Core Viewpoint - PTC Therapeutics has received marketing authorization from the European Commission for Sephience™ (sepiapterin) to treat phenylketonuria (PKU) in patients of all ages and disease severities, marking a significant advancement in treatment options for this rare metabolic disorder [1][2][3] Group 1: Product Information - Sephience™ (sepiapterin) is indicated for the treatment of hyperphenylalaninaemia (HPA) in both adult and pediatric patients with PKU, acting as a natural precursor of the enzymatic co-factor BH4, which is essential for the enzyme phenylalanine hydroxylase (PAH) [4] - The product functions as a dual pharmacological chaperone, improving the activity of defective PAH enzymes and effectively reducing blood phenylalanine (Phe) levels [4] - The European launch of Sephience will begin in Germany in the first half of July 2025, with ongoing review for approval in other countries including Japan and Brazil [3] Group 2: Clinical Evidence - The European approval is based on statistically significant results from the Phase 3 APHENITY trial, demonstrating a durable treatment effect and allowing participants to liberalize their diet in the long-term extension study [2] Group 3: Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, leveraging scientific expertise and a global commercial infrastructure to maximize value for patients [6] - The company aims to provide access to best-in-class treatments for patients with unmet medical needs, supported by a robust and diversified pipeline of transformative medicines [6]

Ad hoc announcement pursuant to Art. 53 LR Idorsia to receive USD 50 million (an additional USD 30 million) approval milestone payment in return for a reduction in Simcere potential sales milestones and tiered royalty payments. Allschwil, Switzerland – June 23, 2025Idorsia Ltd. (SIX: IDIA) today announced that Simcere Pharmaceuticals Group Ltd (2096.HK, “Simcere”) has received approval for QUVIVIQ® (daridorexant) from the Chinese National Medical Products Administration for the treatment of adult patients w ...

Core Viewpoint - Sarepta Therapeutics, Inc. is under investigation for potential violations of federal securities laws related to the safety profile of its product Elevidys, which has been linked to fatal acute liver failure in patients [1][2]. Company Overview - Sarepta Therapeutics is a biopharmaceutical company focused on developing treatments for rare diseases, with Elevidys being its most significant product aimed at treating Duchenne muscular dystrophy [2]. Product Safety Concerns - The company has previously emphasized the safety of Elevidys, claiming that its benefits outweigh the risks. However, it has been revealed that Elevidys can cause fatal acute liver failure in some patients [2]. Stock Performance - Following the announcement of a patient's death due to acute liver failure on March 18, 2025, Sarepta's stock price dropped by $27.81, or over 27%, from $101.35 to $73.54 per share [3]. - After a second patient death was reported on June 15, 2025, Sarepta's stock fell by $15.24, or more than 42%, from $36.18 to $20.94 per share [3]. Legal Implications - Investors in Sarepta may have legal options available to them, as indicated by the investigation and the potential for class action lawsuits [4].

BOSTON, June 20, 2025 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) today announced that it has postponed its 2025 Annual Meeting of Stockholders (the “Annual Meeting”), which was scheduled to be held on June 25, 2025, in light of the previously announced acquisition (the “Acquisition”) by BioMarin Pharmaceutical Inc. (“BioMarin”). If the Acquisition is completed, there will be no Annual Meeting involving public stockholders. If the Acquisition is not completed, the Board of Directors will take su ...

Core Viewpoint - Cyclacel Pharmaceuticals has successfully completed a private placement of its convertible Series F Preferred Stock, raising gross proceeds of $3.0 million, which will be utilized for working capital and general corporate purposes [1][2]. Group 1: Financing Details - The private placement involved the issuance of convertible Series F Preferred Stock and warrants to purchase a total of 9,810,000 shares of common stock at varying exercise prices of $0.51, $0.60, and $0.68 per share [1]. - The offering closed on June 20, 2025, and the warrants will expire five years from the date of issuance [1]. - Each share of Preferred Stock is convertible into 3.27 shares of Common Stock, subject to stockholder approval as per Nasdaq listing rules [3]. Group 2: Use of Proceeds - The net proceeds from the offering are intended for working capital and general corporate purposes, which, along with existing cash, is expected to extend the company's cash runway into the third quarter of 2025 [2]. Group 3: Regulatory and Legal Aspects - The securities issued in the private placement have not been registered under the Securities Act of 1933 and were sold in reliance on Regulation S [4]. - The offering was conducted directly to investors without the involvement of a placement agent, underwriter, broker, or dealer [4]. Group 4: Company Overview - Cyclacel Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative cancer medicines, particularly through its anti-mitotic program, plogosertib, which targets PLK1 in various cancer types [7].

