Core Insights - The company announced positive Phase III data from the CALIBRATE clinical trial for patients with autosomal dominant hypocalcemia type 1, marking a significant advancement for ADH1 patients [2][3]. Group 1 - The webinar was hosted by the company to present the top line results of the CALIBRATE Phase III trial [1]. - The CEO expressed gratitude towards the team and stakeholders involved in the trial, highlighting the importance of the results for patients and their families [2].

Summary of BridgeBio Pharma's Conference Call on ADH1 Calibrate Phase 3 Results Company and Industry Context - **Company**: BridgeBio Pharma - **Industry**: Biotechnology, specifically focusing on rare genetic conditions and therapies Key Points and Arguments 1. **Positive Phase 3 Data**: The Calibrate clinical trial for autosomal dominant hypocalcemia type 1 (ADH1) showed positive results, marking a significant advancement for patients suffering from this condition [2][8][22] 2. **Therapeutic Impact**: Encaleret, the investigational drug, demonstrated the ability to normalize calcium and parathyroid hormone (PTH) levels in a majority of patients, addressing the root cause of ADH1 [5][11][22] 3. **Study Results**: - 76% of participants achieved target serum and urine calcium levels within 24 weeks of treatment with encaleret [8][17] - 91% of participants showed a positive response in PTH levels [18] - Encaleret was well tolerated, with no discontinuations due to adverse effects [9][21] 4. **Prevalence of ADH1**: The estimated prevalence of ADH1 is approximately 1 in 25,000, translating to about 12,000 individuals in the U.S. [9][10] 5. **Mechanism of Action**: Encaleret acts as a calcilytic, targeting the calcium-sensing receptor (CASR) to restore mineral homeostasis, which is disrupted in ADH1 patients [10][11] 6. **Future Studies**: Plans to initiate a phase 2/3 study in pediatric patients and a phase 3 study in chronic hypoparathyroidism are underway [24][26] Additional Important Information 1. **Commercial Strategy**: BridgeBio Pharma is preparing for a potential blockbuster launch of encaleret, leveraging its existing commercial infrastructure from previous product launches [26][29] 2. **Market Dynamics**: The ADH1 market is underrecognized, presenting a significant opportunity for diagnosis and treatment expansion [27][66] 3. **Regulatory Engagement**: The company plans to engage with the FDA for a New Drug Application (NDA) submission in the first half of 2026 [24] 4. **Patient Identification**: Increased awareness and new ICD-10 codes are expected to facilitate the identification of ADH1 patients, enhancing market access [45][66] 5. **Safety Profile**: The safety profile of encaleret appears favorable, with manageable adverse events primarily related to the drug's mechanism of action [21][94] This summary encapsulates the critical insights from the conference call, highlighting the advancements in treatment for ADH1 and the strategic direction of BridgeBio Pharma.

Core Insights - The CALIBRATE study of encaleret for patients with ADH1 met all pre-specified primary and key secondary efficacy endpoints [1] - Encaleret demonstrated significant efficacy in achieving target serum and urine calcium levels compared to conventional therapy [2] - The drug was well-tolerated with no discontinuations related to the study drug [1] Efficacy Results - 76% of participants treated with encaleret achieved both serum and urine calcium within target ranges at Week 24, compared to 4% on conventional therapy at Week 4 (p<0.0001) [1][2] - In a key secondary analysis, 91% of encaleret-treated participants had intact PTH above the lower limit of the reference range at Week 24, compared to 7% on conventional therapy at Week 4 (p<0.0001) [1] - By Day 3 after randomization, 71% of encaleret participants had an albumin-corrected serum calcium within the reference range [4] Safety and Tolerability - Encaleret was well-tolerated, with safety findings consistent with known ADH1 biology [12] - 65 of 67 randomized participants (97%) chose to continue in the long-term extension of the study [5] Future Plans - The company plans to submit a New Drug Application (NDA) to the FDA in the first half of 2026 [1][6] - Registrational studies of encaleret in chronic hypoparathyroidism and pediatric ADH1 are planned for 2026 [1][6] Study Design - The CALIBRATE study enrolled 70 adults with ADH1, randomizing 67 participants in a 2:1 ratio (encaleret: standard of care) [4] - The primary endpoint was defined as the proportion of participants achieving target serum and urine calcium levels [2]

