Thu.Jun 13, 2024

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With phase 3 miss, Pfizer's DMD candidate is no threat to Sarepta's Elevidys ahead of key label expansion: analysts

Fierce Pharma

With a phase 3 miss following a fatality in a related study and marking what analysts are calling the “final nail” in Pfizer’s Duchenne muscular dystrophy (DMD) gene therapy coffin, Sarepta Therape | With a phase 3 miss and severe safety signals, Pfizer's Duchenne muscular dystrophy gene therapy candidate is no competition to Sarepta's established Elevidys, according to analysts.

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Modifier gene therapy – clinical development and manufacturing considerations

European Pharmaceutical Review

Tell us about Ocugen’s lead programme, OCU400. What’s the latest and what’s next? OCU400 is a modifier gene therapy aimed at treating retinitis pigmentosa and Leber congenital amaurosis (LCA). In April 2024, Ocugen received US Food and Drug Administration (FDA) clearance to initiate the Phase III liMeliGhT clinical trial for OCU400 for retinitis pigmentosa.

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Pfizer expects 8 blockbuster cancer drugs to come from each of 4 focus areas: exec

Fierce Pharma

Pfizer's oncology division plans to have eight blockbusters by 2030, with contributions from each of its four focused therapeutic areas.

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Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval

MedCity News

A Pfizer gene therapy for Duchenne muscular dystrophy failed its Phase 3 clinical trial. But analysts draw distinctions between that therapy and Sarepta Therapeutics’ Elevidys, which awaits an FDA decision that could grant it full regulatory approval. The post Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval appeared first on MedCity News.

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Optimizing Clinical Supply Strategy: Navigating Challenges & Finding Your Ideal Model

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Talc plaintiffs file for restraining order to block Johnson & Johnson's 3rd bankruptcy attempt

Fierce Pharma

As Johnson & Johnson has tried to resolve talcum powder lawsuits that have been consolidated in a multidistrict litigation in New Jersey, the company has had to deal with two groups of plaintif | J&J has had to deal with two groups of plaintiffs in its talc lawsuits—one that wants to accept a settlement and the other that doesn’t. Now, the latter group has asked a New Jersey federal court to issue a restraining order to block a J&J subsidiary from filing for bankruptcy.

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The Potential for AI in Drug Design

MedCity News

Despite misconceptions about its use, artificial intelligence (AI), through modalities such as machine learning models, is poised to enable companies to expedite the drug design process. The post The Potential for AI in Drug Design appeared first on MedCity News.

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How Healthcare Leaders Are Reacting to the Supreme Court Preserving Abortion Pill Access

MedCity News

The Supreme Court sided with the FDA and unanimously ruled to uphold access to abortion pill mifepristone. The decision quells worries about what it might look like if the Supreme Court sided with the physician group — many healthcare leaders feared that if the top court could overturn mifepristone’s FDA approval, the same could happen to other drugs.

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Investment firm Great Point Partners scoops up German CDMO Lyocontract

Fierce Pharma

On a mission to debut life science companies on the global stage, healthcare investment firm Great Point Partners is making a play for private German contract development and manufacturing organiza | On a mission to debut life science companies on the global stage, healthcare investment firm Great Point Partners is making a play for private German contract development and manufacturing organization Lyocontract.

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Flagship, ProFound set off on obesity drug hunt for Pfizer

pharmaphorum

Pfizer has had some setbacks in its efforts to develop drugs for the lucrative obesity market, but could see its pipeline swell thanks to its $7 billion partnership with venture capital firm Flagship Pioneering. Flagship has just announced that it will work with biotech startup ProFound Therapeutics to find first-in-class protein therapeutics with potential in obesity, confirming this is the first programme in the alliance with Pfizer signed last July.

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Haemophilia drug could reduce treated bleeding episodes

European Pharmaceutical Review

A next-generation FVIIIa mimetic bispecific antibody has demonstrated statistically significant and superior reduction of treated bleeding episodes in haemophilia A, new headline data from a pivotal Phase IIIa trial show. Mim8 was evaluated in participants aged 12 years or older as both a once-weekly and once-monthly treatment versus no prophylaxis treatment and prior coagulation factor prophylaxis treatment.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Mahana’s DTx for IBS gets permanent reimbursement in Germany

pharmaphorum

Mahana Therapeutics has granted permanent reimbursement in Germany for its Cara Care digital therapeutic for irritable bowel syndrome

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AmeriHealth Caritas Exec: Worst Thing Insurers Can Do Is Be Paralyzed by AI Challenges

MedCity News

Payers face numerous challenges when it comes to AI, but that doesn’t mean they shouldn’t use the technology, according to Neil Gomes, senior vice president and chief digital officer of AmeriHealth Caritas. The post AmeriHealth Caritas Exec: Worst Thing Insurers Can Do Is Be Paralyzed by AI Challenges appeared first on MedCity News.