WALTHAM, Mass., June 20, 2025 (GLOBE NEWSWIRE) -- Zenas BioPharma, Inc. (“Zenas” or the “Company”) (Nasdaq: ZBIO), a clinical-stage global biopharmaceutical company committed to being a leader in the development and commercialization of therapies for autoimmune diseases, today announced that on June 16, 2025 (the “Grant Date”), the Compensation Committee of the Company’s Board of Directors granted a non-qualified stock option to purchase 112,000 shares of the Company’s common stock to a newly hired employee ...

Company Overview - ArriVent BioPharma, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancer patients [2] - The company aims to leverage its team's extensive drug development experience to advance its lead candidate, firmonertinib, and a pipeline of novel therapeutics [2] Product Information - Firmonertinib is an oral, mutation-selective EGFR inhibitor effective against both classical and uncommon EGFR mutations, including PACC and exon 20 insertion mutations [3] - The drug was approved in China in March 2021 for first-line treatment of advanced NSCLC with specific EGFR mutations [3] Regulatory Designations - Firmonertinib received Breakthrough Therapy Designation from the U.S. FDA for treating previously untreated locally advanced or metastatic non-squamous NSCLC with EGFR exon 20 insertion mutations [4] - The drug also holds Orphan Drug Designation from the U.S. FDA for treating NSCLC with various EGFR mutations [4] Clinical Trials - Firmonertinib is currently undergoing a global Phase 3 trial for first-line NSCLC patients with EGFR exon 20 insertion mutations and a Phase 1b study for patients with EGFR PACC mutations [5] - The drug is also part of a clinical combination study targeting advanced or metastatic NSCLC patients with classical EGFR mutations, in collaboration with Beijing InnoCare Pharma Tech Co., Ltd. [5] Market Context - Lung cancer is the leading cause of cancer-related deaths globally, with NSCLC accounting for approximately 85% of all cases [6] - Uncommon EGFR mutations, such as exon 20 insertion and PACC mutations, represent significant unmet medical needs, with exon 20 mutations constituting about 9% and PACC mutations about 12% of all EGFR mutations [6]

Core Insights - The European Commission has approved VYVGART® (efgartigimod alfa) for subcutaneous injection as a monotherapy for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins [1][4] - VYVGART is the first targeted IgG Fc-antibody fragment for CIDP and introduces a novel mechanism of action for CIDP treatment in over 30 years [2] - The approval is based on positive results from the ADHERE clinical trial, the largest study of CIDP patients to date, and is valid across all 27 EU Member States, as well as Iceland, Norway, and Liechtenstein [4] Company Overview - Halozyme Therapeutics, Inc. specializes in biopharmaceutical solutions aimed at improving patient experiences and outcomes, particularly through its ENHANZE® drug delivery technology [5] - The company has impacted over one million patients through its commercialized products and has licensed its ENHANZE® technology to major pharmaceutical companies including Roche, Takeda, and Pfizer [5] - Halozyme also develops drug-device combination products using advanced auto-injector technologies to enhance patient comfort and adherence [6] Strategic Partnerships - The approval of VYVGART represents a significant milestone in Halozyme's partnership with argenx, expanding access to CIDP treatment in Europe [3] - The company continues to collaborate with various pharmaceutical and biotechnology firms to leverage its ENHANZE® technology for improved drug delivery [5][6]

Core Insights - GH Research PLC has submitted a complete response to the FDA regarding the clinical hold of its Investigational New Drug Application (IND) for GH001, ahead of schedule [1][2] - The company is focused on developing GH001, a treatment for treatment-resistant depression (TRD), utilizing a proprietary inhalation method [3] Company Developments - The response to the FDA includes comprehensive data and completed toxicology studies, demonstrating the company's commitment to regulatory compliance and patient care [2] - GH001 has shown promising results in the Phase 2b GH001-TRD-201 trial, achieving a significant reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) score by -15.5 points compared to placebo on Day 8 (p<0.0001) [3]

VANCOUVER, British Columbia, June 20, 2025 (GLOBE NEWSWIRE) -- Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE:7JO) (“Rakovina” or the “Company”) is pleased to announce that the effective date for the previously announced share consolidation (the “Consolidation”) will be June 24, 2025. As outlined in the Company’s news release dated May 15, 2025, the Consolidation will be conducted on the basis of one (1) post-consolidation common share (“Post-Consolidation Share”) for every ten (10) pre-consolidation common s ...