Core Insights - Several biotech and healthcare stocks experienced significant after-hours trading gains due to clinical updates, strategic deals, and regulatory milestones [1] Company Summaries - **Genenta Science S.p.A. (GNTA)**: Shares closed at $2.40, down 27.71% during regular trading, but rebounded to $2.45, up 2.08% in after-hours. The volatility was driven by a $15 million registered direct offering and a strategic collaboration announcement [2] - **EDAP TMS S.A. (EDAP)**: Closed at $1.81, down 22.58%, but rose 8.29% to $1.96 in after-hours trading. The recovery appears technical, with no new news, and is supported by upcoming investor meetings at the UBS Global Healthcare Conference [3] - **BridgeBio Pharma Inc. (BBIO)**: Ended regular trading at $64.50, up 1.48%, and climbed 6.71% to $68.83 after hours. The increase reflects anticipation for Phase 3 CALIBRATE trial results for ADH1, with topline data expected on October 29 [4] - **OrthoPediatrics Corp. (KIDS)**: Closed at $16.91, down 0.29%, but surged 6.45% to $18.00 after releasing Q3 financial results, reporting a GAAP loss per share of $(0.50) despite 12% revenue growth [5][6] - The company raised its full-year 2025 revenue guidance to $233.5 million to $234.5 million, indicating projected growth of 14% to 15% compared to the prior year [7] - **Modular Medical Inc. (MODD)**: Dipped 1.30% to $0.5392 but rallied 5.38% to $0.5682 after hours following the successful completion of its Stage 1 ISO 13485:2016 audit, a key step toward CE Mark certification [8] - **Vivani Medical Inc. (VANI)**: Rose 3.09% to $1.67 and added 3.65% to $1.7310 after closing a $15.7 million capital raise, which included a registered direct offering and private placement, indicating insider confidence [9] - **Amarin Corp. plc (AMRN)**: Slipped 1.56% to $18.94 but rebounded 5.07% to $19.90 after the FDA revised fenofibrate drug labeling, which may favor Amarin's Vascepa in the lipid management space [10]

Core Insights - BridgeBio Pharma, Inc. is set to release topline results from the CALIBRATE Phase 3 trial for autosomal dominant hypocalcemia type 1 (ADH1) on October 28, 2025, before market opening [1] - A conference call will be held by management at 8:00 a.m. ET on the same day to discuss the trial data [1] Company Overview - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on discovering, creating, testing, and delivering transformative medicines for genetic diseases [3] - Founded in 2015, the company has a pipeline that includes programs from early science to advanced clinical trials [3] - The team consists of experienced professionals dedicated to applying advances in genetic medicine to benefit patients quickly [3]

Core Insights - BridgeBio Pharma (BBIO) shares experienced a significant increase of 17.1%, closing at $63.56, driven by impressive trading volume and a prior 8.4% gain over the last four weeks [1][2] Company Performance - The stock's rally followed the announcement of positive top-line data from the pivotal phase III FORTIFY study for BBP-418, which met all primary and secondary interim analysis endpoints and showed good tolerability without new safety concerns [2] - The company is expected to report a quarterly loss of $0.88 per share, reflecting a year-over-year decrease of 2.3%, while revenues are projected to reach $105.47 million, marking a substantial increase of 3763.3% compared to the same quarter last year [3] - The consensus EPS estimate for the upcoming quarter has been revised 3.1% higher in the last 30 days, indicating a positive trend that typically correlates with stock price appreciation [4] Industry Context - BridgeBio Pharma is categorized within the Zacks Medical - Generic Drugs industry, which includes other companies like Supernus Pharmaceuticals (SUPN), that recently closed at $52.72, down 0.2% [5] - Supernus has seen a 12.3% return over the past month, with its consensus EPS estimate increasing by 14.3% to $0.3, although this represents a significant decline of 71.7% from the previous year [6]

Core Insights - BridgeBio Pharma, Inc. (NASDAQ:BBIO) is a biotechnology company focused on developing treatments for genetic diseases, particularly rare diseases with significant unmet medical needs [1] - The company has received a price target of $69 from Raymond James, indicating a potential increase of approximately 9.17% from its current stock price of $63.21 [2][6] - Positive interim results from the Phase III FORTIFY trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 have been announced, marking a significant milestone for the company [3][6] - Following the announcement of trial results, BBIO's stock price increased to $63.56, reflecting a 17.14% rise and strong investor confidence [4][6] - BBIO's market capitalization stands at approximately $12.15 billion, indicating its substantial presence in the biotech industry, with a trading volume of 8,339,257 shares today [5]

BridgeBio Pharma (NASDAQ:BBIO) shares rose after it announced positive results from its late-stage study of BBP-418 on patients with limb-girdle muscular dystrophy type 2I/R9. The study successfully met all its main and secondary goals, showing a safety profile that aligns with ...

Core Insights - The webinar is focused on the interim analysis results of the Phase III FORTIFY study for Limb-Girdle Muscular Dystrophy Type 2i/R9 conducted by BridgeBio Pharma [1] Group 1 - The conference is hosted by Krista, the conference operator, indicating a formal presentation setting [1] - Dr. Neil Kumar, the Chief Executive Officer of BridgeBio Pharma, is set to present the findings [1]

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