Encaleret
Search documents
13 Biotech Stocks Charging Ahead With New 52-week Highs - Will Near-term Catalysts Drive More Gains?
RTTNews· 2025-10-14 13:49
"All there is to investing is picking good stocks at good times and staying with them as long as they remain good companies", said Warren Buffet, reminding us that patience and discipline are key to successful investing. Stocks quietly climbing to new 52-week highs could be a sign that the market is recognizing consistent performance, strong fundamentals, or upcoming catalysts.The following are some of the stocks that hit new 52-week highs yesterday. Do you already own any of them? Are any on your watchlis ...
BridgeBio Pharma (NasdaqGS:BBIO) Earnings Call Presentation
2025-09-10 12:00
Encaleret & ADH1 Overview - Encaleret is an investigational oral calcilytic drug being developed as a potential treatment for Autosomal Dominant Hypocalcemia Type 1 (ADH1)[35, 38] - ADH1 is caused by activating variants in the CASR gene, leading to dysregulation of calcium homeostasis, decreased blood calcium, and increased urinary calcium[16, 17] - Diagnosis of ADH1 requires genetic testing, but is often delayed with a median age of 25 years[23, 24] - Conventional therapy for ADH1, including calcium and activated vitamin D, does not correct the underlying pathophysiology and may worsen long-term complications[17, 27] Clinical Development & Results - Phase 2 study of encaleret in 13 ADH1 patients showed rapid and sustained normalization of serum calcium, urine calcium, and PTH levels over 42 months[49, 56, 57] - In the Phase 2 study, 69% of participants responded to encaleret, achieving both blood and urine calcium in the target range at Week 24[59] - Encaleret was generally well-tolerated over 42 months of outpatient administration in the Phase 2 study, with most Treatment-Emergent Adverse Events (TEAEs) being mild[54] - The CALIBRATE Phase 3 study of encaleret in ADH1 is ongoing, with topline results expected in Fall 2025[62, 63] Market Opportunity - The estimated prevalent population of ADH1 in the US is approximately 12,000 individuals, with 73% being symptomatic[72] - ADH1 is emerging as the leading isolated cause of nonsurgical hypoparathyroidism, accounting for approximately 20% of all cases[76, 78] - The company projects peak year sales of encaleret to be over $1 billion, drawing a comparison to the XLH market[73]
BridgeBio to Present Phase 2 Proof-of-Concept Data for Encaleret in Post-Surgical Hypoparathyroidism
Globenewswire· 2025-09-02 11:30
Core Insights - BridgeBio Pharma, Inc. is set to present Phase 2 data on post-surgical hypoparathyroidism and skeletal dysplasia at the ASBMR Annual Meeting 2025 in Seattle from September 5-8, 2025 [1] Group 1: Post-Surgical Hypoparathyroidism Presentation - An oral presentation titled "The Oral Calcilytic Encaleret Reduced Urinary Calcium While Maintaining Blood Calcium in Individuals with Post-Surgical Hypoparathyroidism" will be presented by Dr. Iris Hartley on September 6 at 11:30 am PT [2] Group 2: Skeletal Dysplasia Poster Sessions - A poster session on "Infigratinib Low Dose Therapy Is an Effective Strategy in Improving Bone Growth in a Hypochondroplasia Mouse Model" will be presented by Dr. Bhavik Shah on September 5 at 5:00 pm PT and September 7 at 2:00 pm PT [2] - Another poster session titled "Infigratinib Improves Skull Measures in a Mouse Model of Crouzon/Pfeiffer Syndromes" will also be presented by Dr. Bhavik Shah on September 6 at 2:00 pm PT [3] Group 3: Company Overview - BridgeBio Pharma, Inc. focuses on discovering, creating, testing, and delivering transformative medicines for genetic diseases, with a pipeline that includes early science to advanced clinical trials [3] - Founded in 2015, the company aims to leverage advances in genetic medicine to expedite patient care [3]
BridgeBio Reports Second Quarter 2025 Financial Results and Business Updates
GlobeNewswire News Room· 2025-08-05 20:01
Core Insights - BridgeBio Pharma reported strong commercial progress with 3,751 unique patient prescriptions for Attruby as of August 1, 2025, indicating robust month-over-month growth in treatment-naïve patients [1][3] - The company achieved total second-quarter revenue of $110.6 million, driven by $71.5 million in U.S. Attruby net product revenue, $1.6 million in royalty revenue, and $37.5 million in license and services revenue [1][12] - Attruby's clinical profile was reinforced by new analyses from the ATTRibute-CM study, showing significant benefits for patients, including a 59% relative risk reduction in mortality and a 43% reduction in annual frequency of cardiovascular hospitalization due to atrial fibrillation [1][8] Commercial Progress - The launch of Attruby has accelerated, with nearly double the product revenue this quarter, attributed to growing adoption among healthcare providers [4] - The company ended the quarter with $756.9 million in cash and marketable securities, positioning it well for ongoing commercial activities and upcoming clinical trials [2][9] Pipeline Overview - Key upcoming milestones include topline results from the CALIBRATE study for encaleret in ADH1 expected in fall 2025, and