registrational trial remains ongoing, with topline results expected to be announced in the first half of 2024
- Phase 1/2 trial of BBP-631 for treatment of congenital adrenal hyperplasia (CAH) continuing to progress with an update planned by the end of 2023
- Three lead KRAS programs are advancing, with an Investigational New Drug (IND) application planned for first-in-class direct KRASG12C (ON) inhibitor BBO-8520 in 2023 as well as a recent selection of a clinical candidate for PI3Kα:RAS breaker with the intention to file an IND application in 2024
- Ended the quarter with $353 million in cash, cash equivalents, marketable securities, and short-term restricted cash, and $50 million of investments in equity securities, providing runway into 2H 2024
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