Core Insights - Dyne Therapeutics Inc. has announced positive topline results from its Phase 1/2 DELIVER trial for z-rostudirsen, a treatment for Duchenne muscular dystrophy (DMD) [1][3] - The trial demonstrated a statistically significant increase in muscle content-adjusted dystrophin expression to 5.46% of normal at 6 months, meeting its primary endpoint [3] - Dyne Therapeutics is preparing to submit a Biologics License Application for US Accelerated Approval in Q2 2026 [3] Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing therapeutics for neuromuscular diseases in the US [4] - The investigational drug z-rostudirsen utilizes Dyne's FORCE platform to produce near full-length dystrophin [2] Regulatory Designations - Z-rostudirsen has received multiple designations from the US FDA, including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease, as well as Orphan Drug designations from the FDA, EMA, and MHLW in Japan [3] Clinical Trial Results - The DELIVER trial also provided positive long-term clinical data from its ongoing open-label extension and long-term extension portions [2] - Functional improvements were observed across all six prespecified clinical endpoints at 6 months compared to baseline, with declines noted in the pooled placebo group [3]

Core Insights - Praxis Precision Medicines Inc. is recognized as a promising biotech stock by Wall Street analysts, particularly due to its advancements in drug development for essential tremor and epilepsy [1][4] Group 1: Drug Development Progress - The company successfully completed a pre-New Drug Application meeting with the US FDA for Ulixacaltamide, an oral drug aimed at treating essential tremor, with plans to submit a New Drug Application early next year [1] - Positive results were confirmed from the EMBOLD study, which evaluated riluzole for patients with SCN2A- and SCN8A-related developmental and epileptic encephalopathies, showing a 53% placebo-adjusted reduction in seizures without serious adverse events [2] - Updated Phase 2 RADIANT trial data indicated that the candidate vormatrigine achieved nearly 100% median seizure reduction in focal onset seizures and about 80% in generalized epilepsy [3] Group 2: Analyst Ratings and Price Target - Analysts at TD Cowen reiterated a Buy rating on Praxis and raised the price target from $251 to $353, reflecting confidence in the company's epilepsy pipeline [3] - The interim EMBOLD results revealed that relutrigine provided a 53% placebo-adjusted reduction in seizures, contributing to the positive outlook from analysts [3]

Biomarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best biotech stocks to buy according to Wall Street analysts. On November 18, BioMarin Pharmaceutical (NASDAQ:BMRN) presented at the Jefferies London Healthcare Conference, where CEO Alexander outlined progress on the company’s $500 million cost transformation program, now about two-thirds complete, and announced a goal of reaching a 40% non-GAAP operating margin next year, up from 19% in 2023. Leerink Cuts BioMarin Pharmaceutical Inc. (BMRN) Price ...

Core Insights - TuHURA Biosciences is advancing its Phase 3 program of IFx-2.0 as an adjunctive therapy with Keytruda® for advanced Merkel cell carcinoma, with enrollment completion targeted for Q4-2026 [1][13] - The company raised $15.6 million in a recent equity financing transaction, providing a cash runway to achieve key milestones across its development programs [1][2] - A mini KOL symposium highlighted the potential of targeting VISTA in acute myeloid leukemia (AML) and the combination with menin inhibitors for NPM1 mutated relapsed/refractory AML [1][3] Company Developments - The Phase 3 trial of IFx-2.0 has been initiated, marking a significant step in the company's strategy to address resistance to cancer immunotherapy [2][13] - The merger with Kineta has added a Phase 2 ready VISTA inhibiting antibody to the company's pipeline, enhancing its therapeutic offerings [2][14] - Preliminary data from the IFx-2.0 basket trial and insights on inhibiting Delta Opioid Receptor (DOR) are expected to be presented at scientific conferences in 2026 [3] Clinical Insights - VISTA is identified as the only checkpoint significantly upregulated in AML, particularly in high-risk subtypes, contributing to low response rates in patients treated with menin inhibitors [11] - The combination of TBS-2025 (VISTA inhibiting antibody) with menin inhibitors shows promise in improving survival outcomes in murine models of AML [11] - Key opinion leaders expressed enthusiasm for the potential of TBS-2025 in combination therapies for high-risk AML and patients unfit for intensive treatments [3][11]