from the PROPEL 3 study for infigratinib in achondroplasia expected in early 2026 [2][11] - The FORTIFY Phase 3 study of BBP-418 for LGMD2I/R9 has completed enrollment, with topline results anticipated in fall 2025 [1][6] Financial Performance - Total revenues for the three months ended June 30, 2025, were $110.6 million, a significant increase from $2.2 million in the same period last year, primarily due to the success of Attruby [12][13] - Operating costs for the quarter were $244.8 million, up from $177.7 million year-over-year, largely driven by increased selling, general, and administrative expenses related to the commercial launch of Attruby [14][15] Key Program Updates - Attruby is positioned as a near-complete transthyretin stabilizer for treating transthyretin amyloid cardiomyopathy (ATTR-CM), with ongoing studies to further validate its efficacy [7][31] - The company is also advancing its pipeline with BBP-418 and encaleret, aiming to address significant unmet needs in genetic diseases [9][11]
Novel Human Genetics Evidence Confirms Estimates of Genetic Prevalence, Underdiagnosis, and Potentially Greater Symptom Burden of Gain-of-Function CASR Variants Associated with ADH1
Globenewswire· 2025-07-23 11:30
Core Insights - The study confirms the genetic prevalence of Autosomal Dominant Hypocalcemia Type 1 (ADH1) and highlights a significant gap in diagnosis and care for affected individuals [1][2][3] Group 1: Genetic Findings - The average frequency of gain-of-function CASR variants is approximately 3.7 per 100,000, equating to around 25,000 carriers in the US and EU [1][6] - Only about 20% of individuals with genetic variants linked to ADH1 have an established diagnosis, indicating a major gap in disease recognition [1][2] - Nine novel gain-of-function CASR variants were identified, associated with symptoms consistent with ADH1 [1] Group 2: Clinical Implications - ADH1 is a common genetic form of hypoparathyroidism, presenting serious symptoms like seizures and irregular heart rhythms due to hypocalcemia [2][5] - The findings emphasize the importance of genetic testing in uncovering the underlying causes of hypoparathyroidism [2][3] Group 3: Clinical Trials and Future Developments - BridgeBio's Phase 3 clinical trial of encaleret for ADH1 is fully enrolled with 71 participants, expected to report topline results in the second half of 2025 [4] - If successful, encaleret would be the first approved therapy for ADH1, with plans to initiate a study in chronic hypoparathyroidism in 2026 [4][8] Group 4: Company Overview - BridgeBio Pharma focuses on discovering and delivering transformative medicines for genetic diseases, with a commitment to applying advances in genetic medicine [9]
BridgeBio Reports First Quarter 2025 Financial Results and Business Updates
GlobeNewswire News Room· 2025-04-29 20:01
Core Insights - BridgeBio Pharma reported $36.7 million in net product revenue for Attruby in its first full quarter, with 2,072 unique patient prescriptions written by 756 prescribers as of April 25, 2025 [1][2] - The company has made significant progress in its clinical trials, including the full enrollment of the observational run-in study for hypochondroplasia Phase 2 trial ahead of schedule [1][6] - BridgeBio ended the quarter with $540.6 million in cash and cash equivalents, excluding anticipated regulatory milestone payments of $105 million for ex-U.S. approvals of BEYONTTRA [1][9] Commercial Progress - The first full quarter of Attruby net product revenue was $36.7 million, with 2,072 unique patient prescriptions written by 756 healthcare providers since FDA approval in November 2024 [2][13] - The Chief Commercial Officer highlighted a 42% reduction in all-cause mortality and a 50% decrease in cardiovascular hospitalizations at 30 months for patients treated with Attruby [3] Pipeline Overview - Acoramidis for ATTR-CM is approved in the U.S., EU, Japan, and UK, with the first participant in the ACT-EARLY Phase 3 trial expected to be dosed in 2025 [4] - BBP-418 for LGMD2I/R9 and encaleret for ADH1 have completed enrollment in their Phase 3 studies, with topline results expected in the second half of 2025 [4][6] - Infigratinib for achondroplasia is fully enrolled, with topline results anticipated in early 2026 [4][6] Key Program Updates - Attruby has shown strong clinical efficacy and safety, contributing to its growth in the ATTR-CM treatment paradigm [5][6] - The Phase 2 proof-of-principle study of encaleret in hypoparathyroidism showed 78% of participants achieving normal calcium levels within 5 days, with plans to advance to a registrational study in 2026 [6][7] Financial Updates - Cash and cash equivalents decreased to $540.6 million as of March 31, 2025, primarily due to operating activities and loan repayments [9][10] - Total revenues for the three months ended March 31, 2025, were $116.6 million, down from $211.1 million in the same period the previous year, mainly due to a decrease in license and services revenue [10][11] - The company recorded a net loss attributable to common stockholders of $167.4 million for the quarter, compared to a loss of $35.2 million for the same period in 2024 [20]