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SCOTUS rejects challenge to abortion pill mifepristone

pharmaphorum

SCOTUS rejects lawsuit challenging the FDA's regulation of abortion pill mifepristone, in a setback for the anti-abortion lobby

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Being an Ally Isn’t Just for Pride Month

ALULA

Every June, companies plaster rainbow flags across their logos in a show of LGBTQIA+ "allyship" for Pride Month. But true allyship isn't just a celebration - it's an ongoing commitment to supporting LGBTQIA+ individuals every day.

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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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WuXi buoyed as BIOSECURE Act is left out of US spending bill

pharmaphorum

Shares in WuXi Biologics and WuXi AppTec rose after the BIOSECURE Act was omitted from legislation due to be followed up in congress.

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FDA approves AstraZeneca’s Farxiga for paediatric T2D patients

Pharmaceutical Technology

The US FDA has granted approval for AstraZeneca's Farxiga for paediatric patients aged 10 years and above with type-2 diabetes.

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Future of Pfizer’s Duchenne gene therapy in doubt

pharmaphorum

Pfizer is “evaluating next steps” for its Duchenne muscular dystrophy (DMD) gene candidate after it failed a phase 3 trial

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Alzheon secures $100m to advance Alzheimer’s disease treatment

Pharmaceutical Technology

Alzheon has secured $100m in a Series E funding round to develop and commercialise ALZ-801 for Alzheimer's disease (AD).

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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Global medicines and the future outlook

pharmaphorum

In a conversation held earlier in the year as the four-year anniversary of the pandemic approached, the pharmaphorum podcast had as guest Murray Aitken, executive director of the IQVIA Institute for Human Data Science, to discuss the state of global medicines and the future outlook when it comes to research and development, and usage and spending appetites.

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LifeArc funds new projects to address and prevent global threat of dengue fever

PharmaTimes

The mosquito-borne viral disease is responsible for an estimated 390 million cases globally

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How Covered Entities and Pharma Companies Can Better Collaborate on Drug Discount Programs

MedCity News

Three things drug manufacturers and CEs can do to build trust and enable effective collaboration The post How Covered Entities and Pharma Companies Can Better Collaborate on Drug Discount Programs appeared first on MedCity News.

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Streamlining Aseptic Processes through Automation

PharmaTech

Leveraging automation advancements can help improve efficiency in aseptic processing and speed up commercialization of critical drug products.

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Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research

Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud

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Sysmex receives Longitude Prize on AMR for antimicrobial testing system

PharmaTimes

AMR is designated as one of the top ten global public health threats facing humanity by WHO

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How to increase specialist engagement in accountable care organizations

Clarify Health

Medicare is a pioneer in advancing value-based care, specifically its use of Accountable Care Organization (ACO) frameworks. Alternative Payment Models (APMs) are payment approaches that incentivize quality and value in healthcare delivery by rewarding providers for cost-efficient care and improved patient outcomes. Progressive APMs incentivize ACOs to deliver the best possible care, especially for patients with complex conditions that require multiple costly treatments—which describes the major

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Revamp your MLR review with these 7 data-driven strategies

Vodori

In today's fast-paced marketing landscape, ensuring the efficiency and effectiveness of promotional materials is more crucial than ever. The Medical, Legal, and Regulatory (MLR) review process plays a vital role in this, acting as the gatekeeper for compliance and quality.

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AbbVie Presents New Data from Antibody-Drug Conjugates Across Multiple Indications at ASCO 2024

PharmExec

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses data presented at ASCO from ABBV-400 and ABBV-706 in multiple studies.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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How to improve MLR team collaboration amidst changes

Vodori

In the dynamic world of medical, legal, and regulatory (MLR) reviews, change is a constant. Teams frequently evolve, with new members joining, processes being updated, and goals shifting to align with the latest regulatory requirements and market demands. In such a fluid environment, effective collaboration becomes more critical than ever. Ensuring that your MLR team can adapt seamlessly to these changes is essential for maintaining efficiency, compliance, and high-quality outputs.

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Craig Tooman: Silencing the Distractions

PharmExec

Craig Tooman, CEO of Silence Therapeutics, on steering through the rough biotech funding and investment waters post-pandemic—and how a retooled organizational approach has the company in a stronger position today in the growing field of gene silencing through siRNA drugs.

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Despite detractors, Lilly’s adcomm win shows amyloid and tau will likely dominate Alzheimer’s — for now

PharmaVoice

Lilly is the latest to approach the coveted Alzheimer’s approval, but a slate of drugmakers are lining up clinical trials in amyloid and tau to take a shot at improving patient outcomes.

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Biopharma Leadership in Times of Transition

PharmExec

How pharma and biotech leaders can navigate today’s challenging landscape—focusing on revamped approaches in business operations, talent strategy and development, and more to meet the evolving demands.

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.