Core Insights - The Phase 3 sunRIZE study of ersodetug for congenital hyperinsulinism did not meet its primary or key secondary endpoints, showing a 45% reduction in hypoglycemia events at the highest dose compared to a 40% improvement in the placebo group, which was not statistically significant [1][2] - The study involved 63 participants across multiple countries and aimed to evaluate the efficacy and safety of ersodetug in patients experiencing hypoglycemia despite standard care [8] Study Results - The primary endpoint assessed the change in average weekly hypoglycemia events, which did not show significant improvement with ersodetug compared to placebo [2] - The key secondary endpoint, measuring average daily percent time in hypoglycemia, also did not demonstrate significant results, with a 25% reduction in time in hypoglycemia for the 10 mg/kg dose versus a 5% increase in the placebo group [2] - Dosing regimens included 5 and 10 mg/kg administered every other week for three doses, followed by every four weeks for the remaining 24-week treatment duration, achieving target drug concentrations across all age groups [3] Safety Observations - Safety data from the study were generally favorable, supporting the safe use of ersodetug in both pediatric and adult patients [4] - Two participants experienced serious hypersensitivity reactions leading to early discontinuation, but the incidence of serious allergic reactions was low compared to other biologic treatments [4] - The most common adverse event reported was hypertrichosis, which was mild and self-limiting [4] Company Statements and Future Plans - The company expressed disappointment over the study results but indicated that certain aspects warrant further investigation and a thorough evaluation to understand the outcomes better [5] - The company plans to meet with the FDA under the Breakthrough Therapy Designation to discuss next steps for the program [5] - An ongoing Phase 3 study for tumor hyperinsulinism (upLIFT) is expected to release topline results in the second half of 2026 [5] About Ersodetug - Ersodetug is a fully human monoclonal antibody designed to decrease insulin receptor over-activation, potentially treating hypoglycemia from any form of congenital or acquired hyperinsulinism [9] - The company focuses on rare diseases, particularly hypoglycemia caused by hyperinsulinism, and aims to provide meaningful treatment options for affected patients [10]

Chaired by globally renowned nuclear medicine expert Prof. Ken Herrmann, M.D. Other Board members James Cook, Jason Lewis, Ph.D., and Michael Morris, M.D. bring significant clinical and industry expertise, supporting transition from early clinical validation to strategic development ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as ...

Data demonstrates the significant and sustained tumor regression achieved by the CAPTN-3 platform across two distinct tri-specific antibodies, IM1240 and IM1305, targeting different tumor antigens Transcriptomic analysis across ~11,000 TCGA samples shows NKG2A expression is strongly associated with tumor expression of 5T4 or TROP2, supporting inclusion of the NKG2A arm in CAPTN-3 designs NKG2A arm significantly contributes to IM1240 anti-cancer immune activity in PD1-resistant patient-derived explants REHOV ...

Core Viewpoint - The discussion focuses on addressing acute agitation associated with schizophrenia and bipolar disorders, emphasizing the need for patient-centered innovations in non-clinical settings [1][3]. Group 1: Company Mission and Commitment - BioXcel Therapeutics aims to transform patient care by advancing solutions that alleviate the burden of acute agitation on patients, caregivers, and the healthcare system [3]. - The company recognizes the significant challenges posed by episodes of acute agitation, particularly outside clinical environments, highlighting the urgency for practical innovations [3]. Group 2: Event Structure and Participants - The event features a roundtable discussion moderated by Anjalee Khemlani, an award-winning journalist known for her in-depth reporting on healthcare [2]. - The participation of clinical experts is intended to provide valuable insights into the management of agitation in real-world settings [2].

Core Points - Exelixis is hosting its 2025 R&D Day focused on building next-generation oncology franchises [1] - The event features guest speakers who will discuss specific disease focus areas [1] - The company will make forward-looking statements regarding future developments and performance [2] Company Developments - Exelixis is emphasizing potential advancements in discovery, product development, and regulatory matters [2] - The company is exploring market and growth opportunities in the oncology sector [2] Risks and Uncertainties - Actual results may differ materially from the company's forward-looking statements due to various risks [2] - Key risk factors include product commercial success, market competition, and regulatory approval processes [2]

Core Insights - IDEAYA Biosciences has completed the targeted enrollment of 435 patients in the Phase 2/3 trial (OptimUM-02) for darovasertib in combination with crizotinib for treating first-line HLA*A2-negative metastatic uveal melanoma [1][2] - The company anticipates reporting median progression-free survival (PFS) data in Q1 2026 to support a potential accelerated approval filing in the U.S. [1][6] - Darovasertib has received FDA Breakthrough Therapy Designation and Fast Track designation for its use in metastatic uveal melanoma [4] Trial Details - The OptimUM-02 trial is a multi-arm, multi-stage, open-label study comparing the darovasertib and crizotinib combination against investigator's choice of treatment [3] - Primary endpoints include median PFS and median overall survival (OS), which will support potential accelerated and full approval filings in the U.S. [3] - Previous data from the Phase 1/2 trial (OptimUM-01) indicated a median OS of 21.1 months and median PFS of 7.0 months for the combination treatment [3] Regulatory Designations - Darovasertib has been designated as an Orphan Drug by the U.S. FDA for uveal melanoma, including metastatic cases [4] - The company is also enrolling patients in a pivotal Phase 3 trial of single-agent darovasertib in the neoadjuvant setting for primary uveal melanoma (OptimUM-10) [4] Company Overview - IDEAYA Biosciences focuses on precision medicine in oncology, aiming to develop transformative therapies for cancer through a combination of drug discovery, structural biology, and bioinformatics [5] - The company has a pipeline targeting synthetic lethality and antibody-drug conjugates for solid tumor indications [5]