Biomarin Pharmaceutical

BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder

6 days ago

• BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701. • INZ-701 is a Phase 3 enzyme replacement therapy being developed for ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones with no currently approved treatments. • The first pivotal data readout for INZ-701 in children is expected in early 2026, with potential regulatory approval and market launch in 2027.

Takeda Spotlights Three Late-Stage Readouts as Potential "Inflection Point" Amid Vyvanse Generic Competition

13 days ago

• Takeda Pharmaceutical is positioning three upcoming late-stage clinical trial readouts as a strategic "inflection point" to offset revenue losses from Vyvanse's generic competition. • The Japanese pharmaceutical giant is focusing on new product launches and pipeline advancement to maintain growth momentum, with particular emphasis on rare disease and neuroscience therapeutic areas. • Industry analysts view Takeda's pipeline strategy as critical for the company's long-term financial stability, as it navigates the challenging transition period of losing exclusivity for one of its top-selling medications.

Phenylketonuria Pipeline Shows Robust Growth with 25+ Therapies in Development

a month ago

• DelveInsight's latest report reveals a robust phenylketonuria (PKU) pipeline with over 20 companies developing 25+ therapeutic candidates, signaling significant advancement in treatment options for this rare metabolic disorder. • PTC Therapeutics' sepiapterin has reached registration stage with the FDA accepting its New Drug Application, while Otsuka Pharmaceutical's JNT-517 advances in Phase III trials as a first-in-class oral therapy for PKU patients regardless of age or genotype. • Novel approaches including enzyme replacements, gene therapies, and innovative oral formulations are transforming PKU management beyond traditional dietary restrictions, with companies like BioMarin, Sanofi, and Relief Therapeutics actively developing next-generation treatments.

New Report Reveals Critical Insights into Clinical-Stage Pharma Partnerships from 2020-2025

2 months ago

• A comprehensive new industry report analyzes over 1,670 clinical-stage partnering agreements in pharma and biotech from 2020-2025, providing unprecedented access to deal structures and financial terms. • The report reveals detailed intelligence on how licensing agreements typically grant exclusive rights across Phase I-III trials, with multi-component structures involving collaborative R&D and commercialization strategies. • Business development professionals can now access actual contract documents and payment triggers often missing from press releases, enabling more effective negotiation strategies and competitive deal structuring.

iOnctura Initiates Phase II OCULE-01 Trial of Roginolisib for Metastatic Uveal Melanoma

2 months ago

• iOnctura has dosed the first patient in its randomized Phase II OCULE-01 study evaluating roginolisib, an allosteric PI3Kδ modulator, in patients with metastatic uveal melanoma. • The European Medicines Agency recently granted Orphan Drug Designation for roginolisib, providing 10 years of market exclusivity in the EU, complementing the FDA's ODD designation received in 2023. • In previous Phase I trials, roginolisib demonstrated excellent safety and doubled overall survival to 16 months in metastatic uveal melanoma patients compared to historical controls.

Incyte's Povorcitinib Shows Significant Efficacy in Phase 3 Trials for Hidradenitis Suppurativa

2 months ago

• Incyte's oral JAK1 inhibitor povorcitinib demonstrated statistically significant results in two Phase 3 trials (STOP-HS1 and STOP-HS2) for moderate to severe hidradenitis suppurativa, meeting primary endpoints at both 45mg and 75mg doses. • The drug showed particularly strong efficacy in patients previously exposed to biologics, with up to 45% achieving HiSCR50 response compared to 19.5% on placebo, addressing a critical unmet need in treatment-resistant cases. • With a favorable safety profile and rapid onset of action, povorcitinib could become the first oral targeted therapy for hidradenitis suppurativa, with regulatory submissions planned worldwide based on these positive results.

Kodiak Sciences Completes Enrollment in GLOW2 Phase 3 Trial for Diabetic Retinopathy Treatment

2 months ago

• Kodiak Sciences has successfully enrolled over 250 patients in its GLOW2 Phase 3 trial of tarcocimab tedromer for diabetic retinopathy, with topline data expected in Q1 2026. • The GLOW2 study mirrors the successful GLOW1 trial design with an additional loading dose, potentially offering a twice-yearly dosing regimen that could transform treatment for millions of diabetic retinopathy patients. • If successful, Kodiak plans to file a single Biologics License Application for tarcocimab covering wet AMD, diabetic retinopathy, and retinal vein occlusion following completion of the DAYBREAK study in Q2 2026.

FDA Approves Genentech's TNKase for Acute Ischemic Stroke with Simplified Administration

3 months ago

• The FDA has approved Genentech's TNKase, a clot-dissolving agent, for treating acute ischemic stroke in adults, marking the company's second stroke treatment approval. • TNKase offers a streamlined five-second intravenous bolus administration, presenting a significant advantage over the current standard treatment Activase's 60-minute infusion protocol. • The approval is supported by a multi-center non-inferiority study across 22 Canadian stroke centers, demonstrating comparable safety and efficacy to Activase in patients with disabling neurological deficits.

Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments

3 months ago

• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027. • KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks. • The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.

Pfizer Discontinues Beqvez Gene Therapy for Hemophilia B Amid Low Market Demand

3 months ago

• Pfizer has announced the cessation of development and commercialization of Beqvez, its FDA-approved gene therapy for hemophilia B, citing limited interest from patients and healthcare providers. • The decision marks Pfizer's complete exit from viral gene replacement therapies, following earlier withdrawals from Duchenne muscular dystrophy and hemophilia A programs. • The company will shift focus to other treatments including Hympavzi for hemophilia A, while maintaining support for existing Beqvez clinical trial participants.

AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR

3 months ago

• The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine. • Marketed AAV therapies include Luxturna for hereditary retinal dystrophy, Zolgensma for spinal muscular atrophy, and Glybera for lipoprotein lipase deficiency, with numerous promising candidates in late-stage development. • Improved diagnostic technologies, regulatory support, and significant investment from companies like Spark Therapeutics, Novartis, and BioMarin are accelerating the development of next-generation AAV therapies for previously untreatable genetic disorders.

Anavex's Blarcamesine Shows Promise in Long-Term Alzheimer's Treatment, EMA Filing Accepted

4 months ago

• Anavex Life Sciences' blarcamesine demonstrates sustained cognitive and functional benefits in early Alzheimer's patients over three years of continuous oral treatment. • The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for blarcamesine, a potential novel oral treatment for Alzheimer's disease. • Blarcamesine targets upstream Alzheimer's pathology through autophagy enhancement and has shown a favorable safety profile in clinical trials. • Anavex is also evaluating blarcamesine for other CNS indications, including Parkinson’s disease and Rett syndrome.

BridgeBio Pharma Receives Optimistic Buy Recommendation Amid Promising Drug Developments

4 months ago

Citi analyst David Lebowitz maintains a Buy rating on BridgeBio Pharma, citing strong initial sales data for its drug Attruby and a well-progressing pipeline, with a price target of $45.00.

European Commission Approves BioMarin's Roctavian for Severe Hemophilia A

4 months ago

• The European Commission granted conditional marketing approval for BioMarin's Roctavian (valoctocogene roxaparvovec) for severe hemophilia A in adults without Factor VIII inhibitors. • Roctavian, a one-time gene therapy, delivers a functional Factor VIII gene using an adeno-associated virus vector, potentially offering a curative treatment option. • BioMarin plans to launch Roctavian in Europe with a 'net' price of approximately €1.5 million, starting in Germany and followed by France, contingent on reimbursement agreements. • BioMarin intends to refile for Roctavian's approval in the US, anticipating a decision by late March 2023, with a potential price range of $2 to $3 million after discounts.

Pfizer Abandons Hemophilia A Gene Therapy, Jeopardizing Sangamo's Financial Stability

5 months ago

• Pfizer has terminated its partnership with Sangamo Therapeutics for the hemophilia A gene therapy, giroctocogene fitelparvovec, despite promising Phase III trial results. • This decision by Pfizer casts doubt on the commercial viability of first-generation hemophilia A gene therapies, influenced by the slow market uptake of BioMarin's Roctavian. • Sangamo's stock plummeted by 56% following the announcement, as the company loses potential milestone payments of up to $220 million from Pfizer. • Sangamo now intends to explore all options for advancing the gene therapy, including seeking a new collaboration partner, while facing financial challenges with limited cash reserves.

Korea Approves Voxzogo for Pediatric Chondrodysplasia Treatment

5 months ago

The Korean Ministry of Food and Drug Safety (MFDS) has approved Voxzogo (vosoritide) for treating chondrodysplasia in children aged four months and older. This breakthrough therapy, developed by BioMarin Pharmaceutical, represents the first treatment option for pediatric chondrodysplasia patients in Korea, offering new hope for those affected by this rare genetic disorder.

Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024

5 months ago

• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach. • Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein. • A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease. • The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.

Viridian's Veligrotug Shows Promise in Phase 3 Trial for Chronic Thyroid Eye Disease

5 months ago

• Viridian Therapeutics' veligrotug met all primary and secondary endpoints in the THRIVE-2 Phase 3 trial for chronic thyroid eye disease (TED). • The trial demonstrated statistically significant improvements in proptosis, diplopia, and clinical activity scores (CAS) after five infusions of veligrotug. • A Biologics License Application (BLA) submission for veligrotug is on track for the second half of 2025, potentially transforming TED treatment. • Viridian is advancing VRDN-003, a subcutaneous anti-IGF-1R antibody, with topline data expected in the first half of 2026.

Hemophilia Treatment Advances: Gene Therapy, Prophylaxis, and On-Demand Therapies Highlighted at ASH 2024

5 months ago

• ASH 2024 highlighted significant progress in hemophilia care since Hemlibra's approval, including gene therapies for hemophilia A and B, offering curative potential. • Despite advances, unmet needs persist, including spontaneous bleeds in prophylaxis patients, limited gene therapy access, and concerns about long-term risks like liver toxicity. • Emerging therapies like Pfizer's giroctocogene fitelparvovec and Sanofi's fitusiran show promise, with ongoing trials and regulatory reviews potentially expanding treatment options. • Novel on-demand treatments like Staidson's bemiltenase alfa offer alternatives, particularly in price-sensitive markets, demonstrating a 12-hour bleed clearance rate of 83%.

Hemophilia B Therapeutics: Advances in Clinical Trials and Emerging Therapies

6 months ago

• Several companies are actively involved in developing therapies for Hemophilia B, with Belief Biomed's drug candidates reaching Phase III clinical trials. • Recent trials include Novo Nordisk's assessment of Nonacog Beta Pegol in Chinese patients and ApcinteX Ltd's study of SerpinPC in severe Hemophilia A or B. • Emerging therapies like BBM-H901 (Belief Biomed) and Fidanacogene elaparvovec (Pfizer/Spark Therapeutics) are under investigation across various clinical stages. • Hemophilia B therapies are being developed using various routes of administration, including oral, intravenous, and subcutaneous, and molecule types, such as gene therapy and recombinant fusion proteins.