Ascendis Pharma A/S (NASDAQ:ASND) ranks among the best biotech stocks to buy. UBS reaffirmed its 307 price target and Buy rating on Ascendis Pharma A/S (NASDAQ:ASND) on September 23 in response to MBX’s clinical study for the treatment of persistent hypoparathyroidism. Given Yorvipath’s higher placebo-adjusted response rates of 52.9% at 4 weeks and 69% at 26 weeks in its clinical trials, UBS maintains that Ascendis Pharma’s medication continues to lead the hypoparathyroidism treatment space, despite supp ...

Summary of BridgeBio Pharma (BBIO) 2025 Conference Call Company Overview - **Company**: BridgeBio Pharma - **Key Products**: Acoramidis, Encaleret - **Focus Areas**: Limb-girdle muscular dystrophy, autosomal dominant hypocalcemia type 1 (ADH1) Key Points Acoramidis Launch and Performance - The launch of acoramidis is described as having a strong start, with increasing momentum in both treatment naive and switch settings [2][3] - Acoramidis is noted for its superior clinical profile, being the only near-complete stabilizer on the market [2] - Key clinical outcomes include: - 50% reduction in hospitalization at 30 months - 40% reduction in all-cause mortality (ACM) and hospitalization at 30 months [3] - Metrics such as gross-to-net revenue have stabilized in the 30% to 40% range, with compliance around 80% [3][5] - New patient additions have accelerated, with 3,751 patients reported by Q2, up from 2,072 in Q1, indicating a growth rate of approximately 90 to 100 patients per week [8][9] Market Dynamics and Competitive Landscape - The discontinuation of Pfizer's Vyndaqel in 2026 is seen as an opportunity for BridgeBio to capture patients who would have been prescribed Vyndaqel, as these physicians are already predisposed to using stabilizers [12] - The polymorph patent covering both Vyndaqel and Vyndamax is expected to protect acoramidis from generic competition, raising the bar for any potential generic manufacturers [13][14] Encaleret Development - Encaleret is being developed for ADH1, with a prevalence rate of about 1 in 25,000, translating to approximately 12,000 individuals in the U.S. [22] - The diagnosis rates for ADH1 are lagging behind prevalence due to the lack of indicated treatments [22][23] - Phase two results showed that 70% of patients normalized both blood and urine calcium levels, which is a significant improvement over conventional therapies [34][35] - The phase three study is designed to evaluate the concurrent normalization of blood and urine calcium over six months, informed by phase two results [35] Limb-Girdle Muscular Dystrophy Insights - Ribitol is crucial for muscle function as it is used by FKRP to glycosylate alpha-dystroglycan, a stabilizing protein in muscle fibers [45] - Phase two results indicated an approximate doubling of glycosylated alpha-dystroglycan levels in patients, with a significant reduction in creatine kinase (CK) levels [52][53] - The study aims for a 5% absolute change in glycosylated alpha-dystroglycan and a 40% reduction in CK for potential accelerated approval [56][57] Competitive Landscape for Hypoparathyroidism - Encaleret is positioned against Yorvipath, with potential advantages such as being an oral medication and offering a urine calcium benefit [43][44] - The treatment goals focus on normalization of blood calcium, urine calcium, and phosphate, rather than complete independence from conventional therapy [41] Future Outlook - The company is optimistic about the continued growth of acoramidis and the potential for encaleret to address unmet needs in hypoparathyroidism and ADH1 [14][44] - Upcoming data presentations and interim analyses are expected to provide further insights into the efficacy and market positioning of both acoramidis and encaleret [56][57] Additional Important Insights - The company emphasizes the importance of genetic testing for undiagnosed patients with ADH1, suggesting that many patients with hypoparathyroidism are not genotyped [28][23] - The phase three trial for encaleret is designed to be powered for success based on the promising phase two results, indicating a strong confidence in the product's potential [35][40]

Group 1 - The article expresses a beneficial long position in the shares of ASND, indicating a positive outlook on the company's stock performance [1] - The author emphasizes that the opinions presented are personal and not influenced by any business relationships with the mentioned companies [1] - There is a clear distinction made that the commentary does not constitute personalized investment advice, urging readers to conduct their own due diligence [2] Group 2 - The article highlights that past performance is not indicative of future results, which is a common disclaimer in investment discussions [3] - It is noted that the views expressed may not reflect those of Seeking Alpha as a whole, indicating a diversity of opinions among analysts [3] - The article clarifies that Seeking Alpha does not act as a licensed securities dealer or investment adviser, reinforcing the need for independent research by investors [3]